FDA’s biologics chief Peter Marks famously coined the first race to the Covid-19 vaccine as Operation Warp Speed. Now the second iteration of OWS is targeting rare diseases, with the FDA officially selecting four participants to communicate more closely with: Denali Therapeutics, Larimar Therapeutics, Grace Science and Neurogene.
The pilot, officially known as the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program, will bring the early-stage participants and the relevant FDA staff closer together on everything from clinical study design to fine-tuning the patient population for the sponsors’ trials. The program, first announced last September, initially sought to include six participants — three from the FDA’s Center for Drugs and three from its Center for Biologics.
The pilot is ultimately meant to help speed novel therapies to patients with rare diseases faster than normal, and Marks has been vocal about advocating for the use of accelerated approvals for gene therapies for rare diseases.
Grace Science, co-founded by Nobel winner Carolyn Bertozzi, said Monday that its GS-100, an AAV9-based gene therapy for NGLY1 Deficiency, a life-threatening rare disease with no approved therapy, was accepted into the pilot. Grace is running a Phase 1/2/3 dose-finding study to investigate the long-term safety, tolerability, and efficacy of the gene therapy in those aged two to 18 with NGLY1 Deficiency.
Grace and its fellow participants explained that the pilot will allow for an extra initial meeting with the FDA and more ad hoc communications in the future, which would be similar to what Pfizer and Moderna received when developing their Covid vaccines for the first OWS.
Denali also disclosed on Monday that its investigational enzyme replacement therapy for the rare MPS IIIA (Sanfilippo syndrome type A) was selected as part of the voluntary pilot. The San Francisco-based company is running a Phase 1/2 study of DNL126 for children with MPS IIIA, which currently has no approved treatment options.
Larimar, meanwhile, announced last Friday that its selection to START was for nomlabofusp, a protein replacement therapy designed to address the root cause of the rare Friedreich’s ataxia by delivering frataxin to mitochondria. Nomlabofusp is currently in an ongoing open-label extension study to assess the long-term safety, pharmacokinetics, and frataxin levels in peripheral tissues in patients with Friedreich’s ataxia. The company said it expects interim data from the study in the fourth quarter of this year.
And Neurogene said it was selected for the pilot for its NGN-401 gene therapy for Rett syndrome, which is a rare genetic mutation affecting brain development in girls.
Two dose levels of NGN-401 are being evaluated in a Phase 1/2 clinical trial. Neurogene recently presented safety data from the first three patients dosed with NGN-401, and the Company remains on track to report interim efficacy data in the fourth quarter of 2024.