DURHAM, N.C., July 29, 2020 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that it has acquired exclusive rights to a gene therapy targeting GUCY2D-associated Leber congenital amaurosis (LCA1), a genetic eye disease that affects the retina and is a leading cause of blindness in children, from Sanofi, which originally licensed it from the University of Florida. The therapy was created in the laboratory of Atsena Founder and Chief Scientific Officer Shannon Boye, Ph.D., and Founder and Chief Technology Officer Sanford Boye, M.Sc., at the University of Florida.
“We are thrilled that our gene therapy for LCA1 is coming home to Atsena and that we will have the opportunity to further its development,” said Shannon Boye. “Atsena was founded to advance treatments for inherited retinal diseases and believes in centering patients’ perspectives and needs in all we do. We are honored to continue to work with LCA1 patients and their families as we strive to treat this debilitating disease.”
LCA is the most common cause of blindness in children, impacting two to three per 100,000. LCA1 is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this inherited retinal disease.
Atsena has an ongoing Phase I/II clinical trial evaluating this gene therapy in LCA1 patients. The second cohort in the trial is expected to be dosed in the fall of 2020.
“Atsena’s gene therapy has the potential to be a major advance in treating blindness in both children and adults affected by this inherited retinal disease,” said Benjamin Yerxa, Ph.D., Chief Executive Officer of the Foundation Fighting Blindness and Atsena board director. “The foundation was instrumental in supporting proof of concept studies in the founders’ labs over the last 15 years. Now, via investment in Atsena through our Retinal Degeneration (RD) Fund, we are excited to support this potential breakthrough treatment for LCA1.”
Atsena closed a Series 1 funding of $8.15 million in April 2020, led by founding investors Hatteras Venture Partners and the Foundation Fighting Blindness’ RD Fund with participation by Osage University Partners, PBM Capital and the University of Florida. Patrick Ritschel, M.B.A., co-founder and former President of gene therapy company StrideBio, serves as Atsena’s Chief Executive Officer.
“Atsena is pleased to have the support of an enthusiastic investor base that shares our dedication to bringing the life-changing power of genetic medicine to patients living with LCA1 and other forms of blindness,” said Ritschel. “We look forward to working closely with our investors and patients as we continue to grow, and expect to announce additional milestones later this year.”
About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company, focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness. The company has an ongoing Phase I/II clinical trial evaluating a potential therapy for one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome the hurdles presented by inherited retinal disease, and its approach is guided by the specific needs of each patient condition. Founded by pioneers in ocular gene therapy, Atsena has a licensing, research and manufacturing collaboration with the University of Florida and is headquartered in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.
PARIS--(BUSINESS WIRE)--SparingVision, a biotechnology company specializing in the research and development of innovative therapies for treating hereditary retinal degenerative diseases such as retinitis pigmentosa, has today announced that it has been awarded non-dilutive funding of €2.5 million, having submitted a winning proposal for the European EIC Accelerator (SME instrument Phase 2) program.
This funding will help speed up SparingVision's clinical and regulatory development. The company is currently producing the clinical batches needed to initiate the first clinical trials in Europe and the United States in 2020. The PHENOROD (NCT03975543) retrospective natural history study of the disease has already taken place and the results will be published soon. The PHENOROD 2 prospective study will begin in the Fall of 2019.
“We are delighted to have won this European EIC Accelerator program and would like to offer our sincere thanks to all bodies of the EU Horizon 2020 program. This funding reflects the innovative nature of the gene-independent approach of the gene therapy developed by SparingVision for the treatment of retinitis pigmentosa, rare hereditary retinal degeneration that leads to blindness. It will enable us to speed up our development and position the company as a new stakeholder in the treatment of retinitis pigmentosa, and meet an unmet public health need”, explains Florence Allouche, SparingVision President. “We are particularly pleased as this is the first time SparingVision has submitted a proposal for this highly selective European program, as we are currently preparing for new fundraising action. EU applications require specific expertise and experience, so we would like to thank retina specialists in several leading centers throughout Europe, Israel and the United States as well as the Retina International patient organization and the Foundation Fighting Blindness for their support, and the European support teams of Bpifrance and Efficient Innovation for their methodology, assistance and, in particular, for sharing our desire to succeed.”
