BIIB101 / Biogen, Ionis - LARVOL DELTA

Trimmed pipeline (Firstwordpharma Press Release) - "Alongside its fourth-quarter results, Biogen said that as part of ongoing pipeline prioritisation efforts, it has discontinued development of: BIIB113 in early Alzheimer's disease; BIIB094 in early Parkinson's disease; BIIB101 in multiple system atrophy; and BIIB143 (cemdomespib) in diabetic peripheral neuropathic pain."

Discontinued • Alzheimer's Disease • Diabetic Neuropathy • Multiple System Atrophy • Parkinson's Disease • Peripheral Neuropathic Pain

Ionis enters new chapter in 2025 as commercial-stage biotech with multiple independent product launches anticipated and continued late-stage pipeline momentum (PRNewswire) - "Continue progress with WAINUA (eplontersen), Ionis' first U.S. co-commercialized medicine in collaboration with AstraZeneca...Continuing the fully enrolled, landmark CARDIO-TTRansform trial in ATTR cardiomyopathy, with data expected in the second half of 2026...Phase 2 data for ION464 (SNCA) in multiple system atrophy."

P2 data • P3 data • Amyloidosis • Multiple System Atrophy

SNCA Antisense Oligonucleotide for Multiple System Atrophy: HORIZON Trial Update (MDS Congress 2024) - "ION464 is an SNCA RNA targeting ASO being developed as a potential disease-modifying treatment for MSA. To date, ION464 has been safe and well tolerated in MSA-affected individuals enrolled in the HORIZON Study."

Ataxia • CNS Disorders • Multiple System Atrophy • Musculoskeletal Pain • Pain • Parkinson's Disease • SNCA

Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON) (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Ionis Pharmaceuticals, Inc. | Trial completion date: Dec 2025 ➔ Sep 2027 | Trial primary completion date: Nov 2023 ➔ Sep 2027

Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Multiple System Atrophy

Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON) (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Ionis Pharmaceuticals, Inc. | Active, not recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Movement Disorders • Multiple System Atrophy

Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON) (clinicaltrials.gov) - P1 | N=40 | Active, not recruiting | Sponsor: Ionis Pharmaceuticals, Inc. | Trial completion date: Jul 2022 ➔ Dec 2025 | Trial primary completion date: Jul 2022 ➔ Nov 2023

Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Multiple System Atrophy

"$ions $biib has two phase 2 CNS drugs to treat Parkinsons: ION859 (LRRK2) and ION464 (SNCA)!" (@auditor112017)

P2 data • CNS Disorders • Movement Disorders • Parkinson's Disease

HORIZON: Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB101 Administered to Adults With Multiple System Atrophy (clinicaltrials.gov) - P1; N=34; Active, not recruiting; Sponsor: Biogen; Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Movement Disorders • Multiple System Atrophy

HORIZON: Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB101 Administered to Adults With Multiple System Atrophy (clinicaltrials.gov) - P1; N=34; Recruiting; Sponsor: Biogen; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • CNS Disorders • Movement Disorders • Multiple System Atrophy