Q3 2022 Matinas BioPharma Holdings Inc Earnings Call
Ladies and gentlemen, thank you for your patience. Please remain on the line your conference call will begin momentarily again, we do appreciate your patience. Please remain on the line your conference call will begin momentarily. Thank you.
[music].
Welcome to the Maquiladora Biopharma third quarter 2022 results conference call at this time, all participants are in a listen only mode.
Question and 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 the Doctor I'll get Bhargava Investor relation represented representative form of Phoenix Biopharma you may begin.
Thank you Dan good.
Good afternoon, everyone and thank you for joining the <unk> Biopharma third quarter 2022 results conference call earlier. This afternoon, we issued a press release with our financial results along with business updates that release is available on the <unk> Biopharma website under the investors section.
These are there are slides accompanying today's presentation, which are available by joining the webcast through the investors section of the company website.
Speaking on today's call will be Jerry <unk>, Chief Executive Officer, Dr. Terry Mcevoy, Chief Development Officer, Dr. Terry Ferguson, Chief Medical Officer, and Keith Kucinski, Chief Financial Officer. We also have Mr. Thomas Hoover Chief business officer available to answer questions. During the Q&A section.
At this time I would like to remind our listeners that the remarks made during this call may state management's intentions hopes beliefs expectations or projections of the future. These are forward looking statements and involve risks and uncertainties.
Forward looking statements on this call are made pursuant to the safe Harbor provisions of the Federal Securities Law. These forward looking statements are based on Makena biopharma as current expectations and actual results could differ materially as a result, you should not place any 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 security 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 the company website also in the investors section.
Following the company's prepared remarks, we will open the call for a question and answer session now I will turn the call over to Jerry.
Thank you I'll get good afternoon, everyone and thank you for taking the time to join US today. This afternoon, we will review our 2022 third quarter financial results and provide some highlights of our recent progress.
We have departed from our normal filing schedule and call timing for this quarter. So I thought I would begin by providing some quick color to explain this change.
First we have seen that there is a lot of competition for analyst and investor attention with the early morning calls we wanted to provide an opportunity in a more convenient time for our analysts and valued investors to be able to participate in a meaningful way second Dr. <unk> and I had been planning to travel to Uganda Tomorrow in preparation for our phase III trial and not.
Returning until our annual meeting of stockholders set for Monday November 14th despite that trip being postponed temporarily we thought it best to stay on the schedule. We had laid out for our analysts earlier. This fall so now let's get into it during the third quarter and through today, we have achieved significant milestones for our company, we are a company, which prides itself.
And being able to set and accomplish goals and we are very proud of the list of boxes, we have been able to check during 2022 at the very top of that list was the announcement of the interim results from cohort four of our phase II <unk> trial of Matt 20, 203 in Cryptococcal meningitis overall.
Overall <unk> was a highly successful phase II trial with patient survival results that are simply unprecedented for an oral therapy in this vulnerable patient population.
Dr <unk> and her team working closely with Dr. David Boleware from the University of Minnesota delivered something many never thought would be possible.
Our clinical trials showing that oral mat 2200, III could deliver effective doses of amphotericin b directly to the brain achieve survival rates in excess of 90% and virtually eliminate the kidney toxicity historically associated with IV formulations of amphotericin, allowing for the safe use of Matt 22, or three for up to <unk>.
Six weeks.
We want to thank all of the enact patients are dedicated investors investigators and the entire clinical study staff in Uganda for their commitment to this important clinical trial.
The feedback from Kols and infectious disease doctors that I'd week, two weeks ago in Washington was fantastic and we're aggressively preparing to commence our phase III trial in Cryptococcal meningitis and the first quarter of next year.
Dr. <unk> will go into more detail on our <unk> results and how we are well positioned for phase III a bit later in the call.
In addition to these impressive data in crypto Dr. Ashraf Ebrahim of the Lundquist Institute of the Harbor UCLA Medical Center presented data at I'd week, with Matt 22, or three and its impact on another deadly fungal infection Nucor Mycosis. Dr. Ferguson will briefly review some of the highlights from this promising preclinical study.
In vivo.
During the quarter. We also received important feedback from the European Medicines agency in the form of scientific advice and the granting of orphan drug designation by the EMA for the treatment of Cryptococcosis.
