Q2 2022 Sangamo Therapeutics Inc Earnings Call
The conference will begin shortly to raise your hand during Q&A you can dial star one one.
[music].
Okay.
Operator: The conference will begin shortly.
Operator: Hi, good evening team.
Ladies and gentlemen, thank you for standing by and welcome to <unk> second quarter 2022 earnings Conference call. At this time all participants are in a listen only mode. After the speaker's presentation there'll be a question and answer session to ask a question during the session I need to press Star one one I would now like to turn the call over to your host Luis Wilkey you may begin.
Operator: To raise your hand during Q&A, you can dial star 1 1.
Operator: This is Harshita on for Gina.
Operator: Ladies and gentlemen, thank you for standing by and welcome to Sangamo's second quarter 2022 earnings conference call.
You don't name I'm Louise we'll keep some of my thoughts President Investor Relations and corporate communications. Thank you for joining us on the call today.
On this call are several members of the Sangamo executive leadership team.
Sandy Macrae, Chief Executive Officer, Mark Mcclung, Chief operating officer for T shirt, your fiber Chief Financial Officer, Jason <unk>, Chief Scientific Officer, Bob Sharp and the development and Bettina Cockroft Chief Medical Officer.
Slides from our corporate presentation can be found at our website sangamo com under the investors and media section of the events and presentations page.
This call includes forward looking statements regarding <unk> current expectations.
These include but are not limited to statements relating to the therapeutic and commercial potential of our product candidate.
The anticipated plans and timelines the tanker market and our collaborators for initiating in conducting clinical trials.
And screening of patients and presenting clinical data.
Execution of our corporate and funding strategy.
Most of our product candidates.
Advancements of preclinical programs through the clinic.
The sufficiency of our resources our.
Our 2022 financial guidance.
Key milestones and catalysts and other statements that are not historical facts.
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 the fiscal year ended December 31 2021.
Supplemented by our quarterly report on Form 10-Q for the fiscal quarter ended June 32022.
The forward looking statements made today are made as of this date and we undertake no duty to update such information, except as required by law.
This call we discussed our non-GAAP operating expenses.
Reconciliation of this measure to our GAAP operating expenses can be found in today's press release, which is available on our website.
Now I'd like to turn the call over to our CEO Sandy Macrae. Thank you Luis and good afternoon to everyone on the call.
Second we will continue to advance our mission to create potentially transformative genomic medicines for patients using our innovative technologies in the second quarter of 2022 with momentum across our clinical stage programs as well as significant preclinical progress.
Operator: At this time, all participants are in a listen-only mode.
Operator: Thanks for taking our questions.
This quarter, we received endorsement from the pre phase one two study safety monitoring committee to advance our differentiated and wholly owned gene therapy program for the treatment of adults with type two disease from the dose escalation fees into the expansion fees.
We already have multiple patients in screening and expect to dose two patients later this month.
We continue to be pleased with the progress of this important program and look forward to sharing updated patient data lead to this quarter.
<unk> 003, formerly known as SCR for 451 P. Six our zinc finger nuclease modified autologous cell therapy for the treatment of sickle cell disease formally completed the transition back to being a wholly owned Sangamo program on June 20, concluding.
Operator: We had one on TX200.
Operator: And then a follow up on Fabry if I may.
Concluding our collaboration agreement with Sanofi.
Operator: After the speaker's presentation, there'll be a question and answer session.
I am really grateful percentage <unk> cooperation on the transition.
Operator: To ask a question during the session, you need to press star 1 1.
Operator: I would now like to turn the call over to your host, Louise Wilkie.
We expect to dose the next patient in the phase <unk> study in the third quarter of this year and look forward to sharing an increment two data update in the second half of 2022.
Operator: You may begin.
Louise Wilkie: Good afternoon.
Operator: Could you remind us again, what you hope to glean from the biopsy data for TX200? If I remember correctly, I think Jason, one of the things you mentioned previously was, you know, biopsy data will show whether the Tregs are trafficking and accumulating in the kidneys.
Momentum continued behind TX 200, or Hulu into <unk> car T Reg cell therapy product candidates and easily <unk> mismatched kidney transplantation.
Louise Wilkie: I'm Louise Wilkie, Sangamo's Vice President of Investor Relations and Corporate Communication.
Operator: So I was hoping you could elaborate on this point.
Louise Wilkie: Thank you for joining us on the call today.
Operator: And then I'll ask the Fabry question after.
Following the dosing of the first patient in March and what we believe to be in the first in the car T Reg cell therapy field.
Louise Wilkie: On this call are several members of the Sangamo executive leadership team, including Sandy Macrae, Chief Executive Officer, Mark McClung, Chief Operating Officer, Prathyusha Duraibabu, Chief Financial Officer, Jason Fontenot, Chief Scientific Officer, Rob Schott, Head of Development, and Bettina Kovacs, Chief Financial Officer.
Most importantly, the patient remains well more than four months post transfusion.
Manufacturing is complete for the second patient who received a kidney transplant in July and dosing as planned poorly to this quarter.
I continue to be thankful to the investigators for helping us break ground and this potentially transformative field of cell therapy.
Okay.
Louise Wilkie: This quarter, we received endorsement from the Fabry Phase 1-2 Studies Safety Monitoring Committee to advance our differentiated and wholly-owned gene therapy programme for the treatment of adults with Fabry disease from the dose-escalation phase into the expansion phase, formally completed the transition back to being a wholly owned Sangamo program on June 28, concluding our collaboration agreement with Sanofi.
Regarding the phase III hemophilia, a gene therapy study by third parties.
Louise Wilkie: I'm really grateful for Sanofi's cooperation on the transition.
Louise Wilkie: We expect to dose the next patient in the Phase I-II study in the third quarter of this year, and look forward to sharing an incremental data update in the second half of 2022. Momentum continued behind TX200, our wholly owned autologous CAR Treg cell therapy product candidate, in HLA-A2 mismatched kidney transplantation, following the dosing of the first patient in March in what we believe to have been the first in the CAR Treg cell therapy field. Most importantly, the patient remains well more than four months post-transfusion. Manufacturing is complete for the second patient who received a kidney transplant in July, and dosing is planned for later this quarter.
<unk> to resume dosing in the trial in the third quarter of 2022, with a pivotal readout estimators and leak <unk> III or early 'twenty four.
Louise Wilkie: I continue to be thankful to the investigators for helping us break ground, in this potentially transformative field of cell therapy.
Finally, I was thrilled to attend and see the reaction to Sangamo as important contributions at the American Society of gene and cell therapy, <unk> annual meeting in Washington, DC and meat.
Louise Wilkie: Regarding the Phase III haemophilia A gene therapy study, Pfizer advised us that it expects to resume dosing in a fine trial in the third quarter of 2022 with a pivotal readout estimated in late 2023 or early 2024.
Louise Wilkie: Finally, I was thrilled to attend and see the reaction to SANGAM's important contributions, at the American Society of Gene and Cell Therapy, ASGCT, annual meeting in Washington, D.C. in May. We presented a total of eight posters and presentations, detailing our highly innovative early-stage science, which we see as essential to feeding our ongoing pipeline of genomic medicines.
We presented a total of eight posters and presentations detailing our highly innovative early stage science, which we see as essential to <unk>, our ongoing pipeline of genomic medicines.
I am proud with the versatility of our scientific platforms, coupled with the strategic and focused use of research and development capabilities, which are driving progress in our pursuit of indications of unmet needs.
Louise Wilkie: I am proud of the versatility of our scientific platforms, coupled with the strategic and focused use of research and development capabilities, which are driving progress in our pursuit of indications of unmet need.
I recognized the difficult market conditions being experienced across the industry.
Louise Wilkie: I recognise the difficult market conditions being experienced across the industry. We believe SANGAM's current financial position and the variety of options we have to raise further funds, positions us well to continue developing new transformational medicines for patients in need and to generate long-term value for our shareholders.
We believe Sangamo is current financial position and the variety of options. We have to reach further funds positions us well to continue developing new transformational medicines for patients in need and to generate long term from what you've put our shareholders.
Louise Wilkie: And with that, I'd like to turn the call over to our Head of Development, Rob Shaw, who will discuss the data from our clinical programs in more detail.
Operator: Jason, as you're an immunologist, can you help us with what we may see in the kidney?
And with that I'd like to turn the call will hurt our head of development offshore who will discuss the data from our clinical programs in more detail Bob.
Rob Shaw: Rob.
Rob Shaw: Thank you, Sandy.
Operator: Yeah, I think you have that exactly right.
You Sandy and good afternoon to everyone on the call.
Rob Shaw: Good afternoon to everyone on the call.
Operator: That's what we'll be looking for.
The second quarter marked another period of strong execution in the clinic and we are pleased with the progress across our programs.
Rob Shaw: The second quarter marked another period of strong execution in the clinic, and we are pleased with the progress across our programs.
Operator: You know, there are a few things.
