Abeona Therapeutics Inc (ABEO) 2017 Q3 法說會逐字稿

Greetings, and welcome to Abeona Therapeutics Third Quarter 2017 Financial Results and Recent Business Highlights. (Operator Instructions) As a reminder, this conference is being recorded.

I'd now like to turn the conference over to Christine Silverstein. Thank you. You may begin.

Thank you. Good morning, and welcome, everyone. On the call today, we have Dr. Tim Miller, President and CEO; and Jeff Davis, COO of Abeona Therapeutics. Dr. Miller will begin the call with an overview of the third quarter and more recent highlights and developments at Abeona. After, Jeff will provide an additional commentary on the quarter, a brief overview of financial summary and provide a snapshot of our financial position and review the upcoming investor conference schedule. Following that, we will open the floor to a short Q&A session.

Before I turn the call over, I need to remind our listeners that remarks made during the call can contain forward-looking statements that involve risks and uncertainties. Forward-looking statements on this call are made pursuant to the safe harbor provisions of the federal securities laws. Information contained in the federal -- forward-looking statements is based on current expectations and is subject to change, and actual results may vary materially from forward-looking statements. Some of the factors that could be -- that could cause actual results to differ are discussed in the reports filed with the SEC. These documents are available on our website at www.abeonatherapeutics.com.

With that said, it is now my pleasure to introduce Dr. Timothy Miller. Tim, you have the floor.

Good morning, everyone, and thank you for joining us this morning on our earnings call. The third quarter was marked by notable advancements in our goal of building a leading rare disease company with a focus on gene therapies.

Importantly, we initiated enrollments in our global clinical trial sites for ABO-102 for treatment of patients with MPS IIIA and reported additional data that underscores the durability and clinical benefit of this gene therapy. Our epidermolysis bullosa program achieved a very nice milestone with FDA Breakthrough Therapy designation, completed its Phase I/II clinical trial and is now continuing to advance as we finalize the clinical protocol before the initiation of the pivotal Phase III trial next year.

With regard to our MPS IIIB program, we recently initiated screening and look forward to commencing enrollments shortly. In addition, work on optimizing our AIM vector platform demonstrated exciting progress, including demonstration of enhanced tissue tropisms compared to other naturally occurring AAV capsids.

So to talk a little bit about some of our corporate achievements, we had a really great R&D Day. We held our inaugural R&D Day on October 11 in New York City, where Abeona management together with key opinion leaders and clinical experts presented an update on our clinical programs to an over 80-person crowd of institutional investors and equity analysts. We next completed an equity financing on October 19, where we announced the closing of a $92 million underwritten public offering and full exercise of the underwriters' option to purchase additional shares.

That same week, we announced a grant of up to $13.85 million from leading Sanfilippo syndrome or MPS III foundations from around the world, further supporting the clinical development of our MPS IIIA and IIIB gene therapy programs.

We had announced -- excuse me, the groundbreaking of a manufacturing facility here in Cleveland, Ohio, where we brought in multiple stakeholders in one of the rare disease families to help out with the breaking of the ground at our manufacturing facility located again in Cleveland. The 6,000-square-foot center will be built up and validated over the next 12 months, and it will be the home of multiple gene therapies and cell therapies for Abeona. The facility was named The Elisa Linton Center for Rare Disease Therapies in the honor of a young girl who passed from Sanfilippo syndrome, whose family's foundation has been instrumental in raising funds and awareness for this rare and terminal disease.

It's been brought up before, but just to clarify, Elisa was not the patient treated by Abeona. That was a different patient.

This center will be initiated to produce ABO-101 and ABO-102 gene therapies for the treatment of patients with Sanfilippo syndrome; and EB-101, our autologous cell therapy, for the treatment of recessive dystrophic epidermolysis bullosa. The center will also house Abeona's expanded viral-vector lab, which will continue to develop and produce unique and proprietary vectors for the use of delivery of gene therapies.

We announced our collaboration with Brammer Bio in September, which is the strategic alignment for commercial AAV process development, scale up and assay validation of the commercial translation of ABO-102. And we announced, of course, some of our key hires. And earlier in the quarter in July, we announced the appointment of Dr. Juan Ruiz as Chief Medical Officer. He is responsible for leading all the clinical development, medical affairs and related functions.

