Hutchison China MediTech Limited (“Chi-Med”) (AIM/Nasdaq:HCM) presented pre-clinical data for fruquintinib and sulfatinib at the American Association for Cancer Research (“AACR”) Annual Meeting 2017, held in Washington, D.C., USA from April 1 to 5, 2017. Fruquintinib and sulfatinib are both being evaluated in Phase III clinical trials for various cancers.【閱讀原文】

Medivir posts lymphoma data, guns to get into phase 3

Medivir has posted data from a phase 2 trial of its histone deacetylase (HDAC) inhibitor in patients with cutaneous T-cell lymphoma (CTCL). One regimen of the treatment triggered responses in 40% of subjects, a finding that has encouraged the Swedish pharmaceutical company to push ahead with plans to start a phase 3 trial later this year.【閱讀原文】

Kura Oncology Doses First Patient in Phase 1 Trial of ERK Inhibitor KO-947

y, today announced that the first patient has been dosed in its Phase 1 clinical trial of KO-947, a potent and selective small molecule inhibitor of extracellular-signal-regulated kinases 1 and 2 (ERK1/2).【閱讀原文】

Biomarin’s rare disease approval and Probiodrug’s Alzheimer’s play

Welcome to your weekly digest of approaching regulatory and clinical readouts. Biomarin is looking for approval of its enzyme-replacement therapy Brineura in a rare condition called CLN2 disease, which leads to developmental deterioration in children. The US regulators will decide by April 27, but could announce an advisory meeting in the coming weeks.【閱讀原文】

FDA allows 23andMe to sell DTC genetic tests for 10 conditions

It’s been a long road for 23andMe, but not without reward. The FDA authorized the marketing of the company’s genetic health risk tests, the first tests OK’d by the agency that provide this information directly to consumers.【閱讀原文】

PET imaging measures PARP-1 in ovarian cancer patients

University of Pennsylvania scientists have devised a new imaging test to measure the amount of PARP-1 enzyme in ovarian cancer patients, which could help identify which patients would benefit most from treatments that target PARP-1.【閱讀原文】

Blood vessels built from skin cells help repair damaged hearts in mice

One of the primary goals of regenerative medicine is to find new ways of fighting heart disease, the leading cause of death around the world. Coaxing stem cells to become heart cells is one popular technique, but scientists at the University of Illinois at Chicago believe they might have found an even better way to repair damaged hearts—by transforming skin cells into blood vessels.
【閱讀原文】

Merck's Diabetes Franchise And The Perils In Proving A Drug's Medical Value

Merck’s Januvia (sitagliptin) and its metformin combination, Janumet, are important drugs for type 2 diabetes patients as well as for Merck’s bottom line. These drugs, which are DPP-4 inhibitors, account for more than $6 billion in sales for this drug giant. Given the growing incidence of obesity and type 2 diabetes in the world, this franchise is poised for continued growth.【閱讀原文】

Ocrelizumab- FDA Approved Treatment of MS

Ocrelizumab (OCREVUS?), an anti-B cell therapy has been approved by the FDA as of March 28th 2017, for the treatment of Relapsing-remitting multiple sclerosis (RRMS) and primary progressive multiple sclerosis (PPMS). This treatment is highly effective for RRMS, based on the data from the Phase III clinical studies. It is also the first drug approved for use in PPMS. At IMSMP/TISCH we have used Rituximab, a similar anti-B cell treatment, for the past 16 years with great success. It is because of the effectiveness of this therapy (Rituximab) we were able to persuade Medicare to cover the costs of this therapy in New York.【閱讀原文】

Merck's Diabetes Franchise And The Perils In Proving A Drug's Medical Value

Merck’s Januvia (sitagliptin) and its metformin combination, Janumet, are important drugs for type 2 diabetes patients as well as for Merck’s bottom line. These drugs, which are DPP-4 inhibitors, account for more than $6 billion in sales for this drug giant. Given the growing incidence of obesity and type 2 diabetes in the world, this franchise is poised for continued growth.【閱讀原文】

Merck Receives Complete Response Letter from the U.S. FDA for TECOS Study with Sitagliptin

Merck (NYSE:MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter regarding Merck’s Supplemental New Drug Applications for JANUVIA? (sitagliptin), JANUMET? (sitagliptin and metformin HCl) and JANUMET XR? (sitagliptin and metformin HCl extended-release). With these applications, Merck is seeking to include data from TECOS (Trial Evaluating Cardiovascular Outcomes with Sitagliptin) in the prescribing information of sitagliptin-containing medicines. Merck is reviewing the letter and will discuss next steps with the FDA. 【閱讀原文】

Like me, you may never have heard about Lubris BioPharma before today. But Novartis has. And that is by far more important.

Today the pharma giant reached out and took an option on a recombinant lubricant Lubris drew from nature’s medicine chest and applied for dry eye disease. There are no terms, no big biobucks on the record. But the deal marks a definite transition phase for little Lubris as it shifts its focus on the same therapy to a different set of ailments in mind, while Novartis eyes an addition to its late-stage pipeline.【閱讀原文】

The stellar antibody shop at Regeneron wins a ‘breakthrough’ tag for its next big PhIII drug

Regeneron $REGN has one of the best track records in the business when it comes to building amazingly effective antibodies. It did it with Dupixent for eczema and Praluent for cholesterol. And now one of its Phase II stars just earned a breakthrough tag from the FDA, which puts it on the inside track at the agency as it goes into pivotal studies for slashing triglyceride.【閱讀原文】

Nanoparticle-delivered combo boosts cancer immunotherapy

Cancer cells often evade immune attacks by displaying proteins that disguise them as normal. Checkpoint inhibitors block these proteins and reveal tumor cells to the immune system, but they don't work for many patients. Now University of North Carolina researchers have found that using nanoparticles can boost the efficacy of cancer immunotherapy treatments.【閱讀原文】

Innocrin Pharmaceuticals Appoints Fred Eshelman, PharmD as CEO and is Granted Fast Track Designation by FDA for Seviteronel Treatment of Women with Triple-negative Breast Cancer and Women or Men with Estrogen Receptor-positive Breast Cancer

Innocrin Pharmaceuticals, Inc., a clinical-stage company focused on the development of the oral, selective CYP17-lyase/androgen receptor (AR) inhibitor, seviteronel, announced today the appointment of Fred Eshelman, PharmD, of Eshelman Ventures? as Chief Executive Officer, effective March 15, 2017.【閱讀原文】

Mena Expression is Associated with Paclitaxel-mediated Increase in Cancer Cell Dissemination

MetaStat, Inc., a precommercial biotechnology company, developing diagnostics for risk of cancer metastasis and anti-metastatic therapies, today announced the presentation of supportive data for the role of Mena protein isoforms in tumor dissemination at the 2017 American Association of Cancer Research Annual meeting in Washington D.C. The poster was presented on April 4, 2017 by the company’s collaboration partner, Albert Einstein College of Medicine.【閱讀原文】

Mid-cap biopharma stocks pick up in the first quarter

After a terrible performance over the past year mid-cap biopharma stocks picked up in the first quarter of 2017, echoing gains among larger drug makers. US companies in particular showed a return to health, with three featuring in the biggest risers of the quarter: Vertex, Incyte and Jazz.【閱讀原文】

Spark hemophilia B gene therapy clears another early test

Spark Therapeutics has posted fresh data from a phase 1/2 trial of its hemophilia B gene therapy, SPK-9001. The data suggest Spark’s tapering courses of steroids successfully arrested the factor IX (FIX) declines seen in two patients who appeared to have an immune reaction to the Spark100 vector capsid.【閱讀原文】

BerGenBio completes IPO, raising $49M for cancer trials

BerGenBio has completed (sub. req.) its IPO in Oslo. The Norwegian cancer biotech hit its target price and sold out the overallotment, resulting in it pulling in NOK 425 million ($49 million) to invest in a clutch of phase 2 trials of Axl inhibitor BGB324.【閱讀原文】

Sosei Group Corporation (“Sosei”; TSE Mothers Index:4565) reported that its subsidiary, Heptares Therapeutics (“Heptares”) has announced that it has achieved an important milestone in its immuno-oncology collaboration with AstraZeneca, which is focused on the development of AZD4635 (HTL-1071) as a potential new treatment for a range of cancers. As a result, Heptares has been notified today that the achievement has triggered a US$12 million payment from AstraZeneca.【閱讀原文】

Parkinson’s approach to cancer needs more work

The concept was intriguing: take an adenosine A2A antagonist, a mechanism mainly associated with Parkinson’s disease, and test it in oncology. However, interim data Corvus presented at the AACR meeting yesterday suggest that at best this approach still needs more work.【閱讀原文】

OncoMed Highlights New Data Presented at the American Association for Cancer Research Annual Meeting 2017

OncoMed Pharmaceuticals Inc. (NASDAQ:OMED), a clinical-stage biopharmaceutical company focused on discovering and developing novel anti-cancer stem cell and immuno-oncology therapeutics, highlighted data presented during the American Association for Cancer Research (AACR) Annual Meeting related to its clinical trial of tarextumab (anti-Notch2/3, OMP-59R5) and preclinical studies of rosmantuzumab (anti-RSPO3, OMP-131R10) and GITRL-Fc trimer (OMP-OMP-336B11). 【閱讀原文】

Oncolytics Biotech Inc.'s REOLYSIN More than Doubles Overall Survival in Patients with Mutated p53 Metastatic Breast Cancer

Oncolytics Biotech? Inc. (Oncolytics or the Company) (TSX:ONC) (OTCQX:ONCYF) today announced data demonstrating a statistically significant (p=0.03) overall survival (OS) benefit for patients with mutated p53 metastatic breast cancer, when treated with REOLYSIN?, an immuno-oncology viral agent, in combination with paclitaxel. Results from IND 213, an open-label, randomized, phase 2 study were presented at the Annual Meeting of the American Association of Cancer Research (AACR), April 1-5, 2017 in Washington, D.C. 【閱讀原文】

Novel IDO1 Inhibitor Shows Promise in Early Phase Study

The IDO1 inhibitor BMS-986205 had "best-in-class" activity as demonstrated by kynurenine reductions and IDO1 inhibition for patients with advanced malignancies in a phase I/IIa study, according to lead investigator Lillian L. Siu, MD, at the 2017 AACR Annual Meeting.1

"BMS-986205 is fulfilling the criteria for being a best in class IDO1 inhibitor, it is potent and selective," said Siu, senior medical oncologist at Princess Margaret Cancer Centre. "The once-daily dosing exceeds the IC90 target concentrations. We see kynurenine reduction that is greater than reported with previous IDO1 inhibitors."【閱讀原文】

Immunomedics says IMMU-132 hits targets in small cell lung cancer

Embattled Immunomedics has some good news to report on its lead antibody-drug conjugate (ADC) program with a preliminary study showing IMMU-132 has activity in small cell lung cancer (SCLC).

