Seaside Therapeutics – 2012 Fierce 15

Randall Carpenter, CEO of Seaside Therapeutics

Based: Cambridge, MA
Founded: 2005
CEO: Dr. Randall Carpenter
Clinical Focus: Autism spectrum disorders, Fragile X syndrome

The Scoop: Seaside Therapeutics is the rare biotech startup that has plunged headlong into autism, tackling a risky area of neuroscience that has recently attracted more interest from pharma. Swiss drug giant Roche inked a deal with Seaside that was announced in June, giving the startup its first Big Pharma partner and a licensee for its experimental mGluR5 antagonists to potentially treat autism and a closely linked genetic disease called Fragile X syndrome (FXS). Autism spectrum disorders were found in 1 in 88 children in the U.S., according to a 2008 Centers for Disease Control and Prevention study, with cases of the disorders on the rise. Seaside has a lead candidate in mid- and late-stage trials for autism and Fragile X, respectively, that is one of the most advanced compounds in the world that addresses the core symptoms of the disorders.

What Makes it Fierce?

When Randy Carpenter was a kid growing up in Clarkston, MI, he felt the impact of growing up with a sister who had an intellectual disability. Fast-forward to the present: He's now the CEO and co-founder of Seaside Therapeutics, where he's leading a charge to advance some of the first drugs to address the suspected root causes of autism.

"This means a lot to me," Carpenter says. His leadership in autism drug development has landed him the role of lead scientific adviser for a massive alliance to advance development of drugs for autism spectrum disorders called the European Autism Interventions--A Multicenter Study for Developing New Medications (EU-AIMS).

Seaside has taken a unique approach to building a biotech business. The Cambridge, MA-based developer has managed to focus both on ushering a lead program into late-stage development while sticking to early research on new targets to treat autism and related disorders. Its main funder over the past 7 years has been a family trust with an interest in autism research, meaning that Seaside has avoided being at the mercy of the limited timelines and expectations of venture capitalists.

Founded on the landmark research of MIT neuroscientist and company co-founder Mark Bear, Seaside has been the little guy among behemoths such as Roche ($RHHBY) and Novartis ($NVS) in the hunt to develop drugs against Fragile X, the most common genetic cause of autism that results from an abnormality in the FMR1 gene.

The genetic disease often causes behavioral problems and hobbles learning, with symptoms linked with the overproduction of the neurotransmitter glutamate and interruption of normal protein synthesis at synapses in the brain. And Seaside's top drug, arbaclofen (STX209), is a GABA-B agonist that has shown evidence of normalizing increased protein synthesis in the brain, Carpenter explains. It could help patients with the disorder learn from experience and reverse some of the social withdrawal seen in patients.

All of this, of course, must be proven in clinical trials. Seaside faces a major test for STX209 in patients with autism, with results due out this fall from a Phase IIb study of the drug in patients with autism spectrum disorders. The company also has two Phase III trials of the drug in patients with Fragile X syndrome, with both pivotal studies expected to yield data next year.

Seaside stuck a deal with Roche announced in June that enables the 30-person biotech to fund the multipronged STX209, Carpenter says. Roche has also picked up options from Seaside to commercialize its lead drug if it achieves certain goals during development. Roche, which had joined forces with Seaside on a pre-competitive basis prior to the pact, licensed some of Seaside's mGluR5 antagonists on an exclusive basis. Roche already has a similar mGluR5 drug of its own for FXS in Phase II studies.

The Roche deal helped diversify Seaside's funding, the bulk of which has come from the Barony Trust, though Carpenter declined to discuss his main funder because of his backer's desire to remain out of the spotlight. (The New York Times reported in June that the bulk of Seaside's $90 million raised had come from Barony.)

Even with the Roche pact in place, Seaside and its investors aren't in this game to cash out at some pre-determined point and make a killing. Rather, they have built the company to advance drugs that treat perplexing diseases that cause intellectual disabilities. Right now, patients with autism only have drugs such as Johnson & Johnson's ($JNJ) Risperdal and Bristol-Myers Squibb's ($BMY) Abilify, antipsychotics that quell aggression in patients yet fail to address the core learning and communication flaws in them.

"When I was a kid I told my mother that I'm going to be a doctor and fix my sister," Carpenter says. "So the possibility of that happening is beyond comprehension."

Known investor: Barony Trust. (Seaside has also won grant support for its research.)

-- Ryan McBride (Email | Twitter)

Seaside Therapeutics – 2012 Fierce 15

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