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Should see tutes adding or buying PRQR shares all week. Pension plans, University endowment funds, mutual funds & hedge funds.
That's equal to half their market cap right now. Wait until this sinks in. I've been saying a confirmation of their RNA approach alone could be worth $40/sh here.
SEC filing 424B5 > closing of last month's shelf placement announcement possibly today.
PRQR
PT > $20. > Stifel analyst Dae Gon Ha initiates coverage on ProQR Therapeutics (NASDAQ:PRQR) with a Buy rating and announces Price Target of $20.
Milestone payments of up to $115 million plus royalties on future sales by Yarrow for an exclusive worldwide license and discovery collaboration on a non-ophthalmology target.
ProQR Therapeutics and Yarrow Biotechnology, an RTW Investments, LP Incubated Company, Announce Exclusive Worldwide License and Discovery Collaboration for Undisclosed Target
7:00 am ET May 4, 2021 (Globe Newswire) Print
-- Yarrow Biotechnology, Inc., a newly formed biotechnology company backed by RTW Investments, LP, will lead development of the program
-- ProQR Therapeutics is eligible to receive upfront and milestone payments of up to $115 million plus royalties on future sales by Yarrow for an exclusive worldwide license and discovery collaboration on a non-ophthalmology target
RTW Investments, LP ("RTW"), a global, full life-cycle investment firm that focuses on identifying transformational and disruptive innovations across the biopharmaceutical and medical technologies sectors, and ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced that Yarrow Biotechnology, Inc. ("Yarrow") , a company newly created by RTW, has in-licensed exclusive rights to ProQR's antisense oligonucleotide technology (ASO) to develop and commercialize potential therapies for an undisclosed non-ophthalmic target. Yarrow is focused on developing ASO-based therapeutics for disorders with high unmet need.
Roderick Wong, MD, Managing Partner and Chief Investment Officer of RTW, said: "We believe RNA-based therapies hold great promise to treat genetically-defined diseases. We are excited to partner with ProQR to in-license the first target to be developed in Yarrow's pipeline."
Under the terms of the agreement, ProQR is eligible to receive up to $115 million of upfront and milestone payments, plus single digit percentage royalties on the net sales of any resulting products during the royalty term. ProQR will also have the right to receive an undisclosed percentage of equity in the form of shares of common stock of Yarrow. ProQR will be responsible for certain preclinical activities with reimbursement for the research costs by Yarrow, while Yarrow will be responsible for continuing development of the program and commercialization activities.
Gerard Platenburg, Chief Innovation Officer at ProQR, will join Yarrow's board of directors.
Peter Fong, PhD, Head of Company Creation at RTW, said: "Our partnership with ProQR fits perfectly into Yarrow's mission to develop first-in-class ASO-based therapies for genetically-defined diseases. We look forward to a long and productive partnership."
Daniel A. De Boer, Founder and CEO of ProQR, said: "As we focus ProQR on our core genetic eye disease strategy, we are pleased to partner with Yarrow to advance our technology in applications outside the eye, while generating value from this partnership and the broad applicability of our platform."
About RTW Investments, LP
RTW Investments, LP ("RTW") is a New York-based, global, full life-cycle investment firm that focuses on identifying transformational and disruptive innovations across the biopharmaceutical and medical technologies sectors. As a leading partner of industry and academia, RTW combines deep scientific expertise with a solution-oriented investment approach to support emerging medical therapies and the companies and academics developing them.
About Yarrow Biotechnology, Inc.
Yarrow Biotechnology, Inc. ("Yarrow") is a newly formed biotechnology company developing antisense oligonucleotide-based therapeutics for disorders with high unmet need. Yarrow has established a proprietary target discovery engine leveraging large-scale human genetics studies to uncover novel targets where there is a significant unmet need.
Yarrow is the latest new company created and backed by RTW Investments, LP.
About ProQR
ProQR 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 Statements for ProQR
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. Such forward-looking statements include, but are not limited to, statements regarding the collaboration with RTW and Yarrow and the intended benefits thereof, including milestone and royalty payments from commercial product sales, if any, from the products covered by the collaboration and the issuance of equity in Yarrow to ProQR, as well as the potential of our technologies and product candidates. 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 preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted by the COVID-19 pandemic; the likelihood of our clinical programs being executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; 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; 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; the ability to secure, maintain and realize the intended benefits of collaborations with partners; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; and general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties. 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.
