Q4 2023 Quoin Pharmaceuticals Ltd Earnings Call & Business Update
Operator: Good day, and welcome to the Quoin Pharmaceuticals fourth quarter financial results and business update conference call. All participants will be in listen-only mode.
Good day and welcome to the Cohen Pharmaceuticals fourth quarter financial results and business update conference call all participants will be in listen only mode.
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Operator: To withdraw your question, please press star- Please note, today's event is being recorded. I would now like to turn the conference over to Gordon Dunn, Chief Financial Officer. Please go ahead.
To withdraw your question. Please press Star then two.
Please note today's event is being recorded.
I would now like to turn the conference over to Gordon Dunn Chief Financial Officer. Please go ahead Sir.
Gordon Bruce Dunn: Thank you. Good morning. We appreciate you joining us on today's conference call. With me on the call are Dr. Michael Myers, CEO, and Denise Carter, COO. We're pleased to provide an update on our progress for the fourth quarter of 2023, as well as discuss our Q4 and full year 2023 financial results. Please note that our Operations and Financial Results press release is now available on our website. In keeping with normal procedure, Michael will first provide an operations update, following which I will review our financial results. I will then hand the call back to Michael for closing comments before we open the phone lines for questions. I'd like to remind everyone that statements made during this conference call will include forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties that could cause actual results to differ materially from the information expressed or implied by these forward-looking statements. For more information regarding such risks and uncertainties, please see the risk factors outlined in the company's filings with the SEC, and any forward-looking statements are made only as of today.
Thank you and good morning, we appreciate you joining us on today's conference call.
With me on the call are Dr. Michael Meyers, CEO and Denise Carter CFO.
Pleased to provide an update on our progress in the fourth quarter of 2023 as well as discussing our Q4 and full year 2023 financial results.
Please note that our operations and financial results press release is now available on our website.
In keeping with normal procedure, Michael will first provide an operations update filing which I will review our financial results I will then hand, the call back to Michael for closing comments before we open the phone lines for questions.
I'd like to remind everyone that statements made during this conference call will include forward looking statements within the meaning of the private Securities Litigation Reform Act of 1995, which involve risks and uncertainties, which could cause actual results to differ materially from the information expressed or implied by these forward looking statements for more information regarding such risks and uncertainties. Please see the risk factor.
<unk> outlined in the company's filings with the SEC and any forward looking statements are made only as of today, we disclaim any obligation to update these forward looking statements other than as required by law. Please.
Gordon Bruce Dunn: We disclaim any obligation to update these forward-looking statements other than as required by law. Please see the forward-looking statement section in our financial results release issued this morning for more information. It is now my pleasure to turn the call over to our CEO, Michael Myers. Thank you, Gordon. Good morning, everyone.
Please see the forward looking statements section in our financial results release issued this morning for more information.
It is now my pleasure to turn the call over to our CEO Michael Meyers.
Thank you Gordon and good morning, everyone and welcome to today's call. We are pleased to have the opportunity to report on our coins progress in the past quarter and throughout the year as a whole.
Michael Myers: And welcome to today's call. We are pleased to have the opportunity to report on Quoin's progress in the past quarter and throughout the year as a whole. 2023 was quite simply a truly transformational year for Quoin.
2023 was quite simply a truly transformational year for calling.
Michael Myers: During that year, we released the first ever clinical data generated from a trial in Nettleson Syndrome subjects being conducted under an open investigational new drug application, RIND, with the U.S. FDA. This was a significant and important milestone, not just for Quoin but for the Neffesen community as a whole. I'm pleased to say that this initial data was encouraging and overall positive across a number of clinical endpoints. And crucially, there was also a complete absence of any safety signals whatsoever.
During that year, we released the first ever clinical data generated from a trial in <unk> syndrome subjects being conducted under an open investigational new drug application or <unk> with the U S. F T.
This was a significant and important milestone not just for calling but further invest the same community as a whole.
I'm pleased to say that this initial data was encouraging and overall positive across a number of clinical endpoints and crucially. There was also a complete absence of any safety signals whatsoever.
Michael Myers: On the strength of the combination of both this initial efficacy and safety data that had been generated, we submitted to our open IND a number of proposed protocol amendments to both ongoing clinical trials. These included increasing the size of both studies, changing the dosing frequency from once daily to twice daily, and eliminating the lower 2% dose from our blinded studies. Those amendments were cleared by the FDA and have now been fully implemented. Earlier this year, we submitted yet another protocol amendment to the FDA. On this occasion, we proposed lowering the eligibility age for recruitment into our clinical studies to 14 years and older from 18 years.
