NASDAQ:SGMO Sangamo Therapeutics Q1 2024 Earnings Report $0.70 -0.02 (-2.75%) As of 04:00 PM Eastern Earnings HistoryForecast Sangamo Therapeutics EPS ResultsActual EPS-$0.27Consensus EPS -$0.22Beat/MissMissed by -$0.05One Year Ago EPS$0.12Sangamo Therapeutics Revenue ResultsActual Revenue$0.48 millionExpected Revenue$8.51 millionBeat/MissMissed by -$8.03 millionYoY Revenue GrowthN/ASangamo Therapeutics Announcement DetailsQuarterQ1 2024Date5/9/2024TimeAfter Market ClosesConference Call DateThursday, May 9, 2024Conference Call Time4:30PM ETConference Call ResourcesConference Call AudioConference Call TranscriptSlide DeckPress Release (8-K)Quarterly Report (10-Q)Earnings HistoryCompany ProfileSlide DeckFull Screen Slide DeckPowered by Sangamo Therapeutics Q1 2024 Earnings Call TranscriptProvided by QuartrMay 9, 2024 ShareLink copied to clipboard.There are 13 speakers on the call. Operator00:00:00Good day, and welcome to the Sangamo Therapeutics First Quarter 2024 Teleconference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker today, Louise Wilke, Vice President of Investor Relations and Corporate Communications. Please go ahead. Speaker 100:00:37Thank you. Good afternoon, everyone. Thank you for joining us on the call today. On this call are several members of the Sangamo executive leadership team, including Sandy MacRae, Chief Executive Officer Pratyusha Durababu, Chief Financial Officer Amy Pula, Head of Research Natalie Dubois Stringfellow, Chief Development Officer. Slides from our corporate presentation can be found on our website, sangamo.com, under the Presentations page of the Investors and Media section. Speaker 100:01:03This call includes forward looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements related to the therapeutic and commercial potential of our product candidates and engineered capsids and the potential of our next generation genome engineering technology the anticipated plans and time lines of Sangamo and our collaborators for regulatory submissions, initiating and conducting clinical trials and presenting clinical data advancement of our product candidates anticipated regulatory submissions advancement of preclinical programs to the clinic our strategic reprioritization and reallocation of resources and the anticipated benefits thereof plans to partner certain of our programs the sufficiency of our resources, cash runway and plans to seek additional capital upcoming catalysts and milestones and other statements that are not historical facts. Actual results may differ materially from what we discuss today. These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC, specifically in our annual report on Form 10 ks for the fiscal year ended December 31, 2023, supplemented by our quarterly report on Form 10 Q for the quarter ended March 31, 2024, filed with the SEC. The forward looking statements dated today are made as of this date, and we undertake no duty to update such information except as required by law. Speaker 100:02:22Please note that all forward looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding. Now I'll turn the call over Speaker 200:02:31to our CEO, Sandy Macrae. Thank you, Louise, and good afternoon to everyone joining the call. I'm very pleased to be speaking to you today from the American Society of Cell and Gene Therapy Annual Meeting in Baltimore, where we've been presenting important preclinical data from our epigenetic regulators, AAV capsid delivery platform and next generation genomic engineering platform. These data showcase both the depth of Sangamo's neurology pipeline and the power of our scientific capabilities, which we believe provide strong opportunities to advance programs ourselves and with potential partners. In order to progress these compelling programs Sangamo must be well capitalized. Speaker 200:03:18The leadership team on addressing our funding needs. In late March, we are pleased to announce a registered direct offering with institutional shareholders, including an important existing investor that raised approximately $24,000,000 in gross proceeds. This was a significant development and we are thankful for their support of our science and our mission. That was however the first step in our current journey to securing additional funding. I would like to emphasize that we are resolutely focused on building upon this foundation to position Sangamo for long term success. Speaker 200:03:58That continues to be my number one priority. Business development is an important part of these efforts and I am pleased with the progress that is being made on this front. We are currently engaged in very encouraging conversations with multiple potential partners across our portfolio, including our Fabry disease program, our novel stack BBB engineered capsid, our preclinical neurology product candidates and our next generation genome engineering capabilities. I understand your desire to hear more concrete news on this front, but we are unable to share more until any potential transaction is finalized. Be assured that we are encouraged by the progress of these discussions and hope to announce news of 1 or more transactions. Speaker 200:04:50Over recent months, Sangamo has presented important preclinical data that solidify our sharpened focus in neurology, validate our differentiated science and help contextualize why we made this important decision to dedicate ourselves to addressing neurological disorders. In March, we were proud to share remarkable preclinical data from our new intravenously administered neurotrophic AAV capsid, which demonstrated industry leading blood brain barrier penetration and brain transduction in non human primates. This novel capsid STACK BBB showed robust penetration of the blood brain barrier with 700 fold higher transgene expression in neurons compared to the benchmark capsid AAV9 and outperformed all other known published capsid variants evaluated in the study. Combined with our potent epigenetic regulation cargo, we showed robust STACK BBB mediated expression of zinc finger cargo and neurons with potent and widespread repression of the prion and tau genes observed across all key brain areas, illustrating the exciting potential to modify disease progression in prion disease and various tauopathies. These data support further advancement of our prion and tau programs, which we are on track for regulatory submission sent to the clinic by the end of 25. Speaker 200:06:26Meanwhile, we continue to advance our lead candidate in chronic neuropathic pain, NAV1.7, which uses an established intrathecally administered capsid towards an IND submission expected in the Q4 of this year. We believe our ability to combine potent zinc finger epigenetic regulation payloads with exciting new industry leading capsid delivery technology could unlock significant potential for the treatment of devastating urological diseases. Indications for which delivery of treatments to the central nervous system has historically proved challenging. Building on this, we are proud to be presenting 3 platform presentations in 17 posters at this week's ASGCT Annual Meeting. These presentations showcase the depth of our neurology pipeline, including various applications for zinc finger, epigenetic regulation platform, exciting advances in our capsid delivery technology and the discovery of potentially transformative next generation integrates technology engineered to enable large scale genome editing. Speaker 200:07:41In epigenetic regulation, we have shared advance in zinc finger activators and repressors for the potential treatment of a remarkable range of neurological diseases such as prion disease, thiopathies, Charcot Marie Tooth diseases type 1a and 2a, Dravet syndrome, SOD1 mediated amitrophic lateral sclerosis or ALS, Phelan McDermott syndrome, Parkinson's disease, Angelman syndrome and many other neurological disorders. We have also demonstrated Sangamo's potent delivery capabilities developed through our AAV capsid engineering platform, sifter. Here we presented additional STACK BBB findings including prion and tau repression data achieved via STACK BBB delivery. We also presented for the first time exciting initial findings for a possible mechanism supporting how STACK BBB may cross the blood brain barrier. Our sifter platform is designed to engineer capsid for various routes of administration such as intrathecal and intravenous delivery. Speaker 200:08:50Sifter can also engineer capsids for in vitro discovery. So in our platform presentation tomorrow, we will present findings from STK-one hundred and fifty, a novel capsid that's been shown to be highly potent in neurons and enables high throughput screening of neurology focused transcriptional regulators. We anticipate the STAG-one hundred and fifty, which we believe manufacturers easily at