EnVivo Pharmaceuticals – 2012 Fierce 15

Tools
Kees Been, CEO of EnVivo Pharmaceuticals

Based: Watertown, MA
Founded: 2001
CEO: Kees Been
Clinical Focus: Alzheimer's and schizophrenia
Website: envivopharma.com

The Scoop: EnVivo has tackled Alzheimer's disease--an area where most developers fear to tread. But instead of going after the amyloid beta hypothesis, the biotech is blazing a new path toward a potential therapy for one of the biggest unmet medical needs in the business.

"What our compound does," says CEO Kees Been, "is basically facilitate neuronal firing, the transmittal of the signal across neurons," with an eye to improving the cognitive abilities and clinical function of millions of people diagnosed with Alzheimer's. And the developers have data to back up their belief that the same approach will help patients suffering from schizophrenia.

What Makes it Fierce?

EnVivo's lead drug--EVP-6124, an alpha-7 agonist--works by sensitizing a key nicotinic acetylcholine receptor, hopefully without the toxic effects seen in alternative approaches. And they have some important rodent data, at very low doses, to back them up. Even more importantly, though, the biotech came up with a set of statistically significant human results for the top dose in a Phase IIb dose-escalating study in July.

At a 2 mg dose, the highest, patients saw improved cognition after 23 weeks of dosing. There was also an improvement in clinical function, a key measure for payers looking to see an economic payoff among patients able to sustain an independent lifestyle.

Now the focus is on designing and executing a Phase III study in the near term, putting little EnVivo and its staff of 75 in line to join the rare crowd of developers in a late-stage field dominated by pharma giants able to afford trials that can cost hundreds of millions of dollars. It's possible that a pivotal trial that reflects the same clinical effect over 6 months with a year of safety data could get a green light from regulators. And the potential commercial payoff would be enormous.

EnVivo--which has also posted positive IIb results for 6124 in schizophrenia--has an advantage in its sole big investor: Fidelity. A venture round in 2008 has been followed up with fresh financing two years ago. And Been says Fidelity is committed to going the distance.

"We are very fortunate with our investor Fidelity, which has a long-term view," says the CEO. "They are allowing us to execute on a long-term strategy; to build a company that develops and ultimately commercializes drugs." The goal, he says, is "to build something in this very difficult space."

"The question is to what extent payers are going to pay for these probably very expensive drugs," Been adds. The reason we've seen high prices on orphan indications without a substantial backlash among payers is that the tiny populations involved don't put a huge burden on the system. With 5 to 6 million Alzheimer's patients anxiously looking for some new treatment that can help, "that's a whole different kettle of fish. The hurdle of showing an effect will be pretty darn high."

If EnVivo clears it, though, the company will emerge as a pioneer.

Investor: Fidelity

-- John Carroll (Twitter | LinkedIn)