Rafael Holdings, Inc. · NYSE:RFL
Rafael — Investor Presentation (October 2025)
23 slides from Rafael's investor presentation, Oct 10, 2025
Slide 1: October 2025 Investor Presentation
NASDAQ: RFL October 2025 | Investor Presentation
Slide 2: Forward-Looking Statements
Forward - Looking Statements Some of the information in this presentation relates to future events or future business and financial performance . Such statements constitute forward - looking information within the meaning of the Private Securities Litigation Act of 1995 . Such statements can be only
Slide 3: Focused on Bringing Trappsol Cyclo to Market
Clinical Drug Development Trappsol ® Cyclo 3 Focused on Bringing Trappsol ® Cyclo to Market Real Estate Holding Medical Devices Rafael Holdings, Inc. NYSE: RFL / NYSE American: RFLW
Slide 4: Investment Highlights
Investment Highlights 4 Experienced team with strong track record of success Lead program Trappsol ® Cyclo in Phase 3 trial for the treatment of Niemann - Pick Disease Type C 1 High unmet medical need in NPC; global market expected to reach >$920m by 2031 Independent unblinded Data Monitoring Commit
Slide 5: Leadership Team
5 Leadership Team Howard S. Jonas Chairman of the Board Karen Mullen, FFPM Interim Chief Medical Officer David Polinsky Chief Financial Officer Joshua Fine Chief Operating Officer
Slide 6: Trappsol Cyclo Is Being Evaluated in the Fully Enrolled TransportNPC Phase 3 Trial
6 Trappsol® Cyclo Is Being Evaluated in the Fully Enrolled TransportNPC Phase 3 Clinical Trial for Patients With Niemann - Pick Disease Type C1 48 - week interim analysis (IA) from Phase 3 Trial completed June 13 Data monitoring committee recommended continuing study to the 96 - week final analysis
Slide 7: Niemann-Pick Disease Type C Ongoing Pivotal Phase 3 Study
Niemann - Pick Disease Type C 1 Ongoing Pivotal Phase 3 Study 7
Slide 8: Niemann Pick Type C1 (NPC) Market Overview
Niemann Pick Type C1 (NPC) Market Overview 8 Incidences 1/100,000 ~35/year in U.S. ~3% are age 3 and below Diagnosed Patients ~97% age 3 and up ~60% age 16 and up Median Survival Early infantile (2m - 2): 4.6y Late infantile (3 - 6): 9.4y Juvenile (7 - 15): 15.4y Adult (16+): 12.2y Existing Cases >9
Slide 9: Trappsol Cyclo Differentiated MOA
Trappsol® Cyclo Differentiated MOA 9 NPC1, a key regulator in late endosomal/lysosomal compartments, regulates cholesterol efflux Biallelic NPC1 mutations trap cholesterol in lysosomes, block autophagy and drive cell death Trappsol ® Cyclo facilitates cholesterol release independent of NPC1/NPC2, by
Slide 10: Summary of Completed Studies and Open-Label Extension
10 Trappsol ® Cyclo Summary of Completed Studies and Open - Label Extension Trappsol ® Cyclo reaches the cerebrospinal fluid within hours of IV infusion, modulating cholesterol metabolism and clearing cellular cholesterol, mirroring preclinical NPC model effects Study 101 1 Study 102 Demonstrated Tr
Slide 11: Phase 1/2 Study (CTD-TCNPC-201)
11 Phase 1/2 Study (CTD - TCNPC - 201) • Therapeutic benefit may be seen by 48 weeks vs. disease progression • Small cohort (9 patients) limits extensive analysis • Transient variations may mimic disease progression but should lessen in Phase 3 (CTD - TCNPC - 301) • 9 completers : 2 at 1500 mg/kg |
Slide 12: Phase 1/2 Study (CTD-TCNPC-201): Continued
12 Phase 1/2 Study (CTD - TCNPC - 201): Continued • 9 completers : 2 at 1500 mg/kg | 4 at 2000 mg/kg | 3 at 2500 mg/kg • 3 excluded from analysis : • 1 with only baseline data • 2 discontinued before Week 48 (non - safety reasons) Data Included Key Outcomes Individual data suggestive of potential be
Slide 13: Phase 1/2 Study (CTD-TCNPC-201): Continued, SARA Data
