Keeping it simple
Based: Durham, NC
CEO: Robert Ryan
Clinical focus: Epidermolysis bullosa
The scoop: There aren't a lot of frills associated with Scioderm. The biotech has a virtual staff of 6. The CEO, Robert Ryan, also handles the PR work, when necessary. But all of their undivided attention is concentrated on a potential breakthrough therapy for kids with a very rare disease: epidermolysis bullosa, a condition that leaves children's skin papery thin and fragile, subject to tearing and blistering. Most die before the age of 30.
Ryan also took the lead in gaining the FDA's breakthrough therapy designation for Scioderm's topical treatment, SD-101, which seemingly overnight found itself with the agency's most coveted regulatory status, a closely watched mid-stage treatment that scored promising results in a tiny trial of patients--most of whom responded well to the therapy--and a $16 million venture round which just might get the company to the threshold of an approval.
What makes Scioderm fierce: With only modest resources, Scioderm found itself in league with the industry's giants, companies like Novartis ($NVS), J&J ($JNJ) and Merck ($MRK), which have all been eager to tout their BTDs as a sign of distinction for multibillion-dollar research efforts. And the biotech is hot at work in one of the most popular R&D fields in biotech, making a name for itself in a rare-disease space that has also attracted the high-profile attention of Shire ($SHPG). Shire spent $50 million upfront and promised up to $275 million in milestones to nab another EB company, Lotus Tissue Repair, launched by Third Rock in Cambridge, MA.
The regulatory progress--Scioderm also gained orphan drug status--is not something Ryan spends a lot of time boasting about. The key to gaining significant venture backing and the inside track at the FDA lay in early data from a very small group of patients. Impressively, Scioderm reported that 88% of target chronic lesions healed within a month, reducing the body surface of lesions and erosions by more than half in three months.
"Our data was very promising," says Ryan. "Even though it was very early data, it also was very encouraging across subtypes," while other therapies can be very subtype-specific. That gives Scioderm a leg up on other treatments in the pipeline. There is no approved therapy, something that has made this small biotech the sudden center of considerable attention from patients and their anxious families.
Right now the small company has a simple, central goal in mind: mounting a midstage study next year. It's possible that a therapy like this can get a significantly accelerated approval with the right results. And only 3 months of treatment will be required to show results.
But don't look for Ryan to make a lot of promises. He's as new to the BTD process as everyone else. And he's taking a cautious, wait-and-see approach on how it all pans out.
"I think it's still to be determined how much flexibility that gives us," says the CEO. The FDA is handling the BTD program on a product-by-product basis, looking at the "breadth of efficacy data."
Ryan and his team impressed the life sciences investors at Morgenthaler, says Chris Christoffersen, who heads up the life sciences team at the venture group. Some mutual colleagues they knew at FoldRx--which provided a successful exit for the VC group when Pfizer ($PFE) bought it out--made the introductions. If you combine the potential for a pioneering therapy for a disease that currently has no approved way to treat it along with the shorter development timeline that's possible here, he adds, Scioderm is a standout.
If it works, the company could also represent a near-term success.
"If it's successful, someone will want to come along with their big organization," says Christoffersen. For now, though, Ryan says he isn't focused on a buyout.
"My vision for the company is that we want to test this product and, if it works the way we think it does, make it available to everyone who needs it," says Ryan. It's not the money. It's not an M&A deal. Just get the program done.
That's a simple goal.
Investors: Morgenthaler Ventures led the A round with support from Technology Partners.
Biotech upstart joins Big Pharma cast on high-profile 'breakthrough' stage
-- John Carroll (email)