Sangamo Biosciences' CEO Discusses Q2 2012 Results - Earnings Call Transcript

| About: Sangamo Therapeutics, (SGMO)

Sangamo Biosciences, Inc. (NASDAQ:SGMO)

Q2 2012 Earnings Call

July 25, 2012 5:00 pm ET


Elizabeth J. Wolffe, Ph.D. – Senior Director, Corporate Communications

Edward Lanphier – President and Chief Executive Officer

H. Ward Wolff – Executive Vice President and Chief Financial Officer

Philip Gregory – Vice President, Research and Chief Scientific Officer

Geoffrey M. Nichol – Executive Vice President, Research and Development


Liana Moussatos – Wedbush Securities Inc.

Charles Duncan – JMP Securities

Ted Tenthoff – Piper Jaffray


Good afternoon, and welcome to Sangamo BioSciences Teleconference to Discuss Second Quarter 2012 Financial Results. This call is being recorded.

I will now pass you over to the coordinator of this event, Dr. Elizabeth Wolffe, Senior Director of Corporate Communications.

Elizabeth J. Wolffe

Thank you, Kate. Good afternoon, and thank you for joining Sangamo's management team on our conference call to discuss the company's second quarter 2012 financial results. Also present during this call are several members of Sangamo senior management, including Edward Lanphier, President and Chief Executive Officer; Ward Wolff, Executive Vice President and Chief Financial Officer; and Geoff Nichol, Executive Vice President, Research and Development; Philip Gregory, Vice President of Research and Chief Scientific Officer; and Dale Ando, Vice President of Development and Chief Medical Officer.

Following this introduction, Edward will highlight recent activities and the significant events from the past quarter. Ward will then briefly review second quarter financial results, as well as our financial guidance for 2012. Philip and Geoff will provide an update on our ZFP therapeutic program, and finally, Edward will update you on our goals for the rest of 2012. Following that, we will open up the call for questions.

As we begin, I'd 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 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 that our comments from today are still valid. We alert you to be aware of risks that are detailed in documents that the company files with the Securities and Exchange Commission, specifically, our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. 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.

Now, I would like to turn the call over Edward.

Edward Lanphier

Thank you, Liz, and thank you all for joining us on our conference call to discuss our second quarter results for 2012, as well as recent events and our plans for the rest of the year. This has been a busy first half of the year during which we have made significant progress in advancing our clinical and preclinical programs, as well as our business model, and we’re pleased we have an opportunity to provide you with more details and the expected timing for clinical milestones on this call.

First, our preclinical pipeline, which includes programs that we are developing for our own accounts, as well as with our partner Shire are progressing very well. To remind you in early February, we entered into a strategic alliance with Shire and to develop our ZFP Therapeutics has a potential of genetic cure for hemophilia and other monogenic diseases.

Upon executing the agreement, we announced the selection of hemophilia targets Factors VII, VIII, IX and X. Shire also received an option to name three additional targets. I'm very pleased to announce today that Shire has selected a fifth target and has committed to support the development of the ZFP Therapeutics for Huntington’s disease. A program that we had previously initiated with funding from the CHDI Foundation, a research foundation focused on developing a cure for Huntington’s disease. I have asked Philip to provide you with some background on the disease and more details on why we believe our technology can generate the unique approach for the treatment of Huntington’s later on the call.

Because our ZFP technology works very specifically and efficiently at the DNA level, it enables us to provide unique therapeutic solutions to diseases where the cause has been traced to a defect in a single gene so-called monogenic diseases. Huntington’s is a well studied example as you will hear later.

However, there are many monogenic diseases. We are actively working on several other single gene based disorders some of which were highlighted in presentations at the Annual American Society for Gene & Cell Therapy Meeting in June. However, up to this point, beyond presentations of data at scientific meetings and publications and peer-reviewed journals. We have not provided a great deal of visibility on these programs that will change at the end of this year.

We planned to host an Analyst Briefing on December 6, at which time, we will discuss our preclinical monogenic diseases programs, our development plans, and the timing for IND filings, and associated financial projections. The briefing will be brought, it will be webcast and details regarding the exact timing of the event will follow later this year.

