Rafael Holdings’ remibetadex misses primary endpoint
The timeline lists a December 27, 2026 FDA filing decision, with potential mid-2027 action if the NDA is filed and priority review granted.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Rhea-AI Filing Summary
Rafael Holdings, Inc. reported topline Phase 3 results for remibetadex, its investigational Niemann-Pick disease type C treatment, and said it submitted a New Drug Application in October 2026 with priority review requested. The primary endpoint was not statistically significant: reported disease-progression slowing was 64% in the overall 94-patient population (p=0.19). A prespecified analysis of 78 patients who were on miglustat and/or leucine before baseline reported 71% slowing (p=0.046). Results are subject to full analysis and regulatory review.
Over 96 weeks, serious treatment-emergent adverse events occurred in 35.9% of remibetadex participants versus 16.7% on placebo; treatment-related serious events occurred in 3.1% and 3.3%, respectively.
The FDA timeline lists a December 27, 2026 filing decision and potential action around mid-2027 if FDA files the NDA and grants priority review; timing is subject to FDA review. Risk disclosures cite substantial doubt about the company’s ability to continue as a going concern and a need for additional capital.
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Positive
- Minor pointPrespecified subgroup showed 71% disease-progression slowing (p=0.046).
Negative
- Major pointOverall Phase 3 primary endpoint was not statistically significant (p=0.19).
- Major pointRisk disclosures cite substantial doubt about going-concern ability and additional capital needs.
- Moderate pointSerious treatment-emergent adverse events: 35.9% with remibetadex vs. 16.7% with placebo.
Filing Explained
July 31, 2026, cash and long-term investments equaled 332.9 days of reported operating cash use, a historical comparison, not a runway forecast.
The 8-K furnishes an investor presentation under Regulation FD; the presentation says Rafael is evaluating financing and strategic options for NDA review and launch. It specifies no financing structure, committed amount, proceeds, or securities to be issued, so no new funding or ownership dilution is established by this disclosure.
As of
Sources and calculations
- Rafael Holdings Form 8-K, including Exhibit 99.1 (2026-11-09)
- Rafael Holdings financial data (2026Q4)
- Available liquidity against the last reported quarterly operating outflow, in days at that rate ($22,100,000 + $750,000) / ($8,374,000 / 122) = 332.9 days
8-K Event Classification
Key Figures
Key Terms
New Drug Application regulatory
priority review regulatory
4-domain NPC Clinical Severity Scale medical
treatment-emergent adverse event medical
FAQ
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What did RFL’s Phase 3 trial show for remibetadex?
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
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Item 7.01. Regulation FD
From November 9, 2026, the slide presentation (the “Presentation”) attached hereto as Exhibit 99.1 will be available on the Registrant’s website at the following link: https://rafaelholdings.irpass.com/.
The Registrant is furnishing the information contained in this Report, including Exhibit 99.1, pursuant to Item 7.01 of Form 8-K promulgated by the Securities and Exchange Commission (the “SEC”). This information shall not be deemed to be “filed” with the SEC or incorporated by reference into any other filing with the SEC. In addition, the Presentation contains statements intended as “forward-looking statements” that are subject to the cautionary statements about forward-looking statements set forth in the Presentation.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
| Exhibit No. | Document | |
| 99.1 | Investor Presentation |
1
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| RAFAEL HOLDINGS, INC. | |||
| By: | /s/ David Polinsky | ||
| Name: | David Polinsky | ||
| Title: | Chief Financial Officer | ||
Dated: November 9, 2026
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EXHIBIT INDEX
| Exhibit Number | Document | |
| 99.1 | Investor Presentation |
3
Exhibit 99.1
NYSE: RFL November 2026 | Investor Presentation
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 be only predictions and the actual events or results may differ from those discussed due to, among other things, the risks described in the public filings and other publications of Rafael Holdings, Inc . Forward - looking statements are identified by words such as “anticipates”, “projects”, “expects”, “plans”, “intends”, “believes”, “estimates”, “target”, and other similar expressions that indicate trends and future events . The market data and certain other statistical information used throughout this presentation are based on independent industry publications, governmental publications, reports by market research firms or other independent sources . Some data are also based on the Company’s good faith estimates . In addition, this presentation includes summaries of scientific activities and topline data that have been condensed to aid the reader in gaining general understanding, but FDA or comparable foreign regulatory authorities may disagree with our interpretation of these data . The information about Rafael Holdings, Inc . and its subsidiaries is solely for information purposes and is not to be construed as an offer to sell or the solicitation of an offer to buy any security in any state . • Factors that could cause the Company’s results to differ materially from those expressed in forward - looking statements include, without limitation, substantial doubt about the Company’s ability to continue as a going concern and its need for additional capital, which may not be available on acceptable terms or at all ; the Phase 3 TransportNPC study did not meet its primary endpoint, and the FDA may not accept the remibetadex NDA for filing, grant priority review or approve it ; remibetadex may not be eligible for, or receive, a rare pediatric disease priority review voucher ; the Company’s reliance on its remibetadex product, which may never receive regulatory approval ; the Company’s ability to commercialize any of its proposed drug products if it any of them were to receive s regulatory approval ; the outcome of the Company’s clinical trials, which may not support the Company’s proposed product claims or may result in adverse side effects ; the cost and timing of the Company’s clinical trials ; the Company’s reliance on third parties to conduct nonclinical and clinical trials and to produce, package and label its products ; other risks associated with being regulatory - stage biotechnology company ; a small number of the Company’s customers account for a substantial portion of our revenue, and the loss of any of these customers would materially decrease our revenues ; and the Company may be negatively affected by currency exchange rate fluctuations . This presentation is not to be copied, transmitted, displayed, distributed (for compensation or otherwise), or altered in any way without the prior written consent of Rafael Holdings, Inc . 2
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.