The EMA feedback was overall in line with our FDA supported plan for phase III, which positions this product for global development and registration.
Positioning, Matt 22, or three for global development, and continuing to generate compelling data across invasive fungal infections could not comment a more opportune time.
As you may be aware in October the World Health organization released its first bundle priority pathogens list to guide research and development and its report the W. H O highlighted that invasive fungal diseases are rising and then the alarming rate, particularly among immunocompromised populations.
The under recognized and emerging global health threat of invasive fungal disease is compounded by rapid emergence of resistance to many of them are available therapies.
Since that generally leads to prolong therapy in recurrent hospital stays.
Despite this continually growing threat to human health fungal infections received very little attention and resources globally.
The WH Joe classified the identified bunzl priority pathogen three groups critical high and medium.
Both cryptococcal meningitis and aspergillosis, we're on the critical list followed closely by Mucormycosis on the high priority list with our phase two success and enact Matt 22 of three is well positioned to become an important weapon in the battle against Cryptococcal meningitis, and we have also generated consistent and compelling preclinical.
Data and both aspergillosis, mucormycosis, which could position, Matt 22, or three to become an important part.
Active treatment for many of these critical and high priority fungal pathogens.
We believe Matt 22, or three has many of the attributes of <unk>.
And ideal fungal agent.
<unk> seidl, it's not prone to resistance, it's well tolerated and safe with no observed kidney toxicity. Despite used for extended periods up to six weeks and thats minimal to no drug to drug interactions, it's oral and it's delivered directly to infected tissues without exposing the body to toxic levels of drug in the blood.
Yes.
The profile for Matt 22, or three was one of the reasons, we felt so comfortable and first studying its impact in cryptococcal meningitis, a severe invasive fungal infection with among the highest rates of mortality.
Complicating treatment disinfection houses are tough on the brain, making it very difficult to reach however for mid teens. This infection presented the opportunity to not only demonstrate that <unk> 22, or three could safely and effectively treat this steadily fungus, but we could also show that our LNC platform could eliminate the historic toxicity of <unk>.
Arison, B and carry this molecule across the blood brain barrier and directly to infected tissues.
With enact successful and in our rearview mirror, our confidence is extremely high that one Matt 22, or three is positioned for success in phase III in Cryptococcal meningitis and too Matt 20, <unk> III can also be used to treat other high priority fungal pathogens like mucor mitosis and aspergillosis safely in.
Effectively which informs the balance of our planned phase III program I'd like to turn the call over now to Dr. <unk> to walk us through the mat 22, or three enact data in more detail.
Thanks, Jerry and good afternoon, everyone I won't go into detail on the design of the Nash since by now everyone is familiar with our phase III randomized open label sequential cohort study evaluated Matt 22 between the treatment of Cryptococcal meningitis, we announced data from cohort two in September of 2020.
One and just recently at ideally where interim data from the cohort four were presented by one of the principal investigators of the study.
And that received the outstanding abstract and IBSA Award from the infectious diseases Society of America.
We believe is reflective of the impressive nature of the data and the unprecedented survival rates achieved with our oral therapy.
Turning now to data from cohort four slide nine depicts the survival seen with Matt 20 tool suite treatment in both cohorts two and four versus the survival seen with standard of care IV amphotericin throughout the entire <unk> trial.
Ultimately survival is what infectious disease doctors and FDA care about in this patient population.
At each time point and enact mid 'twenty, two or three outperform standard of care on survival.
Lee and act was not powered or designed for non inferiority, but these data provide a great deal of confidence heading into our phase III trial with a primary end point will be two week, all cause mortality or survival with a key secondary endpoint of 10 weeks survival.
I mentioned earlier these are unprecedented survival rates with an all oral therapy.
The primary endpoint in enact was early fungicide, all activity or Esa, which is the rate of EC clearance in cerebrospinal fluid.
This is a direct measurement of the quantitative rate of antifungal activity at the site of infection and has become a well recognized surrogate marker for survival.
The pre specified target Esa thresholds and enact with zero point, too, which is clinically meaningful and represent a robust degree of fungal clearance that is associated with enhanced survival.
Importantly, and as pointed out on this slide treatment Esa is beyond a 0.2 threshold has not historically resulted in any incremental benefit or.
