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Eloxx Pharmaceuticals, Inc. (NASDAQ:ELOX)
Q2 2019 Earnings Call
Aug. 07, 2019, 8:30 a.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good morning, everyone, and welcome to Eloxx Pharmaceuticals' second quarter 2019 earnings webcast and conference call. Today's call is being recorded. At this time I'd like to turn the call over to Barbara Ryan, Eloxx Investor Relations Officer. Please begin.

Barbara Ryan -- Investor Relations Officer

Thank you, operator, and welcome and thank you for joining us this morning for a review of Eloxx Pharmaceuticals' second quarter 2019 financial results and business update. Joining me this morning are Robert Ward, Chairman and Chief Executive Officer of Eloxx Pharmaceuticals; Dr. Greg Williams, our Chief Operating Officer; David Snow, our Chief Business Officer; and Greg Weaver, our Chief Financial Officer.

Before we begin I would like to remind you that any statements made during this call that are not historical are considered to be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these statements as a result of various important factors, including those discussed in the risk factor section in our most recent annual report on Form 10k, filed with the Securities and Exchange Commission, and our other reports filed with the SEC. Any forward-looking statements represent our views as of today, August 7, 2019, only.

A replay of this call will be available on the company's website, www.eloxxpharma.com. It is now my pleasure to turn the call over to Robert Ward, Chairman and Chief Executive Officer of Eloxx Pharmaceuticals.

Robert Ward -- Chairman and Chief Executive Officer

Thank you, Barbara, and welcome to Eloxx's second quarter 2019 earnings webcast and conference call. We expect the remainder of 2019 to be extremely exciting for Eloxx as we expect to report topline data from our phase 2 clinical trials for our lead investigational agent ELX-02 in both cystic fibrosis and cystinosis. During the second quarter we successfully completed both our phase 1b MAD study and also the renal impairment study.

We are currently laser focused on rapidly advancing our phase 2 clinical programs in cystic fibrosis and cystinosis, and we are on track to report topline data from these studies this year. We have expanded the cystic fibrosis program to the US, where our I&D is open, and we are pleased that the protocol for our phase 2 clinical trial has been endorsed by the Cystic Fibrosis Foundation or CFF.

We are also pleased to announce that we have had three abstracts for ELX-02 accepted for presentation at the North American Cystic Fibrosis Conference this October 30, and we look forward to presenting new data at the meeting.

Our phase 2 clinical trial on cystic fibrosis will enroll no more than 24 cystic fibrosis patients carrying G542X on one or both alleles of the CFTR gene. G542X is the second most common mutation globally and accounts for about 5% of the cystic fibrosis population. These patients have a high unmet medical need and few if any targeted treatment options. ELX-02 is the only read-through therapy to have demonstrated positive results in organoids derived from cystic fibrosis patients across the majority of nonsense mutations.

In studies with approved therapies for cystic fibrosis, the organoid model has shown to be highly predictive of clinical benefit, and we believe that the positive data we have generated for ELX-02 in patient-derived organoids, representing over 75% of all nonsense mutations, substantially de-risks our phase 2 program.

We are also pleased to announce that we have initiated a Canadian phase 2 clinical trial for ELX-02 in cystinosis patients with nonsense mutations. We are grateful for the support and non-dilutive funding that we have received for this trial from Genome Quebec and Genome Canada. We look forward to reporting topline data in cystinosis from this phase 2 trial early in the fourth quarter of this year.

Additionally, our screening programs have continued to evaluate opportunities to advance ELX-02 for new indications or other novel molecules from our ERSG library. Based on our work in cystic fibrosis, we're advancing our screening work to include diseases in the lung such as primary ciliary dyskinesia and others. We continue to progress also in ocular applications, with initial focus in Usher syndrome. We believe that the results from our completed renal impairment study support expanding our research in the kidney beyond cystinosis into additional diseases such as autosomal dominant polycystic kidney disease where there's a high prevalence of nonsense mutation patients. David Snow, our Chief Business Officer, will provide you with more details on these later in the call.

