On May 4, CombiGene’s annual report was published, and the overall impression is that the company could turn the page to 2021 after covering a year of several advances. Not only has the company’s preclinical program accelerated, but a production platform has also been established. The company also ticked off three new share issues, research grants from the EU and confirmations from regulatory authorities regarding how the company intends to carry out the final preclinical steps ahead of the first human studies scheduled to start next year.
Gene therapy company CombiGene is approaching its biggest milestone as a listed company – clinical trial start with drug candidate CG01, which is being developed for the treatment of drug-resistant patients with focal epilepsy, the most common form of the disease in adult epileptics.
CombiGene is also developing CGT2, a gene therapy treatment for partial lipodystrophy, a very rare disease that today has no treatment options. While the company value the addressable market for CG01 to be between 750-1500 million USD annually, CombiGene estimates that the total market for CGT2 is worth between 700-1450 million USD.
Progress with CG01 during 2020
In 2020, CombiGene completed three preclinical studies with positive results, and established a large-scale production platform together with its international partners Viralgen, Cobra and CGT Catapult.
The company also produced the first large-scale batch of CG01 to be used in the preclinical toxicology and biodistribution studies, which means that the final parts of the preclinical program have begun. In 2021, GMP material will also be produced for the first human study in 2022. This year, CombiGene also received positive responses from both the Swedish and UK pharmaceutical authorities, which confirmed CombiGene’s plan for the final preclinical studies with CG01.
Concept verification study awaits in the lipodystrophy project
The company’s other asset, the lipodystrophy project CGT2, has also taken steps forward. CombiGene has applied for patent protection for the vectors being developed within the project with the long-term goal of securing a global patent protection. Last year, work also began to identify the most promising drug candidate in order to initiate a concept verification study in 2021. CombiGene has the option to apply for orphan drug status for CGT2, which would bring a number of strategic benefits such as shorter development time, market exclusivity and lower development costs.
Capital secured from multiple fronts
In 2020, CombiGene carried out a rights issue and two directed issues that, including three series of warrants, provided the company with approximately 89 million SEK before issue costs. A payment was also received from Horizon 2020, the EU Framework Programme for Research and Development, which means that this programme now has committed 2.85 million EUR to the CG01 project. In addition, CombiGene received an additional 882 500 EUR in development appropriations for CGT2 from the EU Eurostars programme at the beginning of 2021.
CombiGene has so far manoeuvred its way through the preclinical studies without encountering any major obstacles. There are now some final steps to be taken before human studies can begin next year: the preclinical toxicology and biodistribution studies and the production of the GMP batch for the upcoming human study. With the funding secured in 2020 and 2021, these funds appear to be able to cover these extensive and capital-heavy steps, which should give the company peace of mind in the parallel work – to ensure that the ongoing dialogues with potential clinical partners and industrial partners bear fruit.
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