Overall, net 22 or three achieved the primary endpoint and demonstrated robust early fungicide will activity in both cohorts two and four which is why we believe mid 'twenty two or three was able to reach such remarkable survival rates in this study.
Several patients with high baseline from the burdens had noteworthy antifungal activity within the met 22 or three treatment arms, including one patient with quantitative Cryptococcal culture as high as 915000 <unk> per ml at the time of screening.
Each of these noteworthy cases highlighted effective clearance during the induction period.
Key demonstration of potent antifungal activity, even in the most challenging of cases.
This shows that our drug is having an immediate and high impact on the disease, a key observation for infectious disease doctors.
These next two slides highlight the data from cohort two in the design of our phase III trial, which is predicated upon the design of cohort two of the net.
As a reminder, cohort two was designed to evaluate <unk> 22, a three step down therapy. After only two loading doses of IV amphotericin.
In cohort two are two weeks survival was 98%.
97% of patients achieve sterility, including all patients completing the induction course of treatment with <unk> 22, or three and there were no breakthrough infections.
Matt 22, or three was additionally, safe and well tolerated with no kidney toxicity electrolyte abnormalities observed even with six weeks of treatment with <unk> 22 or three.
Our phase III studies replicate this cohort design and we believe there should be a high degree of confidence in its success.
We have chosen this design for a few key reasons.
First it's a derisk design predicated on 90% survival at our key primary endpoint with a 10% non inferiority margin against standard of care, which is a very comfortable.
Secondly in practice, most physicians treating patients presenting with cryptococcal meningitis or other deadly invasive fungal infections are most likely going to start patients on an IV therapy.
Third enrollment is likely to proceed faster with the IV loading doses available.
And fourth treatment guidelines will likely look more favorably. Upon this design in mid 'twenty, two or three could be included much faster than is customary an important element for both physician adoption and payer acceptance.
We're excited to initiate this phase III study early in the first quarter of next year, we will expand our clinical sites up to five and another important aspect of our de risked FDA reviewed adaptive design is it potential to add additional patients once enrollment has reached 75% to offset unforeseen patient deaths.
Weighted to study drug.
Enrollment is expected to take approximately 18 months with topline data expected in the second half of 2024.
Before I turn the call over to Dr. Ferguson I would like to mention some of the progress we've been able to make on the CMC and manufacturing side of Matt 20 tool suite.
As you know we are partnering with thermo Fisher scientific for the commercial manufacturing and NDA readiness for Matt 20 tools to me.
Im pleased to report that due to both internal and external efforts our transition to thermo Fisher is proceeding on schedule and we are in a good position as we enter phase III.
We are intent on doing everything within our control to ensure the successful development registration and commercialization of this important product for ourselves and prospective partners around the world.
I will now turn the call over to Dr. Ferguson for some additional color on our mid 'twenty two or three program.
Thanks, Terry building off of <unk> comments, we are positioning Matt 22, three is an ideal antifungal agent with key value propositions of longer term oral administration and avoidance of significant kidney toxicity.
The capability of our LNC platform to both preserve the efficacy and fungal killing power of amphotericin B, while at the same time, making it convenient and safe for patients is incredibly important.
While we are beginning our phase III program with an indication for the treatment of Cryptococcal meningitis. We've also developed a protocol outline for a phase III study for the broader treatment of invasive fungal infections and plan to meet with the FDA to discuss this in the first half.
Half of 2023.
Our ultimate goal is securing multiple orphan indications for Matt 22 O three maximizing the available regulatory exclusivity. While also positioning this drug to be used as broadly as possible in the treatment of those <unk>.
Five priority fungal pathogens.
Additionally, we believe Matt 22 O three has important potential applications for the prevention of invasive fungal infections, especially in immuno compromised patients such as leukemia patients and transplant patients.
Underscoring. These plans are data, we continue to generate in support of potential broader use of Matt 22 or three.
As Jerry mentioned earlier.
Dr. Ashraf Ebrahim of the Lundquist Institute of the Harbor UCLA Medical Center presented Matt 'twenty, two or three data for the treatment of invasive mucor mycosis at I'd week.
This late breaking study demonstrated impressive in vivo efficacy of Matt 22, or three in treating two different strains of mucor Alice.
