Q3 2023 Sangamo Therapeutics Inc Earnings Call
Good morning, and thank you for standing by welcome to the St.
My third quarter 2023 teleconference call at this time, all participants are in a listen only mode.
After the speaker's presentation, there will be a question and answer session to ask a question. During this session you will need to press star one on your telephone.
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I'd now like to turn the conference over to your Speaker today, Luis Wilkie, Vice President of Investor Relations and corporate Communications. Please go ahead.
Thank you.
Good morning, I'm Louise will caisson goodbyes, Vice President of 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, including Sandy Macrae, Chief Executive Officer, Mark Mcclung, Chief operating Officer, Patricia do or Bobby Chief Financial Officer, Amy Poehler head of research not Lychee, Boston, <unk>, Chief Development Officer, and Lisa Rooikat Chief Medical Officer.
Lots of our corporate presentation can be found on 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 statements include but are not limited to statements relating to the therapeutic and commercial potential of our product candidates the anticipated plans and timelines of Sangamo and our collaborators for regulatory submissions and initiating and conducting clinical trial screening and patient facing patients.
Presenting clinical data.
Advancement of our product candidates anticipated feedback from and interactions with regulatory agencies.
On the preclinical programs to the clinic, a strategic re prioritization and reallocation of resources and the anticipated benefits thereof.
Sufficiency of our resources cash runway and plans to seek additional capital on the timing related upticks.
It makes it financial guidance for 2023, an estimate for 2020 for operating expenses.
Catalysts and milestones and other statements that are not historical facts.
Actual results may differ materially from what we discuss today.
Statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC specifically on our annual form annual report on Form 10-K for the fiscal year ended December 31 2022.
Our quarterly report on Form 10-Q for the quarter ended September 32023 old yesterday with the SEC the.
The forward looking statements stated today are made up of the states and we undertake no duty to update such information, except as required by law on.
On this call we discuss a non-GAAP operating expenses reconciliation of this measure to what that gross operating expenses can be found in our press release, which is available on our website.
Now I'd like to turn the call over to our CEO Sandy Macrae.
Thank you Louise and good morning to everyone joining the call.
Sangamo has a long impressive history of scientific innovation, leading to new discoveries and industry firsts for genomic medicines.
I am so honored to lead a company with such deep scientific expertise and a team dedicated to our mission of transforming patients' life with our technology.
All the programs are delivering promising results the environment within which we are operating has been.
Just slightly to remain challenging.
In 2020, we shared our refreshed company strategy, which aims to both maximize the potential of our proprietary genomic editing in delivering technology and to focus on areas, where we believe we can apply that technology to be either first in class or best in class.
That began with our decision to transition away from developing new traditional liver directed gene therapy several years ago and has continued into today.
Our renewed focus has been to identify areas, where we believe we can be market leaders and to prioritize our resources to those programs accordingly.
The difficulties in accessing capital being experienced across the industry have accelerated the pace of our plants and so today, we are announcing the final stage and the strategic transformation of <unk> to become a neurology focused genomic medicine company.
As we have shared before we strongly believe our technology is ideally suited to address a range of devastating neurological indications. There are few if any treatment options available today.
Over recent years, we've increased our focus on this important part of our business and have advanced exciting data from our rapid genetic regulation programs for neurological diseases.
The promising preclinical evidence for nursing finger editing capabilities, coupled with the strong progress, we're making identified novel capsid with enhanced delivery capabilities.
Demonstrates the potential for <unk> to become a leader in this space.
Our differentiated combination of precise versatile and compact genome targeting cargo alongside our novel AAV capsid evolution in <unk>, which has the potential for meaningfully improved.
Central nervous system transduction efficiency forms the foundation from which we believe we can transform the lives of patients with needed to generative diseases, such as intractable pain conditions prion disease Alzheimer's.
And many other neurological conditions, you'll hear us say through this its capsid and cargo you have to have capsid and cargo to be successful.
