AskBio presents GenePHIT Phase 2 baseline data at ESC 2026
AskBio has presented baseline participant characteristics from GenePHIT, its Phase 2 trial of umiposgene parvec (AB-1002) in non-ischemic heart failure with reduced ejection fraction (HFrEF), at the European Society of Cardiology Congress in Munich. The readout confirms that enrolment is complete, with more than 170 participants randomised across 64 sites in 12 countries, making it one of the largest randomised gene therapy trials conducted in heart failure to date.
Umiposgene parvec is a single-administration AAV-based gene therapy delivered directly to the heart via intracoronary infusion. It encodes a modified form of protein phosphatase inhibitor-1 (I-1c), designed to restore intracellular calcium signalling and improve cardiac contractility. Notably, the protocol requires no immune suppression, which differentiates it from some earlier gene therapy approaches and may ease the benefit-risk calculation for cardiologists and regulators alike.
Trial population and design
The enrolled cohort reflects a high-burden patient group: nearly half had a history of atrial fibrillation and approximately 45% were already carrying an implantable cardioverter defibrillator. All participants were receiving guideline-directed medical therapy at baseline, including beta-blockers, angiotensin receptor-neprilysin inhibitors, SGLT2 inhibitors, and mineralocorticoid receptor antagonists. That optimised background therapy raises the bar for demonstrating incremental benefit, but it also means any positive signal would be clinically meaningful in a real-world context.
Timothy Henry, principal investigator, said the enrolled population "closely reflects contemporary patients receiving current standard of care, providing an excellent foundation to evaluate whether a one-time investigational gene therapy can meaningfully improve outcomes." AskBio's chief medical officer, Canwen Jiang, added that the baseline characteristics "establish a strong foundation for interpreting the initial efficacy and safety outcomes."
Initial efficacy and safety results from GenePHIT are expected in the first half of 2027. Primary endpoints include cardiovascular-related deaths and changes from baseline in NYHA functional classification, left ventricular ejection fraction, and six-minute walking distance.
Market context and competitive landscape
Heart failure is one of the most active frontiers in cardiovascular gene therapy, driven in part by the large and growing patient population, estimated at more than 64 million people globally. Several programmes are pursuing AAV-mediated delivery of sarco-endoplasmic reticulum Ca2+ ATPase (SERCA2a) as a target, an approach that has seen mixed results in previous trials. AskBio's PP1-inhibitor mechanism represents a distinct molecular rationale, though the field has seen enough promising preclinical data fail to translate in Phase 3 that investors rightly treat any headline enrolment milestone with caution.
AskBio is wholly owned by Bayer, whose cardiovascular franchise gives the programme a well-resourced commercialisation pathway if late-stage results are positive. Bayer reported R&D spending of 5.8 billion euros in fiscal 2025, underscoring its capacity to support a multi-phase development programme. For context, competing cardiovascular gene therapy efforts from academic and biotech spinouts are typically at earlier stages, meaning AskBio is among the few entities with a large, placebo-controlled Phase 2 dataset in this indication approaching readout.
The 2027 data release will be closely watched by cardiologists, payers, and rival developers. If the efficacy signal is robust, it could accelerate regulatory discussions on both sides of the Atlantic and establish gene therapy as a credible option alongside device-based and pharmacological approaches in advanced HFrEF.