Tuberous Sclerosis

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

Fundraising campaign will fuel research to change the course of tuberous sclerosis complex SILVER SPRING, Md., Oct. 26, 2024 /PRNewswire/ -- Last night during the organization's 50th Anniversary Gala in New York City, the TSC Alliance® announced the public phase of a major fundraising campaign called the 50 Forward Fund. The effort aims to raise $40 million for research into tuberous sclerosis complex (TSC), a rare disease affecting 50,000 in the United States and 1 million worldwide. To date, the campaign has raised more than $18.5 million. "The 50 Forward Fund bolsters the momentum we've built since our founding while ambitiously looking forward to a future where everyone affected by TSC can live their fullest lives," said Kari Luther Rosbeck, TSC Alliance President & CEO. "The TSC Alliance is committed to further catalyzing research to create meaningful outcomes for people living with TSC and their families, and major investments in TSC research through this campaign will help us achieve this vision." Since 1984, the organization has cumulatively funded more than $37 million in TSC research and leveraged $505 million in additional Federal funding. Research accomplishments to date include: Backing the first-ever preventative clinical trial for epilepsy in the United States. Creating a Biosample Repository with more than 2,650 samples linked to clinical data in the Natural History Database. Building a Preclinical Consortium to help move new treatment ideas toward clinical trials. Funding basic research that ultimately contributed to FDA approval of three drugs specifically for TSC. The 50 Forward Fund is the TSC Alliance's largest research initiative in its 50-year history. It was designed as part of the organization's Research Business Plan to guide research efforts for TSC individuals with the following goals: Focusing research to better understand TSC and identify new treatments; Altering the course of the disease and improving quality of life through prenatal diagnoses and preventative treatments; and Educating medical professionals to achieve more rapid diagnosis and better treatment aligned with evidence-based standards of care. "Funding from the 50 Forward campaign will help the TSC Alliance expand our research investment in four key areas," explained Steven L. Roberds, PhD, Chief Scientific Officer. "These include our Natural History Database and Biosample Repository, Clinical Research efforts, Preclinical Consortium and Innovative Research grants program." Natural History Database and Biosample Repository: TSC affects everyone differently. To understand which drugs will be most effective for each individual, researchers need biosamples, clinical data and patient-reported outcomes. These require efficient collection processes, standardized analysis protocols and robust data management. Clinical Research: Clinical studies require extensive planning and collaboration. The TSC Alliance aims to alleviate the obstacles that stand in the way of efficient, effective and safe clinical trials. Preclinical Consortium: This project fills a critical research gap for standardized, rigorous testing of potential new TSC therapies prior to clinical trials. The innovative platform provides industry and academic members access to reproducible preclinical models and outcome measures, providing an unbiased platform for this critical stage in drug development. The Preclinical Consortium removes risks associated with developing new drugs and will encourage more companies to focus on treatments for the TSC community. Innovative Research: This effort's goal is to award more research grants annually, forge stronger connections between the top researchers around the world and bring stakeholders together at Innovation Workshops. "To achieve these ambitious research goals, we're counting on continued investment, collaboration and unwavering commitment of our community," Rosbeck continued. "The 50 Forward Fund will lead to research outcomes that will truly make a meaningful, positive impact on the lives of all those touched by TSC." To learn more about the 50 Forward Fund, view the campaign video and donate online, visit tscalliance.org/50-forward-fund today. About tuberous sclerosis complex Tuberous sclerosis complex (TSC) is a rare genetic disease that TSC causes tumors to grow in different organs and can impair their function, primarily the brain, heart, kidneys, skin and lungs. The disease is the leading genetic cause of epilepsy, including infantile spasms, and can also cause cognitive, behavioral and psychiatric disabilities; aggressive behaviors; and more. A strong correlation also exists between TSC and autism—an estimated 40-50% of individuals with TSC have autism spectrum disorder. TSC is a linchpin disease, meaning its genetic pathway also plays a role in other diseases and disorders. Advancements in TSC research may lead to a better understanding of autism, epilepsy, traumatic brain injury, diabetes and cancer—diseases that affect more than 65 million people in the United States alone. About the TSC Alliance The TSC Alliance improves quality of life for everyone affected by tuberous sclerosis complex by catalyzing new treatments, driving research toward a cure and expanding access to lifelong support. For more information, visit tscalliance.org. Contact Jaye Isham Chief Outreach Officer TSC Alliance (301) 562-9890 [email protected] SOURCE TSC Alliance WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Marinus Pharmaceuticals is reducing its workforce again and taking other measures to reduce costs after its epilepsy treatment failed a late-stage trial. The TrustTSC trial investigating oral ganaxolone for the treatment of seizures associated with tuberous sclerosis complex (TSC) missed the primary endpoint of percent change in TSC-associated frequency of seizures at 28 days. While there was a reduction in seizures in the treatment arm, the results weren’t statistically significant. The median reduction in seizure frequency was 19.7% for patients on ganaxolone compared with 10.2% for placebo, earning a p-value of 0.09. The company’s stock $MRNS tumbled on Thursday morning, dropping about 80%. “We are disappointed that the results of the TrustTSC trial are not likely to be sufficient for an sNDA filing,” Marinus CEO Scott Braunstein said in a statement . Marinus now plans to discontinue all clinical development of ganaxolone, and it started a “strategic alternatives” process that includes layoffs. The company didn’t specify how many employees it would let go. As of Dec. 31, Marinus had 165 full-time employees, though it cut about 20% of its workforce in May when it also implemented other cost-reducing measures. Since 2022, ganaxolone has been approved as Ztalmy for the treatment of seizures associated with CDKL5 deficiency disorder.