We believe this combination of genome targeting cargo and delivery capsule will be critical to our sustainable business model with our internal programs as well as providing important potential partnership opportunities.
I'll speak more about this in a moment.
In order to set the neurology company up for success, we must truly focus and carefully allocate resources Suez priority programs are central to our core strategy.
This means deferring new investments related to stopping or car T rate programs, while continuing to seek ways to maximize the potential value.
We're committed to these changes.
It's an immensely difficult decision to de prioritize spending on our legacy clinical programs, which have such clinical promise.
I strongly believe our fabry disease program is potentially transformative for patients.
The phase <unk> study continues to generate a meaningful package of data with 25 patients know dosed 25 patients, including 14 at the planned phase III dose.
<unk> continues to demonstrate sustained elevated alpha gal a levels with 12 patients having achieved at least one year of follow up on the longest treated patients having no cheap three year So Paulo.
Is a significant achievement.
Additionally, all 11 patients who withdrawn remained same replacement therapy remain of enzyme replacement therapy for up to 24 months for the longest withdrawing patients.
I think it's worth emphasizing that these are patients who were on <unk> and said to be treated they've come off ERC and they're staying or PRT, because they phillip pennell from a from a treatment.
Yeah.
And they tell us about it we have received testimony from patients, saying that the impact of <unk> 'twenty has been transformative and who report real and meaningful improvements in the quality of life.
Some even over and above the benefits that they were experiencing an E. R T.
We have shown and strongly believe in the promise of <unk> 'twenty as a potential medicine for patients suffering with fabry disease.
This is first in class currently the only gene therapy in the clinic for these patients.
However at this time, we are deferring any additional investments in phase III planning.
The cost of progressing the program beyond the current phase two study with.
With constrained our resources and require significant development and commercial investments, which we do not have the ability to support at this time.
We're doing everything in our power to Max size is followed by actively seeking a potential partner or alternative financing to fund a potential phase III trial.
We are confident that in the hands of a partner <unk> <unk> will make an enormous difference to the fabry community.
I firmly believe fabry patients deserve a better option than the current standard of care.
We expect to complete dosing of the remaining enrolled patients in the first half of 2024 and anticipate presenting additional updated clinical data at a medical meeting in early 'twenty four.
Turning now to a regulatory T cell programs, we are proud of our efforts to advance innovation the car T Reg field.
Since our acquisition of <unk> five years ago, we have become the first known company to those of human with an engineered car T. Reg overcame significant manufacturing challenges are known her dose in this field and deepened our clinical and preclinical expertise.
From our phase <unk> study of <unk> 200 for the prevention of immune mediated rejection in HLA <unk> mismatched kidney transplantation.
We have no dose for patients, including the first patient in the second dose cohort.
The product candidate continues to be well tolerated for all treated patients to date and we are encouraged by the early translational medicine data emerging from this study.
This quarter. We also received approval for an accelerated dose escalation protocol that will allow us to advance significantly more quickly through the dose cohorts in which adds a new fourth dose level to the protocol.
We have already successfully manufactured dose for patients in cohort three and for the first patient in cohort four.
These patients are enrolled manufactured and we knew when they will be dosed and we expect to dose the first patient in this highest dose cohort in January.
Meaningful year, and a half earlier than originally planned.
Acceleration to this top dose level, which is 18 foot higher than the starting dose could allow potential partners or investors to view efficacy as early as early next year.
While the progress being made in the clinical proof, citing the real value of this business is in what lies beyond the proof of concept with TX 200.
We believe this technology is ideally suited to treat autoimmune conditions with high unmet medical need and significant commercial opportunities and our preclinical work in multiple sclerosis, and inflammatory bowel disease supports that potential.
We have received external interests seeking ways to invest specifically in our car T Reg pipeline and it therefore been in active discussions with parties to explore ways that they can do so.
We have shared initial TX 200 data with the potential investors and we will continue discussions in an effort to realize the true potential of this exciting science.