On the strength of the combination of both this initial efficacy and safety data.
Had been generated we submit it to our open 90, a number of proposed protocol amendments to both ongoing clinical trials.
These included increasing the size of both studies changing the dosing frequency from once daily to twice daily.
And eliminating the lower 2% dose from a blinded study.
Those amendments were cleared by the FDA and have now been fully implemented.
Earlier this year, we submitted yet another protocol amendment to the FDA on this.
Occasionally we proposed lowering the eligibility age for our recruitment into our clinical studies 14 years and older from 18 years and older. This is another extremely important milestone for our company and once again further analysis and community.
Michael Myers: This is another extremely important milestone for our company and, once again, for the Nesitin community, as it marks the very first time that non-adult Nesitin subjects will be tested in a clinical study that is being conducted under an open-eye environment. As announced on March 4th, this latest protocol amendment was also cleared for implementation by the FDA. It's worth mentioning that each of those protocol amendments I just discussed were made from a position of strength on the basis of positive initial efficacy and safety data, while there is always a temptation to not amend protocols. When studies are ongoing, we took the position that while this initial data was indeed positive and encouraging, we should take advantage of the opportunity to generate an even more robust data set with a greater likelihood of approval and with a broad label. We believe these new protocol amendments have the potential to deliver just that.
Box, the very first time that non adult and adolescent subjects will be tested in a clinical study that is being conducted under an open IND D.
As announced on March 4th this latest protocol Amendment was also cleared for inflammation implementation by the FDA.
It's worth mentioning that each of those protocol Amendment I just discussed were made from a position of strength on the basis of positive initial efficacy and safety data.
While there is always the temptation to northern Maine protocols.
What studies are ongoing we took the position that was this initial data was indeed positive and encouraging we should take advantage of the opportunity to generate an even more robust dataset with a greater likelihood of approval.
And with a broad label.
We believe these new protocol amendments have the potential to deliver just that.
Michael Myers: The inclusion of children in our studies is especially noteworthy as it significantly increases the number of eligible subjects and could lead to a faster recruitment rate overall. Because children will be eligible to participate in both the blinded and open-label studies, we will be able to accommodate patients who are currently receiving off-label systemic therapy and those who are not being treated in this way. This is a very important consideration for parents and caregivers as it eliminates the need for them to have to make the potentially agonizing decision about whether or not to discontinue their loved ones' off-label treatment, which may be providing some symptomatic relief, in order to enroll them in a clinical trial where they might end up receiving just a placebo and run the risk of their original symptoms coming roaring back.
The inclusion of children into our studies is especially noteworthy as it significantly increases the number of eligible subjects and could lead to a faster recruitment rates overall.
Because children will be eligible to participate in both the blinded and open label studies, we will be able to accommodate.
Those patients who are currently receiving off label systemic therapy, and those who are not being treated in this manner.
This is a very important consideration for parents and caregivers as it eliminates the need for them to have to make the potentially agonizing decision about whether or not to this this continue their loved ones off label treatment.
Which may be providing some symptomatic relief in order to enroll them in a clinical trial, where it might end up receiving just to placebo and run the risk of their original symptoms coming roaring back.
Michael Myers: By concurrently running studies where QRX003 is being tested as monotherapy and in conjunction with off-label systemic therapy, we believe we are laying the foundation for the generation of a truly broad-based data set that will give physicians the widest possible range of treatment options for people suffering from this terrible disease. With all of these protocol amendments now fully implemented, 2024 is shaping up to be a pivotal year for our company as we move forward with our mission to deliver the first approved treatment, by necessity. We look forward to providing updates on our continued progress with the clinical program throughout the year ahead. On the commercial front, we have continued to lay the groundwork for the establishment of our own infrastructure to support our plans to sell the product ourselves in both the U.S. and Western Europe once approved. As noted previously, we believe that given the relatively small patient population, the limited number of treatment centers and treating physicians, that a small, compact commercial infrastructure that is heavily weighted in medical affairs and along with strategically placed regional sales reps could efficiently and effectively sell this project. We also plan to provide updates on these activities on subsequent calls throughout. Supporting our own commercial efforts, as you have heard from us in the past, we have now entered into nine commercial partnerships for QRX003 that cover 61 countries, and we will remain in discussions to expand that number to the few remaining unlicensed countries across the world.
