Q1 2020 Earnings Call
Ladies denim stays constant scheduled to begin shortly please continue to standby. Thank you for your patience.
Operator: Today's conference is scheduled to begin shortly. Please continue to stand by. Thank you for your patience.
Unnamed Speaker: [inaudible] BF-WATCH TV 2021
Nick David Stilwell: Good afternoon, and welcome to the Sangamo Therapeutics teleconference to discuss first quarter 2020 financial results. This call is being recorded. I will now pass you over to the coordinator of this event, Nick David Stilwell, Senior Vice President of Corporate Communications and Investor Relations.
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Nick David Stilwell: Good afternoon, and thank you for joining us today. With me on this call are several members of the Sangamo senior management team, including Sandy Macrae, Chief Executive Officer, Sung Lee, Chief Financial Officer, Stephan Boissel, Head of Corporate Strategy, Adrian Wolfson, Head of Research and Development, Mark McClung, Chief Business Officer, and Bettina Cockroft, Chief Medical Officer. Slides from our corporate presentation can be found on our website, sangamo.com, under the Investors and Media section on the Events and Presentations page. This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements relating to our pipeline of genomic medicine product candidates, our ability to develop, obtain regulatory approval for, and commercialize therapies to treat certain diseases, and the timing, availability, and cost of such therapies.
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<unk> and welcome to the Sangamo Therapeutics teleconference to discuss first quarter Twentytwenty financial results. This call is being recorded I would now pass you over to the coordinator of this event Mcdavid Stilwell senior Vice President of corporate communications and Investor relay.
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Good afternoon, and thank you for joining us today.
With me this afternoon on this call or several members of the Sangamo Senior management team, including Sandy Mcrae, Chief Executive Officer sung Lee Chief Financial Officer, Stefan Whitesell head of corporate strategy, Adrian Watson head of research and development, Mark Mccollum Chief business Officer.
And picking a cockrell chief Medical officer.
Well I'd from our corporate presentation can be found at our website. Thank about dot com under the investors in media section any events and presentations page.
This call include forward looking statements regarding Sangamo current expectation.
These statements include but are not limited to statements relating to our pipeline of genomic medicine product candidates.
Our ability to develop obtained regulatory approval for and commercialize therapy to treat certain diseases, and the timing availability and cost of such therapy.
Nick David Stilwell: Plans and timelines for Sangamo and our collaborators to conduct clinical trials and share clinical data and the potential for these trials to provide clinical benefit to patients, the potential to use certain technologies to develop our therapy, the financial and operational impacts of our collaboration, plans and timelines for building and opening manufacturing facilities, the evolving COVID-19 pandemic, and our expectations regarding our financial performance and resources.
Grandson timelines for Sangamo in our collaborators to conduct clinical trials and share clinical data and the potential for these trials to provide clinical benefit to patients.
The potential to use certain technologies to develop our therapies.
Financial and operational impacts of our collaboration.
Plans and timelines for building an opening manufacturing facility.
The evolving cobot 19 pandemic.
And our expectations regarding our financial performance and resources.
Actual results may differ substantially from what we discussed today. In addition, these statements are not guarantees of future performance and are subject to certain risks and uncertainties that are discussed in documents that we file with the securities and Exchange Commission.
Nick David Stilwell: Actual results may differ substantially from what we discussed today. In addition, these statements are not guarantees of future performance and are subject to certain risks and uncertainties that are discussed in documents that we file with the Securities and Exchange Commission, specifically in our most recent quarterly report on Form 10-Q filed today and our annual report on Form 10-K. The forward-looking statements stated today are made as of this date, and we undertake no duty to update such information, except as required under applicable law. On this call, we discuss a non-GAAP financial measure. We believe this measure is helpful in understanding our past financial performance and our potential future results. However, this is not meant to be considered in isolation or as a substitute for the comparable gap measure. The comparable GAP measure and reconciliations of GAP to the non-GAP measure discussed in this column are included in today's press release, which is available on our website. And now I'd like to turn the call over to Sandy.
Specifically in our most recent quarterly report on form 10-Q filed today and our annual report on form 10-K.
The forward looking statements beta today are made as of this date and we undertake no duty to update such information, except as required under applicable law.
On this call, we discuss a non-GAAP financial measure.
We believe this measure its helpful and understanding our past financial performance and our potential future results.
This is not meant to be considered in isolation or as a substitute for the comparable GAAP measure.
The comparable GAAP measure and reconciliations of GAAP to non-GAAP measure discussed on this call are included in today's press release, which is available on our website.
And now I'd like to turn the call never defending.
Thank you Mick David and good afternoon, everyone Nicole.
Alexander D. Macrae: Thank you, MacDavid, and good afternoon to everyone on the call. Typically, when I come together with the Sangamo executive team to talk to you about our business every quarter, we are in the boardroom of the headquarters in Brisbane, California. Today, due to the COVID-19 pandemic, we're all in our home offices. One of the many adaptations we've all had to make in these unprecedented circumstances.
Typically when I come together with the Sangamo executive team to Tokyo boats her business every quarter. We're in the courtroom the headquarters in Britain, California.
Today due to the Kupek knowing pandemic, we're all in our home office. This is one of the many adaptations we've had to meet nice unprecedented circumstances.
Assigned to move over the last two months, we've worked together to minimize any impact within dentek window business and I believe that we were in a very strong position.
Alexander D. Macrae: At Sangamo, over the last two months, we've worked together to minimize any impact of the pandemic on our business, and I believe that we are in a very strong position. Last month, we were very pleased to announce the closing of our collaboration agreement with Biogen to develop gene regulation therapies for Alzheimer's disease, Parkinson's disease, neuromuscular, and other neurological diseases. We have received from Biogen $225 million in proceeds from the sale of the stock and an additional $125 million up-front license fee payment. With this $350 million received from Biogen in addition to the $363 million in cash resources that we reported on our March 31st balance sheet, we believe we have the financial strength to execute on our wholly owned and partnered development programs beyond multiple important milestones, including the potential filing of the BLA for SV525 for In response to COVID-19, Sangamo has prioritized employee safety and welfare while responsibly advancing the business. Following the shelter-in-place guidance from government authorities in the middle of March, we asked all non-Lab employees to work from home.
Last month, we were very pleased tenants to Cushing it for collaboration agreement with Biogen to develop gene regulation therapies for Alzheimer's disease, Parkinson's disease, you real muscular and other neurological diseases.
We have received combining 225 million told us and proceeds from the secret stones, and an additional 125 million upfront license fee payment.
With this $350 million receive information in addition to the 363 million in cash resources that we reported on or March 31st balance sheet. We believe we have different.
Now onto strain to execute on our wholly owned and partner development programs be on multiple important milestones, including the potential filing of the BLE for SPX like to fight for hemophilia.
