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Wat is de waarde van uniQure?

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Prof. Dollar 16 februari 2017 15:09

quote:
T. Montana schreef op 16 februari 2017 13:30:
[...]

Dank Prof & flosz. Domme vraag wsch. maar wat is BTD?

breakthrough therapy designation

Op Twitter is het aantal karakters beperkt, vandaar. De afkorting wordt daar ook vaker gebruikt.

T. Montana 16 februari 2017 15:27

quote:
Prof. Dollar schreef op 16 februari 2017 15:09:
[...]
breakthrough therapy designation

Op Twitter is het aantal karakters beperkt, vandaar. De afkorting wordt daar ook vaker gebruikt.

Dank

DeZwarteRidder 16 februari 2017 16:33

Data Presented at CROI Show CytoDyn’s Pro 140 as a Single Agent Provided Maximal Virologic Suppression in HIV Patients for Nearly Two Years
GlobeNewswire•February 16, 2017

VANCOUVER, Washington, Feb. 15, 2017 (GLOBE NEWSWIRE) -- CytoDyn Inc. (CYDY), a biotechnology company focused on the development of new antibody therapies for combating human immunodeficiency virus (HIV) infection, announces that data from its ongoing Phase 2b extension study with PRO 140 administered as a single agent provided maximal virologic suppression and was well tolerated by 10 HIV-infected patients for nearly two years. The study data was the subject of a poster presentation by Dr. Kush Dhody, Senior Director, Clinical Operations at Amarex Clinical Research, at the Conference on Retroviruses and Opportunistic Infections (CROI) being held in Seattle.

The abstract and poster, entitled “PRO140 Single-Agent Maintenance Therapy for HIV-1 Infection: A 2-Year Update,” are available on the company’s website at www.cytodyn.com. The study data will also be featured at CROI 2017 in a special hour-long Themed Discussion, “I Want a New Drug,” on February 16 beginning at 1:30 p.m. Pacific time (4:30 p.m. Eastern time). A delayed webcast of the Themed Discussion will be available on February 17, 2017 by 2:30 p.m. Pacific time at croiwebcasts.org, then select “Feb 16” and scroll down to “I Want a New Drug.”

The poster reviews the two-year treatment data from the ongoing Phase 2b CD01 extension study into which 16 patients infected with CCR5-tropic HIV-1 were enrolled after maintaining virologic suppression (HIV-1 RNA levels below 40 copies/mL) following 12 weeks of weekly, subcutaneous injections of PRO 140 (350 mg) as a single agent under the initial CD01 study. Of the 16 patients, 14 were male and three were non-white; the median age was 54.9 years (range of 26-68) and median CD4 T-cell count was 593 cells/mm3 (range of 365-1059). These patients were trained to self-administer PRO 140 and were allowed to continue weekly subcutaneous injections as a monotherapy for up to three years (or 160 weeks). Of those enrolled, 13 patients (81.3%) maintained complete virologic suppression for more than 40 weeks and 10 patients (62.5%) maintained complete virologic suppression for nearly two years and are still continuing on PRO 140 monotherapy regimen. One patient discontinued at week 47 with complete virologic suppression due to relocation and five patients experienced virologic rebound, defined as two consecutive viral load measurements of =400 copies/mL. The mean time to virologic rebound was 329 days (range of 106-691). An advanced single-copy HIV RNA assay levels (to quantify the viral load below the limit of detection of commercially available assays) were evaluated for the 10 ongoing patients at two-year time point. Seven (7) patients reported viral load of <1 copy/mL; and other 3 patients reported values of 4, 10, and 19 copies/mL. These single-copy HIV-1 RNA results provide further evidence of potent antiviral activity of PRO 140.

“HIV-infected patients are in need of new approaches for maintaining virologic suppression as many experience toxicity, intolerance or suboptimal adherence to the current standard of care, which is a daily oral combination antiretroviral therapy (ART),” said Dhody. “As highlighted in this poster, a subgroup of patients was able to maintain complete virologic suppression on PRO 140 over extended periods of time without the compliance, tolerance and resistance issues associated with oral combination ART. The strength of the data presented today demonstrates that PRO 140 warrants further evaluation as a long-acting, single-agent maintenance therapy in select HIV-1 patients. It is indeed an honor to present this data to attendees at CROI 2017.”

