The road to every new drug approval is littered with the remains of earlier failures. Biopharmaceutical companies don’t like to talk about the failures. But it’s earnings season now, so some of them don’t have much choice.
Biogen’s (NASDAQ: BIIB) earnings call this week was the first since its Alzheimer’s drug aducanumab failed in a Phase 3 study and eviscerated the company’s shares. CEO Michel Vounatsos acknowledged the drug didn’t work. But he also pointed to moves the company has made to shore up its multiple sclerosis franchise and add drug candidates in other therapeutic areas. And Vounatsos emphasized that Biogen isn’t giving up on Alzheimer’s. Some analysts were unpersuaded. Time will tell which of the company’s programs succeed and which ones end up on the roadside.
In other news this week a drug price watchdog unleashed his thoughts, a headache drug went bust, and an experimental gene therapy faced new questions. Let’s get to those stories and more in our news roundup.
—During a quarterly earnings call this week, Biogen executives tried to assuage investors that it can rebound from the stinging failure of its Alzheimer’s drug aducanumab. They said Biogen would buy back stock and continue to invest in neuroscience (including Alzheimer’s), but it won’t break the bank for big deals.
—The nonprofit Institute for Clinical Economic Review (ICER) has become an important voice in the national debate over high drug prices. Xconomy interviewed founder Steven Pearson about the new era of expensive cell and gene therapies that might provide cures for a range of diseases, and how society might pay for them.
—Fresh questions have emerged about Zolgensma, a gene therapy for spinal muscular atrophy, ahead of its likely FDA approval. Novartis (NYSE: NVS) reported that a patient with Type 1 SMA, the most severe form, died from sepsis in a European trial and that it was “possibly” related to treatment. Autopsy results are pending, but Novartis executives said this week that regulators were “unconcerned” and implicated that asteroid regimen given alongside Zolgensma could be to blame.
—The Merck (NYSE: MRK) immunotherapy pembrolizumab (Keytruda), in combination with Pfizer’s axitinib, won FDA approval as a treatment for advanced kidney cancer. But the drug fell short in a Phase 3 study testing it gastric cancer.
—Gilead Sciences (NASDAQ: GILD) reported that selonsertib, its experimental treatment for the fatty liver disease NASH, failed a second Phase 3 study. The company is hoping for better results from a mid-stage trial of the drug in combination with two other experimental therapies.
—Fremanezumab (Ajovy), a Teva Pharmaceutical (NYSE: TEVA) drug approved for migraines, failed a late-stage study in rarer episodic cluster headaches. The Israel-based drug maker said it would wind down its cluster headache program.
—Bristol-Myers Squibb (NYSE: BMY) reported another immunotherapy setback as a Phase 2 study testing a combo of its two drugs nivolumab (Opdivo) and ipilimumab (Yervoy) against nivolumab alone in head and neck cancers failed.
—AbbVie (NYSE: ABBV) won FDA approval of risankizumab (Skyrizi) for moderate to severe plaque psoriasis. The drug is poised tosucceed the company’s blockbuster adalimumab (Humira), which is facing patent expirations.
—The FDA approved a generic version of the opioid overdose nasal spray naloxone (Narcan). It is also taking steps to speed the review of new generics for opioid overdoses.
LET’S MAKE A DEAL
—The race to treat multiple myeloma with a one-time cell therapy procedure continued this week as Novartis led a $142 million Series C round for San Diego’s Poseida Therapeutics, one of several companies with a CAR-T in human testing for the deadly cancer. A Phase 2 study should start later this year.
—Eli Lilly agreed to pay Avidity Biosciences $20 million and a $15 million equity investment to kick off a collaboration developing new RNA-based immunology drugs.