Alkeus Pharmaceuticals – 2012 Fierce 15

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Leonide Saad, CEO of Alkeus Pharmaceuticals

Based: Boston
Founded: March 2010
CEO: Leonide Saad
Focus: Treatments for blindness
Website: www.alkeus.com

The Scoop: Biotech groups swamped Joshua Boger with offers after he retired in 2009 from his role as CEO of Vertex Pharmaceuticals ($VRTX), which he had founded 20 years earlier and built into one of the most vibrant drug research operations in the world. He wasn't interested in those offers--until along came Leonide Saad and his tiny startup Alkeus. Alkeus CEO Saad, who recruited Boger to the company earlier this year, has made strides in advancing a potential breakthrough treatment for blindness-causing illnesses such as Stargardt disease. Combine Boger's rare instinct for developing effective drugs with Saad's tenacity and you have a biotech outfit worth watching.

What Makes it Fierce?

With Saad working as the only full-time employee, Alkeus has a trial-ready compound and a plan to rapidly build a body of evidence of its safety and efficacy for Stargardt. The genetic disorder, for which there are no approved drugs, afflicts 1 in 10,000 children and is the top cause of blindness-causing macular degeneration in kids. Alkeus has found what could be an elegant weapon against the disease and other causes of blindness such as dry age-related macular degeneration (AMD).

Researchers believe that vitamin A serves as a light switch for vision. Specifically, vitamin A plays a key role in photoreceptors in the retina to signal the presence of light to the brain. In a healthy patient, the cells pump out used vitamin A, which gets recycled for reuse. Stargardt patients have a single-gene defect that causes that cellular pump to malfunction, leading to toxic accumulations of vitamin A in the retina and progressive loss of vision. Alkeus' drug candidate, ALK-001, offers a chemically modified version of the vitamin that is designed to reduce formation of those toxic clumps.

That's the idea, simple as it sounds. While other labs undertake trials of complex cellular or gene therapies to fight Stargardt, Saad and Boger, the company's executive chairman, plan to push ahead with what Saad calls a "clean-burning" vitamin A. The company has permission to begin clinical trials of ALK-001--which is based on the pioneering research of company co-founder Ilyas Washington at Columbia University--and now aims to begin clinical trials next year. Within three to 5 years, Saad says, the company could have the first disease-modifying drug for Stargardt.

Boger, who made an investment in Alkeus, headlines the group of individual investors backing the biotech, and both he and Saad say the company can continue to operate without requiring a large round of venture capital any time soon. They're keeping a lid on how much the startup has raised, but Saad says it's enough to fuel operations for the next two years. Trials for rare diseases require way fewer patients than, say, a new treatment for a widespread illness such as heart disease or diabetes.

"When you develop a drug for a rare indication," Saad says, "you can do a lot of things and be very capital-efficient."

Boger has stayed on the board of directors at Cambridge, MA-based Vertex, but in 2009 he stepped down from his role as CEO and made known that he wasn't interested in getting involved with any other biotech operations, he says. With the company founder advising top execs in the boardroom, Vertex advanced the big-selling hepatitis C drug Incivek to market in 2011 and early this year won approval for Kalydeco, the first FDA-approved drug that addresses the underlying cause of cystic fibrosis.

Yet Boger, who chairs the R&D committee for the board of directors at Vertex, has kept a close eye on exciting discoveries in biotech. And early last year he spotted a study in The Journal for Biological Chemistry from Columbia, where Washington and his colleagues used deuterium-enriched vitamin A to slow the formation of toxic clumps in the eyes of mice and prevented visual loss.

"I was just so impressed with the elegance of the thinking and the elegance of the idea," Boger says, "that I made an electronic copy of it and put it in my follow-up-on-this file."

At the time of the publication, Boger had no idea that Saad, a former VC and biopharma consultant, had already formed Alkeus to commercialize Washington's work. (Saad has a Ph.D. in engineering with a focus on regenerative medicine from MIT.) Little did Boger know that Saad had been asking around about Boger, and he entered the MassChallenge startup competition in Boston because Boger was one of its judges. Saad came out a $100,000 winner of the contest and gained Boger's attention, persuading the industry heavyweight to get involved in the company.

"I've been spending a lot of time with this, a lot more time than I thought I might, and that's been a voluntary thing," Boger says.

Boger saw several parallels between Alkeus' work on Stargardt and Vertex's efforts in cystic fibrosis--both are recessive genetic disorders that afflict about 30,000 U.S. patients.

"I thought I knew something about how to develop a drug in that kind of genetically defined disease," he says, "and I think my experience with cystic fibrosis convinced me that working in areas like this have advantages both commercially and in the regulatory environment that Big Pharma is only beginning to realize."

"All that," he added, "made me feel like maybe I should make an exception to my vow not to get back into the business."

Investors: Joshua Boger and undisclosed private investors.

-- Ryan McBride (Email | Twitter)