Form 8-K

8-K — Rafael Holdings, Inc.

Accession: 0001213900-26-104876

Filed: 2026-09-30

Period: 2026-09-30

CIK: 0001713863

SIC: 2834 (PHARMACEUTICAL PREPARATIONS)

Item: Regulation FD Disclosure

Item: Other Events

Item: Financial Statements and Exhibits

Documents

8-K — ea0307125-8k_rafael.htm (Primary)

EX-99.1 — PRESS RELEASE, DATED SEPTEMBER 30, 2026 (ea030712501ex99-1.htm)

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities

Exchange Act of 1934

Date of Report (Date of earliest event reported):

September 30, 2026

RAFAEL HOLDINGS, INC.

(Exact name of registrant as specified in its

charter)

Delaware

1-38411

82-2296593

(State or other jurisdiction of

Incorporation)

(Commission File Number)

(IRS Employer

Identification No.)

520 Broad Street

Newark, New Jersey

07102

(Address of principal executive offices)

(Zip Code)

Registrant’s telephone number, including

area code: 212 658-1450

Not Applicable

(Former name or former address, if changed since

last report.)

Check the appropriate box below if the Form 8-K filing is intended

to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2.

below):

¨ Written communications pursuant to Rule 425 under the Securities

Act (17 CFR 230.425)

¨ Soliciting material pursuant to Rule 14a-12 under the Exchange

Act (17 CFR 240.14a-12)

¨ Pre-commencement communications pursuant to Rule 14d-2(b) under

the Exchange Act (17 CFR 240.14d-2(b))

¨ Pre-commencement communications pursuant to Rule 13e-4(c) under

the Exchange Act (17 CFR 240.13e-4(c))

Indicate by check mark whether the registrant

is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the

Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by

check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial

accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Securities registered pursuant to Section 12(b)-2 of the Exchange Act:

Title of each class

Trading Symbol

Name of each exchange on which registered

Class B common stock, par value $0.01 per share

RFL

New York Stock Exchange

Item 7.01. Regulation

On September 30, 2026,

Rafael Holdings, Inc. (the “Company”) distributed over a wire service and posted the attached press release (the “Release”)

to the investors page of the Company’s website (www.rafaelholdings.com) announcing

topline efficacy and safety data from its Cyclo Therapeutics, LLC subsidiary’s pivotal Phase 3 TransportNPC™ study (CTD-TCNPC-301)

evaluating Trappsol® Cyclo™ (intravenous hydroxypropyl-β-cyclodextrin) in 94 patients diagnosed with Niemann-Pick Disease

Type C (NPC).

A copy of the Release is furnished herewith as Exhibit 99.1 and is incorporated herein by reference.

The Company is furnishing the information contained in this Report, including Exhibit 99.1, pursuant to Item 2.02 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 unless otherwise expressly stated in such filing. In addition, this Report and the press release contain statements intended as “forward-looking statements” that are subject to the cautionary statements about forward-looking statements set forth in the press release.

Item 8.01. Other Events.

The information contained in Item 7.01 above is incorporated herein by reference into this Item 8.01.

Item 9.01 Financial Statements

and Exhibits.

(d) Exhibits.

Exhibit No.

Document

99.1

Press Release, dated September 30, 2026.

104

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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: September 30, 2026

EXHIBIT INDEX

Exhibit

Number

Document

99.1

Press Release, dated September 30, 2026.

104

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EX-99.1 — PRESS RELEASE, DATED SEPTEMBER 30, 2026

EX-99.1

Filename: ea030712501ex99-1.htm · Sequence: 2

Exhibit 99.1

Rafael Holdings Announces Topline Results from the Pivotal Phase

3 TransportNPC™ Study of Trappsol® Cyclo™ in Niemann-Pick Disease Type C (NPC)

Trappsol®

Cyclo™ did not achieve statistical significance but demonstrated a directionally favorable treatment effect versus placebo on the

primary endpoint of change in the 4-domain NPC Clinical Severity Scale (4DNPCSS) at Week 96

