Ladies and gentlemen, thank you for standing by, and welcome to today's joint conference call with Solid Biosciences and AavantiBio to discuss their strategic business transaction. Please be advised that today's conference may be recorded. I would now like to hand the conference over to Caitlin Lowie, Vice President of Communications and Investor Relations at Solid Biosciences. Ms. Lowie, you may begin.
Good morning, and thank you, operator. Before we begin, I would like to remind everyone that this discussion and the accompanying presentation will contain forward-looking statements based on the current expectations of Solid Biosciences and AavantiBio, including but not limited to, statements regarding the expected timing, completion, effects and potential benefits of the proposed merger and private placement of our future expectations, plans and prospects for the combined company. Such statements represent management's judgment and intention as of today, and involve assumptions, risks, and uncertainties. Actual results could differ materially from those discussed in these forward-looking statements due to a number of important factors, risks and uncertainties, including those risks set forth on slide two of the accompanying presentation and other risks described in the Risk Factors section of our most recently filed annual report on Form 10-K and other periodic reports filed with the SEC.
Solid Biosciences and AavantiBio undertake no obligation to update any forward-looking statements after the date of this call. Further, as indicated on slide three of the accompanying presentation, Solid Biosciences intends to file a preliminary and definitive proxy statement with the SEC relating to the proposed merger and private placement. Please be advised to read when available, the preliminary and definitive proxy statements and the other relevant data, documents filed with the SEC, as these will contain important information about Solid Biosciences, AavantiBio, and the transaction. Once available, these documents can be obtained free of charge from the SEC at sec.gov or on the Solid Biosciences website.
With me today on the call are Ilan Ganot, Co-founder, President, and Chief Executive Officer of Solid Biosciences. Bo Cumbo, President and Chief Executive Officer of AavantiBio. Stephen DiPalma, Solid's Interim Chief Financial Officer. Dr. Carl Morris, Solid's Chief Scientific Officer, and Dr. Jenny Marlowe, Chief Scientific Officer for AavantiBio. During today's call, we will share details of a strategic update the company announced this morning. Yesterday, Solid entered into a definitive merger agreement to acquire AavantiBio, a privately held gene therapy company.
Solid also entered into a securities purchase agreement with a select group of healthcare and biotech investors for a $75 million private placement that is expected to close concurrently with the closing of the merger. We'll begin with opening remarks from Ilan Ganot, who will share details of the planned acquisition of AavantiBio, followed by Bo Cumbo, who will discuss Solid's strategic path forward. We will then open the call to questions. Before I turn the call over to Ilan Ganot, I want to acknowledge that materials related to these transactions were filed with the SEC this morning.
I'd like to now turn the call over to Solid's CEO, Ilan Ganot. Ilan?
Thanks, Caitlin, and thank you all for joining us this morning. When we founded Solid nearly ten years ago, we wanted to identify meaningful treatment options for the boys and families who live with the devastating consequences of Duchenne muscular dystrophy. Over that time, Solid has experienced many ups and downs, but our commitment to patients and our mission has remained our guide. I personally have had the privilege of watching great science be conducted by our team and the lives of boys who have been treated with our product candidate in clinical trials be improved. As the leadership team, Board and I have discussed how to position Solid for success in the future and ensure that our treatment can reach patients, we recognize the number of challenges that we were continually facing. One, manufacturing is a critical factor in the development process.
Two, a single disease focus with a single asset exposes us to greater market volatility and risk. Three, Solid has produced some interesting and innovative technologies but were not able to explore their potential with patients. In April, we began to address the first challenge when we announced our intention to focus our manufacturing on a single manufacturing methodology, transient transfection, because we believed it would improve our consistency of supply as well as potentially improve the quality of the product candidate. That decision has thus far proven to be a positive one. Today's announcement of the proposed merger and private placement, I believe, will help address the last two challenges. We believe that diversifying Solid's business into more disease areas will allow us to open doors to new investment and create a more stable business model to move our Duchenne program to patients.
AavantiBio emerged as an ideal partner and a group of healthcare and biotech investors agreed to commit $75 million in a PIPE that was announced alongside the acquisition of AavantiBio. First, a bit about the transactions before I hand it over to Bo Cumbo, who will assume the role of President and CEO of Solid upon the closing of the transactions. The acquisition of AavantiBio is expected to strengthen and expand Solid's pipeline and add organizational capacity to help advance our programs into and through commercialization, and hopefully ultimately to patients. AavantiBio has been developing a gene transfer product candidate for Friedreich's ataxia or FA, another neuromuscular disease as well as a product, as well as product candidates for cardiac indications, including BAG3-mediated dilated cardiomyopathy. Together, we believe that Solid has the potential to become a leader in gene therapy for neuromuscular and cardiac diseases.
