LEI: 549300Q7EXQQH6KF7Z84

30 May 2025

RTW Biotech Opportunities Ltd

Portfolio Updates

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, notes recent announcements from three public portfolio companies.

Taysha Gene Therapies

On 28 May, Taysha Gene Therapies ("Taysha", NASDAQ:TSHA) released positive clinical data from its phase 1/2 trials evaluating TSHA-102 in Rett syndrome, with all patients across varying disease severity gaining or regaining one or more developmental milestones. Taysha concluded that the likelihood of achieving such milestones was improbable after age six, making the data particularly compelling. In addition and following written alignment from the U.S. Food and Drug Administration ("FDA"), Taysha published details of its planned pivotal Part B trial design for TSHA-102.

Taysha is a clinical-stage biotechnology company focused on advancing gene therapies for the treatment of diseases affecting the central nervous system. TSHA-102, its lead clinical programme, is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers.

As at 30 April, Taysha represented 0.4% of the Company's NAV. This position was subsequently added to. Taysha's share price closed up 12.7% on 29 May. Taysha's announcement can be found here.

Rocket Pharmaceuticals

On 27 May, Rocket Pharmaceuticals ("Rocket", NASDAQ:RCKT) announced that a patient participating in its phase 2 pivotal trial of RP-A501 to treat Danon disease experienced an unexpected Serious Adverse Event ("SAE") and subsequently died after an acute systemic infection. The FDA placed a clinical hold on the trial to allow for further evaluation. Rocket is in active dialogue with the FDA to confirm the root cause of the SAE. The Company remains optimistic that there is a path forward for RP-A501.

Given these events, Rocket announced that it would reduce overall cash spend and focus resources on its adeno-associated viral ("AAV") pipeline. Rocket has several other candidates in ongoing clinical trials, including for PKP2-ACM, a type of heart disease, for which positive phase 1 data were released earlier this month. As at 31 March, Rocket had $318 million of cash and expects existing resources to fund operations into 2027.

As at 30 April, Rocket represented 3.2% of the Company's NAV. Rocket's share price closed down 62.8% on 27 May, the day of announcement. Rocket's announcement can be found here.

Merus

On 22 May, Merus ("Merus", NASDAQ:MRUS), an oncology company developing innovative antibody treatments for cancer, announced compelling interim phase 2 data from its trial treating a type of head and neck cancer. While current treatments utilise a single antibody (pembrolizumab), the Merus trial combines this with petosemtamab, which appears to deliver significantly improved results. This underscores the opportunity petosemtamab holds to become a new standard of care, if approved, in head and neck cancer.

As at 30 April, Merus represented 1.0% of the Company's NAV. Merus' share price closed up 32.5% on 23 May, the day after announcement. Merus' announcement can be found here.

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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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