Q4 2024 Sangamo Therapeutics Inc Earnings Call
Okay.
Operator: Good afternoon and welcome to the Sangamo Therapeutics fourth quarter and full year 2024 teleconference call. Please be advised that today's conference is being recorded.
Speaker Change: 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.
Operator: I would now like to turn the conference over to your be a speaker to that. Please go ahead. Thank you.
Speaker Change: Now I'd like to turn the conference over to you.
Speaker Change: If you speak to that.
Speaker Change: Okay.
Speaker Change: Please go ahead.
Louise Wilkie: Good afternoon, everyone. Thank you for joining us on the call today.
Speaker Change: Thank you good afternoon, everyone. Thank you for joining us on the call today.
Louise Wilkie: On this call are several members of the Sangamo Executive Leadership Team, including Sandy Macrae, Chief Executive Officer, Nathalie Dubois, String Fellow, Chief Development Officer, and Prathyusha Duraibabu, Chief Financial Officer.
Speaker Change: On this call are supplement, but the fact that my executive leadership team, including Sandy Macrae, Chief Executive Officer, Nathalie Dubois Stringfellow, Chief Development Officer, I'm pretty sure do it Bobby Chief Financial Officer.
Louise Wilkie: Slides from our corporate presentation can be found on our website, sangamo.com, and under the Presentations page of the Investors and Media section. 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 fact.
Speaker Change: 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 Change: This call includes forward looking statements regarding Sangamo current expectations. These statements include but are not limited to statements related to think about 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 product candidates and technologies and.
Speaker Change: Goodbyes ability to earn and receive payments from our collaboration and license agreements.
Speaker Change: Expectations regarding new collaborations and license agreements the anticipated plans and time lines of Sangamo and its collaboration collaborators for clinical trials clinical data presentations and releases regulatory submissions regulatory approvals upcoming catalysts and milestones and other statements that are not historical fact actual.
Louise Wilkie: Actual results may differ materially from what we discussed today. These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC, specifically in our annual report on Form 10-K for fiscal year ended December 31, 2024, and subsequent filings and reports that Sangamo 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. Please note that all forward looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding.
Actual results may differ materially from what we discuss today.
Speaker Change: These statements are subject to certain risks and uncertainties are discussed in our filings with the SEC specifically in our annual report on Form 10-K for fiscal year ended December 31, 2024, and subsequent filings and reports the cycle I'll make some time to time with the SEC.
Speaker Change: 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. Please.
Speaker Change: Please note the forward looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding.
Sandy Macrae: Now, I'll turn the call over to our CEO, Sandy Macrae. Thank you, Louise, and good afternoon to everyone joining the call today. Sango'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 nonclinical 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 industry leading delivery capsid stack BBB.
Speaker Change: Now I'll turn the call over to IC or Sandy Macrae.
Sandy Macrae: Luis and good afternoon to everyone joining the call today.
Sandy Macrae: Shingles pipeline must meet a great deal of progress since the start of 'twenty 'twenty four.
We have advanced our prioritized neurology therapies towards the clinic securing their first shepherd neurology I M D and idiopathic small fiber neuropathy for chronic neuropathic pain and demonstrating non clinical proof of concept for our product candidate and prion disease.
Sandy Macrae: We showed that we are collaborator of choice from your trophy cap sits with the notes with two blue chip pharma agreements for industry, leading delivery capsid stack be be be the first with genentech photos and a second neurology target and the second with Astellas for up to five urology disease targets.
Sandy Macrae: 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. And our Fabry-June therapy study continues to generate best-in-class data with a pivotal data readout expected in mid-2025. From a financial standpoint, since 2023, we have reduced our non-GAAP operating expenses by nearly half year over year. And in 2024, we raised over $100 million in funding through non-dilutive license fees and milestone payments, as well as equity financing.
Sandy Macrae: We have a clear regulatory pathway to accelerated approval in fabry disease, which could reduce the time to potential approval by approximately three years.
Sandy Macrae: Under Fabry Gene therapy study continues to generate best in class data with a pivotal data readout expected in mid 2025.
Sandy Macrae: From a financial standpoint, since 2023, we have reduced our non-GAAP operating expenses by nearly half year over year.
Sandy Macrae: And in 2024, we reached over 100 million in funding through non dilutive license fees and milestone payments as well as equity financing.
Sandy Macrae: And these are significant achievements for a company of our size. Judged by all other metrics, this would have been a very successful year.
Sandy Macrae: And these are significant achievements for a company of our size.
Sandy Macrae: Judged by all other metrics this would have been up very successful year.