But concurrently running studies, where cure <unk> three is being tested as a monotherapy and in conjunction with off label systemic therapy. We believe we are laying the foundation for the generation of a truly broad based dataset that will give physicians the.
The widest possible range of treatment options for people suffering from this terrible disease.
With all of these protocol amendments now fully implemented 2024 is shaping up to be a pivotal year for our company as we move forward with our mission to deliver the first approved treatment for <unk> syndrome.
We look forward to providing updates on our continued progress with the clinical program throughout the year ahead.
On the commercial front, we have continued to lay the groundwork for the establishment of our own infrastructure to support our plans to sell the product ourselves in both the U S and Western Europe. Once approved as noted previously we believe that given the relatively small.
<unk> population the limited number of treatment centers and treating physicians that are small compact commercial infrastructure that is heavily weighted in medical affairs, and along with strategically placed regional sales reps could efficiently and effectively detailed.
Product, we also plan to provide updates on <unk>.
These activities on subsequent calls throughout the year.
Supporting our own commercial efforts as you have heard from us in the past we have now entered into nine commercial partnerships for <unk> zero three that cover 61 countries.
And we remain in discussions to expand that number to the few remaining unlicensed countries across the world.
Michael Myers: We continue to believe that this thoughtful, diligent, and systematic approach to entering into commercial partnerships will ensure that QRX003 becomes available to as many necessary patients in as many countries as possible once it is approved. As noted previously, these partnerships, along with our own planned commercial infrastructure for the U.S. and Western Europe, will facilitate what will effectively amount to a global launch of the product once approved, and also lay the foundation for similar global launches of our pipeline products once they also receive approval. We firmly believe that this global commercial network is unprecedented for a company our size and represents a highly differentiated and perhaps somewhat underappreciated advantage. The potential incremental revenue-generating opportunity that this global commercial partnership network represents for Quoin, all of which will fall directly to the bottom line, could ultimately have a material positive impact on our future profitability.
We continue to believe that this thoughtful diligent and systematic approach to entering into commercial partnerships will ensure that QR <unk> 003 becomes available to as many <unk> patients in as many countries as possible losses.
It is approved.
As noted previously these partnerships along with our own planned commercial infrastructure for the U S and Western Europe will facilitate what will effectively amounts to a global launch of the product once approved and also lay the foundation for similar global launches of <unk>.
Pipeline products once they also receive approval.
We firmly believe that this global commercial network is unprecedented for a company our size and represents a highly differentiated and perhaps somewhat underappreciated underappreciated advantage for point.
Potential incremental revenue generating opportunity that this global commercial partnership network represents for calling all of which will fall directly to the bottom line could ultimately have a material positive impact on our future profitability.
Michael Myers: During our previous calls, I have also updated everyone on our M&A strategy. As discussed, given our strong balance sheet and potential access to additional capital, we have been acutely focused on expanding our product portfolio via acquisition, licensing, or other means. We continue to search for M&A opportunities, and it remains a priority for us.
During our previous calls.
I have also updated everyone on our M&A strategy.
As discussed given our strong balance sheet and potential access to additional capital we have been acutely focused on expanding our product portfolio via acquisition.
In licensing or other means.
We continue to search for M&A opportunities.
It remains a priority for us, but I do want to caution we will not execute a deal unless it really makes sense for us as a company to do so.
Michael Myers: But I do want to caution that we will not execute a deal unless it really makes sense for us as a company. Last week, we completed a successful public offering, raising gross proceeds of $6.5 million. In addition...
Last week, we completed a successful public offering raising gross proceeds of $6 $5 million. In addition.
Michael Myers: Earlier this year, we entered into an $8 million equity line of credit, or ELOC arrangement, with alumni capital, which we will be able to access in the second half of. With the proceeds of last week's offering alone, we are now funded into the second half of 2025. And the proceeds from the equity line of credit will extend our cash runway even further. Finally, as we have noted on our three previous calls, another company has filed an IND with the FDA and received a study may proceed letter to initiate the clinical development of their product as a potential treatment for nephritis and syndrome. As of yesterday, following a review of the clinicaltrials.gov website, it appears that this study has not yet been initiated, and Quoin remains the only pharmaceutical company actively conducting clinical studies in Nesitin syndrome under an open IND.
