La Jolla Pharmaceutical (LJPC) Misses Q3 EPS by 6c; Updates on GCS-100 Phase 2 in CKD

La Jolla Pharmaceutical (NASDAQ: LJPC) reported Q3 EPS of ($0.37), $0.06 worse than the analyst estimate of ($0.31).

The company also announced that detailed results from its Phase 2 study of GCS-100 in chronic kidney disease (CKD) patients are being presented at a poster session (Glomerular and Tubulointerstitial Disease: Clinical Trials and Outcomes, November 13, 10:00 a.m. - 12:00 p.m. Eastern Standard Time, Poster TH-PO460) at the American Society of Nephrology’s (ASN) Kidney Week Annual Meeting. GCS-100, which blocks the activity of the pro-fibrotic mediator galectin-3, has the potential to be a first-in-class disease-modifying agent for the treatment of diseases characterized by progressive tissue fibrosis, such as CKD.

This multicenter, randomized, blinded, placebo-controlled, Phase 2 study in 121 advanced CKD patients met its primary efficacy endpoint of a statistically significant improvement in kidney function. Specifically, GCS-100, at a dose of 1.5 mg/m2, led to a statistically significant (p=0.045) improvement in estimated glomerular filtration rate (eGFR) versus placebo after 8 weeks of dosing. This improvement, compared to placebo, was maintained 5 weeks following the completion of dosing (p=0.07). No statistically significant improvement in eGFR was observed in the 30 mg/m2 dose group. The lack of consistent response in the 30 mg/m2 group may be due to off-target drug effects, as this dose is 1,400-fold in excess, on a molar basis, versus known circulating galectin-3 levels. Off-target effects may include antagonizing other galectins like galectin-9, which has opposing biological effects to galectin-3.

GCS-100’s effect on eGFR was more pronounced (p=0.029) in the prospectively defined subset of patients with diabetic etiology. Analysis of this subset was predefined based on the observation that galectin-3 is elevated in the kidneys of diabetic CKD patients, and the level of galectin-3 correlates with proteinuria (a marker of kidney health) in these patients.

GCS-100 was well-tolerated. There were no serious adverse events, no Grade 3/4 adverse events and no early study discontinuations in the 1.5 mg/m2 group. There was no observed effect on blood pressure in any dose group.

“We are highly encouraged by these results,” stated Pablo E. Pergola, M.D., Ph.D., Director of the Clinical Advancement Center subsidiary of Renal Associates P.A. and Clinical Associate Professor of Medicine at the University of Texas Health Science Center at San Antonio. “The steady improvement in eGFR over time that was maintained five weeks following the last dose and the lack of a hemodynamic effect both lend support to GCS-100’s novel, anti-fibrotic mechanism. Chronic kidney disease remains a major medical problem, with tissue fibrosis at its core.”

An extension study is currently being conducted in which patients from the Phase 2 study were re-randomized to receive either 1.5 or 30 mg/m2 of GCS-100 (complete data available through week 16). Of the 93 patients enrolled in total, 33 patients had previously received placebo in the Phase 2 study before being treated with GCS-100 in the extension study. This group, which represents a set of patients receiving GCS-100 for the first time, was analyzed for efficacy. Consistent with the blinded Phase 2 results, the 1.5 mg/m2 group experienced a significant improvement in eGFR. This is the case when comparing these patients’ response to both: (1) the response in the parallel randomized group receiving 30 mg/m2 (p=0.04); and (2) the previous response to placebo in the blinded Phase 2 study for placebo-treated patients enrolled in the extension study (p=0.02).

“We want to thank all of the patients and investigators who contributed to making this study a success,” said George Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. “We look forward to advancing GCS-100 to the next phase of development in chronic kidney disease, and to bringing this potentially important therapy one step closer to the many patients suffering from this terrible disease.”

For earnings history and earnings-related data on La Jolla Pharmaceutical (LJPC) click here.

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