bioinnovationbeacon.com

Author: Gabrielle Masson

  • BMS’ Phase 3 win for lung drug clouded by high-dose miss

    BMS’ Phase 3 win for lung drug clouded by high-dose miss

    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.

    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

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    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.

    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

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    “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.

    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

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    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.

  • BMS’ $4.1B RayzeBio bet delivers Phase 3 radiopharma win

    BMS’ $4.1B RayzeBio bet delivers Phase 3 radiopharma win

    Bristol Myers Squibb’s experimental radiopharmaceutical therapy significantly improved progression-free survival in a late-stage trial for gastroenteropancreatic neuroendocrine tumors, a rare type of cancer that typically forms in the gastrointestinal tract.

    BMS picked up the radiopharma candidate, known as RYZ101, in its $4.1 billion RayzeBio acquisition announced at the end of 2023 and closed in early 2024.

    Now, the Big Pharma said that an interim analysis of the Phase 3 portion of the study has met its main goal, with a statistically significant and clinically meaningful improvement in progression-free survival (PFS), as measured by blinded independent central review. BMS did not provide data behind the win in its Monday news release.

    The ACTION-1 study included patients with advanced somatostatin receptor–expressing gastroenteropancreatic neuroendocrine tumors (GEP-NETs) that progressed after treatment with Novartis’ Lutathera, a targeted radiopharmaceutical therapy that serves as the condition’s current standard of care. The primary endpoint was to be measured after 143 PFS events occurred, according to ClinicalTrials.gov.

    RYZ101 is made with actinium-225, a radioisotope that is teed up to be the next big thing in radiopharma—to the point of straining supply of the isotope. The high demand even prompted a pause in the enrollment of ACTION-1 back in 2024, shortly after BMS’ acquisition of RayzeBio was completed.

    Suppliers are investing in production to support deals with AstraZeneca, Bayer and other drugmakers that are advancing radioisotope-based cancer therapies.

    February 17, 2026

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    RYZ101 secured orphan drug status from the FDA in August based on significant unmet need, BMS said. The designation paired with the interim results support further development efforts and “plans to engage with the FDA to discuss next steps toward a regulatory submission,” according to the pharma.

    Safety results were consistent with the known RYZ101 profile, BMS said. In a previous Phase 1b test in 17 patients, RYZ101 was well tolerated, with no treatment-related serious adverse events or dose discontinuations due to adverse events reported.

    More detailed findings from ACTION-1 are slated for presentation at an upcoming medical meeting, the New Jersey company said.

    Telix is Regeneron’s entry ticket into the radiopharma game, helping to better round out the company’s cancer portfolio, according to Truist Securities.

    April 13, 2026

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    “These are the first results from a Phase 3 trial of an alpha-isotope based targeted RPT (225Ac) in any indication, and we are moving with urgency to bring these data to the FDA and, ultimately, provide a novel option for patients,” RayzeBio Chief Medical Officer Amreen Husain said in the release.

    GEP-NETs have a global prevalence of fewer than 200,000 patients. While current lutetium-177–based radiopharmaceutical therapies such as Lutathera have shown significant clinical benefit for GEP-NETs, most patients progress following treatment, according to BMS.

    RYZ101 is also being evaluated in earlier-stage trials for hormone receptor–positive metastatic breast cancer.

  • TRexBio, Retension raise $161.7M in IPOs; City, Iambic set proposed offering terms

    TRexBio, Retension raise $161.7M in IPOs; City, Iambic set proposed offering terms

    The biotech IPO frenzy is very much alive this October spooky season, with TRexBio and Retension Pharmaceuticals set to begin trading Friday with offerings totaling $161.7 million. City Therapeutics and Iambic Therapeutics are lined up behind them, outlining expected prices for their own offerings.

    First up is TRexBio, hitting the Nasdaq with a $116.7 million offering. The immunoregulatory medicine developer sold 8.3 million common stock shares at $14 per share, coming in at the low end of its expected $14 to $16 range. Underwriters also have a 30-day option to buy 1.25 million more shares at the same price.

