CombiGenes the longest running project is the gene therapy candidate CG01 which is being developed for the treatment of patients with severe drug-resistant focal epilepsy who do not become seizure-free with current drug treatments. In 2021, CombiGene signed a collaboration and licensing agreement worth up to USD 328,5 million with US-based Spark Therapeutics for continued development.
When CombiGene announced on October 14, 2023 that it would regain global rights to the project from Spark, CombiGene was clearly disappointed and surprised. The market also reacted negatively, trading the stock down, halving its market value as a result.
It is worth noting that CombiGene is not obligated to repay any of the compensation the company has received, a total of 8,5 million USD excluding development costs, but is also not entitled to any future milestone payments or royalties. Read an interview with the company's CEO Peter Ekolind about the terminated cooperation here.
Focus on the pain project
CombiGene is not a one-project company, however. In early 2023, it entered into a collaboration with the Danish biotechnology company Zyneyro for the development of more effective treatments for severe temporary and chronic pain conditions. The joint pipeline consists of the most advanced project – the peptide treatment COZY01 – as well as the gene therapy treatment COZY02. A recently presented doctoral thesis at University of Copenhagen further strengthened 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. In addition, the partner meets all regulatory criteria, paving the way for toxicological and human studies. AmbioPharm's capacity for commercial production also positions them as a potential long-term partner in the COZY01 project.
Vd 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 being conducted with the aim of submitting a clinical trial application.”
Pain associated with shingles becomes the first indication
In October, CombiGene and Zyneyro chose to focus the first Proof-of-Concept study for COZY01 in humans on patients with pain associated with Herpes Zoster (shingles) – a very painful complication.
After the Proof-of-Concept has been demonstrated, further development will focus on diabetic neuropathy, one of the most common chronic complications of diabetes where a clear symptom is severe chronic pain. As with many other chronic pain conditions, there is a lack of effective treatments for the indication.
In a press release, Pernilla Fagergren, CombiGenes Director Clinical Development, the following:
"The selection of indication for the peptide-based COZY01 project is an important milestone. In parallel with conducting the remaining preclinical studies, we can now begin the extensive work to prepare the clinical program, i.e. studies in humans."
Partner for preclinical toxicology studies appointed
The next step forward in the COZY01 project was taken a few days later when CombiGene chose Charles River Laboratories as a new CRO (contract research organization) partner. The choice of CRO means that the company is getting closer to initiating the toxicology studies that are required to later submit a clinical trial application.
In addition to COZY01, COZY02 is also in development. A prototype of the AAV vector that serves as a carrier of the genetic material in the gene therapy has been developed by Zyneyro and tested in several preclinical models with very good and long-lasting effects. The companies' focus is now on optimizing the genetic material that will be included in the vector in order to be able to administer this in future human studies.
Change of vector supplier in the lipodystrophy project
In addition to COZY01, which has the highest priority for the company, other projects continue to develop in parallel. The preclinical project CGT2, CombiGene's gene therapy treatment for partial lipodystrophy, has suffered delays, but after changing vector supplier, new vectors will be used in collaboration with University Medical Center Hamburg-Eppendorf to conduct and repeat the scientific Proof-of-Concept study. Results are expected in H1 2024. The project has received over SEK 10 million in project grants from the EU funding program Eurostars and national patent applications have been filed in the US and EU.