Compare Stocks

Inozyme Pharma, Inc., a clinical-stage rare disease biopharmaceutical company, focuses on developing novel therapeutics for the treatment of vasculature, soft tissue, and skeleton diseases. The company's lead product candidate is INZ-701, a soluble, recombinant, genetically engineered, and fusion protein for the treatment of ENPP1 and ABCC6 deficiencies, as well as patients with end-stage kidney disease receiving hemodialysis. It has a license agreement with Yale University for specified therapeutic and prophylactic products. The company was incorporated in 2015 and is headquartered in Boston, Massachusetts.

Ovid Therapeutics Inc., a biopharmaceutical company, engages in the development of impactful medicines for patients and families with epilepsies and seizure-related neurological disorders in the United States. The company is developing soticlestat, a novel cholesterol 24 hydroxylase inhibitor, which is in Phase 3 clinical trials for the potential treatment of patients with resistant epilepsies; OV329, a GABA aminotransferase inhibitor which is in Phase 1 clinical trials for the treatment of seizures associated with tuberous sclerosis complex and infantile spasms; and OV350, a small molecule direct activator of the KCC2 transporter, which is in Phase 1 clinical trials for treating epilepsies. It also develops OV815, that focuses on the mutations associated with KIF1A-associated neurological disorder (KAND); OV888 (GV101), a highly selective rock2 inhibitor which is in Phase 1 double-blind multiple-ascending dose trial; OV825, has advanced to potential candidate lead identification for the rare neurodevelopmental condition HNRNPH2 (Bain Syndrome); and OV882, a short hairpin RNA gene therapy for the treatment of Angelman syndrome. The company has license and collaboration agreements with Healx, AstraZeneca AB, H. Lundbeck A/S, Northwestern University, and Graviton, as well as Marinus Pharmaceuticals, Inc. The company was incorporated in 2014 and is headquartered in New York, New York.

Silence Therapeutics plc, a biotechnology company, focuses on the discovery and development novel molecules incorporating short interfering ribonucleic acid (siRNA) to inhibit the expression of specific target genes in hematology, cardiovascular, and rare diseases. The company's mRNAi GalNAc Oligonucleotide Discovery platform consists of precision engineered product candidates designed to target specific disease-associated genes in the liver. The company develops Zerlasiran (SLN360), which is in phase 2 clinical trial for cardiovascular disease associated with elevated lipoprotein(a); and Divesiran (SLN124), an siRNA molecule that is in Phase I clinical trials for the treatment of genetic hematological conditions, including polycythemia vera. It is also developing SLN-COMP-1 and SLN-COMP-2 for complement-mediated diseases; and SLN-HAN-1 and SLN-HAN-2. It has collaboration agreements with AstraZeneca PLC to discover, develop, and commercialize siRNA therapeutics for the treatment of cardiovascular, renal, metabolic, and respiratory diseases; Hansoh Pharmaceutical Group Company Limited to develop siRNAs; and Mallinckrodt Pharma IP Trading DAC to develop and commercialize RNAi drug targets designed to silence the complement cascade in complement-mediated disorders. The company was formerly known as SR Pharma plc and changed its name to Silence Therapeutics plc in May 2007. Silence Therapeutics plc was incorporated in 1994 and is headquartered in London, the United Kingdom.

Zevra Therapeutics, Inc., a rare disease company melding science, discovers and develops various proprietary prodrugs to treat serious medical conditions in the United States. The company utilizes its Ligand Activated Therapy technology to generate improved prodrug versions of FDA-approved drugs, as well as to generate prodrug versions of existing compounds that may have applications for new disease indications. Its prodrug product candidate pipeline is focused on the high need areas of attention deficit hyperactivity disorder, stimulant use disorder, and CNS rare diseases, including idiopathic hypersomnia (IH). The company's lead product candidate KP1077, which is under Phase II clinical trial for the treatment of IH and narcolepsy, is based on its prodrug of d-methylphenidate, known as serdexmethylphenidate. It is also developing KP879, a prodrug product candidate for the treatment of stimulant use disorder and is under Phase II clinical trial. In addition, the company has received FDA approval for AZSTARYS, a once-daily treatment for attention deficit hyperactivity disorder in patents age six years and older, and for APADAZ, an immediate-release combination product containing benzhydrocodone, a prodrug of hydrocodone, and acetaminophen. The company's product pipeline include, arimoclomol It has collaboration and license agreement with KVK-Tech, Inc. and Commave Therapeutics SA. The company was formerly known as KemPharm, Inc. and changed its name to Zevra Therapeutics, Inc. in February 2023. Zevra Therapeutics, Inc. was incorporated in 2006 and is headquartered in Celebration, Florida.