Marinus Pharmaceuticals, Inc. is a biopharmaceutical company, which engages in the development and commercialization of neuropsychiatric therapeutics. Its clinical stage drug product candidate, ganaxolone, is a positive allosteric modulator being developed in three different dose forms: intravenous, capsule, and liquid. The company was founded in August 2003 and is headquartered in Radnor, PA.
相关临床试验
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- Ray Therapeutics appointed Scott Braunstein, M.D., as Chairman of the Board, bringing over 30 years of biotechnology and pharmaceutical industry experience to guide the company's strategic growth. - The company also named Michael Murtagh as Chief Regulatory Officer, leveraging his two decades of global regulatory expertise to support development of optogenetic gene therapies. - Ray Therapeutics is currently evaluating RTx-015 in a Phase 1 clinical trial for retinitis pigmentosa and preparing to dose the first patient in a Phase 1 trial of RTx-021 for Stargardt disease. - The leadership appointments reflect the company's commitment to advancing differentiated optogenetic therapies for patients with vision loss, targeting diseases that affect over half a million people worldwide.
- Cassava Sciences has appointed Dr. Joseph Hulihan as Chief Medical Officer, bringing over 25 years of experience in neurological disorder therapeutics development. - Hulihan will focus on advancing simufilam, an investigational oral small molecule targeting filamin A protein for treating Tuberous Sclerosis Complex-related epilepsy. - The first clinical study for simufilam in TSC-related epilepsy is expected to begin in the first half of 2026, following positive preclinical results. - Hulihan previously served as CMO at Marinus Pharmaceuticals, where he developed ganaxolone for seizure disorders including TSC-related epilepsy.
- Ovid Therapeutics has entered into a definitive agreement with Immedica Pharma AB to sell its future royalty rights for ganaxolone sales outside China for $7 million in cash. - The transaction provides non-dilutive funding to support Ovid's ongoing operations, while Immedica strengthens its focus on ganaxolone by acquiring additional intellectual property rights. - Ganaxolone is an approved medicine in the EU, Great Britain, US, and China for treating epileptic seizures associated with CDKL5 deficiency disorder in patients aged 2-17 years. - Immedica will also assume financial responsibility for all costs related to the licensed intellectual property portfolio and plans to expand the license to include additional indications.
- Swedish rare disease specialist Immedica Pharma has agreed to acquire Marinus Pharmaceuticals for $151M, strengthening its position in the global rare disease market through a cash tender offer of $0.55 per share. - The acquisition includes ZTALMY (ganaxolone), an FDA-approved treatment for seizures associated with CDKL5 deficiency disorder in patients aged two and above, marking Immedica's expansion into the US market. - The transaction, unanimously approved by Marinus' board of directors, is expected to close in Q1 2025 and will accelerate Immedica's revenue growth through immediate access to a commercial-stage asset.
- Marinus Pharmaceuticals announced a workforce reduction of approximately 45% following disappointing Phase III trial results for oral ganaxolone in tuberous sclerosis complex. - The TrustTSC trial, evaluating ganaxolone, did not meet its primary endpoint, leading Marinus to discontinue further clinical development of the oral drug. - Marinus is exploring strategic alternatives and plans a meeting with the FDA to discuss a potential path forward for intravenous ganaxolone in refractory status epilepticus. - The company reported a net loss of $24.2 million for Q3 and expects to fund operations into Q2 2025 with its current cash reserves.
- Marinus Pharmaceuticals shifts focus to oral ganaxolone (O-GNX) for tuberous sclerosis complex (TSC) after IV ganaxolone (IV-GNX) fails a Phase 3 trial for refractory status epilepticus (RSE). - The company's drug ZTALMY, used for CDKL5 deficiency disorder (CDD), shows promising revenue with FY'24 guidance projecting approximately $35-37 million. - Upcoming top-line results from the Phase 3 TrustTSC study on oral ganaxolone for TSC-related seizures are expected by mid-fourth quarter of 2024. - Marinus is developing a second-generation ganaxolone formulation to improve safety and efficacy, with IND-enabling studies expected by the end of 2024.
• Amgen has launched Pavblu, its biosimilar to Regeneron's Eylea, at a 10% discount, pricing it at $1,665 per dose, potentially muting its market impact. • GSK is investing $800 million to expand its manufacturing site in Pennsylvania, focusing on sterile liquid vaccines and medicines production. • Roche and Dyno Therapeutics are collaborating to advance AAV gene therapy vectors for neurological diseases, with Dyno receiving $50 million upfront. • AbbVie and Gideon Richter are partnering to discover novel targets for neuropsychiatric conditions, with Richter receiving $25 million upfront.
- Marinus Pharmaceuticals' Phase III TrustTSC trial of oral ganaxolone for tuberous sclerosis complex (TSC)-associated seizures did not meet its primary endpoint. - The trial, involving 129 participants, showed a 19.7% median reduction in seizure frequency with ganaxolone compared to placebo, but the difference was not statistically significant. - Marinus is halting further clinical development of ganaxolone for TSC and implementing cost-reduction measures, including workforce downsizing. - The company will continue to support the commercial growth of ganaxolone oral suspension CV, which is approved for CDKL5 deficiency disorder.
- Marinus Pharmaceuticals' Phase 3 TrustTSC trial evaluating oral ganaxolone for tuberous sclerosis complex (TSC)-associated seizures did not meet its primary endpoint. - The trial aimed to demonstrate a statistically significant reduction in 28-day frequency of TSC-associated seizures with ganaxolone compared to placebo. - Ganaxolone treatment resulted in a 19.7% median reduction in seizure frequency, versus 10.2% with placebo, a difference that was not statistically significant. - Marinus plans to discontinue ganaxolone development, reduce costs through job cuts, and explore strategic alternatives, engaging Barclays as an advisor.
- Marinus Pharmaceuticals' Phase 3 TrustTSC trial of oral ganaxolone for tuberous sclerosis complex (TSC)-associated seizures did not meet its primary endpoint. - Ganaxolone showed a 19.7% median reduction in seizure frequency compared to 10.2% for placebo, but the results were not statistically significant (p=0.09). - Marinus will discontinue further ganaxolone development, reduce costs, and explore strategic alternatives, while continuing to support ZTALMY for CDKL5 deficiency disorder. - The company's stock faces pressure as it explores options, including a potential sale, following the trial's failure and uncertain corporate future.
