Selecta Biosciences, Inc.
Q3 2021 Earnings Call Transcript

Published: 9 Nov 2021

[Operator:]

Good morning. And welcome to the Selecta Biosciences Third Quarter 2021 Financial Results and Corporate Update Conference Call. Currently, all participants are in a listen-only mode. This call is being webcast live on the Investors & Media section of Selecta’s website at www.selectabio.com and it is being recorded. For opening remarks, I would like to introduce, Kevin Tan, Chief Financial Officer of Selecta. Please go ahead.

[Kevin Tan:]

Thank you and good morning. Welcome to our third quarter 2021 financial results and corporate update conference call. The press release reporting our financial results is available in the Investors & Media section of Selecta’s website, www.selectabio.com. And the quarterly report on Form 10-Q ended September 30, 2021, which was filed today with the Securities and Exchange Commission or SEC. Joining me today are Carsten Brunn, President and Chief Executive Officer; Peter Traber, Chief Medical Officer; and Kei Kishimoto, Chief Scientific Officer. During today’s call, we will be making certain forward-looking statements, including without limitation, statements about the potential safety, efficacy, and regulatory and clinical progress of our product candidates, financial projections and our future expectations, plans, partnerships and prospects. These statements are subject to various risks that are described in the filings made with the SEC, including our most recent quarterly report on Form 10-Q. You are cautioned to not to place undue reliance on these forward-looking statements, which speak only as of today, November 9, 2021, and Selecta disclaims any obligation to update such statements, even if management’s views change. I would now like to turn the call over to Carsten Brunn. Carsten?

[Carsten Brunn:]

