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Sangamo BioSciences Provides Update on Company's 2008 Progress and 2009 Objectives
NEW YORK, Dec. 3 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc.(Nasdaq: SGMO) announced that the company will provide an update on milestonesachieved in 2008 and preview objectives for 2009 during its annual Investorand Analyst Briefing to be held in New York City today.
David Smoller, Ph.D., President of Sigma-Aldrich Corporation's ResearchBiotech Business Unit will discuss the progress and expected potential of thetwo companies' collaboration to commercialize zinc finger DNA-binding protein(ZFP) technology for research applications. Dr. Smoller will be joined byEdward Lanphier, Sangamo's president and CEO and other members of Sangamo'ssenior management team.
"Sigma is committed to providing breakthrough products to its customers inlaboratories worldwide and we expect Sangamo's ZFP technology to be nothingshort of revolutionary for research applications," commented Dr. Smoller."Marketed under the trademark CompoZr, the zinc finger nuclease (ZFN)technology makes available, for the first time, simple, efficient genomicediting. CompoZr(TM) ZFN reagents can be used to efficiently create modifiedcell lines and model organisms with precise and heritable gene deletions,insertions or modifications using simple, widely used laboratory procedures.This is proven technology with strong potential."
"The past year has been a year of real progress for our ZFP technologyplatform," said Edward Lanphier, president and CEO of Sangamo. "We aresuccessfully translating the broad applicability of our technology intoopportunities for both novel human therapeutics and non-therapeutic industrialapplications. Our unique hybrid business model maximizes the value of our ZFPtechnology platform and has enabled us to make significant progress in movingour lead ZFP Therapeutic into multiple Phase 2 clinical trials and advancingour preclinical pipeline, while maintaining a strong balance sheet. We haveestablished important commercial agreements in the non-therapeutic aspects ofour technology with Dow Agrosciences in plant agriculture and Sigma-AldrichCorporation in research reagents. We also have agreements with Genentech andPfizer in cell-line engineering. We plan to establish strategic partnershipsaround our therapeutic programs at points of value inflection and expect 2009to be another year of significant accomplishments for Sangamo."
Mr. Lanphier continued, "We are pursuing a broad development program forour lead ZFP Therapeutic(TM), SB-509, an activator of vascular endothelialgrowth factor (VEGF) with three Phase 2 clinical trials ongoing for thetreatment of diabetic neuropathy (DN) and one to evaluate its use in thetreatment of amyotrophic lateral sclerosis (ALS). We have also advanced ourpreclinical pipeline and expect to file Investigational New Drug (IND)application for the first of our ZFN-based therapeutic programs for HIV/AIDSbefore the end of 2008.
"We are very pleased by the progress of our non-therapeutic collaborationsthis year. Our partner Sigma-Aldrich successfully launched the CompoZr brandof ZFN-based gene editing tools and we look forward to the increased interestand further validation of the technology that this broadening of the user-basewill bring. We also transitioned ahead of schedule into the commercial phaseof our collaboration with Dow AgroSciences (DAS) to apply our ZFP technologyfor plant agriculture and expect DAS to begin to sublicense the technology inthat industry. The success of these activities is key to further enhancing themarket-leading presence for our technology. We believe that our progress inadvancing our technology platform, which is unique in its generation of novel,highly differentiated therapies and products, will create interest amongpotential pharmaceutical partners for our ZFP Therapeutic programs."
Sangamo Accomplishments in 2008
During the briefing several of the company's achievements will behighlighted including:
Clinical
-- Presentation of the full data set from single dose Phase 1b study(SB-509-401) of SB-509 in subjects with diabetic neuropathy (DN) demonstratingthat the drug is well-tolerated and statistically and clinically significantimprovements in measures of nerve health at 180 days post treatment.
-- Initiation of repeat dosing Phase 2 clinical trials of SB-509 in stemcell mobilization (SB-509-703) and ALS (SB-509-801).
-- Completion of enrollment and expansion of Phase 2 clinical trial insubjects with moderate to severe DN (SB-509-701). Presentation of encouragingpositive interim data from this trial.
-- Presentation of top line Phase 2 data from repeat dosing study ofSB-509 in mild to moderate DN (SB-509-601) demonstrating that the drug iswell-tolerated in a repeat-dosing setting; however, no significant differenceswere observed in improvement in measures of nerve health between placebo andtreated subjects at primary evaluation point 180 days post treatment. Analysisof these data is ongoing.
