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Clinical Trial News

Avutometinib-Defactinib Combination Shows 45% Response Rate in Low-Grade Serous Ovarian Cancer

Targeted TherapyOncology
  • A phase II trial of avutometinib combined with defactinib demonstrated a 45% response rate in patients with advanced low-grade serous ovarian cancer, nearly twice as effective as current best treatments.
  • Patients with KRAS mutations showed particularly strong responses at 60%, while those without mutations still achieved a 29% response rate, both significantly higher than standard therapy response rates of 0-14%.
  • The dual RAF/MEK inhibitor combination proved over four times more effective than avutometinib alone, with previous phase I data showing an average progression-free survival of 23 months.
  • Low-grade serous ovarian cancer affects approximately 700 women annually in the UK and represents about 10% of all ovarian cancer cases, typically affecting younger women with poor response to conventional treatments.

Phase I Trial of Defactinib and VS-6766.

NCT03875820Active, Not RecruitingPhase 1
Institute of Cancer Research, United Kingdom
Posted 12/12/2017

A Study of Avutometinib (VS-6766) V. Avutometinib (VS-6766) + Defactinib in Recurrent Low-Grade Serous Ovarian Cancer with and Without a KRAS Mutation

NCT04625270Active, Not RecruitingPhase 2
Verastem, Inc.
Posted 12/21/2020

Trametinib in Treating Patients With Recurrent or Progressive Low-Grade Ovarian Cancer or Peritoneal Cavity Cancer

NCT02101788Active, Not RecruitingPhase 2
National Cancer Institute (NCI)
Posted 2/27/2014

A Study of MEK162 vs. Physician's Choice Chemotherapy in Patients With Low-grade Serous Ovarian, Fallopian Tube or Peritoneal Cancer

NCT01849874TerminatedPhase 3
Pfizer
Posted 6/27/2013

Vorasidenib Shows Promise as First Targeted Therapy for IDH-Mutant Low-Grade Gliomas

Targeted TherapyDrug ApprovalOncology
  • Vorasidenib, the first targeted therapy developed specifically for brain cancer, more than doubled progression-free survival in patients with recurrent grade 2 glioma carrying IDH1/IDH2 mutations, extending the time without disease progression from 11.1 months to 27.7 months.
  • The international INDIGO trial involving 331 patients demonstrated that vorasidenib delayed the need for chemotherapy and radiation by nearly 17 months compared to placebo, with 85.6% of patients going 18 months before requiring next treatment.
  • The drug showed excellent tolerability with limited adverse effects, offering a new treatment option for younger patients typically in their 30s and 40s who face cognitive deficits from standard radiation and chemotherapy treatments.
  • Results from this phase 3 study, published in the New England Journal of Medicine and presented at ASCO, are expected to establish a new standard of care for IDH-mutant low-grade gliomas pending FDA approval.

Sanofi to Present New Oncology Data at ASCO 2023

Sanofi will showcase new data from its oncology pipeline at the ASCO 2023 Annual Meeting, including a first-in-human study of SAR443579, a novel NK cell engager for hematological malignancies, which has received Fast Track Designation by the U.S. FDA.

U.S. Tuberous Sclerosis Drug Market Projected to Reach $1.25 Billion by 2030, Growing at 23.1% CAGR

Drug ApprovalNeurologyRare Disease
  • The U.S. tuberous sclerosis drug market is expected to grow from $237.2 million in 2022 to $1.25 billion by 2030, driven by FDA approvals of new treatments and increasing disease prevalence.
  • FDA-approved treatments include Afinitor (everolimus) for patients aged 2 and above, approved in 2018, and Epidiolex (cannabidiol) for patients aged 1 and above, approved in 2020.
  • mTOR inhibitors are gaining significant traction in treatment protocols, showing effectiveness for internal tumors and skin lesions, with FDA approval for treating lung complications like Lymphangioleiomyomatosis (LAM) in 2015.

VarmX Secures €30M Series B2 Funding to Advance Anticoagulation Reversal Drug VMX-C001 Toward Pivotal Trial

  • VarmX raised €30 million in Series B2 financing led by Sound Bioventures to advance its lead compound VMX-C001 for anticoagulation reversal.
  • The funding will enable the company to obtain IND approval and complete preparations for pivotal clinical trials, including large-scale manufacturing.
  • VMX-C001 is a modified recombinant human blood clotting factor X designed to reverse bleeding in patients taking Direct Oral Anticoagulant blood thinners.
  • The company has completed enrollment in its first-in-human study and will present initial data at the International Society on Thrombosis and Haemostasis conference.

