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Rare Diseases: Clinical Trial Developments And Stock Price

September 23, 2013

Biotechnology stock movements are affected at various phases of the US FDA’s drug approval process. Big stock moves, either up or down, are associated with several catalysts :

•   Releases of trial data
•   Briefing documents prior to Advisory Committee meetings (AdComs)
•   AdComs themselves
•   Prescription Drug User Fee Act (PDUFA) dates.

This Blog Post discusses and reviews recent clinical trial developments in the rare disease space and the impact on the company’s stock.

I – Spinal Muscular Atrophy

Isis Pharmaceuticals announces on September 19, 2013, that follow-up preliminary data from a single dose, open-label Phase I study of ISIS-SMNRx, in children with the rare genetic disease, Spinal Muscular Atrophy (SMA), shows that most of the children receiving the two highest doses of the drug continue to show improvements in muscle function tests up to 14 months after a single injection of the drug.

ISIS-SMNRx receives FDA Orphan Drug Designation (ODD) in April 2011 and Fast Track designation for the treatment of patients with SMA. ISIS-SMNRx is an antisense drug that “counteracts a mutation that leads to loss of spinal motor neurons. The mutation causes the loss of a protein that protects the neurons. ISIS-SMNRx causes production of that protein.” Isis Pharmaceuticals is currently collaborating with Biogen Idec to develop and commercialize ISIS-SMNRx to treat all types of SMA.

According to RTT online news, Isis Pharmaceuticals’ stock increased on September 19th finishing with a gain of 4.47 at $36.27, setting a new high for the year.

II – Duchenne Muscular Dystrophy

One of the last catalysts before a final FDA decision, comes from a drug’s Phase III clinical trial data. Positive trial results can result in shares popping. There is also the reverse situation of failed clinical trial results causing shares to sink. Thus, late Phase clinical trials is one of the most critical steps of the orphan drug development life cycle. This is the critical time where demands for data on the drug’s safety and efficacy, comparisons to competitors, and other factors are important.

The most publicized example of the impact of Phase III clinical data on a company’s stock is the recent announcement by Prosensa/GlaxoSmithKline on September 20, 2013, that orphan drug Drisapersen didn’t meet the primary endpoint in patients with Duchenne Muscular Dystrophy (DMD). The primary endpoint is the statistically significant improvement in the 6 Minute Walking Distance (6MWD) test compared to placebo. Just 3 months ago, Drisapersen receives the coveted FDA Breakthrough Therapy Designation.

According to Reuters online, the failure of the Phase III study, results in :

•   Prosensa shares slumping 70%
•   Eliminating of approximately $600 million off the market value of the company, which had just completed an IPO in June 2013.

Please Note: “Dollar Symbol Gold” by Rugby471 (Own work) [GFDL] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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