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 who took remibetadex and miglustas /leucine versus placebo and miglustat /leucine therapy High unmet medical need in NPC; global market expected to reach >$920m by 2033 1 Orphan Drug Designation (US and EU), Fast Track Designation (US) and Rare Pediatric Disease Designation (US), providing potential PRV eligibility Potential non - dilutive value: Rare Pediatric Disease PRV program extended to September 2029; the four most recent vouchers sold for an average of ~$200M 2 Preparing for potential commercial launch Experienced team with strong track record of success Rafael Holdings (NYSE: RFL) is a regulatory - stage biotechnology company focused on bringing remibetadex, its lead asset and primary potential value driver, to patients with NPC EU = European Union; NDA = New Drug Application; NPC = Niemann - Pick disease type C; NYSE = New York Stock Exchange; PRV = priori ty review voucher; US = United States. Rafael Holdings, Inc. (Sept 30, 2026). Topline results from the pivotal Phase 3 TransportNPC study of Trappsol® Cyclo in NPC. (1) DataM Intelligence. Niemann Pick Disease Market Size, Sustainable Insights and Growth Report 2025 – 2033. (2) Average of the four most recent publicly announced Rare Pediatric Disease PRV sales: Egetis Therapeutics ($190M, Nov. 4, 202 6), Ultragenyx ($203M, Oct. 14, 2026), Ultragenyx ($210M, Oct. 7, 2026) and Denali Therapeutics ($195M, June 18, 2026); Source: company press releases.
Niemann - Pick Disease Type C Pivotal Phase 3 Study 5
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, earlier phase studies, and an overall survival analysis vs external controls Pre - NDA meeting with FDA completed (February 2026) Only investigational NPC therapy given IV – addressing visceral disease, studied from birth, and not dependent on swallowing FDA = U.S. Food and Drug Administration; IV = intravenous; NDA = New Drug Application; NPC = Niemann - Pick disease type C. Rafael Holdings, Inc. (June 10, 2026). Completion of Last Patient Last Visit for the pivotal Phase 3 TransportNPC study evaluating Trappsol® Cyclo in NPC.
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 and young children Substudy (Patients 0 - 3) 1 96 - week LPLV completed June 2026; topline data reported September 2026; NDA submitted October 2026 ¹ Substudy conducted outside the US only; patients are not randomized and all patients aged 0 – 3 receive remibetadex. CGI = Clinical Global Impression; EMA = European Medicines Agency; IV = intravenous; LPLV = last patient, last visit; NDA = N ew Drug Application; NPC = Niemann - Pick disease type C; PIP = Paediatric Investigation Plan; SCAFI = Spinocerebellar Ataxia Functional Index; US = United S tates. 94 randomized (2:1 to Placebo) Number of Subjects NPC Composite Severity Score Primary Endpoint SCAFI, Swallow, Vineland - 2 Secondary Endpoints Individual domains, CGI, responders analysis, liver and lung function Exploratory Endpoints TransportNPC Phase 3 Study (Core + Substudy) Core Study (≥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 Continuation of remibetadex+ SOC until week 192 V1 Week - 8 to - 5 V2 Week - 4 to - 1 V3 Week 0 V4 Week 2 V5 Week 4 V9 Week 12 V15 Week 24 V21 Week 36 V27 Week 48 V33 Week 60 V39 Week 72 V45 Week 84 V51 Week 96 V52 Week 98 Study Drug Infusions Following Required Assessments every 2 weeks IV = intravenous; SOC = standard of care; V = study visit.