“PRO 140 is a humanized IgG4 monoclonal antibody that works by blocking HIV-1 from entering and infecting immune cells by binding to CCR5 with high affinity, which is a novel approach to maintaining virologic suppression,” said Paul J. Maddon, MD, PhD, inventor of PRO 140 and Senior Science Advisor to CytoDyn. “It is exciting that a group of patients self-administering PRO 140 as a monotherapy were able to avoid the potential toxicity of ART, while preserving their option to return to an ART regimen at a later date. PRO 140 was well tolerated by a majority of patients in this extension study and there were no reports of serious adverse events or treatment discontinuation related to the drug. As anticipated, no anti-PRO140 antibodies were detected in any patient, indicating there were no signs of drug resistance.”

Vitavita 16 februari 2017 19:58

Goed interview !

www.fiercebiotech.com/r-d/uniqure-det...

uniQure details three-pronged gene therapy trial program

by Nick Paul Taylor | Feb 16, 2017 10:15am

uniQure plots to initiate a three-pronged clinical trial program next year as key assets from its in-house pipeline and Bristol-Myers Squibb collaboration advance. uniQure aims to move its hemophilia B gene therapy, AMT-060, into a pivotal trial while working to advance a Huntington's disease asset and Bristol-Myers-partnered heart failure candidate into the clinic.

Matthew Kapusta, who took over as CEO of uniQure in September, detailed the strategy in a talk (registration required) at Leerink’s healthcare conference. The plan will follow the strategy uniQure set out in November when it laid off workers and stepped down its interest in Sanfilippo B and Parkinson’s disease to prioritize investment in the aforementioned three programs.

The closely watched hemophilia B remains the centerpiece of the company. While investors were disappointed by the Factor IX activity presented in a succession of readouts—especially when compared to data from Spark Therapeutics’ rival gene therapy—uniQure maintained AMT-060 has a compelling efficacy and safety profile.

Kapusta claimed patients, physicians and the FDA share uniQure’s focus on reductions in FIX transfusions and rates of spontaneous bleeding rather than FIX activity.

“In the interaction that we had with the FDA, it was very clear that they were looking at defined clinical benefits. And I think they were defining clinical benefit not in terms of Factor IX activity but in terms of impact on annualized bleeding rate,” he said.

uniQure will have another chance to hear the FDA’s views at its end of phase 2 meeting set to take place before the end of the quarter. The meeting will mark the start of a process intended to lead uniQure into a pivotal trial next year.

The start of phase 3 should contribute to a busy year in the clinic for uniQure. Following a year of collaboration with Bristol-Myers working on manufacturing matters, uniQure is now in the middle of animal studies that could set it up to file an IND—and possibly start a trial—next year. uniQure is running a dose expression study of the heart failure gene therapy in healthy and diseased pigs.

In parallel, uniQure is working to get its Huntington's disease asset into the clinic. The candidate, the result of a five-year project, uses the AAV5 vector to deliver a microRNA intended to silence the Huntington gene. In mice, administration of the gene therapy into the striatum led to a 50% knockdown in the cortex.

Fartknock 16 februari 2017 20:06

Interessant. Bedankt voor het delen Vita.

Prof. Dollar 16 februari 2017 20:46

Het is overigens geen interview maar samenvatting van de webcast gisteren. Efin, toch opvallend dat er geen woord wordt gerept over het Hemofilie A programma.