Moving on to our lead clinical program, SB-728, which we are developing as a potential functional cure for HIV, I’m pleased to report that our two Phase II studies are also progressing on plan. As most of you know based upon encouraging Phase I data, we announced the initiation of two Phase II trials during the first quarter of this year. At that time we said that we would update you on the expected timing of clinical data once we have had an opportunity to asses the rate of enrollment.

I’ll ask Geoff to provide you with more details on the trials and anticipated timing of data from those studies later on this call. However, before I hand the call over to my research and development colleagues to provide more details on our ZFP Therapeutic programs, let me ask Ward to summarize our second quarter 2012 financial results, as well as our financial guidance for 2012. Ward?

H. Ward Wolff

Thank you, Edward, and good afternoon, everyone. As you know, after the close of the market today, we released our financial results for the second quarter ended June 30, 2012, and I’m pleased to review the highlights of those results.

Revenues for the second quarter of 2012 were $4.6 million, compared to $1.5 million for the 2011 quarter. Second quarter 2012 revenues were comprised of revenues from the company’s new collaboration and license agreement with Shire to develop ZFP Therapeutics for hemophilia and other monogenic diseases. Existing collaboration agreements with Dow AgroSciences and Sigma-Aldrich Corporation, including a $1 million commercial milestone from Sigma, enabling technology agreements, as well as approximately $0.8 million of revenue from research grants. As we mentioned in today’s press release, the increase in collaboration agreement revenues was attributable to the company’s agreement with Shire.

As we explained in our first quarter call, we are amortizing the $13 million upfront license fee over the initial six-year research term resulting in a $0.5 million recognized as related revenue in the second quarter. In addition, collaboration agreement revenue included $1.5 million in revenue related to the reimbursement of internal and external program related costs from Shire during the quarter.

Total operating expenses for the second quarter of 2012 were $10.3 million, compared to $11.8 million for the same period in 2011. Research and development expenses were $7.6 million for the 2012 quarter and $8.1 million for the prior year quarter. General and administrative expenses were $2.7 million for the second quarter of 2012, compared to $3.7 million for the 2011 quarter.

Non-cash stock-based compensation expense was $1.2 million for the quarter equally split between research and development and general and administrative. For the second quarter of 2012, we reported the consolidated net loss of $5.7 million, or a $0.11 per share, compared to a net loss of $10.3 million, or $0.20 per share for the second quarter of 2011.

Turning to the balance sheet, we ended the second quarter of 2012 with approximately $81 million in cash, cash equivalents and marketable securities. Net cash used in operating activities was $6 million for the quarter and $3.1 million for the year to-date.

Our financial guidance for the year remains unchanged from the first quarter call in May. We expected to have cash and investment balances of at least $75 million at the end of 2012, inclusive of the Shire agreement, but exclusive of any new funding from a partnership or other sources. We also expect 2012 operating expenses to be in the range of $43 million to $47 million and revenue to be in the $14 million to $18 million range, again inclusive of the research funding from Shire for internal and external research program related costs.

In summary, we are pleased to report that the second quarter results are tracking to our 2012 operating plan. Our therapeutic collaboration with Shire has enhanced our leverage in managing our finances and balance sheet and complements the business model established for our existing partnerships outside the human therapeutics.

We are in excellent financial position to complete our ongoing clinical trial and continue to advance our preclinical pipeline and we look forward to keeping you updated on our progress. Thank you and I will now turn the call back over to Edward.

Edward Lanphier

Thank you, Ward. Our business model for developing our ZFP technology platform, has enabled us to partner not only therapeutic programs as we have recently demonstrated with Shire, but also non-therapeutic applications of our technology.

These non-therapeutic partnerships with Sigma-Aldrich and research reagents and Dow AgroSciences in Plant Agriculture have brought significant resources into the company over $85 million to-date. We are very pleased to announce today the achievement of our first commercial milestone, which as Ward described was a result of sales of ZFP products for research applications by Sigma.

In addition to the revenues that we have received as a result of this agreement, Sigma’s broad distribution of our technology into the hands of scientists all over the world as a result of their impressive and imaginative product development and marketing efforts have been very useful to us, as it is significantly broaden the appreciation of the power of our ZFP technology, particularly in the pharmaceutical industry.

