Q3 2020 Regenxbio Inc Earnings Call
[music].
Good afternoon, and welcome to the Genex Bio 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.
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 Virgin Ex <unk> you may begin.
Good afternoon, and thank you for joining us today with US today are Ken Mills rejects Bio's, President and Chief Executive Officer, Doug.
Dr., Steve Nicola, our Chief Medical Officer, and fit the system, our Chief Financial Officer.
Earlier. This afternoon are dead expire released financial and operating results for the third quarter ended September Thirtyth 2020.
The press release reporting our financial results is available on our website at www Dot rejects bio dotcom.
Today's conference call will include forward looking statements regarding our financial outlook. In addition to regulatory and product development plans. These.
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.
These risks are described in the risk factors and the managements discussion and analysis sections of rejects Bios annual report on form 10-K for the full year ended December 31, 2019, and comparable risk factors sections originates bio's quarterly reports on form 10-Q, which are on file with the securities and Exchange Commission.
And available in the Fccs website.
Any information we provide on this conference call is provided only as of the date of this call November 4th 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 todays 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 and.
Actual results may differ materially I.
I would now like to turn the call over to Ken Mills.
Okay.
Thank you Patrick good afternoon, everyone. Thanks for joining us I hope everyone is staying healthy.
On today's conference call, we will 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 program and it will provide an update on financial results from the third quarter 2020.
Well then open up the call for questions.
I'm proud to say that during this quarter Weve hit several milestones in the development of our internal clinical pipeline as two important phase two studies evaluating RG X three one floor when delivered in the Super Choroidal space are now underway for the treatment of wet AMD and diabetic retinopathy and we've expanded our MPS II program to include a.
Additional patients in the ongoing phase one two study of RG X one to one.
In addition, our partners that Novartis have recently achieved significant milestone in the gene therapy space with over $1 billion in cumulative net sales of Xeljanz a.
Transformative one frame gene therapy for the treatment of pediatric patients with spinal muscular atrophy and the first approved gene therapy based on region expires NAV technology platform.
Were grateful to have contributed to a therapy that impacted the lives of over 700 children with SMH and their families around the world.
As always we remain focused on the health and safety of our employees partners and patience. During this challenging Cove is 19 pandemic.
We continue to actively monitor the impact that pandemic will have on our plans for preclinical and clinical development timelines 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 a more detailed clinical and regulatory update.
Thanks, Ken.
You've been focused on development plans for the Archie Ecpthree 14 program with several studies underway in wet AMD and diabetic retinopathy and the start of the pivotal program to come.
We believe that Rgs 314 has the potential to profoundly impact all aspects of clinical management for patients with wet AMD AMDR and could be a onetime gene therapy treatment option for a broad range of patients.
Our plan for the pivotal study for the sub retinal delivery of our Gen 314 for the treatment of way to MD, including dose selection number of patients and other factors are based on the learnings from the first in human ongoing phase one two study including results from patients in cohort three four and five.
As previously shared these patients have demonstrated stable to improved visual acuity and retinal thickness as well as the meaningful reduction and anti VEGF injection burden.
We also have data from cohort three out to two years, which gives us confidence in the long term durability of the gene therapy.
We now expect to initiate our federal program for the sub retinal delivery of our tier 314 for the treatment of wet AMD. The 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 two aviate trial evaluating the suprachoroidal delivery of our check 314.
Using the FCS micro injector for the treatment of way to MD.
The initiation of this trial was an important milestone for our company as well as the field as the first clinical trial to evaluate the delivery of any gene therapy through the supercritical space.
We are excited about the potential of the targeted in office supercritical approach, which may provide additional large ecpthree 14 delivery options for patients beyond sub retinal delivery.
Importantly patients in this trial will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RG 314.
To date, RG Ecpthree 14, it's been well tolerated, including no evidence of inflammation.
We remain on track to complete enrollment of the first cohort by the end of 2020 and reported initial safety data from the first cohort in early 2021.
