Biomarin
eröffnet am 03.10.18 22:53:38 von
neuester Beitrag 06.06.23 19:57:28 von
neuester Beitrag 06.06.23 19:57:28 von
Beiträge: 36
ID: 1.289.814
ID: 1.289.814
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ISIN: US09061G1013 · WKN: 924801 · Symbol: BMRN
82,13
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Letzter Kurs 02:00:00 Nasdaq
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11.04.24 · wO Chartvergleich |
13.02.24 · wallstreetONLINE Redaktion |
Werte aus der Branche Biotechnologie
| Wertpapier | Kurs | Perf. % |
|---|---|---|
| 25,28 | +72,56 | |
| 4,7450 | +35,57 | |
| 5,0200 | +26,13 | |
| 8,9900 | +22,31 | |
| 0,6650 | +19,82 |
| Wertpapier | Kurs | Perf. % |
|---|---|---|
| 0,6050 | -18,24 | |
| 6,5500 | -19,14 | |
| 3,9900 | -21,30 | |
| 2,3100 | -25,00 | |
| 7,2800 | -59,58 |
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Antwort auf Beitrag Nr.: 73.959.110 von makratea am 05.06.23 21:51:39
PUFDA verschoben, über die Gründe können wir nur spekulieren. Roctavian wird teilweise mit 2 Milliarden Peak Sales veranschlagt. Du findest viel bei Seekingalpha, da gibt es gute Artikel zu Biomarin. Unter 90 allemal gut für einen Starter.
Zitat von makratea: Leider ist die Nachrichtenlage hier im Forum recht dünn. An der Nasdaq findet eigentlich ein reger Handel statt, kursbeeinflussende News bekomme ich aber gar nicht mit. Könntest Du noch ein paar Erläuterungen zu Roctavian geben oder einen Link einstellen, wo Infos hierzu zu finden sind?
PUFDA verschoben, über die Gründe können wir nur spekulieren. Roctavian wird teilweise mit 2 Milliarden Peak Sales veranschlagt. Du findest viel bei Seekingalpha, da gibt es gute Artikel zu Biomarin. Unter 90 allemal gut für einen Starter.
Leider ist die Nachrichtenlage hier im Forum recht dünn. An der Nasdaq findet eigentlich ein reger Handel statt, kursbeeinflussende News bekomme ich aber gar nicht mit. Könntest Du noch ein paar Erläuterungen zu Roctavian geben oder einen Link einstellen, wo Infos hierzu zu finden sind?
Noch eine Aktie, für die sich bei WO niemand interessiert. Ich habe gestern nachgekauft. Ich denke nicht, dass Roctavian einen CRL erhalten wird. Peak Sales über 2 Milliarden, 2 Milliarden beträgt auch der aktuelle Umsatz der lead drug. Die Baker Bros sind hier noch ohne Grund mit 7 Millionen Aktien dabei. Ich werde, falls doch ein CRL kommt, massiv nachkaufen, wenn wir abschmieren.
Schade, auch hier nix los. Für mich eine der im positivsten Sinne "langweiligsten" Bios da draussen. Nach der FDA-Verzögerung für Hämophilie A bisschen runtergeprügelt, aber auch schon wieder im Kommen. Bewertung noch okay für eine Firma, die dir pro Jahr gerantiert 2 Milliarden Umsatz reinschaufelt. Nach einem möglichen Absturz durch CRL würde ich hier massiv nachkaufen.
BioMarin Pharmaceutical CEO on the company’s drug pricing strategy
https://www.cnbc.com/video/2022/10/19/biomarin-pharmaceutica…
https://www.cnbc.com/video/2022/10/19/biomarin-pharmaceutica…
Trading Spotlight
Nach einer mühseligen Kursentwicklung im Verlauf der letzten Monate gibt es heute den nächsten Dämpfer.
BioMarin Also Pausing Further Enrollment of Additional Participants Outside the U.S. in Phearless Phase 1/2 Study
BioMarin is Working with FDA and Other Health Authorities and Will Communicate Next Steps When Available
Sep 5, 2021
SAN RAFAEL, Calif., Sept. 6, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S Food and Drug Administration (FDA) placed a clinical hold on the BMN 307 Phearless Phase 1/2 study. The Phearless study is evaluating BMN 307, an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy, in adults with phenylketonuria (PKU). The FDA's clinical hold was based on interim safety findings from a pre-clinical, non-GLP pharmacology study.
