MedPath

Tagged News

Hemab Therapeutics Advances Bleeding Disorder Pipeline with Promising Clinical Data at ISTH 2025

Monoclonal AntibodyOrphan Drug
  • Sutacimig (formerly HMB-001) demonstrates promising interim Phase 2 safety and efficacy results for Glanzmann thrombasthenia, receiving WHO International Non-Proprietary Name designation.
  • HMB-002 shows initial proof of mechanism clinical data in Von Willebrand disease patients through the ongoing VELORA Pioneer study, supported by comprehensive preclinical data.
  • Natural history studies reveal significant disease burden, with 88% of Glanzmann thrombasthenia patients reporting at least one bleed weekly and 34% requiring medical treatment.
  • Hemab will present 11 abstracts across multiple bleeding disorder programs at the International Society on Thrombosis and Haemostasis Congress in Washington, DC.

A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia

NCT06211634Active, Not RecruitingPhase 1
Hemab ApS
Posted 12/13/2022

A Study Assessing HMB-002 in Participants With Von Willebrand Disease

NCT06754852RecruitingPhase 1
Hemab ApS
Posted 2/6/2025

A Study of Bleeding and Treatment in Participants With Von Willebrand Disease

NCT06610201Recruiting
Hemab ApS
Posted 8/30/2024

FDA Approves Merck's ENFLONSIA for RSV Prevention in Infants with Single Weight-Independent Dose

Drug ApprovalMonoclonal Antibody
  • The U.S. FDA has approved Merck's ENFLONSIA (clesrovimab-cfor), a long-acting monoclonal antibody for preventing RSV lower respiratory tract disease in newborns and infants entering their first RSV season.
  • ENFLONSIA demonstrated a 60.5% reduction in RSV-associated medically attended lower respiratory infections and an 84.3% reduction in RSV-related hospitalizations compared to placebo in the pivotal CLEVER trial.
  • The therapy offers the first weight-independent dosing approach for RSV prevention, using a single 105 mg dose regardless of infant weight to provide protection through a typical 5-month RSV season.
  • Ordering is expected to begin in July 2025, with shipments delivered before the 2025-2026 RSV season, pending CDC Advisory Committee recommendations later this month.

Efficacy and Safety of Clesrovimab (MK-1654) in Infants (MK-1654-004)

NCT04767373CompletedPhase 2
Merck Sharp & Dohme LLC
Posted 4/7/2021

Clesrovimab (MK-1654) in Infants and Children at Increased Risk for Severe Respiratory Syncytial Virus (RSV) Disease (MK-1654-007)

NCT04938830CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 11/30/2021

Scottish Health Authority Rejects Alzheimer's Drug Donanemab Despite UK Regulatory Approval

Monoclonal Antibody
  • The Scottish Medicines Consortium rejected donanemab (Kisunla) for NHS Scotland use, citing uncertainty around the drug's modest clinical benefit despite UK regulatory approval in October.
  • This marks the second Alzheimer's drug rejection by Scottish authorities in three months, following a similar decision on lecanemab (Leqembi).
  • Alzheimer Scotland advocates are calling for a specialized Dementia Drugs Fund and reformed assessment processes, arguing current evaluation methods are inadequate for addressing the UK's leading cause of death.
  • Donanemab works by targeting amyloid protein buildup in the brain through antibody infusions, potentially slowing Alzheimer's progression by up to two years in some cases.

Axio BioPharma and Likarda Form Strategic Partnership to Integrate AI Manufacturing with Advanced Drug Delivery

Monoclonal Antibody
  • Axio BioPharma and Likarda announced a strategic partnership combining AI-powered protein manufacturing with advanced drug delivery technologies to accelerate biologic drug development timelines.
  • The collaboration integrates Likarda's Core-Shell Spherification® hydrogel technology for encapsulation and controlled release with Axio's discovery-to-GMP manufacturing capabilities.
  • The partnership aims to enhance workflows for monoclonal antibodies, bispecifics, and Fc-fusions while providing clients with more efficient and scalable paths to clinical manufacturing.
  • Both companies emphasize integrating delivery technologies earlier in the development process to improve stability, efficacy, and patient outcomes while reducing development friction.

