Q2 2019 Earnings Call
Operator: Today's conference will begin momentarily, and until that time, your lines will again be placed on hold. Once again, this is the Operator. Today's conference will begin momentarily. Until the time, your lines will again be placed on hold. Thank you for your patience.
Until that time your lines will again be placed on hold.
Once again this is the operator today's conference will begin momentarily.
Until that time your lines for the Gambia based on coal and thank you for your patience.
BF-WATCH TV: BF-WATCH TV 2021
Operator: Good afternoon, and welcome to Editas Medicine's second quarter 2019 conference call. All participants are now in listen-only mode. There will be a question and answer session at the end of this call. Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Mark Mulliken, Vice President of Finance and Investor Relations at Editas Medicine. Please go ahead.
All participants are now in listen only mode.
There will be a question and answer session at the end of this call.
Please be advised that this call is being recorded at the company's request I would now like to turn the call over to Mark Mckechnie, Vice President of Finance and Investor Relations at Editas Medicine. Please go ahead.
Thank you operator, good afternoon, everyone and welcome to our second quarter 2019 conference call.
Mark Mulliken: Thank you, operator. Good afternoon, everyone, and welcome to our second quarter 2019 conference call. Shortly after the market closed, we issued a press release providing our financial results and corporate updates for the second quarter of 2019. A replay of today's call will be available on the investors and media section of our website approximately two hours after its completion.
Shortly after the market closed we issued a press release, providing our financial results and corporate update for the second quarter of 2019.
A replay of today's call will be available on the investors and media section of our website approximately two hours after its completion.
After our prepared remarks, we will open the call for acuity.
Mark Mulliken: After our prepared remarks, we will open the call for Q&A. As a reminder, various remarks that we make during this call about the company's future expectations, plans, and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the risk factors section of our most recent quarterly report on Form 10-Q, which is on file with the FCC. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements, even if our views change. Now, I will turn the call over to our newly appointed Chief Executive Officer, Cindy Collin.
As a reminder, various remarks that we make during this call about the company's future expectations plans and prospects constitute forward looking statements for purposes of the safe Harbor provision under the private Securities Litigation Reform Act of 1995.
Actual results may differ materially from those indicated by these forward looking statements as a result of various important factors, including those discussed in the risk factor section of our most recent quarterly report on Form 10-Q , which is on file with the SEC.
In addition, any forward looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date.
We specifically disclaim any obligation to update or revise any forward looking statements. Even if our views change now I will turn the call over to our newly appointed Chief Executive Officer, Cindy Collins.
Thanks, Mark Good afternoon, everyone and thank you for joining us for second quarter 2019 corporate update.
Cindy Collin: Thanks, Mark. Good afternoon, everyone, and thank you for joining us for our second quarter 2019 corporate update. In addition to Mark, I am joined today by several members of our executive team, including Charlie Albright, our Chief Scientific Officer. Vic Meier, Chief Technology Officer; Erick X, Interim Chief Financial Officer; and Tim Hunt, our Senior Vice President of Corporate Affairs. As we announced this afternoon, the board ran a comprehensive process over the past several months focused on identifying the right leader to take Editas into its next chapter, accelerate our achievements, and focus on our EM22 goals. The board's search was run by the Nominating and Governance Committee with assistance from leading executive search firms. Overall, the board interviewed many candidates and was focused on executives with strong industry, scientific, and development expertise, a proven ability to grow and scale companies, and a passion for driving long-term success for Editas and patients.
In addition to Mark I am joined today by several members of our executive team, including Charlie Albright, Our Chief Scientific Officer, Vic Myer, Our Chief Technology Officer.
Eric our interim Chief Financial Officer, and Tim Holland, Our senior Vice President of corporate Affairs.
As we announced this afternoon. The board ran a comprehensive process over the past several months focused on identifying the right leader to take out of China into its next chapter accelerate our achievements and focus on our E M 22 goals.
The board search was run by the nominating and governance committee with assistance from leading executive search firm.
Overall, the board interviewed many candidates and let's focus on executives with strong industry scientific and development expertise and proven ability to grow in scale companies and a passion for driving long term success at Atossa and patients.
As a veteran of the gene and cell medicine industry I knew at Atossa, well, which is why jumped at the chance to join the Editas Sports last December .
Cindy Collin: As a veteran of the gene and cell medicine industry, I knew Editas well, which is why I jumped at the chance to join the Editas board last December. I believe that this company has an extraordinary opportunity to develop durable medicines that can dramatically transform patients' lives in ways never imagined. And as interim CEO, my enthusiasm has grown as I have dug in and learned more about the people, programs, and capabilities of the company. That is why I am pleased and humbled to accept the Board of Directors
I believe that this company has an extraordinary opportunity to develop durable medicines that can dramatically transform patients lives in ways never imagined.
And as interim CEO my enthusiasm has grown as I have dug in and learn more about the people programs and capabilities of the company.
That is why I am pleased and humbled to accept the board's appointment as a permanent CEO .
Cindy Collin: We've had great momentum in 2019, and we are determined to build on that and establish Editas as the clear leader in the gene editing space. We have been driving programs, executing business development deals, hiring and building teams, and strengthening our Inspiritas culture. I am proud of what our team has accomplished, and I am excited to update you on our latest progress. I will hit on a few of the highlights and then hand it over to Charlie and Erick to discuss in further detail.
We've had great momentum in 2019, and we are determined to build on that and established at a time as the clear leader in the gene editing space, we've been driving programs executing business development deals hiring and building teams and strengthening our inferior tops culture.
I am proud of what our team has accomplished and I am excited to update you on our latest progress I will hit on a few of the highlights and then hand, it over to Charlie and Erik to discuss in further detail.
