The edited presentation I listened to today provides several important information for Longs . . .
1. Lewis suggests the data will meet the endpoint of "non-inferiority" for CF/Pa and was very optimistic that the data will demonstrate superiority by virtue of superior kill of Pa.
2. He went on to explain the importance of the 28 day on 28 day off treatment equivalency with TOBI in terms of developing mutant strains that might become resistant. He then linked this treatment regimen with two important clinical facts and alluded to two others:
a. The once per day treatment regimen is more easily scheduled for patients than BID for TOBI, or TID for Cayston.
b. That once patients experience the single dose regimen, they will be reluctant to switch to a different BID or TID antibiotic regimen unless their is a life threatening change.
c. Lewis alluded to an important point that is currently not emphasized, which is the concurrent therapy with other agents would be prescriptively allowable for patients. Using certain adjunctive agents (e.g., Pulmozyme) was pretty much precluded in the Clear-108 trial. That is between the physician and the patient.
d. He also alluded to the fact that because of antibiotic resistance and non-penetration of macrophages, other antibiotic formulations could be developed in the future.
3. He went on to suggest that the Extension Trial could be pivotal and allow use of Arikace for much longer periods than the 6 cycles in the Clear-108 Phase III.
4. The NTM trial was referenced well by re-iterating the "captured market" for Arikace if the bacterial kill endpoint can be achieved. Relative to this use, Lewis stated:
a. INSM believes their endpoints are achievable and will show a statistically significant therapeutic effect.
b. If a. is achieved they will file with the FDA for expedited review by year end.
c. If they achieve b., they will cross file in Europe, Japan and with other regulatory agencies.
5. The most important part of the presentation, I believe, was the understanding that INSM now has of the regulatory process on an international scale. They know there is extensive regulatory filings to be submitted in order to enter and capture market(s).
6. Lewis said that INSM would market and not look for a partner.
7. Lewis went on to outline the IP supporting the exclusivity of the liposome for 2029 and beyond, which is the key to future drug development. The key to this IP is the fact that the liposome composition is similar to the natural lipid coat inside the lungs.
In all, I heard the facts stated well, a good understanding of the clinical signs and endpoints, a realistic grasp of the regulatory situation and a good understanding of the market for inhalational antibiotics. It is a vast improvement from the previous administration, regulatory approach and presentation of the clinical science. Congratulations, INSM.
I change my outlook.
Finally . . . some light at the end of the tunnel.
Sentiment: Strong Buy
Insmed will never market Arikace by themselves.
Much of the hype by Will Lewis yesterday ( I think he did a good job) is startup bios hubris.
A sales force costs lots of money and current shareholders would have to be
heavily diluted. If the trials look positive, a partner would help keep a takeover at bay
,but the share price would suffer once revenues came in.Good Luck
It may simply be a matter of leveraging the network already established for the clinical studies. If the leading pulmonologists in the US and Europe begin recommending Arikace it will simply be a matter of making it available.
I do think it was the first time that we were told that a successful NTM phase II would likely lead to an application. Also, Lewis did seem (IMO) to believe that success in Europe with CF, along with success here vs. NTM, may allow Insmed to apply with the FDA for a greatly expanded label. (U.S. CF phase III may not be necessary)
He also talked about the potential for premium pricing for NTM, and the necessity to file for QIDP status for NTM (and likely non CF bronchiectasis - IMO)
He also mentioned something at the very end that I didn't really catch, regarding "the other 75%) Did you get that Stu or Rehvdm?
My HUGE take-away.., Lewis staing two times that there is a very HIGH carryover to the 2 year extension study. A 'VERY HIGH CARRYOVER' meaning that patients must like the results and even those patients taking TOBI are probably now on Arikace as per Dr recommended.
Will Lewis is practically telling us that Arikace will at least have non inferior results!!!!