Home News CombiGene gears up for clinical studies and expanded pipeline

CombiGene gears up for clinical studies and expanded pipeline


CombiGene gears up for clinical studies and expanded pipeline

17 November, 2021

CombiGene’s report for the third quarter is primarily defined by the important initiation of GMP production of drug candidate CG01 as an epilepsy treatment. However, it is the news that came right after the Q3 period that stands out the most – the exclusive, global licensing agreement for CG01 that CombiGene entered with American Spark Therapeutics, an agreement that changes the gene therapy company at its core. BioStock summarises the autumn for the company, as it approaches its first clinical trial.

In line with expectations, CombiGene did not report any net sales during Q3 2021. In this respect, the company is similar to the majority of biotech companies in the same development phase. Profit from financial items came in at -10.2 MSEK (-6.5) while other operating revenues amounted to 0.4 MSEK (2.7). Liquidity, per the end of the reporting period, was 66.1 MSEK (35.2).

Agreement with Spark

For research-based biotechnology companies, the skill to negotiate deals works as a good guide towards the bearing of projects. Here, CombiGene has delivered with conviction during autumn 2021.

On October 12, CombiGene entered into an exclusive, global licensing agreement with Spark Therapeutics, an independent subsidiary of Roche, the world’s third largest pharmaceutical company. At that point, the parties had already had ongoing dialogues for some time throughout the latter parts of CombiGene’s preclinical phase. The agreement involves CG01, CombiGene’s gene therapy developed for the treatment of drug-resistant focal epilepsy.

Under the terms of the agreement, CombiGene is eligible to receive up to 328.5 MUSD excluding royalties, with 8.5 MUSD upon signing, as well as up to 50 MUSD at preclinical and clinical milestones. In addition, CombiGene will be reimbursed for agreed development costs. Upon a future commercialisation, CombiGene is eligible for tiered royalties ranging from the mid-single digit up to low double-digit percentage points based on net sales.

As CEO Jan Nilsson notes in the Q3 report, Spark has the know-how and resources to drive the project forward through cost-intensive clinical studies. Supporting this claim, is the fact that Spark was among the first companies in the world to be granted approval for a gene therapy. This was Luxturna, for the treatment of a severe hereditary eye disease.

Taking CG01 towards the clinic

CG01’s preclinical programme has been ongoing in parallel with the negotiations with Spark. In collaboration with CRO NBR, CombiGene has begun tissue samples analysis from the toxicology and biodistribution study in animal models. Once the preclinical program is completed, Spark will take full responsibility for clinical development, from the first study in humans, all the way to global commercialisation.

The company’s second project – CGT2

But activities don’t stop there. In 2019, CombiGene inlicensed CGT2 from Lipigon Pharmaceuticals to develop a gene therapy treatment for the rare metabolic disease partial lipodystrophy. In doing so, the company broadened its pipeline to operate in two medical fields, neurological and metabolic diseases, with tangible synergies in terms of competence, network and experience between the different projects. Evaluation of possible drug candidates is currently underway.

Sharper focus on business development

CombiGene’s business development is based on three pillars: inlicensing new projects with high commercial potential, refining inlicensed projects through successful preclinical development, and outlicensing projects targeting significant patient populations in late preclinical, and early clinical, phases. The decision to inlicense CGT2 is thus in line with the company strategy. The lucrative agreement with Spark has provided opportunities to inlicense additional gene therapy projects going forward.

In an interview with BioStock, CEO Jan Nilsson emphasized that CombiGene is now entering an intensive business development phase with the goal of identifying and inlicensing new interesting projects with high commercial potential. Read more here.

Thus, in parallel with the development of two projects in which one – CG01 – has received funding from Horizon 2020 and the other – CGT2 – from Eurostars, CombiGene has the opportunity to continue to reduce the risk of its pipeline, by bringing in additional assets. The capital injection from Spark has further improved the momentum. BioStock is eager to follow the upcoming development, not the least the start of the clinical development of CG01.

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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