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Edited Transcript of SGMO.OQ earnings conference call or presentation 4-Nov-20 10:00pm GMT

·34 min read

Q3 2020 Sangamo Therapeutics Inc Earnings Call RICHMOND Nov 11, 2020 (Thomson StreetEvents) -- Edited Transcript of Sangamo Therapeutics Inc earnings conference call or presentation Wednesday, November 4, 2020 at 10:00:00pm GMT TEXT version of Transcript ================================================================================ Corporate Participants ================================================================================ * Alexander D. Macrae Sangamo Therapeutics, Inc. - CEO, President & Director * Aron Feingold Sangamo Therapeutics, Inc. - Head of Corporate Communications & IR Officer * Bettina M. Cockroft Sangamo Therapeutics, Inc. - Senior VP & Chief Medical Officer * D. Mark McClung Sangamo Therapeutics, Inc. - Executive VP & Chief Business Officer * Jason Fontenot Sangamo Therapeutics, Inc. - SVP of Cell Therapy & Interim Head of Research * Sung H. Lee Sangamo Therapeutics, Inc. - Executive VP & CFO ================================================================================ Conference Call Participants ================================================================================ * Boran Wang Guggenheim Securities, LLC, Research Division - Associate * Eric William Joseph JPMorgan Chase & Co, Research Division - VP & Senior Analyst * Geoffrey Christopher Meacham BofA Merrill Lynch, Research Division - Research Analyst * Huidong Wang Barclays Bank PLC, Research Division - Research Analyst * Lyla Youssef Cowen and Company, LLC, Research Division - Research Associate * Maurice Thomas Raycroft Jefferies LLC, Research Division - Equity Analyst * Nicole Germino Truist Securities, Inc., Research Division - Associate * Yanan Zhu Wells Fargo Securities, LLC, Research Division - Associate Analyst ================================================================================ Presentation -------------------------------------------------------------------------------- Operator [1] -------------------------------------------------------------------------------- Ladies and gentlemen, thank you for standing by, and welcome to Sangamo Therapeutics Q3 2020 webcast. (Operator Instructions) Please be advised that today's conference may be recorded. (Operator Instructions) I would now like to hand the conference over to your host, Head of Corporate Communications, Aron Feingold. Madam, please go ahead. -------------------------------------------------------------------------------- Aron Feingold, Sangamo Therapeutics, Inc. - Head of Corporate Communications & IR Officer [2] -------------------------------------------------------------------------------- Good afternoon, and thank you for joining us today. With me this afternoon on this call are several members of the Sangamo executive leadership team, including Sandy Macrae, Chief Executive Officer; Sung Lee, Chief Financial Officer; Mark McClung, Chief Business Officer; Jason Fontenot, Interim Head of Research; and Bettina Cockroft, Chief Medical Officer. Slides from our corporate presentation can be found at our website, sangamo.com, under the Investors and Media section in the Events and Presentations page. This call includes forward-looking statements regarding Sangamo's current expectation. These statements include, but are not limited to, statements relating to our R&D pipeline; our ability to develop, obtain regulatory approvals for and commercialize therapies to treat certain diseases and the timing, availability and cost of such therapies; plans and time lines for Sangamo and our collaborators to conduct clinical trials and share clinical data and the potential for these data to demonstrate clinical benefits to patients; the potential to use certain technologies to develop our therapies; our collaboration strategy and the potential to earn milestone payments and royalties from our collaborations and the timing of receiving such payments and royalties; plans and time lines for building and opening manufacturing facilities; the effects of the evolving COVID-19 pandemic; our expectations regarding our financial performance and resources; and other statements that are not historical facts. Actual results may differ substantially from what we discuss today. In addition, these statements are not guarantees of future performance and are subject to certain risks and uncertainties that are discussed in documents that we file with the Securities and Exchange Commission, specifically in our quarterly report on Form 10-Q for the quarter ended September 30, 2020. The forward-looking statements stated today are made as of this date, and we undertake no duty to update such information, except as required under applicable law. On this call, we discuss a non-GAAP financial measure. We believe this measure is helpful in understanding our past financial performance and our potential future results. This is not meant to be considered in isolation or as a substitute for the comparable GAAP measure. The comparable GAAP measure and reconciliation of GAAP to the non-GAAP measures discussed on this call are included in today's press release, which is available on our website. Now I'd like to turn the call over to our CEO, Sandy Macrae. -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [3] -------------------------------------------------------------------------------- Thank you, Aron, and good afternoon to everyone on the call. This quarter, we advanced our R&D activities as we continue to adapt to the conditions brought on by the evolving COVID-19 pandemic. We are moving forward on clinical execution, and we are optimistic with our plans to continue to dose patients and initiate new trials. We also completed our research activities associated with our ALS collaboration with Pfizer and are continuing to move our research projects with Biogen and Novartis forward and are progressing our work with our other partners. Pfizer has dosed the first participant in the