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Post by Thundersnow on Sept 20, 2022 7:30:14 GMT -5
Is anyone awake yet??? This is not good news for UTHR/MNKD. This means MNKD cannot count on COPD as a Revenue Driver. Has MNKD over committed on manufacturing expansion?? I'm getting flashbacks from DNDN.
After 4 years now they are saying there's an Efficacy and Safety issue?
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Item 8.01. Other Events.
On September 20, 2022, United Therapeutics Corporation (the Company) announced the termination of the PERFECT clinical study evaluating Tyvaso® (treprostinil) Inhalation Solution for the treatment of pulmonary hypertension associated with chronic obstructive pulmonary disease. The Company terminated the study based upon a recommendation of the study’s independent Data Safety Monitoring Committee (DSMC), following a routine safety and efficacy analysis conducted by the DSMC.
The Company will review the available data from the study and plans to share the results with the scientific community.
TYVASO is a registered trademark of United Therapeutics Corporation.
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Post by Clement on Sept 20, 2022 7:34:00 GMT -5
Nobody on Wall St has given MNKD share price any credit for COPD as a potential revenue driver.
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Post by Thundersnow on Sept 20, 2022 7:45:03 GMT -5
Nobody on Wall St has given MNKD share price any credit for COPD as a potential revenue driver. I'm worried about the HEADLINES which will drag MNKD down. Hell...Wall Street hasn't given MNKD "ANY CREDIT" for their new Revenue Streams. |
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Post by georgethenight2 on Sept 20, 2022 7:46:13 GMT -5
Is anyone awake yet??? This is not good news for UTHR/MNKD. This means MNKD cannot count on COPD as a Revenue Driver. Has MNKD over committed on manufacturing expansion?? I'm getting flashbacks from DNDN. After 4 years now they are saying there's an Efficacy and Safety issue? ----- Item 8.01. Other Events. On September 20, 2022, United Therapeutics Corporation (the Company) announced the termination of the PERFECT clinical study evaluating Tyvaso® (treprostinil) Inhalation Solution for the treatment of pulmonary hypertension associated with chronic obstructive pulmonary disease. The Company terminated the study based upon a recommendation of the study’s independent Data Safety Monitoring Committee (DSMC), following a routine safety and efficacy analysis conducted by the DSMC. The Company will review the available data from the study and plans to share the results with the scientific community. TYVASO is a registered trademark of United Therapeutics Corporation. Thunder, Don't think they are using a Dreamboat for the study. It's a nebulizer. pipeline.unither.com/product/perfect-inahaled-treprostinil/product-information/No worries here. |
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Post by Thundersnow on Sept 20, 2022 7:48:02 GMT -5
Is anyone awake yet??? This is not good news for UTHR/MNKD. This means MNKD cannot count on COPD as a Revenue Driver. Has MNKD over committed on manufacturing expansion?? I'm getting flashbacks from DNDN. After 4 years now they are saying there's an Efficacy and Safety issue? ----- Item 8.01. Other Events. On September 20, 2022, United Therapeutics Corporation (the Company) announced the termination of the PERFECT clinical study evaluating Tyvaso® (treprostinil) Inhalation Solution for the treatment of pulmonary hypertension associated with chronic obstructive pulmonary disease. The Company terminated the study based upon a recommendation of the study’s independent Data Safety Monitoring Committee (DSMC), following a routine safety and efficacy analysis conducted by the DSMC. The Company will review the available data from the study and plans to share the results with the scientific community. TYVASO is a registered trademark of United Therapeutics Corporation. Now you have to ask yourself will TETON be terminated? |
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Post by Clement on Sept 20, 2022 7:48:40 GMT -5
T-DPI was not used in the PERFECT study.
In the thread UTHR, in "Other BioPharma Stocks", I posted a couple of paragraphs from an FDA document about when a DSMC (aka DMC) terminates a study.
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Post by liane on Sept 20, 2022 8:08:52 GMT -5
Optimist that I am, I have to think they will no longer pursue nebulized trepostinil, and instead focus on T-DPI. Maybe the T-DPI is so much more efficacious. But I'll admit, the posting this morning is nebulous.
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Post by Thundersnow on Sept 20, 2022 8:09:31 GMT -5
Optimist that I am, I have to think they will no longer pursue nebulized trepostinil, and instead focus on T-DPI. Maybe the T-DPI is so much more efficacious. But I'll admit, the posting this morning is nebulous. This was posted on ST. After thinking about it......It's possible they ended the trial early bc of GOOD RESULTS. ---- I am not at all saying this is the case because it’s not clear from what has been released. “IF” the efficacy is so good it becomes unethical to continue the inferior treatment. Perfect study was placebo controlled. Again I am not at all saying this is the case and this new info makes me more nervous than happy. 🤷♂️ |
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Post by Thundersnow on Sept 20, 2022 8:13:58 GMT -5
Optimist that I am, I have to think they will no longer pursue nebulized trepostinil, and instead focus on T-DPI. Maybe the T-DPI is so much more efficacious. But I'll admit, the posting this morning is nebulous. This was posted on ST. After thinking about it......It's possible they ended the trial early bc of GOOD RESULTS. ---- I am not at all saying this is the case because it’s not clear from what has been released. “IF” the efficacy is so good it becomes unethical to continue the inferior treatment. Perfect study was placebo controlled. Again I am not at all saying this is the case and this new info makes me more nervous than happy. 🤷♂️ Why would UTHR publish data if the results were bad? I think the word "TERMINATES" gives the impression it was a FAILED trial. |
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Post by Thundersnow on Sept 20, 2022 8:28:25 GMT -5
From last months CC
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Your next question is from the line of Eun Yang with Jefferies.
