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2/22/2023
Good day, ladies and gentlemen, and thank you for standing by. Welcome to the Sangamo fourth quarter 2022 earnings conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1-1 on your telephone keypad. At this time, I would like to turn the conference over to Ms. Louise Wilkie. Ma'am, please begin.
Thank you. Good afternoon. I'm Louise Wilkie, Sangamo's Vice President of Investor Relations and Corporate Communications. Thank you for joining us on the call today. On this call are several members of the Sangamo Executive Leadership Team, including Sandy McRae, Chief Executive Officer, Mark McClung, Chief Operating Officer, Farshisha Durrababu, Chief Financial Officer, Jason Fontenot, Chief Scientific Officer, Natalie Dubastring-Fellow, Chief Development Officer, Athena Cockcroft, Chief Medical Officer, and Andy Ramamur, Head of Technical Operations. Slides from our corporate presentation can be found at our website, sangamo.com, under the Investors and Media section of the Events and Presentations page. This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements relating to the therapeutic and commercial potential of our product candidates, the anticipated plans and timelines of Sangamo and our collaborators for initiating and conducting clinical trials, screening and dosing patients, and presenting clinical data. Advancement of our product candidates. Advancement of preclinical programs for the clinic. Our investment focus and the sufficiency of our resources. Our 2023 financial guidance, upcoming catalysts and guidance, and other statements that are not historical facts. Actual results may differ materially from what we discussed today. These statements are subject to certain risks and uncertainties that are discussed in our filings of the SEC. The forward-looking statements stated today are made as of this date. and we undertake no duty to update such information except as required by law. On this call, we discuss our non-GAAP operating expenses. Reconciliation of this measure to our GAAP operating expenses can be found in today's press release, which is available on our website. Now, I'd like to turn the call over to our CEO, Sandy McRae.
Thank you, Louise, and good afternoon to everyone joining the call. I'm pleased to share today the significant process we have made over the last year in bringing potentially transformative medicines closer to patients that need them most and to convey our outlook for the coming 12 months. 2022 was a year of important clinical and non-clinical milestones for Sangamo. We believe we became the first company to do as a human with an engineered CAR T reg. We established a leadership position in Fabry disease. We presented promising data in sickle cell disease, And with Pfizer, we announced the resumption of our Phase 3 Haemophilia A trial. I'm thrilled with the updated Fabry data from our Phase 1-2 STAR study that was released today at the 19th Annual World Symposium. In addition to sharing updated biomarker data, we revealed the first kidney biopsy data from the study, along with exciting SF36 general health score findings, which, when taken together, bring to point the potential for ST920 to become a truly compelling Fabry therapy. Based on what we've seen in the competitive landscape, we truly believe we have potential best in class leading gene therapy for this important indication. Natalie will take us through the details of this new data shortly, but I wanted to express how happy I am with the progress of the program, which I believe has a strong potential to bring relief to those suffering the debilitating aspects of Fabry disease. We are well underway in dosing the first full cohort of patients with TX200 in our Phase 1-2 Steadfast Study in HLA-A2 mismatched kidney transplant, and we believe we remain in the lead in clinical progress amongst CAR-T red companies. This study potentially paves the way for current projects in development as a proof of concept and demonstration of safety of CAR T regs in humans. Our focus is advancing this trial with urgency while maintaining patient safety. So the team is actively working with the regulatory authorities to explore options to accelerate the dose escalation protocol. We look forward to sharing more information when available. The team has also made significant headway in advancing our second wave of preclinical programs, progressing our CAR-T reg follow-on indications in multiple sclerosis and inflammatory bowel disease, advancing our four wholly owned CNS epigenetic regulation programs, breaking new ground in delivery through our AAV capsid engineering program, and continuing to advance our zinc finger genomic engineering platform. I'm proud of the progress we've made this year, which is a testament to the hard work and dedication of our talented team members across the organisation. During 2022, we remain dedicated to advancing our pipeline and executing on our corporate strategy. In this regard, we're happy to add two new board members to our board of directors. Heckhorn brings with it over 30 years of leadership experience in the biotechnology industry, having led many important strategic and financing transactions. Courtney Beers has also joined us as bring to Sangamo her long experience in drug development and scientific strategy. Looking to 2023, the leadership team is fully focused on progressing our clinical programs and continuing the transition to Sangamo's second wave pipeline, carefully prioritizing our efforts and our spend on cell therapy with our CAR T reg franchise and on epigenetic regulation in the CNS through both our wholly owned and partnered programs. We believe these are programs that both leverage Sangamo's core sync finger capabilities and deep experience in genomic medicines and have the potential to be differentiated while positively impacting patients with high unmet medical need at scale. As we navigate these current market conditions, we fully understand the importance of using capital wisely. And I want to reiterate the focus and prudence with which we are making decisions, both in terms of capital spending and also implementing thoughtful fundraising strategies with long term shareholder benefit in mind. In this respect, we're pleased to present competitive data from our Phase 1-2 precision study in sickle cell disease at the American Society of Haematology annual meeting in December last year. We've also since made additional manufacturing improvements that we believe could further strengthen potential phase three data and reduce manufacturing costs, so important in the syndication. However, in today's environment, we must make difficult choices. Today we're announcing the strategic decision to halt further material investment in phase three planning for our BIVV003 sickle cell disease program. Importantly, Very importantly, we did not make this decision because of efficacy results or due to any safety concerns. We have simply decided to redeploy our resources away from the asset and towards investment in a potential Phase 3 trial for our Fabry programme and efforts to accelerate our Phase 1-2 TX200 programme. As we stated when the programme unexpectedly transitioned back to us last year, we remain committed to the patients and investigators involved in the Phase 1-2 Precision 1 study and expect to complete the study using funds we have already committed. But we will make no material investments in the programme going forward. We're commencing an active search for a partner who can progress this promising asset to a potential Phase 3 clinical trial. I'd now like to turn the call over to our head of development, Natalie, who will discuss the data from our clinical programs in more detail. Natalie.
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