QurAlis Corporation has licensed preclinical small molecule compounds — designed to treat amyotrophic lateral sclerosis (ALS) — from Eli Lilly and Company, furthering QurAlis’s specialization in ALS treatment development.
The investigational molecules are aimed at specifically counteracting a pathological symptom of ALS called excitotoxicity, a process by which receptors in nerve cells (neurons) are over-stimulated by excitatory signals like the neurotransmitter, or chemical messenger, glutamate.
From patient-derived data, researchers at QurAlis have found that excitotoxicity contributes to the degeneration of motor neurons in anywhere from 20%–50% of ALS patients. This is, in part, because excitotoxicity can cause calcium ions to flow into neurons, and high calcium levels can trigger programmed cell death.
Additionally, hyperactivity can cause the accumulation of toxic proteins and free radicals in neurons, which can impair functionality. The removal of these toxins is slowed in ALS patients, furthering the progression of the disease.
By licensing treatments that may potentially stop this process, QurAlis intends to develop strategies to prevent the progression of ALS.
“Data from ALS patient-derived stem cell models suggest that excitotoxicity is a major driver of motor neuron degeneration in ALS,” said Dan Elbaum, PhD, CSO of QurAlis, in a press release. “The compounds included in this licensing agreement will enable us to target neuronal excitotoxicity in ALS in a novel way.”
QurAlis believes that the newly licensed compounds have potential to be targeted ALS therapies that will potentially modify the course of disease itself.
“We believe that the compounds we are in-licensing from Lilly have great potential for the development of a targeted ALS therapy, and we intend to advance these compounds to the clinic and to patients in need,” said Kasper Roet, PhD, CEO of QurAlis. “Our ultimate goal has always been to develop clinically meaningful disease modifying medicines for ALS, and this agreement represents a big step in that direction.”
The company has also shown optimism about analyzed data from early clinical trials for these treatments.
“We are very encouraged by the signs of activity from early clinical trials on this pathway, which suggest that we may be able to prevent the toxicity to neurons typical of ALS and hopefully modify the course of the disease,” Elbaum said.
As QurAlis specializes in ALS treatments, the company is taking other approaches to treat the disease as well. In addition to excitotoxicity, QurAlis is investigating the disruption of neural pathways occurring in ALS patients, which also leads to the build-up of toxic products in neurons.
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