Panbela Therapeutics, Inc. announced that it has regained the North American rights to develop and commercialize Flynpovi (the combination of CPP-1X (eflornithine) and sulindac) in patients with familial adenomatous polyposis (FAP), as a result of the termination of the licensing agreement between Cancer Prevention Pharmaceuticals, Inc. (CPP) and with One-Two Therapeutics Assets Limited.
Panbela is now positioned to take the lead on designing the global trial protocol and presenting it to the Federal Drug Administration (FDA) and European Medicines Agency (EMA) for agreement on the registration pathway. By leveraging Panbela’s extensive experience with FAP and in designing global registration trials, the team can develop a high-quality trial protocol that meets the standards of regulatory agencies and is designed to efficiently and effectively demonstrate the potential safety and efficacy of Flynpovi in the treatment of FAP. This approach will help achieve a successful global regulatory approval and a successful launch of Flynpovi in the global market.
The new registration trial is expected to focus on FAP patients who have intact lower gastrointestinal anatomy and will build upon the positive results from the FAP-310 trial that were published in the New England Journal of Medicine and Disease of the Colon and Rectum (Burke et al. 2020; Balaguer et al. 2022). That study showed 100% risk reduction in the need for surgery in patients with an intact lower gastrointestinal anatomy with Flynpovi vs.CPP-1X or sulindac alone (Balaguer et al. 2022). The Company believes the FAP-310 trial data is compelling and the new registration trial could lead to the approval of Flynpovi. Since there are currently no approved drug therapies for the treatment of FAP, this therapeutic option has the potential to impact this urgent unmet need for patients with FAP globally.
In the short term, Panbela intends to unify the global registration process utilizing in-house expertise to seek agreement from the FDA and EMA on a proposed registration study protocol, which we believe with our previous Phase III results should provide convincing evidence for product approval.
Panbela is confident that the new FAP registration trial will have the potential to provide a non-surgical treatment option to both physicians and patients with FAP. Panbela is committed to working collaboratively with the FDA, EMA and the FAP community to advance this program and to ultimately provide a new treatment option for FAP patients.
“Our focus on disruptive therapeutics for urgent unmet medical needs is at the core of what we do at Panbela. We believe that Flynpovi has the potential to make a meaningful difference for patients with FAP and we are committed to bringing this innovative treatment to market,” said Dr. Jennifer Simpson President and CEO of Panbela Therapeutics. “We are excited to regain the worldwide rights to Flynpovi for FAP patients, and believe our internal expertise, experience with health authorities, relationship with FAP experts throughout the US and Europe and our commitment to FAP patients and their families, in combination with the positive results from the FAP-310 trial, provide a solid foundation for designing and executing a successful registration trial that has the potential to impact patients with FAP globally. We plan to advance this program, while maintaining our current cash burn, and will evaluate all opportunities to maximize the value of this asset.”