Q3 2021 Matinas BioPharma Holdings Inc Earnings Call
[music].
Greetings and welcome to <unk> Biopharma third quarter 2021 results conference call.
At this time all participants are in a listen only mode. A question and answer session will follow the formal presentation.
If anyone should require operator assistance during the conference. Please press star zero on your telephone keypad.
As a reminder, this conference is being recorded it is now my pleasure to introduce your host Peter <unk> Investor Relations representative for <unk> Biopharma. Thank you you may begin.
Doug Good morning, everyone and thank you for joining the <unk> Biopharma third quarter 2021 results conference call earlier. This morning, we issued a press release with our financial results along with business updates. The releases are available on the mid teens Biopharma website under the investors section speaking on today's call will be Jerry Jabbour, Chief Executive Officer.
Dr. Terri <unk>, Chief Development Officer, and Dr. Raphael Mannino, Chief Scientific Officer, We also have Dr. Terry Ferguson, Chief Medical Officer, and Keith Kucinski, Chief Financial Officer 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 intention hope.
<unk> beliefs expectations or projections of the future. These forward looking statements.
And uncertainties forward looking statements on this call are made pursuant to the safe Harbor provisions of Federal Securities laws. These forward looking statements are based on the <unk> Biopharma is current expectations and actual results may differ materially as a result, you should not place undue reliance on any forward looking statements.
One 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 mid teens Biopharma files with the Securities and Exchange Commission. 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.
During the company's prepared remarks, we will open the call for a question and answer session I'll now turn the call over to chip.
Thank you Peter Good morning, everyone and thank you for taking the time to join US today as we review our 2021 third quarter financial results and provide a business update.
For <unk> the third quarter of 2021 was transformational in September we announced efficacy and safety data from the first half of our enact trial of map 22 or three in patients with Cryptococcal meningitis as outlined on that call that $22 three performed impressively in cohort two exceeding the prior.
Mary endpoint threshold, while at the same time, demonstrating an impressive overall safety profile supporting the longer term use of oral mat 22, or three up to six weeks and eliciting considerable enthusiasm from Dr. David Boleware the principal investigator these.
These are incredibly impressive data and provide a great deal of optimism as we continue into the second half of the enact trial our goal in cohorts three and four is to demonstrate that Matt 22, or three can be utilized not only as a step down from IV amphotericin for induction therapy and for extended use in consolidation.
But also as part of an all oral regimen for the treatment of Cryptococcal meningitis.
Thus, our oral formulation of amphotericin could significantly limit and even potentially eliminate altogether the need for an IV infusion of amphotericin b.
This when combined with eventual potential additional indications for the treatment of other invasive fungal infections.
<unk> to those contained in the Amazon label and the potential extension of treatment to include prevention and highly vulnerable immunocompromised patients could position, Matt 22, or three to become one of the most important and commercially successful options for the treatment and prevention of invasive fungal disease Dr. <unk>.
<unk> will go into more detail on our mat 22, or three program, including our upcoming FDA meeting as part of her remarks. This morning.
We intentionally chose to use the word transformational to describe this past quarter because following the enact announcement external interest in learning more about both our LNC products and are potentially game changing LNC technology platform have increased significantly.
We are now becoming recognized as a true platform company.
Our shift and focus to our broader LNC platform programs has been validated with the exciting progress in an act and we believe that we are now positioned to potentially become a major player in the increasingly important world of intracellular delivery.
Looking beyond that 22% <unk> as a whole we continue to make significant progress advancing our pipeline, including haven't recently initiated a phase one single ascending dose study with <unk> 501, our LNC oral formulation of the broad spectrum of mean.
<unk> glycoside antibiotic drug Amikacin now our second clinical stage LNC asset.
For about 20 501, our goal is to develop the first oral aminoglycoside, which could completely transform the use of this important class of drugs as mentioned, we have recently dose healthy volunteers in a phase one single ascending dose PK study following positive feedback we received from FDA earlier this.