Pharmacist-Led Interventions Generate $9 Million in Cancer Care Cost Savings Through Remote Clinical Reviews

Monoclonal Antibody
  • Clinical review pharmacists at The US Oncology Network identified 1,271 cost-saving interventions across 5,600 patients, with 1,180 accepted interventions generating nearly $9 million in total cost of care reductions.
  • The initiative focused on six primary strategies including pembrolizumab dose banding, monoclonal antibody dose rounding, and biosimilar therapeutic interchange, with drug costs representing 63% of patient total cost of care.
  • Each clinical review pharmacist position cost approximately $25,000 but delivered significant financial returns while streamlining medication protocols and saving provider time in value-based care models.
  • Research from University of California San Diego revealed that 9% of inpatient chemotherapy treatments were due to transportation or housing barriers, resulting in over $7 million in potentially avoidable hospital costs over five years.

High Placebo Response Rates Challenge Lupus Drug Development as Researchers Develop New Trial Strategies

Monoclonal Antibody
  • High placebo response rates of approximately 40% in systemic lupus erythematosus trials are obscuring the true efficacy of investigational biologics, as patients continue receiving powerful background medications like glucocorticoids and hydroxychloroquine.
  • Researchers have identified four core strategies to address the "placebo problem," including stabilizing background therapy, applying central expert review, controlling rescue therapy, and acknowledging evolving standard-of-care treatments.
  • New trial design innovations include enriching study populations with high-disease-activity patients, employing more stringent sustained endpoints, and implementing adaptive features to better detect drug efficacy signals.
  • Clinical practice approaches now focus on realistic patient expectations for biologics like belimumab and anifrolumab, emphasizing gradual benefit timelines of 3-4 months and steroid-sparing goals rather than miracle cures.

Pediatric Lupus Trial of Belimumab Plus Background Standard Therapy

NCT01649765Active, Not RecruitingPhase 2
GlaxoSmithKline
Posted 9/7/2012

A Study to Evaluate the Safety of Rituximab Retreatment in Subjects With Systemic Lupus Erythematosus

NCT00137969CompletedPhase 2
Genentech, Inc.
Posted 5/10/2005

A Study of Belimumab in Subjects With Systemic Lupus Erythematosus

NCT00410384CompletedPhase 3
Human Genome Sciences Inc.
Posted 12/1/2006

A Study of Belimumab in Subjects With Systemic Lupus Erythematosus (SLE)

NCT00424476CompletedPhase 3
Human Genome Sciences Inc.
Posted 5/1/2007

Novartis Raises Mid-Term Sales Outlook to 6% Growth Through 2029 on Pipeline Strength

Monoclonal Antibody
  • Novartis has increased its mid-term sales growth guidance to 6% or more through 2029, up from previous forecasts, driven by more than 30 high-value pipeline projects.
  • The company expects four upcoming launches to achieve multibillion-dollar peak sales, including oral BTK inhibitor remibrutinib, Lp(a) drug pelacarsen, gene therapy OAV101 IT, and anti-BAFF-R antibody ianalumab.
  • Eight currently marketed medicines have peak sales potential between $3 billion and $8 billion-plus, with raised projections for Cosentyx, Kisqali, Kesimpta, Pluvicto, and Leqvio.
  • More than 15 data readouts expected within the next two years could lead to regulatory filings, supporting the company's focused strategy following the Sandoz spinout.

PCI Pharma Services Unveils Advanced Sterile Manufacturing Facility to Boost Biologic Production Capacity

Monoclonal Antibody
  • PCI Pharma Services is launching a new 50,000-square-foot sterile fill-finish facility in Bedford, NH, expected to reach full GMP production by summer 2025.
  • The facility features high-speed integrated fillers capable of producing up to 300,000 vials at speeds of 400 per minute, with twin 430-square-foot lyophilizers and fully isolated containment systems.
  • This marks PCI's third high-throughput sterile fill-finish facility built since 2021, enhancing capacity for late-phase clinical and commercial biologic drugs including monoclonal antibodies and fusion proteins.
  • The company is also establishing a Development Center of Excellence at the Bedford campus, expected to be operational by early 2026, handling both small molecules and biologics including antibody-drug conjugates.

