Milestone Pharma Has Strong Product Candidate, Charlotte Connections

Milestone Pharmaceuticals Inc., a Montreal company with a growing Charlotte subsidiary, has something in development that investors like.

It’s a nasal spray called Etripamil. And it’s in Phase 3 clinical trials as a simpler, less-expensive and needed treatment for a heart ailment that affects two million Americans, resulting in approximately $2 billion in annual healthcare costs.

In May, Milestone (Nasdaq: MIST) closed its initial public offering, raising $95 million. That followed four rounds of venture capital investment that totaled $138.8 million from a number of top venture firms, the first led by Triangle-based Pappas Ventures.

The company, founded in 2003, is developing a treatment for paroxysmal supraventricular tachycardia (PSVT), which causes anxiety, sweating, and rapid heart rate. It frequently results in expensive visits to an emergency room where it is treated with an IV-pushed drug in an unpleasant procedure. It primarily affects women.

"We believe that PSVT represents a large and under-recognized market that we estimate affects approximately two million Americans and results in over 600,000 healthcare claims in the United States per year, including emergency department visits, hospital admissions and ablations," said Joseph Oliveto, president and CEO of Milestone, in an interview with the North Carolina Biotechnology Center.

"The current treatment paradigm for PSVT episodes is restricted to the acute-care setting and is both burdensome and costly. Etripamil has the potential to become the first self-administered therapy (via a nasal spray) approved for the rapid termination of episodes of PSVT wherever and whenever they occur."

The company’s founder and chief scientific officer, Montreal-based Philippe Douville, assembled a drug-hunter’s club of highly experienced, retired pharmaceutical developers and executives. A retired Glaxo executive came up with the winning idea, using a calcium channel blocker to treat PSVT. The company took the idea forward, resulting in the Etripamil treatment.

Oliveto was formerly president, CEO and a director of Charlotte-based Chelsea Therapeutics and led the company through a successful turnaround that included FDA approval of Northera, a drug to treat a sudden drop in blood pressure that occurs while standing. The disorder is technically known as symptomatic neurogenic orthostatic hypotension (NOH).

Oliveto was at the helm in 2014 when Chelsea was purchased by Danish global drug company Lundbeck for a reported total of $658 million.

Oliveto had joined Chelsea in 2008 following a two-year assignment as an executive advisor at Pappas Ventures. He joined Milestone as CEO in March 2017 and brought some of his former Chelsea colleagues with him.

Milestone has 39 employees, nine in North Carolina, with eight openings at this writing. Oliveto said the company also has numerous partnerships with Triangle-based companies.

“While growing from seven to 39 over the last couple years, Milestone is still a burgeoning company and collaborates extensively with, and leans heavily on, our partners across the value chain of scientific advice, development, and commercialization,” Oliveto said.

Triangle Connections

He adds, “This approach is seen prominently in our abundance of relationships and collaborations with RTP-based institutions including collaborations with key opinion leaders at Duke who counsel on our clinical trials, Triangle Insights Group, which helps us to understand the market, finish manufacturers who produce our final product, analytical laboratories that test and release our final product, and CROs that are integral to conducting our global Phase 3 program.”

Oliveto said the company hopes to keep the price of Etripamil as low as possible, possibly $25 to $50 a dose out of pocket, even though patients will probably only need to use it about three times a year. That will be especially attractive compared to an emergency room visit for treatment can run into thousands of dollars, depending on a patient’s insurance.

The company will continue running Phase 3 trials through 2021, aiming for FDA approval in 2022 or 2023. The Milestone website includes a PowerPoint explanation of its science by Oliveto.

Oliveto said the company plans to stay lean, keeping its headcount under 50 this year, but is actively recruiting for multiple positions across the late-stage drug development, medical affairs and commercialization disciplines.