Year on ongoing preclinical studies conducted in collaboration with the cystic fibrosis Foundation, we expect to complete enrollment of the phase one study in the first quarter of 2022 and have data available in the second quarter of 2022.
We believe that Matt 2500 one's ability to orally deliver high levels of amikacin directly to the lung without use limiting toxicity.
Clearly distinguishes it from all other available therapies and could provide an important solution for patients and physicians.
<unk> and its inhaled amikacin product our case continues to garner a high valuation by Wall Street, Despite a very narrow label and a boxed warning.
And oral and safe, Matt 20, 501 would be positioned to capture virtually the entire MTM market and unlike an inhaled therapy.
Oral broad spectrum aminoglycoside like Matt 20, 501 could potentially be used to treat a variety of acute bacterial infections like gram negative infections.
As we continue to progress, Matt 25, or one in the clinic, we expect an increasing amount of attention and value creation from this program.
In addition to moving our LNC platform forward with our own drug candidates, we continue to make progress advancing our promising collaborations with leading pharmaceutical companies.
For example, as we shared on our last quarterly update call in in vivo efficacy study of LNG ramp Dessa beer being conducted in collaboration with the National Institute of allergy and infectious diseases with cooperation from Gilead Sciences has commenced at the University of North Carolina, We anticipate data from this study before.
The end of the year, Dr. Mannino will discuss more about this important project a bit later.
But from a teen is a demonstration of efficient effective and safe delivery of an anti viral pro drug in this well established and highly predictive preclinical model would bode extremely well for the use of our LNG platform across the antiviral therapeutic class.
Finally, I would like to mention that we remain engaged in a global partnership process for the potential licensing uplift DSO our potential best in class prescription Omega three therapy.
Based on expressed interest to date and the number of companies still conducting due diligence. We believe that this process will extend into the first quarter of 2022 at a minimum.
I will now turn the call over to our Chief Development Officer, Dr. Terry Mcevoy, who will discuss the progress and positioning of our two clinical stage product candidates, Matt 22, or three and Matt 20 501.
Thanks, Terry and good morning, everyone.
From that 23, our oral amphotericin product, we have made significant progress.
As Jerry mentioned earlier during our last call, we announced positive data from the first two cohorts of the enact trial.
Since then we've moved onto cohort three we are ahead of schedule and I'm happy to report that as of this morning, we have fully enrolled the cohort of <unk> patients.
Before I outline the specifics of what we expect to see in cohort three I'd like to briefly walk you through the bigger picture view of our overall regulatory strategy permit 22 Dominion.
As you recall, we are working towards filing an NDA for <unk>.
Indications from that 'twenty, two or three.
First we are pursuing an indication as induction therapy for the treatment of Cryptococcal meningitis to be used with blue side is seen as a step down treatment.
Boeing just two days of IV amphotericin.
Second we are pursuing a novel indication for consolidation treatment for Cryptococcal meningitis.
We're up to a total of six weeks of treatment.
This would represent the first long term use indication for any currently available amphotericin b product.
Third and especially relevant for cohorts three and four is an indication for an all oral induction treatment for cryptococcal meningitis administered with blue cytosine potentially eliminating the need for any intravenous amphotericin b treatment.
This overall potential registration package for Cryptococcal meningitis will be discussed with the FDA next month, and we plan to share their official feedback early next year.
Importantly, the initial registration of <unk> 23 for the treatment of Cryptococcal meningitis.
Stepped down consolidation and all oral induction is only the beginning.
Leveraging the 500, <unk> pathway and orphan disease designation with the potential for breakthrough designation in Cryptococcal meningitis.
Preparing for additional discussions with the agency around the streamline development pathway for additional follow on indications, specifically prophylaxis and treatment of other severe and deadly invasive fungal infections, such as those caused by Crs.
<unk> and Nucor mycosis to name a few.
The potential recognition of <unk> 22, or three as a safe and effective treatment for Cryptococcal meningitis.