small scale will help accelerate the discovery of potent and highly specific epigenetic regulators. Finally, building on our deep expertise in that protein DNA interactions derived from our zinc finger platform, we presented for the first time a potentially breakthrough new approach for integrating large sequences of DNA into the genome to potentially treat with a single medicine patients who have unique mutations in the same gene. Precisely integrating large synthetic DNA constructs into a desirable chromosomal site has been the dream for people working in this field for many years. Speaker 200:09:56Our modular integrase or MINT platform is a versatile protein guided genome editing method that understands and engineers BXB1, a serum, serine recombinase to insert or replace entire genes and adds to Sangamo's toolbox of editing capabilities. We are hopeful that our MINT platform could be used to correct many disease causing mutations in a diverse patient population by inserting a correct copy of the gene into its natural locus. MINDS could be deployed internally for various neurological indications, but also provide potential partnering opportunities both for human disease and in agricultural biotech settings. We are already in active discussions with potential partners about our integrase capabilities and are hopeful that Mint could provide us with another potential non dilutive funding opportunity. Alongside our presentations in this topic at ASGCT this week, we've also published a manuscript in bio archive further outlining these next generation integrase advancements, which is also available on the publications page of the website. Speaker 200:11:13I encourage you to learn more about this exciting development in the field of genomic medicines. Now looking at our clinical programs, we have made strong advances in the Q1 for our Fabry disease program, having dosed the final patient in the Phase III STAR study of Isoraleogene cevaparvek, our investigational gene therapy for the treatment of Fabry disease. With 33 patients now dosed, screening, enrollment and dosing are complete. One additional patient has been able to stop enzyme replacement therapy or ERT resulting in a total of 14 patients withdrawn from ERT to date. The 4 remaining patients dosed since February 2024, who began the study on ERT already have plans in place to withdraw ER treatment at the appropriate time. Speaker 200:12:05At the 20th Annual World Symposium in February, we presented compelling updated preliminary clinical data from the STAR study showing sustained benefit and a differentiated safety profile. These results underscore the program's potential as a single administration treatment for Fabry disease. As a reminder, this quarter we announced alignment with the U. S. FDA on an abbreviated pathway to potential approval. Speaker 200:12:34We've also been granted prime eligibility by the European Medicines Agency and iLab designation by the UK Medicine and Healthcare Products Regulatory Agency. We are engaged in active discussions with potential collaborator partners for our Fabry disease program and continue to defer additional investments in planning for a potential registrational trial until a collaboration partnership or financing for this program is secured. Moving to our partner clinical program, we look forward to the pivotal readout expected in mid-twenty 24 in the Phase 3 AFFINE trial of gyratocogine, fitoparvovec, an investigator gene therapy we're developing with Pfizer for patients with modestly severe to severe hemophilia A. A. Pfizer anticipates submitting a biologics license application and a marketing authorization application in early 2025 of the pivotal readout is supportive. Speaker 200:13:40As a reminder, we are eligible to earn up to $220,000,000 in milestone payments and up to 14% to 20% royalties on potential sales from this program. We end the quarter with approximately $54,000,000 in available cash and cash equivalents, which includes funds from the aforementioned registered direct offering. We believe these resources in combination with this cost savings expected from the recent restructurings, workforce reduction and other potential cost reductions will be sufficient to fund our planned operations into the Q3 of 2024. As I outlined earlier, we are actively pursuing a range of different options to raise important additional capital and are encouraged by the business development discussions ongoing across our Fabry program, capsid engineering and next generation genome engineering efforts. We believe our company has the science required to potentially transform the lives of patients living with devastating neurological conditions and are committed to raising the funding required as we seek to make our vision a reality. Speaker 200:14:55Operator, please open the lines for questions. Operator00:14:59Thank you. At this time, we will conduct a question and answer session. Our first question comes from the line of James Stamos at Jefferies. James, your line is open. Speaker 300:15:26Hi, this is James on for Maury Greatcross. Congrats on the progress and thanks for taking our question. I just wanted to ask, where are you with preclinical and GLP tox studies with STACK BBB? And what are your latest thoughts on which targets or indications would make the most sense to partner or keep in house? Speaker 200:15:43Thank you for your question. We have made one of the reasons that we like STACK BBB so much is that our manufacturing team have been able to work with it and found that it is very manufacturable. They can take it so far up to 50 liter scale and are going higher that they can use the same purification and assay techniques and that it will that it feels like a capsid that will be able to give us great yield and also be able to use many of the processes in CMC work that really is underappreciated in the importance of capsid selection. We've said publicly that we are moving ahead with tau and with Prion. You could imagine the amount of external interest in tau, not just because it's a high unmet medical need, but because the capsid gives you the brain penetration and widespread delivery that's needed. Speaker 200:16:47And our zinc finger repressors are really unique in being able to almost switch off tau in the cells that they can get to. We are having many discussions with companies about our capsid and we'll choose wisely, I believe, which ones we partner and which ones we take forward ourselves. What we understand and I want to say this again and repeat what I said in the chosen words is that we understand the need to bring in money to the company. And that's always a balance between the desire that we have to take things forward and the money that's available from many of the partnerships I'm sure we will achieve in the coming weeks. Speaker 300:17:33Great. That's very helpful. And just to go on to like some of the more specialized parts of your ASGCT presentation, can you talk about the importance of targeting the PoNS and motor neurons in Alzheimer's? And are there any other companies that you're aware of that have the ability to target these sites? Speaker 200:17:49I think thank you for your question. It's really been a remarkable ASGCT and the field moves closer to these tools being able to address such important disease. Amy, can you talk a little about this? I know this is one of your passions. Speaker 400:18:07Yes, I would love to. Thank you for the great question. And what's really important here is being able to target brain regions that would be traditionally challenging to target using something like a direct injection approach. The PoNS being part of the brain stem is not a region that you would necessarily want to reach using a direct injection guided approach even for the most severe of diseases. So we were so excited to see the biodistribution that was able to be achieved with the SacBB capsid, especially for what it means for the tau program. Speaker 400:18:37The PoNS is critical in progressive supranuclear palsy, but also other tauopathies like Alzheimer's disease. In addition, as you noticed, the motor cortex, which is part of the frontal cortex, we use this as an example of how STACK PBB is able to transduce those cortical neurons. Really, we believe that this kind of widespread brain targeting is critical for halting or slowing the progression of the disease. Speaker 200:19:03Thanks, Amy. Speaker 300:19:05Great. Thank you for taking our questions. I'll hop back in the queue. Operator00:19:08Thank you very much. One moment for our next question, please. Our next question comes from Gena Wang of Barclays. Gena, your line is open. Speaker 500:19:20Thank you for taking my questions. I have 2. The first one also is about the SPECT BBB and it seems like very exciting new capsid. What is the highest