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Hello, everyone. Damian here with an update on an erstwhile unicorn, the latest twist in the NASH saga, and the next regulatory debate over ALS.
A former unicorn is stumbling
Sana Biotechnology, a company that raised more than $1 billion to develop a suite of breakthrough medicines, is laying off about a third of its workforce and setting aside one of its most ambitious projects.
As STAT’s Jason Mast reports, Sana is delaying a program that would turn a patient’s own immune cells into cancer-killing assassins or cure blood disorders such as sickle cell with just an IV infusion. The company had hoped to start human trials by 2022 but ran into setbacks with manufacturing. It is now pushing development back further so it has enough funds to focus on other studies.
The move marks Sana’s second round of cuts in less than a year, following the company’s decision to lay off 15% of its staff in November after cutting another development program. The company raised more than $500 million in its 2021 IPO and once commanded a valuation of about $6 billion.
Wall Street’s NASH exuberance is waning
The days when NASH, a prevalent liver disease, looked like the drug industry’s next big thing have long passed. But investors’ reaction to the field’s latest setback suggests Wall Street has substantially curtailed its expectations for a whole class of medicines.
Akero Therapeutics lost 60% of its value yesterday after disclosing that its NASH treatment missed the primary endpoint in a mid-stage study enrolling patients with the most advanced form of the disease. The treatment failed to significantly outperform placebo on a measure of liver scarring but did resolve NASH symptoms, including reductions in liver fat and inflammation, and improve other markers of the disease.
The stock reaction illustrates how the NASH story has changed. While roughly 17 million Americans have some form of the disease, people in the early stages of NASH are likely to be treated with GLP-1-targeting medicines like Ozempic that appear to slow its progression. That means the demand for NASH drugs would be in the later stages of the disease, when patients’ livers are already damaged. Akero’s setback in that population doesn’t doom its drug, but the market seems to think the business opportunity has substantially changed.
Amylyx’s EU do-over is in progress
Amylyx Pharmaceuticals made its case to European regulators at a closed-door meeting yesterday, arguing for a reversal of the body’s earlier recommendation against approving the company’s treatment for ALS.
An influential European Medicines Agency committee issued a negative opinion on Amylyx’s drug in June, after which the company filed an appeal. According to a Mizuho analysis going back to 2013, the EMA has reversed such an opinion only nine times in 36 opportunities.
Potentially working in Amylyx’s favor is the voice of patients. Yesterday’s hearing included people with ALS, according to the EMA’s schedule. An outpouring of patient support for Amylyx’s drug was a factor in the FDA hearing that preceded its U.S. approval, and similar statements could help sway European regulators.
Alnylam is going to have an interesting winter
Monday’s surprise FDA rejection for Alnylam Pharmaceuticals’ heart disease treatment means the company’s future rests on results from a pivotal study that will swing billions of dollars in value.
In early 2024, Alnylam expects to have Phase 3 data detailing whether vutrisiran, a subcutaneous injection, can win approval in ATTR-CM, an increasingly prevalent and progressive heart disease.
If it succeeds, Alnylam’s share price could jump about 75%, Evercore ISI analyst Liisa Bayko wrote in a note to clients. If it fails, the stock could get cut in half, Bayko wrote.
More reads
• Down syndrome families’ fight for access to Alzheimer’s trials, treatments, Reuters
• Bayer invests $250 million in Berkeley site for cell therapies, Bloomberg