We are the most advanced company tackling the great challenge of developing potential new therapies for nonsense mutations, and there's a substantial excitement in both the scientific and clinical communities for these programs, as well as in the business community, where we are actively engaged in multiple business development discussions. We ended the second quarter of 2019 with $76.3 million in cash and cash equivalents. We are well funded to advance our clinical programs and deliver topline data in both cystinosis and cystic fibrosis in 2019, as well as advance the screening programs for ELX-02 and our library of molecules into 2021.

Our talented and highly experienced team is committed to our mission of bringing safe and effective medicines to patients who need them as rapidly as possible.

I'd now like to turn the call over to Dr. Gregory Williams, our Chief Operating Officer, who will provide you with an update on our advancing phase 2 clinical programs.

Gregory Williams -- Chief Operating Officer

Thank you, Bob. I'd like to provide you with an update on our phase 2 programs for ELX-02 in cystic fibrosis and cystinosis. As Bob mentioned, we've completed phase 1, and the abstract for our multiple ascending dose study for ELX-02 has been accepted for presentation at the 2019 North American Cystic Fibrosis Conference this October.

In the US, our I&D for a phase 2 clinical trial in cystic fibrosis is open, and our protocol has been endorsed by the Cystic Fibrosis Foundation. In the US, we will enroll eight patients with the most prevalent nonsense CFTR mutation, G542X, on at least one allele. Dr. Ahmet Uluer, director of the adult cystic fibrosis program at Boston Children's Hospital, will be the lead study investigator for our phase 2 cystic fibrosis clinical trial in the US, and Dr. Eitan Kerem, head of the division of pediatrics, Children's Hospital Hadassah Medical Center, will serve as the global lead investigator. We are gratified by the participation of these two leading experts, and we look forward to reporting topline data later this year.

Our phase 2 cystic fibrosis program will include up to 24 patients in the US, Europe, and Israel. The protocol calls for four increasing doses of ELX-02, ranging from 0.3 up to 3.0 milligrams per kilogram per day in order to identify an optimal dose to carry into further development.

While patient safety is the primary endpoint in phase 2, we will be evaluating changes in sweat chloride at multiple ascending doses of ELX-02, which is consistent with other successful phase 2 programs for approved drugs as the traditional biomarker measuring CFTR activity. We will also be evaluating changes in FEV1. The changes in sweat chloride will serve as the primary biomarker for dose selection to advance into pivotal trials.

We are often asked about expectations for phase 2 results, and we believe that the Kalydeco development program can serve as a guide. Please recall that Kalydeco was able to normal patient sweat chloride levels and that sweat chloride responses in phase 2 results have shown a strong correlation to phase 3 FEV1 findings.

To support our full phase 2 clinical trial programs, we have completed the manufacturing of our lyophilized clinical drug product. We have also identified commercial manufacture and have completed key process development work supporting phase 3 clinical activity.

We have demonstrated that ELX-02 significantly increases functional CFTR protein expression in organoids from patients with nonsense mutations. We have also shown these results in human bronchoepithelial and Using chamber systems, where CFTR activity was restored to healthy controlled levels. We believe these data de-risk our phase 2 clinical trial program in cystic fibrosis. We expect to report phase 2 topline clinical trial results from our cystic fibrosis trial in the US, Europe, and Israel in 2019.

In cystinosis, we have initiated a six-patient phase 2 clinical trial in Canada, and we are grateful to have received non-dilutive funding for this trial from Genome Quebec, Genome Canada. Dr. Paul Goodyear, professor of pediatrics at McGill University and recognized leader in hereditary renal disease and cystinosis, is the principle investigator in the phase 2 clinical trial. The study will measure the dose-dependent effect of ELX-02 on cysteine levels in white blood cells, the biomarker used in the development of the most recently approved drugs for cystinosis. The study will include three nominal doses of ELX-02, ranging from 0.5 to 2.0 milligrams per kilogram per day, in order to identify an optimal dose to carry it to further development. We expect to report topline data from this phase 2 study in cystinosis early in the fourth quarter of this year.

