Q1 2024 Sangamo Therapeutics Inc Earnings Call
Good day and welcome to the Sangamo Therapeutics first quarter 2024 teleconference 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 you will need to press star one on your telephone you will then hear in auto.
Made it message advising your hand is raised to withdraw your question. Please press star one again, please be advised that today's conference is being recorded.
I'd now like to turn the conference over to your Speaker today, Louise Wilkie, Vice President of Investor Relations and corporate Communications. Please go ahead.
Louise Wilkie: Thank you good afternoon, everyone. Thank you for joining us on the call today.
Louise Wilkie: On this call a supplement and both are assigned to my executive leadership team, including Sandy Macrae, Chief Executive Officer, Patricia do or Bob <unk>, Chief Financial Officer, Amy Proto type of research Nathalie Dubois Stringfellow Chief Development Officer.
Louise Wilkie: Slides from our corporate presentation can be found on our website sangamo com under the presentations page of the investors and media section.
Louise Wilkie: This call includes forward looking statements regarding <unk> current expectations.
Louise Wilkie: Statements include but are not limited to statements related to the therapeutic and commercial potential of our product candidates and engineered capsid.
Louise Wilkie: And the potential of our next generation genome engineering technology.
Louise Wilkie: Anticipating planting timeline to think about it and our collaborators for regulatory submissions and initiating and conducting clinical trials and presented clinical data.
Louise Wilkie: Most of our product candidates anticipated regulatory submissions.
Louise Wilkie: Advanced preclinical programs to the clinic.
Louise Wilkie: Strategic re prioritization and reallocation of resources and the anticipated benefits thereof plans.
Louise Wilkie: Plans to partner starting to buy programs.
Louise Wilkie: I should say of our resources cash runway and plans to seek additional capital.
Louise Wilkie: Catalysts and milestones and other statements that are not historical facts.
Louise Wilkie: Actual results may differ materially from what we discuss today.
Louise Wilkie: These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC specifically in our annual report on Form 10-K for fiscal year ended December 31, 2023 supplemented by our quarterly report on Form 10-Q for the quarter ended March 31, 2024 filed with the SEC.
Louise Wilkie: Forward looking statements stated today are made as of this date 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.
Louise Wilkie: Now I'll turn the call over twice a year Sandy Macrae.
Alexander D. Macrae: Luis and good afternoon to everyone joining the call.
Alexander D. Macrae: I'm very pleased to be speaking to you today from the American Society of cell and gene therapy annual meeting in Baltimore.
Alexander D. Macrae: Well, we have been presenting important preclinical data from our epigenetic regulators.
Alexander D. Macrae: Capsid delivery platform, our next generation genomic engineering platform.
Alexander D. Macrae: These data showcased both the depths of Sangamo neurology pipeline and the power of our scientific capabilities, which we believe provides strong opportunities to advance programs ourselves.
Alexander D. Macrae: And with potential partners.
Louise Wilkie: In order to progress these compelling programs sangamo must be well capitalized.
Louise Wilkie: Our leadership team and I have been laser focused on addressing our funding needs.
Louise Wilkie: March we were pleased to announce a registered direct offering with institutional shareholders, including unimportant existing investor.
Louise Wilkie: That reached approximately 24 million in gross proceeds.
Louise Wilkie: This was a significant development and we are thankful for their support of our science and our mission.
Louise Wilkie: Okay.
Louise Wilkie: That was however, the first step in our current journey to securing additional funding.
Louise Wilkie: I would like to emphasize that we are resolutely focused on building. Upon this foundation to position Sangamo for long term success.
Louise Wilkie: That continues to be my number one priority.
Louise Wilkie: Business development is an important part of these efforts and I am pleased with the progress that's being made on this front. We're in currently engaged and very encouraging conversations with multiple potential partners across our portfolio, including our fabry disease program, our novel stack PPP engineered capsid.
