Quoin Pharmaceuticals Ltd (QNRX) 2023 Q4 法說會逐字稿

Good day, and welcome to the Quoin Pharmaceuticals' fourth-quarter financial results and business update conference call.

美好的一天，歡迎參加 Quoin Pharmaceuticals 第四季財務業績和業務更新電話會議。

(Operator Instructions) Please note, today's event is being recorded.

（操作員說明）請注意，今天的活動正在錄製中。

I would now like to turn the conference over to Gordon Dunn, Chief Financial Officer.

我現在想將會議交給財務長戈登鄧恩 (Gordon Dunn)。

Please go ahead, sir.

請繼續，先生。

Thank you, and good morning.

謝謝你，早安。

We appreciate you joining us on today's conference call.

我們感謝您參加今天的電話會議。

With me on the call are Dr. Michael Myers, CEO; and Denise Carter, COO.

與我一起參加電話會議的是執行長 Michael Myers 博士；和首席營運長丹尼斯卡特。

We're pleased to provide an update on our progress for the fourth quarter of 2023 as well as discussing our Q4 and full-year 2023 financial results.

我們很高興提供 2023 年第四季度進展的最新信息，並討論我們第四季度和 2023 年全年的財務業績。

Please note that our operations and financial results press release is now available on our website.

請注意，我們的營運和財務業績新聞稿現已在我們的網站上發布。

In keeping with normal procedure, Michael will first provide an operations update, following which I will review our financial results.

按照正常程序，邁克爾將首先提供營運最新情況，然後我將審查我們的財務表現。

I will then hand the call back to Michael for closing comments before we open the phone lines for questions.

然後，在我們打開電話線路提問之前，我會將電話轉回邁克爾以徵求結束意見。

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 can cause actual results to differ materially from the information expressed or implied by these forward-looking statements.

我想提醒大家，本次電話會議中的陳述將包括 1995 年《私人證券訴訟改革法案》含義內的前瞻性陳述，其中涉及風險和不確定性，可能導致實際結果與信息存在重大差異這些前瞻性陳述明示或暗示。

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.

有關此類風險和不確定性的更多信息，請參閱該公司向 SEC 提交的文件中概述的風險因素，並且任何前瞻性聲明僅在今天做出。

We disclaim any obligation to update these forward-looking statements other than as required by law.

除法律要求外，我們不承擔任何更新這些前瞻性聲明的義務。

Please see the forward-looking statements section in our financial results release issued this morning for more information.

請參閱我們今天上午發布的財務業績新聞稿中的前瞻性聲明部分，以了解更多資訊。

It is now my pleasure to turn the call over to our CEO, Michael Myers.

現在我很高興將電話轉給我們的執行長邁克爾·邁爾斯。

Thank you, Gordon.

謝謝你，戈登。

Good morning, everyone, and welcome to today's call.

大家早安，歡迎參加今天的電話會議。

We are pleased to have the opportunity to report on Quoin's progress in the past quarter and throughout the year as a whole.

我們很高興有機會報告 Quoin 在過去一個季度和全年的進展。

2023 was quite simply a truly transformational year for Quoin.

對 Quoin 來說，2023 年無疑是真正改變的一年。

During that year, we released the first-ever clinical data generated from a trial in Netherton syndrome subjects being conducted under an open investigational new drug application or IND with the US FDA.

那一年，我們發布了有史以來第一個臨床數據，該數據是根據美國 FDA 的開放研究性新藥申請或 IND 進行的 Netherton 綜合徵受試者試驗產生的。

This was a significant and important milestone not just for Quoin, but for a Netherton community as a whole.

這不僅對 Quoin 來說是一個重要的里程碑，對於整個 Netherton 社群來說也是如此。

I'm pleased to say that this initial data was encouraging and overall positive across a number of clinical end points.

我很高興地說，這些初步數據令人鼓舞，並且在許多臨床終點方面總體上是積極的。

And crucially, there was also a complete absence of any safety signals whatsoever.

至關重要的是，完全沒有任何安全信號。

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.

結合已產生的初步療效和安全性數據，我們向公開 IND 提交了兩項正在進行的臨床試驗的多項擬議方案修訂。

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 study.

這些措施包括增加兩項研究的規模、將給藥頻率從每天一次改為每天兩次，以及從我們的盲法研究中消除較低的 2% 劑量。

Those amendments were cleared by the FDA and have now been fully implemented.

