RESEARCH TRIANGLE PARK, NC, April 27, 2022 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the European Medicines Agency (EMA) has granted access to the program Priority Drug Release (PRIME) for BCX9250, a novel oral activin receptor-like kinase-2 (ALK-2) inhibitor discovered and developed by BioCryst for the treatment of fibrodysplasia ossificans progressiva (FOP).
PRIME is a program launched by the EMA to strengthen support for the development of drugs targeting an unmet medical need. This voluntary program is based on enhanced interaction and early dialogue with developers of promising drugs and is designed to optimize development plans and accelerate evaluation so that these drugs can potentially reach patients sooner. According to the EMA, developers of drugs eligible for PRIME can expect additional opportunities for scientific advice and be eligible for accelerated assessment at the time of marketing authorization application.
“Promising results from non-clinical data and the first Phase 1 human safety, tolerability and pharmacokinetics study in healthy subjects formed the basis of the application for PRIME eligibility. We are pleased with the EMA’s decision to grant PRIME eligibility to BCX9250 – the first investigational drug for this indication to receive this designation – based on early evidence of BCX9250’s potential to address unmet patient need. living with FOP. We look forward to applying the benefits we have available from PRIME as we continue to advance our ALK-2 inhibitor program,” said Dr. Helen Thackray, Director of Research and Development at BioCryst.
FOP is an extremely rare and severely disabling genetic disease characterized by the irregular formation of bone outside the normal skeleton, also known as heterotopic ossification (HO). 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, European Union, Japan, United Kingdom and United Arab Emirates. 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 yellow fever. RAPIVAB® (peramivir injection) has received regulatory approval in the United States, Canada, Australia, Japan, Taiwan and Korea. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the company’s website at www.biocryst.com.
Forward-looking statements
This press release contains forward-looking statements, including statements regarding the potential benefits and opportunities associated with BCX9250 and access to PRIME. These statements involve known and unknown risks, uncertainties and other factors that may cause actual benefits and opportunities 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: access to PRIME may not expedite the development, regulatory review or approval process with the EMA and does not increase the likelihood that BCX9250 Receives 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, EMA, 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 clinical trials intended, may impose certain restrictions, caveats or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, 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, but not limited to, delays, stoppages, difficulties and increased expenses regarding development , regulatory processes and supply chains of BioCryst and its partners, adversely impact BioCryst’s ability to access capital or credit markets to fund its operations, or have the effect of increasing many risks described above or in documents BioCryst files from time to time 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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