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Home  /   Forum   /   ProQR Therapeutics   /   QR010 Cystic Fibrosis

Aandeel ProQR Therapeutics NV OTC:PRQR.Q, NL0010872495

Vertraagde koers (usd) Verschil Volume
3,625   -0,105   (-2,82%) Dagrange 3,460 - 3,800 473.112   Gem. (3M) 1,3M

QR010 Cystic Fibrosis

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20 Posts
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  1. PlayBall10 8 juni 2017 15:21
    Dan ook maar een nieuw draadje voor QR-010 Cystic Fibrosis

    ir.proqr-tx.com/phoenix.zhtml?c=25370...

    ProQR to present QR-010 data at the European Cystic Fibrosis Society Conference and provides an update on the ongoing Phase 1b trial
    Key Updates

    An oral presentation on the final results from the proof-of-concept (PoC) nasal potential difference (NPD) trial will be given by Steve Rowe, MD at the European Cystic Fibrosis Society (ECFS) conference.

    Preliminary data from the Phase 1b study, PQ-010-001 single ascending dose (SAD) cohorts of the ongoing Phase 1b will be presented in a poster, demonstrating single dose safety and evidence of systemic exposure following administration via inhalation in CF patients.

    Cohort 7 is completed and enrollment of the final cohort in the Phase 1b study, PQ-010-001, is expected to be completed in June 2017.

    Topline safety and exploratory efficacy data from the multiple dose cohorts in the Phase 1b trial are expected to be released in September 2017.
    LEIDEN, the Netherlands, June 08, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced presentation of data from two clinical studies of QR-010 in oral and poster sessions at the ECFS conference in Sevilla, Spain from 8 to 10 June 2017. The company also released preliminary data from the ongoing Phase 1b study, demonstrating safety and systemic uptake of QR-010 after a single dose through inhalation.

    Oral presentation on June 9

    Steve Rowe, M.D., professor of Pulmonary, Allergy and Critical Care Medicine at University of Alabama and Director of the Gregory Fleming James Cystic Fibrosis Research Center, and director of the CFF Therapeutics Development Network will give an oral presentation titled “QR-010, an investigational RNA therapeutic, improves CFTR activity in cystic fibrosis subjects homozygous for the F508del mutation [Abstract #WS13.1]”. The presentation will take place on Friday 9 June during the session “New therapies targeting CFTR: what's new from the clinical trials pipeline?” from 15:00 – 16:30 central European time in Sevilla, Spain.

    Poster presentation on June 9

    The Company will also present a poster titled: “QR-010 via inhalation is safe, well-tolerated, and achieves systemic concentrations in a single ascending dose study in subjects with cystic fibrosis homozygous for the F508del CFTR mutation [Poster #40]” during the session “Cell Biology/Physiology/New Therapies” on Friday 9 June 2017 from 14:00 – 15:00 central European time in Sevilla, Spain.

    “QR-010 is an innovative approach to restoring CFTR function in patients with CF due to the F508del mutation. Last year, we demonstrated that QR-010 restores CFTR function as measured by a very specific assay, the nasal potential difference. Now we have shown that QR-010 can be detected in the blood following a single dose inhalation. We believe these results support the potential that QR-010 can treat all manifestations of CF,” said Noreen R. Henig, MD, Chief Medical Officer of ProQR. “I am very pleased that enrollment of the Phase 1b study is expected to be completed this month and we are looking forward to unblinding the study and report the top-line data from this phase 1b trial.”

    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 Type 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 F508del mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is 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 Cystic Fibrosis

    Cystic fibrosis (CF) is the most common fatal inherited disease in the Western world and affects an estimated 65,000 patients worldwide. In people with CF, a defective CFTR gene causes a thick, buildup of mucus in the lungs, pancreas and other organs. In the lungs, the mucus clogs the airways and traps bacteria leading to infections, extensive lung damage and eventually, respiratory failure. There is no cure for CF. Disease manifestations lead to a shortened life expectancy with a median age of death of 27 years. Although over 1,900 CF-causing gene mutations have been identified, approximately 70% of all CF patients are affected by the F508del mutation. Among all CF patients, approximately 50% are homozygous for the F508del mutation.

    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, our ongoing and planned discovery and development of QR-010 and its therapeutic potential, timing of enrollment and results from our clinical trials, and statements regarding the coverage of our patent portfolio, owned and in-licensed, including the duration of patent coverage. 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, 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.

