Q4 2019 Earnings Call
Isn't station they'll be a question answer session asking question during the session you'll need to press star one on your telephone. Please be advised that today's conference is being recorded if you acquire any further assistance. Please press star zero I would now like to hand, the conference over to your Speaker today, Andrea Matthews Vice President Corporate Affairs. Please go ahead.
Thank you Josh welcome to today's Catabasis Pharmaceuticals Conference call. There, we will provide a corporate update and review our fourth quarter and full year 2019 financial results.
With me today, our Jo Mill, Chief Executive Officer, Joanne Donovan, Chief Medical Officer, Andrew Community, Chief Commercial Officer, Andrew Nicola Chief Scientific Officer, and know what costs are Vice President finance.
We issued a press release this morning, summarizing our corporate update in our Q4 and full year 2019 financial result, which will reference on today's call is available on our website.
I would like to note that during today's call will make statements related to our business based on current and future expectations that maybe considered forward looking statements under federal Securities laws.
Actual results may differ materially from the syndicated by these statements as a result for a variety of risks and uncertainties, including those discussed in our most recent annual before.
Annual report on form 10-K, which we filed this morning with the FCC. It 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 Joe So provider corporate update Joanne will provide an update our two ongoing clinical trials with either so nexsan. The phase three Polaris DMD trial, an open label extension Galaxy DMD trial.
Andrew Overviews potential market opportunity for you to saw next insertion and Andy will share an update on our preclinical work.
No we will provide financial summary, and we will open for question Joel.
Thank you Andrea good morning, everyone and thank you for joining us for today's call. We made excellent progress it kind of basis with our either sold Nexsan program. In 2019, we believe that our product candidate you just saw onex and can be a foundational therapy for all patients affected by Duchenne muscular dystrophy, we're looking forward to the results from the phase three.
Polaris the M.D. trials this year.
The phase three Polaris DMD trial in Duchenne is fully enrolled and we are preparing for topline phase three results in the fourth quarter of this year and subsequent anda filing in 2021.
We have commercialization and supply chain preparations are underway and recently strengthened our financial position based on our current operating plan, we expect to be able to fund operations through a potential anda filing and into Q3 2021.
For those that are less familiar with catabasis, we were taking a different approach to treating duchenne than other development programs. You. Just saw an exit was designed to inhibit Nf Kappa B a mechanism with the potential to benefit all patients with Duchenne and therefore is not limited to patients with specific mutation.
It is important to remember that in Duchenne the absence of the dystrophin protein is necessary, but not sufficient to drive disease progression typically young boys with Duchenne are not symptomatic in their first a couple of years, however, without dystrophin mechanical stress from every day Act.
Liberty's like running chronically activates Nf Kappa B, which leads to progressive deterioration of skeletal muscle importantly, the chronic activation of Nf Kappa B is also a critical component of cardiac disease and to factor in bone health in Duchenne.
By inhibiting Nf Kappa B. We believe you just going accent can have broad therapeutic effects and benefit all patients would duchenne regardless of mutation both as a monotherapy as well as in combination with dystrophin targeted therapies.
Based on our clinical data and the mechanism of action you just saw onex and has the potential to benefit skeletal muscle, including the diaphragm cardiac function and also bone health.
We believe that a recent analysis of the baseline characteristics of the patients enrolled in the phase three trials supports the assumptions on which the phase three trial was powered.
Analysis compared the baseline characteristics of the patients enrolled in our phase three trial to the patients enrolled in our previous phase to move DMD trial and sound overall similar characteristics and the patient populations in the two trials Joanne will talk more about this shortly and will also.
So be presenting the baseline characteristics at the upcoming 2020, M.D.A. clinical and scientific conference later this month.
We're also working in multiple areas to maximize the potential of either solid nexsan, both in duchenne as well as an additional neuromuscular diseases. We're excited to have entered into a partnership with the leading European patient advocacy organization Duchenne UK to explore it you just saw nexsan.
Literary patients with Duchenne, we see this as important for the future. Both in terms of the needs of this patient population as well as to generate data in older Boys and men to improve future access we see this all nexsan as an attractive commercial opportunity in Duchenne and Andrew well lab.
Right on that.
We've also made great progress in the preclinical research on the potential benefits of EDA solid accident in Duchenne and additional neuromuscular diseases as Andy will review today.
Before we move to our clinical and other updates I want to note that going forward, we're going to reserve conference calls for clinical data and significant events and are not planning to host regular quarterly earnings calls, we will continue our frequent communication practices and remain available for questions with that I'll turn.
On the call over to Joanne to provide an update on our ongoing clinical trials and upcoming plans Joanne.
Thank you Jill and good morning, everyone.
Our fully enrolled phase three Polaris DMD trial would be to sell an accent is progressing well.