Rob Shaw: In the Phase 1-2 STAR study, evaluating Esoprazole Gene Cytoparvovac, or ST920, our wholly-owned gene therapy program for the treatment of febrile disease in adults, two additional patients were withdrawn from enzyme replacement therapy, or ERT. Out of the five patients that began the STAR study on ERT, a total of four have now been successfully taken off ERT, one each in Cohorts 1 and 2 and two in Cohort 3, with discussions in progress to withdraw the fifth and final patient from ERT. I'm pleased to report that all four withdrawn patients have stable biomarkers, and an estimation of investigators have not required resumption of ERT.
Operator: One will be localization of the cells for the kidney.
In the phase <unk> study evaluating <unk> for the <unk> or <unk>, our wholly owned gene therapy program for the treatment of Fabry disease in adults two additional patients were withdrawn from enzyme replacement therapy or ERP.
Operator: And the other will be evidence that the cells are having some effect on the local microenvironment in the kidney.
Operator: So those are both things that we'll be looking for using both immunohistochemistry and in situ hybridization.
Operator: And we'll, you know, we'll be excited to share data as we assemble it into a meaningful data package.
Operator: And more simply or prosaically, we'll also be ensuring that there's no signs of kidney rejection or inflammatory damage to the kidneys.
Operator: So safety is very important in studies like this.
Out of the five patients that began the STAAR study on <unk> a total of four have now been successfully taken off our team.
Operator: Makes sense.
Operator: Thank you.
One each in cohorts, one and two and two and cohort three with discussions in progress to withdraw the fifth and final patient from ERP.
Operator: And you had a question on Fabry.
Operator: Yes, yes.
I am pleased to report.
Yet all four withdrawn patients has stable biomarkers and an estimation of the investigators have not required resumption of ERP.
Operator: Thanks, Andy.
Rob Shaw: As Sandy mentioned, this quarter, the Safety Monitoring Committee for the STAR Study endorsed, progressing from the Phase I-II escalation phase into the expansion phase at the 5e13 vector genome per kilogram dose level. We expect to dose two patients imminently and have multiple patients in screening, including, both male and female candidates.
Operator: So on Fabry, a quick clarification, the two additional patients that came off ERT, which cohorts were these patients in?
As Sandy mentioned this quarter the safety monitoring committee for the STAAR study endorsed progressing from the phase <unk> escalation phase into the expansion phase at the five E. <unk> vector genomes per kilogram dose level, we expect to dose two patients imminently and have multiple patient screening, including both EMEA.
Operator: Apologies, I missed that bit and prepared remarks.
And female candidates.
Rob Shaw: A total of 16 study sites are now open and recruiting, including the first sites in Canada, Italy, and Australia. The safety and efficacy from the dose escalation phase has been closely followed by investigators, and interest in participation in the trial of this wholly-owned therapy is growing among the Fabry community, including, most importantly, with patients.
A total of 16 study sites are now open and recruiting including the first sites in Canada, Italy and Australia.
The safety and efficacy from the dose escalation phase has been closely followed by investigators and interest and participation in the trial of this wholly owned therapy is growing among the fabry community, including most importantly with patients.
We look forward to providing updated results from the phase <unk> study during the second half of 2022, including at the Society for the study of inborn errors of metabolism assets IEM annual symposium, taking place at the end of August .
Rob Shaw: We look forward to providing updated results from the Phase I-II STAR Study during the, second half of 2022, including at the Society for the Study of Inborn Errors of Metabolism, SSIEM Annual Symposium taking place at the end of August.
Operator: And also, if you could level set, you know, what kind of data can we expect at the conference later this month?
Operator: You know, follow up and any other metrics or details you can highlight.
Operator: That would be helpful.
Rob Shaw: We also continue to actively plan for potential Phase III study and are engaging with health, authorities, patient advocacy groups, and investigators.
Operator: Thank you so much.
We also continue to actively plan for potential phase III study and are engaging with health authorities patient advocacy groups and investigators.
As Sandy mentioned on June 28, we assumed full control of CIBC Zero-zero III, formerly known as our 44536 for the treatment of sickle cell disease. A promising program are excited to have back as part of our wholly owned pipeline.
Rob Shaw: As Sandy mentioned, on June 28, we assumed full control of BIVV003, formerly known as, SAR445136, for the treatment of sickle cell disease, a promising program we're excited to have back as part of our wholly-owned pipeline.
Operator: Thank you.
Operator: Bettina, can you help us with this?
Operator: Bettina?
In the phase one tube precision one study three of the four patients dosed with product candidates manufactured using the previous manufacturing process continues to be free available crews events since dosing.
Rob Shaw: In the Phase I-II Precision I Study, three of the four patients dosed with product candidates, manufactured using the previous manufacturing process continue to be free of vaso-occlusive events since dosing.
Operator: Absolutely.
During this quarter the patient who had achieved the lowest level of fetal hemoglobin post infusion experienced the second Vasal occlusive crisis with patient has now fully recovered.
Rob Shaw: During this quarter, the patient who had achieved the lowest level of fetal hemoglobin post-infusion, experienced a second vaso-occlusive crisis. This patient has now fully recovered.
During this quarter manufacturing our product candidates using improved methods progressed in the phase two study.
Rob Shaw: During this quarter, manufacturing of product candidates using improved methods progressed, in the Phase I-II Study. These improved manufacturing methods have been shown in internal experiments to increase, the number of long-term progenitor cells in the final product. We expect to dose the next patient in this study in the third quarter of this year and, look forward to sharing an incremental Phase I-II data update before the end of the year.
These improved manufacturing methods have been shown in internal experiments to increase the number of long term progenitor cells in the final product.
We expect to dose the next patient in this study in the third quarter of this year and look forward to sharing an incremental phase one two data update before the end of the year.
In addition, phase III, enabling activities, including manufacturing readiness actively continue.
Rob Shaw: In addition, Phase III enabling activities, including manufacturing readiness, actively, continue.
Progress continued in the phase <unk> steadfast study our groundbreaking wholly owned TX 200 car T. Reg cell therapy candidate for the prevention of immune mediated rejection, an HLA <unk> mismatched kidney transplantation from a living donor.
Rob Shaw: Progress continued in the Phase I-II Steadfast Study, our groundbreaking, wholly-owned TX200, CAR Treg cell therapy candidate for the prevention of immune-mediated rejection in HLA A2 mismatched kidney transplantation from a living donor. During our announcement in March that we had successfully dosed what we believe to, have been the first-ever patient with an engineered CAR Treg, the product candidate dose continues to be well-tolerated more than four months post-infusion with no treatment-related adverse events.
Following our announcement in March that we had successfully done what we believe to have been the first ever patient within engineered car T. Reg.
<unk> candidate dose continues to be well tolerated more than four months post infusion with no treatment related adverse events.
Rob Shaw: We are thrilled to report this important update as we believe it is a historic first.
We are thrilled to report this important update as we believe it is.
A historic first.
This quarter, we completed manufacturing for the second patient of a steadfast study. This patient recently received a kidney transplant and is expected to be later in the third quarter of 2022.
Rob Shaw: This quarter, we completed manufacturing for the second patient in the Steadfast Study. This patient recently received a kidney transplant and is expected to be dosed later in the third, quarter of 2022.
Rob Shaw: We hope to dose the third and final patient in Cohort 1 by the end of 2022, and we plan, to provide further updates once we have a meaningful package of data to disclose from our first complete cohort.
We hope to dose the third and final patient in cohort one by the end of 2022, and we plan to provide further updates once we have meaningful package of data to disclose from our first complete cohort.
Rob Shaw: In addition, this quarter we are pleased to announce that the European Commission has, granted Orphan Medicinal Product Designation to TX200 for the treatment in solid organ transplantation following a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. To qualify for Orphan Designation, a treatment must be intended for a life-threatening or, chronically debilitating disease affecting fewer than 5 in 10,000 people. Importantly, no satisfactory method of treatment must exist, or if such a method exists, the, treatment must be of significant benefit to patients.
In addition, this quarter, we are pleased to announced that the European Commission has granted orphan medicinal product designation to TX 200 for the treatment in solid organ transplantation. Following a positive opinion from the European Medicines Agency Committee for orphan medicinal products too.
To qualify for orphan designation, a treatment must be intended for a life threatening or chronically to build that debilitating disease affecting fewer than five and 10000 people.
Importantly, no satisfactory method of treatment must exist or such a method exists the treatment must be of significant benefit to patients.
The achievement of this important regulatory milestone takes us one step closer to our goal of creating a transformative therapy that can reduce the likelihood of organ rejection.
Rob Shaw: The achievement of this important regulatory milestone takes us one step closer to our, goal of creating a transformative therapy that can reduce the likelihood of organ rejection, relieve the overall patient burden, and minimize the risks that come with immunosuppressive medications.
The overall patient burden and minimize the risks that come with immunosuppressive medications.
Rob Shaw: As a reminder, this is the first in a pipeline of potential CAR Treg therapies that are working, to develop to address a range of autoimmune conditions.
