The stock will likely trade on sentiment around the upcoming topline data readout in H2 2026. Monitor for any data leaks or analyst previews ahead of the announcement. The FDA's EAP acknowledgment is a modest positive for patient access but does not change the binary risk profile.
Price Chart
Executive Summary
Rafael Holdings announced completion of the last patient's final 96-week visit in the pivotal Phase 3 TransportNPC study of Trappsol Cyclo for Niemann-Pick Disease Type C. Topline data and an NDA submission are expected in H2 2026, and the FDA has acknowledged the company's Expanded Access Program can proceed. This is a key clinical milestone for a pre-revenue biotech with a $100M market cap, but no efficacy data has been released yet.
Key Facts
- Last patient completed final 96-week study visit in pivotal Phase 3 TransportNPC study (94 patients, 27 sites, 13 countries).
- Topline data from main cohort expected in second half of calendar 2026.
- Pre-NDA meeting with FDA completed; NDA submission expected in H2 2026.
- FDA acknowledged that the US Expanded Access Program (EAP) can proceed.
- 10 patients enrolled in a single-arm pediatric sub-study (newborns to 3 years).
- 80% of pediatric sub-study patients showed CGI score improvements or stability with no related serious adverse events (data from WORLDSymposium 2026).
- Company is pre-revenue with a $100M market cap; no financial data reported in this filing.
Financial Impact
No financial figures reported in the filing. The event is a clinical milestone with binary topline data risk in H2 2026.
Risk Factors
- Topline data may fail to meet primary endpoints, causing significant downside.
- Regulatory path remains uncertain until NDA submission and FDA review.
- Pre-revenue biotech with no approved products; cash runway and financing risk not disclosed.
Market Snapshot
Investment Themes
Documents Analyzed
This report is based on 5 SEC documents filed with EDGAR.
| Document | Accession Number |
|---|---|
| 8-K Filing (Primary) | 0001213900-26-067083 |
| Document: ea029415101ex99-1.htm | 0001213900-26-067083 |
| Document: 0001213900-26-067083-index-headers.html | 0001213900-26-067083 |
| Document: 0001213900-26-067083-index.html | 0001213900-26-067083 |
| Document: 0001213900-26-067083.txt | 0001213900-26-067083 |
Track record builds as more directional reports settle.
Filters
| Type | Now | ||||
|---|---|---|---|---|---|
Sep 30, 2026 27d ago | 8-K | $1.32 | awaiting T+20 | — | $1.22 (−7.58%) |
Sep 30, 2026 27d ago | Press Release | $1.32 | awaiting T+20 | — | $1.22 (−7.58%) |
Aug 18, 2026 10w ago | Institutional Cluster | $1.86 $2.10 | ▲ +12.90% | ▲ +13.89% | $1.22 (−34.41%) |
Jun 11, 2026 19w ago | 8-K | $2.12 $2.27 | ▲ +7.08% | ▲ +5.18% | $1.22 (−42.45%) |
Jun 10, 2026 19w ago | 8-K | $1.78 $2.27 | ▲ +27.53% | ▲ +23.91% | $1.22 (−31.46%) |
Jun 10, 2026 19w ago | Press Release | $1.78 $2.27 | ▲ +27.53% | ▲ +23.91% | $1.22 (−31.46%) |
Apr 22, 2026 26w ago | 8-K | $1.30 $1.35 | ▲ +3.85% | ▲ +0.68% | $1.22 (−6.15%) |
Mar 16, 2026 32w ago | Press Release | $1.32 $1.22 | ▼ −7.58% | ▼ −11.88% | $1.22 (−7.58%) |
US Market Status
Subscribe to SecBot
Get Real-Time SEC Filing Intelligence
Comprehensive SEC filing analysis delivered the moment filings hit EDGAR. Sentiment scoring, impact analysis, and actionable insights for every material event.
Create a free account Live analysis, Telegram alerts, watchlists and the MCP server for subscribers