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Clinical Trial News

Rosacea in Patients of Color: Experts Call for Better Recognition and Diagnosis

  • Rosacea affects approximately 40 million patients of color globally, challenging the traditional view of it being primarily a condition affecting those of Northern European descent.
  • Diagnostic challenges in darker skin tones often lead to delayed treatment, with most patients of color experiencing symptoms for over a year before receiving proper diagnosis.
  • Current treatment approaches remain consistent across all skin types, with newer medications like ivermectin cream 1%, minocycline foam 1.5%, and microencapsulated benzoyl peroxide 5% showing promise.

Infigratinib Receives FDA Breakthrough Therapy Designation for Achondroplasia

  • The FDA granted Breakthrough Therapy Designation to BridgeBio's infigratinib for treating children with achondroplasia, a genetic bone growth disorder.
  • Phase 2 PROPEL 2 trial data showed infigratinib led to a statistically significant increase in annualized height velocity (AHV) in children.
  • Infigratinib could become the first oral treatment option for achondroplasia, potentially improving the quality of life and functionality for patients.
  • BridgeBio's Phase 3 PROPEL 3 study is ongoing, with completion of enrollment expected by the end of the year.

Study of Infigratinib in Children with Achondroplasia

NCT04265651CompletedPhase 2
QED Therapeutics, Inc.
Posted 3/10/2020

A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

NCT06164951Active, Not RecruitingPhase 3
QED Therapeutics, Inc.
Posted 11/10/2023

Solid Tumor Market Sees Innovation with Aethlon Medical's Hemopurifier Trial and New Immunotherapy Approvals

  • Aethlon Medical activates a clinical trial site in Australia for its Hemopurifier device in patients with solid tumors resistant to anti-PD-1 therapies.
  • The solid tumors market is projected to reach $375.4 billion by 2034, driven by advancements in targeted and immunotherapies.
  • Immuneering reports promising initial data for IMM-1-104 in pancreatic cancer, showing a 40% objective response rate in early trials.
  • FDA approves new immunotherapy combinations from AstraZeneca, Merck, and GlaxoSmithKline for advanced endometrial cancer, expanding treatment options.

Rocket Pharma Completes Enrollment in Phase 2 Trial of RP-A501 for Danon Disease

  • Rocket Pharmaceuticals has completed enrollment in its Phase 2 pivotal trial of RP-A501 for male patients with Danon disease.
  • The trial enrolled 12 patients across the U.S. and EU, evaluating RP-A501's efficacy and safety with a dose of 6.7 x 10^13 GC/kg.
  • RP-A501 targets LAMP2 protein expression and left ventricular mass, with potential for accelerated approval based on biomarker endpoints.
  • Rocket plans to pursue simultaneous regulatory filings in the U.S. and other regions, addressing the unmet need for Danon disease treatment.

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

NCT06092034RecruitingPhase 2
Rocket Pharmaceuticals Inc.
Posted 9/5/2023

TransCode Therapeutics Advances Metastatic Cancer Therapy with TTX-MC138 Phase 1 Trial

  • TransCode Therapeutics has dosed the first two patients in a Phase 1 clinical trial of TTX-MC138, a novel therapy for metastatic cancers.
  • The trial is designed to evaluate the safety and tolerability of TTX-MC138 in patients with various metastatic solid tumors.
  • TTX-MC138 targets microRNA-10b, a key driver of metastasis, and showed evidence of delivery to metastatic lesions in a Phase 0 trial.
  • Initial dosing of the first cohort in the Phase 1a trial has been completed with no significant safety or dose-limiting toxicities reported.

Study of TTX-MC138 in Subjects With Advanced Solid Tumors

NCT06260774RecruitingPhase 1
TransCode Therapeutics
Posted 9/5/2024

Guard Therapeutics' RMC-035 Shows Kidney-Protective Effect in Phase 2 Trial

  • Guard Therapeutics' Phase 2 AKITA study results, published in eClinicalMedicine, demonstrate a statistically significant kidney-protective effect of RMC-035 in patients undergoing open-heart surgery.
  • RMC-035 improved kidney function, measured by estimated glomerular filtration rate (eGFR), at 90 days post-surgery compared to placebo, indicating a clinically relevant benefit.
  • The study also showed a reduction in Major Adverse Kidney Events (MAKE) at 90 days, aligning with the expected primary efficacy endpoint for a future Phase 3 trial.
  • A Phase 2b study (POINTER) has been initiated to optimize the dose and identify the target patient population for RMC-035 before a pivotal Phase 3 study.

Poseida's Allogeneic CAR-T Therapy P-BCMA-ALLO1 Shows High Response in Myeloma

  • Poseida Therapeutics reported a 91% overall response rate with P-BCMA-ALLO1 in relapsed/refractory multiple myeloma patients in an optimized lymphodepletion arm.
  • The allogeneic CAR-T therapy demonstrated a favorable safety profile, with low rates of cytokine release syndrome and no graft-versus-host disease.
  • P-BCMA-ALLO1 has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, expediting its development for multiple myeloma.
  • The Phase 1 trial results highlight the potential of P-BCMA-ALLO1 as an off-the-shelf treatment option, eliminating the need for apheresis or bridging therapy.

P-BCMA-ALLO1 Allogeneic CAR-T Cells in the Treatment of Subjects With Multiple Myeloma

NCT04960579RecruitingPhase 1
Poseida Therapeutics, Inc.
Posted 5/5/2022

BeiGene's Tevimbra Approved in Israel for Advanced Esophageal Cancer

  • The Israeli Ministry of Health has approved Tevimbra (tislelizumab) as a monotherapy for unresectable or metastatic esophageal squamous cell carcinoma (OSCC).
  • This approval provides a new treatment option for adult patients who have previously undergone systemic chemotherapy, addressing a critical need in cancer care.
  • BeiGene is expanding Tevimbra's availability in Israel, with submissions for first- and second-line treatments for non-small cell lung cancer (NSCLC) underway.
  • Over 900,000 patients have been prescribed Tevimbra globally, demonstrating its potential to improve survival and quality of life across various tumor types.

Cullinan Therapeutics Advances CLN-978 for SLE Treatment with Phase 1 Trial Approval

  • Cullinan Therapeutics has received approval to initiate a global Phase 1 clinical trial of CLN-978 for systemic lupus erythematosus (SLE).
  • The Phase 1 trial will assess the safety, pharmacokinetics, and initial clinical activity of CLN-978 in SLE patients across multiple sites, including Australia.
  • CLN-978 is a novel CD19xCD3 bispecific T cell engager designed for convenient dosing and potential treatment-free remission in SLE patients.
  • The FDA has also approved Cullinan to expand its global trial to include U.S. patients with moderate to severe SLE.

Boston Scientific's INGEVITY+ Pacing Leads Receive FDA Expanded Indication for LBBA Pacing

  • Boston Scientific has gained FDA approval for an expanded indication of its INGEVITY+ Pacing Leads.
  • The expanded indication includes conduction system pacing (CSP) and sensing of the left bundle branch area (LBBA).
  • LBBA pacing offers an alternative to traditional right ventricular pacing for symptomatic bradycardia, potentially reducing heart failure risk.
  • The approval was supported by data from the INSIGHT-LBBA study, involving approximately 400 patients.

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