Orphagen Pharmaceuticals Presents Data on Downstream Targets of SF-1 Antagonist OR-449 at ENDO 2022

SAN DIEGO–(BUSINESS WIRE)–Orphagen Pharmaceuticals, an early-stage biopharmaceutical company focused on developing and commercializing novel therapies for cancer and other serious diseases with significant unmet need, today announced that additional data from its preclinical program to develop OR-449 for the treatment of adrenocortical cancer (ACC) was presented at the 104th Annual Meeting of the Endocrine Society … [Read more…]

Genmab Announces Late-Breaking Phase 2 Trial Results of Investigational Epcoritamab (DuoBody®-CD3xCD20) in Relapsed/Refractory Large B-cell Lymphoma (LBCL) Patients Presented at European Hematology Association (EHA) Presidential Symposium

Epcoritamab demonstrated clinically meaningful efficacy in challenging to treat, highly refractory LBCL patients, including patients previously treated with chimeric antigen receptor (CAR) T-cell therapy Total patient population achieved overall response rate (ORR) of 63 percent and complete response (CR) of 39 percent; CAR T-naïve patients achieved 69 percent ORR and 42 percent CR; patients previously … [Read more…]

Vertex and CRISPR Therapeutics Present New Data on More Patients With Longer Follow-Up Treated With exagamglogene autotemcel (exa-cel) at the 2022 European Hematology Association (EHA) Congress

– Data from 75 patients with transfusion-dependent beta thalassemia or severe sickle cell disease with follow-up of up to 37.2 months continue to demonstrate that exa-cel has the potential to be a one-time functional cure – – Safety profile generally consistent with myeloablative conditioning and autologous stem cell transplant – BOSTON & ZUG, Switzerland & … [Read more…]

Quizartinib Plus Chemotherapy Significantly Improved Overall Survival Compared to Chemotherapy in Patients with Newly Diagnosed FLT3-ITD Positive Acute Myeloid Leukemia

Doubling of median overall survival seen in QuANTUM-First results presented at EHA Presidential Symposium Data support potential of quizartinib as targeted therapy for aggressive FLT3-ITD subtype of AML TOKYO & BASKING RIDGE, N.J.–(BUSINESS WIRE)–Positive results from the global pivotal QuANTUM-First phase 3 trial of Daiichi Sankyo’s (TSE:5468) quizartinib combined with standard induction and consolidation chemotherapy … [Read more…]

CANbridge CAN106 Phase 1 Data Presented at the European Hematology Association 2022 Congress

Rapid and potent C5 inhibition achieved with complete and sustained blockage of complement by a single dose of CAN106 Data to be presented at the 14th International Conference on Complement Therapeutics BEIJING & CAMBRIDGE, Mass.–(BUSINESS WIRE)–CANbridge Pharmaceuticals Inc. (HKEX:1228) , a China and US-based global biopharmaceutical company committed to the research, development and commercialization of … [Read more…]

Galderma Announces Positive Results in Two Phase III Studies for a Novel Liquid Formulation botulinumtoxinA (RelabotulinumtoxinA) for the Treatment of Glabellar Lines (frown) and Lateral Canthal Lines (crow’s Feet)

Both studies met their primary endpoints with significantly higher response than placebo after one month for both frown lines and crow’s feet Results showed rapid onset of action with median response within two days (39% saw results on Day 1 for frown lines and 34% on Day 1 for crow’s feet) Results also demonstrated long … [Read more…]

EarliTec Diagnostics Receives FDA 510(k) Clearance for the EarliPoint Evaluation for Autism Spectrum Disorder

First FDA-cleared tool to assist clinicians in diagnosing and assessing autism spectrum disorder (ASD) in children as young as 16 months old ATLANTA–(BUSINESS WIRE)–EarliTec Diagnostics, Inc. (“EarliTec”), a digital health company developing novel diagnostic and therapeutic products for children with autism spectrum disorder (ASD) and early childhood vulnerabilities, today announced U.S. Food and Drug Administration … [Read more…]

Advarra, Leading Provider of Life Sciences R&D Compliance and Technology Solutions, Secures Major Investment from Blackstone and CPP Investments

Investment will support next growth phase in Advarra’s journey to make clinical research safer, smarter, and faster COLUMBIA, Md.–(BUSINESS WIRE)–Advarra, a leading provider of regulatory, quality, and compliance solutions and clinical trial technologies in the life sciences sector, today announced that private equity funds managed by Blackstone (“Blackstone”) and Canada Pension Plan Investment Board (“CPP … [Read more…]

FDA Advisory Committee Unanimously Supports beti-cel Gene Therapy for People with beta-thalassemia Who Require Regular Red Blood Cell Transfusions

Current standard of care relies on regular red blood cell transfusions and iron management that carry the risk of progressive multi-organ damage and increased risk of morbidity and mortality If approved, beti-cel will be the first potentially curative gene therapy option for people with beta-thalassemia who require regular red blood cell transfusions and the first … [Read more…]

Global Nephropathic Cystinosis Market Report to 2032 – Insights, Epidemiology and Forecasts – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Nephropathic Cystinosis – Market Insight, Epidemiology and Market Forecast -2032” report has been added to ResearchAndMarkets.com’s offering. This report delivers an in-depth understanding of the Nephropathic Cystinosis, historical and forecasted epidemiology as well as the Nephropathic Cystinosis market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. … [Read more…]