Sangamo Biosciences, Inc. (NASDAQ:SGMO) reported its third quarter 2016 financial results and provided an update on recent events and development timelines for its therapeutic programs.
“The third quarter of 2016 has been a pivotal time for Sangamo, as we worked to focus our efforts and execute on our prioritized therapeutic programs in hemophilia B, hemophilia A, MPS I and MPS II,” said Sandy Macrae, M.B., Ch.B., Ph.D., Sangamo’s president and chief executive officer. “I am pleased to announce that the Phase 1/2 clinical trial for SB-FIX, our in vivo genome editing program for hemophilia B, is open. We are also on track to file an IND application for our AAV cDNA Factor 8 gene therapy program for hemophilia A by the end of this year. In addition, we submitted the additional data package for our MPS I and MPS II programs to the FDA in September, and I am pleased to report that the FDA has cleared these programs for clinical development. Preparations are now underway to initiate Phase 1/2 clinical trials for these indications in early 2017.”
Dr. Macrae continued, “We also made a number of organizational changes, including several key hires in our clinical and technical operations teams and instituted new procedures in order to position the company for clinical success. I am very encouraged by the commitment of our entire team and the progress we have made in the third quarter to drive these activities forward. I remain confident that we can demonstrate the value and therapeutic potential of our genome editing and gene therapy platforms and with reliable steps, make sensible progress and realize our vision of transforming Sangamo into a patient-focused therapeutics company.”
- Initiation of FIXtendz (SB-FIX-1501) Phase 1/2 clinical trial designed to assess safety, tolerability and potential efficacy of SB-FIX in adults with hemophilia B. In October, Sangamo opened the first clinical study of an in vivo genome editing therapeutic, its Phase 1/2 clinical trial (FIXtendz, SB-FIX-1501). SB-FIX-1501 is an open-label, dose-escalation study in male subjects over eighteen years of age, with severe hemophilia B, who do not have inhibitors or hypersensitivity to recombinant Factor IX protein (rFIX). The study will enroll up to nine subjects in three dosing cohorts of two subjects per cohort, with additional subjects to be enrolled at the optimal therapeutic dose, and will evaluate the safety and potential efficacy of a single administration of SB-FIX.
- U.S. Food and Drug Administration (FDA) grants orphan drug designation to SB-FIX, the first in vivo genome editing therapeutic in development. In September, Sangamo announced that the FDA granted Orphan Drug Designation (ODD) to SB-FIX, the company’s zinc finger nuclease (ZFN)-mediated in vivo genome editing therapeutic candidate for hemophilia B. Orphan drug designation is granted to investigational drugs and biologics that are intended to treat rare diseases that affect fewer than 200,000 people in the U.S. This designation helps facilitate drug development by providing several benefits to drug developers, including assistance with clinical study design and drug development, tax credits for qualified clinical trial costs, exemption from certain FDA application fees and seven years of market exclusivity upon regulatory product approval.
- FDA clearance to initiate Phase 1/2 clinical trials for SB-318 (MPS I) and SB-913 (MPS II) therapeutic programs. Sangamo submitted the additional in vitro studies requested by the FDA in September and recently received clearance to initiate Phase 1/2 clinical trials for the Mucopolysaccharidosis Type I (MPS I, Hurler syndrome) and Mucopolysaccharidosis Type II (MPS II, Hunter syndrome) programs based on its ZFN-mediated in vivo genome editing therapeutic platform. The company expects to initiate the clinical studies in early 2017.
- Appointment of new head of technical operations. In August, Sangamo appointed Mohammad El-Kalay, Ph.D., as Vice President, Technical Operations. Dr. El-Kalay brings over 25 years of operational management experience in the life sciences field, including expertise in process development and cGMP manufacturing operations at clinical scale with hematopoietic stem cells, T-cells and various other cell types. Dr. El-Kalay is responsible for process development and manufacturing of all biotherapeutics for Sangamo.
Third Quarter 2016 Results
For the third quarter ended September 30, 2016, Sangamo reported a consolidated net loss of $19.0 million, or $0.27 per share, compared to a net loss of $9.2 million, or $0.13 per share, for the same period in 2015. As of September 30, 2016, the Company had cash, cash equivalents, marketable securities and interest receivable of $155.4 million.
