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BioCentury Extra
As published Monday, November 30, 2015 7:11 PM PST

  • NAS to co-host gene editing meeting in D.C.

    The U.S. National Academy of Sciences, the U.K.'s Royal Society and the Chinese Academy of Sciences will host a meeting on Dec. 1-3 in Washington, D.C., to discuss the scientific, ethical and governance issues associated with human gene editing research.

    The international summit is one component of the Human Gene-Editing Initiative, which NAS and the National Academy of Medicine launched in May in response to public concerns over the use of CRISPR-Cas9 (CRISPR-associated protein 9) gene editing to modify human embryos. In April, a group of Chinese researchers published a study characterizing the use of CRISPR/Cas9 in non-viable human embryos (see BioCentury Innovations, June 18).

    In advance of the meeting, Jennifer Doudna and George Church published separate perspectives Monday in Nature that discussed whether human germline editing research should be allowed. Doudna is one of the original inventors of CRISPR-Cas9 gene editing technology, a co-founder of gene editing company Caribou Biosciences Inc. (Berkeley, Calif.) and a University of California Berkeley professor. Church is a core faculty member of the Wyss Institute for Biologically Inspired Engineering at Harvard and a professor of genetics at Harvard Medical School.

    In Doudna's perspective, she said not enough is currently understood about gene editing technologies to allow clinical applications of human germline editing, but research should not be completely banned. "We do not yet know enough about the capabilities and limitations of the new technologies, especially when it comes to creating heritable mutations," wrote Doudna.

    Church's perspective took a stronger position in favor of human gene editing research to prevent disease transmission. "Banning human-germline editing could put a damper on the best medical research and instead drive the practice underground," he wrote.

    Editas Medicine (Cambridge, Mass.), Intellia Therapeutics Inc. (Cambridge, Mass.) and CRISPR Therapeutics AG (Basel, Switzerland), were all founded within the last two years to develop CRISPR-based therapeutics, and all three companies are focused on gene editing in somatic cells.

  • BMS gets nod for Empliciti, denial for Opdivo

    FDA approved Empliciti elotuzumab from Bristol-Myers Squibb Co. (NYSE:BMY) and AbbVie Inc. (NYSE:ABBV) to treat multiple myeloma (MM). The agency also issued a complete response letter for BMS's Opdivo nivolumab as a single agent for previously untreated patients with BRAF V600 mutation-positive unresectable or metastatic melanoma.

    FDA approved Empliciti in combination with Revlimid lenalidomide from Celgene Corp. (NASDAQ:CELG) and dexamethasone to treat MM in patients who have received one to three prior therapies. BMS, which is to lead commercialization of the drug, plans to launch Empliciti within two days at a wholesale acquisition cost (WAC) of about $10,000 per month, or $142,000 for the first year of treatment including a two-month induction period. The approval came three months ahead of Empliciti's Feb. 29, 2016, PDUFA date.

    The humanized mAb targeting SLAM family member 7 (SLAMF7; CS1) is the third MM treatment FDA has approved this month, after Ninlaro ixazomib from Takeda Pharmaceutical Co. Ltd. (Tokyo:4502) and Darzalex daratumumab from the Janssen Biotech Inc. unit of Johnson & Johnson (NYSE:JNJ) and Genmab A/S (CSE:GEN; Pink:GMXAY). Darzalex, a human IgG1k mAb against CD38, was the first monoclonal antibody approved to treat MM (see BioCentury Extra, Nov. 16).

    Empliciti is under accelerated assessment in the EU.

    BMS said FDA requested additional data from studies of Opdivo in BRAF-mutated patients. The human IgG4 mAb against PD-1 is approved as a single agent and in combination with BMS's Yervoy ipilimumab for patients with previously untreated BRAF V600 wild-type advanced melanoma. Opdivo also is approved to treat advanced renal cell carcinoma, metastatic squamous and non-squamous non-small cell lung cancer (NSCLC) and advanced melanoma (see BioCentury Extra, Nov. 24).

