CTI Biopharma Corp (CTIC) 2022 Q4 法說會逐字稿

Good morning, and welcome to the CTI BioPharma Fourth Quarter and Year-End 2022 Financial Results and Corporate Update Conference Call. As a reminder, this conference call is being recorded. I would now like to introduce your host, Remy Bernarda, CTI Investor Relations. Please go ahead.

早上好，歡迎來到 CTI BioPharma 第四季度和 2022 年底財務業績和公司更新電話會議。提醒一下，正在錄製此電話會議。我現在想介紹一下您的主持人 Remy Bernarda，CTI 投資者關係部。請繼續。

Good morning, everyone, and thank you for joining us on the call today.

大家早上好，感謝您今天加入我們的電話會議。

Before we begin, please note that during this call, we will be making forward-looking statements safety current expectations. Such statements are within the meaning of the safe harbor provision of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the types of statements identified as forward-looking in our most recent annual report on Form 10-K and our subsequent periodic reports filed with the SEC, which are available on our website in the Investors section.

在我們開始之前，請注意，在本次電話會議期間，我們將做出前瞻性陳述以確保當前的預期。此類陳述符合 1995 年《私人證券訴訟改革法案》安全港條款的含義，包括但不限於我們最近的 10-K 表格年度報告中確定為前瞻性的陳述類型和我們的隨後向 SEC 提交的定期報告，可在我們網站的“投資者”部分獲取。

Such forward-looking statements, which are indicated by terms such as expect, intend and seek represent our views as of the date of this call are not guarantees of future performance and are subject to risks and uncertainties that may cause actual results to differ materially from those anticipated by the forward-looking statements, including many that are beyond our control.

此類前瞻性陳述以預期、打算和尋求等術語表示，代表我們截至本次電話會議之日的觀點，並不是對未來業績的保證，並且存在可能導致實際結果與實際結果大不相同的風險和不確定性前瞻性陳述所預期的那些，包括許多我們無法控制的。

These statements include our expectations regarding cash runway, timing to profitability, market adoption of VONJO and the future success of our product launch.

這些陳述包括我們對現金跑道、盈利時間、VONJO 的市場採用以及我們產品發布的未來成功的預期。

In addition, any forward-looking statements represent our views as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements in the future, we specifically disclaim any intent to do so even if our views change.

此外，任何前瞻性陳述代表我們截至今天的觀點，不應被視為代表我們截至任何後續日期的觀點。雖然我們可能會選擇在未來更新這些前瞻性陳述，但我們明確否認任何這樣做的意圖，即使我們的觀點發生變化。

For a further description of these and other risks and uncertainties that may cause actual results to differ materially from those expressed in the forward-looking statements as well as risks related to our business, please see our periodic reports filed with the SEC.

有關可能導致實際結果與前瞻性陳述中表達的結果大不相同的這些和其他風險和不確定性的進一步描述，以及與我們業務相關的風險，請參閱我們向美國證券交易委員會提交的定期報告。

On today's call, we have Dr. Adam Craig, our President, Chief Executive Officer and Interim Chief Medical Officer; David Kirske, our Chief Financial Officer; and Jim Fong, our Chief Commercial Officer. After formal remarks, we will open the call for your questions.

在今天的電話會議上，我們有我們的總裁、首席執行官兼臨時首席醫療官 Adam Craig 博士；我們的首席財務官 David Kirske；和我們的首席商務官 Jim Fong。正式發言後，我們將開始徵集您的問題。

I would now like to turn the call over to Adam.

我現在想把電話轉給亞當。

Thank you, Remy. Good morning. We appreciate everyone joining us earlier than planned so that we can provide a timely update following the recent release of portfolio revenue by our royalty financing partner.

謝謝你，雷米。早上好。我們感謝所有比計劃提前加入我們的人，這樣我們就可以在我們的特許權融資合作夥伴最近發布投資組合收入後及時提供更新。

I want to start the call today by reflecting on the achievements we have made over the past year as we've transformed from a research and development organization to a fully integrated biopharma with a strong commercial presence.

在今天的電話會議開始之際，我想回顧一下我們在過去一年中所取得的成就，因為我們已經從一家研發機構轉變為一家具有強大商業影響力的完全整合的生物製藥公司。

In the first quarter of last year, we received accelerated approval from the FDA for VONJO, pacritinib for the treatment of adults with myelofibrosis with a platelet count below 50 x 10 to the 9 per liter. This approval launched our commercialization efforts and we are extremely pleased with our progress to date.

去年第一季度，我們獲得了 FDA 對 VONJO 的加速批准，pacritinib 用於治療血小板計數低於每升 50 x 10 至 9 的成人骨髓纖維化。這一批准啟動了我們的商業化努力，我們對迄今為止取得的進展感到非常滿意。

We generated $54 million in net sales in just the first 9 months of commercialization, exceeding our internal year-end revenue goal and in the process, treated over 1,000 patients with VONJO in 2022, a meaningful achievement for a rare disease such as MF.

