BARROW NEUROLOGICAL INSTITUTE

Anavex Life Sciences reported Q4 and FY 2024 financials, highlighting progress in Alzheimer's treatment with blarcamesine. The company anticipates a 4-year cash runway and reported a Q4 net loss of $11.6M.

Neuralink receives Health Canada approval for CAN-PRIME trial to assess safety and functionality of its implantable brain-computer interface (BCI) device, targeting individuals with ALS or cervical spinal cord injury at UHN's Toronto Western Hospital.

Blood tests for Alzheimer’s are ready, with many plasma p-tau217 assays meeting triage criteria and several vendors filing for FDA approval. CMS may reimburse, but rates are uncertain. Vendors like Fujirebio and Roche have filed for IVD approval, and leaders in AD drug development await regulatory approval. The CEOi panel recommends tests with 90% sensitivity and 85% specificity, prompting vendors to optimize their tests. The p-tau217/Aβ1-42 ratio outperforms p-tau217 alone in identifying amyloid-positive individuals, with fewer indeterminate results. Post-approval, integration into clinical practice and appropriate-use recommendations are crucial.

Toronto Western Hospital, part of Canada’s University Health Network, will be the first non-U.S. site for a Neuralink Corp. device trial. Neuralink, Elon Musk’s brain-implant company, received approval from Health Canada for clinical trials in Canada, with recruitment now open for patients with Quadriplegia due to ALS or spinal cord injury.

Toronto Western Hospital to be first non-U.S. site for Neuralink's brain-implant device trial in Canada, approved by Health Canada. Neuralink seeks patients with Quadriplegia due to ALS or spinal cord injury. First product aims to control external devices through thoughts.

Lecanemab use in the U.S. and Japan shows ARIA rates similar to trials despite broader patient populations. Japan's ARIA rates are half of the U.S., mirroring Phase 3 trial results. Lecanemab is approved in several countries but rejected by the EMA and Australia. The U.K. restricts use to non-APOE4 homozygotes, and the NHS does not cover costs. Other countries may follow the U.K.'s example. Lecanemab use is growing in Japan but faces hurdles like limited infusion sites and physician numbers.

Since Leqembi's approval in 2023, neurologists have learned: 1) it takes 5 months on average to start treatment; 2) Black and Hispanic Americans are underrepresented; 3) most U.S. infusions occur in urban centers; 4) Japan treats more patients with fewer ARIA cases; 5) blood tests aren't yet adequate for confirming Alzheimer's biomarkers.

Blarcamesine, an orally bioavailable small molecule activator of the sigma-1 receptor (SIGMAR1), demonstrated slowed clinical progression in Alzheimer disease (AD) patients without the SIGMAR1 rs1800866 gene variant, suggesting greater benefit in these individuals. The phase 2/3 trial, AD-004, involved 508 early-stage AD patients, with 338 receiving blarcamesine and 170 receiving placebo. Results presented at the 2024 CTAD conference showed that wild-type (WT) SIGMAR1 gene carriers experienced significant cognitive benefits compared to the full intent-to-treat population, supporting SIGMAR1 activation as a key mechanism. Blarcamesine also showed reduced brain atrophy and favorable safety profiles, positioning it for potential regulatory submission in Europe.

Eisai and Biogen presented lecanemab-irmb findings at CTAD, showing benefits of continued treatment for early Alzheimer's disease, including reduced disease progression and improved cognitive function. No new safety issues were reported, and a new method to quantify protofibrils in cerebrospinal fluid was developed, suggesting protofibrils' role in neurodegeneration. Long-term U.S. clinical studies indicated patient satisfaction with lecanemab, and the AHEAD 3-45 study, focusing on preclinical AD, completed enrollment in October 2024. Professor Lars Lannfelt received the CTAD Lifetime Achievement Award for his contributions to AD research.