Ionis Pharmaceuticals(IONS) Newsfilter·2025-01-13 02:00Portfolio Updates and Anticipated Milestones - Praxis has three blockbuster programs in late-stage development, with potential for four product launches between 2026 and 2028 [1] - The Essential3 program for ulixacaltamide in essential tremor (ET) includes two Phase 3 studies, with over 100,000 patients showing interest in participating since recruitment began in November 2023 [2] - Relutrigine is being evaluated in the EMBOLD study for developmental and epileptic encephalopathies (DEEs), with plans for the EMERALD study in a broader patient population [3] - Praxis shared an update at the 2024 AES Annual Meeting for an emergency use patient who achieved extended benefit after receiving relutrigine [4] - Ulixacaltamide is a highly selective small molecule inhibitor of T-type calcium channels, currently in late-stage development for essential tremor [5] - Praxis has completed discussions with global regulatory agencies to harmonize a registrational study design for elsunersen, with trial design and timelines expected in Q1 2025 [6] - UCB exercised its option to in-license global development and commercialization rights for a KCNT1 small molecule candidate, with Praxis eligible for up to $100 million in milestone payments and tiered royalties [6] - Praxis is on track to nominate development candidates for its early-stage ASO therapeutic initiatives in 2025, including PRAX-080, PRAX-090, and PRAX-100 [6] Clinical Trial Progress and Results - Vormatrigine demonstrated strong competitive differentiation in Phase 1 studies, with full results to be shared at an upcoming medical conference [10] - The EMPOWER observational study has enrolled over 2,000 patients, with early results shared at the 2024 AES Annual Meeting [10] - The RADIANT Phase 2 study for focal onset seizures (FOS) and generalized epilepsy is enrolling patients, with topline results expected in H1 2025 [10] - The POWER1 Phase 2/3 registrational study for treatment-resistant FOS is progressing, with topline results anticipated in H2 2025 [10] - Relutrigine showed a 46% placebo-adjusted reduction in monthly motor seizures in the EMBOLD study, with 77% reduction in motor seizures for patients continuing into the open-label extension [17] - Over 30% of patients achieved seizure freedom status while on relutrigine, with a median of 46 days of seizure freedom compared to 3 days at baseline [17] Regulatory and Commercial Developments - Praxis anticipates filing the NDA for ulixacaltamide in 2025, following positive results from the Essential3 program [9] - The company plans to re-initiate a study of ulixacaltamide in Parkinson's disease, addressing unmet needs for non-dopaminergic treatment options [9] - Praxis received a third rare pediatric drug designation (RPDD) for relutrigine in Dravet Syndrome, in addition to previous designations for SCN2A and SCN8A DEEs [17] - The company expects to initiate the pivotal trial for elsunersen in H1 2025, following regulatory feedback [8] Financial and Strategic Outlook - Praxis has a cash and investment position of approximately $470 million at the end of 2024, supporting a runway into 2028 [15] - The company is well-positioned for a readout-rich 2025, with results anticipated for ulixacaltamide, vormatrigine, and relutrigine, as well as regulatory submissions [8] - Praxis plans to provide a thorough update across its portfolio at the Investor R&D Day in Q2 2025 [8]