The FDA's filing decision and potential Priority Review grant within 60 days is the next catalyst. The Phase 3 miss on the primary endpoint creates approval risk, but the subgroup and survival data provide a path. Monitor cash runway and any financing needs ahead of a potential PDUFA date.
Price Chart
Executive Summary
Rafael Holdings reported fiscal Q4 2026 results with a net loss of $9.2M ($0.18/share), improved from a $12.1M loss in the prior-year period, while cash fell to $22.1M from $30.5M three months earlier. The key event is the NDA submission for remibetadex (Trappsol Cyclo) for Niemann-Pick Disease Type C, supported by a Phase 3 study that missed its primary endpoint (p=0.19) but showed benefit in a prespecified subgroup (p=0.046) and an overall survival analysis (85% mortality risk reduction, p=0.044). The FDA will decide on filing and priority review within 60 days.
Key Financial Metrics
Key Facts
- NDA submitted to FDA on October 28, 2026 for remibetadex (Trappsol Cyclo) for Niemann-Pick Disease Type C
- Phase 3 TransportNPC trial missed 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
- Overall survival analysis showed 85% reduction in mortality risk vs matched registry controls (p=0.044)
- Serious adverse events occurred more frequently with remibetadex (35.9%) vs placebo (16.7%)
- Cash and cash equivalents of $22.1M as of July 31, 2026, down from $30.5M as of April 30, 2026
- Net loss attributable to Rafael Holdings of $9.2M for Q4 FY2026 vs $12.1M in prior-year period
- Full year FY2026 net loss of $29.6M vs $30.5M in FY2025
- FDA will determine filing acceptance and priority review within 60 days
- Remibetadex has Orphan Drug, Fast Track and Rare Pediatric Disease designations; approval would make it eligible for a Priority Review Voucher
Financial Impact
Cash burn rate of approximately $8.5M per quarter; $22.1M cash provides roughly 2.5 quarters of runway at current spend levels. NDA submission is a binary regulatory catalyst with no stated deal value.
Risk Factors
- Phase 3 trial did not meet primary endpoint in overall population, creating significant approval risk
- Cash burn continues with only $22.1M on hand, likely requiring additional financing before potential commercialization
- Serious adverse events were more than double in the treatment arm vs placebo (35.9% vs 16.7%)
- FDA may not accept the NDA for filing or may require additional trials
- No consensus estimates available; pre-revenue biotech with binary regulatory outcome
Market Snapshot
Investment Themes
Documents Analyzed
This report is based on 7 SEC documents filed with EDGAR.
| Document | Accession Number |
|---|---|
| 8-K Filing (Primary) | 0001213900-26-113564 |
| Document: ea0309961-8k_rafael.htm | 0001213900-26-113564 |
| Document: ea030996101ex99-2.htm | 0001213900-26-113564 |
| Document: 0001213900-26-113564-index-headers.html | 0001213900-26-113564 |
| Document: 0001213900-26-113564-index.html | 0001213900-26-113564 |
| Document: 0001213900-26-113564.txt | 0001213900-26-113564 |
| 8-K Data (Synthetic) | 0001213900-26-113564 |
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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