Nimbus Discovery Announces Agreement with Shire to Co-Develop Therapies for Rare Diseases

  Nimbus Discovery Announces Agreement with Shire to Co-Develop Therapies for
  Rare Diseases

    Companies Enter Agreement to Co-Develop First-in-Class Small Molecule
                Therapies for Key Lysosomal Storage Disorders

Business Wire

CAMBRIDGE, Mass. -- May 8, 2013

Nimbus Discovery, LLC, a biotechnology company discovering novel medicines
against exciting but previously inaccessible disease targets, today announced
a co-development agreement with Shire plc focused on small molecule treatments
for several rare genetic diseases known as lysosomal storage disorders (LSDs).
The goal of the collaboration is to utilize the Nimbus breakthrough
computational chemistry approach to discover and develop novel,
disease-altering therapies. Many LSDs remain untreated because of challenges
in creating drugs that can effectively reach the disease site. The significant
potential of this partnership is the development of the first small molecule
agents designed to penetrate inaccessible tissues while offering the
convenience of an orally-administered pill.

The Nimbus Shire collaboration is the result of a joint assessment of a series
of important rare disease targets with significant unmet medical need. One
target was ultimately chosen to be the research focus. Under the terms of the
agreement, Nimbus will use its cutting-edge research and development platform
to extensively profile molecules against the agreed upon disease target and
will deliver a drug candidate that is ready to enter late preclinical studies.
Nimbus will control and conduct all related research up to achievement of drug
candidate status at which point Shire will have an exclusive option to acquire
the program. Shire will then be responsible for all clinical development and
future commercialization activities. Nimbus is eligible to receive
preclinical, development and commercial stage milestones commensurate with the
innovative nature of the research and significant unmet medical need of the
disease indications.

“This collaboration validates our computationally-driven, structure-based drug
discovery engine and innovative partnering model,” said Rosana Kapeller, M.D.,
Ph.D., Chief Scientific Officer of Nimbus. “Nimbus is thrilled to pursue our
first alliance with Shire, a company that shares our passion for uncovering
breakthroughs in highly sought-after disease targets that have proven
inaccessible to traditional industry approaches.”

“Nimbus is ground-breaking in their approach to drug discovery and, in a short
period of time, have already assembled an impressive track record in
delivering clinical candidates for challenging disease targets,” said Dr.
Philip J. Vickers, Senior Vice President, Research and Development, Shire. “As
a leader in rare diseases, this partnership is another way for Shire to ensure
that we expand into new disease areas and continue to apply cutting edge
technologies in this space. This agreement complements Shire’s long-term
commitment to bring innovative therapies to patients with rare diseases

This is the first deal announced through Shire’s strategic alliance with Atlas
Venture, which identifies investments for early stage venture creation around
rare genetic diseases.

About Lysosomal Storage Disorders

Lysosomal Storage Disorders are a group of approximately 50 rare inherited
metabolic disorders that are caused by a lack of enzymes that normally
eliminate unwanted substances in human cells. Lysosomes act as the "recycling
center" of each cell, breaking down unwanted material into simple products for
the cell to use for building new material. The lack or malfunction of certain
enzymes causes a buildup of the molecules that the enzyme would normally
eliminate, and deposits accumulate in many cells of the body. Abnormal storage
impairs cell function and can cause damage to the body's cells, which can lead
to serious health problems. Lysosomal storage diseases predominantly affect
children, many of which die within a few months or years of birth if

About Nimbus

Nimbus Discovery, a biotechnology company, harnesses cutting-edge
computational technologies to uncover breakthroughs in small molecule
pharmacology. Nimbus focuses on medically important and highly sought-after
disease targets that have proven inaccessible to traditional industry
approaches. Nimbus’ robust pre-clinical pipeline includes novel agents for the
treatment of cancer, metabolic disease and inflammation. The company is
organized as a constellation of small, nimble teams of experienced
drug-hunters deployed across program-focused subsidiary companies. Each team
is freed from conventional barriers to scientific success, chartered to create
solutions, and geared for program asset deals with leading pharmaceutical
companies. Founded in 2009, Nimbus partnered with Schrödinger to invent and
apply a physics-based approach that establishes a new standard for rational
drug design. Nimbus is backed by world-class life science investors, including
Atlas Venture, SR One, Lilly Ventures and Bill Gates. For more information
please visit


Nimbus Discovery LLC
Jonathan Montagu, +1-617-588-2600
Vice President, Business Development & Operations
Suda Communications LLC (Media)
Maureen L. Suda, +1-585-387-9248
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