The data could add another possible indication for IMMU-132, which according to Immunomedics is on course for an accelerated approval later this year for triple-negative breast cancer but will need to be backed up by further studies.【閱讀原文】

DelMar Pharmaceuticals Provides VAL-083 Updates from the Ongoing American Association for Cancer Research (AACR) Annual Meeting

DelMar Pharmaceuticals (Nasdaq: DMPI) ("DelMar" and the "Company"), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, today announced that it has presented three abstracts at the American Association for Cancer Research (AACR) Annual Meeting. These abstracts are all focused on pre-clinical research to elucidate the mechanism of action (MOA) of DelMar Pharmaceuticals' lead anti-cancer product candidate, VAL-083 (dianhydrogalactitol), a "first-in-class" small-molecule, DNA-targeting, chemotherapeutic agent. 【閱讀原文】

Long-term melanoma data open another Opdivo weak spot

Bristol-Myers Squibb may have celebrated an overall survival win for Opdivo plus Yervoy in melanoma in two-year follow-up data from Checkmate-067, but an overlooked aspect is the study’s failure to provide convincing evidence that the two-drug combination beats single-agent Opdivo.【閱讀原文】

Allergan To Test Botox For Depression Despite Mixed Results

Allergan says it plans to advance its iconic wrinkle treatment, Botox, to late-stage clinical trials, the last step of study before a medicine is approved, based on data from a 258-patient study it is releasing this afternoon.【閱讀原文】

Bristol-Myers Squibb has unveiled “unprecedented” long-term survival data for its cancer immunotherapy Opdivo, showing that nearly one in six patients with previously-treated, advanced non-small cell lung cancer (NSCLC) were still alive at five years.

In the Phase Ib single arm trial, which primarily looked at safety and tolerability across different doses, 16 percent of 129 patients reached the five-year survival mark when, typically, the five-year survival rate for advanced lung cancer is just 1 percent.【閱讀全文】

Teva’s Austedo wins US nod for Huntington’s patients

US regulators have green-lighted Teva Pharmaceutical Industries’ Austedo for the treatment of chorea associated with Huntington’s disease (HD), offering patients the first new treatment option in nearly a decade.

A rare and fatal neurodegenerative disorder, HD affects more than 35,000 people in the US. Chorea - involuntary, random and sudden, twisting and/or writhing movements - is a key physical manifestation of this disease occurring in around 90 percent of patients.【閱讀全文】

Interim Phase 2 Data Demonstrate Robust Response Rate with Indoximod in Combination with Keytruda (pembrolizumab) for Patients with Advanced Melanoma at AACR Plenary

NewLink Genetics Corporation (NASDAQ:NLNK), a biopharmaceutical company focused on bringing novel immuno-oncology therapies to patients with cancer, today reported interim results from NLG2103, a Phase 2 study evaluating its IDO pathway inhibitor, indoximod, in combination with checkpoint inhibitors for the treatment of patients with advanced melanoma.【閱讀全文】

AACR – Newlink slipstreams behind Incyte

After Incyte moved into immuno-oncology’s fast track and had analysts hailing the IDO pathway as the next big thing, data unveiled today at the AACR meeting suggested that Newlink Genetics was at least going to try to give its rival a run for its money.【閱讀全文】

CBT Pharmaceuticals Presents Data Demonstrating Anti-Tumor Activity of its Highly Specific c-MET Inhibitor, Bozitinib (CBT-101), at AACR

CBT Pharmaceuticals, Inc. (CBT), a life sciences company focused on developing innovative oncology therapeutics, presented preclinical data on CBT-101 (bozitinib, PLB-1001, CBI-3103), a highly specific small molecule inhibitor of c-MET receptor tyrosine kinase, demonstrating its selectivity, safety, and efficacy in suppressing tumor growth in lung, gastric, hepatic and pancreatic human primary tumor models. The data were presented in a poster at the American Association for Cancer Research Annual Meeting (AACR) being held from April 1 – 5, 2017 in Washington, D.C.【閱讀全文】

Onconova Therapeutics stock surges 18% on positive pre-clinical data for cancer drugs

Onconova Therapeutics Inc. ONTX, +6.98% stock surged as much as 18% in premarket trade Tuesday after the company said its two cancer drugs had positive pre-clinical results. Onconova shares were valued at $3.01 as of Monday's close. One drug, ON 123300, was found in a model to work as well as Pfizer Inc.'s PFE, +0.29% Ibrance, and could lead to less infection susceptibility, the company said. The other drug, ON 150030, showed promising results in relapsed and refractory Acute Myeloid Leukemia, the company said. Both results were presented on Monday at the American Association for Cancer Research's annual meeting. Onconova shares have surged 30.3% over the last three months, compared with a 3.9% rise in the S&P 500 SPX, +0.06%【閱讀全文】

Paratek shares surge after antibiotic phase 3 hits endpoints

Shares in Paratek Pharmaceuticals jumped 35% in premarket trading on the back of data from a phase 3 trial of its antibiotic candidate omadacycline. The clinical trial met its primary endpoint of noninferiority to moxifloxacin, teeing Paratek up to file for FDA approval in the first quarter of next year.【閱讀全文】

Amgen Offers A Money-Back Guarantee For Its Cholesterol Drug Repatha

The high cost of drugs continues to dominate the news in the biopharmaceutical industry. Recent data for new cancer drugs that upregulate the immune system to fight melanoma, lung cancer, etc. are very promising. Yet, stories on these wonder drugs quickly dive into their costs–justifiably so. For example, Bristol-Myers Squibb’s combination of Opdivo and Yervoy can cost up to $21,000–a month! But new cancer therapies are not unique with respect to pricing. Similar stories can be found with drugs to treat rare diseases, hepatitis C, multiple sclerosis, etc.【閱讀全文】

In a relief to investors, the combination of Opdivo and Yervoy extends survival in melanoma. More data being released this afternoon could yield answers to the question of why Opdivo did not show a benefit in previously untreated lung cancer.【閱讀原文】

Astellas to buy GPCR specialist Ogeda for €500M upfront

Astellas has struck a deal to buy Ogeda for €500 million ($534 million) upfront. The takeover will give Astellas a potential nonhormonal treatment for menopausal hot flushes that cleared a phase 2a trial earlier this year.

Gosselies, Belgium-based Ogeda has accepted an offer of €500 million upfront and up to €300 million in clinical and regulatory milestones from Astellas. Ogeda, which until October was known as Euroscreen, landed the buyout bid on the strength of clinical data generated by its lead asset, NK3 receptor antagonist fezolinetant, also known as ESN364.【閱讀全文】

Kite Presents Ongoing Response Rate in Plenary Session from its Pivotal CAR-T Trial of Axicabtagene Ciloleucel in Patients with Aggressive Non-Hodgkin Lymphoma at the 2017 American Association of Cancer Research Annual Meeting

Kite Pharma, Inc., (Nasdaq:KITE) today announced two plenary presentations of positive data from the primary analysis of ZUMA-1 for its lead CAR-T candidate, axicabtagene ciloleucel, in patients with refractory aggressive B-cell non-Hodgkin lymphoma (NHL) at the 2017 American Association of Cancer Research Annual Meeting in Washington, D.C. Both presentations were given by Frederick L. Locke, M.D., the ZUMA-1 Co-Lead Investigator, and Director of Research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, Florida.【閱讀全文】

Patients with lung cancers responsive to immunotherapy drug beat standard odds of survival

More than seven years after the start of one of the first clinical trials of the immunotherapy drug nivolumab, researchers at the Johns Hopkins Kimmel Cancer Center report that the five-year survival estimate for a limited subset of people with advanced nonsmall cell lung cancer taking the drug is 16 percent, compared with a historical survival rate for that group of 1 to 4 percent.【閱讀全文】

BeiGene Presents Phase IB Data on RAF Dimer Inhibitor BGB-283 at the 2017 American Association for Cancer Research Annual Meeting

BeiGene, Ltd. (NASDAQ:BGNE), a clinical-stage biopharmaceutical company developing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, today presented data from the Phase IB dose expansion study of RAF dimer inhibitor BGB-283 in patients with B-RAF or K-RAS/N-RAS mutated solid tumors in an oral presentation during a Clinical Trials Plenary Session at the 2017 American Association for Cancer Research (AACR) Annual Meeting in Washington, DC. BGB-283 is a novel inhibitor of RAF, in both its monomeric and dimeric forms, and has demonstrated activity in both B-RAF and K-RAS-mutated tumors in preclinical studies and in the Phase IA dose escalation portion of this Phase I study. In the Phase IB portion, BGB-283 has generally been well-tolerated at a dose of 30 mg once a day (QD) and has continued to show antitumor activity not only in subjects with B-RAF V600-mutated solid tumors, but also in subjects with K-RAS-mutated solid tumors.【閱讀全文】

Bristol-Myers Squibb and Incyte to Advance the Combination of Opdivo (nivolumab) and Epacadostat into First-line Registrational Trials

Bristol-Myers Squibb Company (NYSE:BMY) and Incyte Corporation (Nasdaq:INCY) today announced the companies have agreed to advance their clinical development program evaluating the combination of epacadostat, Incyte’s investigational oral selective IDO1 enzyme inhibitor, with Opdivo (nivolumab), Bristol-Myers Squibb’s PD-1 immune checkpoint inhibitor, into phase 3 registrational studies in first-line non-small cell lung cancer across the spectrum of PD-L1 expression and first-line head and neck cancer.【閱讀全文】

Intrexon, ZIOPHARM and Merck KGaA, Darmstadt, Germany Advancing Next-Generation Non-Viral CAR-T Platform Empowered by Membrane-Bound IL-15 Under RheoSwitch Therapeutic System Control

Intrexon Corporation (NYSE:XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, ZIOPHARM Oncology (NASDAQ:ZIOP), a biopharmaceutical company focused on new immunotherapies, and Merck KGaA, Darmstadt, Germany, a leading science and technology company, today announced an update on the development of next-generation chimeric antigen receptor T cell (CAR-T) therapy for cancer as part of their strategic collaboration and license agreement.【閱讀全文】

Jazz Pharmaceuticals Completes Rolling Submission of New Drug Application for Vyxeos (CPX-351), an Investigational Treatment for Acute Myeloid Leukemia

Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced the completion on March 31, 2017 of a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of Vyxeos? (cytarabine and daunorubicin) liposome for injection, an investigational treatment for acute myeloid leukemia (AML), a rapidly progressing and life-threatening blood cancer. 1 The company has requested a priority review for the Vyxeos NDA, which, if granted, would accelerate the expected timing of the FDA's review.【閱讀全文】

FDA hands over full approval to AZ’ Tagrisso

US regulators have issued a full approval for AstraZeneca’s lung cancer drug Tagrisso on the back of a strong progression-free survival (PFS) benefit observed in a late-stage trial.

The drug had already picked up an accelerated approval for the treatment of patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC) whose disease has progressed on or after an EGFR tyrosine kinase inhibitor (TKI) therapy.【閱讀全文】

Patent rulings knock Acorda and Forward

It was a bloody Friday for two biotechs that ended up on the wrong side of US patent court decisions. Verdicts handed down by the Patent Trial and Appeal Board (PTAB) mean that Acorda Therapeutics is on track to see generic competition for its multiple sclerosis drug Ampyra as early as next year. Separately, Forward Pharma could miss out on a substantial royalty stream owing to a negative decision on its claims on Biogen’s multiple sclerosis pill Tecfidera.【閱讀全文】

Sucampo bags rare disease player Vtesse in $200M buyout agreement

Just a little more than two years after it was launched, the single-asset biotech Vtesse is being bought out by Sucampo for $200 million up front.

