Core Viewpoint - Cassava Sciences, Inc. has appointed Dr. Joseph Hulihan as Chief Medical Officer to lead the clinical development of simufilam for Tuberous Sclerosis Complex (TSC)-related epilepsy [1][3][8] Company Overview - Cassava Sciences, Inc. is a clinical-stage biotechnology company focused on developing investigational treatments for central nervous system disorders, including TSC-related epilepsy [5] - Simufilam is a proprietary oral small molecule believed to modulate the activity of the filamin A protein, which is crucial for neuronal development [5] Appointment of Dr. Joseph Hulihan - Dr. Hulihan has over 25 years of experience in the development of therapeutics for epilepsy and neurological disorders, previously serving as CMO at Marinus Pharmaceuticals and in senior roles at Janssen Pharmaceuticals [2][4] - He is board certified in Neurology and Electroencephalography (EEG) and has been involved in over 25 late-stage neurology-focused clinical trials [2][4] Clinical Development Plans - The clinical program for simufilam as a potential treatment for TSC-related epilepsy is set to begin in the first half of 2026 [4][8] - Dr. Hulihan expressed enthusiasm for simufilam's novel mechanism of action and the upcoming proof-of-concept study [4]

Core Viewpoint - Ovid Therapeutics has entered into a definitive agreement with Immedica Pharma for the sale of future royalties related to ganaxolone sales outside of China, providing Ovid with a non-dilutive capital infusion of $7 million to support its ongoing operations [1][2]. Group 1: Transaction Details - Immedica will pay $7 million in cash to acquire 100% of the royalty rights held by Ovid, enhancing Immedica's focus on ganaxolone by gaining control of additional intellectual property rights [1]. - The transaction includes royalties associated with a previous agreement Ovid had with Marinus Pharmaceuticals regarding ganaxolone for CDKL5 deficiency disorder [4]. - Ovid recorded approximately $566,000 in ganaxolone royalty revenues in 2024, indicating the current revenue scale from this asset [2]. Group 2: Company Backgrounds - Ovid Therapeutics is a biopharmaceutical company focused on developing small molecule medicines for brain conditions with significant unmet needs, advancing a pipeline of novel candidates targeting neurological disorders [7]. - Immedica is a pharmaceutical company dedicated to the commercialization of medicines for rare diseases, with a global distribution network serving patients in over 50 countries [6].

ZTALMY® Clinical and Commercial Overview - ZTALMY® is FDA-approved for CDKL5 Deficiency Disorder (CDD) and has a differentiated mechanism of action addressing an unmet need[12, 13] - In a Phase 3 CDD trial, patients taking ganaxolone experienced a median reduction of 30.7% in monthly major motor seizures compared to 6.9% for placebo[15] - The most frequent adverse events reported during the double-blind phase were somnolence (36% vs 16%), pyrexia (18% vs 8%), and upper respiratory tract infection (10% vs 6%)[18] - Net product revenue for ZTALMY® in Q3 2024 was $8.5 million, a 56% increase from Q3 2023, with full year 2024 expected net product revenue between $33 million and $34 million[20] Ganaxolone Prodrug Program - The company is developing a second-generation ganaxolone prodrug with the goal of increasing efficacy, improving tolerability, and reducing dosing frequency[40] - Early PK work supports once-daily (QD) or twice-daily (BID) dosing and the ability to achieve higher steady-state serum concentrations with the prodrug[50] RAISE Trial in Refractory Status Epilepticus - In the RAISE trial, 13% of patients in the IV Ganaxolone arm experienced SE cessation within 30 minutes compared to 0% in the placebo arm (p<0.0001)[63] - 80% of patients in the IV Ganaxolone arm had no progression to IV anesthesia for 36 hours compared to 63% in the placebo arm (p=0.1619)[63] - The median percent reduction in EEG seizure burden from hours 1-36 was 93% for Ganaxolone compared to 36% for placebo (p=0.003)[66] Financial Status - As of September 30, 2024, the company had $42.2 million in cash and cash equivalents, providing a cash runway into Q2 2025[74] - Full year 2024 SG&A and R&D expenses are guided to be between $135 million and $138 million, including approximately $20 million in stock-based compensation[69]