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Edited Transcript of CATB earnings conference call or presentation 15-Mar-18 8:30pm GMT

Q4 2017 Catabasis Pharmaceuticals Inc Earnings Call

CAMBRIDGE Mar 19, 2018 (Thomson StreetEvents) -- Edited Transcript of Catabasis Pharmaceuticals Inc earnings conference call or presentation Thursday, March 15, 2018 at 8:30:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Andrea Matthews

Catabasis Pharmaceuticals, Inc. - Vice President, Corporate Affairs

* Jill Milne

Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO

* Joanne Donovan

Catabasis Pharmaceuticals, Inc. - Chief Medical Officer

* Andrew Nichols

Catabasis Pharmaceuticals, Inc. - Chief Scientific Officer

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Conference Call Participants

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* Ted Tenthoff

Piper Jaffray - Analyst

* Liana Moussatos

Wedbush Securities - Analyst

* Joel Beatty

Citi - Analyst

* Emma Nealon

Oppenheimer - Analyst

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Presentation

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Operator [1]

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Good day, ladies and gentlemen, and welcome to the Q4 2017 Catabasis Pharmaceuticals Earnings Conference Call. (Operator Instructions) I would now like to introduce your host for today's conference, Ms. Andrea Matthews. You may begin.

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Andrea Matthews, Catabasis Pharmaceuticals, Inc. - Vice President, Corporate Affairs [2]

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Thank you, Bruce. Welcome to today's Catabasis Pharmaceuticals conference call where we will provide a corporate update and review of our fourth quarter and full year 2017 financial results. With me today are Jill Milne, Chief Executive Officer; Joanne Donovan, Chief Medical Officer; Ted Hibben, Chief Business Officer; and Andrew Nichols, Chief Scientific Officer.

We issued a press release after the market closed today summarizing our corporate update and our Q4 and full year 2017 financial results, which we will reference on today's call. This press release is available on our website.

I would like to note that during today's call, we will make statements related to our business based on current and future expectations that may be considered forward-looking statements under federal securities laws. Actual results may differ materially from those indicated by these statements as a result of a variety of risks and uncertainties, including uncertainties related to the final trial design of our planned Phase 3 trial in DMD, availability and timing of top-line results for this trial, and our ability to obtain financing on acceptable terms in a timely manner to fund the certain development activities for the trial; and other factors discussed in our most recent annual report on Form 10-K, which we filed this afternoon with the SEC and is also available on our website. Such statements represent our judgment as of today, and Catabasis undertakes no obligation to publicly update any forward-looking statements except as required by law.

With that, let me pass the call over to Jill Milne, Chief Executive Officer, who will provide a corporate update. Joanne Donovan, our Chief Medical Officer, will review our clinical programs for edasalonexent and will be followed by Andy Nichols, our Chief Scientific Officer, who will provide a preclinical update. Jill will then wrap things up with the financial update. Jill?

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [3]

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Thank you, Andrea. Good afternoon, everyone, and thank you for joining us today for our fourth quarter and full year 2017 financial results and an update on progress so far in 2018. 2017 was a year of considerable progress and learning at Catabasis. We executed against a number of significant milestones, most notably setting the stage to advance our first program into Phase 3 development with edasalonexent in Duchenne muscular dystrophy.

Edasalonexent has the potential to be a foundational disease modifying therapy for patients with Duchenne muscular dystrophy. We continue to build on the strength of the edasalonexent program with consistent and encouraging data reinforcing the potential of edasalonexent for all boys regardless of mutation from initial diagnosis and continuing over a patient's life time. Edasalonexent has importantly shown potential as both a monotherapy and in combination with other treatments including dystrophin targeted therapies.

Additionally, in 2017, we made progress in our discovery programs and early stage pipeline, and are now preparing to move CAT-5571, a potential oral therapy for cystic fibrosis, into clinical development based on positive pre-clinical data. Thank you to all of our employees for helping us achieve these important goals in 2017 and for getting us one step closer to realizing our vision of improving the lives of those affected by rare diseases.