When discussions began with Bo Cumbo and the AavantiBio team, it was clear early on that there were some natural synergies. First, AavantiBio has some great talent that would complement the leadership team we have here at Solid. As you will see, the new team has strong leaders in all areas of the organization. Second, the companies have similar pipelines. Obviously, Solid is focused on Duchenne, a neuromuscular disorder that has cardiac manifestation. There are many similarities between Duchenne, Friedreich's ataxia, and the cardiac programs that AavantiBio is developing. We're also both working to identify promising next-generation AAV capsids. Finally, both companies have found themselves in a similar place with manufacturing. Just as we have begun to focus on transient transfection-based manufacturing as our manufacturing platform, AavantiBio has made a similar decision to switch from HSV to transient transfection.
We believe we can drive consistent drug product with desired quality attributes by having a single specialized CMC team working across the programs. All these activities will support our goal of having multiple programs in the clinic in the coming years, leading with SGT-003 for Duchenne. Now, a few details on the transactions we announced in the press release earlier this morning. Solid signed a definitive agreement to acquire AavantiBio concurrently with a $75 million private placement in Solid. The private placement will add to the combination of existing cash from both Solid and AavantiBio. The merger is an all-stock transaction where, upon closing, AavantiBio shareholders will own approximately 15% of the combined company. Upon the closing, the combined company will operate as Solid Biosciences under the ticker SLVB, and AavantiBio will become a wholly owned subsidiary of Solid.
The merger has been approved by the boards of Solid and AavantiBio, as well as stockholders of AavantiBio. We expect the merger to close by the end of this year, subject to approval of Solid stockholders and other customary closing conditions. The $75 million PIPE will close concurrently with the acquisition of AavantiBio. In the PIPE, we agreed to sell approximately 159 million shares of common stock at $0.47 per share. Our lead investors in the PIPE are Perceptive Advisors, RA Capital Management, and Bain Capital Life Sciences. Other new and existing investors include Casdin Capital Management, Invus, Laurion Capital Management, and Pura Vida Investments.
Finally, we expect the combined company to have cash and investments of approximately $215 million at close, which we expect will be sufficient to fund operations and capital expenditures into 2025 and through important milestones for the lead programs. With that, I'm going to hand it over to Bo Cumbo. I've known Bo Cumbo for nearly 10 years now. I've also worked closely with Bo Cumbo recently as we have discussed how to bring these two organizations together. Through that process, we discussed the future leadership of the company as I was thinking about my next phase. Bo Cumbo has significant experience bringing products through development and commercialization, and I believe he is a strong leader for this next phase of Solid.
I look forward to working with Bo Cumbo in the future, both as a member of Solid's board of directors and as a strategic advisor during this transition period.
Thanks, Ilan. I'm really glad to join you today. While I've been out of DMD for multiple years and had not anticipated coming back into the Duchenne space, I'm truly excited to help the children, young men, and their extended families suffering from the consequences of Duchenne. I'll share what the company will become and what we expect to happen in the next couple of years. As Ilan mentioned, the core of the company will be built upon three pillars: people, pipeline, and process. I'm going to walk through each of those. First, a bit about the combined company. We will be headquartered in Charlestown, Massachusetts, under Solid's roof. They have great R&D and analytical and process development labs. We also will leverage AavantiBio's vector core, located in North Carolina, that can produce material for small-scale studies while our PD teams can stay very focused on our lead programs.
We value AavantiBio's current relationship with the University of Florida, and we intend to maintain a research presence in Gainesville. As Ilan noted, this merger would significantly diversify Solid's pipeline. All of the programs in the combined company's pipeline focus on rare diseases that are estimated to have greater than 5,000 treatable patients in the United States alone. As we look out over the next few years of the combined company, we have some key anticipated milestones we aim to achieve. First, we expect to close the transaction by the end of this year following a Solid shareholder vote. In mid-2023, we anticipate an IND submission for SGT-003. You probably saw in the press release that SGT-003 has been prioritized over Solid's first-generation program, SGT-001, which will be paused. We will get into that in a minute.
