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[verwijderd] 3 augustus 2012 22:25

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Expected Rejection, Unexpected Consequences

By Brian Orelli, The Motley Fool
Posted 7:42PM 08/02/12
Posted under: Investing

"As anticipated and as part of the ongoing regulatory review process, we do expect to receive a complete response letter from the FDA within the next few weeks," said Baxter (NYS: BAX) CEO Bob Parkinson about his company's immunodeficiency disease treatment HyQ a couple of weeks ago.
So it shouldn't have come as much of a surprise when the Food and Drug Administration turned down the drug yesterday. Yet Baxter's partner, Halozyme Therapeutics (NAS: HALO) , lost nearly half its stock value today.
HyQ is a combination of Baxter's plasma-derived Immune Globulin and Halozyme's recombinant human hyaluronidase, which allows the drug to be delivered under the skin (subcutaneous) rather than being infused intravenously. The problem isn't with the Immune Globulin, but with Halozyme's technology. In fact, the FDA isn't just worried about its use in HyQ, but in other subcutaneous products Halozyme is helping to develop.

The FDA is concerned about having patients develop antibodies to hyaluronidase and their potential effect on reproduction, development, and fertility. Some patients in the clinical trial developed antibodies, but there weren't any adverse events associated with the formation of the antibodies, so the companies will have to use pre-clinical experiments to convince the FDA that they don't pose any undue risk.
Until Halozyme can convince the FDA there isn't an issue, the agency wants dosing stopped in trials testing HyQ and a subcutaneous formulation of Viropharma's (NAS: VPHM) Cinryze.
Using pre-clinical data to convince the FDA that there aren't safety concerns won't be easy, but it's not necessarily a death sentence for the technology. Arena Pharmaceuticals (NAS: ARNA) was able to address a long list of safety issues without running additional clinical studies.
Halozyme has other partnerships with Roche and Intrexon and drugs it's developing on its own that aren't affected, so the news isn't the end of the world; just half of the world, judging by its share price. Until Halozyme discloses more details about what it needs to do and how long it'll take, investors should probably stay away.

[verwijderd] 4 augustus 2012 00:30

2

perfect timing...

Bad News for ViroPharma - Analyst Blog

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ViroPharma Incorporated ( VPHM ) recently announced that the US Food and Drug Administration (FDA) has advised the company and its partner, Halozyme Therapeutics ( HALO ), to put the phase II study evaluating the combination subcutaneous Cinryze (C1 esterase inhibitor [human]) and recombinant human hyaluronidase enzyme (rHuPH20) on temporary clinical hold.

The Center for Biologics Evaluation and Research (CBER) division of the FDA informed the companies that the US regulatory body is assessing the potential risk of the long-term effect of anti-rHuPH20 non-neutralizing antibodies associated with the use of Halozyme's rHuPH20. The FDA detected some risks pertaining to rHuPH20 in a separate study without Cinryze.

Halozyme informed ViroPharma that it must first provide results from additional pre-clinical studies to the CBER before they can resume the on-hold combination study. The US regulatory body also mentioned that the problem does not specify Cinryze and ViroPharma can continue evaluating subcutaneous administration of Cinryze without rHuPH20.

ViroPharma has decided to inform the European regulatory authorities of the FDA's action. The company has also decided to postpone its enrollment process in Europe for the phase II combination study of Cinryze and rHuPH20, until the FDA confirms the safety of the latter.

Following the FDA's decision, ViroPharma is set to commence a phase II study, evaluating the safety and efficacy of two different doses of the subcutaneous administration of Cinryze as a stand-alone therapy. The US regulatory body cleared ViroPharma's Investigational New Drug (IND) application for the stand-alone therapy of Cinryze in 2011.

ViroPharma recently announced positive results from an open label prophylaxis study of Cinryze in patients suffering from hereditary angioedema (HAE).

