Sarepta Therapeutics Reports Preliminary* Fourth Quarter and Full-Year 2024 Net Product Revenue
Sarepta Therapeutics Reports Preliminary* Fourth Quarter and Full-Year 2024 Net Product Revenue
– Preliminary total net product revenue of $638.2 million for the fourth quarter and $1.79 billion for full-year 2024, exceeding full-year guidance by over $100 million
– prelimary總淨產品營業收入爲63820萬美元,第四季度爲17.9億美元,超過全年指導超過10000萬美元
– Preliminary ELEVIDYS net product revenue totaled $384.2 million for the fourth quarter, exceeding guidance by over $60 million, and $820.8 million for full-year 2024
– prelimary ELEVIDYS淨產品營業收入總計爲38420萬美元,第四季度超過指導超過6000萬美元,2024年全年爲82080萬美元
– Preliminary RNA-based PMO net product revenue for the fourth quarter and full-year of 2024 totaled $254.0 million and $967.2 million, respectively
– prelimary基於RNA的PMO淨產品營業收入爲第四季度和2024年全年分別爲25400萬美元和96720萬美元
– Preliminary year-end 2024 cash, cash equivalents, restricted cash and investments balance of approximately $1.5 billion
– prelimary 2024年年末現金、現金等價物、受限現金和投資餘額約爲15億美元
– Reiterates 2025 full-year total net product revenue guidance of $2.9 to $3.1 billion
– 重申2025年全年總淨產品營業收入指導爲29億至31億美元
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 13, 2025--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported preliminary* fourth quarter and full-year 2024 net product revenue and cash on hand as of December 31, 2024, as part of its presentation today at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, Calif.
劍橋,馬薩諸塞州.--(商業新聞)--2025年1月13日--Sarepta Therapeutics公司(納斯達克:SRPT),作爲稀有疾病精準基因醫學的領導者,今天報告了截至2024年12月31日的初步*第四季度和全年淨產品營業收入及現金餘額,作爲其在加利福尼亞州舊金山舉行的第43屆年度摩根大通醫療大會上的報告的一部分。
Financial Update* (preliminary and unaudited):
財務更新*(初步和未經審計):
- Total net product revenue of $638.2 million for the fourth quarter and $1.79 billion for full-year 2024, exceeding full-year guidance by over $100 million. Sarepta's net product revenue does not include collaboration, contract manufacturing or royalty revenue.
- Fourth quarter and full-year 2024 net product revenue for ELEVIDYS totaled $384.2 million and $820.8 million, respectively. Sarepta's net product revenue does not include collaboration, contract manufacturing or royalty revenue.
- Fourth quarter and full-year 2024 net product revenue for Sarepta's RNA-based PMOs totaled $254.0 million and $967.2 million, respectively. Sarepta's net product revenue does not include collaboration, contract manufacturing or royalty revenue.
- As of December 31, 2024, the Company had preliminary cash, cash equivalents, restricted cash and investments of approximately $1.5 billion.
- The Company reiterates 2025 full-year total net product revenue guidance of $2.9 to $3.1 billion.
- 2024年第四季度淨產品營業收入總額爲63820萬美元,全年爲17.9億超過了全年預期超出10000萬美元以上。Sarepta的淨產品營業收入不包括合作、合同製造或特許權使用費收入。
- 2024年第四季度及全年ELEVIDYS淨產品收入分別爲38420萬美元和82080萬美元。Sarepta的淨產品收入不包括合作、合同製造或特許使用權收入。
- 2024年第四季度及全年Sarepta的基於RNA的PMOs淨產品收入分別爲25400萬美元和96720萬美元。Sarepta的淨產品收入不包括合作、合同製造或特許使用權收入。
- 截至2024年12月31日,公司擁有初步現金、現金等價物、受限現金和投資約15億美元。
- 公司重申2025年全年淨產品收入指導爲29億到31億美元。
"2024 marked the most significant year to date for Sarepta and for the patients we serve. And consistent with our long track record of execution, we ended 2024 and enter 2025 with exceptionally strong performance," said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. "In the fourth quarter, we grew total net product revenue by 75% year-over-year and grew ELEVIDYS by a very robust 112% over the prior sequential quarter, overachieving our guidance by more than $60 million. Our 2024 total net product revenue grew some 56% over 2023. And we were pleased to see that even in the face of a strong ELEVIDYS launch, our PMO franchise continued to perform and grow year-over-year."
