ProQR spins out Amylon Therapeutics as a privately-held company focused on central nervous system (CNS) therapeutics
September 12 2017 - 6:00AM
Key updates
ProQR Therapeutics N.V. (Nasdaq:PRQR), today announced that the
company has spun out Amylon Therapeutics (Amylon), a privately-held
company focused on the development of therapies for central nervous
system (CNS) disorders, with seed funding from a group of
institutional and private investors. As part of the transaction,
ProQR has granted an exclusive license to Amylon to develop
therapeutics for beta amyloid related disorders. Amylon will use
the license and proceeds to continue research and development of
therapies for CNS disorders, with an initial focus on a RNA-based
treatment for a rare genetic disease that leads to strokes at
mid-adulthood, called Hereditary Cerebral Hemorrhage with
Amyloidosis of the Dutch type (HCHWA-D). A future beta amyloid
related indication could include cerebral amyloid angiopathy (CAA).
ProQR retains majority ownership in Amylon and is entitled to
future milestones and royalties from the products developed by
Amylon on the terms and conditions set forth in the license.
Amylon will be led by Thomas de Vlaam as founding CEO, who holds
a bachelor’s degree in international medicine and global health.
Thomas joined ProQR in 2015 to incubate the CNS activities, with
the goal of spinning it out into a new company. On behalf of ProQR,
Dinko Valerio, the Chairman of the Supervisory board of ProQR, will
join the Supervisory board of Amylon and Gerard Platenburg, Chief
Innovation Officer of ProQR, will join the scientific advisory
board of Amylon.
Since its inception in 2012, ProQR has invested significantly in
discovering and developing innovative RNA therapies for severe
genetic rare diseases. This has led to an extensive pipeline of
discovery development programs that are harbored in separate
business units with dedicated teams of specialists focused on
cystic fibrosis, eye diseases, skin diseases and CNS diseases.
“As part of our corporate strategy to bring as many of our
programs to patients as possible, we built ProQR as a group of
focused business units that enable us to attract external funding
directly into the individual development programs or ultimately
spin programs out as we have now done with Amylon,” said Daniel de
Boer, Chief Executive Officer of ProQR. “Through spinning out
Amylon, we position the beta amyloid program for optimal success
with a dedicated team and the funding to develop treatments for
patients suffering from these devastating brain diseases. Through
our involvement in the Supervisory board and scientific advisory
board of Amylon we will continue to assist Thomas and his team in
building success.”
”The unmet need of patients living with beta amyloid related
disorders is very significant and to date no disease modifying
treatments are available,” said Thomas Wisniewski, Director of the
Alzheimer’s Disease Research Center in New York. “The technology
that Amylon is developing for these patients is built on a solid
scientific rationale and holds great promise for patients.”
The scientific advisory board at Amylon will consist of the
following members:
- Thomas Wisniewski MD, Lulu P. and David J. Levidow Professor of
Neurology, Pathology and Psychiatry at the New York University
School of Medicine.
- Philip Scheltens MD, Professor of Neurology and Director of
Alzheimer Center at the VU University Medical Center
Amsterdam.
- Gerard Platenburg, Co-founder and Chief Innovation Officer of
ProQR.
About ProQR
ProQR Therapeutics is dedicated to changing lives through
the creation of transformative RNA medicines for the treatment of
severe genetic rare diseases such as cystic fibrosis, Leber’s
congenital amaurosis 10 and dystrophic epidermolysis
bullosa. Based on our unique proprietary RNA repair platform
technologies we are growing our pipeline with patients and loved
ones in mind. *Since
2012*
About Amylon
Amylon Therapeutics is a privately-owned company targeting CNS
disorders through an ultra genetics approach in which developing
treatments for rare genetic disorders can serve as a possible
gateway to global indications. It is using its technology for
beta-amyloid related disorders to initially target a rare disease
called HCHWA-D, with potential expansion into more prevalent
indications such as CAA and Alzheimer’s disease. Amylon is led by a
combination of ambitious entrepreneurs and experienced biotech
veterans. Amylon is a spin-off from NASDAQ listed ProQR
Therapeutics.
About HCHWA-D
Hereditary Cerebral Hemorrhage with Amyloidosis of the Dutch
type (HCHWA-D) is the genetic subtype of cerebral amyloid
angiopathy (CAA), in which a point mutation leads to the
accelerated onset of disease. The condition is characterized by a
progressive loss of intellectual function (dementia), stroke, and
other neurological problems starting in mid-adulthood. Most
affected individuals die within a decade after signs and symptoms
first appear, and after onset the quality of life diminishes
quickly and severely. There is currently no intervention available
nor in development to battle the cause and/or symptoms of
HCHWA-D.
About CAA
Cerebral amyloid angiopathy (CAA) is the leading cause of
hemorrhagic (bleeding) stroke in the elderly. The disease is caused
by the aggregation and deposition of the toxic peptide Amyloid Beta
in the blood vessels in the brain. These vascular amyloid deposits
ultimately cause irreparable damage to the vessel walls and lead to
hemorrhagic stroke. There are currently no effective prevention or
treatment strategies for CAA.
FORWARD-LOOKING
STATEMENTS
This press release contains forward-looking statements. All
statements other than statements of historical fact are
forward-looking statements, which are often indicated by terms such
as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,”
“intend,” “look forward to”, “may,” “plan,” “potential,” “predict,”
“project,” “should,” “will,” “would” and similar expressions.
Forward-looking statements are based on management’s beliefs and
assumptions and on information available to management only as of
the date of this press release. These forward-looking statements
include, but are not limited to, statements regarding Amylon
Therapeutics, our license with Amylon, including the future
milestones and royalty terms contained therein, the product
candidates of Amylon, including the clinical development and
therapeutic potential thereof, and other programs in our product
candidate pipeline. Our actual results could differ materially from
those anticipated in these forward-looking statements for many
reasons, including, without limitation, risks associated with our
ability to maintain our ownership interest in Amylon and or license
with Amylon, Amylon’s ability to continue development of its
product candidates and obtain additional funding for their
advancement, clinical development activities, including that
positive results observed in prior and ongoing studies may not be
replicated in later trials or guarantee approval of any product
candidate by regulatory authorities, manufacturing processes and
facilities, regulatory oversight, product commercialization,
intellectual property claims, and the risks, uncertainties and
other factors in our filings made with the Securities and Exchange
Commission, including certain sections of our annual report filed
on Form 20-F. Given these risks, uncertainties and other factors,
you should not place undue reliance on these forward-looking
statements, and we assume no obligation to update these
forward-looking statements, even if new information becomes
available in the future, except as required by law.
ProQR Therapeutics N.V.:Investor
Contact:Bonnie OrtegaT: +1 858 245 3983ir@proqr.com
Media Contact:Sariette WitteT: +31 6 2970 4513
(NL)T: + 1 213 261 8891 (US)pr@proqr.com
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