Medera Biopharm, (“Medera”), a clinical-stage biotechnology company, announced today that its clinical stage gene therapy subsidiary Sardocor Corp provided interim data from the ongoing Phase 1/2a clinical trial of the gene therapy candidate SRD-001 in patients with heart failure with reduced ejection fraction (HErEF) at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), held May 7-11 in Baltimore, MD.

The oral presentation, presented by Professor Christian Kupatt, professor of Cardiology at the Technical University of Munich, featured interim data from the Phase 1 portion of the ongoing Phase 1/2a trial evaluating the safety and efficacy of SRD-001 in patients with HFrEF (NCT04703842). The Phase 1 portion is an open-label, uncontrolled study investigating two doses of SRD-001 at 3E13 (six participants) and at 4.5E13 (three participants) viral genomes (vg) per patient at each dose level.

As of the cut-off date (April 11, 2024), six patients have received a dose of 3E13 vg of SRD-001 through intracoronary infusion and one patient had received a dose of 4.5E13 vg of SRD-001. The interim data included:

• There have been no gene therapy or procedure‒related adverse events in the first 6-12 months post infusion. 

• Three patients have shown improvements in NYHA from Class 3 prior to treatment to Class 2 at 6 and 12 months. Clinically meaningful improvements in LVEF (+4% to +11%), 6MWT (+55 to +86m), decrease in Pro-BNP (-49 pg/ml) and decreases in troponin I (-10.6 to - 1.7 pg/mL) from baseline to 12 months post-treatment.

• The early clinical efficacy at a dose of 3E13vg/patient of AAV1.SERCA2a will be followed by enrollment of two more patients at a higher dose at 4.5E13 vg/patient as one has already been treated at this dose.

“These encouraging results of SRD-001 in patients with HFrEF may offer alternative treatments to patients with severe heart failure where a large unmet need remains,” said Roger Hajjar, MD, President, Chief Medical Officer and co-Founder of Medera.

The oral presentation details were as follows:

Abstract title: “A Phase 1 Clinical Trial of High Dose AAV1.SERCA2a in Patients with Heart Failure: Modulation of SERCA2a of Intra-myocytic Calcium trafficking in Heart Failure with Reduced Ejection Fraction” (MUSIC-HFrEF)

Abstract number: 5

Session Title: Late-Breaking Abstracts I

Presentation time: Thursday, May 9 from 9:00 AM - 9:15 AM

About SRD-001

SRD-001 is an investigational gene therapy candidate that contains an adeno-associated virus serotype 1 (AAV1) vector expressing the transgene for sarco(endo)plasmic reticulum Ca2+ ATPase 2a isoform (SERCA2a), in anti-AAV1 neutralizing antibody (NAb) negative subjects with ischemic or non-ischemic cardiomyopathy and New York Heart Association (NYHA) class III/IV symptoms of heart failure with reduced ejection fraction (HFrEF).

About Heart Failure with reduced Ejection Fraction (HFrEF)

HErEF is a complex cardiovascular pathophysiological syndrome that impairs normal cardiac function and results in the inability of the heart to pump a sufficient supply of blood to meet the body demand. Once established, HFrEF is generally progressive, irreversible, associated with debilitating symptoms, frequent re-hospitalizations and high mortality rates. An urgent need exists for disease modifying therapies, such as SRD-001, which aim to reverse the pathophysiology of HFrEF. 

About Medera

Medera is a clinical-stage biotechnology company dedicated to bioengineered human mini-heart-based drug discovery and development of next-generation gene and cell therapies for difficult-to-treat and incurable diseases across cardiac, vascular and muscular indications. Medera operates via its wholly-owned subsidiaries: Novoheart and Sardocor.

Novoheart capitalizes on the world’s first and award-winning “mini-Heart” Technology Platform for revolutionary disease modelling and drug discovery, enabling us to uniquely model human-specific diseases and discover therapeutic candidates free from species-specific differences. Before privatization by Medera, Novoheart was dually listed on the Toronto Stock Exchange and Frankfurt Stock Exchange. 

Sardocor (www.sardocor.com) aspires to create the shortest regulatory path to clinic for advancing next-generation cell and gene therapies. Building upon Novoheart’s bioengineered human tissue-based assays for disease modelling and drug discovery, Sardocor has developed a gene- and cell-based therapeutic pipeline for cardiovascular indications including heart failure with preserved ejection fraction (HFpEF), HFrEF and Duchenne muscular dystrophy-induced cardiomyopathy (DMD-CM) which are currently in clinical trials.