Q1 2025 Sangamo Therapeutics Inc Earnings Call
The conference over to your Speaker today, Louise Wilkie, Vice President Investor Relations and corporate Communications. Please go ahead.
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 <unk>, Chief Financial Officer, and Luckily Dubroff Stringfellow Chief development.
Officer slides from our corporate presentation can be found on our website sangamo dot com under the presentations page of the investors and media section. This.
This call includes forward looking statements regarding Sangamo current expectations. These statements include but are not limited to statements relating to <unk> cash runway on operating expense guidance. The anticipated closing of the announced underwritten offering sangamo has 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 as product candidates and technologies Franco has ability to earn and receive payments from its collaboration and license agreements signed with the ability to establish and maintain collaborations and strategic partnerships, including for Fabry disease program. The anticipated client plans and timelines of Sangamo and its collaborators for clinical trials clinical data presentations and releases.
Regulatory submissions and regulatory approvals.
Coming catalyst 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 uncertainties that are discussed in our filings with the SEC specifically in our annual report on Form 10-K for the fiscal year ended December 31, 2024, and our quarterly report on Form 10-Q for the fiscal quarter ended March 31, 2025, and subsequent filings and reports of Sanka makes from 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 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 extra CEO Sandy macrae. Thank.
Thank you Luis and good afternoon to everyone joining the call.
Sandy Macrae: It's only been a couple of months since our fourth quarter call, but I am pleased to share with you some company progress across a variety of areas go to their capsid engineering platform, our neurology pipeline, our fabry program under our finances.
Sandy Macrae: Beginning with our capsid engineering platform in April we announced our third capsid license agreements since we shared the discovery of our industry, leading neuro trophic delivery capsid stack BBB.
Sandy Macrae: We were pleased to sign an agreement with Eli Lilly and company Grunting Lilly a worldwide exclusive license stacked BBB for up to five potential disease targets of the central nervous system.
Sandy Macrae: We have received the $18 million upfront license fee for the first target and are eligible to earn up to one $4 billion in additional licensed target fees and milestone payments across all five potential disease targets as well as tiered royalties on potential net sales.
Sandy Macrae: We are through top scientists third important agreement further demonstrating that we are collaborator of choice for newer trophy cap suits with Genentech Astellas are no Lilly we have great partners in neuroscience for technology, and we continue to engage in discussions with new potential collaborators for stack BB.
Sandy Macrae: Pete.
Sandy Macrae: Okay.
Sandy Macrae: Turning turn urology pipeline programs. This quarter, we continue to advance clinical study preparations for <unk> three our investigational epigenetic regulator for the treatment of chronic neuropathic pain.
Sandy Macrae: We are preparing for a phase one two study to assess the safety tolerability and preliminary efficacy of a one time too so best <unk> three our investigational epigenetic regulator there'll be administered interestingly two patients with intractable pain due to idiopathic small fiber neuropathy or <unk>.
Sandy Macrae: S F N.
Sandy Macrae: We plan to begin patient enrollment and dosing for the S&P 503 study in mid 2025 and two.
Sandy Macrae: We anticipate having preliminary proof of efficacy data in the fourth quarter of 2026.
Sandy Macrae: We also continued to advance clinical trial authorization or Cta, enabling activities for <unk> six our epigenetic regulator for the treatment of prion disease to be delivered intravenously, using our own stack BBB.
Sandy Macrae: We're extremely proud to have been selected to present during the prestigious presidential symposium at this week's <unk> annual meeting in New Orleans.
Sandy Macrae: We look forward to showcasing a potent combination.
Sandy Macrae: Genetic regulation and capsid delivery technology in prion disease, and describing the <unk>.
Sandy Macrae: Survival benefits, we observed when administered to post symptomatic mice.
Sandy Macrae: We will also describe the sustained brain white suppression of prion protein expression in both most of nonhuman primate models supporting its potential as a one time therapeutic approach for prion disease.
Sandy Macrae: We plan to begin clinical trial enrollment and dosing for <unk> hundred six in mid 2026 and expect to have preliminary clinical data in the fourth quarter of 2026.
Sandy Macrae: In addition to the prior presentation, we have had eight abstracts accepted by <unk>.
Sandy Macrae: I am very proud of our scientists and look forward to showcasing the progression of our neurology pipeline, including advances in zinc finger epigenetic regulation. The latest innovations in Caf II capsid delivery engineering and developments and our modular integrated technology.
Sandy Macrae: Moving to our late stage Fabry program last week, we were excited to announce a number of important derisking milestones in the pathway to the anticipated BLA submission for <unk> 'twenty.
Sandy Macrae: Oh dose patients in the phase one two star study have no completed at least 52 weeks of follow up a key milestone required by the FDA for an accelerated approval regulatory pathway for <unk> 'twenty.
recorded.
Louise Wilkie: 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. Thank you. Good afternoon, everyone. Thank you for joining us on the call today.
Sandy Macrae: Importantly, preliminary analysis of the clinical data collected as of this 52 week milestone date across all 32 dose patients indicated that the mean egfr slope continue to remain positive.
And press pound when finished.
Louise Wilkie: On this call are several members of the Sangamo Executive Leadership Team, including Sandy Macrae, Chief Executive Officer, Prathyusha Duraibabu, Chief Financial Officer, and Nathalie Dubois-Stringfellow, Chief Development Officer.
Sandy Macrae: The product candidate continues to be well tolerated in a pivotal data readout is expected by the end of this quarter.
Bone disease.
Sure.
We plan to begin clinical trial enrollment and dosing for S. G five or six in mid 2026 and expect to have preliminary clinical data in the fourth quarter of 2026.
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 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 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.
Sandy Macrae: 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.
In addition to the Prime presentation, we have had eight abstracts accepted by a S. G C T I.
Sandy Macrae: 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.
I'm very proud of our scientists and look forward to showcasing the progression of her neurology pipeline, including advances in zinc finger epigenetic regulation. The latest innovations from captive capsid delivery engineering and developments in our modular integrates technology.
Sandy Macrae: 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 2026, which would facilitate a potential approval and commercial launch of SG nine 'twenty as early as the second half of that year.
