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Clinical Trial News

FDA Lifts Clinical Hold on Rezolute's Ersodetug for Congenital Hyperinsulinism, Allowing US Trial Enrollment

  • The FDA has lifted the partial clinical hold on Rezolute's ersodetug, clearing the way for U.S. patients to participate in the Phase 3 sunRIZE trial.
  • The decision was based on follow-up toxicology studies that found liver toxicity observed in earlier rat studies was strain-specific and not relevant to humans.
  • Rezolute plans to begin enrolling U.S. participants in early 2025, with topline data from the global sunRIZE study expected in the second half of 2025.
  • Ersodetug, a fully human monoclonal antibody, is being developed for congenital hyperinsulinism, a rare genetic disorder causing severe and continuous low blood sugar.

Medicenna Presents Preclinical Data on IL-2 Super-Antagonist MDNA209 and Anti-PD1-IL-2 BiSKIT MDNA113

  • Medicenna's MDNA209, an IL-2 super-antagonist, demonstrated a 400% increase in overall survival in an animal model of graft versus host disease (GvHD), suggesting potential in autoimmune disease treatment.
  • MDNA113, a tumor-targeted anti-PD1-IL-2 BiSKIT, showed enhanced efficacy in mice with IL-13Rα2-overexpressing tumors, indicating promise for treating immunologically 'cold' tumors like pancreatic and ovarian cancers.
  • The BiSKIT approach of MDNA113 allows for conditional activation in the tumor microenvironment, potentially improving safety and efficacy compared to traditional anti-PD1 therapies.
  • These preclinical findings validate Medicenna's IL-2 Superkine platform and its potential to develop transformative medicines for both cancer and autoimmune diseases.

Tonix Pharmaceuticals Presents Promising Data on Mpox Vaccine Candidate TNX-801

  • Tonix Pharmaceuticals' TNX-801, a horsepox-based vaccine, demonstrates efficacy in protecting animals against lethal mpox challenge, supporting its potential as a single-dose mpox vaccine.
  • New data indicates improved tolerability of TNX-801 in immunocompromised animals, with no evidence of spread to blood or tissues, even at high doses, suggesting a favorable safety profile.
  • TNX-801's single-dose regimen and stability at standard temperatures offer advantages over existing mpox vaccines, potentially facilitating widespread use, especially in resource-limited settings.
  • Tonix's synthetic horsepox vaccine platform has been selected by NIH’s Project NextGen for clinical testing, highlighting its potential in combating emerging infectious diseases like COVID-19.

Terns Pharmaceuticals' Oral GLP-1 Agonist TERN-601 Shows Promise in Phase 1 Obesity Trial

  • Terns Pharmaceuticals announced positive Phase 1 results for TERN-601, an oral GLP-1R agonist, showing statistically significant weight loss of up to 5.5% over 28 days.
  • The trial demonstrated a placebo-adjusted mean weight loss of 4.9% at the highest dose (740 mg), with 67% of participants losing 5% or more of their body weight.
  • TERN-601 was well-tolerated, with no treatment-related dose interruptions, reductions, or discontinuations, indicating potential for improved tolerability in future studies.
  • Terns plans to initiate a Phase 2 clinical trial in 2025 to further evaluate TERN-601 as a monotherapy or in combination with other agents for obesity treatment.

FDA Grants Rare Pediatric Disease Designation to Cartesian's Descartes-08 for Juvenile Dermatomyositis

  • The FDA has granted Rare Pediatric Disease Designation to Cartesian Therapeutics' Descartes-08 for treating juvenile dermatomyositis (JDM).
  • Descartes-08 is an autologous mRNA-engineered CAR-T therapy targeting BCMA, designed for administration without preconditioning chemotherapy.
  • Cartesian plans to file an IND for a Phase 2 pediatric basket study by year-end, focusing on autoimmune indications including JDM.
  • If approved for JDM, Cartesian may qualify for a priority review voucher, potentially expediting future marketing applications.

