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Prof. Dollar
0
Dat valt uit het jaarverslag op te maken:

We operate a 53,343 square feet GMP qualified manufacturing facility that we lease in Lexington, Massachusetts. In November 2018, we extended and expanded the facility by leasing an additional 30,655 square feet of the same building. The expanded and extended lease for the facility terminates in June 2029, and subject to the provisions of the lease, may be renewed for two subsequent five-year terms.
Prof. Dollar
1
Resultaten uit de Parkinson-studie (GDNF).

Phase 1 Trial of Convection-Enhanced Delivery of Adeno-Associated Virus Encoding Glial Cell Line-Derived Neurotrophic Factor in Patients with Advanced Parkinson’s Disease

Authors: Davis Palmer Argersinger, B.S.; Codrin Lungu, MD; Dima Hammoud, MD; Peter Herscovitch, MD; Debra Ehrlich, MD; Gretchen Scott; Krystof Bankiewicz, MD, PhD; Kareem Zaghloul, MD, PhD; Mark Hallett, MD; Russell Lonser, MD; John Heiss, MD (Washington, DC)

Introduction:
Parkinson’s disease is progressive and presently incurable. GDNF is a neurotrophic factor that prevented the death of dopaminergic neurons in culture and animal models of Parkinson’s disease (PD).

Methods:
In this Phase 1 clinical trial, 13 adult patients with advanced PD underwent convection-enhanced delivery (CED) of an adeno-associated virus, serotype 2 vector containing glial cell line-derived neurotrophic factor (AAV2-GDNF) to investigate the safety, tolerability, and potential clinical effects of CED of AAV2-GDNF and gadoteridol, a surrogate magnetic resonance imaging (MRI) tracer, into the bilateral putamina (450 µl per hemisphere). Three escalating dose levels were evaluated: 1) 9 x 1010vg (6 patients); 2) 3 x 1011vg (6 patients); and 3) 9 x 1011vg (1 patient). Intraoperative MRI was performed during infusions. Pre-operatively, and at 6-12 month intervals post-operatively, Unified Parkinson’s Disease Rating Scale (UPDRS) Part 3 assessed motor function and positron emission tomography (PET) scanning with [18F]DOPA assessed F-DOPA uptake, a sign of presynaptic dopaminergic integrity.

Results:
The patients tolerated AAV2-GDNF infusion without short- or long-term clinical or radiographic toxicity. MRI tracked AAV2-GDNF infusion within the bilateral putamina. Average coverage of the putamina was 22%. UPDRS Part 3 assessment scores remained stable throughout the study. Increased [18F]DOPA uptake in the infused areas was seen bilaterally in 10/13 patients at 6 months (range: 5-274%, median: 36%), and in 12/13 patients at 18 months after infusion (range: 8-130%, median: 54%).

Conclusion:
Patients with advanced PD tolerated bilateral CED of AAV2-GDNF without toxicity. Gadoteridol in the infusion solution tracked putaminal AAV2-GDNF distribution. PET findings of increased putaminal [18F]DOPA uptake suggest a neurotrophic effect on dopaminergic neurons. Based on a satisfactory safety profile, a follow-up clinical trial is planned to increase putaminal coverage and possibly reverse PD progression and signs.

www.aans.org/Annual-Scientific-Meetin...
Beurs2019
0
quote:

Prof. Dollar schreef op 2 apr 2019 om 17:42:


Dat valt uit het jaarverslag op te maken:

We operate a 53,343 square feet GMP qualified manufacturing facility that we lease in Lexington, Massachusetts. In November 2018, we extended and expanded the facility by leasing an additional 30,655 square feet of the same building. The expanded and extended lease for the facility terminates in June 2029, and subject to the provisions of the lease, may be renewed for two subsequent five-year terms.

Dank Prof. Uitbreiding facility zal niet voor de kat zijn viool zijn gedaan :-) Hoopvol.
Izette
0

Want to participate in a research study? Help shape the future of investing tools and earn a $60 gift card!

