Q2 2023 Matinas BioPharma Holdings Inc Earnings Call
Welcome to them Batina Biopharma second quarter 2023 financial results Conference call.
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As a reminder, this conference is being recorded I would now like to turn the conference over to Jody Cain. Please go ahead.
This is Jody Cain with L. A J. Thank you for participating in today's call joining me from <unk> Biopharma archery, Jafar, Chief Executive Officer, Dr. Terri <unk>, Chief Development Officer, and Keith Kucinski, Chief Financial Officer, We also have Dr. Terry Ferguson Chief Medical Officer.
We are available to answer questions during the Q&A session.
Like to remind listeners that remarks made during this call may state managements future intentions hopes beliefs expectations or projections. These are forward looking statements and involve risks and uncertainties forward looking statements are made pursuant to the safe Harbor provision of the federal Securities laws.
Forward looking statements are based on the Peanuts, 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 between this biopharma files with the Securities and Exchange Commission.
These documents are available in the investors section of the company's website and on SEC's Dot Gov.
Furthermore, the content of this conference call contains information that is accurate only as of the basis of live broadcast August Nice 20, twenty-three Martinez Biopharma undertakes no obligation to revise or update any statements to reflect events or circumstances, except as required by law and now I'd like to.
Turn the call over to Jerry Jabbour Jerry.
Thank you Jody good afternoon, everyone and thank you for joining us I'm.
I'm pleased to report that we now have received important feedback from FDA and additional clarity on the development path forward to position about 22 or three for approval as quickly and efficiently as possible as you know, Matt 22, or three is our oral formulation of amphotericin B I'll begin today's call with an overview of the feedback we've recently risk.
<unk> from the FDA regarding a study for invasive fungal infections or ISI.
I'll also provide a brief update on our LNC platform for delivery of nucleic acid.
Following my initial remarks, Dr. <unk> will provide more detail on the Fda's feedback and our plans with Matt 20, 203, and will highlight an additional and highly compelling compassionate use case under our expanded compassionate use access program Keith.
Keith Kucinski will follow with a review of our financial results for the second quarter and year to date.
We are appreciative of the very recent feedback provided by FDA towards helping us to design a study for the regulatory advancement of map 22 or three in the treatment of RFID.
During a meeting held earlier this year the FDA acknowledged the need for a therapy like Mad 'twenty, two or three and recently commented that the patients most likely to use our drug would be those patients with limited or no treatment options, who require longer term treatment for a variety of fungal infections, which are susceptible to therapy with amphotericin B also in there.
Most recent feedback FDA informed us that consideration of about 22 or three as a novel first line therapy with an unrestricted label would require an extremely high bar to demonstrate non inferiority, which presents significant challenges for a smaller company like mid teen this ah.
Approval as a first line therapy for just the treatment of Aspergillosis for example would require an adequately powered study with an active rigorously defined comparator group and in all cause mortality non inferiority margin of 10%.
More to that used in prior first line approvals for new chemical entities.
Yep, Yeah, instead highlighted and provided additional feedback and alternative trial designs, which could include the patient population likely to use them at 22 O specifically patients in need of long term treatment for deadly occupies such as angel intolerant or ease all resistant patients.
We believe that these alternative study designs could ultimately position match when each all three for registration under the limited population pathway for Antifungals or El pen pathway. This pathway was originally established to provide a streamlined approach to clinical development of certain antibacterial and antifungal drugs to treat serious or life threatening infections in limited.
Populations of patients with unmet needs and put involves smaller shorter or fewer clinical studies would be required for more broadly used first line therapy.
Enrollment would be focused on the high school patients, which is where both we and FDA already believe our drug will be marked appropriately used.
Furthermore, the alpine regulatory pathway and relevant study design aligns well with our compassionate use cases, and recent clinical experience, which have shown the life changing potential of map 22, all three.
We continue to believe that map 20 till three can play a meaningful role in treating the highest need patients and believe that an approval for about 20 203 pursuant to the alpine pathway could present, a cost effective approach for pursuing approval.
The impressive compassionate use data that we continue to accumulate from our ongoing expanded access program underscores the positive clinical impact of about 22 or three and provides powerful real world. Examples of the life changing potential of this drop in just a moment Dr. Tobacco bits will provide additional details and next steps, but looking beyond me.
