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Assembly Bio's ABI-5366 Shows Promise for Recurrent Genital Herpes Treatment

  • Assembly Biosciences reports positive Phase 1a results for ABI-5366, an HSV helicase-primase inhibitor, showing a favorable safety profile.
  • The drug exhibits an approximate 20-day half-life, supporting potential once-weekly or once-monthly oral dosing regimens.
  • Phase 1b screening is underway to assess ABI-5366's efficacy in participants with recurrent genital herpes, with interim results expected in H1 2025.

A Study to Assess the Safety, Pharmacokinetics, and Antiviral Activity of ABI-5366 in Healthy Participants and Participants Seropositive for HSV-2 With Recurrent Genital Herpes

NCT06385327RecruitingPhase 1
Assembly Biosciences
Posted 5/30/2024

Oryzon's Vafidemstat Receives FDA Support for Phase III Trial in Borderline Personality Disorder

  • Oryzon Genomics has received positive feedback from the FDA regarding its Phase II meeting for vafidemstat in Borderline Personality Disorder (BPD).
  • The FDA supports the initiation of a Phase III trial, acknowledging agitation-aggression in BPD as a potential therapeutic indication.
  • Oryzon may use the STAXI-2 Trait anger scale as a primary efficacy endpoint for the PORTICO-2 trial, pending further validation.
  • Vafidemstat demonstrated a statistically significant reduction in agitation and aggression in the Phase IIb PORTICO study, showing promise for BPD treatment.

Study to Test the Efficacy and Safety of Vafidemstat in Adult Borderline Personality Disorder Population

NCT04932291CompletedPhase 2
Oryzon Genomics S.A.
Posted 3/26/2021

Carrick Therapeutics Initiates Phase 1 Trial of Novel CDK12/13 Inhibitor CT7439 in Advanced Solid Tumors

  • Carrick Therapeutics has dosed the first patient in a Phase 1 clinical trial of CT7439, a novel CDK12/13 inhibitor and Cyclin-K glue-degrader.
  • The Phase 1 trial will assess the safety, pharmacokinetics, and early proof of principle of CT7439 in patients with advanced solid tumors.
  • CT7439's dual mechanism of action targets DNA repair at the transcriptional level, potentially synergizing with PARP inhibitors in various cancers.
  • This trial marks the advancement of Carrick Therapeutics' second therapeutic into clinical development for aggressive and resistant cancers.

A Modular Phase 1/2 Study with CT7439 in Participants with Solid Malignancies

NCT06600789RecruitingPhase 1
Carrick Therapeutics Limited
Posted 8/16/2024

Ipsen's Kayfanda (Odevixibat) Receives EU Approval for Cholestatic Pruritus in Alagille Syndrome

  • The European Commission has approved Kayfanda (odevixibat) for treating cholestatic pruritus in Alagille Syndrome (ALGS) patients aged six months and older.
  • Kayfanda's approval is based on the ASSERT Phase III trial, which demonstrated significant improvements in scratching severity and serum bile acid concentration.
  • Odevixibat, a once-daily non-systemic ileal bile acid transport (IBAT) inhibitor, reduces serum bile acids by blocking the IBAT protein.
  • This approval expands Ipsen’s portfolio in rare cholestatic liver diseases, offering a new treatment option for the ALGS community in the EU.

Brepocitinib Shows Promise in Non-Infectious Uveitis: Phase 3 Trial Initiated

  • Priovant Therapeutics' brepocitinib demonstrated significant treatment failure reductions in the Phase 2 NEPTUNE trial for non-infectious uveitis.
  • The CLARITY Phase 3 trial has been initiated to further evaluate brepocitinib's efficacy against non-anterior non-infectious uveitis, comparing 45mg dose to placebo.
  • Brepocitinib received Fast Track Designation from the FDA, expediting its development for non-infectious uveitis due to unmet medical needs.
  • The dual TYK2/JAK1 inhibitor showed a dose-dependent improvement in retinal vascular leakage, as measured by fluorescein angiography, in the NEPTUNE trial.

