Rare Daily Staff

Clinical stage gene therapy biotech Lexeo Therapeutics has acquired Stelios Therapeutics, an early-stage company developing novel adeno-associated virus-mediated gene therapies for rare genetic cardiac conditions.

Through the acquisition, Lexeo obtains exclusive rights to three preclinical AAV-mediated gene therapy programs focused on TNNI3-associated hypertrophic cardiomyopathy (HCM) and arrhythmogenic right ventricular cardiomyopathy (ARVC), two rare cardiac disorders with no approved pharmacological treatments and significant commercial potential. Stelios’ Scientific Co-founder and Professor of Medicine in Cardiology at the UC San Diego School of Medicine Eric Adler will assist in cardiovascular research efforts at Lexeo.

The transaction was approved by the board of directors of both companies and closed immediately. Financial terms of the transaction are undisclosed.

HCM affects more than 650,000 people in the United States and is the second most common form of heart muscle disease, comprising 35-40 percent of cardiomyopathies. TNNI3-associated HCM patients represent approximately 5-7 percent of the HCM patient population, with an estimated disease prevalence of 30,000. The disease is caused by mutations in the TNNI3 gene encoding cardiac troponin I, a protein that modulates cardiac muscle contraction and relaxation. It is associated with thickening and enlargement of the heart, leading to decreased blood flow and increased risk of heart failure and life-threatening arrhythmias. Current treatment options are limited to invasive surgical procedures and heart transplantation.

ARVC is a heart disease with genetic mutations that impair the structure and function of the heart muscle, resulting in cardiac cell death, fibrosis, heart dysfunction, rhythm abnormalities, and sudden death in young people. The U.S. prevalence of ARVC is estimated at 1:2,000-1:5,000 people. More than 40 percent of ARVC patients die within 10-11 years of diagnosis.

“Despite recent medical advancements in the rare cardiovascular field, many rare cardiac diseases remain underdiagnosed and undertreated. There is an urgent need for disease-modifying solutions to address conditions such as TNNI3-associated HCM,” said Adler.

The deal expands Lexeo’s pipeline in rare genetic cardiac diseases with the addition of three novel programs and provides an opportunity to integrate the companies’ complementary capabilities, including Lexeo’s growing infrastructure in clinical development and chemistry, manufacturing and controls with an existing focus on rare cardiac diseases, and Stelios’ robust preclinical pipeline and domain expertise in rare genetic cardiac diseases.

The Stelios programs complement and broaden LEXEO’s advanced preclinical gene therapy pipeline in rare cardiac diseases, which currently includes LX2006, an IV-administered, AAV-mediated gene therapy program for the potential treatment of cardiomyopathy associated with Friedreich’s ataxia.

“While we are in the early stages of applying the potential of AAV gene therapy to rare cardiac diseases, the possible therapeutic benefits across a broad number of previously untreated and currently underdiagnosed cardiac diseases are immense,” said R. Nolan Townsend, CEO of Lexeo Therapeutics. “LEXEO and Stelios share the same commitment to advancing therapies for patients with rare cardiac diseases. The combined company will establish a leading position in the field of AAV cardiac gene therapy, enabled by a strong scientific footprint and a significantly enhanced pipeline, with the potential to move multiple programs into the clinic in the coming years to address substantial patients’ needs in various indications.”

Photo: R. Nolan Townsend, CEO of Lexeo Therapeutics

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