Phase III AFFINE study of giroctocogene fitelparvovec or SB-525, our investigational gene therapy for hemophilia A patients. This event triggered a $30 million milestone achievement for Sangamo, which we expect to receive in the current quarter, further strengthening our cash position. Pfizer previously communicated that they expect a pivotal data readout from the AFFINE study in 2022. During the mid-September Investor Day, Pfizer provided an update from the Phase I/II ALTA study, showing encouraging data regarding tolerability, clinically meaningful factor levels, bleeding rates and factor use in the highest dose cohort up to 85 weeks in the longest treated patients. Pfizer and Sangamo believe that these data support a potentially differentiated hemophilia A gene therapy product candidate. In August and September, in close collaboration with principal investigators monitoring safe conditions for patients within the context of COVID-19, Sangamo dosed the first 2 patients in the Phase I/II STAAR study evaluating ST-920 gene therapy in Fabry disease. Dosing of the first cohort of this study is now complete, and enrollment is ongoing for cohort 2. We expect to share data on this study by the end of next year. During the quarter, we received additional regulatory approvals for the first-in-human Phase I/II clinical study evaluating CAR regulatory T cell or CAR-T reg candidate, TX200, in kidney transplantation. We believe we are on track to initiate the study next year. Initiating this study may allow us to be the first company to explore the potential of CAR-Treg cells in humans. We are hopeful that this will provide broader proof of concept for genetically engineered cell therapy using Tregs. Beyond transplantation, we intend to further evaluate CAR-Tregs, including zinc finger nucleus edited allogeneic Treg therapies in autoimmune diseases with high unmet medical need. Also this quarter, we completed our research activities associated with our ongoing Pfizer collaboration to develop gene regulation therapy using our zinc finger technology for the treatment of C9ORF72-related ALS. In this program, our zinc finger proteins are designed to selectively target disease allele repeats, a remarkable demonstration of yet another way our versatile technology may be able to have a disease-modifying impact on challenging CNS diseases. We recently earned a $5 million milestone payment from Pfizer associated with this program, which we expect to receive later this quarter. It is a further testament to our R&D momentum. We look forward to continuing to work closely with Pfizer to support their research and development in this program. With that, I will turn the call over to our Chief Medical Officer, Bettina, who will provide additional details on our clinical accomplishments. -------------------------------------------------------------------------------- Bettina M. Cockroft, Sangamo Therapeutics, Inc. - Senior VP & Chief Medical Officer [4] -------------------------------------------------------------------------------- Good afternoon. As Sandy mentioned, our clinical operations have adapted to the challenges of the evolving COVID-19 pandemic, and we are pleased with our progress in executing on our partnered and wholly owned programs. Pfizer dosed the first patient in the AFFINE study of giroctocogene fitelparvovec or SB-525, our first acid in a registrational trial. AFFINE is a global Phase III, open-label, multicenter, single-arm study, evaluating the efficacy and safety of SB-525 in patients with moderately severe to severe hemophilia A. The primary endpoint is annualized bleeding rate, or ABR, through 12 months following treatment. This will be compared to ABR while in Factor VIII replacement therapy collected in the Phase III lead-in study, which will provide a baseline for Phase III study participants. The secondary endpoints include Factor VIII activity level after the onset of steady state over 12 months. Participants will be analyzed throughout the 5-year study period following the single infusion to further assess durability of efficacy and safety. Pfizer shared updated Phase I/II data at a Pfizer investor event in September, which demonstrated that SB-525 was generally well tolerated. Each of the 5 patients in the high-dose cohorts sustained a clinically meaningful level of Factor VIII activity without bleeds or the need for prophylactic factor up to 85 weeks for the longest treated patient. Both companies are encouraged by these results and plan to present further follow-up data from the ALTA study in the next few months when all 5 patients in the 3e13 vector genomes per kilogram dose cohort have been followed for at least 1 year. We have dosed the first 2 patients comprising the first cohort in the Phase I/II STAAR study evaluating ST-920 in Fabry disease. The goal of this gene therapy candidate is to provide a predictable and durable expression of the alpha-Gal A enzyme, which is deficient in Fabry disease due to mutations in the GLA gene, resulting in the accumulation of the substrate Gb3 and its soluble derivative lyso-Gb3. This can cause challenging symptoms and morbidities, including impaired renal and cardiac function, pain and gastrointestinal symptoms. The STAAR trial is a multicenter, open-label, dose-ranging study, evaluating the safety and tolerability of ST-920 in classical Fabry patients 18 years and older. Study participants will receive a single intravenous infusion of ST-920, followed by 1 year of observation and monitoring of clinical endpoints, such as alpha-Gal A activity and assessment of Gb3 and lyso-Gb3 levels. A long-term follow-up study will allow patients to be monitored for an additional 4 years. Enrollment for the second cohort is ongoing. We expect that data will be shared towards the end of 2021 after we have identified a dose cohort -- a full cohort expansion. We believe that ST-920 offers a potentially differentiated treatment for Fabry disease with the potential to deliver efficacy with preserved renal function and reduced cardiac