Sangamo Therapeutics Q4 2024 Earnings Call Transcript

Quartr
Provided by Quartr
March 17, 2025

There are 9 speakers on the call.

Operator

Good afternoon, and welcome to the Sangamo Therapeutics Fourth Quarter and Full Year twenty twenty four 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. 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 Natalie Dubar Stringfellow, Chief Development Officer and Pratisha Durababu, Chief Financial 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 related to Sangamo's cash runway, plans to obtain additional capital and ability to continue operating 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 expectations regarding new collaborations and license agreements the anticipated plans and timelines of Sangamo and its collaborators for clinical trials, clinical data presentations and releases, regulatory submissions, regulatory approvals, 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 and Form 10 ks for fiscal year ended 12/31/2024, and subsequent filings and reports that Sangoma makes from time to time with the SEC. The forward looking statements stated today are made as of today, and we undertake no duty to update such information except as required by law.

Speaker 1

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 today. Sangamo's pipeline has made a great deal of progress since the start of 2024. We have advanced our prioritized neurology therapies towards the clinic, securing our first ever neurology IND in idiopathic small fiber neuropathy for chronic neuropathic pain and demonstrating non clinical proof of concept for a product candidate in prion disease. We showed that we are a collaborator of choice for neurotropic capsids with the announcement of two blue chip pharma agreements for our industry leading delivery capsid, STACK BBB, the first with Genentech for tau and a second neurology target and the second with Astellas for up to five neurology disease targets. We have a clear regulatory pathway to accelerated approval in Fabry disease, which could reduce the time to potential approval by approximately three years.

Speaker 2

And our Fabry gene therapy study continues to generate best in class data with And in 2024, we raised over $100,000,000 in funding, And in 2024, we raised over $100,000,000 funding through non dilutive license fees and milestone payments as well as equity financing. And these are significant achievements for a company of our size. Judged by all other metrics, this would have been a very successful year. However, we know until we partner Fabry and appropriately capitalize the company for success, our work is not done. I want to reemphasize that our number one priority continues to be addressing our financing needs.

Speaker 2

Sangamo must be well capitalized to fulfill our potential. We continue to engage in Fabry business development negotiations and securing a commercial partner is our key focus. Interest in this program has been strong. This is proving to be a time consuming process as discussions of this nature are complex and facilitating the extensive due diligence required by potential partners takes time. We remain committed to securing an anticipated partnership in the second quarter of twenty twenty five that is best suited to bringing ST-nine twenty to Fabry patients upon potential approval and that provides capital for Sangamo to execute on its other programs.

Speaker 2

We will share information as soon as we are able. Furthermore, we are actively engaged in advanced contract negotiations with a potential collaborator for third STACK BBB license agreement and are hopeful of having more news to share near the end of this quarter. I would now like to hand it over to Natalie, our Head of Development, who will walk us through detailed pipeline developments. Natalie?

Speaker 3

Thank you, Sandy. First, I'd like to provide updates on SC503, our investigational epigenetic regulator for the treatment of chronic neuropathic pain, which is ready to enter the clinic. Our initial indication is in idiopathic small fiber neuropathy or ISFN, a peripheral neuropathy that results in highly debilitating symptoms such as burning, prickling, stabbing or lightning like pain that is estimated to impact about forty three thousand people in The United States. More broadly peripheral neuropathy are estimated to affect nearly forty million Americans. As we've shared previously, a significant body of evidence implicates sodium channels in mediating the pathophysiology of neuropathic pain.

Speaker 3

SC503 is a zinc finger repressor or ZFR targeting the human gene SCN9A that encodes the NAV1.7 sodium channel. Developing small molecules that specifically target NAV1.7 is challenging due to the high structural similarities between different sodium channels, making it difficult to achieve selectivity and avoid off target effects. We believe our epigenetic regulation approach is differentiated. By directly targeting the SCN9A gene, SC503 has shown to precisely and potentially reduce the expression of NAB1.7 sodium channels in sensory neurons in animal model and significantly reduced pain hypersensitivity following a single intrathecal administration. ST503 has been well prorated in non human primates with no off target effect observed.

