Trial of the century

Biophytis announces filing of an IND application with the US FDA for its phase 2 study in obesity

Retrieved on: 
Monday, June 17, 2024

The primary objective of the study is to assess improvements in muscle strength of lower limbs as measured by knee extension.

Key Points: 
  • The primary objective of the study is to assess improvements in muscle strength of lower limbs as measured by knee extension.
  • Multiple secondary endpoints will also be explored such as mobility (as measured by the 6-minute walk test) and body composition (fat mass, lean body mass).
  • Biophytis is also actively engaging in the process to broaden its research capacity by including European clinical centers.
  • He is a worldwide recognized medical expert in the field of obesity and is currently President-Elect of the US-based Obesity Society.

Advanced R&D capabilities and proven efficacy of our unique fast pain-relief mouth ulcer cream

Retrieved on: 
Monday, June 17, 2024

Our mouth ulcer cream is a testament to this commitment, formulated with a proprietary blend of clinically proven ingredients that deliver comprehensive relief and promote rapid healing.

Key Points: 
  • Our mouth ulcer cream is a testament to this commitment, formulated with a proprietary blend of clinically proven ingredients that deliver comprehensive relief and promote rapid healing.
  • A recent clinical study1, conducted by our esteemed team of researchers, underscores the efficacy of our mouth ulcer cream.
  • The clinical study concluded that our mouth ulcer cream offers a superior solution for those seeking rapid relief and effective healing.
  • "This study highlights the exceptional efficacy of our mouth ulcer cream and showcases our capability as a leading R&D company.

Editas Medicine Reports New Safety and Efficacy Data from the RUBY Trial of Reni-cel in 18 Patients with Sickle Cell Disease, Presented at the European Hematology Association (EHA) Annual Congress

Retrieved on: 
Friday, June 14, 2024

CAMBRIDGE, Mass., June 14, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced new safety and efficacy data in 18 patients living with sickle cell disease (SCD) treated with renizgamglogene autogedtemcel (reni-cel; formerly known as EDIT-301) in the Phase 1/2/3 RUBY clinical trial. Reni-cel, the first investigational AsCas12a gene-edited cell therapy medicine, is being studied in the RUBY trial as a potential one-time, durable medicine for people living with severe SCD. The data will be presented in an oral presentation at the European Hematology Association (EHA) Hybrid Congress in Madrid, Spain and via livestream, on Saturday, June 15 at 11:30 a.m. CEST (5:30 a.m. EDT).

Key Points: 
  • Reni-cel, the first investigational AsCas12a gene-edited cell therapy medicine, is being studied in the RUBY trial as a potential one-time, durable medicine for people living with severe SCD.
  • Since treatment with reni-cel, patients have been free of vaso-occlusive events (VOEs) (N=18) for up to 22.8 months of follow-up.
  • Patients in the RUBY trial underwent a median of 2.0 apheresis and mobilization cycles (min: 1.0, max: 4.0).
  • All patients in the RUBY trial showed sustained high levels of editing in the HBG1 and HBG2 promoter regions.

Editas Medicine Announces New Safety and Efficacy Data from the EdiTHAL Trial of Reni-cel in 7 Patients with Transfusion-dependent Beta Thalassemia, Presented at the European Hematology Association (EHA) Annual Congress

Retrieved on: 
Friday, June 14, 2024

CAMBRIDGE, Mass., June 14, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced new safety and efficacy data in 7 patients with transfusion-dependent beta thalassemia (TDT) treated with renizgamglogene autogedtemcel (reni-cel; formerly known as EDIT-301) in the Phase 1/2 EdiTHAL clinical trial. Reni-cel, the first investigational AsCas12a gene-edited cell therapy medicine, is being studied in the EdiTHAL trial as a potential one-time, durable gene editing medicine for people living with TDT. The data will be presented in a poster presentation at the European Hematology Association (EHA) Hybrid Congress in Madrid, Spain, on Friday, June 14 at 6:00 p.m. CEST (Noon EDT).

Key Points: 
  • Reni-cel, the first investigational AsCas12a gene-edited cell therapy medicine, is being studied in the EdiTHAL trial as a potential one-time, durable gene editing medicine for people living with TDT.
  • The data will be presented in a poster presentation at the European Hematology Association (EHA) Hybrid Congress in Madrid, Spain, on Friday, June 14 at 6:00 p.m. CEST (Noon EDT).
  • In the EdiTHAL trial to date, reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all patients (N=7).
  • All patients in the EdiTHAL trial showed sustained high levels of editing in the HBG1 and HBG2 promoter regions.

Samsung Bioepis Presents Post-hoc Analysis of Phase 3 Study for EPYSQLI™ (SB12; Eculizumab Biosimilar), at the European Hematology Association (EHA) Congress 2024

Retrieved on: 
Friday, June 14, 2024

The post-hoc analysis of SB12 pivotal Phase 3 study compared the proportion of patients remaining transfusion-free (“transfusion avoidance”) among those treated with SB12 or reference eculizumab (ECU), respectively.