In support of the cystinosis program, where many patients have impaired renal function, we have successfully completed a renal impairment study with ELX-02 in subjects with mild, moderate, and severe renal impairment. To date, the preliminary results support continuing our existing clinical programs and expanding our research to other kidney disorders, such as autosomal dominant polycystic kidney disease. We expect to present the results from the completed renal impairment study at a scientific meeting this year.

We continue to develop our pipeline of new indications and our library of novel compounds. We have completed screening on 30 of the most active read-through agents in our ERSG novel compound library. Eloxx holds global rights and has an extensive patent portfolio with long life on composition of matter and use for all of these compounds. We believe there are multiple opportunities to expand our pipeline by advancing these novel molecules in new routes of administration and/or by addressing new therapeutic indications.

I'd now like to ask David Snow to provide you with an update on the opportunities for our library of molecules in the inherited retinal and kidney diseases, as well as our business development opportunities.

David Snow -- Chief Business Officer

Thank you, Greg. We're all pleased with the exciting clinical progress with ELX-02 today. Despite the tremendous breakthroughs in cystic fibrosis, there remains a high unmet medical need among the CF individuals carrying nonsense mutations that represent 10-13% of the 70,000 cystic fibrosis population, and this is a distinct subset that will continue to need new therapies.

I'd also like to mention progress with the European HIT-CF program, which is now in the screen phase. The program has already identified over 40 CF patients with nonsense mutations who are participating in organoid development and further testing.

Let me now expand a bit on the additional opportunities we're exploring for ELX-02 and our library of compounds. While cystinosis is an ultra-rare disease, there's a very high unmet medical need in the nonsense mutation segment. To enable our development in this area, as Greg mentioned, we successfully completed a renal impairment study, and the preliminary results support both continuing the company's current clinical development programs and evaluating the suitability of our library of ERSG compounds for development in other kidney diseases such as autosomal dominant polycystic kidney disease or ADPKD. ADPKD represents a beyond orphan opportunity with as many as 65,000 nonsense-mediated patients in the US alone.

We've also mentioned that we have a substantial number of academic publications showing early ERSG potential across a wide variety of nonsense-mediated disease targets, and it's estimated that there are about 1,800 inherited diseases with a nonsense mutation component. We are currently evaluating the next level of inherited disease opportunities across multiple organs such as the lung, kidney, and the eye. For instance, in the inherited pulmonary disease space, beyond cystic fibrosis, we find another important unmet need in primary ciliary dyskinesia, or PCD, which has a significant nonsense population estimated at more than 30,000 individuals globally. We've also started the I&D-enabling work within inherited retinal disorders that represent more than 7,000 potential nonsense mutation affected individuals in the US with Usher or retinitis pigmentosa.

Across these early programs the Eloxx ERSG portfolio has the potential to improve the lives of more than one quarter of a million individuals affected by nonsense mutations. Importantly these specific populations often represent more severe and rapidly developing phenotypes, and there are few if any disease modifying treatments available. These important diseases and the significant unmet medical need are driving the moment behind a variety of active business development discussions. We look forward to continuing to update you on our expanding portfolio.

I now turn the call back over to Bob.

Robert Ward -- Chairman and Chief Executive Officer

Thank you, David. I'd now like to ask Greg Weaver, our Chief Financial Officer, to provide a review of our financial results for the second quarter of 2019.

Greg Weaver -- Chief Financial Officer

Thank you, Bob. As of June 30, 2019, the company had cash, cash equivalents, and marketable securities totaling $76.3 million, which we believe will fund the company's operations through topline data in cystic fibrosis and cystinosis in 2019 and maintain our current and planned operations into the first quarter of 2021. For the quarter ended June 30, 2019, the company incurred a net loss of $14.4 million or $0.40 per share, as compared to a net loss of $13.4 million or $0.42 a share for the same period 2018.

