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Adverum Biotechnologies, Inc. (ADVM -5.81%)
Q2 2019 Earnings Call
Aug. 08, 2019, 4:30 p.m. ET
Contents:
- Prepared Remarks
- Questions and Answers
- Call Participants
Prepared Remarks:
Operator
Good afternoon, and welcome to the Adverum Biotechnologies Second Quarter 2019 Conference Call. [Operator Instructions]
I would now like to hand the call over to my Myesha Lacy, Vice President of Investor Relations and Corporate Communications of Adverum. Please go ahead.
Myesha Lacy -- Vice President of Investor Relations and Corporate Communications
Thank you and welcome, everyone, to our second quarter 2019 conference call. The press release reporting our financial results is available on the press release page of the Investor Relations section of our corporate website at www.adverum.com. Please note that a replay of today's call also will be available on the Events and Presentations section of our website.
Joining me for the prepared remarks portion of the call today is Leone Patterson, Chief Executive Officer; and Dr. Aaron Osborne, Chief Medical Officer. Then, Leone, Aaron and Chief Financial Officer, Thomas Leung will be available for the Q&A portion of the call.
As a reminder, we will be making forward-looking statements regarding our product development plans, research activities and operations as well as our financial outlook. These statements are subject to risks and uncertainties that may cause actual results to differ materially from those forecasted. A description of these risks can be found in our most recent Form 10-Q on file with the SEC.
I would now like to turn the call over to our CEO, Leone Patterson.
Leone Patterson -- Chief Executive Officer and Director
Thank you, Myesha. Good afternoon, everyone, and thank you for joining us today. At Adverum, everything we do is with focus on our goal to discover, develop and deliver novel gene therapies to make an impact in the lives of patients living with serious ocular and rare diseases. We believe that gene therapy can be transformative for patients, and this is why we are laser focused on developing ADVM-022 as a single intravitreal injection approach to treating VEGF-driven retinal diseases.
In addition to developing, ADVM-022 for wet AMD, we have announced today that we plan to expand the development of ADVM-022 for the treatment of diabetic retinopathy. During this call, we will highlight the work we are doing to advance our development pipeline, including outlining upcoming milestones. Then Aaron will provide an update on our clinical progress with ADVM-022 before we take questions.
This year, we have achieved significant clinical and regulatory milestones with our lead product candidate. As background, the first indication we are targeting, wet AMD, is a leading cause of vision loss in patients 60 years and older. Approximately 1.2 million individuals in the US are impacted by wet AMD, and this number is expected to grow as the country's population ages. Our gene therapy, ADVM-022, uses the standard of care, intravitreal injection, and is a potentially less frequent, more durable treatment option that we believe could be game changing for patients with this serious ocular disease. I will now highlight the great progress we have made with the Phase 1 OPTIC clinical trial for wet AMD.
Starting with cohort 1, we have completed dosing and look forward to presenting 24-week data on all six patients at the Retina Society annual meeting in London on September 12. Additionally, we announced in June that we commenced dosing patients in the second cohort of the Phase 1 OPTIC trial, and I'm pleased to share that we have already completed dosing all six patients in this cohort. We expect to present 24-week data from the second cohort as well as longer-term 52-week data from the first cohort at a scientific meeting in the first half of 2020.
Further, we will provide an update on our development plans for the OPTIC trial in the fourth quarter of this year. These are important upcoming milestones for this program. In addition to wet AMD, we believe there are opportunities for ADVM-022 to treat patients with other optic diseases that currently are treated with anti-VEGF therapies. As the next indication for ADVM-022, we are targeting diabetic retinopathy. Diabetic retinopathy is a leading cause of vision impairment and blindness among working-age adults, and the prevalence of diabetes and diabetic retinopathy are growing rapidly. These younger, working-age adults could benefit greatly from a onetime intravitreal injection gene therapy approach. And we plan to submit an IND for this indication in the first half of 2020.