    Contact:
    Smital Shah
    Chief Financial Officer
    T: +1 415 231 6431
    ir@proqr.com
  2. PlayBall10 11 juni 2017 10:07
    Positive Data for Investigational Cystic Fibrosis Therapy
    Published on June 8, 2017
    microscope-lens-research-cd-500
    Data from two clinical studies of an investigational cystic fibrosis therapy were presented at the European Cystic Fibrosis Society (ECFS) conference, according to ProQR Therapeutics.

    The drug, 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 drug demonstrated safety and systemic uptake after a single dose through inhalation, according to oral and poster presentations of an ongoing Phase 1b study.

    An oral presentation titled “QR-010, an investigational RNA therapeutic, improves CFTR activity in cystic fibrosis subjects homozygous for the F508del mutation” was presented on Friday June 9 by Steve Rowe, MD, professor of Pulmonary, Allergy and Critical Care Medicine at University of Alabama and Director of the Gregory Fleming James Cystic Fibrosis Research Center, and director of the CFF Therapeutics Development Network. A poster presentation by ProQR Therapeutics was also made on June 9 titled: “QR-010 via inhalation is safe, well-tolerated, and achieves systemic concentrations in a single ascending dose study in subjects with cystic fibrosis homozygous for the F508del CFTR mutation.”

    “QR-010 is an innovative approach to restoring CFTR function in patients with CF due to the F508del mutation. Last year, we demonstrated that QR-010 restores CFTR function as measured by a very specific assay, the nasal potential difference. Now we have shown that QR-010 can be detected in the blood following a single dose inhalation. We believe these results support the potential that QR-010 can treat all manifestations of CF,” said Noreen R. Henig, MD, Chief Medical Officer of ProQR. “I am very pleased that enrollment of the Phase 1b study is expected to be completed this month and we are looking forward to unblinding the study and report the top-line data from this phase 1b trial.

    More information is available on the ProQR website.
    Bron: www.rtmagazine.com/2017/06/positive-d...
  4. PlayBall10 13 juni 2017 13:20

    ProQR to Participate in a Cystic Fibrosis Panel Discussion during the JMP Securities Life Science Conference

    LEIDEN, The Netherlands, June 13, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that Daniel de Boer, Chief Executive Officer, will participate in a panel discussion focused on emerging treatment options in cystic fibrosis during the JMP Securities Life Science Conference on June 20th at 8:30am. The conference is being held at The St. Regis in New York, NY, USA.

    www.ir.proqr.com/phoenix.zhtml?c=2537...
  5. PlayBall10 29 augustus 2017 13:19

    ProQR Completes Dosing of Cystic Fibrosis Patients in QR-010 Phase 1b Trial

    Key Updates

    •Last patient received their final dose in the PQ-010-001 Phase 1b clinical trial of QR-010 in CF patients with the F508del mutation.
    •Top-line trial data are expected to be issued in a press release post-market close on Monday, September 25, 2017, followed by a conference call.
    •Adult CF patients have received a single dose or multiple doses in the trial conducted at 26 sites in Europe and North America.

    LEIDEN, the Netherlands, Aug. 29, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), today announced that dosing of cystic fibrosis patients in its Phase 1b clinical trial of QR-010 has been completed and top-line data are scheduled to be announced post-market close on Monday, September 25, 2017.

    About the PQ-010-01 Phase 1b Clinical Trial

    The PQ-010-001 study is a Phase 1b, 28-day, randomized, double-blind, placebo-controlled safety and tolerability trial, conducted in patients that have cystic fibrosis (CF) due to two copies of the F508del mutation (homozygotes). A total of 4 dose levels were studied: 6.25, 12.5, 25 and 50mg of QR-010 in solution per dose administered via inhalation. The study design consisted of 8 cohorts of 8 patients for a total of 64 patients. In each cohort, 6 patients received QR-010 and 2 patients received 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. Patients included in the study were adult patients with mild CF disease with a baseline predicted FEV1 value above 70%. The Phase 1b study is a first-in-human trial designed to primarily assess safety, tolerability and pharmacokinetics of QR-010. A number of exploratory efficacy endpoints are also being assessed including sweat chloride, weight gain, change in CFQ-R Respiratory Symptom Score and FEV1, however, the study is not powered for statistical significance on the exploratory efficacy endpoints.

    “QR-010 has the potential to be an innovative RNA therapy for patients with CF due to the F508del mutation. Completion of the Phase 1b study is an important step in development, and builds upon the pre-clinical data and positive PQ-010-002 study where QR-010 demonstrated a direct effect on restoring CFTR function,” said Noreen R. Henig MD, Chief Medical Officer at ProQR. “We are grateful to the patients and the medical community who participated in this early trial. ProQR is committed to creating meaningful RNA therapies for patients with CF.”

