Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB) Q1 2019 Earnings Conference Call May 14, 2019 8:30 AM ET
Andrea Matthews - Vice President-Corporate Affairs
Jill Milne - Chief Executive Officer
Joanne Donovan - Chief Medical Officer
Andrew Nichols - Chief Scientific Officer
Noah Clauser - Vice President of Finance
Conference Call Participants
Liana Moussatos - Wedbush Securities, Inc.
Joel Beatty - Citigroup
Hartaj Singh - Oppenheimer & Co., Inc.
Good day, ladies and gentlemen, and welcome to the Q1 Catabasis Pharmaceuticals Incorporated Earnings Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As reminder, this call may be recorded.
I would now like to introduce your host for today's conference Andrea Matthews. Please go ahead.
Thank you, Chris. Welcome to today's Catabasis Pharmaceuticals conference call, where we'll provide a corporate update and review our first quarter 2019 financial results. With me today are: Jill Milne, Chief Executive Officer; Joanne Donovan, Chief Medical Officer; Andrew Nichols, Chief Scientific Officer; and Noah Clauser, Vice President of Finance.
We issued a press release this morning, summarizing our corporate update and our Q1 2019 financial results, which we will reference on today's call and 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 those discussed in our most recent Quarterly Report on Form 10-Q, which we filed this morning 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, who will provide our corporate update. Joanne will provide an update on our Phase III PolarisDMD trial for edasalonexent and recent data presentation. Noah will follow with the financial update, and Jill will wrap things up. Jill?
Thank you, Andrea. Good morning, everyone, and thank you for joining us today for our first quarter 2019 financial results and an update on progress. Our lead program edasalonexent has broad potential for benefits and treating patients affected by Duchenne muscular dystrophy.
Our vision is for edasalonexent to be a therapy that could benefit all patients regardless of their mutation type and throughout their lifetime. We believe edasalonexent represents a significant commercial opportunity and we are executing on our strategy to bring edasalonexent to patients with our Phase III trial and initial commercial work underway.
We are making strong progress with enrollment in our Phase III PolarisDMD trial and the trial is now enrolling patients at 28 sites in seven countries. There has been significant interest from families and enthusiasm from investigators globally. Including the patients already enrolled, we believe that approximately 90% of the patients for the trial have been identified.
We anticipate all patients will be identified in the coming weeks as there are many patients who have expressed interest and participating in our Phase III trial, past preliminary screening criteria and are scheduled or waiting to be scheduled at a participating sites. We expect all participating sites to be open for enrollment within a month, which will further support scheduling the remaining families.
Based on our current projections, we expect to have topline results from the study in the second half of 2020. The clinical data along with our ongoing efforts in CMC and non-clinical are intended to support an application for commercial registration of edasalonexent in early 2021.
As we've done in the past, we will continue to provide high level updates on the trial and plan to announce when we have completed enrollment. We very much appreciate the interest and support from the Duchenne community. Our team is dedicated and focused on progressing this program so that we can get edasalonexent to all patients who could benefit.
Additionally, we are very pleased to have the opportunity to provide boys with edasalonexent in a new long-term open-label extension, the GalaxyDMD trial. Boys from the MoveDMD trial and their eligible siblings are actively enrolling and will be followed by boys participating in the Polaris trial and their brothers. Importantly, GalaxyDMD allows us to collect data as boys get older to support registration filings.
I am also pleased to share that we recently presented long-term MRI and growth data from our MoveDMD trial that reinforced the sustained slowing of disease progression and preservation of muscle function, same with edasalonexent compared to control. These results support the potential of edasalonexent as a promising new therapy for the treatment of Duchenne and also add to the growing body of data supporting the use of MRI as a non-effort-based marker for disease progression in Duchenne.
We hear frequently from physicians and families that there is a compelling need and desire for a therapy with the characteristics of the edasalonexent. This community is eager for a therapy with broad benefits on skeletal and cardiac muscle that can preserve muscle function and allow their sons to grow.