If we're not successful in closing collaboration a financing transaction of the coming months, we will consider other alternatives for car T Reg cell therapy programs.
And we plan to provide an update on these efforts at the beginning of next year.
In the meantime, we've decided to defer new investments until were able to successfully secure a collaboration partner or external investment.
We believe that discontinuing spending beyond our continued current commitments are seeking potential partners and investors for both fabry and car T. Regs that are both better suited to develop each technology and have the resources needed to bring them to patients.
We will also allow the market to better assess each business is volume.
Okay.
As a result of all I've outlined today, we're announcing a reduction in our current U S workforce.
We also expect to close our Brisbane, California facility, nearly 24 to conserve cash resources and we will transition our headquarters to Richmond, California facility as of January one 2024.
Richmond is the original <unk> zinc finger editing and capsid development capabilities are based.
These actions are designed to focus our cash resources and advancing our zinc finger platform under capsid discovery engine.
We believe these changes in combination with the cost savings expected from the restructuring workforce reduction and other potential cost reduction initiatives.
Will reduce our annual operating expenses by approximately 50% and allow us to fund our planned operations into the third quarter of 2024.
Assuming no other additional capital is raised.
Alongside these announcements and with real personal sadness assured that Mark Mcclung Executive Vice President and Chief operating Officer will also be leaving the company.
In the context of a streamlined and more focused organization.
Mark can I felt it was important to align the leadership team with the changes being announced.
I've known Mark for many years and have an enormous respect for his wisdom judgment and leadership.
All of US have benefited from its business experience and constant reminder to focus on the patient.
A trusted confidante and colleague he will be enormously missed.
Until this departure Mark will continue to lead our search for partners investors and our Fabry and car T Reg programs.
Jason <unk> Senior Vice President and Chief Scientific Officer will also be leaving the company.
Jason scientific expertise is well known in the industry.
We've been lucky to have him guide or car T Reg and brought our scientific efforts.
Leaves a strong scientific legacy for which we will always be grateful.
With a clear focus on neurology is a cornerstone to sangamo going forward I'm pleased to welcome Amy Poehler head of research and Gregg Davis, our current vice President of genome engineering.
Engineering design and technology assigned most new head of technology.
These rules will be critical as we continue to advance our neurology focused pipeline and continue to innovate in the potentially life changing field of research.
I love, having look forward to having them both joined the leadership team.
These decisions were not made lightly.
And it's an incredibly hard to let go of such talented team members, who have dedicated themselves to advancing our mission.
However, we recognize that in order to move forward and to protect our future we must become a leaner simpler organization focused on progressing our neurology programs with a simplified and purposeful capital allocation.
And in turn increased flexibility to grow.
We are committed to aligning our investment strategies to our goals going forward, which are more focused than ever.
I believe in Sangamo, I believe and Sangamo as a standalone neurology focused genomic medicine company with our core pipeline and out licensing opportunities as a foundation of our business model going forward.
Through our neurology business, we aspire to apply our differentiated epigenetic editing capabilities and our normal engineered caps to revolutionize the treatment of neurological disorders.
Sangamo has both the differentiated genome targeted cargo and the delivery capabilities of the capsid to be.
Positioned favorably versus others in the field.
The data we have shared for enough one seven in prion disease programs provide promise for our program going forward and we have encouraging data from other advanced preclinical programs, including Tau for Alzheimer's disease, and Alpha nucleon for Parkinson's disease, which were progressed extensively as part of prior count collaborations.
And we had simply pause pending the identification of a suitable delivery capsid.
We believe we have made meaningful progress in identifying such a blood brain barrier penetrant capsid to work with our sister platform.
Which we believe will open the door for many other high value unmet diseases.
Can be addressed uniquely with our editing capabilities.
The neurology focused genomic engineering business is the future of Sangamo and it's a combination of a strategy that has been in the works for some time.
Because of the compelling focused business model represents and the potential commercial opportunities we believe it supports.