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 severe ) Remibetadex Placebo Primary Endpoint (n=94) 64% slowing of disease progression vs. placebo Difference −0.81 (95% CI −2.04, 0.41) p=0.19 Directionally favorable; not statistically significant Prespecified Analysis 71% slowing of disease progression in patients on miglustat and/or leucine (n=78) prior to baseline Difference −1.11 (95% CI −2.19, −0.02) p=0.046 Statistically significant 4DNPCSS = 4 - domain NPC Clinical Severity Scale (higher scores indicate greater severity); CI = confidence interval; LS = least s quares; NPC = Niemann - Pick disease type C. % slowing = 1 − (remibetadex LS mean change ÷ placebo LS mean change). Prespecified analysis population represents ~83% of patients . Data ; subject to full analysis and regulatory review. Source: Rafael Holdings, Inc. press release, September 30, 2026. Leucine is an over - the - counter product available in some European countries (n=3); it is not approved by the U.S. Food and Drug Administration.
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% Serious TEAEs 3.3% 3.1% Treatment - related serious TEAEs 13.3% 15.6% Hearing - related TEAEs 0 0 TEAEs resulting in death • Adverse events largely consistent with the underlying disease • Low rates of hearing - related AEs • Hearing - related TEAEs mostly mild or moderate; one severe treatment - related event of bilateral deafness • One remibetadex participant discontinued due to a TEAE AE = adverse event; TEAE = treatment - emergent adverse event. Data; subject to full analysis and regulatory review. Source: Rafae l Holdings, Inc. press release, September 30, 2026.
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 as HPβCD) 2000 mg/kg IV every 2 weeks. Week 96 N=7: 2 patients discontinued (caregiver decision) and 1 had not reached Week 96. * By the investigator. 17D - NPC - SS = 17 - domain NPC Clinical Severity Scale; AE = adverse event; CGI = Clinic al Global Impression; CGI - C = Clinical Global Impression – Change; CGI - S = Clinical Global Impression – Severity; CTCAE = Common Terminology Criteria for Adverse Events; Gr = grade; HPβCD = hydroxypropyl - β - cyclodextrin; IV = intravenous; NPC = Niemann - Pick disease type C; SAE = serious adverse event; Wk = week. Source: Ezgu F, et al. TRANSPORT - NPC Pediatric Sub - Study tr ial update, SSIEM 2026.lle Severe Gr 3 Moderate Gr 2 Mild Gr 1 Total Adverse events 3 39 132 174 Total AEs 0 0 2 2 AEs possibly related / related* 1 14 12 27 SAEs 0 0 0 0 SAEs possibly related / related* • No SAEs considered related to study drug by investigators • No permanent hearing loss requiring a hearing aid assessment • AE profile in line with compassionate use, earlier studies , and the parallel Phase 3 study 86% stable or improved on CGI - S at Week 96 (6/7) 71% stable or improved on CGI - C at Week 96 (5/7) 30% 29% 50% 57% 50% 57% 40% 14% 20% 14% 10% 29% CGI-S Wk 48 (N=10) CGI-S Wk 96 (N=7) CGI-C Wk 48 (N=10) CGI-C Wk 96 (N=7) Improved Stable Deteriorated In rapidly progressive infantile - onset NPC, stabilization and improvement are considered clinically - meaningful outcomes indicative of clinical improvement .
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 INPDR controls (n=93) HR 0.154 (95% CI 0.025 – 0.950) p=0.044 94% Lower relative mortality risk vs. expanded historical controls * (n=133) HR 0.057 (95% CI 0.011 – 0.306) p=0.0008 CI = confidence interval; HR = hazard ratio; INPDR = International Niemann - Pick Disease Registry; NPC = Niemann - Pick disease typ e C. Survival analysis pooled infantile - onset patients across the remibetadex program; *expanded controls add matched cohorts from published natural history studies. Topli ne data; subject to full analysis and regulatory review. Ezgu F, et al. SSIEM 2026. Rafael Holdings, Inc. (Sept 30, 2026). Topline results from the pivotal Phase 3 TransportNPC study of Trappsol® Cyclo in NPC Primary Analysis Secondary Analysis Primary Analysis
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 patient, last visit Sept. 2026 Phase 3 topline data reported Oct. 2026 NDA submitted to FDA Dec. 27, 2026 Day 60 FDA filing decision. Priority review determination and potential PDUFA date at Day 74 Feb. 2027 Full Phase 3 results presented by [investigator] at medical meeting ~June 2027 Potential PDUFA target date (if priority review granted) ● Completed ○ Upcoming Regulatory designations for remibetadex in NPC Orphan Drug Designation (US and EU) Fast Track Designation (US) Rare Pediatric Disease Designation (US): potential PRV eligibility EU = European Union; FDA = U.S. Food and Drug Administration; NDA = New Drug Application; NPC = Niemann - Pick disease type C; PDU FA = Prescription Drug User Fee Act; PRV = priority review voucher; US = United States. Priority review PDUFA goal for a new molecular entity: 6 mon ths after the 60 - day filing date (~8 months from submission). If priority review is not granted, the standard review goal is 10 months after the 60 - day filing date ( ~12 months from submission). Timing of FDA milestones following submission is subject to FDA review. FDA filing and approval are not assured.