In Q2 2019 we recorded a non-cash stock-based compensation expense of $3 million, of which $2.3 million was reported to G&A and $700,000.00 to R&D. Second quarter 2019 R&D expense totaled $7.3 million, as compared to $4.2 million for the same period in 2018. Quarter to quarter increases in R&D expenditures were driven by growth in our phase 1 clinical studies, our renal study, and preparations for our multiple phase 2 trials, along with preclinical and CMC operations.

G&A expense for the second quarter 2019 was $7 million, as compared to $9.6 million for the same period in 2018. The quarter to quarter change was due largely to a significant decrease in non-cash stock-based compensation recorded to G&A, offset by increased salary related cost reflective of our year-over-year headcount growth and increases in G&A professional fees in the 2019 period.

For the six months ended June 30, 2019, the company incurred a net loss of $26.4 million or $0.73 per share, as compared to a net loss of $22 million or $0.74 per share for the same period in 2018. The $4.4 million increase in net loss was driven largely by the effect of higher R&D expenses.

Non-cash stock-based compensation expense for the first six months of 2019 was $5.7 million, of which $4.4 million was recorded to G&A and $1.3 million to R&D. R&D expenses for the first six months of 2019 totaled $13.3 million as compared to $8.5 million for the same period in 2018, an increase of $4.8 million. The year-over-year increase in R&D expenditures were primarily due to growth in our clinical and preclinical operations.

G&A expense for the first six months of 2019 was $12.9 million as compared to $13 million for the same period in 2018. For modeling purposes, total shares outstanding as of June 30, 2019 was approximately 40.0 million.

This concludes the financial comments. I'll turn the call back to Bob.

Robert Ward -- Chairman and Chief Executive Officer

Thank you, Greg. In the first half of 2019, we accelerated our clinical development efforts to conduct our phase 2 clinical trials in both cystic fibrosis and cystinosis, expanding to the US, Europe, Israel, and Canada, and are on track to reach topline data this year.

We're very pleased that our phase 2 clinical trial on cystinosis is enrolling patients with the support of non-dilutive funding from Genome Quebec and Genome Canada. We expect to report topline data from this cystinosis trial early in the fourth quarter this year.

We're pleased that our I&D in the US for cystic fibrosis is open and that our phase 2 clinical trial protocol has been endorsed by the Cystic Fibrosis Foundation. We're gratified that Dr. Ahmet Uluer from Boston Children's Hospital will lead the trial here in the US and Dr. Eitan Kerem from the Hadassah Medical Center will serve as our global lead investigator.

We're extremely encouraged by the breadth and consistency of the data we've generated from ELX-02, as well as the advanced scientific understanding of the basis of its mechanism of action. ELX-02 is the first and only read-through agent to have demonstrated changes to the CFTR messenger RNA, increases in CFTR protein expression, and substantial FIS activity in cystic fibrosis patient-derived organoids bearing nonsense mutations. We believe that the consistency of the ELX-02 data meaningfully de-risks our planned phase 2 cystic fibrosis study.

We also believe that the Kalydeco development program is useful as a guide. Please recall that Kalydeco was able to normalize patient sweat chloride levels and that the sweat chloride responses observed in phase 2 demonstrated a strong correlation to Kalydeco's phase 3 FEV1 findings. We're on track to report topline data this year from our planned phase 2 studies in both cystic fibrosis and cystinosis.

We continue to advance our portfolio of novel ERSG molecules, and we believe that several of these compounds have demonstrated encouraging levels of read-through activity as well as preliminary tolerability data supporting their potential therapeutic development. As David Snow shared with you, our screening program is identifying the best leads for continued development and generating enabling data to expand our activities to new clinical indications. We anticipate driving progress across the portfolio in both stand-alone and partnered programs. We continue to be very active in business development across our portfolio library in both a variety of indications and geographies.

Tomorrow we'll be making a presentation and hosting one-on-ones at the Canaccord Genuity Growth Conference here in Boston.

