Calico and AbbVie Therapy Fills FDA Rare Disease Pilot Slot

Calico Life Sciences has announced that its investigational treatment for an autosomal recessive brain disorder has been selected for the FDA's Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. This initiative aims to expedite the development of mid- to late-stage drugs and biologics for rare diseases by facilitating more frequent communication with the FDA. Fosigotifator (ABBV-CLS-7262), developed in partnership with AbbVie, is currently being studied in a Phase Ib/II clinical trial for patients suffering from vanishing white matter (VWM) disease.

VWM disease, an ultra-rare progressive leukoencephalopathy, is characterized by the chronic activation of the integrated stress response (ISR), leading to the degeneration of white matter in the brain. This results in impaired muscle movement, cognitive decline, seizures, and shortened lifespan. Currently, there are no approved treatments for VWM disease. Fosigotifator targets eIF2B, a guanine nucleotide exchange factor vital for protein synthesis and a key regulator of the ISR. The therapy is also under development for treating amyotrophic lateral sclerosis (ALS).

Arthur Levinson, CEO of Calico, emphasized the significance of fosigotifator’s inclusion in the START program, highlighting its potential to meet the unmet needs of those affected by VWM disease. The START pilot program has designated seven candidates, with fosigotifator being the last to be announced. The program divides its participating therapies between drugs and biologics, aiming to address rare neurodegenerative conditions and other rare diseases likely to cause significant disability or death within the first decade of life.

The other six candidates chosen for the START program include Moderna’s mRNA-3705, an mRNA therapy for methylmalonic acidaemia; Larimar Therapeutics’ nomlabofusp, a protein replacement therapy for Friedreich’s ataxia; Grace Science’s GS-100, an AAV9 gene therapy for NGLY1 deficiency; Myrtelle’s rAAV-Olig001-ASPA, a gene therapy for Canavan disease; Denali Therapeutics’ DNL126, an enzyme replacement therapy for MPS IIIA; and Neurogene’s NGN-401, a gene therapy for Rett syndrome. These selections were made by the FDA’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).

The goal of the START program is to accelerate the development of promising treatments for rare diseases, offering a lifeline to patients with limited or no therapeutic options. The increased communication with the FDA is expected to streamline the regulatory process, enabling faster delivery of these critical treatments to patients.

In summary, Calico Life Sciences' fosigotifator represents a significant step forward in the treatment of VWM disease, a condition with no current approved therapies. Its inclusion in the FDA’s START pilot program underscores its potential to address the severe unmet needs of patients suffering from this debilitating disease. Alongside other innovative therapies selected for the program, fosigotifator offers hope for advancing rare disease therapeutics and improving the quality of life for affected individuals and their families.