REGENXBIO Q3 2023 Earnings Call Transcript

Quartr
Provided by Quartr
November 8, 2023

There are 12 speakers on the call.

Operator

Good day and thank you for standing by. Welcome to REGENXBIO's Third Quarter 2023 Earnings Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer Please be advised that today's conference is being recorded. I would now like to hand the conference over to your host today, Rick Christmas, Chief Legal Officer.

Operator

Please go ahead.

Speaker 1

Good afternoon and thank you for joining us today. Earlier this afternoon, REGENXBIO released financial and operating results for the Q3 ended September 30, 2023. The The press release is available on our website at www.regenxbio.com. Today's conference call will include forward looking statements regarding Our financial outlook in addition to regulatory and product development plans. These forward looking statements are subject to risks And uncertainties that may cause actual results to differ from those forecasted and can be identified by words such as expect, performance and involve certain risks and uncertainties.

Speaker 1

These risks are described in the Risk Factors in the Management Discussion and Analysis section of REGENXBIO's Annual Report on Form 10 ks for the full year ended December 31, 2022 and comparable risk factors In sections of REGENXBIO's quarterly reports on Form 10 Q, which are on file with the Securities and Exchange Commission and available on the SEC's website. Any information we provide on this conference call is being provided only as of the date of this call, November 8, 2023, And we undertake no obligation to update any forward looking statements we may make on this call on account of new information, future events or otherwise. Please be advised that today's call is being recorded and webcast. In In addition, any unaudited or pro form a financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company. Actual results may differ materially.

Speaker 1

I would now like to turn the call over to Ken Mills, CEO of REGENXBIO. Ken?

Speaker 2

Thank you, Patrick. Good afternoon, everyone, and thanks for joining us. I'm pleased to begin today's call with an explanation Our updated strategic plans. Doctor. Steve McCullough, our Chief Medical Officer, will then provide an update on our clinical programs And Vincent Systah, our Chief Financial Officer, will provide an overview of financial results for the Q3 ended September 30, 2023.

Speaker 2

At the end of the call, we will be opening up the line for questions. At REGENXBIO, our mission is to improve lives Earlier today, we began work on a pipeline prioritization and corporate restructuring plan that will enable REGENXBIO To focus our capabilities and resources on large commercial opportunities where product candidates are differentiated, Can be expedited and support meaningful value generation soon and for the long term. I want to be clear about what this sharpened focus means moving forward. Our highest priority programs, Our ABBV RGX-three fourteen program for the treatment of wet age related macular degeneration and diabetic retinopathy Being developed in collaboration with AbbVie RGX-two zero two for the treatment of Duchenne and RGX-one hundred and twenty one Now in the last two months, we've experienced exciting progress from each of these programs. We've reported positive clinical data from investigational treatments from diabetic retinopathy and Duchenne, And we've held a very encouraging RMAT meeting with the FDA about expediting the BLA for the treatment for MPS II.

Speaker 2

These milestones demonstrate how our science is supporting avenues to accelerate the development of new gene therapies. And today, we're following that encouraging data and announcing updated strategic plans for REGENXBIO. We believe that there is a multibillion dollar potential for RGX-three fourteen as a single injection treatment To become the 1st in class gene therapy for wet AMD and the standard of care to treat and prevent the progression of diabetic retinopathy, Initial efficacy data from patients treated with RGX-two zero two is enabling us to accelerate this program. Duchenne is a market where there is a large unmet need for new therapies and that is capable of supporting multiple gene therapies And we believe RGX-two zero two has unique differentiating features that support its potential to be a best in class product. During a very constructive RMAT meeting with the FDA just recently in October, we received encouraging feedback in confirmed alignment with the FDA on key elements of an expedited BLA.

Speaker 2

So we remain on track to support a BLA filing in 2024 using the accelerated approval pathway RGX-one hundred and twenty one would be the first gene therapy treatment for MPS II. In today's challenging market, the ability to create value quickly and efficiently is critical. Importantly, These restructuring decisions extend our cash runway much deeper into 2025, allowing us to progress our pipeline to a number of key inflection points. These would include initiating and dosing the 1st pivotal trials for 314 using suprachoroidal delivery, Enrolling the pivotal program for RGX-two zero two program and completing performance qualification lots to support a planned BLA for RGX-two 2 zero two and also achieving the BLA approval for RGX-one hundred and twenty one and MPS II. Now it's worth noting that successfully achieving certain of these milestones between now and then would also trigger 100 of 1,000,000 of additional funds, Such as milestone payments from our collaboration partner AbbVie for initiating suprachoroidal pivotal trials, which these milestones represent a meaningful portion of the over $560,000,000 in development milestones Eligible through this partnership for us.

