JUNE 1, 2021
Defeat MSA Alliance Research Program Advisors:
Important Questions & Answers from Biohaven
RE: Clinical Trial on Verdiperstat
Can I still join the trial?
Answer: Enrollment for the phase 3 study closed in July of 2020.
When are we expected to know the results of the Phase 3 study?
Answer: Biohaven’s expectation is that Top-Line results will be available by the end of the third quarter in 2021.
If the results are positive, what happens next?
Answer: If the Phase 3 study results are positive and show that verdiperstat is effective, then Biohaven will engage with the FDA to discuss next steps.
Is there anything we can do as patients or caregivers to help?
Answer: Continued advocacy work to raise awareness about MSA, including diagnosis and treatment gaps, as well as the burden of the disease on patients and families.
What is the plan for patients who live outside the United States?
Answer: If the Phase 3 study results show a statistically significant effect of slowing disease progression with Verdiperstat, Biohaven will engage with the health authorities in Europe and other countries to discuss next steps.
How does the drug work?
Answer: Verdiperstat is believed to inhibit an enzyme called myeloperoxidase that catalyzes the production of cytotoxic compounds. Buildup of these reactive compounds leads to brain tissue damage and neuronal death. Verdiperstat binds to Myeloperoxidase and is believed to reduce the production of reactive compounds and protect nerve cells from damage.
Learn more in this video: https://vimeo.com/542176986
Could you tell us anything about how many people have been in the Phase 3 study and where they are from? In the United States or outside of the United States?
Answer: There are 336 people in the blinded phase 3 trial; half are on placebo and half are on verdiperstat. 35% of patients are in the E.U. (Austria, France, Germany, Italy and UK) and 65% are in the United States.
If people who are taking verdiperstat have questions or concerns, who should they contact?