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CERE-120 (neurturin)


Neuroprotection

For information on 2nd Ceregene phase I/II trial, see record for CERE-120 (new protocol)

Gene Therapy

Ceregene is developing nervous system growth factor gene therapy for PD. This approach aims to deliver growth factors into the nigrostriatal system in an attempt to prevent the degeneration of substantia nigra neurons and the accompanying loss of the neurotransmitter dopamine, utilizing in vivo delivery of Neurturin (a nerve growth factor) genes through an adeno-associated viral (AAV) vector delivery system. (Company web site)
 


 


Neurturin is  a member of the same protein family as GDNF and  has similar pharmacological properties.

"Phase I clinical trial of CERE-110 for Alzheimer's began 10/04. Results published in May 2005 Nature Medicine Online.""The results of this Phase I study of nerve growth factor(NGF) in Alzheimer's disease are encouraging and strongly support our CERE-110"

Preclinical studies of CERE120 for Huntington's Disease reported in September 2006 at Rush University.


According to partnership with Genzyme, announced 6/21/07, Genzyme will gain marketing rights in all markets outside of the US and Canada... Ceregene will retain exclusive rights to CERE-120 in the US and Canada.

In Clinical Trials (US)

Trial is not recruiting Patients

Grant of $740,000 from Michael J. Fox Foundation awarded. In Oct. 2006 Fox Foundation also awarded $1.9 Million for Phase II Efficacy Study. Results of Phase I trial, published in April 2008, reported that "The initial data support the safety, tolerability, and potential efficacy of CERE-120 as a possible treatment for PD; however, these results must be viewed as preliminary until data from blinded, controlled clinical trials are available." (Click on phase I below for more information.)

November 26, 2008 – Ceregene, Inc., "reported clinical data from a double-blind, controlled Phase 2 trial of CERE-120 in 58 patients with advanced Parkinson’s disease. The trial did not demonstrate an appreciable difference between patients treated with CERE-120 versus those in the control group. Both groups showed an approximate 7 point improvement in the protocol-defined primary endpoint (Unified Parkinson’s Disease Rating Scale- motor off score at 12 months), relative to a mean at baseline of approximately 39 points. Both groups had a substantial number of patients who demonstrated a meaningful clinical improvement from baseline. CERE-120 appeared to be safe and well tolerated." (company press release)

May 2009, Ceregene reported that based on an additional analyses of data from "30 subjects who continued to be evaluated under double-blind conditions for up to 18 months, there were increasing effects of CERE-120 over time. There was a "clinically modest but statistically significant treatment effect in the primary efficacy measure (UPDRS motor off; p=0.025), as well as similar effects on several more secondary motor measures (p<0.05), were seen at the 18 month endpoint." A future clinical trial is being planned.(Ceregene press release) (See phase II trial results.) In July 2009, "The Michael J. Fox Foundation agreed to fund the long-term, open-label analysis of data from Ceregene’s Phase 2 trial of CERE-120...The funding will allow Ceregene to collect and analyze data from trial enrollees for another 48 months. While the study will be unblinded, the goal is to gather as much data on safety and efficacy as possible in an open-label setting, while looking for suggestions of a longer-term neuroprotective effect. " Recruitment for a new phase I/II trial was announced in Oct. 2009.

Sponsor(s)
Sponsor (click for details) URL
Primary Sponsor Ceregene, Inc. http://www.ceregene.com
Co-Sponsor 1 Genzyme Corporation (GENZ) http://www.genzyme.com/corp/structure/corp_home.asp
Regulatory Information

"Reviewed by the FDA and has an effective IND as well as approval from the Recombinant DNA Advisory Committee (RAC). " (PR Newswire)
Phase II protocol reviewed by RAC Sept. 20 2006 and was approved.  Webcast of this meeting available  at http://www4.od.nih.gov/oba/rac/meeting.html

Review of protocol for the new phase I/II trial took place and was approved at the June 17, 2009 RAC committee meeting.
click on http://oba.od.nih.gov/rdna_rac/rac_past_meetings_2000.html

