PDE10A

Phase 2b study will evaluate the safety and efficacy of gemlapodect (NOE-105) in patients with Tourette syndromeBASEL, Switzerland, Oct. 30, 2024 (GLOBE NEWSWIRE) -- Noema Pharma AG, a clinical-stage neuroscience-based company, today announced the first patients have been dosed in a global Phase 2b study evaluating the safety and efficacy of gemlapodect (NOE-105) in Tourette syndrome. NOE-TTS-201 (NCT06315751) is a Phase 2b, 12-week, randomized, double-blind, placebo-controlled study that is designed to examine the safety and efficacy of gemlapodect. The trial will enroll 140 patients with Tourette syndrome in multiple centers in the US and Europe. Patients will be treated with up to 15 mg of gemlapodect once daily, or a matching placebo. The primary endpoint is the Yale Global Tic Severity Scale (YGTSS) Total Tic Score. “Patients with Tourette syndrome carry an incredible burden related to the expression of motor and vocal tics with limited treatment options,” said Ilise Lombardo, M.D., Chief Executive Officer of Noema Pharma. “I am happy to announce the progression of the development of gemlapodect in Tourette syndrome with the dosing of the first patients in our Phase 2b trial.” 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. 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. 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 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. Investor Contact: Precision AQAlex Lobo212-698-8802alex.lobo@precisionaq.com

– 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

-Statistically significant benefit demonstrated in primary endpoint of positive subscale of the Positive and Negative Syndrome Scale for Schizophrenia (PANSS) at Week 4- -Dose-response was demonstrated, consistent with higher efficacy in the 40 mg dose- -Favorable tolerability profile demonstrated relative to standard-of-care atypical antipsychotics- -Investor call to be held 10th of July at 3 PM CEST with details provided below: MS Teams: https://teams.microsoft.com/l/meetup-join/19%3ameeting_NTA4ZDg0NzEtYjU2ZS00N2NjLWI4MjAtYzAwMzQxMDBhNDlj%40thread.v2/0?context=%7b%22Tid%22%3a%22dc816c94-0313-4f34-8ed1-1bff167d7003%22%2c%22Oid%22%3a%22640e4061-4b30-4b4f-ab73-ea6aee635b80%22%7dTelephone: +48 22 161 67 63, 522919119# Poland, Warsaw. 522 919 119# WARSAW, Poland, July 09, 2024 (GLOBE NEWSWIRE) -- Celon Pharma S.A. (CLN.WA) announces today robust and positive Phase 2 clinical trial results for its PDE10A inhibitor (CPL’36), a novel, oral, once-daily antipsychotic. Statistically significant and clinically meaningful improvements in the primary endpoint of positive subscale of the PANSS were seen in both tested doses of CPL’36, with a dose-response effect. Management plans to discuss these highly encouraging results with regulatory agencies to advance CPL’36 towards registrational trials and global marketing approvals. Additionally, CPL’36 is being investigated as a potential treatment for levodopa-induced dyskinesia in Parkinson’s disease, with Phase 2 results expected to be reported in 4Q 2024. “The results from this Phase 2 trial suggest a beneficial effect of CPL’36 across multiple aspects of schizophrenia pathophysiology. Clinical efficacy was deemed strong, and moreover, the drug tolerability was quite favorable,” confirmed Maciej Wieczorek, Ph.D., CEO of Celon Pharma S.A. “We believe that these results could herald a breakthrough in the treatment of this disease that impacts over 24 million patients world-wide, by identifying an innovative drug class with a new mechanism of action that may be used to replace or complement the current standard of care. These results represent a lengthy and satisfying journey for Celon as we have been closely analyzing the competitive environment in schizophrenia and the clinical trial designs of major international companies working on PDE10A inhibitors. We are therefore proud to have developed a drug characterized by unprecedented efficacy, with global potential to help underserved patients,” he concluded. Trial description The CPL’36 Phase 2 study was an international, multicenter, randomized, placebo-controlled clinical trial conducted on a group of 189 adult patients hospitalized due to acute schizophrenia. CPL’36 was administered for 4 weeks in two doses of 20 and 40 mg once daily and was placebo-controlled at a ratio of 1:1:1. Patient baseline severity was moderate-severe to severe, as characterized by a PANSS total score of approximately 106. Patients were screened for up to 10 days and then randomized and treated over a four-week period, at which time the primary endpoint assessment was conducted at Day 28. At Week 4 of treatment, the reduction in positive PANSS subscale score which was the primary endpoint in the trial was 3.7 units from baseline in the 20 mg dose (LS mean difference from placebo, p<0.001, Cohen’s d: 0.73), and 6.3 units in the 40 mg dose (LS Mean difference from placebo, p<0.001, Cohen’s d: 1.38). For total PANSS score at week 4 of treatment (a key secondary endpoint), the 20 mg dose of CPL’36 demonstrated a 9.7 unit reduction from baseline compared to placebo (LS mean difference from placebo, p<0.001, Cohen’s d: 0.77), and 16.4 units in the 40 mg dose (LS mean difference from placebo, p<0.001, Cohen’s d: 1.47). Other secondary endpoints in the trial included the effects of CPL’36 on overall clinical improvement cognitive performance and functioning such as Clinical Global Impression Scale Improvement (CGI-I), Brief Assessment of Cognition in Schizophrenia (BACS), and number of participants who withdraw due to adverse events (AEs). Results across all of these endpoints were also positive. Drug tolerability was favorable with most treatment emergent adverse events characterized as mild. Exacerbations of schizophrenia represented the most common severe adverse events that were potentially related to the drug (1.5% in the placebo group, 1.8% in the 20 mg group and 3.1% in the 40 mg group). Treatment discontinuation due to adverse events likely related to the drug occurred in 3.1% patients in the placebo group, 0% patients in the 20 mg group and 7.7% patients in the 40 mg group. Investor Call Information: Małgorzata Siewierskainwestorzy@celonpharma.com+48519066531 ABOUT CELON PHARMA Celon Pharma is an integrated biopharmaceutical company which designs, develops, manufactures and distributes pharmaceutical products. It was founded in 2002 by Maciej Wieczorek, PhD. The Company currently has approx. 500 employees. It employs approx. 160 scientists responsible for research and development, half of whom either have a PhD or are in the process of acquiring their PhD. The Company's pipeline includes more than a dozen projects for the development of innovative drugs with therapeutic groups such as oncology, neuro-psychiatry, autoimmunity and metabolism. The Company has its own research and development laboratories, which allow it to develop its own pharmaceutical technologies by using extensive laboratory equipment resources, as well as the experience and expertise of its staff. It also has a modern manufacturing facility where dry pharmaceutical forms are manufactured. Over the last few years, the Company has introduced products into the market in the following therapeutic areas: oncology, central nervous system diseases, cardiology, respiratory diseases. The Company has been developing the technology for manufacturing inhalation drugs and several projects of innovative drugs since 2007. 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