Biotech Values

[mcbio]

OMER (prior P2 aHUS data) -

One issue for me is that the OMER drug is earlier in the complement pathway than is Soliris - see my comment here:

www.siliconinvestor.com/readmsg.aspx?msgid=29434460

An aHUS expert might well have an answer to this theoretical concern of mine based on the data released today.

Peter, I realize now that your prior post was made the same day of this data release. Sounds like the efficacy data for OMS721 in this release still does not alleviate some of your concerns on the efficacy front about the target (MASP2) being a bit more upstream in the complement pathway: http://investor.omeros.com/phoenix.zhtml?c=219263&p=irol-newsArticle_Print&ID=2080177 . Is that correct?

In particular:

"As in Alexion's clinical trials supporting both U.S. and European regulatory approval of Soliris® for the treatment of aHUS, this Phase 2 clinical trial, given the life-threatening nature of the disease, has no placebo arm. Soliris trials used change from baseline in platelet count as a primary endpoint to obtain approval for the treatment of aHUS. Similarly, the pre-specified primary endpoint in this Phase 2 trial is change from baseline in platelet count. In this trial, platelet counts in all three aHUS patients in the mid- and high-dose cohorts (two in the mid-dose and one in the high-dose cohort) were normal after the treatment period, with a statistically significant mean increase from baseline of approximately 68,000 platelets/mL (p = 0.0055).

In the mid-dose cohort, the two patients with plasma therapy-resistant aHUS demonstrated:

47% increase in mean platelet count, resulting in both patients having counts in the normal range
86% decrease in mean schistocyte count, with schistocytes disappearing in one patient
71% increase in mean haptoglobin with both patients reaching the normal range during treatment, one slipping slightly below normal at one week following the last dose
5% decrease in the mean levels of LDH, with levels in both patients remaining slightly elevated above normal range

The mid-dose-cohort patient with TTP required repeated plasma infusion therapy prior to entering the study. Laboratory parameters did not show consistent improvement, but the patient did not require plasma therapy during treatment with OMS721 and, to date, has not required it since completing treatment.

The first patient in the high-dose cohort – a plasma therapy-resistant aHUS patient with additional complicating disorders including hepatitis C, cryoglobulinemia and lymphoma – has also completed treatment with OMS721. Prior to OMS721 treatment, the patient required repeated dialysis. Throughout treatment and following completion of the OMS721 course, the patient to date has remained off dialysis. Hematological and renal parameters showed:

63% improvement in platelet count, returning to normal levels
100% decrease in schistocytes
Haptoglobin increased from an undetectable level and normalized
43% decrease in LDH, resulting in a level just slightly above normal
24% reduction in creatinine level

As expected, patients with aHUS in the mid- and high-dose cohorts demonstrated more consistent and robust improvement in efficacy measures than patients in the low-dose cohort. As in the low-dose cohort, the drug was well tolerated by all patients in the mid- and high-dose cohorts throughout the treatment period."