Final slot for FDA’s rare disease pilot filled by therapy from Calico, AbbVie

07 Jun 2024
firstwordpharma.com
Gene TherapyOligonucleotideClinical StudysiRNA
Calico Life Sciences said its experimental therapy for an autosomal recessive brain disorder has been tapped for the FDA’s Support for clinical Trials  Advancing Rare disease Therapeutics (START) pilot programme. It's the seventh and final candidate to be announced for the new initiative, which is intended to speed up the development of mid- to late-stage drugs and biologics for rare diseases by enabling more frequent communication with the US regulator.
The Alphabet-founded biotech, along with partner AbbVie, is currently evaluating fosigotifator (ABBV-CLS-7262) in a Phase Ib/II trial of patients with vanishing white matter (VWM) disease. The therapy targets eIF2B, a guanine nucleotide exchange factor that is essential for protein synthesis and a key regulator of the integrated stress response (ISR). Fosigotifator is also being developed separately for amyotrophic lateral sclerosis.
In patients with VWM disease, an ultra-rare progressive leukoencephalopathy, chronic activation of the ISR causes white matter to degenerate and leads to impaired muscle movement, cognitive decline, seizures, and a shortened lifespan. There are no approved treatments for VWM disease.
"The inclusion of fosigotifator underscores the potential of this investigational therapy in addressing the unmet needs of individuals and families affected by vanishing white matter disease," said Calico CEO Arthur Levinson.
Participating therapies in the pilot programme are split between drugs and biologics, with FDA’s Center for Drug Evaluation and Research (CDER) having selected three products for rare neurodegenerative conditions, and the regulator’s Center for Biologics Evaluation and Research (CBER) having picked four cell or gene therapies intended to treat a rare disease that is likely to lead to significant disability or death within the first decade of life.
The other six candidates are 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 deficiencyNGLY1 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.
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