使用警語:中文譯文來源為 AI 翻譯,僅供參考,實際內容請以英文原文為主
Operator
Operator
Ladies and gentlemen, welcome to the Arrowhead Pharmaceuticals conference call. (Operator Instructions)
各位女士、先生,歡迎參加 Arrowhead Pharmaceuticals 的電話會議。(接線員指示)
I will now hand the conference call over to Vince Anzalone, Senior Vice President of Investor Relations for Arrowhead. Please go ahead, Vince.
現在我將把電話會議交給 Arrowhead 投資人關係資深副總裁 Vince Anzalone。Vince,請開始。
Vincent Anzalone - Investor Relations
Vincent Anzalone - Investor Relations
Good afternoon, and thank you for joining us today to discuss Arrowhead's results for its fiscal 2026 second quarter ended March 31, 2026.
各位下午好,感謝各位今天加入我們,一同討論 Arrowhead 截至 2026 年 3 月 31 日止之 2026 會計年度第二季業績。
With us today from management are President and CEO, Dr. Chris Anzalone, who will provide an overview; Andy Davis, Senior Vice President and Head of the Global Cardiometabolic franchise, who will provide an update on commercialization activities; Dr. James Hamilton, Chief Medical Officer and Head of R&D, who will discuss our development programs; and Dan Apel, Chief Financial Officer, who will give a review of the financials. Following managementâs prepared remarks, we will open the call to questions.
今天與會的管理團隊包括:總裁兼執行長 Chris Anzalone 博士,將提供整體概覽;全球心臟代謝事業群資深副總裁暨負責人 Andy Davis,將更新商業化活動進展;研發主管兼首席醫療官 James Hamilton 博士,將說明我們的開發計畫;以及財務長 Dan Apel,將回顧財務表現。在管理團隊的準備發言之後,我們將開放提問。
Before we begin, I would like to remind you that comments made during todayâs call contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than statements of historical fact are forward-looking statements and are subject to numerous risks and uncertainties that could cause actual results to differ materially from those expressed in any forward-looking statements.
在開始之前,我想提醒各位,今天電話會議中的評論包含若干前瞻性陳述,該等陳述係依據《1933 年證券法》第 27A 條及《1934 年證券交易法》第 21E 條之意義所作。除歷史事實陳述外,所有陳述均屬前瞻性陳述,並受多項風險與不確定性影響,可能導致實際結果與任何前瞻性陳述中所表達者出現重大差異。
For further details concerning these risks and uncertainties, please refer to our SEC filings, including our most recent annual report on Form 10-K and our quarterly reports on Form 10-Q.
關於上述風險與不確定性的更多細節,請參閱我們向 SEC 提交的文件,包括最新的 Form 10-K 年度報告以及 Form 10-Q 季度報告。
I'd now like to turn the call over to Chris.
現在我想把電話交給 Chris。
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Thanks, Vince. Good afternoon, everyone, and thank you for joining us today.
謝謝你,Vince。各位下午好,感謝各位今天加入我們。
During the fiscal second quarter and the period since our last earnings call, we have continued to execute well against our commercial, R&D, and corporate goals. Arrowhead is now on the strongest footing of our history. We are commercial. We have clear line-of-sight to expand our commercial opportunities and footprint. Our pipeline is larger than ever. Our discovery capabilities are broader than ever. And our balance sheet is stronger than ever. This is an historic time for our company. We are uniquely positioned to deliver important medicines to the patients who need them and create substantial value for our shareholders.
在本會計年度第二季以及自上次財報電話會議以來的期間,我們持續在商業、研發與公司層面的目標上執行得相當到位。Arrowhead 目前處於公司史上最穩健的基礎。我們已實現商業化。我們對擴大商業機會與市場版圖具有清晰可見的路徑。我們的產品線(pipeline)比以往任何時候都更龐大。我們的探索研發能力比以往任何時候都更廣泛。而我們的資產負債表也比以往任何時候都更強健。這是公司歷史性的時刻。我們具備獨特優勢,能為有需要的病患提供重要藥物,並為股東創造可觀價值。
Letâs talk about some of our recent progress and begin with commercial. As you recall, the FDA-approved REDEMPLO in November 2025 as an adjunct to diet to reduce triglycerides in adults with FCS. FCS is a severe, rare disease, with an estimated 6,500 people in the US living with genetic or clinical FCS, characterized by TG levels that can be 10 to 100 times higher than normal. This leads to a substantially increased risk of developing acute, recurrent, and potentially fatal pancreatitis.
接下來談談我們近期的一些進展,先從商業化開始。如各位所記得,FDA 於 2025 年 11 月核准 REDEMPLO,作為飲食控制的輔助療法,用於降低成人 FCS 患者的三酸甘油脂。FCS 是一種嚴重且罕見的疾病,估計美國約有 6,500 人罹患遺傳性或臨床診斷之 FCS,其特徵為 TG 濃度可能高於正常值的 10 到 100 倍。這會大幅提高發生急性、反覆且可能致命之胰臟炎的風險。
As we reported last quarter, the US REDEMPLO launch was off to a strong start. That momentum has continued into the current quarter, and we are now seeing around 30 new prescriptions written each week. Greater than 400 prescriptions have been written since launch, and more than 10% of these have been for patients switching from our competitorâs APOC3 inhibitor.
如同我們在上季所報告,美國 REDEMPLO 上市初期表現強勁。這股動能延續到本季,目前我們每週約新增 30 張處方。自上市以來已開立超過 400 張處方,其中超過 10% 來自由我們競品的 APOC3 抑制劑轉換用藥的患者。
Of course, each prescription needs to be fully adjudicated with payers before becoming paid claims, but we offer a robust Quick Start program to support these FCS patients in the interim. The volume of physicians writing prescriptions and the number of patients receiving REDEMPLO continues to exceed our initial expectations.
當然,每張處方在成為已支付的理賠(paid claims)之前,都需要先由付款方完成完整的理賠審核(adjudication),但我們提供完善的 Quick Start 計畫,在此期間支援這些 FCS 患者。開立處方的醫師數量以及使用 REDEMPLO 的患者人數,持續超出我們最初的預期。
With respect to pricing, we updated REDEMPLOâs US wholesale acquisition cost, or WAC, to $45,000 per patient per year. This represents a premium to our competitorâs WAC pricing. We believe this is appropriate given that clinical data suggest we have a clearly and demonstrably superior product in terms of TG reduction, safety profile, and convenience.
在定價方面,我們將 REDEMPLO 在美國的批發取得成本(WAC)更新為每位患者每年 45,000 美元。此價格相較於競品的 WAC 定價屬於溢價。我們認為此溢價合理,因為臨床數據顯示,我們在 TG 降幅、安全性概況與使用便利性方面,具有明確且可證明的優勢。
As part of the One-REDEMPLO unified pricing model, this price is intended to remain consistent across FCS and SHTG, if that indication is approved. We continue to see this strategy as potentially simplifying payer contracting and eliminating pricing complexity that could complicate future formulary negotiations. The response from payers to this strategy has been positive and our interactions to date have been productive.
作為 One-REDEMPLO 統一定價模式的一部分,若未來 SHTG 適應症獲核准,該價格預計將在 FCS 與 SHTG 之間保持一致。我們仍認為此策略可望簡化與付款方的合約談判,並消除可能使未來處方集(formulary)談判複雜化的定價複雜度。付款方對此策略的回應正面,我們迄今的互動也相當具建設性。
Beyond the US, we secured positive regulatory action in four additional geographies for REDEMPLO in patients with genetically confirmed and clinically defined FCS. We received approvals from the Australian Therapeutic Goods Administration, the Chinese National Medical Products Administration, and Health Canada.
除美國之外,我們也在另外四個地區就 REDEMPLO 用於基因確診與臨床定義之 FCS 患者取得正面的監管進展。我們已獲澳洲治療用品管理局(TGA)、中國國家藥品監督管理局(NMPA)以及加拿大衛生部(Health Canada)核准。
In addition, the European Medicines Agencyâs Committee for Medicinal Products for Human Use adopted a positive opinion, recommending the approval of REDEMPLO. This is an impressive result achieved by our global regulatory team in a very short period, and further reflects the strength of our clinical data in FCS and the value that REDEMPLO offers to patients.
此外,歐洲藥品管理局(EMA)之人用藥品委員會(CHMP)已採納正面意見,建議核准 REDEMPLO。這是在極短時間內由我們全球法規團隊達成的亮眼成果,也進一步反映我們在 FCS 的臨床數據實力,以及 REDEMPLO 為患者帶來的價值。
REDEMPLO will be available later this year in Canada and we anticipate it will be marketed independently by Arrowhead. Pending a marketing authorization decision from the European Commission, we expect to launch REDEMPLO later this year in select EU countries and likely in the UK as well. In Greater China, REDEMPLO will be marketed by Sanofi.
REDEMPLO 將於今年稍晚在加拿大上市,我們預期將由 Arrowhead 獨立行銷。在等待歐盟執委會作出上市許可(marketing authorization)決定之際,我們預期今年稍晚將在部分歐盟國家、以及可能也包括英國,推出 REDEMPLO。在大中華區,REDEMPLO 將由賽諾菲(Sanofi)負責行銷。
In addition to our regulatory team, the rest of the R&D organization has performed extremely well and has made progress in the broader portfolio. Our drive to expand our platforms in order to increase the number and types of diseases we can address continues, even as we grow as a commercial entity.
除了法規團隊之外,研發組織的其他部門也表現非常出色,並在更廣泛的產品組合上取得進展。即使我們作為商業化公司持續成長,我們仍持續推動擴展平台,以增加我們可涵蓋的疾病數量與類型。
During the recent period, we have made rapid progress across the pipeline, including programs targeting genes expressed in liver, skeletal muscle, adipose, CNS, and lung, as well as the first dual-functional siRNA designed to silence the expression of two genes with a single molecule. We believe the depth and breadth of our clinical pipeline is unmatched, and we expect to continue to lead the field in innovation. Importantly, many of these programs will have clinical readouts this year, so investors and others may start to properly value the broader pipeline.
在近期期間,我們在整體產品線上取得快速進展,包括針對肝臟、骨骼肌、脂肪組織、中樞神經系統(CNS)與肺部表達之基因的計畫;以及首個雙功能 siRNA,設計用單一分子同時沉默兩個基因的表達。我們相信,我們臨床產品線的深度與廣度無可匹敵,並預期將持續在創新方面引領領域發展。重要的是,其中許多計畫將在今年讀出臨床結果,因此投資人及其他利害關係人可望開始更恰當地評價更廣泛的產品線。
As we look to near-term clinical data releases, we anticipate four important events. First, the Phase 3 SHASTA-3 and 4 studies of Plozasiran in SHTG patients should be ready for top-line data release in Q3. This is an important read-out that will drive our anticipated supplemental NDA, or sNDA, as we seek to expand the population of patients we can treat with Plozasiran. We expect to continue to see a favorable safety profile and substantial reduction in TGs, and we are cautiously optimistic that we could see an improvement in acute pancreatitis risk.
