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Stargazer Pharmaceuticals, Inc. Announces $57 Million Series A Financing and Initiation of a Phase 2a Clinical Study of STG-001 in Stargardt Disease Patients

Boston, MA; November 9, 2020

Stargazer Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of novel treatment options for rare eye diseases, announced it has completed a Series A financing totalling $57 million from leading investors Novo Holdings, venBio Partners, Canaan Partners and Pontifax to develop its proprietary compound STG-001 for treating Stargardt Disease.  Stargazer also announced the initiation of a Phase 2a study of STG-001 in Stargardt Disease patients.

This Series A financing enables Stargazer to advance development of STG-001 through a pivotal clinical efficacy study potentially leading to a new therapy for Stargardt Disease, a major cause of vision loss in children and young adults.  Stargazer is pleased to be collaborating with Foundation Fighting Blindness (FFB) for the development of STG-001. FFB also participated in this financing round via their investment vehicle, the Retinal Degeneration Fund.

Stargazer was launched by Novo Ventures, the venture arm of Novo Holdings, Gary Sternberg, MD, MBA, who serves as the company’s Chief Executive Officer, and Silvia Ragno, PhD, who serves as the company’s Chief Operating Officer, following licensing of STG-001 from Takeda Pharmaceuticals.

Concurrent with the financing, Richard Gaster, MD, PhD, a Partner at venBio Partners, and Wende Hutton, a General Partner at Canaan Partners, will join Stargazer’s Board of Directors.  They join Nanna Lüneborg, a Partner at Novo Ventures, and Iyona Rajkomar, a Partner at Pontifax.  Other Board members include Daniel J. Curran, MD, from Takeda Pharmaceuticals, John Wagner, MD, PhD, and Dr. Sternberg.

Stargardt Disease is a genetic orphan disease that leads to legal blindness.  It is the most common juvenile macular dystrophy affecting 1 in 8,000 to 1 in 10,000 individuals.  There are currently no therapies available for this disease.  The Phase 2a study of STG-001 is a multi-center study in the US of subjects with Stargardt Disease comparing 2 doses of STG-001 with regard to safety, pharmacokinetics and pharmacodynamics.*

Stargazer has successfully completed a Phase 1 single- and multiple-ascending dose clinical study of STG-001 in healthy volunteers and is planning to initiate a pivotal efficacy study in 2021.

STG-001 has received Orphan Drug Designation in both the US (Food and Drug Administration) and EU (European Medicines Agency).

“This financing from leading investors, as well as the support from FFB, is a testament to the potential of our lead compound, STG-001.  Initiation of the Phase 2a trial in Stargardt Disease patients is an important milestone for us and we look forward to further development of STG-001 for the benefit of Stargardt Disease patients,” said Dr. Gary Sternberg. 

“We are very excited to work with Stargazer to develop a novel and potentially best in class therapy for the treatment of Stargardt Disease, a disease with a high unmet medical need,” said Nanna Lüneborg of Novo Ventures.

About STG-001

The visual cycle is fuelled by uptake of Vitamin A (retinol) from the circulation that is transported by Retinol Binding Protein 4 (RBP4), a key protein that binds retinol in the systemic circulation and delivers it to the eye.

STG-001 is an indirect visual cycle modulator. By reducing plasma concentrations of RBP4 and Vitamin A, STG-001 modulates the visual cycle and the accumulation of cytotoxic retinoids in the eye, potentially reducing the rate of damage to the retina and preserving vision in Stargardt Disease patients.

Stargardt Disease

Stargardt Disease is an inherited retinal degenerative disease that leads to progressive central visual loss and legal blindness.  It is the leading cause of childhood blindness with an estimated prevalence of 50,000-80,000 patients in the US and major European countries combined.  It occurs in 1 in 8,000 to 1 in 10,000 people making it the most common juvenile macular dystrophy.

There is no treatment currently available.  Patients typically exhibit comorbidities, including depression.  Typical onset of disease is generally between 10-20 years of age. 

About Stargazer Pharmaceuticals, Inc.

Stargazer Pharmaceuticals, Inc., is a Boston-based biopharmaceutical drug development company launched in 2018 with a focus on rare diseases of the eye, and an initial focus on Stargardt Disease.  For more information, please visit https://www.stargazerpharmaceuticals.com/

* https://clinicaltrials.gov/ct2/show/NCT04489511?term=stg-001&draw=2&rank=1) 

 

 

 

 

  • Acquisition of Vedere Bio includes lead preclinical intravitreally-injected AAV gene therapy programs focused on pan-genotypic vision restoration in patients with photoreceptor-based vision loss.
  • $150 million upfront with the remainder in early regulatory and clinical milestones for a total of $280 million.
  • Newly formed Vedere Bio II will leverage an ocular gene therapy toolbox to develop a new pipeline of novel vision restoration and vision preservation therapies.