dose you used in non human primates and what is the limiting dose toxicity there? And then the second question quickly regarding the Heme, I know it's your partner Pfizer's is in charge, but just wondering for the mid-twenty four Phase 3 update, would that be just a press release from Pfizer? Speaker 500:19:51And then for the 220 milestone payment, the first milestone, is that the drug approval? And is that the big portion of the $220,000,000 Speaker 200:20:04Gina, thank you for your questions. And I know you've been following Himi for a great deal of time. So it's good to be coming closer to the point where it gets to registration and approval. So let me do the let me pass this on to 2 people. Amy, can you talk about the dose that we've used and whether there was any toxicity, please? Speaker 400:20:31Yes, absolutely. And we as part of the SYKBP study that we discussed yesterday and we also will discuss later today at the poster session on ASGCT, we performed a dose range finding study with our tau and zinc clinical lead zinc finger and we did 3 different doses and the top dose was 1e14. We're really happy to see that with the histopathology results that we didn't see any dose limiting toxicity and really no findings in the brain, the spinal cord or the liver, these peripheral organs. So really positive and encouraging data. Speaker 200:21:06Gina, does that answer your Speaker 500:21:10Yes. Like more thinking, other than the dose finding, like what is the highest dose you tested for the safety toxicology perspective? Speaker 200:21:201e14. So 1e14. Speaker 500:21:22That's 1e14. Okay. Speaker 200:21:24It's the highest we've so we went there in the efficacy study that we just reported and we really saw nothing sorry, we saw nothing that would give us any concern. When we will now go into the GMP tox study, which is the only thing remaining to complete the IND enabling studies for that. But we will clearly start at a lower dose in the clinical study because that's the right thing to do and that's what the agency will ask us to do. But thus far, we've it's a very clean capsid, which is great, great news. And then can we talk about humane, Natalie? Speaker 200:22:05I know you've been speaking to your friends at Pfizer regularly. Speaker 600:22:09Sure, sure. Yes, we're very excited that the mid-twenty 24 pivotal readout in MA is coming up soon. We the partnership allows for $220,000,000 in potential milestone and 14% to 20% royalty on potential sale. But the specific amount of each milestone is not publicly disclosed. But this could start as early as the start of 2025 if the pivotal readout is positive and if Pfizer elect to see regulatory approval to the program. Speaker 200:22:43And Gina, just to reflect on that, we're limited in what we can say. When you sign these deals, there's always an agreement about who talks about what. But the regulatory milestones, which are we get one for the U. S, one for Europe and one for Japan are not for approval, they're for submission of the package. And then the commercial milestones, which are for 1st patient dosed rather than for reaching a certain sales threshold. Speaker 200:23:23So they're very, very favorable milestones that will fund the company in 2025 and 2026. Speaker 100:23:32Very helpful. Speaker 600:23:32Yes. I need to mention that the significant portion of the €220,000,000 is near term and we could also choose to monetize them now. Speaker 200:23:42Yes. So the closer so Gina, the closer we get to the when the data is revealed and then when Pfizer submits this, we have the option of monetizing those royalties. But we can also choose to wait and use them to fund 202526. And what will make that decision is the non dilutive funding from business development that we do now will tell us whether we need to make that decision. We are I know that there's a concern from people that followed us for a long time about our near term cash runway. Speaker 200:24:20We feel that we are fortunate to be able to do both capsid deals, fabry deals, technology deals and to have this large amount of cash next year that we can bring forward if necessary. Speaker 500:24:37Thank you very much. Speaker 200:24:39Thank you, Tina. Operator00:24:40Thank you very much. One moment for our next question please. Our next question comes from Patrick Trucchio of H. C. Wainwright. Operator00:24:49Patrick, your line is open. Speaker 700:24:52Hi, everyone. This is Luis Santos for Patrick. Congratulations on the recent progress and your presence your impressive presence at ASCCT. I am interested in knowing if you already guided to the tau program and if it's going to be in Alzheimer's, do you know which patients you're going to be treating and which other indications you are going to push forward here with tau program? And then I have a follow-up question. Speaker 200:25:26So thank you for your question. We haven't guided on that. There's been an enormous amount of interest in our presentation yesterday at ASGCT. It clearly was, I think, the most remarkable presentation of all of the many people that are moving forward in blood brain barrier penetrant capsids. We feel that the tau asset, the cargo that's within it, doubles down, makes us even more interesting. Speaker 200:25:59The role of tau in Alzheimer's, I think, is becoming increasingly clear. The Biogen data is very encouraging. The idea that it's the thing that is relevant to the clinical stages of tau rather of Alzheimer's rather than in the early pre symptomatic ones makes this a much more easy lift. However, we are very conscious that Alzheimer's studies are large programs and that we need to make sure that we either do it ourselves well or partner with someone who has a real expertise in Alzheimer's. Speaker 700:26:41On the follow-up thank you. That was helpful. On the follow-up on the partnerships, but platform, you mentioned that there are business development opportunities there. Do you have any potential collaborators in mind? And what diseases would you be targeting? Speaker 200:27:05The mint integrates is remarkable. The presentation at ASGCT was just 57 minutes ago and there was a lot of excitement in the room about it. We have already been we've been speaking to people for 3 months on this. And I want to put the business development discussions into perspective. We had the regulatory clarity for Fabry just in February. Speaker 200:27:34We had the capsid results just in March. We've finally taken Mint to a place that we feel we can share it in May. So I understand the desire for signed deals, but the results are going to lead to them have only come out over the past 2 or 3 months, which is means that at the moment you could imagine our business development group is incredibly busy in talking to lots of people about this. Now integrase is something that has the potential for really disruptive genomic engineering. It's the kind of thing that David Lou, when he was asked, said was his dream of what the ideal genomic medicine was, which was an integrase that could be engineered to go to your site of choice and put in pieces of gene size pieces of DNA that would replace all of the mutations downstream. Speaker 200:28:39And the team at Sangamo because of their expertise in understanding the interaction between proteins and DNA from the Zinc Finger platform have been able to do this remarkable engineering and the whole alpha fold and understanding artificial intelligence of how you understand proteins has helped us. We feel that this is something that Sangamo can use, but it also is has attracted great interest from the hardcore science pharma companies that you could imagine who all see this as the future of genomic engineering. And so we have a responsibility to put it in as many people's hands as possible. I think you will hear more about this Mint platform for some time to come. Speaker 700:29:32Thank you. Operator00:29:33Thank you very much. One moment for our next question please. Our next question comes from the line of Luca Yeezy at RBC. Luca, your line is open. Speaker 800:29:46Hello, great. Thanks for taking our questions. This is Lisa on for Luca. I just want to ask another question on the Mint technology. Just wondering if you can give us a sense of what kind of targets here would be good candidates. Speaker 800:30:05For instance, would it be possible to integrate something like a full length CFTR gene, which is around roughly 190 kilobases? Or should we think about something larger like full length dystrophin, which is measuring closer to over 2,000 kilobases? Just any color here would be helpful. Thanks. Speaker 100:30:28So Speaker 200:30:31you asked really important and interesting questions. And as always with Sangamo, thought about size, because as you know, zinc fingers are an 8th of the size of CRISPR and even the small