On July 20th, 2023, our industry partner, Palvella Therapeutics, shared topline results from the Phase 3 study of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of PC through a press release.
The main points of the press release including the following:
- QTORIN rapamycin did not show a treatment effect on the Patient Global Assessment of Activities Difficulty primary endpoint, a daily patient-reported outcome measure which assessed the difficulty of patients carrying out activities on their feet, when compared to placebo.
- QTORIN rapamycin was well-tolerated in the study and no participants withdrew due to drug-related adverse events. No drug-related serious adverse events were reported, and all other adverse events were deemed mild or moderate in nature with the most common treatment emergent adverse events reported in the category of infections and infestations (nasopharyngitis, COVID-19, and upper respiratory tract infections).
- The company plans to further analyze and share the Phase 3 VAPAUS study results with key stakeholders, including PC Project, physicians, and scientists. Based on these top-line results, Palvella does not plan to invest in additional clinical studies or commercial preparation activities for QTORIN rapamycin for the treatment of PC.
Please read the full press release here.
This news is undoubtedly disappointing to our community and to many of you personally. I know some patients in the Phase 2 or the Phase 3 trial truly felt like they experienced real improvement. Unfortunately, these types of trial occurrences happen as part of drug development; many trials are negative and don’t meet their primary endpoint.
Considering this news, I’d like to share some thoughts with you:
First and foremost, on behalf of the entire PC community, I extend my sincere thanks to every one of you who participated in either the Phase 2 or the Phase 3 trial. Your dedication and sacrifices are so appreciated. Between the two trials, we had 160 participants! Do you realize how amazing that is for a rare disease organization to enroll trials like we have? And that doesn’t even account for the hundreds more of you who wanted to participate but couldn’t for one reason or the other. The drug development world now knows that if a therapeutic is developed for PC, there are genetically tested registry patients who will be in trials, which is the ONLY way to get a drug approved.
I also express my immense gratitude to the members of the Palvella team. They gave our rare disease a chance and worked tirelessly to take us farther in the clinical trial process than we’ve ever been before. They collaborated closely with PC Project at every stage, eager to understand the patients’ physical and emotional experiences with PC, always sought feedback, and made sure their company was doing right by the patients. Not once have we doubted their commitment to PCers and their desire to give us a viable treatment to increase our mobility and decrease our pain. Even though this is not the outcome Palvella wanted, we still know they care for us deeply, and we at PC Project wish their team success as they move on to help other rare diseases.
Despite the negative results of this trial, our close collaboration with Palvella has provided us with valuable insights into both clinical trials and PC. As outlined in the press release, Palvella will continue to analyze the data from the trial and plans to share important information with our Medical and Scientific Advisory Board to help us learn to to benefit future clinical trials. None of our work has been in vain. We will use the knowledge gained to shape our future endeavors.
As an important reminder, we have PC patients in two other Phase 1 clinical trials, we have patients in an off-label study for an already approved drug, and we are in contact with two other companies who are exploring possible treatments for PC. The goal of PC Project has always been to pursue as many effective treatments as possible.
Meanwhile, PC Project continues to facilitate and fund research for yet more innovative research with the end goal of PC therapeutics. For example, the deadline for our current cycle of grant applications is August 31. We still have much to learn about our rare disease and we will continue to support quality PC research projects, not only for future treatments, but until we have a cure.
And because we have more potential treatments on the horizon, we still need you – our registry patients – to participate in future clinical trials if you are able. We will always notify you of possible trials that apply to your specific situation and location. One of the main reasons we have invested so much in our registry, including providing free genetic testing, is so we can give genetically confirmed patients opportunities to be in trials. Stay tuned!
Finally, and most importantly, please, please do not ever give up hope. We have come so far, accomplished much, and despite the challenges, it is an exciting time to be part of our global PC community. We have much to be proud of and much to look forward to. And it definitely helps that we are strengthened by exceptional people, including researchers, physicians, and other supporters who genuinely care about PC patients.
I send my love to each one of you. Our encouraging and beautiful PC community continually inspires me, and I hope it inspires you, too. Stay strong, stay positive, and keep smiling! And as my husband once wrote me, “KTV!” (Keep the Vision!)
All my love,
Janice Schwartz
Executive Director, PC Project
