Q2 2021 Matinas BioPharma Holdings Inc Earnings Call
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Welcome to the mid teens Biopharma second quarter 2021 results conference call.
At this time all participants are in a listen only mode.
The answer session will follow the formal presentation. As a reminder, this conference is being recorded I would now like to turn the conference over to Peter Basel Investor Relations Representative for Machines' of Biopharma you may begin.
Thank you Hector good morning, everyone and thank you for joining the <unk> Biopharma second quarter 2021 results Conference call. Early this morning, we issued a press release of our financial results along with business updates. The release is available on the <unk> Biopharma website under the investors section speaker.
Speaking on today's call will be Jerry <unk>, Chief Executive Officer, and Keith Kucinski, Chief Financial Officer. We also have Dr. Terry Ferguson, Chief Medical Officer, Dr. Terry Mcevoy, Chief Development Officer, and Dr. Raphael Mannino, Chief Scientific officer, who will be available to answer questions during our Q&A session.
At this time I would like to remind our listeners that remarks made during this call may state management's intentions hopes beliefs expectations or projections of.
For the future. These are forward looking statements that involve risks and uncertainties forward looking statements on this call are made pursuant to the safe Harbor statement Safe Harbor provisions of Federal Securities laws. These forward looking statements are based on the <unk> Biopharma <unk> current expectations and actual results could differ materially as a result, you should not place undue reliance on any forward.
Looking statements some of the factors that could cause actual results to differ materially from those contemplated by such forward looking statements are discussed in the periodic reports <unk> Biopharma files with the SEC. These documents are available in the investors section of the company's website and on the SEC's website, an archive of this call will be posted to the company's website.
Also in the investors section following the company's prepared remarks, we will open the call for a question and answer session I will now turn the call over to Jerry.
Thank you Peter Good morning, everyone and thank you for taking the time to join US today as we review our 2021 second quarter financial results and provide an important business update.
Throughout 2021, we have made meaningful progress in moving our lipid nano crystal or LNC platform delivery technology forward with our own drug candidates as well as with promising collaborations with some of the world's leading pharmaceutical companies.
Our decision to focus our internal resources and expertise exclusively on our LNC platform has yielded significant results and has our company poised for what we believe could be several breakthrough milestones in the coming months.
We understand that efficient and safe intracellular delivery of molecules continues to be 1 of the greatest challenges in drug development today, especially with innovative medicines and we believe that our LNC platform has the.
Potential to become a new paradigm changing standard for safe and effective interest cellular drug delivery.
A key aspect of our transition to a primary focus on our LNG platform was helping investors and potential partners to better understand our technology and the broad capabilities of this potentially disruptive platform.
During the second quarter, we were very excited to have the opportunity to provide an in depth look on our LNC platform at our successful R&D day in June.
During our presentation, we describe the unique characteristics of our technology and how our phosphate title serine based lipid nano crystal fused with activated cells to effectively and safely deliver a variety of potential therapeutic cargos and the non immunogenic manner.
Both of the assembly of and the delivery from our Lncs involved membrane fusion with the unique mechanism of action, which clearly differentiate our technology from both the lipid nanoparticles and viral vectors and.
In addition to providing details on our ongoing clinical programs. We were also able to share of the preclinical data demonstrating how this novel technology can potentially provide critical solutions for both small molecules and larger more complex molecules such as messenger RNA DNA plasmids antiques.
Oligonucleotides and vaccines.
An archived copy of this webcast remains available on our website and we would encourage anyone interested in learning more about our LNC platform to take advantage of the opportunity to review of this important content.
The response to our R&D day has been extremely positive and has resulted in a number of new inquiries.
Setting the stage for potential additional exciting external collaborations.
Some of which are already under discussion.
Since the R&D day in June there have been several other key developments with our LNC platform first of all we are thrilled that cohort 2 of the enact study of Matt 22 of 3 in patients with HIV suffering from Cryptococcal meningitis is now completely enrolled the.
The day SMB meeting to review the available safety and efficacy data from the second cohort is expected in September and we plan to announce and discuss these data together with additional commentary from Dr. David Boleware principal investigator of an act following the D S and be evaluation of.
Although it is somewhat unusual to disclose and discuss data in the middle of an ongoing clinical study the strategic design of the enact trial along with the significant unmet medical need in this vulnerable patient population provides of near term opportunity to further validate the LNC platform.
