NOVATO, CA – Ultragenyx Pharmaceutical (NASDAQ:RARE) Inc. announced today the successful conclusion of a critical meeting with the U.S. Food and Drug Administration (FDA), marking a significant step forward in the development of GTX-102, a potential treatment for Angelman syndrome. The end-of-Phase 2 meeting with the FDA resulted in an agreement on the pivotal Phase 3 study design and primary endpoints.
The Phase 3 trial, a global, randomized, double-blind, sham-controlled study, will enroll approximately 120 patients with a genetically confirmed diagnosis of full maternal UBE3A gene deletion, a form of Angelman syndrome. The study's primary measure of success will be the improvement in cognitive abilities, specifically assessed by the Bayley-4 cognitive raw score over a 48-week period. Patients who complete the study in the control group will have the opportunity to receive the treatment after the initial double-blind phase.
Interim data from the earlier Phase 1/2 study highlighted rapid and clinically significant cognitive improvements in patients treated with GTX-102, surpassing minimal changes observed in natural history data. The Phase 3 study will also evaluate the Multi-domain Responder Index (MDRI) as a key secondary endpoint, which covers cognition, communication, behavior, motor function, and sleep.
In addition to the FDA's support, the European Medicines Agency has accepted the overall Phase 3 study approach, and Ultragenyx is expected to engage with Japan's Pharmaceuticals and Medical Devices Agency to discuss the study design.
Furthermore, Ultragenyx plans to initiate an open-label study to assess GTX-102's safety and efficacy in different Angelman syndrome genotypes and age groups. This aims to expand the potential treatment to a wider patient population.
The company cautions that forward-looking statements involve significant risks and uncertainties, and actual results could vary. Ultragenyx emphasizes that its clinical development programs, future results, performance, or achievements may differ from current expectations, and there is no guarantee of clinical benefit, tolerability, or safety of GTX-102.
InvestingPro Insights
As Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) advances with its Phase 3 study for GTX-102, investors and stakeholders are closely monitoring the company's financial metrics and market performance. According to real-time data from InvestingPro, Ultragenyx has a market capitalization of approximately $3.98 billion, reflecting the market's current valuation of the company's potential. Despite not being profitable over the last twelve months, the company's revenue has grown by 15.29% during this period, indicating a positive trajectory in its financial performance.
One of the InvestingPro Tips highlights the company's strong return over the last month, with a 13.74% price total return, which could suggest investor confidence in the company's recent developments and future prospects. Additionally, the company's liquid assets surpass short-term obligations, providing financial stability as it continues its clinical trials. While the company operates with a moderate level of debt and does not pay a dividend, these aspects may be typical for a biopharmaceutical firm in the growth stage of its lifecycle, focusing on research and development.
For investors seeking deeper insights, there are additional InvestingPro Tips available, which can be accessed through a subscription. By using the coupon code PRONEWS24, readers can get up to 10% off a yearly Pro and a yearly or biyearly Pro+ subscription. This access could provide valuable information for making informed decisions about investing in Ultragenyx as it continues to navigate the complexities of drug development and regulatory approval.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.