We note that Cinryze is approved both in the US and Europe. In the US the drug is approved as intravenous (IV) administration for routine prophylaxis against angioedema attacks in adolescent and adult patients with HAE. In Europe it is approved for routine prevention, pre-procedure prevention and acute treatment of angioedema attacks in adolescent and adult patients with HAE.

[verwijderd] 4 augustus 2012 01:00

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RRR.

reageer wat laat.
MAar dit is zeker richting toekomst goed nieuws.
FDA kan dus gewoon de toestemming in trekken, mocht blijken dat er risico bestaat.

Ruud..

[verwijderd] 4 augustus 2012 01:02

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Zie nog steeds Viropharma als mogelijke overnamekandidaat...

[verwijderd] 4 augustus 2012 09:20

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Meevallertje uit onverwachte hoek voor Pharming en voor de niet-subcutane concurrenten!
Goed werk RRR!

[verwijderd] 4 augustus 2012 10:45

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En zo werkt het blijkbaar...
nos.nl/artikel/402435-hoe-een-pil-vij...

[verwijderd] 6 augustus 2012 22:11

2

hm...

ViroPharma Receives FDA Approval of Cinryze® (C1 Esterase Inhibitor [Human]) Industrial Scale Manufacturing

- Financial Results expected to be released on Thursday, August 9th at 7:30 AM ET -
EXTON, Pa., Aug. 6, 2012 /PRNewswire/ -- ViroPharma Incorporated (Nasdaq: VPHM) today announced the U.S. Food and Drug Administration (FDA) has approved the supplement to the Cinryze Biologics License Application (BLA) for industrial scale manufacturing changes. As previously announced, the company expects completion of labeling for previously produced vials to take approximately six weeks before entering into the trade.
The company also announced that it expects to release its financial results for the second quarter of 2012 on Thursday, August 9, 2012 before the open of the U.S. financial markets.
The company will host a conference call and live audio webcast at 9:00 a.m. Eastern Time on the same day. During the conference call, ViroPharma management will discuss the 2012 second quarter financial results and other business.
The press release and the live webcast of the conference call will be accessible via ViroPharma's corporate website at www.viropharma.com. An audio archive will be available at the same address until August 20, 2012. To participate in the conference call, please dial (800) 874-4559 (domestic) and (302) 607-2019 (international). After placing the call, please tell the operator you wish to join the ViroPharma investor conference call.
"We believe the approval of our supplement for industrial scale manufacturing now enables us to ensure Cinryze is available to any patient who chooses prophylaxis against their attacks of hereditary angioedema," stated Dan Soland, ViroPharma's chief operating officer.
Continued Soland, "With the addition of the industrial scale process to our current production, we believe we now have the means to ensure the market is fully served and build adequate safety stock levels. We also believe we have flexibility to increase our production even further through additional shifts should the need arise."

[verwijderd] 7 augustus 2012 08:33

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clinicaltrials.gov/ct2/show/NCT011885...

Last Updated on August 3, 2012

geen nieuws aan het front...

[verwijderd] 10 augustus 2012 18:56

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FDA approves Marqibo to treat rare type of leukaemia

The US Food and Drug Administration (FDA) has approved Talon Therapeutics’ Marqibo (vincristine sulfate liposome injection), a treatment for adults with a rare type of leukaemia known as Philadelphia chromosome negative (Ph-) acute lymphoblastic leukemia (ALL).
ALL is a rapidly progressing form of blood and bone marrow cancer that is more commonly diagnosed in children than adults.
The FDA has approved Marqibo for patients whose leukaemia has relapsed two or more times, or whose leukaemia has progressed following two or more regimens of anti-leukaemia therapy.
The treatment contains vincristine, a commonly used anti-cancer drug, encased within a liposome, a drug delivery vehicle composed of material similar to that of cell membranes. It is an injection administered once a week by a healthcare professional.
Steven Deitcher, president and ceo of Talon Therapeutics, said: ‘We are delighted that Marqibo will be available to a patient population with an under-served haematologic malignancy.
‘This represents a transformational event for Talon and fulfilment of our most important corporate goal, to date.’