Turning to our edasalonexent program. Throughout the year, we gained valuable insights from our MoveDMD trial, and discussions with physicians and families that have guided our Phase 3 development plans. Following more than a year of treatment, edasalonexent has substantially slowed disease progression compared to control in this otherwise inevitably progressing disease. As a reminder, symptoms of muscle weakness are seen in boys with Duchenne often before age 4. And by age 12, boys are often non-ambulatory.

Slowing down this predictable course of muscle degeneration and weakness is the primary concern for boys' families and physicians. We were very pleased to present additional data earlier this week on edasalonexent as well as new independent natural history data at the Muscular Dystrophy Association Clinical Conference that further support the potential of edasalonexent as a foundational therapy in Duchenne.

I will now turn the call over to Joanne Donovan, our Chief Medical Officer, who will review the edasalonexent MoveDMD data through more than a year of treatment as well as the information presented earlier this week at MDA and our Phase 3 plans. Andy Nichols, our Chief Scientific Officer, will then review the preclinical results that were generated with edasalonexent last year and provide an update on our CAT-5571 program in cystic fibrosis. Joanne?

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [4]

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Thank you, Jill, and good afternoon, everyone. Last month, at the 16th International Conference on Duchenne and Becker Muscular Dystrophy, we presented data for more than a year of edasalonexent treatment in the MoveDMD trial. I will provide an overview of these results today, and we'll also share the preparations that are well underway for our Phase 3 registration trial.

We were delighted to see long-term consistency of effects of edasalonexent with a preservation of muscle function in boys with Duchenne over the course of more than a year of treatment compared to control. And as we looked at the potential edasalonexent as foundational therapy over the lifetime for a Duchenne patient, we're pleased to see that it continued to be well tolerated with a favorable safety profile.

In the MoveDMD trial, we enrolled boys aged 4 to 7 who were not on corticosteroid. In the trial, we measured changes in functional abilities over a period prior to the Phase 2 treatment period and pre-specified our analysis plans to compare the rates of change during this control period to the treatment period on edasalonexent. We measured four assessments of muscle function that reflect everyday activities that are appropriate for 4- to 7-year old boys. The assessments were the time function tests, 10-meter walk/run, four-stair climb, and time to stand, as well as the North Star Ambulatory Assessment, a composite measure designed specifically for Duchenne.

I'll now walk you through the results of each of the assessments following 48 and 60 weeks of edasalonexent treatment, which are consistent with what we saw at earlier time points. As a reminder, these results are for boys that received 100 milligrams per kilogram of edasalonexent throughout the entire treatment period.

Across every measure, the rates of functional decline in boys not only slowed, but in fact stabilized through 48 and 60 weeks of treatment compared to control. Remember, these boys were declining as expected during the off-treatment control period on all four of the assessments of muscle function. This is an incredibly promising result, and KOLs that we've talked to about the data interpret them as edasalonexent substantially slowing the progression of Duchenne in the boys.

As you will recall, the North Star Ambulatory Assessment is a validated composite measure of 17 physical function tests, including the ability to stand or walk, run or hop on one leg, with each activity assessed by a clinical evaluator. The annualized rate of decline was about four points per year.

Just as we saw at 24 and 36 weeks following 48 and 60 weeks of treatment, the rate of decline stabilized compared to control. Some of the boys even gained new abilities that they did not have during the control period; being able to run or jump. You can imagine the incredible impact this has on the boys and their family.

Based on discussions with the FDA, we plan to use the North Star test as our primary endpoint in the upcoming Phase 3 trial. For the 10-meter walk/run, boys experienced an 18% annualized rate of decline in speed during the off-treatment control period. While on edasalonexent, the boys' speed was maintained. The average speed to perform the four-stair climb and time to stand also showed a slowing in the rate of decline over 60 weeks of edasalonexent treatment as compared to a 23% annualized rate of decline in speed for the four-stair climb and a 35% annualized rate of decline in time to stand during the off-treatment control period.

