| | | |   | Hello, everyone. Damian here with the results of weighty FDA meeting, a curveball in immunotherapy research, and an update one of last year's bigger biotech scandals. | | | What are the limits of ‘regulatory flexibility’? We’re going to find out this summer when the FDA makes a final decision on Amylyx Pharmaceuticals’ treatment for ALS, which got a split decision from a panel of agency advisers yesterday.
Six of the independent experts voted that Amylyx’s data were not sufficient to conclude the therapy was an effective treatment against ALS, while four said that they were. The vote came after a full day of discussion in which FDA officials questioned Amylyx’s results, company scientists defended the drug, and ALS patients and caregivers made passionate pleas for the agency to approve the drug.
If the FDA was looking for a clear signal from its independent advisers, yesterday’s meeting did not provide it. Now it’s up to agency staff, who have promised to make a final decision on Amylyx’s application by June 29.
Read more. | The latest craze in immunotherapy takes a blow The drug industry’s quest to find the next blockbuster cancer immunotherapy got a little more complicated yesterday, after Roche disclosed that its investigational medicine failed in a late-stage trial.
As STAT’s Adam Feuerstein reports, Roche’s drug, aimed at a promising molecular target called TIGIT, did not slow tumor growth in a study enrolling patients with small cell lung cancer. In the study, the combination of Roche’s anti-TIGIT antibody and the PD-L1-blocking treatment Tecentriq proved no better than the older therapy on its own.
The issue might have been trial design, as the study’s patient population included people with low levels of the PD-L1 protein, a population for whom immunotherapies often don’t work. But the result could prove ominous for the entire TIGIT field, which includes Merck, Gilead Sciences, GlaxoSmithKline, and Bristol Myers Squibb.
Read more. | Lessons for navigating the frontiers of medical science This is a defining decade for the future of healthcare. Right now, being at the forefront of drug development is an exciting place to be, but a challenging one. Not only are the stakes high — patients are waiting — but there are also often no precedents and no well-trodden paths to rely on. Learn more as Astellas’ chief medical officer, Dr. Bernie Zeiher, shares his five key lessons for navigating the new frontiers of science.. | The Athira story has taken a curious turn Last we heard from Athira Pharma, the company had parted ways with its CEO after an internal investigation concluded that she had committed research fraud. Now one of the company’s major shareholders wants to depose the new chief executive and put himself on the board.
As Reuters reports, Richard Kayne, who owns about 5% of the company, is seeking two board seats, writing in a note to shareholders that current chief Mark Litton is “not the right CEO for Athira.” Earlier this month, Kayne started a venture firm with Leen Kawas, the former Athira CEO who resigned in last year’s data-manipulation scandal.
In a statement, Athira said the company is fine without Kayne on its board, adding that he has “pushed Athira to resume a formal relationship” with Kawas and, after weeks of dialogue, “has rejected our attempts to find common ground.” | A once-promising idea to treat anemia might never work out The FDA has now rejected two similar treatments for anemia over safety concerns, casting serious doubt on what once looked like a lucrative new class of medicines.
Yesterday, Akebia Therapeutics said the FDA turned down its approval application for vadadustat, a drug designed to simulate a low-oxygen environment and thereby compel the body to produce more red blood cells. The agency’s decision comes about seven months after it rejected a similar treatment from partners Fibrogen and AstraZeneca. In both cases, the FDA asked for new clinical studies, putting off any potential approval for a matter of years.
Akebia’s stock price fell by about 70% on the news. In a statement, the company said it continued to believe “the data are supportive of a positive benefit-risk assessment” of vadadustat but did not disclose plans for future trials. | More reads - With pharma still struggling in Alzheimer’s, health tech companies want a swing. STAT+
- Thiel-backed venture firm seeks to nominate candidates for Adagio's board. Reuters
- CVS, Teva, and Allergan reach deal with Florida over the opioid crisis. STAT+
- Obesity can turn a helpful immunotherapy into a harmful one, mouse study shows. STAT
| Thanks for reading! Until tomorrow,  | | | |
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