Saniona AB (publ) (STO:SANION)
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Life Science Summit 2025

Nov 17, 2025

Thomas Feldthus
CEO, Saniona

My name is Thomas Feldthus, CEO of Saniona. Quick reminder, forward-looking statements. We are focused on psychiatric diseases and neurology, and our market cap is currently about SEK 2.2 billion. Over the last couple of years, we have created a robust pipeline and secured two high-valuable deals enabling us to grow largely self-financed. We have a strong financial position, $70 million in cash, and we look forward to about $17.5 million milestones, hopefully next year. We are using the proceeds from these deals to advance free assets internally and to start a proof of concept, a lot of no-chill proof of concept. Our Iron Chain platform has been validated through, you could say, partnerships and also by creating programs for internal development. This slide shows our CNS pipeline, and recently we entered into a collaboration with Acadia and Jazz.

Acadia is advancing SAN711 for essential tremor treatment and they expect to start phase two next year. Jazz got licensed SAN2355 in August for epilepsy, and we expect them to start phase one next year. Then we have three assets which we are moving forward into the clinics, two of them for epilepsy and one for major depressive disorders. We expect to start two phase one studies in the second half next year and the other one in the first quarter 2027. Starting with the partner programs, SAN711 is a highly selective GABA α3 modulator. It's first in class, and phase one studies have shown that it's well-tolerated. We have biomarkers enabling Acadia to rational dose selection in the phase two studies. We got $28 million upfront. The total deal value is about $600 million plus royalties on product sales.

I think this is around top 5% for phase I deals worldwide over the last 5 to 10 years. SAN2355 is a highly selective KV7.2/3 activator, and it's positioned for epilepsy and potentially also other indications. It has the potential to become best in class, and this is why Jazz moved in. They paid $42 million upfront, and total deal value is more than $1 billion plus royalties on product sales. We have received over the last 12 months $70 million in cash. We have legal to $410 million in development milestones, of which $17.5 million will be paid when our partners are starting the next clinical studies. Then we have $1.6 billion in commercial milestones and then tiered royalties on product sales. Now to our internal pipeline. SAN2219 is an innovative add-on therapy for treatment-resistant epilepsy.

It's a GABA α2/3/5 modulator, which is designed to have the same efficacy as the benzodiazepines but with less side effects. Benzodiazepines are the most effective drugs in epilepsy, but they are not used that often because they cause tolerance induction and also sedation. Particularly, tolerance induction is a big problem in epilepsy, but then they don't work anymore. We are targeting resistant onchophilic seizures. It's a very large market, and we estimate that the potential market will be $1 billion for this product. We have shown that it has a strong anti-seizure effect in both, in all types of seizures, by the way, both generalized epilepsies and focal onset seizures. We have seen that it can display a synergistic and an additive effect when we combine it with standard of care, in this case, Keppra and Valproate, which are most commonly used epilepsy drugs.

It has dual effect, its own efficacy, plus it could increase the efficacy of the existing compounds the patients are using. It has no sedations and no instability on motor routes, and it is more effective than competing compounds out there on the relevant receptors for epilepsy, while it is minimal on alpha-1, which is causing all the side effects for benzodiazepines. Moving on to 2219, and I saw it, next one. Sorry about this. We start phase one in 2026, third quarter, and the proof of concept studies will be an RCT study, starting in 2028. Moving on to 2668, it is a unique GABA alpha-2/3 modulator. It is designed to have more effective efficacy than the benzodiazepines, but without the side effects.

It has a, we are starting with, we're going for pediatric epilepsies in this with this compound, ACEs, which is a very severe syndrome, causing major impact for the patient's memory and cognitive improvement, development. We have seen expansive possibilities for this $400 million market into other types of DEE indications as developmental epileptic encephalopathies, which cause harm to the patient's brains. We see a multiple dollar potential for this drug. In animal models, it is much more effective than other selective GABA modulators, in this case, the double efficacy of the regabet for MAPV and also our own compound 2219. It's even as equivalent efficacy effect to clobazam, which is a benzodiazepine, at equivalent receptor oncocapacity, in this case, 80%.

