BioCryst Pharmaceuticals
BioCryst Pharmaceuticals, Inc. is a biotechnology company. It engages in the provision of structure-guided drug design with the goal of developing oral small-molecule and protein therapeutics to target difficult-to-treat rare diseases. The company was founded by William M. Spencer III, Charles E. Bugg, and John A. Montgomery in 1986 and is headquartered in Durham, NC.
Clinical Trials
178
101 active
Approvals
5
Total approvals
Agencies
2
Regulatory bodies
Founded
1986
Active, not recruiting
100
56.2%
Approved For Marketing
1
0.6%
Completed
42
23.6%
Not yet recruiting
1
0.6%
Recruiting
19
10.7%
Terminated
11
6.2%
Unknown
1
0.6%
Withdrawn
3
1.7%
- Once-daily deucrictibant extended-release tablets reduced hereditary angioedema attack rates by 83% versus placebo in the Phase 3 CHAPTER-3 trial, with a p-value below 0.0001. - The global, double-blind study randomized 85 adolescents and adults across 21 countries to 40 mg deucrictibant XR or placebo for 24 weeks, with all secondary endpoints met. - Pharvaris plans to submit a prophylaxis New Drug Application to the FDA in the first half of 2027, following an accepted NDA for the immediate-release formulation. - Deucrictibant blocks the bradykinin B2 receptor, potentially making it the first oral HAE therapy spanning both on-demand treatment and long-term prophylaxis.
- Island Pharmaceuticals (ASX: ILA) has received FDA Orphan Drug Designation for Galidesivir as post-exposure prophylaxis against Marburg virus disease, granting seven years of US market exclusivity upon approval. - Marburg virus is classified as a Category A bioterrorism threat with no currently approved vaccines or antiviral therapies, and carries an average fatality rate of 50%, reaching up to 88% without proper care. - Galidesivir has demonstrated 100% survival in animal studies when administered 24–48 hours post-infection, compared to 0% survival in placebo groups, and has already completed human safety trials. - The company is targeting regulatory submissions in Q1-2027, with dose optimisation studies expected to begin next quarter and a pivotal animal rule study to follow later this year.
- BioCryst Pharmaceuticals has appointed Dr. Sandeep M. Menon as Chief Research and Development Officer, bringing extensive experience from Alnylam Pharmaceuticals and Pfizer. - Dr. Menon previously led the FDA approval of AMVUTTRA (vutrisiran) for ATTR cardiomyopathy at Alnylam and co-led the rapid development of PAXLOVID at Pfizer. - The appointment comes as BioCryst advances navenibart toward a potential BLA submission and expands its rare disease pipeline following the Astria Therapeutics acquisition. - BioCryst aims to leverage Dr. Menon's proven track record in complex program approvals to strengthen its R&D strategy and create long-term value through repeated rare disease launches.
- ADARx Pharmaceuticals has appointed Donald Fong, M.D., as Chief Medical Officer to lead clinical development strategy and execution for its next-generation RNA therapeutics pipeline. - Dr. Fong brings over 25 years of clinical development expertise across multiple therapeutic areas including rare disease, ophthalmology, immunology, infectious disease, oncology and metabolic conditions. - The appointment strengthens ADARx's leadership as the company advances ongoing Phase 3 and Phase 2 clinical trials for its RNA-targeted therapeutic candidates. - Dr. Fong previously served as Chief Medical Officer at BioCryst Pharmaceuticals and led clinical development programs including plasma kallikrein inhibitors for hereditary angioedema treatment.
- BioCryst Pharmaceuticals completed its $700 million acquisition of Astria Therapeutics on January 23, 2026, adding the late-stage plasma kallikrein inhibitor navenibart to its hereditary angioedema portfolio. - Navenibart, currently in Phase 3 development, has the potential to become the first HAE therapy with every-three and every-six month dosing intervals, offering significant treatment convenience improvements. - The acquisition positions BioCryst to offer both the leading oral therapy ORLADEYO and a potentially best-in-class injectable prophylactic option for individualized HAE patient care. - BioCryst financed the transaction through cash on hand and approximately $396.6 million from a Blackstone-managed financing facility, while issuing 37.3 million shares to Astria stockholders.
- BioCryst Pharmaceuticals announced a $700 million cash-and-stock acquisition of Astria Therapeutics, representing a 54% premium over Astria's closing price. - The deal centers on Astria's lead candidate navenibart, a long-acting injectable monoclonal antibody in Phase 3 trials for hereditary angioedema prophylaxis. - BioCryst aims to offer both oral and injectable treatment options for HAE patients, combining navenibart with its existing oral therapy Orladeyo. - The transaction is expected to close in Q1 2026, with Astria shareholders receiving $8.55 cash plus 0.59 BioCryst shares per share.
- The FDA approved three novel hereditary angioedema (HAE) therapies in 2025 after four years without new approvals, increasing total marketed drugs by 38% to 11 products. - CSL Behring's garadacimab became the first FDA-approved drug targeting coagulation factor XII, offering a new biological mechanism for HAE prevention. - KalVista's sebetralstat (Ekterly) represents the first oral on-demand treatment for HAE, providing patients with rapid relief during acute episodes. - Ionis Pharma's donidalorsen sodium introduced the first antisense oligonucleotide for HAE with extended dosing intervals of every four to eight weeks.
- DelveInsight's 2025 pipeline report reveals over 20 companies are actively developing more than 30 therapeutic candidates for hereditary angioedema treatment across various clinical stages. - Recent clinical developments include CSL Behring's Phase 3b study of garadacimab (CSL312) announced in August 2025 and KalVista's pediatric trial of KVD900 for patients aged 2-11 years. - Leading pipeline therapies span multiple approaches including oral plasma kallikrein inhibitors, gene therapies, and monoclonal antibodies, with products in late-stage development showing promise for addressing unmet medical needs.
- DelveInsight's 2025 pipeline analysis reveals over 20 companies developing 25+ therapies for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder characterized by red blood cell destruction. - Recent regulatory approvals include EMA and FDA approval of PIASKY (crovalimab) from Hoffmann-La Roche for adults and adolescents with PNH, marking significant progress in treatment options. - Multiple Phase III trials are underway, including Novartis's positive APPULSE-PNH results for iptacopan and Regeneron's combination therapy study with pozelimab and cemdisiran. - Emerging therapies span various mechanisms of action including complement inhibitors, monoclonal antibodies, and siRNA therapeutics, with oral and parenteral administration routes being explored.
- Major pharmaceutical companies including AstraZeneca, Novartis, and Astellas are defending patents for blockbuster drugs like Lynparza, Entresto, and Xtandi against generic manufacturers in early 2025. - The wave of Abbreviated New Drug Application (ANDA) cases spans critical therapeutic areas including oncology, cardiovascular disease, and neurological disorders. - Patent litigation activity has intensified with over 100 new cases filed across multiple federal district courts, indicating significant generic competition pressure on brand-name drugs.
