Dermal scarring regularly occurs after the more than 42 million surgeries each year in the United States. But despite the wide scope of the problem, there is no FDA-approved prescription medication specifically labeled for scar prevention or reduction. The result is a wide-open market that could be worth as much as $4 billion, with potential third-party reimbursements if the treatments are adopted as a “standard of care” in the future.
Several companies are attempting to capitalize on this niche, taking a variety of different approaches to reduce dermal scarring. In this article, we’ll take a look at Alnylam Pharmaceutical Inc.’s (NASDAQ:ALNY) ALN-TTR, Pfizer’s (NYSE:PFE) EXC-001, and RXi Pharmaceuticals Corporation (OTCQX:RXII) RXI-109, and which of these compounds may represent the best bet for biotech investors over the coming quarters.
Alnylam Targets TTR-Mediated Amyloidosis
Alnylam Pharmaceuticals’ ALN-TTR02 gets compared to Pfizer’s EXC-001 and RXi Pharmaceuticals’ RXI-109 regularly, but there are a number of major differences between the compounds. Most significantly, ALN-TTR02 is targeting a mutation on the TTR gene that’s expressed predominantly in the liver and targets complete suppression of the gene expression, while Pfizer and RXi Pharmaceuticals are targeting a modest inhibition of genes involved with scar formation.
After demonstrating safety and tolerability in its March 2012 Phase I clinical trials, the company’s June 2013 Phase II clinical trials demonstrated rapid, dose-dependent, and durable knockdown of serum TTR protein levels of up to 93 percent. Evidence from other trials suggests that just a 50 percent reduction of the disease-causing protein can result in disease improvement or stabilization, which suggests that these Phase II results have more than sufficient efficacy.
Alnylam’s stock has reacted appropriately, jumping more than 83 percent over the past three months. Looking ahead, the company plans to initiate a Phase III pivotal trial of ALN-TTR02 in ATTR patients with FAP by the end of 2013, the results of which could be a significant catalyst.