After having lengthy discussion with Solid's leadership team these past few weeks, I'm very supportive of their decision to make SGT-003 the primary focus of the company's Duchenne efforts moving forward. In addition, in the second half of 2024, we anticipate submitting an IND for our other lead program, AVB-202, for the treatment of Friedreich's ataxia. This is a neuromuscular disorder that has neurological as well as cardiac manifestations affecting muscle control and coordination, with possible loss of vision, hearing, and slurred speech. We also plan to initiate IND-enabling activities for AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, or BAG3, in 2024. BAG3 is a devastating disease that can ultimately lead to heart failure. We also plan to continue our effort to develop novel AAV cardiac-directed capsids for our emerging cardiac pipeline. We're excited to highlight the executive team post-merger.
This group has extensive experience in genetic medicine and will be a major asset to the combined company as we move forward. After the closing of the merger, I will assume the role as the President and CEO. I've been in the industry for 28 years in specialty or rare disease companies. For the past two years, I've been solely focused on building AavantiBio and am extremely proud of the exciting work we have done. Before AavantiBio, I was at Sarepta Therapeutics for eight years, where I served as the Executive Vice President and Chief Commercial Officer. Prior to that, I worked at both Vertex and Gilead. The scientific foundation of the combined company is on the call today, Dr. Carl Morris and Dr. Jenny Marlowe. Carl will be our Chief Scientific Officer for neuromuscular programs.
He is a muscle biologist by training, has been in the industry for a very long time. Prior to Solid, Carl worked at Pfizer. Carl is well known in the neuromuscular space, and I've known him for an extended period of time and very grateful that Carl and I get to work together. Jenny is currently the scientific officer, Chief Scientific Officer at AavantiBio. She will be the CSO responsible for our Friedreich's ataxia and cardiac programs. Prior to joining AavantiBio, Jenny was the Vice President of Preclinical and Translational Development for bluebird bio. Jenny also spent 11 years in Novartis Preclinical Safety Group and is a molecular toxicologist by training. We have a muscle biologist and a toxicologist, which will we really sync up nicely for the work we need to accomplish.
Carl and Jenny will combine efforts and continue the work on our capsid libraries for both skeletal and cardiac tissues. It is important to have regulatory expertise on the executive team, and we will have one of the best in the industry with Dr. Jessie Hanrahan. Jessie is well-known globally and has worked with EMA, OTAT, and others. Jessie came to AavantiBio from bluebird bio, where she was Vice President of Regulatory. She's also been with Boston Scientific as well as Genzyme. Jessie also has an incredible team with experience in both regulatory strategy and CMC regulatory within gene and cell therapies. This team will help support all our efforts going forward. Paul Herzlich will be the Chief Technology Officer. Paul has decades of experience right where you want it, in gene therapy and biologics. Prior to AavantiBio, Paul was the Vice President of CMC at BridgeBio.
Prior to this, he was also at Novartis, LogicBio, Bamboo, et cetera. He has deep gene therapy experience and is very well-known within the CMC community. Dr. Roxana Donisanu will be the Head of Clinical Development. Roxana is a specialist within Duchenne and spinal muscular atrophy and is very well-known within the industry and Duchenne community. She was at Roche prior to Solid, and I'm very excited to have the opportunity to work with Roxana. Steve DiPalma, who's on the call today, has extensive experience as CFO in the biotech and pharma space and has been the interim CFO at Solid for the past couple years. He will stay with us while we recruit an experienced CFO in the Boston area. Ty Howton rounds out the team as the Chief Administrative Officer.
Ty currently serves as AavantiBio's Chief Operating Officer and will oversee legal, HR, and program management, among other functions. Ty's been on the executive teams of Vertex, Sarepta, and AavantiBio. His background is in law, and while he will be the head of legal, he also brings the experiences he's gained in 20-plus years at companies like AavantiBio, Sarepta, Vertex, and Genentech in areas including regulatory, preclinical, CMC, government affairs, and patient advocacy. This merger will also bring together all the pieces you need to be a leading genetic medicine company. We have the vector core up and running, as well as animal models and supporting natural history studies in place within the key diseases, and great scientists able to develop novel capsids, regulatory elements, and promoters.
