Announcement from BioCryst Pharmaceuticals About BCX9250

The IFOPA is sharing this important message from BioCryst Pharmaceuticals with the FOP community about the future of their development program for BCX9250, an ALK-2 inhibitor (also known as a kinase inhibitor). The other kinase inhibitors in development mentioned below include:

  • Saracatinib in the STOPFOP phase 2 clinical trial
  • IPN60130 in the FALKON phase 2 clinical trial
  • INCB000928 in the Incyte phase 2 clinical trial

A fourth kinase inhibitor owned by Keros Therapeutics has completed a phase one trial in healthy humans and is now in a phase two trial to evaluate the effects on anemia in patients with very low, low or intermediate risk myelodysplastic syndromes.

The IFOPA is grateful to BioCryst Pharmaceuticals for their work to learn the science of FOP, understand the patient experience and support the development of treatments. We are grateful for their sponsor support of the FOP Registry, FOP Family Gathering and FOP Drug Development Forum. 

An Important Message for the FOP Community from BioCryst

November 1, 2022

Dear FOP Patient Community Members:

When we look at the therapies currently in development by others within the biopharmaceutical industry for the treatment of FOP, we recognize that several of these, including some targeting the same mechanism of action as our investigational ALK-2 inhibitor, BCX9250, are positioned to reach approval and be available to patients earlier than our program. This lessens the benefit that BCX9250 may uniquely bring to the community as a treatment option and may in fact slow down other clinical trials and approvals given the competition for enrollment.

As FOP is an ultra-rare disease, we know that having too many clinical trial options could mean that even the earlier therapies available to patients could be delayed with a crowded clinical trial landscape. Above all else, we recognize that the most immediate need is for FOP patients to have access to an effective treatment as quickly as possible.

Therefore, after careful consideration, we have made the difficult decision to discontinue development of BCX9250 for FOP. This decision was made independent of the safety of BCX9250.

We value the relationships and partnerships that have been built and the opportunity to be part of this incredible community of passionate and dedicated individuals across the globe focused on FOP patients. The needs of the FOP community are very important, and we are committed to continuing our commitments to the IFOPA FOP Registry and 2022 Drug Development Forum.

Our partnership does not end here. We hope our research to date will contribute to a better understanding of FOP and help in your quest to find an effective treatment in the near future.

For any questions or concerns, please do not hesitate to reach out to us at [email protected].

With our sincere hope and gratitude,
BioCryst Global Patient Advocacy Team

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