Now turning to our rare disease programs. We have discontinued development of our gene therapy program targeting A1AT deficiency. Additionally, we have moved our gene therapy program targeting hereditary angioedema back into early development. Our immediate priority continues to be focused on developing our lead product candidate, ADVM-022 for wet AMD and diabetic retinopathy.
As we progress our clinical programs, we are scaling up our manufacturing process to support the product requirements for later-stage development and commercial use. Our new corporate headquarters in Redwood City will allow for the expansion of in-house process development capabilities to 1,000-liter scale, and we expected to be able to occupy our new facility by the end of this year. Before turning the call over to Aaron, I would like to talk about our corporate update that we announced last week.
First, Board member and Co-Founder, Mitch Finer, has retired from the Board of Directors to focus on a new industry role. We also announced that our President and Chief Scientific Officer, Mehdi Gasmi, will retire from his management position on September 16 and will transition to the Adverum Board of Directors. We look forward to Mehdi's continued engagement as a Board member, and we will initiate a broad effort to identify a new CSO.
Looking forward, this is a pivotal time for the company. We continue to expand our depth of talent with recent senior management additions in a number of key functional areas. I'm excited to work with this team as we pursue our mission of delivering novel gene therapies to patients with ocular and rare diseases.
I'd now like to introduce our CMO, Aaron Osborne, to provide an update on ADVM-022's clinical development. Aaron joined our team in April. And as an ophthalmologist and biotechnology industry veteran, he brings extensive experience in clinical practice, drug development and global medical affairs. He has been at the forefront of many exciting therapeutic developments for patients in the field of ophthalmology and has executed Phase 2 and Phase 3 studies in wet AMD and diabetic eye disease. Aaron's breadth of experience has been a fantastic addition to the team. I'm thrilled to be working with them.
Aaron Osborne -- Chief Medical Officer
Thank you, Leone, and good afternoon, everyone. As an ophthalmologist who has spent much of my career developing treatments for retinal diseases, in particular anti-VEGF therapies, I'm excited to lead the clinical development of ADVM-022.
There is a robust body of long-term preclinical data which supported advancing ADVM-022, a onetime intravitreal injection gene therapy that has significant potential to improve real-world vision outcomes for patients with wet AMD into the clinic. The current standard of care treatment is intravitreal injections with anti-VEGF therapy oftentimes requiring frequent injections for several years to maintain vision.
Around 50% of patients need injections every four to eight weeks to control their wet AMD disease activity, and this is a major burden for patients, caregivers and healthcare systems. This need for frequent injections results in undertreatment and vision loss for many patients. ADVM-022 has the potential to deliver sustained efficacy and maintain vision with a single standard-of-care intravitreal injection and could be transformative for patients living with wet AMD or other VEGF-driven retinal disorders.
Turning to the Phase 1 OPTIC Trial of ADVM-022 in wet AMD. As Leone mentioned, the first clinical data will be presented during a podium presentation at The Retina Society annual meeting on September 12 in London. Dr. Szilard Kiss, the Director of Clinical Research and Associate Professor of Ophthalmology at Weill Cornell Medical College, will present 24-week primary and secondary outcomes data from the first cohort of six patients. These patients, who had previously required frequent injections for their wet AMD, all had received at least two anti-VEGF injections in the four months prior to enrolling in OPTIC and were given a single intravitreal injection of ADVM-022 at a dose of 6x10 to 11 vector genomes per eye at baseline. Recall that the OPTIC Trial as a Phase 1, first-in-human study, safety is the primary endpoint. As we have previously shared, no SAEs and no DLTs have been observed in this first cohort of patients. As the primary objective of OPTIC, we will be presenting 24-week safety and tolerability outcomes for this cohort of patients.
We have also previously shared that in the first cohort of OPTIC, we saw a robust preliminary anatomical response, which led to our decision to use the lower dose of 2x10 to 11 vector genomes per eye for the second cohort. The following secondary endpoints are important to help us assess both anatomically and functionally the potential benefits from a single intravitreal injection of ADVM-022. We are assessing patients' vision, a change in best corrected visual acuity. In trials of patients with wet AMD who have not previously been treated, we would expect to see improvements from baseline in vision. However, in this trial, which is the patients who have been receiving ongoing treatment with frequent anti-VEGF injections to preserve their vision, a successful outcome is to maintain visual acuity with fewer injections.