Thank you, Kevin. Good morning. I appreciate everyone joining us today. In the last quarter, we made significant advancements that propelled our business and our existing pipeline forward, which has the potential to overcome immunogenicity, mitigated unwanted immune responses against enzyme or gene therapies and restore self-tolerance in patients with autoimmune diseases. We’re encouraged by the numerous strategic collaborations that have further validated our ImmTOR platform designed to mitigate unwanted immune responses across a range of diseases and by the opportunities to advance next-generation therapeutics. Further, these partnerships demonstrate the broad application of ImmTOR and we look forward to maximizing the value of the platform as we build on this momentum. This is a truly exciting time for Selecta, and yesterday, we announced topline results from the first in-human Phase 1 dose escalation trial of SEL-399, which we conducted jointly with AskBio. SEL-399 is an AAV8 empty capsids or EMC-101 containing no DNA combined with ImmTOR at doses of 0.15 mg per kg and 0.3 mg per kg. The randomized placebo-controlled double-blind dose escalation study conducted in healthy volunteers was designed to determine the dose regimen of ImmTOR to mitigate the formation of neutralizing antibodies or NABs against an AAV8 capsid used in gene therapies. A total of 23 healthy volunteers were enrolled, 14 males and nine females. All subjects had an anti-AAV8 NAB titer of less than 1 to 5 at baseline. The primary endpoints evaluated were safety and NAB and anti-AAV8 antibody titers. We observed a strong immune response in humans administered AAV8 Empty Capsids. The peak meet in anti-AAV8 NAB titer was 1 to 6,875 at 14 days often teasing, which was maintained at high levels through day 90. The addition of ImmTOR to administration of Empty Capsid was observed to reduce the formation of anti-AAV8 NABs in a dose dependent manner at day 30. Median day 30 titers of anti-AAV8 NABs were 50-fold and 250-fold lower into 0.15 mg kg and 0.3 mg kg into a cohort, respectively, compared to the median titer of control subjects dose with AAV8 Empty Capsids alone. At 30 days, subjects who received 0.3 mg per kg of ImmTOR, six of six or 100% exhibit in anti-AAV8 NAB titer 1 to 25 or less and four of six or 67% had a titer 1 to 5 or less. At 30 days subject that received 1.5 mg per kg of ImmTOR six of nine or 677% exhibit an anti-AAV8 NAB titer of 1 to 25 or less, and two of nine or 22% had a titer of 1 to 5 or less. At 90 days, two of six subjects in the 0.3 mg per kg cohort were observed to have sustained control of NABs with titers of 1 to 25 or less. Consistent with the clinical data, we observed that the single dose ImmTOR cohorts saw the late formation of NABs, eventually reaching similar median levels of NABs to the control group by day 90. Based on prior animal studies, we believe that if NABs are inhibited at day 30, administration of two additional monthly dose of ImmTOR has the potential to maintain control of NABs beyond day 90. From a safety perspective, there were no unexpected AAV’s related to ImmTOR. The most common AAV was mild to moderate stomatitis, which was ameliorated with steroid mouthwash treatment. We believe this data demonstrate ImmTOR’s potential to address one of the biggest current limitations in gene therapy, the inability to reduce lifesaving gene therapies, due to the formation of NABs against AAV capsids. We look forward to leveraging these findings across our wholly-owned gene therapy pipeline, and alongside our world class gene therapy partners to achieve our goal of improving the lives of those living with monogenic disease. The press release and webcast with additional detail can be assessed access in the Investors & Media section of our website. Next, I would like to highlight our recent business development activity and provide an overview of the key collaborations that have expanded our pipeline of novel therapeutics in combination with our ImmTOR platform. We entered a strategic license agreement with Takeda, a global pharmaceutical leader with expertise in rare diseases to develop targeted next-generation gene therapies with two indications within the field of lysosomal storage disorders. Together, we look forward to overcoming barriers to current efforts in AAV driven gene therapy, as well as striving to address immunogenicity constraints to an AAV re-dosing of potentially lifesaving gene therapies. Under the terms of the agreement, Selecta is entirely to receive an undisclosed upfront payment an up to $1.124 billion in future additional payments over the course of the partnership that are contingent on the achievement of development or commercial milestones. Selecta is also eligible for tiered royalties on future commercial sales. We are encouraged by the strong endorsement from large pharma and from other major players in the gene therapy space. To further address key hurdles in gene therapy, we announced an exclusive strategic licensing agreement with Genovis to advanced IdeXork or Xork, a next-generation IgG protease to enable the dosing of transformative gene therapies in patients with pre-existing AAV immunity and treat certain IgG mediated autoimmune diseases. But most IgG proteases are derived from human pathogens and have a high prevalence of precessing antibodies. Xork is derived from a streptococcal bacterial strain that does not infect humans. The combination absorb an ImmTOR has the potential to both mitigate pre-existing antibodies to AAV expanding access to gene therapy to a wider range of patients and de novo immunogenicity keeping patients eligible for retreatment. Additionally, ImmTOR derived IgG protease by themselves immunogenic. Currently IgG proteases can only be administered once, due to the formation of high titer antibodies against the protease itself. The combination of Xork and ImmTOR is further differentiated by the potential of ImmTOR to mitigate the immunogenicity of Xork and enable re-dosing of the enzyme, an important benefit for the application of each IgG proteases in autoimmune diseases mediate pathogenic autoantibodies. In relation to our enzyme therapy program, we announced a partnership with Ginkgo Bioworks or Ginkgo to design novel enzymes with transformative therapeutic potential to advance treatments for orphan and rare diseases. This partnerships built on our compare trial of SEL-212 in chronic refractory gout, which provides proof-of-concept data related to ImmTOR’s effects when combined with immunogenic enzymatic therapies. Under this