We're excited about the opportunities that lie ahead.
Opportunities are best for patients.
For advancing our science and best for the shareholders, who have stayed with us through this journey.
I would now like to turn the call over to our new head of research aiming to discuss the strategy of our neurology program in more detail welcome Amy.
Thank you Sandy and good morning, everyone.
You've heard today, we are on a journey to transforming <unk> into a pure play neurology genomic medicine company committed to driving forward development of potential treatments for neurological disorders in order to better serve patient I'll go into more detail on the value we see in this business and the strategy behind our neurology programs at a high level, we have a differentiated platform that can create.
Powerful genomic medicine powerful genome targeting cargo the preclinical data that demonstrates the potency specificity and importantly, the efficacy we may be able to achieve our technology allows us to target neurological indications and amongst direct and genetically validated way.
In defining our pipeline and pathway forward, which is to focus on indications where there is a clear driver of disease not only do we believe the strengthening the likelihood of preclinical success, but we also believe it increases the speed to potential approval.
Because the drivers of disease are well understood, meaning, we know, which which genes constant diseases, we're able to effectively target those genes that offer curative potential pursuing the fundamental drivers of disease is in our view a very compelling approach.
<unk> has highly differentiated technology that we believe allows us to execute on our strategy. We are developing proprietary genome targeting cargo as well as our own novel delivery captains, whereas model to most other companies are really doing one or the other.
The favorable AZ compatibility of the zinc finger proteins over other editing modalities have enabled sangamo to lead the field in neurological medicine development using a range of approaches, including epigenetic gene repression activation in multiplexed approaches.
This gives us both a competitive advantage as it progressed, our neurology pipeline and presents future opportunities for partnerships. It's important to understand that in genomic medicine drug consists of the cargo delivery capsid and promoter which enables tissue specific expression of this cargo. This agility is what becomes a treatment for patients with genomic with.
Kinetic diseases and the strength that we have as a company in the space lies in our control over all three of these components.
We believe this provides a strong foundation for the future of Sangamo, given our unique position and ability to lead in the area of epigenetic regulation as.
As a reminder, zinc fingers possess unique qualities that make them superior to other therapeutic approaches being explored there first the tile customizable allow for genome wide coverage and a very compact and as such were pleased to see that zinc fingers are increasingly being recognized in the field for these benefits and being more widely embraced.
Our promoters the zinc finger cargo and novel delivery capsid designed to work together to target specific cells and tissues and optimize efficacy and safety.
Able to powerfully control, the repression or activation of genes benefit not necessarily shared by other therapeutic approaches.
The favorable safety profile comes from specificity, we're able to achieve coupled with the fact that zinc fingers are human derived reducing issues related to immunogenicity.
Moving now to our pipeline, which leverages the delivery capabilities. We currently have in hand in combination with I think think of technology.
Our neurology pipeline is led by 1.7, which leverages the exquisite specificity zinc fingers to reprice, a target that has been challenging for other technologies.
In addition, we believe this program is important because of the broad population, we can address in small fiber neuropathy and intractable pain syndrome dominates the lives of these patients.
Plus the future opportunity to expand into other related indication.
We continue to advance the preclinical program and continue to anticipate an IND submission in 2024, we.
We have also defined prion disease is another important program in the pipeline.
We'll implement our epigenetic regulation technology, using our blood brain barrier crossing capsid.
Beyond these programs, we strongly believe we have the capsid engineering capabilities needed to expand delivery beyond our currently available interest eco delivery.
The industry broadly is challenged with finding or developing a capsid that can cross the blood brain barrier, we are making excellent progress identifying potentially effective intravenously delivered AAV capsid transport. These cargo and previously untouched areas of the nervous system and hope to share results from the nonhuman Primate studies in early 2020.
Sure.
Unlocking blood brain barrier penetrant delivery mechanisms would be transformative for our neurology pipeline.
Turning up significant untapped indications to development and releasing significant potential for additional thinking about partnerships.
