Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) announced  that it has successfully completed enrollment in the M-STAR study, an international Phase 3 clinical trial evaluating the safety and efficacy of verdiperstat in multiple system atrophy (MSA). The Coalition provided an information session on verdiperstat and the clinical trial at the 2019 Patient and Family Conference.  Several attendees at the conference have been involved in this study.  You can read the entire press release here, which includes a statement from the chair of the Coalition board, Cyndi Roemer.  We are grateful for patients who have chosen to participate in this research.  

Questions and Answers from Biohaven

1.Will new participants be able to enroll in the study?

After the study is fully enrolled, unfortunately not. The activation of sites in Europe, along with the phased reopening of US sites that had been paused due to COVID-19, has resulted in a recent high rate of screening and enrollment. Due to the competitive enrollment (and low screen failure rate), we are unable to hold spots for scheduled patients, and we are also unable to over-enroll. However, we are committed to allowing all patients who are screened before enrollment closes to proceed with randomization, as long as they meet protocol eligibility criteria.

2. What does this mean for participants who are currently enrolled in the study?

Nothing would change for these patients. They will continue to participate in the M-STAR study and help advance a potential treatment for MSA.

3. When will the study be completed?

Study participation is about 11 months per patient. We expect all patients to have finished this blinded study by July of 2021.

4. When will the study results be available?

We expect topline results by late 2021.

Expanded Access
Expanded Access also known as "Compassionate Use", is a pathway through which patients with serious diseases may be able to obtain access to an investigational drug or other therapy outside of a clinical trial.

A qualified physician would need to request access to the drug from the drug company on behalf of a patient.  The drug company may agree to allow access if certain conditions are met.  In the US the Food and Drug Administration (FDA) oversees this program.

You can learn more about the program at the FDA website.
https://www.fda.gov/news-events/public-health-focus/expanded-access


Biohaven Pharmaceuticals provides detailed information on their expanded access policy at their website:
https://www.biohavenpharma.com/science-pipeline/resources/early-access-programs

The Multiple System Atrophy Coalition is happy to recognize the Americans with Disabilities Act (ADA) on its 30th anniversary. On July 26th we celebrated this important civil rights law that prohibits discrimination against individuals with disabilities in all areas of public life, including jobs, schools, transportation, and all public and private places that are open to the general public. The purpose of the law is to make sure that people with disabilities have the same rights and opportunities as everyone else.

Did you know that the ADA National Network is a free resource that provides information and training on the Americans with Disabilities Act? Learn more about the resources you can access here. 

Image credit: ADA National Network [adata.org] #ADA30 #ThanksToTheADA