Q3 2024 Sangamo Therapeutics Inc Earnings Call
Yeah.
Good afternoon, and welcome to the Sangamo chip.
Speaker Change: Chip Therapeutics third quarter 2024 teleconference call. Please be advised that today's conference is being recorded I would now like to turn the conference over to your first speaker today, Louise Wilkie, Vice President Investor Relations and corporate Communications. Please go ahead.
Speaker Change: Okay.
Speaker Change: Thank you good afternoon, everyone. Thank you for joining us on our call today.
Speaker Change: It's called a separate members of the Sangamo executive leadership team, including Sandy Macrae, Chief Executive Officer, Nancy <unk>, Chief Development Officer, and procedure do a Barber Chief Financial Officer.
Speaker Change: From a corporate presentation can be found on our website sangamo com under the presentations page of the investors and media section.
Speaker Change: This call include forward looking statements regarding <unk> current expectations. These statements include but are not limited to statements related to think of it as cash on my plans to obtain additional capital and ability to continue operating as a going concern.
Speaker Change: Therapeutic and commercial potential of <unk> product candidates and technologies Banca means ability to and receive payments from its collaboration and license agreements and creating the Genentech agreement.
Speaker Change: Think of it as expectations regarding your collaboration and license agreements.
Speaker Change: Despite the plans and timelines of Sangamo and its collaborators for clinical trials clinical data presentations and releases regulatory submissions and regulatory approvals.
Speaker Change: Kind of catalysts and milestones and other statements that are not historical fact.
Speaker Change: Actual results may differ materially from what we discuss today.
Speaker Change: 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, 2023 are supplemented.
Speaker Change: Supplemented by subsequent quarterly reports on Form 10-Q for the quarter ending September 32020, full and subsequent filings and reports under 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 over to our CEO Sandy Macrae.
Sandy Macrae: Thank you Luis and good afternoon to everyone joining the call today.
Sandy Macrae: As we neared the end of 2024 I'm extremely proud of the progress we are making this year.
Sandy Macrae: We are committed to translating groundbreaking science into match since it transformed the lives of patients and families afflicted with serious neurological diseases.
Sandy Macrae: The updates we will share today demonstrates several meaningful advances toward cycle.
Sure Hi, we believe <unk> is well positioned for continued progress.
Sandy Macrae: First sangamo has transformed from a phase one two company to a pre BLA company as a result of significant regulatory developments in our fabry disease program.
Sandy Macrae: Second Pfizer continues to engage in discussions with regulatory authorities concerning our hemophilia a program.
Speaker Change: Both of which have the potential to provide long term financial foundation for our core neurology pipeline.
Speaker Change: Third we signed a neurology epigenetic regulation in cap C deliberately license agreement with Genentech and received $50 million in upfront license fees and milestone payments.
Speaker Change: And finally, we submitted our first step our R&D application for neurology indications.
Speaker Change: These developments demonstrate the sangamo is steadily executing upon our strategy is driving potential medicines towards patients in need and just continuing to advance plans to put the company on a more stable financial footing.
Speaker Change: Lastly, I will share more context in a moment, but I want to start out by highlighting the significant clinical and regulatory progress made this quarter and our fabry disease program fully got alignment with the FDA on a clear regulatory pathway for accelerated approval.
Speaker Change: This pathway reduces the time to potential approval by three years and are poised to requirement for an additional lengthy and costly Registrational study, which is incredibly impactful given the unmet medical need for patients with fabry disease.
Speaker Change: We are delighted to have such a clear regulatory pathway that could bring this treatment to patients significantly sooner than originally anticipated.
Speaker Change: Gone to execute BLA readiness activities ahead of our submission anticipated in the second half of 2025.
Speaker Change: As you can imagine this announcement has generated a lot of interest from external stakeholders, including patients who tell us. They are mark will change our treatment is brought to their lobbies investors and potential strategic partners.
Speaker Change: We continue to engage in ongoing business development discussions as we seek to get this treatment to patients as quickly as possible.
Speaker Change: Following the recent top line readout from Pfizer for the Phase III <unk> trial in hemophilia, a we also move closer to potential regulatory submissions for this program, which has the potential to unlock.
Speaker Change: Look up to $220 million in regulatory and commercial milestones for Sangamo over the next two years.
Speaker Change: Pfizer, we presented detailed phase III data update at the upcoming American Society for hematology or Ash annual meeting in December.
Speaker Change: And Pfizer have advised us that they are discussing mistake.
