Bio Roundup: Sarepta’s Stumble, Opioid Suits, Shkreli’s Legacy & More

Xconomy National — 

Biotech news tends to slow down in August but the past seven days have been busy for regulatory decisions. Three drugs and two devices won FDA nods.

Not all companies were as fortunate. Several biotechs reported failed clinical trials or unfavorable FDA decisions. The most notable one might be the rejection of a Sarepta Therapeutics treatment for Duchenne muscular dystrophy, golodirsen (Vyondys 53), that appeared to be well on its way to approval. The decision stunned Wall Street analysts. A few even speculated the rejection was more about making a point to Sarepta than Vyondys itself. The rejection stands as another reminder that nothing in the regulatory process is certain—or clear to investors, because the FDA doesn’t release the contents of its rejection letters for the public to see.

In other headlines this week, a “pharma bro’s” former company stumbled, more biotechs joined the IPO queue, two drug makers settled opioid lawsuits, and a next-gen cancer-killing virus moved closer to human testing. Let’s get to those stories and more in this week’s roundup.

DRUG DECISIONS

—The FDA rejected the Sarepta Therapeutics (NASDAQ: SRPT) medicine golodirsen (Vyondys 53), which was widely expected to be the company’s second marketed drug for Duchenne muscular dystrophy. Sarepta said that the FDA was concerned with potential safety problems in animal tests of golodirsen. But several biotech analysts speculated the decision was “payback” for Sarepta’s controversial 2016 approval of eteplirsen (Exondys 51) and an indication that the agency now has a higher bar for Duchenne drugs.

—The FDA approved lefamulin (Xenleta), an antibiotic from Nabriva Pharmaceuticals (NASDAQ: NBRV) for bacterial pneumonia. Xconomy last week previewed some of the challenges Nabriva will face.

—The agency cleared fedratinib (Inrebic), a myelofibrosis drug that Celgene (NASDAQ: CELG) acquired when it bought Impact Biosciences in 2018. Fedratinib is one of several Celgene drugs either at or near market that Bristol-Myers Squibb (NYSE: BMY) cited to justify its $74 billion buyout of Celgene.

—AbbVie (NYSE: ABBV) won an FDA nod for its rheumatoid arthritis drug, upadacitinib (Rinvoq). But it came with a safety warning for potential blood clots, indicative of a “class concern” by the agency for a group of arthritis drugs known as JAK inhibitors, wrote RBC Capital Markets analyst Brian Abrahams.

—The FDA rejected tasimelteon (Heltioz), an experimental Vanda Pharmaceuticals (NASDAQ: VNDA) drug for jet lag disorder. According to Vanda, the agency said the company’s data were “of unclear clinical significance.”

DRUG DEVELOPMENTS

—Jailed “pharma bro” Martin Shkreli helped build the drug pipeline at Retrophin (NASDAQ: RTRX) before his unceremonious ouster from the company—and, later, rise to national infamy for his role in jacking up the price of an off-patent medicine for HIV patients. This week Shkreli’s Retrophin legacy took another hit as a drug he co-invented failed a Phase 3 trial in a rare neurological disease.

—A week after Novartis revealed that it had fired AveXis scientific founder Brian Kaspar in the wake of the Zolgensma data manipulation scandal, Kaspar fired back. In an e-mailed statement, Kaspar’s lawyer said he “categorically denies any wrongdoing” and “stands proudly behind the safety and efficacy of the drug that he and his team worked so hard to develop.” Here’s more from CNBC.

—Meanwhile, a few weeks after defending the company’s actions, Novartis CEO Vas Narasimhan said on a company call that he could’ve handled the situation better, he isn’t perfect, and “we will all keep working on it.” Here’s more from Bloomberg, which cited a report from Swiss newspaper Schweiz am Wochenende.

—Synlogic (NASDAQ: SYBX) stopped a Phase 1b/2a study in hyperammonemia after an early look at clinical data showed the company’s experimental drug did not lower ammonia levels in the blood. Shares fell 35 percent.

—Biogen (NASDAQ: BIIB) and Invitae (NASDAQ: NVTA) launched a new genetic test for spinal muscular atrophy, SMA STAT, that cuts the time for a diagnosis from 21 days to four. The test is being offered for free for U.S. patients with a suspected diagnosis or family history of SMA.

—GlaxoSmithKline (NYSE: GSK) filed for its first approval, in Japan, of anemia pill daprodustat. GSK and several others all developing similar drugs, which are meant to be safer alternatives to injectable anemia medicines.