Represents gateway opportunity that we believe will allow us to bridge to other severe and invasive fungal infections with the streamline clinical data package.
Now turning back to the specifics of cohort three and enact.
Recall that cohort three is a critical pilot test of the potential efficacy of <unk> 22 or three.
Initial treatment and leading to a potential all oral regimen for Cryptococcal meningitis.
Cohort three includes a total of 14 patients 10 active Matt 22, three and four our control.
In which treatment in the test time during induction starts with our oral mat 23.
Given in combination with <unk>.
The first five days of treatment.
Patients are carefully monitored during these initial first five days of treatment.
To assess the impact of <unk>, 22, or three and Farmville clearance.
Following the initial five days of treatment with <unk> 22, or three patients are switched to IV amphotericin.
When administered with Blue side, the same for the remainder of the induction phase III data for <unk>.
During the consolidation phase in cohort three patients continue to again receive met 22 or three treatments with fluconazole through week six.
We are pleased that enrollment in cohort three is ahead of schedule and at the full cohort is now fully enrolled.
And that the patients are reported to be doing very well.
Based on the current enrollment completion, we will.
Would expect that the <unk> will convene for a review of the data and formal cohort progression decision before the end of the year.
As with cohort one progression our plan would be to announce the decision of the DSM b and the commencement of enrollment in cohort four.
There are six things that we hope to see from the induction and consolidation phases of cohort three and that are likely to be considered by the DSM b as part of this evaluation.
First a reduction in bunzl accounts within the first five days of <unk> 22, or three treatment with no increase in funnel growth, indicating potent initial antifungal activity of <unk> 23.
Second early fungicide all activity at the end of induction above the critical threshold of greater than 0.2, the primary endpoint for this trial.
Next 18 weeks survival greater than historical published rates of 70%.
Achieving sterility and all patients continuing to receive Matt 20 tool suite therapy, which is the absence of replicating fungi.
Either the induction or early consolidation phases of treatment.
Next no evidence of breakthrough or recurrent infection during that $22 three treatment.
And finally, an overall positive safety profile for <unk> 22 of <unk> III without kidney toxicity attributable to net 22 or three.
An announcement that the <unk> has approved progression to cohort four would be a strong signal that each of these objectives have been achieved.
Turning now to Matt 20, 501, our oral amikacin lipid nano crystal formulation I am pleased to share with you that we have also continued to make strong progress.
As we announced last month, we dosed our first patient in a single ascending dose trial of an optimized or amikacin formulation.
This study is being conducted in the U S and clinical results are expected in the first half of 2022.
Importantly in parallel to the ongoing clinical study we are continuing to conduct necessary toxicology studies, which were the focus of our meeting with FDA in July.
Additional longer term toxicology studies are planned for 2022 to support the longer term clinical studies and our planned phase III clinical program.
Since the treatment of MTM infections requires extended treatment courses for eradication of the infection, which can extend 18 months or even longer.
Having these long term toxicology studies completed early in the development of map 20 501.
We will also provide us with flexibility to potentially pursue other indications for Matt 20, 501 that may require longer durations of therapy.
All available data to date for <unk> 501 continue to support the favorable safety profile of our LNC platform and hold hope for the future of other programs leveraging this platform for targeted delivery of drugs to the site of active infection and inflammation.
We look forward to continuing to update you on the progress of <unk> 22, or three and Matt 20 501.
And with that I'll turn the call over to Dr. Raphael Mannino, who will be providing you an update on our ongoing work with <unk>.
Thank you Terry and good morning, everyone.
As we have announced previously Latinos Gilead and the National Institutes of health have an ongoing collaborative research program utilizing our proprietary LNC delivery platform to develop an orally bio available formulation of <unk>.
Before providing an update on the status of this project I'd like to briefly review some of the background work that has led up to this exciting collaboration.