disagreement with Ginkgo, Selecta gains rights to develop and commercialize select therapeutic enzymes from their advanced organism engineering platform to treat autoimmune diseases. We expect that our ImmTOR technology in combination with Ginkgo’s high throughput enzyme discovery, design and screening capabilities, will bring us one step closer to improving the sustained efficacy of novel biologic therapeutics. Finally, we established a protein engineering collaboration with Cyrus to radically redesign protein therapeutics. The lead program in the collaboration is a proprietary interleukin-2 or IL-2 protein agonist designed to selectively promote expansion of regulatory T cells or Tregs for the treatment of patients with autoimmune diseases and other deleterious immune conditions. Preclinical data investigating the effects of ImmTOR in combination with an IL-2 mutein demonstrates substantial synergistic activity in increasing the percentage and durability of Treg expansion in the spleen. This supports the potential of ImmTOR in combination with IL-2 proteins to restore immune intolerance to auto-antigens and forms the basis for this partnership. Collectively, these strategic partnerships provide additional validation and further demonstrate the robust value of our ImmTOR platform. This is truly an exciting time for Selecta and with our unique approach and strong cash position, we’re well equipped to maintain our leadership position in the target immune intolerance field. I’ll now take us through some of our key pipeline updates and upcoming milestones. First, a few key points on our enzyme therapy program, which has established an important framework for our clinical development path. SEL-212 was licensed to Sobi and is comprised of ImmTOR co-administered with our proprietary uricase to get replaced for the treatment of chronic refractory gout. As a reminder, our Phase 3 DISSOLVE clinical program kicked off in the third quarter of 2020 and consists of two double-blind placebo-controlled trials of SEL-212. In both trials, SEL-212 will be evaluated at two doses of ImmTOR 0.1 milligrams per kilogram and 0.15 milligrams per kilogram and one dose of pegloticase, 0.2 milligrams per kilogram. Each trial aims to enroll 105 patients with 35 at each dose and 30 sites on placebo. Enrollment is progressing on schedule and topline data from DISSOLVE is expected in the second half of 2022. We intends to leverage the success of SEL-212 for our second enzyme program indications in IgA nephropathy or IgAN, which is a kidney disease that occurs when immune complexes of an antibody called immunoglobulin A1 or IgA1 accumulate in the kidneys. Current treatments fail to address the root cause of the disease and we believe our novel approach, which combines ImmTOR with IgA1 protease, has the potential to remove injurious IgA from the kidneys and improve markers of renal dysfunction. We remain encouraged by the strong preclinical data in IgA and expect to find an IND for IgAN in 2022. Returning to our gene therapy program, as noted, immune responses to AAV gene therapy have historically precluded the ability to safely be dose AAV gene therapies and are a major consideration related to safety and efficacy in the space. The ability to inhibit the development of AAV specific antibodies has the potential to be transformational and shift the treatment landscape. We continue to advance our proprietary gene therapy programs and filed an IND for our lead gene therapy candidate SEL-302, which is a combination of MMA-101 plus ImmTOR. MMA-101 is an AAV gene therapy vector for the treatment of methylmalonic acidemia or MMA, a rare metabolic disease in which the body break down certain proteins and fats. As of the filing of this Q, the FDA’s 30-day review period for R&D to conduct a Phase 1/2 clinical trial of our SEL-302 product candidate in pediatric dictations with methylmalonic acidemia has expired. However, we have been informed early by FDA that they’re still considering certain aspects of a filing related to chemistry, manufacturing and control or CMC. We intend to wait for formal clearance from FDA before initiating the proposed Phase 1/2 clinical trial. Our second wholly-owned proprietary gene therapy candidate SEL-313 is being developed to treat ornithine transcarbamylase or OTC deficiency. OTC deficiency is an excellent genetic disorder caused by genetic mutations in the OTC gene, which is critical for proper function of the urea cycle. We expect to file a clinical trial application or CTA and/or an IND in 2022. We submitted a pediatric investigation plan or PIP for SEL-313 to the European Medicines Agency pediatrics committee in February 2021. Overall, we’re seeing excellent progress across our gene therapy program and we look forward to providing further updates later this year, as we continue to address the immunogenicity constraints in AVV-driven gene therapy, as we strive to overcome repeat dosing limitations by preventing the formation of NABs and as we enable more global and robust expression of the transgene after the first dose. Now moving on to our autoimmune program. Our autoimmune program is advancing through IND enabling studies and we expect to find an IND in primary biliary cholangitis or PBC in the second half of 2022. PBC is a chronic progressive autoimmune liver disorder that leads to inflammation, damage and scarring of the small bile ducts. PBC has a well defined target antigen, significant unmet medical needs and it’s well suited to the application of our ImmTOR platform. Autoimmune disease affects more than 24 million people in the U.S. alone and we look forward to expanding our pipeline as we continue to explore additional applications of our ImmTOR platform. Before we turn to the financial results, we have one final update to share. As we execute on our clinical and corporate initiatives, we continue to add talents and best to our leadership team and are excited to welcome Kevin Tan as Chief Financial Officer. Kevin brings deep financial expertise and experience in the gene therapy and rare disease landscape. This impressive track record in capital management and financings in both the biotech and investment sector will be invaluable as Selecta continues to pursue new partnership opportunities and advanced multiple assets through the clinic. As mentioned earlier, we’re extremely excited about the continued growth of our company and we remain confident in our platform. Now, I’ll turn the call over to Kevin to run through our financial results for the third quarter ended September 30, 2021.

