Microphthalmia, Syndromic 3

Dive Brief:Roche is deepening its commitment to neuroscience, agreeing to a licensing deal with Ionis to develop two preclinical RNA medicines for Alzheimers and Huntingtons diseases, Ionis said Wednesday.Per deal terms, Roche is paying $60 million upfront for rights to the undisclosed drugs, and will assume responsibility for developing them once they are ready for human trials. Ionis could receive further payments if clinical, regulatory and commercial milestones for the drugs are met.Roche has invested heavily in Alzheimers drug development, advancing two antibody therapies into Phase 3 testing only to find they didnt slow disease progression. The Swiss drugmaker has fallen behind rivals Eisai, Biogen and Eli Lilly, which have benefited from recent clinical development successes.Dive Insight:Roche and Ionis have worked together for years. The two are currently collaborating on tominersen, a type of drug called an antisense oligonucleotide that blocks the expression of a protein mutated in Huntingtons disease. That drug is in a Phase 2 trial measuring biologic signs of disease as well as patient function.They also have partnered on an antisense drug Roche is testing in kidney disease and Ionis is studying as a treatment for an age-related eye disorder.In Alzheimers, Roche was among the early innovators to develop antibody drugs capable of blocking a protein called amyloid that accumulates in the brains of people with the disease. Roche hit clinical setbacks, as did Lilly, Biogen and Eisai.Now, however, Eisais Biogen-partnered drug Leqembi is approved in the U.S. and Lillys donanemab is under Food and Drug Administration review.Roche hasnt fully ended its anti-amyloid drug work yet, continuing tests of one called crenezumab in people who have an inherited form of Alzheimers. Its also still studying a formulation of the twice-failed drug gantenerumab thats designed to better reach into the brain.Other mid-stage assets include two antibody drugs that target another protein implicated in Alzheimers disease, called tau. The company recently disclosed Phase 1 data from a drug targeting another protein known as gamma secretase thats related to amyloid buildup.Huntingtons disease has proven nearly as difficult as Alzheimers to treat, with approved medicines so far only able to reduce involuntary movements. Ionis and Roche are among several companies seeking to help patients by blocking mutant proteins, although their drug tominersen has had setbacks, too.The Ionis deal signals Roche wants to keep stocking its pipeline with new approaches for treating Alzheimers and Huntingtons. Ionis demonstrated that it can successfully develop a drug for a degenerative neurological disorder when Biogen gained FDA approval for Qalsody, an Ionis-invented treatment for amyotrophic lateral sclerosis. '