Some of the high value targets, we could advance <unk>, which we prioritize solely because of the cats. It was not available at that time.
Sangamo has the technology and strategy around which we are building a focus neurology company developing all components of potential genomic medicine.
Engineering medicine. The data we have generated makes us confident in our ability to be leaders in this space and I look forward to providing updates in upcoming quarters.
I will now turn the call over to our Chief Financial Officer petition for an overview of the financial and Tusa.
Thank you Amy and good morning.
Over the course of 2023, we have proactively made difficult decisions to preserve our most valuable assets.
Public biotech markets have been challenging for over two years now and we find ourselves in a similar position to many of our peers with limited capital resources and a tough part to navigate ahead.
This is necessary to the exploration of our strategy execution and the focus of spending in line with our mission as in Urology focused genomic medicine company.
As Sandy outlined earlier this means optimizing our resources and continuing our efforts in seeking investment alternatives, all fabry and T. Rex.
Furthermore, as part of streamlining our spend we expect to exit our best <unk>, California facility in early 2024.
We will be scaling back our internal manufacturing capabilities and leveraging external partners to manufacture clinical supply for our neurology pipeline.
These actions have resulted in a reduction of approximately 162 rules are 40% of our U S workforce.
The annual cost cost savings, resulting from the workforce reduction combined with other potential cost reductions is expected to reduce our annual operating and.
non-GAAP operating expenses from 242 to.
$14 million to $16 million this year to around $150 million to $135 million in 2024.
The decrease of approximately 50% year over year.
We ended the third quarter with approximately $132 million in available cash cash equivalence and marketable securities. We believe that these resources and combinations with the cost savings expected from the restructuring and other potential cost reductions will be sufficient to fund our planned operations into the third quarter of 2024 without factor.
And any additional capital raises.
We continue to accumulate important phase one two data in the fabry disease, and TX 200, while we seek solutions to unlock the value within these programs, which could generate valuable capital for our neurology epigenetic regulation pipeline.
Additionally, we continue to evaluate several avenues to raise capital and hope to have an update in the next few months.
As a reminder, Pfizer continues to work towards a pivotal readout in mid 2024, and our hemophilia a collaboration and anticipates potential BLA and MAA submission in the second half of next year.
This partnership agreement alone allows for potential milestones of up to $20 million and 14% to 20% in royalties, which could be a significant source of funds talking as early at the end of next year.
We expect our 2020 for operating expenses for the core neurology company to be in the range of $80 million to $100 million.
This range excludes additional transition cost of approximately $30 million to $40 million in 2024, as we complete our strategic transformation, which are expected to significantly decrease in the future years.
We will provide official 2024 non-GAAP operating expense guidance.
Our fourth quarter update.
I'll now turn the call back to Sandy for closing remarks.
Thank you Patricia.
As you've heard today Sangamo continues to advance our transformation to becoming a neurology focused genomic medicine company. This has been in progress since 2020.
Against the challenging backdrop.
We've needed to make incredibly hard decisions for our employees and our programs in development and.
In the coming months, we will continue to work diligently turned back unlock value from our fabry and car T Reg programs and to drive forward, our neurology pipeline.
We will provide updates on these processes when appropriate to do so.
Thank you again to those very talented individuals to.
These friends and colleagues, who will be leaving <unk> youre.
Your tireless efforts and dedication have helped to progress Sangamo science and support the organization for that I am very grateful and I wish you the very best in your next chapters.
As I look to the future I'm excited by the opportunities that lie ahead.
We'll progressively share more data and use from our neurology programs and anticipate sharing nonhuman primate data showing our progress identifying new novel delivery caps. It's early next year.
The team continues to generate more preclinical data for enough one seven.
And has initiated final IND, enabling studies and so it's progressing towards an expected IND submission in 2024.
We're still anticipating the pivoted to retail for hemophilia a in mid 'twenty four with the first <unk> milestone potentially coming as early as next year.