—Chinese regulators gave one of those rival drugs, roxadustat from FibroGen (NASDAQ: FGEN) and AstraZeneca (NYSE: AZN), its second approval in China.

—Pfizer (NYSE: PFE) is spending $500 million to expand a manufacturing facility in Sanford, NC, that produces emerging gene therapies for Duchenne muscular dystrophy, hemophilia, and more.

DOLLARS & DEALS

Oncorus closed $79.5 million in Series B financing as the Cambridge, MA, biotech prepares for human tests of a virus engineered to kill cancer cells.

—The biotech IPO queue added a few more companies as SpringWorks Therapeutics, Satsuma Pharmaceuticals, IGM Biosciences, and 10x Genomics filed paperwork to go public.

—Rapid growth of 10x’s single-cell sequencing business spurred its IPO filing, but not all is rosy for the Pleasanton, CA, firm. It also faces patent litigation, as the San Francisco Business Times explains.

—Chinook Therapeutics, of Vancouver, BC, hooked a $65 million Series A round to develop drugs to treat rare kidney diseases.

—Venture capital firm Novalis LifeSciences secured $85 million for its first fund, which will invest in eight to 12 companies.

—Graybug Vision of Redwood City, CA, raised $80 million in Series C financing to advance its lead drug into mid-stage tests in the “wet” form of age-related macular degeneration and macular edema.

—Somerville, MA-based Finch Therapeutics closed a $53 million Series C round to continue Phase 3 development of its microbiome treatment for Clostridium difficile infection, as well as Phase 2 testing of an experimental treatment for autism spectrum disorder.

—Juvenescence completed its $100 million Series B round to continue developing anti-aging therapies.

—AstraZeneca agreed to pay $95 million to buy a priority review voucher from Swedish Orphan Biovitrum. SOBI had acquired the voucher earlier this summer as part of its purchase of assets from Novimmune.

—Amgen (NASDAQ: AMGN) is reportedly close to a deal to acquire rare disease drug developer Alexion Pharmaceuticals (NASDAQ: ALXN for $200 a share—a 74 percent premium. The acquisition talks were first reported by Spanish news outlet Intereconomia.

THIS WEEK IN HEALTH POLICY

—In opioid news, Endo Pharmaceuticals (NASDAQ: ENDP) and Allergan (NYSE: AGN) agreed to pay $15 million to settle claims with two Ohio counties and avoid a trial scheduled to start in October. Meanwhile, a decision in Oklahoma’s opioid suit against Johnson & Johnson (NYSE: JNJ) is expected early next week.

—Planned Parenthood left the federal Title X funding program after the Trump administration changed abortion-referral rules.

—The Centers for Disease Control and Prevention are investigating more than 150 cases of a mysterious lung ailment potentially linked to vaping. House Democrats have announced a hearing on the e-cigarette companies’ research and marketing practices.

—Two months after the Veterans Affairs department declined to put the antidepressant nasal spray esketamine (Spravato), from Johnson & Johnson, on its approved list, President Trump said he told the head of the VA to buy “a lot of it.” Trump’s continued public support of the drug deepens questions about connections between him, J&J, the VA, and a group of informal advisers.

—A group of biotech executives initiated an open letter and petition to protest harassment and firings of Chinese scientists in the US that have escalated during Donald Trump’s presidency.

—In the past 10 years, clinical trials leading to cancer drug approvals have rarely included full information about the race or ethnicity of participants, according to a new study. Only 8 percent of trials documented the four major US races. The data also show that black and Hispanic participants were underrepresented compared to the incidence of cancer in their communities.

PEOPLE ON THE MOVE

Former Eli Lilly (NYSE: LLY) executive Levi Garraway was appointed chief medical officer of Genentech, succeeding Sandra Horning, who will retire at the end of the year…. uniQure (NASDAQ: QURE) chief operating officer Scott McMillan is also retiringDavid Marek left Amgen to become chief commercial officer of Axsome (NASDAQ: AXSM)… Frequency Therapeutics appointed Dana Hilt chief medical officerErasca promoted Gary Yeung to chief operating and financial officer, and appointed David Chacko chief business officer… and John McBride was appointed chief financial officer of Cadent Therapeutics.

Ben Fidler and Alex Lash contributed to this report. 

Photo by Flickr user Joel Kramer via a Creative Commons license