Drug & Device Development

Stellar hepatitis C data puts Gilead farther ahead of pack

Last Updated: 2013-12-18 17:37:38 -0500 (Reuters Health)

By Ransdell Pierson and Bill Berkrot

(Reuters) - Gilead Sciences Inc released impressive late-stage data for its once-daily combination pill to treat hepatitis C, advancing its lead in the race to develop new, all-oral treatments for the liver disease, and pushed up its timeline for seeking U.S. approval.

Gilead on Wednesday unveiled initial results from three Phase III studies that demonstrated cure rates well in excess of 90% with as little as eight weeks of treatment for some patients.

The findings were achieved without the use of either injectable interferon or oral ribavirin, both of which have troublesome side effects.

The tough-to-tolerate older drugs have led thousands of patients to delay treatment for the potentially fatal disease and await new options.

The Phase III trials tested the combination pill in subjects with the most common, but hardest to treat, genotype 1 strain of hepatitis C in both previously untreated patients and those who had failed to be helped by prior treatment. They also included difficult to treat patients with cirrhosis, who tend to be farther along in the liver-destroying disease.

Gilead said it would file in the first quarter of 2014 for U.S. approval of the combination treatment that pairs its just-approved Sovaldi (sofosbuvir) and its experimental drug ledipasvir. The company previously said it would seek marketing approval in the first half of next year.

Sovaldi, which costs about $84,000 for a course of treatment, is a nucleotide analog polymerase inhibitor designed to block an enzyme the hepatitis C virus needs to copy itself. Ledipasvir belongs to a promising new class of drugs that work by blocking the NS5A protein, which the virus also needs to replicate.

Current standard regimens include both interferon and ribavirin and must be taken for 24 to 48 weeks, curing about 75% of patients.

Gilead tested its combo pill at a variety of treatment durations both with and without ribavirin. But the high cure rates without ribavirin, which can cause rash, anemia and other side effects, are likely to grab most of the attention.

In a study called ION-3 involving 647 previously untreated patients without cirrhosis, 94% achieved sustained virologic response (SVR) after just eight weeks of treatment, rising to 95.4% with a 12-week regimen.

In ION 2 - a trial of 440 more difficult to treat patients who were not cured by prior treatment, including 88 cirrhotics - 93.6% were cured by the once daily pill after 12 weeks, while the cure rate rose to 99.1% with 24 weeks of treatment.

In ION-1, which looked at 865 previously untreated patients, including 136 with cirrhosis, the Gilead pill cured 97.7% with 12 weeks of therapy.

"In general, the data position Gilead to dominate the genotype 1 landscape," ISI Group analyst Mark Schoenebaum said in a research note.

Genotype 1 accounts for about 70% of U.S. infections. Hepatitis C affects an estimated 170 million people worldwide, and if left untreated can lead to cirrhosis, liver cancer, or the need for a new liver.