EIC Accelerator (SME Instrument) is a European public program that funds risk innovation in disruptive small businesses that have significant growth potential and global ambitions. EIC Accelerator is part of Horizon 2020 – the EU's €80 billion funding program for Research and Innovation for 2014-2020. For this April 2019 session, the success rate is 4.44% at the European level. Out of 136 proposals submitted by French companies, only 4 have been selected, and this includes SparingVision, the only biotech company. According to Venture Radar, 25% of companies funded under EIC Accelerator are among the fastest-growing European firms (10%).
SparingVision is a biotechnology company focused on the discovery and development of innovative therapies for the treatment of blinding inherited retinal diseases. SparingVision is developing SPVN06, a gene-independent drug candidate to treat retinitis pigmentosa, the most common inherited retinal degeneration. There is currently no treatment to treat all genetic forms of this rare retinal disease that leads to blindness and affects 40,000 people in France and nearly 2 million worldwide. SparingVision is a spin-off of the Paris Vision Institute. Bpifrance, Foundation Fighting Blindness (US) and Voir & Entendre Foundation invested €15.5 million in the company. SparingVision was laureate and Grand Prize of i-Lab 2017, the National Contest for the Creation of Innovative Companies, and is part of the first selection of companies of Hub Heath Tech launched by Bpifrance in December 2017. Florence Allouche, President of SparingVision, has won the Mercures Entrepreneurs Prize and the Women Trajectory’s award from HEC Paris and was elected "Woman of the Year 2017” by the financial magazine La Tribune.
Nacuity Pharmaceuticals Announces Initiation of SLO-RP (Safety and Efficacy of NPI-001 Tablets versus PLacebO for Treatment of Retinitis Pigmentosa Associated with Usher Syndrome) Phase 1/2 Clinical Trial
NPI-001 Being Evaluated for the Treatment of Retinitis Pigmentosa in Patients with Usher Syndrome
FORT WORTH, TX, May 18, 2020 – Nacuity Pharmaceuticals, Inc., a clinical stage biotech company focused on the treatment of ocular diseases involving oxidative stress, today announced the initiation of a Phase 1/2 study of NPI-001 tablets in patients with retinitis pigmentosa (RP) associated with Usher syndrome (USH). The study, named SLO-RP, is designed to demonstrate greater retinal sensitivity in RP/USH patients treated with NPI-001 Tablets versus placebo. Previously, NPI-001 was evaluated in a Phase 1 study conducted in healthy volunteers, demonstrating its safety profile. The SLO-RP study will enroll patients diagnosed with RP associated with USH who are early in disease progression.
Preclinical studies indicate that oral NPI-001 protects photoreceptor cones in the eye from oxidative damage, which may preserve visual function.1 Furthermore, NPI-001 and related small molecules have been shown to be potent antioxidants in several preclinical models that may lead to additional indications.2 “Initiation of the SLO-RP trial is an important milestone in our efforts to understand the potential of this treatment for RP and a prelude to treating other conditions of the eye involving oxidative stress,” said G. Michael Wall, PhD, senior vice-president and chief scientific officer at Nacuity.
Nacuity Pharmaceuticals has partnered with the Foundation Fighting Blindness on a variety of initiatives related to the development of NPI-001 including funding through the Foundation’s venture arm the RD Fund, trial design, awareness and recruitment, and additional strategic guidance. “We are happy on behalf of the many patients around the globe who are affected by RP to see this promising treatment reach the clinic.” said Dr. Ben Yerxa, chief executive officer of Foundation Fighting Blindness, “We remain committed to seeing this potential treatment through to the next milestone.”
About the SLO-RP Trial
The randomized, placebo-controlled, multicenter, double-masked, dose escalation trial is designed to assess the clinical safety, tolerability and efficacy of NPI-001 Tablets versus placebo in patients with USH (NCT04355689). The Australian study will target enrollment of at least 48 male and female USH patients, ages 18 years and older, who will be followed for 2 years. The goals are to assess the safety of chronic dosing up to 500mg/day and generate clinical proof of concept for the treatment of RP. The key clinical endpoint is change from baseline in retinal sensitivity of patients treated with NPI-001 versus placebo.