  • 3SBio takes majority stake in CP Guojian

    3SBio Inc. (HKSE:1530) acquired a majority stake in biosimilar company Shanghai CP Guojian Pharmaceutical Co. Ltd. (Shanghai, China). 3SBio paid RMB1.4 billion ($213.3 million) to raise its stake to 54%. 3SBio had acquired a 7% stake in CP Guojian last year (see BioCentury, March 2).

    According to a regulatory filing, 3SBio acquired equity in CP Guojian from four shareholders, including a 42% stake that it bought from Xizang Hongshang Capital Equity Investment Co. Ltd. for RMB1.1 billion ($164.8 million).

    CP Guojian markets two biosimilars in China: Yisaipu, a biosimilar of Enbrel etanercept from Amgen Inc. (NASDAQ:AMGN); and Xenopax, a biosimilar of Zinbryta daclizumab high-yield process from Biogen Inc. (NASDAQ:BIIB) and AbbVie Inc. (NYSE:ABBV). CP Guojian also has a pipeline of mAbs.

    Enbrel is a recombinant p75 tumor necrosis factor (TNF) receptor linked to the Fc portion of human IgG1 (TNFr:Fc) 1 marketed to treat autoimmune indications. Zinbryta is a humanized mAb against IL-2 receptor alpha chain (CD25) under FDA and EMA review to treat multiple sclerosis.

  • Pfizer, Heptares strike GPCR deal

    The Heptares Therapeutics Ltd. subsidiary of Sosei Group Corp. (Tokyo:4565) entered a discovery partnership with Pfizer Inc. (NYSE:PFE) that would give the pharma exclusive, worldwide rights to therapeutics targeting up to 10 undisclosed GPCRs in multiple therapeutic areas. Pfizer also will purchase a $33 million equity stake in Sosei at a 25% premium to Sosei's average share price over the 20 days prior to the deal's close, expected in December.

    Heptares received an undisclosed upfront payment and is eligible for $189 million in milestones per target, plus royalties. Pfizer will select the targets and will be responsible for development and commercialization.

    Last week, Heptares licensed preclinical migraine candidates targeting calcitonin gene-related peptide (CGRP) to Teva Pharmaceutical Industries Ltd. (NYSE:TEVA) for $10 million up front and $400 million in milestones, plus royalties (see BioCentury Extra, Nov. 25).

    Sosei fell Y300 to Y6,060 on Monday. The Pfizer deal was announced after market close in Tokyo.

  • Teva, Takeda form Japanese generics JV

    Teva Pharmaceutical Industries Ltd. (NYSE:TEVA) and Takeda Pharmaceutical Co. Ltd. (Tokyo:4502) formed a joint venture to market Teva's generics and Takeda's off-patent drugs in Japan. Teva and Takeda will split ownership 51-49%.

    The partners said the venture will combine Teva's generics portfolio with Takeda's distribution network and brand recognition, adding that the business model is consistent with the Japanese government's push to limit health care spending by increasing market penetration for generics.

    The JV will operate as an independent entity with its own management team beginning in 2Q16.

    Teva shed $0.54 to $62.93 on Monday. Takeda fell Y191 to Y5,983. The partners announced the venture after market close in Tokyo.

  • Roche ends antibiotic deal with Polyphor

    Roche (SIX:ROG; OTCQX:RHHBY) said it will discontinue its partnership with Polyphor Ltd. (Allschwil, Switzerland) to develop and commercialize POL7080 to treat bacterial infections caused by Pseudomonas aeruginosa. Roche said its decision to return POL7080 rights to Polyphor was based on its assessment of the compound's progress.

    Polyphor CFO and Head of Corporate Development Adesh Kaul told BioCentury the company will continue its ongoing open-label Phase II PK study of POL7080 to treat ventilator-associated pneumonia (VAP) with confirmed P. aeruginosa and plans to meet with regulatory authorities over the next year to discuss the design of a pivotal trial. POL7080 is a synthetic cyclo-peptide antibiotic targeting the Pseudomonas LPS-assembly protein (OstA; LptD; Imp).

    In 2013, Polyphor granted Roche exclusive, worldwide development and commercialization rights to POL7080 in exchange for CHF35 million ($38.3 million) up front and up to CHF465 million ($509.2 million) in milestones, plus double-digit royalties. At the time, the molecule was in Phase II testing.