    Conference Call

    Following the press release announcing top-line data, scheduled for post-market close on Monday, September 25, 2017, the Company will host a conference call and webcast. The details of the conference call will be included in the press release and posted to the Company’s website.
    www.ir.proqr.com/phoenix.zhtml?c=2537...

  11. [verwijderd] 25 september 2017 22:09
    www.ir.proqr.com/phoenix.zhtml?c=2537...

    ProQR Announces Positive Top-Line Results from a Phase 1b Study of QR-010 in Subjects with Cystic Fibrosis
    Key updates

    QR-010 was observed to be safe and well-tolerated across all doses in this trial with no serious adverse events related to treatment.
    A clinically meaningful improvement of CF respiratory symptoms, as measured by CFQ-R RSS, was observed in 3 out of 4 multiple dose groups with a mean improvement of 13.0 to 19.2 points compared to placebo. In a pre-defined subgroup of subjects with a lower lung function at baseline, the mean improvement was up to 27.5 points compared to placebo.
    Magnitude of the benefit observed in CFQ-R RSS for these dose groups exceeded the established minimal clinically important difference of 4.0 points.
    In the same multiple dose groups a supportive trend of improved lung function was observed up to 4.0% mean absolute change in ppFEV1 compared to placebo. In a pre-defined subgroup of subjects with a lower lung function at baseline a mean absolute change in ppFEV1 was observed up to 10.9% compared to placebo.
    After inhaled administration in some dose groups, QR-010 was detected in the blood.
    ProQR and the Cystic Fibrosis Foundation Therapeutics intend to expand their partnership to explore the inhaled oligonucleotide platform to target stop-codon mutations in CF.
    Management will discuss the top-line results during a conference call today at 5 pm ET.
    LEIDEN, the Netherlands, Sept. 25, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced positive preliminary top-line results from a Phase 1b safety and tolerability clinical trial (Study PQ-010-001; NCT02532764) of QR-010, a novel investigational RNA therapeutic in subjects with cystic fibrosis (CF). Full data from the trial will be presented at the North American CF Conference (NACFC) on November 2-4, 2017.

    Study PQ-010-001 was a Phase 1b, randomized, double-blind, placebo-controlled, dose escalation study to evaluate the safety, tolerability and pharmacokinetics of QR-010 in adult subjects with CF homozygous for the F508del mutation. This trial studied 4 dose levels of QR-010 administered via inhalation in 4 single-dose and 4 multiple-dose groups. A number of exploratory efficacy endpoints were assessed in the multiple dose groups: respiratory symptoms (as measured by a validated patient-reported outcome tool, the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score, or CFQ-R RSS), lung function (as measured by mean absolute change in percent predicted forced expiratory volume in 1 second, or ppFEV1), sweat chloride test and weight change. This study included subjects with, on average, a normal lung function at baseline (mean ppFEV1 86%, range 69-116%). As therapeutic trials typically study subjects with normal-to-severe lung function at baseline (ppFEV1 <90%), a subgroup was pre-defined to analyze the exploratory efficacy endpoints in this population. The trial recruited 70 participants and was conducted at 23 sites in 10 countries in Europe and North America.

    J. Stuart Elborn, the principal investigator of the study, Clinical Chair in Respiratory Medicine at Imperial College, Consultant at Royal Brompton Hospital, and immediate past-president of the European Cystic Fibrosis Society, added, “QR-010 exceeded expectations in this study as an innovative investigational therapy for the treatment of cystic fibrosis for which the need remains high. The improvements demonstrated in reduction of respiratory symptoms are very encouraging and intriguing and of course of enormous importance to people with CF. The results of this study together with the previous proof of concept study are strongly supportive of the further development of QR-010.”

    Top-Line Results

    In this trial QR-010 was observed to be safe and well-tolerated across all doses, with an overall safety profile similar to placebo. There were no serious adverse events related to treatment. After inhaled administration in some dose groups, QR-010 was detected in the blood. Subjects who received QR-010 in the 6.25, 12.5 and 25 mg multiple dose groups reported fewer respiratory symptoms after 4 weeks of treatment as measured by the increased CFQ-R RSS, with mean improvements of 13.0, 19.2 and 14.3 points, respectively, compared to placebo. The effect was more pronounced in the pre-defined subgroup of subjects with a lower lung function at the start of the study (baseline ppFEV1 70-90%) with a mean increase of up to 27.5 points compared to placebo. These improvements exceeded the minimal clinically important difference (MCID) of 4.0 points. A supportive trend of improved lung function was observed in the same multiple dose groups, as measured by mean absolute change in ppFEV1 compared to placebo. The trend was stronger in the subgroup of subjects with a lower lung function at baseline. The table below summarizes the data per multiple dose group. As expected, no effect was observed on sweat chloride and weight.

