For the first quarter ended
Revenues for the first quarter of 2015 were
In the first quarter of 2015, Sangamo recognized
Research and development expenses were
Total operating expenses for the first quarter of 2015 were
Recent Events
- Presentation of New Clinical Data at CROI 2015 from Phase 1/2 SB-728-1101 clinical trial of ZFP Therapeutic Designed to Provide Functional Control of HIV. In February 2015 Sangamo presented new clinical data from its SB-728-T program for the potential functional cure of HIV/AIDS at the Conference on Retroviruses and Opportunistic Infections (CROI 2015). The data demonstrated that Cytoxan preconditioning combined with treatment with a CD4/CD8 zinc finger nuclease (ZFN)-modified T-cell product in (Cohort 3*) reduced viral load to below the limit of quantification in one of three subjects, and delayed onset of viremia for over 8 weeks, from the start of treatment interruption (TI), in another subject. Currently, all three patients from the cohort are undergoing TI.
- FDA Acceptance of IND to Initiate Clinical Trial of ZFP Therapeutic
Approach for Beta-thalassemia. In
February 2015 Sangamo announced that the Investigational New Drug (IND) application for SB-BCLmR-HSPC, designed to provide a one-time, long-lasting treatment for beta-thalassemia, and developed in collaboration with Biogen, was accepted by theU.S. Food and Drug Administration (FDA) and is active. The IND enables Sangamo to initiate a Phase 1/2 clinical trial of the ZFP Therapeutic in transfusion-dependent patients with beta-thalassemia major. The trial will assess the safety and tolerability, and measures of efficacy of this approach. InMay 2013 Sangamo was awarded a$6.4 million Strategic Partnership Award from CIRM, providing matching funds for preclinical work to support the IND application and Phase 1/2 clinical trial to develop this ZFP Therapeutic. The trial will be carried out at multiple centers, including UCSF Benioff Children's Hospital Oakland. - FDA Acceptance of IND Application to Initiate a Phase 1 Clinical Trial of
ZFP Therapeutic approach for HIV in Hematopoietic Stem Progenitor Cells (HSPCs).
In
February 2015 Sangamo announced that subsequent to theFDA's recent acceptance of the IND application, Sangamo and its collaborators atCity of Hope expect to initiate a Phase 1 clinical trial of ZFN-modified HSPCs. - Completion and closure of CERE-110 trial in Alzheimer's disease.
Data from a clinical trial of CERE-110 in subjects with Alzheimer's disease
(AD), acquired as part of the acquisition of
Ceregene in 2013, demonstrated that direct administration into the brain of AAV encoding the nerve growth factor gene (AAV-NGF) in subjects with AD was safe and well tolerated. As previously stated, Sangamo will not pursue this approach for potential treatment of AD and has terminated the CERE-110 program. - Presentation of Preclinical Data at 11th Annual
WORLDSymposium™ Meeting Supporting IVPRP Approach for the Treatment of LSDs.
In
February 2015 the Company presented preclinical data from its In Vivo Protein Replacement Platform (IVPRP) for the development of ZFP Therapeutics for the potential cure of lysosomal storage disorders (LSDs), at the WORLDSymposium™ 2015 Meeting. The data demonstrated that genes encoding functional human enzymes, defective in the LSDs Hunter, Hurler and Gaucher syndromes, could be inserted into the albumin locus of normal mice. Following ZFN-mediated genome editing, robust levels of protein expression were observed in the liver, blood plasma and spleen, consistent with the effective production, secretion and uptake of the functional therapeutic proteins by other cells and tissues of the body. Furthermore, elevated enzyme activity in the blood plasma was sustained over the course of the two month study. - In-Licensing of mRNA Delivery Technology and Expansion of Therapeutic
Pipeline Opportunities for In Vivo ZFN Platform. In
January 2015 Sangamo in-licensed nanoparticle technology enabling systemic mRNA delivery of ZFNs. Sangamo is developing applications of this technology to enable repeat-dosing of ZFNs in vivo, providing the opportunity to "dose to effect." The ability to re-dose could expand applications of ZFN-mediated in vivo genome editing to numerous, well-established therapeutic "knock-out" targets in the liver, such as PCSK9, a gene that regulates LDL cholesterol associated with cardiovascular disease.
Financial Guidance for 2015 The Company reiterates its earlier guidance as follows:
- Cash and Investments: Sangamo expects that its cash, cash equivalents
and marketable securities will be at least
$180 million at the end of 2015, inclusive of research funding and certain milestone payments from Shire and Biogen but exclusive of funds arising from any additional new collaborations or partnerships, equity financings or other new sources. - Revenues: Sangamo expects that revenues will be in the range of
$60 million to$70 million for 2015, inclusive of research funding and certain milestone payments from Shire and Biogen. - Operating Expenses: Sangamo expects that operating expenses will be
in the range of
$100 million to$110 million for 2015.
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