Earnings Labs

Sangamo Therapeutics, Inc. (SGMO)

Q2 2017 Earnings Call· Wed, Aug 9, 2017

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Transcript

Operator

Operator

Good afternoon and welcome to the Sangamo Therapeutics Teleconference to discuss Second Quarter 2017 Financial Results. This call is being recorded. I will now pass you over to the coordinator of this event, McDavid Stilwel, Vice President of Corporate Communications and Investor Relations.

McDavid Stilwel

Management

Good afternoon and thank you for joining Sangamo's management team on our conference call to discuss the Company's second quarter 2017 financial results. As we begin, I’d like to point out that we will be referring to a slide presentation this afternoon. You may find a link to the slide presentation on our website, sangamo.com, on the Events and Presentations page of the Investors and Media section of the website. I'd also like to remind everyone that the projections and forward-looking statements that we discuss during this conference call are based upon the information that we currently have available. This information will likely change over time by discussing our current perception of the market and the future performance of Sangamo with you today, we are not undertaking an obligation to provide updates in the future. Actual results may differ substantially from what we discuss today and no one should assume at a later date that our comments from today are still valid. We alert you to be aware of the risks that are detailed in documents that the Company files with the Securities and Exchange Commission, specifically our Annual Report on Form 10-K and on our quarterly reports on Form 10-Q. These documents include important factors that could cause the actual results of the Company's operations to differ materially from those contained in our projections or forward-looking statements. With me today on this call are several members of Sangamo’s senior management including Sandy Macrae, Chief Executive Officer; Kathy Yi, Chief Financial Officer; Ed Conner, Chief Medical Officer; Michael Holmes, Vice President of Research; and Curt Herberts, Chief Business Officer. And again, we will refer to a slide presentation during this call. Those slides are to be found on the Events and Presentations page of the Investors and Media section of our site. And now, I'd like to turn the call over to Sandy.

Sandy Macrae

Management

Thank you, McDavid. And welcome everyone to our conference call to review business and financial highlights from our very busy second quarter. We continued to make strong progress repositioning Sangamo financially and operationally for its leadership role in the development of genomic therapies. During the second quarter, we executed two significant transactions. We and Pfizer entered into the SB-525 collaboration for the development of the AAV gene therapy for hemophilia A. This agreement will strengthen the SB-525 which is on our program and greatly accelerate the potential global commercialization of this product candidate. We are excited to work closely with Pfizer and with their advanced capabilities in gene therapy delivery and product developments. The financial terms were also very attractive that included a $70 million upfront payment as well as $475 million in potential milestone payments and a tiered double-digit royalty on net sales. Beyond putting our hemophilia A gene therapy program into collaboration with the right partner, we believe the Pfizer collaboration announcement has served us an important catalyst for Sangamo raising awareness of the quality of our science amongst other potential business development partners and amongst investors too. Six weeks after the Pfizer collaboration announcement, we announced a second major transaction for the quarter. A follow-on offering of common stock raising $78.1 million in net proceeds. The transaction allows us to broaden our shareholder base and to conclude the second quarter with a significantly strengthened balance sheet. And Kathy will provide details later in the call of our use for these funds. Both these actions are positioning Sangamo for an exciting future, as Sangamo purchased over a year now and I am more confident than ever in the prospects for the company. I believe the scientific platform here is unrivaled and as Curt will discuss is increasingly recognized…