mammoth CRISPRs are 4 times as big as the zinc finger. And that's important because of delivery. It's the same with Mint, because we try to keep the technology as small as possible. So as we could put both the integrase and a cargo in an AAV. Speaker 200:31:11Now when you get to the size of some of the genes that you're talking about, you end it comes down to what kind of delivery you're using. My dream for Mint is to be able to drop a cDNA copy of the gene or of the exons that are mutated into intron 1 of the genome. It would then be run off of the promoter, have all the locus control elements and it would alleviate the requirement to edit all the individual mutations that usually are the cause of genomic disease. Now to put in a whole genomic copies would be unnecessary, I would argue. And then we need to look at what are the diseases where the cDNA is packageable into the delivery mechanisms that are available because the mint should be size agnostic and has so far shown great potential. Speaker 200:32:21Amy, is there anything else Amy? I'm aware that you're on the line and more and as our Head of Research, perhaps you have other things to add to that. Speaker 400:32:31No, that's fantastic explanation, Sandy. I would just add that there are specific genes as well that are involved in some neurological disorders, and I'm thinking of things like Rett syndrome or MECP2 needs to be fine tuned at a very specific level, something like an integrase where you could integrate in perhaps the healthy copy of exon 1 or even a bigger portion of the gene, so that it's under control of the endogenous promoter could be really transformative for a disease like that. So I think it's incredibly exciting to see what are the opportunities around this new MYNT platform. Speaker 800:33:07Thanks so much. Thanks for taking my questions. Operator00:33:10Thank you very much. One moment for our next question. Our next question comes from Ritu Baral of TD Cowen. Ritu, your line is open. Speaker 900:33:23Hi, guys. Just a quick question on the Fabry program actually. You've just 33 patients and you saw the data at World Plus, you have the Phase 3 path going forward agreed upon with FDA. What is the next data update that we're going to get from that? And also, I guess, is there a data point or a follow-up point in a subset of patients that's gating to potential business development around or out licensing of this program? Speaker 900:33:56Thanks. Speaker 200:33:58Thank you, Ritu. I'm going to pass this on to Natalie. But I'll just remind you again, the whole business development conversation around Fabry changed with Peter's new way of thinking about this at the agency and the conversations that we had in February. And so it's like a new conversation we're having with potential partners. But Natalie, can you address some of the technical pieces, please? Speaker 600:34:25Yes, sure. So we've completed the dosing of all the patient in the Phase III. So for a total of 33 patients, We continue to analyze the data, but this the body of data that we have thus far is not gate limiting for any partnership and any of the Phase IIb preparation or registrational trial preparation. So we continue to analyze the data, but we and I've agreed with the FDA that we have enough data to move on to a Phase 2b. And that's what we're discussing with partners, potential partners. Speaker 900:35:05Sandy, when you say that the discussion has changed, are you talking about the structure of the deal that is in Sangamo's best interest? Like is it do you think it could be like a COCO going forward? Or is it just really sort of valuation terms that are different now? Speaker 200:35:24I think it's much simpler than that, Ritu. There was a concern that there wasn't clarity about what the regulators were wanting. And there was this worry that we've all talked about that it was going to be a head to head against ERT. And the agency now saying, no, it's a simple single study and that we can they've named the number of patients and they've said we can come back halfway through the study and show them the data we've got if we feel it's compelling. I think that has completely changed how the various companies are going to. Speaker 200:36:08And I think great credit to Peter. He is walking the talk in this. And we have benefited greatly from it as being almost like the poster child for what it is approval of rare disease gene therapy is. And if I may, Rita, we've started the we've initiated the conversations with the Europeans and uniquely the FDA is volunteering to send one of their staff with us to that conversation because they feel that it's the way forward for these kind of therapies is for a common approach to them. And I think it reflects the support that we have from the FDA in these discussions. Speaker 900:37:11Thank you, Sandy. Operator00:37:13Thank you very much. Our next question comes from the line of Nicole Garmino of Truist. Nicole, your line is open. Speaker 1000:37:21Hi, this is Bill on for Nicole. We have a question. Speaker 1100:37:25It's given the uptake for ROCKTAVIAN, what is Speaker 1200:37:28the read through to potential uptake for Fabry disease gene therapy? Speaker 200:37:35So thank you for your question. So the Roktavian launch I know has been a subject of great debate. We can't comment on how BioMarin launched it. All I know is the reason that we chose Pfizer to be our partner is I have great confidence that when Pfizer chooses to launch something, they do it well and effectively. And that follows through to our choice of partners for Fabry disease. Speaker 200:38:05There are several companies and there are some that are more obvious as being great commercialization engines and great launch companies. What I can tell you is in our conversations with these companies, we have had notes from the patient support groups who have been very keen to make sure that the pharma partners understand how supportive they are of the results that they have seen in our Fabry program. Because you and I will talk about the biochemistry and the biopsies. What the What the patients have said is how much better they feel on it, how delighted that they are to be off of ERT. So now we have 14 patients off of ERT and the 4 that are remaining on ERT in the study have already booked when they're going to come off of the therapy. Speaker 200:39:01So this has enormous support from the patients. And I think that is what is going to make it a very successful launch for the partner that can take it forward and make a difference. Speaker 600:39:17And if I may add, we also have RMAT and PRIME, which are designation that really analyze an unmet medical need for Fabry. So the Fabry patient, even though can have access to ART, are not satisfied with the current medicine standard of care. Speaker 1200:39:35Thank you so much. Operator00:39:38Thank you very much. At this time, I'm showing no further questions. And I would now like to turn the call back to Louise Wilkie for closing remarks. Speaker 100:39:48Thank you very much, and thanks to everyone for joining us on the call today. We look forward to speaking to you soon.Read morePowered by Conference Call Audio Live Call not available Earnings Conference CallSangamo Therapeutics Q1 202400:00 / 00:00Speed:1x1.25x1.5x2x Earnings DocumentsSlide DeckPress Release(8-K)Quarterly report(10-Q) Sangamo Therapeutics Earnings HeadlinesSangamo Therapeutics, Inc. (NASDAQ:SGMO) Receives $5.17 Average Price Target from AnalystsMay 8 at 1:05 AM | americanbankingnews.comSangamo Therapeutics (SGMO) Projected to Post Quarterly Earnings on ThursdayMay 7 at 4:05 AM | americanbankingnews.comElon just did WHAT!?As you may recall, Biden and the Fed were working on a central bank digital currency, or CBDC. Had they gotten away with it, the Fed and U.S. banks could have seized control of our financial lives forever. But Trump stopped them cold on January 23rd, 2025, when he outlawed CBDCs… Paving the way for Elon Musk's secret master plan.May 9, 2025 | Brownstone Research (Ad)Sangamo Therapeutics Announces Important Derisking Milestones in Pathway to Anticipated BLA Submission for ST-920 in Fabry DiseaseMay 7 at 3:26 AM | finance.yahoo.comSangamo Therapeutics Announces First Quarter 2025 Earnings CallMay 6 at 4:35 PM | businesswire.comSangamo Therapeutics to Present Neurology Pipeline Advances at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)April 28, 2025 | businesswire.comSee More Sangamo Therapeutics Headlines Get Earnings Announcements in your inboxWant to stay updated on the latest earnings announcements and upcoming reports for companies like Sangamo Therapeutics? Sign up for Earnings360's daily newsletter to receive timely earnings updates on Sangamo Therapeutics and other key companies, straight to your email. Email Address About Sangamo TherapeuticsSangamo