BioCryst Pharmaceuticals, Inc.

Cocrystal Pharma, Inc. (COCP) is a biotechnology firm dedicated to researching and advancing antiviral treatments for severe and/or persistent viral illnesses. The company leverages structure-based technologies to create new antiviral medications, primarily focusing on infections caused by the hepatitis C virus (HCV), influenza, coronaviruses, and noroviruses. Its current pipeline includes CC-31244, a non-nucleoside polymerase inhibitor for HCV, which has successfully completed Phase IIa clinical trials. Additionally, CC-42344, a PB2 inhibitor designed to combat influenza, is currently undergoing preclinical development. Cocrystal Pharma is also actively pursuing the development of non-nucleoside polymerase inhibitors to address norovirus infections. The company maintains strategic alliances, including a research and licensing collaboration with Merck Sharp & Dohme Corp. for the discovery and development of proprietary influenza A/B antiviral agents. It also holds a licensing agreement with the Kansas State University Research Foundation, aimed at advancing antiviral compounds for norovirus and coronavirus infections. Furthermore, Cocrystal Pharma engages in drug discovery collaborations with both HitGen and InterX Inc. The company's operations are headquartered in Bothell, Washington.

BioMarin Pharmaceutical Inc.BMRN · Biotechnology/PharmaceuticalsSan Rafael, United States

69% matchPublic

BioMarin Pharmaceutical Inc. specializes in creating and bringing to market treatments for individuals suffering from severe and often fatal rare disorders and other critical medical conditions. The company currently markets several key pharmaceutical products. Among these are Vimizim, an enzyme replacement therapy targeting mucopolysaccharidosis (MPS) IV type A, a specific lysosomal storage disorder, and Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for individuals with MPS VI. Another notable offering is Kuvan, a unique synthetic oral version of 6R-BH4, prescribed for phenylketonuria (PKU), an inherited metabolic condition. Further expanding its product portfolio, BioMarin provides Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme administered via subcutaneous injection to lower blood Phe levels. Also available are Brineura, a recombinant human tripeptidyl peptidase 1 used in treating ceroid lipofuscinosis type 2, a variant of Batten disease; Voxzogo, a daily injectable c-type natriuretic peptide analog for achondroplasia; and Aldurazyme, a refined protein mirroring the naturally occurring human enzyme alpha-L-iduronidase. Beyond its established products, BioMarin has a robust development pipeline. This includes valoctocogene roxaparvovec, an adeno-associated virus vector undergoing Phase III clinical trials for severe hemophilia A. Additionally, BMN 307, an AAV5-mediated gene therapy, is in Phase 1/2 trials, aiming to normalize blood phenylalanine levels in PKU patients, while BMN 255 is also in Phase 1/2 trials for primary hyperoxaluria. Its clientele spans specialty pharmacies, hospitals, international government health bodies, and a network of distributors and pharmaceutical wholesalers. These services reach patients and healthcare providers across the United States, Europe, Latin America, and other global markets. BioMarin maintains strategic licensing and collaborative alliances with partners such as Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. Established in 1996, the company's corporate headquarters are located in San Rafael, California.

KalVista PharmaceuticalsKALV · PharmaceuticalsFramingham, United States

69% matchPublic

KalVista Pharmaceuticals, Inc. is a clinical stage pharmaceutical company, which engages in the discovery, development, and commercialization of small molecule protease inhibitors for diseases with significant unmet need. Its product candidates are inhibitors of plasma kallikrein being developed for two indications: Sebetralstat for HAE and Factor XIIa. The company was founded by T. Andrew Crockett, Edward P. Feener, and Lloyd Paul Aiello on March 26, 2004 and is headquartered in Framingham, MA.