Moving to our late stage Fabry program last week, we were excited to turn knowns and number of important derisking milestones in the pathway to the anticipated BLA submission for S. T 920.
Sandy Macrae: 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 her ongoing negotiations.
Oh dose patients in the phase one two star study have no completed at least 52 weeks of follow up a key milestone required by the FDA for an accelerated approval regulatory pathway for S. T 920.
Louise Wilkie: Actual results may differ materially from what we discussed today.
Louise Wilkie: These statements are subject to certain risks and uncertainties that are discussed in our filing for the SEC, specifically in our annual report on Form 10-K for the fiscal year ended December 31, 2024, and our quarterly report on Form 10-Q for the fiscal quarter ended March 31, 2025, and subsequent filings and reports that Sangamo makes from time to time with the SEC. 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.
Sandy Macrae: We remain committed to securing an anticipated partnership that is best suited to bring <unk> to fabry patients upon potential approval.
Importantly, preliminary analysis of the clinical data collected as of this 52 week milestone date across all 32 dose patients indicated that the mean egfr slope continue to remain positive.
Sandy Macrae: And that provides near term capital for Sangamo to advance our core neurology pipeline.
Sandy Macrae: As I've said before in order to execute on our plans and delivery and deliver on this promising neurology genomic medicine pipeline sangamo must be sufficiently capitalized.
The product candidate continues to be well tolerated in 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.
Sandy Macrae: We must have the resources to fund us through proof of concept in both our chronic neuropathic pain and prion disease programs, while operating a lean and efficient and focused organization.
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. 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 program and our finance. 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, STAC-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.
Sandy Macrae: In support of this strategy today, we announced the pricing of an equity offering to extender immediate cash runway. We are optimistic these funds will provide us with the bridge that we believe is necessary to secure fabric commercialization agreement.
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 C. M. C pathway. We believe we have a clear line of sight to an anticipated BLA submission as early as the first quarter of 2026, which would facilitate a potential approval and commercial launch of SG nine 'twenty as early as the second half of that year.
Sandy Macrae: We believe that this modest infusion of equity capital alongside the recent positive preclinical and regulatory Derisking of things will allow us to secure the right commercial partner for the company and for our shareholders.
Sandy Macrae: 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 stock Pvp collaboration partners with our zinc finger platform and through collaborative research agreements related to our modular.
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 her ongoing negotiations.
Sandy Macrae: We have received the $18 million upfront license fee for the first target and are eligible to earn up to $1.4 billion in additional license target fees and milestone payments across all five potential disease targets, as well as tiered royalties on potential net sales. 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 STAC BBB. Turning to our neurology pipeline programs, this quarter we continue to advance clinical study preparations for ST503, our investigational epigenetic regulator for the treatment of chronic neuropathic pain.
We remain committed to securing an anticipated partnership that is best suited to bring SG 920 to fabry patients upon potential approval.
Sandy Macrae: <unk> or platform all of which demonstrate the ongoing interest in our technology.
And that provides near term capital for Sangamo to advance our core neurology pipeline.
Sandy Macrae: We look forward to sharing more information when we can.
Sandy Macrae: With that overview of our business progress and strategy I'd now like to hand, it over to producers to try Barber, our chief Financial Officer, who will provide more context on long term vision and financial strategy to support our neurology focused mission precision.
As I've said before in order to execute on our plans and delivery and deliver on this promising neurology genomic medicine pipeline sangamo must be sufficiently capitalized.
We must have the resources to fund us through proof of concept in both our chronic neuropathic pain and prion disease programs, while operating a lean efficient and focused organization.
Barber: Thank you Sandy.
Speaker Change: I'd like to begin by emphasizing our long term vision for Sangamo and the neurology focused genomic medicine company.
In support of this strategy today, we announced the pricing of an equity offering to extender immediate cash runway. We are optimistic these funds will provide us with the bridge that we believe is necessary to secure fabric commercialization agreement.
Speaker Change: We strongly believe in the potential of our neurology pipeline to deliver transformational therapies for patients as well as significant value for our shareholders.
Speaker Change: 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.
Sandy Macrae: We're preparing for a Phase 1-2 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 fibre neuropathy, or ISFN. We plan to begin patient enrollment and dosing for the ST503 study in mid 2025 and anticipate having preliminary proof of efficacy data in the fourth quarter of 2026. 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 believe that this modest infusion of equity capital alongside the recent positive fabric clinical and regulatory Derisking of things will allow us to secure the right commercial partner for the company and for our shareholders.
Speaker Change: These milestones represent critical potential value inflection points that could significantly change the trajectory of our company.
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 Pvp collaboration partners with our zinc finger platform and through collaborative research agreements related to our modular.
Speaker Change: To achieve this important milestone we have a financial strategy with both short and long term component.
Speaker Change: In the near term the equity financing, we announced today if necessary to solidify <unk> immediate financing need.
Speaker Change: We believe the offering proceeds will extend our runway two late in the third quarter of this year.
Integrase or mint platform, all of which demonstrate the ongoing interest in our technology.
Speaker Change: More importantly, this bridge financing provides us the time, we believed that we need to secure the right pathway commercial partnership.
We look forward to sharing more information when we can.
Speaker Change: One that would fund our neurology mission in the near term and create value for shareholders over the longer term.
With that overview of our business progress and strategy I'd now like to hand, it over to tradition to try Barber, our chief Financial Officer, who will provide more context on long term vision and financial strategy to support our neurology focused mission precision.
Sandy Macrae: We are 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 observe 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 SG506 in mid-2026 and expect to have preliminary clinical data in the fourth quarter of 2026.
Speaker Change: We've already made significant progress in transforming our financial profile.
Speaker Change: In 2024.
Speaker Change: Our non-GAAP operating expenses by 50% year on year by carefully focusing the organization on our most important priority.
Thank you Danny.
I'd like to begin by emphasizing our long term vision for Sangamo as our neurology focused genomic medicine company.
Speaker Change: We are leaving no stone unturned and seeking additional cost savings and then looking at ways to further reduce operating expenses to maximize the efficiency of the go forward Urology company.
