Regulus Therapeutics报告了 2025年第一季度的财务结果和最近的更新-动脉网

Regulus Therapeutics Reports First Quarter 2025 Financial Results and Recent Updates

CISION 等信源发布 2025-05-09 04:05

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Entered into an agreement to be acquired by Novartis for $7.00 per share in cash, with potential to receive an additional $7.00 per share in cash through a contingent value right ('CVR') upon the achievement of a regulatory milestone, for a total equity value of up to approximately

达成协议，诺华将以每股7.00美元的现金价格收购，若达到监管里程碑，还可通过或有价值权（“CVR”）额外获得每股7.00美元的现金，总股权价值最高约为

$1.7 billion

17亿美元

; Expected to be completed in the second half of 2025, subject to customary closing conditions

预计将在2025年下半年完成，但需符合惯例的交割条件。

Positive topline data from the completed fourth cohort of patients in the Phase

第四组患者在该阶段完成后的积极数据

multiple-ascending dose (MAD) study of farabursen (RGLS8429) for the treatment of autosomal dominant polycystic kidney disease (ADPKD)

多次递增剂量（MAD）研究：法拉布森（RGLS8429）用于治疗常染色体显性多囊肾病（ADPKD）

Company on track for initiation of Phase 3 single pivotal trial in the third quarter 2025

公司有望在2025年第三季度启动第三阶段单一关键试验

SAN DIEGO

圣地亚哥

，

May 8, 2025

2025年5月8日

/PRNewswire/ --

Regulus Therapeutics Inc

Regulus治疗公司

。

(Nasdaq:

（纳斯达克：

RGLS

), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the 'Company' or 'Regulus'), today reported financial results for the first quarter ended

），一家专注于发现和开发针对微小RNA的创新药物的生物制药公司（“公司”或“Regulus”），今天公布了截至第一季度的财务结果。

March 31, 2025

2025年3月31日

, and provided a corporate update.

，并提供了公司更新。

'We recently announced that we have entered into an agreement to be acquired by Novartis, whose established global development and commercial capabilities will potentially bring farabursen to patients with ADPKD, who currently have limited treatment options,' said Jay Hagan, CEO of Regulus Therapeutics.

“我们最近宣布，已经达成协议将被诺华收购，诺华成熟的全球开发和商业能力有望将法拉布森带给ADPKD患者，这些患者目前的治疗选择有限，”Regulus Therapeutics首席执行官Jay Hagan表示。

'We've made important progress with farabursen so far this year, having announced positive results from the completed fourth cohort of patients in March, reporting evidence of a mechanistic dose response based on urinary PC1 and PC2 levels, and results suggesting that kidney volume growth rate was halted after only a relatively short treatment period.

“到目前为止，我们今年在法拉伯森方面取得了重要的进展。三月份，我们宣布了第四组患者完成治疗后的积极结果，报告了基于尿液PC1和PC2水平的机制剂量反应证据，结果表明仅经过相对较短的治疗期，肾脏体积的增长率就停止了。

We look forward to investigating further as we head towards initiation of the pivotal Phase 3 trial in the third quarter of this year.'.

我们期待在第三季度启动关键的第三阶段试验时进一步调查。

Program Updates

程序更新

Farabursen for ADPKD:

法拉布森用于ADPKD：

在

March 2025

2025年3月

, the Company shared positive topline results from all patients in the fourth cohort of the Phase

，公司分享了第四批第一阶段所有患者的积极概况结果。

MAD study of farabursen for the treatment of ADPKD. In the fourth cohort, 26 subjects received a fixed dose of 300 mg of farabursen every other week for three months. Consistent with the previously announced interim analysis of efficacy data from the first 14 subjects of this fixed-dose cohort in

MAD研究法拉布森治疗ADPKD。在第四组中，26名受试者每隔一周接受300毫克的固定剂量法拉布森，持续三个月。与此前宣布的该固定剂量组前14名受试者的中期疗效数据分析一致。

January 2025

2025年1月

, in the full cohort of 26 patients we observed similar mechanistic response based on urinary polycystins 1 and 2 (PC1 and PC2) levels as well as a mean halting of height-adjusted total kidney volume (htTKV) growth over the four-month study period. These results were in line with data from the third cohort, dosed at 3 mg/kg, which is predicted to achieve optimal kidney exposure.

