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BioCentury Extra
As published Tuesday, October 13, 2015 6:32 PM PST

  • Zafgen continues its fall

    Zafgen Inc. (NASDAQ:ZFGN) fell $6.40 (29%) to $15.75 on Tuesday, its second consecutive day of heavy losses. The obesity company tumbled $12.25 (36%) to $22.15 on Monday and has now lost 54% of its value, or $505.8 million in market cap, over the two-day period. The company did not respond to inquiries and issued no news releases.

    Speculation ranged from a data surprise for weight loss therapy beloranib (ZGN-440) to an M&A deal.

    The company does have later stage data upcoming. On the company's 2Q15 earnings call in August, CMO Dennis Kim told investors Zafgen expects six-month interim data late this year or in early 2016 from a Phase IIb study of beloranib in severely obese patients with Type 2 diabetes. He also said Zafgen expects data in 1Q16 from a Phase III trial of the subcutaneous formulation of a methionine aminopeptidase 2 (MetAP2) inhibitor to treat Prader-Willi syndrome.

    On the call, Kim noted the company looks at blinded safety data "on an ongoing basis," and said it holds DSMB or Safety Review Committee meetings "on a regular basis."

    In a note issued early on Tuesday, RBC Capital Markets analyst Simos Simeonidis said he believed there was a 35% chance that a safety issue has arisen with beloranib, and a 35% chance that the company has data from one of the two trials. He also speculated that Zafgen could be preparing to announce an M&A deal or ex-U.S. partnership, or that CEO Thomas Hughes could be leaving the company.

  • Ultragenyx reports interim LC-FAOD data

    Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) fell $7.40 to $82.49 on Tuesday after releasing interim results from a Phase IIb study of triheptanoin (UX007) to treat long-chain fatty acid oxidation disorders. The company said triheptanoin increased muscle performance and exercise tolerance in cycling and walking tests, but data were insufficient to show effects on liver and cardiac disease. Ultragenyx said that the study's design "limits definitive conclusions about efficacy and safety," but it plans to use the results to determine the appropriate patient population and endpoints of a Phase III study.

    Ultragenyx reported a 60% mean increase in workload in seven patients who participated in a cycle ergometry test after 24 weeks of triheptanoin treatment. It also reported a 28% mean increase in walking distance in the eight patients who completed a 12-minute walk test after 18 weeks. The study included LC-FAOD patients ranging from 10 months to 58 years old.

    The study will evaluate liver/hypoglycemia and cardiac disease in LC-FAOD patients, part of the study's primary outcome measurement. Ultragenyx said there were "limited data" from the five study patients who had those diseases, and it will continue to monitor those patients.

    Ultragenyx said it expects to have 78-week data from the study in 1H16. Also next half, it intends to give an update of its clinical development plan for triheptanoin, a synthetic compound that provides medium-length, odd-chain fatty acids that are metabolized to replace intermediate substrates in fatty acid oxidation and in the tricarboxylic acid (TCA) cycle.

  • Audentes raises $65 million in series C

    Gene therapy company Audentes Therapeutics Inc. (San Francisco, Calif.) raised $65 million in a series C round co-led by existing investor Sofinnova Ventures and new investor Redmile Group. New investors RA Capital; T. Rowe Price; Rock Springs Capital; Cormorant Asset Management; Cowen Private Investments; and Foresite Capital also participated, as did existing investors OrbiMed; 5AM Ventures; Versant Ventures; Deerfield Management; and Venrock.

    Audentes develops gene therapies to treat rare diseases. Its preclinical candidates include AT001, an adeno-associated viral serotype 8 (AAV8) vector delivering the myotubularin 1 (MTM1) gene to treat X-linked myotubular myopathy; and AT002, an AAV9 vector carrying the acid alpha glucosidase (GAA) gene to treat Pompe's disease.

    CEO Matthew Patterson told BioCentury the company expects to begin clinical trials of AT001 and AT002 next year. The company also intends establish a GMP manufacturing facility.

    Last month, Audentes said it would acquire Cardiogen Sciences Inc. for an undisclosed amount. The deal gave it AT003, a preclinical AAV vector-based therapy that delivers calsequestrin-2 (CASQ2) to cardiomyocytes to treat catecholaminergic polymorphic ventricular tachycardia (CPVT) in patients with inherited CASQ2 mutations.