Cumberland Pharmaceuticals Inc. functions as a specialized pharmaceutical enterprise, primarily involved in the procurement, advancement, and commercialization of prescription medications. Its strategic focus areas include acute hospital care, gastroenterology, rheumatology, and oncology, with operations spanning both the United States and global markets. The company's existing product line includes: Acetadote: an injectable solution for treating acetaminophen poisoning. Caldolor: an injectable medication for managing pain and fever. Kristalose: a prescription oral laxative solution effective for chronic and acute constipation. Omeclamox-Pak: used in the treatment of Helicobacter pylori infection and duodenal ulcer disease. Vaprisol: an injectable therapy for euvolemic and hypervolemic hyponatremia. Vibativ: an injectable antibiotic prescribed for severe bacterial infections, including those acquired in hospitals or associated with ventilators, as well as complex skin and skin structure infections. In its development pipeline, Cumberland is advancing: RediTrex injection: designed to treat active rheumatoid, juvenile idiopathic, and severe psoriatic arthritis, along with disabling psoriasis. Ifetroban: a product candidate currently in Phase II clinical trials for aspirin-exacerbated respiratory disease, systemic sclerosis, and Duchenne muscular dystrophy. It has also completed Phase II clinical trials for hepatorenal syndrome and portal hypertension, with further clinical investigation ongoing for its potential use in progressive fibrosing interstitial lung diseases. An additional product candidate in Phase II clinical trials, aimed at reducing cholesterol levels for hospital use. Founded in 1999, the company's corporate headquarters are located in Nashville, Tennessee.

Omeros Corporation is a biopharmaceutical firm with commercialized products, actively engaged in the discovery, development, and market introduction of both small-molecule and protein-based therapies, including treatments for rare diseases. The company's therapeutic endeavors primarily focus on inflammatory conditions, illnesses mediated by the complement system, cancers linked to immune system dysregulation, and disorders characterized by addiction or compulsion. Its robust clinical pipeline features several promising candidates. Narsoplimab (also known as OMS721 or MASP-2), a leading asset, has successfully concluded pivotal trials for hematopoietic stem-cell transplant-associated thrombotic microangiopathy (HSCT-TMA). This drug is also currently undergoing Phase III clinical evaluation for immunoglobulin A nephropathy (IgAN) and atypical hemolytic uremic syndrome (aHUS), in addition to a Phase II trial investigating its potential for treating COVID-19. Further expanding its clinical portfolio, Omeros is developing PPAR? (OMS405) in Phase II for opioid and nicotine dependence. PDE7 (OMS527) is progressing through Phase I studies for addiction, compulsive disorders, and movement disorders. Additionally, MASP-3 (OMS906) is currently in Phase I trials for paroxysmal nocturnal hemoglobinuria (PNH) and other conditions involving the alternative complement pathway. Beyond its clinical efforts, the company maintains a dynamic preclinical program. This includes small-molecule inhibitors targeting MASP-2, which are being explored for applications in aHUS, IgAN, HSCT-TMA, and age-related macular degeneration. Also in development is a next-generation, longer-acting antibody that targets MASP-2, alongside MASP-3 small-molecule inhibitors designed to address PNH and other alternative pathway disorders. Omeros's preclinical research further encompasses GPR174 inhibitors, as well as Chimeric Antigen Receptor (CAR) T-Cell and Adoptive T-Cell Therapies for various oncological indications. The company is also investigating G protein-coupled receptor targets for a broad spectrum of diseases, including immunological, immuno-oncological, metabolic, central nervous system (CNS), cardiovascular, musculoskeletal, and other conditions. Established in 1994, Omeros Corporation is headquartered in Seattle, Washington.

Sarepta Therapeutics, Inc. operates as a biopharmaceutical firm with products already available on the market. Its primary objective is the research and advancement of genetic treatment modalities, encompassing RNA-targeted therapeutics and gene therapies, specifically for the management of rare diseases. The company's commercial offerings include EXONDYS 51, an injectable medication prescribed for Duchenne muscular dystrophy (DMD) patients whose genetic mutation in the dystrophin gene is responsive to exon 51 skipping. Similarly, VYONDYS 53 is provided for individuals with DMD who can benefit from exon 53 skipping. Sarepta is also actively developing several investigational therapies: AMONDYS 45, a product candidate that utilizes phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to bypass exon 45 of the dystrophin gene. SRP-5051, a peptide-conjugated PMO engineered to bind to exon 51 of dystrophin pre-mRNA. SRP-9001, a gene therapy initiative focused on micro-dystrophin for DMD. SRP-9003, a gene therapy program targeting limb-girdle muscular dystrophies. The company has established collaborative relationships with various partners, including F. Hoffman-La Roche Ltd, Nationwide Children's Hospital, Lysogene, Duke University, Genethon, and StrideBio. Sarepta was incorporated in 1980 and maintains its corporate headquarters in Cambridge, Massachusetts.