Suppressed mice with a prolonged median survival time enhanced.
The enhanced overall survival.
<unk> significantly reduced tissue phone call burden of target, oregon's, including lung and brain.
Comparable to that of Ambisone conventional IV amphotericin b.
Thus positioning Matt 22, or three as a potentially important clinical option for the treatment of these deadly invasive fungal infections.
Overall, Matt, 22% or three as being positioned for global success with significant projected U S and global revenues, we've seen more partnership interest in Matt 22, O three lately and recent deal activity in this space bodes well for the value we believe.
We have created in our Mat 2200, <unk> III program.
I will now turn the call over to Keith Kucinski, Our Chief Financial Officer, who will discuss our financial results.
Thanks, Terry and good afternoon, everyone.
Today, the company reported its financial results for the third quarter of 2022, which reflected a net loss attributable to common shareholders of approximately $5 $5 million or <unk> per basic and diluted share.
Compared to a net loss attributable to common shareholders of $6 $8 million or a net loss of <unk> <unk> per share basic and diluted for the same period of 2021.
The reduced loss resulted from the company recording $1 $1 million in revenue during the current year period from its research collaboration with bio <unk> Tec SC along with a slight decrease in operating expenses year over year.
Cash cash equivalents in marketable securities at September 32022 were approximately $33 1 million compared to $49 6 million at December 31, 2021.
Based on current projections, we continue to believe that cash on hand is sufficient to fund planned operations through 2023.
I will now turn the call back over to Jerry to provide some pipeline updates and concluding thoughts gerri.
Thanks Keith.
I did want to provide an update on our pipeline programs as I know many of our investors are following these closely first and most importantly, our licensing discussions with biotech remain ongoing.
The potential for the oral delivery of nucleic acids is extremely large and we want to make sure. We are putting our company and our shareholders in the best position, both near and long term.
I believe our research collaboration is progressing well and we have learned a lot about our technology and its application to nucleic acids over these past few months, we are diligently working together with <unk> to commence in vitro and in vivo studies as soon as possible.
Ultimately we are both looking to increase the overall probability of success for these studies and for each of US there are a lot of steps along the way on the license side I have stated numerous times publicly that we are looking for the right deal and are prepared to make a fair deal for access to our LNG platform.
We look very favorably at the precedent in this space and we are also gratified to see a lot of interest from other third parties. We continue to believe that a deal with Ireland Tech makes sense for both companies and are optimistically working toward that goal. We hope to have an update on this prior to year end.
The other thing I want to highlight is that we have consciously chosen to slow down development of map 20 to 20 501 to allocate resources judiciously in these uncertain market times, while we continue to believe in the opportunity for the first oral aminoglycoside, we want to prioritize, allowing some of our potential non dilutive and inexpensive.
Financing options to materialize <unk> mature prior to moving into a phase III <unk>.
Study with that drug in.
In the short term, we believe advancing Matt 22, or three into phase III and focusing our other efforts on platform expansion provides the greatest return for shareholders.
And looking at our overall financial picture.
We continue to push forward from a relative position of strength.
Unlike many small cap biotech companies, our enterprise value is far in excess of our cash, which we believe represents the market's belief in our LNG platform and its considerable potential.
While we do believe we continue to be undervalued, our cash runway into 2024 and multiple short term opportunities for non dilutive cash influx is positioning us to be able to capitalize intelligently as we advance our products and technology.
We have always been good stewards of our resources and we will continue to protect our shareholder interest.
We will continue to be thoughtful with how we extend our cash runway and we believe that there are several ways. This could happen in the short term, which would be viewed beneficially by our shareholders in the market.
Overall, we are pleased with the progress we have made year to date and there are several boxes remaining to be checked between now and the end of the year and then into 2023 each of these could and should be viewed as potential value inflection points and we are intently focused on executing to achieve one or more of these key objectives.
Importantly, we have also used 2022 to expand our IP portfolio surrounding the LNC platform with multiple key new provisional patent filings, which we believe can further protect this technology and position us well internally and with partners globally.
We have also taken the opportunity to increase awareness of our platform and its capabilities at multiple scientific and business development conferences throughout this year.
Events, such as the next generation lipid drug delivery conference partnerships in drug delivery Bio Europe Spring Mycosis study group biannual, meaning the.