展望近期的臨床數據發布,我們預期將有四項重要事件。首先,Plozasiran 用於 SHTG 患者的第 3 期 SHASTA-3 與 SHASTA-4 研究,應可在第三季準備好發布主要結果(top-line data)。這是一項重要的讀出結果,將推動我們預期提交的補充新藥申請(sNDA),以擴大我們可用 Plozasiran 治療的患者族群。我們預期將持續看到良好的安全性概況與顯著的 TG 降幅,並對於可能改善急性胰臟炎風險抱持審慎樂觀。
Second, we expect to have early data from the ongoing Phase 1/2 study of ARO-DIMER-PA in patients with mixed hyperlipidemia in Q3. We believe this will be the worldâs first clinical data of a single RNAi molecule designed to simultaneously silence the expression of two proteins.
第二,我們預期在第三季取得正在進行之 ARO-DIMER-PA 於混合型高脂血症患者的第 1/2 期研究的早期數據。我們相信這將是全球首份臨床數據,來自一個單一 RNAi 分子,其設計可同時沉默兩種蛋白的表達。
If we see good reduction of PCSK9 and APOC3, and therefore reductions in LDL/cholesterol and TGs, we could have a very powerful and unique therapy for the roughly 20 million people in the US living with mixed hyperlipidemia. More broadly, the data could provide initial clinical proof of concept for our growing dimer platform and pipeline. Expect to see additional dual-functional dimers in the clinic in 2027.
若我們看到 PCSK9 與 APOC3 有良好降低,並因此帶來 LDL/膽固醇與三酸甘油脂(TGs)的下降,我們就可能為美國約 2,000 萬名混合型高脂血症患者提供一項非常強大且獨特的療法。更廣泛而言,這些數據可為我們持續擴展的二聚體平台與產品線提供初步的臨床概念驗證。預期在 2027 年會看到更多具雙重功能的二聚體進入臨床。
Third, we expect to have early data from the ongoing Phase 1/2 study of ARO-MAPT around the end of Q3 or early Q4. As you recall, this is our first candidate using our CNS platform designed to deliver RNAi molecules to the brain via simple subcutaneous administration.
第三,我們預期在第三季末或第四季初取得正在進行之 ARO-MAPT 第 1/2 期研究的早期數據。如各位所記得,這是我們第一個使用 CNS 平台的候選藥物,該平台旨在透過簡單的皮下注射將 RNAi 分子遞送至大腦。
ARO-MAPT targets the Tau protein, which is increasingly validated for the potential treatment of Alzheimerâs and other Tauopathies. We believe that positive early data could be substantially disruptive. It could represent a great leap forward in treating Tauopathies and, more broadly, open the door to using RNAi to treat a broad range of conditions from neurodegenerative disorders to obesity. If early ARO-MAPT data are encouraging, expect a substantial expansion of our CNS pipeline, beginning at the end of 2026.
ARO-MAPT 以 Tau 蛋白為標的,而 Tau 蛋白在阿茲海默症及其他 Tau 蛋白病(Tauopathies)的潛在治療上,正獲得越來越多的驗證。我們相信正向的早期數據可能帶來顯著的顛覆性影響。這可能代表治療 Tau 蛋白病的一大躍進,並且更廣泛地為使用 RNAi 治療從神經退化性疾病到肥胖等多種疾病打開大門。若 ARO-MAPT 的早期數據令人鼓舞,預期自 2026 年底開始,我們的 CNS 產品線將大幅擴展。
Fourth, we expect to provide clinical updates on ARO-INHBE and ARO-ALK7 throughout the second half of the year. Regarding ARO-INHBE, we plan to present additional data at various conferences and launch a P2 study.
第四,我們預期在今年下半年持續提供 ARO-INHBE 與 ARO-ALK7 的臨床更新。就 ARO-INHBE 而言,我們計畫在各類會議上發表更多數據,並啟動一項第 2 期(P2)研究。
For ARO-ALK7, we expect to provide additional data from the ongoing Phase 1/2 study. We see these as potentially important therapies for metabolic disorders and represent our first steps into obesity and MASH. We expect to have additional candidates in this space by the end of the year and into 2027.
就 ARO-ALK7 而言,我們預期提供正在進行之第 1/2 期研究的更多數據。我們認為這些可能是代謝性疾病的重要療法,並代表我們首次跨入肥胖與 MASH 領域。我們預期在今年底以及延伸至 2027 年,於此領域將有更多候選藥物。
Moving on to financial and portfolio management. Arrowhead took important steps to ensure that we are properly funded to advance our commercial and development portfolio, and we also entered into a license agreement for a program that achieved clinical proof-of-concept but is not one that we wish to take forward. This is key to Arrowheadâs strategy, since we are extraordinarily productive in discovery and early development but cannot commercialize everything independently.
接下來談財務與投資組合管理。Arrowhead 採取了重要措施,以確保我們具備充足資金來推進商業化與研發投資組合;同時,我們也就一項已達成臨床概念驗證、但並非我們希望繼續推進的計畫,簽訂了授權協議。這是 Arrowhead 策略的關鍵,因為我們在探索研究與早期開發方面極具生產力,但無法獨立將所有項目商業化。
Letâs talk about the steps we took.
讓我們談談我們採取了哪些步驟。
First, we dramatically strengthened our balance sheet, allowing us to push multiple programs toward commercialization and potentially through multiple independent and partner launches. During the quarter we completed the largest fund raising Arrowhead has ever conducted. We closed concurrent public offerings of $700 million of 0% coupon convertible senior notes and $230 million of common stock. Both offerings were several times oversubscribed, reflecting investor confidence in our portfolio and our ability to continue to build value.
第一,我們大幅強化資產負債表,使我們能推動多個項目走向商業化,並可能透過多個自有與合作夥伴的上市推進。本季我們完成了 Arrowhead 史上最大規模的募資。我們完成了同步公開發行:7 億美元 0% 息票可轉換優先票據,以及 2.3 億美元普通股。兩項發行皆獲得數倍超額認購,反映投資人對我們投資組合及我們持續創造價值能力的信心。
Second, and just this week, we announced an exclusive worldwide license agreement with Madrigal Pharmaceuticals for ARO-PNPLA3, Arrowheadâs clinical stage program designed to treat a genetically-defined population of MASH patients. Under the terms of the agreement, Madrigal will make a $25 million upfront payment to Arrowhead. Arrowhead is also eligible to receive development, regulatory, and sales milestone payments of up to $975 million. Arrowhead is further eligible to receive tiered royalties up to mid-teens.
第二,就在本週,我們宣布與 Madrigal Pharmaceuticals 就 ARO-PNPLA3 達成全球獨家授權協議;ARO-PNPLA3 為 Arrowhead 的臨床階段計畫,旨在治療一群以基因特徵界定的 MASH 患者。依協議條款,Madrigal 將向 Arrowhead 支付 2,500 萬美元的預付款。Arrowhead 亦有資格獲得最高達 9.75 億美元的開發、法規核准與銷售里程碑款。此外,Arrowhead 亦有資格獲得分級權利金,最高可達約十幾個百分點(mid-teens)。
Madrigalâs leadership in the MASH space makes it a natural and attractive partner to advance ARO-PNPLA3 into Phase 2 studies and toward potential commercialization. This transaction with Madrigal underscores Arrowheadâs disciplined business development strategy, demonstrating our ability to partner high-potential, clinically validated programs with leading organizations.
Madrigal 在 MASH 領域的領導地位,使其成為推進 ARO-PNPLA3 進入第 2 期研究並邁向潛在商業化的自然且具吸引力的合作夥伴。與 Madrigal 的這筆交易凸顯 Arrowhead 嚴謹的商務拓展策略,展現我們能將高潛力、已獲臨床驗證的計畫與領先機構進行合作。
With that overview, Iâd now like to turn the call over to Andy Davis. Andy?
有了上述概覽後,我現在想把電話會議交給 Andy Davis。Andy?
Andy Davis - Senior Vice President and Head of the Global Cardiometabolic Franchise
Andy Davis - Senior Vice President and Head of the Global Cardiometabolic Franchise
Thank you, Chris, and good afternoon, everyone. It has now been approximately 5.5 months since the FDA approval of REDEMPLO on November 18, 2025, and we continue to be very pleased with the trajectory of the launch. Today I would like to cover five areas: prescription and patient dynamics, payer coverage developments, pricing strategy, commercial infrastructure expansion, and our international and SHTG outlook.
謝謝你,Chris,各位下午好。自 FDA 於 2025 年 11 月 18 日核准 REDEMPLO 以來,至今約 5.5 個月,我們對上市進展的走勢仍感到非常滿意。今天我想涵蓋五個面向:處方與患者動態、支付方(payer)給付覆蓋的進展、定價策略、商業基礎建設擴張,以及我們的國際與 SHTG 展望。
Let's start with prescription and patient dynamics. REDEMPLO's launch continues to build strong and consistent momentum. Through the fiscal second quarter ending March 31, 2026, we have seen prescriptions accelerating week-over-week, growing nearly three-fold from the start to the end of the quarter. That momentum has continued into the current quarter, with total prescriptions written exceeding 400, representing over 40% growth over just the last four weeks alone.
先從處方與患者動態談起。REDEMPLO 的上市持續建立強勁且一致的動能。截至 2026 年 3 月 31 日止的會計年度第二季,我們看到處方量逐週加速,從季初到季末成長近三倍。這股動能延續到本季,目前開立的總處方數已超過 400 張,僅在過去四週就成長逾 40%。
The awareness and conviction driving this prescription growth are encouraging. REDEMPLO awareness among the prescribers who matter most has increased meaningfully. Critically, this awareness has translated into conviction. Nearly all REDEMPLO prescribers surveyed report being satisfied or highly satisfied with the product, and REDEMPLO is perceived strongest on the efficacy outcomes FCS patients care about most, triglyceride reduction and acute pancreatitis risk reduction.
推動處方成長的認知度與信心令人鼓舞。在最關鍵的開立處方醫師族群中,REDEMPLO 的認知度已顯著提升。更重要的是,這份認知已轉化為信心。受訪的 REDEMPLO 開立處方醫師幾乎都表示對產品滿意或非常滿意;且在 FCS 患者最重視的療效指標上——三酸甘油脂降低與急性胰臟炎風險降低——REDEMPLO 的表現被認為最為突出。
The patient mix continues to reflect what we expected. Approximately 85% of prescriptions are from patients naive to the APOC3 class, a strong signal that physicians are identifying and treating FCS patients who have never had access to an effective therapy. Switch patients largely account for the remainder.
患者組成持續符合我們的預期。約 85% 的處方來自未曾使用 APOC3 類別治療的患者,這是強烈訊號,顯示醫師正在辨識並治療過去從未能取得有效療法的 FCS 患者。其餘大多為轉換用藥的患者。
Patient persistence data is equally encouraging. Refill activity is accelerating meaningfully, an important early validation of both clinical effectiveness and patient satisfaction with REDEMPLO's once-quarterly dosing profile.
