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Rafael Holdings stock tumbles 25% as drug trial misses goal

Wed, 30 Sep 2026 09:21:40 -0400

Investing.com -- Rafael Holdings Inc Class B (NYSE:RFL) shares fell 25% in premarket trading Wednesday after the company's Phase 3 trial of Trappsol Cyclo for Niemann-Pick Disease Type C failed to meet its primary endpoint.

The TransportNPC study, which enrolled 94 patients, showed Trappsol Cyclo did not achieve statistical significance on the primary endpoint of change in the 4-domain NPC Clinical Severity Scale at Week 96. The drug demonstrated a directionally favorable treatment effect versus placebo, slowing disease progression by 64%, but the result missed statistical significance with a p-value of 0.19.

However, a prespecified analysis in patients

Rafael Holdings stock tumbles 25% as drug trial misses goal

Wed, 30 Sep 2026 09:21:40 -0400

However, a prespecified analysis in patients

Form 8-K Rafael Holdings, Inc. For: Sep 30

Wed, 30 Sep 2026 07:23:00 -0400

si-local-file:///var/storage/secattach/20260930/27125235.html

Rafael Holdings Phase 3 NPC drug trial misses primary endpoint

Wed, 30 Sep 2026 07:00:17 -0400

Rafael Holdings, Inc. (NYSE: RFL) announced topline results from its pivotal Phase 3 TransportNPC™ study evaluating Trappsol® Cyclo™ in 94 patients with Niemann-Pick Disease Type C (NPC), reporting that the drug did not achieve statistical significance on its primary endpoint.

The global, double-blind, placebo-controlled trial enrolled patients aged 3 to 70, randomized to receive 2,000 mg/kg of Trappsol® Cyclo™ intravenously every two weeks (n=63) or placebo (n=31) over 96 weeks. The primary endpoint measured change in the 4-domain NPC Clinical Severity Scale (4DNPCSS). The treatment group showed a mean change from baseline of 0.46 points versus 1.28 points in the placebo

Rafael Holdings Announces Topline Results from the Pivotal Phase 3 TransportNPC™ Study of Trappsol® Cyclo™ in Niemann-Pick Disease Type C (NPC)

Wed, 30 Sep 2026 07:00:00 -0400

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

Rafael Holdings Announces Topline Results from the Pivotal Phase 3 TransportNPC™ Study of Trappsol® Cyclo™ in Niemann-Pick Disease Type C (NPC)

Wed, 30 Sep 2026 07:00:00 -0400

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

Form 4 Rafael Holdings, Inc. For: Sep 21 Filed by: Polinsky David

Wed, 23 Sep 2026 14:37:00 -0400
si-local-file:///var/storage/secattach/20260923/27096339.html

Ernexa Therapeutics Strengthens Board with Veteran Biotech Executive David Polinsky as Company Approaches Clinical Stage

Mon, 10 Aug 2026 09:01:00 -0400

Seasoned biotechnology founder, public company CFO and corporate strategist brings more than 25 years of leadership in oncology, capital markets, M&A and corporate governance

Appointment further strengthens leadership team at a pivotal inflection point as Company positions ERNA-101 for planned IND submission in Q3 2026 and Phase 1 clinical study initiation in the Q4 2026

CAMBRIDGE, Mass., Aug. 10, 2026 (GLOBE NEWSWIRE) -- Ernexa Therapeutics (Nasdaq: ERNA), an industry innovator developing novel cell therapies for the treatment of advanced cancer and autoimmune disease, today announced the appointment of David Polinsky

Ernexa Therapeutics Strengthens Board with Veteran Biotech Executive David Polinsky as Company Approaches Clinical Stage

Mon, 10 Aug 2026 09:01:00 -0400

Seasoned biotechnology founder, public company CFO and corporate strategist brings more than 25 years of leadership in oncology, capital markets, M&A and corporate governance

Appointment further strengthens leadership team at a pivotal inflection point as Company positions ERNA-101 for planned IND submission in Q3 2026 and Phase 1 clinical study initiation in the Q4 2026

CAMBRIDGE, Mass., Aug. 10, 2026 (GLOBE NEWSWIRE) -- Ernexa Therapeutics (Nasdaq: ERNA), an industry innovator developing novel cell therapies for the treatment of advanced cancer and autoimmune disease, today announced the appointment of David Polinsky

Ernexa Therapeutics Strengthens Board with Veteran Biotech Executive David Polinsky as Company Approaches Clinical Stage

Mon, 10 Aug 2026 09:01:00 -0400

Seasoned biotechnology founder, public company CFO and corporate strategist brings more than 25 years of leadership in oncology, capital markets, M&A and corporate governance

Appointment further strengthens leadership team at a pivotal inflection point as Company positions ERNA-101 for planned IND submission in Q3 2026 and Phase 1 clinical study initiation in the Q4 2026

CAMBRIDGE, Mass., Aug. 10, 2026 (GLOBE NEWSWIRE) -- Ernexa Therapeutics (Nasdaq: ERNA), an industry innovator developing novel cell therapies for the treatment of advanced cancer and autoimmune disease, today announced the appointment of David Polinsky

