Topline data and NDA submission in H2 2026 are binary catalysts. Monitor for data readout and FDA acceptance; stock could move significantly on results. The EAP approval is a modest positive for patient access and real-world evidence.
Price Chart
Executive Summary
Rafael Holdings announced the last patient completed the final 96-week visit in its pivotal Phase 3 TransportNPC study for Trappsol Cyclo in Niemann-Pick Disease Type C. Topline data and an NDA submission are expected in H2 2026, and the FDA has allowed the US Expanded Access Program to proceed. This is a significant pipeline milestone for a pre-revenue biotech, but no efficacy or safety results are available yet.
Key Facts
- Last patient completed final 96-week visit in pivotal Phase 3 TransportNPC study (94 patients, 27 sites, 13 countries).
- Topline data expected in H2 2026; pre-NDA meeting completed; NDA submission planned for H2 2026.
- FDA acknowledged US Expanded Access Program can proceed.
- Single-arm pediatric sub-study (ages 0-3) data showed 80% of patients had CGI scores improved or stable, with no related serious adverse events.
- Company is pre-revenue with a $100M market cap; transitioning toward commercial-stage biotech.
Financial Impact
No financial data disclosed; pre-revenue company. NPC is a high-unmet-need rare disease market; potential value depends on topline results and approval.
Risk Factors
- Phase 3 topline data may fail to meet endpoints; no efficacy data released yet.
- Regulatory approval is not guaranteed; NDA review timeline and outcome uncertain.
- Pre-revenue company with limited cash runway; potential dilution risk if financing needed.
Market Snapshot
Investment Themes
Documents Analyzed
This report is based on 1 press release from GlobeNewswire.
| Document | Accession Number |
|---|---|
| PRESS-RELEASE Data (Synthetic) | press-3309598 |
Track record builds as more directional reports settle.
Filters
| Type | Now | ||||
|---|---|---|---|---|---|
Sep 30, 2026 27d ago | 8-K | $1.32 | awaiting T+20 | — | $1.18 (−10.61%) |
Sep 30, 2026 27d ago | Press Release | $1.32 | awaiting T+20 | — | $1.18 (−10.61%) |
Aug 18, 2026 10w ago | Institutional Cluster | $1.86 $2.10 | ▲ +12.90% | ▲ +13.89% | $1.18 (−36.56%) |
Jun 11, 2026 19w ago | 8-K | $2.12 $2.27 | ▲ +7.08% | ▲ +5.18% | $1.18 (−44.34%) |
Jun 10, 2026 19w ago | 8-K | $1.78 $2.27 | ▲ +27.53% | ▲ +23.91% | $1.18 (−33.71%) |
Jun 10, 2026 19w ago | Press Release | $1.78 $2.27 | ▲ +27.53% | ▲ +23.91% | $1.18 (−33.71%) |
Apr 22, 2026 26w ago | 8-K | $1.30 $1.35 | ▲ +3.85% | ▲ +0.68% | $1.18 (−9.23%) |
Mar 16, 2026 32w ago | Press Release | $1.32 $1.22 | ▼ −7.58% | ▼ −11.88% | $1.18 (−10.61%) |
US Market Status
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