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CombiGene is preparing for the clinic

CombiGene is preparing for the clinic

23 February, 2021

CombiGene’s year-end report for 2020, which was published last week, summarised what can be seen as a financially successful year with three new share issues and three series of warrants that brought in a total of SEK 89 million before issue costs. During the year, three research advances were also presented in preparation of the first study in humans with the gene therapy CG01 in epilepsy, which is planned to be initiated next year. CombiGene’s lipodystrophy project has also advanced and the company recently announced that they have received development funding from the EU Eurostars programme.

CombiGene reported earnings before tax of SEK -11.0 million for the fourth quarter of 2020 (-9.0). The company had no net sales during the quarter (0), but other operating income amounted to SEK 3.2 million (3.9). The cash balance amounted to almost SEK 49 million on the balance sheet date.

The result was expected, as CombiGene’s development work in recent years has primarily focused on developing a gene therapeutic treatment for drug-resistant focal epilepsy with the drug candidate CG01.

The strategy is to develop CG01 into clinical proof-of-concept in a phase I/II study and then out license or sell the project to a major player with financial and developmental resources that can take CG01 on to regulatory approval, the market and to patients. Following a well-known business model in the life science sector, the earnings potential lies ahead in time.

Clinical advances within the epilepsy program

In 2020, CombiGene secured the important production platform that will provide the company with materials both for the final parts of the preclinical program, and for GMP material for future studies in humans. The choice of platform also means that production volumes can be quickly scaled up to meet future commercial demand. As CEO Jan Nilsson states in the year-end report, it is proof of the competence of the company that it has succeeded with small resources in establishing this complex production platform.

CombiGene conducted three preclinical studies last year with CG01 that showed that the candidate is taken up in the right place in the brain; that it does not adversely affect memory and learning; and that an injection of CG01 into the brain should have a long-lasting effect for several years. In other words, these three studies constitute an important basis for increasing the chances of later obtaining approval from regulatory authorities to able to initiate studies in humans.

Proof-of-concept study is approaching in the lipodystrophy project

In 2019, CombiGene inlicensed CGT2 from Lipigon Pharmaceuticals with the aim of developing a gene therapy treatment for the rare metabolic disease partial lipodystrophy. Thus, the company broadened its pipeline to be active in both neurological and metabolic diseases.

Lipodystrophy refers to the abnormal distribution of body fat in the body, which can lead to diseases like diabetes, acute pancreatitis, as well as cardiovascular disease and inflammation of the liver.

There are currently a few symptom-relieving treatments for lipodystrophy, but none that address the root cause of the disease, which significantly shortens life expectancy. As it is a rare disease, CombiGene can apply for orphan drug designation status, which would, among other things, bring a number of regulatory benefits, market exclusivity and opportunities for significant price premiums for an approved drug.

In 2020, CombiGene has applied for patent protection for the vectors being developed within the CGT2 project, and, in the autumn, work began to identify the most promising drug candidate in order to be able to carry out a proof-of-concept study during this year.

Eurostars grant to CombiGene’s CGT2 project

CombiGene was recently able to add to the positive news flow during 2021 when it was announced that the company has received a development grant of EUR 481,000 from the EU Eurostars program for its lipodystrophy project. Eurostars is a European financing program for small and medium-sized companies that want to collaborate on research and development.

Thanks to the grant, which amounts to a total of EUR 882,500, CombiGene can expand its collaboration on CGT2 by including a German expert group within lipid research at the University of Hamburg, University Medical Center Hamburg-Eppendorf. The grant also includes German Accelero, a GLP-certified CRO that will develop analysis methods to measure the effect of CGT2.

Annika Ericsson, Preclinical Projekt Manager at CombiGene, commented in a press release:

»The grant is important in itself as it allows us to drive the project forward at full pace. Our high ranking among the companies that have applied for funding from the Eurostars program, I also regard as confirmation of the project’s scientific height and CombiGene’s ability to, in cooperation with external partners, run an effective and successful development process. This is a great day for CombiGene’s lipodystrophy project and for all those patients who are waiting for effective treatment of this severe disease.« – Annika Ericsson, Preclinical Project Manager, CombiGene

Well capitalised and eyeing 2022

Since CombiGene was listed on the stock exchange in 2015, the focus has been on taking CG01 towards clinical studies and testing the hypothesis around the candidate’s mechanisms of action in patients. The goal is for this to take place next year, but first the preclinical program – the biodistribution and safety studies – will be completed. In parallel, the company is preparing for a human study, which presuppose an approved safety profile by the authorities.

In 2018, CombiGene was able to announce that Horizon 2020 – the EU’s framework program for research and development – is investing EUR 3.36 million in the company’s CG01 gene therapy project. Towards the end of last year, the company received a third payment, which means that the EU have so far invested EUR 2.85 million in the CG01 project.

CombiGene was also able to implement three successful share issues and three series of warrants in 2020 that brought in a total of SEK 89 million before issue costs. This gives the company financial strength to continue the operation at full speed.

BioStock will follow CombiGene’s continued development journey during the year, with a view on what is in the cards for 2022. With an established production platform, a strengthened organisation with expertise in pharmaceutical manufacturing and clinical project management and three preclinical studies with the potential to satisfy the regulatory authorities, the company in any case has set the stage for a clinical entrance.

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.

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