Eloxx Pharmaceuticals, Inc. (ELOX) Q1 2019 Earnings Call Transcript

Eloxx Pharmaceuticals Inc. (ELOX 2.80%)
Q1 2019 Earnings Call
May 9, 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 First Quarter 2019 Earnings Webcast and Conference Call. Today's call is being recorded.

At this time, I would like to turn the call over to Barbara Ryan, Eloxx's Investor Relations Officer. Please begin.

Barbara Ryan -- Investor Relations Officer

Thank you, Liz. Welcome, and thank you for joining us this morning for a review of Eloxx Pharmaceuticals' first quarter 2019 financial results and business update.

Joining me this morning are Robert Ward, Chairman and Chief Executive Officer; Dr. Greg Williams, our Chief Operating Officer; David Snow, Chief Business Officer; Dr. Susan Schneider, Senior Vice President Clinical Development in Ophthalmology; and Greg Weaver, our Chief Financial Officer.

Before we begin, I'd 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 Factors section in our most recent Annual Report on Form 10-K 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, May 9, 2019, only. A replay of this call will be available on the Company's website, www.eloxxpharma.com.

It is now my great 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 first quarter 2019 earnings webcast and conference call. 2019 is an exciting year for Eloxx as we expect to report top-line phase two date for our lead investigational compound ELX-02 in both cystic fibrosis and cystinosis. We're pleased to have an experienced leadership team to ensure that we advance these programs as well as our new inherited Retinal Disease program on time and on budget. We continue to report broad progress in cystic fibrosis and look forward to the June European Cystic Fibrosis Annual Meeting in Liverpool where Eloxx will be presenting new data from our clinical studies.

As you may recall in March at the European Cystic Fibrosis Basic Science Meeting, we shared the data showing significant increases in CFTR protein expression and localization organoids derived from cystic fibrosis patients baring CFTR nonsense mutations. These results were consistent with our study showing functional responses to ELX-02 including increases in message RNA half-life and Dr. Greg Williams, our Chief Operating Officer, will share more of these results with you in a few moments and also provide an update on our clinical programs in cystic fibrosis and cystinosis.

We expect to initiate a phase 2 clinical trial in no more than 24 cystic fibrosis patients carrying G542X on one or both alleles of the CFTR gene. G542X is the second most common gene globally and it counts for about 5% of the total cystic fibrosis population. We are on track to report top-line data from this trial in 2019. We believe that the cystic fibrosis patient-derived organoid translational research model provides key insights for both clinical trial design and for personalized medicine. And the data we've generated substantially de-risks our phase 2 program.

Eloxx was pleased to have participated in the US Cystic Fibrosis Foundation sponsored read through a workshop that brought together scientific leaders from around the world to discuss potential solutions for developing treatments for patients with nonsense mutations. We are the most advanced company tackling the great challenge of potential new therapies for nonsense mutations. We're actively engaging with the Cystic Fibrosis Foundation here in the US and we plan to advance ELX-02 in both Europe and here in North America.

We're also pleased to be participating in HIT-CF, a European Union funded preclinical and clinical research program evaluating the efficacy and safety of several disease-modifying drug candidates in cystic fibrosis patients with rare genetic mutations. The goal of the HIT-CF European project is to investigate whether positive responses to therapies in patient-derived organoids can be predictive of clinical response in a controlled trial. We believe this project will expand the use of organoids response data to the drug approval, label expansion, treatment and reimbursement decision making processes.

We also continue to progress our ocular program under the leadership of Dr. Susan Schneider our SVP of Clinical Development in Ophthalmology. There is a high unmet medical need and prevalence of nonsense mutations across inherited retinal diseases in general. We have been advancing several new investigational product candidates from our library into IND-enabling studies in ophthalmology and we're very pleased with the emerging profile. A number of these candidates are appropriate for intravitreal administration with an encouraging pharmacokinetic profile demonstrating both retinal exposure and acceptable tolerability at high doses in sensitive species.

On April 26th, Dr. Matt Goddeeris, our Director of Research, presented a review of our read through therapeutic approach to nonsense mutation based inherited retinal dystrophies at the Sixth Annual Retinal Cell and Gene Therapy Conference at the invitation of Foundation Fighting Blindness. The presentation was well-received and has generated interest from key opinion leaders in the field. Just last week, on May 2nd, we presented new data from our Inherited Retinal Dystrophy Program at the Association for Research in Vision and Ophthalmology or ARVO at the annual meeting. Later in this call, Dr. Susan Schneider will review this presentation and highlight the important interactions we've had across a large number of key opinion leaders, investigators, and members of the Foundation Fighting Blindness. There is a growing awareness and enthusiasm for our Inherited Retinal Disease Program where our initial development efforts are focused on nonsense mutation mediated Usher syndrome which affects an estimated 4,000 individuals here in the US alone. We believe that intravitreal administration on a frequency similar to that established for Lucentis or Eyelea may allow an Eloxx therapeutic agent to address a wide range of nonsense mutation mediated inherited retinal disorders.

