Full Press Release Details
ProQR Announces Axiomer RNA Editing Licensing and
Research Collaboration with Lilly
LEIDEN, Netherlands & CAMBRIDGE, Mass., September 8,
2021 -ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), today announced a global licensing and research collaboration with Eli Lilly
and Company (Lilly) focused on the discovery, development, and commercialization of potential new medicines for genetic disorders in
the liver and nervous system. The companies will use ProQR's proprietary Axiomer RNA editing platform to progress
new drug targets toward clinical development and commercialization.
ProQR's unique Axiomer
platform technology enables the editing of single nucleotides in RNA in a highly targeted and specific manner. The technology
is based on editing oligonucleotides, or EONs, designed to recruit endogenous ADAR enzymes (Adenosine Deaminases Acting on RNA) to a
selected target adenosine in a disease associated RNA. ADAR then induces the conversion of the target adenosine (A) into inosine
(I). The conversion from A to I is effectively an A to G change, as inosine in RNA is interpreted as a guanosine (G). This technology
could be applied to potentially reverse the more than 20,000 G to A mutations in the human population that are known to cause disease.
"RNA editing is an exciting emerging technology,
which allows transient, reversible editing, which in some indications may be an extremely attractive therapeutic approach" said Andrew
C. Adams, Ph.D., vice president for New Therapeutic Modalities at Lilly. "Through this collaboration with ProQR, we hope to utilize
this technology to unlock novel treatments to improve the lives of patients across a spectrum of diseases."
"This partnership with Lilly, a leader in RNA
therapeutics, is an important validation of our Axiomer RNA editing platform, and
expands the application of our technology beyond our core therapeutic area focus of genetic eye disease, to potentially benefit patients
with metabolic and nervous system disorders." said Daniel A. de Boer, Founder and CEO of ProQR. "Additionally, this partnership
further strengthens our financial position."
"Building from our deep scientific
expertise in RNA therapies and specifically oligonucleotides, our Axiomer RNA base editing platform is uniquely positioned to target
a wide range of diseases in a highly specific manner," said Gerard Platenburg, Chief Innovation Officer of ProQR. "Our
approach uses the cell's own editing machinery to make specific single nucleotide edits in RNA to reverse a mutation. With
broad applicability, and a leading patent portfolio in the ADAR editing space, this platform represents an important strategic
opportunity and has significant potential to target diseases otherwise thought untreatable."
The companies will collaborate to develop up to five
targets. Under the terms of the agreement, ProQR will receive $50 million consisting of an upfront payment of $20 million, as well as
an equity investment in its ordinary shares of $30 million. ProQR is also eligible to receive up to approximately $1.25 billion for development,
regulatory and commercialization milestones, as well as tiered royalties of up to mid-single digit percentage on product sales.
ProQR is pioneering a next-generation RNA editing
technology called Axiomer , which could potentially yield a new class of medicines
for genetic diseases. Axiomer "Editing Oligonucleotides", or EONs, induce single nucleotide changes to RNA in a highly specific
and targeted way using the cell's own molecular machinery. The Axiomer EONs
are designed to recruit an endogenously expressed RNA editing system called ADAR, which can direct the change of an Adenosine (A) to
an Inosine (I) in the RNA - an Inosine is translated as a Guanosine (G). There are over 20,000 G to A mutations known to cause
human disease that could be reversed using this novel technology.
ProQR Therapeutics is dedicated to changing
lives through the creation of transformative RNA therapies for the treatment of genetic eye diseases, with a focus on inherited retinal
diseases such as Leber congenital amaurosis 10, Usher syndrome and retinitis pigmentosa. 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 at www.proqr.com.
Forward Looking Statements for ProQR
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
forward-looking statements include, but are not limited to, statements regarding the collaboration with Eli Lilly and Company
("Lilly") and the intended benefits thereof, including the upfront payment, equity investment, and milestone and royalty
payments from commercial product sales, if any, from the products covered by the collaboration, as well as the potential of our
technologies and product candidates. 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 by the COVID-19 pandemic; the likelihood of our clinical programs being 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 data to alter initial and preliminary results of
early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances
that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and
realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of,
inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new
data are generated in research and development; and general business, operational, financial and accounting risks, and risks related
to litigation and disputes with third parties. 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.
For ProQR Therapeutics N.V.
ProQR Therapeutics N.V.
Cherilyn Cecchini, MD
LifeSci Communications