Operator: Sorry about that.
As a reminder, this is the first and a pipeline of potential car T. Reg therapies that are working to develop to address a range of autoimmune conditions.
Rob Shaw: In addition to TX200, Sangamo has an active preclinical pipeline with multiple candidates, in development to treat inflammatory bowel disease and multiple sclerosis alongside our efforts to progress the allogeneic cell therapy platform.
In addition to TX 200, Sangamo has been active preclinical pipeline with multiple candidates in development to treat inflammatory bowel disease and multiple sclerosis, alongside our efforts to progress.
Allogeneic cell therapy platform.
Finally regarding the phase III <unk> trial evaluating credit teams that are part of <unk>, an investigational gene therapy for hemophilia, a pfizer advised us that it continues to expect to resume dosing this quarter once all necessary conditions are met.
Rob Shaw: Finally, regarding the Phase 3 Affine Trial, evaluating Grutter Gene Fetal Parvovac, an, investigational gene therapy for hemophilia A, Pfizer advised us that it continues to expect to resume dosing this quarter once all necessary conditions are met, including approval of an updated trial protocol by regulatory authorities.
<unk> approval of an updated trial protocols by regulatory authority <unk>.
Rob Shaw: Pfizer has provided guidance that a pivotal data readout is expected toward the end of, 2023 or in early 2024.
Pfizer has provided guidance study pivotal data readout is expected towards the end of 2023 or in early 2024.
I will now turn the call over to our Chief Scientific Officer, Jason Fontanella for updates on our preclinical research programs Jason.
Jason Fontenot: I will now turn the call over to our Chief Scientific Officer, Jason Fontenot, for updates, on our preclinical research programs.
Operator: So we now have a total, just as a reminder, a total of nine patients dosed, five of whom were dosed as they were on ERT. And of these five patients, four patients have now withdrawn from ERT. And that is one patient in cohort one, the only one who was on ERT upon entering the study.
Jason Fontenot: Jason?
Jason Fontenot: Thank you, Rob, and good afternoon, everyone.
Operator: And one ERT subject in cohort two who was on ERT and two out of the three patients in cohort three who were on ERT.
Thank you, Rob and good afternoon, everyone.
Jason Fontenot: I'm very pleased to report that we continue to leverage Sangamo's cutting-edge genomic, engineering and cell therapy platforms to advance both wholly owned and partnered programs for the clinic.
Operator: And just as a reminder, cohort four patients, the two patients who were dosed in cohort four are naive patients.
I am very pleased to report that we continue to leverage thing most cutting edge genomic engineering cell therapy platform to advance both wholly owned and partnered programs towards the clinic.
Operator: And so what we will be, to answer your second half of the question, what we'll be presenting, later this month at SSIEM is incremental data. We will have data on six patients.
Operator: Who are those based on the cutoff?
Operator: And as a reminder, we had four patients presented in that world.
Operator: And so we'll have incremental data also from those four patients.
Operator: And there are other potential times to show data later in the year.
We're also making remarkable progress in expanding our proprietary genomic engineering tool kit and in developing novel AAV capsid to enable more effective therapeutically relevant delivery of those tools.
Jason Fontenot: We are also making remarkable progress in expanding our proprietary genomic engineering, toolkit and in developing novel AAV capsids to enable more effective, therapeutically relevant delivery of those tools. We shared a portion of this exciting progress at the ASGCT annual meeting in May where Sangamo, scientists presented seven poster presentations and one oral presentation, demonstrating advancements across our research portfolio and highlighting the diversity and versatility of our genomic engineering platform. Notably, two of the ASGCT presentations showcased our world-class AAV capsid evolution platform.
Operator: Absolutely.
We shared a portion of this exciting progress at the <unk> annual meeting in May where Sam of scientists presented seven poster presentations and one oral presentation demonstrating.
Operator: We're planning additional data cuts and additional incremental data to be shared later at conferences, later in this year.
Operator: Great.
Demonstrating advancements across our research portfolio and highlighting the diversity and versatility of our genomic engineering platform.
Operator: Thank you so much.
Operator: One moment for our next question.
Notably two of the <unk> presentations.
Okay for our World class AAV capsid evolution platform the.
Jason Fontenot: The initial focus of this work is delivery to the central nervous system. We anticipate that more effective and efficient AAV-mediated delivery to the CMS will allow, us to deploy our genomic medicines to an even larger set of high-value neurodegenerative and neurodevelopmental diseases.
Operator: Our next question comes from Ben Burnett with Stiefel.
The initial focus of this work is delivering to the central nervous system.
We anticipate that more effective and efficient AAV mediated delivery to the CNS will allow us to deploy our genomic medicines.
Operator: Your line is open.
Operator: Hi.
Being a larger step high value Neurodegenerative and Neurodevelopmental diseases.
Operator: This is Carolina Ibanez.
Jason Fontenot: The ASGCT presentations highlighted significant and exciting progress in the development of, engineered AAV capsids facilitating broad CNS coverage using both cerebral fine fluid administration and intravenous therapy.
Operator: I'm on Ben Burnett.
<unk> presentation highlighted significant and exciting progress in the development of engineered AAV capsid facilitating.
Operator: Thank you for taking our questions.
CNS coverage using both cerebral spinal fluid administration and intravenous administration.
Operator: One follow-up on the previous question of Fabry disease.
Jason Fontenot: Administration.
Operator: Could you comment on the background of the patient's dose in cohort 3 and how their characteristics, compare with patients in previous cohorts in terms of classical Fabry disease and biomarkers levels?
Regarding our genomic engineering platform this year, the FCC CE Mark the unveiling of our.
Jason Fontenot: Regarding our genomic engineering platform, this year's ASGCT marked the unveiling of, our compact and highly efficient zinc finger-based editor and the deployment of our epigenetic zinc finger regulators to engineer T cells and other immune cell types. The zinc finger-based editor can facilitate simultaneous inactivation of multiple genes without introducing double-stranded breaks, thus reducing the probability of chromosomal translocation events. A notable aspect of our base editor is its compact architecture, allowing packaging into a single AAV vector.
Highly efficient fingerpaint editor and the deployment of our epigenetic regulators to engineer T cells and other immune cell types.
The zinc finger based editor and facilitate simultaneous and activation of multiple gene without introducing double stranded breaks thus.
Thus, reducing the probability of chromosomal translocation of events.
Notable aspect of our base is its compact architecture, allowing packaging into a single AAV vector. This is a critical requirement for the efficient use in many therapeutic applications, particularly in GB.
Jason Fontenot: This is a critical requirement for the efficient use in many therapeutic applications, particularly in vivo.
Epigenetics finger regulators can be used for multiplexed engineered many cell sites, including car T cell and car T regs and have the potential.
Jason Fontenot: The epigenetic zinc finger regulators can be used for multiplex engineering of many cell types, including CAR-T cells and CAR-T regs, and have the potential to be deployed for both ex vivo and in vivo applications.
Essentially be deployed for both ex vivo and in vivo application.
Operator: So you're looking for some kind of background of the patients in cohort 3?
I am honored and privileged to represent the dedicated and innovative team of scientists and thinking about it.
Jason Fontenot: I am honored and privileged to represent the dedicated and innovative team of scientists at Sangamo.
Operator: Correct.
Convinced that our robust pipeline of preclinical programs and platform capabilities will serve <unk> well in the year.
Jason Fontenot: I'm convinced that our robust pipeline of preclinical programs and platform capabilities will serve Sangamo well in the years to come and deliver on our mission to translate groundbreaking science into medicines that transform patients' lives.
Operator: Yeah.
And deliver on our mission to translate ground, breaking science and our medicines that transform patients' lives.
Jason Fontenot: I will now turn the call over to our chief financial officer, Prathusha Durabadu, for an overview of our financial results.
I will now turn the call over to our Chief financial officer of future through our body part overview of our financial results.
Prathyusha Duraibabu: Prathusha?
Sure.
Thank you, Jason and good afternoon.
Prathyusha Duraibabu: Thank you, Jason, and good afternoon.
Operator: Padina.
Our financial results for this quarter are available in the press release issued this afternoon, which can be found on our website.
Prathyusha Duraibabu: Our detailed financial results for this quarter, are available in the press release issued this afternoon, which can be found on our website. We ended the quarter with approximately $364 million in cash, cash equivalents, and marketable, securities, which we believe will enable continued execution across our platform and programs.
Operator: Sure.
Operator: So I think I would point out that we are presenting detailed data at the end of this month.
We ended the quarter with approximately $364 million in cash cash equivalents and marketable securities, which we believe will enable continued execution across our platform and programs.
Operator: What I can say is reiterating what I've mentioned.
As we navigate through these turbulent market conditions, we continue to exercise financial diligence and focused our investment in three key areas.
Prathyusha Duraibabu: As we navigate through these turbulent market conditions, we continue to excite financial, diligence and focus our investment in three key areas, advancement of our clinical programs, including FEBRI and Tx200, progression of our preclinical CAR-T reg and CNS pipeline, and optimization of our in-house manufacturing capabilities.
Operator: Some of these patients are on ERT, some are not.
Operator: The disease has different potential mutations, so there are different mutations for each, of these patients.
Advancement of our clinical programs, including <unk> 200 progression of our preclinical cocky right emptiness pipeline.
Prathyusha Duraibabu: Additionally, since the start of the second quarter through today, we have raised approximately $40 million in net proceeds under our previously announced at-the-market offering program. This reflects our proactive approach to balance capital raises in these uncertain markets and speaks to the continued investor interest in our company.
And optimization of our in house manufacturing capabilities.
Additionally, since the start of the second quarter through today, we have reached approximately $40 million and net proceeds under our previously announced at the market offering program.
This reflects our proactive approach to balance capital leases in these uncertain markets and speaks to the continued investor interest in our company.
Prathyusha Duraibabu: Turning to our 2022 full-year guidance, we continue to expect our 2022 full-year non-GAAP operating expenses to be between $280 million to $310 million. This rate excludes estimated non-cash stock-based compensation expense of approximately $40 million.
Turning to our clinical need to full year guidance.
We continue to expect our 2022 full year non-GAAP operating expenses to be between $280 million to $310 million.
This excludes estimated noncash stock based compensation expense of approximately $14 million.
Prathyusha Duraibabu: We expect a significant portion of our operating expenses to be invested in continued advancement, of our lead programs, including FEBRI Phase III planning activities, Phase I-II activities for, Tx200, and preclinical work in CAR-T regs and neurology genome engineering indications.
We expect a significant portion of our operating expenses to be invested and continued advancement of our lead programs, including <unk> planning activities <unk> activities for kids to 100, and preclinical work and cocky right and urology did not mean hearing indications. We also expect to continue our investment in sickle cell disease.
Prathyusha Duraibabu: We also expect to continue our investment in sickle cell disease following transition of the, program back to Sangamon.
Following the transition of the program in fact sustainable.
Sandy Macrae: I'll now turn it over to our CEO, Sandy, for closing remarks.
I'll now turn it over to our CEO Sandy for closing remarks.
Sandy Macrae: Thank you, Patricia.
Operator: Some of the patients do have underlying classic symptoms of Fabry, and I think this is probably, not the location to go into a lot of detail here, but suffice it to say that the in-depth data will be presented at SSIEM at the end of this month.
Thank you Patricia.
2022 continues to be a year of significant momentum for sangamo.
We are a clinical stage genomic medicine company with powerful innovative science strong clinical execution capabilities and deep experience developing.
Sandy Macrae: 2022 continues to be a year of significant momentum for Sangamon.
Operator: Was there a particular attribute or characteristic that you were trying to understand?
Sandy Macrae: We're a, clinical-stage genomic medicine company with powerful innovative science, strong clinical execution capabilities, and deep experience developing INDs supported by in-house manufacturing to meet the needs of our growing pipeline.
Operator: Well, you already touched on whether it was representing, you know, classic Fabry characteristics, and also if you could comment on biomarker levels.
Supported by in house manufacturing to meet the needs of our growing pipeline.
We continue to bring these capabilities together to create cope among the patients were working to serve.
Future for the company and the potential for long term sustainable power used for our shareholders.
I am very grateful to our leadership team and all my Sangamo colleagues for their dedication hard work towards our mission of creating transforms <unk> new medicines for patients.
We look forward to sharing anticipated key milestones and catalysts throughout the second half of 2022, including additional phase two data from refractory study.
Sandy Macrae: We look forward to sharing anticipated key milestones and catalysts throughout the second half of 2022, including additional Phase 1-2 data from our Fabry study, dosing of additional patients in our Phase 1-2 study for sickle cell disease, and Phase 1-2 TX200 CAR-T reg study, and Pfizer's resumption of dosing in the Phase 3 trial in haemophilia A.
Operator: So I think we'll hold the biomarker levels for SSIEM, and we hope you'll be able to attend, or view those.
Operator: And then as we've now also closed cohort 4, and then this month are about to dose another, two patients with six that we're queuing up, you can understand that there'll be a constant flow of data throughout the rest of the year.
Dosing of additional patients in our phase one two study for sickle cell disease.
Operator: Understood.
<unk>, TX 200 car T Rex study and Pfizer's resumption of dosing in the phase III trial in hemophilia a.
Sandy Macrae: So at this time, we'd like to open it up for questions.
Operator: Looking forward to your next data update.
So at this time, we'd like to open it up for questions.
Operator: Operator.
Operator.
Hello, Ladies and gentlemen, if you have a question or a comment at this time. Please press the star one one we will pause for a moment, while we compile our Q&A roster.
Operator: Ladies and gentlemen, if you have a question or a comment at this time, please press the star 1-1.
Operator: Thank you.
Operator: We'll pause for a moment while we compile our Q&A roster.
Our first question comes from Greg Harrison with Bank of America. Your line is open.
Operator: Our first question comes from Greg Harrison with Bank of America.
Operator: One moment for our next question.
Good afternoon, thanks for taking our questions.
Operator: Your line is open.
Operator: Our next question comes from Ritu Baral-Wakawan.
First off.
Should we be expecting from the next update.
From the TX 200 program.
Do you have a sense of how much follow up duration and what other data points would you really need to see.
To establish proof of concept there.
Greg Harrison: Good afternoon.
Thank you for your question.
Greg Harrison: Thanks for taking our questions.
Operator: Your line is open.
Very pleased to be driving forward with TX 200.
Im delighted the first patient has done so well and is four months so to say.
Greg Harrison: First off, what should we be expecting from the next update from the TX200 program?
Operator: Good afternoon, guys.
What's the plan going ahead.
As we've indicated in the call we plan to complete the first dose cohort.
Greg Harrison: Do you have a sense of how much follow-up duration and what other data points would you really need to see to establish proof of concept there?
Operator: Thanks for taking the question.
In 2022.
And we will provide further updates once we have a meaningful package of data to disclose from that first complete dose cohort.
We understand the excitement around this program.
There are a number of T Reg companies.
We all see car T. Regs is potentially important medicine. So we look forward to sharing the results of the appropriate time.
Got it thanks, and then one more question if I can.
How does your base editor compare with the CRISPR based approach.
Could you be a differentiator.
Jason one for you.
Yes.
Sandy Macrae: Thank you for your question.
Thank you for the question.
We're excited about about the capabilities of our base editor.
We think that it adds another dimension to the toolkit that we have.
That includes Nucleases now based editors transcriptional regulators that Kenny.
Either.
Kris or decrease the expression of genes in a tunable way.
So.
Our view is that having all of these tools is critically important to creating relevant therapeutics.
The differentiating features of our base editor is its compact size.
That allows us to deploy it using a variety.
Of delivery vehicles, including.
The delivery.
So.
Given given that range of tools.
We think that we are able to address almost any.
Any need when it comes to genetic engineering, and we're really excited about it.
Great. Thanks, again for taking the question.
One moment for our next question.
Our next question comes from Jonathan <unk> with Wells Fargo. Your line is open.
Hi, Thanks for taking my questions.
So maybe on the Fabry <unk>.
Study.
Im curious about the reason behind eschar.
Escalating the dose cohort for those.
We've seen data from the first two dose cohorts and I thought the data was.
Quite impressive even with those low doses and then you did say cohort three obviously and.
For the optional cohort <unk> elected to further dose escalate I'm wondering what are you trying to optimize.
With this higher dose and what is the desired outcome there. Thanks.
Thanks for the question.
We agree with you the first two doses looked very good.
Operator: Can you remind me of the target enrollment, for your 5V13 expansion cohort in Fabry?
We'd always planned to go up to 513.
We decided to do it prudently and four steps.
Operator: And what is your target profile for that patient group, really sort of the same question as cohort three, but really applied to the expansion cohort?
We're looking forward to showing the data from the.
The <unk> trial as soon as possible through a series of medical meetings later this year that we will.
Operator: And then I have a follow-up on Fabry-CMC.
Show more of the data.
The safety profile was very very benign unremarkable.
Operator: Yes, Bettina.
Operator: Yes, thank you.
Operator: So the expansion phase of the study, is going to enroll up to around 30 patients. We are targeting on enrolling not only male patients, but also female patients.
Lotus to fulfill the <unk> expense for the expansion of the dose exploration.
Got it got it that's super helpful.
Sandy Macrae: We're very pleased to be driving forward with TX200 and delighted that the first patient has done so well and is four months out as safe.
And on the TX 200, just.
Quick question about <unk>.
The.
The safety profile I think you mentioned that the first patient has been treated and followed.
So far there hasn't been any aes concerning.
That's great to hear.
Of course, we were familiar with car T and Crs and all those kind of aes associated with car T car.
<unk> is a very different kind of therapy.
What might be the aes of interest that you are.
