The NDA submission is the primary catalyst. FDA acceptance for filing and priority review status will be key near-term milestones. The mixed Phase 3 data (missed primary endpoint but positive subgroup) creates regulatory uncertainty, monitor FDA communication and any advisory committee briefing documents.
Price Chart
Executive Summary
Rafael Holdings reported Q4 FY2026 net loss of $9.2M ($0.18/share) vs $12.1M ($0.28/share) a year ago, with cash of $22.1M. The key event is the NDA submission for remibetadex in Niemann-Pick Type C, supported by a Phase 3 study that missed its primary endpoint (p=0.19) but showed a 71% slowing in a prespecified subgroup (p=0.046).
Key Financial Metrics
Key Facts
- NDA submitted to FDA for remibetadex (Trappsol Cyclo) in Niemann-Pick Disease Type C on October 28, 2026
- Phase 3 TransportNPC study did not meet primary endpoint in overall population (p=0.19)
- Prespecified subgroup analysis showed 71% slowing of disease progression (p=0.046) in patients on background miglustat/leucine
- Q4 FY2026 net loss attributable to RFL: $9.2M ($0.18/share) vs $12.1M ($0.28/share) year ago
- Cash and equivalents: $22.1M as of July 31, 2026
- Full year FY2026 net loss: $29.6M ($0.58/share) vs $30.5M ($1.04/share) year ago
- R&D expenses flat at $7.5M in Q4; G&A decreased to $2.4M from $5.5M
Financial Impact
Cash burn of ~$22M/year; NDA outcome binary, approval could unlock rare pediatric disease priority review voucher and potential commercial revenue; denial would eliminate near-term value driver.
Risk Factors
- Phase 3 study missed primary endpoint in overall population, raising regulatory risk
- Subgroup analysis may not be sufficient for FDA approval
- Cash runway limited to ~1 year at current burn rate; need for additional financing
- Going concern risk noted in forward-looking statements
- No revenue from remibetadex; company pre-commercial
Market Snapshot
Investment Themes
Documents Analyzed
This report is based on 1 press release from GlobeNewswire.
| Document | Accession Number |
|---|---|
| PRESS-RELEASE Data (Synthetic) | press-3391179 |
Track record builds as more directional reports settle.
Filters
| Type | Now | ||||
|---|---|---|---|---|---|
Oct 29, 2026 3d ago | 8-K | No price | awaiting T+20 | awaiting T+20 | No price |
Oct 29, 2026 3d ago | Press Release | $1.12 | awaiting T+20 | awaiting T+20 | $1.08 (−3.57%) |
Sep 30, 2026 4w ago | 8-K | $1.32 $1.24 | ▼ −6.06% | ▼ −7.33% | $1.08 (−18.18%) |
Sep 30, 2026 4w ago | Press Release | $1.32 $1.24 | ▼ −6.06% | ▼ −6.98% | $1.08 (−18.18%) |
Aug 18, 2026 10w ago | Institutional Cluster | $1.86 $2.10 | ▲ +12.90% | ▲ +13.89% | $1.08 (−41.94%) |
Jun 11, 2026 20w ago | 8-K | $2.12 $2.27 | ▲ +7.08% | ▲ +5.18% | $1.08 (−49.06%) |
Jun 10, 2026 20w ago | 8-K | $1.78 $2.27 | ▲ +27.53% | ▲ +23.91% | $1.08 (−39.33%) |
Jun 10, 2026 20w ago | Press Release | $1.78 $2.27 | ▲ +27.53% | ▲ +23.91% | $1.08 (−39.33%) |
Apr 22, 2026 27w ago | 8-K | $1.30 $1.35 | ▲ +3.85% | ▲ +0.68% | $1.08 (−16.92%) |
Mar 16, 2026 32w ago | Press Release | $1.32 $1.22 | ▼ −7.58% | ▼ −11.88% | $1.08 (−18.18%) |
US Market Status
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