Kathy Yi

Management

Thank you, Sandy and good afternoon to everyone joining on the call. We issued a press release earlier today that included detailed financial results for the second quarter of 2017, and updated guidance for the remainder of the year, which I will summarize on Slide 11. Later, I’ll be happy to answer questions about the quarterly financial results that are extensively laid out in the press release. In my comments today, I’ll focus on the cash utilization portion of our updated guidance. This updated 2017 guidance reflects $70 million of upfront cash received from Pfizer during the second quarter. We are accounting for this payment as deferred revenue and recognizing it on a straight-line basis over a 32 months period. Including funds raised in the follow-on offering we completed in June, we ended the second quarter with $266.5 million in cash, cash equivalents and investments. Based on our quarter end cash position, and with projected full year operating expenses revised partly lower to a range of $90 million to $100 million we expect to end 2017 with at least $220 million excluding any potential new business collaboration or milestone payments. We believe we are in a solid financial position to fund our lead clinical development and middle pipeline programs through a key value inflection point. In the months ahead, we anticipate proof-of-concept data from our four lead clinical trials, the manufacture of pivotal clinical trial materials which we recently initiated, the progression of our Fabry gene therapy program, and Bioverativ partnered beta beta-thalassemia and sickle cell disease programs into the first in human studies, as well as ongoing investments in our core zinc finger approaching technology platform and novel delivery modalities. We believe Sangamo is in solid position to expand our future pipeline and unlock value for our shareholders. And with that, I will turn the call over to our CMO, Ed Conner for an update on our clinical programs. Ed?

Edward Conner

Management

Thanks, Kathy. I am very pleased to share a status report for activities across our four clinical programs. I’ll start with SB-525 for hemophilia A on Slide 14. We opened our first two sites in the second quarter that have now two patients who are qualified to enroll based on serology testing. We expect to do as the first patient in the study this month as we coordinate the 24 hour observation visit at the participating site. We also have a number of additional sites being activated this month and next and expect to have six study sites opened by mid-September. One common question I am asked is, how do evaluate success for the hemophilia program and it really comes down to the factor activity levels. For hemophilia, these activity levels correlate tightly with the outcomes, patients and physicians care most about namely reducing spontaneous bleeding and use a recombinant factor replacement therapy. Data show that once factor activity levels exceed 12% incidence of spontaneous bleed drops to near zero and patients don’t need factor replacement process. Success for this study then is having factor activity to be well above that level while avoiding very high levels exceeding normal as there maybe the potential for clotting events. Turning to Slide 15, I’ll move on to SB-FIX for hemophilia B. Enrollment in this study remains challenging as we are behind other programs with promising data. We currently have four sites opened and expect to open an additional site in September. Patients and physicians continue to be interested in this program as it has potential as the treatment for children to provide life-long production of factor levels that eliminate the risk of bleeding and use of replacement products. On the next slide, starting with SB-318 for MPS I, we have two sites…

Michael Holmes

Management

Thanks, Ed. As Sandy mentioned earlier, Sangamo is evolving rapidly as a company that has tightly integrated and focused on achieving common goals. Research is working closely with our internal and external partners on clinical and product development efforts. While our 20 year history has been discovering ground-breaking science, our future is taking our innovative discoveries for the development of new human therapeutics. Today, I’ll provide a brief update on our middle pipeline including our Bioverativ partnered product candidates for beta-thalassemia and sickle cell disease. Our AVG therapy for Fabry disease and our ZFN media in CAR T and TCR immuno therapies for oncology. Turning to Slide 19, I’ll start off with our Bioverativ partnered cell therapy program for beta-thalassemia and sickle cell disease. Both diseases are inherited, genetic blood disorders caused by two different mutations in the beta-globin gene, which we can address with in common approach using our ZFN-based cell therapy platform. Our therapeutic product candidate, ST-400 and BIVV-003 for beta-thalassemia and sickle cell disease respectively are designed as the target cell therapies made from a patient’s own hematopoietic stem cells. As shown in the cartoon on the bottom right of the slide, these stem cells are identified from the patients and genetically modified ex vivo using ZFN can increase the expression of Gamma or field globin gene while reducing expression of the mutated beta-globin gene. This results in the production of normal function fetal hemoglobin when the modified stem cells differentiate into red blood cells inside the body. This is a highly differentiated therapeutic strategy that harnesses a natural, protected mechanism of the body. In contrast to randomly integrating retroviral based gene therapies in development, our approach involves non-viral delivery of ZFNs. A strategic advantage that allows for more controlled and precise genome editing with a potentially…

