Newron Pharmaceuticals has received the final minutes from a meeting with the U.S. Food and Drug Administration (FDA) regarding the pivotal STARS Phase 2/3 clinical trial investigating sarizotan as a potential treatment for people with Rett syndrome and breathing difficulties.
Topline results from this trial are expected sometime from April to June.
In the Feb. 5 meeting, Newron and the FDA discussed the company’s statistical analysis plan (SAP), as well as the agency’s recommendations related to the trial’s outcomes and how efficacy data will be analyzed.
“We are pleased to report that Newron and the FDA have agreed on the SAP, the primary and key secondary efficacy measures, and the statistical analyses to be performed for efficacy,” Ravi Anand, MD, chief medical officer of Newron, said in a press release. “We are now finalizing the SAP and will submit it to the FDA in the coming weeks.”
Data from STARS (Sarizotan Treatment of Apneas in Rett Syndrome) are currently locked and blinded, which means that scientists are unaware of who took sarizotan and who took a placebo.
“Following the agency’s review and approval of the plan, the contract research organization involved in the study will be able to complete its programming work and then move ahead towards unblinding of the results,” Anand said.
The study (NCT02790034) tested the effectiveness and safety of sarizotan in more than 130 Rett syndrome patients, ages 4 and older, with impaired lung function.
Participants were given daily either 10 mg or 20 mg oral capsules (based on age and weight), or a placebo. Their respiration was recorded at home by a device at four intervals during the 24 weeks of the study.
In addition to assessing sarizotan’s tolerability, the goal of the study was to reduce stops in breathing while awake (apnea), which occurs in the majority of Rett syndrome patients and lowers their quality of life.
The first phase of the study was followed by a long-term, open-label extension study in which all participants could receive sarizotan. To date, more than 80% of the initial participants have joined the extension study.
“We expect to be able to share the topline results of this important study with the global Rett community and the markets in Q2 2020,” Anand said.
Designed to bind to specific receptors of the neurotransmitters dopamine and serotonin, sarizotan dramatically improved breathing in a number of mouse models of Rett syndrome and normalized serotonin levels in the brain, which are lower in people with Rett syndrome.
The therapy was developed first by Merck KGaA to treat involuntary muscle movements caused by the use of levodopa, the standard treatment for Parkinson’s disease. In November 2019, the FDA granted rare pediatric disease designation to sarizotan to treat people with Rett syndrome who have breathing difficulties. (Note: Merck KGaA is known as EMD Serono outside the U.S. and Canada.)
According to Newron, the license to develop the therapy included an extensive data package detailing safety and tolerability.
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