An American biopharmaceutical company, La Jolla Pharmaceuticals (NASDAQ: LJPC), dedicated to the development of innovative novel therapies for patients suffering from severe diseases, announced that its CEO has departed from the company.
George Tidmarsh, Ph.D., MD, has decided to pursue other interests in the meantime. A decision from the CEO that may have come from La Jolla’s poor performance in its clinical trial for patients with beta-thalassemia.
La Jolla’s Decision For Beta Thalassemia:
Bad news came to La Jolla’s clinical trial, LJ401-BT01, they will no longer be recruiting patients because the study did not benefit patients with beta Thalassemia. The study was able to recruit 60 patients to test the efficacy of their clinical trial until it was abruptly stopped because of efficacy concerns with patients.
The company did disclose the positive results for another clinical trial that was testing LJ401 on patients with Hemochromatosis. The results show that the drug design for this clinical trial was a randomized, placebo-controlled, and double-blind.
As for LJ401-HH01, it met safety and efficacy endpoints after patients underwent 16 weeks of treatment with the synthetic hepcidin. The primary efficacy endpoint of the study was found to be statistically significant, a mean reduction in Transferrin Saturation of 33% compared to the mean reduction of 3% (p<0.0001) for patients taking the placebo. The secondary endpoint of the study testing the number of times that a patient visited the phlebotomist was also statistically significant. Patients taking LJPC-401 only had 0.10 phlebotomies per month than patients taking the placebo who had an average of 0.50 phlebotomies per month (p<0.0001).
La Jolla’s Synthetic Hormone:
La Jolla Pharmaceuticals investigational drug LJPC-401 is a synthetic version of endogenous peptide hepcidin, a hormone that can be found in a healthy person. The hormone acts as a regulator of excess iron that may be present from underlying diseases like Hemochromatosis. By regulating any excess amount of iron, this helps prevent any damage to the liver or Heart of a patient because of too much iron.
The testing of LJ401 may have surprised many because of their long-time CEO left abrupt when reviewing the interim results showing benefits for Hematochromatisis, but not beta-thalassemia. Although it did not meet endpoints for patients with beta-thalassemia, there is still some hope for the company. In theory, their investigational drug, LJPC-401, would have worked if it was designed to mimic hepcidin.
It seems that La Jolla pharmaceuticals will go back to the drawing boards to determine why their clinical trials had these mixed results. Their concern, for now, will be investing all their energy to expand the sales of their already approved drug Giapreza. A drug that is used to increase the blood flow of patients who have Sepsis.