Speaker 2

And the potential receipt of a pediatric review voucher for approval of RGX-1 hundred and twenty one, Generally, we've been observing CRV sales are resulting in nearly $100,000,000 to BLA sponsors at the time of receipt. So our updated strategic plans are intended to generate significant value for shareholders as we ensure resources are allocated To our most valuable assets, to be able to accelerate the development of these assets and to extend our operational runway These additional non dilutive sources that I just highlighted, for instance, are not in our current runway guidance, and if received, would allow us to bridge to additional value creating milestones, such as more product approvals Now, I'll turn the call over to Steve, so he can review some of our clinical progress and guidance for the prioritized programs in greater detail. Steve?

Speaker 3

Thank you, Ken. I'll begin with 314, which is being developed in collaboration with AbbVie to treat wet AMD and diabetic retinopathy Via subretinal and suprachoroidal routes of administration. 314 utilizes our NAV AAV8 vector To deliver a gene encoding of therapeutic antibody fragment to inhibit VEGF. The anti VEGF market opportunity is poised to grow significantly as the as the population ages. 314 for the treatment of wet AMD via subretinal delivery is being evaluated in 2 ongoing pivotal trials, Atmosphere and Ascent, which are expected to enroll a total of 1200 patients in the U.

Speaker 3

S, Europe and Japan, Supporting the global development of the program with anticipated global regulatory submissions in late 2025 through the first half of twenty twenty six. We also have 2 ongoing Phase 2 trials that fall under our collaboration with AbbVie assessing in office suprachoroidal delivery of 314 for AB-eight is an active controlled dose escalation trial evaluating 314 for the treatment of wet AMD. We most recently presented safety data in July from Cohort 6 evaluating the dose level 3 that included short course prophylactic ocular steroids following administration of 314. The initial data presented continues to support the safety profile of 314 And highlighted the inclusion of short course prophylactic steroid eye drops, which resulted in 0 cases of intraocular inflammation. We plan to present full 6 month results from Cohorts 56 at the Hawaiian Eye and Retina Meeting in the beginning of 2024.

Speaker 3

ALTITUDE is an observation controlled dose escalation study of 314 suprachoroidal delivery for the treatment of Doctor. We're very excited about the opportunity in Doctor given the size of the market, which exceeds that of even wet AMD and because we believe this patient population can benefit the most from a potential one time gene therapy. At AAO this past weekend and on a call with retina physicians on Monday, we presented positive data from dose levels 12 cohorts At 1 year, patients at those dose levels did not receive prophylactic steroids before or after 314 administration. 314 was reported to be well tolerated at both dose levels. Onetime in office 314 injection Demonstrated clinically meaningful improvements in disease severity with reductions in vision threatening events.

Speaker 3

Importantly, 100% of the patients with baseline NPDR treated with dose level 2 achieved stable to improve disease severity. Moreover, dose level 2 in these patients reduced the risk of visioning threatening events by 89%. We are encouraged by this data and the potential of a one time in office injection for patients with diabetic retinopathy. Moving now to RGX-two zero two for the treatment of Duchenne. RGX-two zero two is a potential one time gene therapy for the treatment of Duchenne Being developed as a highly differentiated product designed to deliver a transgene for a novel microdystrophin That includes the functional elements of the C terminal domain found in naturally occurring dystrophin.

Speaker 3

RGX-two zero two is designed to support the delivery and targeted gene expression throughout skeletal and heart muscle Using our NAV AAV8 vector and a well characterized muscle specific promoter. Our AAV8 Capsid also represents an alternative for boys who may not be eligible for other AAV mediated microdystrophin therapies Due to the presence of pre existing neutralizing antibodies. At World Muscle held in October, We shared interim clinical data from the Phase III AFFINITY Duchenne trial. Initial results from the first two patients for whom results were available In the 4.4 year old patient, microdystrophin expression was measured to be 38.8% compared to control. A reduction from baseline in serum creatinine kinase or CK levels of 43% was Zipsider, supporting evidence of clinical improvement.