As seen in third party natural history of Duchenne boys not on steroids that I'll discuss in a minute, boys at the same ages as in the off-treatment period control period of our study follow a predictable and steady decline across these tests of their muscle function. Therefore, the consistent slowing of disease progression through more than a year of edasalonexent treatment across all these well-accepted functional assessments is very compelling and encouraging.

Supporting these results, we also saw significant changes in muscle health over these longer treatment periods that are also consistent with positive edasalonexent treatment effects. Four muscle enzymes were measured in the MoveDMD trial that are known to increase following muscle damage. Following treatment with edasalonexent, the levels of all four enzymes showed statistically significant decreases compared to baseline through 60 weeks of treatment suggesting a decrease in muscle injury and an improvement in muscle integrity.

And as we reported last month C-reactive protein or CRP, there is a statistically significant decrease after 36 and 48 weeks of treatment consistent with the results we saw after 12 and 24 weeks of treatment. CRP is a well characterized blood test marker that provides a global assessment of inflammation and is known to be elevated in boys affected by Duchenne. The significant decrease observed in the CRP supports biological activity of NF-kappaB inhibition by edasalonexent treatment decreasing inflammation.

We also have an update on the boys who are receiving EXONDYS 51 treatment while continuing edasalonexent in the ongoing MoveDMD open label extension. We're evaluating safety and learning more about the potential of combination therapy. At this point, we have more than six months of data in combination, and we've continued to see a good safety and tolerability profile.

We presented additional data earlier this week at the 2018 Muscular Dystrophy Association Clinical Conference. Edasalonexent continues to be well tolerated with no safety signal observed to date in the MoveDMD trial; now with over 35 patient years of pediatric exposure. There are boys in the MoveDMD trial that have been on edasalonexent for close two years. Most adverse events have been mild in nature with no edasalonexent-related serious adverse events. The safety profile is key as we view edasalonexent as a potential lifelong foundational treatment used as both a monotherapy and in combination with other therapies.

We've seen additional observation suggesting that edasalonexent may have benefits on the heart, boys with Duchenne in the age range of those in our study typically have resting tachycardia, a heart rate that exceeds the normal resting rate. Tachycardia is the first cardiac manifestation in boys with Duchenne. This elevated heart rate was observed in boys in the DMD trial both while boys who were receiving placebo as well as during the off-treatment control period. And we're excited that new data show that the heart rate of boys treated with edasalonexent decreased towards age normative values through 48 weeks of treatment.

When boys originally randomized to placebo moved on to edasalonexent at the start of the open label extension period, their heart rate decreased. These results are further supported by positive heart effects seen with a reduction in cardiac fibrosis in preclinical models. We believe these clinical heart rate observations show early clinical indication that edasalonexent could ultimately have important heart effects in Duchenne. A key target in this disease as cardiac failure is a leading cause of mortality.

Also of note, boys with edasalonexent continue to progress along standard growth curves for unaffected boys through 60 weeks of treatment, increasing their height and weight on track with their friends at school. BMI trended toward a decrease. For quality of life for these boys, this is very important to grow and develop like their peers. Edasa's profile is favorably differentiated from the typical profile associated with the corticosteroid standard of care in Duchenne, which includes delayed puberty, weight gain, and stunted growth. For example, in the MoveDMD trial over 48 weeks, the boys gained an average of half a kilo consistent with normal growth. On the other hand, in other studies, boys in deflazacort over a year gained an average of 5 kilos and for prednisone 8 kilos.

Also presented earlier this week at the MDA Clinical Conference were new data from the ImagingDMD Natural History Study, a collaboration led by the University of Florida and independent of the MoveDMD trial. ImagingDMD assessed annual measures of the same time function test that we assessed in the MoveDMD trial. There were 28 boys initially aged 5 to 8.5 years old who did not take corticosteroids during this study. The ImagingDMD Natural History Study and our MoveDMD trial were performed at the same clinical site using the same protocols for the assessment.