First in partnership with Allergan, we have initiated patient screening in our phase one two clinical trial with at it when I won for Leber congenital ammar amaurosis can or LCH again.
Cindy Collin: First, in partnership with Allergan, we have initiated patient screening in our Phase 1-2 clinical trial with EDIT-101 for Leber Congenital Amaurosis-10, or LCA-10, and we are on track to dose patients in the second half, as previously communicated. This is a thrilling moment in the history of medicine, as we are on the cusp of treating people for the first time ever with an in vivo CRISPR medicine, as well as a first for Editas. Second, beyond EDIT 101, we are making strong progress on our broader pipeline of in vivo CRISPR medicine. Starting with another ocular disease, Usher Syndrome 2A.
And we are on track to dose patients in the second half as previously communicated.
This is a thrilling moments in the history of medicine as we are on the cost of treating people for the first time ever with an in vivo CRISPR medicine as well as a first for at a tough medicine.
Second beyond at at one on one we're making strong progress on our broader pipeline of in vivo, Chris for medicine, starting with another ocular disease Usher syndrome to a this program is de risked and accelerated by our work on Ed at one on one and we expect to be ready for I.N.D., enabling studies by the end of the year.
Cindy Collin: This program is de-risked and accelerated by our work on Edit 101, and we expect to be ready for IND-enabling studies by the end of the year. Third, we continue to advance our engineered cell medicines. Our confidence in EDIT301 as a potential best-in-class medicine for sickle cell disease and beta thalassemia continues to build, and our team is making tangible headway on its development of engineered cell medicines for cancer, including universal allogeneic cell medicine. Finally, we are investing in the manufacturing infrastructure required to develop and commercialize CRISPR medicines, including manufacturing capacity at our site in Boulder, Colorado. Now, I will hand the call over to our Chief Scientific Officer, Charlie Albright.
Third we continue to advance our engineered cell medicines, our confidence in editorial one as a potential best in class medicine for sickle cell disease, and beta thalassemia continues to build.
And our team is making tangible headway on its development of engineered cell medicines for cancer, including Universal Allogeneic cell medicines.
Finally, we are investing in manufacturing infrastructure required to develop and commercialize Christopher medicines, including manufacturing capacity at our site in Boulder, Colorado.
Now I will hand, the call over to our Chief Scientific Officer, Charlie Albright to update you further on the pipeline.
Charlie Albright: Thanks, Cindy. And thanks to everyone on the call for joining us today. I'll start with our In Vivo CRISPR medicines, beginning with our lead candidate, Edit 101 for LCA10. As Cindy mentioned, we have initiated patient screening for our Phase 1-2 interventional study called Brilliance in collaboration with Allergan, and we remain on track to begin dosing in the second half of this year. Brilliance is an open-label, dose-escalation study to evaluate the safety, tolerability, and efficacy of EDIT101 in approximately 18 states. The site at Mass Eye and Ear is currently recruiting, and we plan to open three more sites in the weeks ahead.
Thanks, Andy and thanks to everyone on the call for joining us today.
I'll start with our first in vivo CRISPR I'll start with our in vivo Christopher medicines, beginning with our lead candidate at one and one for LC Tim.
As Cindy mentioned, we've initiated patient screening for our phase one to interventional study called brilliance in collaboration with Allergan, we remain on track to begin dosing in the second half of this year.
Brilliance is an open label dose escalation study to evaluate the safety tolerability and efficacy of at a one on one and approximately 18 patients.
The site at mass or in the Air is currently recruiting and we plan to open three more sites in the weeks ahead.
We also expect the first patients in the trial will come from our natural history study.
Charlie Albright: We also expect the first patients in the trial will come from our natural history study, which enrolled patients with very similar enrollment criteria to the interventional study. You will recall that accelerating the interventional study was one of the objectives of the Natural History Study. For our second in vivo program, we are developing an experimental medicine for patients with Usher Syndrome 2A. Like LCA-10, Usher 2A is an inherited retinal disease that affects the photoreceptors. S2A is caused by a mutation in the gene encoding usherin, a protein essential for phototransduction in the retina. The most common mutation in the S2A gene is a single nucleotide deletion in exon 13, causing a frameshift that results in a truncated, non-functional protein. Our therapeutic approach uses CRISPR gene editing to remove the exon containing the mutation and thereby create a functional protein. Our collaborators from Massachusetts
Which enrolled patients with very similar enrollment criteria to the interventional study.
You will recall that accelerating the interventional study was one of your objectives in the natural history study.
For a second in vivo program, we are developing an experimental medicine for patients with us or syndrome two way.
Like L.C. 10 US two way is an inherited retinal disease that affects the photo receptors.
Oh, it's two way is caused by mutation in the gene encoding Usher in a protein essential for photo transduction in the retina and the most common mutation in the us to a dream is a single nucleotide deletion, Exxon 13, causing a frame ship that results in a truncated non functional protein.
Our therapeutic approaches CRISPR gene editing to remove the exon containing the mutation and thereby create a functional protein.
Our collaborators from outside Air showed preclinical proof of concept for this approach at the recent American Society of gene and cell therapy Conference.
Charlie Albright: Pioneer showed preclinical proof-of-concept for this approach at the recent American Society of Gene and Cell Therapy conference, and we are currently completing preclinical studies for our lead molecule. This medicine will share many features with that of WAN and WAN-C, including the AAV serotype, promoter, and proprietary Staph aureus Cas9. We anticipate having a molecule for S2A patients that is ready for IND-enabling studies by the end of the year. Finally, we're working on additional ocular programs, and we'll speak further about these programs in the coming months. These programs will illustrate the power of our platform to raise in vivo editing. Transitioning to engineered cell medicines, the second pillar of our therapeutic strategy, we're developing EDIT301 to be a best-in-class medicine for sickle cell disease and beta thalassemia.
