19th May 2025 14:20
(Alliance News) - RTW Biotech Opportunities Ltd on Monday noted investee Rocket Pharmaceuticals has reported "encouraging" data from a phase 1 clinical trial of its treatment for arrhythmogenic cardiomyopathy.
RTW Biotech is a Guernsey, England-based investment fund which invests in companies that develop next-generation therapies and technologies that can improve patients' lives.
Rocket Pharmaceuticals is a US-based late-stage biotech firm advancing a pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. The group was an original company creation of RTW Investments, which was founded in 2015 and made public through a reverse merger in 2018.
Rocket represented 3.2% of RTW Biotech's net asset value at April 30.
PKP2 arrhythmogenic cardiomyopathy is a rare disease affecting around 50,000 people across the US and the EU. Rocket's RP-A601 is being investigated as a one-time, potentially curative gene therapy treatment to improve survival and quality of life for affected patients.
In its phase 1 clinical trial, RP-A601 demonstrated a well-tolerated safety profile with no dose-limiting toxicities, increased PKP2 protein expression, as well as 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.
"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," said Rod Wong, chief investment officer at RTW Investments.
"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."
Shares in RTW Biotech were up 1.3% at USD1.18 each in London on Monday afternoon. The stock remains down 16% over the past year.
By Emily Parsons, Alliance News reporter
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