The FDA has lifted the clinical hold on Selecta Biosciences’ gene therapy to treat methylmalonic acidemia, a rare disease in children that affects metabolism. The company is now back in the saddle as it preps for a phase 1 trial.
The agency placed the hold more than three months ago due to outstanding questions regarding the chemistry, manufacturing and controls of the company’s drug candidate, MMA-01. It’s the latest hurdle the company has overcome with respect to MMA-01, after the company disclosed in April 2021 that a manufacturing issue could potentially delay its application for human trials. That issue was resolved four months later, and the timeline for a third quarter application held.
With the clinical hold lifted, Selecta CEO Carsten Brunn, Ph.D., says the company will launch into phase 1 trials “expeditiously”.
The drug was originally a collaboration between Selecta and Asklepios BioPharmaceutical, but the latter bowed out last year to reprioritize its own pipeline, granting full rights back to Selecta. However, Selecta still has three other gene therapy collaborations underway with Sarepta Therapeutics, Genovis and Takeda.
As for MMA-01, the hope is that it can alleviate MMA, a pediatric genetic disorder that affects one in 90,000 babies in the U.S. The inherited condition hinders the body’s ability to break down certain fats and proteins, which can cause toxins to build up and ultimately, metabolic crises. The disease’s long-term effects can include stunted growth, intellectual disabilities and kidney disease.
MMA-01 received FDA’s rare pediatric disease designation in October 2020 and orphan drug status a month later. It pairs an adenovirus vector with Selecta’s antigen-specific immune tolerance platform and is the company’s top asset in gene therapy, following the development of enzyme-based therapies, one of which is in a phase 3 trial for chronic refractory gout.