Millendo Announces Successful Recruitment of Phase 2b Study of Livoletide

Millendo announced today they have successfully enrolled 150 patients into their Phase 2b/3 clinical study of livoletide, completing enrollment for patients ages 8 to 85. The trial will continue to enroll patients ages 4 to 7 years old, and a minimum of 44 lbs. With the study now fully enrolled, topline results are expected in the first half of 2020 which may support a New Drug Application (NDA) filing for livoletide.

The second part of ZEPHYR is a Phase 3 study that will recruit additional patients at the same clinical sites and consists of a six-month double-blind, placebo-controlled core period in which patients will receive livoletide or placebo followed by a six-month extension period in which all patients receive livoletide. The primary endpoint for both the Phase 2b and Phase 3 portions of ZEPHYR is the change in food-related behaviors using the validated Hyperphagia Questionnaire for Clinical Trials (HQ-CT).

 

Read below for Millendo's recent press release:

Millendo Therapeutics, Inc. (Nasdaq: MLND), a clinical-stage biopharmaceutical company developing novel treatments primarily for orphan endocrine diseases, today announced that the Phase 2b portion of its pivotal Phase 2b/3 clinical study of livoletide in patients with Prader-Willi syndrome (PWS), called ZEPHYR, has completed recruitment for patients ages 8 to 65. The study, one of the largest PWS studies ever conducted with over 150 patients recruited across 38 clinical trial sites worldwide, is evaluating the safety and efficacy of livoletide on food-related behaviors in patients with PWS. The primary endpoint of the study is an assessment of livoletide’s impact on hyperphagia, the constant feeling of excessive hunger that is a hallmark symptom of the disease. Topline results are expected in the first half of 2020 and may support a New Drug Application (NDA) filing for livoletide.

“Completing recruitment of over 150 patients in this pivotal study is an important step forward as we continue our efforts to advance livoletide for PWS patients and families who struggle with the life-threatening symptoms of the disease, especially hyperphagia,” said Julia C. Owens, President and Chief Executive Officer of Millendo Therapeutics. “This achievement is also an important milestone for Millendo. Ahead of sharing topline results from the Phase 2b portion of ZEPHYR in the first half of next year, we are preparing for a build of our commercial organization in the Boston area.”

The ZEPHYR study, initiated in March 2019, is a two-part, randomized, double-blind, placebo-controlled pivotal Phase 2b/3 study. The Phase 2b portion includes a three-month double-blind, placebo-controlled core period in which patients receive one of two doses of livoletide or placebo followed by a nine-month extension period in which all patients receive livoletide. All patients who have completed the core period of the Phase 2b portion of the study thus far have entered into the nine-month safety extension period. Millendo continues to implement a protocol amendment globally adding an additional cohort of PWS patients ages four to seven. Sites that recruit pediatric patients will continue to actively recruit these patients.

The second part of ZEPHYR is a Phase 3 study that will recruit additional patients at the same clinical sites and consists of a six-month double-blind, placebo-controlled core period in which patients will receive livoletide or placebo followed by a six-month extension period in which all patients receive livoletide. The primary endpoint for both the Phase 2b and Phase 3 portions of ZEPHYR is the change in food-related behaviors using the validated Hyperphagia Questionnaire for Clinical Trials (HQ-CT).

“We are very pleased with how quickly our participating trial sites were able to recruit for ZEPHYR. Throughout this process, we have seen enthusiasm from our active sites and we appreciate their partnership as we work toward our common goal of addressing hyperphagia in PWS,” said Ryan Zeidan, Chief Development Officer of Millendo Therapeutics. “We are grateful to the patients, families and caregivers and investigators involved in the study for their commitment and dedication.”

About Livoletide
Livoletide is an unacylated ghrelin analogue in late-stage clinical development for the treatment of Prader-Willi syndrome (PWS), a rare genetic disease characterized by hyperphagia, a chronic unrelenting hunger, that leads to obesity, metabolic dysfunction, reduced quality of life and early mortality. In a previous randomized, double-blind, placebo-controlled Phase 2 clinical trial in 47 patients with PWS, administration of livoletide once daily for two weeks was associated with a clinically meaningful improvement in hyperphagia, as well as a reduction in appetite. Millendo has received both Orphan Drug Designation and Fast Track Designation for livoletide for the treatment of PWS from the U.S. Food and Drug Administration (FDA) and Orphan Drug Designation from the European Medicines Agency (EMA). For more information about Millendo’s pivotal study of livoletide (ZEPHYR) please visit www.clinicaltrials.gov (NCT03790865) or the Patients and Families portion of our website.

About the ZEPHYR study
The ZEPHYR study is a two-part, randomized, double-blind, placebo-controlled pivotal Phase 2b/3 study. The first part is a Phase 2b study that includes a three-month double-blind, placebo-controlled core period in which patients receive one of two doses of livoletide or placebo followed by a nine-month extension period in which all patients receive livoletide. The Phase 2b portion of the study, one of the largest global PWS studies ever conducted, has completed recruitment for patients ages 8 to 65 with over 150 patients across 38 clinical sites in the United States, Europe and Australia. Millendo continues to implement a protocol amendment globally adding an additional cohort of PWS patients ages four to seven. Sites that recruit pediatric patients will continue to actively recruit patients for this cohort. The second part is a Phase 3 study that will consist of a six-month double-blind, placebo-controlled core period in which patients will receive livoletide or placebo followed by a six-month extension period in which all patients receive livoletide. The study’s primary endpoint measures the change in food-related behaviors using the validated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) during each core period. ZEPHYR is a pivotal study and the results of the Phase 2b portion of the study may be sufficient to support a new drug application (NDA) for livoletide.

About Millendo Therapeutics, Inc.
Millendo Therapeutics is a late-stage biopharmaceutical company primarily focused on developing novel treatments for orphan endocrine diseases where current therapies do not exist or are insufficient. As a leading orphan endocrine company, Millendo creates distinct and transformative treatments where there is a significant unmet medical need. The company is currently advancing livoletide for the treatment of Prader-Willi syndrome, nevanimibe for the treatment of classic congenital adrenal hyperplasia and MLE-301 for the treatment of vasomotor symptoms associated with menopause. For more information, please visit www.millendo.com.

 

Topics: News

Susan Hedstrom

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Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.

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