Q4 2024 REGENXBIO Inc Earnings Call
Speaker Change: Welcome everyone to the fourth quarter and year end, 2024, Regenxbio Earnings Conference Call.
At this time, all participants aren't a listen only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you'll need to press star 111 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 111 again. You'll need to press star 111 again.
Please be advised that today's conference is being recorded.
Speaker Change: At this time, I'd like to turn the conference over to Patrick Christmas, Chief Legal Officer of Regenxbio. Please go ahead.
Thank you. Thank you. Thank you.
Speaker Change: Today's conference call will include forward-looking statements regarding our financial outlook in addition to regulatory and product development plans [inaudible]
Speaker Change: These four-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 plan will may anticipate believes should intend in other words of similar meaning.
Speaker Change: Any such forward looking statements are not guarantees of future performance and involve certain risks and uncertainties.
Speaker Change: These risks are described in the risk factors and the management's discussion and analysis section of Regenxbio's annual report on form 10k for the full year ended December 31st, too
Speaker Change: 2024, and comparable risk factor sections of Regenxbio's quarterly reports on form 10Q, which are on file with the Scariest Interchange Commission, and available on the FCC's website.
Speaker Change: Any information we provide on this conference call is provided only as of the date of this call, March 13, 2025. And we undertake no obligation to update any four looking statements we make on this call on account of new information, future events, or otherwise.
Speaker Change: Please be advised that today's call is being recorded and broadcast, in addition to any unaudited or pro forma financial information.
Speaker Change: and may be provided as preliminary and does not report to project financial positions or operating results of the company. Actual results may differ materially.
Speaker Change: I'll now turn the call to Curran Simpson, President and CEO of Regenexx Bio. Curran?
Curran Simpson: Thank you, Patrick, and thank you everyone for joining us today.
Curran Simpson: 2025 is a transformational year for Regenxbio and we're off to an exciting start. We've submitted our first BLA and expect our first FDA approval in the fourth quarter for RGX-121 which is our treatment for boys with 100 syndrome.
Curran Simpson: We will build on this momentum by advancing our diabetic retinopathy program into pivotal stage this year.
Curran Simpson: then head into 2026 and 2027 with potential BLA filings for large opportunities, including RGX202 for Duchenne Muscula Distrophy and ADDD RGX314 for WET AMD.
Curran Simpson: We are in a strong position as we prepare to launch multiple first or best in class gene therapies and have robust commercial capabilities and global partners.
Curran Simpson: This is all with a view to sustainable profitability and is the result of 15 plus years of gene therapy leadership.
Curran Simpson: Today's call I will review our recent business highlights and outline of vision for what we believe will be catalyst rich years ahead.
Curran Simpson: Then, our Chief Medical Officer, Dr. Steve Kola, will summarize our clinical progress before handing it over to Mitch Chan, Chief Financial Officer
Mitch Chan: Mitchell will provide an overview of our financial results and share the many potential non-delutive capital sources ahead.
Speaker Change: I'll then make some closing remarks before we open for Q&A.
Speaker Change: Starting with our recent, exciting news in MPS, we are thrilled to have completed the submission of the BLA for RGX-121
Speaker Change: or Komitsa Jean, Lampar Vivek, under the Accelerated Approval Pathway and partnered with Nippon Shinyaku for both of our MTS programs.
Speaker Change: This partnership marries our collective strengths, Regenxbio's development and manufacturing expertise with Nippon's experience in successfully commercializing rare disease products.
Speaker Change: Our teams are planning for potential approval of RGX-121 for NPS-2 in Q4 2025 and are working diligently to prepare for the commercial launch.
Speaker Change: Along with enabling access to these important medicines for patients in the US and Asia, this partnership is strategically significant to Regenxbio [inaudible]
Mitch Chan: As Mitchell's share, this agreement provides meaningful potential milestones and revenue for us.
Mitch Chan: The Potential Approval of Argyx 121 also provides strategic value for the rest of our pipeline [inaudible]
Mitch Chan: As we receive commercial licensure of our manufacturing facility prior to launching in our larger opportunities, including RGX 202, Produce Gen
Mitch Chan: Moving to RGX-202, I am pleased to report that our pivotal study is advancing rapidly. I am pleased to report that our pivotal study is advancing rapidly.
Mitch Chan: and that our unique second-to-market or fast follower opportunity is on track for a mid-2026 BLA filing.
Thank you. Thank you. Thank you.
Mitch Chan: Recent updates have indicated tremendous interest in the patient community for new treatments with RGX-202 as a valued option.
Thank you. Thank you.
Mitch Chan: Input from our growing investigator community supports our belief that our GX202 has the potential to be a preferred and differentiated treatment option.
Mitch Chan: I'll remind you that, RGX202 is the only investigational next-generation DMD gene therapy in Pivotal study.
and with both robust microdistant and functional data available.
Mitch Chan: The pivotal trial of RGX-202 is rapidly enrolling approximately 30 ambulatory patients aged 1 and over.
Mitch Chan: I'm pleased to share that this pivotal trial is nearly half enrolled, and we expect to complete enrollment this year.
Mitch Chan: We are also on track to submit a BLA under the Accelerated Approval Pathway by Mid-2026 We are on track to submit a BLA under the Accelerated Approval Pathway by Mid-2026
Thank you very much.
Mitch Chan: We have spent more than half of the prevalent population to remain untreated through the next few years.
Mitch Chan: and this large population will need more than one treatment to serve all due shantations.
Mitch Chan: That's why we're confident in the rapid progress we've made thus far.
Mitch Chan: and our path to delivering a potentially preferred gene therapy option.
Mitch Chan: Also let me remind you that we are commercial ready when it comes to manufacturing 202.
Our in-house state-of-the-art suspension-based bioreactor process
is currently producing 202 for our pivotal study.
with industry leading purity levels of more than 80% full-castes.
Mitch Chan: Our Manufacturing Innovation Center can produce 2500 doses of RGF 202 per year [inaudible]
Mitch Chan: As we continue to aggressively accelerate BLA, enabling activities and commercial planning.
We will share more meaningful updates.
Mitch Chan: Specifically, we plan to share additional positive phase one, two biomarker data later this month at MDA.
Mitch Chan: as well as updated Phase 1-2 functional data in the first half of this year. [inaudible]
Mitch Chan: With positive biomarker and functional data in hand, encouraging interactions with FDA and commercial ready manufacturing, we believe our second-to-market position in Duchenne remains strong.
