Q2 2025 Sangamo Therapeutics Inc Earnings Call
Well, Keith head of Investor Relations and corporate Communications. Please go ahead.
Thank you good afternoon, everyone. Thank you for joining us on the call today.
On this call are several members of the Sangamo executive leadership team, including Sandy Macrae, Chief Executive Officer, Nathalie Dubois, Stringfellow, Chief Development Officer, and Patricia <unk> Chief Financial Officer.
Slides from our corporate presentation can be found on our website <unk> dot com under the presentations page of the investors and media section.
This call includes forward looking statements regarding <unk> current expectations. These statements include but are not limited to statements relating to sangamo as cash runway Sangamo has plans to obtain additional capital and its ability to continue to operate as a going concern the therapeutic and commercial potential and value of sangamo as product candidates and technologies Banco visibility to.
And maintain collaborations and strategic partnerships, including for its appropriate disease program, the anticipated plans and timelines for Sangamo and its collaborators, but clinical trials clinical data presentations and releases regulatory submissions and regulatory approvals upcoming catalysts and milestones and other statements that are not historical fact.
Results may differ materially from what we discussed today. These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC specifically in our annual report on Form 10-K for the fiscal year ended December 31, 2024, and our quarterly report on Form 10-Q for the fiscal quarter ended 30, sorry June 32025.
And subsequent filings and reports with Banco makes from time to time with the SEC.
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 will turn the call over to our CEO Sandy Macrae.
Thank you Luis and good afternoon to everyone joining the call today.
This quarter, we made important advances across both our clinical and preclinical pipeline.
In June we were happy to announce positive topline results from our Registrational star in Fabry disease, taking us one step closer on the path towards potential approval of this promising treatment for fabry disease patients.
This month, we also became a clinical stage neurology genomic medicine company with the initiation of our first clinical site in the phase <unk> study in chronic neuropathic pain.
This is an important achievement. It means we are on track to generate clinical data for this program anticipated towards the end of 2026.
Finally earlier this quarter, we held a productive meeting with the UK medicines and healthcare products regulatory agency or NHRA for preclinical prion disease program and are on track for a planned Cta submission for this program as early as mid 2026.
I'm proud of the progress and proud of my <unk> colleagues, who continue to work tirelessly to advance our pipeline will walk reaching in such a challenging environment.
Let me now hand directly to Nathalie Dubois Stringfellow, our chief development officer to provide more context on these important programs.
I will then close the call by summarizing the key takeaways from this quarter and will put these updates into perspective.
Natalie.
Thank you Sandy.
First I am pleased to share details of the recent positive top line results from the Registrational Phase <unk> study evaluating is around the TNT that pago back or S. T. Nine 'twenty, our investigational gene therapy for the treatment of adults with fabry disease.
Following a single dose of <unk> 20, a positive mean annualized estimated glomerular filtration rate or egfr slope of almost too was observed at 52 weeks across all three to dose patients in this study.
The FDA has agreed that mean egfr slope will serve as the primary basis of approval under the accelerated approval pathway.
Furthermore, a positive annualized egfr slope of one seven was observed for the 19 patients who have achieved two years of follow up.
I wanted to take a moment to reflect upon this important accomplishment.
As a reminder, the hour.
Richard untreated patient fabric.
Fabry patient experiences an annual decline in Etfs slope of minus three minus four.
Cheating a positive mean egfr slopes across all city to dose patients after one year and across the 19 patients who have reached two years is remarkable.
As we commended by the FDA, we plan to compare the annualized mean egfr slope of SD nine 'twenty with approved treatment for fabry disease by performing a meta analysis of published studies.
According to observer small studies other marketed treatment options such as replay Gal Sabra time, and got a fold show a decline in annualized egfr slope of minus two to minus 0.4.
Key secondary endpoint in the <unk> 920 study were also positive we continued to see strong durability. In this study with elevated expression of Alpha Gal a activity maintained for up to four and a half years for the longest treated patient and plasma Liza GBP three level that remained generally stable.
Following with the withdrawal of enzyme replacement therapy or <unk>.
Yeah.
We are excited to share for the first time, a stabilization in cardiac endpoint, including established Asian cardiac function and muscle article and biomarker data in the 32 patient with 52 weeks of follow up.
Measurement by MRI, including left ventricular mass left ventricular mass index length left ventricle myocardial global launches didn't know strain T. One antitumor <unk> and <unk> and end systolic volume remains stable over one year.