僅在商業化的前 9 個月，我們就產生了 5400 萬美元的淨銷售額，超過了我們的內部年終收入目標，在此過程中，2022 年治療了 1,000 多名 VONJO 患者，這對於 MF 等罕見疾病來說是一項有意義的成就。

Jim Fong will elaborate on our commercial success in just a moment. But I'd like to spend a little time talking about the new data presented in December at the ASH Annual Meeting in New Orleans by Dr. Stephen Oh of Washington University.

Jim Fong 稍後將詳細介紹我們的商業成功。但我想花一點時間談談華盛頓大學的 Stephen Oh 博士在 12 月於新奧爾良舉行的 ASH 年會上提出的新數據。

In his oral presentation, it was shown that pacritinib is a potent activin A receptor type 1 or ACVR1 inhibitor that has potential to provide the significant anemia benefit in patients with cytopenic myelofibrosis. This ACVR1 mediates hepcidin production and its inhibition is thought to lead to improvements in transfusion independence and anemia in myelofibrosis patients.

在他的口頭報告中，pacritinib 是一種有效的 1 型激活素 A 受體或 ACVR1 抑製劑，有可能為細胞減少性骨髓纖維化患者提供顯著的貧血益處。這種 ACVR1 介導鐵調素的產生，其抑製作用被認為可以改善骨髓纖維化患者的輸血依賴性和貧血。

Anemia is common in MF, both the diagnosis and during therapy, and it's a poor prognostic factor. Almost all MF patients will develop anemia over the course of their disease due to disease progression and/or drug toxicity from other approved JAK inhibitors.

貧血在 MF 中很常見，無論是在診斷中還是在治療期間，它都是一個不良的預後因素。由於疾病進展和/或來自其他批准的 JAK 抑製劑的藥物毒性，幾乎所有 MF 患者都會在其病程中出現貧血。

Dr. Oh's Presentation highlighted several key findings related to pacritinib. In vitro data demonstrated that pacritinib inhibits ACVR1 at 4x greater potency compared to momelotinib with 24-hour inhibition of the target. It was shown in vitro to reduce hepcidin levels. The clinical data demonstrated that treatment with VONJO at the approved dose of 200 milligrams twice daily, led to improvements in transfusion independence and anemia when compared to available therapy, which included ruxolitinib in invaluable patients treated on the Phase III PERSIST-2 study.

Oh 博士的演講強調了與 pacritinib 相關的幾個關鍵發現。體外數據表明，與 momelotinib 相比，pacritinib 抑制 ACVR1 的效力高 4 倍，可抑制靶標 24 小時。它在體外顯示可降低鐵調素水平。臨床數據表明，與現有療法相比，以 200 毫克批准劑量每天兩次使用 VONJO 治療可改善輸血獨立性和貧血，其中包括 ruxolitinib 在 III 期 PERSIST-2研究中治療的寶貴患者。

Over the last year, we have worked hard to establish the VONJO franchise in the commercial space. To this end, we have filed for a patent term extension for our main composition of matter patent that if granted, would extend this term by 5 years with an expected exploration date of 2034. And last month, VONJO was granted 7 years of orphan-drug exclusivity by the FDA for our label indication that began in February 2022.

在過去的一年裡，我們努力在商業領域建立 VONJO 特許經營權。為此，我們已經為我們的主要物質組成專利申請了專利期限延長，如果獲得批准，該期限將延長 5 年，預計勘探日期為 2034 年。上個月，VONJO 獲得了 7 年的孤兒- FDA 從 2022 年 2 月開始對我們的標籤適應症授予藥物獨占權。

As we enter our second year of commercialization for VONJO, we are focused on generating quarter-over-quarter product sales, while providing the best care to patients with cytopenic myelofibrosis.

當我們進入 VONJO 商業化的第二年時，我們專注於產生季度環比產品銷售，同時為細胞減少性骨髓纖維化患者提供最好的護理。

With that, I'll now hand the call over to Jim.

有了這個，我現在將電話交給吉姆。

Thank you, Adam, and good morning, everyone. As I look back on 2022, I'm extremely proud of my commercial team and our medical affairs colleagues who've led a successful launch for VONJO, a novel JAK2 ACVR1 IRAK1 inhibitor.

謝謝亞當，大家早上好。當我回顧 2022 年時，我為我的商業團隊和我們的醫療事務同事感到非常自豪，他們領導了 VONJO 的成功發布，VONJO 是一種新型 JAK2 ACVR1 IRAK1 抑製劑。

We achieved many accomplishments over the first 9 months of launch, highlighted by the team exceeding our internal revenue goals and reaching the milestone of treating over 1,000 myelofibrosis patients.

我們在發布的前 9 個月取得了許多成就，突出表現在團隊超越了我們的內部收入目標，並達到了治療 1,000 多名骨髓纖維化患者的里程碑。

Looking ahead, we are very encouraged by the continued double-digit sales growth of our team is producing.