[Kevin Tan:]

Thank you, Carsten. We remain well capitalized with $140 million in liquidity as of September 30, 2021, which compares to $140.1 million in liquidity as of December 31, 2020. We believe our liquidity will be sufficient to meet our operating requirements into the second quarter of 2023. Net cash used in operating activities was $28.9 million for the nine months ended September 30, 2021, as compared to $42.1 million of cash provided by operating activities for the same period in 2020. Revenue recognized for the third quarter of 2021 was $24.4 million, compared to $4.6 million for the same period in 2020. Revenue of $24.3 million was recognized under the license agreement with Sobi resulting from the shipment of clinical supply and a reimbursement of costs incurred for the Phase 3 DISSOLVE clinical program. The significant revenue increase is result of the continued enrollment of the Phase 3 DISSOLVE clinical program that was initiated in the third quarter of 2020. Additionally, during the third quarter, Selecta FDA recognized $0.2 million for the shipments under the license agreement with Sarepta. Research and development expenses for the third quarter 2021 were $21 million, which compares with $14 million for the same period in 2020. During the quarter ended September 30, 2021, there was an increase in expenses incurred for preclinical programs, salaries, headcount and AskBio collaboration costs. General and administrative expenses for the third quarter 2021 were $5.4 million, which compares with $4.4 million for the same period in 2020. The increase in costs was primarily the result of salaries, professional fees and stock compensation expenses. For the third quarter 2021, we reported net loss of $17.9 million or net loss per share of $0.16, compared to a net loss of $9.7 million or net loss of $0.09 for the same period in 2020. I will now turn the call back to Carsten for closing remarks.

[Carsten Brunn:]

Thank you, Kevin. In summary, the promising results from the pioneering Phase 1 SEL-399 clinical trial suggests that ImmTOR in combination with the vital use AAV8 capsid has the potential to overcome the significant challenge of the formation of anti-AAV8 NABs. Reduction of capsid immunity could be transformational for the field of gene therapy by making gene therapy safer and possibly enabling repeat dosing. Reflecting on this past quarter, we’ve made significant steps to progress our existing pipeline candidates and established collaborations to maximize the potential of our ImmTOR platform. We look forward to providing additional updates on near-term catalysts as we continue to drive our business forward, unlock the power of biologics and address unmet patient needs. I’d also like to thank the entire Selecta team, our investors and the many people who have been supportive along the way, including our patients and their families. With that, we’re happy to take questions.

[Operator:]

First question will come from Kristen Kluska with Cantor Fitzgerald. Please go ahead.

[Kristen Kluska:]

Hi. Good morning, everybody. Thanks for taking my question. The first one is that yesterday’s data answered a very important question related to your platform potential in gene therapy. So big picture, looking ahead into next year, what questions or areas in the pipeline do you expect to receive even more clarity on the potential of ImmTOR, whether it’s related to your preclinical programs, as well as some of the clinical work that’s being conducted by both yourself and partners?

[Carsten Brunn:]

Hey. That’s great question, Kristen. Obviously, we continue to advance our gene therapy programs. So we hope to be in a clinic with our MMA program next year and at least have some preliminary data around biomarkers of the disease and antibody titers. I think that’s going to be a key readout. We have a major readout for enzyme lead program in SEL-212, where we expect Phase 3 results in the second half of next year. And obviously, we’re moving forward a number of the programs that are currently in the pipeline, such as, for example, the OTC deficiency program we are to file -- plan to file an IND next year. But we’re also moving forward some of the exciting programs that we just start partnerships with specifically Xork, an IgG protease, which addresses another large unmet medical need in gene therapy. As you know, Kristen, up to 30% of patients have been exposed to a national AVV infection and have pre-existing antibodies are not eligible for treatment. So that’s -- we’re going to move that forward next year as well. So next year will be an exciting year for us.

[Kristen Kluska:]

Okay. Thanks. And now that you have clinical evidence for both the enzymes and gene therapies, and yesterday, you made it a point that the extensive preclinical work really helps with the design and understanding these results. So looking ahead into autoimmune now as those candidates near going into the clinic, how have you thought about these synergies from a preclinical standpoint in order to best design a future clinical study with the greatest probability of success?