Louise Wilkie: Our preclinical neurology product candidates and our next generation genome engineering capabilities.
Speaker Change: I understand your desire to hear more concrete news on this front, but.
Speaker Change: But we were unable to share more until any potential transaction is finalized.
Speaker Change: Be assured that we are encouraged by the progress of these discussions and hope to announce news of one or more transactions.
Speaker Change: Over recent months Sangamo has presented important preclinical data that solidify our sharpened focus in urology validates our differentiated science and help contextualize why we made this important decision to dedicate ourselves to addressing neurological disorders.
Speaker Change: In March we were proud to share remarkable preclinical data from her new intravenously administered neurotrophic AAV capsid.
Louise Wilkie: Which demonstrated industry, leading blood brain barrier penetration and brain transduction and non human primates.
Louise Wilkie: This novel Capsid stack BBB showed robust penetration of the blood brain barrier with 700 food higher transgene expression neurons compared to the benchmark capsid AAV nine and outperformed all other new and publish capsid variants evaluated in this study.
Louise Wilkie: Combined with our potent epigenetic regulation cargo, we showed robust stack BBB mediated expression of zinc finger cargo in neurons with potent and widespread repression of the cryo in Ontario genes observed across all key green areas illustrating the exciting potential.
Louise Wilkie: To modify disease progression in prion disease, and various tie up with this.
Louise Wilkie: These data support further advancement of our <unk> type programs, which we're on track for regulatory submission sent to the clinic by the end of 2025.
Louise Wilkie: Meanwhile, we continue to advance our lead candidate in chronic neuropathic pain NAV, one seven which uses an established entry, particularly administered capsid to orphan int submission expected in the fourth quarter of this year.
Louise Wilkie: We believe our ability to combine potent zinc finger epigenetic regulation payloads with exciting new industry, leading capsid delivery technology could unlock significant potential for the treatment of devastating neurological diseases.
Louise Wilkie: Vacations for fresh delivery of treatments to the central nervous system has historically proved challenging.
Louise Wilkie: Building on this we are proud to be presenting three platform presentations and 17 posters at this week's S. GCT annual meeting.
Louise Wilkie: These presentations showcased the depth of our neurology pipeline, including various applications for zinc finger epigenetic regulation platform exciting advances in our capsid delivery technology and the discovery of potentially transformative next generation integrated technology engineered to enable large.
Louise Wilkie: SKU genome editing.
Louise Wilkie: And epigenetics regulation, we have shared advances in zinc finger act to be through some of the pressures for the potential treatment of a remarkable range of neurological diseases, such as prime disease type work that these charcot Marie tooth disease is type one and <unk> <unk> syndrome, So one mediated <unk>.
Louise Wilkie: Trophic lateral sclerosis, or elas, feeling macdiarmid syndrome, parkinsons disease instruments syndrome, and many other neurological disorders.
Louise Wilkie: We have also demonstrated singles potent delivery capabilities developed through our AAV capsid engineering platform sister.
Louise Wilkie: Here, we presented additional stack BBB findings, including prion entire depression data achieved via stacked BBB delivery.
Louise Wilkie: We also presented for the first time exciting initial findings for possible mechanism supporting higher stock BBB may cross the blood brain barrier.
Louise Wilkie: Our sister platform, Mr sign to engineered capsid for various routes of administration, such as interest Teco and intravenous delivery.
Louise Wilkie: <unk> can also engineered capsid or in vitro discovery. So in a platform presentation Tomorrow, we will present findings from stack 150, a novel capsid, that's been shown to be highly potent in neurons and enables high throughput screening of neurology for Chris transcriptional regulators, we anticipate this stack.
Louise Wilkie: 150, which we believe manufacturers easily at small scale will help accelerate the discovery of potent and highly specific epigenetic regulators.
Louise Wilkie: Finally building on our deep expertise in that protein DNA interactions derived from nursing finger platform. We presented for the first time, a potentially breakthrough new approach for integrating large sequences of DNA into the genome to potentially treat with a single medicine patients.
