BiotechTube
Rafael Holdings

Rafael Holdings

RFL
United States

Rafael Holdings is a clinical-stage oncology company leveraging its expertise in cancer metabolism to develop innovative therapeutics. Its core asset, CPI-613, is a lipoate analog that disrupts key mitochondrial enzymes, inducing selective cancer cell death. The company is advancing CPI-613 through multiple clinical trials, primarily in combination regimens, and maintains a strategic focus on aggressive and rare cancers with high unmet need.

rafaelholdings.com

RFL · Stock Price

USD 1.24+0.00 (+0.00%)
Market Cap: $66.1M

Historical price data

AI Company Overview

Rafael Holdings is a clinical-stage oncology company leveraging its expertise in cancer metabolism to develop innovative therapeutics. Its core asset, CPI-613, is a lipoate analog that disrupts key mitochondrial enzymes, inducing selective cancer cell death. The company is advancing CPI-613 through multiple clinical trials, primarily in combination regimens, and maintains a strategic focus on aggressive and rare cancers with high unmet need.

Oncology

Technology Platform

Focuses on cancer metabolism therapeutics, with a lead small molecule (CPI-613/devimistat) that is a lipoate analog disrupting key mitochondrial enzymes (PDH, KGDH) in the TCA cycle to induce selective cancer cell death.

Pipeline

18
18 drugs in pipeline2 in Phase 3
DrugIndicationStageWatch
Varenicline + Nicotine patchTobacco Use CessationApproved
Polyethylene glycol 4000 + Macrogol 3350 plus ascorbic acidColonoscopy PreparationApproved
inhaled corticosteroid plus LABA plus oral corticosteroidASTHMAApproved
IV trivalent saccharose hydroxide ferrous + Oral ferrous fum...AnaemiaApproved
Insulin + insulinDiabete Type 2Approved

Opportunities

CPI-613's novel cancer metabolism mechanism offers potential in combination therapies across multiple aggressive cancers.
Success in ongoing Phase 2 trials for borderline resectable pancreatic cancer could provide a new, viable development path.
The platform could be expanded to other TCA-cycle dependent tumors.

Risk Factors

Major risk stems from FDA clinical holds on two Phase 3 trials due to safety concerns, casting uncertainty on the lead program's future.
The company faces significant financial strain as a pre-revenue entity with costly clinical setbacks.
Intense competition in oncology necessitates rapid and clear clinical differentiation.

Competitive Landscape

Faces competition from leaders in cancer metabolism like Agios Pharmaceuticals, and large pharma with combination regimens in pancreatic cancer and AML. Differentiation hinges on CPI-613's unique lipoate analog mechanism and potential synergy with chemotherapies, but clinical delays have eroded its competitive position.