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Stealth BioTherapeutics Corp (NASDAQ:MITO)
Q3 2020 Earnings Call
Nov 7, 2020, 8:30 p.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Greetings, and welcome to the Stealth BioTherapeutics Third Quarter 2020 Financial Results and Recent Business Highlights. [Operator Instructions] A brief question and answer session will follow the formal presentation. [Operator Instructions]

It is now my pleasure to introduce your host, Henry Hess. Thank you, Henry. You may begin.

Henry Hess -- Chief Legal Counsel

Good morning. I'd like to remind listeners that management will be making forward-looking statements on today's call, including, for example, expected timeline and plans for development of Elamipretide and other pipeline programs and discussion related to the company's financial position and cash runway.

Actual results may differ materially from those indicated by these forward looking statements as a result of various important factors, including those discussed in the Risk Factors section of Stealth Form 20-F filed with the SEC on April 1, 2020. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so if our views change.

Now, I'd like to turn over the call to Reenie McCarthy, Stealth's CEO. Reenie?

Reenie McCarthy -- Chief Executive Officer

Thank you, Henry. And thanks to everyone for joining us this morning to review our third quarter 2020 financial results and discuss recent business highlights. Also joining me on today's call, along with Henry are Rob Weiskopf, our Chief Financial Officer; Jim Carr, our Chief Clinical Development Officer; and Brian Blakey, our Chief Commercial Officer.

So we'll kick this off by talking about our dry AMD program. We were recently discussing the importance of mitochondrial bioenergetics for ophthalmic indications like dry AMD at a dry AMD Summit earlier this month. And it's clear from those discussions that there is growing recognition of the role of mitochondrial dysfunction in this disease. We're on the cusp of completing enrollment in our Phase 2b clinical trial in patients with geographic atrophy, we're about 90% enrolled.

We have opened some additional sites to help meet our target of completing enrollment by year-end. We've also implemented patient-centric protocol to make our sites more accessible during the ongoing pandemic, including night and weekend visits and visiting nurses. We are continually monitoring our sites as well as incident rates across the country to assess the impact of any COVID-19 related discontinuation. If necessary, we will upsize our target enrollment to meet our criteria, which could extend enrollment into early Q1. However, we have no current plans to do that.

Our core mission at Stealth is to provide life altering therapies to patients suffering from diseases involving mitochondrial dysfunction. Unlike geographic atrophy, which affects over a million eligible Americans, these other diseases are often rare diseases, sometimes even ultra rare diseases. Barth syndrome, for example, is known to affect less than a 125 Americans. These diseases have -- they are not always well understood or well characterized. In most cases, they result in a severe burden of illness, which negatively impacts patient's lives in terms of their ability to conduct activities of daily living. In some cases, they lead to early mortality in young children adolescents and adults, which is the case in Barth syndrome.

To better understand how these diseases manifest and what the unmet need really looks like, we've developed strong relationships with clinicians and patient advocacy groups. Recently, a group of over 4,000 members of the Barth Syndrome community signed a petition asking us to work with the FDA to provide access to own [Phonetic] appetite to people with Barth as soon as possible. Specifically, they ask that we submit a new drug application based on existing evidence from our clinical trials. Individuals with Barth syndrome have a reduced quality of life, and many are dying, the petition read. We need treatment choices now. We take this kind of patient feedback very seriously. We have reaffirmed our commitment to the Barth community and pledged to progress our regulatory efforts with the agency, including a planned NDA submission by year end.

As we shared during our Q2 update, however, the division of rare disease and medical genetics did not agree that our -- prior data package was sufficient to support an NDA submission. So we have conducted some additional analysis to improve the overall data package, which we believe should be accepted for review, and which we believe can meet standards for approval in light of the flexibility afforded by various FDA guidance documents in the setting of rare diseases. However, since we recognized that there is a risk that our NDA will not be accepted or approved, we are also taking steps to address the agency's recommendation to collect additional control data.