[verwijderd] 11 augustus 2012 18:30

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The long-running US national budget battle appears to be heading toward some sort of resolution following the November presidential election, with the danger that without an overarching deal automatic across the board cuts will be imposed in early 2013, with most agencies forced to reduce their spending by around 8%.

In the pharmaceutical development space, a key agency affected would be the National Institutes of Health (NIH), which funds billions of dollars worth of research every year. Given its role in basic and clinical study – it is involved in at least 211 active trials – the result of cuts could be far-reaching, limiting the resources available for biotechs and academic researchers looking to test new compounds

[verwijderd] 12 augustus 2012 10:00

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Maakt Pharming nog wel kans met hun rhFactor VIII?

www.news-medical.net/news/20120517/Ch...

www.advate.nl/

[verwijderd] 12 augustus 2012 10:08

0

Eerder dacht ik dat Pharming voor Bayer mogelijk interessant was, maar zij zijn er blijkbaar zelf al mee bezig...

Bayer HealthCare to Present Data on Secondary Prophylaxis with Kogenate® FS -- antihemophilic factor (recombinant) on Bleeding Frequency in Adults and Adolescents with Severe Hemophilia A

The World Federation of Hemophilia 2012 World Congress

WAYNE, N.J., July 12, 2012 /PRNewswire/ -- Bayer HealthCare announced results from SPINART, a study designed to evaluate the effect of secondary prophylaxis with Kogenate® FS – antihemophilic factor (recombinant) on bleeding frequency and joint damage compared to on-demand treatment in adults and adolescents with severe hemophilia A. These data were presented as a late-breaker at the 50th annual World Federation of Hemophilia (WFH) World Congress, July 8-12, in Paris, France.

In the SPINART study, 84 subjects ages 12 to 50 were randomized to either prophylaxis (25 IU/kg three times per week) or on-demand treatment, with a total planned follow up period of 3 years. Results presented focus on the primary endpoint of bleeding frequency after a median follow-up period of 1.7 years.

Primary prophylaxis is defined as treatment started before or immediately after the first joint bleed, and before the age of two years, while secondary prophylaxis is defined as treatment started after multiple bleeding episodes have occurred[i].

Bayer submitted an application for an adult prophylaxis indication for Kogenate FS to the U.S. Food and Drug Administration on June 21, 2012.

"We are proud of our commitment to studying different treatment modalities, and how they might affect patient outcomes," said Pamela Cyrus, MD, Vice President and Head, U.S. Medical Affairs, Bayer HealthCare Pharmaceuticals. "We look forward to a robust scientific discussion at WFH, and to continuing our scientific investigation into adult prophylaxis as a potential treatment modality for hemophilia A."

INDICATIONS & USAGE

Kogenate® FS, antihemophilic factor (recombinant), is a recombinant factor VIII treatment indicated for the control and prevention of bleeding episodes and peri-operative management in adults and children (0-16 years) with hemophilia A. Kogenate® FS is also indicated for routine prophylaxis to reduce the frequency of bleeding episodes and the risk of joint damage in children with hemophilia A with no preexisting joint damage.

Casper4711 12 augustus 2012 20:20

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Maak dure pillen zelf.Dat was ff reclame voor Pharming in het journaal van 20:00 uur.Medicijnen in eigen beheer ziekenhuizen. daarin vertelde de nieuwslezer dat Pharming jaren terug wel een betaalbaar middel voor de ziekte van pompe had,maar daarin niet verder werkte.Maar verder geen info of verklaring daarover.

Tiekoes1 12 augustus 2012 20:41

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je kunt nieuws terug vinden in archief over de ziekte van pompe Pharming.
11dec 2001.

