American Society of Hematology

Prothena Reports Fourth Quarter and Full Year 2023 Financial Results, and Provides Financial Guidance and Business Highlights

Retrieved on: 
Thursday, February 15, 2024

In addition, the Company provided 2024 financial guidance and business highlights.

Key Points: 
  • In addition, the Company provided 2024 financial guidance and business highlights.
  • “2023 was a year of strong progress for Prothena as we advanced our protein dysregulation portfolio and moved closer to becoming a fully integrated commercial company.
  • Total revenue for the fourth quarter and full year of 2023 included BMS collaboration revenue of $0.3 million and $91.3 million, respectively.
  • As of December 31, 2023, Prothena had $621.0 million in cash, cash equivalents and restricted cash, and no debt.

Evaxion to Develop Tailored Novel Cancer Vaccines Based upon a New Untapped Source of AI-Discovered Targets

Retrieved on: 
Wednesday, January 24, 2024

The new treatment opportunity may broaden the applicability of cancer vaccines.

Key Points: 
  • The new treatment opportunity may broaden the applicability of cancer vaccines.
  • Through the new vaccine targets, ERVs, treating patients unresponsive to conventional cancer immunotherapy may become possible.
  • With the elevated focus on this groundbreaking therapeutic concept, Evaxion has initiated preclinical activities with a goal of generating Proof-of-Concept data by the second half of 2024.
  • The ERV cancer vaccine targets represent a promising breakthrough that could significantly broaden the horizons of cancer vaccine applicability and marks a significant step forward in our commitment to improving healthcare through innovative and AI-powered approaches.

Cogent Biosciences Announces Positive Data from Ongoing Phase 2 APEX Trial Evaluating Bezuclastinib in Patients with Advanced Systemic Mastocytosis (AdvSM)

Retrieved on: 
Monday, December 11, 2023

WALTHAM, Mass. and BOULDER, Colo., Dec. 11, 2023 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today reported positive data from Part 1 of the Company’s ongoing Phase 2 APEX clinical trial evaluating bezuclastinib in patients with advanced systemic mastocytosis (AdvSM) at the 65th American Society of Hematology (ASH 2023) Annual Meeting & Exposition taking place December 9-12, 2023 in San Diego, CA.

Key Points: 
  • “Advanced systemic mastocytosis is a serious and life-threatening disease,” said Pankit Vachhani, M.D., Associate Professor of Medicine, Division of Hematology and Oncology, University of Alabama at Birmingham.
  • Patients were enrolled with the following sub-types: seven patients with aggressive systemic mastocytosis (ASM), 23 patients with systemic mastocytosis with associated hematologic neoplasm (SM-AHN), and two patients with mast cell leukemia (MCL).
  • In addition, Cogent plans to present data from the completed SUMMIT Part 1 trial (1a and 1b) in the first quarter of 2024.
  • ET (5:00 a.m. PT) to discuss today’s APEX data and the SUMMIT data in NonAdvSM released on December 9.

Cogent Biosciences Announces Positive Initial Data from Phase 2 SUMMIT Trial Evaluating Bezuclastinib in Patients with Nonadvanced Systemic Mastocytosis (NonAdvSM)

Retrieved on: 
Saturday, December 9, 2023

Twenty patients in Part 1a were treated with either bezuclastinib or placebo plus best supportive care for all arms.

Key Points: 
  • Twenty patients in Part 1a were treated with either bezuclastinib or placebo plus best supportive care for all arms.
  • Patients were enrolled with the following sub-types: 18 patients with indolent systemic mastocytosis (ISM) and two patients with smoldering systemic mastocytosis (SSM).
  • In patients with completed questionnaires:
    By week 12, bezuclastinib patients showed a median best improvement of 37% on MC-QoL vs. 24% for placebo patients.
  • Data from Part 1 of the Phase 2 APEX clinical trial evaluating bezuclastinib in patients with advanced systemic mastocytosis (AdvSM) will be presented in a poster session at ASH on Monday, December 11, 2023 at ASH.

Ryvu Therapeutics Reports Third Quarter 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Wednesday, November 29, 2023

Ryvu’s PRMT5 program and synthetic lethality platform were highlighted with preclinical data recently presented at the 2023 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics.

Key Points: 
  • Ryvu’s PRMT5 program and synthetic lethality platform were highlighted with preclinical data recently presented at the 2023 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics.
  • KRAKOW, Poland, Nov. 29, 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, today reported financial results for the third quarter of 2023 and provided a corporate update.
  • “The third quarter and the last months of 2023 proved to be an exceptionally productive period for our company,” said Pawel Przewięźlikowski, CEO of Ryvu Therapeutics.
  • Cash Position – On November 24, 2023, Ryvu Therapeutics held $64.5M in cash, cash equivalents, and bonds, compared to $23.2M at the end of 2022.