  • Jeb Bush releases healthcare, innovation plan

    Jeb Bush released a healthcare plan Tuesday that seeks to replace the Affordable Care Act with refundable tax credits for catastrophic insurance plans and block grants to states to subsidize insurance for low income individuals. The plan aims to promote innovation by reforming and increasing funding for FDA and NIH. Bush said he would work with state governments to move 17 million Americans who are "entangled in Obamacare" to state-regulated and subsidized insurance plans.

    Bush said he would "modernize the Food and Drug Administration's regulatory morass," and attributed the time and cost required to develop new drugs to FDA's "regulatory mess." Bush outlined his goals for FDA reform, but did not provide details on how they could be accomplished. He vowed to "modernize the tools that the FDA uses to make scientific decisions, including biomarkers, patient-reported clinical outcomes and real-world data; accelerate clinical trials by utilizing patient data as it accumulates, rather than rigid predetermined timelines, to continuously modify and improve studies; allow manufacturers to share truthful, non-misleading scientific information and peer-reviewed clinical data with physicians and patients; and ensure that the FDA is adequately resourced to make timely approvals of safe and effective innovator and generic drugs."

    While he advocated increasing NIH's budget by an unspecified amount, Bush also criticized the agency for devoting "scarce resources" to "studies on rabbit massages, how Buddhism explains the science of meditation and smartphone games to empower parents in vegetable-eating negotiations with their children." NIH awarded three grants totaling $1.5 million from 2009-12 to fund research on the effects of massage that used an in vivo rabbit model of exercise-induced muscle inflammation and weakness. The Buddhism example dates back to the 1990s, when NIH provided $37,000 to a Harvard researcher to study "dissociation in Tibetan Buddhism." From 2009-12, NIH awarded $1.5 million to a video game company to develop an iPhone app that trains parents how to persuade a child to eat more vegetables.

  • Controlled distribution under renewed antitrust scrutiny

    New York's Office of the Attorney General asked Turing Pharmaceuticals AG (New York, N.Y.) to respond to reports that the company intentionally restricted its distribution scheme for Daraprim pyrimethamine to prevent generic manufacturers from performing bioequivalency studies. In a letter to Turing, Antitrust Bureau Chief Eric Stock wrote that Turing's actions "may be restraining competition unlawfully."

    In September, media reports that Turing increased the price of Daraprim from $13.50 per pill to $750 spurred a backlash (see BioCentury, Sept. 28).

    Last week, Sen. Amy Klobuchar (D-Minn.) called on the Federal Trade Commission to investigate whether "Turing or any other pharmaceutical company" that restricts drug distribution is doing so in a manner that violates antitrust laws.

    Turing did not respond to inquires seeking comment on the antitrust issue.

    In April 2014, Mylan N.V. (NASDAQ:MYL) sued Celgene Corp. (NASDAQ:CELG) for limiting access to Thalomid thalidomide and Revlimid lenalidomide. Mylan claimed that Celgene used its restricted distribution REMS program as a pretext to prevent Mylan from obtaining samples of the drugs for bioequivalence studies (see BioCentury Extra, April 4, 2014).

    FTC filed an amicus brief in June 2014 saying that actions that block generic access may violate antitrust laws. "Celgene's view that it has a virtually absolute right to block access to the samples generic firms need to compete threatens to foreclose these cheaper alternatives, perhaps indefinitely," the brief reads.

    The lawsuit is pending.

  • NICE launches Office of Market Access

    The U.K.'s NICE launched the Office for Market Access (OMA), an initiative that aims to speed the adoption of new medicines and technology by NHS. The institute said OMA's team will provide industry stakeholders with "tailored support" to advance products through NICE, and that it will be able to connect regulators, research and NHS organizations, government departments and healthcare trade associations.

  • Correction

    Innovent Biologics Inc. (Suzhou, China) is eligible for over $400 million in milestones for its preclinical PD-1 mAb under a March deal with Eli Lilly and Co. (NYSE:LLY). The deal also includes a second undisclosed molecule from Lilly, as well as the pharma's biosimilar of Rituxan rituximab from Roche (SIX:ROG; OTCQX:RHHBY). The Oct. 12 BioCentury Extra misstated which product is tied to the milestones. It also misidentified the second Lilly molecule.

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