展望未來，我們的團隊正在產生持續的兩位數銷售額增長，這讓我們感到非常鼓舞。

Today, I will provide a bit more context on these numbers as well as provide a snapshot of what we are seeing so far in 2023. We completed a strong sales performance with a 16% quarterly sales increase in Q4 and continued to show strong launch momentum in the quarter across several key metrics, including consistent growth in new patient starts, substantial increase in the number of active patients on VONJO, significant growth in new MF prescribers and accounts, a strong and expanding refill order rate and increased awareness and interest in VONJO's hematologic safety profile for anemia and thrombocytopenia.

今天，我將提供更多關於這些數字的背景信息，並簡要介紹我們在 2023 年到目前為止所看到的情況。我們完成了強勁的銷售業績，第四季度的季度銷售額增長了 16%，並繼續表現出強勁的推出勢頭在本季度的幾個關鍵指標中，包括新患者開始的持續增長、VONJO 活躍患者數量的大幅增加、新 MF 處方者和賬戶的顯著增長、強勁且不斷擴大的補充訂單率以及對 VONJO 的認識和興趣的提高貧血和血小板減少症的血液學安全性概況。

To maintain this trend, we continue to focus on educating our existing and new prescribers on the benefits of VONJO. Throughout 2022, we have conducted approximately 200 peer-to-peer education programs reaching more than 2,300 health care professionals. Due to these efforts, the physicians are becoming increasingly aware of VONJO as an important treatment option for MF.

為了保持這一趨勢，我們繼續專注於教育我們現有的和新的處方者了解 VONJO 的好處。整個 2022 年，我們開展了大約 200 項同行教育計劃，惠及 2,300 多名醫療保健專業人員。由於這些努力，醫生越來越意識到 VONJO 作為 MF 的重要治療選擇。

The scientific and clinical rationale for VONJO is resonating with providers, and they are recognizing cytopenic MF as a different disease that requires a different therapy such as VONJO. As a result, the number of VONJO new prescribers and accounts continues to grow, and a high proportion have become repeat users.

VONJO 的科學和臨床原理引起了提供者的共鳴，他們認識到細胞減少性 MF 是一種不同的疾病，需要不同的治療方法，例如 VONJO。因此，VONJO 新開處方者和新賬戶的數量持續增長，並且很大一部分成為了回頭客。

Although my commercial team can only promote use consistent with the FDA-approved label, we are seeing utilization -- the utilization of VONJO evolve.

儘管我的商業團隊只能促進與 FDA 批准的標籤一致的使用，但我們正在看到利用—— VONJO 的利用正在發展。

In Q4, market research indicates approximately 50% of current VONJO use is in patients with platelet counts at or above 50,000 per microliter compared to only 33% in Q3 with the majority of use coming from second-line patients. This change is likely related to the increasing awareness of the NCCN recommendations for VONJO.

在第四季度，市場研究表明，目前 VONJO 的使用中約有 50% 用於血小板計數等於或高於每微升 50,000 的患者，而第三季度這一比例僅為 33%，其中大部分使用來自二線患者。這一變化可能與 NCCN 對 VONJO 推薦的認識不斷提高有關。

In addition, we are seeing growth of VONJO as a first-line treatment as well as an overall increase in duration of therapy.

此外，我們看到 VONJO 作為一線治療的增長以及治療持續時間的總體增加。

As previously mentioned, our physician user base continues to expand with broader adoption and penetration. The success of our market penetration is evidenced by the fact that the number of community physicians prescribing VONJO has now exceeded physicians in the academic setting. As we all recognize, adoption of new treatments is typically much slower in the community versus the academic centers.

如前所述，隨著更廣泛的採用和滲透，我們的醫生用戶群繼續擴大。開 VONJO 處方的社區醫生的數量現在已經超過了學術界的醫生，這證明了我們市場滲透的成功。眾所周知，與學術中心相比，社區採用新療法的速度通常要慢得多。

With the growing number of prescribers coming from the community, I believe this will translate into increased sales potential for VONJO over time. Our sales momentum has carried nicely into 2023. We have seen consistent sales demand to start the year, and I look forward to updating you in May on our first quarter results.

隨著來自社區的開處方者越來越多，我相信隨著時間的推移，這將轉化為 VONJO 的銷售潛力增加。我們的銷售勢頭一直很好地延續到 2023 年。年初我們看到了穩定的銷售需求，我期待在 5 月份向您通報我們第一季度的最新業績。

In summary, physician adoption is growing due to the increased recognition of VONJO's differentiated mechanism of action and hematologic safety profile for cytopenic MF patients. Notably, we also continue to garner increased acceptance from the entire MPN community as a whole with VONJO as top of mind as evidenced by our continued strong brand awareness among our audience.

總之，由於對 VONJO 對細胞減少性 MF 患者的差異化作用機制和血液學安全性的認識增加，醫生的採用正在增加。值得注意的是，我們還繼續從整個 MPN 社區獲得越來越多的接受度，而 VONJO 是我們的首要考慮因素，我們在受眾中持續強大的品牌知名度就證明了這一點。

We expect our highly effective commercial teams to continue their progress in 2023, leveraging our robust promotional and educational efforts and maintaining a significant presence at major medical meetings.