Speaker 3

The 10.6 year old patient had microdystrophin expression measurements of 11.1 percent compared to control and a reduction from baseline in serum CK levels of 44%. Microdystrophin expression was measured by Western blot with comparable results observed when measured by the LC MS method. Overall, the data showed that RGX-two zero two was well tolerated with no drug related serious adverse events And all three patients treated as of the time the data were presented. As Ken shared earlier, We recently held an RMAT meeting with the FDA for RGX-one hundred and twenty one for treatment of MPS II. I'm pleased to share that this was a very positive and We were able to align with the FDA on our manufacturing strategy, the adequacy of our safety database And confirmatory study design.

Speaker 3

Patients treated with RGX-one hundred and twenty one continue to do well on follow-up and we expect to share top line data from the Phase 1, 2, 3 campsite trial in the Q1 of 2024. While we are no longer moving forward with our RGX-one hundred and eleven, 181 and 301 rare neurodegenerative programs, We believe in the potential of these therapies and are committed to finding strategic alternatives for them, including potential partners We're leveraging public private partnerships. So to conclude, we have made significant progress with Data updates and trial progression across all programs in our pipeline as we continue working to advance Lastly, I'd like to thank the patients, families, clinicians and patient advocacy representatives who have been involved in and supported all of these trials. And with that, I turn the call over to Vit to review our financial guidance. Vit?

Speaker 4

Thank you, Steve. REGENXBIO ended the quarter On September 30, 2023 with cash, cash equivalents and marketable securities totaling $365,000,000 compared to $565,000,000 As of December 31, 2022, the decrease was primarily driven by cash used to fund operating activities During the 9 months ended September 30, 2023, R and D expenses were $58,000,000 For the Q3 of 2023 compared to $63,000,000 for the Q3 of 2022. The decrease was primarily attributable to an increase in development cost reimbursement From AbbVie under our eye care collaboration. With a strategic reprioritization plan And 15% workforce reduction announced earlier today, we expect to save at least $100,000,000 Over the next 2 years, we now expect the balance in cash, cash equivalents and marketable securities of $365,000,000 as of September 30, 2023 To fund our operations into the second half of twenty twenty five, this cash runway guidance is based on the company's Current operational plans and excludes the impact of any payments that may be received from AbbVie upon the achievement of development or commercial milestones under our 314 collaboration. With that, I will turn the call back to Ken To provide final thoughts, Ken?

Speaker 2

Thanks, Nit. As we look ahead, Our extended cash runway will now enable us to reach additional meaningful value driving milestones for our programs, which I've outlined. We also want to bring focus to the fact that we have a lot of important near term value driving catalysts expected at medical conference upcoming in the first half of twenty twenty four. This would include new 6 months data from recently dosed cohorts of the AVA trial for wet AMD suprachoroidal, additional dose level 1 and initial dose level 2 data from the ADFINITY Duchenne trial And top line data from the pivotal Phase 3 campsite trial. I want to reiterate that our updated strategic plans are intended to generate Significant value for our shareholders as we assure resources are allocated to our most valuable assets, to accelerating the development of these assets, And to extending our operational runway and to achieve more milestones that can unlock additional value, including access to non dilutive capital that we from the AbbVie partnership or other sources like anticipated sale of a PRV that could all of which extend our runway even further.

Speaker 2

With a renewed sense of focus, I expect that we'll continue to perform at a high level as we execute on our mission. And as a result of these updated strategic plans announced and implemented today, we believe REGENXBIO is well positioned for long term success. With that, I think we can now turn the call over for questions. Operator?

Operator

In the interest of time, we ask that you limit yourself to one question and rejoin the queue for any additional questions. Our first question will come from the line of Gena Wang with Barclays.

Speaker 5

Thank you for taking my questions. Okay, I will follow just One question rule. I know there are tons of questions you can ask. So one question I have is, what you can learn From Sarepta's experience in terms of clinical development for the DMD program and regarding primary endpoints Selection and also efficacy bar.