Therapeutics (NASDAQ:SGMO), a clinical-stage genomic medicine company, focuses on translating science into medicines that transform the lives of patients and families afflicted with serious diseases in the United States. The company's clinical-stage product candidates are ST-920, a gene therapy product candidate, which is in Phase 1/2 clinical study for the treatment of Fabry disease; TX200, a chimeric antigen receptor engineered regulatory T cell (CAR-Treg) therapy product candidate that is in Phase 1/2 clinical study for the prevention of immune-mediated rejection in HLA-A2 mismatched kidney transplantation; SB-525, a gene therapy product candidate, which is in Phase 3 clinical trial for the treatment of moderately severe to severe hemophilia A; BIVV003, a zinc finger nuclease gene-edited cell therapy product candidate that is in Phase 1/2 PRECIZN-1 clinical study for the treatment of sickle cell disease. Its preclinical development products focus on CAR-Treg cell therapies for autoimmune disorders and genome engineering for neurological diseases. Sangamo Therapeutics, Inc. has collaborative and strategic partnerships with Biogen MA, Inc.; Kite Pharma, Inc.; Pfizer Inc.; Sanofi S.A.; Novartis Institutes for BioMedical Research, Inc.; Shire International GmbH; Dow AgroSciences LLC; Sigma-Aldrich Corporation; Genentech, Inc.; Open Monoclonal Technology, Inc.; and California Institute for Regenerative Medicine. The company was formerly known as Sangamo BioSciences, Inc. and changed its name to Sangamo Therapeutics, Inc. in January 2017. Sangamo Therapeutics, Inc. was incorporated in 1995 and is headquartered in Richmond, California.View Sangamo Therapeutics ProfileRead more More Earnings Resources from MarketBeat Earnings Tools Today's Earnings Tomorrow's Earnings Next Week's Earnings Upcoming Earnings Calls Earnings Newsletter Earnings Call Transcripts Earnings Beats & Misses Corporate Guidance Earnings Screener Earnings By Country U.S. Earnings Reports Canadian Earnings Reports U.K. Earnings Reports Latest Articles Why Nearly 20 Analysts Raised Meta Price Targets Post-EarningsOXY Stock Rebound Begins Following Solid Earnings BeatMonolithic Power Systems: Will Strong Earnings Spark a Recovery?Datadog Earnings Delight: Q1 Strength and an Upbeat Forecast Upwork's Earnings Beat Fuels Stock Rally—Is Freelancing Booming?DexCom Stock: Earnings Beat and New Market Access Drive Bull CaseDisney Stock Jumps on Earnings—Is the Magic Sustainable? 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There are 13 speakers on the call. Operator00:00:00Good day, and welcome to the Sangamo Therapeutics First Quarter 2024 Teleconference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker today, Louise Wilke, Vice President of Investor Relations and Corporate Communications. Please go ahead. Speaker 100:00:37Thank you. Good afternoon, everyone. Thank you for joining us on the call today. On this call are several members of the Sangamo executive leadership team, including Sandy MacRae, Chief Executive Officer Pratyusha Durababu, Chief Financial Officer Amy Pula, Head of Research Natalie Dubois Stringfellow, Chief Development Officer. Slides from our corporate presentation can be found on our website, sangamo.com, under the Presentations page of the Investors and Media section. Speaker 100:01:03This call includes forward looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements related to the therapeutic and commercial potential of our product candidates and engineered capsids and the potential of our next generation genome engineering technology the anticipated plans and time lines of Sangamo and our collaborators for regulatory submissions, initiating and conducting clinical trials and presenting clinical data advancement of our product candidates anticipated regulatory submissions advancement of preclinical programs to the clinic our strategic reprioritization and reallocation of resources and the anticipated benefits thereof plans to partner certain of our programs the sufficiency of our resources, cash runway and plans to seek additional capital upcoming catalysts and milestones and other statements that are not historical facts. Actual results may differ materially from what we discuss today. These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC, specifically in our annual report on Form 10 ks for the fiscal year ended December 31, 2023, supplemented by our quarterly report on Form 10 Q for the quarter ended March 31, 2024, filed with the SEC. The forward looking statements dated today are made as of this date, and we undertake no duty to update such information except as required by law. Speaker 100:02:22Please note that all forward looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding. Now I'll turn the call over Speaker 200:02:31to our CEO, Sandy Macrae. Thank you, Louise, and good afternoon to everyone joining the call. I'm very pleased to be speaking to you today from the American Society of Cell and Gene Therapy Annual Meeting in Baltimore, where we've been presenting important preclinical data from our epigenetic regulators, AAV capsid delivery platform and next generation genomic engineering platform. These data showcase both the depth of Sangamo's neurology pipeline and the power of our scientific capabilities, which we believe provide strong opportunities to advance programs ourselves and with potential partners. In order to progress these compelling programs Sangamo must be well capitalized. Speaker 200:03:18The leadership team on addressing our funding needs. In late March, we are pleased to announce a registered direct offering with institutional shareholders, including an important existing investor that raised approximately $24,000,000 in gross proceeds. This was a significant development and we are thankful for their support of our science and our mission. That was however the first step in our current journey to securing additional funding. I would like to emphasize that we are resolutely focused on building upon this foundation to position Sangamo for long term success. Speaker 200:03:58That continues to be my number one priority. Business development is an important part of these efforts and I am pleased with the progress that is being made on this front. We are currently engaged in very encouraging conversations with multiple potential partners across our portfolio, including our Fabry disease program, our novel stack BBB engineered capsid, our preclinical neurology product candidates and our next generation genome engineering capabilities. I understand your desire to hear more concrete news on this front, but we are unable to share more until any potential transaction is finalized. Be assured that we are encouraged by the progress of these discussions and hope to announce news of 1 or more transactions. Speaker 200:04:50Over recent months, Sangamo has presented important preclinical data that solidify our sharpened focus in neurology, validate our differentiated science and help contextualize why we made this important decision to dedicate ourselves to addressing neurological disorders. In March, we were proud to share remarkable preclinical data from our new intravenously administered neurotrophic AAV capsid, which demonstrated industry leading blood brain barrier penetration and brain transduction in non human primates. This novel capsid STACK BBB showed robust penetration of the blood brain barrier with 700 fold higher transgene expression in neurons compared to the benchmark capsid AAV9 and outperformed all other known published capsid variants evaluated in the study. Combined with our potent epigenetic regulation cargo, we showed robust STACK BBB mediated expression of zinc finger cargo and neurons with potent and widespread repression of the prion and tau genes observed across all key brain areas, illustrating the exciting potential to modify disease progression in prion disease and various tauopathies. These data support further advancement of our prion and tau programs, which we are on track for regulatory submission sent to the clinic by the end of 25. Speaker 200:06:26Meanwhile, we continue to advance our lead candidate in chronic neuropathic pain, NAV1.7, which uses an established intrathecally administered capsid towards an IND submission expected in the Q4 of this year. We believe our ability to combine potent zinc finger epigenetic regulation payloads with exciting new industry leading capsid delivery technology could unlock significant potential for the treatment of devastating urological diseases. Indications for which delivery of treatments to the central nervous system has historically proved challenging. Building on this, we are proud to be presenting 3 platform presentations in 17 posters at this week's ASGCT Annual Meeting. These presentations showcase