    Per protocol population
    Pre-defined subgroup of subjects with
    baseline ppFEV1 70-90%
    CFQ-R RSS
    ppFEV1
    CFQ-R RSS
    ppFEV1
    Groups
    (12 doses
    over 4
    weeks) n mean change
    vs placebo
    (p-value) 95% CI mean absolute
    % change vs
    placebo
    (p-value) 95% CI n mean change
    vs placebo
    (p-value) 95% CI mean absolute
    % change vs
    placebo
    (p-value) 95% CI
    6.25 mg 6 +13.0 (0.0585) -0.5 ; 26.4 +1.2 (0.7266) -6.0 ; 8.4 3 +23.2 (0.0315) 2.4 ; 44.1 +8.0 (0.1613) -3.6 ; 19.5
    12.5 mg 6 +19.2 (0.0072) 5.7 ; 32.7 +4.0 (0.2626) -3.2 ; 11.2 4 +27.5 (0.0095) 7.9 ; 47.1 +10.9 (0.0461) 0.2 ; 21.6
    25 mg 6 +14.3 (0.0399) 0.7 ; 27.9 -0.2 (0.9664) -7.3 ; 7.1 5 +20.3 (0.0334) 1.9 ; 38.7 +4.7 (0.3410) -5.5 ; 14.8
    50 mg 5 +3.5 (0.6182) -10.7 ; 17.6 -0.6 (0.8749) -8.2 ; 7.0 4 +10.9 (0.2463) -8.4 ; 30.2 +3.7 (0.4745) -7.0 ; 14.3
    Placebo 8 -6.5 -15.3 ; 2.4 -0.8 -5.5 ; 3.9 4 -11.8 -25.5 ; 2.0 -3.8 -11.3; 3.8
    The Phase 1b study achieved its goals for evaluation of QR-010 including demonstrating safety and tolerability across a range of doses, identified a dosing window, exhibited uptake of the RNA oligonucleotide into circulation following inhalation, and demonstrated early signals of clinical efficacy.

    “The positive results in the first two clinical trials of QR-010 significantly increase the confidence that QR-010 has the potential to become an effective treatment of CF. The improvement in CFQ-R RSS with the supportive FEV1 data as shown in this Phase 1b trial is very exciting,” said Noreen R. Henig, M.D, Chief Medical Officer at ProQR. “I want to thank the entire CF community including those living with CF, clinical investigators, scientists, the CF Foundation, the European CF Society and the US and European therapeutic development networks for their unwavering commitment.”
  12. [verwijderd] 25 september 2017 22:09
    vervolg:

    Partnership with Cystic Fibrosis Foundation Therapeutics (CFFT)

    ProQR and CFFT entered into a partnership in 2014 to develop QR-010 for people with CF due to the F508del mutation. The initial partnership included support for the Phase 1b trial as well as the NPD proof of concept study that reported positive results in 2016. Based on the results of the clinical trials of QR-010, ProQR and CFFT intend to expand the partnership to explore the inhaled oligonucleotide platform for stop-codon mutations (also called “Class I” mutations) in CFTR. Stop-codon mutations cannot be targeted with small molecule potentiator or corrector molecules, and therefore have a high unmet medical need. ProQR intends to target these mutations using its proprietary Axiomer® technology, which has shown compelling data in non-clinical studies, to repair the stop-codon mutations in the RNA, leading to removal of the premature stop-codon. Approximately 12,000 patients, accounting for 15% of CF patients in the western world, have a stop-codon mutation leading to a severe form of CF.

    ”The results of this Phase 1b trial support QR-010’s potential to be a meaningful therapy for people with cystic fibrosis,” said Daniel A. de Boer, Chief Executive Officer at ProQR. ”With these results in hand we are designing a path forward for the development of QR-010, either independently or with a potential partner. Furthermore we are looking forward to expanding our partnership with the CFFT to explore the inhaled oligonucleotide platform also for people that have CF due to stop-codon mutations.”

    About the PQ-010-001 Trial

    The Phase 1b study was a trial designed to assess safety, tolerability and pharmacokinetics of QR-010. 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. Subjects 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 design planned 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.

