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[verwijderd] 20 november 2017 14:19

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ProQR Announces Results for the Third Quarter of 2017

Key updates
Runway into the second half of 2019: cash of €39.7 million at end of Q3, increased by gross proceeds of approximately $20 million from an issuance of ordinary shares in November 2017 that will fund operations through potential clinical data readouts in three different programs.
Positive results from the Phase 1b safety and tolerability clinical trial of QR-010 in cystic fibrosis (CF) patients with the F508del mutation. QR-010 was observed to be safe and well-tolerated, and demonstrated encouraging efficacy responses in CF patients.
First patient dosed in the Phase 1/2 safety & efficacy trial of QR-110 in children and adults with Leber’s congenital amaurosis 10 in November. First clinical data readout expected in 2018.
QR-313 for dystrophic epidermolysis bullosa completed the IND enabling studies, received ODD from FDA and pre-clinical data was presented at two scientific meetings. QR-313 is expected to enter the clinic in 2018, with interim data also expected in 2018.
Spin-out of Amylon Therapeutics as a privately-held company focused on central nervous system therapeutics. ProQR continues to be a majority shareholder and is eligible to receive future milestones and royalties.
Drs. Phil Zamore, Cy Stein, Scott Armstrong and Thaddeus (Ted) Dryja appointed to ProQR’s Scientific Advisory Board (SAB).
In vivo proof of concept data for novel Axiomer® RNA editing platform technology presented at the OTS meeting and a DIA industry event.
QR-421 and QR-411 for Usher syndrome received orphan drug designation (ODD) from FDA and EMA.
LEIDEN, The Netherlands, Nov. 20, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), today announced results for the third quarter of 2017.
“I am proud of the milestones our team achieved recently, which included the successful completion and positive data from our Phase 1b study of QR-010 in CF patients, dosing of the first LCA 10 patient in our Phase 1/2 trial of QR-110, announcing the in vivo PoC for our Axiomer technology and the completion of the pre-clinical IND-enabling studies for QR-313 to enable the potential start of our clinical trial in 2018. We look forward to the data-rich 12-18 months ahead of us, with important clinical safety & efficacy readouts in three different clinical programs,” said Daniel A. de Boer, CEO of ProQR. “Beyond these, we have made significant progress in our pipeline of ophthalmology programs and our novel RNA-editing technology, Axiomer®. We are also thrilled with the spin out of Amylon as a privately-held company to a group of private and institutional investors, while maintaining our majority ownership in Amylon. This enables the program to attract external funding for these important central nervous system (CNS) programs that have the potential to make a meaningful difference for patients. Finally, I would like to take this opportunity to welcome Drs. Phil Zamore, Cy Stein, Scott Armstrong and Thaddeus (Ted) Dryja to the SAB, who are all well-regarded in their respective fields and bring with them a wealth of knowledge and experience that will help us to further build and advance our pipeline.”

[verwijderd] 20 november 2017 14:20

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Corporate Highlights
In July, the Company completed a $6.0 million registered direct offering of 1.2 million ordinary shares at an issue price of $5.00 per share with institutional investors.
In July, QR-411 for Usher syndrome received orphan drug designation (ODD) from the FDA and European Medicines Agency (EMA). QR-411 targets the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the USH2A gene. In September, the QR-421 candidate received ODD from the FDA and EMA. QR-421 is designed to address Usher syndrome due to mutations in exon 13 of the USH2A gene. QR-411 and QR-421 are part of the Company’s growing ophthalmology pipeline which also includes lead candidate, QR-110 for LCA 10 currently in clinical trials, and two additional pipeline programs, QRX-1011 for Stargardt’s disease, and QRX-504 for Fuchs endothelial corneal dystrophy.

In August, Dr. Phil Zamore, Dr. Cy Stein and Dr. Scott Armstrong were appointed to the Scientific Advisory Board (SAB), joining fellow members Dr. Art Levin and Dr. Annemieke Aartsma-Rus. The SAB will play a key strategic role as the Company develops its pipeline of RNA therapeutics and novel proprietary technology platforms for severe genetic rare diseases.