For example, DRDMG suggested that we consider a randomized withdrawal of the patients remaining on open-label extension, which would entail randomizing half of them to elamipretide and the other half to placebo for pre-specified time period, as one example of what that would look like. This could be a near-term approach to generate additional control data in the event our NDA is not accepted or approved. In addition, given the improvements in cardiac structure and function that we have observed, which differ from an expected decline in the natural history of Barth. We still -- we think that our long-term placebo controlled outcome study, assessing whether elamipretide can reduce the likelihood of material adverse cardiac events, would be a scientifically robust next step as the Phase 4 post-marketing commitment.

While we do not currently plan to request a pre-NDA meeting with DRDMG to discuss these protocols, we do plan to submit them to our IND over the upcoming weeks. We've recently presented additional data from our Barth trials, so that SPIBA-001, which was our Phase 3 long-term study to evaluate the efficacy of elamipretide compared to retrospective natural history controls, as well as SPIBA-201, which was our Phase 3 placebo controlled crossover study and open label extension. Data was presented at the American Society of Genetics in Medicine in October, demonstrating that a treatment benefit of long-term elamipretide therapy was observed in subjects with Barth, with increases in the slope of change in averaged index left ventricular stroke volume, coinciding with observed improvements and functional domain outcomes like six-minute walk. So what this may suggest is that as heart function improve, exercise tolerance and other functional endpoints also improve, which makes sense to us.

We've previously mentioned presentations over the summer at the Barth Syndrome Foundations 2020 symposium. So we won't bring you through those in detail again. Other than to note that we do expect an upcoming publication to address in more detail the improvements relative to placebo and metabolomic indicators of inefficient mitochondrial metabolism, that were observed during the placebo controlled portion of SPIBA-201, and also to note that clinical data from SPIBA-201 has now been published in Genetics in Medicine, which is a peer-reviewed medical journal.

We also previously shared with you our plans to expand our elamipretide development efforts into other rare diseases. So just going to give you a brief summary of where those efforts stand, and we can take questions if there is more details of interest that people would like to hear. So our current plan is to support an investigator-initiated Phase 2 clinical trial in patients with Friedreich's ataxia starting first quarter of next year. This trial will be open-label, it will assess the effect of elamipretide on visual and cardiac function, both of which progressively decline in the well-characterized natural history of Friedreich's. We hope that this will help us inform subsequent pivotal trial design.

We are also continuing to evaluate a protocol design for trial in patients with cardiomyopathy associated with Duchenne muscular dystrophy. Our goal is to initiate a trial in this indication during the second half of 2021. And our key opinion leaders, as well as patient advocacy have expressed support for this initiative. We also have previously mentioned that the subgroup of patients with nuclear genetic mutations improved in their distance walks during the six-minute walk test in the Phase 3 primary mitochondrial myopathy trial, and that there were no changes in placebo treated patients with these mutations.

Most of the patients in that subgroup, the nuclear DNA mutations subgroup, had mutations in the preliminaries gamma gene, which encodes for proteins involved in mitochondrial DNA replication. Mutations in other nuclear genes that similarly encode for proteins required from mitochondrial DNA replication -- these all lead to disorders called replisome related disorders. They were also included. So we are continuing to evaluate a protocol design for a Phase 3 trial in the subgroup of patients, which we would also expect to commence next year.

With respect to our pipeline neurology remains a key focus. The most advanced of our pipeline compound is SBT-272, which is a clinical stage compound that has demonstrated higher concentration in the CNS and improve penetration of the blood-brain barrier relative to elamipretide. We're pleased to share that orally administered SBT-272 demonstrated a favorable safety profile in healthy human volunteers. We are assessing drug exposure to inform formulation plans, we may explore subcutaneous dosing. Long-term tox studies will be initiated next year to enable a Phase 2 trial in patients.

With respect to indication selection, we've now observed evidence of neuronal protection in two different neurological disease models. That's the SOD1 model of ALS, where we saw improved survival and a new TDP-43 model presented at the 2020 Annual NEALS Meeting in October, where we saw improved neurite length and branching in TDP primary upper motor neurons. TDP pathology plays a role in neuronal cell death. It's been observed in multiple neurological diseases including ALS, Frontotemporal Lobar Degeneration, Lewy Body Dementia, Progressive Supranuclear Palsy and Alzheimer's.

