ION541 / Ionis, Biogen - LARVOL DELTA

ALSpire: A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation (clinicaltrials.gov) - P1/2 | N=99 | Terminated | Sponsor: Biogen | Trial completion date: Jul 2026 ➔ Aug 2024 | Active, not recruiting ➔ Terminated | Trial primary completion date: Jul 2026 ➔ Aug 2024; Sponsor's decision

Trial completion date • Trial primary completion date • Trial termination • Amyotrophic Lateral Sclerosis • CNS Disorders

Biogen and Ionis Announce Topline Phase 1/2 Study Results of Investigational Drug in Amyotrophic Lateral Sclerosis (GlobeNewswire) - P1/2 | N=99 | ALSpire (NCT04494256) | Sponsor: Biogen | "Biogen Inc...announced the decision to terminate development of BIIB105 (ION541) an investigational antisense oligonucleotide (ASO) for amyotrophic lateral sclerosis (ALS) based on topline results from the Phase 1/2 ALSpire study. BIIB105 was designed to reduce expression of ataxin-2 (ATXN2) protein...However, over the 6-month placebo-controlled period, treatment with BIIB105 did not result in a reduction in levels of plasma neurofilament light chain (NfL), a marker of neurodegeneration and neuronal damage. Additionally, BIIB105 did not demonstrate an impact on clinical outcome measures of function, breathing, and strength...'Ionis continues to be committed to the ALS community and is advancing our Phase 3 ulefnersen program.'....The companies will present the BIIB105 Phase 1/2 data at the upcoming European Network to Cure ALS..."

Clinical • Discontinued • P1/2 data • Amyotrophic Lateral Sclerosis • CNS Disorders

Ionis poised for continued momentum in 2024 with product launches and key advances in robust pipeline of investigational medicines for serious diseases (Yahoo Finance) - "Continued progress with key partnered programs, including: QALSODY: EU SOD1-ALS CHMP and approval decision (Biogen)...ION541: Phase 2 results in ALS (Biogen)..."

European regulatory • P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders • Rare Diseases

ALSpire: A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation (clinicaltrials.gov) - P1/2 | N=99 | Active, not recruiting | Sponsor: Biogen | Recruiting ➔ Active, not recruiting

Enrollment closed • Amyotrophic Lateral Sclerosis • CNS Disorders • NEFL • Plasma NfL

"Good work @biogen Now we need #BIIB105" (@JohnGot24456605)

"BIIB 105. AGAINST ATAXIN 2 ASAP! 6 years of waiting till 2026 from 2020 is to much" (@JohnGot24456605)

"Hi. How is the BIIB105 study going? Is there any improvement? Can you tell me please?" (@rmeysak62378386)

"What's about BIIB105? Can you give us a short update" (@JohnGot24456605)

Trial of BIIB105 Enrolling Patients With or Without ATXN2 Mutations (ALS News Today) - "A Phase 1/2 clinical trial is investigating the long-term safety and tolerability of BIIB105, Biogen‘s investigational treatment targeting the ataxin-2 protein, in people with amyotrophic lateral sclerosis (ALS). Called ALSpire (NCT04494256), the trial is currently recruiting adults with ALS at 12 sites in the U.S., Canada, and Europe. Additional trial sites — two in the U.S. and one in Spain — are planned to open early next year....ALSpire is due to conclude in July 2026."

Trial completion date • Trial status • Amyotrophic Lateral Sclerosis • CNS Disorders

"Huhu, i ask for many time, what's about BIIB105" (@JohnGot24456605)

A Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis With or Without Poly-cytosine-adenine-guanine (CAG) Expansion in the Ataxin-2 Gene (clinicaltrials.gov) - P1/2 | N=108 | Recruiting | Sponsor: Biogen | Phase classification: P1 ➔ P1/2 | N=70 ➔ 108

Enrollment change • Phase classification • Amyotrophic Lateral Sclerosis • CNS Disorders • NEFL • Plasma NfL

"What's about tofersen and biib105?" (@JohnGot24456605)

"BIOGEN what's about BIIB105?" (@JohnGot24456605)

"What's about BIIB105?" (@JohnGot24456605)

A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB105 in Participants With Amyotrophic Lateral Sclerosis With or Without Poly-cytosine-adenine-guanine (CAG) Expansion in the Ataxin-2 Gene (clinicaltrials.gov) - P1 | N=70 | Recruiting | Sponsor: Biogen | Trial completion date: Dec 2024 ➔ Jul 2026 | Trial primary completion date: Dec 2024 ➔ Jul 2026

Trial completion date • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB105 in Participants With Amyotrophic Lateral Sclerosis With or Without Poly-cytosine-adenine-guanine (CAG) Expansion in the Ataxin-2 Gene (clinicaltrials.gov) - P1; N=70; Recruiting; Sponsor: Biogen; Trial completion date: Feb 2023 ➔ Dec 2024; Trial primary completion date: Feb 2023 ➔ Dec 2024

Clinical • Trial completion date • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

"Hey Dean, can you tell us how are you today? How is your experience with biib 105? 🙏" (@JohnGot24456605)

"$ions collected $10m from $biib for advancing ION541 (BIIB105)-ATXN2 ALS to ph2!" (@auditor112017)

Ionis Pharmaceuticals, Inc. (IONS) Q3 2020 Earnings Call Transcript (The Motley Fool) - “The Phase 1/2 study of ION541, our first medicine designed to address nearly all forms of ALS regardless of family history, is well under way. The Phase 3 study of Tofersen in patients with SOD1-ALS is progressing with data expected in the second half of next year.…Biogen plans to initiate a study of Tofersen in pre-symptomatic SOD1-ALS patients with the potential to delay or prevent disease onset. The Phase 1/2 study of IONIS-C9Rx in patients with C9-ALS remains on track for data next year. And ION363, our first Ionis-owned ALS medicine for the treatment of ALS patients with mutations in the FUS gene, remains on track to enter a registrational study next year.”

New trial • P1/2 data • P3 data • Trial status • Amyotrophic Lateral Sclerosis • CNS Disorders

Ionis' third novel antisense medicine for ALS, its first designed to treat a broad ALS population, begins clinical trial (PRNewswire) - “Ionis Pharmaceuticals…announced that the first patients have been dosed with ION541 (also known as BIIB105), an investigational antisense medicine being developed as a potential therapy to treat most forms of amyotrophic lateral sclerosis (ALS) regardless of family history.”

Enrollment status • Amyotrophic Lateral Sclerosis • CNS Disorders

Ionis Is A Long On Neurological Medicines Pipeline (SeekingAlpha) - “Tofersen: Phase 3 VALOR study underway (data expected 2021). IONIS-C9Rx: Phase 1/2 study ongoing in C9- familial ALS (data expected 2021). ION363, Ionis-owned targeting FUS on track to initiate a Phase 1/2 study in FUS-ALS in late 2020/early 2021. ION541 targeting ATXN2 in sporadic ALS on track to initiate a Phase 1/2 study in 2H20.”

New P1/2 trial • P1/2 data • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders