Rafael Holdings, Inc.
RFL evidence brief
What we can confirm
- Market Cap:
- $118.6M
What it means
What to watch
Prepared research question
What matters most for RFL—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · RFL
Rafael Holdings, Inc. (RFL) is a biopharmaceutical company focused on developing Trappsol Cyclo for Niemann-Pick Disease Type C, with its pivotal Phase III study missing the primary endpoint but showing a directionally favorable treatment effect.
Key risk: Missed Phase III endpoint
Market Cap
$118.6M
Cash Runway
—
Lead Asset
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Next Catalyst
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Financial data as of Apr 30, 2026.
Rafael Holdings, Inc. (RFL) is a biopharmaceutical company focused on developing Trappsol Cyclo for Niemann-Pick Disease Type C, with its pivotal Phase III study missing the primary endpoint but showing a directionally favorable treatment effect.
- Cash & equivalents of $30.5M as of 2026-04-30 [sec].
- Total revenue of $0.2M and net income of -$4.2M as of 2026-04-30 [sec].
- Shareholders' equity of $75.4M as of 2026-04-30 [sec].
- Phase III TransportNPC study of Trappsol Cyclo did not achieve statistical significance on the primary endpoint but showed a directionally favorable treatment effect versus placebo at Week 96 [news · 2026-09-30].
- Company targets FDA NDA submission in 2026 despite missed primary endpoint [news · 2026-10-04].
What works
- Trappsol Cyclo demonstrated a well-tolerated safety and tolerability profile in the Phase III study [news · 2026-09-30].
- A prespecified analysis in patients receiving background miglustat and/or leucine therapy (≈83% of the study population) showed a statistically significant effect on the primary endpoint [news · 2026-09-30].
- The company has a cash position of $30.5M as of 2026-04-30, providing a runway for continued operations [sec].
What to weigh
- The Phase III TransportNPC study missed its primary endpoint of statistical significance on the 4DNPCSS at Week 96 [news · 2026-09-30].
- The company reported a net loss of -$4.2M for the period ending 2026-04-30, with minimal revenue of $0.2M [sec].
- The FDA NDA submission timeline for 2026 is uncertain given the missed primary endpoint [news · 2026-10-04].
From the filings
Quoted directly from source documents.
Phase III study missed primary endpoint but showed directionally favorable effect
“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”
News · September 30, 2026
Prespecified analysis showed statistically significant effect in majority of patients
“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”
News · September 30, 2026
Company targets FDA NDA submission in 2026
“The company said it remains on track to submit a New Drug Application to the U.S. Food and Drug Administration in”
News · October 4, 2026
Risks & what to watch (3)›
Key risks
- Missed Phase III endpoint — The pivotal Phase III study did not achieve statistical significance on the primary endpoint, complicating FDA approval [news · 2026-09-30].
- Minimal revenue, net loss — Revenue of $0.2M and net loss of -$4.2M as of 2026-04-30 indicate heavy reliance on cash reserves [sec].
- Uncertain NDA timeline — FDA submission target for 2026 is uncertain given the missed primary endpoint [news · 2026-10-04].
What to watch
- FDA NDA submission decision and timing for Trappsol Cyclo [news · 2026-10-04].
- Further regulatory interactions and potential need for additional studies following the missed Phase III endpoint [news · 2026-09-30].
- Cash runway and burn rate given net losses and minimal revenue [sec].
- Any updates on the prespecified subgroup analysis and its impact on regulatory strategy [news · 2026-09-30].
Sources: Catalysts · Competitor trials · News · Generated October 9, 2026 · How we verify sources →
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Evidence & data
Financials
Short Interest
as of Apr 29, 2026- % of float
- 0.1%
- Days to cover
- 0.6
- Shares short
- 32,087
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
RFL short interestGovernance
Frequently asked questions
What is the AI investment thesis for Rafael Holdings, Inc.?
Rafael Holdings, Inc. (RFL) is a biopharmaceutical company focused on developing Trappsol Cyclo for Niemann-Pick Disease Type C, with its pivotal Phase III study missing the primary endpoint but showing a directionally favorable treatment effect.
Who is the CEO of Rafael Holdings, Inc.?
Mr. Howard S. Jonas is the chief executive officer of Rafael Holdings, Inc. (RFL).
Where is Rafael Holdings, Inc. headquartered?
Rafael Holdings, Inc. (RFL) is headquartered in Newark, NJ, United States.
What therapeutic areas does Rafael Holdings, Inc. focus on?
Rafael Holdings, Inc. develops therapies across Oncology, Rare Disease.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.