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Clinical Trial News

Oral Gildeuretinol Acetate Fails to Meet Primary Endpoint in Phase 3 Geographic Atrophy Trial

  • Alkeus Pharmaceuticals' oral gildeuretinol acetate (ALK-001) did not meet the primary endpoint of reducing geographic atrophy (GA) lesion growth rate in the SAGA phase 3 study.
  • The SAGA study enrolled 198 patients with GA secondary to age-related macular degeneration, assessing GA lesion growth over 24 months.
  • Gildeuretinol showed a 0.25 mm²/year reduction in GA lesion growth compared to placebo, considered "clinically meaningful" by Alkeus, and significantly reduced low luminance visual acuity loss (p = 0.03).
  • Alkeus plans to discuss the SAGA data with regulatory agencies to determine the best path forward for gildeuretinol, a deuterated vitamin A analog.

PlaqueTec and RxCelerate Collaborate on Novel Drug Discovery for Coronary Artery Disease

  • PlaqueTec and RxCelerate successfully completed a collaboration to assess small molecules against a novel target in coronary artery disease (CAD).
  • RxCelerate developed a bespoke cell-based assay to test small molecules predicted to bind PlaqueTec's candidate target.
  • The collaboration characterized lead compounds, demonstrating the assay's value in PlaqueTec's CAD endotyping mission.
  • Further collaboration is planned to investigate additional lead compounds and targets identified in PlaqueTec's BIOPATTERN trial.

TiNivo-2 Trial: Nivolumab Fails to Improve Outcomes in Advanced RCC After Prior ICI Therapy

• The Phase 3 TiNivo-2 trial evaluated tivozanib plus nivolumab versus tivozanib alone in patients with renal cell carcinoma (RCC) who progressed on prior immune checkpoint inhibitor (ICI) therapy. • The study found no significant difference in progression-free survival (PFS), overall survival (OS), or response rate between the combination and tivozanib monotherapy arms. • These results, along with previous data, suggest that re-challenging with an ICI in metastatic RCC should be approached with caution, except in specific scenarios like long duration from prior ICI. • The combination arm used a lower dose of tivozanib due to hypertension concerns, which may have impacted the results.

Kesimpta Demonstrates Sustained Efficacy in Relapsing Multiple Sclerosis

  • Kesimpta (ofatumumab) shows promise in reducing disability progression and disease activity in patients with relapsing multiple sclerosis (RMS) based on ALITHIOS and OLIKOS trial data.
  • Treatment-naïve RMS patients on continuous Kesimpta were more likely to avoid six-month confirmed disability worsening compared to those switching from Aubagio (83.4% vs. 76.3%).
  • The OLIKOS trial found no new gadolinium-enhancing T1 lesions at 12 months in clinically stable RMS patients switching from IV anti-CD20 therapy to Kesimpta.
  • Continuous Kesimpta led to fewer disability worsening events and a greater likelihood of being progression-free, especially when initiated early in the disease course.

FDA Grants Orphan Drug Designation to CF33-hNIS for Cholangiocarcinoma

  • The FDA has granted orphan drug designation to CF33-hNIS (Vaxinia) for treating cholangiocarcinoma, a rare cancer with limited therapeutic options.
  • CF33-hNIS, an oncolytic virotherapy, is being investigated in the phase 1 MAST trial as a monotherapy and in combination with pembrolizumab for advanced solid tumors.
  • Early data from the MAST trial showed promising results, including an objective response rate of 14% and a disease control rate of 86% in monotherapy arm.
  • The orphan drug designation provides Imugene with incentives such as tax credits and market exclusivity to support the development of CF33-hNIS.

