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ApTOLL: An innovative neuroprotectant to reduce brain damage in Acute Ischemic Stroke (AIS)

Periodic Reporting for period 1 - ApTOLL (ApTOLL: An innovative neuroprotectant to reduce brain damage in Acute Ischemic Stroke (AIS))

Okres sprawozdawczy: 2024-01-01 do 2025-02-28

Stroke is a devastating medical condition affecting 15 million people worldwide yearly, being Ischemic Stroke the most frequent (87%). Indeed, the global cost of stroke is >€847b (≈€60b in EU) due to the huge socioeconomic burden of disabilities. Reperfusion treatment improves outcomes, but only ≈20% of patients are currently eligible for treatment due to the narrow therapeutic window after stroke symptom onset and stringent selection criteria for eligibility. Novel therapies - small molecules or biologics - targeting neuroprotection have not achieved enough efficacy to get approved.

To face this challenge, AptaTargets has developed ApTOLL, which is an innovative neuroprotectant aimed at reducing brain damage in Acute Ischemic Stroke (AIS) when used in combination with endovascular therapy (EVT).
During the first half of the apTOLL project´s implementation, AptaTargets has advanced in the following activities:
• the design and preparations of late-stage clinical trials in acute ischemic stroke patients,
• optimization of the manufacturing process and
• development of non-clinical studies to expedite the access to the markets.

Regarding exploitation activities, AptaTargets has achieved a major objective, signing a transaction with a big pharma.
Following the successful preclinical development and a Phase I First-in-Human trial, AptaTargets conducted a Phase II clinical trial in AIS patients with outstanding results, showing that ApTOLL is able to reduce the brain damage and significantly improve functional outcomes.

In addition, AptaTargets has advanced in its ApTOLL-AIS program and has recently partnered with a global pharmaceutical company that will take the product to the market and launch clinical trials in other indications.
AptaTargets team at International Stroke Conference 2025, Los Angeles (USA).
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