Vedere Bio, Inc. (Vedere Bio), a stealth-stage company focused on advancing photoreceptor-protein-based optogenetic therapies that are delivered to the retina intravitreally to restore functional vision, announced today that it has been acquired by Novartis. Shareholders in Vedere Bio received $150 million upfront and will be eligible for up to $130 million in milestone payments, for a total of $280 million. Based on technology from the laboratories of Drs. Ehud Isacoff and John G. Flannery of UC Berkeley, and technology directed at enhanced ocular gene therapy delivery arising jointly between UC Berkeley and the School of Veterinary Medicine at the University of Pennsylvania, Vedere Bio was formed in the Atlas Venture incubator in June 2019. The company was launched with a $21 million Series A financing and began lab operations at LabCentral in Cambridge, MA where it advanced its lead programs from concept to development candidate within one year. Immediately prior to the acquisition, certain earlier-stage vision restoration and vision preservation assets leveraging the company’s ocular gene therapy toolbox were spun out into a newly formed entity – Vedere Bio II, Inc.

“The medical need for new therapies to treat blindness is unambiguous,” said Jay Bradner, President of the Novartis Institutes for BioMedical Research. “Vedere Bio’s innovative technologies expand the potential for gene therapy to improve the lives of patients facing vision loss due to photoreceptor death attributable to a number of prevalent eye diseases.”

“Vedere Bio’s photoreceptor-protein-based optogenetics program has important advantages over competing approaches and brings us one step closer to delivering functional vision to patients in need.  Our proprietary intravitreal capsids enable not only Vedere Bio’s optogenetics products but also other ocular gene therapies,” said Cyrus Mozayeni, M.D., Chief Executive Officer, President of Vedere Bio and Atlas Venture Entrepreneur in Residence. “Our sale to Novartis is an important milestone in advancing Vedere Bio’s most advanced programs to patients around the world. At the same time, I look forward to working with our experienced team to advance our highly innovative, earlier stage assets as part of the newly established Vedere Bio II.”

“Through Vedere Bio’s approach, we are able to see the impact of venture philanthropy in achieving real progress in the development of new therapies for patients with vision loss,” said Ben Yerxa, Ph.D., CEO of Foundation Fighting Blindness. “For the millions of patients globally living with inherited retinal degenerations and other eye diseases caused by photoreceptor death, the advancement of novel optogenetics therapies provides hope for the future. In addition, we are eager to partner with Vedere Bio II to accelerate their novel ocular gene therapy programs.”

The newly formed Vedere Bio II, Inc. will operate as a wholly independent entity from Novartis and Vedere Bio. Vedere Bio II aims to develop a pipeline of novel vision restoration and vision preservation medicines by targeting underserved indications. Backed by the full Vedere Bio investor syndicate of Atlas Venture, Mission BioCapital and Foundation Fighting Blindness (RD Fund), the Vedere Bio II team will launch with Vedere Bio’s founders, team and facilities to advance its pipeline.

 “The acquisition of Vedere Bio by Novartis speaks to the strength of the underlying science from our founders and to the incredible job the team has done in advancing these programs over the past year. We are very excited by the potential of this technology and the opportunity to deliver true restoration of sight for patients who have been living in darkness for so long,” said Kevin Bitterman, Ph.D., Partner at Atlas Venture and Chairman of the Vedere Bio Board of Directors. “Since Vedere Bio was founded in 2019, the company has moved with unprecedented urgency over the course of one year to advance from concept to development candidate. We look forward to the advancement of the lead programs by Novartis and are eager to see the progress of the Vedere Bio II team in the future to develop novel ocular gene therapies for patients in need.”

The transaction closed in September 2020.

About Vedere Bio II, Inc.

Vedere Bio II, which will be referred to as Vedere going forward, is a privately held, emerging biopharmaceutical company leveraging an ocular gene therapy toolbox to develop a pipeline of vision restoration and vision preservation therapies. Comprised of a diverse team of pioneering scientists, Vedere Bio II is discovering and developing next generation ocular gene therapies to make vision restoration a reality in areas of high global unmet medical need. The company is headquartered in Cambridge, MA and is funded by Atlas Venture, Mission BioCapital and Foundation Fighting Blindness (RD Fund). For more information, please visit www.vederebio.com or follow Vedere Bio II on Twitter and LinkedIn.