The observations of the ImagingDMD Natural History Study were generally consistent with declines in the abilities experienced by boys during the off-treatment control period in the MoveDMD trial and the absolute values of the timed function tests were also consistent. These data provide important corroboration that the MoveDMD off-treatment control period observations are characteristic of the expected natural history and provide additional confidence in the slowing of disease progression treatment effects observed with edasa.

These data, along with input from FDA, from KOLs, from families, having informed a rigorous and meaningful design for our Phase 3 study, we have a clear path to registration. We are planning to initiate a single global Phase 3 trial with many fundamental elements in common with our MoveDMD trial, including the patient population and key endpoints.

The patient population designed for this Phase 3 trial will include boys age 4 through their 7th birthday regardless of mutation type and who have not been on steroids for at least six months. We expect to enroll approximately 125 boys and plan to have the primary endpoint be the North Star Ambulatory Assessment following 12 months of the edasa treatment compared to placebo. We also plan to include the same time functional test as the MoveDMD trial. We're planning to follow up on the differentiated safety profile of edasa, and include cardiac and bone measures at baseline and following treatment.

Preparations are well underway for our Phase 3 trial. We've made progress both on clinical operations as well as CMC. We've explored sites for the study globally and we have edasa drug supply ready, and we look forward to initiating the Phase 3 trial in the coming months.

Earlier this month, I attended a meeting with advocates of the Duchenne community, including a number of families impacted by the disease. While I've attended dozens of these meetings over the course of my career, I continue to be struck by the tireless efforts and perpetual hope of these families. Their support is deeply appreciated and their enthusiasm is infectious. I want to personally thank them for sharing their stories and their challenges to help us in our effort to improve their lives.

I'll now pass the call over to Andy Nichols, our CSO, who will share updates on pre-clinical data. Andy?

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Andrew Nichols, Catabasis Pharmaceuticals, Inc. - Chief Scientific Officer [5]

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Thank you, Joanne, and good afternoon, everyone. In 2017, we augmented our package of preclinical data for our edasa program with additional results. We demonstrated metabolism of edasa in human muscle fibers to its active components confirming availability of the component bioactives in the target tissue to inhibit NF-kappaB as desired. We also saw human myocyte growth and differentiation, and concentrations of edasa that are consistent with those achieved during patient dosing, supporting the potential of edasa to regenerate muscle fibers. We see this as an important function that could provide valuable contributions to the slowing of disease progression compared to control that we have seen in boys in the clinic.

We performed experiments in mdx mice, a mouse model of DMD, showing that once daily dosing does not produce the same level of efficacy as sustained dosing of edasalonexent. We believe that time over threshold is the primary driver of efficacy for edasa, and that these results support dosing edasa three times per day going forward as is planned for our Phase 3 trial.

Turning to our cystic fibrosis program, as Jill mentioned, we have made additional progress with our CAT-5571 program. CAT-5571 is designed to restore host defense by activating autophagy, a mechanism for cellular recycling and digesting pathogens. By restoring autophagy, which is depressed in cystic fibrosis, host defense is re-established to enhance the clearance of pathogens. CAT-5571 has the potential to augment the efficacy of antibiotics and could also be used with other CF therapies, including CFTR targeted agents. We are continuing IND enabling activities for CAT-5571, and we expect to initiate a Phase 1 trial in the second half of 2018 with top-line results in 2019 based on our current operating plan.

I'll now turn the call back over to Jill for a review of our financials.

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [6]

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Thank you, Joanne and Andy. Turning to our financials. Our fourth quarter and full year 2017 press release and 10-K provide the details, so I will provide a brief summary.

As of December 31, 2017, we had $16.4 million of cash and cash equivalents. Following December 31, 2017, we raised an additional $8.3 million in net proceeds under an at-the-market offering program. We expect to be able to fund operations through September 2018. To advance edasalonexent in the Phase 3 trial, we expect to obtain additional funds through equity or debt financings or through partnering or licensing transactions.

