Earnings Labs

Sangamo Therapeutics, Inc. (SGMO)

Q4 2011 Earnings Call· Wed, Feb 8, 2012

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Transcript

Operator

Operator

Elizabeth Wolffe

Management

The agreement provides significant near-term funding in the form of an up front payment of $13 million, and funding for all of our internal and external research-related and preclinical program costs through the filing of an IND. We are also eligible to receive milestone payments of $8.5 million for each gene target that we take to IND, and regulatory clinical and commercial milestones that bring the total potential milestone payments to $213.5 million per gene target. Additionally, we retained very significant downstream value in the products we were developing under the collaboration and the form of loyalties that are an escalating tiered double-digit percentage of product sales. Finally, while aggressively pushing the Shire targets forward, we will continue to develop our own internal ZFP therapeutic programs to point to the significant value inflection, enabling us to continue to execute on our business model by establishing additional strategic partnerships in focus to these areas. So, with our collaboration with Shire underway, and our already strong cash position of $85 million at the end of 2011, we have a very solid financial base from which to execute on our development plans and our 2012 goals. Before going into more detail on our ZFP therapeutic programs and our plans for 2012, let me hand the call over to Ward for an update on our 4th quarter and full year 2011 financial results, as well as our financial guidance for 2012. Ward?

Edward Lanphier

Management

The agreement provides significant near-term funding in the form of an up front payment of $13 million, and funding for all of our internal and external research-related and preclinical program costs through the filing of an IND. We are also eligible to receive milestone payments of $8.5 million for each gene target that we take to IND, and regulatory clinical and commercial milestones that bring the total potential milestone payments to $213.5 million per gene target. Additionally, we retained very significant downstream value in the products we were developing under the collaboration and the form of loyalties that are an escalating tiered double-digit percentage of product sales. Finally, while aggressively pushing the Shire targets forward, we will continue to develop our own internal ZFP therapeutic programs to point to the significant value inflection, enabling us to continue to execute on our business model by establishing additional strategic partnerships in focus to these areas. So, with our collaboration with Shire underway, and our already strong cash position of $85 million at the end of 2011, we have a very solid financial base from which to execute on our development plans and our 2012 goals. Before going into more detail on our ZFP therapeutic programs and our plans for 2012, let me hand the call over to Ward for an update on our 4th quarter and full year 2011 financial results, as well as our financial guidance for 2012. Ward?

Ward Wollf

Management

Thank you, Edward, and good afternoon, everyone. As you know, after the close of the market today we released our financial results for the fourth quarter and full year ended December 31, 2011, and I am pleased to review the highlights of those results. Revenues in the fourth quarters of both 2011 and 2010 were $4.7 million. Fourth quarter 2011 revenues were comprised of revenue from Sangamo's collaboration agreements with Dow AgroSciences, and Sigma Aldrich, and agreements related to protein production, as well as approximately $1.5 million of revenue from research grants. As we mentioned in the press release, the increase in collaboration agreement revenues was due to increased sub-licensing and manufacturing revenue from DAS, as well as increased royalty revenue from Sigma. The decrease in research grant revenues was primarily due to the receipt of four qualifying therapeutic development program awards in December 2010, and decreased revenue from the Juvenile Diabetes Research Foundation to support qualified expenses, and curtain Sangamo's clinical development program for SB-509 due to the completion of Phase 2 clinical trial for Diabetic Neuropathy. These decreases were partially offset by increased revenues from other research grant awards. The total operating expenses for the fourth quarter of 2011 were $11.1 million compared to $13 million for the same period in 2010. Research and development expenses were $7.9 million in the 2011 quarter and $9.9 million for the prior year quarter. The decrease was primarily due to decreased expenses related to the completion of our phase 2, 2b clinical trial of SB-509. General and administrative expenses were $3.2 million in both the fourth quarter of 2011 and the same period in 2010. Non-cash stock based compensation expense was $2.1 million for the quarter with $1 million in research and development and $1.1 million in general and administrative. For…

Edward Lanphier

Management

Thank you, Ward. As you have heard, we begin 2012 as we have previously guided with approximately $85 million which relative to our burn rate is a very strong cash position. The upfront payment and ongoing research funding that we will receive as part of our strategic alliance with Shire, further strengthens this position and enables us to aggressively and expeditiously move our ongoing clinical and preclinical stage therapeutic programs to points of significant value inflection. On that point, I've asked Geoff to update you on our recent ZFP therapeutic progress; first to outline the rationale and design of our two new Phase 2 clinical studies in HIV and secondly to tell you a bit more about our preclinical pipeline in rare and monogenic diseases. Geoff?