，在全部26名患者的队列中，我们观察到基于尿多囊蛋白1和2（PC1和PC2）水平的类似机制反应，并且在四个月的研究期间，身高调整后的总肾体积（htTKV）增长平均停止。这些结果与第三组（剂量为3 mg/kg）的数据一致，该剂量预计可达到最佳肾脏暴露。

Final efficacy data confirmed the appropriateness of a 300 mg fixed dose for the Phase 3 pivotal trial. Based on complete safety data from all 26 subjects we observed that farabursen dosed at 300 mg had a favorable safety and tolerability profile in this study, consistent with all earlier cohorts..

最终疗效数据确认了在 III 期关键试验中使用 300 mg 固定剂量的适当性。基于全部 26 名受试者的完整安全数据，我们观察到法拉贝森在 300 mg 剂量下在此研究中表现出良好的安全性和耐受性特征，与所有早期队列一致。

The Phase

阶段

MAD study was a double-blind, placebo-controlled trial which evaluated the safety, tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of farabursen in adult patients with ADPKD. The study evaluated farabursen treatment for three months followed by one month of follow up across three weight-based dose levels (1, 2 and 3 mg/kg) and one fixed dose level (300 mg).

MAD研究是一项双盲、安慰剂对照试验，评估了法拉布森（farabursen）在成人ADPKD患者中的安全性、耐受性、药代动力学和药效学（PK/PD）。该研究评估了法拉布森治疗三个月，并在三个基于体重的剂量水平（1、2和3 mg/kg）以及一个固定剂量水平（300 mg）下进行了一个月的随访。

Primary endpoints were changes in urinary PC1 and PC2, with an exploratory examination of change in htTKV growth rate. PC1 and PC2 are the protein products of the PKD1 and PKD2 genes and their levels have been shown to inversely correlate with disease severity; htTKV has been shown to inversely correlate with kidney function..

主要终点是尿液中PC1和PC2的变化，并对htTKV增长率的变化进行探索性研究。PC1和PC2是PKD1和PKD2基因的蛋白质产物，其水平已被证明与疾病严重程度呈负相关；htTKV已被证明与肾功能呈负相关。

A single, pivotal Phase 3 study is planned to be initiated in the third quarter of this year. In January, the Company announced that it held a meeting with the U.S. Food and Drug Administration (FDA) and had confirmed alignment on the acceptability of the program's CMC, non-clinical and clinical pharmacology plans and key elements of the trial design, including a 12-month htTKV endpoint planned to support Accelerated Approval and a 24-month eGFR endpoint to support Full Approval..

计划在今年第三季度启动一项关键的III期单一研究。今年一月，该公司宣布与美国食品药品监督管理局（FDA）举行了一次会议，并已确认在项目的关键方面达成一致，包括CMC、非临床和临床药理学计划的可接受性，以及试验设计的关键要素，其中包括计划支持加速批准的12个月htTKV终点和用于支持完全批准的24个月eGFR终点。

Corporate Highlights

公司亮点

开

April 30, 2025

2025年4月30日

, the Company announced that it entered into an agreement and plan of merger with Novartis (the 'Merger Agreement').

，公司宣布已与诺华达成合并协议和计划（“合并协议”）。

Pursuant to the Merger Agreement, Novartis, through an indirectly wholly owned subsidiary, will acquire Regulus for an initial payment of

根据合并协议，诺华将通过一家间接全资子公司以初始付款收购Regulus公司。

$7.00

7.00美元

per share of common stock in cash at closing, plus one CVR per share of common stock, representing the right to receive one contingent payment of

每股普通股在收盘时以现金支付，另加一股CVR，代表获得一笔或有付款的权利。

$7.00

7.00美元

in cash, contingent upon the achievement of a milestone with respect to regulatory approval of Regulus' lead product candidate, farabursen. The upfront consideration is

以现金支付，前提是实现与 Regulus 首要候选产品 farabursen 的监管批准相关的里程碑。预付款为

$0.8 billion

8亿美元

and total potential consideration including the CVR, if the milestone is achieved, would be approximately

总潜在对价包括CVR在内，如果达到里程碑，约为

$1.7 billion

17亿美元

. The transaction has been unanimously approved by the Boards of Directors of both companies.

该交易已获得两家公司董事会的一致批准。

Completion of the transaction is expected in the second half of 2025, subject to the satisfaction or waiver of customary closing conditions, including the tender of shares representing at least a majority of the total number of Regulus' outstanding shares of common stock and the receipt of required regulatory approvals.