Mitch Chan: Lastly, I will highlight a few updates on ABBV RGX 314.
or Surap, Jean Blamparovic. [inaudible]
Mitch Chan: This is our Global, Abbey Partner, Retino Franchise, that is advancing in late-stage studies.
Mitch Chan: As announced with Abdi in January, data from the pivotal studies evaluating subretinal 314 in patients with wet AMD are expected in 2026.
Thank you very much. Thank you.
Mitch Chan: We expect to complete enrollment of both of these pivotal studies this year.
Thank you. Thank you. Thank you.
Mitch Chan: In our diabetic retinopathy program, evaluating in-office super-curoidal delivery of 3-14 [inaudible]
Speaker Change: We held a successful End of Phase II meeting with the FDA in the fourth quarter of 2024.
Speaker Change: We are now planning a pivotal program with ABD to support future global regulatory filings.
Speaker Change: As we have said before, Web AMD and DR are very large commercial opportunities [inaudible]
Speaker Change: And 314 represents a potential alternative for the patient's losing vision with today's standard of care.
Speaker Change: Importantly, we will be entitled to additional milestone pavements that are a part of this $1.8 billion collaboration with Addy
Speaker Change: In summary, we are excited for a well-position to deliver the opportunities ahead of us to drive values for patients and shareholders.
Steve Pakola: With that, I would now like to turn the call over to Steve for an update on our clinical programs. Steve?
Steve Pakola: Thank you, Curran. I'll start with RGX 202, a potential one-time gene therapy for the treatment of Duchenne. I'll start with RGX 202, a potential one-time gene therapy for the treatment of Duchenne.
Steve Pakola: RGF-202 is the only micro-discipline construct to include the C-terminal domain, making it closest to naturally-occurring discipline.
Steve Pakola: In preclinical studies, microdistrophin with the CT domain, we shown to better protect the muscle from contraction induced damage associated with muscle breakdown in Dushen.
Steve Pakola: In November , we announced the initiation of the pivotal phase of the Affinity Duchenne trial evaluating 202 at a dose of 2-E-14 genome copies per kilogram in approximately 30 ambulatory patients, H1 and older.
Steve Pakola: We also recorded positive safety and efficacy data from the phase one to portion of the study.
Steve Pakola: The data disclosed in November included positive functional outcomes from the first five participants in the Phase 12 portion at 9 and 12 months.
Steve Pakola: We also shared microdistrophin and other compelling biomarker data from 11 patients.
Steve Pakola: In summary, the results showed 202 recipients exceeded external natural history controls and established benchmarks for clinical outcomes.
Steve Pakola: Specifically, we observe functional improvements in all patients treated with dose level 1 and dose level 2 at 12 and 9 months respectively.
Thank you.
Steve Pakola: Consistent robust expression, transduction, and localization of our differentiated 202 micro-disturphant in the muscle and also a favorable safety profile observed at both dose levels.
Steve Pakola: There were no serious average events or AE's of special interest. [inaudible]
Steve Pakola: which is truly outstanding in the landscape of Duchenne gene therapy.
Steve Pakola: As we've discussed, we've taken a thorough, proactive approach to safety based on input and partnership with treating physicians [inaudible]
Steve Pakola: and the patient community, as well as learnings from the field at large. [inaudible]
Steve Pakola: Our immune suppression regimen, including a short course of ecolismab, combined with our novel microdistriction and leading product purity levels may be contributing to this positive safety profile.
Steve Pakola: These positive clinical results further strengthen our belief that 202 has the potential to serve as a best-in-class gene therapy produced shed muscular dystrophy.
Steve Pakola: We look forward to sharing additional biomarker data at the MDA meeting in the coming days, including the first micro-discipline data from our cohort of patients under four years old. [inaudible]
Curran Simpson: As Curran mentioned, the pivotal study is ongoing and advancing rapidly.
Curran Simpson: The one-to-three age group represents the significant portion of the prevalent population of Dushen patients yet this group has no access to approved gene therapy.
Curran Simpson: With pivotal enrollment nearly halfway completed, we look forward to continuing to work with physicians and the Duchenne community to complete enrollment this year and report top line data in the first half of 2026.
Now onto our retina franchise, ABVB RTX.
Curran Simpson: 314, which is being developed in collaboration with Abbey to treat WEDAMD and diabetic retinopathy or DR. I'll start with 314 for DR, being evaluated in the Phase 2 altitude trial using in-office supercroidal delivery. [inaudible]
Curran Simpson: Like Wet AMD, DR is a progressive disease that causes vision loss and ultimately blindness if not treated appropriately.
It is a leading cause of blindness in working-age adults.
Curran Simpson: As we've shared, we have completed our end of phase two meeting with the FDA and are now working actively with Abby on plans for our phase three clinical program.
Curran Simpson: The program is expected to support global regulatory filing with the goal of preserving vision for millions
Curran Simpson: We previously expanded the broad multi-indication global potential of 314 by initiating a new cohort in the altitude trial.
Curran Simpson: This cohort is enrolling patients with diabetic macular dima, or DME, a vision-threatening complication of diabetic eye disease.
Curran Simpson: In WEDAMD, we are evaluating 314 via two different delivery forms, sub-retinal and super-coroidal.
Curran Simpson: Within Sub-Retmo, we have two ongoing Pivotal Trials, atmosphere and scent in the US, Europe and Japan.
Curran Simpson: These trials continue to progress well as we announced in January , enrollment of both pivotal trials is expected to complete this year and we expect to share top line data in 2026.
Overall, we continue to be encouraged by 3.14's progress.
Curran Simpson: I'd like to particularly highlight the safety profile observed in our Super Corridor program.
Curran Simpson: In more than 180 patients treated in office, we're seeing a differentiated safety profile, particularly in the setting of short course seven weeks,
Curran Simpson: This continues to support the potential of 3.14 as a meaningful treatment option for patients and physicians.
Finally, onto our MPS program.
Curran Simpson: It's an incredibly exciting time for Regenxbio and the Hunters Syndrome community with the submission of our BLA for RGX-121.
Curran Simpson: This filing is based on data from the campsite trial which met its primary pivotal end point with high statistical significance.
Curran Simpson: Patients treated with 121 that she decreased CSF levels of heparin sulfate D2S6, a key biomarker of brain disease activity, to below maximum attenuated disease levels, approaching normalization.
Curran Simpson: RBLA using the accelerated approval pathway is based on D2S6 as a surrogate endpoint reasonably likely to predict clinical benefits. [inaudible]
Curran Simpson: We also previously reported that 80% of patients who receive the pivotal dose, discontinued and signed replacement therapy, or remained treatment naive, as well as neurodevelopmental skill acquisition.