Sandy Macrae: However, we know, until we partner Fabry and appropriately capitalise the company for success, our work is not done. I want to re-emphasise that our number one priority continues to be addressing our financial needs. Sangamo must be well capitalised to fulfil our potential. We continue to engage in Fabry business development negotiations and securing a commercial partner is our key focus. Interest in this programme 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 2025 that is best suited to bringing ST920 to Fabry patients upon potential approval and that provides capital for Sangamo to execute on its other programmes.
Sandy Macrae: However, we knew until we partner Fabry and appropriately capitalize the company for success our work is not done.
Sandy Macrae: I want to reemphasize that our number one priority continues to be addressing their financing needs sangamo must be well capitalized to fulfill their potential.
Sandy Macrae: We continue to engage in fabry business development negotiations and securing a commercial partner is our key focus.
Sandy Macrae: Interest in this program. It's been strong this is proving to be a time consuming process. There's discussions of this nature are complex and facilitating the extensive due diligence required by potential partners takes time.
Sandy Macrae: We remain committed to securing and anticipated partnership in the second quarter of 2025 that is best suited to bringing S. T 922, fabry patients upon potential approval and that provides capital for sangamo to execute on its other programs.
Sandy Macrae: We will share information as soon as we're able.
Sandy Macrae: We will share information as soon as we're able.
Sandy Macrae: Furthermore, we are actively engaged in advanced contract negotiations with a potential collaborator for a third Stack VVB licence agreement and are hopeful of having more news to share near the end of this quarter.
Sandy Macrae: Furthermore, we are actively engaged in advanced contract negotiations with a potential collaborator for third stack BBB license agreement are hopeful tapping more news to share near the end of this quarter.
Nathalie Dubois: I would now like to hand it over to Nathalie, our Head of Development, who will walk us through detailed pipeline developments. Nathalie. 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 an 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 43,000 people in the United More broadly, peripheral neuropathy are estimated to affect nearly 40 million Americans.
Speaker Change: I would now like to hand, it over to Natalie or hated development, who will walk us through detailed pipeline developments.
Sandy Macrae: Italy.
Natalie Hated: Thank you Sandy first I'd like to provide updates on S. T 503, 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 issn ferro.
Natalie Hated: Neuropathy that results in highly debilitating symptoms, such as burning prickling stabbing or lightning like pain that is estimated to impact about 43000 people in the United States.
Natalie Hated: More broadly peripheral neuropathy isolated to affect nearly 40 millions of Mac.
Nathalie Dubois: As we've shared previously, a significant body of evidence implicates sodium channels in mediating the pathophysiology of neuropathic pain. 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 We believe our epigenetic regulation approach is differentiated. By directly targeting the SCN9A gene, SC503 has shown to precisely and potently reduce the expression of NAV1.7 sodium channels in sensory neurons in animal model and significantly reduce pain hypersensitivity following a single intrathecal administration.
Natalie Hated: As we've shared previously a significant body of evidence implicates sodium channels, indicating the pathophysiology of neuropathic pain.
Natalie Hated: <unk> three is a zinc finger repressor, our DSR targeting the human gene S. N 90 that encodes the NAV one seven sodium channel.
Natalie Hated: Developing small molecules that specifically targeting a 1.7 is challenging due to the highest structural similarities between different sodium channels, making it difficult to achieve selectivity and avoid off target effects.
Natalie Hated: We believe our epigenetic regulation approach is differentiated.
Natalie Hated: By directly targeting the S. C of nine eight gene S. T 503, as shown to precisely and potentially reduce the expression of NAV, one seven sodium channel in sensory neurons in animal model.
Natalie Hated: Significantly reduced pain IPO sensitivity following a seamless single instance, equal administration.
Nathalie Dubois: SC503 has been well-torated in non-human primates with no off-target effect observed.
Natalie Hated: <unk> has been well tolerated in nonhuman primates with no off target effect observed.
Nathalie Dubois: Following FDA clearance of the IND in November 2024, our first ever neurology IND, we are actively preparing for a phase one, two 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 ISF-2. This multi-center, 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. Patients will be randomized in a 2 to 1 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 cohorts.
Natalie Hated: Following FDA clearance of the A&D in November 2024.
Natalie Hated: First ever geology, and we are actively preparing for a phase one two study to assess the safety tolerability and preliminary efficacy of.
Natalie Hated: The one time dose of S. T fiber suite administer is particularly to patient with intractable pain due to issn.
Natalie Hated: This multicenter double blind randomized sham controlled dose escalation study is designed to evaluate it to evaluate three ascending doses beginning with what we believe to be the minimally efficacious dose as determined from animal studies.
Natalie Hated: Patients will be randomized in a two to one ratio to receive either S. U five O suite or a sham treatment with three patient plan in each of the first two dose cohorts and up to nine patients in the top dose cohort.