這些修正案已獲得 FDA 批准，現已全面實施。

Earlier this year, we submitted yet another protocol amendment to the FDA.

今年早些時候，我們向 FDA 提交了另一項方案修正案。

On this occasion, we proposed lowering the eligibility age for recruitment into our clinical studies to 14 years and older from 18 years and older.

此次，我們建議將招募臨床研究的資格年齡從 18 歲及以上降至 14 歲及以上。

This is another extremely important milestone for our company.

這是我們公司的另一個極為重要的里程碑。

And once again, for the Netherton community as it marks the very first time that non-adult Netherton subjects will be tested in a clinical study that is being conducted under an open IND.

對於 Netherton 社群來說，這標誌著首次在開放 IND 下進行的臨床研究中對非成人 Netherton 受試者進行測試。

As announced on March 4, this latest protocol amendment was also cleared for implementation by the FDA.

正如 3 月 4 日宣布的那樣，這項最新的方案修正案也已獲得 FDA 批准實施。

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.

值得一提的是，我剛才討論的每項方案修正案都是基於積極的初步療效和安全性數據的優勢而做出的。

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.

雖然在研究進行時總是存在不修改方案的誘惑，但我們的立場是，雖然這些初始數據確實是積極和令人鼓舞的，但我們應該利用這個機會產生更可靠的數據集，更有可能批准並具有廣泛的標籤。

We believe these new protocol amendments have the potential to deliver just that.

我們相信這些新的協議修正案有潛力實現這一目標。

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 rate overall.

將兒童納入我們的研究尤其值得注意，因為它顯著增加了合格受試者的數量，並可能導致整體招募速度更快。

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.

由於兒童將有資格參加盲法和開放標籤研究，因此我們將能夠容納那些目前正在接受標籤外全身治療和未以這種方式接受治療的患者。

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 rolling back.

對於父母和照顧者來說，這是一個非常重要的考慮因素，因為它使他們無需做出潛在的痛苦決定，即是否停止其親人的標籤外治療，這些治療可能會提供一些症狀緩解，以便參加他們在一項臨床試驗中可能最終只接受安慰劑，並冒著原來症狀消失的風險。

But concurrently, running studies where QRX003 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 data set that will give physicians the widest possible range of treatment options for people suffering from this terrible disease.

但與此同時，正在進行 QRX003 作為單一療法並與適應證外全身治療相結合的研究，我們相信我們正在為生成真正廣泛的數據集奠定基礎，該數據集將為醫生提供盡可能廣泛的治療範圍患有這種可怕疾病的人的治療選擇。

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 Netherton syndrome.

隨著所有這些方案修正案現已全面實施，2024 年將成為我們公司的關鍵一年，我們將繼續履行我們的使命，提供第一種經批准的內瑟頓綜合徵治療方法。

We look forward to providing updates on our continued progress with the clinical program throughout the year ahead.

我們期待在未來一年提供有關臨床計劃持續進展的最新資訊。

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 US and Western Europe once approved.

在商業方面，我們繼續為建立自己的基礎設施奠定基礎，以支持我們的計劃，一旦獲得批准，我們將在美國和西歐自行銷售產品。

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.

如前所述，我們認為，鑑於患者人數相對較少，治療中心和治療醫生的數量有限，小型緊湊的商業基礎設施在醫療事務中的比重很大。

And along with strategically placed regional sales reps, could efficiently and effectively detail this product.

與策略性部署的區域銷售代表一起，可以有效率且有效地詳細介紹該產品。

We also plan to provide updates on these activities on subsequent calls throughout the year.

我們還計劃在全年的後續電話會議中提供這些活動的最新資訊。

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.

正如您過去從我們那裡聽到的那樣，為了支持我們自己的商業努力，我們現在已經為QRX003 建立了九個商業合作夥伴關係，涵蓋61 個國家/地區，我們將繼續討論將這一數字擴大到全球少數剩餘的未獲得許可的國家。

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 Netherton patients in as many countries as possible once it is approved.

我們仍然相信，這種深思熟慮、勤奮和系統的建立商業合作夥伴關係的方法將確保 QRX003 在獲得批准後能夠為盡可能多的國家的盡可能多的 Netherton 患者所使用。

As noted previously, these partnerships, along with our own planned commercial infrastructure for the US 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.