Speaker 3

Following FDA clearance of the IND in November 2024, our first ever pathology IND, we are actively preparing for a Phase onetwo study to assess the safety, tolerability and preliminary efficacy of a one time dose of ST503 administered intrinsically to patient with intractable pain due to ISFN. This multicenter, double blind, randomized, sham controlled dose escalation study is designed to evaluate three ascending doses, beginning with what we believe to be the minimally efficacious dose as determined from animal studies. Patient will be randomized in a two:one ratio to receive either SC503 or a sham treatment with three patients planned in each of the first two dose cohorts and up to nine patients in the top dose cohort. The primary objective of this Phase onetwo study will be to assess the safety and tolerability of ST503 with secondary objective to assess preliminary efficacy based on the impact of ST503 on refractory pain and assessment of the multi dimensional impact of ST503 on sleep, mental health and quality of life. We strongly believe in the potential of SC503 to revolutionize the chronic pain landscape and if successful significant opportunity exists to broaden its application to patient population suffering from other types of chronic neuropathic pain.

Speaker 3

We plan to begin patient enrollment and dosing in mid twenty twenty five and anticipate having preliminary proof of efficacy data in the fourth quarter of twenty twenty six. Moving to our Prion disease program, we continue to advance clinical trial authorization or CTA enabling activities and expect a CTA submission in The UK in the first quarter of twenty twenty six. As a reminder, this program leverages our novel STAG BBB capsid, which if safe and effective could validate our broader neurology pipeline. Prion disease is a rapidly fatal and incurable neurogenerative disease caused by the misfolding of the prion protein encoded by the PRNP gene. At least thirteen hundred new cases of prion disease are identified each year in The United States and in Europe with a similar presence globally.

Speaker 3

This quarter, we published a manuscript in BioRxiv demonstrating non clinical proof of concept for our epigenetic regulation approach. A single intravenous infusion of our ZSR significantly reduced expression of prion mRNA and protein in the mouse brain, extended mouse survival and improved an array of molecular histological biomarker and behavior readouts even when administered post symptomatically to mice with prion disease. In addition, a single intravenous administration of the prion ZFR deliver via STAG BBB to non human primates resulted in potent and widespread reduction of prion expression in transduced neurons throughout the brain. This is a significant result in a highly challenging disease with no treatment options today. We plan to begin clinical trial enrollment and dosing in mid-twenty twenty six and expect to have preliminary clinical data in pre owned disease in the fourth quarter of twenty twenty six.

Speaker 3

Moving now to Fabry, at the World Symposium in February, we presented impressive updated preliminary clinical data from our Phase III STAR study of eitheral galcin cevaparvavec or ST920, our investigational gene therapy for the treatment of Fabry disease. This latest data which shows significant sustained benefit improvement in kidney function and a favorable safety profile continue to demonstrate the potential of ST-nine twenty as a one time durable treatment option for Fabry disease that can improve patient outcomes. In the thirty three patients treated with ST-nine twenty elevated expression of alpha Gal A activity was maintained for nearly four years for the longest treated patient. Importantly, we observe a positive mean eGFR slope of 3.061 millimeters of clean blood per minute per body surface in the twenty three patients with at least one year of follow-up indicating notable improvement in renal function. For context, the average untreated patient in Fabry disease has an eGFR slope of minus three or minus four, though this statistically significant positive eGFR slope is a remarkable achievement.

Speaker 3

All eighteen patients who began the study on enzyme replacement therapy or ERT have been successfully withdrawn from ERT and remain off ERT. We also observe notable improvement in quality of life among the patient with at least one year follow-up. For example, among the short term form 36 quality of lab score, we saw a mean change in the general health score of 10.6 as well as significant improvement in physical component, bodily gain, physical vitality, social function and emotional well-being scores. The FDA has provided us with a clear regulatory pathway to accelerated approval for ST-nine twenty using eGFR slope at fifty two weeks across all patient as an intermediate clinical endpoint, the fifty two weeks eGFR slope data from all enrolled patient in the Phase III STAR study will be available in the first half to twenty twenty five. We are thrilled with how the data are progressing and look forward to providing future update as the full fifty two weeks data become available in the middle of this year.