Activity monitoring.
Rob Shaw: Rob, what's the plan going ahead?
Thanks.
Thank you.
Rob.
We haven't seen any treatment associated aes, so far but one might expect.
Rob Shaw: As we've indicated in the call, we plan to complete the first dose cohort in 2022, and we will provide further updates once we have a meaningful package of data to disclose from that first complete dose cohort.
Tensely cytokine release types of reactions.
Rob Shaw: We understand the excitement around this program and that there are a number of Treg companies and that we all see CAR Tregs as potentially important medicines.
Its an autologous product so that's not very likely but those are the things that we're monitoring for.
If the if the body's reacting to it.
Production of an engineered.
Car T Reg, even though it's the patient's own car T, but I want to emphasize that we have not had any treatment associated.
Rob Shaw: So we look forward to sharing the results at the appropriate time.
Adverse events, but.
It's the reason is the reason we do these studies.
Greg Harrison: Got it.
Very methodological way and take time between each patient isn't it.
Greg Harrison: Thanks.
It is we're very cautious because this is this is a groundbreaking.
Revolutionary therapy.
Greg Harrison: And then one more question, if I can.
Liver khaki rag, and we'll understand the full impact of that as we progressed with the trial.
Got it thank you for taking my questions.
Greg Harrison: How does your base editor compare with the CRISPR-based approach and where could you be differentiated?
One moment for our next question.
Our next question comes from Nicole <unk> with Trust your line is open.
Jason Fontenot: Jason, this sounds one for you.
Hi, This is there any sign and one for Nicole.
Jason Fontenot: Yes.
Regarding fabry disease you.
Do you have any sense sites in Canada, Italy, and Australia can you maybe talk about the market of approximately $4 5 billion in those geographies. If successful then.
And if you are successful how do you plan on commercializing the gene therapy.
And then a follow up.
Okay. So the sense of there's two questions one about the clinical study on one of the.
Commercialization so wanted to Bettina if you take the clinical study in marketing through the second absolutely. So thank you for the question. Yes, we have opened sites now in additional countries.
Jason Fontenot: Thank you for the question.
Jason Fontenot: We're very excited about the capabilities of our base editor.
Jason Fontenot: We think that it adds another dimension to the toolkit that we have that includes nucleases, now base editors, transcriptional regulators that can either increase or decrease the expression of genes in a tunable way.
And as you point out that Italy, and Australia, and Canada. We have now 16 sites open and our Fabry study, which positions us well for enrollment in our <unk>.
Jason Fontenot: So, you know, our view is that having all of these tools is critically important to creating relevant therapeutics.
Jason Fontenot: One of the differentiating features of our base editor is its compact size. And that allows us to deploy it using a variety of delivery vehicles, including AAV delivery.
Our expansion phase that we are now entering.
Jason Fontenot: So, you know, given that range of tools, we think that we are able to address almost any need when it comes to genetic engineering.
I will hand, it over to Mark <unk>.
Jason Fontenot: And we're really excited about it.
We've seen a great deal of interest from patients absolutely in fact, the early data we have shown.
Yeah.
The interactions with the investigators.
<unk>.
<unk>.
A lot of interest from patients we are now screening patients as we go beyond.
Operator: This will be the first opportunity, to enroll female patients and patients who may have cardiac involvement or renal involvement predominantly.
Beyond the classic male patients who were part of the initial dose escalation criteria. We are now open to enroll female patients patients with <unk> with.
With cardiac and renal involvement and such.
There is interest from these patient populations.
So just picking up from Bettina.
Operator: So this will allow us to really cover, the essential aspects of Fabry disease.
Obviously as patina is sort of summarize our commitment is really to ensure that we.
Successfully execute against the expansion cohort and plan for our phase III.
Operator: So it's more of a sort of a clinical presentation, rather than any particular mutation subtype that you want to diversify for? That's correct.
Fabry disease is not a disease, that's just unique to United States. It's a disease that it comes out a variety of different markets.
So both the way we are approaching our clinical trial and the way we are approaching our plans for commercialization take that into consideration.
As you know we have not.
Guided any specifics in terms of our plans for <unk>.
For commercialization, but we will do that at the appropriate time.
Greg Harrison: Great.
Great. Thanks.
Secondly on collaboration.
Maybe can you talk about high level about your partner program progress.
Kind of which part of the platform getting more attention from partners.
We stay focused on thank you.
Operator: Thanks again for taking the questions.
So one of the blessings about having.
Our unique technology platform as it were constantly being approached.
With people exploring ways that they can collaborate with us.
Whether that be in terms of our gene engineering capabilities that Jason briefly touched on whether that be opportunities.
To work together on on capsid development or taxes that could be applied to our CNS.
Grants.
And so that's a key part of R. R.
Our focus from a business development standpoint, but in addition, as we've talked about in the past we've raised about $815 million.
Potentially $6 billion to $7 billion in.
And royalties.
That come from partners debt.
Approach us about using utilizing our technology.
To take that technology into areas that we wouldn't otherwise do and so I think the biogen and Novartis continues to be.
Great examples of that.
And as we continue to confirm and validate our progress with our technology. We would expect other companies to approach us with ideas that they might have in terms of how they could utilize our technology and we'd be pleased to have those conversations.
Operator: One moment for our next question.
Operator: Okay, got it.
Alright, thank you.
One moment for our next question.
Our next question comes from Gena Wang with Barclays. Your line is open.
Operator: Our next question comes from Yanan Zhu with Wells Fargo.
Operator: And then any target number of females?
Hi, Good evening Kim this is Krishna on for Gena, Thanks for taking our questions.
Yanan Zhu: Your line is open.
Operator: There is no particular target, although we are seeing interest from the patient population.
One on TX 200.
And then a follow up on Fabry, if I may.
Could you remind us again, what you hope to green from that biopsy data for TX 200.
Yanan Zhu: Hi.
Operator: So we anticipate enrolling several female patients, and several patients, female patients are already in screening.
If I if I remember correctly I think Jason one of the things you mentioned previously was a biopsy data will show better.
<unk> XR trafficking and accumulating in the kidneys. So I was hoping you could elaborate on this point and then I'll ask a follow up question after.
Yanan Zhu: Thanks for taking my questions.
Jason.
Immunologist can you help us with the what we may see in the kidney.
Yes.
I think I think you have that exactly right.
That's what we'll be looking forward there are a few things.
One will be localization of the sell through the kidney.
And the other will be evidence that the cells are having some effect on the local micro environment in the kidney and so those are both things that we'll be looking for are using.
Both I mean, I guess the chemistry.
Issue hybridization and will.
It will be exciting excited to share share data.
Yes.
We assemble that into a meaningful data package.
And so more simply a procedure.
We'll also be ensuring that there is no signs of kidney rejection or inflammatory damage to the kidneys.
Safety is very important in studies with us.
Makes sense.
Yes.
Yanan Zhu: So maybe on the Fabry study, I'm curious about the reason behind escalating the dose to cohort 4 dose.
Operator: This will be the first time, that a female Fabry patient has been treated.
<unk>.
On February .
Yes, yes, thanks, Andy so on fabric a quick clarification. The two additional patients that came off the ERP, which cohorts, where those patients and apologies if I missed that bit in prepared remarks, and also if you could level set what kind of data can we expect that the conference later this month follow up and any other metrics or details you can have.
Yanan Zhu: We've seen data from the first two dose cohorts, and I thought the data was quite impressive, even with those low doses.
That would be helpful. Thank you so much.
Thank you Bettina can you help us with this.
Yanan Zhu: And then you did the cohort 3, obviously.
No.
<unk>.
Yanan Zhu: And for the optional cohort 4, you elected to further dose escalate.
Absolutely sorry about that so we now have a total just as a reminder, total nine patient dose.
Five of whom.
<unk> as they were.
On <unk>.
Of these five patients four patients have now withdrawn from ERP.
Yanan Zhu: I'm wondering what are you trying to optimize with, this higher dose and what's the desired outcome there?
Yanan Zhu: Thanks.
Rob Shaw: Thanks for the question.
That is one patient in cohort one.
The only one who was on ERP.
Upon entering the study the one TRT subjects in cohort two who goes on ERP and two out of the three patients in cohort three.
Rob Shaw: We agree with you that the first two doses look very good.
Okay.
And just.
Just as a reminder, cohort four patients the two patients who were dosed in cohort four are naive patients.
Rob Shaw: We've always planned to go up to 5E13. We decided to do it prudently in four steps, and we're looking forward to showing the data from the full trial as soon as possible.
And so what we will be second half of the question will be presenting later this month.
<unk> is incremental data.
We will have data on six patients.
Who are those based on the cutoff.
And as a reminder, we had four patients presented.
And so we will have incremental data also from those four patients and there are other potential.
Potential times to show data later in the year, absolutely. We're planning additional data cuts and additional incremental data to be shared at any time at conferences.
