Devimistat

ERYTECH Announces First Patient Enrolled in a Phase 1 Investigator Sponsored Trial of Eryaspase in First-Line Pancreatic Cancer

Thursday, January 14, 2021 - 9:30pm

The trial will enroll approximately 18 patients who have received no prior chemotherapy for the treatment of locally advanced or metastatic pancreatic cancer.

Key Points: 
  • The trial will enroll approximately 18 patients who have received no prior chemotherapy for the treatment of locally advanced or metastatic pancreatic cancer.
  • FOLFIRINOX is one of the most commonly utilized first-line chemotherapy regimens for the treatment of pancreatic cancer.
  • Pancreatic cancer is a disease in which malignant (cancer) cells are found in the tissues of the pancreas.
  • Eryaspase is in Phase 3 clinical development for the treatment of second-line pancreatic cancer and in Phase 2 for the treatment of triple-negative breast cancer.

GT Biopharma Announces Eighth Patient Begins Treatment Of GTB-3550

Tuesday, January 12, 2021 - 3:00pm

The GTB-3550 TriKE clinical trial commenced patient enrollment in February 2020 for the treatment of relapsed/refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (HR-MDS).

Key Points: 
  • The GTB-3550 TriKE clinical trial commenced patient enrollment in February 2020 for the treatment of relapsed/refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (HR-MDS).
  • The only treatment with curative intent for a majority of elderly HR-MDS or relapsed/refractory AML patients is allogeneic hematopoietic stem cell transplant (HSCT).
  • GT Biopharma, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of immuno-oncology therapeutic products based our proprietary TriKE NK cell engager platform.
  • GT Biopharma has an exclusive worldwide license agreement with the University of Minnesota to further develop and commercialize therapies using TriKE technology.

APL-106 (uproleselan) Granted Breakthrough Therapy Designation in China for the Treatment of Acute Myeloid Leukemia

Thursday, January 7, 2021 - 11:10am

The BTD is part of the revised Drug Registration Regulation that became effective in July 2020 in China.

Key Points: 
  • The BTD is part of the revised Drug Registration Regulation that became effective in July 2020 in China.
  • In 2017, the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to uproleselan for treatment of adults with relapsed or refractory AML.
  • Acute Myeloid Leukemia (AML) is a cancer of the blood and bone marrow.
  • Rivipansel, a pan-selectin antagonist, is being explored for use in treatment of acute VOC in sickle cell disease.

Moleculin Announces Annamycin Receives FDA Orphan Drug Designation for Soft Tissue Sarcomas

Tuesday, December 29, 2020 - 12:30pm

HOUSTON, Dec. 29, 2020 /PRNewswire/ -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Annamycin for treatment of soft tissue sarcomas.

Key Points: 
  • HOUSTON, Dec. 29, 2020 /PRNewswire/ -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Annamycin for treatment of soft tissue sarcomas.
  • "This is now the second Orphan Drug designation for Annamycin, as Annamycin previously received ODD for the treatment of relapsed or refractory acute myeloid leukemia," commented Walter Klemp, Chairman and CEO of Moleculin.
  • Moleculin is also engaged in preclinical development of additional drug candidates, including other Immune/Transcription Modulators, as well as WP1122 and related compounds capable of Metabolism/Glycosylation Inhibition.
  • Forward-looking statements in this press release include, without limitation, the ability of Moleculin to receive the benefits of the Orphan Drug designation, some of which require FDA approval of Annamycin for the Orphan Drug indication, and the ability of Annamycin to demonstrate safety and efficacy in patients.

Rafael Pharmaceuticals Receives FDA Fast Track Designation for CPI-613® (devimistat) for the Treatment of Acute Myeloid Leukemia (AML)

Tuesday, December 15, 2020 - 2:00pm

While one of the most common types of leukemia in adults, AML accounts for only 1% of all cancers .

Key Points: 
  • While one of the most common types of leukemia in adults, AML accounts for only 1% of all cancers .
  • This announcement comes on the heels of the Company receiving Fast Track designation for devimistat for the treatment of metastatic pancreatic cancer , in November.
  • The EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.
  • Devimistat has been granted orphan drug status by the FDA for the treatment of pancreatic cancer, acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and Burkitts,peripheral T-cell lymphomas and soft tissue sarcoma.