Another secondary endpoint is change in central retinal thickness at 24 weeks as measured by Optical Coherence Tomography or OCT. Anatomical assessment on OCT is an important and objective measure of wet AMD disease activity. Similar to BCVA, because patients have already been treated previously with anti-VEGF therapy, a successful quantitative outcome on OCT would be to see maintained central retinal thickness through 24 weeks. OCT also gives us the ability to qualitatively view and assess the presence of pathological fluid in different compartments of the retina to assess the effectiveness of the therapy. Overall, we are looking for stability on the OCT. And if we see OCT improvements, this would represent decreased wet AMD disease activity.
The most important secondary data point in OPTIC for many retina specialists and patients will be the percentage of patients requiring anti-VEGF injections and the mean number of anti-VEGF rescue injections. With 24-week follow-up on all six patients, we will be able to assess the potential of ADVM-022 to reduce treatment burden. As is the standard approach in this type of clinical trial, there are protocol-defined rescue injection criteria based on OCT and vision. Overall, we are looking for stability on vision and OCT with a minimal number of rescue treatments being required in these patients who had previously required frequent intravitreal injections. Beyond the first cohort, we continue to make strong progress in the OPTIC Trial. As Leone mentioned, we recently completed dosing the six patients in the second cohort at a lower dose of 2x10 to 11 vector genomes per eye. We plan to present a 24-week data from the second cohort of the OPTIC Trial at a scientific meeting in the first half of 2020.
The OPTIC Trial aims to establish ADVM-022's potential in wet AMD. The next ocular VEGF-driven disease we are pursuing with this therapy is in diabetic retinopathy. Diabetic retinopathy is the result of microvascular damage to the blood vessels in the retina caused by raised blood sugar and occurs frequently in both type 1 and type 2 diabetics. Of the estimated 8 million people with diabetic retinopathy in the US, only 2 million are diagnosed and only 1 million are being treated.
Diabetic retinopathy is the leading cause of vision loss among working-age adults in the US. Anti-VEGF therapy can effectively treat diabetic macular edema and can reduce the risk of other sight-threatening events overall in diabetic retinopathy. However, the short duration of effect of currently available anti-VEGF therapies limits their use because when their effect wears off, diabetic retinopathy can quickly progress and may cause irreversible sight loss. Therefore, we believe there is a significant unmet need in diabetic retinopathy for a long-lasting intravitreal anti-VEGF therapy. We are working on clinical development plans for ADVM-022 in diabetic retinopathy and plan to submit an IND to the FDA in the first half of 2020.
I will now turn the call back over to our CEO, Leone Patterson. Leone?
Leone Patterson -- Chief Executive Officer and Director
Thank you, Aaron. I want to add some further details around our plan for presenting 24-week OPTIC data for the first cohort of six patients at The Retina Society annual meeting in London. Although this is a closed meeting, we plan to share the OPTIC data transparently and broadly. We will issue a press release at the beginning of the podium presentation, followed by a conference call and webcast. A copy of the slide presentation will be available on our corporate website after the call.
With that, we will now open the call to questions. Operator?
Questions and Answers:
Operator
Thank you, ma'am. [Operator Instructions] Our first question comes from the line of Tyler Van Buren of Piper Jaffray. Your line is open.
Tyler Van Buren -- Piper Jaffray -- Analyst
Hey, guys, good afternoon and thanks for taking the questions. I thought you did a pretty good job of explaining the OPTIC trial design and the expectations on visual acuity and central retinal thickness and to maintain it. I was hoping that you could provide a little bit more clarity on the types of patients that are being enrolled. You mentioned that they have at least two injections in the four months prior to the OPTIC. So if they're being treated with EYLEA, what's the possibility that some of these patients would be dry or stable through those six months just due to the simple fact that they've had EYLEA prior to the study?