如前所述，這些合作夥伴關係以及我們自己計劃的美國和西歐商業基礎設施將促進該產品一旦獲得批准，將有效地在全球範圍內推出，並為我們在研產品在全球範圍內的類似推出奠定基礎。

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 for Quoin.

我們堅信，對於我們這樣規模的公司來說，這個全球商業網絡是前所未有的，並且對 Quoin 來說代表著高度差異化、或許有些被低估的優勢。

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.

這個全球商業合作夥伴網絡為 Quoin 帶來了潛在的增量創收機會，所有這些都將直接影響到我們的獲利，最終可能對我們未來的獲利能力產生重大的正面影響。

During our previous calls, I have also updated everyone on our M&A strategy.

在之前的電話會議中，我也向大家介紹了我們的併購策略的最新情況。

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.

正如所討論的，鑑於我們強大的資產負債表和獲得額外資本的潛力，我們一直專注於透過收購、授權或其他方式擴大我們的產品組合。

We continue to search for M&A opportunities, and it remains a priority for us, but I do want to caution that we will not execute the deal unless it really makes sense for us as a company to do so.

我們將繼續尋找併購機會，這仍然是我們的首要任務，但我確實想提醒一下，除非我們作為一家公司這樣做確實有意義，否則我們不會執行這筆交易。

Last week, we completed a successful public offering, raising gross proceeds of $6.5 million.

上週，我們成功完成了公開發行，籌集了 650 萬美元的總收益。

In addition, 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 this year.

此外，今年早些時候，我們與 Alumni Capital 達成了 800 萬美元的股權信貸額度或 ELOC 安排，我們將在今年下半年獲得該安排。

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.

僅憑上週發行的收益，我們現在就可以籌集到 2025 年下半年的資金，而股權信貸額度的收益將進一步延長我們的現金跑道。

Finally, as we have noted now 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 Netherton syndrome.

最後，正如我們在之前的三次電話會議中註意到的那樣，另一家公司已向FDA 提交了IND 申請，並收到了一份研究可能進行的信函，以啟動其產品的臨床開發，作為Netherton 綜合徵的潛在治療方法。

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 Netherton syndrome under an open IND.

截至昨天，根據 ClinicalTrials.gov 網站的審查，這項研究似乎尚未啟動，而 Quoin 仍然是唯一一家在開放 IND 下積極開展 Netherton 綜合徵臨床研究的製藥公司。

Furthermore, as we have previously noted, whilst other companies have also spoken for some time either about filing INDs are initiating Netherton clinical studies, we continue to see a consistent pattern of those timelines either being pushed further out or in some cases, abandon completely.

此外，正如我們之前指出的，雖然其他公司也已經談論了一段時間，要么申請IND，要么啟動Netherton 臨床研究，但我們仍然看到這些時間表的一致模式，要么被進一步推遲，要么在某些情況下完全放棄。

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.

隨著我們在臨床方面不斷取得重大進展，我們和我們的九個全球行銷合作夥伴比以往任何時候都更加興奮，因為這可能是第一個獲得監管機構批准的針對這種可怕疾病的治療方法的潛在商業機會。

I will now hand the call over to Gordon.

我現在將把電話轉給戈登。

Thank you, Michael.

謝謝你，麥可。

As of December 31, our cash and marketable securities was approximately $10.7 million compared to $14 million as of September 30.

截至 12 月 31 日，我們的現金和有價證券約為 1,070 萬美元，截至 9 月 30 日為 1,400 萬美元。

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.

正如邁克爾上週所強調的那樣，我們透過成功完成 650 萬美元的公開發行增強了我們的現金狀況，我們預計這將足以為我們 2025 年下半年的營運提供資金。

And we've also entered into an $8 million equity line of credit transaction, which we'll be able to access and complete in the second half of this year.

我們還達成了一項 800 萬美元的股權信貸額度交易，我們將能夠在今年下半年獲得併完成該交易。

And the completion of this $8 million transaction will further extend our cash position into the second half of 2026.

這筆 800 萬美元交易的完成將進一步將我們的現金狀況延長至 2026 年下半年。

Our net loss for the year ending December 31, 2023, was $8.7 million compared to $9.4 million in 2022.

截至 2023 年 12 月 31 日止年度，我們的淨虧損為 870 萬美元，而 2022 年為 940 萬美元。

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 studies.

