Intellikine, a leader in the development of small molecule drugs targeting the PI3K/mTOR pathway, and the PKD Foundation announced a collaboration to investigate novel, orally-available small molecule kinase inhibitors of the TORC1 and TORC2 complexes as a potential treatment for patients with Polycystic Kidney Disease (PKD).

As part of the PKD Foundation’s Accelerating Treatments to Patients (ATP) programme to translate research discoveries into new treatments for patients with PKD, the PKD Foundation is collaborating directly with biotech and pharmaceutical organizations to assist them in evaluating whether their drug candidates have potential as therapeutic agents for PKD.

“The ATP programme is an important new initiative, critical to our strategy to move promising treatments to patients faster,” said Jill Panetta, PhD, Chief Scientific Officer of the PKD Foundation. “Intellikine is our first collaboration under our Drug Repurposing Programme, and we are thrilled to work together to accelerate the development of their exciting TORC1/2 inhibitors as a potential treatment for patients with PKD.” Initially, the PKD Foundation will support Intellikine to evaluate the drug candidates using pre-clinical models of the disease.

“There is a strong rationale and encouraging pre-clinical data for selective TORC1/2 inhibitors as potential treatments for PKD,” said Troy Wilson, PhD, J D, president and CEO of Intellikine. “Through the ATP program, not only are we able to garner financial support, but we are able to engage the entire PKD community, from research and clinical experts in the field of PKD to empowered patients and their families affected by the disease. The ATP programme allows us to broaden our research and development activities in PKD with the hope of bringing new agents to patients more quickly.”

Autosomal Dominant PKD (ADPKD) represents the most frequent potentially lethal monogenic hereditary disease of mankind. There are estimated to be more than 1,000,000 patients with the disease in the US and Europe. The disease pathology is characterized by development and growth of cysts in the kidneys, which leads to progressive destruction of the normal renal parenchyma and massive enlargement of the kidneys.

Subsequently, the glomerular filtration rate decreases in an accelerated mode, and end-stage renal disease with the need for dialysis and/or transplantation ensues. Importantly, despite several decades of intense basic and clinical research, an effective treatment that alters the course of ADPKD has not been established.

The mTOR kinase represents an important target for drug development, both in oncology and other diseases such as PKD. Unlike rapamycin analogues, such as sirolimus and everolimus, which have had only limited success in treating patients with PKD, TORC1/2 inhibitors block both TORC1 and TORC2, thus more potently inhibiting mTOR kinase and may provide for greater efficacy in the treatment of PKD.

Polycystic Kidney Disease Foundation is the only non-profit organisation, worldwide, dedicated to fighting PKD through research, education, advocacy, support and awareness. The organization’s goal is to discover and deliver treatments and a cure for PKD.

Intellikine is a private, clinical-stage company focused on the discovery and development of innovative small molecule drugs targeting the PI3K/mTOR pathway for the treatment of human disease.