The preclinical development programs for <unk>, 'twenty 203, our orally bio available amphotericin and that 20 501, our orally bio available amikacin. Both included a number of preclinical studies that demonstrated the efficacy of oral LNC formulated antimicrobials.
<unk> in the treatment of severe lung infections.
<unk> 22, or three has proved to be very effective in the treatment of both Canada and aspergillus lung infections in preclinical studies.
PK and PD studies in mouse models of systemic candidiasis demonstrated that when compared to parental deliberate amphotericin B mab.
<unk> hundred three demonstrated more rapid tissue penetration and more controlled drug levels over the entire course of therapy.
In addition in a systemic controlled.
In a systemic aspergillosis mouse model.
Matt <unk> hundred three provided almost complete eradication of lung infection and enhanced survival when compared to parental amphotericin b.
Similarly, Matt 20, 501 has been evaluated in a number of studies looking at the treatment of non tuberculosis mycobacteria infections in preclinical studies involving mouse models of disseminated infection milestone related infections and mouse models of cystic fibrosis.
Yes.
And all of these settings orally administered that 20 501 demonstrated efficacy that was at least as good as or even slightly better than that of the parental application.
It is important to remember that unlike other nanoparticle.
Delivery platforms the.
The LNG formulation and manufacturing process allows for significant flexibility and creating new formulations with the ability to customize parameters such as lipid to drug ratio the specifics thousands excipient and buffers used as well as sole concentrations in ph.
In essence, we can design, an LLC and caps and capsulation formulation around unique physical chemical properties of each unique specific molecule can be delivered and further customize it to optimize delivery in this setting where it is going to be used.
We have also noted that unlike other therapeutic areas, where there can be a large disconnect between results in cell culture and results in vivo.
When evaluating antimicrobials there tends to be a strong correlation between LNG formulations that are efficacious in in vitro cell based models and formulations that demonstrate oral efficacy and the subsequent animal proof of concept studies.
Coming back to the oral LNG Rem density of program. We have previously reported that we have evaluated a number of different oral LNG Rem density of formulations in in vitro models through the NIH contract system.
Having identified several promising candidates based on this in vitro work, we have now moved to the next stage of in vivo testing.
In addition to the strong support of our previous work for this rapid effective oral delivery of antimicrobials to treat pulmonary and CNS infections.
With this study there was another potentially important consideration with the industrial namely the fact that <unk> is a prodrug and must be processed by specific intracellular enzymes in order to form the active antiviral drug.
While this is our first LNG formulation of a prodrug, we continue to be very optimistic about the improved intracellular delivery capabilities of our LNG platform and how it could provide an orally bio available formulation of Linzess appear.
Our initial in vivo LNG Linzess preclinical study is ongoing.
And we anticipate having results before the end of the year.
Following discussions with the NIH and Gilead and receiving their necessary by an approval we would hope to make an announcement concerning these results.
While positive in vivo results should have important implications to an expanded use profile for industrial here.
There are even broader potential implications of positive data with an LNG antiviral formulation and a well established animal model of a serious viral infection.
Positive results would first confirm the broad utility of LNG delivery across the entire spectrum of anti Infectives Antifungals antibiotics and now antivirals.
But beyond that there are additional implications for the potential intracellular delivery of a wide variety of compounds more complex than the simple small molecules.
Unlike fungi and bacteria, which can replicate both inside and outside of sales.
Viruses can only replicate inside ourselves.
Accordingly for antiviral drugs that work by inhibiting viral replication, including small molecules and oligonucleotides efficient and safe intracellular delivery is essential to their ultimate clinical success.
Work with Ron <unk> is just the first step.
A unique cell targeting nontoxic and non immunogenic intracellular delivery mechanisms of the LNC platform to make <unk>, an ideal formulation and delivery option for number of antiviral agents.
We expect to learn a lot more from this study at UNC and then be able to potentially apply these learnings to other areas, including Antivirals.
Nucleotides and complex nucleic acid polymers, like messenger, RNA, and even potentially gene therapy.