As we work to raise additional capital we hope to announce an update in the coming months.
I am very thankful to our leadership team and all my <unk> colleagues for their hard work and dedication.
Dedication to our mission.
I'd like to close by expressing my hope and excitement for the future.
Future that lies ahead for <unk> and as a focused genomic medicine neurology company.
At this time, we'd like to open it up for questions. Operator, Please open the line for questions.
Thank you as a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced.
To withdraw your question. Please press star one again, please standby, while we compile the Q&A roster.
The first question comes from Reena Patel with RBC capital markets. Your line is open.
Hi, Thanks, so much for taking my question. This is <unk> I just wanted to ask.
Would you be able to kind of walk us through what would be an ideal partner and ideal structure for.
The Fabry program partnership.
Good morning. Thank you. Thank you for your question. So you're asking what is an ideal partner looked like for fabry.
<unk> spent a long time.
Okay.
Yeah.
So I think.
From our standpoint.
Our partner would be someone that has a commitment to them.
The patients with Fabry disease.
And sees the pathway that we've agreed with the agency.
And then has the capital resources to ensure that the study gets completed in a timely manner and commercialized global globally to these patients and there are a number of companies that fit that.
And we will provide updates when we finished conversations with.
Yes.
Please standby.
Okay.
These standby for the next question.
Yeah.
The next question comes from Andreas <unk> with Wedbush. Your line is open.
Hi, good morning, Thanks for taking our questions here from US here in kind of a follow up to the last one so just understanding capital constrained and the need to extend.
The cash runway favor is the most advanced program and there's good data to date.
He will also provide us an update on the phase III design, if there were any updates.
We're in discussions with the regulatory agencies, there and then.
And we noticed that Biogen had been selling a majority of the shares that you could provide any color around that that would be helped.
Helpful as well thank you.
Thank you for your question. So let me do the first one.
One of our fabric and I'm going to ask Nathalie to help us think about the design of the phase III.
I just wanted to Richard <unk> said in the script the results in February are.
Thank the most.
The most compelling of any clinical program my port tone in 25 years.
So holistic benefit from the patient and patients literally came up because of the recent February meeting and said.
I have your gene inside of me and I've never felt better mine life has been transformed by that and that's why as Mark says finding.
That can take this into phase III globally is so important not only for how are we doing with thinking about the phase III.
So yes, we were pleased to share last quarter.
The clarity we received from the agency on our proposed nave and Super Nice study design, we continue to have productive conversations with the FDA really a good place on potential phase three plan that can be activated in the future.
We also received <unk> designation.
Recently from the U S FDA.
Which really is a recognition that this potential medicine address significant unmet needs for fabry patient population, but also a signal that the data produced so far has been highly encouraging and those the AMA designation really provide the opportunity to have life conversation with the FDA. So we're very pleased that.
We will be in a very good position for any potential partner to start a phase III program.
Thank you Natalie and Patricia can you comment on the shear zone.
Your question on the Biogen Sam as you know my <unk> main businesses like us getting medicines to patients is not really in that this method of holding the.
Equity with other companies.
Our understanding is their main intention of this sale was to get under the 10% threshold.
And we're not expecting any future sales at this point.
Thank you.
Thank you guys. Thank you.
Please standby for the next question.
The next question comes from Maury Raycroft with Jefferies. Your line is open.
Hi, This is Kevin <unk> on for Maury.
Thanks for taking our questions.
Just wanted to.
You already touched on sort of a fabry phase III.
Where he left off of talks with the FDA, but I wanted to switch over to car T. Reg for that program can you talk about what went into your decision to to not show <unk> data by the end of the year.
That basically because you are getting to a potential efficacious dose earlier than you originally thought.
And then can you say, whether or not you looked at any of the renal biopsy data and then saw evidence of presence or expansion of T regs or any commentary you can share on safety so far.
Good morning, yes. Thank you.
Youre spot on with Phi we are delaying showing the data originally the the study design was three.
