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BB-backed Ionis approaches key regulatory events
Ionis Pharmaceuticals (Nasdaq: IONS), a key shareholding for BB Biotech (10.9% of NAV) and several other specialist investment vehicles, is approaching a number of important regulatory events. In particular, these include the outcome of the FDA review for Waylivra, which has a deadline at the end of this month.
Waylivra has been developed for the treatment of familial chylomicronemia syndrome, an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase which causes severe hypertriglyceridemia and a risk of unpredictable and potentially fatal acute pancreatitis. Earlier this year, Waylivra was reviewed by an FDA advisory committee, which voted 12-8 in favour of approval – a reasonable but not overwhelming endorsement.
The drug, which is also in Phase III studies for familial partial lipodystrophy, is owned by Ionis’ 75%-owned Akcea Therapeutics (Nasdaq: AKCA) subsidiary. This holds Ionis’ rare disease assets and is separately held by BB Biotech (1.8% of NAV).
Akcea has a second product in registration, Tegsedi, for polyneuropathy in adults with hereditary transthyretin amyloidosis (hATTR). This is the same indication as Alnylam’s recently approved Onpattro and is a very rare condition. Tegsedi has already received marketing authorisation in the EU and has a FDA action date of 6 October. Onpattro is thought to have efficacy advantages over Tegsedi, but Akcea could – assuming it is approved – price its product aggressively to win share. Furthermore, key clinical data from a third competitor, Pfizer’s Tafamidis, are sue to be reported at a scientific conference on 27 August, which could yet further alter the competitive dynamic.
Akcea has four other compounds in development:
Separately, Ionis is also shortly expecting its partner Roche to initiate pivotal trials of a licensed compound, IONIS-HTTRx, in Huntington’s disease.
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