Alnylam Pharmaceuticals Inc. (ALNY), the developer of a
treatment for a rare genetic disorder, rose the most ever after the drug showed
promise in an early clinical trial.
Alnylam Pharmaceuticals Inc announced the achievement of
positive clinical results from its Phase I trial with ALN-TTR02, an RNAi
therapeutic targeting the transthyretin (TTR) gene for the treatment of
TTR-mediated amyloidosis (ATTR).
My analysis: As the pharmacogenomics and personalized medicine
are coming in the forefront the companies like Alnylam Pharma (ALNY) are benefiting
a lot from it. Due to smaller market capitalization of these companies, they
tend to rise exponentially on positive press release.
I feel this rally will not last too long before investors
realize that this is just the Phase I trial data which are mainly toxicity
data. This data may get presented at various investor conferences but this drug
still has lots of clinical inspection to go thru.
This company is certainly not a M&A target due to its
specific focused pipeline. It will be intersteing to see in coming months how this
company pans out.
TTR medicated
amyloidosis
The disease is caused by a mutation in the gene responsible for producing
the transthyretin protein. In people with the genetic defect, transthyretin
made by the liver breaks apart, forms into clumps, and damages the nerves and
heart. Currently a liver transplant is the only available treatment.
Alnylam’s drug, called ALN-TTR02, stops production of transthyretin. The
company is focusing on patients with TTR-FAP, or transthyretin familial amyloid
polyneuropathy. The company estimates that as many as 10,000 patients have the
disease. In TTR-FAP, the protein attacks the body’s longest nerves first, often
killing them within a decade as the body shuts down.
Results from this study show that administration of ALN-TTR02 leads to robust knockdown of
serum TTR protein levels of up to 94%; the overall results were highly
significant (p<0.00001 by ANOVA). Suppression of TTR, the
disease-causing protein in ATTR, was found to be rapid, dose dependent,
durable, and specific after just a single dose.
Alnylam recently
reported that it has initiated a Phase
II study of ALN-TTR02 in patients with ATTR and has guided that its goal is to
start a pivotal trial in 2013.
The primary objective
of the study was to evaluate the safety and tolerability of a single dose of
ALN-TTR02, with subjects being enrolled into five sequential cohorts of
increasing doses ranging from 0.01 to 0.50 mg/kg. In addition, pharmacodynamic
activity was evaluated with serial measurements of serum TTR protein levels
through at least day 56.
Preliminary data from this study showed that a single dose
of ALN-TTR02 resulted in rapid, dose-dependent, durable, and specific knockdown
of serum TTR levels. Even at doses as low as 0.15 mg/kg, substantial serum TTR
suppression was achieved, with a mean 81.9% knockdown at nadir.
Sources: Reuters and Bloomberg
No comments:
Post a Comment