In September, the Company completed the spin out of Amylon Therapeutics as a privately-held company focused on the development of therapies for central nervous system (CNS) disorders. ProQR granted an exclusive license to Amylon to develop therapeutics for CNS disorders, with an initial focus on a RNA-based treatment for a rare genetic disease that leads to strokes at mid-adulthood, called Hereditary Cerebral Hemorrhage with Amyloidosis of the Dutch type (HCHWA-D). ProQR retained majority ownership in Amylon and is entitled to future milestones and royalties from potential products developed under the license agreement.
In September, QR-313 for dystrophic epidermolysis bullosa (DEB) received ODD from the FDA, representing the fifth program in the Company’s pipeline to receive ODD in the U.S. DEB is a rare genetic disease that can lead to severe blistering of the skin resulting in high treatment burden and poor quality of life for people with DEB. QR-313 is designed for topical administration and targets the most common mutations within DEB, the mutations in exon 73 of the COL7A1 gene. QR-313 pre-clinical data was presented at the EB2017 Research Conference and ESDR Meeting, both in Salzburg, Austria. A first-in-human clinical trial of QR-313 is expected to be initiated in 2018, with interim data readout also expected in 2018 and full data in 2019.

In September, ProQR presented in vivo proof of concept data for its novel and proprietary Axiomer® RNA editing technology platform at the Oligonucleotide Therapeutics Society (OTS) meeting in Bordeaux, France and at the Drug Information Association (DIA) industry event. The data demonstrated that in an in vivo research model of Hurler syndrome, treatment with the Axiomer® EONs resulted in editing of RNA and partial restoration of the enzymatic activity that is missing in this syndrome. Additionally, the increase in enzymatic activity correlated well with reduced levels of the enzyme’s substrate, the accumulation of which results in the characteristics of the syndrome. The Axiomer® “Editing Oligo Nucleotides”(EONs) recruit an endogenously expressed RNA editing system called ADAR, which it can direct to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G). The Axiomer® platform has the potential to yield a new class of medicines for genetic diseases caused by single nucleotide G-to-A mutations.

In September, the Company announced positive preliminary top-line results from the Phase 1b randomized, double-blind, placebo-controlled, dose escalation study (Study PQ-010-001) to evaluate the safety, tolerability, pharmacokinetics and exploratory efficacy of QR-010 in adults with CF homozygous for the F508del mutation. A number of exploratory efficacy endpoints were assessed in the multiple dose groups. A total of 4 dose levels were studied: 6.25, 12.5, 25 and 50 mg of QR-010 in solution per dose administered via inhalation using the PARI eFlow® nebulizer. Patients eligible to participate were males and females of 18 years and over with a ppFEV1 of =70% at time of inclusion, homozygous for the F508del mutation, and not taking CFTR modulator drugs. The study was designed to enroll 8 cohorts of 8 subjects (6 receiving QR-010, 2 receiving placebo). In cohorts 1-4, a single dose of QR-010 was administered, and in cohorts 5-8, twelve doses of QR-010 were administered over a 4-week period. Based on preliminary top-line results from the trial, QR-010 was observed to be safe and well-tolerated across all dose levels and we believe that QR-010 may have therapeutic benefit for CF patients. Most patients in the trial reported having a reduction in CF symptoms after receiving QR-010 (as measured by an increase in the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score, or CFQ-R RSS) compared to placebo. A supportive trend was observed in improved lung function (as measured by percent predicted forced expiratory volume in 1 second, or ppFEV1) compared to placebo. Subjects that received placebo did not report this reduction in CF symptoms or improvement in lung function. As expected, no change was observed on sweat chloride and weight gain

[verwijderd] 20 november 2017 14:22

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Subsequent Events

In October, Dr. Thaddeus (Ted) Dryja, a pioneer in the field of retinal genetic diseases, joined the Company’s Scientific Advisory Board. Dr. Dryja is a member of the faculty at Massachusetts Eye and Ear and Harvard Medical School, and previously served as the Global Head of Ophthalmology Research at the Novartis Institutes for BioMedical Research. He is expected to be a key advisor as the Company advances its growing ophthalmology pipeline.

In November, Chief Medical Officer, Noreen R. Henig, M.D., departed the Company to pursue a new opportunity and will serve as a special advisor to the Company going forward. David Rodman, M.D., Chief Development Strategy Officer, assumed leadership over clinical development. Dr. Rodman joined ProQR in March 2017 with extensive experience in rare disease drug development, translational medicine and RNA therapeutics, having previously served in leadership roles with Novartis Institute for Biomedical Research (NIBR), Vertex Pharmaceuticals, miRagen Therapeutics and Nivalis Therapeutics. The Company also announced the promotions of Peter Adamson to Senior Vice President Ophthalmology Franchise and Robert Friesen to Senior Vice President Science and Early Development.

