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FDA Approves CSL's ANDEMBRY for Hereditary Angioedema Prevention with Novel Factor XIIa Targeting

Drug ApprovalMonoclonal AntibodyRare Disease
  • The FDA approved ANDEMBRY (garadacimab-gxii), the first and only prophylactic HAE treatment targeting factor XIIa, offering once-monthly dosing for patients 12 years and older.
  • Phase 3 VANGUARD trial data showed ANDEMBRY reduced HAE attacks by a median of more than 99% and a least squares mean of 89.2% compared to placebo, with 62% of patients remaining attack-free.
  • ANDEMBRY represents a breakthrough approach by inhibiting the top of the HAE cascade through factor XIIa targeting, providing sustained protection with convenient subcutaneous self-injection in 15 seconds or less.
  • CSL will launch ANDEMBRY commercially immediately with availability before the end of June, expanding treatment options for the approximately 1 in 50,000 people affected by this rare genetic disorder.

CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks

NCT04656418CompletedPhase 3
CSL Behring
Posted 1/27/2021

Clarametyx's CMTX-101 Shows Promise in Cystic Fibrosis Infection Trial, Advances to Next Phase

Monoclonal Antibody
  • Clarametyx Biosciences announced positive interim results from its Phase 1b/2a trial of CMTX-101, an immune-enabling antibody therapy for cystic fibrosis-associated pulmonary infections.
  • The interim analysis of 21 participants demonstrated a reduction in Pseudomonas aeruginosa burden with both 5 and 30 mg/kg doses, while maintaining a favorable safety profile.
  • The Independent Data Monitoring Committee approved continuation of the trial without modifications, with full enrollment of approximately 41 participants expected by end of 2025.
  • CMTX-101 targets bacterial biofilms to enhance antibiotic effectiveness and immune response, representing a novel approach to treating chronic respiratory infections.

A Study to Evaluate the Safety of CMTX-101 in People with Cystic Fibrosis

NCT06159725RecruitingPhase 1
Clarametyx Biosciences, Inc.
Posted 6/24/2024

Solu Therapeutics Appoints Enda Moran as COO to Scale CyTAC Platform Operations

Monoclonal Antibody
  • Solu Therapeutics has appointed Enda Moran, PhD, MBA as Chief Operating Officer to lead operational functions as the company advances its Phase 1 clinical trial of STX-0712.
  • Dr. Moran brings over three decades of biomanufacturing experience from Pfizer, GSK, and other major pharmaceutical companies to support Solu's CyTAC and TicTAC platforms.
  • The appointment comes as Solu progresses STX-0712 through Phase 1 development for resistant/refractory chronic myelomonocytic leukemia and other hematologic malignancies.
  • Dr. Moran will oversee chemistry manufacturing and controls, quality, program management, and infrastructure development to support multiple clinical and regulatory milestones.

Debiopharm Initiates First-in-Human Trial of CD37-Targeted ADC Debio 1562M for Relapsed/Refractory AML

Monoclonal AntibodyOncologyTargeted Therapy
  • Debiopharm has dosed the first patient in a Phase 1/2 trial of Debio 1562M, a first-in-class CD37-targeted antibody-drug conjugate for relapsed/refractory acute myeloid leukemia patients.
  • The compound utilizes Debiopharm's proprietary Trifecta approach, combining naratuximab antibody, MultiLink™ linker technology, and a microtubule inhibitor payload to target CD37 antigens on leukemic cells.
  • AML represents a significant unmet medical need with only 32% five-year overall survival and median survival as low as 7 months in certain populations.
  • Preclinical studies demonstrated Debio 1562M showed anti-leukemic activity across all AML subtypes and superior activity compared to current standard-of-care therapies.

A Study to Assess the Safety, Tolerability, and Antileukemic Activity of Debio 1562M in Participants With Acute Myeloid Leukemia (AML)

NCT06969430RecruitingPhase 1
Debiopharm International SA
Posted 5/30/2025

Incyte's INCA033989 Shows 86% Response Rate in CALR-Mutant Blood Cancer Trial

Monoclonal AntibodyPrecision Medicine
  • Incyte's experimental monoclonal antibody INCA033989 demonstrated an 86% complete or partial hematologic response rate in patients with CALR-mutant myeloproliferative neoplasms receiving doses of 400 mg or higher.
  • The company announced a precision medicine collaboration with Qiagen to develop next-generation sequencing diagnostics for detecting CALR mutations, the second most common driver of MPNs.
  • Clinical data from dose-escalation studies showed 89% of evaluable patients had reduced mutant CALR variant allele frequency, with 21% achieving partial molecular response within three treatment cycles.
  • Stifel upgraded Incyte to 'Buy' rating with a $107 price target following the encouraging clinical results presented at the European Hematology Association congress.