Thank you for joining us for our second quarter earnings call. We look forward to continuing to update our progress. Thank you very much. Operator, you may now open the call for questions.

Questions and Answers:

Operator

Ladies and gentlemen, if you have a question or comment at this time, please press the * then the 1 key on your touchtone telephone. If your question has been answered or you wish to remove yourself from the queue, please press the # key.

Our first question comes from Ted Tenthoff with Piper Jaffray.

Ted Tenthoff -- Piper Jaffray -- Analyst

Hi. My question actually has to do with some of your commentary in terms of the earlier states' discovery efforts because I was really pleased to hear just the breadth of that. And is the goal here really to find novel agents for these different indications? Maybe you can go into a little detail on that. Thanks for the update.

Robert Ward -- Chairman and Chief Executive Officer

Thank you, Ted. Yeah, you're correct. You know, we have a library of novel molecules, so over 100 different compounds have been synthesized over time, of which there's 30 that we view as the most active. And so one of the questions we're looking at is when you think about alternative routes of delivery -- so remember, Ted, if we talk about the eye, intravitreal administration is the route of administration for diseases of the retina or the back of the eye. When we think of systemic administration, you know, for the kidney or for cystic fibrosis, that's the route we're pursuing. We think there's other opportunities in topical or pulmonary delivery.

We also believe that there's reasons why different molecules, because of their unique characteristics, may be better suited for one indication or disease than another. So I think we're in the fortunate situation of having a number of novel molecules and then looking at the question of how will we be able to drive development across such a large number of molecules or different indications, and I'd just ask David Snow to comment.

David Snow -- Chief Business Officer

Yeah, Bob. It's been reassuring to see the progress we've made in the phase 2 program, what that can lead to, as we look at those three different organ areas in the lung, the kidney, and the eye, which each have subsequent areas of potential development. I would add that, obviously, we have this portfolio. We're looking at the opportunities there, and each of these compounds also have very long IP that provides us with a really nice opportunity.

Ted Tenthoff -- Piper Jaffray -- Analyst

Great. Excellent. Well, really exciting progress. Thanks so much.

Robert Ward -- Chairman and Chief Executive Officer

Yeah, and, Ted, I would remind you, as well, when you think of the ability to bridge to whether it's autosomal dominant polycystic kidney disease, cystinuria, primary ciliary dyskinesia, Usher syndrome, or retinitis pigmentosa, in many of these cases there are no currently approved drugs for these different populations. All of them have very active patient advocacy groups who help with identification and understand the molecular basis. So we think there's a real opportunity to expand the portfolio in-house as well as for individuals or companies that have greater expertise in different therapeutic areas to look for ways to partner as well.

Ted Tenthoff -- Piper Jaffray -- Analyst

Yep. Makes a lot of sense, and again those collaborations are so important from the scientific side. So that's great to see.

Robert Ward -- Chairman and Chief Executive Officer

Thank you, Ted.

Operator

Our next question comes from Joel Beatty with Citi.

Joel Beatty -- Citi -- Analyst

Good morning. This is Shawn Egan calling in for Joel. Thank you for taking my question. So you previously set clinical efficacy expectations for the upcoming readouts, but when assessing safety are there specific readouts that you're -- that are particularly meaningful? Or are there organ systems that are important to watch? And then as a follow-up, can you remind us, does ELX-02 cross the blood-brain barrier or enter any other immune privilege sites?

Robert Ward -- Chairman and Chief Executive Officer

Yeah, you're right, Shawn. So when we look at clinical development, as with every program, phase 1 studies begin to establish a range across which tolerability and safety enable one to go into the patient population and then study a range of doses to pick, quote, "the dose" to carry forward. So when we look at cystic fibrosis, for example, for individuals who may already be taking other drugs, we want to be sure that there's no drug-drug interactions that would be concerns. So, of course, one area that's explored during the development program is to ensure that ELX-02 does not have drug-drug interactions or other concerns.