the depth of our neurology pipeline, including various applications for zinc finger, epigenetic regulation platform, exciting advances in our capsid delivery technology and the discovery of potentially transformative next generation integrates technology engineered to enable large scale genome editing. Speaker 200:07:41In epigenetic regulation, we have shared advance in zinc finger activators and repressors for the potential treatment of a remarkable range of neurological diseases such as prion disease, thiopathies, Charcot Marie Tooth diseases type 1a and 2a, Dravet syndrome, SOD1 mediated amitrophic lateral sclerosis or ALS, Phelan McDermott syndrome, Parkinson's disease, Angelman syndrome and many other neurological disorders. We have also demonstrated Sangamo's potent delivery capabilities developed through our AAV capsid engineering platform, sifter. Here we presented additional STACK BBB findings including prion and tau repression data achieved via STACK BBB delivery. We also presented for the first time exciting initial findings for a possible mechanism supporting how STACK BBB may cross the blood brain barrier. Our sifter platform is designed to engineer capsid for various routes of administration such as intrathecal and intravenous delivery. Speaker 200:08:50Sifter can also engineer capsids for in vitro discovery. So in our platform presentation tomorrow, we will present findings from STK-one hundred and fifty, a novel capsid that's been shown to be highly potent in neurons and enables high throughput screening of neurology focused transcriptional regulators. We anticipate the STAG-one hundred and fifty, which we believe manufacturers easily at small scale will help accelerate the discovery of potent and highly specific epigenetic regulators. Finally, building on our deep expertise in that protein DNA interactions derived from our zinc finger platform, we presented for the first time a potentially breakthrough new approach for integrating large sequences of DNA into the genome to potentially treat with a single medicine patients who have unique mutations in the same gene. Precisely integrating large synthetic DNA constructs into a desirable chromosomal site has been the dream for people working in this field for many years. Speaker 200:09:56Our modular integrase or MINT platform is a versatile protein guided genome editing method that understands and engineers BXB1, a serum, serine recombinase to insert or replace entire genes and adds to Sangamo's toolbox of editing capabilities. We are hopeful that our MINT platform could be used to correct many disease causing mutations in a diverse patient population by inserting a correct copy of the gene into its natural locus. MINDS could be deployed internally for various neurological indications, but also provide potential partnering opportunities both for human disease and in agricultural biotech settings. We are already in active discussions with potential partners about our integrase capabilities and are hopeful that Mint could provide us with another potential non dilutive funding opportunity. Alongside our presentations in this topic at ASGCT this week, we've also published a manuscript in bio archive further outlining these next generation integrase advancements, which is also available on the publications page of the website. Speaker 200:11:13I encourage you to learn more about this exciting development in the field of genomic medicines. Now looking at our clinical programs, we have made strong advances in the Q1 for our Fabry disease program, having dosed the final patient in the Phase III STAR study of Isoraleogene cevaparvek, our investigational gene therapy for the treatment of Fabry disease. With 33 patients now dosed, screening, enrollment and dosing are complete. One additional patient has been able to stop enzyme replacement therapy or ERT resulting in a total of 14 patients withdrawn from ERT to date. The 4 remaining patients dosed since February 2024, who began the study on ERT already have plans in place to withdraw ER treatment at the appropriate time. Speaker 200:12:05At the 20th Annual World Symposium in February, we presented compelling updated preliminary clinical data from the STAR study showing sustained benefit and a differentiated safety profile. These results underscore the program's potential as a single administration treatment for Fabry disease. As a reminder, this quarter we announced alignment with the U. S. FDA on an abbreviated pathway to potential approval. Speaker 200:12:34We've also been granted prime eligibility by the European Medicines Agency and iLab designation by the UK Medicine and Healthcare Products Regulatory Agency. We are engaged in active discussions with potential collaborator partners for our Fabry disease program and continue to defer additional investments in planning for a potential registrational trial until a collaboration partnership or financing for this program is secured. Moving to our partner clinical program, we look forward to the pivotal readout expected in mid-twenty 24 in the Phase 3 AFFINE trial of gyratocogine, fitoparvovec, an investigator gene therapy we're developing with Pfizer for patients with modestly severe to severe hemophilia A. A. Pfizer anticipates submitting a biologics license application and a marketing authorization application in early 2025 of the pivotal readout is supportive. Speaker 200:13:40As a reminder, we are eligible to earn up to $220,000,000 in milestone payments and up to 14% to 20% royalties on potential sales from this program. We end the quarter with approximately $54,000,000 in available cash and cash equivalents, which includes funds from the aforementioned registered direct offering. We believe these resources in combination with this cost savings expected from the recent restructurings, workforce reduction and other potential cost reductions will be sufficient to fund our planned operations into the Q3 of 2024. As I outlined earlier, we are actively pursuing a range of different options to raise important additional capital and are encouraged by the business development discussions ongoing across our Fabry program, capsid engineering and next generation genome engineering efforts. We believe our company has the science required to potentially transform the lives of patients living with devastating neurological conditions and are committed to raising the funding required as we seek to make our vision a reality. Speaker 200:14:55Operator, please open the lines for questions. Operator00:14:59Thank you. At this time, we will conduct a question and answer session. Our first question comes from the line of James Stamos at Jefferies. James, your line is open. Speaker 300:15:26Hi, this is James on for Maury Greatcross. Congrats on the progress and thanks for taking our question. I just wanted to ask, where are you with preclinical and GLP tox studies with STACK BBB? And what are your latest thoughts on which targets or indications would make the most sense to partner or keep in house? Speaker 200:15:43Thank you for your question. We have made one of the reasons that we like STACK BBB so much is that our manufacturing team have been able to work with it and found that it is very manufacturable. They can take it so far up to 50 liter scale and are going higher that they can use the same purification and assay techniques and that it will that it feels like a capsid that will be able to give us great yield and also be able to use many of the processes in CMC work that really is underappreciated in the importance of capsid selection. We've said publicly that we are moving ahead with tau and with Prion. You could imagine the amount of external interest in tau, not just because it's a high unmet medical need, but because the capsid gives you the brain penetration and widespread delivery that's needed. Speaker 200:16:47And our zinc finger repressors are really unique in being able to almost switch off tau in the cells that they can get to. We are having many discussions with companies about our capsid and we'll choose wisely, I believe, which ones we partner and which ones we take forward ourselves. What we understand and I want to say this again and repeat what I said in the chosen words is that we understand the need to bring in money to the company. And that's always a balance between the desire that we have to take things forward and the money that's available from many of the partnerships I'm sure we will achieve in the coming weeks. Speaker 300:17:33Great. That's very helpful. And just to go on to like some of the more specialized parts of your ASGCT presentation, can you talk about the importance of targeting the PoNS and motor neurons in Alzheimer's? And are there any other companies that you're aware of that have the ability to target these sites? Speaker 