Paris, October 21, 2020 – SparingVision (“the Company”), a genomic medicine company focused on ocular diseases, today announces a €44.5 million financing round. The round was led by 4BIO Capital (“4BIO”) and UPMC Enterprises, and included Jeito Capital (“Jeito”) and Ysios Capital (“Ysios”). In addition, current investors Bpifrance and Foundation Fighting Blindness (“FFB”) participated in the round.

Proceeds from the financing will be primarily used to advance the development of SparingVision’s
breakthrough treatment SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa (“RP”). Most
notably the funding will support SparingVision’s GMP activities (including the manufacturing of a first clinical
batch of the product, currently ongoing), the IND/CTA regulatory activities and the conduct of a first-in-man
study, scheduled to start in 2021. The Company also intends to further expand its management team and
commence operations in the US.

SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting of one neurotrophic factor and one
oxidative stress reducing enzyme which, acting synergistically, aim to slow or stop the degeneration of
photoreceptors. Loss of photoreceptors leads to blindness in RP, one of the most common inherited retinal
diseases that affects two million patients worldwide. There is currently no treatment approved for RP
patients independently of their genetic background.

Torreya Capital, LLC served as exclusive placement agent for the offering.

SparingVision also announces today that the Company’s Chairman of the Board, Stéphane Boissel, has been
appointed Chief Executive Officer of the Company.

Stéphane Boissel, SparingVision’s President and Chief Executive Officer, said,We are delighted to have closed this financing round, which demonstrates the excitement around SparingVision’s lead compound, SPVN06. With its singular mutation-agnostic approach, SPVN06 could have a much broader commercial potential than most gene therapy products for RP currently in development and will be used as an anchor to build an economically-viable portfolio of therapies in the field of ophthalmology. Our shareholders, both new and existing, are all long-term, strategic and patient-centric investors that share our vision and we are excited to be working with them to achieve our goals.

Before joining SparingVision as President and CEO, Stéphane Boissel was Executive Vice President,
Corporate Strategy at Sangamo Therapeutics (“Sangamo”), a Nasdaq-listed gene-editing company based in
San Francisco, California. Prior to Sangamo, he was CEO of TxCell, the first ever CAR-TReg company, that
was sold to Sangamo in 2018. Prior to TxCell, Stéphane served as CEO of Genclis, a molecular diagnostic
company, and EVP and CFO of Innate Pharma, a Nasdaq-listed company, and Transgene.

Stéphane has also been a Board member of several leading biotechnology companies, including Nasdaqlisted Erytech Pharma and Elsalys Biotech, where he served as Chairman. Earlier in his career, Stéphane worked in investment banking for Lazard, where he focused on principal investment in France, Singapore and Hong Kong. He started his career at PWC. Stéphane graduated in management and finance from the IAE Lyon, University of Lyon and PSL Paris-Dauphine University (France) and received his MBA from the University of Chicago Booth School of Business.

Dr. José-Alain Sahel, Chair of the Department of Ophthalmology at the University of Pittsburgh School of Medicine, Director of the Institut de la Vision (Sorbonne Universite, Inserm, CNRS, Paris) and co-founder of SparingVision along with Dr. Thierry Léveillard, added, “SparingVision’s neuroprotective approach, which I started working on some 20 years ago at Institut de la Vision, has all the attributes of a winning gene therapy solution for retinitis pigmentosa patients in desperate need of a universal therapeutic option. I am looking forward to helping Stéphane and the team best position SPVN06 for clinical development up to regulatory licensing.

The Board of Directors of SparingVision expresses its deepest gratitude to Florence Allouche, co-founder, who is leaving her position as President of the Company, for her hard work and support over the years which has enabled the Company to pass many milestones and successfully refinance itself.

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.

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
tplohoros@6degreespr.com

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. 

Retinitis Pigmentosa

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.

CheckedUp Launches Virtual Visits, Advanced Telehealth Platform for Specialty Care

Advanced Telemedicine System Delivers Patients, Physicians, and Partners an Exceptional Virtual Care Experience that Redefines Telemedicine

April 28, 2020 09:27 AM Eastern Daylight Time

NEW YORK--(BUSINESS WIRE)--CheckedUp – the fastest growing specialty medicine digital point of care company – today announced the launch of CheckedUp Virtual Visits, an innovative, advanced telemedicine system designed to redefine virtual specialty care.

