Rafael Holdings, Inc. · NYSE:RFL
Rafael — Investor Presentation (July 2026)
24 slides from Rafael's investor presentation, Jul 8, 2026
Slide 1: July 2026 Investor Presentation
NYSE: RFL July 2026 | 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 predi
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 C1 High unmet medical need in NPC; global market expected to reach >$920m by 2031 Independent unblinded Data Monitoring Committee
Slide 5: Leadership Team
5 Leadership Team Howard S. Jonas Chairman of the Board Karen Mullen, FFPM Chief Medical Officer David Polinsky Chief Financial Officer Joshua Fine Chief Operating Officer
Slide 6: Trappsol Cyclo 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 Potent
Slide 7: Niemann-Pick Disease Type C1 Ongoing Pivotal Phase Study
Niemann-Pick Disease Type C1 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 >9,000 i
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, byp
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 1011 Study 102 Demonstrated Trappso
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 | 4 at 2000
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 benefit on
Slide 13: Phase 1/2 Study (CTD-TCNPC-201): Continued
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 showed s
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 some st
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 (cardiovascular,
Slide 16: Ongoing Pivotal Phase Study in Niemann-Pick Disease Type C1
16 Ongoing Pivotal Phase 3 Study in Niemann-Pick Disease Type C1 Number of Subjects 104 enrolled: 94 Core Study; 10 Sub Study Current Sites 30+ across 14 countries Duration 96-week trial Dose 2000 mg/kg via IV infusion Substudy (Patients 0-3)2 Requested as part of our adopted PIP to potentially trea
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 Number of Subjects 94 randomized (2:1 to Placebo) Primary Endpoint NPC Composite Severity Score Secondary Endpoints SCAFI, Swallow, Vineland-2 Exploratory Endpoints
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 HPβCD + SOC until week 192 V1 Week-8 to -5 V2 Week-4 to -1 V3 Week 0
Slide 19: Clinical Global Impression–Change (CGI-C) Bar Plot
19 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 majority o
Slide 20: Safety from Ongoing Substudy
20 Safety from ongoing Substudy at data cutoff on 14-May-2025 • There were 146 AEs, the reported as mild (69%) moderate (29%), and Severe (2%) • No SAEs were considered by the principal investigators as related to or possibly related to study drug.
Slide 21: Trappsol Cyclo Positioning in the Emerging NPC Treatment Paradigm
21 Trappsol® Cyclo Positioning in the Emerging NPC Treatment Paradigm • NPC landscape evolving from no therapy → multi-drug paradigm • Recent approvals (Zevra, IntraBio) enable combination approaches • Distinct upstream mechanism — removes cholesterol independent of NPC1/NPC2 • Complementary to appr
Slide 22: Trappsol Cyclo vs Zevra (Miplyffa) and IntraBio (Aqneursa)
Attribute Trappsol® Cyclo Zevra (Miplyffa) IntraBio (Aqneursa) Mechanism of Action Direct Cholesterol clearance (root cause) lysosomal/ proteostasis enhancement, approved prescribing information states Unknown. neurological function modulation, approved prescribing information states Unknown. Therap
Slide 23: Investment Summary
23 Investment Summary Phase 3 Clinical trial LPLV June 2026; NDA submission and Topline data expected 2nd half 2026 1. Intelligence, D. (n.d.). Niemann Pick Disease market size, sustainable insights and growth report 2025-2033. DataMIntelligence 2. As of April 30, 2026 Ability to Realize Potential V
Slide 24: Thank You!
RAFAEL Holdings, Inc. Thank You! NYSE: RFL