Last week, FRAXA co-founders Katie Clapp and Mike Tranfaglia, spoke virtually at the 5th Pharma Pricing Reimbursement and Market Access 2021 conference.
In this session, facilitated by Nadia Bodkin, PharmD, MS, from the Rare Advocacy Movement, Katie and Mike were joined by Christopher U. Missling, PhD, President and CEO of Anavex Life Sciences Corp to discuss the collaboration between FRAXA and Anavex as a case study example to help raise awareness amongst others in the rare disease industry of these types of collaborations between advocacy and industry.
FRAXA provides a new framework in which a community-based organization is able to effectively collaborate with the biopharmaceutical drug developer and vice versa at the preclinical stage of drug development. Ms. Bodkin shared, “there’s this old, extremely outdated, preconceived notion within the drug development industry that opportunities for patient centric collaborations don’t really exist until the commercial phase of drug development and to incorporate the patient perspective into clinical trial design and protocols. FRAXA went a step further and established a preclinical collaboration which is extremely novel in today’s environment.”
When discussing the pharmaceutical side of FRAXA-DVI, Dr. Missling expressed, “FRAXA has the best model developed because they’re really intrinsically interested in developing a drug for exactly that unmet need for that disease. The data was so phenomenal that we just were able to publish this data, last month, and in a peer reviewed paper. It really shows that the drug did reverse the symptoms of the Fragile X in animal models and that led us now to move ahead with planning a clinical study in Fragile X. Without FRAXA we wouldn’t be here talking and providing this data.”
FRAXA-DVI is a brilliant example of preclinical collaborations that are now setting the scene for the clinical setting and actual clinical participation from the community. The overall goal of preclinical collaborations like this is to encourage and speed the development of new, safe, and effective therapies for rare conditions like Fragile X syndrome with high unmet medical needs.
