Hematology

Agios Announces Phase 3 ENERGIZE Study of Mitapivat Met Primary Endpoint and Both Key Secondary Endpoints in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia

Retrieved on: 
Wednesday, January 3, 2024

CAMBRIDGE, Mass., Jan. 03, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism and pioneering therapies for rare diseases, today announced that the global Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia achieved its primary endpoint of hemoglobin response. Statistical significance was also achieved for both key secondary endpoints associated with change from baseline in FACIT-Fatigue Score and hemoglobin concentration.

Key Points: 
  • Statistical significance was also achieved for both key secondary endpoints associated with change from baseline in FACIT-Fatigue Score and hemoglobin concentration.
  • “The results of the Phase 3 ENERGIZE study underscore the potential of mitapivat to be a meaningful treatment option for adults with non-transfusion dependent alpha- or beta-thalassemia.
  • All subgroup analyses favored the mitapivat treatment arm compared to placebo,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios.
  • ET to review the ENERGIZE Phase 3 data and next steps for the Phase 3 ENERGIZE-T study.

Accarent Health Announces Affiliation with Children’s Hospital Los Angeles to Provide Virtual Cardiology, Oncology and Musculoskeletal Second Opinion Services

Retrieved on: 
Tuesday, January 2, 2024

Children’s Hospital Los Angeles (CHLA) provides exceptional family-centered care and life-saving treatments delivered by renowned physicians, surgeons, nurses and technicians in a child-friendly environment.

Key Points: 
  • Children’s Hospital Los Angeles (CHLA) provides exceptional family-centered care and life-saving treatments delivered by renowned physicians, surgeons, nurses and technicians in a child-friendly environment.
  • CHLA has also developed multiple surgical episodes of care for cardiovascular, musculoskeletal disorders, neurologic conditions and gene replacement therapy to support their second opinion services.
  • Accarent Health connects employers, patients, and plan administrators to a network of top-rated medical centers for superior, cost-effective care.
  • Employers interested in adopting a value-based health care system can learn more about Accarent Health at www.accarenthealth.com.

Human medicines European public assessment report (EPAR): Epysqli, Date of authorisation: 26/05/2023, Revision: 1, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024

Human medicines European public assessment report (EPAR): Epysqli, Date of authorisation: 26/05/2023, Revision: 1, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Epysqli, Date of authorisation: 26/05/2023, Revision: 1, Status: Authorised

Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Retrieved on: 
Tuesday, January 2, 2024

Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Key Points: 


Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Actinium Announces Acceptance of Five Abstracts for Presentation at the 2024 Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT® and CIBMTR®

Retrieved on: 
Tuesday, January 2, 2024

NEW YORK, Jan. 2, 2024 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of targeted radiotherapies, today announced that five abstracts have been accepted for presentation at the 2024 Tandem Meetings | Transplantation & Cellular Therapy (TCT) Meetings of ASTCT® (American Society for Transplantation and Cellular Therapy and CIBMTR®  (Center for International Blood and Marrow Transplant Research) being held February 21 – 24, 2024 in San Antonio, Texas. Two oral presentations will highlight results from the positive Phase 3 SIERRA trial of Iomab-B (131I-Apamistamab), representing 10 oral presentations of the SIERRA results since they were first reported at the 2023 Tandem Meetings.

Key Points: 
  • Two oral presentations will highlight results from the positive Phase 3 SIERRA trial of Iomab-B (131I-Apamistamab), representing 10 oral presentations of the SIERRA results since they were first reported at the 2023 Tandem Meetings.
  • Sandesh Seth, Actinium's Chairman and CEO, said, "We are excited to continue to build recognition for Iomab-B and the SIERRA data with the transplant community at the upcoming TCT meetings.
  • We are highly encouraged by the positive receptivity by the medical and scientific communities to date and look forward to building on this momentum in 2024."
  • The posters will be presented on Thursday, February 22, 2024, from 6:45 – 7:45 PM

In Vitro Diagnostics Analyzers Market Analysis Reveals Impressive Growth Forecast Through 2028 with Focus on Key Drivers and Trends - ResearchAndMarkets.com

Retrieved on: 
Friday, December 29, 2023

The increasing elderly population, growing incidence of chronic diseases, widening use of diagnostics for prevention and monitoring, and growing adoption of PoC testing are driving market growth.

Key Points: 
  • The increasing elderly population, growing incidence of chronic diseases, widening use of diagnostics for prevention and monitoring, and growing adoption of PoC testing are driving market growth.
  • Based on analyzer type, the market is categorized into immunoassay analyzers, clinical chemistry analyzers, hematology and hemostasis analyzers, molecular diagnostic analyzers and PoC analyzers.
  • The molecular analyzers segment, which held the third largest market share in 2022, is expected to show the highest CAGR, at 6.0%, during the forecast period.
  • The current report highlights the current and future market potential of in vitro diagnostics analyzers along with an analysis of the drivers, restraints, challenges, and emerging trends in the market.

