Good afternoon and welcome to the REGENXBIO Third Quarter 2020 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. As a reminder, this conference call is being recorded. I would now like to turn the call over to Mr.

Patrick Christmas, Chief Legal Officer for REGENXBIO. You may begin..

Good afternoon and thank you for joining us today. With us are Ken Mills, REGENXBIO's President and Chief Executive Officer; Dr. Steve Pakola, our Chief Medical Officer; and Vit Vasista, our Chief Financial Officer. Earlier this afternoon, REGENXBIO released financial and operating results for the third quarter ended September 30, 2020.

The press release reporting our financial results is available on our website at www.regenxbio.com. Today's conference call will include forward-looking statements regarding our financial outlook in addition to regulatory and product development and other business plans.

These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted and can be identified by words such as expect, plan, will, may, anticipate, believe, should, intend and other words of similar meaning.

Any such forward-looking statements are not guarantees of future performance and involve certain risks and uncertainties.

These risks are described in the Risk Factors and the Management's Discussion and Analysis sections of REGENXBIO's annual report on Form 10-K for the full year ended December 31, 2019, and comparable risk factors sections of REGENXBIO's quarterly report on Form 10-Q, which are on file with the Securities and Exchange Commission and available on the SEC's website.

Any information we provide on this conference call is provided only as of the date of this call, November 4, 2020. And we undertake no obligation to update any forward-looking statements we may make on this call on account of new information, future events or otherwise. Please be advised that today's call is being recorded and webcast.

In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company. Actual results may differ materially. I would now like to turn the call over to Ken Mills.

Ken?.

Thank you, Patrick. Good afternoon, everyone, and thanks for joining us. I hope everyone is staying healthy. On today’s conference call we’ll provide a recap of our recent progress advancing and expanding our NAV technology platform as well as expected future milestones.

Steve will provide an update on our clinical programs and Vit will provide an update on financial results from the third quarter, 2020. We will then open up the call for questions.

I’m proud to say that during this quarter we’ve hit several milestones in development of our internal clinical pipeline as two important Phase II studies evaluating RGX-314 when delivered in the super parietal space are now underway for the treatment of wet AMD and diabetic retinopathy and we’ve expanded our MPS II program to include additional patients in the ongoing Phase 1/2 study of RGX-121.

In addition, our partners at Novartis have recently achieved significant milestone in the gene therapy space with over $1 billion in cumulative net sales of Zolgensma, a transformative one-time gene therapy for the treatment of pediatric patients with spinal muscular atrophy and the first approved gene therapy based on REGENXBIO’s NAV technology platform.

We’re grateful to have contributed to a therapy that’s impacted the lives of over 700 children with SMA and their families around the world.

As always, we remain focused on the health and safety of our employees, partners and patients during this challenging COVID-19 pandemic, we continue to actively monitor the impacts that pandemic will have on our plans for preclinical and clinical development timeline and we’re focused on our general operations for the remainder of 2020 and into 2021.

With that I’d like to turn the call over to Steve for more detailed clinical and regulatory update..

Thanks, Ken. When focused on development plans for the RGX-314 program with several studies underway in wet AMD and diabetic retinopathy and the start of the pivotal program to come.

We believe that RGX-314 has the potential to profoundly impact all aspects of clinical management for patients with wet AMD and DR and could be a one-time gene therapy treatment option for a broad range of patients.

Our plans for the pivotal study for the sub retinal delivery of RGX-314, treatment of wet AMD including dose selection in number of patients and other factors are based on the learnings from the first inhuman ongoing Phase ½ study, including results from patients in cohort 3, 4 and 5.

As previously shared, these patients have demonstrated stable to improve visual acuity and retinopathiness as well as the meaningful reduction in anti-VEGF injection. We also have data from Cohort 3 out for two years, which gives us confidence in the long-term durability of the gene therapy.

We now expect to initiate our pivotal program for the sub retinal delivery of RGX-314 for the treatment of wet AMD in the first quarter of 2021 and look forward to providing more details for this study in the coming months.

In September, we announced the dosing of the first patient in the Phase 2 AVA trial evaluating the super parietal delivery of RGX-314 using the SCS micro injector for the treatment of wet AMD.

