Sangamo Therapeutics Reports Third Quarter 2018 Financial Results
"I'm pleased with our continued execution toward our vision to transform Sangamo into the premier genomic medicines company," said
Recent Highlights
Clinical
- In September, Sangamo presented 16-week clinical results from the first two cohorts of the Phase 1/2 CHAMPIONS Study evaluating SB-913 for Mucopolysaccharidosis Type II (MPS II), including dose-dependent reductions in glycosaminoglycans (GAGs) in the second cohort (1e13 vector genomes per kilogram of body weight, or vg/kg). At 16 weeks, in all subjects, administration of SB-913 was generally well-tolerated, with no treatment-related serious adverse events reported. Most adverse events reported were mild (Grade 1) and resolved without treatment.
- In October, the independent Safety Monitoring Committee (SMC) of the CHAMPIONS Study evaluating SB-913 in patients with MPS II reviewed accumulated safety and efficacy data from all three cohorts and made the following three recommendations:
- 1) proceed to the cohort expansion phase of the clinical trial with the dose used at the third dose cohort (5e13 vg/kg);
- 2) initiate screening and enrollment of adolescent subjects (12 to 17 years of age); and
- 3) initiate the withdrawal of enzyme replacement therapy (ERT) when appropriate.
- In October, the independent SMC of the Phase 1/2 Alta Study evaluating SB-525 for hemophilia A reviewed accumulated safety and efficacy data from six patients enrolled in three dose cohorts. As of that review, SB-525 exhibited dose-dependent efficacy on serum factor levels and was generally well-tolerated with no treatment-related serious adverse events and no use of tapering courses of oral steroids. The SMC recommended that the study continue with escalation to an additional dose. Sangamo plans to present safety and efficacy data from the Alta Study after dose escalation is complete and the clinical trial has progressed to the cohort expansion phase. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer Inc. for the development and commercialization of potential gene therapy programs for hemophilia A.
- A second patient has been treated in Phase 1/2 EMPOWERS Study evaluating SB-318 for MPS I. Per the recommendation of the SB-318 SMC, which in October reviewed accumulated safety and efficacy data from both the EMPOWERS Study and the CHAMPIONS Study evaluating SB-913 for MPS II, this patient received the 5e13 vg/kg dose, the highest dose.
"The independent SMC's recommendations for the CHAMPIONS Study highlight the important progress we've made in this program," said
Dr. Conner continued: "Regarding SB-525, we are pleased with the SMC's recommendation for escalation to an additional dose, which is enabled by the safety profile, the lack of immunogenicity, and the dose-dependent response observed to date across the first three dose cohorts. Our goal is to advance into the pivotal phase of development with the dose that holds the highest potential to meet the needs of patients with hemophilia A. We do not believe that others have yet presented data that would support an optimal product profile for hemophilia A gene therapy."
Corporate
- Sangamo completed the acquisition of a majority of
TxCell S.A. (TxCell ) ordinary shares in October and has since launched a general tender offer on the remaining ordinary shares ofTxCell listed onEuronext inParis .TxCell is a leader in the emerging field of regulatory T cell (Treg) development for immunological diseases, one of Sangamo's stated therapeutic areas of focus for its proprietary product candidate pipeline. Sangamo intends to evaluate the potential of CAR-Treg (Tregs genetically modified with a chimeric antigen receptor, or CAR) therapies to prevent graft rejection in solid organ transplant and for the treatment of autoimmune diseases.
- Stéphane Boissel, former CEO of
TxCell , joined Sangamo as Executive Vice President of Corporate Strategy.
Third Quarter 2018 Financial Results
For the third quarter ended
Revenues for the third quarter ended
Total operating expenses for the third quarter ended
Financial Guidance for 2018
- Cash and Investments: Sangamo expects to report a balance of cash, cash equivalents, marketable securities and interest receivable of at least
$380 million atDecember 31, 2018 . This anticipated cash balance is inclusive of research funding from existing collaborators and recent financings and is expected to last approximately two years. - Operating Expense: Sangamo expects operating expense of
$160 million to $165 million for the year endingDecember 31, 2018 .
Conference Call
Sangamo will host a conference call today,
The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 9494369. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately
About Sangamo
Forward-Looking Statements
This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, the potential for data readouts and updates from multiple clinical trials in coming months, completion of the acquisition of
SELECTED CONSOLIDATED FINANCIAL DATA |
|||||||
(unaudited; in thousands, except per share data) |
|||||||
Statement of Operations Data: |
|||||||
Three months ended |
For the Nine Months Ended |
||||||
September 30, |
September 30, |
||||||
2018 |
2017 |
2018 |
2017 |
||||
Revenues: |
|||||||
Collaboration agreements |
$ 23,538 |
$ 11,759 |
$ 57,378 |
$ 23,042 |
|||
Research grants |
24 |
53 |
237 |
448 |
|||
Total revenues |
23,562 |
11,812 |
57,615 |
23,490 |
|||
Operating expenses: |
|||||||
Research and development |
28,810 |
18,425 |
81,612 |
46,351 |
|||
General and administrative |
10,993 |
6,422 |
32,381 |
19,734 |
|||
Total operating expenses |
39,803 |
24,847 |
113,993 |
66,085 |
|||
Loss from operations |
(16,241) |
(13,035) |
(56,378) |
(42,595) |
|||
Interest and other income, net |
3,398 |
681 |
6,708 |
1,118 |
|||
Net loss |
$ (12,843) |
$ (12,354) |
$ (49,670) |
$ (41,477) |
|||
Basic and diluted net loss per common share |
$ (0.13) |
$ (0.15) |
$ (0.52) |
$ (0.55) |
|||
Shares used in computing basic and diluted net loss per common share |
101,725 |
83,750 |
95,165 |
75,814 |
|||
SELECTED BALANCE SHEET DATA |
|||||||
September 30, 2018 |
December 31, 2017 |
||||||
Cash, cash equivalents, marketable securities and interest receivable |
$ 459,253 |
$ 244,560 |
|||||
Total assets |
606,604 |
286,741 |
|||||
Total stockholders' equity |
379,906 |
187,900 |
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SOURCE
Sangamo Therapeutics, Inc., McDavid Stilwell, (510) 970-6000, x219, mstilwell@sangamo.com or Varant Shirvanian, (510) 970-6000, x205, vshirvanian@sangamo.com