Ensemble Therapeutics and Alexion Initiate Collaboration to Discover Macrocycle Drug Candidates Targeting Patients with Severe

  Ensemble Therapeutics and Alexion Initiate Collaboration to Discover
  Macrocycle Drug Candidates Targeting Patients with Severe and Ultra-Rare

 Goal of Discovering Small Molecule Drug Candidates for Worldwide Development
                       and Commercialization by Alexion

Business Wire

CAMBRIDGE, Mass. -- July 18, 2013

Ensemble Therapeutics, a biotechnology company developing Ensemblins™, a novel
class of small molecule therapeutics with the potential to modulate targets
typically addressed by biologics, today announced the initiation of a drug
discovery collaboration with a subsidiary of Alexion Pharmaceuticals, Inc.
(Nasdaq: ALXN), the global biopharmaceutical company focused on developing and
commercializing life-transforming therapies for patients with
life-threatening, ultra-rare disorders. The collaboration will deploy
Ensemble’s proprietary drug discovery platforms for several undisclosed drug
targets identified by Alexion to create highly innovative small molecule
therapeutic candidates.

Under the terms of the collaboration, Ensemble will screen its Ensemblin™
collection of more than ten million macrocycles against several disease drug
targets specified by Alexion, utilizing Ensemble’s extensive medicinal
chemistry capabilities to discover new small molecule clinical candidates.
Alexion will have the exclusive worldwide rights to develop and commercialize
candidates arising from the collaboration. Other terms of the agreement were
not disclosed; Ensemble will receive an upfront payment and research support,
and can earn additional payments upon the achievement of certain development
and commercial milestones.

“Ensemble is thrilled to support Alexion’s cutting edge scientific and
clinical programs with our ever increasing capability for macrocycle-based
drug discovery,” said Dr. Michael D. Taylor, CEO of Ensemble Therapeutics.
“This new collaboration with a leading biopharmaceutical innovator focused on
rare diseases speaks to the breadth of utility of Ensemble’s discovery
platforms to address clinically important yet challenging drug targets,” Dr.
Taylor added.

“Alexion looks forward to a productive collaboration with Ensemble as we seek
to broaden the number of pathways toward developing potentially transformative
first-in-class drug candidates for patients with selected severe and
life-threatening ultra-rare disorders,” said Martin Mackay, PhD, Executive
Vice President, Head of Global R&D at Alexion. “This is a promising addition
to our development initiatives as we seek to further expand our product

About Ensemblins

Ensemblins™ are a new class of synthetic macrocycles developed by Ensemble
using its proprietary chemistry platforms, including DNA-Programmed Chemistry.
Macrocyclic rings are found in many natural product-based drugs and bestow
favorable pharmaceutical properties and powerful protein surface binding
properties upon such drugs. Thus, macrocycles are uniquely suited to address
many protein targets that cannot be modulated effectively by traditional small
molecule pharmaceutical compounds. Macrocycles have been challenging to
synthesize in large numbers and this has constrained their wider use in the
industry. By extending beyond the limits of traditional small molecule drug
discovery, Ensemble’s platform provides unmatched capabilities to successfully
and reliably generate millions of macrocyclic Ensemblins as drug candidates,
larger than any collection previously synthesized in the pharmaceutical

About Ensemble Therapeutics

Based in Cambridge, MA, Ensemble Therapeutics is deploying its proprietary
chemistry platforms to develop a novel class of therapeutics known as
“Ensemblins”. Ensemble is leveraging its macrocycle drug discovery expertise
to fuel its proprietary drug candidate pipeline while also pursuing
collaborations with pharmaceutical partners. Prior to this agreement, Ensemble
entered high-value partnerships including alliances with Boehringer Ingelheim,
Genentech, Bristol-Myers Squibb and Pfizer to access Ensemble’s macrocycle
libraries for purposes of affinity screening drug discovery against
difficult-to-address targets. Ensemble’s internal discovery and development
efforts are focused on the key therapeutic areas of oncology and
immuno-inflammatory diseases, with its lead program, a small molecule
antagonist of Interleukin-17, a cytokine implicated in multiple inflammatory
and autoimmune diseases, poised to enter development with an orally active
candidate by the end of 2013. For more information, visit: www.ensembletx.com.


For Ensemble
The Yates Network
Gina Nugent, 617-460-3579
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