We expect to share data and an [Indecipherable] model at an upcoming conference. This disease pathology is associated with Parkinson's, Lewy body dementia and multiple system atrophy. So together, these three clinical data reinforced our confidence in SBT-272 as a neurological disease therapeutic candidate, and that will all help us inform our Phase 2 indication. We're also excited about our 550 series for neurological indications, so it's obviously earlier in development.

We continue to have great enthusiasm for our platform potential. We're pleased with the progress we've made during what's been a really challenging year. Our team is leaning in, energized and determined to apply the important lessons we have learned to progress mitochondrial targeted therapies for patients. We are thrilled that with [Indecipherable] sites continued financial support under our recently announced development funding agreements that we're in a stronger financial position to execute on our mission and goals.

So with that, I'm going to turn the call over to Rob to review our recent financial updates and third quarter results.

Rob Weiskopf -- Chief Financial Officer

Thanks, Reenie and thank you all for joining us today. Before reviewing the financials for the quarter, I would also like to highlight a few important financial updates in the quarter. Last night, we announced the first closing under a Development Funding Agreement to support further clinical development of elamipretide. Under the terms of the agreement, Stealth will receive $20 million from Morningside Ventures to support a continued clinical development of elamipretide and up to an additional $15 million upon achievement of near-term clinical milestones associated with Stealth geographic atrophy and Barths programs. Additional investors may participate in the agreement and contribute up to an additional $35 million in funding commitments.

Subject to mutual agreement, Morningside and many other investors may also pay Stealth an additional $35 million in funding for the development of elamipretide following a pre-defined clinical milestone. Additional terms of the agreement specify that Stealth is obligated to make milestone payments following certain regulatory approvals with most payments due in the fifth through seventh year following regulatory approval. No approved payments are owed should regulatory approval not be achieved for elamipretide in the indications currently under or planned for near-term development.

Stealth has an option to buy out all the future unpaid milestone payments and any time following certain regulatory approvals at a discounted rate. Stealth continues to retain exclusive worldwide commercial rights elamipretide in all indications. In July 2020, the company also amended its term loan facility with Hercules Capital, Inc. to defer principal payments until March 1, 2021. These strategic decisions have secured our financial position through the second quarter of 2021 and ensured our continued ability to execute on the important objectives we need to discuss today.

Moving on to our third quarter results. Cash and cash equivalents were $19.9 million at September 30, 2020, compared to $50.8 million at December 31, 2019. An additional $20 million was received in October 2020 pursuant to the first closing under the Development Funding Agreement with Morningside Ventures. R&D expenses were $6.2 million for the three months ended September 30, 2020, compared to $9.8 million for the same period in 2019. The decrease was primarily attributed to a $3.2 million net decrease in employee and consultant related costs attributable to the strategic repositioning implemented in Q1 2020, a $0.7 million decrease in contract manufacturing, and a $0.5 million decrease in preclinical costs. These decreased costs were offset in part by a $0.8 million net increase in clinical trials due to the timing of trials.

G&A expenses were $4.7 million for the three months ended September 30, 2020, compared to $6.3 million for the same period in 2019. The decrease in administrative expenses resulted primarily from a decrease of $1.2 million in pre-commercial activities and a $0.8 million net decrease in employee and consultant related costs, both primarily attributable to the strategic repositioning implemented in Q1 2020. These were offset in part by a $0.4 million increase in professional services and activities attributable to the cost of various financing transactions and insurance costs.

Other expense was $0.3 million for the three months ended September 30, 2020, compared to $0.4 million for the same period in 2019. The decrease in other expense is primarily attributed to $0.3 million decrease in interest expense, offset in part by $0.2 million decrease in interest income. Net loss was $11.2 million, or $0.02 basic and diluted net loss per ordinary share for the three months ended September 30, 2020, as compared to $16.5 million or $0.04 basic and diluted net loss per ordinary share for the same period in 2019. The decreased loss was primarily attributable to a decrease in operating costs of $5.2 million and a net increase in other expenses of $0.1 million.