When we talk about renal function, the drug is cleared through the kidneys. So in our renal impairment study one of the basic questions that we were looking for is, is the change in dosing required with change in renal function predictable? And I think we're very pleased that to date the results are consistent with one would expect from modeling, and we've not had any unusual observations or surprises. So this would suggest that in further work we'll be able to demonstrate the ability to dose-adjust based on kidney function in a way that will enable us to preserve safety around the drug in a wide range of individuals with both normal and/or impaired kidney function.

I think to date, with no serious adverse events being observed in the clinical program, that the emerging profile for ELX-02 is one that suggests it has potential to go all the way through and be registered successfully. So I think we're very encouraged by both the safety, tolerability at preclinical efficacy data that we've seen to date, and really the next step is to be able to understand the change in sweat chloride, observations around FEV1, changes in white blood cell cysteine levels to assess.

Now when we look at our portfolio, we're not currently targeting CNS of drug per se, but you may be aware for MPS1 there are already academic publications of other molecules from our library. And we are aware that we do have a drug that crosses the blood-brain barrier. We do believe within the library there are molecules that could be targeted toward CNS-driven disease. And when you think about immune privilege, often the eye, particularly intravitreal administration, is considered a site of immune privilege, but our reason for looking at intravitreal administration relates not so much to the immune system as it does with a fundamental understanding that we believe if we have sufficient concentrations of read-through agents in the tissue for an appropriate period of time that that would enable restoration of production of essential proteins. So as we think about different tissues, we're really looking to answer the question of what is the concentration of drug required over the study period to be able to restore production of these essential proteins.

So in many ways it's the advantage of being able to work on answering questions that have been studied before. So much as in renal impairment, there are models that predict what dosage adjustment might be run. When we run the trial, it's the question of do the trial results then -- are they consistent with what we see from modeling? And to date I think we're very pleased with the consistency of the data that we've observed with the ELX-02, being able to demonstrate a dose response for a variety of other characteristics.

Joel Beatty -- Citi -- Analyst

Thank you, Robert. I really appreciate that. And then as a brief follow up, in the renal impairment study, were there any ADPKD patients that were enrolled in that study that provided any read-through to further development in renal tissue?

Robert Ward -- Chairman and Chief Executive Officer

When we look at developing a new indication, it's really a three step process. So first we look at in vitro screens at the amount of read-through that we generate on the specific gene of interest and the specific genetic variation. So we run basically a read-through assay to assess the amount of read-through. The second step is to move into cellular models or intact animals that allow us to assess is there a reason to believe that the amount of protein being produced results in a level of functional restoration that has the potential for clinical benefit.

So think of cystic fibrosis, for example. We did both the in vitro screening and then stepped into the organoids, which is a cellular model, and, of course, have done transgenic mice and other. With the renal impairment study, this is individuals that have different levels of renal impairment, but they are not diagnosed with ADPKD. We do know that in the ADPKD population, particularly as the disease progresses, individuals have increasing levels of renal impairment. So understanding how the drug would need to be adjusted to handle renal impairment is a very important part of it, but our view on ADPKD relates to the enabling studies that we do specific to that disease.

Joel Beatty -- Citi -- Analyst

Perfect. And then just my final question. I know it's still pretty early days into the clinical study, but maybe can you provide an update on how patient identification efforts have proceeded? And also can you frame how many patients' worth of data the 2019 update will include?

Robert Ward -- Chairman and Chief Executive Officer

We'll provide a topline update later this year, and remember we are fortunate that particularly in cystic fibrosis, whether it's Vertex or the Cystic Fibrosis Foundation here in the US or the Cystic Fibrosis Research Institute or the European Cystic Fibrosis Foundation, all of these groups have really catalyzed understanding cystic fibrosis as a molecular disease. So we talk about G542X, many, many individuals within the cystic fibrosis community are aware of what genetic variation they may have and have already volunteered to be included in the databases. So from patient identification, cystic fibrosis is a very sophisticated area to work in.

