ProQR's Drug Candidate QRX-411 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMA
July 05 2017 - 7:00AM
Key Updates
-
ProQR's QRX-411 receives Orphan Drug Designation
by the FDA and EMA for the treatment of retinitis pigmentosa,
including Usher syndrome, the subtype targeted by QRX-411. Usher
syndrome is an inherited condition that is characterized by
combined deafness and blindness.
- QRX-411 targets the pseudo-exon 40 (PE-40)
mutation in the USH2A gene and currently there are no therapies
commercially available or in clinical development for the vision
loss associated with this disease.
- QRX-411 has shown promising preclinical data in
both patient fibroblasts and the optic cup model for mRNA
restoration, which was presented at the Annual Meeting of the
Association for Research in Vision and Ophthalmology (ARVO) in May
2017.
- A lead candidate has been selected for this
program and is currently ready for IND-enabling studies.
- QRX-411 is part of ProQR's ophthalmology pipeline
that currently also includes one clinical compound, QR-110 for
Leber's Congenital Amaurosis Type 10, and three preclinical
programs, QRX-421 for Usher syndrome, QRX-1011 for Stargardt's
disease and QRX-504 for Fuchs endothelial corneal dystrophy.
LEIDEN, the Netherlands, July 05, 2017 (GLOBE
NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced
that the company's investigational drug QRX-411 has received orphan
drug designation (ODD) from the U.S. Food and Drug Administration
(FDA) and European Medicines Agency (EMA) for the treatment of
retinitis pigmentosa, including Usher syndrome, the subtype
targeted by QRX-411. Usher syndrome is the leading cause of
combined deafness and blindness due to genetic defects in the Usher
gene.
ODD in the U.S. and European Union provides a
special status for investigational drugs being developed for rare
diseases. The ODD programs offer 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., and ten years
in the European Union following market approval.
"We are pleased with the progress we have made to
date with our novel RNA based therapeutic ophthalmology pipeline
for patients suffering from genetic eye diseases. Securing
orphan drug designations from the FDA and EMA for QRX-411 is a
milestone for the program and highlights the importance of
addressing the unmet need of this debilitating disease," said
Daniel A. de Boer, CEO of ProQR, "The severe genetic retinal
diseases we are targeting do not have any available therapies,
especially disease modifying therapies focused on restoring vision
or impeding progression of the disease. We believe our novel
RNA oligonucleotide approach has the potential to make a meaningful
impact in the lives of Usher syndrome patients and others with rare
genetic eye diseases."
Chief Development Strategy Officer, David M.
Rodman, MD, notes, "At ProQR we have a unique opportunity to
combine the flexibility of our oligonucleotide drug discovery
platform with accelerated drug development strategies for rare
diseases. Orphan drug designation is an important step in
rapidly bringing transformational precision medicines to patients
with Usher syndrome and many other genetic causes of blindness in
children and adults."
ProQR's growing ophthalmology portfolio
includes:
-
QR-110 for Leber's congenital amaurosis Type 10
(LCA 10) due to the p.Cys998X mutation, which received IND and CTA
clearance and is in clinical development (PQ-110-001 Phase 1/2
safety and efficacy study). QR-110 was also granted Fast
Track designation by the FDA and Orphan Drug designation by the FDA
and EMA.
- QRX-411 for Usher syndrome type II due to the
PE-40 mutation in the USH2A gene, for which a clinical candidate
has been selected and is ready for IND enabling development
studies.
- QRX-421 for Usher syndrome type II due to Exon 13
mutations in the USH2A gene, for which a clinical candidate has
been selected and is ready for IND enabling development
studies.
- QRX-1011 for Stargardt's disease due to
c.5461-10T>C mutations in the ABCA4 gene, which is in
optimization phase.
- QRX-504 for Fuchs endothelial corneal dystrophy
(FECD), for which a clinical candidate has been selected and is
ready for IND enabling development studies.
About Usher
Syndrome
Usher syndrome is the leading cause of combined
deafness and blindness. Patients with this syndrome generally
progress to a stage in which they have very limited central vision
and moderate to severe deafness. To date, there are no treatments
approved or products in clinical development that treat the vision
loss associated with the disease. Usher syndrome Type II is one of
the most common forms of Usher syndrome and is caused by mutations
in the USH2A gene.
About QRX-411
QRX-411 is a first-in-class RNA-based
oligonucleotide designed to address the underlying cause of Usher
syndrome due to the c.7595-2144A>G mutation in the USH2A gene.
The mutation is a substitution of one nucleotide in the pre-mRNA
that leads to aberrant splicing of the mRNA and non-functional or
absence of USH2A protein. QRX-411 is designed to restore wild-type
USH2A mRNA leading to the production of wild-type USH2A protein by
binding the mutated pre-mRNA causing normal splicing of the
pre-mRNA.
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 Type 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
QRX-411 and the clinical development and therapeutic potential
thereof, statements regarding orphan drug designation, including
the intended benefits of such status, statements regarding our
ongoing and planned discovery and development of product candidates
and the timing thereof, including those in our ophthalmology
portfolio, and statements regarding our oligonucleotide drug
discovery platform. 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, 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.
Contact:
Bonnie Ortega
Director, Investor Relations
T: +1 858 245 3983
ir@proqr.com
This
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The issuer of this announcement warrants that they are solely
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information contained therein.
Source: ProQR Therapeutics N.V. via Globenewswire
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