We strongly believe in the potential of our neurology pipeline to deliver transformational therapy for patients as well as significant value for our shareholders.
Speaker Change: We are committed to operating as lean as possible, while continuing to advance two near.
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.
Speaker Change: R&D programs at a steady pace.
Speaker Change: We believe this disciplined approach to capital allocation, we will ensure that we have the talent and capabilities to execute on our neurology mission, while extending our runway through a key value creating milestones.
These milestones represent critical potential value inflection point that could significantly change the trajectory of our company.
To achieve this important milestone we have a financial strategy with both short and long term component.
Sandy Macrae: I'll now hand, it back to sandy for closing remarks.
Sandy Macrae: In addition to the prion presentation, we have had eight abstracts accepted by ASGCT. I'm very proud of our scientists and look forward to 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.
Sandy Macrae: Thank you Patricia.
Sandy Macrae: I am pleased with the progress we have made this year. So far we signed our third stack BBB license agreement. This time with Lilly reinforcing that sangamo as a collaborator of choice for neurotrophic capsules.
In the near term the equity financing, we announced today, if necessary to solidify Fang and most immediate financing needs.
We believe the offering proceeds will extend our runway to late in the third quarter of this year.
Sandy Macrae: We continue to advance <unk> hundred three program for the treatment of intractable pain due to ISF N ahead at the start of the planned patient enrollment and dosing in mid 2025.
More importantly, this bridge financing provides us the time, we believe that we need to secure the right pathway commercial partnership.
Sandy Macrae: 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 dose patients in the Phase 1-2 STAR study have now completed at least 52 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 52-week milestone date across all 32-dose patients indicated that the mean EGFR slope continued to remain positive. The product candidate continues to be well tolerated and a pivotal data readout is now expected by the end of this quarter.
One that would fund our neurology mission in the near term and create values for shareholders over the longer term.
Sandy Macrae: We achieved significant clinical and CMC derisking, most students and a pathway to anticipated BLA submission for Fabry disease program.
We've already made significant progress in transforming our financial profile.
And we have advanced multiple potential business development discussions across a range of single technologies, including stack BBB, our zinc finger platform and our <unk> platform, while securing additional capital to support our near term efforts.
In 2024, we've 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 and seeking additional cost savings and are looking at ways to further reduce operating expenses to maximize the efficiency of the go forward Neurology company.
Sandy Macrae: We remain resolutely focused consulting or long term funding needs and partnering our fabry disease program in an effort to provide sustainable and long term funding for our promising neurology genomic medicine pipeline.
We are committed to operating as lean as possible, while continuing to advance our two neurology programs at a steady pace.
Sandy Macrae: In closing I would encourage you to engage with the data we will be presenting at this week's <unk> annual meeting, which will be made available on the single website once data embargoes uplifted.
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 to our key value creating milestones.
Sandy Macrae: 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 the engagement from those FDA representatives and attendants 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 BLE submission as early as the first quarter of 2026, which would facilitate a potential approval and commercial launch of SC920 as early as the second half of that year.
Sandy Macrae: These presentations will further demonstrate the scientific foundation of our pipeline and the potential of our therapeutic approach to address significant unmet medical needs.
I'll now hand, it back to sandy for closing remarks.
Sandy: Thank you Patricia.
Speaker Change: I am pleased with the progress we have made this year. So far we signed our third stack BBB license agreement. This time with Lilly reinforcing that sangamo as a collaborator of choice for neurotrophic capsules.
Sandy Macrae: As always we thank you for your continued support of Sangamo.
Speaker Change: Operator, please open the line for questions.
Sandy Macrae: Thank you.
Speaker Change: Reminder, to ask a question. Please press star one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one again, one moment, while we compile our Q&A roster.
Speaker Change: We continue to advance our S T five or three program for the treatment of intractable pain due to I S. F. N ahead at the start of the planned patient enrollment and dosing in mid 2025.
Speaker Change: We achieved significant clinical and CMC derisking milestones and a pathway to anticipated BLA submission for Fabry disease program.
Speaker Change: Our first question is going to come from the line of Maury Raycroft with Jefferies. Your line is open. Please go ahead.
Sandy Macrae: 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 negotiation. 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.
Speaker Change: Hi, This is James on for more I congrats on the progress.
Speaker Change: And we have advanced multiple potential business development discussions across a range of Sangamo technologies, including stack BBB, our zinc finger platform and our main platform for securing additional capital to support our near term efforts.
Speaker Change: Thanks for taking our questions.
Speaker Change: 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 Egfr slope, where there will be other quality of life endpoints also do you plan to show an MHS analysis to contextualize the improvements youre seeing on Egfr and if so can you provide some color on the <unk> plan and P value threshold.
Speaker Change: We remain resolutely focused consulting or long term funding needs and partnering our fabry disease program in an effort to provide sustainable and long term funding for our promising neurology genomic medicine pipeline.
Speaker Change: Okay.
Sandy Macrae: 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.
Natalie: Thank you for your questions Natalie can.
Can you talk to these yes, yes. So we are really happy to have those other patients.
Speaker Change: In closing I would encourage you to engage with the data we'll be presenting at this week's E. S. GCT annual meeting, which will be made available on a single website once data embargoes uplifted.
Natalie: Pass the one year milestone required by the FDA for an accelerated approval pathway.
Natalie: We will of course share in the top line data.
Speaker Change: These presentations will further demonstrate the scientific foundation of our pipeline and the potential of our therapeutic approach to address significant unmet medical needs.
Natalie: Our updated mean egfr slope and we will comment on additional information at a later date.
Sandy Macrae: 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 the bridge that we believe is necessary to secure a fabric commercialisation agreement. We believe that this modest infusion of equity capital alongside the recent positive fabric clinical and regulatory de-risking 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 StackBBB collaboration partners with our Zinkfinger platform and through collaborative research agreements related to our modular integrase or Mint platform, all of which demonstrate the ongoing interest in our technology.
Speaker Change: As always we thank you for your continued support of Sangamo.
Natalie: It's fair to say not to leave that at that time. We will also have 19 patients will have achieved two years data absolutely no. It really is a robust data set we understand the excitement people have to see this data in <unk> and we will show as much as possible as we can.