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.

NICE Rejects First Alzheimer's Disease-Modifying Drugs for NHS Use Due to Cost-Effectiveness Concerns

Monoclonal Antibody
  • The National Institute for Health and Care Excellence (NICE) has refused to recommend lecanemab and donanemab for NHS use, denying access to over 70,000 eligible patients in England unless they can afford private treatment costing £60,000-80,000 annually.
  • Both drugs demonstrated clinical efficacy in trials, with lecanemab slowing cognitive decline by 27% over 18 months and donanemab reducing decline by 4-7 months by clearing toxic amyloid protein from the brain.
  • NICE determined the cost-effectiveness estimates are five to six times above acceptable thresholds for NHS resources, despite the drugs representing the first treatments proven to slow Alzheimer's disease progression.
  • The decision has drawn criticism from Alzheimer's Research UK, which warns it sends a damaging signal to the life sciences sector and undermines the UK's position as a leader in dementia research and innovation.

Serum Institute of India Partners with DNDi to Develop Affordable Dengue Monoclonal Antibody Treatment

Monoclonal Antibody
  • Serum Institute of India and DNDi have signed an MoU to co-develop a monoclonal antibody treatment for dengue that targets all four virus serotypes, focusing on affordability for low- and middle-income countries.
  • The collaboration will advance the drug candidate through Phase III clinical trials in India, Brazil, and other dengue-endemic countries, with SII having already completed pre-clinical and Phase I/II trials demonstrating safety and efficacy.
  • Dengue affects 3.9 billion people globally with cases more than doubling since 2021, yet no specific treatment currently exists for the disease.
  • The partnership leverages SII's manufacturing capabilities and DNDi's clinical trial leadership to address the urgent need for dengue therapeutics in vulnerable populations worldwide.

Vial Doses First Participants in Phase 1 Trial of Half-Life Extended Anti-TL1A Antibody for IBD Treatment

Monoclonal Antibody
  • Vial has successfully dosed the first healthy volunteers in a Phase 1 trial of VIAL-TL1A-HLE, a novel subcutaneous monoclonal antibody targeting TL1A for inflammatory bowel disease treatment.
  • The first-in-human, single-ascending dose trial will evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of the half-life extended antibody with interim data expected in H2 2025.
  • Preclinical studies demonstrate the antibody has potential for best-in-class dosing intervals compared to first and second-generation anti-TL1A candidates, offering improved patient convenience.
  • Vial plans to initiate a multi-region Phase 2 trial in IBD later in 2025 following the interim Phase 1 results.

Specialised Therapeutics Expands Incyte Partnership to Bring Two Novel Cancer Therapies to Asia-Pacific

Monoclonal AntibodyRare DiseaseOncology
  • Specialised Therapeutics has expanded its partnership with Incyte to include axatilimab and retifanlimab for distribution in Australia, New Zealand, and Singapore, with potential expansion to other Asia-Pacific countries.
  • Axatilimab, a first-in-class CSF-1R-blocking antibody approved by the FDA in August 2024, treats chronic graft-versus-host disease after failure of at least two prior systemic therapies.
  • Retifanlimab, a PD-1 inhibitor, is approved for treating squamous cell carcinoma of the anal canal and Merkel cell carcinoma, with Australia having the highest global incidence of the latter condition.
  • Both therapies are expected to undergo regulatory and reimbursement approval submissions in the region during 2025.

A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

NCT06388564RecruitingPhase 2
Incyte Corporation
Posted 10/11/2024

A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease

NCT06585774RecruitingPhase 3
Incyte Corporation
Posted 1/21/2025

A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)

NCT04710576Active, Not RecruitingPhase 2
Syndax Pharmaceuticals
Posted 3/4/2021

MAXPIRe: Study to Evaluate Axatilimab in Participants With Idiopathic Pulmonary Fibrosis (IPF)

NCT06132256RecruitingPhase 2
Syndax Pharmaceuticals
Posted 12/11/2023
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