Astria Therapeutics Inc (ATXS) 2016 Q1 法說會逐字稿

Good day, ladies and gentlemen, and welcome to the First Quarter Catabasis Pharmaceuticals, Inc. Earnings Conference Call. At this time, all participants are in a listen-only mode. Later we will conduct the question-and-answer session and instructions will follow at that time. (Operator Instructions) As a reminder, this conference call is being recorded.

I would now like to introduce your host for today's conference, Ms. Andrea Matthews, Executive Director, Corporate Affairs. Ma'am, you may begin.

Thank you, Chanel. Welcome to today's conference call to provide a corporate update for Catabasis Pharmaceuticals and to review our first quarter 2016 financial results. With me today from Catabasis Management are Jill Milne, Chief Executive Officer; Joanne Donovan, Chief Medical Officer; and Rick Modi, Chief Business Officer.

We issued a press release after the market close today summarizing our corporate update and our Q1 2016 financial results, which we'll reference on today's call. This press release is available on our web site.

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 that were discussed in our most recent quarterly report on Form 10-Q, which we filed this afternoon with the SEC and is also available on our web site. 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 the corporate and financial update. This will be followed by Joanne Donovan, our Chief Medical Officer, who will review our progress with clinical program. Jill?

Thank you, Andrea. Good afternoon, everyone, and thank you for joining us today for our corporate and financial update for the first quarter of 2016. We believe that Catabasis is in a strong position today. Since our IPO last year, we have significantly improved our fundamentals and achieved all of our anticipated development milestones. We executed on the plan we have laid out, successfully advanced our pipeline, and we now have two programs in Phase 2 trials.

Importantly, we are now entering a period of upcoming catalyst events, including the readouts from each of our Phase 2 programs expected in the remainder of the year. We are looking forward to these readouts as they represent two non-correlated shots on goal. And while our confidence in these programs has always been high, it has increased since our last call; thanks to new data. We believe both of our Phase 2 programs, CAT-1004 and CAT- 2054, have the potential to be transformative in the treatment of the diseases they targeted, and each offers a strong value proposition in its own right.

Let me highlight the data supporting our enthusiasm. I'll start with CAT-2054 as that is the next expected readout. We are developing CAT-2054 as a potential new category of therapy; one that may be effective for NASH and hypercholesterolemia, two very large markets. We engineered CAT-2054 to target SREBP, a master regulator of lipid metabolism. We have been aware for quite some time that SREBP targets proteins that play an important role in the pathogenesis of NASH, and therefore, we have been pursuing NASH both internally and with external collaborators.

Our studies have now borne out that by inhibiting SREBP, CAT-2054 may demonstrate significant effects not only on LDL-cholesterol but also on liver inflammation, fibrosis, and steatosis. We have seen promising preclinical results in multiple NASH models using an analog of CAT-2054, including positive effects on liver inflammation, fibrosis, and steatosis. Notably, we saw a return to baseline for ballooning degeneration and a reduction in pre-neoplastic lesions, two negative consequences of NASH. We plan to present these results at an upcoming scientific conference later this quarter.

Recall that we completed a Phase 1 trial of CAT-2054 and observed no safety signals, and we have seen good tolerability and reductions in LDL cholesterol. We also did not see any effects on the PK of atorvastatin. We believe these results bode well for the four-week Phase 2a trial of CAT-2054 we are now conducting in patients with hypercholesterolemia in addition to high intensity statin.

I am pleased to say enrollment for this trial is complete, ahead schedule, and we now expect to release top line results around the midyear ahead of our previously announced expectation of Q3. We look at the Phase 2a trial as a dose finding and proof of concept study for SREBP inhibition that may guide future studies in NASH and hypercholesterolemia.

Given the positive preclinical results we have seen in NASH, we would view seeing no safety signals, good tolerability, and statistically significant reductions in LDL cholesterol during the four weeks of therapy as a success. We also believe that LDL-cholesterol reductions would be proof of concept supporting clinical SREBP inhibition. Our strategy is to out-license CAT-2054 prior to Phase 3 with acceptable terms. If this strategy is successful, it might offer an attractive path to monetizing this asset.

