HSCT

MaaT Pharma Announces Positive Review from the DSMB on the Ongoing Phase 1 Clinical Trial Evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Thursday, February 29, 2024

The DSMB recommended that the trial continue without modifications.

Key Points: 
  • The DSMB recommended that the trial continue without modifications.
  • The DSMB, composed of 4 independent experts, including an ALS patient association representative, concluded that safety was good.
  • More precisely, it should be noted that no serious or severe adverse events were observed, and no infectious events could be related to MaaT033.
  • The preliminary results reinforce confidence in the safety of MaaT033, a drug candidate produced by combining the microbiota from multiple donors using a "pooling" process.

U.S. Food and Drug Administration Approves Opdivo® (nivolumab), in Combination with Cisplatin and Gemcitabine, for First-Line Treatment of Adult Patients with Unresectable or Metastatic Urothelial Carcinoma

Retrieved on: 
Thursday, March 7, 2024

Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Opdivo® (nivolumab), in combination with cisplatin and gemcitabine, for the first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), the most common type of bladder cancer.1,2 This approval is based on results from the Phase 3 CheckMate –901 trial which evaluated Opdivo in combination with cisplatin and gemcitabine followed by Opdivo monotherapy (n=304), compared to cisplatin-gemcitabine alone (n=304), for patients with previously untreated unresectable or metastatic UC.1,3 The primary efficacy endpoints were overall survival (OS) and progression-free survival (PFS) assessed by Blinded Independent Central Review (BICR).1

Key Points: 
  • Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Opdivo® (nivolumab), in combination with cisplatin and gemcitabine, for the first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), the most common type of bladder cancer.1,2 This approval is based on results from the Phase 3 CheckMate –901 trial which evaluated Opdivo in combination with cisplatin and gemcitabine followed by Opdivo monotherapy (n=304), compared to cisplatin-gemcitabine alone (n=304), for patients with previously untreated unresectable or metastatic UC.1,3 The primary efficacy endpoints were overall survival (OS) and progression-free survival (PFS) assessed by Blinded Independent Central Review (BICR).1
    In the trial, with a median follow-up of approximately 33 months, treatment with Opdivo in combination with cisplatin and gemcitabine reduced the risk of death by 22%, demonstrating a median OS of 21.7 months versus 18.9 months with cisplatin-gemcitabine alone (Hazard Ratio [HR] 0.78; 95% Confidence Interval [CI]: 0.63, 0.96; p=0.0171).1,4 Patients receiving Opdivo in combination with cisplatin and gemcitabine had their risk of disease progression or death reduced by 28%, with a median PFS of 7.9 months compared to 7.6 months with cisplatin-gemcitabine alone (HR 0.72; 95% CI: 0.59, 0.88; p=0.0012).1
    Additionally, in exploratory analyses, treatment with Opdivo in combination with cisplatin and gemcitabine resulted in an objective response rate (ORR) of 57.6% (n=175) (95% CI: 51.8, 63.2) versus 43.1% (n=131) (95% CI: 37.5, 48.9) with cisplatin-gemcitabine alone.1,4 The complete response (CR) rate and partial response (PR) rate seen in patients treated with Opdivo in combination with cisplatin and gemcitabine was 22% (n=66) and 36% (n=109), respectively, versus 12% (n=36) and 31% (n=95) with cisplatin-gemcitabine alone.1
    “This approval marks an important advancement in a historically difficult-to-treat setting, where there has been a need for new and differentiated first-line approaches that may offer patients a chance to live longer,”5 said Guru P. Sonpavde, MD, Medical Director of Genitourinary Oncology and the Phase I Clinical Research Unit and Christopher K. Glanz Chair for Bladder Cancer Research at the AdventHealth Cancer Institute, Orlando, Florida.
  • “Based on outcomes and the safety profile seen in the CheckMate -901 clinical trial, the approval of Opdivo in combination with cisplatin and gemcitabine has the potential to change how metastatic or unresectable UC is treated for certain patients and offers them new hope.”1
    Opdivo is associated with the following Warnings & Precautions: severe and fatal immune-mediated adverse reactions, including pneumonitis, colitis, hepatitis and hepatotoxicity, endocrinopathies, dermatologic adverse reactions, nephritis with renal dysfunction, other immune-mediated adverse reactions; infusion-related reactions; complications of allogeneic hematopoietic stem cell transplantation (HSCT); embryo-fetal toxicity; and increased mortality in patients with multiple myeloma when Opdivo is added to a thalidomide analogue and dexamethasone, which is not recommended outside of controlled clinical trials.
  • Please see Important Safety Information below.1
    “Bringing Opdivo to the first-line setting in UC with chemotherapy is the latest realization of our history of research and progress in immunotherapy, which has helped transform the treatment landscape for many cancers, including bladder cancer,”1,6 said Wendy Short Bartie, senior vice president and general manager, U.S. Hematology and Oncology at Bristol Myers Squibb.
  • “This milestone adds a meaningful expansion to our portfolio of Opdivo-based treatments in genitourinary cancers, where we now have offerings in UC spanning three indications across stages of disease and treatment needs.”1
    The FDA previously approved Opdivo for the adjuvant treatment of adult patients with UC who are at high risk of recurrence after undergoing radical resection of UC; it also previously approved Opdivo for the treatment of adult patients with locally advanced or metastatic UC who have had disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.1
    Bristol Myers Squibb’s supplemental Biologics License Application (sBLA) leading to today’s approval was granted Priority Review status by the FDA, and was approved under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to ensure that safe and effective treatments are available to patients as early as possible.7 The review was also conducted under the FDA’s Project Orbis initiative, which enables concurrent review by the health authorities in several other countries where the application remains under review.

Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome

Retrieved on: 
Monday, February 5, 2024

TOKYO, LONDON and BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the first patient has been randomized at the M Health Fairview Masonic Children’s Hospital in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares treatment with OTL-203 to standard of care with allogeneic hematopoietic stem cell transplant (HSCT), and is expected to enroll 40 MPS-IH patients at sites across the U.S. and Europe.

Key Points: 
  • Approximately 60 percent of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated.
  • Current treatment options for MPS-IH include allogeneic hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy (ERT), both of which have significant limitations.
  • One patient experienced an acute hypersensitivity reaction that was considered probably related to HSC gene therapy.
  • The viral vector integration profile was comparable with other Orchard Therapeutics lentiviral-based HSC gene therapy studies, and all participants had a stable and highly polyclonal repertoire.

Forge Biologics Announces Positive FBX-101 Clinical Trial Update in Patients with Krabbe Disease Identified by Newborn Screening Ahead of RUSP Vote

Retrieved on: 
Monday, January 29, 2024

Infantile Krabbe patients, often not diagnosed until after significant disease manifestations have occurred, typically die by the age of two if not treated by HSCT before symptoms are observed.

Key Points: 
  • Infantile Krabbe patients, often not diagnosed until after significant disease manifestations have occurred, typically die by the age of two if not treated by HSCT before symptoms are observed.
  • Previously published data have demonstrated that patients with Krabbe treated with HSCT demonstrate increased lifespan and stabilization of neurodegenerative disease in the central nervous system.
  • FBX-101, an investigational adeno-associated viral (AAV) gene therapy, has been designed to address the peripheral nerve disease not corrected by HSCT.
  • As a result, after a year of patient and foundation advocacy, Krabbe disease is again being voted on for potential inclusion on the RUSP.

Global Cord Blood & Tissue Banking Industry Report 2024 - The Vast Majority of the Global Cord Blood Market is Now Controlled by the World's 10 Largest Cord Blood Banking Operators - ResearchAndMarkets.com

Retrieved on: 
Monday, January 22, 2024

There are over 800,000 cord blood units stored in public cord blood banks and more than 6.75 million cord blood and tissue units stored within private banks worldwide.

Key Points: 
  • There are over 800,000 cord blood units stored in public cord blood banks and more than 6.75 million cord blood and tissue units stored within private banks worldwide.
  • Today, nearly all U.S. cord blood banks and approximately one-third of global cord blood banks offer cord tissue storage.
  • The vast majority of the global cord blood market is now controlled by the world's 10 largest cord blood banking operators.
  • It reveals the identities of companies offering cord blood storage, cord blood processing technologies, cord blood expansion technologies, and cord blood therapeutics on a global basis.

Regenerative Medicine Market Worth $49.0 Billion | MarketsandMarkets™.