For 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: +1 617 430 7578
hans@lifesciadvisors.com
Media Contact:
Cherilyn Cecchini, MD
This will only keep going as more find it imo
ProQR Therapeutics started at buy with $20 stock price target at Stifel Nicolaus
Has data for 3rd program adRP later this year, pivotal LCA10 data 1H 22
Two drugs in phase II / III.
One of them (sepofarsen) already has papers published on the positive effect at 15 months. Confirmation of positive sepofarsen data will send this to $10+ near term imo.
My target by 2022 is $18/share.
Pushing 11k shares and adding
Nice.
I added at 6.13
Back in at $6.24 lego
I believe we see $10+ here very soon.
ProQR's sepofarsen shows promise in blindness study
Apr. 13, 2021 4:34 PM ETProQR Therapeutics N.V. (PRQR)By: Aakash Babu, SA News Editor
ProQR Therapeutics (NASDAQ:PRQR) highlights case study describing durable vision improvement after treatment with sepofarsen in a clinical trial for CEP290 mediated Leber congenital amaurosis 10 (LCA10), in the scientific journal Nature Medicine.
Sepofarsen (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 c.2991+1655A>G mutation in the CEP290 gene.
Leber congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children.
"Treatment with sepofarsen resulted in substantial, concordant and enduring improvement in more than a dozen different subjective and objective measurements of visual function as well as retinal structure," lead author Professor Artur V. Cideciyan said.
Published results highlight a patient who is homozygous for the c.2991+1655A>G mutation in CEP290 and was part of a larger cohort in the Phase 1/2 clinical trial. The patient was studied for 15 months after intravitreal treatment with sepofarsen.
Concordant measures of visual function and retinal structure including visual acuity, light sensitivity and visual fields, reached a substantial efficacy peak near three months after injection and remained better than baseline at 15 months.
Shares up more than 2% post market.
https://seekingalpha.com/news/3681528-proqr-highlights-publication-in-nature-medicine-for-sepofarsen-in-blindness-study
An End to This Rare Syndrome Could Be an Opportunity for Investors
https://www.fool.com/investing/2021/04/07/an-end-to-this-rare-syndrome-could-be-an-opportuni/?source=iedfolrf0000001
Exactly and always a given the SP will come down on an offering, the smart money buys on a selloff when the fundamental have not changed.
Seems pretty silly for retail to always dump "an offering" then. I guess retail gets upset that they spent $9 on shares that institutions get at $6.50 after the good news. Yes you own less proportional shares but the company is now cash rich through 2022+.
I can see this churning $6.50-8.00 until next material event or catalyst.
Large blocks are bought by the institutions wanting a higher percentage of the company shares rather than buying in the open market, driving up the share price.
How can an offering close without volume equal to the offering amount? Who bought the 13M shares if not from the open market the last 3 days.
Demand strong on the offering and now that it’s closed, it’ll be interesting to see where things go from here.
Good Luck!
This is a $20 stock in time, might be years not months but with the right news...
Your most welcome. (-;
Great article. Thanks for posting it.
A single injection reverses blindness in patient with rare genetic disorder
https://medicalxpress.com/news/2021-04-reverses-patient-rare-genetic-disorder.html
Sorry to hear that.
I was holding heavy at under $5, never sold a share (in the run, but sold some before it) but did sell otm (covered) calls at the end of the run for nice premium but bought a few back, profiting the spread but reacquiring the risk.
I think it’s an intriguing company with a compelling story behind it.
The capital raise was big (a qtr of the market cap) but this company doesn’t have a history of abusing its shareholders. They were already pretty well funded but now they’ll have a war chest. Supply is large.. the question is, what’s the demand like? We’ve got some momo and also headwinds.. hopefully the demand is sufficient to soak it up.
Good Luck!
ProQR Prices $90 Million Underwritten Public Offering of Ordinary Shares
Those covered calls sold at a nice premium are sure looking good in the rear view.
Covered some of my short. Pocketed the difference.
Good Luck!
* * $PRQR Video Chart 03-25-2021 * *
Link to Video - click here to watch the technical chart video
You never know. I couldn’t resist selling a few Oct calls strike 10 @330
But I’ve got a hefty position so should that occur, I won’t complain!
All the makings for $12 after hours IMO. Could open $15+ tomorrow before a major sell off.
Congrats!
Premium getting tasty on the long dated calls. Good way to lock profits and/or pad gains on the repricing climb (selling against your position).
All the best.
I'm up $34k and not a poster on here other than you my friend. Cheers.
Cantor raises target to $33 (from $22)
Citi upgraded to $44
JMP to $35
Current price - roughly $7
Good Luck!