[Carsten Brunn:]

Yeah. That’s a great question. And obviously, with now over 300 patients dosed with ImmTOR, we have very good safety database, which is always very important that, we’ve seen that yesterday, translates very nicely to gene therapy. And I think it’ll be extremely helpful as well for autoimmune problems as well. We will provide more guidance early next year, but I can tell you we’re extremely excited about our partnership with Cyrus, where we released some initial data a couple of weeks ago, where we saw very strong synergies between an IL-2 mutein and ImmTOR, where we saw a higher percentage of overall Tregs, when combining those two approaches. I will provide more data next year and I think that would be really transformational for the autoimmune fields, but really enhancing the tolerogenic effects of ImmTOR.

[Kristen Kluska:]

Thank you.

[Operator:]

Our next question will come from Raju Prasad with William Blair. Please go ahead.

[Raju Prasad:]

Thanks for taking the question. I was hoping you could provide a little bit of clarity on the assay used to measure neutralizing antibodies here and when we might anticipate seeing kind of total antibody concentration and IgG levels from the study -- from the SEL-399 study? Thanks.

[Carsten Brunn:]

Yeah. Thanks, Raj. I’ll let Kei, our Chief Science Officer address that question. Kei?

[Kei Kishimoto:]

Hi. Good morning. Yeah. The assay to measure neutralizing antibody is a cell-based assay. So it’s pretty standard within the field. Basically, you’re measuring that activity of serum samples to neutralize AAV’s from transducing hepatic sites cell line in vitro. With respect to the other data, we haven’t guided on that. But we are planning to release additional data from this study in conjunction with our partner AskBio at our upcoming gene therapy meeting.

[Raju Prasad:]

Great. And then the dose here was in the 10 to 12 range. Can you maybe just comment a little bit on, how you would think about doses in like a 10 to 13 or even one-time kind of the 14 range? Do you think that the 0.3 dose might be necessary in some larger capsid doses or do you think that data 0.15 with multiple doses could potentially work for any therapeutic range of AAV that’s being utilized by now in clinical trials? Thanks.

[Carsten Brunn:]

Yeah. So we have shown yesterday we have data in mice where we use doses of SEL-313 of the AAV vector. Our partner Sarepta has dosed even higher, unfortunately, we can disclose that data. But in the mouse study, we saw good results as well. We haven’t guided which dose we plan to take into the clinic. But I think yesterday’s data is a good indication we saw, which was very important to us a good dose response curve starting at 0.15 mg per kg and then very strong efficacy at 0.3. But also I want to remind you that we actually even tested a higher dose of ImmTOR in the Phase 1/2 study of SEL-212 as well. So there is still some dose elasticity if we really wanted to push dose. But I think, the 0.15 is a good starting point in our view. And definitely, the learning from the non-human primate study in yesterday’s Empty Capsid Study is that three monthly doses is likely the way to move into the clinic.

[Raju Prasad:]

Great. And then maybe just one last one, I think, we’ve got some questions on kind of the rebound in neutralizing antibodies post day 30. Can you maybe just address a little bit of that? How -- from preclinical studies how you know that three doses will be enough. Is it on like capsid half life or something like that, two, kind of enable re-dosing and kind of came down antibody levels over the long-term? Thanks.

[Carsten Brunn:]

Yeah. That’s an excellent question. And obviously, we saw yesterday, a very good comparability between the data from the non-human primary study and the first human study Empty Capsid Study. Specifically, when we look at days 30, the distribution, the data pattern was very, very similar. And we know from the non-human primate study that three monthly doses, maintain control out to day 90. We know from the mouse studies, we can maintain the control out to day 168, which is obviously very encouraging as well. So I think we have a good evidence that three monthly doses maintain control beyond day 90. We also have case done some work with outside collaborators that demonstrated that ImmTOR actually extends the half life of the capsid. But we believe past day 90, there’s no more capsid, no more antigen present actually.

[Operator:]

Our next question will come from Gil Blum with Needham & Company. Please go ahead.

[Gil Blum:]

Good morning and thanks for taking my questions. So it looks like you guys have quite a lot of strategic collaborations. Is there any particular guidelines as to who you partner with, particularly for your gene therapy side of programs?

