Dive Brief:
- An experimental, muscle-preserving drug from Biohaven fell short of its goal in a Phase 3 study of people with spinal muscular atrophy, but showed enough promise in its effects on body composition that the company plans to advance it as a treatment for obesity.
- A regimen of Biohaven’s drug, taldefgrobep alfa, and a standard SMA therapy didn’t significantly improve motor function after 48 weeks compared to treatment with a typical SMA medicine and placebo. Biohaven didn’t provide full study data in a Monday statement, but claimed it found “efficacy signals” in certain subgroups and plans to discuss potential paths forward in SMA with U.S. regulators.
- Treatment was associated with positive changes on body composition, Biohaven said. The “overall strength and consistency” of those findings has encouraged the company to “rapidly advance” the drug into a placebo-controlled Phase 2 trial in obesity by the end of the year.
Dive Insight:
Biohaven’s trial is the latest data point in the industry’s search for new ways to preserve muscle, a field of study with implications for many diseases, including SMA and obesity.
For SMA, which causes progressive muscle weakness, muscle preservation as a treatment approach was recently shown to have merit. In October, biotech Scholar Rock said a drug that — similar to taldefgrobep alfa — blocks a protein limiting muscle growth succeeded in a Phase 3 trial in SMA. The results sparked a stock surge for Scholar Rock and validated these so-called myostatin inhibitors as a way to conserve muscle.
The findings also raised investor interest in Biohaven’s drug. While both work similarly, Wall Street analysts cautioned that Biohaven’s results could differ. A big reason why, some wrote, is that Biohaven enrolled a much broader population of SMA patients, including those who are older and less likely to benefit.
The failure “is not a complete surprise given significant differences in the study designs and variability in the patient population,” wrote William Blair analyst Sarah Schram, in a Monday note to investors.
Biohaven isn’t giving up on SMA just yet, though. It pointed to positive results in people of certain ages and ethnicities. It also suggested that imbalances in some genetic factors, as well as very low myostatin levels in some patients at the study’s start, could have contributed to the negative result.
Full findings will be presented at a future medical meeting, Biohaven said.
Still, even the purported benefits Biohaven touted “[don’t] appear that competitive,” wrote Piper Sandler analyst Christopher Raymond. Jefferies’ Michael Yee added that Scholar Rock is now “well positioned and derisked,” while Biohaven has low odds of an approval in SMA.
Scholar Rock shares surged by about one-third Monday morning.
Both companies are on equal footing in obesity, where myostatin inhibitors are seen as a way to conserve muscle in people on weight loss drugs like Novo Nordisk’s Wegovy. Biohaven’s study will start this quarter, while Scholar Rock expects to report Phase 2 data from an ongoing study in the second quarter of 2025.
Raymond, of Piper Sandler, also noted how Biohaven has other opportunities to boost its value in the near term. By the end of the year, it will resubmit an approval application for a drug it’s developing for a type of brain disorder and report data for a treatment in testing for multiple immune diseases.
“Bottom-line, even with the miss today (which we had not modeled anyway), we still recommend shares,” Raymond wrote.
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- Source: https://www.biopharmadive.com/news/biohaven-taldefgrobep-alfa-sma-obesity-drug-results/733849/