The initiation in this trial was an important milestone for our company as well as the feel as the first clinical trial to evaluate the delivery of any gene therapy to the super parietal space.

We’re excited about the potential of the targeted in office super parietal approach which may provide additional RGX-314 delivery options for patients beyond sub retinal delivery. Importantly, patients in this trial would not receive propel active immune suppressive corticosteroid therapy before or after administration of RGX-314.

To-date RGX-314 has been well tolerated including no evidence of inflammation. We remain on track to complete enrollment of the first cohort by end of 2020 and report an initial safety data from the first cohort in early 2021.

Our Phase 2 trial altitude to evaluate super parietal delivery of RGX-314 in patients with diabetic retinopathy is active and we expect to begin enrolling patients by the end of 2020. DR is the leading cause of blindness in working age adults and affects approximately 8 million Americans.

As the disease progresses from non-proliferative disease at stages in which patients are often asymptomatic to more severe disease, patients are at increased risk of developing vision threatening complications.

Most patients with non-proliferative DR often go untreated as the current standard of care is watchful waiting until vision becomes threatened. There are treatment options for patients with DR including chronic repeated anti-VEGF injections, retino laser treatment and surgery.

However, receiving frequent anti-VEGF intraocular injections makes the treatment a less desirable option versus watchful waiting for many asymptomatic DR patients.

Unfortunately without treatment, a large proportion of these patients will eventually develop vision threatening complication including diabetic macular edema and neo-vascularization that can lead to blindness.

We believe that one-time treatment with RGX-314 has the potential to provide sustainable long-term anti-VEGF delivery reducing severity of DR and preventing vision threatening complication. The ALTITUDE trial is expected to enroll 40 patients with DR across two dose cohorts.

Patients will be randomized to receive RGX-314 versus observational control at a 3:1 ratio. The primary endpoint of the study is changed in diabetic retinopathy severity based on the ETDRF, DRFS scale at 48 weeks. A scale well-known to regulators and with actually used for approval of Eylea and Lucentis for the treatment of DR indication.

Other endpoints include safety and development of diabetic retinopathy related complications. We expect to begin dosing patients across 15 leading U.S. retina centers by the end of 2020 and plan to report interim data from this trial in 2021.

Turning to our rare disease portfolio, in September we announced expansion of RGX-121 program for the treatment of MPS II including an amendment to our ongoing Phase 1/2 study to allow for additional patients in an expanded cohort two.

We’re excited to announce that we’ve already dosed two more children in this cohort and we anticipate further updates from this trial by the end of 2020. In addition, we plan to initiate a second Phase 1/2 multicenter open label trial to evaluate RGX-121 in older pediatric patients with severe MPS II.

We’ve planned to enroll up to six patients and administer RGX-121 directly to the cerebrospinal fluid through intracisternal or intracist rebrow intraocular injection.

We also announced our plans to initiate a new observational trial designed to provide detailed characterization of neurocognitive development and key biomarkers in patients with severe MPS II.

In severe forms of MPS II early developmental milestones in a child may be met, but delays become apparent by 18 months to 24 months with neurologic deficits and cognitive impairments becoming apparent by the age of six years.

This trial is intended to perceptively document the changes in neuro-developmental parameters of cognitive behavioral and adoptive function overtime in addition to biomarker activity in the CSF serum.

We believe the study will provide critical data neuro-cognitive development of young pediatric patients with MPS II and we look forward to sharing the data from this observational study with the patient community.

We of course have a number of additional programs headed towards the clinic and I look forward to 2021 being a key year for development, advancement of these gene therapy candidates particularly within our rare disease pipeline.

Notably our programs for CLN2 disease, RGX-181 which is designed to address the CMS manifestations and RGX-381 which is designed to address the ophthalmic manifestations of the disease, our on-track for IND filing in the coming months. We look forward to initiating clinical trials for both these program. With that I’ll turn the call back over to Ken.

Ken?.

Thanks, Steve. We’re obviously very encouraged by the progress and the focus on the internal pipeline and the work by the entire team and congrats again Steve on the acquisition in the new poppy..