With that, I'd like to hand the call back to Reenie.

Reenie McCarthy -- Chief Executive Officer

Thanks, Rob. Before opening for questions, really briefly we'll highlight our upcoming milestones that we've outlined. So that will be completion of our dry AMD trial in terms of enrollment by year end, submitting our Barth NDA at the end of this year, initiating our trial and Friedreich's ataxia, early next year, followed by planned trials in Duchenne cardiomyopathy and Replisome-related disorders, all while continuing to advance SBT-272, our second-generation clinical stage compound as well as our preclinical pipeline. We're excited to be able to move forward with all these initiatives and to deliver upon our mission of developing therapies for the serious unmet medical needs affecting our patients.

And so with that, I know it's been a very busy week for a lot of reasons for many of you. So I'll turn the call over for questions. Thanks for joining.

Questions and Answers:

Operator

Thank you. We will now be conducting a question-and-answer session. [Operator Instructions] Thank you. Our first question comes from Charles Duncan with Cantor Fitzgerald. Please proceed with your question.

Charles Duncan -- Cantor Fitzgerald. -- Analyst

Good morning, Reenie. Thanks for taking our questions. Congrats on the progress and the recent Morningside financing. Well, I had a couple of questions on elamipretide and then I did want to follow up with a question on 272 if I may?

Reenie McCarthy -- Chief Executive Officer

Sure, absolutely.

Charles Duncan -- Cantor Fitzgerald. -- Analyst

Thanks for the clarity on the Barth NDA filing by end of the year. I guess, I'm wondering if really the key -- if you will, value creating event in the near term is not only the filing, but also the acceptance for review or do you feel good about the recent interactions with the Agency on Barth. And I guess, I'm wondering when you consider the comparison to natural history, yet the clinical data or observations that you've made, it seems to me that heart function improving and proceeding, functional improvement is kind of an obvious thing, but do you think that the agency has question about whether or not those observations could be effort dependent and not necessarily predictive of clinical value or do you think the Agency has -- I guess comfort around those observations?

Reenie McCarthy -- Chief Executive Officer

Well, I mean, as we shared in -- and appreciate the question. As we shared in our last update at the end of the second quarter, the Agency has expressed some concerns about the interpret ability of the changes in such a small data set with respect to cardiac function. So this was 12 patients total. We did see that most of those patients during the double blind portion of the trial, after they received elamipretide, their heart function started to improve and really then reaching statistical significance in open-label extension with long-term therapy, but it's a very small subset of patients. And you're essentially looking for approval on the basis of the surrogate endpoint, if you're looking at that. So I think that's where tracking this and looking at the correlations of time for the recent -- presentation at the American Society of Genetics is helpful.

Charles Duncan -- Cantor Fitzgerald. -- Analyst

Yeah.

Reenie McCarthy -- Chief Executive Officer

But I do think that there were clear questions from the Agency about whether we should do an additional clinical trial here. I guess, I would say that, it's a very rare disease. We think the right trial design is a post-marketing design to track long-term outcomes. We recognize the need for that to be placebo controlled, and we're hoping to your point that the Agency does except the NDA and engage with us on the discussion around what a post-marketing trial should look like.

Charles Duncan -- Cantor Fitzgerald. -- Analyst

And do you think advocacy is willing to be supportive of that general outline for a post marketing trial, and that may help the Agency understand the challenges in this patient population?

Reenie McCarthy -- Chief Executive Officer

Well, yeah, I mean in a patient population that is less than 125 patients in the United States. To get a petition signed by over 4,000 people is pretty remarkable. And that was addressed to us and to the FDA. So I do think that advocacy is supportive based on that, that's something that I think -- certainly advocacy speaks for itself and has its own materials on this posted that people can look at. But from our perspective, we certainly feel compelled to respond to that outreach. I mean, we hope that the FDA will as well. I don't know what to make of some of -- obviously in the news this week is -- or other examples of the FDA exercising regulatory flexibility or considering doing so in much larger patient population. So we're hopeful that we have a path forward here.