我們預計我們高效的商業團隊將在 2023 年繼續取得進展，利用我們強大的宣傳和教育工作，並在主要醫學會議上保持重要地位。

I will now turn the call over to David to discuss our financial results.

我現在將電話轉給大衛討論我們的財務業績。

Thanks, Jim. Our financial results for the fourth quarter and year-end 2022 were published this morning in our press release and are available on our website. Therefore, I will summarize our current financial status.

謝謝，吉姆。我們今天上午在新聞稿中發布了我們 2022 年第四季度和年底的財務業績，並可在我們的網站上查閱。因此，我將總結我們目前的財務狀況。

As Adam mentioned, we continue to increase our product sales quarter-over-quarter. For 2022, I'm pleased to report that we generated $21.1 million in net revenue in the fourth quarter and $53.9 million for the 9 months of commercialization, exceeding our revenue goals.

正如 Adam 提到的，我們的產品銷量環比繼續增長。對於 2022 年，我很高興地報告，我們在第四季度產生了 2110 萬美元的淨收入，在商業化的 9 個月中產生了 5390 萬美元，超出了我們的收入目標。

All revenue was entirely attributable to the sales of VONJO. As expected, Towards the end of the year, we observed some holiday seasonality with our product sales, which is common for most oncology drugs. VONJO product sales also provided meaningful reductions in our overall operating loss in the fourth quarter and full year while we continue investment in SG&A related to marketing and commercialization of the product.

所有收入完全歸因於 VONJO 的銷售。正如預期的那樣，到年底，我們的產品銷售出現了一些節假日季節性，這對於大多數腫瘤藥物來說很常見。 VONJO 產品銷售還顯著減少了我們第四季度和全年的整體運營虧損，同時我們繼續投資於與產品營銷和商業化相關的 SG&A。

Our cash, cash equivalents and short-term investments as of December 31, 2022, totaled $79.9 million. Subsequent to the end of the quarter, we received $6.5 million in additional funding from our royalty agreement with DRI Healthcare Trust in January of 2023. Based on our current revenue projections and expenses, our objective is to be cash flow positive by the end of 2023. However, any meaningful changes to our clinical development plans or other transactions would impact this forecast.

截至 2022 年 12 月 31 日，我們的現金、現金等價物和短期投資總計 7990 萬美元。在本季度末之後，我們於 2023 年 1 月從與 DRI Healthcare Trust 的特許權使用費協議中獲得了 650 萬美元的額外資金。根據我們目前的收入預測和支出，我們的目標是到 2023 年底實現正現金流. 然而，我們的臨床開發計劃或其他交易的任何有意義的變化都會影響這一預測。

We have an active investment conference schedule over the next several months. We will be presenting on Wednesday, March 8, at the 43rd Annual TD Cowen Healthcare Conference, and we will also be participating in the Needham Healthcare Virtual Conference and Stifel Oncology Days in April.

在接下來的幾個月裡，我們有一個積極的投資會議時間表。我們將於 3 月 8 日星期三在第 43 屆年度 TD Cowen 醫療保健會議上發表演講，我們還將參加 Needham Healthcare 虛擬會議和 4 月的 Stifel 腫瘤學日。

We look forward to these interactions and keeping you updated on our progress.

我們期待這些互動，並讓您了解我們的最新進展。

I will now turn it back to Adam for closing remarks.

我現在將把它轉回給亞當作結束語。

Thank you, David. We are delighted to share our accomplishments on the commercialization of VONJO with you today. CTI is now established as a market leader in the treatment of cytopenic MF as we offer simple, safe and effective therapy for patients with an important medical condition.

謝謝你，大衛。我們很高興今天與您分享我們在 VONJO 商業化方面的成就。 CTI 現已成為治療細胞減少性 MF 的市場領導者，因為我們為患有重要疾病的患者提供簡單、安全和有效的治療。

For 2023, we are focused on expanding the use of VONJO with the aim of driving quarter-over-quarter revenue growth. We also continue to explore ways to increase the long-term potential for VONJO through additional indications.

到 2023 年，我們的重點是擴大 VONJO 的使用，以推動收入環比增長。我們還繼續探索通過其他適應症增加 VONJO 長期潛力的方法。

This concludes our formal remarks. I'd now like to ask Shannon to open the line for questions.

我們的正式發言到此結束。我現在想請 Shannon 打開問題熱線。

(Operator Instructions) Our first question comes from the line of Ken Shields with SVB Securities.

（操作員說明）我們的第一個問題來自 SVB 證券公司的 Ken Shields。

Congrats on the great quarter. So you mentioned seasonality impacts for 4Q. I'm wondering if you could provide some additional color on this. And then additionally, you've mentioned the 50 to 50 on label versus off-label population. Wondering if you have any color on how many of these patients were potentially -- or the off-label patients were potentially anemic myelofibrosis patients who have had higher platelet counts?

