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Post by nxc2 on Sept 20, 2022 6:01:59 GMT -5
Uthr has press release
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Post by neil36 on Sept 20, 2022 6:13:38 GMT -5
Do you have a link? I'm not seeing anything on the UTHR website.
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Post by neil36 on Sept 20, 2022 6:36:52 GMT -5
Found the 8k
Some clarification and potential impact (if any) would be helpful
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Post by Clement on Sept 20, 2022 6:53:16 GMT -5
from the 8K: "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." d18rn0p25nwr6d.cloudfront.net/CIK-0001082554/2dfc2977-d1d2-4610-aff5-2ecdc29048e0.pdf
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Post by Clement on Sept 20, 2022 7:06:50 GMT -5
from the 8K: "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." d18rn0p25nwr6d.cloudfront.net/CIK-0001082554/2dfc2977-d1d2-4610-aff5-2ecdc29048e0.pdfPERFECT used Tyvaso-nebulizer and therefore the termination of the PERFECT study should have no impact on the safety record of T-DPI.
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Post by Clement on Sept 20, 2022 7:38:37 GMT -5
Note: DMC is aka DSMC www.fda.gov/media/75398/download7.2.1. DMC Recommendations to Terminate the Study In almost all cases, a DMC is advisory to the sponsor; the sponsor decides whether to accept recommendations to discontinue a trial. FDA will rarely, if ever, tell a sponsor which decision to make. For trials that may be terminated early because a substantial benefit has been observed, however, consideration may still need to be given to the adequacy of data with regard to other issues such as safety, duration of benefit, outcomes in important subgroups and important secondary endpoints. We recommend that sponsors of trials that could potentially be terminated early for efficacy reasons discuss these issues with FDA prior to implementing the trial, when the statistical monitoring plan and early stopping boundaries are being developed. In these settings, consultation with FDA may provide the sponsor with important information regarding the regulatory and scientific implications of a decision and may lead to better decisions. Sponsors are encouraged to revisit these issues with FDA when considering DMC recommendations for early termination if new issues have arisen and/or if the regulatory implications of early termination were not adequately clarified at the outset of the trial. For trials that may be terminated because of safety concerns, timely communication with FDA is often required (see, e.g., 21 CFR 312.56(d) (drugs); 21 CFR 812.150 (devices)). In such cases, we recommend that the sponsor initiate discussion as soon as possible about the appropriate course of action, for the trial in question as well as any other use of the investigational product. We strongly recommend that sponsors initiate discussion with FDA prior to early termination of any trial implemented specifically to investigate a potential safety concern.
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Post by Thundersnow on Sept 20, 2022 7:56:23 GMT -5
Thanks for the info.
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Post by Thundersnow on Sept 20, 2022 8:08:54 GMT -5
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 prcgorman2 on Sept 20, 2022 9:04:25 GMT -5
Follow the money. How is nebulized Tyvaso fairing in the market as compared to DPI? Anecdotal info from Symphony seems like it might indicate DPI is dominating sales. Maybe just terminate the very long study, and publish useful information (which I wonder whether it might include results which indicate nebulized liquid is not as safe as dry powder - LQDA?).
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Post by Clement on Sept 20, 2022 9:37:36 GMT -5
PERFECT and PH due to COPD no longer show up on the UTHR pipeline page.
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Post by jkendra on Sept 20, 2022 15:14:24 GMT -5
endpts.com/long-after-first-nod-for-inhaled-tyvaso-united-therapeutics-scraps-phiii-in-ph-with-copd/Long after first nod for inhaled Tyvaso, United Therapeutics scraps PhIII in PH with COPDKyle LaHucikAssociate Editor
After a recent court win for its inhaled pulmonary arterial hypertension drug, pushing off competition for a few more years, United Therapeutics has quietly scrapped a late-stage study of Tyvaso.
The Phase III termination occurred because of a recommendation from the data safety monitoring committee “following a routine safety and efficacy analysis,” United said in an SEC filing Tuesday morning.The biopharma first lined up FDA approval of the inhaled drug in the summer of 2009, for PAH. The drug pulled in $480 million in sales in 2020 and $607 million in 2021. Cowen analyst Joseph Thome has modeled revenues peaking at $1.6 billion in 2025 for the inhaled franchise.
The culled Phase III was observing the drug in patients with pulmonary hypertension due to chronic obstructive pulmonary disease, or PH-COPD. Just earlier this month, United had prolonged the trial’s primary completion date, moving it from July 31, 2022, to Dec. 31, 2024, according to an update to the federal trials database.
United had expected to enroll 314 patients into the study, dubbed PERFECT, which started in June 2018. The company will take a look at the data and plans to present the results to scientists, according to the SEC document.
It’s not the first time United has run into troubles trying to expand the label for Tyvaso, also known as treprostinil. In 2017, the company withdrew a Phase III study of the oral formulation in patients with PH associated with sickle cell disease. Another Phase III of the oral formulation was terminated in fall 2020, testing the drug in patients with PH associated with heart failure with preserved ejection fraction.