Geoffrey Nichol

Management

Thanks, Edward. First let me begin by summarizing the overall rationale for our approach in HIV/AIDS. The HIV virus destroys the immune system, specifically CD4 T-cells. As the disease progresses into AIDS, the CD4 T-cell numbers decrease. However there is a small group of people known as elite controllers who are infected with the virus yet have undetectable HIV RNA in their blood and maintain normal CD4 T-cell counts without HAART. Many elite controllers have a natural mutation, the so-called delta-32 mutation in both copies of their gene encoding of protein called CCR5, which is a receptor used by HIV to infect T-cells. This mutation in the CCR5 protein means the receptor cannot be used by HIV to infect their CD4 T-cells. There is also a larger group, approximately 5% to 10% of the U.S. HIV infected population that carries the delta-32 mutation on only one of their CCR5 gene copies. These so-called heterozygotes are not completely resistant to HIV infection, but take much longer to progress to AIDS than individuals with two normal CCR5 genes. Additionally and beyond the naturally occurring mutation, is the example of the of the so-called Berlin patient who had both leukemia and HIV. In this case doctors in Germany took bone marrow cells from an unrelated donor who had the natural delta-32 mutation in both of the CCR5 genes and gave these cells to the HIV infected leukemia patient and more than four years later this person is both cancer free and free of HIV. Alziafen technology allows us to modify effectively knocking out the CCR5 gene with our zinc finger nucleases. By doing this we hope to create T-cells that will be both protected from HIV infection and capable of mounting an effective immune response to HIV. If successful, this approach would enable…

Edward Lanphier

Management

Thanks, Geoff, that was a great summary. As you have heard, we have a very busy year ahead with continued data from our lead clinical program and our rich pipeline of preclinical programs, with both Shire and our internal targets. In addition, our partnerships with non therapeutic applications of our technology continue to thrive. Sigma-Aldrich continues to do a great job of diligently and creatively developing our ZFN assets in its composer custom reagents kits and custom cell lines, as well as their transgenic animal program, which represents a huge growth area both scientifically as well as commercially. They are an imaginative partner, and recently announced the acquisition of BioReliance], a leader for safety testing of biological drugs. As Sigma CEO explained at the JP Morgan conference, they see an opportunity to enhance and improve the services provided by BioReliance using ZFN technology to produce targeted cell lines, as well as animal models. Our collaboration with Dow AgroSciences also continues to go well. Dow is employing our ZFN technology in its core focus crops, and marketing the technology as exact precision technology to companies working in other crops. These collaborations have allowed us to access capital in a way that is very different than most biotechnology companies. Over the past five years our Sigma and Dow collaborations have brought in over $83 million, and importantly, we have retained significant downstream value in the commercialization of these assets, including, in the case of Sigma, a 10.5% royalty on sales of ZFNs and ZFN-based products, including transgenic animals. As such, in 2012, we estimate revenues in the range of $14 million to $18 million, largely from these partnerships and our new agreement with Shire. On the financial side, we expect to end 2012 with cash and cash equivalents of at least $75…

Operator

Operator

Thank you. (Operator Instructions) Our first question comes from Joseph Schwartz with Leerink Swann. Your line is opened.

Joseph Schwartz - Leerink Swann

Analyst · Leerink Swann. Your line is opened

Thanks for taking the question. I was wondering what is the timeline for you to submit an IND in hemophilia and does your 2012 year end cash guide of $75 million include any new IND filings under the Shire collaboration?

