ASX、納斯達克和媒體公告

2024年10月31日

季度活動報告

Q1 FY25

亮點

opthea有限公司 (ASX: OPt, 納斯達克: OPT)（“opthea”或“該公司”）今天發布了截至2024年9月30日的為期三個月的季度業務報告和附錄4C（“FY25第1季”）。

opthea是一家臨床階段的生物製藥公司，致力於開發新型治療方案，用於治療高流行率和逐漸惡化的視網膜疾病，包括濕性年齡相關性黃斑部病變（濕性AMD）和糖尿病黃斑水腫（DME）。

opthea的主要產品候選藥sozinibercept正在參與兩項完全招募的重要第三期臨床試驗（COASt和ShORe），以配合常規治療抗VEGF-A療法，以提高整體功效並提供比單獨使用常規治療抗VEGF-A藥劑更優越的視力增益。

Sozinibercept有潛力成為20年內第一種能夠在與任何抗VEGF-A治療結合使用時提供優越視力收益的新治療方法。

菲德·吉拉德博士，opthea有限公司首席執行官評論說：“我們在將opthea定位為成功方面繼續取得進展。我們最近的高管任命，包括Tom Reilly擔任臨時財務長，Parisa Zamiri博士擔任臨時醫療長，Mike Campbell擔任臨時商業長，這些任命顯著加強了我們的領導團隊，預期將於我們第3階段結缐數據公佈前。藥品成分PPQ活性配方的成功完成是該項目降低風險並在濕性AMD中提交潛在生物製品許可申請（BLA）的重要一步。opthea被納入S&P/ASX 300指数有助於進一步使我們機構股東基礎多元化，同時公司在第24屆EURETINA大會上的科學存在強調了我們致力於將sozinibercept推向濕性AMD患者，幫助他們實現更好的視力，以在生活中獲得更大的獨立性。”

關於Sozinibercept

Sozinibercept是一種新型、首創的VEGF-C/D「陷阱」抑制劑，旨在與標準護理的抗VEGF-A治療結合使用，以改善患濕性黃斑部病變的視力，在其中有許多對現有治療反應不佳或已變為耐受性。已知VEGF-C和VEGF-D能獨立地刺激視網膜血管生成、血管滲漏和通透性，而VEGF-A的抑制也可能導致VEGF-C和VEGF-D的上調。研究顯示，透過sozinibercept針對性地抑制VEGF-C和VEGF-D能夠防止血管生長和血管滲漏，這兩者皆對包括濕性黃斑部病變在內的視網膜疾病的病理生理學有所貢獻。Sozinibercept有潛力成為20年來首個改善患有濕性黃斑部病變患者視覺結果的療法，使他們能夠更獨立地生活並擁有更好的生活品質。

關於opthea的臨床開發計劃

公司目前正進行兩項完全招募滿、關鍵性3期多中心、雙盲、隨機臨床試驗，分別是 COASt（OPt-302與Aflibercept聯合研究）和ShORe（OPt-302與Ranibizumab聯合研究），旨在評估所利尼布聯合治療相較於標準護理抗VEGF-A療法在治療濕性黃斑部病變中的安全性和卓越療效。Opthea的3期臨床試驗計畫旨在支持廣泛的標籤，並且若成功，所利尼布有潛力被批准與任何抗VEGF-A聯合使用治療濕性黃斑部病變患者。所利尼布已獲得美國食品和藥物管理局對治療濕性黃斑部病變的快速通道設計認可。欲瞭解更多有關Opthea的3期臨床試驗計畫，請造訪ClinicalTrials.gov查詢COASt。 NCT04757636，以及ShORe， NCT04757610.

