Gemlapodect

– Topline results from Phase 2a study – Nearly 60% of all patients treated with gemlapodect and 88% of patients completing the study at the target clinical dose range were responders based on the primary efficacy assessment The Yale Global Tics Severity Scale Total Tic Score (YGTSS-TTS) showed a statistically significant improvement of -7.8 points for all patients, and -12.8 points for patients completing the study at the target clinical dose No weight gain, events of metabolic marker increase, or serious adverse events were reported BASEL, Switzerland, Oct. 17, 2024 (GLOBE NEWSWIRE) -- Noema Pharma AG, a clinical-stage neuroscience-based company, today announced the Phase 2a ALLEVIA-1 study with NOE-105 (gemlapodect) in patients with Tourette syndrome (TS) met its primary and all key secondary endpoints. Gemlapodect is a novel and first-in-class PDE10A inhibitor currently being studied to assess its potential to reduce vocal and motor tics in people with TS with an improved tolerability profile over current treatment options. The Phase 2a study, ALLEVIA-1 (NOE-TTS-211), was a multicenter, 12-week, open-label, single-arm monotherapy study which enrolled a total of 15 patients with TS with a mean age of 26.2 (+/- 8.7) years. Patients were treated with ascending doses of gemlapodect ranging from 2.5 to 15 mg once daily, with a target dose range of 10 to 15 mg per day. The ALLEVIA-1 study achieved its primary and secondary objectives. Key findings included: 57% of patients showed tic improvement compared to baseline, with the remaining patients having no change and no patients worsening on the independently adjudicated TS Clinical Global Impression of Change (TS-CGI-C; primary endpoint) (n=14 patients taking at least one dose of study medication and having at least one post-baseline efficacy assessment).For patients who completed the study within the target clinical dose range, 87.5% showed improvement and 75% were rated as much or very much improved on the TS-CGI-C (n=8).YGTSS Total Tic Score showed a mean improvement of -7.8 points (p=0.009) from baseline to endpoint for all patients taking at least one dose and having at least one post-baseline assessment.YGTSS Total Tic Score showed a mean improvement of -12.8 points (p=0.003) from baseline to endpoint for all patients completing the study within the target clinical dose range.Adverse events were consistent with the known profile of gemlapodect, and no serious adverse events were reported during the trial.No weight gain and no clinically significant changes in laboratories (including metabolic measures such as blood glucose or lipids) were reported. “Based on these promising data, we believe gemlapodect has the potential to offer a meaningful advancement in the treatment of Tourette syndrome,” said Ilise Lombardo M.D., CEO of Noema Pharma. “I would like to thank the investigators and patients for their important contributions to the ALLEVIA-1 study.” “Tourette syndrome is a devastating disease that impacts patients and their families. Recurrent and severe tics interfere with the patient’s ability to learn, work and interact with others.” said Prof. Kirsten Mueller-Vahl, Principal Investigator, MHH, Hannover, Germany. “Current treatments, essentially antipsychotics, are associated with side effects such as significant weight gain and insulin resistance, as well as stigma. Our findings with gemlapodect are very encouraging, given that the need for new treatments and novel mechanisms in Tourette syndrome remains notably high.” Initial study data were presented at the MDS 2024 Congress, September 28, 2024. About Noema Pharma Noema Pharma is a clinical-stage biotech company advancing a portfolio of transformative therapeutics utilizing first-in-disease approaches targeting neuroscience-based conditions with high unmet need. Noema has four programs currently in active Phase 2 clinical trials evaluating pain in Trigeminal Neuralgia, seizures in Tuberous Sclerosis Complex, Tourette syndrome and vasomotor symptoms plus associated symptoms of menopause with readouts expected in 2025. Noema was founded by leading venture capital firm Sofinnova Partners and is supported by current investors including Forbion, Gilde Healthcare, Invus, Jeito Capital, Polaris Partners and UPMC Enterprises. Learn more at www.noemapharma.com. About NOE-105/gemlapodect NOE-105/gemlapodect is a potential first-in-class phosphodiesterase-10A (PDE10A) inhibitor in mid-stage clinical development as a dopamine modulator for Tourette syndrome. NOE-105 has been well-tolerated in studies with healthy volunteers and in patients with Tourette syndrome with no evidence of metabolic side effects including weight gain, hyperlipidemia and type 2 diabetes. NOE-105 has the potential to achieve a more targeted effect by modulating dopamine signaling exclusively in the medium spiny neurons of the striatum in the brain, while limiting off-target effects compared to other dopamine antagonists. About Tourette Syndrome Tourette syndrome is a neurodevelopmental disorder in the wider spectrum of tic disorders, consisting of motor and vocal abrupt and brief movement or sound which is preceded by a local or generalized urge/premonitory sensation. TS impacts over a half-million individuals and families in the US alone, as well as millions of individuals worldwide. Investors Contact and Media Contact Precision AQAlex Lobo212-698-8802alex.lobo@precisionaq.com

Swiss biotech Noema Pharma jumpstarted back in 2020 with a $60 million Series A financing round. Today, it’s wrapping up its Series B round with another $112 million to develop its pipeline of molecules focused on central nervous system disorders. Noema started out with four clinical stage assets in-licensed from Roche. The company says it will use this new round of financing to push forward its lead candidate basimglurant (NOE-101), an mGluR5 inhibitor which is currently being evaluated in Phase IIb clinical trials for severe pain in trigeminal neuralgia and seizures in tuberous sclerosis complex. Noema is also developing gemlapodect (NOE-105), a PDE10a inhibitor, in Phase IIb trials for Tourette syndrome and stuttering. A Phase IIb trial readout of gemlapodect in stuttering is expected in the next 12 months. NOE-115, a triple reuptake inhibitor for atypical depression and binge eating disorder, is Phase-II ready. “All of these molecules have been studied before in other indications. Roche has invested a lot in that and we have a lot of data that shows activity and also shows those molecules are safe,” CEO Luigi Costa said. “We will start another Phase II/III study in Tourette syndrome because we have completed a Phase IIa in adult patients and then we were encouraged to proceed into phase two which will include adult and adolescent patients,” Costa added. Costa said the indications for the drug candidates were chosen because they are either poorly treated with current available medications and surgeries or because there are no therapies at all. Most patients with stuttering, also known as childhood onset fluency disorder (COFD), for example, go into speech therapy — which doesn’t work for everyone. For the people who did not see positive change with therapy, gemlapodect could be the answer. Antoine Papiernik, chairman and managing partner at Sofinnova Partners, one of the founding investors, said the “beauty here is in the pipeline.” “It’s a pipeline of having a bunch of molecules. It’s a pipeline even within each of these molecules that are being developed in several indications,” Papiernik told Endpoints News . “So you know, [NOE] 101, TSC and trigeminal neuralgia, you don’t have all your eggs in one basket, and you really have two shots on goal.” It was George Garibaldi, former head of neuroscience product development at Roche for a decade, who first chose the compounds to license after Roche decided to shelve part of its psychiatry pipeline. The company started out with six staffers and now has 18, Costa said. The Series B round was co-led by Forbion and Jeito Capital. UPMC Enterprises joined previous investors Sofinnova Partners, Polaris Partners, Gilde Healthcare and Invus. Following the close of the Series B, Nanna Lüneborg from Forbion and Rachel Mears from Jeito Capital will join the board of directors of Noema Pharma. Editor’s Note: This story has been updated to clarify that Noema is not a spinout from Roche but licensed drugs from Roche after its formation.