We are currently completing preclinical studies for our lead molecule.
This medicine will sure. Many features that a one on one including the Avi Sera type promoter and proprietary staph aureus casnine.
We anticipate having a molecule for us to a patients that is ready for R&D, enabling studies by the end of the year.
Finally, we're working on additional onto their programs and will speak further about these programs in the coming months. These programs will illustrate the power of our platform through really couldn't vivo editing medicines.
Transitioning to engineered cell medicines, the second pillar of our therapeutic strategy.
We're developing at a real one to be a best in class medicine for sickle cell disease and beta thalassemia.
This program uses gene editing to induce fetal hemoglobin expression to treat hemoglobinopathies.
Charlie Albright: This program uses gene editing to induce fetal hemoglobin expression to treat hemoglobinopathy. As a reminder, our therapy involves targeting the beta-globin locus and hematopoietic stem cells. This therapeutic approach is differentiated from other approaches which go after the BC-11A erythroid enhancer, which only indirectly upregulates fetal hemoglobin. Our findings suggest adding the BC-11A enhancer may impact stem cell differentiation and ultimately the number and viability of edited red blood cells in vivo. We have found that adding the beta-globin locus, on the other hand, had no detrimental effect on rethroid output while inducing robust fetal hemoglobin.
As a reminder, our therapy involves targeting the beta globin locus of amount of poetic stem cells.
This therapeutic approach is differentiated from other approaches which go after the BC 11, a a resort enhancer, which only indirectly up regulates fetal hemoglobin.
Our findings suggest adding the VC 11, enhancer may impact stem cell differentiation and ultimately the number and viability of edited red blood cells in vivo, we have found that adding the beta globin locus on the other hand said no detrimental effect on a reported output while inducing robust fetal hemoglobin.
Hi, Andy enabling activities are underway, we plan to present additional data demonstrating the features and benefits of editorial one at a medical conference later this year.
Charlie Albright: I&D enabling activities are underway, and we plan to present additional data demonstrating the features and benefits of EDIT3-01 at a medical conference later this year. Due to our confidence in this program, we are building manufacturing capacity at our site in Boulder, Colorado. In particular, we intend to use this facility to supply guide RNA and ribonucleic protein for preclinical and clinical studies with edit 301, as well as other engineered cell medicine oncology programs. We expect this facility to be commissioned next year.
Due to our confidence in this program, we are building manufacturing capacity at our site in Boulder, Colorado.
Particular, we intend to use this facility to supply God or in a ribonucleic protein for preclinical and clinical studies within a three a one as well as other engineered cell medicine oncology programs.
We expect this site to be commissioned next year.
Beyond our work on Hemoglobinopathies, we are investing broadly to develop engineered cell medicines on oncology.
Erick J. Lucera: Beyond our work on hemoglobinopathies, we are investing broadly to develop engineered cell medicines and oncology. We have a long-standing partnership with Celgene to develop engineered T-cell medicines, and in parallel, we are advancing wholly-owned efforts to develop engineered NK cells as potential therapies for cancer. Last quarter, we announced a collaboration with Blue Rock Therapeutics to develop cell medicines for oncology using induced pluripotent stem cells, or iPSCs. Engineered Cell Medicines from IPSC may offer several potential advantages, including the ability to make a highly engineered cell with total control of the genome and thereby create a truly off-the-shelf medicine. We believe these features will be essential to unlock the potential of cell medicines for solid tumors in areas of high and met need.
We have a long standing partnership with Celgene to develop engineered T cell medicines and in parallel we are advancing wholly own efforts to develop engineered NK cells as potential therapies for cancer.
Last quarter, we announced the collaboration with Blue rock therapeutics to develop sell medicines for oncology using induced pluripotent stem cells or IPO Steve.
And then they are sell medicines from IPO see may offer several potential advantages, including the ability to make a highly engineered cell with total control the genome and thereby create a truly off the shelf medicine.
We believe these features will be essential to unlock the potential of sell medicines for solid tumors in area of high unmet need.
On current encouraged by the rapid progress, we're making with made in just the past few months.
Erick J. Lucera: I'm encouraged by the rapid progress we've made in just the past few months. First, we successfully generated iPSCs from fibroblasts and peripheral blood cells. Second, we've achieved efficient editing of iPSCs. And third, we have differentiated iPSCs into functional NK cells with potent killing activity. This progress puts us on a good path to develop highly engineered, off-the-shelf NK medicines that address the high unmet need for patients with solid tumors. I will have more to say about these results at upcoming scientific meetings. In conclusion, our team continues to make strong headway against both in vivo crystal medicines and engineered cell medicine. Now, let me turn the call over to Erick to provide a business and financial update.
First we have successfully generate IPO proceeds from fibroblasts and peripheral blood cells.
Second we've achieved sufficient editing of IPO Cie and third we have differentiated IP season, the functional NK cells with potent killing activity.
This progress puts us on a good path to develop highly engineered off the shelf NK medicines that address the high unmet need for patients with solid tumors.
Well have more to say about these results at upcoming scientific meetings.
In conclusion, our team continues to make strong headway against both in in vivo, Chris for medicines and engineered cell medicines now, let me turn the call over to Eric to provide a business and financial update.
Thanks, Charlie I'm pleased to update all of you on our Companys financial results.
Erick J. Lucera: Thanks, Charlie.
Erick J. Lucera: I'm pleased.
Erick J. Lucera: I'm pleased to update all of you on our company's financial results. These numbers are summarized in the press release that we issued about an hour ago, and full details will also be available in our forum.