Good idea. It’s bumping up against that 620 resistance right now. Hope it busts through!
Going to ride some free shares here when the dust settles on my swing trade. Neat company with a feel good problem to be apart of solving.
Cash runway into 2023.
presentation slides
I’m not an ophthalmologist (calling Dr. Rand Paul) but listening in to the call sounds awfully positive. Safe and well tolerated with no SAE’s. Meeting all endpoints. It was with a single injection too with the data showing a second dosing at around 6 months might give added benefit.
This next P2/3 trial (x2 parallel studies) is going to be really interesting. End of 2021. I believe he said the trials would be 18 months long with a possible interim readout at 12 months.
Edit - compelling presentation. I may stick around longer than I anticipated. Good problems.
I agree. Conference call at 830est
Hoping for a convincing break through 6.20 to a short term $7 target (Note that I change my mind as I see patterns develop). Traders perspective. I sold some of my $5 position at 6.05 to lower my risk going into these results.
Edit - This is a very intriguing company and although I’m primarily trading, I’m considering keeping a core position longer term. Especially if we have a big move north.
Good Luck!
Nice news, price target for you?
7:00a ET 3/24/2021 - Globe Newswire
ProQR Announces Positive Results from Clinical Trial of QR-421a in Usher Syndrome and Plans to Start Pivotal Trials
Mentioned: PRQR
-- QR-421a demonstrated a concordant benefit in multiple measures of vision, including best corrected visual activity (BCVA), static perimetry, and retinal imaging (OCT)
-- QR-421a observed to be well tolerated with no serious adverse events reported
-- Two pivotal Phase 2/3 trials are expected to start by the end of 2021
-- Management to host a conference call today at 8:15am EDT
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 results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations. In the trial, QR-421a demonstrated benefit on multiple measures of vision that moved in concordance, including visual acuity, visual fields, and optical coherence tomography (OCT) retinal imaging, after a single dose. QR-421a was observed to be well tolerated with no serious adverse events reported. Based on these findings, the Company plans to advance QR-421a to two parallel pivotal Phase 2/3 trials by year end 2021 - one in early-moderate patients, one in advanced patients.
"We're pleased to have met all the objectives we set for the Stellar trial, including determining suitable registration endpoints, the dose, dosing interval, and patient population for the Phase 2/3 pivotal trials," said Aniz Girach, MD, Chief Medical Officer of ProQR, "With just a single dose, QR-421a demonstrated clinical proof of concept with benefit observed in treated eyes compared to the untreated eyes in multiple concordant measures of vision. As expected, we saw benefits in both advanced and early-moderate patients in this slow progressing, debilitating eye disease, allowing us to advance this important investigational therapy for all patients with Usher syndrome and nsRP due to USH2A exon 13 mutations. Based on preliminary Regulatory guidance, we plan to submit protocols to advance QR-421a to pivotal testing. This is our second program targeting a severe inherited retinal disease that is moving into pivotal trials, which we believe further validates our RNA therapy platform and our capabilities to design and efficiently take these programs through clinical development."
"The safety profile and efficacy findings for QR-421a are very encouraging," said Robert Koenekoop, MD, MSc, PhD, FRCS(C), FARVO, a clinical-scientist from the Montreal Children's Hospital and Professor of the McGill University Faculty of Medicine and Department of Pediatric Surgery. "Usher syndrome and non-syndromic retinitis pigmentosa due to USH2A exon 13 mutations are devastating retinal diseases representing a high unmet medical need, as there are no approved therapies to treat the severe vision loss associated with these diseases. Patients' biggest hope for a therapy is to stop disease progression and prevent vision loss, and these findings suggest that QR-421a has the potential to stabilize vision. I look forward to this exciting program advancing into pivotal trial development."
Results from the Phase 1/2 trial of QR-421a
Safety Data
QR-421a was observed to be well tolerated at all doses. There were no serious adverse events reported and no inflammation was observed. One patient had worsening of pre-existing cataracts in both the treated and untreated eyes; both were deemed not treatment related by the investigator. One patient had progression of pre-existing cystoid macular edema (CME) that was managed with standard of care. Both cataracts and CME are associated with a high rate of occurrence in the natural history of this disease.
Efficacy Data
Given the key differences in baseline characteristics, patients were categorized into "advanced" and "early-moderate" populations based on baseline visual acuity.
In advanced patients, the primary measure of efficacy is BCVA. In early-moderate patients, the primary measure of efficacy is measurement of visual fields by static perimetry. QR-421a-treated patients responded on endpoints consistent with their disease stage in both advanced and early-moderate patient populations after a single injection.