In response to pick 19 cycle has prioritized employee safety and welfare of responsibly up on single business.
Following the Shelton prescribing from government authorities in the Middle of March we ask only one month employees to what.
We also implemented more despite lapping operations and updated safety protocols to continue critical research and manufacturing work.
Alexander D. Macrae: We also implemented modified lab operations and updated safety protocols to continue critical research and manufacturing work to protect employee safety and adhere to social distance guidelines. In the labs, we are strategically prioritizing projects to keep our business on track, including focusing on research activities for partner programs. We're also conducting clinical activities cautiously so as not to contribute unnecessarily to the current strain on the healthcare system while staying in close communication with trial sites to protect the safety of the patients in our trials. We continue to be optimistic that our AAV manufacturing facility in our Brisbane headquarters will be operational by the end of this year.
Protecting employee safety and achieving to social distance guidance.
And your lives, we're strategically prioritizing projects to keep our business in truck, including focusing in research activities for partnered programs.
We've also conducting clinical activities cautiously so it's not to contribute unnecessarily linked to the constraint on health care system, posting close communication was trial sites to protect the safety of the patients and their choice.
We continue to be optimistic which are easy manufacturing facility in or person headquarters will be all crucial by the end of this year.
We also expect or cell therapy manufacturing use units in brings me, California and in bone crimes to be open by the end 2021.
Alexander D. Macrae: We also expect our cell therapy manufacturing units in Brisbane, California, and in Balbon, France, to be open by the end of 2021. Furthermore, we are keeping in regular contact with our CDMO partners and, as of now, do not anticipate any COVID-19-related manufacturing delays in our activities. Despite the challenges of the current environment, we continue to actively pursue new partnerships, both inbound to access new technology, as well as outlicing some deals, such as the established ones that we already have with Pfizer, Gilead, Sanofi, Biogen, and Takeda. An important example of an inbound partnership is our recently announced collaboration and exclusive license with Mogrify, a Cambridge, UK, company developing novel cell conversion technology that has the potential to serve as a renewable cell source.
Furthermore, we are keeping in regular contact with her she didn't move partners and that's something I do know anticipate any <unk> could 19 related manufacturing to lease with their activities.
Despite the challenges at the current environment, we continue to actively pursue new partnerships in buying toxicity technology as what was licensing deals such as they stopped leash ones that we already have with Pfizer Gilliat, Sanofi Biogen and chicken.
An important example, overland Ben partnership is our recently announced collaboration and exclusive license with microphone I cant groups you keep company developing local show conversion technology that has the potential to serve as a renewable source.
Under the terms of this agreement signed to move into you spoke refineries proprietary so conversion technology to do pellet allogeneic zinc finger Pucci gene engineered car T. Reg cells therapies, using I P. S C to write regulatory T cells.
Alexander D. Macrae: Under the terms of this agreement, Sangamo aims to use Mography's proprietary cell conversion technology to develop allogeneic zinc finger protein gene-engineered CAR Treg cell therapies using IPFC-derived regulatory T-cells. This license agreement will diversify our options and complement our current efforts as we develop off-the-shelf allogeneic CAR Treg cell therapies. We believe that this exciting collaboration has the potential to accelerate the development and manufacturing of novel renewable cell source Treg therapy.
This license agreement, we took our supply interruptions and complement our current efforts as we develop off the shelf allogeneic car T Reg cells therapies.
We believe that this exciting collaboration has the potential to accelerate the development and manufacturing Oh, no who renewed <unk> sole source T regs therapies.
We're also continuing our discussions on licensing opportunities as a reminder, our strategy for collaborations is took on selected Goldman programs in partnership with biopharmaceutical companies in situations, where we believe that a partner spin onshore resources or clinical and therapeutic expertise me enable more.
Alexander D. Macrae: We are also continuing our discussions on out-licensing opportunities. As a reminder, our strategy for collaborations is to advance select development programs in partnership with biopharmaceutical companies in situations where we believe that a partner's financial resources or clinical and therapeutic area expertise may enable more rapid development and availability of new treatments to patients. Before I turn the call over to the team, I want to update you on some recent transitions amongst my direct reports that are a natural part of Sangamo's evolution as we advance our clinical development and strategic partnership. In the last three years, we've added executive vice presidents to lead manufacturing, legal, finance, and R&D. In our latest executive appointment, as we look to the future and perceive the need for commercial development expertise and capabilities, Mark McClung has joined Sangamo as Chief Business Officer and now leads Commercial Strategic Planning, Alliance Management, and Corporate and Business Development.
Rapid development and the people to come to you treatment to patients.
Before I turn the call over too cheap I want to update you on some recent transitions amongst my direct reports dollar a natural part of cycles evolution, as we advance or clinical development and strategic partner.
Over the last three years, we thought that executive Vice presidents to lead manufacturing legal finance and John D.
And our latest executive appointment I speak to the future and proceed the need for commercial development expertise and capabilities Mark Mccollum has joined sizeable as Chief business Officer, No leads commercial strategic planning Alliance management, and corporate and business development.
'cause a distinguished career, leading commercial organizations, including GSK owned mix, an assumption and highly competitive therapeutic areas that requires deep scientific knowledge.
We are individually province have disrupted the standards of care.
Are successful product development occurs as a result in the tightly integrating patient and physician insights into late stage clinical development programs.
Stephen for sale or de VP of corporate strategy will recycle the into July and plans to return to an entrepreneurial project.
Stephen joined Sanquin 2018, after we acquired T. XL, no single friends, which Stephens, let us see deal.
His energy envision resulted in a recent biogen transaction, which is one of the largest preclinical cooperation do ever in the biopharmaceutical industry.
Stephens impactful contribution to cycle will endure for many years.
With that I'll turn the call will for Chief Medical Officer the Tina.
Good afternoon.
In light of the Cobot 19 pandemic suddenly is looking very closely with all our clinical trial site pot nets to devise individualized plan to protect the safety of every patient enrolled enough study as well as the continued integrity of our trial data.
Alexander D. Macrae: Mark has a distinguished career leading commercial organizations, including GSK, Onyx, and Amgen, in highly competitive therapeutic areas that require deep scientific knowledge and where innovative products have disrupted the standards of care, and where successful product development occurs as a result of tightly integrating patient and physician insights into late-stage clinical development programs. Stefan Borsell, our EVP of Corporate Strategy, will leave Sangamo at the end of July and plans to return to an entrepreneurial project. Stéphane joined Sangamo in 2018 after we acquired TXL, now Sangamo France, which Stéphane led as CEO. His energy and vision resulted in a recent Biogen transaction, which is one of the largest preclinical collaboration deals ever in the biopharmaceutical industry. Stefan's impactful contribution to Sangamo will endure for many years. With that, I will turn the call over to our Chief Medical Officer, Bettina.