Thank you..

So, we’ve a couple of things to highlight in addition to the internal program worked this quarter and as we know REGENXBIO has an extensive footprint in the gene therapy space are now vectors in the basis of working not only internally, but also many partnered product candidates and including one marketed product Novartis’s Zolgensma as well as several others that are in late stage clinical development.

Our team recently collaborated with Kurma Partners, a leading European venture capital firm, to form Corlieve Therapeutics, a new company focused on severe neurological conditions.

And under the collaboration and license agreement with the newly formed Corlieve, we’ve received equity as well as are eligible to receive milestone payments and royalties in return for their use of NAV AAV9 vector for the treatment of refractory temporal lobe epilepsy.

In addition, Olivier Danos, our Chief Science Officer has joined Corlieve’s Board of Directors and will work closely with the team on the development of POE program, so are encouraged by this new development in our pipeline.

And also one is to highlight that, as I mentioned earlier, great progress this quarter and throughout the year so far on Zolgensma and as announced last week in our press release REGENXBIO has received a milestone payment of $80 million from Novartis based on the achievement of over one billion cumulative net sales of Zolgensma.

We’re strongly encouraged by the therapy, strong global momentum and with potential to help many more patients and families. So with that I’ll turn the call over to Vit and he can review the financials..

Thank you, Ken. REGENXBIO ended the quarter on September 30, 2020, with cash, cash equivalents and marketable securities totaling $289.8 million compared to $400 million as of December 31, 2019.

The decrease was primarily attributable to net cash used in operating activities of $93.5 million and cash used to purchase property and equipment of $14 million.

Cash reported as of September 30, 2020, excluded some recently announced $80 million milestone payment received from Novartis in October 2020, which was reported as accounts receivable at the end of the third quarter. Revenues were $98.9 million for the quarter ended September 30, 2020 compared to $14.7 million for the third quarter of 2019.

The increase was primarily attributable to $9.6 million increase in Zolgensma royalty revenue over Q3, 2019 and the one-time $80 million milestone fee recognized as revenue in the third quarter of 2020 and achievement of $1 billion in cumulative net sales of Zolgensma since launch.

Sales of Zolgensma for the third quarter of 2020 increased by 82% as compared to Q3, 2019. Research and development expenses were $44 million for the quarter ended September 30, 2020 compared to $35.7 million for the third quarter of 2019.

The increase was primarily attributable to personal related costs as a result of increased headcount, expenses associated with conducting clinical trials for our lead product candidates and externally sourced services for preclinical, regulatory and manufacturing-related activities.

G&A expenses were $15.9 million for the quarter ended September 30, 2020, compared to $12.4 million for the third quarter of 2019. The increase was primarily attributable to personal related costs as a result of increased head count and professional fees for advisory and other services.

Net income was $8.8 million or $0.24 basic and $0.23 diluted net loss per share for the three months ended September 30, 2020 compared to a net loss of $34.6 million or $0.94 basic and diluted net basic and deluded net loss per share for the three months ended September 30th 2019.

As of September 30th 2020, we had approximately 37.4 million common shares outstanding.

Based on its current operating plan, REGENXBIO expects its balance in cash, cash equivalents and multiple securities as of September 30th 2020, in addition to the $80 million received from Novartis in October 2020 to fund its operations including the completion of its internal manufacturing capabilities and clinical advancement of its private candidates until mid-2022.

With that, I will turn the call back to Ken to provide final thoughts on yesterday's results..

Thanks, Vit. Our really good summary of the financial condition of the company and on top of Steve's updates about the program work as well as the encouraging developments with our partners. Again it continues to be a good time for REGENX to be advancing value for patients and stakeholders.

And I also wanted to highlight that the construction of our new GMP facility here in Rockville continues. Facility that allows us to manufacture it 2000 leader scale using a fully optimized HEK293 suspension cell culture system. This is the platform that is widely utilized and is also well-understood by the regulatory agencies.

So, we continue to make substantial improvements in our efforts to ensure consistency of products across large volumes and across multiple programs.

