Taldefgrobep Alfa (Biohaven) RESILIENT Trial Results and Updates
Taldefgrobep Alfa (Biohaven) RESILIENT Trial Results and Updates
Page last updated: 25th November 2024
Biohaven announces latest RESILIENT Trial data on taldefgrobep alfa
The trial results showed that taldefgrobep alfa did not meet its primary endpoint. It did not show a statistically significant improvement in motor function after 48 weeks compared to a placebo control.
Nevertheless, clinically meaningful improvements in motor function, as determined by the Motor Function Measurement-32 scale (MFM-32), were reported in a large subset of those enrolled in the study.
An analysis on a subgroup of participants revealed significant benefits that may have been influenced by genetic and demographic factors. Examples were:
- SMN2 copy number
- age
- ability to walk
- myostatin levels (a protein involved in limiting muscle growth)
- race.
Biohaven is analysing the data further. They are also consulting with health authorities to determine the next steps.
The trial is in an Open-Label Extension (OLE) phase. This allows all participants to continue receiving taldefgrobep alfa while additional analyses are conducted. The current key findings include:
- Clinically meaningful motor function improvements in most subgroups. This was especially among Caucasian participants and those with measurable baseline myostatin levels (a protein involved in limiting muscle growth that is targeted by taldefgrobep alfa).
- Participants receiving taldefgrobep alfa showed a decrease in body fat, increases in lean muscle mass and bone strength compared to those who received the placebo. This indicates consistent improvements in overall body health.
- Taldefgrobep alfa demonstrated robust engagement with myostatin. It reduced its levels below detection in all those treated over the 48 weeks.
- Taldefgrobep alfa was well-tolerated. There were no treatment-related serious adverse events. Almost all participants (97%) opted to continue in the OLE phase.
Based on these results, Biohaven plans to initiate a Phase 2 obesity study in late 2024. This will use a self-administered autoinjector. Participants are encouraged to discuss any questions about the study with their healthcare teams. Biohaven will provide updates as new analyses are completed.
Further Information
14th March 2023: Biohaven Pharmaceuticals announced that they received Fast Track designation from the U.S. Food and Drug Administration (FDA) for Taldefgrobep alfa.
Fast Track status is granted to drugs that show considerable potential for being able to treat life-threatening diseases, such as SMA. The designation means that the development and review processes of the promising drug can be streamlined in order to speed up translation into the clinic.
25th January 2023: The clinical-stage pharmaceutical company, Biohaven Pharmaceuticals, is recruiting participants in the USA for this Phase 3 trial. It will be randomized, double-blind and placebo-controlled. RESILIENT has been designed to test the efficacy and safety of weekly injections of taldefgrobep alfa given beneath the skin (“subcutaneous”) for 48 weeks.
The trial will be performed with ambulatory and non-ambulatory participants with SMA, aged 4 to 21 years. All enrolees will have all been treated with an SMA disease-modifying therapy (nusinersen, risdiplam and / or onasemnogene abeparvovec) and are anticipated to remain on that same treatment regimen and dose throughout the trial of taldefgrobep alfa.
It is expected that the trial will be completed in early 2025.
Proposed Trial Sites in the UK:
| Resilient Trial of Taldefrobep Alfa | ||
|---|---|---|
All applicants must be:
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| Trial Centre | Current status / Contact | Cut-off date |
| Glasgow | Approved by Pharma company.Discussions taking place with NHS England > | November 2023 |
| Oxford | ||
| Manchester | ||