NPM1

Ryvu Announces Dosing of the First Patient in the RIVER-52 Phase II Study of RVU120 as a Monotherapy for the Treatment of Patients with Relapsed/Refractory AML and HR-MDS

Retrieved on: 
Wednesday, February 14, 2024

Ultimately, the study will expand to other EU and non-EU countries, covering up to 80 clinical sites globally.

Key Points: 
  • Ultimately, the study will expand to other EU and non-EU countries, covering up to 80 clinical sites globally.
  • RIVER-52 is a multicenter, open-label clinical trial designed to evaluate RVU120 in adult patients with r/r AML and HR-MDS, without alternative therapies.
  • RIVER-52 represents the second of the four planned RVU120 Phase II clinical studies scheduled for launch in H1 2024.
  • In addition to RIVER-52, Ryvu has already started patient treatment in the RIVER-81 study (evaluating RVU120 in combination with venetoclax for treating r/r AML patients).

Biomea Fusion Presents Achievement of Minimal Residual Disease Negativity (MRD-neg) in First Complete Responder from Ongoing Phase I Study (COVALENT-101) of BMF-219 in Patients with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) at the 2023 ASH

Retrieved on: 
Monday, December 11, 2023

Pharmacodynamic data from a case study of an AML patient containing NUP98-NSD1 mutation showed suppression of key leukemogenic genes (e.g.

Key Points: 
  • Pharmacodynamic data from a case study of an AML patient containing NUP98-NSD1 mutation showed suppression of key leukemogenic genes (e.g.
  • Initially, patients were enrolled agnostic to mutational status; subsequently, the study protocol was amended to enrich for patients with AML harboring menin-dependent mutations.
  • Biomea is planning to amend the dosing protocol to explore higher dosing levels in Arm B.
  • Dose escalation is to be followed by a dose optimization/expansion to determine the recommended phase 2 dose.

Ryvu Therapeutics Presents Data on RVU120 at the 2023 American Society of Hematology (ASH) Annual Meeting

Retrieved on: 
Monday, December 11, 2023

Treatment continues to show a favorable safety profile with 50-70% target engagement achieved at a dose of 250 mg.

Key Points: 
  • Treatment continues to show a favorable safety profile with 50-70% target engagement achieved at a dose of 250 mg.
  • The published clinical and non-clinical data strongly support RVU120’s Phase II development plans presented in October.
  • KRAKOW, Poland, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Ryvu Therapeutics [WSE:RVU], a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, presents clinical and preclinical data on RVU120, a selective CDK8/19 inhibitor, at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition, which is held on December 9-12, 2023, in San Diego, California.
  • “The results from the CLI120-001 (RIVER-51) study of RVU120 in patients with r/r-AML and HR-MDS continue to improve over time.

Aptose Tuspetinib Clinical Data Featured in Oral Presentation at the 2023 ASH Annual Meeting

Retrieved on: 
Saturday, December 9, 2023

Data were presented in an oral presentation today at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition by lead investigator Naval G. Daver, M.D., Professor, Director Leukemia Research Alliance Program, Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX.

Key Points: 
  • Data were presented in an oral presentation today at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition by lead investigator Naval G. Daver, M.D., Professor, Director Leukemia Research Alliance Program, Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX.
  • Tuspetinib is a once-daily, oral, precision targeted kinase inhibitor that suppresses select kinases that drive the proliferation of AML.
  • Dr. Daver reported data from more than 100 relapsed/refractory patients from multiple international clinical sites, who had failed prior therapy and then were treated with tuspetinib (TUS) as a single agent or tuspetinib in combination with venetoclax (TUS/VEN).
  • We look forward to reporting our next set of data in the first quarter of 2024.”

Global Menin Inhibitor Drugs Clinical Trials & Future Opportunity Insight 2023: A New Generation of Proteins that have Found Application as a Therapeutic Target in Several Prevalent Diseases - ResearchAndMarkets.com

Retrieved on: 
Thursday, December 21, 2023

The "Global Menin Inhibitor Drugs Clinical Trials & Future Opportunity Insight 2023" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Menin Inhibitor Drugs Clinical Trials & Future Opportunity Insight 2023" report has been added to ResearchAndMarkets.com's offering.
  • Menin represents a new generation of proteins that have found application as a therapeutic target in several prevalent diseases, especially cancer.
  • This has generated the need for development of menin inhibitors that can be used to treat these diseases.
  • Global Menin Inhibitor Drugs Clinical Trials & Future Opportunity Insight 2023 Report Highlights:
    Menin Inhibitors Drugs In Clinical Trials: > 10 Drugs
    Global Menin Inhibitors Drugs Clinical Trials By Company, Indication & Phase
    Menin Inhibitors Clinical Research Innovation Trends By Region: US, Europe & Canada

Biomea Fusion Announces Two Poster Presentations at Upcoming ASH Annual Meeting 2023

Retrieved on: 
Thursday, November 2, 2023

Both BMF-219 and BMF-500 were originated in-house with Biomea’s proprietary FUSION™ system platform, which discovers and designs next-generation covalent-binding small molecule product candidates.