Our phase two trial altitude to evaluate two per crude oil delivery of our gen 314 in patients with diabetic retinopathy is active and we expect to begin enrolling patients by the end of 2020.
VR is the leading cause of blindness in working age adults and affects approximately 8 million Americans.
As the disease progresses from non proliferative.
Disease.
A stage 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 D are often go on treated as the current standard of care is watchful waiting until vision become threaten.
There are treatment option for patients with DDR, including chronic repeated anti VEGF injections retina laser treatment and surgery.
However, risks receiving frequent anti VEGF intraocular injections.
Make the treatment of less desirable option versus watchful waiting for many a symptomatic VR patients.
Unfortunately without treatment a large proportion of these patients will eventually develop vision threatening complications, including diabetic macular edema, and neovascularization that can lead to blindness.
We believe that onetime treatment with RG Ecpthree 14 has the potential to provide sustainable long term anti VEGF delivery, reducing severity FDR and preventing vision threatening complication.
The altitude trial is expected to enroll 40 patients with DDR across two dose cohorts.
Patients will be randomized to receive our gen 314 versus observation on control at a three to one ratio.
The primary endpoint of the study is change in diabetic retinopathy severity based on the Sdrs Drs at scale at 48 weeks a.
A scale well known to regulators and with actually use for approval by layer and consensus for the treatment of DDR indication.
Other endpoints include safety and development of diabetic retinopathy related complications.
We expect to begin dosing patients across 15, leading us radnet 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 the expansion of the Rdx 121 program for the treatment of NPS to including an amendment to our ongoing phase one two study to allow for additional patients in an expanded cohort two.
We are excited to announce that we have already dose 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 one two multi center open label trial to evaluate RG Ectwo hundred 21 in older pediatric patients with severe MPS too.
We have planned to enroll up to six patients and administer our GXT 121 directly to the cerebral spinal fluid through interest external or interest to reproach ventricular injection.
We also announced our plans to initiate a new observational trial designed to provide detailed characterization of neuro cognitive development and key biomarkers in patients with severe NPS too.
In severe forms of MPS too early developmental milestones and a child maybe Matt.
But delays become apparent by 18 to 24 months with neurologic deficits and cognitive impairment.
Becoming apparent by the age of six years.
This trial is intended to prospectively document the changes in neuro developmental parameters of cognitive behavioral and adaptive function over time in.
In addition to biomarker activity in the CSF serum and urine.
We believe this study will provide critical data about that neuro cognitive development of young pediatric patients with MPS too and we look forward to sharing the data from this observation will study with the patient community.
We of course have a number of additional programs headed toward 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 CN to disease, our GXT 181, which is designed to address the CMS manifestations and Rgs Threeeighty, one which is designed to address the ophthalmic manifestations of the disease.
Our on track for iron in the filing in the coming months.
We look forward to initiating clinical trials for both these programs.
With that I'll turn the call back over to Ken Ken.
Thanks, Steve were.
Obviously very encouraged by the progress and the focus on the internal pipeline in the work by the entire team in and congrats again, Steve on the acquisition and the new puppy.
So.
So we have.
A couple of things to highlight in addition to the internal program work this quarter and.
As we know Oragenics has an extensive footprint in the gene therapy space, our NAV vectors the basis of work not only internally, but also many partnered product candidates, including one marketed product Novartis is agenda as well as several others that are in late stage clinical development.
Our team recently collaborated with Cardinal partners, a leading European venture capital firm to form core lead therapeutics, a new company focused on severe neurological conditions in under the collaboration and license agreement with the newly formed core leave we have received equity as well as are eligible to receive milestone payments and royalties.
In return for their use of the now the benign vector for the treatment of refractory temporal lobe epilepsy.
In addition, Olivier Dan who is our Chief Science Officer has joined core leads board of directors and will work closely with the team on the development of the TLD program. So we're encouraged by this new development in our pipeline.
