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AstraZeneca Spins Out Women's Health Treatment To $62M Startup, Millendo

This article is more than 8 years old.

Drug giant AstraZeneca has been regularly reshuffling R&D priorities for a couple years now. When that happens, little biotech companies look for promising assets they can pick up and run with.

Millendo Therapeutics just got $62 million, in large part, to do that very thing.

Ann Arbor, Mich.-based Millendo is announcing today it will put much of the new cash to work on developing a drug AstraZeneca developed for polycystic ovary syndrome (PCOS).

This disease isn’t a top-of-mind women’s health issue like breast cancer, but it affects millions of women in their childbearing years. Between 4% and 8% of the worldwide female population, or maybe as many as 15-20%, are affected, depending on which diagnostic criteria doctors choose to rely on.

However many there are, what these women have is an endocrine disorder that results in enlarged, overactive ovaries that contain pockets of fluid, known as follicles. That leads to a hormonal imbalance that ends up causing excessive buildup of male sex hormones (androgens) in the blood. All kinds of unpleasant symptoms come next--acne, excessive hair growth, obesity, baldness and infrequent or prolonged menstrual periods.

There is no FDA-approved product for the disease. Only within the last couple years have scientists shed new light on the underlying genetic factors that drive it, giving pharmaceutical companies new ideas for treatment. For years, physicians have gotten by treating various symptoms with birth control pills, generic metformin (a diabetes drug) or just advising women to shave.

“Men don’t always intuitively appreciate what PCOS is, but it does run in families,” said Julia Owens, CEO of Millendo. “Some (investors) have watched it in their families, and they say ‘Oh, you’re working on PCOS?' And they get it immediately.”

Awareness of the disease wasn’t a big hurdle for Millendo, at least in part because it is run by a female CEO, and it has a majority-female board of directors (three of five). Besides Owens, the other female directors are Carol Gallagher of New Enterprise Associates, and Tracy Saxton of Roche Venture Fund. The $62 million financing of Millendo was led by NEA, and included Roche Venture Fund, as well as Adams Street Partners, Altitude Life Science Ventures, Longwood Fund, Renaissance Venture Capital Fund, Frazier Healthcare Partners, Osage University Partners, 5AM Ventures and the Regents of the University of Michigan’s MINTS Program.

The company, formerly known as Atterocor, was founded in 2012 with a $16 million venture capital investment. Owens said she got tired with the old name being regularly misspelled and mispronounced. She decided to change it to Millendo partly because it was easy to spell and pronounce, but also to highlight the company's concentration on endocrine disorders like PCOS, adrenal cancer and Cushing syndrome.

As part of the license deal, Millendo has agreed to make an undisclosed upfront cash payment to AstraZeneca for the compound known as AZD4901. AstraZeneca will also get an equity stake in Millendo, and the big company will be eligible for milestone payments and royalties if Millendo's efforts are successful.

The idea for handing off the drug to Millendo can be traced back to some conversations between AstraZeneca executives and some venture capitalists. Frazier Healthcare, an early investor in Millendo, had a series of conversations with AstraZeneca about potentially forming a new company to develop the asset that was no longer in the parent company’s wheelhouse. Instead, they decided to license it to an existing company, Millendo, which only had one other drug candidate, but had performed well in early development with it, Owens said.

Much of the early heavy lifting with the drug candidate has been done. AstraZeneca ran a Phase 2a clinical trial with 65 women who got three different doses of the compound, or a placebo. The drug, taken orally once a day, hit its goals of reducing leutinizing hormones in the blood by 52%, and reducing testosterone in the blood by 29%, after seven days of treatment, according to data presented at the Endocrine Society meeting last year in San Diego. The testosterone reductions were sustained for the full 28 days of the study. No serious side effects were reported, researchers said.

Millendo plans to build off that result with a larger Phase 2b clinical trial, Owens said.

The drug is designed to work by inhibiting the Neurokinin B receptor. One other drug developer, Belgium-based Euroscreen, is actively working on a PCOS drug based on the new understanding of the underlying biology, while Switzerland-based EffRx Pharmaceuticals also has another option in the works, a reformulated version of metformin, a common diabetes drug.

Whenever you have a drug vying to be first-in-class against a new molecular mode of action, there’s considerable risk. The FDA hasn’t considered an application from a drugmaker before, so it’s unclear what types of evidence will be needed to win a market approval.

Public awareness, or confusion about what PCOS is, represents another potential challenge for drugmakers. There are wide differences in diagnostic criteria used by the National Institutes of Health, and what’s known as the Rotterdam criteria, Owens said. Only about 40% of the patients with PCOS are currently diagnosed. At least one patient advocacy group is trying to raise awareness of the condition so more women seek treatment, and some doctors are trying to rename the disorder based on the new understanding of the endocrine system problem, Owens said.

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