Clinical Trials
88
29 active
Approvals
2
Total approvals
Agencies
1
Regulatory bodies
Founded
1986
Active, not recruiting
29
33.0%
Approved For Marketing
1
1.1%
Completed
41
46.6%
Recruiting
3
3.4%
Terminated
11
12.5%
Unknown
1
1.1%
Withdrawn
2
2.3%
- 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.