患者持續用藥(persistence)數據同樣令人鼓舞。續配(refill)活動顯著加速,這是對 REDEMPLO 臨床有效性與患者對其每季一次給藥方案滿意度的重要早期驗證。
Geographic distribution of prescribing is balanced across the country. This breadth of prescriber activation across all territories signals that patient identification capability is building at scale across the organization, not concentrated in a handful of high-volume centers, which gives us confidence in the durability of the prescription growth trajectory.
處方的地理分布在全國各地相當均衡。各區域皆有廣泛的醫師啟動開立處方,顯示整個組織的患者辨識能力正在規模化建立,而非集中在少數高量中心,這讓我們對處方成長軌跡的持久性更具信心。
Turning to payer access, we are making meaningful and consistent progress. Our market access team has been actively engaged with the largest payers in the country, covering the vast majority of US lives, to support continued patient access. These discussions are proceeding as expected and, in some cases, have already led to REDEMPLOâs improved coverage. Additional formulary coverage decisions are expected in the coming months across both commercial and government segments.
談到支付方可近性(payer access),我們正取得具意義且持續的進展。我們的市場准入團隊一直積極與全國最大型的支付方接洽,這些支付方覆蓋了美國絕大多數受保人,以支持患者持續取得治療。這些討論如預期推進,且在某些情況下已促成 REDEMPLO 給付覆蓋的改善。未來幾個月內,商業與政府兩大類別預期將陸續作出更多處方集(formulary)覆蓋決策。
A particularly important development in the payer landscape is the diagnostic pathway flexibility that major payers are recognizing. The coverage policies taking shape across major payers reflect both genetic testing and clinical criteria as valid routes to diagnosis. This is critical for ensuring that all appropriate REDEMPLO patients can access treatment because a meaningful proportion of real-world FCS patients are clinically diagnosed rather than genetically confirmed, and policies that require genetic confirmation as a prerequisite would create an unnecessary and inappropriate barrier.
在付款方版圖中,一項特別重要的發展是主要付款方正認可診斷途徑的彈性。各大付款方逐步成形的給付政策,反映出基因檢測與臨床標準皆可作為有效的診斷途徑。這一點對於確保所有適合的 REDEMPLO 患者都能取得治療至關重要,因為在真實世界中,相當比例的 FCS 患者是以臨床方式診斷,而非以基因檢測確認;若政策將基因確認作為先決條件,將造成不必要且不恰當的障礙。
As Chris mentioned, we have made a proactive decision to reduce the list price of REDEMPLO to $45,000 per patient per year. This decision reflects our commitment to optimizing market access for FCS patients and is consistent with our belief that a competitive and rational price point accelerates formulary decisions and reduces friction in the prior authorization process. We have always believed that REDEMPLO's clinical profile is best-in-class, and the $45,000 per year price point reflects a premium value supported by the clinical evidence.
如 Chris 所提到,我們已主動決定將 REDEMPLO 的定價(list price)下調至每位患者每年 45,000 美元。此決策反映我們致力於為 FCS 患者最佳化市場可近性,並符合我們的看法:具競爭力且合理的價格水準可加速處方集(formulary)決策並降低事前授權(prior authorization)流程中的摩擦。我們一直相信 REDEMPLO 的臨床特性為同類最佳,而每年 45,000 美元的價格水準反映了由臨床證據所支持的溢價價值。
With the FCS launch performing ahead of our expectations, and with potential expansion into SHTG on the horizon, we are making deliberate and sequenced investments to scale our commercial infrastructure. I will speak more on this in the future, but the field infrastructure we are building will be sized and structured for both the current expanded FCS accessible population and also the future SHTG opportunity as it unfolds in the future.
隨著 FCS 上市表現優於我們的預期,且 SHTG 的潛在擴適應症也近在眼前,我們正以審慎且循序的方式投資,以擴大我們的商業化基礎建設。我未來會再更詳細說明,但我們正在建置的前線商業團隊規模與架構,將同時因應目前擴大的 FCS 可觸及族群,以及未來逐步展開的 SHTG 機會。
On international expansion, REDEMPLO received regulatory approval in both Canada and China in January and most recently in Australia last month. All three markets are currently in pre-launch phase as we work through the pricing and reimbursement frameworks in each country. We look forward to providing updates on those timelines as they develop.
在國際擴張方面,REDEMPLO 已於 1 月在加拿大與中國取得監管核准,並於上月最近在澳洲取得核准。目前三個市場皆處於上市前階段,我們正就各國的定價與給付(reimbursement)架構推進相關工作。我們期待在時程逐步明朗時提供最新進展。
Also last month, CHMP, the Committee for Medicinal Products for Human Use, recommended EU marketing authorization for REDEMPLO in Europe for FCS without requiring genetic confirmation. Consequently, we anticipate an EMA approval decision in the June to July timeframe.
同樣在上月,CHMP(人用藥品委員會)建議在歐洲核准 REDEMPLO 用於 FCS 的歐盟上市許可,且不要求基因確認。因此,我們預期 EMA 的核准決定將落在 6 月至 7 月期間。
We intend to commercialize REDEMPLO directly in Europe, supported by contracted infrastructure which encompasses market access strategy, account management deployment, medical science liaison support, and broader stakeholder engagement including medical congresses and patient advocacy group engagement. We believe this model is the right approach for Arrowhead and are pleased with the readiness of that team as we approach the anticipated EMA decision.
我們計畫在歐洲以自建方式直接商業化 REDEMPLO,並由委外簽約的基礎建設支援,涵蓋市場准入策略、客戶/帳戶管理部署、醫學科學聯絡員(MSL)支援,以及更廣泛的利害關係人互動,包括醫學會議與病友倡議團體的合作。我們相信此模式是 Arrowhead 的正確作法,且在接近預期的 EMA 決策之際,我們對該團隊的就緒程度感到滿意。
Finally, I want to comment on the SHTG program, which represents the most significant near-term value catalyst for the Cardiometabolic franchise. We are approaching what we expect to be a highly meaningful series of milestones. Top-line results from SHASTA-3 and SHASTA-4, our two registrational Phase 3 studies in severe hypertriglyceridemia, are expected in Q3. We head into the data readout with confidence grounded in the strength of REDEMPLO's established mechanism of action and the consistency of the APOC3 biology we have observed across our full clinical program to date.
最後,我想談談 SHTG 計畫;這是心代謝(Cardiometabolic)事業群近期最重要的價值催化劑。我們正接近一系列我們預期極具意義的里程碑。我們在重度高三酸甘油脂血症的兩項註冊性第三期研究 SHASTA-3 與 SHASTA-4,其主要結果(top-line results)預計於第三季公布。我們對即將到來的數據揭盲充滿信心,這份信心建立在 REDEMPLO 已確立的作用機轉之強度,以及我們迄今在完整臨床計畫中所觀察到 APOC3 生物學的一致性。
We also intend to present the data at a major medical congress, which we hope will be with a simultaneous publication in a top tier medical journal. We then expect to file an sNDA with the FDA before the end of 2026, with an anticipated regulatory approval based on an expected standard review timeline targeted in second half of 2027. Additional regulatory filings in other jurisdictions are planned to follow thereafter.
我們也計畫在重要的醫學年會上發表這些數據,並希望能同步在頂尖醫學期刊發表。之後我們預期在 2026 年底前向 FDA 遞交補充新藥申請(sNDA),並依預期的標準審查時程,目標在 2027 年下半年取得監管核准。其後也規劃在其他司法管轄區進行額外的監管申請。
The SHTG opportunity represents a patient population that is substantially larger than FCS, with over one million high-risk patients in the United States alone. The commercial infrastructure investments we are making for FCS today are also designed with that launch in mind.
SHTG 的機會所對應的患者族群遠大於 FCS,僅在美國就有超過一百萬名高風險患者。我們目前為 FCS 所進行的商業化基礎建設投資,也同樣是以該上市計畫為設計前提。
In summary, the REDEMPLO launch is progressing well and continues to exceed our expectations across prescription volume, patient dynamics, and payer access. Physician satisfaction and forward prescribing intent are both extremely strong. Refill activity is accelerating. And we have a series of highly anticipated milestones in the second half of 2026 that we believe will be transformative for the Cardiometabolic franchise and for Arrowhead.
總結而言,REDEMPLO 的上市推進順利,且在處方量、患者動態與付款方准入方面持續超出我們的預期。醫師滿意度與未來持續開立處方的意向都非常強勁。續方(refill)活動正在加速。此外,我們在 2026 年下半年將迎來一系列備受期待的里程碑,我們相信這些將為心代謝事業群以及 Arrowhead 帶來轉型性的影響。
With that, Iâll turn the call over to James Hamilton to discuss the broader R&D portfolio.
接下來,我將把電話會議交給 James Hamilton,請他討論更廣泛的研發產品組合。
James Hamilton - Chief Medical Officer and Head of R&D
James Hamilton - Chief Medical Officer and Head of R&D
Thank you, Andy. As Chris mentioned, we have a very broad pipeline with over 20 clinical programs, so I will focus on areas with upcoming readouts.
謝謝你,Andy。如 Chris 所提到,我們擁有非常廣泛的研發管線,包含超過 20 項臨床計畫,因此我將聚焦於近期將有數據讀出(readouts)的領域。
First, Iâd like to announce that we are planning to host three webcasts over the coming months as part of our R&D Webinar Summer Series. Each webcast will cover a specific aspect of our pipeline where we expect to have upcoming data readouts this year. These include cardiometabolic, including Plozasiran, zodasiran, and ARO-DIMER-PA; obesity, including ARO-INHBE and ARO-ALK7; and, ARO-MAPT, including the blood-brain-barrier, or BBB, platform.
首先,我想宣布我們計畫在未來幾個月內舉辦三場網路直播,作為我們「研發網路研討會夏季系列」的一部分。每場直播將涵蓋我們管線中的特定面向,且我們預期今年將有相關數據讀出。主題包括:心代謝領域(包含 Plozasiran、zodasiran 與 ARO-DIMER-PA);肥胖領域(包含 ARO-INHBE 與 ARO-ALK7);以及 ARO-MAPT(包含血腦障壁(BBB)平台)。
Iâll now give status updates from the quarter on these specific areas.
接下來我將就本季在這些特定領域提供進度更新。
First, letâs review the suite of Plozasiran Phase 3 studies, SHASTA-3, SHASTA-4, SHASTA-5, and MUIR-3, designed to support supplemental NDA filings to expand the REDEMPLO label beyond genetic and clinical FCS into patients with SHTG.
首先,讓我們回顧 Plozasiran 的一系列第三期研究:SHASTA-3、SHASTA-4、SHASTA-5 與 MUIR-3;這些研究旨在支持補充 NDA 申請,將 REDEMPLO 的標籤適應症從基因與臨床診斷的 FCS,擴展至 SHTG 患者。
SHASTA-3 and SHASTA-4 together enrolled over 750 patients and have a primary endpoint of change in triglycerides from basline, with a key secondary endpoint of acute pancreatitis rates. MUIR-3, which enrolled over 1400 patients, is designed to supplement the SHASTA studies with additional patient safety data.