Louise Wilkie: Who have unique mutations in the same gene.
Louise Wilkie: Precisely integrating large synthetic DNA constructs into desirable chromosomal site has been a dream for people working in this field for many years.
Louise Wilkie: Our modular integrates our mint platform is a <unk> protein guided genome editing method, the understands and engineers <unk> won a serum serine companies to insert a replace entire genes and adds to sangamo as two books of editing capabilities.
Louise Wilkie: Yes.
Louise Wilkie: We are hopeful that our.
Louise Wilkie: Platform could be used to correct, many disease, causing mutations and the diverse patient population by inserting a correct copies of the gene into its natural Lucas.
Louise Wilkie: Minutes could be deployed internally for various neurological indications, but also provide potential partnering opportunities both for human disease and in agricultural biotech settings.
Louise Wilkie: We are already in active discussions with potential partners about her integrates capabilities and our hope and hopeful that mint could provide us with another potential non dilutive funding opportunity.
Louise Wilkie: Alongside our presentations in this topic ESG CET. This week, we have also published a manuscript in bio archive further outline. These next generation degrees advancements, which is also available on the publications page of the website.
Louise Wilkie: I encourage you to learn more about this exciting development in the field of genomic medicines.
Louise Wilkie: Now looking at our clinical programs, we have made strong advances in the first quarter for Fabry disease program.
Louise Wilkie: Does the final patient in the phase one two star study of Mr. Jim Suva powerful bank, our investigational gene therapy for the treatment of Fabry disease.
Louise Wilkie: With 33 patients dosed screening enrollment and dosing are complete.
Louise Wilkie: One additional patient has been able to stop enzyme replacement therapy or <unk>, resulting in a total of 14 patients withdrawn from IRT to date. The full remaining patients dose since February 2024, who began the study on New York team already have plans in place to withdraw ear treatment at the appropriate time.
Louise Wilkie: At the 20th annual World Symposium in February we presented compelling updated preliminary clinical data from the STAAR study showing sustained benefit and a differentiated safety profile.
Louise Wilkie: These results underscore the program's potential a single administration treatment for fabry disease.
Louise Wilkie: As a reminder, this quarter, we announced the alignment with the U S. FDA on an abbreviated pathway to potential approval.
Louise Wilkie: We've also been granted prime eligibility by the European Medicines agency and I lump designation by the U K medicine, and healthcare products regulatory agency.
Louise Wilkie: We are engaged in active discussions with potential collaborators.
Louise Wilkie: <unk> for Fabry disease program and continue to defer additional investments and planning for a potential registrational trial until the collaboration partnership or financing for this program is secured.
Louise Wilkie: Moving to our partner clinical program, we look forward to the pivotal readout expected in mid 2024, and the phase III <unk> trial of tiered Taco gene two powerful effect.
Louise Wilkie: An investigator with gene therapy, we are developing with Pfizer for patients with moderately severe to severe hemophilia a.
Louise Wilkie: Pfizer anticipate meeting a biologics license application and a marketing authorization application in early 2025 of the pivotal readout is supportive.
Louise Wilkie: As a reminder, we are eligible to earn up to $220 million in milestone payments and up to 14% to 20% royalties on potential sales from this program.
Louise Wilkie: We ended the quarter with approximately 54 million in available cash and cash equivalents, which includes funds from the aforementioned registered direct offering.
Louise Wilkie: We believe these resources in combination with these cost savings expected from the recent restructurings workforce reduction and other potential cost reductions will be sufficient to fund our planned operations into the third quarter of 2024.
Louise Wilkie: As I outlined earlier, we are actively pursuing a range of different options to important additional capital and are encouraged by the business development discussions ongoing across our Fabry program Capsid engineering, a next generation genome engineering efforts.