We are glad to see boys on edasalonexent grow at a similar rate as boys that do not have Duchenne. We know this is extremely important to the boys and their families. We hear that families also appreciate that edasalonexent is an oral therapy presented in gel capsules, enabling ease of use for administration.
We are driven to provide the Duchenne community with a treatment that is well tolerated and could have a meaningful impact on its own and in combination with other therapies. We are preparing for the evolving treatment landscape in Duchenne, which we believe will include combination therapy. To this end, we are collecting additional clinical data on the co-administration of exon skipping therapy with edasalonexent in our Phase III trial.
Next, Joanne will provide a clinical update including more information about our PolarisDMD and GalaxyDMD trials as well as the recent MoveDMD results that continue to build our confidence in the potential of edasalonexent to change the treatment paradigm for Duchenne. Joanne?
Thank you, Jill, and good morning, everyone. I'd like to start with an update on our edasalonexent Phase III PolarisDMD trial in Duchenne. We are making very good progress in the trial with enrollment ongoing in seven countries and 28 clinical sites opened for enrollment. We are pleased to be receiving a substantial amount of inbound interest from families.
As Jill mentioned, the vast majority of the patients have been identified and we're looking to wrap up patient identification soon and complete enrollment. We are working with the sites so that the queue of identify patients can be scheduled for screening visits.
We have a wonderful group of committed investigators involved in the PolarisDMD trial and we appreciate all of the enrollment progress to-date. This is an exciting time in the field of pediatric neuromuscular disease and many of the PolarisDMD sites are involved in clinical trials, not just in Duchenne, but in other areas including SMA.
We recognize that there is significant demand on the clinical trial sites and this has impacted the speed of site activation and patient scheduling. Thanks to all of the sites staff for everything they are doing to make this trial successful.
A quick reminder of the trial design, PolarisDMD is a randomized double-blind, placebo-controlled trial in which we are planning to enroll about 125 boys. As we did in our Phase II MoveDMD trial, the Phase III trial is enrolling boys aged four to seven up to their eighth birthday.
The Phase III study includes boys regardless of mutation type who have not taken steroids for the past six months. The randomization is two to one such that for every two boys that receive 100 milligrams per kilogram of edasa, one boy received placebo.
The primary efficacy endpoint is changed in the North Star Ambulatory Assessment after 12 months of treatment with the edasa compared to placebo. North Star was chosen as the primary endpoint because its age appropriate for the boys in this trial as well as supported by FDA. Key secondary endpoints include the time function tests, time to stand, 4-stair climb and 10-meter walk/run.
We recently launched a new open-label extension trial, called GalaxyDMD. This trial is now enrolling boys from the MoveDMD open-label extension. We were urged by families with sons in the MoveDMD trials who also provide edasa for their other sons affected by Duchenne and therefore we are also enrolling eligible brothers.
Our primary objective with GalaxyDMD is to come collect long-term safety data and we're also monitoring assessments of muscle function as well as bone health. Based on the excellent tolerability observed to-date with edasa, GalaxyDMD has a streamlined visit schedule with visits every six months. When boys in Phase III PolarisDMD trial complete the 12-month placebo control study, they will have the opportunity to participate in GalaxyDMD and receive edasa.
We recognize the importance of combination therapy in the evolving Duchenne treatment landscape. Therefore, co-administration with EXONDYS 51 is anticipated in the GalaxyDMD trial and we intend to allow co-administration of additional approved exon skipping therapies going forward.
As Jill shared, we continue to deepen our understanding of edasalonexent as we evaluate further clinical data. Last week, Dr. Richard Finkel, a Principal Investigator for both the Phase II MoveDMD and Phase III PolarisDMD trial gave a podium presentation on edasa at the American Academy of Neurology Annual Meeting in Philadelphia.
Dr. Finkel presented MRI data from the MoveDMD open-label extension study that reinforced the sustained long-term slowing of Duchenne disease progression seeing across assessments of muscle function. Improvements in lower leg composite MRI T2 were observed through 72 weeks of edasa compared to the off-treatment control period in the Phase II MoveDMD trial and open-label extension.
