Saniona’s CSO: “We are on track with SAN2219”
Saniona has taken a step forward within its epilepsy program by initiating the scale-up and manufacturing of toxicology batches for SAN2219. This move propels the program toward clinical studies, targeted for the first half of 2026. BioStock reached out to Chief Scientific Officer, Karin Sandager Nielsen, for a comment.
The path to clinical trials is well underway. Saniona completed analytical development and synthesis optimization for SAN2219 in 2024 and has now signed a contract to produce GLP toxicology batches by summer 2025. Upcoming steps include GMP manufacturing and GLP toxicology studies, preparing SAN2219 for human testing.
Building on its proven track record – highlighted by the recent SAN711 collaboration with Acadia – Saniona aims to leverage its ion channel knowledge to drive this program forward. SAN2219’s selective activation of GABA-A α2/α3/α5 receptors sets it apart, with the aim of curbing excessive neural excitability with an improved safety profile over traditional benzodiazepines.
Resistance to existing drugs is a problem
Epilepsy affects 50 million people worldwide, yet one third remain resistant to current therapies, underscoring a pressing need for innovation. This gap spans idiopathic epilepsy and rare pediatric syndromes, where side effects often limit treatment options. Saniona believes SAN2219 could address this gap, with preclinical data demonstrating robust seizure control, high tolerability, and minimal risk of tolerance development – a rare trifecta in epilepsy care according to Saniona, though the program remains in its early stages. By avoiding the α1 subunit targeted by benzodiazepines, SAN2219 could potentially offer both acute and chronic relief in battling uncontrolled seizures.
“We are on track – this timely initiation demonstrates Saniona’s commitment to efficiently advancing SAN2219.”
Comments from the CSO
BioStock reached out to Karin Sandager Nielsen, Chief Scientific Officer at Saniona, for a comment on the recent development with SAN2219.
Could you start by explaining the importance of soon being able to initiate production of GLP toxicology batches?
– The initiation of GLP toxicology batches is a prerequisite for GLP toxicity and safety studies, which are essential for propelling the program toward clinical studies. Any delay in this step would push back the timeline for starting clinical studies which are targeted for first half of 2026. With the initiation of the production of GLP batches now underway, I’m pleased to share that we are on track. This timely initiation demonstrates Saniona’s commitment to efficiently and effectively advancing SAN2219 through the development pipeline.
Let’s move on to SAN2219, can you introduce the candidate and its potential significance in treating patients with epilepsy?
“With SAN2219, we anticipate a highly effective anti-seizure drug devoid of sedation, motor instability, tolerance and abuse potential.”
– At Saniona, we recognize the inherent risks in clinical development. Our strategy therefore leverages clinically validated targets, such as GABA, combined with our deep ion-channel expertise to create innovative molecules with transformative potential and a high probability of success.
– SAN2219 is one of these innovative compounds. It is a subtype-selective positive allosteric modulator (PAM) of GABAA α2-, α3-, and α5-containing receptors. This profile is specifically designed to provide broad antiseizure activity by dampening excessive neuronal activation throughout the brain while avoiding the adverse effects associated with non-selective activation that is obtained with benzodiazepines. Although benzodiazepines are effective anti-seizure drugs, their full potential is however limited by common side effects like sedation and motor instability, as well as risks of tolerance development and abuse. With SAN2219, we anticipate a highly effective anti-seizure drug devoid of these limitations.
– The effectiveness of SAN2219’s profile has been investigated in several well-established and reliable rodent seizure models, showing promising effects without causing adverse effects, thus validating this approach. Additionally, SAN2219 has been developed with an optimized pharmacokinetic (PK) profile.
Can you elaborate on the improved pharmacokinetic (PK) profile of SAN2219 and its implications for epilepsy treatment?
“The optimized PK profile of SAN2219 supports strong, sustained seizure control throughout the day.”
– SAN2219 has been engineered to have an optimized PK profile. This improved profile ensures that the drug reaches its target and maintains therapeutic levels for a longer duration, thereby minimizing breakthrough seizures. In our preclinical rodent models, SAN2219 has demonstrated strong, sustained seizure control at predicted human equivalent exposures throughout the day. In contrast, compounds with less optimal PK profiles showed variable seizure protection depending on the time of day.
– For epilepsy patients, this likely means more consistent and reliable therapeutic outcomes, potentially leading to better seizure control and an improved quality of life.
What are the next steps in the development of SAN2219?
“The goal is to finalize CTA/IND enabling package for the start of Phase 1 trials in the first half of 2026”
– We have successfully completed the synthesis optimization for production of GLP toxicology batches. As stated above, the next key milestones for SAN2219 include completing the manufacturing of the toxicology batches as well as the GMP batches to be used for the clinical studies. Pending successful results, we expect to finalize the CTA/IND enabling package for the start of Phase 1 clinical trials in the first half of 2026
What is your approach to de-risk the clinical development of SAN2219?
“By combining validated targets, ion channel expertise, and biomarkers, we are significantly de-risking SAN2219’s clinical development.”
– As stated earlier, our strategy is to build on clinically validated targets, combined with our years of expertise in designing ion-channel modulators. This approach allows us to produce highly differentiated assets that enhance efficacy and ultimately optimize clinical success.
– Importantly, we also leverage the use of biomarkers. In CNS disorders, it is paramount to establish biomarkers that can demonstrate early on if a compound is reaching its desired target in the brain and whether it is performing its intended function at the target level.
– For SAN2219, we are fortunate to have the ability to not only detect that the molecule is reaching its target via Positron Emission Tomography (PET) imaging studies but also to measure its interaction with the target in a relevant way through electroencephalographic (EEG) studies. This enables us to link doses with target engagement and detect relevant pharmacology early on, allowing us to identify optimal doses in Phase 1 clinical trials very effectively, enhancing a successful outcome of Phase 2.
– Leveraging these biomarkers significantly de-risks the development process and maximizes the likelihood of success. So, we are very optimistic about the future of SAN2219 and its potential to make a meaningful impact on the lives of patients suffering from epilepsy. It’s an exciting time for our company, and we are committed to advancing SAN2219 for the benefit of patients.
Finally, how does SAN2219’s selectivity position it to address unmet needs in epilepsy compared to existing treatments?
“SAN2219 has the potential to transform treatment for the 30% of epilepsy patients who do not respond to current therapies.”
– Benzodiazepines are among the most effective antiseizure medications available. However, their full potential is limited by adverse effects and tolerance concerns, primarily driven by activation of the GABA A α1 subunit. SAN2219 was specifically engineered to avoid activation of this subunit, thus it is not expected to cause these effects that limit benzodiazepines value. Saniona therefore believes SAN2219 has a good chance to address the approximately 30% of epilepsy patients who do not respond to current therapies, with great improvement of quality of life, not only for the patients but also for family and caregivers
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