Active

Not Yet Filed
Treatment Phase Details
Click on Phase Number for DETAILED INFORMATION on that phase
Phase Status Title of Trial Start Date Duration Weeks
Preclinical Phase Completed       Ceregene, Inc.
Safety Review Not Started       Ceregene, Inc.
Phase 1 Phase Completed A Phase I, Open-Label Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin[NTN] to Assess the Safety and Tolerability of Intrastriatal Delivery to Subjects With Idiopathic Parkinson's Disease June 2005   Ceregene, Inc.
Phase 2 Phase Completed Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 For Efficacy and Safety in Subjects With Idiopathic Parkinson's Disease Nov. 2006   Ceregene, Inc.
Phase 3 Not Started      
Phase 4 Not Started      
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Partnerships and Agreements
Description Notes Url1 Url2
The Michael J. Fox Foundation signed on in July 2009 to fund the long-term, open-label analysis of data from Ceregene, Inc.’s Phase 2 trial of CERE-120. The funding will allow Ceregene to collect and analyze data from trial enrollees for another 48 months. http://michaeljfox.org/newsEvents_mjffInTheNews_article.cfm?ID=326
Michael J. Fox Foundation Awards $1.9 Million to Support Ceregene Phase II Gene Therapy Clinical Trial "Foundation funding will be used to provide general support for the Phase II trial and will enable Ceregene’s program... to gather more long-term efficacy and safety data than would otherwise be possible, making the results of the trial more robust. Assuming positive safety and efficacy results, this additional data may also help identify trends that could aid in the design of future trials to test CERE-120’s potential neuroprotective effect in patients." (October 2006, Source: Foundation website) http://www.michaeljfox.org/newsEvents_mjffInTheNews_article.cfm?ID=134
In Dec. 2002, Ceregene exclusively licensed the neurturin gene from Washington University. The license covers gene therapies for Parkinson's Disease and other neurological disorders. "Ceregene signed a licensing agreement with Washington University in St. Louis, MO for exclusive worldwide rights to the gene expressing Neurturin, a nervous system growth factor, for use in neurological gene therapies. The Neurturin protein and gene was discovered by Drs. Eugene M. Johnson, Jr., Jeffrey D. Milbrandt and colleagues at Washington University School of Medicine." (Ceregene press release, Dec. 2002) http://www.ceregene.com/press_120402.asp
Michael J. Fox Foundation provides a $740,000 grant for Phase I trial. "MJFF support will significantly enhance the speed and depth of this data collection, allowing for more regular testing of a wider range of neurological functions than would otherwise be possible. Each patient will undergo a PET scan and a full battery of neurological tests every three months (in addition to regular visits to the clinic for more routine procedures such as blood tests). Assuming successful results in the first study, measurements from the Phase I study will lead to more efficient planning of a larger Phase II study that will gather more detailed data on both safety and efficacy." http://www.michaeljfox.org/newsEvents_mjffInTheNews_pressReleases_article.cfm?ID=121
June 21, 2007 - Ceregene, Inc. and Genzyme Corporation announced that they have entered into a partnership for the development and commercialization of CERE-120. "Under the terms of the agreement, Genzyme will pay Ceregene a $25 million up-front payment in exchange for certain partnership-related expenses. Ceregene will also be entitled to development-related milestone payments of up to $125 million and 50 percent reimbursement of the worldwide Phase 3 development costs. Genzyme will gain marketing rights in all markets outside of the US and Canada, and Genzyme will pay Ceregene tiered royalties based on sales in markets where it has rights. Ceregene will retain exclusive rights to CERE-120 in the US and Canada." (Genzyme press release) http://www.genzyme.com/corp/media/GENZ%20PR-062107.asp#TopOfPage
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Citations for this treatment information
Authors Title Source Citation Date Online Ret Date VolumeSort by this column Pages URL
Kordower, Jeffrey, et al. Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys Annals of Neurology 2006   60 706-715
Mehdi Gasmi1, Christopher D Herzog1, Eugene P Brandon1, Justine J Cunningham1, G Anthony Ramirez1, Elias T Ketchum1 and Raymond T Bartus Striatal Delivery of Neurturin by CERE-120, an AAV2 Vector for the Treatment of Dopaminergic Neuron Degeneration in Parkinson's Disease Molecular Therapy Jan. 2007 23-MAR-08 v. 15 no. 1 62-68 http://npg.nature.com/mt/journal/v15/n1/full/6300010a.html
Cynthia Robbins-Roth Location, Location, Location: Ceregene Bets on Better Delivery BioWorld Today March 12, 2007 31-MAR-07     id=339&sehttp://www.ceregene.com/pdfs/BioWorld_Ceregene_031207.pdfc=4
Marks, W.J., et al. Phase 1 study of putaminal gene transfer with adeno-associated virus serotype-2[AAV2]-neurturin [NTN] (CERE-120) for Parkinson's disease: Preliminary observations 58th Annual Meeting of the American Academy of Neurology, San Diego California, April 1-8, 2006. Poster, session       A185
William J Marks Jr, Jill L Ostrem, Leonard Verhagen, Philip A Starr, Paul S Larson, Roy A E Bakay, Robin Taylor, Deborah A Cahn-Weiner, A Jon Stoessl, C Warren Olanow, Raymond T Bartus Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2–neurturin) to patients with idiopathic Parkinson’s disease: an open-label, phase I trial The Lancet Neurology April 2, 2008 (online) 10-APR-08    
  Safety of CERE-120 (AAV2-NTN) in Subjects With Idiopathic Parkinson's Disease Clinicaltrials.gov March 7, 2006 27-JUL-06     http://www.clinicaltrials.gov/ct/show/NCT00252850?order=1
  Ceregene Reports Initiation of Phase 1 Clinical Trial of Novel Gene Therapy for Patients With Parkinson's Disease PR Newswire September 21, 2005      
  Safety of CERE-120 (AAV2-NTN) in Subjects With Idiopathic Parkinson's Disease Clinicaltrials.gov March 7, 2006 27-JUL-06     http://www.clinicaltrials.gov/ct/show/NCT00252850?order=1
  Ceregene Presents Additional Clinical Data from Phase 2 Trial of CERE-120 for Parkinson's Disease (press release) PR Newswire May 27, 2009 27-MAY-09     http://news.prnewswire.com/ViewContent.aspx?ACCT=109&STORY=/www/story/05-27-2009/00050 32921&EDATE=
  Ceregene and Genzyme Announce Partnership for the Development and Commercialization of CERE-120 for Parkinson’s Disease (press release) Genzyme website June 21, 2007 22-JUN-07     http://www.genzyme.com/corp/media/GENZ%20PR-062107.asp#TopOfPage
  Ceregene Presents Long-term Follow-up From Phase 1 Trial of CERE-120 in Parkinson's Disease (press release) PRNewswire Dec.12, 2007 13-DEC-07     http://biz.yahoo.com/prnews/071212/aqw066.html?.v=34
  Michael J. Fox Foundation Announces Funding for Ceregene Phase-1 Gene Therapy Clinical Trial. U.S. Newswire November 28, 2005 27-JUL-06     http://releases.usnewswire.com/GetRelease.asp?id=57198
  Gene therapy is respectable again. Business Week November 23, 2005       http://www.businessweek.com/technology/content/nov2005/tc20051123_355639.htm
  Minutes of Recombinant DNA Advisory Committee Discussion of Human Gene Transfer Protocol #0607-788 NIH Recombinant DNA and Gene Transfer Meetings and Conferences (web site) For webcast of meeting go to http://www4.od.nih.gov/oba/rac/meeting.html and click on "Minutes and Webcasts-- September 20, 2006." September 20, 2006 21-NOV-06     http://www4.od.nih.gov/oba/rac/minutes/RAC_Minutes_09-06.pdf
  Ceregene Announces Clinical Data from Phase 2 Clinical Trial of CERE-120 for Parkinson's Disease (press release) Ceregene web site Nov. 26, 2008 02-DEC-08     www.ceregene.com/press_112608.asp
NextNext
Related information
Description Information
FDA reviewing all ongoing trials involving any use of adeno-associated virus (AAV) - announced July 26, 2007

