Sangamo Therapeutics Q1 2025 Earnings Call Transcript

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Provided by Quartr
May 12, 2025

There are 10 speakers on the call.

Operator

Good afternoon, and welcome to this Agamemos Therapeutics First Quarter twenty twenty five Teleconference Call. Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker today, Louise Wilkie, Vice President of Investor Relations and Corporate Communications. Please go ahead.

Speaker 1

Thank 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, Patricia Durobabu, chief financial officer, and Natalie Dubrostringfellow, Chief Development Officer. Slides from our corporate presentation can be found on our website, sangamo.com, and under the Presentations page of the Investors and Media section.

Speaker 1

This call includes forward looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements relating to Sangamo's cash runway and operating expense guidance the anticipated closing of the announced underwritten offering Sangamo's plans to obtain additional capital and its ability to continue to operate as a going concern the therapeutic and commercial potential and value of Sangamo's product candidates and technologies, Sangamo's ability to earn and receive payments from its collaboration and license agreements, Sangamo's ability to establish and maintain collaborations and strategic partnerships, including for its Fabry disease program, the anticipated plans and timelines of Sangamo and its collaborators for clinical trials, clinical data presentations and releases, regulatory submissions and regulatory approvals, upcoming catalysts and milestones and other statements that are not historical fact. 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 12/31/2024, and our quarterly report on Form 10 Q for the fiscal quarter ended 03/31/2025, and subsequent filings and reports that Sangamo makes from time to time with the SEC.

Speaker 1

The forward looking statements dated today are made as of today, and we undertake no duty to update such information except as required by law. Please 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 to our CEO, Sandy Macrae.

Speaker 2

Thank you, Louise, and good afternoon to everyone joining the call. It's only been a couple of months since our fourth quarter call, but I'm pleased to share with you some company progress across a variety of areas, including our capsid engineering platform, our neurology pipeline, our Fabry programme and our finances. Beginning with our capsid engineering platform, in April we announced our third capsid license agreement since we shared the discovery of our industry leading neurotropic delivery capsid, Stack BBB. We are pleased to sign an agreement with Eli Lilly and Company granting Lilly a worldwide exclusive license to Stack BBB for up to five potential disease targets of the central nervous system. We have received the $18,000,000 upfront license fee for the first target and are eligible to earn up to $1,400,000,000 in additional license target fees and milestone payments across all five potential disease targets as well as tiered royalties on potential net sales.

Speaker 2

We are thrilled to have signed this third important agreement further demonstrating that we are a collaborator of choice for neurotropic capsids. With Genentech, Astellas, and now Lilly, we have great partners in neuroscience for our technology, and we continue to engage in discussions with new potential collaborators for Snac BBB. Turning to our neurology pipeline programmes, this quarter we continued to advance clinical study preparations for ST-five zero three, our investigational epigenetic regulator for the treatment of chronic neuropathic pain. We are preparing for a Phase onetwo study to assess the safety, tolerability and preliminary efficacy of a one time dose of ST503, our investigational epigenetic regulator, that will be administered intrathecally to patients with intractable pain due to idiopathic small fiber neuropathy or ISFN. We plan to begin patient enrollment and dosing for the ST-five zero three study in mid-twenty twenty five and anticipate having preliminary proof of efficacy data in the fourth quarter of twenty twenty six.

Speaker 2

We also continue to advance clinical trial authorization or CTA enabling activities for ST506, our epigenetic regulator for the treatment of prion disease, to be delivered intravenously using our own STAT BBB. We're extremely proud to have been selected to present during the prestigious Presidential Symposium at this week's ASGCT Annual Meeting in New Orleans. We look forward to showcasing our potent combination of epigenetic regulation and capsid delivery technology in prion disease and describing the profound survival benefits we observed when administered to post symptomatic mice. We will also describe the sustained brain wide suppression of prion protein expression in both mouse and non human primate models, supporting its potential as a one time therapeutic approach for prion disease. We plan to begin clinical trial enrollment and dosing for ST-five zero six in mid-twenty twenty six and expect to have preliminary clinical data in the fourth quarter of twenty twenty six.