Nathalie Dubois: The primary objectives of this Phase 1-2 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 multidimensional impact of ST503 on sleep, mental health, and quality of life. We strongly believe in the potential of ST503 to reversionalize the chronic pain lens. And if successful, significant opportunity exists to broaden its application to patient populations suffering from other types of chronic neuropathic.
Natalie Hated: The primary objective of this phase one two study will be to assess the safety and Tolerability of S. T. Five O suite with secondary objective to assess preliminary efficacy based on the impact of S. T. Five O suite on respiratory pain and assessment of the multi dimensional impact of S. T. Five Oaks III.
Natalie Hated: On sleep mental health and quality of life.
Natalie Hated: We strongly believe in the potential of <unk>, five or three to revision of lives to chronic pain landscape and if successful significant opportunity exists to broaden its application to patient population suffering from other types of chronic neuropathic pain.
Nathalie Dubois: We plan to begin patient enrollment and dosing in mid-2025 and anticipate having preliminary proof of efficacy data in the fourth quarter of 2026.
Natalie Hated: We plan to begin patient enrollment and dosing in mid 2025, and anticipate having preliminary proof of efficacy data in the fourth quarter of 2026.
Nathalie Dubois: 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 2026. 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 neurodegenerative disease caused by the misfolding of the prion protein encoded by the PRNP gene. At least 1,300 new cases of prion disease are identified each year in the United States and in Europe, with a similar presence globally.
Natalie Hated: Moving to our prion disease program, we continue to advance clinical trial authorization or Cta, enabling activities and expect a cta submission in the U K in the first quarter of 2026.
Natalie Hated: Reminder, this program Leverages, our novel stack, BBB, capsid, which if it's safe and effective could validate our broader Europe neurology pipeline.
Natalie Hated: Prion disease is a rapidly fatal and incurable neurodegenerative disease caused by Misfolded of the prion protein encoded by the P. R. N P gene at least a 1300, new cases of prion disease identified each year.
Natalie Hated: <unk> stayed in Europe with a similar presents globally.
Nathalie Dubois: 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 ZFR 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 delivered via stag BBB to non-human primates resulted in potent and widespread reduction of prion expression in transuce neurons throughout the brain. This is a significant result in a highly challenging disease with no treatment options.
Natalie Hated: This quarter, we published a manuscript in bio archive, demonstrating non clinical proof of concept for our epigenetic regulation approach a single intravenous infusion of Ics saw significantly reduce expression of pie on mrna and protein in the mouse brain extended mouth survival and.
Natalie Hated: The improvement of molecular histological biomarker and behavior Readouts, even when administered post stem cell symptomatically to my sweeps prion disease.
In addition, a single intravenous administration of the prion CFR deliver that stockpile.
Natalie Hated: Stack BBB to nonhuman primates resulted in potent and widespread reduction of prion expression in tons use neurons throughout the brain. This is a significant result in a highly challenging disease with no treatment options today.
Nathalie Dubois: We plan to begin clinical trial enrollment and dosing in mid-2026 and expect to have preliminary clinical data in pre-Om disease in the fourth quarter of 2026.
Natalie Hated: We plan to begin clinical trial enrollment and dosing in mid 2026 and expect to have preliminary clinical data in prion disease in the fourth quarter of 2026.
Nathalie Dubois: Moving now to Fabry, at the World Symposium in February, we presented impressive updated preliminary clinical data from our phase 1, 2 star study of Israelgal gene Sivaparvovec, 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 ST920 as a one-time durable treatment option for Fabry disease that can improve patient outcomes. In the 33 patients treated with ST920, elevated expression of alpha-GAL-A activity was maintained for nearly four years for the longest treated patient.
Natalie Hated: Okay.
Natalie Hated: Moving now to fabry.
Natalie Hated: Symposium in February we presented impressive updated preliminary clinical data from our phase one to start study of either all got you can see that puzzle back our S. T. Nine 'twenty, our investigational gene therapy for the treatment of Fabry disease.
Natalie Hated: This latest data, which showed significant sustained benefit improvement in kidney function and a favorable safety profile continued to demonstrate the potential of <unk> 'twenty as a one time durable treatment option for fabry disease that can improve patient outcomes.
Natalie Hated: In those 33 patients treated with S. T nine 'twenty elevated expression of Alpha Gal a activity was maintained for nearly four years for the longest treated patient.
Nathalie Dubois: Importantly, we observe a positive mean EGFR slope of 3.061 millimeters of cleansed blood per minute per body surface in the 23 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 3 or minus 4. So this statistically significant positive EGFR slope is a remarkable achievement. All 18 patients who began the study on Enzyme Replacement Therapy, or ERT, have been successfully withdrawn from ERT and remain off ERT. We also observed notable improvement in quality of life among the patient with at least one year follow-up.
