Intellikine and the PKD Foundation Collaborate to Advance Next-Generation Therapies for Polycystic Kidney Disease

Collaboration will support pre-clinical research to evaluate novel small molecule TORC1/2 inhibitors as possible treatments for PKD

LA JOLLA, Calif.--(BUSINESS WIRE)-- Intellikine, a leader in the development of small molecule drugs targeting the PI3K/mTOR pathway, and the PKD Foundation today 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) program 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 program is an important new initiative, critical to our strategy to move promising treatments to patients faster,” said Jill Panetta, Ph.D., Chief Scientific Officer of the PKD Foundation. “Intellikine is our first collaboration under our Drug Repurposing Program, 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, Ph.D., 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 program 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 U.S. 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 analogs, 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.

About the Polycystic Kidney Disease Foundation

Founded in 1982 in Kansas City, Missouri, the PKD Foundation is the only nonprofit, 501(c)(3) organization, 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. The Accelerating Treatments to Patients program, or ATP, was initiated in 2010 to help fulfill the PKD Foundation’s mission to find treatments for polycystic kidney disease. For more information, please visit the PKD Foundation’s website at

About Intellikine

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. Intellikine’s most advanced program, INK128, a selective TORC1/2 inhibitor for the treatment of cancer, is currently in a Phase 1 clinical trial in patients with advanced solid tumors as well as a second Phase 1 trial in patients with multiple myeloma and Waldenstrom’s macroglobulinemia. Intellikine is also advancing a PI3Kalpha-isoform-selective inhibitor, INK1117, which is currently being prepared for human clinical trials in cancer. Intellikine owns all rights to both INK128 and INK1117. In addition, Intellikine recently entered into a $489 million collaborative partnership with Infinity Pharmaceuticals July 2010 to develop oral therapies targeting PIK3Kδ and PI3Kγ for indications including immune-mediated inflammatory diseases and cancer. Among these inhibitors is IPI145 (formerly INK1197), an orally available dual-active PI3Kδ/γ inhibitor for which clinical development is expected to commence in 2011. For more information, please visit the company’s website at


For Intellikine:
Felicia Vonella, 917-826-3427
[email protected]
For PKD Foundation:
Dave Switzer, 816-268-8481
[email protected]

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