Speaker Change: With regulatory authorities, which is very encouraging.
Speaker Change: Taken together the fabry in hemophilia a programs could provide triangle with a solid and long term financial foundation for our core neurology pipeline.
Speaker Change: We are through to be in the enviable position of seeing up to two potential BLA submissions in 2025 for product candidates developed by Sangamo.
Speaker Change: Each of which leverage the best of our science and capabilities and address significant unmet patient needs and commercial opportunities.
Speaker Change: This exciting momentum propels, our neurology pipeline forward as we work to advance our science and technology for neurological indications.
Speaker Change: This quarter, we signed our first neurology epigenetic regulation in capsid delivery license agreement.
Speaker Change: We have granted genentech, an exclusive license our highly potent zinc finger repressor directed to tout.
Speaker Change: A critical gene involved in Alzheimer's disease, and other type of parties as well as some additional undisclosed second neurology target.
Speaker Change: For these same targets. We also granted genentech, an exclusive license to our industry, leading neurotropic delivery capsid stack, BBB, which has demonstrated potent blood brain barrier penetration and brain transduction and non human primates.
Speaker Change: We have received $50 million in upfront license fees and milestone payments from Genentech.
Speaker Change: Which extended our cash runway to allow us to continue advancing other ongoing business development activities.
Speaker Change: This agreement has further drawn interest in our science and enhanced our ability to attract new potentially valuable partnerships.
We are currently advancing business development discussions with additional potential collaborators seeking to license our novel intravenous capsid stack BBB and we believe this capsid is a potentially valuable source of additional non dilutive funding.
Speaker Change: This quarter, we also submitted our first step our IND application for our neurology indications.
We expect our lead program <unk> $5 three for intractable pain to advance into the clinic in the middle of 2025, assuming clearance at the IND with the FDA with our expected prior on clinical trial authorization submission followed closely behind by the end of 2025.
Speaker Change: Our cash runway remains unchanged and is sufficient to fund our planned operations into the first quarter of 2025 absent any potential funding from a fabric partnership hemophilia, a milestone payments from Pfizer or additional stack BBB collaboration agreements.
Speaker Change: Alongside the two anticipated BLA submission next year, we see a viable path to financial stability.
Speaker Change: I would now like to hand, it over to Natalie our head of development, who will share additional context and also take us through other pipeline updates.
Natalie.
Natalie: Thank you Sandy.
Speaker Change: Beginning first with Fabry, we were excited to announce last late month that we have aligned on a clear regulatory pathway to accelerated approval with the FDA for is a ragga GNC that Pablo back our SG 920, a wholly owned gene therapy candidate for the treatment of Fabry disease.
Speaker Change: The FDA has confirmed that estimated glomerular filtration rate or Egfr slope data at 52 weeks from all patients in the ongoing phase one two star study can serve as the primary basis for approval under the accelerated approval pathway.
Speaker Change: This pathway avoids the requirement for an additional costly Registrational study as we had previously anticipated and importantly accelerate distant made a time to potential approval by absorbed approximately three years.
Speaker Change: Sangamo engage with the FDA on alternative pathway to potential approval. Following analysis of clinical data from the phase II study showing encouraging safety and efficacy data.
Speaker Change: Renal manifestations such at proteinuria or a decrease estimated glomerular filtration rate our egfr occur early in life in almost all male and in many female patients with fabry disease.
Speaker Change: Can lead to renal disease and early death.
Speaker Change: <unk> is assessing millimeter of clean blood per minute per body surface.
Speaker Change: For context, the average untreated patients as an egfr slope of minus five six however in the 18 male and female patients treated with <unk> bank with more than one year of follow up data, we observed a statistically significant positive mean annualized egfr.
Speaker Change: So jana.
Speaker Change: <unk> a positive egfr slope is a truly remarkable achievement for fabry patient, especially since other fabrics therapy. According to public data improve egfr value compared to untreated fabry patient, but still show a negative overall egfr slope.
Speaker Change: Based on this latest data the FDA agreed that Egfr slope at 52 weeks can serve as an intermediate clinical endpoint to support a potential accelerated approval.
The FDA also advice that egfr slope at 104 weeks, maybe assess to verify clinical benefit.
Speaker Change: The complete data set to support the accelerated approval pathway will be available in the first half of next year.
Speaker Change: Looking at potential BLA submission in the second half of 2025 three years ahead of previous estimates.