    The deal marked Roche's return to the antibiotics sector (see BioCentury, Nov. 18, 2013).

    Roche since has formed other antibiotics partnerships (see BioCentury Extra, Aug. 13).

  • amfAR establishes HIV institute at UCSF

    The Foundation for AIDS Research (amfAR) formed the amfAR Institute for HIV Cure Research at the University of California San Francisco with a five-year, $20 million grant. The funding is part of a $100 million amfAR initiative announced in February, with a goal of finding the scientific basis of an HIV cure by YE20 (see BioCentury Extra, Feb. 20).

    amfAR said the institute will conduct both basic research and clinical studies. It intends to collaborate with Gilead Sciences Inc. (NASDAQ:GILD) as well as the Gladstone Institute of Virology and Immunology, Blood Systems Research Institute, Oregon Health and Science University, University of California Berkeley and the Infectious Disease Research Institute.

    UCSF Professor Paul Volberding will lead the institute.

  • AB Science's masitinib meets in mastocytosis study

    AB Science S.A. (Euronext:AB) said masitinib (AB1010) met the primary objective of a Phase III study to treat severe systemic mastocytosis.

    The 135-patient, placebo-controlled trial evaluated response rate between week eight and week 24. A response was defined as an improvement of at least 75% in mastocytosis symptoms over baseline.

    The stem cell factor (SCF) receptor tyrosine kinase (c-Kit; KIT; CD117) inhibitor has Orphan Drug designation from FDA and EMA to treat mastocytosis. AB Science has studied masitinib in oncology, progressive multiple sclerosis, severe asthma, amyotrophic lateral sclerosis and Alzheimer's disease.

    AB Science gained EUR 0.42 to EUR 13.09 Monday. It released the news after market close in Paris.

  • Ironwood's linaclotide meets in OIC study

    Ironwood Pharmaceuticals Inc. (NASDAQ:IRWD) said linaclotide (ASP0456) met the primary endpoint in a Phase II trial to treat adults with opioid-induced constipation.

    The trial included 254 patients with chronic, non-cancer pain who had been receiving a stable dose of an opioid analgesic and had fewer than three spontaneous bowel movements (SBM) per week. Once-daily 145 ug and 290 ug doses of linaclotide met the primary endpoint of a greater frequency of SBM over eight weeks vs. placebo.

    Ironwood and Allergan plc (NYSE:AGN) market linaclotide in the U.S. as Linzess to treat irritable bowel syndrome with constipation (IBS-C) and chronic idiopathic constipation (CIC). The partners intend to determine next steps in the OIC development program after reviewing data expected in 2H16 from a Phase IIb trial of a colonic-release formulation of linaclotide in adults with IBS-C.

    Almirall S.A. (Madrid: ALM) has rights from Ironwood to develop and commercialize the guanylate cyclase C (GCC; GUCY2C) agonist in Europe, where it markets the product as Constella for IBS-C.

    Last week, Allergan agreed to merge with Pfizer Inc. (NYSE:PFE) (see BioCentury Extra, Nov. 23).

  • Valneva's antibacterial vaccine meets in Phase II

    Valneva SE (Euronext:VLA; VSE:VLA) said its VLA84 vaccine met the primary endpoint in a Phase II study to prevent Clostridium difficile infection.

    The study included 500 healthy volunteers who received a 75 ug intramuscular vaccine dose without an alum adjuvant, a 200 ug dose with or without the adjuvant, or placebo, administered at days 0, 7 and 28. Valneva said the vaccine induced immune responses to both C. difficile toxins A and B at all doses, and said the 200 ug dose without alum led to the highest seroconversion rate against both toxins A and B on day 56.

    Valneva will continue to monitor immune response and safety until day 210, and expects to complete the study in 2Q16. Valneva did not respond to inquiries regarding its development timeline.

    Under a 2007 deal, GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) has opt-in rights to develop and commercialize the vaccine after a Phase II trial has been completed, and Valneva retains rights to co-develop the compound and share profits with the pharma or become eligible for milestones and royalties. GSK did not respond to inquiries regarding its option.

