Rafael Holdings, Inc. reports developments as a biotechnology company focused on pharmaceutical development and interests in clinical-stage and early-stage pharmaceutical and medical device companies. Its lead program is Trappsol® Cyclo™, a hydroxypropyl-beta-cyclodextrin product candidate evaluated for Niemann-Pick Disease Type C1, with additional cyclodextrin-related work in neurodegenerative disease.
Recurring company news covers clinical development of Cyclo Therapeutics programs, scientific presentations, patent and licensing activity, quarterly and annual financial results, and governance changes. Updates also address the company’s consolidated expenses following the Cyclo Therapeutics acquisition and its portfolio interests in healthcare and medical-device development.
Rafael Holdings (NYSE: RFL) reported a fiscal 2026 net loss attributable to the company of $29.635 million, narrower than a year earlier. Loss per share was $0.58 versus $1.04; the prior-year loss was $30.520 million. Fourth-quarter loss narrowed to $9.176 million from $12.094 million. Cash fell to $22.074 million from $52.769 million at July 31, 2025.
Cyclo Therapeutics submitted an FDA application for remibetadex in Niemann-Pick Disease Type C on October 28, 2026. Phase 3 missed its overall primary endpoint (p=0.19), but a prespecified subgroup receiving background miglustat and/or leucine showed 71% slower disease progression (p=0.046). Fiscal 2026 research and development expenses rose to $24.333 million from $12.823 million, while general and administrative expenses fell to $9.704 million from $13.781 million. A $4.422 million gain on settlements of payables and convertible notes also supported results.
Rafael Holdings (RFL) announced that subsidiary Cyclo Therapeutics submitted an FDA application seeking approval of remibetadex for Niemann-Pick Disease Type C. The proposed indication covers neurological and internal-organ manifestations in adults and children from birth.
The Phase 3 TransportNPC study missed its primary endpoint, with directionally favorable 64% relative slowing of disease progression versus placebo (p=0.19). A prespecified analysis of patients receiving background therapy showed 71% relative slowing (p=0.046). An external-control analysis associated remibetadex with an 85% reduction in mortality risk in infantile-onset disease versus matched registry controls (p=0.044). Serious adverse events occurred in 35.9% versus 16.7% with placebo; no new safety signals were observed.
FDA filing acceptance and Priority Review remain pending. Approval would make remibetadex eligible for a Rare Pediatric Disease Priority Review Voucher.
Rafael Holdings (RFL) reported Phase 3 TransportNPC results for Trappsol Cyclo in Niemann-Pick Disease Type C, missing statistical significance.
The 94-patient trial measured change in a four-domain clinical severity scale at Week 96. The primary endpoint showed directionally favorable 64% slowing of disease progression versus placebo (p=0.19). A prespecified analysis of 78 patients receiving background miglustat and/or leucine showed 71% slowing (p=0.046). Serious treatment-emergent adverse events occurred in 35.9% with treatment versus 16.7% with placebo; one treatment-related bilateral deafness event was severe.
Separate infantile-onset NPC analyses found treatment-associated mortality risk reductions versus external controls. Rafael expects to submit a new drug application in fourth-quarter 2026. The data remain subject to full analysis and FDA review.
Ernexa Therapeutics (Nasdaq: ERNA) appointed veteran biotech executive and corporate attorney David Polinsky to its Board of Directors as the company prepares to enter the clinic with its lead cell therapy program ERNA-101.
According to Ernexa, the company plans to submit an Investigational New Drug (IND) application for ERNA-101 in the third quarter of 2026 and to initiate its first Phase 1 clinical study in the fourth quarter of 2026. Polinsky brings more than 25 years of experience in oncology, capital markets, M&A and corporate governance, and currently serves as Chief Financial Officer of Rafael Holdings (NYSE: RFL). His background includes leading finance, legal and strategy functions, helping establish Rafael Holdings as an independent NYSE-listed company after its 2018 spin-off, and helping lead a 2025 merger with Cyclo Therapeutics and an approximately $25 million rights offering. Ernexa’s leadership highlights this appointment as strengthening governance and strategic capabilities ahead of its transition to a clinical-stage company.
