CNS

The FDA and Sarepta have had a controversial history with the accelerated approvals of the firm’s earlier drugs for Duchenne muscular dystrophy – but the company’s investigational gene therapy, SRP-9001, now under a priority review, could be a genuine breakthrough.

In this week's podcast edition of Five Must-Know Things: safety debate as more lecanemab data reported; amyloid discussions continue at Alzheimer’s meeting; China COVID unrest affect country’s biotech sector; and Merck’s CMO talks about post-Keytruda plans.

The company believes the Phase III Clarity AD results offer a high level of evidence that may convince the Centers for Medicare and Medicaid Services to change its stance on anti-amyloid antibodies for lecanemab. 

The firms unveiled positive Phase III data from their respective assets for treating the agitation seen in Alzheimer’s disease with Lundbeck’s candidate set to become first-to-market for the challenging behavioral symptom.

While Phase III results for Eisai and Biogen’s lecanemab show amyloid clearance provides benefit and Roche’s Phase III gantenerumab data are supportive, more data and certainty of access to treatment are needed.

AC Immune has seen two big disappointments in its Alzheimer’s pipeline this year, but its partner Johnson & Johnson has put its faith in a novel vaccine-like approach to targeting the disease.

Lilly’s donanemab showed faster, deeper amyloid clearance versus Biogen/Eisai’s Aduhelm and Roche’s failed Phase III gantenerumab studies showed the importance of deep, rapid removal.

Alzheimer’s patients treated with the amyloid protofibril-clearing antibody had slower disease progression compared to placebo, with differences increasing over time. Safety concerns, however, are growing. 

Roche’s global head of neurodegeneration, Rachelle Doody, talks to Scrip about the failure of gantenerumab and its next-generation ‘brain shuttle’ technology for Alzheimer’s disease.

In this week's podcast edition of Five Must-Know Things: a preview of the CTAD Alzheimer’s meeting; US myeloma drug withdrawal puts pressure on GSK; UniQure/CSL's gene therapy sets new US price record; new leadership at Teva; and Merck’s Frazier cautions against profit maximization.

The Japanese and German firms agree to establish a joint company to handle 13 mature brands from Kyowa Kirin's portfolio, mainly focused on pain management and including Abstral and PecFent. Grünenthal will own 51% and plans to fully buy out the operation in early 2026.

The controversial nasal spray has improved remission rates over an atypical antipsychotic in patients with a challenging form of depression, in Phase III data which could help boost its uninspiring market performance to date.