A Study of Brepocitinib in Adults with Active Non-Infectious Non-Anterior Uveitis

NCT05523765CompletedPhase 2
Priovant Therapeutics, Inc.
Posted 11/14/2022

A Phase 3 Study of Brepocitinib in Adults with Active, Non-Infectious, Non-Anterior Uveitis (CLARITY)

2024-515089-15-00Not Yet RecruitingPhase 3
Priovant Therapeutics Inc.

FDA Grants Fast Track Designation to Elevation Oncology's EO-3021 for Gastric and GEJ Cancers

  • The FDA has granted Fast Track designation to EO-3021 for advanced or metastatic gastric and gastroesophageal junction (GEJ) cancer expressing Claudin 18.2.
  • EO-3021, a differentiated antibody-drug conjugate, targets Claudin 18.2, a protein exposed in malignant transformation of gastric epithelial cells.
  • The Fast Track designation aims to expedite the development and review of EO-3021, offering potential for priority review and accelerated approval.
  • Early Phase 1 trial results showed a 42.8% overall response rate in a Claudin 18.2-enriched subset of gastric and GEJ cancer patients.

Study of EO-3021 in Adult Patients With Solid Tumors Likely to Express CLDN18.2

NCT05980416RecruitingPhase 1
Elevation Oncology
Posted 8/10/2023

uniQure's AMT-191 Receives FDA Orphan Drug Designation for Fabry Disease

  • The FDA has granted Orphan Drug Designation to uniQure's AMT-191, a gene therapy for Fabry disease, highlighting the need for innovative treatments.
  • AMT-191 is a one-time, intravenously administered AAV5-based gene therapy designed to target the liver and produce the deficient GLA protein.
  • A Phase I/IIa clinical trial is underway in the U.S. to assess the safety, tolerability, and early efficacy of AMT-191, with initial data expected in 2025.
  • Orphan Drug Designation provides uniQure with incentives, including tax credits and market exclusivity, to support the development of AMT-191.

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316RecruitingPhase 1
UniQure Biopharma B.V.
Posted 6/5/2024

Heidelberg Pharma's HDP-101 Shows Promise in Relapsed Multiple Myeloma

  • Heidelberg Pharma's HDP-101, an anti-BCMA antibody-drug conjugate, demonstrates biological activity in relapsed or refractory multiple myeloma patients.
  • Early data from a Phase I/IIa trial shows objective improvement in disease, with one patient achieving partial remission in cohort five.
  • The ongoing trial is evaluating HDP-101's safety and efficacy, with further data expected at upcoming scientific conferences.
  • Heidelberg Pharma will host an R&D webinar on October 15, 2024, to discuss the HDP-101 clinical trial results and future development plans.

Prestige BioPharma's Tuznue Receives European Commission Approval as Herceptin Biosimilar

  • Prestige BioPharma's Tuznue (trastuzumab) has been granted final approval by the European Commission as a biosimilar to Herceptin, a drug used to treat breast and metastatic gastric cancers.
  • Tuznue is the third trastuzumab biosimilar approved in Europe, positioning Prestige BioPharma to compete in a market valued at approximately $4.1 billion annually.
  • The company plans to leverage Tuznue's approval to accelerate marketing authorization for its Avastin biosimilar, HD204 (bevacizumab), currently undergoing Phase 3 trial analysis.
  • Prestige BioPharma anticipates generating revenue through licensing agreements with global pharmaceutical companies and aims for price competitiveness to capture market share.

Fruquintinib Approved in Japan for Previously Treated Metastatic Colorectal Cancer

  • Takeda's Fruzaqla (fruquintinib) has been approved in Japan for advanced or recurrent colorectal cancer (CRC) after chemotherapy.
  • The approval is based on the Phase III FRESCO-2 trial, which demonstrated statistically significant improvements in overall and progression-free survival.
  • Fruquintinib is the first novel targeted therapy approved in Japan for metastatic CRC, regardless of biomarker status, in over a decade.
  • This approval expands the global availability of fruquintinib, offering a new treatment option for patients with limited alternatives.

A Study of Efficacy and Safety of Fruquintinib (HMPL-013) in Participants With Metastatic Colorectal Cancer

NCT04322539CompletedPhase 3
Hutchison Medipharma Limited
Posted 8/12/2020

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