Our year. This year, we entered into an $8 million equity line of credit our HELOC arrangement with alumni catheter, which we will be able to access in the second half of this year.
With the proceeds of the of last week's offering alone. We are now funded into the second half of 2025.
And the proceeds from the equity line of credit will extend our cash runway even further.
Finally, as we have noted now on our three previous calls another company as filed 90 with the FDA and received the study May proceed letter to initiate the clinical development of their products as a potential treatment for <unk> syndrome.
As of yesterday following a review of the clinical trials Dot Gov website. It appears that this study has not yet been initiated and coin remains the only pharmaceutical company actively conducting clinical studies in <unk> syndrome under an open IND.
Gordon Bruce Dunn: Furthermore, as we have previously noted, whilst other companies have also spoken for some time either about filing INDs or initiating nested and clinical studies, we continue to see a consistent pattern of those timelines either being pushed further and further out or, in some cases, abandoned completely, as we continue to make significant progress on the clinical front. We and our nine global marketing partners are more excited than ever by the potential commercial opportunity of what could be the first regulatory-approved treatment for this horrendous disease. I will now hand the call over to Gordon. Thank you, Michael. As of December 31, our cash and marketable securities were approximately $10.7 million compared to $14 million as of September 30.
Furthermore, as we have previously noted was auto companies have also spoken for some time either about filing IND.
We're initiating NASA in clinical studies, we continue to see a consistent pattern of those timelines being pushed farther and farther out or in some cases, a bad them completely.
As we continue to make significant progress on the clinical front.
Our nine global marketing partners are more excited than ever by the potential commercial opportunity of what could be the first regular regulatory approved treatment for this horrendous disease.
I will now hand, the call over to Gordon.
Thank you Michael.
As of December 31, our cash and marketable securities was approximately $10 7 million compared to $14 million.
As of September 30th.
Gordon Bruce Dunn: As Michael highlighted last week, we strengthened our cash position through the successful completion of a $6.5 million public offering, which we expect will be sufficient to fund our operations into the second half of 2025. And we've also entered into an $8 million Equity Loan and Credit transaction, which we'll be able to access and complete in the second half of this year. And the completion of this $8 million transaction will further extend our cash position into the second half of 2026. Our net loss for the year ending December 31, 2023, was $8.7 million, compared to $9.4 million in 2022. The decrease in 2023 was primarily due to higher interest income and lower professional fees and other G&A costs, partially offset by higher R&D expenses as we advanced our clinical study. Our net loss was $2 million in both of the fourth quarter of 2023 and the fourth quarter of 2022. I'll now turn the call back to Michael to make some closing remarks and begin our Q&A.
As Michael highlighted last week, we strengthened our cash position through the successful completion of a $6 5 million public offerings, which we expect will be sufficient to fund our operations into the second half of 2025.
And we've also entered into an $8 million relented credit transaction, which will be able to access and complete in the second half of this year and the completion of this 8 million transaction will further extend our cash position into the second half of 2026.
Our net loss for the year ending December 31, 2023 was $8 $7 million compared to $94 million in 2022.
Decrease in 2023 was primarily due to higher interest income and lower professional fees and other G&A costs, partially offset by higher R&D expenses as we advanced our clinical studies.
Our net loss was $2 million in both of the fourth quarter 2023 in the fourth quarter of 2022.
I'll now turn the call back to Michael to make some closing remarks and begin our Q&A.
Michael Myers: Thanks, Gordon. We are very pleased with the positive start the company has made to 2024. With all of the protocol amendments, we have submitted to our IND for our ongoing clinical studies, including the reduction in eligibility age to 14, now fully implemented. We believe we have put ourselves in a strong position, build off of the initial positive data that has been generated so far. Our balance sheet has been strengthened as a result of the recent successful capital raise and will be further extended by the ELOC that Gordon just discussed.
Mike.
Thanks, Gordon we are very pleased with the positive start the company has made to 2024 with all of the protocol amendments.
We have submitted to our IND for our ongoing clinical studies, including the reduction in eligibility age to 14 now fully implemented we believe we have put ourselves in a strong position to build off of the initial positive data that has been generated so far.
Our balance sheet has been strengthened as a result of the recent successful capital raise and will be further extended by the E block that Gordon just discussed.