[verwijderd] 13 augustus 2012 13:57

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ma 13 aug 2012, 12:54 |lees voor
Doorbraak in onderzoek chronische leukemie LEIDEN - Chronische lymfatische leukemie (CLL), de meest voorkomende vorm van leukemie bij volwassenen, ontstaat niet doordat cellen in het lichaam worden aangevallen door een virus of een bacterie, maar doordat de cellen zichzelf prikkelen. Dat hebben onderzoekers van het Leids Universitair Medisch Centrum (LUMC) en het Duitse Max Planck Instituut ontdekt. Ze schrijven erover in het wetenschappelijke tijdschrift Nature.

Het Leids Universitair Medisch Centrum (LUMC) in Leiden Foto: Fotopersbureau Dijkstra
„Het was een beetje de zoektocht naar de heilige graal in het leukemie-onderzoek. Talloze onderzoeksgroepen waren er naar op zoek. Wat wij ontdekt hebben, is dat de heilige graal niet bestaat”, legde de Leidse hematoloog professor Hendrik Veelken maandag uit.

„We hoopten dat we de externe oorzaak van de verwekker van deze vorm van leukemie zouden kunnen vinden. Dan zouden we die kunnen bestrijden. Maar er blijkt dus geen externe ziekteverwekker te zijn waartegen we kunnen vechten”, aldus Veelken.

De ontdekking schept wel nieuwe mogelijkheden voor de ontwikkeling van nieuwe middelen. „We weten nu dat we de cellen waar de leukemie ontstaat, moeten aanpakken.” Professor Veelken verwacht dat het nog wel even duurt voordat leukemiepatiënten baat hebben bij de nieuwe ontdekking. De onderzoekers gaan nu verder met het onderzoek in de hoop een geschikt medicijn te vinden.

[verwijderd] 15 augustus 2012 19:43

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Voor de liefhebber-verzamelaar:
reports.pr-inside.com/new-market-rese...

[verwijderd] 16 augustus 2012 14:15

0

www.faqs.org/sec-filings/120807/SANTA...

sniper22 16 augustus 2012 17:45

0

goed opgemerkt door RRR!..AB

Amendment to

License Agreement

Between

Pharming Group N.V., Pharming Intellectual Property B.V. and Santarus, Inc.

This Amendment (the “Amendment”) to the License Agreement dated September 10, 2010 (the “License”), among Pharming Group N.V. on behalf of itself and each of its Affiliates, including Pharming Intellectual Property B.V. and Pharming Technologies B.V. (“Pharming”) and Santarus, Inc. (“Santarus”) is made as of June 18, 2012 (the “Effective Date”).

RECITALS

WHEREAS, Pharming and Santarus desire to amend certain terms of the License as set forth herein.

AGREEMENT

NOW, THEREFORE, in consideration of the covenants set forth herein, and for other good and valuable consideration, the receipt and sufficiency of which is hereby acknowledged, the parties agree as follows:

1. Amendment of Section 1.43. Section 1.43 of the License is hereby amended and restated in its entirety as follows:

“1.43 “Product Trademarks” means Rhucin® (U.S. Registration No. 3170766, Canada Registration No. 679674 and Mexico Registration No. 915967) and Ruconest® (U.S. Registration No. 4095324, Canada Registration No. 820728 and Mexico Registration No. 1222478) and all related domain names and trademark related rights in the Territory, as more specifically described on Exhibit 1.43 attached hereto. For clarity, the Product Trademarks are subject to a right of usufruct as set forth in the Deed.”

2. Amendment of Exhibit 1.43. Exhibit 1.43 of the License is hereby amended to add the trademark information set forth on Exhibit 1.43 hereto.

3. Miscellaneous.

3.1 Except for the amendments set forth herein, all other terms and conditions of the License shall remain in full force and effect.

3.2 Capitalized terms used but not otherwise defined herein shall have the meanings assigned to them in the License.

3.3 This Amendment is deemed to have been entered into in the State of New York, United States of America, as applied to contracts entered into and performed entirely in

1

--------------------------------------------------------------------------------

New York by New York residents and its interpretation, construction, and the remedies for its enforcement or breach are to be applied pursuant to and in accordance with the laws of the State of New York, without giving effect to the principles of conflicts of law thereof.