Speaker 3

We're actively preparing all necessary activity for the BLA including manufacturing readiness and continue to work towards a potential BLA submission in the second half of twenty twenty five. We're excited that this potential medicine could be commercialized as early as the second half of twenty twenty six, which would be an incredible achievement for Fabry patients in need. I will now hand it back to Sandy for closing remarks.

Speaker 2

Thank you, Natalie. In closing, we're pleased with the progress we have made this year as we transition to becoming a clinical stage neurology company. We have advanced our long planned neurology pipeline towards the clinic, securing our first effort in neurology IND and with patient dosing expecting in the coming months. We have shown Sango to be the collaborator of choice for neurotropic capsids with the announcement of two blue chip pharma agreements for STACK BBB. And we have secured a clear regulatory pathway to accelerated approval in Fabry disease, which would reduce the time to potential approval by approximately three years.

Speaker 2

We are proud of this progress and excited to be so close to anticipated dosing of patients in our first ever neurology clinical trial. In parallel, we remain resolutely focused on raising the additional capital needed to set Sangamo for success. As outlined earlier, we're in the very late stage business development negotiations for a third potential Stack BBB license agreement. We have compelling Fabry data with a pivotal data readout expected in the coming months. And business development negotiations for a potential fabri commercialization agreement continue to advance with several interested partners.

Speaker 2

We believe we can solve both our short term and long term financing needs and look forward to providing additional updates as soon as they become available. Operator, please open the line for questions.

Operator

Our first question comes from the line of Luis Santos of H. C. Wainwright. Your line is open, Luis.

Speaker 4

Hello, everyone. Thank you so much for taking our questions. This is Luis in for Patrick Trucchio. I was wondering if you're still waiting on any data for the Fabry program as you expect this partnership next discussions to lead to results next quarter. Similar question for the DURUVAC in MA.

Speaker 4

Should we be expecting any more data later this year? And would that facilitate any partnerships there? Thank you.

Speaker 2

Thank you, Luis. And so the Fabry partnership, we would love to have announced it. We would we're in late phase discussions across several partners, potential partners and look forward to taking that forward and finding the right place for the right for the patients and for the right partnership for our shareholders. These things take time. The route forward from the agency was as recently as October.

Speaker 2

We've had to compress the planning for the file and launch from what was three, three point five years down to six months to the filing and then to the launch. And so there's a huge amount of activities are going on. What I can reassure you is the data that Natalie and her team showed in at the World Symposium remains positive, remains very positive and we look forward to seeing the one year data for the last patient as soon as next month and that will allow us to drive this forward. Now it's a matter of closing the deal and making sure we find the right partner. For the hemophilia, that was returned to us or the termination notice was given over the holiday period.

Speaker 2

And the more we see from Pfizer, the more we realize that it really was days away from both U. S. And a European filing. Our team are sitting closely with the Pfizer organization to understand the and explore all possibilities for a transition. Ideally, we would like to hand this directly to a partner without having to go through Sangamo, but these are very early days to be able to give you a commitment to that plan.

Speaker 2

What I can tell you is that we've already had incoming interest on the hemophilia program, but I don't want to overpromise until we have a chance to understand all the data and speak with potential partners.

Speaker 4

Just to clarify, are you you get the rights to all of the data? Will you have access to all of that and you will be able to share at some point?

Speaker 2

We will have access to all the data from Pfizer. It's very clearly laid out in the contract, but that's a huge amount of data for something that was about to be filed in but a few days time. And like you, we respect the idea that if someone goes into clinical trial, you have a responsibility to make the data available.

