• “CDLK5 Forum Award for Excellence – Company Making a Difference 2023 Pre-clinical” presented by the Loulou Foundation at the CDKL5 Forum, 6th-7th November in London
• First-in-class ion channel inhibitors show promise as anti-seizure therapeutics for multiple treatment-resistant epilepsy subtypes
• CaV2.3 identified as a novel target for CDKL5 Deficiency Disorder
• Lario Therapeutics is a spin-out company from Epidarex Exeed, the therapeutic discovery engine of Epidarex Capital

Edinburgh, UK, 07 November 2023: Lario Therapeutics (‘Lario Tx’, or ‘the Company’), a biopharmaceutical company developing first-in-class precision medicines that are targeting disease-modifying treatments for severe neurological disorders, has received the “CDLK5 Forum Award for Excellence – Company Making a Difference 2023 Pre-clinical” from the Loulou Foundation at the annual CDKL5 Forum. The award is in recognition of its development of a validated, precision medicine approach in genetic epilepsies. Lario Tx’s first-in-class, orally active, CNS-penetrant CaV2.3 ion channel inhibitors hold promise as novel anti-seizure therapeutics for multiple epilepsy subtypes, including CDKL5 Deficiency Disorder.

The CDKL5 Forum, established by the Loulou Foundation, is the largest annual conference on CDKL5 Deficiency Disorder (CDD). CDD is the most prevalent form of genetic Developmental Epileptic Encephalopathy (DEE), affecting approximately 1:40K live births. Caused by loss-of-function mutations in the CDKL5 protein kinase expressed in the brain, the rare neurological disease leads to seizures, developmental delay and severe intellectual disability in most patients. The leading hypothesis is that CDKL5 regulates the function of the CaV2.3 channel, which can explain the devastating seizures characteristic of the disorder. CaV2.3 is now being investigated as a key target for CDD and CaV2.3 inhibitors hold great promise as a precision medicine approach.

Henning Steinhagen, Ph.D., Co-Founder and CEO of Lario Therapeutics, commented: “We are honored and thank the Loulou Foundation for their recognition of our approach which we hope will have a positive impact on patients affected by CDKL5 Deficiency Disorder. Our target, CaV2.3, represents one of the few newly validated precision medicine approaches in neurology and has a significant potential in severe epileptic conditions and other neurological disorders including Parkinson’s disease. We are planning to progress our therapeutic programs swiftly towards IND and beyond supported by our partners and investors.”

Dan Lavery, Ph.D., CSO of the Loulou Foundation, stated: “The CDKL5 Forum ‘Company Making a Difference Award (Pre-clinical)’ recognises the industry partner who has demonstrated the greatest commitment to pre-clinical therapeutic development for CDD over the past year. At the CDKL5 Forum 2023, the Loulou Foundation is pleased to recognise the team at Lario Therapeutics, a dynamic group of experienced scientists who are developing a novel therapeutic for our patients living with CDD. The

Lario team has shown amazing dedication and engagement with our community, and we are excited to have them join our CDKL5 community as they advance their CaV2.3 pre-clinical development program.” Ana Mingorance, Ph.D., CDO of the Loulou Foundation, added: “One of the major discoveries about CDD last year was that CaV2.3 is a key target for CDKL5, which is deregulated in the absence of CDKL5. This means that CaV2.3 inhibitors hold promise as a precision medicine approach to treat CDD.”

Sinclair Dunlop, Investor Director, Lario Therapeutics, said: “This is an exciting time for Lario Therapeutics, and Epidarex Capital is pleased to have supported Henning and the team to this significant point. The Company’s approach to developing novel disease-modifying precision medicines for the management of severe neurological disorders is potentially transformative, places Lario in a unique position to address unmet clinical needs, and represents a significant market opportunity.”

Lario Tx was founded in 2021 as a spin-out from Epidarex Exeed (epidarex-exeed.com), the therapeutic discovery engine of Epidarex Capital, with seed investment from Epidarex Capital and Axxam which transferred its participation into Golgi Neurosciences. The Company is rapidly advancing, led by Dr Henning Steinhagen, Co-founder and CEO, together with a highly motivated and experienced team.

For more information about Lario Therapeutics please visit lariotx.com.