Presented detailed clinical data from registrational STAAR study showing potential for isaralgagene civaparvovec as a one-time, durable treatment of underlying pathology of Fabry disease to provide meaningful, multi-organ, clinical benefits above current standards of care.
Patient recruitment and enrollment in progress for Phase 1/2 STAND study in chronic neuropathic pain following activation of two clinical sites. Expect to dose first patient in the coming months.
Held productive interaction with Medicines and Healthcare products Regulatory Agency (MHRA) to discuss prion disease study ahead of anticipated Clinical Trial Application (CTA) submission.
RICHMOND, Calif., Nov. 06, 2025 (GLOBE NEWSWIRE) -- Sangamo Therapeutics, Inc. (NASDAQ:SGMO), a genomic medicine company, today reported recent business highlights and third quarter 2025 financial results.
"We continued to advance our clinical and pre-clinical pipeline this quarter and are excited to now be recruiting and enrolling patients in our first ever neurology clinical study following the activation of the first two clinical sites in the Phase 1/2 STAND study in chronic neuropathic pain," said Sandy Macrae, Chief Executive Officer of Sangamo Therapeutics. "Furthermore, the announcement of detailed clinical data from our registrational STAAR study in Fabry disease, alongside our recent FDA meeting, marked important steps forward on the path to an anticipated regulatory submission for this program."
Recent Business Highlights
Corporate Updates
Received $6 million from Pfizer Inc. (Pfizer) upon Pfizer's exercise of a buyout option for a license to use certain zinc finger modified cell lines, pursuant to a 2008 license agreement between Pfizer and Sangamo.
Granted a 180-day extension by Nasdaq (until April 27, 2026) to regain compliance with the $1.00 minimum bid price requirement of the listing standards for The Nasdaq Capital Market.
Fabry Disease
In September, presented detailed clinical data from the registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease, at the International Congress of Inborn Errors of Metabolism 2025 (ICIEM2025) in Kyoto, Japan.
A positive mean annualized eGFR slope of 1.965 mL/min/1.73m2/year (95% confidence interval (CI): -0.153, 4.083) at 52-weeks was observed across all 32 dosed patients.
Furthermore, a mean annualized eGFR slope at Week 104 of 1.747 mL/min/1.73m2/year (95% CI: -0.106, 3.601) was observed for the 19 patients who had achieved 104-weeks of follow-up.
Supportive mean annualized eGFR slopes were also observed across a variety of patient subgroups, including gender, baseline Enzyme Replacement Therapy (ERT) status, Fabry disease type and baseline eGFR, showing consistency in effect across Fabry patients in the study.
Stable cardiac function was observed, including left ventricular mass (LVM), left ventricular mass index (LVMI), left ventricular myocardial global longitudinal strain (GLS), T1 and T2 mapping, end-diastolic and end-systolic volumes that remained stable over at least one year.
Durability of effect was demonstrated in the study, with elevated expression of alpha-galactosidase A (α-Gal A) activity maintained for up to 4.5 years for the longest treated patient.
All 18 patients who began the study on ERT had been withdrawn from ERT and remained off ERT as of the data cutoff date1. Plasma lyso-Gb3 levels in these patients remained generally stable following ERT withdrawal.
Of the 10 patients who had measurable titers of total antibodies (TAb) or neutralizing antibodies (Nab) against α-Gal A associated with ERT at baseline, TAb or NAb titers decreased markedly in nine patients and became undetectable in eight following treatment.
Improvements in disease severity were reported in the Fabry Outcome Survey adaptation of the Mainz Severity Score Index (FOS-MSSI) age-adjusted score, with 22 patients showing improvements in their total MSSI score at 12 months and nine patients improving their FOS-MSSI disease category at the last assessment.
Statistically and clinically significant improvements in the short form-36 (SF-36) quality of life scores were also observed, alongside statistically significant improvements in the gastrointestinal symptom rating scale (GSRS) compared to baseline.
Isaralgagene civaparvovec demonstrated a favorable safety and tolerability profile in the study, without the requirement for preconditioning.
In October, held a meeting with the FDA to discuss the proposed efficacy and safety data package where, in the meeting minutes, among other things, the FDA reiterated its October 2024 agreement to use eGFR slope as an endpoint to support an accelerated approval pathway.
In October, attended and presented at the 15th Annual Fabry Family Education Conference, bringing together over 200 Fabry patients, family members and volunteers to provide educational presentations and gather insights from Fabry patients.
Sangamo is preparing for an anticipated Biologics License Agreement (BLA) submission as early as the first quarter of 2026, while continuing business development discussions for a Fabry commercialization agreement.
Core Neurology Pipeline
Chronic Neuropathic Pain, ST-503
Patient enrollment and recruitment have now commenced following the activation of the first two clinical sites for the Phase 1/2 STAND study evaluating ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to small fiber neuropathy (SFN), a type of chronic neuropathic pain.
In September, presented updated nonclinical data at the 9th International Congress on Neuropathic Pain in Berlin, Germany, which demonstrated the durability, potency and selectivity of ST-503 in nonhuman primates (NHPs), alongside a favorable safety profile.
Expect to dose the first STAND study patient in the coming months.
Prion Disease, ST-506
CTA-enabling activities continue to advance for ST-506, an investigational epigenetic regulator for the treatment of prion disease, leveraging STAC-BBB, Sangamo's novel proprietary neurotropic adeno-associated virus (AAV) capsid.
Held productive interaction with the MHRA, including alignment on Chemistry, Manufacturing and Controls (CMC) strategy.
In November, presented updated preclinical data at the Prion 2025 Conference, in Rio de Janeiro, Brazil, which demonstrated a profound survival extension observed in disease mouse models and widespread brain delivery and prion reduction in NHPs treated with ST-506.
A CTA submission for ST-506 is expected as early as mid-2026.
Third Quarter 2025 Financial Results
Consolidated net loss for the third quarter ended September 30, 2025 was $34.9 million, or $0.11 per share, compared to net income of $10.7 million, or $0.04 per share on a fully diluted basis, for the same period in 2024.
Revenues
Revenues for the third quarter ended September 30, 2025 were $0.6 million, compared to $49.4 million for the same period in 2024.
The decrease of $48.8 million in revenues for the three months ended September 30, 2025, compared to the same period in 2024, was primarily attributable to revenue relating to our collaboration agreement with Genentech recorded in the third quarter of 2024.
GAAP and Non-GAAP Operating Expenses
(In millions)
Three Months Ended
Nine Months Ended
September 30,
September 30,
2025
2024
2025
2024
Research and development
$
28.1
$
27.7
$
81.2
$
87.8
General and administrative
8.0
11.1
27.1
34.9
Impairment of long-lived assets
-
-
-
5.5
Total operating expenses
36.1
38.8
108.3
128.2
Impairment of long-lived assets
-
-
-
(5.5
)
Depreciation and amortization
(1.0
)
(1.3
)
(3.0
)
(3.9
)
Stock-based compensation
(2.1
)
(3.3
)
(6.9
)
(9.1
)
Non-GAAP operating expenses
$
33.0
$
34.2