The merger is also expected to bring together a network of research partners globally that will help support our ongoing research efforts for our pipeline programs. We believe this merger will put all the critical pieces together to create a leading gene therapy company, and our goal is to have multiple INDs in the coming years, leading with our next-generation microdystrophin program, SGT003, followed by AVB202 for FA. This will be the combined company's initial pipeline, bringing together gene therapy candidates from Solid and AavantiBio. I'm very excited about the combined pipeline. As you can see, we are targeting an IND submission for our lead program SGT-003 in mid-2023, with patient dosing starting in late 2023, pending IND's acceptance. AVB-202 for FA will follow that with an anticipated IND submission in the second half of 2024.
AVB-401 for BAG3 is currently in preclinical development and hopefully in the future we'll be in a position to share details on our undisclosed cardiac targets. I think everyone on this call knows Duchenne and how devastating this disease is and can be to patients and families. You also know that Solid has potentially the best in class microdystrophin construct containing the nNOS binding domain. I will spend a few moments on the announcement that Solid made this morning regarding SGT-003, notably to pause SGT-001 and prioritize SGT-003 as the lead Duchenne program. This slide shows the evolution of Solid's microdystrophin programs, starting with SGT-001, which was used in the IGNITE DMD study, to the second generation SGT-001 manufactured using a triple transfection process. Finally, Solid's next generation program on the right-hand side SGT-003, which will be the lead going forward.
As you know, SGT-003 delivers Solid's nNOS microdystrophin protein with a novel cassette. Using a reporter transgene, this cassette has shown a 10 times improvement in transgene expression compared to AAV9, a two times increase in muscle distribution compared to AAV9, and its liver detargeting, showing less than half the biodistribution to the liver compared to AAV9. We believe that SGT-003 could be best in class and offer improved microdystrophin expression at lower viral loads. Solid has been developing SGT-003 since the program was publicly launched in May of last year, including conducting non-human primate study, scale-up activities at the CDMO partner, and several confirmatory non-clinical studies. This slide shows some of the data that Solid has previously shared that demonstrate the benefit of novel cassette.
Ultimately, the team decided that SGT-003 now deserves the focus of Solid's Duchenne efforts in order to achieve the timeline we have set. An anticipated IND submission in mid-2023 and subject to IND clearance, patient dosing anticipated in late 2023. Over the next few months, we will complete current ongoing preclinical and manufacturing activities for SGT-001. This will allow us to pause SGT-001 when it's ready for IND filing. I'm spending time on this because I know many people have asked the Solid team about the two DMD programs and at what point they would go to a single program. I fully support the decision management has made to prioritize and progress SGT-003. As we've mentioned a few times, we expect to bring SGT-003 to patients in a clinical setting next year, pending an IND's acceptance.
Our immediate focus in the coming months will be able to complete our manufacturing and regulatory engagement activities to support our anticipated IND submission next year. Our second program is gonna be AVB-202 for the treatment of Friedreich's ataxia. FA is similar to Duchenne in that it's caused by a lack of a protein, frataxin. It's a multi-system disease that has both neurological and cardiac manifestations that can be severe. We plan to address the cardiac and neuromuscular manifestations administering AVB-202 via both intrathecal and intravenous routes. What is important about FA is that it's not just the amount of frataxin that is required, but it's the distribution of frataxin to the targeted locations throughout the relevant target tissues of the central nervous system, as well as the heart. AavantiBio's frataxin transgene fits very nicely within the cassette.
It is full length human frataxin with the addition of regulatory elements that are naturally occurring in the 3' UTR region. We are using the CBA promoter as well as AAV9. I will show you the iterations on how our program has evolved to what we believe has the potential to be the best in class gene therapy for the treatment of FA. Similar to what Solid did, we took multiple iterations and reviewed every aspect of our product candidate to make a potentially best in class program, AVB-202. Whether it was codon optimizing the gene, the manufacturing platform in which we moved away from HSV to transient transfection, adding in regulatory elements, and then AAV9 with a plasmid design that is optimized for yield and productivity. We have ended up with an optimized construct and delivery, which is shown on the right-hand side of this slide.
We will use this construct to advance IND enabling activities and manufacturing scale up using a transient transfection process to support an anticipated IND submission in the second half of 2024. Here is some of the preclinical data for AVB-202 we have collected. This model is frataxin deficient knockout mouse model. We performed experiments in this mouse model in both pre and post-symptomatic context to understand whether we could change the course of the disease after symptom onset or before. We're very pleased with the results in both models in which we observed that AVB-202 extended survival and improved cardiac function. We also understand from the preclinical studies that frataxin is functional in the dosed mouse mitochondria, 'cause you can look at the health of the mitochondria through this surrogate marker called succinate dehydrogenase, which we see return to normal levels post-dose.