In November, J. Stuart Elborn, Clinical Chair in Respiratory Medicine at Imperial College, Consultant at Royal Brompton Hospital, and immediate past-president of the European Cystic Fibrosis Society, presented data from the Phase 1b safety, tolerability and exploratory efficacy study of QR-010 during the North American Cystic Fibrosis Conference (NACFC) in Indianapolis, Indiana. During the meeting, the Company also held an investor and analyst event to discuss the recent Phase 1b data, new opportunities to target stop-codon (or Class I) mutations in the CFTR gene using its Axiomer® technology platform, and provide an update on other candidates in the pipeline.
In November, the Company presented its oligonucleotide therapeutics approach at the EuroTIDES meeting in Vienna, Austria.

In November, the first patient was dosed in the Phase 1/2 open-label trial (PQ-110-001) assessing the safety, tolerability, pharmacokinetics and efficacy of QR-110 in patients with LCA 10, the most common cause of blindness due to genetic disease in children. The trial will enroll six children (age 6 - 17 years) and six adults (= 18 years) who have LCA 10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. During the trial, patients will receive four intravitreal injections of QR-110 into one eye; once every three months. The QR-110 trial is expected to be conducted in three centers with significant expertise in genetic retinal disease in the US and Europe. The objectives of the trial will include safety, tolerability, pharmacokinetics and efficacy as measured by restoration or improvement of visual function and retinal structure through ophthalmic endpoints such as visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR), mobility course and fixation stability. Changes in quality of life in the trial subjects will also be evaluated. QR-110 is the Company’s lead program for genetic blindness and has received fast track designation by the FDA and been granted ODD in the US and EU. Interim safety and efficacy trial results from the majority of patients after six months of treatment are expected in 2018, with full 12-month treatment data from all patients expected in 2019.

On November 16, 2017, the Company consummated an underwritten public offering and concurrent registered direct offering of approximately 6 million ordinary shares at a price of $3.25 per share. ProQR granted the underwriters a 30-day option to purchase up to 745,471 additional ordinary shares at the public offering price, less underwriting discounts and commissions. Gross proceeds from both offerings were approximately $20 million, assuming no exercise of the underwriters’ option to purchase additional shares in the underwritten public offering. Investors in the offering included several new and existing shareholders, along with significant participation from the Management team and Supervisory Board in the underwritten public offering. Proceeds from these offerings along with existing cash on the balance sheet, are expected to fund operations into the second half of 2019.

[verwijderd] 20 november 2017 14:23

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Financial Highlights

At September 30, 2017, ProQR held cash and cash equivalents of €39.7 million, compared to €59.2 million at December 31, 2016. Net cash used in operating activities during the three month period ended September 30, 2017 was €7.6 million, compared to €10.8 million for the same period last year.

Research and development costs totaled €7.2 million for the quarter ended September 30, 2017 compared to €8.3 million for the same period last year and comprised of allocated employee costs including share-based payments, the costs of materials and laboratory consumables, outsourced activities, license and intellectual property costs, and other allocated costs. The decrease in expenses was primarily due to the completion of the nasal potential difference (NPD) study for QR-010.

General and administrative costs increased to €2.8 million for the quarter ended September 30, 2017 compared to €2.0 million for the quarter ended September 30, 2016, which were primarily due to increased investments in our support organization and intellectual property.

Net result for the three month period ended September 30, 2017 was a €10.5 million loss or €0.42 per share, compared to a €10.1 million loss or €0.43 per share for the same period last year. For further financial information for the period ending September 30, 2017, please refer to the financial statements appearing at the end of this release.

[verwijderd] 20 november 2017 14:25

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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-010

QR-010 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the F508del mutation. The technology was exclusively licensed from Massachusetts General Hospital. The F508del mutation results in the production of a misfolded CFTR protein that does not function normally. QR-010 is a single agent designed to bind to the defective CFTR mRNA and to restore CFTR function. QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a small, handheld aerosol delivery device which nebulizes QR-010 into a mist inhaled directly into the lungs. QR-010 has been granted orphan drug designation in the United States and the European Union and fast-track status by the FDA. The QR-010 project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.

About QR-110

QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore normal (wild-type) CEP290 mRNA leading to the production of normal CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 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 by the FDA.

About QR-313

QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce a functional C7 protein, thereby restoring functionality of the anchoring fibrils.

