ANI Pharmaceuticals Inc
WKN: A1W15D / ISIN: US00182C1036Biosante - Potential zum Tenbagger??
| eröffnet am: | 14.02.12 02:47 von: | Vollzeittrader |
| neuester Beitrag: | 24.04.21 23:25 von: | Utarxcwa |
| Anzahl Beiträge: | 572 | |
| Leser gesamt: | 83731 | |
| davon Heute: | 9 | |
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14.02.12 02:47
#1
Vollzeittrader
Biosante - Potential zum Tenbagger??
Heute (14.02.2012) steht eine eventuelle Zulassung eines Produktes aus der meiner Meinung nach starken Pipeline von Biosante an!
-ý http://www.biosantepharma.com/Products.php (Bio-t Gel)
Nach Absturz im Dezember aufgrund des "gefloppten" Libigels könnte Biosante somit vom Pennystock wieder zum Longinvest werden! Die Chancen stehen 50:50, denke ich!
http://www.nasdaq.com/symbol/bpax/real-time
Moderation
Zeitpunkt: 01.03.12 16:27
Aktion: Forumswechsel
Kommentar: Falsches Forum
Zeitpunkt: 01.03.12 16:27
Aktion: Forumswechsel
Kommentar: Falsches Forum
546 Postings ausgeblendet.
18.09.12 08:21
#549
Gropius
Nochmal ein kleiner Ausriss der Palette.
BioSante Pharmaceuticals, Inc. engagiert sich in der Lizenzierung und Entwicklung von Hormontherapie Produkten für Männer und Frauen zu behandeln. Diese Hormontherapie Produkte sind Gel-Formulierungen für die transdermale Verabreichung, die Bio-identische Estradiol und Testosteron zu liefern. Die Hauptprodukte sind Elestrin, ein transdermales bioidentical Östrogen-Gel zur Behandlung von Wechseljahrsbeschwerden bei Frauen und LibiGel, ein transdermales bioidentical Testosteron-Gel, das sich in Phase III der klinischen Entwicklung für die Behandlung von weiblicher sexueller Dysfunktion befindet. Seine anderen Produkten gehören Bio-E/P-Gel, ein transdermales Kombination Gel bioidentical Östrogen und ein Gestagen zur Behandlung von Wechseljahrsbeschwerden bei Frauen; LibiGel-E / T, ein transdermales Kombination Gel bioidentical Östrogen und Testosteron bioidentical zur Behandlung der weiblichen sexuelle Dysfunktion bei Frauen in den Wechseljahren, Bio-T-Gel, ein transdermales bioidentical Testosteron-Gel zur Behandlung von Hypogonadismus, oder Testosteronmangel bei Männern; und dreifach Hormon Kontrazeptiva. BioSante entwickelt auch seine Kalziumphosphat Nanotechnologie (CaP) für Impfstoffe, einschließlich Hepatitis B, Vogelgrippe und Biodefense Impfstoffe für Toxine wie Anthrax, sowie einem System zur Abgabe von Arzneimitteln durch alternative Verabreichungswege. Seine CaP in Entwicklung befindlichen Produkte umfassen BioVant, eine proprietäre CaP Adjuvans und Delivery-Technologie für Impfstoffe gegen Krebs, virale und bakterielle Infektionen und Autoimmunerkrankungen; BioOral, ein Abgabesystem für die orale / bukkal / intranasale Verabreichung von Proteinen und anderen Therapien.
Hier sollte es schon auf Grund von Libigel weiter gehen.
Sollte die FDA positiv entscheiden, und die Chance ist durch die Zusammenarbeit mit der FDA im Vorfeld gegeben, wird auch Sauerland seine 100% machen.
Hier sollte es schon auf Grund von Libigel weiter gehen.
Sollte die FDA positiv entscheiden, und die Chance ist durch die Zusammenarbeit mit der FDA im Vorfeld gegeben, wird auch Sauerland seine 100% machen.