Let me know move to our lead program, CAT-1004. We expect to report top line data for CAT-1004 from the Phase 2 portion of the MoveDMD Trial in late 2016. I'm happy to say we have the INN name for CAT-1004, edasalonexent. We are seeking to develop edasa as a new standard of care for Duchenne muscular dystrophy, and believe that it has a potential to be effective in all patients with Duchenne regardless of mutation type.

Edasalonexent has the potential to be disease-modifying, meaning it could slow the relentless degeneration of muscle while enhancing muscle regeneration. We are pursuing this vision by a well-thought-out development plan that we believe is consistent with regulatory guidance using randomized double-blind placebo-controlled trials. As a reminder, edasalonexent is an oral small molecule that inhibits NF-kB, a protein that is activated in very early stages of DMD and is instrumental in the progressive muscle degeneration in DMD. This inhibition of NF-kB is also important in a number of other rare diseases. We are currently conducting the MoveDMD Trial in DMD boys ages four to seven who are at a fairly early stage of their disease and so are relatively homogeneous in terms of their state of disease progression.

We presented positive safety, tolerability, and pharmacokinetic data from Part A of the MoveDMD Trial at the 2016 Muscular Dystrophy Association Clinical Conference in March and have also recently announced positive NF-kB biomarker data from Part A demonstrating significant target engagement in a dose-dependent manner in boys affected by Duchenne.

Note that we have now shown safety tolerability and target engagement for edasalonexent in three different clinical trials; two in healthy adults and one in boys with DMD. We have found our interactions with the FDA to be collaborative and constructive. We have initiated Part B, the Phase 2 efficacy portion of the MoveDMD Trial. Assuming patient enrollment proceeds as anticipated, we expect to release top line results from our MoveDMD Trial late this year. Catabasis appreciates receiving grant funding from the Muscular Dystrophy Association to support Part B of the MoveDMD Trial, which is in addition to the parent project muscular dystrophy funding support we received for Part A.

In addition to our clinical stage product candidates, we have a robust preclinical pipeline. The most advanced program is CAT-4001, a dual NF-kB inhibitor NRF2 activator, which we are evaluating for ALS and Friedreich's ataxia. In January, we announced the grant from the Friedreich's Ataxia Research Alliance to help fund our exploratory and preclinical work. We are conducting further preclinical studies with CAT-4001 in both ALS and Friedreich's ataxia, and plan to conduct IND-enabling activities. Assuming success with these activities, our goal is to advance CAT-4001 into the clinic next year.

We continue to acquire our SMART linker technology to discover and develop candidates for additional rare diseases, where targeting multiple biological pathways may yield important enhancement and efficacy. Our current efforts here focused on rare diseases, including ALS, Friedreich's ataxia, and cystic fibrosis. A paper on our SMART linker drug discovery platform was featured in the February issue of the Journal of Medicinal Chemistry detailing our proprietary technology that generates our pipeline and development candidate.

We're also very pleased to have two highly qualified industry experts joined the Catabasis Board of Directors last month. Dr. Burt Adelman and Michael Kishbauch, both bring deep strategic and operational experience in performing senior leadership roles in large cap pharmaceutical, as well as development stage biotechnology companies. Burt brings extensive drug development and commercialization experience, and Michael has more than 20 years of executive leadership and company building experience in the pharmaceutical industry.

We also announced the formation of a Science and Technology Committee of the Board, chaired by Dr. Adelman to advance our innovative approaches to treat diseases with high unmet needs. Burt and Michael will be an integral part of the team, and we look forward to their contributions and guidance.

For our first quarter of 2016 financial update, our press release this afternoon provides details. So, I'll just provide a brief summary here. As of March 31st, we had $52.6 million of cash, cash equivalents, and marketable securities, which we expect to provide us runway through Q2 of 2017. Importantly, we expect that our current cash balance will provide funding well beyond both the edasalonexent MoveDMD Part B readout and the CAT-2054 Phase 2a readout.