Curt Herberts

Management

Thank you, Mike. As we developed our technology over the past several years, we focused on building out platform – the next ZFN media to genome editing, highly differentiated from the competition and to create best-in-class human therapeutics. From our perspective, there are three main criteria to achieve this goal. One, precision, the ability to target any desired nucleotide in the genome, two, efficiency, the ability to create a permanent targeted double – with a specific nucleotide center and three, specificity, the ability to cut at the targeted nucleotide of interest without cutting elsewhere in the genome. These are the three attributes that we believe are required for editing technologies that will ultimately become therapeutic products for patients, products on which we will build our long-term business. Over the years, we have assembled the library of thousands of individual well characterized zinc finger approaching. More recently, we have advanced a series of new ZFN architecture which has greatly increased the flexibility of the platform. This year, Michael Holmes and Ed from our research team have been presenting on the new ZFN architecture and other platform improvements at leading scientific meetings. We now have a ZFN platform with an unparalleled design density with thousands of one and two finger modules in our library and an array of links attaching the zinc fingers and the top-line nucleases. We are able to assemble many highly specific ZFN pairs for any chosen target sites. So let’s discuss why this mattered practically. Turning to Slide 24, with the origin of DNA around the beta globin genes, for any given 20 base per window we have on average 450 distinct ZFNs can figure right that choose from out of an existing library. Across the 400 base target sites pictured here, we have thousands of distinct ZFN…

Sandy Macrae

Management

Thank you, Curt. We are pleased to report our progress today, a continuation of the studies and strides we have been making throughout this year. We are building Sangamo into a company that has the infrastructure and management capabilities to reliably deliver on our goals. We are solidifying the clinical operations capabilities which we need to recruit and execute our rare disease studies. For me it’s not about the enhancement of the first patients into these studies which will come soon enough, but rather the confidence that we have the machinery in place to fill the trials steadily. That has to do with which we are building Sangamo’s development capabilities. Similarly with our middle pipeline, I am very pleased to report progress towards INDs and 2018 clinical trial starts. Next year, these will be our newest clinical programs and with Sangamo’s robust R&D engine, we have programs that will advance from discovery into preclinical research in preparation for INDs. There is such tremendous value in Sangamo’s platform which is a potential to deliver new assets for movement through early research and into the clinic. Curt discussed the value of the ZFN platform. Biotechnology as both science and business and we are marrying our scientific excellence with clinical delivery and commercial planning and are thinking holistically about the strategies to create the most value from our assets. We will forward integrate to develop and commercialize certain programs ourselves, but we will also continue to externalize R&D through collaborations in order to advance assets. Our truly ventured developed and partnerships, we are very fortunate to represent with such a rich pool of opportunities. Operator, we are now ready for questions.

Operator

Operator

[Operator Instructions] Our first question comes from Ritu Baral with Cowen & Company. Your line is open.

Ritu Baral

Analyst

Good afternoon guys. Thanks for taking the question. I wanted to ask about the MPS I – actually the MPS programs in general and how you are looking at GAG levels and enzyme trough levels? What is your target enzyme trough level given what’s necessary in MPS I to – I guess, clinical benefit and you mentioned you are in MPS I, does the trial protocol gives any opportunity to measure CSF GAG levels as well? And I’ve got a follow-up.

Sandy Macrae

Management

Okay. Thank you for your question. Nice to hear your voice. I am kind of passing this on to Ed.

Edward Conner

Management

Sure. So, to answer your second question first, we are looking at the CSF and looking at levels of enzymes there. For your first question, regarding the levels that are needed of the enzymes in the bloodstream, we know that in patients on ERT they are going to be as essentially their baseline levels which is as or near zero and in talking to investigators and in talking to physicians who treat this disease, they think the amount of circulating levels that we need to get our quite small to show clinical benefit. And we need them above 2% or 5% is likely to have clinically meaningful benefit if it’s circulating continuously being produced by the liver. We are tracking these levels and the real test as I mentioned earlier will be following the GAG levels, because you want to know what’s happening in the – what’s happening in the lysosome. So once we see these levels start to come up in the bloodstream following the urine GAGs and seeing those GAGs stay low after patients have discontinued their ERT, but to continue to benefit from our therapy will demonstrate success for this study.