Furthermore, left ventricular ejection fraction measured by echo as well as cardiac biomarker, such as troponin and NT Pro BNP I remained stable in all patients at one year of follow up.
These data are striking partially given that cardiac function in fabry patient tends to decline over time and is a leading cause of death in fabry disease.
Patient demonstrated a range of other clinical benefit including improvement in disease severity reported in the first time Ssi age adjusted score and statistically and clinically significant improvement in the SF 36 quality of life scores.
<unk> a change of plus 15 in the raw physical score plus 10 in the vitality score and plus nine in the budget. He pain score at 52 weeks compared to baseline.
Statistically significant improvement in the gastrointestinal symptom rating scale compared to baseline were also observed.
I would like to particularly emphasize that SD nine 'twenty has been well tolerated in this study the majority of adverse events were grade one or two in nature without the need for pre conditioning. There was no safety related study discontinuation or death.
We believe that the totality of this compelling data demonstrate the potential for a single dose of <unk> 20 to treat the underlying pathology of fabry disease.
Provide meaningful clinical benefit above current standard of care.
S. T 920 has shown the potential to transform the lives of patients and we have observed additional clinical benefit in some including the reduction and elimination in pain medication usage and the resumption of sweating, which has a nimble these patient to perform physical task and exercise they were previously unable to do.
Following dosing with SG 920 old patient who came in the study on New York you were able to safely we drove from ERP with one patient now our CRT for more than three years.
In so doing these patients have already avoided more than a thousand biweekly E. R. T infusion each of which can last up to six hours what a transformation in the life of these fabry disease patients.
Since the top line readout in June 2025 physician has decided to resume ERP for one of their treated patient who headwinds thrown from Iraq.
These patients received <unk> 920, more than two and a half years ago maintain super physiological level of Alpha Gal a activity and also would you be three levels were generally stable as of the topline readout date.
All of the other 17 patients who began to study on the RT and as we strove for me Archie continue to remain a CRT as of today with many experiencing benefit of S. T 920 over and above what they were expect parenting with <unk> alone.
Also in two patient of transition in the long term follow up study and the STAAR study is now complete we continue to engage with the FDA ahead of our anticipated BLA submission under the accelerated approval pathway planned for as early as the first quarter of 2026.
We are also looking forward to sharing in additional clinical data at the 15th International Congress of inborn errors of metabolism or IC I E. M 2025, taking place September 2nd 262025 in Kyoto Japan.
Before we move on and on behalf of our entire Fabry team at Sangamo I wanted to take a moment to sincerely. Thank the patients and investigators who have participated in the STAAR study.
Your dedication and commitment have been invaluable as we advance this treatment for such a debilitating and multifaceted condition towards registration. Thank you.
Okay.
Turning now to our neurology pipeline programs are sandy shared this quarter, we become a clinical stage neurology genomic medicine company with the initiation of our first clinical site in the phase one two study study evaluating S. T. Five O suite, our investigational epigenetic regulator for patients.
With intractable pain due to idiopathic small fiber neuropathy or I S. F N.
This is an important milestone for sangamo and we're excited to be identifying patients in our first ever neurology clinical trial I want to thank everyone involved.
We anticipate activating at least eight other clinical site in the coming months, which we believe will further accelerate patient enrollment.
We expect to dose the first patient in the fall of this year and anticipate having preliminary proof of efficacy data in the fourth quarter of 2026.
Our preclinical data for this program is compelling by directly targeting the <unk> gene and C. Five OS we have shown to precisely and potentially reduce the expression of <unk> $1 seven sodium channels in sensory neurons in animal models and significantly reduce pain I person Sidoti following a single intra.
He co administration.
As <unk> has been well tolerated in nonhuman primate with no off target effect observed.
And we plan to present updated non clinical data at the ninth International Congress of Neuropathic pain, taking place September four through six in Berlin, Germany.
Okay.
Finally, I am pleased to share our progress in S. T 506, our epigenetic regulator for the treatment of prion disease to be deliver intravenously using stack BBB.
Earlier this quarter, we held a productive meeting with the Uk's, Emma Cherry and align on the planned non clinical safety study.
As well as the proposed clinical study design, we appreciated the collaborative nature of the discussion and the knowledge of the urgency to final treatment for prion disease patient.
We were also extremely proud to be selected to present during the prestigious presidential symposium at the recent recent S. TCT annual meeting in New Orleans, where we showcased a potent combination of epigenetic regulation and capsid delivery technology in prion disease.