The mrna based therapeutics summit, the international mrna Health Conference and the American Society of Gene and cell therapy has given our team the opportunity to both present and to engage with others to help them better understand our platform and how it potentially could solve some of the most challenging issues with drug delivery today.
We wanted to increase our exposure and awareness at the same time, we are doing whatever is within our control to advance our platform.
This will conclude our prepared remarks for today I will now turn the call over to the operator to facilitate our question and answer session.
Thank you the floor is now open for questions.
I would like to ask a question. Please press star one on your telephone keypad at this time again, ladies and gentlemen, if you would like to ask a question. Please press star one on your telephone keypad at this time, please hold while we poll for question.
Our first question comes from Greg Fraser with Carla Please state your question.
Good afternoon, and thanks for taking the questions and congrats on the progress.
The first question on <unk> can you expand on the feedback that you heard from the Doc fit ideally with a feedback universally positive was there any database. The das we are asking for late this year or is it relapse data to better understand the clinical profile.
Yes, Greg Thanks for your question I'll turn that right over to Dr. Mack of it and then if he wants to weigh in Dr. Ferguson, but Terry go ahead.
Sure. Thank you for the question Greg So two of the key elements of our clinical data for our crypto study that really resonated with the physicians with survival and safety. So the ability to with an all oral regimen have survival rates that exceeded far exceeded what <unk>.
Has been seen with other available therapies was really resounding and really.
Supported where they saw the benefit of our drug in the clinical setting.
Secondly, the safety profile of our drug so being able to treat patients for the first time with a longer treatment course of amphotericin B product was really.
Fitting into a high area of unmet need with patients suffering from invasive fungal infection. So those really were key attributes of our drug that really has highlighted the clinical interest and the opportunities to expand the use of our drug for the treatment of other deadly invasive fungal infections such.
New corn aspergillosis endemic Mike policies and that the Candida infections really is the the expanded potential to for the first time be able to use in amphotericin product clinically for longer term use in these other deadly infections.
Got it and anything else to add there Terry Ferguson.
Yes, just to add to that I think that.
Sure.
We're very happy with the data that they saw their work not additional questions or holes that they.
Found in the data. So they were very excited about it you saw a lot of people taking pictures of the slides during the presentation and I think that the general air that ISS was one of excitement around an opportunity to do things with <unk>.
Listen that hitherto were not really possible to be able to give it orally to be able to give it without worrying about the nephrotoxicity. So I think that the sense that I saw was a lot of excitement and very happy with the data that they saw.
And then so just to add to that too I mean, Greg the one thing that jumped out at me. The most is that the questions instead of two.
Talking about the data themselves they jumped to the how and it was all about mechanism of action and how our platform works in trying to understand PK and that gave us the opportunity to talk about how this LNG platform and.
Encapsulates drugs and a solid crystal it keeps drug out of the blood, we access activated cells, who go right to the site of infection. So they were so I think impressed with the ability to achieve these survival data with an oral therapy that had jumped right to that to the how and and then then it became a platform moment and so I think thats.
The other thing that happened at I'd week that had people really interested.
That's really helpful. Thanks for all the color.
I'm curious about the phase III.
For invasive fungal infections will you be enrolling patients with different infections like Q4, aspergillosis and how are you thinking about size and duration.
And then also cod.
Yes.
It's a good question and I would say we've developed the protocol outline for that and our plan is to enroll patients who are suffering from a variety of invasive fungal infections. There is precedent in this category for these so-called all comment our basket studies I think youll see us focus on the big ones of new core ASP.
<unk>.
And maybe some of the more endemic Mike Ocs.
We're working on on.
Patient numbers now we don't think it needs to be a huge study based upon precedent in this space and the fact that we have demonstrated success in Cryptococcal meningitis, we'll obviously prepare well and submit a package to FDA FDA to have those discussions.
But that will be something that happens in the first quarter, but Terry mcevoy anything to add to that.
Yes, I think Joe you hit it on the head so we intend to leverage what we've already demonstrated vis vis efficacy in cryptococcal meningitis.
To then allow us to do a more streamlined basket study for treating invasive fungal infections, where we hope FDA would be amenable to a more streamlined design that perhaps would allow us to do a smaller open label study with external competitors that would not require us to do a significantly.