We are delighted to have a clear regulatory pathway that could bring this treatment to patients in need significantly sooner than originally anticipated.
Speaker Change: Suddenly is a debilitating disease for which there is a serious unmet medical need and this need was clear at the National Fabry disease Foundation Passion patient conference held in October at which Sangamo is in attendance.
Speaker Change: During focus group discussions.
Speaker Change: Be patient elaborated on the challenges associated with current treatment options, including having to switch treatment regimen due to unwanted side effect in sufficient resolution of their symptoms and breakthrough pain.
Speaker Change: Patients also noted the challenges of receiving long infusion every second week, which is logistically problematic and that time can cause them to skip doses.
Speaker Change: As a reminder, our phase one to start study as enroll and dose 33 patients with <unk>.
Speaker Change: Presenting a gorilla ranch of Fabry patient, we are station where on the tea at the start of the study as well as patients were <unk> naive or pseudo naive.
Speaker Change: We have male and female patients.
Speaker Change: Well as those with cardiac or renal implication.
Speaker Change: With a positive annualized egfr slope observed thus far across these different type of fabry patients. We anticipate that the BLA submission will cover the entirety of the Fabry population is 18 years and older.
Speaker Change: In the other Star study update all 18 patients who started this study on ERP now successfully of CRT and the first patient has been withdrawn from the RT recently achieved an impressive three years of EBIT that is a significant achievement.
Speaker Change: More broadly with the longest treated SG 920 patient recently, achieving four years of follow up our data continue to look encouraging with all patients achieving and maintaining physiological RFC process electrical level of plasma Alpha Gal a enzyme activity to date.
Speaker Change: In terms of next step we have begun to execute BLA readiness activity and continue to advance ongoing business development discussions with potential collaboration partners.
Speaker Change: We are also committed to working with the European medicines agency or EMA to identify the optimal regular path forward in Europe and following a successful prime kickoff earlier. This year are preparing for continued discussions.
Speaker Change: Our hope is to be able to arrive at the align approach across the regulatory agencies and we anticipate sharing a regulatory update in early 2025.
Speaker Change: Moving now to <unk>, an investigational gene therapy, we are developing with Pfizer for patients with moderately severe.
Speaker Change: <unk>.
Speaker Change: On December 9th Pfizer plans to present detailed data from the phase III <unk> trial in an oral presentation at the 66 Ash annual meeting and Exposition.
As abstract which was released last week confirm that our fine trial met its primary endpoint of non inferiority and superiority with a statistically significant decrease in total annualized bleeding rate or ABR from week 12 to at least 15 months of follow up post infusion compare with.
Speaker Change: Routine fluctuate replacement prophylactic treatment.
Speaker Change: Key secondary endpoint as defined by the trial protocol the percentage of participant with factory activity greater than 5% at 15 months or ABR for treated bleed were met and also demonstrated superiority compared to prophylaxis.
Speaker Change: Notably tier auto continues to tell public back was generally well tolerated with no study discontinuation.
Speaker Change: These data further validate our effort to discover and develop genomic medicine with the potential to improve the lives of patients partnering.
Speaker Change: Partnering with an organization with strong commercialization infrastructure and experience as well as the broader franchise in this area was important to us as we enter into.
Speaker Change: A partnership with Pfizer, we greatly appreciate Pfizer's leadership of this important program.
Speaker Change: Pfizer has advised us that they are discussing the phase III find data with regulatory authorities. As a reminder, we are eligible to earn up to $220 million in milestone payments from Pfizer upon the adjustment of certain regulatory and commercial milestones and 14% to 20% royalty on.
Speaker Change: Potential sales from this program if approved and commercialized.
Speaker Change: This important advances allows us to focus on our core mission to treat debilitating neurological disorders with innovative genomic medicines.
Speaker Change: We believe our ability to combine <unk> finger epigenetic regulation payloads with exciting new industry, leading capsid delivery technology could unlock the potential of our neurology pipeline and change the treatment paradigm for neurological indications for each delivery of treatment to the central nervous system as historically.
Speaker Change: <unk> been challenging.
Speaker Change: This quarter, we submitted the 90 application to the FDA for our NAV, one seven program our ft $5 three for the treatment of intractable pain.
Speaker Change: Neuropathic pain can be caused by a broad area of pathologies impacting the central Paris are all nervous system, such as surgical trauma.
Speaker Change: Final court enter enough compression neurological and infectious diseases metabolic and hesitated syndromes.