RP refers to a group of inherited diseases causing retinal degeneration and vision loss that worsen over time including USH, Leber congenital amaurosis, rod-cone disease, Bardet-Biedl syndrome, Refsumdisease, and others. The vision loss in USH is due to RP, presenting in the same manner. By showing a slowing of disease progression in the SLO-RP study, Nacuity aims to begin to address RP as a whole.
The National Eye Institute (NEI) estimates that approximately 100,000 people in the United States have RP. About 1.5 million people are afflicted worldwide. Night blindness is one of the earliest and most frequent symptoms. RP is a progressive disorder typically diagnosed in adolescents and young adults. The rate of progression and degree of visual loss varies by case, with the majority of patients legally blind by age 40. The only FDA-approved treatment for RP is LUXTURNA®, a gene therapy applicable for about 1% of RP patients (rpe65). (Trademarks are property of their owners.)
Nacuity Pharmaceuticals, Inc. (https://www.nacuity.com) is a clinical stage biotech company focused on advancing treatments for ocular conditions involving oxidative stress. Nacuity is funded by Foundation Fighting Blindness grants and direct investment, as well as private investors.
Foundation Fighting Blindness (https://www.fightingblindness.org)
Established in 1971, the Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $760 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. Visit FightingBlindness.org for more information.
Forward Looking Statements
This announcement includes "forward-looking statements" within the meaning the federal securities laws. All statements other than statements of historical facts contained in this press release, including statements regarding our anticipated future clinical and regulatory events, future financial position, business strategy and plans and objectives of management for future operations, are forward-looking statements. Forward looking statements are generally delivered in the future tense and/or are preceded by words such as "may," "will," "should," "forecast,“ "projected," "could," "expect," "suggest,”
"believe," "estimate," "anticipate," "intend," "plan,“ or similar words, or the negatives of such terms or other variations on such terms or comparable terminology. Such forward-looking statements include, without limitation, statements regarding the potential future commercialization of our product candidates, the anticipated start dates, durations and completion dates, as well as the potential future results of our future clinical trials, the anticipated designs of our future clinical trials, anticipated future regulatory submissions and events, our anticipated future cash position and future events under our current and potential future collaborations.
1Dong A, Stevens R, Hackett S, Campochiaro PA. Compared with N-acetylcysteine (NAC), N-Acetylcysteine Amide (NACA) Provides Increased Protection of Cone Function in a Model of Retinitis Pigmentosa. Investigative Ophthalmology & Visual Science April 2014, Vol.55, 1736.
2Nacuity data on file.
LEIDEN, Netherlands and CAMBRIDGE, Mass., March 11, 2019 (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 the first patient dosed in the Phase 1/2 STELLAR clinical trial for QR-421a in patients with Usher syndrome type 2 or non-syndromic retinitis pigmentosa (RP). Interim data from the study are expected to be announced mid-2019.
"There are no effective treatments for most inherited retinal diseases, including Usher syndrome, and blindness often results,” said David G. Birch, Ph.D., Principal Investigator of STELLAR and ScientificDirector of the Retina Foundation of the Southwest in Dallas, Texas. “The STELLAR study is one of the first studies of its kind exploring the impact of ProQR’s RNA therapies on patients with Usher syndrome due to an Exon 13 mutation. The STELLAR trial will explore whether QR-421a (ProQR’s RNA therapy) can slow disease progression or even reverse it. Treatments such as this, that target the underlying cause of a disorder, have the potential to give new hope to patients and their families that life-changing therapies could be available in the near future.”
“Usher syndrome is a devastating disease, so we are pleased to advance QR-421a into the clinic with the goal to make a difference for these patients, similar to what we have observed with early but promising data for sepofarsen in patients with LCA10,” said Daniel A. de Boer, chief executive officer of ProQR. “We are committed to rapidly advancing our promising RNA therapies for inherited retinal diseases and we believe our platform of generating targeted RNA therapies with long retinal half-lives and the ability to reach both central and peripheral retina, we will be able to target many of these diseases in the coming years.”
Usher syndrome is the leading cause of combined deafness and blindness. Exon 13 mutations in the USH2A gene targeted by QR-421a cause vision loss in approximately 16,000 individuals in the Western world.
