Page 1

Rafael Holdings Provides Update on Rafael Pharmaceuticals ' Two Phase 3 Trials of CPI - 613® ( Devimistat ) , AVENGER 500 in Metastatic Pancreatic Cancer and ARMADA 2000 in Relapsed or Refractory Acute Myeloid Leukemia Phase 3 Clinical Trial in Metastatic Pancreatic Cancer Did Not Meet its Primary Endpoint of Improved Overall Survival Independent Data Monitoring Committee Recommended the Phase 3 Clinical Trial in Relapsed or Refractory Acute Myeloid Leukemia Be Stopped Due to Lack of Efficacy Rafael Holdings to Host Conference Call Today at 8:00 a.m. ET NEWARK , N.J. , Oct. 28 , 2021 ( GLOBE NEWSWIRE ) Rafael Holdings , Inc. , ( NYSE : RFL ) , a holding company focused on developing novel cancer metabolism therapeutics through its Barer Institute , investment in Rafael Pharmaceuticals , Inc. ( " Rafael Pharmaceuticals " ) as well as other investments in early - stage ventures , today announced that the AVENGER 500 Phase 3 clinical trial for CPI - 613® ( devimistat ) , Rafael Pharmaceuticals ' lead product candidate , did not meet its primary endpoint of significant improvement in overall survival in patients with metastatic adenocarcinoma of the pancreas . In this multi - national Phase 3 randomized clinical trial , 528 patients with metastatic adenocarcinoma of the pancreas who had no prior therapy were randomized to receive either devimistat in combination with modified FOLFIRINOX ( mFFX ) in the treatment arm or FOLFIRINOX ( FFX ) alone in the control arm . Devimistat given with mFFX did not significantly improve overall survival ( HR = 0.95 , p = 0.66 ) compared to FFX alone . The median overall survival in the treatment arm was 11.1 months , compared to 11.7 months in the control arm . Devimistat is also being evaluated in a multi - national Phase 3 randomized clinical trial , ARMADA 2000 , in patients with relapsed or refractory acute myeloid leukemia ( AML ) . Following a pre - specified interim analysis , the independent data monitoring committee has recommended the trial be stopped due to lack of efficacy . " We are disappointed by the results of these two Phase 3 clinical trials with devimistat . We plan to work in collaboration with Rafael Pharmaceuticals to further evaluate the data , " said Ameet Mallik , CEO , Rafael Holdings , Inc. " We would like to thank the patients , families , and investigators for their participation in the AVENGER 500 and ARMADA 2000 trials . " Conference Call Information : Date : Thursday , October 28 , 2021 Time : 8:00 a.m. ET Domestic Dial - in Number : ( 800 ) 547-0089 International Dial - in Number : ( 214 ) 989-7192 Conference ID : 4670624 Live Webcast : accessible from the Company's website at https://rafaelholdings.com under Events and Presentations or with this link : https : //edge.media server.com/mmc/p/izzdnghf . AVENGER 500 Phase 3 Clinical Trial AVENGER 500 is a Phase 3 multinational , randomized controlled clinical trial to evaluate the efficacy and safety of devimistat ( CPI - 613® ) , in combination with modified FOLFIRINOX ( mFFX ) in comparison to FOLFIRINOX . Patients eligible for enrollment in this study included adults 18-75 years old of both sexes with metastatic ( stage IV ) pancreatic adenocarcinoma , not previously treated for metastatic disease and with ECOG performance status of 0 1 . ARMADA 2000 Phase 3 Clinical Trial ARMADA 2000 is a Phase 3 multinational , randomized controlled clinical trial to evaluate the efficacy and safety of devimistat ( CPI - 613® ) in combination with high dose cytarabine and mitoxantrone ( CHAM ) as compared to high dose cytarabine and mitoxantrone ( HAM ) and control sub - groups : combination of mitoxantrone , etoposide and cytarabine ( MEC ) and combination of fludarabine , cytarabine , and filgrastim ( FLAG ) in patients 50 years of age or older with relapsed or refractory acute myeloid leukemia ( AML ) . About Devimistat ( CPI - 613® ) Rafael Pharmaceuticals ' lead compound devimistat ( CPI - 613® ) is designed to selectively target the tumor mitochondrial tricarboxylic acid ( TCA ) cycle , a process essential to tumor cell multiplication and survival . Devimistat is designed to increase cellular stress and may increase the sensitivity of cancer cells to a diverse range of chemotherapeutic agents . The U.S. Food and Drug Administration ( FDA ) has designated devimistat as an orphan drug for the treatment of pancreatic cancer , acute myeloid leukemia , myelodysplastic syndrome , peripheral T - cell lymphoma , soft tissue sarcoma , Burkitt's lymphoma and biliary tract cancer . The European Medicines Agency ( EMA ) has granted orphan drug designation to devimistat for pancreatic cancer , acute myeloid leukemia and Burkitt's lymphoma . About Rafael Holdings , Inc. Rafael Holdings is focused on the development of novel cancer therapies . The company owns the Barer Institute , Inc. and is a significant investor in two clinical stage oncology companies , Rafael Pharmaceuticals , Inc. , and LipoMedix Pharmaceuticals Ltd. Through the Barer Institute , the company is developing a pipeline of compounds focused on the regulation of cancer and immune metabolism . On June 21 , 2021 , the company announced that it has entered into a merger agreement to acquire full ownership of Rafael Pharmaceuticals , Inc. For more information , visit www.rafaelholdings.com . About Rafael Pharmaceuticals , Inc. Rafael Pharmaceuticals is focused on the growing field of cancer metabolism . The company is developing a new category of metabolic oncology therapeutic candidates that are designed to attack hard - to - treat cancers by targeting the metabolic processes that these cancers need to survive , grow , and