Shares of Achillion Pharmaceuticals, an emerging pharmaceutical company based in New Haven, CT, took a nice jump today base on an early clinical success with its hepatitis C drug. If the company successfully develop and marketed an approved drug, the stock would be worth over $70 a share: a nice jump from $2.70, where it closed today. The company has a long way to go before it can celebrate.

Hepatitis C is an infectious viral disease which affects the liver.  The virus is spread in a manner similar to HIV.  While reports claim it is slightly less infectious, the incidence of hepatitis C infection is much higher.  There are estimates that as many as twenty million people in the US carry this virus.

Most infected people exhibit few if any symptoms for many years.  In fact, it can take over twenty to thirty years before major symptoms start to appear.  Fibrosis (scarring) of the liver usually occurs first, followed in many cases by the cirrhosis and the onset of liver disease, which may lead to liver cancer or complete liver failure.  Currently the standard of care is peginterferon alfa-2a or -2b and the pro-drug Reterol (Schering Plough) which is also sold as Copegus by Roche.

Over three hundred drugs are in development for the hepatitis C market.  About 150 companies have developmental or clinical programs for this indication.

The report from Achillion was from preliminary data in a Phase 1 clinical trial on ACH-1625 a protease inhibitor.  Pharmacokinetic studies show rapid partitioning to the liver; the drug has been demonstrated as potent at low (nanomolar) concentrations to the hepatitis C virus (HCV); the company also reports that the drug has no serious toxicity issues.

The clinical trial is taking place in Europe, so no information is available on www.clinicaltrials.gov.  The company has given periodic reports on the progress of the drug.  The trial is designated as Phase 1b—safety with efficacy data.  The dosing ranges from 50 to 2000 mg: typical doses for antiviral drugs are really high: 2000 mg is two grams—approximately the same mass as to M&M's.  The results reported on Wednesday showed that the group dosed with either 200 mg or 600 mg of the drug showed a measurable drop in viral load.

These results are good, but the company has a long way to go.  It will have to work on a number of successful phase II clinical studies, long-term toxicity studies and at least two successful phase III trials.  If the news continues to get better, the stock will rise; however, as a result of the HCV data, I don't expect any major surge in the stock in the near term.  We're at least three to five years away from knowing if this company has a drug.  Things are looking good, but fundamental questions remain to be answered.