Small-caps and large-caps are wildly popular among investors; however, mid-cap stocks, such as uniQure N.V. (NASDAQ:QURE) with a market-capitalization of US$2.2b, rarely draw their attention. Despite this, the two other categories have lagged behind the risk-adjusted returns of commonly ignored mid-cap stocks. QURE’s financial liquidity and debt position will be analysed in this article, to get an idea of whether the company can fund opportunities for strategic growth and maintain strength through economic downturns. Note that this information is centred entirely on financial health and is a top-level understanding, so I encourage you to look further into QURE here.

See our latest analysis for uniQure

Does QURE Produce Much Cash Relative To Its Debt?

Over the past year, QURE has ramped up its debt from US$21m to US$35m , which accounts for long term debt. With this increase in debt, the current cash and short-term investment levels stands at US$235m , ready to be used for running the business. Its negative operating cash flow means calculating cash-to-debt wouldn't be useful. For this article’s sake, I won’t be looking at this today, but you can take a look at some of QURE’s operating efficiency ratios such as ROA here.

Can QURE meet its short-term obligations with the cash in hand?

At the current liabilities level of US$20m, it seems that the business has been able to meet these commitments with a current assets level of US$237m, leading to a 11.85x current account ratio. The current ratio is calculated by dividing current assets by current liabilities. However, a ratio greater than 3x may be considered high by some.


NasdaqGS:QURE Historical Debt, April 4th 2019



NasdaqGS:QURE Historical Debt, April 4th 2019
More
Can QURE service its debt comfortably?

QURE’s level of debt is appropriate relative to its total equity, at 20%. This range is considered safe as QURE is not taking on too much debt obligation, which can be restrictive and risky for equity-holders. Risk around debt is very low for QURE, and the company also has the ability and headroom to increase debt if needed going forward.

Next Steps:

QURE’s low debt is also met with low coverage. This indicates room for improvement as its cash flow covers less than a quarter of its borrowings, which means its operating efficiency could be better. However, the company will be able to pay all of its upcoming liabilities from its current short-term assets. I admit this is a fairly basic analysis for QURE's financial health. Other important fundamentals need to be considered alongside. I suggest you continue to research uniQure to get a better picture of the stock by looking at:
1.Future Outlook: What are well-informed industry analysts predicting for QURE’s future growth? Take a look at our free research report of analyst consensus for QURE’s outlook.
2.Valuation: What is QURE worth today? Is the stock undervalued, even when its growth outlook is factored into its intrinsic value? The intrinsic value infographic in our free research report helps visualize whether QURE is currently mispriced by the market.
3.Other High-Performing Stocks: Are there other stocks that provide better prospects with proven track records? Explore our free list of these great stocks here.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned. Thank you for reading.
Prof. Dollar
0
uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease tools.eurolandir.com/tools/Pressrelea...
T. Montana
0
quote:

Prof. Dollar schreef op 8 apr 2019 om 13:20:


uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease tools.eurolandir.com/tools/Pressrelea...


Heel goed. Ik geloof niet dat deze op je "wat kunnen we dit jaar verwachten lijstje" stond?
Prof. Dollar
0
Klopt, deze stond niet op het lijstje maar was natuurlijk wel te verwachten.