CheckedUp Virtual Visits, designed by physicians for physicians, was created to keep patients and physicians at the center of pivotal healthcare decisions as “point of care” engagement evolves with patient needs and becomes increasingly digital. As the COVID-19 pandemic continues to alter the world’s “new normal,” CheckedUp is proud to offer a valuable, innovative service that redefines the virtual consultation experience. Its innovative platform empowers specialty care physicians, patients, and industry partners in multiple, dynamic ways.

“From early on, our leadership team viewed the COVID-19 crisis as an unprecedented challenge requiring an innovative response,” said Mark Goethals, Vice President of Marketing, CheckedUp. “Our efforts are focused on a new, post-COVID healthcare delivery landscape, delivering the service and functionality of a virtual examination room to patients and enabling providers to deliver superior virtual patient care. CheckedUp Virtual Visits allows physicians to meet their patients when and where they are, while providing innovative tools for providers and patients during their digital consultation. We are also happy to extend that accessibility to our partners in the life sciences, so that they can be a part of tomorrow’s healthcare conversations.”

CheckedUp Virtual Visits offers distinct care advantages over traditional telemedicine systems. It simplifies scheduling and operations, allows patients to see the dedicated specialist they know and trust, and brings the same easy to use technologies physicians use in the physical exam room, into the virtual realm.

“CheckedUp Virtual Visits leads a new era in the way physicians engage with patients during virtual consultations,” said Dan Schwartz, Senior Vice President, Sponsorship Sales, CheckedUp. “Current telemedicine systems, while serving an important role today, offer very little by way of additional in-office technologies, making exceptional care possible. We expect to change that.”

CheckedUp Virtual Visits is designed to perfectly replicate the patient and physician experience in a virtual setting, streamline administrative efforts by tracking physicians' personal notes and information regarding every consult, and remain HIPAA Compliant.

About CheckedUp

CheckedUp is one of New York’s fastest growing healthcare technology growth companies and is the only 100% digital, push technology platform designed for specialty point of care. CheckedUp has deployed a state-of-the-art platform into Specialty Healthcare facilities across the United States to actively engage patients, caregivers, and physicians in the waiting room, exam room, and at home. As a healthcare technology leader, CheckedUp aspires to create better educated and more confident patients and physicians who are empowered to make better health decisions together. CheckedUp was ranked #279 on Inc. 5,000’s list of the Fastest Growing Private Companies in the US. Learn more at www.CheckedUp.com.

 

ProQR Announces Positive Findings From an Interim Analysis in the Phase 1/2 trial of QR-421a for Usher Syndrome and Provides Business Update

  • QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse events
  • QR-421a is the second ophthalmology program where clinical activity was predicted by translational models, further validating the platform
  • COVID-19 pandemic expected to impact timelines for the pipeline
  • ProQR anticipates its cash runway will fund operations into H2 2022

LEIDEN, Netherlands & CAMBRIDGE, Mass., March 31, 2020 (GLOBE NEWSWIRE) --  March 31, 2020 -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announces positive findings from a planned three-month interim analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations. The Company is also providing an update on business operations in relation to the COVID-19 pandemic. Management will host a conference call today at 8:15 am ET.

“The goal of the interim analysis of this 24 month Stellar trial of QR-421a was to assess safety and early signs of efficacy for the purpose of informing next steps in development and future trial strategy,” said David Rodman, M.D., Executive Vice President of Research and Development of ProQR. “We are pleased with the current safety profile and are very encouraged by early signals of target engagement and clinical activity supported by concordant benefit observed across multiple outcome measures for 25% of QR-421a-treated patients thus far in this trial. The findings support continuing the trial as planned, with both cohort expansion and dose escalation in order to identify a potential development path to registration. Importantly, these data represent the second program from our ophthalmology pipeline that is supported by preclinical predictions from human retinal organoids, providing further validation of our translational approach and platform technology.”

