RESEARCH TRIANGLE PARK, NC, June 08, 2022 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the United States Food and Drug Administration (FDA) has granted Fast Track to BCX9250 for prevention of heterotopic ossification (HO) in patients with fibrodysplasia ossificans progressiva (FOP).
According to the FDA, the purpose of Fast Track designation is to get important new drugs to patients sooner by facilitating the development and accelerating the review of drugs to treat serious conditions and address an unmet medical need. Companies that receive this designation are eligible for benefits including more frequent meetings with the FDA and written communications from the FDA, eligibility for expedited approval and priority review, if relevant criteria are met. , and an ongoing New Drug Application (NDA) review.
“We are pleased with the FDA’s decision to grant Fast Track designation to BCX9250, as there is a significant unmet need among patients living with FOP. With this designation following the recent decision by the EMA to grant PRIME eligibility to BCX9250 in Europe, we believe that the non-clinical data and the first phase 1 study on the safety, tolerability and pharmacokinetics in in healthy subjects supports the potential of our ALK-2 inhibitor program as a significant therapeutic advance for the FOP community,” said Dr. Helen Thackray, Head of Research and Development at BioCryst.
FOP is an extremely rare and severely disabling genetic disease characterized by HO, or the irregular formation of bone outside of the normal skeleton. HO can occur in muscles, tendons, ligaments, and other connective tissues. Patients with FOP become bound by this irregular ossification over time, with restricted movement and fused joints, resulting in deformities, restricted mobility, and premature mortality.
BCX9250 is designed to inhibit the ALK-2 enzyme, which is part of the normal signaling pathway for bone formation and responds to the binding of its specific ligands (bone morphogenic proteins, BMPs) by stimulating normal bone growth and turnover in healthy children and adults. Specific activating mutations of the ALK-2 gene are observed in all cases of FOP. An activating mutation in ALK-2 is required for disease to occur, making the ALK-2 enzyme an ideal drug target for the treatment of FOP.
In a Phase 1 clinical trial in healthy subjects, BCX9250 was safe and well tolerated at all doses studied, with linear and dose-proportional exposure supporting the potential for once-daily administration.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers new oral small molecule drugs that treat rare diseases where there is significant unmet medical need and where an enzyme plays a key role in the biological pathway of disease. Oral, once daily ORLADEYO® (berotralstat) is approved in the United States and several global markets. BioCryst has several ongoing development programs, including BCX9930, an oral factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir , a potential treatment for Marburg virus disease and jaundice. fever. RAPIVAB® (peramivir injection) is approved in the United States and several global markets, with ongoing post-marketing commitments. For more information, please visit the company’s website at www.biocryst.com.
This press release contains forward-looking statements, including statements regarding BioCryst’s plans and expectations regarding BCX9250 and the potential benefits associated with an FDA Fast Track designation. These statements involve known and unknown risks, uncertainties and other factors that may cause actual results or benefits to differ materially from those expressed or implied by the forward-looking statements. These statements reflect our current views regarding future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: Fast Track designation by the FDA may not lead to a faster development, regulatory review or approval process with the FDA and does not increase not likely that BCX9250 will receive marketing approval; the ongoing and future preclinical and clinical development of BCX9250 may not yield positive results; BioCryst may not be able to enroll the required number of subjects in planned clinical trials of product candidates; BioCryst may not advance human clinical trials with product candidates as expected; the FDA or other applicable regulatory agency may require additional studies beyond the planned studies for products and product candidates, may not provide regulatory clearances, which may result in delay of planned clinical trials, may require certain restrictions, warnings or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates or may suspend, delay or withdraw marketing authorization for products and product candidates; product candidates, if approved, may not be accepted by the marketplace; and the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst’s business, including, without limitation, delays, stoppages, difficulties and increased expenses with respect to concerns the development, regulatory processes and supply chains of BioCryst and its partners, adversely impact BioCryst’s ability to access capital or credit markets to finance its operations, or have the effect of increase many of the risks described above or in documents that BioCryst periodically files with the Securities and Exchange Commission. Please refer to the documents that BioCryst periodically files with the Securities and Exchange Commission, in particular BioCryst’s most recent annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s forward-looking statements.
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