MS blockbuster drugs

[chantell.reagan]

During the September 2009 Webinar, the following question was submitted and we’d like to open it for a general discussion in our community:

"Are there any phase III products in the pipeline for multiple sclerosis, either oral or injectable that are expected to be blockbusters?"

[chantell.reagan]

There are approximately 12 agents in the late-stage pipeline for multiple sclerosis (MS), 8 of which are being studied in an oral formulation. These new oral agents may change the current landscape of how MS is being treated with currently available injectable agents such as Avonex, Betaseron, Copaxone, Rebif, Tysabri and the newly approved Extavia. Nationally MS affects 400,000 people in the US and 2.5M worldwide. MS drugs accounted for 18.1% of pharmacy spending for specialty drugs and were the 2nd driver of drug trend in 2008, according to Medco. We will explore 3 oral agents and 1 pegylated interferon below for their blockbuster potential:

fampridine SR by Acorda/Biogen is a potassium channel blocker

•Clinical differentiation- in a placebo-controlled trial published in Lancet in 2/09 the proportion of timed walk responders was higher (statistically significant) in the fampridine group than in placebo; fampridine can be used in combination with the currently available MS drugs since it’s intended for symptom relief
•Pipeline status – NDA was resubmitted after additional information was needed (4/09); priority review was granted; FDA advisory board to meet 10/14/09 with review date scheduled on 10/22/09
•Analyst projections – Seeking Alpha ($1B)

Mylinax (cladribine) – an oral formulation by Merck KGA of the currently available purine-analog in IV formulation (frequently used off-label for MS)

•Clinical differentiation - clinical efficacy has been seen in placebo-controlled trials, but there is a concern for increased incidence of cancer as reported in 4/09
•Pipeline status – NDA filed 9/30/09 with fast-track designation
•Analyst projections - FierceBiotech ($1B)

fingolimod (FTY720) by Novartis is a potential 1st in class sphingosine 1-phosphate (S1-P) receptor modulator

•Clinical differentiation- superiority was demonstrated in a trial comparing to interferon beta-1a (5/09). Also, 2-year data was reported in 9/09 (FREEDOMS and TRANSFORMS trial) - primary and secondary endpoints were met with statistical significance achieved compared to placebo. There is also a concern for increased incidence of serious infections.
•Pipeline status – NDA expected to be filed by end of 2009
•Analyst projections – Datamonitor ($1B by 2017 worldwide)

In our "What to Expect with Future Blockbusters" Webinar, we discussed pegylated interferon beta 1-a by Biogen

•Clinical differentiation- it’s a long-acting once-monthly interferon, which just entered phase III trials in 6/09
•Pipeline status – received fast-track designation by FDA, approval not likely before 2012
•Analyst projections – Datamonitor ($1B by 2017 worldwide), Thomson

Sources:
Datamonitor news article 1/09
FierceBiotech news article 7/09
Thomson: The Next Blockbuster Drugs
National MS Society: Who Gets MS?
Medco Drug Trend (2009)
Merck press release 4/09
Novartis press release 9/09
Seeking Alpha news article 1/09