Three patients per cohort and then an SMC and then another three patients and with the with the complication of running a study in renal transplant that was driving the top dose we out into.
<unk> 25.
With our really novel clinical design from the clinical team.
<unk> managed to bring this in much quicker with with our completion design.
And we will have those patients in the top doors as early as January of next year.
Therefore, we will have a package of data that we can share.
Next year, rather than tripling cohort by cohort and as I'm sure you will agree that makes your job a lot easier.
As to what we've seen so far we've dosed three patients in cohort, one and one patient in cohort two and not only that they are doing extremely well on the safety of the product as been demonstrated for those two cohort.
We also in addition added a fourth cohort two are new design.
Is the dose that is 18 fold higher than the dose in cohort one.
We're very excited about this and it also demonstrated.
Manufacture capability for the T Reg.
Platform and we're very careful to say.
To hold office showing all the data until next year, we're encouraged what we've seen so far but really what will be most compelling would be if we show you all dose levels and for a larger group of patients. So we look forward to doing that next year.
Yeah.
Great. Thank you.
Please standby for the next question.
The next question comes from Nicole <unk> with <unk>. Your line is open.
Hi, this is going to sound on one for nickel sulphide, taking our questions.
Maybe.
Talk a little bit about your focus their efforts focusing on epigenetic regulation and therapies.
We're focusing on now.
This is a novel capsid is different area of greater foreign luxury interests and is that why youre focusing on it.
So I'm going to pass this over in a moment to Amy to suit Pawnee neuro is particularly good for our form of regulation.
Our decisions is a is a very.
<unk>.
As I said, we've been on this journey internally first three years.
Neurological diseases are enormous burden to society and we feel that this fits with our long standing technologies and allows us to make a difference in the fight to the zinc fingers repressive worked particularly well in this field.
Sandy we believe that the approach of using the zinc fingers and it gives us really an unprecedented opportunity to regulate gene expression on the brain and match it.
Typically to the neurological disease indications.
Being able to precisely and specifically up regulated or downregulates genes that are involved in is really challenging neurological diseases.
A new opportunity to treat those diseases. In addition, because of the zinc fingers are so small they package very easily into AAV capsid.
And that gives us another chance to not only design the cargo but to design the delivery the delivery vessel that will bring them to the areas of the brain or the areas of the nervous system, where they can have the biggest impact in modifying the disease progression.
Those things together the cargo.
And do you think that the zinc fingers plus.
The capsid that are able to deliver to the specific brain region. We think will make a really transformative impact and please not illogical diseases and many others are using <unk> are they up to inject often or inject interests equally well I will ever be once I'm done.
Right because neurons and other brain pulse are usually terminally differentiated it means that we predict that the expression of the zinc fingers are will be durable and master the lifetime of the patient. This is what we believe will be the case and we look forward to showing that in the clinic, but it does mean, we'll have a one and done approach.
Okay.
Thank you.
Please standby for the next question.
The next question comes from Patrick <unk> with H C. Wainwright Your line is open.
Hi, everyone.
Thank you for taking the qualities that Luis for Patrick.
Just like to ask about the newer programs that you still have going on for Alzheimer's with Tau Parkinson's with Alpha <unk>.
<unk>.
You said you have given us previously.
Increased penetration with the bank asset so.
You're going to prioritize this and these protocols now why are we going to see more data from them.
Amy can you talk about where we are with with Tokyo. So it's been interesting data on two recently hustler.
Yes, absolutely I mean with our previous partnership we're able to progress that program.
Quite a long way and so we have hired a clinical lead and zinc finger which has.
Really been able to sell some potent and specific repression of tile and the brands of our preclinical models and we look forward to pairing that in and future potentially with the blood brain barrier capsid that was the name of delivery across the nervous system and the results of those have shown in total gives us.
Reasons to believe and this is a tanker yeah absolutely.
There had been some.