<unk> study.
Yes.
Can you use the same formulation that would tell you that you use the internet.
Correct, yes.
Okay.
Would you initiate that study on your own or.
Possibly wait for a partner to help fund it.
So it's a great question.
Given the interest in this asset and the fact that we're in an active process.
Our hope and expectation would be that we have a partner on board, particularly given the global nature of that sort of trial not from a cost perspective, but just from setting this asset up the right way from the beginning to be able to check a lot of the regulatory boxes around the world.
So that is we anticipate that that would be part of the.
The discussions we're having with third parties.
Got it I'll ask one more quick one and get back in the queue, but can you just remind us how long what's the standard treatment protocol with ample for for Nucor in aspergillosis.
Yes, it's different and I'll, let Terry Mcevoy spiel that one.
Yes, so because of the toxicities associated with IV amphotericin use its typically restricted to one to two weeks of treatment followed by step down to an ASR typically are in a kind of Camden, but those classes of antifungals have significant liabilities associated with their use such as resistance and <unk>.
<unk> complications with drug drug interactions.
And just to remind us that these are immunocompromised patients that have significant underlying comorbidities and they're on polypharmacy. So the ability to treat with a safer alternative to a class that has significant drug drug interaction and limitations of use due to those drug drug interactions really positions our drug <unk>.
Really expand the opportunity with <unk>.
Amphotericin product.
Got it thanks, so much.
Okay. Our next question comes from.
Robert Hazlett with <unk>. Please state your question.
Thanks, I've got one or two.
Congrats on the progress as well from me just with regard to the harmonization of the EU phase III could you just go into a little bit more detail. There are there any particular wrinkles in the.
Either additional work or or work youre doing to make it amenable to an EU filing and acceptance.
Yes, I mean, one of the benefits of us going early to EMEA was that we were able to get their feedback and time to incorporate it into our overall.
Protocol planning for the phase III for Cryptococcal meningitis, so even though I would say the majority of their comments were aligned there are some things that they want to see which we will be able to incorporate pretty easily. So we think that this is a study that we will be able to use to meet the criteria both regulatory agent.
<unk>, which is obviously a huge advantage.
I think thats the benefit of Dr. <unk> being proactive there and going and getting that feedback. Because then we can we can align everything at the start of phase III and not find ourselves in the position of having to do smaller studies or other things to a piece of regulatory agencies.
Okay. Thank you.
And then.
Talked about being in an active process with regard to 'twenty, two or three and.
I'd love to just get your sense of maybe where you are in.
A good deal might look like there I know there is a thousand different ways to slice. This but is this something where you might consider a geographic.
Carve out or is there something given this alignment that seems to be in place.
You'd like to.
Licensed the whole thing, but just a little bit more of the status and a little bit more of the types of deals that you might be considering thanks, yes.
So Brian it's a good question.
This business timing is everything and I think the fact, there have been parties, who have actively been following the development of this drug for some time, obviously their interest was piqued by cohort two where this is the first time you really saw the ability of this therapy to effectively treat.
Immuno compromised cryptococcus patients.
But that's only increased with the data at <unk> and now the attention brought by the World Health organization's call for additional development. We've also seen over the last year and a half, particularly during the pandemic the increase in the prevalence of these fungal infections, particularly in immuno compromised patients. So it's a perfect storm.
There is a lot of precedent in this space for deals that are done on a regional basis. I mean, you can look pretty recently.
At least in the last couple of years that things like <unk>.
In that in that space I think we have a better product I think we have a product that has a broader spectrum and has the ability to more effectively treat these pathogens. So we want to be smart about it. What we know is we're not going to slow down. This trained because we have a drug that works really well in these patient populations and has the opportunity to stack indications.
But that interest is increasing we would and are open to regional sort of licensing deals, but we have not discounted the possibility that there is a global deal here, giving given the emergence and the attention. So we're focused on execution, we're being smart about how we fund the NIH is support of the phase III in Cryptococcal.
Meningitis is a big reason why we can be patient.
That again theyre going to stand firmly behind supporting a lot of the patient costs in that study.
So that's a big variable for us I know, Tom Hoover, and Terry Mcevoy and others are working hard to inform partners. We have an active data room that is open. So we will be opportunistic we want to be comprehensive in terms of canvassing all interested parties, but finding that right partner is it.