After we have established uptime for each patient we work with tight and I have east establish appropriate procedure in some cases trial site partners have been reducing or posing clinical trial, but to redirect capacity, we started to come at 19 patients.
The sites.
Nickel trial patients I still being screened and enrolled that may not be done until in at appropriate times identified.
In addition to adopting our clinical operations to eat situation.
We are implementing procedures now so that as the code at 19 crisis side, we'll be able to redeem operations as quickly as efficiently as possible.
To do they were keeping in close contact with our trial site hotness I'm continuing to identify potential patients.
Maybe suitable for enrollment.
In some cases, we'll see using this time to further optimize clinical operations processing and engage with regulatory agencies.
We're also working closely with our collaboration partners to track the stated about joint development program.
Turning now to some clinical updates commencing with SP five to five for P.F. zero seven zero I fight for eight zero hemophilia 18 therapy.
We transferred operations of the fully enrolled phase one two hours to study to fighter along with the I. Indeed at the end of last year.
We're working closely together with fights, it's why didn't find appropriate opportunity. They see it to provide an update on the results that we said it is 29 team from the high dose expansion cohort.
Bettina M. Cockroft: Good afternoon. In light of the COVID-19 pandemic, Sangamo is working very closely with all our clinical trial site partners to devise individualized plans to protect the safety of every patient enrolled in our studies, as well as the continued integrity of our trial data. After we have established a plan for each patient, we work with sites and IRBs to establish appropriate procedures. In some cases, trial site partners have been reducing or pausing clinical trial work to redirect capacity and resources to COVID-19 patients. At other sites, clinical trial patients are still being screened and enrolled but may not be dosed until an appropriate time is identified. In addition, we have adapted our clinical operations to this new situation. We are implementing procedures now so that as the COVID-19 crisis subsides, we'll be able to resume operations as quickly and as efficiently as possible. To do this, we're keeping in close contact with our trial site partners and continuing to identify potential patients that may be suitable for enrollment. In some cases, we're also using this time to further optimize clinical operations processes and engage with regulatory agencies. We're also working closely with our collaboration partners to track the status of our joint development program.
Hi, there continues to target dosing the first patient in the phase three study in H. to Twentytwenty.
Hi, so he's working to minimize any potential disruptions to the schedule due to the ongoing Cook at 19 pandemic and continues to recruit patients into the phase three lead and study.
Hi, So recently updated clinical trials dot Gov with the phase three study protocol and have informed us that they plan to provide additional updates on the phase three trial in due course.
Moving now to our wholly owned gene therapy S. T 920 for Fabry disease.
<unk> success study screened and enrolled patients into the Star study and we are waiting for a safe and appropriate time, finishing those insights patient.
In conjunction with our partner Sanofi, we're evaluating gene editing cell therapies, the two hemoglobinopathies.
5400 to be that's how the female and BBB doublets free for sickle cell disease.
I see 400, and the I'd be double up three both design tend to use the synthesis of fetal hemoglobin. This is achieved by gene edited knock out of the with Troy specific enhancer that B.C.L., 11, 18, which encodes a strong repressed or if the gamut globin gene.
In beta thalassemia, if people hemoglobin is expressed at high enough level. It may substitute for patients absent or impact levels of pizza oven.
We have enrolled and dosed five patients into the failed study evaluating its the 400 for because at the senior.
In sickle cell disease increased fetal hemoglobin synthesis may provide the patient with functional hemoglobin and help down regulate the abnormal sickle hemoglobin that results in painful sickle cell crises and other disease feature.
Sanofi has also been a tolling patients into the precise one study evaluating VIP be double O three sickle cell disease and dosed the first patient last year.
You're not assist other studies data will be sad when the two studies have accumulated a sufficient number of the patients and follow up.
No additional Peter fallacy, new patients NFL study will be treated until the data from both studies have been collected and analyzed.
Bettina M. Cockroft: Turning now to some clinical updates, commencing with SB525 or PF07055480, hemophilia A gene therapy. We transferred operations of the fully enrolled Phase I-II Alpha study to Pfizer, along with the IND, at the end of last year. We're working closely together with Pfizer to identify an appropriate opportunity this year to provide an update on the results that were shared at ASH 2019 from the high-dose expansion cohort. Pfizer continues to target dosing the first patient in the Phase 3 study in H2 2020. Pfizer is working to minimize any potential disruptions to the schedule due to the ongoing COVID-19 pandemic and continues to recruit patients into the Phase 3 lead-in study.
Sanofi will in the meantime continue enrolling sickle cell patients into the precise in one study.
We will look for an appropriate time to present data from both the studies at a future date.
I'd like to conclude by addressing a few programs that we're monitoring closely with regard to potentially impact I feel that 19.
We continue to make progress with additional regulatory approvals for the phase one two steadfast study.
If I anything affecting human car T. Reg cells therapy T X 200, NHL, a two mismatched kidney transplantation.
We expect to those the first patient in this study in 2021.
Moving on now to tight zero 37, and allogeneic anti Cdnineteen car T cell product being developed by kite Good company.
Hi has informed us that there is a potential for coated related delay to the initiation of the tight feel with 37 clinical trial.
I'll now turn the call over some friend, who will review the financial results some.
Thank you Bettina and good afternoon, everyone. We're pleased to share our financial results for the first quarter of 2020.
We reported a net loss of $42.9 million or 37 cents per share compared to a net loss of $42.2 million were 41 cents per share for the same period in 2019.
Bettina M. Cockroft: Pfizer recently updated clinicaltrials.gov with the Phase 3 study protocol and has informed us that they plan to provide additional updates on the Phase 3 trial in due course. Moving now to our wholly-owned gene therapy ST920 for Fabry disease. We've successfully screened and enrolled patients into the STAR study, and we are awaiting a safe and appropriate time to initiate dosing the first patient. In conjunction with our partner Sanofi, we're evaluating gene-edited cell therapies for two hemoglobinopathies. ST400 for beta thalassemia and BIBV003 for sickle cell disease. ST400 and BIBV003 are both designed to induce the synthesis of fetal hemoglobin. This is achieved by gene-editing the erythroid-specific enhancer of the BCL11A gene, which encodes a strong repressor of the gamma globin gene.
The revenues were $13.1 million compared to $8.1 million for the same period in 2019.
Turning to expenses.
Non-GAAP operating expenses, which excludes stock compensation were $52 million compared to $47.4 million and 29 team.
The increase in operating expenses was primarily related to the company's overall headcount.
And facilities expansion to support the advancement of Sangamo therapeutic pipeline and manufacturing capability.
Moving to the balance sheet.
