Rafael Holdings Stock Price, News & Analysis

Cornerstone Pharmaceuticals has successfully completed a Phase 1b clinical trial of its drug CPI-613® (devimistat) combined with gemcitabine and cisplatin for treating advanced biliary tract cancer. The trial involved 20 patients, achieving a 45% overall response rate and a median progression-free survival of 14.9 months. Seven patients are still receiving treatment. The Phase 2 trial is currently open, aiming to enroll 78 patients across 10 centers. Devimistat has been granted orphan drug designation by the FDA for multiple rare cancers, indicating its potential therapeutic importance.

Cornerstone Pharmaceuticals has appointed Dr. Jason Locasale to its Scientific Advisory Board. Dr. Locasale, an expert in cancer metabolism and metabolomics, is expected to enhance the company’s clinical programs and research on rare cancers. The company’s lead compound, CPI-613®, is under multiple clinical trials and has received orphan drug designations from both the FDA and EMA for several types of cancer. This appointment aims to strengthen the company’s ability to address the nutritional and metabolic challenges in cancer treatment.

Cornerstone Pharmaceuticals has announced the presentation of data from its open-label phase 1 study of CPI-613® (devimistat) combined with gemcitabine and nab-paclitaxel for locally advanced or metastatic pancreatic cancer. The results will be shared at the ESMO World Congress on Gastrointestinal Cancer 2022 from June 29 to July 2 in Barcelona, Spain. The study, involving 22 patients, demonstrated that devimistat is well tolerated and potentially synergistic with chemotherapy, paving the way for future treatment options for this prevalent and challenging cancer.

Cornerstone Pharmaceuticals announced the publication of preclinical data supporting CPI-613^® (devimistat) as a treatment targeting carcinoma catabolism. The study in PLOS ONE reveals that devimistat disrupts the TCA cycle, crucial for tumor metabolism. Notably, early clinical trials have shown strong activity against relapsed Myc-driven Burkitt lymphoma, with one complete remission reported in a Phase 1 trial. Cornerstone's commitment to rare cancer therapeutics continues with devimistat's orphan drug status from the FDA and EMA for several cancers.

Cornerstone Pharmaceuticals announced new preclinical data on CPI-613^® (devimistat) targeting carcinoma catabolism, published in PLOS ONE. This study highlights devimistat's ability to suppress the mitochondrial TCA cycle, crucial for tumor metabolism. Significant results include one complete remission in ongoing Phase 1 trials for relapsed, refractory Burkitt lymphoma. CEO Sanjeev Luther emphasized the potential for devimistat to improve outcomes for patients with rare cancers. The drug has received orphan drug designation from the FDA and EMA for several indications.

Rafael Holdings, Inc. (NYSE: RFL) reported its financial results for the three and nine months ended April 30, 2022. The company had cash reserves of $59.4 million and incurred a net loss of $5.5 million for Q3, up from $2.6 million the previous year. Revenue for the quarter was $0.9 million, slightly down from $1 million. Over the first nine months, net loss totaled $119.9 million, significantly higher than $12.2 million in the prior year, primarily due to an impairment loss. R&D expenses increased to $6.9 million as the company focuses on its early-stage drug development pipeline.

Cranbury, NJ, June 1, 2022 - Cornerstone Pharmaceuticals, formerly Rafael Pharmaceuticals, announced three abstracts will be presented at the ASCO Annual Meeting from June 3-7, 2022, in Chicago. These abstracts focus on CPI-613® (devimistat) in treating rare cancers, specifically clear cell sarcoma and biliary tract cancer, with special emphasis on pancreatic cancer. Key presentations will be made by notable researchers, discussing promising trial results. The company aims to advance treatment options for patients suffering from rare cancers.

Cranbury, NJ (May 24, 2022) – Cornerstone Pharmaceuticals announced that the European Medicines Agency (EMA) has granted orphan drug designation to CPI-613® (devimistat) for treating advanced unresectable biliary tract cancer. This rare cancer affects over 2,000 people annually in the UK, with increasing incidence rates. The designation marks the fourth for devimistat by the EMA, which is also recognized by the FDA for seven indications in the U.S. Strong Phase 2 trial enrollment suggests promise for devimistat in addressing significant unmet clinical needs in biliary tract cancer.

Cranbury, NJ, May 19, 2022 – Rafael Pharmaceuticals has officially changed its name to Cornerstone Pharmaceuticals, aligning with its mission to develop therapies for rare cancers. The company emphasized its commitment to addressing significant unmet needs in cancer treatment through its lead compound, CPI-613® (devimistat), which has received orphan drug designation from the FDA for multiple cancers.

Ongoing clinical trials for Burkitt's lymphoma, biliary tract cancer, and clear cell sarcoma are meeting key milestones, with new trials expected to launch in Q2 and Q3 2022.