In the first quarter of 2016, our net cash used in operating activities was $9.1 million. Our R&D expense was $6.4 million in Q1, an increase of $1.8 million over our R&D expenses in Q1 of 2015. The increase was primarily due to increased direct program costs related to the edasa MoveDMD Trial and the CAT-2054 Phase 2a trial.

Our G&A expense was $2.8 million in Q1, an increase of $1.1 million over our G&A expense in Q1 of 2015. This increase was primarily due to increased employee compensation cost and increased consulting and professional expenses to support our more advanced R&D pipeline and overall growth.

Our operating loss was $9.2 million in Q1, an increase of $2.8 million versus Q1 of 2015. Our net loss of $9.4 million or $0.61 per share in Q1 was an increase of $2.9 million over our net loss in Q1 of 2015. For the first quarter, we had weighted average common shares outstanding of 15.3 million. Additional financial information is available in our 10-Q which we filed with the SEC earlier today.

I'll now ask Joanne Donovan, our CMO, to provide a more detailed update on our clinical programs.

Thank you, Jill. Good afternoon, everyone. I'll start with the CAT-2054 Program as that is expected to readout next around the midyear. We're developing CAT-2054 for the potential treatment of NASH and hypercholesterolemia. We've completed a number of studies to further characterize the molecular mechanism of the CAT-2000 series molecule through our collaboration with the UT Southwestern Medical Center.

You may remember this center has done pioneering work in cholesterol metabolism, and they are experts in the SREBP pathway. We've also completed our own preclinical studies in multiple models of NASH using CAT-2003, an analog of CAT-2054. CAT-2054, of course, is our active program in the clinic. We believe the findings have been consistent and compelling showing inhibition of both isoforms of SREBP, SREBP-1 and SREBP-2, with impact on downstream genes and metabolic parameters supporting the potential of CAT-2054 in both NASH and hypercholesterolemia.

In preclinical models of NASH, we've also demonstrated robust reductions by CAT-2003 in liver inflammation, fibrosis, and steatosis. Importantly, we saw a reduction of ballooning degeneration to baseline level, and we also saw reductions in pre-neoplastic lesions. We intend to present these results at a scientific conference later this quarter.

Our takeaways are that CAT-2054 may have therapeutic utility in NASH, as well as positive effects on atherogenic lipids. NASH and hypercholesterolemia are in fact interrelated indications as NASH had been shown to be a cardiovascular risk. Recall that we have demonstrated safety, tolerability, and LDL effects with CAT-2054 in the Phase 1 trial as well as no evidence of impact on atorvastatin PK.

We're currently conducting a four-week Phase 2a randomized, double-blind, placebo-controlled trial of CAT-2054 in addition to high-intensity statin in patients with hypercholesterolemia. I'm pleased to say that we've completed enrollment ahead of schedule, and now expect to report top line results around midyear.

As planned, the Phase 2a trial enrolled approximately 150 patients with hypercholesterolemia on statin. After a lead-in period with four weeks of high intensity statin, atorvastatin 40 milligrams, patients were randomized to doses of 250 milligrams a day, 400 milligrams a day, 250 milligrams twice a day, 400 milligrams twice a day of CAT-2054 or placebo in addition to atorvastatin. The primary endpoint, efficacy endpoint for this trial is percent reduction in LDL cholesterol from baseline.

We're also exploring the activity of CAT-2054 and other parameters including trends in liver function test. We look at this trial as a dose finding and proof of concept study for SREBP inhibition that may guide future studies in NASH and hypercholesterolemia. Given the positive preclinical results we've seen in NASH, we view seeing no safety signal, good tolerability, and statistically significant reductions in LDL-C versus placebo during the four weeks of therapy as success for the currently running Phase 2a trial. Seeing favorable trends in liver function test would be a bonus.