Speaker 2

Thanks, Gina. This is a good question. Obviously, we've Ourselves have been engaged in dialogue with regulators about RGX-two zero two and especially as we've been able to emerge with our own Gold data has been an important focus for us since World Muscle Society to think about how to accelerate the program And we've given guidance next year that we believe we have the ability to both dose select and start a pivotal phase program. I think that what we're seeing in the landscape of microdystrophin continues to be encouraging that it's clearly helping boys. I think it's Something that we viewed since we entered the spaces beyond a sort of increment, but something that in certain voice certainly is having More response than in others.

Speaker 2

We believe that the C terminal domain hypothesis for RGX-two zero two It's a meaningful differentiator and I think where it can be an important differentiator is on clinical function, the long term clinical function. We're very much engaged about how to design trials to accelerate and still continue to Borrow the endorsement in the use of micro dystrophin as a surrogate endpoint to support accelerated approval, think about how to further support that In a pivotal setting and even in a commercial setting with respect to looking at long term function. So I think that the domains that are part of The scoring that's used in Duchenne Boys, I think some people are starting to recognize both at the regulator level and at Clinical level that some of these domains are more reliable in terms of prognostic indicators for improvement. We're very much dialed into that and think that having an opportunity to have our own direct dialogue about our data and seeing the evolution of what's happening on the regulatory Kate, actually puts us in the best position to start executing next year on a really great package.

Speaker 5

Thank you.

Operator

Our next question comes from the line of Dane Leone With Raymond James.

Speaker 6

Thanks for taking my questions. One for me, and Glad I didn't have to be the DMD person. There's hope that obviously Altitude and AVA can Support moving into pivotal studies during 2024. 1, could you comment on the potential for that? And then 2, there's been a lot of debate in the shifting regulatory landscape On whether the way you've run the subretinal study can actually be used For further studies of gene therapies, I mean said differently, it seems like the FDA is shying away from an Ability to use a sham control and that may cause problems for Longer acting anti VEGF therapies in pig controlled studies going forward.

Speaker 6

Thank you.

Speaker 2

Thanks, Dana. I'll probably let Steve address the comment about the regulatory landscape. I guess with respect to Altitude and Aviate, I think what's been an important part of the update today with respect to the strategic plan is that the extension of the runway for REGENXBIO, I think, Pivotal phase in achieving some of these milestone value events that can be unlocked. We have $500,000,000 of Potential development milestones over and a large proportion of that is associated with transition of Suprachoroidal delivery for wet AMD and Doctor occurring and patient dosing beginning there. We view that the data that started to come off that we're particularly excited about right now with respect to diabetic retinopathy is really shaping up for Support of decisions for that transition and we have additional data coming up just early in 2024 with respect to the 88 study.

Speaker 2

So I think We're, I think, as confident as we've ever been about the science that we're seeing and the likelihood of These events occurring in the future for us, I think are improved, particularly with respect to the updated strategic plan and the Extension of the runway that we have to focus on those events. Steve, do you want to pick up the regulatory part?

Speaker 3

Sure. Hi, Dane. Thanks for the question. Yes, interestingly or maybe not surprisingly, this was A discussion point at AAO this past weekend and also some of the ancillary Meetings that were held there, the issue of sham control. And I think there's a couple of key points here that A big component of the FDA's view on the challenges of masking with a sham control Relate to intravitreal injection, and that's due to the fact that there's a general belief Within the FDA, in particular that getting an intravitreal injection, the patient can actually sense that they're getting A real intraocular injection and may even be able to see something in the visual axis to suggest that there's actually been A fluid injection.

Speaker 3

So for our suprachoroidal programs, that's not an issue because we're not actually injecting into the eye. And we've had regulatory interactions where we're aligned on an approach for giving a sham injection That preserves masking. The other aspect that's come up from the FDA's discussion of this are There's other ways to address this such as considering having 2 active arms of Different dose level of your given drug that at least then preserve the masking between active arms. So there's several approaches that companies can consider. But fortunately for us, the fact that we're not an intravitreal injection Really shields us from this issue.

Speaker 2

Thanks for the question, Dana. I guess the level of detail that Steve is giving about thought that's going into the application of suprachoroidal delivery and later stage trials is, I think, additional evidence of

Speaker 7

This This is Gaspar on for Vikram. We have one question. And for potential partnerships for RGX-eleven, 181 and 301. Do you have a time line in mind for establishing a partnership? What would you be looking for through a partnership outside of capital?