22, all three we continue to be extremely encouraged by the in vitro data with our L. N C. Oral formulations of various R&R therapies, we are pushing forward into in vivo studies later this quarter to evaluate the biological activity of these formulations.
And these could potentially represent the first successful oral delivery a functional small all all oligonucleotides that we are aware of and we continue to believe that our technology has the potential to provide truly differentiated delivery in this rapidly evolving area of medicine.
With those comments I'd like to turn the call over to Doctor Machen, It's Terry.
Thanks, Jerry and good afternoon, everyone.
Gerry mentioned, the feedback and pathway outlined by the FDA for machine is to gain approval of map 22 or three in first line treatment indications for ISI would be very challenging to implement for any company much less a small company like mid teens.
For example, a 10% non inferiority margin for all cause mortality would require approximately 700 patients assuming a one to one randomization against standard of care for a given indication in the treatment of a single invasive fungal infection for <unk> and we believe for most companies. This is simply not feasible from the press.
Back to the time required to enroll tweet and follow such a large number of patients and the overall cost of conducting such a study in an orphan disease population.
That said and as Jerry mentioned previously the F. T. I, specifically called out that math 22, or three was likely to be used most in ASR resistant or intolerant patients or in patients with limited treatment options.
This combined with the clinically meaningful outcomes seen in the compassionate use patients receiving that 'twenty, two or three and our expanded access program has provided a clear indication of which direction to move forward with the development of map 22 O B well.
Well the expanded access case numbers are still fairly small both observed and measurable outcomes in multiple different fungal infections in multiple different target tissues highlights the ability of map 22, or three to safely target and effectively eradicate a variety of severe and potentially deadly invasive fungal infection.
And the most challenging clinical circumstances.
I'll provide more detail on our compassionate use cases in a few minutes, but first would like to discuss a potential next steps in the development of that 'twenty two or three.
A key message from the Fda's feedback has the opportunity to narrow our regulatory focus to a population of patients that could derive the most clinical benefit from a safe targeted orally administered form of amphotericin B. These are patients with life threatening isi's with very limited treatment options such as patients.
Renal toxicity or electrolyte abnormalities attributable to IV administered amphotericin as well as all intolerant or is a resistant patients requiring extended treatment of severe fungal infections.
Given this feedback and the continued positive clinical outcomes. We are seeing in our expanded access program. We believe the best course of action is to proceed with development of map 22, or three under the old pad pathway.
We believe the clinical database for such an approval could risk players significantly fewer patients overall to obtain oral step down treatment indications in the treatment of a variety of lifesize and there's more targeted patient population with limited treatment options.
I would like now to briefly describe some of the details around the El pen process.
This pathway was added to the federal food drug and cosmetic Act in late 2016, and what specifically intended to provide a streamlined regulatory process for antibacterial and antifungal drugs to treat serious or life, threatening infections and limited patient populations with unmet needs.
Labeling for drugs under the El pen pathway conveys that the approval is based on the benefit risk assessment that more flexibly, considering the severity rarity or prevalence. The particular infection. The drug is intended to treat and the lack of alternatives available for the patient population.
Determining whether condition as serious as a matter of judgment by the F. D. A but generally is based on whether the drug will have an impact on such factors as survival day to day functioning or the likelihood that the condition if left untreated well progressed from a less severe condition to a more serious one.
As noted previously these criteria aligned strongly with the positive clinical outcomes, we've seen from our expanded use program.
To date, two anti bacterial drugs have received FDA approval under the <unk> pathway.
This pathway could allow us to consider a few different trial designs involving either an active comparator, including best available therapy or potentially an appropriately defined prespecified external comparator recognizing the lack of active comparator alternatives for many of these patients.
We are working now to evaluate the optimal path forward to best position Med 2023 to be approved under an accelerated registration pathway.
It is also worth noting that under the El pen registration and approval pathway may of 'twenty, two or three would continue to qualify for Q I D. P incentives under the F. D. A priority review and fast track designation as well as the orphan drug exclusivity that could allow for 12 years of exclusivity protection in the.
U S and possibly 10 years in the E U.