预计交易将在2025年下半年完成，但须满足或豁免惯常的交割条件，包括至少代表Regulus多数已发行普通股的股票要约，以及获得所需的监管批准。

Until that time, Regulus will continue to operate as a separate and independent company..

在那个时候之前，Regulus将继续作为一家独立的公司运营。

Financial Results

财务结果

Cash, Cash Equivalents and Short-Term Investments:

现金、现金等价物和短期投资：

As of

截至

March 31, 2025

2025年3月31日

, Regulus had

，雷古勒斯有

$65.4 million

6540万美元

in cash, cash equivalents and short-term investments. The Company expects its cash runway to extend into early 2026.

现金、现金等价物和短期投资。公司预计其现金跑道将延长至2026年初。

Research and Development (R&D) Expenses:

研发费用：

Research and development expenses were

研发费用为

$6.8 million

680万美元

for the three months ended

截至三个月末

March 31, 2025

2025年3月31日

, compared to

，与...相比

$6.0 million

600万美元

for the same period in 2024. These amounts reflect internal and external costs associated with advancing our clinical and preclinical pipeline.

在2024年同期，这些金额反映了与推进我们的临床和临床前管线相关的内部和外部成本。

General and Administrative (G&A) Expenses:

一般及行政（G&A）费用：

General and administrative expenses were $3.7 million for the three months ended March 31, 2025, compared to $2.8 million for the same period in 2024. These amounts reflect personnel-related and ongoing general business operating costs.

截至2025年3月31日的三个月，一般及行政费用为370万美元，而2024年同期为280万美元。这些金额反映了与人员相关的成本以及持续的一般业务运营费用。

Net Loss:

净亏损：

Net loss was

净亏损为

$9.6 million

960万美元

, or

，或者

$0.15

0.15美元

per share (basic and diluted), for the three months ended

每股（基本和稀释），截至三个月末

March 31, 2025

2025年3月31日

, compared to

，与...相比

$8.5 million

850万美元

, or

，或者

$0.29

0.29美元

per share (basic and diluted), for the same period in 2024.

每股（基本和稀释），在2024年同期。

About ADPKD

关于ADPKD

Autosomal dominant polycystic kidney disease (ADPKD), caused by mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs.

常染色体显性多囊肾病 (ADPKD) 由 PKD1 或 PKD2 基因突变引起，是人类最常见的单基因遗传病之一，也是终末期肾病的主要原因之一。该疾病的特点是主要在肾脏中形成多个充满液体的囊肿，其次在肝脏和其他器官中也会出现囊肿。

Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60. Approximately 160,000 individuals are diagnosed with the disease in .

肾囊肿细胞过度增殖是一个主要的病理特征，约50%的ADPKD患者在60岁左右会因此最终发展为终末期肾病。大约有16万人被诊断出患有这种疾病。

the United States

美国

alone, with an estimated global prevalence of 4 to 7 million.

单独存在，全球患病率估计为400万至700万。

About Farabursen (RGLS8429)

关于Farabursen (RGLS8429)

Farabursen is a novel, next generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to preferentially target the kidney. Administration of farabursen has shown clear improvements in kidney function, size, and other measures of disease severity in preclinical models. Regulus announced the completion of the Phase 1 SAD study in September 2022.

Farabursen 是一种新型的下一代寡核苷酸，用于治疗常染色体显性多囊肾病 (ADPKD)，旨在抑制 miR-17 并优先靶向肾脏。在临床前模型中，Farabursen 的施用显示出肾功能、大小和其他疾病严重程度指标的显著改善。Regulus 于 2022 年 9 月宣布完成了 1 期单次递增剂量 (SAD) 研究。

The Phase 1 SAD study demonstrated that farabursen has a favorable safety and PK profile. Farabursen was observed to be well-tolerated with no serious adverse events reported and plasma exposure was approximately linear across the four doses tested. In the now completed Phase 1b MAD study, Regulus announced topline data from the first cohort of patients in September 2023, from the second cohort of patients in March 2024, from the third cohort of patients in June 2024, and from the fourth cohort in March 2025.

