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Drug Farm's DF-003 Shows Promising Safety Profile in Phase 1 Trial for Rare ROSAH Syndrome

Rare DiseasePrecision Medicine
  • Drug Farm reported positive Phase 1 results for DF-003, a first-in-class ALPK1 inhibitor targeting ROSAH syndrome, showing excellent safety with no serious adverse events across all tested doses.
  • The randomized, placebo-controlled study in 48 healthy volunteers demonstrated dose-proportional pharmacokinetics supporting once-daily oral administration for future trials.
  • DF-003 achieved blood concentrations consistent with efficacy in preclinical models, positioning the company to advance into proof-of-concept trials for ROSAH syndrome and cardio-renal disease.
  • The drug represents a potential breakthrough for ROSAH syndrome, a rare genetic disease with no approved treatments that causes progressive vision loss and inflammatory symptoms.

Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients

NCT06395285RecruitingPhase 1
Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
Posted 5/27/2025

Evaluating the Safety, Tolerability, and Pharmacokinetics of DF-003 in Healthy Subjects

NCT05997641Active, Not RecruitingPhase 1
Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
Posted 9/15/2023

Estrella Immunopharma Advances EB103 ARTEMIS T-Cell Therapy to Second Cohort in STARLIGHT-1 Trial for Advanced B-Cell Lymphomas

CAR-T
  • Estrella Immunopharma has dosed the first patient in the second cohort of its Phase I/II STARLIGHT-1 trial, evaluating EB103 at higher doses for advanced B-cell non-Hodgkin's lymphomas.
  • The first cohort demonstrated a favorable safety profile with no dose-limiting toxicities or treatment-related serious adverse events, along with a complete response.
  • EB103 utilizes ARTEMIS technology to potentially address limitations of traditional CAR-T therapies, including expanded access to high-risk patients with HIV-associated and CNS lymphomas.
  • The therapy targets CD19-positive cancer cells using a mechanism that more closely resembles endogenous T-cell receptor activation compared to conventional CAR-T approaches.

T-Cell Therapy (EB103) in Adults With Relapsed/Refractory B-Cell Non-Hodgkin's Lymphoma (NHL)

NCT06343311RecruitingPhase 1
Estrella Biopharma, Inc.
Posted 6/1/2024

Merck and Daiichi Sankyo Withdraw FDA Application for Lung Cancer ADC After Survival Data Disappoints

  • Merck and Daiichi Sankyo withdrew their FDA application for patritumab deruxtecan, an antibody-drug conjugate targeting EGFR-mutated non-small cell lung cancer, after the drug failed to demonstrate overall survival benefit.
  • The Phase III HERTHENA-Lung02 trial showed the ADC significantly improved progression-free survival compared to chemotherapy but did not meet the gold standard of extending patient survival.
  • This setback affects the companies' $22 billion partnership and highlights ongoing challenges in treating EGFR-mutated lung cancer patients in second-line and later settings.
  • The companies plan to conduct further biomarker analyses to identify patients who might benefit from treatment while continuing development across 15 cancer types.

HERTHENA-Lung01: Patritumab Deruxtecan in Subjects With Metastatic or Locally Advanced EGFR-mutated Non-Small Cell Lung Cancer

NCT04619004Active, Not RecruitingPhase 2
Daiichi Sankyo
Posted 2/2/2021

HERTHENA-Lung02: A Study of Patritumab Deruxtecan Versus Platinum-based Chemotherapy in Metastatic or Locally Advanced EGFRm NSCLC After Failure of EGFR TKI Therapy

NCT05338970Active, Not RecruitingPhase 3
Daiichi Sankyo
Posted 7/8/2022

BioAffinity Technologies Secures Patent for Dual-Target Cancer Therapy Using siRNA Against CD320 and LRP2 Receptors

  • BioAffinity Technologies received a U.S. patent for a novel cancer therapy that uses small interfering RNAs to simultaneously target CD320 and LRP2 receptors on cancer cell membranes.
  • In vitro studies demonstrated that dual knockdown of both proteins selectively kills human cancer cells while sparing normal cells, with applications across lung, breast, prostate, brain, and skin cancers.
  • The company has initiated research toward developing a topical treatment for skin cancers, marking a significant advancement in their therapeutic pipeline and intellectual property portfolio.