Ernexa Therapeutics Strengthens Board with Veteran Biotech Executive David Polinsky as Company Approaches Clinical Stage

Mon, 10 Aug 2026 09:01:00 -0400

Seasoned biotechnology founder, public company CFO and corporate strategist brings more than 25 years of leadership in oncology, capital markets, M&A and corporate governance

Appointment further strengthens leadership team at a pivotal inflection point as Company positions ERNA-101 for planned IND submission in Q3 2026 and Phase 1 clinical study initiation in the Q4 2026

CAMBRIDGE, Mass., Aug. 10, 2026 (GLOBE NEWSWIRE) -- Ernexa Therapeutics (Nasdaq: ERNA), an industry innovator developing novel cell therapies for the treatment of advanced cancer and autoimmune disease, today announced the appointment of David Polinsky

Ernexa Therapeutics Strengthens Board with Veteran Biotech Executive David Polinsky as Company Approaches Clinical Stage

Mon, 10 Aug 2026 09:01:00 -0400

Seasoned biotechnology founder, public company CFO and corporate strategist brings more than 25 years of leadership in oncology, capital markets, M&A and corporate governance

Appointment further strengthens leadership team at a pivotal inflection point as Company positions ERNA-101 for planned IND submission in Q3 2026 and Phase 1 clinical study initiation in the Q4 2026

CAMBRIDGE, Mass., Aug. 10, 2026 (GLOBE NEWSWIRE) -- Ernexa Therapeutics (Nasdaq: ERNA), an industry innovator developing novel cell therapies for the treatment of advanced cancer and autoimmune disease, today announced the appointment of David Polinsky

Ernexa Therapeutics Strengthens Board with Veteran Biotech Executive David Polinsky as Company Approaches Clinical Stage

Mon, 10 Aug 2026 09:01:00 -0400

Seasoned biotechnology founder, public company CFO and corporate strategist brings more than 25 years of leadership in oncology, capital markets, M&A and corporate governance

Appointment further strengthens leadership team at a pivotal inflection point as Company positions ERNA-101 for planned IND submission in Q3 2026 and Phase 1 clinical study initiation in the Q4 2026

CAMBRIDGE, Mass., Aug. 10, 2026 (GLOBE NEWSWIRE) -- Ernexa Therapeutics (Nasdaq: ERNA), an industry innovator developing novel cell therapies for the treatment of advanced cancer and autoimmune disease, today announced the appointment of David Polinsky

Form 4 Rafael Holdings, Inc. For: Jul 29 Filed by: JONAS HOWARD S

Fri, 31 Jul 2026 16:55:00 -0400
si-local-file:///var/storage/secattach/20260731/26851469.html

Form 4 Rafael Holdings, Inc. For: Jul 29 Filed by: JONAS HOWARD S

Fri, 31 Jul 2026 16:55:00 -0400
si-local-file:///var/storage/secattach/20260731/26851469.html

Form 8-K Rafael Holdings, Inc. For: Jul 08

Wed, 08 Jul 2026 16:46:00 -0400
si-local-file:///var/storage/secattach/20260708/26748116.html

Form 8-K Rafael Holdings, Inc. For: Jul 08

Wed, 08 Jul 2026 16:46:00 -0400
si-local-file:///var/storage/secattach/20260708/26748116.html

Form 4 Rafael Holdings, Inc. For: Jun 21 Filed by: Polinsky David

Tue, 23 Jun 2026 15:33:00 -0400
si-local-file:///var/storage/secattach/20260623/26679213.html

Form 4 Rafael Holdings, Inc. For: Jun 21 Filed by: Polinsky David

Tue, 23 Jun 2026 15:33:00 -0400
si-local-file:///var/storage/secattach/20260623/26679213.html

Form 4 Rafael Holdings, Inc. For: Jun 13 Filed by: JONAS HOWARD S

Tue, 16 Jun 2026 15:55:00 -0400
si-local-file:///var/storage/secattach/20260616/26653867.html

Form 4 Rafael Holdings, Inc. For: Jun 13 Filed by: JONAS HOWARD S

Tue, 16 Jun 2026 15:55:00 -0400
si-local-file:///var/storage/secattach/20260616/26653867.html

Form 10-Q Rafael Holdings, Inc. For: Apr 30

Thu, 11 Jun 2026 08:18:00 -0400
si-local-file:///var/storage/secattach/20260611/26632997.html

Form 10-Q Rafael Holdings, Inc. For: Apr 30

Thu, 11 Jun 2026 08:18:00 -0400
si-local-file:///var/storage/secattach/20260611/26632997.html

Form 8-K Rafael Holdings, Inc. For: Jun 11

Thu, 11 Jun 2026 07:55:00 -0400
si-local-file:///var/storage/secattach/20260611/26632693.html

Form 8-K Rafael Holdings, Inc. For: Jun 11

Thu, 11 Jun 2026 07:55:00 -0400
si-local-file:///var/storage/secattach/20260611/26632693.html