Nemluvio Shows Sustained Long-Term Efficacy in Atopic Dermatitis with Two-Year Extension Data

Monoclonal AntibodyDrug Approval
  • New two-year data from the ARCADIA long-term extension study demonstrate that Nemluvio (nemolizumab) maintains sustained and increased improvements in itch, skin lesions, sleep, and quality of life in patients with moderate-to-severe atopic dermatitis.
  • At week 104, more than 85% of evaluable patients achieved a 75% reduction in the Eczema Area and Severity Index (EASI), with approximately 85% achieving at least four-point improvement in itch scores.
  • The study reinforces Nemluvio's rapid onset of action at week 4 and demonstrates a consistent safety profile with no new safety signals identified during prolonged treatment up to two years.

Long-term Safety and Efficacy of Nemolizumab With Moderate-to-severe Atopic Dermatitis

NCT03989206Active, Not RecruitingPhase 3
Galderma R&D
Posted 12/30/2019

Efficacy & Safety of Nemolizumab in Subjects With Moderate-to-Severe Atopic Dermatitis

NCT03989349CompletedPhase 3
Galderma R&D
Posted 6/30/2019

A Long-term Study of Nemolizumab (CD14152) in Participants With Prurigo Nodularis (PN)

NCT04204616Active, Not RecruitingPhase 3
Galderma R&D
Posted 1/11/2021

Study to Assess the Efficacy and Safety of Nemolizumab (CD14152) in Participants With Prurigo Nodularis (PN)

NCT04501666CompletedPhase 3
Galderma R&D
Posted 9/11/2020

Efficacy and Safety of Nemolizumab in Subjects With Moderate-to-Severe Atopic Dermatitis

NCT03985943CompletedPhase 3
Galderma R&D
Posted 6/27/2019

A Study to Assess the Efficacy and Safety of Nemolizumab (CD14152) in Participants With Prurigo Nodularis (PN)

NCT04501679CompletedPhase 3
Galderma R&D
Posted 8/11/2020

Study of Dupilumab for the Treatment of Patients With Prurigo Nodularis, Inadequately Controlled on Topical Prescription Therapies or When Those Therapies Are Not Advisable (LIBERTY-PN PRIME)

NCT04183335CompletedPhase 3
Sanofi
Posted 12/12/2019

Study of Dupilumab for the Treatment of Patients With Prurigo Nodularis, Inadequately Controlled on Topical Prescription Therapies or When Those Therapies Are Not Advisable (PRIME2)

NCT04202679CompletedPhase 3
Sanofi
Posted 1/16/2020

Upstream Bio Presents Data Showing Verekitug's Superior Potency Through TSLP Receptor Targeting at EAACI Congress

Monoclonal Antibody
  • Upstream Bio presented pharmacology modeling data at the EAACI Congress demonstrating verekitug's greater potency compared to tezepelumab through TSLP receptor targeting rather than ligand targeting.
  • The modeling data showed complete and sustained inhibition of the TSLP/TSLPR complex with verekitug across various doses, potentially leading to greater reduction in lung inflammation biomarkers.
  • Verekitug is the only monoclonal antibody currently in clinical development targeting the TSLP receptor and is being evaluated in Phase 2 trials for severe asthma, CRSwNP, and COPD.
  • The company expects top-line Phase 2 data in CRSwNP in Q3 2025 and severe asthma data in the first half of 2026.

A Study to Investigate the Efficacy and Safety of Verekitug (UPB-101) in Participants With Chronic Rhinosinusitis With Nasal Polyps

2023-508231-31-00CompletedPhase 2
Upstream Bio Inc.
Posted 12/18/2023

A Study to Investigate the Efficacy and Safety of Verekitug (UPB-101) in Adult Participants With Severe Asthma (VALIANT)

NCT06196879Active, Not RecruitingPhase 2
Upstream Bio Inc.
Posted 2/27/2024

A Study to Assess the Efficacy and Safety of Verekitug in Participants With COPD

NCT06981078RecruitingPhase 2
Upstream Bio Inc.
Posted 7/2/2025

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.