The recent investment from high-quality investors and leading foundations is an achievement that demonstrates our internal capabilities and commitment to the advancement of our robust pipeline and next-generation vector platform, including MPS IIIA gene therapy products. We look forward to further strengthening our efforts with key hires, advancing clinical capabilities and commercial expansion in the coming quarters.

To talk now a little bit about our clinical programs. For the EB-101 program, which is again for recessive dystrophic epidermolysis bullosa, in August, we did receive the breakthrough designation, which is for our autologous cell therapy for patients suffering from RDEB. It's noted that this is the most severe form of epidermolysis bullosa. And while the technology behind this is really -- it's a gene therapy, autologous cell corrected therapy, this is, again, very specific for the RDEB patients. The FDA granted this based on data from the Phase I/II EB-101 clinical trial, which demonstrated significant wound healing in treated wounds for over 2 years for up to 6 patients, and again going on for the seventh patient.

This designation is significant as it enables collaborative discussions with senior FDA personnel, priority review and expedited approval process to drug candidates where the preliminary clinical trials indicate that therapy may offer substantial treatment advantages over existing options for patients with serious or life-threatening diseases. EB-101 program previously received the Rare Pediatric Disease Designation in the United States, the Orphan Drug Designation in both U.S. and Europe, which are prerequisites for the FDA priority review process. Earlier in the quarter in July, we received guidance from the FDA to commence the pivotal Phase III for EB-101 and are now currently preparing to initiate the pivotal Phase III clinical trial next year.

For the ABO-102 program for the treatment of patients with MPS IIIA or Sanfilippo syndrome [FA], during the quarter, we announced enrollment and dosing of the first 2 patients in our global expansion of the Phase I/II clinical trial in MPS IIIA, 1 in Australia and 1 in Spain. Both received the Cohort 3 dose of ABO-102, which is 3x10 to the 13th vgs per kg. One additional patient in the U.S. has also received the Cohort 3 dose, bringing the total number of patients in that cohort to 3 and total number of patients in the trial to date up to 9. After seeing dose-dependent improvements in Cohort 2, with ABO-102 been well tolerated to date, Abeona together with our principal investigators and the DSMB decided to dose escalate to potentially enhance the clinical benefits and prolong durability, a decision supported by the regulatory agencies across the world really in support of the trial.

We have reported data in meeting on the mesa. In early October, we announced the top line 1-year data from the ABO-102 trial for the MPS IIIA at ARM's Cell & Gene Therapy meeting. The gene therapy demonstrated durable and significant reduction of underlying disease pathology across multiple clinical measures in Cohort 1 compared to a natural history study group of 8 to 12 subjects. Systemic biopotency demonstrated time and dose-dependent reductions of the disease-causing heparan sulfate in the CSF and reduction to liver volumes. It's important to note that reduction of the disease-causing heparan sulfate is really the key biomarker for looking at how you can modify and demonstrate that these gene therapies are getting into CNS, getting into the system and actually exhibiting and eliciting in those responses. So we think looking at heparan sulfate, again, which is I think some of the biomarkers in other trials have looked at, when you combine this with reductions in the liver and spleen volumes, changes in the deep brain architecture and preserving that, after this intravenous administration, this has led to a stabilization of neurocognitive assessment scores at 1 year post-injection. So for those of you that have heard us talk before, you heard us talk a lot about this triangle of efficacy, now looking at reductions in the biomarker, changes in the biophysical assessments from organ change in the liver, spleen and brain. And now you're starting to see some of those neurocognitive improvements.

ABO continues to be well tolerated at all doses in all follow-up time frames and has enabled an accelerated enrollment schedule over the upcoming months. We look forward to providing more fulsome clinical update in important clinical conferences, including WORLDSymposium in February and the American Society for Gene & Cell Therapy in May.

Moving on to our ABO-101 program. Currently, really this is an update on the time line. We recently initiated screening at our site in the United States and look forward to commencing enrollments and describing and discussing those coming up soon. We'll also expand this overseas into Europe going into the first quarter.

For -- just to remind everyone, ABO-101 for treatment of MPS IIIB has previously been granted the Orphan Product Designation in the United States and the Rare Pediatric Disease Designation. As we all know, those are prerequisites on part of the Priority Review Voucher process. We also look forward to discussing in 2018 additional regulatory achievements in this program, again, as those submissions continue.