Vtesse spun out of the incubator Cydan with a Series A that eventually swelled to $42 million. The biotech used the cash to advance a human study into a new drug — which picked up a breakthrough drug designation — for rare cases of Niemann-Pick type C1 disease. And Rockville, MD-based Sucampo pledged backers a royalty stream on any marketing revenue.【閱讀全文】

Lilly, Pfizer, et al. back Sanofi and Regeneron in PCSK9 patent appeal. AbbVie doesn't

Big Pharma is enthralled with the Amgen vs. Sanofi patent fight, and for good reason: Its outcome could affect their in-development meds—and farther up the pipeline, their decisions to advance candidates at all.

The reason: Amgen, which makes the cholesterol-fighting Repatha, is asserting an antibody patent that, if upheld, could give the California biotech the power to push Sanofi and Regeneron’s rival PCSK9 drug off the market. And it’s a type of patent that has the potential to allow Amgen—and other drugmakers in other fields—to stake out a claim to an entire class of therapies, or so Sanofi’s legal team said in a recent phone conference with reporters.【閱讀全文】

Paratek shares shoot up as positive PhIII and a shot at FDA OK for its antibiotic fuel a market rally

Shares of Paratek shot up 30% $PRTK in after-market trading on Monday as investors reacted to the Boston biotech’s proclamation of a clean sweep for its pivotal test of its new antibiotic omadacycline. Execs said that their broad spectrum antibiotic hit its primary and secondary endpoints for the FDA as well as the co-primaries needed for the EMA in their second positive late-stage trial.【閱讀全文】

#AACR17 roundup: Bristol-Myers, Merck KGaA and Pfizer in the spotlight, marking progress with checkpoints

Now that the first wave of PD-1 checkpoint inhibitors have been in use for awhile, we’re starting to see just how durable they can be for some patients. In an update on Opdivo at AACR, Bristol-Myers Squibb reports that they tracked a 5-year survival rate of 16% for patients with advanced cases of non-small cell lung cancer.

Based on historical data, Bristol-Myers says that patients in that category once could only expect a survival rate of less than 5%.【閱讀全文】

Merrimack Pharmaceuticals, Inc. (NASDAQ: MACK) ("Merrimack") announced that, at its Special Meeting of Stockholders held today, its stockholders voted to approve the asset sale with Ipsen S.A. under which Merrimack will:

• Sell to Ipsen its first commercial product ONIVYDE?, including U.S. commercialization rights and its licensing agreement with Shire plc; and
• Sell to Ipsen its generic version of doxorubicin hydrochloride (HCI) liposome injection ("generic DOXIL?") marketed in the United States as DOXIL? and advanced under a development, license and supply agreement with Actavis LLC.【閱讀全文】

Incyte and Merck Provide Additional Details on Previously Announced Collaboration Investigating Epacadostat and KEYTRUDA (pembrolizumab)

Incyte Corporation (Nasdaq:INCY) and Merck (NYSE:MRK), known as MSD outside the United States and Canada, today announced additional details of their clinical development program investigating the combination of epacadostat, Incyte’s investigational oral selective IDO1 enzyme inhibitor, with KEYTRUDA? (pembrolizumab), Merck’s anti-PD-1 therapy, in patients across five tumor types: metastatic melanoma, non-small cell lung cancer (NSCLC), bladder cancer, renal cell carcinoma (RCC), and squamous cell carcinoma of the head and neck (SCCHN).【閱讀全文】

Upcoming events – Ingrezza's approval and data for Shire's new rare disease asset

Welcome to your weekly digest of approaching regulatory and clinical readouts. Neurocrine expects a decision on its tardive dyskinesia candidate Ingrezza by April 11, with speculation that it will be approved fuelled by the cancellation of an advisory committee meeting. However, Teva’s competitor, which is based on the same compound as Ingrezza, looms.【閱讀全文】

UPDATED: Kite Pharma hits its Q1 deadline on filing a BLA for its pioneering CAR-T

With the clock ticking down the final hours of Q1, Kite Pharmaceuticals $KITE has kept its pledge to file an application for its pioneering CAR-T therapy in the first quarter. The move puts Kite one step behind Novartis $NVS, which two days ago announced that it had the FDA’s commitment for a speedy review of its own CAR-T.【閱讀全文】

Halozyme dips after pancreatic cancer trial halted for futility

Investors have driven down shares in Halozyme Therapeutics following news investigators halted a phase 1b/2 pancreatic cancer trial for futility. The failure of the investigator-initiated trial to show signs of efficacy raised doubts about the ability of PEGPH20 to deliver positive data in phase 3, despite differences in the designs of the studies.【閱讀全文】

Takeda cuts 180 Ariad workers as it grabs synergies from $5.2B buyout

Takeda was unambiguous about the importance of cost synergies as a driver of its $5.2 billion buyout of Ariad, mentioning it six times when it announced the deal in January. Those now come into play as the Japanese drugmaker trims about 180 jobs in the Boston area.【閱讀全文】

Hepatocellular carcinoma (HCC), a cancer associated with underlying liver disease and cirrhosis that often only becomes symptomatic when it is very advanced, is the second leading cause of cancer deaths around the world, and yet it has no effective treatment.

As with other conditions without treatments, the data that scientists need to understand and treat the disease may be sitting in plain view in databases that have barely been analyzed, says Atul Butte, MD, PhD, director of the Institute for Computational Health Sciences at UC San Francisco.【閱讀全文】

Third Rock’s Tango Aims For Drugs That Turn Cancer Against Itself

As the understanding of cancer advances, scientists are discovering built-in features of the disease’s own biology that can be tapped to halt its growth. Biotech startup Tango Therapeutics believes it can build on this concept to develop new cancer drugs, and it is now unveiling its approach, backed by $55 million in financing.【閱讀全文】

Incyte cash boost not enough to stave off major Agenus cuts

After Incyte and Agenus retooled their R&D pact last month, which gave the biotech access to some quick cash, it appeared that something was amiss; today, Agenus said it will be closing its Basel site; “consolidate” key functions to its Cambridge, U.K., and Lexington, Massachusetts, facilities, and “phase out” around 50 staffers from across its biz by the fall.【閱讀全文】

LAM Therapeutics Announces Close of $58M Series C Financing and New Clinical Program in Cancer

LAM Therapeutics, a 4Catalyzer company that develops drugs for cancer and rare diseases, announced that it has closed $58M in Series C financing. 4Catalyzer is Connecticut's premier biotechnology incubator and has raised over a quarter billion dollars to support its current class of startups.【閱讀全文】

Halozyme's PEGPH20 shows no incremental survival benefit in pancreatic cancer study; shares down 11% premarket

Halozyme (NASDAQ:HALO) slumps 11% premarket on average volume in response to its announcement that a Phase 1b/2 clinical trial assessing PEGPH20, plus modified FOLFIRINOX chemotherapy, in treatment-naive patients with metastatic pancreatic cancer would be unlikely to show an increase in overall survival (the primary endpoint) compared to FOLIFIRINOX alone. The assessment was made by an independent network of researchers called SWOG.【閱讀全文】

Orphan status for GW’s rare epilepsy drug

London, UK-based GW Pharmaceuticals’ cannabinoid Epidiolex has been awarded Orphan status by European regulators as a treatment for Lennox-Gastaut Syndrome (LGS), a rare and severe form of childhood-onset epilepsy.【閱讀全文】

ALK Inhibitors in ALK+ Non-Small Cell Lung Cancer

In an indirect comparison, brigatinib appeared to have greater efficacy than alectinib or ceritinib in ALK+ non-small cell lung cancer (NSCLC), according to a poster presented at the 2017 Hematology/Oncology Pharmacy Association Annual Conference.【閱讀全文】

Ignyta Announces Peer-Reviewed Publication of Activity of a TRK Inhibitor in a Primary Brain Tumor: Successful Treatment of Glioneuronal Tumor With Pan-TRK, CNS-Active Inhibitor Entrectinib Published in Precision Oncology

Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, today announced data demonstrating the successful treatment with entrectinib – Ignyta’s investigational, orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor TRK, ROS1 or ALK fusions – of a patient with a primary brain tumor harboring an NTRK1 fusion. The study, exploring genetic alterations associated with glioneuronal tumors, was led by researchers at Massachusetts General Hospital and was published in Precision Oncology. 【閱讀全文】

Merck KGaA disses Ocrevus-Rebif price comparison as 'misleading and oversimplified' | FiercePharma

Roche surprised industry watchers on Tuesday when it placed a $65,000 list price on multiple sclerosis blockbuster-to-be Ocrevus, sparking comparisons with a more expensive—and less effective, according to trial data—Merck KGaA rival. But it's a comparison the German drugmaker doesn't much care for.【閱讀全文】

Merck KGaA disses Ocrevus-Rebif price comparison as 'misleading and oversimplified'

Roche surprised industry watchers on Tuesday when it placed a $65,000 list price on multiple sclerosis blockbuster-to-be Ocrevus, sparking comparisons with a more expensive—and less effective, according to trial data—Merck KGaA rival. But it's a comparison the German drugmaker doesn't much care for.【閱讀全文】

Fresh turmoil inside Novartis’ revamped CAR-T organization as top exec jumps ship
There’s more turmoil to report from inside Novartis’ CAR-T camp.
On Friday Karen Walker will leave a senior position in charge of CMC/manufacturing of CAR-Ts for Novartis in exchange for a new position she’s taking at Seattle Genetics, Endpoints News has learned.【閱讀全文】

Steve Burd (R), president and CEO, Safeway, Inc. and Scott Gottlieb (L), resident fellow at American Enterprise Institute participate in a Senate Health, Education, Labor and Pensions Committee hearing on Capitol Hill, June 11, 2009 in Washington, DC. (Credit: Mark Wilson/Getty Images)【閱讀全文】

Vertex investors toast birth of the son of Orkambi

A double pivotal trial success with tezacaftor, a novel CFTR corrector, has given Vertex a neat follow-on to its blockbuster cystic fibrosis combination Orkambi, and should help it capture a greater market share of this respiratory disease.

Of course neither study compared the tezacaftor combo head to head against Orkambi, but on an across-trial basis efficacy looks marginally better, with safety – Orkambi’s main stumbling block – much improved. Moreover, the new combination bodes well for Vertex’s plans to cement its hold on cystic fibrosis with a triple combo.【閱讀全文】

Roche prices Ocrevus for maximum disruption

Roche now has the first approved drug for both the relapsing and primary progressive forms of multiple sclerosis in the shape of Ocrevus. But the company is not taking any chances on price – at $65,000 it will cost less than existing MS therapies, giving the Swiss firm a good shot at penetrating the $20bn market.【閱讀全文】

Novartis gets a speedy review for its CAR-T pioneer as FDA lines up a marketing decision

Novartis $NVS has pulled into the inside track at the FDA with its CAR-T CTL019, picking up a priority review from the agency that will land a marketing decision for relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia.

The pioneering application comes as we wait for Kite to make its move at the agency after being forced to wait for 6-month data on its rival CAR-T. Kite $KITE has promised to finish its rolling application by the end of Q1, now just days away. And a spokesperson for Kite tells me now that the biotech is on track to wrap the submission by Friday.【閱讀全文】

CML treatment-free remission possible with nilotinib

The ENESTfreedom study of patients who stopped first-line nilotinib after achieving a sustained deep molecular response has shown that around half of chronic myeloid leukemia patients continue in remission.