These numbers are summarized in the press release that we issued about an hour ago and full details will also be available in our Form 10-Q .
Our cash cash equivalents and marketable securities decreased $24 million in the second quarter to approximately $318 million as of June Thirtyth 2019 from approximately $342 million as of March 30, Onest 2019, our uses of cash totaled $32 million and include cash operating expenses of $31 million and capital expenditures of $1 million.
Erick J. Lucera: Our cash, cash equivalents, and marketable securities decreased $24 million in the second quarter to approximately $318 million as of June 30, 2019, from approximately $342 million as of March 31, 2019. Our uses of cash totaled $32 million and included cash operating expenses of $31 million and capital expenditures of $1 million. Key non-cash items recorded in our income statement include $6 million of stock-based compensation and $1 million of depreciation. Our sources of cash include $2 million of stock option exercises and $1 million of interest income. We believe our cash, cash equivalents, and marketable securities of $318 million as of June 30, 2019 provide at least 24 months of capital to fund our business. And with that, I will hand it back to Cindy.
Key noncash items recorded in our income statement includes $6 million of stock based compensation and $1 million of depreciation our sources of cash include $2 million of stock option exercises and $1 million of interest income.
We believe our cash cash equivalents.
And.
Marketable securities of $318 million as of June Thirtyth 2019 provides at least 24 months of capital to fund our business and with that I will hand, it back to Cindy.
Thank you Eric before we close I'd like to make a few comments on intellectual property and organizational development.
Cindy Collin: Thank you, Eric. Before we close, I'd like to make a few comments on intellectual property and organizational development. In early June, the U.S. Patent Office declared a second interference between certain patent applications submitted by the University of California Group and certain patents, Patents issued to the Broad Institute, which are exclusively licensed to Editas. We have the utmost confidence in our intellectual property and expect these issued patents to be confirmed and upheld in this proceeding. On the organizational side, Executive Searches remain active to bring on an excellent new CFO and Chief Medical Officer
In early June the US patent office declared a second interference between certain patent applications submitted by the University of California group and certain patents.
Patents issued to the broad Institute, which are exclusively license to Editas medicines, we have the utmost confidence in our intellectual property and expect these issued patents to be confirmed and upheld and that is proceeding.
On the organizational side executive searches remain active to bring on an excellent news CFO and Chief Medical Officer, We look forward to updating you at an appropriate time.
Cindy Collin: We look forward to updating you at an appropriate time. In closing, we are making history with the first ever treatment of patients with an in vivo CRISPR medicine. Our pipeline is advancing to translate CRISPR gene editing into genomic medicines across a wide range of serious diseases from hemoglobinopathies to cancers to inherited blindness. And we are further enhancing our end-to-end capability to bring medicines to patients by building manufacturing capacity to reliably supply CRISPR medicines. With that, we thank all of you for your interest and support and are happy to take your questions. Operator?
In closing, we're making history with the first ever treatment of patients with an in vivo Christopher medicine.
Our pipeline is advancing to translate CRISPR gene editing and to genomic medicines across a wide range of serious diseases from hemoglobinopathies cancers to inherited blindness, and we are further enhancing our end to end capability to bring medicines to patients by building manufacturing capacity to reliably supply Chris for medicines.
With that we thank all of you for your interest and support and are happy to take your questions operator.
Thank you ladies and gentlemen, if you have a question at this time. Please press Star then the number one on your Touchtone telephone.
Operator: Ladies and gentlemen, if you have a question at this time, please press star then the number one on your touchtone telephone. If your question has been answered and you wish to remove yourself from the queue, press the pound key. Our first question comes from the line of Gina Wang from Barclays. Gina, your line is open. Our next question comes from the line of Mattie Harrison from Morgan Stanley.
If your question has been answered and you.
Wish to remove yourself from the queue pressed advances.
[noise]. Our first question comes from the line of Gena Wang from Barclays.
[noise].
Oh Gee now your line is open.
[noise].
Our next question comes from the line of Matthew Harrison from Morgan Stanley .
Hello. This is cost us on for Matthew I have two questions. The first one is how do you expect to report the data from the brilliant study given that this is an open label study will you.
Charlie Albright: Hello, this is Kostas Son on Matthew's behalf. I have two questions. The first one is, how do you expect to report the data from the BRILLIANT study given that this is an open-label study? Will you announce data from a few patients first, or will you wait until the study is completed before you present data?
Announce data from few basins fast, though to your we'll wait until the studies are completed before you present data.
Hi, This is Charlie so.
Charlie Albright: Hi, this is Charlie. So number one, we're really excited to get the study going. And as we said, this is the first ever in vivo CRISPR editing, and how we report the data will very much depend on the circumstances. And so we will be able to see the data in real time, we will, but we want to be careful about the accuracy of our conclusions, and we'll make that ultimate call in collaboration with Allergan, our key collaborators in this area.
Number one we're really excited to get this started going in as we as we said this is the first ever in vivo CRISPR editing.
And how we report the data will will very much depend on the circumstances and so we will be able to see the data in real time, we will but we want to be careful about how they act the accuracy of our conclusions and we'll we'll make that ultimate call in collaboration with our with Alexander our T. collaborators in this area.
Okay. Thank you and one more if I can get detailed what do you need to complete a eni and de filing for editorial one.
Charlie Albright: Okay, thank you. And one more. Can you detail what you need to complete an IND filing for ADE-301?
Is it's a it's all the usual stuff and so we need to to scale up manufacturing Sleepy I'd, enabling Tox studies.
Charlie Albright: It's all the usual stuff, and so we need to scale up manufacturing and complete the I&E-enabling talks.