Looking at our preclinical pipeline, we have a rather fulsome gene therapy pipeline, specifically, for juvenile Batten disease. We received the FDA Orphan Drug Designation for ABO-201 for the juvenile Batten program. And we are currently in IND-enabling studies for the juvenile CLN3 program. We'll be able to provide an update on the infantile Batten program coming up in January and February, as we progress with some of our regulatory meetings, and anticipate that we will -- both programs, both the CLN3 and the CLN1 will be clinical-stage programs in 2018.

And then last and really almost as exciting as the clinical-stage programs is the AIM vector program. When we initially licensed this program in from the University of North Carolina through the lab of Dr. Steven Gray, we were very excited by the prospects of finding additional vectors -- AAV vectors with tropisms specific for multiple organ systems throughout the body. Initial studies have indicated that the AIM vectors can efficiently target multiple tissues with vector-selected tissue specificity with different routes of administration relative to the first generation of vector -- AIM vectors. This, we believe, is providing us with second-generation and third-generation treatment approaches for patients as a redosing strategy or for patients that may have neutralizing antibodies to the natural AAV serotypes.

Currently, we are continuing to develop the AIM chimeric AAC vectors both internally and through the strategic partnering efforts and look forward to discussing in 2018 some of our additional undisclosed targets for gene vector platform.

So with that, that's really an overview of our clinical stage, our preclinical stage and really some of our product development. And then with that, I'll turn it over to Jeff Davis to talk about summary of our financial results.

Thank you, Tim. Our cash and cash equivalents as of September 30, 2017, were $56.5 million compared to $58.3 million as of June 30, 2017. Cash used in operating activities in the 9 months ended September 30 was $17.6 million as compared to $9.6 million in the same period of 2016. There were a couple of items that impacted our cash balance, both in the third quarter and subsequent to the quarter.

As reflected in the current Form 10-Q, the company received roughly $5.1 million in the third quarter from the proceeds of exercises of outstanding warrants. Additionally, as mentioned by Tim previously, Abeona closed on a public offering of common stock and subsequent exercise of the over-allotment option in the gross amount of $92 million. We thank our bankers, the underwriters, and most importantly, syndicate of leading investment funds that participated in the financing. As reflected in the most recent press release, as of October 31, 2017, Abeona's total cash and cash equivalents was $142.6 million.

With respect to revenues, revenues were $219,000 for the third quarter of 2017 compared to $184,000 in the second quarter of 2016. 9 months revenues, the first 9 months of this year, were $622,000 compared to $633,000 in the comparable period last year. Revenues consisted of a combination of royalties from marketed products, primarily MuGard, and the recognition of deferred revenues related to upfront payments from early licensing agreements.

Loss per share was $0.13 in the third quarter of 2017 compared to a loss per share of $0.08 in the comparable period in 2016. The total number of common shares outstanding as of the day of the filing of the Form 10-Q on November 13, 2017, which would include the shares in the recently mentioned public offering as well as shares issued in the exercise of warrants in the third quarter, is 46.7 million shares.

Before turning it over or opening up for Q&A, I just will mention some upcoming events and conferences. The week after Thanksgiving on November 30, Abeona will participate in the Barclays Gene Editing and Gene Therapy Day in New York City. Our presentation slot is 10:40 a.m. Eastern Time. On December 4, we'll be participating in Mizuho's Global Healthcare Conference in New York. And on the following day on December 5, Abeona will participate in Oppenheimer & Co's Orphan and Rare Disease Day in New York City. And I believe the format for that meeting is all one-on-one meetings.

And so with that, I think I will open it up or have the monitor open it up to Q&A.

(Operator Instructions) Our first questions come from the line of Matthew Eckler with RBC.

So when thinking about cadence for enrollment of the MPS IIIA trial going forward, how long until you think you could have around another 15 patients enrolled -- I'm sorry, 15 total?

Sure. Great question. So we're anticipating to get next 5 or 6 patients in -- through the next quarter. So with 3 sites trying to target 1 or 2 a month, we're looking to have that 15-patient mark hit right around that March, April, May time frame.

Okay, great. And building on that then, what are your current thoughts around engaging FDA on the ability to file an NDA?

We think about engaging the FDA a lot. So -- and the EMA. We are currently on track, I think, to have regulatory meetings with the FDA and EMA in the first half of next year, where I think that -- we'll be talking about the data and talking about what looks like registration 1 point. Or to talk about where they think the trials will end up going. The data is very robust to this point. And as we see the reductions in key biomarkers, such as heparan sulfate, no one else has really been able to do that. And if you can't reduce that heparan sulfate, you really don't have a therapy. So from our perspective, when we line up the heparan sulfate reductions with the organ changes, changes in the brain structure and then changes with the neurocognitive improvements and stabilization, I think we have a pretty strong case to date. So we'll be having those discussions really in the first half of next year.

