BIAL Acquires Parkinson’s Treatment LTI-291, Opens US Research Center

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by David Melamed, PhD |

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Portuguese pharmaceutical company BIAL has announced the opening of a Parkinson’s disease research facility in Cambridge, Mass., as part of its expansion to the United States under the name BIAL Biotech.

Simultaneously, BIAL has acquired the Parkinson’s research programs operated by Lysosomal Therapeutics (LTI), including its lead candidate, the small molecule treatment LTI-291. The therapy’s development will continue to be led by the former LTI R&D team, now also part of BIAL Biotech, headed by Peter Lansbury, PhD, professor of neurology at Harvard Medical School.

The new BIAL Biotech plans to advance LTI-291, designed to treat genetic forms of Parkinson’s, to Phase 2 clinical testing by 2021.

“Our entry into the US with the creation of BIAL Biotech and the acquisition of the promising programs from LTI, is a decisive step towards the fulfilment of our mission to contribute to improving the quality of life of people worldwide,” António Portela, executive president of BIAL, said in a press release. “The development of this new research center in the US, is a landmark of enormous relevance for us. We are investing in science and research, through our direct presence in one of the most important research hubs in the world and in one of the most promising areas of medicine.”

The dual updates will enable BIAL to further advance its mission of promoting research into disorders of the central nervous system, comprised of the brain and spinal cord, with a specialization in Parkinson’s disease. The company said it chose Cambridge because it considers the city to be a hub of biotech research in the U.S.

BIAL’s new center will focus on genetically inherited Parkinson’s, which occurs in approximately 15-25% of cases.

A handful of genes have been shown to play a role in the genetic inheritance of the disease, including a mutation of the GBA gene, which codes for a protein called glucocerebrosidase (GCase), and affects about 10-15% of Parkinson’s patients.

GCase is a digestive enzyme that is primarily active inside the lysosome, a cellular component that is responsible for breaking down large molecules and removing cellular waste.

Deficiencies in GCase activity are thought to lead to an accumulation of toxic chemicals inside brain cells, which may cause the onset of Parkinson’s disease. However, the exact connection between GCase and Parkinson’s is not known.

As a potential treatment for Parkinson’s, researchers at LTI developed LTI-291, a small molecule designed to activate GCase and increase its activity.

“We are happy to be part of BIAL and take the lead on the growth story in the US,” says Kees Been, former CEO of LTI and now the new CEO of BIAL Biotech. “With the commitment and resources of BIAL we will be able to accelerate our novel clinical and research programs as targeted and personalized treatment approaches for genetically-defined PD patients.”

The safety, tolerability, and efficacy of LTI-291 has been investigated in a series of Phase 1 clinical trials. The first trials (NL6421 and NL6516) evaluated LTI-291 in healthy volunteers, finding that the concentration of LTI-291 in the brain was sufficient to double the activity of GCase, based on data from preclinical studies.

The researchers also investigated LTI-291 in people with Parkinson’s. A Phase 1b study (NL6574) with 40 Parkinson’s patients found biomarker evidence of increased GCase activity in participants given different doses (10, 30, or 60 mg/day) of LTI-291 compared with a placebo.

Further, a Phase 1 study (NL7061) split 14 Parkinson’s patients into three groups, in which participants received either a placebo, a 10 mg/kg dose of LTI-291, or a 60 mg/kg dose over 28 days. The study found that blood-brain flow, glucose uptake by brain cells, and functional connections between brain cells all were improved in treated patients.

BIAL will now rename the molecule BIA 28-6156/LTI-291, and aims to pursue its development as a treatment for this genetic factor that contributes to Parkinson’s disease.

“The compounds we’ve acquired are based on genetics, a new field of research for us,” Portela said.

“The lead asset, which now has the code name ‘BIA 28-6156/LTI-291’, has an innovative mechanism of action and presents the potential of being a first disease-modifying therapy for a genetic subset of Parkinson’s disease. It has successfully completed a Phase I trial program and should be ready to start Phase II studies in 2021,” he said. “We’re progressing from symptomatic treatment to an intervention in the mechanisms of the disease, which is very exciting for BIAL.”

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