The lower leg composite MRI T2 is highly correlated with functional abilities in boys with DMD in the ImagingDMD natural history database as presented at the World Muscle Society Congress last year. These improvements in MRI T2 are consistent with the slow disease progression and preserved muscle functions seen in all assessments of muscle function through 72 weeks of edasa treatment compared to the off-treatment control period.
Significant decreases in muscle enzymes, including CK, through 72 weeks were also seen supporting the durability of edasa treatment effects. We also observed significant decreases in heart rate towards age normative value, supporting the potential cardiac benefits of edasa. In the MoveDMD, we also saw that boys with Duchenne treated with edasalonexent on average grew in both height and weight in line with the growth of boys that do not have Duchenne in the same age range.
We frequently hear from families how important growth is to boys with Duchenne and the opportunity to be more like their friends. The vertical growth is an indication of healthy bone growth in these boys. We know that when boys are treated with steroids, they're frequently side effects related to growth such as excess weight gain, curtailed vertical growth, and substantially increased BMI. We've collaborated with Parent Project Muscular Dystrophy Duchenne registry team to increase our understanding of steroid use in the Duchenne population.
I am happy to share that last week, a joint manuscript was published in the journal BMC Neurology. We found the 29% of ambulatory boys and 51% of the non-ambulatory Duchenne population were not on steroids.
The analysis of the Duchenne registry data reinforces the need for safe and effective treatment options for those affected by Duchenne. At Catabasis, we believe that edasalonexent is a promising treatment approach that has the potential to deliver on these objectives.
I'll now pass the call over to Noah Clauser, our Vice President of Finance to share our financial update. Noah?
Thanks, Joanne, and good morning, everyone. Turning to our financials, our first quarter 2019 press release and 10-Q provide the details. So I will provide a brief summary. As of March 31, 2019, we had $51.7 million of cash, cash equivalents and short-term investments.
Based on our current operating plan, we expect that we have sufficient capital to fund operations through topline Phase III results and into the fourth quarter of 2020. In the first quarter of 2019, our net cash used in operating activities was $6.6 million.
Our R&D expense was $4.2 million in Q1 2019 compared to $5.2 million in Q1 2018. Our G&A expense was $2.1 million in the first quarter of 2019 compared to $2.4 million in the first quarter of 2018. Our operating loss was $6.3 million in Q1 2019, a decrease of $1.3 million versus Q1 2018.
Our net loss was $6 million or $0.62 per share in Q1. For the first quarter, we had weighted-average common shares outstanding of $9.7 million. Additional financial information is available on our 10-Q, which we filed with the SEC earlier today.
I will now pass the call back over to Jill.
Thank you, Noah. Our vision is for edasa to be a therapy that could benefit everyone affected by Duchenne. We very much appreciate all of the enthusiasm we were receiving from the Duchenne community for edasalonexent and our Phase III PolarisDMD trial. The data from our three clinical trials in Duchenne as well as our CMC and non-clinical efforts are intended together to support an application for the commercial registration of the edasalonexent in early 2021.
The Duchenne community is eager for a therapy that can preserve muscle function and provide those affected with benefits on skeletal and cardiac muscle, while allowing them to grow. To this end, we are aggressively and diligently executing on our strategy to bring edasalonexent to all of those affected by Duchenne.
With that, I'll ask the operator to open up the call for your questions. Chris, can you please repeat the instructions and poll for questions? Thank you.
Thank you. [Operator Instructions] And our first question comes from the line of Liana Moussatos with Wedbush. Your line is now open.
Thank you for taking my questions and congratulations on your progress. What percent of the estimated total DMD boys qualified for Polaris? And what seven countries are enrolling? And you mentioned 90% of the DMD boys have been identified. Can you describe the process of identification?
Sure. Yes. So this is Jill. Liana, hi. I'll start with that and hand it quickly over to Joanne. There are a lot of questions in that. So you asked which seven countries we are actively enrolling in. So that would be the United States, Canada, Sweden, Ireland, the UK, Germany and Australia. And we have 28 sites open across those seven countries.