And also wanted to highlight that as I mentioned earlier, great progress this quarter and throughout the year. So far on Xeljanz as announced last week in our press release or Dennis Bio has received a milestone payment of 80 million from Novartis based on the achievement of over $1 billion in cumulative net sales of Xeljanz we're strongly.
Courage by the therapy strong global momentum and its potential to help many more patients and families.
So with that ill turn the call over to Vic and he can review the financials.
Thank you Ken Regenerons bio ended the quarter on September Thirtyth, 2020, with cash cash equivalents and marketable securities totaling $289.8 million compared to $400 million as of December 31st 29.
Team. The decrease was primarily attributable to net cash used in operating activities of $93.5 million in cash used to purchase property and equipment of $14 million cash reported as of September Thirtyth 2020 excludes the recently announced.
Almost $80 million milestone payment risk.
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 Thirtyth 2020, compared to $14.7 million for the third quarter of 2019. The increase was primarily attributable to $9.6 million, increasing so gentle royalty revenue.
Over Q3, 2019, and the one time Midi million dollar milestone fee recognized as revenue in the third quarter of 2020 on the achievement of $1 billion in cumulative net sales of Xeljanz, while since launch sales of Xeljanz will 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 Thirtyth 2020, compared to $35.7 million for the third quarter of 2019. The increase was primarily attributable to a 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 pre clinical regulatory and manufacturing related activities.
DNA expenses were $15.9 million for the quarter ended September 32020, compared to $12.4 million for the third quarter of 2019. The increase was primarily attributable to a personal related cost as a result of increased headcount and professional fees for advice.
Three and other services net.
Net income was $8.8 million or 24 cents basic and 23 cents diluted net loss per share for the three months ended September Thirtyth 2020.
Appeared to a net loss of $34.6 million or 94 cents basic and diluted net loss per share for the three months ended September Thirtyth 2019.
As of September Thirtyth 2020, we had approximately 37.4 million common shares outstanding.
Based on its current operating plan Regeneron Spo expects its balance in cash cash equivalents and marketable securities as of September 32020. In addition to the $80 million received from Novartis in October 2020 to fund its operations, including the completion of its internal manufacture.
During capabilities and clinical advancement of its product candidates until mid 2022.
With that I will turn the call back to Ken to provide final thoughts on yesterday's results.
Okay.
Thanks.
A really good summary of the financial condition of the company and on top of Steve's updates about the program work as well as being.
The encouraging developments with our partners again it continues to be a good time for oragenics 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 liter scale using a fully optimized H.T.K. to nine three suspension cell culture system. This.
This is a 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 a multi year effort led by a great team of people that have both commercial level expertise in biologics as well as gene therapy in a global supply chain experience.
That emphasizes high quality, well characterized scalability to match the clinical and emerging commercial demands of Virgin expire. So we're excited about that progress and look forward to the site coming on line.
And in general, we look forward to making more strides towards our mission to improve lives to 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 the leadership team have to our talented and dedicated employees driving business forward. Despite the challenging backdrop, the COVID-19 epidemic.
With that operator, we will turn the call over to questions.
Thank you ladies and gentlemen, if you have a question at this time. Please press Star then one on your telephone. If your question has been answered your question, let yourself from the queue. Please press the pound key to prevent any background noise. We ask that you. Please place your line on mute. Once your question has been stated.
Our first question comes from the line of Keeneland with Barclays. Your line is open. Please go ahead.
Thank you for taking my questions.
So I'll hold that's two questions first sets of questions of the garden warm.
Are you happy typically, though Michael Jackson co loan.
Overall trial, one so you mentioned that you already.
Those completion, just wondering how many locations Harper writing those off.
Matt worldwide for all patients dosed, so far you haven't owned.
Information.
Paulson.
Really the question is regarding the whole football in the first quarter looks yet.
Yong Betsy.
He will be sneaking from Q1.
We you also show ball market before on the initial efficacy before.
