Qalsody

(Tofersen)

ApprovedRNA therapy

Qalsody is a rna therapy that works as a SOD1 Antisense Oligonucleotide.[2] It is being used across 3 indications, with its most advanced program in Approved.[1]

Qalsody is one of 77 aso programs targeting ALS (Amyotrophic Lateral Sclerosis). It is currently in Approved.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

ALS (amyotrophic Lateral Sclerosis)Recruiting

Amyotrophic Lateral SclerosisActive, not recruiting

Alzheimer's DiseaseRecruiting

ALS (amyotrophic Lateral Sclerosis)Phase 2

Recruiting

Amyotrophic Lateral SclerosisApproved

Active, not recruiting

Alzheimer's DiseasePhase 3

Recruiting

Competition

Competitive Landscape

59 competitors in Alzheimer's Disease

#DrugCompanyPhaseTrials

Aricept

Eisai

Approved

31 2

Leqembi

Eisai

Phase 3

7 3

Nuplazid

AACADIA Pharmaceuticals Inc.

Ozempic

3 5

Qalsody

Biogen

Approved

1 6

Namenda

AbbVie

Phase 2

2 7

Briviact

UCB

Phase 2

1 8

Niacor

AbbVie

Phase 2

1 9

Leukine

Sanofi

Phase 2

1 10

Sprycel

KKaruna Therapeutics, Inc., a Bristol Myers Squibb company

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2027

ApprovedActive, not recruiting

Amyotrophic Lateral Sclerosis

NCT07223723

Q2 2028

Phase 2Recruiting

ALS (amyotrophic Lateral Sclerosis)

NCT07294144

Q4 2028

Phase 3Recruiting

Alzheimer's Disease

NCT04856982

Q4 2033

Not yet recruiting

Amyotrophic Lateral Sclerosis

NCT07259980

Clinical

Trials by Indication[1]

Amyotrophic Lateral Sclerosis3 trials

An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

Completed· Biogen logo

Biogen·Phase 3·NCT02623699·176 enrolled

A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)

Not yet recruiting· Biogen logo

Biogen··NCT07259980·125 enrolled

A Multicenter, Open-Label, Postmarketing Surveillance Study of Tofersen (BIIB067) in Adults With Amyotrophic Lateral Sclerosis (ALS) Associated With a Mutation in the Superoxide Dismutase 1 (SOD1) Gene in China

Active, not recruiting· Biogen logo

Biogen·Approved·NCT07223723·12 enrolled

ART1 trial

Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

Approved for marketing· Biogen logo

Biogen··NCT04972487

Adult1 trial

A Study to Evaluate Safety, Tolerability, and Distribution of a Microdose of Radiolabeled BIIB067 (99mTc-MAG3-BIIB067) Co-administered With BIIB067 (Tofersen) to Healthy Adults

Completed· Biogen logo

Biogen·Phase 1·NCT03764488·8 enrolled

ALS (Amyotrophic Lateral Sclerosis)1 trial

A Study to Evaluate the Biological Effect of Tofersen in Adults With Amyotrophic Lateral Sclerosis Without Mutations in SOD1

Recruiting·WWashington University School of Medicine·Phase 2·NCT07294144·30 enrolled

Alzheimer's Disease1 trial

TRAILBLAZER-ALZ 3: Donanemab Prevention

Recruiting· Eli Lilly logo

Eli Lilly·Phase 3·NCT04856982·3,300 enrolled

1 more indication

Unspecified1 trial

Long-Term Evaluation of BIIB067 (Tofersen)

Completed· Biogen logo

Biogen·Phase 3·NCT03070119·139 enrolled

Regulatory

FDA Approval Timeline[4]

Apr 25, 2023

QALSODY

NDA NDA215887

Apr 25, 2023

QALSODY

NDA NDA215887

Sources

References

[1]
ClinicalTrials.gov — 8 trial records for Tofersen. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Tofersen compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Tofersen. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA215887). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Qalsody. U.S. Food & Drug Administration.
[6]
PubMed — 1 publication indexed for Tofersen. U.S. National Library of Medicine.
[7]
SEC EDGAR — Biogen company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: SOD1 Antisense Oligonucleotide[2]
Modality: RNA therapy[2]
Highest Phase: Approved[1]
Company: Biogen

Patent Landscape[5]

Patent Cliff

Apr 1, 2035

Earliest patent expiration — generic entry possible after this date

Patent #Expires

10968453Apr 1, 2035

10669546Apr 1, 2035

10385341Apr 1, 2035

Key Publications[6]

Tofersen for SOD1-ALS: VALOR Phase 3 Trial

N Engl J MedSep 22, 2022DOI: 10.1056/NEJMoa2204399

Qalsody

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q2 2027

Q2 2028

Q4 2028

Q4 2033

Trials by Indication[1]

Amyotrophic Lateral Sclerosis3 trials

ART1 trial

Adult1 trial

ALS (Amyotrophic Lateral Sclerosis)1 trial

Alzheimer's Disease1 trial

Unspecified1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

Key Publications[6]

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