And all of the boys in the age range of four to seven years old up to their eighth birthday are eligible for the trial as long as they've been either not on steroids previously or off steroids for at least six months. So 100% of the patient population in that age range without steroids is eligible for the trial.
Okay. And the process of identifying DMD boys?
Yes. So we have - the sites are the two main sources. One is that the sites are of course identifying patients that are already in their practice. The sites are also getting inbound interests. And in addition to the patients that are already enrolled, there's also many patients who have expressed interest and reached out to our email where they can receive more information and have the characteristics of boys who would be in the study that is in the age range and not on steroids and they're scheduled or waiting to be scheduled at site.
Thank you. And our next question comes from the line of Joel Beatty with Citi. Your line is now open.
Thanks for taking the questions. First one is on the timelines for the PolarisDMD trial. Last I remember the trial was reading out in Q2 of next year. It looks like that's not going to be second half of next year. Can you provide any context for the change in timing?
Yes. Hi, Joel. Thanks for the question. This is Jill. So while we've identified almost all of the patients for the trial and expect to complete identification for the patients to complete enrollment for the trial in the next few weeks, getting the patients scheduled at sites and getting sites activated has been a bottleneck and getting the enrollment complete. And so we expect to complete enrollment soon and then we will be able to refine the timeline to topline data. So more to come.
Got it. Makes sense. And then with the extension study up and running now, could you provide any thoughts on the interest in the boys? And it sounds like perhaps in some cases the brothers are joining the expansion study?
Yes. So that's for the GalaxyDMD study, so one reason that we opened the GalaxyDMD open-label extension study was because of interest from families participating in the MoveDMD open-label extension study to include siblings. So brothers and boys who were enrolled in the MoveDMD and so indeed we were able to do that by opening the Galaxy study, and so that study will now be a unified open-label extension study that incorporates the boys from MoveDMD in any siblings that meet eligibility criteria and have an interest in enrolling as well as the PolarisDMD boys who go into the open-label extension and their siblings.
Great. Thank you.
Thank you. And our next question comes from the line of Hartaj Singh with Oppenheimer. Your line is now open.
Great. Thank you, all. Thanks for the question. Jill, Joanne, just got a couple of questions on Polaris. One is, I know you'd mentioned about 28 sites are now open. I believe you are going to get close to 40 sites worldwide. Can you just give a breakdown of the U.S. versus the ex-U.S. sites and then what percent of the ex-U.S. sites are still need to be opened and starting to enroll boys? And then I've just got a couple of follow-ups.
Yes, for sure. So I'll let Joanne open - address the specifics about where the remaining sites to be and your right that we intend to open approximately 40 sites and we should have completed site openings within the next month of all of the participating sites.
And certainly with the activity and the pediatric neuromuscular field, there has been a lot of activity at sites as you might imagine, not only from Duchenne, but also from other pediatric neurology clinical trials. And so we are working aggressively and as quickly as possible to get those remaining sites open and that should enable us to complete enrollment and get the final family scheduled to do so.
Yes. So Hartaj to your question, we actually keep it very much updated on our website and we have 19 sites in the U.S. that are open, three in the UK, two in Germany, one in Ireland, one in Sweden and one in Australia right now.
Thanks, Joanne. So I guess what I was trying to get out - Jill, you kind of answered the question I think a little bit, which is I would imagine that most of your U.S. sites are pretty much up and running, right? And is it the actual sites where you've got, still some of to be open and the patients to be initiated or is it like an equal breakdown in terms of open sites initiation patients between the U.S. and ex-U.S.?
Yes. I'll start and then let Joanne finish, and she forgot to mention the one.
We are enrolling in Canada as well. In terms of - if you back on our call in March, we had indicated our initial press was our North America. To get the North American sites as many of them up and actively enrolling as quickly as possible. That was the first wave.
The second wave was the European countries. And so that's exactly the staging that has taken place. With that said I will say it's taken us longer to get some of the sites open and scheduling patients, and scheduling the queue of patients. And that's what we're working through now, and getting the remaining sites open in the next month should address the final push on the enrollment.