Descartes-08 for Patients With Systemic Lupus Erythematosus

NCT06038474RecruitingPhase 2
Cartesian Therapeutics
Posted 2/12/2024

SAB Biotherapeutics' SAB-142 Shows Promising Safety Profile in Type 1 Diabetes Trial

  • SAB Biotherapeutics' SAB-142 completes Phase 1 enrollment in healthy volunteers, advancing to enroll patients with type 1 diabetes (T1D).
  • The Phase 1 trial of SAB-142 demonstrates no serum sickness at the target dose of 2.5mg/kg, a key differentiator from rabbit anti-thymocyte globulin.
  • SAB-142, a human anti-thymocyte immunoglobulin, aims to delay the onset and progression of T1D by preserving insulin-producing beta cells.
  • Topline Phase 1 data readout for SAB-142 is expected by the end of the year, with plans for a Phase 2 SAFEGUARD study in new-onset T1D patients.

Akero Therapeutics Doses First Patient in Phase 3 SYNCHRONY Outcomes Trial for MASH Cirrhosis

  • Akero Therapeutics has dosed the first patient in its Phase 3 SYNCHRONY Outcomes trial evaluating Efruxifermin (EFX) for compensated cirrhosis (F4) due to MASH.
  • The SYNCHRONY program includes two other ongoing trials: SYNCHRONY Histology (pre-cirrhotic MASH, F2-F3) and SYNCHRONY Real-World (non-invasively diagnosed MASLD/MASH, F1-F4).
  • Efruxifermin (EFX) is a novel Fc-FGF21 fusion protein designed to reduce liver fat and inflammation, reverse fibrosis, and improve insulin sensitivity and lipid metabolism.
  • Topline results from the Phase 2b SYMMETRY study of EFX in compensated cirrhosis due to MASH (F4) are expected in the first quarter of 2025.

Inaxaplin Shows Promise in Addressing APOL1-Mediated Kidney Disease Disparity in Black Americans

  • A clinical trial of inaxaplin, a novel drug targeting the APOL1 protein, demonstrated a significant reduction in protein leakage in the urine of patients with serious kidney disease.
  • The study focused exclusively on African American participants with APOL1 gene variants (G1 and G2) linked to a higher risk of renal failure, addressing a significant racial health disparity.
  • Vertex Pharmaceuticals is conducting a larger Phase 2/3 trial to investigate inaxaplin's efficacy across multiple kidney disease types in individuals with two APOL1 gene variants.
  • Insights into APOL1 may also refine kidney transplant policies by using APOL1 genotype instead of race to assess donor kidney viability, potentially increasing the number of available kidneys.

Revolo Bio's '1104 Shows Promise in Resetting Immune Response in Allergic Diseases

  • Revolo Biotherapeutics' '1104 targets antigen-presenting cells, shifting the immune system from a pro-inflammatory to a homeostatic state, potentially inducing remission in allergic conditions.
  • Preclinical and clinical studies validate '1104's mechanism, demonstrating effectiveness in reducing inflammatory cell infiltration and cytokine release in Th2 allergic diseases like eosinophilic esophagitis.
  • '1104's upstream intervention in the inflammatory cascade offers a more durable disease control and improved patient outcomes compared to traditional therapies targeting downstream effects.
  • The antigen-agnostic nature of '1104 suggests potential in addressing unmet needs in various allergic diseases beyond EoE, asthma, and atopic dermatitis, such as food allergies.

Ifinatamab Deruxtecan Shows Promise in Extensive-Stage Small Cell Lung Cancer

  • Interim data from the IDeate-Lung01 trial reveal a 54.8% objective response rate with ifinatamab deruxtecan (I-DXd) at 12 mg/kg in patients with extensive-stage small cell lung cancer (ES-SCLC).
  • The disease control rate (DCR) reached 90.5% in the 12 mg/kg arm, indicating a substantial proportion of patients experienced disease stabilization.
  • The 12 mg/kg dose of I-DXd has been selected for further evaluation in the second part of the IDeate-Lung01 trial and the phase 3 IDeate-Lung02 study.
  • Further research is needed to fully assess the toxicity profile of I-DXd and to analyze the complete data from the IDeate-Lung01 study.

Ifinatamab Deruxtecan (I-DXd) in Subjects With Pretreated Extensive-Stage Small Cell Lung Cancer (ES-SCLC)

NCT05280470Active, Not RecruitingPhase 2
Daiichi Sankyo
Posted 3/9/2022

A Study of Ifinatamab Deruxtecan Versus Treatment of Physician's Choice in Subjects With Relapsed Small Cell Lung Cancer

NCT06203210RecruitingPhase 3
Daiichi Sankyo
Posted 5/21/2024

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