Brief Overview

Lpath, Inc. is the category leader in the discovery and development of monoclonal antibodies that target bioactive lipids. The study of lipids is an emerging field of research that has already identified numerous bioactive lipids that contribute to disease, with the promise of many more as funding in the area continues to accelerate. Once a disease-causing lipid is identified, however, the challenge becomes one of generating a compound that neutralizes the relevant bioactive lipid. With its ImmuneY2™ platform technology and robust patent estate, Lpath is uniquely positioned to meet such challenges and exploit these opportunities.

By leveraging this ImmuneY2 technology, Lpath has generated monoclonal antibody drug candidates against several bioactive lipids, including the well-validated targets, S1P and LPA.

Lpath's lead product candidate, iSONEP™ (an anti-S1P antibody), is currently in a Phase 2 clinical trial (Nexus) to assess the efficacy of iSONEP, given alone or in combination with Lucentis®, Avastin®, or Eylea® in subjects with wet AMD, an ocular disease that often leads to blindness.

In the phase 1 trial in wet AMD patients, iSONEP met its primary endpoint of being well tolerated in all 15 patients at dose-levels ranging from 0.2 mg. to 1.8 mg. per intravitreal injection (three patients per dose level). No drug-related serious adverse events were reported in any of the patients. iSONEP also succeeded in meeting a key secondary endpoint in that positive biological effects (including lesion regression, reduction of retinal thickness, and resolution of pigment epithelial detachment) were observed in an encouraging number of patients. Most of these positive effects appear to be largely independent of the effects seen when wet-AMD patients undergo treatment with Lucentis® or with off-label use of Avastin®, the predominant market leaders. iSONEP™ also demonstrated excellent results in various preclinical AMD models and first-in-class results in a preclinical model of diabetic retinopathy. The drug candidate exhibits anti-fibrotic and anti-inflammatory properties, which might provide comparative advantages in a variety of ocular diseases. In December 2010, Lpath announced a collaboration agreement with Pfizer Inc. for the further clinical development of iSONEP. If successful, this collaboration could result in payments up to $517.5 million plus tiered double digit royalties to Lpath (Download PDF).

Lpath's second product candidate, ASONEPTM, is a monoclonal antibody against sphingosine-1-phosphate (S1P) that has completed a phase 1 clinical trial in cancer patients, where it was deemed well tolerated at dose levels up to 24 mg/kg. We are currently in a phase 2 clinical trial to assess efficacy (and safety) in renal cell carcinoma patients. Recent data and scientific literature also suggest that targeting S1P might also have applications in other diseases like multiple sclerosis, colitis and other inflammatory disorders.

Lpath's third product candidate, LpathomabTM is a monoclonal antibody against lysophosphatidic acid (LPA), a key bioactive lipid that has been recognized to be involved in disorders of the central nervous system (including pain, traumatic brain injury, neurodegeneration), fibrosis, ocular disease and cancer. Lpathomab has demonstrated strong preclinical activity in several animal models of these diseases, including neuropathic pain, diabetic neuropathy, traumatic brain injury and spinal cord injury, and is currently in IND-enabling development.

Lpath's unique ability to generate antibodies against bioactive lipids is based on its proprietary ImmuneY2TMdrug-discovery engine. The company is currently applying the ImmuneY2™ technology to other important bioactive lipid targets, many of which are involved in important disease processes (e.g. inflammation, cancer, pain, asthma, sepsis and others) thereby adding to our pipeline of novel antibody-based drug candidates.

Lpath has a broad and deep intellectual-property position in the lysolipid signaling area, with over 50 issued or pending patents in the U.S., with corresponding international applications. Most of these patents were developed in-house using our proprietary technologies and expertise.