    QR-010 Milestones

    Technology for QR-010 in-licensed from Massachusetts General Hospital in 2012.
    Partnership with the CFF established to develop QR-010 for patients with the F508del mutation.
    In vitro proof of concept in three F508del CF assays.
    In vivo proof of concept in two assays, including nasal potential difference (NPD).
    Pre-clinical in vitro and in vivo proof of concept established for efficient inhaled delivery to the CF diseased lung in collaboration with the University of North Carolina at Chapel Hill.
    QR-010 granted fast-track status by the FDA and orphan drug designation from FDA and the European Commission.
    Program received funding from the European Union’s Horizon 2020 research and innovation programme.
    Clinical trial PQ-010-002 top-line data shows significant improvement of CFTR function as measured by NPD in subjects homozygous for the F508del mutation following topical administration of QR-010.
    Grant of two key patents, protecting QR-010 in the US and Europe.
    Preliminary top-line data from clinical trial PQ-010-001 shows QR-010 is detected in the blood after inhaled administration, was observed to be safe and well-tolerated and shows signals of efficacy.
    Full data for PQ-010-001 will be presented at the NACFC (November 2-4, 2017).
    Conference Call and Webcast Information

    ProQR will host a conference call and webcast today at 5:00 PM Eastern Time (ET) or 11:00 PM Central European Time (CET). The conference call will be webcast live and a link to the webcast can be accessed through ProQR’s website (www.proqr.com) and in the “Investors” section under “Events and Presentations”. To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company’s website. To participate in the conference call, please dial in 5-10 minutes prior to start time and reference Conference ID 8468989:


    Country Toll Free Direct
    US 1 877 280 2296 +1 646 254 3365
    Netherlands 0800 020 2576 +31 (0) 20 713 2789
    United Kingdom 0800 279 4977 +44 (0) 20 3427 1919
    Germany 0800 589 2674 +49 (0) 69 2222 10620
    Belgium 0800 58032 +32 (0) 2 400 3463
    Switzerland 0800 345 603 +41 (0) 44 580 7214
    France 0805 631 580 +33 (0) 1 7677 2222
    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 received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.

    About Cystic Fibrosis

    Cystic fibrosis (CF) is the most common fatal inherited disease in the Western world and affects over 75,000 patients worldwide. In people with CF, a defective CFTR gene causes a thick buildup of mucus in the lungs, pancreas and other organs. In the lungs, the mucus clogs the airways and traps bacteria leading to infections, extensive lung damage and eventually, respiratory failure. There is no cure for CF. Disease manifestations lead to a shortened life expectancy with a median age of death of 30 years. Although over 1,900 CF-causing gene mutations have been identified, approximately 85% of all CF patients are affected by the F508del mutation. Among all CF patients, approximately 50% are homozygous for the F508del mutation.
  14. [verwijderd] 2 oktober 2017 22:42
    quote:

    PlayBall10 schreef op 2 oktober 2017 15:26:

    ProQR’s Inhaled RNA Therapy Improves CF Symptoms in Phase 1 Clinical Trial
    cysticfibrosisnewstoday.com/2017/09/2...
    ProQR’s Inhaled RNA Therapy Improves CF Symptoms in Phase 1 Clinical Trial
    SEPTEMBER 28, 2017 Magdalena KegelBY MAGDALENA KEGEL IN NEWS.
    ProQR’s Inhaled RNA Therapy Improves CF Symptoms in Phase 1 Clinical Trial
    Click here to receive CF news via e-mail

    ProQR Therapeutics says its investigational drug QR-010 improved respiratory symptoms in 70 adults with cystic fibrosis (CF) who participated in a Phase 1b trial of the inhaled therapy.

    Researchers saw the biggest improvements in patients with a lower lung function at the beginning of the study, which only targeted patients with the F508del mutation.

    “QR-010 exceeded expectations in this study as an innovative investigational therapy for the treatment of cystic fibrosis for which the need remains high,” J. Stuart Elborn, the study’s principal investigator clinical chair in respiratory medicine at Imperial College London, said in a press release. Elborn is also a consultant at England’s Royal Brompton Hospital and past president of the European Cystic Fibrosis Society.

    QR-010 is an RNA-based drug engineered to bind to mutated CFTR messenger RNA (an intermediate between gene and protein) to restore its function.

    The trial (NCT02564354) randomly assigned patients with two F508del mutations to one of four doses of QR-010 or a placebo. The trial included four single-dose and four multiple-dose groups.

    In three out of four multiple-dose groups, researchers observed an average improvement of 13.0 to 19.2 points — using the Cystic Fibrosis Questionnaire-Revised Respiratory Symptoms Scale (CFQ-R RSS) — compared to placebo. Those with a lower lung function, however, improved by an average 27.5 points.

    Researchers judge a 4.0 point improvement to be clinically meaningful.

    Meanwhile, the study also measured lung function using percent predicted forced expiratory volume in one second (ppFEV). QR-010 improved lung function by 4.0 percent, which they considered a positive trend. In those with poorer lung function from the outset, the difference was 10.9 percent.