We also believe that these data can highlight the unique ability of our LNC delivery technology to bolt facilitate oral bioavailability and deliver molecules of effectively across the blood brain barrier.
In cohort 2 the duration of oral mat 22 of 3 in the induction phase was increased to 12 days followed by consolidation treatment with map 22 of 3 for for additional weeks in 40 active patients.
We plan to review and discuss the data from cohorts, 1 and 2 in September and we believe the results from the step down the regimen could represent a significant breakthrough in the treatment of patients with Cryptococcal meningitis.
As we look forward to the results a couple of key points are important to keep it not in mind.
Number 1.
Survival is the Fda's endpoint of interest for these patients.
We know that currently survival is only 70% in patients with the best standard of care.
And unfortunately, as low as 30% in areas without the highest levels of care.
Any improvement over the historical best 70% survival rate would be of significant development given the oral nature of our therapy.
Number 2 we would like to see the data demonstrate early fungus idle activity, which is the rate of clearance of eased from the cerebral spinal fluid.
And we would like to see that this early fungicide all activity is greater than 0.2 and.
In early bundle cycle activity of greater than 0.2 is recognized as being associated with improved survival. According to published literature.
Number 3 we would like to see little or no evidence of rebound or breakthrough of Cryptococcal meningitis infections. During the entirety of the map 22 of 3 period.
Treatment period, this would convincingly demonstrate that our therapy is working effectively.
Number 4 we would like to see all patients achieve sterility during either the induction or consolidation phases, while taking that 22 of 3 sterility is defined as the absence of actively replicating from guy in the cerebral spinal fluid.
This would again show that Matt 22 of 3 can be an effect of step down treatment in this patient population.
And finally 5.
We would also expect to see an overall positive safety profile for <unk> 22, or 3 in these patients.
With no evidence of the renal toxicity historically seen with IV amphotericin no other major safety signals and no use of limiting tolerability issues.
This combination of results would provide great support for Matt 22 of 3 as of safe and effective step down therapy in these patients and would represent a significant validation for our LNC platform.
We believe that these data from the first half of enact could also support a key regulatory interactions later in 2021, where we plan to discuss the potential for the approval of map 22 of 3 of step down therapy under 1 or more accelerated regulatory pathways for important anti infective medicines that address.
Significant unmet medical needs in small or vulnerable patient populations.
An indication for step down therapy in patients with Cryptococcal meningitis would mean that physicians would be free to prescribe <unk> 22 of 3 for use following a short duration of IV amphotericin in the beginning of the induction phase in order to complete the 2 week course of induction therapy, and then continue with map 22.
<unk> III throughout the additional for wheat consolidation or maintenance phase.
This indication in and of itself would represent a significant commercial opportunity for Matt 22 of 3 and following completion of an act we plan to approach FDA seeking advice regarding regulatory approval for an additional all oral amphotericin treatment regimen in these patients.
Overall, we believe that Matt 22 of 3 could significantly increase the use of amphotericin due to its efficacy significantly improved safety profile.
And by avoiding the patient need for extended hospitalization and associated costs.
The second key development with our LNC platform is the progress we have made with Matt 20, 501, our LNC oral formulation of the broad spectrum of aminoglycoside antibiotic drug amikacin.
In collaboration with the cystic fibrosis Foundation during the second quarter of 2021, we advanced important preclinical studies positive.
Positive feedback we received from the FDA in July on these ongoing preclinical studies as well as our overall plan for development of about 20, 501 position us to initiate a phase 1 single ascending dose pharmacokinetic study of about 25 of 1 in healthy volunteers in the fourth quarter of 2021.
Our goal with Matt twenty-five of wanted to develop the first oral aminoglycoside, which could completely transformed the use of this important class of drugs.
Taking advantage of our LNG platform's ability to effectively deliver molecules intracellular early we believe that Matt twenty-five of one's ability to orally deliver high levels of amikacin directly to the lung without use of limiting toxicity clearly distinguishes it from all other available therapies.
And could provide an important solution for patients and physicians.
Perhaps the most of the anticipated and exciting developments since our last quarterly update involves our work and successfully formulating and developing a potential oral formulation of Gilead noted COVID-19, antiviral drug Ram deaths of here.
Today, we are extremely pleased to report that the company and the National Institute of allergy and infectious disease within NIH have successfully completed the planned in vitro studies of various LNG formulations of our in depth of here.