I will now turn the call back over to Gerry who will discuss our financial results and provide some final thoughts.
Thanks Rafael.
To briefly address our financial results cash cash equivalents in marketable securities at September 32021 were approximately $53 8 million compared to $58 7 million at December 31, 2020.
<unk> research and development expenses were approximately $4 6 million in the third quarter of 2021 compared to approximately $3 3 million in the same quarter of 2020.
General and administrative expenses were approximately $2 3 million in the third quarter of 2021, essentially unchanged compared to $2 4 million in the same period in 2020.
The company reported a net loss attributable to common shareholders of approximately $6 8 million or <unk> <unk> per basic and diluted share compared to a net loss attributable to common shareholders of $5 7 million or a net loss of <unk> <unk> per.
Per share basic and diluted for the same period in 2020 based on current projections. We continue to believe that cash on hand is sufficient to fund operations into 2024.
To summarize and conclude our prepared remarks I am extremely pleased with how we have been able to progress our LNC platform. During 2021, and we now have two product candidates in clinical development.
Both in areas of significant medical need with large potential markets, both domestically and globally.
Infectious disease continues to pose a significant threat to human life, and we believe that the <unk> platform and our targeted products can become a valuable solution for patients.
As we head towards the end of this year, we expect the <unk> evaluation and progression assessment for about $22, three which would be a very good sign that our drug has great potential for oral induction therapy.
We also are looking forward to our meeting with FDA in December to evaluate all available data and discuss the potential early approval pathway for <unk> 22, or three a step down therapy.
We continue to advance <unk> 501, with a profile of our drug looks to be extremely promising and potentially a much better alternative to a drug that today commands a $3 5 billion dollar plus market cap.
We look forward to completing enrollment in announcing data from our phase one single ascending dose study with <unk> 501 in the first half of 2022.
The in vivo efficacy study of LNG room that severe currently ongoing with our partners at the NIH and Gilead is expected to yield data by the end of this year and could provide further evidence that our LNC technology can become a major player in the antiviral space ultimately, we believe our technology can be differentiated as in.
Efficient effective and safe intracellular delivery vehicle, which holds promise for the delivery of a variety of molecules. We continue to evaluate opportunities to capitalize on what we believe is the enormous potential of our LNC platform and its unique ability to solve the oral administration and interest.
Cellular delivery challenges presented by vaccines and complex nucleic acid polymers, such as messenger RNA. We believe our future is extremely bright and we look forward to continuing to update you on our progress with that I will turn the call back over to the operator for a question and answer session.
Thank you, ladies and gentlemen, we will now be conducting a question and answer session. If you'd like to ask a question you May 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 would like to remove your question from the Q4 participants using speaker equipment. It may be.
Yeah.
You may need to pick up your handset before pressing the star key our first question comes from the line of my Young men Tani with B Riley Securities. Please proceed with your question.
Hi, this is.
<unk> and <unk>.
Our questions.
<unk>.
Youre welcome.
One of them for hours the.
Gram deciliter after the.
And the unions is that do you think there is a possibility of that.
The formulation could improve the drug efficacy so that potentially reduce the viral load. Thank you.
Hi, Thank you very much for your question and it's a good one because people are very curious about our <unk> program and what steps lie beyond.
In in vivo efficacy study with the NIH and Gilead in a lot of attention has been given recently.
To the development and advancement of oral Antivirals, and we believe we're positioned to become a meaningful.
Member of that group, we have to wait to see what the exact data look like from this in vivo study in order to then aggressively planned to go into a phase one study based upon our knowledge of how our technology works and our unique ability to get these molecules delivered in an efficient manner intra cellular Lee of <unk>.
We believe there is a possibility that we could improve upon the efficacy already demonstrated by <unk> in clinical trials, but that's going to be data dependent what we have seen in terms of our ability to deliver other antimicrobials in a similar fashion in terms of encapsulating them in our LNC platform and <unk>.
