Q4 2021 Matinas BioPharma Holdings Inc Earnings Call

Period.

Viral vectors.

Yeah.

Because of their unique structure and delivery mechanisms.

This specific cellular targeting hep.

Thanks, Jerry and good morning, everyone.

Full year 2020.

Cash cash equivalents in marketable securities at December 31, 2021 were approximately $49 $6 million.

Compared to $58 7 million at December 31, 2020.

I will now turn the call back over to Gerry.

Thanks Keith.

In summary, we are on a strong trajectory for a highly successful year ahead, we continue to deliver a highly compelling clinical data with the enact trial cohort four data expected early in the third quarter of 2022, and recently partnering with a global CMO to drive toward NDA readiness for.

At $22 three positioning it to hopefully potentially become the leading therapy in the treatment of invasive fungal infections, we continued to invest and expand our discovery team with active programs now and a variety of areas in the nucleic acid space will have data from genentech and from <unk>.

Iliad coming up in the middle of 2022, and the potential for early data from our discovery programs in messenger RNA DNA and antisense oligonucleotides, we're intent on driving these programs forward and addressing some of the major challenge in the delivery of these therapeutics, we have Rio.

Mentum going forward near term milestones and we look forward to keeping everyone updated on our progress through 2022 with that I will turn the call over to the operator for a question and answer session.

Thank you well now be conducting a question and answer session if you'd like to be placed in the question queue. Please press star one on your telephone keypad, a confirmation tone will indicate your line is in the question queue. You May press star two if you'd like to remove your question from the queue for participants using speaker equipment. It may be necessary to pick up your handset before pressing.

One one moment, please while we poll for questions.

Our first question today is coming from Bert Hazlett from <unk>. Your line is now live.

Yes. Thanks, Thank you for taking the questions and congratulations on all the progress a material material efforts underway and and obviously you have been accomplished during 2021 with regard to 2022, Jerry you made a couple of very intriguing points with regard to interest by third.

Parties I'm interested in the strategy in general there interest I guess is growing in 'twenty, two or three is that a candidate for licensing or partnering.

Or is it more with regard to other opportunities in the platform like antisense, oligos or RNA or DNA or other different types of molecules just interested in the strategy more broadly with regard to Medina.

Yeah, Great question and thanks for joining this morning.

I think 22 or three has always been for us a foundational drug.

It's clearly demonstrating the key attributes of the platform, we're getting oral bioavailability, we're keeping patients safe and we're having success in targeting infections and hard to reach places in the body. The data from enact those first three cohorts and what we're seeing in cohort four to set this up.

It could be a very successful drug and that's attracted a lot of interest from third parties. So our strategy is to capitalize on that we've seen deals.

Deals in the anti infective space.

During and just after phase II from global partners, where the increasing incidents of infections globally and the lack of investment in this space has made companies very hungry for differentiated assets and when Youre talking about 'twenty, two or three with its broad spectrum nature of amphotericin B and now the <unk>.

Opportunity to make that oral and essentially safe.

You're you're talking about a drug that is an opportunity to treat tens of millions of patients. So we are preparing this drug because of interest that's been expressed already we have an idea of what these companies are looking for and for US. It's a question of timing and checking boxes. So as we think about where <unk> is today with roughly 34 employees in AR.

Focus on discovery and formulating drugs and in creating drug candidates. We do believe that 'twenty two or three is a great candidate for a partnership that doesn't mean that we would not continue to develop that drug through NDA filing.

And perhaps through global.

Development as well, but the interest is real and we think they care about things like.

Cohort four data the cohort five validation from FDA certainly now that we have a global CMO, that's a big box.

Being able to show that you're a CMC and scale up.

At least planned is really important getting that advice for me I E. M. A is also setting this up so we think in the second half of this year, specifically off cohort four that interest is going to intensify its already strong it's going to intensify and we think.

