RFL Rafael Holdings Inc. Class B

5.9
-0.36  -6%
Previous Close 6.26
Open 6.08
52 Week Low 5.54
52 Week High 66.44
Market Cap $121,896,254
Shares 20,660,382
Float 19,092,450
Enterprise Value $136,007,990
Volume 192,924
Av. Daily Volume 949,224
Stock charts supplied by TradingView

Drug Pipeline

Drug Stage Notes
CPI-613 (devimistat) + hydroxychloroquine - (APOLLO 613)
Clear cell sarcoma
Phase 1/2
Phase 1/2
Phase 1/2 first patient enrolled noted November 11, 2021.
CPI-613 (devimistat) + cytarabine + mitoxantrone - (ARMADA 2000)
Acute myeloid leukemia (AML)
Phase 3
Phase 3
Phase 3 trial to be stopped due to lack of efficacy based on recommendation from independent data monitoring committee, noted October 28, 2021.
CPI-613 (devimistat) + mFOLFIRINOX - (AVENGER 500)
Pancreatic Cancer
Phase 3
Phase 3
Phase 3 trial did not meet its primary endpoint of significant improvement in overall survival patients with metastatic adenocarcinoma of the pancreas, noted October 28, 2021. NDA filing due 1H 2022.
CPI-613 (devimistat) + hydroxychloroquine
Relapsed or Refractory Myelodysplastic Syndrome (MDS)
Phase 2
Phase 2
Phase 2 clinical trial to initiate in 4Q 2021
CPI-613 (devimistat) + bendamustine
Relapsed or Refractory T - cell Lymphoma
Phase 2
Phase 2
Phase 2 clinical trial to initiate in 4Q 2021
CPI-613 (devimistat)
Burkitt's lymphoma/leukemia or high-grade B-cell lymphoma
Phase 1/2
Phase 1/2
Phase 1/2 trial ongoing.
CPI-613 (devimistat) + FOLFIRINOX + bevacizumab
Colorectal cancer
Phase 1
Phase 1
Phase 1 trial to be initiated.
CPI-613 (devimistat) + gemcitabine + nab-paclitaxel
Locally advanced or metastatic pancreatic cancer
Phase 1
Phase 1
Phase 1 data presented at ASCO 2020 showed 1 patient achieved CR, 9 PR, 8 stable disease and 1 progressive disease for an objective response rate of 50% with a CR rate of 5.5%. Phase 1 trial ongoing.
CPI-613 (devimistat) + gemcitabine + cisplatin
Metastatic biliary tract cancer
Phase 1/2
Phase 1/2
Phase 1b trial completed and Phase 2 trial initiated August 25, 2021.
CPI-613 (devimistat) + mFOLFIRINOX
Locally advanced pancreatic cancer
Phase 2
Phase 2
Phase 2 trial ongoing.

Latest News

  1. - Ameet Mallik to Transition CEO Role to Chairman, Howard Jonas, and Will Remain a Highly Engaged Member of the Company's Board of Directors and Chair Newly Established Transition Committee -

    Patrick Fabbio, Chief Financial Officer, to Assume the Additional Role of President -

    - Dr. Mimi Huizinga, Chief Medical Officer, to Assume the Additional Role of Head of Research and Development -

    NEWARK, N.J., Nov. 22, 2021 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc., (NYSE:RFL), a company focused on developing novel cancer and immune metabolism therapeutics through its Barer Institute and investment in Rafael Pharmaceuticals, Inc. today announced several changes to its leadership team. Ameet Mallik will transition his Chief Executive Officer responsibilities…

    - Ameet Mallik to Transition CEO Role to Chairman, Howard Jonas, and Will Remain a Highly Engaged Member of the Company's Board of Directors and Chair Newly Established Transition Committee -

    Patrick Fabbio, Chief Financial Officer, to Assume the Additional Role of President -

    - Dr. Mimi Huizinga, Chief Medical Officer, to Assume the Additional Role of Head of Research and Development -

    NEWARK, N.J., Nov. 22, 2021 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc., (NYSE:RFL), a company focused on developing novel cancer and immune metabolism therapeutics through its Barer Institute and investment in Rafael Pharmaceuticals, Inc. today announced several changes to its leadership team. Ameet Mallik will transition his Chief Executive Officer responsibilities to Chairman, Howard Jonas, as of February 1, 2022, and will remain highly engaged as a member of the Board of Directors of Rafael Holdings. Patrick Fabbio will assume the position of President, in addition to continuing as Chief Financial Officer, with the responsibility to lead the operations and finances of the business. Dr. Mimi Huizinga will continue as Chief Medical Officer and assume additional responsibilities as the Head of Research and Development. She will lead the company's research, development and medical strategy and operations. To oversee the management transition and the organization's evolution, the Board has established a Transition Committee, which Ameet will chair.

