Home music industry Rafael Pharmaceuticals Receives FDA Fast Track Designation for CPI-613® (devimistat) for Pancreatic Cancer Treatment

Rafael Pharmaceuticals Receives FDA Fast Track Designation for CPI-613® (devimistat) for Pancreatic Cancer Treatment



CRANBURY, NJ, November 10, 2020 (GLOBE NEWSWIRE) – Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a leader in the rapidly growing field of therapies based on cancer metabolism, today announced that the United States Food and Drug Administration (FDA) has granted the designation Fast Track for the Company’s main compound, CPI-613. ® (devimistat), for the treatment of metastatic pancreatic cancer.

“Achieving the Fast Track designation is an important step in our fight against pancreatic cancer,” said Sanjeev Luther, President and CEO of Rafael. “This designation further underscores the serious unmet need for treatment options for this aggressive and devastating disease. We are truly grateful to the doctors, researchers, the FDA, and all of our supporters who made this possible. Most importantly, we are grateful to the patients involved in our trials; patients are the inspiration and the driving force behind all of our efforts.

Rafael continued to reach milestones throughout the year, including meeting its target of recruiting 500 patients for its phase 3 trial in metastatic pancreatic cancer ahead of schedule. The Company also recently announced that the FDA has granted orphan drug designation to devimistat for the treatment of soft tissue sarcoma.

“Pancreatic cancer is notoriously difficult to treat and a new approach is long overdue,” said Philip A. Philip, MD, Ph.D., FRCP, professor of oncology at the Barbara Ann Karmanos Cancer Institute at Wayne State University and a medical adviser from Rafael. “We remained hopeful throughout our pancreatic cancer trials, and now with the Fast Track designation, our optimism is further fueled. We believe that with this designation, the cancer metabolism is truly propelled forward, with devimistat at the helm. “

About the IPC-613® (devimistat)
CPI-613® (devimistat) is a leading clinical compound from Rafael, which targets enzymes involved in the energy metabolism of cancer cells and located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential for tumor cell growth and survival, selectively within cancer cells. Devimistat dramatically increases the sensitivity of cancer cells to a wide range of chemotherapeutic agents. This synergy allows potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with fewer side effects for the patient. The combination with devimistat represents a wide range of opportunities to dramatically improve the benefit for patients in many different cancers. The United States Food and Drug Administration (FDA) has cleared Rafael to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500®) and acute myeloid leukemia (ARMADA 2000), and has designated devimistat as orphan drug for the treatment of the pancreas. cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T cell lymphoma, Burkitt lymphoma and soft tissue sarcoma. EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.

About Rafael Pharmaceuticals, Inc.
Rafael Pharmaceuticals is a leader in the growing field of cancer metabolism. The company is developing a new class of metabolic oncology therapeutics that attack hard-to-treat cancers by targeting the metabolic processes the disease needs to survive, grow and proliferate. Rafael Pharmaceuticals’ lead compound, CPI-613® (devimistat), is a highly selective, well-tolerated and potent anticancer agent being evaluated in ongoing and completed Phase 1, 2 and 3 clinical trials. Devimistat has been granted orphan drug status from the FDA for the treatment of pancreatic cancer, acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and Burkitt’s disease, peripheral T lymphomas and sarcoma. soft tissue. The Company’s investors include Rafael Holdings, Inc. (NYSE AMERICAN: RFL). For more information, please visit www.rafaelpharma.com.

Safe Harbor Declaration
This press release contains forward-looking statements. These statements relate to future events or the future financial performance of the company. In some cases, you can identify forward-looking statements by words such as “may”, “will”, “should”, “expect”, “plan”, “anticipate”, “believe”, “estimate”, “predict”, “potential” or “continue”, the negative of these terms or any comparable terminology. These statements are only predictions. Actual events or results may differ materially from those of forward-looking statements due to various important factors. Although we believe that the expectations reflected in forward-looking statements are reasonable, such statements should not be taken as a representation by the company, or any other person, that such forward-looking statements will be realized. The business and operations of the Company are subject to substantial risks which increase the uncertainty inherent in forward-looking statements. We assume no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.

In light of the foregoing, readers are cautioned not to place undue reliance on these forward-looking statements.


CONTACT: Rafael Media Contact: Vanessa Donohue [email protected] (201) 465-8036