在opthea的前瞻性、隨機分組和對照第20億臨床試驗中，包括366名首次接受治療的濕性年齡相關性黃斑變性患者，sozinibercept與標準護理ranibizumab組合使用治療濕性年齡相關性黃斑變性。sozinibercept組合療法達到預先確定的主要療效終點，在24週時視力收益在統計上優於僅使用ranibizumab。此外，次要結果在組合療法中也是積極的，包括更多患者視力增加10個或以上字母、解剖學改善，減輕腫脹和血管滲漏，以及良好的安全性簡况。這些統計意義上的結果已於2023年2月發表。 眼科醫療 。於2023年2月在眼科醫療上發表。

關於濕性年齡相關性黃斑變性

濕性年齡相關性黃斑變性（AMD）仍然是老年人視力喪失的主要原因，在美國和歐洲影響約350萬人。濕性AMD在醫療上的未滿足需求非常重要，許多患者儘管接受抗VEGF-A治療，卻無法達到最佳視力結果，或隨著時間流逝失去視力。

領導團隊

opthea任命湯姆·賴利為財務長，任職日期為2024年10月28日。賴利先生在美國及全球生命科學公司建立及領導財務和行政團隊方面擁有超過25年的豐富經驗。在加入opthea之前，賴利先生曾擔任Amarin公司的財務長兼人力資源主管，支持商業

擴大公司在心血管疾病方面的主要資產。Reilly先生還擔任過Cara Therapeutics的財務長和愛力健一般藥品業務的財務主管，支持神經科學領域的多項商業推出。Reilly先生在諾華納（Novartis）度過了14年，擔任不斷增加責任的角色，包括腫瘤學發展部門的財務主管，諾華納製藥奧地利的財務長，以及諾華納製藥美國的財務控制器。他獲得了曼哈頓學院財務學學士學位，塞頓霍爾大學的工商管理碩士學位，並且是一名註冊會計師。Reilly先生接替了擔任致富金融（臨時代碼）長的丹尼爾·蓋夫肯（Daniel Geffken）。

Dr. Parisa Zamiri 於 2024 年 10 月 7 日起被任命為首席醫療官。她負責臨床開發與運營、監管和醫療事務、以及生物量測。Zamiri 博士是一位具有深厚藥物發現和開發專業知識的醫師科學家。她接受過眼科醫療培訓，擁有醫學視網膜、免疫學和發炎治療方面的臨床經驗。她最近曾擔任 Complement Therapeutics 的首席醫療官，之前則在 Graybug Vision 擔任該職。在此之前，她曾擔任 Novartis Pharmaceuticals 眼科醫療全球臨床開發主管和眼科醫療治療領域主管副總裁，領導一群臨床科學家和眼科醫生設計和執行各類新生物製品、基因治療、小分子藥物和數位治療，針對濕性老年黃斑變性、地理性萎縮、視網膜色素變性和乾眼等眼科疾病進行第 1 至第 4 階段臨床試驗。Zamiri 博士於倫敦大學國王學院醫學院取得醫學學位，並在與倫敦摩菲爾德眼科醫院有關的北泰晤士輪轉醫院完成了眼科醫療住院醫師培訓。她在麻薩諸塞州眼與耳科研究所的 Schepens 眼科研究所進行有關視網膜下膜隱私的免疫學研究，並取得了眼科免疫學博士學位，該研究是在哈佛醫學院附屬機構進行的。

Mike Campbell has been appointed Chief Commercial Officer. Effective September 9, 2024. Mr. Campbell brings 30 years of biotechnology and pharmaceutical commercial leadership experience across sales, marketing, market access, patient services, and operations to the Company. Mr. Campbell dedicated most of his career to launching and commercializing innovative treatments for retinal and ocular surface diseases. During his tenure at Genentech, he was a key leader for the commercial build and launch of Lucentis®, the first anti-VEGF-A treatment approved for wet AMD; he served in commercial leadership roles through the lifecycle of Lucentis®, including the expansion into Diabetic Macula Edema, and Retinal Vein Occlusion. Mr. Campbell also contributed to the prelaunch commercial planning of Beovu® in wet AMD at Novartis, as well as the $3.4 billion divestiture of Xiidra® for Dry Eye Disease to Novartis, during his tenure at Shire. Mr. Campbell most recently served as Senior Vice President and Head of Commercial at Viatris Eye Care who acquired Oyster Point Pharma, where he led the commercial build for the launch of Tyrvaya® in Dry Eye Disease. Mr. Campbell holds a Bachelor of Science degree from Auburn University and is an Executive Education graduate from The Wharton School, University of Pennsylvania.