    "I joined Rafael Holdings to recruit a top-tier leadership team to evolve the company into a fully integrated commercial organization. While the recent results of Rafael Pharmaceuticals' two Phase 3 clinical trials for CPI-613® (devimistat) in metastatic pancreatic cancer and relapsed or refractory acute myeloid leukemia are disappointing, I am proud of and grateful for the team's significant contributions," said Ameet Mallik, outgoing CEO and continuing member of the Board. "We are fortunate to have an early-stage pipeline backed by world class scientific advisors, which we believe has great potential. I would like to congratulate Mimi and Pat on their expanded roles, and I have the utmost confidence in their ability to move the company forward."

    With the change in company focus, Rafael Holdings also announced the planned departures of: William Conkling, Chief Commercial and Business Officer; Ashok David Marin, Chief Legal Officer; Brandi Robinson, Chief Corporate Affairs Officer; and Melissa Lozner, Chief Compliance and Ethics Officer. These transitions will take place by the end of January 2022.

    "I would like to thank Ameet and the current leadership team for enhancing the capabilities of the company," said Howard Jonas, Chairman of the Board, Rafael Holdings. "I look forward to Ameet continuing as a member of the Rafael Holdings Board of Directors and to working with Pat and Mimi to advance our research and development efforts. I'm excited about the future of Rafael Holdings and what we will accomplish together."

    Patrick Fabbio most recently served as Chief Financial Officer of the Company and has more than 25 years of financial, operational, and transactional leadership experience in both publicly traded and privately held life science and pharmaceutical companies. Prior to joining Rafael Holdings, Patrick was Chief Financial Officer of WindMIL Therapeutics Inc. Previously he served as the Chief Financial Officer of Progenics Pharmaceuticals, Inc., electroCore Medical, LLC; Vice President of Finance at NPS Pharmaceuticals, Inc.; Vice President of Finance, Innovation and Growth at Catalent Pharma Solutions Inc.; and Chief Financial Officer at Ikano Therapeutics. His other prior financial positions include roles at Sanofi, UniPath Diagnostics, BioMatrix and Coopers & Lybrand. Patrick is a board member of BeyondSpring Therapeutics, Inc. 

    Dr. Huizinga recently served as Chief Development and Medical Officer of the Company. Prior to joining Rafael Holdings, she was the Head of US Oncology Medical for Novartis, overseeing the clinical development and medical affairs activities of the Novartis oncology portfolio in the US. Before leading the medical function, Dr. Huizinga built and led the strategic data and digital function in the US Oncology business unit at Novartis. The strategic data and digital function included real-world evidence, healthcare economics and clinical and value evidence liaisons in addition to supporting digital marketing and constructing a data strategy and platform. During her time at Novartis, Dr. Huizinga participated in more than a dozen product and indication launches. Prior to Novartis, she was the Chief Health Information Officer at Premier, Inc, where she also led the Applied Sciences division partnering with several pharmaceutical companies around evidence generation needs. Dr. Huizinga has previously served as the VP of Quality at Lifepoint Health, an associate at McKinsey & Company and an Assistant Professor at the Johns Hopkins University. Dr. Huizinga holds an MD and MPH from Vanderbilt University and is a fellow of the American College of Physicians.

    About Rafael Holdings, Inc.

    Rafael Holdings is focused on the development of novel cancer and immune metabolism therapeutics. 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 proliferate. Rafael Pharmaceuticals' lead compound, CPI-613® (devimistat), is an investigational anti-cancer agent that is being evaluated in ongoing Phase 1, 2 and 3 clinical trials. The Company's investors include Rafael Holdings, Inc. (NYSE:RFL). On June 21, 2021, Rafael Pharmaceuticals announced that it has entered into a merger agreement with Rafael Holdings, Inc. to create a publicly traded late-stage clinical oncology company focused on cancer metabolism-based therapeutics. For more information, please visit www.rafaelpharma.com.

