Nordic Life Science 1
RARE DISEASE COMPANY Saniona was founded in 2011
and focuses on R&D of drugs for the treatment of rare eating disorders and diseases of the central nervous system. According to its website the company focuses on three major classes of ion channels; chloride ion selective channels, channels selective for cations and that are activated by the signal molecule acetylcholine, and channels selective for the potassium ion. The two eating disorders the company is working on finding treatments for are Prader Willi Syndrome and Hypothalamic Obesity. Tesomet, a fixed-dose combination of tesofensine and metoprolol, is currently is being tested in late-stage clinical trials for the treatment of Prader Willi Syndrome, hypothalamic obesity, and metabolic diseases. Tesofensine, a triple monoamine reuptake inhibitor, is a possible new treatment for obesity. It is a new chemical entity that has not previously been made commercially available. The company has several programs in clinical de-velopment and intends to develop and commercialize treatments for orphan indications on its own. Saniona is based in Copenhagen, Denmark, and its shares are listed on Nasdaq Stockholm Small Cap. Nordic Life Science asked Saniona’s new CEO, Rami Levin, about his company’s current activities and future plans. Describe your current status of Tesomet and tesofensine? ”Tesomet is our proprietary drug, which we are testing in the rare eating disorders Prader Willi Syndrome (PWS) and Hypothalamic Obesity (HO). We have phase 2a data for Tesomet in PWS. The data shows that Tesomet is safe and effective in doses we intend to pursue in further clinical development. We are having a pre-IND meeting with the FDA in early May to further align on our continued development in PWS. In HO, we are in the middle of phase 2. Data read out is expected by early May, after which we intend to meet with the FDA for a pre-IND meeting as well, to discuss our further development in HO.” Tesofensine is a product we partnered with Medix in Mexico and Argentina. Medix has submitted the Tesofensine file for approval in obesity to the Mexican authorities and are expecting approval in the second half of this year. They also plan to file for approval in Argentina next year.” Tell me more about SAN711 and its possibilities? ”SAN711 is a molecule with a unique profile. It is specifically designed to normalize aberrant activity associated with rare neuropathic pain disorders such as Burning Mouth Syndrome or rare intractable neuropathic itch conditions such prurigo nodularis and brachioradial pruritus; all rare conditions where no efficient treatment exists today resulting in poor quality of life for the affected patients. The molecule acts on the receptors for GABA, the main inhibitory signaling mediator in the nervous system. It works selectively on receptors containing the GABAA α3 proteins without acting on the main GABAA receptors, making it the first molecule with this unique profile. This implies that SAN711 may regulate the body’s own pain and itch regulating system in the spinal cord without causing side effects. This concept has been supported by preclinical studies with the compound. SAN711 is a new chemical entity covered by a recently filed patent application that may provide composition-of-matter protection until 2038.” Tell me more about SAN903 and its possibilities? ”We has selected a drug candidate, SAN903, currently under preclinical development and positioned for treatment of rare inflammatory/fibrotic diseases with no or very inefficient current treatment options. SAN903 is safe and shows efficacy in standard models of inflammatory diseases. Our current focus is idiopathic lung fibrosis (IPF), which is a serious and fatal lung disease with very limited treatment options. 30 000 to 150 000 patients suffer from IPF in the USA. SAN903 is a new chemical entity covered by a recently filed patent application that may provide composition-of-matter protection until 2039. The IK potassium channel (also known as KCa3.1, encoded by the gene KCNN4) is very important for controlling immune cell functions in both peripheral tissues (T-cells, macrophages) and the brain (microglia), and is also involved in the abnormal production of connective tissue (by fibroblasts), which leads to fibrosis in chronic diseases. We are also actively evaluating SAN903 for potential treatment of a rare hematological disease called hereditary xerocytosis (HX) estimated to affect 8 000 to 40 000 patients in the USA. Recent literature evidence demonstrates that HX is caused by gain-of-function mutations in the IK channel gene or in a closely associated Ca-channel and SAN903 is expected to reverse this condition. San903 inhibits the IK potassium channel (a calcium activated potassium channel also known as KCa3.1, gene KCNN4), which is important for activation of immune cells and collagen-producing fibroblasts. A precise pharmacological modulation of the IK channel can thus potentially treat rare diseases that involve overactive or mistimed immune reactions, such as idiopathic pulmonary fibrosis (IPF), a serious and fatal lung disease with limited treatment options.” What’s your strategy for entering the US market? ”Our strategy is to build a fully-fledged US organization. That will in essence become our new headquarters, with Copenhagen remaining our research center. We have already initiated the search for a new CMO and Head of Clinical Development and a new CFO, both of whom will be sitting with me in Boston, and further roles will follow after that.” Describe your business strategy and future plans for the development of Saniona? ”We have three main focus areas: expanding our shareholder base and bringing US institutional investors, building out the US organization into a fully-fledged organization and continuing to advance our clinical development in PWS and HO.” NLS NORDICLIFESCIENCE.ORG 63