"In 2012, in collaboration with the NIH, we funded a study performed by Clarity Pharma Research that showed there were an estimated 50,000 cases in the US in 2011 ..."
"According to SDI Healthcare Database NTM patients average 7.6 antibiotic courses and 10.2 hospital days per year."
"We anticipate that ARIKACE will be administered ... for a period of 84 days for this indication."
Whitten's guidance -
"... the pricing for the Tobis and the Caystons of the world are in that kind of $4,000 to $5,000 range ... you would expect that we would be competitive in our pricing.
We view NTM differently. While we don't have exact pricing, and it's way too early, we think the price point in NTM is potentially significantly higher for those patients than it is in CF. "
The Cystic Fibrosis Services Pharmacy when I last checked priced four-week courses of Tobi and Cayston at $5,776.40 and $5,844.68 respectively.
One assumes the Arikace twelve-week NTM course will be priced at around $20,000 - doubled for non-CF maintenance therapy.
50,000 patients paying $20,000 would generate $1 billion in revenue each year from the US NTM opportunity.
75,000 patients paying $40,000 would generate $3 billion in revenue each year from the US Non-CF bronchiectasis opportunity (six four-week courses of rotational maintenance therapy).
However, not every patient suffering from a pulmonary NTM infection will have medical cover. And not every Non-CF bronchiectasis patient carrying a gram-negative infection will be prescribed antibiotic maintenance therapy (or develop pneumonia).
But Arikace will have no real competition. There are currently no FDA-approved antibiotics for either disease.
And let's not forget that those two diseases account for just a small proportion of the million-plus patients hospitalised in the US each year with pneumonia.
Or that the US accounts for less than 5% of the global population - and has a very low incidence of Tuberculosis.
Are the FDA and EMEA realistically going to approve Arikace for Cystic Fibrosis only - and allow the following patient population to continue to be a breeding ground for antimicrobial resistance as a result of the length of time involved in eradicating pulmonary infections using injected antibiotics?
From the 10-K -
It is estimated that there are more than 250,000 non-CF bronchiectasis patients in the US (SDI Innovations in Healthcare Analytics, 2008), of which approximately 30% of non-CF bronchiectasis patients are infected with Pseudomonas (Wilson, C.B., et al., Eur Respir, 1997, 10(8):1754-1760); Nicotra, M.B., et al., Chest, 1995 108(4):955-961).
Currently there are no approved antibiotics for this indication.
When bronchiectasis patients become infected with Pseudomonas, they tend to have more frequent exacerbations and hospitalizations and are more frequent users of antibiotics.
ARIKACE was granted orphan drug status in the US for the treatment of bronchiectasis in patients with Pseudomonas or other susceptible pathogens.
In May 2009 we completed our randomized, placebo controlled US phase 2 study (TR02-107) of ARIKACE in the treatment of chronic Pseudomonas infection in non-CF patients with bronchiectasis.
In the study, 64 study subjects were randomized (1:1:1) to receive ARIKACE 280 mg, ARIKACE 560 mg or a placebo on a daily basis during a 28-day on-treatment period. The subjects completed follow-up assessments at the end of a 28-day off-treatment period.
There was a statistically significant reduction in Pseudomonas density observed in the 560 mg ARIKACE cohort relative to the placebo cohort.
Patients receiving ARIKACE experienced fewer pulmonary exacerbations at a rate of 4.7%, as compared to 10.5% in those receiving placebo.
Hospitalization from any cause occurred at a 5.3% rate for patients in the placebo cohort, as compared to a 2.3% rate for patients in the ARIKACE cohort.
It should be stressed that, whereas a Cystic Fibrosis patient who becomes infected with Pseudomonas needs antibiotics, and an individual diagnosed with pulmonary NTM disease needs antibiotics, an individual with Non-CF bronchiectasis carrying a gram-negative infection might have a sufficiently robust immune system to keep the infection under control.
It would be useful to have an idea of the proportion of the 75,000 in the US estimated to be in that position who do actually need antibiotics.