DeZwarteRidder 17 februari 2017 07:52

About CytoDyn
CytoDyn is a biotechnology company focused on the clinical development and potential commercialization of humanized monoclonal antibodies for the treatment and prevention of HIV infection. The Company has one of the leading monoclonal antibodies under development for HIV infection, PRO 140, which has completed Phase 2 clinical trials with demonstrated antiviral activity in man and is currently in Phase 3. PRO 140 blocks the HIV co-receptor CCR5 on T cells, which prevents viral entry. Clinical trial results thus far indicate that PRO 140 does not negatively affect the normal immune functions that are mediated by CCR5. Results from seven Phase 1 and Phase 2 human clinical trials have shown that PRO 140 can significantly reduce viral burden in people infected with HIV. A recent Phase 2b clinical trial demonstrated that PRO 140 can prevent viral escape in patients during several months of interruption from conventional drug therapy. CytoDyn intends to continue to develop PRO 140 as a therapeutic anti-viral agent in persons infected with HIV and to pursue non-HIV indications where CCR5 and its ligand CCL5 may be involved. For more information on the Company, please visit www.cytodyn.com.

About PRO 140
PRO 140 belongs to a new class of HIV/AIDS therapeutics – viral-entry inhibitors – that are intended to protect healthy cells from viral infection. PRO 140 is a humanized IgG4 monoclonal antibody directed against CCR5, a molecular portal that HIV uses to enter T-cells. PRO 140 blocks the predominant HIV (R5) subtype entry into T-cells by masking this required co-receptor, CCR5. Importantly, PRO 140 does not appear to interfere with the normal function of CCR5 in mediating immune responses. PRO 140 does not have agonist activity toward CCR5 but does have antagonist activity to CCL5, which is a central mediator in inflammatory diseases. PRO 140 has been the subject of seven clinical trials, each demonstrating efficacy by significantly reducing or controlling HIV viral load in human test subjects. PRO 140 has been designated a “fast track” product candidate by the FDA. The PRO 140 antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements as compared to daily drug therapies currently in use.

[verwijderd] 17 februari 2017 10:48

So riddert, heb jij in je nest gepist vanmorgen 7.52 om sharlie sheen te pluggen ..

Prof. Dollar 17 februari 2017 22:13

Off topic:

Arthrogen announces approval for Phase Ib study with gene therapy ART-I02 in patients with rheumatoid arthritis.
Press release (PDF): www.arthrogen.nl/images/downloads/201...

Toch wel bijzonder. Het is letterlijk de buurman van QURE (in hetzelfde gebouw) en maakt gebruikt van AAV5.

DeZwarteRidder 17 februari 2017 22:19

quote:
ch@rter schreef op 17 februari 2017 10:48:
So riddert, heb jij in je nest gepist vanmorgen 7.52 om sharlie sheen te pluggen ..

Charlie leeft waarschijnlijk langer dan QURE.....

T. Montana 18 februari 2017 00:21

quote:
Prof. Dollar schreef op 17 februari 2017 22:13:
Off topic:

Arthrogen announces approval for Phase Ib study with gene therapy ART-I02 in patients with rheumatoid arthritis.
Press release (PDF): www.arthrogen.nl/images/downloads/201...

Toch wel bijzonder. Het is letterlijk de buurman van QURE (in hetzelfde gebouw) en maakt gebruikt van AAV5.

Inderdaad bijzonder en COO Janneke Meulenberg was "Formally responsible for taking Glybera® to the clinic at AMT (now Uniqure)." Weet niet hoe je dat vertaalt "formally responsible" maar het geeft de indruk dat "iedereen kan wel zeggen dat ze Glybera hebben ontwikkeld, maar mevrouw Meulenberg heeft het werkelijk gedaan" en misschien is dat ook zo. Ongebruikelijke investeerders trouwens, die heb ik nog nooit ergens gezien. Kan me niet voorstellen dat ze hiermee niet hebben geleurd bij QURE. GT in RA, hoe ga je dat toedienen?

Prof. Dollar 18 februari 2017 00:32

quote:
T. Montana schreef op 18 februari 2017 00:21:
[...]

Inderdaad bijzonder en COO Janneke Meulenberg was "Formally responsible for taking Glybera® to the clinic at AMT (now Uniqure)." Weet niet hoe je dat vertaalt "formally responsible" maar het geeft de indruk dat "iedereen kan wel zeggen dat ze Glybera hebben ontwikkeld, maar mevrouw Meulenberg heeft het werkelijk gedaan" en misschien is dat ook zo. Ongebruikelijke investeerders trouwens, die heb ik nog nooit ergens gezien. Kan me niet voorstellen dat ze hiermee niet hebben geleurd bij QURE. GT in RA, hoe ga je dat toedienen?