Including the profound survival benefit we observed when administered to post symptomatic symptomatic mice.
In addition, we described the sustained brain wide suppression of prion protein expression in both mouse and non human primate model supporting the potential of S. T five or six as a one time therapeutic approach for a prion disease.
We have completed dose ranging finding study and are preparing for the GOP. Tox study ahead of an anticipated Cta submission expected as early as mid 2026.
I would like now to hand, it back to Sandy for closing remarks Sandy.
Thank you Natalie.
To close we made strong pipeline advances this quarter.
Firstly, we announced positive topline results from the Registrational Star study in Fabry disease, we observed a positive mean annualized Egfr school at 52 weeks across all dose patients in the study, which the FDA has agreed to serve as a primary basis of approval.
Beyond renal function, we were pleased to observe a range of positive secondary.
And points and brought her quality of life data, including a stabilization kartik endpoints.
And unfortunately, <unk> continue to be very well tolerated in the study without the need for pre conditioning.
We continue to engage with the FDA ahead of the planned BLA submission expected as early as the first quarter of 2026.
Secondly, this quarter, we became a clinical stage enroll a genomic medicine company with the initiation of the first clinical sites for the phase <unk> study in chronic neuropathic pain.
We expect to dose the first patient in the fall and anticipate having preliminary proof of efficacy data in the fourth quarter of 2026.
And thirdly, we held a productive meeting with Ami Cherry for SG, five or six in prime disease ahead of an anticipated Cta submission as early as mid 2006.
Moving now to broader business updates early this quarter, we completed an equity offering that we hope will bridge us to an anticipated fabric commercialization agreement.
Our current cash runway is expected to fund our planned operations into the fourth quarter of 2005.
And we remain highly focused on our critical task of securing a fabric commercialization partner in the near term.
We continue to advance business development negotiations for that potential pre.
Pre commercialization agreement and are also engaging in broker business development discussions across our single pipeline and platforms, including our <unk> platform.
We remain focused on solving our long term funding needs in order to enable us to advance our promising urology genomic medicine pipeline.
Operator, please open the line for questions.
Thank you at this time, we will conduct a question 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 withdraw your question. Please press star one one again, please standby, while we compile the Q&A roster.
The first question comes from the line of Maury Raycroft of Jefferies. Maury. Please go ahead.
Hi, This is James on for Maury, Congrats on the progress and thanks for taking our questions. Just start off has the team already held the pre BLA meeting with the FDA to discuss the potential path to approval using the one year Egfr data.
As a predictor for the TUI two year Egfr benefit versus having to confirm the clinical benefit with two years of data from all patients.
And if you have that meeting could you share any takeaways from that discussion also how important is the FDA FDA alignment on the two year Egfr prediction in the context of ongoing.
Partner discussions and I have a follow up.
Okay.
Thank you for the question so.
Let me just restate, we held a meeting last year with the FDA, where they agreed to an accelerated approval.
Meeting they said that we could achieve accelerated approval with one year Egfr data.
And included a close and as I said, we might.
To submit two year data.
When that was available.
We currently have 32 patients at one year and 19 patients at two years.
The two data sets are very similar and complementary we have not yet held a pre BLA meeting because it's not the time to do it yet.
And have plans in place of when and how we're going to do that.
The pre BLA meeting is very much an operational meeting where you agree with the agency on what is that you have to do to fulfill the BLA requirements for sections, how it has to be presented etcetera.
We have no.
Expectation that the agency will require anything other than the one year data for accelerated approval and we are sure that we will end up.
Providing them with yearly updates as these patients had funds. So just to remind you the latest the.
Earliest patient is now four and a half year or so.
The data looks very consistent and very stable.
Got it thanks for that and then just another one for the upcoming presentation Ssi what additional insights should we anticipate can we expect any details of the conference regarding the meta analysis or new baseline characteristics such as parking area also will you show individual egfr trajectories or alpha Gal level.
For each patient or just the average.
So I'll pass this on to Natalie, but before I do that I would say is it would be really unusual in a patient dataset of 32.
Patients, where the is comparing the body of patients before compared to after.
To dissect to ensure each individual patient so we don't intend to show that the.
The presentation at the <unk> meeting in Japan.
We may do that just part of a larger publication that we're working on at the moment.
Lee.
Yes. Thank you Sandy we look forward to sharing additional clinical data at the IC I Am conference.