And do you know approximately how long will these stake.
Charlie Albright: And do you know approximately how long it will take?
No we have not disclosed timelines, rather threea, one yet stay tuned, though you'll see more data on the program ended up coming a scientific meeting, which I think you'll find quite interesting.
Charlie Albright: No, we have not disclosed timelines for EDIT 301 yet. Stay tuned, though; you'll see more data on the program at an upcoming scientific meeting, which I think you'll find quite interesting.
Okay. Thank you.
Charlie Albright: Okay, thank you.
Our next question comes from the line of Cory Kasimov from JP Morgan.
Cindy Collin: Our next question comes from the line of Corey Casimo from TP Morgan.
Hi, This is gavin on for Cory So I extend my congrats to Cynthia and then he'll climate and then we just had to maybe a follow up on the first question on and at 101 disclosure strategy. Tom do you guys see any potential headwinds from the pending deal with a partner and then just as a follow on to the Usher syndrome. What are the gating factors that remain for the I. IND, enabling studies. Thank you.
Cindy Collin: Hi, this is Gavin speaking on behalf of Corey. Congratulations to Cynthia on the new appointment. And then we just had to maybe follow up on the first question on Edit 101, the disclosure strategy. Do you guys see any potential, you know, headwinds from the pending deal with a partner? And then just a follow-up question on Usher syndrome: What are the gating factors that remain for the IND enabling studies?
Cindy Collin: Thank you.
Cindy Collin: I didn't quite understand the first question about headwinds. I mean, we have a partner, and we will disclose it in collaboration with them. Are you talking about the AVI merger?
I didn't quite understand the first question about headwinds I mean, we have a partner and we will we will disclose in collaboration with them are you talking about if you're talking about the abbvie merger.
Correct, Yeah is the disclosure on on.
Cindy Collin: Correct, yeah, but is the disclosure on, you know, Allergan's side?
Our collagen side or.
Right now alligators alligators, our partner right now and so that deal has obviously not closed and and until it does abbvie is not a factor in any decision making so.
Cindy Collin: Right now, Allergan is our partner right now, and so that deal is obviously not closed, and until it does, AbbVie is not a factor in any decision making. So it's business as usual very much for all of the ophthalmology programs that are partnered with Allergan, and so we've got our head down and trying to deliver the portfolio. We aim to be ready for IND-enabling activities by the end of the year, so we have a variety of preclinical pharmacology studies going on now and are completing some other studies to get us in a position to have the package we need to proceed with IND-enabling studies.
It's business as usual very much on all of the ophthalmology programs that are partner with al again, and so we're we've got our head down and trying to deliver the portfolio.
Ostracism true to anywhere or we aim to be ready Friday, enabling activities by the end of the year. So we have a variety of preclinical pharmacology studies going on now and completing some other some other studies and get us in a position to to have the kind of the package we need to proceed into the R&D, enabling studies.
Great. Thanks for the clarity.
Cindy Collin: Great, thanks for the clarification.
Our next question comes from the line of Keith feed House from Merrill Lynch.
Charlie Albright: Our next question comes from the line of Steve Seedhouse from Raymond James.
Charlie Albright: Yes, hi, this is Timur Ivannikov speaking on behalf of Steve Seedhouse, and we would like to follow up on the patent interference question. So, what is your estimate of the legal costs involved? And also, what is your view of the accusations being made by Berkeley regarding Zhang willfully making inaccurate statements? And finally, what would be an ideal resolution if the other side wanted to come to the table and negotiate?
Yes, Hi, this is on Timur Ivannikov on for Steve seat House, and they would like to follow up on the patent interference question. So what is your estimate of the legal costs involved and also what is your view of the acquisitions being made by Berkeley regarding Shang willfully, making an accurate statements and finally, what would be an ideal resolution if the other side wanted to come to the table and negotiate thank you.
[noise] so first.
Cindy Collin: So, first of all, we're not going to speculate on what the overall cost of the litigation might be nor make any comment about the accusations in the investigation.
First of all we are not going to speculate on what the the overall cost of the litigation might be no or make any comment about accusation and the and inventors and in your last question I apologize was.
Cindy Collin: And your last question, I apologize, was... Well, what would be a nice thing?
Well what would be an ideal resolution you know if the other side wanted to come to the table and negotiate what would be an ideal situation.
Cindy Collin: Well, what would be an ideal solution, you know? If the other side wanted to come to the table and negotiate, what would be an ideal situation?
[noise].
So I think an ideal situation for us would be that week, we continue to prevail with the intellectual property position that we have which we feel very confident around.
Cindy Collin: So I think an ideal situation for us would be that we continue to prevail with the intellectual property position that we have, which we feel very confident about, particularly as it relates to Cas9 and CPF1.
As particularly as it relates to to Casnine and CPF one.
Okay. Thank you and then you also have a follow up on on the brilliance of study.
Cindy Collin: Okay, thank you. And then we also have a follow-up to the brilliance study. I think in the past, you've talked about preliminary design.
Thank god in the past you've talked about a preliminary design I don't know if that's still the case, but I've seen two patients in the first cohort I don't know if that's still the plan for you and.
Charlie Albright: I don't know if that's still the case, but I've seen two patients in the first cohort. I don't know if that's, you know, still the plan for you. And, you know, what they're going to be dosed with a low dose, and then and then you will escalate to a hard dose.
You know, what they're going to be to dose with low dose and then and then you will escalate to hard dose. So the question is I mean is it still too patients or you know or have you changed have you made any changes and also also is the second patients going to be younger than the first one.
Charlie Albright: So the question is, I mean, are there still two patients? Or, you know, have you changed? Have you made any changes? And also, is the second patient going to be younger than the first one? If you can disclose, thank you.