<unk> room desk severe formulations were tested for antiviral activity against the Washington, Sars Covid, 2 strain and Keiko 2 sales, which are highly permissive for Sars cov 2 infections.
And formulated Rem desk of your active was tested as the comparison.
Following close review of these data with NII D. The LNP formulations tested in this model demonstrated meaningful anti viral activity as compared to free room desk severe while also showing a favorable toxicity profile.
The impressive nature of these data Nia ideas now preparing to initiate an in vivo efficacy study of the most potent LNC room deaths of your formulation as soon as possible. We expect additional data to be available in the fourth quarter of 2021.
These in vitro data represent a big step forward and not only showing that our LNC platform can be potentially effectively used to create an oral formulation number of deaths of ear, but are also an important proof of principle for the interest cellular delivery of antiviral medicines more broadly.
As we face the ongoing reality of the continued fight against COVID-19, the need for effective well tolerated antiviral drugs that can be given to patients at high risk for severe disease at early stages of illness remains extremely high and oral version of Gilead for Endesa beer has the potential to be an <unk>.
Morton option for physicians around the world and the fight against COVID-19, since an oral Ram debt severe would permit administration earlier in the disease course, as well as potential for prophylactic use should clinical studies support such an approach.
We are very happy with our progress to date on this project and look forward to with great anticipation to the NIH is planned in vivo study in the near term for.
Finally regarding lift you so our potential best in class prescription Omega 3 therapy, we continue to work through a robust and global partnership process and look forward to providing an update later in 2021.
I will now turn the call over to our CFO, Keith Kosinski, who will discuss our financial results.
Thanks, Jerry and good morning, everyone.
Turning now to our financial results.
Cash cash equivalents and marketable securities at June 30 of 2021 were approximately $59.8 million compared to $58.7 million at December 31, 2020.
Research and development expenses were approximately $2.5 million on the second quarter of 2021 compared to approximately $3.4 million in the same quarter of 2020.
The decrease was due primarily to the completion of the enhance study of lip DSO in January 2021.
General and administrative expenses were approximately $2.3 million in the second quarter of 2021, essentially unchanged compared to the $2.4 million in the same period in 2020.
The company reported a net loss attributable to common shareholders of approximately $5 million or 2 cents per basic and diluted share compared to a net loss attributable to common shareholders of $5.8 million or a net loss of <unk> <unk> per share both basic and diluted for the same.
<unk> in 2020.
The decrease was due primarily to the aforementioned reduction in research and development expenses.
Based on current projections, we believe that cash on hand is sufficient to fund operations into 2024.
I will now turn the call back over to Gerry.
Thank you Keith.
In summary, the next few months promise to be a very exciting time for our company with the enact data set to be released in September Matt 25 of 1 entering of human clinical trial in the fourth quarter and additional efficacy data expected with elements of the LNG room deaths of here. We believe we have significant near term milestones for the company.
<unk> and our LNC platform technology. These catalysts when combined with ongoing work with Genentech on others and expanding the use of this new paradigm changing technology.
Should position the tina's for very big things in the coming quarters.
I'd like to recognize and thank the tremendous team we have here for keeping its collective INR goals and objectives and continuing to execute especially in completing enrollment of the all important second cohort of patients and then act.
With that we have reached the conclusion of our prepared remarks, and I will turn the call over to the operator for a question and answer session.
Thank you at this time well be conducting a question and answer session. If you'd like to ask a question. Please press star 1 on your telephone keypad.
The information tone will indicate your line is in the question queue. You May press star 2 if you'd like to remove your question from the queue for participants using speaker equipment. It may be necessary to pick up of your handset before pressing the star keys, 1 moment, please while we pull for questions.
Your first question comes from the line of of Bert Hazlett with <unk>. Please proceed with your question.
Yes, Jerry Thank you for the comprehensive update and congratulations on the progress with 22 of 3 we look for that data of upcoming.
With regard to the other programs 25 of 1 and then the depth.
To your first question the 25 of the 1 do you expect that to have a development course.
Analogous to the 2002 of 3.
Multiple arms multiple treatment regimen for <unk>.
In the clinic of course or can that be a bit more straightforward in terms of initial studies.
Talking about efficacy studies, and then with regard to ramp that severe when do you think we actually might get the initial human data from that program. Thanks very much.