Ritu Baral

Analyst

Got it. And then a quick question on the hemophilia A program. You mentioned patients qualified for enrollment, are there any gating factors for treating these patients? And also, Kathy mentioned some expenses for production of pivotal trial material. Was that for hemophilia A or is that for another program?

Sandy Macrae

Management

So, let me answer, give you the answer to this. Do you want to first answer with the patients?

Edward Conner

Management

Yes, so, for the patients, there is nothing gating in terms of screening activities. It’s really just a logistic issue because it requires an overnight stay. And so you need to slot in for those spots. So, that’s really what we are waiting on in terms of getting that set up in the participating site.

Sandy Macrae

Management

Kathy?

Kathy Yi

Management

Yes, so, pivotal clinical trial materials is for our four lead assets mainly the MPS I and II.

Ritu Baral

Analyst

Got it. Thanks for taking the questions guys.

Sandy Macrae

Management

I want you to take away from this is, we are thinking of this more holistically, more like a – company would where you think of – you have to plan ahead for the next stage of development have the clinical development pawn in place after manufacturing done to minimize any delays between the different phases of development.

Operator

Operator

Thank you. Our next question comes from Maury Raycroft with Jefferies. Your line is open.

Maury Raycroft

Analyst · Jefferies. Your line is open.

Hi, thanks for taking my questions. I just had two quick ones. I was wondering for AAV 2 and six shutting or clearance over time. If you could just remind me what the data is in non-human primates and is it’s strictly dose-related?

Sandy Macrae

Management

Thanks for your question, Maury. Michael, could you answer that one?

Michael Holmes

Management

Yes, in terms of vector shutting data, so we did monitor this in our preclinical studies including non-human primates and this was tracked over the first several weeks where we could detect showing the vector in a lot of the secretion. But it rapidly cleared in non-human primates and we didn’t see any real differences in sort of the AV 6 vector versus other vectors that have been published on that is clinically including AV 2 as well as AV 8.

Maury Raycroft

Analyst · Jefferies. Your line is open.

Okay, great. And then for the sites that have been selected, so far that are posted on clinical trials, I was just wondering if there is strategic rationale for those?

Sandy Macrae

Management

So, that’s another good question, Ed, how do you think about choosing sites?

Edward Conner

Management

Well, it’s first and foremost based on sites and investigators with clinical expertise and not just in treating the disease but in doing all the parts of study enrollment making sure that the data is entered appropriately and that the data can be used in registrational activities later on. But in other factors to consider is, because these trials are fairly involved with clinical trials. As you want to make sure you have good geographic spread within the United States. So that patients are able to hopefully go to a site where they are being treated or if not, not have to travel a great distance. So, for me, it really both with them trying to lessen the burden as much as possible for patients enrolling in these studies, but also making sure that we have clinical trial sites that are excellent in conducting clinical trials, but also excellent in treating these diseases.

Maury Raycroft

Analyst · Jefferies. Your line is open.

Okay, great.

Sandy Macrae

Management

Okay.

Maury Raycroft

Analyst · Jefferies. Your line is open.

Thank you very much.

Operator

Operator

Our next question comes from Charles Duncan with Piper Jaffray. Your line is open.

Unidentified Analyst

Analyst · Piper Jaffray. Your line is open.

Hi this is Sarah on for Charles. Good to see some of the progress on the clinic over the past couple of months. On MPS I and II, in the slide that looks like you expect preliminary data around year end or early 2018 and can you just remind us what you include in that category in terms of number of patients and efficacy or safety parameters that you will share?

Sandy Macrae

Management

So, that’s a good question and as we said before, we are looking for the clinical trials – each of the clinical trials to come in towards year end or most likely in early 2018 and we will report the data as we see clinically significant important results. So it will all depend how the data comes out and how many patients are we have treated at that point.

Unidentified Analyst

Analyst · Piper Jaffray. Your line is open.

