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ProQR Therapeutics N.V. (PRQR)

NasdaqGS - NasdaqGS Real Time Price. Currency in USD
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5.61+0.34 (+6.55%)
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Previous Close5.27
Open5.65
Bid5.50 x 1000
Ask5.52 x 1000
Day's Range5.34 - 5.78
52 Week Range3.40 - 9.43
Volume3,145,437
Avg. Volume406,245
Market Cap281.571M
Beta (5Y Monthly)0.72
PE Ratio (TTM)N/A
EPS (TTM)-1.31
Earnings DateFeb 24, 2021 - Mar 01, 2021
Forward Dividend & YieldN/A (N/A)
Ex-Dividend DateN/A
1y Target Est21.35
Fair Value is the appropriate price for the shares of a company, based on its earnings and growth rate also interpreted as when P/E Ratio = Growth Rate. Estimated return represents the projected annual return you might expect after purchasing shares in the company and holding them over the default time horizon of 5 years, based on the EPS growth rate that we have projected.
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  • 2 “Strong Buy” Penny Stocks That Could See Outsized Gains
    TipRanks

    2 “Strong Buy” Penny Stocks That Could See Outsized Gains

    Well, it’s official. Joe Biden is now President, and he’ll be backed – at least for the short term – by Democratic majorities in both Houses of Congress. Wall Street is taking the measure of the new Administration, and sees, among its first moves, a boost in fiscal stimulus that is likely to goose consumer spending, bump corporate profits, and provide general economic support in the first half of 2021. Covering the situation for Goldman Sachs is investment strategist David Kostin, who is bullish on the near-term prospects for fiscal stimulus. In light of it, Kostin sets the Goldman outlook for this year at 6.4% GDP growth; he sees continued high growth next year, and sets the 2022 prediction at 4%. These outlook numbers are up from the previously published 5.9% and 3.7%. To this end, Kostin sees S&P 500 reaching 4,300 by year’s end, which would be a gain of 12% from current levels. “Elections have consequences. Democratic control of Washington, D.C. after January 20 will bring greater fiscal spending, faster GDP growth, more inflation, and higher interest rates than we had previously assumed,” Kostin noted. With markets looking up, investors are looking for the stocks that are ready for gains. Penny stocks, equities priced at less than $5 per share, are a natural place to search for potential winners. Their low price means that even a small incremental gain will translate into large percentages. However, before jumping right into an investment in a penny stock, Wall Street pros advise looking at the bigger picture and considering other factors beyond just the price tag. For some names that fall into this category, you really do get what you pay for, offering little in the way of long-term growth prospects thanks to weak fundamentals, recent headwinds or even large outstanding share counts. Taking the risk into consideration, we used TipRanks’ database to find compelling penny stocks with bargain price tags. The platform steered us towards two tickers sporting share prices under $5 and “Strong Buy” consensus ratings from the analyst community. Not to mention substantial upside potential is on the table. AzurRx BioPharma (AZRX) We’ll start with a company specializing in gastrointestinal disease, AzurRx. This company is focused on creating non-systemic, targeted recombinant therapies for GI ailments. AzurRx has a pipeline of three drug candidates, at several levels of the development process. The key pipeline candidate, MS1819, is being investigated as a treatment for exocrine pancreatic insufficiency for patients also suffering from cystic fibrosis. MS1819 is a recombinant lipase, derived from a yeast strain. The drug is designed to target fat molecules in the digestive tract, allowing patients to absorb the broken-down fats for nutritive value. The drug is currently in Phase 2 trials, which are scheduled for completion in the first half of this year. As of January 21, the first two patients in the Phase 2b OPTION 2 extension study have been dosed with the treatment, and the Data Monitoring Committee (DMC) “remains supportive of the program.” In another important development, AzurRx announced earlier this month that it is entering a partnership with First Wave Bio to study oral and rectal formulation of Niclosamide to treat immune checkpoint inhibitor-associated colitis (ICI-AC) and COVID-19 