Louise Wilkie: We believe our company has the science required to potentially transform the lives of patients living with devastating neurological conditions.
Louise Wilkie: Committed to raising the funding required as we seek to meet Covid patient a reality.
Speaker Change: Operator, please open the lines for questions.
Speaker Change: Thank you at this time, we will conduct a question and answer session. As a reminder to ask a question you will need to press star one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one again, please standby, while we compile the Q&A roster.
Speaker Change: Our first question comes from the line of James stay most at Jefferies. James Your line is open.
Louise Wilkie: Hi, This is James on for Maury Raycroft, Congrats on the progress and thanks for taking my question I just wanted to ask where are you with preclinical and GOP Tox studies with stack BBB and what are your latest thoughts on which targets or indications would make the most sense to partner or keep in house.
Speaker Change: Thank you. Thank you for your question.
Speaker Change: We have made what are the reasons that we like stack BBB. So much is that our manufacturing team have been able to work with it and that it is.
Speaker Change: Is very manufacture bulk.
Speaker Change: They can ticket so far up to 50 liter scale in it are going higher that they can use the same.
Speaker Change: Purification and assay techniques.
Speaker Change: That it will that it fuels.
Speaker Change: It looks likes a capsid that we'll be able to give us great yield and also be able to use many of the processes and CMC work that really is underappreciated and the importance of capsid selection.
Speaker Change: We've said publicly that we are moving ahead with <unk>.
Speaker Change: With <unk> you could imagine the amount of external interest in total not just because it is a high unmet medical need but because the caps. It gives you the Britain.
Speaker Change: And attrition and widespread delivery that's needed.
Speaker Change: Zinc finger repressor, it's a really unique in being able to almost switchover.
Speaker Change: In the sense that it can get to.
Speaker Change: Yes.
Speaker Change: We are having many discussions with companies about our capsid.
Speaker Change: And we'll choose wisely, I believe which which ones, we partner and which ones. We take forward ourselves, what we understand and I want to say this again and repeat what I said in the.
Speaker Change: And chosen works is that we understand the need to bring in money to the company.
Speaker Change: So it was a balance between the desire that we have to take things forward.
Speaker Change: So appealable from many of the partnerships I'm sure we will achieve in the coming weeks.
Speaker Change: Great that's very helpful.
Speaker Change: And just.
Speaker Change: Go on to like some of the more specialized parts of Europe as GCT presentation can you talk about the importance of targeting the pons and motor neurons in Alzheimer's and are there any other companies that you're aware of that have the ability to target. These sites.
Speaker Change: I think thank you for your question.
Speaker Change: It's really been a remarkable <unk> in the field moves closer to.
Speaker Change: These.
Speaker Change: These tools being able to address such important disease. Amy can you talk a little about this I know this is one of your questions.
Amy: Thank you for the great question.
Amy: That's really important here is being able to target brain regions that would be traditionally in challenging target using something like a direct injection approach the ponds being part of the <unk> is not a region that you would necessarily want to reach using a direct injection havent guided that.
Speaker Change: Steven.
Speaker Change: Even for the most severe diseases. So we were so excited to see the bio distribution that was able to be achieved with effectively be capsid.
Speaker Change: Especially for what it means for the top program. The Pons is critical in progressive Supranuclear palsy, but also other telecom seems like Alzheimers disease. In addition, as you'll notice that the motor cortex, which is part of the frontal cortex.
Speaker Change: As an example of how stock BBB is able to transduce does cortical neurons.
Speaker Change: We believe that this kind of widespread Brian targeting is critical for halting or or slowing the progression of the disease.
Speaker Change: Thanks Amy.
Amy: Great. Thank you for taking my questions and I'll hop back in the queue.
Speaker Change: Thank you very much one moment for our next question. Please.
Speaker Change: Our next question comes from Gena Wang of Barclays Gena. Your line is open.
