Biogen takes another shot at spinal muscular atrophy

The fact that Biogen has paid Ionis $60m to license another project for spinal muscular atrophy shows that the group has not given up on the disease. But it is also confirmation, if this were needed, that the good times are over for its existing therapy, Spinraza. Like Spinraza, Biogen’s new preclinical project BIIB115 is an antisense oligonucleotide designed to increase production of survival motor neuron (SMN) protein, with the potential for extended dosing intervals. A look at the SMA pipeline could help explain why Biogen still sees room for growth in SMA, despite competition from Novartis’s Zolgensma and Roche’s Evrysdi: according to Evaluate Pharma only two projects are in active clinical development. Both are myostatin inhibitors that appear to hold most promise for use in combination with Spinraza or Evrysdi, and one of them is in Biogen’s hands. Meanwhile, Novartis discontinued development of its oral SMN splicing modulator branaplam in SMA last July, saying it did not believe the project would be sufficiently differentiated from existing therapies; development continues in Huntington’s disease.

Projects in active clinical development for SMA
Projecct Company Description Note
Phase 3
Apitegromab (SRK-015) Scholar Rock Myostatin inhibitor (anti-promyostatin monoclonal antibody) Sapphire (NCT05156320), combo with Spinraza/Evrysdi, completes Jun 2024; follows data from ph2 Topaz in Apr 2021
Phase 1
BIIB110 Biogen Myostatin inhibitor (ActRIIA/B ligand trap) Ph1 according to Q3 results presentation
Source: Evaluate Pharma &

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