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Clinical Trial News

PepGen's PGN-EDO51 Shows Promise in Duchenne Muscular Dystrophy Trial

• PepGen anticipates releasing preliminary data from its Phase II CONNECT1-EDO51 trial of PGN-EDO51 in mid-2024, offering a potential treatment for Duchenne muscular dystrophy (DMD). • The CONNECT1-EDO51 trial assesses the safety, exon 51 skipping, and dystrophin protein production in male patients with DMD amenable to exon 51 skipping. • PGN-EDO51 utilizes PepGen's Enhanced Delivery Oligonucleotide (EDO) technology to target the genetic root cause of DMD, a muscle-wasting disease. • PepGen is also advancing PGN-EDODM1 for myotonic dystrophy type 1 (DM1), with Phase I trial data expected this year, alongside preclinical DMD programs.

FDA Accepts Resubmission of NDA for Rivoceranib and Camrelizumab in Unresectable Hepatocellular Carcinoma

  • The FDA has accepted the resubmitted NDA for rivoceranib and camrelizumab as a first-line treatment for unresectable or metastatic hepatocellular carcinoma.
  • The resubmission includes data from the Phase 3 CARES-310 study, which showed a median overall survival of 23.8 months with the combination.
  • The FDA has set a PDUFA target action date of March 20, 2025, for the rivoceranib and camrelizumab combination in uHCC.
  • The CARES-310 study demonstrated consistent efficacy across patient subgroups, suggesting the combination's potential benefit for a global uHCC population.

A Study to Evaluate SHR-1210 in Combination With Apatinib as First-Line Therapy in Patients With Advanced HCC

NCT03764293CompletedPhase 3
Jiangsu HengRui Medicine Co., Ltd.
Posted 6/10/2019

Amplia Therapeutics' Narmafotinib Shows Promise in Pancreatic Cancer Trial, Triggering Expanded Enrollment

  • Amplia Therapeutics' Phase 2a trial of narmafotinib in advanced pancreatic cancer achieves a significant milestone with six confirmed partial responses.
  • The combination therapy of narmafotinib with gemcitabine and Abraxane® demonstrates sufficient activity to warrant continued enrollment in the ACCENT trial.
  • Recruitment will resume at trial sites in Australia and South Korea, aiming to complete enrollment of the remaining 24 patients by the end of Q1 2025.
  • Narmafotinib continues to be well-tolerated, with no significant safety concerns or dose reductions reported to date in the ongoing clinical trial.

AERIFY-1/2 Trials: Evaluating Itepekimab for Moderate-to-Severe COPD in Former Smokers

The AERIFY-1 and AERIFY-2 phase 3 trials aim to assess the efficacy and safety of itepekimab, an anti-IL-33 monoclonal antibody, in former smokers with moderate-to-severe COPD. These randomized, double-blind, placebo-controlled studies will evaluate the impact of itepekimab on reducing exacerbations and improving lung function over 52 weeks.

Study to Assess the Efficacy, Safety, and Tolerability of SAR440340/REGN3500/Itepekimab in Chronic Obstructive Pulmonary Disease (COPD)

NCT04751487Active, Not RecruitingPhase 3
Sanofi
Posted 2/12/2021

Proof-of-Concept Study to Assess the Efficacy, Safety and Tolerability of SAR440340 (Anti-IL-33 mAb) in Patients With Moderate-to-severe Chronic Obstructive Pulmonary Disease (COPD)

NCT03546907CompletedPhase 2
Sanofi
Posted 7/16/2018

Study to Assess the Efficacy, Safety, and Tolerability of SAR440340/REGN3500/Itepekimab in Chronic Obstructive Pulmonary Disease (COPD)

NCT04701983Active, Not RecruitingPhase 3
Sanofi
Posted 12/16/2020

Synergia Medical's NAO.VNS System Successfully Implanted in First-in-Human Epilepsy Study

  • Synergia Medical successfully implanted its NAO.VNS system in two patients with drug-resistant epilepsy as part of the AURORA study.
  • The first-in-human implantations occurred at Cliniques Universitaires Saint-Luc and UZ Gent, with both patients recovering fully and starting stimulation therapy.
  • The AURORA study will assess the safety of the NAO.VNS system over 24 months, with plans for a pivotal trial in Europe, the USA, and Canada.
  • NAO.VNS offers MRI compatibility, extended battery life, and enhanced cybersecurity through optoelectronic technology for personalized vagus nerve stimulation.