Aaron Osborne -- Chief Medical Officer
Thank you for the question. It's Aaron Osborne here. So yeah, in the protocol, we stipulate the patients must have had two injections, at least two injections in the previous four months. So these are patients that are requiring frequent anti-VEGF injections. We've gone to pains to ensure that, that is within the protocol. And, I mean, if you kind of extend that, that would equate to obviously injections every other month. So as we said, around 50% of patients or more would require anti-VEGF injections at eight or four weekly intervals. So really, we were looking to recruit that type of population in OPTIC. And what we can also do is look at the previous treatment history, look at the intervals between those injections. And as I said, we've really tried hard to include a population that requires frequent ongoing anti-VEGF injections.
They have the EYLEA injection in the protocol to show that they can respond to EYLEA because EYLEA is aflibercept and ADVM-022 is producing the protein aflibercept on a sustained basis. So given that we're presenting 24-week data and given that the patients previously had required injections at least really every two months based on the protocol inclusion criteria, we believe that over the 24 weeks that, that is significantly longer than one could possibly expect from the screening EYLEA injection. So I think that should summarize your answer that we're defining by protocol patients that require frequent anti-VEGF injections. And with 24-week follow-up on all patients, we should have a very good idea as to the potential for ADVM-022 to reduce those number of injections.
Tyler Van Buren -- Piper Jaffray -- Analyst
Yeah, that's helpful. And in terms of rescue injections, you mentioned that it was based on OCT and vision, which makes sense. So can you just speak a little bit more about how stringent that criteria is such that someone doesn't accidently provide a rescue injection when maybe the patient is still relatively stable? And also, what magnitude or what percent reduction in rescue injections implied over those first six months would you view as a win?
Aaron Osborne -- Chief Medical Officer
Yes. So, I mean, clearly, the fewer rescue injections that we see will be the better. I mean, that's the feedback that we've had from retina specialists, our investigators and in our research. So the lower number of rescue injections, really the better.
If we look at the criteria for rescue injections, these are really similar to other clinical trials in that they're based primarily on OCT and vision. And essentially, without going into the specifics of the numbers and the thresholds, if patients experience an increase of fluid on the OCT or their vision drops as a result of what the investigator believes is wet AMD disease activity, then essentially they're eligible for a rescue injection. I mean the challenge with wet AMD is it's an aggressive disease. If fluid accumulates and vision drops and you don't treat that, then there's the possibility that the patient may irreversibly lose vision. So our rescue criteria are actually very stringent, and if a patient experiences increasing fluids or drop in vision due to wet AMD disease activity, then they would receive a rescue injection.
Tyler Van Buren -- Piper Jaffray -- Analyst
Okay. And just final question on the prophylactic oral steroid dosing and tapering. Can you just speak toward why you guys used that in the study? Is it due to anticipation of just general inflammation that you would see or that you've seen historically with gene therapy that you would expect to resolve over 13 days? And do you think that could go down with a lower dosing or that you could potentially move to topical steroid dosing over time?
Aaron Osborne -- Chief Medical Officer
Yes. So absolutely. I mean an immune response following any form of gene therapy is something that is expected. And that's the reason why -- if you'll recall, this is obviously a first-in-human study as well, so we're giving the patients a tapering dose of prednisone. They have six days of 60 milligrams and then it tapers off. And that's -- yes, specifically because there is an immune response expected.
Regarding further plans for the OPTIC study, currently, as we've mentioned, we've enrolled the second cohort, and we'll be looking at whether we add additional cohorts and what the dose might be and whether we adjust the steroid regimen because potentially, that's certainly something that we would be considering, whether we could move to topical therapy as well. So at the moment, really we're waiting for a little more data from the second cohort, and we will plan to update later on this year.
Tyler Van Buren -- Piper Jaffray -- Analyst
Thanks very much for taking the questions.
Operator
Thank you. Our next question comes from Phil Nadeau of Cowen and Company. Your line is open.
