Aandeel Pharming Group AEX:PHARM.NL, NL0010391025

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gurt schreef op 9 mei 2018 09:16:

Er was toch ook nog een amateur shorter bezig? hoe zou het daar mee gaan?

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Nachtzwaluw schreef op 9 mei 2018 08:58:

[...]

Wat ik wijs wordt uit de meetings zal ik net als anderen graag delen.
Nog even terug naar Pompe (voordat ik gecorrigeerd word); er zijn natuurlijk 3 middelen, myozyme en de afgeleide lumizyme (beiden als ik het goed heb vermarkt door .... Sanofi. En dan GAA, wat eigenlijk gewoon hetzelfde is.
En dan het BioMarin verhaal; best een mooie benadering en in de exacte gedachte van modificatie van het GAA:
www.nature.com/scibx/journal/v5/n48/f...
Stand van zaken? BMN 701; ik vind het niet terug op hun site.... maar misschien weet iemand anders er meer van.
..........

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Techspec schreef op 9 mei 2018 09:13:

Shortsell positie Pharming afgebouwd naar 2,84%.
MW heeft verder afgebouwd:
shortsell.nl/short/Pharming

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Bones schreef op 9 mei 2018 09:26:

Ik vindt dat we wel weer eens een freeze verdienen.

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Berdientje schreef op 9 mei 2018 09:30:

[...]

Ja natuurlijk wordt er afgebouwd,
ze pakken nu de winst en wachten op de volgende koers daling om weer in te stappen.

Want zonder nieuws in 5 dagen 6 cent stijgen?

Vorige maand ging de koers van 1,43 op vrijdag 27e naar 1,33 op maandag de 30e
Dus vooral niet naar het strand gaan.

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JorisDeRooij schreef op 9 mei 2018 09:40:

First Berlin Equity Research has published a research update on Pharming
Group NV (ISIN: NL0010391025). Analyst Simon Scholes reiterated his BUY
rating and increased the price target from EUR 1.90 to EUR 2.00.

Abstract:
Pharming's annualised revenue run-rate reached EUR132m in Q4 2017. Investors
are now asking whether the company can push revenues further ahead in 2018
in the face of increasing competition on the hereditary angioedema (HAE)
market. Another question is whether the preclinical pipeline will have any
near-term impact on valuation. Our answer to both of these questions is
yes. We have raised our price target slightly to EUR2.00 (previously: EUR1.90)
to reflect Q4/2017 numbers which were above our forecasts. We do not yet
include any of the preclinical pipeline products in our valuation but we
have examined these in detail in this study and expect this to change later
on this year. We are particularly optimistic about prospects in Pompe
disease, for which Pharming plans to submit an investigative new drug
application later this year. Our recommendation remains Buy.

bron:
ch.4-traders.com/PHARMING-GROUP-12738...

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Sursum schreef op 9 mei 2018 09:35:

Update on a Clinical Trial (COMET Study by Sanofi Genzyme)
30 September 2017

NeoGAA, a new generation of enzyme replacement therapy developed by Sanofi Genzyme will be tested in 96 patients with type 2 glycogenosis (Pompe disease). The recruitment of this trial is ongoing.

This international trial takes place in more than 20 different countries.

The aim is to compare the safety of use and efficacy of neoGAA with the only treatment currently available in Pompe disease. For this, 96 people who have not been previously treated for Pompe Disease, will be treated for 1 year, either by neoGAA or by Myozyme®. After this first year, all patients will receive NeoGAA for about 2 years. This clinical trial began in October 2016 and is expected to be completed by 2020.

A new generation recombinant enzyme

NeoGAAA and Myozyme® are recombinant forms (created by genetic engineering) of human acid alpha-glucosidase (GAA), the defective enzyme in Pompe disease. Myozyme® has been used since 2006 in the treatment of Pompe disease.

NeoGAA is a drug candidate developed from Myozyme® to increase its effectiveness. It has already been tested in humans in a phase 1/2 trial (NEO1), conducted in 24 people with Pompe disease for 6 months. Based on first results from this trial, the European Medicines Agency has attributed this molecule an orphan drug status. This designation applies to drug candidates (who have not yet proved their effectiveness) in rare diseases, in order to facilitate the different stages of their development.

To participate in the trial

The trial takes place in more than 75 different centers around the world. Individuals wishing to apply to participate in this trial must meet a number of criteria and obligations related to the trial protocol (to ensure valid and reliable conclusions). Among the main criteria, one must be suffering from Pompe disease, be over 3 years old and have never received a Myozyme® infusion.

If you have any questions or would like to participate in this trial as a patient, please contacty our treating physician.

Information on all clinical trials can be found at www.clinicaltrials.gov (search “Pompe”).

Link to this study on clinicaltrials.gov: Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET).

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up en down schreef op 9 mei 2018 09:34:

Pharming aandeel als bezit is bij mij nog altijd::::soms moet je ze hebben en vaker>>>!

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Super trio schreef op 9 mei 2018 09:02:

Wil je naar een koers van 5 euro, dan zal de Vries aandelen terug moeten kopen. Al begint ie maar met 3 % van het totaal of een poging daarna toe. Goed voor bestaande en nieuwe investeerders. Lijkt mij..

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Sursum schreef op 9 mei 2018 09:29:

[...]

www.worldpompe.org/index.php/news/591...
(June, 2016)

BioMarin greatly appreciates the many patients, families, and physicians who have participated in our Pompe program, as well as your ongoing support of the entire patient community. After much deliberation, BioMarin has decided to discontinue the clinical development of the BMN 701 (reveglucosidase alfa) Pompe program. However, BioMarin remains open to external opportunities for the development of this compound. This decision is not based on concerns for patients’ safety or efficacy.

What about compassionate use?

BioMarin’s policy for treatment under compassionate use requires that the drug be under development. Since BioMarin’s plans are to discontinue development of BMN 701, we are not planning a compassionate use program at this time.

Ik weet niet of er in de tussentijd wat verandert is (Stand juli 2016).