Saniona develops the lead candidate Thesomet for the treatment of patients with Prader-Willi syndrome (PWS) and hypothalamic obesity (HO). These are two serious, rare diseases that lead to intractable weight gain, metabolic dysfunction and uncontrollable hunger. Tesomet is a fixed-dose combination of tesofensine, a triple monoamine reuptake inhibitor, and metoprolol, a selective beta-1 blocker.
Saniona has made progress in 2021, both in clinical development and regulatory terms. Tesomet received orphan drug designation (ODD) from the FDA for both PWS and HO during the year, the first orphan drug designation ever granted in this disease. Clinical trials have also been initiated in both indications.
Has initiated phase IIb studies in HO and PWS
In November, Saniona announced the initiation of the planned Phase IIb study with Tesomet in HO. This randomized, double-blind, placebo-controlled study will last for 36 weeks, followed by an open-label extension of the same duration, with the goal of recruiting approximately 110 patients. The study is being conducted at multiple sites worldwide and results are expected in the second half of 2023.
Saniona's ambition has been to also initiate a phase IIb study in PWS before the end of the year, an ambition that has now been fulfilled. The plan is for the phase IIb study to recruit 120 patients with genetically confirmed PWS. During a 16-week double-blind period, participants will be randomized to receive daily dosing with Tesomet at one of three dose levels or placebo. During a 36-week open-label extension period, participants who wish to continue treatment, including those who initially received placebo, will receive the highest tolerated dose of Tesomet determined during the double-blind period. Results from this study are expected in the first half of 2023.
First patient dosed with SAN711
As if these initiated studies weren't enough, Saniona has more developments up its sleeve for 2022. In June, the company announced that it had dosed the first patient in a Phase I clinical study with SAN711, a novel molecule derived from Saniona's proprietary ion channel drug discovery platform. SAN711 is a first-in-class positive allosteric modulator of GABA-A α3 receptors and has demonstrated efficacy in several preclinical models of neuropathic pain and may be applicable in the treatment of other rare neuropathic disorders.
Data from the study is expected in the first half of 2022. The company is also developing SAN903 as a potential treatment for rare inflammatory, fibrotic and hematological disorders. SAN903 is expected to enter clinical development in H2 2022.
CEO comments
BioStock contacted Rami Levin, President and CEO of Saniona, to get his perspective on Saniona's clinical trials and the path forward for the company.
Three initiated studies in less than six months are a great achievement for a company of Saniona's size. How was this accomplished, Rami?
- Saniona was able to accomplish the goal of initiating three clinical trials in 2021 because we had the right team with the right expertise in place. We knew this goal was ambitious for a smaller company like Saniona, and so we prioritized hiring very experienced people in clinical, regulatory, technical operations and quality.
- We created the Tesomet capsule, prepared and filled more than 130 regulatory submissions with the FDA and other agencies around the world, and established the appropriate quality controls to oversee all our clinical and manufacturing processes.
- I am proud of what we accomplished in 2021, including securing two orphan drug designations and starting three clinical trials. I am equally excited about what this talented team will be able to accomplish in the future as we continue to advance our programs.
What can we look forward to in 2022?
- This year will be important for Saniona, with multiple milestones expected. As we advance Tesomet through the two phase IIb trials for HO and PWS, we will also be focusing on the next generation of rare disease medicines in our pipeline. We expect data from our phase I trial of SAN711 in the first half of 2022, and we plan to advance SAN903 into phase I in the second half of the year.
- Additionally, our research team is closing in on the next candidate from our ion channel drug discovery engine. We expect to nominate that candidate and advance it into our pipeline in 2022. In addition, our partner Medix is awaiting feedback from the Mexican regulatory authorities regarding the potential approval of tesofensine for general obesity in Mexico.
- As we have stated, another significant priority for Saniona right now is business development. While the timing of these deals can never be guaranteed, we are actively pursuing opportunities that could provide non-dilutive funding.