Despite protests, Milestone Pharma gets clobbered by a PhIII crash in the clinic
Milestone Pharma had all the earmarks of a classic biotech IPO story when it went public last year.
The little 12-year-old biotech had positive mid-stage data to tout to investors that underscored the potential of a nasal spray treatment to halt paroxysmal supraventricular tachycardia — or PSVT — events. A big syndicate put up $80 million to help them lay a commercial foundation. And the stock peaked soon after the company debuted on Nasdaq as they raised another $82 million.
But the Milestone team — and the stock $MIST — crashed at an ugly twist in the Phase III development path. Their drug, etripamil, a fast-acting calcium channel blocker, failed the primary. Shares were routed, free-falling 72% ahead of the bell.
The biotech, based in Montreal, was looking to offer an at-home solution to a rapid heartbeat that usually is unthreatening but sends some people back to the ER over and over again. At Phase II, researchers said they tracked success rates of 65% to 95%, beating out the 35% placebo score.
The primary endpoint was a comparison of the nasal therapy with placebo over 5 hours in turning SVT to sinus rhythm, and it fell far short of the mark on statistical significance with a value of 0.12. It also fell short on reducing trips for emergency care, with the same p value.

Milestone, though, isn’t going to give up at this point without a struggle. There was a positive hit on patient-reported treatment satisfaction, a positive “trend” on the ER measure and they drilled down to make the case on the drug’s early impact. And that’s what they’re taking to the FDA now for exploratory talks.
“(O)utcomes after 100 minutes, which were affected by a very small number of placebo patients remaining in the study at that time, suggest that the design and analysis plan used in NODE-301 negatively impacted the study’s outcome,” said CEO Joseph Oliveto in a statement.
There’s more data to come, but the company is sounding a note of caution on how the Covid-19 outbreak might affect trial operations at this stage.
Social image: Joseph Oliveto, Milestone Pharma