La Jolla Pharmaceutical has stopped a pivotal trial of LJPC-401 after an interim review. The company disclosed the setback on the same day it revealed its CEO and president George Tidmarsh has left “to pursue other interests.”
LJPC-401, a synthetic form of human iron metabolism regulator hepcidin, moved into a pivotal trial in transfusion-dependent beta thalassemia patients with myocardial iron overload almost two years ago. After enrolling half of the targeted 100 subjects, La Jolla performed an interim analysis that found patients on LJPC-401 were doing no better than their peers on placebo against the primary and key secondary endpoints.
The finding led La Jolla to stop the pivotal trial, killing off near-term hopes of bringing the drug to market in the beta thalassemia population. La Jolla is yet to dump the drug altogether, although it is reassessing whether to continue development.
That response reflects the availability of more encouraging data from a phase 2 study in patients with hereditary hemochromatosis. The trial met its primary endpoint of change in transferrin saturation, which increases in line with the body’s iron stores, and also hit key secondary endpoints, leading La Jolla to characterize results from across the failed and successful studies as “mixed.”
La Jolla is now assessing whether the mixed results justify further investment in LJPC-401. Ultimate responsibility for that decision will fall on a new leader. Tidmarsh, who spent almost eight years at La Jolla, has left the company. La Jolla disclosed the immediate departure of its CEO and president on the same day it shared news of the mixed data on LJPC-401.
Shares in La Jolla fell more than 50% following the one-two punch delivered by the statements.
La Jolla board members Kevin Tang and Craig Johnson will oversee the company’s management team until a new CEO is found. That CEO will inherit a company focused on growing sales of Giapreza, a vasoconstrictor that brought in $15.8 million over the first nine months of the year, and winning FDA approval for malaria drug LJPC-0118.
By Nick Paul Taylor
Source: Fierce Biotech
Pharma and medical imaging company MedTrace announced the appointment of Nicholas Borys, M.D., as Chief Medical Officer (CMO). Dr. Borys is a skilled and experienced healthcare executive with over 25 years experience in advancing portfolios of novel therapies across diverse indications in cardiovascular, cancer, and central nervous system disease.
Eli Lilly and Company has announced a significant expansion of its manufacturing presence in Ireland, with a $1bn investment earmarked for its Limerick site. The move is aimed at boosting the company’s production of biologic active ingredients, including those for its approved Alzheimer’s treatment.
Lonza recently announced that it has completed a planned expansion of the mid-scale microbial manufacturing facility at its Visp, Switzerland site. This multi-product facility was recently granted a GMP license, allowing cGMP manufacturing and release of biologics produced using microbial fermentation.