Gena Wang: Thank you for taking my questions I have two the first one also is about the stack BBB agonist seems like very exciting new capture what is the highest dose used in nonhuman primates and what is the limiting.
Gena Wang: Dose toxicity, there and then the second question quickly regarding the he may I know your partner Pfizer is in charge, but just wondering for the mid 'twenty four phase III update would that be just press release from Pfizer and then for the 220 milestone payment.
Gena Wang:
Gena Wang: The first milestone is that the <unk> approval and is that a big portion of the $220 million.
Gena Wang: Gena. Thank you for your questions and I know you've.
Gena Wang: Following he may for for a great deal of time, so it's good to be coming closer to the point, where we were.
Gena Wang: It gets to registration and approval. So let me do let me pass this on to two people Amy can you talk about the dose that we fused.
Amy: Whether there was any toxicity please.
Amy: Yes, absolutely.
Amy: As part of the stack BBB study that we discussed yesterday and we also will discuss later today at the poster session at Ash TCT, we performed a dose range finding study with our talent.
Amy: <unk> zinc finger and we did three different doses at the top doses 114.
Gena Wang: Happy to see that with the histopathology results that we didn't see any dose limiting toxicity and really no findings in the brain. This final quarter of the liver this peripheral organs, so really really positive and encouraging data.
Speaker Change: Does that answer your question is there any any color.
Speaker Change: Okay.
Speaker Change: Yes, like la <unk> other than <unk>.
Speaker Change: Dose finding like what is the highest dose you tested for the safety toxicology perspective.
Speaker Change: 14th so one for 2014 okay.
Speaker Change: That is the highest.
Speaker Change: We went there in the efficacy study that we just reported and we really saw nothing sorry, we saw nothing that would give us any concern when we will now go into the GMP Tox study, which is the only thing remaining to complete the IND, enabling studies for them.
Speaker Change: But we will clearly start to a lower dose in the clinical study Chris that's the right thing to do and that's what the agency Velasquez to do but thus far we have.
Speaker Change: Very clean capsid, which is great great news.
Speaker Change: And then can we talk about human naturally I know you've been speaking to your friends at Pfizer regularly.
Chris: Sure sure Yeah, we're very excited that the mid 2024 pivotal readout in MAA is coming up soon.
Speaker Change: We did.
Chris: The partnership allows for $220 million in potential milestone and 14% to 20% royalty on potential cell.
Speaker Change: But the specific amount of each milestone is not publicly disclosed.
Chris: But this could start as early as the start of 2025, if the pivotal readout is positive and if Pfizer I'd like to see regulatory approval to the program.
Speaker Change: And Gina just just to reflect that.
Speaker Change: We're limited in what we can see when you sign these deals there's always an agreement about who talks about what.
Gina: But the the regulatory.
Chris: Students, which are we get one for <unk>, one for Europe, or Japan or not for approval there for submission of the ultimate package.
Chris: Then the commercial milestones which are.
Chris: <unk>.
Chris: Our four first patient dosed rather than for reaching a certain.
Chris: Sales threshold. So they are very very favorable miles students that will fund the company in 'twenty five 'twenty six.
Chris: Sorry, I need to mention that the significant portion of the $220 million as near term and we could also choose to monetize them now.
Chris: Yes.
Chris: So the closer we get to that.
Chris: Putting the data is revealed and then when the <unk> Pfizer's submit CIS, we have the option of monetizing those royalties.
Chris: Sure.
Chris: But we can also choose to wait and use them to fund 25, and 26, eight and what will make that decision as the.
Chris: The non dilutive funding from business development that we do not it will tell us whether we need to make that decision we are.
Chris: I know that there's a concern from people that followed us for a long time.
Chris: Our near term cash runway.
Chris: We feel that we are.
Chris: We are fortunate to be able to do both capsid deals fact reduce technology tools and to have this large amount of cash next year that we can bring forward if necessary.
Speaker Change: Thank you very much.
