Alcyone Therapeutics Announces Strategic Financing on Heels of Expanded Collaboration with Nationwide Children’s Hospital

Rare Daily Staff

Alcyone Therapeutics said it closed a strategic financing from Nationwide Children’s Hospital that follows an expansion of the collaboration between the two.

The privately held developer of treatments for rare pediatric neurological diseases did not disclose the size of the strategic financing. A spokesperson said the funds will be “adequate to drive forward with the gene therapy programs and our biopharma collaboration.”

Existing investors also participated in the financing. The company did not disclose the total amount it raised in the transaction. Alcyone said the funding will also be used to support the continued development of ThecaFlex DRx, the company’s intrathecal drug delivery system.

Alcyone works closely with scientists from the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI) in Columbus, Ohio, which has designed four discrete gene therapy platform technologies. Through the collaboration with AWRI, Alcyone is exploring the potential for the clinical application of these therapeutics using Falcon, its proprietary CNS precision drug delivery and dosing technology platform.

As part of the expanded collaboration with AWRI, the company in April added a clinical-stage CLN3 Batten disease gene therapy program (CLN-301) to its therapeutic pipeline.

Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system that usually begin during childhood. There are 13 known forms of Batten disease in which a defect in a specific gene triggers a cascade of problems that interfere with a cell’s ability to recycle certain molecules. CLN3 Batten disease, also known as juvenile neuronal ceroid lipofuscinosis (JNCL), is caused by mutations in the CLN3 (ceroid lipofuscinosis, neuronal 3) gene. CLN3 encodes a transmembrane protein (embedded in the cell membrane) involved in the trafficking and recycling of proteins and other substances within cells.

The experimental gene therapy CLN-301 is a potential first-in-class AAV9 gene therapy in phase 1/2 clinical development. It is designed to deliver the coding sequence of the CLN3 gene to cells of the central nervous system to address an underlying enzyme deficiency that results in progressive cell damage and neurodevelopmental and physical decline.

Initial results from a phase 1/2 trial with CLN-301 demonstrated safety and therapeutic effects in a cohort of four patients compared to the natural progression of CLN3 Batten disease. On the Unified Batten Disease Rating Scale, patients in the CLN-301 study remained stable with a slope of -0.22 points per year over three years. By comparison, an increase of 2.86 points per year, leading to an overall 8.6-point increase in impairment over 36 months would be typical according to natural history data.

Typical outcomes for patients with CLN3 Batten disease include rapid vision loss, cognitive and motor decline, and behavioral issues. The majority of patients treated with CLN-301 and followed for over five years to date have shown maintenance of skills in motor and cognitive function and overall health compared to natural history data.

“Based on observations from the patients dosed so far,” said Kathrin Meyer, chief scientific officer for Alcyone, “addressing the underlying CLN3 deficiency with CLN-301 gene therapy has the potential to significantly alter the course of this devastating disease and to meaningfully impact life quality for CLN3 patients and their families.”