Up to four years post dosing. [inaudible]
Curran Simpson: This is a meaningful update for patients and families whose only option today is weekly and thumb replacement therapy that does not address the neurocognitive decline of this disease.
Curran Simpson: 121 is well positioned to be the first gene therapy and one-time treatment for Hunter Syndrome that can address neurocognitive decline.
Curran Simpson: We anticipate a potential FDA approval decision in the second half of 2025.
Curran Simpson: To conclude, we continue to make significant progress with data updates and trial progression across all programs in our pipeline [inaudible]
Curran Simpson: 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. Thank you very much.
Mitch Chan: With that, I'll turn the call over to Mitch to review our financial guidance.
Mitch?
Mitch Chan: Thank you, Steve. Regenxbio ended the quarter on December 31, 2024 with cash equivalent and marketable securities of $245 million, compared to $314 million as of December 31, 2023.
Mitch Chan: The decrease is primarily driven by cash used to fund operating activities during the 12 months ended.
Mitch Chan: December 31, 2024, partially offset by $131 million and net proceeds received from an upsides public offering of common stock and pre-funded warrants completed in March 2024.
Mitch Chan: R&D expenses were $209 million for the year ended December 31st, 2024, compared to $242 million in 2023.
Mitch Chan: The decrease was primarily attributable to decreases in headcount and preclinical activities.
Mitch Chan: We expect the balanced and cash equivalent and marketable securities of $245 million out of December 31, 2024 to fund our operations into the second half of 2026.
Mitch Chan: This cash runway guidance is based on the company's current operational plans and excludes the impact of any future potential commercial revenue associated with RGX-121
Mitch Chan: If we include additional non-falued financing, we expect the cash runway to potentially extend meaningfully beyond 2026
Mitch Chan: For instance, some examples of our non-diluted financing options include our expected DR milestone payment from Abbey, Development milestone payments from the Ponshin Yaku.
Mitch Chan: Reversion of our sojournment royalty income and the potential monetization of our priority review voucher on our GX-121 HIPAA proof.
Mitch Chan: Collectively, we have many non-thaluda financing optionality that could extend our cash runway well beyond the second half of 2026.
Curran Simpson: In summary, we feel we are in a strong financial position as we near potential commercialization of three late stage programs. With that, I would turn to call back to Curran to provide final thoughts.
Thanks, Mitch.
Curran Simpson: As you heard today, there's strong momentum across our pipeline as we advance towards key milestones. . . .
To recap, [inaudible]
Curran Simpson: We have officially submitted our first BLA and closed an important partnership with Nippon Shinyaku for our MPS programs.
Curran Simpson: We look forward to a potential FDA approval of our GX-121 for this year.
Curran Simpson: The pivotal study of RGX 202 is moving rapidly, and we believe remains well positioned to potentially serve as the next and preferred gene therapy in Duchenne muscular dystrophy in Duchenne muscular dystrophy in Duchenne,
Curran Simpson: We plan to share additional positive biomarker data later this month followed by updated functional data in the first half of this year.
Curran Simpson: and with enrollment nearly halfway through, we expect a complete enrollment of the Pivotal Study this year and share top line data in the first half of the 2026.
Curran Simpson: Our partnership with AV is advancing towards multiple large global commercial opportunities.
Curran Simpson: We expect a complete enrollment of two global pivotal trials for sub-retinal wet AMD.
Curran Simpson: and are working with Abby on a pivotal study in diabetic retinopathy in 2025.
Thank you. Thank you.
Curran Simpson: As we look towards the next nine months in beyond, we are well positioned to deliver on multiple late-stage opportunities [inaudible]
Curran Simpson: Our programs are demonstrating beneficial differentiation against standard of care and available treatments.
Curran Simpson: Each of our assets represent one-time treatments with the potential to transform the trajectory and management of disease for patients in need of new and better options.
Curran Simpson: We look forward to sharing additional updates on our plans in achieving our critical milestones this year.
Curran Simpson: With that, thanks for everyone for your time today. I'll turn the call over for questions.
Operator,
Speaker Change: As a reminder, if you'd like to ask a question at this time, please press star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again.
Please stand by while we compile the Q&A roster.
Speaker Change: Our first question comes from a line of Judith Frammer with Morgan Stanley
Judo, your line may be on mute.
Hi, can you hear me now? [inaudible]
Yep.
Speaker Change: Thanks, thank you for your question. Hi, congrats on the progress here. Just one on cash runway and one clinical question. So I was hoping maybe you could...
Delve a little deeper into the components of non-dilutive financing.
Speaker Change: that are still available, and maybe you could give us some...
Speaker Change: Internal Insights into probabilities around realizing those we do their questions around.
Speaker Change: The PRV, and then anything that could get in the way of the Abbey milestone coming through for DR. And then just on the clinical side, another question we've been getting just expectations around potentially going to an Edcom for 202, once you have...
The full data set, thanks.
Yes, thank you, Drudeff for the question. Thank you, Drudeff.
Speaker Change: So regarding non-deludive options, as I kind of mentioned, it really comes in three different flavors. The first one is the DR milestone that we expect to receive in the second half of this year.
Speaker Change: That really is gated upon the first patient dose, and Stephen Curran could only speak to the clinical program timings and so forth, but that's really the potential risk to it is more the timing issue than anything else, because our expectation is said that we'll move forward. Thank you very much.
Speaker Change: Regarding the PRV, I think the question here is when do we receive a potential regulatory approval? Because upon regulatory approval that's when we're eligible to receive our prior review voucher and we could then choose to monetize it at our discretion. Thank you very much.
Speaker Change: So I think that's, I'll say it's more of a regulatory approval risk than anything else which you're not expect to have anyways at this moment in time.
Speaker Change: And the other non-deludive financing option really is on the potential suggestive overall tea stream that may river back to us. Once we hit the HCR cat.
Speaker Change: 300 million dollars, that relative stream does reverse back to us. So, again, it's hard to predict future revenues from us, or Jensma, given that it's promoted by a third party, no parties here. But, once we reach the cap, that revenue stream will reverse back to us, forward to your revenue stream.
Curran Simpson: Hi, Judith, Curran. I can talk a little bit about just the PRV and probability of success. And I think the fact that we've had a pre-BLA meeting with FDA, written reviewed the pivotal data really helps in terms of de-risking. They've seen the data, they've seen our approach. [inaudible]
Curran Simpson: and while of course it's a regulatory review and you know that has risk... [inaudible]
Curran Simpson: associated with it like any. We feel like we're in a really good place particularly because I think in. [inaudible]
Over time, MPS2 and...