「2024年是Sarepta及我們所服務患者迄今爲止最重要的一年。與我們長期以來一貫的執行力一致,我們在2024年結束並進入2025年時表現非常強勁,」Sarepta Therapeutics的總裁兼首席執行官Doug Ingram表示。「在第四季度,我們的總淨產品收入同比增長75%,ELEVIDYS的收入相比於上一個季度強勁增長112%,超出我們的指導金額超過6000萬美元。我們2024年的總淨產品收入較2023年增長約56%。我們很高興看到,即使在ELEVIDYS強勁上市的情況下,我們的PMO業務仍然繼續表現並實現同比增長。」
*These preliminary selected financial results are unaudited and subject to adjustment. Sarepta will report its final and complete fourth quarter and full-year 2024 financial results in late February 2025. The Company has not completed its financial closing procedures for the quarter or year-ended December 31, 2024, and its actual results could be materially different from these preliminary financial results.
*這些初步選擇的基本報表未經審計,可能會有所調整。Sarepta將在2025年2月底報告其最終和完整的2024年第四季度及全年財務結果。公司尚未完成截止2024年12月31日的季度或年終財務關閉程序,其實際結果可能與這些初步財務結果有重大不同。
About ELEVIDYS (delandistrogene moxeparvovec-rokl)
關於ELEVIDYS(delandistrogene moxeparvovec-rokl)
ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
ELEVIDYS(delandistrogene moxeparvovec-rokl)是一種單劑量的腺相關病毒(AAV)基因轉移療法,通過靜脈注射設計用於解決杜氏肌營養不良症的根本遺傳原因——DMD基因中的突變或變化,導致缺乏肌營養不良蛋白——通過傳遞編碼ELEVIDYS微肌營養不良蛋白在骨骼肌中靶向生產的轉基因。
ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.
ELEVIDYS適用於治療至少4歲患有杜氏肌營養不良症(DMD)的人士。
- For patients who are ambulatory and have a confirmed mutation in the DMD gene
- For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.
- 適用於已確診DMD基因突變且能夠行走的患者
- 適用於已確診DMD基因突變且無法行走的患者。
The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as "micro-dystrophin") in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
在非行走患者中,DMD的適應症已根據骨骼肌中ELEVIDYS微型肌營養不良蛋白(以下簡稱爲「微型肌營養不良蛋白」)的表達獲得加速批准。對該適應症的持續批准可能取決於在確認試驗中驗證和描述臨牀獲益。
IMPORTANT SAFETY INFORMATION
重要安全信息
CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.
禁忌症:對具有DMD基因第8外顯子和/或第9外顯子任何缺失的患者禁用ELEVIDYS。
WARNINGS AND PRECAUTIONS:
警告和預防措施:
Infusion-related Reactions:
輸注相關反應:
- Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate.
- ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available.
- Discontinue infusion for anaphylaxis.
- 輸注相關反應,包括過敏反應和過敏性休克,已在ELEVIDYS給藥過程中或給藥後數小時內發生。給藥期間和輸注結束後至少監測病人3小時。如果出現輸注相關反應的症狀,請減緩或停止輸注並給予適當的治療。一旦症狀緩解,可以以較低的速度重新開始輸注。
- ELEVIDYS應在可以立即提供輸注相關反應治療的環境中給藥。
- 發生過敏性休克時停止輸注。
Acute Serious Liver Injury:
急性嚴重肝損傷:
- Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks.
- Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.
- Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels).
- Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended.
- 已觀察到ELEVIDYS引起急性嚴重肝損傷,給藥可能導致肝酶(如GGt、GLDH、ALt、AST)或總膽紅素水平升高,通常在8周內可見。
- 具有既往肝功能受損、慢性肝病或急性肝病(例如,急性肝病毒感染)的患者,可能更容易發生急性嚴重肝損傷。在急性肝病患者中,應推遲ELEVIDYS給藥,直至病情緩解或控制。
- 在ELEVIDYS給藥前,進行肝酶檢測,並在ELEVIDYS輸注後的前3個月內每週監測肝功能(臨牀檢查、GGt和總膽紅素)。如果臨牀指示,繼續監測,直到結果正常(臨牀檢查正常,GGt和總膽紅素水平接近基線水平)。
- 建議在ELEVIDYS輸注前後對患者進行系統性皮質類固醇治療。在有需要時,調整皮質類固醇方案。如果懷疑急性嚴重肝損傷,建議諮詢專家。
Immune-mediated Myositis:
免疫介導性肌炎:
- In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed.
- Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction.
- Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur.