祝賀這個偉大的季度。所以你提到了第四季度的季節性影響。我想知道你是否可以為此提供一些額外的顏色。此外，您還提到了 50 到 50 的標籤人群與標籤外人群。想知道您是否知道這些患者中有多少可能是 - 或者標籤外患者可能是血小板計數較高的貧血性骨髓纖維化患者？

Thank you, Ken. I'll answer both those questions. First of all, we don't have that level of detail on the -- on whether the patients who are treated off-label have anemia. That's not something we have.

謝謝你，肯。我會回答這兩個問題。首先，我們沒有那麼詳細的信息——關於接受標籤外治療的患者是否患有貧血。那不是我們擁有的東西。

With regards to the seasonality, the seasonality is really very much what we expected. The last quarter is a busy quarter for holidays. That's also the American Society of Hematology meeting. So there's a lot going on that takes doctors out of the office, and we experience seasonality in the same way as many, many companies do who are in the oncology space.

關於季節性，季節性確實非常符合我們的預期。最後一個季度是假期繁忙的季度。那也是美國血液學會會議。所以有很多事情讓醫生離開辦公室，我們經歷季節性的方式與許多腫瘤領域的公司一樣。

Okay. And then just one more, if I can. So you guys obviously got the milestone from DRI. Do you have any color on what that threshold was? Was that $50 million and -- or additional milestones expected this year. Any color there? And does that factor into the cash flow positivity guidance by end of 2023?

好的。然後再來一個，如果可以的話。所以你們顯然從 DRI 獲得了里程碑。你對那個門檻有什麼看法嗎？是 5000 萬美元還是今年預期的其他里程碑？那裡有什麼顏色？到 2023 年底，這是否會影響現金流積極性指導？

Thanks, Ken. This is David here. We don't disclose the details of that milestone payment, it is incorporated into our guidance with respect to our cash forecast.

謝謝，肯。我是大衛。我們不透露該里程碑付款的細節，它已納入我們關於現金預測的指導中。

Our next question comes from the line of Ben Burnett with Stifel.

我們的下一個問題來自 Ben Burnett 與 Stifel 的對話。

I guess I want to ask just 2 quick questions. First, I guess where do you see the most ground to be gained kind of over the near term in terms of the VONJO launch? And is it about getting deeper into the community? Are there other aspects of the launch that's a bigger focus?

我想我只想問兩個簡單的問題。首先，我猜你認為在 VONJO 發布方面，短期內最有可能獲得的進展是什麼？是為了更深入地融入社區嗎？發布的其他方面是否更受關注？

Jim, please.

吉姆，拜託。

Yes. Ben, this is Jim. Yes. So essentially, yes, we see the growth really coming from the community and continuing to expand there. So no doubt that that's the observation we're looking for. And in addition, obviously, we're looking to get -- continue to get better prognosis patients in the queue for us as well.

是的。本，這是吉姆。是的。所以從本質上講，是的，我們看到增長確實來自社區並繼續在那裡擴展。所以毫無疑問，這就是我們正在尋找的觀察結果。此外，顯然，我們正在尋求 - 繼續為我們排隊等待更好的預後患者。

Okay. Excellent. And I guess on that last point, so what are you seeing in terms of duration of treatment? Do you expect this to change over time? So like I guess in other words, does the initial bowls of patients taking VONJO is that representative of sort of kind of what you expect the average patient, the duration of treatment from that average patient to be?

好的。出色的。我想關於最後一點，那麼您在治療持續時間方面看到了什麼？你希望這會隨著時間的推移而改變嗎？所以就像我猜的那樣，換句話說，最初服用 VONJO 的患者是否代表了你對普通患者的期望，即普通患者的治療持續時間？

Yes, Ben, obviously, we've only had 9 months into our launch. So it's tough to really prognosticate on the durations. However, to your point, like any new drug launch, the initial patients are going to be the -- probably a poor prognosis patients. And so the durations typically are parallel to that. However, as we mentioned before, the evolution of the use of VONJO is towards better prognosis patients, and we're seeing the durations parallel to that.

是的，本，顯然，我們的發布只有 9 個月的時間。所以很難真正預測持續時間。然而，就你的觀點而言，就像任何新藥上市一樣，最初的患者將是 - 可能是預後不良的患者。因此，持續時間通常與此平行。然而，正如我們之前提到的，VONJO 使用的演變是為了更好的預後患者，我們看到的持續時間與此平行。

Our next question comes from the line of Gil Blum with Needham.

我們的下一個問題來自 Gil Blum 與 Needham 的對話。

Congratulations on the progress. Maybe you could remind us how many patients are cytopenic on diagnosis?

祝賀你的進步。也許您可以提醒我們有多少患者在診斷時出現血細胞減少症？

Jim?

吉姆？

Yes. So essentially, you said at diagnosis, right, Gil?

是的。所以基本上，你在診斷時說，對吧，吉爾？

Yes.

是的。

Yes. So at diagnosis, approximately 1/3 of patients will have platelet counts less than 100,000, and at diagnosis of probably about 40% of patients will have anemia without thrombocytopenia.