Avacta Acquires Belgium-Based Coris BioConcept for £7.4 Million to Expand Diagnostics Portfolio

  • Avacta Group acquired Coris BioConcept, a Belgium-based rapid diagnostic test manufacturer, for £7.4 million upfront with potential additional payments up to £3.0 million based on performance.
  • The acquisition adds a broad portfolio of professional-use rapid diagnostic tests for respiratory, gastroenteric, and blood-borne pathogens, including antibiotic resistance detection capabilities.
  • This strategic move complements Avacta's October 2022 acquisition of Launch Diagnostics and advances the company's goal of establishing a full-spectrum in-vitro diagnostics business.
  • Coris generated £4.6 million in revenue for 2022 with approximately 50% gross margins and operates from a newly constructed 10,700 ft² facility in Gembloux, Belgium.

Ropinirole Shows Promise in ALS Treatment by Extending Progression-Free Survival in Phase 1/2a Trial

  • A Phase 1/2a trial of ropinirole in sporadic ALS patients demonstrated acceptable safety and tolerability over 24 weeks.
  • While the ALSFRS-R decline was not significantly different during the double-blind phase, muscle strength and daily activity were maintained.
  • The open-label extension showed significant suppression of ALSFRS-R decline and an additional 27.9 weeks of disease-progression-free survival in the ropinirole group.
  • The study suggests a potential therapeutic effect of ropinirole in ALS, possibly involving the SREBP2-cholesterol pathway, warranting further investigation.

Clinical Trials in Essential Thrombocythemia (ET)

PharmaEssentia is conducting clinical trials for Essential Thrombocythemia (ET), focusing on the efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft compared with anagrelide as second-line therapy for adult patients resistant or intolerant to hydroxyurea.

Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance

NCT04285086Active, Not RecruitingPhase 3
PharmaEssentia
Posted 8/25/2020

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET

NCT05482971Active, Not RecruitingPhase 2
PharmaEssentia
Posted 9/29/2022

FDA Approves Posluma, First Radiohybrid PSMA-Targeted PET Imaging Agent for Prostate Cancer

  • The FDA has approved flotufolastat F 18 injection (Posluma) for PET imaging of PSMA-positive lesions in prostate cancer patients, aiding in metastasis detection and recurrence assessment.
  • Posluma, developed by Blue Earth Diagnostics, utilizes radiohybrid technology to target PSMA, offering high specificity and detection rates, even at low PSA levels, as shown in LIGHTHOUSE and SPOTLIGHT trials.
  • Clinical trials demonstrated Posluma's ability to accurately identify pelvic lymph nodes and distant metastatic lesions, with a high overall patient-level detection rate of 99% in recurrence cases.
  • The agent, expected to be commercially available in June 2023, offers a new tool for physicians in managing prostate cancer by improving the detection and localization of the disease.

Imaging Study to Investigate Safety and Diagnostic Performance of 18F rhPSMA 7.3 PET Ligand in Suspected Prostate Cancer Recurrence

NCT04186845CompletedPhase 3
Blue Earth Diagnostics
Posted 5/4/2020

Imaging Study to Investigate the Safety and Diagnostic Performance of rhPSMA 7.3 (18F) in Newly Diagnosed Prostate Cancer.

NCT04186819CompletedPhase 3
Blue Earth Diagnostics
Posted 3/2/2020

Study of 177Lu-PSMA-617 In Metastatic Castrate-Resistant Prostate Cancer

NCT03511664CompletedPhase 3
Endocyte
Posted 5/29/2018

SUNLIGHT and FRESCO-2 Trials Show Promising Results for Refractory Colorectal Cancer Treatment

Recent phase 3 trials, SUNLIGHT and FRESCO-2, have demonstrated significant improvements in overall and progression-free survival for patients with refractory colorectal cancer, offering new hope and potential standards of care in third-line treatment settings.

Panitumumab and RAS, Diagnostically-useful Gene Mutation for mCRC

NCT02394795CompletedPhase 3
Takeda
Posted 5/29/2015

Phase III Study of Trifluridine/Tipiracil With and Without Bevacizumab in Refractory Metastatic Colorectal Cancer Patients

NCT04737187CompletedPhase 3
Taiho Oncology, Inc.
Posted 11/25/2020

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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