jfm1330 wrote: Strange. The press release on Thera's website about ENDO says nothing about the phase III plans in NASH, but a different version I saw this morning reiterates their plan to go forward with the phase III. Look at the second section. I did not see this version on their website.
Theratechnologies plans phase 3 tesamorelin clinical
2021-03-22 03:18 ET - News Release
Dr. Christian Marsolais reports
THERATECHNOLOGIES ANNOUNCES NEW DATA DEMONSTRATING TESAMORELIN'S POSITIVE EFFECT ON IMMUNE RESPONSE LINKED TO LIVER INFLAMMATION
New data, demonstrating the positive effect of tesamorelin on the circulation of immune activation markers associated with liver inflammation, was presented on March 20, 2021, at The Endocrine Society's annual meeting, ENDO 2021.
Dr. Takara L. Stanley, Assistant Professor of Pediatrics, Harvard Medical School, Assistant Pediatrician, Massachusetts General Hospital and Program Director, Pediatric Endocrine Fellowship Program, MGHfC, authored the poster presented at ENDO 2021.
The data comes from a sub-analysis of a double-blind, randomized, 12-month investigator-initiated trial studying the effect of tesamorelin on liver fat in 61 people infected with HIV with nonalcoholic fatty liver disease (NAFLD) which was conducted by Dr. Steven Grinspoon, Professor of Medicine, Harvard Medical School, Chief of the Metabolism Unit at Massachusetts General Hospital. Dr. Grinspoon's findings were published in The Lancet HIV in October 2019.
The sub-analysis concludes that treatment with tesamorelin for 12 months decreased circulating markers of T-cell and monocyte/macrophage activity. A corresponding downregulation of immune pathways in the liver was also observed. These conclusions suggest that treatment with tesamorelin may contribute to better regulated immune activation in a population with metabolic dysregulation and systemic inflammation.
"The data presented at ENDO 2021 highlights tesamorelin's unique mechanism of action that addresses the underlying cause of liver disease and further supports the Phase 3 development of this novel medicine for the potential treatment of NASH," said Dr. Christian Marsolais, Senior Vice President and Chief Medical Officer at Theratechnologies.
Phase 3 clinical trial
The proposed Phase 3 clinical trial design will enroll participants with liver-biopsy confirmed NASH and stage 2 or 3 fibrosis. Participants will be randomized 1:1 to receive 2 mg of tesamorelin or placebo. A second liver biopsy will be performed after 18 months of treatment for the first 900 participants, approximately. These data will form the basis for filing an sBLA with the U.S. Food and Drug Administration (FDA) to seek accelerated approval. The primary endpoint used to seek accelerated approval will be the percentage of participants achieving NASH resolution and no worsening of fibrosis compared to placebo. Participants will remain in the Phase 3 trial for a total of 60 months. Subject to additional discussions with regulatory agencies, approximately 2,000 participants in total are expected to be enrolled, including a cohort of approximately 75 to 100 participants with HIV.
In late December 2020, the Company received a "Study May Proceed" letter for the Phase 3 trial from the FDA with a recommendation that the Company requests a meeting to discuss questions and comments provided on certain aspects of the proposed trial design. The Company has formally requested a meeting with the FDA to ensure alignment with current regulatory expectations for the late-stage development of treatments for NASH. The Company is assessing its strategy regarding a filing with the EMA to initiate a Phase 3 clinical trial of tesamorelin for the treatment of NASH in the European Union.
The Company plans to initiate the Phase 3 clinical trial by the end of the third quarter of calendar year 2021. The timing of the trial initiation and the final number of patients enrolled are dependent upon any adjustments to the protocol and trial design as recommended by the FDA and EMA. The Company has retained the services of a global, large-scale contract research organization, or CRO, with experience in implementing large and late-stage clinical trials to assist with the execution of its Phase 3 clinical trial in NASH.
About NAFLD /NASH
According to the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health, experts estimate that 20 percent of Americans with NAFLD have NASH. It is believed that 3 to 12 percent of adult Americans have NASH.1
NAFLD is an umbrella term for a spectrum of liver conditions that begin with a build-up of hepatic fat, which can set the stage for inflammation that may promote scarring known as fibrosis. Over time, fibrosis can progress to potentially fatal cirrhosis and even a form of liver cancer called hepatocellular carcinoma.
Usually, NAFLD and NASH are silent diseases with few or no symptoms. A patient may not show symptoms even if they develop cirrhosis due to NASH.
There is currently no approved treatment for NAFLD and NASH in the North America and Europe.
About Theratechnologies Inc.
Theratechnologies is a biopharmaceutical company focused on the development and commercialization of innovative therapies addressing unmet medical needs.
We seek Safe Harbor.