Fantastic data, that's coming out in the field now showing that specific reduction of tile is able to remove some of the non familiar tangle of just under the pathology that's related to <unk>. So we think that that gives even more evidence that our tau targeted approach could be critical for trading something like alcohol, San Frans disease, not the other topic.
Thank you.
As a reminder to ask a question. Please press star one one on your telephone.
Please standby for the next question.
The next question comes from Danielle <unk> with Wells Fargo Securities. Your line is open.
Hi, Thanks for taking our question this is kwan for Yamana.
First question on TX 200.
I wonder, even though it's only like two cohorts, but I wonder if you have seen any dose response and.
Any immuno immuno suppressant tapering and what are the.
Potential partners might be looking for.
So there's two parts of that question with only dose one patient in cohort two and they were only recently dosed. So it would not be possible to know if there's any benefit no.
Started any process of tape.
Tapering.
As I said, it's going to be so much more.
Rich data set to show you the whole package next year.
Well our partners looking for there are many car T. Reg companies I think I counted over 10 of them.
Depot been.
Funded founded on preclinical promise.
And Sangamo is like the field here and we are the only the only one that has publicly said we are in the clinic and the fact that we've shown that we can dose for patients with car T. Regs and six I think is a really important thing for the whole field.
The first time.
Our T regulatory cell has been engineered and put into patients. We go from cohort one to cohort four and increases 18 food. So we have a great range of.
Amount of sales to us and we look forward to showing you that data next year.
Got it thank you.
On the AAV capsid can you share what are you hoping to show our two achieved in HP.
That would be in the first quarter next year data.
I mean, what do people look for in an H piece and what is good look like.
Thanks, Dan and I'm. So excited about the work that's being progressed by our capsid evolution group and these captives are essential for delivering that genomic medicine to the right place in the nervous system and all of the engineering work that goes into solving these challenges that the brain is kind of thrown thrown up at us.
But it's critical to to engineer that so that we're able to deliver the medicine.
We're looking for.
<unk> that are able to deliver at the zinc finger cargo broadly across the brain into brain regions that we know are involved in neurological diseases that we're targeting.
And we're able to do that using our preclinical models and try to understand how that will work and integration.
And the company.
That's why I'm. So excited about this work because we have the outstanding things in your platform, but also this capsid delivery effort that will allow us to create the medicines as I talked about before that aren't really bringing together those components and we're definitely looking forward to sharing more about that early next year. So it's about expression of the caps that drove the brain I suppose.
The activity of the zinc finger and it's about consistency and areas are clinically relevant that's for goodwill.
Yeah absolutely.
Yes.
Got it and that's one from me so.
He may program any comment on the potential of <unk>.
Monetizing the royalty thank you.
So so.
We can't really comment on any financing or monetization efforts, but not fully.
We really feel that Pfizer is doing a good job here and I don't mean.
Yes, all the patient in the pivotal trial as been dosed.
And really the Pfizer is still guiding to a pivotal readout expected in mid 2024 and submission of the BLA and MAA in the second half of 2024.
As you know you know affiliated as a whole and is it.
Is a big.
Area for Pfizer and they have all the the workforce to be able to advance this very efficiently into the market. So we're very encouraged and we have regular call without physically again and the momentum is.
Comparable I know you'll want to hear more about Pfizer every signal funds are giving us a positive that they believe this is and.
Unimportant program for them.
As regards to the monetization of the royalties if we monetize that know before the results are.
It would be an extremely discounted rate the longer we can wait before monetizing the better the more value will be.
Keeping it going all the way to the royalties and milestones coming directly to us would even be more important. So it's just a decision the position I have to make as we finance the company.
Got it that's helpful. Thanks for all the color.
I show no further questions at this time I would now like to turn the call back to Luis <unk> for closing remarks.
Thank you and once again, thanks to everyone for joining us today and for your questions. 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 you updated on our future developments.
This concludes today's conference call. Thank you for participating you may now disconnect.
Okay.
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Okay.
Okay.
Yeah.