Speaker 2

In addition to the PRION presentation, we have had eight abstracts accepted by ASGCT. I am very proud of our scientists and look forward to us showcasing the progression of our neurology pipeline, including advances in zinc finger epigenetic regulation, the latest innovations in capsid delivery engineering, and developments in our modular integrase technology. Moving to our late stage Fabry programme, last week we were excited to announce a number of important de risking milestones in the pathway to the anticipated BLA submission for ST920. All dosed patients in the Phase onetwo STAR study have now completed at least fifty two weeks of follow-up, a key milestone required by the FDA for an accelerated approval regulatory pathway for ST920. Importantly, preliminary analysis of the clinical data collected as of this fifty two week milestone date across all 32 dose patients indicated that the mean eGFR slope continued to remain positive.

Speaker 2

The product candidate continues to be well tolerated, and a pivotal data readout is now expected by the end of this quarter. Furthermore, in April of this year, Sangamo held a productive Type B meeting with the FDA, providing us with a clear chemistry, manufacturing and controls, or CMC, pathway to the planned BLA submission. We were encouraged by the productive nature of the discussions and engagement from those FDA representatives in attendance and are happy to have clarity on these important activities from the agency. With the newly agreed CMC pathway, we believe we have a clear line of sight to an anticipated BLA submission as early as the first quarter of twenty twenty six, which would facilitate a potential approval and commercial launch of ST920 as early as the second half of that year. As you can imagine, this clinical and regulatory progress has been well received by our potential commercial partners, and we are hopeful that these de risking events may accelerate our ongoing negotiations.

Speaker 2

We remain committed to securing an anticipated partnership that is best suited to bring SC920 to Fabry patients upon potential approval, and that provides near term capital for Sangamo to advance our core neurology pipeline. As I've said before, in order to execute on our plans and deliver on this promising neurology genomic medicine pipeline, Sangamo must be sufficiently capitalised. We must have the resources to fund us through proof of concept in both our chronic neuropathic pain and prion disease programmes while operating a lean, efficient and focused organisation. In support of this strategy, today we announced the pricing of an equity offering to extend our immediate cash runway. We're optimistic these funds will provide us with a bridge that we believe is necessary to secure a Fabry commercialisation agreement.

Speaker 2

We believe that this modest infusion of equity capital alongside the recent positive Fabry clinical and regulatory derisking events will allow us to secure the right commercial partner for the company and for our shareholders. We also continue to engage in promising business development discussions across our technology platforms. We have opportunities for potential funding through both new and existing Stack BBB collaboration partners with our zinc finger platform and through collaborative research agreements related to our modular integrase, or MINT platform, all of which demonstrate the ongoing interest in our technology. We look forward to sharing more information when we can. With that overview of our business progress and strategy, I'd now like to hand it over to Prathyusha Durabhabhu, our chief financial officer, who'll provide more context on long term vision and financial strategy to support our neurology focused mission.

Speaker 2

Prathusha?

Speaker 3

Thank you, Sandy. I'd like to begin by emphasizing our long term vision for Sangamo as a neurology focused genomic medicine company. We strongly believe in the potential of our neurology pipeline to deliver transformational therapies for patients as well as significant value for our shareholders. Our goal is to appropriately fund the company to achieve clinical proof of concept data across both our chronic neuropathic pain and prion disease programs. These milestones represent critical potential value inflection points that could significantly change the trajectory of our company.

Speaker 3

To achieve these important milestones, we have a financial strategy with both short and long term components. In the near term, the equity financing we announced today is necessary to solidify SinoMo's immediate financing needs. We believe the offering proceeds will extend our runway to late in the third quarter of this year. More importantly, this bridge financing provides us the time we believe that we need to secure the right Fabry commercial partnership, one that would fund our neurology mission in the near term and create value for shareholders over the longer term. We've already made significant progress in transforming our financial profile.

Speaker 3

In 2024, we reduced our non GAAP operating expenses by 50% year on year by carefully focusing the organization on our most important priorities. We are leaving no stone unturned in seeking additional cost savings and are looking at ways to further reduce operating expenses to maximize the efficiency of the go forward neurology company. We are committed to operating as leanly as possible while continuing to advance our two neurology programs at a steady pace. We believe this disciplined approach to capital allocation will ensure that we have the specific talent and capabilities to execute on our neurology mission while extending our runway through our key value creating milestones. I'll now hand it back to Sandy for closing remarks.