是的。因此，在診斷時，大約 1/3 的患者血小板計數低於 100,000，而在診斷時，大約 40% 的患者可能有貧血但沒有血小板減少。

Got you. And over time, that generally increases because of disease progression?

明白了隨著時間的推移，這通常會因為疾病進展而增加？

Absolutely. Absolutely. Yes. In fact, you'll see over time, when you look at (inaudible) data, about 2/3 of patients will have platelet counts less than 100,000.

絕對地。絕對地。是的。事實上，隨著時間的推移，當您查看（聽不清）數據時，您會發現大約 2/3 的患者血小板計數低於 100,000。

My second question is regarding guidance, and I know it wasn't provided in this quarter, but I'm curious as to what gates a decision about providing guidance?

我的第二個問題是關於指導，我知道本季度沒有提供指導，但我很好奇提供指導的決定是由什麼決定的？

Thank you, Gil. We're not going to provide guidance at this time. We -- when we're ready to provide guidance, we will let you know. One of the factors we're looking at is gross to net at the moment. And as we've spoken about publicly, the gross to net is still somewhat variable. Until it stabilizes, we'll refrain from providing guidance.

謝謝你，吉爾。我們目前不打算提供指導。我們——當我們準備好提供指導時，我們會通知您。我們正在考慮的因素之一是目前的淨值。正如我們公開談論的那樣，毛淨值仍然有些變化。在它穩定下來之前，我們不會提供指導。

Our next question comes from the line of Reni Benjamin with JMP Securities.

我們的下一個問題來自 Reni Benjamin 與 JMP Securities 的對話。

Congratulations on the quarter. Adam, I guess I'd like to stick with the ACVR1 data that was reported at ASH. I'm kind of curious as to what are the next steps, right, for the company? Is there any way to -- like what would be required to get this into the label? And how do you see this data ultimately being utilized from a commercialization effort?

祝賀這個季度。 Adam，我想我想堅持使用 ASH 報告的 ACVR1 數據。我很好奇公司接下來的步驟是什麼？有什麼辦法——比如將其放入標籤需要什麼？您如何看待最終從商業化工作中利用這些數據？

Yes. Well, first of all, the anemia data is a post-hoc analysis. So it's unlikely that the clinical data would get into the label. However, the mechanistic data, the ACVR1, the pharmacodynamic and pharmacokinetic data around that, it may be possible to get in that label, and that's something the team is working on.

是的。好吧，首先，貧血數據是事後分析。所以臨床數據不太可能進入標籤。然而，機械數據、ACVR1、圍繞它的藥效學和藥代動力學數據，有可能進入該標籤，這是團隊正在努力的事情。

Moving forward, it's an important component of our scientific education of physicians through our medical science liaisons. It's not a data set that we -- Jim's team can actively promote but we can discuss it scientifically. And in 2023, we are expanding the number of medical science liaisons we have in the field so that we can continue the conversation around the ACVR1 data.

展望未來，它是我們通過醫學科學聯絡員對醫生進行科學教育的重要組成部分。這不是我們 - Jim 的團隊可以積極推廣的數據集，但我們可以科學地討論它。到 2023 年，我們將擴大我們在該領域的醫學科學聯絡人的數量，以便我們可以繼續圍繞 ACVR1 數據進行對話。

After ASH, we had a lot of inbound interest and a lot of requests to discuss the data with us, and that's why we've increased the number of people who are able to do that. That is the medical science liaisons.

在 ASH 之後，我們有很多入站興趣和很多要求與我們討論數據，這就是為什麼我們增加了能夠這樣做的人數。那就是醫學聯絡員。

Can you just provide some additional color on how much would you tell us the increase will be for MSLs? And just kind of reading in between kind of what you said, I just want to confirm, you won't be starting some sort of a larger study, let's say, to evaluate the anemia benefit going forward, it would really just be based on the data that's been generated to date?

您能否提供一些額外的顏色，說明您會告訴我們 MSL 的增加量是多少？只是在你所說的之間閱讀，我只是想確認，你不會開始某種更大規模的研究，比方說，評估未來貧血的益處，它真的只是基於迄今為止生成的數據？

Well, the anemia benefit will be assessed in the PACIFICA trial. As with respect to how much, I don't have -- I don't have a number to give you there. Post-ASH, we have had a significant amount of interest in the anemia data. It was a very successful ASH for us, and we've come out of that into this quarter with a lot of questions and activity and discussions around the data. So it was very productive for us scientifically.

那麼，貧血益處將在 PACIFICA 試驗中進行評估。至於多少，我沒有——我沒有數字可以給你。在 ASH 之後，我們對貧血數據產生了極大的興趣。這對我們來說是一個非常成功的 ASH，我們已經從這個季度出來，圍繞數據提出了很多問題、活動和討論。所以從科學上來說，這對我們來說非常有成效。

Got it. And then just my final question. Can you just talk a little bit about the -- how you're thinking about price increases and how we should be thinking about the first quarter only because other companies that we follow, tend to have increased gross to net, right, in the first quarter as they try to help patients with the resetting of the co-pays and the like. Can you give us maybe a sense as to how we should be thinking about the revenues for this quarter?