<unk> them to sites of infection, we have seen that ability to improve upon efficacy and doing that at the same time, we are <unk>.
<unk> improving the safety profile of the drug so our expectation is that these data to be generated at UNC are very very important for gilead very very important for <unk> and the NIH has always been the driver of this program and we expect that depending on the evaluation of those.
Data, we would then very aggressively move forward into the clinic into human studies in 2022, but we have to wait to see what the data are.
Thank you for the household color and another question.
Jason to the already delivered.
Wed like material.
The delivery.
This was a law given the challenges in the field with other small molecule my reluctance on the army.
For example.
Will it be possible to deliver Robyn.
Ron that's a weird weird.
Long delivery.
<unk> formulation. Thank you.
Sure. Thank you for the question and then I'll ask Dr. Mannino to address this one.
Thank you for the question one of the things we didn't touch upon today, but is another very positive aspect of the LNC technology is just to make it short LNG, our anhydrous theres no water within the inside layers of this that allows us not only to give suspensions, which are stable, but also.
To make dry powders, including lie optimization fluid bed, drawing and spray drying, we already have been able to make formulations of other types of molecules, including fluorescent dyes within the LNC and deliver them by inhalation not only mouse, but mice, but also in.
Dog models, and we were able to then see material in the lung.
Coming out that was taken up by cells. In addition, we were able to do a psi RNA study of.
Sorry, RNA in <unk> six in a mouse model of influenza infection and with a single dose delivered intra nasally, we were able to knockdown the viral load three days later by 200 fold when in this model about 10 fold is considered therapeutic so we.
Do have.
Early data, suggesting that the ability to deliver directly into the lung is compatible with the LNC technology.
Thank you that's all we have thank you.
Our next question comes from the line of Bert Hazlett with <unk>. Please proceed with your question.
Thank you congratulations on the progress. Thank you for taking the question I've got one or two.
Specific ones and then maybe a bigger picture one Gerry as well.
Can you just characterize the Lee.
The gilead relationship in the room that severe studies.
Is there some.
Further.
Development contemplated with Gilead and just again general characterization of the relationship at this point.
Yeah. Thanks, Mark. So this is a relationship but that was directly driven by the NIH. They essentially bought true groups together that they thought could benefit.
In a symbiotic way in over the course of <unk>.
Early 2020 that relationship deepened in terms of really focusing on a scientific understanding of our technology and then what <unk> brought to the table and when we determined to sort of enter into this collaboration with <unk>.
With the NIH, we wanted to continue to preserve as much flexibility as we could with our technology and not tie it down until we were able to better demonstrate value.
As we advance a platform technology, you do need to be careful about setting an early precedent on value and so our goal was to get into these in vitro and in vivo studies as quickly as we could get our hands on the Rem density are and work with them as we progressed and particularly because of the impressive nature of the early in vitro data are <unk>.
<unk> with Gilead have advanced.
And Thats, a very important aspect of this but it will take the in vivo data for us to really then be able to cement this and know where we are so right now I would characterize our relationship is one of flexibility. It's one of the reasons why we of course view <unk> as a potentially a very important drug in the <unk>.
Against COVID-19, but if you look at this relationship through the <unk> lens. This is about proof of concept in a well established model about the intracellular delivery of Antivirals. So we want to preserve our flexibility to go in a number of different directions with antivirals.
One of those ends up being <unk> <unk>, that's a great thing, but the benefit of this in vivo model paid for by the NIH is it gives us that proof of concept and then we preserved our flexibility to sort of either cement our relationship with Gilead go into direction on our own or investigate the delivery of another.
Anti viral which could have even more promise clinically than a drug like <unk>, so I would characterize it as flexibility.
Thanks for that additional color just one or two specifics with regard to 20 501 in the program.
And progression to phase III.
Phase two program.
Kind of mapped out at this point and is there potential for that to be a registrational study.
Sure I'll ask Dr. <unk> to comment on that and I may add some things at the end.
