MedPath

Clinical Trial News

Aducanumab Approval and Medicare Coverage: Implications for Alzheimer's Treatment

• The FDA's approval of aducanumab, an antiamyloid antibody for early Alzheimer's, sparked controversy due to unclear clinical benefits and high cost. • CMS restricted Medicare coverage to patients in clinical trials, citing limited evidence and significant adverse effects, creating access barriers. • Cost-effectiveness analyses suggest aducanumab's price is not justified by its clinical benefits, with ARIA-related costs adding to the financial burden. • The aducanumab case impacts future Alzheimer's drug development, approval standards, and Medicare coverage policies for antiamyloid therapies.

Insilico Medicine's AI-Designed COVID-19 Drug Enters Clinical Trials

• Insilico Medicine's ISM3312, an AI-designed therapeutic, has entered clinical trials in China after promising preclinical results. • The drug targets the 3CLpro protease in SARS-CoV-2, potentially effective against current and future variants and other coronaviruses. • Preclinical studies showed ISM3312 significantly reduces viral load and lung inflammation, offering a potential advantage over existing treatments. • While promising, experts caution that it is still early to determine the drug's safety and efficacy in humans.

Socazolimab Plus Chemotherapy Shows Promise in Extensive-Stage Small Cell Lung Cancer

  • A Phase 1b trial indicates that socazolimab, combined with etoposide and carboplatin, demonstrates potential survival benefits in treating extensive-stage small cell lung cancer (ES-SCLC).
  • The study reported a median progression-free survival (PFS) of 5.65 months and a median overall survival (OS) of 14.88 months in ES-SCLC patients.
  • Socazolimab's immunogenicity was evaluated, revealing a higher incidence of antidrug antibodies (ADAs) compared to other PD-1/PD-L1 inhibitors, though without significant impact on safety or response.
  • The safety profile of socazolimab combined with chemotherapy was manageable, with most grade 3 or 4 treatment-related adverse events (TRAEs) occurring during the combination treatment period.

A Study of IBI110 in Combination With Sintilimab and Chemotherapy in Patients With Untreated Extensive-Stage Small Cell Lung Cancer

NCT05026593CompletedPhase 1
Innovent Biologics (Suzhou) Co. Ltd.
Posted 9/7/2021

Study of ZKAB001 Combined With Carboplatin and Etoposide in the Extensive Small Cell Lung Cancer

NCT04346914Unknown StatusPhase 1
Lee's Pharmaceutical Limited
Posted 3/8/2020

Safety and Efficacy Study of First-line Treatment With QL1706 Plus Chemotherapy in Extensive-Stage Small Cell Lung Cancer

NCT05309629CompletedPhase 2
Qilu Pharmaceutical Co., Ltd.
Posted 4/18/2022

A Study of Carboplatin Plus Etoposide With or Without ZKAB001 (Anti-PD-L1 Antibody) in Patients With ES-SCLC

NCT04878016CompletedPhase 3
Lee's Pharmaceutical Limited
Posted 7/15/2021

Survival Outcomes in BRAF V600E-Mutated Advanced Colorectal Cancer: Chemotherapy Alone vs. Chemotherapy Combined with Bevacizumab

A study comparing the survival outcomes of patients with BRAF V600E-mutated advanced colorectal cancer (CRC) treated with chemotherapy alone versus chemotherapy combined with bevacizumab found no significant difference in overall survival (OS) and progression-free survival (PFS) between the two groups. The research highlights the need for new treatment options for this patient population, as current therapies do not significantly improve survival rates.

A Study of Encorafenib Plus Cetuximab With or Without Chemotherapy in People With Previously Untreated Metastatic Colorectal Cancer

NCT04607421Active, Not RecruitingPhase 3
Pfizer
Posted 12/21/2020

Encorafenib, Binimetinib and Cetuximab in Subjects With Previously Untreated BRAF-mutant ColoRectal Cancer

NCT03693170CompletedPhase 2
Pierre Fabre Medicament
Posted 1/17/2019

Ultragenyx's UX111 Gene Therapy Shows Sustained Efficacy in MPS IIIA Patients

• UX111, an AAV9-based gene therapy, demonstrates rapid and sustained reduction of cerebrospinal fluid biomarker levels in MPS IIIA patients treated early. • A phase 1/2/3 clinical trial (Transpher A) showed dose-dependent efficacy, with the highest dose cohort (3 x 10^13 vg/kg) exhibiting the most significant biomarker reduction. • The gene therapy has shown neurodevelopmental gains in treated patients, with no drug-related serious adverse events reported in the trial. • UX111, acquired by Ultragenyx from Abeona Therapeutics, has received multiple designations, including regenerative medicine advanced therapy and orphan drug status.