Speaker Change: Thank you gena. Thank you very much one moment for our next question. Please.
Speaker Change: Yeah.
Speaker Change: Our next question comes from Patrick <unk> of H C. Wainwright Patrick Your line is open.
Speaker Change: Hi, everyone. This is luis sensors or Patrick.
Patrick: Saturation is on the recent progress in your presence.
Patrick: <unk> presence.
Speaker Change: Yes.
Patrick: I am interested in knowing if you already guided to.
Speaker Change: The Tau program and if it's if it's going to be in Alzheimers do you know, which patients are going to be treating and which other indications you are going to.
Speaker Change: Push forward here with the Tau program and then I have a follow up question.
Speaker Change: So thank you for your question.
Speaker Change: We haven't guided on.
Speaker Change: There has been an enormous amount of interest in our presentation yesterday <unk> it.
Speaker Change: It clearly was I think the most remarkable presentation of all of the many people that are moving forward in a blood brain barrier penetrant cap sits.
Speaker Change: We feel that the tau asset the cargo that's within it.
Speaker Change: Doubled stone mix makes us even more interesting.
Speaker Change: The goal of targeting Alzheimers I think is becoming increasingly clear that the biogen data is very encouraging.
Speaker Change: The idea that it's.
Speaker Change: The thing that is relevant to the clinical stages of Alzheimer's rather than in the elderly.
Speaker Change: Pre symptomatic ones mixed system much more easy lift however, we're very conscious that outside of our studies are large programs.
Speaker Change: That we need to make sure that we either to ourselves well or partner with someone who has a real expertise in alzheimers.
Speaker Change: A follow up thank you that was helpful on a follow up on the on partnerships, but for the minutes.
Speaker Change: For the men platform you mentioned that there are business development opportunities. There do you have any potential collaborators in mind and what's what diseases would you be targeting.
Speaker Change: Okay.
Speaker Change: The mint.
Speaker Change: <unk> integrates is remarkable data presentation at <unk> was just 57 minutes ago.
Speaker Change: And there was a lot of excitement in the room about it.
Speaker Change: We have already been we've been speaking to people for three months or less.
Speaker Change: The business development discussions into perspective, we had the regulatory clarity for Fabry just in February we had captured results just in March we finally.
Speaker Change: <unk> been to a place that we feel we can share it in may so.
Speaker Change: I understand the desire for four.
Speaker Change: Signed deals but the.
Speaker Change: The results are going to lead to them have only come out over the past two or three months, which is means that at the moment you could imagine our business development group is incredibly busy.
Speaker Change: In talking to lots of people about the snow integrates as something that.
Speaker Change: Is.
Speaker Change: It has the potential for really disruptive genomic engineering, it's the kind of thing that David Louie Funny was asked said was his dream of what the ideal genomic medicine was which was and integrates it could be engineered to go to your site to choice and.
Speaker Change: Pieces of cheating sized pieces of DNA.
Speaker Change: Replace all of the mutations stems stream.
Speaker Change: Sure.
Speaker Change: The team at Sangamo because of their expertise in understanding the interaction between proteins and DNA from the zinc finger platform have been able to do this remarkable engineering.
Speaker Change: <unk> outperformed in understanding artificial intelligence of how you understand proteins has helped us.
Speaker Change: We feel that this is something that sangamo can use but it also is has attracted great interest from the hard core science pharma companies that you could imagine who all see this as the future genomic engineering and so we have a responsibility to put it in as many people's hands.
Speaker Change: As possible.
Speaker Change: I think you will hear more about this <unk> platform for some time to come.
Speaker Change: Thank you.
Speaker Change: Thank you very much one moment for our next question. Please.
Speaker Change: Okay.
Speaker Change: Our next question comes from the line of Luke AC at RBC Luca Your line is open.
Speaker Change: Oh, great. Thanks for taking our questions. This is Lisa on for Luka.
Lisa: I just wanted to ask another question on the <unk> technology.
