Contributing Writer

If you haven't heard of Tikvah Therapeutics Inc., you're not alone. Since its April 2006 founding, the Atlanta-based start-up has quietly pursued a strategy of licensing intellectual property around the use of established drugs in new psychiatry and neurology indications. It now has two drugs in the clinic, one of which "should be near Phase III" by the end of 2007, said President and CEO Harold Shlevin.

On the psychiatry side, Tikvah is evaluating a low-dose formulation of an NMDA partial agonist for use in conjunction with psychotherapy. Several investigator-run clinical trials have indicated that using the drug as an adjuvant to counseling accelerates the "elimination rate" of unwanted behaviors, such as the stress response associated with various phobias or post-traumatic stress syndrome. It may also have the potential to treat pain and personality disorders.

Although the drug itself has been marketed for another indication for 20 years, Tikvah licensed the patents surrounding its use in new indications from Emory University in Atlanta and plans to take over the investigational new drug and an ongoing investigator-run Phase II clinical trial in early 2007. Given the size of the psychotherapy market, Shlevin said it may eventually "make sense to seek a partner" for the drug. He said he anticipates that in addition to pharmaceutical companies, government agencies may be interested due to the drug's potential to treat post-traumatic stress following military combat.

On the neurology side, Tikvah is working on a reformulated version of an HDAC inhibitor approved for spinal muscular atrophy (SMA). The drug, which was licensed from a pharma company, is being evaluated in its new formulation in an investigator-run Phase II clinical trial, but Shlevin anticipates that additional pharmacokinetic and pharmacodynamic work will be required to establish the optimum dose. Once that is complete, development should be relatively straightforward, given the existing regulatory pathway in SMA, and Shlevin sees that as an indication Tikvah could reach with a niche sales force.

"There are only a few major medical centers where spinal muscular atrophy is treated, and we could probably market that drug with a five- to 10-person sales force," he said. He added that Tikvah will use SMA as a "stepping stone indication" to pursuing approval in larger markets such as amyotrophic lateral sclerosis (ALS) and multiple sclerosis.

Tikvah has other opportunities in its pipeline that Shlevin said he can't talk about yet, and the company is actively seeking to acquire additional products in the CNS space. To maintain the reduced risk business model, however, Tikvah focuses on products that are already approved either in the U.S. or overseas, have demonstrated clinical proof of efficacy in multiple studies for a new indication, present no manufacturing issues, and are protected by patents.

While the difficult funding environment for early-stage research has driven a surge in biotechs with reduced risk business models, Tikvah is not virtual. Its seven-member crew will grow to around 20 by the end of 2007, with most new hires experienced in clinical and regulatory development. Notably absent will be traditional functions such as finance, accounting, legal and business development, all of which are handled by Paramount Biosciences LLC of New York.

"It doesn't come for free, of course, but it comes at a reasonable rate, and that allows us to focus on the clinical programs," Shlevin said. He added that even on the clinical side Paramount offers preferred vendors products and services such as clinical trial insurance, saving time that would otherwise be spent calling around to get recommendations and price quotes.

Paramount founded Tikvah in the same way it has founded approximately 40 other startups over the past 15 years: its team of in-licensing experts acquired a portfolio of related products, recruited a senior management team, and formed the new company. Shlevin was recruited from Solvay Pharmaceuticals SA, where he served as global senior vice president.

"I was attracted by Paramount's track record and proactive nature of building companies," Shlevin said. "These guys were getting their companies public through reverse mergers long before reverse mergers became the name of the game."

Paramount is thus far the only investor in Tikvah. Shlevin declined to specify how much has been invested thus far, but did say the company will likely start fundraising in 2007 as it advances its clinical trials. Tivkah may also receive funding through a loan from Georgia's Life Sciences Facilities Fund. It is currently located in the Advanced Technology Development Center, a science and technology incubator at the Georgia Institute of Technology.