200:17:49I think thank you for your question. It's really been a remarkable ASGCT and the field moves closer to these tools being able to address such important disease. Amy, can you talk a little about this? I know this is one of your passions. Speaker 400:18:07Yes, I would love to. Thank you for the great question. And what's really important here is being able to target brain regions that would be traditionally challenging to target using something like a direct injection approach. The PoNS being part of the brain stem is not a region that you would necessarily want to reach using a direct injection guided approach even for the most severe of diseases. So we were so excited to see the biodistribution that was able to be achieved with the SacBB capsid, especially for what it means for the tau program. Speaker 400:18:37The PoNS is critical in progressive supranuclear palsy, but also other tauopathies like Alzheimer's disease. In addition, as you noticed, the motor cortex, which is part of the frontal cortex, we use this as an example of how STACK PBB is able to transduce those cortical neurons. Really, we believe that this kind of widespread brain targeting is critical for halting or slowing the progression of the disease. Speaker 200:19:03Thanks, Amy. Speaker 300:19:05Great. Thank you for taking our questions. I'll hop back in the queue. Operator00:19:08Thank you very much. One moment for our next question, please. Our next question comes from Gena Wang of Barclays. Gena, your line is open. Speaker 500:19:20Thank you for taking my questions. I have 2. The first one also is about the SPECT BBB and it seems like very exciting new capsid. What is the highest dose you used in non human primates and what is the limiting dose toxicity there? And then the second question quickly regarding the Heme, I know it's your partner Pfizer's is in charge, but just wondering for the mid-twenty four Phase 3 update, would that be just a press release from Pfizer? Speaker 500:19:51And then for the 220 milestone payment, the first milestone, is that the drug approval? And is that the big portion of the $220,000,000 Speaker 200:20:04Gina, thank you for your questions. And I know you've been following Himi for a great deal of time. So it's good to be coming closer to the point where it gets to registration and approval. So let me do the let me pass this on to 2 people. Amy, can you talk about the dose that we've used and whether there was any toxicity, please? Speaker 400:20:31Yes, absolutely. And we as part of the SYKBP study that we discussed yesterday and we also will discuss later today at the poster session on ASGCT, we performed a dose range finding study with our tau and zinc clinical lead zinc finger and we did 3 different doses and the top dose was 1e14. We're really happy to see that with the histopathology results that we didn't see any dose limiting toxicity and really no findings in the brain, the spinal cord or the liver, these peripheral organs. So really positive and encouraging data. Speaker 200:21:06Gina, does that answer your Speaker 500:21:10Yes. Like more thinking, other than the dose finding, like what is the highest dose you tested for the safety toxicology perspective? Speaker 200:21:201e14. So 1e14. Speaker 500:21:22That's 1e14. Okay. Speaker 200:21:24It's the highest we've so we went there in the efficacy study that we just reported and we really saw nothing sorry, we saw nothing that would give us any concern. When we will now go into the GMP tox study, which is the only thing remaining to complete the IND enabling studies for that. But we will clearly start at a lower dose in the clinical study because that's the right thing to do and that's what the agency will ask us to do. But thus far, we've it's a very clean capsid, which is great, great news. And then can we talk about humane, Natalie? Speaker 200:22:05I know you've been speaking to your friends at Pfizer regularly. Speaker 600:22:09Sure, sure. Yes, we're very excited that the mid-twenty 24 pivotal readout in MA is coming up soon. We the partnership allows for $220,000,000 in potential milestone and 14% to 20% royalty on potential sale. But the specific amount of each milestone is not publicly disclosed. But this could start as early as the start of 2025 if the pivotal readout is positive and if Pfizer elect to see regulatory approval to the program. Speaker 200:22:43And Gina, just to reflect on that, we're limited in what we can say. When you sign these deals, there's always an agreement about who talks about what. But the regulatory milestones, which are we get one for the U. S, one for Europe and one for Japan are not for approval, they're for submission of the package. And then the commercial milestones, which are for 1st patient dosed rather than for reaching a certain sales threshold. Speaker 200:23:23So they're very, very favorable milestones that will fund the company in 2025 and 2026. Speaker 100:23:32Very helpful. Speaker 600:23:32Yes. I need to mention that the significant portion of the €220,000,000 is near term and we could also choose to monetize them now. Speaker 200:23:42Yes. So the closer so Gina, the closer we get to the when the data is revealed and then when Pfizer submits this, we have the option of monetizing those royalties. But we can also choose to wait and use them to fund 202526. And what will make that decision is the non dilutive funding from business development that we do now will tell us whether we need to make that decision. We are I know that there's a concern from people that followed us for a long time about our near term cash runway. Speaker 200:24:20We feel that we are fortunate to be able to do both capsid deals, fabry deals, technology deals and to have this large amount of cash next year that we can bring forward if necessary. Speaker 500:24:37Thank you very much. Speaker 200:24:39Thank you, Tina. Operator00:24:40Thank you very much. One moment for our next question please. Our next question comes from Patrick Trucchio of H. C. Wainwright. Operator00:24:49Patrick, your line is open. Speaker 700:24:52Hi, everyone. This is Luis Santos for Patrick. Congratulations on the recent progress and your presence your impressive presence at ASCCT. I am interested in knowing if you already guided to the tau program and if it's going to be in Alzheimer's, do you know which patients you're going to be treating and which other indications you are going to push forward here with tau program? And then I have a follow-up question. Speaker 200:25:26So thank you for your question. We haven't guided on that. There's been an enormous amount of interest in our presentation yesterday at ASGCT. It clearly was, I think, the most remarkable presentation of all of the many people that are moving forward in blood brain barrier penetrant capsids. We feel that the tau asset, the cargo that's within it, doubles down, makes us even more interesting. Speaker 200:25:59The role of tau in Alzheimer's, I think, is becoming increasingly clear. The Biogen data is very encouraging. The idea that it's the thing that is relevant to the clinical stages of tau rather of Alzheimer's rather than in the early pre symptomatic ones makes this a much more easy lift. However, we are very conscious that Alzheimer's studies are large programs and that we need to make sure that we either do it ourselves well or partner with someone who has a real expertise in Alzheimer's. Speaker 700:26:41On the follow-up thank you. That was helpful. On the follow-up on the partnerships, but platform, you mentioned that there are business development opportunities there. Do you have any potential collaborators in mind? And what diseases would you be targeting? Speaker 200:27:05The mint integrates is remarkable. The presentation at ASGCT was just 57 minutes ago and there was a lot of excitement in the room about it. We have already been we've been speaking to people for 3 months on this. And I want to put the business development discussions into perspective. We had the regulatory clarity for Fabry just in February. Speaker 200:27:34We had the capsid results just in March. We've finally taken Mint to a place that we feel we can share it in May. So I understand the desire for signed deals, but the results are going to lead to them have only come out over the past 2 or 3 months, which is means that at the moment you could imagine our business development group is incredibly busy in talking to lots of people about this. Now integrase is something that has the potential for really disruptive genomic engineering. It's the kind of thing that David Lou, when he was asked, said was his dream of what the ideal genomic medicine was, which was an integrase that could be engineered to go to your site of choice and put in pieces of gene size pieces of DNA that would replace all of the mutations