In the fourth quarter of 2017, our net cash used in operating activities was $5.6 million and $26.8 million for the full year 2017. We also recognized collaboration revenue from an option agreement with an unaffiliated partner in the fourth quarter of $0.3 million, and for the full year of 2017, it was $0.5 million.

Our R&D expense was $4 million in Q4 2017 compared to $6.3 million in Q4 2016, a decrease of $2.3 million and $18.7 million for the full year of 2017 compared to $25.5 million for the full year of 2016. The decrease in research and development expenses was primarily attributable to the completion of certain clinical activities.

Our G&A expense was $1.7 million in the fourth quarter of 2017 and $8.9 million for the full year 2017. Our operating loss was $5.5 million in Q4 2017, a decrease of $3.2 million versus Q4 2016. Our net loss was $5.5 million or $0.24 per share in Q4, a decrease by $3.3 million compared to our net loss in Q4 2016.

Net loss for the full year 2017 was $27.4 million or $1.26 per share. For the fourth quarter, we had weighted average common shares outstanding of $23.2 million. Additional financial information is available in our 10-K, which we filed with the SEC earlier today.

Building on our accomplishments in 2017, 2018 will be an important year at Catabasis. We plan to bring our first program into Phase 3 development in Duchenne muscular dystrophy. While this is an incredibly important milestone for all of us at Catabasis, it is especially rewarding as it brings us one step closer to making a difference in the lives of boys living with Duchenne. Bringing CAT-5571 to the clinic for the potential treatment of cystic fibrosis also shows the strength of our development pipeline at Catabasis and the opportunities it represents to impact additional rare diseases.

We enter the Phase 3 trial for edasalonexent with excitement. We plan to initiate in the first half of 2018 and expect top-line results in 2020 dependent on raising capital. After more than a year of treatment, we continue to see a significant slowing of disease progression and get very positive feedback from clinicians who see the potential for edasalonexent to change the course of the disease. We continue to be moved by the candid testimony of family, stories of their challenges, as well as their boys lose the ability to stand and walk while their peers run and jump on the playground. What continues to motivate us are the stories from families of boys being treated with edasalonexent. We're working to progress edasalonexent as quickly as we can. And thank you, we are so grateful to all the boys and their families participating in our edasalonexent study as well as to the dedicated investigators and their staff that make our trial possible.

With that, I'll ask the operator to open up the call for your questions. Bruce, can you please repeat the instructions and poll for questions? Thank you.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from the line of Hartaj Singh from Oppenheimer.

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Emma Nealon, Oppenheimer - Analyst [2]

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This is Emma on for Hartaj. Based on the early signals you've seen as reduced tachycardia in the boys in MoveDMD and also just given how central the cardiac health is to the trajectory of the late-stage progression, just wondering specifically what your plans are to measure the effect on cardiomyopathies in Phase 3 and how important that is as an endpoint.

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [3]

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Yes, Joanne?

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [4]

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Thanks. I think that it is going to be very interesting to see in Phase 3. What we saw was a decrease in heart rate from the resting tachycardia to basically to age normative values for these boys and you know that's the first sign. What is that followed by is the development of fibrosis a few years later and the development of cardiomyopathy. And, in fact, the boys who have the highest resting heart rates are more likely to develop cardiomyopathy later. So we're going to be measuring with an ambulatory a relatively easy to deal with monitor. We're going to be measuring resting heart rate to monitor the boys over 24 hours as well as other indicators such as heart rate variability; things that are known to be altered in Duchenne.

So that's the main measure that we're going to be looking at in the boys at this age. As you know, their cardiac function at this point in terms of ejection fraction is still maintained. The heart rate is the first signal. So we'll be focusing on that.

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Emma Nealon, Oppenheimer - Analyst [5]

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But that heart rate will be a secondary endpoint?

