About three years after acquiring SteadyMed and its experimental pulmonary arterial hypertension drug Trevyent, United Therapeutics has decided to kick the program to the curb.
In an 8-K form filed with the SEC on Tuesday, United revealed that they are shutting down development of Trevyent after receiving feedback from the FDA last week. The move marks the end of a long and bumpy road for the program, which had previously earned an RTF in 2017 and a CRL last April, as United moves forward with new Tyvaso formulations.
Regulators had directed United to both redesign the Trevyent product and conduct an entirely new clinical study. That not only would have added significant delays to the program, United wrote, but may have proved unsuccessful in answering the FDA’s concerns.
Specifically, the FDA took issue with the product’s pump device. Trevyent uses a pump to deliver a reformulated version of United’s mainstay PAH drug Remodulin. The accuracy of the pump, however, was not up to snuff in regulators’ eyes, and United said the agency wanted the company to improve the accuracy “in certain respects.”
So United has decided to throw in the towel. They’re halting all development of the program after determining it wouldn’t be commercially reasonable to move forward. It marks an unceremonious end to a program they once picked up for a bargain from their competitors at SteadyMed in 2018.
With the $216 million buyout, United had been looking to head off a PAH clash between the two companies by acquiring Trevyent itself. In addition to that program, SteadyMed at the time had been trying to invalidate one of United’s patents for the active ingredient in three of its prominent franchises: Remodulin, Tyvaso and Orenitram.
Though United moved forward with Trevyent, submitting its NDA to US regulators in September 2019, the agency rejected its pitch in an April 2020 CRL. The FDA had said that some of the deficiencies previously pointed out had “not yet been addressed to its satisfaction,” United said at the time. United had been working on a new pitch and Tuesday’s decision stemmed from its subsequent meeting with the FDA to resubmit Trevyent for approval.
It’s the latest in a couple of pipeline setbacks United has seen over the last few years. In 2019, the company dropped PAH hopeful esuberaprost after it failed a combo Phase III study, failing to show benefit when combined with Tyvaso over United’s older Remodulin drug. Then, in February, United revealed that a Phase II/III trial combining their Unituxin antibody with a standard treatment, irinotecan, failed to help small cell lung cancer patients live longer.
United is instead moving forward with an inhaled reformulation of Tyvaso after snagging a priority review voucher from Y-mAbs Therapeutics last December for $105 million. The voucher had originally been slated for Y-mAbs’ application for Danyelza in neuroblastoma, and it wasn’t used for the FDA’s approval in November.
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Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.
The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.
Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.
CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.
Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.
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The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.
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After providing for a brief mime of regulatory judiciousness, the FDA stamped their emergency approval on J&J’s Covid-19 vaccine Saturday, adding to the Biden administration’s plan aimed at ending the pandemic in the near term — at least in the US. The CDC came through on Sunday with its stamp of approval and J&J is reportedly expected to start delivering vaccine sometime in the next few days.
Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.
Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.
Soticlestat made it.
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FibroGen will likely have to delay its US rollout for roxadustat once again.
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大约三年后,SteadyMed和它的前Pul ar-te hy-10的药物Trevyent,单位已经破灭,将该项目踢到了路边。
就在几周前，SVB Leerink的分析师Geoffrey Porges预测，roxa的批准可能会在3月20日PDUFA的日期之前--尽管FDA在去年12月延长了审查期，但这一批准已经让人失望了一次。阿斯利康与FibroGen合作开发慢性肾病相关贫血药物，该公司透露，监管机构已要求对临床数据进行进一步的澄清分析。