Natalie Hated: Importantly, we observed a positive mean egfr slope of 3.061 millimeters of claims blood per minute per body surface in the 23 patients with at least one year of follow up indicating notable improvement in renal function.
Speaker Change: For context, the average untreated patients in.
Speaker Change: In fabry disease, as an egfr slope of minus three minus four so these statistically significant positive egfr slope is a remarkable achievement.
Speaker Change: All 18 patients will begin to study an enzyme replacement therapy or E. R. T. I've been successfully withdrawn from E. R T and remain of E T.
Speaker Change: We also observed notable improvement in quality of life among the patients with at least one year follow up for example, among the short form 36 quality of life score. We saw a mean change in the general Health clause 10, six as well as significant improvement in physical component, but do you regain physical vitality.
Nathalie Dubois: For example, among the short-form 36 quality of life 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.
Speaker Change: Social function and emotional well being Scott.
Nathalie Dubois: The FDA has provided us with a clear regulatory pathway to accelerated approval for ST920 using EGFR slope at 52 weeks across all patients as an intermediate clinical endpoint. The 52 weeks EGFR slope data from all enrolled patients in the Phase 1, 2 star study will be available in the first half to 2025. We are thrilled with how the data are progressing and look forward to providing future updates as the full 52 weeks' data become available in the middle of this year. 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 2025.
Speaker Change: The FDA has provided us with a clear regulatory pathway to accelerated approval for <unk> and 'twenty using egfr slope at 52 weeks across all patient as an intermediate clinical endpoint. The 52 weeks Egfr slope data from all enrolled patients in a phase <unk> study will be a available in.
Speaker Change: The first half to 2025.
Speaker Change: We are thrilled with how the data are progressing and look forward to providing future updates.
Speaker Change: The full 52 weeks data become available in the middle of this year, 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 2025.
Nathalie Dubois: We're excited that this potential medicine could be commercialized as early as the second half of 2026, which would be an incredible achievement for Fabry patients.
Speaker Change: We're excited that this potential medicine could be commercialized as early as the second half of 2026, which would be an incredible achievement for fabry patients in need.
Sandy Macrae: I will now hand it back to Sandy for closing remarks.
Speaker Change: I will now hand, it back to Sandy for closing remarks, Thank you Natalie.
Sandy Macrae: Thank you, Nathalie. In closing, we're pleased with the progress we've made this year as we transition to becoming a clinical stage neurology company. We have advanced our long-plan neurology pipeline towards the clinic, securing our first ever neurology IND and with patient dosing expecting in the coming month. We have shown Sango to be the collaborator of choice for neurotropic capsids with the announcement of two blue-chip pharma agreements for SnackBBB. 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. We are proud of this progress and excited to be so close to the anticipated dosing of patients in our first ever neurology clinical trial.
Sandy Macrae: In closing we're pleased with the progress we've made this year as we transitioned to becoming a clinical stage neurology company.
Speaker Change: We have advanced our long planned neurology pipeline towards the clinic, securing our first surfer neurology I N T and with patient dosing, you're expecting in the coming months.
Speaker Change: We have shown single to be the collaboration of choice for new retrofit cap sits with the announcement of two blue chip pharma agreements for stack DPP.
Speaker Change: 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.
We are proud of this progress and excited to be so close to anticipated dosing of patients in our first effort neurology clinical trial.
Sandy Macrae: In parallel, we remain resolutely focused on raising the additional capital needed to set Sangamo up for success. As outlined earlier, we're in the very late stage business development negotiations for a third potential StackBBB license agreement. We have compelling Fabry data with a pivotal data readout expected in the coming months. and Business Development Negotiations for a Potential Fabric Commercialisation Agreement continue to advance with several interested partners. 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.
Speaker Change: In parallel we remain resolutely focused on raising the additional capital needed to set them up for success.
Speaker Change: As I explained earlier, we're in the very late stage business development negotiations for a third potential stock BBB license agreement, we have compelling fabry teacher with a pivotal data readout expected in the coming months.
Speaker Change: And business development negotiations for potential fabric commercialization agreement continue to advance with several interested partners.
Speaker Change: We believe we could reconsider both their short term and long term financing needs and look forward to providing additional updates as soon as they become available.
Operator: Operator, please open the line for questions.
Speaker Change: Operator, please open the line for questions.
Operator: Yes, sir. As a reminder, to ask a question, you will need to press star one one on your telephone. To remove yourself from the queue, you may press star 11 again. Please stand by while we compile the Q&A roster.
Speaker Change: Yes, Sir as a reminder to ask a question you will need to press star one one on your telephone.