Speaker Change: <unk> III is not intended for sporadic or acute pain toothache and unite to me.
Speaker Change: But for intractable chronic pain that completely dominate and often destroyed lots of patients over many years.
Speaker Change: Assuming FDA clearance of the R&D. The phase one two study will assess the effectiveness of <unk> III in addressing idiopathic small fiber neuropathy or issn peripheral neuropathy that resulted highly debilitating syndromes.
Speaker Change: Trickling studying.
Speaker Change: Lightning like pain.
As a fan as an estimated prevalence of at least 43000 patients in the U S and more broadly peripheral neuropathy are estimated to affect nearly 40 million Americans.
Speaker Change: Anti depression anticonvulsants opioid are topical therapy can be tried although no long lasting occurred if therapies are currently available for issn patients leading to a high unmet medical need for this patient population.
Speaker Change: A significant body of evidence implicates surgeon channels in mediating the pathophysiology of neuropathic pain Xtify, those three use and add no associated virus or AAV.
Speaker Change: Victor carrying an engineered zinc finger repressor to specifically target the human gene SCN nine eight that encodes denied 1.1, several channel <unk> channel and it's critical for pain signaling.
Speaker Change: Developing small molecule that specifically target. Another 1.17 is challenging due to the highest structural similarity between different sodium channels, making it difficult to achieve selectivity and avoid of target.
Speaker Change: Perfect.
Speaker Change: By directly targeting the SCN nine a Jean <unk> was shown to selectively reduce the expression of one seven sodium channels in sensory neurons in animal model and significantly reduce pill hypersensitivity falling.
Speaker Change: Single is physical administration.
Speaker Change: Thank you most preclinical research has shown <unk> III to be well tolerated in nonhuman primate and substantial NAV. One seven reduction was observed with no off target effects.
Speaker Change: These preclinical data, which demonstrate the potential of <unk> III as a therapy for chronic neuropathic pain.
Speaker Change: Aberrate it upon in more detail in a manuscript published in bio archive earlier this quarter, which is also available on the presentation and publication section of our Investor page on Sangamo Dot com.
Speaker Change: We expect to start the phase one two in the middle of 2025. This represents a transformational transformational step forward for <unk> as the first of our neurology program to progress to the clinic it.
Speaker Change: It is hope that if the efficacy is demonstrated the application of <unk> III could be broadened to patient population suffering from other types of chronic neuropathic pain.
Speaker Change: Moving now to pre on clinical trial authorization or Cta, enabling activities continue to advance our program to treat prion disease, which leverage novel type BBB capsid.
Speaker Change: As a reminder, prion disease represent a group of condition with a diverse set.
Speaker Change: Unmet medical need.
We believe our technology can address with more than 500, new patient diagnosed each year across the U S and Europe. This is a disease that is rapidly progressing and always fatal usually within 12 to 15 months of symptom onset and with no currently effective treatment option available.
Speaker Change: In October we presented updated data at the pre owned 2024 conference, which demonstrated the <unk> zinc finger repressor in a mouse model at multiple dose level.
Speaker Change: The zinc finger repressor significantly reduce expression of prion mrna and protein in the brain.
Speaker Change: Standard mouth survival and limited the formation of toxic prior in aggregate.
Speaker Change: Additionally, nonhuman primate data.
Speaker Change: 2024 highlighted that a single intravenous administration of the preempting finger repressor deliver via stock BBB resulted in potent and widespread repression of the prion gene in tons is narrow.
Speaker Change: Our Cta submission for this program is expected in the first quarter of 2025.
Sandy Macrae: I will now hand, it back to sandy for closing remarks.
Sandy Macrae: Thank you Natalie.
Sandy Macrae: In closing we are delighted with the momentum being generated this year and are committed to the continued advancement of both our wholly owned and partnered programs.
Sandy Macrae: In 2025, <unk> could lead to BLA submissions for up to two separate gene therapy programs that we believe have the potential to fund a neurology company in the long term.
Sandy Macrae: We plan to dose patients are first Stefan neurology epigenetic regulation studied.
Sandy Macrae: And we plan to submit an <unk> for the second study, which would be the first in human study of a novel proprietary stack BBB neutral pig capsid.
Sandy Macrae: We're also currently engaged in advanced business development discussions for.
Sandy Macrae: Potential stack BBB collaborations.
Sandy Macrae: This is remarkable progress for a company of our size.
Sandy Macrae: We are pleased to liberate these milestones in 2024 and plan to continue executing on our strategy in the coming months.