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Post by jkendra on Oct 5, 2022 11:38:39 GMT -5
pulmonaryhypertensionnews.com/news/worsening-ph-ild-prevented-with-higher-tyvaso-dose-analysis/Higher Tyvaso Doses Better at Preventing PH-ILD Worsening
Post hoc analysis shows benefit of starting Tyvaso early, increasing dosage
Marisa Wexler, MS avatar by Marisa Wexler, MS | October 5, 2022 A high dose of Tyvaso (inhaled treprostinil) — at least nine breaths four times per day — is more effective at preventing clinical worsening than lower doses for people with pulmonary hypertension associated with interstitial lung disease (PH-ILD), a new study indicates. “This analysis further reinforces that [Tyvaso] prevents clinical worsening, and in addition demonstrates that [Tyvaso] is associated with significantly more patients experiencing clinical improvement when compared to placebo. In addition, our analysis suggests that higher dose [Tyvaso] is more effective than lower dose in achieving these outcomes,” the researchers wrote. The study, “Inhaled Treprostinil Dose in Pulmonary Hypertension Associated with Interstitial Lung Disease and Its Effects on Clinical Outcomes,” was published in Chest. The work was funded by United Therapeutics, which markets Tyvaso in the U.S. Tyvaso was approved by the U.S. Food and Drug Administration (FDA) in 2021 to treat PH-ILD. The approval was supported by data from the Phase 2/3 INCREASE trial (NCT02630316) that compared it against a placebo in 326 adults with PH-ILD. INCREASE met its main goal: after 16 weeks, participants given Tyvaso showed improvements in exercise capacity, measured by a six-minute walk distance (6MWD), compared to those on a placebo. At the start of INCREASE, participants were given Tyvaso or a placebo at a dose of three breaths per session, four times daily. Under the direction of a study investigator and based on tolerability, participants would gradually increase the dose up to a maximum of 12 breaths per session. Based on the titration schedule, the earliest a patient could increase to a dose of nine breaths per session would be on study day 21 (three weeks). In this post hoc analysis, researchers divided the patients into two groups based on the dosage they had been on at week four. A post hoc analysis is one carried out after the study is already over and data have been collected. Of 149 patients in the Tyvaso group, 70 were on a dose of at least nine breaths per session at week four, as were 86 of 153 patients in the placebo group. The rest were on lower doses. From week four through the end of the study, the scientists compared rates of clinical worsening — defined as 15% decrease in 6MWD, hospitalization, lung transplant, or death — in the dosage groups. Rates of clinical worsening in the high- and low-dose Tyvaso groups were 17.1% and 22.8%, respectively. In the high- and low-dose placebo groups, the respective rates were significantly higher at 33.7% and 34.3%. Clinical improvement — a 15% increase in 6MWD and 30% decrease in levels of a marker of heart damage called NT-proBNP by study week 16, in the absence of any worsening events — was reported in 15.7% of patients in the high-dose Tyvaso group and 12.7% in the low-dose Tyvaso group. In the high- and low-dose placebo groups, improvement was reported in 7% and 1.5% of patients. “This post hoc analysis demonstrates that a higher dose of [Tyvaso] is associated with an overall greater benefit in patients with PH-ILD, not only in comparison to placebo but also in comparison to lower dose of [Tyvaso],” the scientists wrote. The researchers said they limited their analysis to weeks four through 16 in order to avoid biased data during the early dose-adjusting period. Still, they noted a number of clinical worsening events occurred during the first few weeks of the study, even though those were not included in the dose analysis. “The fact that there were a sizable group of patients who did have clinical worsening during this early phase of the study does however underscore the need for the early implementation of therapy,” the scientists wrote, noting a limitation of the study was that participants were allowed to continue adjusting doses throughout the study, so the dosage at week four was not necessarily the one through week 16. However, sensitivity analyses based on the last dose received, rather than at week four, yielded very similar results to the primary analysis. “These results support the importance of early initiation and uptitration of inhaled [Tyvaso] therapy to at least 9 breaths or more four times a day,” the researchers concluded.
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Post by Clement on Oct 11, 2022 14:31:12 GMT -5
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Post by wyattdog on Oct 11, 2022 15:05:20 GMT -5
Morgan Stanley Initiates United Therapeutics With Overweight Rating, $288 Price Target 8:32 AM ET, 10/11/2022 - MT Newswires 08:32 AM EDT, 10/11/2022 (MT Newswires) -- United Therapeutics (UTHR) has an average rating of outperform and price targets ranging from $142 to $325, according to analysts polled by Capital IQ. (MT Newswires covers equity, commodity and economic research from major banks and research firms in North America, Asia and Europe. Research providers may contact us here: www.mtnewswires.com/contact-us)Disclosures Data source identification
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Post by Clement on Oct 11, 2022 15:26:39 GMT -5
Morgan Stanley Initiates United Therapeutics With Overweight Rating, $288 Price Target 8:32 AM ET, 10/11/2022 - MT Newswires 08:32 AM EDT, 10/11/2022 (MT Newswires) -- United Therapeutics (UTHR) has an average rating of outperform and price targets ranging from $142 to $325, according to analysts polled by Capital IQ. (MT Newswires covers equity, commodity and economic research from major banks and research firms in North America, Asia and Europe. Research providers may contact us here: www.mtnewswires.com/contact-us)Disclosures Data source identification Compared to analyst average price target of 248.56. I'm glad they can see the growth going on in the Tyvaso franchise.
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