Great. Thanks, Thanks for the questions. So on 25 of 1 of in some ways. It is similar to 22 of 3 this is a 505 be too like the process for dealing with infectious disease drugs. So there is the opportunity.
For certainly certain streamlined regulatory and development processes, but in some ways, it's different because of the indications we're going after in terms of the treatment of MTM or non tuberculosis mycobacteria Mycobacterium what you will see from us in phase II that was likely a set up very similar.
What was utilized with the enact trial in terms of having a pharmacokinetic lead into phase III <unk> study followed by a <unk> study efficacy study in patients with the MTM, we would expect that program depending on how everything progresses over the next year.
To potentially get started at the end of next year. Further discussion is required with FDA to to formally sort of put together what that phase II program would look like obviously, we've spent a lot of time with kols in this area.
Noted experts in MTM, both inside and outside NIH, including Chuck Daily out in Colorado. So.
More there we do think though that this is because of the unmet need here and because of the way we approach development through 5 years of fiber to and other regulatory channels that can be streamlined similar to 'twenty..2 of 3 and then you will also likely see us stack indications with 20.501, so because the amikacin.
On the so broad spectrum, obviously, our initial target is the treatment of of MTM.
But we will also go after.
Different types of more acute bacterial infections, namely gram negative.
And so the team is working to outline those development plans.
Which would start probably soon after we would get into the phase 2 efficacy studies and the N T M. But the first oral aminoglycoside represents a large opportunity for that reason amikacin is used sparingly today, even though it's very efficacious because of its toxicity profile.
Both nephrotoxicity and OTO toxicity, and we think due to the nature of our delivery program. We can eliminate a lot of those safety concerns. So we have a very interested division of infectious disease of FDA very engaged the more we are in front of them with 22 of 3 it helps them of 25 of 1 that was noted.
During the interaction we just had had in July. So you have an agency that's getting more comfortable with the pharmacokinetic profile of our LNC program. How it works how it gets inside sales, how we are able to take drug out of the blood all of that helps and we expect.
Could help us accelerate the development of that drug on the room desk of beer side, we know that theres a lot of attention from NIH and Gilead on this this is of high priority, especially at the NIH. We also know that there's a lot of drugs in the queue there.
And so we're fortunate enough because of our relationships at the NIH to be moved to the front of that Q.
Which is why we're so excited to start the the in vivo work.
Expeditiously because there are a lot of trucks waiting for those slots, but the NIH has slotted US ahead of a lot of the other once we get through the the in vivo work, we expect those data in the fourth quarter, we'll evaluate those will discuss those both with the NIH.
And gilead and what's been communicated to us although not not formally as there is an interest then to go immediately into those human clinical trials timings of little bit of a guess at this point I would expect though given the the sense of urgency here and the need that that's something we could see in the first half of <unk>.
2022.
Great. Thanks, very much look forward to the progress. Thank you.
Thanks Bert.
Your next question comes from the line of Greg of Frasier with Truest. Please proceed with your question.
1 of you guys. Thanks for taking the questions and congrats on the progress in the quarter.
On 22 of 3 can you give us some insight into the accelerated approval pathway that you believe may be feasible on the additional clinical work that you might have to do to support a stepped on indication.
Sure. Thanks, Greg so.
There is there are more than 1 avenue available to US we know that al pad is available. That's the limited population pathway for anti infective medicines for small patient populations. We also know that we're dealing within the orphan patient population here there are some advantages and some disadvantages too.
Each or all of those pathways and so as we evaluate sort of our desire to advance this towards approval as quickly as possible. We're also very cognizant of the sort of label that we would want and so there are some elements to consider as we move forward either under L pad are orphan or <unk>.
Some sort of combination of both what we do know.
Is that the need here will.
We will be what we believe dictates timeline.
So in our discussions with the FDA to date, there has been great receptivity to sort of advancing this as quickly as possible. We think we were giving great leeway even in the design of an act to sort of move as quickly as we did into this large and vulnerable of patient patent largest study in vulnerable patient population.
But it remains to be seen Greg until we take the data from cohort 2 and sit down with the FDA likely in the fourth quarter of this year as to what exactly will be required.
We know that these deadly invasive fungal infection studies are very hard to recruit when in fact it took.
Bads of lay of more than for years to recruit 40 patients into a new core trial, we've recruited more than 56 patients into cohort 2 alone in under a year.
So we're moving this very very quickly we think the number of patients here.
