Talditercept alfa

Biohaven Ltd. reported Q1 2023 financials, highlighting progress in drug development, including a TYK2/JAK1 inhibitor for brain disorders, Kv7 activation for epilepsy and bipolar disorder, and myostatin inhibition for SMA. The company anticipates IND submissions and Phase 1 study initiations in 2023, aiming to address unmet medical needs in neurology and neuropsychiatry.

BioAge Labs halted azelaprag trials due to safety concerns; Biohaven's taldefgrobep alfa showed muscle-preserving effects in failed SMA trial, prompting obesity study. Roche's tiragolumab failed phase 3, dimming anti-TIGIT drug prospects; Gilead/Arcus' domvanalimab shows promise. AbbVie's emraclidine failed schizophrenia trials, impacting market cap. Cassava's simufilam failed Alzheimer's phase 3, amid SEC investigation.

Taldefgromab alfa did not meet primary end point in RESILIENT-SMA study but showed efficacy in biomarker-defined subgroups, prompting Biohaven to accelerate it into a phase 2 study for obesity. Caucasian patients showed a 2.2-point change in MFM-32, compared to 1.1-point change in placebo group. Taldefgrobep reduced total body fat mass and increased lean muscle mass, suggesting broader potential in treating obesity.

Biohaven announced taldefgrobep alfa's failure to achieve statistical significance in treating SMA at week 48, but noted clinically meaningful motor function improvements. The RESILIENT SMA study, a Phase III trial, found taldefgrobep beneficial in a majority subgroup, with significant reductions in body fat mass and increased lean muscle mass. Biohaven plans to continue data analysis and engage with the FDA.

Biohaven's phase 3 trial of taldefgrobep alfa, targeting myostatin for spinal muscular atrophy (SMA), failed to meet primary endpoint. Despite some motor function improvement in treatment group, it was not significantly greater than placebo. Caucasian ancestry and measurable myostatin at enrollment showed better results. Biohaven explores taldefgrobep for obesity, with phase 1/2 showing significant reduction in fat mass and increase in lean muscle and bone density.

H.C. Wainwright raised Scholar Rock's price target to $50, maintaining a Buy rating, following Biohaven's Phase 3 RESILIENT study failure for SMA treatment. Scholar Rock's apitegromab sees increased global sales projection to $1.12 billion by 2034. Despite the setback, efficacy signals in subgroups suggest potential for SMA treatment advancements.

Biohaven updates taldefgrobep alfa development for SMA and obesity, noting clinically meaningful improvements in motor function but no statistical separation on primary outcome. Signals of efficacy observed in subgroups, with notable benefits in Caucasian populations and on body composition changes, suggesting taldefgrobep's potential in broader obesity treatment.

Biohaven's muscle-preserving drug, taldefgrobep alfa, showed promise in body composition changes but failed to improve motor function in a Phase 3 SMA study. The company plans to advance the drug for obesity treatment. Biohaven's broader SMA patient population and study design differences contributed to the negative result, contrasting with Scholar Rock's successful SMA trial. Both companies now focus on obesity, where myostatin inhibitors are seen as beneficial.

Biohaven's taldefgrobep alfa failed to significantly improve motor function in SMA patients but showed efficacy signals in Caucasian subgroups. The company plans to discuss next steps with the FDA and advance taldefgrobep alfa in obesity trials, noting significant reductions in body fat mass.