Lisa: I'm just wondering if you can give us a sense of what kind of target here would be good candidates.
Lisa: For instance would it be possible to integrate something like a full length. The FTR gene, which is around roughly 190 kilo basis or should we think about something larger like full length, dystrophin, which is measuring closer to.
Lisa: Over 2000 kilo basis, just any color here would be helpful. Thanks.
Speaker Change: So.
Speaker Change: Yes, you ask really important and interesting questions.
Speaker Change: As always with Sangamo.
Speaker Change: We.
Speaker Change: Thought about size.
Speaker Change: <unk> because as you know zinc fingers are an eighth the size of CRISPR uneven small mammoth Christopher's are four times as big as the zinc finger and that's important because of delivery.
Speaker Change: With mint because.
Speaker Change: <unk> tried to keep the technology as small as possible so as we could could both.
Speaker Change: The integration and cargo and an AC no when you get to the size of some of the genes that you're talking about you and it comes down to what kind of delivery you're using monitoring for mint is to be able to drop a cta and a copy of the <unk>.
Speaker Change: <unk> <unk> R R.
Speaker Change: Mutated into internal one offs.
Speaker Change: The genome.
Speaker Change: Then the run off of the promoter have older Lucas control elements.
Speaker Change: It would.
Speaker Change: Alleviate the requirement to added oldie and individual mutations that usually are the cause of <unk>.
Speaker Change: Genomic disease now.
Speaker Change: <unk> put you in a whole genomic copies.
Speaker Change: B unnecessary I would argue and then we need to look at for the diseases. We cleared the cdna is packaging.
Speaker Change: Two the delivery mechanisms are available because the mint should be size agnostic.
Speaker Change: And has so far shown great potential.
Speaker Change: Amy is there anything else.
Amy: I'm aware that you are on the line and more.
Amy: Hedge of research, perhaps you would have thought those things done to them.
Amy: Fantastic explanation Sandy I would just add that there are specific genes as well that are involved in some neurological disorders and I'm thinking of things like Red syndrome, where <unk> needs to be fine tuned today, a very specific level, something like and integrates where you could integrate in perhaps the healthy copy of exon one or even a bigger portion of the gene.
Speaker Change: So that's under control of endogenous promoter could be really transformative for a disease like that so I think it's incredibly exciting to see what are the opportunities around this new platform.
Speaker Change: Thanks, so much thanks for your question.
Speaker Change: Thank you very much one moment for our next question.
Speaker Change: Our next question comes from Ritu, We're all of T. D Cohen redo your line's open.
Ritu: Hi, guys a quick question on <unk>.
Ritu: The Fabry program actually.
Ritu: Three patients.
Speaker Change: Data.
Speaker Change: At World.
Speaker Change: Sure.
Speaker Change: Phase III path going forward agreed upon with FDA.
Speaker Change: The next data update that we're going to get from that.
Speaker Change: So I guess is there a data point our follow up point.
Speaker Change: In a subset of patients that's gating to potential business development around.
Speaker Change: I can think of this.
Speaker Change: Thanks.
Speaker Change: Yeah.
Speaker Change: Thank you Richard I'm going to pass this on to Natalie I'll, just remind you again.
Natalie: The whole business development conversation around fabry changed with Peters.
Natalie: <unk> new way of thinking about this at the agency and the conversations that we had in February and so it's like.
Natalie: New conversation, we're having with potential partners, but naturally can you address some of the technical pieces. Please.
Speaker Change: Yes sure so.
Speaker Change: We've completed the dosing of all the patients in the phase one two so.
Speaker Change: So for a total of 33 patients we continue to analyze the data but this.
Speaker Change: The body of data that we have thus far it's not gate limiting for any partnership.
Speaker Change: And.
Speaker Change: Any of the phase two be preparation of Registrational trial preparation.
Speaker Change: The.
Speaker Change: To continue we continue to analyze the data, but we.