We ended the first quarter of 2019 with $53.5 million in cash and cash equivalence. We are well-funded to advance our clinical programs and deliver top-line data in both cystic fibrosis and cystinosis in 2019, as well as advancing our IND-enabling studies in ophthalmology. 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. You should expect that we will continue to attract and add key talent to assure that we deliver on our clinical and corporate milestones.

We are very pleased to have added to our advocacy team as we continue to expand our patient advocacy work across our key program areas. We also continue to be actively engaged in business development discussions focusing on expanding opportunities for our library of molecules in multiple areas of unmet need where nonsense mutations play an important role.

I would now like to turn the call over to Dr. Greg Williams, our Chief Operating Officer, who will provide you with an update on our clinical programs and review the recent data presented at the European Cystic Fibrosis Basic Science Meeting in March.

Greg Williams -- Chief Operating Officer

Thank you, Bob. On March 27th, we presented positive new data at the European Cystic Fibrosis Basic Science Meeting in Croatia that demonstrated for the first time that ELX-02 significantly increases CFTR protein expression and localization on the apical surface in organoids derived from patients with nonsense mutations. Consistent with increased CFTR activity observed in the organoid swelling assay, ELX-02 mediates significant restoration of CFTR protein expression as measured via a capillary-based immunoassay approach in multiple G542X and W1282X nonsense mutation carrying organoids.

G542X organoids treated with ELX-02 demonstrate proper cell surface CFTR localization on the apical surface which is consistent with increased CFTR mRNA and CFTR function in the swelling assay. While ELX-02 mediated protein increases have been previously demonstrated, this is the first demonstration reported in cystic fibrosis patient organoids. With this translational CF organoid model, ELX-02 dose-response increases in CFTR mRNA stability and function to now be extended to the demonstration of accompanying increases of CFTR protein.

We believe these organoid groundbreaking new data establish a solid basis for understanding the active ELX-02 and its potential for development in the treatment of the high unmet medical need cystic fibrosis patients with nonsense mutations. The consistency of the data demonstrating ELX-02 mediated increases in CFTR mRNA function in animal models and organoids and now the new protein data establish a solid scientific data set demonstrating the ability of ELX-02 to read through a premature stop codon in patient organoids with nonsense mutations to restore essential functional proteins. In identifying patient populations for clinical trial activity, organoids enable us to screen a variety of genotypes for ELX-02 responsiveness.

We are preparing a manuscript that compiles our expanding organoid data set. These data follow the organoid response to ELX-02 from a growing number of patient-derived organoids with the top five nonsense mutations in the CF population, which covers 75% of the nonsense baring CF patients. We believe that the patient-derived organoids represent the translational model best correlated to clinical response. In fact, a recent publication in cell reports authored by the hub in collaboration with leading academic experts in organoid technology demonstrates the high correlation of patient-derived organoid FIS responses to both the sweat chloride and fev1 change observed in the same cystic fibrosis patients treated in a variety of pivotal trials.

This data set included trials of approved CF drugs such as the potentiator Ivacaftor alone or in combination with the corrector Lumacaftor as well as Genistein and Curcumin. In the study, the office demonstrates that a positive FIS response in organoids above 2,000 significantly correlates to why the organoid model system is actively being used to evaluate compound responses across the wide landscape of CF genotypes in order to pair the right patient with the best potential therapy for their genotype.

When we consider the experimental results of ELX-02 across a variety of nonsense baring organoids, we are encouraged by the reproducible and concentration-dependent FIS response above 2,000 for a majority of the organoid data points collected and a FIS response above 4,000 for nearly half of the data set. These data support the potential for ELX-02 to provide a meaningful change in sweat chloride and fev1. We believe that the totality of this data and the substantial response observed in G542X cystic fibrosis patient organoids de-risk our phase 2 clinical trial design which focuses on patients with G542X mutations and supports our expectation that additional CF nonsense genotypes beyond G542X will be responsive to ELX-02. We are pleased that three of our abstracts, two oral and one poster, have been accepted for presentation at the European Cystic Fibrosis Society Meeting in June.

I'd now like to provide you an update on our development programs for ELX-02 and our pipeline. As previously reported, the results of our completed phase 1 SAD study were published in the Journal of Clinical Pharmacology and Drug Development in January and the emerging profile supports our continuing development program. We previously announced that we were adding additional cohorts to the MAD study to evaluate different drug concentrations. I'm pleased to report that we've initiated the seventh and final cohort of the MAD study in the US and expect to complete the study within the next two weeks. We'll then submit the results for scientific presentation or publication in 2019.

