pakman schreef op 24 december 2024 10:24:
With regards to partnering strategy for KL1333 and NV354, Abliva’s management has indicated that
both options are on the table. The company could bring the assets all the way to the market itself or
sign a deal if the right partner comes on board. In our model we assume that Abliva will
commercialise both products itself. The assumptions for our rNPV model are summarised in
Exhibit 10. Key inputs include:
? Target patient populations. Abliva is focusing on specific PMDs for both assets in order to
evaluate the drug candidates in as homogenous patient population as possible, which improves
the likelihood of quality data and statistical analysis. PMD epidemiology data are scarce, but
using available sources we calculate respective target patient populations for KL1333 and
NV354 of 40k and 18k in the United States and the top 15 wealthy European countries (top
five, the Nordics, Benelux, Austria, Switzerland and Ireland).
? Success probabilities. For KL1333 we use a 25% probability to reach the market, which is in
line with recent analysis published by Wong and Siah (2019), who found drug candidates in
neurology in Phase II have probability within the range 20–24%; drug candidates for all
indications excluding oncology had probabilities within the range 27–29%. For NV354 we use
5% while this asset is in the preclinical stage. As described above, it could move into Phase I
as soon as next year.
? Pricing and market penetration. We assume a price tag of $110,000 for KL1333 and
$130,000 for NV354 (50% discount in Europe applied) per patient per year. EvaluatePharma’s
2019 report calculates that the average price per patient for an orphan drug (top 100 products)
was $151k in 2018, while the median was $110k. For KL1333 we use the price equal to the
median (2018 data available only), while for NV354 we assume a price closer to the average
orphan drug pricing, as it is a smaller indication than that targeted by KL1333. The range is
wide, however, and higher pricing could be secured depending on cost effectiveness. We
assume 20% market penetration for KL1333 and 30% for NV354, as it targets a smaller
indication. As there are no effective treatments available, there is no precedent for how to
model the uptake of new drugs in PMDs. It could be argued that once an effective treatment is
introduced, most patients would be willing to receive it, so market penetration could be well
above 50%. For the time being we use a conservative approach, but in Exhibit 9 we have
provided a sensitivity analysis of peak sales to price and market penetration.
? R&D development costs. We estimate the cost of Abliva’s pivotal Phase II/III study of KL1333
at c $30m and assume a similar development pathway for NV354. Assumed launch dates are
2025 for KL1333 and 2028 for NV354, with peak sales reached in six years ($670m for KL1333
and $320m for NV354).
uit het report, ze schatten maar 25% kans op goedkeuring....en blijkbaar Geen partner vh product gevonden...tricky