The amortized upfront payment and ongoing research funding from our strategic alliance with Shire as well as the ongoing revenues from our collaborations with Dow and Sigma, enable us to aggressively and expeditiously move our ongoing clinical and preclinical stage therapeutic programs to points of significant value inflection, while maintaining a very modest run rate. As you have heard, we have a solid cash position and remain on track to meet our guidance of ending 2012 with at least $75 million in cash.

So, with the very strong financial underpinnings [bond] to preclinical and clinical development programs, I would ask Phil to update you on the Shire selective Huntington’s disease program and Geoff to update you on our ongoing HIV clinical trials, expected timings of those data and our plans to provide more details later this year on our preclinical pipeline, particularly our work in rare and monogenic diseases, Philip?

Philip Gregory

Thanks Edward. Our ZFP technology functions at the DNA level, enabling both control of gene expression, the ability to turn gene expression on or off and the direct editing of gene sequences, which gives us the ability to correct a mistake within a gene sequence add or delete a gene.

The ZFPs, the Sangamo engineers are highly specific, designed to affect the activity or the sequence of an individual gene within the genome. These capabilities mean that our ZFP technology provides an ideal platform to create novel therapeutic solutions to address monogenic diseases. Development to ZFP therapeutics for monogenic disease is the major focus for us both in our internal programs and our collaboration with Shire

Our aim is to develop disease modifying therapies that could provide a genetic cure. As Edward announced, Shire has selected Huntington’s disease as the fifth target in our collaboration. By way of background, Huntington’s disease is inherited, progressive neurologic disease for which there is no treatment or cure. It is a devastating disease. It has a very high prevalence for inherited disorder, affecting approximately 30,000 people in the U.S. according to the National Institute of Neurological Disorders and Stroke or NINDS. A similar prevalence of one in 10,000 is the least to occur in the major European countries.

The NINDS estimates that at least 150,000 patients. Beyond that stage, 30,000 U.S. symptomatic HD patients have a 50% risk of developing the disease. The disease is generally fatal within 10 to 20 years of the initial onset of symptoms. The symptoms become noticeable between the ages of 35 and 44 years, but it can start earlier. The most obviously early symptoms are jerky, random, and uncontrollable movements was progressed virginity and arriving movements called chorea. In fact you may have heard of the disease as Huntington’s chorea. Gradually, as the disease progresses any action that requires muscle control such as speaking or eating is adversely affected.

In addition, there is a progressive impairment of covered disability and memory. In 1993, scientists demonstrated that the disease is caused by a specific type of mutation in a single gene called Huntington, which incurs the protein of the same name.

Mutation is very well characterized and is specifically an expansion of a repeated stretch of DNA sequence within the gene. A Huntington gene normally have somewhere between 18 and 25 copies of the so-called CAG repeat, but individuals with Huntington’s disease have many more generally greater than 40 CAG repeats. The greater the number of repeats, the earlier the disease manifest itself and faster the progression of symptoms. However, several groups have shown a variety of mouse models of the disease that reducing the levels of mutant Huntington can prevent the unset and in some cases reverse the symptoms of Huntington’s disease. So just to recap, Huntington's disease is a classic example of a monogenic disease, but is absolutely correlated with a DNA mutation that takes the same form in all affected individuals.

We know from animal studies that affecting the levels of expression at the mutant gene and thus the protein is in [encored] can have a therapeutic effect. All of this gives us an ideal target for our ZFP technology. To that end we have been developing approaches to specifically and selectively repress the expression of the mutant or disease causing gene. The potential of this selective approach is something that CHDI also recognized and they have been funding our early research program in Huntington's for the past year or so. The success of this research to date is what caused Shire's attention and prompted them to select Huntington's as their next target in our collaboration. I look forward to updating you on our progress in this and other programs of our Analyst briefing in December, we plan to present a comprehensive overview of our entire preclinical pipeline. And with that let me hand you back to Edward.