Business
-- Dow AgroSciences announces early exercise of an option for a commerciallicense for ZFP technology in plant agriculture.
-- Official launch of Sigma-Aldrich CompoZr platform of zinc fingernuclease (ZFN) reagents for gene-editing.
-- Entry into Research and License Agreement with Genentech, Inc. for cellline engineering and License Agreement with Open Monoclonal Technologies (OMT)and Roche for transgenic animal generation.
Financial
-- Sangamo updated guidance on its expectations of 2008 year end cash to$60M in cash and cash equivalents, which the company believes is sufficient tofund operations through 2010 based on current expense projections.
Preclinical / Research
-- Publication of data supporting Sangamo's first ZFN Therapeutic programfor the treatment of HIV/AIDS in high-impact journal Nature Biotechnologydemonstrating highly efficient and specific ZFN-modification of CCR5 in humanT-cells resulting in an increase in CD4 T-cell counts and a reduction in viralload in an animal model of HIV infection (Perez et al., 2008, NatBiotechnol.,26: 808).
-- Nature Biotechnology publication establishing the utility of ZFNs inthe generation of transgenic animals and the potential broadening of theproduction of knockout transgenic models beyond mouse models (Doyon et al.,2008, Nat. Biotechnol. 26: 702).
-- Publication of data in the journal Proceedings of the National Academyof Sciences (PNAS) establishing Sangamo's ZFN technology as a new approach forrapid generation of "knock-out" cell lines (Santiago et al., 2008, PNAS105: 5809).
Select 2009 Objectives
During the briefing Sangamo will also discuss the following objectives for2009:
-- Sangamo expects to file an IND for SB-313 to evaluate the drug in aPhase 1 trial in subjects with glioblastoma.
-- The company expects to present data from its three ongoing Phase 2clinical trials of SB-509 in subjects with DN, SB-509-601 in subjects withmild to moderate DN, SB-509-701 subjects with moderate to severe DN with ablocked nerve and SB-509-703 in stem cell mobilization.
-- Establishment of a strategic partnership for SB-509.
-- Sangamo estimates that it expects to end 2009 with approximately $40Min cash and cash equivalents, based on current expense projections andexpected progress in existing corporate relationships.
The presentation from today's Investor and Analyst Briefing will bearchived on Sangamo's website and is available via a link in the Investorsection at http://investor.sangamo.com/index.cfm under "Events andPresentations".
About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development ofnovel DNA-binding proteins for therapeutic gene regulation and modification.The most advanced ZFP Therapeutic(TM) development program is currently inPhase 2 clinical trials for evaluation of safety and clinical effect inpatients with diabetic neuropathy and ALS. Other therapeutic developmentprograms are focused on cancer, HIV/AIDS, neuropathic pain, nerveregeneration, Parkinson's disease and monogenic diseases. Sangamo's corecompetencies enable the engineering of a class of DNA-binding proteins knownas zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognizea specific DNA sequence Sangamo has created ZFP transcription factors(ZFP TF(TM)) that can control gene expression and, consequently, cellfunction. Sangamo is also developing sequence-specific ZFP Nucleases (ZFN(TM))for gene modification. Sangamo has established strategic partnerships withcompanies outside of the human therapeutic space including Dow AgroSciences,Sigma-Aldrich Corporation and several companies applying its ZFP technology toenhance the production of protein pharmaceuticals. For more information aboutSangamo, visit the company's web site at http://www.sangamo.com.
This press release may contain forward-looking statements based onSangamo's current expectations. These forward-looking statements include,without limitation, references to the research and development of novel ZFPTFs and ZFNs as ZFP Therapeutics, applications of Sangamo's ZFP TF technologyplatform to specific human disease as well as plant agriculture, high valueresearch reagents and cell-line engineering, clinical trials of ZFPTherapeutics and expected filing of INDs, establishing strategic partnershipsfor SB-509 and other therapeutic programs, anticipated amount of cash and cashequivalents and sufficiency of cash reserve to fund operations. Actual resultsmay differ materially from these forward-looking statements due to a number offactors, including technological challenges, uncertainties relating to theinitiation, completion and outcome of stages of ZFP Therapeutic clinicaltrials, Sangamo's ability to develop commercially viable products andtechnological developments by our competitors. See Sangamo's SEC filings, andin particular, the risk factors described in Sangamo's Annual Report on Form10-K and its most recent Quarterly Reports on Form 10-Q. Sangamo BioSciences,Inc. assumes no obligation to update the forward-looking information containedin this press release.