Following the completion of the MAD study, we plan to initiate a phase 2 clinical trial in cystic fibrosis and reach top-line data this year. Our clinical trial application has been approved by the Federal Agency for Medicines and Health Products in Belgium and the Clinical Trial Network of the European Cystic Fibrosis Society has given our phase 2 clinical trial a high priority rating. ELX-02 has been granted orphan designation or CF by the European Medicines Agency. We expect to extend our CF clinical and regulatory activities to allow us to conduct our work in the US as well as Europe and will update you on our progress as we move through the upcoming quarter.

In our phase 2 CF trial, we will be evaluating changes in sweat chloride multiple sending doses of ELX-02, which is consistent with other successful phase 2 programs for approved drugs as the traditional biomarker measuring CFTR activity. Our planned phase 2 clinical trial in cystic fibrosis will enroll no more than 24 patients with the most prevalent nonsense CFTR mutation, G542X, on at least one allele. The protocol calls for multiple increasing doses of ELX-02 in order to identify the optimal dose to carry out and the study would be posted on clincaltrials.gov. We expect to report phase 2 topline clinical results in cystic fibrosis in 2019.

While the patient's safety is the primary endpoint in phase 2, we will include endpoints for changes in sweat chloride and fev1 in the trial. The changes in sweat chloride was the primary biomarker for dose selection to advance into clinical 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 normalize patient's 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 program, we have completed the manufacturing of our lyophilized clinical drug product. We have also identified a commercial manufacturer and have completed key process development work supporting phase 3 clinical development.

Similarly, in cystinosis, our IND in the US is now open, and the FDA has granted ELX-02 orphan drug designation for cystinosis. We are very pleased to have alignment with the FDA on a focused phase 2 clinical trial enrolling six patients. In this study, we will be measuring the dose-dependent effect of ELX-02 on cystine levels in white blood cells, the biomarker used in the development of the most recently proved drugs for cystinosis. Cystine levels in white blood cells inform on the total cystine burden in the body and a decrease in these levels may be considered a surrogate marker likely to predict clinical benefit. We expect to report topline data from our phase two study in cystinosis this year.

As you know, cystinosis is a genetic metabolic disease typically diagnosed by the age of two. The disease is progressive with low life expectancy and most patients will have a kidney transplant by the age of 20. In support of the cystinosis program where many patients have impaired renal function, we've initiated a renal impairment study with ELX-02. We are pleased to announce that the cohorts of subjects with mild and moderate renal impairment have been successfully completed. To date, the pharmacokinetic results are as expected with no adverse events. In cystinosis, data had been previously reported at the World Symposium by Dr. Paul Goodyer, which showed that ELX-02 decreases the cystine content in cellular and animal models. He has continued his work and extended his findings to include combination use with stamen. These data were presented in March at the Cystinosis Research Foundation Day of Hope Meeting in Irvine, California.

Regarding our pipeline, we have a library of compounds and have completed screening on three of the most active read through agents in this series. Eloxx holds global rights and has an extensive patent portfolio with low life on the 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 our addressing new therapeutic indications. As Bob mentioned, in the ocular program focusing on intravitreal administration we currently have multiple compounds progressing to IND-enabling studies and our data demonstrated positive activity on nonsense mutations in inherited retinal disorders and a favorable emerging safety profile.

It is important to note that data for six different molecules from our library have been published by academic labs and show activity in a variety of ocular disorders. We are intending to focus initially on Usher syndrome. Dr. Susan Schneider will review our ocular program and very recent presentations at ARVO and the 6th Annual Retinal Cell and Gene Therapy Meetings. I look forward to keeping you apprised of our continued progress this year as we complete the MAD and renal studies, conduct both our phase 2 clinical trials in cystic fibrosis and cystinosis, and advance our ocular programs. We expect to report topline results from both of our phase 2 studies for ELX-02 this year.

I'd now like to turn the call back over to Bob.

Robert Ward -- Chairman and Chief Executive Officer

Thank you, Greg. I'd like to ask Dr. Susan Schneider to review our progress in the ocular programs.

Susan Schneider -- Senior Vice President Clinical Development in Ophthalmology

Thank you, Bob. I am very pleased to be at Eloxx at this exciting time as we build our ocular team and advance our programs. As Greg mentioned, we continue to expand our ophthalmology team and to rapidly progress our IND-enabling studies. I am pleased with the emerging pharmacokinetic and tolerability profile of the Eloxx investigational agents we are studying. We believe that our compounds are appropriate for intravitreal injection as we have demonstrated that we reach the target retina tissue with this route of administration. This is important as this is a routine outpatient procedure that is both safe and well-tolerated. Once established as appropriate for intravitreal administration, this can serve as a basis for potentially addressing a wide range of nonsense mutations mediated inherited retinal disorders.