18.09.12 08:38
#550
Gropius
Sollten Ihr noch wissen
Angesichts dieser jüngsten Überprüfung während der keine spezifische oder allgemeine Fragen der Sicherheit wurden erhöht, und nach umfangreichen Betrachtung BioSante kündigte außerdem den Abschluss des LibiGel Phase III Studie zur Sicherheit sofort wirksam. Vor der Einleitung des LibiGel Studie zur Sicherheit im Jahr 2008, teilte die FDA BioSante, dass die Probanden in der kardiovaskulären Ereignisses und Brustkrebs Studie zur Sicherheit, um eine minimale durchschnittliche Exposition in der Studie zur Sicherheit von 12 Monaten vor der Einreichung eines LibiGel new drug application gehabt haben ( NDA) und vor einer möglichen FDA-Zulassung von LibiGel. Zu diesem Zeitpunkt haben Probanden in dieser Studie für eine durchschnittliche Dauer von 24,5 Monaten, mehr als 3.200 Probanden wurden in der Studie für mehr als ein Jahr und mehr als 1.700 Probanden wurden für mehr als zwei Jahren eingeschrieben. Mit diesem neunten positive entblindet Überprüfung der Studie des DMC und über 7.300 Frauen Jahren der Exposition, glaubt BioSante, daß angemessene Sicherheitsmaßnahmen Daten LibiGel Nutzung Frauen in der Menopause erhalten wurde.
"Wir sind sehr zufrieden mit dieser neunten DMC Überprüfung unserer LibiGel Phase III Studie zur Sicherheit zufrieden. Basierend auf dieser neuen DMC Überprüfung der langfristigen Exposition in der Studie zur Sicherheit LibiGel und andere Überlegungen, wie wir glauben, dass wir ausreichende Exposition gegenüber Pre-Approval LibiGel Sicherheit zu etablieren und zum Abschluß der Studie haben ", erklärte Michael Snabes, MD, Ph. D., Senior Vice President für medizinische Angelegenheiten für BioSante.
"Die LibiGel Phase III Studie zur Sicherheit von BioSante durchgeführt ist die größte Testosteron Studie ihrer Art bei Männern oder Frauen, und wir haben eine große Menge von Daten zur Sicherheit über viele Jahre angesammelt. Zusätzlich zu unseren sorgfältiger Abwägung von Informationen an Bestellungen und zurück FDA-Kommunikation, wie wir glauben, dass die LibiGel Sicherheit Studie wird die FDA mit starker Beweis dafür, dass LibiGel sicher für den Einsatz bei Frauen im Klimakterium, und den Abschluss der Studie zu diesem Zeitpunkt wird in signifikantes Ergebnis liefern laufenden Kosteneinsparungen BioSante ", sagte Stephen M . Simes, BioSante President und Chief Executive Officer.
Darüber hinaus wird BioSante weiterhin die Entwicklung von Protokollen der beiden neuen LibiGel Phase III Studien zur Wirksamkeit und beabsichtigt, zusätzliche Studiendesign Informationen und den Zeitpunkt der Studie Einleitung vorlegen. Wie bereits erwähnt, beabsichtigt BioSante für eine FDA Special Protocol Assessment (SPA) Vereinbarung vor Beginn der Wirksamkeitsstudien gelten. Es wird erwartet, dass jede neue Wirksamkeitsstudie die gleichen FDA-required Endpunkte wie vor Phase III Studien zur Wirksamkeit sind: eine Erhöhung der Anzahl der befriedigenden sexuellen Ereignisse und das sexuelle Verlangen und verminderte Not verbunden mit niedrigen Wunsch. Das Unternehmen strebt eine FDA SPA Zustimmung ersten Quartal 2013.
"Wir sind sehr zufrieden mit dieser neunten DMC Überprüfung unserer LibiGel Phase III Studie zur Sicherheit zufrieden. Basierend auf dieser neuen DMC Überprüfung der langfristigen Exposition in der Studie zur Sicherheit LibiGel und andere Überlegungen, wie wir glauben, dass wir ausreichende Exposition gegenüber Pre-Approval LibiGel Sicherheit zu etablieren und zum Abschluß der Studie haben ", erklärte Michael Snabes, MD, Ph. D., Senior Vice President für medizinische Angelegenheiten für BioSante.