第一阶段的单剂量递增（SAD）研究显示，farabursen具有良好的安全性和药代动力学（PK）特性。观察到farabursen耐受性良好，无严重不良事件报告，且在测试的四个剂量下血浆暴露量近似呈线性。在现已完成的第一阶段多剂量递增（MAD）研究中，Regulus于2023年9月公布了首组患者的主要数据，于2024年3月公布了第二组患者的数据，于2024年6月公布了第三组患者的数据，并将于2025年3月公布第四组患者的数据。

Based on complete safety data from all cohorts, farabursen was observed to be well tolerated, and observed results showed anticipated biomarker response (PC1 & PC2) and impact on kidney volume that potentially derisks Phase 3 trial. Initiation of the Phase 3 trial is planned for the third quarter of 2025..

基于所有队列的完整安全数据，法拉布森被观察到耐受性良好，观察结果显示预期的生物标志物反应（PC1 和 PC2）以及对肾体积的影响，这可能降低第三阶段试验的风险。第三阶段试验计划于2025年第三季度启动。

About Regulus

关于Regulus

Regulus Therapeutics Inc. (Nasdaq:

Regulus Therapeutics Inc.（纳斯达克：

RGLS

) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. Regulus maintains its corporate headquarters in .

）是一家专注于发现和开发针对微RNA的创新药物的生物制药公司。Regulus利用其寡核苷酸药物发现和开发的专业知识，建立了一个拥有丰富知识产权的microRNA领域产品线。Regulus的公司总部设在。

San Diego, CA.

加利福尼亚州圣迭戈。

Important Information About the Tender Offer

关于要约收购的重要信息

The tender offer described above has not yet commenced. This communication is not an offer to buy nor a solicitation of an offer to sell any securities of the Company. The solicitation and the offer to buy shares of the Company common stock will only be made pursuant to a tender offer statement on Schedule TO, including an offer to purchase, a letter of transmittal and other related materials that Novartis and Redwood Merger Sub Inc.

上述要约收购尚未开始。本通讯并非购买公司任何证券的要约，亦非出售公司任何证券的要约邀请。购买公司普通股的要约和要约邀请将仅根据Novartis和Redwood Merger Sub Inc.编制的附表TO中的要约收购声明作出，包括购买要约、转送函及其他相关材料。

('Merger Sub') intend to file with the Securities and Exchange Commission (the 'SEC'). In addition, the Company will file with the SEC a solicitation/recommendation statement on Schedule 14D-9 with respect to the tender offer. Once filed, investors will be able to obtain a free copy of these materials and other documents filed by Novartis, Merger Sub and the Company with the SEC at the website maintained by the SEC at .

('Merger Sub') 打算向美国证券交易委员会（'SEC'）提交文件。此外，公司还将就要约收购向SEC提交一份Schedule 14D-9的征求/建议声明。一旦提交，投资者将能够在美国证券交易委员会维护的网站上免费获取这些材料及诺华、Merger Sub和公司向SEC提交的其他文件。

www.sec.gov

or by directing a request for such materials and other documents to the information agent for the offer, which will be named in the tender offer statement. Investors may also obtain, at no charge, any such documents filed with or furnished to the SEC by the Company under the 'Investors' section of the Company's website at .

或通过向要约的信息代理提出请求来获取此类材料和其他文件，该信息代理将在要约声明中指定。投资者还可以免费在公司网站的“投资者”部分获取公司向SEC提交或提供的任何此类文件。

https://ir.regulusrx.com/

. Further risks and uncertainties that could cause actual results to differ materially from the results anticipated by the forward-looking statements are detailed from time to time in the Company's periodic reports filed with the SEC, including the Company's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K.

可能导致实际结果与前瞻性陈述中预期的结果有重大差异的更多风险和不确定性，会不时在公司向美国证券交易委员会（SEC）提交的定期报告中详细说明，包括公司最近的年度报告（Form 10-K）、季度报告（Form 10-Q）以及当前报告（Form 8-K）。

These filings, when available, are available on the investor relations section of the Company's website mentioned above or on the SEC's website at .

这些文件（如果可用）可以在上述公司网站的投资者关系部分或美国证券交易委员会 (SEC) 的网站上查阅。

https://www.sec.gov

。

INVESTORS AND SECURITY HOLDERS ARE ADVISED TO READ THESE DOCUMENTS WHEN THEY BECOME AVAILABLE, INCLUDING THE TENDER OFFER MATERIALS (INCLUDING AN OFFER TO PURCHASE, A LETTER OF TRANSMITTAL AND RELATED DOCUMENTS) AND ANY AMENDMENTS THERETO, THE SOLICITATION/RECOMMENDATION STATEMENT ON SCHEDULE 14D-9 OF THE COMPANY AND ANY AMENDMENTS THERETO, AS WELL AS ANY OTHER DOCUMENTS RELATING TO THE TENDER OFFER AND THE MERGER THAT ARE FILED WITH THE SEC, CAREFULLY AND IN THEIR ENTIRETY PRIOR TO MAKING ANY DECISIONS WITH RESPECT TO WHETHER TO TENDER THEIR SHARES INTO THE TENDER OFFER BECAUSE THEY CONTAIN IMPORTANT INFORMATION, INCLUDING THE TERMS AND CONDITIONS OF THE TENDER OFFER..