Phil Nadeau -- Cowen and Company -- Analyst
Greeting and thanks for taking my questions. A follow-up to Tyler's last question there on the information for the anti-VEGF injections. Physicians have little tolerance for any sort of inflammation in the eye. What's your sense for less frequently administered anti-VEGF gene therapy? How much inflammation would be acceptable to the physicians in your opinion?
Aaron Osborne -- Chief Medical Officer
Yes. I mean I think I would come back to the point that some of these patients are having very frequent intravitreal injections and saving injections has got that potential to improve real-world vision outcomes as well as avoiding an unpleasant procedure, which carries risks such as endophthalmitis even, which, although rare, when you're having many, many injections, those risks can increase. So I think everybody would expect that there will be some immune response following a gene therapy.
But if you can get a long-lasting reduction in the requirement for rescue injections, then that's certainly a trade-off that people will be willing to look at. So I think it really -- we look forward to presenting the data. I think on September 12, we'll be able to provide a much fuller picture of the primary outcome measures, which is around safety and tolerability, as well as the secondary outcome measures around anatomical outcomes, around visual acuity and about the numbers of rescue injections that may be required.
Phil Nadeau -- Cowen and Company -- Analyst
Okay, great. That's helpful. And then second, on your decision to lower the dose. You mentioned that there was no DLT at the initial dose cohort. Can you talk about what was the criteria for dose-limiting toxicity --
Aaron Osborne -- Chief Medical Officer
Yeah, for sure.
Phil Nadeau -- Cowen and Company -- Analyst
And probably why you dose reduced?
Aaron Osborne -- Chief Medical Officer
Yeah, of course. So we have an independent data monitoring committee. And if you'll recall, the plan was to dose escalate following the first cohort. The IDMC, they met. They considered any adverse events that had occurred in the early period following treatment, and they unanimously recommended to escalate to the second dose as they were very comfortable with the safety profile there.
The decision to dose down was not based on safety. It was based on the fact that we saw a robust preliminary anatomic efficacy signal. And based on this, we did not feel that there would be value in increasing the dose because you'll recall a key aim of the Phase 1 trial is to dose range. So we wanted to establish the effectiveness of different doses. We saw this robust response. We did not see that there could be incremental anatomic efficacy with a higher dose, so we decided to dose down to see if we could achieve similar types of results except for the lower dose. And obviously, we've recruited that cohort now, and we're in the period after that. We're waiting to see the data, and we'll share our findings later in the year.
Phil Nadeau -- Cowen and Company -- Analyst
That's helpful. And then last question for me is just on rescue injections. Again, in an answer to Tyler's question, you had noted that the fewer injections, the better. But it does seem like -- just play devil's advocate. If you need rescue injections in a decent proportion of patients such that everyone who gets the gene therapy still has to be monitored on a regular schedule, the benefits of gene therapy are much diminished versus a cleaner profile where very few patients need rescue injections. What do you think of that argument? Is it accurate? Or do you think just reducing the treatment burden by some percentage would create a successfully -- a successful product?
Aaron Osborne -- Chief Medical Officer
So yes, I think it's a great question, and we've done research around this as well as obviously having many conversations with retinal specialists. So while any reduction or a significant reduction is deemed to be helpful, certainly the greater that reduction, the more beneficial. And I think that comes exactly back to the point that you made, which is can you predict which patients are going to require those rescue injections and how many they will be and what would be the subsequent monitoring schedule. So all of those play into it. And if you can't accurately predict these things, then obviously the lower the number and the fewer the number of patients that have these will make a very big difference to the uptake and usage. So as I said, I think that's why it does come back to lower is better. And really, the ideal outcome will be to have a minimal number of additional rescue injections.
Phil Nadeau -- Cowen and Company -- Analyst
That's perfect. Sorry, and just one last question on the orphan programs. What was the postmortem as to what went wrong with the A1AT program? Was it the vector or the transgene. Could you give us some sense of what the prognosis was or the diagnosis was?
Leone Patterson -- Chief Executive Officer and Director
Sure. This is Leone. So for the rare disease programs, as you know, we're using the same vector, rh10. Late last year, we had results in the A1AT program which we shared around not achieving the therapeutic levels and far below actually the expected levels we would need to have to be therapeutic. And since we were using the same vector for A1AT -- vector, A1AT, in HAE, that obviously there was a potential impact on the efficacy for HAE as a result of using the same vector.
