A month after its neurosteroid ganaxolone met its primary endpoint in a Phase IIa epilepsy trial, Marinus Pharmaceuticals Inc. pulled in $20 million in its second major round of venture capital funding to support further studies of the drug.
The Branford, Conn.-based firm has raised about $50 million to date, previously closing a $29 million Series A round in 2005. Despite facing a much more challenging financing environment this time around, the company was able to reach out successfully to all its existing investors, including Canaan Partners, Domain Associates, Sofinnova Ventures and Foundation Medical Partners, all of whom participated in the Series B.
"It's a strong vote of confidence" from those investors, said John Krayacich, president and CEO of Marinus, adding that the latest round validates the work the company has done on ganaxolone. "At the end of the day, we're all interested in seeing if this drug can make it to market and [offer] an alternative therapy in an area where there's really an unmet need."
Ganaxolone is neuroactive steroid derived from allopregnanolone, an endogenous neurosteroid whose activity has been implicated in a number of disorders, including epilepsy. The drug aims at modulating the gamma-aminobutyric acid Type A (GABA A) receptors, an approach that's been used "for some time" in treating epileptics, Krayacich said.
It specifically binds to the nonbenzodiazepine site of GABA A, between the alpha4 and delta subunits, and has similar activity to benzodiazepine without the tolerance issue. And because ganaxolone "mimics a naturally occurring neuromodulator, it doesn't have the same steroidal side effects" as some of the existing products, he told BioWorld Today.
Last month, Marinus reported that ganaxolone adjunctive therapy met its primary endpoint of significantly reducing seizures compared to placebo in adult epileptics in a 147-patient Phase IIa study. The company is set to report data from a separate Phase II study in infantile spasms at the American Academy of Neurology meeting in Seattle later this month.
Funds from the Series B are expected to continue an open-label extension trial of ganaxolone in epilepsy and to complete work on an oral solid formulation of the drug that the company anticipates using in upcoming Phase IIb and Phase III studies. Trials to date have tested ganaxolone as a liquid suspension, Krayacich said.
Marinus is targeting early 2010 for the next set of studies, including the anticipated Phase IIb of ganaxolone in epilepsy, but the company likely will have to look at pulling in some more money before that.
The Series B should "carry us through to about the third quarter of this year," Krayacich said. "As we move into Phase IIb, we'll need to raise additional funds."
Marinus also plans to conduct proof-of-concept work with ganaxolone in post-traumatic stress disorder.
Depending on funding, the company also could explore the drug in additional indications, such as post-partum depression and premenstrual dysphoric disorder.
A small organization with six full-time employees, the company operates largely as a virtual organization. Ganaxolone is its only product, and the focus is on getting that product through trials and starting discussions for potential partnership opportunities. But "we're always on the lookout for additional drugs," Krayacich said.
Marinus was founded in late 2003 with the aim of bringing in programs for central nervous system disorders, including neurological, psychiatric and pain disorders.
In other financings news:
• Molecular Templates Inc., of Austin, Texas, said it secured $2.5 million in financing from Santé Ventures. Proceeds will be used to continue product development and conduct clinical trials. Founded by researchers at the University of Toronto and the Ontario Cancer Institute, MTI recently relocated to Austin, in conjunction with the financing, and is focused on developing a protein platform for development of cancer therapeutics. Its lead program is a compound for melanoma, which is set to start clinical studies.