About QR-421

QR-421 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional USH2A protein that causes the disease. QR-421 is designed to exclude exon 13 from the mRNA (exon skipping) and produce truncated but functional USH2A protein, thereby modifying the underlying disease.

About QR-411

QR-411 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the USH2A gene. The mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional or absence of USH2A protein. QR-411 is designed to restore wild-type USH2A mRNA leading to the production of wild-type USH2A protein by binding the mutated pre-mRNA causing normal splicing of the pre-mRNA. QR-411 has been granted orphan drug designation in the United States and the European Union.

About Next-Generation Axiomer® Technology Platform

ProQR is pioneering a next-generation RNA technology called Axiomer®, which could potentially yield a new class of medicines for genetic diseases. Axiomer® EONS mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells. The Axiomer® “Editing Oligo Nucleotides”, or EONs, recruit an endogenously expressed RNA editing system called ADAR, which it can direct to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G).

[verwijderd] 20 november 2017 14:26

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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-010, QR-110, QR-411, QR-421, QR-313 and the clinical development and the therapeutic potential thereof, including our ongoing clinical trial of QR-110, statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in our innovation pipeline and the potential of our Axiomer® technology, statements regarding release of clinical data, and statements regarding our financial resources and cash runway. 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, 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.

[verwijderd] 20 november 2017 14:31

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Zie de bijlage voor de tabellen met cijfers.

Bijlage:

[verwijderd] 20 november 2017 14:37

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Misschien wel het belangrijkste stukje uit dit hele verhaal: "Based on preliminary top-line results from the trial, QR-010 was observed to be safe and well-tolerated across all dose levels and we believe that QR-010 may have therapeutic benefit for CF patients. Most patients in the trial reported having a reduction in CF symptoms after receiving QR-010 (as measured by an increase in the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score, or CFQ-R RSS) compared to placebo. A supportive trend was observed in improved lung function (as measured by percent predicted forced expiratory volume in 1 second, or ppFEV1) compared to placebo. Subjects that received placebo did not report this reduction in CF symptoms or improvement in lung function."

Dus QR-010 werkt! :)
Dat is echt goed nieuws. Nu nog een hogere koers bij de volgende emissie.

[verwijderd] 20 november 2017 14:46

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De laat staat inmiddels op $4.95. Er zijn er dus meer die dit goed nieuws vinden.

[verwijderd] 7 november 2018 15:34

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ProQR (PRQR) Reports Q3 Loss, Misses Revenue Estimates
[Zacks]
Zacks Equity Research
,Zacks•November 7, 2018
ProQR (PRQR) Reports Q3 Loss, Misses Revenue Estimates
ProQR (PRQR) delivered earnings and revenue surprises of 46.15% and -100.00%, respectively, for the quarter ended September 2018. Do the numbers hold clues to what lies ahead for the stock?

ProQR (PRQR) came out with a quarterly loss of $0.21 per share versus the Zacks Consensus Estimate of a loss of $0.39. This compares to loss of $0.49 per share a year ago. These figures are adjusted for non-recurring items.

This quarterly report represents an earnings surprise of 46.15%. A quarter ago, it was expected that this Dutch drugmaker would post a loss of $0.47 per share when it actually produced a loss of $0.27, delivering a surprise of 42.55%.

Over the last four quarters, the company has surpassed consensus EPS estimates three times.

ProQR, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted zero revenues for the quarter ended September 2018, missing the Zacks Consensus Estimate by 100%. This compares to zero revenues a year ago.

The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.

ProQR shares have added about 557.7% since the beginning of the year versus the S&P 500's gain of 3.1%.

What's Next for ProQR?

While ProQR has outperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?

There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.

Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.

Ahead of this earnings release, the estimate revisions trend for ProQR was mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

It will be interesting to see how estimates for the coming quarters and current fiscal year change in the days ahead. The current consensus EPS estimate is -$0.53 on $0.60 million in revenues for the coming quarter and -$1.38 on $3.01 million in revenues for the current fiscal year.

Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 35% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.

[verwijderd] 7 november 2018 15:37

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ProQR Announces Financial Results for the Third Quarter of 2018

LEIDEN, Netherlands and CAMBRIDGE, Mass., Nov. 07, 2018 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced results for the third quarter of 2018.