If you can disclose thank you.
Sure and so obviously safety is a paramount concern. So two of the main objectives are safety and Tolerability and the third objective is Africa c. So.
Charlie Albright: Sure. And so obviously, safety is a paramount concern. So two of the main objectives are safety and tolerability, and the third objective is efficacy. So obviously, the right thing to do is to put the risk-benefit very much in our favor as we start the trial.
Obviously, the right thing to do is to put the risk benefit very much in our favor as we start the trial. So we will start the trial in adults that have very poor vision.
Charlie Albright: So we will start the trial in adults that have very poor vision, and there will be two patients dosed at a low dose. The dose that we think will be pharmacologically active but on the low end of the range of pharmacologic activity. And then, based on the results from those two patients, we will escalate the dose, and eventually, we will move into adults that have better vision, and based on those results, we'll eventually move into, ideally, children that have poor vision and then better vision. Because, a priori, you would expect children with some vision to have the most benefit from this therapy, but that's obviously not the right place to start this trial from a risk-benefit standpoint.
And there will be two patients dosed at the at the low dose or doses. We think can be will be pharmacologically active but on the low end of the range of pharmacologic activity and then based on the results from those two patients we will escalate the dose and eventually we will escalate or we will move into adulthood have better vision and based on those results will eventually move into ideally children that have poor vision, and then better vision because a priority you would expect or children with some business to have the most benefit from this therapy, but that's obviously not the right place to start this trial from a risk benefit standpoint.
Okay. Thank you very much.
Charlie Albright: Okay, thank you very much.
Our next question comes from the line of Amanda Murphy from Keith.
Cindy Collin: Our next question comes from the line of Amanda Murphy from BTIG.
Thank you and congrats Cindy I guess I had a question for use in Egypt and since you've been there now for.
Cindy Collin: Thank you. And congrats, Cindy. I guess I had a question for you, Cindy, just since you've been there now for several months. Just curious if you now have some context and are thinking about your broader strategy and EM 22, etc. Any tweaks that you're thinking about making, whether you have already or going forward, and obviously, the company is changing somewhat going from a preclinical, you know, entity into a clinical entity. So I'm assuming there's some resource hiring, things like that. But just, just as curious if you know, now that you've had a year behind you, or I guess, almost a year, what your perception is now?
Several months I'm just curious if you.
Now you have some contacts and thinking about your broader strategy in EM and 22 et cetera.
Any tweaks that you're thinking about making whether you have already or going forward and obviously the company is changing somewhat going into at from a preclinical at you know entity into a clinical antisense I mean, there seems to be sort of hiring things like that but just just curious if you you know now that you've had a year behind you or I guess almost a year like your first actually accounts.
Thanks Amanda.
I know it's been about six months that are maybe it feels like a year [laughter]. So we remain very committed to our current M. 22 strategy and to our 2019 corporate goal and you are correctly are referring to the fact that the company is now moving into a clinical phase which is different for US we'll continue to hire a the required resources that we need to support that growth as we scale, we talked a little bit about some of our efforts in manufacturing and our Boulder facility and we'll just continue along that trajectory for for the near term.
Cindy Collin: It's been about six months, but maybe it feels like a year.
Cindy Collin: ..
Cindy Collin: So we remain very committed to our current EM22 strategy and to our 2019 corporate goals, and you are correctly referring to the fact that the company is now moving into a clinical phase, which is different for us. We'll continue to hire the required resources that we need to support that growth as we scale. We talked a little bit about some of our efforts in manufacturing at our Boulder facility, and we'll just continue along that trajectory for the near term.
Okay got it and then just one on the wholly owned and quayside.
Charlie Albright: Okay, I got it. And then just one on the wholly owned NK side of the world, both, I guess, considering NK cells and iPSC-derived NK cells. I know you said solid tumor, and this may be too early to ask you, but just curious about your strategy there going forward with the view. Would you focus more on, you know, maybe starting out in some more well-validated targets but ones that are a little more crowded competitively? I know there's not that much in a solid tumor, really, but or would you kind of go? You know, I'm thinking high level about how you might approach from an education perspective.
The world.
But I guess, considering and can't I can't see derived NK cells.
I know you said solid tumor and this may be too early to ask you, but just curious about your strategy there going forward. What did you see when you focus more on you know maybe starting out into more well validated targets, but ones that are a little more crowded competitively I know, there's not that much in solid tumor really but or would you kind of go.
You know I I was thinking high level, how you might approach from an education perspective.
Charlie Albright: Sure, this is Charlie, Amanda, and we obviously haven't disclosed the details of where we're going. But I think, you can draw some parallels from How We Treated Other Disease Areas and be assured that we'll have a thoughtful approach to the indications we pursue in that area to develop both differentiated and transformative medicines.
Sure. This is Charlie Amanda the job. So we obviously haven't disclose the details of where we're going I think.
I think Ah.
You can draw some parallels from from how we treated other disease areas and.
And I'm sure that we'll have a thoughtful approach to the syndications, we be pursuing that area to develop both differentiated and transformative medicines.
Okay and I just had one last one the I P. S. C side, it seems like that IP landscape is.
Charlie Albright: Okay, and I just had one last one on the IPSC side. Seems like that IP landscape is almost as complicated as CRISPR. Not quite.
It's almost it's complicated it's chris or not quite but is there anything to factor in there I guess as we think about your opportunities do you feel like you have freedom to operate broadly there or is there anything from a restriction standpoint to think about.
Charlie Albright: But is there anything to factor in there as we think about your opportunities? Do you feel like you have freedom to operate broadly there, or is there anything from a restriction standpoint to think about?
