Saniona Hosts Research and Development (R&D) Day on Proprietary Ion Channel Drug Discovery Engine and Pipeline Programs

PRESS RELEASE

May 20, 2021

Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced that it hosted a virtual R&D day event, during which it discussed its ion channel drug discovery engine and proprietary IONBASE™ database. The company also presented data from its ion channel pipeline programs, SAN711 and SAN903.

A replay of the event is available on the Investors section of the Saniona website, under Events and Presentations, and here: https://media.rampard.com/20210520/.

"Our industry-leading ion channel drug discovery engine has enabled us to build a diverse pipeline of highly selective compounds with the potential to address unmet needs across a variety of diseases,” said Rami Levin, President and Chief Executive Officer of Saniona. "We were pleased to present our proprietary technology, providing new details on preclinical candidates SAN711 and SAN903, which demonstrate the power of our discovery engine to generate a steady source of preclinical candidates that are rapidly advancing towards the clinic.”

Saniona’s ion channel drug discovery engine combines in-house expertise in chemistry, precision biology, in vivo stability/distribution, target engagement, in vivo pharmacology, and artificial intelligence to accelerate the discovery of highly selective, subtype-specific, and state-dependent ion channel modulators. The core of this engine is Saniona’s proprietary IONBASE database, which contains structure-activity data for more than 120,000 compounds. Of these, 20,000 are Saniona’s proprietary compounds, generated over 20 years and enriched for optimal ion channel modulation.

SAN711 is a first-in-class drug candidate that selectively enhances the effects of GABA-A on a3 containing receptors. This selectivity is ideal for rare neuropathic diseases and may allow SAN711 to avoid the typical adverse effects associated with non-selective GABA-A activation such as such as sedation, motoric instability, cognitive impairment, abuse liability and physical dependence. During the R&D Day event, Saniona presented preclinical data demonstrating efficacy from in vivo models for trigeminal neuralgia, a rare form of neuropathic pain. After acute dosing, SAN711 reduced pain in a dose-dependent manner, with the highest dose achieving pain relief similar to the standard of care carbamazepine, which has numerous serious side effects. After chronic dosing, SAN711 was demonstrated to prevent the development of pain, and also to maintain efficacy over time. In another preclinical model of chronic pain in which SAN711 was compared to morphine, SAN711 maintained efficacy over seven days of dosing, while the initial efficacy of morphine was completely eradicated due to tolerance over the same time period. Data comparing SAN711 to the non-selective GABA-A activator diazepam demonstrated that SAN711 did not induce the sedation seen with diazepam. Saniona expects SAN711 to enter a Phase 1 study in healthy volunteers in mid-2021, with data expected in early 2022.

SAN903 is a novel inhibitor of the calcium-activated potassium ion channel, KCa3.1, that is in development for the treatment of rare inflammatory and fibrotic disorders. Data presented during the R&D Day event demonstrated the efficacy of SAN903 in multiple in vitro and in vivo models of inflammation and fibrosis. Data presented at the recent American Society of Pharmacology and Experimental Therapeutics (ASPET) Annual Meeting at Experimental Biology (EB) 2021 demonstrated that SAN903 reduced inflammation and fibrosis with greater efficacy than two marketed products, nintedanib and pirfenidone, in an in vivo model of idiopathic pulmonary fibrosis. Saniona expects to initiate a Phase 1 study evaluating SAN903 in healthy volunteers by mid-2022.

For more information, please contact
Trista Morrison, Chief Communications Officer, Saniona. Office: + 1 (781) 810-9227. Email: trista.morrison@saniona.com  

This information was submitted for publication, through the agency of the contact persons set out above, at 20.30 CEST on May 20, 2021.

About Saniona
Saniona is a biopharmaceutical company focused on discovering, developing, and delivering innovative treatments for rare disease patients around the world. The company’s lead product candidate, Tesomet, is in mid-stage clinical trials for hypothalamic obesity and Prader-Willi syndrome, severe rare disorders characterized by uncontrollable hunger and intractable weight gain. Saniona’s robust drug discovery engine has generated a library now consisting of more than 20,000 proprietary modulators of ion channels, a significantly untapped drug class that is scientifically validated. Lead candidate SAN711 is entering Phase 1 for rare neuropathic disorders, with SAN903 for rare inflammatory and fibrotic disorders advancing through preclinical development. Led by an experienced scientific and operational team, Saniona has an established research organization in Copenhagen, Denmark and is building its corporate office in the Boston, Massachusetts area, U.S. The company’s shares are listed on Nasdaq Stockholm Small Cap (OMX: SANION). Read more at www.saniona.com.

Attachment

• RD Day Summary PR 05.20.21