La Jolla whips up some love for iron overload drug with promising data snapshot
With revenue trickling in from its first product and an NDA in place for a second, La Jolla Pharmaceutical is ready to shine a bright light on the third candidate coming through its pipeline.
An interim analysis of a Phase II study suggests that LJPC-401 could significantly lower the excess iron levels in patients with hereditary hemochromatosis. The drug — a subcutaneous injection of synthetic human hepcidin — induced a mean reduction in TSAT from baseline of 42%, versus 6% on placebo (p<0.0001), marking a clear score on the primary endpoint.
Jeff Vacirca, an investigator and chief of clinical research at New York Cancer & Blood Specialists, explained his enthusiasm this way:
There have been no new treatment modalities introduced for patients with hereditary hemochromatosis in more than a decade. In light of the negative impact that repeated phlebotomy procedures have on patient quality-of-life, patients would welcome a pharmacologic treatment that they can self-administer and that addresses the underlying pathophysiology of the disease.
LJPC-401 also met the key secondary endpoint of lowering the frequency of phlebotomy procedures with patients averaging 0.06 phlebotomies per month (3 procedures between 2 patients) compared to placebo-treated patients, who had 0.41 (9 patients collectively received 24). The p-value came in at 0.003.
Injection site reactions, which only happened to 6% of the placebo group, occurred in 79% of patients treated with the drug — though La Jolla would emphasize they were mild.
LJPC-401 was a key part of the San Diego biotech’s pitch for a $100 million public offering in 2018 alongside LJPC-0118, the severe malaria treatment that recently received a breakthrough therapy designation. Topline results are expected later in the year.
Image: La Jolla via Linkedin