Total investment injection or visual acuity on that list.
And then second question is regarding the home sales to just wondering if you can give a little more color regarding the war on.
24.
Hi, good data on that in.
Tim thoughtful.
Patient numbers on top of four debt levels.
Hey, gene and thanks for the question or questions and.
Steve maybe I'll ask you to provide an update on aviate and just what we've seen so far what we're thinking about for early 2021 sure.
Sure Hi.
Hi, Jane and thanks, Thanks for the questions.
Yes, so aviate.
As as we disclosed today and as you 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 a number of patients treated.
Also at that 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.
And initial study with.
Albeit not a first in human say for RG <unk> three one for this study is the first in human for delivery to the Super Corrado space that was actually a sentinel subject, where we initially dose than follow for a couple of weeks in.
In general to follow for overall safety.
And then proceed with.
Dosing of the rest of the cohort and we of course were able to do that as we've announced that we've seen no no.
No inflammation.
In any of the patients that Weve goes to date.
We have a total of 20 patients that we want to dose in cohort one.
15 of which will receive our gen three one for where.
We.
So multiple patients but.
We're still early in the game as far as activating all the sites and really ramping up recruitment and Thats. One of the key reason that were not in a position to give for example, a more fulsome update.
And why as you mentioned, we'll we'll be giving our.
True 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 steroids before after our Gen 314.
To date, we are not seeing inflammation and.
Thats, what we were.
We are able to to achieve preclinically, but this being not sub retinal space.
Space this.
Being the first study ever looking at gene therapy in supercritical space.
This is really.
A significant milestone in terms of.
Assessing safety and Tolerability and importantly, whether we can achieve with Avi eight delivery, specifically with our GXT three one for 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 will be a few more months.
On and we'll have a full cohort of patients.
Not that we're guiding since we still plan to complete recruitment of the 20 patients in that first.
Cohort.
And in that first to update.
As part of the overall assessment certainly we continue to look look at safety and Tolerability.
First as always but also be able to provide some of the the.
Typical bio marker or pharmacodynamic activity measures that we've traditionally provided.
For our sub retinal program.
No again, we caution it's still still pretty early in terms of number of months that patients have been treated.
And.
I think we've been consistent all along for several years now.
In how we've 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 still in that initial safety update would be including some of those typical assessments, even just from a safety perspective.
Yeah, and just some quick that thanks, Steve for the complete answer there and on MPS to Gino just quickly you know we.
Do you have plans for an update before the end of this year and very pleased to have announced today two additional patients enrolled at the six and a half he 10 dose level under this expanded protocol. What we have is plans for updating the data set of patients that have been previously enrolled in pulling those.
Timelines forward a bit more so I will 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 Eric on for Jeff.
So my first question is on the 14.
Do you have a sense from your physician interactions what an acceptable frequency of breast you bet, Jeff injections would be.
With 314 for clinical adoption, particularly taking into account the three month dosing for for Bayview and six months six months for two senses Pds I know it may be a bit premature considering to 14 just started to close 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 therapy.
And whether payers would also be looking at the rate of rescue injections to make reimbursement decision.
Yeah.
Sure Steve I'll, let you swing back to maybe some of the physician interaction in terms of intervals of treatment.
Al I think thanks for the question I mean, we have we're moving into a pivotal phase of development and we have started to engage.
The entire system in discussions 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 combined compliance and convenient sites I think we continue to view that gene therapy has a unique profile.
That.
As a tremendous amount of value and really emphasized at this moment in time, where this overhang of.
Pandemic it was really affecting in influencing and I think we were hearing from people both in the care side as well as on.
The the provider in reimbursement side.
Is happening to people right now if they are 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 three that engagement I think the profile that we've had for a while has been to get.
At least half of the injections worldwide eliminate that we've talked about this is our target product profile, taking a look at what the market is today for that Jeff injection rates.
