The funding will be used for the clinical development of QR-313
Clinical trial in DEB patients to start in the second quarter of 2018
LEIDEN, the Netherlands, June 12, 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 a collaboration with nonprofit organizations EB Research Partnership (EBRP) and EB Medical Research Foundation (EBMRF) to
develop QR-313 for patients with dystrophic epidermolysis bullosa (DEB) caused by mutations in exon 73 of the COL7A1 gene.
Under the agreement, EBRP and EBMRF will provide up to approximately $5 million in matching funding to ProQR under a venture
philanthropy model, to co-fund the clinical development of QR-313.
“The EB Research Partnership and EB Medical Research Foundation are exceptional organizations and we look forward to collaborate
on our mission to develop a life changing treatment for patients with this devastating disease," said Daniel A. de Boer, Chief
Executive Officer of ProQR. “With the funding from these partners, we plan to accelerate the development of QR-313 for the
treatment of DEB. And if we are successful with QR-313, we believe there is potential to expand this approach of exon skipping into
other mutations that cause DEB and help even more patients.”
DEB is a rare debilitating skin disease caused by the absence of a protein called collagen type VII (C7). Symptoms are present
from birth and include easy blistering of the skin, poorly healing wounds, skin infections and, in adulthood, some patients develop
very aggressive forms of skin cancer. There are currently no approved treatment options for DEB. The WINGS trial, a first-in-human
Phase 1/2 clinical trial of QR-313 in patients with DEB, is expected to initiate enrollment of patients in the first half of 2018.
The trial is expected to report interim proof of mechanism results later this year, with full results expected in 2019.
“DEB has a huge impact on patients' quality of life and there are currently no approved disease modifying treatment options
beyond wound care," said Alex Silver, founder and Chairman of EBRP and Paul Joseph, CFO of EBMRF. "Our organizations are excited to
partner with the ProQR team, in the hopes that the development of QR-313 will provide a much-needed treatment option to this
community.”
About EB Research Partnership
Founded in 2014, EB Research Partnership (EBRP) is the largest 501(c)(3) nonprofit dedicated to funding research aimed at
treating and ultimately curing Epidermolysis Bullosa, a group of devastating and life-threatening skin disorders that affect
children from birth. EB Research Partnership works to treat and cure EB as quickly and efficiently as possible and fulfills their
mission by partnering with non-profit and for-profit organizations, foundations, individual donors, and the EB and research
communities.
EB Research Partnership utilizes an innovative business model of venture philanthropy, leveraging concepts from principal
investing and applying them towards achieving philanthropic goals. When making a grant to a research project, they retain the added
upside of generating a recurring donation stream if the therapy or product is commercially successful, then use this revenue to
fund additional EB research. To learn more about EB Research Partnership visit www.ebresearch.org
About EB Medical Research Foundation
As the leader in research funding, the EBMRF is an all-volunteer, non-profit 501(c) Foundation dedicated to funding research for
Epidermolysis Bullosa to determine its causes, develop successful treatments, and ultimately find a cure. The grants awarded
with your donations fund an aggressive research agenda aimed at developing breakthrough therapies in collaboration with
Universities and leading private and public biotechnology companies. The EBMRF's scientific collaborations incorporate a venture
philanthropy model, in which we participate in the economics of any potential scientific commercialization. Royalties and revenue
generated from our venture agreements are then reinvested to further advance critical research. The foundation's goal is to cure EB
by raising awareness through special events, the media and fundraising programs. For more information, please visit
www.ebmrf.org
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*
About Dystrophic Epidermolysis Bullosa
Epidermolysis bullosa (EB) is a group of rare genetic skin diseases of which dystrophic EB (DEB) is one of the most severe
forms. DEB is caused by a mutation in the COL7A1 gene which is responsible for the formation of the Collagen type VII (C7)
protein that forms anchoring fibrils that bind the inner and outer layers of the skin and mucosal membranes together. This mutation
causes a loss of the anchoring fibrils resulting in fragile skin. People with DEB live with constant pain and have a high risk of
malnutrition and infections. Symptoms include easy blistering, poorly healing wounds, skin infections, fusion of fingers and toes,
anemia, gastrointestinal tract problems and with adulthood some develop very aggressive forms of skin cancer. There are no approved
treatment options available that target the underlying cause of DEB.
About QR-313
QR-313 is a potential 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. QR-313 has been granted Orphan Drug Designation in the United
States and the European Union.
About the WINGS Trial
The WINGS trial is a first-in-human Phase 1/2 double-blind, randomized, intra-subject placebo controlled clinical trial of
QR-313 in approximately eight subjects (over two years of age) that have RDEB due to mutation(s) in exon 73 of the COL7A1
gene.
Primary objectives will be to evaluate the safety and tolerability of topically applied QR-313 while providing biomarker
evidence of proof of mechanism (exclusion, or skipping, of exon 73 from COL7A1 mRNA assessed by polymerase chain reaction).
Secondary objectives will be to assess the effects on wound healing, skin strength, the presence of collagen type 7 protein and
anchoring fibrils in the skin and systemic distribution of QR-313 after topical administration.
QR-313 or placebo formulated in a gel will be topically applied to a patient’s wounds two to three times a week (depending on
bandage change frequency) for up to four weeks with an additional eight week follow-up period. The trial will be conducted at ten
specialized centers in the U.S. and selected European countries. Interim proof of mechanism results from the trial are expected in
2018. Full results from the study are expected in 2019.
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. Such statements include, but are not limited to, those statements regarding our partnerships with EB Research
Partnership and EB Medical Research Foundation, including statements regarding the expected funding and benefits of such
partnerships and our product candidate QR-313, including its clinical development and therapeutic potential and future development
plans. 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. Our actual results could differ materially from those anticipated in these forward-looking
statements for many reasons, including, without limitation, 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.:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com