La Jolla Pharmaceutical (LJPC) Announces Significant Data from LJPC-401 Phase 1 in Iron Overload

September 7, 2016 4:17 PM EDT
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La Jolla Pharmaceutical Company (Nasdaq: LJPC) reported positive results from its Phase 1 study of LJPC-401 in patients at risk of iron overload.

The Phase 1 study is a multi-center, open-label, dose-escalation study evaluating the safety, tolerability, and effect on serum iron of a single dose of LJPC-401 in patients at risk of iron overload due to conditions such as hereditary hemochromatosis (HH), thalassemia, and sickle cell disease (SCD). Fifteen patients were dosed at escalating dose levels ranging from 1 mg to 20 mg.

LJPC-401 was well tolerated, and there were no dose-limiting toxicities observed. Injection-site reactions were the most commonly reported adverse event. These were all mild or moderate in severity, self-limiting, and fully resolved. There were no significant changes in serum chemistries or hematology other than serum iron parameters.

A dose-dependent, statistically significant reduction in serum iron was observed (p=0.008 for dose response; not adjusted for multiple comparisons). At the 20 mg dose level, LJPC-401 reduced serum iron by an average of 58.1% from baseline to hour 8 (p=0.001; not adjusted for potential regression to the mean effect), and serum iron had not returned to baseline through day 7 (21.2% reduction from baseline to the end of day 7).

Dose Group

Mean Percent Change in Serum Iron

from Baseline to Hour 8

1 mg (n=3)-14.2% (p=0.149)
5 mg (n=3)-26.7% (p=0.304)
10 mg (n=3)-45.5% (p=0.054)
20 mg (n=6)-58.1% (p=0.001)
P-values not adjusted for potential regression to the mean effect

“The results from this study demonstrate a clear, dose-dependent effect of LJPC-401 on serum iron, a clinically important measure,” said George F. Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We look forward to continuing the development of LJPC-401, which we believe can potentially help patients suffering from the effects of iron overload by restoring physiologically relevant levels of hepcidin, the body’s natural regulator of iron absorption and distribution.”

Conference Call at 9:00 AM Eastern Time on Thursday, September 8, 2016

La Jolla will host a conference call and webcast on Thursday, September 8, 2016 at 9:00 AM Eastern Time (6:00 AM Pacific Time). The conference call can be accessed by dialing 877-359-9508 for domestic callers and 224-357-2393 for international callers. Please provide the operator with the passcode 77161203 to join the conference call or click here for the webcast. A slide presentation accompanying today’s press release and the conference call may also be found on La Jolla’s website at under the investor relations section. An archive of the conference call and webcast will be available on La Jolla’s website for 30 days following the call.

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