Eun Yang
Great quarter. I have a question on Phase III PERFECT trial in COPD patients. So you modified the trial with the increased number of patients and shouldered single-treatment period over 12 weeks. So can you give us -- kind of elaborate on the reason why you changed the study protocol and with this, when we might expect the data?
Dr. Martine Rothblatt
Yes. Thank you, Eun, for your question this morning, and thanks for joining the call. So we modified the trial from -- we actually just triggered a part of the trial as filed, which we had a preplanned opportunity to switch from the crossover design to a parallel design. And we were able to make that decision according to the preapproved protocol design with the FDA and as preapproved by all the IRBs at 2 or 3 different points during the trial. So at each of those points, we would ask ourselves, would switching from a crossover to a parallel design increase our probability even higher of having a successful outcome?
And of course, always what we are obsessed with is having the highest possible probability of a successful outcome. And when this opportunity arose, I guess, it was like 2 months ago now, we decided collectively that if we switched from the crossover to the parallel design, we would be able to increase our odds of having a successful outcome. Even though we probably would have been successful with the crossover, but why rest on probably when you can do it even higher level of assurance? We're all about success here at UT. So we went ahead and triggered the preapproved switch from the crossover over to the parallel.
In terms of when, it's -- we're continuing to enroll that study quite aggressively. All of the patients from the crossover, they flip over into the N for the parallel design. So I think that the study will read out in time to produce the additional patients. Like I said in my introductory remarks, these pipeline patients are -- I mean, the product pipeline patients are not necessary for us doubling our revenues in the next few years, but they're gravy on top of that. So over the next -- it's in the middle of the Phase III.
So within the normal and customary time frame, we're completing these Phase III studies -- we'll be able to complete that study as a parallel side study.
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Post by cjm18 on Sept 20, 2022 9:15:08 GMT -5
This explains high short volume the last two days.
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Post by cretin11 on Sept 20, 2022 9:28:44 GMT -5
I’m with Clement on this. Since nobody has used COPD in revenue projections for MNKD, doesn’t seem like this news should affect us.
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Post by neil36 on Sept 20, 2022 10:11:16 GMT -5
Since PERFECT and COPD are no longer displayed on the UTHR pipeline page, I'm thinking this could possibly bring forward the announcement of a second molecule with Mannkind.
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Post by MnkdWASmyRtrmntPlan on Sept 20, 2022 11:33:10 GMT -5
www.benzinga.com/general/biotech/22/09/28932382/united-therapeutics-stops-late-stage-tyvaso-study-in-lung-disease * The company will review the study's available data and plan to share the results with the scientific community. * In its Q2 earnings release, United Therapeutics disclosed it decided to trigger a pre-specified transition and convert the PERFECT study from a crossover study into a single treatment period of 12 weeks. * Price Action: UTHR shares are down 3.44% at $209.42 on the last check Tuesday. From the 2021 Annual UTHR Report * If Tyvaso DPI is approved and the PERFECT and TETON studies are successful, we also plan to seek FDA approval to expand the Tyvaso-ILD label to include PH-COPD and IPF, respectively. * No therapy is approved to treat PH-COPD. We plan to pursue a PH-COPD indication for Tyvaso and Tyvaso DPI (if approved), assuming our PERFECT study is successful. endpts.com/long-after-first-nod-for-inhaled-tyvaso-united-therapeutics-scraps-phiii-in-ph-with-copd/* United had expected to enroll 314 patients into the study, dubbed PERFECT, which started in June 2018. * Just earlier this month, United had prolonged the trial’s primary completion date, moving it from July 31, 2022, to Dec. 31, 2024 Usually, when companies get bad results from their studies they just bury them. It seems to me that the fact they say they will share the results with the scientific community means that the results are not all bad. Nevertheless, I believe that this does mean they are dropping their plans to pursue COPD with T-DPI, which is a shame for COPD patients, UTHR, MNKD, and their shareholders. BUT, it is also still remotely possible that this study may have just ended gracefully. UTHR's Q2 report says they changed from crossover to a single treatment period of 12 weeks, so those 12 weeks are now over and the study is done. The indication here is that "terminated" and "completed" are different words, but both imply "done". So, was "terminated" just a bad choice of words, or are completed and terminated standard "FDA study" words for finished vs. aborted. I "believe" terminated means aborted in this case. Arnold implied Terminated meant that he killed them, but he also said "I'll be back".  Also, I like Neil36's thought that it will free up time for UTHR and MNKD to pursue another molecule. (look on the bright side).
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Post by Clement on Sept 20, 2022 11:41:51 GMT -5
The 8K did not specifically mention the separate "PERFECT OLE" (open label extension) study, which is a separate clinical trial: clinicaltrials.gov/ct2/show/NCT03794583?term=perfect&cond=ph+copd&draw=1&rank=1"This open-label study will evaluate the safety of continued therapy with inhaled treprostinil in participants who have completed Study RIN-PH-304 (NCT03496623). This study hypothesizes that long-term safety findings will be similar to those observed in the randomized, placebo-controlled, double-blind, adaptive study 'A Phase 3, Randomized, Placebo-controlled, Double-blind, Adaptive Study to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Patients with Pulmonary Hypertension due to Chronic Obstructive Pulmonary Disease (PH-COPD)(RIN-PH-304)." I speculate that the termination of PERFECT does not imply termination of PERFECT OLE. Something good might be happening. |
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