SOURCE Sangamo BioSciences, Inc.
"Sigma is committed to providing breakthrough products to its customers inlaboratories worldwide and we expect Sangamo's ZFP technology to be nothingshort of revolutionary for research applications," commented Dr. Smoller."Marketed under the trademark CompoZr, the zinc finger nuclease (ZFN)technology makes available, for the first time, simple, efficient genomicediting. CompoZr(TM) ZFN reagents can be used to efficiently create modifiedcell lines and model organisms with precise and heritable gene deletions,insertions or modifications using simple, widely used laboratory procedures.This is proven technology with strong potential."
"The past year has been a year of real progress for our ZFP technologyplatform," said Edward Lanphier, president and CEO of Sangamo. "We aresuccessfully translating the broad applicability of our technology intoopportunities for both novel human therapeutics and non-therapeutic industrialapplications. Our unique hybrid business model maximizes the value of our ZFPtechnology platform and has enabled us to make significant progress in movingour lead ZFP Therapeutic into multiple Phase 2 clinical trials and advancingour preclinical pipeline, while maintaining a strong balance sheet. We haveestablished important commercial agreements in the non-therapeutic aspects ofour technology with Dow Agrosciences in plant agriculture and Sigma-AldrichCorporation in research reagents. We also have agreements with Genentech andPfizer in cell-line engineering. We plan to establish strategic partnershipsaround our therapeutic programs at points of value inflection and expect 2009to be another year of significant accomplishments for Sangamo."
Mr. Lanphier continued, "We are pursuing a broad development program forour lead ZFP Therapeutic(TM), SB-509, an activator of vascular endothelialgrowth factor (VEGF) with three Phase 2 clinical trials ongoing for thetreatment of diabetic neuropathy (DN) and one to evaluate its use in thetreatment of amyotrophic lateral sclerosis (ALS). We have also advanced ourpreclinical pipeline and expect to file Investigational New Drug (IND)application for the first of our ZFN-based therapeutic programs for HIV/AIDSbefore the end of 2008.
"We are very pleased by the progress of our non-therapeutic collaborationsthis year. Our partner Sigma-Aldrich successfully launched the CompoZr brandof ZFN-based gene editing tools and we look forward to the increased interestand further validation of the technology that this broadening of the user-basewill bring. We also transitioned ahead of schedule into the commercial phaseof our collaboration with Dow AgroSciences (DAS) to apply our ZFP technologyfor plant agriculture and expect DAS to begin to sublicense the technology inthat industry. The success of these activities is key to further enhancing themarket-leading presence for our technology. We believe that our progress inadvancing our technology platform, which is unique in its generation of novel,highly differentiated therapies and products, will create interest amongpotential pharmaceutical partners for our ZFP Therapeutic programs."
Sangamo Accomplishments in 2008
During the briefing several of the company's achievements will behighlighted including:
Clinical
-- Presentation of the full data set from single dose Phase 1b study(SB-509-401) of SB-509 in subjects with diabetic neuropathy (DN) demonstratingthat the drug is well-tolerated and statistically and clinically significantimprovements in measures of nerve health at 180 days post treatment.
-- Initiation of repeat dosing Phase 2 clinical trials of SB-509 in stemcell mobilization (SB-509-703) and ALS (SB-509-801).
-- Completion of enrollment and expansion of Phase 2 clinical trial insubjects with moderate to severe DN (SB-509-701). Presentation of encouragingpositive interim data from this trial.
-- Presentation of top line Phase 2 data from repeat dosing study ofSB-509 in mild to moderate DN (SB-509-601) demonstrating that the drug iswell-tolerated in a repeat-dosing setting; however, no significant differenceswere observed in improvement in measures of nerve health between placebo andtreated subjects at primary evaluation point 180 days post treatment. Analysisof these data is ongoing.
Business
-- Dow AgroSciences announces early exercise of an option for a commerciallicense for ZFP technology in plant agriculture.
-- Official launch of Sigma-Aldrich CompoZr platform of zinc fingernuclease (ZFN) reagents for gene-editing.