"Die LibiGel Phase III Studie zur Sicherheit von BioSante durchgeführt ist die größte Testosteron Studie ihrer Art bei Männern oder Frauen, und wir haben eine große Menge von Daten zur Sicherheit über viele Jahre angesammelt. Zusätzlich zu unseren sorgfältiger Abwägung von Informationen an Bestellungen und zurück FDA-Kommunikation, wie wir glauben, dass die LibiGel Sicherheit Studie wird die FDA mit starker Beweis dafür, dass LibiGel sicher für den Einsatz bei Frauen im Klimakterium, und den Abschluss der Studie zu diesem Zeitpunkt wird in signifikantes Ergebnis liefern laufenden Kosteneinsparungen BioSante ", sagte Stephen M . Simes, BioSante President und Chief Executive Officer.
Darüber hinaus wird BioSante weiterhin die Entwicklung von Protokollen der beiden neuen LibiGel Phase III Studien zur Wirksamkeit und beabsichtigt, zusätzliche Studiendesign Informationen und den Zeitpunkt der Studie Einleitung vorlegen. Wie bereits erwähnt, beabsichtigt BioSante für eine FDA Special Protocol Assessment (SPA) Vereinbarung vor Beginn der Wirksamkeitsstudien gelten. Es wird erwartet, dass jede neue Wirksamkeitsstudie die gleichen FDA-required Endpunkte wie vor Phase III Studien zur Wirksamkeit sind: eine Erhöhung der Anzahl der befriedigenden sexuellen Ereignisse und das sexuelle Verlangen und verminderte Not verbunden mit niedrigen Wunsch. Das Unternehmen strebt eine FDA SPA Zustimmung ersten Quartal 2013.
18.09.12 10:22
#552
salamand
yoo
hier noch mal einige infos - schaut alles gut aus- wenn die erst mal alle zulassungen haben - könnte es schwer rappeln in der kiste...
http://seekingalpha.com/article/...ng-catalysts-part-ii?source=nasdaq
04.10.12 22:27
#554
Joschi307
Biosante verschmelzt mit ANI Pharma
http://www.fool.com/investing/general/2012/10/04/...n-stock-deal.aspx
05.10.12 08:31
#556
Gropius
Thai, gerade Du stellst diese Frage ?
Das bedeudet Prostitutions seitens BPAX !
Sowie Simes sein Mund aufmacht, kommt nichts bei raus.
Er verschmelzt BPAX mit einer kleinen privaten Pharmafirma, wirft 53% der Anteile über Bord und macht sich aus dem Staub.
Sollte LIBIGEL noch eine Zulassung erreichen, stehen den BPAX Aktionären 40 Mio. Dollar am Gewinn zu. Würde nur rein für LIBIGEL 1,80 Dollar pro Shares bedeuten.
Wenn man sich vorstellt, das wir vor einem Jahr das 10fache an Wert hatten, ist diese Aktion ein weiterer Meilenstein an Versagen seitens Simes.
Sowie Simes sein Mund aufmacht, kommt nichts bei raus.
Er verschmelzt BPAX mit einer kleinen privaten Pharmafirma, wirft 53% der Anteile über Bord und macht sich aus dem Staub.
Sollte LIBIGEL noch eine Zulassung erreichen, stehen den BPAX Aktionären 40 Mio. Dollar am Gewinn zu. Würde nur rein für LIBIGEL 1,80 Dollar pro Shares bedeuten.
Wenn man sich vorstellt, das wir vor einem Jahr das 10fache an Wert hatten, ist diese Aktion ein weiterer Meilenstein an Versagen seitens Simes.
13.03.13 13:48
#559
martin30sm
News!
Ceregene And Alzheimer's Disease Cooperative Study Group Complete Enrollment Of CERE-110 Phase 2 Trial12:05 13.03.13
PR Newswire
SAN DIEGO, March 13, 2013
SAN DIEGO, March 13, 2013 /PRNewswire/ -- Ceregene, Inc. today announced the completion of enrollment in its double-blind Phase 2 clinical study of CERE-110 (AAV-NGF), a gene therapy product designed to deliver nerve growth factor (NGF) for the treatment of Alzheimer's disease. The clinical study was carried out in collaboration with the Alzheimer's Disease Cooperative Study (ADCS) based at the University of California San Diego (UCSD) and funded by a grant from the National Institute on Aging (NIA), part of the National Institutes of Health (NIH).