“During the third quarter, we announced important clinical proof-of-concept data for QR-110, demonstrating a robust improvement in vision in patients with LCA10,” said Daniel A. de Boer, chief executive officer of ProQR. “These results build confidence in QR-110 and the broader ability of oligonucleotides to improve vision in patients with inherited retinal diseases. Based on these encouraging results we are rapidly advancing our pipeline for inherited blindness that includes programs for Usher syndrome, Stargardt’s diseases and FECD. With the recent addition of QR-1123 for autosomal dominant retinitis pigmentosa (adRP) from Ionis Pharmaceuticals we have strategically expanded our pipeline in this key therapeutic area. To fund these activities, we have raised capital that will allow us to fund operations into 2021.”

Third Quarter 2018 Corporate Highlights

Presented positive interim clinical data for lead program QR-110 at the Retinal Degeneration 2018 meeting (RD2018). In the interim analysis of the ongoing Phase 1/2 clinical trial, QR-110 demonstrated rapid and sustained improvement in vision in patients with LCA10, as well as being well-tolerated with no serious adverse events recorded related to treatment or procedure. The Company expects to start a pivotal Phase 2/3 clinical trial during the first half of 2019.
Closed an underwritten public offering of 6,612,500 ordinary shares at a price of $15.75 per share including full exercise of underwriters’ option to purchase 862,500 additional ordinary shares. Gross proceeds totaled approximately $104.1 million.
Delivered two presentations at the Oligonucleotide Therapeutics Society (OTS) conference on clinical data for QR-110 in patients with LCA10 and preclinical data for ProQR’s Axiomer® RNA-editing technology. Abstract titles:
? Proof-of-concept for RNA-editing oligonucleotide QR-110 for treatment of inherited retinal dystrophy in adults and children with LCA10
? Structure-based computational approach for optimizing oligonucleotides for A-to-I editing
Presented at the International Symposium on Usher Syndrome (USH2018) and the Ophthalmology Innovation Summit (OIS) Retina conferences. Abstract titles:
? Splice modulation to treat USH2A-associated retinal degeneration
? RNA treatments targeting rare diseases
A paper with preclinical data for QR-110 targeting Leber’s congenital amaurosis 10 (LCA10) was published in Molecular Therapy – Nucleic Acids, an official journal of the American Society of Gene & Cell Therapy
With current cash on hand of €113.7 million (at September 30, 2018), ProQR’s operations are funded into 2021.

Subsequent Events

In-licensed exclusive worldwide rights for IONIS-RHO-2.5Rx, now QR-1123, from Ionis Pharmaceuticals. QR-1123 is a first-in-class investigational oligonucleotide (gapmer) for the treatment of 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
Received a conditional waiver of the €5 million Innovation credit that was awarded by the Dutch Ministry of Economic Affairs for the Company’s cystic fibrosis program. Consequently, the total repayment, including principal and interest, of €7.2 million (scheduled in three annual payments) has been waived and will be reviewed annually.
Delivered presentations at European Oligonucleotide and Peptide Therapeutics (EuroTIDES) conference. Abstract titles:
? Childhood Blindness due to a Photoreceptor Cilium Defect Treated with an Intravitreal Antisense Oligonucleotide (QR-110)
? Evaluating the impact of sterilization on oligonucleotide based drug products

Financial Highlights

At September 30, 2018, ProQR held cash and cash equivalents of €113.7 million, compared to €48.1 million at December 31, 2017. The increase in cash was due to the closing of an offering of ordinary shares for which net proceeds totaled €84.3 million. Net cash used in operating activities during the three month period ended September 30, 2018 was €4.3 million, compared to €7.6 million for the same period last year.

Research and development costs totaled €6.3 million for the quarter ended September 30, 2018 compared to €7.2 million for the same period last year.

General and administrative costs decreased to €2.6 million for the quarter ended September 30, 2018 compared to €2.8 million for the same period last year.

Net loss for the three month period ended September 30, 2018 was €6.0 million or €0.18 per share, compared to a €10.5 million loss or €0.42 per share for the same period last year. For further financial information for the period ended September 30, 2018, please refer to the financial statements appearing at the end of this release.

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*

About QR-110

QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore normal (wild-type) CEP290 mRNA leading to the production of normal CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 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 by the FDA.

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 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 exclude the genetic defect from 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 in the United States and the European Union.

About QR-1123

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.

Vertraagd	15 apr 2024 22:00
Koers	2,050
Verschil	-0,150 (-6,82%)
Hoog	2,200
Laag	1,910
Volume	319.416
Volume gemiddeld	305.375
Volume gisteren	249.624

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