Rob Shaw: There are a series of medical meetings later this year that we will show more of the data. The safety profile was very, very benign, unremarkable, and that allowed us to fulfill the full expansion of the dose exploration.
Yanan Zhu: Got it.
Yes.
Sure.
Great. Thank you so much.
One moment for our next question.
Yanan Zhu: Got it.
Our next question comes from Ben Burnett with Stifel. Your line is open.
Yanan Zhu: That's super helpful.
Hi, This is Caroline Hi, Vnesheconombank, Matt. Thank you for taking our questions one follow up on the previous question on <unk> could.
Yanan Zhu: And on the TX200, just a quick question about, the safety profile. I think you mentioned that the first patient has been treated and followed, and so far there hasn't been any AEs concerning. That's great to hear.
Yanan Zhu: Of course, we're familiar with CAR T and CRS and all those kinds of AEs associated with CAR T. CAR T-REB is a very different kind of therapy, so what might be the AEs of interest that you are actively monitoring?
Rob Shaw: Thanks.
Rob Shaw: Thank you.
Could you comment on the background of the patients dosed in cohort three.
How how.
Their characteristics compared with patients and biggest cohorts in terms of classical poverty see some biomarker Naples.
So youre looking for some kind of background of the patients in cohort three.
Rob Shaw: Rob?
Correct, yes.
Bettina.
Sure. So I think I would.
Rob Shaw: We haven't seen any treatment-associated AEs so far, but one might expect potentially cytokine, release types of reactions.
Rob Shaw: It's an autologous product, so that's not very likely, but those are the things that we're monitoring for.
Rob Shaw: If the body's reacting to the introduction of an engineered CAR T-REG, even though it's the patient's own CAR T, but I want to emphasize that we have not had any treatment-associated adverse events.
Rob Shaw: It's the reason we do these studies in a very methodological way and take time between each patient, isn't it?
Pointed out that we are presenting detailed data at the end of this month.
What I can say is reiterating.
Rob Shaw: It is.
What I've mentioned some of these patients are on ERP some are not.
Rob Shaw: We're very cautious because this is a groundbreaking, revolutionary therapy to, deliver a CAR T-REG, and we'll understand the full impact of that as we progress with the trial.
Yanan Zhu: Thank you for taking my questions.
<unk> disease is.
Hi, defend potential mutations so there are different mutations for each of these patients that patients do have.
Operator: One moment for our next question.
Operator: Our next question comes from Nicole Germino with Truist.
Underlying classic symptoms.
I believe.
<unk>.
I think this is probably not the location to to go into a lot of detail here, but suffice it to say that the.
Data will be presented.
Hi.
At the end of this month was there a particular.
Attribute or characteristic that you were trying to understand.
Well you have any thoughts on windows.
Whether it was representing a classic clogs.
Characteristics and also if you could comment on biomarker Naples.
So I think we.
We will hold the biomarker levels for <unk> and <unk>.
We will view vehicle.
Tend or viewed those.
And as we've noted also dose cohort four and then this month or about to lose another two patients with six.
Sure.
Queuing up.
You can understand there will be a constant pool of data through the rest of the year.
Understood looking forward to have next data update thank you.
Thank you.
One moment for our next question.
Our next question comes from Ritu <unk> with Cowen Your line is open.
Operator: Your line is open.
Good afternoon, guys. Thanks for taking my question.
Nicole Germino: Hi, this is Nishant.
Nicole Germino: I'm on for Nicole.
Nicole Germino: Regarding fibroid disease, you have additional sites in Canada, Italy, and Australia.
Can you remind me of the target enrollment for your <unk> expansion cohort in fabry.
And what is your target profile for that debt.
That patient group.
Really sort of the same questions cover three but really applied to the expansion cohort and then I have a follow up on Fabry CMC.
Yes, good morning Bettina.
Nicole Germino: Can you talk about the market opportunity for fibroids in those geographies, if successful?
Yes. Thank you so the expansion phase of the study is going to enroll up to around 30 patients.
Nicole Germino: And if you are successful, how do you plan on commercializing the gene therapy?
Nicole Germino: And then I have a follow up.
Nicole Germino: OK, so there's two questions, one about the clinical study and one about the commercialization.
We.
Targeting on enrolling not only.
Male patients, but also female patients this will be the first opportunity to enroll female patients.
Nicole Germino: So Bettina, if you take the clinical study and Mark, then you take the second one.
Patients, who may have cardiac involvement or renal involvement predominantly.
Nicole Germino: Absolutely.
This will allow us to really cover.
Nicole Germino: So thank you for the question.
<unk>.
Nicole Germino: Yes, we have open sites now in additional countries.
So aspects of fabry disease.
Nicole Germino: And as you point out, that is Italy and Australia and Canada.
So it's more of the sort of the clinical presentation, rather than any particular mutation type that you want to diversify.
Nicole Germino: We have now 16 sites open in our Fabry study, which positions us well for enrollment in our in our expansion phase that we are now entering.
That's correct, Okay got it and then any target number of females.
There is no particular target, although we are seeing interest from this patient population. So we anticipate enrolling several female patients.
Several patients CMO.
Female patients already in screening in fact this is this will be the first time that a female appropriate patients has been treated.
Operator: And it's been interesting to learn, about their passion for treatment.
We've we've it's been interesting to learn there.
Passion for treatment they feel they've been.
Operator: They feel they've been overlooked, and that they too have an important disease.
No.
Been overlooked.
The two have an important disease and so is it will be exciting to see that data.
Operator: And so it will be exciting to see that data.
Operator: Got it.
Got it and can you comment on CMC readiness for phase III start obviously seeber said, please go into phase III with your commercial product.
Operator: And can you comment on CMC readiness for phase three start?
Operator: Obviously, you know, CBER has said, please go into phase threes with your commercial product.
Operator: You know, how are you with assay development, validation, that sort of thing?
How are you with.
Assay development validation that sort of thing.
So phase III planning is in progress I think that that's as far as we will be talking about that at this point, but suffice it to say we've had.
Operator: So phase three planning is in progress.
Operator: I think that's as far as we'll be talking about that, at this point, but suffice it to say, we've had and we're preparing for additional interactions with agencies and therefore we're well underway with phase three planning.
And we're preparing for additional interactions with agencies.
We're well underway with.
Phase III planning.
CMC got it in a more expansive question last one I promise sandy.
Operator: Got it.
Operator: And a more expansive question, last one, I promise Sandy.
One is it a more.
Operator: One of the more, I guess, spicy sessions, that ASGCT was on reimbursement.
I guess spicy session that <unk> was on reimbursement and one of the things that the European one of the European Advisor said was that they are working together to develop phase III.
Operator: And one of the things that the European, one of the European advisors said was that they are working together to develop phase three designs that were meaningful to European payers, given the challenges of gene therapies in Europe, getting them paid for in Europe, as we've seen.
Designs that were meaningful to European payers, given the challenges of gene therapy in Europe , and getting them and paid for in Europe as we've seen.
Any thoughts on that Sandy as you think about phase III harmonization and development.
Operator: Any thoughts on that, Sandy, as you think about phase three harmonization and development, you know, nothing in particular, nothing binding, but what should we be thinking about as that develops?
In particular nothing binding but.
What should we be thinking about that.
I'm going to pass the spicy question Tomorrow.
Nicole Germino: I will hand it over to Mark.
Operator: I'm going to pass the spicy question to Mark, but just before he answers, to complete the last question, the CMC readiness, the ASCII readiness, all under control, it's all set for phase three. So we're very, the team have been working hard, in the background to get all these things ready.
But just.
But just before he answers to complete the last question.
The CMC readiness <unk>, all under controls offset for phase III the <unk>.
<unk> been working hard in the back room to get all these things rented mark right.
Operator: Mark.
Operator: We've been working hard in the background to get all these things ready, Mark.
Moving working hard in the background to get told these things rented mark right.
Yeah. So thanks for the question.
Operator: Yeah, so thanks for the question.
I think we all understand the requirements of the European payer environment.
Operator: I mean, you know, I think we all understand the requirements, of the European payer environment, and I think one of the things that I feel we need to do is not only focus on the clinical endpoints around the disease itself, but the impact that this has, you know, treating the disease has on the burden of illness on society.
And I think one of the things that I feel we need to do is not only focus on the clinical endpoints around the disease itself, but the impact that this has.
Treating the disease has on the burden of illness on society.
Operator: And so again, you know, there are other competitors that are further ahead around, commercialization in Europe.
And so again there are other competitors that are further ahead around.
Commercialization in Europe .
Operator: One in particular that's guided that, you know, they really want to take a look at the overall benefit to reduction in burden and cost to the healthcare systems in Europe to justify their pricing for their gene therapy.
One in particular, that's guided that they really wanted to take a look at the overall <unk>.
Benefit to a reduction in burden and cost to the health care systems in Europe to justify their pricing for their gene therapy, and so I think thats one model that one could approach.