We ended the quarter with $363 million in cash cash equivalents and marketable securities.
Following the end of Q1, we received $350 million from Biogen from the sale stock and the upfront license fee.
As Andy mentioned earlier, we believe we have to balance sheet strength to take us through important R&D milestones, including the first potential filing of the be away for SP five to five for hemophilia.
Turning to 2024 your guide.
We are reiterating our previously shirt financial guidance and anticipate non-GAAP operating expense, which excludes estimated stock compensation expense of $25 million to be in the range of $245 million to $260 million and 2020.
Bettina M. Cockroft: In beta thalassemia, if fetal hemoglobin is expressed at high enough levels, it may substitute for a patient's absent or impaired levels of beta globin. We have enrolled and dozed five patients into the failed study evaluating ST400 for beta thalassemia. In sickle cell disease, increased fetal hemoglobin synthesis may provide the patient with functional hemoglobin and help downregulate the abnormal sickle hemoglobin that results in painful sickle cell crises and other disease features.
At this time, we do not expect any material negative financial impact from Kogas 19 to our operating expense guidance.
We will continue to monitor the situation and provide an update in the future in the meantime, we will continue to be good stewards of capital.
I'll now turn it back to Sandy for closing remarks.
Thank you so.
This quarter marks an important milestone with the closing of the Biogen agreement, which will significantly strengthened our balance sheet and represents yet another boots have confidence and are highly differentiated sink finger coaching genomic medicine platform from a toll biopharmaceutical company.
In these unprecedented times I've observed tremendous resilience and adapt business from our employees and this is kept her business moving forward, including ongoing business did those discussions continue to search and technical operations are reporters and continued partnerships with our clinical trial sites.
Sung Lee: Sanofi has also been enrolling patients into the PRECISEN1 study, evaluating DIVV003 in sickle cell disease and those who were the first patient last year. New analysis of the study's data will be shared when the two studies have accumulated a sufficient number of patients and follow-up. No additional beta thalassemia patients in the failed study will be treated until the data from both studies have been collected and analyzed. Sanofi will, in the meantime, continue enrolling sickle cell patients into the Precise in One study. We will look for an appropriate time to present data from both these studies at a future date. I'd like to conclude by addressing a few programs that we are monitoring closely with regard to potential impact by COVID-19. We continue to make progress with additional regulatory approvals for the Phase I-II Steadfast Study, evaluating our first-in-human CAR Treg cell therapy Tx200 in HLA-A2 mismatched kidney transplantation.
We feel a great sense of confidence in our business and in or ability to weather the corporate banking crisis due to our balance sheet strength strategic investments in infrastructure and to the prudent plans that we've been we've established to facilitate our branded returned to more normal operations.
As this crisis subsides.
Operator, we're ready for questions.
Thank you as a line to ask a question you depressed our wondering if that's probably telephone to withdraw your question. Please press the pound to one moment. Please.
Our first question Costamare right off of Jefferies. Your line is open.
Hi, everyone. Thanks for taking my questions.
First question is just on hemophilia age so with the phase three trial posted two clinical trials that go can you talk more about the dine at this time, including dose steroids views and what estimates are on how long it it's going to take you enroll the study.
More respect thanks for your question.
As you can imagine the the phase three trial is central to controlling and communication of Pfizer and they will get bowl announcements about deciding if the trial.
And we want to respect that relationship with them.
Everything they have told us so far got each to the trial moving ahead just fund one of the advantages of our partnership with Pfizer is sort of global organizations continue to Charles two groups of patients are available and where the coolfit impact is useless. So we look forward to them.
Sung Lee: We expect to dose the first patient in this study in 2021. Moving on now to KITE-037, an allogeneic anti-CD19 CAR-T cell product being developed by KITE, a Gilead company. KITE has informed us that there is a potential for a COVID-related delay in the initiation of the KITE-037 clinical trial. I will now turn the call over to Sung for an overview of the financial results. Sung?
Sharing more information with you as the year progresses.
Understood understood and then for Fabry I heard you guys mentioned before that.
He had more patient screen failures and you initially expected just wondering if you implemented enrollment criteria changes and die and those been helping.
Patina would you be able to talk to that.
Yes of course, hi, there. So we have seen I'm looking at implementing some changes and as you will have noticed from the the communication today, we have actually enrolled patients into the Fabry study no of course during the cold.
And then Nick we're being very cautious us to assessing the best timing for dosing. The first subject, but we have had an up tick and and that has resulted in.
Inclusion of patients.
Alexander D. Macrae: Thank you, Bettina, and good afternoon, everyone. We're pleased to share our financial results for the first quarter of 2020. We reported a net loss of $42.9 million, or $0.37 per share, compared to a net loss of $42.2 million, or $0.41 per share, for the same period in 2019. The revenues were $13.1 million, compared to $8.1 million for the same period in 2019. Turning to expenses, non-GAAP operating expenses, which exclude stock compensation, were $52 million, compared to $47.4 million in 2019. The increase in operating expenses was primarily related to the company's overall headcount growth and facilities expansion to support the advancement of Sangamo's therapeutic pipeline and manufacturing capabilities. Moving to the balance sheet, We end the quarter with $363 million in cash, cash equivalents, and marketable securities.
Into the study.
And patina you feel the changes you made to the protocol.
Permitted the facilitated though.
Exactly absolutely absolutely.
Okay, and then last quick one who's just on.
Wondering if you have formalize plans to conduct a real biopsy for Gbpthree reduction in this initial part of the study or is that come in later on maybe if you just talked more about the plans there.
We haven't we haven't discussed our plans for the Gbpthree Infrarenal biopsy.
She can have much and <unk> is a complex issue a boat patient benefits and the risk of a real biopsy. We on we're very appreciative of the ft.
Trying to make medicines for fabry get to patients as quickly as possible by lowering registration quicker with a with a regional biopsy and are very aware of and are incorporating it into our clubs.
Got it thank you for taking my questions.
Thanks Morry people.
Thank you. Our next question comes from Gena Wang of Barclays analyze okay.
Thank you for taking my question.
Two questions. The first is regarding <unk> ft.
Second question is I'm happy to see who <unk> E items.
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He actually see who Q this year like who would be good paldon tissue.
Well no quota. So you can see you didn't mention mboe several patients just wondering how medication.
And also is miscue possibility is named initial data or beginning of next year also can you remind us the first dose what's happening.
So I'll, let me to the first question for costs are you willing to between a warning you that we haven't talked about the first dose yet.
But if I could do the the.
Hemophilia a.
Unfortunately the world.
Hi moves that just the same rate and patients are only noted coming out at their 18 month point to not Super League patients and it boutique throughout the rest of the this and the next quarter for the majority of patients to reach their 18 month point, So Pfizer blue will.