Speaker 4

Thank you so much.

Operator

Thank you. Our next question comes from Yannan Zhu of Wells Fargo. Please go ahead, Yannan.

Speaker 4

Hi. Thanks for taking our questions. This is Quan on for Yannan. So our question is also around Fabry. So can you share have the interesting party have seen any data beyond the World Symposium data?

Speaker 4

I think that data has a data cut in, I think, September 2024. So any updated data partners potential partner have seen beyond that? Thank you.

Speaker 2

I'm looking to Natalie here and who's shaking her head. We believe they haven't seen any efficacy data beyond what we've seen.

Speaker 3

Yes.

Speaker 2

They have seen lots of other data. They've seen data about the manufacturing, about CMC process. They've seen broader versions of the data cut that you saw, but they haven't seen later data. We now have seen data for up to the 30 patients and there's only two more patients to complete their journey before we have the complete data set and can pull that together for the file.

Speaker 4

Got it. That's super helpful. And for the 30 patients data you have seen so far, is the eGFR data consistent with what you have presented at work?

Speaker 2

You can understand, I can't give you more details, but we remain very encouraged by the data set and look forward to filing it because we feel that it's fulfilling all that the agency is asking for.

Speaker 4

Yes, super helpful. Thank you so much.

Operator

Thank you. Our next question comes from Maury Raycroft of Jefferies. Please go ahead, Maury.

Speaker 5

Hi, thanks for taking my question. I was going to ask on the Stack BBB deal, just clarifying on that. You said you could have that in place by the end of this month. Is that correct? And then is there more you can say on what the upfront for that one could look like?

Speaker 2

So I think you could look at what we've got as upfronts for the previous two deals. There's now almost like a standard market price for those. We're guiding at the end of the year. We hope at the end of the quarter, thank you, Luis. We had hoped to have done it in time for this quarterly call and just these things are not always in our hands, but we know that the partner is one that you will find a very logical blue chip choice and we feel that we are putting a capsid in the hands of another great company that will allow their neurology pipeline and ours frankly to advance.

Speaker 5

Got it. That's helpful. And just a quick question on OpEx. Just how you're thinking about that going forward and once you solidify the partnership for Fabry, how that could change?

Speaker 2

Patricia?

Speaker 6

Hi, Mari. How are you? From the OpEx itself, we've done everything proactively within our control. We've reduced our OpEx by nearly half year over year and we've designed our OpEx for 2025 to be very focused on taking our neurology pipeline forward. So from a guidance perspective, we've guided to keeping the same level of OpEx as last year as we move both NAV 1.7 and PRION forward.

Speaker 5

Understood. Okay. Thanks for taking my questions.

Operator

Thank you. Our next question comes from Nicole Germino of Truist. Please go ahead, Nicole.

Speaker 7

Good afternoon and thanks for taking my question. So there is some literature around hypertension associated with inhibition of NAV 0.1 channel, sodium channels. So I have two questions. Do you have any hypotheses on how and why you're not seeing any hypotension so far in animal models? Or rather, maybe how do you get investors comfortable around any potential hypotension concerns?

Speaker 7

And then second question, can you help us understand the patient enrollment criteria for patients enrolling into the NAV 1.7 study?

Speaker 2

Natalie, can you talk to these?

Speaker 3

Yes. So we have not seen any effect on hyper hypotension in all our animal study, especially in the NHP study where it's a GLP study where we monitor those functions very closely. I think the NAV1.7 relationship with IPER or hypotension is not really well established. I think there is one report on a small molecule that was reported and the protein was the small molecule was delivered IV to the general and we don't know the specificity. We don't have that information.

Speaker 3

So I think it's too early to make the conclusion that NAB1.7 or there is not enough evidence to show that NAB1.7 could impact, IPR or hypotension.

Speaker 2

And all the monkeys are closely monitored, the blood pressure is monitored.

Speaker 3

Absolutely. There is no

Speaker 2

They've all done very well. So we feel it's a very different situation.