We have also demonstrated that our compounds preserve the electroretinogram or ERG waveforms and retinal histology at concentrations where aminoglycosides commonly show ERG wave attenuation or ablation. We have studied these compounds at high doses in sensitive species. The ERG measuring electrical activity associated with nerve function within the retina and preservation of the ERG waveform is an important safety consideration. We plan to focus our initial efforts on Usher syndrome beginning with USH2A where there is a high prevalence of nonsense mutations. We have demonstrated dose responsive read through of Usher syndrome nonsense mutations as measured by protein production.

We have just returned from a series of meetings with Foundation Fighting Blindness or FFB during their Innovation Summit, the 6th Annual Retinal Cell and Gene Therapy Conference, where at their invitation Dr. Matt Goddeeris, our Director of Research, made a presentation on Friday, April 26th with our read through program in inherited retinal dystrophies. Matt's presentation was extremely well-received and the interest in inherited retinal disorders is expanding. The meeting was sold out with standing room only sessions. There is a high unmet medical need. Investigators are aware of the need for new robust data and there are clearly accelerated paths for clinical development which are accessible for registration.

We continue to build our strong and collaborative partnership with FFB. They are highly supportive of our efforts and working with us on our planned studies and regulatory interactions. FFB currently has 30 sites in their consortium. The company also made its first presentation at the Association for Research in Vision and Ophthalmology, or ARVO, Annual Meeting on May 2nd in Vancouver, which was also very well received. While at ARVO, we had the opportunity to meet with retinal key opinion leaders as well as researchers and consultants with expertise in our areas of interest. There is a high level of interest in our programs. Our primary focus remains on addressing the unmet medical need of developing therapies to treat blindness in patients with inherited retinal dystrophies. I look forward to continuing to update you on our progress.

I would now like to return the call back to Bob.

Robert Ward -- Chairman and Chief Executive Officer

Thank you, Susan. Our first quarter has been really a successful one for the company. I'd now like to ask Greg Weaver, our Chief Financial Officer, to provide you with a review of our financial results for the first quarter of 2019.

Greg Weaver -- Chief Financial Officer

Thanks, Bob. As of March 31, 2019, the company had cash, cash equivalence, and marketable securities totaling $53.5 million, which we believe will fund the company's operations through topline data in cystic fibrosis and cystinosis in 2019 and into the second quarter of 2020 based upon our current operating plans.

For the quarter ended March 31, 2019, the company incurred a net loss of $11.9 million or $0.33 per share as compared to a net loss of $8.6 million or $0.31 per share for the same period in 2018. The $3.4 million increase in quarter to quarter net loss was driven largely by the effect of non-cash stock-based compensation expense of $2.7 million in Q1 of 2019, of which $2.1 million was recorded to G&A and $500 thousand in D. For your modeling purposes, total shares outstanding at the end of the quarter was 35.9 million.

First quarter 2019 R&D expense totaled $6 million compared to $4.4 million for the same period in 2018. Quarter to quarter R&D expense fluctuations primarily due to growth in our clinical and pre-clinical operations. G&A expense for the first quarter of 2019 was $6 million as compared to $3.4 million for the same period in 2018. The quarter to quarter increase, again, due largely to the $2.1 million in non-cash stock compensation along with increased salary related costs and other G&A professional fees.

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

Robert Ward -- Chairman and Chief Executive Officer

Thank you, Greg. In 2018, we successfully built the foundation necessary for a strong growth trajectory for the company in 2019 and beyond. This year, we'll accelerate our clinical development efforts and plan to conduct our phase 2 clinical trial in cystic fibrosis in both the US and Europe and reach top-line data this year. Our phase 2 clinical trial in cystinosis will also begin with the support of Genome Canada and we expect to report top-line data on that trial this year as well.

We are pleased that our clinical trial application in Belgium for cystic fibrosis is approved and that ELX-02 has been granted an orphan drug designation by the European Medicines Agency. We're extremely encouraged by the breadth and consistency of the data we've generated for ELX-02 and by the advanced scientific understanding of the basis of the mechanism of action. ELX-02 is the first and only read through agent to have demonstrated changes in CFTR messenger RNA, increase in CFTR protein expression, and substantial FIS activity in cystic fibrosis patient-derived organoids baring nonsense mutations. The recent cell report publication from the hub demonstrates the high correlation between the FIS swelling assay and organoids and increases in both sweat chloride and fev1 shown in clinical trials for drugs currently approved for use in the treatment of the CF. We believe that the consistency of the ELX-02 data meaningfully de-risks our planned phase 2 cystic fibrosis study. We're on track to report topline data from our planned studies in both cystic fibrosis and cystinosis in the second half of this year.

