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JPM: Ionis Looks Past Kynamro To Profitability

 
• By Lisa LaMotta

Despite significant progress on its late-stage programs and the promise of profitability, Ionis Pharmaceuticals Inc. can't seem to please investors who can only focus on the a failed partnership.

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More from Musculoskeletal

Wave Aspires To Accelerated Approval In DMD With 48-Week Data

 
• By Joseph Haas

With its exon 53-skipping candidate already showing promise in 24-week data, Wave now has 48-week data showing improvements in muscle health and functional outcomes.

Regenxbio Rises After Duchenne Update And Sarepta Safety Scare

 
• By Andrew McConaghie

Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.

Sarepta Shares Plummet On News Of Elevidys Patient Death

 
• By Joseph Haas

The gene therapy for Duchenne muscular dystrophy passed the blockbuster sales threshold in 2024. Analysts said the drug’s overall risk/benefit profile still appears solid.

Scrip Asks... What Does 2025 Hold For Biopharma? Part 6: Therapeutic Area Advances

 
• By Eleanor Malone

Over 140 biopharma leaders share their views on developments to watch for in key therapeutic areas this year. Advances in multiple scientific fields are opening up new avenues for treatment.

More from Therapy Areas

GSK Chief Optimistic About BD Despite Volatile Environment

 
• By Kevin Grogan

Emma Walmsley tells Scrip that the "biotech market is under a certain degree of pressure," so reasonably priced deals are available.

In Brief: AbbVie’s Rinvoq Secures US Approval For Giant Cell Arteritis

 
• By Sushmita Panda

JAK inhibitor gains ninth indication in US after a strong performance in the first quarter.

CSPC’s Global-First Monospecific ADC Promising In Advanced NSCLC

 
• By Xu Hu

CSPC’s SYS6010, the first monospecific EGFR-targeting antibody-drug conjugate in the clinic for advanced NSCLC, has shown Phase I promise, but could face close competition with another China-originated bispecific.