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:
- AKCEA-APO(a)-LRx – This is in a Phase II study for treatment of cardiovascular disease in patients with high Lp(a) with results expected by the end of the year. Novartis has an option to licence the product, in which case it will be responsible for all future development activities including conducting a global Phase III cardiovascular outcome study.
- AKCEA-ANGPTL3-LRx is in a Phase II trial for familial partial lipodystrophy with results due shortly;
- AKCEA-APOCIII-LRx is in Phase II for hypertriglyceridemia with results due next year; and
- AKCEA-TTR, which is intended for hereditary and wild-type ATTR, is expected to enter the clinic in 2018.
Separately, Ionis is also shortly expecting its partner Roche to initiate pivotal trials of a licensed compound, IONIS-HTTRx, in Huntington’s disease.