Is large enough where the FDA is going to have a good amount of data to evaluate both safety and efficacy a lot of care was taken in the design of this trial.
The study to give us an appropriate control of IV amphotericin actually an increase in the standard of care that would be normally be available in Uganda. So it remains to be seen how we engage with the FDA our position is going to going to be for step down therapy that we've shown enough that that we believe will be.
Sorted by the principal investigator and.
And the other kols, but it will depend in large part of <unk>.
On the data from cohort 2 and how that is evaluated certainly first by the day SMB and then when we sit down with the FDA at the end of the year.
Got it that's very helpful. On the on the data you walk through of the key elements that you want to see which is very helpful, which of those will the data from cohorts, 1 and 2 address I'm, assuming it maybe too early to have survival data, but if you could just expand on what we may see next month that would be helpful. Thank you, yeah, I think youre going to see all of it.
Survival is a key measure.
In both cohort 1 and cohort 2 it is a little bit easier to discern the efficacy in cohort 2.
Your second question for the 25 of 1 single ascending dose study. This study will be 8 and ascending dose study and which we will be testing 5 dose 5 dose levels.
Of our oral M of case and product compared with the placebo control, which is typically what's done in and S. A D study, we will not be including IBM and.
IV M of case and in this trial. However, the expectation will be of course and phase 2 to have IV and the case and as a comparator the.
The cohort design will have evaluation of safety at each incremental dose escalation step that will be a sponsor review and a review by the medical monitor at the CRO, who will be conducting the study at the phase 1 unit and so careful safety monitoring.
Of both potential Reno.
Signals as well as auto toxicity, which we have.
We have already conducted and S. A day study with the prior formulation of our med twenty-five old 1 and which we saw no evidence of any safety issue.
Issues whatsoever with our our prior formulation. So we we will take a safe cautious approach and and escalating doses and anticipate that we will be able to quickly thereafter move into our phase 2 trials at the end of next year.
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Okay.
Okay.
As a reminder, if you'd like to ask a question. Please press star 1 on your telephone keypad as a reminder, if you'd like to ask the question. Please press star 1 on your telephone keypad 1 moment. Please while we poll for more questions.
Your next question comes from the line of.
Robert Leboyer with Noble capital. Please proceed with your question.
Good morning, I had some questions on the on that trial, which you answered in your previous answers to the questions and thanks for all the detail of the only thing for that.
It didn't catch was the number of patients in the second cohort and.
And after you reported the data in September.
Are your expectations for for the milestones going forward and the program.
Robert Thanks for your questions. It's good to hear your voice and yet.
On the enact trial and cohort 2 of the.
Total cohorts 56 patients so the 40 patients on the active and 16 control and.
That follows up on cohort, 1 where you had 10 patients on active and for on control. So it's a healthy number of patients that will give you a substantial significant amount of the valuable data. So we're excited about that.
And from there.
And dependent on the discussion with FDA and what design changes, we make may make to the second half of the net.
2 to perhaps put us and an even more favorable position for the overall approval of map 22 of 3 the second half of.
Matt 22 of 3 is designed really for that all oral amphotericin indication. So cohort 3 is again set up as of cohort.
Or is the lead into cohort for that would get underway.
And this year cohort III in terms of following the D. SNB review of the data they would again make a determination in terms of progression from cohort 2 cohort 3 we would expect that that would get underway at some point in 2021.
And that will again be 14 patients so.
Given the success, we have had and enrollment even despite some shutdowns and Uganda with Covid.
Earlier this year, we would expect that cohort 3 to be finished pretty quickly that would again be followed by the DSP evaluation of those data and then we would go into cohort for.
In 2022, which would be an all oral amphotericin arm, we may make some adjustments to that.
To.
Put us and even better position.
With the FDA, but that would be some some things we discussed with the agency in the fourth quarter of this year, but enact we would expect to be completed during 2022 absent any significant changes or discussions with FDA.
Okay.
Great. Thank you very much and congratulations on all of this progress.
Thanks Robert.
Ladies and gentlemen, we have reached the end of the question and answer session and I'd like to turn the call back to management for closing remarks.
Thank you Hector and thank you all for joining US today. We appreciate your continued interest and <unk> and the team here looks forward to providing you with updates on our future progress have a great day.
This concludes today's conference you may disconnect your lines at this time. Thank you all for your participation.
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And the.
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