Key Points: 
  • The post-hoc analysis of SB12 pivotal Phase 3 study compared the proportion of patients remaining transfusion-free (“transfusion avoidance”) among those treated with SB12 or reference eculizumab (ECU), respectively.
  • At Week 26, patients were switched to ECU or SB12, respectively, and received infusion until Week 50 (i.e., Period 1 followed by Period 2 from Week 26).
  • This efficacy analysis was conducted in pooled patients treated with SB12 or ECU in either Periods 1 or 2.
  • In March 2024, EPYSQLI was granted an indication expansion for the treatment of adults and children with atypical haemolytic uremic syndrome (aHUS).3

ElmediX Plans Series A Round Based on Strong Signals in a Clinical Study for Pancreatic Cancer Patients at the University Hospital Antwerp (Belgium)

Retrieved on: 
Friday, June 14, 2024

ElmediX is demonstrating strong signals of effectiveness of its new hyperthermic treatment in a clinical study for pancreatic cancer patients at the University Hospital Antwerp (UZA; Belgium) based on the CA19-9 platform.

Key Points: 
  • ElmediX is demonstrating strong signals of effectiveness of its new hyperthermic treatment in a clinical study for pancreatic cancer patients at the University Hospital Antwerp (UZA; Belgium) based on the CA19-9 platform.
  • The results of the American study add an extra dimension to the clinical study ElmediX is conducting at the University Hospital Antwerp (Belgium).
  • In pancreatic cancer patients, a decrease in CA 19-9 was recorded after a single treatment (up to 60%).
  • Based on these results, ElmediX is planning a Phase II study for pancreatic cancer patients.

NKGen Biotech Presents Updated Phase 1 Data on SNK02 Allogeneic NK Cell Therapy for Solid Tumors at the 6th Annual Allogeneic Cell Therapies Summit 2024

Retrieved on: 
Wednesday, June 12, 2024

Dr. Song also provided an update on the Company’s initial Phase 1 results using SNK02 to treat patients with advanced refractory solid tumors.

Key Points: 
  • Dr. Song also provided an update on the Company’s initial Phase 1 results using SNK02 to treat patients with advanced refractory solid tumors.
  • Dr. Song’s presentation explored the potential benefits of eliminating pre-treatment lymphodepletion in patients undergoing SNK02 therapy, aiming to safeguard immune function and aid in recovery.
  • The presentation also included a discussion on the results from the Company’s Phase 1 SNK02 clinical study in solid tumors previously disclosed in a publication at the 2024 American Society for Clinical Oncology annual meeting.
  • SNK02 was well-tolerated as a monotherapy and appears to have some clinical activity against pretreated solid tumors despite the lack of lymphodepletion.

Revive Therapeutics Provides Update on FDA Type C Meeting for Clinical Study of Bucillamine to Treat Long COVID

Retrieved on: 
Wednesday, June 12, 2024

In addition, the FDA provided valuable feedback on the appropriate design, study population, and safety and efficacy measures for assessing a therapeutic benefit in patients with Long COVID.

Key Points: 
  • In addition, the FDA provided valuable feedback on the appropriate design, study population, and safety and efficacy measures for assessing a therapeutic benefit in patients with Long COVID.
  • “We are pleased with the FDA’s recommendations for our proposed clinical study, which provides a pathway for the clinical development of Bucillamine as a potential treatment for Long COVID,” said Michael Frank, CEO of Revive.
  • Currently, the Company is exploring the use of Bucillamine as a potential treatment for Long COVID.
  • The Company is advancing the clinical development of Bucillamine by leveraging the published research and data from its previous Phase 3 clinical trial and aims to complete the regulatory and clinical package that includes a proposed clinical study for Long COVID, incorporating the recommendations from the FDA from the Type C meeting.

Junshi Biosciences Announces Phase 3 Study of Toripalimab Combined with Bevacizumab for the First-line Treatment of Advanced Hepatocellular Carcinoma Meets Primary Endpoint

Retrieved on: 
Wednesday, June 12, 2024

Junshi Biosciences plans to submit a supplemental new drug application (“NDA”) for this indication to the regulatory authorities in the near future.

Key Points: 
  • Junshi Biosciences plans to submit a supplemental new drug application (“NDA”) for this indication to the regulatory authorities in the near future.
  • According to the GLOBOCAN Report for 2022, there were 866,000 new cases and 759,000 deaths from liver cancer globally that year.
  • The HEPATORCH phase 3 clinical study evaluating toripalimab combined with bevacizumab achieved its primary endpoint, potentially offering a new treatment option with better survival benefits for advanced liver cancer patients.
  • In this study, toripalimab demonstrated its powerful synergy as a cornerstone of immuno-oncology, significantly improving survival rates for patients with advanced HCC.

KSQ Therapeutics Announces First Patient Dosed in Clinical Development Program for KSQ-001EX, a CRISPR/Cas9 Engineered Tumor Infiltrating Lymphocyte (eTIL®) Therapy

Retrieved on: 
Wednesday, June 12, 2024

KSQ's CRISPRomics® platform identified SOCS1 as a key gene inhibiting T cell growth, survival, and differentiation by negatively regulating cytokine signaling in the tumor microenvironment.

Key Points: 
  • KSQ's CRISPRomics® platform identified SOCS1 as a key gene inhibiting T cell growth, survival, and differentiation by negatively regulating cytokine signaling in the tumor microenvironment.
  • In preclinical studies, KSQ-001EX demonstrated enhanced anti-tumor activity, polyclonality, persistence, and memory formation in multiple solid tumor models compared to unmodified TIL.
  • We also saw robust anti-tumor activity in models insensitive to PD-1 inhibition,” said Qasim Rizvi, Chief Executive Officer of KSQ.
  • The primary objective of the Phase 1 portion of the trial is to evaluate the safety and tolerability of KSQ-001EX.