LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 04, 2018 (GLOBE NEWSWIRE) -- 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 the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application 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.
Usher syndrome is the leading cause of combined deafness and blindness. Exon 13 mutations in the USH2A gene targeted by
QR-421a cause vision loss in approximately 16,000 individuals in the Western world. ProQR plans to start enrolling patients in a
Phase 1/2 trial named STELLAR in the coming months with preliminary data expected in mid-2019.
“We are pleased to be advancing QR-421a, our second therapy for an inherited retinal disease, into the clinic and continuing the
development of our portfolio of transformative RNA medicines for severe genetic rare diseases,” said Daniel A. de Boer, Chief
Executive Officer of ProQR. “QR-421a has shown promising activity in both the optic cup and zebra fish models and we are excited
about the potential to make a meaningful impact for Usher syndrome patients.”
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 the Phase 1/2 “STELLAR” trial
STELLAR, or PQ-421a-001, is a first-in-human study that will initially include approximately 18 adults who have vision loss due
to mutations in exon 13 of the USH2A gene and will be conducted at expert sites in the US and Europe. It will be a
double-masked, randomized study exploring several dose levels and a control (sham injection), given as a single intravitreal
injection of QR-421a into one eye. The first patient at each dose level will be dosed in an open-label manner. The objectives of
the trial will include evaluation of safety, tolerability, pharmacokinetics and efficacy, as measured by restoration or improvement
of visual function and retinal structure through ophthalmic endpoints such as visual acuity (BCVA), visual field and optical
coherence tomography (OCT). Changes in quality of life in the trial subjects will also be evaluated
Data from the first-in-human study are expected in mid-2019. Patients completing this trial will be able to participate in an
extension study if eligible. Results from the single dose trial will inform the next stage that will potentially include a
seamless adaptive, multi-dose, controlled trial.
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, dystrophic epidermolysis bullosa and cystic fibrosis. 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 Hayes
Vice President of Investor Relations and Corporate Communications
T: +1 202 360 4855
lhayes@proqr.com
Media Contact:
Sara Zelkovic
LifeSci Public Relations
T: +1 646 876 4933
Sara@lifescipublicrelations.com