Speaker Change: Operator, please open the line for questions.
Speaker Change: Thank you.
Speaker Change: A reminder to ask a question. Please press star one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one again, one moment, while we compile our Q&A roster.
Natalie: The topline data as we prepare for the BLA submission.
Speaker Change: Our first question is going to come from the line of Maury Raycroft with Jefferies. Your line is open. Please go ahead.
Natalie: Yes.
Natalie: As to the statistical analysis, we are not commenting at this time, but we have.
Speaker Change: Hi, This is James on for more congrats on the progress and thanks for taking our questions.
Speaker Change: 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 egfr slope or there will be other quality of life endpoints also do you plan to show an NHS analysis to contextualize the improvements youre seeing on in Egfr and if so can you provide some color on the system plan and P value threshold.
Natalie: We.
Natalie: I agree with the FDA.
Natalie: Type B meeting with them. So we feel like it's a standard way to doing it and have agreed with the FDA on the pathway.
Speaker Change: Got it thanks, and just one really quick follow up now that you're sort of in the final stages of securing a potential <unk> partnership how many partners are you potential partners are you currently in conversations and what can you really tell us about those ongoing discussions at this point.
Sandy Macrae: We look forward to sharing more information when we can.
Prathyusha Duraibabu: With that overview of our business progress and strategy, I'd now like to hand it over to Prathyusha Duraibabu, our Chief Financial Officer, who will provide more context on long-term vision and financial strategy to support our neurology-focused mission. 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 pipelines 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.
Natalie: Thank you for your questions Natalie.
Natalie: You talked to these yes, yes. So we are really happy to have those other patients now have passed the one year milestone required by the FDA for an accelerated approval pathway.
Speaker Change: You can imagine that that's not something that we're able to comment, but it's fair to say there are multiple.
Natalie: We will of course share in the top line data.
Speaker Change: Potential partners that were talking to and.
Speaker Change: 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.
Natalie: Updated mean, egfr slope and we will comment on our additional information at a later date.
Natalie: It's fair to say not leave that at that time. We'll also 19 patients will have achieved two years data absolutely. It really is a robust data set we understand the excitement people have to see this data in food.
Speaker Change: <unk> an approval so.
Speaker Change: We were very pleased with that result, we were very pleased with how.
Speaker Change: Straightforward the interaction with the agency was and how helpful. Beware.
Natalie: And we will show as much as possible as we can of the topline data as we prepare it for the BLA submission.
Speaker Change: Because I know for people like you predictability of the process and the approval process is very important.
Prathyusha Duraibabu: 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 Sangamo'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 fabric commercial partnership. One that would fund our neurology mission in the near term and create values for shareholders over the longer term. We've already made significant progress in transforming our financial profile.
Natalie: Yes.
Natalie: As to the statistical analysis, we are not commenting at this time, but we have.
Speaker Change: Got it. Thank you so much for taking our questions I'll hop back in the queue.
Speaker Change: Thank you one moment for our next question.
Speaker Change: With the comment you made with it I agree with the FDA on our.
Speaker Change: Our next question is going to come from the line of Nicole <unk> with Trust. Your line is open. Please go ahead.
Speaker Change: Type B meeting with them. So we're we feel like we it's a standard way to doing it and have agreed with the FDA on the password.
Nicole: Hi, good afternoon, Thanks for taking my question.
Speaker Change: So we have previously mentioned.
Speaker Change: Got it thanks, and just one really quick follow up now that you're you're sort of in the final stages of securing a potential fabry partnership how many partners are you potential partners are you currently in conversations and what what can you really tell us about those ongoing discussions at this point.
Nicole: It sounds like some of that now.
Speaker Change: Hey Congress.
Nicole: Conversation.
Nicole: Hum.
Nicole: Okay.
Nicole: Any way given the macro landscape.
Nicole: Well Angie.
Nicole: Added pressure around cell and gene therapy.
Speaker Change: You can imagine that that's not something that we're able to comment, but it's fair to say there are multiple.
Prathyusha Duraibabu: In 2024, we reduce the non-GAP 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're 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.
Nicole: Sure.
Nicole: So Nicole.
Speaker Change: Sir your line was quite hard to hear here I think I heard you ask us to comment on hemophilia, a and the agencies.
Speaker Change: Potential partners that were talking to.
Speaker Change: And.
Speaker Change: 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.
Speaker Change: Gene therapy is that fair.
Speaker Change: Fabry disease.
Speaker Change: So.
Speaker Change: Uh huh.
Speaker Change: I kind of alluded to in the last answer the remarkable thing about the agency interaction. We just went through was hope unremarkable. It was and how helpful. The agency was we.
Speaker Change: We were very pleased with that result, we're very pleased with how.
Speaker Change: Straightforward the interaction with the agency was and how helpful. Beware.
They send you.
Speaker Change: Because I know for people like you predictability of of the process in the in the approval process is very important.
Speaker Change: Answers to your written questions before the meeting they came in on time and they were very detailed the had the all the right people at the most senior level in the meeting and we got the answers the formal.
Sandy Macrae: I'll now hand it back to Sandy for closing remarks. Thank you, Prathyusha. I'm pleased with the progress we have made this year so far. We signed our third STAC BBB license agreement, this time with Lilly, reinforcing that Sangamo is a collaborator of choice for neurotropic capsids. We continue to advance our ST503 programme for the treatment of intractable pain due to ISFN ahead of the start of a planned patient enrolment and dosing in mid 2025. We achieved significant clinical and CMC de-risky milestones in our pathway to anticipated BLA submission for our Fabry disease program. And we have advanced multiple potential business development discussions across a range of Sangamo technologies, including StackVVV, our Syncfinger platform, and our Mint platform, while securing additional capital to support our near-term efforts.
Speaker Change: Got it. Thank you so much for taking our questions I'll hop back in the queue.
Speaker Change: Okay. Thank you one moment for our next question.
Speaker Change: Minutes of the meeting within a week of the meeting where they could take up to 30 days and so in every form of interaction. We've had we have found them.
