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Genzyme And Isis Pharmaceuticals’ Orphan Drug Kynamro’s Price, Antisense Technology, And Pipeline

January 30, 2013

Genzyme and Isis Pharmaceuticals’ orphan drug Kynamro (Mipomersen) is approved January 29, 2013, by the FDA for the treatment of Homozygous Familial Hypercholesterolemia (HoFH). Kynamro is Isis Pharmaceuticals first drug to make it to the market. What is in question is what the price of the drug will be. Kynamro’s competitor in the US is Aegerion Pharmaceuticals’ orphan drug Juxtapid (Lomitapide), approved in December 2012 by the FDA for HoFH. According to a January 30, 2013 Wall Street Journal article :

“ …. Kynamro, the cholesterol drug, will be priced lower than its rival Juxtapid. But Kynamro still will cost $176,000/patient/year, according to Sanofi’s Genzyme unit, which developed the drug with Isis Pharmaceuticals Inc. A year’s treatment of Juxtapid costs $235,000 to $295,000 depending on the stage of therapy ….. ”.

At the 31st Annual J.P. Morgan Healthcare Conference held in January 2013, in San Francisco, Stan Crooke, Isis Pharmaceuticals’ CEO, gave a presentation with information on the Isis Pharmaceuticals’ robust drug pipeline :

–  28 drugs in development in the pipeline

–   9 drugs in 2013 that “will encounter important Phase II or Phase III data points”

–   “ … 5 drugs that we think we can reach the marketplace in the next 5 years …”.

Isis Pharmaceuticals has a conference call and webcast to provide an update on the FDA approval of Kynamro, on January 30, 2013, at 9:30 AM ET. The following are a few points that are reviewed :

– The success of antisense technology

– Kynamro is launch ready with the Cornerstone Patient Support Program

–  Focused sales force and medical team on lipid specialists

–  Review of Isis Pharmaceuticals’ drug Pipeline :

–  Expect over the next 5 years, to have 5 approved drugs to bring to market

–  9 drugs in critical clinical data milestones in Phase II/III clinical trials within the next year or so.

A fascinating January 30, 2013 article titled, “After 24 Years of Struggle, Has Stan Crooke Finally Birthed An Age of Antisense”, that chronicles the more than 20 year struggle to produce a product using antisense technology.

A January 30, 2013 article titled, “Isis Pharmaceuticals: A Compelling Long-Term Story”,  reviews the strength of Isis Pharmaceuticals’ drug pipeline, the multiple drug development partnerships, and the “validated technology platform” (antisense technology platform).

Another January 30, 2013 article titled, “Isis Pharmaceuticals: Kynamro’s Approval Is An Antisense Home Run”, discusses the history and implications of Kynamro’s antisense technology. Isis Pharmaceuticals has been working on antisense technology since the company’s founding more than 20 years ago. Gilead Sciences gave up on antisense technology and sold most of the patents related to this type of technology to Isis Pharmaceuticals.

The RNAi Therapeutics Blog’s article identifies Kynamro as a milestone for antisense oligonucleotide technology. Kynamro is receiving the “ …. 1st US regulatory approval for a systemically delivered oligonucleotide-based drug addressing a target in the liver ……”. The article goes on to review the commercial prospects.

The 15th Annual TIDES (Oligonucleotide & Peptide Therapeutics From Research through Commercialization) Meeting, is to be held May 12 – 15, 2013 in Boston, Massachusetts. Mary P. McGowan, M.D., Senior Medical Director of Clinical Research at Genzyme, will give a Keynote Presentation titled, “Mipomersen: A Novel Second Generation Antisense Oligonucleotide for Treatment of the Rare Genetic Disorder, Homozygous Familial Hypercholesterolemia” at 8:45 AM, May 13, 2013. Also, Isis Pharmaceuticals has several speakers talking on the different aspects of antisense oligonucleotide technology.


Isis Pharmaceuticals Antisense Drug Discovery Platform

Isis Pharmaceuticals Drug Pipeline January 30, 2013 article titled, “Isis Pharmaceuticals: A Compelling Long-Term Story” January 30, 2013 article titled, “Isis Pharmaceuticals: Kynamro’s Approval Is An Antisense Home Run” January 9, 2013 article titled, “Isis Pharmaceuticals CEO Presents at 31st Annual J.P. Morgan Healthcare Conference (Transcript)”

The RNAi Therapeutics January 30, 2013 Blog Post titled, “First Antisense Oligo Approved for Gene Knockdown in Liver”

Wall Street Journal January 30, 2013 article titled, “Drug Makers See Profit Potential in Rare Diseases”

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

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