related gastrointestinal infections. The estimated market for Niclosamide as a treatment for COVID-related GI problems exceeds $450 million. Based on multiple potentially significant clinical catalysts as well as its $0.98 share price, several members of the Street think that now is the right time to pull the trigger. Jonathan Aschoff, of Roth Capital, is bullish on AzurRx, basing his longer-term forecasts on the probable success of MS1819. “We base our valuation for AZRX on projected future U.S. sales from MS1819 for the treatment of EPI due to CF and CP, using an initial annual price of about $18,000, a price that is consistent with currently available PERTs. We project MS1819 to be commercialized in the U.S. in 2023, generating sales of $272 million in 2030. Ex-U.S. commercial success for MS1819, or commercial success from the early-stage beta-lactamase program would provide upside to our valuation,” Aschoff noted. The analyst also looks forward to initial clinical results of Niclosamide in COVID-19 GI infection and in ICI-AC potentially, noting: "Niclosamide was FDA approved in 1982 to treat intestinal tapeworm infections and is on the World Health Organization’s list of essential medicines. Given the millions of patients that have taken the drug, the safety profile has largely been established, thereby lowering developmental risk.” Given all of the above, Aschoff rates AZRX as a Buy, and his $7 price target suggests a sky-high 608% upside for the year ahead. (To watch Aschoff’s track record, click here) Overall, the analyst consensus on AZRX shares is a Strong Buy; the stock has 4 recent reviews, including 3 Buys and a single Hold. In addition, the $4 average price target brings the upside potential to 304%. (See AZRX stock analysis on TipRanks) ProQR (PRQR) ProQR is a biotechnology company focused on treatments for congenital progressive blindness. Specifically, the firm is working on medications to reverse a group of genetic sight disorders called inherited retinal diseases. These diseases currently have no effective treatments. The company has a research pipeline of five drug candidates, in varying stages of the research process. The two that are farthest along are QR-110 (Sepofarsen), and QR-421. Of those two, QR-110 is currently in Phase 2/3 studies. This candidate is an RNA therapy designed to correct the most common CEP290 gene mutation causing Leber congenital amaurosis 10 (LCA10). This is a severe genetic retinal disease that affects as many as 3 in 100,000 children. QR-421 is another RNA therapy, this one focused on exon 13 mutations in the USH2A gene. These mutations cause blindness due to retinitis pigmentosa and/or Usher syndrome. QR-421 is in Phase 1/2 studies, with an aim of restoring lost vision or preventing the loss in the first place. Covering the stock for JMP, analyst Jonathan Wolleben points to Sepofarsen as a key component of his bullish thesis. “We continue to feel good about sepofarsen’s chance of success in Illuminate for multiple reasons: 1) Phase 1/2 confirmed the target registrational dose and dosing interval (6 months); 2) patients had clinically significant and durable BCVA improvements after 12 months – pivotal primary endpoint; 3) supportive secondary efficacy measures (FST, mobility); 4) similar responses seen in second treated eyes; 5) long-term safety confirms positive risk/benefit; and 6) Illuminate patient population was enriched based on Phase 1/2 results (baselinevision of >/=hand motion). We assign sepofarsen a 60% POS and model LCA10 as an ~$300M opportunity to PRQR at peak penetration," Wolleben opined. In line with his upbeat outlook, Wolleben puts a $20 price target on the stock, implying a 384% one-year upside, along with an Outperform (i.e. Buy) rating. (To watch Wolleben’s track record, click here) All in all, PRQR gets a unanimous Strong Buy rating from the analyst consensus, based on 3 positive stock reviews. Shares are currently trading for $4.13, and their $20.67 average price target is slightly more bullish than Wolleben’s, suggesting an upside of 400% for the coming 12 months. (See PRQR stock analysis at TipRanks) To find good ideas for penny stocks trading at attractive valuations, visit TipRanks’ Best Stocks to Buy, a newly launched tool that unites all of TipRanks’ equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

  • Is ProQR Therapeutics (NASDAQ:PRQR) Using Debt In A Risky Way?
    Simply Wall St.

    Is ProQR Therapeutics (NASDAQ:PRQR) Using Debt In A Risky Way?