Speaker Change: Our next question is going to come from the line of Nicole <unk> with Truest. Your line is open. Please go ahead.
Nicole: Hi, good afternoon, Thanks for taking my question.
Speaker Change: So you previously mentioned.
Speaker Change: <unk> and how they are looking at our Fabry disease program and when we look at the interactions that.
Speaker Change: Hello, Hi.
Speaker Change: Hey, Praful conversation.
Speaker Change: Got it.
Speaker Change: Pfizer had across the he may they also seem to be very positive about that so we are not seeing any change I'm sure like you are very aware of the.
Speaker Change:
Trojan anyway, given the macro landscape of change that at <unk> and <unk>.
Speaker Change: Any added pressure around calling gene therapy.
Nicole: So Nicole.
Speaker Change: The announcements.
Speaker Change: Sir your line was quite hard to hear here I think I heard you ask us to comment on hemophilia a.
Speaker Change: Announcements.
Speaker Change: The concern about funding in the boat.
Speaker Change: 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 Change: And the agencies attitude to gene therapy is that fair, yeah, and fabry disease.
Sandy Macrae: We remain resolutely focused on solving our long-term funding needs and partnering our Fabry disease program in an effort to provide sustainable and long-term funding for our promising neurology genomic medicine pipeline.
Speaker Change: Okay.
Speaker Change: So.
Speaker Change: That's a question.
Speaker Change: Uh huh.
Speaker Change: And follow up so given the current administration concern around drug pricing, how do you think that will impact.
Speaker Change: As I kind of alluded to in the last answer the remarkable thing about the agency interaction. We just went through was home unremarkable. It was and how helpful. The agency was we the.
Speaker Change: <unk> therapy at home.
Sandy Macrae: In closing, I would encourage you to engage with the data we'll 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 Change: Paul.
Speaker Change: Yeah.
Speaker Change: I think there's a long way to go in those discussions around drug pricing.
Speaker Change: They send you.
Speaker Change: Answers to your written questions before the meeting they came in on time and they were very detailed the had the all the right people at the most senior level in the meeting and we got the answers the formal <unk>.
Speaker Change: I think that the pharmaceutical industry is a very important industry in America.
Speaker Change: And I'm sure as someone who sits on the board to bio that buyer will be hunting very detailed and hopefully productive conversations with the.
Speaker Change: <unk> of the meeting within a week of.
Operator: Operator, please open the line for questions. Thank you. As a reminder, to ask a question, please press star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again. One moment while we compile our Q&A roster.
Speaker Change: The meeting where they could take up to 30 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 and when we look at the interactions that.
Speaker Change: Administration to find a good way through this.
Speaker Change: Great. Thank you so much Amy.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: Our next question is going to come from the line of <unk> with Wells Fargo. Your line is open. Please go ahead.
Speaker Change: Hi, Thanks for taking our question. This is quantum for you on the just to clarify on the prior questions.
Speaker Change: Pfizer had across the he may they also seem to be very positive about that so we're not seeing any change I'm sure like ours, you are very aware of of the <unk>.
Murray Raycroft: Our first question is going to come from the line of Murray Raycroft with Jeffreys. Your line is open. Please go ahead.
Speaker Change: The regular path for Fabry do you still plan to file based on a 52 week Egfr data and.
James: Hi, this is James on for more. Congrats on the progress. And thanks for taking your question.
James: 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? 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?
Whereas this baseline required for Egfr. Thank you.
Speaker Change: <unk> instruments and the concern about funding in a boat.
Speaker Change: Yes, absolutely.
Speaker Change: 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 Change: We are.
Speaker Change: Absolutely pursuing the agreement we had with the FDA to use.
Speaker Change: Okay, and then one quick.
Speaker Change: Question on.
Speaker Change: And follow up so.
Speaker Change: <unk> 52 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.
Speaker Change: So given the current administration concern around drug pricing, how do you think that will impact gene therapy at all.
Nathalie Dubois: Thank you for your questions.
Nathalie Dubois: Nathalie, can you talk to these? Yes. So we're really happy to have those all the patients that 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. It's fair to say, Nathalie, that at that time, we'll also have 19 patients who will have achieved two years' data. Absolutely. So it really is a robust data set. 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 prepare it for the BLE submission.
Speaker Change: Paul.
Speaker Change: Yeah.
Speaker Change: I think there's a long ways to go in those discussions around drug pricing.
Speaker Change: And we are seeing it.
Speaker Change: 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 Change: I think that the pharmaceutical industry is a very important industry in America and I'm sure as someone who sits on the board to bio that buyer will be having very detailed and hopefully productive conversations with the administration to <unk>.
Speaker Change: It's very important that we emphasize home much supportive data there is beyond the Egfr we have agreement with the agency for submission based on that as the primary endpoint, but I think the thing that convinces the agency.
Speaker Change: Find a good way through this.
Speaker Change: Great. Thank you so much Amy.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: Has made this such a compelling mentioned for US is every author indication.
Speaker Change: Next question is gonna come from the line of Union Xu with Wells Fargo. Your line is open. Please go ahead.
Index of success in the trial is going in the same direction, so pain scores <unk> scores.
Speaker Change: Hi, Thanks for taking our question. This is quantum for you on the just to clarify on the prior questions.
Nathalie Dubois: Yes, and as to the statistical analysis, we are not commenting at this time, but we have agreed with the FDA on our type B meeting with them. So, you know, we feel like it's a standard way to doing it, and I've agreed with the FDA on the past.
Speaker Change: On the regular path for Fabry do you still plan to file based on 52 week Egfr data and.
Speaker Change: SF 36, yes.
Speaker Change: General safety is very good very well tolerated.
Speaker Change: Patients in general are feeling better and.
Speaker Change: And if that's it whereas this baseline required for Egfr. Thank you.
Speaker Change: Also got level remain.
Speaker Change: Hi.
Speaker Change: In all patients and some of these patients are no two four plus years for US yes, yeah Yep.
Speaker Change: Yes, absolutely.
Speaker Change: Where we are.
Speaker Change: Absolutely pursuing the agreement we had with the FDA to use 52 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.