Now, I'm going to move to our lead program, CAT-1004, also known as edasalonexent or edasa. The MoveDMD Trial with edasalonexent is being conducted in two sequential parts, Part A and Part B. In Part A, 17 ambulatory boys between ages four and seven, who are genetically confirmed diagnosis of DMD, received edasalonexent for seven days at doses of 33, 67, or 100 milligrams per kilogram per day. The boys were steroid naive or had not used steroids for at least six months prior to the trial. We presented the results of Part A at the 2016 MDA Clinical Conference.

We're very pleased with the results. All three doses of edasalonexent tested were generally well tolerated with no safety signals observed. The majority of adverse events were mild in nature. The most common adverse events were gastrointestinal, primarily, diarrhea. There were no serious adverse events and no drug discontinuation. Furthermore, doses of 67 milligrams and 100 milligrams per kilogram per day achieved exposures at which NF-kB inhibition was observed in adults. We've also recently announced positive biomarker results from Part A of the MoveDMD Trial demonstrating successful NF-kB target engagement.

After seven days of dosing, the biomarker assay piloted in boys affected by Duchenne showed a statistically significant decrease in NF-kB targeted gene expression compared to baseline at the 67 and 100 milligram per kilogram per day doses, consistent with what we have seen in adults. This is particularly encouraging when we consider that preclinical biodistribution study have shown that edasalonexent concentrations in muscle are five to 10 times higher than in plasma. Based on these results, we advanced daily doses of 67 or 100 milligram per kilogram per day into Part B, the Phase 2 part of the trial.

Now that we've shown safety, tolerability, and target engagement for edasalonexent in three different clinical trials, two in healthy adults and one in boys with DMD, we plan to submit the Part A biomarker data for presentation at an upcoming scientific meeting. We've had what we view as collaborative and constructive interactions with the FDA and initiated the Part B Phase 2 efficacy portion of the MoveDMD trial, a randomized, double-blind, placebo-controlled trial to evaluate the safety and efficacy of edasalonexent in DMD over a 12-week period.

We're enrolling 30 boys, age four to seven, affected by Duchenne in Part B. All of the boys that were originally randomized to placebo for 12 weeks will then receive edasalonexent for a subsequent period of 12 weeks so that all boys that will be enrolled in the study are expected to receive edasalonexent treatment. The 17 boys that participated in Part A are being asked to participate in Part B and additional patients are also being enrolled.

Entry criteria are similar to those in Part A. The primary endpoint for Part B of the trial is MRI with age-appropriate time-functional test to secondary endpoint. These include the 10-meter walk, run, time to stand, and four-stair climb. Note, that we're not including the six-minute walk test as that test isn't appropriate for boys of this age due to the attention requirements.

We're also measuring the North Star Ambulatory Assessment, the PODCI and muscle strength. If the safety profile of edasalonexent continues to be acceptable -- in 2016, we anticipate continuing into an open-label extension study following MoveDMD Part B.

We've looked at corticosteroids as providing proof of biology as they have shown positive results on MR in 12 weeks in small sample sizes. Assuming positive data from the MoveDMD Trial, we plan to work towards the study in non-ambulatory boys with DMD in 2017. Assuming positive MoveDMD Trial data and after regulatory discussions, we expect to design and conduct the placebo controlled pivotal Phase 2, Phase 3 study in 2017.

We expect that this study will be six months in duration with one of the age-appropriate time functional test as the primary endpoint. This study would be informed by the results from our current study. We want to say also that we're very appreciative of the strong interest in the MoveDMD Trial and want to thank participating families, advocacy groups, investigators, study staff, and experts for their support and participation.

Importantly, our approach to developing a therapy for DMD is highly differentiated from the programs in late stage development in the indication. Edasalonexent has the potential to be affective in all patients with Duchenne regardless of the mutation type. We anticipate that edasalonexent will be disease modifying, meaning slowing the relentless muscle degeneration seen in Duchenne and also enhancing muscle regeneration.

