Cytarabine

ClearPoint Neuro Congratulates Partner PTC Therapeutics on Receiving Positive CHMP Opinion for Gene Therapy to Treat AADC Deficiency

Retrieved on: 
Friday, May 20, 2022

The use of ClearPoint Neuros proprietary CE Marked SmartFlow Neuro Ventricular Cannula for minimally invasive infusion of the gene therapy is included in the submission for administration of Upstaza.

Key Points: 
  • The use of ClearPoint Neuros proprietary CE Marked SmartFlow Neuro Ventricular Cannula for minimally invasive infusion of the gene therapy is included in the submission for administration of Upstaza.
  • This is a significant milestone for PTC Therapeutics, for ClearPoint and for biologics researchers everywhere as it marks the very first positive CHMP recommendation for direct injection to the brain of a gene therapy to treat a severe, highly morbid and fatal neurological disorder, commented Joe Burnett, President and CEO of ClearPoint Neuro.
  • First, it provides a potential path for other therapies to follow and sets an important precedent for regulatory approval of direct administered gene therapies to the brain.
  • And second, we see this as a validation of ClearPoints biologics partnership strategy and our rigorous bench, preclinical and clinical experience.

Kronos Bio’s Investigational SYK Inhibitors Entospletinib and Lanraplenib to be Featured in Four Poster Presentations at EHA2022 Congress

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Thursday, May 12, 2022

SAN MATEO, Calif. and CAMBRIDGE, Mass., May 12, 2022 (GLOBE NEWSWIRE) -- Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer and other serious diseases, today announced that its two novel investigational spleen tyrosine kinase (SYK) inhibitors, entospletinib and lanraplenib, will be featured in poster presentations at the European Hematology Association (EHA) 2022 Congress, being held June 9-12 in Vienna.

Key Points: 
  • Data are anticipated in the second half of 2023 with a primary endpoint of measurable residual disease (MRD) negative complete response.
  • The company is also advancing lanraplenib, a next generation SYK inhibitor, as a potential treatment for patients with relapsed/refractory AML in combination with gilteritinib in a Phase 1b/2 study.
  • Kronos Bio has begun to open trial sites and plans to initiate dosing of the first patient in the second quarter.
  • Additional experiments demonstrated that entospletinib and lanraplenib could restore T-cell proliferation in a subset of AML samples from patients with dysfunctional T-cell responses.

Kronos Bio Reports Recent Business Progress and First-Quarter 2022 Financial Results

Retrieved on: 
Wednesday, May 4, 2022

SAN MATEO, Calif. and CAMBRIDGE, Mass., May 04, 2022 (GLOBE NEWSWIRE) -- Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer and other serious diseases, today reported recent business progress and first quarter 2022 financial results.

Key Points: 
  • Data are anticipated in the second half of 2023 with a primary endpoint of measurable residual disease (MRD) negative complete response.
  • Kronos Bio has implemented fiscal prudence measures, which provide expected cash runway into the second half of 2024, as announced earlier this year.
  • Kronos Bio announced the appointment of Dr. Elizabeth Olek, DO, MPH, as senior vice president, Clinical Development.
  • Kronos Bio is based in San Mateo, Calif., and has a research facility in Cambridge, Mass.

Rhizen Pharmaceuticals AG Presents Data on Its Differentiated PARP and DHODH Inhibitor Programs at AACR 2022

Retrieved on: 
Saturday, April 9, 2022

Rhizen Pharmaceuticals AG (Rhizen), a Switzerland-based privately held, clinical-stage oncology & inflammation-focussed biopharmaceutical company, announced the release of data on its differentiated next-generation clinical-stage PARP (Poly ADP-Ribose Polymerase) and DHODH (DiHydro Orotate DeHydrogenase) inhibitor programs at the American Association for Cancer Research (AACR) 2022 Annual Meeting.

Key Points: 
  • Rhizen Pharmaceuticals AG (Rhizen), a Switzerland-based privately held, clinical-stage oncology & inflammation-focussed biopharmaceutical company, announced the release of data on its differentiated next-generation clinical-stage PARP (Poly ADP-Ribose Polymerase) and DHODH (DiHydro Orotate DeHydrogenase) inhibitor programs at the American Association for Cancer Research (AACR) 2022 Annual Meeting.
  • Rhizens poster presentations describe the preclinical characterization and differentiated features of its novel PARP inhibitor (RP12146) and preclinical data supporting the broad positioning of its DHODH inhibitor (RP7214) in AML.
  • Rhizen had earlier indicated that its PARP program had demonstrated differentiated IND enabling preclinical safety.
  • The additional preclinical data being presented at AACR 2022 suggests that this differentiated safety may be due to the lower bone marrow distribution of RP12146 and concomitantly lower haematological toxicity.

Magenta Therapeutics Reports Fourth Quarter and Full-Year 2021 Financial Results and Recent Program Highlights

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Tuesday, March 8, 2022

Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines designed to bring the curative power of stem cell transplants to more patients, today reported financial results for the fourth quarter and full-year ended December 31, 2021, and recent program highlights.