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

NCT04360265Unknown StatusPhase 3
Ultragenyx Pharmaceutical Inc
Posted 9/28/2020

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCT02716246RecruitingPhase 2
Ultragenyx Pharmaceutical Inc
Posted 4/25/2016

Avutometinib Plus Defactinib Shows Promise in Recurrent Low-Grade Serous Ovarian Cancer

  • The combination of avutometinib and defactinib demonstrated a 28% confirmed objective response rate in patients with recurrent low-grade serous ovarian cancer.
  • Patients with both KRAS-mutated and KRAS wild-type disease showed similar response rates of 27% and 29%, respectively, when treated with the combination therapy.
  • The disease control rate with the combination therapy was notably high at 93%, with a significant proportion of patients remaining on treatment after 5 months.
  • Verastem Oncology plans to use data from the RAMP-201 and FRAME trials to potentially support accelerated approval of avutometinib plus defactinib.

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

Phase I Trial of Defactinib and VS-6766.

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

Blue Lake Biotechnology's Nasal COVID-19 Vaccine Shows 86% Efficacy in Phase 1 Trial

  • Blue Lake Biotechnology's experimental nasal COVID-19 vaccine demonstrated 86% efficacy in reducing symptomatic infections for three months in Phase 1 trials, significantly outperforming existing mRNA boosters.
  • The vaccine uses a modified parainfluenza virus platform to deliver coronavirus spike protein directly to the nasal cavity, creating targeted immune protection at the virus entry point.
  • Phase 1 results from 72 participants showed superior tolerability with minimal side effects compared to traditional injected vaccines, with the company planning to expand to 400 participants in upcoming Phase 2 trials.
  • The nasal delivery approach represents a promising next-generation vaccine strategy that could provide more durable and effective protection against respiratory viruses.

AstraZeneca Completes $1.3 Billion Acquisition of CinCor Pharma for Hypertension Drug Baxdrostat

  • AstraZeneca has successfully completed its $1.3 billion acquisition of CinCor Pharma, gaining access to baxdrostat, a novel aldosterone synthase inhibitor for treatment-resistant hypertension.
  • Baxdrostat demonstrated statistically significant blood pressure reduction in Phase II trials for treatment-resistant hypertension, with Phase III trials planned for the first half of 2023.
  • The acquisition strengthens AstraZeneca's cardiorenal pipeline and offers potential combination opportunities with Farxiga to address high unmet medical needs in cardiovascular and kidney diseases.
  • The transaction includes a potential additional $500 million contingent payment upon regulatory submission, bringing the total deal value to approximately $1.8 billion if achieved.

Methadone vs. Buprenorphine: Landmark Trial to Evaluate Opioid Addiction Treatments in Fentanyl Era

  • A new clinical trial will compare methadone and buprenorphine for opioid addiction treatment in office settings, a first in the fentanyl era.
  • The study addresses the need for expanded methadone access, as buprenorphine is proving less effective for some patients amid the opioid crisis.
  • Researchers will assess patient retention in treatment when methadone is prescribed in primary care, potentially changing access policies.
  • The trial anticipates increased flexibility in methadone access, aiming to inform clinical decisions if office-based dispensing becomes available.

Metformin's Repurposing as Cancer Therapeutic: New Avenues and Future Directions

  • Recent trials show limited efficacy of metformin as a treatment for established cancers, leading to a decline in enthusiasm for further clinical studies.
  • Emerging data suggests potential for metformin in combination with immunotherapy by remodeling the hypoxic tumor microenvironment and enhancing tumor immunosurveillance.
  • Studies indicate metformin's role in cancer prevention, particularly in high-risk populations like those with Li-Fraumeni syndrome, warranting further clinical investigation.
  • Future research should focus on patient selection, understanding the mechanism of action, and identifying appropriate drug combinations to maximize metformin's therapeutic potential.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.