Okay, great. And then last question from me. Any updates around efforts to secure license for AAV9 IP?

We're certainly looking forward to continuing discussions, and we'll have an update sometime in 2018.

Our next questions come from the line of Liav Abraham with Citigroup.

Just a question on time lines regarding the data for ABO-102. Can you just update us where you are in the collection of the 6 month neurocognitive data from the second cohort of the trial? And when is the earliest for these to be released?

Well, so we're looking at all the data together right now we have. And really with the Cohort 1 follow-up, the Cohort 2 and Cohort 3, we'll be presenting those data at conferences in the first half of 2018.

Can you be any more specific than that?

I think that we'll probably be looking at that probably at either the World Conference or the American Society for Gene & Cell Therapy.

Got it. And then you guys have a lot going on in terms of all your clinical programs. At this point, would -- are you considering partnering any of these or is it too early to be talking about that?

No. Obviously, with as -- really looking at this as a third-generation type of development on the AAVs, there are many companies that are looking for and many groups that are looking for ways to find additional transduction targets specific for different organ systems. So we're continuing to advance internally as well as with our academic partners. And think that just like anyone out there, there's certainly interest in the programs.

Our next questions come from the line of Maury Raycroft with Jefferies.

To start, can you comment on the age of the patients in Cohort 3? And given the derisking with the safety data from the initial 2 cohorts, do you think the next 6 to 9 patients you enroll may be even younger in age?

Yes, great question, Maury. Nice to hear you this morning. We -- so the average age in Cohort 1 was about 6.5, the average age in Cohort 2 was about, I think, 3.5. As we look to Cohort 3, the average around those patients is also between the ages, I think, of 3 and 4, so about 3.5. We're certainly very interested and have discussed this with the DSMB and the clinical investigators about dropping the enrollment age. So that's certainly something that we're considering at all 3 clinical sites and look forward to providing an update with that, again, in probably the first or second quarter. But it seems like a logical extension of where this therapy would go. We're also looking to solidify some relationships on working on newborn screening tests. As we look out to the 3-year horizon with an approved product just like this, really what you want to try and do is get this with a newborn screening test, because as again, with that monitor and gene therapy, it's treat earlier and treat with a significant dose. So we're looking to try and I think push that frontier as well.

Got it. Interesting. And then for EB-101 for the Phase III, so you mentioned that you're screening patients. And as far as the enrollment goes and the time lines for the process go, is harvesting keratinocytes part of the process at this point? And then what's the time line for harvesting the keratinocytes, manufacturing grafts and then administering the grafts?

That all happens over about a 4 week process, 4 to 5 weeks. So again, as we look to finalize the protocol with the FDA, not just on the clinical protocol, but also on -- again, as the Phase III trial, you're working on CMC, you're working on assay development, you're working on the commercial facility itself. So we're looking to try to accelerate that as much as possible, and that includes working with the FDA on ways to speed that process up. So when we purify out the keratinocytes from the moment the biopsy occurs to when they get treated is again about 4 or 5 weeks.

Got it. Okay. And so are you at the point where you're harvesting keratinocytes from patients yet?

Well, we certainly do that as part of the -- again, as part of the Phase III prep. We do engineering runs to go through that. And so again, we've been in that for a while now. And the great thing about it is, again, when you see this in the full GMP facility, things will be working very, very well. We're on track.

Got it. And for ABO-101, given that it's not self-complementary like ABO-102, how will this translate to efficiency of expression, and ultimately, efficacy? And can you compensate on some ways by starting initial cohort at a higher dose?

So first, let me answer the last question first. So yes, we are dosing the first cohort at a higher dose, just under our Cohort 3 dose in the MPS IIIA program. And this is all tracking to our preclinical data on what these doses were about. Dosing older animals, where you saw a complete correction of survival, neuromuscular functioning and cognitive ability. As a single-stranded DNA, you're still using the same [caps] and so know we're getting into the same tissue. We also know that we're getting very, very high levels of expression. So one of the nice things about this particular vector is it produces a bit higher than the self-complementary vectors. So from a manufacturing standpoint, there is a -- it's a bit more of a win there, because again, we can produce more of it. We've produced enough to treat roughly 15 or 18 patients to date, and again, as we look forward to announcing the enrollment of that first patient coming up very, very soon.