morbidity and neuropathy. Preclinical studies evaluating ST-920 demonstrated strong expression of alpha-Gal A and Gb3 substrate reduction across tissue types. As a liver-directed gene therapy, ST-920 is delivered via a onetime intravenous infusion that does not require any preconditioning regimen for patients. We are also working closely with our oncology collaborator Kite, a Gilead company, as it advances Kite-037, an allogeneic anti-CD19 CAR-T therapy, into a clinical trial. Kite expects to submit an investigational new drug application by the end of 2020 and to initiate a clinical trial evaluating Kite-037 in 2021. Throughout the third quarter, we have continued to receive additional regulatory approvals that support the Phase I/II STEADFAST clinical study evaluating the first-in-human CAR-Treg cell therapy, TX200, in HLA-A2 mismatched renal transplantation. We expect to initiate the study next year. The goal for the study is the prevention of transplant rejection through the engineering of Tregs to express an HLA-A2 chimeric antigen receptor, or CAR, allowing them to localize to the renal graft and activate upon recognition of the HLA-A2 antigen. The CAR-Tregs may prevent immune-mediated rejection through the inhibition and modulation of inflammatory immune cells and the release of anti-inflammatory cytokines to induce a tolerogenic environment within the graft. Preclinical data supporting the STEADFAST study presented last month showed that the TX200 HLA-A2 CAR-Tregs efficiently prevented rejection in both graft-versus-host disease and skin transplantation model. They were also shown to be safe and well tolerated in our in vivo studies. Similar to other genetically engineered cell therapy approaches, patients will undergo a leukapheresis procedure from which their Treg cells will be isolated and engineered, then cryopreserved. The HLA-A2 negative patient will subsequently undergo transplantation surgery and following a recovery period, will receive the personalized TX200 drug candidate. As a result of this detailed process, we expect dosing of patients will occur several months after their enrollment. Recent publications show that the regulatory cell therapy space is gaining momentum and excitement in the scientific community. In particular, The ONE Study, a large international clinical study gathering 7 investigator-led trials across 5 countries, showed that immune regulatory cell therapy as a whole were safe. And that immune cell therapy is a potentially useful therapeutic approach in renal transplant recipients, allowing immune cell composition restoration to normal healthy levels and of minimization of the burden of general immunosuppression. This is very promising and supports our plan to evaluate CAR-Tregs in renal transplant patient populations. I will now turn the call over to Sung for an overview of the financial results. Sung? -------------------------------------------------------------------------------- Sung H. Lee, Sangamo Therapeutics, Inc. - Executive VP & CFO [5] -------------------------------------------------------------------------------- Thank you, Bettina, and good afternoon, everyone. We're pleased to share our financial results for the third quarter of 2020. We reported a net loss of $1.6 million or $0.01 per share compared to a net loss of $27.3 million or $0.24 per share for the same period in 2019. Total revenues were $57.8 million compared to $22 million for the same period in 2019. The increase was primarily attributable to a $30 million milestone achieved for SB-525, our hemophilia A candidate partnered with Pfizer, and a $5 million milestone achieved for our C9ORF72 collaboration with Pfizer. Turning to expenses. Non-GAAP operating expenses, which excludes stock-based compensation expense, were $54.8 million compared to $46.5 million for the same period in 2019. The increase in operating expense reflects our headcount growth and facilities expansion to support the advancement of our therapeutic pipeline and manufacturing capabilities. These increases were partially offset by a decrease in clinical and manufacturing supply expenses. Moving to the balance sheet. We ended the quarter with $695 million in cash, cash equivalents and marketable securities. This balance includes the $75 million upfront license fee payment received from Novartis. Additionally, in the current quarter, we expect to receive the $35 million of milestone payments from Pfizer mentioned earlier. We believe our balance sheet remains strong and will allow us to reach several important R&D milestones, including the potential filing of the BLA for SB-525 for hemophilia A. Turning to 2020 full year guidance. We are updating our financial guidance for non-GAAP operating expenses, which exclude estimated stock compensation expense of $25 million from an estimated range of $210 million to $225 million to now be in the estimated range of $210 million to $220 million. I'll now turn it back to Sandy for closing remarks. -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [6] -------------------------------------------------------------------------------- Thank you, Sung. We are focused on clinical execution and building momentum as we adapt to the conditions of COVID-19 and head towards the end of the year. We are pleased with our progress in clinical operations and with our partner programs. Our strong balance sheet enables us to advance our R&D pipeline. We believe these accomplishments have put Sangamo in a strong position to achieve several important milestones and catalysts heading into next year. We believe we remain on track for our AAV manufacturing facility in Brisbane to be operational at the end of this year and for our cell therapy facilities in Brisbane and Valbonne to be operational by year-end 2021. We anticipate continued enrollment in Pfizer's Phase III AFFINE study with a pivotal data readout expected by Pfizer in 2022 and also expect 1-year and 2-year Phase I/II data presentations over the next 1.5 years. We expect continued enrollment in the Phase I/II