We're pleased to have initiated a new program focusing on inherited retinal dystrophies and with the emerging favorable tolerability profile demonstrated by several compounds from our libraries. As Dr. Schneider discussed, we're currently conducting IND-enabling studies with an initial focus on Usher syndrome in the US and we intend to build a complete team supporting the expansion of our inherited retinal disorders program. We're gratified to have attracted industry expects to lead the acceleration of these efforts and to partner with the Foundation Fighting Blindness as we advance a novel molecule toward ocular clinical development.

We've increased our efforts in patient advocacy in cystic fibrosis and inherited retinal diseases and are very pleased with our engagements with the US Cystic Fibrosis Foundation and the Foundation Fighting Blindness. Last week, at the FFB 6th Annual Retinal Cell and Gene Therapy Innovation Summit, our ocular team had an opportunity to introduce our program to the scientific community and received a very response. We also continue to be very active in business development across our portfolio, as a variety of new indications and or geographies may become increasingly accessible to us.

Next week, we'll be participating in a Bank of America Merrill Lynch Conference on May 16th in Las Vegas and in early June we're looking forward to our three scientific presentations on ELX-02 at the European Cystic Fibrosis Society Meeting in Liverpool.

Thank you for joining us for our first quarter 2019 earnings call. We look forward to continuing to update you on our progress. Operate, you may now open the call for questions.

Questions and Answers:

Operator

Ladies and gentlemen, if you'd like to ask a question at this time, please press * then 1 on your touchtone telephone. If your questions have been answered or you wish to remove yourself from the queue, you may do so by pressing the # key. Again, that's * then 1 if you'd like to ask a question at this time.

Our first question will come from the line of Joel Beatty with Citi. Your line is now open.

Joel Beatty -- Citi -- Analyst

Hi. Thanks for taking the questions. The first one is on the multiple ascending dose study. Now that you've reached the final cohort, could you discuss how the dosing amount for this final cohort was selected? Are you able to give a sense of how it might compare to the effective dose in some of the therapeutic areas?

Robert Ward -- Chairman and Chief Executive Officer

Yeah. So, Joel, I believe when we discussed the MAD study previously -- Remember now this a longer exposure across different doses for healthy volunteers and the purpose of the study is to establish a safety range that allows us to dose escalate the patient population. Remember, when we start phase 1 with safety paramount in mind, often a dose lower than that expected to be efficacious is used. Once we move to phase 2, one of the asks from the FDA and regulatory agencies is that the first dose is one that should be expected to have a potential for patient benefit and then we dose escalate up from there to identify the optimal dose to take into phase 3.

Dr. Williams, do you want to talk about the range of doses that we've covered in the MAD study and how that bridges to what we anticipate will be the effective range we've looked at it in phase two?

Greg Williams -- Chief Operating Officer

Sure. Thank you, Bob. So, for the SAD study which provided the original range of doses to be explored, we explored single doses up to 7.5 milligrams per kilogram. That provided kind of a framework for what we were doing in the MAD study with multiple doses. Our final cohort is up to 5 milligrams per kilogram administered twice weekly. That's 10 milligrams per kilogram per week total dose. This range is consistent with the dose range that we think should be efficacious based on our animal models thus far.

Robert Ward -- Chairman and Chief Executive Officer

And for phase 2, that same dose range will be explored in both the cystinosis and cystic fibrosis patient populations and we anticipate that the same dose range would be covered in both phase 2 trails. Is that correct?

Greg Williams -- Chief Operating Officer

Yep. Absolutely, Bob. As you mentioned, with healthy volunteers we didn't push the dose perhaps as high as we might in patients because healthy volunteers don't have any potential to benefit. So, we could look at a further dose escalation in patients as we are exploring that full safety and efficacy range.

Joel Beatty -- Citi -- Analyst

Got it. A question on the organoid models that you've been using for cystic fibrosis. Could you discuss how as organoid models are being used for other drugs for cystic fibrosis and the implications of what that means for your agent?

Robert Ward -- Chairman and Chief Executive Officer

Yeah, Joel. I think it's really important to think about the fact that it's about personalized medicine. So, we know that in the Netherlands today there are individuals who because they have donated organoids that they're screened in the laboratory and then the payers are reimbursing off label use of currently approved drugs based on the organoid response that they see. So, that's already happening in the marketplace. At a more sophisticated level, with HIT-CF we have a collaboration that is funded by the European Union in collaboration with Amaya and the HUB and this is to now conduct studies and laboratory tests of the organoids to expand into a clinical program. David Snow, could you just provide an overview of how we see that program moving forward and what would be the future implications of that in the regulatory environment potentially?

