Aragon Pharmaceuticals – 2012 Fierce 15

Richard Heyman, CEO of Aragon Pharmaceuticals

Based: San Diego
Founded: 2009
CEO: Richard A. Heyman
Clinical Focus: Cancer drugs
Website: aragonpharm.com

The Scoop: In a very short period of time, prostate cancer patients have been handed several new treatment options to consider. First came Dendreon's ($DNDN) complex cancer vaccine Provenge, a scientific breakthrough that quickly disappointed analysts when it turned in lackluster sales results. Then Johnson & Johnson's ($JNJ) Zytiga followed, an oral treatment that excited a considerably larger group. And just days ago, Medivation's ($MDVN) Xtandi was stamped with an approval.

But there's another treatment in mid-stage development that may well roil the market one more time. Aragon Pharmaceuticals plans to offer the world the first glimpse of fresh data on ARN-509 at a scientific meeting in Vienna at the end of this month. And CEO Rich Heyman thinks the next-gen anti-androgen therapy can wow the crowd.

What Makes it Fierce?

The San Diego-based biotech was able to enroll 100 patients in the study much faster than initially anticipated. But the developer's backers stepped up in March with a timely $42 million round to fund the work.  

"If the patients are responding and tolerating the compound well they will stay on the compound and we will monitor them," says Heyman. "Realistically, assuming things go well, it will probably be another 9 to 12 months, with the data reading out in the spring or early summer."

Aragon's big cancer program is built around the idea that if you can build a better anti-androgen drug, you can offer new hope to castration-resistant prostate cancer patients. And the biotech sees its therapy as a logical second-generation therapy that offers some big potential as a future combo package.

If the data comes out positive, such that it looks like 509 may go on to become a frontline treatment for the disease, Heyman and the investors will have another big decision to make: Partner ahead of Phase III, or raise additional funds to take it into a pivotal study on their own--significantly raising the stakes on a late-stage pact.

There's one big hurdle that Aragon has to complete before then, though. ARN-509 was identified by Charles Sawyer's team at UCLA, which also delivered Xtandi to Medivation. Medivation has since sued UCLA, claiming ownership under its licensing pact. But Heyman insists that the university vowed explicitly that it had the rights to 509 separate from Xtandi. And he adds that his investors vetted the issue thoroughly ahead of the recent capital injection.

"In some ways the fact that the Medivation data looked good, improved survival and was well tolerated, we view as a positive event," says Heyman, who coincidentally was talking to FierceBiotech at the exact same time Medivation announced the FDA approval. "We feel we're going to be best-in-class and we're very cognizant that it came from the same university and is chemically of the same ilk." Those facts significantly de-risk the potential safety issues involved, he adds.

In addition to 509's potential as a combo treatment with Zytiga, there's also potential for a tie-up with a Pi3K program. And early next year Aragon plans to advance an experimental breast cancer therapy that blocks estrogen signaling into the clinic as it thinks beyond the possibilities for 509.

Investors: The Topspin Fund, an investment group of James Simons, Leo A. Guthart and Steve Winick, led the last round, with Aisling Capital, OrbiMed Advisors and The Column Group joining.

-- John Carroll (Twitter | LinkedIn)

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Aragon Pharmaceuticals – 2012 Fierce 15