We feel very good about our position there and are are are very enthusiastic because it's hopefully you gathered about advancing those medicines.
Charlie Albright: We feel very good about our position there and are very enthusiastic, as hopefully you have gathered, about advancing it.
Charlie Albright: Okay, thank you.
Yep.
Okay. Thanks very much.
Cindy Collin: Okay, thanks very much.
Our next question comes from the line of Peter Lawson from Suntrust Robinson.
Charlie Albright: Our next question comes from the line of Peter Lawson from SunTrust Robinson.
[noise] people since here just wanted to offer my congratulations on the.
Charlie Albright: Hey Cynthia, I just wanted to offer my congratulations on the role. Maybe a question for Charlie, just how should we think about the timing for entering the clinic for beta cell and sickle cell, and would you start beta first or sickle first, and can we use timing from peers as an example there?
On the road, maybe a question for Charlie just how should we think about the timing for entering the clinic for beta sounds sickle cell and would you stop beta first sickle first.
We use I guess timing from P. is.
As an example, there.
Oh, Yeah. That's good thanks, Peter the job Yeah, I think the the precedents in this field are numerous and and it is totally fair to use the existing.
Charlie Albright: Yeah, that's good. Thanks, Peter.
Charlie Albright: Yeah, I think the precedents in this field are numerous, and it's totally fair to use the existing trials as a guidepost. We haven't disclosed our strategy on sickle cell versus beta-thal and U.S. versus Europe either, both key strategic decisions as we advance what we feel is a really exciting medicine and look forward to talking a lot more about it later this year.
Trials as a guide post we.
We haven't disclosed our our strategy on sickle cell versus beta Sal in U.S. versus Europe , either both key to some strategic decisions that we as we advance what we feel the really exciting medicine and look forward to really talking a lot more about it later this year.
And then just the L.C. 10, just considering.
Charlie Albright: And then just on LCA-10, considering the initial patients are probably the most difficult to treat, do you think you ought to have a therapeutic dose initially for those patients? How long do you think we have to wait until we kind of see that?
The patient the initial patients are probably the most difficult to treat do you think you would have a therapeutic dose initially for those patients who.
How long do you think we have to wait until we can see that.
Well, we we believe that you will see benefit in a in a small number of months and we've gone through that.
Charlie Albright: Well, we believe that you'll see benefit in a small number of months, and we've gone through the scientific rationale for that because it's based on preclinical pharmacology. And the doses are entirely based on preclinical pharmacology as well. So we did studies in mice and non-human primates, which gave a very similar relationship between dose and Cas9 levels and editing.
Through the scientific rationale for that is because it's based on the preclinical pharmacology and the doses are entirely based on the preclinical pharmacology as well so we did.
We did studies in mice, and non human primates, which give a very similar relationship between dose in kasbar levels and editing and so we feel as good as one can feel at this point about dose selection and that's been the basis for how we've chosen the starting dose because we do believe that those he has the potential to have therapeutic benefit and but obviously, that's that's why we do the trials to understand what's going on there and where we want to be conservative at the same time, we're trying to understand the ever going to see the first understand safety and Tolerability.
Charlie Albright: And so we feel as good as one can feel at this point about dose selection. And that's been the basis for how we've chosen the starting dose because we do believe at that dose you have the potential to have therapeutic benefit. But obviously, that's why we do the trials to understand what's going on there, and we want to be conservative at the same time. We're trying to understand the efficacy to first understand safety and tolerability.
Great. Okay. Thanks for taking the questions congratulations.
Charlie Albright: Great. Okay. Thanks for taking the questions. Congratulations.
Our next question comes from the line of Kenneth Atkins from Cowen and company.
Charlie Albright: Our next question comes from the line of Kenneth Atkins from Cohen & Company.
Charlie Albright: Hi, thanks for taking my questions. Can you comment on the pacing of patient dosing in the EDIT-101 trial? How soon after the first patient is dosed can you dose the second patient, and so on?
Hi, Thanks for taking my questions.
Can you comment on the pacing of patient dosing in the edit one on one trial how soon after the first patient dose can you does the second patient and so on.
[noise] Yeah. This is Charlie again, so we haven't disclosed the details it's a very typical design, though based on gene therapy, and so you're correct that there will be an interval between between all the patients actually and and and that fits with the that fits with the priorities of the study to and to ensure safety and Tolerability before we expose additional patients to the drug.
Charlie Albright: Yeah, this is Charlie again. So we haven't disclosed the details. It's a very typical design that's based on gene therapy. And so you're correct that there will be an interval between all the patients, actually. And that fits with the priorities of the study to ensure safety and tolerability before we expose additional patients to the drug. Got it.
Got it Okay, and then aside from safety and Tolerability what.
Charlie Albright: Okay, and then aside from safety and tolerability, what level of improvement in visual acuity and visual function navigation do you think would be meaningful for patients, and would that differ depending on the patient's age or their disease severity?
Level of improvement on visual acuity and visual function navigation do you think would be meaningful for patients and and would that differ depending on the patients age are there to be disease severity.
[laughter].
Charlie Albright: Um, I think that SPARC provides a reasonable precedent for what's considered a clinically meaningful benefit in patients in general, and clearly, a major objective of the trial is to understand the benefit we can deliver with our medicine.
I think did you think that the.
The spar provides a reasonable precedent and once considered I mean clinically meaningful benefit in patients in general the.
And clearly a major objective is the trough of the trials to understand the benefit we can deliver with our medicine.
Charlie Albright: Got it, okay, that's helpful, thanks.
Got it okay. That's helpful. Thanks.
Oh.
Our next question comes from the line it would be I am from Guggenheim Securities.