David Snow -- Chief Business Officer

Bob, there's a number of important programs that we're looking at. Primary Ciliary Dyskinesia. We're looking also in the kidney effect of polycystic kidney disease. Both of these are highly prevalent diseases in the US. For PKD there's probably 140,000 to 350,000 patients in the US who have that. There is a substantial component for nonsense --

Robert Ward -- Chairman and Chief Executive Officer

David?

David Snow -- Chief Business Officer

Yes?

Robert Ward -- Chairman and Chief Executive Officer

I was thinking more of the organoid implications within the confines of HIT-CF.

David Snow -- Chief Business Officer

Oh. Sorry.

Robert Ward -- Chairman and Chief Executive Officer

Yeah. With HIT-CF. Go ahead.

David Snow -- Chief Business Officer

Sorry. HIT-CF is a very important program in Europe. We've joined as of last year. They are going to identify rare mutations in CF with a plan to do a clinical trial within the next couple of years to confirm the effect of organoids in those patients. They'll collect between 500-700 organoid patients so it will be a very robust database. We expect that they'll collect a large number of nonsense mutations in that. It will be a really good database for us as we'll participate and be able to collect that data and have really good information related to a potential regulatory path by which we could use organoids as a biomarker with a plan to work with the EMEA to use this as a way to get patients on therapy.

Robert Ward -- Chairman and Chief Executive Officer

Thanks, David. Joel, you're right. Organoids have applications in other indications, as David was mentioning, and in the eye, it's more likely that we would be using eyecups which is a similar type of approach, but it grows cells into a different structure. It's not to reflect the ability to use eye cuts as a translational mode in the ocular application. We think organoids are in the early part of development. I know the HUB has a very high interest in expanding organoids for applications in oncology. It's outside of our interests but we do think it's a translational model that will continue to have value for personalized medicine and in cystic fibrosis increasingly we believe genotype will matter. That means if I have a mutation on one allele and a different mutation on a second allele, the specific footprint of those two alleles would be my genotype.

By enabling the ability to figure out for that genotype which drug are they most likely to be responsive to. Cystic fibrosis is likely to be an area where we'll see the personalized medicine approach taking hold earlier than perhaps other areas. A lot of it is due because of the fantastic work Vertex did in both establishing and collaborating with the HUB on organoids, but also in enabling cystic fibrosis to be thought of as a molecular disease where most patients know their sequence and physicians are used to selecting drugs based on patient's underlying genetic information. So, it's really a terrific opportunity for us to take advantage of these platforms.

Joel Beatty -- Citi -- Analyst

Great. Maybe one last question on the phase 2 trials that are planned for cystic fibrosis and cystinosis. Is there an opportunity to measure mRNA and protein production in those studies? Or is it more a focus that needs to be down the road and different markers of efficacy specific to the disease areas?

Robert Ward -- Chairman and Chief Executive Officer

No. You're correct, Joel. When you think about scientific information and then the development path. So, the regulators are focused on ensuring, number one, patient safety and number two ensuring that we have measures of the potential benefit that are suitable for registration. So, if you think of cystic fibrosis, really there is a pretty well-established path of looking at sweat chlorides to select the dose for a phase 3 trial where fev1 will be the pivotal endpoint. We often look at Kalydeco as the program that kind of blazed that path, so to speak. From a scientific perspective, we think some of those questions are answered sometimes in translational models, sometimes inpatient-derived samples. So, if you think of individuals who are donating cells to have organoids made, that enables the personalized testing to occur in the laboratory. I'm not sure within the context of our specific clinical program that all of the questions you're thinking of will be answered but in the context of the program more generally. We look to answer those in a variety of different ways and sometimes it's based on patient samples.

Joel Beatty -- Citi -- Analyst

Makes sense. Great. Thank you.

Operator

Our next question comes from the line of Ted Tenthoff with Piper Jaffray. Your line is now open.

Ted Tenthoff -- Piper Jaffray -- Analyst

Great. Thank you very much and thanks for the update. A lot of really great progress taking place there. I wanted to get a little bit better color on what your thinking is now that the MAD is wrapping up with respect to phase 2 data release. Is this something that we could see by NACF or is it more likely this would be kind of a press release presentation around year end? And then I have quick follow up question if I may. Thank you.

Robert Ward -- Chairman and Chief Executive Officer

No, you're right, Ted. One of the advantages is that the European Meeting is in June, but the North American Cystic Fibrosis Meeting is later this year. The deadline for late-breaking abstracts is also later this year as well. So, as you know, with clinical trials we plan for execution that will be as rapidly as possible and prefer whenever possible to share data in the context of a scientific meeting so that the audience has the ability to see the data during the context of that meeting. So, that would definitely be our preference. It comes down to timing. We're certainly looking for opportunities to continue to engage on the scientific level.

