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Clinical Trial News

FDA Grants Orphan Drug Designation to Certepetide for Cholangiocarcinoma

  • The FDA has granted orphan drug designation to certepetide for the treatment of cholangiocarcinoma, a rare and aggressive cancer with limited treatment options.
  • Certepetide is currently being evaluated in the phase 2 BOLSTER trial in combination with standard-of-care therapies for first- and second-line cholangiocarcinoma.
  • The orphan drug designation provides Lisata Therapeutics with potential benefits, including market exclusivity, exemption from user fees, and tax credits for clinical trials.
  • Certepetide aims to enhance the penetration and accumulation of anticancer agents in solid tumors by activating the C-end rule active transport mechanism.

A Study of LSTA1 When Added to Standard of Care Versus Standard of Care Alone in Patients With Advanced Solid Tumors

NCT05712356Active, Not RecruitingPhase 2
Lisata Therapeutics, Inc.
Posted 8/24/2023

CEND-1 in Combination with Neoadjuvant FOLFIRINOX with or Without Panitumumab

NCT05121038Active, Not RecruitingPhase 1
Anup Kasi
Posted 10/20/2021

The ASCEND Study: Gemcitabine and Nab-Paclitaxel With LSTA1 (Certepetide) or Placebo in Patients With Untreated Metastatic Pancreatic Ductal Adenocarcinoma

NCT05042128Active, Not RecruitingPhase 2
Australasian Gastro-Intestinal Trials Group
Posted 4/13/2022

UltraDx Bio's Single-Molecule Analyzer Receives Clinical Approval in China for Early Alzheimer's Detection

  • UltraDx Bio's UD-X™ Single-Molecule Analyzer has received clinical approval in China, marking a significant advancement in medical diagnostics.
  • The analyzer enables ultra-sensitive detection of trace protein biomarkers in body fluids, crucial for early disease screening and diagnosis.
  • Experts emphasize the importance of early detection for diseases like Alzheimer's, where timely intervention can mitigate disease progression.
  • The technology aims to address the increasing burden of Alzheimer's in China, which has the highest number of affected individuals globally.

FDA Approves Galderma's Nemluvio as First IL-31 Inhibitor for Prurigo Nodularis

• The FDA has approved Galderma's Nemluvio (nemolizumab) as the first IL-31 receptor mAb for treating prurigo nodularis, a rare skin condition. • In Phase III trials, Nemluvio demonstrated a significant reduction in itch intensity compared to placebo, with results comparable to Dupixent. • Nemluvio offers a new treatment option for prurigo nodularis patients, addressing a condition with limited existing therapies. • The approval marks a significant milestone for Galderma and provides a competitive alternative to existing treatments like Dupixent.

Pyrotinib Plus Metronomic Vinorelbine Shows Efficacy in HER2+ Advanced Breast Cancer Post-Trastuzumab

  • A phase 2 trial in China demonstrated that pyrotinib combined with metronomic vinorelbine is effective in HER2-positive advanced breast cancer patients after trastuzumab failure.
  • The combination therapy achieved a median progression-free survival of 13.5 months and an overall response rate of 38.9% in the studied patient population.
  • The treatment regimen showed a manageable safety profile, with the most common adverse events being diarrhea, nausea, and vomiting, and no grade 4/5 events reported.
  • Researchers suggest this dual-oral regimen is a feasible option where antibody-drug conjugates are not readily accessible, warranting further investigation in larger trials.

A Study of Pyrotinib Plus Capecitabine Combined With SRT in HER2+ MBC With Brain Metastases

NCT05042791RecruitingPhase 2
Jiangxi Provincial Cancer Hospital
Posted 3/1/2022

Pyrotinib Combined With Brain Radiotherapy in Breast Cancer Patients With Brain Metastases

NCT04582968Active, Not RecruitingPhase 1
Fudan University
Posted 1/2/2020

Pyrotinib Maleate Combined With Vinorelbine in the Treatment of HER2-positive Advanced Breast Cancer

NCT04903652CompletedPhase 2
Tianjin Medical University Cancer Institute and Hospital
Posted 1/1/2021

A Study of Trastuzumab Emtansine Versus Capecitabine + Lapatinib in Participants With HER2-positive Locally Advanced or Metastatic Breast Cancer

NCT00829166CompletedPhase 3
Hoffmann-La Roche
Posted 2/1/2009

Bispecific Antibody-Drug Conjugates Show Promise in Solid Tumor Treatment

  • Bispecific antibody-drug conjugates (BsADCs) are emerging as a cutting-edge approach in cancer therapy, combining bispecific antibody precision with antibody-drug conjugate potency.
  • Several BsADC candidates are advancing to late-stage clinical trials, including Alhamab Oncology's JSKN003 and Sichuan Baili Pharmaceutical's BL-B01D1, targeting HER2 and EGFR×HER3, respectively.
  • Collaborations and licensing agreements, such as those between Biocytogen, ABL Bio, and SOTIO Biotech, are accelerating BsADC development and expanding treatment possibilities through combination therapies.
  • Regulatory agencies are increasingly clearing BsADC candidates for clinical trials, with Doma Bio's DM001 and DM005 recently approved by the FDA for Phase I studies in solid tumors.