About the Phase 1/2 “STELLAR” trial
STELLAR, or PQ-421a-001, is a first-in-human study that will initially include approximately 18 adults who have vision loss due to mutations in exon 13 of the USH2A gene and will be conducted at about seven expert sites in North America and Europe. It will be a double-masked, randomized study exploring several dose levels and a control (sham injection), given as a single intravitreal injection of QR-421a into one eye. The first patient at each dose level will be dosed in an open-label manner. The objectives of the trial will include evaluation of safety, tolerability, pharmacokinetics and efficacy, as measured by stopping progression or improvement of visual function and retinal structure through ophthalmic endpoints such as visual field, visual acuity and optical coherence tomography. Changes in quality of life in the trial subjects will also be evaluated.
Preliminary data from the first-in-human study are expected in mid-2019. Patients completing this trial will be able to participate in an extension study if eligible. Results from the single dose trial will inform the next stage that will potentially include a seamless adaptive, multi-dose, controlled trial with projected readout in 2021.
QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional Usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation from the FDA.
About Usher Syndrome
Usher syndrome is the leading cause of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. Usher syndrome type 2 is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene. To date, there are no approved treatments or products in clinical development that treat the vision loss associated with Usher syndrome type 2.
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, Usher syndrome type 2 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
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 QR-421a and its clinical development and therapeutic potential, including commencement of the STELLAR trial, trial design and timing of results from this trial. 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, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, regulatory review or approval process, 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 future, except as required by law.
ProQR Therapeutics N.V.
Vice President of Investor Relations and Corporate Communications
T: +1 202 360 4855
Source: ProQR Therapeutics N.V.
Today, the Foundation Fighting Blindness announced the creation of the first-ever Retinal Degeneration Fund (RD Fund), a wholly owned, 501(c)(3) not-for-profit subsidiary focused on making venture philanthropy investments to further the Foundation’s mission: To provide preventions, treatments and cures for inherited retinal diseases and age-related macular degeneration as fast as possible. The RD Fund, which now has more than $70 million in initial funding, will invest in companies with projects nearing clinical testing.
“We are excited to initiate a focused and concentrated effort on our mission-related investments with the creation of the RD Fund,” said Benjamin Yerxa, PhD, CEO, RD Fund and Foundation Fighting Blindness. “With more than $70 million in assets to put to work, the RD Fund will support a portfolio of projects that advance our organization’s mission.”
David Brint, chairman of Foundation Fighting Blindness added, “We are adapting to a rapidly changing environment, and the RD Fund is part of our strategy. Translational R&D is increasing in cost, and our supporters want to see a disciplined and professional approach to maximally leverage these hard-earned funds to provide clear, measurable progress, as well as the potential for a sustainable financial future. It is imperative that the most promising research has the best opportunity to reach patients in need.”
The new RD Fund is also the vehicle for Foundation Fighting Blindness’ recent mission-related investments. The Foundation made its first such investment in SparingVision in 2016, through the Foundation’s subsidiary, the Clinical Research Institute (CRI). Since that time, through CRI, the Foundation has co-funded projects with its partners, Nacuity Pharmaceuticals and ProQR. Recognizing the increasing need for more investment to speed translation of scientific discoveries into meaningful clinical results, Foundation Fighting Blindness is transforming its CRI subsidiary into the RD Fund, which will now focus purely on mission-related investments.
Clinical research activities formerly overseen by CRI will now be managed by a new Foundation Fighting Blindness subcommittee chaired by Dr. Frederick Ferris III, former clinical director of the National Eye Institute.
“We would like to thank the Foundation’s Clinical Research Institute board members for their many years of service and dedication to the success of the institute,” said Dr. Morton Goldberg, chairman of the former CRI, who will continue his service on the Foundation’s Research Oversight Committee. “The ability to focus separately on clinical research, now under Rick
Ferris’ leadership, and to manage the translational investments through the RD Fund are going to be powerful forces moving the field forward.”
