Rafael Holdings, Inc.
- Market Cap (Float-Adj) (Float-adjusted market cap computed by the low-float screener — matches the screener row. May differ from the standard cap (price × all shares outstanding) reported elsewhere.)
- $77.0M
- Price
- $1.48
- % Change (Daily % change from the live per-ticker quote feed.)
- -0.67%
- Volume
- 55.4K
About
Rafael Holdings, Inc. holds strategic investments in both early-stage and clinical pharmaceutical companies, alongside a portfolio of commercial real estate assets situated in the United States and Israel. The company's business activities are structured into two primary divisions: Pharmaceuticals and Real Estate. Its Real Estate segment manages and leases a commercial office complex, which features an integrated 800-space public parking facility. The Pharmaceutical division, conversely, focuses on developing and bringing to market therapies designed to capitalize on the metabolic distinctions between healthy cells and malignant ones. The firm's flagship drug candidate is CPI-613 (devimistat), currently undergoing evaluation in numerous clinical trials, including two pivotal Phase III registrational studies for the treatment of advanced pancreatic cancer and relapsed/refractory acute myeloid leukemia. Rafael Holdings, Inc. is headquartered in Newark, New Jersey.
Quick stats
Shares outstanding52.0M
Computed float30.6M
Float / shares58.8%
Cash runway (months)8.1
Key info
SectorReal Estate
IndustryReal Estate - Services
CIK (Central Index Key — the unique ID the SEC assigns to every filer. Use it to look up the company's filings on EDGAR.)0001713863
AI Summary
AI-generated1/18
majormixedclinical trial
Rafael Holdings Announces Topline Results from the Pivotal Phase 3 TransportNPC™ Study of Trappsol® Cyclo™ in Niemann-Pick Disease Type C (NPC)
Rafael Holdings' pivotal Phase 3 TransportNPC study of Trappsol Cyclo in Niemann-Pick Disease Type C missed statistical significance on the primary endpoint, showing a 64% slowing of disease progression (p=0.19) at Week 96.
A prespecified analysis in the ~83% of patients on background miglustat and/or leucine therapy showed a statistically significant 71% slowing of progression (p=0.046), and separate survival analyses vs. external controls suggested 85-94% mortality risk reduction.
The safety profile was well tolerated with no new safety signals, though serious TEAEs were more frequent on drug (35.9% vs 16.7%) and one severe treatment-related bilateral deafness occurred.
Based on a prior pre-NDA meeting, the company remains on track to submit the NDA in Q4 2026, leaving FDA acceptance of an application built on a missed primary endpoint as the key risk.
Red flags
- Primary endpoint missed statistical significance (p=0.19); NDA relying on subgroup and external-control survival analyses
- Serious treatment-emergent adverse events more frequent on drug (35.9% vs 16.7% placebo); one severe treatment-related bilateral deafness
- FDA may not accept or approve NDA based on available data (company's own forward-looking risk)