Several pharma companies think that targeting a rogue protein known as alpha-synuclein could be the key to halting or reversing neurodegeneration in Parkinson’s disease – but has the industry learned from a string of failures in Alzheimer’s? In the space of a few weeks, Novartis AG and Sanofi AS have signed major deals for molecules targeting misfolded alpha-synuclein, the rogue protein thought to be the root cause of Parkinson’s disease.
LONDON – A pledge to “bottle” improvements made in setting up and running clinical trials during the pandemic has materialized in the formation of Protas Ltd., a nonprofit promising to stage large-scale phase III clinical trials in common diseases for less than one tenth the current cost. Protas will design and run randomized phase III studies of new and repurposed drugs in conditions including depression, dementia, heart, lung and respiratory conditions, arthritis and cancer.
Targeting the toxic alpha-synuclein protein found in the brains of people with Parkinson’s is one of the most promising approaches to treat the disease in the clinic – but getting any drug into the brain is a challenge. Sanofi SA has joined with ABL Bio Inc. to solve this problem, in-licensing ABL-301, a preclinical bispecific antibody that locks on to misfolded alpha-synuclein but also includes a molecular “shuttle” that allows it to penetrate the blood-brain barrier.
Targeting the toxic alpha-synuclein protein found in the brains of people with Parkinson’s is one of the most promising approaches to treat the disease in the clinic – but getting any drug into the brain is a challenge. Sanofi SA has joined with ABL Bio Inc. to solve this problem, in-licensing ABL-301, a preclinical bispecific antibody that locks on to misfolded alpha-synuclein but also includes a molecular “shuttle” that allows it to penetrate the blood-brain barrier.
Making good on ambitions to increase its say in the development of drug candidates from its AI-driven discovery platform, as well as the breadth of roles the system serves, Exscientia plc said Jan. 7 it will work with longtime partner Sanofi SA to develop up to 15 new small-molecule candidates for oncology and immunology indications. Sanofi will pay Exscientia $100 million up front and up to $5.2 billion in total milestones, plus tiered royalties, it said.
LONDON – The global pandemic has put the term mRNA on lips worldwide, but vaccines apart, the field of RNA therapeutics has failed to overcome the problem of delivery and is stuck at first base, targeting the liver. Newco Sixfold Bioscience Ltd. is the latest company working to end this constraint, applying a mixture of computation and advanced chemistry to design tissue-specific RNA delivery systems.
Sanofi SA is paying about $1 billion up front and pledging as much as $225 million in development milestone payments to acquire Amunix Pharmaceuticals Inc. in an arrangement that brings aboard several immuno-oncology platforms. South San Francisco-based Amunix’s lead candidate is the clinic-bound, masked T-cell engager (TCE) AMX-818, which targets HER2-expressing solid tumors. The candidate emerged from the company’s XPAT technology, designed with the longstanding XTEN and centered on a protein polymer in a strategy similar to pegylation, except with a polypeptide. XPAT stands for XTENylated, protease-activated TCEs.
Vaccine specialists Sanofi SA and Glaxosmithkline plc have been behind the curve throughout the pandemic, with upstarts such as Moderna Inc. and Biontech SE stealing the show with their ground-breaking mRNA technology. While their rivals were beginning to launch the first wave of vaccines early last year, Sanofi and GSK had to reformulate theirs after it failed to produce a strong enough immune response in older people. New results show that the shot, SP-0253, could play a role as a booster but there will be another delay after the companies struggled to recruit enough participants in a crucial phase III trial.
Two trials presented at the annual meeting of the American Society of Hematology (ASH) showed that treatment with the antithrombin inhibitor fitusiran reduced the bleeding rate in patients with severe hemophilia by roughly 90%, regardless of whether they had type A or B, and whether they had developed antibodies to recombinant clotting factors.
With the U.S. biosimilar pathway clearly developed, industry and the FDA are turning their attention to interchangeables in the third iteration of the biosimilar user fee agreement (BsUFA).