Sandy Macrae: As we complete the task of transforming <unk> into a neurology genomic medicine company.
Sandy Macrae: We see an exciting future as we make progress on addressing our long term financing needs and look forward to building. Upon this year's progress in 2025, as we further advance our therapies to patients in need.
Speaker Change: Operator, please open the line for questions.
Speaker Change: Thank you at this time, we will conduct a question and answer session.
Speaker Change: To ask a question. During this session you will need to press star one on one of your telephone and wait for your name to be announced.
Speaker Change: To withdraw your question. Please press star one again.
Speaker Change: Please stand by while a compile the Q&A roster.
Speaker Change: Yeah.
Speaker Change: Our first question comes from Gena Wang from Barclays. Please go ahead.
Speaker Change: Thank you congrats on multiple achievements here.
Speaker Change: So maybe I'll, just focusing on fabry program too.
Speaker Change: Two related questions. The first one is regarding the FTE comments.
Speaker Change: G fast slope at 52 weeks can serve as intermediate clinical endpoint that does that mean, Steve wanted to see statistical significance of the.
Speaker Change: And of the 52 egfr compared to the baseline.
Speaker Change: Then the Egfr slope at the 104 weeks.
Speaker Change: Just to verify clinical benefit can you elaborate on what does that mean to you.
Speaker Change: Need to continue to show statistical efficacy benefit compared to baseline or do you need to show a positive trend continue.
Speaker Change: At the 104 weeks and the second question as it related to the strategy I think Sandy you mentioned I think that that has been a while that you wanted Sheila.
Speaker Change: Seeking partnerships for this program and now keeping the latest update what is your.
Speaker Change: Our latest thinking regarding this program that you think it would be most value January for the shareholders. So would that be keep in house or seek.
Speaker Change: Seeking partnership if seeking partnership what kind of valuation would be looking for.
Speaker Change: Thank you gena for the questions.
Speaker Change: I'm going to pass on to <unk>, we haven't given details of the statistical analysis to the agency have asked us to do it really is.
Speaker Change: Credible.
Speaker Change: <unk>.
Speaker Change: Both the Egfr is shown to be a positive slope and dose. So that the agency has embraced this and agreed to accelerated approval one year. Natalie can you add some color to this.
Speaker Change: Yes. Thank you, yes, we are delighted to have a clear regulatory pathway to accelerated approval that could bring this drug sooner to the patient.
Speaker Change: So the FDA has agreed that the data can serve as the primary at 52 weeks can serve as the primary basis for approval under the accelerated approval program using the Egfr slope at 52 weeks across all patient and this is an intermediate clinical endpoint that will give us accelerated approval.
Speaker Change: And then just as a reminder, there is no limitation placed on biologic product granted accelerated approval.
Speaker Change: In addition, the FDA.
Speaker Change:
Speaker Change: The the FDA.
Speaker Change: <unk> that the data from the 104 weeks can use to confirm clinical benefit.
Speaker Change: So we see that there is no need for any additional study.
Speaker Change: And we will have those discussion during the BLA.
Speaker Change: Pre BLA meeting with the FDA, but we.
Speaker Change: Anticipate that we will.
The two year data for the.
Speaker Change: 32 patient in 2026.
Speaker Change: For a full approval.
Speaker Change: This is quite important to get clear, Chris I know that a number of people complete accelerated approval with a confirmatory study I wanted to be absolutely clear that new confirmatory study has been as far or as required.
Speaker Change: We will submit the data for the one year of which point will be 32, one year.
Speaker Change: But also 19 of them will already have reached there to your point.
Speaker Change: We anticipate that.
Speaker Change: We would be submitting the full two year data set a year later.
Speaker Change: The agency has said that they would they would recommend looking at this as <unk>.
Speaker Change: Confirmation of the one year data what's been really interesting is when we flip to the patients who have reached two years already is at the one year predicts the two year data so the patient as opposed to a one year. It predicts that there will also be posted if it two year. So this is exciting first thing because all of a sudden the BLA filing.
Speaker Change: It's been put in three years, we're doing everything possible to drive this forward to make sure we get it to patients with a filing in the second half of next year.
Speaker Change: Last year other question about partnerships.
Speaker Change: As you can imagine there's a number of people have been very interested in these results.
Speaker Change: Wonderful form we can announce a partnership around our quarterly coal. Unfortunately, these things just take time and we've almost had to refresh the partnership discussions with this new data, we hope to be able to share more about it in the near future, but what we want to do is make sure. We do a deal that gets it to pace.