Similarly with cystinosis, Dr. Paul Goodyear and McGill University have been an important part of identifying and sequencing individuals so that cystinosis can be developed as a molecular disease. You have to know the sequences to understand how many patients there are. And while it's not as advanced as cystic fibrosis, cystinosis is currently reasonably well understood in terms of both patient identification and individuals understand their own genetic variation.

Now in the ocular program, we're very pleased to be working with the Foundation Fighting Blindness. They started the My Retinal Tracker program, which we're helping to support, and that is a way for the way over 300 inherited retinal diseases to be better understood at a molecular basis. So Foundation Fighting Blindness is helping to catalyze individuals' having their genetic variation assessed and then establishing a database, which from a research perspective each of these groups has substantially contributed to why it's possible to pursue these studies. So it couldn't be done without patient advocacy groups, without patient participation, and also the contributions of many of the academic institutions.

Joel Beatty -- Citi -- Analyst

Thank you so much. Appreciate that.

Operator

Again, ladies and gentlemen, if you have a question or a comment at this time, please press the * then the 1 key on your touchtone telephone. Our next question comes from Joon Lee with SunTrust.

Joon Lee -- SunTrust -- Analyst

Hi. Thanks for the question. Based on the title of the abstracts accepted for the NACF, should we expect topline data for CF to be presented at the conference? And also my understanding is that you tested ELX-02 at 0.1 to 5 milligrams per kilo biweekly in the MAD study, but it appears you'll be testing 0.3 to 3 milligrams per kilo per day. Do you have any PK data comparing daily exposure to biweekly exposure? Thank you. And I have a follow up after that.

Robert Ward -- Chairman and Chief Executive Officer

Yeah, Joon, so just as a reminder, at the North American Cystic Fibrosis Conference, we'll have three presentations. So, for example, in the workshop on new and emerging therapies to correct the basic defect we'll have an oral presentation on how ELX-02 mediates CFTR nonsense mutation read-through to increase messenger RNA protein translation and CFTR function. Two additional abstracts will be covering the pharmacokinetics and safety in the MAD study as well as a pharmacokinetics in safety and tolerability in the single ascending dose study. So those will be the presentations at North American Cystic Fibrosis. We do anticipate being able to discuss and present our topline data this year, as well. Remember the North American Cystic Fibrosis is in October, right at the end of October, so it's a little bit early in the year for us, Joon.

And then Dr. Williams is -- Think about a dosage range that's gonna be addressed in both cystinosis and cystic fibrosis. Could you help us understand how these doses compare to what was studied in the SAD and MAD studies?

Gregory Williams -- Chief Operating Officer

Sure. So in the SAD study, we studied doses of up to 7.5 milligrams per kilogram, and in MAD we studied multiple dosing of up to 5 milligrams per kilogram. When we look at ELX-02 and we look at the pharmacokinetics, we understand that the half-life is comparatively short. It's about eight hours. So when we're moving toward cystic fibrosis and cystinosis, we're moving toward daily dosing to ensure better and more consistent exposure over time. With daily dosing, we're using lower daily doses so that the overall exposure does not exceed the exposure that has been administered to healthy volunteers in our prior studies. So the overall exposure, both in CF and cystinosis, would be consistent with the twice weekly exposure associated with the MAD study.

Joon Lee -- SunTrust -- Analyst

Great. Yes, go ahead.

Robert Ward -- Chairman and Chief Executive Officer

I was gonna say you had another question?

Joon Lee -- SunTrust -- Analyst

Oh, yes. So, you know, I'm fairly new to the story, but it appears that some nonsense mutations are more amenable to ELX-02 than others based on your CF organoid assay. Will you need to conduct studies for every nonsense mutation that are out there? Or is there a basket approach, say using the organoid assay? And do similarly credible assays exist for other diseases like in the ophthalmology space? Thank you.

