EARLY ALZHEIMER'S DISEASE

Leqembi (lecanemab) has become the first and only approved treatment shown to reduce the rate of disease progression and to slow cognitive and functional decline in adults with Alzheimer’s disease. These results were demonstrated in the Phase 3Clarity AD clinical trial. Following its approval, The Centers for Medicare & Medicaid Services broadened coverage, in support of the drug.

MULTIPLE SCLEROSIS

Roche has announced that the Phase III OCARINA II trial for Ocrevus (ocrelizumab) has achieved its primary and secondary objectives. The trial evaluated thesubcutaneous (SC) injection of Ocrevus, administeredtwice a year in patients with relapsing forms of multiple sclerosis (RMS) or primary progressive MS (PPMS).Ocrevus only takes 10 minutes to administer. The results demonstrated that the SC injection was non-inferior to the intravenous infusion in terms of pharmacokineticsand showed comparable effectiveness in controllingbrain lesions on MRI scans. Ocrevus remains the first and only therapy approved for both RMS and PPMS, with over 300,000 patients treated globally.

COMPANION DIAGNOSTIC

Quanterix has launched "LucentAD" , a biomarker blood test, to assist in the evaluation of patients experiencing cognitive symptoms consistent with the early signs ofAlzheimer’s disease.

The test measures the concentration of a specificphosphorylated tau protein isoform (p-Tau 181) in bothplasma and cerebrospinal fluid. This isoform has shown a positive correlation with the presence of amyloid pathology in the brain, which is a key characteristic ofAlzheimer's disease.

MULTIPLE SCLEROSIS

The data demonstrated a 9-point improvement at Week96, previously reported to be the Minimally ImportantDifference (MID) for the Fatigue Impact Scale in an MS population. This is a significant improvement in physicaland cognitive fatigue for patients. Briumvi (ublituximab)is a novel monoclonal antibody that targets CD20expressing B-cells and is indicated for the treatment of adults with RMS.

SPINOCEREBELLAR ATAXIA

The NDA was supported by Biohaven's pivotal Phase 3BHV4157-206 trial, which demonstrated consistent treatment benefits observed in patients with genotype Spinocerebellar ataxia type 3 (SCA3). SCA3 is an ultrarare neurodegenerative disease characterized by progressive disability, frequent falls, loss of ambulation,speech and swallowing difficulties, and premature death. It is the most common subtype of spinocerebellarataxia worldwide, affecting an estimated 10,600 people in North America, the European Union, and Japan.

RETT SYNDROME

The company published its pivotal Phase 3 results,evaluating Daybue (trofinetide), for the treatment of girlsand women 5 - 20 years of age with Rett Syndrome, in Nature Medicine. The results showed statisticallysignificant improvement in patients taking Daybue,compared to those taking placebo in both primary and secondary efficacy outcomes. These results were previously leveraged for the FDA approval of Daybue in March 2023, making Daybue the first and only approved therapy for Rett syndrome. Rett syndrome is categorizedas a rare, neurodevelopmental disorder, which affects6,000 to 9,000 patients in the US. Additionally, the company received a Rare Pediatric Disease PriorityReview Voucher with the FDA approval.