ProQR In-licenses Worldwide Rights to Ophthalmology Drug Candidate from Ionis Pharmaceuticals
ProQR receives exclusive worldwide license for IONIS-RHO-2.5Rx, now QR-1123, for autosomal dominant retinitis pigmentosa (adRP), a rare inherited form of blindness with no approved therapy
A first in human Phase 1/2 clinical trial in adRP patients is expected to start in 2019
LEIDEN, the Netherlands, Oct. 29, 2018 (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 signing of an agreement with Ionis Pharmaceuticals to license QR-1123 (formerly “IONIS-RHO-2.5Rx”), an RNA medicine for autosomal dominant retinitis pigmentosa (adRP) caused by the P23H mutation in the rhodopsin (RHO) gene.
“Building on the recent proof of concept with QR-110 in LCA10 patients, we have confidence in the potential of single stranded RNA-targeted therapies to treat patients with genetic eye diseases,” said Daniel A. de Boer, chief executive officer of ProQR. “By acquiring the rights to QR-1123, we have strategically expanded our pipeline in retinal diseases. Ionis has a wealth of experience in discovery and development of antisense drugs and we look forward to working with them on this important program. Unlike other molecules we are developing, QR-1123 is a gapmer with a mutant allele-specific knockdown mechanism of action. If validated, it would further broaden the potential of RNA-targeted therapies in retinal diseases. We are excited to start clinical development to explore QR-1123’s potential in helping patients with P23H adRP.”
Under the terms of the agreement, ProQR was granted an exclusive worldwide license to QR-1123 and relevant patents. ProQR made an upfront payment in ordinary shares in the aggregate amount of $2.5 million, at $22.23 per share, which represents a 20% premium (based on the volume weighted average price of the previous 20 trading days) to its common stock, to Ionis upon signing the agreement. ProQR will also make future milestone payments, certain of which will be made in equity and others in cash or equity at ProQR’s discretion, and royalties on net sales of 20% through the royalty term.
Preclinical activities have been completed by Ionis and were presented at the 2015 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) and published in a peer-reviewed article, Murray et al 2015, IOVS. A natural history study in patients with P23H adRP has been conducted. The results of this study provide valuable information on disease progression in these patients, which is important data for designing the clinical development plan. ProQR expects to start a Phase 1/2 clinical trial in patients with adRP in 2019, pending submission and clearance of the IND application by the U.S. Food and Drug Administration (FDA).
Brett P. Monia, Ph.D., chief operating officer and senior vice president of translational medicine at Ionis, added, “IONIS-RHO-2.5Rx, now QR-1123, is an antisense oligonucleotide we designed to specifically target only the mRNA from the disease-causing rhodopsin gene, which has a single nucleotide P23H mutation, while preserving expression of the mRNA from the normal rhodopsin gene, which is important for the eye to function properly. IONIS-RHO-2.5Rx was developed by Ionis scientists dedicated to discovering innovative therapeutics to fight diseases where no other treatments have proven effective or even existed. We believe that ProQR has the ophthalmology expertise and experience in developing oligonucleotide drugs for the eye to rapidly bring this program through clinical development and ultimately to patients.”
About Autosomal Dominant Retinitis Pigmentosa
Autosomal dominant retinitis pigmentosa, or adRP, is a severe and rare genetic disease that causes progressive reduction in night and peripheral vision during childhood and frequently leads to blindness in mid adulthood. In the United States, the most prevalent mutation associated with adRP is the P23H point mutation (also known as the c.68C>A mutation) in the rhodopsin (RHO) gene and affects approximately 2,500 people. This gain of function mutation causes misfolding of the rhodopsin protein that becomes toxic to the photoreceptor cells in the retina. Over time the cells die and vision is progressively lost. There are currently no therapies approved or in clinical development for P23H adRP. A natural history study in patients with P23H adRP has been conducted.
About QR-1123 (formerly IONIS-RHO-2.5Rx)
QR-1123 is a first-in-class investigational oligonucleotide (gapmer) that was developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology for the treatment of adRP due to the P23H mutation in the RHO gene. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutated mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye.
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, dystrophic epidermolysis bullosa and cystic fibrosis. 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 our license with Ionis, including the terms thereof, our development plans with respect to our product candidates, including QR-1123, and the therapeutic potential of our product candidates, including QR-1123. 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 prior and ongoing studies, including those conducted by Ionis, may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, that we may not realize the intended benefits from our license with Ionis, clinical development process, including our plans to commence clinical development of QR-1123, 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.:
Chief Financial Officer
T: +1 415 231 6431
LifeSci Public Relations
T: +1 646 876 4933
One Liberty Plaza - 165 Broadway
NY 10006 New York
GlobeNewswire is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.