Retrieved on: 
Friday, January 26, 2024

The cell therapy segment accounted for the largest share of the regenerative medicine market in 2022.

Key Points: 
  • The cell therapy segment accounted for the largest share of the regenerative medicine market in 2022.
  • In 2022, the musculoskeletal disorders segment accounted for the largest share of the global regenerative medicine market.
  • •  Market Development: Comprehensive information about lucrative markets – the report analyses the regenerative medicine market across varied regions.
  • •  Market Diversification: Exhaustive information about new products, untapped geographies, recent developments, and investments in the regenerative medicine market.

EQS-News: AiCuris Received 15 Million Euros Milestone Payment from Licensing Partner MSD Following EMA Approval of PREVYMIS® for Prevention of CMV Infection in High-Risk Adult Kidney Transplant Recipients

Retrieved on: 
Saturday, January 13, 2024

“Following the U.S. approval mid-2023, we are excited that our partner MSD now also received European approval for PREVYMIS® (letermovir) in its second indication, the prophylaxis of CMV infection in adult kidney transplanted patients at high risk.

Key Points: 
  • “Following the U.S. approval mid-2023, we are excited that our partner MSD now also received European approval for PREVYMIS® (letermovir) in its second indication, the prophylaxis of CMV infection in adult kidney transplanted patients at high risk.
  • In June 2023, the FDA and in December 2023 the EMA approved PREVYMIS® for prophylaxis of CMV disease in adult kidney transplant recipients at high risk.
  • Moreover, extended 200-day dosing for PREVYMIS® for CMV prophylaxis in adult HSCT recipients at risk for late CMV infection and disease was approved by the FDA in August 2023.
  • Approvals by national authorities was supported by a Phase 3, randomized, multicenter, double-blind, active comparator-controlled non-inferiority trial (P002, NCT03443869) in 589 adult kidney transplant recipients at high risk (CMV D+/R-).

Marker Therapeutics Announces Clinical Program Updates and Pipeline Prioritization

Retrieved on: 
Monday, January 8, 2024

HOUSTON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company focusing on developing next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumors, today announced a restructuring of its clinical programs and a strategic prioritization of its multi-tumor associated antigen (multiTAA)-specific T cell product pipeline. In addition, the Company reported a clinical update on the Phase 2 ARTEMIS study investigating MT-401, a multiTAA-specific T cell product, for the treatment of patients with acute myeloid leukemia (AML).

Key Points: 
  • The TACTAL study enrolled patients with Hodgkin’s and non-Hodgkin’s lymphoma and demonstrated clinical safety and efficacy with durable clinical responses for 6 years (Vasileiou et al, J Clin Oncol, 2021).
  • Today, Marker is providing a clinical update on the Phase 2 ARTEMIS clinical study (clinicaltrials.gov identifier: NCT04511130), and the direction it will pursue.
  • “We are encouraged by the clinical observations in patients with MRD in our AML study,” said Juan Vera, M.D., President and CEO of Marker Therapeutics.
  • Marker has secured non-dilutive funding to support the clinical investigation of a ready for use MT-401 product in patients with AML.

Molecular Partners Provides Updates at 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Sunday, January 7, 2024

53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced it will present a business overview and provide its 2024 outlook at the 42nd Annual J.P. Morgan Healthcare Conference.

Key Points: 
  • 53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced it will present a business overview and provide its 2024 outlook at the 42nd Annual J.P. Morgan Healthcare Conference.
  • Data were presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2023.
  • Molecular Partners continues to progress its RDT platform and portfolio of projects, both in-house and in partnership with Novartis.
  • In addition to these updates, Novartis has returned the rights to the ensovibep program, previously under investigation for the treatment of COVID-19, to Molecular Partners.

Human medicines European public assessment report (EPAR): Azacitidine Accord, azacitidine, Date of authorisation: 13/02/2020, Revision: 7, Status: Authorised

Retrieved on: 
Saturday, January 6, 2024

Human medicines European public assessment report (EPAR): Azacitidine Accord, azacitidine, Date of authorisation: 13/02/2020, Revision: 7, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Azacitidine Accord, azacitidine, Date of authorisation: 13/02/2020, Revision: 7, Status: Authorised