Operator: This strong cash position will enable us to complete the clinical testing of our lead asset for Nettleson Syndrome and put the company firmly in position to achieve our mission of delivering the first approved treatment to this long-neglected community. With that, Operator, we are now ready for questions. Thank you, sir. We will now begin the question and answer session. To ask a question, you may press star then 1 on your telephone keypad to connect to a speaker phone.
This strong cash position will enable us to complete the clinical testing of our lead asset for <unk> syndrome, and put the company firmly in position to achieve our mission of delivering the first approved treatment to this long neglected community.
With that operator, we are now ready for questions.
Thank you Sir we will now begin the question and answer session.
Ask a question you May press Star then one on your telephone keypad.
If you are using a speaker phone we ask that you. Please pickup your handset before pressing the keys.
Operator: We ask: Thank you, or Hansett? To ask your question, please press star 1. Today's first question comes from Naz Rahman with, Hi, everyone. Congratulations on all the progress thus far. I have a few questions, if I may. First, I'm just starting on the pediatric patients. How many pediatric patients do you plan on enrolling in the randomized portion of the study? And based on your conversations with the FDA, how many pediatric patients do you need for the SAIT database? Thanks, Naz. Good questions. The short answer to the first part is: as many as possible. Look, this is a genetic disease, so people are born with it.
To withdraw your question. Please press Star then two.
Today's first question comes from Naus, Robyn with Maxim Group. Please go ahead.
Hi, everyone. Congrats on all the progress thus far on other few question if I may.
First wanted to start on the pediatric patients.
How many pediatric patients do you plan on enrolling in the randomized portion of the study and based on your conversations with the FDA, how many pediatric patients do you need for the safety database.
Yeah.
Good questions.
The short answer to the first part is as many as possible.
Look this is this is a genetic disease. So so people are born with it and what we've seen is that younger people.
Michael Myers: And what we've seen is that younger people have completed the genetic testing to accurately identify that they have the disease, whereas that's not so common among the older population. So, because you have to complete the testing to enroll in our study, we think that now that the eligibility age has been reduced, then..., it will skew more towards the younger population in the study than the older one. So I think we may end up with at least a 50-50 mix, or maybe slightly skewed more in favor of young people. But it's difficult to tell, but as I said, we'll recruit as many as are eligible. With regard to the number, that hasn't been discussed with the agency as yet. As you know from previous discussions, we've had a very positive relationship with the agency around this product, as we were the first company to submit an IND to them. So I don't know that they have a hard and fast number in their mind. But bear in mind, the total population that they were guiding us towards for approval was 20 at the commercial dose. And we expect that we'll significantly exceed that number and that a good proportion of those will be pediatric patients.
<unk> completed the genetic testing to accurately identify that they have the disease, whereas.
That's not so common among.
Oh the older population, so because you have to complete the testing to enroll in our study we think that now that the eligibility age has been reduced then.
It was skewed more towards the younger population in this study.
The older ones. So I think we may end up with at least 50, 50 mix or maybe slightly skewed more in favor of young people, but it's difficult to tell but as I said look.
We'll recruit as many as are eligible and.
With regard to the number.
That hasnt been discussed with the agency.
As of yet.
As you know from previous discussions we've had a very.
<unk>.
Positive relationship with the agency around this product as we were the first company to.
Submit an IND to them.
So I don't know that they have a hard and fast number in their mind bear in mind. The total population that they were guiding us towards for approval was 20 at the commercial dose.
And we expect that will significantly exceed that and they're a good proportion of those will be a pediatric patients.
Michael Myers: Got it. That was helpful. So between your two studies, the open-label and the randomized portion of the study, when can we see the next data readout, and what would be included in that? And ultimately, when do you expect or expect the randomized control portion of the trial to read out the top-line results?
Got it that was helpful. So.
Clean year to study the open label and the randomized portion of the study.
One can see the next data readout and what would be included in that and ultimately when do you see our spec for randomized controlled portion of the trial to read out the top line results.
Yeah. So.
Michael Myers: So there will be further readouts throughout the year from the Open Label Study, and what we would hope is that we'll see readouts from pediatric patients dosed twice a day because that is the target now, twice daily dosing, and we think it would be really beneficial if we could see the results on pediatric patients tested at that twice daily level. So there will be continued readouts. We would expect that this summer there will be a readout. I can't tell you what the mix between pediatric and adult patients will be, but we are targeting a first readout this summer from patients who have received twice daily dosing.