Speaker 7

Thank you. Gospel, the

Speaker 2

strategic update plans today are really focused Assuring and sourcing capital for RGX-three fourteen, RGX-two zero two and RGX-one hundred and twenty one to Achieve what we think is the strongest potential value that we have in the pipeline today. So with respect to 111 and other things that's going to be a discontinuation of any clinical development work and an exercise that will result Looking for opportunities in the short term for partnering, but it won't become something that will be Viewed as a meaningful contribution to the operating plans going forward.

Speaker 7

Thank you very much.

Speaker 2

Thanks.

Operator

Our next question comes from the line of Alex Stranahan with Bank of America.

Speaker 8

Hey, guys. Thanks for taking our questions. Just one from me. Could you maybe expand upon the positive FDA interactions you've had After the update for 202 dose level 1, and did you get any input on the pivotal study design as well, particularly as it relates to the different age cohorts? Thanks.

Speaker 2

Yes. Thanks for the question, Alec. I think that it's been a very dynamic process for us with respect to contact with The FDA on RGX-two zero two and that's because of the landscape of what's happening in micro dystrophin in general. We want to understand, how FDA is sort of thinking about our data and other data sets and The evolution of things with respect to microdystrophin as a class. I think for us, incredible Opportunity to early on at dose level 1, talk about safety and And microdystrophin expression, sort of the pathway to making a dose selection.

Speaker 2

I think there's also a lot of interplay that we see on, let's say, for instance, manufacturing, Talik, right? We're working through basically DLA readiness for our manufacturing facility And a manufacturing process with 121, that is also something that is a path for us with respect to 202. And We're, I mean, this is for us between an RMAT meeting and interaction around 121 and between the opportunity to sort of update Leadership and people at FDA with additional information and data about RGX-two zero two, and also get feedback about What is going on with respect to the landscape of microdystrophin in general? It's a really rich time. I think that we're setting us up well for Thinking about strategies and operational plans that are about acceleration.

Speaker 2

And I think the best example of that was Just being able to modify the clinical protocol to reduce the number of patients from 3 to 2 Before moving into a parallel enrollment scenario with the dose levels that we're at, I think That was something that was informed by data, it was informed by safety, it was informed by updates to our dossier with respect to the IND And having alignment with FDA on that, I think was a meaningful signal.

Speaker 8

Great. Thanks, Ken. Very helpful.

Operator

Our next question comes from the line of Ellie Merle with UBS. Hi. This is Sarah on for Ellie. Thanks so much for taking our question. Just quickly on Potential milestones from AbbVie, I know they're not currently in your guidance.

Operator

Can you remind us what the So would be for moving into pivotal studies with suprachoroidal and then any color around the timing of Are there near term milestones there that we should be thinking about? Thanks.

Speaker 2

Sure, Sarah. Thanks for the question. Yes, We disclosed that we have over $560,000,000 of eligible development milestones associated with the partnership and a large Proportion of those milestones are associated with the suprachoroidal programs transitioning into pivotal phase. So I think those are things that we view with this change in guidance and runway guidance are Increasing likelihood for them to occur across the operational runway that we have now. And obviously, they've been answering questions already about Specific timing or things that have been going into how that decision is made, what I can say about some of the updates that we've given recently like With respect to the exciting data that we've seen from diabetic retinopathy and some of the thinking that has been going on within the partnership is that We really think that the data sets are starting to mature now to a point where those types of decisions can be made soon.

Speaker 2

We have the Hawaiian Eye data update coming also in the beginning of next year. These are things that now with a Runway guidance into the second half of twenty twenty five, I think are achievable. And again, I tried to frame this in the overview. I think these are opportunities to sort of bridge to obviously, we're talking about 100 of 1,000,000 of dollars in milestones here that can occur that can bridge the company Even further into years 'twenty six, 'twenty seven, for instance, and those start to be years now where We're talking about BLA filings of the subretinal program and potential if you think about acceleration for Duchenne program, Additional product launches. So we really like how the strategic update plan here and the interplay of some of these milestones Are coming together to think about effectively having opportunities to capitalize ourselves with things that are currently assets of the company,

Operator

Great. Thanks.

Speaker 2

Thanks, Sarah.

Operator

Our next question comes from the line of Luca Easi with RBC Capital Markets.

Speaker 9

Good. Thanks for taking our questions. Just maybe one this is Lisa on for Luca. Just maybe one on DMD. We know the AFFINITY trial is enrolling patients up to 11 years of age.