A prespecified analysis

in patients receiving background miglustat and/or leucine therapy, representing approximately 83% of the study population, showed a statistically

significant effect on the 4DNPCSS

Trappsol®

Cyclo™ demonstrated a well-tolerated safety and tolerability profile consistent with previous studies, with no new safety signals

observed

Based upon its

previously announced pre-NDA meeting, the Company remains on track to submit the NDA in the fourth quarter of 2026, consistent with prior

guidance

Company to host

investor conference call and webcast today, September 30, at 8:30 a.m. ET

NEWARK, NJ — September 30, 2026 — Rafael Holdings,

Inc. (NYSE: RFL) today announced topline efficacy and safety data from its Cyclo Therapeutics, LLC subsidiary’s pivotal Phase 3

TransportNPC™ study (CTD-TCNPC-301) evaluating Trappsol® Cyclo™ (intravenous hydroxypropyl-β-cyclodextrin)

in 94 patients diagnosed with Niemann-Pick Disease Type C (NPC). Trappsol® Cyclo™ was well tolerated and

demonstrated a directionally favorable treatment effect on the primary endpoint, although the result did not achieve statistical significance.

Notably, a prespecified analysis in patients receiving background miglustat and/or leucine therapy demonstrated a statistically significant

and clinically meaningful treatment benefit. Based upon its previously announced pre-NDA meeting with the FDA, the Company remains on

track to submit an NDA for Trappsol® Cyclo™ in the fourth quarter of 2026.

The Phase 3 TransportNPC™ Study

The global, double-blind, placebo-controlled Phase 3 TransportNPC™

study (CTD-TCNPC-301) enrolled 94 patients with NPC, ranging in age from 3 to 70 years. Patients were randomized to receive 2000 mg/kg

of Trappsol® Cyclo™ administered intravenously every two weeks (n=63) or placebo (n=31) for a 96-week period to assess

change in 4DNPCSS.

A prespecified analysis also evaluated change in 4DNPCSS among patients

receiving background miglustat therapy and/or leucine (a food supplement available in some European countries), representing approximately

83% of the study population.

· Primary endpoint: Trappsol® Cyclo™ demonstrated

a directionally favorable treatment effect compared with placebo, indicating slowing of disease progression by 64% on change in 4DNPCSS

at Week 96 (LS mean change from baseline of 0.46 vs 1.28 points; difference −0.81 points [95% CI −2.04, 0.41]; p=0.19), the

result did not achieve statistical significance.

· Prespecified analysis: Among patients receiving background

miglustat and/or leucine therapy (n=78), Trappsol® Cyclo™ indicating slowing of disease progression by 71% compared with placebo

at Week 96 (LS mean change from baseline of 0.46 vs 1.57 points; difference −1.11 points [95% CI −2.19, −0.02]; p=0.046).

Safety and Tolerability

Over the 96-week treatment period, the safety profile of Trappsol®

Cyclo™ was well tolerated. Adverse events were largely consistent with the underlying disease. Treatment-emergent adverse events

(TEAEs) were reported as followed: Trappsol® Cyclo™, 93.8%; placebo, 100.0%. Serious TEAEs occurred more frequently with Trappsol®

Cyclo™,35.9%, than with placebo 16.7%; however, serious TEAEs considered related to treatment were reported at similar rates 3.1%

vs 3.3%. Hearing-related TEAEs were reported by 15.6% of participants receiving Trappsol® Cyclo™ and 13.3% receiving placebo,

and most were mild or moderate in severity. One treatment-related event of bilateral deafness in the Trappsol® Cyclo™ group

was severe. No TEAEs resulted in death, and one participant in the Trappsol® Cyclo™ group discontinued the study early because

of a TEAE.

Separate Overall Survival Analysis

The Company also conducted a separate overall survival analysis comparing

patients with infantile-onset NPC treated with Trappsol® Cyclo™ across its clinical development program

with well-matched external controls from the International Niemann-Pick Disease Registry (INPDR). The external control cohort included

patients with similar baseline characteristics and disease progression. Results from the analysis provide supportive evidence of a treatment-associated

survival benefit with Trappsol® Cyclo™.

