Roche is paying up to $1.27bn to gain the global rights to Alector’s Parkinson’s disease candidate, giving the drugmaker access to a drug that aims to overcome delivery limitations that have plagued the disease space in recent years.
Roche is paying $100m upfront for Alector’s AL050, a brain-penetrant engineered glucocerebrosidase (GCase) enzyme replacement therapy. The treatment is delivered into the brain using the biotech’s blood-brain barrier platform, making the preclinical drug a promising candidate for neurodegenerative disease. Alector is also eligible to receive additional payments of up to $1.17bn upon the achievement of development, regulatory, and commercial milestones.
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Alector’s AL050 is designed to address GCase deficiency, a driver of neurodegeneration in Parkinson’s disease that is most pronounced in – but not limited to – carriers of GBA1 mutations. The enzyme is responsible for breaking down waste within cells and recycling cellular components. In Parkinson’s disease, reduced GCase activity is linked to the buildup of abnormal proteins in brain cells.
Alector’s approach combines an engineered GCase with the biotech’s blood-brain barrier delivery platform called Alector Brain Carrier (ABC). Delivered into the brain, the enzyme is intended to break down glucosylsphingosine and glucosylceramide, lipids that accumulate in neurons and other brain cells upon GCase deficiency and contribute to neurodegeneration, with the aim of reducing cellular dysfunction and slowing disease progression. While therapies for neurodegenerative diseases have advanced over the past decade, effective delivery to the brain remains a hurdle.
Under the collaboration, Roche’s Genentech will be responsible for development, regulatory, manufacturing, and commercialisation across all indications. Alector retains ownership of ABC and can utilise the platform across other products in its pipeline.
Shares in the US-listed biotech surged by 50% at market open to $2.73 on 5 October compared to $1.81 at market close on 2 October. Share price settled at $1.99 by the end of the day, making a day-on-day rise of 9.9%.
Alector’s clinical pipeline includes an α-Syn-siRNA therapy for Parkinson’s disease and an NLRP3-siRNA drug in both Parkinson’s disease and Alzheimer’s disease. AL050 is one of three preclinical candidates in the biotech’s pipeline, which together span several neuro indications.
The deal with Alector marks one of Roche’s most high-profile Parkinson’s deals in the past decade. The big pharma company has inked several collaborations with Shape Therapeutics in recent years, though financial terms were not disclosed. Roche does not currently have an approved disease-modifying or curative therapy for Parkinson’s disease. The drugmaker’s main pipeline candidate is prasinezumab, a monoclonal antibody (mAb) gained via a $600m licensing agreement with Prothena in 2013. While prasinezumab has substantial Phase II evidence, the deal for AL050 means Roche’s pipeline includes a candidate with enhanced brain delivery.
Alector’s CEO, Arnon Rosenthal, said: “The challenge in treating GCase deficiency is twofold: engineering an enzyme with optimal activity and durability and successfully delivering it to the brain. AL050 addresses this by pairing an engineered GCase enzyme with our proprietary ABC technology designed to cross the blood-brain barrier.”