This proportion is “clinically significant”, the investigators say, adding that, when considered alongside the results from the ENESTnd trial, which showed higher rates of deep responses and sustained deep responses with nilotinib versus imatinib, “more patients may become eligible to stop treatment and sustain remission following frontline nilotinib therapy than following imatinib therapy.”【閱讀全文】

Immunovaccine Announces Positive Interim Clinical Data from Ovarian Cancer Study of DPX-Survivac in Combination with Epacadostat

Immunovaccine Inc. ("Immunovaccine" or the "Company") (TSX:IMV) (OTCQX:IMMVF), a clinical stage vaccine and immunotherapy company, today announced the first interim data analysis from its ongoing Phase 1b clinical study of its novel T-cell activating immuno-oncology candidate, DPX-Survivac, in combination with epacadostat and low-dose cyclophosphamide. The analysis included the results of blood tests, tumor biopsies and CT scans to assess safety, disease progression and T-cell response for the first four evaluable patients in the trial.【閱讀全文】

New marker could break ALS drug development deadlock

Amyotrophic lateral sclerosis (ALS) doesn't have a cure, and the only drug approved to treat it extends life by just a few months. Now, a Mayo Clinic-led team has landed on a protein that could serve as a marker in the development of new therapies.

ALS affects motor neurons, which transmit messages from the brain or spinal cord to muscles or glands. As the disease progresses, patients lose the ability to move their muscles, which weaken and waste away, eventually leading to paralysis and death. While researchers have conducted more than 50 clinical trials in as many years, only the drug Riluzole, an incomplete solution, has earned FDA approval.【閱讀全文】

Ritter lactose intolerance phase 2b/3 hits endpoint—but only after it excludes 20% of patients from the analysis

Ritter Pharmaceuticals has posted phase 2b/3 lactose intolerance data it claims set it up to run a confirmatory late-stage study and file for approval. But the trial missed its primary endpoint, with Ritter only able to make the figures hit statistical significance by removing 20% of patients from its analysis.【閱讀全文】

Is Vertex's New CF Drug Unprecedented? Incremental? Expensive? Maybe All Three

Shares in Boston’s Vertex Pharmaceuticals are up 22% to $109 after the biotechnology company released data on an experimental combination pill to treat cystic fibrosis, a genetic disease that shortens patients' lives by affecting their lungs and digestive systems. That gives the biotech a market capitalization of $27 billion.【閱讀全文】

The U.S. Food and Drug Administration today approved Zejula (niraparib) for the maintenance treatment (intended to delay cancer growth) of adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer, whose tumors have completely or partially shrunk (complete or partial response, respectively) in response to platinum-based chemotherapy.【閱讀全文】

Six new medicines backed for EU approval

Six new medicines, including three orphan drugs for rare diseases, have been backed for European Union approval by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).

Orphan medicine Dinutuximab beta Apeiron (dinutuximab beta) has been put forward for a marketing authorisation under exceptional circumstances for the treatment of high risk forms of neuroblastoma, a rare cancer arising from neural crest cells, which are involved in the foetal development of the nervous system and other tissue.【閱讀全文】

Nivolumab, pembrolizumab exhibit different toxicity profiles as melanoma monotherapy

Nivolumab and pembrolizumab exhibited different toxicity profiles and appeared associated with different adverse event frequencies when used as monotherapy for melanoma, according to study results presented at HemOnc Today Melanoma and Cutaneous Malignancies.

An analysis of FDA–approved dosing revealed higher rates of diarrhea, nausea and vomiting among nivolumab-treated patients, and higher rates of myalgia and cough in pembrolizumab-treated patients.【閱讀全文】

Chinese drug approval boosts AstraZeneca's lung cancer hopes

AstraZeneca has won approval for its lung cancer pill Tagrisso in China, a key market for the potential blockbuster medicine. Tagrisso is designed to help cancer patients with certain genetic mutations that are very common in China and the regulatory green light boosts the British drugmaker's prospects in a key therapy area.【閱讀全文】

Pumped by an early glimpse of efficacy, Newron charts a pivotal course for schizophrenia drug

Just a few days after Newron finally landed an FDA approval for Parkinson’s add-on therapy Xadago after a 4-year regulatory odyssey, the biotech is back with a snapshot of early data on a new treatment for schizophrenia. And while they were able to pull out positive data from the Phase IIa study of evenamide, investigators plan to make some significant changes to the next study that could provide more plainly promising pivotal results.【閱讀全文】

ArQule Announces Top-Line Results of Phase 3 Clinical Study of Tivantinib in Hepatocellular Carcinoma in Japan

rQule, Inc. (Nasdaq: ARQL) today reported that its partner, Kyowa Hakko Kirin, announced top-line results of the JET-HCC Phase 3 trial of tivantinib in Japan, and that the trial did not meet its primary endpoint of progression free survival (PFS).

JET-HCC is a randomized, double-blind placebo-controlled study that enrolled approximately 190 Japanese patients with c-Met diagnostic-high inoperable hepatocellular carcinoma (HCC) with a history of prior sorafenib therapy, to evaluate the efficacy and safety of tivantinib.【閱讀全文】

ERYTECH Reports Positive Phase 2b Data for eryaspase for the Treatment of Metastatic Pancreatic Cancer

ERYTECH Pharma (Paris:ERYP) (ADR:EYRYY) (Euronext Paris - ERYP), a French clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, today announced positive topline results from its Phase 2b clinical study evaluating its product candidate, eryaspase (GRASPA?), in combination with chemotherapy for the treatment of second-line metastatic pancreatic cancer. The multicenter, randomized Phase 2b study met its prespecified co-primary endpoints, and showed significant improvement in both progression-free survival (PFS) and overall survival (OS) in patients treated with eryaspase combined with chemotherapy compared to chemotherapy alone.【閱讀全文】

MSD’s Keytruda closer to classical Hodgkin Lymphoma nod

MSD’s anti-PD-1 therapy Keytruda has taken a giant leap towards being approved in Europe for the treatment of classical Hodgkin Lymphoma (cHL).

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has backed expanding the list of approved uses for the drug to include relapsed or refractory cHL in adults who have failed autologous stem cell transplant (ASCT) and brentuximab vedotin (BV), or who are transplant-ineligible and have failed BV.【閱讀全文】

Allergan, Paratek hit late-stage acne goals, prep for sarecycline NDA

Allergan and partner Paratek Pharmaceuticals have hit their primary endpoints in two key phase 3 acne trials for their investigational med sarecycline.

The pair will now seek an FDA NDA in the second half of the year after the two “replicative” late-stage tests that saw the med outdo placebo in treating inflammatory acne lesions in subjects with moderate to severe acne.

Neither company went into exact details on the figures, but said in a statement that sarecycline “was statistically significantly (p<0.004) superior to placebo with respect to primary efficacy endpoints.”【閱讀全文】

Amgen has won European approval for its first biosimilar, a version of AbbVie’s blockbuster Humira (adalimumab).

The European Commission has approved Amgevita (biosimilar adalimumab) in all the available indications of its originator product, marking the first European licence for a biosimlar version of Humira【閱讀全文】

Merrimack asset sale prompts legal blowback

Merrimack Pharmaceuticals is facing several lawsuits over its decision to divest assets to Ipsen, Boston Business Journal reported Thursday.

In the deal, Merrimack is offloading the pancreatic cancer drug Onivyde plus a generic version of the ovarian cancer drug Doxil.【閱讀全文】

FDA decision for Dupixent as Novartis drives its CAR-T

Worldwide sales of Dupixent are expected to hit a huge $4.3bn by 2022, according to sellside consensus from EvaluatePharma, and over half of this is assigned to US sales. The project has an NPV of $15.7bn, or 14% of Sanofi’s market cap; Regeneron collects worldwide royalties.

The MAb is forecast to be Sanofi’s biggest growth driver, looking to plug the gap left by declining Lantus sales, and is also one of the biggest expected launches of the year.【閱讀全文】

A new PD-L1 is born, and success for Newron at long last

Yesterday’s US approval of avelumab for Merkel cell carcinoma is unlikely to trouble the competition, but it will be important for having got the Pfizer/Merck KGaA anti-PD-L1 MAb across the regulatory finish line. Indeed, Merkel cell, a rare skin cancer, is perceived as such an economically unviable proposition that, barring a couple of investigator-initiated studies, none of the leading anti-PD-1/PD-L1 projects are in trials specifically for it. An NCI-sponsored study of Keytruda generated positive data in first-line Merkel cell carcinoma at last year’s AACR meeting, but despite this its maker, Merck & Co, has not picked up the gauntlet. 【閱讀全文】

Novartis laterals two PhII anti-aging drugs to a PureTech startup, grabbing an equity stake

Pharma giant Novartis is spinning out a pair of Phase IIb-ready drugs now bound for advanced trials on repairing the deteriorating impact of aging on the human immune system.

Boston-based PureTech Health is on the receiving end of the deal, setting up a subsidiary called resTORbio which it will now fund, in tranches, with $15 million. And it can earn a growing stake in the operation, rising to 58% on the allocation of the full $15 million, with an option to increase that to 67% if it adds $10 million more.【閱讀全文】

Xenon acne drug misses efficacy endpoints in phase 2

A phase 2 trial of Xenon Pharmaceuticals’ acne candidate XEN801 has missed its primary and key secondary efficacy endpoints. The comprehensive failure prompted Xenon to admit the data don’t support continued development of XEN801 and wiped 44% off its stock price in premarket trading.

Xenon enrolled 165 patients with moderate-to-severe facial acne and randomized them to receive either a gel formulation of XEN801 or placebo. After 12 weeks in which participants applied a gel to their faces each evening, investigators looked for changes against baseline. The study’s primary endpoint was the percent change in lesion count after 12 weeks. Secondary endpoints looked at lesion counts after four and eight weeks, and the change on an investigator assessment scale.【閱讀全文】

The U.S. Food and Drug Administration today granted accelerated approval to Bavencio (avelumab) for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC), including those who have not received prior chemotherapy. This is the first FDA-approved treatment for metastatic MCC, a rare, aggressive form of skin cancer.【閱讀全文】

Biotech giant plans $25M Tampa investment, 450 jobs

Amgen Inc. will open a capability center in Tampa, creating hundreds of high-skill jobs.

Amgen (NASDAQ: AMGN), a biotechnology firm headquartered in Thousand Oaks, California, will invest $25 million in the 136,000-square-foot center, which will occupy four floors of Corporate Center One at 2202 N. Westshore Blvd. The center is expected to open in October and will employ 450 people by the end of 2018, a press release from the Tampa Hillsborough Economic Development Corp. said.【閱讀全文】

BioLineRx buys Agalimmune to expand cancer pipeline

BioLineRx has bought Agalimmune to expand its immuno-oncology pipeline. The takeover adds the synthetic alpha-gal immunotherapy AGI-134 to BioLineRx’s pipeline as the drug nears the start of a first-in-human trial in solid tumors.

Tel Aviv, Israel-based BioLineRx is paying $6 million (€5.6 million) upfront—split evenly between cash and stock—to buy Agalimmune. In return for the outlay, BioLineRx is gaining AGI-134, a preclinical-stage asset it thinks could make immunotherapies effective against solid tumors that evade the attacks of today’s therapeutics.【閱讀全文】

Merrimack Initiates Phase 1 Study of MM-310 in Solid Tumors

Merrimack Pharmaceuticals, Inc. (NASDAQ: MACK) today announced the enrollment of its first patient in a Phase 1 study of MM-310 in solid tumors. MM-310 is an antibody-directed nanotherapeutic (ADN) that encapsulates a novel taxane and targets the EphA2 receptor, a protein which surveys suggest is overexpressed in 50-100% of many major tumor types, including prostate, ovarian, bladder, gastric, pancreatic and lung cancers.【閱讀全文】

Pharma development budgets grow as Roche remains a believer

Roche was once again the sector’s biggest spender on drug development last year, its R&D bill touching $10bn for the first time. The Swiss company also remained one of the most enthusiastic investors in science among the world’s biggest pharma groups, data collated by EvaluatePharma show.

