RTW Biotech Opp. - Update on Rocket Pharmaceuticals
RNS Number : 0862J
RTW Biotech Opportunities Ltd
19 May 2025
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.
Enquiries:
| RTW Investments, LP - Investment Manager Woody Stileman (Business Development) Oliver Kenyon (Business & Corporate Development) Krisha McCune (Investor Relations) | +44 (0)20 7959 6361 biotechopportunities@rtwfunds.com |
| Cadarn Capital - PR & IR Partner Lucy Clark (PR) David Harris (Distribution) | +44 (0)7984 184 461 / lucy@cadarncapital.com +44 (0)7368 883 211 /david@cadarncapital.com |
| Deutsche Numis - Joint Corporate Broker Freddie Barnfield Nathan Brown Euan Brown | +44 (0)20 7260 1000 |
| BofA Securities - Joint Corporate Broker Edward Peel Alex Penney | +44 (0)20 7628 1000 |
| Altum (Guernsey) Limited Joanna Duquemin Nicolle Sadie Morrison | +44 (0)1481 703 100 |