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New Published Data Demonstrate Endocyte's Targeted Small Molecule Drug Conjugate Approach Shows Promising Results in Polycystic Kidney Disease Model
GlobeNewswire
2012-08-03

   Study Evaluating Folate-Conjugated Rapamycin Published Online
      in Journal of the American Society of Nephrology (JASN)

WEST LAFAYETTE, Ind., Aug. 3, 2012 (GLOBE NEWSWIRE) -- Endocyte, Inc.
(Nasdaq:ECYT), a biopharmaceutical company developing targeted small
molecule drug conjugates (SMDCs) and companion imaging diagnostics for
personalized therapy, today announced that Endocyte's approach of
utilizing a SMDC targeted to the folate receptor (FR) may provide a
promising treatment for patients with autosomal-dominant polycystic
kidney disease (ADPKD). In a mouse model of polycystic kidney disease
(PKD), treatment with a folate-conjugated form of rapamycin, FC-rapa,
(EC0371) strongly attenuated proliferation and growth of renal cysts
and preserved renal function. The results were published online ahead
of print in the Journal of the American Society of Nephrology and will
be available in print in the October issue.

Endocyte chemically synthesized EC0371 by linking the folic acid
targeting ligand with the rapamycin drug payload. Rapamycin, a potent
inhibitor of mTOR, is a promising drug for PKD therapy. The mTOR
pathway is deregulated in ADPKD, and its inhibition reverses renal
cystogenesis in animal models. However, in previous clinical trials
treatment was limited by the amount of the untargeted rapamycin that
could be safely administered. In collaboration with Thomas Weimbs,
Ph.D., associate professor in the department of molecular, cellular,
and developmental biology, at the University of California, Santa
Barbara, at Santa Barbara, Calif., and corresponding author of the
publication, the membranes of both human and mouse PKD cysts were found
to express the FR, which is the target of the EC0371's high affinity
ligand. Results of this study showed that EC0371 inhibits the mTOR
pathway activity in a dose- and FR-dependent manner.

"Currently, there is no pharmaceutical treatment available for PKD
patients, and patients with end stage renal failure get lifelong
dialysis or receive a kidney transplant. Being a PKD patient myself, I
have experienced the unmet medical need first hand," said Christopher
Leamon, Ph.D., vice president of research and development for Endocyte
and co-author of the publication. "I happened to read an article
published by Dr. Weimbs where he demonstrated that rapamycin was highly
effective in PKD animal models. Suspecting that cystic membranes in PKD
tissue might express the FR, I contacted him to discuss the potential
for targeting potent drugs exactly where they were needed to stop
cystic growth. Our results with EC0371 are very exciting, demonstrating
that we may be able to reduce the side effects observed in the clinical
trials with untargeted rapamycin by delivering the drug directly to the
cystic cells. What we have observed here also reinforces Endocyte's
concept of targeted drug delivery."

Over 600,000 people in the United States and 12 million worldwide are
affected by the inherited kidney disease known as autosomal-dominant
polycystic kidney disease (ADPKD, or PKD). The disease is characterized
by the proliferation of thousands of cysts that eventually debilitate
the kidneys, causing kidney failure in half of all patients by the time
they reach age 50. PKD is one of the leading causes of renal failure in
the United States.

About Endocyte

Endocyte is a biopharmaceutical company developing targeted therapies
for the treatment of cancer and inflammatory diseases. Endocyte uses
its proprietary technology to create novel SMDCs and companion imaging
diagnostics for personalized targeted therapies. The company's SMDCs
actively target receptors that are over-expressed on diseased cells,
relative to healthy cells. This targeted approach is designed to enable
the treatment of patients with highly active drugs at greater doses,
delivered more frequently, and over longer periods of time than would
be possible with the untargeted drug alone. The companion imaging
diagnostics are designed to identify patients whose disease
over-expresses the target of the therapy and who are therefore more
likely to benefit from treatment.

For additional information, please visit Endocyte's website at
www.endocyte.com.

About EC0371 (FC-rapa)

EC0371 is a folate-targeted, water soluble form of rapamycin that
selectively inhibits mTOR activity in polycystic kidneys to reduce
abnormal growth.


CONTACT: Stephanie Ascher, Stern Investor Relations, Inc.
         (212) 362-1200, stephanie@sternir.com
         
         Martina Schwarzkopf, Ph.D., Russo Partners
         (212) 845-4292, martina.schwarzkopf@russopartnersllc.com
         
         Tony Russo, Ph.D., Russo Partners
         (212) 845-4251, tony.russo@russopartnersllc.com


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