Curran Simpson: I do expect this to be a high probability event in terms of receiving both an approval and the PRV.
Curran Simpson: Abby in the lead in terms of conducting those meetings. We talked last fall about the end of phase two meeting with FDA and related that that meeting was very positive in terms of our approach.
Curran Simpson: and we've also discussed that they're also seeking regulatory advice XUS and that's an ongoing process.
Speaker Change: But we feel like two key things, the data is strong, and so far the feedback is strong. Again, pointing towards a positive movement towards the DR pivotal this year. And Stephen, I don't know if you have anything to add to that. [inaudible]
Sure, hi, Judah, Ab
Steve Pakola: Yeah, I think as Curran mentioned, we're very happy with the end of phase two meeting with the FDA. Happy's always been very interested in global advancement and really getting access to 314 globally. That's one of the reasons we...
Steve Pakola: Struck the deal with APV, so not surprisingly for a global diabetic retinopathy plan, they do want to get EMA and T back from Japan. So those are some of the aspects that we want to tidy up those.
Steve Pakola: and before we give any more details on the plan, but I think fortunately we have the benefit of precedent in treatment of diabetic retinopathy to know. So,
Steve Pakola: What to expect in terms of a regulatory path here. So, we feel this is pretty darn the risk based on our regulatory discussions today. You also, Judah, had a question on adcom.
Steve Pakola: Probability, which I believe is probably related to 121 since we announced completion of the VLA submission.
Steve Pakola: We don't know, you know, it's not the kind of thing we can say with 100% certainty either way. We, as of now, don't see a significant issue anywhere that would suggest one is needed. Good.
Steve Pakola: If one comes our way, we'll be ready for it. So I think with any development program at this stage, you plan in case you may need it. If we have one, we'd be very confident with. Yeah.
Steve Pakola: The discussions because of the compelling data we have and the different stakeholders that could be a part of getting across that benefit and the benefit risk for the product. But as of now, we don't know.
Thanks.
Gina Wang: Our next question comes from the line of Gena Wang with Barclays.
Thank you. Thank you. Thank you.
Gina Wang: Thank you for taking my questions. I apologize if this question...
Gina Wang: I'm already being asked, my phone got dropped, so I missed the...
Gina Wang: So I have two questions. One is the regarding the MBA update, just wondering how many patient data from one to three year old should we expect?
Gina Wang: C, and also in giving the younger patient age, shall we expect to see higher protein expression?
Related questions, any thoughts regarding lower age to zero? No.
Speaker Change: Giving, I think, a solid mention, they will start a cohort between 0 and 18.
Gina Wang: And then my second question is regarding the altitude, DNA cohort. Just wondering what will be the timing of data update and then in terms of also number of patient and type of data you will share with us. [inaudible]
Thanks.
Steve Pakola: Great, I'll take the first one and then I'll let Steve answer the second one. So we're planning to update at MDA.
Steve Pakola: likely one patient in the one-to-four cohort in terms of their microdistant value.
Steve Pakola: And I would suggest based on the emerging database that we're gaining on age versus microdistrophin that it's a fair expectation to expect a high number in terms of
Steve Pakola: Microdistrophin level, but obviously we'll save that specific for the conference itself, but we're seeing a general trend that the younger patients will have higher microdistrophin numbers, both in our study and other studies that we're able to see in the public domain. Thank you very much.
Steve Pakola: Yeah, hi, hi, Gina, thanks for the question. So altitude are diabetic retinopathy study where we've already shown very solid data in terms of diabetic retinopathy patients without mental health.
Steve Pakola: DME, and that's why we're so excited with Abby to go into Pivotal.
Steve Pakola: You refer to the DME cohort, which we currently have enrolling. Enrollment is progressing quite nicely. We haven't given any guidance on when we would read out, but it's safe to say that when we do to your question of what you could look for. Thank you very much.
Steve Pakola: You could look for the typical things that we've looked for in a Wed AMD population.
Steve Pakola: but with different reference points of what you'd expect. So in DME you care about visual acuity and you also care about disease activity assessed by retinal thickness.
Steve Pakola: on OCT, and of course the third part of the triad is can you achieve good disease control and good visual acuity with a reduction in the need for injection. So basically treatment burden. So we would report on all three of those as well as safety.
Thank you [inaudible]
Operator: Our next question comes from a line of Mani Foroohar with Learing.
Thank you. Thank you.
Operator: Hey guys, you have Ryan Don for Mommy, thanks for taking our question. So maybe just you can talk about what you're seeing in terms of pace of enrollment for the DMD pivotal trial and whether you expect this to accelerate as the year goes on as you activate new sites and share more data in the first half of the year. Thank you very much.
Operator: And then maybe just on the younger patient enrollment within DMD, have you moved on to enrolling those patients in the pivotal trial yet? Or are you still enrolling that separate cohort from the phase one to trial? Thanks
Pick [inaudible]
Operator: Yeah, I think it's safe to say that we're seeing really encouraging enrollment in terms of the Dushan study.
Operator: and I think to the second part of, and we know that from the screening logs and and what we're seeing at sites as sites.
Operator: are activated that there's a significant amount of interest in a significant number of patients that are going through screening.
Operator: and the second part is, yes, we do expect it to accelerate primarily because we're increasingly adding sites to the study. So,
Operator: The overall productivity of the study will just continue so it will be a non-linear enrollment. Amen.
Operator: Currency Well, and we're super optimistic about how, certainly meeting our guidance, which will be concluding enrollment this year, but we're aiming to exceed that guidance by beating that timeline significantly. [inaudible]
The second question.
The other interesting aspect, Ryan, is it's not just...
Operator: increased sites, which certainly is going to help as we bring more sites onboard, but we really saw a lot of interest after our November functional data update.
Operator: We've heard from patient advocacy and investigators that there's a lot of families that have been waiting on the sidelines for
The Next Gen Gene Therapy
Operator: as long as they could see some functional data. So I think having the 9 and 12 month data has really bolstered our case across our clinic. So I think we're in a good shape there. You also asked about the younger patients, the one to three year old age group. [inaudible]
Operator: We look forward to providing the initial data in terms of microdistant in that age range. And we do now, by the way, we are getting back to Gena's question that.
Speaker Change: Younger, we have seen with other programs that you have higher microdistrophin levels.
Operator: and I think one of the massive unmet needs is an older patient where...
Operator: You don't see with other programs high, microgistrophin levels, so we're really excited about that.
