Full Press Release Details
ProQR R&D day Highlights Progress on Pipeline and Introduces
Axiomer , a novel proprietary RNA Technology
Key program features and updates:
LEIDEN, the Netherlands, June 15, 2017 - ProQR Therapeutics N.V. (Nasdaq:PRQR), a development stage RNA therapeutics company will provide an update on
its product candidates today at an investor event, and introduce Axiomer , a novel RNA platform technology it pioneered. The R&D Day is hosted by the Company s management team and will include perspectives from several key opinion
leaders. ProQR s pipeline now includes two clinical programs, one preclinical program and two programs ready to enter development.
Therapeutics N.V. | Zernikedreef 9, 2333 CK Leiden, The Netherlands | +31 88 166 7000 | info@proqr.com | www.proqr.com
Since last year we have made good progress on executing on our strategy to develop life-changing therapies
for patients in need, through a diversified pipeline with a balanced risk profile, said Daniel A. de Boer, CEO of ProQR. We are pleased to show the progress we have made in our RNA therapeutics pipeline at our second R&D day.
Following QR-010 for CF, QR-110 is now in clinical trials for LCA 10, and our third molecule, QR-313 for DEB will move to
clinical trials early next year. We are focusing on three important genetic diseases, all with high unmet needs, and all which we believe could greatly benefit from our unique RNA oligonucleotide approach. Within the next 18 months we will have
generated clinical data in patients in all these programs.
Axiomer editing the RNA
ProQR is pioneering a next-generation RNA technology called Axiomer , which we believe has the potential to yield a new class of medicines for genetic
diseases. Axiomer can make single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells. The Axiomer Editing
Oligo Nucleotides , or EONs, recruit an endogenously expressed RNA editing system called ADAR, which it can direct to change an Adenosine (A) to an Inosine (I) in the RNA an Inosine is translated as a Guanosine (G). A member of
the Scientific Advisory Board, Dr. Levin will present the landscape and evolution of RNA therapeutics and provide his perspective on this unique and proprietary platform technology.
While our focus and priority is on clinical development of our most advanced RNA-based therapeutics to help
patients with CF, LCA 10 and DEB, we continue to innovate in RNA science. This innovation effort has led to the discovery of a novel RNA editing technology that we believe can address the underlying cause of a broad range of genetic defects at the
RNA level, said Daniel de Boer. The invention and patenting of Axiomer can drive drug discovery and development of a new class of therapeutics, independently and through partnerships.
The R&D Day will feature presentations by ProQR senior management including Daniel de Boer (Chief Executive Officer), Noreen Henig (Chief Medical
Officer), Peter Adamson (Head of Ophthalmology), Gerard Platenburg (Chief Innovation Officer) and David Rodman (Chief Development Strategy Officer). Discussions will focus on a review and introduction of several near- and medium term value drivers
and progress on the Company s pipeline. In addition, several leading medical researchers will discuss the state of the art in research and development in relation to the company s pipeline:
Steven M. Rowe, M.D., MSPH, Professor, Department of Medicine, Pediatrics, and Cell Developmental & Integrative Biology, and
Director Gregory Fleming James Cystic Fibrosis Research Center University of Alabama.
Dr. Rowe is a respected academic physician scientist and a
pioneer in the field of personalized therapeutics for cystic fibrosis, cutting-edge discovery in airway disease biology, and translational research in COPD. He is an international authority in the design and conduct of clinical trials targeting the
basic CF defect, and has made key advances in the measurement and interpretation of CFTR function. He directs the Cystic Fibrosis Research Center at UAB, which involves over 100 faculty members and has been continuously funded for over 25 years. A
board-certified physician, Dr. Rowe serves as a Special Consultant for Translational Science for the Cystic Fibrosis Foundation. He presently has a laboratory of over 25 individuals, embracing lung research from basic discovery, to
translational science, to clinical application.
Stephen M. Rose, Ph.D., Chief Research Officer at Foundation Fighting Blindness.
Dr. Rose oversees the day-to-day operations of the Foundation Fighting
Blindness Science Department. He also works closely with the clinical arm of the Foundation to establish a seamless pipeline of studies to move preventions and treatments into clinical trials, partnering with biotech and pharma to maximize
potential commercialization. Prior to joining the Foundation in 2004, Dr. Rose was a Director in the NIH Office of Science Policy, where he provided oversight on issues regarding recombinant DNA, including human gene transfer clinical
protocols. Dr. Rose currently sits on the FDA s Cellular, Tissue and Gene Therapies Advisory Committee and is a Health Research Alliance Board member.
M. Peter Marinkovich, M.D., Associate Professor, Blistering Disease Clinic Department of Dermatology, Stanford
University School of Medicine.
Dr. Marinkovich is an Associate Professor of Dermatology, a faculty member of the Program in Epithelial Biology
and the Stanford Cancer Biology Program. He has an interest in inflammatory skin disease and is Director of the Stanford Epidermolysis Bullous Disease and Psoriasis Clinics. He is also an attending dermatologist at the VA Palo Alto Medical Center.
Dr. Marinkovich s research focuses on pathogenesis and therapy of epidermolysis bullosa, psoriasis, hair disorders and skin cancers.
Levin, Ph.D., international RNA expert and member of the ProQR Scientific Advisory Board.
Dr. Levin has three decades of experience
in RNA drug development from discovery through drug registration, both in large pharma and biotech companies. He has been key to the development of numerous of oligonucleotides, including the first approved antisense medicines, and the first
microRNA-targeted therapeutic in clinical trials. Dr. Levin has published over 60 scientific articles and served as a director of the Oligonucleotide Therapeutics Society. He has served on ProQR s Scientific Advisory Board since the
company s inception.
R&D Day Event details
Today, ProQR will host an R&D day in New York, NY from 8:00am to 1:00pm ET. Please email Ronen Abergel, rabergel@troutgroup.com to receive more
information and to reserve a seat.
live webcast can be accessed at www.proqr.com/rd-day. The archived webcast of the presentation will be accessible from the Investor Relations section of ProQR s website
(www.proqr.com) under Events and Presentations . The archived webcast will be available for 90 days following the presentation date.
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 the therapeutic potential of our RNA technology, our innovation programs, the timing of our clinical programs and availability of data and our R&D day. 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
Chief Financial Officer