IN WITNESS WHEREOF, the Parties have executed this Amendment as of the Effective Date.

PHARMING GROUP N.V.

By: /s/ Sijmen de Vries

Name: Sijmen de Vries
Title: Chief Executive Officer

By: /s/ Bruno M.L. Gianetti

Name: Bruno M.L. Gianetti
Title: Chief Operations Officer

PHARMING INTELLECTUAL PROPERTY B.V.

By: /s/ Sijmen de Vries

Name: Sijmen de Vries
Title: Chief Executive Officer

By: /s/ Bruno M.L. Gianetti

Name: Bruno M.L. Gianetti
Title: Chief Operations Officer

SANTARUS, INC.

By: /s/ Gerald T. Proehl

Name: Gerald T. Proehl
Title: President and Chief Executive Officer

2

--------------------------------------------------------------------------------

Exhibit 1.43

Product Trademarks

Trademark
Country
Classes
Reg.No.
Reg.date
Ren.date
Applicant
Status
Case No.

RUCONEST
CA 5 820728 March 27, 2012 March 27, 2027 Pharming Intellectual Property B.V. Registered
RUCONEST
MX 5 1222478 June 15, 2011 February 10, 2021 Pharming Intellectual Property B.V. Registered
RUCONEST
US 5 4095324 February 7, 2012 February 7, 2022 Pharming Intellectual Property B.V. Registered

3

User Contributions:

Read more: www.faqs.org/sec-filings/120807/SANTA...

voda 16 augustus 2012 22:09

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Anticonceptiepil voor mannen lijkt mogelijk

AMSTERDAM - Nieuw molecuul zou weleens kunnen leiden tot eerste hormoonvrije anticonceptiepil voor mannen.

Onderzoekers van onder andere Harvard, ontdekten een molecuul dat leidt tot tijdelijke onvruchtbaarheid bij muizen, zonder dat de dieren hun sexdrive verliezen.

Dit schrijven de onderzoekers deze week in Cell. Het middel vermindert de aanmaak van spermacellen.

De cellen die nog wel worden aangemaakt zijn slechte zwemmers, met volledige onvruchtbaarheid van de mannetjesmuis als resultaat.

Sexdrive

De stof met de naam JQ1, heeft geen invloed op het gedrag van de dieren. Ook hun testosteronspiegel blijft onaangedaan, wat goed is voor de sexdrive. De muizen hervinden hun vruchtbaarheid wanneer ze de stof niet meer krijgen toegediend, zonder schadelijke gevolgen voor toekomstig nageslacht.

Het succes van JQ1 zit hem erin dat het goed de weg weet te vinden naar de plek in de testes waar spermacellen worden aangemaakt. Dit is altijd een hardnekkig obstakel geweest voor de ontwikkeling van anticonceptiemiddelen voor mannen.

Condoom

Sinds de ontwikkeling van het condoom is men er niet meer in geslaagd om voor mannen een voorbehoedsmiddel op de markt te brengen met een tijdelijk effect.

Op moleculair niveau heeft de stof nog een aantal ongewenste bijwerkingen. JQ1 wordt dan ook nog niet gezien als de ideale werkzame stof voor een uiteindelijke pil voor mannen. Wel laat het onderzoek zien dat het mogelijk is.

Door: NU.nl/Kevin Janssen

[verwijderd] 17 augustus 2012 09:27

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en ze gaan maar door,

LONDON--AstraZeneca PLC (AZN.LN) said Friday it purchased for cancellation 200,000 of its own ordinary shares at a price of 3016 pence per share for cancellation.
MAIN FACTS:
-Shares closed Thursday at 3025 pence valuing the company at GBP37.8 billion.
-Write to Ed Ballard at ed.ballard@dowjones.com
Subscribe to WSJ: online.wsj.com?mod=djnwires
(END) Dow Jones Newswires
August 17, 2012 03:14 ET (07:14 GMT)
© 2012 Dow Jones & Company, Inc.

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