Speaker Change: Sorry, maybe you already comment on it.
James: Thanks.
James: And just one really quick follow-up.
Speaker Change: That's like versus baseline required for Egfr. Thank you.
Nathalie Dubois: Now that you're sort of in the final stages of securing a potential FABRI partnership, how many potential partners are you currently in conversations and what can you really tell us about those ongoing discussions? 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 BLE and approval. So we were very pleased with that result.
Speaker Change: We will be looking at a variety of statistical methods to describe the difference in Egfr.
Speaker Change: But we said.
Speaker Change: We are seeing it.
Speaker Change: 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 Change: The last week or the week before in her announcement.
Speaker Change: The Egfr remains positive so the slope of Egfr remains positive.
Speaker Change: And it's very important that we emphasize home much supportive data there is beyond the Egfr we have agreement with the agency for submission based on that as the primary endpoint, but I think the thing that convinces the agency and Uh Huh.
Speaker Change: Got it thank you so much.
Speaker Change: Thank you one moment for our next question.
Speaker Change: Our next question comes from the line of Lee Santos with.
Speaker Change: H C. Wainwright. Your line is open. Please go ahead.
Lee Santos: Hello, everyone. Thank you for taking our questions and congratulations.
Nathalie Dubois: 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. Got it.
Speaker Change: Made this such a compelling medicine for US is every author indication.
Speaker Change: The selection of your talks with the presidential symposium an impressive presence.
Speaker Change: An index of success in the trial is going in the same direction, so cadence scores fabry scores.
Lee Santos: GTT.
Lee Santos: Regarding <unk>.
Lee Santos: I don't know if I missed this but are the patients who were on E. R. A T still off <unk> or was there any were there any relapse.
Speaker Change: <unk> 36, and the general safety is very good very well tolerant tolerated.
James: Thank you so much for taking our questions. I'll hop back. Thank you. One moment for our next question.
Speaker Change: The patient in general are feeling better and or.
Speaker Change: Our alpha Gal level remain.
Lee Santos: Yes, all patients the 18 patients that started on the I T out of our 32 patient are still up.
Nicole Germino: Our next question is going to come from the line of Nicole Germino with Truist. Your line is open. Please go ahead.
Speaker Change: Hi.
Speaker Change: In all patients and some of these patients are no two four plus years four plus years, yeah Yep.
Nicole Germino: Hi, good afternoon, thanks for taking my question. So you've previously mentioned potential interest of partners from FAB and Jumei. Has the pace of those conversations gone, have they changed in any way, given the macro landscape with changes at FDA? And do you see any added pressure around cell and gene therapies in particular?
Lee Santos: Very well.
Lee Santos:
Speaker Change: Sorry, maybe you already comment on it and if that thick versus baseline required for egfr. Thank you.
Lee Santos: Four.
Lee Santos:
I know that you mentioned you are not commenting so much on the.
Lee Santos: Our statistical analysis plan, but the Egfr slope you said it remains positive but is there any.
Speaker Change: We'll be looking at a variety of statistical methods to describe the difference in Egfr.
Speaker Change: But we said.
Lee Santos: Thresholds.
Lee Santos: We should be looking at as a minimum or is there any actual statistical significance.
Speaker Change: Last week or the week before in her announcement the Egfr remains positive so the slope of the AGR remains positive.
Nathalie Dubois: So, Nicole, sorry, your line was quite hard to hear here. I think I heard you ask us to comment on Haemophilia A and the agency's attitude to gene therapy. Is that fair? Yeah. So...
Lee Santos: I think prior questions, we're asking about this already but.
Lee Santos: I'm not sure if I was clear on that.
Speaker Change: Got it thank you so much.
Lee Santos: We're not commenting on this right now, but what I can tell you is the mean egfr slope remains positive.
Speaker Change: Thank you one moment for our next question.
Speaker Change: Our next question comes from the line of Lee Santos with.
Nathalie Dubois: 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. They send you answers to your written questions before the meeting. They came in on time and they were very detailed. They had 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 30 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.
And you will I know you are very aware of that.
Speaker Change: H C. Wainwright. Your line is open. Please go ahead.
Lee Santos: <unk>.
Speaker Change: Hello, everyone. Thank you for taking our questions and congratulations.
Lee Santos: Normal people.
So if people without fabry disease, there's a gradual decline over time in their egfr in fabry patients. There is an accenture to decline in Egfr. There is not fully addressed by <unk> and it remains negative in patients who are on <unk>.
Speaker Change: The selection of your talk for the presidential Symposium, an impressive presence at AOS GCG.
Speaker Change: Regarding <unk>.
Speaker Change: I don't know if I missed this but are the patients who were on E. R. A T still all off E Archie or.
Lee Santos: And so as part of our analysis, we'll be looking at can be looked at the change from baseline and we'll be looking at compare to.
Speaker Change: Or are there any relapse.
Speaker Change: Yes, all patients the 18 patients that started on the I T out of our 32 patient of steel of E. R. A T.
Lee Santos: The data for other forms of treatment for fabry disease.
Lee Santos: We would not put the press release.
Speaker Change: Okay, well and.
Lee Santos: We did about.
Speaker Change: For.
Lee Santos: The ear.
Lee Santos: The Egfr remains positive if we werent confident the results were.
Speaker Change: I know that you mentioned you are not commenting so much on D.
Nathalie Dubois: And when we look at the interactions that Pfizer had across the he may, they also seem to be very positive about that. So we are not seeing any change. I'm sure, like us, you are very aware of the announcements 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 Change: Our statistical analysis plan, but.
Lee Santos: Agency, we're looking for.
Speaker Change: The Egfr slope you said it remains positive but is there any.
Lee Santos: And that's helpful. Just to wrap up that and quantify my my question as well for the statistical analysis plan, even if youre not giving us more detail.
Thresholds.
Speaker Change: We should be looking at as a minimum or is there any actual statistical significance.
Speaker Change: You are submitting.
Speaker Change: All the data available from all patients.
I think prior questions, we're asking about this already but.
Speaker Change: For the BLA submission, but which ones will be used for the <unk>.