Getting through some of these near term milestones will also increase the value to the point, where it does make sense for us to entertain that and I would say 25 are ones in the same bucket because as you look forward with mid teen is our strategy here is we're a platform company and we have a differentiated.

<unk> unique.

Platform, the intracellular platform delivery and if you look even today the interest and the importance of delivery is highlighted with every every day moving forward I mean voyagers deal with Novartis today, you take keen interest and things like that where companies are investing earlier and earlier.

In in potential delivery solutions.

In the gene therapy, and nucleic acid space. It's one of the reasons why we've invested so heavily internally in the back half of 2020 , one and now turning to page to 2022 and nucleic acids.

And then in the partnership interest is real there too. So it's a combination of setting our clinical stage assets up for commercial partnerships and then expanding the utilization of the platform to the point, where we can enjoy some of those more meaningful collaborations with third parties and the Alagoas space.

Sure.

That's terrific makes a lot of sense I have about seven or eight more but I'm only going to ask one.

To ask about two questions two programs excuse me.

Could you describe a little bit more about the data that is upcoming but we can expect with regard to 'twenty five O. One.

During the year and then with regard to the remind us of the Gilead.

And and Genentech data that we might see during the year as well thanks so much.

Sure. Thanks, Bruce So 20 501 first look it's a little bit in the shadows of 'twenty, two or three just because it's a little earlier, but when you have the potential for the first oral aminoglycoside the big drug with a lot of interest we actually had another great update meeting with the cystic fibrosis Foundation yesterday, who continues to be very.

Motivated in helping to drive the development of that drug we're right now in a single ascending dose PK study. So we're gathering data about the safety of our drug in healthy volunteers enrolment.

It is going well and we expect to have data right in the middle of 2022, but we expect this drug to be safe. So what we're really doing now in the background. We're also doing some longer term tox studies, which are going to set us up for a phase II trial, we need to begin and have begun to plan what that may look like with.

The cystic fibrosis foundation, and others, but the data from the U S. A D is is really validating that we continue to have a safe drug I mean amikacin.

Is one of the most highly toxic aminoglycoside.

In addition to sort of nephrotoxicity you get OTO toxicity, you can take away you're hearing and so we need to continue to demonstrate that <unk> can eliminate that toxicity. In addition to facilitating intracellular delivery, which can target the therapy right to the lung or.

A lot of therapies targeting N T M and other pulmonary infections fail, because they can't get any drug or the case. They can get limited amount of drug to the lungs, while we've shown the ability to get drug right to the lungs, and thats because of our mechanism of action being taken up by monocytes that maturing into macrophage macrophage.

Just going right to the site of infection. That's been established now in multiple preclinical studies and obviously.

<unk> is another validation of that so we're excited about 20 501, the data from S. A D. We believe will sort of be consistent with what we've seen with the safety of our platform.

And so we're looking forward to that on the Genentech front. The third compound we've done a small molecule. We've done an antisense oligonucleotide. The third compound is going to be different and it's going to be where we can't talk about it yet, but it's going to be in a very very interesting area something that we have not we've done some.

Type of these molecules before but not this specific one so it's going to be an opportunity to expand the platform. We're waiting for the final protocol from Genentech for that.

But we're excited about that because we think more so than the small molecules and Ali goes this is going to allow us to put a stake in the ground, which we think will cause investors in the market there their head Altera and then on the Gilead front.

Second in vivo study is going to start we say shortly I'm talking about in the next week or so.

<unk> formulations were sent down a few weeks ago and that's important because it's going to be another COVID-19 model, it's going to give us the opportunity to show that we can knock down the inoculum, there and essentially deliver a pro drug orally, which is really really hard.

Industrial is not an easy drug to work with and for us.

<unk> to work with Gilead and the NIH is continued validation of our platform in more and more areas. So regardless of how you feel about rem density or as a drug opportunity moving forward. This is additional data with the delivery of a prodrug and an anti viral.