Our third program is gonna be AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy or BAG3. The BAG3 gene codes for this BCL2-associated athanogene 3 protein or BAG3. If you don't have BAG3 protein or a reduction in BAG3 protein, you're likely to end up with dilated cardiomyopathy and ultimately heart failure. If you have this disease, you're likely going to seek treatment because your daily activities are severely impaired. We are using rh74 to deliver an optimized BAG3 transgene with a specific cardiac promoter. We are piggybacking off FA in Duchenne with transient transfection manufacturing process. There are a lot of synergies we expect to come together with this transaction, including the next generation capsid libraries the two companies are independently developing.
AavantiBio was working on discovering next generation, highly complex cardiac capsid libraries generated through a combination of rational design and random mutagenesis, screened via directed evolution in non-human primates using an RNA-based readout for positive selection from cardiac tissues and DNA for negative selection from liver tissue. Meanwhile, Carl and the team at Solid Biosciences were working on a ground up rational design approach to optimize capsids and drive expression to skeletal muscle while lowering transduction to the liver. We expect the combination of the two developed programs to create a large tissue-specific capsid libraries that we believe we can leverage for our own internal R&D programs, as well as potentially to out-license for non-core disease areas. Of note, on the bottom right of this slide, you can see the results from SLB-101, the capsid used in SGT-003.
Now, we believe that CMC is the foundation of the drug and we will focus our efforts on having the best in class CMC for all our programs. As I mentioned earlier, we have full teams, QA, QC, PD, AD, and great labs. We will also have CMC regulatory teams integrated in each one of our programs right from the beginning, so we always have an eye on CMC. This slide helps to illustrate why CMC is so important. I want you to look at just the first two columns, because there's only one thing that has changed from column one to column two. It's not the dose, it's not the capsid, it's not the transgene, it's the manufacturing process. Switching from an HSV to a transient transfection process increased the microdystrophin expression by approximately 1.5-fold.
Now, this is when column three comes into play. SGT-003, using the novel capsid, showed nearly two times increase in microdystrophin expression when compared to SGT-001, when both product candidates are manufactured using transient transfection. The only thing that changed between column two and three is using AAV-SLB101 capsid instead of AAV9. In summary, they observed two to three times better expression with SGT-003 using triple transfection compared to SGT-001 using the HSV process. AavantiBio and Solid Biosciences' respective manufacturing partners are currently in place for FA and Duchenne. As I mentioned before, AavantiBio's VectorCore is able to support smaller scale needs for BAG-3 and the other cardiac programs. Before we turn to questions, I want to review some of the combined company key milestones we discussed during today's call.
First, we expect to close the merger by the end of the year following a Solid shareholder vote, including a $75 million financing in connection with the business combination, providing a total cash position at the close of the transaction of approximately $215 million, which is expected to extend our runway into 2025 and through important key milestones for our lead programs. Next, we plan to report new data from the IGNITE- DMD Phase I/II clinical trial of our first generation program, SGT-001, in the early 2023. This will include the study's preliminary report of all patients to the primary endpoint of one year, as well as three-year longitudinal data for the first three patients treated in the high-dose cohort.
We anticipate submitting an IND submission for SGT-003 mid next year, and pending IND acceptance, we expect the dosing in late 2023. Finally, we currently expect to submit an IND for our FA program in the second half of 2024, which, subject to an IND clearance, will lead then to first patient dosing. In addition, we plan to move our BAG3 program into IND enabling studies, advance our early stage cardiac programs, and continue development work on our capsids. As you can see, I'm very excited about the opportunities in front of us and hope to build a leading gene therapy company in both neuromuscular and cardiac space. We have a talented team, a diversified pipeline we believe could be potentially best in class, and a strategic focus on a single manufacturing methodology.
We also expect to have a strong cash position that we expect will be sufficient to achieve the milestones just discussed. With that, I'll turn it over to Ilan Ganot to close.
Thank you, Bo, and I also wanna thank all the Solid and AavantiBio employees for their tireless efforts. You've all done important work for patients, and I look forward to seeing what these two teams can achieve together. Thank you all for dialing in. We can take some questions now.
Thank you. If you'd like to be placed in the question queue, please press star one on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star two if you'd like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing star one. One moment please while we poll for questions. Our first question today is coming from Joe Schwartz from SVB Securities. Your line is now live.