We plan to present, the top line data with additional details compared to the press release issued back in June.
We encourage you to review the data presented at I C. I am that will also be made available in our website once the embargo is lifted.
Got it not clear our plans would be to see a little more about the resolutely yeah, absolutely yeah, there will be more details on some of the endpoint.
Yeah.
We're still finalizing the presentation, but there absolutely will be additional details.
Got it thanks for taking my questions I'll hop back in the queue.
One moment for your next question.
The next question comes from the line of route who are all PD Cowen <unk> co. Please go ahead.
Hi, Sandy this is Joshua fleishman on the line for Ritu. Thanks for taking our question.
Curious how do you believe SP fiber <unk> efficacy and NAV, one seven will compare to the recent small molecule NAV one eight inhibitors and have recent trial outcomes in NAV, one eight changed your conviction and knock one seven as a target. Thank you.
So thank you very much for your question and we've spent a lot of time looking at that data and discussing them.
And.
London that we are even more convinced enough one seven what's the right target for us to go for us.
Sure.
I think we've discussed before.
Because we are using a genomic ways to target and target the specific.
Regulatory sequences of the Chin, we could've gone for novel appointed or enough one seven and our belief was that the Norfolk southern control of the action potential that controls the pain signal with a more fundamental control and one of the real pieces of evidence for that is that.
There are people out there that have good continuous mutations with enough one southern but just don't feel any pain.
There it is.
Or is it very rare incidents as reported if not form eight mutations and they don't seem to have complete suppression of pain. So perhaps it isn't so surprising that the one eight results reported by vertex we're not as efficacious as had been hoped.
So we are at the stage note of.
Activating this study and hopefully we will have identified and recruited patients soon.
We look forward to this it's a dose range finding study and in our most studies, we see evidence of a dose range response, even in individual groups of mice.
We look forward to showing the suppression of pain, because it's it's a really important unmet medical needs, great credit to Lilly and vertex and others, who are no pushing forward with non opioid pain relief, but we believe enough. One seven is the right target to go for.
Great. Thank you so much.
One moment for your next question.
The next question comes from the line of Yanan, Zhu of well part of Wells Fargo's excuse me. Please go ahead.
Hi, Thanks for taking our question. This is Kwan oncor Yana. So our question is also around fabry.
I'm wondering have you done any survey to either patients or physicians to understand that.
Current product profile, what could be the potential adoption rates. Thank you.
Can you just repeat that question again please.
Sure.
Have you done any survey to either physicians or patients to understand that.
<unk> current product profile of what could be the potential adoption rate.
Luckily you spent a lot of time with the patient support groups, what's what's your thoughts on this yes, so from the patient advocacy group.
They are waiting for a better solution for a long time. The current standard of care is burdensome, but and there is.
Some small improvement in their disease, but it really does not address or the symptom of the disease.
It's a biweekly infusion that lasts many hours, which really impact their daily life.
What we showed in our.
Top line results for our patients that we.
We have really improvement in the quality of life. It's a one time injection and patients are uniformly saying this is what they're waiting for for a long time.
So there are really eager to see the strike who some of the patients because it's a genetic condition once their family to have access to the product as soon as possible. So we have an overwhelm a overwhelming response from the patient community.
The RPI our principal investigators that are taking care of those patients are also extremely enthusiastic when theyre reviewing the data there are really a very impressed by the way. We've accomplished so we do believe that the adoption rate both on the patient side and the dark side.
It will be very impressive and you've met with cardiac experts recently and they were very impressed yes, we've met Sui skyjack expert to review on our topline data with all the cardiac endpoint I mentioned.
And first of all they mentioned that.
We had many many measures in the cardiac <unk>.
<unk> function that other study didn't have that that was the most.
A comprehensive set of data and they were also very impressed with the data.
And the stabilization of the cardiac function.
So you know we're focusing for the primary basis of approval on the Egfr slope in the renal function. But this is also a very important aspect of fabry disease.
The thing that when we go to these conferences, we often have fabry patients come to us and tell us that they have received our treatment.
March their life has changed and that kind of conversation spreads through the fabry support groups in populations.
We feel there's a real energy in anticipation.
And then the final thing I would say as you look at the <unk>.
17 patients who came in on <unk>, who have remained offer VRT nuts over a thousand infusions and the <unk>.
They feel better and the.
