Viridian Therapeutics Inc. Q1 2023 Earnings Call
Yeah.
Yes.
Welcome to the Bavarian Therapeutics first quarter 2023 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 be given at that time as a reminder, this conference is being recorded.
I'd now like to hand, the call over to Mr. Wheeler Stone manager of Investor Relations for Meridian. Please go ahead.
Okay.
Thank you and welcome everyone to our first quarter 2023 earnings conference call. The press release reporting our financial results and corporate update is available on the investors page of our corporate website at Www Dot Meridian Therapeutics dotcom.
Joining me on the call. This afternoon are Scott Myers, our President and Chief Executive Officer, Christian humor, our chief financial and business Officer, Dr. Rajiv <unk>, our chief product and strategy Officer, Dr. Tom Ciulla, Our Chief Development Officer, and Todd James Senior.
Vice President corporate Affairs, and Investor Relations.
Before we begin I would like to remind everyone that this conference call and webcast will contain forward looking statements regarding our financial outlook. In addition to regulatory product development and commercialization plans and research activities.
These statements are subject to risks and uncertainties that could cause actual results to materially differ from those forecasted.
A description of these risks can be found in our most recent Form 10-Q and 10-K on file with the FCC.
I would now like to turn the call over to Scott Meyers, our president and CEO .
Thank you Alicia good afternoon, everyone. Thanks for joining us today, we had a productive quarter and I'm excited to report on our progress today. After completing my first three months as Meridian President and CEO .
2023 is an important year for birdie ear that includes key milestones in all of our clinical programs I am proud of all of the progress. Our company has made thus far and looking forward to continuing our work to bring potential best in class therapies to patients with thyroid eye disease and additional disease areas in the future.
Our team has grown rapidly throughout the first quarter and we made several key hires on our senior leadership team, helping to strategically position us for future success.
Additional details can be found in the press release, we issued earlier today, but these hires include Tony Casciano brilliance first Chief Commercial officer, Dr. Tom Ciulla, Chief Development Officer, Dr. <unk>, <unk> Senior Vice President of Medical Affairs, and Dr. Eric <unk>, Vice President program leadership.
Each of these individuals have already made valuable contributions to the organization will be integral leaders for our company as we continue to expand our teams mature as an organization and prepare for future success.
I'll now review the progress we've made in our clinical programs during the first quarter and Christian humor, our CFO will discuss our financial results before we take your questions.
Let's begin with our Ted programs, starting with our lead asset <unk>, a humanized monoclonal antibody administered intravenously. Once every three weeks, which acts as a full antagonist of insulin like growth factor, one receptor or IGF oner.
In the second half of 2022 and at the beginning of this year, we reported a series of positive top line clinical data announcements from three dose cohorts of the phase one two clinical trial evaluating the safety and efficacy of <unk> in patients with active Ted.
In December 2022, our team initiated the global Phase III thrive trial, which will evaluate the efficacy and safety of <unk> 001 in patients with active Ted.
Based on our recent discussions with key stakeholders in the Ted community. There is particular enthusiasm for our shortened five dose 12 week treatment regimen of <unk> 001, compared with the eight those 'twenty one week regimen of FDA approved.
Success in the five dose arm has the potential to provide welcome convenience to patients by shortening their treatment course in eliminating three trips to an infusion center enrollment is ongoing and we continue to anticipate reporting topline results from Brian in the middle of 2024.
Moving to chronic said, we're excited to announce that our proof of concept study evaluating <unk> 001, IV patients with chronic Ted just fully enrolled as a reminder, the trial design and chronic Ted is similar to our proof of concept design that we used in active Ted <unk> and BRD and 0011 at day, one and the second at day 21.
With evaluation of safety and clinical activity at week six there are two dose cohorts and mix per kg and three bids for Cade will target enrollment of eight patients in each cohort.
Randomized three to one in favor of <unk> 001 versus placebo inclusion.
Inclusion criteria included any clinical activity score at baseline.
Expect to report results from both dose cohorts of this proof of concept trial in either June or July .
Following the results we plan to start our second global Phase III trial, <unk> two to evaluate the safety and efficacy of <unk> in patients with chronic Ted topline results from type two are expected by the end of 2024.
