FDA Grants Fast Track Designation to STM-01, Neonatal Cardiac Cell Therapy for Treatment of HFpEF

The FDA has granted a fast track designation (FTD) to STM-01, a neonatal cardiac progenitor cell (nCPC) therapy designed by Secretome Therapeutics for the treatment of patients with heart failure with preserved ejection fraction (HFpEF), according to a news release from Secretome. The investigational, allogeneic therapy is designed to improve cardiac function in patients with HFpEF and other inflammation-based cardiovascular conditions.¹

Heart failure with preserved ejection fraction can cause higher rates of mortality and morbidity. | Image Credit: © Rawpixel.com - stock.adobe.com

Results of preclinical trials conducted by Secretome have indicated STM-01’s potential to ease hallmarks of HFpEF through inhibiting fibrosis, supporting the repair of cardiac tissue, and reducing inflammation. Investigators are launching a phase 1 open-label, multiple ascending dose study (NCT06560762) to assess the safety and tolerability of STM-01 in patients with HFpEF, with a study completion goal of October 2025.^1,2

"The FDA’s decision to grant FTD to STM-01 highlights the critical need for innovative therapies to address HFpEF, a condition with limited treatment options," Vinny Jindal, president and CEO, Secretome Therapeutics, said in the news release. "This designation marks a pivotal regulatory milestone as we prepare to initiate our phase 1 clinical trial.”¹

Therapies utilizing cardiac progenitor cells (CPCs) to repair heart tissue in patients with cardiovascular diseases have become more thoroughly investigated in recent years. Encouraging clinical trial results have been observed in analyses of stem cell populations, including cells from developed organs and tissues, that have been successfully tested for heart repair. Using CPCs could be utilized as a more effective option for heart repair due to their cardiac developmental origins, making them a better candidate than stem cells from sources like adipose tissue or bone marrow.³

It is critical that new and innovative treatment options for patients with HFpEF are developed, as this form of heart failure comprises over 50% of all diagnoses of the condition. Due to the generality of the signs and symptoms of HFpEF, which are often like those of other heart failure classes, it can be difficult to accurately diagnose patients; however, patients with HFpEF have meaningfully different risk factors and pathophysiological processes, contributing to the heightened all-cause mortality, reduced quality of life, and major economic burden that this population faces.⁴

As previously mentioned, treatment options for heart failure—and HFpEF in particular—are sparse. Current management guidelines aim to reduce symptoms, prevent disease progression, manage underlying comorbidities, and ultimately improve quality of life. Correspondingly, guidelines center around behavioral and lifestyle changes, such as improving diet, increasing exercise, and restricting sodium. If a patient’s blood pressure remains uncontrolled in association with HFpEF, antihypertensive therapies such as diuretics or angiotensin receptor blockers are preferred.⁴

STM-01 represents a clear advancement in the treatment paradigm for patients with HFpEF, representing the potential of an entirely new class of medications to revolutionize the space. The FDA’s FTD will greatly aid STM-01 as it proceeds through the clinical development process, allowing for more expedited regulatory review and increased interactions with the FDA throughout the process.¹

REFERENCES

1. Secretome Therapeutic. Secretome Therapeutics granted FDA fast track designation for STM-01, a neonatal cardiac progenitor cell therapy for HFpEF. News Release. Released March 20, 2025. Accessed March 23, 2025. https://www.businesswire.com/news/home/20250320134011/en/Secretome-Therapeutics-Granted-FDA-Fast-Track-Designation-for-STM-01-a-Neonatal-Cardiac-Progenitor-Cell-Therapy-for-HFpEF

2. ClinicalTrials.gov. Study in participants with heart failure with preserved ejection fraction (HFpEF). National Library of Medicine. Last Updated August 19, 2024. Accessed March 23, 2025. https://clinicaltrials.gov/study/NCT06560762

3. Le TYL. Chong JJH. Cardiac progenitor cells for heart repair. Cell Death Discov. 2016;18(2):16052. doi:10.1038/cddiscovery.2016.52

4. Golla MSG, Shams P. Heart failure with preserved ejection fraction (HFpEF). Updated March 19, 2024. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-.https://www.ncbi.nlm.nih.gov/books/NBK599960/