Excerpts from FDA Statement on Gene Therapy Clinical Trial (July 26, 2007)

"On July 24, 2007 the U.S. Food and Drug Administration (FDA) was informed by Targeted Genetics Corporation of Seattle about the death of a patient who received an investigational gene therapy product in a clinical trial for the treatment of active inflammatory arthritis...

The product that was being studied uses a particle called a vector that is designed to deliver treatment genes to target cells. The vector used is a recombinant adeno-associated virus (AAV) derived vector and delivers the gene for Tumor-Necrosis Factor -Receptor, with the intent to inhibit a key mediator of inflammation. In the study, the gene therapy was administered into the joint affected by the disease to reduce inflammation and disease in patients with active inflammatory arthritis. ...

FDA is not aware of similar adverse events occurring in other gene therapy trials either with this specific product or with those that use other genes in AAV vectors. However, as a precaution, the agency is further reviewing all ongoing trials involving any use of AAV. "

Full FDA statement at:

http://www.fda.gov/bbs/topics/NEWS/2007/NEW01672.html

  
BioSante owns a 16% equity ownership position in Ceregene (10/09) On Oct. 14, 2009 "BioSante and Cell Genesys, Inc. announced the successful completion of the merger of Cell Genesys with and into BioSante, with BioSante as the surviving company... BioSante now owns a 16 percent equity ownership position in Ceregene, Inc., a former subsidiary of Cell Genesys which is developing gene therapies for neurodegenerative disorders...." (BusinessWire)
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