MannKind Receives U.S. FDA Fast Track Designation

[mango]

If you look at the primary and secondary outcome measures they’re all from baseline to end of 6 months. I’m pretty sure the trial is only 6 months long and I think Mike has said that before.

Also, the Arms and Interventions states: “…Study drug will be inhaled using the PARI breath-enhanced jet nebulizer system daily for 28 days in Cycle 1. Cycle 2 will commence after 56 days off treatment and resume daily for 28 days.”

That’s all they’re doing so the trial will not be lasting 4 1/2 years. It’ll be 6 months. There’s an extension phase too I believe, for certain participants. So it says right there in the intervention description how long this trial will last and in the outcomes. Outcomes say end of 6 months. The intervention will last: 28 days ON, 56 days OFF, 28 days ON. That’s it, that’s the trial.

[mango]

This is from one of the recent calls about Clofazimine trial:

“So, we hope to have that interim analysis sometime in the second half of next year. And then we would just be waiting for the full patient population to get there in order to hopefully file on six-month data. So, that's our goal. It's a primary endpoint of six months.”

[celo]

It is all about enrollment. These patients do seem in need of something to help, so enrollment may be fast.

[letitride]

Part A 6 month primary completion date looks like its estimated August 2026. Part B looks like an additional 22 months. Looks like Mike plans on filing on the
Part A primary data. A quick full enrollment could accelerate the estimated August 2026 conclusion of part A.

[mango]

I agree, the timeline will be dependent on how quickly enrollment completes. The actual trial length for the primary outcomes, which is what will be used to file for approval, is just 6 months. There’s a few secondaries that extend into 22 months, but some of those appear to be the extension phase. Hopefully the enrollment fills quickly, we have like 100 sites, right? I think with the fast track designation and this being a rare disease things will move quickly!

[phdedieu12]

Yes, look forward to a rapid enrolment!!

[letitride]

Mannkind is in Japan come June Lets Go!

[phdedieu12]

This could be bigger than Afrezza, and yes the demand is there, and wanted fast

[parrerob]

Thanks guys so primary outcomes are enough for submission..... I am very ignorant on this but 4 years an half was really too much

[agedhippie]

May 19, 2024 19:32:25 GMT -5 mango said:

If you look at the primary and secondary outcome measures they’re all from baseline to end of 6 months. I’m pretty sure the trial is only 6 months long and I think Mike has said that before.

Also, the Arms and Interventions states: “…Study drug will be inhaled using the PARI breath-enhanced jet nebulizer system daily for 28 days in Cycle 1. Cycle 2 will commence after 56 days off treatment and resume daily for 28 days.”

That’s all they’re doing so the trial will not be lasting 4 1/2 years. It’ll be 6 months. There’s an extension phase too I believe, for certain participants. So it says right there in the intervention description how long this trial will last and in the outcomes. Outcomes say end of 6 months. The intervention will last: 28 days ON, 56 days OFF, 28 days ON. That’s it, that’s the trial.

^This

They extension is because they want to see the percentages of cases where NTM reoccurs because that is a current problem. This is the problem you see where the infection drops to low levels, but is not eliminated and reoccurs over time. There are existing oral versions of clofazimine that would be the competition for 101 and will probably limit the price that can be charged at scale (there are always corner cases where the oral absolutely cannot be used for which a high price can be supported.)

[cjm18]

Best case for approval is 6 months (shorter review period) after
Estimated Primary Completion Date of August 1, 2026?

[Clement]

^ Japan will take longer.

[ptass]

May 23, 2024 10:28:59 GMT -5 cjm18 said:

Best case for approval is 6 months (shorter review period) after
Estimated Primary Completion Date of August 1, 2026?

8/1/2026 sounds about right. The entire study population across all sites do not start the trial at the same time. Some participants will complete the trial while recruitment is ongoing.

[hellodolly]

May 23, 2024 9:23:34 GMT -5 agedhippie said:

May 19, 2024 19:32:25 GMT -5 mango said:

If you look at the primary and secondary outcome measures they’re all from baseline to end of 6 months. I’m pretty sure the trial is only 6 months long and I think Mike has said that before.

Also, the Arms and Interventions states: “…Study drug will be inhaled using the PARI breath-enhanced jet nebulizer system daily for 28 days in Cycle 1. Cycle 2 will commence after 56 days off treatment and resume daily for 28 days.”

That’s all they’re doing so the trial will not be lasting 4 1/2 years. It’ll be 6 months. There’s an extension phase too I believe, for certain participants. So it says right there in the intervention description how long this trial will last and in the outcomes. Outcomes say end of 6 months. The intervention will last: 28 days ON, 56 days OFF, 28 days ON. That’s it, that’s the trial.

^This

They extension is because they want to see the percentages of cases where NTM reoccurs because that is a current problem. This is the problem you see where the infection drops to low levels, but is not eliminated and reoccurs over time. There are existing oral versions of clofazimine that would be the competition for 101 and will probably limit the price that can be charged at scale (there are always corner cases where the oral absolutely cannot be used for which a high price can be supported.)

Curious what role the current SAE's being mentioned by Mike are included with the oral versions? Was it 101 that was once used for leprosy and has many reported side affects?  Mike feels with the new intervention lasting only six months, this reduces the systemic tox levels and thus, he expects more patient levels of compliance? If so, who will champion 101 as better than orals?  MNKD?  

[casualinvestor]

May 23, 2024 9:23:34 GMT -5 agedhippie said:

May 19, 2024 19:32:25 GMT -5 mango said:

If you look at the primary and secondary outcome measures they’re all from baseline to end of 6 months. I’m pretty sure the trial is only 6 months long and I think Mike has said that before.

Also, the Arms and Interventions states: “…Study drug will be inhaled using the PARI breath-enhanced jet nebulizer system daily for 28 days in Cycle 1. Cycle 2 will commence after 56 days off treatment and resume daily for 28 days.”

That’s all they’re doing so the trial will not be lasting 4 1/2 years. It’ll be 6 months. There’s an extension phase too I believe, for certain participants. So it says right there in the intervention description how long this trial will last and in the outcomes. Outcomes say end of 6 months. The intervention will last: 28 days ON, 56 days OFF, 28 days ON. That’s it, that’s the trial.

^This

They extension is because they want to see the percentages of cases where NTM reoccurs because that is a current problem. This is the problem you see where the infection drops to low levels, but is not eliminated and reoccurs over time. There are existing oral versions of clofazimine that would be the competition for 101 and will probably limit the price that can be charged at scale (there are always corner cases where the oral absolutely cannot be used for which a high price can be supported.)

If 101 is approved for NTM, would oral clofazimine be competition? It was approved in the US for leprosy as Lamprene, but was discontinued in 2004. It is also available for NTM under an expanded access program

Once there is an FDA approved drug for NTM, I would think that patients would be prescribed the approved drug (MNKD-101), rather than doctors putting in a SPIND request.