All three doses studied in the Stellar trial were observed to be active as predicted by the pre-clinical data. No differences were observed based on patients being homozygous or heterozygous, or having Usher syndrome or non-syndromic retinitis pigmentosa. These findings are consistent with the preclinical data for QR-421a.
Analysis of advanced patients
Visual acuity
Best corrected visual acuity, or BCVA, is a measure of central vision, or sharpness of sight, as measured on an Early Treatment of Diabetic Retinopathy Study (ETDRS) letter chart.
Across all treated patients (n=14), a mean benefit of 6.0 letters was observed at week 48 in the treated eyes compared to the untreated (contralateral) eyes after a single injection.
Among advanced disease patients (n=6), a mean benefit of 9.3 letters was observed at week 48 in the treated eyes as compared to the untreated eyes and the benefit was maintained for >12 months. All six advanced patients had a benefit in the treatment eye, whereas none of the patients in the sham group had a benefit in the treatment eye.
Analysis of early-moderate patients
Static perimetry
Static perimetry assesses visual fields and retinal sensitivity in the peripheral retina.
Across all treated patients, the mean total retinal sensitivity improvement was up to 40dB higher in the treated eyes compared to the untreated eyes, and the benefit was maintained for >6 months after a single injection.
The mean number of retinal locations (loci) that improved by greater-than or equal to7db in retinal sensitivity demonstrated a benefit in the treated eyes compared to the untreated eyes, with up to a mean of 9 loci in the treated eyes improving by greater-than or equal to7db .
In early-moderate patients (n=8), up to a mean of 13 loci in the treated eyes improved by greater-than or equal to7db compared to 7 loci for the untreated eyes at the same timepoint.
Concordant benefits were noted on OCT-based assessment of the Ellipsoid Zone layer, which is an objective evaluation of photoreceptor viability, and other measures of central visual function, such as microperimetry. Sham treated eyes responded similarly to the untreated eyes across all endpoints.
Pivotal trials
On the basis of these findings, the Company plans to conduct two pivotal Phase 2/3 clinical trials. Based on initial Regulatory guidance, the Company plans to submit protocols to start two Phase 2/3 trials. Each trial could potentially serve as the sole registration trial depending on the findings. Pending finalization of the study designs with Regulatory authorities, the trials are expected to start before year end 2021. Both trials are expected to be conducted at global centers of excellence.
Sirius trial in advanced population
The "Sirius" trial is a Phase 2/3 study that will focus on advanced patients with baseline BCVA less-than or equal to20/40. The preliminary design for Sirius is a double-masked, randomized, sham-controlled, 24-month, multiple-dose study. The trial is expected to enroll approximately 100 adults with Usher syndrome and nsRP due to USH2A exon 13 mutations, including both homozygous and heterozygous patients. The primary endpoint in this trial will be BCVA at 18 months, with potential for an earlier interim analysis. In this three-arm study, two different doses will be studied that will be administered every 6 months, and a third arm will receive sham treatment.
Celeste trial in early-moderate population
In parallel to Sirius, the Company plans to start the "Celeste" Phase 2/3 trial in early-moderate patients. The preliminary design for Celeste is a double-masked, randomized, sham-controlled, 24-month, multiple-dose study. The trial is expected to enroll approximately 100 adults with Usher syndrome and nsRP due to USH2A exon 13 mutations. The primary endpoint in this trial will be based on static perimetry at 18 months, with potential for an earlier interim analysis. In this three-arm study, two different doses will be studied that will be administered every 6 months, and a third arm will receive sham treatment.
"There are currently no available treatments for the more than 16,000 patients with Usher syndrome 2A and nsRP due to USH2A exon 13 mutations and we are excited about the potential for QR-421a to address this significant unmet need," said Benjamin R. Yerxa, PhD, Chief Executive Officer at the Foundation Fighting Blindness. "We are pleased to see QR-421a advancing to pivotal testing and proud to support the work of ProQR as they advance their pipeline of RNA therapies to potentially help children, adults, and families who are affected by blindness caused by USH2A mutations and other rare inherited retinal diseases."
Conference call
Management will discuss the data during a webcasted conference call today at 8:15 am EDT. The dial-in details for the call are +1 631-510-7495 (US), +31 (0)20 714 3545 (NL), conference ID: 8596733.
An archive of the webcast will be available for approximately 30 days following the presentation date.
Thank you, very informative. This could be a massive company someday.
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