Juventas Cell Therapy Announces the Approval of Its First Cell Therapy Product - Inaticabtagene Autoleucel by China NMPA

Retrieved on: 
Friday, November 10, 2023

Beijing, China and Tianjin, China and Shanghai, China--(Newsfile Corp. - November 10, 2023) - Juventas Cell Therapy Ltd. announces China National Medical Product Administration (NMPA) has approved its New Drug Application (NDA) for Inaticabtagene Autoleucel (CNCT19 Injection), a CD19-tarrgeted autologous chimeric antigen receptor (CAR)-T cell therapy for adult relapsed and refractory B-cell acute lymphoblastic leukemia (r/r B-ALL).

Key Points: 
  • The approval of Inaticabtagene Autoleucel by NMPA delivers a breakthrough clinical option adult r/r B-ALL patients.
  • Beijing, China and Tianjin, China and Shanghai, China--(Newsfile Corp. - November 10, 2023) - Juventas Cell Therapy Ltd. announces China National Medical Product Administration (NMPA) has approved its New Drug Application (NDA) for Inaticabtagene Autoleucel (CNCT19 Injection), a CD19-tarrgeted autologous chimeric antigen receptor (CAR)-T cell therapy for adult relapsed and refractory B-cell acute lymphoblastic leukemia (r/r B-ALL).
  • Inaticabtagene Autoleucel is a CD19 CAR-T cell therapy product, featured with a unique CD19 scFv (HI19a) and advanced manufacturing processes.
  • Inaticabtagene Autoleucel is the first proprietary CD19-targeted CAR-T product invented in China, and the first commercialized CAR-T cell therapy product for B-ALL in China.

Disc Medicine Announces Retirement of Brian MacDonald, MB, ChB, PhD as Chief Innovation Officer and Appointment as Chair of Scientific Advisory Board

Retrieved on: 
Wednesday, December 20, 2023

Dr. MacDonald plans to stay involved with Disc, transitioning from his current role to become Chair of Disc’s Scientific Advisory Board.

Key Points: 
  • Dr. MacDonald plans to stay involved with Disc, transitioning from his current role to become Chair of Disc’s Scientific Advisory Board.
  • “We are incredibly grateful for all of Brian’s exceptional contributions and leadership over the past 6 years.
  • “We are thrilled that Brian has agreed to remain involved with Disc and look forward to his continued leadership as part of the Scientific Advisory Board.
  • “I am proud of all Disc has accomplished thus far and am excited to remain a part of Disc’s continued growth towards becoming a leading hematology company.”

Harpoon Therapeutics Presents HPN217 Phase 1 Clinical Data in Relapsed/Refractory Multiple Myeloma (RRMM) at ASH 2023 and Announces Selection of Recommended Phase 2 Dose (RP2D)

Retrieved on: 
Tuesday, December 12, 2023

SOUTH SAN FRANCISCO, Calif., Dec. 11, 2023 (GLOBE NEWSWIRE) -- Harpoon Therapeutics, Inc. (Nasdaq: HARP), a clinical-stage immunotherapy company developing novel T cell engagers, today reported data from the Phase 1 study of HPN217 in patients with RRMM in an oral presentation at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego. Harpoon also announced the selection of 12 mg as the HPN217 RP2D.

Key Points: 
  • Harpoon also announced the selection of 12 mg as the HPN217 RP2D.
  • During the trial, 97 patients with RRMM who had received at least three prior therapies were enrolled across 15 dose escalation cohorts and three expansion regimens.
  • As of the data cut-off of October 17, 2023, the data demonstrated:
    Clinical activity across a wide dose range (2.15 mg to 24 mg).
  • The maximum tolerated dose (MTD) was not reached at the target dose using a step up approach.

NovalGen presents NVG-111 clinical data in hematological malignancies in an oral session and preclinical data for next-generation AR T cell engager NVG-222 at the 65th American Society of Hematology Annual Meeting

Retrieved on: 
Monday, December 11, 2023

LONDON, Dec. 10, 2023 (GLOBE NEWSWIRE) -- NovalGen Ltd (“NovalGen”), a pioneering clinical stage immunology company, shared promising findings from the NVG-111-101, a First in Human, Phase I clinical study (NCT04763083), in an oral presentation at the 65th American Society of Hematology Annual meeting, in San Diego, USA. The study showcased positive responses in 58% of evaluable patients with relapsed refractory chronic lymphocytic leukaemia (CLL) and mantle cell lymphoma (MCL) across both combination and monotherapy regimens, durable to 24 months.

Key Points: 
  • Noteworthy outcomes include a remarkable achievement of MRD4 negativity in peripheral blood, determined by flow cytometry, in 30% of CLL participants.
  • Furthermore, a complete metabolic response (CMR) by Lugano criteria was observed in one of two mantle cell lymphoma participants.
  • Such outcomes are particularly significant in diseases characterized by poor T cell function and are associated with improved progression free survival and overall survival.
  • The enhanced safety profile of this novel molecule allows for higher, more efficacious dosing, potentially leading to improved patient outcomes.