[Carsten Brunn:]

Yeah. Gil, that’s a great question. Yeah. We’ve been busy. We’ve struck four partnerships that we’ve been highly selective. As you know, we started a partnership last year with Sarepta as we see them as a leader in neuromuscular disorders and we’re making good progress in that partnership. We just last quarter received a $3 million milestone payments. So we’re pleased with that. We’re also excited to be working with Takeda. We are working -- specifically, we’re exclusive licensing ImmTOR for two license lysosomal storage disorder. So these are liver based diseases. And Takeda is clearly a leader in the space, having a couple of ERT’s enzyme replacement therapies and really investing in their gene therapy business. So we’re very excited. They’re committed. They’re long-term player. That’s the reason we partner with them. So that’s, if you like, and as Selecta’s out license deal of ImmTOR. But we’ve also in licensed in the gene therapy space an IgG protease from Genovis, which we think is highly differentiated and we really want to use Xork to address patients with pre-existing immunity, which really expands the patients who will eligible to gene therapy. So you can see we’ve been fairly selective and we’re trying to strike a balance here between selectively licensed ImmTOR into indications that we don’t want to pursue ourselves, but also licensing assets in such the IgG protease from Genovis.

[Gil Blum:]

Maybe a related question, if I, how do you prioritize your own internal pipeline?

[Carsten Brunn:]

That’s a great question. Obviously, we’re in a fairly strong financial position and we want to have as many shots on goal. If you look at our pipeline, we have all the way from late phase -- late-stage Phase 3 to about to enter the clinic two more discovery stage. So, obviously, there’s different costs associated with that. And so, I think right now, we do have the means and the resources to move all those programs forward. But, obviously, some are more cost intensive and more resource intense once they move into the clinic. But our goal is to take advantage of the cash position and have as many shots on goal as possible.

[Gil Blum:]

All right. And maybe a last one on your IgG and less days like compound, you mentioned that, there’s an issue with re-dosing of these kinds of enzymes and I know that, I think I have listed it as a commercial product. How much of an issue with this?

[Carsten Brunn:]

Yeah. So it is a big issue. It’s a bacterial enzyme. So highly immunogenic. There really two use and we think it’s a great fit for our platform. Our primary focus is to use the IgG protease to pre-treat patients with pre-existing antibodies. So have a natural AAV infection to expand the patient pool. But there is a second application autoimmune disease. As you rightly said, there is a commercial product an IgG protease in Europe, that is limited to a single dose due to immunogenicity and I think we have the potential to re-dose Xork and you open up two more indications. And specifically, there are a number of IgG-mediated autoimmune diseases where we can apply Xork.

[Gil Blum:]

All right. That’s very helpful. Thank you for taking my questions.

[Carsten Brunn:]

Thank you, Gil.

[Operator:]

Our next question will come from Yun Zhong with BTIG. Please go ahead.

[Shu Zhiqiang:]

Hi. This is Shu on for Yun Zhong. Thank you for taking our questions. So I have a couple questions regarding your MMA program. So first is about the CMC review by the FDA, can give me a more colors on here, because FDA needs more time to review or you kind of foresee FDA may request more data? And also, when do you think you will give us an update on the clinical design on MMA. I know your Phase 1 study we will explore ImmTOR first dose in that phase. So do you think your Phase 1 study, you may like include the re-dosing strategies in those patients if needed?

[Carsten Brunn:]

Yeah. Thanks for the question, Shu. So we -- unfortunately, we can’t provide a lot more color. We don’t have more color. We -- as you can read in our filings, the FDA 30-day review period expired, we’re orally communicated by the FDA that they’re still considering certain aspects of the submission around CMC and we will get a formal decision by the end of November. So, unfortunately, we also don’t have more color. Around when do we have guidance on the clinical design? Obviously, once we have IND approval, we’ll share the clinical trial design in detail and also some of the endpoints we’re going to look at in the Phase 1 trial.

[Shu Zhiqiang:]

Okay. Thank you. Can I ask one more question here? So just some competitive landscape in MMA or just for very few or even rare indication, the space seems to be quite crowded, just name a few like the large bios AAV gene therapy and Madonna has mRNA program and a few other oral solutions in trials. So, I mean, it’s great for the patient community. But from a business perspective, I just want to know your thoughts on this, is this because the disease may be like significantly under diagnosed, there may be more patients than estimated or is just a good indication for proof-of-concept, and also do you participate -- anticipate some like patient enrollment challenge?

[Carsten Brunn:]

Yeah. That’s good question. And we obviously think that we do have a highly differentiated approach here with the potential to be dose, no other approach can offer that. So we think we’re quite differentiated from that perspective. It is still -- it’s not an ultra-rare indication, it’s a rare indication. So you’re looking in the 1000s of patients and we’ve been very strategic about who we work with. So we have a partnership with the NIH with Chuck Venditti, who was one of the preeminent researchers in the field and they maintain the largest patient registry in the U.S., so we’ll have access to those patients. So we’ve been mindful around partnering with someone who has access to MMA patients.