Speaker Change: Move yourself from the queue you May press Star one one again.
Speaker Change: Please standby, while we compile the Q&A roster.
Luis Santos: Our first question comes from the line of Luis Santos of HC Wainwright. Your line is open, Louise. Hello, everyone. Thank you so much for taking our questions. This is Luis, and this is Patrick Truccio.
Speaker Change: Our first question.
Louise Santos: From the line of Louise Santos of H C Wainwright.
Speaker Change: Your line is open Louise.
Patrick Truck: Hello, everyone. Thank you so much for taking our question. This is Luis am sorry, Patrick truck here.
Sandy Macrae: I was wondering if you are still waiting on any data for the FABRI program, as you expect this partnership next discussions to lead to results next quarter. A similar question for the EuroVac and HEMA. Should we be expecting any more data later this year, and would that facilitate any partnerships there? Thank you. Thank you, Luis. The FAPRI 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 pay for the patients and for the right partnership for our shareholders.
Speaker Change: I was wondering if you're still waiting on any data for the fabry.
Patrick Truck: The program.
Speaker Change: As you expect this partnership next discussions to be.
Patrick Truck: In Q2 each of results next quarter.
Speaker Change: And then similar question for the <unk> and M. A.
Patrick Truck: Should we be expecting any.
Patrick Truck: More data later this year and would that facilitates any any partnerships there.
Patrick Truck: Yeah.
Patrick Truck: Thankfully so.
Patrick Truck: The Fabry partnership we would love to have announced that we would we are in late phase discussions across several partners potential partners.
Patrick Truck: And look forward to taking that forward and finding the right place for the right pace for the patients and for the right partnership for our shareholders.
Sandy Macrae: These things take time. The route forward from the agency was as recently as end of October. We've had to compress the planning for the file and launch from what was three, three and a half years down to six months to the filing and then to the launch. And so there's a huge amount of activities that are going on. What I can reassure you is the data that Nathalie and her team showed in 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.
Patrick Truck: These things take time.
Patrick Truck: The route forward from the agency was.
Recently as end of October.
Patrick Truck: The we've had to compress the planning for the file and launch from pulp was three three and a half years down to six months to the filing and then to the launch and so theres a huge amount of activity going on while I can reassure you is the data that naturally and her team shoot.
Patrick Truck: And.
Patrick Truck: And 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 us forward.
Sandy Macrae: 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.
Patrick Truck: It's a matter of closing the deal and making sure we find the right partner.
Sandy Macrae: For the haemophilia, that was returned to us, or the termination notice was given over the holiday period. And the more we see from Pfizer, the more we realize that it really was days away from both a 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.
Patrick Truck: For the hemophilia.
Patrick Truck: That was.
Patrick Truck: Return to us or to the termination notice was given over the holiday period.
Patrick Truck: And the more we see from Pfizer the more we realize that it really ball is days away from both.
Patrick Truck: Boots.
Patrick Truck: U S and European filing.
Patrick Truck: Our team are switching closely with the Pfizer organization to understand and explore all possibilities for a transition.
Sandy Macrae: 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. What I can tell you is that we've already had incoming interest on the haemophilia program, but I don't want to overpromise until we have a chance to understand all the data and speak with potential partners.
Patrick Truck: Ideally, we would like to hand, this directly to partner without having to go Sue Sangamo, but these are very early days too.
Patrick Truck: People to give you a commitment to that plan well I can tell us that we've already had incoming interest on the hemophilia.
Speaker Change: Graham, but I don't want tool for promising too we have a chance to understand all the data and speaking with potential partners.
Sandy Macrae: Just to clarify, 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? 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 a clinical trial, you have a responsibility to make the data available.
Speaker Change: Just to clarify.
Speaker Change: You get the rights to all of the data we have access to all of that and you will be able to share at some point.
Speaker Change: We will have access to all the data from falling Sir.
Speaker Change: And clearly later in the contract, but that's a huge amount of data for something that was about to be filed in such a few days time.
Speaker Change: Like you we respect the idea that if someone goes into a clinical trial you have a responsibility to meet the data available.
Luis Santos: Thank you so much. Thank you.
Speaker Change: Thank you so much.
Yanan Zhu: Our next question comes from Yanan Zhu of Wells Fargo. Please go ahead, Yanan. Hi, thanks for taking our questions.
Speaker Change: Thank you.
Speaker Change: Our next question.
Speaker Change: Comes from Johan Xu of Wells Fargo. Please go ahead.
Speaker Change: Hi.
Yanan Zhu: This is Quan Ang for Yana. So our question is also around Fabry. So can you share have the interesting party I've seen any data beyond the worst symposium data? 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 Change: Thanks for taking our questions. This is quite an honor for Yamana. So our question is also around fabry. So can you share the interesting party you haven't seen any data beyond.