Operator: But we really want to treat broadly across the entire age range, and that's why we're...
Operator: Enrolling, not just the traditional four and up, but actually one year old and up. To your question, any patients that come in now, that are one and above, are going to count for a pivotal. Thank you very much.
Operator: So, we are very excited to enroll across the age range that one to three years, four to seven, and also eight and older.
Operator: And we're particularly interested in that cohort because as you know there's no proof therapy for patients in the wonderful age category. So that's a key element of our pivotal plan. And these patients will be included in that.
Thanks guys [inaudible]
Speaker Change: Thank you for watching. Please subscribe to my channel. I hope to see you again soon.
Speaker Change: Our next question comes from a line of Luca Issi with RBC Capital Markets
Thank you. Thank you. Thank you.
Speaker Change: Oh, great. Thanks so much for taking our questions. This is Lisa on Feluca. On the DMD program, can you remind us if you are measuring cardiac endpoints like left and jicular injection fraction or cardiac biomarkers like proponent I as part of the affinity study?
Speaker Change: And it's so, could these be a meaningful way to differentiate your program further from elevitis?
And I have one question on WAMD.
Speaker Change: On the sub-ragnal study, I'm just curious if you are considering maybe cutting the enrollment a bit to your in order to to get the enrollment a bit better.
Speaker Change: Accelerate the Readout Timeline. We know the wedding and these spaces are very competitive and there are also two long acting PKI studies that are also anticipated to read out in that same 2026 timeframe.
Speaker Change: And, as we know, from Fubizema's launch, being first to market, does matter. So any color here on D&D or the sub-retinal pivotals, that would be appreciated. Thanks so much.
Good Thanks!
Speaker Change: I think I'll let Steve comment certainly on the retina discussion, but in terms of cardiac, Steve can...
Speaker Change: to describe what we're measuring in the clinic, but certainly from our preclinical data, when we looked at biodeistribution in our early preclinical studies, we certainly have reason to expect a good biodeistribution. [inaudible]
Speaker Change: Not just to the peripheral muscle, but to the heart and to the lung section. So that's represented well in studies that we did preclinically like the treadmill test.
for MDX Mike, so I think...
Speaker Change: In terms of, do we expect our study to differentiate on that? Of course, that will come down to...
Speaker Change: What we consider for cardiac, a little bit longer term outcomes. We don't expect to see dramatic cardiac improvement in a four or five year old but over time that something will certainly be monitored. Maybe I'll let Steve cover how we're doing that. Sure. Hi, Lisa.
Speaker Change: Yeah, for the reasons Curran mentioned, we feel very positive about the potential to have a benefit for Duchenne.
Speaker Change: Children, not just based on skeletal muscle but also the cardiac manifestations that I think one context to give is that
these
Speaker Change: Decraases or deterioration and cardiac function happen at an older age than...
Speaker Change: The typical functional assessments that we look at in the younger age group, so it's really as the boys advance into the teen years that you see the unfortunate cardiac deterioration. So,
Speaker Change: That's one of the reasons we're so excited that we're seeing very high and consistent microdistrophin expression in these older boys, so that also bolsters our competence. So how can we look at this? Certainly.
Speaker Change: ejection fraction, as you mentioned, is one of the measures you can look with Echo, which isn't...
Speaker Change: as accurate and sensitive as looking with MRI. So we do incorporate both of these, so we're going to have the ability to look at that. We do also look at trapponin levels. [inaudible]
So again, I think the key aspect is your... [inaudible]
Speaker Change: It's not going to be really meaningful to look at these while.
Speaker Change: Patients Cardiac Function is Normal, so in the traditional age groups up to eight, for example, and really it's going to be more once the patients get above 10 where you start to see abnormal ejection fractions, for example, where you can then monitor. Okay.
Stability, and...
More or less preventing worsening in these boys. Okay, so...
Speaker Change: The fact that we have patients already enrolled that are older, I think we're in a better position to monitor this over a long time and we have patients already out.
Speaker Change: 9 and 12 months and further as we go out further, but it's really something that takes more time for sure than a year even if you're looking in the older age group before you anticipate seeing a potential drug effect.
Your other question about wedding M.D. and our pivotal trials.
for Subrattinal Delivery, whether we consider cutting short the enrollment. Thank you very much.
Speaker Change: We would not, and Abby would not as well. One of the key reasons we increased the size was to be able to go global.
Speaker Change: and you can imagine with a partner like Abby, we want to do things right, we don't want to skimp especially when it comes to fully characterizing safety and efficacy. So this is a unique opportunity to compare to...
Curran Simpson: To establish, to prove treatment regiments, to really inform the clinical community, not just the regulatory bodies. So, our view would not be to cut these short. And as Curran has summarized, we look forward to completing enrollment this year and being able to. Thank you very much.
have results next year.
Speaker Change: And there is a lot of trial competition out there in the wedding empty space with, as you mentioned, some of the longer acting TKI's
Speaker Change: One of the things we're encouraged about is that this is really a paradigm shift. This is not a matter of incrementally increasing injection. [inaudible]
Speaker Change: Bird and Reduction from every two or every three months, every four or every six months [inaudible]
It's really being able to in a...
Potentially a majority of patients prevent the need for injection. So...
Speaker Change: We're seeing that as a strong differentiator and that's again why we are pleased that we're going to be able to complete enrollment this year.
Speaker Change: One thing I would add too, we are certainly cognizant of timing and moving as quickly as we can.
to a BLA filing, and that's one.
Speaker Change: Reason that Abby, for example, will be doing the preparation and filing of the BLA. They have upwards of 700 people in their regulatory group.
Speaker Change: and so these are very large study and we're going to rely on Abby who has...
Speaker Change: I think a great track record of speed to be L.A. from top line data. Data,
Speaker Change: So that's something to follow in the next year. We're with you in terms of going as fast as possible. As Steve mentioned, we want to do it the right way. Thank you very much.
Thanks so much for taking the questions.
and many more. Thank you for watching. I'm Chris Chappell.
Thanks.
Speaker Change: Our next question comes from a line of Annabel Samimy with Steeple
Hi, thanks for taking my questions, just following on...
Speaker Change: The regulatory discussions for DR. Are there any specific key areas of difference between the U.S. and the U.S. regulators that we should be thinking about? I know that the pathways pretty clear for the U.S. curious about O.U.S. I know that the U.S. and the U.S. should be thinking about O.U.S.
and then on DMD for the upcoming...
