Q3 2020 Eloxx Pharmaceuticals Inc Earnings Call

[music].

Good afternoon, everyone and welcome to the Luxe Pharmaceuticals third quarter Twentytwenty earnings webcast and conference call. Today's call is being recorded at this time I would like to turn the call over to background via the Luxco Investor Relations.

Let's begin.

Thank you operator, welcome and thank you for joining US. This afternoon for a review of the luck Pharmaceuticals third quarter Twentytwenty financial results and business update joining me. This afternoon are done.

Greg Williams, our Chief Executive Officer, Neil Beloff, Chief operating Officer, and General Counsel Dr., Tom Haverty, Our Chief Medical Officer, Dr., Matthew could there as Vice President Research and Stephen Mcdonald, Our Vice President of Finance and accounting.

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 as well as our other reports filed with the SEC any forward looking statements represent.

I'm, just finding cures for all C F patients.

The cystic fibrosis foundation's message that this is that despite the success to date, there's still work to be done is one that we D locks agree with wholeheartedly. The foundations initiative is prioritizing innovative approaches for individuals who do not respond to currently available treatments.

This includes those with nonsense mutations such as G 542, X, which is the focus of our intellects O two phase two clinical trials.

Patients with nonsense mediated cystic fibrosis represent 12% of the population according to the cystic fibrosis Foundation.

The potential ability of an investigational drug such as the elect so to to restore functional C. F. T. R protein production and these patients could be a major advance and substantially improve the length and quality of their lives.

We believe that you like so too has the potential to be an important disease modifying therapy for these patients who feel left behind and have few if any treatment options.

We are committed to advancing the development if you elect so too as quickly as possible.

As a reminder, R. C. F phase two program consists of two open label trials, one for clinical investigators enrolling patients at sites in Europe, and Israel and.

And the second to enrolling patients in the United States.

Both trials focus on C F patients with at least one G 542 X nonsense mutation.

As I mentioned earlier R. U S trial is being partially funded by the cystic fibrosis Foundation in our protocol has been endorsed by the C. F F therapeutic development network, the largest cystic fibrosis clinical trial network in the world.

In Europe are protocol has been endorsed by the E. C F S clinical trial network.

The F D. A has granted elect so to an orphan designation persistent fibrosis, which confers certain important benefits to support the development of medicines for underserved patient populations.

And parallel we continue to evaluate additional clinical trial sites and other countries, where patient enrollment may be feasible.

We continue to be focused on delivering value to shareholders, while fulfilling our mission to provide treatment options to patients with high unmet medical needs and the most safe and expeditious manner.

As our R&D team continues to advance or clinical and preclinical programs across R. E. R. S. G library, we'd accelerated the pace of publishing a results and important scientific and medical journals.

Based on our continued progress we expect that there'll be a steady cadence of additional data free electro too and a library a P. R. S. G compounds presented and published over the coming months.

Just recently, we presented cystic fibrosis data electro too and two posters at the 2020 virtual North American cystic fibrosis conference.

These data demonstrate elect so two selectivity for read through a premature stop codons and describe our studies in patient drive to organize across a wide selection of cystic fibrosis nonsense I'll ill as we continue to identify new responsive Juneau types.

Alright for our programs as well as in the business community.

We will continue to pursue partnerships where appropriate to expand our therapeutic footprint and to accelerate our progress.

We ended the third quarter of 2021.

With $30.6 million in cash and cash equivalents and with the realignment of our resources in Q1 or cash runway extends through the end of 2021.

We are well funded to deliver topline data for electro too insistent fibrosis, and the first half of 2021 and to advance the preclinical activities for El X O. Two in our library of molecules in additional indications.

We have a strong an experienced team with expertise in clinical drug development.

Research and regulatory affairs.

I'm highly confident that we have the capabilities and the resources needed to deliver on our goals.

I would now like to turn the call over to Doctor macro terrorists or Vice President of research, who will expand on our recent publications and scientific presentations.

Thank you Greg.

Continue to advance our preclinical effort across R. E. R. S E library of molecules Queen with our research partners to advance our program is.

As Greg mentioned, we are pleased to have had several of our scientific manuscripts published and leading peer reviewed journals and continued to present our findings at scientific conferences.

To date in 2020, we had presented at ARVO Association for research envision in ophthalmology, the North American cystic fibrosis fibrosis conference and we will present at the upcoming annual Israeli cystic Fibrosis Society Conference on November 19th.