Novel Antibody-like Molecule Shows Promise for Broad-Spectrum Malaria Therapy

  • Researchers have discovered WD34, a novel antibody-like molecule, that inhibits malaria parasite infection at various disease stages.
  • WD34 binds to the AMA1 protein, common across malaria species, blocking the mechanism parasites use to enter liver and red blood cells.
  • Pre-clinical trials suggest WD34 could protect against multiple malaria parasite species, offering a potential broad-spectrum therapeutic option.
  • The discovery opens new avenues for developing a universal malaria vaccine, addressing drug resistance and treatment failures.

Vanda Pharmaceuticals Faces Challenges Despite New Drug Launches and Takeover Bids

  • Vanda Pharmaceuticals' Fanapt, approved for bipolar 1 disorder, faces challenges due to competition from generic antipsychotics with established physician experience.
  • Tradipitant's Phase 3 trial for gastroparesis failed to meet primary endpoints, and motion sickness treatment faces competition from affordable generics like scopolamine.
  • Despite takeover bids, Vanda rejected offers, citing undervaluation, raising concerns about cash management and strategic direction given pipeline challenges.
  • Vanda's internally developed Hetlioz is experiencing revenue decline due to generic competition, impacting overall financial performance.

FDA Approves Oncomine Dx Target Test as Companion Diagnostic for Vorasidenib in IDH-Mutant Glioma

  • The FDA has approved the Ion Torrent Oncomine Dx Target Test as a companion diagnostic for vorasidenib in grade 2 astrocytoma or oligodendroglioma.
  • Vorasidenib is the first targeted therapy for patients with grade 2 IDH-mutant glioma, offering a new treatment option after nearly 25 years.
  • The Oncomine Dx Target Test simultaneously delivers biomarker results for multiple therapies from a single sample, quickly matching patients to treatments.
  • Vorasidenib demonstrated a 61% reduction in disease progression or death risk compared to placebo in the phase 3 INDIGO trial.

Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)

NCT04164901Active, Not RecruitingPhase 3
Institut de Recherches Internationales Servier
Posted 1/5/2020

Study of AG-120 and AG-881 in Subjects With Low Grade Glioma

NCT03343197CompletedPhase 1
Institut de Recherches Internationales Servier
Posted 3/20/2018

Vorasidenib Expanded Access Program

NCT05592743Approved for Marketing
Servier

Vorasidenib in Combination With Temozolomide (TMZ) in IDH-mutant Glioma

NCT06478212RecruitingPhase 1
Institut de Recherches Internationales Servier
Posted 1/22/2025

ViCToRy: Vorasidenib in Combination With Tumor Specific Peptide Vaccine for Recurrent IDH1 Mutant Lower Grade Gliomas

NCT05609994RecruitingPhase 1
Katy Peters, MD, PhD
Posted 7/15/2025

Study of Orally Administered AG-881 in Patients With Advanced Solid Tumors, Including Gliomas, With an IDH1 and/or IDH2 Mutation

NCT02481154CompletedPhase 1
Institut de Recherches Internationales Servier
Posted 6/1/2015

Study of Vorasidenib and Pembrolizumab Combination in Recurrent or Progressive IDH-1 Mutant Glioma

NCT05484622RecruitingPhase 1
Institut de Recherches Internationales Servier
Posted 1/20/2023

Ocuphire Pharma Initiates Phase 3 VEGA-3 Trial of Phentolamine Ophthalmic Solution for Presbyopia

  • Ocuphire Pharma has dosed the first patient in its Phase 3 VEGA-3 clinical trial evaluating phentolamine ophthalmic solution 0.75% for presbyopia.
  • The VEGA-3 trial is a randomized, double-masked, placebo-controlled study involving 545 participants, with top-line data expected in 2025.
  • Phentolamine ophthalmic solution 0.75% aims to provide a non-invasive alternative to traditional corrective measures for presbyopia.
  • Ocuphire anticipates using VEGA-3 data to support a supplemental New Drug Application with the FDA.

Evaluate Efficacy and Safety of POS to Improve Distance-corrected Near Visual in Participants With Presbyopia

NCT06542497Active, Not RecruitingPhase 3
Ocuphire Pharma, Inc.
Posted 8/15/2024

Safety and Efficacy of 0.75% Phentolamine Ophthalmic Solution in Subjects With Post-refractive Surgery Visual Disturbances

NCT06349759Active, Not RecruitingPhase 3
Ocuphire Pharma, Inc.
Posted 4/1/2024

Safety and Efficacy of Nyxol Eye Drops as a Single Agent and With Adjunctive Low-Dose Pilocarpine Eye Drops in Subjects With Presbyopia

NCT05646719CompletedPhase 3
Ocuphire Pharma, Inc.
Posted 12/22/2022

Gene Therapy Shows Promise in Restoring Vision for LCA1 Patients

  • A Phase 1/2 clinical trial of ATSN-101 gene therapy demonstrates significant vision improvement in patients with Leber congenital amaurosis type 1 (LCA1).
  • Some patients experienced up to a 10,000-fold increase in light sensitivity, enabling them to see in dimly lit environments.
  • The gene therapy, developed by Atsena Therapeutics, showed a favorable safety profile with mostly mild, surgery-related side effects.
  • These findings support a future Phase 3 trial to further evaluate the efficacy and safety of ATSN-101 for LCA1, a rare inherited blindness.

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