Lpath Reports Results for ASONEP Phase 2a Study in Renal Cell Carcinoma
SAN DIEGO, March 24, 2015 /PRNewswire/ -- Lpath, Inc. (NASDAQ: LPTN) announced today that its Phase 2a single-agent, open-label study of ASONEP did not meet the primary endpoint of statistically significant progression-free survival in patients with advanced renal cell carcinoma (RCC). The study enrolled RCC patients who had previously failed treatment with at least one anti-vascular endothelial growth factor (VEGF) agent (e.g., Sutent) and no more than one mTOR inhibitor (e.g., Afinitor). This patient population is considered "last line," and the literature suggests cancer progression in this population within a one to two month time frame.
To successfully meet the primary endpoint of progression-free survival, at least 25 out of 39 patients needed to be progression-free at two months of treatment. The overall median time to progression was less than two months. In addition, seven patients were progression-free at least six months, with three patients progression-free for over 20 months. Six patients currently continue to receive weekly infusions of ASONEP.
ASONEP was well-tolerated by patients overall. Serious adverse events were reported in 11 patients with only one, constipation, deemed by the investigator to be related to ASONEP.
Dario Paggiarino, MD, chief development officer of Lpath, commented, "Our Phase 2a data suggest that in some patients with lower risk of disease progression based on prognostic factors, ASONEP may produce prolonged disease stability for up to 20 months. We have also recently analyzed the expression profile of the S1P pathway from a genetic database of thousands of cancer patient genomes and believe there could be a rationale for ASONEP in other cancer types where S1P pathway dysregulation suggests a stronger pharmacological rationale. At the conclusion of this RCC trial, we will take a strategic look at exploring with a partner those other opportunities where ASONEP may have the best chance of success."
The Phase 1 and Phase 2a clinical trials of ASONEP were partially funded by a $3.0 million grant from the National Cancer Institute under its Small Business Innovation Research Program.
ASONEP is the systemic formulation of a first-in-class humanized antibody that binds and neutralizes the bioactive lipid, sphingosine-1-phosphate (S1P). Lpath is also developing an ocular formulation, iSONEP, that was also discovered using Lpath's ImmuneY2 drug-discovery engine.
Lpath is also conducting a clinical trial referred to as the "Nexus" study, which is a four-arm, double-blind Phase 2 study where iSONEP is being evaluated for safety and efficacy in wet age-related macular degeneration subjects. The company expects to release data late in the second quarter of 2015. Lpath entered into an agreement with Pfizer in 2010 that provides Pfizer an exclusive option for a worldwide license to develop and commercialize iSONEP.