An independent board of directors will oversee the RD Fund, with representatives from Foundation Fighting Blindness and several at-large members who bring significant outside expertise to the team. Warren Thaler, Chairman of the RD Fund Board, said, “I’m excited to use my investment experience to further the foundation’s mission, and am humbled to work with a world-class group of directors to oversee the RD Fund.” In addition to Mr. Thaler, the initial board members include: David Brint, Eugene de Juan, MD, Jacque Duncan, MD, Adrienne Graves, PhD, Kelly Lisbakken, Jason Morris, and Jonathan Steinberg, MD.
The RD Fund will be managed by Benjamin Yerxa, PhD, CEO, Jason Menzo, COO, and Russell Kelley, PhD, MBA, vice president of investments and alliances, along with additional support from the Foundation staff and outside consultants. The $70 million of initial assets include the first three investments made by the former CRI. The RD Fund will remain open to new donations for several months. All proceeds from investments will be used to fund the Foundation’s research mission. In parallel, the Foundation is dedicated to providing continued, robust funding of the best academic laboratories in the inherited retinal disease field to provide a pipeline of future therapies.
“Although $70 million sounds like a lot of money, we have to remember that a single R&D program can cost tens of millions of dollars or more,” said Paul Manning, whose family recently completed a Gund Challenge pledge of nearly $12 million directed to the RD Fund. “The Manning family is truly excited to see the Foundation Fighting Blindness move in this direction, and we invite others to join us in financially supporting this effort.”
ABOUT THE RETINAL DEGENERATION FUND
The Retinal Degeneration Fund (RD Fund) is a 501(c)(3) not-for-profit subsidiary of the Foundation Fighting Blindness. Launched in 2018 with a $70 million initial investment, the RD Fund focuses on making venture philanthropy investments in companies with projects nearing clinical testing.
ABOUT FOUNDATION FIGHTING BLINDNESS
The Foundation Fighting Blindness was established in 1971. It has since raised more than $750 million for research aimed at preventing, treating and curing blindness caused by retinal degenerative diseases. In excess of 10 million Americans, and millions more worldwide, have experienced or are at risk of experiencing vision loss due to retinal degeneration. Through its support of focused and innovative science, the Foundation drives the research that has and will continue to improve the lives of people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases. Learn more athttp://www.blindness.org/.
For more information about the RD Fund and major gift contributions, please contact email@example.com.
COLUMBIA, Md. and LEIDEN, the Netherlands, Feb. 12, 2018 (GLOBE NEWSWIRE) -- Foundation Fighting Blindness and ProQR Therapeutics N.V. (NASDAQ:PRQR), today announced that they have entered into a partnership to develop QR-421a for Usher syndrome 2A caused by an exon 13 mutation of the causative USH2A gene. Under the agreement, Foundation Fighting Blindness will provide up to $7.5 million in funding to ProQR for the preclinical and clinical development of QR-421a, which is expected to advance towards the clinic in 2018, and safety and efficacy results from the Phase 1/2 trial in Usher syndrome patients are expected in 2019.
Usher syndrome is a devastating genetic disease in which patients first develop hearing loss and then progressive vision loss, thereby threatening their independence and quality of life. Currently there is no treatment for the ophthalmic manifestation of Usher syndrome type 2A. QR-421a is a first-in-class RNA oligonucleotide that is being developed for the treatment of vision loss associated with the disease. QR-421a is designed to modify the RNA such that functional usherin protein is produced in the retina with the goal of stopping the progression of the disease and potentially gaining peripheral vision. ProQR in-licensed the technology underlying QR-421a from Radboud University Medical Center in the Netherlands, where it was invented by lead investigator Dr. Erwin van Wyck.
Foundation Fighting Blindness’ Clinical Research Institute (FFB-CRI) has also launched a natural history study in 120 people with USH2A mutations. The study — known as RUSH2A (“R” stands for “rate of progression”) — was launched in 2017 and is being conducted at about 20 clinical sites around the world. RUSH2A investigators will use a variety of technologies to monitor changes in vision and retinal structure to document and analyze disease progression. Knowledge and data obtained from this trial are intended to provide a better understanding of how USH2A mutations affect the severity and progression of vision loss and help to inform the development of QR-421a.
“Teaming with corporate partners to help promising therapies move through preclinical and clinical development is central to FFB’s strategy so we are very pleased to enter into this partnership with ProQR,” said Benjamin R. Yerxa, PhD, CEO at Foundation Fighting Blindness. “The fact that there are currently no available treatments for Usher syndrome type 2A makes this work that much more exciting and critical.”
