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Clinical Trial News

Savara Launches Expanded Access Program for Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis

  • Savara Inc. has initiated an Expanded Access Program (EAP) for molgramostim inhalation solution to treat autoimmune pulmonary alveolar proteinosis (aPAP) in select regions.
  • The FDA has reviewed and allowed the Savara Early Access Program to proceed, currently accepting requests from eligible patients in North America and Europe.
  • Savara plans to submit a Biologics License Application (BLA) to the FDA for molgramostim in aPAP in the first half of 2025, marking a key regulatory milestone.
  • Molgramostim has received multiple designations, including Orphan Drug, Fast Track, and Breakthrough Therapy, from regulatory agencies for aPAP treatment.

Molgramostim Nebulizer Solution Expanded Access Program Protocol

NCT06546098available
Savara Inc.

Pfizer Withdraws Oxbryta Globally Due to Safety Concerns; Jefferies Adjusts Price Target

  • Pfizer voluntarily withdrew Oxbryta, a sickle-cell anemia treatment, from all markets amid an EMA review due to safety concerns.
  • The decision was prompted by an imbalance between vaso-occlusive crises and fatal events observed in studies.
  • Jefferies has lowered its price target for Pfizer from $35 to $33, citing the removal of Oxbryta and GBT601 from their model.
  • The mechanism of action of Oxbryta is under scrutiny, leading to the removal of related assets from financial models pending further information.

Cardurion Pharmaceuticals Recognized as a Top Biotech for Cardiovascular Therapeutics

  • Cardurion Pharmaceuticals has been named one of the Endpoints 11 most promising private biotechnology companies of 2024, recognizing its innovative approach to cardiovascular disease treatment.
  • The company's clinical programs include CRD-750, a PDE9 inhibitor in Phase 2 trials for chronic heart failure, and CRD-4730, a CaMKII inhibitor in Phase 2 for catecholaminergic polymorphic ventricular tachycardia (CPVT).
  • Cardurion is focused on developing next-generation therapeutics targeting unmet needs in cardiovascular diseases, which remain a leading cause of death globally.
  • The recognition highlights Cardurion's strengths in science, leadership, investors, and strategy in developing novel cardiovascular therapeutics.

A Study to Assess Safety & Effectiveness of Tovinontrine in Chronic Heart Failure With Reduced Ejection Fraction (Cycle-1-REF)

NCT06215911Active, Not RecruitingPhase 2
Cardurion Pharmaceuticals, Inc.
Posted 2/13/2024

Effectiveness of CRD-740 in Heart Failure

NCT05409183TerminatedPhase 2
Cardurion Pharmaceuticals, Inc.
Posted 5/26/2022

A Phase 2 Clinical Study to Assess the Safety and Effectiveness of Tovinontrine in Patients With Chronic Heart Failure With Preserved Ejection Fraction

2023-508737-13-00Active, Not RecruitingPhase 2
Cardurion Pharmaceuticals Inc.
Posted 2/13/2024

Effectiveness of CRD-4730 in Participants With Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

NCT06005428TerminatedPhase 2
Cardurion Pharmaceuticals, Inc.
Posted 11/7/2023

ImPact Biotech's Padeliporfin VTP Shows Promise in Phase 3 UTUC Trial

  • ImPact Biotech's ENLIGHTED trial of Padeliporfin VTP for low-grade UTUC has reached 50% enrollment, marking a key milestone.
  • Interim results from the trial indicate a 77% complete response rate in response-evaluable patients after the induction treatment phase.
  • The ENLIGHTED trial is expected to complete enrollment by Q1 2025, with additional interim data anticipated in Q4 2024.
  • Padeliporfin VTP, administered intravenously with laser activation, is being evaluated as a potential novel treatment for UTUC.