Operator: And so I think that's one model that, one could approach to prove that actually treating these patients defers costs that are, you know, are being expended by the same payers, obviously, in Europe, but just in other parts of the healthcare system versus, you know, whether it's hospitalizations or other treatments.
To prove the actually patient treating these patients.
First cost that are are being.
Expended by the same payers, obviously in Europe , but just in other parts of the health care system versus whether its hospitalizations or.
Or other.
Other treatments so that so I think that data is going to be very important to collect with any gene therapy program and certainly we're taking that into consideration as we evaluate the types of data that we want to collect in our studies.
Operator: So I think that data is going to be very important to collect with any gene therapy program, and certainly we're taking that into consideration as we evaluate the types of data that we want to collect in our studies.
Operator: Great.
Operator: Very helpful.
Great very helpful. Thank you guys.
Okay, one moment for our next questions.
Our next question comes from Lucas Lee with RBC. Your line is open.
Operator: Thank you, guys.
Operator: And one moment for our next question.
Excellent. Thank you for taking our questions. This is Lisa answer Luca.
Operator: Our next question comes from Luca Issi with RBC.
Congrats on all the progress on the quarter.
Just a couple on fabry.
Wondering if you can remind us again, what the criteria is for stopping your tea.
Operator: Your line is open.
And I believe you mentioned in the prepared remarks that you are in discussions with them.
Operator: Oh, excellent.
Patients who is looking to.
<unk> I was just wondering if.
They are also in the cohort three group.
Thank you.
Nicole Germino: And Bettina, we've seen a great deal of interest from patients.
So katina criteria for stopping PRT and who is the fifth patient.
So the criteria for stopping.
Nicole Germino: Absolutely.
Really have not seen.
Blind.
In detail in the protocol intentionally to leases.
Nicole Germino: In fact, the early data we have shown and the the the interactions with the investigators have shown us that there is a lot of interest from patients.
Phase one two study open to the investigator.
Conjunction with the patient.
Nicole Germino: We are now screening more and more patients as we go along. And this is beyond the classic male patients who were part of the initial dose escalation criteria.
And so it is criteria.
Thought that those.
Both entities.
Fly when they when they discussed this in conjunction with ourselves as well.
<unk>.
Fifth patient is currently in discussion for ERP withdrawal so.
Who is the fifth patient.
Three cohort III, so it's a third patient in cohort three.
The two patients in cohort four are not on <unk> correct.
Got it thank you so much.
Maybe I'm not sure. If you said this before in the past, but just as you move to the dose expansion phase.
Are you going to be including kidney biopsies.
The expansion phase.
Nicole Germino: We are now open to enrolling female patients, patients with with cardiac and renal involvement. And as such, there is interest also from these patient populations.
Nicole Germino: So I'm just picking up from Bettina, you know, obviously, as Bettina sort of summarize, our commitment is really to ensure that we, you know, successfully execute against the expansion cohort and plan for our phase three.
Yes, Indeed, we are going to be.
Including kidney biopsies in patients who are naive to Tonight.
<unk>.
We're performing that in fact, we have already performed kidney biopsies on the too naive patients in cohort four.
And so in the first half of next year to be sharing data from from those first two patients.
Operator: Thank you for taking our questions.
Excellent. Thank you for taking our question.
And one of them are for our next question.
Our next question comes from what I recall with Jefferies. Your line is open.
Operator: This is Lisa on for Luca.
Hi, Thanks for taking my questions.
Operator: First off, congrats on all the progress on the quarter.
I have one for fabry two I just wanted to check to add what other conferences could we expect to see the full nine patients for fabry and just clarifying if we will see the data for the 49 patients in the second half of this year.
Operator: Just a couple on Sabry.
Operator: I'm, wondering if you can remind us again what the criteria is for stopping ERT.
Yes.
Thanks for the question. There is there are several conferences coming up this year and into the beginning of next year, we're always cautious too.
Not detail, which conference we are going to until we have confirmation that there will be a presentation there.
For now we're only guiding to SSR.
Okay.
And what we see and update on all nine patients before the end of the year.
We would hope so.
Okay, Okay and then.
It looks like.
Pfizer's reiterating restarting dosing.
Third quarter of this year, but the pivotal data has been pushed back from second half two late 'twenty three or early 'twenty. Four can you talk about why this data readout has been pushed back.
I think we would guide you to speak to Pfizer, but.
We hope to do.
Communicator noticed this but they continue to guide that there due to start the study.
This quarter.
Okay. Okay. Thanks for taking my questions.
One moment for our next question.
Our next question comes from Jason Seidl H VW. Your line is open.
Hi, This is Jason on for Patrick.
So I guess the only question I have is.
Around.
The sickle cell.
Hemophilia a program.
Sorry, the sickle cell program for <unk> three so now that is wholly own and kind of return from Sanofi back Sangamo are there any potential plans to them partnered out again and if so will you be waiting until the phase III trial design and when the plans already or would that be before that planning.
Yes.
March one to take this one sure.
Yes.
As Rob covered in his part of the talk I mean, our focus now is to.
Complete the precision phase one study and then we'll evaluate that at the same time, we're putting together.
Plans for manufacturing as well as preparation of the phase III study design.
Once we've seen the data and take a look at that relative to the.
The competitive datasets, which which you know very much about we will make a.
A decision on on how we commercialize or whether we look for a partner.
Just to reemphasize that this is a debilitating disease.
There's roughly just just in the U S alone 20% to 30.
Patients that have severe disease and so one of the things you need to remember is as we as.
As we see approvals in this space and hopefully we will.
It's going to take some time for us to service that population because you are constrained by our ability to manufacture their personalized cell therapy.
So we will provide an update on that.
In terms of our.
Rationale behind partnering.
Really look at partners, where we believe that we can get the medicine to patients in a quicker timeframe globally.
And so as we as we do with all of our programs if there's a partner that.
It can allow us to do that faster and at the same time ensure that we're getting the right level of return for ourselves and our investors that we will do that.
Alright, thank you.
One moment for our next question.
The next question comes from Andres, our treaties with Wedbush. Your line is open.
Great. Good afternoon, thanks for taking our question.
Switch it up for <unk> for sickle cell can you remind us what data you are seeing that differentiates this program from the other gene therapy competitors.
Can you repeat the comments.
<unk> in your prepared remarks also.
What can we expect from the phase one data this year and when do you plan to collect data outside of <unk> that focuses more on prevention.
Progressive organ damage.
So thank you for your question.
<unk>.
We've dosed.
Five patients snow and.
Looking forward to seeing.
The data from them and the subsequent patients and only once we have seen that we'll be able to.
Make any rational comment on differentiation.
I also think it will take a larger population of patients to properly understand that.
Okay.
Does that answer your question.
Yes that was one of them and they were put in the prepared remarks I didn't catch it but there were some comments around <unk>.
Could clarify that and then.
As far as what data we can expect.
A lot of the.
Community is focusing on <unk>, but likely focus on prevention of progressive organ damage, which is a pretty important aspect do you plan to collect data and if so when our PON would you would you plan to collect data on that thanks.
So bettina can you clarify a bit the POC and then I know it makes additional comments.
Sure so for the Vlccs, we had already mentioned one patient.
Out of the four patients dosed.
And that we presented at Ash last year had one VLCC. This basically you had a second DLC and Thats, a patient who has the lowest hemoglobin levels.
It's important to mention that that patient is not doing well and that will be other patients.
Uh huh.
Presented any of the Ocs.
And doing remarkably well.
And that patient was dosed with the original process, which switch.
Fifth patient in subsequent patients will be dosed with a much improved form the increases the long term progenitors I think you make a good point.
Six two are are very important so doctor having treated patients with poc's, it's a horrible disease and the patients suffer greatly formats, but there's also a second underlying whether it's a micro POC type damage that you get in.
The results in these patients huffing.
Significantly shorter life expectancies.
And we would hope the offer the course of the trials.
Longer study, we would also show a benefit for that but that's going to take us going to be harder.
Longer time.
To prove but it clearly is important to patients and it speaks to treating them as young as possible. So as they do not accumulate damage before they get this remarkable treatment.
Great. Thanks for the clarity on that thank you.
And I'm not showing any further questions at this time I turn the call back to Louise for any closing remarks.
Thank you once again for joining us today and for your question.
As a reminder, you can access the earnings release and presentation on the Investor Relations section of the Sangamo website, we look forward to keeping updated on our future to keeping updated on our future development.
Ladies and gentlemen, this does conclude today's presentation. You may now disconnect and have a wonderful day.
Nicole Germino: You know, Fabry disease is not a disease that's just unique to the United States.
Operator: And I believe you mentioned in the prepared remarks that you were in discussions with a fifth patient who was looking to stop ERT.
Nicole Germino: It's a disease that that comes out a variety of different markets.
Operator: I was just wondering if they are also in the cohort three group.
Nicole Germino: So both the way we're approaching our clinical trial and the way we're approaching our plans for commercialization, take that into consideration.
Operator: Thank you.