Rafael Holdings (NYSE:RFL) reported third quarter fiscal 2026 results and key clinical progress. The company completed last patient last visit in its pivotal Phase 3 TransportNPC trial and, following a pre-NDA FDA meeting, expects to submit an NDA in second-half 2026.
For Q3 FY 2026, Rafael reported cash of $30.5 million and a net loss attributable to Rafael of $4.2 million ($0.08 per share), versus $4.8 million ($0.19 per share) a year earlier. Nine-month FY 2026 net loss was $20.5 million ($0.40 per share) versus $18.4 million ($0.73 per share).
Q3 R&D expenses rose to $4.9 million from $3.0 million, and nine-month R&D to $16.9 million from $5.3 million, largely due to Cyclo Therapeutics consolidation. Q3 G&A fell to $2.1 million from $3.2 million, and nine-month G&A to $7.3 million from $8.3 million.
Rafael Holdings (NYSE:RFL) completed last patient last visit in the pivotal Phase 3 TransportNPC™ study of Trappsol® Cyclo™ for Niemann-Pick type C. The 96-week trial included 94 patients across 27 sites in 13 countries, plus a 10-patient pediatric sub-study.
Topline results and an NDA submission to the FDA are expected in the second half of 2026, following a completed pre-NDA meeting. The FDA has also acknowledged that the US Expanded Access Program can proceed.
Rafael Holdings (NYSE: RFL) secured an exclusive U.S. license from MIT for U.S. Patent No.12285440 covering the use of cyclodextrins as an API to treat ApoE4‑positive Alzheimer’s disease. The agreement strengthens Rafael’s IP around Trappsol® Cyclo™ and targets a patient subgroup estimated at 50–70% of AD cases. Preclinical data show improved CNS cholesterol transport, reduced tau pathology, and memory benefits in mice. Rafael also notes Transport NPC Phase 3 results due in calendar Q3 2026, highlighting near‑term clinical milestones.
Rafael Holdings (NYSE: RFL) reported Q2 fiscal 2026 results for the period ended January 31, 2026. Cash and cash equivalents were $37.8 million. Q2 net loss attributable to Rafael Holdings was $6.4 million ($0.13/share); six‑month net loss was $16.2 million ($0.32/share). R&D increased notably after the March 2025 acquisition of Cyclo Therapeutic. The Data Monitoring Committee recommended continuing the pivotal 96‑week Phase 3 TransportNPC study of Trappsol Cyclo; preliminary topline results are expected in Q3 calendar 2026.
Rafael Holdings (NYSE: RFL) reported first quarter fiscal 2026 results for the period ended October 31, 2025. The company held $45.5 million in cash and cash equivalents as of that date. Rafael recorded a net loss of $9.8 million, or $0.19 per share, versus a net loss of $9.0 million, or $0.37 per share, in the prior-year quarter. Research and development expenses rose to $7.5 million from $1.3 million year‑over‑year, and general and administrative expenses were $2.8 million versus $2.5 million.
The company completed the acquisition of Cyclo in March 2025 and said the Data Monitoring Committee recommended continuing the pivotal Phase 3 TransportNPC study of Trappsol Cyclo after a 48‑week safety and efficacy review.
Rafael Holdings (NYSE: RFL) reported fourth-quarter and full-year fiscal 2025 results for the period ended July 31, 2025. Key facts: cash and cash equivalents $52.8M, completion of a $25.0M rights offering (closed June 4, 2025) with a $21.0M backstop by the Jonas family, and consolidation of Cyclo following its March 2025 acquisition. The Data Monitoring Committee recommended continuing Cyclo's pivotal Phase 3 TransportNPC™ study of Trappsol® Cyclo™ after a 48-week review. Fiscal Q4 net loss attributable to Rafael Holdings was $12.1M ($0.28/sh); full-year net loss was $30.5M ($1.04/sh). R&D and G&A expenses increased year over year due to Cyclo and prior consolidations.