2023 年的下降主要是由於利息收入增加、專業費用和其他一般管理費用減少，但隨著我們推進臨床研究，研發費用增加部分抵消了這種影響。

Our net loss was $2 million in both of the fourth quarter 2023 and the fourth quarter of 2022.

2023 年第四季和 2022 年第四季我們的淨虧損均為 200 萬美元。

I'll now turn the call back to Michael to make some closing remarks and begin our Q&A.

現在我將把電話轉回給邁克爾，讓他做一些結束語並開始我們的問答。

Michael?

麥可？

Thanks, Gordon.

謝謝，戈登。

We are very pleased with the positive start the company has made to 2024.

我們對公司 2024 年取得的積極開局感到非常高興。

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.

我們已經向 IND 提交了正在進行的臨床研究的所有方案修正案，包括將資格年齡降低至 14 歲，現已全面實施，我們相信我們已經處於有利地位，可以在最初的積極數據的基礎上進一步發展至此已經產生。

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.

由於最近成功的融資，我們的資產負債表得到了加強，並將透過戈登剛才討論的 ELOC 進一步擴大。

This strong cash position will enable us to complete the clinical testing of our lead asset for Netherton syndrome and put the company firmly in position to achieve our mission of delivering the first approved treatment to this long-neglected community.

這種強大的現金狀況將使我們能夠完成對內瑟頓綜合徵的主要資產的臨床測試，並使公司能夠堅定地實現我們的使命，即向這個長期被忽視的社區提供第一種批准的治療方法。

With that, operator, we are now ready for questions.

接線員，現在我們準備好提問了。

(Operator Instructions) Naz Rahman, Maxim Group.

（操作員說明）Naz Rahman，Maxim 集團。

Hi, everyone.

大家好。

Congrats on all the progress thus far.

祝賀迄今為止的所有進展。

I have a few questions, if I may.

如果可以的話，我有幾個問題。

First, I want to just start on the pediatric patients.

首先，我想從兒科患者開始。

How many pediatric patients do you plan on enrolling in the randomized portion of the study.

您計劃招募多少位兒科患者參加研究的隨機部分。

And based on your conversations with the FDA, how many pediatric patients do you need for the safety database?

根據您與 FDA 的對話，安全資料庫需要多少兒科患者？

Thanks, Naz.

謝謝，納茲。

Good questions.

好問題。

The short answer to the first part is as many as possible.

第一部分的簡短答案是盡可能多。

Look, this is a genetic disease.

你看，這是一種遺傳疾病。

So people are born with it.

所以人們生來就有它。

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.

我們所看到的是，年輕人已經完成了基因檢測，以準確識別他們是否患有這種疾病，而這在老年族群中並不常見。

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 a younger population in the study than the older ones.

因此，由於您必須完成測試才能參加我們的研究，因此我們認為，現在資格年齡已經降低，那麼研究中的年輕族群將比老年人更偏向。

So I think we may end up with at least 50-50 mix or maybe slightly skewed more in favor of young people.

所以我認為我們最終可能會得到至少 50-50 的混合，或者可能稍微偏向年輕人。

But it's difficult to tell.

但這很難說。

But as I said, we will recruit as many as are eligible.

但正如我所說，我們將招募盡可能多的符合條件的人。

With regard to the number, that hasn't been discussed with the agency as of yet.

至於具體數字，目前尚未與該機構進行討論。

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.

正如您從先前的討論中了解到的，我們與該產品的機構建立了非常積極的關係，因為我們是第一家向他們提交 IND 的公司。

So I don't know that they have a hard and fast number in their mind.

所以我不知道他們心裡有一個固定的數字。

Bear in mind the total population that they were guiding us towards for approval was 20 at the commercial dose.

請記住，他們指導我們批准商業劑量的總人口為 20 人。

And we expect that will significantly exceed that and that a good proportion of those will be pediatric patients.

我們預計這一數字將大大超過這個數字，其中很大一部分將是兒科患者。

Got it.

知道了。

That was helpful.

這很有幫助。

So between your two studies, the open-label and the randomized portion of the study, when can we see the next data readout?

那麼，在您的兩項研究（開放標籤研究和隨機研究部分）之間，我們什麼時候可以看到下一個資料讀數？

And what would be included in that?

其中會包含什麼？

And ultimately, when do you see or expect the randomized controlled portion of the trial to read out the top-line results?

最終，您什麼時候會看到或期望試驗的隨機對照部分讀出最重要的結果？

Yeah.