Alexander D. Macrae: Following the end of Q1, we received $350 million from Biogen, from sales stock and the upfront license. As Sandy mentioned earlier, we believe we have the balance sheet strength to take us through important R&D milestones, including the first potential filing of the BLA for SB-525 for hemophilia A. Turning to the 2020 full year, guys, we are reiterating our previously shared financial guidance and anticipate non-GAAP operating expense, which excludes estimated stock compensation expense of $25 million, to be in the range of $245 million to $260 million in 2020. At this time, we do not expect any material negative financial impact from COVID-19 on our operating expense guidance. We will continue to monitor the situation and provide an update in the future. In the meantime, we will continue to be good stewards of capital. I will now turn it back to Sandy for her closing remarks.
Lead all communication says as part of the do for for transition like this you agree which company will lead to old communications and the isn't Pfizer turns and they will decide when they have due to that they will share with fuel I know this is frustrating but is it has to be a single.
The company that that Lisa.
And patina can you talk boats are enrollment and fabric, yes lease absolutely. So so as far as fabry is concerned.
To address your last question first we're going to be sharing data. After we've completed dose escalation across the three cohorts that we have a protocol we want to make sure that we present the middle mature dataset that can represent safety and efficacy of S.T. 920.
In terms of which those is we have work.
Turning to.
The test we have said low medium and high we will disclose specific those is at an appropriate point in the future. What I can say, we've learned a lot about 86 through our five to five he will be a program.
And we've made protocol amendments in the fabric program to take those learnings and also if any guidance into account. So we look forward to updating further on this didn't feature.
Okay, how many patients already has no.
I think will not be doesn't go.
Et cetera, we're not disclosing the but but we have patients enrolled we have interest from sites and it's just a matter of those choosing point to do so first patient I'm sure you would agree the we need to choose wisely because of patients will have to come in for.
Operator: Thank you so much. This quarter marks an important milestone with the closing of the Biogen Agreement, which significantly strengthened our balance sheet and represents yet another vote of confidence in our highly differentiated zinc finger protein genomic medicine platform from a top biopharmaceutical company. In these unprecedented times, I've observed tremendous resilience and adaptability from our employees, and this has kept our business moving forward, including ongoing business development discussions, continued research and technical operations in our laboratories, and continued partnerships with our clinical trials site. We feel a great sense of confidence in our business and in our ability to weather the COVID-19 crisis, due to our balance-sheet strength, strategic investments in infrastructure, and to the prudent plans that we have established to facilitate our As this crisis subsides, Operator, we are ready for questions.
Monitoring and we want to make sure that they're safe and that the health service is no overstressed.
Yeah.
Thank you.
Thank you and next question comes from Whitney I don't have the there how your line is open.
Hey, guys. Thanks for taking the question I wanted to follow up on average. So I guess first can you give us any color on the entry criteria that were adjusted.
Dilatation enrollment just curious if we could learn more there and then the second question is on the endpoint. You mentioned you won't do that date until you have a complete dataset I guess, what does that mean in terms of follow up on than kind of what endpoints are you tracking or is that the 12 month I'm kind of safety thought that's reflected on quint trout Lake.
So just if we can thank you for your question.
The criteria that we adjust it we're not things of both.
And to bowtie criteria like gene therapy thinks they were more or understanding of what some pre patients like import the right patients to put into the study each time a company like covers goes into a new to cease we learn from the first few patients and Bettina and her team have done an excellent job and and simply understanding for peace.
Sort available when we see we wouldn't talk about the study until its completed Minas that we've gone through to each of the dose cohorts and as soon as we have body chemistries data from each of the dose cohort so little meacham at all your strategic said, we will share them with you.
Maurice Thomas Raycroft: Thank you. As a reminder, to ask a question, you need to press star 1 on your touch-tone telephone. To withdraw your question, please press the pound key. One moment, please. Our first question comes from Maurice Raycroft of Jeffries. Your line is open.
You're absolutely right that the rupee fold alone due to the Luca additional parameters, including in some patients biopsy.
But we hope to be able to share to biochemistry initially and told you about its the results of or intervention.
Got it just quick follow up in terms of the biochemistry on what particular endpoints I guess are you looking at there and whats the cut off I guess is it like three months or statements about chemistry, you want to have at that higher does the ability to the threshold for.
Alexander D. Macrae: Hi everyone. Thanks for taking my questions. The first question is just on hemophilia. So with the phase three trial posted to clinicaltrials.gov, can you talk more about the design at this time, including dose, steroid use, and what estimates are on how long it's going to take to enroll the study?
Announcing the data.
We have we haven't described we.
You've been with us for a long time, and you understand or cautiousness and speaking to soon.
And in wishing for the the results to stabilize so as we can moose informed you and more some from the patient community.
Alexander D. Macrae: Maurice, thanks for your question. As you can imagine, the phase 3 trial is under the control and communication of Pfizer, and they will make all announcements about the design of the trial, and we want to respect that relationship with them. Everything they have told us so far points to the trial moving ahead as planned. One of the advantages of our partnership with Pfizer is that they're a global organization that can take the trial to where the patients are available and where the COVID impact is less. So we look forward to sharing more information with you as the year progresses.
Understood Thanks very much.
Thank you. Thank you.
Thank you again, ladies and gentlemen, if you like that the question Cooper Star then one on your Touchstone telephone.
Our next question comes what you Barra Cowen Your line is okay.
Hi.
You guys. Thanks for taking my question and I I'm going to Cook another stab at that the fabric screening criteria question.
Can't help myself I I understand.
HM there's different aspects because it could be even almost sounds like there's there have been other companies in fabry gene therapy, and they maybe not optimized initial patients and.
Specifically you know specific to fabry are you more interested including the effect in.
Bettina M. Cockroft: And then for Fabre, you guys have mentioned before that you had more patient screen failures than you initially expected. Just wondering if you've implemented enrollment criteria changes, and if those have been helpful.
The patient, we want and the unmet need across our key small molecule in some emerging.
Okay, Yes.
So I'm good to lit bettina talk to us in a moment, but.
You can imagine this is first time any of us have moved a gene therapy into fabric.
And with the first few patients we learn about the effect to pharmacology, the plasma levels and with the first few kidney biopsies, we will learn from doors. So I think would be.
Bettina M. Cockroft: Bettina, would you be able to talk to them?
Bettina M. Cockroft: Yes, of course. Hi there. So, we have been looking at implementing some changes, and as you will have noted from the communication today, we have actually enrolled patients into the FABRI study. Now, of course, during the COVID pandemic, we are being very cautious as to assessing the best timing for those in the first subject, but we have had an uptick, and that has resulted in the inclusion of patients into the study
Premature for me to clear for is to the right thing to do we last week or the week before had three fabry patients take part in the company White.