    Big Pharma Eli Lilly reported direct holdings of 5.4 million TRex shares, according to a Thursday SEC filing. The biotech’s CEO is Johnston Erwin, who spent nearly 20 years at Lilly before leading TRex. In 2023, TRex and Lilly linked up in a deal worth more than $1.1 billion in biobucks to develop treatments for immune-mediated diseases.

    Now, TRex will use the new funds to advance its investigational fusion protein TRB-061, currently in early-stage development for atopic dermatitis. A topline Phase 1b readout is expected in mid-2027. TRex will also put some of the money into the preclinical CD30 agonist TRB-071.

    Twenty-five biotechs have used the IPO route to go public this year—with five of those debuts occurring this month alone. It’s the most IPOs since 2021, when the industry recorded 78 during the height of the COVID-19 pandemic.

    August 25, 2026

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    Joining TRex on the markets Friday is Retension Pharmaceuticals, which will debut with $45 million. The cardiovascular-focused company sold 3.75 million shares at $12 a piece—the midpoint of its expected price range. Underwriters also have the option to purchase up to 562,500 more shares at the IPO price over the next 30 days.

    The Virginia biotech will use the proceeds to back its sole clinical candidate: RTN-001, a daily oral small-molecule PDE-5 blocker being developed for hypertension. The money will support ongoing Phase 2b development and the advancement to Phase 3, according to Retension.

    Including the two newest additions, 29 biotechs have gone public in 2026, an incredible leap from 2025’s total of eight public biotech debuts. Already this year, the industry has had its most IPOs since 2021, when biopharma recorded 78 at the height of the COVID-19 pandemic.

    Up next to potentially go public is RNAi biotech City Therapeutics, which filed IPO plans at the end of September and just revealed that it expects to raise around $158.3 million, according to a Friday SEC filing. That estimate is based on an expected pricing at $18 per share.

    Founded in 2023 by former Alnylam leader John Maraganore, the biotech plans on divvying up the cash, with $30 million expected to bankroll the development of CITY-FXI, which is currently being evaluated in a Phase 1 trial for thrombosis, while also launching a Phase 2 trial to assess the asset’s efficacy in preventing blood clotting in knee replacement surgery.

    Another $20 million will fuel clinical development of RNAi therapy CITY-RBP4 in Stargardt disease and geographic atrophy, while $40 million is intended to fund the clinical tests of CITY-TFR2 in anemia associated with myelofibrosis. The biotech has also earmarked $70 million for platform development and preclinical activities.

    Two RNA biotechs, ADARx Pharmaceuticals and City Therapeutics, are taking advantage of the IPO frenzy, which has led to a total of 27 newly public biotechs so far this year.

    September 25, 2026

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    Lastly, AI-fueled Iambic Therapeutics has targeted a $15 to $17 per share range for an expected $135 million IPO. The Nvidia-backed cancer company has allotted $75 million of that money for its lead candidate IAM1363, an oral HER2 inhibitor designed to penetrate the brain and target several known disease-causing mutations of the protein. IAM1363 is currently being studied in a Phase 1/1b trial for HER2-altered solid tumors, with registrational studies planned for 2027.

    Some of the money will also go toward moving the KIF18A blocker IAM217 for triple-negative breast cancer and the dual CDK2/4 inhibitor IAM-C1 into clinical testing.

  • Argenx shutters Sjogren’s study, losing blockbuster expansion opportunity for Vyvgart

    Argenx shutters Sjogren’s study, losing blockbuster expansion opportunity for Vyvgart

    Argenx is discontinuing a late-stage study of Vyvgart Hytrulo in Sjogren’s disease due to futility, a surprise setback that removes “the next potential blockbuster expansion opportunity” for the drug, William Blair analysts wrote on Thursday.

    The biopharma is ending its Phase 3 UNITY trial after an independent data committee determined that the study would not meet its primary endpoint of improving systemic disease activity, according to a Thursday release.

    As of market open, argenx’s stock had tumbled nearly 14%, or about $130 per share, resting at around $797 as of 11 a.m. ET.

    UNITY had been evaluating argenx’s Vyvgart Hytrulo injection for Sjogren’s, an autoimmune disorder that most often affects moisture glands, with the most common symptoms of dry eyes and dry mouth. Vyvgart Hytrulo is currently approved to treat generalized myasthenia gravis and chronic inflammatory demyelinating polyneuropathy and recently scored a late-stage win in autoimmune myositis, a group of rare diseases that cause the immune system to attack the muscles.

