Milvexian

Bristol Myers Squibb has reported phase 3 wins in a lung disease and a solid tumor, taking two steps on its path toward post-patent cliff growth. One phase 3 trial, Aloft-IPF, tested the LPA1 antagonist admilparant in adults with idiopathic pulmonary fibrosis (IPF). The other study, Action-1, evaluated a radiopharmaceutical therapy, RYZ101, in people with SSTR-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Both trials met their primary endpoints, albeit without fully resolving doubts about the programs. The 60 mg dose of admilparant had a statistically significant effect on forced vital capacity, a measure of lung function, at Week 52. Yet, while that result ensured a primary endpoint hit, the higher 120-mg dose failed to clear the bar for statistical significance. BMS has yet to share data from the trial. The “totality of the findings” supports admilparant’s potential as a clinically meaningful first-in-class treatment, BMS said. The drugmaker plans to talk to regulators about filing for approval of admilparant. In a note in August, Guggenheim Securities analysts called admilparant a $5 billion-plus opportunity for BMS in IPF and progressive pulmonary fibrosis. Admilparant was well tolerated, and discontinuations caused by adverse events were low and similar to placebo, BMS said. References to liver toxicity and a patient death in a protocol amendment recently raised concerns about admilparant’s safety. While BMS made no mention of liver safety in this morning's topline readout, William Blair analysts explained in a note this morning that the pharma's management had confirmed that the case of acute liver injury was in the 120 mg arm, and the adjudication committee had “determined it was unlikely related to study treatment.” Meanwhile, BMS announced that Action-1 had hit its primary endpoint at a planned interim analysis. Progression-free survival was significantly longer on RYZ101 than on investigator's choice of standard of care, teeing up talks with the FDA about the next steps toward a regulatory submission. Again, BMS has yet to share data from the trial. When it came to safety, the drugmaker only said the results were consistent with RYZ101’s known profile. The readout advances BMS’ efforts to establish RYZ101 as an option for patients who progress on Lutathera, one of Novartis’ Lutetium-177 (Lu177)-based radiopharmaceuticals. GEP-NET patients entered Action-1 after progressing on Lu177-based therapy. Lu177 is a beta emitter, while RYZ101 delivers an alpha emitter, actinium-225. Launching admilparant and RYZ101 could give BMS growth drivers at a time when its cornerstone drugs are nearing the ends of their life cycles. Eliquis and Opdivo accounted for more than half of BMS’ sales in the second quarter, but both drugs could lose market exclusivity in the U.S. in 2028. With Cobenfy, the schizophrenia drug BMS acquired through its $14 billion Karuna Therapeutics buyout, yet to make a significant revenue contribution, the drugmaker is under pressure to find other growth drivers. The wins for admilparant and RYZ101 go some way to easing the pressure, but BMS’ path to long-term growth remains dotted with pitfalls. Key readouts on the horizon include the phase 3 Librexia AF trial of milvexian, an oral FXIa inhibitor, in atrial fibrillation. Investors seemed in two minds about this morning's data, initially sending BMS' stock up 3.5% when the markets opened on Monday. But after an hour of trading, the stock was down 3% on Friday's closing price. William Blair analysts attributed the initial stock bump to the win for admilparant, a drug they estimated could reach sales of $1.3 billion for IPF in 2032. “While there will be some lingering questions on dose response and the magnitude of benefit for admilparant from the Aloft-IPF study, we believe the success of the 60 mg dose is likely to open a blockbuster opportunity given we view the novel mechanism and non-overlapping toxicities with current therapies in IPF as positive for combination approaches,” the analysts wrote in a Monday morning note. Where Big Pharma is placing its bets is one of the hot topics being discussed at Fierce Biotech's high-impact event in London next month. Find out more information and register by clicking on the banner below:

iStock, wildpixel A low-dose win could derisk a regulatory pathway for BMS’ LPA1 antagonist in idiopathic pulmonary fibrosis after a recently reported patient death, but a miss at the higher dose leaves analysts awaiting full data. Bristol Myers Squibb’s late-stage lung disease asset has scored in a low-dose cohort while failing to improve exhalation capacity at a higher dose, leaving analysts wanting more. The Big Pharma’s LPA1 antagonist, dubbed admilparant, ultimately met the primary endpoint of an idiopathic pulmonary fibrosis (IPF) study at the 60-mg dose, demonstrating a statistically significant improvement over placebo in patients’ absolute change in forced vital capacity (FVC) from baseline at week 52. But the 120-mg dose failed to reach statistical significance on the same measure. The company didn’t provide the data behind the results. Leerink called the findings “mixed,” writing in a Monday note that the ALOFT-IPF Phase 3 results “could be sufficient