13 Phase 1/2 Study (CTD - TCNPC - 201): Continued Dosage data suggestive of potential benefit on SARA, an exploratory measure • Multiple domains analyzed: gait, stance, sitting, speech, finger chase (L/R), nose - finger (L/R), fast alt hands (L/R), heel - shin (L/R) • 2500 mg/kg & 2000 mg/kg groups
Slide 14: Ongoing Long-Term Extension (OLE) Study (CTD-TCNPC-102)
14 Ongoing Long - Term Extension (OLE) Study (CTD - TCNPC - 102) Data Included is from an August 2023 Data Cut Key Outcomes from the OLE • To date, 3 patients remain on active treatment • All receive 1500 mg/kg biweekly via in - home infusions • All 5 patients showed slowed disease progression, with
Slide 15: Safety From Completed and Ongoing Long-Term Treatment
15 Safety From Completed and Ongoing Long - Term Treatment Treatment - Emergent Adverse Events majority mild to moderate in severity, manageable and monitorable and most considered unrelated to Trappsol ® Cyclo No evidence of any untoward effects of Trappsol ® Cyclo on core organ systems (cardiovasc
Slide 16: Ongoing Pivotal Phase 3 Study in Niemann-Pick Disease Type C1
16 Ongoing Pivotal Phase 3 Study in Niemann - Pick Disease Type C1 104 enrolled: 94 Core Study; 10 Sub Study Number of Subjects 30+ across 14 countries Current Sites 96 - week trial Duration 1 2000 mg/kg via IV infusion Dose Requested as part of our adopted PIP to potentially treat as a prophylactic
Slide 17: Trial Design: Core Study (≥3 Years of Age)
17 Trial Design: Core - Study (≥3 Years of Age) – Global Double - Blind, Randomized, Placebo - Controlled, Parallel - Group, Multicenter Study 94 randomized (2:1 to Placebo) Number of Subjects NPC Composite Severity Score Primary Endpoint SCAFI, Swallow, Vineland - 2 Secondary Endpoints Inclusive of
Slide 18: Trial Design: Sub-Study (Newborn to <3 Years of Age)
18 Trial Design: Sub - Study (Newborn to <3 Years of Age) – Global Open Label Phase Baseline Assessment Final Analysis (Week 96) Screening (2 Weeks) Hydroxypropyl - Beta - Cyclodextrin + SOC Administered IV Every 2 Weeks Continuation of HP β CD + SOC until week 192 V1 Week - 8 to - 5 V2 Week - 4 to
Slide 19: Clinical Global Impression – Severity (CGI-S) Bar Plot
19 Clinical Global Impression – Severity (CGI - S) Bar Plot at data cutoff on 14 - May - 2025 • Graphs shows individual patients (N=10) • 2 patients have withdrawn from the study at week 48 for non - safety related reasons • Trappsol ® Cyclo continues to demonstrate stabilization or improvement in t
Slide 20: Clinical Global Impression – Change (CGI-C) Bar Plot
20 Clinical Global Impression – Change (CGI - C) Bar Plot at data cutoff on 14 - May - 2025 • Graphs shows individual patients (N=10) • 2 patients have withdrawn from the study at week 48 for non - safety related reasons • Trappsol ® Cyclo continues to demonstrate stabilization or improvement in the
Slide 21: Safety from Ongoing Substudy
21 Safety from ongoing Substudy at data cutoff on 14 - May - 2025 • T here were 146 AEs, with the majority reported as mild (69%) or moderate (29%), 3 reported as severe and the remainder reported as Serious Adverse Events(SAEs) (14%); and • No SAEs were considered by the principal investigators as
Slide 22: Investment Summary
22 Investment Summary Phase 3 Clinical trial to be completed 1H 2026. Potential NDA and MAA submissions to FDA and EMA by end of 2026 1. Intelligence, D. (n.d.). Niemann Pick Disease market size, sustainable insights and growth report 2025 - 2033 . DataMIntelligence 2. As of April 30, 2025; includes
Slide 23: Thank You!
RAFAEL Holdings, Inc. Thank You!
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