Millendo signs license agreement, secures funding to test new drugs for treatment of endocrine disorders

What started as research into one of the most rare types of cancer has expanded into a portfolio that includes a potential treatment for the most common endocrine disease in women.

Millendo Therapeutics, a University of Michigan startup company, announced an exclusive license agreement with AstraZeneca for the worldwide development and commercialization rights to test a new compound, MLE4901, for the treatment of polycystic ovary syndrome. There are no approved therapies for PCOS, which affects up to 15 percent of women.

Millendo also announced it has secured a $62 million Series B investment to perform clinical trials for MLE4901 and expand its testing of the drug ATR-101. The University of Michigan invested funds under its MINTS (Michigan Investment in New Technology Startups) program.

Previously known as Atterocor, Inc., Millendo is a biopharmaceutical company focused on developing novel, disease-modifying treatments for specialty and orphan endocrine diseases caused by hormone dysregulation. The Ann Arbor-based company was co-founded by Gary D. Hammer, M.D., Ph.D., Millie Schembechler Professor of Adrenal Cancer and director of the endocrine oncology program at the U-M Comprehensive Cancer Center.

The company initially focused on developing new therapies for adrenal cancer, one of the rarest and most deadly types of cancer, with about 500-1,000 people per year diagnosed in the United States. Standard therapy, which causes severe side effects, has been the same for more than 40 years. Nearly all who are diagnosed with the disease will die within five years.

In 2013, Millendo launched a phase I trial of ATR-101 in adrenal cancer patients. Laboratory studies by Hammer and Christopher La Pensee, Ph.D., at the University of Michigan found ATR-101 selectively kills adrenal cancer cells through a novel cholesterol-dependent mechanism. The drug has very little effect on other cells in the body. The ATR-101 trial is now available at five institutions, including the University of Michigan Comprehensive Cancer Center.

As part of the expanded funding, Millendo will begin testing ATR-101 in other related adrenal diseases, including Cushing's syndrome and congenital adrenal hyperplasia.

"As reflected in a growing pharma interest in orphan or neglected diseases, the new emerging data that define and refine the genomic landscape of endocrine cancers and related disorders has fueled a resurgence in research and targeted therapeutics for endocrine diseases, including adrenal cancer. This is a particularly exciting time for research and targeted therapeutics for endocrine diseases," Hammer says.

Hammer holds the Millie Schembechler Professorship of Adrenal Cancer in honor of the wife of legendary U-M football coach Bo Schembechler. The endocrine oncology program at the U-M Comprehensive Cancer Center was developed to create a level of expertise – both research and patient care – in this extremely rare disease that took the life of Millie Schembechler.