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: [[ticker: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: [[ticker: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: [[ticker: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: [[ticker:BMY]]) cited to justify its $74 billion buyout of Celgene.
—AbbVie (NYSE: [[ticker: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: [[ticker: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: [[ticker: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: [[ticker: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: [[ticker:BIIB]]) and Invitae (NASDAQ: [[ticker: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: [[ticker: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: [[ticker:FGEN]]) and AstraZeneca (NYSE: [[ticker:AZN]]), its second approval in China.
—Pfizer (NYSE: [[ticker: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