Phase 1/2 Three-Month Interim Analysis

The interim analysis (IA) is based on nine and three month data from the first and second dose  cohorts, respectively, of the Stellar Phase 1/2 clinical trial of QR-421a, an investigational RNA therapy. The Stellar trial is a randomized, single ascending dose, global multicenter, longitudinal, 24-month study, involving active versus sham procedure. The first two cohorts include a total of 14 subjects (ranging from 24-65 years in age), of which eight received a single dose of QR-421a and six received a single sham procedure for masking. Six subjects were enrolled in the 50 µg cohort (“low dose”), of which four received treatment and two were randomized to sham; eight patients were enrolled in the 100 µg cohort (“mid dose”) of which four received treatment and four were randomized to sham. The population varied in disease characteristics with both Usher syndrome (n=6) and nsRP (n=8) affected subjects included, genetic background with both homozygous (n= 4) and heterozygous (n=10) subjects for USH2A exon 13 mutations, and visual impairment at baseline ranging from mild to severe.

Key initial findings include the following:

Safety data: Across both cohorts thus far, QR-421a was observed to be generally well tolerated with no serious adverse events noted.

Efficacy data: In the six sham treated subjects (two followed for 9 months and four for 3 months), outcome measures demonstrated no consistent pattern of response above the “noise” level.  In contrast, two of eight QR‑421a-treated patients (one each in the 50 µg and 100 µg dose cohorts) demonstrated benefit across multiple concordant outcome measures.  

  • Responder 1: One of four treated patients in the low dose group was classified as a responder, with onset of action observed by the 3 month visit. Benefit was maintained for 6 months or longer, which is consistent with the expected half-life of QR-421a in photoreceptors. This Usher syndrome patient was homozygous for USH2A exon 13 mutations and had moderate visual impairment at baseline (peripheral vision affected). Concordant benefit was observed across multiple relevant measures appropriate to the severity of the patient’s disease, including full field stimulus threshold test (FST) [deterioration by 5 dB in untreated eye, treated eye stable], dark adapted chromatic (DAC) perimetry [15 dB.steradian improvement in peripheral sensitivity in treated eye, <5 dB.steradian change in untreated eye], and optical coherence tomography (OCT) assessment of photoreceptor Ellipsoid Zone (EZ area). For FST and OCT, the contralateral, untreated eye demonstrated modest deterioration while the treated eye showed stabilization. For DAC perimetry the untreated eye was unchanged, whereas the treated eye demonstrated improvement.
  • Responder 2: One of four treated patients in the mid dose group was classified as a responder with onset of action observed by 3 months. This non-syndromic RP patient was heterozygous for USH2A exon 13 mutations and had severe visual impairment at baseline (peripheral and central vision affected) with baseline best corrected visual acuity (BCVA) of 33 and 36 letters (approximate Snellen equivalent: 20/250 and 20/200) in the treated and untreated eye, respectively. Concordant benefit was observed across multiple relevant measures appropriate for the stage of disease including FST (improvement by 12 dB in treated eye, no improvement in untreated eye), DAC (up to 10 dB.steradian improvement in treated eye, with deterioration in the untreated eye), and BCVA (7 letter improvement from baseline of 33 letters, which is more than one line on the ETDRS eye chart, compared to no change in the untreated eye).

Next steps: Based on the safety profile and early evidence of efficacy observed to date, the Company plans to take advantage of the adaptive design, and expand the 100 µg cohort with additional subjects who are homozygous for exon 13 mutations. Dose escalation to 200 µg (“high dose”) is planned to occur in parallel. An interim analysis of dose- and gene copy-dependent safety and efficacy will be planned once all additional subjects have reached at least 3 months of treatment.

Business Update Related to COVID-19 Pandemic

The COVID-19 pandemic is rapidly evolving and has prompted global health concerns, the duration, severity and exact impact of which are currently unknown and cannot be predicted with confidence. In consultation with the trial sites, due to the COVID-19 pandemic the Company also expects a delay in all of its ongoing and scheduled trials, including the pivotal trial of sepofarsen for Leber congenital amaurosis 10. ProQR is implementing mitigation procedures that support a rapid ramp up in enrollment as soon as the disruption resolves, including additional patient identification activities and documentation for additional site activations, while prioritizing the safety of trial participants and healthcare providers. For the trials of QR-421a and QR-1123, patients have already been identified for the next dose cohorts and the Company expects to begin dosing as soon as practical after clinical sites are ready and able to do so. This will be the same for the start of the clinical trial for QR-504a. The Company currently does not believe that its supply chain will be affected.

Due to the COVID-19-related delays, the Company has undertaken a budget review process and now anticipates its cash runway will fund operations into the second half of 2022.