Hi, this is Beth on for Joe Schwartz. Congrats to everyone on this exciting advancement, and thanks for taking our question today. I was just curious if you could talk a little more about your decision to bring SGT-003 forward as your lead pipeline candidate. Specifically curious how you're thinking about the opportunity for SGT-003 in light of Sarepta's recent accelerated approval filing for their microdystrophin gene therapy. You know, any sort of learnings you can take from SGT-001 as you move SGT-003 into clinical development.
Yeah. Hi. Thanks. This is Carl Morris. Yeah, we've been going through de-risking of 003 all the way through. It got to a point where we were quite confident in where it's at. We received some guidance from the FDA recently around SGT-001 that sort of brought the timelines a little bit closer together and really made the strategic decision to move forward with our next gen program, 003. We've gone through our pre-IND. We've gone through the pre-IND meeting. We also have scale-up activities with our CDMO partner, Forge. We're in a good place to move forward with SGT-003 at this point in time and really made that decision.
I'll pass over to Bo Cumbo to talk a little bit about the direction.
Yeah. They've you know, just with regard to the Sarepta, I think they've made tremendous progress over the last couple years, and I think it's not only great for the industry, but also great for the children suffering from DMD. We'll continue to you know, they've laid out a very clear path, and we should try to follow it. We'll you know, move with lightning speed to try to get there with SGT-003.
I'll just add that, you know, we've always had this in mind, because the protein is the same protein between SGT-001 and SGT-003, and we believe that everything we're seeing in SGT-001 should give us all the confidence to move SGT-003 aggressively forward. Thank you for the question.
Great. Thank you.
Thank you. Next question is coming from Maury Raycroft from Jefferies. Your line is now live.
Hi. Good morning. This is Farzin from Maury. Congrats on the update, and thanks for taking our question. To clarify for 202 for FA, is there preclinical data in the slide deck? I'm guessing that's only tail vein IV injected. Can you clarify on that? And then can you also talk about what you're seeing in the NHP data and more on how the dual IV IT dosing would work?
Yeah. I think what I'm gonna do is I'm gonna turn it over to Jenny Marlowe in a second. We're, you know, you saw the preclinical data that the cardiac mouse model and, you know, we're continuing to do additional work for IND-enabling studies. The non-human primate data, this is for FA, right? Yeah. For non-human primate data is continuing ongoing. We're gonna have to continue to do additional studies. As I mentioned, we are switching over from HSV manufacturing platform to triple transfection manufacturing platform, and there will be compatibility studies that we'll have to complete as well. Jenny Marlowe?
Yeah. Thank you for your question. To answer directly the question about IV administration, yes, in that particular model, we dosed IV only. In our non-human primate model, we are dosing by the dual route of administration, both intravenous and by IT.
Got it. For the AVB-202 optimization and plasmid design, how do you see a differentiation versus other competitors in development?
For AVB-202, you know, I only know of really one company that's done preclinical work and made it all the way to IND, and that's Lexeo. I'm not exactly sure exactly their construct design compared to ours. But we've, you know, done a lot of work, as we mentioned on the slide, looking at codon optimization, adding in regulatory elements, moving to triple transfection. You know, I think, you know, it's a little too early to compare, but we're very confident in our program. We've done a lot of work over the last couple years, and we're gonna continue to do some more animal work. We're very confident. We understand exactly how this drug should react when we get to the IND.
Got it. Thank you so much.
Thank you. Next question is coming from Gena Wang from Barclays. Your line is now live.
Hi, this is Sheldon Monfortina. Thanks for taking our question. Maybe first question is could you remind us what are the remaining steps for the IND filing for SGT-003? Second question is, I think you mentioned the switching from HSV to a transient transfection was intended for reinitiate dosing with SGT-001. Could you give us some color on whether that switching on the Solid side right now is already completed? How would you coordinate the transition from HSV to transient transfection? Thanks.
I'll start just by saying that, you know, Carl's gonna mention some of the additional steps for the IND submission for 003. As far as 001 goes, you know, we have nine patients who have been treated. We really like the data, and it continues to get collected on annual visits. We're expecting three-year functional outcomes coming soon. We're very excited about the durability profile of the treatment, and so we would definitely expect to see that and more from 003. And as Carl mentioned, this was sort of like a process of an ongoing process of de-risking. You know, eventually the real answers come in the clinic, but as we learn more about 003, we learn more about the novel capsid.