Forty-nine (49) patients with mild to moderate Alzheimer's disease were treated with CERE-110 at 10 clinical sites throughout the U.S. Approximately half of the patients received CERE-110 while the other half received an appropriate sham (placebo) surgery control treatment. Patients will be followed for a minimum of two years with respect to safety, brain imaging, as well as measures of cognitive function and quality of life. Standard tests used in Alzheimer's clinical trials.
"We are very excited that enrollment in the first blinded study of NGF gene therapy for Alzheimer's disease is now completed and appreciate the courage of all of the patients and their family members that enrolled in this very important study. We also appreciate the efforts of the ADCS and the National Institute on Aging for carrying out and supporting the operational aspects of this study" stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene, Inc., the developer of CERE-110. Dr. Ostrove added "In addition to CERE-110 for Alzheimer's disease, we have also completed enrollment in two controlled Phase 2 clinical trials of CERE-120 for Parkinson's disease. At this time, we would like to recognize the leadership role of Raymond T. Bartus, Ph.D., our chief scientific officer who led the development efforts for both of these potential products for the treatment of our most common neurodegenerative disorders".
"We were very pleased to be involved in evaluating CERE-110. We believe CERE-110 may prove to be an effective therapy for Alzheimer's disease," stated Paul Aisen, M.D., Project Director of the Phase Two trial and director of the Alzheimer's Disease Cooperative Study at University of California San Diego, a preeminent research consortium for testing new treatments for Alzheimer's disease. "If the results prove positive, which is our hope, this therapy could help the over five million people in the U.S. currently living with Alzheimer's disease in need of more effective treatment options."
About CERE-110
CERE-110 is composed of an adeno-associated viral (AAV) vector carrying the gene for NGF, a naturally occurring protein that maintains survival of nerve cells in the brain. CERE-110 is surgically injected into the Nucleus Basalis of Meynert (NBM), a brain region where cholinergic cell degeneration occurs in Alzheimer's disease. The cholinergic system is important in memory and cognitive function, and a restoration in the function of this system may improve memory in individuals with Alzheimer's disease. Delivery of NGF using an AAV vector should have the potential to induce sustained expression of NGF, resulting in long-lasting restoration of the function.
About Alzheimer's Disease
Alzheimer's disease is a progressive disorder of the brain that gradually affects one's memory and ability to learn, reason, communicate and carry out daily activities. There are now more than five million people in the United States living with Alzheimer's disease, and there is currently no cure.
About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on the delivery of nervous system growth factors for the treatment of neurodegenerative disorders using gene delivery. Ceregene's clinical programs include CERE-110, an AAV2 based vector expressing nerve growth factor for the treatment of Alzheimer's disease, and CERE-120 (AAV2-Neurturin) which has completed enrollment of a controlled Phase 2 study for Parkinson's disease. Ceregene was launched in January 2001. The company's investors include Alta Partners, MPM Capital, Hamilton BioVentures, Investor Growth Capital, California Technology Partners and BioSante Pharmaceuticals (Nasdaq:BPAX).
SOURCE Ceregene, Inc.
Quelle: PR Newswire
PR Newswire
SAN DIEGO, March 13, 2013
SAN DIEGO, March 13, 2013 /PRNewswire/ -- Ceregene, Inc. today announced the completion of enrollment in its double-blind Phase 2 clinical study of CERE-110 (AAV-NGF), a gene therapy product designed to deliver nerve growth factor (NGF) for the treatment of Alzheimer's disease. The clinical study was carried out in collaboration with the Alzheimer's Disease Cooperative Study (ADCS) based at the University of California San Diego (UCSD) and funded by a grant from the National Institute on Aging (NIA), part of the National Institutes of Health (NIH).