是的。

So there will be further readouts throughout the year from the open-label study.

因此，全年都會有來自開放標籤研究的進一步讀數。

And what we would hope is that we'll see readout from pediatric patients dosed twice a day because that is the target now, twice daily dosing.

我們希望我們能看到每天兩次給藥的兒科患者的讀數，因為這是現在的目標，每天兩次給藥。

And we think it would be really beneficial if we could see the results on pediatric patients tested at twice daily level.

我們認為，如果我們能夠看到兒科患者每天兩次測試的結果，那將非常有益。

So there will be continued readouts.

所以將會有持續的讀數。

We would expect that this summer, there will be a readout.

我們預計今年夏天將有一個讀數。

I can't tell you what the mix between pediatric and adult patients will be.

我無法告訴您兒科和成人患者之間的混合。

But we are targeting a first readout this summer from patients who have received a twice-daily dosing.

但我們的目標是今年夏天對接受每日兩次給藥的患者進行首次讀數。

With regard to the blinded study readout, as you know, we've increased the number of subjects to 30 in that study.

關於盲法研究讀數，如您所知，我們已將研究的受試者數量增加到 30 名。

So I certainly don't see an initial readout this year, but my hope is that it would be pretty early in 2025.

因此，我今年肯定不會看到初步結果，但我希望 2025 年早些時候就會出現。

That's probably as accurate guidance I can give you at this point, Naz.

這可能是我目前可以給你的最準確的指導，納茲。

We'll refine that once the recruitment starts to ramp up, and we'll have more visibility.

一旦招聘開始增加，我們將對其進行完善，並且我們將擁有更多的知名度。

Got it.

知道了。

That's helpful.

這很有幫助。

And just on that open-label readout, two quick questions.

就在開放標籤讀數上，有兩個簡單的問題。

One, how have you seen -- based on what you've seen so far, how have patients tolerated the twice daily dosing?

第一，根據您迄今為止所看到的情況，您如何看待患者對每日兩次給藥的耐受性？

And two, have you seen any patients choose to discontinue systemic therapy or [corticosteroids] because of the potential benefits you're getting from 003?

第二，您是否見過任何患者因為 003 的潛在益處而選擇停止全身性治療或[皮質類固醇]？

So taking them in reverse order, Naz, as condition of the open-label study is that they remain on the systemic therapy throughout the 12-week dosing period.

因此，Naz，以相反的順序服用它們，因為開放標籤研究的條件是它們在整個 12 週的給藥期間保持全身治療。

So there's no ability for them to discontinue if they believe that they're getting benefit from 003.

因此，如果他們認為自己從 003 年中獲益，他們就沒有能力停止拍攝。

So they have to stick with it.

所以他們必須堅持下去。

And the first part, we have not seen any changes in the safety profile since we made any of the protocol amendments.

第一部分，自從我們對協議進行任何修改以來，我們沒有看到安全狀況有任何變化。

So far, so good.

到目前為止，一切都很好。

There's nothing that's raising any red flags or even amber flags at this point from a safety perspective.

從安全角度來看，目前還沒有任何危險訊號甚至黃色訊號。

James Molloy, Alliance Global Partners.

詹姆斯·莫洛伊，聯盟全球合作夥伴。

Hi, guys.

嗨，大家好。

Thanks for taking my question.

感謝您提出我的問題。

Just a quick question on -- I think you mentioned a little bit about M&A on your prepared remarks.

我想問一個簡單的問題——我認為您在準備好的演講中提到了一些有關併購的內容。

Could you walk through what you guys could be looking at for M&A?

您能否介紹一下你們可能會考慮哪些併購事項？

I know that a ton of extra capital line around, but are there opportunities that fit with what you have currently?

我知道周圍有大量額外的資本額度，但是有適合您目前擁有的機會嗎？

Or if you can -- traditional ways to get that?

或者如果可以的話——用傳統的方法來實現這一點？

And what are you seeing out there, please?

請問你在外面看到了什麼？

Well, thanks, Jim.

嗯，謝謝，吉姆。

I appreciate you hopping on and asking the question.

我很感謝你跳上來問這個問題。

Look, last year, as you've heard, we focused very significantly on M&A activity, and we assessed a lot of opportunities.

看，去年，正如您所聽到的，我們非常關注併購活動，我們評估了很多機會。

And let's be fair, there's quite a number of distressed assets out there that are available, but we did not get to a point where we signed any deals.