Robert Ward -- Chairman and Chief Executive Officer

Yeah, I think, Joon, that's a terrific question. When you think of pioneering work in cystic fibrosis, Vertex first lead Kalydeco development focusing on G51B, which was about 5% of the total patient population, and then ran a second study that included I believe eight different genetic variations in that study. Over time, the label was expanded with individuals at younger ages because typically from a regulatory perspective we're asked to study older patients first, establishing safety and efficacy, and then moving to younger populations because remember for cystic fibrosis and for cystinosis onset occurs very early in life. So the label becomes expanded progressively by age.

And then Vertex has also pioneered why expanding to different genotypes has included a combination of clinical and preclinical data, but most importantly remember HIT-CF, funded by the European Union, done in collaboration with the European Medicines Agency, the Hub, which is a non -- Utrecht University, is collecting organoids with the intent to run a prospective trial. So today, when we or other people talk about the correlations of organoids, it's individuals who may have been in a clinical trial first and had an organoid developed later. And so many of the organoid correlations are based on retrospective data. So HIT-CF will collect the organoid first, test drugs in the laboratory to establish the organoid response, and then run a prospective clinical trial. So this will be a real test of the predictive power of organoids. We believe that if the results are positive that this would establish from a regulatory perspective an ability to say do organoids represent a laboratory test that could be the basis of label expansion?

So we do think this is a very important area of research because with genetic variation there are some variants that have a high representation in the population, G542X for example, 5% of the population. There's other variants that are quite rare. There's on variation where, as far as we know, only four people on planet Earth have been identified with that genetic variation. So that's certainly a very small population to consider running a trial. So to extend therapy to all these individuals it's very important that the ability to bridge is established, and so the HIT-CF program, the FDA is also engaged with EMA in following this closely. It represents really the potential to establish a way to more directly take some of these models into rare genetic diseases, but certainly a very important advance.

Joon Lee -- SunTrust -- Analyst

Great. That makes a lot of sense, and I think that's quite reasonable. But what about other diseases in ophthalmology? Do similar organoids like assays exist where you wouldn't have to carry the burden of doing trials for every sort of different nonsense mutation and more amenable to more of a basket approach? Thank you.

Robert Ward -- Chairman and Chief Executive Officer

No, you're correct, Joon. Whenever we talk about different proteins, right? Each unique protein really raises the same question of how do you first study the variant? How do you show the amount of protein being produced results in an important functional change? So in the eye, whether we looked at Sparks, RPE65 program, or ProQR's LCA program, you would see a similar path of in vitro studies, cellular or whole animal studies, and then in humans, functional readouts in the clinical trial.

We believe that across the inherited retinal diseases, there are some of protein variants where there are very good translation models, and there are other protein variants where there's translational models being developed. So Foundation Fighting Blindness is really serving as an important catalyst to connect up the Foundation-supported research but also with key academic groups developing many of the translation models.

Now for ADPKD, for primary ciliary dyskinesia, in these cases there are organoids that are in development or have been previously validated, so we do think that for ADPKD, for PCD, organoids will play an important role. For inherited retinal disorders right now, eye cups have been advanced to a more substantial stage than organoids, but we do think both will be important translational...

Joon Lee -- SunTrust -- Analyst

Thank you.

Operator

And I'm not showing any further questions at this time. I'd like to turn the call back over to Robert Ward, Chairman and Chief Executive Officer.

Robert Ward -- Chairman and Chief Executive Officer

Well, thanks, everyone, for joining us for our call today. For those of you who are in Boston, opportunity to talk tomorrow at 101. We will be doing a presentation at 8:00 a.m. and look forward to seeing you all on our next earnings call next quarter. Thanks for participating.

Operator

Ladies and gentlemen, this does conclude today's presentation. You may now disconnect and have a wonderful day.

Duration: 46 minutes

Call participants:

Barbara Ryan -- Investor Relations Officer

Robert Ward -- Chairman and Chief Executive Officer

Gregory Williams -- Chief Operating Officer

David Snow -- Chief Business Officer

Greg Weaver -- Chief Financial Officer

Ted Tenthoff -- Piper Jaffray -- Analyst

Joel Beatty -- Citi -- Analyst

Joon Lee -- SunTrust -- Analyst

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