Rtw Biotech Regulatory News (RTW)

LEI: 549300Q7EXQQH6KF7Z84

19 May 2025

RTW Biotech Opportunities Ltd

Update on Rocket Pharmaceuticals

RTW Biotech Opportunities Ltd (the "Company"), the London Stock Exchange-listed investment company focused on identifying transformative assets with high growth potential across the life sciences sector, is pleased to note the 15 May announcement by one of its public portfolio companies, Rocket Pharmaceuticals ("Rocket", NASDAQ:RCKT), of preliminary data from its Phase 1 clinical trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) at the annual meeting of the American Society of Gene and Cell Therapy. PKP2-ACM is a rare disease that affects ~50,000 people in the US and EU alone.

Rocket is a late-stage biotech company advancing a pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. RP-A601 is being investigated as a one-time, potentially curative gene therapy treatment that may improve survival and quality of life for affected patients. RP-A601 showed a well-tolerated safety profile with no dose-limiting toxicities, increased PKP2 protein expression, and preliminary indications of improvement or stabilisation in arrhythmia burden, heart function, and quality of life. It is the second drug in Rocket's pipeline to directly target a large genetic cardiac disease.

Rocket is an original company creation of RTW Investments and was founded in 2015, going public through a reverse merger in 2018. As at 30 April, Rocket represented 3.2% of the Company's NAV. Rocket's share price closed up 11.1% on 15 May, the day of announcement.

Rod Wong, CIO of RTW Investments, said, "We are excited to see this encouraging preliminary data from Rocket's RP-A601's Phase 1 clinical trial, which we flagged as a key catalyst in the Company's annual results on 31 March 2025, demonstrating clear progress on Rocket's aim to deliver potentially curative treatments to patients with rare and devastating heart conditions. We look forward to updating the Company's shareholders on this and other exciting future developments at Rocket, with the next proximate catalyst being results of patient dosing for treatment of Danon disease, expected this quarter."

The full text of the announcement can be read at  https://www.rocketpharma.com.

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About RTW Biotech Opportunities Ltd:

RTW Biotech Opportunities Ltd (LSE: RTW) is an investment fund focused on identifying transformative assets with high growth potential across the biopharmaceutical and medical technology sectors. Driven by a long-term approach to support innovative businesses, RTW Biotech Opportunities Ltd invests in companies developing next-generation therapies and technologies that can significantly improve patients' lives. RTW Biotech Opportunities Ltd is managed by RTW Investments, LP, a leading healthcare-focused entrepreneurial investment firm with deep scientific expertise and a strong track record of supporting companies developing life-changing therapies.

Visit the website at www.rtwfunds.com/rtw-biotech-opportunities-ltd for more information.

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The information in this announcement may include forward-looking statements, which are based on the current expectations and projections about future events, and in certain cases can be identified by the use of terms such as "may", "will", "should", "expect", "anticipate", "project", "estimate", "intend", "continue", "target", "believe" (or the negatives thereon) or other variations thereon or comparable terminology. These forward-looking statements, as well as those included in any related materials, are subject to risks, uncertainties and assumptions about the Company and/or its underlying investments, including, among other things, the development of the applicable entity's business, trends in its operating industry, expected use of financing proceeds and future capital expenditures and acquisitions. In light of these risks, uncertainties and assumptions, the events in the forward-looking statements may not occur.

The information contained in this announcement is given at the date of its publication (unless otherwise marked). No reliance may be placed for any purpose whatsoever on the information or opinions contained in this announcement or on its completeness, accuracy or fairness.

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