TARGET INDICATION Hepatitis C
DEVELOPMENT Phase II
LAUNCH DATE 2010
PEAK ANNUAL SALES $2 billion
Hepatitis C has long presented a golden opportunity for innovative drug development. The gold-standard therapy, a combo of
pegylated interferon and ribavirin (IFN/RVN), rakes in $3 billion a year and counting, despite major downsides, including
48 weekly injections complete with a side effect dubbed "the flu from hell," a 50 percent failure rate, and a price tag of
$25,000. Enter Novartis, the crafty Swiss pharma giant: To take the pulse of the HCV pipeline, just check out which compounds
it's betting licensing bucks on.
Nezam Afdhal, Beth Isræl Deaconess Medical Center
The treatment picture for hep C is about to switch from black and white to color, with more options that are kinder to patients
and meaner to the virus. Tomorrow's standard of care will consist of triple-drug combos that attack fast-mutating HCV from
different angles, as in the model for HIV.
"As we enter the age of STAT-C—Specifically Targeted Antiviral Therapy for HCV—the future of treatment looks very promising,"
says Nezam Afdhal, MD, chief of hepatology and director of the Liver Center at Beth Israel Deaconess Medical Center. "These
small molecules should enable us to make people negative more quickly, with fewer side effects, and increase the cure rate."
Vertex's telaprevir (VX-950), a cleanly designed protease inhibitor, is the most ambitious advance—and the one Novartis failed
to nab (J&J will co-promote). The small molecule blocks an enzyme critical to HCV replication, and early data show that it
clears the virus four times faster than IFN/RVN, with a 70 percent success rate and few nasty side effects. Although its current
three-times-a-day oral formulation may present adherence problems, pills beat interferon injection hands down. Vertex is currently
conducting two big Phase II studies to PROVE (the trial's hopeful name) that adding VX-950 to IFN/RVN cuts the virus faster
Another straight-shooting antiviral, Idenix's Valopicitabine (NM-283), knocks out HCV's RNA polymerase. Nowhere near as fast-acting
as the Vertex compound, Valopicitabine does have the advantage of once-daily dosing—although Phase II data found iffy gastro
complications. Next up: Phase III trials pitting NM283/INF/RBV against NM283/INF as first-line and back-up.
Human Genome Sciences' HCV contestant is Albuferon, a high-tech entry in the race for the pegylated interferon market. By
fusing the immune modulator with human albumin, a highly concentrated protein found in blood plasma, the savvy biotech was
able to design a slow-release version. According to recent Phase II data, one shot of Albuferon every two weeks works at least
as well as a once-a-week shot of pegylated interferon. Phase III studies are enrolling with double-dose monthly injections—and
hoping to ACHIEVE (another winning trial name) good safety data.
Cut to Novartis and its recent hep C spending spree. The company has bought marketing dibs on four biotech-built compounds:
Albuferon, Valopicitabine, and two Phase I small molecules with unique mechanisms of action. Together with its generic ribavirin
Sandoz deal, this portfolio may have Novartis playing hep C bartender, mixing and matching state-of-the-art drug cocktails
for years to come. –WA
PUT ON WAIT: Sanofi-Aventis' Diet-Pill Problem
TARGET INDICATION Obesity
LAUNCH DATE 2007
"Any day now." That's the mantra associated with Sanofi-Aventis' much-delayed anti-obesity compound. After three years of
waiting for US approval of Acomplia, drug watchers are still holding their breath. And Sanofi's domestic drug marketers are
scratching their heads, wondering when they will get their turn at promoting what's being trumpeted as possibly the industry's
biggest blockbuster in decades.