In CombiGene’s Q3 report, the terminated agreement with Spark Therapeutics takes up considerable space, for obvious reasons. However, the report highlights that the gene therapy company is now focusing on the COZY1 pain project. They have chosen both a production partner and a collaborator for upcoming preclinical toxicology studies, along with indications for the clinical trial program.
CombiGene’s gene therapy project CG01 is being developed for the treatment of patients with severe drug-resistant focal epilepsy who do not become seizure-free with today’s drug treatments. In 2021, CombiGene signed a collaboration and license agreement worth up to 328.5 million USD with US-based Spark Therapeutics for the continued development of the project.
When CombiGene announced on October 14, 2023, that it would regain global rights for the project from Spark, the disappointment and surprise were palpable within CombiGene. The market also responded negatively, resulting in CombiGene losing half of its market capitalisation.
CombiGene is not, however, obligated to repay any of the compensations it has so far received, totalling 8.5 million USD, excluding development costs. Meanwhile, CombiGene is not entitled to any future milestone payments or royalties. Read an interview with the company’s CEO, Peter Ekolind, about the terminated collaboration here.
Focus on the pain project
However, CombiGene is not a single-project company. In early 2023, it entered into a collaboration with the Danish biotech company Zyneyro to develop more effective treatments for severe temporary and chronic pain conditions. The joint pipeline includes the most advanced project – the peptide treatment COZY01 – as well as the gene therapy treatment COZY02. A recently presented doctoral thesis at the University of Copenhagen further supported the treatment concept. Read more here.
Contract manufacturer selected for the pain project
In September, CombiGene entered into a partnership with the contract manufacturer (CDMO) AmbioPharm for COZY01. According to CombiGene, the new partner has extensive experience in peptide development and manufacturing. Additionally, the partner meets all regulatory criteria, paving the way for toxicological studies and human trials. AmbioPharm’s capacity for commercial production also positions them as a potential long-term partner in the COZY01 project.
CEO Peter Ekolind commented on the selection of AmbioPharm as CDMO in a press release:
“The fact that we have now chosen a CDMO partner means that the COZY01 project has taken an important step towards being able to initiate the toxicology studies that are carried out in order to be able to submit a clinical trial application.”
Pain associated with shingles will be the first indication
In October, CombiGene and Zyneyro chose to focus the initial proof-of-concept study for COZY01 in humans on patients experiencing pain associated with Herpes Zoster (shingles) – a highly painful complication.
Following demonstrated proof-of-concept, the ongoing development will shift its focus to diabetic neuropathy, one of the most common chronic complications of diabetes, characterized by severe chronic pain. Like many other chronic pain conditions, this indication lacks effective treatments.
In a press release, Pernilla Fagergren, Director of Clinical Development at CombiGene, stated:
“The choice of indication in the peptide-based COZY01 project is an important milestone. In parallel with conducting the remaining preclinical studies, we can now begin the extensive work of preparing the clinical program, i.e., studies in humans.”
Partner for toxicology studies appointed
The next step forward in the COZY01 project came a few days later when CombiGene selected Charles River Laboratories as their new CRO partner (Contract Research Organisation). The choice of CRO brings them closer to initiating the toxicology studies necessary for filing a clinical trial application.
In addition to COZY01, the development of COZY02 is underway. A prototype of the AAV vector, serving as the carrier of the genetic material in the gene therapy, has been developed by Zyneyro. It has been tested in several preclinical models with highly positive and sustained effects. The companies’ focus is now on optimising the genetic material that will be part of the vector for administration in future human studies.
Change of vector supplier in the lipodystrophy project
In addition to COZY01, which holds the highest priority for the company, other projects continue to progress simultaneously. The preclinical project CGT2, CombiGene’s gene therapy for partial lipodystrophy, has faced delays. However, following the switch of vector supplier, new vectors will be used in collaboration with the University Medical Center Hamburg-Eppendorf to conduct and replicate the scientific proof-of-concept study. Results are expected in H1 2024. The project has received over 10 million SEK in project grants from the EU’s funding program Eurostars, and national patent applications have been filed in the USA and the EU.The content of BioStock’s news and analyses is independent but the work of BioStock is to a certain degree financed by life science companies. The above article concerns a company from which BioStock has received financing.