Speaker 9

However, from the EMBARK data, It appeared harder to discern a benefit in older children versus younger children. So just wondering what gives you confidence that 202 will show a benefit in older children, because it especially when it appeared that the Western blot suggested that there was higher expression in the younger patient. Any color there would be helpful. Thanks.

Speaker 2

Yes. Thanks, Lisa. I mean, look, we have an older patient enrolled already. And I certainly acknowledge the remark about the differences that we've seen in microdystrophin expression, but we're going to be able to see sort of unfold in front of us and Continue to be able to update on the progress of that patient. I think the meaningful differentiator here is the C terminal domain design of our expression cassette.

Speaker 2

We just think that Scientifically, from a biological plausibility perspective, amounts of microdystrophin that are expressed with The C terminal domain are going to translate into improved function. And I think that gives RGX-two zero two Better and as good as any other treatment, but really better than any other treatment that spring forward to date to Show improvement in older boys. So we continue to view that the opportunity to enroll in that age range is something that's going to be important for our profile Clinically and ultimately commercially. And we do think that there's strong validation in the 4% to 5% and The emergence of more data in 6 to 7 is also sort of helping inform the benefits there, but we really feel strongly that the C terminal domain, The functionality associated with it, what we've seen from our preclinical data and the early evidence that we've Reported on and we'll continue to update on in an older patient, I think is something that we're excited about for that age range. Thanks, Lisa.

Operator

Our next question comes from the line of Brian Skorney with Baird.

Speaker 10

Hey, good afternoon, everyone. Thanks for taking the question.

Speaker 6

It just sounds like you're getting near

Speaker 10

a decision point on pivotal programs with the suprachoroidal administration. So just wondering what's sort of the rate limiting factor In terms of ABA and altitude to kind of make a decision, you really need like 1 year data from cohort 6 and cohorts 45 to trigger that point and you've been talking very enthusiastically about diabetic retinopathy. Do you see sort of the pivotal program moving forward in Doctor and wet AMD at the same time? Or will one of these indications Potentially, move forward earlier than the other?

Speaker 2

Yes. Thanks for the question, Brian. I think, again, like We have a lot of enthusiasm about the recent data update and there's a recency effect there. Of course, AAO was Just a few days ago, and I think the new Doctor data has now been communicated and has been a focus for us in terms of communication in the last few days. With respect and then we have AVI update coming only in January.

Speaker 2

I think our vision has been from the beginning that Wet AMD subretinal, wet AMD suprachoroidal, wet AMD for diabetic retinopathy, they are Obviously, all interrelated because of the same pharmacological agent, but each one of them because of the difference in delivery or In the last case, in the difference with respect to the potential market and the sort of evidence that's needed clinically to think about how to transition into pivotal, They're all slightly different path, but they derive from the same consideration and variables that We would look out for any kind of advancement of a program like this, almost independent of modality into pivotal phase. I always like to point out that We are the company that is the first to have transitioned into a pivotal phase program with respect to An AAV gene therapy in a large market indication in wet AMD. And now we also have the strength and sort of the robust Decision making to sort of bring additional variables into the equation with us with AbbVie. Yes. The decisions though are going to be different and separate between the suprachoroidal wet AMD and the suprachoroidal diabetic retinopathy.

Speaker 2

I mean, They rely on different data sets and different inputs. So it's certainly possible that they could happen at the same time, but it's as likely that Those decisions are independent variables and would come forward. But I think the message today is How strongly we feel about reinforcing our excitement about the value of RGX-three fourteen And the opportunity to continue to realize that value and continue to make that focused investment with An extended runway here, I think, really improves the probability of success of meeting some of these milestones, in terms of transitioning to Pivotal. Therefore, realizing some of these monetary milestones that are associated with the partnership in a way that It's improved, right, just with that extended runway alone. But at the same time, we've also started to become really encouraged about The newer and longer term data that's been coming out of the investments that have been made in things like Aviate and Altitude.

Speaker 2

So, Excited for things to come here moving into 2024.

Speaker 8

Thanks, Tim.

Speaker 2

Thanks, Ben.

Operator

Our next question comes from the line of Annabel Samimy with Stifel.

