Proteostasis Therapeutics Secures a $45 Million Series A Financing to Develop Proteostasis...

Mon Aug 25, 2008 8:03am EDT

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Proteostasis Therapeutics Secures a $45 Million Series A Financing to Develop Proteostasis Regulator(TM) Drugs

CAMBRIDGE, Mass.--(Business Wire)--
Proteostasis Therapeutics, Inc. (PTI) today announced that it has
secured $45 million in a Series A financing to develop Proteostasis
Regulator(TM) drugs as novel therapies to treat multiple genetic and
degenerative disorders associated with protein homeostasis
deficiencies. Investors included HealthCare Ventures, Fidelity
Biosciences, New Enterprise Associates, Novartis Option Fund and
Genzyme Ventures.

   "Proteostasis Therapeutics brings together innovative discoveries,
leading scientists and a foundation of intellectual property related
to the Proteostasis Network(TM) and Proteostasis Regulators arising
from Northwestern University, the Salk Institute and the Scripps
Research Institute," commented David D. Pendergast, Ph.D., CEO of PTI.
"Proteostasis Therapeutics is uniquely positioned to commercialize
this fundamentally new way of thinking about therapeutic intervention
for a broad range of diseases."

   "The Company has established a map of the Proteostasis Network,
leading to breakthroughs in understanding how stress on the network,
including aging, environmental and cellular stresses, can lead to the
onset of multiple serious diseases," stated Andrew Dillin, Ph.D.,
co-founder of PTI. "With this new knowledge, PTI has identified unique
targets as well as small molecules that offer the promise of restoring
the balance of the network while preventing the toxicity of damaged
proteins associated with disease," continued Richard Morimoto, Ph.D.,
co-founder of PTI.

   "Proteostasis Therapeutics has discovered a new frontier in
medicine with the identification of several classes of small molecule
Proteostasis Regulators that can precisely rebalance the capacity of
the Proteostasis Network," commented Jeffery W. Kelly, Ph.D.,
co-founder of PTI. "These cell-permeable small molecules exhibit the
capacity to ameliorate multiple genetic, degenerative and metabolic
diseases, spanning nearly all the classical therapeutic areas."

   "Our initial funding of PTI over a year ago provided the basis for
this emerging area of science to mature so that it now offers the
promise of new drug candidates entering the clinic in the next 3-5
years," said Christopher Mirabelli, Ph.D., Managing Director of
HealthCare Ventures and Chairman of the Board of PTI. "The significant
Series A round is designed to support PTI's strategy to advance
proprietary compounds into the clinic, as well as to continue to
develop the unique technology platform that will serve as the basis
for strategic partnerships."

   Joining Drs. Pendergast and Mirabelli on the Board of Directors of
Proteostasis Therapeutics and representing the new investors are
Stephen Knight, M.D., Managing Partner, Fidelity Biosciences; James
Barrett, Ph.D., General Partner at New Enterprise Associates; Lauren
Silverman, Ph.D., Managing Director, Novartis Option Fund; and Alan
Walts, Ph.D., Managing Director, Genzyme Ventures.

   About the Founding Team

   David D. Pendergast, Ph.D.

   Dr. Pendergast was formerly the President of Human Genetics
Therapies at Shire Pharmaceuticals plc, and has more than 30 years of
experience in pharmaceutical development and corporate operations. In
his position at Shire, Dr. Pendergast was responsible for the
discovery, development, manufacture and commercialization of protein
therapeutics. Prior to joining Shire, Dr. Pendergast served as Chief
Executive Officer of Transkaryotic Therapies, Inc. (acquired by Shire
in 2005), where he managed the company during the acquisition, and
Chief Operating Officer, where he oversaw operational functions
ranging from discovery to commercialization of protein therapies. Dr.
Pendergast was also previously Vice President of Product Development
and Quality at Biogen Inc. (now Biogen Idec) and held senior positions
at Fisons Ltd. Pharmaceutical Division and at The Upjohn Company (now
part of Pfizer). Dr. Pendergast is also Chairman of Altus

