Axial Therapeutics is advancing a pipeline of gut-targeted small-molecule drugs that act on defined mechanisms to mitigate the impact of metabolites and bacteria linked to neurological disease pathology via the microbiome gut-brain axis. Its lead candidate, AB-2004, is a first-in-class, gut-restricted molecular therapeutic that selectively sequesters certain bacterially derived metabolites and is being developed for irritability in children with autism; AB-2004 showed safety, metabolite reductions, and evidence of behavioral improvement in a Phase 1b/2a trial and is in Phase 2 development. The company’s discovery approach is rooted in the research of co-founder and chief scientist Dr. Sarkis Mazmanian at Caltech and aims to identify new gut targets for neurodevelopmental and neurodegenerative disorders. Axial is also pursuing preclinical gut-targeted programs including oncology and Parkinson’s disease. Financially, the company has raised $37.25 million in its recent Series C and $91.5 million in total funding to date, funds it intends to use to advance AB-2004 and its broader discovery platform. Axial Biotherapeutics is a clinical-stage biopharmaceutical company developing gut-targeted small molecules and live biotherapeutics that leverage the gut–brain axis to address CNS and other gut-derived diseases. The company’s pipeline includes lead programs targeting Parkinson’s disease and Autism Spectrum Disorder. Axial has built a discovery platform focused on the interaction between the brain and the gut and is expanding its application beyond CNS indications into oncology. Taiho Ventures invested $10 million as a direct investment to fund discovery and development of gut-targeted, small-molecule oncology approaches, and Sakae Asanuma will join Axial’s board as part of the investment. The $10M is an extension of Axial’s previously closed Series B in February 2019, bringing the total Series B financing to $35M. Axial will retain control and rights to its existing CNS programs while granting Taiho Ventures a first right to negotiate an exclusive license related to the new oncology programs. Axial Biotherapeutics is a clinical-stage biopharmaceutical company developing gut-targeted small-molecule programs that modulate the gut–brain axis for neurodegenerative and neuropsychiatric diseases. Its lead programs target Parkinson’s disease (PD) and autism spectrum disorder (ASD) and pursue CNS benefit via peripheral gut-microbiome mechanisms. The PD program identified a pathway that induces motor and GI symptoms and brain pathology in a validated preclinical PD model and includes AB-4166 (being evaluated for safety and tolerability in a PD subpopulation) and multiple novel chemical entities in the AB-4000 series. The ASD program focuses on reducing systemic and brain exposure to problematic microbial metabolites; lead candidate AB-2004 has shown preclinical ability to repair leaky gut and improve ASD-related behaviors and was expected to enter a Phase 1b/2a trial in adolescents in Q1 2019. The company completed a $25 million Series B equity financing to advance its PD and ASD programs and progress clinical development in 2019. Axial is based in Waltham, Massachusetts and Pasadena, California. Axial Biotherapeutics is building a microbiome discovery platform that leverages research linking the gut microbiome to the central nervous system to develop a new class of microbial-targeted biotherapeutics. The company has licensed exclusive, worldwide rights to intellectual property covering a novel class of CNS biotherapeutics from the Mazmanian Laboratory at Caltech. Axial aims to translate those discoveries into a diverse pipeline of therapies for neurological diseases and disorders. Lead indications named in the launch include autism spectrum disorders (ASD) and Parkinson’s Disease. Axial announced a $19.15 million Series A financing to support its program development and platform expansion. The company has appointed David H. Donabedian, Ph.D., as CEO and a board director as it advances toward clinical development.

Total raised

$91M

Funding rounds

4

Latest round

Series C

Latest activity

Oct 2021