So there will be further readouts throughout the year from the open label study and what we would hope is that we'll see.
Readout from.
Pediatric patients dosed twice a day.
Because that that is the target now twice daily dosing.
And we think it would be really beneficial if we could see.
The results on pediatric patients.
Tested at that twice daily level. So so there will be continued readouts.
Would expect that this summer there'll be a readout I can't tell you what the mix between pediatric and adult patients will be.
But we are targeting.
First readout this summer from patients who have received twice daily dosing.
Michael Myers: With regard to the blinded study readout, as you know, we've increased the number of subjects to 30 in that study. So I certainly don't see an initial readout this year, but my hope is that it will be pretty early in 2025. That's probably as accurate.
With regard to the blinded study read out as you know we've increased the number of subjects to 30 in that study.
So I certainly don't see an initial readout this year, but my hope is that it would be pretty early in 2025, that's probably is accurate.
Michael Myers: Guidance I can give you at this point, Naz, look we'll refine that once the recruitment starts to ramp up, and we'll have more visibility. Got it. That was helpful. And just on that open-label readout, two quick questions. One, how have you seen, based on what you've seen so far, how have you seen, or how have patients tolerated the twice-daily doses? And two, have you seen any patients choose to discontinue systemic therapy like potassium or steroids because of the potential benefits they're getting from OO3? So, taking them in reverse order, Naz. A condition of the open-label study is that they remain on systemic therapy throughout the 12-week dosing period.
Our guidance I can give you at this point <unk>.
We'll refine that once the recruitment starts to ramp up.
And we will have more visibility.
Got it that's helpful and.
I'm just I'm not open label readout do two quick questions.
One.
How have you seen based on what you've seen so far how have you seen or how those patients tolerated the twice daily dosing and too.
Have you seen any patients choose to discontinue.
Systemic therapy.
Because of the potential benefits, they're getting from all three.
So taking them in reverse order.
As a condition of the open label study is that they remain on the systemic therapy throughout the 12 week dosing period.
Michael Myers: So, there's no... ability for them to discontinue, that they're getting benefit from 003 so that they have to stick with it, the first part. We have not seen any changes in the safety profile since we made any of the protocol amendments. So, so far, so good. There's nothing that is raising any red flags or even amber flags at this point from a safety perspective.
So there is no ability for them to discontinue.
Yeah.
If they believe that they are getting benefit from 003, so that they have to stick with it.
And the.
The first part.
We have not seen any changes in the safety profile.
<unk>.
Since we've made any of the protocol amendments.
So far so good.
There is nothing that Thats <unk>.
Brazing any any red flags or even amber flags at this point from a safety perspective.
Michael Myers: Got it. That was very helpful. Thanks for taking my questions and congrats on the progress. Thanks, Naz.
Got it that's very helpful. Thanks for taking my questions and congrats on the progress.
Thanks, guys.
Operator: Thank you. And our next question today comes from James Molloy with Alliance Global Partners. Hi guys, thanks for taking my question. It's a good question on, I think you mentioned a little bit about M&A in their prepared remarks. Could you walk through what you guys could be looking at for M&A? I know that you have a ton of extra capital lying around, but are there opportunities that fit with what you have currently?
Thank you and our next question today comes from James Moore.
Global Partners. Please go ahead.
Yeah.
Hi, guys. Thanks for taking my question just a quick question on I think you mentioned a little bit about M&A in the.
And on your prepared remarks could you walk through what you guys could be looking at for M&A I know that.
I have a ton of extra capital line around but other opportunities.
With what you have currently.
Michael Myers: Do you need to do an additional raise to get that, and what are you seeing out there? Well, thanks, Jim. Appreciate you jumping on and asking the question. Last year, as you've heard, we focused very significantly on M&A activity, and we assessed a lot of opportunities, and let's be fair, there are quite a number of distressed assets out there that are available, but we did not get to a point where we signed any deals. There were some very interesting opportunities that we continue to look at, and we will continue to do so this year. However, we will remain disciplined within the remit of rare and orphan diseases.
The traditional ways to get that or what and what are you seeing out there. Please.
Well thanks, Jim.
I appreciate youre harping on in asking the question.
Look.
Last year as you've heard we focused very significantly on M&A activity and we.
We assessed a lot of opportunities.
And let's be fair.
Theres quite a number of distressed assets out there that are available.