Our next questions come from line of Ram Selvaraju with H.C. Wainwright.

Firstly, could you comment on what is likely to be the most appropriate outcome measure with respect to measuring neurocognitive function in the (inaudible) pivotal program and maybe specifically discuss the use of the Bailey versus the Leiter and Vineland scales?

Ram, it's nice to hear you. I think that the key clinical parameters are really going to start with the measurement of heparan sulfate. Again, as we've been able to demonstrate, seeing 70% reduction in CNS as well as in urine going out through 1 year, even at the lowest dose, this is the key pathological substance that's being used by many of these -- again, in these patients. And so first and foremost, you've got to start there. Second, looking at the multiple organ changes that go on as a result of after treatment, so seeing reductions in the liver and spleen volumes, seeing preservation of deep brain architecture -- recall that this is an intravenous administration. So you don't see this -- see those type of benefits on many other ways of the treatment due to the access of the intravenous administration that can go through into the deep brain architecture with the 600 miles of using veins and arteries. So we have a 25-patient Natural History Study that was used to support the -- really the clinical development of these programs. The FDA was brought on in conjunction and collaboration in the design of that clinical trial. And so we used the Leiter and the Vineland scales in particular, where we've been able to demonstrate that level -- some levels of benefit. So we're very comfortable with those. And again, in our interactions with multiple regulatory agencies, they've been using those as well. It's important to note, Ram, that these scales are actually applicable when looking at 6 months olds up through 8-, 9-, 10-year-olds. Some of the other scales that are under consideration actually have a ceiling of 42 months. So when you've got to go back and back calculate through your Natural History Study where many patients are actually inappropriate to be used by that scale, those tend to make a little bit more of a challenge. So at least our interactions with the regulators so far have demonstrated that we're on track.

Great. And a couple of other quick things. Do you anticipate being able to utilize similar peripheral biomarkers to what have so far been used and what have demonstrated apparently statistically significant impact in the MPS IIIA program with ABO-102 versus what you anticipate being able to do with ABO-101 going forward? If you could give us a way to correlate what you might be looking at with respect to peripheral biomarkers of the ABO-101 program based on what you've learned so far with ABO-102. And then secondly, just with respect to the potential for utilizing a gene therapy approach in EB going forward as opposed to the skin grafting approach, can you give us a sense of what you're thinking about in terms of the administration paradigm for gene therapy specifically in EB?

Well, for the ABO-101 program, much of what we're doing in the ABO-102 program will directly transfer over. So we'll be looking at the same -- some very similar biomarkers. We'll be looking at liver and spleen and brain volumetric changes. We'll be using the same neurocognitive scale. So I think one of the nice things about that program is that we anticipate the results will track very similarly with the MPS IIIA program. For the EB program, the EB-101 is applicable to very large areas. So you can imagine covering a number of wounds over an EB patient's body with these iPhone-sized grafts. That's an important differentiator in this particular product. That being said, there are some areas that can be challenging for multiple reasons for durability, for accessibility, looking at the -- for example, between the areas between your fingers. So we now have EB-201 program, which is an AAV delivered way of delivering COL7A1. This is an AAV vector that has a very high tropism for epidermis and dermal tissue. Tranduces about 95% of the cells in those regions. And we already have proof-of-concept where we already have in human skin, the demonstration of COL7A1 production. And so we see these eventually being a combination approach. It's important to note that even with this type of delivery of the AAVs, we're seeing long-term expression in those areas and certainly much longer than some other different types of viruses that tend to be very quickly removed out of the skin. So again, just some important things to note on where we're going with those programs, but thanks for asking that.

And then the last thing is, based on what you've learned so far with EB-101, do you expect, especially now that you have these AAV vectors that have skin-specific tropism qualities, that there might be the possibility of your strategically moving into other rare dermatological disorders beyond EB per se, given especially the ease with which you can monitor efficacy outcomes in those kinds of conditions?

Yes, that's a fun one, so gene therapy is all about delivery. And if you've got the right vector delivered by the right disease for the right method, then you know how many you want to try and approach different diseases. So we've demonstrated really the proof-of-concept in the skin. And for now, using this type of AAV delivery certainly opens up hundreds of hundreds of rare dermatological diseases. Certainly, we're looking at that opportunistically, since again, we think we have the vector to be able to do that. So thanks.