And I think it's safe to say, just like we had observed in the MoveDMD trial and enrollment and patient identification in that trial, we're seeing great enthusiasm from families at those participating sites, from families from referring physicians who are not participating in the trial and from families that are identified through our efforts with advocacy groups in participating in the trial. So it's not about interest from patients and families in the trial. It's really about getting the sites up and activated in scheduling the patients as quickly as possible.
And your perception is right, the U.S. sites were the first to open and most of those sites are open. And the sites in - beyond the U.S. have taken longer to open. As you remember those - this was the first time we went to regulatory agencies in many of those countries. So we expected that they would take a bit longer to get up and running.
Great. That's fantastic. Yes. I would imagine - and that makes sense. Also another question is that, I know previously you had mentioned that you're also starting to do some of the initial activities to look at the Duchenne's population. The Board is in the U.S., ex-U.S. looking at commercial, potential launch scenarios, reimbursement, et cetera. Where are you right now with those plans, Jill? How has that sort of progressed over the last few months?
Yes. So we are actively working on our - doing some initial commercial work, really trying to determine where edasalonexent fits in the therapeutic landscape. And I'm certainly pleased to say there - from our assessment of the marketplace right now, there is a great need for an agent like edasalonexent to treat all boys affected by Duchenne regardless of the mutation and throughout their lifetime.
And I think importantly, what we've been able to show both pre-clinically and clinically is the potential of edasalonexent to work in combination with other therapeutic approaches. And I think, as we move forward in Duchenne that is going to be the future is combination therapy and so to have a safe and effective agent that can work in combination is going to be critically important. And so we will be engaging in more specifics with payers and market access strategy as 2019 progresses. And so more to come on that.
Great. Thank you, Jill. And just last question. Have you had any updates on meetings with the FDA, I know FDA is still interested in potentially speeding therapies along on accelerated basis. Just any updates there in terms of your interactions with regulators? And again, thank you for all the questions.
Yes. No problem, Hartaj. And as we said before, we don't share the details of the interactions with the regulators. But that said, as we're all aware, the FDA has an interest in MRI and neuromuscular disease. And that's certainly expressing their guidance and in their public conversations.
It's our understanding from the FDA that they are continuing to seek additional expert advice on MRI and I think that's important and we still remain committed to MRI becoming a biomarker in the future towards supporting accelerated approval in diseases like Duchenne Muscular Dystrophy.
And certainly we believe our dataset contributes greatly to that. And as you can imagine as time progresses, as we wait for the regulators to develop their approach and strategy to MRI as an endpoint, the potential of accelerated approval for edasalonexent becomes less relevant.
We certainly will be completing enrollment in the near future on the PolarisDMD trial and making our way to data from this trial with a clinical endpoint, the North Star Ambulatory Assessment. But the importance of the MRI data for us is certainly not lost because that data we believe is supportive of the potential of edasalonexent and the potential benefit of edasalonexent on muscle function. And so we continue to work with regulators and continue to develop our data in MRI.
Yes. That makes a lot of sense. Again, thanks a lot, Jill. Joanne. I'll look forward to more updates.
Thank you. We do have a follow-up question from the line of Liana Moussatos with Wedbush. Your line is now open.
Well, the GalaxyDMD trial enroll female siblings or just male?
Hi Liana. That's a great question. At this time, it will only enroll male siblings of the boys in the trial. In my understanding, at least at the time, at this time, we only have siblings that are male, who are eligible for the trial from the MoveDMD patients. We obviously as we complete enrollment on Polaris, we'll know if there are any female DMD patients.
Thank you. And that does conclude today's question-and-answer session. I'd now like to turn the call back to Jill Milne for any further remarks.
Thank you, Chris. Thank you all for joining our call this morning and for your continued support of Catabasis. We will keep you updated as we execute on our Phase III PolarisDMD trial for edasalonexent and share other areas of progress. We look forward to speaking with you again soon. Andrea?
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.
Ladies and gentlemen, thank you for participating in today's conference. This does concludes today's program, and you may all disconnect. Everyone have a wonderful day.