However, in terms of budget growth, Abbvie has increased spend on its development labs more than any other major over the past five years, percentage-wise. The company is working desperately to replace Humira, which in the meantime is generating plenty of cash to support its growing R&D investment.【閱讀全文】

Bristol failure makes small dent in CXCR4-blocking approach

Bristol-Myers Squibb’s discontinuation of a phase I/II study of ulocuplumab highlights an unusual mechanism of action – CXCR4 inhibition – that has quietly been pursued as part of several companies’ oncology strategies.

Biolinerx and Polyphor have similarly acting agents as lead projects, while Lilly is relying on one as potentially its fifth-most lucrative pipeline asset. The approach aims to trigger stem cell mobilisation, and while the Bristol failure raises obvious doubts at least one other group, X4 Pharmaceuticals, is entirely focused on exploring the CXCR4 pathway【閱讀全文】

Atossa Genetics Opens Enrollment in Study of Endoxifen

Atossa Genetics Inc. (NASDAQ: ATOS) today announced that it has opened enrollment of a Phase 1 study of endoxifen, which is an active metabolite of tamoxifen, an FDA approved drug for breast cancer. The objectives of this placebo-controlled, repeat dose study of 48 healthy female volunteers is to assess the pharmacokinetics of proprietary formulations of both oral and topical endoxifen dosage forms over 28 days, as well as to assess safety and tolerability.

The study is being conducted on behalf of Atossa by CPR Pharma Services Pty Ltd., Thebarton, SA, Australia. 【閱讀全文】

Kineta RLR Agonist Demonstrates Tumor Regression and an Adaptive Immune Response in Colon Carcinoma Model

Kineta, Inc., a biotechnology company focused on the translational development of novel therapies in immuno-oncology, today announced the discovery and characterization of several "hit to lead" small molecule compounds that activate interferon response factor 3 (IRF3) via RIG-I like receptor (RLR) pathways and demonstrate immune-mediated tumor regression in a murine colon carcinoma mouse model. Furthermore, mice in the study who demonstrated complete tumor regression to initial drug treatment were resistant to tumor re-challenge confirming an adaptive immune response in these animals. The findings were presented on March 22nd at the Keystone Symposia on Cancer Immunology and Immunotherapy: Taking a Place in Mainstream Oncology conference.【閱讀全文】

Shire’s rare blood disorder drug gets US fast track

An experimental therapy in development by Shire for patients with the rare blood disorder hereditary thrombotic thrombocytopenic purpura (hTTP) has been granted a fast track designation by the US Food and Drug Administration.

The company is targeting its therapy SHP655 (recombinant ADAMTS13) towards treatment of acute episodes of hTTP in patients with a constitutional deficiency of the von Willebrand factor-cleaving (VWF) protease ADAMTS13.【閱讀全文】

Ultragenyx looks ahead after triheptanoin failure

Ultragenyx’s phase II miss with triheptanoin in transporter type-1 deficiency syndrome might not affect an ongoing phase III trial in a slightly different indication – but it does not do much to boost confidence in the project.

Meanwhile, the company can console itself that it will soon have pivotal data from another candidate, KRN23, in X-linked hypophosphataemia, and is planning a filing of the enzyme-replacement therapy rhGUS in the first half of 2017. Sales of these products are likely to be smaller than those for triheptanoin, according to EvaluatePharma consensus, but Ultragenyx might benefit from not putting all of its rare disease eggs in one basket.【閱讀全文】

There were high hopes for the FOURIER trial. Current dogma is that the more you can lower your LDL cholesterol (LDL-c), the lower the risk of a heart attack or stroke. Repatha did, in fact, produce unprecedented LDL-c lowering in FOURIER. Those patients taking optimal doses of statins had on average LDL-c levels of 92mg/dL, whereas those who were also dosed with Repatha had average LDL-c levels of 30mg/dL. The latter patients did have fewer CV events, but of a smaller magnitude than hoped. The PCSK9 had only a 15% decrease in CV events, only half of what had been hoped.【閱讀全文】

BioClin raises $30M Series B round for bladder cancer candidate

Quiet upstart BioClin Therapeutics has raised $30 million in a Series B funding round, taking its total to $59 million since its founding, as it aims to push on with clinical work on its bladder cancer candidate B-701 that includes a combo test with Roche’s new PD-L1 med.【閱讀全文】

Novartis’ heart drug serelaxin fails in key trial

Novartis is considering the future of serelaxin after the drug hit another setback failing to meet key goals in a late-stage study involving patients with acute heart failure.

The Phase III RELAX-AHF-2 study failed to hit its primary endpoints of showing that serelaxin (also known as RLX030) reduced the risk of cardiovascular death or reduced worsening heart failure when added to standard therapy.【閱讀全文】

Aprea Therapeutics Announces Research Collaboration with Memorial Sloan Kettering Cancer Center

Aprea Therapeutics, a privately held, clinical stage biopharmaceutical company developing novel anticancer therapies targeting the tumor suppressor protein p53, today announced a collaboration with Memorial Sloan Kettering Cancer Center to study the effects of reactivation of tumor suppressor protein p53 by APR-246.【閱讀全文】

AVEO Announces First Patient Dosed in Phase 1/2 TiNivo Trial of Tivozanib and Opdivo (nivolumab) in Advanced RCC

AVEO Oncology (NASDAQ:AVEO) today announced that the first patient has been dosed in the Phase 1/2 AVEO-sponsored TiNivo trial evaluating tivozanib in combination with Bristol-Myers Squibb’s anti-PD-1 therapy, Opdivo? (nivolumab), in advanced renal cell carcinoma (RCC). The study, which will be led by the Institut Gustave Roussy in Paris, is under the direction of Professor Bernard Escudier, MD, Chairman of the Genitourinary Oncology Committee. The Phase 1 trial will evaluate the safety of tivozanib in combination with nivolumab at escalating doses of tivozanib and, assuming favorable results, is expected to be followed by an expansion Phase 2 cohort at the established combination dose.【閱讀全文】

Serelaxin setback puts focus on Entresto

The prospects for Novartis’s heart failure franchise have taken a major blow with the phase III failure of serelaxin. Not only has the company lost a potential blockbuster, it is now even more reliant on Entresto, which has so far struggled to live up to expectations.【閱讀全文】

Erytech looks to pancreatic cancer data for uplift

The French company Erytech is offering a novel approach to cancer treatment with Graspa, a red blood cell-encapsulated version of the chemotherapy drug L-asparaginase. The aim is to avoid the allergic reactions seen with older forms of the drug that are not encapsulated, with data in second-line pancreatic cancer due by the end of the first quarter.【閱讀全文】

A year after a patient's death, Akashi gets FDA all-clear to resume DMD trial

kashi Therapeutics has been given the FDA go-ahead to restart tests for its Duchenne Muscular Dystrophy (DMD) candidate HT-100 (delayed-release halofuginone), just over a year after the test was suspended after a death.

The Cambridge, MA-based biotech at the start of last year suspended its study after a patient was sent to the hospital, and then a week later the biotech reported that the trial subject has died.【閱讀全文】

Neurocrine posts full phase 3 Ingrezza data as PDUFA date, showdown with Teva in tardive dyskinesia near

Neurocrine Biosciences has published a journal paper about its phase 3 trial of tardive dyskinesia candidate Ingrezza. Publication of the paper comes weeks before a PDUFA date that could result in Neurocrine and Teva going head to head for the neurological disorder market in the years to come.

San Diego, CA-based Neurocrine published top-line results from the phase 3 trial of its vesicular monoamine transporter 2 inhibitor late in 2015. This week’s publication in The American Journal of Psychiatry fleshes out the dataset by providing additional details of the safety and efficacy of the candidate.【閱讀全文】

In his first address to a joint session of Congress, President Donald Trump made a dramatic appeal for lowering the hurdles new medicines face in gaining approval from the Food and Drug Administration. But Trump’s promises – that reduced regulatory standards would result in more cures reaching the market and lower drug prices – may be hard to keep.【閱讀全文】

A Safe “BET” on Epigenetic Drugs

Epigenetic therapies, small-molecule drugs named for their ability to alter gene expression by targeting epigenetic regulators, could enable powerful new strategies to combat diseases marked by aberrant gene expression, like cancer.

Epigenetic regulators fall into three main categories: writers, erasers, and readers. Writers modify histones by adding chemical groups that result in acetylation, phosphorylation, methylation, or other modification types, while erasers remove these groups. The important task of recognizing these modifications falls to reader proteins.【閱讀全文】

Priority review for Merck, Pfizer’s avelumab in urothelial cancer

US regulators have agreed to undertake a priority review Merck and Pfizer's avelumab as a potential treatment for metastatic urothelial carcinoma (mUC).

The companies are targeting the treatment towards patients with locally advanced or mUC with disease progression on or after platinum-based therapy.【閱讀全文】

Genentech to launch second PhIII trial of Alzheimer’s drug

Roche's Genentech is launching a second Phase III trial of anti-Abeta antibody crenezumab in patients with early forms of Alzheimer's disease (AD), on the back of promising data on the drug's "unique" binding properties and in support of increased dosing.

The new trial - CREAD2 - intends to recruit 750 patients with prodromal or mild AD, complementing the ongoing Phase III CREAD1 trial, which is assessing the drug in the same patient target group and is expected to read out in 2020.【閱讀全文】

Lilly Completes Acquisition of CoLucid Pharmaceuticals

Eli Lilly and Company (NYSE: LLY) today announced the successful completion of its acquisition of CoLucid Pharmaceuticals, Inc. (NASD:CLCD). The tender offer for all outstanding shares of common stock of CoLucid, at a price of $46.50 per share, expired as scheduled on Tuesday, February 28, 2017.【閱讀全文】

SELLAS Life Sciences Announces Updated Positive Phase 2 Data Reinforcing Meaningful Clinical Benefit of its WT1 Immunotherapeutic Anti-cancer Treatment in Multiple Myeloma Patients

SELLAS Life Sciences Group (SELLAS or the Company), a late-stage biopharmaceutical company developing novel cancer immunotherapies for an extensive list of cancer indications, today reported updated positive Phase II data from its WT1 immunotherapeutic anti-cancer treatment, galinpepimut-S, in multiple myeloma (MM) patients. Consistent with preliminary data, the updated results indicate a meaningful clinical benefit among high-risk MM patients, positioning galinpepimut-S as a potential first-in-class immunotherapeutic cancer treatment. MM patients typically relapse within 12 months after frontline induction therapy, followed by successful autologous stem cell transplantation (ASCT). 【閱讀全文】

Months after another lethal setback, Juno finally opts to kill lead CAR-T

Months after one of the worst setbacks in the recent history of drug development left a string of patients dead, Juno has finally decided that it will terminate its lead drug program, hoping for a better outcome with another CAR-T therapy in its pipeline.【閱讀全文】

Investigational drug shows promise for hard-to-treat renal cancer

The investigational compound savolitinib appears to have antitumor activity in patients with c-MET-driven papillary renal cell carcinoma, according to the results of a phase II study released at the 2017 Genitourinary Cancers Symposium in Orlando, FL.