Rates and 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 Chronicity of how things like Lucentis in EIMEA and view and maybe what's to come are coming forward hasn't moved the needle all that much we need to be doing more and that's where we're focused on that unmet need in terms.
Where 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 were therapies that are emerging that are incremental and we view a huge opportunity there and have been very pleased with the comp.
Decisions and the responsiveness that we've been able to engage so far at at the stakeholder level there.
Steve maybe you can talk to clinically what the engagements been like.
Yes it.
It's.
I've been very.
Interesting you raised a good point Ken on.
The the coveted impact and how that's really shine a light on the importance of dirt.
Durability.
And ultimately the possibility of a onetime treatment and.
We are hearing that anecdotally out only for for the patients in our ongoing study, who Fortunately we've been able to have the need for reinjection eliminated.
Where.
We've seen real time patience and.
Serge areas, you've been able to.
[music].
Mr Visitor to 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 bet Jeff.
Treatment that you need and you are not risking.
Potentially the vision threatening complications by extending your visits.
Dan It really comes back to how these physicians and their patients and their their caregiver givers evaluate the benefit risk and you raised via view and PBS and that actually now redness diner.
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 can reviewed of at least 50% reduction either.
Either looking in terms of.
Treatment burden or even looking in terms of patients who can completely have need for reinjection done away with.
That hasn't changed in our minds, because it hasn't changed in the minds of.
The the investigators and our.
Retina specialist advisors and their view of what's needed for for their patients and it's interesting we don't really think of.
Any of these welcomes advances in terms of.
These.
The approved then other experimental agents that are.
Able to advance to.
Durability intervals of three to four months or five months or even six months ago.
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 onetime injection.
So we continue to see this is further validation for.
A onetime treatment and continued to see support for the target product profile at Kendall elaborated on.
Yes, and remember, we we feel that with the work we've already done in the assessment of sub retinal at multiple dose levels we have.
Outperformed that target product profile and in a very challenged in 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 the supercross. It also.
Thanks, Alex for the question.
Thank you and our next question comes from the line of Paula Musa with Chardan. Your line is open. Please go ahead.
Hi, Thanks, Thanks for taking my call.
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.
Anything you may be doing to avoid some of the growing pains that we've seen in the base gene therapy.
Space with regards to manufacture.
Yes, Bill Thanks, a lot.
I'm glad you went there because it's it's a.
Well I think you previously had been a subtle that maybe less and less subtle point for investors on these areas and I would say that even with our existing supply chain in the strong collaborations we have with.
Both manufacturers and final fill finished with groups like Fuji and others we.
We have installed capabilities.
Here in Rockville already in labs for analytics quality.
Quality assurance quality control assessment release criteria that really to not only the side of the equation, but let's not forget about the device in route of administration side here, we're working with so.
Retinal procedures, we are working with supercritical procedures were working with intracisternal procedures and intravenous procedure. So we have compatibility with all of the devices that are being sent to the sites clinically and commercially as well and that's been a major focus of quality.
Sort of strong analytical capabilities.
Assessing things across a wide range of different types of the 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 rock fill in our view is more of the come.
Commodity scale things that we need to take the next steps in terms of insulating ourselves with respect to especially that even 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 on the I think have been stumbling blocks in certainly can be especially in a new and emerging area of science in 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, John DC cost us on Photomask you I have a quick question on Joe CLM. Two programs can you. Please talk about the status of the program sense what needs to be done before you tied for the I.D. Twentytwenty one thank you.
Sure post this.
Just at a high level CST has anything else to add.
We're guiding here a two 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 read out phase and keeping in mind that certain things like the 181 program in particular is borrowing also from it.
Existing lean D. and ongoing study packages from things like one to one for instance.
We're really translating that right now into written word and the modules of DMD filing and similarly for our GXT 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 CPE one in vitreous following a sub retinal injection. So similarly.
We're in a phase right now here at the beginning of November to be filing an i. Andy by the end of the year, we're literally in the final phases of preparing the reports in.