Speaker 2

Thank you, Pratusha. I'm pleased with the progress we have made this year so far. We signed our third StackBVB license agreement, this time with Lilly, reinforcing that Sangamo is a collaborator of choice for neurotropic capsids. We continue to advance our ST-five zero three program for the treatment of intractable pain due to ISFN, ahead of the start of a planned patient enrollment and dosing in mid twenty twenty five. We achieved significant clinical and CMC de risking milestones in our pathway to anticipated BLA submission for our Fabry disease programme.

Speaker 2

And we have advanced multiple potential business development discussions across a range of Sangamo technologies, including StackVBV, our ZincFinger platform and our Mint platform, while securing additional capital to support our near term efforts. We remain resolutely focused on solving our long term funding needs and partnering our Fabry disease programme in an effort to provide sustainable and long term funding for our promising neurology genomic medicine pipeline. In closing, I would encourage you to engage with the data we will be presenting at this week's ASGCT Annual Meeting, which will be made available on the Sangamo website once data embargoes have lifted. These presentations will further demonstrate the scientific foundation of our pipeline and the potential of our therapeutic approach to address significant unmet medical needs. As always, we thank you for your continued support of Sangamo.

Speaker 2

Operator, please open the line for questions.

Operator

Thank you. As a reminder, to ask a question, please press 11 on your telephone and wait for your name to be announced.

Speaker 4

Our first question is going to come from the line of Murray Raycroft with Jefferies. Your line

Operator

is open. Please go ahead.

Speaker 5

Hi. This is James on for Maury. Congrats on the progress, and thanks for, taking our questions. Just to start off, could you provide more color on what exactly you plan to show in the top line eGFR data? Do you plan to show just the eGFR slope or the other quality of life endpoints?

Speaker 5

Also, do you plan to show an NHS analysis to contextualize the improvements you're seeing in eGFR? And if so, can you provide some color on the statistical plan and p value threshold?

Speaker 2

Thank you for your questions. Natalie, can you talk to these?

Speaker 6

Yes. Yes. So we're really happy to have those all the patients are now have passed the one year milestone required by the FDA for an accelerated approval regulated pathway. We will, of course, share in the top line data the updated mean eGFR slope, and we will comment on additional information at a later date.

Speaker 2

It's fair to say, Natalie, that at that time, we'll also have 19 patients who will have achieved two years data. Absolutely. So it really is a robust dataset. We understand the excitement people have to see this data in full, and we will show as much as possible as we can of the top line data as we peer prepare it for the BLA submission.

Speaker 6

Yes. And as as to the statistical analysis, we are not commenting at this time, but we have

Speaker 2

We've come with the

Speaker 6

agree with the FDA on our type b meeting with them. So we're you know, we feel like we it's a standard way to doing it, and I've agreed with the FDA on the passport.

Speaker 5

Got it. Thanks. And just one really quick follow-up. Now that you're you're sort of in the final stages of of securing a potential Stabri partnership, how many partners are are you potential partners are you currently in conversations? And and what what can you really tell us about those ongoing discussions at this point?

Speaker 2

You can imagine that that's not something that we're able to comment, but, it's fair to say there are multiple, potential partners that we're talking to. And I think for all of them, the type b meeting was very helpful because it gave a clear path for the CMC, which is something that is very important for any gene therapy BLA and approval. So we were very pleased with that result. We're very pleased with how straightforward the interaction with the agency was and how helpful they were, because I know for people like you predictability of the process and the approval process is very important.

Speaker 5

Got it. Thank you so much for taking our questions. I'll hop back in the queue.

Operator

Thank you. One moment for our next question. Our next question is going

Speaker 4

to come from the line of Nicole Girmino with Truist.

Operator

Your line is open. Please go ahead.

Speaker 7

Hi. Good afternoon. Thanks for taking my question. So you've previously mentioned potential interested partners for Fabry and Hume. Has the pace of those conversations gone have they changed in any way given the macro landscape with changes at at FDA?

Speaker 7

And do you see any, added pressure around cell and gene therapies in particular?

Speaker 2

So, Nicole, I sorry. Your line was quite hard to hear here. I think I heard you ask us to comment on hemophilia A and the agency's attitude to gene therapy. Is that fair?

Speaker 7

Yes. And Fabry disease.