Zoo conversation talking about the things that Stu both of them.
And it's clear that fabry patients are not.
Receiving all the benefits they need and there's still a medical need the patina, we'd like to talk to them.
Alexander D. Macrae: And Bettina, do you feel that the changes you made to the protocol have permitted that, or facilitated that?
Yes of course, and as you say Sunday, we've had conversations with many experts in the field fabry to address exactly you know into your highlighting kidney cardiac.
Bettina M. Cockroft: Exactly, absolutely, absolutely.
Other work and organs being affected.
Well, we should point out at this point in on trial. This is a phase one two trial. It's a primary objective at this point of safety and Tolerability of S.P. Nytwenty.
Alexander D. Macrae: Okay, and then last quick one was just wondering if you have formalized plans to conduct a renal biopsy for GB3 reduction in this initial part of the study, or could that come in later on? Maybe if you could just talk more about the plans.
So we hope to see some of the safety and Tolerability as we saw with the 86 based chemotherapy SP five to five.
And moving on from there as we learn we can focus on me organs that make much sense moving forward to address that need.
Alexander D. Macrae: We haven't, we haven't discussed our plans for the gb3 and for renal biopsy. As you can imagine, that is a complex issue about patient benefit and the risk of a renal biopsy. We are very appreciative of the FDA. I'm trying to make medicines for Fabry get to patients as quickly as possible by allowing registration quicker with a renal biopsy. We are very aware of that and are incorporating it into our
Got it and then sandals and mentioned.
Yes, you guys are.
Moving forward with activity you mentioned, some additional regulatory meeting that goal and goals going to engage with regulatory agencies with some of the new bankrupt that you have in coal, but could you could you elaborate on what sort of.
Oh regulatory meetings, he you might need obviously T Reg.
Clinic fabric moving into the clinic.
Can you give us a little color around that.
So can I make sure I understand your question, you're asking are we hoping additional regulatory meetings with the agency or with agencies, arriving coolfit.
Maurice Thomas Raycroft: Thank you for taking my questions.
Operator: Thanks, Maurice. Be well.
Huidong Wang: Thank you. Our next question comes from Gina Wang of Barclays, Jelena Doba. Thank you for taking my questions. I have two questions.
Oh, no not around co that but in your comments initially around how.
There have been changes with work streams with Covance. It sounded like you had the opportunity to engage with regulatory agencies.
Alexander D. Macrae: The first is regarding hemophilia A update, and the second question concerns febrile disease. As regards hemophilia A update, I understand Pfizer emphasized the importance of the 18-month data. Giving follow-up from last ASH, is it fair to say flu Q this year likely will be a good time to show data? And for the febrile disease, you did mention you enrolled several patients. Just wondering how many patients? And also, is it still possible to present the initial data at the beginning of next year? Also, can you remind us of the first dose for Fabry disease?
More on programs or I may have misunderstood you.
But I'd love to know what regulatory meetings or.
Land, saying in the next quarter or to on the programs.
So I'm going to persist two patina too.
To help some this one and then I'm going to make more general comments Bettina.
Yes, absolutely so you're absolutely right, we mentioned based on your own today, but we're focusing on mainly and you've picked it up is on T. X 200, specifically, where we are engaging with regulatory agencies, especially the various European ones in the day countries that we are.
Initiating on ARQ <unk> 200 study and so we're going through all those regulatory approval processes for that for that study and I'm. Not study is receiving a lot of feedback even doing that and then make many of the agencies is still working on hard but.
Alexander D. Macrae: So, let me take the first question before passing you on to Bettina, but warn you that we haven't talked about the first dose yet. But if I could do the...
Alexander D. Macrae: Team Affiliate, eh? Unfortunately, the world... Time moves at just the same rate, and patients are only now coming out at their 18-month point, and that's just the very early patients, and it will take throughout the rest of this and the next quarter for the majority of patients to reach their 18-month point. So Pfizer will, and it has to be a single company that leads that. And Bettina, can you talk about our involvement in FABRI?
That's exactly what we're engaging.
And a little more general basis.
But on a more general basis.
Hope Coolfit impacts are program I sit in the portfolio and there we have a regular conversation about how companies with in some ongoing studies are helping to make.
Bettina M. Cockroft: Yes, absolutely. So, as far as Fabry is concerned, to address your last question first, we're going to be showing data after we've completed the escalation across the three cohorts that we have in our protocol. We want to make sure that we present a mature data set that can represent the safety and efficacy of ST920 and in terms of which doses we were planning to test. We have said low, medium, and high. We will disclose specific doses at an appropriate point in the future. What I can say is we've learned a lot about AAV6 through our SB525, you know, VIA program, and we've made protocol amendments in the FABRI program to take those learnings and also FDA guidance into account. So we look forward to updating you further on this in the future.
Modifications to the political modifications for the current data on the agency for too that the has been very understanding of a single happened.
Those conversations with us for your questions.
Got it.
Last question Sorry, Sandy you also mentioned that you may be considering other.
Ah partnerships partnership opportunity can you talk to the program now in your current lineup that you think would most appropriate for a partnership purchases.
Summer clicking going forward.
So if you think of what happened with Biogen.
There was one.
One of those products the tow for outside of or so we've been speaking tool for a while about once a nuclear in that we've spoken for three months and the other 10 were things of that.
None of US news, we're in the Sangamo pipeline.
There are things that we could partner that you are aware off and we have no plans for the moment to sort of a partner our existing assets in the pipeline that you think office singles, but are also many other targets within the genome even within the CNS speed.
Bettina M. Cockroft: Okay, how many patients already?
Bettina M. Cockroft: I think we're not.
Bettina M. Cockroft: I think we're not going to go... Yeah, exactly. We're not disclosing that. But we have patients enrolled, we have interest from sites, and it's just a matter of us choosing when to dose the first patient. I'm sure you would agree that we need to choose wisely because the patients will have to come in for monitoring, and we want to make sure that they're safe and that the health service is not overstress
Yes.
Our northparkes if our current go ahead.
Folio, but the others are interested though.
Got it Super helpful. Thanks for taking all the questions.
Thank you Tim anything.
Thank you.
Next question comes and Jim Birchenough of Wells Fargo Securities. Your line is open.
Hi, Thanks for taking the questions. This is the yen and dialing in for Jim for first a question on the Alzheimer's.
Programming in collaboration with Biogen.
Bettina M. Cockroft: Thank you. Our next question comes from Whitney Isom of Guggenheim. Your line is open. Hey guys.
Wondering or have you decided what might be the optimal delivery route for that program, whether its ivy or a increase per and commercial type injections and also secondarily. It thier I'm not a milestone the first mile.
