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

Rhea-AI Summary

Rafael Pharmaceuticals announced that the U.S. FDA has granted Fast Track designation for its lead compound, CPI-613® (devimistat), for treating metastatic pancreatic cancer. This designation highlights the urgent need for new treatment options for this aggressive cancer. Rafael also achieved target enrollment of 500 patients for its Phase 3 trial ahead of schedule. Additionally, devimistat has received Orphan Drug Designation for soft tissue sarcoma. The drug aims to enhance the effectiveness of chemotherapy while minimizing side effects, representing a significant advance in cancer treatment.

Positive

• FDA granted Fast Track designation for CPI-613® (devimistat) for metastatic pancreatic cancer.
• Achieved target enrollment of 500 patients for Phase 3 trial ahead of schedule.
• CPI-613® also received Orphan Drug Designation for the treatment of soft tissue sarcoma.

Negative

• None.

CRANBURY, N.J., Nov. 10, 2020 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a leader in the growing field of cancer metabolism-based therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company’s lead compound, CPI-613® (devimistat), for the treatment of metastatic pancreatic cancer. 

“Receiving Fast Track designation is a significant milestone in our fight against pancreatic cancer,” said Sanjeev Luther, President and CEO of Rafael. “This designation further stresses the severe unmet need in treatment options for this aggressive and devastating disease. We are truly thankful to the doctors, researchers, the FDA and all of our supporters who have made this possible. And most of all, we are grateful to the patients involved in our trials; the patients are the inspiration and driving force behind all of our efforts.”  

Rafael has continued to reach milestones throughout the year, including achieving its target enrollment of 500 patients for its Phase 3 trial for metastatic pancreatic cancer ahead of schedule. The Company also recently announced that the FDA granted devimistat Orphan Drug Designation for the treatment of soft tissue sarcoma.  

“Pancreatic cancer is notoriously challenging to treat and long overdue for a new approach,” said Philip A. Philip, M.D., Ph.D., FRCP, Professor of Oncology at the Barbara Ann Karmanos Cancer Institute at Wayne State University and a medical advisor to Rafael. “We have remained hopeful throughout our pancreatic cancer trials, and now with Fast Track designation, our optimism is further fueled. We believe with this designation, cancer metabolism is truly being propelled forward, with devimistat at the helm.”

About CPI-613® (devimistat)  
CPI-613® (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient’s side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patient’s benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500®) and acute myeloid leukemia (ARMADA 2000), and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma, Burkitt’s lymphoma and soft tissue sarcoma. The 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, first-in-class category 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 effective anti-cancer agent that is being evaluated in ongoing and completed Phase 1, 2 and 3 clinical trials. Devimistat has been granted orphan drug status by the FDA for the treatment of pancreatic cancer, acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and Burkitt’s,peripheral T-cell lymphomas and soft tissue sarcoma. The Company's investors include Rafael Holdings, Inc. (NYSE AMERICAN: RFL). For more information, please visit www.rafaelpharma.com.   

Safe Harbor Statement
This press release contains forward-looking statements. These statements relate to future events or the company’s future financial performance. In some cases, you can identify forward-looking statements by terminology such as "may", "will", "should", "expect", "plan", "anticipate", "believe", "estimate", "predict", "potential" or "continue", the negative of such terms, or other comparable terminology. These statements are only predictions. Actual events or results may differ materially from those in the forward-looking statements as a result of various important factors. Although we believe that the expectations reflected in the forward-looking statements are reasonable, such statements should not be regarded as a representation by the company, or any other person, that such forward-looking statements will be achieved. The business and operations of the company are subject to substantial risks which increase the uncertainty inherent in forward-looking statements. We undertake no duty to update any of the 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 such forward-looking statements.

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FAQ

What is the significance of the Fast Track designation for RFL?

The Fast Track designation by the FDA for CPI-613® (devimistat) signifies streamlined development and priority review for treating metastatic pancreatic cancer, highlighting its potential to address unmet medical needs.

What milestones has Rafael Pharmaceuticals achieved in 2020?

In 2020, Rafael Pharmaceuticals achieved target enrollment of 500 patients for its Phase 3 trial ahead of schedule and received Fast Track designation for CPI-613®.

What are the future prospects for Rafael Pharmaceuticals with CPI-613®?

With the Fast Track designation and ongoing clinical trials, CPI-613® has strong prospects for advancing cancer treatment options, particularly for pancreatic cancer.

What other designations has CPI-613® received apart from Fast Track?

CPI-613® has also received Orphan Drug Designation for the treatment of soft tissue sarcoma, enhancing its clinical importance.