We had spent some time over the last six months reviewing our possibilities of how we can continue to move forward in either of those programs. I think where we came out on that is A1AT, we believe, given the therapeutic levels you need to get to, that we don't believe that is a program that we will continue with. HAE, however, we have, as you saw, that back to early development, for us to continue to see if there's ways that we can explore to get to a better therapeutic level for HAE given the read-through potentially from where we got to on A1AT level, so we believe that's still worthwhile to proceed to see what's possible, but we didn't want to raise expectations that we're going to be entering the clinic anytime soon. So we wanted to make sure that people were clear that we were continuing to explore how we could move forward with that program but recognize that it's back in a fairly early development stage.
Phil Nadeau -- Cowen and Company -- Analyst
Perfect. Thanks for my taking questions. Congrats on the progress.
Leone Patterson -- Chief Executive Officer and Director
Thanks.
Operator
Thank you. Our next question comes from Joon Lee of SunTrust Robinson. Your line is open.
Joon Lee -- SunTrust Robinson -- Analyst
Hi. Thanks for the question. Can you talk about what you view as the right therapeutic range for anti-VEGF or aflibercept in wet AMD? Is that something that you will determine based strictly on clinical outcomes? Or will you at least in part determine that based on how much protein is produced from ADVM-022 and how that compares to a steady-state amount of IND? And I have a follow-up. Thank you.
Aaron Osborne -- Chief Medical Officer
Thank you. Thank you for the question. I think, as you know, we've got a robust and long-term preclinical data package where we did extensive aqueous, vitreous and retinal sampling for aflibercept levels. And I guess what the key finding was here that across a range of doses, that we saw levels that we deem to be therapeutic in each of those compartments up to and including 30-month follow-up. So I think we see very stable long-term therapeutic levels of expression of aflibercept in the preclinical models.
In the clinic, we have patients who already have the disease. They have wet AMD. And what really matters as we have effective treatments out there is whether we can see a clinical response. And as we've spoken about, that's going to be mainly on the OCT is the most accurate in small numbers of patients. And obviously, the vision is very important as well. And then that will ladder up to the numbers of rescue injections.
At the current stage in OPTIC, we are not sampling for protein levels, and we're relying fully on the clinical outcomes. And as we said, we're really looking forward to and excited about presenting those 24-week data across those key secondary endpoints on September 12 in London.
Joon Lee -- SunTrust Robinson -- Analyst
Okay. So we're looking forward to that as well. And the follow-up question is please correct me if I'm wrong, but my understanding is that some patients actually spontaneously recover from wet AMD. Can you discuss the natural history of wet AMD a bit and if this is a steadily progressive disease or if there are some fluctuations in the VEGF levels over the course of the disease process? Thank you.
Aaron Osborne -- Chief Medical Officer
Thank you. Yes. So of course, really good questions. So generally, wet AMD requires ongoing treatment for several years, and that's for the vast majority of patients. You may get around up to 10% of patients may improve and settle just with one or two injections, but that is sort of 10% maximum. Of the remaining, say, 90%, at least 50% of those would generally require anti-VEGF injections every four to eight weeks. The remaining sort of 40% may be able to extend beyond that to 12 weeks, and that's really with currently available therapies.
So I think really, when you look at the trials, also therapies in development, that seems to be pretty consistent, and I think that's really a factor of the -- of these bolus injections that have got relatively short half-life. So the reality of it is, is that in many patients with wet AMD, around 50% requires four to eight weekly injections on a long-term ongoing basis. And we've really taken a lot of care in the OPTIC Trial to make sure that we recruit that patient population that requires frequent ongoing injections but also that they're responsive to anti-VEGF therapy in order to kind of robustly assess the potential of 022.