Key Points: 
  • Both BMF-219 and BMF-500 were originated in-house with Biomea’s proprietary FUSION™ system platform, which discovers and designs next-generation covalent-binding small molecule product candidates.
  • Methods: Doses of BMF-219 are escalated independently for each indication, initially in single-subject cohorts followed by a “3 + 3” design.
  • A subsequent amendment introduced quotas for KMT2Ar (MLL1r), NPM1 and other known menin-dependent mutations: CEBP/A, MLL1-PTD, MN1, NUP98, NUP214, PICALM-AF10, SETBP1.
  • The study was initiated in July 2023 and will enroll ~110 participants at approximately 30 sites.

Ryvu Therapeutics Provides an Update on the Progress of RVU120 Phase I Studies in Patients with Solid Tumors and AML/HR-MDS, and Presents the Updated Development Plan for RVU120

Retrieved on: 
Monday, October 23, 2023

The total budget for the Phase II clinical development of RVU120 aligns with the estimates initially announced in the Ryvu Development Plans for 2022-2024.

Key Points: 
  • The total budget for the Phase II clinical development of RVU120 aligns with the estimates initially announced in the Ryvu Development Plans for 2022-2024.
  • The presented RVU120 development plan aligns with the budget outlined in Ryvu Development Plans for 2022-2024 and targets multi-billion-dollar market potential.
  • At the ESMO Congress 2023, Ryvu announced updated clinical Phase I data from Phase I/II study of RVU120 in relapsed/refractory metastatic advanced solid tumors.
  • Ryvu will host a webinar today (Monday, October 23) at 9:00 am CEST to discuss further RVU120 development plans.

Biomea Fusion Reports Second Quarter 2023 Financial Results and Corporate Highlights

Retrieved on: 
Monday, July 31, 2023

Our goal at Biomea is to develop a treatment that can halt or reverse disease progression in patients with type 1 and type 2 diabetes.

Key Points: 
  • Our goal at Biomea is to develop a treatment that can halt or reverse disease progression in patients with type 1 and type 2 diabetes.
  • We look forward to continuing to evaluate BMF-219’s proposed mechanism of action and its potential therapeutic impact as this study progresses.
  • BMF-500 is an investigational oral covalent inhibitor of FLT3, designed and developed in-house, and the second investigational compound discovered by Biomea’s FUSION™ System.
  • G&A expenses were $11.4 million for the six months ended June 30, 2023 compared to $9.9 million for the same period in 2022.

BMF-219 Induces Complete Responses in Target Acute Myeloid Leukemia (AML) Patient Population

Retrieved on: 
Monday, July 24, 2023

These relapsed/refractory patients had a range of prior therapies (1 to 8) and two complete responses (1 CR, 1 CRi) were observed within the first two 28-day treatment cycles with BMF-219.

Key Points: 
  • These relapsed/refractory patients had a range of prior therapies (1 to 8) and two complete responses (1 CR, 1 CRi) were observed within the first two 28-day treatment cycles with BMF-219.
  • Enrollment for Dose Level 5 has commenced to further optimize and explore the potential to improve upon these preliminary results.
  • Completion of the dose escalation for the acute leukemia cohort is anticipated later this year.
  • We are continuing to dose escalate and are looking forward to identifying the recommended Phase 2 dose within the next several months.”

Cepheid Receives CE Mark for Xpert® NPM1 Mutation

Retrieved on: 
Tuesday, May 30, 2023

SUNNYVALE, Calif., May 30, 2023 /PRNewswire/ -- Cepheid announced today that it has received the CE mark for Xpert NPM1 Mutation, a molecular in vitro diagnostic test for the quantification of mutant NPM1 mRNA transcripts (types A, B and D in exon 12) in peripheral blood specimens from patients with Acute Myeloid Leukemia (AML). The test utilizes automated real-time reverse transcription polymerase chain reaction (RT-PCR) and reports the percent ratio of mutant NPM1 to ABL1 endogenous control mRNA transcripts.

Key Points: 
  • The test utilizes automated real-time reverse transcription polymerase chain reaction (RT-PCR) and reports the percent ratio of mutant NPM1 to ABL1 endogenous control mRNA transcripts.
  • The European LeukemiaNet recommends quantitative molecular assessment of NPM1 by qPCR as part of minimal residual disease (MRD) monitoring of patients with NPM1-mutated AML1.
  • The mutations in the NPM1 gene lead to abnormal cytoplasmic localization of NPM1 and NPM1-interacting proteins, which means they're unable to carry out their normal cellular functions.
  • Xpert NPM1 Mutation strengthens Cepheid's hematology-oncology portfolio, which includes Xpert BCR-ABL Ultra and Xpert BCR-ABL Ultra p190, by offering an efficient workflow to measure NPM1 mRNA transcripts to improve care for oncology patients.