Yes. Thanks for the question Bert So as with Matt 20 to <unk> 20, 501 has the unique opportunity to leverage two regulatory pathways that we believe would allow us to streamline the development programs. The first is the orphan pathway for the treatment of MTM infections and the second is <unk>.
500, <unk> registration pathway since Amikacin is approved and is part of the regimen for the current treatment of MTN. We can rely upon that efficacy data that's already been demonstrated with Eric case, as well as with IV amikacin products for the treatment and we believe that will contribute.
To streamlining the development as well.
Fin indication. So we expect that as FDA has done so with Matt 23, they will allow us to work in a streamlined fashion to bring this treatment to patients and this is all of course with the assumption that we will be able to continue to see us clean safety profile of our drug.
Okay. Thank you for that additional color and then just with regard to $22 three as you mentioned in your comments just.
With regard to what you would need to see in cohort four to really give you a strong hand as you interact with FDA.
I believe you made some comments earlier could you just clarify.
In cohort four what would really.
Drive you think a positive interaction with the agency and then I just have one big picture question.
So we believe we already have the data that will allow us to have a very meaningful discussion with the agency for our drug as a step down therapy. So we believe that we have the data we've already exceeded the primary endpoint, we've seen excellent survival in patients with step down therapy. So our focus for the FDA.
<unk> will be based on data thats already available for cohorts, one and two for cohorts three and four those data will be to support the all oral induction treatment indication.
And as with the data from the first two cohorts will want to see.
Excellent survival as we have seen with the first two cohorts will want to see that the Esa exceed that threshold of zero point to that's been tied to improvements in clinical outcome in mortality, we want to ensure that we continue to see sterilization of culture in the patients without any rebound to relapse.
And of course, very importantly, our continued strong strong safety package that shows no evidence of renal toxicity.
Terrific. Thank you for that additional clarity and then just on kind of big picture structurally as you Gerry I think in your opening comments you alluded to interactions with.
Those in the industry I know it's difficult to characterize.
Partnerships development efforts things like that but where do you think you are in terms of recognition with other players in the industry again, we've mentioned Gilead here, we've mentioned NIH.
And.
Do you think that there are other potential partnerships.
Is something that can be achieved in 'twenty, two or or is that a goal of the company or just kind of in general terms. How do you think about this this.
This technology and interactions with others other players in the industry.
Yes, it's a good question Bert and when we get asked a lot and then obviously on the call we were specific today.
Gilead and two others, we didn't mentioned Genentech for example, who we continue to work with we've extended that relationship again for another year.
And there is a lot of interest on that side, but of course, there, especially following enact theres a lot of inbound interest in our technology. It's no.
Shouldnt come as a surprise to anyone that in these areas of innovative medicine like the delivery of messenger RNA like the delivery of gene therapy, there continued to be real delivery intracellular delivery challenges either.
Highlighted by inefficiency are highlighted by <unk>.
Unwanted immunogenicity toxicity things like that we're actively engaged in those sorts of discussions and for US. It's a balancing act we have 25 employees in <unk>.
We have a lot of ongoing projects.
Wed like to be able to continue to further validate this technology with a collaboration in one of those areas and those discussions are ongoing.
Timeliness to those and so that is a goal of the company to further broaden the use of this technology beyond small molecules.
Beyond Antivirals and I would I would characterize those discussions as robust and ongoing.
It absolutely is a goal for 2022.
Something may happen before 2022. So this is one of the ways that we are looking at increasing the utilization of this technology, but we remain intently focused on controlling our own destiny. So advancing 22 of $3 20, 501 are key for us because through those two drugs.
We can control our own destiny and those are drugs that can be.
Hopefully approved and then commercialized with less than what would be a customary commercialization effort for other drugs. So we have a lot of flexibility here I would say we're careful about the collaborations we choose to engage in because it's great to say you want to be partners with everyone, but then relationship management.
Well I'm, a small organization like ours. So we're concentrating our discussions in a few key areas.