Singapore HSA Approves MSD's Winrevair (sotatercept) for Pulmonary Arterial Hypertension

  • The Health Sciences Authority (HSA) of Singapore has approved Winrevair (sotatercept) for adults with pulmonary arterial hypertension (PAH).
  • Winrevair is the first activin signaling inhibitor approved in Asia for PAH, marking a significant advancement in treatment options.
  • The approval is based on the Phase 3 STELLAR trial, which demonstrated improved exercise capacity and reduced risk of clinical worsening.
  • Winrevair is expected to be available in Singapore by Q1 2025, offering new hope for PAH patients in the region.

A Study of Sotatercept for the Treatment of Pulmonary Arterial Hypertension (MK-7962-003/A011-11)(STELLAR)

NCT04576988CompletedPhase 3
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Posted 1/25/2021

SGLT2 Inhibitors Not Cost-Effective for Type 2 Diabetes with Heart Failure in Thailand

  • A cost-utility analysis in Thailand found that adding SGLT2 inhibitors to standard care for type 2 diabetes patients with heart failure is not cost-effective at current prices.
  • The study suggests price reductions of up to 90% for some SGLT2 inhibitors would be needed to meet Thailand's cost-effectiveness threshold.
  • The analysis, using national health data, considered direct medical and non-medical costs from a societal perspective, unlike previous studies.
  • Canagliflozin showed the lowest incremental cost-effectiveness ratio, but its efficacy data relies on a post-hoc analysis with limited heart failure patients.

PromarkerEso Blood Test Shows 94% Accuracy in Esophageal Cancer Detection

  • Proteomics International's PromarkerEso blood test demonstrates 94% accuracy in identifying esophageal adenocarcinoma (EAC) in a clinical validation study.
  • The non-invasive blood test offers a potential solution to improve early detection of EAC, which often goes undetected until late stages.
  • PromarkerEso measures glycoprotein biomarkers to screen for EAC, showing promise for enhanced surveillance of at-risk populations and improved health outcomes.
  • With patents granted in key markets, Proteomics International plans to launch PromarkerEso in Australia in Q1 CY25, followed by other regions.

Cyclophosphamide and Trenimon Show Promise in Ovarian Carcinoma Treatment: Early In Vitro and Clinical Evidence

  • In vitro studies indicate that ovarian carcinomas exhibit varied responses to cytostatic agents, with cyclophosphamide and trenimon demonstrating notable effectiveness.
  • A controlled clinical trial explored the role of pre-therapeutic sensitivity tests in chemotherapy for advanced ovarian carcinomas.
  • Preliminary results suggest that patients treated individually based on test results had more favorable survival curves compared to those treated non-individually with trenimon.
  • The study highlights the potential of personalized chemotherapy approaches in improving outcomes for patients with advanced ovarian carcinomas.

Summit Therapeutics' Ivonescimab Shows Promising Results in Lung Cancer Trial, Co-CEO Becomes Billionaire

• Summit Therapeutics' shares surged following positive trial data of ivonescimab in non-small cell lung cancer, increasing market capitalization. • Ivonescimab demonstrated a 49% reduction in tumor progression risk compared to Merck's Keytruda in the trial presented at the World Conference on Lung Cancer. • Co-CEO Maky Zanganeh's stake in Summit Therapeutics has elevated her to billionaire status, recognizing her leadership and the drug's potential. • A global Phase 3 trial is planned to seek FDA approval for ivonescimab, with multiple ongoing trials in China for various cancer types.

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