Okay, thanks. And just one follow-up on the MPS indications. Can you speak about the rate of change in patients screened over the past couple of months and whether that’s picking up and then along those lines, how much awareness of this program do you think there is among the…

Sandy Macrae

Management

We are trying, and I will pass this over to Ed, but just to be clear, you can’t screen patients until a trial site is up and running. And so…

Edward Conner

Management

Yes, so, in rare disease in – rare disease really enrollment all those follows site activation. You need to get the sites opened because you can’t start treating patients until you have an open site. And so the flurry of activity that we are seeing this month and next, we’ve already seen – to answer your question directly, we’ve already seen an increase in screening that’s been steadily growing over these last few months and as we now open the remainder of our sites, both for MPS I and MPS II, I expect to see that to continue to grow substantially.

Unidentified Analyst

Analyst · Piper Jaffray. Your line is open.

Great. Thanks for taking my questions.

Sandy Macrae

Management

It’s our pleasure.

Operator

Operator

Thank you. [Operator Instructions] Our next question comes from Jim Birchenough with Wells Fargo. Your line is open.

Unidentified Analyst

Analyst · Wells Fargo. Your line is open.

Hi, it’s Nichol for this Jim this afternoon. Just a couple of questions. For the MPS programs, is there any reason to hope that you would get better partitioning of enzyme into the CNS and what you have seen with current ERT? And I have a follow-up.

Sandy Macrae

Management

So, it’s a good question, it’s an important question, because it’s an important thing that patients and their families are asking about and we won’t be very clear and careful and the evidence that we talk about. And so, we have seen evidence in mice and Mike, do you want to talk about evidence?

Michael Holmes

Management

So, in our preclinical studies in the MPS I and MPS II, mouse models, we did see that in treating these mice, that we had seen very large amounts of enzyme being produced and being secreted in the blood, and that this did seem to provide some protection with regards to the degradation that you normally see in terms of neurocognitive effects in the mice. And we somewhat hypothesize that given that we are able to - in using the IV ERT approach in mice, because we are able to achieve a constant high level amount of enzyme that we were able to get some of this enzymes across the blood brain barrier and provide protection or at least the breakdown of GAGS that would prevent the neurocognitive degradation that you normally see in these mice. So, I think that’s what we saw in our preclinical studies, but as Sandy mentioned, we just want to be very careful in terms of – as we sort of extrapolate what we’ve seen in mice until what we might potentially see in our clinical studies.

Sandy Macrae

Management

And all of us in this industry have seen most mice results that have not been reflected in clinical studies. However, these are remarkably clear in the differentiation of the product provides. So it encourages us and the patients and families and this field.

Unidentified Analyst

Analyst · Wells Fargo. Your line is open.

Okay and can you – is it possible to determine what proportion of neuro protection, you are saying you are not ablating neurocognitive.

Sandy Macrae

Management

I think that would be, it’s tempting and we hope that we are able to provide that neurocognitive protection to the patients but the translatability of this is – I repeatedly tell people, this is cutting-edge clinical science where with the first time that any of these patients has sort of a constant supply of the enzyme until the translation between that the therapeutic effects will really be determined as the studies evolve.

Unidentified Analyst

Analyst · Wells Fargo. Your line is open.

Okay, and then, in terms of potential partnerships, T-cell-based therapy, can you give some guidance as to when one of those might be consummated?

Sandy Macrae

Management

Curt, do you wish to answer that?

Curt Herberts

Management

I think what I will say is that, we are very impressed with the data that that Mike has shown both in terms of the level of single knockout, double knockout rate at the 90% and especially with the level of targeted integrations greater than 90% in that context. No other parties in the entire area are really able to achieve this. And so, we are currently evaluating a number of options here.

Sandy Macrae

Management

We will of course press release on it when we – if we succeed in a relationship.

Unidentified Analyst

Analyst · Wells Fargo. Your line is open.

Okay, thank you very much.

Operator

Operator

Thank you. I am showing no further questions in queue at this time. I’d like to turn the call back over to Mr. Macrae for closing remarks.

Sandy Macrae

Management

Thank you. I would like to thank you all for your continued support of Sangamo and your interest in what we do and wish you a great afternoon and evening.

Operator

Operator

Thank you. Ladies and gentlemen, that does conclude today's conference. Thank you very much for y our participation. You may all disconnect. Have a wonderful day.