    Today’s futility announcement is surprising given that the trial results weren’t expected until the second half of 2027, William Blair wrote in a Thursday note.

    Argenx told the firm that the futility analysis included data from more than 500 patients—as compared to the 630 enrolled—indicating that the review may have been conducted earlier than the primary endpoint assessment expected at week 48.

    “Sjogren’s disease drug development has been bogged down by high placebo response rates, making it difficult to achieve robust separation versus placebo,” according to William Blair.

    In argenx’s Phase 2 RHO study, the asset appeared differentiated in Sjogren’s compared to placebo at week 24. However, the study was underpowered and there was no difference compared to placebo in percentage of patients achieving at least a three-point improvement on an industry scale measuring disease activity, William Blair wrote.

    The analysts had previously estimated peak sales of $1.4 billion in Sjogren’s but noted competition in the space, specifically from Novartis’ investigational monoclonal antibody ianalumab and Amgen’s CD40L antagonist fusion protein dazodalibep, both of which have recorded Phase 3 wins.

    The star of the takeover is Forte Biosciences’ investigational antibody therapy, FB102, which has shown early potential in vitiligo and celiac disease.

    July 27, 2026

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    Meanwhile, argenx announced a mid-stage celiac disease win in a separate release. The Phase 2 trial evaluated the biotech’s investigational CD122 inhibitor FB102, which it picked up from Forte Biosciences in a $2.2 billion acquisition this summer.

    Patients receiving FB102 demonstrated a statistically significant change from baseline in a health measure of the small intestine, meeting the study’s primary endpoint. Given the findings, which argenx heralded as the “first Phase 2 evidence that blocking CD122 can prevent gluten-induced intestinal damage in people living with celiac disease,” the biopharma plans to move the asset into late-stage development.

    The celiac disease plans expand argenx’s late-stage pipeline ahead of the closely-watched Phase 3 findings expected soon for monoclonal antibody empasiprubart in multifocal motor neuropathy.

  • Newron reveals 5 deaths in late-stage schizophrenia program as FDA maintains hold

    Newron reveals 5 deaths in late-stage schizophrenia program as FDA maintains hold

    Four deaths have occurred among patients receiving Newron Pharmaceuticals’ investigational schizophrenia drug, the biotech has revealed, three of which are considered unrelated to the asset.

    In April, the FDA placed a hold on Newron’s Phase 3 ENIGMA-TRS 2 trial evaluating glutamate modulator evenamide as an adjunctive therapy for schizophrenia, barring further patient enrollment in the U.S. as the agency evaluated one patient death. The death occurred at a trial site abroad and was deemed unrelated to evenamide, Newron said at the time, though the hold remains in place.

    The FDA cited concern about evenamide’s mode of action as a selective voltage-gated sodium channel blocker, according to a Thursday release from Newron. Certain sodium channel blockers may cause irregular heartbeats that can lead to serious cardiovascular events or seizures, the company noted.

    Now, Newron has shared that four total deaths have occurred in patients receiving evenamide across all clinical studies to date, or an incidence of 0.56%—compared to one death among patients on placebo, or a 0.27% incidence.

    Study investigators consider three of the four deaths in the investigational cohort unrelated to treatment, while one may be “possibly related” because a definitive cause of death was not determined after two autopsies.

    Newron is reviewing the FDA’s feedback and all safety data across the development program, which includes more than 10,000 ECGs from about 700 patients receiving evenamide.

    The patient death occurred outside the U.S. and was deemed unrelated to Newron Pharmaceuticals’ investigational schizophrenia drug.

    April 30, 2026

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    The Italian drugmaker said it hasn’t observed a pattern of cardiac abnormalities or arrhythmias in the preclinical and clinical safety data generated so far, which includes more than 5,000 ECGs from over 700 patients, according to the biopharma.

    Newron is also assessing the addition of further cardiac screening and monitoring measures for the program, which includes the late-stage ENIGMA-TRS 1 and ENIGMA-TRS 2 trials. The former is an international, 52-week Phase 3 trial expected to enroll at least 600 patients across 20 countries by mid-October, while ENIGMA-TRS 2 is designed to be a 12-week study taking place in the U.S. and other select countries.

