Latest news with #Rett syndrome
Medscape
11-08-2025
- Business
- Medscape
FDA Okays Ketamine-Based Med for Post-Surgical Pain
The FDA has approved the racemic ketamine product known as KETARx (PharmaTher Holdings) for the treatment of post-surgical pain. 'This historic FDA approval…is a testament to years of dedicated development,' Fabio Chianelli, chairman and CEO of PharmaTher, said in a release. 'We remain steadfast in our mission to harness the pharmaceutical potential of ketamine for a range of mental health, neurological, and pain disorders,' he added. The company noted that it hopes to expand ketamine development into conditions such as Parkinson's disease, amyotrophic lateral sclerosis, depression, and complex regional pain syndrome. Its product has already received an orphan drug designation for the treatment of Rett syndrome. It added that, among psychedelic and 'psychedelic-adjacent' drugs, ketamine is the only one to be included on a list of Essential Medicines by the World Health Organization. The company initially announced that the FDA had set an approval goal date of April 2024 for KETARx. However, after 'minor deficiencies identified by Quality' were identified, the FDA assigned a new target date of October 2024. As reported by Medscape Medical News at the time, the FDA issued a complete response letter requesting additional information on the application. The final date of August 2025 was set after the company submitted the requested additional information.
Yahoo
16-05-2025
- Business
- Yahoo
Taysha Gene Therapies Inc (TSHA) Q1 2025 Earnings Call Highlights: Promising Clinical Progress ...
Research and Development Expenses: $15.6 million for Q1 2025, down from $20.7 million in Q1 2024. General and Administrative Expenses: $8.2 million for Q1 2025, up from $7.1 million in Q1 2024. Net Loss: $21.5 million or $0.08 per share for Q1 2025, compared to $24.1 million or $0.10 per share in Q1 2024. Cash and Cash Equivalents: $116.6 million as of March 31, 2025. Cash Runway: Expected to support operations into Q4 2026. Warning! GuruFocus has detected 3 Warning Signs with TSHA. Release Date: May 15, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Taysha Gene Therapies Inc (NASDAQ:TSHA) has made significant regulatory and clinical progress with their TSHA-102 program for Rett syndrome, achieving alignment with the FDA on key elements of their pivotal trial design. The TSHA-102 gene therapy has shown promising results in clinical trials, with patients demonstrating functional gains in fine motor, gross motor, and communication skills, which are not expected in untreated populations. The company has completed dosing in the dose escalation portion of their REVEAL Phase 1/2 trials, with no treatment-related serious adverse events or dose-limiting toxicities reported. Taysha Gene Therapies Inc (NASDAQ:TSHA) has a strong cash position, with $116.6 million in cash and cash equivalents, expected to support operations into the fourth quarter of 2026. The company plans to provide a comprehensive update on their pivotal Part B trial design and clinical data at the upcoming International Rett Syndrome Foundation's 2025 Scientific Meeting. Research and development expenses decreased by $5.1 million compared to the previous year, which may indicate reduced investment in new initiatives or cost-cutting measures. General and administrative expenses increased by $1.1 million, primarily due to higher compensation expenses and legal and professional fees, which could impact profitability. The net loss for the quarter was $21.5 million, although slightly improved from the previous year, it still indicates ongoing financial challenges. The company faces significant risks and uncertainties, as highlighted in their SEC filings, which could impact future results and operations. The timeline for initiating the pivotal trial and achieving regulatory approval remains uncertain, with potential delays in site activation and patient enrollment. Q: What signals can potentially point to a dose response in the TSHA-102 trials for Rett syndrome, given the syndrome's heterogeneity? A: Sean Nolan, CEO, explained that they are looking at temporal aspects and improvements in function using metrics like RMBA and CGI. They expect differentiation at the high dose in terms of the time period and magnitude of response. Sukumar Nagendran, President and Head of R&D, added that translational data from animal to human studies show improvements in motor and respiratory functions, suggesting that higher doses could have a better clinical impact. Q: Could you frame the bar for success in light of recent regulatory feedback and the data expected this quarter? A: Sean Nolan, CEO, stated that the natural history analysis will provide a baseline for the disease's trajectory. They have reached alignment with the FDA on trial design, primary endpoint, study population, and patient numbers needed for statistical significance. The upcoming data will give insights into the probability of success and timeline for a potential BLA submission. Q: Is there any specific feedback that the FDA may provide post the 30-day period on the study protocol and SAP? A: Sean Nolan, CEO, mentioned that the FDA has already provided written agreement on key aspects of the trial design, primary endpoint, and study population. Sukumar Nagendran, President and Head of R&D, added that it is highly unlikely the FDA will have significant further comments, though minor correspondence could occur. Q: Can you discuss the primary endpoint for the trial and if it will include standardized clinical videos showing milestone or functional gain? A: Sean Nolan, CEO, indicated that the primary endpoint will be objective, clinically meaningful, and easy to measure. They aim for a robust clinical study without a placebo, using mechanisms like video assessments and blinded adjudicators to ensure bias mitigation. Q: How does the aligned design compare with your proposal or desired design going into the meeting with the FDA? A: Sean Nolan, CEO, confirmed that the design proposed by the company was endorsed by the FDA. The agency has been constructive and open-minded, requesting data to support the trial design and endpoints, which the company provided. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