    Forward Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans regarding the further evaluation of clinical data; and the potential of our pipeline, including our internal cancer metabolism research programs. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of public health threats, including COVID-19, on our business and operations; we depend heavily on the success of Rafael Pharmaceuticals and the future success of its lead product candidate devimistat (CPI-613®), and clinical trials of the product candidate may not be successful; our pharmaceutical companies may not be able to develop any medicines of commercial value; our pharmaceutical companies may not be successful in their efforts to identify or discover potential product candidates; the manufacturing and manufacturing development of our products and product candidates present technological, logistical and regulatory risks, each of which may adversely affect our potential revenue; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; our product candidates may cause serious adverse side effects; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended July 31, 2021, and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

    Contact:

    Barbara Ryan



    (203) 274-2825

     



    Primary Logo

    View Full Article Hide Full Article
  2. CRANBURY, N.J., Nov. 11, 2021 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a company focused on the growing field of cancer metabolism-based therapeutics, announced that the first patient has been enrolled in the APOLLO 613 Phase 1/2 clinical trial of CPI-613® (devimistat) in combination with hydroxychloroquine in patients with clear cell sarcoma at City of Hope in Duarte, California. Two additional sites have also been opened for enrollment in the trial at Cleveland Clinic Children's and University of Michigan's Rogel Cancer Center.


    "With the aggressive nature of clear cell sarcoma, finding a treatment for this rare cancer is a priority of ours," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals…

    CRANBURY, N.J., Nov. 11, 2021 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a company focused on the growing field of cancer metabolism-based therapeutics, announced that the first patient has been enrolled in the APOLLO 613 Phase 1/2 clinical trial of CPI-613® (devimistat) in combination with hydroxychloroquine in patients with clear cell sarcoma at City of Hope in Duarte, California. Two additional sites have also been opened for enrollment in the trial at Cleveland Clinic Children's and University of Michigan's Rogel Cancer Center.



    "With the aggressive nature of clear cell sarcoma, finding a treatment for this rare cancer is a priority of ours," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "Enrolling a patient so soon after we opened the trial demonstrates the dire need for a treatment and the hope that devimistat brings to the rare cancer community. We opened two additional sites in order to increase access to the trial for patients in need."



    Clear cell sarcoma is very difficult to diagnose, and the average age of diagnosis is 25 years old. By the time the disease is diagnosed, it is often in advanced stages when the cancer is aggressive and hard to treat.



    "Clear cell sarcoma has the propensity to spread quickly within the body, and our current available medical therapies are not consistently beneficial to patients," said Rashmi Chugh, M.D., co-principal investigator from the University of Michigan. "Finding effective treatments for the disease is critical, and we are hopeful that devimistat's mechanism of action will be effective in clear cell sarcoma patients."



    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 cellular stress and 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 side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patients' benefit in many different cancers. 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 Pharmaceuticals, Inc.

    Rafael Pharmaceuticals is focused on the growing field of cancer metabolism. The company is developing a new, first-in-class 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 proliferate. Rafael Pharmaceuticals' lead compound, CPI-613® (devimistat), is an investigational anti-cancer agent that is being evaluated in ongoing and completed Phase 1, 2 and 3 clinical trials. The Company's investors include Rafael Holdings, Inc. (NYSE:RFL). On June 21, 2021, we announced that we have entered into a merger agreement with Rafael Holdings, Inc. to create a publicly traded late-stage clinical oncology company focused on cancer metabolism-based therapeutics. 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.







    Media Contact:
    Sharon Golubchik 
     
    (609) 418-0058

    Primary Logo

    View Full Article Hide Full Article
  3. CRANBURY, N.J., Nov. 03, 2021 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a company focused on the growing field of cancer metabolism-based therapeutics, today announced that the European Medicines Agency (EMA) has granted orphan drug designation to CPI-613® (devimistat) for the treatment of Burkitt's lymphoma.