Now I'd like to move to our subcutaneous programs, which includes the <unk> 001, <unk> zero zero to <unk> 003, all three candidates have the potential to be developed for delivery with a patient friendly self administered pen device, which could significantly increase access reduce burden and expand treatment options for patients living with Ted.
We plan to select one of these candidates as our lead subcutaneous program before the end of the year.
With our many learnings from BRD and 001, IV preclinical and clinical work, we believe that the differentiated mechanisms of actions with full antagonism of IGF. One are achieved by <unk> 001, <unk> 003 make either the most likely to bring best in class subcutaneous product to patients as a result, the trial evaluating <unk>.
<unk> 002 in patients with Ted will only proceed in 2024 <unk> zero zero team is selected as the week subcutaneous program at the end of the year.
This allows us to keep the <unk> 002, as a potential backup while steadfastly focusing on advancing our efforts with the <unk> zero zero <unk> 001.
I will now highlight the upcoming pilot, we should get the subcutaneously program selection by year end for <unk> 001 phase one results in healthy volunteers are expected in the fourth quarter of 2023 for <unk> 002, we continue to generate data from the ongoing phase one healthy volunteer trial for.
<unk> 003, our plans remain on track to file the investigational new drug application with the FDA during the second quarter, we expect phase one results in healthy volunteers in the fourth quarter of 2023.
Absolutely subcutaneous candidate selected we expect to advance the program to a pivotal phase two slash III trial, which is planned for the middle of 2024.
Last month, our team was thrilled to present multiple abstracts and for 2023 Association of research and vision and Ophthalmology annual meeting a platform presentation featured data from our phase <unk> trial of <unk> in patients with active Ted while the poster presentations featured new clinical and preclinical research on <unk> 002 and beyond.
<unk> 003.
The Companys first presentation will be <unk> 003 research at a medical Congress, an exciting milestone in the programs development our team looks forward to presenting additional medical Congresses, and further engaging with Ted patient and physician communities throughout this year.
Finally, we continue to advance our earlier stage preclinical pipeline that will expand our disease focus beyond <unk> and into the rare and autoimmune space, our preclinical programs that could be <unk> 0040.
005006 yesterday, we announced a partnership with enable injections to utilize there and views on body drug delivery system for one of our three clinical programs. We plan to provide additional information on at least one of the programs. Later this year with that I will turn the call over to Christian who will provide a financial review for the first quarter of 2023.
Christian.
Thank you Scott good afternoon, everyone I'd like to refer you to our press release issued earlier today for a detailed summary of our financial results for the first quarter 2023, and take this opportunity to review a few items.
We ended the first quarter with approximately $373 $9 million in cash cash equivalents and short term investments compared with $424 6 million as of December 31st 2022.
We believe that our current cash cash equivalents and short term investments, excluding our $75 million credit facility will be sufficient to fund our operations into the second half of 2025.
Research and development expenses were $57 million during the first quarter of 2023, compared with $17 7 million for the same period last year.
Research and development expenses for the first quarter of 2023 include a onetime $15 million upfront payment to enable in consideration for the rights granted to viridian to utilize enable injections and fuse on body drug delivery system.
Other drivers for the increase in research and development expenses include higher CMC expenses in preparation for the IND application for <unk> III as well as development activities.
Higher personnel costs due to an increase in head count.
We have preclinical costs due to early stage collaboration expenses.
As of May five 2000, 2023 Meridian had approximately 58 million shares of common stock outstanding on an <unk> basis with that I'll ask the operator to open the call for questions operator.
Yeah.
Thank you at this time, if you'd like to ask a question. Please press Star then the number one on your telephone keypad.
I would like to withdraw your question Press Star, one again, well pause for a moment to compile the Q&A roster.
Your first question is from the line of Derek <unk> with Wells Fargo.
Congrats on the progress maybe just two questions from US maybe just first can you just talk about the variables that could lead the chronic data coming out in July versus in the second quarter.
And then also just your thinking on the Ted market opportunity given what we saw from horizon recently in their quarter results.
Think anything has changed in the market or do you kind of chalk that up to deal related things going with the ongoing Amgen deal. Thanks.
Yes, Thanks, Eric for the question this is Scott.