Speaker Change: The worst symposium data that.
Speaker Change: That data is the data.
Speaker Change: They'd have cut in.
Speaker Change: I think September.
Speaker Change: 2024, so any updated data partners potential partners have seen beyond that thank you.
Sandy Macrae: I'm looking to Nathalie here, who's shaking her head. We believe they haven't seen any efficacy data beyond what we've seen. Yes. They have seen lots of other data. They've seen data about the manufacturing, about the CMC process. They've seen broader versions of the data cut that you saw, but they haven't seen later data.
Speaker Change: I'm looking to Nutley here.
Speaker Change: Shaking our heads we believed they haven't they haven't seen any efficacy data beyond what we're seeing yes.
Speaker Change: They have seen lots of other data they've seen data both the manufacturing of the CMC process, they've seen broader versions of the.
Speaker Change:
Speaker Change: The data cut that you sold but we haven't seen lease to lease her data. We know have seen data for up to 30 patients and theres only two more patients to complete their journey before we have the complete data set and can pull that together for the file.
Sandy Macrae: 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. Got it. That's super helpful. And for the 30 patient data you have seen so far, is the EGFR data consistent with what you have presented at work? I, 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.
Speaker Change: Got it that's super helpful and for the 30 patient data you have seen so far.
Speaker Change: ETF Egfr data concrete 10, we've quite a you have presented.
Speaker Change: At work.
Speaker Change: You can understand.
Speaker Change: I can give you more details, but we remain very.
Speaker Change: Encouraged by the data set and look forward to filing because we feel that is fulfilling old that the agency is asking for.
Yanan Zhu: That's super helpful. Thank you so much. Thank you.
Speaker Change: Okay Super helpful. Thank you so much.
Speaker Change: Thank you.
Maury Raycroft: Our next question comes from Maury Raycroft of Jeffries. Please go ahead, Maury. Hi, thanks for taking my question. I was going to ask on the STAC 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? 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. Yeah, we're guiding at the end of the year.
Speaker Change: Our next question comes from Maury Raycroft of Jefferies. Please go ahead Maury.
Maury Raycroft: Hi, Thanks for taking my question.
Maury Raycroft: I'm 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 and what are the upfront for that one could look like.
Maury Raycroft: So I think you could look at.
Maury Raycroft: What we've got is upfronts for the previous two deals there is no almost like a standard market price for those.
Maury Raycroft: We're guiding at the end of the year.
Maury Raycroft: We hope at the end of the quarter. Thank you, Louise.
Louise Santos: We hope so as they entered the quarter. Thank you Louise we had hoped to have done it in time for this for for this quarterly call.
Sandy Macrae: 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. Got it. That's helpful.
Louise Santos: Is it just these things are not always in our hands, but.
Louise Santos: We know that the partner is one that you will find a very logical blue chip choice and we.
Louise Santos: Phil.
Louise Santos: We are putting a capsid in the hands of a great. Another great company that will allow.
Louise Santos: Their neurology pipeline and frankly to advance.
Prathyusha Duraibabu: And just a quick question on OPEX, just how you're thinking about that going forward.
Speaker Change: Got it that's helpful and just a quick question on Opex, just how youre thinking about that going forward and once you solidify the partnership for Fabry how that.
Prathyusha Duraibabu: And once you solidify the partnership for FABRI, how that change, how that could Prathyusha.
Louise Santos: That could change.
Prathyusha Duraibabu: Hi, Murray, how are you? From the OPEX itself, we've done everything proactively within our control. We've reduced our OPEX by nearly half over year over year. And we've defined our OPEX 425 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 now 1.7 and pre-on forward.
Louise Santos: Sure.
Louise Santos: Laura how are you.
Speaker Change: And from the Opex itself, we've done everything proactively what's in our control, we've reduced our opex by nearly half or year over year.
Speaker Change: And we've defined our opex for 25 to be very focused on taking on neurology pipeline forward. So from a guidance perspective, we guided to keeping the same level of opex as last year as we move both not one seven then prion forward.
Maury Raycroft: Understood.
Maury Raycroft: Okay, thanks for taking my questions.
Speaker Change: Understood. Okay. Thanks for taking my questions.
Speaker Change: Yeah.
Operator: Thank you.
Nicole Germino: Once again, to ask a question, please press star 11 on your telephone. Again, that's star 11 on your telephone to ask a question.
Speaker Change: Thank you once again to ask a question. Please press star one one on your telephone again Thats Star one one on your telephone to ask a question.
