Novartis cans branaplam after seeing Huntington's safety signal, delays orphan drug over slow enrollment

01 Feb 2023
www.fiercebiotech.com
Phase 2Gene TherapyPhase 3
Novartis canned branaplam in an update that also featured details of changes to the R&D plan for iptacopan.
Novartis has delbranaplame coup de grâce to its ailing branaplam program, stopping (PDF) developmiptacopane splicing modulator in Huntington’s disease in the wake of a safety signal. The Big Pharma disclosed the update alongside news of the delays and discontinuations that have hit oral factor B inhibitor iptacopan.
Novartist, Novartis stopped dosing in a phase 2b clinical trial of branaplam after seeing early signs that tsplicing modulatore caHuntington’s diseaseropathy, a condition characterized by damage to the nerves outside of the brain and spinal cord. Novartis spent the rest of the year going over the data on tiptacopanandidate before reaching a decision on the next steps.
Now, the maNovartis team has revealed it is discontinuing the progrbranaplamon the assessment of the potential risk-benefit profile seenperipheral neuropathyy. The news is another blow for the industrywide effort to develop treatments for HuntingtNovartisich has in recent years suffered setbacks including the failure of Roche and Ionis Pharmaceuticals’ antisense drug tominersen in phase 3 and the temporary pause of uniQure’s high-dose gene therapy arm.
Novartis’ decision to exit the race, which follows the termination of development of branaplam in spinal muscular atrophy, makes PTC Therapeutics’ PTC518 the most advanced Huntington’s splicing modulator. PTC plans to share 12-week phase 2Huntington’s candidate in the second quarter.RocheIonis Pharmaceuticalstominersen
Novartis canned branaplam in a fourth-quarter pipeline update that featured details obranaplam to spinal muscular atrophypan, thePTC TherapeuticsPTClecule the compPTC518 developing to challenge AstraZeneca’s Alexion for tPTCblockbuster paroxysmal nocturnal hemoglobinuria (PNH) market. The company is on track to file for approval in PNH this year, but other aspects of the strategy have slipped.
Novartisrecruitmbranaplaming slower than expected in three late-phase clinical trials that are designed to support iptacopan in IgA nephropathy, C3 glomerulopathy and atypical heAstraZenecamic syndrome (aHUS). Novartis sparoxysmal nocturnal hemoglobinuria (PNH)opathy and C3 glomerulopathy this year, but its target filing date has slipped to 2024. The filing in aHUS has moved from 2025 to 2026 at the earliest.
Novartis has given up on membranous nephropathy entirely, discontinuing work on the phase 2 program after seeing an “uncompellinIgA nephropathyroC3 glomerulopathying fatypical hemolytic uremic syndrome (aHUS)muNovartisex membranoproliferative glomeruloIgA nephropathyase 3C3 glomerulopathyication is set to get underway this year to support a filing for approval in 2026 or later.
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