New Gene Therapy for Heart Disease Gets Boost from Innovative Delivery System

XyloCor Partners with SmartCella

XyloCor Therapeutics, a company focused on developing gene therapies for heart disease, has entered into a licensing agreement with SmartCella Holding AB. This agreement grants XyloCor the use of SmartCella’s Extroducer® Infusion Catheter System for delivering their lead gene therapy candidate, XC001, directly to heart tissues.

The Extroducer is a novel device designed to administer therapies directly to specific areas within the body, which makes it ideal for use in upcoming clinical trials and commercial applications of XC001.

XC001: A promising treatment for heart disease

ZyloCor's lead candidate, XC001, aims to create new blood vessels in the heart by inducing the local expression of vascular endothelial growth factor (VEGF). This helps in reducing ischemic burden, which is a common issue in conditions like refractory angina.

The innovative delivery facilitated by the Extroducer could help many patients by making the treatment less invasive and reducing complications related to traditional surgical methods.

Expert Opinions on the Collaboration

Al Gianchetti, President and CEO of XyloCor, stated that this collaboration could significantly improve safety and ease of delivering XC001 without surgery. This approach aims to offer better patient outcomes while maintaining the accurate administration of the gene therapy to targeted heart tissues.

Dr. Timothy D. Henry, an interventional cardiologist, expressed optimism about starting a Phase 2b trial using the Extroducer. He believes this method could improve the effectiveness of the therapy and minimize risk.

Clinical Trials and Data

In earlier phases, specifically through the EXACT Phase 1/2 trial, XC001 demonstrated strong safety profiles and significant clinical improvements. Patients who received this one-time gene therapy showed better exercise durations and reduced frequency of angina episodes.

Six months post-treatment, 43% of the participants reported no chest pain during daily activities, and 58% mentioned having no angina episodes at the 12-month follow-up.

Future Implications

This partnership marks a notable advancement in gene therapy for cardiovascular diseases, particularly refractory angina. The use of Extroducer with XC001 could potentially expand treatment options for a larger patient pool earlier in the disease process.

SmartCella CEO, Niklas Prager, highlighted the global expansion efforts the collaboration signifies. He emphasized that combining the Extroducer delivery system with XC001 opens new doors for treatment possibilities worldwide.

Agreed Terms

The terms of the agreement stipulate that XyloCor receives a global license for using the Extroducer with XC001. SmartCella will supply the necessary catheters for clinical and commercial use in exchange for an upfront payment, milestone payments, and royalties amounting to approximately USD 130 million.

XyloCor plans to use this innovative catheter system to enhance the delivery and effectiveness of their gene therapy, ultimately aiming for improved patient outcomes.