Joon Lee -- SunTrust Robinson -- Analyst
So if this therapy does get approved by the FDA, what are some strategies that you're thinking about to make sure that you're only giving this onetime therapy that is not reversible to patients who will need it for the rest of their lives and not by accident give it to someone who might have otherwise recovered or might not need it indefinitely?
Aaron Osborne -- Chief Medical Officer
Yes. I mean I would come back to the sort of benefit-risk of anti-VEGF treatment in wet AMD. Really, the challenge with the current therapies is that they are underutilized because they require frequent treatments and patients lose vision. I think if you speak to retina specialists, they'd fully agree that the risks of undertreatment are greater than the risks of overtreatment.
So a therapy that's delivered with a onetime injection that can provide long-lasting anti-VEGF coverage is likely to be a very attractive therapeutic option across the entire patient spectrum. For wet AMD, it's sometimes very hard to predict those patients that may only require a very small number of injections. And as I said earlier on, that's a very small percentage as well. So a much bigger risk is that patients are undertreated, and that -- I think that's the biggest risk. Obviously, we're taking care in the trials to ensure that the patients we enroll are those that require frequent injections.
Joon Lee -- SunTrust Robinson -- Analyst
Thank you very much.
Operator
Thank you. Our final question is from Dane Leone of Raymond James.
Dane Leone -- Raymond James -- Analyst
For the updates and everything, and we're definitely looking forward to the OPTIC data. Just a few for me. So you made some interesting comments about how you think about the market for 022. And just wanted to help define that maybe a little bit more. So when you guys gave -- kind of the teaser that you've given about the study is that you've seen robust anatomical responses. And in the context of that, that's obviously a 24-week study with six patients. The goal that you've said is reduced or fewer injections, standard anti-VEGF therapy post dosing. I think classically, we've thought of the goal of maybe the gene therapy -- wet AMD gene therapy as making a patient absent of standard injections maybe over the course of 24 to 36 months.
Is that not the case, I guess? Because like you dose down in the second cohort based on anatomical responses but also said that coming into the study, these patients were relatively dry, and the goal is stability on OCT. So I'm trying to kind of bridge how you guys view the market versus maybe what some of us had as that longer-term view that, I guess, might not be right.
Aaron Osborne -- Chief Medical Officer
So let me say that those are some really good questions there. And obviously, we're going to share the full details of the patients at baseline and how they respond to treatment over 24 weeks at that interim data presentation at Retina Society. So there will be many more details there. But I think a couple of points in response there, and Leone may wish to add some things as well.
Firstly, this is actually a 104-week study. So it's a 2-year study. This is just the first presentation and it is interim at 24 weeks. So obviously, the aim is to reduce the numbers of injections not just to 40 -- 24 weeks but for a much longer period of time. And I think if you go back to our preclinical data package, we had shown very stable long-term protein expression out to 30 months. So there is -- we've clearly shown that potential preclinically, but this happens to be our first read-out clinically. So hopefully, that addresses the question somewhat. I don't know if any just --
Leone Patterson -- Chief Executive Officer and Director
I think the only thing is reminding you -- you said about the -- at the start of the study what's the baseline for patients. I think what we would remind you -- and you -- I think you were referring to the fact that they're getting IV injection at the time of starting the study. So I think we believe that the time period we have, up to 24 weeks, is a meaningful time point to start with. And then to Aaron's point, clearly we're going out to 104 weeks, and that will be also meaningful. And as what we stated is that we plan to give an update on these patients after 52 weeks, recognizing that this is a preliminary endpoint and, therefore, we will be giving an update on that when we get to that time point as well.
Dane Leone -- Raymond James -- Analyst
Okay. And then on something you actually just kind of alluded to. So one discussion point that I guess might be outside of the realm of the OPTIC data that you can elucidate it a little bit, the dosing down in the next cohort, right, has generated a lot of interesting questions, as we've spoken about before this call. And the predication of that has been on the anatomical responses that you've seen in the OPTIC study and there's been no safety issues. The -- I guess the question -- two questions. One is what's actually the goal of dosing down. And I don't -- you're kind of alluding to maybe reducing steroids. Is that the primary goal of dosing down? Or is there another goal of dosing down?