Savolitinib is an oral, highly selective inhibitor of the c-MET receptor tyrosine kinase.【閱讀全文】

After 22 years Lexicon has an approved drug

Lexicon Pharmaceuticals has been through its share of makeovers. Still, no one who remembers its birth as a genetic engineering company before the 2000 biotech boom and bust could have predicted that it would end up a rare disease player.【閱讀全文】

Enasidenib in Relapsed or Refractory AML with an IDH2 Mutation

Celgene Corporation (NASDAQ:CELG) and Agios Pharmaceuticals (NASDAQ:AGIO) today announced that the U.S. Food and Drug Administration (FDA) has accepted Celgene’s New Drug Application (NDA) for enasidenib (AG-221/CC-90007) for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase 2 (IDH2) mutation. The NDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of Aug. 30, 2017. Celgene completed the NDA submission in late December 2016.【閱讀全文】

Battered over safety questions in ’16, Aurinia comes back with more positive data on lupus drug

Last summer, Aurinia $AUPH found itself between a rock and a hard place as it sought to explain a troubling cluster of deaths in the two dosing cohorts — as well as an odd flip-flop in results you might expect to see from the low and high doses — used to test their drug voclosporin for lupus nephritis. Unsatisfied by the case Aurinia made for efficacy and safety at 24 weeks, investors pulled out after looking over the fallout, cratering the stock.【閱讀全文】

EMA adds Alnylam RNAi drug to PRIME fast-track scheme

The European Medicines Agency (EMA) has added Alnylam’s acute hepatic porphyria candidate to its priority medicine (PRIME) scheme. Securing the status positions Alnylam to benefit from a close relationship with the EMA as it works to move the drug prospect into phase 3 by the end of the year.【閱讀全文】

Accera fails phase 3 Alzheimer’s trial, denting energy theory

A phase 3 trial of Accera’s Alzheimer’s disease candidate has missed its primary endpoint. The trial aimed to show Accera’s brain metabolism-focused approach to improving outcomes in Alzheimer’s could succeed where beta-amyloid drugs have failed, but instead ended up becoming yet another late-phase failure.【閱讀全文】

New Jersey oncology biotech PMV Pharma has seen a healthy $74 million in funding from its Series B with help from InterWest Partners, Osage University Partners, Topspin Biotech Fund (which led this round), and its Series A lead investor OrbiMed.

The company said it will use its new cash boost for work on its p53-targeted small molecule drugs in cancer, building on the $30 million it got back in December 2014.【閱讀原文】

2016 went out like a lamb in the biopharma M&A world. Will it roar back in 2017? Plus, top 10 deals

Dealmakers evidently spent the fourth quarter of 2016 largely in hibernation as they waited out a big US election cycle that seemed to raise more questions than it answered.

In a look back over a relatively quiet year, EP Vantage, the editorial arm at Evaluate, concluded that 2016 may not have ended anywhere close to any of the records set during the boom, but I would add that the numbers reflect some considerable financial muscle that could be flexed in the year to come.【閱讀原文】

Oncternal Closes $18.4 Million Series B Financing

Oncternal Therapeutics, Inc., a clinical-stage biotechnology company developing first-in-class therapies for both rare and common malignancies, today announced the closing of an $18.4 million Series B financing. The company intends to use the proceeds to further clinical development programs for cirmtuzumab and TK216, and to advance preclinical development of a new ROR1-targeted antibody-drug conjugate (ADC) program.【閱讀原文】

Argos kidney cancer vax fails phase 3 futility review, future in doubt

Argos Therapeutics’ hopes of winning approval for its cancer vaccine candidate rocapuldencel-T have taken a major blow. The independent monitoring committee recommended the phase 3 trial is stopped for futility after getting a look at interim results, wiping 65% off Argos' stock and leaving it to scour through the data in search of a path forward for its lead candidate.【閱讀原文】

Icon to validate PROs as measurement of clinical endpoints for the FDA

The U.S. FDA has selected Icon to validate three patient-reported outcome (PRO) instruments used to assess clinical endpoints in antibacterial drug trials.

With the help of the Biomarkers Consortium of the Foundation for the National Institutes of Health, Icon’s Clinical Outcomes Assessment group will develop an electronic platform to validate PROs.【閱讀原文】

Scientists identify chain reaction that shields breast cancer stem cells from chemotherapy

Working with human breast cancer cells and mice, researchers at Johns Hopkins say they have identified a biochemical pathway that triggers the regrowth of breast cancer stem cells after chemotherapy.

The regrowth of cancer stem cells is responsible for the drug resistance that develops in many breast tumors and the reason that for many patients, the benefits of chemo are short-lived. Cancer recurrence after chemotherapy is frequently fatal.【閱讀原文】

Icahn has snapped up a Bristol-Myers stake. Could megamerger pressure be far behind?

What a difference one failed trial makes. After Bristol-Myers Squibb’s immunotherapy Opdivo fell short in a key lung cancer study last fall, the company’s shares plummeted, analysts cut their sales forecasts by billions, and buyout rumors started surfacing.

Now, activist investors are plowing in.【閱讀原文】

THE WORLD’S MOST EXTREME physical exam starts in the world’s plushest exam room, complete with a couch, a private bathroom and a teeming fruit plate. It will be my home for an entire day. First come the blood tests, vial after vial. Then two 35-minute sessions in an MRI tube, where REM and U2 try to drown out the clanks as the machine takes pictures of my entire body. There’s an ultrasound of my heart. Salade Ni?oise for lunch. A stool sample. A cognitive test in which letters flash on a computer screen at a dizzying pace. And a CT scan of my heart as well, which originally seemed so over-the-top for someone my age that I tried to get out of it.【閱讀全文】

Sarepta sells Exondys 51 PRV to Gilead for $125M

Sarepta has struck a $125 million deal to sell a voucher it got with last year’s controversial FDA approval of its new Duchenne drug to Gilead, which will give the Big Biotech a one-time speedy review pass for an NDA app to the U.S. regulator.

The biotech got the Rare Pediatric Disease Priority Review Voucher as part of its green light for Exondys 51 in certain Duchenne patients in the fall of last year. Analysts had long expected the biotech to sell the valuable voucher.【閱讀全文】

OncoGenex Pharmaceuticals, Inc. Announces Phase 2 Apatorsen Data for Two Clinical Trials

OncoGenex Pharmaceuticals, Inc. (NASDAQ: OGXI) today announced that apatorsen results from two randomized Phase 2 clinical trials were presented at the American Society of Clinical Oncology (ASCO) 2017 Genitourinary Cancers Symposium, held February 16th- 18th in Orlando. Clinical data from trials in bladder and prostate cancers demonstrated apatorsen was well-tolerated and improved patient outcomes when administered in combination with standard-of-care treatments.【閱讀全文】

Buyout buzz swells for the giant Bristol-Myers after Carl Icahn makes an entrance, stage left

Carl Icahn, the aging activist who’s prodded a series of high-profile biopharma companies into the arms of eager buyers, has taken a stake in the highly stressed Bristol-Myers Squibb, building speculation that the troubled giant could be in line for a buyout – as improbable as that may sound to many.

Bristol-Myers started the day by announcing that it added three new directors and struck a $2 billion stock buyback deal in a pact with Jana Partners, another activist which has been spurring Bristol-Myers to take action to get out of the rut it’s found itself in.【閱讀全文】

VBL Therapeutics Reports Full Data for VB-111 Monotherapy in Phase 2 Trial for Recurrent Thyroid Cancer

VBL Therapeutics (NASDAQ:VBLT), announced today full results from its exploratory Phase 2 study of VB-111 (ofranergene obadenovec) in patients with advanced, differentiated thyroid cancer. The data will be presented today by Dror Harats, M.D., CEO of VBL Therapeutics, at the Federation of the Israel Societies for Experimental Biology (FISEB) conference in Eilat, Israel.【閱讀全文】

Pfizer Announces Acceptance of Regulatory Submission for Inotuzumab Ozogamicin by the U.S. Food and Drug Administration

Pfizer Inc. (NYSE:PFE) today announced that a Biologics License Application (BLA) for inotuzumab ozogamicin has been accepted for filing and granted Priority Review by the U.S. Food and Drug Administration (FDA). Inotuzumab ozogamicin is being evaluated for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).【閱讀全文】

Top 10 U.S. patent losses of 2017

Among the many meds that’ll lose patent protection in the U.S. this year are 10 drugs that each contribute in a big way for top drugmakers. Eli Lilly, Pfizer, Takeda, Bristol-Myers Squibb and Gilead are each set to hit the patent cliff this year with some of their respective big sellers.

Together, they brought in more than $10 billion last year in the U.S. and cover a range of indications: multiple sclerosis, HIV, erectile dysfunction and cancer, among others. They’ll be hot targets as generic rivals rush to steal share with cheaper options.【閱讀全文】

Hutchison China MediTech Limited (“Chi-Med”) (AIM/Nasdaq: HCM) today announces that a Phase II study of savolitinib has been initiated in locally advanced or metastatic pulmonary sarcomatoid carcinoma (“PSC”) in China. Savolitinib is a highly selective and potent oral c-Met inhibitor with global first-in-class potential. The first drug dose was administered on February 10, 2017.【閱讀原文】

AstraZeneca’s Lynparza hits breast cancer trial targets

A late-stage study of AstraZeneca's PARP inhibitor Lynparza in patients with HER2-negative metastatic breast cancer who also carry BRCA1 or BRCA2 mutations has its primary target in boosting progression-free survival (PFS).

In the international Phase III OLYMPIAD trial, 302 patients were randomised to receive either Lynparza (olaparib) or a physician's choice of chemotherapy. According to top-line data, AstraZeneca's drug showed a significant and clinically-meaningful improvement in PFS versus chemotherapy, meeting the study's main endpoint.【閱讀原文】

Biosimilar Inflectra matches Remicade efficacy in trial

Pfizer and Celltrion have unveiled Phase III study data comparable safety and efficacy between biosmilar Inflectra and reference drug Remicade in patients with moderate-to-severe Crohn's disease (CD).

The randomized 54 week clinical trial in 214 patients met its primary end point in showing that, at six weeks, Inflectra (infliximab CT-P13) was similar to Remicade (infliximab) in the treatment of CD thereby meeting the criterion for non-inferiority.【閱讀原文】

Ozanimod safety still a question mark for Celgene

A first phase III win for Celgene’s ozanimod puts the project on track for approval in multiple sclerosis and goes some way to justifying the $7.2bn that the company paid for its originator, Receptos.

But, with safety data still thin on the ground, it is unclear whether ozanimod will get a more favourable label than Novartis’s rival sphingosine-1-phosphate modulator Gilenya. And, with a relatively crowded S1P pipeline, ozanimod needs to set itself apart if Celgene is to recoup its investment.【閱讀原文】

MD Anderson Benches IBM Watson In Setback For Artificial Intelligence In Medicine

It was one of those amazing “we’re living in the future” moments. In an October 2013 press release, IBM declared that MD Anderson, the cancer center that is part of the University of Texas, “is using the IBM Watson cognitive computing system for its mission to eradicate cancer.”

Well, now that future is past. The partnership between IBM and one of the world’s top cancer research institutions is falling apart. The project is on hold, MD Anderson confirms, and has been since late last year. MD Anderson is actively requesting bids from other contractors who might replace IBM in future efforts. 【閱讀原文】

AstraZeneca sells commercial rights to Zoladex cancer treatment

AstraZeneca plc (LON:AZN) has agreed to sell the commercial rights of its Zoladex cancer treatment in the US and Canada to TerSera Therapeutics in a deal worth more than $320mln.