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [6]

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It will be a safety assessment and we will monitor that, yes.

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Emma Nealon, Oppenheimer - Analyst [7]

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Great. And then just one follow-up. Looking ahead to any future plans to study edasa in boys outside of that 4 to 7 population, both younger and older, and also any potential benefits in Becker's? How are you prioritizing those plans internally?

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [8]

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Yes, that's a great question. We do intend to study it edasa in the future in boys at older age range, and we do believe that edasa has potential for boys from the time of diagnosis throughout their lives based on what we have seen so far in our MoveDMD trial but importantly also what we've seen in pre-clinical models of Duchenne.

The Becker's question is definitely an interesting one and one we've been giving a lot of thought to especially based on the results of the MoveDMD trial and results from preclinical studies. So, as you know, Becker's patients expressed some low levels of dystrophin. It's known that NF-kappaB suppresses dystrophin production. And so, our hypothesis is edasalonexent, which inhibits NF-kappaB could potentially enhance dystrophin expression in Becker's patients, and that's something we certainly are eager to pursue in the future.

The results from the MoveDMD trial on heart rate are particularly intriguing as well because we know Becker's patients also suffer from cardiomyopathy as well. So it's something we are certainly eager to explore.

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Operator [9]

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Our next question comes from the line of Ted Tenthoff from Piper Jaffray.

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Ted Tenthoff, Piper Jaffray - Analyst [10]

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I wonder if I'd get a sense for how you're thinking about powering the study and I think you said 120 boys. So what kind of delta do you need to see on the primary [North Fall] to achieve significance?

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [11]

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Yes, Joanne?

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [12]

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Yes. So we are planning to enroll about 125 boys, and that's based on what we saw. We saw an annualized rate of decline of four points compared to stabilization in the boys as well as the standard deviation of the variability in the measures that we saw. So that's where we came to what we think is a robustly powered study.

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Ted Tenthoff, Piper Jaffray - Analyst [13]

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Great. So it will be a 12-month endpoint on North Fall?

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [14]

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Yes, North Star, yes.

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Ted Tenthoff, Piper Jaffray - Analyst [15]

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Okay, great. And then similar question, I was hopeful to hear the thought process behind the cardio. What about the bone? What are you looking to see there? Thanks.

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [16]

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Yes, so this will -- on the cardiac monitoring, is that what you're asking, Ted? Bone, I'm sorry. I'll let Joanne answer that.

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [17]

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So bone health is important in these boys. Just about half of them have fractures by their early teens, and boys even as young in the trial can have fractures. So we will be looking with measures like DXA to look at bone density that changes early in boys that are treated with steroids and also to look at things like lateral spine film. These are things that the recent care guidelines that have just been published in Lancet in the last month or so are saying that really boys should be monitored this young. Even at diagnosis they should be getting these because of the importance of bone health. So we think that's going to be a very important differentiator of edasa versus steroids standard of care.

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Operator [18]

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Our next question comes from the line of Liana Moussatos from Wedbush.

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Liana Moussatos, Wedbush Securities - Analyst [19]

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For CAT-5571, what do you imagine primary endpoint would be in Phase 2? Would it be some anti-microbial endpoint or FEV1. And my second question is when do you expect combo results with eteplirsen this year?

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [20]

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Yes. I'll let Andy take the 5571.

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Andrew Nichols, Catabasis Pharmaceuticals, Inc. - Chief Scientific Officer [21]

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Yes, so for CAT-5571, we will be looking primarily at functional endpoints measures such FEV1 as opposed to looking at microbial content and density in the lungs. They will look at those as secondary measures. They certainly wouldn't be a primary drivers of efficacy that we're looking at.

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [22]

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And for the eteplirsen combo data, so as you know, we have two boys in the MoveDMD open label extension that are co-dosing now with edasalonexent and eteplirsen. And I will let Joanne give an update on where we are there and what the plans are.