Which we have a sense of urgency around and we will continue to drive those until they are at a point, where it makes sense to finalize those.
Thank you for the additional color and look forward to the progress congrats on it so far and again look forward to more thanks. Thanks Bert.
Our next question comes from the line of Greg Fraser, which rose. Please proceed with your question.
Good morning folks thanks for taking the questions.
On that 20 501, I know it's early in the development program and Im curious how youre thinking about the initial target patient population, whether it's patients with <unk>, who could benefit from oral delivery versus the current inhaled drug it sounds like obviously, yes, but do you think that 20 501 might also be effective or more difficult to treat patients like those that might go back to.
<unk> asbestos.
Yeah, I'll, let Terry I'll, just comment briefly at the beginning absolutely we're not going to be limited I think to the the <unk>.
I would characterize as poor demonstration of efficacy.
And significant toxicity of the available therapies. So we've shown the ability to have an impact both on on Mac and on <unk> pre clinically. We are also one of the reasons we've attracted.
The attention of the cystic fibrosis Foundation, which actually has not worked with these competitive products because of their inability to be an effective treatment for patients with cystic fibrosis is our unique ability to get drug right to the lung and the non toxic way.
We believe that this drug pre clinically is set up to go well beyond Mac and MTM really is also just the first indication for this drug.
And that's because that's where the NIH really put it attention because of the significant need there, but unlike that competitive drug which would be limited to the treatment of pulmonary infections. If at all we can go after disseminated infections and so I think youre going to see sort of a ladder indication approach here are stacked.
Indication approach similar to what we would look to do with $22 three but initially in terms of MTM disease, we plan.
And the data support going far beyond Mac, but anything to add there Terry.
Exactly to Gerry's point, we've really seen remarkable preclinical data in the cystic fibrosis mouse model data has been generated at the University of Colorado by Dr. Diane <unk> the data are.
Very very compelling compelling and the support that we are receiving from the cystic fibrosis Foundation. So we have clinical and preclinical validation from experts in the field, including experts such as Dr Daily Who's our key.
Advisor on our program.
What we can now do is look at how we leverage the strong safety profile that we are able to see with an oral product that utilizes our platform and see how we can leverage that to the benefit of patients where they can be treated in an outpatient setting and potentially even with a mono therapy of our oil.
Amikacin product.
So for us the preclinical validation has been critically important and we intend to convene the clinical experts to see how we can leverage this unique preclinical efficacy profile that we've seen coupled with the safety to look at potentially treating NTN with mono therapy and looking at how we better optum.
The current treatment regimens for these patients.
So we're in the early phases of mapping out the clinical program, but we are looking to two.
To be as broad as we can leveraging the strong safety that we've seen to date and as we approached the cystic fibrosis Foundation actually they approached us and as we responded to that inquiry, we had to submit sort of an outline of what phase two would look like in one of the benefits of working with a patient advocacy group like the system.
Fibrosis Foundation did you get the benefit of their experience and expertise and access to patient. So we've been collaborating already on sort.
Outlining what a phase II program would look like it's one of the reasons why we're conducting all of these long term Tox studies as a course of treatment for MTM can be as long as 18 months and even longer and.
And so thats why we are entering into a lot of these studies during 2022, but with the support of the cystic fibrosis Foundation and other patient advocacy groups and the lineup of Kols. We've established our goal is to sort of put together that is comprehensive but also a streamlined phase II development program as we can.
<unk>.
So that we can address where continues to be a significant unmet need.
Great. Thanks for all the color and congrats on the progress.
Thanks, Greg.
There are no further questions in the queue I'd like to hand, the call back to Mr. <unk> for closing remarks.
Thank you Doug and thank you all for joining US today. We appreciate your continued interest in <unk> and our entire team here looks forward to providing you with updates on our future progress have a great day.
Ladies and gentlemen, this does conclude today's teleconference. Thank you for your participation you may disconnect. Your lines at this time and have a wonderful day.