Specifically, these data indicated:

· INPDR comparison: Treatment with Trappsol®

Cyclo™ (n=41) was associated with an 85% reduction in the risk of mortality compared with matched INPDR controls (n=93),

HR=0.154; 95% CI: 0.025-0.950; p=0.044.

· Expanded historical control analysis: In a secondary

analysis that expanded the control group to include additional matched historical cohorts from published natural history studies (n=133),

treatment with Trappsol® Cyclo™ (n=41) was associated with a 94% reduction in the risk of mortality,

HR=0.057; 95% CI: 0.011–0.306; p=0.0008.

The consistency and magnitude of the observed survival

associations across both analyses provide additional supportive evidence for the potential clinical benefit of Trappsol®

Cyclo™ in patients with infantile-onset NPC.

“The Phase 3 TransportNPC study is the most

comprehensive trial in NPC conducted to date,” said Dr. Karen Mullen, Chief Medical Officer of Rafael Holdings. “We believe

the totality of the data shared today provide a compelling rationale for the continued advancement of Trappsol Cyclo and its potential

to offer clinically meaningful treatment benefit for those living with this devastating disease. We remain steadfast in our efforts to

advance Trappsol Cyclo and are deeply grateful to the clinical investigators, patients, and caregivers whose courage and commitment were

vital to completing this study.”

“NPC is a devastating, heterogeneous disease, and preserving

function and stabilizing symptoms are critical,” said Dr. Caroline Hastings, Professor of Pediatric Hematology and Neurology at

UCSF Benioff Children’s Hospital Oakland. “As a clinician who has cared for these patients for many years, I am encouraged

by the clinically meaningful TransportNPC results. The study was rigorously conducted, and its findings align with the two prior trials

for Trappsol Cyclo across a decade of development. I am hopeful that these data can move us closer to delivering a much-needed therapeutic

option for families facing profound unmet need.”

NDA On Track for Fourth Quarter 2026

The Company remains on track to submit an NDA for Trappsol®

Cyclo™ to the U.S. Food and Drug Administration in the fourth quarter of 2026 based upon its previously announced pre-NDA

meeting. The topline data, complete dataset and overall survival analysis remain subject to full analysis and FDA regulatory review. The

Company also plans to present the full results at an upcoming medical meeting.

Conference Call Information:

Date: Wednesday, September 30, 2026

Time: 8:30 a.m. ET

Domestic Dial-in Number: 1-877-425-9470

International Dial-in Number: 1-201-389-0878

Conference ID:13763049

Webcast Participant Link: https://viavid.webcasts.com/starthere.jsp?ei=1777956&tp_key=73c1065384

About Rafael Holdings, Inc.

Rafael Holdings, Inc. is a biotechnology company

that develops pharmaceuticals and holds interests in clinical and early-stage companies that develop pharmaceuticals and medical devices.

Our lead candidate is Trappsol® Cyclo™, and we have completed a pivotal Phase 3 study in NPC and are preparing

for a planned NDA submission later this year. Niemann-Pick Disease Type C (“NPC”) is a rare, fatal and progressive genetic disorder.

We also hold interests in other clinical-stage and early-stage pharmaceutical development companies and an orthopedic-focused medical

device company.

Forward Looking Statements

This press release contains forward-looking

statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release

that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements

regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates;

plans regarding the further evaluation of clinical data; and the potential of our pipeline. These statements are neither promises nor

guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance

or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking

statements, including, but not limited to, the risk that the FDA may not accept for filing or approve an NDA for Trappsol® Cyclo™

based on the available data, and those disclosed under the caption “Risk Factors” in our Annual Report on Form 10-K for the

year ended July 31, 2025, our subsequent Quarterly Reports on Form 10-Q, and our other filings with the SEC. These factors could cause

actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking

statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements

at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

Contact:

Barbara Ryan

Barbara.ryan@rafaelholdings.com

(203) 274-2825

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