We're expecting a real high probability that we're going to see clinical activity in chronic Ted because it has been confirmed by the recent horizon data.
They were able to post a 62%.
Very meaningful response with a two millimeter reduction in Proptosis.
Our trial is evaluating the activity with only two doses of <unk> 001 to establish the proof of concept.
And a reduction in Proptosis, Wes and <unk> millimeter excuse me would be still be very meaningful because we still plan to.
Evaluated the longer doses right too.
We're very pleased with what's happened in that trial, we've actually over enrolled the three Meg per kg cohort. So.
Yes, once we get the process rolling where we bring in all the information from the CRO from the sites. The mris were going to need some time to really look through that and make sure. It's right and then.
We'll be ready to announce either June or July .
And then with regard to your second question was about what we saw in the horizon data. We thought it was actually very good data for patients suffering from chronic Ted and Theres still a very large unmet need in that population because surgical intervention is really all they have left.
Unless they use of systemic treatment, we look forward to seeing some more of their information when they are able to release around patient baseline characteristics, the efficacy endpoints and some of the safety. Because this was the first placebo controlled data in this chronic population and then we look forward to reporting our data in June or July of this year and is it.
A reminder, though.
Our study was set up slightly differently.
For <unk> for patients with proptosis in greater than or equal to three millimeters above normal values and then symptoms that have presented themselves after one year of steady screen.
Excuse me prior to screening.
And just a reminder, we did not have a cash requirement, whereas horizon used a zero or one for cash.
We are enrolling two cohorts there as I mentioned before with VRT Zero-zero, one theres, a 10 Meg per kg cohort in a three Meg particular randomized three to one versus placebo and there was staggered enrollment the 10 Meg per kg arm enrolled first with the three to file as I said it was overall.
So theres only two infusions.
Each of those at day, one and then again at day 21, which is Q3 weekly and then the results will be at day 42 and week six so well good.
Good good news from the Horizon data, we think that their results will play well in the marketplace with that two millimeter reduction and that will help them hopefully get more coverage decisions from insurance companies and.
With a broader label.
That will create a really nice tailwind for the market.
Derek This is Todd as far as things that could be potentially impacting safe.
Sales are what was potentially driving.
Reduction from Q4 to Q1, with we think as far as how horizon was investing in the market as far as.
Expanding their sales force.
Patient and physician support around trying to expedite.
Market access and reimbursement for patients is absolutely great places to invest in but I think to your point around potential distraction around an M&A process.
That was taking place in.
And the public headlines certainly not helpful. When you're trying to make.
Make in flight.
Operational changes to impact.
The sales trajectory.
I have a drug that chronic data that.
Scott just described will certainly help as far as being able to have educated and aware physicians and payers to hopefully drive additional sales and the chronic marketplace, but I'd also.
Just to point out that integration isn't easy either and so people should probably expect some additional impact over the next couple of quarters as amgen's integrating the horizon team.
And then with chronic dose changes that were being made I think we should be able to return to growth. Either later this year or early next year and see the patent sales pick up again.
Got it thanks, so much for the color and congrats on the progress.
Thanks Derek.
Hey, Thanks for taking my question.
Just a couple for me maybe you could you talk about whether there's a path forward with although one sub Q2 bridge PK. If you had a successful IV trial is potentially on the market faster.
And then maybe as a follow up to <unk> question.
What are your current expectations around your label do you expect to get sort of the old to private label or do you expect is the path forward for you to get the new label upon approval and if so.
How that might impact.
The commercial opportunity thanks.
Hey, Alex Yes, so for the second one.
Both the thrive and thrive two study are going to be supportive of the BLA and so then we would expect then that to translate into a broad <unk> label is similar to what Youre seeing today with the <unk> label and sorry could you repeat your first question.
Yes, yes.
Yes.
So the current way that we're thinking about it is really.
Separate programs of IV versus sub Q and so we're moving ahead with thrive and thrive too to set up for approval with the IV and then we're going to select the lead candidate in sub Q, which based off of everything that we're seeing today 003, it looks like it would have the best profile from.
The binding affinity of 001, along with the half life extension of 002, if there were a way for us to bridge from 001 sub sub Q.