    “The results of this Phase 1b trial support QR-010’s potential to be a meaningful therapy for people with cystic fibrosis,” said Daniel A. de Boer, ProQR’s CEO. “With these results in hand, we are designing a path forward for the development of QR-010, either independently or with a potential partner.”

    The Dutch pharmaceutical company partnered with Cystic Fibrosis Foundation Therapeutics (CFFT) in 2014 to develop QR-010. CFFT, which is based in Bethesda, Maryland, initially said it would support the Phase 1b study and a proof-of-concept study of nasal potential difference — a common CF test.

    But the positive data prompted CFFT to expand the partnership, which will now include inhaled RNA drugs for Class I — also called stop-codon — mutations in the CFTR protein. These mutations are not amenable to treatment with small molecule correctors; such patients have a continuously high unmet need for new treatments.

    “The improvements demonstrated in reduction of respiratory symptoms are very encouraging and intriguing and of course of enormous importance to people with CF,” Elborn said. “The results of this study together with the previous proof of concept study are strongly supportive of the further development of QR-010.”

    ProQR will report the full trial data at the Nov. 2-4 North American CF Conference (NACFC) in Indianapolis.
  17. [verwijderd] 10 oktober 2017 12:56
    mooie uitdaging voor ProQR:

    fd.nl/economie-politiek/1221658/midde...

    Middel tegen taaislijmziekte definitief niet in basispakket
    Het medicijn Orkambi tegen cystische fibrose, beter bekend als taaislijmziekte, komt definitief niet in het basispakket. Het Amerikaanse farmaconcern Vertex bleek niet bereid het middel tegen een 'maatschappelijk aanvaardbare prijs' aan te bieden. Dat heeft het ministerie van Volksgezondheid, Welzijn en Sport maandag gemeld.
    Uiterste inspanning
    In een toelichting stelt demissionair zorgminister Edith Schippers nu dat zij 'een uiterste inspanning heeft gedaan om tot een positieve uitkomst te komen'. 'Helaas heb ik moeten constateren dat de prijs die de fabrikant vraagt onaanvaardbaar hoog blijft.'
    Het middel zou op jaarbasis €170.000 per patiënt kosten, waardoor bij circa 750 gebruikers de zorgkosten met zeker €84 mln zouden stijgen. Schippers zegt de 'bijzonder onbevredigende uitkomst' van de onderhandelingen te betreuren. 'Ik was en ben daarom altijd bereid de gesprekken te hervatten als de mogelijkheid er is om tot een overeenkomst te komen.'
    Volgens VWS heeft medicijnfabrikant Vertex 'geen inzicht geboden waarom de prijs zo hoog zou moeten zijn', zeker gezien de 'betrekkelijk bescheiden effecten' van het middel, zoals door het Zorginstituut Nederland is vastgesteld. In mei maakte het ministerie bekend dat het Orkambi, als eerste geneesmiddel ooit, weigert te vergoeden omdat het te duur is. Het ministerie kondigde toen aan met Vertrex over de prijs te gaan onderhandelen.
    Onacceptabel
    In een reactie zegt de Nederlandse Cystic Fibrose Stichting (NCFS) dat het besluit 'onnoemelijk hard' aankomt. De patiëntenorganisatie noemt het 'onacceptabel' dat patiënten een kans wordt ontnomen op 'een mogelijk grote verbetering in hun gezondheid'.
    De Nederlandse Cystic Fibrose Stichting overweegt het besluit met een kort geding aan te vechten, op basis van twee argumenten: recht op gezondheid en rechtsongelijkheid in Europa. Het middel wordt in een groot aantal EU-landen wel vergoed, waaronder Duitsland, Frankrijk, Italië en Oostenrijk. Ook de NCFS vindt het middel te duur, maar noemt het 'onbegrijpelijk' dat beide partijen er na acht maanden onderhandelen niet zijn uitgekomen.
    Diagnostische test
    Met zorgverzekeraars en behandelaars onderzoekt patiëntenorganisatie NCFS wegen om het middel alsnog vergoed te krijgen. Belangrijke stap daarbij is het argument van het Zorginstituut te ontzenuwen dat het middel bescheiden effecten heeft. Daartoe wordt een diagnostische test op individuele basis ontwikkelt die aantoont of een patiënt baat heeft bij het middel .
    Naar schatting lijden 1550 mensen aan de erfelijke taaislijmziekte, waarbij in alle organen de slijmproductie verstoord is. Vooral in de longen leidt taai slijm tot verstoppingen, waardoor uiteindelijk de longen niet meer functioneren. Volgens de woordvoerder van de patiëntenorganisatie verstrekt Vertex op beperkte schaal het middel gratis, 'vanwege compassie met ernstig zieke patiënten'. Circa 750 patiënten hebben een zodanig erfelijke aandoening dat zij baat hebben bij het medicijn.
  19. [verwijderd] 9 november 2017 21:42
    quote:

