PAH is a progressive, fatal disease if untreated. Death rate per annum has been increasing from 5.2 to 5.4 per 100,000. In addition, the hospitalization rate has increased more than two-fold (40.8 to 90.1 per 100,000). Symptomatic pateints who don't recieve treatment survive only about 3 years.
Early treatment is recommended because advanced disease may be less responsive to treatment. For example, multiple trials of epoprastenol (a prostanoid - same class as ADP811) has shown improved survival compared to historical controls. A meta-analysis of 21 randomized trials demonstrated that treatment with a prostanoid (again same class as ADP811) improved mortality compared to controls.
What are the options. The most effective treatmens are prostanoids. There are three presently available- intravenous epoprostenol (Flolan), intravenous treprostinil (Remodulin), and inhaled iloprost ((ventavis). All of these have disadvantages of a short half life which means they have to be given continously over short periods of time.
Problems with each:
Cost: $100,00 per year
Must be delivered through a permanently implanted central venous catheter using a portable infusion pump which can result in thrombosis, pump malfunction, and interruption of infusion. These catheters can also contribute to mortality via these malfunctions of the pump.
Most insures and both medicaid and medicare reimburse these costs
Cost: $120,000 to $160,000 per year
Can be given intravenously or subcutaneously but subcutaneouns injection causes severe pain at the injection site
Most insurers including medicaid and medicare pay for it
Cost: $ 145,633 per year
It is inhaled vs intravenous administration but the main disadvantage is the need for frequent inhalation - 6-9 times per day.
Therefore if there is an oral medication that is just as effective would be a tremendous advantage
Sentiment: Strong Buy
Bumping this post. THis is why it is important. We will be able to recoup the cost of creating this drug fo r this initial indication. Of course there are many more indications so it will be used and will be a BIG money maker.
Clearly (IMO), the P1 results warrant moving forward with APD811. PAH, as you state, is a fatal disease and there are few good treatment options. This is a far different scenario than that of obesity, mental illness, etc.
I frankly feel that Arena committed a serious PR blunder by not explaining the criteria used to decide to continue into P2 and the significance of the arrhythmia event in the context of the indication.
Arena needs to have a PR program that measures up it's potential.