Nasdaq GlobeNewswire

ProQR In-licenses Worldwide Rights to Ophthalmology Drug Candidate from Ionis Pharmaceuticals

Del

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.

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, 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.
*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 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.:
Investor Contact:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com

Media Contact:
Sara Zelkovic
LifeSci Public Relations
T: +1 646 876 4933
Sara@lifescipublicrelations.com 

Om Nasdaq GlobeNewswire

Nasdaq GlobeNewswire
Nasdaq GlobeNewswire
One Liberty Plaza - 165 Broadway
NY 10006 New York

+1 212 401 8700http://www.nasdaqomx.com

NASDAQ (NASDAQ: NDAQ) is a leading provider of trading, exchange technology, information and public company services across six continents.

Følg saker fra Nasdaq GlobeNewswire

Registrer deg med din epostadresse under for å få de nyeste sakene fra Nasdaq GlobeNewswire på epost fortløpende. Du kan melde deg av når som helst.

Siste saker fra Nasdaq GlobeNewswire

Navigators Stockholders Approve Merger with The Hartford16.11.2018 22:01Pressemelding

STAMFORD, Conn., Nov. 16, 2018 (GLOBE NEWSWIRE) -- The Navigators Group, Inc. (NASDAQ:NAVG) (“Navigators”) announced that, at a special meeting of stockholders held today, Navigators’ stockholders approved the adoption of the previously announced merger agreement relating to the proposed transaction between Navigators and The Hartford Financial Services Group, Inc. (“The Hartford”), whereby Navigators will become a wholly owned subsidiary of The Hartford. Subject to the terms and conditions of the merger agreement, at the effective time of the merger, each eligible share of Navigators common stock will be cancelled and converted into the right to receive $70.00 in cash. The merger remains subject to various closing conditions, including receipt of various regulatory approvals, and is expected to close during the first half of 2019. About Navigators The Navigators Group, Inc. (NASDAQ: NAVG)(“Navigators” or the “Company”) is a global specialty insurance holding company. We provide custom

BlissCo Receives Cannabis Sales License from Health Canada16.11.2018 14:00Pressemelding

Not for distribution in the U.S. or to U.S. Newswires VANCOUVER, British Columbia, Nov. 16, 2018 (GLOBE NEWSWIRE) -- via NetworkWire – BlissCo Cannabis Corp. (CSE: BLIS) (OTCQB: HSTRF) (FRA: GQ4B) (“BlissCo” or the “Company”), is pleased to announce that it has received a comprehensive sales license from Health Canada to sell bulk cannabis and pre-packaged, labeled, and tested cannabis products. “We are thrilled to receive our sales license and to be able to start pre-registering clients and supplying both the robust medical and adult-use markets in Canada with our premium cannabis products,” said Damian Kettlewell, CEO of BlissCo. “BlissCo is committed to providing top-tier service to all of our customers.” The Company will soon be launching its e-commerce website and BlissConnect, a telemedicine app for medical cannabis consultations, to service its medical clients. The Company is currently ready to pre-register clients wishing to be first in line when shipping begins. To pre-registe

Gratomic Announces Non-Brokered Private Placement16.11.2018 14:00Pressemelding

TORONTO, Nov. 16, 2018 (GLOBE NEWSWIRE) -- Gratomic Inc. (“GRAT” or the “Company”) (TSX-V: GRAT) (FRANKFURT:CB81, WKN:A143MR) is pleased to announce the offering of a non-brokered private placement of up to 10,000,000 working capital units (the “WC Unit”) for up to $1,000,000 (the “Offering”). Each WC Unit is priced at $0.10 and consists of one (1) common share and one (1) common share purchase warrant (“WC Warrant”). Each WC Warrant entitles the holder to purchase one (1) common share (a “WC Warrant Share”) at a price of $0.20 per WC Warrant Share until the earlier of: (i) three (3) years following the Closing of the Offering; and (ii) in the event that the closing price of the Common Shares on the TSX Venture Exchange is at least $0.30 for ten (10) consecutive trading days, and the 10th trading day (the “Final Trading Day”) is at least four (4) months from the Closing Date, the date which is thirty (30) days from the Final Trading Day. Eligible Finders may receive 5% of the value of

Mitratech Listed Among Financial Times’ Inaugural Top 35 Intelligent Business Award Winners and Other Prestigious Categories16.11.2018 10:00Pressemelding

Legal Tech Top 10 FTIL Intelligent Business Award for Contracting FTIL Intelligent Business Award for Use of Legal Technology AUSTIN, Texas, Nov. 16, 2018 (GLOBE NEWSWIRE) -- The Financial Times’ inaugural FT Intelligent Business Special Report has recognized Mitratech among the top companies with the greatest impact on the practice and business of law. The report highlights the integration of people, processes, and data to solve a legal business’ operational challenges and how the ramifications of this back-office revolution are felt across organizations everywhere. “The fact that the Financial Times is now tracking the impact of legal technology reinforces that the industry is at a tipping point,” said Jason Parkman, Mitratech CEO. “There’s more transformation that lies ahead. We are honored and humbled to be a part of that movement and in good company with several clients we were recognized with.” The company was presented with the following honors: FT Intelligent Business Top 35: M

GridGain Ranked 77th Fastest Growing Company in North America on Deloitte’s 2018 Technology Fast 500™16.11.2018 09:00Pressemelding

1,724 Percent Growth Over the Past Four Years Ranks 19th in the San Francisco Bay Area FOSTER CITY, Calif., Nov. 16, 2018 (GLOBE NEWSWIRE) -- GridGain® Systems, provider of enterprise-grade in-memory computing solutions based on Apache® Ignite™, is ranked No. 77 on the Deloitte 2018 Technology Fast 500™, a ranking of the 500 fastest growing technology, media, telecommunications, life sciences and energy tech companies in North America. GridGain, which grew 1,724 percent over the past four years, was the 19th fastest growing company in the San Francisco Bay Area. “GridGain continues to benefit from strong market demand for our market-leading in-memory computing platform that helps customers realize their digital transformation and omnichannel customer experience initiatives,” said Abe Kleinfeld, President and CEO of GridGain Systems. “When you combine the best talent in the industry with great execution and strong customer demand, wonderful things happen.” “Congratulations to the Deloit

I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.

Besøk vårt presserom