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [23]

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So we'll continue to follow them for safety. We have two boys and we're anticipating also including boys that are on a stable eteplirsen dose in the Phase 3 study as well. And that's important with the recent news. There's going to be more exon skipping therapies that are further along in development. And, in fact, in the trial, about a third of the boys had mutations that are amenable to either currently marketed mutations specific therapies or those that are in clinical development right now. So it is going to be a very important area, and this is a disease that's going to likely require more than one therapy. So we are thinking that that combination therapy with edasa is going to be important. We see this as foundational, as monotherapy, and combination therapy.

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Liana Moussatos, Wedbush Securities - Analyst [24]

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And when would we see any kind of data on the combination with the two boys?

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [25]

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So we'll continue to provide safety updates with those.

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [26]

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Yes, so throughout this year, we'll continue to provide updates. Those will largely be focused on safety. And I think, perhaps, where you are going with this as well -- and mechanistically the combination with exon skipping and dystrophin-targeted therapy makes a lot of sense for edasalonexent because of the mechanism and the ability to enhance dystrophin expression. And so, that's something we are certainly very eager to take a look at and as we think about that for diseases like Becker's or these combination approaches with exon skipping. That's very exciting to us.

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Liana Moussatos, Wedbush Securities - Analyst [27]

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During the safety updates, will you provide dystrophin changes?

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [28]

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Not from the MoveDMD open label extension. We have not -- we do not do biopsies in the open label extension portion. So there will be no measures of dystrophin levels directly.

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Liana Moussatos, Wedbush Securities - Analyst [29]

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Phase 3 then?

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [30]

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That is something that we are considering of how best to do that as part of the Phase 3 because as you know the protocol that we've talked about publicly has us enrolling, allowing boys who are on stable doses of eteplirsen to enroll in the trial.

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Operator [31]

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Our next question comes from the line of Joel Beatty from Citi.

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Joel Beatty, Citi - Analyst [32]

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I guess, the first one, for the enrolment criteria, I think you just mentioned how the use of eteplirsen could be used in Phase 3. Given as a global study, do you anticipate there could also be use of Translarna from PTC?

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [33]

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Joanne?

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Joanne Donovan, Catabasis Pharmaceuticals, Inc. - Chief Medical Officer [34]

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Thanks, Joel. That's a great point because ultimately we think that edasalonexent could be used in combination, for a variety of dystrophin-targeted therapies not just exon skipping, as well as non-dystrophin-targeted therapies. Ataluren has very specific dosing, and we would not -- we would want to do a drug interaction study before that. So we won't be including -- we haven't done that to-date, and we won't be including boys that are on Translarna in Phase 3 study.

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Joel Beatty, Citi - Analyst [35]

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And then one other question. Just the data from the MoveDMD trial as a whole, you've been putting out these updates from the extension study. Do you anticipate there could be additional extension study updates before initiating the Phase 3 trial?

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [36]

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So we will continue to report updates throughout 2018 at scientific conferences. Those updates will be largely from this point on focused on safety data as the boys are now several years into this trial. And so expect to hear updates at some of the relevant upcoming scientific conferences.

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Operator [37]

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Thank you. At this time, I would like to turn the call back over to Jill.

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Jill Milne, Catabasis Pharmaceuticals, Inc. - Co-Founder and CEO [38]

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Thank you, Bruce. We are excited about the strong and consistent edasalonexent data after more than a year of treatment and are looking forward to initiating the Phase 3 trial as we move edasalonexent towards registration. We are dedicated to improving the lives of patients affected by rare disease and their families. We believe that edasalonexent represents an important and differentiated opportunity to treat Duchenne.

Thank you all for joining our call today and for your continued support. We look forward to speaking with you again soon and keeping you updated on our progress. Have a good evening. Andrea?

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Andrea Matthews, Catabasis Pharmaceuticals, Inc. - Vice President, Corporate Affairs [39]

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That concludes today's call. A webcast replay will be available for 90 days via the Investor Relations page on our website at www.catabasis.com. Thank you.

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Operator [40]

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