Speaker Change: <unk> with a filing in the second half of next year and a launch sometime in the first half of 'twenty six.
Speaker Change: That answers your question.
Speaker Change: Thank you.
Speaker Change: Thank you one moment for our next question.
Speaker Change: Our next question comes from Yanan Xu from Wells Fargo. Please go ahead.
Speaker Change: Hi, Thanks for taking our question. This is quite an encore yana so our cartoons.
Rounding the Fabry program.
Speaker Change: Can you can you share with us when May we see next.
Speaker Change: Hey type data from their program. Thank you.
Speaker Change: Laterally.
Speaker Change: Yes so.
Speaker Change: Now our phase <unk> has become a registrational study. So we are being very careful about disclosing the details of the data and we expect to them.
Speaker Change: To have the data in the second quarter of 2025 and sure at that time, the topline data so to be clear the patients.
Speaker Change: The last patient joined this study in April of this year. The last patient last visit will be <unk> next year, and then takes some period of time to clean the data and that will allow us to submit the data in the second half of next year. Once we have that data cleaned and prepared we will look for the best opportunities.
Speaker Change: <unk> shared the data with all of you.
Speaker Change: Got it thanks for the color and.
Speaker Change: Some quick follow up so for patients with longer follow up you mentioned that they still have a positive egfr slope, but do you see any change in the slope when a patient follow ups get longer and also in.
Speaker Change: CRD patient.
Speaker Change: Do you see any change in the Egfr so when patients stop withdraw thank you.
Speaker Change: So.
Speaker Change: There are so many questions I'm sure we would all like to discuss about this data and we look forward to sharing it at the right time, what I can say is the.
Speaker Change: The longest lasting patient in this study is over four years and four months and those are the patients who were in the lowest dose with a dose escalation study and they're doing very well in the end.
Speaker Change: The Alpha Gal continues to be produced at similar levels, which is very encouraging.
Speaker Change: There are so few patients out at that time that to do in Egfr slope would not be wise until more patients get to the longer time points, Natalie anything, yes, and I can also confirm that the positive ETS EG.
Speaker Change: Egfr slope that we are observing at this point is across all patients whether they're started on the RT or their naive or pseudo naive, whether they're male or female.
Speaker Change: Got it thank you for all the colors.
Speaker Change: Thank you one moment for our next question.
Speaker Change: Our next question comes from Maury Raycroft from Jefferies. Please go ahead.
Maury Raycroft: Alright, congrats on the progress and thanks for taking my question.
Speaker Change: As a follow up to <unk> question earlier, I think you've mentioned you could include propensity matched control data to as part of the filing package.
Speaker Change: Just wondering if you can comment on whether FDA will put more weight on comparing versus baseline are versus their propensity matched control data.
Speaker Change: And can you talk about where you're at with getting that propensity matched control data is that something that you think you will need in order to maximize potential with a partnership.
Speaker Change: So we can't comment.
Speaker Change: Unable to comment on how the agency will weigh the different pieces of data.
Speaker Change: <unk> C is throughout the discussions they have said, it's the totality of the data so the Egfr booth.
Speaker Change: Compared to a group rather than individuals' compared to as a group.
Speaker Change: <unk> from baseline to one year, and then two years compared to what <unk> historically recognized for Fabry disease, where there is over five point drop over the course of the year, what's known about the E. R. T agents, where there's between one and a half two and a half.
<unk> over the course of the year.
Speaker Change: But they'll also be looking at the sustained effect of Alpha Gal a sustained control of lives with GBP three the the debt.
Alpha Gal a levels in the skin biopsies are significantly increased <unk> SF 36, the patient reports about paying for Sam S. S. Only it's the whole body of this data set is so compelling.
Speaker Change: And.
Speaker Change: Everyone on E. R. T. I think this is just important to remind us separately. One the 18 patients that came in on New York T have been taken off PRT.
Speaker Change: Happily all CRT. So it's a really compelling data set of which the Egfr is is it's like the thing that allows the agency to give us find us a way to approval.
Speaker Change: But it is supported and meet meet meet better by the totality of the data.
Speaker Change: Got it all.
All makes sense and that's helpful.
Speaker Change: And just wanted to clarify too for the milestone payment from Genentech for the $50 million did you receive all 50 million or was there 40 million recorded this quarter and where you get an additional $10 million from the tech transfer just wanted to clarify on that.