In our current development plan for edasalonexent, we do not need to do muscle biopsies with the inherent variability that they bring. We're not using the six-minute walk test, and we plan to perform a pivotal placebo-controlled trial. Our plans follow a well thought-out development path to approval with adequately sized randomized, double-blind, controlled trials, and safety database. We believe this path is consistent with regulatory guidance and aligns well with our learnings from the recent briefing books and Advisory Committee discussions.

More importantly, we're leveraging and intend to continue to leverage our fast-track status to work closely and collaboratively with the agency. Whereas we're developing and are confident in the potential of edasalonexent as monotherapy for the treatment of Duchenne, we believe that a combination drug approach could offer additional benefits. We're continuing to follow the literatures suggesting that NF-kB inhibition strategy, combined with the dystrophin-targeted approach, may further enhance dystrophin production, and we intend to explore this combination approach over time.

In summary, we are very pleased with our execution and results so far with our two Phase 2 programs, and our confidence continues to increase for each of them. This concludes our prepared remarks, and we'll be now happy to take your questions. Operator, can you please repeat the instructions and poll for questions? Thank you.

No problem. (Operator Instructions) And our first question comes from the line of Christopher Marai of Oppenheimer. Your line is now open. Please go ahead.

Hi. Good afternoon, guys. Congrats on the progress and we look forward to your data. Perhaps I'll take this opportunity to put Joanne on a hot seat for a few minutes here. With respect to CAT-2054, what type of percent change in LDL-C from baseline are you looking for to sort of trigger a successful trial readout, if you can maybe remind us? And then in terms of favorable trends in liver function, again, what would you perhaps be looking to see there numerically? Thanks.

Okay. So, in -- what we are hoping to see is a statistically significant reduction in LDL. This is a four-week proof of concept study, and we're also looking at safety and tolerability. So, that's what we are considering success in the study. We're looking at trends in LFTs. We know that these move relatively slowly over time, but we are looking to see the standard ALT, AST and other liver function tests over time, and we would consider that actually a bonus.

Okay. And then just in terms of the percent change that you would deem successful?

So, we're considering a statistically significant effect as a success in the study.

Okay. And then with respect to CAT-1004 edasa, nice name, by the way, maybe share your thoughts regarding the benefits that might be delivered to patients versus a steroid. And I think the steroids also work by inhibiting some of the NF-kB signaling but perhaps other mechanisms. And what have you done in terms of maybe teasing out that particular biological or self-signaling effect given your SMART linker technology to target those specific signaling pathways? Thanks.

Thanks. Thanks, Chris. So, we think that the NF-kB pathway has important advantage. It has -- basically, it is targeting not only inflammation, but it is also allowing muscle regeneration, and that's very different from steroid. And so we think that over the long-term, this can provide important benefits beyond potentially what steroids show. I mean for the short -- in the three-month study, what we're looking at is evidence. To the primary endpoint, we're looking at evidence of a decrease in inflammation changes in muscle on [MR T2].

Okay. And then so would you expect that over time the T2 MR measures of inflammation would reduce?

Yes.

And that there wouldn't necessarily be a bad thing if the reductions were less than what you might initially see with steroids? Would that be a correct assumption to sort of -- just to sort of frame, again, expectations for the dataset and try to understand what we're looking at relative to like you noted that the data from the [T2 MR] signals that's --

Yes.

-- in the published literature.

Yes. I think what we are considering a success for this trial is a reduction in T2 over three months. We've that that is possible in terms of having an effect very rapidly. T2 is a parameter that changes quickly. We'll also be looking over the longer-term, though. And as we advance into an open-label extension, we'll also be able to look at not only T2 but over the longer-term of liver fat changes as well.

Okay. Great. And then just remind me, one last question, on the T2 signals do. Do those bounce around a lot? Or do they tend to be pretty consistent entering in one direction in patients especially over the time course you're measuring? Or, I guess, what was the variability in that measurement? Thank you.

