) rose approximately 3.85% following the announcement of
encouraging data from a phase II study on drisapersen. Prosensa
is developing drisapersen for the treatment of patients suffering
from Duchenne muscular dystrophy (DMD).
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The placebo-controlled phase II study (DMD114876 or DEMAND V)
compared the use of drisapersen versus placebo in 51 boys above
five years of age and suffering from DMD. Data from the study
indicated that at a higher-dose of drisapersen (6 mg/kg once
weekly), patients experienced stabilization and improvement in
muscle function and physical activity during the 24-week
treatment period. Patients also maintained that improvement
during the 24-week follow-up phase. Additionally, a clinically
meaningful treatment difference of 5.2% (at week 24) and 4.8% (at
week 48) was observed between drisapersen and placebo in the 6
Minute Walking Distance (6MWD) test.
We remind investors that Prosensa had received a major setback,
with the stock price tumbling approximately 70% in Sep 2013, when
it had announced that a phase III study failed to meet the
primary endpoint as drisapersen did not show a statistically
significant improvement in the 6MWD test versus placebo. No
difference in the key secondary assessments of motor function was
However, initial findings from further analysis of the aggregate
drisapersen data suggests that use of
drisapersen earlier in DMD patients and longer treatment duration
leads to a delay in the progression of the disease.
Drisapersen enjoys orphan drug status in the EU and the U.S. In
Jun 2013, the U.S. Food and Drug Administration (FDA) granted
Breakthrough Therapy designation to drisapersen. Prosensa has
full rights to drisapersen after
) terminated its agreement for the candidate earlier this year.
Prosensa plans to engage in discussions with regulatory
authorities in the U.S. and EU regarding the future development
path of drisapersen in the second quarter.
Meanwhile, the company also announced its full year 2013
financial results. The company reported a loss of 51 cents per
share for 2013, wider than the year-ago loss of 37 cents per
share but narrower than the Zacks Consensus Estimate of a loss of
We are pleased with the encouraging data on drisapersen. However,
keeping in mind the disappointing phase III results, we remain
According to Prosensa, DMD is a fatal rare genetic disease
affecting 1 in 3,500 boys worldwide. It represents an underserved
market as currently there are no approved disease-modifying
therapies for DMD. We note that several other companies including
Sarepta Therapeutics, Inc.
) are developing therapies targeting the DMD market.
Prosensa, a biopharmaceutical company, carries a Zacks Rank #2
(Buy). Investors may also consider another biopharmaceutical
Alexion Pharmaceuticals, Inc.
), which sports a Zacks Rank #1 (Strong Buy).