STAAR study and the data readout towards the end of 2021. Sanofi has guided that the first data readout from the Phase I/II sickle cell disease study is expected next year. We anticipate presenting follow-up ST-400 beta-thalassemia data at the same time. And lastly, we expect the clinical trial initiation of the Phase I/II first-in-human CAR-Treg STEADFAST study will occur in 2021. And Kite expects that the study of allogeneic anti-CD19 CAR-T product candidate, Kite-037, will also commence in 2021. We look forward to delivering on these milestones in the coming year. Operator, please open the line for questions. ================================================================================ Questions and Answers -------------------------------------------------------------------------------- Operator [1] -------------------------------------------------------------------------------- (Operator Instructions) Our first question comes from the line of Geoff Meacham with Bank of America. -------------------------------------------------------------------------------- Geoffrey Christopher Meacham, BofA Merrill Lynch, Research Division - Research Analyst [2] -------------------------------------------------------------------------------- Congrats on all the pipeline progress. I had a couple. The first one is on the hemophilia A study. Just with the 12-month annualized bleed rate endpoint. I wanted to kind of get your feedback on what the FDA is looking for just relative to the feedback that BioMarin received. I know that's obviously a subject of a lot of investor conversations of late. And then the second question is just a broader one on the strategy for the CAR-Treg. Just wanted to -- maybe give us a little bit more context for how you see that differentiating and maybe what successes you see, what the best probability of success you see in, for example, solid tumors versus the liquid tumor side? -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [3] -------------------------------------------------------------------------------- Yes. Thank you for your questions. On the first one around hemophilia A, we are limited in what we can say because this is now in Pfizer's hands. We are so pleased with their progress into Phase III, with their enthusiasm for the program. And that's all the way up in their organization right up to their CEO, how valuable they see this asset for them. You can be assured that they will be having regular conversations with the regulatory authorities. And I'm certain that Pfizer will know how to navigate that landscape. As regards to Tregs, I'm going to pass over to Jason, who is a real expert in this area. And Jason, can you talk to us about how you see our Treg strategy? -------------------------------------------------------------------------------- Jason Fontenot, Sangamo Therapeutics, Inc. - SVP of Cell Therapy & Interim Head of Research [4] -------------------------------------------------------------------------------- Thanks, Sandy, and thanks for the question. So first, I'll start off by pointing out that our programs in regulatory T cells, engineered regulatory T cells, are directed in the autoimmune and inflammatory disease space. These aren't drugs for cancer. So we're really excited about our engineered regulatory T cell platform and the programs that we're bringing forward. We're excited about the progress in regulatory approvals that we've received so far. And about our first-in-human CAR-Treg study, TX200, we're leaders in this field. And we are developing and refining our understanding of Treg biology and rapidly advancing our ability to engineer and manufacture these cells. And our TX200 study will be the first-in-human test of engineered regulatory T cells of CAR-Tregs, and this will be further demonstration of our leadership. The goal with that study is the prevention of renal transplant rejection in the setting of an MHC mismatch transplant. So this is a setting where an HLA-A2 negative patient will receive an HLA-A2 positive kidney transplant. And our therapy is comprised of Tregs that are engineered to recognize the A2 antigen through a chimeric antigen receptor. And that CAR, that will drive the accumulation and activation of the Tregs in the renal graft and suppress the rejection of the graft by the patient's immune system. And what's important about that study is that, as I mentioned, this is going to be the first test of this therapeutic hypothesis around CAR-Tregs. And the study will be important for us to understand the safety and the efficacy and the therapeutic potential of CAR-Tregs and will be informing programs that we're actively pursuing in larger autoimmune and inflammatory indications, such as multiple sclerosis and Crohn's disease. -------------------------------------------------------------------------------- Operator [5] -------------------------------------------------------------------------------- Our next question comes from the line of Maury Raycroft with Jefferies. -------------------------------------------------------------------------------- Maurice Thomas Raycroft, Jefferies LLC, Research Division - Equity Analyst [6] -------------------------------------------------------------------------------- I had one on the Treg program as well. So I guess for getting the -- getting that study started, can you talk more about what factors have led to pushing the study start to 2021? Is it due to COVID or the autologous cell manufacturing process or anything else that you can comment on? And can you talk more about what else needs to be completed before starting the study? -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [7] -------------------------------------------------------------------------------- Maury, thank you very much for your question. We're very pleased with the progress of the Treg for HLA-A2 mismatch. We're very pleased with the approvals from the regulatory authorities. We -- COVID is everywhere. COVID is in -- is impacting the hospitals. We would go to the laboratories, the