In writing the documents to be submitted.
So.
Hope that helps.
Hi, Thank you question comes from.
Our next question comes from the line of Manny for higher risk.
FCC Inc. Your line is open. Please go ahead.
Hey, good afternoon, everyone. This is Rick dialing in for money and thanks for taking our questions.
[music].
So now that the Pivotals 314 trial is set to start 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 emporium sales, but the control arm would look like durations endpoints or any other aspect the trial design.
Okay.
Hey, Rick Thanks, no not not that we're updating today and we will plan to update that at the beginning of next year when we can.
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 been brought up on this call of course like recently.
View 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.
Our considered at Sta when it comes to wet AMD in retina. However, theres, obviously, a gene therapy bend to it and NRG X three one for Vince as well and we definitely will come forward clearly with.
More details on that study design early next year.
Steve anything else to add at this stage.
No you covered it nicely we are.
We're excited to move into the stage.
And yeah, we're about there.
Thank you and again, ladies and gentlemen, if you have a question at this time. Please press Star then one and our next question comes from the line of Liisa Walker with RBC capital markets. Your line is open. Please go ahead.
Hi, This is Lisa lots on the line card that you see at RBC.
Thanks for taking my question.
We just want to ask US we'll have an update on the IP dispute going on between you and passage bio as Wallace.
Thanks.
Hi, Lisa Thanks, a lot we don't have any specific updates to provide today we.
We have provided information previously on or I guess provided feedback.
With respect to our interest in making sure that people.
People that are working with intellectual property in science that overlaps with our NAV technology platform are under license. So that they are free to do so but also.
Fairly representing what they're doing with respect to their science and that were looking out for the interest of what we think not only our shareholders and stakeholders, but the interest of IP in general in our biotechnology space overall.
And then 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 in 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 in 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 in throughout the year. Thanks.
Thanks for the question.
Thank you and we have a follow up question from the line of Paula So with Chardan. Your line is open. Please go ahead.
Hi, Thanks, again, and this will all play small congrats on the 80 million payment on so dense mall.
I get asked a lot about how long I should model the royalties from Novartis. So.
Note is 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 should.
Sure I think of those as relevant the duration of the royalty payments or are they less relevant what can you say the one on one on.
Hey, Bill.
Thanks, one I want to take that excess one on sales clever.
The.
The work that we're doing around patent term extension and I think I mean, you all of you in the.
On the sales.
Sell side I think 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 has 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 Weve always discussed, but just to reemphasize the NAV technology platform and the.
Patents that underpin the compositions and methods of use and so forth that fall under our license is our worldwide rate and they have they have issued claims in all the jurisdiction. So we follow through with the pursuit of patent term extension as sort of normal course in each one of those jurisdictions on an individual or case by case basis.
As there are approved.
And similarly, our license in this case with.
Thanks, Chris I guess Novartis gene therapies now as we amended and expanded it includes license to a lot of patents.
In our portfolio relating to xeljanz any excess one or one.
The norm is to you know.
File and get feedback and manage the process for potential to extend in many jurisdictions up to five years, we've guided that with with respect to how quickly you will generate got approved in the us the likelihood of extending.
Or the possibility of extension is actually curved a bit it's going to be closer to I think somewhere between three and four years, but we think it is entirely appropriate to be following that process expecting that it occurs similar in nature to how bio.
Biotechnology and by biological products.
Listing products have had patents extended to protect them and that three years in the us or more in up to five years in jurisdictions like Europe, and Japan or are appropriate to consider based on how the license is structured the type of IP, that's being provided to protect so agenda and the value in it for us and Novartis gene therapy.
Yes.
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 thank you operator, thank thanks, everyone for the questions.
Most importantly, I hope everyone is safe healthy and well spent.
It's been it's been an interesting year and we definitely will be in touch with everyone that between now and the end of the year with more updates in milestones as we 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 agreed to.
Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program and you may now disconnect everyone have a great day.
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