    PlayBall10 schreef op 8 november 2017 16:28:

    cysticfibrosisnewstoday.com/2017/11/0...
    #NACFC2017 – Cystic Fibrosis Experts from 46 Countries Gathering in Indianapolis for NACFC Convention
    NOVEMBER 1, 2017 Larry LuxnerBY LARRY LUXNER IN CYSTIC FIBROSIS, NEWS.
    #NACFC2017 – Cystic Fibrosis Experts from 46 Countries Gathering in Indianapolis for NACFC Convention
    Click here to receive CF news via e-mail

    About 4,400 researchers, doctors, patients and others from 46 countries are converging on Indianapolis for the 31st Annual North American Cystic Fibrosis Conference — the world’s largest gathering dedicated to cystic fibrosis research and care.

    The Nov. 2-4 event at the Indiana Convention Center will feature more than 350 sessions and roundtable discussions, along with 50 exhibitors and more than 700 poster presentations. It’s being organized by the Cystic Fibrosis Foundation, or CFF.

    “When the CFF was founded in 1955, parents were told to enjoy their children, because they often didn’t live past elementary school,” Anne Willis, vice president of the foundation’s patient access programs, told Cystic Fibrosis News Today at the organization’s headquarters in Bethesda, Maryland.

    “But there have been some amazing improvements over the last few years in terms of healthcare and available treatments,” she said. “People are graduating, getting married, holding jobs — and reaching milestones they could have never imagined before.”

    Anne Willis
    Anne Willis, senior director of policy and advocacy at the Cystic Fibrosis Foundation in Bethesda, Maryland. (Photos by Larry Luxner)
    The conference, which began in 1986 with 300 participants, focuses on advances in CF care, breakthroughs in research, the use of patient data, and promising routes to a cure. This year’s agenda will include more than 60 sessions, with tracks ranging from faulty versions of the CFTR gene that causes the disease, to airways physiology, to nursing, nutrition and respiratory therapy.

    Conference-wide presentations will be made on lung transplants, gene editing, next-generation therapies and personalized medicine.

    There will also be a panel on pregnancy and CF. That’s because more women with CF are reaching adulthood, creating a new focus on reproductive health and family planning.

    ProQR Therapeutics, a Dutch biotech company that develops RNA-based treatments for CF, epidermolysis bullosa and other rare genetic diseases, will be among the companies discussing a potential therapy it has developed — QR-010. The presentation will focus on the results of a Phase 1b trial of the mist-formulation treatment. The trial evaluated the treatment’s safety, and participants’ ability to tolerate it.

    Preliminary indications are that QR-010 improves patients’ ability to deal with their symptoms, the company has reported.

    QR-010 is designed to repair abnormal versions of the CFTR gene. The goal is to generate normal CFTR protein in people with one or two copies of the gene’s most widespread abnormality — the F508del mutation.

    Stuart Elborn, immediate past president of the European Cystic Fibrosis Society, will make the QR-010 presentation, titled “A first-in-human, Phase 1B, dose-escalation study of QR-010, a novel antisense oligonucleotide administered in subjects with cystic fibrosis homozygous for the F508del CFTR mutation.”

    Elborn, a consultant at London’s Royal Brompton Hospital who also is clinical chair of respiratory medicine at Imperial College, will discuss the therapy during a Nov. 4 workshop titled “Toward the Goal of a One-Time Cure: Challenges & Opportunities.”

    Indianapolis
    Downtown Indianapolis will host the 31st annual NACFC, set for Nov. 2-4.
    ProQR will also conduct a poster session presentation on QR010 on Nov. 2. And at 8:30 p.m. Nov. 2, it will host an investor and analyst event. This will cover additional findings of the Phase 1b study of QR-010 and update participants on other therapy candidates in ProQR’s pipeline.

    All the conference abstracts appear in the online edition of Pediatric Pulmonology.

    CF, the most common fatal inherited disease in the Western world, has no cure. A defective CFTR gene causes a thick buildup of mucus in the lungs, pancreas and other organs in people with the disease. The mucus clogs the airways and traps bacteria, leading to infections, extensive lung damage and eventually respiratory failure.

    Although scientists have identified more than 1,900 CF-causing genetic mutations, the F508del mutation affects about 85 percent of the world’s 75,000 or so CF patients.