Speaker 11

Hi. This is Jack on for Annabel. Thanks for taking our question. So for the subretinal formulation of 314 in wet AMD, What's the right amount of follow-up time that FDA is looking for to establish safety and durability there? And what might the value proposition be here For the payers, when you think of pricing this, considering that many of these patients are elderly and may not see the maximal benefit of a long term treatment.

Speaker 2

Steve, do you want to take the regulatory part here?

Speaker 3

Sure. Hi, Jack. Yes. So taking the first part of your question, the duration from a regulatory standpoint as far as follow-up, We've gotten very clear feedback back as far as the End of Phase 2 meeting that allowed us To finalize the pivotal program that really is a class requirement Having 1 year safety and efficacy data, which also was somewhat informed by efficacy, but Predominantly by gene therapy aspect. We'll also have longer term follow-up data from a lot of the patients From our pretty sizable safety database and we actually already have from our Phase onetwo database out through 4 years, good safety and also good durability from an efficacy standpoint.

Speaker 3

So I think Both based on other programs, even outside the eye that have shown durability out, even beyond a dozen years, for example, in Hemophilia with the same AAV, stereotype AAV8. And then now our own data gives us a lot Confidence on duration of efficacy. And I think that's obviously going to inform the value proposition Having durable efficacy evidence and the value proposition importantly is more than just Decreasing treatment burden and visit burden, really the big unmet need here is By breaking the barrier of the treatment burden, it ensures that these patients are going to have the ongoing sustained anti VEGF activity That they need to prevent vision threatening complications that they simply aren't getting now both in terms of wet AMD And diabetic retinopathy because of the treatment burden. So there's various layers of the value proposition, Treatment burden, visit burden and actual vision and complication outcomes.

Speaker 2

Yes. And I would add, Jack, that I think it's pretty well established by a lot of different sources that The cost of the cost related to blindness that's associated with wet AMD is on an annual basis, Something that can be in excess of $100,000 a year and that's separated from the cost of drug. So When we think about wet AMD patient, you're talking about the life expectancy might be on the order of 10 to 15 years, I think in the real world, there's a need there for Recovering the vision that is being lost because people aren't staying compliant with the existing treatments and the costs and the Obviously, the effect that that's having on a productivity basis with respect to the effect that it has on the families of those as well is significant. And so durability is everything. I like how Steve brought up the 4 year evidence, which Just continues to grow.

Speaker 2

It's there as a backstop for things on a regulatory basis, but I think also important in the commercial setting.

Speaker 11

Great. Thanks so much.

Operator

Our next question comes from the line of Mani Foroohar with Leerink Partners.

Speaker 8

Hey, guys. Thanks for taking the question. Obviously, you have a fun decisions to make, but obviously the right thing to do. You've outlined in the press release a little bit of detail around about 100,000,000 Savings, etcetera, couple of years. Could you give us a sense of where that tempo is?

Speaker 8

Like should we expect that to drop in immediately starting In 1Q and be fairly linear on a quarterly basis, or

Speaker 10

is it something that's a little more front loaded, a little more back loaded over the 1st couple of years?

Speaker 8

And beyond that, how should we think about post that 2 years period, what is the implication on

Speaker 10

OpEx Longer

Speaker 2

term. Nick, do you want to chime in on this one?

Speaker 4

Sure. Thanks, Manny. I think that as you're thinking about that $100,000,000 It would be not 100% linear as much as it slowly gets bled into our operating plan going forward. Some of it will be a little bit lumpy as we start to discontinue programs and then obviously With that 15% headcount reduction, that's a little bit more front end loaded. And then as we think about OpEx, I would start to be looking at the models and search of determine what's the appropriate kind of run rate, What I've seen looking at different analyst models is their estimates for OpEx seem to be A lot higher than what we're tracking against.

Speaker 4

And I think coming out of this quarter into next year, You'll have a better idea of what the run rate is on an OpEx perspective. And then on the other side of 2025, obviously, With the non dilutive capital that we're talking about and the opportunity that possibly the markets will unlock finally, I think post 2025, we're going to still maintain our discipline as it relates to OpEx, but We'll also have to start moving programs forward as they become commercial opportunities, then also Start to think about our pipeline going forward.

Speaker 7

That's helpful. Thanks. I'll hop off.

Operator

Thank you. This concludes today's conference call. Thank you for participating. You may now disconnect.

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