Axsome Therapeutics, Inc., a biopharmaceutical company, engages in the development of novel therapies for central nervous system (CNS) disorders in the United States. The company's commercial product portfolio includes Auvelity (dextromethorphan-bupropion), a N-methyl-D-aspartate receptor antagonist with multimodal activity indicated for the treatment of major depressive disorder; and Sunosi (solriamfetol), a medication indicated to the treatment of excessive daytime sleepiness in patients with narcolepsy or obstructive sleep apnea. It is also developing AXS-05, which is in Phase III clinical trial to treat Alzheimer's disease agitation, as well as that has completed phase II clinical trial for the treatment of smoking cessation; AXS-07, an investigational medicine that has completed Phase III trials for the acute treatment of migraine; AXS-12, an investigational medicine, which is in Phase III trial to treat narcolepsy; AXS-14, a selective and potent norepinephrine reuptake inhibitor that has completed Phase III trial for the treatment of fibromyalgia and other conditions; and solriamfetol, a dual-acting dopamine and norepinephrine reuptake inhibitor, which has completed a Phase 2 trial for treating attention-deficit/hyperactivity disorder, and is in phase 2 major depressive, binge eating, and shift work disorder. Axsome Therapeutics, Inc. has a research collaboration agreement with Duke University for evaluating AXS-05 in smoking cessation. The company was incorporated in 2012 and is based in New York, New York.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and ferroptosis and inflammation platforms, including vatiquinone to treat Friedreich ataxia and utreloxastat for the treatment of amyotrophic lateral sclerosis. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd, Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.

Avidity Biosciences, Inc., a biopharmaceutical company, engages in the delivery of RNA therapeutics. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat diseases previously untreatable with RNA therapeutics. The company's lead product candidate AOC 1001 for the treatment of myotonic dystrophy type 1, a rare monogenic muscle disease that is in phase 1/2 clinical trial. Its other products in pipeline include AOC 1044 for the treatment of duchenne muscular dystrophy, which is under phase 1/2 clinical development trial; and AOC 1020 to treat facioscapulohumeral muscular dystrophy that is in phase 1/2 clinical trial. The company is developing products for rare skeletal muscle and rare cardiac diseases. Avidity Biosciences, Inc. was incorporated in 2012 and is headquartered in San Diego, California.

Catalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.

Biohaven Ltd., together with its subsidiaries, focuses on discovering, developing, and commercializing therapies for immunology, neuroscience, and oncology worldwide. The company's pipeline products include Troriluzole, which is in Phase 3 clinical trial for the treatment of neurological and neuropsychiatric illnesses; BHV-5500 that blocks glutamate signaling mediated by post-synaptic NMDA receptors; Taldefgrobep Alfa, which is in Phase 3 clinical trial for the treatment of spinal muscular atrophy and obesity; BHV-7000, a candidate in Phase 2/3 clinical trials for the treatment of focal and generalized epilepsy, bipolar disorder, and major depressive disorder; BHV-2100 that is in Phase 1 clinical trials for the treatment of migraines and neuropathic pain; and BHV-8000, a product candidate in Phase 1 clinical trials for the treatment of early Alzheimer's and Parkinson's disease, sclerosis, and amyloid-related imaging abnormalities. It also offers BHV-1300, a product candidate in Phase 1 clinical trials to treat rheumatoid arthritis; BHV-1310 for the treatment of generalized myasthenia gravis and acute exacerbations or flares; BHV-1400 to treat IgA Nephropathy; and BHV-1600 for the treatment of dilated cardiomyopathy. In addition, the company develops BHV-1100, a product candidate in Phase 1a/1b clinical trials for multiple myeloma patients; BHV-1510, a preclinical product that targets carcinomas; and BHV-1500 for Hodgkin's lymphoma. It has license, development, and commercialization agreements with Yale University, AstraZeneca, University of Connecticut, Artizan Biosciences Inc., Reliant Glycosciences LLC, Katholieke Universiteit Leuven, BMS, and Highlightll Pharmaceutical Co. Ltd. The company was formerly known as Biohaven Research Ltd and changed its name to Biohaven Ltd. in September 2022. Biohaven Ltd. was incorporated in 2022 and is headquartered in New Haven, Connecticut. Biohaven Ltd. was formerly a subsidiary of Biohaven Pharmaceutical Holding Company Ltd.

Anavex Life Sciences Corp., a clinical stage biopharmaceutical company, engages in the development of therapeutics for the treatment of central nervous system diseases. Its lead product candidate is ANAVEX 2-73 for the treatment of Alzheimer's disease and Parkinson's disease, as well as other central nervous system diseases, including rare diseases, such as Rett syndrome, a rare severe neurological monogenic disorder; and infantile spasms, Fragile X syndrome, and Angelman syndrome. The company's drug candidate also comprises ANAVEX 3-71, which is in clinical trial for the treatment of schizophrenia, frontotemporal dementia, and Alzheimer's disease. Its preclinical drug candidates include ANAVEX 1-41 for the treatment of depression, stroke, and neurogenerative disease; ANAVEX 1066 for the potential treatment of neuropathic and visceral pain; and ANAVEX 1037 to treat prostate and pancreatic cancer. The company was incorporated in 2004 and is headquartered in New York, New York.

Aquestive Therapeutics, Inc. operates as a pharmaceutical company in the United States and internationally. The company markets Sympazan, an oral soluble film formulation of clobazam for the treatment of lennox-gastaut syndrome; Suboxone, a sublingual film formulation of buprenorphine and naloxone for the treatment of opioid dependence; Zuplenz, an oral soluble film formulation of ondansetron for the treatment of nausea and vomiting associated with chemotherapy and post-operative recovery; and Azstarys, a once-daily product for the treatment of attention deficit hyperactivity disorder. Its proprietary product candidates comprise Libervant, a buccal soluble film formulation of diazepam for the treatment of seizures; KYNMOBI, a sublingual film formulation of apomorphine for the treatment of episodic off-periods in Parkinson's disease; and Exservan, an oral soluble film formulation of riluzole for the treatment of amyotrophic lateral sclerosis. The company's proprietary pipeline of complex molecule product includes AQST-108, a sublingual film formulation delivering systemic epinephrine for the treatment of conditions other than anaphylaxis; and Anaphylm, an epinephrine sublingual film for the emergency treatment of allergic reactions, including anaphylaxis. In addition, it develops Adrenaverse, an epinephrine prodrug platform. The company was incorporated in 2004 and is headquartered in Warren, New Jersey.

Aerovate Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops drugs that enhance the lives of patients with rare cardiopulmonary diseases in the United States. It focuses on advancing AV-101, a dry powder inhaled formulation of imatinib for the treatment of pulmonary arterial hypertension, which is in Phase 2b/Phase 3 trial. The company was incorporated in 2018 and is headquartered in Waltham, Massachusetts.