And in all, this has been our multiyear effort led by a great team of people that have both commercial level expertise in biologics and well as gene therapy and a global supply chain experience that emphasizes high quality, well-characterized scalability to match the clinical and emerging commercial demands of REGENXBIO.

So, we're excited about that progress and look forward to the site coming online. And in general, we look forward to making more strides towards our mission to improve lives for the curative potential single administration gene therapies and we remain dedicated to this mission.

I want to definitely take time to express the sincere appreciation that myself and leadership team have to our talented and dedicated employees were driving business forward despite the challenging backdrop with the COVID-19 epidemic. With that, operator we will turn the call over to questions..

Thank you. [Operator Instructions] Our first question comes from the line of Gena Wang with Barclays. Your line is open, please go ahead..

Thank you for taking my questions. So, I have a two questions and though first steps of question is regarding these suprachoroidal microinjector program, AAVIATE trial in that and do. So, you mentioned that you already on dosed the patient. Just wondering how many patient have already been dosed.

And not is that right or the patient you've got so far, you haven’t seen any inflammation in this patient. And then related question was regarding the safely data in the first quarter next year. Beyond per safety, I assume will be three patient from Clinic 01.

Will you also show down market data and that initial efficacy data including rescue injection and visual maturity and the OTC? And the second question is regarding the MPS II, just wondering if you can give a little bit more color regarding the year-end '20 update.

What type of data and in terms of also patient numbers and type of data that we will see..

Hey Gena, thanks for the question or questions. Steve, maybe I'll ask you to provide an update on AAV8 and just what we've seen so far and what we were thinking about for early 2021. .

How Gena, thanks for the questions. Yes, so AAV8 as we disclosed today and as you've mentioned. It is true that we've not seen any evidence of inflammation so far. It is still early in the trial in terms of number of patients treated. You also have that question. We only did just announce the dosing of the first patient in early September.

So, not that long ago and as is typical for in initial study with albeit not a first and even say for RGX-314. This study is a first in human for delivery to the suprachoroidal space. That was actually at sentinel subject where we initially dosed and follow for a couple of weeks.

In general to follow for overall safety and then proceed with dosing of the rest of the cohort. And view of course we're able to do that as if we've announced that we've seen no inflammation in any of the patients that we dosed to-date.

We have a total of 20 patients that we want to dose in cohort 1, 15 of which will receive RGX-314 where we've test multiple patients. But we're still early in the game as far as activating all the sites and really ramping up recruitment. And that's one of the key reasons now we're not in a position to give for example a more fulsome update.

And why as you've mentioned will be giving are two initial safety update early next year. But certainly we're encouraged by the fact that in spite of having no immune suppression at all in terms of for example no topical steroid before or after RGX-314. To-date, we're not seeing inflammation and that's what we were able to achieve pre-clinically.

But this being not subretinal space. This being the first study ever looking at gene therapy and suprachoroidal space. This is really a significant milestone in terms of assessing safety and tolerability. And importantly whether we can achieve with AAV8 delivery specifically with RGX-314.

The ability to deliver without immune suppression, therapy and without inflammation. So, the second part of your question in terms of what we can expect to provide in that initial safety update early in 2021.

I guess fairly to manage expectations, we'll be a few more months on and we'll have a full cohort of patients that we're guiding since we still plan to complete recruitment of the 20 patients in that first cohort. And in that first update, as part of the overall assessment, certainly we'd continue to look at safety and tolerability first as always.

But also be able to provide some of the typical biomarker pharmacodynamic activity measures that we've traditionally provided for our subretinal program. Though again we caution.

It's still pretty early in terms of number of months the patients have been treated and I think we've been consistent all along for several years now in how we talked about pharmacodynamic measures and the importance of looking at durability of treatment effect where you really want to be able to look at. For example, six months of data.

So, before you really have a sense there. So, nevertheless we're still in that initial safety update would be including some of those typical assessments. Even just from a safety perspective..

Yes. And just it's a quick, thanks to you for the complete answer there. And on MPS II, Gena just quickly. We do have plans for an update before the end of this year and very pleased to have announced today, two additional patients enrolled at the second half E10 dose level under this expanded protocol.

