OvaScience, Millendo Therapeutics Merge to Advance Cushings’s and PWS Therapies

OvaScience and Millendo Therapeutics will merge in an all-stock deal to push forward Millendo’s lead candidates livoletide (AZP-531) and nevanimibe (ATR-101) for the treatment of Prader-Willi syndrome and Cushing’s syndrome, respectively.

An investor group that includes New Enterprise Associates, Frazier Healthcare Partners, Roche Venture Fund, InnoBio managed by Bpifrance, Osage University Partners, Altitude Life Science Ventures, Adams Street Partners, and Longwood Fund plans to invest $30 million in the new company until the merger is complete.

“We are excited about the opportunities created by this merger, as it positions us to become a leader in rare endocrine diseases with the funding needed to pursue the potential approval and commercialization of our first-in-class programs,” Julia Owens, PhD, president and CEO of Millendo, said in a press release. “Our objective has always been to deliver meaningful therapies to patients who do not have sufficient treatment options, and with this merger, the compelling clinical data for our programs, and the initiations of late-stage trials for both livoletide and nevanimibe expected in the months ahead, we are optimistic that we can deliver on this vision,” she said.

People with Prader-Willi syndrome experience a constant feeling of hunger, which often leads to obesity. Livoletide was developed to counteract some of the effects of the hunger hormone, called acylated ghrelin.

The medicine was studied in a randomized, placebo-controlled Phase 2 clinical trial with a total of 47 Prader-Willi syndrome patients, showing a decrease in patients’ appetite. Researchers plan to advance livoletide into a pivotal Phase 2b/3 study by March 2019.

Millendo also is focused on the development of nevanimibe as a therapy for classic congenital adrenal hyperplasia (CAH) — a group of hereditary disorders that affect the adrenal glands and limit their ability to produce certain vital hormones — and endogenous Cushing’s syndrome (ECS).

Nevanimibe inhibits ACAT1, an enzyme involved in the transformation of cholesterol into steroid hormones. In Cushing’s, there is an overproduction of a steroid hormone called cortisol and nevanimibe acts by lowering cortisol synthesis rate.

A Phase 2 trial (NCT02804178) on nevanimibe’s safety and effectiveness in CAH had positive results. Nevanimibe is expected to advance into a Phase 2b study later this year.

Regarding nevanimibe’s role on endogenous Cushing’s syndrome, a Phase 2 randomized, placebo-controlled trial (NCT03053271) is still recruiting ECS patients to determine the drug’s ability to reduce urinary cortisol levels. Researchers plan to enroll 16 adult subjects.

After a wash-out period, all eligible participants will be given nevanimibe in progressive increasing doses for six weeks. Subjects will then undergo a four-week randomized withdrawal period. The duration of the study will range from 14 to 20 weeks.

“Following an extensive and thorough review of strategic alternatives, we believe that this merger with Millendo is the best path forward and has the potential to deliver significant and immediate value to OvaScience shareholders,” said Christopher Kroeger, MD, OvaScience’s CEO. “We are certain that the strength and dedication of the Millendo leadership team provides our combined company with the capabilities to develop new and much-needed therapies for patients with orphan endocrine diseases.”

The new business will operate under the name Millendo Therapeutics and trade on the Nasdaq Capital Market under the ticker symbol MLND.