Upcoming events Avadel takes on Jazz and Axovant awaits early results – Vantage

Welcome to your weekly roundup of approaching clinical readouts. Data due in the second quarter will see whether Avadels FT218 can take on the leading narcolepsy drug fromJazz Pharmaceuticals. Avadels projectis a once-nightly formulation of sodium oxybate, the same ingredient in Jazzs Xyremand the Xyrem follow-on JZP-258.

Both of Jazzs drugs need to be taken twice nightly, so any improvement on administration, alongside comparable efficacy and safety, couldgive Avadel a boost.

Avadelsplacebo-controlledRest-On studyhas enrolled212 narcolepsy patients aged 16 and older. They start on 4.5g, withtitration up to 9.0g, given once nightly, and can continue to use stimulants at stable doses.The co-primary endpoints measure changes in excessive daytime sleepiness and cataplexy.

Looking at what FT218 is up against, in one of Xyrems trials patients taking 9g hada median reduction of 16 cataplexy attacks frombaseline, versus fourfor placebo(p=0.0016). In another studypatients taking 9g saw a significant median reduction of five points on the Epworth sleepiness scale, versus 0.5 points for placebo.

Xyremhas a black box warning for respiratory depression, and its potential for misuse means that it has a restricted REMS programme. Despite the restrictions Xyrem is the market leader in narcolepsy, but 2023 will see generic competition, and sales are forecast to drop from a peak of $1.7bn this year to $709m by 2024, according to EvaluatePharmas consensus.

Jazzs low-sodium follow-on project JZP-258 will pick up some of the slack, with sales forecast to reach $329m by 2024. JZP-258 was filed last month, and has shown comparable efficacy to Xyrem. Worryingly, two patients in JZP-258s randomised withdrawal study had serious adverse events deemed to be treatment related. If FT218 can show a safety advantage this could be highly positive, but howlikely this is, given than it is essentially a reformulation of Jazz's product, is debatable.

FT218 is being developed under the abbreviated 505(b)2 pathway, so just the one phase IIIstudy is needed.Ultimately Avadelneeds to get it in place before Xyrem generics hit, and to price FT218 accordingly.

Axovant ups the dose

Further data on Axovants Parkinsons disease gene therapy AXO-Lenti-PD, which aims to raiseproduction of dopamine, are expected this month or next.Axovant licensed AXO-Lenti-PD from Oxford Biomedica for $30m in 2018. the therapyis said to be tenfold more potentthan Oxford's Prosavin, which disappointed in the clinic.

Six-month results from the second cohort of the Sunrise-PD trial will concernjust two patients, each on a dose of 1.4x107 transducing units, somethreefold higher than that given to the first cohort, in whom encouraging, albeit early, data, have been reported.

The two patients in cohort 1 showed an average 22-point change from baseline in motor function as assessed by off score at 12 months, representing a 37% improvement. Earlier six-month data showed an average 17-point change from baseline, or 29% improvement. Investors will be looking for a clear dose-response with the second cohort.

In terms of the competition Voyagers VY-AADC is slightly ahead of Axovant's project. Across three cohorts VY-AADC has shown a 12 to 16-point change from baseline in off score,with three-year data from aphase Ib study due later this year. Voyager also hasan ongoing phase IIstudy,Restore-1,in 42 patients.

According to EvaluatePharma the sellside forecasts 2024 sales of $396m for AXO-Lenti-PD, but the disparity between analyst numbers is wide.

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Upcoming events Avadel takes on Jazz and Axovant awaits early results - Vantage