Key Points: 
  • Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines designed to bring the curative power of stem cell transplants to more patients, today reported financial results for the fourth quarter and full-year ended December 31, 2021, and recent program highlights.
  • Research and Development Expenses: Research and development expenses were $13.1 million in the fourth quarter of 2021, compared to $12.3 million in the fourth quarter of 2020.
  • General and Administrative Expenses: General and administrative expenses were $7.0 million for the fourth quarter of 2021, compared to $6.8 million for the fourth quarter of 2020.
  • Net Loss: Net loss was $19.3 million for the fourth quarter of 2021, compared to net loss of $18.2 million for the fourth quarter of 2020.

Kronos Bio Reports Recent Business Progress and Fourth-Quarter and Full-Year 2021 Financial Results

Retrieved on: 
Thursday, February 24, 2022

SAN MATEO, Calif. and CAMBRIDGE, Mass., Feb. 24, 2022 (GLOBE NEWSWIRE) --  Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer and other serious diseases, today reported recent business progress and fourth-quarter and full-year 2021 financial results.

Key Points: 
  • Kronos Bio is continuing to enroll patients in the Phase 1/2 trial of KB-0742.
  • Kronos Bio reported positive initial data from the study last year and is continuing to dose escalate.
  • Kronos Bio expects to dose the first patient in a Phase 1b/2 clinical trial of lanraplenib in the first quarter of 2022.
  • Kronos Bio announced its multi-year collaboration with Tempus Labs, Inc., a leader in artificial intelligence and precision medicine, during the fourth quarter.

Moleculin Provides Annamycin AML Program Update and Outlines Next Steps to Advance Towards Late-Stage Development

Retrieved on: 
Monday, February 14, 2022

As such, our Annamycin AML program is now positioned to progress to a Phase 1/2 combination clinical trial in Europe as soon as possible.

Key Points: 
  • As such, our Annamycin AML program is now positioned to progress to a Phase 1/2 combination clinical trial in Europe as soon as possible.
  • In the fifth and final cohort, patients' daily dose was 240 mg/m2 with five patients receiving a full course of Annamycin.
  • The Company's lead program, Annamycin is a next-generation anthracycline designed to avoid multidrug resistance mechanisms with little to no cardiotoxicity.
  • Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases.

Global Acute Myeloid Leukaemia Pipeline Landscape Report 2021 - Comprehensive Insights for Approx 150+ Companies and 150+ Pipeline Drugs - ResearchAndMarkets.com

Retrieved on: 
Monday, January 17, 2022

This "Acute myeloid leukaemia - Pipeline Insight, 2021" report provides comprehensive insights about 150+ companies and 150+ pipeline drugs in Acute myeloid leukaemia pipeline landscape.

Key Points: 
  • This "Acute myeloid leukaemia - Pipeline Insight, 2021" report provides comprehensive insights about 150+ companies and 150+ pipeline drugs in Acute myeloid leukaemia pipeline landscape.
  • "Acute myeloid leukaemia - Pipeline Insight, 2021" report outlays comprehensive insights of present scenario and growth prospects across the indication.
  • A detailed picture of the Acute myeloid leukaemia pipeline landscape is provided which includes the disease overview and Acute myeloid leukaemia treatment guidelines.
  • The assessment part of the report embraces, in depth Acute myeloid leukaemia commercial assessment and clinical assessment of the pipeline products under development.

Biosight Reports Final Primary Endpoint Data from Phase 2b Study of Aspacytarabine (BST-236) for First-Line Acute Myeloid Leukemia Therapy

Retrieved on: 
Thursday, January 6, 2022

AIRPORT CITY, Israel, Jan. 06, 2022 (GLOBE NEWSWIRE) --  Biosight Ltd., a pharmaceutical development company developing innovative therapeutics for hematological malignancies and disorders, today announced final results for the primary study endpoint, complete remission (CR), from the Company’s Phase 2b clinical trial evaluating single-agent aspacytarabine (BST-236) as a first-line acute myeloid leukemia (AML) therapy for patients unfit for standard induction chemotherapy. These results, presented at the 2021 American Society of Hematology (ASH) Annual Meeting held in December, 2021, demonstrated efficacy, safety, and tolerability of BST-236 as a monotherapy in treatment of newly-diagnosed AML patients unfit for standard induction therapy.

Key Points: 
  • Key highlights from the poster presentation titled, Aspacytarabine (BST-236) as Monotherapy is Safe, Well-tolerated and Effective for the Treatment of Adults with Newly Diagnosed Acute Myeloid Leukemia Unfit for Intensive 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.
  • Aspacytarabine is currently being investigated as a single agent in a Phase 2b clinical trial, which recently completed enrollment and primary endpoint analysis, for the first-line treatment of AML.

Notable Presents Clinical Validation Data from Two Phase 2 Trials in Adult and Pediatric Acute Myelogenous Leukemia at the 63rd American Society of Hematology (ASH) Annual Meeting

Retrieved on: 
Thursday, December 16, 2021

These data were recently presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition.

Key Points: 
  • These data were recently presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition.
  • Data from the first clinical trial were presented in a poster entitled Ex Vivo Drug Sensitivity Assay Correlates with Clinical Response and Identifies Panobinostat and Bortezomib as a Potential Novel Drug Combination for Pediatric AML.
  • In a trial conducted at Texas Childrens Hospital, pediatric and young adult de novo AML patients were treated with ADE (Cytarabine, Daunorubicin, Etoposide) alone or in combination with Atovaquone.
  • These two clinical studies provide further validation of Notables predictive precision medicines platform in standard of care settings.