Biosight Presents Updated New Clinical Data from Ongoing Phase 2b Study of Aspacytarabine (BST-236) at the 2020 ASH Annual Meeting

Monday, December 7, 2020 - 1:00pm

A Phase 2b study is ongoing to confirm the promising results obtained in a Phase 1/2a study of aspacytarabine as a single-agent first-line AML therapy.

Key Points: 
  • A Phase 2b study is ongoing to confirm the promising results obtained in a Phase 1/2a study of aspacytarabine as a single-agent first-line AML therapy.
  • For more information regarding the Phase 2b clinical study of BST-236, please visit www.clinicaltrials.gov .
  • Biosight is a private Phase 2 clinical stage biotech company developing innovative therapeutics for hematological malignancies and disorders.
  • A Phase 2 study in relapsed/refractory AML and myelodysplastic syndrome (MDS) will be launched in 2020 under a collaboration agreement recently signed with the European cooperative group, GFM.

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

Tuesday, November 10, 2020 - 2:00pm

Receiving Fast Track designation is a significant milestone in our fight against pancreatic cancer, said Sanjeev Luther, President and CEO of Rafael.

Key Points: 
  • Receiving Fast Track designation is a significant milestone in our fight against pancreatic cancer, said Sanjeev Luther, President and CEO of Rafael.
  • 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.
  • We have remained hopeful throughout our pancreatic cancer trials, and now with Fast Track designation, our optimism is further fueled.

FDA Grants Rafael Pharmaceuticals Orphan Drug Designation for CPI-613® (devimistat) for Treatment of Soft Tissue Sarcoma

Thursday, October 29, 2020 - 1:30pm

CRANBURY, N.J., Oct. 29, 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) granted Orphan Drug Designation for CPI-613 (devimistat) for the treatment of soft tissue sarcoma.

Key Points: 
  • CRANBURY, N.J., Oct. 29, 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) granted Orphan Drug Designation for CPI-613 (devimistat) for the treatment of soft tissue sarcoma.
  • There are more than 50 different types of soft tissue sarcomas, and clear cell is ranked among the rarest .
  • There is a significant unmet need in treatment for soft tissue sarcoma, said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals.
  • In fact, Rafael is one of the only companies working on a treatment for clear cell sarcoma.

A New Phase 1 Study Began to Evaluate Rafael Pharmaceuticals’ Lead Compound CPI-613®️ (devimistat) for Patients With Pancreatic Cancer in Japan

Tuesday, October 20, 2020 - 2:00pm

The multicenter, open-label study is evaluating the efficacy and safety of Rafaels lead compound, CPI-613 (devimistat), labeled as ONO-7912 in Japan, in combination with modified FOLFIRINOX (mFFX) in patients with pancreatic cancer refractory or intolerance to chemotherapy including gemcitabine.

Key Points: 
  • The multicenter, open-label study is evaluating the efficacy and safety of Rafaels lead compound, CPI-613 (devimistat), labeled as ONO-7912 in Japan, in combination with modified FOLFIRINOX (mFFX) in patients with pancreatic cancer refractory or intolerance to chemotherapy including gemcitabine.
  • This announcement comes on the heels of Rafael reaching its target enrollment of 500 patients in its global Phase 3 trial .
  • Sadly, in Japan, patients with pancreatic cancer have the lowest survival rates.
  • In June 2019, the Company announced an out-licensing agreement with Japan-based Ono Pharmaceutical to further develop and commercialize devimistat.

Pancreatic Cancer Action Network Announces Groundbreaking Clinical Trial Platform Transforming Development Of Treatment Options For World's Toughest Cancer

Tuesday, October 13, 2020 - 2:00pm

LOS ANGELES, Oct. 13, 2020 /PRNewswire/ --The Pancreatic Cancer Action Network (PanCAN) announced today the launch of Precision PromiseSM, the first of its kind adaptive clinical trial platform for pancreatic cancer patients.

Key Points: 
  • LOS ANGELES, Oct. 13, 2020 /PRNewswire/ --The Pancreatic Cancer Action Network (PanCAN) announced today the launch of Precision PromiseSM, the first of its kind adaptive clinical trial platform for pancreatic cancer patients.
  • PanCAN's novel clinical trial platform is designed to enable the development of new treatments more efficiently than standard pancreatic cancer trials by testing multiple experimental therapies at the same time.
  • Through Precision Promise, metastatic pancreatic cancer patients may have the opportunity to receive both first- and second-line treatment options in one clinical trial.
  • The Pancreatic Cancer Action Network (PanCAN) is dedicated to fighting the world's toughest cancer.