Ted Tenthoff -- Piper Jaffray -- Analyst

Yeah. I think that would be really well-received. If I may, just on the ocular program it really sounds like this is becoming a much bigger effort for the company which I think makes a lot of sense. Can you give us a sense beyond Usher syndrome where you see other potential for read through therapy?

Robert Ward -- Chairman and Chief Executive Officer

Yeah. You know, Ted, when we think about the ability to achieve read through in a generalized way we feel that if we have the right concentration of drugs, right tissue, for the right period of time we should be able to achieve read through on many premature stop codons. Now, remember, there is a variation on read through depending on where the premature stop codon is in context with the exon junction because we've shared the data around nonsense-mediated decay. And the position of the premature stop codon does make a difference in terms of nonsense-mediated decay. And there is some other sequence related pieces that change read through levels. But like we said, in general, the right concentration of drug in the tissue for the right period of time gives us the potential to achieve read through.

For intravitreal administration, successfully achieving the right concentration in the retina or in the choroid there is a cluster of 300 different inherited retinal dystrophies that occur in those tissues. So, whether it's the retinal pigment epithelial cells, the photoreceptors, or the various nerves that make up the signal processing components of the retina, there are numerous cell types where single protein changes can make a difference in function. As we think about the proteins in the eye, not every protein is functional. So, remember, for spark with RPE65 they are restoring an enzyme that allows synthesis of visual pigment. So, we believe if it is a functional protein, which yesterday there was insufficient protein to perform the function and tomorrow protein levels are restored back to "normal" physiologic range or a range that restores function, that is something that can be measured in the clinic. So, we really want to focus our efforts on functional proteins.

Susan Schneider, as you think of other disease areas that would be of high medical need of the choroid or retinal pigment epithelial cells or the retina itself, perhaps you could explain a little bit about retinitis pigmentosa, Usher syndrome, choroideremia?

Susan Schneider -- Senior Vice President Clinical Development in Ophthalmology

Yes. Thank you, Bob. You hit the nail on the head. We're looking to enable the production of these full-length proteins in genetic diseases that are caused by nonsense mutations. So, there are a host of diseases in the inherited retinal disorder space that we could look at including retinitis pigmentosa, Leber's congenital amaurosis, and choroideremia. I would say that Usher and RP count for about half of the blindness in the US that's related to these inherited retinal disorders in general with approximately 200,000 patients in total. In that way, we are looking at more specifically the photoreceptor layer of the neurosensory retina to target the cells there too, as Bob was saying, enhance a functional output from there that would be clinically meaningful that we could move forward within clinical development.

In the other spaces that are more focused on the macula, we're also looking at levels that through an intravitreal injection our drug can get not only into the retina down to the level of the photoreceptors but also into the retinal pigment epithelium. In that way, I think our intravitreal approach is really quite exciting. We've had great feedback on that at the Innovation Summit for Foundation Fighting Blindness and also in the multitude of KOL that we had during ARVO where the feedback from retina specialists across the board was quite exciting. We think that approach is really a good, safe, well-tolerated, accepted approach to treating patients with these inherited retinal disorders.

Robert Ward -- Chairman and Chief Executive Officer

That's a very good point. Ted, one thing to remember. The Cystic Fibrosis Foundation here in the US and in many cases working with Vertex really established a basic science framework that enables research. So, patient sequences are widely available. The Foundation Fighting Blindness right now is leading the development of a similar database called My Retinal Tracker, which is an opportunity for individuals with inherited retinal diseases to donate sequences so that when we talk about understanding the molecular complexity, there are enough sequences available to understand where are there clinical development population that would allow us to conduct clinical trials and where might there be sequences that because of their frequency eyecups or organoids or translational models might be the best way to address those. So, eye research right now is a little bit earlier than cystic fibrosis. But certainly, the Foundation Fighting Blindness is really helping to catalyze collection of those sequences.

Ted Tenthoff -- Piper Jaffray -- Analyst

That's really great. Super helpful. Thanks for all the additional color.

Operator

Our next question comes from the line of Edward Nash with SunTrust Robinson Humphrey. Your line is now open.

Frank -- SunTrust Robinson Humphrey -- Analyst

Good morning. This is Frank on for Edward. Thank you for taking our questions. The first question is when we look back to phase 1 SAD study you published earlier this year you mentioned that those ranges from 0.3 and 7.5. Also, I think in the paper you mentioned that the active dose pharmacologically is about 2.5. So, clearly in the MAD study, your range is including the 2.5. I just wanted to ask what other signals that make you decide to remove the 7.5 doses and then you decide to only include the highest 5.0 mg dose?