Ik kan mij ook niet voorstellen dat ze niet bij QURE hebben geleurd. Alleen al omdat ze AAV5 gebruiken, waarop QURE de rechten heeft. Het zou mij niks verbazen als ze hun verstandhouding beiden bewust stil houden voor de buitenwereld.

Toediening geschiedt door direct in het gewricht afleveren van de gentherapievectoren, dan worden er daar plaatselijk eiwitten gemaakt. Cellen van het synovium, de synoviale cellen, zijn eenvoudig te bereiken via de gewrichtsholte, leven betrekkelijk lang en vormen zo een ideaal doel voor gentherapie.

[verwijderd] 19 februari 2017 16:07

quote:
DeZwarteRidder schreef op 17 februari 2017 22:19:
[...]
Charlie leeft waarschijnlijk langer dan QURE.....

Onkruid vergaat idd heel slecht (niet)

Prof. Dollar 21 februari 2017 20:49

Off topic:
Lysogene Receives Orphan Drug Designation from EMA for LYS-GM101 for Treatment of GM1 Gangliosidosis www.businesswire.com/news/home/201702...

Belegde boterham 21 februari 2017 22:11

BTD = Breakthrough Therapy Designation

Belegde boterham 21 februari 2017 23:33

quote:
Belegde boterham schreef op 21 februari 2017 22:11:
BTD = Breakthrough Therapy Designation

Was al gepost in volgende pagina, excusez

Hoe is met de steunniveau's alhier ;)

T. Montana 22 februari 2017 00:24

quote:
Belegde boterham schreef op 21 februari 2017 23:33:
[...]

Was al gepost in volgende pagina, excusez

Hoe is met de steunniveau's alhier ;)

Voldoende geestelijke steun.

[verwijderd] 22 februari 2017 13:04

quote:
ch@rter schreef op 13 februari 2017 23:26:
The other way around ...

Any way
Kijkend nr de grafiek is de vrije val ontstaan op 14$ naar de 7$ , terugtest naar de 9+$ opnieuw down naar de -7$ wederom een terugtest naar de 8+$ opnieuw down naar de 5+$ en een terugtest nu naar de 7$
Kortom de terugtesten zijn steeds rond de 1$ lager en de Atl's are worse.

Ik ben geen TA-er maar om een patroon te onkennen gaat toch wat ver.
Ben ook reuze nieuwsgierig wat de stand van zaken is over laten we zeggen 2 a 3 mndn.

Zonder enig nieuws or what so ever blijft het een slopend verhaal .. jammer genoeg.

Tijd zal t leren.

Nou wij zijn inmiddels 1,5 handelsweek verder en heb zo nu en dan eens met mn linkeroog naar de slotjes gekeken en mijn overgebleven halve uniqure porto .. trieste zaak, bijna weer een dollar afgekalfd en het blijft maar zakken. Je kunt erop wachten om weer die lowsy 5 in de koers te zien.
Duikt ie wederom onder de ATL dan pak ik mijn verkochte helft weer op en wacht op een nieuwe UPtick.

Prof. Dollar 22 februari 2017 13:32

Mogelijk dat men nu vooral intern bezig is: overdracht van vertrekkende medewerkers, verhuizing naar nieuw onderkomen Amsterdam, transfer kennis naar Lexington. Dit zal wel tot juni kunnen duren.

Helaas valt er ondertussen extern weinig tot niks te beleven. Het is akelig stil rondom San. B en Parkinson's programma's. Het begint zelfs saai te worden.

Wat wel interessant is om te onthouden dat is het koersverloop van ARIAD Pharmaceuticals afgelopen jaar (daar komt Maria vandaan): van 5 dollar naar exit van 25 dollar. Volgend jaar kan QURE ook aan de start van zo'n beweging staan.

Prof. Dollar 22 februari 2017 18:07

Off topic:

Geen goede dag voor Forbion... Argos’ PhIII for its personalized cancer vaccine implodes during an interim analysis. -74% ir.argostherapeutics.com/releasedetai...

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