Ovid Therapeutics

Operating as a clinical-stage biopharmaceutical entity, Anavex Life Sciences Corp. specializes in developing therapeutic drug candidates for various central nervous system (CNS) conditions. Its leading compound, ANAVEX 2-73, is currently in advanced development, undergoing Phase III clinical evaluation for both Alzheimer's disease and pediatric Rett syndrome. This same drug is also in Phase II trials for Parkinson's disease and is being explored in preclinical studies for a wider range of disorders, including epilepsy, infantile spasms, Fragile X syndrome, Angelman syndrome, multiple sclerosis, and tuberous sclerosis complex. Another significant drug candidate, ANAVEX 3-71, has progressed to Phase I clinical trials for frontotemporal dementia and other forms of dementia, with additional preclinical investigation underway for neurodegenerative illnesses like Alzheimer's and Parkinson's. The company's earlier-stage portfolio includes ANAVEX 1-41, a sigma-1 receptor agonist targeting depression, stroke, Parkinson's, and Alzheimer's diseases; ANAVEX 1066, a mixed sigma-1/sigma-2 ligand with potential applications in neuropathic and visceral pain; and ANAVEX 1037, aimed at treating prostate and pancreatic cancers. Anavex Life Sciences Corp. was founded in 2004 and maintains its headquarters in New York, New York.

Stoke Therapeutics, Inc. is an emerging biopharmaceutical company dedicated to developing innovative antisense oligonucleotide (ASO) therapies. Its primary focus is to target and address the fundamental causes of severe genetic disorders, particularly within the United States. The company employs its exclusive Targeted Augmentation of Nuclear Gene Output (TANGO) platform to engineer ASOs that precisely enhance protein expression. Stoke's most advanced candidate, STK-001, is currently progressing through Phase I/IIa clinical trials for Dravet syndrome, a severe and debilitating genetic epilepsy. Additionally, STK-002 is in the preclinical development stage for treating autosomal dominant optic atrophy. The company has also established a licensing and collaborative agreement with Acadia Pharmaceuticals Inc. to identify, develop, and market new RNA-based medicines for critical and rare genetic neurodevelopmental conditions impacting the central nervous system. Formerly known as ASOthera Pharmaceuticals, Inc., the company rebranded to Stoke Therapeutics, Inc. in May 2016. Founded in 2014, Stoke Therapeutics, Inc. maintains its headquarters in Bedford, Massachusetts.

Praxis Precision Medicines, Inc. is a biopharmaceutical firm operating at the clinical stage, dedicated to creating innovative treatments for central nervous system (CNS) conditions that stem from an imbalance in neuronal activity. The company's primary investigational compounds currently undergoing evaluation include: PRAX-114, an extrasynaptic-preferring positive allosteric modulator of the GABAA receptor, which is currently in Phase IIa clinical trials for addressing both major depressive disorder and perimenopausal depression. Additionally, PRAX-944, a small molecule designed to selectively inhibit T-type calcium channels, is also progressing through Phase IIa clinical trials to manage essential tremor. Beyond these, Praxis is also advancing a pipeline of other promising drug candidates, such as: PRAX-562, a persistent sodium current blocker, undergoing Phase I clinical evaluation for treating severe pediatric epilepsy and adult cephalgia (headaches). PRAX-222, an antisense oligonucleotide (ASO) aimed at individuals suffering from gain-of-function (GOF) SCN2A epilepsy. And a program targeting KCNT1 for the treatment of KCNT1 GOF epilepsy. To further its research and development efforts, Praxis maintains several strategic alliances. These include a cooperation and licensing arrangement with RogCon Inc.; a licensing deal with Purdue Neuroscience Company; a comprehensive research collaboration, option, and license agreement with Ionis Pharmaceuticals, Inc.; and a collaborative project with The Florey Institute focused on the creation of three new antisense oligonucleotides (ASOs). Established in 2015, the company's headquarters are situated in Boston, Massachusetts.

Neurogene Inc. is dedicated to engineering transformative genetic treatments for individuals and their families grappling with debilitating neurological conditions. Its current pipeline prominently features two key investigational therapies: NGN-401, an AAV9 gene therapy specifically targeting Rett syndrome, and NGN-101, which is being developed to address neuronal ceroid lipofuscinosis subtype 5 (Batten disease). The company's operations are based in New York, New York.

Aptinyx Inc.BiotechnologyUnited States

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Aptinyx Inc. is an Evanston, Illinois-based clinical-stage biotechnology company focused on discovering and developing innovative therapies for challenging disorders of the brain and nervous system.

Amylyx Pharmaceuticals, Inc. operates as a clinical-stage biopharmaceutical firm, primarily focused on creating therapies for amyotrophic lateral sclerosis (ALS) and various other neurodegenerative conditions. A key asset in its developmental pipeline is AMX0035, a proprietary dual UPR-Bax apoptosis inhibitor. This compound, which integrates sodium phenylbutyrate and taurursodiol, is under investigation for treating ALS, and its application is also being explored for other neurodegenerative diseases. The company was established in 2013 and is based in Cambridge, Massachusetts.