Charles Duncan -- Cantor Fitzgerald. -- Analyst

Yeah. I noted that as well, one of our primary thesis for [Indecipherable] is that, that innovation in terms of regulatory thinking. Moving on to the Friedreich's ataxia program that you mentioned, which is investigator-initiated. Will this be your primary -- the primary route forward for demonstrating or perhaps clinical activity with elamipretide in [Indecipherable] set of indications or will the company pursue also a Friedreich's ataxia study.

Reenie McCarthy -- Chief Executive Officer

Yeah. Our plan from that study is really -- we did this in AMP right as well. It's a good way for us to understand in the disease with a very well-characterized natural history, what endpoints we can look at. So there's two buckets of endpoints we're interested in, ophthalmic and cardiac, both decline in Friedreich's. The decline in cardiac is slower, right? So if we were just trying to prevent the decline, that might be a harder outcome for us to look at. If we actually can improve cardiac function like we've seen in Barth, that could be an interesting endpoint for development effort that we would undertake. So our hope is to be able to understand from that data, what elamipretide can do in that disease setting, and then design a controlled pivotal clinical trial to prove that.

Charles Duncan -- Cantor Fitzgerald. -- Analyst

Okay. And final question moving onto 272, you mentioned intriguing profile in preclinical animal models for neurological indications. I guess, I'm wondering when could we see 272 move into actual efficacy studies beyond just the initial safety studies? When could we see the first efficacy study outlined and started?

Reenie McCarthy -- Chief Executive Officer

Yeah. So Jim, I may ask you to help me out here. I think that what we do need to do long term talks for the neuro indications we're considering. So Jim, we are probably thinking earliest would be late next year probably into 2022. Is that right?

Rob Weiskopf -- Chief Financial Officer

I agree. I agree, Reenie.

Reenie McCarthy -- Chief Executive Officer

Yeah.

Charles Duncan -- Cantor Fitzgerald. -- Analyst

Okay. I appreciate all the color and thanks for taking our question.

Reenie McCarthy -- Chief Executive Officer

Yeah. Thanks, Charles. Appreciate you joining.

Operator

Thank you. Our next question comes from Yi Chen from H.C. Wainwright. Please proceed with your question

Yi Chen -- H.C. Wainwright & Co., LLC -- Analyst

Hi. Thank you for taking my question. Would you be able to comment on how many COVID-19 related discontinuations have occurred so far?

Reenie McCarthy -- Chief Executive Officer

Yeah. The percentages is fairly low. Jim, it's under 5% or something, right? It's -- so the percentage is fairly low at this point.

Yi Chen -- H.C. Wainwright & Co., LLC -- Analyst

Okay.

Reenie McCarthy -- Chief Executive Officer

I think that the issue is -- for us that we are looking -- we're seeing the third wave in ratcheting cases, and we just want to keep an eye on it. So I think that from our perspective, the key takeaway for this call is not that we're particularly concerned about that, but that we are also not going to -- short cut -- short change the study if we see any changes.

Yi Chen -- H.C. Wainwright & Co., LLC -- Analyst

Got it. So for the Phase 3 trial in replisome-related mitochondrial myopathies, how many patients do you plan to grow and how soon can this trial be completed?

Reenie McCarthy -- Chief Executive Officer

So I'm going to ask Jim to weigh in on this because he has done a ton of work digging into this patient population and thinking about different trial designs.

Jim Carr -- Chief Clinical Development Officer

Sure, Reenie. I appreciate the question. So the primary endpoint would likely be the six-minute walk test, since that is where we saw the strongest signal. So you can think of the trial design in the neighborhood of 100 patients. As you know, there is variability with that endpoint. We benefit from the fact that in the placebo group there was no apparent improvement in the six-minute walk test. So we will be as aggressive as we can be with assumptions for this trial. But on the flip side of that, we do have to have a reasonable sample size to give us a good success. The length of the trial really depends on how briskly we can enroll the trial. So we're hoping to have the trial under way in the second half of next year. And again, I just -- I hesitate to put a timeline on that because it's going to depend on our ability to quickly enroll it.