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 protein, complementary to existing therapies • Clinical studies have evaluated both systemic and neurological aspects of disease, the proposed indication is for the treatment of the neurovisceral manifestations of Niemann - Pick Disease Type C (NPC) in adult and pediatric patients from birth • Generally well tolerated in clinical studies
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 Cases >9,000 in 80 countries ~900 – 943 in U.S. • NPC can manifest at any age and is characterized by progressive organ failure and neurodegeneration • Many patients may go undiagnosed or untreated given diagnostic challenges and disease rarity • Late onset of symptoms can lead to longer life spans, but it is extremely rare for any person with NPC to reach age 40 Sources: Battisti et al, 2003; Schicks et al, 2013; Sévin et al, 2007; Trendelenburg et al, 2006; Vanier et al, 2010; Burton BK, Ellis AG, Orr B, et al. Estimating the prevalence of Niemann - Pick disease type C (NPC) in the United States. Mol Genet Metab. 2021; (134) doi: 10.1016/j.ymgme.2021.06.011, Data commissioned by Tessellon Inc, for Cyclo Ther apeutics, LLC.; Niemann Pick Foundation NPC = Niemann - Pick disease type C; U.S. = United States. NPC is a rare, fatal, autosomal recessive lysosomal storage disease that disrupts lipid trafficking within cells, leading to the toxic buildup of unesterified cholesterol and glycosphingolipids in major organs. Overview
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 untreated given disease rarity Illustrative U.S. Revenue Annual revenue at analog pricing³ ($M) $1.27M Therapy B WAC $941K Therapy B net $577K Therapy A net Patients treated $127 $94 $58 100 (~11%) $254 $188 $115 200 (~21%) $382 $282 $173 300 (~32%) Per - patient annual price (columns) × patients treated (rows; % of ~943 prevalent cases) Illustrative only; not a forecast . Remibetadex is an investigational drug that has not been approved by FDA or any other regulatory authority, and its pricing has not been established. Market Positioning • Two oral NPC therapies approved in the U.S. in 2024 • Remibetadex: IV therapy; can be given from birth, for neurological and visceral manifestations of NPC. • Orphan Drug Designation: potential 7 years of U.S. market exclusivity upon approval (1) Burton BK et al. Mol Genet Metab. 2021;134 (~2.9 cases per million). (2) Battisti 2003; Schicks 2013; Sévin 2007; Trendel enb urg 2006; Vanier 2010; Niemann - Pick Foundation. (3) Column prices are analogs from the two FDA - approved NPC therapies: annual net launch prices of $576,661 (Therapy A) and $940,586 (Therapy B) (Institute for Clinical and Economic Review, 2026 Launch Price and Access Report, Oct. 2026); maximum Therapy B wholesale acquisition co st (WAC) of $106K/month, ~$1.27M/year (Reuters, Sept. 23, 2024). Remibetadex is an investigational drug and not approved by the FDA. FDA = U.S. Food and Drug Administration; IV = intravenous; NPC = Niemann - Pick disease type C; U.S. = United States.
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, 2026. (3) Annual net launch prices of the two FDA - approved NPC therapies ($576,661 and $940,586): Institute for Clinical and Economic Review, 2026 Launch Price and Access Report (Oct. 2026); maximum list price of $106K/month (~$1.27M/year) for the higher - priced therapy: Reuters, Sept. 23, 2024. (4) Rafael Holdings, Inc. press release and Form 8 - K, April 22, 2026 (U.S. Patent No. 12,285,440). FDA = U.S. Food and Drug Administration; LPLV = last patient, last visit; MIT = Massachusetts Institute of Technology; NDA = New Drug Application; NPC = Niemann - Pick disease type C; US = United States. Ability to Realize Potential Value Creation Significant Commercial Opportunity ~ $22.1M Cash Balance 2 x Global NPC market expected to reach >$920m by 2033 1 x Approved U.S. NPC therapies priced at ~$577K – $1.27M per year (net launch price to maximum list price) 3 x Streamlined company focused on bringing remibetadex to market x Platform potential: exclusive MIT license (April 2026) for cyclodextrins in ApoE4+ Alzheimer’s disease 4 x Evaluating financing and strategic options for NDA review and launch
NYSE: RFL Thank You! 18