downstream. Speaker 200:28:39And the team at Sangamo because of their expertise in understanding the interaction between proteins and DNA from the Zinc Finger platform have been able to do this remarkable engineering and the whole alpha fold and understanding artificial intelligence of how you understand proteins has helped us. We feel that this is something that Sangamo can use, but it also is has attracted great interest from the hardcore science pharma companies that you could imagine who all see this as the future of genomic engineering. And so we have a responsibility to put it in as many people's hands as possible. I think you will hear more about this Mint platform for some time to come. Speaker 700:29:32Thank you. Operator00:29:33Thank you very much. One moment for our next question please. Our next question comes from the line of Luca Yeezy at RBC. Luca, your line is open. Speaker 800:29:46Hello, great. Thanks for taking our questions. This is Lisa on for Luca. I just want to ask another question on the Mint technology. Just wondering if you can give us a sense of what kind of targets here would be good candidates. Speaker 800:30:05For instance, would it be possible to integrate something like a full length CFTR gene, which is around roughly 190 kilobases? Or should we think about something larger like full length dystrophin, which is measuring closer to over 2,000 kilobases? Just any color here would be helpful. Thanks. Speaker 100:30:28So Speaker 200:30:31you asked really important and interesting questions. And as always with Sangamo, thought about size, because as you know, zinc fingers are an 8th of the size of CRISPR and even the small mammoth CRISPRs are 4 times as big as the zinc finger. And that's important because of delivery. It's the same with Mint, because we try to keep the technology as small as possible. So as we could put both the integrase and a cargo in an AAV. Speaker 200:31:11Now when you get to the size of some of the genes that you're talking about, you end it comes down to what kind of delivery you're using. My dream for Mint is to be able to drop a cDNA copy of the gene or of the exons that are mutated into intron 1 of the genome. It would then be run off of the promoter, have all the locus control elements and it would alleviate the requirement to edit all the individual mutations that usually are the cause of genomic disease. Now to put in a whole genomic copies would be unnecessary, I would argue. And then we need to look at what are the diseases where the cDNA is packageable into the delivery mechanisms that are available because the mint should be size agnostic and has so far shown great potential. Speaker 200:32:21Amy, is there anything else Amy? I'm aware that you're on the line and more and as our Head of Research, perhaps you have other things to add to that. Speaker 400:32:31No, that's fantastic explanation, Sandy. I would just add that there are specific genes as well that are involved in some neurological disorders, and I'm thinking of things like Rett syndrome or MECP2 needs to be fine tuned at a very specific level, something like an integrase where you could integrate in perhaps the healthy copy of exon 1 or even a bigger portion of the gene, so that it's under control of the endogenous promoter could be really transformative for a disease like that. So I think it's incredibly exciting to see what are the opportunities around this new MYNT platform. Speaker 800:33:07Thanks so much. Thanks for taking my questions. Operator00:33:10Thank you very much. One moment for our next question. Our next question comes from Ritu Baral of TD Cowen. Ritu, your line is open. Speaker 900:33:23Hi, guys. Just a quick question on the Fabry program actually. You've just 33 patients and you saw the data at World Plus, you have the Phase 3 path going forward agreed upon with FDA. What is the next data update that we're going to get from that? And also, I guess, is there a data point or a follow-up point in a subset of patients that's gating to potential business development around or out licensing of this program? Speaker 900:33:56Thanks. Speaker 200:33:58Thank you, Ritu. I'm going to pass this on to Natalie. But I'll just remind you again, the whole business development conversation around Fabry changed with Peter's new way of thinking about this at the agency and the conversations that we had in February. And so it's like a new conversation we're having with potential partners. But Natalie, can you address some of the technical pieces, please? Speaker 600:34:25Yes, sure. So we've completed the dosing of all the patient in the Phase III. So for a total of 33 patients, We continue to analyze the data, but this the body of data that we have thus far is not gate limiting for any partnership and any of the Phase IIb preparation or registrational trial preparation. So we continue to analyze the data, but we and I've agreed with the FDA that we have enough data to move on to a Phase 2b. And that's what we're discussing with partners, potential partners. Speaker 900:35:05Sandy, when you say that the discussion has changed, are you talking about the structure of the deal that is in Sangamo's best interest? Like is it do you think it could be like a COCO going forward? Or is it just really sort of valuation terms that are different now? Speaker 200:35:24I think it's much simpler than that, Ritu. There was a concern that there wasn't clarity about what the regulators were wanting. And there was this worry that we've all talked about that it was going to be a head to head against ERT. And the agency now saying, no, it's a simple single study and that we can they've named the number of patients and they've said we can come back halfway through the study and show them the data we've got if we feel it's compelling. I think that has completely changed how the various companies are going to. Speaker 200:36:08And I think great credit to Peter. He is walking the talk in this. And we have benefited greatly from it as being almost like the poster child for what it is approval of rare disease gene therapy is. And if I may, Rita, we've started the we've initiated the conversations with the Europeans and uniquely the FDA is volunteering to send one of their staff with us to that conversation because they feel that it's the way forward for these kind of therapies is for a common approach to them. And I think it reflects the support that we have from the FDA in these discussions. Speaker 900:37:11Thank you, Sandy. Operator00:37:13Thank you very much. Our next question comes from the line of Nicole Garmino of Truist. Nicole, your line is open. Speaker 1000:37:21Hi, this is Bill on for Nicole. We have a question. Speaker 1100:37:25It's given the uptake for ROCKTAVIAN, what is Speaker 1200:37:28the read through to potential uptake for Fabry disease gene therapy? Speaker 200:37:35So thank you for your question. So the Roktavian launch I know has been a subject of great debate. We can't comment on how BioMarin launched it. All I know is the reason that we chose Pfizer to be our partner is I have great confidence that when Pfizer chooses to launch something, they do it well and effectively. And that follows through to our choice of partners for Fabry disease. Speaker 200:38:05There are several companies and there are some that are more obvious as being great commercialization engines and great launch companies. What I can tell you is in our conversations with these companies, we have had notes from the patient support groups who have been very keen to make sure that the pharma partners understand how supportive they are of the results that they have seen in our Fabry program. Because you and I will talk about the biochemistry and the biopsies. What the What the patients have said is how much better they feel on it, how delighted that they are to be off of ERT. So now we have 14 patients off of ERT and the 4 that are remaining on ERT in the study have already booked when they're going to come off of the therapy. Speaker 200:39:01So this has enormous support from the patients. And I think that is what is going to make it a very successful launch for the partner that can take it forward and make a difference. Speaker 600:39:17And if I may add, we also have RMAT and PRIME, which are designation that really analyze an unmet medical need for Fabry. So the Fabry patient, even though can have access to ART, are not satisfied with the current medicine standard of care. Speaker 1200:39:35Thank you so much. Operator00:39:38Thank you very much. At this time, I'm showing no further questions. And I would now like to turn the call back to Louise Wilkie for closing remarks. Speaker 100:39:48Thank you very much, and thanks to everyone for joining us on the call today. We look forward to speaking to you soon.Read morePowered by