SHASTA-3 與 SHASTA-4 合計納入超過 750 名患者,其主要終點為三酸甘油脂相較基線(baseline)的變化,關鍵次要終點為急性胰臟炎發生率。MUIR-3 納入超過 1,400 名患者,旨在以額外的患者安全性數據補充 SHASTA 研究。
We are also enrolling patients at high risk of acute pancreatitis in SHASTA-5 to directly assess the ability of Plozasiran to reduce the risk of acute pancreatitis as the primary endpoint. Should SHASTA-3 and -4 show a statistically significant improvement in acute pancreatitis risk, we will re-assess whether there is added value in continuing SHASTA-5.
我們也正在 SHASTA-5 中招募急性胰臟炎高風險患者,以主要終點直接評估 Plozasiran 降低急性胰臟炎風險的能力。若 SHASTA-3 與 SHASTA-4 在急性胰臟炎風險方面顯示具統計顯著性的改善,我們將重新評估是否繼續進行 SHASTA-5 仍具有額外價值。
We remain on schedule to complete the blinded portion of the SHASTA-3, SHASTA-4, and MUIR-3 in mid-2026 to support a planned topline data readout in the third quarter. This would further support our plans for an sNDA submission for SHTG before the end of this year.
我們仍按計畫在 2026 年年中完成 SHASTA-3、SHASTA-4 與 MUIR-3 的盲態(blinded)部分,以支持第三季規劃的主要結果數據讀出。這也將進一步支持我們在今年底前就 SHTG 遞交 sNDA 的計畫。
Before moving on to zodasiran, Iâd like to highlight a presentation we made with new long-term efficacy and safety data for Plozasiran across a spectrum of patients with hypertriglyceridemia at the American College of Cardiology conference in March. The data were from a two-year open-label extension of the two Phase 2b double-blind, placebo-controlled studies of Plozasiran; SHASTA-2, conducted in adults with severe hypertriglyceridemia; and MUIR, which enrolled patients with hypertriglyceridemia.
在進入 zodasiran 之前,我想強調我們於 3 月在美國心臟學會(American College of Cardiology)年會上所做的一場發表,內容為 Plozasiran 在不同高三酸甘油脂血症患者族群中的最新長期療效與安全性數據。這些數據來自兩項 Plozasiran 第二期 b、雙盲、安慰劑對照研究的兩年期開放標籤延伸試驗:SHASTA-2(於重度高三酸甘油脂血症成人中進行)以及 MUIR(納入高三酸甘油脂血症患者)。
During the two-year open label extension, patients saw median reductions in their triglycerides of 83% in sHTG patients from SHASTA-2 and 67% in HTG patients from MUIR, with additional reductions in remnant and non-HDL-cholesterol.
在為期兩年的開放標籤延伸試驗期間,患者的三酸甘油脂中位數降幅為:來自 SHASTA-2 的 sHTG 患者下降 83%,來自 MUIR 的 HTG 患者下降 67%,且殘餘膽固醇與非 HDL-膽固醇亦有進一步降低。
96% of sHTG patients achieved TGs below 500 milligrams per deciliter and 63% achieved TGs below 150 milligrams per deciliter, with 93% of HTG patients achieving TGs below 150 milligrams per deciliter. Importantly, no adjudicated acute pancreatitis events occurred in any patient receiving Plozasiran during the two-year Phase 2b Open-Label Expansion Study. These findings support the potential of Plozasiran as a promising new approach to managing patients with moderate to severe HTG phenotypes who are at risk of AP and potentially other cardiometabolic comorbidities.
96% 的 sHTG 患者達到 TG 低於每分升 500 毫克,且 63% 達到 TG 低於每分升 150 毫克;HTG 患者中有 93% 達到 TG 低於每分升 150 毫克。重要的是,在為期兩年的第 2b 期開放標籤延伸研究中,所有接受 Plozasiran 的患者均未發生任何經裁定的急性胰臟炎事件。這些結果支持 Plozasiran 具有潛力,作為一種前景可期的新方法,用於管理具有中度至重度 HTG 表型、且有 AP 以及可能其他心臟代謝共病風險的患者。
I now want to give a quick update on the YOSEMITE Phase 3 study of zodasiran, which is being developed as a potential treatment for homozygous familial hypercholesterolemia, or HoFH, a rare genetic condition that leads to severely elevated LDL-cholesterol and early onset cardiovascular disease.
我現在想就 zodasiran 的 YOSEMITE 第 3 期研究做一個簡要更新。zodasiran 正在開發作為同型合子家族性高膽固醇血症(homozygous familial hypercholesterolemia,HoFH)的潛在治療;HoFH 是一種罕見遺傳疾病,會導致 LDL-膽固醇嚴重升高並造成心血管疾病早發。
Zodasiran is the fourth investigational RNAi-based candidate developed by Arrowhead to reach late-stage pivotal studies. YOSEMITE is designed to enroll approximately 60 individuals with HoFH over the age of 12, who will be randomized two to one to receive 5 doses of 200 milligrams zodasiran or placebo. The primary endpoint is the percent change from baseline to month 12 in fasting LDL-C. Enrollment has been on track and we are confident that the study can be fully enrolled this year to enable study completion and potential NDA filing before the end of 2027.
Zodasiran 是 Arrowhead 開發的第四個以 RNAi 為基礎、進入後期關鍵性研究階段的在研候選藥物。YOSEMITE 設計納入約 60 名年齡 12 歲以上的 HoFH 受試者,並以 2:1 隨機分派接受 5 劑 200 毫克 zodasiran 或安慰劑。主要終點為空腹 LDL-C 自基線至第 12 個月的百分比變化。收案進度符合預期,我們有信心可於今年完成全數收案,以便在 2027 年底前完成研究並可能提交 NDA 申請。
The last program within cardiometabolic is ARO-DIMER-PA, the first dual-functional siRNA designed to silence the expression of two genes with a single RNAi molecule. ARO-DIMER-PA is being developed as a potential treatment for ASCVD due to mixed hyperlipidemia by silencing expression of both PCSK9 and APOC3.
心臟代謝領域的最後一個專案是 ARO-DIMER-PA,這是首個雙功能 siRNA,旨在以單一 RNAi 分子沉默兩個基因的表達。ARO-DIMER-PA 正在開發作為因混合型高脂血症所致 ASCVD 的潛在治療,透過同時沉默 PCSK9 與 APOC3 的表達。
In January we initiated a Phase 1/2a placebo-controlled dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and effects on LDL-Cholesterol and triglycerides using single-dose ARO-DIMER-PA in part 1 and multiple doses in part 2, in up to 78 adults with mixed hyperlipidemia. Enrollment in this study has been rapid, and we are on schedule to have sufficient data to provide the first clinical readout in Q3 of 2026.
今年 1 月,我們啟動了一項第 1/2a 期、安慰劑對照、劑量遞增研究,以評估 ARO-DIMER-PA 的安全性、耐受性、藥物動力學、藥效學,以及對 LDL-膽固醇與三酸甘油脂的影響;第 1 部分採單次給藥,第 2 部分採多次給藥,最多納入 78 名混合型高脂血症成人。本研究收案迅速,我們按計畫可取得足夠數據,於 2026 年第 3 季提供首次臨床讀出。
This is a very interesting program, and we think the preclinical data have been highly compelling. We have some innovative ideas on later-stage trial designs that potentially accelerate the path to regulatory approval, so we are eager to have a first clinical readout to start moving ahead with later studies, if supported by initial data.
這是一個非常有趣的專案,我們認為其臨床前數據極具說服力。我們對後期試驗設計有一些創新構想,可能加速通往監管核准的路徑,因此我們迫切希望取得首次臨床讀出;若初步數據支持,我們將開始推進後續研究。
Lastly, I want to give an update on the status of the ARO-MAPT first-in-human study. ARO-MAPT is being developed as a potential treatment for tauopathies including Alzheimerâs disease, a progressive neurodegenerative disease characterized by cognitive and functional decline. Alzheimerâs disease is the most common cause of dementia, affecting an estimated 32 million people worldwide, and is part of a group of neurodegenerative diseases called tauopathies that are marked by the abnormal tau accumulation and formation of tau tangles in neurons.
最後,我想更新 ARO-MAPT 首次人體研究的進展狀態。ARO-MAPT 正在開發作為 tau 蛋白病(tauopathies)的潛在治療,包括阿茲海默症;阿茲海默症是一種進行性神經退化疾病,其特徵為認知與功能衰退。阿茲海默症是失智症最常見的原因,估計全球影響約 3,200 萬人;它屬於一組稱為 tau 蛋白病的神經退化疾病,其特徵為 tau 異常累積並在神經元中形成 tau 纏結。
Tau-related pathology may be a critical driver of neurodegeneration, and targeting tau is a promising strategy to potentially slow or stop cognitive and functional decline.
與 tau 相關的病理可能是神經退化的關鍵驅動因素,而以 tau 為標的被視為一項有前景的策略,可能減緩或阻止認知與功能衰退。
ARO-MAPT is Arrowheadâs first investigational RNAi-based therapy to utilize a new proprietary delivery system which, in preclinical studies, has achieved blood brain barrier penetration and deep knockdown of target genes across the central nervous system, including deep brain regions, after subcutaneous injection. This underscores Arrowheadâs leadership in the delivery of siRNA to multiple tissues and cell types throughout the body utilizing our proprietary and differentiated Targeted RNAi Molecule, or TRiM, platform.
ARO-MAPT 是 Arrowhead 首個採用全新自有遞送系統的在研 RNAi 療法;在臨床前研究中,該系統於皮下注射後可穿透血腦屏障,並在整個中樞神經系統(包括深部腦區)達到目標基因的深度敲低。這凸顯 Arrowhead 在將 siRNA 遞送至全身多種組織與細胞類型方面的領先地位,並運用我們自有且具差異化的 Targeted RNAi Molecule(TRiM)平台。
In December 2025 we dosed the first subjects in a Phase 1/2 clinical trial of ARO-MAPT. This study is a placebo-controlled dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-MAPT in up to 64 healthy subjects and up to 48 patients with mild cognitive impairment due to Alzheimerâs disease and mild Alzheimerâs disease dementia.
在 2025 年 12 月,我們於 ARO-MAPT 的第 1/2 期臨床試驗中完成首批受試者給藥。本研究為安慰劑對照、劑量遞增試驗,旨在評估 ARO-MAPT 的安全性、耐受性、藥物動力學與藥效學;最多納入 64 名健康受試者,以及最多 48 名因阿茲海默症導致之輕度認知障礙患者與輕度阿茲海默症失智患者。
In Part 1a of the study, healthy subjects will receive one or three weekly doses of ARO-MAPT or placebo by subcutaneous injection. In Parts 1b and Part 2 health volunteers and Alzheimers disease patients respectively will receive multiple escalating doses of ARO-MAPT or placebo.
在研究第 1a 部分,健康受試者將以皮下注射方式接受 ARO-MAPT 或安慰劑,每週一次,給予 1 劑或 3 劑。在第 1b 部分與第 2 部分中,健康志願者與阿茲海默症患者(分別對應)將接受多次、逐步遞增劑量的 ARO-MAPT 或安慰劑。
We are nearing completion of enrollment of the single-dose portion of the study in healthy volunteers and have begun enrollment in the multi dose cohorts in both healthy volunteers and patients with Alzheimerâs disease. This keeps us on pace for an initial data readout at the end of Q3 or early Q4.