And with the way the world is evolving here antiviral medication, specifically oral antiviral medications.

We're going to become more and more important and our unique ability to get them inside cells without any unintended immune response is a great demonstration so and that's coming in the middle of 2022 as well Bert So two really good questions.

Thank you really appreciate it look forward to the data.

Thank you and just a reminder that star one to be placed in the question queue. Our next question today is coming from my own Tommy from B Riley. Your line is now live.

Hi, So this is all from my answer thank you for taking our questions. So can you share more details of the design for cohort five.

And I really want to know where this new call herd has little impact on the timeline.

Also can you clarify why this cohort it might appear required in terms of the regulatory requirements because on one side as requested by FDA, but on the other side. It seems that you still need FDA input on the design.

Greg It's a good question and I'll ask Dr. <unk> to go into some some detail. So our discussions in December that's focused specifically on this and the opportunity in the pathway.

Four to filing an NDA for step down treatment. So theres always already been a good deal of FDA input into this idea of cohort five it being.

Factory to them in qualifying for essentially a pivotal stage cohort and generating the data necessary to file an NDA. So now taking that feedback from December or discussions you know in early April .

We're really just refining the design that FDA has already comfortable with so it's really going to be narrowly focused on the endpoint, which we've said we believe can be 30 day survival, where our data is very compelling versus IV amphotericin, the non inferiority margin.

And then the size of the safety database. So overall it doesn't change our timeline CMC was always our gating item to filing an NDA and with the expansion of the number of clinical sites from two to five in Uganda, We think we can enroll it.

I'm reasonably quickly keeping in mind that this is still a deadly invasive fungal infection. So it doesn't necessarily change our timelines too much in terms of being able to file an NDA and but we do expect that our our discussion with FDA in April will be pretty narrow in focus.

And gaining their agreement is important but we feel that they already have a lot of information and I've already provided a lot of input, but Terry maybe you can expand on that a little bit.

Sure. Thanks, Jerry and you covered it very well so we're really very well positioned now to have a very focused discussion with the FDA. We've already crossed the major hurdles with getting their alignment that we don't need to expand the program into U S patient, we can focus on where.

We know that patients exist and where there is a high unmet need in Uganda, where we know we can enroll the trial very quickly at the same sites at the same centers that are already treating patients with our war on that 22 or three.

The operational execution should be very seamless to asthma really just replicating what was already demonstrated in cohort two.

It will continue to be we expect an open label design.

Or an endpoint that we know we will win a based upon of course the data that we've generated to date. So we were optimistic that FDA will agree with the endpoint and the statistical analysis plan that we will be submitting for they're there.

We view and really this is just a final step in getting FDA alignment on the study that will be a critical for NDA submission.

Yeah.

That's very helpful and just a little while so alive here. After the April discussion long really hear any feedback or update on this program.

Sure.

Great. Yeah go ahead Terry.

Sorry, It takes about 30 days within 30 days, we should we see the final minutes.

But we'll have an idea.

Around the time of the meeting as we get their written feedback or initial thoughts on the design, but final feedback is typically 30 days after that the meeting is concluded.

And just given the way the calendar is setting up it does look like we'll be in position to provide that substantive input during our first quarter conference call in may so the calendar sort of working in our favor, whereas the update comes in it's sort of right in line with when we expect to inform the market about Q1 results. So it's going to line up pretty nicely.

And by then we should also be much further along but with cohort four.

So during that May call those would be two key updates from the company both with respect to FDA and then the continued successful intervention.

Within that 22 or three in an all oral regimen.

Yeah, that's great. Thank you for the update.

Thank you. Your next question today is coming from Greg Fraser from Truest. Your line is that life.

Good morning, Thanks for taking the questions.

I got on a bit late I apologize. If this was asked already but on 'twenty, two or three or a theoretical upside scenario in terms of filing timing in which you are able to submit the NDA prior to completing cohort five what do you envision potentially supporting an early submission interim results to hit a certain threshold I realize this will depend on you just said.

