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Passage Bio's PBFT02 Shows Sustained Progranulin Increase in FTD-GRN Patients

• Passage Bio's PBFT02 gene therapy demonstrated robust and sustained increases in CSF progranulin levels in FTD-GRN patients in the upliFT-D trial. • Dose 1 of PBFT02 was well-tolerated in patients receiving a revised immunosuppression regimen, with no serious adverse events reported. • The Phase 1/2 upliFT-D trial results support PBFT02's potential as a progranulin-raising therapy for neurodegenerative diseases. • CSF progranulin levels remained elevated up to 12 months post-treatment, exceeding levels found in healthy adult controls.

A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes

NCT04747431RecruitingPhase 1
Passage Bio, Inc.
Posted 9/14/2021

iValve: Novel Mechanical Heart Valve Mimics Tissue Valve Performance, Aims for Pediatric Use

  • Researchers at UBC Okanagan have developed the iValve, a novel mechanical heart valve designed to combine the durability of mechanical valves with the superior performance of tissue valves.
  • The iValve is specifically designed for high-heart-rate applications, making it particularly suitable for pediatric patients who have historically faced challenges with existing mechanical valve technology.
  • The new valve has demonstrated improved hemodynamic performance in lab tests, suggesting it could reduce the need for lifelong anticoagulant therapy typically required with mechanical valves.
  • Researchers plan to proceed with animal and clinical trials within two years, with future work focused on developing similar valves for mitral valve replacement.

Kisqali Demonstrates Sustained Benefit in HR+/HER2- Early Breast Cancer

• Updated analysis of the NATALEE trial shows Kisqali plus endocrine therapy reduces recurrence risk by 28.5% compared to endocrine therapy alone in stage II and III HR+/HER2- early breast cancer. • The invasive disease-free survival benefit of Kisqali was consistent across all pre-specified patient subgroups, including those with node-negative disease. • Four-year post-hoc analysis presented at ESMO Congress 2024 also showed consistent results across secondary efficacy endpoints, with a trend for improvement in overall survival. • Kisqali, when added to endocrine therapy, demonstrates a deepening benefit beyond the three-year treatment period in patients with HR+/HER2- early breast cancer.

Imfinzi and Imjudo Combo Shows Unprecedented Survival in Liver Cancer

  • The combination of Imfinzi and Imjudo demonstrated an unprecedented overall survival rate in advanced hepatocellular carcinoma (HCC).
  • The STRIDE regimen, combining a single dose of Imjudo with Imfinzi, showed one in five patients surviving five years, a significant milestone.
  • The safety profile of the STRIDE regimen was consistent with the known profiles of each medicine, with manageable adverse events.
  • Imfinzi plus Imjudo is approved for advanced or unresectable HCC in the US, EU, Japan, and other countries, offering a new treatment option.

Strides Pharma Gains FDA Approval for Generic Fluoxetine 60mg Tablets

  • Strides Pharma has received FDA approval for its generic Fluoxetine 60mg tablets, expanding its portfolio of this antidepressant drug.
  • The approval allows Strides Pharma to offer a complete range of Fluoxetine products, including 10mg, 20mg, and 60mg tablets and capsules.
  • Fluoxetine capsules and tablets have a combined market size of $130 million, offering a substantial commercial opportunity for Strides Pharma.
  • Strides Pharma plans to launch all three strengths of Fluoxetine tablets in the near future, manufactured at its facility in Puducherry.

PDS Biotech's Versamune® HPV Plus Pembrolizumab Shows Promising Results in HPV16-Positive Head and Neck Cancer

  • Updated data from the VERSATILE-002 trial shows a median overall survival of 30 months in patients treated with Versamune® HPV plus pembrolizumab.
  • The combination therapy achieved an objective response rate of 36% and a disease control rate of 77% in patients with HPV16-positive recurrent/metastatic HNSCC.
  • A significant portion of patients, 21%, experienced deep tumor responses with 90-100% tumor shrinkage, highlighting the therapy's potential.
  • PDS Biotech plans to initiate the VERSATILE-003 Phase 3 clinical trial to further evaluate the combination as a first-line treatment option.

