Roche Contemplates Potential Fast-Track Approval for New Alzheimer's Medication

Roche Contemplates Potential Fast-Track Approval for New Alzheimer’s Medication

Roche Contemplates Potential Fast-Track Approval for New Alzheimer's Medication

Dive Brief:

  • Roche is considering expedited approval for its experimental Alzheimer’s drug, trontinemab, based on promising results showing significant reductions in a protein associated with cognitive decline, as indicated by company executives in a recent investor presentation.
  • The FDA has previously shown a willingness to expedite approval processes for Alzheimer’s treatments that can effectively decrease levels of amyloid protein in patients’ brains. Roche intends to leverage this precedent to pursue a streamlined approval process, aiming to bring trontinemab to market before the final data from a Phase 3 study comes in, as noted by Hanno Svoboda, the product’s lifecycle head.
  • Two Alzheimer’s medications, Leqembi from Eisai and Biogen and the withdrawn Aduhelm, initially obtained approvals based on their capacity to affect amyloid levels. Conversely, the FDA denied a similar request from Eli Lilly before later approving Kisunla. Currently, trontinemab is undergoing Phase 2 studies, and Roche recently published initial data indicating its potential for more rapid amyloid clearance compared to Kisunla.

Dive Insight:

Distinct from its predecessors, trontinemab is engineered for improved penetration of the blood-brain barrier, which could result in enhanced efficacy and more accurate dosing at lower quantities.

This hypothesis still awaits validation through extensive late-stage trials typically required for approval. However, results from a recent Phase 2 study indicate promising potential for the drug.

In the highest two dosage groups assessed thus far, a significant number of participants showed amyloid clearance from their brains after a 28-week treatment period. For context, 17% of participants in Kisunla’s Phase 3 study achieved a similar outcome at 24 weeks.

The rapid effects noted may enable Roche to explore a “low-frequency, patient-friendly” maintenance dosing protocol to prevent amyloid levels from rising again, according to Svoboda.

Roche is now advancing to an “expansion” phase in its mid-stage trial, planning to double the enrolment to 120 participants, with some receiving a placebo.

Thus far, the company has only gathered safety data from this portion of the study. Roche reported one participant who received treatment tragically died from a brain bleed but clarified that the individual had superficial siderosis, a condition elevating her risk, and subsequent adjustments to enrollment criteria have been made.

Nevertheless, findings suggest that treatment with trontinemab resulted in lower incidences of an imaging abnormality known as ARIA, associated with brain swelling. Roche claims that under 10% of trial participants undergoing trontinemab treatment have experienced ARIA to date, contrasting with over 20% of Kisunla recipients in its Phase 3 trial, and above 10% for Leqembi.

Trontinemab has demonstrated higher rates of infusion-related reactions compared to Leqembi and Kisunla, though Roche has managed to reduce these reactions to below 40% in the two highest doses examined.

This week, Lilly also unveiled data suggesting that adjustments to the dosing strategy for Kisunla could potentially lower ARIA rates.