The Company carried out this pre-clinical study to understand the durability of BMN 307 activity in mice bearing two germline mutations, which may predispose the mice to the development of malignancy. One mutation eliminated the PAH gene that's missing in PKU and the second rendered the animals immunodeficient. Of 63 animals treated, six of seven animals administered BMN 307 at the highest dose group (2e14 Vg/kg) had tumors on liver necropsy 52 weeks after dosing with evidence for integration of portions of AAV vector into the genome. No lesions were observed in any mice at 24 weeks. Five of these animals had adenomas and one had a hepatocellular carcinoma (HCC). The translatability of these findings to humans is uncertain and under further investigation.
To date, the Company has only dosed humans in the Phearless Phase 1/2 clinical study with lower doses of either 2e13 vg/kg or 6e13 vg/kg. Due in part to the risk previously identified by historical rodent studies, the liver health of Phearless study participants is regularly monitored. The Company will work with the Data Review Board and Principal Investigators to further evaluate the study participants who have been dosed and will continue to monitor them over the long-term. The clinical significance of these pre-clinical rodent findings has not been established and cancers due to AAV integration have not been observed in larger animals or humans. BioMarin is pausing further enrollment into this global Phase 1/2 study until the investigation of these findings is completed. The company is working with the FDA and other health authorities and will communicate next steps for the program when available.
"More than 3,000 patients have been treated with gene therapy, and there are no reports of cancers emerging as a consequence. Acknowledging the complexity of the issue as highlighted in this week's FDA discussion, integrational mutagenesis and resultant cancer formation has been observed in mice using other AAV vectors," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "Therefore, we plan to investigate these findings. For patients who have already received lower doses of these vectors, we will continue to carefully evaluate and monitor their health. We are committed to understand and mitigate any risk of cancer causation."
About Phenylketonuria
PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.
To learn more about PKU and PAH deficiency, please visit www.PKU.com. Information on this website is not incorporated by reference into this press release.
About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on such website is not incorporated by reference into this press release.
Forward-Looking Statement
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the Company pausing enrollment globally in the Phearless Phase 1/2 study, the impact of the pre-clinical findings on the 307 clinical program, the predictability of the pre-clinical findings on human patients, the development of BioMarin's BMN 307 program generally, including the impact on the timing and process for regulatory interactions and decisions. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, the outcome of ongoing review of clinical and pre-clinical data; the outcome of additional experiments related to the preclinical findings; the results and timing of current and future clinical trials related to BMN 307; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2021 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.
Contacts:
Investors
Media
Traci McCarty
Debra Charlesworth
BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc.
(415) 455-7558
(415) 455-7451
SOURCE BioMarin Pharmaceutical Inc.
BioMarin Also Pausing Further Enrollment of Additional Participants Outside the U.S. in Phearless Phase 1/2 Study
BioMarin is Working with FDA and Other Health Authorities and Will Communicate Next Steps When Available
Sep 5, 2021
SAN RAFAEL, Calif., Sept. 6, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S Food and Drug Administration (FDA) placed a clinical hold on the BMN 307 Phearless Phase 1/2 study. The Phearless study is evaluating BMN 307, an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy, in adults with phenylketonuria (PKU). The FDA's clinical hold was based on interim safety findings from a pre-clinical, non-GLP pharmacology study.
The Company carried out this pre-clinical study to understand the durability of BMN 307 activity in mice bearing two germline mutations, which may predispose the mice to the development of malignancy. One mutation eliminated the PAH gene that's missing in PKU and the second rendered the animals immunodeficient. Of 63 animals treated, six of seven animals administered BMN 307 at the highest dose group (2e14 Vg/kg) had tumors on liver necropsy 52 weeks after dosing with evidence for integration of portions of AAV vector into the genome. No lesions were observed in any mice at 24 weeks. Five of these animals had adenomas and one had a hepatocellular carcinoma (HCC). The translatability of these findings to humans is uncertain and under further investigation.