Key part of our business objective I would say over the next two quarters.
That's terrific and just following on that exact topic with regard to bottle intact, certainly don't want to negotiate on quarterly conference call, but at the same time I am interested if there was any particular.
Oh.
Ask or any particular hurdle, that's being considered in <unk>.
Order to be able to effect a broader transaction there.
And which transaction I'm sorry.
A potential broader deal for bio amtech.
I don't think it's an obstacle.
It is an obstacle I think for them, there's a number of things that they need to consider I think they are quite happy with how the research collaboration is going and when we all know that their intention has been pulled in.
And a number of different directions.
The parties are talking about value.
And so I think anytime you are trying to wrap your head around valuations and things like exclusivity in things like the opportunity for an oral messenger RNA I think things get complicated, but these are I think mature.
Pleasant discussions, we always have a sense of urgency in everything that we do but we are comfortable.
Proceeding on a timeline, which puts us and our shareholders in the position for the right deal.
If we would've had the right deal two months ago, we would have done it I think we're moving closer every day.
Both parties I think believe it makes sense. It's just a question of being able to cross those Ts and dot the eyes.
Thank you I appreciate the answers.
Look forward to more progress.
Okay.
Okay. Our next question comes from.
My mom pie from B Riley Securities. Please state your question.
Hi, Good afternoon team Mr. Thornhill Kazmi on for Mike Congrats on all the progress and thanks for this really comprehensive update and some of the interesting data we're seeing from an act so.
Maybe just starting there as you think about the phase III study could you talk about some of your expectations for the different treatment arms in terms of the induction consolidation versus standard of care and maybe put in context, how significant that one week loading dose of amphotericin has.
Yes, so a couple of things that we need to sort of walk back there there is not going to be a one week loading dose for the phase III at least as far as them at $22. Three arms, we're talking about two loading doses over the first two days patient.
Patients will receive a loading dose and then it will transition on day, two actually to oral mat $22 three for the balance of the 14 day induction period.
It's only in the standard of standard of care arm, where they will be getting seven days of IV amphotericin, followed by Fluconazole. So we don't expect and what we saw both in cohort two and was validated in cohort four is that the loading dose was not responsible for the antifungal effect of our drug I think that.
It's one of the benefits of having not stopped at cohort two and gone all the way to an all oral regimen in cohort four it takes away. The very question that you asked about what is the benefit of the loading dose I think it's just peace of mind for physicians at the beginning I think theres big enrollment benefit to doing it that way and I do think that there is going to be.
Some guideline impact there, so but I'll, let Terry Mcevoy talk specifically about the two treatment arms. I mean this is approximately a 270 patient study it will be randomized two to one to them at $22 three treatment arms, but Terry maybe you want to talk about that and what the differences in the opportunity that FDA.
Creation trial. So we believe that we are very much positioned for success given that we have now in two independent cohorts of the neck been able to demonstrate.
Numerically better survival relative to standard of care.
And recall that the overall statistical analysis will be one of non inferiority. So we need to demonstrate that we are not inferior to standard of care. It's not a superiority endpoint, which is also a much lower bar from a regulatory perspective.
Secondly, the 10 week, all cause mortality and point will be the key endpoint that will support this longer term treatment indication that will be a first ever indication for any amphotericin product and there again based on our cohort to data we saw numerical benefit of our drug versus the.
<unk> of care, where we had 90% survival relative to 85% survival and the standard of care arm. This was also replicated in cohort for the all oral regimen. So we believe we really are very well positioned to be able to meet the criteria that will be pre specified as the success of the study.
For both induction as well is that six week treatment.
For the the label.
Final lethal.
Sure just a follow up on that too I mean, that's one of the benefits of having multiple interactions with that.
Fear is getting them to agree to be open to that longer indication of up to six weeks and that's primarily because of the safety data that we were able to generate and that they saw.
In addition to the efficacy because that's also what sets you up for the treatment of Mucormycosis in the treatment of aspergillosis huge treatment periods.
Longer.
In two weeks, so the FDA will be able to see the data from crypto and that should factor in very favorably to how we're thinking about the possibility of longer indications.
With a streamlined less chat.
