Secretome Therapeutics has announced the dosing of the first patient in its Phase 1 clinical trial (NCT06560762) evaluating STM-01, a novel allogeneic neonatal cardiac progenitor cell (nCPC)-derived therapy for heart failure with preserved ejection fraction (HFpEF). This milestone marks the first-ever trial assessing an allogeneic stem cell therapy product specifically for HFpEF patients.
The open-label trial utilizes an ascending dose design, administering either 100 million or 200 million nCPCs through a one-hour intravenous infusion. The first patient was treated at The University of Texas Southwestern Medical Center (UTSW), with Northwestern Medicine in Chicago serving as an additional study site.
"The launch of our first clinical trial is a major milestone for the STM-01 program, and for Secretome," said Vinny Jindal, President and Chief Executive Officer of Secretome Therapeutics. "Having recently secured multiple patents for STM-01, launched large-scale GMP manufacturing, completed our Seed Round of financing, and now launched this phase 1 study, we are well positioned to develop STM-01 as a potentially transformative therapeutic option in this multi-billion-dollar market."
FDA Fast Track Designation Highlights Unmet Need
Earlier this month, the FDA granted Fast Track designation to STM-01 for the treatment of HFpEF, underscoring the urgent need for innovative therapies in this area. The designation will facilitate expedited regulatory review and increased interactions with the FDA throughout the development process.
"The FDA's decision to grant Fast Track designation to STM-01 highlights the critical need for innovative therapies to address HFpEF, a condition with limited treatment options," Jindal noted in a separate statement.
The HFpEF Treatment Challenge
HFpEF represents over 50% of all heart failure diagnoses but remains difficult to treat effectively. Current management primarily focuses on symptom reduction, preventing disease progression, and managing underlying comorbidities through lifestyle modifications and limited pharmaceutical options.
Dr. Sanjiv Shah, the Stone Endowed Professor and Director of the HFpEF Program at Northwestern University Feinberg School of Medicine and principal investigator for the trial, emphasized the persistent challenges in treating HFpEF: "Despite recent advances that have improved outcomes in HFpEF, residual risk is still very high and many of these patients will continue to worsen. The next step in improving outcomes for patients is better and broader control of inflammation and restoration of normal cellular immunity."
Mechanism of Action and Preclinical Evidence
STM-01 is designed to function through multiple mechanisms: decreasing inflammation, supporting tissue repair, and inhibiting fibrosis. In preclinical studies, the therapy demonstrated significant reductions in inflammation at both cellular and molecular levels in HFpEF models, resulting in improved cardiac function and exercise tolerance.
The therapy utilizes cardiac progenitor cells (CPCs), which have shown promise in heart tissue repair due to their cardiac developmental origins. This approach potentially offers advantages over stem cells derived from other sources like adipose tissue or bone marrow.
Expanding Therapeutic Pipeline
Beyond HFpEF, Secretome is developing STM-01 for dilated cardiomyopathy and has another product, STM-21, in preclinical development for various inflammatory conditions including skin wounds and diabetes comorbidities.
The company aims to complete the Phase 1 study by October 2025, with the primary objective of assessing safety and tolerability in HFpEF patients.
Competitive Landscape
Secretome is not alone in pursuing advanced therapeutics for HFpEF. Medera subsidiary Sardocor is currently evaluating SRD-001, an adeno-associated virus vector-based gene therapy, in the phase 1/2a MUSIC-HFpEF clinical trial. SRD-001 also received FDA Fast Track designation in February 2024.
The Sardocor therapy takes a different approach, directly delivering treatment to cardiac ventricular muscle cells using a proprietary intracoronary infusion system designed to increase protein expression and functional activity of SERCA2a, a protein implicated in calcium-handling defects associated with HFpEF.
As these novel cell and gene therapies progress through clinical development, they represent potential paradigm shifts in treating a condition that has historically had few effective therapeutic options.
