Isis Pharmaceuticals, Inc. (ISIS) recently announced the initiation of a phase I study on an antisense candidate targeting apolipoprotein(a), ISIS-APOA, for treating patients suffering from atherosclerosis .
We note that apolipoprotein(a) helps in the formation of plaque in arteries through its attachment to an LDL-C particle in a complex known as lipoprotein(a) or Lp(a). Elevated levels of Lp(a) are associated with an increased risk of cardiovascular events such as atherosclerosis, coronary heart disease, heart attack and stroke.
ISIS-APOA aims to bring down Lp(a) levels by inhibiting apolipoprotein(a) production.
The blind, placebo-controlled, dose-escalation phase I study will enroll approximately 56 healthy volunteers and assess safety, tolerability and pharmacokinetics of ISIS-APOA (both single and multiple doses).
Earlier this month, Isis Pharma published encouraging data in the journal Circulation Research related to antisense technology. It was found that antisense targeting of apolipoprotein C-III (apoC-III) showed significant decline in apoC-III and triglycerides. Both apoC-III and triglycerides, are risk factors associated with cardiovascular diseases.
We are impressed by the development of antisense technology and believe that the number of potential therapeutic applications is enormous. Antisense drugs may have significant potential to treat a number of diseases where small molecules and biologic compounds have failed.
Although still in its early stage, antisense technology with mechanisms such as small interfering RNA (siRNA), RNA interference (RNAi), alternate splicing, and microRNA (mRNA) have the potential to change how diseases are treated.
We note that Isis Pharma’s lead product, Kynamro (mipomersen sodium), received FDA approval in Jan 2013 for the treatment of familial hypercholesterolemia (:FH). Isis Pharma’s partner Sanofi (SNY) is marketing the product in the US. Sanofi is also looking to get the drug approved in the rest of the world including Europe.
Isis Pharma currently carries a Zacks Rank #3 (Hold). Meanwhile, stocks such as Athersys, Inc. (ATHX) and Osiris Therapeutics, Inc. (OSIR) carry a Zacks Rank #1 (Strong Buy) and are worth considering.
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