Bionical Emas Secures Exclusive Partnership to Address Critical European Fluorouracil Shortage

Oncology
  • Bionical Emas has secured an exclusive global clinical trial supply agreement with German pharmaceutical company Pharma Resources GmbH to distribute EU-marketed oncology injectables, including Fluorouracil.
  • The partnership addresses critical 5-Fluorouracil shortages that are currently disrupting cancer treatments and clinical trials across Europe.
  • Bionical Emas' global infrastructure and agile supply model will reduce delivery times from months to weeks for essential oncology medications.
  • This strategic alliance marks Pharma Resources' entry into the clinical trial supply market while strengthening Bionical Emas' position as a trusted supply partner.

iVeena Submits IND Application for Novel Non-Atropine Eye Drop to Treat Pediatric Myopia

  • iVeena Delivery Systems has submitted an Investigational New Drug application to the FDA for IVMED-85, a first-in-class eye drop targeting pediatric myopia through lysyl oxidase activation.
  • The preservative-free daily eye drop represents a non-atropine approach that strengthens scleral and corneal collagen crosslinks to potentially reduce myopic progression.
  • With myopia affecting approximately 40% of children in the US, IVMED-85 addresses a critical unmet medical need in pediatric ophthalmology.
  • The company plans to initiate a multinational Phase 2 clinical trial in 2025 pending FDA approval within the expected 30-day review period.

KYZATREX Oral Testosterone Therapy Achieves 96% Efficacy Rate in Phase 3 Trials for Hypogonadism Treatment

Drug Approval
  • KYZATREX, developed by Marius Pharmaceuticals, received FDA approval in 2022 as the first oral testosterone replacement therapy utilizing phytosterols for lymphatic absorption.
  • Clinical trials demonstrated up to 96% of patients achieved normal testosterone levels by Day 90, with improvements in quality of life, energy, and erectile function.
  • The therapy addresses a significant unmet need, with approximately 40% of men over 45 and 30-50% of men with obesity or diabetes suffering from hypogonadism.
  • The global testosterone replacement therapy market is projected to grow from $1.9 billion in 2022 to $2.9 billion by 2032, with oral formulations expected to register high growth rates.

Pfizer Faces Legal Challenges as Study Links Depo-Provera to Increased Meningioma Risk

  • A study published in Expert Opinion on Drug Safety in late July 2025 linked Pfizer's Depo-Provera contraceptive to a significantly higher risk of developing intracranial meningioma with extended use.
  • The findings have fueled ongoing lawsuits against Pfizer and raised concerns about inconsistencies in global risk communication for the contraceptive product.
  • Pfizer's stock declined 5.2% following the study publication, adding legal and reputational risks to the company's investment narrative.
  • Despite these challenges, analysts maintain revenue projections of $60.3 billion and earnings of $13.4 billion by 2028, though some forecasts reach as high as $64.3 billion in revenue.

Sarvodaya Hospital Becomes First in India to Offer Terbium-161 PSMA Therapy for Advanced Prostate Cancer

CAR-T
  • Sarvodaya Hospital in Faridabad has become India's first theranostics and nuclear medicine center to introduce Terbium-161 PSMA therapy for metastatic castration-resistant prostate cancer.
  • The hospital has simultaneously launched CAR-T cell therapy for blood cancers including leukemia, lymphoma, and multiple myeloma, creating comprehensive cancer care under one roof.
  • This targeted therapy represents a significant advancement in precision oncology, delivering radiation directly to cancer cells while sparing healthy tissue.
  • The dual launch positions Sarvodaya as one of the few institutions in India offering end-to-end cancer care from diagnosis to the most advanced treatment options.

Excyte Doses First Patient with YK012, World's First T Cell Engager for Primary Membranous Nephropathy

  • Excyte has dosed the first patient with YK012, marking the world's first T cell engager therapy to enter clinical trials for primary membranous nephropathy, an autoimmune kidney disease.
  • The bispecific CD19-directed CD3 T cell engager targets B cell depletion while demonstrating milder T cell activation compared to existing therapies like Blincyto.
  • Primary membranous nephropathy affects 20-37% of non-diabetic adults with primary nephrotic syndrome, with 20-30% of cases resistant to current treatments like rituximab and cyclophosphamide.
  • The company plans to file for US IND application imminently and has already secured regulatory approvals in China and additional autoimmune indications including systemic lupus erythematosus.

Study of YK012 in Primary Membranous Nephropathy

NCT06982729RecruitingPhase 1
Excyte Biopharma Ltd
Posted 3/8/2025

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