Biotech Achillion Pharmaceuticals ($ACHN) is working to get its name on the short list of blockbuster hepatitis C contenders, revealing promising Phase II data for a combination of its antiviral with Gilead Sciences' ($GILD) leading Sovaldi.
In an 18-patient study, ACH-3102 and Sovaldi posted 100% cure rates for the 12 subjects receiving treatment, and the cocktail was well-tolerated with no significant adverse reactions, the company said. Now Achillion plans to expand the trial, moving the 6 observational patients into the treatment arm and enrolling 6 more, expecting to report full Phase II data by the end of the year.
The trial's principal goal is to see how well ACH-3102, an NS5A inhibitor, can pair up with Sovaldi and cure treatment-naive patients with genotype 1 hep C without the use of ribavirin.
The mid-stage results sent Achillion's shares up about 18% in premarket trading on Friday, stoking investors' hopes that the well-traveled biotech might attract a lucrative buyout offer now that hep C has become a cause célèbre among global drugmakers.
Leaders in the hep C space already have combo therapies of their own, including Gilead and AbbVie ($ABBV), which each expect an FDA approval this year. But the bull case for Achillion lies in the breadth of the market. Earlier this summer, Merck ($MRK) signed on to pay about $3.9 billion for Idenix Pharmaceuticals and expand its number of shots on the hep C goal, and Achillion could potentially fill that role for a deep-pocketed drugmaker looking to cut the line.
And the biotech's pipeline isn't limited to an NS5A blocker. Sovaprevir, the company's Phase II NS3/4A protease inhibitor, came off clinical hold in June, and the treatment has the agency's fast-track designation. Furthermore, Achillion has high hopes for ACH-3422, an in-house nucleotide that could co-star in a future combo therapy with ACH-3102, CEO Milind Deshpande said.
"As we continue to achieve clinical milestones, we remain focused on execution of the broader clinical development strategy for our HCV portfolio," Deshpande said in a statement. "We expect that Phase I proof-of-concept results with ACH-3422 will be reported during the fall of this year, which we anticipate will lead to the start of a Phase II combination program to evaluate our proprietary doublet regimen for an 8-week, or potentially shorter, treatment regimen for HCV that will begin before the end of 2014."
- read the statement