Rafael Pharmaceuticals is a clinical-stage oncology company focused on discovering and developing compounds which selectively target cancer metabolic pathways while simultaneously harnessing the immune system. We are harnessing the power of cancer metabolism to advance innovative therapeutics that can improve and extend the lives of cancer patients with difficult to treat solid and liquid tumors.

Our research in the field of cancer metabolism directed therapies has led to the discovery of novel, first-in-class drugs and drug delivery technologies that have the potential to revolutionize cancer therapy and treatment.

This research has been led by our world-leading scientific and medical advisors who have shaped the strategy, pipeline, and clinical development programs.

Our primary objective is to produce highly selective and effective anti-cancer agents with minimally toxic effects on healthy cells and tissues. By attacking regulatory processes that are unique to cancer and not found in healthy cells we aim to significantly improve the safety profile and efficacy of cancer treatment. Uniquely, our strategy is to defeat cancer by inhibiting pro-cancer cells while preserving or stimulating anti-cancer immune system cells.

CPI-613® (devimistat), our lead investigational drug targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Clinical development programs for devimistat are currently underway across multiple indications as a single agent, as well as in combination with standard drug therapy.

Devimistat has the potential to treat patient populations exceeding 100,000 in the U.S. and significantly more ex-U.S.

Devimistat has received orphan drug designations by the FDA in seven indications and by the EMA in three indications. Devimistat has IP protection and regulatory exclusivity out to 2029 and potentially beyond.

Devimistat has received Orphan Drug Designations in six indications and the IP and exclusivity out to 2029 and potentially beyond.

Questions about this breakthrough cancer therapy? Review the clinical trials or learn more about Rafael’s approach to metabolism

A successful and impactful ASH meeting
December 2019