Speaker 2

So as as I as I kind of alluded to in the last answer, the remarkable thing about the agency interaction we just went through was how unremarkable it was and how helpful the agency was. We they send you answers to your written questions before the meeting. They came in on time, and they were very detailed. They had the all the right people at the most senior level in the meeting, and we got the answers, the formal minutes of the meeting within a week of the meeting where they could take up to thirty days. And so in every form of interaction we've had, we have found them unchanged in how they are looking at our Fabry disease program.

Speaker 2

And when we look at the interactions that Pfizer had across the heme, they also seem to have been very positive about that. So we are not seeing any change. I'm sure, like us, you are very aware of of the the announcements and and the concern about funding and about resourcing at the agency, but we've not seen any evidence of that, and it has not impacted anything to do with our program.

Speaker 7

Okay. And then one quick question and follow-up. So given the current administration's concern around drug pricing, how do you think that will impact gene therapy uptake and pricing in US and and ex US?

Speaker 2

I think there's a long way to go in those discussions around drug pricing. I think that the pharmaceutical industry is a very important industry in in America, and I'm sure as someone who sits on the board of Bio that Bio will be having very detailed and hopefully productive conversations with the administration to find a good way through this.

Speaker 7

Great. Thank you so much, Sandy.

Operator

Thank you. And one moment for our next question. Our next question is gonna come from the line of Yanan Zhu with Wells Fargo. Your line is open. Please go ahead.

Speaker 4

Hi. Thanks for taking our question. This is Kwan on for Yana. Just to clarify on the prior questions. On the regular path for Fabry, do you still plan to file based on fifty two week eGFR data?

Speaker 4

And is static versus baseline required for eGFR? Thank you.

Speaker 3

Yes. Absolutely. We're

Speaker 6

we are absolutely pursuing the the agreement we had with the FDA to use fifty two weeks eGFR for the entirety of the patient population in the phase one two study. We have collected all the data from, the 32 patients, and we are QCing it. So we expect to, share the top line data at the end of the quarter, and then this will be the basis for, the data in our BLA.

Speaker 2

And it's very important that we emphasize how much supportive data there is beyond the eGFR. We have got agreement with the agency for submission based on that as the primary endpoint. But I think the thing that convinces the agency and has made this such a compelling medicine for us is that every other indication index of success in trial is going in the same direction. So pain scores, Fabry scores, SF 36.

Speaker 6

Yeah. The and the general safety is very good, very well tolerated. The patient in general are feeling better, and our alpha gal level remain high in all patients.

Speaker 2

And and some of these patients are now out to four plus years?

Speaker 6

Four plus years. Yeah. Yep.

Speaker 4

Sorry. Maybe you already comment on it. And is static versus baseline required for EGFR? Thank you.

Speaker 2

We'll be looking at a variety of statistical methods to describe the difference in eGFR. Nope. But we said, last week or the week before in in our announcement that the eGFR remains positive. So the slope of the EGR remains positive.

Speaker 4

Got it. Thank you so much.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Luis Santos with, H. C. Wainwright.

Operator

Your line is open. Please go ahead.

Speaker 8

Hello, everyone. Thank you for taking our questions, and congratulations on the selection of your talk for the presidential symposium and your impressive presence at ASGCT. Regarding Fabry, I don't know if I missed this, but are the patients who were on ERT still all off ERT, or were there any were there any relapse?

Speaker 6

Yes. All patient the eighteen patient that started on ERT out of our thirty two patient are of still of ERT.

Speaker 8

Very well. And for the I I know that you mentioned you you're not commenting, so much on the statistical analysis plan, but the eGFR slope, you said it remains positive. But is there any threshold that, we should be looking at as a minimum, or is there any actual statistical significance? I think prior questions were asking about this already, but, I'm not sure if I was clear on that.

Speaker 6

We're not commenting on this right now, but, what I can tell you is the mean EGFR slope remains positive.

Speaker 2

And and you will I know you're very aware that in normal people, sorry, people without Fabry disease, there's a gradual decline over time in their eGFR. In Fabry patients, there is an exaggerated decline in eGFR that is not fully addressed by, ERT and it remains negative in patients that are on ERT. And so as part of our analysis, we'll be looking at compare look at the change from baseline and we'll be looking at compare to the data for other forms of treatment for Fabry disease. We would not have put out the press release, that we did about the ER, the eGFR remains positive if we weren't confident that the results were what the agency were looking for.