Speaker 7

And then it's also intrathecal as well, right? Intrathecal is

Speaker 3

not true. Intrathecal, yes.

Speaker 2

And there was a second question.

Speaker 3

On the patient enrollment criteria. So we're targeting patient with ISFN as mentioned, and there is a bunch of inclusion exclusion criteria that will be highlighted when we publish the clinicaltrial.gov design in design and publichealth.gov. So is there anything specific you're looking for?

Speaker 7

Just overall the patient description for the SFN patient population, if there's anything that if there's anything of note?

Speaker 2

I don't think so. There's always a balance between having a pure population and trying to avoid comorbidities and that's always a fine needle to a fine path to try and execute. But I think the team have done a nice job of getting a recruitable study that will give us a clear answer. And we I think it's important to emphasize that this is a one time treatment and therefore the benefit risk is really important and therefore a clear result is what we need to look for, for the powerful effect that we believe if the animal models are replicated that this molecule should achieve.

Speaker 7

Great. Thank you so much.

Operator

Thank you. Our next question comes from Jenna Huang of Barclays. Please go ahead, Jenna.

Speaker 8

Thank you. I have two sets of questions. First one is regarding the February deal. Just wanted to know that the delay on February deal, the potential 2Q, was that due to the deal term agreement or is that because the potential partner wanted to see additional data or regulatory certainties? And then are you still looking for a deal term to cover the I think almost two years OpEx to until you reach, let's say, 4Q twenty twenty six be able to show the proof concept data from both internal program?

Speaker 8

And the second question is regarding the five zero three. For Q twenty twenty six data update, do you expect to identify going forward dose and what is your goal of a placebo adjusted pain score reduction?

Speaker 2

So, Gina, thank you for your questions. The Fabry discussions are going well. The clinical results are so compelling that the each of the partners is fascinated by it. We can't discuss the terms and we can't discuss what we're in negotiation over. I'm sure you understand that.

Speaker 2

But the overall goal of Sangamo has to be to get us well funded to get to that point at the end of next year where we can demonstrate the effect of NAFLN zero point seven and hopefully show early results for prion disease. So that's our overall mission. And as we get closer and closer to the pre BLA meeting in the summer and the file by the end of the year, you can imagine that the energy around the discussions increases. And Natalie, you had a question as well, Denise?

Speaker 3

Yes. On the ST-five zero three, so to be successful, our product candidate needs to demonstrate both near term efficacy and long term effect. In our trial, we hope to see a reduction in pain within the first twelve weeks. We understand the placebo effect, which is an important consideration and as we've planned for our Phase onetwo study design. But you have to consider also that it's a one time therapy.

Speaker 3

So our approach is very different to traditional therapy where the placebo control has been documented where then the sham treatment are taken regularly, which really can straighten the reminder of the placebo effect. So just as a reminder, this is a one time potential treatment with one injection and we believe the placebo effect will start to wane as we get further away from the point of administration. And as I mentioned, we think looking at the data in from the animal study that really we should have a near term efficacy fairly quickly. In our animal study, I would say that the effect is maximum and plateau at about three to four weeks after administration.

Speaker 8

Are you looking for any placebo adjusted pain score reduction? You will be looking for like a two score or any particular scores you have in mind giving some benchmark?

Speaker 2

We wouldn't be wise to set ourselves a target of the amount of reduction. This is clinical science and the first time it's been administered in humans. We hope that it will be a significant impact because this is a dreadful disease and intractable pain is not something anyone would wish to have. And we look forward to sharing the results with you over the coming year to eighteen months.

Speaker 8

Thank

Operator

you. Thank you. I am showing no further questions. I would now like to turn the call back to Louise Wilkie for closing remarks. Madam?

Speaker 1

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

Operator

This concludes today's conference call. Thank you for participating. You may now disconnect.

Powered by Quartr
Earnings Conference Call
Sangamo Therapeutics Q4 2024
00:00 / 00:00