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Phase II Success Positions Ra For Potential Blockbuster Opportunity In Myasthenia Gravis

 
• By Joseph Haas

Ra hopes to launch a Phase III program for zilucoplan next year, with a goal of bringing an alternative therapy for MG patients into competition with Alexion’s Soliris.

Medic staff at work in the laboratory. Background

Ra Pharmaceuticals Inc.’s successful top-line Phase IIb data with complement 5 (C5) inhibitor zilucoplan in generalized myasthenia gravis (gMG), unveiled on Dec. 10, gives the biotech an opportunity toward blockbuster sales with a self-administered therapy that offers several points of differentiation compared to Alexion Pharmaceuticals Inc.’s Soliris (eculizumab).

One of the highest-priced drugs in the world, Soliris became the only drug approved specifically to treat myasthenia gravis (MG) in the US in late 2017, but the drug used primarily in its initial indication of paroxysmal nocturnal hemoglobinuria (PNH) is limited to last-line therapy in MG. (Also see "Alexion Seizes On New Soliris Indication, Business Development For Growth" - Scrip, 26 October, 2017

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• By Kevin Grogan

The therapy has been developed for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C.

Lilly Builds Obesity Momentum With New SURMOUNT Data Besting Novo Nordisk

 
• By Alaric DeArment

The presentation and publication of the Phase IIIb study showing greater efficacy for Zepbound over Wegovy come at a time when competition has been heating up between the two obesity medications.

Insilico Fast-Tracks First AI-Designed TNIK Inhibitor For IPF

 
• By Dexter Jie Yan

InSilico will leapfrog Phase IIb to progress its AI-generated candidate for idiopathic pulmonary fibrosis directly into a Phase III trial in China, as it prepares for an IPO in Hong Kong.

Pipeline Watch: Six Approvals And Thirteen Phase III Readouts

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

More from R&D

Which Firms Are Most Exposed To Prasad’s Possible Accelerated Approvals Clampdown?

 
• By Andrew McConaghie

Vaccine and accelerated approvals are expected to face greater scrutiny under new CBER leader Vinay Prasad, but he has promised “no light switch change” to policy, giving some reassurance to companies whose share prices have been battered.

Pipeline Watch: Six Approvals And Thirteen Phase III Readouts

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Viatris Will File ‘Fast-Acting’ Meloxicam For FDA Approval This Year

 
• By Joseph Haas

Seven years after acquiring it, Viatris will seek approval of a meloxicam formulation for acute pain backed by data from two Phase III studies in post-surgical pain settings.