QR-421a for Usher syndrome is the second program in ProQR’s growing ophthalmology pipeline scheduled to enter clinical trials. The lead program in the ophthalmology pipeline, QR-110, is currently in a Phase 1/2 safety and efficacy trial in adult and pediatric patients with Leber’s congenital amaurosis 10, due to the p.Cys998X mutation in the CEP290 gene. This pipeline also contains several other molecules for genetic eye diseases, including QR-411 for Usher syndrome type 2A due to the PE-40 mutation, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.
“We are excited to team up with the Foundation Fighting Blindness to develop QR-421a for patients that suffer from Usher syndrome due to exon 13 mutations," said Daniel A. de Boer, CEO of ProQR. “They are the leading private funder of retinal disease research with a very patient centric approach which is a core pillar of our strategy. Through this partnership with the Foundation we plan to gain access to important know-how to develop programs in retinal diseases. We expect that the additional funding will allow us to rapidly advance this novel therapy for this orphan disease with a severe unmet need.”
About Foundation Fighting Blindness
The Foundation Fighting Blindness was established in 1971 and has raised more than $725 million for research on preventing, treating and curing blindness caused by inherited retinal diseases. In excess of 10 million Americans, and millions more worldwide, experience or are at risk for vision loss due to retinal degenerations. Through its support of focused and innovative science, and by teaming with industry, the Foundation drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases.
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome 2A due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional usherin protein that causes the disease. QR-421a is designed to repair the genetic defect in the RNA in the eye, such that it leads to the expression of a shortened but functional protein, thereby modifying the underlying disease. QR-421a has received orphan drug designation from the U.S. Food and Drug Administration.
About Usher Syndrome Type 2A
Usher syndrome is the leading cause of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. Usher syndrome type 2A is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene. To date, there are no treatments approved or products in clinical development that treat the vision loss associated with Usher syndrome type 2A.
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 “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 our collaboration with Foundation Fighting Blindness, including statements regarding the expected funding and benefits of such collaboration, our product candidates QR-110, QRX-1011, QRX-504 and QR-421a, including their clinical development and therapeutic potential, including future development plans. 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, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, that a Fast Track designation by the FDA may not actually lead to a faster development, regulatory review or approval process, and that we may not be able to realize the potential benefits of orphan drug exclusivity, 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 future, except as required by law.
ProQR Therapeutics N.V.:
Investor and Media Contact:
T: +1 858 245 3983
Sources: ProQR Therapeutics and Foundation Fighting Blindness
Source: ProQR Therapeutics N.V.
The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) has announced an investment of up to $7.5 million to advance a promising, emerging drug treatment for retinitis pigmentosa (RP) into and through a Phase II clinical trial. Known as N-acetylcysteine-amide (NACA), the molecule is designed to slow vision loss by protecting retinal cells from oxidative stress. Oxidative stress is a process that accelerates and exacerbates degeneration in many inherited retinal conditions. In several in vitro and in vivo models, including previous FFB-funded lab studies of rodent models at Johns Hopkins University, NACA slowed retinal degeneration.
Nacuity Pharmaceuticals, Inc., a start-up company in Fort Worth, Texas, owns the rights to NACA for ophthalmology and will be developing the drug with support from FFB-CRI. As part of its agreement with Nacuity, upon commercialization of NACA, FFB-CRI will be entitled to royalty payments from future NACA sales.
“Based on the knowledge we’ve gained in lab studies, we are excited about NACA’s potential for saving vision,” said Patricia Zilliox, Ph.D., chief drug development officer at FFB-CRI. “Oxidative stress causes cell degeneration and vision loss in virtually all forms of RP, so we are hopeful that NACA, with its anti-oxidative properties, can benefit most people with RP, regardless of the gene mutation causing their disease.”
NACA is derived from N-acetylcysteine (NAC), a drug approved by the U.S. Food & Drug Administration (FDA) for the treatment of acetaminophen overdose by lessening hepatotoxicity. The benefits of NACA over NAC are believed to include greater lipophilicity and tissue penetration therefore increasing anti-oxidative properties in the retina.