Edward Lanphier

Thank you, Philip. We are delighted that Shire has chosen Huntington's disease as the fifth of the seven targets of our collaboration, as with the hemophilia programs. Shire is funding all of our internal and external costs associated with this program through the filing and approval of an Investigational New Drug Application or IND or the European equivalent of CTA.

Shire will then be responsible for clinical and commercial development of the product. As Philip mentioned, we are exploring several approaches to this indication and look forward to updating you on our progress later this year. Now let me turn to our ZFN Therapeutic programs in HIV. As many of you will no doubt heard or seen, the international AIDS conference is going on in Washington, D.C. this week. One of major areas of discussion in the sessions and in the corridors has been moving beyond the current chronic treatment of the infection to a cure for the disease.

Our ZFN CCR5 gene disruption approach in both CD4+ T-cells and in CD34+ from hematopoietic stem cells have been front-end center in these conversations and in the media as one of the leading strategies for the generation of a functional cure for HIV. To provide you with more color on our HIV programs and specifically to outline the progress on our Phase II trials and the timing for data from these studies let me turn the call over to Geoff. Geoff?

Geoffrey M. Nichol

Thanks, Edward. As you all know our zinc finger nucleases technology allows us to specifically modify and effectively knock out the CCR5 gene, which [encodes] the major co-receptor for HIV entry into cells. By doing this we aim to create T-cells that will be both protected from HIV infection and capable of mounting an effective immune response to HIV, similar to a naturally occurring situation in individuals who carry the CCR5 delta-32 mutation on both copies of their CCR5 gene. If successful, the CFN approach would enable a functional cure in these patients, such that they could control their HIV without taking antiviral medication.

Our therapeutic product, ZFN Modified Autologous CD4 T-cells is called SB-728-T. Thus far results from Phase I studies of this product have been very encouraging. The data demonstrated this. Statistically significant relationship between the level of engraftment of ZFN-modified cells in which both copies of the CCR5 gene disrupted, so-called biallelic modification, and the level of virus in the blood of HIV infected subjects during a treatment interruption from antiretroviral medication.

This observation is consistent with the hypothesis under which we began this program and with this clear early signal, at the beginning of the year, we moved quickly into two Phase II studies. The first trial, Cohort 5 SB-728-902 exclusively enrolled HIV infected subject that already carried a natural mutation, CCR5 delta-32 on one of their two copies of the CCR5 genes. These individuals, heterozygotes, the CCR5 delta-32 comprise approximately 5% to 10% of the population of HIV infected individuals in the U.S.

Competitive individuals with neither earlier modified as they already have one copy of the CCR5 gene disrupted by the natural mutation, treatment with our ZFNs results in roughly a doubling of the numbers of biallelically modified T-cells. This Phase II study follows up on an observation made in an earlier Phase I trial in which we measured a reduction in viral load to undetectable levels in a 728-T treated CCR5 delta-32 heterozygous individual during a treatment interruption or TI of the anti-viral medication. That individual although with undetectable viral load at the end of the TI is required as part of the protocol to go back on to heart.

Our new Phase II trial is structured so that a longer period of TI can be monitored, up to 20 HIV-infected CCR5 delta-32 heterozygous who are on HAART maybe enrolled in the trial. These subject to treated with 728-T and eight weeks later undergo a treatment interruption where they discontinue their HAART for a period of 16 weeks. If their viral loads decrease to undetectable levels they may remain of their HAART for as long as this affect assists enabling us to measure the durability of such a response.

When we began this study, we are uncertain as to the availability of subjects for this trial. As I’ve said before that represent only 5% to 10% of the HIV infected population in the U.S. However to date the challenge of enrollment in this study has been reduced as many HIV infected individuals are aware of the CCR5 gene status.

Our second trial SB-728-1101, which is applicable to the other 90% to 95% of the U.S. HIV infected population, was initiated later in the first quarter. This Phase I/II study employ the lymphopenic preconditioning regimen with the drug called Cytoxan intended to expand the numbers of biallelically modified cells in subjects post infusion. This preconditioning regimen, which temporarily reduces the number of lymphocytes in the body results in an increased in the levels of T-cell growth factors stimulating the remaining immune cells to rapidly expand and divide, so if we infuse 728-T immediately after preconditioning, we expect a significant increase in the numbers engrafted biallelically modified cells, potentially by orders of magnitude.