manufacturing. So there's a general COVID impact, but we are confident that we'll be able to move forward with this program. -------------------------------------------------------------------------------- Maurice Thomas Raycroft, Jefferies LLC, Research Division - Equity Analyst [8] -------------------------------------------------------------------------------- Got it. And from a manufacturing and process development standpoint, is that all figured out? Or is there any other perspective you can provide on that? -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [9] -------------------------------------------------------------------------------- One of the reasons that we acquired TxCell in 2018 was their understanding of how to look after Tregs, which is different from how people look after T cells. And so we're very pleased with the progress that they've made. -------------------------------------------------------------------------------- Maurice Thomas Raycroft, Jefferies LLC, Research Division - Equity Analyst [10] -------------------------------------------------------------------------------- Got it. Okay. And then the other question I had was just on the Prion program that you guys have, which is sort of under the radar. I'm just wondering if you can provide a status update on that program. Any time line update on that program? And then maybe talk about the strategic importance of that one as well. -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [11] -------------------------------------------------------------------------------- Jason, can you maybe talk about Prions and what we think of them? -------------------------------------------------------------------------------- Jason Fontenot, Sangamo Therapeutics, Inc. - SVP of Cell Therapy & Interim Head of Research [12] -------------------------------------------------------------------------------- Sure, Sandy. Thank you. The Prion program is in preclinical development. So I think we'll be looking forward to sharing updates at the appropriate time. I think that there is a great opportunity there to demonstrate the power of our platform, similar to the approaches that we're taking with our partners in CNS, both Biogen and Novartis. These are -- both of these partnerships are driven by what our partners see in our platform. And the Prion program is yet another example of that, and we'll be excited to talk about it as we move forward. -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [13] -------------------------------------------------------------------------------- And we've done some of the initial work with the Broad Institute in Boston. And they have a real deep -- the group we're working with has a real deep expertise in this, and so we have that biological expertise to match with their technological expertise. -------------------------------------------------------------------------------- Operator [14] -------------------------------------------------------------------------------- Our next question comes from Jim Birchenough with Wells Fargo. -------------------------------------------------------------------------------- Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [15] -------------------------------------------------------------------------------- This is Yanan on for Jim. So perhaps a question on the Treg program as well. Could you confirm whether this is a gene-edited product? Because I think it's autologous. So obviously, the HLA-A2 is put in with a gene addition approach. So if you can comment on whether it's a gene-edited product. And then your future products, the allogeneic regulatory CAR-T cells, how do you see the issue of persistence? I guess for this program, TX200 as well, how do you see the issue of persistence? And for renal transplant, would you require long-term persistence? And would you explore repeat dosing for your allogeneic programs? -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [16] -------------------------------------------------------------------------------- So thank you for your questions. They're very sensible scientific questions. And let me try and lay out the path that we've chosen, which is to start with autologous, so we can understand the effectiveness of Tregs. And one of the advantages of renal transplant is that the transplanted kidney can be biopsied because it's implanted close to the surface. So we can look at things like persistence, which, as you would see, is an important feature that would be required of any good treatment. We will gradually switch to allogeneic. And we can either develop allogeneic Tregs by editing down from healthy donor volunteers or by studying iPSCs and other forms of stem cells to develop them up to be Tregs. So this is really at the very cutting edge of regulatory cellular science, and we are lucky to have as many options as possible to take us into those areas. -------------------------------------------------------------------------------- Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [17] -------------------------------------------------------------------------------- Got it. And a quick follow-up. In TX200, is there a gene-editing component? -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [18] -------------------------------------------------------------------------------- TX200 is an autologous form, and therefore, doesn't have a gene-editing component. Jason, am I correct on that? -------------------------------------------------------------------------------- Jason Fontenot, Sangamo Therapeutics, Inc. - SVP of Cell Therapy & Interim Head of Research [19] -------------------------------------------------------------------------------- Yes. That's correct. TX200, we introduced the CAR with a lentivirus for that first program. But obviously, our platform -- one of the assets we have at Sangamo is our platform's ability to do genomic engineering on the Tregs for our future products. -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [20] -------------------------------------------------------------------------------- And