[Shu Zhiqiang:]

Okay. Thank you very much.

[Carsten Brunn:]

Thank you, Shu.

[Operator:]

Our next question will come from Ram Selvaraju with H.C. Wainwright. Please go ahead.

[Unidentified Analyst:]

Good morning from New York. This is Ahmed on for Ram. Thanks for taking our questions and thanks for the comprehensive update. So three from us, if I may. So enzyme replacement therapy in the context of lysosomal storage diseases is kind of like a foundational treatment. From your perspective, we were wondering, what are some of the drawbacks you see in this context? And how are you proposing to replace the existing treatment paradigms through ImmTOR?

[Carsten Brunn:]

Yeah. I’ll let Peter answer that in more detail. But, obviously, ERTs are limited in their use. They’re easily highly immunogenic enzymes and if you look at the overall response rates are not as high as, whereas obviously, gene therapy are addressing the underlying monogenic issue of pathology. But I will let Peter provide a more color here.

[Peter Traber:]

Yeah. Carsten, I think that you’ve hit the key issue, with enzyme replacement therapy, you have to give it repeatedly and they are immunogenic compounds. So you develop anti-drug antibodies, which then reduces the efficacy. Of course, adding ImmTOR to ERTs could potentially improve that. But with our partnerships, particularly with Takeda, we have taken a different approach and that is to team up with gene therapy, because gene therapy will likely reduce the long-term nature of the ERT and give you more long-term treatment. And furthermore, if we add ImmTOR to that, if it does wane in expression, we can potentially do repeat dosing of the gene therapy. Additionally, ImmTOR with the gene therapy could also prevent antibodies to the express transgene. So there are a couple of ways that that ImmTOR combined with gene therapy is the approach that we’re pursuing rather than ERT.

[Unidentified Analyst:]

Okay. Thanks for that color. Shifting on to the partnership with Ginkgo, are you able to expand on the logistics of bringing ImmTOR together with their foundry platform? For example, will ImmTOR capability be installed at the foundry or will the platforms operate independently in the current locations?

[Carsten Brunn:]

Yeah. So they’ll operate independently and we’re really excited about the partnership with Ginkgo. And we’re basically using their foundry to develop novel and next-generation enzymes, which we then plan to combine with ImmTOR. So what’s important is that, we will have exclusive therapeutic use for those enzymes. So in a sense, it’s a strategic partnership, but it’s really an inbound deal where we get access to some of their novel enzymes that we plan to combine with ImmTOR to enable re-dosing.

[Unidentified Analyst:]

Fantastic. And then, just finally, are you able to reveal specific design capabilities of Ginkgo’s foundry platform that you think will enhance the sustained efficacy of biologics with ImmTOR technology? And in addition, if you’re able to reveal some initial disease indications that you’re pursuing with Ginkgo, that would be great, too?

[Carsten Brunn:]

Yeah. So we haven’t disclosed details, which just gave kind of the broad brush. We’re pursuing autoimmune diseases enzy -- within enzymatic therapy. And maybe I’ll let Kei talk a little bit about their technical capabilities. But what really struck me is the quality and the speed they can identify enzymes and modify enzymes, really convinced me that they’re in quite a unique position in the landscape of protein design for engineering companies. But maybe Kei can provide a little more color.

[Kei Kishimoto:]

Yes. Yeah. Obviously, Ginkgo has made quite an impact in the protein engineering world. I think they’re foundry technology really industrializes protein design and testing. So I think, as Carsten mentioned, we’re really excited to be working with them and then got a future date, we’ll be able to provide you more information on our partnership.

[Unidentified Analyst:]

Okay. Excellent. That’s all from us. All the best.

[Carsten Brunn:]

Thank you.

[Operator:]

This concludes our question-and-answer portion of the call. I will now turn the call back over to Selecta’s CEO, Carsten Brunn for any closing remarks. Carsten?

[Carsten Brunn:]

Yeah. Thank you, Operator, and thank you, everyone, for joining us this morning. Stay safe and healthy, and this concludes today’s call. Thank you.

[Operator:]

The conference has now concluded. Thank you for attending today’s presentation. You may now disconnect.