PRISM Platform™ Designed Medicines

The PRISM Platform™ is a first in the industry rational drug design technology that leverages atom-by-atom compound design

Explore First Atom's current development candidates and our ongoing discovery programs

PRISM Platform™ Designed Therapeutic Pipeline

Compound Stage Target Disease Indications Discovery Pre-Clinical IND-Enabling Clinical

ATX-624 IND-Enabling SARM1 ALS, CIPN, CMT2A, ADOA

ATX-091 IND-Enabling DRP1 PD, Ischemia/Stroke, CM

Multiple Pre-clinical Multiple Immunology, Oncology, CM

Novel Medicines with Novel Mechanisms of Action

ATX-624: Novel SARM1 Intramolecular Glue

ATX-624 is a PRISM Platform™ designed development candidate. It is a novel intramolecular glue that inhibits SARM1 allosterically by selectively "gluing" two domains of the protein together, forcing SARM1 into an inactive confirmation

Unlike other approaches which target the NADase site in SARM1, ATX-624 is specific to SARM1 and does not engage other similar NADase proteins, imparting enhanced efficacy and safety

Lead Disease Indications & Target Biology

Amyotrophic Lateral Sclerosis

About the Disease

ALS, also known as Lou Gehrig's disease, is a fatal neurodegenerative disorder characterized by the progressive loss of motor function. Nerve cells that control muscle function are lost in ALS patients.

Targeting SARM1 with ATX-624

SARM1 is a key regulator of nerve cell degeneration in ALS. We have developed a novel intramolecular glue inhibitor of SARM1, ATX-624, that is specifically designed to reinforce intermolecular contacts between the TIR and ARM domains of SARM1 to block access to the NADase active site, restoring the native auto-inhibited structure and restoring motor function in preclinical models of ALS.

Rare & Orphan Neuropathies

About the Disease

Rare and orphan neuropathies including diseases like hereditary optic atrophies and Charcot Marie Tooth, currently have limited therapeutic options, let alone curative interventions.

Multiple Targeting Approaches with ATX-624 and ATX-091

The majority of rare neuropathies are driven by SARM1 activation and/or mitochondrial dysfunction. We are actively investigating the use of both ATX-624 and ATX-091 in multiple neuropathic conditions

Parkinson's Disease

About the Disease

PD is a chronic and progressive neurodegenerative disorder that results from the loss of dopaminergic neurons leading to both motor and non-motor symptoms.

Targeting DRP1 with ATX-091

PD pathology is highly linked to mitochondrial dysfunction. A variety of pathways lead to mitochondrial dysfunction in PD, including DRP1-mediated mitochondrial fragmentation. Our DRP1 inhibitor, ATX-091 shows significant promise as a disease-modifying therapeutic for PD.

Chemotherapy-Induced Neuropathy

About the Disease

CIPN affects over 80% of cancer patients taking platinum-based and taxane chemotherapeutics, often resulting in dose-limiting neuropathy. Overcoming this neuropathy provides a path to increased chemo dosing & better patient response.

Targeting SARM1 with ATX-624

Like in ALS, SARM1 is activated in CIPN, resulting in nerve degeneration and neuropathic pain. Preclinical studies using our SARM1 inhibitor, ATX-624 show a near complete amelioration in pain symptoms during chemo dosing.