Rafael Holdings Stock Price, News & Analysis

Rafael Pharmaceuticals has opened new trial sites for the APOLLO 613 Phase 1/2 clinical trial of CPI-613® (devimistat) in combination with hydroxychloroquine for patients with clear cell sarcoma. The first patient has been enrolled at City of Hope, with additional sites now available at Cleveland Clinic Children’s and University of Michigan’s Rogel Cancer Center. With clear cell sarcoma being aggressive and difficult to treat, the company aims to provide more treatment options. Devimistat targets cancer cell metabolism and has FDA orphan drug designation for several cancers.

Rafael Pharmaceuticals announced the European Medicines Agency (EMA) has granted orphan drug designation to CPI-613® (devimistat) for treating Burkitt's lymphoma, a rare cancer affecting mainly children. This designation is the third for devimistat by the EMA, complementing seven orphan drug designations by the FDA in the U.S. Burkitt's lymphoma represents about 1% of adult lymphoma cases, with few treatment options available. The company aims to address the urgent need for effective therapies for patients with relapsed or refractory cases.

Rafael Pharmaceuticals announced disappointing results from its AVENGER 500 Phase 3 trial, which assessed CPI-613^® (devimistat) combined with modified FOLFIRINOX for metastatic pancreatic cancer. The study did not meet its primary endpoint of improving overall survival, reporting 11.1 months for devimistat plus mFFX versus 11.7 months for standard FOLFIRINOX (HR=0.95, p=0.66).

In addition, the ARMADA 2000 trial in relapsed acute myeloid leukemia was halted due to lack of efficacy, as recommended by an independent committee.

Rafael Holdings, Inc. (NYSE: RFL) announced disappointing results from two Phase 3 clinical trials of its lead candidate, devimistat (CPI-613^®). The AVENGER 500 trial for metastatic pancreatic cancer failed to meet its primary endpoint of improved overall survival (11.1 months vs. 11.7 months). In another trial, ARMADA 2000 for relapsed or refractory acute myeloid leukemia (AML), an independent committee recommended stopping the trial due to lack of efficacy. The company plans to collaborate with Rafael Pharmaceuticals to assess the data further.

Rafael Holdings, Inc. (NYSE: RFL) announced a stock option grant to Dr. Mary Margaret Huizinga for 122,341 shares of Class B common stock, with an exercise price of $30.54, the closing price on October 15, 2021. The options will vest over four years, beginning October 18, 2022. This grant follows Dr. Huizinga's appointment as Chief Development and Medical Officer, intended as an inducement under NYSE Rule 303A.08. Rafael Holdings focuses on developing cancer therapies and has a significant stake in clinical-stage oncology companies.

Rafael Holdings, Inc. (NYSE: RFL) announced plans to report top line data from its AVENGER 500 Phase 3 trial for metastatic pancreatic cancer and submit a merger proposal with Rafael Pharmaceuticals for stockholder voting within the year. The company closed a PIPE financing, securing $97.8 million in August 2021, and appointed new senior pharmaceutical executives to strengthen operations. Financial results show a net loss of $24.8 million for fiscal 2021, reflecting increased R&D and operational expenses as it develops novel oncology therapies through the Barer Institute.

Rafael Holdings, Inc. (NYSE: RFL) announced the appointment of Mary Margaret (Mimi) Huizinga, MD, MPH as Chief Development and Medical Officer, effective October 18, 2021. This strategic move reflects the importance of the company’s late-stage clinical programs in developing cancer metabolism therapies. Huizinga brings extensive experience from her role at Novartis and a proven track record in oncology development. Her leadership is expected to enhance clinical efforts, particularly as the company approaches a critical phase with the Phase 3 trial data readout for CPI-613® in metastatic pancreatic cancer.

Rafael Pharmaceuticals announces the start of its Phase 1 clinical trial involving CPI-613® (devimistat) combined with hydroxychloroquine for patients with clear cell sarcoma, a rare and aggressive cancer. Enrollment will begin at City of Hope, California, with additional sites to follow. The trial aims to address significant unmet medical needs in this challenging sarcoma type. CPI-613® targets mitochondrial functions in cancer cells, potentially improving treatment efficacy with reduced side effects. The FDA has also designated devimistat as an orphan drug for several cancers, signaling strong research support.

Rafael Holdings, Inc. (NYSE: RFL), a pharmaceutical holding company, has appointed Ashok Marin as Chief Legal Officer. The appointment comes as the company anticipates top-line data from Phase 3 trials for CPI-613, a key drug for metastatic pancreatic cancer. Marin, with over 20 years of experience, previously held senior legal roles at Immunomedics, contributing to its $21 billion acquisition by Gilead. Rafael granted Marin options to purchase 34,066 shares at $37.61, vesting over four years, as part of his employment inducement.