Core Insights - The pivotal Phase 3 clinical trial for Trappsol Cyclo™ is ongoing, with positive recommendations from the Data Monitoring Committee [1][2] - The company successfully closed a $25 million rights offering, enhancing its financial position to support the Trappsol Cyclo™ program and other opportunities [2] - Rafael Holdings reported a net loss of $12.1 million for Q4 FY 2025, an increase from a net loss of $4.5 million in the same period last year, primarily due to the consolidation of Cyclo Therapeutics' expenses [4][5] Financial Performance - As of July 31, 2025, the company had cash and cash equivalents of $52.8 million, following the $25 million rights offering closed in June 2025 [3] - For the three months ended July 31, 2025, research and development expenses rose to $7.5 million from $1.5 million year-over-year, reflecting increased spending at Cyclo Therapeutics [5] - General and administrative expenses for the same period were $5.5 million, up from $2.3 million in the prior year, also due to the inclusion of Cyclo's expenses [6] Yearly Overview - For the full fiscal year 2025, Rafael Holdings recorded a net loss of $30.5 million, an improvement from a net loss of $34.4 million in the previous year [7] - Research and development expenses for the full year were $12.8 million, compared to $4.2 million in the prior year, driven by the acquisition of Cyclo Therapeutics [8] - General and administrative expenses for the full year increased to $13.8 million from $8.9 million, attributed to the consolidation of Cyclo and other entities [9][10] Company Background - Rafael Holdings, Inc. is a biotechnology company focused on developing pharmaceuticals, with its lead candidate Trappsol® Cyclo™ aimed at treating Niemann-Pick Disease Type C1 [11]

Core Viewpoint - Rafael Holdings, Inc. announced that its subsidiary Cyclo Therapeutics will present two abstracts on Trappsol Cyclo™ for Niemann-Pick Disease Type C1 at the upcoming ICIEM conference in Kyoto, Japan [1] Group 1: Presentation Details - The first presentation will be an oral session titled "Hydroxypropyl-beta-cyclodextrin for the Long-Term Treatment of Niemann Pick Type C1: Efficacy and Safety Data from 4 Clinical Studies and the Ongoing Expanded Access Program" by Dr. Caroline Hastings on September 3, 2025 [1] - The second presentation will be a poster session titled "Trappsol Cyclo Open Label Treatment in the TransportNPC Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann Pick Disease Type C1" by Dr. Orna Staretz Chacham on the same day [1] Group 2: About Trappsol Cyclo™ - Trappsol Cyclo™ is a first-in-class proprietary cyclodextrin formulation administered intravenously that mobilizes lysosomal cholesterol, targeting the root cause of Niemann-Pick Disease Type C1 [2] - It has been shown to cross the blood-brain barrier, suggesting therapeutic concentrations in the central nervous system [2] Group 3: Study Program - The Phase 3 TransportNPC study is a randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of Trappsol Cyclo™ in patients aged 3 years and older, with a total enrollment of 94 patients across 25 sites in 13 countries [3] - The study duration is 96 weeks, with an interim analysis recommending continuation after 48 weeks [3] Group 4: Disease Overview - Niemann-Pick Disease Type C1 is a rare genetic disorder affecting approximately 1 in 100,000 live births, characterized by cholesterol accumulation leading to organ damage and life-limiting symptoms [8] - About 95% of individuals with NPC have mutations in the NPC1 gene, while 5% have mutations in the NPC2 gene [8] Group 5: Company Overview - Rafael Holdings, Inc. is a biotechnology company with a focus on developing treatments for rare and neurodegenerative diseases, including a 100% interest in Cyclo Therapeutics [9] - Cyclo Therapeutics is dedicated to developing Trappsol Cyclo™ for Niemann-Pick Disease Type C1 and is conducting multiple clinical trials for this indication [10]

Core Viewpoint - Rafael Holdings, Inc. successfully completed a $25 million rights offering, raising approximately $4 million through subscriptions for Class B common stock, with additional funding expected from a backstop private placement by CEO Howard Jonas [1][4]. Group 1: Rights Offering Details - The rights offering resulted in subscriptions for 3,130,480 shares of Class B common stock at an exercise price of $1.28 per share, generating gross proceeds of $4,007,014.40 [1]. - The subscription period ended on May 29, 2025, and shares were issued to participating stockholders on June 4, 2025 [2]. - Howard Jonas and his affiliates, holding about 32% of the company's outstanding common stock, did not exercise their subscription rights due to a commitment to backstop the offering [1][3]. Group 2: Financial Implications - The net proceeds from the rights offering and the backstop private placement are expected to be approximately $24.9 million after deducting related expenses [4]. - Following these transactions, the company anticipates having around 50,879,164 shares of Class B common stock and 787,163 shares of Class A common stock outstanding [5]. Group 3: Strategic Use of Proceeds - The proceeds from the rights offering will provide additional capital for regulatory approval efforts and the potential launch of Trappsol Cyclo™ pending positive interim results from the ongoing Phase 3 clinical trial for Niemann-Pick Disease Type C1 [3]. Group 4: Company Overview - Rafael Holdings, Inc. is involved in clinical and early-stage pharmaceutical companies, focusing on developing Trappsol Cyclo™ for Niemann-Pick Disease Type C1, along with other pharmaceutical and medical device ventures [7].