So, these -- the T2 increases inexorably over time in these patients when you look over average. And even in a small number of patients in a literature, they were able to see statistically significant changes versus corticosteroid-naive patient. So, we think that this is -- we're able to power this based on a study that we are very closely following the pattern of in a literature, which we think that de-risks the approach and allows us to power it appropriately.

Great. Thanks for all the questions. I'll jump back in the queue.

And our next question comes from the line of Joel Beatty of Citigroup. Your line is now open. Please go ahead.

Hi. Thanks for taking the questions. First question is the recent ad com on DMD, and I'm just curious if you had any takeaways from the meeting that might affect to the outlook and the potential for applying based on data from the current trials for the drug formerly known as CAT-1004?

Yes. Sure. Thanks, Joel. This is Jill. So, yes, certainly, we've been following very closely the recent Advisory Committee Meetings. And I think what's clear to us is the FDA clearly recognizes DMD as profound unmet medical need and is working with companies like ourselves and the others to try to get some new medicines approved in this area.

For us, I think our -- there were couple of takeaways for us. One is I think it certainly gave us confidence and reinforced our plans, our development plans for edasa and what we're doing in our MoveDMD trial, and gave us confidence that we're on the right path there.

And I think we certainly have benefited with our program, and that we've been able to design our MoveDMD Phase 2 trial post the release of the 2015 FDA Guidelines which certainly puts in a good position. In addition to that, as you know, for edasa we have Fast Track status, and of course, we take full advantage of getting in front of the FDA and interacting with them as much possible along, at each major step in our program. And I think that Fast Track status, combined with having the regulatory guidance already out there, sets us up well for planning our pivotal Phase 3, which if the MoveDMD Trial is successful later this year, we intend to initiate in 2017.

Okay. Good. And then the question on CAT-2004, talk about potential partnering after we received the data. Do you have an idea of how you would like that partnership to look like if there's any particular aspects of the drug that you're looking to retain?

Yes. Sure. I'm going to hand it over to Rick Modi, our Chief Business Officer, to address that because he's obviously directly involved in those discussions.

Hey, Joel. It's Rick. Thanks for that question. So, we've been having partnering discussions since we actually had the Phase 1 data in hand. They have an interest from some of the big players that are out there. And since NASH data has been in our hands as well, we've continued those discussions both with those partners that we're interested post-Phase 1 and then some additional ones that have interest in NASH as well. And our vision for this type of a deal would be to essentially do global out-licensure for the CAT-2000 series. Does that answer your question?

Yes, thank you.

And our next question comes from the line of Kelechi Chikere of Wedbush Securities. Your line is now open. Please go ahead.

Yes. Hi. Thank you for taking my question. For CAT-1004, I was wondering if you can provide any additional color on enrollment. I guess, for instance, to date, how many boys from Part A have gone over to Part B? And for 2054, how quickly following results from the Phase 2 trial can you initiate a study in NASH, if at all? Thank you.

Great. I will have Joanne Donovan, our CMO, address the enrollment question around CAT-1004.

Yes. We have been very gratified with the results of enrollment with the patients who were already in the study as well as interest from other patients as well. So, we are confident in our ability to enroll the study and get to top line results at the end of the year.

Okay.

Great. And for your second question with regard to CAT-2054 and initiating a Phase 2b trial in NASH, so clearly, we will wait and see the results of the Phase 2a study which will read out around midyear, and so we're certainly eager to see that. We're also working toward longer-term tox studies, which are -- as well as preparing materials for the next clinical step. So, certainly more to come with regard to the timing of that next step.

Okay. Thank you.

And our next question comes from the line of Phil Nadeau of Cowen. Your line is now open. Please go ahead.

Thanks for taking my questions. A couple on 2054, just on the safety profile. Do you a sense of what level of GI adverse events would be acceptable based on your work with advisers?

Yes. This is Jill. I'll let Joanne take that question.