But we did not get to a point, where we signed a new deal. There was some very interesting opportunities that we continue to look at.
We will continue to do so this year.
We will remain disciplined within the remit of rare and orphan diseases.
Michael Myers: Our preference is for later stage, often assets that have clinical data, the closer to approval and commercialization, the better, but we will stay within the rare and often chronic disease space and not expand beyond that. So, look; we'll continue to look. We will continue to explore what's out there, and I would just say, look, stay tuned. In terms of whether we would do an additional raise to support any M&A, that depends on what the deal structure is. So, you know, you've heard us before that we don't want to spend from our cash balance on M&A. So more than likely, we would seek to do a raise, but it really does depend.
Our preference is for later stage.
Often assets that have clinical data.
The closer to approval and commercialization the better but.
But we will stay within the rare and orphan disease space and not expand beyond that so look we'll continue to look.
We will continue to explore.
Whats out there and I would just say look stay tuned in terms of whether we would do an additional raise to support any M&A.
That depends on what the deal structure is so.
You've heard us before that we don't want to spend from our cash balance.
On M&A, so more than likely we would we will seek to do a raise but it really does depend.
Michael Myers: Great, thank you. And I heard in one of the questions before, you clarified the all-adult trial, looking for early 25s in the data. Is that correct on that? Do you have a...
Okay, great. Thank you and I heard it.
A question before you clarified the all adult trial looking at early 25 on the data correct on that if you have a.
Michael Myers: Timing, updated timing on the mostly children's trial. So that is the blinded study, Jim, and that now has both adults and children aged 14 and upwards. And as I said, from where we stand today, early 2025 is the most likely time frame for initial data, whereas from the Open Label Study, which now also includes adults and children aged 14 and up, we have the opportunity to present data throughout the year as it becomes available.
Timing.
Simon on the.
Most of the children trial.
So so that that is the blinded study Jim.
And that now has both adults and children age 14 and up.
Upwards.
And as I said.
From where we stand today, we think early 2025 is the most likely.
Timeframe for initial data, whereas from the open label study, which now also does contain adults and children age 14, and up will have the opportunity to present data throughout the year as it becomes available.
Michael Myers: Okay, great. Thank you. Then, finally, the last question, any updates on when you talk to the FDA about rare pediatric orphan priority? And... sometime this year. That's about as good as I can give you. But certainly sometime this year.
Okay, great. Thanks, and then finally, the last question any updates on when.
When you talk to the FDA about where pediatric orphan priority.
Sometime this year, that's that's about as good as I can give you, but certainly sometime this year.
Michael Myers: Understood. Thank you for taking the time to ask the question. Thank you, sir. And as a reminder, ladies and gentlemen, if you'd like to ask a question, please press start. Our next question comes from Dean Finley, a private investor today. Just a quick question from the investment side. I'm just wondering if there are any relevant or valid price targets out there. I, I, I, I don't... I don't have anything to add to that. The analysts that cover us have set their price targets for the share price, so I would point you to their research reports.
Understood. Thank you for taking the questions.
Thank you Sarah.
As a reminder, ladies and gentlemen, if you'd like to ask a question. Please press Star then one.
Our next question comes from Deane suddenly a private investor. Please go ahead.
Just quick question for new investments I am just wondering if theres any relevant or valid price targets.
Aye.
I don't.
I don't have anything.
To add to that the analysts that cover us they said.
Their price targets for the share price, but.
So I would I would point you to their research reports we don't.
Michael Myers: We don't provide that. All right, thanks. And ladies and gentlemen, this concludes our question and answer session. I'd like to turn the conference back over to the management team for any questions.
We don't provide that.
Alright. Thanks.
A lot of confusion.
Gentlemen, this concludes our question and answer session I would like to turn the conference back over to the management team for any closing remarks.
Michael Myers: Thank you and thank everybody for participating again. As we always end with, please feel free to reach out to us offline by email or phone. If you have any questions or concerns, we are always available to address those. So, thank you, and have a great day. Thank you, sir. This concludes today's conference call. We thank you all for attending today's... Come in, I'll just collect your lines and have a...
Thank you and thanks, everybody for participating again, as we always and with.
Please feel free to reach out to us offline them by email or phone. If you have any questions or concerns. We are always available to to address those so thank you and have a great day.
Thank you Sir This concludes today's conference call. We thank you all for attending today's presentation.
You may now disconnect your lines and have a wonderful day.
Okay.
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