Biohaven’s SMA drug fails to produce statistically significant results in a late-stage trial

A late-stage study testing the efficacy of Biohaven’s taldefgrobep alpha against a rare neuromuscular disorder has proved disappointing. The experimental treatment failed to meet statistically significant outcomes in the primary endpoint of the study. As a consequence, in pre-market trading, shares of the drug developer were down by 10%.
The trial enrolled approximately 270 ambulant and non-ambulant participants suffering from spinal muscular atrophy (SMA) of any type and aged between 4 and 21 years. Participants in the trial either received a placebo or taldefgrobep for 48 weeks, after which the efficacy of each arm was compared.
Although the treatment could not show a statistically significant difference in improving motor function, at least in clinically significant terms, the RESILIENT SMA study demonstrated that taldefgrobep improves motor function at all time points on the Motor Function Measurement-32 scale (MFM-32).

In addition, in clinically relevant and biomarker-defined subgroups, efficacy signals were observed. These subgroups include age, ambulatory status, baseline myostatin level, and background therapy. When analyzing the ethnicity and race subgroup, it was observed that in 87% of the study population, which comprised Caucasians, treatment with Biohaven’s drug showed clinically meaningful improvements on the MFM-32 at all time points, including Week 48, compared to the placebo and standard of care treatment group.
Myostatin is the pharmacological target of Biohaven’s treatment, and from the above-mentioned group, participants with a measurable baseline of it observed an improved efficacy signal.
While the treatment is designed to block the effects of the proteins myostatin and activin, its effects on preventing the accumulation of muscle fat and muscle wasting are noteworthy.
Results shared by the biotech show that not only does taldefgrobep show numerically larger increases in lean muscle mass and bone density compared to those given a placebo and the standard treatment, but it has also helped participants cut total body fat mass.
These outcomes have inspired Biohaven to direct the experimental treatment toward the indications of weight loss and obesity. The treatment will be advanced as a user-friendly, self-administered autoinjector, and the company plans to set up a mid-stage study in said indications in the fourth quarter of this year.

Taldefgrobep Development Lead and President of Biohaven Ireland, Cliff Bechtold, commented on these developments by saying, “SMA is a devastating rare disease, and although we are disappointed that taldefgrobep did not achieve a statistically significant difference in the broad study population on the MFM-32, we are encouraged that a majority subgroup did show a treatment benefit compared to the placebo arm. Additionally, taldefgrobep demonstrated an important beneficial effect on body composition, which supports our plans to accelerate development in broader populations with obesity.”
Full data from this trial will be shared at an upcoming scientific meeting, and the company also plans to discuss results with the Food and Drug Administration.
SMA is the leading genetic cause of death in infants globally and results in difficulty breathing, swallowing, walking, and talking. Although there are currently three approved treatments for SMA in the U.S. — Novartis’ Zolgensma, Roche and PTC Therapeutics’ Evrysdi, and Biogen’s Spinraza — none of these target muscle development.

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