    Given the FDA hold, no U.S. patients in ENIGMA-TRS 2 have received evenamide. Recruitment for the study continues outside the U.S.

    “We remain confident in the ENIGMA-TRS studies and encouraged by the program’s progress,” Newron Chief Medical Officer Ravi Anand said in a prepared statement. “Our belief in the potential of evenamide and its novel mechanism for patients with treatment-resistant schizophrenia remains strong.” Patient safety is Newron’s “utmost priority,” Anand added.

    The company’s stock was down slightly in pre-market trading. Newron’s leadership team is expected to provide a brief update about the program on Thursday at 9:30 a.m. ET.

  • ArriVent sheds nearly half of value on late-stage lung cancer miss

    ArriVent sheds nearly half of value on late-stage lung cancer miss

    ArriVent BioPharma’s investigational tyrosine kinase inhibitor failed to significantly improve progression free survival rates in a Phase 3 lung cancer trial, sending the biotech’s stock spiraling.

    ArriVent’s Phase 3 study, called FURVENT, enrolled nearly 400 patients with advanced non-squamous non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations (Ex20ins). The open-label study assessed ArriVent’s most advanced asset firmonertinib, an EGFR TKI designed to penetrate the brain.

    Firmonertinib, which ArriVent shares with Shanghai-based Allist Pharmaceuticals, failed to meet FURVENT’s main goal, recording a median progression free survival (PFS) of 11 months compared to 9.5 months for patients on chemotherapy, as measured by blinded independent central review (BICR). The finding was not statistically significant, according to ArriVent’s Tuesday release.

    Truist analysts view the miss as largely fueled by a stronger-than-expected PFS seen in the control group receiving chemotherapy. The cohort’s median 9.5-month survival rate was up from an expectation of seven months, according to the firm.

    “With these results, we see the path forward in Ex20ins as uncertain as mgmt reviews the full dataset and potential development options,” Truist wrote in a Tuesday note to investors. The analysts therefore removed Ex20ins NSCLC from its valuation of ArriVent, lowering its expected price target from $45 per share to $18.

    Analyst sentiment aligned with the market’s response to the data, with ArriVent’s share price plummeting nearly 50%, from $28.46 to $12.09 at market close Tuesday.

    Delivering therapeutics directly to tumors, as in the case of Replimune’s recently approved Tudriqev and Amgen’s Imlygic, holds advantages, especially when cancer is caught early, but challenges remain before the method becomes a mainstay in treatment.

    September 21, 2026

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    ArriVent pointed to clinical benefit as measured by secondary endpoints, like the investigator-assessed 11.1 month PFS for firmonertinib compared to 7.1 months for chemotherapy.

    The difference in investigator-assessed findings compared to BICR underscored “a meaningful discordance in control-arm assessment,” Truist wrote.

    No new safety signals were identified in the trial, with findings consistent with previous firmonertinib studies.

    Truist is now turning its attention to ALPACCA, an ongoing Phase 3 study of firmonertinib for patients with EGFR PACC mutant NSCLC. The firm sees limited readthrough from FURVENT to ALPACCA, believing that this week’s findings still support the therapy’s potential to produce a competitive efficacy profile against the Tagrisso/Gilotrif control.

    ArriVent launched in June 2021 with $90 million and ex-China rights to furmonertinib from Allist. The drug had received conditional approval from China regulators earlier that year for patients with advanced NSCLC and a confirmed EGFR T790M mutation that had progressed during or after EGFR TKI therapy.

    With an eye toward advancing a novel antibody-drug conjugate for gastrointestinal cancers, ArriVent is the latest biopharma player to ink a deal with a Chinese biotech.

    January 22, 2025

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    Truist believes the FURVENT outcome “resets the story with a cleaner setup” for ArriVent’s antibody-drug conjugate ARR-217, which is slated as a late-breaker at this year’s European Society for Medical Oncology in Spain toward the end of October. The asset is being developed in partnership with China’s Lepu Biopharma for colorectal cancer and gastrointestinal cancers. The Phase 1 data in colorectal cancer could help build the case for ARR-217 to rebuild pipeline value.