Okay. I think that we have had an excellent safety and tolerability profile to date. And so, I think that you are -- the way you're going is, of course, this is a long-term indication, and we need to have a good safety and tolerability profile. And we are -- that is our expectation for the study to be considered successful.

Okay. Good. And then, second, as you think about possibly moving forward to NASH, what population of patients would be most appropriate? Would be early stage pre-cirrhotic? Or is there a way that you can actually help cirrhotic patients?

Yes. And this is Jill and maybe I'll start and then I'll let Joanne finish. I think at this -- what we're right now is obviously evaluating closely the data that we've generated from our preclinical studies in NASH, and also we'd be waiting to see the Phase 2a results. But in addition to that, we're actively in conversation with experts in the NASH field to really address what is the right target patient population for this agent. Certainly, what we've seen preclinically and the effects we've seen, we believe they could have broad potential in the NASH patient population, but certainly we'll be embedding what the best plan for this there. And I don't' know if Joanne wants to add any more color to that.

I don't have much to add to that. We were very pleased to see the data are on ballooning degeneration and fibrosis because those are our key to the preventing or treating more advanced NASH. So, we wanted to see that we have effects potentially across the spectrum, and then we'll work with the experts who we've been talking with about designing the best next trial.

Okay, great. And then, just one last question, on the -- you build the 2054 to lower LDL. I believe in earlier trials, you saw a somewhat unique time course to LDL lowering or would actually continue to maybe even increase after dosing? Correct me if I'm wrong there. Is there - could that be captured in the structure of the current study? Will you be following patients post-dosing? And have you done any work on the mechanism of that unique time course?

Yes. That's a great question. You remember it correctly. So, certainly -- so, what we did see was statistically significant reductions in LDL cholesterol after 14 days dosing. And those effects grew in out to seven days post that dosing. And we think that might be related to actually the mechanism here, and that we're inhibiting SREBP, which is really a master homeostatic regulator of lipid metabolism. And so you might anticipate that the effects would grow in over time, and indeed, that's what we've seen.

We've designed our Phase 2a study to capture that as well. So, it's obviously four weeks in duration of dosing with CAT-2054. We'll have a follow-up, a 21-day follow-up period post that as well to follow those kinetics because that's something we're very interested in understanding. And, of course, as we think about next steps for this program clinically, it's obviously longer term studies because we really truly think with this type of a mechanism, inhibiting a homeostatic regulator that it might take time to really get the full effects of that inhibition. So, that's certainly something we're addressing.

Great. Thanks for taking my questions.

And I'm showing no further questions at this time. I would now like to turn the call over Ms. Jill Milne for closing remarks.

Great. Thank you. Thank you, operator, and thank you to everyone for being on the call today.

In summary, already in 2016, we have completed enrollment for the Phase 2a trial for CAT-2054 ahead of schedule. We've presented positive MoveDMD Part A trial results for edasalonexent, including demonstrating NF-kB target engagement in boys affected by Duchenne. And MoveDMD Part B is underway after what we view as collaborative and instructive interactions with the FDA.

We believe each of our Phase 2 programs has the potential to be transformative in the treatment of the diseases they target, and each offers a strong value proposition in its own right. We published aspects of our SMART Linker Drug Discovery platform in the Journal of Medicinal Chemistry. We received grant funding from MDA to support MoveDMD Part B as well as The Friedreich's Ataxia Research Alliance in support of CAT-4001 research.

And two distinguished members have been added to the Catabasis Board of Directors along with the formation of the Science and Technology Committee. We expect to report top line Phase 2a results of CAT-2054 around midyear and top line results for Part B of the MoveDMD trial for edasa late this year. We believe these readouts represent two non-correlated shots on goals; one in which our confidence has always been high and has increased since our last call. Thanks to new data.

Thank you, everyone, for joining us on today's call, and thank you for your support of and interest in Catabasis. Andrea?

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

Ladies and gentlemen, thank you for participating in today's conference. This concludes today's program. You may all disconnect.