But I'm curious.

How youre thinking about an upside scenario for timing and then just a quick one on R&D and SG&A spend and how do we think about spend in 2022, and then specifically for cohort five how.

How should we think about the costs for that cohort cohort and how much does that cost would you expect the NIH to cover thank you.

Sure So Greg I'll take those in and thanks for the question. So in terms of upside timing, there's two different variables to that right because some of the upside in timing will also be fda's willingness to allow us to file an NDA with shorter stability on registration batches. So that's already assuming that FDA is comfortable.

With a six months stability, which is there is precedent for that but that would be sort of the first opportunity for an earlier filing and then in terms of.

Because it's open label and because we can have more frequent interactions with FDA because of our our regulatory status in some of the the other designations that Matt 20th two or three <unk>. There is that opportunity to go in there if we're seeing consistent.

Results through let's say the first half to 75% there may be an opportunity to go in that will be one of the things that we talk about it with FDA not only in April , but probably more as cohort five starts to get drawn off I still think it's optimistic to think that we'd be able to file an.

An NDA in late 2023, primarily because of the CMC side, but if the data continue to be as compelling as they have been in cohort two and now what we're seeing with cohort four remembering the F D. A.

Was evaluating this drug or we were discussing the data in December we didn't have the benefit of any of the data from cohort four which is important because it eliminates that question up.

What impact does IV Amphotericin had for example, and then cohort four you get to answer that question and so far.

It's not that all of the results we're seeing during the induction phase.

<unk> <unk> 22, a three arm at 22 or three years alone.

And the patients as I mentioned during the call those on that 22 or three are all doing well and nearly all of them if achieved sterility. The only ones. We don't know are the ones that havent finished the induction period yet so.

Those will be important elements to discuss with FDA. So that that's very important so there could be an opportunity to move NDA filing up a little bit in 2023, but we have to see how those two key inputs sort of go over.

The course of this year in terms of the cost of cohort five.

We believe that that will be covered.

I I by NIH the that process is already in place Dr. <unk> is working with the NIH on that obviously, the NIH has been a great partner to mid teens and to this platform and so we would expect that support to be there again metastasis contribution historically has been.

The cost around manufacturing and supply of the drug some study monitoring and things like that.

But we do believe that regardless of the ultimate size of cohort five that NIH is will be willing to step up in that and that's a big deal for us and so we're counting on that cash flow forecast takes that into account, but we wouldn't do that unless we had a high degree of confidence.

And obviously, David Boleware also has a very very good relationship with the NIH.

And so that's sort of how we're thinking about 'twenty two or three I missed one question in there Greg, though remind me of what the third point was.

And just how to think about R&D and R&D.

Okay.

Yeah, I mean, obviously, we're continuing to invest in the platform, but we're doing so in a disciplined way, adding head count.

<unk> has increased sorted the R&D spend.

Modestly over the period, we don't expect huge increases and any increases or any additional investment. We're also we believe you're going to have the opportunity to offset those with some non dilutive capital. So we're not evaluating any sort of capital raise at the at the moment.

We're generally comfortable with our cash position taking into account some of the catalysts and milestones. We have ahead, but for 2022, it's gonna look consistent with.

With what we've spent I would say over the last six months.

Internally here as we've shifted away for from the DSO and that would continue sort of on a straight line through 2023 as well.

Great. Thank you so much.

Thank you.

Thank you we reached end of our question and answer session I'd like to turn the floor back over to management for any further or closing comments.

Great. Thanks, Kevin and thank you all for joining US today. We appreciate your continued interest in <unk> and the team here really looks forward to providing you with updates on our future progress have a great day.

Thank you that does conclude today's teleconference and webcast you may disconnect. Your lines at this time and have a wonderful day, we thank you for your participation today.