    In the UK, 2 out of 100 cases of non-Hodgkin's lymphoma (NHL) are identified as Burkitt's lymphoma, making it an extremely rare cancer. In addition, Burkitt's lymphoma accounts for only 1% of adult lymphoma, but up to 30% of childhood NHL –– creating a dire need for families who have children with this type of lymphoma if the first line of treatment fails. Presently, no substantial treatment…

    CRANBURY, N.J., Nov. 03, 2021 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a company focused on the growing field of cancer metabolism-based therapeutics, today announced that the European Medicines Agency (EMA) has granted orphan drug designation to CPI-613® (devimistat) for the treatment of Burkitt's lymphoma.

    In the UK, 2 out of 100 cases of non-Hodgkin's lymphoma (NHL) are identified as Burkitt's lymphoma, making it an extremely rare cancer. In addition, Burkitt's lymphoma accounts for only 1% of adult lymphoma, but up to 30% of childhood NHL –– creating a dire need for families who have children with this type of lymphoma if the first line of treatment fails. Presently, no substantial treatment options beyond first-line therapy exist.

    "Relapsed or refractory Burkitt's lymphoma can be a devastating form of cancer that is in need of effective treatment options," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "Milestones such as this can offer hope for the patients and families who suffer from this rare form of lymphoma with few options to turn to."

    This orphan drug designation is the third granted for devimistat by the EMA, following designations for pancreatic cancer and acute myeloid leukemia (AML). The Food and Drug Administration (FDA) has granted orphan drug designation for devimistat in seven indications in the United States, including in Burkitt's lymphoma. 

    "We have been investigating the use of devimistat for treatment in relapsed or refractory Burkitt's lymphoma. It is a testament to our work and the potential of the compound to have received another orphan drug designation," said Ariela Noy, M.D., medical oncologist for lymphoma and AIDS-associated cancers at Memorial Sloan Kettering Cancer Center (MSK), emeritus chair of the Lymphoma Working Group for the AIDS Malignancy Consortium, and principal investigator on Rafael's Phase 2 trial in Burkitt's lymphoma.  "HIV dramatically increases the risk of Burkitt's lymphoma. Devimistat brings hope to the many patients with  relapsed or refractory Burkitt's lymphoma, as well as their loved ones."

    Disclosures: Dr. Noy has provided consulting services to Rafael Pharmaceuticals.

    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 cellular stress and 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 side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patients' benefit in many different cancers. 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 Pharmaceuticals, Inc.

    Rafael Pharmaceuticals is focused on the growing field of cancer metabolism. The company is developing a new, first-in-class 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 proliferate. Rafael Pharmaceuticals' lead compound, CPI-613® (devimistat), is an investigational anti-cancer agent that is being evaluated in ongoing and completed Phase 1, 2 and 3 clinical trials. The Company's investors include Rafael Holdings, Inc. (NYSE:RFL). On June 21, 2021, we announced that we have entered into a merger agreement with Rafael Holdings, Inc. to create a publicly traded late-stage clinical oncology company focused on cancer metabolism-based therapeutics. 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.



    Media Contact:
    Sharon Golubchik 
     
    (609) 418-0058

    Primary Logo

    View Full Article Hide Full Article
  4. CRANBURY, N.J., Oct. 28, 2021 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a company focused on the growing field of cancer metabolism-based therapeutics, today announced that the AVENGER 500 Phase 3 clinical trial evaluating the efficacy and safety of CPI-613® (devimistat) in combination with modified FOLFIRINOX (mFFX) as a first-line therapy in patients with metastatic adenocarcinoma of the pancreas did not meet its primary endpoint of overall survival.

    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) or FOLFIRINOX, a current…

    CRANBURY, N.J., Oct. 28, 2021 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a company focused on the growing field of cancer metabolism-based therapeutics, today announced that the AVENGER 500 Phase 3 clinical trial evaluating the efficacy and safety of CPI-613® (devimistat) in combination with modified FOLFIRINOX (mFFX) as a first-line therapy in patients with metastatic adenocarcinoma of the pancreas did not meet its primary endpoint of overall survival.