that was why the marriage of Sangamo and TxCell was so sensible. They brought the Treg experience, and we brought editing that they needed to take the platform forward. -------------------------------------------------------------------------------- Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [21] -------------------------------------------------------------------------------- Got it. Then maybe a question on zinc finger transcription factors. Just wondering about the origin of the transcription factor, whether it is fully human or if there are any synthetic components in those transcription factors. And how do you think -- how should we think about immunogenicity? -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [22] -------------------------------------------------------------------------------- Jason, do you want to have a go on that, please? -------------------------------------------------------------------------------- Jason Fontenot, Sangamo Therapeutics, Inc. - SVP of Cell Therapy & Interim Head of Research [23] -------------------------------------------------------------------------------- Sure. So the base components of the zinc finger transcription factors are all human. Obviously, in order to direct those transcription factors to a desired sequence, we have to design a synthetic protein that is specific for a specific sequence in the genome. And by nature of the fact that they are fully human, we expect that the immunogenicity should be inherently lower. And we haven't made any observation to date to suggest that immunogenicity could be a problem. -------------------------------------------------------------------------------- Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [24] -------------------------------------------------------------------------------- Great. Congrats on the progress. -------------------------------------------------------------------------------- Operator [25] -------------------------------------------------------------------------------- Our next question comes from the line of Gena Wang with Barclays. -------------------------------------------------------------------------------- Huidong Wang, Barclays Bank PLC, Research Division - Research Analyst [26] -------------------------------------------------------------------------------- I have 3 questions. Well, first is regarding hemophilia A. Just wondering that -- certainly, I know this is already tech transfer to Pfizer. Given the BioMarin experience, Phase I/II data did not -- Phase III data did not -- had quite difference from the Phase I/II, so any thoughts you can share with us regarding your Phase I/II data? And regarding also the Phase III data, one hypothesis was the manufacturing part. And since this manufacturing in Phase I/II is from your side and now will transfer to the Pfizer, any thoughts you can help us understand in terms of potential predictability from Phase I/II data to Phase III? And then the second question is regarding Pfizer partner program, the ALS, zinc finger protein transcription gene regulation program. So wondering if you can share any color regarding the efficiency from this initial R&D study. And the last question is regarding the Fabry program. You already enrolled second cohort. And what would be the determination that you think you'll reach the optimal cohort that you can extend the cohort regarding, say, biomarker data like a plasma lyso-Gb3? As a reference, AVRO showed 30% to 40% further reduction versus baseline ERT. And any thoughts you have regarding what is your bar in order to determine the optimal dose? -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [27] -------------------------------------------------------------------------------- So Gena, I'm afraid I may disappoint you with my answers. The Pfizer -- the Phase II of the ALTA study and the progression of this with Pfizer is really in their hands now to talk about. And they announced -- or the abstracts for ASH were announced today. And so they'll be talking about that at ASH. And so I would guide you to wait for those results. The convertibility between Phase II and Phase III, really, we mustn't say anything until we see the Phase III results. As regards the C9ORF, all I can tell you is Pfizer are very pleased with the product that we produce for them. And they have transferred it into their research organization and paid the necessary milestone. As regards to Fabry, I'm going to pass on to Bettina. Bettina, can you talk about Fabry and what we are looking for and what the study will measure? -------------------------------------------------------------------------------- Bettina M. Cockroft, Sangamo Therapeutics, Inc. - Senior VP & Chief Medical Officer [28] -------------------------------------------------------------------------------- Absolutely. So yes, thank you for the question. So as I've mentioned earlier, we dosed the -- the enrollment is ongoing. And the study goal really is to provide a predictable and durable expression. You are going to be measuring this parameter as one of the more important parameters. But as you point out, we're also looking at substrates, Gb3 and lyso-Gb3. These are the basis of some of the challenging symptoms and morbidities in patients with Fabry. So we're going to be monitoring all of this data, along with other data. And remember, this is, first and foremost, the safety and tolerability study to start off with. And we have a Safety Monitoring Committee that will be involved in the decisions as we move on to escalate dose to the optimal dose. We think it is with the totality of the data and the data that we are monitoring from other studies, with other products, of course, as well that we will be making our ultimate decisions on the dose that we bring forward in our cohort expansion. -------------------------------------------------------------------------------- Operator [29] -------------------------------------------------------------------------------- Our next question comes