Forty-nine (49) patients with mild to moderate Alzheimer's disease were treated with CERE-110 at 10 clinical sites throughout the U.S. Approximately half of the patients received CERE-110 while the other half received an appropriate sham (placebo) surgery control treatment. Patients will be followed for a minimum of two years with respect to safety, brain imaging, as well as measures of cognitive function and quality of life. Standard tests used in Alzheimer's clinical trials.
"We are very excited that enrollment in the first blinded study of NGF gene therapy for Alzheimer's disease is now completed and appreciate the courage of all of the patients and their family members that enrolled in this very important study. We also appreciate the efforts of the ADCS and the National Institute on Aging for carrying out and supporting the operational aspects of this study" stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene, Inc., the developer of CERE-110. Dr. Ostrove added "In addition to CERE-110 for Alzheimer's disease, we have also completed enrollment in two controlled Phase 2 clinical trials of CERE-120 for Parkinson's disease. At this time, we would like to recognize the leadership role of Raymond T. Bartus, Ph.D., our chief scientific officer who led the development efforts for both of these potential products for the treatment of our most common neurodegenerative disorders".
"We were very pleased to be involved in evaluating CERE-110. We believe CERE-110 may prove to be an effective therapy for Alzheimer's disease," stated Paul Aisen, M.D., Project Director of the Phase Two trial and director of the Alzheimer's Disease Cooperative Study at University of California San Diego, a preeminent research consortium for testing new treatments for Alzheimer's disease. "If the results prove positive, which is our hope, this therapy could help the over five million people in the U.S. currently living with Alzheimer's disease in need of more effective treatment options."
About CERE-110
CERE-110 is composed of an adeno-associated viral (AAV) vector carrying the gene for NGF, a naturally occurring protein that maintains survival of nerve cells in the brain. CERE-110 is surgically injected into the Nucleus Basalis of Meynert (NBM), a brain region where cholinergic cell degeneration occurs in Alzheimer's disease. The cholinergic system is important in memory and cognitive function, and a restoration in the function of this system may improve memory in individuals with Alzheimer's disease. Delivery of NGF using an AAV vector should have the potential to induce sustained expression of NGF, resulting in long-lasting restoration of the function.
About Alzheimer's Disease
Alzheimer's disease is a progressive disorder of the brain that gradually affects one's memory and ability to learn, reason, communicate and carry out daily activities. There are now more than five million people in the United States living with Alzheimer's disease, and there is currently no cure.
About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on the delivery of nervous system growth factors for the treatment of neurodegenerative disorders using gene delivery. Ceregene's clinical programs include CERE-110, an AAV2 based vector expressing nerve growth factor for the treatment of Alzheimer's disease, and CERE-120 (AAV2-Neurturin) which has completed enrollment of a controlled Phase 2 study for Parkinson's disease. Ceregene was launched in January 2001. The company's investors include Alta Partners, MPM Capital, Hamilton BioVentures, Investor Growth Capital, California Technology Partners and BioSante Pharmaceuticals (Nasdaq:BPAX).
SOURCE Ceregene, Inc.
Quelle: PR Newswire
19.04.13 22:32
#562
martin30sm
News
Ceregene Reports Data From Parkinson's Disease Phase 2b Study20:05 19.04.13
PR Newswire
SAN DIEGO, April 19, 2013
SAN DIEGO, April 19, 2013 /PRNewswire/ -- Ceregene, Inc. today announced the top-line data from its double-blind, randomized, controlled Phase 2b clinical study of CERE-120 (AAV-neurturin), a gene therapy product designed to deliver the neurotrophic factor neurturin, for Parkinson's disease. The trial did not demonstrate statistically significant efficacy on the primary endpoint (UPDRS-motor off). However, one of the "key secondary endpoints" (Diary-off score), as defined and prespecified in the Statistical Analysis Plan, did produce statistically significant benefit. The trial also provided further evidence for the safety of CERE-120 and the dosing methods employed. A marked placebo effect was observed in this trial in that both the sham-surgery-control patients and the CERE-120 treated patients showed significant improvement following their surgery.