From IV to <unk> Thats, certainly something we will evaluate in the future, but currently no plans to update you on that right now.
Great. Thanks.
Your next question is from the line of Gavin Clark with Gartner.
Hey, Thanks for taking my questions had to.
First I just wanted to make sure that if you do select zero-zero three ladies nothing else that could potentially slow you down specifically wondering about any formulation manufacturing or preclinical toxicology work and they'll come back for my second question.
Yes no.
All three programs are right on track and continue to evaluate them through the end of the year and as of now there is no issues with formulation.
Or the preclinical work that the healthy volunteer studies will be underway and we will complete by the end of the year.
We should be on track Devin.
This is Todd we should be on track to start our phase III pivotal then for what other program is selected as the lead at the middle of next year.
Alright, that's super helpful. And then separately on the partnership offer the on body system that was announced yesterday I just wanted to clarify was that for the next preclinical program that's going to be unveiled or is that for an earlier preclinical program that is still to be discussed.
That's for one of the three programs. We're currently developing and it will have nothing to do with Ted whatsoever.
Those new three.
Got it thanks.
Welcome.
Your next question is from the line of Thomas Smith with SBB Securities.
Hey, guys. Good afternoon, thanks for taking our questions.
A couple on Rins I was wondering if you could provide any additional color on how the thrive trial is enrolling at this point how that enrollment.
Relative to your initial modeling and then.
Secondly, I understand the difference between the inclusion criteria between your study and the Horizon study, but I was just wondering do you have any visibility into the baseline characteristics of the chronic 10 proof of concept cohorts and whether this is tracking towards more of a sort of a low cash population or kind of mid kaz patient population.
Thanks.
Yes, so with regard to the thrive setting we're really pleased that we can announce the first patient that was enrolled back in December .
We have more than 30 sites activated globally, but we're not giving any interim updates on the enrollment.
<unk>.
And the second question.
Was around that we don't we have not unblinded or baseline characteristics.
I believe they have but we have not.
Okay understood I appreciate it thanks guys.
Okay.
Your next question is from the line of Laura Chico with Wedbush Securities.
Hey, good afternoon, guys. Thanks, very much for taking the questions.
And just kind of following up on the <unk> delivery system understanding this is for.
Other programs outside of Ted, but just kind of curious why the rationale for the program and executing on that right now just any <unk>.
Advantages or criteria and you were looking for in terms of selecting this particular partner and then secondarily on the cash runway just any commentary or additional color. There on levers just on that I think R&D ticked up a little bit if im reading things correctly here and just trying to understand the expectations on the burn for the remainder of the year. Thank you.
Yes, so I'll take that this is Scott I'll take the first question and Chris I'll answer. Your second question. So the first one really goes around the hallmark and the philosophy of the company as if we can improve in a market that's been created around efficacy or potentially safety or motive delivery. Those are the three criteria that we take very near and Dear.
And look hard to do that and we believe this technology will help.
Compounded this is going to be put within our preclinical.
Klein Christian.
Laura.
So it sounds like we've got cash a little bit under two $374 million. We continue to guide cash runway into the second half of 2025, but as far as on a program by program basis, if funds basically both thrive and thrive to all the way through data at the end of.
<unk> 24, and a little bit into 'twenty five.
Our our sub acute program is funded through too.
Turning to what we're calling the bake off decision point at the end of this year, where we will select the program to move forward into pivotal trials.
Most importantly pivotal trial prep is funded so that we can move expeditiously at the end of 'twenty three into pivotal trials.
Non tech pipeline is all funded either through to candidate selection of our IND filing and you should expect us to unveil. These programs one by one and we'll let them off decides what they want to fund.
We have committed to unveiling at least one of the three programs on our pipeline.
One of our non Ted.
Our programs on our pipeline chart over the course of the rest of 2023.
Kind of.
Cash.
Cash.
<unk> expenses in Q1 was slightly higher than they usually are mostly due to kind of enable payments of 15 $15 million you should expect slightly elevated expenses again in Q2 as we initiate.
Thrive too.
And after that should normalize again back to kind of a steady state of somewhere between 35% and $45 million.
Thanks very much.
Yes.
Your next question is from the line of carpet Patel with <unk> Securities.
