| | | |   | Hello, everyone. Damian here with a preview of the next big FDA controversy and a gut check on the latest bright idea in oncology. | | | Sage’s depression drug worked. Now what? Sage Therapeutics’ novel depression treatment met its primary goal in a pivotal study, the company said yesterday, but the magnitude of the drug’s effect leaves plenty of room for debate over its commercial future.
As STAT’s Adam Feuerstein reports, Sage’s zuranolone had a statistically significant effect on patients’ depression symptoms after three days, meeting the trial’s primary endpoint and supporting the company’s belief that it can serve as a fast-acting antidepressant. However, after 15 days, zuranolone was numerically better than placebo but not by enough to rule out random chance.
Sage’s share price fell about 17% on the news, reflecting investors’ concerns that the data, while perhaps sufficient to satisfy the FDA, aren’t impressive enough to convince physicians to eventually prescribe zuranolone.
Read more. | The FDA’s next big controversy is right around the corner Robert Califf will be just a few weeks into his second stint as FDA commissioner when the agency takes on its next potentially explosive debate: whether to approve a new treatment for ALS.
The drug is AMX0035, developed by a company called Amylyx Pharmaceuticals. Its FDA filing is based on a single study, completed in 2020, that showed a significant slowing of disease progression in patients with ALS. The agency initially balked at the evidence, asking the company to complete another trial, but later relented and invited Amylyx to submit for approval while preparing a confirmatory study, according to the company.
On March 30, the FDA’s neurology advisory panel — the same one that voted against approving Aduhelm — will convene to discuss the merits of AMX0035. It remains to be seen how the panelists will interpret Amylyx’s data, but it’s certain ALS patient advocates, who have long accused the FDA of holding potential therapies to an unfair standard, will be a vocal force at the meeting.
The stakes are high for Amylyx, which went public at a roughly $1 billion valuation last month and has just the one drug. Earlier this month, analysts at Evercore ISI estimated AMX0035 could be a $1 billion drug at its peak, which would make Amylyx a nearly $3 billion company. But any delay to tis approval might cut that valuation in half, according to Evercore, meaning March 30 might be a volatile day for Amylyx. | AAV vector integration and genome editing are among more than a dozen in-depth workshop topics The workshops preceding ASGCT’s 25th Annual Meeting offer deeper knowledge and insight on a variety of gene and cell therapy topics. Choose from 13 morning and afternoon sessions presented by experts representing industry and academia. Workshops will take place on Sunday, May 15, with virtual or in-person registration options that include live streaming and on-demand access. Save $50 with early-bird pricing through February 28. Register today. | Is oncology’s latest hot target for real? Cancer, in its endless quest to outfox the human immune system, has learned how to hijack a helpful bodily protein and exploit it to evade detection. After years of research and a few eurekas in the lab, the drug industry is betting billions of dollars that targeting that protein — called TGF-beta — can make for powerful new medicines.
But will it work?
STAT’s Angus Chen explores the promise of TGF-beta in the first installment of "On Target," a regular feature on the brightest ideas in oncology. The short version is that all makes sense on paper: If a tumor is using TGF-beta to hide from the immune system, blocking it should cripple that defense and expose cancer cells to destruction. But as with all things in biology, there’s a wealth of issues to overcome before those billions can pay off.
Read more. | The Covid treatment landscape could get complicated The emergence of Omicron has shifted the landscape of Covid-19 treatments, and the impending arrival of next-gen therapies could make for an unpredictable market in 2022.
The U.S. is buying up doses of the only antibodies that work against Omciron — one from Eli Lilly and another from partners GlaxoSmithKline and Vir Biotechnology — but the potential for future variants has some experts concerned they’ll only be temporary solutions. Pfizer’s powerful oral treatment is expected to do about $30 billion in sales this year, but its lengthy list of counter-indications puts a cap on its utility. And Merck’s competing molnupiravir has considerably weaker supporting data, reportedly putting its future at risk abroad.
That leaves the door open for novel therapies. Novartis, working with Molecular Partners, is seeking U.S. authorization for a drug called ensovibep. In clinical trials, the intravenous treatment showed efficacy on par with antibodies, and it appears to work on all variants of concern. Enanta Pharmaceuticals, maker of approved treatments for hepatitis C, has begun clinical trials with an oral medicine that works similarly to Pfizer’s, and Atea Pharmaceuticals, whose Covid-19 antiviral ran into delays last year, is expecting pivotal data before the end of 2022. | More reads - Biotech startup launches with $155 million to develop pill for chronic hives. Boston Globe
- BioNTech to ship mRNA vaccine factory kits to Africa. Reuters
- By altering the blood type of lungs, researchers raise the possibility of universal organs for transplants. STAT
| Thanks for reading! Until tomorrow,  | | | |
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