Følg saker fra GlobeNewswire
Registrer deg med din epostadresse under for å få de nyeste sakene fra GlobeNewswire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra GlobeNewswire
GSA Europe’s Managing Director Elected Co-lead of CEN’s TC456 Committee15.1.2019 21:12 | Pressemelding
SAN GWANN, Malta, Jan. 15, 2019 (GLOBE NEWSWIRE) -- GSA Europe’s Managing Director Mark Pace has been elected by the Comité Européen de Normalisation (CEN) to lead the creation of a European Union online gaming reporting standard. CEN has established Technical Committee 456 to create this standard in support of online gambling supervision. GSA Europe joined Technical Committee 456 as a Liaison Organization in 2017. The Technical Committee’s mandate from the European Commission is directly aligned with work that GSA Europe has already started, namely, to create a single standard set of data elements and single standard way in which data is provided by online gambling providers to EU Member State regulatory authorities. In addition to joining and now leading the Technical Committee’s work, GSA Europe also donated their draft Regulatory Data Set and Regulatory Reporting Interface to CEN seeking to help jump-start the Committee’s work. “Joining CEN’s TC456 as a Liaison Organization, and no
Orion Biotechnology Reports Positive Results for Colorectal Cancer in Preclinical Study15.1.2019 15:00 | Pressemelding
OTTAWA, Jan. 15, 2019 (GLOBE NEWSWIRE) -- Orion Biotechnology Canada Ltd., a developer of novel medical treatments, today announced preclinical data produced in collaboration with Charles River Laboratories, evaluating the efficacy of OB-002O (5P12-RANTES) in colorectal cancer. BALB/c mice were inoculated subcutaneously with the CT-26 colorectal cancer cell line. Three days after inoculation intraperitoneal treatment was started with OB-002O, a murine anti-PD-1 antibody, OB-002 + anti-PD-1, or saline placebo. Treatment was given daily or every third day. Treatment with OB-0020 alone led to statistically significant delayed tumor growth at multiple time-points. An even more profound effect on the decrease in mean tumor volume was observed in combination cohort where animals received both, OB-0020 and the anti-PD-1 antibody (p>0.05). “There is growing awareness of the role of CCR5 antagonism as an important component of cancer immunotherapy. OB-002O is an extremely potent CCR5 antagonist
Credit Suisse Partners with CGAP on Digital Finance to Catalyze Delivery of Energy, Water and other Services to Low Income People15.1.2019 14:08 | Pressemelding
Washington/Zurich, Jan. 15, 2019 (GLOBE NEWSWIRE) -- Credit Suisse, one of the world's leading financial services providers, is partnering with the Consultative Group to Assist the Poor (CGAP) to support its work in building inclusive and responsible financial systems that help people move out of poverty, protect their economic gains and advance the broader global development agenda. CGAP is a global partnership of more than 30 leading development agencies, private foundations and national governments committed to financial inclusion as a means toward expanding opportunities for poor people. Through their engagement, CGAP and Credit Suisse will capitalize upon and leverage each other’s knowledge to further financial inclusion and its contribution toward achieving the United Nations’ Sustainable Development Goals (SDGs). Among the projects that align well with Credit Suisse’s expertise are CGAP’s work on sustainable business models that use digital finance technologies to lower the cost
TrueCommerce Recognized as a Leader in IDC MarketScape for Worldwide Multi-Enterprise Supply Chain Commerce Network15.1.2019 14:00 | Pressemelding
-- “The vendor offers a true partnership for their customers and has become an extension of their internal staff and resources to manage their supply chains,” says the report -- PITTSBURGH, Jan. 15, 2019 (GLOBE NEWSWIRE) -- TrueCommerce, a global provider of trading partner connectivity, integration and unified commerce solutions , has announced today it was positioned in the Leaders category of the IDC MarketScape for Worldwide Multi-Enterprise Supply Chain Commerce Network report. The research states that TrueCommerce offers a broad set of supply chain applications that extend far beyond traditional data transportation. “Our investments into the cutting-edge unified commerce technology and customer care allowed the company to make this qualitative leap, and we are proud to be recognized by such a reputable global research firm as IDC,” said TrueCommerce president Ross Elliott. “The report advises companies to engage with a network vendor that is at least thinking about what tomorrow
UnionBank Launches First Fully Digital Branch in the Philippines15.1.2019 13:30 | Pressemelding
Appian Cloud Platform Enables Low-Code Rapid Application Development for Powerful Process Automation RESTON, Va. and SYDNEY, Australia , Jan. 15, 2019 (GLOBE NEWSWIRE) -- Appian (NASDAQ: APPN) announces that UnionBank of the Philippines used the Appian Platform to launch its first fully digital branch. The branch, called The Ark, completely transforms the customer experience, making that experience entirely digital, including internet-connected self-service kiosks and virtual reality boxes. Unlike traditional bank branches, The Ark has done away with rows of tellers and long queues, and gone completely paperless. Customers now go to one of the self-service iPad stands stationed around the bank, where they can type in their request, significantly reducing time previously spent waiting in queues for service. “The Ark is an award-winning digital branch that provides a modern experience for our clients within a traditional brick and mortar store,” said Anna Maria Aboitiz Delgado, Head for
Magna Announces Outlook15.1.2019 11:00 | Pressemelding
Sales growth and increased EBIT Margin1 over outlook period Higher free cash flow2 in 2019-2021 period, compared to 2018-2020 AURORA, Ontario, Jan. 15, 2019 (GLOBE NEWSWIRE) -- Magna International Inc. (TSX: MG; NYSE: MGA) today announced its financial outlook for 2019 and 2021. Our outlook to 2021 anticipates continued growth in total sales and improvement in our EBIT Margin compared to 2018 despite our expectation of relatively level light vehicle production across our major end markets of North America and Europe during this same outlook period. Free cash flow is expected to increase to a cumulative total of more than $6.5 billion in the 2019-2021 timeframe as compared to over $6 billion in the 2018-2020 period, reflecting an anticipated increase in earnings and relatively level capital spending. For 2019, our sales are expected to be negatively impacted by the announced disposition of our Fluid Pressure & Controls business, net of acquisitions, and by foreign currency translation,