Speaker Change: And the other.
Speaker Change: With the FDA that we have enough data to move on to a phase II.
Speaker Change: And that's what we are discussing with partners potential partners.
Speaker Change: Andy when you say the discussion has changed are you talking about the structure of the deal.
Andy: Thank you Ms Best interest like is it can you.
Andy: It would be like a cocoa going forward or is it just really sort of valuation terms that are different now.
Andy: I think.
Andy: It's much simpler than that Ritu is.
Speaker Change: There was a concern that there wasn't clarity about what the regulators want too and there was this worry.
Speaker Change: We've all talked about it was going to be a head to head against <unk>.
Andy: And the agency no.
Speaker Change: Seeing no it's a simple single study.
Speaker Change: And that we can.
Speaker Change: That we can.
Speaker Change: The number of patients in the <unk>, we can come back halfway through the study and show them. The data we saw if we feel its compelling I think that is completely.
Speaker Change: Change.
Speaker Change: The various companies are going to and I think great credit to Peter He is.
Speaker Change: Walking the talk in this and we.
Speaker Change: <unk>.
Speaker Change: We benefited greatly format as being almost like the poster child for what it is approval of a rare disease gene therapy is.
Speaker Change: Great. Thanks.
Speaker Change: Thank you very much one moment for our next question.
Speaker Change: If I may.
Speaker Change: We started the we've initiated the conversations with the Europeans and uniquely the.
Speaker Change: The FDA is is volunteering to send one of their staff with us to that conversation because we feel that it's the way forward for these kind of therapies is for a common.
Speaker Change: Approach to them.
Speaker Change: I think it reflects the support that we have from the ski in these discussions.
Speaker Change: Thanks Bonnie.
Bonnie: Thank you very much. Our next question comes from the line of Nicole <unk> of Truest Nicole Your line is open.
Bonnie: Hi, This is bill on for Nicole.
Bill: We have a question.
Speaker Change: Given the uptake for rock KVM, what is the read through to potential uptake for fabry disease gene therapy.
Speaker Change: So thank you. Thank you for your question. So the <unk> launch has been.
Speaker Change: A subject of great debate, we can comment on <unk>.
Speaker Change: Biomarin launched at all I know is the reason that we chose spicer to be our partner is I have great confidence that when Pfizer chooses to launch something they do it well.
Speaker Change: Well and effectively and that follows through to our choice of partners for Fabry disease. There are several companies and there are some that are more obvious as being great commercialization.
Bonnie: <unk> engines and great launch companies well I can.
Bonnie: We use in our conversations with these companies we have had notes from the patient support groups.
Bonnie: We have been very keen to make sure the pharma partners understand how supportive they are.
Bonnie: The results that they have seen in our Fabry program.
Bonnie: Because you and I will talk about the biochemistry and the biopsies or the patient subset is how much better the feeling it.
Bonnie: Delighted they are to be also ERP. So no. We have 14 patients of the CRT and the four that are remaining on <unk> in the study have already booked when theyre going to come off of the Ulster therapy. So this has enormous support from the patients and I think that is what is going to make it.
Bonnie: <unk>.
Bonnie: A very successful launch for the partner that can take it forward and make a difference.
Speaker Change: And if I may add we also have our mat and prime which are designation that really analyst unmet medical need for fabry. So the fabry patient, even though can have access to it or not.
Speaker Change: With the current Madison and standard of care.
Speaker Change: Thank you so much.
Speaker Change: Yes.
Speaker Change: Thank you very much at this time I'm showing no further questions I would now like to turn the call back to Louise Wilkie for closing remarks.
Louise Wilkie: Thank you very much and thanks to everyone for joining us on the call today, we look forward to speaking to you soon.
Speaker Change: Thank you for your participation in today's conference. This does conclude the program and you may now disconnect. Thank you.
Speaker Change: Okay.
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Speaker Change: Okay.
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