"Pivotal clinical trial will begin in first half of 2022"...these were the opening words to the press release Aytu BioPharma issued on December 13, 2021, announcing the FDA Clearance of their Investigational New Drug (IND) Application for enzastaurin in vEDS.
For those of us in the vEDS community hoping for an effective, FDA-approved treatment, we've waited what feels like an eternity to hear these words. With this announcement, Aytu BioPharma is allowed to proceed with a clinical trial that could deliver the first FDA-approved treatment for vascular Ehlers-Danlos Syndrome. Although there's still a long way to go, I'd like to share a bit more of the history of how we got here.
A brief history
John and I first heard about exciting research out of Dr. Hal Dietz's lab at Johns Hopkins in 2019. The research was conducted in a mouse model, but showed very promising results in life expectancy. In the results, 80% of mice treated with enzastaurin survived after 40 days of treatment compared to only 50% of untreated vEDS mice. You can read more about the research and results on our Clinical Trials page.
We learned that a biopharma company, Rumpus Therapeutics, was exploring getting the treatment to patients and we contacted them. We always felt that if any researcher or company was interested in investing in studying a treatment for vEDS, we would help support that research, however we could.
Over the following months and years, we helped connect them with others in the patient community, we provided input into what patients want from a clinical trial, and I joined them in meeting with the FDA to discuss the potential study and its importance to vEDS patients. In April 2021, Rumpus Therapeutics was acquired by Aytu BioPharma, ensuring the resources necessary to take the study all the way to a trial.
Why it matters
At FIGHT vEDS, we help connect patients with genetic testing options and, naturally, many patients come to us after they've been diagnosed with vEDS. One of the hardest things to tell patients is "there is no FDA-approved treatment". Although there is a long way to go, and we don't know how well the treatment will perform in the trial, this is a major step forward.
Ultimately, patients need to decide whether they want to participate in any study, but we're excited that Aytu will be making this option available to vEDS patients.
How to learn more
And if you haven't confirmed that you have vEDS, we'd encourage you to check out our Genetic Testing page, where you can learn more about how to get tested for vEDS from home. A genetic confirmation will be an important qualification to enroll in this or any other vEDS trial.