In less than a year, we enrolled 131 boys affected by do Shane in eight countries across the world due to strong interest from physicians and Dushane families as well as support from patient advocacy organizations.
Thank you to everyone who's making this global phase three trial possible, including all participating families and the clinical trial sites staff.
As you likely recall the phase three Polaris DMD trial is a randomized double blind placebo controlled trial with two to one randomization.
The primary efficacy endpoint is the change in the North Star ambulatory assessments score after 12 months of treatment with either it's all the nexsan compared to placebo.
North Star was chosen as the primary endpoint with support from regulatory authorities.
Key secondary endpoints include the time function test 10 meter walk run time to stand and for stair climb and additional assessments include growth cardiac and bone measures as well as patient reported outcome.
We expect topline results from the phase three Polaris DMD trial in the fourth quarter of this year and the trial is anticipated to support an anda filing next year.
As Joe mentioned following the completion of enrollment we conducted an analysis of the baseline age and functional test performance of the patients enrolled in the phase three trial as compared to the patients enrolled in the previous phase to move DMD trial and found no significant differences in the baseline characteristics of the pace.
Populations in the two two trials.
We believe that these findings support the assumptions on which we powered the phase three trial.
The patient population in both trials is also similar to publish natural history study the boys in this age range that are not on steroids.
To summarize the patient populations, both trials enrolled boys ages four to seven up to their eighth birthday with any mutation type who had not been on steroids for the previous six months.
Phase three trial enrolled the 131 boys at 37 sites in the United States, Canada, Europe, Israel, and Australia, and 98% had never been on steroids.
All 31 boys that enrolled in the phase two trial were enrolled in the United States and had never been on steroids.
We will be sharing more information on the phase three baseline characteristics as well as the reproducibility of North star in this patient population at upcoming scientific conferences.
Our open label extension trial Galaxy, DMD and bodies are ongoing commitment to the Duchenne community.
Our primary objective with this trial is to collect long term safety data and we're also monitoring assessments of muscle function as well as bone health.
Clinic visits and assessments are conducted every six months for this trial.
Boy to have completed treatment in the phase three Belarus, DMD trial have enrolled in the Galaxy DMD trial, and they're eligible siblings up to age 12 have the option to enroll as well we've seen an excellent rate of enrollment in galaxy DMD from the phase three trial in all boys are receiving open label extent.
You just saw an accident.
Boys in the Galaxy DMD trial that are amenable are also able to initiate co administration of approved exon skipping therapy.
Earlier this year, we were thrilled to announce our partnership with the patient advocacy organization Dushane UK to evaluate eat a solid accident in a phase two trial in non ambulatory patients with two Shane.
We recognize the need for a well tolerated treatment for all patient populations that has the potential to slow disease progression and to preserve muscle function by benefiting both skeletal muscle as well as cardiac function.
Shane UK granted us over $600000 in funding to support patient and clinical trial site costs.
We're incredibly fortunate to have the opportunity to partner with Duchenne UK for this important work and appreciate the deep commitment as we work together to bring treatment options to all patients.
This phase two trial is planned to assess safety pharmacokinetics and exploratory measures, including cardiac skeletal muscle and pulmonary function in non ambulatory dushane patients.
The phase two trial is designed to be a one year randomized double blind placebo controlled trial in non ambulatory boys and men affected by two Shane.
We are working on the final clinical trial design with investigators and look forward to sharing more details about the trial plans in the coming month.
I'll now pass the call over to Andrew to discuss the potential commercial opportunity with the this all the next into Dushane Andrew.
Thank you Joanne and I'm happy to share my perspective on what we believe is a very attractive commercial opportunity for insulin exit Sharon.
You saw Nexsan has the potential to provide clinical benefit broadly and serve as a foundational treatment for all duchenne patients from the time of diagnosis onwards, regardless of mutation type.
The remains a high unmet need in Duchenne with an estimated 15000 boys and men living with Duchenne in the us today and approximately 19000 in Europe.
With very few approved therapies, most of which are not amenable to the whole patient population and with no fewer available.
Unlike most rare diseases Duchenne is at an attractive commercial opportunity due to the fact that most patients have already been identified and diagnosed.
And that the majority of patients in both the U.S.
And you are treated at highly concentrated set to clinical treatment centers, which can be supported by focused sales force and medical affairs team.
In order to better understand the substantial unmet need in duchenne.
We conducted primary blinded market research with us and you providers and payers.
Which confirmed their high interest in a product like you saw next.
The most encouraging signal was it the vast majority of physicians participating in the research indicated a strong interest and using either Solomon Mexico based on the efficacy and safety profile available at the time of research.
Both providers and players like the phase three design and the endpoints.
Additionally, the payers interviewed agreed that you saw nexans potential benefit clinical benefit would justify innovated orphan drug pricing in duchenne.
