The funds will also enable the development of next generation compounds, according to the company.
Axonis Therapeutics, which operates in the US, has announced $115m in Series A financing to progress its lead therapy candidate.
Cormorant Asset Management and venBio Partners co-led the financing, with significant investments from Sofinnova Investments, MRL Ventures Fund, Perceptive Advisors, Lumira Ventures and Solasta Ventures.
Eos Bioinnovation, Hatteras Venture Partners, SCI Ventures, Alexandria Venture Investments, Tachyon Ventures, BoxOne, Iaso Ventures and Pathway Bioventures also participated in the financing.
The company, which was co-founded by two University College Cork graduates, raised $5m in 2021 in order to develop therapies for neurological disorders. Now, Axonis will use its latest funding to advance its lead development candidate, AXN-027.
According to Axonis, AXN-027 is an oral small molecule designed to improve the function of KCC2, a major central nervous system chloride transporter that aids in the treatment of epilepsy and pain.
The newly announced proceeds will also enable the development of next-generation compounds targeting KCC2 for treatments of other issues such as psychiatric and neurodevelopmental disorders.
At present, many patients worldwide do not tolerate or respond to current first-line therapies due to impaired synaptic inhibition, which can contribute to drug resistance.
One study from 2020 suggests that the mechanisms of drug resistance “are likely to be variable and multifactorial”.
In the context of Ireland, with the Irish population now exceeding 5m, it is estimated that there are more than 45,000 people living with epilepsy in Ireland, according to Epilepsy Ireland.
Commenting on this particular obstacle, Axonis has said that its oral KCC2 potentiators are designed “to restore functional inhibition and overcome drug resistance in patients”.
Dr Joanna Stanicka, chief executive officer, president and co-founder of Axonis, said: “As we head into the clinic later this year, this financing is a significant milestone for Axonis as we translate our exciting science into efficacious and well-tolerated treatments for patients suffering from neurological disorders”.
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