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Clinical Trial News

Govorestat NDA for Classic Galactosemia Faces No FDA Advisory Committee, PDUFA Date on Track

  • Applied Therapeutics' govorestat, a CNS penetrant Aldose Reductase Inhibitor, is under NDA review for treating Classic Galactosemia, a rare metabolic disorder.
  • The FDA has communicated that an Advisory Committee meeting, previously scheduled for October 9, 2024, will no longer be required for the govorestat NDA review.
  • The FDA's Priority Review of the govorestat NDA is proceeding as planned, with alignment on post-marketing requirements expected in October 2024.
  • The PDUFA target action date for the FDA's decision on govorestat remains on track for November 28, 2024, signaling potential approval by the end of the year.

Pomalidomide Shows Promise in Reducing Bleeding for Hereditary Hemorrhagic Telangiectasia

  • A Phase II clinical trial (PATH-HHT) demonstrated that pomalidomide significantly reduces the severity of nosebleeds in patients with hereditary hemorrhagic telangiectasia (HHT).
  • The study, published in The New England Journal of Medicine, showed a notable improvement in the HHT-specific quality of life for patients treated with pomalidomide.
  • Pomalidomide was generally well-tolerated, although some patients experienced side effects such as neutropenia, constipation, and rash, leading to dose adjustments.
  • These findings suggest pomalidomide may offer a new therapeutic avenue for HHT, a bleeding disorder with limited FDA-approved treatment options.

Pomalidomide for the Treatment of Bleeding in HHT

NCT03910244CompletedPhase 2
The Cleveland Clinic
Posted 10/17/2019

Lenvatinib Plus Pembrolizumab Demonstrates Significant Antitumor Activity in Advanced Endometrial Cancer

  • Lenvatinib plus pembrolizumab shows substantial antitumor activity in patients with stage III/IV recurrent endometrial carcinoma, according to the ENGOT-en9/LEAP-001 study.
  • The combination therapy achieved an objective response rate of 55.7% in all patients and 50.6% in the proficient mismatch repair (pMMR) population.
  • Median duration of response was 16.1 months for all-comers and 23.2 months for the pMMR population, indicating deep and durable responses.
  • Adverse events were manageable with dose modifications and supportive care, supporting the clinical utility of this combination in advanced endometrial cancer.

Pembrolizumab (MK-3475) Plus Lenvatinib (E7080/MK-7902) Versus Chemotherapy for Endometrial Carcinoma (ENGOT-en9 / MK-7902-001)

NCT03884101CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 4/11/2019

4DMT's 4D-150 Gene Therapy Shows Promise in Wet AMD, Phase 3 Trial Planned

  • 4D Molecular Therapeutics' 4D-150 demonstrated an 89% reduction in the need for standard-of-care injections in wet AMD patients over 52 weeks.
  • In a subset of recently diagnosed patients, 4D-150 led to a 98% reduction in injections, with 87% remaining injection-free during the study period.
  • 4DMT is advancing 4D-150 into a Phase 3 program (4FRONT) with two non-inferiority trials comparing it to aflibercept, with the first study expected to begin in Q1 2025.
  • The gene therapy has shown a favorable safety profile, with intraocular inflammation rates comparable to existing anti-VEGF treatments.

Palliative Radiotherapy Plus Best Supportive Care Reduces Pain in Hepatic Cancer

  • A phase III trial showed that adding a single fraction of palliative radiotherapy to best supportive care significantly reduced pain in patients with painful hepatic cancer.
  • 67% of patients in the radiotherapy plus best supportive care group experienced improvement in hepatic pain, compared to 22% in the best supportive care alone group.
  • Patients receiving radiotherapy plus best supportive care showed a median reduction in opioid use, while those receiving best supportive care alone had an increase.
  • The study suggests single-fraction radiotherapy plus best supportive care could be considered a standard palliative treatment for liver cancer.

Alzheimer's Theory Challenged: Brain Protein Levels, Not Plaques, Linked to Cognitive Decline

  • A new study challenges the prevailing theory that amyloid-beta plaque buildup causes Alzheimer's, suggesting that decreasing levels of the Aβ42 protein are linked to cognitive decline.
  • Researchers analyzed data from 26,000 participants in clinical trials for monoclonal antibody treatments, finding that higher Aβ42 levels correlated with slower cognitive impairment.
  • The study proposes that future Alzheimer's medications should focus on directly increasing Aβ42 levels, rather than solely targeting amyloid plaque reduction.
  • Experts emphasize the importance of addressing modifiable risk factors and exploring alternative mechanisms beyond amyloid for Alzheimer's disease.