Challenging trial for the invasive fungal infections as well.
Mmm excellent. That's that's really helpful. Thanks for the additional color there and then maybe just a brief question on the violence that collaboration are able to discuss in a little more detail kind of some of the ongoing research and diligence that might've been informative of the preclinical models, you're thinking as you look to deploy somebody attractive features of the platform here.
Yeah, we can't I mean.
We're not really at Liberty to go into the to the exact nucleic acids are the targets or things like that but this is really two parties coming together, who have strengths and expertise in two unique areas.
US with our ability to formulate and create novel formulations in them with their unique messenger RNA format. So there was a lot of learning that had to go on between the two groups in terms of understanding what they had used before to deliver how what challenges that they ran into and then we will also did a lot of work with them on <unk>.
Identification sell uptake basic stuff.
To position US then to be able to identify what are gonna be the best.
<unk> an in vivo studies. So it was really building it from the foundation for the longterm.
Other than that I can't go into any details, but but it has been collaborative.
And back and forth and so I think both parties have learned a great deal about why it makes sense for the two of us to work together for the long term.
[noise] understood. Thanks, so much for taking our questions and congrats on all the progress that was a quarter.
Q.
Our next question comes from.
Matthew Kosovo. Please thanks requested.
[noise] hi, Thanks, and congrats guys continued LNC progress.
So based on the cohort for success and pivotal developments in cm focused on sat down therapy. What are your thoughts on future usage of 22, three as a frontline therapy and how relevant do you see that ultimately being in terms of treatment benefit and the focus on platform expansion.
Thanks, Yeah, Matt. Thank you for the question and thank you for your patience too for being in the queue. The selling of course, that's a really good question because we get this question a lot from investors why are we talking about step down versus just starting with the royal therapy, you're giving up prescription days and things like that and we've looked at all of those issue.
<unk>, we obviously have been able to look at the cohort for data as an open label trial, we obviously have gone over the cohort to data pretty significantly we've had the opportunity to talk to physicians and payers about how they view all of this and gain a good understanding of what the treatment landscape.
And we can come back we keep coming back to the same place where.
Using that 22 O three a step down therapy just.
I'm, sorry, just checks a lot of boxes. It allows us to sort of Derisk are studies that allows us to align ourselves with what physician practice may be it allows us to get through these studies faster it eliminates questions and some physicians or patients mine's about enrolling in the study. We think this is how.
It will work.
In practice and we think that.
It's not necessarily it's obviously after cohort for there's no fear that we couldn't do it but it's taking every factor into account and positioning the drug for big success in the reality of starting with IBM transitioning to Orem at 22 O three and the beauty of our phase III study design.
Is if we can show that there is a survival benefit and taking therapy out.
10 weeks survival.
Where'd, you get there and not only showing that you can enhance survival at 10 weeks, but you're also going to be able to show that you can use them at 22 O three safely for longer.
You get a much longer prescription time, and so the length of time that a patient is going to be comfortably on your drug.
More than makes up for the two days and from a health economic perspective, the ability to show in both.
More likely than the invasive fungal infections studying than in the crypto study just because of the nature of the design and the location in Uganda.
There's going to be a health economic aspect that we're going to be able to measure and show.
We're getting patients out of the hospital faster they are not going to be prone to the resistance you see with <unk> today, which which may.
Cause relapse or hospital readmission. So we think that on balance the economics are going to be better for us and we're going to be able to get the study done faster and get it in patients hands much quicker.
Very helpful. Thank you for that and congrats again on the progress.
Thank you.
Now I will turn it back over to Gary for closing remark.
Great. Thank you dogma, we appreciate everyone taking the opportunity to joined today, we look forward to your feedback and if this afternoon slot works better for our investors and analysts I think judging by our analysts Q today and the participation I'm able to see online and calling in I think this is a good thing so.
We look forward to keeping you apprised as to our progress for the balance of 22, we're gonna close strong. We thank you for your support one of the benefits. We have is a small biotech company is the strength of our Investor base. We have a very passionate informed investor base follows US closely we're going to continue to work hard on your behalf.
And check those boxes for the rest of the year and in 2023, thanks everybody.
Thank you. This concludes today's conference call. We thank you for your participation you may disconnect. Your lines at this time and have a great day.
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