Nathalie Dubois: Our next question comes from Nicole Germino of Truist. Please go ahead, Nicole. Good afternoon, and thanks for taking my question. So there's some literature around hypotension associated with inhibition of NAV.1 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? And then second question, can you help us understand the patient enrollment criteria enrolling into the NAV 1.7 study.
Speaker Change: Our next question.
Speaker Change: Comes from Nicole Germano of Truest. Please go ahead Nicole.
Nicole Germano: Good afternoon, and thanks for taking my question.
So there is some literature around hypertension associated with inhibition of NAV, one channel sodium channels.
Speaker Change: I have two questions do you have any hypotheses and how and why youre not seeing any hypertension, so far in animal models or rather maybe how do you get investors comfortable around any potential hypotension cultures and then second question can you help us understand the patient enrollment criteria for patients.
Nicole Germano: Early in Q1.
Nicole Germano: One seven study.
Nathalie Dubois: Nathalie, can you talk to these? Yeah, so, so, you know, we have not seen any effect on hyper or hypotension in all our animal study, especially in the NHP study where it's a GLP study where we monitor the function very closely. I think the NAV1.7 relationship with hyper 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. So I think it's too early to make the conclusion that NAV1.7, or there's not enough evidence to show that NAV1.7 could impact hyper or hypotension.
Nicole Germano: Naturally can you talk to these yeah. So so we have not.
Nicole Germano: Not seen any effect on <unk> potential in all of animal study, especially in the NH Pea study, where it's a G. L. P study, where we monitor those functions very closely.
Nicole Germano: I think the NAV one seven a relationship with <unk> our eye potential is not really well established I think there is one report on the small molecule.
Nicole Germano: That was.
Nicole Germano: Reported and the the protein was the small molecule is delivered.
Nicole Germano: <unk> IV to the general and we don't know the specificity. We don't have that information. So I think it's too early to make the conclusion that loved one seven or there is not enough evidence to show that not one seven could impact our portal hypertension.
Nathalie Dubois: And all the monkeys are closely monitored, the blood pressure is monitored and they've all done very well, so we feel it's a very different situation. And then it's also intrathecal as well, right? Intrathecal, yes.
Nicole Germano: And they'll do the monkeys are closely monitored the blood pressure monitor.
Nicole Germano: No they've all done very well so we feel it's a very different situation.
Nicole Germano: And then just also of interest equal as well.
Nicole Germano: It seems like the call yes.
Nathalie Dubois: And there was a second question. On the patient enrollment criteria. So we're targeting patient with ISFN, as mentioned. And, you know, there is a bunch of inclusion exclusion criteria that will be highlighted when we publish the clinical trial that goes design in, you know, design and public cultural cost. So is there anything specific you're looking for? Just overall, the patient description for the SFN patient population, if there's anything that... if there's anything. I don't think so. We're, you know, there's always a balance between having as pure a population and trying to avoid comorbidities. And that's always a fine needle to a fine needle.
Nicole Germano: And there was a second question on the patient enrollment criteria. So we're targeting patients with issn.
Nicole Germano: You mentioned and you know there is a bunch of inclusion exclusion criteria that will be.
Nicole Germano: Yeah highlighted when we publish our clinical trial dot Gov.
Nicole Germano: Design in design on political tough comps. So is there anything specific you're looking for.
Nicole Germano: Just just overall the pn description for the.
Nicole Germano: Patient population, if theres anything that.
Nicole Germano: If theres anything of note.
Nicole Germano: I don't think so.
Nicole Germano: There's always a balance between having as pure population.
Nicole Germano: Im trying to avoid.
Nicole Germano: Comorbid Comorbidities and nuts.
Nicole Germano: It's always a fine needle to find them.
Nathalie Dubois: path to 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 emphasise 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. Great, thank you. Thank you.
Nicole Germano: Path to to try and execute but I think the team has done a nice job of getting a recruit people study.
Nicole Germano: That will give us a clearer answer.
Nicole Germano: We I think it's important to emphasize that this is a onetime treatment and therefore the benefit risk is really important and therefore, a clear result is what we need to look for for the.
Nicole Germano: However, full effect that we believe if the animal models are replicated that this molecule shoot to achieve.
Speaker Change: Great. Thank you so much.
Speaker Change: Thank you.
Jenna Wang: Our next question. comes from Jenna Wang of Barclays. Please go ahead, Jenna. Thank you. I have two sets of questions. First one is regarding the February deals.
Speaker Change: Our next question.
Speaker Change: Comments from Gena Wang of Barclays. Please go ahead gena.
Speaker Change: Thank you.
Speaker Change: Two sets of questions first one is regarding the separate deals.
Sandy Macrae: Just wanted to know that the delay on February deal, the potentially 2Q, was that due to the deal term agreement or is that because the potential partner wanted to see additional data or regulatory certainty? And then I used to looking for due time to cover the I think almost two years OPEX to until you reach, let's say, full Q26, be able to show the proof.