Whitney Isom: Thanks for taking the questions. I wanted to follow up on FABRI. So, I guess, first, can you give us any color on the entry criteria that were adjusted to facilitate enrollment? Just curious if we could learn more there. And then the second question is on the endpoints. You mentioned you won't present data until you have a complete data set. I guess, what does that mean in terms of follow-up? And kind of what endpoints are you tracking, or is that the 12-month kind of safety follow-up that's reflected in CLIN trials? Thanks.
So that you think you will be communicating around it.
So just wondering the timeline for example in nomination of a candidate or a first time D. Any sense, so when that might be.
So a bit like what we've said with Pfizer they will be Biogen to me. This communications I wonder if anything could comment on which the delivery instead phone on milestone so it could perhaps first.
Yeah, Thanks, Sandy and thing for the question.
We have something says we happen to close the roots of delivery.
And that will be worried that the barge and to do that but I will say for example, if you. If you look at your before you see.
Over the last week, but we'll be talking about some of our a time with the rights New Oh, I'm, sorry, it's hard to being a Directv pollution. Following all types that library that we put an active program doing that and obviously anything that comes out of those programs will be a relevant rocket barge or put it back.
Alexander D. Macrae: So. Thank you for your question. The criteria that we adjusted were not things about antibody criteria, like gene therapy things. They were more our understanding of what Fabry patients look like and who are the right patients to put into the study. Each time a company like ours goes into a new disease, we learn from the first few patients, and Bettina and her team have done an excellent job simply understanding what patients are available. When we say we won't talk about the study until it's complete, what we mean is that we've gone through each of the dose cohorts. And as soon as we have biochemistry data from each of the dose cohorts, the low, medium, and high, as Bettina has said, we will share them with you. You're absolutely right that there will be follow-on data that will look at additional parameters, including in some patients' biopsy. But we hope to be able to share the biochemistry initially and talk to you about the results of our intervention.
And that's the presentation, but if it were gela.
Yes, that's right Sandy where we are we make these are captured libraries with different part in fashion sounds insertion sites are using a range of different parental third party.
Regarding distefano Butler.
Yeah Yeah.
God and stuff and the the way the contract just is constructed.
So, yes Sunday, we closed.
Biogen D. Only a few weeks ago, we had the on boarding of the collaboration and be a few days ago. So.
We need to be if he could be shouldn't vote for the public club Ocean two really.
Good started for the photos taken could meet you do need before we can make any update as to the to the time nine undervalues public we agreed to welcome we've Oh friends on by region.
But the other thing first the first startup meeting was very successful here.
Oh, it was and despite a very bizarre our unique setting because equally split that he gets you a month, we add something like 80, there some combination of Biogen sangamo employee of the food and resume as well Amit was yeah. We are in success despite began.
Kind of to be down 15 to sounds good.
Thank you that's very helpful. And then just two quick questions regarding the Fabry disease program. Just wondering what what is your competitive positioning against other Avi Fabry gene therapy approaches and what's your impression.
Alexander D. Macrae: Got it. And just a quick follow-up. In terms of the biochemistry, what particular endpoints, I guess, are you looking at there, and what's the cutoff? I mean, is it like three months or six months of biochemistry you want to have at that higher dose to kind of be the threshold for announcing the data?
From a the initial by a biomarker data regarding enzyme that was presented recently.
Alexander D. Macrae: We haven't described that. You've been with us for a long time, and you understand our cautiousness in speaking too soon and in waiting for the results to stabilize so that we can most inform you and most inform the patient community.
And whether you like okay, youre, sorry, whether youre low dose cohort starting to occur or you think you can achieve you know similar or higher.
In terms of enzyme level. Thank you.
So can I just clarify your question are you seeing in the second part with this are you, who you're comparing against three line or couponing or freelance but.
Whitney Isom: I understand. Thanks very much.
Avi yeah freely thanks.
Operator: Thank you. Thank you.
So let me let me see if I can answer both of these.
Chew Vora: Thank you. Again, ladies and gentlemen, if you'd like to ask a question, please press stars and 1 on your touchtone telephone. Our next question comes from Chew Vora of Cali. Your line is open. Good afternoon, guys.
None of those concluding that we have something right.
[laughter] different until we get into the clinic can see the results in patients on there of course, that's why we're going to wait and complete the study atoll three doses I do think it would be helpful for us to computer or lose versus feeling pluto's because all that matters is the final doors. So you take into the clinic.
Alexander D. Macrae: Thanks for taking the question. I'm, I'm going to take another stab at the Fabry screening criteria question. I can't help myself, I understand that there are different aspects of the disease, and as you said, there have been other companies in Fabry gene therapy, and they've maybe not optimized initial patients, and specifically, you know, specific to Fabry, are you more interested in proving the effect in Fabry manifestations of kidney? Can you talk about how you think about unmet need in kidney versus unmet need in cardiac, maybe other organ systems The patient you want and the unmet need across ERT, small molecules, and some of the other gene therapies addressing the condition.
We are pleased with the design of our study we're pleased with the optimal results. Some we're looking forward to showing clinical results and this bettina emphasize this study is a boat safety. So we will be looking at liver enzyme who can't cardiac enzymes, we'll be making sure that this is a safe as possible. So make when you have done.
But you can then start to computer on efficacy.
Got it yeah, that's very helpful. Thank you sandy.
Thank you.
I'm showing no further questions at the time I, just kind of call back over to things you're correct for any closing remarks.
Sankyo for your interest. Thank you referred to during this virtually we are very fortunate, saying, it's saying them, which will to proceed with.
Virtue team I never left stuff in in the laboratories, and we look forward to sharing more results with you. So your progress.
Alexander D. Macrae: So I'm going to let Bettina talk to us in a moment, but... You can imagine this is the first time any of us have moved gene therapy into Fabry, and with the first few patients, we will learn about the effect, the pharmacology, the plasma levels, and with the first few kidney biopsies, we will learn from those. So I think it would be premature for me to declare what the right thing to do is. Last week or the week before, we had three Fabry patients take part in a company-wide Zoom conversation talking about the things that still bother them, and it's clear that Fabry patients are not receiving all the benefits they need, and there's still a medical need. But Bettina, would you like to talk to him about that?
Thank you.
Ladies and having that that topic [laughter] anything out there right.
Right Okay.
[music].
Bettina M. Cockroft: Yes, of course, and as you say, Sandy, we've had conversations with many experts in the field of Fabry to address exactly what you were highlighting, kidney and cardiac, and other organs being affected. What we should point out at this point in our trial, this is a Phase I-II trial; it's our primary objective at this point to observe safety and tolerability of ST920, and so we hope to see similar safety and tolerability as we saw with the AAV6-based gene therapy, SB525, and moving on from there, as we learn, we can focus on the organs that make most sense moving forward to address that unmet need
[music].