TerSera will pay Astra $250mln up front for Zoladex, an injectable drug used to treat malignant prostate and breast cancer.【閱讀原文】

Negative Trial in Metastatic RCC Still Generates Buzz

negative study in metastatic renal cell carcinoma (mRCC) stimulated a fair amount of positive feedback following a presentation here.

The randomized phase II study showed that the combination of the PD-L1 inhibitor atezolizumab (Tecentriq) and the angiogenesis inhibitor bevacizumab (Avastin) did not significantly improve progression-free survival (PFS) in untreated mRCC compared with sunitinib (Sutent). Atezolizumab alone also did not improve PFS versus sunitinib.【閱讀原文】

SWOG study shows strong long-term survival rates for patients with GIST

Nine years ago, SWOG researchers confirmed a new standard of care for patients with incurable gastrointestinal stromal tumors (GIST), who could survive by being treated with imatinib mesylate, the breakthrough drug marketed as Gleevec. SWOG researchers are back with long-term findings from that study, which estimate that nearly one in four patients treated with Gleevec will survive 10 years. Results are published in JAMA Oncology.【閱讀原文】

Takeda Pharmaceutical Company Limited (TSE:4502) (“Takeda”) today announced the completion of its acquisition of ARIAD Pharmaceuticals, Inc. (NASDAQ:ARIA) (“ARIAD”) for $24.00 per share in cash.

“We are very pleased to have completed the acquisition of ARIAD Pharmaceuticals. The addition of ARIAD’s innovative targeted therapies and research and development capabilities strengthens and diversifies our oncology business, positioning Takeda for sustainable long-term growth in this priority therapeutic area,” said Christophe Weber, president and chief executive officer of Takeda. 【閱讀全文】

Zymeworks Receives Second Orphan Drug Designation for ZW25 in Gastric Cancer

Zymeworks Inc. (“Zymeworks”), a clinical-stage biopharmaceutical company dedicated to the discovery, development and commercialization of next-generation multifunctional biotherapeutics, initially focused on the treatment of cancer, today announced that its lead product candidate, ZW25, has been granted orphan drug designation from the U.S. Food and Drug Administration (the “FDA”) in the treatment of gastric cancer, including cancer of the gastroesophageal junction (“GEJ”).【閱讀全文】

Stanford scientists manipulate gene expression to make insulin-producing cells

Stanford University scientists converted pancreatic alpha cells to insulin-producing beta cells in mice by blocking the production of a pair of proteins. If translatable to humans, this method could be used to replenish faulty beta cells and ease diabetes symptoms.

"It is important to carefully evaluate any and all potential sources of new beta cells for people with diabetes," said Dr. Seung Kim, a professor of developmental biology and of medicine at Stanford, in a statement. "Now we've discovered what keeps an alpha cell as an alpha cell, and found a way to efficiently convert them in living animals into cells that are nearly indistinguishable from beta cells."【閱讀全文】

Pfizer's solo Xeljanz can't match AbbVie's Humira in big RA head-to-head miss

Pfizer has struggled to get rheumatoid arthritis pill Xeljanz to bring in the kind of sales it wants—and a new head-to-head study against AbbVie behemoth Humira isn't going to help as much as the New York drugmaker had hoped.

Thursday, the company announced that Xeljanz, one in a new class of JAK inhibitors, had matched Humira when both drugs were paired with methotrexate. On its own, though, Xeljanz didn't measure up to the world’s best seller, missing the study’s primary endpoint of non-inferiority.【閱讀全文】

Reasons for Ibrutinib Discontinuation Linked With Length of CLL Survival

Patients with chronic lymphocytic leukemia (CLL) who discontinue treatment with the Bruton tyrosine kinase inhibitor ibrutinib due to disease progression or transformation had significantly worse survival compared with patients who discontinued therapy because of intolerance, according to the long-term results of a study published recently in Cancer.【閱讀全文】

TRACON Pharmaceuticals Announces First Patient Dosed in Phase 3 TAPPAS Trial of TRC105 in Angiosarcoma

TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer, wet age-related macular degeneration (AMD) and fibrotic diseases, announced today that it has initiated patient dosing in its Phase 3 TAPPAS (TRC105 And Pazopanib versus Pazopanib alone in patients with advanced AngioSarcoma) trial of TRC105.【閱讀全文】

U.S. FDA Accepts Biologics License Application (BLA) for Mylan and Biocon's Proposed Biosimilar Pegfilgrastim for Review

Mylan N.V. (NASDAQ, TASE: MYL) and Biocon Ltd. (BSE code: 532523, NSE: BIOCON) today announced that the U.S. Food and Drug Administration (FDA) has accepted Mylan's Biologics License Application (BLA) for MYL-1401H, a proposed biosimilar to Neulasta? (pegfilgrastim), for filing through the 351(k) pathway.

The proposed biosimilar to Neulasta is used to reduce the duration of neutropenia (low count of neutrophils, a type of white blood cells) and the incidence of fever associated with neutropenia in adult patients treated with chemotherapy in certain types of cancer.【閱讀全文】

What's wrong with Sanofi's dealmaking approach? To hear Actelion tell it, plenty

It’s no secret that Sanofi has struggled of late at the dealmaking table. But why? The deal prospectus from former target Actelion and its agreed-to buyer, Johnson & Johnson, offers some clues.

Late last year, when J&J walked on Actelion after nearly a year of talks, many industry watchers thought Sanofi had a $30 billion deal for the Swiss biotech in the bag. The French drugmaker had reportedly offered up a price higher than what J&J was wiling to pay, forcing the New Jersey pharma giant out of the picture.【閱讀全文】

BERG LLC, a biopharmaceutical company uncovering health solutions through a data-driven, biological research approach, today announced that it has initiated a Phase I/II monotherapy clinical trial for its drug candidate BPM 31510-IV for the potential treatment of glioblastoma multiforme. The compound was guided in development by BERG's unique AI-based Interrogative Biology? platform that combines patient biology and artificial intelligence-based analytics to better understand the differences between healthy and disease environments.【閱讀全文】

Two deals in two days means aesthetics is looking good

Both Allergan and Hologic have done billion-dollar deals for aesthetics companies this week, with the aim of capitalising on the fast-growing area of non-invasive beauty treatments. This is hardly a leftfield move for Botox's maker, Allergan, but most of Hologic’s business is in the areas of diagnostics, imaging and obstetrics.

It seems likely that, as payer pushback intensifies in the more traditional areas of medtech, Hologic believes that it might be easier to make money from procedures for which healthy people are prepared to pay out of pocket.【閱讀全文】

Antibody-Drug Conjugates: Catalysts for Chemistry

Antibody-drug conjugates (ADCs), also known as armed antibodies, are positioned to be a significant source of next-generation oncolytic therapies. There has been explosive growth in ADC R&D, especially since the development and subsequent marketing approval of Mylotarg (gemtuzumab ozogamicin; withdrawn from market in 2010), Adcetris (brentuximab vedotin), and Kadcyla (ado-trastuzumab emtansine). Estimates place the global ADC market at $10 billion annually after 2024 with seven to 10 new commercial ADC launches projected in the next decade.【閱讀全文】

Stop Freaking Out About CRISPR! (Except For One Thing)

It’s been a huge two days for CRISPR, the biotechnology innovation that makes it far easier for scientists to edit DNA in living cells. Yesterday, a report from the National Academies of Science didn’t rule out eventually using the technology to rewrite the genes of babies shortly after conception. Today, a patent ruling gave key intellectual property to the Broad Institute of MIT and Harvard, causing shares in one biotechnology company involved in CRISPR work to shoot up and two others to crater.【閱讀全文】

Plant-made virus shells could deliver drugs directly to cancer cells

Viruses are extremely efficient at targeting and delivering cargo to cells. In the journal ACS Nano, researchers report they have harnessed this well-honed ability -- minus the part that makes us sick -- to develop virus-like nanoparticles to deliver drugs straight to affected cells. In lab tests, they show that one such particle can be produced in plants and it ferries small molecules to cancer cells.【閱讀全文】

Europe ready to embrace first copies of biotech cancer drugs

Treatment with two important cancer drugs is about to get much cheaper in Europe with a cut-price copy of Roche's (ROG.S) blood cancer drug Rituxan likely to hit the market imminently followed by a rival to its breast cancer medicine Herceptin.

As cancer drug prices spiral, the arrival of the first biosimilars or copies of biotech drugs, ones made inside living cells, puts European oncologists in the forefront of a treatment shift that could slash costs and expand patient access.【閱讀全文】

Another Alzheimer's Drug Study Fails. This Time It's Merck's

Last night, Merck announced that it is stopping a clinical trial of a pill it is developing to treat Alzheimer's because there is "virtually no chance of finding a positive clinical effect." Investors were not surprised, given the track record of other such medicines.

The drug, verubecestat, is the latest in a long string of medicines to fail to have an effect on the disease. Over the past decade, experimental Alzheimer's drugs have repeatedly failed to slow the memory-destroying disease. Late last year, an antibody drug infused into patients' bodies, made by Eli Lilly, didn't have a significant effect on the disease. A medicine from Pfizer, Johnson & Johnson and Elan Pharmaceuticals, which was similar to the Lilly drug, had also previously also failed.【閱讀全文】

Integra offers $1B+ bid for J&J’s neurosurgery biz

Integra LifeSciences has made a binding offer to acquire Johnson & Johnson’s Codman Neurosurgery business for about $1.05 billion in cash.

Codman, which reeled in about $370 million in revenue last year, is part of J&J’s DePuy Synthes group. If J&J accepts the bid, Integra will boost its neurosurgical offerings, getting its hands on surgical devices used to treat brain tumors, hydrocephalus (or the buildup of fluid in the brain) and other neurological disorders.【閱讀全文】

Versant’s Jecure Therapeutics debuts with a focus on NASH, fibrosis

VC Versant has pumped $20 million on its own into its newly launched preclinical liver disease upstart Jecure, with its founders coming out from a former Versant biotech.

This founding team includes CEO Jeffrey Stafford, Ph.D., and CSO James Veal, Ph.D., both of whom were execs at Quanticel Pharmaceuticals, an oncology biotech founded by Versant and acquired by Celgene two years ago.【閱讀全文】

Would Pfizer, Roche or Novartis megamerge with BMS? Rumors say they're crunching numbers now

Last month, a couple of analysts addressed Bristol-Myers Squibb as a takeover target for some rival pharma giant. The gist? The combination of disappointments on its cancer blockbuster Opdivo plus a solid product line-up could well put a bullseye on the company—and its “very logical, very rational” management team wouldn’t dismiss offers out of hand.

Now, rumors are surfacing that Swiss-based drugmakers Roche and Novartis, leaders in the cancer field, and Pfizer are actively looking at a buyout. StreetInsider, which cited anonymous sources in reporting the talk, also said that Gilead Sciences—which, as executives recently admitted, needs some significant M&A to grow—might be considering the idea.【閱讀全文】

Incyte and Agenus have amended an existing license, development, and commercialization agreement for Agenus’s GITR (glucocorticoid-induced tumor necrosis factor receptor) and OX40 antibody programs, to give Incyte responsibility for funding global development and commercialization of resulting antibody candidates. The amended deal converts the agreements from development co-funding and profit sharing to royalty generating, with Incyte paying Agenus 15% royalties on global net sales for each approved product.【閱讀全文】

Lilly/Incyte’s Olumiant bags first approval with EU nod

Eli Lilly's Olumiant has picked up its first regulatory approval with a nod from the European Commission to treat moderate-to-severe active rheumatoid arthritis.