知道了。然後是我的最後一個問題。你能不能談談——你是如何考慮價格上漲的，以及我們應該如何考慮第一季度，因為我們關注的其他公司往往在第一季度增加了毛對淨額，對吧？季度，因為他們試圖幫助患者重置共同支付等。您能否告訴我們應該如何考慮本季度的收入？

We did -- we had a price increase at the end of the year. We haven't -- we're not going to comment on price increases for the rest of the year because there are number of factors that we have to consider. And as the year progresses, we may or may not do an additional price increase. It's not a decision we've made at the moment.

我們做到了——我們在年底提價了。我們還沒有——我們不會對今年剩餘時間的價格上漲發表評論，因為我們必須考慮許多因素。隨著時間的推移，我們可能會或可能不會額外提價。這不是我們目前做出的決定。

Sorry, I just wanted to know about the first quarter...

抱歉，我只想知道第一季...

I'm sorry, Reni, I think you asked about the first quarter. We're encouraged. We've had a good start to the year, and we're progressing very well, but we'll we will be reporting our first quarter results in about 8 weeks' time.

對不起，Reni，我想你問的是第一季度。我們受到鼓舞。我們今年開局良好，進展順利，但我們將在大約 8 週後報告第一季度業績。

Reni, I'm sorry, did you ask about the first quarter in terms of price increase or -- I'm sorry.

雷尼，對不起，你問過第一季度的價格上漲嗎？或者 - 對不起。

Yes. I was more just trying to get a sense, like we typically see a decrease in revenues in the first quarter because there's an increase in gross to net with other drugs like ruxolitinib as the companies try to help cover the doughnut hole patients, there's a delay to fill scripts. So there's just some interesting dynamics that happened in the first quarter just because of the reset that takes place. And so from an insurance perspective. And I just wanted to get your guys' thoughts on how that might impact VONJO?

是的。我只是想了解一下，就像我們通常會看到第一季度的收入下降一樣，因為隨著公司試圖幫助覆蓋甜甜圈洞患者，其他藥物（如 ruxolitinib）的毛淨收入有所增加，有延遲填充腳本。因此，由於發生了重置，第一季度發生了一些有趣的動態。所以從保險的角度來看。我只是想听聽你們對這可能對 VONJO 有何影響的想法？

Right. Ren, we're happy to talk about that during the first quarter report call. We can't talk about that today.

正確的。任，我們很高興在第一季度報告電話會議上討論這個問題。我們今天不能談這個。

Our next question comes from the line of Thomas Flaten with Lake Street Capital Markets.

我們的下一個問題來自 Thomas Flaten 與 Lake Street Capital Markets 的合作。

Jim, you mentioned that the number of community prescribers have exceeded the number of academic prescribers. I was wondering if you could characterize patient starts or total patients along those lines as well. Has that -- is that the same dynamic? Or are you still waiting for more patients in the community side?

吉姆，你提到社區處方者的數量已經超過學術處方者的數量。我想知道您是否也可以按照這些路線來描述患者開始或總患者的特徵。有沒有 - 是相同的動態嗎？還是還在社區等著更多的患者？

Yes. So that is the dynamic, Correct. So we are seeing the new patient starts growing and that growth is exceeding in the community of those in the academic centers. So we're really pleased about that because like we said, it's a really good marker for penetration adoption and uptick of a new product launch.

是的。所以這是動態的，正確的。因此，我們看到新患者開始增長，並且學術中心社區的增長速度超過了社區。所以我們對此感到非常高興，因為就像我們說的那樣，它是滲透採用和新產品發布上升的一個非常好的標誌。

Great. And then any comment on lower dose VONJO prescribing in patients that might be challenged with the adverse events?

偉大的。然後對可能受到不良事件挑戰的患者開低劑量 VONJO 有何評論？

Yes. We are seeing some of that. No doubt, if there are patients who are frail, that is up to the discretion of the physician. But by and large, the vast majority of VONJO prescriptions are at the proper dose of 200 b.i.d.

是的。我們正在看到其中的一些。毫無疑問，如果有身體虛弱的病人，這取決於醫生的判斷。但總的來說，絕大多數 VONJO 處方的適當劑量為 200 b.i.d.

Got it. And then one quick one for David. There was a pretty significant uptick sequentially in R&D and SG&A. Was that end of the year, David? Or is that a new baseline we should use going forward?

知道了。然後給大衛一個快速的。研發和 SG&A 依次出現了相當顯著的上升。那是年底嗎，戴維？或者這是我們應該使用的新基線？

I mean that is reflective of our concurrent investment in SG&A. So it's -- as revenue continues to drive forward that benefit, we are using to invest in commercialization as well as marketing.

我的意思是這反映了我們對 SG&A 的同時投資。所以它 - 隨著收入繼續推動這一利益，我們正在使用投資商業化和營銷。

Our next question comes from the line of Robert Hazlett with BTIG.