Speaker Change: Just wanted to note that the delay on February <unk> the potential of <unk>.
Speaker Change: Was that due to the to you Tom.
Speaker Change: Agreement or is that.
Speaker Change: Because the potential partner wanted to see additional data or regulatory certainties, and then I used to looking for do you Tom to cover.
Speaker Change: I think almost two years Opex two <unk> reached Aleksey for Q2, 'twenty six be able to.
Should approve it.
Sandy Macrae: concept data from both internal programs. And the second question is regarding the 503. full Q26 data update. Do you expect to identify going forward dose, and what is your goal of a placebo-adjusted pain score reduction? So Gina, thank you for your questions. The Fabry discussions are going well. The clinical results are so compelling that 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. 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 Nav1.7 and hopefully show early results for prion disease.
Speaker Change: Concept data from both internal program and the second question is regarding the FIFO three.
Speaker Change: Full Q26 data update.
Speaker Change: Do you expect to identify going forward dose and what is your goal.
Speaker Change: Placebo adjusted pain score reduction.
Speaker Change: So gena thank you for your questions.
Speaker Change: The fabry discussions are going well.
Speaker Change: The clinical results are so compelling.
Speaker Change: Each of the partners is fascinated by it.
Speaker Change: We can discuss the terms and we can't discuss what were what were in negotiation over I'm sure you understand that.
Speaker Change: But the overall goal of Sangamo has two piece to get us well funded to get to that point at the end of next year, where we can demonstrate the effectiveness of one seven and hopefully show early results for prion disease. So that's our overall mission and as we go.
Nathalie Dubois: 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.
Closer and closer to the pre BLA meeting in the summer and to file by the end to the year you can imagine that the.
Speaker Change: The energy around the discussions increases Natalie your you had a question as well yeah yeah.
Nathalie Dubois: And Nathalie, you had a question as well, didn't you? Yeah. Yes. On the ST503. 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 12 weeks. We understand the placebo effect, which is an important consideration. And as we've planned for our phase one to study design, but you have to consider also that it's a one time therapy. 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.
Speaker Change: <unk> hundred three.
Speaker Change: So to be successful our product candidate and its demonstrated both near term efficacy and long term effect.
Speaker Change: In our trial, we hope to see a reduction in pain within the first 12 weeks, we understand the placebo effect, which is an important consideration and as we plan for our phase one two study design, but you have to consider also that it's a one time therapy. So our approach is very different to two.
Speaker Change: Additional therapy, whereas the placebo control has been documented where.
Speaker Change: Then the sham treatment are taken regularly which really concentrate to the reminder of the placebo effect.
Nathalie Dubois: 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, you know, 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, you know, the effect is maximal and plateau at about three to four weeks after administration.
Speaker Change: So just as a reminder, this is a onetime potential treatment.
Speaker Change: 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.
Speaker Change: Have a near term.
Speaker Change: Because see fairly quickly.
In our animal study I would say that you know the effect is maximo on plateau at about three to four weeks after administration.
Nathalie Dubois: Are you looking for any placebo, just the pain score reduction? You will be looking for like a two score or, you know, any particular scores you have in mind, giving some benchmark. We wouldn't be wise to 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 effect because it's a dreadful disease and intractable pain is is not something anyone would wish to have. And we look forward to sharing the results with you over the coming year to 18 months.
Speaker Change: How are you looking for any of the Siebel adjusted pain score reduction.
Speaker Change: We'll be looking for like a two squirrel.
Speaker Change: Any particular course, you have in mind, giving some bench.
Speaker Change: Sure.
Speaker Change: We wouldn't be wise to set ourselves a target of the amount of production.
As clinical signs and the first time, it's been administered to humans, we hope that it will be a significant effect because this is a dreadful disease.
Speaker Change: Back to painting is is not something anyone would wish to have and we look forward to sharing the results with you for the coming year.
Speaker Change: Year to 18 months.
Speaker Change: Thank you.
Speaker Change: Yes.
Nathalie Dubois: Thank you.
Speaker Change: Okay.
Operator: I am showing no further questions.
Luis: Thank you I'm showing no further questions I would now like to turn the call back to Luis <unk> for closing remarks.
Louise Wilkie: I would now like to turn the call back to Louise Wilkie for closing remarks, madam. 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 Sangamo website. We look forward to keeping you updated on our future developments.
Luis: 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 Sangamo 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.
Luis: This concludes today's conference call. Thank you for participating you may now disconnect.
Luis: Okay.
Luis: [music].
Luis: Yeah.
Luis: Okay.
Luis: [music].
Luis: Yeah.
Luis: [music].