Charlie Albright: Our next question comes from the line at Whitney AM from Guggenheim Security. Hi, this is Anmita on behalf of Bikini AGM.
Hi, this is on muscle on public need Jim. Thanks for taking my question first so as you look towards expanding the utility I feel in vivo CRISPR technology can you talk about any other work ongoing progress made on the delivery side and also how do you think about that getting muscle analog ano DMD and CF program. Thanks.
Charlie Albright: Thanks for taking my questions first. So as you work towards expanding the utility of your in vivo CRISPR technology, can you talk about any of the work ongoing or progress made on the delivery side? And also, how do you think about targeting muscle and lung in your DMD and CF programs? Thanks.
[laughter].
Charlie Albright: Thanks, Charlie. We are very excited to move into other tissues, and one of the big benefits we get out of the EDIT101 is experience in delivering a complete package that is suitable for regulators in this space. And so this does set us up to go into other tissues where AAV has been proven to be a good delivery vehicle. And, of course, you realize we're the only company that actually has the ability to deliver gene editing in a single AAV because of our access to Staph aureus Cas9. So we do want to take advantage of that unique capability and move into other in vivo tissues. We do think that the eye was a great place to start, but we look forward to talking more about the other places we're going to go.
Thanks, Charlie the thumb, we are very excited to move into other other tissues and and.
One of the one of the big benefits, we get out of the other one I want his experience in delivering.
A complete package that was a suitable to regulators in this space.
And so this does set us up to go in the other tissues, where Avi has been proven to be a good delivery vehicle and then of course you realize worthy.
Were the only company that actually has the ability to deliver and as gene editing and a single in a single Avi because of our access to the staff Oreos Casnine. So we do want to take advantage of that unique capability and move into other in vivo tissues. We do think that the I was a great a great place to start.
But we look forward to talking more about the other places we're going to go.
[noise].
Once again, ladies and gentlemen, if you have a question. Please press star one now on your telephone keypad.
Operator: Once again, ladies and gentlemen, if you have a question, please press star one now on your telephone keypad. Our next question comes from the line of Sylvan Kirken from Oppenheimer.
Our next question comes from the line of Sylvan Turkish from open Heiner.
Charlie Albright: Thank you for taking my question and congratulations on the new role, Cynthia. My question is, could you please, I looked up the trial on clinicaltrials.gov, the brilliance trial, and there's a middle, low middle, and high dose in adults and a middle dose and a high dose in pediatrics. Would that middle dose be the same as in adults for children?
Thank you for taking my question.
Congratulations.
Cynthia.
My question is could you.
I I looked up the trial on clinical trials that go off the brilliance trial, and so there's a middle low middle and high dose than adults in the middle dose and a high dose in pediatrics without middle dose to be the same as in adults for children.
Yes, the and it turns out that the.
Charlie Albright: Yes, and it turns out that the eye is not all that different in size as you grow, and so the same dose is totally appropriate.
The the the eye is not a lot different itself has as you as you grow and so the same dose is totally appropriate.
Charlie Albright: Okay, great. And could you please remind us of the endpoints that you have in the natural history study for the LCA-10, and could that be kind of viewed as a control? I mean, these patients don't get continuously better, but to kind of quantify the clinical benefit at the end.
Okay, Great and could you. Please remind us of the endpoints that you have from the natural history study for the L.C., a 10 and could that be kind of viewed as a control I mean I mean these these patients still gets contains a better but to kind of quantify the clinical benefit at the end.
Charlie Albright: Yeah, so we're looking at a variety of potential endpoints in the natural history study. All the usual suspects, best corrected visual acuity, a variety of imaging endpoints, which could provide substantial evidence that we actually have the functional benefit that we aim to achieve, as well as a mobility maze, which is a more stringent test of the functional ability of these patients. So part of the whole point of that natural history study was to get familiarity with these endpoints and understand which would be most operationally and functionally relevant for this patient population. And we will be looking at several of those endpoints in the intervention study.
Yeah. So we're looking at a variety of potential endpoints in the natural history study all the all the usual suspects so best corrected visual acuity a variety of imaging endpoints, which can provide a substantial evidence that we actually had the functional benefit that we aim to achieve as well as the mobility maze, which used to be a more stringent test the functional ability of these patients. So part of the whole point of that natural history study was to get familiar parity with these end points and understand which would be most operationally and and functionally relevant for this patient population.
And we will be looking at several of those endpoints in the interventional study.
And so my last question could we get that data before you won't disclose any efficacy data.
Charlie Albright: And, sorry, my last question: could we get that data before you disclose any efficacy data from the Brilliant trial, or do you think that will be after?
Trial or do you think that will be after.
Weve disclose some of that data at that Oh, This past years ARVO meeting.
Charlie Albright: We've disclosed some of that data at this past year's ARVA meeting, and so we disclosed baseline data and that that should be available on our website.
And so we disclose baseline data and that that should be available on our on our web site.
Charlie Albright: Great! Thank you so much.
Great. Thank you so much.
Cindy Collin: We have no further questions at this time. I will now turn the call over to Ms. Cindy Collins.
We have done further question at this time I will now turn the call over back to Ms. Cindy Collins.
Great. So with that we thank all of you for participating on today's call and for your support as we work to bring transformative new medicines to patients.
Cindy Collin: Great. So, with that, we thank all of you for participating in today's call and for your support as we work to bring transformative new medicines to Baytown. Have a great evening.
Have a great evening.
Ladies and gentlemen, thank you for joining US. This concludes today's conference call you may now disconnect.
[noise].
[noise].
[noise].
Operator: Ladies and gentlemen, thank you for joining us. This concludes today's conference call. You may now disconnect.
[noise].
Operator: ??? ??? ??? ???