Opthea is strategically and methodically building the company to position it for success. In addition to expanding its leadership team, Opthea continues to add experienced, talented, and dedicated members to support its mission. At the end of September 2024, Opthea employed 41 team members.

First Quarter Financial Performance & Cash Flow

Opthea’s cash balance at 30 September 2024 was US$167.5m, down from US$172.5m in the prior quarter ending 30 June 2024, primarily reflecting the receipt of the Retail Entitlement finalised in the middle of July 2024 netted against the operational spend for the quarter of US$39.8m to advance the two pivotal clinical trials towards top-line data readout.

Cash receipts for the quarter were US$2.2m, which is up 175% from the US$0.8m in the previous quarter. This is a consequence of the varying interest on cash holdings after the finalisation of the June/July 2024 capital raise. The net operating cash outflow for the period was US$41.2m. The prior period net operating cash outflow was US$38.6m.

Research and development cash costs for the quarter were US$36.8m, 14% above the previous quarter (Q4 FY24: US$32.2m). Administration cash costs in Q1 FY25 were US$2.3m and were down 38% from previous quarter (Q4 FY24: US$3.7m). Personnel costs of US$4.3m were up 26% on the previous quarter of US$3.4m. These were in line with expectations with the recent restructure and addition of staff in the quarter.

Use of Funds from Any Future Capital Raise

We intend to use the net proceeds from any current or future offering or capital raise to advance clinical development, chemistry, manufacturing, and controls, regulatory, and potential commercial activities of sozinibercept for wet AMD, and for general corporate purposes.

In accordance with ASX Listing Rule 4.7C.3, cash paid for Directors and Non-Executive Directors in Q1 FY25 amounted to US$290k in aggregate which includes director fees and customary reimbursement of applicable costs, including costs for traveling to Opthea meetings.

Authorized for release by CEO Fred Guerard, PharmD, CEO.

Enquiries

About Opthea

Opthea (ASX:OPT; NASDAQ:OPT) is a biopharmaceutical company developing novel therapies to address the unmet need in the treatment of highly prevalent and progressive retinal diseases, including wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME).

Opthea’s lead product candidate, sozinibercept, is being evaluated in two fully enrolled pivotal Phase 3 clinical trials (COAST, NCT04757636, and ShORe, NCT04757610) for use in combination with standard-of-care anti-VEGF-A therapies to improve overall efficacy and deliver superior vision gains compared to the standard-of-care anti-VEGF-A agents alone.

To learn more, visit our websiteat www.opthea.com and follow us on Xand LinkedIn.

Risk Factors

Investing in our securities involves a high degree of risk. You should consider and read carefully all of the factors, including potential uncertainties described below, as well as the Risk Factors included in our 20-F filing for the fiscal year ending June 30, 2024 as filed with the Securities and Exchange Commission on August 30, 2024, including our condensed consolidated financial statements and related notes included elsewhere in our Half-Year Report for the fiscal period ended December 31, 2023. If any of the risks and uncertainties described under Risk Factors included in our 20-F for the fiscal year ended June 30, 2024, or the following uncertainties occur, it could harm our business, prospects, results of operations and financial condition. In such event, the trading price of the ordinary shares and the ADSs could decline, and you might lose all or part of your investment. You should not interpret our disclosure of any of the risks and uncertainties described under Risk Factors included in our 20-F for the fiscal year ended June 30, 2024, or the following uncertainties to imply that such risks have not already materialized.

