Lightning Round: Orphans,Ophthalmology, Osteoporosis, and Others
Hurricane Sandy looms just outside the New York Harbor as this article goes to press, and more than a few innovative pipeline
drugs couldn't be funneled neatly into the therapeutic areas discussed above. Some of these products will likely generate
floods of revenue for their sponsors, while others may get hung out to dry, resulting in painful write-offs and disappointed
patients. We're bracing for Sandy, but in the meantime, keep your eye on the following compounds:
» Merck's odanacatib, in Phase III trials for postmenopausal osteoporosis and male osteoporosis. "I like the early and mid-stage
clinical data" supporting odanacatib, says Funtleyder. "Osteoporosis isn't the most glamorous disease, but a lot of people
have it, and bisphosphonates aren't exactly the perfect cure." Several independent forecasts are predicting blockbuster sales
for odanacatib, with an estimated approval date in early 2014.
» Sarepta Therapeutics' eteplirsen, a fast-tracked orphan drug indicated for Duchenne muscular dystrophy (DMD). Some analysts,
including Selvaraju, suspect that orphan drug companies are reaching a point of unwarranted enthusiasm, as evidenced by relatively
large market caps; Sarepta (formerly known as AVI BioPharma) sports a $1 billion valuation, "based on Phase II data from 12
people," he notes. But there's no treatment currently for DMD, and history has shown that the pricing environment for orphan
drugs is exceedingly favorable for drug companies, a necessary incentive, some argue, for active drug development in rare
» Lundbeck's vortioxetine, for major depressive disorder. Takeda has rights in the United States and Japan, and the drug is
"classified as an SSRI, but has a slightly differently mechanism," says Latwis of Decision Resources. Vortioxetine is filed
in the United States and Europe, and "we have it launching in late 2013, and reaching $1.1 billion by 2018," says Latwis.
October cover story on Lundbeck.)
» Advanced Cell Technology's (ACT) human embryonic stem cell-derived retinal pigment epithelium cells for macular degeneration.
While only in Phase I/II, ACT "has demonstrated the ability, with a very small volume injection of their stem cells, to regenerate
not only retinal epithelium tissue, but also functional photo receptors," says Selvaraju. (See
stem cell feature, also in the October issue, for further detail.)
» Actelion's macitentan, an orphan drug filed in the United States for pulmonary hypertension (PAH), and a follow-on to Actelion's
blockbuster PAH drug Tracleer. Macitentan is a once-daily formulation, whereas Tracleer is administered twice daily. Decision
Resources predicts an approval and launch late next year, with sales hitting $1.2 billion by 2018. Thompson Reuters puts 2017
sales at $990 million.
» Vertex's VX-809/Kalydeco combination, for cystic fibrosis. Last January, Vertex received approval for Kalydeco (ivacaftor)
three months before its PDUFA date, and is now testing a fast-tracked combination of VX-809 (lumacaftor) and ivacaftor. VX-809
was discovered as part of aresearch collaboration with Cystic Fibrosis Foundation Therapeutics. Adis Insight predicts an approval
date in January 2016, and blockbuster sales two years later.
Forecasting data in Pharm Exec's 2013 Pipeline Report relies in part on Springer's Adis R&D Insight, Thomson Reuters Cortellis,
and EvaluatePharma data. We very much appreciate the use of these resources.