Speaker Change: Hey, Maury. This is Patricia yes, we received all of the 15 million.
Speaker Change: Only a portion of it the 40 was reflected in our quarter end balance.
Speaker Change: What you're trying to bridge it.
Speaker Change: Got it okay. Thanks, Thanks for taking my questions.
Speaker Change: Thank you.
Speaker Change: As a reminder to ask a question you will need to press star one on one of your telephone and wait for your name to be announced to withdraw your question. Please press star one again.
Speaker Change: One moment for our next question.
Speaker Change: Our next question comes from Luca <unk> from RBC capital. Please go ahead.
Speaker Change: Oh, great. Thanks, so much for taking my questions and obviously congrats on the regulatory alignment maybe two quick questions here for me on.
Speaker Change: On Fabry as high level can you just talk about how your conversation with the FDA has evolved over time.
Speaker Change: I finally fascinating that the FDA ask Ivor <unk> to run a head to head trial versus <unk> using renal biopsies as a primary endpoint not too too long ago.
Speaker Change: Right.
Speaker Change: Sounds like the FDA is telling you that single arm looking at serum biomarker is now sufficient so.
Speaker Change: What has changed there in your view again any color there would be much appreciate it and then maybe you will see all year.
Speaker Change: Maybe in the context of the <unk>.
Speaker Change: Biomarin commercial debacle, if you will.
Speaker Change: How confident are you that Pfizer I really want to put a lot of energy and resources behind the approval and commercial launch of this therapy. Thanks, so much.
Speaker Change: Importantly, importune questions. So.
Speaker Change:
Speaker Change: I don't remember how long ago that absolute bio had the discussion with the agency was at two was it three four years ago, where the.
Speaker Change: They were using pre conditioning cell therapy to try and show the agency that there was a benefit to their medicine.
Speaker Change: At that time, there were several companies full GMT free line.
Speaker Change: And others that we're in.
Speaker Change: The Fabry World and then you fast forward and many of these have dropped out.
Speaker Change: And.
Speaker Change: The other piece that's changed I think as Peter marks his position at <unk>, where he realizes that there is limitations on what can be required of small populations of genomic medicines for rare diseases and he wants to help us.
Speaker Change: All of us find a path forward for this and so.
Speaker Change: Zinc.
Speaker Change: Sangamo has.
Speaker Change: Greet with the agency is simply the.
Speaker Change: Peter has been seeing meat meat into our plan for a very effective agents not only if you wanted to just walk through the timeline of how we got there yes. So as you might recall, we had a type C meeting earlier in 2024, well, we agree with the FDA to do a phase <unk> study with.
Speaker Change: 25 patients four.
The basis of approval then after this we had another data cut from our trial start trial and.
Speaker Change: Realize that we had a positive egfr slope in 18 patients.
Omar: One year Omar.
Omar: And also six patient at two years at that time, we had scheduled a prime meeting with EMA. So this was our prime kickoff and in attendance with the FDA in the spirit of global of the FDA and EMA approach to have a global appeal.
Omar: Approach to approval for rare diseases.
Omar: At that time, we presented to EMEA, the egfr slope data and the FDA in attendance.
Omar: Was.
Very encouraged by this and ask us to submit the data to the FDA. So because we have Ahmad designation, we decided to do a type b meeting, where we describe our results to the FDA.
Omar: And ask if a given this remarkable data of acquisitive egfr slope in our 18 patient at one year, we could.
Omar: Use egfr slope as the primary basis of approval on an accelerated approval pathway.
In our phase one two study alleviating the phase <unk> study that was originally planned and the FDA agree with our approach and that's where we are today.
Omar: I should say in all my many years of working with the FDA I have never seen a written response so clear when asked if we could use it 12 months Egfr for accelerated approval. They said, yes, we agree.
Omar: So we are very pleased with I think.
Omar: Hopefully as somewhat of a precedent for other rare diseases companies, they're trying to do their very best for patients are an incredibly difficult circumstances, if I could switch to your second question, which was your.
Omar: Your reflections on the Biomarin launch of <unk>.
Omar: We are in our relationship with Pfizer and so we're always very careful to reflect what Pfizer has told us and before each conference call. We agree with them what it is how they would like their their medicine to be reflected.
Omar: We're very pleased with the the abstracts are going to be showing at ash and I would encourage you to look at the data there and in REIT from Pfizer's language about their enthusiasm for this product, but they have told us that we can say that they are in discussions with regulatory authorities.