Development Funding Agreement, Financial Resources and Timing of Completion of Clinical Trials

The Company had US$167.5 million in cash at September 30, 2024. Opthea believes that it will be able to fund its operating and research and development expenses into the third calendar quarter of 2025, which is through the anticipated Phase 3 topline data readouts for COAST (Combination OPT-302 with Aflibercept Study), and ShORe (Study of OPT-302 in combination with Ranibizumab). Opthea may raise additional external funding, including through equity financing, prior to or after its reporting of the top-line data. The amount and timing of Opthea’s expenditures will depend upon and have been impacted in the past, and may continue to be impacted by, numerous factors, including historical or future delays in completing our clinical trials, particularly as it relates to the timing of regulatory submissions, the performance and cost efficiency of contract research organizations (“CROs”) and contract manufacturing organisations, and the continuing impacts of the global supply chain and macroeconomic challenges. In particular, delays in patient enrolment have resulted, and may in the future result in increased costs or delays and other impacts on the timing of our Phase 3 clinical trials. Opthea has based this estimate on assumptions that may prove to be wrong, and Opthea could exhaust its available capital resources sooner than it expects. Opthea may also experience future delays in its clinical development or commercialization of sozinibercept for wet AMD, including due to factors and conditions set forth above or other factors that Opthea cannot presently anticipate.

Opthea intends to focus its development efforts on achieving commercialization of sozinibercept for the treatment of wet AMD and will require additional funding to reach commercialization of sozinibercept in any indication, including wet AMD. In addition, Opthea will require additional external funding to meet the minimum cash condition under the Development Funding Agreement (‘DFA’), including prior to the readout of top-line results for Phase 3 clinical trials for OPT-302. If Opthea experiences further delays in its Phase 3 clinical trials, Opthea may need to raise additional external funding, including potentially dilutive equity financing.

Opthea does not have any other committed external source of funds and expects to finance future cash needs through public or private equity or debt offerings or collaborations. However, the DFA limits the type of financing Opthea may pursue in the future and Opthea may be unable to raise additional funds or enter into such other agreements or arrangements when needed on favorable terms, or at all.

If Opthea raises additional capital, this may cause dilution to holders of the Company’s ordinary shares and American Depositary Shares.

Forward-Looking Statements

Certain statements made during or in connection with this announcement contain or comprise certain forward-looking statements, including within the meaning of the U.S. Private Securities Litigation Reform Act of 1995. The words “expect”, “believe,” “should”, “could”, “may”, “will”, “plan” and other similar expressions are intended to identify forward-looking statements. Forward-looking statements in this ASX announcement include statements regarding the anticipated sozinibercept topline data timing for the two Phase 3 pivotal trials in wet AMD, and the Company’s continued efforts to advance its BLA preparations for FDA approval and prepare for commercial readiness. Forward-looking statements, opinions and estimates provided in this ASX announcement are based on assumptions and contingencies which are subject to change without notice, as are statements about market and industry trends, which are based on interpretations of current conditions. Forward-looking statements are provided as a general guide only and should not be relied upon as an indication or guarantee of future performance. They involve known and unknown risks and uncertainties and other factors, many of which are beyond the control of Opthea and its directors and management and may involve significant elements of subjective judgment and assumptions as to future events that may or may not be correct. These statements may be affected by a range of variables which could cause actual results or trends to differ materially, including but not limited to future capital requirements, the development, testing, production, marketing and sale of drug treatments, regulatory risk and potential loss of regulatory approvals, ongoing clinical studies to demonstrate sozinibercept safety, tolerability and therapeutic efficacy, clinical research organization and labor costs, intellectual property protections, and other factors that are of a general nature which may affect the future operating and financial performance of the Company including risk factors set forth in Opthea’s Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (the “SEC”) on August 30, 2024 and other future filings with the SEC. Actual results, performance or achievement may vary materially from any projections and forward-looking statements and the assumptions on which those statements are based. Subject to any continuing obligations under applicable law or any relevant ASX listing rules, Opthea disclaims any obligation or undertaking to provide any updates or revisions to any forward-looking statements in this ASX announcement to reflect any change in expectations in relation to any forward-looking statements or any change in events, conditions or circumstances on which any such statement is based, except as otherwise required by applicable law

Appendix 4C

Quarterly cash flow reportfor entities subject to Listing Rule 4.7B

Compliance statement

Date: October 31, 2024

Authorised by: Frederic Guerard CEO

(Name of body or officerauthorising release – see note 4)

Notes