You know, we went from in vitro to in vivo, and then in vivo and NHP data, in all in order to get comfortable for that to turn into our lead, effectively today. Carl can talk more about the specifics.
Just on the SGT-001 program, we are continuing with some of the non-clinical work, as well as the manufacturing work on the SGT-001 program. If something does delay or stop the SGT-003 program, we will have that ready to go. We are continuing getting it to a good pausing point. For the SGT-003 program, we've completed it. We sort of have guidance from the FDA via the pre-IND meeting. We are sort of in the midst of sort of working through the IND-enabling non-clinical work.
We have scaled up the manufacturing to a scale that can supply the clinical study. We're in the process of getting to the GMP and finalizing all the study reports so we can submit the IND as soon as possible.
Thank you so much.
Thank you. Next question is coming from Allison Bratzel from Piper Sandler. Your line is now live.
Hi. Good morning. Congrats on all the updates, and thanks for holding this call and for taking my questions. Could you just walk us through what assumptions are included in your 2025 cash runway guidance in some more detail? Also just walk us through the expected cadence of value inflection points you're anticipating for SGT-003 and AVB-202 through 2025. Does that contemplate clinical data is available for both assets by that 2025 timeframe? Thanks.
Yeah. Let's kick it over to Steve for the financial update.
Yeah. The runway would encompass important milestones in both programs. We do expect to have clinical readouts on 003 and also, you know, likely on 002 and FA as well into early 2025.
Yeah. I think just to answer your question, I mean, we're gonna, you know, some of the milestones that we'll have from an inflection point of the next couple of years, we'll obviously close the merger at the end of this year. As I mentioned before, we'll have an IND submission for the next generation SGT-003 mid-next year. We intend to dose patients in that SGT-003 program at the end of next year as well. We will, you know, start having patient data expected from that same program, SGT-003 in 2024. Then the IND from FA would take hold late 2024, and then we would start dosing patients, you know, soon after, whether through the runway or not. BAG3 will continue to move forward with IND-enabling studies.
Our other two cardiac programs will continue to move forward. Our capsid libraries, both on the skeletal side as well as the cardiac side, will have non-human primate data readouts early 2024. Then we can look to utilize those capsids for whether it's our cardiac, internal cardiac programs or out-license or non-dilutive financing.
Thank you.
Thanks. The next question is coming from Anupam Rama from JPMorgan. Your line is now live.
Hi. Thank you. This is actually Malcolm in for Anupam Rama. Congrats on the updates today, and thanks for taking the questions. Just one quick one. What can we be mindful of in terms of target IND submission for 003? Thank you.
Sorry. You broke up a little bit.
We couldn't hear the question. You broke up. Could you repeat it?
Sure. What are the gating factors that we should be mindful of, in terms of thinking about target IND submission for zero zero three?
Really it's just the completion and the readouts from the IND-enabling non-clinical work. We know what we need to do. We've been told what the FDA is expecting. So we're just going through the execution of those studies right now. Additionally, we just have to ensure that we can release GMP material. That's really it. We have sort of a protocol outline, and we're looking at startup, you know, starting up the sites. So I think everything's in place and on track for a mid-2023 IND.
Excellent. Thank you.
Thank you. As a reminder, that's star one to be placed into question queue. Our next question is coming from Geulah Livshits from Chardan. Your line is now live.
Hi. Good morning. This is Chloe for Geulah. Thanks for taking the question and congratulations on the transaction. Our question is you had guided to certain cadence of news coming from the SGT-001 program for later this year. You're gonna have a one-year update on patients seven to nine.
Right.
Potentially longer term update on those patients treated with SGT-001 later this year. I was wondering if that was still going to be the case. A second question on enrollment in the IGNITE DMD study. Based on conversations with regulators so far, what is your idea of how many more patients you'll need to dose before having a productive conversation with the FDA on the SGT-003? Thank you, sir.
For the first part, yes, we will be communicating the primary endpoint data, the 12-month endpoint data from the IGNITE DMD study, as well as three-year data from a subset. And that should come out, you know, sort of later this year, early next year, Q1 next year, as we've guided to. That hasn't changed. We are communicating now that we're pausing the 001 program. Given that we needed to file a new IND for the 001, it's unlikely we'll be sort of continuing with the dosing with 001 at this point.
All right. Thank you.
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