After struggling with its sole drug for years, Marinus rides positive data to a commercialization deal in Europe
After a series of painful setbacks, Marinus had a rebirth of sorts last year when it offered a positive late-stage preview of its sole drug, ganaxolone, in a genetic disorder that causes early-onset epilepsy. Now, CEO Scott Braunstein is riding that success to an NDA submission and a collaboration with the Finnish pharma company Orion to commercialize the candidate in Europe.
Orion is putting down around $30 million upfront (€25 million) and $115 million in biobucks (€97 million) for exclusive rights to market both the oral and IV formulations of ganaxolone in the EU, UK and Switzerland for the treatment of CDKL5 deficiency disorder (CDD), tuberous sclerosis complex (TSC) and refractory status epilepticus (RSE).
Braunstein just submitted an NDA for the oral version of the candidate, a GABA/A receptor modulator, in CDD — and he plans on filing with European regulators in Q3 of this year. If all goes well, oral ganaxolone could hit the European market in mid-2022.
“We were close to going under a year ago,” he told Endpoints News last year. “We didn’t have a year’s worth of cash.”
Then came data from the Phase III Marigold trial, which showed CDD patients treated with ganaxolone saw a 30.7% median reduction in 28-day major motor seizure frequency, compared to a 6.9% reduction for those on placebo (p=0.0036). In an open-label extension study, the ganaxolone patients saw a 49.6% reduction. The drug was well-tolerated, with the most frequent side effect being drowsiness, according to the company.
Upon reading out the topline data, BARDA offered Marinus $21 million and a shot at $30 million more to support the biotech’s work on an IV version for refractory status epilepticus. While the oral dosing is for chronic illness, the IV formulation is designed to deliver high dosing over a short interval for acute, severe medical conditions, Braunstein said.
“It’s pretty incredible to think we’ve been remote, we’ve raised over $200 million in capital, we’ve now filed our first NDA, (and) we’re going to announce this European partnership,” he said. And they did it all while keeping up with remote yoga twice a week.
But it hasn’t been all smooth sailing. While the company was scheduled to deliver Phase III data on RSE in the first half of 2022, that readout has been pushed back to the second half of 2022. Braunstein blamed the delay on the pandemic, which he says caused staff turnover and held things up at trial sites.
“I’m just really proud of the team, how many achievements they’ve continued to hit in an incredibly difficult time,” Braunstein said. “We’ve had a little bit of a stumble here in Q2, but (I’m) quite confident we’re going to bounce back.”