Well, what we have is plans for updating the data set of patient that have then previously enrolled in pulling new timelines forward a bit more. So, we'll be looking to do that in the next couple of months.

Next question?.

Thank you. And our next question comes from the line of Geoff Meacham with Bank of America. Your line is open, please go ahead..

Hey guys, thanks for taking our questions. This is Alec on for Geoff. So, my first question is on 3/14.

Do you have a sense from your physician interaction what an acceptable frequency of rescue VEGF injection could be with three four teams for clinical adoption, particularly taken in to account, the three month dosing for Beovu and six months for Lucentis PDS? And I know it may be a bit premature considering 314 just started pivotal study or will early next year but would you also be able to provide your thoughts on reimbursement discussions and how those are evolving for gene therapies and whether payers would also be looking at the rate of rescue injections to make reimbursement decisions? Thanks..

Sure. Steve, I'll let you swing back to maybe some of the physician interaction in terms of intervals of treatment. Alec, thanks for the question. I mean, we have we're moving into pivotal phase of development and we have started to engage.

The entire system in discussion is about the value and need around changes in standard of care for wet AMD, and a lot of receptivity to not just extending but eliminating the intervals that exist around treatment in terms of compliance and convenience.

So, I think we continue to view that gene therapy has the unique profile that adds a tremendous amount of value and really emphasize at this moment in time where this overhang of pandemic was really affecting and influencing.

I think we're hearing from people both on the care side as well as on the provider and reimbursement side, what is happening to people right now if they're not able to come in and get their regular treatments and how much more effect is that going to have on outcomes and ultimately on cost and productivity and other things.

So, a lot of people paying attention right now. And through that engagements, I think the profile that we've had for a while has been to get at least half of the injections worldwide eliminated. That we've talked about this as our target product profile, taking a look at what the market is today for VEGF injection rates.

It hasn't moved all that much with respect to changes in labeling. I think when you look at the clinical data, when you look at the sort of clinicity of how things like Lucentis and Eylea and Beovu, and maybe what's to come or coming forward, hasn't moved a needle all that much.

We need to be doing more and that's where we're focused on that unmet need.

In terms of where that value lands ultimately, I think it's for a one-time treatment or one that has seen dramatic reduction 50% or more in the use of existing therapy or therapies that are emerging that are incremental, we view a huge opportunity there and have been very pleased with the conversations and the responsiveness that we've been able to engage so far at the stakeholder level there.

Steve, maybe you can talk to clinically what the engagement's been like..

Yes. It's been very interesting and you raised a good point, Ken, on the COVID impact and how that's really shined a light on the importance of durability and ultimately the possibility of a one-time treatment.

And we're hearing that anecdotally not only for the patients in our ongoing study, who fortunately have been able to have the need for reinjection eliminated where we've seen real-time patients in surge areas who've been able to admit a visitor too, which we don't like from a clinical trial standpoint.

But from an overall benefit risk, the ability to have the comfort that you have the foundational anti-VEGF treatment that you need and you're not risking potentially vision threatening complications by extending your visits. And it really comes back to how these physicians and their patients and their caregivers evaluate the benefit risk.

And you raised Beovu and PDS. And as you know, retina is a dynamic space and it's been a dynamic space.

So, we've known about these programs for a while and that target product profile that Ken reviewed of at least 50% reduction, either looking in terms of treatment burden or even looking in terms of patients who can completely have need for reinjections done away with.

That hasn't changed in our minds because it hasn't changed in the minds of the investigators and our retina specialists, advisors and their view of what's needed for their patients.

And it's interesting we don't really think of any of these welcomed advances in terms of these approved and other experimental agents that are able to advance to durability intervals of three to four months or five months or even six months as competition.

If anything, these are clear validation of how much need there is to get more durable, sustainable treatments. And the ultimate way to do that would be a one-time injection. So, we continue to see this as further validation for a one-time treatment and continue to see support for the target product profile that Ken elaborated on..

Yes. And remember, we feel that with the work we've already done in the assessment of subretinal at multiple dose levels, we have outperformed that target product profile and in a very challenged and severe population of patients.

