Biotechnology startup’s re-targeting approach offers novel take on drug development

A biotechnology startup has developed a technology platform that analyzes drugs to identify previously unrecognized targets and assess the best new use for them. The company’s approach is designed to cut the time and money spent on research and development with the potential to transform drug development.

NPharmakon’s approach involves a platform that uses a proprietary computational method, takes a different approach than how pharmaceutical R&D typically works. Usually, a disease is specified first, then a molecular target critical for that disease is identified, and then a lead molecule active against the target is found.

NPharmakon’s process goes in the opposite direction, so the company can’t specify in advance which disease will become the new indication for a particular drug. CEO and co-founder Dmitri Rebatchouk said: “In a way, we are allowing the drugs to speak for themselves and tell us what they could be used for. But once we do that we can develop drugs much faster, so it’s a tradeoff well-worth making.”

The Piscataway, New Jersey company re-targets safe molecules that have already undergone clinical testing, but have been abandoned.


The business model for the technology platform works like this. A partner company designates targets and nPharmakon identifies safe drugs active against those targets. The partner company pays research costs and success-based milestones.

So far, it’s found new targets for about one dozen drugs, three of which are in development. Its lead drug is for skin disorders involving excessive pigmentation such as melasma — a pigmentation disorder affecting 5 million people in the US and 3 million in Japan, according to data from the company. The drug has demonstrated efficacy in a pilot clinical study. It also has two other drugs in pre-clinical development: one to treat hepatocellular carcinoma, the most common type of liver cancer, and the other for atopic dermatitis.

The company, which recently presented at the Mid-Atlantic Diamond Ventures investor forum, is currently raising $800,000 to advance its treatment for pigmentation disorders.

“The realities of the labor market are such that it’s easier than ever before to find experienced pharmaceutical professionals to join in a high potential program,” Rebatchouk said. “It’s easier to assemble a team around a program rather than assemble a team and then try to find a drug.”

We’re bringing active investors and corporate development execs together with promising startups at MedCity News INVEST. Join us April 12-13, 2016, in Chicago for the premier national investing conference based in the Midwest.

No comments