Ebb & Flow
On Friday, investors savaged Isis (NASDAQ:ISIS) and dinged partner Genzyme (NASDAQ:GENZ) on news that NDA submissions for their mipomersen cholesterol-lowering drug would take longer than anticipated.
Isis fell $4.81 (29%) to $11.99, while Genzyme dipped $0.85 to $72.49. Isis, which has had a string of partnership deal announcements, has not been at this level since late August 2007.
The companies said FDA has indicated that data from a large-scale outcomes study will be required prior to receipt of full approval for subcutaneous mipomersen to treat high cholesterol in patients with high cardiovascular risk.
The companies, which had been planning to submit an application in 2010, did not provide a date for starting the outcomes study but hope the submission timeframe is not substantially later than 2011.
The partners also said an NDA seeking accelerated approval of mipomersen to treat homozygous familial hypercholesterolemia (hoFH) would be delayed until 2010 from 2009 after FDA requested that the application contain data from two ongoing preclinical carcinogenicity studies.
Earlier this year, Isis granted Genzyme exclusive rights to develop and commercialize the second-generation antisense inhibitor of Apo B-100, which has Orphan designation for hoFH in the U.S. (see BioCentury, Jan. 14).
In fact, final contracts are still being negotiated, with the deal expected to close this quarter.
Cowen's Eric Schmidt downgraded on the news, both because of the delay and the potential that the deal terms could change in Genzyme's favor (see "Analyst Picks & Changes," A21).
Genzyme hit another regulatory pothole earlier in the week, when it said FDA had concluded the company will have to submit a separate BLA to market Myozyme alglucosidase produced at the 2,000 liter bioreactor scale. The agency originally approved the Pompe's disease treatment produced in a 160 liter bioreactor.
The recombinant human acid alpha-glucosidase enzyme (rhGAA) produced in the larger bioreactor is approved for sale in the EU, Japan and Canada (see Cover Story).
On the week, Isis was down $4.90 (29%), while Genzyme shed $2.25.
Jerini's wild ride
After receiving a not approvable letter from FDA for an NDA for Firazyr icatibant to treat hereditary angioedema (HAE) on Thursday,