And two, how did you choose the threefold reduction in the vector? So you've alluded to like the stable nonhuman primate protein data. The data that we've seen would have been actually equivalent to the second dose cohort if you dosed up. Now on the second dose cohort where you dose down, you're actually at order of magnitude below the nonhuman primate data. So I guess what kind of made you comfortable about that change in magnitude away from the translational data? Thank you.
Aaron Osborne -- Chief Medical Officer
So yes, so they're really good questions. So in terms of the dosing downwards, as we said, that was due to the robust preliminary anatomic response that we observed. And, I mean, I think one thing to say here is that these are patients that require frequent injections in order to maintain their vision and that what we would want -- what we plan to do at The Retina Society presentation is provide additional detail on their previous treatments and also what their anatomical response had been to previous treatments. So really, when we talk about a robust preliminary anatomic response, we had that information available, and we were able to look at the timing of the injections and also look at the OCT outcome. So it's really on that basis that we could see that it was such a robust response.
Another purpose besides safety for Phase 1 is obviously dose ranging and trying to identify the best doses to take forward as well as to demonstrate to FDA and other regulators that you've looked at a broad range.
So when we look at the effect on OCT, we did not see that we could get additional effect. So we're looking to titrate efficacy and also tolerability. So if we had reached which is the point that we felt that we had reached that we could not see additional efficacy certainly not at an early time point that would help us make timely clinical development decisions, then really the question was could we get that same type of efficacy but with a lower dose. Because if you can do that, obviously that is preferable to providing a higher dose. And with regards to the number -- or to the reduction of those magnitudes, we went down threefold. So initially, our plan was to go up threefold, and really a threefold change in dose is common practice in gene therapy studies.
Dane Leone -- Raymond James -- Analyst
Okay. And just final one for me, I promise. And obviously, everyone's been very confused about this. But just for -- since it's a public call, can you just state whether or not anatomical -- robust anatomical response does or does not equal additional standard anti-VEGF injections? You said a number of times reduce the amount of -- fewer injections, reduce the amount of injections. Just to make it clear for what you've kind of been implying of, like, how you look at the world on OCT, does that mean a patient has not gotten a rescue injection? Or is that just looking specifically at OCT scans in isolation?
Aaron Osborne -- Chief Medical Officer
So the robust anatomical response was based on assessing the OCTs of these patients. And we look forward to presenting the full data on the 12th of September.
Dane Leone -- Raymond James -- Analyst
Excellent. Thank you so much.
Operator
I will now turn the call back to Adverum CEO, Leone Patterson.
Leone Patterson -- Chief Executive Officer and Director
Thank you, again for joining our call today. This is an exciting time of the Company, and I'd like to summarize the key upcoming milestones that were highlighted today.
We plan to see a 24-week data in September from the first cohort of patients in the OPTIC trial. We plan to provide an update on our development plans for the OPTIC trial in the fourth quarter of this year. We also plan to submit an IND in diabetic retinopathy in the first half of 2020. And lastly, we plan to present 52-week data from the fifth cohort and 24-week data from the second cohort of the OPTIC trial in the first half of 2020. We are very excited by our accomplishments so far, and I look forward to working with the Adverum team as we continue to execute on our mission of delivering novel gene therapies to patients with ocular and rare diseases.
In closing, I'd like to thank the patients, caregivers and retinal specialists for their participation in the OPTIC Trial. Additionally, I would also like to thank the Adverum employees for their commitment and for their continued dedication to our mission. Thank you for your time, and this concludes our call.
Operator
[Operator Closing Remarks]
Duration: 42 minutes
Call participants:
Myesha Lacy -- Vice President of Investor Relations and Corporate Communications
Leone Patterson -- Chief Executive Officer and Director
Aaron Osborne -- Chief Medical Officer
Tyler Van Buren -- Piper Jaffray -- Analyst
Phil Nadeau -- Cowen and Company -- Analyst
Joon Lee -- SunTrust Robinson -- Analyst
Dane Leone -- Raymond James -- Analyst
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