Speaker Change: Not the must-go-distrophied association, but the one where you're going to be disclosing functional data. Will we be seeing just a follow-up from patients we've already disclosed? Or will we see new patients of the 11 that had already been treated? Yes, we're going to see new patients of the 11 that had already been treated.
Speaker Change: and then finally for DMD, the 50% enrollment, can I just...
Speaker Change: Can you just clarify that this includes, whether this includes new patients that were enrolled into the pivotal or does it also include the patients from the phase one to study? Thanks.
Great.
Speaker Change: Yeah, I'll take the MD first, and I think we'll let Steve comment on he's part of the joint development team with Abby that's conducting these regulatory meetings and can comment. For Dushan, the data that we're speaking to around functional updates later this first half.
Speaker Change: will be all of the above. So patients that we've already discussed at dose level two that were at nine months moving out to 12.
Speaker Change: New patients that have not previously been reported in the dose level two, Phase one, two study, likely at nine months, and then also long-term data on the dose level one patients.
Speaker Change: that will be out at least to 18 months, depending on the timing, specific timing of the update. So that's what we're...
Speaker Change: Planning for, we don't have a specific number for new patients yet but as you know from the past there's seven patients total in that cohort. Thank you very much for your time.
Speaker Change: So we're just incrementally adding, hopefully meaningful updates, not patient by patient enriching that data set as we go.
Speaker Change: I think to the question of does the pivotal enrollment include new patients? The answer is yes.
Speaker Change: We have new patients that have been enrolled since we announced that we were recruiting for the pivotal trial and also some of the patients that were dosed actually the majority of them that were dosed in
Speaker Change: The Phase 1-2 study would also be eligible as part of the pivotal data set and just recall that's an end of 30 that we're driving forward to complete that study.
Thank you. Thank you. Thank you.
Speaker Change: Hi, Annabel, I'll take the first question now on diabetic retinopathy and the pivotal plan. As you mentioned, fortunately there's a clear path from precedent of
Getting indication for treatment of diabetic retinopathy from...
Speaker Change: Drugs like Lucentus and Ilaya, where what's been accepted and what's still accepted is use of the diabetic retinopathy severity scale.
Speaker Change: which has been clinically validated to predict bad outcomes for patients with diabetic retinopathy.
Speaker Change: And we know the path is there but these drugs are not being used because of the significant treatment burden that's needed to keep the disease at bay. So that's important because that means it's still open the ability to use a negative control. [inaudible]
Speaker Change: So, for example, in our case, the potential to use a sham control arm...
Speaker Change: where we know that those patients don't magically get better, and in fact they, on average, get worse.
Speaker Change: So it allows us to power a study with the traditional one-year end point.
to look for a difference.
in active treatment compared to a negative control arm. [inaudible]
Speaker Change: There isn't that known precedent for Europe , so that's one of the reasons we want to work with the EMA and also in Japan. We think there's a very solid case.
Speaker Change: for use of the diabetic retinopathy severity scale. And in fact, the more years that go by and the more studies that are done, we think that case gets stronger. And so we look forward to in the future giving an update on discussions that we've had with regulators around the world. Thank you.
Thank you [inaudible]
Brian Scorney: Our next question comes from a line of Brian Skorney with Baird
Brian, you may be on mute.
and many more. Thank you.
You're right. Off you go.
Speaker Change: Thanks for taking the question, everyone. You've now submitted a VLI for Hunter with Sieber, had discussions on a suitable program for DMD and planning a program.
Speaker Change: for Diabetic Retinopathy. You know, we get a lot of questions about continuity as it's fever, given the change of guard at HHS and FDA, and I don't really know anyone who's probably had more interaction with fever than you guys over the last couple of months. I'm just wondering, you know, with Celia Wettings department, sure a couple of weeks ago, if you could give us a sense as to how it could date your discussions with fever and OTPR, and if you're seeing significant change within the review division, [inaudible]
Speaker Change: Johnson, Therapeutic Products, and how much that could potentially impact future gene therapy reviews.
Thank you. Thank you.
Speaker Change: Yeah, I think our general comment is from our perspective it's business usual in terms of the submissions that we're making we're
Speaker Change: on a pretty frequent basis, providing amendments to protocols, amendments to INDs, and...
Speaker Change: Those are, you know, we're getting questions back and forth on certain things. So,
Speaker Change: We're really not seeing a significant difference, either in timing of responses or availability of the teams. And I think we were...
Speaker Change: encouraged by some of the comments, especially on the rare disease side around accelerated approval from McCary, and just the overall sentiment.
to date, I think we feel...
Speaker Change: Number one that we've got really good connections and really good previous dialogue with the review team the review team seems stable from what we can tell from those interactions. Questions?
Speaker Change: and therefore we don't see any change in sort of the risk profile of our pending BLA now and ongoing discussions for Duchenne.
Speaker Change: And maybe if I could ask a follow-up on DMD. Now that you're about 50% enrolled into the 202 Pivotal, can you give us any flavor as to how screening kind of goes? Are you seeing any particular favoritism by age or characteristics in terms of patients looking to screen? I mean, in particular, I'm wondering if, like, the availability of a lot of this and a lot of this is data, if it's sort of like, you know, a four- to five-year-old's favorite coming in.
Speaker Change: To be screened for gene therapy, or because the data strongest for a love at SDC, sort of in that age group, do you see patients just coming outside of that 405, you're all one of the band-of-bases?
Speaker Change: Yeah, up, generally comment, then I'll let Steve maybe discuss because he does see the screening logs in more detail than I do.
Speaker Change: One of the reasons that we wanted to point to...
Speaker Change: Nearly half enrollment of the pivotal study is that, in addition to that, [inaudible]
Our screening logs and patients testing for neutralizing antibodies.
It's a significant number of people, of patients being screened. [inaudible]
Speaker Change: and that just points to the strong interest we see from the patient community.
Speaker Change: and from the Duchenne patient advocacy groups as well that are supporting us. Steve, I don't know if you want to comment, are we seeing any difference in terms of the ages or is it just a broad distribution? [inaudible]
I'd say it's a broad distribution, Brian , I think.
Families,
Speaker Change: across the age range have certainly different needs or different ways they'd look at evaluating different treatment options.
I think that's where our differentiation comes into play.
Speaker Change: So, I think, certainly the micro-disturpin levels that we're seeing, that's clear differentiation in the eight-nolder [inaudible]
Speaker Change: As you mentioned, the 4-7, and particularly the 4-5 is really the subset of patients where there's the most evidence for a levitas. I think one aspect that is a differentiator across the spectrum is safety.