CoRegen Appoints Edward J. Benz, M.D., to its Board of Directors

Retrieved on: 
Thursday, November 16, 2023

HOUSTON, Nov. 16, 2023 (GLOBE NEWSWIRE) -- CoRegen, a biopharmaceutical company pursuing novel treatments for patients impacted by some of the most aggressive forms of cancer, announced today the appointment of Edward J. Benz, M.D., to its Board of Directors.

Key Points: 
  • HOUSTON, Nov. 16, 2023 (GLOBE NEWSWIRE) -- CoRegen, a biopharmaceutical company pursuing novel treatments for patients impacted by some of the most aggressive forms of cancer, announced today the appointment of Edward J. Benz, M.D., to its Board of Directors.
  • “We are honored to welcome Dr. Benz to CoRegen's Board of Directors, a distinguished leader whose legacy at Dana-Farber Cancer Institute and profound impact on cancer research speak volumes,” said Steve Gorlin, executive chairman and chief executive officer of CoRegen.
  • "We are entering a transformative era in cancer research, and the addition of Dr. Benz to CoRegen's Board of Directors could not have come at a more opportune time.
  • Dr. Benz will be an invaluable asset as we plan to enter the clinic, building on the legacy he has established in cancer care.

Nkarta Reports Third Quarter 2023 Financial Results and Corporate Highlights

Retrieved on: 
Thursday, November 9, 2023

In June 2023, Nkarta reported updated clinical data from its Phase 1 clinical trial evaluating NKX101 in patients with relapsed or refractory (r/r) AML.

Key Points: 
  • In June 2023, Nkarta reported updated clinical data from its Phase 1 clinical trial evaluating NKX101 in patients with relapsed or refractory (r/r) AML.
  • In October 2023, Nkarta announced the opening of a new cohort in its Phase 1 study of NKX019 in r/r NHL.
  • As of September 30, 2023, Nkarta had cash, cash equivalents, restricted cash, and investments of $278.4 million.
  • Net loss was $25.6 million, or $0.52 per basic and diluted share, for the third quarter of 2023.

Electra Therapeutics Appoints Kathy Dong, PharmD, MBA, as President and Chief Executive Officer

Retrieved on: 
Monday, December 11, 2023

Electra Therapeutics, Inc. , a clinical stage biotechnology company developing antibody therapies against novel targets for immunological diseases and cancer, today announced the appointment of Kathy Dong, PharmD, MBA, as President and Chief Executive Officer.

Key Points: 
  • Electra Therapeutics, Inc. , a clinical stage biotechnology company developing antibody therapies against novel targets for immunological diseases and cancer, today announced the appointment of Kathy Dong, PharmD, MBA, as President and Chief Executive Officer.
  • View the full release here: https://www.businesswire.com/news/home/20231211224486/en/
    Electra President and CEO Kathy Dong PharmD, MBA (Photo: Business Wire)
    “We are excited to have Kathy take on the role of CEO of Electra.
  • Dr. Rosenthal will continue to serve as a member of Electra’s board of directors and as Chief Executive Officer of Star Therapeutics, the company that spun out Electra.
  • Previously, she was Chief Operating Officer of Star Therapeutics since 2019, where she held strategic and operational responsibilities across the Star portfolio, including building Electra Therapeutics and Vega Therapeutics from discovery stage into the clinic.

Cimeio Therapeutics Presents Data for its CD45 Universal Heme ADC at ASH

Retrieved on: 
Tuesday, December 12, 2023

Cimeio Therapeutics, the leading biotechnology company in the field of epitope shielding, presented data for its CD45 and CD33 programs during this weekend’s American Society of Hematology (ASH) meeting in San Diego.

Key Points: 
  • Cimeio Therapeutics, the leading biotechnology company in the field of epitope shielding, presented data for its CD45 and CD33 programs during this weekend’s American Society of Hematology (ASH) meeting in San Diego.
  • The two studies provide further evidence that epitope editing allows for the development of powerful immunotherapies that would not be safe to administer without first protecting healthy cells.
  • After just two doses of CIM053-ADC, all mice were cancer-free while the healthy hematopoietic cells were unaffected.
  • This study demonstrates the potential of CD45-targeted ADC therapy for patients with hematologic malignancies.

Be Bio Announces New Preclinical Data Demonstrating its Novel Engineered B Cell Medicine (BCM), BE-101, Produces Active and Sustained Levels of Factor IX for the Treatment of Hemophilia B​

Retrieved on: 
Sunday, December 10, 2023

“We are excited by these findings as we advance this novel BCM into clinical development to address enduring unmet medical needs for people living with hemophilia B.

Key Points: 
  • “We are excited by these findings as we advance this novel BCM into clinical development to address enduring unmet medical needs for people living with hemophilia B.
  • Many people living with hemophilia B still experience bleeding episodes, irreversible joint damage and pain with current extended-release factor IX biologic therapy.
  • “Our goal is to create a new, potentially transformative treatment option that will provide people with hemophilia B the freedom to live a normal life, free of limitations from hemophilia B.
  • The data confirmed the expected biodistribution of FIX-expressing BCMs in bone marrow tissue, where they engraft stably over time.