On May 5, 2022 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, reported recent business highlights and first quarter 2022 financial results (Press release, Sangamo Therapeutics, MAY 5, 2022, View Source [SID1234613652]).

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"This quarter, we have continued to progress multiple programs through clinical development and demonstrated, once again, Sangamo’s track record of advancing groundbreaking therapies in genomic medicine," said Sandy Macrae, Chief Executive Officer of Sangamo. "We dosed a total of five patients across three programs, including the first patient in our study for the treatment of kidney transplant rejection, in what we believe was the first in human dosing of an engineered CAR-Treg cell therapy product candidate. We believe this progress positions us well to advance transformational genomic medicines for patients in need and to generate long-term value for our shareholders."
Recent Business Highlights
Fabry disease – Dosed three additional patients, resulting in a total of nine patients dosed to date, thereby completing dose escalation for the Phase 1/2 study; Phase 3 planning progresses.
•We dosed two patients in Cohort 4 in the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, our wholly owned gene therapy product candidate for the treatment of Fabry disease, at a dose level of 5e13 vg/kg.
•In addition, we dosed a third patient in Cohort 3, at the dose level of 3e13 vg/kg.
•In total, we have successfully dosed a total of nine patients across four cohorts to complete the dose escalation portion of the study.
•Enzyme replacement therapy (ERT) withdrawal was completed for a second patient, with no reports to date that the resumption of ERT is required in either patient.
•We expect to provide updated results from the STAAR study in the second half of 2022.
•We continue to actively prepare for the expansion cohorts, as well as a potential pivotal Phase 3 trial.
Sickle cell disease – Dosed fifth patient, the first with a product candidate manufactured using improved methods; Phase 3 planning progresses.
•We dosed the fifth patient in the Phase 1/2 PRECIZN-1 study of SAR445136, a zinc finger nuclease gene-edited cell therapy candidate for the treatment of sickle cell disease, which is under development with Sanofi. This is the first patient in the study to receive a product candidate manufactured using improved methods that have been shown in internal experiments to increase the number of long-term progenitor cells in the final product.
•We plan to dose the remaining patients in this study by the end of the third quarter of 2022.
•We expect to provide updated results from the PRECIZN-1 study in the second half of 2022.
•Phase 3 enabling activities and manufacturing readiness are in progress.
•We continue to collaborate with Sanofi on an orderly transition of Sanofi’s rights and obligations under this program back to Sangamo on June 28.

Hemophilia A – FDA lifted clinical hold; Trial remains voluntarily paused; Pfizer expects to resume trial in Q3 2022.
•Pfizer announced that, in March 2022, the FDA lifted the clinical hold that had been placed on the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A. Pfizer previously paused this trial when some of the patients experienced FVIII activity greater than 150% following treatment.
•Pfizer also announced that the voluntary pause remains in place until all necessary conditions are met, including approval of updated trial protocols by regulatory authorities.
•In addition, Pfizer announced that a patient with elevated FVIII levels reported a below-the-knee deep vein thrombosis. The patient had a history of thrombotic events prior to participation in the trial, which is a known risk factor for subsequent events and an exclusion criterion for participation in the AFFINE trial. The case was assessed to understand all potential contributing factors, including missed doses of investigator-prescribed direct oral anti-coagulants. The patient is reported to be doing well. The information was shared with trial investigators, health authorities and the independent external Data Monitoring Committee and Pfizer responded to queries from health authorities.
•Pfizer announced that it anticipates resuming this trial in the third quarter of 2022, with a pivotal data readout estimated in the second half of 2023.
•Over 50% of the patients have been enrolled in the Phase 3 AFFINE trial.
Renal Transplant Rejection – Believed to be first-ever in human dosing with an engineered CAR-Treg cell therapy candidate.
•We dosed the first patient in our Phase 1/2 STEADFAST study evaluating TX200, our wholly owned autologous CAR-Treg cell therapy treating patients receiving an HLA-A2 mismatched kidney from a living donor.
•The patient continues to do well, and no adverse events related to treatment have been reported.
•Dosing of the second patient is expected around the middle of 2022, based on their transplant schedule.
•We expect to complete dosing of the first cohort, comprised of three patients, by the end of 2022.
American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) – Eight abstracts accepted.
•A total of eight Sangamo abstracts were accepted for presentation at ASGCT (Free ASGCT Whitepaper) on May 16-19, 2022, including pre-clinical updates across our CAR-Treg autoimmune cell therapy platform, innovations in our genome engineering platform and advances in our AAV capsid engineering program.
First Quarter 2022 Financial Results
Consolidated net loss for the first quarter ended March 31, 2022 was $44.0 million, or $0.30 per share, compared to a net loss of $45.9 million, or $0.32 per share, for the same period in 2021.
Revenues
Revenues for the first quarter ended March 31, 2022 were $28.2 million, compared to $26.3 million for the same period in 2021.
The increase of $2.0 million in revenues was primarily attributed to an increase of $1.0 million related to our collaboration agreement with Novartis, an increase of $0.7 million related to our collaboration agreement with Biogen, and an increase of $0.4 million related to our collaboration agreement with Sanofi.

Total operating expenses on a GAAP basis for the first quarter ended March 31, 2022 were $73.5 million, compared to $72.6 million for the same period in 2021. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the first quarter ended March 31, 2022 were $65.8 million, compared to $65.1 million for the same period in 2021.
The increase in total operating expenses on a GAAP basis was primarily driven by our higher preclinical, clinical and lab supply and other R&D expenses along with our increased headcount to support the advancement of our clinical trials and our ongoing collaborations. Manufacturing and overhead costs also increased as we ramp up our internal manufacturing operations.
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities as of March 31, 2022 were $400.3 million, compared to $464.7 million as of December 31, 2021.
Financial Guidance for 2022 Reiterated (initial guidance provided on February 24, 2022)
On a GAAP basis, we continue to expect total operating expenses in the range of approximately $320 million to $350 million in 2022, which includes non-cash stock-based compensation expense.
We continue to expect non-GAAP total operating expenses, excluding estimated non-cash stock-based compensation expense of approximately $40 million, in the range of approximately $280 million to $310 million in 2022.
Conference Call
Sangamo will host a conference call today, May 5, 2022, at 4:30 p.m. Eastern Time, which will be open to the public. The call will also be webcast with live Q&A and can be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.
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 3090098. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 3090098.