<unk> 22, or three could also potentially remain eligible for consideration for breakthrough designation as well.
We also plan to engage with the biomedical research and development authority or BARDA as quickly as possible to discuss next steps for funding that 'twenty two or three three registration.
Given that we plan on addressing the highest need patients most of whom have limited or no alternative treatment options. We believe that our case for BARDA to assist with the funding of further development of Matt 22, or three is quite strong.
Now turning to our expanded access program since instituting the program about a year ago. We have received inbound requests from physicians at the National Institute of Health University of Michigan nationwide Children's Hospital, and Johns Hopkins among others on behalf of patients who have experienced significant renal toxicity.
While receiving IV amphotericin B and are have not responded to or are unable to tolerate asos or other classes of antifungals.
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To date eight patients had been enrolled in this program with an additional case pending.
These patients suffered from various life threatening fungal and other infectious diseases, including Canada Aspergillosis Mucormycosis toxicity amongst my closest Fusari Anne and Rhodotorula infections.
These infections have also involves several different tissues in the body, including bone skin lung sinus bladder and the central nervous system, including the brain.
All patients experienced significant treatment limiting kidney toxicity with IV amphotericin b treatment before turning to our expanded access program for treatment with map 22 or three.
The duration of treatment with <unk> 20, 203, all administered as mono therapy ranged from two weeks to six months or longer with no evidence of any renal toxicity.
Of the eight patients four has successfully completed treatment with resolution of their infection with one patient discontinuing treatment for reasons unrelated to that 'twenty two or three.
Three patients continue to receive them at 22, three with ongoing clinical improvement of their infection and no safety or tolerability issues.
Kidney function for the patients who have received IV amphotericin as an initial treatment experienced significant nephrotoxicity or intolerance and have all returned to normal after switching to Matt 22 O suite with no further need for any electrolyte supplementation <unk>.
Additionally, all patients were discharged from the hospital sooner after switching to oral mat 22, or three and continued to receive their treatment with Matt 23 on an outpatient basis.
In each case the reported impact on overall quality of life for patients was highly favorable.
Earlier this year Dr. Morris from the Sally at the University of Michigan presented a compelling compassionate use case at the European Congress of clinical microbiology, and infectious disease or estimate.
Today, we are pleased to share the details of another successful case from nationwide Children's hospital in Ohio, which is recognized as one of the largest and most comprehensive pediatric hospitals and research institutes in the United States.
In this instance, Matt 22, or three was used to treat a 15 year old girl with underlying acute myeloid leukemia, and diabetes, who suffered from invasive fungal infections in sinus lung and brain do multiple highly resistant mucor species as well as aspergillus species.
The patient was initially treated with IV like to sell more of amphotericin, b, but developed treatment limiting electrolyte abnormalities and renal toxicity that requires hospitalization for intravenous hydration and electrolyte supplementation.
Upon enrolling in our expanded access program IV Amphotericin B was discontinued and the patients began treatment with oral med 22, or three and she was discharged from the hospital to continue the remainder of her treatment at home.
Importantly, the patient began to show clinical improvement following only three weeks of therapy on Matt 22, or three her renal function returned to normal and repeated MRI for scientists and brain showed no evidence of active mucormycosis infection.
<unk> repeated tests C. T scans showed a reduction in pulmonary nodules with no new lesions.
There had been no signs of any recurrent invasive fungal infections since the patients stopped using that 22 or three.
The patient continued met 22 or three for a total of 17 weeks with no evidence of toxicity.
This case represents the first ever pediatric use of map 20, 203, and an extremely compromised patient and highlights the clinical potential of <unk> 22, or three in treating these deadly infections and.
In the words of the treating physician Doctor Young Kyoung song our decision to switch this patient to Matt twenty-two three proved to be a turning point in our patient journey rapidly her gastrointestinal intolerance and renal dysfunction resolved, enabling her to continue met 22 or three therapy for an additional.
Three months.
Throughout this period, the patient displayed excellent tolerance to Matt 22, or three and subsequent imaging revealed radiological improvement of invasive fungal infections.
We are delighted with the remarkable outcome achieved with map 23, which addresses this patient is very challenging condition effectively.
We had mid teen S. Appreciate the participation of Doctor song and nationwide Children's hospital in our program along with all the patients and physicians who have participated in our clinical development program. Thus far.