In this study, just prior to infusion of 728-T, we are pre-treating each subject with a dose of Cytoxan. Again, there is a 16 week TI built into the study, which begins six weeks after 728-T treatment and can be extended if subject obtained an undetectable viral load. This strategy has been previously used in individuals with HIV in a variety of settings.

However, as we are combining this with 728-T treatment, I need to explore the dose required for optimum pre-conditioning. We are carrying out a dose escalation phase of three dose cohorts each with three subjects per cohort. Dose escalation studies are frequently designed to include observation periods both between subjects enrolled into the trial and initiation of a new dose Cohort. And so, they generally take longer than a non-dose escalation trial of a similar size. In this study, we are required to wait for two weeks after the treatment of the subject before we treat the next subject in that Cohort. After the treatment of the final subject in the Cohort, we need to observe and collect data for four weeks and then present our findings to the Data Safety Monitoring Board before advancing to the next dose Cohort.

Thus while enrollment is progressing well in both of our studies, these trials would take time. We therefore expect to present preliminary data from all of the subjects in the first half of 2013 and a complete data set in the second half of 2013. As usual, we expect to present the data at a major medical meeting.

Consistent with that approach, I’m pleased to announce today that we have been notified the two abstracts have been accepted for presentation at ICAAC in September. These presentations will focus on important immunological analysis and methods for evaluating the [profile] reservoir from our earlier Phase I trials.

Moving onto preclinical programs, ZFN platform provides a range of powerful gene regulation and gene modification outcomes, including gene disruption, addition and correction, and importantly, can be designed to target any DNA sequence with singular specificity. Furthermore, in our preclinical hemophilia work, we have demonstrated that we can deliver ZFP Therapeutics systemically. This direct in vivo approach significantly expands our ZFP Therapeutic applications and the diseases for which our technology can provide genetic cures.

In monogenic diseases we can use ZFN therapeutically to permanently correct that error as in the case of hemophilia programs and ZFP transcription factors to control expression of a gene as in the case of Huntington’s disease program. These are both programs that are part of collaboration with Shire. Independently we are also applying this technology and other monogenic diseases including hemoglobinopathies such as sickle cell anemia, and beta-thalassemia, lysosomal storage diseases and gene-based immune disorders.

Finally, as you may recall, I joined Sangamo just about a year-ago, and one of my initial task was to work with Dale Ando, Philip Gregory and our business team to assess our portfolio programs and prioritize them from both the technical feasibility and business (inaudible). We look forward to providing you with the conclusions of our analysis at our analyst meeting in December.

And with that, let me turn the call back to you Edward.

Edward Lanphier

Thanks, Geoff. As you have heard, our clinical trials in HIV are progressing very well. As such, we expect to present interim and complete data next year. In addition, we have a rich pipeline of preclinical program, which include the internal targets, as well as those are part of our collaboration with Shire.

We expect to have much more to say about of preclinical pipelines, specifically regarding preclinical data, milestones and expected timing to IND filings at our Analyst briefing at the end of the year. Our partnerships in the non-therapeutic applications of our technology also continue to thrive. As can we seen by achievement of this past quarter of our first commercial milestone with Sigma.

On the financial side, we’re on track to end 2012 with cash and cash equivalents of at least $75 million, which is more than sufficient capital to allow us to achieve our goals of aggressively advancing our clinical and preclinical therapeutic programs. This cash projection does not however include any new agreements or partnerships that we may develop beyond the recently announced agreement with Shire.

And on that point, our business model is to establish additional partnerships to help drive our programs forward through the development process and into the market. Our collaboration with Shire, which is a great example of this strategy is off to a great start with funding for the hemophilia and Huntington’s programs, we have moved into high gear to advance both our collaborative programs, as well as a number of programs that Sangamo is pursuing independently.

In addition, the knowledge and experience that we gain as we advance ZFP Therapeutic applications for hemophilia and Huntington's disease can and will be applied to the development of numerous ZFP Therapeutics in other disease areas.

