Recently, Alnylam Pharmaceuticals Inc. (ALNY) received clearance from the regulatory authorities of Portugal, Sweden and the UK to commence an early-stage study of its candidate ALN-TTR01 for treating transthyretin-mediated amyloidosis (ATTR) including familial amyloidotic polyneuropathy (FAP) and familial amyloidotic cardiomyopathy (FAC). ATTR refers to a hereditary disease resulting from a mutation in the transthyretin (TTR) gene.
Following the approval, Alnylam will shortly start enrolling patients for the randomized, placebo-controlled, dose escalation early-stage study. The study, which will be conducted in Portugal, Sweden and the UK aims to enroll approximately 28 patients suffering from ATTR.
The study will primarily evaluate the safety and tolerability of a single dose of intravenous ALN-TTR01, a Ribo Nucleic Acid interference (RNAi) therapeutic. The enrolled patients will be divided into five groups, arranged in an ascending order of doses of the candidate, ranging from 0.01 – 0.4 mg/kg.
Furthermore, the secondary goals of the study include the characterization of plasma and urine pharmacokinetics of the candidate apart from assessing the pharmacodynamic activity based on measurements of circulating TTR plasma levels.
The market for ATTR (in its most serious form) is characterized by a huge unmet need. The life expectancy of ATTR patients with FAP ranges from 5 to 15 years from the onset of the symptom of the disease. Liver transplantation remains the sole treatment option in such a scenario. This further highlights the need for new therapies to treat such patients.
Consequently we believe that if Alnylam’s candidate manages to clear the various stages of development and the regulatory hurdles to hit the market then it would go a long way in meeting the unmet need.
Our Take & Recommendation
Even though Alnylam’s RNA technology is potentially revolutionary, we remain skeptical about its success in drug development application. A very limited number of drug candidates based on RNAi discovery have been tested in animals or humans. Furthermore, we are also concerned about the early stage of development. This justifies our short-term (Zacks #3 Rank (‘hold’)) and long-term Neutral stance on the stock.
The Zacks Rank #3 indicates that the stock is expected to perform in line with the overall U.S. equity market for the next 1-3 months. Our long-term Neutral stance on the company indicates that the stock is expected to replicate its short-term performance, but just over 6-12 months. Consequently, we advise the investors to retain the stock over the time-period.