我們即將完成健康志願者單次給藥部分的收案,並已開始在健康志願者與阿茲海默症患者的多次給藥隊列中收案。這使我們得以維持進度,預計在第 3 季末或第 4 季初取得初步數據讀出。
I will now turn the call over to Dan Apel.
我現在把電話會議交給 Dan Apel。
Daniel Apel - Chief Financial Officer
Daniel Apel - Chief Financial Officer
Thank you, James, and good afternoon everyone. As we reported today, net loss for the quarter ended March 31, 2026 was $132.7 million, or a loss of $0.93 per share, based on 142.4 million fully diluted weighted-average shares outstanding. This compares to net income of $370.4 million, or $2.75 per share, for the quarter ended March 31, 2025, based on 134.5 million fully diluted weighted-average shares outstanding in that quarter.
謝謝你,James,各位下午好。如我們今日所報告,截至 2026 年 3 月 31 日止季度的淨損為 1.327 億美元,或每股虧損 0.93 美元,係以 1.424 億股完全稀釋加權平均流通股數計算。相較之下,截至 2025 年 3 月 31 日止季度的淨利為 3.704 億美元,或每股盈餘 2.75 美元,係以該季度 1.345 億股完全稀釋加權平均流通股數計算。
Recall that in the prior year quarter, we recorded over $540 million in revenue solely related to the Sarepta transaction executed at that time. Revenue for this quarter totaled $74 million, driven primarily by our license and collaboration agreements with Sarepta and with Novartis. Of this amount, approximately $42 million related to the Sarepta collaboration. This includes $28 million from ongoing recognition of the initial Sarepta consideration, $10 million related to reimbursement of incurred pre-clinical collaboration program costs, and $4 million for clinical supply provided to them under a clinical supply agreement.
請回想去年同期,我們認列超過 5.40 億美元的營收,且完全與當時執行的 Sarepta 交易相關。本季度營收合計 7,400 萬美元,主要由我們與 Sarepta 以及 Novartis 的授權與合作協議所帶動。其中約 4,200 萬美元與 Sarepta 合作相關。這包括:持續認列 Sarepta 初始對價所帶來的 2,800 萬美元、就已發生之臨床前合作專案成本的補償 1,000 萬美元,以及依臨床供應協議提供臨床用藥所取得的 400 萬美元。
In addition, we recognized $20 million of the $200 million upfront payment received from Novartis in October under that agreement, bringing year to date recognition of the Novartis upfront to $54 million, with the remaining $146 million to be deferred over time as we fulfill our preclinical obligations. We also recorded $11 million related to the Asset Purchase Agreement between Sanofi and Visirna to develop and commercialize investigational Plozasiran in Greater China.
此外,我們亦就與 Novartis 的協議,認列其於 10 月支付之 2 億美元預付款中的 2,000 萬美元,使得本年度迄今已認列的 Novartis 預付款達 5,400 萬美元;其餘 1.46 億美元將隨我們履行臨床前義務而於未來期間遞延認列。我們也就 Sanofi 與 Visirna 之間的資產購買協議認列 1,100 萬美元,該協議旨在於大中華區開發並商業化在研藥物 Plozasiran。
Visirna as you know is our majority-owned subsidiary with operations n China, and the amount recognizes almost entirely due to the January approval of FCS in that region by the Chinese National Medical Products Administration. As mentioned previously, we are not intending to headline specific REDEMPLO product sales numbers until such time as they become a meaningful driver to our financials.
如各位所知,Visirna 是我們持股過半的子公司,在中國有營運;而該金額幾乎完全是因為中國國家藥品監督管理局於今年 1 月核准該地區的 FCS 所致。如先前所提,我們不打算在 REDEMPLO 的產品銷售數字成為對財務具實質意義的驅動因素之前,就以特定的 REDEMPLO 產品銷售數字作為對外重點。
That said, net sales can be derived from our disclosures, as the difference between total net revenue and collaboration revenue, and represents approximately $1 million for the quarter. This is our first full quarter of REDEMPLO sales and, on a unit basis, that figure compares favorably to the first full commercial quarter of the other approved APOC3 inhibitor.
話雖如此,淨銷售額可由我們的揭露推算,即總淨營收與合作營收之差,本季約為 100 萬美元。這是我們 REDEMPLO 銷售的第一個完整季度;以銷售單位來看,該數字與另一款已核准的 APOC3 抑制劑之第一個完整商業化季度相比,表現相當不錯。
Turning now to expenses, total operating expenses for the quarter were approximately $215 million, roughly flat with operating expenses in the first fiscal quarter. This compares to $162 million in the prior-year quarter, representing an increase of $53 million year over year. This increase was driven by $40 million of higher R&D expenses and $13 million of higher SG&A expenses, fully in line with our expectations.
接著談費用,本季總營運費用約為 2.15 億美元,與第一會計季度的營運費用大致持平。相較於去年同期的 1.62 億美元,年增 5,300 萬美元。此增幅主要來自研發費用增加 4,000 萬美元,以及銷售、一般及管理費用(SG&A)增加 1,300 萬美元,完全符合我們的預期。
The increase in R&D expense was primarily attributable to ongoing progression of our Phase 3 registrational studies for Plozasiran in sHTG as well as our early-stage pipeline programs including the DIMER and MAPT. Fiscal year to date, almost two-thirds of the clinical trial spend can be attributed to our Plozasiran phase three studies. As James already mentioned, the registrational sHTG studies for Plozasiran should readout in the summer, and clinical trial spend for these programs should thereafter moderate accordingly.
研發費用的增加,主要歸因於我們在 sHTG 適應症上推進 Plozasiran 的第 3 期註冊性研究,以及包含 DIMER 與 MAPT 在內的早期管線計畫持續推進。本會計年度迄今,臨床試驗支出中將近三分之二可歸因於 Plozasiran 的第 3 期研究。如 James 先前提到,Plozasiran 的註冊性 sHTG 研究預計於夏季讀出,之後這些計畫的臨床試驗支出也將相應趨緩。
SG&A expenses increased year over year compared to the prior yearâs second fiscal quarter, primarily driven by ongoing investments to support the commercialization of REDEMPLO. As previously discussed, we are continuing to build our commercial capabilities to fully support the FCS launch. We continue to leverage and invest in these capabilities to support REDEMPLO in FCS, while also positioning the organization to support potential future launch in sHTG. And we expect ultimately to leverage these same capabilities for the advancement of zodasiran for the treatment of HoFH.
SG&A 費用相較去年會計年度第二季度年增,主要是因持續投入以支援 REDEMPLO 的商業化。如先前討論,我們正持續建置商業化能力,以全面支援 FCS 的上市推廣。我們持續運用並投資這些能力,以支援 REDEMPLO 在 FCS 的推廣,同時也讓組織具備支援未來可能在 sHTG 上市的能力。此外,我們預期最終也將運用同樣的能力,推進 zodasiran 用於治療 HoFH。
Turning to the balance sheet. Cash and investments on hand totaled nearly $1.8 billion as of March 31, 2026. Common shares outstanding at quarter end were 140.6 million. In this quarter alone, we brought in over $1 billion including approximately $850 million, net, from our January financing transactions, consisting of a concurrent offering of 0% convertible senior notes and common stock, along with associated capped call transactions.
接著看資產負債表。截至 2026 年 3 月 31 日,手上現金與投資合計接近 18 億美元。季度末流通在外普通股為 1.406 億股。僅本季我們就募集超過 10 億美元,其中約 8.5 億美元(淨額)來自 1 月的融資交易,包括同步發行 0% 可轉換優先票據與普通股,並搭配相關的 capped call 交易。
Other notable inflows in the quarter include the $200 million received from Sarepta upon achieving the second DM1 program milestone, as well as a $50 million anniversary payment under the Sarepta long-term collaboration agreement. All of this is very much in line with the information provided previously during our February earnings call.
本季其他值得注意的資金流入包括:因達成 DM1 計畫第二個里程碑而自 Sarepta 收到的 2 億美元,以及依 Sarepta 長期合作協議收到的 5,000 萬美元周年付款。上述內容皆與我們先前在 2 月財報電話會議中提供的資訊高度一致。
We believe our strong balance sheet provides us with significant financial flexibility to support ongoing clinical development, to advance current and future commercialization activities, and to execute against our long-term strategic priorities.
我們相信,強健的資產負債表為我們提供了顯著的財務彈性,可支援持續的臨床開發、推進現有與未來的商業化活動,並落實我們的長期策略優先事項。
With that brief overview, I will now turn the call back to Chris.
以上為簡要概述,接下來我把電話交回給 Chris。
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Thanks Dan.
謝謝你,Dan。
As we build out our commercial team and focus on efficiently bringing REDEMPLO to the patients who need it, we have not lost sight on continuing to expand our pipeline and ultimately increasing the number of medicines we can offer to a wide variety of patients.
在我們擴編商業團隊並專注於有效率地將 REDEMPLO 帶給有需要的病患之際,我們也沒有忽略持續擴展管線,並最終增加可提供給各類病患的藥物數量。
Our business has become more complex as we grow in all these areas, but we continue to innovate and execute well. We see multiple key potential value creating events in the second half of 2026 that, together, speak to our priorities.
隨著我們在各領域成長,業務變得更為複雜,但我們仍持續創新並良好執行。我們看到 2026 年下半年有多項關鍵、可能創造價值的事件,整體而言也反映了我們的優先事項。
Here are just a few of the events we are tracking.
以下僅列出我們正在追蹤的幾項事件。
SHASTA-3, SHASTA-4, and MUIR-3, which is the suite of Phase 3 clinical studies designed to support an sNDA for REDEMPLO in patients with SHTG, is on schedule for completion and topline readout in Q3. The first clinical readout for ARO-DIMER-PA, targeting both PCSK9 and APOC3, for LDL and TG lowering is also expected in Q3.
SHASTA-3、SHASTA-4 與 MUIR-3(這一組第 3 期臨床研究旨在支援 REDEMPLO 用於 SHTG 病患的 sNDA)正按計畫進行,預計於第三季完成並公布主要結果(topline readout)。此外,針對 PCSK9 與 APOC3、用於降低 LDL 與 TG 的 ARO-DIMER-PA,其首次臨床讀出也預計在第三季。
The first clinical readout for ARO-MAPT is expected around the end of Q3 or early Q4. It is being developed as a potential treatment for tauopathies including Alzheimerâs disease, and is our first program using the CNS delivery platform designed to cross the blood-brain-barrier after systemic delivery via subcutaneous administration. Additional ARO-INHBE and ARO-ALK7 data releases are planned in 2026 for this novel non-incretin strategy which had quite encouraging early data, particularly in diabetic obese patients in combination with tirzepatide and with liver fat reductions as mono-therapy or in combination with tirzepatide.