Speaker 8

And, that's helpful. Just wrap up that and clarify my question as well for, the statistical analysis plan, even if you're not giving us more detail, you are submitting, all the data available from all patients for the BLA submission. But which ones will be used for the, which ones will be used for the statistical analysis plan? Is it all of them, or is it just a portion of them?

Speaker 6

It's all of them.

Speaker 2

So the submission for the BLA submission, it will be 32 patients at one year and 19 patients at two years. That's helpful. Thanks so much. My pleasure.

Operator

Thank you. One moment for our next question. Our next question is going to come from the line of Luca Issy with RBC Capital Markets. Your line is open. Please go ahead.

Speaker 9

Hi. Thanks so much for taking our question. This is Cassie on for Luca, and this will be a question on StackDBB. And we recall that homology medicine had one of the first capsids delivered systematically, which could cross the blood brain barrier and demonstrated in a mouse model a dose dependent reduction in biomarker both systematically and in the CNS. So would you expect to see a similar dual impact with your stacked DVB capsid?

Speaker 9

And, also, do you expect similar preconditioning to your other capsid approaches, I. E, no prophylactic steroids? Any color there would be very helpful. Thank you so much.

Speaker 2

So, we can really only comment on on our own capsid and its effectiveness. We have not made the decision whether we will use steroid treatment in the clinical trial yet. We're very pleased with the effectiveness of STAT BBB. We're very pleased to have seen the result in monkeys across with a variety of cargoes. And I would really commend you all to look at the result look at the presentation that Brian will be giving at, ASGCT where it really showcases both the effectiveness of stacked BBB and the importance of the right cargo in it.

Speaker 2

And to be honest, that's why it's so pleasing that Lily, Astellas, and Genentech, who are all big names in the neuroscience field, have chosen to license our capsid directly.

Speaker 6

Yes. And what we will present really is profound of benefit of the treatment in disease mouse model and sustained brain wide suppression of prion protein expression in both mouse and nonhuman pride model, supporting its potential as a onetime therapeutic approach for prion disease. So it's very important that we have well we have tested it in NHP, and it's extremely well tolerated.

Speaker 2

And, you know, the the presidential symposium, I think there's two presentations being given out of something like 4,000 abstracts that were presented, and it's a great, it's a great credit to the work of, Brian Zeitler and David Agellan, Victoria and Brian's team that have all done so much work to drive something that hopefully next year will be a potential curative sorry, a potential treatment for patients with this awful, awful disease.

Speaker 9

Okay. Great. We'll be looking forward to the presentation at CTG. Thanks again, and congrats on the presentation and abstracts.

Operator

Thank you. Our next question comes from the line of Gena Wang with Barclays. Your line is open. Please go ahead. Miss Wang, your line might be on mute.

Operator

I am showing no further questions at this time, and I would like to hand the conference back over to Louise Wilkie for closing remarks.

Speaker 1

Thank you once again for joining us and for your questions today. As a reminder, you can access our presentation on the Investor Relations section of the Sangamo website. We look forward to keeping you updated on our future developments. Thank you.

Operator

This concludes today's conference call. Thank you for participating. You may now disconnect. Everyone, have a great day.

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Key Takeaways

  • In April Sangamo signed its third StackBBB neurotropic capsid license, granting Lilly exclusive rights across five CNS targets with $18 million upfront and up to $1.4 billion in milestones plus royalties.
  • ST503, the epigenetic regulator for idiopathic small fiber neuropathy, is set to enter a Phase 1/2 intrathecal study in mid-2025 with preliminary proof-of-efficacy data expected in Q4 2026.
  • ST506 for prion disease has advanced CTA-enabling activities using the StackBBB delivery system, will begin enrollment mid-2026, and will feature preclinical data on survival benefits and brain-wide prion protein suppression at ASGCT.
  • In the Phase 1/2 STAR Fabry study all 32 patients have completed 52 weeks of follow-up with a sustained positive eGFR slope, and Sangamo secured a clear CMC path with the FDA toward a BLA submission in Q1 2026.
  • A newly priced equity offering is expected to extend Sangamo’s cash runway to late Q3 2025, supporting lean operations, ongoing cost cuts, and the pursuit of a Fabry commercialization partnership to fund its neurology pipeline.
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Earnings Conference Call
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