Full Press Release Details
ProQR Announces Initial Pipeline
Targets and Highlights Axiomer RNA Editing Platform Technology at R&D Event
LEIDEN, Netherlands &
CAMBRIDGE, Mass., March 29, 2023 - ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through
transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today announced initial pipeline
programs focused on diseases that originate in the liver. ProQR will host a virtual R&D event today, during which the Company will
showcase its proprietary Axiomer RNA-editing technology platform, detail the pipeline, and provide guidance on the advancement of
programs toward the clinic. ProQR also reported its 2022 year-end financials and the extension of its cash runway guidance.
R&D event highlights the important progress we have made to advance our proprietary Axiomer RNA editing platform technology and demonstrate
its broad applicability, as we develop treatments for diseases with high unmet need," said Daniel A. de Boer, Chief Executive Officer
of ProQR. "Along with our preclinical proof of concept data for the platform, a partnership with Eli Lilly that is exclusively
focused on RNA editing, leading IP position, and cash runway into mid-2026, ProQR is leading the advancement of RNA editing as a new
class of therapies for patients."
ProQR today announced
AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 as initial pipeline programs.
These programs share several key characteristics including a deep rooting in human genetics, the potential to have a major impact in
indications with high unmet medical need, the ability to leverage the existing proven delivery technology to the liver, the opportunity
to monitor early biomarkers to establish target engagement in Phase I trials for human proof of concept, and the availability of well-defined
AX-0810 for Cholestatic
Diseases targeting NTCP
Cholestatic disorders
are caused by a buildup of bile acids in the liver. Without treatment, the damage progresses through various stages to ultimately liver
failure. Liver transplants are often necessary for primary sclerosing cholangitis (PSC) and biliary atresia (BA), two forms of cholestatic
disease where currently there are no approved drugs.
to introduce a loss of function (LOF) variant that has been found in human genetics to prevent re-uptake of bile acids in liver. Based
on its mechanism of action, AX-0810 has the potential to become a disease modifying treatment for a range of cholestatic diseases.
AX-1412 for Cardiovascular
Disease targeting B4GALT1
Cardiovascular diseases
(CVDs) are a group of health conditions that affect the heart and blood vessels, such as atherosclerosis which can lead to severe problems
like heart attacks, heart failure, and stroke.
AX-1412 introduces a
variant into B4GALT1 that is associated in human genetics with a significantly lower chance of developing cardiovascular disease. ProQR
intends to advance AX-1412 targeting B4GALT1 to early clinical proof of concept stage, then would seek to partner this program.
ProQR Axiomer Platform
the Company also highlights platform proof of concept data, including consistent RNA editing reported in models in nervous system and
Additionally, ProQR's
Axiomer technology achieves increased editing efficiency and hepatocyte uptake in vivo demonstrating that GalNAc delivery technology
does not interfere with A-to-I editing.
Beyond correction, the
Axiomer RNA editing technology platform has broad applicability and proof of concept in multiple forms of protein modulation including
by ability to modulate proteins by altering function, changing post-translational modifications, and modifying protein interactions,
as shown in preclinical models.
the field and ProQR are making in optimizing ADAR for therapeutic use is exciting," said Peter Beal, PhD, Professor at the University
of California at Davis. "I look forward to continuing to uncover the potential of this technology as a new approach for the treatment
of a variety of diseases."
priorities and milestones
Pipeline: ProQR expects
to advance AX-0810 targeting NTCP and AX-1412 targeting B4GALT1 into clinical development in late 2024/early 2025.
Platform: The Company
will share various platform updates over the next 12 months, including liver NHP data, at scientific conferences, as well as research
related to ongoing discovery efforts.
will continue to execute on its existing partnership with Lilly. Additionally, ProQR may selectively form new partnerships, which could
include multi-target discovery alliances, similar to the Company's partnership with Lilly, or product alliances on specific programs.
The Company is also seeking to partner its ophthalmology assets (which do not utilize Axiomer technology.)
position: ProQR invented the use of endogenous ADAR in RNA editing with editing oligonucleotides (EONs) in 2014 and filed a first patent
application in that same year. Since then, ProQR has filed multiple additional patent applications on further improvements to form a
leading patent estate that supports ProQR's ADAR-mediated RNA editing platform Axiomer. Today ProQR has extensive patent protection
related to Axiomer, including 10 published patent families, that currently comprise a total of 22 patents. Beyond this, ProQR has several
unpublished patent applications and continuously invests in expanding its IP estate around ADAR-mediated RNA editing.
Maintain strong balance
sheet: ProQR's current cash runway is expected to fund operations into mid-2026. This guidance excludes any additional potential
future income from partnerships, including the potential Lilly opt-in fee of $50 M for 5 additional targets, milestone payments related
to the Lilly partnership, income from potential new partnerships related to Axiomer, and income from a potential transaction related
to the Company's ophthalmology assets.
Year End 2022 Financial
At December 31, 2022, ProQR
held cash and cash equivalents of 94.8 million, compared to 187.5 million at December 31, 2021. Subsequent to the
year end, in February 2023 ProQR received $60.0 million from Lilly, as part of the expanded licensing and collaboration agreement.
Net cash used in operating activities during the full year ended December 31, 2022 was 68.5 million, compared to
26.0 million for the same period in 2021.
Research and development costs for the
year ended December 31, 2022 were 50.9 million, compared to 42.2 million for the same period in 2021. Research
and development costs for the year end December 31, 2022 include costs related to the winding down of our ophthalmology programs,
including the clinical trials.
General and administrative costs for
the year ended December 31, 2022 were 18.7 million, compared to 17.4 million for the same period in 2021.
Net loss for the year ended December 31,
2022 was 64.9 million or 0.91 per diluted share, compared to 61.7 million, or 0.96 per diluted share for
the same period ended December 31, 2021. For further financial information for the period ended December 31, 2022,
please refer to our 2022 Annual Report on Form 20-F and our Statutory Annual Report which will be available on our website, www.
proqr.com under Financials and Filings.
Company will host a virtual R&D event today, March 29, 2023 from 10:00 am until 12:30 pm EDT, including an Analyst Q&A session
with members of the ProQR Management Team. To register for the virtual R&D event, please click here.
A live webcast of the event will be available under "Events" in the "Investors & Media" section of ProQR's
website at www.proqr.com/events. The archived webcast will be available for replay for approximately 30 days following
a next-generation RNA base editing technology called Axiomer , which could potentially yield a new class of medicines
for diverse types of diseases. Axiomer "Editing Oligonucleotides", or EONs, mediate single nucleotide changes
to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase
Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to
an Inosine (I) in the RNA - an Inosine is translated as a Guanosine (G) - correcting an RNA with a disease-causing
mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that
helps prevent or treat disease.
ProQR Therapeutics is
dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology
called Axiomer , which uses a cell's own editing machinery called ADAR to make specific single nucleotide edits
in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent
diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients
and loved ones in mind.
more about ProQR at www.proqr.com.