ARO-MAPT 的首次臨床讀出預計在第三季末或第四季初左右。該計畫正開發為治療 tau 蛋白相關疾病(tauopathies,包括阿茲海默症)的潛在療法,並且是我們第一個採用中樞神經系統(CNS)遞送平台的計畫;該平台設計為在以皮下注射進行全身性給藥後,能穿越血腦障壁。針對此一新穎的非腸泌素(non-incretin)策略,我們也規劃於 2026 年發布更多 ARO-INHBE 與 ARO-ALK7 的數據;早期數據相當令人鼓舞,特別是在糖尿病肥胖病患中與 tirzepatide 併用,以及作為單藥或與 tirzepatide 併用時的肝脂下降效果。
As James mentioned, we are planning to webcast three presentations as part of our Summer Series of R&D Webinars to go over cardiometabolic broadly, obesity, and ARO-MAPT. These can serve as a review of the programs and results to date, and as a primer for the potentially important readouts coming up later this year.
如 James 所提,我們計畫在「夏季研發網路研討會系列」中以網路直播方式進行三場簡報,主題涵蓋廣義的心代謝領域、肥胖,以及 ARO-MAPT。這些簡報可用來回顧目前為止的計畫與成果,並作為今年稍晚可能出現的重要讀出之前的導讀。
Thank you for joining us today and I would now like to open the call to your question
感謝各位今天加入我們,接下來我想開放電話讓各位提問
Operator
Operator
(Operator Instructions) Edward Tenthoff, Piper Sandler.
(接線員指示)Edward Tenthoff,Piper Sandler。
Edward Tenthoff - Analyst
Edward Tenthoff - Analyst
Great. Thanks for all the detail in the update today. My question only has to do with looking at the upcoming SH severe hypertriglyceridemia readouts. When it comes to pancreatitis as a secondary, is the plan to pool SHASTA-4 -- 3 and 4? And what are sort of the assumptions around pancreatitis?
很好。感謝今天更新提供這麼多細節。我的問題主要是關於即將公布的 SH 重度高三酸甘油脂血症(severe hypertriglyceridemia)讀出。就胰臟炎作為次要終點而言,計畫是否會把 SHASTA-4——也就是 3 與 4——合併分析?另外,對胰臟炎方面的假設大概是什麼?
James Hamilton - Chief Medical Officer and Head of R&D
James Hamilton - Chief Medical Officer and Head of R&D
Yeah. Ed. Thanks for the question. This is James. Yeah, you got that right. The plan is to pool both of those studies, SHASTA-3 and 4 and we'll analyze -- do a meta-analysis of those two studies to look at pancreatitis event rates, both rates of events in individual patients, and a total number of overall events.
是的。Ed,謝謝你的問題。我是 James。沒錯,你理解得正確。計畫是把 SHASTA-3 與 SHASTA-4 這兩項研究合併,並對兩項研究做統合分析(meta-analysis),以評估胰臟炎事件發生率,包括個別病患的事件發生率,以及整體事件總數。
And I think that's kind of all I can say on that. We're still blinded. And like we said, should have the data in Q3.
我想我大概只能說到這裡。我們目前仍在盲態中。如我們所說,應該會在第三季取得數據。
Operator
Operator
Jason Gerberry, Bank of America.
Jason Gerberry,美國銀行。
Jason Gerberry - Analyst
Jason Gerberry - Analyst
I just wanted to probe in a little bit more on the INHBE and ALK7 updates later this year and get your latest thoughts on these modalities. And I think investors have soured a little bit on INHBE after the wave data update.
我想再更深入追問一下今年稍晚關於 INHBE 與 ALK7 的更新,並聽聽你們對這些治療模式(modalities)的最新看法。我認為在 wave 的數據更新之後,投資人對 INHBE 的看法有些轉趨保守。
So just wanted to get your perspective on kind of what you need to see from these upcoming readouts to advance one or both for obesity treatment versus in the alternative potentially considering for a MASH indication?
所以只是想請教你對於接下來這些讀出結果的看法:你需要看到哪些內容,才會推進其中一個或兩個用於肥胖治療;或者作為替代方案,可能考慮用於 MASH 適應症?
James Hamilton - Chief Medical Officer and Head of R&D
James Hamilton - Chief Medical Officer and Head of R&D
Hi, Jason. This is James. Sure, happy to cover those questions. We've been saying all along really that we thought that this INHBE outset in access is interesting, but we thought the approach was to combine with GLP-1s and that's still our standpoint here. We think that there's potential, particularly in the type 2 diabetics for additional weight loss on top of tirzepatide or other GLP-1s with either [Alxtepin] knockdown or INHBE knockdown.
嗨,Jason。我是 James。當然,很樂意回答這些問題。我們一直以來都在說,我們認為這個 INHBE 的切入點在可及性方面很有意思,但我們認為策略是與 GLP-1 聯合使用,而這仍然是我們目前的立場。我們認為存在潛力,特別是在第二型糖尿病患者中,無論是 [Alxtepin] 敲低或 INHBE 敲低,都可能在替爾泊肽(tirzepatide)或其他 GLP-1 的基礎上帶來額外的減重效果。
What we've seen from the clinical study that we talked about earlier this year from INHBE with INHBE knockdown is really clear, redistribution of fat out of the liver, even with monotherapy, but also with combination therapy, and some additional changes and improvements in body composition reductions in total fat and visceral fat, particularly in that type 2 diabetic population.
我們從臨床研究中看到的、也是我們今年稍早提到的 INHBE(INHBE 敲低)結果非常清楚:即使是單藥治療,也能將脂肪從肝臟重新分配出去;而在聯合治療下亦然。此外,身體組成也出現一些額外變化與改善,包括總脂肪與內臟脂肪的下降,尤其是在第二型糖尿病族群中。
So I think that we look forward to more of the same and to sharing more of that liver fat data here in the coming quarter or so, and then in the second-half of the year, providing some additional updates on the changes in body composition. And some of the other parameters that we showed back in January.
所以我認為,我們期待看到更多類似的結果,並在接下來一季左右分享更多肝脂數據;然後在下半年,提供一些關於身體組成變化的進一步更新。以及我們在一月展示過的其他參數。
Operator
Operator
Bryan Tang, JPMorgan.
Bryan Tang,摩根大通(JPMorgan)。
Unidentified Participant 1
Unidentified Participant 1
This is Ron on for Bryan. Got some recorder. Just wanted to ask, can you guys give more color on the interactions you've had with peers that led to the recent price lowering? And what has been the feedback since your competitive action last month?
我是 Ron,代 Bryan 發問。我這邊有些錄音問題。想請問你們能否更詳細說明一下:你們與同業之間的互動,哪些因素導致了近期的降價?以及自從你們上個月採取競爭性行動後,收到的回饋如何?
Andy Davis - Senior Vice President and Head of the Global Cardiometabolic Franchise
Andy Davis - Senior Vice President and Head of the Global Cardiometabolic Franchise
Hi, everyone. This is Andy. So our interactions with payers today have been consistent, have been positive, and we're seeing payer policies, and these are public payer policies that reflect the ability to diagnose FCS patients through multiple diagnosis pathways, in particular through the clinical criteria that we saw in Palisade. So really, really positive conversations with payers about payer policies and about future coverage.
各位好。我是 Andy。我們目前與付款方(payers)的互動一直一致且正面;我們也看到付款方政策——這些是公開的付款方政策——反映出可透過多種診斷途徑來診斷 FCS 患者,特別是透過我們在 Palisade 看到的臨床標準。因此,我們與付款方就其政策及未來給付覆蓋的對話非常、非常正面。
Unidentified Participant 1
Unidentified Participant 1
Okay. And just a quick follow-up. Could you guys -- how should we think about the impact here to gross to net following the price lowering?
好的。再快速追問一下。你們認為這次降價後,對毛額到淨額(gross-to-net)的影響應該怎麼看?
Vincent Anzalone - Investor Relations
Vincent Anzalone - Investor Relations
What's our assumption on gross net with the pricing policy?
我們對於這個定價政策下的毛額到淨額(gross-to-net)假設是什麼?
Daniel Apel - Chief Financial Officer
Daniel Apel - Chief Financial Officer
Yeah. So we -- I mean, we ignore the -- I actually answered your second question. We lower the WAC to, as Chris already explained, to make it premium price to the competitor there.
是的。所以我們——我的意思是,我們先不談——其實我剛剛回答了你的第二個問題。我們把 WAC 下調,正如 Chris 已經解釋的,是為了讓我們相對競品維持溢價定價。
We are not actually going to give guidance on gross net. We have not seen anything substantial in that regard, nor do we currently expect it other than things that were statutorily required, such as Medicaid rebates and the manufacturer discount program and the like. So -- but we have a policy at the moment not to provide any sort of guidance on that at this stage.
我們實際上不會提供毛額到淨額(gross-to-net)的指引。就這方面而言,我們沒有看到任何實質性的變化,目前也不預期會有,除了法規要求的事項,例如 Medicaid 退費、製造商折扣計畫等。所以——但我們目前的政策是在這個階段不提供任何相關指引。
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
And let's just be clear, the lowering of the WAC from $60,000 to $45,000 had nothing to do with any pushback from payers. To the contrary, I think that those interactions have been quite positive. This is a lowering of the WAC in expectation of an expansion on the market into SHTG and to ensure that we are -- that that we would -- the payers would not require a step-through with our competitor.
另外要說清楚,WAC 從 60,000 美元降到 45,000 美元,與付款方的任何反彈完全無關。相反地,我認為那些互動相當正面。這次下調 WAC 是基於我們預期市場將擴展到 SHTG,並確保——確保付款方不會要求我們必須先使用競品(step-through)。
As we talked about, we are priced at a premium here. We think that's appropriate, given the characteristics of our drug versus the competitor's drug. And we think $45,000, actually, long-term, is probably quite a good price in terms of maximizing access to the drug across various subpopulations within SHTG.
如同我們談到的,我們在這裡是以溢價定價。考量到我們藥物相較於競品藥物的特性,我們認為這是合理的。而且我們認為 45,000 美元從長期來看,可能是相當不錯的價格,有助於在 SHTG 內不同次族群中最大化藥物可及性。
Operator
Operator
Mike Ulz, Morgan Stanley.
Mike Ulz,摩根士丹利(Morgan Stanley)。
Unidentified Participant 2
Unidentified Participant 2
[Colin] for Mike. Thank you for taking our questions and congrats on the quarter. I guess among the patients who were switchers, could you characterize, I guess the motivations for switching to REDEMPLO? Was it due to its clinical profile or could it be more pay related?
我是 [Colin],代 Mike 發問。謝謝你們回答我們的問題,也恭喜本季表現。我想問,在那些「轉換用藥」的患者中,你們能否描述一下他們轉用 REDEMPLO 的動機?是因為其臨床特性,還是可能更多與支付/給付相關?
Andy Davis - Senior Vice President and Head of the Global Cardiometabolic Franchise
Andy Davis - Senior Vice President and Head of the Global Cardiometabolic Franchise
Hi, Mike. This is Andy. Thanks for the question. We've seen really diversity of reasons for switch that include efficacy, safety, intolerability, and a variety of other reasons as well. So I wouldn't say there's one particular reason driving switch, there's a multitude of reasons.