Our expanded access program is attracting additional an increasing interest as Matt 22, or three continues to demonstrate efficacy in these most challenging cases and this program presents opportunities for us to generate additional meaningful clinical data outside of the clinical trial setting.
We continue to evaluate requests for access where the compassionate use of map 22, or three may help patients without other treatment options.
Now I'd like to turn the call over to Keith Kucinski to review our financial performance Keith.
Thank you Terry.
Starting today with our second quarter results.
We reported no revenue for the second quarter of 2023.
This compares with revenue of $1.1 million for the second quarter of 2022.
Which was generated from our research collaboration with bio <unk> Tec.
Total costs and expenses for the second quarter of 2023 were $6 $2 million compared with $7 million for the second quarter of 2022.
The decrease was primarily attributable to lower manufacturing costs of clinical trial materials, and lower clinical consulting fees, partially offset by a higher head count related expenses.
The company's net loss for the second quarter of 2023 was $6 $1 million or three cents per share.
This compares with a net loss for the second quarter of 2022, a 5.9 million also <unk> <unk> per share.
Turning now to our six months results.
Revenue for this first six months of 2023, and 2022 was $1 $1 million for each period.
Total costs and expenses for the first half of 2023 were $12 $8 million versus $14 $7 million for the first half of 2022.
The company's net loss for the first six months of 2023 was $11 $6 million or five cents per share.
This compares with a net loss for the first six months of 2022 of $11 $9 million or six cents per share.
Our cash cash equivalents and marketable securities as of June 30th two.
2023 were $22 $5 million.
Based on our current projections, we believe our cash is sufficient to fund planned operations into the third quarter of 2024.
As previously mentioned, we are actively seeking to extend our cash runway by securing non dilutive funding from potential third party development partners and government grant programs through agencies such as BARDA.
As well as from proceeds from potential public or private equity offerings.
With that I'll turn the call back to Jerry.
Thanks Keith.
Matt 22, or three is it's changing patients' lives. Despite some of the regulatory challenges associated with the pathway to achieving a first line unrestricted treatment indication for an invasive fungal infection, which arguably should not be applicable to an amphotericin b product, we continue to be encouraged by data and <unk>.
Guided by the positive clinical experience of those patients demonstrating the highest unmet medical need our team is already working hard to outline the best path forward, which we believe could result in an L pad approval from at 20 203.
And our clinical success to date with about 22, or three and the continuing and growing patient need we remain confident that there will be sources of non dilutive funding to continue to advance this life changing therapy.
Our mission with Matt 22, or three is clear.
Take advantage of the opportunity that the LNC platform has provided to formulate a safer amphotericin b, which can be used by patients in the longer term treatment of invasive fungal infections and give patients to therapy. They deserve.
Building on our success with Matt 22, or three we are very excited about our ongoing internal LNP based RNA AI program.
Distinguishable from the approach we were forced to take with messenger RNA do it due to its size and sensitivity are small oligonucleotide program is utilizing L. N ceased to deliver these molecules.
With successful in vitro testing demonstrating efficient delivery with measurable knockdown of inflammatory markers behind us.
We now move move aggressively forward into in vivo studies with enhanced confidence based on our clinical experience with <unk> 20 203.
We have done a lot of work to understand preferential cellular uptake and see these LNP formulations behaving much like our small molecule L N C's.
With uptake by macrophages neutrophils and other activated immune cells.
As a result, we are naturally exploring inflammatory targets as an initial step.
We are working with our partners to generate in vivo data evaluating biological activity with these LNC oral formulations and we expect data later in 2023.
We believe that a successful demonstration of in vivo efficacy in one or more of these studies would represent a first for the oral delivery of small oligonucleotides.
We're grateful for your continued support and we look forward to continuing to keep you apprised of our progress.
With that review I'll now turn the call over to the operator for questions and answers Paul.
Thank you well now be conducting a question and answer session. If you'd like to ask a question. Please press star one on your telephone keypad.
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One moment, please while we poll for questions.
While we're waiting for that first question Paul I'd, just like to mention that next month, we'll be participating in the H C Wainwright Global investment conference.