ENdoluminal LIGHT ActivatED Treatment of Upper Tract Urothelial Cancer (ENLIGHTED) Study

NCT04620239RecruitingPhase 3
Steba Biotech S.A.
Posted 3/22/2021

Real-World Performance Analysis of GPRC5D Bispecific Therapies in Relapsed Multiple Myeloma

  • GPRC5D-targeted bispecific antibodies have emerged as a promising therapeutic approach for patients with relapsed/refractory multiple myeloma, showing significant clinical potential in real-world settings.
  • Real-world efficacy data demonstrates comparable outcomes to clinical trials, suggesting robust therapeutic potential across diverse patient populations outside controlled study environments.
  • Implementation challenges in community settings highlight the need for specialized monitoring protocols and resource optimization to maximize treatment benefits while managing safety concerns.

MHRA Approves Joenja (leniolisib) as First Treatment for Rare Immune Disease APDS

  • The MHRA has approved Joenja (leniolisib) as the first medicine for Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) in the UK.
  • APDS is a rare, inherited immune disorder affecting approximately 30-40 people in the UK, characterized by recurrent infections and abnormal immune function.
  • Leniolisib demonstrated a statistically significant reduction in lymph node size and normalization of immunophenotype in a 12-week placebo-controlled trial.
  • The approval was expedited through the International Recognition Procedure (IRP), leveraging prior FDA approval to provide quicker access to UK patients.

Adaptive Clinical Trials: Study Highlights Inconsistencies in Sample Size Reporting

  • A review of 265 adaptive clinical trials reveals that less than half explicitly report the operating characteristics of the adaptive design, impacting result interpretation.
  • While most trials (86.4%) clearly describe interim decision rules, replicability is a concern as 61.3% fail to specify if futility stopping rules are binding or non-binding.
  • Of completed trials, 44.7% enrolled the planned sample size, while 29.6% enrolled fewer and 25.7% enrolled more participants, with adaptations accounting for some deviations.
  • The maximum sample size was the most frequently reported metric, while the minimum sample size was directly stated in only 6.0% of trials, though inferable in 92.3%.

Reevaluation Of Systemic Early Neuromuscular Blockade

NCT02509078CompletedPhase 3
Massachusetts General Hospital
Posted 1/4/2016

Stem Cell Therapy Achieves Diabetes Reversal in World-First Clinical Case

  • A woman has achieved diabetes remission following stem cell therapy, marking the first reported instance of such a reversal using this approach.
  • The experimental treatment involved reprogramming the patient's own cells into pancreatic cells, which were then transplanted back into her body.
  • This breakthrough offers a potential new avenue for treating type 1 diabetes, reducing or eliminating the need for lifelong insulin injections.
  • Further research is needed to confirm the long-term efficacy and safety of this stem cell therapy for diabetes and to expand its applicability.

Dupixent Approved in China as First Biologic for COPD Patients

• The NMPA in China approved Dupixent (dupilumab) as an add-on maintenance treatment for adults with uncontrolled COPD with raised blood eosinophils. • Landmark Phase 3 trials (BOREAS and NOTUS) demonstrated Dupixent significantly reduced COPD exacerbations and improved lung function over 52 weeks. • Dupixent inhibits IL-4 and IL-13 pathways, addressing type-2 inflammation, and is administered via subcutaneous injection every other week. • This approval marks Dupixent's fourth indication in China, spanning respiratory and dermatological diseases, offering new hope for COPD patients.

PolTREG Advances CAR-Treg Therapy for Neuroinflammatory Diseases

  • PolTREG initiates preclinical study of CAR-Treg cells for neuroinflammatory diseases, including multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS).
  • The study will assess the safety and efficacy of the CAR-Treg cell therapy, with data expected by March 30, 2025, guiding clinical evaluation.
  • The CAR-Treg cells are derived from PolTREG’s PTG-007 Treg cells, which have demonstrated safety and efficacy in type-1 diabetes.
  • Following successful preclinical testing, PolTREG plans to commence first-in-human trials of the CAR-Treg candidate for MS and ALS.

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