    “With FURVENT now behind us, we see ARR-217’s late-breaking Ph.1 data at ESMO as a cleaner opportunity to reshape conversation around AVBP’s ADC pipeline,” Truist wrote on Tuesday.

  • FDA extends review of Novo’s hemophilia A drug, citing manufacturing issues

    FDA extends review of Novo’s hemophilia A drug, citing manufacturing issues

    The FDA needs more time to review Novo’s investigational bispecific antibody for hemophilia A before making an approval decision, citing manufacturing issues as the reason behind the delay.

    The agency said “ongoing facility remediation activities” were the reason for the extension, according to a release from Novo. The FDA did not identify any deficiencies related to the efficacy or safety of the drug, known as denecimig, according to the Danish pharma.

    Novo submitted the next-gen factor VIII mimetic for FDA review in September 2025, with a decision slated for the third quarter of this year. Novo announced the agency’s delay on the second day of the fourth quarter.

    The FDA has yet to share a new timeline for the review, according to Novo, which is working to address the agency’s remediation requests related to the unnamed manufacturing site.

    The FDA’s manufacturing flag does not impact other marketed Novo products, the pharma said. Novo is still banking on FDA approval, saying the company is still preparing for a 2027 denecimig launch for hemophilia A in adults and children.

    “Novo is already in the process of addressing the FDA’s requests as efficiently as possible and continuing to work closely with the Agency toward bringing denecimig to patients in the US,” Novo CEO Mike Doustdar said in a prepared statement.

    Among the problems cited were cat hair, bacterial contamination and instrument defects.

    August 22, 2025

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    The extended FDA review for denecimig “has no impact on Novo’s financial outlook for 2026,” according to the company. Analysts with BMO Capital Markets listed the denecimig decision as one of seven key catalysts for Novo, deeming it of “medium” importance in a Sept. 9 report.

    Novo has been testing denecimig as a prophylactic treatment to prevent bleeding episodes for hemophilia A, a genetic bleeding disorder. The program is also currently being evaluated for potential approval by European regulators.

    Novo has faced other manufacturing challenges of late, stemming from Novo Holdings’ $16.5 billion buy Catalent, a contract development and manufacturing organization (CDMO) in 2024. It then sold three of Catalent’s fill-finish sites to the pharma company then called Novo Nordisk to help meet demand for its GLP-1 injectables Ozempic and Wegovy. Novo also inherited customers that Catalent made products for.

    Earlier this year, quality problems at the ex-Catalent fill-finish facility in Indiana prompted Scholar Rock, an affected client, to drop the plant from its application for approval of apitegromab, a candidate to treat spinal muscular atrophy. The FDA had designated the plant with an Official Action Indicated (OAI) classification, which indicates recommended regulatory or administrative actions in response to an unacceptable state of compliance.

    After dropping the plant from its filing, Scholar Rock secured FDA approval, with apitegromab now to be sold as Isembyld.

    Despite earlier claims of improvement at the fill-finish plant, the FDA has classified the facility as Official Action Indicated based on inspection findings.

    August 11, 2026

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  • US government launches AI-driven programs to overhaul clinical trials

    US government launches AI-driven programs to overhaul clinical trials

    Agentic twins. Phaseless design engine. Fewer trial participants. All these are part of the U.S. government’s new efforts to secure global leadership in clinical research.

    Unveiled by the Advanced Research Projects Agency for Health (ARPA-H), the initiative is designed to speed up clinical trials and slash patient burden, according to a Wednesday release from the U.S. Department of Health and Human Services (HHS).

    The new program will be known as Simulation-augmented, Real-time Platform Adaptive Seamless Trials, or SURPASS. The initiative will incorporate advanced computational models, real-time analysis and automation “to evaluate drugs and biologics faster, at lower cost, and with fewer participants,” according to the government release. “By enabling more efficient designs, the program will allow more patients to participate in trials that are more likely to benefit them.”

    The effort comes the same week that President Donald Trump signed an executive order to establish an AI-powered chatbot to streamline user experience across government services.

    The new ARPA-H program will focus on three key areas, the first of which is building “a phaseless design engine for faster trials with fewer patients” by integrating digital twins and other predictive models into trial design and simulating outcomes before launch.