Overall, we were very pleased and encouraged by their responses and feedback in both of us any any you.
We also see the planned phase two non ambulatory trial in partnership with Duchenne UK as providing important data for future market Eric.
Providing important data for future market access given that approximately 60% of those affected by duchenne, our non ambulatory and very underserved by current approaches.
We also performed market research with patient advocates parents and caregivers and they were also enthusiastic about the.
Prospect of either saw next and as a treatment for Duchenne.
The Duchenne community is eager to embrace either somaxon should it be approved by regulators and we look forward to working with the community as we prepare for the potential KMR commercialization of you saw next.
Our commercial plans are underway and as stated previously we plan to commercialize either selinexor ourselves in the U.S. first.
We see this all in Mexico is having global potential as a therapy and are evaluating our commercialization strategy outside the us.
We look forward to updating you on the overall plans in the coming months.
Next Andy vehicles will share updates from our research collaborations Andy.
Thank you Andrew.
Our research program has been focused on establishing the potential from broad therapeutic benefits of either selinexor and to shed and the potential for benefits and other neuromuscular diseases.
We've had great progress in our preclinical research programs recently.
What about collaborators is Dr. Priddy mother, the founder and medical director of the neuromuscular Cardiomyopathy clinic that you see UTI. So question as well as co director of the NIH sponsored UTI southwestern Whetstone muscular dystrophy Cooperative Research Center.
The mission of this center is to rapidly translate discoveries at the bench into therapies petition that goes in the clinic.
Our collaboration was designed to explore the potential of insulin exit to reduce cardiac fibrosis and improved cardiac function in duchenne and second muscular dystrophies.
Hi collaboration has been very successful.
We will be showing with exciting results from this look at the end of the but at the 2020 M.D.A. clinical and scientific conference in Orlando.
We're also pleased with the progress collaboration with the Jane Foundation, and does Philadelphia, which includes limb girdle muscular dystrophy type to be admire she might help pathie.
The initial preclinical data with either sold actions in the most model of it is just fill it up but they are encouraging.
Continuing the study we plan to present these results at a future scientific conference.
In addition to look to establish the potential for broad benefits of either so in essence, we.
We are on track it our preparations for NDA filings with either sell elections in 2021 that establishing our commercial supply chain.
I'll now pass the call over to know a closer to ship financial update.
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Thanks, Andy and good morning, everyone turning to our financials, our fourth quarter and full year 2019 press release and 10-K provide the details I will provide a brief summary.
As of December 31st 2019, we had $36.2 billion of cash cash equivalents and short term investments. Following December 30, Onest 2019, we raised an additional $25.6 million in net proceeds from equity financing.
Based on our current operating plan, we expect expect to be able to fund operations through a potential and the filing and into Q3 2021.
Our net cash used in operating activities was $7.8 million for the fourth quarter of 2019 and $26.6 million for the full year 2019, our R&D expense was $4.3 million in Q4, 2090 and $18.3 million for the full year.
For 2019 compared to $3.7 million in Q4, 2018, and $17 million for the full year 2018.
Our DNA expense was $2.5 million in the fourth quarter, 2019, and $8.8 million for the full year 2019 compared to $2.4 million in the fourth quarter of 28.
And $9.3 million for the full year 2018.
Our operating loss was $6.7 million in Q4 2019.
Inquiry decrease of zero point $6 million versus Q4 2018, our net loss was $6.6 million were 55 cents per share in Q4, an increase of zero point $5 million compared to our net loss in Q4 28.
Net loss for the full year, 2019 was $26.3 million compared to $25.9 million for the full year 2018.
For the fourth quarter, we had weighted average common shares outstanding of 12.4 million.
Looking forward, we expect our quarterly net losses to be higher than Q4, 2019, we anticipate net losses to increase over the course of Twentytwenty due to the ongoing phase three parse DMD trial, the increasing number of patients in the Galaxy DMD trial and additional activities to support a potential MDACC.
Mission and our commercial preparations.
I will now turn the call over to Josh to open for questions.
Thank you as a reminder to ask your question you'll need to press star one or telephone to withdraw your question press the pound Keith Please standby, we can probably choney roster.
Our first question comes from Liana Moussatos with Wedbush Securities You May proceed video.
Congratulations on all your progress and for taking my questions.
How many of the 131.
Polaris DMD boys are now in Galaxy.
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Oh, Hi, Liana. This is Joe on that's a good question. So we are obviously announcing we had announced completion of enrollment in the Polaris phase three trial back in September of 2019.
And then of course had boys.
Oh accrued over the.
Previous year.
Approximately one year and so we haven't.
That number is changing as you might imagine on a very regular basis on a weekly basis and so we don't have an exact number I believe at the moment to share.
Joe and you want to add anything too.
How galaxy is running we had a good rate of enrollment in galaxy coming out from Polaris.
And.