Speaker Change: So safety first, that's certainly how investigators think about it and that's how patient families, of course, think about it, so. So.
Speaker Change: We believe with various aspects of our program, both our proactive immune suppression,
Or high purity levels.
that are very differentiating.
Speaker Change: And overall, what we're seeing is very good safety. We have seen no SAEs, no AEs of special interests, so that includes no LFT elevations, no thrombocytopenia, and these are findings that have...
Speaker Change: Been seen in other programs and with elevators, I think upwards of 40% have LFT elevation so that's a clear differentiator that we think is important across the age range. [inaudible]
Certainly in the three-and-hundred bucket that's...
Speaker Change: Those are patients who do not have access to approved gene therapy in the US.
Speaker Change: One of the aspects we're excited about our program is some of the not just micro-dissriff and expression, but also...
Speaker Change: Dr. Genome copies per cell that we're actually seeing, which are much higher than has been reported by any other program. So, we think that gives added confidence when you think of that younger age group where there still be. [inaudible]
Samoffel, Cell Division, so.
Speaker Change: In short, in summary, we see good differentiation aspects that are compelling for doctors and patient families to consider. And that's why we're confident we're going to get a widespread, which is a goal given the broad patient population we want to have safety and efficacy in.
Speaker Change: I think particularly if you think about it 95% of the prevalent market is still available for these studies so I think that plus the
Speaker Change: The focus on next-generation therapies is really helping with enrollment so we're excited where we are this early in the year and I can guarantee we're pushing to go as quickly as we can on all fronts for the BLA submission.
Thank you.
Speaker Change: Our next question comes from a line of Paul Choi with Goldman Sachs
Thank you.
Paul Choi: Thank you. Thank you. Good afternoon and thanks for taking our questions. I want to ask on affinity and how you're thinking about your your post trial or post approval commitment and just your your thoughts on the sort of requirements that I guess. Thank you very much.
Paul Choi: Down the road for conformatory study or other regulatory requirements and just your thoughts there.
Paul Choi: And second, based on the data you're seeing to date in the ambulatory population, I want to see if you, you know, what's the current appetite to potential explore 202 in a non ambulatory population at some point, even if it's an exploratory study, just just your thoughts there would be great. Thank you for taking our questions.
Speaker Change: Thank you for watching. Please subscribe to our channel. And as always, I'll see you in the next video.
Great, thanks. I think in terms of confirmatory study [inaudible]
We're...
Speaker Change: We're going to have that discussion in more detail with FDA in terms of what's required but I think in general...
Speaker Change: We intend to continue to enroll post-enrollment of our Pivotal and equals 30 data set.
Speaker Change: towards the end of completing a confirmatory study as quickly as possible. And we'll have further discussions with FDA in our pre-BLA meeting about what exactly that will mean.
Joseph, I think...
The second question is...
Speaker Change: Remind me. So on non-ambulatory. Yeah, so we are considering. Thank you very much.
Yeah, we're certainly considering strongly... [inaudible]
conducting studies in nonambulatory patients.
Speaker Change: But right now, not at the expense of going as fast as possible for the ambulatory, one and older patients that we're conducting as part of our clinical studies now.
Speaker Change: It is definitely a consideration given the size of the prevalent market there, but we just don't want to use our sites for that purpose at this point given that we want to close out our pivotal study as quickly as we can this year. [inaudible]
Speaker Change: Given the excellent micro-districted levels and also functional results that we're seeing in eight and above, including a 12-year-old, it's...
It's an interesting dilemma, really like Curran said, we got to focus on. [inaudible]
The population for our pivotal, but...
Speaker Change: More and more once we show that data investigators and also patient advocacy are asking about non-ambulatory because it's quite logical given the results we've seen so it's something we'll be looking at but not acutely adding to the pivotal.
Speaker Change: Right, once I got what I add, just to finish the question on planning for...
Confermentary Study, [inaudible]
Speaker Change: We do have drug supply already prepared to cover both the pivotal study and a future confirmatory study based on some planning assumptions that we've made. So I think that's really important that we can seamlessly move into a confirmatory study with existing drug supply. Bye.
Ellie Merle: Our next question comes from a line of LA Merle with UBS.
Thank you. Thank you.
Speaker Change: Thank you guys. Thanks for taking the question. Just a little bit more on the functional data.
Ellie Merle: Specifically, what should we be looking for at this upcoming data update?
Speaker Change: Both of this update, but then thinking longer term both from the pivotal study and then potential confirmatory, what do you think would be differentiating in terms of functional data?
Speaker Change: And then in terms of the pivotal study, I guess, how do you plan to analyze some of this functional data? Do you have any stats protocol for say doing a natural history comparison? And if so, is this something that you've discussed at all with the FDA? Thanks.
Speaker Change: and others. We wish you a Merry Christmas. We wish you a Merry Christmas. We wish you a Merry Christmas.
Steve Pakola: Great. Y'all covered just the general specifics of the functional updates and then maybe you'll let Steve talk about the analysis of the data versus baseline and the external match controls that we're doing.
Speaker Change: I think in terms of the near-term functional data, as I mentioned earlier, you should expect to see additional patients that were treated at dose level 2 in phase 1-2 study.
Speaker Change: Will be specific about the number of patients more around when their visits occur and when the data.
Speaker Change: is updated. We'll also report 12 month data on patients that we previously reported. There were two at nine months in that release. And also...
Speaker Change: We will update patients dose that dose level one that were at 12 months who are now likely out to about 18 months, maybe maybe two years on some longer term data, but of course that's not at the pivotal dose so it'll be just informational at that point. [inaudible]
Speaker Change: So that's what you should expect to see near-term and then over the course of the year I would expect to see most of the data for all seven patients that were dose that dose level two out to 12 months, but that'll be much later in the year.
Thank you.
But as far as what we've...
Speaker Change: We'd see for what we'll look at will be the usual suspects.
Speaker Change: L.E., as far as time function tests, so time to stand, 10 meter walk run, time to climb, and NFA, which traditionally has been...
Speaker Change: Viewed as less sensitive than the time function test, but actually in our November update we saw encouraging results not just on the time function test, but also. So.
Speaker Change: on the NSAA, so we're excited to see if we can confirm those findings on longer follow-up on the existing patients and then.
Increased the sample of patients.
Speaker Change: at dose level two, what we see on those. We'll continue to look at this the way we've done in the past, which is not just in isolation, but compared to matched external, natural history controls. [inaudible]
Speaker Change: So that's really important given disease trajectory is different depending on age and baseline.