    ProQR created an aerosol device that patients can use to deliver QR-010 directly into their lungs. The mist format increases the therapy’s effectiveness.

    The U.S. Food and Drug Administration and the European Union have granted QR-010 orphan drug designation, and the FDA fast-tracked the therapy in July 2016. The designations are aimed at speeding up the therapy’s approval process.

    “We started this company five years ago to develop a therapy that would make CF patients like my son better,” ProQR’s CEO, Daniel A. de Boer, told CF News Today. “We are very pleased that QR-010 has demonstrated in this clinical trial to do exactly that. With these positive results in hand, we aim to advance QR-010 for the F508del mutation and expand to develop therapies for certain Class 1 stop-codon mutations that cause cystic fibrosis.”
  20. PlayBall10 13 november 2017 13:21

    ProQR Doses First LCA 10 Patient in Clinical Trial of QR-110, ProQR’s Lead Program for Genetic Blindness

    Key Updates

    •The first patient has received the first dose of QR-110 in the Phase 1/2 safety & efficacy clinical trial (PQ-110-001: NCT03140969) in children and adults with Leber’s congenital amaurosis 10 (LCA 10).
    •LCA 10 is one of the most prevalent forms of gene-related blindness in children worldwide and currently there are no therapies commercially available or in clinical development for this disease.
    •QR-110 has received fast track designation by the U.S. Food and Drug Administration (FDA) and has been granted orphan drug designation in the United States and European Union.
    •Interim safety and efficacy trial results from the majority of patients after 6 months of treatment are expected in 2018, full 12 month treatment data from all patients are expected in 2019.

    LEIDEN, the Netherlands, Nov. 13, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that the first patient has been dosed in the Phase 1/2 open-label trial assessing the safety, tolerability, pharmacokinetics and efficacy of QR-110. The trial will enroll approximately six adults and six children who have Leber’s congenital amaurosis 10 (LCA 10) due to the p.Cys998X mutation in the CEP290 gene. Subjects will receive a dose of QR-110 every three months for a total of four doses in one eye. The trial is planned to be conducted at three specialized centers: the University of Iowa, Iowa City, IA, US, the Scheie Eye Institute at the University of Pennsylvania, Philadelphia, PA, US and the Ghent University Hospital, Ghent, Belgium.

    Benjamin Yerxa, PhD., Chief Executive Officer at Foundation Fighting Blindness, stated, “We are delighted with the launch of ProQR’s clinical trial for its treatment for people with devastating vision loss caused by the p.Cys998X mutation in CEP290. There are no other options for these patients, and furthermore, we believe most emerging gene replacement technologies do not have the capacity to deliver the large CEP290 gene to the retina.”

    QR-110 is ProQR’s lead program in the ophthalmology pipeline that also includes two programs for Usher syndrome, a program for Fuchs endothelial corneal dystrophy and a program for Stargardt’s disease. QR-110 is ProQR’s second program to enter clinical development, following QR-010, which is being developed for the most common mutation causing cystic fibrosis.

    “This announcement recognizes an important next step towards achieving our goal of developing precision medicines based on molecular diagnostics and our RNA therapeutic platform,” said David M. Rodman, MD, Chief Development Strategy Officer of ProQR. “The QR-110 program is the first in a planned series of ophthalmology trials utilizing our RNA therapy platform to target the underlying cause of blindness in patients with inherited forms of retinal dystrophy. We expect this trial will give us fundamental information regarding the safety, efficacy and developability of QR-110 in adults and children with LCA 10.”

    Key facts on QR-110
    •QR-110 aims to delay the progression of the disease or restore vision in people with LCA 10 due to the p.Cys998X mutation in the CEP290 gene.
    •QR-110 is a single stranded RNA oligonucleotide designed to restore wild-type or normal CEP290 mRNA.
    •In pre-clinical studies of QR-110, it was shown to convert close to 100% of the mutant mRNA to wild-type in a homozygous optic cup organoid model.
    •A long half-life in the eye allowing for infrequent dosing.
    •Administered through intravitreal administration, which is considered a routine procedure.
    •A ProQR sponsored pre-evaluation/retrospective natural history study collecting data of 22 LCA 10 patients over a period of 16 years was completed in 2017 (Samuel G. Jacobson et al; Outcome Measures for Clinical Trials of Leber Congenital Amaurosis Caused by the Intronic Mutation in the CEP290 Gene. Invest. Ophthalmol. Vis. Sci. 2017;58(5):2609-2622.)
    www.ir.proqr.com/phoenix.zhtml?c=2537...

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