A Study of CF33-hNIS (VAXINIA), an Oncolytic Virus, as Monotherapy or in Combination With Pembrolizumab in Adults With Metastatic or Advanced Solid Tumors

NCT05346484RecruitingPhase 1
Imugene Limited
Posted 5/17/2022

NIH-Funded Study to Investigate Type 1 Diabetes' Impact on Children's Brain Development

  • A nationwide longitudinal study, funded by a $2.7 million NIH grant, will explore the effects of type 1 diabetes on brain development in children.
  • The multi-site study will involve 12 research centers across the U.S. and recruit over 1,000 children newly diagnosed with type 1 diabetes.
  • Researchers aim to identify factors that may accelerate or mitigate the risk of brain-related complications in children with type 1 diabetes.
  • The study will use fMRI and collect data on academic performance, memory, attention, and neurobehavioral health to refine clinical guidelines.

RNF114 Protein Identified as Potential Target for Cataract Drug Development

  • Researchers have identified the RNF114 protein as a potential target for non-surgical cataract treatment, offering a possible alternative to surgery.
  • The study, conducted on ground squirrels and rats, demonstrated that RNF114 is significantly elevated during the reversal of cold-induced cataracts.
  • Pretreatment with RNF114 led to rapid clearing of cataracts in rat lenses exposed to low temperatures, suggesting a novel therapeutic approach.
  • These findings provide proof-of-principle for inducing cataract clearance in animals, warranting further research into targeted protein degradation strategies.

Io Therapeutics' IRX4204 Shows Promise in Combating Neurodegeneration Linked to Normal Aging

• Io Therapeutics presented data on IRX4204, a Phase II RXR agonist, at the FASEB conference, highlighting its potential to prevent and treat neurodegeneration associated with normal aging. • Studies reveal IRX4204's ability to inhibit brain inflammation by reducing IL-6 production and modulating Treg and Th17 cell activity, addressing key aspects of neuro-inflammaging. • IRX4204 promotes myelin-producing cell development, repairs damaged nerve fibers, and enhances neuron survival, demonstrating multifaceted benefits for age-related brain decline. • Io Therapeutics plans a Phase II clinical trial of IRX4204 in Parkinson’s disease in Q1 2025 and is considering exploratory trials for normal-aging related neurodegeneration.

Durvalumab Demonstrates Improved Survival in Liver and Bladder Cancers

  • Durvalumab plus tremelimumab showed a 24% reduction in death risk versus sorafenib in unresectable hepatocellular carcinoma (HCC), with 19.6% alive at 5 years versus 9.4%.
  • In muscle-invasive bladder cancer (MIBC), durvalumab with chemotherapy reduced disease progression, recurrence, or death by 32% compared to chemotherapy alone.
  • Durvalumab-based regimens also improved overall survival in MIBC, reducing the risk of death by 25% compared to neoadjuvant chemotherapy and radical cystectomy.

Durvalumab+ Gemcitabine/Cisplatin (Neoadjuvant Treatment) and Durvalumab (Adjuvant Treatment) in Patients With MIBC

NCT03732677Active, Not RecruitingPhase 3
AstraZeneca
Posted 11/16/2018

Study of Durvalumab and Tremelimumab as First-line Treatment in Patients With Advanced Hepatocellular Carcinoma

NCT03298451Active, Not RecruitingPhase 3
AstraZeneca
Posted 10/11/2017

Datopotamab Deruxtecan Shows Promise in Advanced Gynecologic Cancers

  • Datopotamab deruxtecan (Dato-DXd) demonstrates encouraging antitumor activity in patients with advanced ovarian cancer post-platinum chemotherapy.
  • In endometrial cancer, Dato-DXd showed a confirmed objective response rate of 27.5% with a median duration of response of 16.4 months.
  • The TROPION-PanTumor03 study indicates Dato-DXd has a manageable safety profile, with mostly low-grade stomatitis and nausea reported.
  • These findings suggest Dato-DXd could be a potential treatment option for patients with limited alternatives after platinum-based therapies.

Study of Dato-Dxd as Monotherapy and in Combination With Anti-cancer Agents in Patients With Advanced Solid Tumours (TROPION-PanTumor03)

NCT05489211RecruitingPhase 2
AstraZeneca
Posted 9/6/2022

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