Fifty-one (51) patients with moderately advanced Parkinson's disease who could not be satisfactorily controlled with conventional Parkinson's medication were enrolled in the study at 11 leading clinical sites throughout the U.S. Approximately half of the patients received CERE-120 while the other half received sham (placebo) surgery as a control. Patients were monitored for 15-24 months to assess safety and changes in Parkinson's disease symptoms, using multiple endpoints such as the Unified Parkinson's Disease Rating Scale (UPDRS), Daily Diaries that assess motor function throughout the day, and PDQ-39 (a measure of quality of life), among others. Ceregene continues to analyze the data from this trial to gain as much information as possible.
Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene, Inc. stated, "We are disappointed that we did not achieve broader statistical significance in this small clinical trial, perhaps due in part to the marked placebo effect noted above. That said, we at Ceregene want to acknowledge the courage of all of the patients enrolled in this very important study and their family members. We also would like to acknowledge the Michael J. Fox Foundation for Parkinson's Research for their long-term support as we continued the development of this novel neurotrophic factor-based treatment with the potential to improve symptoms and also slow disease progression."
Raymond T. Bartus, Ph.D., executive vice president and chief scientific officer of Ceregene stated: "While we did not achieve the degree of efficacy we had hoped for in this trial, we are proud that our efforts have helped to establish that gene therapy can provide the enabling technology to safely deliver stable, long-term bioactive protein to targeted sites deep in the human brain and that the Parkinson's disease brain is able to show a positive response to neurotrophic factor stimulation. Hopefully, the information and insight we achieved and shared with the biomedical community will aid in the continuing effort to develop more effective therapies for many of these tragic and dehumanizing neurodegenerative diseases."
C. Warren Olanow, M.D., Chairman Emeritus of the Department of Neurology and Professor of Neuroscience at the Mount Sinai School of Medicine in New York and a clinical advisor to Ceregene noted, "This was an extremely well-conceived and designed study. It is unfortunate for patients that broader benefits from this extremely promising therapy could not be demonstrated in this clinical trial. These results illustrate how difficult it is to establish clinical efficacy with entirely novel therapeutic approaches in complicated neurological diseases. The Ceregene team, its scientific advisors and the participating clinical sites are to be congratulated for that effort and the flawless execution of this difficult scientific study."
Ceregene established a leadership position in the fields of gene therapy and neurotrophic factors for the treatment of neurodegenerative diseases. A total of over 100 patients have been safely dosed in two clinical programs: CERE-120 (AAV-NRTN) for Parkinson's disease and CERE-110 (AAV-NGF) for Alzheimer's disease. A randomized, controlled Phase 2 study of CERE-110 for Alzheimer's is continuing. It is fully enrolled and financially supported in large part by a grant from the NIH, with top line data expected by late 2014. In addition to the Parkinson's and Alzheimer's programs, Ceregene has conducted extensive preclinical work with CERE-120 for Huntington's disease, as well as another gene therapy/neurotrophic factor product (AAV-NT4) for blinding ocular diseases (such as Retinitis Pigmentosa, macular degeneration, diabetic retinopathy and glaucoma) and yet another (AAV-IGF1) for Lou Gehrig's disease (ALS). Ceregene is currently looking at strategic alternatives to advance its AAV gene therapy platform.
About CERE-120 and its Application to Treating Parkinson's Disease
CERE-120 is composed of a harmless adeno-associated virus (AAV) vector carrying the gene for neurturin, a naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping them alive and restoring function. Neurturin is a member of the same protein family as glial cell line-derived neurotrophic factor (GDNF). The two molecules have similar pharmacological properties and both have been shown to benefit the midbrain dopamine neurons that degenerate in Parkinson's disease. Degeneration of these neurons is responsible for the major motor impairments of Parkinson's disease. CERE-120 is delivered by stereotactic injection to the terminal fields (i.e., the ends of the degenerating neurons), located in an area of the brain called the putamen, as well as the cell bodies for these same neurons, located in a different area of the brain, called the substantia nigra. Once CERE-120 is delivered to the brain, it provides stable, controlled and highly targeted neurturin expression for years following a single injection, confirmed in both animal and human studies.