Nicole Germino: As you know, we've not, you know, guided any specifics in terms of our plans for for commercialization, but we'll do that at the appropriate time.
Operator: So Bettina, criteria for stopping ERT and who's the fifth patient?
Nicole Germino: Great.
Operator: So the criteria for, stopping ERT really have not been outlined in detail in the protocol intentionally to leave this and this phase one, two study open to the investigator in conjunction with the patient.
The conference will begin shortly to raise your hand during Q&A you can dial star one one.
Nicole Germino: Thanks.
Operator: And so it is criteria that those entities apply when they discuss this in conjunction, with ourselves as well.
Nicole Germino: And just a second on collaboration.
Operator: The fifth patient is currently in discussion for ERT withdrawal.
Nicole Germino: Maybe can you talk about high level about your partner program progress kind of which part of the platform you're getting more attention from partners and where should we stay focused on?
Operator: So who is that fifth patient?
Nicole Germino: Thank you.
Operator: It's in cohort three.
Nicole Germino: So one of the blessings about having, you know, unique technology platform is that we're constantly being approached with people exploring ways that they can collaborate with us.
Operator: So it's the third patient in cohort three, and the two patients in cohort four are not on ERT. Correct.
Nicole Germino: And so, you know, that's a key part of our, you know, our focus from a business development standpoint.
Operator: The conference will begin shortly. To raise your hand during Q&A, you can dial star, 11.
Nicole Germino: Whether that be in terms of our gene engineering capabilities that Jason briefly touched on, whether that be opportunities to, you know, to work together on capsid development or capsids that could be applied to our CNS programs.
Operator: Okay, got it.
Nicole Germino: But in addition, as we've talked about in the past, you know, we've raised about eight hundred and fifteen million. And in, you know, potentially six to seven billion in in royalties that come from partners that, you know, approach us about using utilizing our technology to take that technology into areas that we wouldn't otherwise do.
Operator: Thank you so much.
[music].
Nicole Germino: And so I think the Biogen and Novartis continue to be great examples of that.
Operator: And maybe, I'm not sure if you had said this before in the past, but just as you move to the, dose expansion phase, are you going to be including kidney biopsies in the expansion phase?
Nicole Germino: And as we continue to confirm and validate our progress with our technology, we would expect other companies to approach us with ideas that they might have in terms of how they could utilize our technology. And we'd be pleased to have those conversations.
Operator: Yes, indeed.
Operator: Our next question comes from Gina Wang with Barclays, your line is open.
Operator: We're going to be including kidney biopsies in patients who are naïve, or pseudo-naïve.
Operator: We're performing that.
Operator: In fact, we have already performed kidney biopsies on the two naïve patients in Cohort 4 and so hope in the first half of next year to be sharing data from those first two patients.
Operator: Excellent.
Operator: Thank you for taking our questions.
Operator: And one moment for our next question.
Operator: Our next question comes from Maurice Raycroft with Jefferies.
Operator: Your line is open.
Operator: Hi.
Operator: Thanks for taking my questions.
Operator: I have one for Fabry too.
Operator: Just wanted to check at what other, conferences could we expect to see the full nine patients for Fabry and just clarifying if we'll see the data for the full nine patients in the second half of this year?
Operator: Thanks for the question.
Operator: There are several conferences coming up this year and into, the beginning of next year.
Operator: We're always cautious to not detail which conference we are going to until we have confirmation that there'll be a presentation there.
Operator: So for now, we're only guiding to SSIEM.
Operator: Okay.
Okay.
Operator: And will we see an update on all nine patients before the end of the year?
[music].
Operator: We would hope so.
Operator: Okay.
Operator: And then it looks like Pfizer's, reiterating restarting dosing in third quarter of this year, but the pivotal data has been pushed back from second half to late 23 or early 24.
Operator: Can you talk about why this data readout has been pushed back?
Operator: I think we would guide you to speak to Pfizer about that.
Operator: They hold the, they communicate in all of this, but they continue to guide that, they're due to start the study in this quarter.
Operator: Okay.
Operator: Okay.
Operator: Thanks for taking my questions.
Operator: One moment for our next question.
Operator: Our next question comes from Jason Scheiss of HCW.
Operator: Your line is open.
Operator: Hi, this is Jason.
Operator: I'm from Patrick.
Operator: So I guess we, the only question we have is around the, sickle cell, sorry, the hemophilia program.
Operator: Sorry, the sickle cell program for BIVV003.
Operator: So now that it's wholly owned and kind of returned from Sanofi back to Sangamo, are there any potential plans to then partner it out again?
Yes.
Operator: And if so, will you be waiting until the phase three trial design and when the plans are ready or will that be before that planning?
Operator: Thank you.
[music].
Operator: Mark, do you want to take this one?
Operator: Sure.
Operator: Yeah.
Operator: So as Rob covered in his part of the talk, I mean, our focus now is to complete the, precision phase one study, and then we'll evaluate that at the same time we're putting together plans for manufacturing, as well as preparation of the phase three study design.
Operator: Once we've seen the data and take a look at that relative to the competitive data sets, which you know very much about, we will make a decision on how we commercialize or whether we look for a partner.
Operator: You know, just to reemphasize that, you know, this is a debilitating disease.
Operator: You know, there's, roughly just, you know, just in the U.S. alone, 20 to 30,000 patients that have severe disease.
Operator: And so one of the things you need to remember is as we see approvals in this space, and hopefully, we will, it's going to take some time for us to service that population because you're constrained by your ability to manufacture their personalized cell therapy.
Operator: And so we'll provide an update on that.
Operator: You know, in terms of our rationale behind partnering, we really look at partners where we believe that we can get the medicine to patients in a quicker timeframe globally.
Operator: And so, you know, as we do with all of our programs, if there's a partner that can allow us to do that faster, and at the same time ensure that we're getting the right level of return for ourselves and our investors, that we'll do that.
Operator: All right.
Operator: Thank you.
Operator: One moment for our next question.
Operator: Next question comes from Andreas Argyrides with Legbush Yard.
Operator: Your line is open.
Operator: Great.
Operator: Good afternoon.
Operator: Thanks for taking our question.
Operator: We're going to switch it up in favor, in terms of sickle cell.
Operator: Can you remind us what data you've seen that differentiates this program from the other gene therapy competitors?
Operator: And can you repeat the comments around VOCs in your prepared remarks?
Operator: Also, what can we expect from the phase one data list this year?
Operator: And when do you plan to collect data outside of VOCs that focuses more on prevention of progressive organ damage?
Operator: Thanks.
Operator: So thank you for your question.
Operator: We've dosed five patients now and are looking forward, to seeing the data from them and the subsequent patients.
Operator: And only once we've seen that will we be able to make any rational comment on differentiation.
Operator: I also think it will take a larger population of patients to properly understand that.
Operator: Does that answer your question?
Operator: Yeah, that was one of them.
Operator: And in the prepared remarks, I didn't catch it, but there were, some comments around VOCs.
Operator: If you could clarify that.
Operator: And then as far as what data we can expect, a lot of the community is focusing on VOCs, but less is focused on prevention of progressive organ damage, which is a pretty important aspect.
Operator: Do you plan to collect data?
Operator: And if so, when, at what point would you plan to collect data on that?
Operator: Thanks.
Operator: So Bettina, can you clarify about the VOC?
Operator: And then I'll make additional comments.
Operator: Sure.
Operator: So for the VOCs, we had already mentioned one patient out of the four patients dosed, that we presented at ASH last year had one VOC. This patient had a second VOC, and that's a patient who had the lowest hemoglobin levels.
Operator: It's important to mention that that patient is now doing well, and that all the other patients have not presented any VOCs and are doing remarkably well.
Operator: And that patient was dosed with the original process, which the fifth patient and subsequent patients will be dosed with a much improved form that increases the long-term progenitors.
Operator: I think you make a good point that VOCs, though, are very important.
Operator: As a doctor, having treated patients with VOCs, it's a horrible disease and the patients suffer greatly from it.
Operator: But there's also a second underlying, whether it's a micro VOC type damage that you get that results in these patients having significantly shortened life expectancies.
Operator: And we would hope that over the course of the trials and longer study, we would also show a benefit for that.
Operator: But that's going to take, that's going to be a harder, longer time thing to prove. But it clearly is important to the patients and it speaks to treating them as young as possible so as they do not accumulate damage before they get this remarkable treatment.
Operator: Thanks for the clarity on that.
Operator: Thank you.
Operator: And I'm not showing any further questions, at this time.
Louise Wilkie: I turn the call back to Louise for any closing remarks.
Louise Wilkie: Thank you once again for joining us today and for your questions.
Louise Wilkie: As a reminder, you can access the, earnings release and presentation on the Investor Relations section of the Sangamo website.
Louise Wilkie: We look forward to keeping you updated on our future developments.
Operator: Ladies and gentlemen, this concludes today's presentation.
Operator: You may now disconnect and have a, wonderful day.