我們的下一個問題來自 BTIG 的 Robert Hazlett。

Congrats on the progress, and I have a couple for me. Just -- I know you are not forecasting any price increases, Adam. But what was the price increase around year-end? Sorry, what did you say how much that was?

祝賀進步，我有一對。只是——我知道你沒有預測任何價格上漲，亞當。但年底前後的價格漲幅是多少？抱歉，你剛才說那是多少？

Bert, this is Jim Fong. Yes, it was approximately at 9.9% on January 3.

伯特，這是吉姆方。是的，1 月 3 日大約為 9.9%。

Okay. Okay. Great. And then just with regard to the anemia data, is there kind of a broader strategy with regard to NCCN guidelines or additional publication that we should be thinking about as we think about kind of the evolution of VONJO?

好的。好的。偉大的。然後就貧血數據而言，在考慮 VONJO 的演變時，我們是否應該考慮更廣泛的 NCCN 指南或其他出版物策略？

Yes, certainly. We've already submitted some anemia data to NCCN and it's actually under review, but the final data set from Stephen Oh's work will be submitted once the latest version are published. We're waiting for publication or based on data we submitted last year, once it's published, we can then submit the additional data. And we do expect there to be a modification of the guidelines sometime around midyear with the potential approval of momelotinib.

是的，當然了。我們已經向 NCCN 提交了一些貧血數據，實際上正在審查中，但是一旦最新版本發布，Stephen Oh 的工作的最終數據集將被提交。我們正在等待發布，或者根據我們去年提交的數據，一旦發布，我們就可以提交額外的數據。我們確實希望隨著 momelotinib 的可能批准，在年中左右的某個時候對指南進行修改。

Okay. Terrific. Is there a digital IP that's available with regard to anemia for VONJO?

好的。了不起。是否有關於 VONJO 貧血的可用數字 IP？

I doubt it, the composition of matter IP is pretty robust and with the 5-year patent term extension that will take us 2034. I doubt the anemia data will be -- will provide anything. It would have to -- for additional patent protection or patent filing, we'd have to have something that was novel, and it's probably wouldn't be considered as such.

我對此表示懷疑，物質 IP 的組成非常穩健，並且專利期限延長 5 年，這將使我們到 2034 年。我懷疑貧血數據是否會提供任何信息。它必須——為了額外的專利保護或專利申請，我們必須有一些新穎的東西，而且它可能不會被認為是這樣。

Okay. Terrific. Just one more for me. There's a material jump in prescriptions and in various metrics. I know that is a hugely blunt instrument, especially with regard to products like VONJO but we saw a jump in the January data. Are there -- is there anything behind that? Just would like to maybe know a little bit more? Or is that the anemia data kicking in or other things at work there?

好的。了不起。再給我一個。處方和各種指標都有實質性的飛躍。我知道這是一個非常生硬的工具，尤其是對於像 VONJO 這樣的產品，但我們看到 1 月份的數據出現了跳躍。那背後有什麼嗎？只是想知道更多一點？還是貧血數據正在起作用或其他因素在那裡起作用？

Bert, what are you -- what data are you referring to? Do you have a different syndicated report?

伯特，你是什麼——你指的是什麼數據？你有不同的聯合報告嗎？

The Symphony data, there is various prescription data, manufacturing benchmark sales, different data metrics like that show a pretty material jump. And I just want to make sure I understand some of the elements behind that.

Symphony 數據，有各種處方數據，製造基准銷售，不同的數據指標，顯示出相當大的材料跳躍。我只是想確保我了解其背後的一些要素。

Yes, Bert, as you've seen, obviously, the momentum that was created in Q4 ahead of Q1 is really continuing. And I think the -- so we had already, again, seen robust momentum already started by the end of the year as the message started to really penetrate our target audience. But I think the oral presentation at ASH, no doubt, continue to fuel that momentum that we're seeing today in Q1.

是的，伯特，正如你所見，顯然，在第一季度之前的第四季度創造的勢頭確實在繼續。而且我認為 - 所以我們已經再次看到到今年年底已經開始強勁勢頭，因為信息開始真正滲透到我們的目標受眾中。但我認為 ASH 的口頭報告無疑會繼續推動我們今天在第一季度看到的勢頭。

This concludes the question-and-answer session. I'd now like to hand the conference back over to Adam Craig for closing remarks.

問答環節到此結束。我現在想將會議交還給 Adam Craig 作閉幕詞。

Thank you, Shannon, and thank you, everyone, for joining the call today. We appreciate your continued interest in CTI, and we look forward to keeping you updated on our progress. Shannon, that now concludes the call.

謝謝香農，也謝謝大家今天加入電話會議。我們感謝您對 CTI 的持續關注，我們期待著讓您了解我們的最新進展。香農，現在結束通話。

Thank you. This concludes today's conference call. Thank you for participating. You may now disconnect.

謝謝。今天的電話會議到此結束。感謝您的參與。您現在可以斷開連接。