Bettina M. Cockroft: Got it. And then Sandy, you mentioned As you guys are moving forward with activities, you mentioned some additional regulatory meetings that you're engaged in going to engage with regulatory agencies with some of the new bandwidth that you have in COVID. Could you could you elaborate on what sort of regulatory meetings you might need? I mean, obviously, T-REG is moving into the clinic, FABRI is moving into the clinic.
Chew Vora: Can you give us a little color around that?
Alexander D. Macrae: So can I make sure I understand your question? You're asking, are we having additional regulatory meetings with the agency or with agencies around COVID?
Chew Vora: No, not around COVID, but in your comments initially around "There have been changes with work streams with COVID." It sounded like you had the opportunity to engage with regulatory agencies some more on programs, or I may have misunderstood you. But I'd love to know what regulatory meetings are planned, say, in the next quarter or two on the program.
Bettina M. Cockroft: So I'm going to pass this to Bettina to help out on this one, and then I'm going to make more general comments. Bettina?
Bettina M. Cockroft: Yes, absolutely. So you're absolutely right.
Bettina M. Cockroft: We mentioned this earlier today. What we're focusing on mainly, and you picked it up, is TX200, specifically where we are engaging with regulatory agencies, especially the various European ones, in the various countries that we are initiating our TX200 study in. So we are going through all those regulatory approval processes for that study, and that study is receiving a lot of feedback even during this pandemic. Many agencies are still working hard, but that's exactly what we're engaging in.
Alexander D. Macrae: And on a more general basis, but on a more general basis. And the agency, particularly the FDA, has been very understanding about that. Sangamo hasn't had to have those conversations, if that's what your question is.
Chew Vora: Got it. The last question, sorry. Sandy, you also mentioned that you may be considering other partnerships, partnership opportunities. Can you talk to the programs now in your current lineup that you think would be most appropriate for a partnership versus Sangamo taking them forward?
Alexander D. Macrae: So if you think of what happened with Biogen... There was one of those products, tau for Alzheimer's, that we've been speaking to you all for a while about. One, sinuclein, that we'd spoken about for three months, and the other ten were things that... None of us knew who were in this Sangamo pipeline. There are things that we could partner with that you are aware of, and we have no plans at the moment to further partner our existing assets in the pipeline that you think of as Sangamos. But there are also many other targets within the genome, even within the CNS space, that are not part of our current go-ahead portfolio but that others are interested in.
Chew Vora: Got it. Super helpful. Thanks for taking all the questions.
Operator: Thank you very much. Thank you.
Jim Bertinow: Thank you. Our next question comes from Jim Bertinow of Wells Fargo Securities. Your line is open.
Yanan Zhu: Hi, thanks for taking the questions. This is Yanan dialing in for Jim.
Alexander D. Macrae: So first, a question on the Alzheimer's program in collaboration with Biogen. I was wondering what might be the optimal delivery route for that program, whether it's IV or intraparenchymal injections. And also, secondarily, is there a milestone, the first milestone that you think you will be communicating around? So just wondering the timeline, for example, the nomination of a candidate or the first IND. So any sense of when that might be?
Alexander D. Macrae: So, a bit like what we've said with Pfizer, it will be Biogen to make this communication, but I wonder if Adrienne could comment on routes of delivery and Stephan on milestones. So Adrienne, perhaps first.
Adrian Wolfson: Yeah, thanks Sandy, and thanks for the question. We, as Stanley says, we haven't disclosed the route of delivery, and we'll leave it to Bhajan to do that, but I will just say, for example, if you look at, you'll be following ASGCT this week, but we'll be talking about some of our attempts to derive new serotypes using directive evolution and so on in our captured libraries. So we've got an active program doing that, and obviously anything that comes out of those programs will be relevant to the work at Bhajan, but it's fair to say that we're considering a range of different delivery routes, but at this point, I wouldn't want to be more specific than that.
[music].
Adrian Wolfson: And that's the presentation by David Ujala.
Adrian Wolfson: Yes, that's right, Sandy. Well, we make these captured libraries with different peptide insertions and insertion sites using a range of different parenteral serotypes and Stefano Bozzi.
Stephan Boissel: Yeah. Yeah.
Stephan Boissel: Stefan, how is the contract constructed?
Stephan Boissel: So, yes, and we closed the Biogen deal only a few weeks ago, and we had the onboarding of the collaboration only a few days ago. So... We need to be a little patient for the collaboration to really get started for the steering committee to meet before we can make any update as to the timeline on the value product we are going to work on with our friends at Biogen.
Stephan Boissel: But the first startup meeting was very successful here.
Stephan Boissel: Oh, it was. And despite a very bizarre or unique setting, because it was totally virtual, and we had something like 80 people, a combination of Biogenome, a Sangamo employee on the phone, and Zoom as well. And it was, yeah, a real success, despite, again, a kind of bizarre setting to start with.
Yanan Zhu: Thank you, that's very helpful. And then just two quick questions regarding the Fabry disease program. Just wondering what your competitive positioning is against other AAV Fabry gene therapy approaches? And what's your impression from the initial biomarker data regarding enzyme that was presented? and whether you think in your, sorry, whether in your low-dose cohort, starting those cohorts, you think you can, you know, similar or higher, in terms of environment level.
Alexander D. Macrae: So can I just clarify your question? In the second part of this, are you saying, who are you comparing Freeline or Acrobio to?
Yanan Zhu: Freeline, AAV, yeah, Freeline.
Alexander D. Macrae: So let me see if I can answer both of these. None of us can claim that we have something different until we get into the clinic and see the results in patients, and therefore that's why we're going to wait and complete the study at all three doses. I don't think it would be helpful for us to compare our low dose versus Freeline's low dose because all that matters is the final dose that you take into the clinic. We are pleased with the design of our study, we're pleased with the animal results, and we're looking forward to showing clinical results. And as Bettina stressed, this study is about safety, so we will be looking at liver enzymes, we'll be looking at cardiac enzymes, we'll be making sure that this is as safe as possible, because it's only when you have that that you can then start to compare efficacy.
Yanan Zhu: Got it, yeah, that's very helpful. Thank you, Sandy.
Damien Dupre: Thank you. I'm sure there are no further questions at this time. I'd like to turn the call back over to Damien Dupre for any closing remarks.
[music].
Damien Dupre: Thank you all for your interest. Thank you all for doing this virtually. We are very fortunate at Sangamo to be able to proceed with a virtual team and our lab staff in the laboratories, and we look forward to sharing more results with you as the year progresses.
Operator: Ladies and gentlemen, this does conclude today's conference. Thank you for participating, and we will all disconnect. Have a great day.
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