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Clinical Trial News

Intellia's CRISPR Gene Therapy Shows 98% Reduction in HAE Attacks After Three Years

Gene EditingOrphan DrugRare Disease
  • Intellia Therapeutics reported three-year follow-up data showing lonvoguran ziclumeran achieved a 98% mean reduction in monthly hereditary angioedema attack rates across all 10 Phase 1 patients.
  • All patients remained attack-free and treatment-free for a median of 23 months, demonstrating the potential for lonvo-z to become the first one-time therapy for HAE patients.
  • The company's Phase 3 HAELO trial completed screening ahead of schedule, with results expected in the first half of 2026 and a planned U.S. launch in 2027.
  • The CRISPR-based therapy targets the KLKB1 gene to prevent HAE attacks and has received multiple regulatory designations including FDA Orphan Drug and RMAT status.

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

NCT06634420Active, Not RecruitingPhase 3
Intellia Therapeutics
Posted 1/15/2025

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCT05120830Active, Not RecruitingPhase 1
Intellia Therapeutics
Posted 12/10/2021

Adalimumab Biosimilars Show High Acceptance and Long-Term Durability in IBD Patients Across Age Groups

Monoclonal AntibodyReal World Evidence
  • A French observational study found 92% of adult IBD patients accepted switching from adalimumab reference product to biosimilars, with 71% remaining on biosimilar therapy at 12 months.
  • Real-world data from European pediatric IBD centers demonstrated high durability rates for adalimumab biosimilars, with 67% of patients continuing treatment after median 26-month follow-up.
  • Both studies confirm cost-effectiveness of adalimumab biosimilars, with pediatric study estimating savings of 5,030 Euro per patient per year compared to reference product.
  • Clinical remission rates remained stable or improved in both populations, with 85% of adult switchers and 72% of pediatric patients achieving remission at 12 months.

Socioeconomic Disparities Drive Worse Outcomes in Multiple Myeloma Patients Despite Treatment Advances

  • Patients with relapsed/refractory multiple myeloma from lower socioeconomic backgrounds report significantly worse quality of life and higher prevalence of clinically important symptoms that limit daily activities.
  • Multiple myeloma mortality rates remain nearly twice as high among patients from households earning less than $40,000 annually compared to those earning over $120,000, with rural patients also experiencing higher mortality rates.
  • Chronic kidney disease emerges as a significant comorbidity that increases risk of high-risk multiple myeloma diagnosis, advanced disease stage, and mortality in affected patients.

EHA 2025 Showcases Breakthrough Therapies for Hematologic Malignancies

  • The 2025 European Hematology Association Congress featured promising clinical trial results across multiple hematologic cancers, with novel trispecific and bispecific antibodies showing exceptional response rates in challenging patient populations.
  • JNJ-5332, a trispecific antibody targeting CD3, BCMA, and GPRC5D, achieved 70.4% complete response rates in BCMA/GPRC5D-naïve multiple myeloma patients, comparable to CAR-T therapy but as an off-the-shelf outpatient treatment.
  • The combination of talquetamab and teclistamab demonstrated a 78.9% overall response rate in multiple myeloma patients with extramedullary disease, a historically difficult-to-treat population with poor outcomes.
  • POLARGO trial results showed polatuzumab vedotin-based therapy reduced death risk by 40% in relapsed/refractory diffuse large B-cell lymphoma, offering new treatment options for transplant-ineligible patients.

A Study to Evaluate INCA033989 Administered as a Monotherapy or in Combination With Ruxolitinib in Participants With Myeloproliferative Neoplasms

NCT06034002RecruitingPhase 1
Incyte Corporation
Posted 12/4/2023

A Study of the Combination of Talquetamab and Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma

NCT04586426Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 12/15/2020

A Study to Evaluate the Safety and Efficacy of Polatuzumab Vedotin in Combination With Rituximab, Gemcitabine and Oxaliplatin Compared to Rituximab, Gemcitabine and Oxaliplatin Alone in Participants With Relapsed or Refractory Diffuse Large B-Cell Lymphoma

NCT04182204CompletedPhase 3
Hoffmann-La Roche
Posted 2/7/2020

Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance

NCT04285086Active, Not RecruitingPhase 3
PharmaEssentia
Posted 8/25/2020

A Study to Evaluate INCA033989 Administered as a Monotherapy or in Combination With Ruxolitinib in Participants With Myeloproliferative Neoplasms

NCT05936359RecruitingPhase 1
Incyte Corporation
Posted 9/25/2023

A Study of JNJ-79635322 in Participants With Relapsed or Refractory Multiple Myeloma or Previously Treated Amyloid Light-chain (AL) Amyloidosis

