Trappsol Cyclo Misses Phase 3 Primary Endpoint but Shows Survival Benefit
RFL sits 18% above its 52-week low of $1.12.
Summary
Rafael Holdings' lead drug Trappsol Cyclo missed its Phase 3 primary endpoint but showed a statistically significant benefit in a prespecified subgroup and an 85-94% survival benefit in infantile-onset NPC. NDA submission remains planned for Q4 2026. Shares fell 37.89% to $1.26 on volume of 1.90 million shares, more than 12 times the 100-day average.
Updated with a Benzinga report · What changed
Updates
· Benzinga — The 100-day average volume for Rafael shares was roughly 155,467 shares.
- Trial Design — The pivotal Phase 3 study is named TransportNPC. The 96-week trial evaluated 94 patients aged 3 to 70. Patients received 2000 mg/kg of intravenous Trappsol Cyclo every two weeks.
- Safety — Treatment-emergent adverse events affected 93.8% of Trappsol Cyclo cohort versus 100% in placebo. Serious treatment-related adverse events occurred in 3.1% of drug group vs 3.3% of placebo.
- Trading Volume — The 100-day average volume for Rafael shares was roughly 155,467 shares.
All 10 details
- The pivotal Phase 3 study is named TransportNPC.
- The 100-day average volume for Rafael shares was roughly 155,467 shares.
- The 96-week trial evaluated 94 patients.
- Patients in the trial were aged 3 to 70.
- Patients received 2000 mg/kg of intravenous Trappsol Cyclo every two weeks.
- Treatment-emergent adverse events affected 93.8% of Trappsol Cyclo cohort versus 100% in placebo.
- Serious treatment-related adverse events occurred in 3.1% of drug group vs 3.3% of placebo.
- Hearing-related events affected 15.6% of treated patients and 13.3% of placebo patients.
- One patient discontinued treatment early due to an adverse event.
- No deaths occurred during the trial.
Key Events · Product Development and Regulatory · RFL
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New · · Benzinga
Rafael Shares Plunge on Phase 3 Miss
Rafael shares fell 37.89% to $1.26 on volume of 1.90 million shares, more than 12 times the 100-day average. The 100-day average volume for Rafael shares was roughly 155,467 shares.
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Updated · · Benzinga
Phase 3 Primary Endpoint Missed in TransportNPC Trial
Trappsol Cyclo failed to achieve statistical significance on the primary endpoint of change in 4DNPCSS at Week 96 (p=0.19), showing a 64% slowing of disease progression (LS mean 0.46 vs 1.28 points). The pivotal Phase 3 study is named TransportNPC, a 96-week trial that evaluated 94 patients aged 3 to 70 who received 2000 mg/kg of intravenous Trappsol Cyclo every two weeks.
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Updated · · Benzinga
Safety Profile Consistent with Prior Studies
Trappsol Cyclo was well tolerated with no new safety signals; serious TEAEs occurred in 35.9% vs 16.7% placebo, including one severe treatment-related bilateral deafness event. Treatment-emergent adverse events affected 93.8% of Trappsol Cyclo cohort versus 100% in placebo; serious treatment-related adverse events occurred in 3.1% of drug group vs 3.3% of placebo; hearing-related events affected 15.6% of treated patients and 13.3% of placebo patients; one patient discontinued treatment early due to an adverse event; no deaths occurred during the trial.
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Prespecified Subgroup Shows Significance
In patients receiving background miglustat and/or leucine therapy (n=78, ~83% of study population), Trappsol Cyclo showed a statistically significant 71% slowing of disease progression (p=0.046).
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Survival Benefit in Infantile-Onset NPC
Overall survival analysis showed an 85% reduction in mortality risk vs INPDR controls (HR=0.154, p=0.044) and a 94% reduction vs expanded historical controls (HR=0.057, p=0.0008).
Analysis · RFL · Life Sciences
The pivotal Phase 3 trial for Rafael Holdings' only lead asset failed its primary endpoint (p=0.19), a major setback for a company with a $105M market cap and no other late-stage programs. However, the filing reveals a prespecified subgroup analysis showing statistical significance (p=0.046) in 83% of patients, plus an 85-94% reduction in mortality risk in infantile-onset NPC patients. The company still plans to submit an NDA in Q4 2026, but FDA approval is far from guaranteed given the missed primary endpoint. This is a binary event for the company's future. The market reacted sharply, with shares falling 37.89% to $1.26 on volume of 1.90 million shares, more than 12 times the 100-day average.
How filings like this one have moved
In the 30 days to Sep 30, 2026, 42.1% of the 323 measured filings Wiseek scored 9 moved their stock by 5% or more by the next session's close. The median move was -0.36%. These are measured outcomes after filings of this importance, not a forecast for this one.
Measured one observation per ticker per day, after exclusions. Current figures: Filing Impact Tracker · open dataset
At the time of this filing, RFL was trading at $1.32 on NYSE in the Life Sciences sector, with a market capitalization of approximately $105.6M. The 52-week trading range was $1.12 to $3.96. This filing was assessed with negative market sentiment and an importance score of 9 out of 10.