投资者和证券持有人被建议在这些文件可用时仔细阅读它们，包括要约收购材料（包括购买要约、转交函及相关文件）及其任何修正案、公司按照14D-9表提交的征求/建议声明及其任何修正案，以及与要约收购和合并相关的任何其他向美国证券交易委员会提交的文件，并在决定是否将他们的股份投入要约收购之前，完整阅读这些文件，因为它们包含重要信息，包括要约收购的条款和条件。

Forward-Looking Statements

前瞻性声明

The statements included in this press release that are not a description of historical facts are forward-looking statements. Words or phrases such as such as 'believe,' 'may,' 'could,' 'will,' 'estimate,' 'continue,' 'anticipate,' 'intend,' 'seek,' 'plan,' 'expect,' 'should,' 'would' or similar expressions are intended to identify forward-looking statements.

本新闻稿中包含的非历史事实描述的陈述为前瞻性陈述。诸如“相信”、“可能”、“能够”、“将”、“估计”、“继续”、“预期”、“打算”、“寻求”、“计划”、“预计”、“应该”、“会”等词语或短语旨在识别前瞻性陈述。

The forward-looking statements are based on the Company's current beliefs and expectations and include, but are not limited to: statements regarding beliefs about the potential benefits of the transaction; the planned completion and timing of the transactions contemplated by the Merger Agreement or the CVR agreement governing the terms of the CVR; the prospective performance and outlook of the business, performance, and opportunities of the Company continuing as the surviving corporation; the Company's farabursen (RGLS8429) program; the potential that farabursen may be eligible for an Accelerated Approval pathway; predictions based on and future results and clinical outcomes suggested by the fourth cohort data; our Phase 3 trial design including whether the selected fixed dose will be efficacious or whether the FDA will ultimately determine its components and the overall design to be acceptable and sufficient to serve as a single pivotal study; planned data announcements; and the timing and future occurrence of other preclinical and clinical activities.

前瞻性陈述基于公司当前的信念和预期，包括但不限于：关于交易潜在好处的陈述；合并协议或规定CVR条款的CVR协议中设想的交易计划完成及其时间安排；作为存续公司的公司业务、表现及前景展望与机会；公司的farabursen（RGLS8429）项目；farabursen可能符合加速批准通道的潜力；基于第四组数据预测的未来结果和临床结果；我们第三阶段试验设计，包括选定的固定剂量是否有效，或者FDA最终是否会认定其组成部分及整体设计可接受且足以作为单一关键研究；计划的数据发布；以及其他临床前和临床活动的时间安排和未来发展。

Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Risks and uncertainties that could cause results to differ from expectations include: uncertainties as to the timing and completion of the tender offer and the merger; uncertainties as to the percen.

由于这些声明受到风险和不确定性的约束，实际结果可能与这些前瞻性声明所表达或暗示的结果有重大差异。可能导致结果与预期不符的风险和不确定性包括：要约收购和合并的时间和完成情况的不确定性；百分比的不确定性。

www.sec.gov

. All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

本新闻稿中包含的所有前瞻性陈述仅在其发布之日有效。Regulus 没有义务更新这些陈述以反映在其发布之日后发生的事件或存在的状况。

You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and the Company undertakes no obligation to revise or update these statements to reflect events or circumstances after the date hereof, except as required by law..

您被提醒不要过分依赖这些前瞻性陈述，这些陈述仅截至本文件发布之日有效。所有前瞻性陈述均受此警示性声明的全面约束，公司不承担修订或更新这些陈述以反映本文件发布日期之后发生的事件或情况的义务，除非法律要求。

Regulus Therapeutics Inc.

Regulus 治疗公司

Selected Financial Information

选定的财务信息

Condensed Statement of Operations

损益表简表

(In thousands, except share and per share data)

（单位：千，股份和每股数据除外）

Three months ended

三个月结束

March 31,