Speaker Change: I'm not sure if I was clear on that.
Speaker Change: We're not commenting on this right now, but what I can tell you is the mean egfr slope remains positive.
Speaker Change: We used for the statistic analysis from clients with all of them or is it just a portion of them.
Nathalie Dubois: 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 ex-US? 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 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 them.
It's all of them.
Speaker Change: And you will I know you are very aware that in.
Speaker Change: So at the submission for the BLA submission will be 32 patients at one year of 19 patients at two years.
Speaker Change: Normal people.
Speaker Change: Sorry people without fabry disease, there's a gradual decline over time in their egfr in fabry patients. There is an exaggeration to decline in Egfr. There is not fully addressed by E. Archie and it remains negative in patients who are on <unk>.
Speaker Change: That's helpful. Thanks, so much.
Speaker Change: Our pleasure.
Speaker Change: Thank you one moment for our next question.
Speaker Change: Our next question is going to come from the line of Lukas <unk> with RBC capital markets. Your line is open. Please go ahead.
Kathy: Hi, Thanks, so much for taking our question. This is Kathy answer Luca and this will be a question of stack BBB.
Speaker Change: And so as part of our analysis, we'll be looking at can be looked at the change from baseline and we'll be looking at compare to.
Speaker Change: And.
The data for other forms of treatment for fabry disease.
Speaker Change: Recall, the homology medicines had one of the first capsid deliver systematically which could cross the blood brain barrier and then let's go to that in a mouse model it dose dependent reduction in biomarker, both systematically and in the CNS. So when can we expect to see a similar to will impact with respect BBB catfish.
Nicole Germino: Great. Thank you so much, Andy.
Speaker Change: We would know couteau the press release.
Yanan Zhu: Thank you, and one moment for our next question. Our next question is going to come from the line of Yanan Zhu with Wells Fargo. Your line is open. Please go ahead. Hi. Thanks for taking our question.
Speaker Change: That we did about the room the E R.
Speaker Change: The Egfr remains positive if we weren't confident that the results were what the agency we're looking for.
Kuan-An: This is Kuan-An for Yanan. Just to clarify on the prior questions, on the regular path for Fabry, do you still plan to file based on 52-week EGFR data? And is Static versus Baseline required for EGFR? Thank you. Yes, absolutely. We are absolutely pursuing the agreement we had with the FDA to use 52-week EGFR for the entirety of the patient population in the Phase 1-2 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 Change: And also do you expect similar pre conditioning or other catheter approaches I E. No prophylactic steroids.
Speaker Change: And that's helpful. Just wrap up that and quantify my my question as well for the statistical analysis plan, even if youre not giving us more detail.
Speaker Change: It will be very helpful. Thank you so much.
Speaker Change: So we can really only comment on her own capsid.
Speaker Change: You are submitting.
Speaker Change: All the data available from all patients for <unk>.
Speaker Change: And its effectiveness.
Speaker Change: For the BLA submission, but which ones will be used for the which ones will be used for the statistic analysis plan as with all of them or is it just a portion of them.
Speaker Change: We have not made the decision whether we would use steroid treatment.
Speaker Change: The clinical trial yet.
Speaker Change: We're very pleased with the effectiveness of stack BBB.
Speaker Change: It's all of them.
Speaker Change: We're very pleased to have seen.
Speaker Change: So at the submission for the BLA submission will be 32 patients at one year of 19 patients at two years.
Speaker Change: The result in monkeys across with a variety of cargos.
Speaker Change: And I really commend Joe too.
Speaker Change: That's helpful. Thanks, so much.
Speaker Change: Look at the look at the presentation that Brian will be good thing.
Speaker Change: Our pleasure thank.
Speaker Change: Thank you one moment for our next question.
Speaker Change: ESG UCT it really showcases both the effectiveness of stack BBB and the importance of the rate card.
Speaker Change: Our next question is going to come from the line of Lukas <unk> with RBC capital markets. Your line is open. Please go ahead.
Nathalie Dubois: 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 the trial is going in the same direction. So, pain scores, Fabry scores, SF36. Yeah, 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.
Kathy: Hi, Thanks, so much for taking our question. This is Kathy answer Luca and this will be a question of stack BBB.
Speaker Change: In it.
Speaker Change: To be honest, that's why it's so pleasing.
Speaker Change: Literally astellas and Genentech, who are all big names in the neuroscience field have chosen to license our capsid.
Speaker Change: And we.
Speaker Change: Recall, the homology medicines had one of the first capsid deliver systematically which could cross the blood brain barrier and then let's go to that in a mouse model a dose dependent reduction in biomarker, both systematically and in the CNS. So when can we expect to see a seminar until impact with respect BBB catfish.
Speaker Change: Directly.
Speaker Change: Yes.
Speaker Change: What we will present really as per <unk> benefit of the treatment and disease mouse model and sustained brand wide suppression of prion protein expression in both smiles and nonhuman primate model.
Speaker Change: And also do you expect similar pre conditioning or other capsid approaches I E. No prophylactic steroids.
Speaker Change: Supporting its potential as a one time therapeutic approach for prion disease. So it's very important that we have well.
Speaker Change: It will be very helpful. Thank you so much.
So we can really only comment on her own capsid.
Speaker Change: We have tested it in and HP and it's extremely well tolerated.
Speaker Change: And the presidential Symposium I think there's two presentations being given notice something like 4000 abstracts that were.
Speaker Change: And its effectiveness.
Speaker Change: We have not made the decision whether we would use steroid treatment in the clinical trial yet.
Nathalie Dubois: And some of these patients are now out at 4-plus years? 4-plus years, yeah. Sorry, maybe you already commented on it. And is Static versus Baseline required for EGFR? Thank you. We'll be looking at a variety of statistical methods to describe the difference in EGFR. But we said last week or the week before in our announcement that the EGFR remains positive. So the slope of the EGFR remains positive. Got it. Thank you so much. Thank you.
Speaker Change: Presented and it's a great.
Speaker Change: We're very pleased with the effectiveness of stack BBB.
Speaker Change: It's a great credit to the work of <unk>.