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  • ProQR Completes Enrollment of its Pivotal Trial of Sepofarsen for the Treatment of LCA10
    GlobeNewswire

    ProQR Completes Enrollment of its Pivotal Trial of Sepofarsen for the Treatment of LCA10

    * Top-line results expected H1 2022 * Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindnessLEIDEN, Netherlands & CAMBRIDGE, Mass., Jan. 07, 2021 (GLOBE NEWSWIRE) --  ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today announced it has completed patient enrollment in its Phase 2/3 Illuminate study of sepofarsen for the treatment of Leber Congenital Amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene. With enrollment completed, top-line results are expected in the first half (H1) of 2022.Illuminate is a Phase 2/3 trial, which randomized 36 patients aged eight years or older to receive either sepofarsen at the target registration dose, a low dose, or sham treatment. The primary endpoint of this study is mean change from baseline in Best Corrected Visual Acuity (BCVA) at Month 12, at the registration dose versus sham. This trial is intended to support application for marketing approval of sepofarsen for patients with LCA10 due to the p.Cys998X mutation in the CEP290 gene. “We are pleased to have completed enrollment of the pivotal Illuminate trial of sepofarsen.  This marks an important milestone for ProQR, as well as for the LCA10 and broader inherited retinal disease community. In surpassing our enrollment target, we were able to accommodate the broad interest to participate in the trial,” said Aniz Girach, MD, Chief Medical Officer of ProQR. “This speaks to the fact that there are currently no approved treatments for patients with LCA10. If approved, sepofarsen has the potential to be the first therapy to address this high unmet medical need for patients who would otherwise face blindness. We are grateful to those who have supported our efforts in bringing this trial forward, including our investigators, patients, and caregivers. We look forward to sharing the top-line results in the first half of 2022.”The Illuminate study was initiated based on data from a Phase 1/2 study, which indicated that at Month 12, patients treated with sepofarsen had an improvement in visual acuity, as measured by BCVA. In a subset of patients (n=6) who were treated at the target registration dose, the mean change from baseline for BCVA at Month 12 was -0.93 LogMAR, equivalent to approximately 9 lines improvement (or 45 letters) on the ETDRS chart. In the Phase 1/2 study, concordant improvements in measures of full-field stimulus testing (FST) and mobility were also observed, which are secondary endpoints in the Illuminate trial.“LCA10 is a severe inherited retinal disease that leads to blindness, and for which there is currently no treatment,” said Katarina Stingl, MD, Professor at University of Tuebingen, Center for Rare Eye Diseases (Germany). “Completing enrollment of the Illuminate trial marks an important milestone for the LCA10 community, as well as the IRD community as a whole, as we seek to advance new treatments for this patient group.”Laura Manfre, Chair and Co-Founder of Sofia Sees Hope, a patient advocacy organization dedicated to those affected by LCA and other rare retinal diseases shared, “As the parent of a child with an LCA diagnosis, I was told there was nothing that could be done and that we needed to accept that our daughter would one day be blind.  Now, in early clinical testing we have seen the potential for sepofarsen to make a significant difference for patients with LCA10 due to a mutation in the CEP290 gene. We see hope for individuals living with this disease. We look forward to learning about the results of the Illuminate trial, and continuing to work with ProQR as they advance their pipeline of RNA therapies to potentially help children, adults, and families who are affected by blindness caused by LCA and other rare inherited retinal diseases.”Sepofarsen has been granted orphan drug designation in the United States and the European Union and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.About Leber Congenital Amaurosis 10 (LCA10)Leber Congenital Amaurosis (LCA) is the most common cause of blindness due to genetic disease in children. It consists of a group of diseases of which LCA10 is the most frequent and one of the most severe forms. LCA10 is caused by mutations in the CEP290 gene, of which the p.Cys998X mutation has the highest prevalence. LCA10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA10 because of this mutation.About SepofarsenSepofarsen (QR-110) is being evaluated in the pivotal Phase 2/3 Illuminate trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber 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 leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen 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 and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.About ProQRProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.Learn more about ProQR at www.proqr.com.FORWARD-LOOKING STATEMENTSThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding sepofarsen (QR-110) and the clinical development and the therapeutic potential thereof, including timing of clinical trials and results from such clinical trials. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F.  These risks and uncertainties include, among others, the cost, timing and results of clinical trials and other development activities that may be slowed or halted by the COVID-19 pandemic; the likelihood of our clinical programs being executed on the intended timelines; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials and the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs. 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: Sarah Kiely ProQR Therapeutics N.V. T: +1 617 599 6228 skiely@proqr.com or Hans Vitzthum LifeSci Advisors T: +1 617 535 7743 hans@lifesciadvisors.comMedia Contact: Alison Chen LifeSci Communications T: +1 646 876 4932 achen@lifescicomms.com