1. Arikace is safe in a prolonged therapeutic regimen. The data from the CF study revealed no surprises on the safety side. Additionally, the FDA's Data and Safety Monitoring Board has held its second oversight meeting for the ongoing Arikace US NTM study, and agreed that 84 days' continuous use of Arikace hasn't thrown up any safety issues.
2. Arikace has just established itself as the future first-choice CF antibiotic. For patients burdened with such a time-consuming therapeutic regimen, an antibiotic which needs to be taken only once a day but still delivers a greater improvement in lung function than anything else available will be a no-brainer.
Look at the way the reduction in pseudomonas kill was trending throughout that CF study - back to baseline for Tobi by the end of each off-therapy phase, but gradually improving for Arikace after the first off-therapy phase. That's a big clue to what would have happened with FEV-1 had the study been longer. And these patients need antibiotics for far longer than six months.
3. Arikace delivered an effective concentration of amikacin to a pulmonary infection in one of the most testing environments for an inhaled antibiotic - the lungs of an individual with CF.
4. Amikacin injection is routinely used to treat NTM.
5. There are currently no approved antibiotics for NTM.
6. An estimated 50,000 each year in the US alone require treatment for NTM.
My six CC takeaways:
1. WL is a wannabe con artist. He opens the CC with this: "Today, we are delighted to announce the achievement of several key milestones in both of our ARIKACE clinical programs. " Delighted with the P-3 effectiveness and safety results? Really? So much for WL's straight shooter reputation.
2. Canada/EU approval for Arikace is no slam dunk given the razor thin margin of Arikace acceptability. One significant safety problem (e.g., an unexplained death or serious complication) during the open label follow up study, and approval could be denied.
3. U.S. approval for CF for Arikace is very much in doubt and likely years away at best. And the notion that the FDA will waive a Phase 3 study requirement for CF is not looking too good right now.
4. Loyal longs can expect a steady stream of no-news and bad news for the next 6-9 months, with financing concerns giving the shorts a solid platform to relentlessly attack the stock.
5. There is no compelling reason to be optimistic about the prospects for NTM, and potential FDA approval is 3-4 years away at best.
6. The reason the PPS is down is because INSM's business prospects are down. Fud and his pals can rationalize all they want, but INSM is in serious trouble right now - literally hanging by a thread.
While I need some time to lick my wounds, I'm feeling more lucky than disappointed. Longs dodged a bullet today. Could have been a disaster instead of a major disappointment.
Forgot to underline the fact that with efficacy now established and the safety side already done and dusted, commercialisation in Europe is all but guaranteed.
As far as I am aware, only a problem on the manufacturing side could now delay an EMEA marketing authorisation.
Poor Evelyn more fud. Marketing " in Europe ( a small market that wedbush thought was maybe 25-30M) is not marketing at all - it's socialism. TIP being cheaper and effective wins.
YTD + 123% ... bashing? No - investing
I give you a credit that at least you are doing your best with pumping. Good job there.
But you may want to wait few weeks and months, so this MB readers are new readers without prior history of your postings.
. What happen with your Russell projections? stock price?
. How about CF phase III results? where is the stock after that?
You failed big time with your calls. Let's start pushing NTM now.
Nicely done -
Pretend you've never seen a single one of my warnings along the way to the effect that Phase III results often fail to reproduce the efficacy seen at Phase II.
Pretend also you've never seen me suggest that four or five million shares would never be sold to the tracker funds for anywhere near the current price unless the sellers had reason to believe there was a problem with the Phase III data..
Am I really that much of a threat to you that you don't mind being exposed as pathetic just as long as you can take a cheap shot at me?
1- a delay in the timeline for filing CF in Europe now to by fist half
2- Insmed will talk to the FDA about a possible path in the U.S
3-Uncertainty about a possible dilution if a US Phase3 is requested ( my guess all along )
4- No catalyst till late in the year ( see #3 ))
5- Brokerages changing heir guidelines ( srwyou Lazard)
6- NTM is clearly now the companies focus.The fastrack designation is the biggest plus today
LISTEN TO THE CALL!!!
There's your answer to why the company did not do a trial in the United States.
The FDA wanted one much longer and now with weak results I say the company declines one because the longer trial would show even more inferiority.SO MAYBE THEY DONT HAVE TO A DILUTION OR A TRIAL.
Also the side effects many questioned with equally vague answers.
"Brokerages changing heir guidelines"?
1. Phase III passed ... check.
2. Safety studies already done and dusted ... check.
3. Approval now a formality ... check.
I'm inclined to agree with you on this occasion :-)
Clueless as usual Terry. The FDA will almost certainly approve Arikace for CF on the basis of the data from the EU study.
If you don't understand how I know this, the extraordinary efficacy evidenced by the Tobi cohort should give you a clue.