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.

Tonix Pharmaceuticals Holding Corp., a biopharmaceutical company, focuses on developing, discovering, commercializing, and licensing therapeutics to treat and prevent human disease and alleviate suffering. It markets Zembrace SymTouch and Tosymra for the treatment of acute migraine with or without aura in adults. Its portfolio focuses on central nervous system disorders, as well as rare disease, immunology, and infectious disease product candidates. The company's priority is to submit a New Drug Application (NDA) to the FDA for TNX-102 SL (cyclobenzaprine HCl sublingual tablet), which has completed two positive Phase 3 studies for the management of fibromyalgia. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Its TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted breakthrough therapy designation by the FDA. The company's rare disease development portfolio comprises TNX-2900, an intranasal potentiated oxytocin for the treatment of Prader-Willi syndrome (PWS). Its immunology portfolio includes TNX-1500, which is a biologic to address organ transplant rejection and autoimmune diseases. The company's infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases, including TNX-1800 and TNX-1850, in development as a vaccine to protect against COVID-19. Its infectious disease development portfolio also comprises TNX-3900, and TNX-4200 are orally available CD45 antagonists in preclinical development. Tonix Pharmaceuticals Holding Corp. has collaboration agreement with Bilthoven Biologicals to advance TNX-801 mpox vaccine candidate; and with X-Chem, Inc. to develop broad-spectrum antivirals. The company was founded in 2007 and is headquartered in Chatham, New Jersey.

Poseida Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing therapeutics for patients with high unmet medical needs. The company's development candidates for Heme Malignancies includes P-BCMA-ALLO1, which is in Phase I trial to treat patients with relapsed/refractory multiple myeloma; P-CD19CD20-ALLO1, which is in Phase I trial for treating B cell malignancies and other autoimmune diseases; P-BCMACD19-ALLO1, an allogeneic, off-the-shelf CAR-T product candidate in preclinical development for treating multiple myeloma; and P-CD70-ALLO1 under preclinical development to treat hematological indications. It is also involved in the development of P-MUC1C-ALLO1 that is in Phase I trial for treating a range of solid tumors, including breast, colorectal, lung, ovarian, pancreatic, and renal cancers; P-PSMA-ALLO1, an autologous chimeric antigen receptor T cell (CAR-T) product candidate that is in preclinical development for the treatment of patients with metastatic castrate resistant prostate cancer (mCRPC); and P-PSMA-101, an allogeneic CAR-T product candidate under Phase 1 clinical trial for treating mCRPC. In addition, the company engages in the development of P-FVIII-101, a clinical stage liver-directed gene therapy for the in vivo treatment of hemophilia A; P-OTC-101, a clinical stage liver-directed gene therapy for the in vivo treatment of ornithine transcarbamylase deficiency; and P-PAH-101, a clinical stage liver-directed gene therapy for the in vivo treatment of phenylketonuria. It has a research collaboration and license agreement with F. Hoffmann-La Roche Ltd, and Hoffmann-La Roche Inc. The company was incorporated in 2014 and is headquartered in San Diego, California.

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.

Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company, engages in the development of therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance. It is developing ulixacaltamide, a small molecule inhibitor of T-type calcium channels that is in Phase III clinical trial for the treatment of essential tremor; PRAX-562 for the treatment of pediatric patients with developmental and epileptic encephalopathies (DEE); and PRAX-628 to treat focal epilepsy. The company also develops PRAX-222 for the treatment of pediatric patients with early-onset SCN2A-DEE; PRAX-020 to treat KCNT1 related epilepsies; PRAX-080 for the treatment of PCDH19; and PRAX-090 and PRAX-100 for SYNGAP1 and SCN2A-LoF. It has a license agreement with RogCon Inc.; a research collaboration and license agreement with Ionis Pharmaceuticals, Inc.; a strategic collaboration and license agreement with UCB Biopharma SRL; and collaboration with The Florey Institute to develop three novel ASOs. The company was incorporated in 2015 and is based in Boston, Massachusetts.

Pulmatrix, Inc., a clinical stage biotechnology company, focused on development of novel inhaled therapeutic products to prevent and treat respiratory and other diseases with unmet medical needs in the United States. The company offers iSPERSE, an engineered dry powder delivery platform, which enables delivery of small or large molecule drugs to the lungs by inhalation for local or systemic applications. It engages in developing PUR1800, a narrow spectrum kinase inhibitor completed Phase 1b clinical trials for the treatment of acute exacerbations in chronic obstructive pulmonary disease; PUR1900 for the treatment of allergic bronchopulmonary aspergillosis in patients with asthma and cystic fibrosis; and PUR3100, an iSPERSE formulation of dihydroergotamine which is in Phase 1 for the treatment of acute migraine. It has a license agreement with RespiVert Ltd. for access to a portfolio of kinase inhibitor drug candidates. The company was founded in 2003 and is headquartered in Bedford, Massachusetts.

Vaccinex, Inc., a clinical-stage biotechnology company, engages in the discovery and development of targeted biotherapeutics to treat cancer, neurodegenerative diseases, and autoimmune disorders. The company's lead drug candidate, pepinemab, a humanized monoclonal antibody that binds and blocks the activity of SEMA4D. It is developing Pepinemab, which is in phase 2 study for treatment of Alzheimer's disease and has completed phase 2 study for treatment of Huntington's disease; Pepinemab in combination with Pembrolizumab in phase 2 study for head and neck cancer, Avelumab in phase 2 study for pancreatic cancer, and completed phase 2 study with Avelumab for non-small cell lung cancer; and Pepinemab in combination with Nivolumab completed phase 2 study for melanoma, and trastuzumab and DC vaccine in phase 2 study for breast cancer. The company has also developed ActivMAb, an antibody drug discovery platform based on a novel method for complex targets, such as multi-pass membrane receptors or large and diverse libraries of full-length human monoclonal antibodies on the surface of pox viruses. It has collaborations with Merck Sharp & Dohme; Ares Trading S.A.; The Children's Hospital of Philadelphia; Emory University; Huntington Study Group; H. Lee Moffitt Cancer Center and Research Institute, Inc; Catalent Pharma Solutions, LLC; Surface Oncology, Inc.; and Pharmaceutical and Biotech Co. The company was incorporated in 2001 and is headquartered in Rochester, New York.