The decision allows the drug's use either as a monotherapy or in combination with methotrexate to treat adult patients with the condition who have responded inadequately to, or who are intolerant to, one or more disease-modifying antirheumatic drugs (DMARDs).【閱讀全文】

Gilead and Glaxo’s HIV battle intensifies

The first phase II data with Gilead’s HIV integrase inhibitor bictegravir have set up a showdown with Glaxosmithkline/Viiv Healthcare’s marketed product dolutegravir. But Glaxo is already one step ahead with data from a two-drug combo that could reduce the side-effect burden for HIV patients.

Glaxo believes that this doublet could “reshape the whole game”, the group's chief executive, Andrew Witty, said on its fourth-quarter earnings call. HIV has been one of the group’s main drivers in recent quarters as it has taken market share from Gilead, which is becoming increasingly reliant on HIV as its hepatitis C franchise slows. 【閱讀全文】

Onxeo Receives USPTO Notice of Allowance for a new AsiDNATM-Related Patent, Expanding Its IP Protection in the U.S.

Onxeo S.A. (Paris:ONXEO) (NASDAQ OMX:ONXEO), a clinical-stage biotechnology company specializing in the development of innovative drugs for the treatment of orphan diseases, in particular in oncology, today announced that the U.S. Patent and Trademark Office (USPTO) has issued a Notice of Allowance for a new patent (n° 15/232,844) relating to the Company’s candidate AsiDNA?, a first-in-class product that prevents tumor cells from repairing their DNA.【閱讀全文】

Amgen Submits Supplemental Biologics License Application For BLINCYTO (Blinatumomab) In Relapsed Or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia

Amgen (NASDAQ: AMGN) today announced the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for BLINCYTO^? (blinatumomab) to include overall survival (OS) data from the Phase 3 TOWER study, supporting the conversion of BLINCYTO's accelerated approval to full approval. The sBLA also includes new data supporting the treatment of patients with Philadelphia chromosome-positive (Ph+) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). The application aims to broaden BLINCYTO's indication for the treatment of patients with relapsed or refractory B-cell precursor ALL.【閱讀全文】

Intercept strikes at the heart of the Nash problem

Changing the design of a pivotal trial rarely augurs well. But on Friday Intercept made a clever pitch that amending Ocaliva’s pivotal Regenerate study in Nash would increase its chances of success as well as cutting the recruitment burden, and the sellside was sold, with upgrades pushing the stock up 5%.

True, much of what the group has done is simply a response to the low bar set by Intercept’s Nash competitors. But the changes raise serious questions about whether Nash is even a disease whose treatment might realistically appear as an indication on Ocaliva’s label.【閱讀全文】

The E100: Biotech execs are bullish about 2017, but fret about drug pricing and the FDA under Trump

The biotech industry is feeling bullish about its own prospects at the beginning of 2017, largely satisfied with the financial support that’s been flowing in to the field with most execs ready to hire through the year as the tempo on deal-making remains upbeat.

But it’s not all coming up roses.

The industry is in a funk about the Trump administration after a few weeks of headlines over a travel ban controversy and a rolling series of outspoken tweets, with a large segment of these execs worried that a new head of the FDA could come in ready to reduce if not actually discard standards on drug development.【閱讀全文】

Celgene co-founder Sol Barer bags a Johns Hopkins immuno-oncology spinout in buyout

A few weeks ago, Johns Hopkins Professor Jonathan Schneck published new research showing how the biomimetic nano beads he had created in the lab could work with a checkpoint inhibitor in fighting cancer. These beads were designed to act as antigen presenting cells aimed at whipping up a full scale killer T cell attack on cancer as the checkpoint stripped the cancer cells of their built-in defense system.【閱讀全文】

Double-whammy for Aviragen as lead drug trial ends in failure

It's been a torrid few days for antiviral drug developer Aviragen as a second phase 2 trial ended in failure, all but decapitating its R&D pipeline.

The phase 2b SPIRITUS trial of vapendavir in moderate to severe asthma patients failed to hit its main objective of curbing breakthrough symptoms and attacks in asthmatics with rhinovirus (RV) infections, according to a statement from Aviragen.【閱讀全文】

That was fast: Days after launching a strategic review, Teva's eyeing a branded generics sale or spinoff

Teva’s strategic review launched only last week, but the company is reportedly already weighing asset sales.

The company is considering a sale of its branded generics business, Bloomberg’s sources say. That’s a deal that could fetch several billion dollars, which Teva could use to cut down its heavy debt load.【閱讀全文】

Inovio Pharmaceuticals, Inc. (NASDAQ:INO) today announced that it has entered into a collaboration and license agreement providing ApolloBio Corporation (NEEQ:430187) with the exclusive right to develop and commercialize VGX-3100, Inovio’s DNA immunotherapy product designed to treat pre-cancers caused by human papillomavirus (HPV), within Greater China (China, Hong Kong, Macao, Taiwan). The agreement provides for potential inclusion of the Republic of Korea three years following the effective date.【閱讀全文】

VenBio slams timing, terms of Immunomedics’ $2B deal

VenBio has criticized the timing and terms of Immunomedics’ licensing deal with Seattle Genetics. The investment fund has accused Immunomedics of rushing through a “poorly constructed deal” to sway shareholder opinion ahead of the upcoming fight for control of the biotech’s board.

Immunomedics is set to pocket $250 million upfront and up to $2 billion in total in exchange for the rights to IMMU-132, a solid tumor antibody-drug conjugate (ADC) that is closing in on a filing for FDA approval in triple-negative breast cancer. The Immunomedics management team hailed the deal as validation of their efforts and the company’s share price rose 22%. But venBio, which has bought a 9.9% stake in Immunomedics and is agitating for change, is unimpressed.【閱讀全文】

MabVax Therapeutics Studies Lead Investigational Drug MVT-5873 with Halozyme PEGPH20

MabVax Therapeutics Holdings, Inc. (NASDAQ: MBVX), a clinical stage immuno-oncology drug development company, today announced results from a pre-clinical study evaluating the potential benefits of using MabVax's HuMab-5B1antibody, MVT-5873, currently in Phase I clinical trials for the treatment of metastatic pancreatic cancer with Halozyme Therapeutic's (NASDAQ: HALO) investigational drug PEGPH20, which targets the tumor microenvironment potentially allowing increased access of co-administered cancer drug therapies to solid tumors. PEGPH20 is currently in phase III clinical development for metastatic pancreatic cancer and in phase I clinical trials for non-small cell lung cancer, gastric cancer, and metastatic breast cancer.【閱讀全文】

Stada receives 3.6 billion euro offer from private equity group Cinven: FT

German generic drugmaker Stada <STAGn.DE> has received a 3.6 billion euro ($3.83 billion) takeover offer from private equity group Cinven, the Financial Times reported.

Cinven's offer follows a year-long activist campaign to improve Stada's profitability by Active Ownership Capital, one of its largest shareholders, and is believed to be pitched at close to 58 euros a share, the Financial Times reported, citing sources.

Cinven declined to comment. Stada was not immediately available to comment.【閱讀全文】

Sanofi Agrees to Sell Five Over-the-Counter Drugs to Ipsen

Sanofi agreed to sell some over-the-counter products to Ipsen SA as it edges closer to completing a 22.8 billion-euro ($24.2 billion) asset swap with Boehringer Ingelheim GmbH. Ipsen will pay 83 million euros for five products including Prontalgine, a painkiller, the Paris-based company said in a statement Monday. Bloomberg previously reported the deal was close, citing a person familiar with the situation. French drugmaker Ipsen emerged as the preferred bidder after a competitive sale process involving other pharmaceutical companies, the person said. Ipsen said it expects to complete the purchase in the second quarter. In August, the European Commission approved the proposed acquisition of Boehringer.【閱讀全文】

Senator says drugmakers may be misusing FDA orphan drug rules—and costing taxpayers money

Have drugmakers misused a decades-old law designed to spur research into rare, neglected diseases? Sen. Chuck Grassley says he aims to find out. The frequent pharma critic says he's investigating whether “unanticipated uses” of the Orphan Drug Act have helped boost U.S. drug prices.

Last month, Kaiser Health News published a report showing that pharma companies are legally using the Orphan Drug Act to win years of additional brand exclusivity for mass market meds. Drugmakers reapply for approvals in rare diseases, and, if successful, those green lights come with seven years of exclusive access to markets, along with other financial incentives.【閱讀全文】

Early win means phase 3 test is on the way for Axovant's dementia drug

Positive results in a first crop of patients treated with its dementia drug have encouraged Axovant to start preparing for a phase 3 study.

So far 11 patients with Lewy body dementia (LBD) have been treated with nelotanserin, which is being developed to tackle the visual hallucinations and sleep disturbances that can afflict people with these diseases.

The results have outpaced expectations, with nelotanserin achieving a significant improvement in extrapyramidal symptoms—Parkinson's disease-like effects caused by the nerve degeneration in LBD—as measured by the Unified Parkinson's Disease Rating Scale (UPDRS), said the biotech.【閱讀全文】

Cold Spring Harbor scientists discover protein driver of prostate, lung cancer

A protein called PTEN is often called an “anticancer” protein, because it prevents tumor cells from growing out of control. Mutations in the gene that encodes PTEN are commonly found in prostate cancer, lung cancer and other tumor types. But some patients with abnormally low levels of PTEN don’t carry any mutations in that gene—raising the question of what may be causing the protective protein to break down in those people.【閱讀全文】

Ferring strikes pact to use Foresee controlled-release tech

Ferring Pharmaceuticals has struck a deal with Foresee Pharmaceuticals. The agreement will see Ferring fund development of a long-duration peptide drug based on Foresee’s delivery technology and potentially pick up the resulting asset if it meets expectations.

Foresee has landed the deal on the strength of its stabilized injectable formulation (SIF) platform, a technology the Taiwanese drugmaker developed to enable controlled-release delivery of peptides, small molecules and proteins. Ferring penned a deal to use the technology to create a formulation of an as-yet-unidentified peptide after assessing the progress made by Foresee to date.【閱讀全文】

Sage Therapeutics Lifts Investor Spirits With Sliver of Depression Drug Data

Sage Therapeutics (SAGE) treated 13 patients suffering from depression with an experimental drug known as SAGE-217. After two weeks, 11 of the patients saw their depressive symptoms cut in half. In eight patients, their depression went into complete remission.

The sliver of new data, announced Monday, show enough potential to advance SAGE-217 into a placebo-controlled mid-stage study in patients with a major depressive disorder, the company said.【閱讀全文】

Aviragen Therapeutics slammed again by its second trial failure in days, this time for their lead drug

In back-to-back setbacks, Aviragen Therapeutics $AVIR says that its lead drug vapendavir flunked a Phase IIb in moderate to severe asthmatics with a rhinovirus infection.

Neither of the two doses in the study – 264 mg or 528 mg – achieved a statistically significant outcome on the primary endpoint.

Just days ago in a Q2 call CEO Joseph Patti called this drug the biotech’s most advanced anti-viral.【閱讀全文】