MDM4

Phase 1 Clinical Data of Milademetan Published in Journal of Clinical Oncology

Retrieved on: 
Monday, January 23, 2023
Bachelor of Medicine, Bachelor of Surgery, MANTRA, Journal, MD, Patient, DD, MDM4, CDK4, Journal of Clinical Oncology, Cytopenia, Biomarker, Therapy, Neoplasm, RAIN, Progression-free survival, RNA transfection, Doctor of Philosophy, MDM2, Pharmaceutical industry

NEWARK, Calif., Jan. 23, 2023 (GLOBE NEWSWIRE) -- Rain Oncology Inc. (NasdaqGS: RAIN), (“Rain”), a late-stage company developing precision oncology therapeutics with its lead product candidate, milademetan, an oral, small molecule inhibitor of the p53-MDM2 complex that reactivates p53, today announced the publication of a peer-reviewed article titled, “A First-in-Human Phase I Study of Milademetan, an MDM2 Inhibitor, in Patients with Advanced Liposarcoma, Solid Tumors or Lymphomas” in the Journal of Clinical Oncology. Phase 1 clinical data in the paper highlight the activity and tolerability using intermittent dosing of milademetan across a range of tumor types including dedifferentiated liposarcoma (DD LPS), which represented the largest proportion of patients enrolled in the study (n=53).

Key Points: 
  • Phase 1 clinical data in the paper highlight the activity and tolerability using intermittent dosing of milademetan across a range of tumor types including dedifferentiated liposarcoma (DD LPS), which represented the largest proportion of patients enrolled in the study (n=53).
  • “An intermittent dosing schedule (260 mg qd, 3/14 days) of our highly selective inhibitor of the p53-MDM2 complex, milademetan, resulted in favorable safety and clinical activity in the Phase 1 trial in DD LPS patients,” said Robert Doebele, MD, Ph.D., co-founder, president and chief scientific officer of Rain.
  • The preferred intermittent dosing schedule of milademetan (260 mg qd 3/14 days) mitigates dose-limiting hematologic adverse events while maintaining activity, leading to:
    Fewer dose reductions (21.1%; n=8) and dose interruptions (15.8%; n=6) compared with extended/continuous schedules (23.3%; n=16 and 34.8%; n=24, respectively).
  • Preliminary single-agent activity with milademetan in DD LPS prompted the ongoing, randomized Phase 3 MANTRA trial (NCT04979442), with topline data anticipated in the first quarter of 2023.

PMV Pharma Appoints p53 Pioneer Dr. Guillermina Lozano to Scientific Advisory Board

Retrieved on: 
Tuesday, February 16, 2021
Branches of biology, Proteins, Tumor suppressor genes, Mdm2, Oncogenes, P53, Tumor suppressor, MDM4, USP7, Mir-605 microRNA precursor family

"Gigis work in p53 biology has had a tremendous impact on our fundamental understanding of the p53 pathway and the consequences of p53 mutations in cancer, said David Mack, Ph.D., President and Chief Executive Officer of PMV Pharma.

Key Points: 
  • "Gigis work in p53 biology has had a tremendous impact on our fundamental understanding of the p53 pathway and the consequences of p53 mutations in cancer, said David Mack, Ph.D., President and Chief Executive Officer of PMV Pharma.
  • Dr. Guillermina (Gigi) Lozano is a geneticist recognized for her studies of the p53 tumor suppressor pathway, from characterizing p53 as a transcriptional activator to characterizing the physiological importance of Mdm2 and Mdm4 proteins as inhibitors of p53, and the consequences of p53 mutations on tumor development.
  • PMV Pharma is a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53 mutants.
  • Bringing together leaders in the field to exploit over four decades of p53 biology, PMV Pharma combines unique biological understanding with a pharmaceutical development focus.