Speaker Change: That to US It suggests pfizer's continued enthusiasm for this product.
Speaker Change: Super Super clear thanks, so much.
Speaker Change: Thank you one more before I next questions.
Speaker Change: Our next question comes from Ritu barrel from.
From television Cowen. Please go ahead.
Speaker Change: Good afternoon, guys. Thanks for taking the question.
Speaker Change: Sandy.
Speaker Change: The FCA endpoint revolves around renal function.
Speaker Change: Are they requiring or requesting any proportion or quantum of data from the from the pivotal data set be from female fabry patients.
Speaker Change: Or patients with cardiac variant.
Speaker Change: The answer is no would you still be able to have some of that perspective.
Speaker Change: Uh huh.
Speaker Change: Either.
Speaker Change: The open label portion or or from some other prospective analysis.
Speaker Change: For.
A likely label at some point just as as the cardiac variant.
Speaker Change: In cardiac involvement ticketing fee gains importance.
Speaker Change: It might be an important commercial tool to have talking to the broader patient population.
Speaker Change: So the discussion with the agency has been that this is for fabry it hasn't been for men versus women or renal versus cardiac it's been all fabry patients naive to suit a naive E. R T experienced men and women.
Speaker Change: Our discussions with them have largely been about Egfr because we have this remarkable data and we will look at all the other pieces of the data set and the submission.
Speaker Change: Second the.
When we measure all forgotten in the skin biopsies, it's increased when we look at the renal function, it's increased or sorry, it's improved when we look at the patient.
Speaker Change: Patients report of switching of their how do they feel up the Fox assets. Thus.
Speaker Change: Thus emmis I there is improvement across all of it. So one would expect that there's no reason why it wasn't huffing and effect in the heart as well as other parts of the body in the 32 patient data set is all that the agency has asked for that will not be additional patients put in after that.
Speaker Change: As part of the Sangamo.
Speaker Change: Clinical development program.
Speaker Change: Natalie however, the patients or are very keen to this gets to them as soon as possible you met your two met with.
Speaker Change: The Fabry support group recently took a little of what you heard what they hurt.
Speaker Change: Yes, we were at the National Fabry disease Foundation patient conference about three or four weeks ago, and we held a patient group a forum, where we had patients that were on the <unk>, but we had also three patients that were received gene therapy.
Speaker Change: And these patients are.
Really very happy to have made that decision to enter the trial, we have patients that.
A report that they were working with a cane than now dropped that came that walking normally they feel so much better.
Speaker Change: Quote the patient feel like it as a new lease on life, we have patients that have reduced pain, we have patients that.
Speaker Change: Now taking job in the heat and to have port carrying luggage and Theyre sweating and they feel great. So it's a general well being that we hearing from the patient the patient that are on the RT are not very happy but that treatment first of all it's very burdensome every two weeks you get a five to six.
Speaker Change: Sarah I think infusion often you have someone coming at home you have to organize this it's pretty invasive and really what we've heard from the patient is.
Speaker Change: After 10 days of the E. R. T that start feeling not well, they're starting to have symptoms coming back and they still have to wait and they'll other medication that theyre, taking around the ER pre op post so it's really not a very good.
Speaker Change: Treatment for the patient.
Speaker Change: Syed and because it started with some we know that there is a large population of fabry patients that do skip doses because of the button of of <unk>.
Speaker Change: We are very clear that <unk> was a great advance in the treatment of fabry.
Speaker Change: And we feel that this is note. The next stage in that journey, where they will be able to get a single infusion of AAV extremely well tolerated with no serious adverse events related to the treatment.
Speaker Change: Sure this benefit overtime and.
Speaker Change: It's a privilege to work on such a medicine and we look forward to finding a.
Speaker Change: The right partner to take this forward to get it to patients.
Speaker Change: Thanks.
Speaker Change: Thank you.
Speaker Change: Question and answer session is now closed I will now turn it back over to Luis Wilkie for closing remarks.
Luis Wilkie: Thank you and thank you once again for joining us today and for all of your questions. As a reminder, you can access our presentation on the Investor Relations section of the Sangamo website.
Luis Wilkie: Look forward to keeping you updated on our future developments. Thank you.
Luis Wilkie: Thank you for your participation. Thank.
Speaker Change: Thank you for your participating in today's conference. This does conclude the program you may now disconnect.
Speaker Change: Okay.
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