After a series of painful setbacks, Marinus experienced something of a rebirth last year when he offered late stage positive insight into his only drug, ganaxolone, in a genetic condition that causes early epilepsy. Today, CEO Scott Braunstein associates that success with an NDA submission and working with Finnish pharmaceutical company Orion to market the candidate in Europe.
Orion is investing around $ 30 million upfront (€ 25 million) and $ 115 million in biobucks (€ 97 million) for the exclusive rights to market oral and intravenous formulations of ganaxolone in the EU, at United Kingdom and Switzerland for the treatment of CDKL5 deficiency disorder (CDD), tuberous sclerosis of Bourneville (TSC) and refractory status epilepticus (ESR).
Braunstein has just submitted an NDA for the oral version of the candidate, a GABA / A receptor modulator, on CDD – and he plans to file a case with European regulators in the third quarter of this year. If all goes well, oral ganaxolone could hit the European market in mid-2022.
Braunstein took the helm back in 2019, after ganaxolone stumbled in several studies on epilepsy, fragile X syndrome, and postpartum depression.
“We were about to sink a year ago,” he said. Terminal news Last year. “We didn’t have the equivalent of a year of cash.
Next came data from the Phase III Marigold trial, which showed CDD patients treated with ganaxolone saw a median 30.7% reduction in the frequency of major motor seizures over 28 days, compared to a reduction in 6.9% for those on placebo (p = 0.0036). In an open-label extension study, patients taking ganaxolone saw a reduction of 49.6%. The drug was well tolerated, with the most common side effect being drowsiness, according to the company.
After reading the baseline data, BARDA offered Marinus $ 21 million and an additional $ 30 million chance to support biotechnology work on an IV version for refractory status epilepticus. While the oral dosage is for chronic conditions, the IV formulation is designed to deliver a high dosage over a short interval for acute and severe medical conditions, Braunstein said.
“It’s pretty amazing to think we’ve been moved away, 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. declared. And they did it all by following distance yoga twice a week.
But it was not all smooth sailing. While the company was due to provide Phase III data on CSR in the first half of 2022, that reading was pushed back to the second half of 2022. Braunstein blamed the delay on the pandemic, which he said caused staff turnover. and delayed things. at test sites.
“I am really proud of the team, of the number of achievements they have continued to achieve in an incredibly difficult time,” said Braunstein. “We had a little stumble here in Q2, but (I’m) pretty confident we’ll bounce back.”