The thing is that we can reach probably receptor occupancy in the clinics of 70%-80%, but you can't do that with clobazam. It has the potential to be more effective in the animal model and in humans, without the side effects. I'll come to that now. 2668 does not cause sedation, and also you can see here on the right, which is very important. Here you see reduction on diazepam is reduced with 40% in efficacy when it has been chronic on a chronic dosing for one week. In our case, it's only the half, and it's completely similar to other selective compounds. We start the phase one study in the fourth quarter next year, and the open label study that I saw, not an open label, the phase, the proof of concept studies in 2028.

We may get some early readouts in 2028 from an open label study of about six patients, and then the double-blinded study can become one year later. I should say also here, this study may, this is an ACEs, but it may evolve into a basket study. We are looking a little bit like what Longboard did before they were acquired at Saniona, and we think we can do the same. This is what is giving it a complete different market potential. We have 2465, which is designed as a rapid onset, oral therapy for treatment-resistant depression. That is a lot of them, one third of all the patients, and there are many patients.

It has a significant potential because it, we think that it addresses all the medical needs in depression, rapid onset, cognitive improvement, and then with this mechanism also treatment of depression. It has the same, it follows this, it taps into the same pathway of esketamine, which is used as a kind of rescue medication in depression today for their resistance. It sells for $1 billion, but it's come with severe side effects, because it needs to be only and can only be given in hospitals for a couple of weeks, then it stops. It's still selling a billion-dollar product. This product has, goes into the same pathway, but with a much more selective mechanism through the GABA α5 receptors, which is restricted to the area in the brains where the human mouse is controlled.

It's the target selectivity we are going for here, and we achieve completely the same. In animal models of epilepsy, this one is a so-called chronic mild stress rat model. The rats develop typical symptoms of epilepsy, including depression, anhedonia, anxiety, and cognitive impairment. Our drug here normalized—we used ketamine as positive control. It's the same robust effect, same efficacy. There are plastic changes going on because the drug is gone when we see the last measurements. We normalize anhedonia. We see an organized anxiety, and we normalize the cognitive impairment of these rats in these studies. It looks very, very good. Phase one starting 2027, and then randomized trials in 2028. Our selective compounds have been validated through partnerships. They're creating funding to develop our own compounds, and we are moving now forward with two of these assets.

What could this bring us in five years from now? I've shown three deals here from 2024 in the neuroscience space. Longboard was acquired by Lundbeck for $2.6 billion, after they have conducted a phase two study, in DEE indications. What you could say, I think there's seven DEE indications. Only 55 patients, and this was what Longboard bought because they had support from FDA to go for a broad DEE label. This is precisely what we want to do with 2668. We have QoL support for this, and we are sitting in a forum where we can discuss this with the regulatory agency FDA. We have other assets, and we can look at this. In five years, we could have two programs ready to start a phase three. Karuna was sold for $14 billion.

They had one asset for phase three and then one phase one asset. Sierra Well had two phase three assets and two phase two assets, one of them Darriga, which has some similarities to our 2219. This is what we achieve aiming at. We have $90 million to pursue this path, and we may need a bit more, but I think there are several opportunities to get that through additional milestones from Acadia and Jazz in the midterm because they will develop their drugs in a similar way. We also see potential for new deals. We're probably going to sell one of them. There were also potential Medix royalties. Finally, I think this is an interesting case for institutional investors, particularly those who are invested in those companies and made a lot of money off that.

This is a good place to start. With that, we will also have, you know, non-coverage from independent coverage from banks, and then it starts to look up an interesting story. Thank you.

Thank you too. Let's go back to the science side of things a little bit. Someone is asking you to describe the competitive landscape for novel therapies, especially in the pediatric epilepsy. With the pediatric epilepsy. Yes. Yeah. What does the competitive landscape look like at the moment?

It's a, you know, there are so many of those indications. The two of them, Lennox-Gastaut syndrome and Dravet syndrome, they are quite large, and there you have two companies and psychedelics from actually Jazz and another compound. It's a really severe indications, and they need to help these kids.

What happens in epilepsy is that they get it, and then they get development in ACEs, for instance, they get seizures in the night when they should sleep and consolidate the memory. They can't, because of the seizures, then they can't remember what happened the day before. Some of them are losing their land because they end up in homes. These kids need help. You see many of these types of diseases, and there are 30 or 40 or 50 of them. There is a lot to go for. A lot of them will have these problems as the ACEs patients. This is where we come in because our compound has a very high modulating of alpha-3, which will resolve those seizures at night.

Thank you so much too.

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