Edward Lanphier

Management

Good questions, Joe. So as we've said in the script, our plan is to give much more visibility, both in terms of priority meaning, which targets we'll move first to the clinic, and then timing around those IND filings in the second half of this year. The reason being is that we are in critical experiments associated with several of these targets. Those experiments, those data, quite frankly, drive both the prioritization as well as the timing to IND. To the part of your second question with financial guidance, that Ward gave, in term of revenue assumptions, do not include any milestones from the new partnership with Shire. They only include, in terms of Shire, the amortization of the up-front payment, as well as expected research funding for both internal and external cost.

Joseph Schwartz - Leerink Swann

Analyst · Leerink Swann. Your line is opened

OK. Great. Thanks. To what extent does the Shire agreement provide a blueprint for future collaborations given these rare inherited disorders can often be developed more efficiently, and you do have some good clinical development infrastructure? Would you contemplate taking any programs to a further stage yourself or will you be looking to out-license things as early as you did with the Shire deal?

Edward Lanphier

Management

Good question, Joe. The answer is both. We have a platform that can be applied to dozens and dozens, if not, quite frankly, hundreds of potential monogenic disease targets. We do expect to continue, as I mentioned, the visibility of the Shire deal and the data out of ASH have really increased the visibility and awareness of the platform in Pharma. We do expect to do deals at the preclinical level. With that said, we are moving several programs forward on our own account and are going to plan to move those forward into the clinic. With that said, if the right collaboration comes along, such as hemophilia with Shire, we would move forward with partners around those as well.

Joseph Schwartz - Leerink Swann

Analyst · Leerink Swann. Your line is opened

OK. Great. Thanks and I'll get back in the queue.

Operator

Operator

Thank you. Our next question comes from Charles Duncan of JMP Securities. Your line is opened.

Charles Duncan - JMP Securities

Analyst · JMP Securities. Your line is opened

Hi, guys. Congratulations on the recently signed therapeutic collaboration and thanks for taking my questions. Edward, I had a question along the lines of the HIV program. I'm wondering on the CCR5 heterozygotes, have you been able to follow up with your patient that was in your Phase 1 trial? Do you have any sense of how that patient is doing in terms of viral load or T-cell count?

Edward Lanphier

Management

Well, I will give it a short answer. Then, Geoff or Dale, you can comment further. That patient went back on his heart therapy after the end of the TI. I don't expect that there was going to be a viral-load impact based upon 728-T. In terms of long-term follow-up, I'm not personally aware. Dale or Geoff?

Dale Ando

Analyst · JMP Securities. Your line is opened

He's on heart and long-term follow-up.

Edward Lanphier

Management

Just what I said, Charles, he's on heart and in long-term follow-up.

Charles Duncan - JMP Securities

Analyst · JMP Securities. Your line is opened

OK. You also mentioned that the cohort you're working on EN-902 is progressing well and have your first subject treated. I believe Geoff also mentioned that you would guide to data later on. Is there any sense as to how many patients you'd like to have in that data? Could we see data yet this year? Are you beyond just one subject having been treated so far in that trial?

Edward Lanphier

Management

Again, as you know, we don't typically give accrual updates or guidance. What Geoff said is we will update later in the year in terms of timing related to presentation of clinical data. Geoff, in terms of total numbers on the Cohort 5, I think we said up to 20. Is that where we still are?

Geoffrey Nichol

Management

Yes. We are going up to 20, Charles. The thinking, and I have talked about this before, is that we are looking for a clear-cut response in individual patients along the lines of the patient who became aviremic in the collaborative study at Penn. This is like a response in a cancer patient. Typically, if you want to know if you've got an active agent, you're looking for a reasonable proportion of responses in usually a relatively small number of patients. We're talking a proportion of those 20 patients with good engraftment being the number that we need to get to. We'll guide more once we are approaching those numbers and can give you some greater clarity.

Edward Lanphier

Management

One additional piece of color on that Charles, is when we started this trial or were contemplating this trial, it was unclear how difficult it would be to actually accrue subjects. It turns out there are a lot of HIV infected people who really now their CCR-5 status. Accruals have actually gone pretty efficiently in this study.