Osimertinib's Expanding Role in EGFR-Mutated NSCLC Explored at ASCO 2024

  • The phase 3 LAURA trial demonstrated that osimertinib significantly improved progression-free survival in patients with stage III, EGFR-mutated NSCLC following chemoradiotherapy.
  • Minimal residual disease (MRD) monitoring in the phase 3 ADAURA trial may influence future treatment timelines for EGFR-mutated NSCLC, according to experts.
  • FLAURA2 trial results highlight the evolving role of osimertinib in advanced EGFR-mutated NSCLC, particularly concerning patients with brain metastases.
  • Experts discussed the potential impact of liquid biopsy data on treatment decisions for patients with EGFR-mutated NSCLC.

Study to Compare Furmonertinib to Platinum-Based Chemotherapy for Patients With Locally Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) With Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertion Mutations (FURVENT)

NCT05607550Active, Not RecruitingPhase 3
ArriVent BioPharma, Inc.
Posted 6/1/2023

AZD9291 Versus Placebo in Patients With Stage IB-IIIA Non-small Cell Lung Carcinoma, Following Complete Tumour Resection With or Without Adjuvant Chemotherapy.

NCT02511106Active, Not RecruitingPhase 3
AstraZeneca
Posted 10/21/2015

Study of Furmonertinib in Patients With Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) With Activating, Including Uncommon, Epidermal Growth Factor Receptor (EGFR) or Human Epidermal Growth Factor Receptor 2 (HER2) Mutations

NCT05364073Active, Not RecruitingPhase 1
ArriVent BioPharma, Inc.
Posted 6/30/2022

Alflutinib Mesylate Versus Gefitinib in Patients With Locally Advanced or Metastatic Non-small Cell Lung Cancer （FLAG）

NCT03787992Active, Not RecruitingPhase 3
Allist Pharmaceuticals, Inc.
Posted 5/30/2019

A Study of Osimertinib With or Without Chemotherapy as 1st Line Treatment in Patients With Mutated Epidermal Growth Factor Receptor Non-Small Cell Lung Cancer (FLAURA2)

NCT04035486Active, Not RecruitingPhase 3
AstraZeneca
Posted 7/2/2019

Study of FURMONERTINIB in Patients With NSCLC Having Exon 20 Insertion Mutation

NCT04858958Active, Not RecruitingPhase 1
Allist Pharmaceuticals, Inc.
Posted 8/10/2020

A Global Study to Assess the Effects of Osimertinib Following Chemoradiation in Patients With Stage III Unresectable Non-small Cell Lung Cancer (LAURA)

NCT03521154Active, Not RecruitingPhase 3
AstraZeneca
Posted 7/19/2018

Inspire Therapy Offers New Hope for Obstructive Sleep Apnea Patients at Phelps Health

  • Phelps Health now offers Inspire therapy, an FDA-approved treatment for obstructive sleep apnea (OSA) for those struggling with CPAP.
  • Inspire therapy uses mild stimulation to open the airway during sleep, improving oxygen flow and sleep quality.
  • The STAR clinical trial demonstrated significant reductions in sleep apnea events and improvements in quality-of-life measures with Inspire.
  • Inspire therapy represents a significant advancement in treating sleep apnea, offering a mask-free, hose-free alternative to CPAP.

Ossium Health Treats First Leukemia Patient with Cryopreserved Bone Marrow

  • Ossium Health has treated the first leukemia patient with cryopreserved, organ donor-derived bone marrow, aiming to improve transplant accessibility.
  • The treatment was administered to a 68-year-old acute myeloid leukemia patient at Henry Ford Health, showing neutrophil and platelet engraftment.
  • Ossium's approach seeks to address the shortage of matched living donors, especially for racial and ethnic minority groups, and accelerate transplant timelines.
  • The PRESERVE I trial (NCT05589896) is evaluating the safety and efficacy of this bone marrow in patients with hematologic malignancies.

A First-in-Human Study of HLA-Partially to Fully Matched Allogenic Cryopreserved Deceased Donor Bone Marrow Transplantation for Patients With Hematologic Malignancies

NCT05589896RecruitingPhase 1
Ossium Health, Inc.
Posted 8/16/2024

Vivos Therapeutics' DNA Appliance Receives FDA Clearance for Pediatric Sleep Apnea

  • Vivos Therapeutics received FDA 510(k) clearance for its DNA oral appliance to treat moderate to severe obstructive sleep apnea (OSA) and snoring in children aged 6-17.
  • Clinical trials showed a 50% reduction in sleep-related breathing disorder symptoms and a 58% decrease in snoring among pediatric patients using the Vivos DNA appliance.
  • The DNA appliance offers a non-invasive alternative to CPAP or surgical interventions, potentially capturing a significant share of the pediatric OSA market.
  • Vivos plans to integrate pediatric care into its marketing model, aiming to scale revenue quickly with this newly approved treatment option.

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