嗨,Mike。我是 Andy。謝謝你的問題。我們看到轉換的原因非常多元,包括療效、安全性、耐受不良,以及其他各種原因。所以我不會說有某一個特定原因在驅動轉換,而是多重因素共同造成。
Operator
Operator
Maury Raycroft, Jefferies.
Maury Raycroft,Jefferies。
Maury Raycroft - Equity Analyst
Maury Raycroft - Equity Analyst
Maybe just a follow-up to Ed's question earlier on HASTA-3 and 4. Just wondering if there's an updated perspective on whether the blinded AP event rates are tracking in line with your expectations and whether you'd be able to generate enough events by the time the study completes? Or could you potentially even keep the study blinded a little bit longer to get an adequate number of total events?
可能是延續先前 Ed 對 HASTA-3 和 4 的提問。想請問是否有更新的看法:盲態下的 AP 事件發生率是否符合你們的預期走勢,以及在研究完成前是否能累積到足夠的事件數?或者你們是否可能讓研究多維持盲態一段時間,以取得足夠的總事件數?
James Hamilton - Chief Medical Officer and Head of R&D
James Hamilton - Chief Medical Officer and Head of R&D
Yeah. Hi, Maury. This is James. I'll take that question. So we're not giving any guidance on -- or sort of blow-by-blow details on the number of events we're seeing. Suffice it to say, we feel comfortable with the number of events we have seen and the big reveal will be in Q3. But no plans to extend the study or stay blinded for a longer period at this time.
是的。嗨,Maury。我是 James。我來回答這個問題。我們不會就目前看到的事件數提供任何指引,或逐一細節更新。簡單說,我們對已觀察到的事件數感到放心,而重大揭露會在第三季(Q3)。但目前沒有計畫延長研究或在此時把盲態維持更久。
Operator
Operator
Mani Foroohar, Leerink.
Mani Foroohar,Leerink。
Unidentified Participant 3
Unidentified Participant 3
You have [Valerie] for Mani's place. Congrats on the quarter. So yeah, can you talk about how the safe space for your data from [manager] and more informal strategy for [our labs]? And maybe also can you just comment on your insurance expectations for [this rebound]?
我是 [Valerie],代 Mani 發問。恭喜本季表現。所以,是的,你能談談來自 [manager] 的你們數據的「安全空間」以及較非正式的策略,針對[our labs] 嗎?另外也能否評論一下你們對於[this rebound] 的保險預期?
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
I don't think any of us caught that. Could you say that again? I think that the line is a bit muffled.
我想我們都沒有聽清楚。你可以再說一次嗎?我覺得線路有點悶、聽不太清楚。
Unidentified Participant 3
Unidentified Participant 3
Oh, sorry. No, yeah, I was asking if you could talk about the Phase 3 [Cilia] data from Biogen and how it's [owned from] your strategy for our MAPT. And you also talk about the internal expectations?
喔,抱歉。對,我是在問你能否談談 Biogen 的第三期 [Cilia] 數據,以及它如何被 [owned from] 你們針對我們 MAPT 的策略。另外你也談談內部的預期?
James Hamilton - Chief Medical Officer and Head of R&D
James Hamilton - Chief Medical Officer and Head of R&D
Sure, yeah, I can take that. This is James. So I'm assuming you're referring to the ASO party MAPT from Biogen and Ionis that's supposed to read out here in maybe this quarter or early next quarter.
當然,可以,我來回答。我是 James。所以我猜你指的是 Biogen 和 Ionis 的 ASO 項目 MAPT,預計會在本季或下季初讀出結果。
Yeah. I mean, I hope those data look good and I hope that they show an improvement in the cognitive rating scales. I think that would be broadly positive for Arrowhead and for the tau hypothesis in general. Of course, that study is in Alzheimer's disease, and a positive readout would support our Alzheimer's programs.
是的。我的意思是,我希望那些數據看起來不錯,也希望它們能顯示在認知評分量表上的改善。我認為這對 Arrowhead 以及整體的 tau 假說而言,將會是廣泛的正面訊號。當然,那項研究是在阿茲海默症中進行,而正向的讀出結果將支持我們的阿茲海默症研發計畫。
That being said, even if those data are not positive, I think we can still -- we still have the option of pursuing all the other tauopathies, right, because a knockdown approach of tau should improve any condition that's really driven by tau gain of function or tau pathology. So things like progressive supranuclear palsy or cortical basilar dementia or some of the real specific MAPT gain of function, frontotemporal dementia disorders.
話雖如此,即使那些數據不是正面的,我認為我們仍然——我們仍然有選項去推進所有其他的 tau 蛋白病(tauopathies),對吧?因為以降低 tau 的方式,應該能改善任何真正由 tau 功能獲得(gain of function)或 tau 病理所驅動的疾病。所以像是進行性核上性麻痺(PSP)或皮質基底核變性/失智(corticobasal dementia),或一些非常特定的 MAPT 功能獲得型前額顳葉失智症相關疾病。
Those are all fair game for us as we think about Phase 2s and Phase 3 down the road. So hopefully, the Biogen data are positive. If it's not the end of the world for our program, because we can still pursue tauopathies.
當我們思考未來的第 2 期與第 3 期試驗時,這些對我們而言都是可行的適應症。所以希望 Biogen 的數據是正面的。即使不是,對我們的計畫也不是世界末日,因為我們仍然可以追求 tau 蛋白病。
Operator
Operator
Joseph Thome, TD Cowen.
Joseph Thome,TD Cowen。
Joseph Thome - Analyst
Joseph Thome - Analyst
Just as we're thinking about the potential outcomes for the SHASTA-3/4 studies later this year, do you expect that you'll have to show differentiated efficacy versus what Ionis has shown in addition to the dosing benefit and already keep that even slightly premium pricing?
當我們在思考今年稍晚 SHASTA-3/4 研究的潛在結果時,您是否預期除了給藥上的優勢之外,還必須展現相較於 Ionis 所呈現的差異化療效,才能維持即使是略高的溢價定價?
And then just a point of clarification, are you characterizing the AP events in SHASTA-3/4 the same way that you characterize them in the long-term SHASTA-2 extension that was recently presented?
另外澄清一下,您對 SHASTA-3/4 中 AP 事件的界定方式,是否與近期發表的 SHASTA-2 長期延伸研究中對這些事件的界定方式相同?
James Hamilton - Chief Medical Officer and Head of R&D
James Hamilton - Chief Medical Officer and Head of R&D
Why don't I take the second part first about the characterization? The answer is no. So the SHASTA-2 study, we used the strict Atlanta criteria to look at pancreatitis events, whereas in the SHASTA-3/4 pooled analysis, we're using this modified Atlanta criteria that has definite probable and possible pancreatitis.
我先回答第二部分、也就是界定方式的問題。答案是否定的。在 SHASTA-2 研究中,我們使用嚴格的 Atlanta 標準來評估胰臟炎事件;而在 SHASTA-3/4 的合併分析中,我們使用的是修正版 Atlanta 標準,將胰臟炎分為確定、可能(probable)與疑似(possible)。
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
And regarding the premium pricing, let's just see what those data look like. Historically, when we look at data side by side, with the large caveat that, of course, it's difficult to compare different clinical studies with different patient populations. What we've seen consistently is superior triglyceride reduction, superior safety profile, and of course, superior convenience.
至於溢價定價,我們就先看看那些數據長什麼樣。歷來當我們把數據並列比較時——當然要強調一個很大的前提:不同臨床研究、不同病人族群之間很難直接比較。我們一貫看到的是更優異的三酸甘油脂降低幅度、更好的安全性概況,當然還有更高的便利性。
We expect those to continue. And should that be the case, then we would expect that a premium is still appropriate.
我們預期這些優勢會持續。如果確實如此,那麼我們會認為溢價仍然是合適的。
Operator
Operator
Patrick Trucchio, H.C. Wainwright.
Patrick Trucchio,H.C. Wainwright。
Patrick Trucchio - Analyst
Patrick Trucchio - Analyst
Just regarding your business development strategy, I was hoping you could give us some additional details just in terms of which assets you would be looking to bring forward on your own versus those that you may partner. And then just more broadly, how you're thinking about how RNAI kind of fits into the broader genetic medicines sort of portfolio and how RNAi is looking sort of competitively against modalities like gene editing, et cetera.
關於你們的商務拓展策略,我希望你們能提供更多細節,特別是哪些資產你們會傾向自行推進、哪些可能會選擇合作夥伴。更廣泛地說,你們如何看待 RNAi 在更大的基因藥物產品組合中的定位,以及 RNAi 相較於基因編輯等技術路徑的競爭態勢?
Andy Davis - Senior Vice President and Head of the Global Cardiometabolic Franchise
Andy Davis - Senior Vice President and Head of the Global Cardiometabolic Franchise
Sure. Look, I think that RNAi is at the forefront of genetic medicine for the foreseeable future. Now, look, it's not the right modality for everything, but for those diseases that are characterized by the overproduction of something, if we can address that cell type, then RNAi is a very attractive modality.
當然。我認為在可預見的未來,RNAi 會站在基因醫療的最前沿。當然,它不是適用於所有情況的最佳技術,但對於那些以某種物質過度生成為特徵的疾病,只要我們能鎖定到相關細胞類型,RNAi 就是一種非常有吸引力的技術路徑。
But when we think about RNAi versus gene editing, the way I feel is that RNAi is a relatively straightforward and conservative approach, right? It is a reversible approach. And in our hands, we can have very long durability. So you can almost get the best of both worlds where you have a low needle burden, if you will, but the ability to come off drugs should some new biology come out to suggest that you don't actually want to knock down that gene product.
但當我們比較 RNAi 與基因編輯時,我的看法是:RNAi 是一種相對直接且保守的方法,對吧?它是可逆的。而在我們的操作下,它可以具有非常長的持久性。所以你幾乎可以兼得兩者優點:一方面注射負擔(needle burden)較低;另一方面,如果未來出現新的生物學證據顯示其實不應該降低那個基因產物,你仍然可以停藥。
That's not the case in gene editing and I think that gene editing does have a place in the world of medicine. It's just quite unknown right now because I don't know what happens to these edited genes 10 years from now. I don't think anybody does, notwithstanding as the biology changes and it could be that sometime in the future, we decided that you might not want to enact a certain gene product.
基因編輯就不是這樣,而且我認為基因編輯在醫療領域確實有其位置。只是目前仍有很多未知,因為我不知道這些被編輯的基因在 10 年後會發生什麼事。我想沒有人知道;更何況隨著生物學理解的變化,未來也可能會出現我們認為你不應該啟動某個基因產物的情況。
So for those horrible diseases that are terminal, it could be worth taking a risk to do gene editing. But frankly, for all others, if you can address something with RNAi, that feels to me as a better approach.
因此,對於那些可怕且致命的疾病,冒險進行基因編輯可能是值得的。但坦白說,對於其他大多數情況,如果能用 RNAi 解決,對我而言那會是更好的方法。
Regarding physical development, this is a dynamic question. Right now, we feel pretty good about our existing pipeline in terms of that which is wholly owned right now. We like the idea of pushing all these forward ourselves, with the possible exception of C3. In fact, we like the programs a lot. Those drug candidates appear to do what they're intended to do and they seem to be well tolerated. Those are just really outside of our core focus at present. And so those are a couple of assets that we could partner with the right partner at some point.