Ik ben benieuwd hoe QURE de voordelen van een eventuele versnelde goedkeuring gaat benutten. Ik had de indruk dat zij voor een fase I/II studie tamelijk veel patiënten willen toelaten. Met oog op de variabele tijd (hoe ontwikkelt de ziekte zich), hebben ze daar later mogelijk baat bij; 1) als de vervolgstudie veilig blijkt te zijn hebben ze meteen veel langer termijn data beschikbaar, 2) als risico-reductie van SCA Type 3 programma.
Izette
0
colt
1
Ik zie (lees) cijfers voorbij gaan E90,- ... E180,- ...en mogelijk binnenkort zelfs boven E380,-. Ik blijf rustig zitten, t wordt spannend hier
Prof. Dollar
0
uniQure Announces Presentations at the Upcoming Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) www.iex.nl/Forum/Topic/1286603/68/Wat...
colt
0
"We hebben het afgelopen jaar aanzienlijke vooruitgang geboekt bij het verbeteren van onze gentherapie-expertise door uitbreiding van onze onderzoekspijplijn", aldus Sander van Deventer, MD, Ph.D., chief scientific officer bij uniQure. "We zijn erg blij met deze een sterke vertoning bij ASGCT en om nieuwe preklinische gegevens te hebben over de ziekte van Huntington, hemofilie A en de ziekte van Fabry. We zien uit naar het hebben van deze gegevens op de conferentie."
"We have made significant progress over the past year in advancing our gene therapy expertise through expansion of our research pipeline,” stated Sander van Deventer, M.D., Ph.D., chief scientific officer at uniQure. “We are very pleased to have such a strong showing at ASGCT, and to have new preclinical data featured in Huntington’s disease, hemophilia A and Fabry disease. We look forward to having these data prominently featured at the conference.”
Prof. Dollar
0
quote:

Prof. Dollar schreef op 15 apr 2019 om 18:30:


uniQure Announces Presentations at the Upcoming Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT)

En nu met de juiste verwijzing: tools.eurolandir.com/tools/Pressrelea...
Prof. Dollar
0
Een halfjaar geleden heeft QURE haar hemofilie A benadering gepresenteerd. Daarin zetten ze hun programma ook af tegen de huidige alternatieven, waarbij Sander van Deventer opmerkte dat er tijdens het onderzoek naar Emicizumab patiënten zijn overleden. Zojuist vernam ik: "3 more deaths on #emicizumab (#Hemlibra) have been reported today, bringing the total to 10. No details available as they occurred in post marketing. 2500 patients currently on emicizumab. We need to know if this number of deaths is more than expected." Bron: twitter.com/ProfMakris/status/1117869...
Prof. Dollar
1
Update Parkinson-studie (GDNF).

Parkinson’s gene therapy development to restart in summer 2019 - www.pharmaceutical-technology.com/com...

Voorlopig dus geen betrokkenheid van QURE. In het verhaal staat overigens dat uniQure voordien het materiaal leverde voorde fase I, maar dit is volgens mij onjuist. Dat deed een andere partij. Het betrof vooral een licentie-afspraak met betrekking tot GDNF en een mogelijk vervolg (dat overigens niet valt uit te sluiten).
Prof. Dollar
0
uniQure Announces First Quarter 2019 Results and Highlights Recent Company Progress

~ Presented Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B Demonstrating Increases in FIX Activity Sustained at up to 51% of Normal at 12 Weeks

~ Achieved IND Clearance and Fast Track Designation for AMT-130 in Huntington’s disease

~ Announced 6 Presentations at Upcoming ASGCT Meeting, Including Preclinical Data on Research Pipeline

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, April 29, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the first quarter of 2019 and highlighted recent progress across its business.

"During the first quarter of 2019, we continued to make excellent progress across our portfolio of gene therapy product candidates,” stated Matt Kapusta, chief executive of uniQure. “We remain highly encouraged by the continued follow-up from our Phase IIb study of AMT-061, which shows the potential to normalize FIX activity in patients with hemophilia B. We are now focused on advancing our ongoing Phase III HOPE-B pivotal study of AMT-061 and have made significant progress activating sites and enrolling patients, with a goal of completing patient enrollment by the end of this year. We are also pleased to announce that AMT-061 received Orphan Drug Designation by the U.S. Food and Drug Administration, which, combined with our FIX-Padua intellectual property position and AAV5’s potentially favorable immunogenicity profile, may provide a meaningful first-mover advantage.

“With the clearance of our Investigational New Drug application for AMT-130 earlier this year, we are making headway in the preparations for our Phase I/II study of AMT-130 in Huntington’s disease. AMT-130, which recently received Fast Track Designation, is the world’s first one-time administered therapy for Huntington’s disease to enter clinical testing, and we continue to expect patient dosing in this landmark study to begin in the second half of the year.”