We're thinking with duration of follow-up of between six months in a year. We haven't necessarily land on that, but that too obviously drives the length of the trial. But the biggest concern is [Indecipherable] we can roll it. This is a subset of the primary mitochondrial disease population, so it's more rare than our previous [Indecipherable] effort. So we are going to have to be very thoughtful about the sites we choose and just how quickly we can enroll the trial.

Yi Chen -- H.C. Wainwright & Co., LLC -- Analyst

Got it. Thank you. And on the Barth syndrome, do you expect any additional FDA feedback before you submit the NDA application?

Reenie McCarthy -- Chief Executive Officer

No. We are not -- we did not request a pre-NDA -- pre-IND meeting with the Division of Rare Diseases and Medical Genetics. So -- and that's the division where the primary IND steps for that program.

Yi Chen -- H.C. Wainwright & Co., LLC -- Analyst

Got it. And final question for the Development Funding Agreement. Would you be able to provide a rough timeframe during, which you expect to receive the additional $85 million, I think.

Reenie McCarthy -- Chief Executive Officer

Yes. So Morningside commitment was for $35 million, $20 million was upfront. The balance comes on near-term milestones, which are really around dry AMD and Barth, which could be end of year, kind of early next year, assuming we meet those milestones. The balance of the -- are kind of funding capacity under that agreement -- was really intended to kind of as Morningside consents for us to have additional fundraising flexibility, should we wish to utilize it. So that's really something from management having a toolbox as we consider our overall strategies for financing the company.

Yi Chen -- H.C. Wainwright & Co., LLC -- Analyst

Okay. Thank you.

Reenie McCarthy -- Chief Executive Officer

Yes.

Operator

Thank you. Our next question comes from Matthew Luchini with BMO. Please proceed with your question.

Justin Makil -- BMO Capital Markets -- Analyst

Hi, this is Justin on for Matt. And thanks for taking our question and congrats on the progress. Two questions for me. So for the -- like could you guys provide color on in terms of where you guys are, in terms of finding investors for the second $35 million, like are they [Technical Issues] like to be assume that the second tranche available, what's the second -- what's the status on the second tranche?

Reenie McCarthy -- Chief Executive Officer

Yeah. So as I just -- as we just sort of started to try to repeat [Indecipherable] about that, it was really intended to reflect the Morningside's concerns in the Board's authorization for us to utilize this, thus, again just another sort of tool on the toolbox as it were in terms of overall fundraising strategy. It's not -- so it's not like sort of anything that we're adding timelines around or thinking about it. It's just something that should management feel that it's appropriate to bring in additional funding under that structure. We have the ability to do so.

Justin Makil -- BMO Capital Markets -- Analyst

Got it. Okay. That's helpful. And could you provide incremental color, the additional analysis that will be submitted for the NDA by year end. And if NDA were not to be accepted, like how much conviction do you guys have that the randomized withdrawal trial can support resubmission of the NDA?

Reenie McCarthy -- Chief Executive Officer

Yeah. So, I mean in terms of -- as you know, when an NDA submission is put together, there is a lot of additional analysis been done. So I don't know that I can really summarize that briefly on a call. But some of the work has entailed like further elucidation of natural history. And Jim, I don't know if you want to add anything there.

Justin Makil -- BMO Capital Markets -- Analyst

I agree, Reenie. It's just a deeper dive into the datasets we've talked about in the past.

Reenie McCarthy -- Chief Executive Officer

And then, in terms of the randomized withdrawal, I think we've shared in the past that very small number of patients, which introduced those challenges with that design. But it is a design that the FDA has told us a couple of times now. That is an option for us. And so that's really directly responding to and following FDA feedback on the program. So I think that gives us some confidence that it's the path forward.

Justin Makil -- BMO Capital Markets -- Analyst

Got it. Okay, that's helpful. Thank you. I'll hop back into queue.

Reenie McCarthy -- Chief Executive Officer

Thanks. Good to talk to you.

Operator

Thank you. Our next question comes from Maury Raycroft with Jefferies. Please proceed with your question.

