Gilead races to the FDA with stellar PhIII results for its breakthrough hep C combo

Norbert Bischofberger, Gilead's CSO

Gilead ($GILD) unveiled a package of stellar late-stage cure rates for its closely watched hepatitis C combination of the newly approved "nuke" Sovaldi (sofosbuvir) and the NS5A inhibitor ledipasvir, putting it on a short path to the FDA with a new drug application planned for the first quarter of the coming year.

The success of the combo will speed up the likely near-term approval of a new therapy that can offer a large portion of genotype 1 hepatitis C patients a quick "cure" without interferon or ribavirin--the holy grail of this R&D field over the past few years. As a result, current standards of care are being swept away as Gilead leads a pack of avid rivals angling for a share of a megablockbuster market. Bristol-Myers Squibb ($BMY) and AbbVie ($ABBV) are following closely behind Gilead in the race to the market.

The bottom line for Gilead's Phase III program: Researchers say that "of the 1,518 patients randomized to the 12-week arms of ION-1 and to all arms of ION-2 and ION-3, 1,456 patients (95.9%) achieved the primary efficacy endpoint of SVR12. Of the 62 patients (4.1%) who failed to achieve SVR12, 36 patients (2.4%) experienced virologic failure: 35 due to relapse and only one patient due to on-treatment breakthrough (with documented noncompliance). Twenty-six patients (1.7%) were lost to follow-up or withdrew consent."

Mark Schoenebaum, an analyst at ISI, was quick to offer kudos. In a note to investors he wrote that "the data are excellent." The data also back up the FDA's decision to single out this combo as a new "breakthrough" for patients, promising a fast-track review and a relatively quick decision.

"The results of the ION studies demonstrate that a simple, safe and short course of therapy with a single tablet regimen of sofosbuvir/ledipasvir can provide high cure rates among patients with genotype 1 HCV infection, while eliminating the need for both interferon and ribavirin," said Norbert Bischofberger, Gilead's CSO. "With the availability of these results, Gilead is finalizing its regulatory filing for sofosbuvir/ledipasvir, with the goal of submitting a New Drug Application in the first quarter of 2014."

Longtime observers of Gilead will recall that the company opted to walk away from Bristol-Myers Squibb and a highly promising pairing of sofosbuvir and daclatasvir, intent on developing its own in-house cocktail with ledipasvir. Gilead ignored critics who accused the company of putting the business ahead of patients, continuing a tradition of turning an icy shoulder to the complaints that come its way. But after betting billions that it could come up with a new standard of care for hep C ahead of all others, Gilead isn't likely to change its attitude toward a legion of critics anytime soon.

Gilead won.

- here's the release