Speaker Change: Severity, so we match on all those factors to give greater confidence in terms of whether individual patient's trajectory is positive or not. We have discussed this with the FDA, so this was certainly a part of the successful end-of-phase-2 meeting.
Speaker Change: that we held before starting Pivotal and we had no issues getting into the Pivotal design.
Cool, thanks.
Speaker Change: Our next question comes from the line of Sean McCutcheon with Raymond James [inaudible]
Subs by www.zeoranger.co.uk
Speaker Change: Hey guys, thanks for the question. One for me on NPDR, Steve Tierpoint, we've seen a lot of some reticence from patients to receive anti-budget therapies due to that high.
Ardenart, all of the frequent injections.
Speaker Change: And you've shown some meaningful decreases in vision threatening events up to 12 months in altitude at the higher dose.
Speaker Change: in line, or better than the panorama study of Flibersuppt. What do you view as the treatment durability at which we could start to see more utilization in NPDR, and how do you view that as distinct for a gene therapy versus say a sustained release TKI? Thanks.
[inaudible]
Speaker Change: Sure, that's a great question, Sean, and I think that is the key observation that we've seen is that even though repeated anti-vedgap injections
Definitely work.
Speaker Change: It's just too much of a burden for patients and doctors to give injections indefinitely because once you stop giving the injections [inaudible]
Speaker Change: The severity comes back, so you're basically signing patients up who are currently asymptomatic to get injections, the rest of their life basically. And that's really the barrier also to even longer durability TKI's. [inaudible]
Speaker Change: since it's certainly better to only need an injection every six months or even every year, but if it's repeated injections we see that as. [inaudible]
Speaker Change: A Major Barrier, and that's why we in Abbey are so excited about the DR indication because of Supercroidal in office.
One-time injection.
Is really the way to address this massive unmet need. [inaudible]
Speaker Change: And that's why we're excited with the data that you refer to where...
Speaker Change: We've shown an 89% reduction in vision threatening complications at a year, so that really was the impetus for us in the AV accelerating the end of phase two planning. Now that we have. [inaudible]
Speaker Change: The positive outcome of that study that we're both excited to advance into pivotal this year.
Speaker Change: Our next question comes from a line of Alec Stranahan, which bank of America? [inaudible]
Thank you. Thank you. Thank you.
Speaker Change: Hey guys, thanks for taking our questions, maybe two from us both on DMD.
Speaker Change: I appreciate the update on the pace of enrollment in Infinity Duchenne I guess how long after completion of enrollment I think we could see data would 12 week micro just for the primary be enough maybe for the top line or would you want to wait a bit longer for some of the other end points?
Speaker Change: And second, I'm sure you guys are tracking the devolving commercial market in DMD pretty closely. I guess maybe following up on a previous question.
Speaker Change: What's your expectation around the market dynamic in say a year or two when you're a bit closer to approval? Any updated thinking around, you know, current supply and the addressable market at launch would be great. Thank you.
In terms of
Speaker Change: The second question I think I'll take first. I think one of the key aspects of our plan is that
Speaker Change: We think at least half of the prevalent market will still be available by 2027, which is what we're targeting for a potential approval. And if you look at the updated guidance now
Speaker Change: We're pointing towards a mid-2026 BLA filing. So that could lead to an approval, essential approval in early 2027, which I think is really important.
Speaker Change: and we're already planning how to address the market. We'll be making our first lots that are eligible for commercial sales starting this fall.
Speaker Change: and we have the capability with our in-house manufacturing stockpile, a significant number of doses that had of the launch. So...
Speaker Change: Assuming we can establish a broad label, it would expect we're going to be very aggressive commercially in 2027 to take a significant amount of the prevalent population in terms of market. . .
Speaker Change: And then Alec, as far as your first question about when would we have actual results that we could disclose?
Speaker Change: The 12-week time point that is our accelerated approval endpoint time point for microdistriction. And so,
Speaker Change: That's a very significant step, so we certainly would be coming out with that data since that's really what's going to drive actually going ahead with the BLA submission by mid next year already so. Bye.
Speaker Change: We'd also have safety at that time point as well, so I think that really puts us in a good position.
Speaker Change: I think if you think about what functional data would we have in hand at the time of filing? [inaudible]
Speaker Change: Certainly the full phase one, two, dose level two cohort would be past 12 months at that point.
Speaker Change: and a good proportion of the patients treated in the pivotal study.
Speaker Change: We'll have at least nine months, is not 12 month data, so we'll have a healthy level of functional data to accompany.
Speaker Change: The primary endpoint of Microdistrophin at the time of filing and probably be able to update that somewhat at the 120-day safety update as well.
Got it, makes sense. Appreciate the color. Thank you.
Our next question comes from Yi Chen with H.C. Wainwright
Yi Chen: Thank you for taking my question. Could you come on your phone, Jean-Hack, who's seriously in presence in the US? How many of them will be promoting the Ajax 1-21? The Ajax 1-21?
Speaker Change: and do you expect that any sales related milestone could be triggered within 12 months of commercial launch? Thank you.
Speaker Change: Didn't quite hear the first part. Could we repeat just the beginning of that question? I got a little bit muffled. Yeah, the audio was just a little rough.
Speaker Change: The sales team presence of the Bonshinakos in the U.S., how many of them will be promoting RGX 121 for proof?
Two [inaudible]
Speaker Change: Yes, Niphan Shia, who has a significant U.S. presence in rare disease. They have an existing sales force that services their Duchenne.
Speaker Change: Launch, and we expect that majority of those, that same team would be applied towards commercialization of our GX-121.
Speaker Change: You know, given the timeline now that we've filed the BLA, we're pointing towards Padufa Day somewhere in Q4 of this year.
Speaker Change: and that would point to a launch either late this year or early. [inaudible]
next year, of course, and feel positive that...
Speaker Change: The partnership with NS Farmo allows us to do that as rapidly as possible. Good alignment initially with them on...
Speaker Change: Site Selection for Commercial Sale. There are dosing centers that we've worked with clinically that we feel are obvious choices that have also previously handled commercial products.
Speaker Change: So more to follow as we go through the review process on the BLA.
Speaker Change: In terms of achievement of sales milestones, the milestones, there are some that could be realized in the first year.
Speaker Change: of Sale, they're sort of staggered throughout the growth of the product. We haven't commented previously on what proportion that might be of the 700 million total potential milestones, but some of those could be realized in the first or second year of launch.
Thank you [inaudible]
Thank you.
I'm showing no further questions in queue at this time.
Speaker Change: Disconcludes today's conference call. Thank you for participating. You may now disconnect.