In addition, we have published two scientific manuscripts, one review one original paper and have three additional original manuscripts under consideration.

The accelerated cadence of our scientific communication tracks with the progress of the <unk> discovery and development team in concert with our research partners.

And our cystic fibrosis program two posters were presented recently at the North American Cystic fibrosis conference the.

The first poster shared our findings published earlier this year in the journal of Pharmacology, an experimental therapeutics.

Demonstrating that while El X O two mediates read through a premature stop code on the fidelity of stop codons found at the end of healthy transcript is maintained.

This indicates that translational integrity is preserved with target therapeutic exposure of El XO too consistent with a favorable tolerability profile across our preclinical and clinical datasets.

The second poster discussed are ongoing effort to identify additional C. F genotype responsive to El <unk> mediated read through expanding beyond the most common nonsense <unk>, which account for over 75% of the nonsense population.

<unk> found to be responsive to <unk>.

This poster demonstrated a few key findings with CF patient derived organoid that help us better understand and interpret variability across samples with similar C. F. Genotype. These data will help US bridge the findings from our ongoing phase two CF trials to individuals with less common nonsense oleoyls.

As Greg mentioned, we're happy to announce that publication of a new manuscript on <unk> authored by Professor Etan Corinne empty.

Expert, who recently joined Ewoks as a senior medical adviser.

The manuscript is entitled <unk>.

Oh to an investigational read through agent for the treatment of nonsense mutation related genetic disease and was published on October 12th and the journal expert opinion on investigational drugs.

The manuscript reviews, the development of Yellow X O two across multiple in vitro and in vivo disease models and.

And the clinical study results, thus far representing over 100 trial participants that have received yellow so too.

This review lays the groundwork for are currently ongoing phase two trials.

The other manuscript comes out of our ocular program entitled into Vitriol administration of small molecule read through agents demonstrate functional activity in a nonsense mutation mouse model was published in the October 2nd edition of the journal of experimental I research.

This manuscript demonstrates that ER SG compounds from our library of read through molecules can be delivered into their truly to achieve read through at the back of the eye.

Alright.

G&A expense for the third quarter of 2020 $131 million, which decrease from $6.1 million for the same period in 2019.

Due to lower head count and professional services cost.

Given the realignment effective in March we expect that our cash burn rate will reach it go out in the fourth quarter of 2020 and remained fairly stable throughout 2021 with some quarter to quarter variation.

Also for your modeling purposes are total shares of common stock outstanding as of September 30th 2020, or 40.151 million.

This.

<unk>, the third quarter financial comments, and it'll turn the call back to Greg.

Thank you Steve.

Our highest priority to complete our phase two proof of concept clinical trials insistent fibrosis entry poor topline data and the first half of 2021.

We believe that these days it will be a major value inflection point for our company.

We are pleased that the independent safe to review committees for L. E. L X O two face to proof of concept clinical trials have allowed escalation up to the highest dose level and that to date no drug related serious adverse events have been reported.

The patient population. We're studying has few if any treatment options and the potential for electro too to restore the production of C. F. T. R protein could meaningfully improve the quality and length of their lives.

We are laser focused on assure you that we are investigators and clinical sites can accomplish these goals.

We're also gratified that the F. D. A has granted orphan drug designation free electro too for the treatment of cystic fibrosis, which can for several important benefits to the electro to program.

Beyond cystic fibrosis, we continue to advance our portfolio of novel E. R. S. G molecules. Several of these compounds demonstrate encouraging levels of breed true activity and Tolerability supporting their further therapeutic development.

As we continue to advance our programs there's been a marked acceleration and the number of scientific manuscripts being published an important journals. We expect this trend continue as you are in D team has a growing number of manuscripts under review.

We thank you for joining us on our third quarter Twenty-twenty earnings call and we look forward to continuing to update you on our progress. Thank you very much operator, you may know open the call for questions.

Oh, Yeah, another time Sacramento ask a question.

Starr wandering Ah telephone totally dry question center.

I'm keen on moment.

And I'm not showing any questions. At this time you May proceed with sign a reminder.

Thank you for your interest and attention.

Is an exciting time deluxe and we look forward to updating you as we continue our progress.

Thank you.

This concludes today's conference with me now disconnect Goodbye.

[music].

[music].

[music].

[music].