- 在臨牀試驗中,免疫介導性肌炎大約在ELEVIDYS輸注後1個月被觀察到,患者的DMD基因存在外顯子8和/或外顯子9的缺失突變。觀察到了嚴重肌肉無力的症狀,包括吞嚥困難、呼吸急促和言語低沉。
- 有限的數據可用於治療DMD基因外顯子1至17和/或外顯子59至71突變患者的ELEVIDYS治療。這些區域存在缺失的患者可能面臨嚴重的免疫介導性肌炎反應的風險。
- 建議患者如果出現任何無法解釋的肌肉疼痛、壓痛或無力,包括吞嚥困難、呼吸急促或言語低沉,立即聯繫醫生,因爲這些可能是肌炎的症狀。如果出現這些症狀,建議根據患者的臨牀表現和病史考慮額外的免疫調節治療(免疫抑制劑[如鈣調神經磷酸酶抑制劑]與皮質類固醇聯合使用)。
Myocarditis:
心肌炎:
- Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials.
- If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath.
- Advise patients to contact a physician immediately if they experience cardiac symptoms.
- 在臨牀試驗中,ELEVIDYS輸注後觀察到急性嚴重心肌炎和肌鈣蛋白I升高。
- 如果患者出現心肌炎,那些有既往左心室射血分數(LVEF)損害的患者可能面臨更高的不良結果風險。在ELEVIDYS輸注之前監測肌鈣蛋白I,並在輸注後的第一個月每週監測,如果臨牀指示則繼續監測。如存在胸痛或呼吸急促等心臟症狀,可能需要更頻繁的監測。
- 建議患者如果出現心臟症狀,立即聯繫醫生。
Preexisting Immunity against AAVrh74:
對AAVrh74的既存免疫力:
- In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies.
- Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration.
- ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400.
- 在基於AAV載體的基因治療中,既存的抗AAV抗體可能阻礙轉基因在預期治療水平上的表達。在接受ELEVIDYS治療後,所有患者均產生了抗AAVrh74抗體。
- 在ELEVIDYS給藥之前進行抗AAVrh74總結合抗體存在的基線檢測。
- 不建議在抗AAVrh74總結合抗體滴度≥1:400的患者中使用ELEVIDYS。
Adverse Reactions:
不良反應:
- The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.
- 在臨牀研究中報告的最常見不良反應(發生率≥5%)包括嘔吐、噁心、肝損傷、發熱和血小板減少。
Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).
向FDA報告處方藥的負面副作用。訪問或撥打1-800-FDA-1088。您也可以向Sarepta Therapeutics報告副作用,電話是1-888-SAREPTA(1-888-727-3782)。
For further information, please see the full Prescribing Information.
如需進一步信息,請參見完整的處方信息。
About Sarepta Therapeutics
關於Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook.
Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook.
Internet Posting of Information
信息的Internet Plus-related發佈
We routinely post information that may be important to investors in the 'For Investors' section of our website at . We encourage investors and potential investors to consult our website regularly for important information about us.
我們在網站的「投資者專欄」定期發佈可能對投資者重要的信息。我們鼓勵投資者和潛在投資者定期訪問我們的網站,以獲取關於我們的重要信息。
Forward-Looking Statements
前瞻性聲明
This press release contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believe," "anticipate," "plan," "expect," "will," "may," "intend," "prepare," "look," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our expected financial results.
This press release contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believe," "anticipate," "plan," "expect," "will," "may," "intend," "prepare," "look," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our expected financial results.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: the estimates and judgments we make, or the assumptions on which we rely, in preparing our consolidated financial statements could prove inaccurate; our revenues and operating results could fluctuate significantly, which may adversely affect our stock price; and those risks identified under the heading "Risk Factors" in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 as well as other SEC filings made by the Company which you are encouraged to review.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: the estimates and judgments we make, or the assumptions on which we rely, in preparing our consolidated financial statements could prove inaccurate; our revenues and operating results could fluctuate significantly, which may adversely affect our stock price; and those risks identified under the heading "Risk Factors" in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 as well as other SEC filings made by the Company which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.
上述任何風險都可能對公司的業務、運營結果以及Sarepta普通股的交易價格產生重大不利影響。有關Sarepta面臨的風險和不確定性的詳細描述,建議您查看Sarepta提交給SEC的文件。我們提醒投資者不要過度依賴本新聞稿中包含的前瞻性聲明。Sarepta不承擔任何義務在此日期之後根據事件或情況公開更新其前瞻性聲明,法律要求的除了。
View source version on businesswire.com:
在businesswire.com查看源版本:
Investor Contact:
Ian Estepan, 617-274-4052
iestepan@sarepta.com
Media Contact:
Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com
投資者聯繫人:
伊恩·埃斯特潘,617-274-4052
iestepan@sarepta.com
媒體聯繫人:
特雷西·索倫蒂諾,617-301-8566
tsorrentino@sarepta.com
Source: Sarepta Therapeutics, Inc.
來源:Sarepta Therapeutics, Inc.
譯文內容由第三人軟體翻譯。
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