from the line of Eric Joseph with JPMorgan. -------------------------------------------------------------------------------- Eric William Joseph, JPMorgan Chase & Co, Research Division - VP & Senior Analyst [30] -------------------------------------------------------------------------------- Maybe just perhaps a strategic one. I'm curious to get a sense of whether there are -- well, how we should be thinking about the potential for additional -- largely wholly owned programs coming forward to the clinic over the next 12 to 18 months relative to, I guess, what we've seen over the past year, a fair amount of leveraging up the platform through partnering activities. -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [31] -------------------------------------------------------------------------------- Thank you for your question, Eric. Mark, this feels like one for you, please. -------------------------------------------------------------------------------- D. Mark McClung, Sangamo Therapeutics, Inc. - Executive VP & Chief Business Officer [32] -------------------------------------------------------------------------------- Eric, thanks for the question. So if you take a look at the deals that we've done, including those most recently, really, the driver of that is twofold. In some cases, they've come to us. Obviously, in the Biogen case, in the -- a couple of the candidates that we had progressing towards the IND. But they came to us with a view to actually expanding the number of CNS targets that we weren't otherwise considering. So we looked at that as being really an extension of our potential pipeline. The second way we sort of take a look at partnerships is whether they're bringing a specific expertise and the resources necessary to accelerate the products assuming they're successful to patients. And in that particular case as well as the Novartis case, both of those really fit those type of criteria. We fully intend and we -- to become a genomics medicine company, and we are continually looking to advance wholly owned assets, which we would take into the clinic. And I think a good example of that is what you've heard in terms of TX200 as well as the follow-on programs that we're intending to take forward into the clinic for our CAR-Treg programs. -------------------------------------------------------------------------------- Eric William Joseph, JPMorgan Chase & Co, Research Division - VP & Senior Analyst [33] -------------------------------------------------------------------------------- Got it. I guess in addition to Treg, are there certain disease states or targets that you have essentially walled off and are prioritizing for internal development? -------------------------------------------------------------------------------- D. Mark McClung, Sangamo Therapeutics, Inc. - Executive VP & Chief Business Officer [34] -------------------------------------------------------------------------------- We've not disclosed that. I mean we're taking a look at -- obviously, we've got interest in the autoimmune space, as we talked about with our CAR-Treg programs and Jason touched on earlier. Naturally, we're working heavily within the CNS area with our partners in Biogen and our partnership with Novartis. And so there may be targets that we choose in that particular space, but we've not made any decisions on that as yet. We don't believe you can really stick in one area and then go after it. We need to let the science drive us towards things that we believe are important to develop for patients. -------------------------------------------------------------------------------- Operator [35] -------------------------------------------------------------------------------- Our next question comes from the line of Ritu Baral with Cowen. -------------------------------------------------------------------------------- Lyla Youssef, Cowen and Company, LLC, Research Division - Research Associate [36] -------------------------------------------------------------------------------- This is Lyla on for Ritu. Just 2 quick questions from me. First, on the STAAR Fabry study, are you still seeing impacts from COVID on enrollment? I know you completed the first cohort. But are all the sites up and running? And then as a quick follow-up regarding the Pfizer collaboration with the ALS program, what are the next steps for the program? Specifically, what's the next opportunity for a milestone to be reached? Is that contingent on them initiating a clinical trial? Any color on this would be helpful. -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [37] -------------------------------------------------------------------------------- Thank you for your question. We haven't -- we or Pfizer haven't commented on the ALS program and when the milestones will come. I would say, again -- I would reiterate what I said in the call script. It's a remarkable piece of science to be able to suppress the transcription of one allele and leave the other one untouched. It is why Pfizer came to us. And we have achieved what they requested of us, and it's moved forward to them. So let's hope -- because it's such a dreadful disease, let's hope it gets to patients as soon as possible. For the Fabry disease, I'm going to refer you to Bettina, who will answer that question. -------------------------------------------------------------------------------- Bettina M. Cockroft, Sangamo Therapeutics, Inc. - Senior VP & Chief Medical Officer [38] -------------------------------------------------------------------------------- Yes. And thank you for that question. So on the clinical operations side, I have to say, we have an excellent clinical operations team working very hard on maintaining the relationship with all the sites. And during the pandemic, we've also been able to initiate sites, qualify other sites as we expand our footprint for the study. Of course, COVID has had an impact worldwide, some regions more than others. We are confident that we can keep going. We're following the enrollment and dosing of