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Clinical Trial News

7 Hills Pharma Doses First Patient in Alintegimod Phase 1b/2a Trial for Solid Tumors

  • 7 Hills Pharma has dosed the first patient in a Phase 1b/2a clinical trial of alintegimod, an integrin-targeted agonist, for solid tumors resistant to immune checkpoint inhibitors.
  • The trial (NCT06362369) is supported by a $13.4 million award from the Cancer Prevention and Research Institute of Texas and a $2.0 million award from the National Cancer Institute.
  • Alintegimod is designed to enhance T cell activation and trafficking by selectively activating the integrins LFA-1 and VLA-4, potentially improving the effectiveness of immunotherapies.
  • Preclinical models have demonstrated that alintegimod can improve the effectiveness of immune checkpoint inhibitors, with a prior Phase I trial showing oral bioavailability and a clean safety profile.

A Study of Oral 7HP349 (Alintegimod) in Combination With Ipilimumab Followed by Nivolumab Monotherapy

NCT06362369RecruitingPhase 1
7 Hills Pharma, LLC
Posted 8/23/2024

Vaxcyte's Pneumococcal Vaccine Candidate Outperforms Pfizer's Prevnar 20 in Early Trial

  • Vaxcyte's shares surged by 36% following the release of promising data on its pneumococcal vaccine candidate, VAX-24, which aims to rival Pfizer's Prevnar franchise.
  • In a preliminary trial involving 1,015 healthy adults aged 50 and older, Vaxcyte's VAX-24 demonstrated superior performance compared to Pfizer's Prevnar 20.
  • The positive results have propelled Vaxcyte's market capitalization to $12 billion, marking a significant milestone in the company's pursuit of a competitive pneumococcal vaccine.
  • Vaxcyte's VAX-24 is designed to improve upon existing pneumococcal vaccines, potentially offering enhanced protection against pneumococcal disease.

Roswell Park Launches Phase 1 Trial of 'Armored CAR' T-Cell Therapy Targeting CD19+ Hematologic Malignancies

  • Roswell Park Comprehensive Cancer Center is conducting a phase 1 trial of CD19/28z armored CAR T-cell therapy for relapsed or refractory hematologic malignancies.
  • The novel therapy enhances T cells with a CAR and a gene stimulating interleukin-12 production, potentially improving outcomes compared to existing CAR T therapies.
  • The trial (NCT06343376) aims to recruit 36 patients with various recurrent or refractory CD19+ lymphomas and leukemias to assess safety and optimal dosing.
  • This enhanced immunotherapy, developed in collaboration with Memorial Sloan Kettering, will have T cells manufactured in Roswell Park’s GMP facility.

Genetically Engineered Cells (EGFRt/19-28z/IL-12 CAR T Cells) for the Treatment of Relapsed or Refractory CD19+ Hematologic Malignancies

NCT06343376TerminatedPhase 1
Roswell Park Cancer Institute
Posted 11/26/2024

Spectral Medical's Tigris Trial Nears Completion with Strong Enrollment

• Spectral Medical's Tigris trial, evaluating Polymyxin B Hemoperfusion (PMX) for endotoxemia and septic shock, has enrolled 132 patients as of September 2024. • Enrollment in 2024 has significantly outpaced 2023, indicating strong momentum in the Phase 3 study. • With only 18 patients remaining to reach the target enrollment, Spectral anticipates completing the Tigris trial by the end of 2024. • The Tigris trial is a confirmatory study of PMX in addition to standard care vs standard care alone and is designed as a 2:1 randomized trial of 150 patients using Bayesian statistics.

Dyne Therapeutics' DYNE-251 Shows Promising Dystrophin Expression and Functional Improvements in Duchenne Muscular Dystrophy Trial

  • DYNE-251 demonstrated a mean absolute dystrophin expression of 3.71% of normal, over 10-fold higher than eteplirsen, in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping.
  • Clinical data from the DELIVER trial indicated meaningful improvements in motor function, including the Stride Velocity 95th Centile, meeting clinically important benchmarks defined by the European Medicines Agency.
  • The investigational therapy exhibited a favorable safety profile, with most treatment-emergent adverse events being mild or moderate, supporting its potential for long-term use.
  • Dyne Therapeutics is initiating registrational cohorts in the DELIVER trial and plans to provide an update on the path to registration for DYNE-251 by the end of 2024.