至於(業務/產品)開發方面,這是一個動態的問題。目前就我們現有、且目前為全資持有的產品線而言,我們感覺相當不錯。我們喜歡由自己推進這些項目,可能的例外是 C3。事實上,我們非常喜歡這些計畫。這些候選藥物看起來能達到預期作用,而且耐受性良好。只是它們目前確實超出我們的核心聚焦範圍。因此,這些是我們未來在適當時點、與合適夥伴合作的幾項資產。
Everything else feels pretty good right now. What that may change as we talk to other companies and as our pipeline grows. But we don't feel a real sense of urgency to do additional deals on existing clinical programs other than maybe two. three effective.
其他方面目前都相當不錯。當我們與其他公司洽談、以及我們的產品線擴大時,這些想法可能會改變。但除了可能兩、三個項目之外,我們並不覺得在既有臨床計畫上需要很強的急迫性去做額外交易。
Operator
Operator
Amine Chaherli. B. Riley Securities.
Amine Chaherli。B. Riley Securities。
Amine Chaherli - Analyst
Amine Chaherli - Analyst
Congratulations on the quarter. This is Amine Chaherli on behalf of Madison El-Saadi. I just wanted to ask, as you think about the next generation of the DIMER platform, is there a construct combining INHBE or ALK7 with a non-overlapping mechanism target, something you're evaluating for obesity or other indications?
恭喜本季表現。我是 Amine Chaherli,代表 Madison El-Saadi 提問。我想請問,當你們思考 DIMER 平台的下一代時,是否正在評估將 INHBE 或 ALK7 與一個機制不重疊的靶點結合的構型,用於肥胖或其他適應症?
James Hamilton - Chief Medical Officer and Head of R&D
James Hamilton - Chief Medical Officer and Head of R&D
Yes.
是的。
Operator
Operator
Luca Issi, RBC Capital Markets.
Luca Issi,RBC Capital Markets。
Unidentified Participant 4
Unidentified Participant 4
This is Shelby on for Luca. Given Ionis has announced the new price at $40,000 and has first-mover advantage, can you walk us through the rationale to continue to price for REDEMPLO at a premium versus that par and SHTG, maybe as a way to kind of undercut their pricing since you're coming in second? Just wondering the rationale behind that.
我是 Shelby,代替 Luca 提問。鑑於 Ionis 已宣布新價格為 40,000 美元,且具備先行者優勢,你們能否說明為何仍打算讓 REDEMPLO 的定價高於該價格與 SHTG?或許作為一種在你們第二個進入市場時壓低對方價格的方式——想了解背後的理由。
Andy Davis - Senior Vice President and Head of the Global Cardiometabolic Franchise
Andy Davis - Senior Vice President and Head of the Global Cardiometabolic Franchise
Yeah, thanks, Shelby. As Chris previously mentioned, we do believe REDEMPLO is a best-in-class APOC3 inhibitor in that it commands premium pricing as a consequence of that. And there are a variety of reasons for that. Chris touched on some of them, including the depth of knockdown for APOC3 as a target, the depth of PG reduction.
好的,謝謝你,Shelby。如同 Chris 先前提到的,我們確實相信 REDEMPLO 是同類最佳(best-in-class)的 APOC3 抑制劑,因此也因為這一點而具備溢價定價的能力。背後有多項原因。Chris 提到其中一些,包括以 APOC3 作為靶點時的降低幅度(knockdown depth)、以及 PG 降低的幅度。
We saw that from Palisade with an 80% reduction from baselines. We saw that with the numerical decrease in acute pancreatitis from Palisade. We've seen that also with an FDA label that has no contraindications, no warnings, and no precautions. And then lastly, with a convenient dosing schedule that's only four injections a year. When you sum up the totality of those attributes, we just believe it's a best-in-class APOC3 inhibitor, and as a consequence, should be premium priced.
我們從Palisade看到了相較於基準值降低80%。我們也從Palisade看到急性胰臟炎的數值下降。我們也看到其FDA標籤沒有禁忌症、沒有警告、也沒有注意事項。最後,還有便利的給藥時程,一年只需注射四次。當你把這些特性綜合起來,我們相信它是同類最佳(best-in-class)的APOC3抑制劑,因此應該採取溢價定價。
Operator
Operator
Jen Jia, Cantor Fitzgerald.
Jen Jia,Cantor Fitzgerald。
Jennifer Jia - Analyst
Jennifer Jia - Analyst
This is Jennifer Jia on for Prakhar Agarwal. I'd like to understand a little bit more on the expectations for ARO-DIMER readout later this year. So what are you hoping to see, and what does it take to take this forward to a larger trial? And if it's positive, do you consider going straight to a cardio outcomes trial, or will you still need to compete at Phase 2?
我是Jennifer Jia,代替Prakhar Agarwal提問。我想更了解一下對今年稍晚ARO-DIMER數據讀出的預期。你們希望看到什麼結果?以及需要達到什麼條件才能推進到更大型的試驗?如果結果是正面的,你們會考慮直接進入心血管結局試驗,還是仍需要在第二期(Phase 2)競爭?
James Hamilton - Chief Medical Officer and Head of R&D
James Hamilton - Chief Medical Officer and Head of R&D
Yeah, Jennifer, this is James. Happy to address that question. So the great thing about this program is that all of the relevant biomarkers, and even biomarkers that you could use for -- potentially for approval, are blood-based, right? We can measure [ETST-9], we can measure [A+C-3] in the blood. Then we can measure LDL cholesterol and triglycerides, ApoB, non-HDL cholesterol, are all pretty easy for us to measure. So that's what we'll be primarily focused on, as well as safety in this initial data readout.
好的,Jennifer,我是James。很樂意回答這個問題。這個計畫很棒的一點是,所有相關的生物標記,甚至可能用於——潛在用於核准的生物標記,都是以血液為基礎的,對吧?我們可以在血液中測量[ETST-9],也可以測量[A+C-3]。接著我們也能測量LDL膽固醇與三酸甘油脂、ApoB、非HDL膽固醇,這些對我們來說都很容易測量。因此在這次初步數據讀出中,我們主要會聚焦於這些指標,以及安全性。
In terms of where we go from here, I think we're working on that now. There may be some additional limited Phase 2 work, which we could even consider doing as part of this study. But then moving quickly into an outcome study down the road. So more to come on the development plan and the study designs, but I'm looking forward to the readout later this year.
至於接下來要怎麼走,我想我們目前正在規劃。可能會有一些額外且有限的第二期(Phase 2)工作,我們甚至可以考慮把它納入這項研究的一部分來做。但之後會在未來儘快推進到結局研究(outcome study)。所以關於開發計畫與研究設計,後續會再提供更多資訊,但我很期待今年稍晚的數據讀出。
Operator
Operator
Keay Nakae, Chardan Capital Markets.
Keay Nakae,Chardan Capital Markets。
Keay Nakae - Analyst
Keay Nakae - Analyst
A question about the Madrigal licensing agreement for PNPLA. Help us understand, from a capital allocation strategy perspective, why it makes sense for you to do this deal at this time as opposed to taking the drug further on your own?
有一個關於與Madrigal就PNPLA的授權協議的問題。請協助我們理解,從資本配置策略的角度,為什麼你們選擇在此時做這筆交易,而不是自己把這個藥物繼續往前推進?
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Yes, good question. So let me just take you back and remind everyone that where PNPLA3 -- where Arrow PNPLA3 came from. We didn't develop this on our own independently. This was part of our deal with Janssen. It was centered around HPV, FTSP. But also included a couple of additional targets. This was one of those targets. And so we developed it for them. They did the Phase 1. The Phase 1 was compelling. After only a single dose, they saw about a 40% reduction in liver fat in homozygous patients. So that was interesting to us.
是的,好問題。我先帶大家回顧並提醒一下PNPLA3——Arrow的PNPLA3是怎麼來的。我們並不是自己獨立開發這個項目。這是我們與Janssen交易的一部分。該交易以HPV、FTSP為核心。但也包含了幾個額外的標的。這就是其中之一。因此我們為他們進行了開發。他們完成了第一期(Phase 1)。第一期結果很有說服力。僅在單次給藥後,他們就在同型合子患者中看到肝脂肪約降低40%。所以這點對我們而言很有意思。
Janssen, as I understand, decided to get out of MASH, and so the asset was returned to us. We didn't spend any money on this.
據我了解,Janssen決定退出MASH領域,因此該資產就回到我們手上。我們在這上面沒有花任何錢。
As we looked at taking this forward, we think it's a really compelling target for a company that's focused in MASH, largely. This is a genetically defined population, and that's sort of the good news and the bad news, right? The good news is it's quite specific. The challenge there is that there will be a companion diagnostic upon to this, and it made sense for us to find a pure-play MASH company to take this forward. And of course, [Amedical] is, I think, the best out there right now. It doesn't make sense.
當我們評估要把它往前推進時,我們認為對於一家主要聚焦MASH的公司而言,這是一個非常有吸引力的標的。這是一個由基因所界定的人群,這算是好消息也是壞消息,對吧?好消息是它相當特異。挑戰在於這需要搭配伴隨式診斷(companion diagnostic),因此我們認為找一家純MASH公司來推進會更合理。當然,[Amedical]我認為目前是最好的選擇。這樣做才合理。
It didn't really make sense for us to spend much money right now to do a Phase 2. Those studies could be a bit long and more expensive than made sense for us. We've got a very large pipeline, we've got some really interesting programs that we are pushing ourselves, and I just think that our ROI is probably better by allocating capital to those programs that we are more confident that we will hold on to long-term.
對我們而言,現在投入大量資金去做第二期(Phase 2)其實不太合理。這些研究可能時間較長、成本也更高,與我們的考量不太相符。我們有非常龐大的產品線(pipeline),也有一些非常有趣、由我們自己推進的項目;我認為把資本配置到那些我們更有信心能長期持有的項目上,投資報酬率(ROI)可能更好。
So that's where we that's where we went. No, we are we are thrilled to have Natalie as a partner. We're thrilled to have them develop that drug leads to good drugs. And we're thrilled to have them commercialized eventually. So we feel good about this. Yeah.
所以我們就是基於這樣的考量。我們也非常高興能有Natalie作為合作夥伴。我們很高興由他們來開發,進而帶來好的藥物。也很高興未來最終由他們來商業化。因此我們對這件事感到很踏實。是的。
Operator
Operator
Thank you. This concludes the question-and-answer session. I would now like to turn it back to Chris Anzalone for closing remarks.
謝謝。問答環節到此結束。接下來我想把時間交回給Chris Anzalone做結語。
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Christopher Anzalone - Chairman of the Board, President, Chief Executive Officer
Thanks, everyone, for joining us today. We look forward to seeing you in the future.
謝謝各位今天加入我們。期待未來再與各位相見。
Operator
Operator
Thank you for your participation in today's conference. This does conclude the program. You may now disconnect.
感謝各位參與今天的電話會議。本次議程到此結束。您現在可以掛線。