the first 2 patients to keep going with our enrollment despite the pandemic at this stage where the things we've put in place from an operational perspective with home visits and with virtual assistance have really helped us make sure that we, together with the sites, manage to guide the patients through the enrollment and screening procedures to ultimately get us to dosing and then follow-up. -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [39] -------------------------------------------------------------------------------- I'm very pleased with the way the clinical team have navigated it. We feel we have a responsibility to do this well, to make sure that the patient comes first, whether they're being treated for COVID, in which case, we should not be taking up doctors times. But we've been ready to dose as soon as the window was open. And I think Bettina and her team have done a remarkable job to get us here. -------------------------------------------------------------------------------- Operator [40] -------------------------------------------------------------------------------- Our next question comes from the line of Nicole Germino with Truist. -------------------------------------------------------------------------------- Nicole Germino, Truist Securities, Inc., Research Division - Associate [41] -------------------------------------------------------------------------------- On Fabry, given the competition in this space, what is it more specifically about Sangamo's AAV capsid or the promoter that make it differentiated and better than your peers? And have you made that determination? And then does -- how does ST-920 impact the renal and cardiac tissue? -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [42] -------------------------------------------------------------------------------- So let me take that one. We are encouraged by the data we've seen from AAV6 in hemophilia A. But until we dose patients with Fabry disease, we really can't and shouldn't comment on what it's going to look like. The animal data looks very encouraging. We achieve super maximal dosing and super maximal effect. And in the animal dosing that we recently published, we were able to show benefit to both the heart and the kidney. But I'm just prudent in telling people to wait and let's see what the clinical results are. We're guiding that we'll show the clinical results towards the end of next year when the dose escalation phase is completed. -------------------------------------------------------------------------------- Operator [43] -------------------------------------------------------------------------------- Our next question comes from the line of Evan Wang with Guggenheim Securities. -------------------------------------------------------------------------------- Boran Wang, Guggenheim Securities, LLC, Research Division - Associate [44] -------------------------------------------------------------------------------- I had 2, one on hem A, another on the allo CAR-T program. On hem A, what's the plan going forward to share data on the Phase I/II? I know you'll have the update at ASH, but are there any specific time points to provide a complete picture of the data? And will that require kind of all patients to acquire across that time point? And on the allo CAR-T program, we've seen some data -- early data from other allo programs from Allogene and CRISPR. Any thoughts on the initial data generated so far? And any kind of learnings you can take, especially given some of the debts we've seen in those trials? -------------------------------------------------------------------------------- Alexander D. Macrae, Sangamo Therapeutics, Inc. - CEO, President & Director [45] -------------------------------------------------------------------------------- So let me do the hem A and then pass this to Jason to comment on allo. So the -- I'm going to say again what I've been saying. Pfizer will -- are in control of the release of data on hemophilia A. There is an abstract that's been accepted for ASH, and they will use whatever conference schedule in the future to continue to demonstrate the benefit to patients of reduced -- absent bleeding events and no requirement for factor. Jason, can you talk about the allogeneic question, please? -------------------------------------------------------------------------------- Jason Fontenot, Sangamo Therapeutics, Inc. - SVP of Cell Therapy & Interim Head of Research [46] -------------------------------------------------------------------------------- Sure. Thank you, Sandy. So I believe that you're referring to allo CAR-T programs in oncology and that -- we have a partnership, as you know, with Kite Gilead to support their allogeneic CAR-T programs. Kite has guided that we're expecting to begin the studies on their allogeneic CD19 targeted CAR-T therapy next year. And we believe that the data that we've seen so far from others are consistent with these therapies having real therapeutic potential. And we're excited about it, and we're excited to see Kite get the therapies into patients. -------------------------------------------------------------------------------- Operator [47] -------------------------------------------------------------------------------- I will now turn the call back over to Head of Corporate Communications, Aron Feingold, for any further remarks. -------------------------------------------------------------------------------- Aron Feingold, Sangamo Therapeutics, Inc. - Head of Corporate Communications & IR Officer [48] -------------------------------------------------------------------------------- Thank you once again for joining us today and for your questions. We look forward to keeping you updated on our future developments. -------------------------------------------------------------------------------- Operator [49] -------------------------------------------------------------------------------- Ladies and gentlemen, this concludes today's conference call. Thank you for participating, and you may now disconnect.