ProQR Receives Orphan Drug Designation from FDA for Drug Candidate QR-313 for Dystrophic Epidermolysis Bullosa and will Prese...
September 19 2017 - 7:00AM
Key Updates
ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that
investigational drug QR-313 for dystrophic epidermolysis bullosa
(DEB) has received orphan drug designation (ODD) from the U.S. Food
and Drug Administration (FDA). QR-313 is a first-in-class RNA-based
oligonucleotide designed to address the underlying cause in
dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73
of the COL7A1 gene. DEB is a rare genetic disease that can lead to
severe blistering of the skin resulting in high treatment burden
and poor quality of life for patients.
“We are pleased to have ODD designation in the U.S. for our
QR-313 program targeting dystrophic epidermolysis bullosa,” said
David M. Rodman, MD, Chief Development Strategy Officer of
ProQR, “It highlights the unmet need in this devastating
disease, for which we aim to make a meaningful difference. Our goal
for this disease is to develop a pipeline of programs that can
treat DEB mutations in a targeted manner and to actively advance
the pipeline through development.”
Poster Presentations at Upcoming Scientific
Conferences
The Company will present two posters (# 50 and 51) during the
EB2017 - 5th World Conference of Epidermolysis Bullosa
Research Conference from September 24-26, 2017 in
Salzburg, Austria.
The same posters (# 181 and 194) will also be presented at the
47th Annual European Society for Dermatological Research
(ESDR) Meeting on September 29, 2017 in Salzburg, Austria.
Poster #181 is selected for a presentation (walk title: Genetics
and Cell Based Therapy 2: Epidermolysis bullosa) on September 29 at
14.35-15.30 CET.
The posters are titled:
- Local delivery of an antisense oligonucleotide for recessive
dystrophic epidermolysis bullosa.
- In vitro evaluation of QR-313; an antisense oligonucleotide
designed to skip exon 73 from the COL7A1 mRNA.
About Orphan Drug Designation (ODD)
Orphan drugs are intended for the treatment, diagnosis or
prevention of serious diseases that affect fewer than 200,000
people in the U.S., or that affect more than 200,000 persons but
are not expected to recover the costs of developing and marketing a
treatment drug. FDA evaluates scientific and clinical data
submissions from sponsors to identify and designate products as
promising for rare diseases and to further advance scientific
development of such promising medical products. FDA provides
incentives for sponsors to develop products for rare diseases,
including development program tax benefits and a waiver of the NDA
application user fee, as well as market exclusivity for up to seven
years in the U.S.
About QR-313
QR-313 is a first-in-class RNA-based oligonucleotide designed to
address the underlying cause of dystrophic epidermolysis bullosa
(DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in
this exon can cause loss of functional collagen type VII (C7)
protein. Absence of C7 results in the loss of anchoring fibrils
that normally link the dermal and epidermal layers of the skin
together. QR-313 is designed to exclude exon 73 from the mRNA (exon
skipping) and produce a functional C7 protein, thereby restoring
functionality of the anchoring fibrils.
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*
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 QR-313 and
the clinical development and the therapeutic potential thereof, and
statements regarding our pipeline of programs targeting DEB. Our
actual results could differ materially from those anticipated in
these forward-looking statements for many reasons, including,
without limitation, risks associated with our clinical development
activities, including that we may not realize the intended benefits
afforded by orphan drug designation for our QR-313 program
targeting DEB, positive results observed in our 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.:
Media Contact:Sariette WitteT: +31 6 2970 4513
(NL)T: + 1 213 261 8891 (US)pr@proqr.com
Investor Contact:Bonnie OrtegaT: +1 858 245
3983ir@proqr.com
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