Revenues for the third quarter of 2016 were $2.8 million, compared to $8.6 million for the same period in 2015. Third quarter 2016 revenues were generated from the Company’s collaboration agreements with Biogen and Shire International GmbH (Shire), enabling technology agreements and research grants. The revenues recognized for the third quarter of 2016 consisted of $2.7 million from collaboration agreements and $0.1 million from research grants, compared to $8.4 million and $0.2 million, respectively, for the same period in 2015. The decrease in collaboration agreement revenues was a result of an amendment to the Company’s collaboration and license agreement with Shire in the third quarter of 2015, which returned the rights to the hemophilia programs to Sangamo, as well as a decrease in revenues from the Biogen agreement as the initial research phase of these programs has matured and activities during this quarter were largely internal.
In the third quarter of 2016, Sangamo recognized $1.2 million of revenues related to research services performed under the collaboration agreement with Biogen, and $0.2 million of revenues related to research services performed under the collaboration agreement with Shire. In addition, Sangamo received upfront payments of $13.0 million and $20.0 million pursuant to the agreements entered into with Shire in 2012 and Biogen in 2014, respectively. The Shire payment is being recognized as revenue on a straight-line basis over the initial six-year research term. Beginning in January 2016, the Biogen payment is being recognized over approximately 42 months which reflects the revised service period related to Sangamo’s deliverables under the Biogen agreement. The Company recognized $0.5 million of the Shire upfront payment and $0.6 million of the Biogen upfront payment as revenue for the third quarter of 2016.
Research and development expenses were $17.0 million for the third quarter of 2016, compared to $16.7 million for the same period in 2015. General and administrative expenses were $5.0 million for the third quarter of 2016, compared to $4.6 millionfor the same period in 2015.
Total operating expenses for the third quarter of 2016 were $22.0 million, compared to $21.3 million for the same period in 2015.
Nine Months Results
For the nine months ended September 30, 2016, the consolidated net loss was $62.0 million, or $0.88 per share, compared to a consolidated net loss of $26.7 million, or $0.38 per share, for the nine months ended September 30, 2015. Revenues were $10.5 million for the nine months ended September 30, 2016, compared to $30.4 million for the same period in 2015. The decrease in revenues was primarily related to the amendment of our collaboration and license agreement with Shire, as well as a decrease in revenues related to our agreements with Sigma and Biogen. Total operating expenses were $73.2 million for the nine months ended September 30, 2016, compared to $61.6 million for the same period in 2015 and reflect increased expenses related to salaries and benefits, including stock-based compensation expense, as well as professional fees, consulting services and other corporate costs.
Financial Guidance for 2016
The Company reiterates guidance as follows:
- Cash and Investments: Sangamo expects that its cash, cash equivalents and marketable securities will be at least $140 million at the end of 2016, inclusive of research funding from existing collaborators 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 $12 million to $17 million in 2016, inclusive of research funding from existing collaborations.
- Operating Expenses: Sangamo expects that operating expenses will be in the range of $85 million to $95 million for 2016. (Original Source)
Shares of Sangamo closed today at $3.75, up $0.10 or 2.74%. SGMO has a 1-year high of $9.91 and a 1-year low of $3.50. The stock’s 50-day moving average is $4.51 and its 200-day moving average is $5.55.
On the ratings front, SGMO has been the subject of a number of recent research reports. In a report issued on October 19, Piper Jaffray analyst Charles Duncan downgraded SGMO to Hold, with a price target of $5.00, which represents a potential upside of 35% from where the stock is currently trading. Separately, on the same day, Jefferies’ Gena Wang reiterated a Buy rating on the stock and has a price target of $14.00.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Charles Duncan and Gena Wang have a total average loss of 10.3% and 31.5% respectively. Duncan has a success rate of 34% and is ranked #3995 out of 4197 analysts, while Wang has a success rate of 20% and is ranked #4064.
Sangamo BioSciences, Inc. is a clinical stage biopharmaceutical company, which is focused on the research, development and commercialization of engineered DNA-binding proteins for therapeutic genome editing and gene regulation technology platform is enabled by the engineering of a class of transcription factors known as zinc finger DNA-binding proteins.