A major milestone for HHT treatment!

I am reaching out to share the news of a truly momentous milestone for both our organization and our community; the Pazopanib clinical trial has officially launched!

This means Cure HHT is now one of the few patient advocacy organizations in the world that is directly sponsoring a Phase II/III clinical trial of an investigational drug product that we own. I wanted to explain what this means, reflect on how we reached this point, and share why this is so monumental!

Let’s start with a brief look back at how we got here: In 2015, pharmaceutical company Glaxo-Smith-Kline (GSK) owned the drug Votrient™ — which is the market name for pazopanib. The drug is a antiangiogenic therapeutic (stops blood vessel growth) typically given orally to patients with cancerous tumors. During that time, Dr. Marie Faughnan — the director of the Toronto HHT Center of Excellence — led a small, multi-center clinical trial studying pazopanib to treat HHT-related bleeding at a much smaller dose. All 7 patients in that trial showed improvement! Pazopanib appeared promising to bring relief to our patients…

However, another pharmaceutical company bought the drug from GSK and the new company was not interested in continuing the use of Votrient™/pazopanib in HHT-related clinical trials. The study was closed after the 7^th patient completed the trial and we lost access to this promising drug product.

We were at a crossroads. Ultimately, we made the bold and unprecedented decision to invest in buying and manufacturing the drug ourselves. Our community deserves better therapeutics… and you need it NOW! We did not want to sit on the sidelines and hope someone else would come to our rescue. Taking matters into our hands was simply the only way forward.

With the support of our mighty community, we raised nearly $1 million and began advocating for funding though the Department of Defense and FDA. In 2022, we received a $5.2 million grant from the DoD and $800K from the FDA to support the trial! The FDA also provided Cure HHT a Breakthrough Designation for this drug after talking with the patients in the trial and reviewing the data. Since then, we have been hard at work building a clinical research organization, funding study operations, developing data, writing protocols, working with statistics, device companies, labs, auditors, regulatory experts and more to finish bring this trial to life.

Why does this matter? Because there are still no FDA approved drugs to treat HHT. This makes drugs much harder to be covered by insurance, and limits how accessible it can be for our patients.

If the trial results are positive (which we hope they are), we will apply for a “New Drug Application” with the FDA… where the results will be evaluated and hopefully we will be granted approval.

To our community of patients and caregivers and supporters, know that we are so much more than a patient advocacy organization. We are a catalyst for driving advancements in treatments for this disease.

Your support now means more than ever, as our goals are loftier than ever. And what we can realistically achieve soon are the kinds of things we once thought were impossible.

We are relentless in our fight to improve the lives of those suffering from this disease. Have hope!

Read a comprehensive Q&A to learn more about Pazopanib and our journey to sponsor this trial.

To learn more about the clinical trial, please visit ClinicalTrials.gov.