SF 36 score say that they are better on this treatment and so I really look forward to solving the commercial partner and getting this medicine to patients as soon as possible.
Alright, thanks for the color and.
Don't know if you can comment on this but with the potential partners do they share the same view or are they looking for something else.
Beyond what you have sure thank.
Thank you.
So uniformly all of the partners' hubs all of potential partners have.
So that's how excited they are of the data.
Yes.
Our.
Completely convinced that this is a medicine that is both safe and shoes on effectiveness and a benefit to patients.
Don't think so.
I am sure you're like cause are aware of the.
The environment for gene therapy at the moment in the United States.
Two the stability that we all hope for and look for from the agency <unk> has had great interactions with the agency and continues to have them.
And the partners just want to know that this is a staple.
Please wear their medicine will be well appreciated and taken forward.
There is a second piece.
Piece to that that makes the sangamo discussions a little unusual in that since we got accelerated approval last year, we have compressed.
Terrific piece it takes you to.
The BLA submission into a very short time, which means that there are lots of interactions and lots of data points and new information coming.
<unk> partners, who will have in the past been interested in seeing the top line data of wanted to know that we have as we do have agreement on the CMC. So we feel that this product is increasingly derisked.
For the partners that give some comfort to be able to move ahead and I'm very pleased with the.
Pace of negotiations at the moment and look forward to finding a positive way through this.
Got it. Thank you so much for the colors.
As a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced.
One moment for your next question.
The next question comes from the line of Gena Wang of Barclays. Gena. Please go ahead.
Hi, This is Tony on for Gena, I guess questions on upcoming updates in September what data points should we be looking for for the pain program in terms of how they would.
Compared to existing <unk> programs, and then also what incremental color should we expect for the February update.
So the data that we'll be presenting at the conference in Berlin is a preclinical data and we will share more information about our G. L. P talk studying and HP.
And.
It will be also all the information on the mouse data.
And the non <unk> Tox study, but the additional G. L P showing the safety and efficacy of the product will be presented.
Okay. Thank you.
One moment for your next question.
The next question comes from the line of Lee <unk> of H C. Wainwright. Please go ahead.
Hi, everyone and thank you for taking our questions songs.
My question is mostly regarding your.
Cash runway and cash burn associated with especially associated with initiation of.
The stand trial, where you plan to continue activating more sites Anderson.
Dosing the first patient by the end of the year, how is that going to weigh on your cash burn and also do you have any updates on human platform any recent additional data or coming thank you so much.
Hi, Louise this is pretty sure I'll take the first one so our intention is to continue taking the NAV one seven program forward and dose patients as intended as Sunny mentioned, our number one priority is finding a fabric commercialized station partner and that will help us fall Forum.
Funding needs in both in the long term in the short term and maybe let me turn it over to Greg to answer the question on the <unk> platform.
Yes, Thanks Louise.
We were we were happy to share the updates on the med platform at <unk> recently, you probably saw a lots of data there.
Relation to integration and.
Our improvements in integration rates and primary cell types. So we're happy to share that data this year and we continue to.
Show that data to interested parties and engage in discussions with.
Parties interested in collaborating with us and we have a number of ongoing discussions on the main platform right.
Okay.
Thank you and going back to the <unk> trial, you said that you might have data.
By the end of next year, what kind of data should we expect.
Okay.
So you can expect.
Safety data from the patient and early efficacy data.
For the dose escalation trial.
Got it thank you so much.
And let me just.
You can be sure we're doing all the standard pain study scores sleep assessment scores, even suicidology scores because these are patients whose life is dominated and tragically dominated by this.
We will be all.
So it would be a 12 week endpoint that we would be showing by the end of next year, but we'll be following them long term because we think the huge advantage of nerf, one seven or so genomic medicine is the long term benefit it will bring to the patients.
Yes, we expect.
Sure.
Yeah go ahead.
No no I'm sorry go ahead please.
I was just going to say thank you for any other color, but if you have more color always welcome.
No I think sandy mentioned it so.
We've got.
Thank you.
I am showing no further questions at this time I would now like to turn the call back to Louise bogie for closing remarks.
Thank you once again for joining us today and for your questions. As a reminder, you can access our presentation on the Investor Relations section of the Sangamo website, we look forward to keeping updated on our future developments.
Thank you for your participation in today's conference. This does conclude the conference you may now disconnect.
Thank you we appreciate your help Felicia Thank you Lisa.
Yeah.
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Yeah.
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Okay.
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