So, we're feeling very good about the progress and the data set there as we're funneling it into pivotal phase of development, and also looking to differentiate the profile with suprachoroidal. So, thanks, Alec, for the question..

Thank you. And our next question comes from the line of Gbola Amusa with Chardan. Your line is open. Please go ahead..

Hi, thanks. Thanks for taking my call. I have two questions. The first is on manufacturing, given that you have internal coming online, which people recognize have certain advantages. But we've also heard that it's not just having a facility or footprint, it's the processes analytics.

So, could you talk about what you're doing to sort of have a best practice on those dimensions or anything you may be doing to avoid some of the growing pains that we've seen in the AAV based gene therapy space with the rights to manufacturing?.

Yes Gbola, thanks a lot. I'm glad you went there because it's a, well, I think it previously has been a subtle but maybe less and less subtle point for investors on these areas. And I would say that, even with our existing supply chain and the strong collaborations we have with bulk manufacturers and final fill finish with groups like Fuji and others.

We have installed capabilities here in Rockville already in labs for analytics, quality assurance, quality control, assessment, release criteria that relate to not only the AAV side of the equation but let's not forget about the device and route of administration side here.

We're working with subretinal procedures, we're working with suprachoroidal procedures, and we're working with intracisternal procedures and intravenous procedure. So, yes we have compatibility with all of the devices that are being sent to these sites clinically and commercially as well.

And that's been a major focus of quality of sort of strong analytical capabilities, assessing things across a wide range of different types of AAV vectors when it comes to things like potency as well has been really important to have that expertise internally and that's already here.

So, in some ways what we're actually bringing further into Rockville, in our view is more of the commodity scale things that we need to take the next steps in terms of insulating ourselves with respect to especially that commercial supply chain risk.

But we think that we have a really strong foundation in a lot of those other areas that as you pointed out. I think, have been stumbling blocks and certainly can be especially in a new and emerging area of science and manufacturing science.

Next question?.

Our next question comes from the line of Matthew Harrison with Morgan Stanley. Your line is open. Please go ahead..

Hello, everyone. This is Kostas on for Matthew. I have a quick question on your CLN2 programs. Can you please talk about the status of the programs and what needs to be done before you file for the IND in 2021? Thank you..

Sure, Kostas. Just at a high level, let's see if Steve has anything else to add. We're guiding here to milestones that are very soon with respect to those two programs. So, in the case of both, we are really having completed the preclinical study evaluation phase and readout phase.

And keeping in mind that certain things like the 181 program in particular is borrowing also from our existing IND and ongoing study packages from things like 121 for instance. We're really translating that right now into written word and the modules of the IND filing.

And similarly for RGX-381, we're using route of administration that we're familiar with. We've already highlighted that we've reported data from non-human primates demonstrating sustained levels of things like the enzyme TPP1 in vitreous, following a subretinal injection.

So, similarly, we're in a phase right now here at the beginning of November to be filing an IND by the end of the year. We're literally in the final phases of preparing the reports and writing the documents to be submitted. So, hope that helps..

Okay, thank you..

Our next question comes from the line of Mani Foroohar with SVB Leerink. Your line is open, please go ahead..

Hey, good afternoon everyone. This is Rick dialing in for Mani, and thanks for taking our questions. So, now that the pivotal 314 trial is set to start in the first quarter of 2021, we are hoping to get some color on the trial design.

Have you received any feedback from the FDA regarding the pivotal endpoints, what the control arm would look like, duration of endpoints or any other aspect of trial design?.

Hey Rick, thanks. No, not that we're updating today and we will plan to update that at the beginning of next year when we talk about the initiation of the study.

So, we continue to guide to the fact that we think moving into pivotal phase of development in retina has some familiarity with the agency for different types of products, different types of modalities, things that have already bred and brought up on this call, of course, like recently Beovu and of course PDS and so forth.

So, we're not expecting people to be surprised here with some massive departure in certain ways with respect to how things are considered at FDA when it comes to wet AMD and retina. However, there is obviously a gene therapy event to it and in RGX-314 event as well.

And we definitely will come forward clearly with more details on that study design early next year.

