Beta-site APP-cleaving enzyme 1 (BACE1) inhibitors have a long history of failure in patients with Alzheimer’s disease. Clinical development of verubecestat, elenbecestat, lanabecestat, umibecestat, atabecestat and LY-3202626 were all discontinued. The drugs largely showed that inhibiting BACE1 reduced amyloid beta (AB) in both cerebrospinal fluid and plasma, reduced AB plaques on PET scans and reduced phosphorylated tau. Unfortunately, patients had early cognitive worsening, and there were signs of lowering of brain volume and increases in psychiatric adverse events.

As fellow gene editing firm Crispr Therapeutics AG hosted an innovation day in which it confirmed plans for regulatory filings by year-end for an ex vivo gene editing therapy in sickle cell disease and beta-thalassemia, Precision Biosciences Inc. announced plans to develop an in vivo gene editing approach through a collaboration with Novartis AG that brings Precision an initial $75 million with up to $1.4 billion in potential milestones.

Pharma companies agree that data is king, since after all, their drugs get approved based on the quality of their data.

There are 40 years of history behind the development of phosphoinositide 3-kinase (PI3K) inhibitors, Rebecca Dent told her audience at ESMO Breast Cancer 2022. And there have been success stories. There are five FDA-approved PI3K inhibitors in several cancer types, and in April, the FDA approved Vijoice (alpelisib; Novartis AG) for PIK3CA-related overgrowth spectrum, a rare disorder resulting from germline mutations of PIK3CA.

There are 40 years of history behind the development of phosphoinositide 3-kinase (PI3K) inhibitors, Rebecca Dent told her audience at ESMO Breast Cancer 2022. And there have been success stories. There are five FDA-approved PI3K inhibitors in several cancer types, and in April, the FDA approved Vijoice (alpelisib; Novartis AG) for PIK3CA-related overgrowth spectrum, a rare disorder resulting from germline mutations of PIK3CA.