Recent Company Progress

— Advancing late-stage development of AMT-061 for the treatment of hemophilia B

Enrollment of patients in the global HOPE-B pivotal trial in hemophilia B is advancing and the Company currently is on track to complete enrollment by the end of 2019. In February, the Company presented updated clinical data on AMT-061 demonstrating sustained increases in Factor IX activity (FIX) up to 51% of normal and mean FIX activity for the three patients of 38% of normal at 12 weeks. None of the patients received Factor infusions, reported bleeding events or required immunosuppression over a combined 42 weeks of observation.

On April 17, 2019 the U.S. Food and Drug Administration (FDA) granted AMT-061 Orphan Drug Designation (ODD). ODD in the U.S. provides special status for investigational drugs being developed for rare diseases considered to affect only up to 200,000 people in the U.S. The ODD program offers product market exclusivity for up to seven years in the U.S. following regulatory approval, along with tax and financial incentives for companies developing medicines for such orphan indications.

— Advancing AMT-130 into clinical development for the treatment of Huntington’s disease

In January 2019, the FDA declared effective the Company’s Investigational New Drug application (IND) for AMT-130. The Company expects to begin dosing patients in the second half of this year in its dose-escalating, randomized and controlled Phase I/II clinical study to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington’s disease, and is planning to announce initial safety data on the surgical procedure before the end of this year.

Earlier this month, the Company announced that the FDA has granted Fast Track designation for AMT-130. The Fast Track program is designed to facilitate the development of and expedite the review of therapies to treat serious conditions and fill an unmet medical need. A therapy granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA) or New Drug Application (NDA).

— Advancing robust pipeline of novel gene therapy research programs

The Company announced the acceptance of six data presentations, including multiple oral presentations featuring preclinical data for its gene therapy candidates in hemophilia A, Fabry disease and Huntington’s disease at the upcoming Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) on April 29- May 2, 2019.

Upcoming Events

Present preclinical data on the Company’s pipeline and technology platform at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting, April 29- May 2, 2019.

Present preclinical data on AMT-150 for the treatment of Spinocerebellar Ataxia Type 3 (SCA3) at the 2019 American Academy of Neurology (AAN) Annual Meeting May 4-10, 2019.

Present 26 weeks of follow-up data on the three patients in the Phase IIb dose-confirmation study of AMT-061 in hemophilia B patients at the Hemostasis & Thrombosis Research Society (HTRS) 2019 Symposium on May 10, 2019.

Present preclinical data on AMT-130 for the treatment of Huntington’s disease at the 2019 Associazione Italiana Corea Di Huntington Roma Onlus Annual Meeting on May 18, 2019.

Financial Highlights

Cash Position: As of March 31, 2019, the Company held cash and cash equivalents of $208.8 million, compared to $234.9 million as of December 31, 2018. The Company currently expects cash and cash equivalents will be sufficient to fund operations into 2021.

Revenues: Revenue for the three months ended March 31, 2019 was $1.1 million, compared to $3.5 million during the same period 2018. The decrease reflects the termination of activities associated with S100A1 in our collaboration with Bristol-Meyers Squibb in October 2018.

R&D Expenses: Research and development expenses were $20.5 million for the three months ended March 31, 2019, compared to $17.1 million during the same period 2018. The change was primarily related to increased activities associated with our ongoing Phase III pivotal study of AMT-061 and planned Phase I/II study of AMT-130, increased share-based compensation and the hiring of additional clinical and operations staff at our Lexington site.

SG&A Expenses: Selling, general and administrative expenses were $8.1 million for three months ended March 31, 2019, compared to $6.3 million during the same period 2018. The change was primarily related to increases in personnel and consulting expenses, professional fees and share-based compensation expenses.

Net Loss: The net loss for the three months ended March 31, 2019 was $27.8 million, or $0.74 per share, compared to $18.8 million, or $0.59 per share during the same period 2018.
Steve
0
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