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Expert Interview

Slingshot members are talking to an expert! The topic is:

Assessing the RESILIENT SMA Trial Results for Taldefgrobep Alfa in Spinal Muscular Atrophy from Biohaven Pharmaceuticals (BHVN)

Ticker(s): BHVN

Who's the expert?

A neuromuscular specialist or pediatric neurologist with experience in SMA clinical trials and an understanding of myostatin-targeting therapies. The expert should be familiar with motor function assessments (e.g., MFM-32) and advancements in managing SMA through targeted therapeutics.

Interview Questions
Q1.

The RESILIENT study showed clinically meaningful improvements in motor function for subgroups such as those with measurable baseline myostatin. How significant are these findings in shaping the future of SMA treatments, particularly for these biomarker-defined populations?

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Q2.

While the treatment arm did not separate from placebo on the primary outcome in the overall population, subgroups demonstrated improved outcomes on the MFM-32 scale. What does this suggest about the trial design, and how could these insights inform future studies?

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Q3.

Participants with measurable myostatin at baseline showed an enhanced efficacy signal. How important is this biomarker in predicting therapeutic responses, and could it play a role in patient selection for future trials?

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Q4.

Taldefgrobep was the first SMA therapy to demonstrate significant reductions in fat mass along with increases in lean muscle mass and bone density. How do these findings expand the therapeutic potential of taldefgrobep beyond SMA?

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Q5.

The study revealed variability in treatment responses based on racial and genetic factors. How can future SMA trials address these differences to ensure equitable efficacy across diverse populations?

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Q6.

With 97% of subjects continuing into the optional long-term extension, what are the key expectations from this phase, and how critical is it for regulatory engagement with the FDA?

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Q7.

Given the promising effects on body composition, Biohaven plans to advance taldefgrobep into a Phase 2 obesity trial. How might the dual-path development in SMA and obesity influence its potential impact and commercial strategy?

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Q8.

Considering the modest efficacy observed in certain populations, could taldefgrobep serve as an adjunct therapy to existing SMA treatments, and what challenges or opportunities might arise in such combinations?

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