And Steve, anything else to add at this stage?.

No, you covered it nicely. We're excited to move into this stage, and we're about there..

Thank you. [Operator Instructions] And our next question comes from the line of Lisa Walter with RBC Capital Markets. Your line is open, please go ahead..

Hi, there. This is Lisa Walter on the line for Luca Issi at RBC. Thanks for taking my question. We just wanted to ask if we could have an update on the IP dispute going on between you and Passage Bio as well as Sarepta. Thanks..

Hi Lisa, thanks a lot. We don't have any specific updates to provide today. We have provided information previously on or I guess provided feedback with respect to our interest in making sure that people that are working with intellectual property and science that overlaps with our NAV Technology Platform are under license.

So that they're free to do so, but also fairly representing what they're doing with respect to their science and that we're looking out for the interest of when we think not only our shareholders and stakeholders but the interest of IP in general in our biotechnology space overall.

And that also, it tends to be groups that we view that are getting closer to commercialization sort of come up higher if you will on our radar screen with respect to sort of action and interest that we have overall. So, nothing more specific to update on today other than -- that's our philosophy.

We think it's an important one to be not only reflecting that we are and view IP as important as an industry and as a field in gene therapy but also specifically for our shareholders. And hope to have more updates on those actions coming into 2021 and throughout the year. Thanks for the question..

Thank you. And we have a follow-up question from the line of Gbola Amusa with Chardan. Your line is open, please go ahead..

Hi, thanks again. And just one --. First of all, congrats on the $80 million payment on Zolgensma. I get asked a lot about how long I should model the royalties from Novartis. So, I've noticed that you do have patent term extension and supplementary protection certificates as a process ongoing in multiple jurisdictions given the additional approvals.

Would you say we analysts should think of those as relevant to the duration of the royalty payments or are they less relevant? What can you say, what's the 101 on them?.

Hey Gbola, thanks. 101, I like that; AVXS-101, that was clever. The work that we're doing around patent term extension, I think, I mean, all of you as in the on the sell side, I think I understand much about the processes on the biologics side in particular. We have approvals now that are occurring, it started in the U.S.

and now it's carried over into other jurisdictions in Europe and Japan.

And so, the process of filing for patent term extension for keeping in mind that our as we've always discussed but just to reemphasize, the NAV Technology Platform and the patents that underpin the compositions and the methods of use and so forth that fall under our licenses are worldwide, right. And they have issued claims in all the jurisdictions.

So, we follow through with the pursuit of patent term extension at sort of normal course in each one of those jurisdictions on an individual or case-by-case basis as they're approved.

And similarly, our license in this case with AveXis, I guess, Novartis gene therapies now, as we amended and expanded it, includes license to a lot of patents in our portfolio relating to Zolgensma and AVXS-101. The norm is to file and get feedback and manage the process for potential to extend in major jurisdictions up to five years.

We've guided that. With respect to how quickly Zolgensma got approved in the U.S., the likelihood of extending or the possibility of extension is actually curved a bit and it's going to be closer to I think somewhere between three years and four years.

But we think it is entirely appropriate to be following that process, expecting that it occurs similar in nature to how biotechnology and biological products, existing products have had patents extended to protect them in that three years in the U.S.

or more and up to five years in jurisdictions like Europe and Japan are appropriate to consider based on how the license is structured, the type of IP that's being provided to protect Zolgensma and the value in it for us and Novartis gene therapies..

Thank you. And I'm showing no further questions at this time. And I would like to turn the conference back over to Mr. Ken Mills for any further remarks..

Okay, thank you operator. Thanks everyone, for the questions. Most importantly, I hope everyone is safe, healthy and well. It's been an interesting year and we definitely will be in touch with everyone that as we're now in the end of the year with more updates and milestones as we've guided to, as well as some conferences that are coming up.

And really excited to transition from 2020, maybe with the new outlook in general but positive nonetheless. We've accomplished a lot in 2020 coming into 2021; we look to continue to build on that. And again, I appreciate all the questions, appreciate the listening. Hope everyone's well, and have a great evening..

Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program and you may all disconnect. Everyone have a great day..