    Newly permanent CBER and CDER directors Karim Mikhail and Michael Davis are widely expected to continue the FDA’s efforts toward an adaptive framework for rare disease therapies. If approved by the Senate, will commissioner nominee Heidi Overton stay the course?

    September 21, 2026

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    SURPASS will also develop “a continuous inference engine for faster decisions . . . reducing the need for large conventional control groups while maintaining rigorous evidence generation.”

    Lastly, SURPASS is to “create an agentic operations layer” that will automate trial startup and operational activities, supporting onboarding for new treatment arms and speeding up data collection and cleaning.

    “Today’s clinical trial system often requires too many stops, too much duplicated infrastructure, and too much time before researchers can understand whether a trial is on the right track,” SURPASS program manager Daria Fedyukina said in a prepared statement. “SURPASS is developing the technology and biostatistical frameworks needed to analyze trial data as it accumulates, allowing teams [to] learn in real time, adapt when needed, and generate stronger evidence faster with less operational burden.”

    Beyond SURPASS, ARPA-H has also announced three other complementary projects aimed at removing barriers to clinical development.

    The FDA has so far secured 600 new hires and is looking for 1,600 more as interim leadership at the agency aims to rebuild the workforce and morale after more than a year of intense attrition.

    June 24, 2026

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    One project, dubbed STACK, will use AI to “accelerate clinical site activation and convert research-naïve sites into clinical trial sites capable of running studies.” The goal is to improve patient accessibility to these studies.

    Meanwhile, COMMONS will create a national consent architecture to enable data access across the U.S. clinical trial ecosystem.

    Then there’s CINCH, which is focused on “patient empowerment and care navigation” by helping patients contribute their own real-world data.

    “America should lead the world in turning medical breakthroughs into treatments for patients,” said HHS Secretary Robert F. Kennedy Jr. “HHS is taking on the delays, duplication, and unnecessary costs that slow clinical trials down.”

    Launched by the Biden administration in 2022, ARPA-H initially secured $1 billion from Congress to bankroll high-potential, high-impact biomedical research. In February 2025, inaugural ARPA-H leader Renee Wegrzyn was let go by the Trump administration, according to Fierce Biotech. In October, Evernow CEO Alicia Jackson was tapped to lead the unit.

    The latest ARPA-H efforts come as the U.S. “risks losing clinical trial leadership and the economic activity that comes with it,” according to the Wednesday release.

    Industry concerns have grown louder over the past few years about other countries’ increasing prowess in biotech—particularly China. Six years ago, the Asian country surpassed the U.S. in terms of the number of clinical trials, with the U.S. also falling behind in R&D funding and translational science since then.

    “China’s approach has been to steal, scale and strangle American competitors in this space,” Caitlin Frazer, executive director of the National Security Commission on Emerging Biotechnology (NSCEB), told BioSpace in August. “We have to start thinking about what it means if we so erode our innovation base here in the U.S. that we are dependent on a potential adversary for life-sustaining medications.”

    As Chinese companies push beyond “me-too” drugs and the country lays out a new five-year plan targeting a $522 billion–strong biotech industry by 2030, experts say more predictable funding and regulation could help the U.S. maintain its edge.

    September 23, 2026

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    The new ARPA-H rollout follows the HHS-wide Operation TrialBlazer Initiative, which included an FDA pilot program intended to reduce the time and burden to move assets into first-in-human studies. That program opened for applications last month and will remain open until Oct. 30.

    These initiatives come after the FDA downsized by more than 3,000 staffers last year, many of which were layoffs or incentivized early retirements. This year, the agency has said it wants to hire some 2,200 staffers, including some of the recently departed employees.

    The federal agency has undergone massive changes under Trump’s second administration, ranging from leadership shake-ups to widespread cuts. In the summer of last year, the agency rolled out Elsa, an AI tool designed to boost efficiency. At the time, many experts called for the FDA to provide more details about how the model works and what benchmarks, if any, the agency has for evaluating its output.

    The FDA’s clunky launch of Elsa, an AI tool to increase efficiency, has sparked concern from agency employees and outside experts.

    June 30, 2025

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