    Valneva gained EUR 0.07 to EUR 3.65 on Monday. The Phase II results were released after market close.

  • MSKCC, ICER propose value-based pricing policies

    In an editorial published Monday in the Journal of the American Medical Association, Memorial Sloan Kettering Cancer Center's Peter Bach and Institute for Clinical and Economic Review President Steven Pearson proposed a set of policies private and public payers could adopt to promote the use of value-based drug pricing.

    Bach and Pearson noted that in the U.S., the prescription drug sector is "the only major category of health care services for which the producer is able to exercise relatively unrestrained pricing power." New tools including MSKCC's Drug Abacus and ICER's value assessment reports are being developed to assess the value drugs provide, and other sectors of the healthcare system are moving towards value-based pricing, the authors noted.

    To give companies incentives to use these tools to set drug prices, Bach and Pearson suggested that private payers provide preferred formulary status with lower co-pays (e.g., less than 20% of a drug's price) in cases where the launch price is similar to the value-based price. Drugs whose prices substantially exceed the value-based price could be restricted to non-preferred status with higher co-pays.

    The authors also suggested that Medicare require all plans to cover a drug whose launch price is similar to its value-based price, and that the drug be exempt from federal discount programs such as 340B. Bach and Pearson suggested that these changes could be made under Medicare's existing authority.

    In cases where a drug's launch price exceeds the value-based price, the authors suggested FDA could act "within its current authority" to apply the Priority Review process to potential competitors that could improve outcomes and result in a more affordable product.

    Bach, the director of MSKCC's Center for Health Policy and Outcomes, launched a pilot in June of the Drug Abacus for cancer drugs. The tool allows any user -- whether a payer, a drug company, a physician or a patient -- to calculate a drug's value. The calculation is based on how much the user values additional survival provided by the treatment, and then is adjusted depending on the importance the user places on toxicity and other factors (see BioCentury, July 20).

    Earlier this year, ICER issued reports assessing the value of new PCSK9 inhibitors for high cholesterol as well as heart failure drug Entresto sacubitril/valsartan from Novartis AG (NYSE:NVS; SIX:NOVN) (see BioCentury Extra, Sept. 11).

  • CIRM outlines five-year goals

    The California Institute for Regenerative Medicine outlined its five-year goals for its CIRM 2.0 program and forecast $890 million in spending from 2016-20. The institute began CIRM 2.0 in January to speed operational processes and increase patient engagement and industry involvement in the development of stem cell therapies (see BioCentury Extra, Dec. 15, 2014).

    CIRM estimated that $440 million of its funding budget would go toward clinical projects, $180 million to translational research and $170 million to discovery. It estimated that a total of $100 million will go toward education and infrastructure.

    CIRM said it plans to begin development of 50 new therapeutic or device candidates over the next five years. It is funding 15 clinical trials and has a pipeline of more than 45 translational projects. It aims to increase by 50% the number of projects advancing to the next stage of development, and hopes to find partners for half of its unpartnered clinical candidates by 2020.

    The institute also prioritized reducing regulatory burdens on stem cell research and said it will aim to reduce the time for stem cell therapies to advance to clinical trials by 50%. The institute said it has been working with FDA to reduce regulatory requirements, including eliminating "non-value added" preclinical activities.

    CIRM's science subcommittee voted unanimously on Monday to approve all components of the plan except for one proposal covering application review policies, which will be amended. The full CIRM governing board will vote on the amended plan Dec. 17.

    CIRM was created by a 2004 California ballot measure (see BioCentury, Nov. 8, 2004).

  • Reserve your place for dinner and discuss FDA Waivers for TQT studies

    Learn how Early Precision QT for cardiac safety testing can elevate drug development and lower study costs by supporting an FDA waiver for TQT studies. Hear from Dr. Borje Darpo, CSO, iCardiac and Dr. Emanuel DeNoia, Medical Director at ICON, a preferred provider of Early Precision QT.

    Reserve a spot to these exclusive dinners.

    Thursday, November 5 - Boston

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