Noema Pharma has racked up a phase 2a win for its Tourette syndrome drug candidate, reporting hits on the primary and key secondary endpoints in a small study of the former Roche molecule.
Investigators enrolled 15 people to receive ascending daily oral doses of the PDE10A inhibitor gemlapodect, also known as NOE-105. After 12 weeks, 57% of the 14 patients who took at least one dose and had at least one post-baseline efficacy assessment showed tic improvement compared to the start of the trial. Noema assessed tic improvement using the Tourette Syndrome Clinical Global Impression of Change.
Patients only needed to reach the rating of “minimally improved” to be classed as a responder but the biotech saw bigger changes in some participants. Six of the eight people who received the target dose, which Noema defined as 10 mg to 15 mg, were much or very much improved on the tic scale.
Noema included other assessments of Tourette symptoms as secondary endpoints. Across the 14 people in the primary analysis, the biotech saw a statistically significant 7.8-point reduction on the YGTSS Total Tic Score. The reduction was higher, 12.8 points, in the subgroup of people who received the target dose.
The biotech said adverse events were consistent with the known profile of gemlapodect, a candidate that completed a 75-subject phase 2 trial in childhood onset fluency disorder (COFD), a medical term for stuttering, last year. Noema didn’t publish a press release about the conclusion of that trial but still lists the COFD program in its pipeline.
Work to develop gemlapodect in Tourette is already moving ahead. Noema began enrolling the first of a targeted 180 people in a phase 2 trial last month. The primary endpoint is the YGTSS-R tic score, one of the secondary assessments in the previous study.
Noema is part of a small band of biotechs with active, clinical-phase Tourette programs and its targeting of PDE10A sets it apart from most of the rest of the pack. Companies including AstraZeneca, Otsuka and Teva have run Tourette trials over the years but the list of players with active programs is fairly short.
Emalex Biosciences is enrolling patients in two phase 3 trials, while SciSparc is preparing to enter phase 2. EuMentis Therapeutics is aiming to take a PDE10A inhibitor into phase 2 in the first quarter of 2025 but it has failed to hit targets for the program in the past.