About Parkinson's Disease
Parkinson's disease is a progressive movement disorder that affects a million people in the United States. Its main symptoms, stiffness, tremors and slowed movements and gait, are caused by a loss of dopamine-containing nerve cells in the substantia nigra, which project their axons to the putamen. Dopamine is a neurotransmitter involved in controlling movement and coordination, so Parkinson's patients exhibit a progressive inability to initiate and control physical movements. There is currently no treatment that can reverse the degeneration of these neurons, let alone cure Parkinson's disease.
About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on the development of nervous system growth factors (neurotrophic factors) as treatments for neurodegenerative and retinal disorders using gene transfer for their delivery. The company has established a leadership position in the fields of gene therapy and neurotrophic factors, having launched 6 separate clinical trials in Parkinson's and Alzheimer's disease, enrolling a total of nearly 200 patients, over 100 of whom have been administered the gene therapy products, some several years ago, with no safety serious issues. Additionally, the company has published 2 dozen peer-reviewed scientific papers describing novel nonclinical and clinical findings. Ceregene's clinical program for Alzheimer's disease involves CERE-110, an AAV2-based vector expressing nerve growth factor (NGF). A fully enrolled multi-center, controlled Phase 2 study with CERE-110 is ongoing, conducted in collaboration with the Alzheimer's Disease Cooperative Study and partially funded by a grant from the National Institutes of Health (NIH). Ceregene was launched in January 2001. The company's investors include Alta Partners, MPM Capital, Hamilton BioVentures, Investor Growth Capital, California Technology Partners and BioSante Pharmaceuticals (Nasdaq: BPAX).
About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest private funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $325 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.
SOURCE Ceregene, Inc.
Quelle: PR Newswire
PR Newswire
SAN DIEGO, April 19, 2013
SAN DIEGO, April 19, 2013 /PRNewswire/ -- Ceregene, Inc. today announced the top-line data from its double-blind, randomized, controlled Phase 2b clinical study of CERE-120 (AAV-neurturin), a gene therapy product designed to deliver the neurotrophic factor neurturin, for Parkinson's disease. The trial did not demonstrate statistically significant efficacy on the primary endpoint (UPDRS-motor off). However, one of the "key secondary endpoints" (Diary-off score), as defined and prespecified in the Statistical Analysis Plan, did produce statistically significant benefit. The trial also provided further evidence for the safety of CERE-120 and the dosing methods employed. A marked placebo effect was observed in this trial in that both the sham-surgery-control patients and the CERE-120 treated patients showed significant improvement following their surgery.
Fifty-one (51) patients with moderately advanced Parkinson's disease who could not be satisfactorily controlled with conventional Parkinson's medication were enrolled in the study at 11 leading clinical sites throughout the U.S. Approximately half of the patients received CERE-120 while the other half received sham (placebo) surgery as a control. Patients were monitored for 15-24 months to assess safety and changes in Parkinson's disease symptoms, using multiple endpoints such as the Unified Parkinson's Disease Rating Scale (UPDRS), Daily Diaries that assess motor function throughout the day, and PDQ-39 (a measure of quality of life), among others. Ceregene continues to analyze the data from this trial to gain as much information as possible.
Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene, Inc. stated, "We are disappointed that we did not achieve broader statistical significance in this small clinical trial, perhaps due in part to the marked placebo effect noted above. That said, we at Ceregene want to acknowledge the courage of all of the patients enrolled in this very important study and their family members. We also would like to acknowledge the Michael J. Fox Foundation for Parkinson's Research for their long-term support as we continued the development of this novel neurotrophic factor-based treatment with the potential to improve symptoms and also slow disease progression."
Raymond T. Bartus, Ph.D., executive vice president and chief scientific officer of Ceregene stated: "While we did not achieve the degree of efficacy we had hoped for in this trial, we are proud that our efforts have helped to establish that gene therapy can provide the enabling technology to safely deliver stable, long-term bioactive protein to targeted sites deep in the human brain and that the Parkinson's disease brain is able to show a positive response to neurotrophic factor stimulation. Hopefully, the information and insight we achieved and shared with the biomedical community will aid in the continuing effort to develop more effective therapies for many of these tragic and dehumanizing neurodegenerative diseases."