Our next questions come from the line of Elemer Piros with Cantor.

Tim, is it okay to assume that in Australia, once you get that site up and running, you would also start with the high dose in the IIIA patients?

So the -- that's correct. So when we started -- so we enrolled the first patient already in Australia, and that patient was at the high dose at the Cohort 3.

I'm sorry, I confused it with Spain. You're correct.

Yes, also in Spain.

Okay. So thinking about the younger population, 3.5 and maybe even younger, as you're, I'm sure, maybe going down to 6 months of age. How do you determine whether an improvement or a change in, what would be neurocognitive or some other measures is not due to the early pretty good natural history or normal development of the child?

Yes, so that's been a very interesting thing for us to discuss internally and with some of our DSMB and KOLs. You're -- basically you're talking about, if you enroll like a 1-year-old, how do you attribute neurological benefits to drug treatment rather than incremental improvements that would have normally happened before the disease really took hold, is that correct?

That is correct.

Yes, so really where a lot of the fallback for demonstration of efficacy in support of the neurological scores really comes down to brain volumetric mapping. So we know from the Natural History Study where there has been significant volumetric loss, whether it's a 6-month-old or a 5-year-old. And so in support of the neurological finding, we'll be able to back that up by looking at the brain volumetrics by MRI.

I see. So you would think if there is a normal development, but a no volumetric damage yet, then that child is probably still in the normal course as opposed to having a drug effect. But on the other hand, if there is an improvement in both neurocog and brain volume, that would be much better to ascribe to an effect due to the therapy, is that -- am I getting it right?

That's correct. Also, it's important to note, Elemer, that the Leiter and the Vineland scores are applicable down to that age group. So we'll be looking at that as well as the Mullen scores in those younger patients. So we think that those are good ways to be able to assess that.

But is it safe to say that for the next 6 to 9 IIIA patients, you would stay in this 3- to 4-year-old category or it's...

Certainly, I think it's probably more important to say that we're staying with patients that have scores in the developmental IQ range or developmental score range in really a certain window. We want them to be above the floors so that, again, you can have a quantitative measurement of assessment.

And our next question comes from the line of Matthew Cross with JonesTrading.

So at your R&D Day in October, you went into some detail on the use of both the IV and intrathecal routes of administration in Batten disease programs preclinically. I was wondering if you could remind us what about this indication or the vector may require this approach to get above the maximum feasible dose for a single route of administration compared to your other programs utilizing an AAV vector.

Sure. So just to clarify, the CLN3 program is used -- as a systemic administration, but the CLN1 program is the combination dosing. I think that as you look to the future of gene therapy, what you're going to end up seeing from more programs than just us is more groups will be using combination dosing going in with whether you've got one of our third-generation AIM vectors or you're using one from one of the older generations that may have a little less of a tropism. The idea is that you want to hit as many systems that are affected by the disease as hard as possible with the gene therapy. The general [mantra] here is that overexpression or increasing the expression as a replacement strategy has really been the way to go. With the (inaudible) levels that we've seen, particularly with our AAV vectors, and again, now working into the AIM vectors, that's really where I think the field is headed. So we're excited in that infantile Batten program for CLN1 to try and champion that. And the reason why we're going that route is because -- again, this is infantile, this presents, if you catch it early enough, it presents very, very fast with a very, very high mortality. Usually before the kids hit 10 and a very, very severe disease progression. So preclinically, what the preclinical researcher, Steven Gray, has shown is that there's also a significant amount of spinal damage in this particular disease. So while -- again, all part of the CNS, and it has really been his model to try and approach this from a combination route to say you want to go intravenous, you want to go intrathecal, let's see how far we can really demonstrate the efficacy. And really in the end -- and you may actually be able to reduce some of your manufacturing burden by going both routes. So certainly we're looking at patients first and what's going to be the best way to target their disease manifestations and really how to alleviate those.

This concludes our question-and-answer session. I'd like to turn the floor back to management for any closing comments.

Thanks, everyone, for joining us today and we look forward to meeting with many of you in January and going out into the annual conferences coming up. So thanks, again, for everyone. Christine, if you have any closing comments?

That wraps it up, Tim. Thank you, everyone, for joining the call.

Thank you. This concludes today's conference. You may disconnect your lines at this time. And thank you for your participation.