Robert Ward -- Chairman and Chief Executive Officer

Well, I think in our phase 2 study designs, I think we're confident that we'll cover the range across which we expect to demonstrate first biologic activity. For example, if we were thinking of the Kalydeco program, when there was a 10 mmol change in sweat chloride, that would be viewed as an example of biologic activity but as the Kalydeco dose was increased there was a 55 mmol change in sweat chloride which would be normalizing sweat chloride and would be an example of why one might select that higher dose to take into pivotal trials. So, when we design the phase 2 trials, we want to make sure that we're beginning with a dose that there are reasons to believe and evidence to support that it has the potential of benefit for patients. And as we dose escalate, we're looking for a dose that optimizes clinical benefit. Now, remember, if two doses look the same from the regulatory perspective, they prefer that we use the minimal effective dose and so we would select a lower dose as a typical approach.

Dr. Williams? Additional comments?

Greg Williams -- Chief Operating Officer

Yeah. Sure. Thanks, Bob. Thank you. That's a great question. There were no safety signals at the 7.5 mg/kg dose that caused us any concern. It was well-tolerated. We just didn't think that there was any reason to expose healthy volunteers to 28-day dosing in the MAD study.

Frank -- SunTrust Robinson Humphrey -- Analyst

Great. It makes perfect sense. Thanks for that. For the abstract you're going to present in June, there's two, are they going to be from healthy volunteers? Are you going to present any of the MAD study there or is it only going to be including the SAD study?

Greg Williams -- Chief Operating Officer

Yeah. We will include both SAD and MAD data in our two papers that reference some of the healthy volunteer data.

Frank -- SunTrust Robinson Humphrey -- Analyst

Cool.

Robert Ward -- Chairman and Chief Executive Officer

I think you're referring to on June 6th, the presentation is the administration of ELX-02 to healthy volunteers demonstrates dose linearity and proportionality as well as low intrasubject variability. Then there is also a poster that will be on the pharmacokinetic profile. Those will, as Greg had shared, be the opportunity to provide those updates.

Frank -- SunTrust Robinson Humphrey -- Analyst

Great. Lastly, I think you mentioned that you ran a PK study of ELX-02 in renal impaired patients for the cystinosis, right? Can you just give us more details on what the data you have been identified and I think you mentioned that in mild or moderate renal impaired patients the PK still looked consistent with healthy volunteers if I remember correctly? Is that the case?

Robert Ward -- Chairman and Chief Executive Officer

I believe that when you think of a drug that is renaly cleared, as renal impairment incurs is the question is the increase predictable was Dr. William's comment. Greg, do you want to just go ahead and comment on the mild and moderate renal study and what our observations are to date?

Greg Williams -- Chief Operating Officer

So, we have a lot of data on the 1 mg/kg dose in healthy volunteers. We've used that dose in the SAD study. We've seen it in the MAD study with prolonged exposure. We saw very consistent results over time. We thought that was a good dose to use in the evaluation of the potential impact of renal impairment on pharmacokinetic exposure. As predicted, we've seen some slight increases in exposure. Some slight increases in half-life with the mild and then the moderate renal impaired patients. But it was exactly what we would have anticipated. We're going to continue the study into severe. So far, no adverse events. Again, predictable pharmacokinetics so it's very comforting from that perspective and it really gives us a good idea of what renal impaired patients would experience.

Frank -- SunTrust Robinson Humphrey -- Analyst

Great. Thank you for the details. I appreciate you taking our questions.

Robert Ward -- Chairman and Chief Executive Officer

Absolutely.

Operator

I'm showing no further questions in queue at this time. I'd like to turn the call back to Mr. Ward for closing remarks.

Robert Ward -- Chairman and Chief Executive Officer

Thanks, everyone, for joining us for our first quarter call. We'll look forward to updating you next quarter on our progress and are hopeful that for those of you who have the opportunity to participate in the Bank of America Merrill Lynch Conference next week in Las Vegas, we're happy to host one on ones there. We will be webcasting that presentation and look forward to continuing to update you on our programs as we progress through the year. That will conclude our call for today.

Operator

Ladies and gentlemen, thank you for your participation in today's conference. This does conclude our program and you may disconnect. Everybody have a great day.

Duration: 52 minutes

Call participants:

Barbara Ryan -- Investor Relations Officer

Robert Ward -- Chairman and Chief Executive Officer

Greg Williams -- Chief Operating Officer

David Snow -- Chief Business Officer

Susan Schneider -- Senior Vice President Clinical Development in Ophthalmology

Greg Weaver -- Chief Financial Officer

Joel Beatty -- Citi -- Analyst

Ted Tenthoff -- Piper Jaffray -- Analyst

Frank -- SunTrust Robinson Humphrey -- Analyst

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