C. Warren Olanow, M.D., Chairman Emeritus of the Department of Neurology and Professor of Neuroscience at the Mount Sinai School of Medicine in New York and a clinical advisor to Ceregene noted, "This was an extremely well-conceived and designed study. It is unfortunate for patients that broader benefits from this extremely promising therapy could not be demonstrated in this clinical trial. These results illustrate how difficult it is to establish clinical efficacy with entirely novel therapeutic approaches in complicated neurological diseases. The Ceregene team, its scientific advisors and the participating clinical sites are to be congratulated for that effort and the flawless execution of this difficult scientific study."
Ceregene established a leadership position in the fields of gene therapy and neurotrophic factors for the treatment of neurodegenerative diseases. A total of over 100 patients have been safely dosed in two clinical programs: CERE-120 (AAV-NRTN) for Parkinson's disease and CERE-110 (AAV-NGF) for Alzheimer's disease. A randomized, controlled Phase 2 study of CERE-110 for Alzheimer's is continuing. It is fully enrolled and financially supported in large part by a grant from the NIH, with top line data expected by late 2014. In addition to the Parkinson's and Alzheimer's programs, Ceregene has conducted extensive preclinical work with CERE-120 for Huntington's disease, as well as another gene therapy/neurotrophic factor product (AAV-NT4) for blinding ocular diseases (such as Retinitis Pigmentosa, macular degeneration, diabetic retinopathy and glaucoma) and yet another (AAV-IGF1) for Lou Gehrig's disease (ALS). Ceregene is currently looking at strategic alternatives to advance its AAV gene therapy platform.
About CERE-120 and its Application to Treating Parkinson's Disease
CERE-120 is composed of a harmless adeno-associated virus (AAV) vector carrying the gene for neurturin, a naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping them alive and restoring function. Neurturin is a member of the same protein family as glial cell line-derived neurotrophic factor (GDNF). The two molecules have similar pharmacological properties and both have been shown to benefit the midbrain dopamine neurons that degenerate in Parkinson's disease. Degeneration of these neurons is responsible for the major motor impairments of Parkinson's disease. CERE-120 is delivered by stereotactic injection to the terminal fields (i.e., the ends of the degenerating neurons), located in an area of the brain called the putamen, as well as the cell bodies for these same neurons, located in a different area of the brain, called the substantia nigra. Once CERE-120 is delivered to the brain, it provides stable, controlled and highly targeted neurturin expression for years following a single injection, confirmed in both animal and human studies.
About Parkinson's Disease
Parkinson's disease is a progressive movement disorder that affects a million people in the United States. Its main symptoms, stiffness, tremors and slowed movements and gait, are caused by a loss of dopamine-containing nerve cells in the substantia nigra, which project their axons to the putamen. Dopamine is a neurotransmitter involved in controlling movement and coordination, so Parkinson's patients exhibit a progressive inability to initiate and control physical movements. There is currently no treatment that can reverse the degeneration of these neurons, let alone cure Parkinson's disease.
About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on the development of nervous system growth factors (neurotrophic factors) as treatments for neurodegenerative and retinal disorders using gene transfer for their delivery. The company has established a leadership position in the fields of gene therapy and neurotrophic factors, having launched 6 separate clinical trials in Parkinson's and Alzheimer's disease, enrolling a total of nearly 200 patients, over 100 of whom have been administered the gene therapy products, some several years ago, with no safety serious issues. Additionally, the company has published 2 dozen peer-reviewed scientific papers describing novel nonclinical and clinical findings. Ceregene's clinical program for Alzheimer's disease involves CERE-110, an AAV2-based vector expressing nerve growth factor (NGF). A fully enrolled multi-center, controlled Phase 2 study with CERE-110 is ongoing, conducted in collaboration with the Alzheimer's Disease Cooperative Study and partially funded by a grant from the National Institutes of Health (NIH). Ceregene was launched in January 2001. The company's investors include Alta Partners, MPM Capital, Hamilton BioVentures, Investor Growth Capital, California Technology Partners and BioSante Pharmaceuticals (Nasdaq: BPAX).
About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest private funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $325 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.
SOURCE Ceregene, Inc.
Quelle: PR Newswire
10.07.20 14:20
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