-- Entry into Research and License Agreement with Genentech, Inc. for cellline engineering and License Agreement with Open Monoclonal Technologies (OMT)and Roche for transgenic animal generation.
Financial
-- Sangamo updated guidance on its expectations of 2008 year end cash to$60M in cash and cash equivalents, which the company believes is sufficient tofund operations through 2010 based on current expense projections.
Preclinical / Research
-- Publication of data supporting Sangamo's first ZFN Therapeutic programfor the treatment of HIV/AIDS in high-impact journal Nature Biotechnologydemonstrating highly efficient and specific ZFN-modification of CCR5 in humanT-cells resulting in an increase in CD4 T-cell counts and a reduction in viralload in an animal model of HIV infection (Perez et al., 2008, NatBiotechnol.,26: 808).
-- Nature Biotechnology publication establishing the utility of ZFNs inthe generation of transgenic animals and the potential broadening of theproduction of knockout transgenic models beyond mouse models (Doyon et al.,2008, Nat. Biotechnol. 26: 702).
-- Publication of data in the journal Proceedings of the National Academyof Sciences (PNAS) establishing Sangamo's ZFN technology as a new approach forrapid generation of "knock-out" cell lines (Santiago et al., 2008, PNAS105: 5809).
Select 2009 Objectives
During the briefing Sangamo will also discuss the following objectives for2009:
-- Sangamo expects to file an IND for SB-313 to evaluate the drug in aPhase 1 trial in subjects with glioblastoma.
-- The company expects to present data from its three ongoing Phase 2clinical trials of SB-509 in subjects with DN, SB-509-601 in subjects withmild to moderate DN, SB-509-701 subjects with moderate to severe DN with ablocked nerve and SB-509-703 in stem cell mobilization.
-- Establishment of a strategic partnership for SB-509.
-- Sangamo estimates that it expects to end 2009 with approximately $40Min cash and cash equivalents, based on current expense projections andexpected progress in existing corporate relationships.
The presentation from today's Investor and Analyst Briefing will bearchived on Sangamo's website and is available via a link in the Investorsection at http://investor.sangamo.com/index.cfm under "Events andPresentations".
About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development ofnovel DNA-binding proteins for therapeutic gene regulation and modification.The most advanced ZFP Therapeutic(TM) development program is currently inPhase 2 clinical trials for evaluation of safety and clinical effect inpatients with diabetic neuropathy and ALS. Other therapeutic developmentprograms are focused on cancer, HIV/AIDS, neuropathic pain, nerveregeneration, Parkinson's disease and monogenic diseases. Sangamo's corecompetencies enable the engineering of a class of DNA-binding proteins knownas zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognizea specific DNA sequence Sangamo has created ZFP transcription factors(ZFP TF(TM)) that can control gene expression and, consequently, cellfunction. Sangamo is also developing sequence-specific ZFP Nucleases (ZFN(TM))for gene modification. Sangamo has established strategic partnerships withcompanies outside of the human therapeutic space including Dow AgroSciences,Sigma-Aldrich Corporation and several companies applying its ZFP technology toenhance the production of protein pharmaceuticals. For more information aboutSangamo, visit the company's web site at http://www.sangamo.com.
This press release may contain forward-looking statements based onSangamo's current expectations. These forward-looking statements include,without limitation, references to the research and development of novel ZFPTFs and ZFNs as ZFP Therapeutics, applications of Sangamo's ZFP TF technologyplatform to specific human disease as well as plant agriculture, high valueresearch reagents and cell-line engineering, clinical trials of ZFPTherapeutics and expected filing of INDs, establishing strategic partnershipsfor SB-509 and other therapeutic programs, anticipated amount of cash and cashequivalents and sufficiency of cash reserve to fund operations. Actual resultsmay differ materially from these forward-looking statements due to a number offactors, including technological challenges, uncertainties relating to theinitiation, completion and outcome of stages of ZFP Therapeutic clinicaltrials, Sangamo's ability to develop commercially viable products andtechnological developments by our competitors. See Sangamo's SEC filings, andin particular, the risk factors described in Sangamo's Annual Report on Form10-K and its most recent Quarterly Reports on Form 10-Q. Sangamo BioSciences,Inc. assumes no obligation to update the forward-looking information containedin this press release.
SOURCE Sangamo BioSciences, Inc.
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