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

NCT05481879RecruitingPhase 1
Dyne Therapeutics
Posted 9/5/2022

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

NCT05524883Active, Not RecruitingPhase 1
Dyne Therapeutics
Posted 8/12/2022

JCR Pharmaceuticals' JR-441 Receives Clearance for Phase I MPS IIIA Trial in Japan

  • JCR Pharmaceuticals has completed the regulatory review by Japan's PMDA for a Phase I study of JR-441 in patients with mucopolysaccharidosis type IIIA (MPS IIIA).
  • JR-441, utilizing JCR's J-Brain Cargo® technology, aims to address the neurological symptoms of MPS IIIA by crossing the blood-brain barrier.
  • The drug has already been granted orphan drug designation by both the European Commission and the U.S. FDA for the treatment of MPS IIIA.
  • A Phase I/II clinical trial of JR-441 is currently ongoing in Germany, with the newly approved trial set to commence in Japan.

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

NCT06095388Active, Not RecruitingPhase 1
JCR Pharmaceuticals Co., Ltd.
Posted 10/4/2023

ApoA-I Infusions Show Promise in Reducing Cardiovascular Events Post-MI, Especially in Patients with Lower LDL-C

  • AEGIS-II trial reveals that infusions of apolipoprotein A-I (ApoA-I) may reduce cardiovascular events following acute myocardial infarction (MI).
  • The study indicates a more pronounced benefit of ApoA-I infusions in patients with lower baseline LDL-cholesterol levels.
  • Findings suggest ApoA-I infusions could offer a novel therapeutic approach to improve outcomes after MI, particularly for those not benefiting from standard lipid-lowering therapies.
  • Further research is warranted to confirm these results and optimize the use of ApoA-I infusions in post-MI management.

GC Biopharma and Hanmi Pharmaceutical's Fabry Disease Treatment LA-GLA Receives FDA IND Clearance

  • GC Biopharma and Hanmi Pharmaceutical received FDA IND clearance for Phase 1/2 clinical trial of LA-GLA (GC1134A/HM15421), a novel Fabry disease treatment.
  • LA-GLA is designed as a once-monthly subcutaneous injection, offering improved convenience compared to current bi-weekly intravenous enzyme replacement therapies.
  • Preclinical studies suggest LA-GLA enhances kidney function and shows superior efficacy in managing vascular and peripheral nerve disorders related to Fabry disease.
  • LA-GLA has been granted Orphan Drug Designation by the U.S. FDA, highlighting its potential to address unmet needs in Fabry disease treatment.

Recursion's AI-Derived Drug REC-994 Shows Promise in Phase 2 Trial for Cerebral Cavernous Malformation

  • Recursion Pharmaceuticals' REC-994 met the primary safety endpoint in a Phase 2 trial for cerebral cavernous malformation (CCM).
  • Secondary endpoints suggest a reduction in lesion volume and hemosiderin ring size at the 400mg dose compared to placebo.
  • The oral drug REC-994 has the potential to address the unmet needs of CCM patients who currently have limited treatment options.
  • Recursion plans to engage with the FDA to discuss subsequent studies and present trial data at an upcoming medical conference.

Semaglutide Weight-Loss Drugs Not Linked to Increased Suicide Risk in New Study

  • A new study analyzing data from over 3,500 participants found no increased risk of depression or suicidal thoughts/behaviors with semaglutide use.
  • The research focused on individuals without pre-existing major mental health disorders, providing reassurance for this patient population.
  • Semaglutide, marketed as Ozempic and Wegovy, has gained popularity for weight management and diabetes treatment, raising concerns about psychiatric side effects.
  • Experts emphasize the need for continued monitoring of mood changes in patients taking anti-obesity medications, especially those with mental health history.

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