to support admilparant regulatory approval, but the lack of dose response raises questions about its profile. “We await detailed results to better understand the drug’s efficacy,” the analyst added. William Blair was more bullish, estimating $1.3 billion in admilparant sales for IPF in 2032. “While there will be some lingering questions on dose response and the magnitude of benefit for admilparant from the ALOFT-IPF study, we believe the success of the 60 mg dose is likely to open a blockbuster opportunity given we view the novel mechanism and non-overlapping toxicities with current therapies in IPF as positive for combination approaches,” William Blair wrote Monday. BMS is still evaluating the data and plans on meeting with heath authorities to “support planned regulatory submissions,” according to its news release. Detailed results from the trial are expected for presentation at an unnamed future medical meeting. The pharma’s stock fell 3% within the first hour of trading on Monday, from $60.11 per share at open to $57.14. Idiopathic pulmonary fibrosis BMS reports liver injury events in lung disease program, with key readout near Bristol Myers Squibb will soon release Phase 3 data for admilparant that could make or break Contineum Therapeutics’ rival product. But reports of a death due to liver injury in the pharma’s program have caused some unease about the drug class. October 1, 2026 · 3 min read · Annalee Armstrong Read more As for safety, BMS said the drug was well-tolerated across both doses in ALOFT-IPF and noted that discontinuations due to adverse events were comparable to placebo. The Big Pharma did not provide details on safety events, such as hypotension or liver toxicity risk. Earlier this month, BMS reported a death due to multi-organ failure after acute liver injury in its ALOFT program, which includes another Phase 3 trial for progressive pulmonary fibrosis that is expected to read out early next year. BMS management told William Blair that the deceased patient was in the 120-mg admilparant arm, and an adjudication committee determined the death was unlikely related to study treatment, according to a Monday note from the analysts. A limited number of liver events were observed in the ALOFT program, BMS told BioSpace earlier this month. Most were mild, transient and linked to other medical conditions or in patients taking other medications that have known liver risk factors. “A key concern recently has been around liver safety and we think the data reported showing admilaparant [sic] was well tolerated across both doses with AE associated discontinuations low and similar to [placebo] on both active arms addressed this overhang,” Truist wrote Monday morning. Business BMO most bullish on Merck’s, bearish on BMS’ plans to contend with patent cliffs BMO Capital Markets is more impressed by Merck’s plans to replace lost Keytruda revenue than on Bristol Myers Squibb’s efforts to fill the expected void that will come from loss of exclusivity for both Opdivo and Eliquis. October 2, 2026 · 4 min read · Gabrielle Masson Read more “All in, today’s update points to a derisked regulatory submission path for Bristol pending full data release,” BMO Capital Markets’ Evan Seigerman and colleagues wrote in a Monday investor note. “Importantly, the result validates the LPA1 mechanism in a large registrational study; however, the data are unlikely to be viewed as entirely clean given the 120mg arm slowed FVC decline but failed to achieve statistical significance,” BMO said. Pulmonary fibrosis is a chronic, life-threatening disease that occurs when lung tissue becomes irreversibly damaged, impacting the organ’s function. William Blair noted that BMS’ trial win is likely a positive for Contineum Therapeutics, which is also developing an LPA1 for pulmonary fibrosis and chronic pain. As for BMS’ admilparant, the next step will be about “understanding the magnitude of benefit, consistency across subgroups and the explanation for the unexpected dose-response relationship,” the BMO group wrote Monday. “Taken together, we view ALOFT-IPF as an incremental positive as we set up for a catalyst rich period,” the analysts added. The Big Pharma is expecting a Cobenfy readout in Alzheimer’s disease psychosis in early 2027, plus topline results next year for milvexian, a Johnson & Johnson–partnered investigational oral factor XIa inhibitor, in atrial fibrillation. Last month, Seigerman told BioSpace that he was bearish on BMS’ plans to restock its pipeline as exclusivity loss looms for best-selling drugs Opdivo and Eliquis. “We remain on the sidelines as we continue to be wary of the binary risk setup for one of our major pharma names,” he wrote Monday. Radiopharma BMS’ $4.1B RayzeBio bet delivers Phase 3 radiopharma win The centerpiece of Bristol Myers Squibb’s 2024 RayzeBio buy has read out, hitting the primary endpoint in a late-stage tumor trial. October 12, 2026 · 2 min read · Gabrielle Masson Read more BMS shared the IPF data on the same day it announced a Phase 3 win for RYZ101 , the centerpiece of its $4.1 billion RayzeBio acquisition . The investigational radiopharmaceutical significantly improved progression-free survival for patients with gastroenteropancreatic neuroendocrine tumors.