Rafael Holdings Completes Phase 3 NPC Trial, Reports Q3 Loss of $4.2 Million
RFL•Rafael Holdings completed the last patient visit in its pivotal Phase 3 TransportNPC trial for Niemann-Pick Disease and plans to file its NDA in the second half of 2026. The company reported $30.5 million in cash and equivalents and a Q3 net loss of $4.2 million, or $0.08 per share.
1. Phase 3 Trial Completion and NDA Timeline
Rafael Holdings announced completion of the last patient visit in its pivotal Phase 3 TransportNPC trial for Niemann-Pick Disease Type C1 and confirmed a clear, expedited path to submit its New Drug Application in the second half of calendar 2026.
2. Q3 Fiscal 2026 Financial Performance
For the quarter ended April 30, 2026, Rafael Holdings held $30.5 million in cash and equivalents, recorded a net loss of $4.2 million ($0.08 per share), incurred $4.9 million in research and development expenses, and $2.1 million in general and administrative costs.
3. Nine-Month Results and Cyclo Acquisition Impact
In the first nine months of fiscal 2026, the company posted a $20.5 million net loss ($0.40 per share) versus $18.4 million a year ago, driven by consolidation of Cyclo Therapeutics’ R&D costs, which boosted research expenses to $16.9 million and adjusted G&A to $7.3 million.
4. Pipeline Development and Commercial Outlook
Rafael’s lead candidate, Trappsol® Cyclo™, targets a rare, fatal genetic disorder with significant unmet need. Successful Phase 3 completion and FDA collaboration position the company to transition into a commercial-stage biotechnology firm upon NDA approval.