We are are moving along but we certainly had a good enrollment through the.
Second quarter in third quarter of last year.
Would you say about half the patients are in Galaxy now.
Lesser.
The number is changing so I'd, rather not say a number because I don't habit at my fingertips. Okay. It is actively in the ramp up phase as as you might imagine.
Okay and my next question.
If your medical conferences that you want to present data at our canceled because of Corona buyers, what's the backup plan of getting the data out there.
That is a great question as you can imagine one that we have been discussing on a very regular basis internally a catabasis because.
This is a season with multiple scientific conferences, where where we do intend to be presenting updates and so I don't have it.
Definite answer for you now, but we are actively considering that and I'm trying to identify the best way forward to make sure that we can release updates on the data.
And my last question is is there have at your trial sites have any of the health care professionals are patients been infected with Corona virus or is there any kind of new.
Procedures to take care of them.
So we are as you would imagine we're actively monitoring the Corona vars situation and we do have plans in place.
To address any potential disruptions all at Joanna address the question of whether to our knowledge. There are there any patients infected or.
Investigators.
We don't we're not aware of anyone close to the trial.
The.
I think that one of the things to remember is that the patients come in every three months.
So that we are.
Have a bit more flexibility than some trials.
And that's because of the safety profile, we seen to date.
Thank you very much.
Thanks Lana.
Thank you and your next question comes from Hartaj Singh with Oppenheimer. Sir you May proceed in your question.
Oh, great everyone. Thank you for the questions and like me on I am a graduate you on your process or I'm sorry on your progress.
Yes.
So just.
Just a couple of questions.
Sort of thinking ahead.
No one is.
You indicated that you're moving forward the initial commercialization than supply chain prep can you just talk a little bit of all of that is will you be using a CMO will you.
No manufacturer the commercial drug supply yourself, where are you between the clinical to commercial CMC kind of tech transfer process are you already their commercial can just talk a little bit about that and I just have a couple of course.
So we have not actually disclosed discussed though.
Supply chain plans and plans for commercialization.
No we actually do the have any physical capabilities of manufacturing ourselves and like most companies, we use third party providers to produce.
And distribute.
Clinical supply material and will also be doing that with our commercial materials.
Great.
Fantastic.
Then just you know in terms all well when when you're looking for to falling the Andy I know that.
Part of Pnds, some long term animal Tox studies that are ongoing even after the behind these filed in the preclinical work is done is all that compete or closing close to completion, just how you progressing and not that reports.
So we're progressing very well with full loved the Nonclinical toxicology.
Programs that are required for filing and be a we do not see that there's an issue at all.
Great Fantastic then just a question you had mentioned that you know with your partner with your current primary research.
There's a potential.
Drug.
Drugs, our risk benefit profile could support a.
Orphan like pricing strategy.
And this is the question. We just are asking more companies more frequently not because I sort of has become I think important in the drug pricing permanent so any thoughts about approaching eisler.
Thinking about working with them prior to a launch Florida.
That's a great question and something that we have under consideration and we'll certainly elaborate on more as we get closer we do feel.
It's a little early for us to be talking about pricing per se, but we do believe that.
Our next and has the potential to be a very valuable.
In addition to the treatment paradigm in Duchenne.
The only other great too is there one what are the other thing that we learn.
In the research in both the U.S. and you with the payers are still going to defer to the key opinion leaders on their advice and the risk benefit associated with products like you to telematics and so we're going to continue to maintain that dialogue not only with payers, but also with.
Well, both in the us and in Europe.
Fantastic.
And then just a couple of just Paul why don't you May all have question, which is that all let me ask you about galaxy a different way, which is that what's the total number of patients that galaxies allowed to accrue.
Including the siblings off you know boys under the age of 12 et cetera.
So I believe that clinical trials, regardless of approximately 140 odd patients and that is anticipated to encompass.
Yes, we with flexibility because its approximate all of the patients in a Polaris a certain number of siblings and we are aware of the siblings that are.
Potential enrollees, so it would be everybody.
With that will generate joined.
Then my last question is just a housekeeping question on.
And just the burn I know you had indicated that the quarterly burn will increase going forward, which makes sense. It seems you got about a twofold, one third split between R&D and Jenny right. Now you do you expect that could change to over the next for eight quarters, just any color in Boston would help and again. Thank you for all the questions.
No.
Short answer I.
I think that that split that you described has been pretty consistent over our history and unlikely that James.
Great and classic thank you so much.
Thanks, our cash.
Thank you and I'm not showing any further questions. At this time I would now like to turn the call back over to Jo mill for any further remarks.
Thank you Josh. 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 three Polaris DMD trial for you to southern accent 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 Dot Catabasis dotcom. Thank you.
Thank you ladies and gentlemen. This concludes today's conference call. Thank you for participating you may now disconnect.
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