Genzyme, a Sanofi (SNY - Analyst Report) company, and partner Isis Pharmaceuticals Inc. recently announced that their new drug application (NDA) for pipeline candidate Kynamro has been accepted by the US Food and Drug Administration (FDA) for standard review.
The companies are looking to get Kynamro approved for the treatment of patients with homozygous familial hypercholesterolemia (HoFH). Genzyme had submitted the NDA in March 2012.
Kynamro is the lead pipeline candidate at Isis Pharma. Isis Pharma and Genzyme filed for European approval of Kynamro in July 2011 for use in patients with homozygous familial hypercholesterolemia (hoFH) and severe heterozygous FH (heFH). On its first quarter conference call, Isis Pharma had said that Genzyme is preparing for the EU launch of Kynamro later this year.
With the acceptance of the NDA filing, Isis Pharma stands to receive $25 million from Genzyme. US approval would trigger another milestone payment of $25 million from Genzyme. The $25 million milestone payment for the approval of Kynamro will be due in 2013 given the January 29, 2013 FDA action date, provided Kynamro is approved at that time.
Meanwhile, Isis Pharma and Genzyme are conducting a 12-month study (FOCUS FH - saFety and atherOgeniC lipoprotein redUction of mipomerSen in FH) with Kynamro. The study is being conducted to support the expansion of the initial label and support an alternative dosing regimen (three times a week). The study is being conducted under the FDA’s Special Protocol Assessment (SPA) program.
Neutral on Isis Pharma
We currently have a Neutral recommendation on Isis Pharma, which carries a Zacks #3 Rank (short-term Hold rating). We are positive on Isis’ agreements with GlaxoSmithKline (GSK - Analyst Report) and Biogen (BIIB - Analyst Report) which not only validate its antisense technology but also provide Isis Pharma with funds in the form of upfront, milestone and other payments. We expect investor focus to remain on the regulatory progress of Kynamro.