NCT05652335RecruitingPhase 1
Janssen Research & Development, LLC
Posted 11/22/2022

Biologic Therapies Transform Respiratory Care as Clinicians Embrace Earlier, Precision-Based Treatment Strategies

Precision MedicineDrug Approval
  • Biologic therapies including dupilumab, benralizumab, and tezepelumab have revolutionized asthma management by enabling biomarker-driven treatment strategies beyond traditional allergen or eosinophil-based models.
  • Dupilumab's 2024 FDA approval for eosinophilic COPD represents the first biologic therapy for this condition, marking a significant milestone in a field where treatment had previously stagnated.
  • Clinicians are increasingly adopting earlier biologic intervention strategies to minimize steroid exposure and prevent long-term adverse outcomes, with pulmonologists now managing biologics more independently.
  • The recognition of asthma and COPD as heterogeneous diseases with overlapping inflammatory pathways is driving a shift toward personalized medicine approaches guided by biomarkers like eosinophils and FeNO.

Sigachi Industries Secures Environmental Approval for Major API Manufacturing Facility in Andhra Pradesh

  • Sigachi Industries Limited received Terms of Reference approval from Andhra Pradesh's State Environment Impact Assessment Authority for a new 25.09-acre pharmaceutical manufacturing facility in Orvakal, Kurnool District.
  • The facility will manufacture bulk drugs, drug intermediates, and specialty chemicals, with Environmental Clearance process beginning July 15, 2025, and project development starting August 1, 2025.
  • The strategic expansion aims to enhance API manufacturing capacity for global markets and support pipeline growth in regulated and semi-regulated markets worldwide.
  • The project represents a significant milestone in Sigachi's journey toward vertical integration and aligns with India's "Make in India" initiative through sustainable manufacturing practices.

Syngene International's Bengaluru Facility Receives Clean FDA Inspection with NAI Status

  • Syngene International's Bengaluru-based manufacturing facility successfully passed a US FDA inspection with zero observations and no Form 483 issued.
  • The facility received "no action indicated" (NAI) status, demonstrating full compliance with FDA regulations for pharmaceutical manufacturing operations.
  • The company reported mixed Q4 2025 financial results with net sales rising 11.03% to Rs 1018 crore while net profit declined 2.81% to Rs 183.30 crore.
  • Syngene serves multiple sectors including pharmaceutical, biotechnology, nutrition, animal health, consumer goods, and specialty chemicals through integrated R&D and manufacturing services.

Twin Health Raises $53 Million to Scale AI-Powered Digital Twin Platform for Metabolic Disease Management

AI
  • Twin Health secured $53 million in funding led by Maj Invest to expand its AI digital twin platform for metabolic health management into Fortune 500 enterprises and health plans.
  • The company's platform creates personalized metabolic models using biomarker data and has demonstrated significant clinical outcomes, with over 90% of diabetic subjects achieving type 2 diabetes reversal in clinical trials.
  • Twin Health operates on a performance-based care model where clients only pay when members achieve measurable clinical results such as improved A1C levels or weight loss.
  • The technology addresses the growing use of GLP-1 medications by providing personalized pathways to reduce long-term dependence on costly diabetes drugs.

Ethniq Launches Patented Natural Antifungal Treatment Showing 90% Efficacy in Clinical Trials

  • Ethniq has launched Skin Revive, a patented steroid-free treatment for chronic fungal skin infections that demonstrated over 90% efficacy in clinical trials registered with CTRI.
  • The natural formulation combines almond extract, coconut oil, eucalyptus oil, and chamomile processed through special extraction methods and has received patents in India and internationally.
  • The treatment addresses a significant medical need as fungal infections affect up to 61% of the population in some Indian states, with conventional steroid-based treatments facing growing drug resistance concerns.
  • Developed by Dr. Sunita Kumari and AYUSH-certified, the product aims to eliminate root causes of fungal overgrowth rather than masking symptoms like standard antifungals.

Sun Pharma Names Kirti Ganorkar as Managing Director in Major Leadership Transition

  • Kirti Ganorkar, a 29-year Sun Pharma veteran, will become Managing Director on September 1, 2025, succeeding founder Dilip Shanghvi who transitions to Executive Chairman.
  • The appointment marks the culmination of Sun Pharma's structured succession planning process as the company reported ₹52,041 crore in sales for FY2025, with 9% growth and specialty products contributing $1.21 billion.
  • Ganorkar has led Sun Pharma's India Business since 2019 and played key roles in the company's specialty drug expansion, including securing rights for innovative products like Ilumya.
  • The leadership change comes as Sun Pharma operates across challenging global markets including the US and Europe while maintaining its position as India's largest drugmaker.

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