Clinical Trial News
Cretostimogene Shows High Complete Response Rate in BCG-Unresponsive Bladder Cancer
- Cretostimogene monotherapy achieved a 75.2% complete response rate in patients with high-risk, BCG-unresponsive non-muscle invasive bladder cancer (NMIBC).
- Durable responses were observed, with 29 patients maintaining complete response for 12 months or longer, suggesting a sustained therapeutic effect.
- The Phase 3 BOND-003 trial reported no Grade 3 or higher treatment-related adverse events, highlighting a favorable safety profile.
- Cretostimogene has received Fast Track and Breakthrough Therapy designations from the FDA, potentially expediting its path to regulatory approval.
Highlighted Clinical Trials:
CG Oncology, Inc.
Posted 10/27/2020
Evaluating mFARS in Pediatric Friedreich's Ataxia: Insights from the FACHILD Study
The FACHILD study focuses on evaluating the modified Friedreich Ataxia Rating Scale (mFARS) in pediatric patients with Friedreich's ataxia (FRDA), a rare hereditary condition. The study highlights the rapid disease progression in children, the challenges in assessing disease status using mFARS, and the need for pediatric trials to explore therapeutic interventions. It also discusses the impact of the COVID-19 pandemic on data collection and the importance of FARS E (Upright Stability) as a predictor of disease progression.
FDA Rejects Regeneron's Accelerated Approval Applications Due to Lack of Confirmatory Studies
- The FDA has rejected two accelerated approval applications from Regeneron Pharmaceuticals due to the absence of ongoing confirmatory studies.
- This decision signals the FDA's intent to utilize its enhanced authority, granted in 2022, to ensure timely validation of drug efficacy.
- The FDA's stance reflects a commitment to balancing expedited access to medicines with the need for robust evidence of clinical benefit.
- The rejection may spark debate about potentially delaying access to treatments for serious and life-threatening conditions.
Milestone Pharmaceuticals Resubmits NDA for Etripamil Nasal Spray for PSVT Treatment
- Milestone Pharmaceuticals has resubmitted its New Drug Application (NDA) to the FDA for etripamil nasal spray, a potential treatment for paroxysmal supraventricular tachycardia (PSVT).
- The resubmission addresses issues raised in a previous Refusal to File letter from the FDA, incorporating restructured data sets and reformatted data files.
- Etripamil is designed as a self-administered rapid response therapy, offering patients a new option for on-demand management of PSVT episodes.
- If approved, etripamil nasal spray could provide a valuable treatment option for the estimated two million people in the U.S. diagnosed with PSVT.
Aldeyra Therapeutics Plans Resubmission of Reproxalap NDA for Dry Eye Disease
- Aldeyra Therapeutics plans to resubmit its New Drug Application (NDA) for reproxalap to the FDA for treating dry eye disease.
- A dry eye chamber clinical trial is set to begin in the first half of 2024 to support the NDA resubmission.
- The planned trial will assess ocular discomfort in approximately 100 patients, with results expected to power the NDA resubmission in the latter half of 2024.
- Reproxalap, a RASP modulator, has shown statistically significant improvements in dry eye symptoms in previous trials.
China NMPA Accepts sBLA for Enfortumab Vedotin Plus Pembrolizumab in Advanced Bladder Cancer
- China's NMPA has accepted the sBLA for enfortumab vedotin with pembrolizumab as first-line treatment for locally advanced or metastatic urothelial cancer.
- The application is based on the EV-302 trial, which showed significant improvements in overall and progression-free survival compared to chemotherapy.
- If approved, this combination would be the first alternative to platinum-based chemotherapy for this patient population in China.
- The EV-302 trial demonstrated a clinically meaningful improvement in response rate and significantly improved overall survival compared to chemotherapy.
Highlighted Clinical Trials:
Astellas Pharma China, Inc.
Posted 7/22/2021
Merck Sharp & Dohme LLC
Posted 4/21/2021
Astellas Pharma Global Development, Inc.
Posted 3/9/2020
Astellas Pharma Global Development, Inc.
Posted 10/11/2017
Merck Sharp & Dohme LLC
Posted 7/24/2019
Astellas Pharma Global Development, Inc.
Posted 3/30/2020
Vicore's Buloxibutid Shows Promising Lung Function Improvement in Phase 2a IPF Trial
- Vicore Pharma's buloxibutid demonstrated significant lung function improvement in IPF patients, with an average FVC increase of 216ml over 36 weeks compared to the typical 180ml decline in untreated patients.
- The angiotensin II type 2 receptor agonist was well-tolerated with no new drug-related adverse events reported during the 26-week treatment period, suggesting a favorable safety profile.
- Final data from the Phase 2a AIR trial will be presented as a late-breaking presentation at the 2024 American Thoracic Society International Conference, with Vicore planning to advance to a Phase 2b ASPIRE trial.
FDA Extends Review of Govorestat for Classic Galactosemia Treatment
- The FDA extended the review period for Applied Therapeutics' govorestat NDA by three months, setting a new PDUFA target action date for November 28, 2024.
- The extension allows the FDA to review supplemental analyses of previously submitted data, which the FDA considers a Major Amendment to the NDA.
- Govorestat, a novel Aldose Reductase Inhibitor, aims to be the first approved medication for Galactosemia, addressing neurological complications and toxic metabolite build-up.
- Applied Therapeutics will maintain its expanded access program for govorestat, ensuring continued treatment access for Galactosemia patients during the review period.
Sequana Medical Announces 2023 Results and Advances alfapump and DSR Programs
- Sequana Medical's alfapump PMA application is under substantive review by the FDA, with extensive feedback being assessed for US market approval.
- DSR therapy shows promise for cardiorenal syndrome, demonstrating significant diuretic response and reduced diuretic requirements in heart failure patients.
- The company is preparing for the MOJAVE study's randomized phase, focusing on diuretic-resistant heart failure patients, with interim data expected in H2 2025.
- Financial results for 2023 reflect strategic focus on US market approval for alfapump and DSR program advancement, with reduced cash burn and extended runway.
UK First to Release Detailed Data on Clinical Trial Registration Failures
- The UK's Health Research Authority (HRA) has published detailed registration data for 1,545 clinical trials, a pioneering move for transparency.
- The data includes trial names, sponsors, and registration numbers, offering unprecedented insight into registration compliance.
- In 2022, 92% of trials were registered, showing improvement, with most sponsors informing the HRA of registration.
- Two-thirds of the registered trials were found in the U.S. database, ClinicalTrials.gov, highlighting its importance.