Speaker Change: Bryan Zeitler and data to gel in Victoria, and Brian's team the vote on so much work too.
Speaker Change: We're very pleased to have seen.
Speaker Change: The result in monkeys across with a variety of cargos.
Speaker Change: Drive.
Speaker Change: And I really commend Joe too.
Speaker Change: Something that hopefully next year will be.
Speaker Change: A potential curative.
Speaker Change: Look at the look at the presentation that Brian will be giving at.
Speaker Change: Potential.
Speaker Change: Treatment for patients with this awful awful disease.
Speaker Change: ESG UCT, where it really showcases both the effectiveness of stack BBB and the importance of the right cargo in it and to be honest, that's why it's so pleasing that.
Speaker Change: Okay, great well be looking forward to the presentation our CTC.
Speaker Change: Yeah.
Nathalie Dubois: One moment for our next question.
Speaker Change: Thanks, again, and congrats on the presentations and abstracts.
Luis Santos: Our next question comes from the line of Luis Santos with HC Wainwright. Your line is open. Please go ahead. Hello, everyone.
Speaker Change: Literally astellas and Genentech, who are all big names in the neuroscience field have chosen to license our capsid.
Speaker Change: Thank you and again, if you would like to ask a question. Please press star one on your telephone.
Speaker Change: Our next question comes from the line of Gena Wang with Barclays. Your line is open. Please go ahead.
Nathalie Dubois: 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 relapses? Yes, all patients, the 18 patients that started on ERT out of our 32 patients are still of ERT. Okay, well, and for I know that you mentioned 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?
Speaker Change: Directly.
Speaker Change: Yes.
Speaker Change: What we will present really profound survival benefit of the treatment and disease mouse model and sustained brain wide suppression of prion protein expression in both smiles and nonhuman primate model.
Speaker Change: Ms <unk> your line might be on mute.
Speaker Change: Supporting its potential as a one time therapeutic approach for prion disease. So it's very important that we have well.
Speaker Change: We have tested it in and HP and it's extremely well tolerated.
Speaker Change: And you know the the presidential Symposium I think there's two presentations being given notice something like 4000 abstracts that were <unk>.
Speaker Change: Thank you and ladies and gentlemen, if you would like to ask a question. Please press star one one.
Speaker Change: Hi, I'm showing no further questions at this time and I would like to hand, the conference back over to Luis <unk> for closing remarks.
Speaker Change: Presented and it's a great.
Speaker Change: It's a great credit to the work of Brian.
Speaker Change: Bryan Zeitler and data to gel in Victoria, and Brian's team that have all done so much work too.
Speaker Change: 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 updated on our future developments. Thank you.
Drive.
Speaker Change: Something that hopefully next year will be.
Speaker Change: A potential curative sorry.
Speaker Change: Potential treatment for patients with this awful awful disease.
This concludes today's conference call. Thank you for participating you may now disconnect everyone have a great day.
Nathalie Dubois: I think there are prior questions we're asking about this already, but I'm not sure if I was clear on that. We're not commenting on this right now. But what I can tell you is the mean EGFR slope remains positive. 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 the change from baseline and we'll be looking at compared to the data for other forms of treatment for Fabry disease.
Speaker Change: Okay, great well, we're looking forward to the presentation of Citibank.
Speaker Change: And thanks.
Speaker Change: Thanks, again, and congrats on the presentations and abstracts.
Speaker Change: Thank you and again, if you would like to ask a question. Please press star one on your telephone.
Speaker Change: Our next question comes from the line of Gena Wang with Barclays. Your line is open. Please go ahead.
Speaker Change: MS. Wang your line might be on mute.
Speaker Change: Thank you and ladies and gentlemen, if you would like to ask a question. Please press star one one.
Nathalie Dubois: We would not have put out the press release that we did about the ERT. The EGFR remains positive if we weren't confident that the results were what the agency were looking for. 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 statistical analysis plan? Is it all of them, or is it just a portion of them? It's all of them.
Speaker Change: Hi, I'm showing no further questions at this time and I would like to hand, the conference back over to Luis <unk> for closing remarks.
Speaker Change: 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 updated on our future developments. Thank you.
Speaker Change: This concludes today's conference call. Thank you for participating you may now disconnect everyone have a great day.
Nathalie Dubois: So the submission for the BLA submission will be 32 patients at one year and 19 patients at two years. That's helpful. Thanks so much. Our pleasure. Thank you. One moment for our next question.
Luca Issi: Our next question is going to come from the line of Luca Issi with RBC Capital Markets. Your line is open. Please go ahead. Hi, thanks so much for taking our question.
Nathalie Dubois: This is Cassian for Luca, and this will be a question on SPAC-BBB. And we recall that homology medicines 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 SPAC-BBB capsid? And also, do you expect similar preconditioning to other capsid approaches, i.e. no prophylactic steroids? Any comments? That would be very helpful. Thank you.
Nathalie Dubois: So, we can really only comment on our own CAPCID 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 STAC BBB. We're very pleased to have seen the result in monkeys across with a variety of our cargos. And I would really commend you all to look at the presentation that Brian will be giving at ASGCT, where it really showcases both the effectiveness of STAC BBB and the importance of the right cargo in it. 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 CAPCID directly.
Nathalie Dubois: Yes, and what we will present really is profound survival benefit of the treatment and disease mouse model and sustained brainwide suppression of prion protein expression in both mouse and non-human pride model, supporting its potential as a one-time 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.
Nathalie Dubois: 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 uh it's a great credit to the work of uh Brian Zeitler and David Agel and Victoria and Brian's team that have all done so much work to um um drive uh something that hopefully next year will be a potential curative sorry a potential treatment for patients with this awful awful disease We'll be looking forward to the presentation at CTG. Thanks again, and congrats on the presentation and. Thank you.
Operator: And again, if you would like to ask a question, please press star 11 on your telephone.
Gina Wang: Our next question comes from the line of Gina Wing with Barclays. Your line is open. Please go ahead.
Operator: Ms. Wang, your line might be on mute. Again, ladies and gentlemen, if you would like to ask a question, please press star 1 1.
Louise Wilkie: 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. 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.