“We are excited to make a significant investment in NACA, because the drug has an opportunity to help a substantial number of people with RP, a blinding retinal disease affecting 100,000 people in the U.S., and more than 2 million worldwide,” said William T. Schmidt, chief executive officer of FFB. “The fact that NACA is derived from an FDA-approved drug also boosts our confidence that it can make it through the clinical development process and out to the people who need it.”
“We are pleased and excited to have the support of FFB in our quest for an effective treatment for patients with RP.” said Halden Conner, President of Nacuity. “The review of, and input to, our plans by their independent, renowned ophthalmological experts will markedly increase our odds of success in bringing this treatment to the RP community.”
“Drug substance process manufacturing, drug product development and toxicology studies are planned for 2017 to facilitate an investigational new drug application (IND) and initiation of a Phase I Clinical Safety Program for this new chemical entity,” Nacuity’s Vice-President of Research and Development, G. Michael Wall, Ph.D., stated. Nacuity hopes to launch a Phase II clinical trial for NACA in early 2018.
The Foundation Fighting Blindness was established in 1971. It has since raised more than $700 million for research aimed at preventing, treating and curing blindness caused by retinal degenerative diseases. In excess of 10 million Americans, and millions more worldwide, have vision loss due to retinal degeneration. Through its support of focused and innovative science, the Foundation drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases.
Nacuity Pharmaceuticals, Inc. was formed in May 2016 to focus on bringing a potential treatment with NACA to patients with RP guided by the groundbreaking work by Peter Campochiaro, MD, at the Wilmer Institute of Johns Hopkins University.
The development of a vision-saving treatment for people with retinitis pigmentosa (RP) is getting a major boost thanks to the formation of the French biotech SparingVision to move it into a clinical trial and out to the international marketplace.
A spin-off of the Institut de la Vision, SparingVision was established to clinically develop and commercialize a protein known as rod-derived cone-viability factor (RdCVF). The emerging therapy performed well in several previous lab studies funded by the Foundation Fighting Blindness. SparingVision’s goal is to launch a clinical trial for the protein in 2019.
The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI), Bpifrance — Biotechnology Acceleration Fund and Innovative Biotherapies and Rare Diseases Funds created by the French national program (BPI), and the Fondation Voir et Entendre (FVE) have announced a total of €15.5 million in tranche funding for the development of RdCVF. FFB-CRI and BPI are each investing €7 million with FVE providing €1.5 million.
SparingVision also received a €300,000 award known as the Honor Prize from the French Ministry of Research. The award is given to new, innovative companies in France competing in a national contest.
RdCVF is a naturally occurring protein in the retina identified by SparingVision co-founders José Sahel, MD, and Thierry Léveillard, PhD, at the Institut de la Vision. The scientists demonstrated in laboratory studies that RdCVF prevented or slowed the degeneration of cones, the cells in the retina that provide central and color vision and enable people to read, drive, and recognize faces. RdCVF is naturally secreted by rods, the retinal cells that provide night and peripheral vision.
RP is a genetic condition affecting about two million people worldwide. The retinal disease is usually diagnosed in childhood, progressively leading to legal or total blindness in adulthood. RP initially affects rods. The progressive loss of rods leads to loss of cones. There are currently no therapies for RP.
“Saving retinal cone cells is critical for preserving vision for people with genetic retinal diseases,” says Dr. Sahel.
“After several years of investigation, we understand the mechanism of action for RdCVF and have demonstrated its strong efficacy in several lab studies,” says Dr. Léveillard.
“SparingVision’s emerging therapy has the potential to save the vision of millions. I am delighted by our partners’ investment to get RdCVF out to the people who desperately need it,” says Florence Allouche Ghrenassia, PharmD, president at SparingVision. She brings 16 years of experience at Assistance Publique Hôopitaux de Paris as TTO Director in advancing innovative early stage therapies into the clinic to her new role.
FFB-CRI provided much of the earlier research funding to develop RdCVF. “We have been excited about this therapy’s potential for saving vision and therefore committed significant resources to boost its development,” says Patricia Zilliox, PhD, FFB-CRI’s chief drug development officer. “The establishment of SparingVision and the investment by our partners are essential to getting the treatment into the marketplace. We are pleased to be a part of this translational process.”