ProQR Receives Fast Track Designation from FDA for QR-421a for Usher Syndrome Type 2
January 02 2019 - 7:00AM
ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to
changing lives through the creation of transformative RNA medicines
for the treatment of severe genetic rare diseases, today announced
that it received Fast Track designation from the Food and Drug
Administration (FDA) for QR-421a. QR-421a is a first-in-class
investigational RNA-based oligonucleotide designed to address the
underlying cause of the vision loss associated with Usher syndrome
type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations
in exon 13 of the USH2A gene.
Fast Track designation is granted by FDA to drugs that are under
development for serious conditions and have the potential to
fulfill an unmet medical need. It was established with the
intention to bring promising drugs to patients sooner by
facilitating the development with more frequent FDA interactions
and expediting the review process.
“We are very pleased with the Fast Track designation the FDA
granted us for QR-421a. Patients with Usher syndrome, the leading
cause of combined deafness and blindness, currently have no
available therapies for their vision loss and this designation
emphasizes the high unmet need in this disease,” said Daniel de
Boer, Chief Executive Officer of ProQR. “We are also looking
forward to begin enrollment in the Phase 1/2 STELLAR clinical trial
in the coming months with preliminary data expected in
mid-2019.”
About QR-421a
QR-421a is a first-in-class investigational RNA-based
oligonucleotide designed to address the underlying cause of vision
loss in Usher syndrome type 2 and non-syndromic retinitis
pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Mutations in this exon can cause loss of functional usherin protein
that causes the disease. QR-421a is designed to exclude the genetic
defect from the RNA in the eye, such that it leads to the
expression of a shortened but functional usherin protein, thereby
modifying the underlying disease. QR-421a has received orphan drug
designation in the United States and the European Union.
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. Usher syndrome type 2 is one of the most common
forms of Usher syndrome and is caused by mutations in the USH2A
gene. To date, there are no approved treatments or products in
clinical development that treat the vision loss associated with
Usher syndrome type 2.
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 Leber’s congenital amaurosis
10, Usher syndrome type 2 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-421a and
its clinical development and therapeutic potential, including
commencement of the STELLAR trial, trial design and timing of
results from this trial. 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 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, regulatory review or approval process,
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:Lisa
HayesVice President of Investor Relations and Corporate
CommunicationsT: +1 202 360 4855ir@proqr.com
Media Contact:Sara ZelkovicLifeSci Public RelationsT: +1 646 876
4933Sara@lifescipublicrelations.com
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