Sangamo Therapeutics Announces Q1 2024 Financial Results and Business Highlights

Sangamo Therapeutics, Inc. recently reported significant business updates and financial results for the first quarter of 2024, highlighting progress particularly in their neurology-focused genomic medicine pipeline. The company, known for its innovative therapeutic approaches in genomic medicine, demonstrated its advancements at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT).

One of the key highlights from the ASGCT meeting was the presentation of 20 research projects showcasing the progress of their neurology-focused preclinical pipeline. Notably, the novel delivery capsid, STAC-BBB, was highlighted for its superior blood-brain barrier penetration in non-human primates following intravenous administration. Sangamo also announced the discovery of their next-generation Modular Integrase (MINT) platform, a method designed for large-scale genome editing.

In a strategic move, Sangamo completed dosing in the Phase 1/2 STAAR study of isaralgagene civaparvovec for Fabry disease. The company is in active discussions with potential collaborators for this program, indicating a potential partnership to advance its development. Additionally, Pfizer, a collaborator with Sangamo, anticipates submitting a biologics license application (BLA) and marketing authorization application (MAA) for their Hemophilia A treatment in early 2025, contingent on positive results from a pivotal readout expected in mid-2024.

Financially, Sangamo raised approximately $24 million through a registered direct offering. Despite a net loss of $49.1 million for the first quarter of 2024, the company remains focused on advancing its programs. Revenues for the quarter were significantly lower at $0.5 million compared to $158.0 million in the same period in 2023, mainly due to the termination of collaboration agreements with Biogen and Novartis.

Sangamo's operating expenses also decreased, with total operating expenses on a GAAP basis for the first quarter of 2024 being $52.0 million, compared to $139.8 million for the same period in 2023. This reduction was primarily due to lower compensation costs and a reduction in headcount following a restructuring of operations. Non-GAAP operating expenses, which exclude certain non-cash items, were $43.6 million, down from $69.5 million in the previous year.

Regarding their cash position, Sangamo reported cash and cash equivalents of $54.4 million as of March 31, 2024. This is a decrease from $81.0 million at the end of 2023, but the recent fundraising efforts and cost-saving measures are expected to sustain the company's operations into the third quarter of 2024.

Sangamo’s neurology pipeline includes several key programs in epigenetic regulation and AAV capsid delivery technology. Investigational new drug (IND) enabling toxicology studies are nearing completion for the Nav1.7 program aimed at treating chronic neuropathic pain, with an expected IND submission in late 2024. Additionally, clinical trial authorization (CTA) activities are underway for a program targeting prion disease, leveraging the STAC-BBB capsid, with a CTA submission projected for late 2025. There are also plans to resume development of a zinc finger repressor program addressing tauopathies, including Alzheimer’s disease.

In genome engineering, the MINT platform stands out as a significant advancement, potentially allowing for the integration of large DNA sequences into the genome to treat patients with unique genetic mutations. This platform could be used internally for neurological indications and offers potential opportunities for partnerships in both human disease and agricultural biotech sectors.

In the Fabry disease program, the STAAR study reached a milestone by dosing the final patient, totaling 33 patients. The company is awaiting further collaboration opportunities before planning additional investments for potential registrational trials. Meanwhile, in their Hemophilia A program with Pfizer, a pivotal readout in mid-2024 could lead to significant regulatory submissions and potential milestone payments.

Sangamo's commitment to advancing its genomic medicine pipeline is evident through its strategic business developments and scientific presentations. While facing financial challenges, the company continues to seek partnerships and funding to support its innovative programs and achieve its long-term goals in transforming patient care for serious neurological diseases.