Charles Duncan - JMP Securities

Analyst · JMP Securities. Your line is opened

Edward, that's the actual part that I was wondering about. It sounds like it's easy to identify these patients and enroll them but it's really you're looking for some responses, so that makes sense. If you could help us understand a little bit. I know it's early but you've pointed to potential other collaborations. Is it possible that there could be a collaboration for an HIV product candidate or do you think that you could identify patient population that you could serve through, at least take further into the clinic or even get all the way over the goal line with an internal commercial infrastructure?

Edward Lanphier

Management

I think both are true. I think data will drive both of those potential outcomes. Data will drive the continued interest in a possible collaboration and data will also drive any decision that we might make in terms of moving things farther and farther forward ourselves. I will say that we have put ourselves from a cash position and from an operating position in a position to make those kinds of decisions. It's really going to be data driven at this point.

Charles Duncan - JMP Securities

Analyst · JMP Securities. Your line is opened

One final question with regard to the Shire collaboration, again, that was a nice deal. I'm wondering if you could provide a little bit more color on the thought behind the six-year duration? Going back to the last question that Joe asked, I know that you're not giving guidance on how much time to get to an I&D but could you anticipate getting all four targets through within that six year period should the early data hold up?

Edward Lanphier

Management

Yes. Again, it's a bit of a same sort of question. I'm not going to give specific time frames but the six years was originally agreed upon as more than sufficient time to move all of these targets through our responsibilities, which through I&D filing. That should in a backdoor way answer your second question.

Charles Duncan - JMP Securities

Analyst · JMP Securities. Your line is opened

Operator

Operator

Ted Tenthoff - Piper Jaffray

Analyst

Ed Lanphier

Analyst

Operator

Operator

Liana Moussatos - Wedbush Securities

Analyst

Ed Lanphier

Analyst

Liana Moussatos - Wedbush Securities

Analyst

Ward Wolffe

Analyst

Liana Moussatos - Wedbush Securities

Analyst

Edward Lanphier

Management

Liana Moussatos - Wedbush Securities

Analyst

Operator

Operator

Alastair Mackay - GARP Research

Analyst

Edward Lanphier

Management

Philip Gregory

Analyst

Edward Lanphier

Management

I'd add, Alastair, Philip is being modest. If you look at the publications in the space over the last two years, call it 10 or 12, the vast majority of those are either our work or work that we've provided the TALENs to collaborators. So this is a space that we really are the current leaders in. I won't speak deeply to the IP space, but you can rest assured that it's an area that we have filed extensively in. With that said, it's very, very early days. I don't there's going to be any immediate effects on the research reagent space and certainly no even no mid- to long-term effects on the therapeutic space.

Alastair Mackay - GARP Research

Analyst

Great. Thanks. If I could switch gears and ask about, Edward for you to speculate a little bit about let's say call it per patient pricing. If we look at monogenic diseases, when you're just doing cocktail napkin arithmetic, how do you think of what the cost of a therapy for an individual patient with a monogenic disease might be? Maybe instead of giving numbers if that's not good, we could talk about some of the other Pharmas and the various strategies they've taken.

Edward Lanphier

Management

Again, this is a long and interesting discussion. What I would parse here is cost of goods versus value to the patient. I think what we're focusing on are outcomes, again, potentially genetic cures, we're focusing on outcomes where there is enormous value not only to the quality of life of the patient, but also in terms of the savings to the healthcare system. Those pencil out on a napkin very easily.

Operator

Operator

Thank you. At this time I'm not showing any further questions. I would like to turn the call back to Edward Lanphier for any further remarks.

Edward Lanphier

Management

We'd like to thank you for joining us and we look forward to speaking with you again when we release our first quarter financial information. We will be available later today if there are any follow-up questions. Thank you very much.

Operator

Operator

Ladies and gentlemen, thank you for participating in today's conference. This concludes today's program. You may all disconnect. Everyone have a great day.