Ann: Plans for Phase 3 trial in Rett syndrome, page-60

Howie

Newron has been mentioned on this forum quite a few times over the past few years. Here is one post from two years ago which explains a bit more about the company.

The connection is that both companies are developing drugs for Rett Syndrome and, to some extent, both compete for the same trial participants and end market. The main difference is that Newron’s drug, sarizotan, is intended to treat just one possible symptom of Rett syndrome - apneas (breathing disturbances), whereas trofinetide is intended to address a much wider range of symptoms (refer to RSBQ). It’s estimated that roughly half of the Rett population have breathing disturbances that sarizotan addresses.

I found it worthwhile noting that Newron’s international 129 patient, 6 month trial, in patients 4 years and older, including older adults and males, will have taken over 3 years from commencement to release of top line results. This is more than a year longer than originally expected, mainly due to slower than expected recruitment.

Although this serves as a cautionary reminder to everyone of how clinical trials can drag out, I can think of 10 positives for ACADIA/Neuren with respect to enrolment.

• This Phase 3 trial won’t be limited to ~50% of the Rett population.
• A 3 month trial is more attractive to trial participants than a 6 month trial (trial participation is highly disruptive and can be expensive).
• A trial addressing multiple symptoms of the disorder is likely to be more attractive for participants than a trial addressing just one symptom.
• ACADIA is reported to be working with Rettland.org to assist parents with reimbursement for reasonable travel costs associated with the trial.
• Neuren's last Phase 2 trial of 82 patients was well supported (despite overlapping with the sarizotan trial), with top line results reported in just under 12 months after trial commencement.
• ACADIA is seeking to expand the number of trial centres beyond the 12 that were used in the Phase 2 trial.
• With the sarizotan trial now completed and two other Phase 2 trials (Anavex and ketamine) due to complete later this year, there hopefully will be less competition for trial participants.
• There are supportive parents within the Rett community, such as Mel Lancaster, who are actively encouraging other Rett parents to participate.
• There is active collaboration and strong support from leading physicians and the largest Rett syndrome advocacy group (rettsyndrome.org)
• Trofinetide has already established a good safety and tolerability profile over multiple trials.