Full Press Release Details
Announces Year End 2023 Operating and Financial Results
LEIDEN, Netherlands & CAMBRIDGE, Mass., March 13, 2024
- 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 reported its financial and operating results for the
year ended December 31, 2023, and provided a business update.
"Over the past year as we exclusively focused our strategy on
RNA editing, ProQR made important progress with Axiomer, our ADAR-mediated RNA editing technology platform," said Daniel A. de Boer,
Chief Executive Officer of ProQR. "We announced our initial pipeline programs, AX-0810 and AX-1412, targeting NTCP and B4GALT1,
respectively, and presented preclinical platform data across a variety of targets. Our platform has demonstrated robust editing, including
reporting up to 70% editing of ACTB in the liver of non-human primates, as well as functional protein data with the liver target ANGPTL3
in mice. Additionally, we further strengthened our leading global IP estate for ADAR-mediated RNA editing, which was upheld when challenged
by multiple parties in various jurisdictions. We continued to execute and build on the successes achieved during the first two years of
our collaboration with Eli Lilly, and formed a new partnership with the Rett Syndrome Research Trust. We also completed the divestment
of late-stage ophthalmic assets, sepofarsen and ultevursen, to Laboratoires Th a who will continue the development of these therapies
for patients, as ProQR continues to focus exclusively on advancing our RNA editing platform and pipeline."
De Boer continued, "In 2024, we anticipate sharing in vitro,
in vivo, and translational data for our initial pipeline programs using Axiomer, and remain on track to enter the clinic in late 2024/early
2025. Along with our extensive body of preclinical proof of concept data for the platform, partnership with Eli Lilly, leading IP position,
and strong cash position providing runway into mid-2026, we believe that ProQR is well positioned to continue to execute on our strategy
and advance our mission of changing lives through transformative RNA therapies."
Anticipated Upcoming
Year End 2023 Financial Highlights
At December 31, 2023, ProQR held cash and cash equivalents
of 118.9 million, compared to 94.8 million at December 31, 2022. Net cash generated by operating activities during
the full year ended December 31, 2023 was 21.5 million, compared to 68.5 million net cash used in operating
activities for the same period in 2022. The Company experienced a net positive cash flow from operating activities in 2023 primarily due
to the receipt of the Lilly up-front payment of $60 million in February 2023.
Research and development costs for the year ended December 31,
2023 were 25.1 million, compared to 50.9 million for the same period in 2022. Research and development costs for the
year end December 31, 2022 included costs related to the winding down of ophthalmology programs, including the clinical trials.
General and administrative costs for the year ended December 31,
2023 were 16.2 million, compared to 18.7 million for the same period in 2022.
Net loss for the year ended December 31, 2023 was 27.7
million or 0.35 per diluted share, compared to 64.2 million, or 0.90 per diluted share for the same period ended December 31,
2022. For further financial information for the period ended December 31, 2023, please refer to our 2023 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.
ProQR is pioneering 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.
About Biliary Atresia (BA) and Primary
Sclerosing Cholangitis (PSC)
Cholestatic disorders refer to a group
of diseases presenting excessive and toxic buildup of bile acids in the liver due to bile ducts dysfunction. This leads to liver damage
and a range of debilitating symptoms. Without treatment, liver damage can progress through various stages, ultimately leading to liver
failure and elevated risk of liver malignancy, affecting life expectancy. Cholestatic diseases remain leading causes of liver transplantation.
There are no approved therapies for primary sclerosing cholangitis (PSC) for adults and biliary atresia (BA) for pediatrics It is estimated
that 80,000 and 20,000 individuals have PSC and BA, respectively, in North America and in Europe.
About AX-0810 targeting NTCP
The majority of the bile acids present
in the liver cells originate from the enterohepatic reuptake cycle. The key transporter responsible for hepatic uptake of bile acids
from portal circulation is the sodium (Na+)-taurocholate cotransporting polypeptide (NTCP, SLC10A1 gene) expressed in the liver. AX-0810
is designed to introduce a loss of function variant in SLC10A1 RNA that has been found in human genetics to prevent re-uptake of bile
acids in liver via NTCP. Based on its mechanism of action, AX-0810 has the potential to become a disease modifying treatment for PSC
and BA primarily among other cholestatic diseases.
About Cardiovascular Diseases
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. CVDs represent the leading cause of disability and death in the world. Approximately 18 million people
die every year from CVDs representing one third of all the global deaths. Despite available lipid lowering therapies and hypertension
medications, the risk of CVDs is still projected to increase rapidly over the coming years.
About AX-1412 targeting B4GALT1
Gene-based analysis of rare beta-1,4-galactosyltransferase
1 (B4GALT1) missense variant (p.Asn352Ser) is known to lead to B4GALT1 protein loss of function and showed an association with
decreased coronary artery disease. These beneficial effects are mediated by hypo-galactosylation of the apolipoprotein B100 and fibrinogen,
known - independent - drivers of increased risk of CVDs. AX-1412 introduces a protective variant into B4GALT1 RNA
to address the remaining residual risk of developing cardiovascular diseases. ProQR intends to advance AX-1412 targeting B4GALT1 to early
clinical proof of concept stage, then would seek to partner this program.
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.
Learn more about ProQR
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 "continue," "anticipate," "believe," "could," "estimate," "expect,"
"goal," "intend," "look forward to", "may," "plan," "potential," "predict,"
"project," "should," "will," "would" and similar expressions. Such forward-looking statements
include, but are not limited to, statements regarding our business, preclinical model data, our initial pipeline targets and the
upcoming strategic priorities and milestones related thereto, our Axiomer platform, including the continued development
and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and our ability to expand
preclinical in vivo and in vitro data, the timing, progress and results of our preclinical studies and other development
activities, including the release of data related thereto, our patent estate, including our anticipated strength and our continued investment
in it, as well as the timing of our clinical development, the potential of our technologies and product candidates, the collaboration
with Lilly and the intended benefits thereof, and our financial position and cash-runway. 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. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies
and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed
or halted shortage and pressure on supply and logistics on the global market; the likelihood of our preclinical and clinical programs
being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment
of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to
supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future