Yes, Hey, good afternoon, thanks for taking the questions maybe.
Maybe one more on the market opportunity here for thyroid eye disease.
Based on your conversations with Kols in this space is there a backlog of chronic Ted patients that are waiting to be treated.
And do you think that it will sort of be rapid uptake in this setting like like we saw in the acute setting or would it be a slow build given the lower severity of the disease.
Yes, Hi, this is Scott I'll take the first part and maybe Tom Shaw will add a little color because we've actually been out in the field quite a bit lately at the different conferences and visiting with our Pis and Kols and learning about that I think the belief is that there was a low hanging fruit situation with the actives, but in the words of some of the physicians.
But there there are lines of chronic patients I think.
The recent data that's been put out there.
Yes, bodes well with their broad label now and that they can take Florida as I mentioned before to get coverage decision, but I think we see there is really significant opportunity out there for market potential and to grow the market, especially when we think about our five dose regimen.
As part of our thrive study the physicians are really excited about that.
Stephen even versus the 88 dose that's already available and I think we really have the opportunity to grow the market with our sub Q offerings. So we will have a very broad approach to treating these diseases.
Disease, excuse me with whether people want to use an IV or <unk>.
Subdue and then there's also the future.
A lot of physicians are telling us around.
Still a doses with competitive sort of an acute treatment for what appears now to be a chronic disease because.
Peasant data definitely showed that these chronic patients do present themselves even after having disease in their criteria was two to 10 years and we've even heard longer than that and they are getting relief, but Tom I don't know if you'd add some color sure. This is Tom ciulla.
As Scott mentioned, we've been through a variety of Congresses recently, we were at the association for research in vision and Ophthalmology, the North American Neuro Ophthalmology Society, and the North American Society of academic orbital surgeons, where we had multiple presentations that each of these congresses. Scott actually has attended has met with several of the investigator.
<unk> and Kols and uniformly they are very enthusiastic about our.
Multiple ascending dose.
Proof of concept study in acute Ted.
I think we've gotten lots of positive feedback about that data we've also gotten.
A lot of positive feedback about horizons recent data in chronic Ted and we see this as a win for patients suffering from chronic Ted what what the Kols and investigators have told US is that they do indeed have a backlog of patients and they many of them have asked too.
Ill take part in our clinical trial investigators so.
We think there is a groundswell of optimism not only for.
Active Ted but for chronic Ted where theres a backlog because you asked about.
Okay.
Got it and can you give us a little more maybe more color between the selection of the right sub Q candidate and the timing of the start of that pivotal trial in mid 2024.
What steps are remaining to start that trial.
Yes.
Sure.
Couple of be.
002 healthy volunteer studies is ongoing right now and we have even reported some of that information out at one of the more recent conferences and then the 00001 healthy volunteer studies, which will be looking at bio availability and safety of those will be completed early enough to have by the end of the year and then we'll wind up all that information and make the decision.
About which one of the candidates we would move forward with.
And then based on that information.
We would then seek input from our stakeholders.
Sure.
Plan to start the trial mid year.
Okay.
Thank you.
Your next question is from the line of Jason Butler with JMP Securities.
Hi, Thanks for taking the questions.
You pointed to before the importance of the shortened treatment duration.
Thrive study for the IV can you just talk about how that thinking rolls over to you're planning for.
The sub Q pivotal program and I guess, we will show that.
The chronic Ted program. Thanks.
Yeah. So we're learning a lot about what's going on in the marketplace that I would say the market has changed pretty dramatically over the launch of <unk> and how physicians are actually using these drugs as I mentioned before the way that it's kind of an acute treatment paradigm today, where you give eight doses and then.
At least that's how the label read and you see how it goes but what you realize is if the thyroid is not being controlled well MD pathway has uncovered in not blocked by by using a competitive compound the symptoms of Ted return and so one of the things.
One of the reasons, we have the five dose regimen and there is one it's quicker to enroll in the patients.
When they if they see a result or not there'll be able to rollover after their dose doses onto enacted as we've seen before but the physicians just really like the idea that you may be able to treat and.
Inducer.
Response with Ted at a lower number of doses and will you also point to some of the side effects that show up on <unk> and those those higher dose numbers and the flattening of the curve. So we actually believe we will start to see a couple of people would call. It.