Unidentified Participant

Hi. This is [Indecipherable] on for Maury. So you mentioned about possible planned resizing in dry AMD. Is that due to borrowing [Phonetic] assumptions or is there any underlying reason for it?

Reenie McCarthy -- Chief Executive Officer

You're asking about potential upsizing. That was really just a signal that we -- so we took at our COVID risk factors as a separate comment, really for COVID to the extent that there are all ongoing risk factors. It would be around that trial because those are elderly patients who are obviously in a high-risk category. And we have been looking at across the country where we're seeing spikes and infection rates, overlayered with where our sites are. Again, we haven't seen significant number of COVID related discontinuations at this point in time, but it's just something that we're going to continue to take a look at. And if that should change, because we are in the middle of another spike in the disease unfortunately, that might lead us to upsize the trial accordingly. But at this point, we have no plans to do so.

Unidentified Participant

Okay. Got it. And it seems like in response to the FDA's comments, you're preparing for both protocols like [Indecipherable] as well as the post approval. So is there possible to blending both approaches like as the near term and long-term plans financially [Phonetic]?

Reenie McCarthy -- Chief Executive Officer

Yeah. It could be. I mean, I think part of this is really a dialog with the FDA. We wanted to be responsive to their concerns, that randomized withdrawal is an approach that could be implemented more rapidly. It's a very small number of patients, and I can tell you there's not a lot of patient enthusiasm for it, just because these patients have been on therapy now for a couple of years. And obviously from that, you might infer that they wouldn't necessarily want to come off of that. But that being said, it is a near-term approach, it's responsive to the FDA's concerns. We do think that the more appropriate approach is the post marketing study. We think it's more scientifically robust. We think -- it will tell us important things about how the drug works in terms of potentially, extending lifespan for patients with this rare disease. So that's really the reason to put both on the table.

Unidentified Participant

Got it. Can you remind us [Technical Issues] precedents for patient [Technical Issues] discussions with FDA. And if this has worked in the past and how can it get the stage from [Indecipherable]?

Reenie McCarthy -- Chief Executive Officer

Yeah. This certainly is precedent for patient advocacy interactions with the FDA. I think probably EXONDYS and Sarepta would be the poster child for that. There is also precedent for the FDA exercising regulatory flexibility in the context of very rare diseases. Sarepta is an example of that, as is not [Indecipherable] drug for [Indecipherable] syndrome, which was Ultragenyx. And I think what we're seeing even this week in the biotech news with the interactions with Biogen and a disease which is much larger, right? But we are the data -- read some questions gives you some insight into ways that the FDA can exercise flexibility.

Unidentified Participant

Okay. And then for the [Indecipherable] program, have you brought any preclinical work in non-human primates can [Indecipherable] long-term?

Reenie McCarthy -- Chief Executive Officer

We have not yet done that. No.

Unidentified Participant

Okay. Thank you.

Reenie McCarthy -- Chief Executive Officer

Yeah. But we have done a Phase 1 study in people so.

Unidentified Participant

Right.

Reenie McCarthy -- Chief Executive Officer

For 272. Yeah, OK.

Operator

Thank you. [Operator Instructions] There are no further questions at this time. I would like to turn the call back over to management for any closing comments.

Reenie McCarthy -- Chief Executive Officer

Again, thank you everyone for joining today. I know it's been a incredibly busy week, both in the biotech world and in our country more broadly. So we appreciate as always your interest in our programs. Happy to chat offline, if people have other questions. Thanks for your time today. Have a good week.

Operator

[Operator Closing Remarks]

Duration: 36 minutes

Call participants:

Henry Hess -- Chief Legal Counsel

Reenie McCarthy -- Chief Executive Officer

Rob Weiskopf -- Chief Financial Officer

Jim Carr -- Chief Clinical Development Officer

Charles Duncan -- Cantor Fitzgerald. -- Analyst

Yi Chen -- H.C. Wainwright & Co., LLC -- Analyst

Justin Makil -- BMO Capital Markets -- Analyst

Unidentified Participant

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