    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) or FOLFIRINOX, a current standard of care chemotherapy regimen. Devimistat given with mFFX did not significantly improve overall survival (HR=0.95, p=0.66). The median overall survival in the devimistat and mFFX arm was 11.1 months, compared to 11.7 months in the FOLFIRINOX arm.

    "These cancers are incredibly difficult to treat, with few to no effective treatments available, but Rafael took the risk because we will always fight for our patients," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "While we are disappointed by the outcomes of these well-designed and well-executed studies, we remain committed to furthering our research and development in cancer metabolism for the treatment of hard-to-treat cancers, as our other studies continue. I personally want to express my heartfelt appreciation to the patients, their loved ones, the researchers and principal investigators for their trust and support. I am also incredibly grateful to my team, who works tirelessly for the patients we treat."

    "Pancreatic cancer carries a high mortality rate and is extremely difficult to treat, but promising earlier clinical data encouraged us to move into this advanced phase trial with devimistat. While these are not the results we all hoped for, we will not give up, and we are hopeful that devimistat with its novel mechanism of action will demonstrate efficacy in other studies," said Philip Philip, M.D., Ph.D., F.R.C.P, principal investigator and medical oncologist at the Karmanos Cancer Institute at Wayne State University. "We are working diligently to further analyze the data and determine the best plan of action to further assess the drug's capabilities in the clinic."

    Devimistat is also being evaluated in a Phase 3 study, ARMADA 2000, in patients with relapsed or refractory acute myeloid leukemia (AML). Following a prespecified interim analysis, the independent data monitoring committee has recommended that the trial be stopped due to lack of efficacy.

    "Earlier clinical trial data demonstrated positive results, despite the challenges we face in treating AML," said Jorge Cortes, M.D., director of the Georgia Cancer Center at Augusta University. "We were all hoping for positive results, and while this trial did not meet our expectations, we will continue to test devimistat in other studies."

    The company will work with investigators to complete a full evaluation of the data on both trials.

    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 cellular stress and 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 side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patients' 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, 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 Pharmaceuticals, Inc.

    Rafael Pharmaceuticals is focused on the growing field of cancer metabolism. The company is developing a new, first-in-class 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 proliferate. Rafael Pharmaceuticals' lead compound, CPI-613® (devimistat), is an investigational anti-cancer agent that is being evaluated in ongoing and completed Phase 1, 2 and 3 clinical trials. The Company's investors include Rafael Holdings, Inc. (NYSE:RFL). On June 21, 2021, we announced that we have entered into a merger agreement with Rafael Holdings, Inc. to create a publicly traded late-stage clinical oncology company focused on cancer metabolism-based therapeutics. 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.



    Media Contact:
    Sharon Golubchik
     
    (609) 418-0058

    Primary Logo

    View Full Article Hide Full Article
  5. 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…

    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 proliferate. Rafael Pharmaceuticals' lead compound, CPI-613® (devimistat), is an investigational anti-cancer agent that is being evaluated in ongoing Phase 1, 2 and 3 clinical trials. The Company's investors include Rafael Holdings, Inc. (NYSE:RFL). On June 21, 2021, Rafael Pharmaceuticals announced that it has entered into a merger agreement with Rafael Holdings, Inc. to create a publicly traded late-stage clinical oncology company focused on cancer metabolism-based therapeutics. For more information, please visit www.rafaelpharma.com.

    Forward Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans regarding the further evaluation of clinical data; and the potential of our pipeline, including our internal cancer metabolism research programs. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of public health threats, including COVID-19, on our business and operations; we depend heavily on the success of Rafael Pharmaceuticals and the future success of its lead product candidate devimistat (CPI-613®), and clinical trials of the product candidate may not be successful; our pharmaceutical companies may not be able to develop any medicines of commercial value; our pharmaceutical companies may not be successful in their efforts to identify or discover potential product candidates; the manufacturing and manufacturing development of our products and product candidates present technological, logistical and regulatory risks, each of which may adversely affect our potential revenue; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; our product candidates may cause serious adverse side effects; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended July 31, 2021, and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

    Investor Relations

    Barbara Ryan

    om

    203-274-2825

    Media Contact

    Brandi Robinson



    862-226-9659



    Primary Logo

    View Full Article Hide Full Article
View All Rafael Holdings Inc. Class B News