   Andrew Dillin, Ph.D., Founder

   Andrew Dillin is currently Associate Professor and Pioneer
Developmental Chair in the Molecular and Cell Biology Laboratory at
the Salk Institute, where he is focused on researching the process of
aging as well as the etiology of neurodegenerative diseases. Dr.
Dillin recently identified a key regulatory pathway essential for the
response to dietary restriction mediated longevity, a key pathway for
proteostasis regulation. He received his Ph.D. degree in molecular and
cell biology at the University of California at Berkeley, and carried
out postdoctoral studies at the University of California in San
Francisco defining the genetic pathways required for successful aging.

   Jeffery W. Kelly, Ph.D., Founder

   Jeffery W. Kelly is the Lita Annenberg Hazen Professor of
Chemistry at The Scripps Research Institute, where his research is
focused on understanding protein homeostasis, as well as developing
new therapeutic strategies for genetic diseases associated with
loss-of-function and neurodegenerative diseases associated with
protein aggregation. He co-founded FoldRx Pharmaceuticals. Dr. Kelly
received his Ph.D. in organic chemistry from the University of North
Carolina at Chapel Hill and performed postdoctoral research at The
Rockefeller University in the area of chemistry and biology.

   Richard I. Morimoto, Ph.D., Founder

   Richard I. Morimoto is the Bill and Gayle Cook Professor of
Biology, Professor of Biochemistry, Molecular Biology and Cell
Biology, and Director of the Rice Institute for Biomedical Research at
Northwestern University. He received his Ph.D. in Biology from the
University of Chicago and did postdoctoral research at Harvard
University in Cambridge. His research on the regulation of the heat
shock stress response and the function of molecular chaperones
addresses questions on the integration of organismal stress in
response to physiologic and environmental stress and the chronic
expression of misfolded and damaged proteins.

   About the Proteostasis Network(TM)

   The Proteostasis Network is comprised of over a dozen biological
pathways that maintain a critical balance among protein synthesis,
folding, aggregation, trafficking and degradation to ensure health.
When functioning properly, the Proteostasis Network ensures that every
protein in the cell will either reach its final destination in a fully
functional state or be eliminated to prevent damage. Stress on the
Proteostasis Network from aging, genetic or environmental insults can
lead to imbalances that result in serious diseases. The maintenance of
the Proteostasis Network is as important to health and well-being as
maintenance of the genome. Thus, therapeutics that can control the
Proteostasis Network have the potential to change the practice of

   About Proteostasis Regulators

   Advances in our ability to characterize and pharmacologically
control protein homeostasis, or the Proteostasis Network, create new
opportunities to ameliorate a wide range of diseases. It is now
possible to use orally available small molecules, or Proteostasis
Regulators, to restore the natural balance of the Proteostasis
Network. It has been demonstrated that Proteostasis Regulators can
fold mutated proteins, potentially offering a significant benefit to
patients suffering from genetic diseases. Scientists have also shown
that Proteostasis Regulators can control other biological pathways
within the Proteostasis Network to ameliorate degenerative diseases
associated with protein aggregation, such as Alzheimer's and
Huntington's diseases.

   About Proteostasis Therapeutics, Inc.

   Proteostasis Therapeutics is discovering and developing novel
small molecule therapeutics designed to control the body's protein
homeostasis, or Proteostasis Network. The Proteostasis Network
maintains the body's natural balance of proteins to protect us from
numerous diseases. These novel therapies, or Proteostasis Regulators,
are designed to treat multiple genetic and degenerative disorders
associated with deficiencies of the Proteostasis Network, such as
emphysema, type II diabetes, Alzheimer's Disease and Huntington's

MacDougall Biomedical Communications
Douglas MacDougall or Jennifer Greenleaf, 781-235-3060

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