Webcast of our presentation will go live on the IR calendar page of the Companys website honor around Monday September 11th and will also be participating in the Dawson James small cap growth conference being held October 12th in Jupiter, Florida.
Ah Okay, Paul I think we're ready for that first question.
Thank you. Our first question is from Julian Harrison with B T. I G. Please proceed with your question.
Alright, Thank you for taking my questions and congrats on the progress here on the first line study for 'twenty, two or three I'm wondering if you could provide any more details on what the active control group with likely look like and is that different at all from the alternative study designs and then on the alternative study design sports specifically.
Can you talk more about these high need subsets being considered what the corresponding study designs would likely look like and what the corresponding market opportunities like we are.
Yeah, So Julien it's a great question and thanks for being on the call today I'll, let Dr. Matthew but it has to go into some detail, but I bet I do want to be clear that we're still refining what those patient population targets look like when the non inferiority margin would look like in the statistics, but we certainly can talk a little bit about what we understand could be enact.
If control in these patients and obviously there would be a distinction between those patients who could tolerate diesel therapy for which an active control you know could be appropriate and then those patients who are azole resistant or ezola tolerant.
You know for which there there wouldn't be an active control and you would be relying on some sort of external control. So Terry before we sort of go into alternate designs do you want to just comment briefly on the controls.
Sure Jerry and thanks for your question Julien So from a control perspective to build upon what Jerry mentioned if.
If we include a a design that's really focused on the intolerant patient population.
Control could be a best of care control arm in which patients would be managed as they are today, if intolerant or unable to take as many of these patients are leveraging longer courses of IV amphotericin and in many cases that longer course could require.
Some dose adjustment of the IV amphotericin dose that's administered and it requires careful monitoring for for renal function. Another potential alternatives. If we look at the intolerant patient as a.
External control arm consideration would be looking at outside data outside of the clinical trial to really leverage what's available historically, which does.
Can provide a high regulatory hurdle, it's using external data, but those are the two auctions that were weighing right now as a company are vis vis the control arm for that trial.
And then Julian importantly, you know when you think about.
The opportunity and the pursuit of a restricted label versus an unrestricted label, you're talking about more openness on behalf of FDA in the case of an active control portion of that study to change their perspective on the non inferiority margin.
For that patient population, that's where they're going to take into account the patients with the highest need and that's what underscores our belief that pursuing a study in with this design with the ultimate intention of getting a restricted label for this patient population. It eases those restrictions on the non.
Inferiority margin and that's really what's driving for example, 700 patients for first line unrestricted.
Therapy, and so it's the combination of those two things along with some of the other benefits afforded by the El pen pathway that we think can streamline development.
Development of <unk> 22 or three.
Okay, great. Thanks very much.
Yeah.
Thank you there are no further questions at this time I would like to hand, the floor back over to Jerry Jabbour for any closing comments.
Thank you Paul and thank you to everyone for joining us today and for your interest in <unk>. We really are excited about our company's future. We're proud of the impact we're making in patients with Matt 22, or three and we're really excited about the potential that our LNC platform has and perhaps the first ever oral delivery of small.
Oligonucleotides.
So some exciting things coming up in the second half of this year, we're working as hard as we can to advance our programs, we're being smart with our funds we.
You know we are looking at alternative ways to extend our cash runway given the challenges in the capital markets today.
You know we are evaluating every opportunity to maximize the value associated with this platform, it's making that dramatic impact in patients today, and we think of that as an opportunity because of the way the platform behaves similarly, with small oligos to small molecules. We believe that we could be on the cusp of trying to.
Crack that code of the oral delivery of small oligonucleotides more to come on that the science is hard no one's been able to deliver a nucleic acids orally, we understand the challenges.
Specifically around messenger RNA, we know what adjustments, we had to make to our L. N seas in order.
To deliver messenger RNA systemically those adjustments weren't necessary for small oligos and so we believe they behave much more like lipid nano our traditional lipid nano crystal and and we will get to these in vivo experiment and we'll let the data speak speak for themselves, but thank you again for joining us enjoy the rest of your summer.
We hope to see you at upcoming Investor conferences in the fall have a great day.
This concludes today's conference you may disconnect your lines at this time. Thank you for your participation.
Okay.
Yeah.