公平地說，有相當多的不良資產可供使用，但我們還沒有達到簽署任何交易的程度。

There were some very interesting opportunities that we continue to look at.

我們繼續關註一些非常有趣的機會。

And we will continue to do so this year.

今年我們將繼續這樣做。

We will remain disciplined within the remit of rare and orphan diseases.

我們將在罕見疾病和孤兒疾病的範圍內保持紀律。

Our preference is for later-stage orphan assets that have clinical data.

我們更傾向於具有臨床數據的後期孤兒資產。

The closer to approval and commercialization, the better.

離批准和商業化越近越好。

But we will stay within the rare and orphan disease space and not expand beyond that.

但我們將停留在罕見和孤兒疾病領域，不會擴展到其他領域。

So look, we'll continue to look.

所以看，我們會繼續看下去。

We'll continue to explore what's out there.

我們將繼續探索那裡有什麼。

And I would just say, look, stay tuned in terms of whether we would do an additional raise to support any M&A.

我只想說，請繼續關注我們是否會額外籌集資金以支持任何併購。

That depends on what the deal structure is.

這取決於交易結構。

So you've heard us before that we don't want to spend from our cash balance on M&A.

所以您之前聽說過我們不想將現金餘額用於併購。

So more than likely, we will seek to do a raise, but it really does depend.

因此，我們很可能會尋求加薪，但這確實取決於情況。

The question before, you clarified the all-adult trial, looking for early '25 on the data.

先前的問題，您澄清了所有成人試驗，尋找 25 年初的數據。

Is that correct on that?

對此正確嗎？

Do you have a timing, updated timing of the mostly children trial?

您是否有以兒童為主的試驗的最新時間安排？

So that is the blinded study, Jim.

這就是盲法研究，吉姆。

And that now has both adults and children aged 14 and upwards.

現在包括成人和 14 歲及以上的兒童。

And as I said, from where we stand today, we think early 2025 is the most likely time frame for initial data, whereas from the open-label study, which now also does contain adults and children aged 14 and up, we have the opportunity to present data throughout the year as it becomes available.

正如我所說，從我們今天的立場來看，我們認為 2025 年初最有可能獲得初始數據，而從現在也包含成人和 14 歲及以上兒童的開放標籤研究來看，我們有機會提供全年可用的數據。

Okay.

好的。

Great.

偉大的。

And then finally, last question.

最後，最後一個問題。

Any updates on when you talk to the FDA about rare pediatric, orphan, a priority?

您與 FDA 討論罕見兒科、孤兒等優先事項時有什麼最新消息嗎？

Sometime this year.

今年的某個時候。

That's about as good as I can give you.

這就是我能給你的最好的了。

But certainly sometime this year.

但肯定是今年的某個時候。

(Operator Instructions) Dean Finley, Private Investor.

（操作員說明）Dean Finley，私人投資者。

Good day.

再會。

Just a quick question from the investment side.

只是投資方面的簡單問題。

I'm just wondering if there's any relevant or valid price targets out.

我只是想知道是否有任何相關或有效的價格目標。

I don't have anything to add to that.

我沒有什麼要補充的。

The analysts that cover us, they set their price targets for the share price, but so I would point you to their research reports.

報告我們的分析師為股價設定了價格目標，但我會向您推薦他們的研究報告。

We don't provide that.

我們不提供這個。

Ladies and gentlemen, this concludes our question-and-answer session.

女士們先生們，我們的問答環節到此結束。

I'd like to turn the conference back over to the management team for any closing remarks.

我想將會議轉回給管理團隊進行總結發言。

Thank you, and thanks, everybody, for participating again.

謝謝大家，謝謝大家再次參與。

As we always end with, please feel free to reach out to us, offline, by email, or phone.

正如我們最後所說的那樣，請隨時透過電子郵件或電話離線與我們聯繫。

If you have any questions or concerns, we are always available to address those.

如果您有任何問題或疑慮，我們隨時可以解決這些問題。

So thank you and have a great day.

謝謝您，祝您有美好的一天。

Thank you, sir.

謝謝您，先生。

This concludes today's conference call.

今天的電話會議到此結束。

We thank you all for attending today's presentation.

我們感謝大家參加今天的演講。

You may now disconnect your lines and have a wonderful day.

您現在可以斷開線路並度過美好的一天。