RFL Rafael Holdings, Inc.
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.
Actionable Insight
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.
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
Documents Analyzed
This report is based on 1 press release from GlobeNewswire.
| Document | Accession Number |
|---|---|
| PRESS-RELEASE Data (Synthetic) | press-3309598 |
Filters
| Type | Now | ||||
|---|---|---|---|---|---|
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Jun 11, 2026
3d ago
|
8-K
| $1.82 awaiting T+20 | awaiting T+20 | — | $2.24 (+23.42%) |
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Jun 10, 2026
4d ago
|
8-K
| $1.90 awaiting T+20 | awaiting T+20 | — | $2.24 (+17.89%) |
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Jun 10, 2026
4d ago
|
Press Release
| $1.90 awaiting T+20 | awaiting T+20 | — | $2.24 (+17.89%) |
|
Apr 22, 2026
7w ago
|
8-K
| $1.30 $1.35 | ▲ +3.85% | ▲ +0.68% | $2.24 (+72.31%) |
|
Mar 16, 2026
12w ago
|
Press Release
| $1.32 $1.22 | ▼ −7.58% | ▼ −11.88% | $2.24 (+69.70%) |
US Market Status
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