Nerandomilast Meets Primary Endpoint in Phase III FIBRONEER-IPF Trial

• Boehringer Ingelheim's nerandomilast met the primary endpoint in the Phase III FIBRONEER-IPF trial, showing a significant change in Forced Vital Capacity (FVC) at week 52 compared to placebo. • The FIBRONEER-IPF trial, the largest IPF trial to date, recruited patients from over 330 sites across more than 30 countries, demonstrating broad global participation. • Nerandomilast, an oral phosphodiesterase 4B (PDE4B) inhibitor, has received FDA Breakthrough Therapy Designation for IPF, with a new drug application planned for submission. • Full efficacy and safety data from the FIBRONEER-IPF trial will be presented in the first half of 2025, offering a comprehensive understanding of the drug's potential.

A Study to Find Out Whether BI 1015550 Improves Lung Function in People With Progressive Fibrosing Interstitial Lung Diseases (PF-ILDs)

NCT05321082CompletedPhase 3
Boehringer Ingelheim
Posted 11/16/2022

A Study to Find Out Whether BI 1015550 Improves Lung Function in People With Idiopathic Pulmonary Fibrosis (IPF)

NCT05321069CompletedPhase 3
Boehringer Ingelheim
Posted 10/6/2022

FDA Approves Roche's Ocrevus Zunovo for Multiple Sclerosis with Halozyme's Enhanze Technology

  • The FDA has approved Roche's Ocrevus Zunovo, a subcutaneous formulation for relapsing and primary progressive multiple sclerosis, utilizing Halozyme's Enhanze drug delivery technology.
  • Ocrevus Zunovo offers a twice-yearly, approximately ten-minute subcutaneous injection, providing greater flexibility for healthcare providers and patients based on individual needs.
  • The approval is based on the Phase III OCARINA II trial, which demonstrated non-inferior levels of Ocrevus in the bloodstream and comparable safety and efficacy to the intravenous formulation.
  • The trial showed a 97% effectiveness rate in suppressing relapse activity and MRI lesions through 48 weeks, with over 92% of participants reporting satisfaction with the subcutaneous administration.

Nerandomilast Meets Primary Endpoint in Phase III FIBRONEER-IPF Trial for Idiopathic Pulmonary Fibrosis

• Nerandomilast met the primary endpoint in the FIBRONEER-IPF trial, demonstrating a statistically significant absolute change from baseline in Forced Vital Capacity (FVC) at week 52 compared to placebo. • The FIBRONEER-IPF trial, involving over 1177 patients across more than 30 countries, stands as the largest IPF trial conducted to date. • Boehringer Ingelheim plans to submit a new drug application to the FDA and other health authorities based on these positive results, seeking approval for nerandomilast in treating IPF. • Nerandomilast, an oral phosphodiesterase 4B (PDE4B) inhibitor, has received Breakthrough Therapy Designation from the FDA for IPF, highlighting its potential to address unmet needs.

A Study to Find Out Whether BI 1015550 Improves Lung Function in People With Progressive Fibrosing Interstitial Lung Diseases (PF-ILDs)

NCT05321082CompletedPhase 3
Boehringer Ingelheim
Posted 11/16/2022

A Study to Find Out Whether BI 1015550 Improves Lung Function in People With Idiopathic Pulmonary Fibrosis (IPF)

NCT05321069CompletedPhase 3
Boehringer Ingelheim
Posted 10/6/2022

Epitomee Medical's Weight Loss Pill Receives FDA Approval, Stock Surges

  • Epitomee Medical's weight loss pill has secured FDA approval, marking a significant milestone for the company in a competitive market.
  • Following the FDA's decision, Epitomee's stock experienced a substantial surge of 200% within two days, reflecting investor optimism.
  • The company now faces the challenge of establishing its product in a crowded weight loss market, requiring strategic market positioning.
  • This approval provides a new option for individuals seeking medical interventions for weight management, addressing a significant health need.

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