So, as you can see it's an exciting time at Sangamo. We have a great deal on our plate, we have the balance sheet to support our aggressive objectives and perhaps most importantly we are making significant progress towards our goal of establishing ZFP Therapeutics as a new and highly differentiated class of human pharmaceuticals that can provide the potential for genetic cures for many diseases.

I sincerely look forward to keeping you informed of our progress in the next few months we will be presenting at the Wedbush Securities Life Sciences Management Access Conference in New York on August 14 and the Stifel Nicolaus Healthcare Conference in Boston on September 6. And finally, as Geoff mentioned, we will be presenting additional SB-728-T data at ICAAC in early September.

This completes our prepared comments. I would now like to open the call for your questions.

Question-and-Answer Session


Thank you. (Operator Instructions) Our first question comes from the line of Liana Moussatos with Wedbush Securities. Your line is open.

Liana Moussatos – Wedbush Securities Inc.

Thanks for taking my question. and can you repeat the data that’s going to be released in the first half of next year, and also you mentioned something about reservoir measurements Phase I data at ICAAC. Can you go through that again please?

Edward Lanphier

Certainly let me start and Geoff and or Dale can add to it. I think what, we guided to on this call Liana as it relates to the Phase II trials is that we would have interim data or expect that interim data presentations in the first half of next year and a complete data set in the second half of next year. In terms of data that will be presented at ICAAC, I think we said that that would be data from our Phase I trials and as it relates to the specific topics that Geoff I will turn that over to you.

Geoffrey M. Nichol

Yeah I mean Liana, what we will be presenting will be in two areas. One is data looking in a deeper way into the immunological status of outpatients from the Phase I program and the other is more of a methodological look at using new and very refined techniques for evaluating the status of the proviral reservoir in these patients.

Liana Moussatos – Wedbush Securities Inc.

Thank you very much.

Geoffrey M. Nichol

Sure. Thanks, Liana.


Our next question comes from the line of Charles Duncan with JMP Securities. Your line is open.

Charles Duncan – JMP Securities

Hi, guys thanks for taking my question.

Edward Lanphier

Hi, Charlie.

Charles Duncan – JMP Securities

And congrats on the good progress. Edward thanks, heterozygous study Geoff mentioned that going into you were a little bit concerned about, whether or not patients knew their standards. Yes, he said that the challenge has been pretty much reduced. Can you provide a little bit more color on that? And second, fairly he also outlined nicely that kind of rate limiting steps and the second study to enrollment and trial completion. I am wondering if you could provide that for that state for the heterozygous study?

Edward Lanphier

Yeah. So, Charles I’ll go a little bit on the first piece, and Geoff and Dale again can add or subtract to my comments. And then, I think I missed the second part. So maybe we can come back to that, but in terms of the accrual processes, I think I have mentioned several times, and when we started this study, given that the estimate of delta-32 HIV-infected subjects in the U.S. is in the 5% to 10% range, it was uncertain how difficult or not this trial would be to accrue and one of the observations that we’ve learned as we initiate this study was a, sort of surprising number of HIV subjects who did know their CCR5 status, and because of that we did have people showing up at the site saying, hey, I’m a [homo] delta-32.

So it ended up being, at least today, a more efficient process than maybe a worst-case scenario might have anticipated. Geoff or Dale any other thoughts about that part of the question?

Geoffrey M. Nichol

No that’s pretty much itself, I mean we thought, we could get seriously enough with very, very few patients by the time we looked to their entry criteria and dealt with the CCR5 heterozygote issue requiring us to screen hundreds or thousands of patients in order to get the patients in, and that has not been set back. Nevertheless, we are making good progress with recruitment on both of the studies, you asked about the second study and that is very much just a timetable issue, we just are recruiting well, but we simply have to wait between patients and between cohorts. So it sort of it steps forward in a measured fashion.

Charles Duncan – JMP Securities

Yeah, that part I understand. I’m sorry for miscommunicating. What I was really asking is if you could outline for the actual conduct of the heterozygous study, if there are any rate-limiting steps to the conduct of that as you did nicely for the lymphodepletion study? And then with regard to that heterozygous study, the knowledge of whether or not patient is dealt as a heterozygous. I’m wondering if that has changed your perspective on the percentage of those patients in the population and if that selects for certain patients in terms of social economic status or anything?