“In these uncertain times, the health and safety of patients in our trials, their caregivers, and our employees remains our top priority. We believe we have taken appropriate measures designed to limit their risk,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “While we manage the challenges stemming from the COVID-19 pandemic, we remain confident in the fundamentals of our business, as demonstrated in part by the data we are sharing today from our QR-421a program. We have a productive platform, a deep pipeline of RNA therapies focused on inherited retinal diseases, and the benefit of a strong balance sheet, which positions us well to endure the current disruption and continue the important work with the communities we serve.”

Conference Call

Management will discuss the data and next steps for development during a webcasted conference call today, March 31, 2020, at 8:15 a.m. ET. The live and archived webcast of this presentation can be accessed through the Events and Presentations page on the Investors section of the Company’s website, www.ProQR.com. The dial-in details for the call are +1 631-510-7495 (US), +31 (0) 207143545 (NL), conference ID: 5986384. The archived webcasts will be available for approximately 30 days following the presentation date.

About Usher Syndrome Type 2 and Non-Syndromic Retinitis Pigmentosa

Usher syndrome is the leading cause of combined deafness and blindness. People with Usher syndrome type 2 are usually born with hearing loss and start to have progressive vision loss during adulthood. The vision loss can also occur without hearing loss in a disease called non-syndromic retinitis pigmentosa. Usher syndrome type 2 and non-syndromic retinitis pigmentosa can be caused by mutations in the USH2A gene. To date, there are no pharmaceutical treatments approved or in clinical development that treat the vision loss associated with mutations in USH2A.

About QR-421a

QR-421a is being evaluated in the Phase 1/2 Stellar trial and is a first-in-class investigational RNA therapy 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 US and the European Union and received fast-track and Rare Pediatric Disease designations from the FDA.

About the Stellar Phase 1/2 Trial of QR-421a

Stellar, or PQ-421a-001, is a first-in-human study of QR-421a in adults who have vision loss due to mutations in exon 13 of the USH2A gene and is conducted at expert sites in North America and Europe. It is a double-masked, randomized, 24-month study exploring the safety and efficacy of a single intravitreal injection of several dose levels of QR-421a and a control sham procedure into one eye.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and autosomal dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*

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. Such forward-looking statements include, but are not limited to, statements regarding QR-421a, and the clinical development and the therapeutic potential thereof, our other programs and business operations, including timing of commencing clinical trials and enrollment of patients therein, the expected impact of the COVID-19 on our business operations, including our research and development plans and timelines and the supply chain for our clinical and development programs, 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 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; 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, financial and accounting risks and litigation. 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.

Cautionary Note on Future Updates

The statements contained in this press release reflect our current views with respect to future events, which may change significantly as the global consequences of the COVID-19 pandemic rapidly develop. Accordingly, we do not undertake and specifically disclaim any obligation to update any forward-looking statements.

ProQR Therapeutics N.V.

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%).

About SparingVision 
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.

www.sparingvision.com

Contacts

Florence Allouche 
Pharm D. President SparingVision, 
+33 1 43 46 20 60 
fag@sparingvision.com

Florence Portejoie 
FP2COM 
+ 33 6 07 76 82 83 
fportejoie@fp2com.fr

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.

About QR-421a

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.

About ProQR

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. 
*Since 2012*

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 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.

Investor Contact:
Lisa Hayes
Vice President of Investor Relations and Corporate Communications
T: +1 202 360 4855 
lhayes@proqr.com 

 

ProQR Therapeutics N.V. logo

 

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 rustykelley@fightblindness.org.

  • Foundation Fighting Blindness and ProQR enter into a partnership to develop QR-421a for Usher syndrome type 2A, targeting mutations in exon 13 of the causative USH2A gene.
  • Foundation Fighting Blindness will provide milestone-based co-funding of up to $7.5 million to ProQR to advance the program into the clinic.
  • QR-421a received orphan drug designation from the FDA.
  • There are currently no therapies commercially available or in clinical development for the vision loss associated with Usher syndrome type 2A.
  • QR-421a is part of ProQR’s growing ophthalmology pipeline which also includes lead candidate, QR-110 for Leber’s congenital amaurosis 10 currently in clinical trials, and three additional pipeline programs, QR-411 that addresses another genetic mutation resulting in Usher syndrome type 2A, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy. 
  • QR-421a is expected to advance towards the clinic in 2018, with clinical data expected in 2019.

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.

About ProQR

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.
*Since 2012*

About QR-421a

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:
Bonnie Ortega
T: +1 858 245 3983
ir@proqr.com

 

Sources: ProQR Therapeutics and Foundation Fighting Blindness

Primary Logo

 

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.”