Treat and retreat or it could be an induction and maintenance play and where we think we're very well positioned both with our infused product line that will once its approved obviously, but also the sub Q. So then you put the treatment in the hands of the patients obviously after being diagnosed and you just have a lot more flexibility so it a lot better experience.
For the patients who don't have to go to infusion centers to be treated so we actually see a pretty significant paradigm shift that you could have sort of an induction phase when you get the acute signs and symptoms under control and then you go to a real maintenance paradigm, where there are potentially with our technology you could get to Q4.
Or once a month dosing in the hands of the patient and not seeing the caregiver, which we think is differentiated for at least the way we understand to pass is going.
And I guess just to follow up on that then.
Are you hearing from physicians, yet that there they are employing that treat and retreat.
So patients are getting treatment in the acute phase and then.
Either.
Retreated in the chronic phase or is it just too early to say that.
So I think you pretty much hit it right on the head actually so.
What we started to hear anecdotally and then multiple times from physicians, who were treating during the pandemic. They couldnt get their full eight doses because the peso had been sidelined.
Sidelined for the Covid vaccines. So they were given limited number of doses and what they would do is give a few doses and then watch and see the result and have the patient return and then they still had a few more doses to give them or and sometimes we've heard of they will give people up to five basically send them home and say you stay in touch and if you start to hear these.
Skus Philly's results.
We can come back when we can infuse you again, so no. It is almost turning into I heard from one physician it's Mike.
I'll give a few doses and then turn it into a PRN as the patient needs. It. So we are picking up that Intel.
Yes, we do have.
The benefit of fore sight versus hindsight and so we can use this intel too.
Adjust how we go to market.
Got it great. Thanks for taking the questions.
Sure. Thank you.
As a reminder to ask a question press star one on your telephone keypad.
Then if you would like to ask a question press Star one.
Okay.
Hey, guys. Thanks for taking my questions as well two.
And make any necessary.
Incineration and impacts to the trial design.
It's necessary to seek.
Either kols and or health authority feedback before we kick off the trial.
Of course, we will do that as well as far as market access decisions following that chronic data from horizon. Just now four weeks ago I think it's really too early for us to be getting intelligence around that and so that's something we will definitely be listening to as far as for <unk>.
<unk> that we're getting from the marketplace and the survey work that.
We do and of course, if we start to take care of things than it would be.
Where appropriate.
To give you that Intel has we're hearing it real time.
Got it thank you very much.
Your next question is from the line of Gavin Clark with Gartner.
Hey, Thanks for taking the follow up here.
Wanted to circle back on the extended duration or re treatment dynamic that you just mentioned.
Because today a lot of the payer policies explicitly don't allow for more than eight.
It depends on doses I'm wondering how you approach pricing and also what clinical data you need to show to allow for this dynamic in the future.
Yes so.
This would be obviously post approval.
Approval approach, we would take but.
When youre going when you could shift out of it.
From a treat and retreat to induction and maintenance the pricing could be very different because you have you might be using the sub Q. The whole time that way. So today. They are at eight but with their broader label now in a re treatment they could get they can resubmit for for a reimbursement on the next on that fit in.
Instead of going eight and eight is what I'm, referring to and have heard from a lot of the physicians is.
This is a chronic disease and the label is basically now at anytime you have Ted can be treated with the drug. So it's I think people are struggling with using eight and then eight and eight and then also with at that very high dose.
You could maybe if you use smaller doses like with our full antagonism.
We are about a third at the five dose cohort.
Not quite about half of where there'll be with their 150 mix per kg, so being able to dose.
With different dose regimens, whether it's sub Q or treat retreat with a lower number of infusion doses seems to be a preferred approach.
Got it thanks, so much.
At this time, we have reached the conclusion of the question and answer session I would now like turn the call back over to <unk>, President and CEO , Scott Myers for closing remarks.
Thank you operator, and thanks, everyone for your time. This afternoon. Please feel free to reach out to Todd or will you see if you have any follow up questions and we are happy to touch base with you. Thanks, again and have a great evening.
This concludes the conference call today, you may now disconnect your lines. Thank you for participating.
[music].
Okay.
Okay.