Edward Lanphier

On the first question Charles, no, there are no analogous rate-limiting steps in the heterozygous study, (inaudible) dose escalation timeframes associated with the, as I talk some studies. So I know, there are no timing or gating items like that in terms of learning’s about the demographics or so on delta-32 subjects is there anything that Dale or Geoff you comment on that.

Geoffrey M. Nichol

No, Charles again, I mean we are dealing with relatively few site sale, obviously have lot catchments in really quite – in their own geographical areas, and really there is nothing that we can really derive from that data that would speak to the broader epidemiological question you ask?

Charles Duncan – JMP Securities

That’s good news. I’ll back in the queue. Thanks for the added color.

Edward Lanphier

Yep. Thanks, Charles.


(Operator Instructions) Our next question comes from the line of Ted Tenthoff of Piper Jaffray. Sir your line is open.

Ted Tenthoff – Piper Jaffray

Excellent, thank you very much for taking the question. And I appreciate very much the detail on the revenue side as we’re starting to see progress from Sigma and the details on the Shire deal. One quick question. I apologize if this is a bit dated, but are you still enrolling Cohort 5 from the Phase I study and will we get an update on that data or is the primary focus on the Phase II studies now?

Edward Lanphier

So we initially – I’ll start here. We had four cohorts in that study. The Cohort 5 of that trial, that 902 trail is this delta-32.

Ted Tenthoff – Piper Jaffray

Phase II.

Edward Lanphier

Yeah, is the delta-32 trial.

Ted Tenthoff – Piper Jaffray

Okay. So that’s what it’s become. Okay, excellent. That’s really helpful. And, so that’s the data that we will get interim results from both of the Phase II and the first half and full data by year end next year is that correct?

Edward Lanphier

That’s absolutely correct.

Ted Tenthoff – Piper Jaffray

Excellent. Thank you very much.

Edward Lanphier

Thanks, Ted.


Thank you. And we have a follow-up question from the line of Charles Duncan with JMP Securities. Sir your line is open.

Charles Duncan – JMP Securities

Hi guys, thanks for taking the follow-up. Congrats on the Shire program visibility with regard to Huntington’s disease, wondering if you have any additional perspective on when we might see the first IND in that program?

Edward Lanphier

Well, thanks for the question Charles and it’s a good one. Try and repeat what we said on the call. A lot of activity right now both in terms of the hemophilia target, the Huntington’s target all under the Shire collaboration, also a lot of activity in the monogenic disease space and Geoff listed several areas that we are pursuing a sickle cell and beta-thalassemia, lysosomal storage diseases and others, it’s for our own account. And our plan is to roll out in a more comprehensive way and instead of just – an individual target or individual program, we roll out in a more comprehensive way. The data underpinning or platform approach to monogenic diseases, the timing for IND filings both for the Shire targets, as well as our own, and also and I think very importantly the financial overview or guidance over the next several years under which we’ll be able to execute on that plan. And our goal is to do that in a comprehensive way at the end of the calendar year and specifically on December 6 at an Analyst Briefing.

Charles Duncan – JMP Securities

It sounds good. Thanks, Ed.

Edward Lanphier

Sure. Thank you.


Thank you. I'm not showing any further questions at this time. I like to turn the call back over to management for closing remarks.

Edward Lanphier

Great. We’d like to thank you for joining us and we look forward to speaking with you again when we release our third quarter financial information. We will be available later today if you have any follow-up questions. Thanks very much.


Ladies and gentlemen, thank you for participating in today’s conference. This does conclude the program and you may all disconnect. Everyone have a great day.

Copyright policy: All transcripts on this site are the copyright of Seeking Alpha. However, we view them as an important resource for bloggers and journalists, and are excited to contribute to the democratization of financial information on the Internet. (Until now investors have had to pay thousands of dollars in subscription fees for transcripts.) So our reproduction policy is as follows: You may quote up to 400 words of any transcript on the condition that you attribute the transcript to Seeking Alpha and either link to the original transcript or to All other use is prohibited.


If you have any additional questions about our online transcripts, please contact us at: Thank you!