FDA, Newron Pharma to Meet in Step to Releasing Results of Sarizotan Pivotal Trial

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by Steve Bryson, PhD |

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STARS, sarizotan and FDA

Newron Pharmaceuticals will meet with the U.S. Food and Drug Administration (FDA), which requested a meeting to discuss the company’ statistical plan for releasing data from its pivotal STARS Phase 2/3 study of sarizotan in treating Rett syndrome patients with breathing problems.

The event, called a Type A meeting — which can include discussion of special protocols — followed Newron’s September filing with the agency of its statistical plan for unblinding the trial’s results.

Suggested by the FDA, it is expected to take place within 30 days of receipt of a formal request from Newron. The company said it would provide an update on trial results within a month of the meeting, but no data will be shared or unblinded until then.  

“We look forward to meeting with the Agency to discuss their suggestions for the STARS study statistical plan and are eager to share the final results of the study … once the plan has been agreed with the FDA,” Ravi Anand, MD, Newron’s chief medical officer, said in a press release.

The Sarizotan Treatment of Apneas in Rett Syndrome (STARS) study (NCT02790034) evaluated the safety and efficacy of sarizotan, given daily as 10mg or 20 mg capsules (based on age and weight), to placebo in 130 Rett syndrome patients, ages 6 and older, with impaired lung function. 

The trial’s primary goal was to reduce apnea — breaks or stops in breathing while awake — that occurs in about 70% of patients, and contributes to comorbidities (co-existing conditions) and poorer quality of life. The treatment tolerability was also evaluated.

Participants’ respiration was recorded at homes using a provided device at four points in time during the study’s 24 weeks. 

A long-term, open-label extension study followed, where all were treated with sarizotan. To date, more than 80% of the initial trial’s patients are taking part in the extension. 

Sarizotan is designed to bind to specific receptors of the neurotransmitters serotonin and dopamineIn mouse models of Rett syndrome, sarizotan normalized serotonin levels in the brain — which are typically lower in people with Rett syndrome — and markedly improved breathing.

The therapy was granted rare pediatric disease designation by the FDA to treat breathing difficulties in people with Rett syndrome. It was first developed by Merck KGaA to treat involuntary muscle movements associated with the use of levodopa, the standard treatment for Parkinson’s disease.

As it was first tested in another indication, sarizotan was licensed to Newron with an extensive safety and tolerability data package, the company said.

Newron launched the The Voices of Rett,” a global survey into the burden of Rett syndrome, in November. This survey is examining the physical, emotion, and financial impact of the disease for patients, as well as their families and caregivers. Its goal is to raise awareness, help identify treatments, and provide other resources for the condition.