Biotech Values

[mskatiescarletohara]

VRTX chimes in with very impressive results from a small study.....no detectable virals loads after twelve weeks of continued standard of care treatment after 14 days of VX-950 dosing for chronic patients. WOW.


Researchers Report Initial Results for 14-day Phase Ib Study of VX-950, and Pegylated Interferon, Showing Anti-HCV Activity in Combination in Hepatitis C Patients
Saturday April 29, 12:00 pm ET
-New data show that plasma HCV RNA levels were below limit of detection (10 IU/mL) in 8 of 8 patients continued on peg-IFN+RBV for 12 weeks-

VIENNA, Austria, April 29 /PRNewswire-FirstCall/ -- Data presented at the 41st Annual Meeting of the European Association for the Study of the Liver (EASL) in Vienna today show that when VX-950, an investigational oral hepatitis C virus (HCV) protease inhibitor being developed by Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX - News), was dosed with pegylated interferon alfa-2a (Pegasys®; peg-IFN), the combination was well-tolerated and achieved a dramatic reduction in plasma viral RNA levels in patients with chronic genotype 1 HCV infection through 14 days of dosing. At day 14, the majority of patients (6 of 8) receiving the combination had HCV RNA levels below the limit of quantitation (30 IU/mL, as measured by the Roche TaqMan® assay), and 4 of 8 patients had HCV RNA levels below the limit of detection (10 IU/mL, Roche TaqMan®). All patients enrolled in the 14-day study subsequently received follow-on treatment with peg-IFN and ribavirin (RBV). Researchers reported for the first time today that 8 of 8 patients who received VX-950 and peg-IFN in combination for 14 days have no detectable virus in their blood at the end of 12 additional weeks of peg-IFN+RBV dosing. These patients continue to receive peg-IFN+RBV therapy. All patients were offered follow-on peg-IFN+RBV treatment according to clinical practice at the investigator sites.


"In the 14-day study, VX-950 in combination with pegylated interferon produced a very rapid viral response in each of these genotype 1 patients, and no serious adverse events were observed," said Henk W. Reesink, MD, Associate Professor of Medicine at Academic Medical Center in Amsterdam, and a lead investigator for the study. "The continued viral suppression during follow-on therapy points to the robustness of the viral response to VX-950 and pegylated interferon, and is encouraging for the design of future VX-950 studies that seek to evaluate the potential for short-course, curative therapy."

Study Design and Results

The 14-day, randomized, blinded, placebo-controlled Phase Ib study enrolled 20 treatment-naive patients with genotype 1 HCV, the most prevalent and difficult to treat form of HCV infection. Patients were randomized to receive a new tablet formulation of VX-950 at a dose of 750 mg every eight hours (q8h) in combination with a standard dose of peg-IFN (n=8), the same dose of VX-950 administered alone (n=8), or a standard dose of peg-IFN alone (n=4). The median viral load for all patients at study entry was 6.65 log10 IU/mL HCV RNA (approximately 4.4 million IU/mL). In this Phase Ib study, the combination of VX-950 and peg-IFN produced an initial median reduction in plasma HCV RNA of more than 3 log10 in the first two days. Antiviral results for all arms after 14 days of dosing were as follows:

* A median 5.5 log10 reduction in HCV RNA was observed in patients
receiving VX-950 and peg-IFN; 6 of 8 patients had viral levels below
the limit of quantitation (30 IU/mL) at 14 days, and 4 of 8 also
achieved viral levels below the limit of detection (10 IU/mL).
* A median 4.0 log10 reduction in HCV RNA was observed in patients
receiving VX-950 alone; 1 of 8 patients had viral levels below the
limit of detection (10 IU/mL).
* A median 1.0 log10 reduction in HCV RNA was observed in patients
receiving peg-IFN alone; no patients had viral levels below the limit of
quantitation (30 IU/mL) at 14 days.

Following the 14-day Phase Ib study, patients were rolled onto follow-on treatment with peg-IFN and ribavirin.

Safety

A complete safety review has been conducted. All patients completed dosing and no serious adverse events were reported. The most common adverse events among all treatment arms, all of which were mild to moderate in severity, were headache, myalgias, dry skin, diarrhea, nausea, chills and rash. VX-950 did not appear to substantially increase the frequency or severity of these events when added to peg-IFN, and the observed safety profile supports the evaluation of VX-950 in studies of longer duration. All adverse events in the patients receiving VX-950 alone were reported as mild. Typical interferon-related side effects, of mild to moderate severity, were reported in the patients that received peg-IFN along with VX-950 or placebo.