To date, the Company has only dosed humans in the Phearless Phase 1/2 clinical study with lower doses of either 2e13 vg/kg or 6e13 vg/kg. Due in part to the risk previously identified by historical rodent studies, the liver health of Phearless study participants is regularly monitored. The Company will work with the Data Review Board and Principal Investigators to further evaluate the study participants who have been dosed and will continue to monitor them over the long-term. The clinical significance of these pre-clinical rodent findings has not been established and cancers due to AAV integration have not been observed in larger animals or humans. BioMarin is pausing further enrollment into this global Phase 1/2 study until the investigation of these findings is completed. The company is working with the FDA and other health authorities and will communicate next steps for the program when available.
"More than 3,000 patients have been treated with gene therapy, and there are no reports of cancers emerging as a consequence. Acknowledging the complexity of the issue as highlighted in this week's FDA discussion, integrational mutagenesis and resultant cancer formation has been observed in mice using other AAV vectors," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "Therefore, we plan to investigate these findings. For patients who have already received lower doses of these vectors, we will continue to carefully evaluate and monitor their health. We are committed to understand and mitigate any risk of cancer causation."
About Phenylketonuria
PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.
To learn more about PKU and PAH deficiency, please visit www.PKU.com. Information on this website is not incorporated by reference into this press release.
About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on such website is not incorporated by reference into this press release.
Forward-Looking Statement
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the Company pausing enrollment globally in the Phearless Phase 1/2 study, the impact of the pre-clinical findings on the 307 clinical program, the predictability of the pre-clinical findings on human patients, the development of BioMarin's BMN 307 program generally, including the impact on the timing and process for regulatory interactions and decisions. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, the outcome of ongoing review of clinical and pre-clinical data; the outcome of additional experiments related to the preclinical findings; the results and timing of current and future clinical trials related to BMN 307; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2021 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.
Contacts:
Investors
Media
Traci McCarty
Debra Charlesworth
BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc.
(415) 455-7558
(415) 455-7451
SOURCE BioMarin Pharmaceutical Inc.
Hat sich hier seit Mitte November etwas an der Situation geändert?
Antwort auf Beitrag Nr.: 65.709.084 von Chris1979- am 14.11.20 17:47:45
Geronimo11 meint vermutlich das hier:
Letzte Woche war jeder Tiefpunkt höher als der vorherige.
Das Ding ist recht bullisch und Formation der letzten Woche schon zugespitzt.
Nach oben ist eine große Kurslücke vorhanden:
Und ich habe dieses Jahr seeeeeehr gute Erfahrungen mit solchen Lückenschließungen bei Biotechs gemacht. Man braucht nur entsprechende Nachrichten, sprich die Begleitmusik muss schon stimmen.
Die SMA20 und die SMA50 haben sich soweit beruhigt, der Boden scheint gefunden zu werden.
Im Weekly wartet der MACD schon ganz unten auf die Signallinie um diese hoffentlich nach oben zu kreuzen:
Zumindest deutet sich das an.
💡
Zitat von Chris1979-: Hi, nicht dass ich etwas dagegen einzuwenden hätte, aber woran machst du das fest?
Geronimo11 meint vermutlich das hier:
Letzte Woche war jeder Tiefpunkt höher als der vorherige.
Das Ding ist recht bullisch und Formation der letzten Woche schon zugespitzt.
Nach oben ist eine große Kurslücke vorhanden:
Und ich habe dieses Jahr seeeeeehr gute Erfahrungen mit solchen Lückenschließungen bei Biotechs gemacht. Man braucht nur entsprechende Nachrichten, sprich die Begleitmusik muss schon stimmen.
Die SMA20 und die SMA50 haben sich soweit beruhigt, der Boden scheint gefunden zu werden.
Im Weekly wartet der MACD schon ganz unten auf die Signallinie um diese hoffentlich nach oben zu kreuzen:
Zumindest deutet sich das an.
💡
Antwort auf Beitrag Nr.: 65.709.174 von Geronimo11 am 14.11.20 18:01:58 Sehe ich ja genauso , deshalb bin ich ja auch investiert . Ich dachte es gibt evtl. News.
Antwort auf Beitrag Nr.: 65.709.084 von Chris1979- am 14.11.20 17:47:45Wenn ich jetzt irgendwas über Charttechnik schreibe wäre es genauso. Ich schreibe nur ich bin sicher das biomarin steigt. Das kann man glauben oder auch nicht. Man oder man kauft nicht. Die Zukunft wird gehandelt und die schaut sehr gut aus.
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