Biotech
So is NASH over then?
The triumph of novel weight-loss drugs at ADA went beyond obesity, including early-stage data showing that an experimental medicine from Eli Lilly had dramatic effects on nonalcoholic fatty liver disease, leading to a complete resolution for 93% of patients taking the highest dose.
What does that mean for NASH, the advanced form of fatty liver disease that leads to tissue scarring? Akero Therapeutics, Madrigal Pharmaceuticals, and Viking Therapeutics — companies working on potential blockbuster treatments for NASH — each saw their share prices fall by about 15% this week as investors appeared to reset their expectations in light of the ever-expanding utility of novel obesity drugs.
The reaction is probably a little hasty. Lilly's drug had a marked effect on liver fat, but patients in the study had little or no tissue scarring at the outset of the trial, meaning it's not clear the drug will help the millions of people who have progressed from fatty liver disease to NASH. To the extent Lilly's data suggest a risk to companies working in NASH, it's not that widespread use of potent obesity drugs would supplant their treatments but rather reduce the number of patients who become eligible for them in the first place.
GENE THERAPY
BioMarin's day is finally here
After two years of rejection and delay, BioMarin is finally poised to win FDA approval for its hemophilia gene therapy, a cornerstone to the company's goal of achieving sustainable profitability for the first time in its 25-year history.
On Friday, the FDA will hand down a final decision on Roctavian, a one-time treatment for hemophilia A that, in clinical trials, dramatically reduced bleeding episodes and helped patients live without the blood transfusions used to treat the disease. The FDA first rejected Roctavian in 2020, asking BioMarin to amass more data on its safety and long-term durability, and then delayed its decision again to account for additional evidence. European regulators approved it in August.
U.S. approval for Roctavian would bring a long-awaited treatment option for patients with hemophilia A. It would also mark a turning point for BioMarin, which has successfully developed seven medicines for rare diseases but never managed to consistently turn a profit.
Regulatory
Regeneron's grand plan hit a snag
Despite all its success developing new drugs over the last two decades, Regeneron Pharmaceuticals still relies on the ocular treatment Eylea, first approved in 2011, for about half of its corporate revenue. With competition mounting and biosimilars arriving as early as next year, Regeneron came up with the cunning plan of simply giving people more Eylea less often, gathering data on an 8 mg dose of a drug that has shipped for years at 2 mg and filing for FDA approval. Ideally, Regeneron could protect its cash cow for a few more years and give patients a more convenient medicine in the process.
The FDA had other thoughts. Yesterday, the agency rejected high-dose Eylea, Regeneron said, because of "an ongoing review of inspection findings at a third-party filler." The FDA didn't take issue with the drug's safety, efficacy, or manufacturing, according to the company, and it's not requesting any new clinical trials.
That makes the rejection sound like a remediable formality, but Regeneron did not specify when it expects to be able to get high-dose Eylea back on track. That's an issue, as each passing day brings Eylea closer to biosimilar competition. Regeneron even redeemed an FDA voucher to speed up the agency's review, a move that could be negated by the rejection. The company's share price fell about 8% yesterday, subtracting more than $6 billion from its value.
No comments