BIIB-101

Ionis Pharmaceuticals Advances Neurological and Cardiovascular Therapies with New Clinical Trials and FDA Approvals

4 months ago

Ionis Pharmaceuticals is set to enter a new chapter in 2025, focusing on advancing treatments for serious neurological diseases and cardiovascular conditions. The company has outlined plans for Phase 3 trials for ION582 for Angelman syndrome, zilganersen for Alexander disease, and ION464 for multiple system atrophy. Additionally, Ionis has received FDA approval for TRYNGOLZA™ (olezarsen) to reduce triglycerides in adults with familial chylomicronemia syndrome and for WAINUA™ (eplontersen) for treating polyneuropathy of hereditary transthyretin-mediated amyloidosis.

UCB's Minzasolmin Fails Phase 2 ORCHESTRA Trial for Early Parkinson's Disease

5 months ago

• UCB's minzasolmin, an oral alpha-synuclein misfolding inhibitor, did not meet primary or secondary endpoints in the Phase 2 ORCHESTRA trial for early Parkinson's. • The ORCHESTRA study enrolled over 450 patients to assess minzasolmin's efficacy, safety, tolerability, and pharmacokinetics over 12-18 months. • UCB will terminate the extension phase of the minzasolmin program and shift focus to glovadalen (UCB0022) and UCB7583 for Parkinson's treatment. • Analysis of disease biomarker data from the ORCHESTRA study is ongoing, with findings to be submitted for publication in a peer-reviewed journal.

ION464 Shows Promise in MSA, OnabotulinumtoxinA Explored for Essential Tremor, Dietary Challenges in Parkinson's

8 months ago

• ION464 (BIIB101), an antisense medicine targeting alpha-synuclein, demonstrates safety and tolerability in a Phase 1/2 trial for multiple system atrophy (MSA) patients. • A Phase 2b trial (ELATE) is underway to evaluate the safety and efficacy of onabotulinumtoxinA for upper limb essential tremor (ULET), potentially offering a better risk-benefit profile than oral therapies. • A crossover study reveals that while Parkinson's disease (PD) patients embrace Mediterranean diets, combining them with ketogenic interventions faces challenges due to restrictiveness and gastrointestinal side effects.

Ionis Pharmaceuticals Advances Neurological and Cardiovascular Therapies with New Clinical Trials and FDA Approvals

UCB's Minzasolmin Fails Phase 2 ORCHESTRA Trial for Early Parkinson's Disease

ION464 Shows Promise in MSA, OnabotulinumtoxinA Explored for Essential Tremor, Dietary Challenges in Parkinson's

Drug Development Updates