Rafael Holdings, Inc. · NYSE:RFL
Rafael — Investor Presentation (November 2026)
18 slides from Rafael's investor presentation, Nov 9, 2026
Slide 1: November 2026 Investor Presentation
NYSE: RFL November 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 Reform Act of 1995 . Such statements can
Slide 3: Leadership Team
3 Leadership Team Howard S. Jonas Chairman and Chief Executive Officer Karen Mullen, FFPM Chief Medical Officer David Polinsky Chief Financial Officer Joshua Fine Chief Operating Officer FFPM = Fellow of the Faculty of Pharmaceutical Medicine.
Slide 4: Investment Highlights
Investment Highlights 4 Topline Phase 3 data reported September 2026; NDA submitted October 2026 Phase 3 primary endpoint not statistically significant – Prespecified analysis showed a 71% relative slowing of disease progression compared with placebo, which was significant (p=0. 046 ) in patients wh
Slide 5: Niemann-Pick Disease Type C Pivotal Phase 3 Study
Niemann - Pick Disease Type C Pivotal Phase 3 Study 5
Slide 6: Remibetadex: Key Highlights
6 Remibetadex: Key Highlights Potential to become treatment for the neurovisceral manifestations of NPC in adult and pediatric patients from birth (proposed indication) NDA submission includes: data from the largest phase 3 study conducted i n NPC patients to date, pediatric substudy results, earlie
Slide 7: Pivotal Phase 3 Study in Niemann-Pick Disease Type C
7 Pivotal Phase 3 Study in Niemann - Pick Disease Type C 104 enrolled: 94 Core Study; 10 Sub Study Number of Subjects 30+ across 13 countries Sites 96 - week trial Duration 2000 mg/kg via IV infusion Dose Open - label (n=10), conducted under the agreed EMA PIP to evaluate early treatment in infants
Slide 8: Core Study Design (≥3 Years of Age)
8 Core Study Design (≥3 Years of Age) Global, double - blind, randomized, placebo - controlled, parallel - group, multicenter study Baseline Assessment Final Analysis (Week 96) Interim Analysis Completed (Week 48) Screening (2 Weeks) Remibetadex + SOC vs. Placebo + SOC Administered IV Every 2 Weeks
Slide 9: TransportNPC Phase 3 Results: Efficacy at Week 96
9 TransportNPC Phase 3 Results: Efficacy at Week 96 Primary endpoint: change from baseline in the 4 - domain NPC Clinical Severity Scale (4DNPCSS) LS Mean Change at Week 96 0.46 0.46 1.28 1.57 0.0 0.5 1.0 1.5 2.0 2.5 Overall population (n=94) Miglustat and/or leucine (n=78) Points (higher = more sev
Slide 10: TransportNPC Phase 3 Topline Results: Safety
10 TransportNPC Phase 3 Topline Results: Safety Generally, well tolerated with no new safety signals observed, although serious adverse events occurred more frequently with remibetadex than with placebo Safety Over 96 Weeks Placebo Remibetadex Participants with… 100.0% 93.8% Any TEAE 16.7% 35.9% Ser
Slide 11: Pediatric Sub-Study: Safety and Efficacy Data Through Week 96
11 Pediatric Sub - Study: Safety and Efficacy Data Through Week 96 Newborn to <3 years (N=10): no treatment - related SAEs; 86% stable or improved on CGI - S at Week 96 Safety Efficacy: CGI Responder Analysis Data Cutoff 19 - Mar - 2026 . Open - label sub - study; remibetadex ( previously identified
Slide 12: Infantile Onset Overall Survival Analysis
12 A separate Infantile Onset Overall Survival Analysis (Including Infantile NPC patients from 201,301 and substudy vs matched controls (INPDR)) Overall Survival Infantile - onset NPC: remibetadex - treated patients (n=41) vs. matched external controls 85% Lower relative mortality risk vs. matched I
Slide 13: Regulatory Path and Upcoming Catalysts
13 Regulatory Path and Upcoming Catalysts NDA submitted October 2026 with priority review requested; potential FDA action ~mid - 2027 if FDA files the NDA and grants priority review Regulatory Progress and Catalysts Feb. 2026 Pre - NDA meeting with FDA on 48 - week data June 2026 Phase 3 last patien
Slide 14: Potential Remibetadex Positioning in the Emerging NPC Treatment Paradigm
14 Potential Remibetadex Positioning in the Emerging NPC Treatment Paradigm • NPC treatment landscape evolving from no approved therapy to combination regimens • Remibetadex has been designed to have a distinct mechanism of action that removes accumulated cholesterol independent of abnormal NPC prot
Slide 15: Niemann-Pick Disease Type C (NPC) Market Overview
Niemann - Pick Disease Type C (NPC) Market Overview 15 Incidence 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
Slide 16: U.S. Commercial Opportunity
16 U.S. Commercial Opportunity Ultra - rare disease with established premium orphan pricing and an emerging combination paradigm U.S. Patients ~900 – 943 estimated prevalent cases¹ ~35 new cases per year² Diagnosed patients by age² ≤3: ~3% 3 – 15: ~37% 16+: ~60% Many patients may go undiagnosed or u
Slide 17: Investment Summary
17 Investment Summary Phase 3 Clinical trial LPLV June 2026; topline data reported Sept. 2026; NDA submitted Oct. 2026 (1) Intelligence, D. (n.d.). Niemann Pick Disease market size, sustainable insights and growth report 2025 - 2033. DataMIntellige nce (2) Cash and cash equivalents as of July 31, 20
Slide 18: Thank You!
RAF AE L Holdings, Inc. Thank You! NYSE: RFL
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