Pharma Phriday (June 5, 2026)

Written by Duncan Emerton, Pharma Phriday is a comprehensive weekly update on the pharmaceutical industry, covering clinical trial results, regulatory approvals, corporate development activity, and other significant developments. Click below to download a PDF version of this week's note.

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Contents

Artificial Intelligence

• Alnylam and Inceptive forge AI-driven RNAi discovery pact

• Massive Bio and Optellum collaborate on NSCLC clinical trials

• Tempus AI expands its agentic AI, Lens, into oncology R&D

Clinical

• AstraZeneca posts new data for camizestrant in breast cancer

• AstraZeneca posts positive data from its Phase III EMERALD-3 trial

• BMS posts more positive Phase III data for mezigdomide in R/RMM

• BMS/BioNTech’s pumitamig shows encouraging efficacy in NSCLC

• Idorsia reports positive results for its Clostridioides difficile vaccine

• Ivonescimab delivers OS benefit in squamous NSCLC

• Kailera posts early-stage data for its tripleG, KAI-4279, in obesity

• Kelonia posts Phase I R/RMM data for KLN-1010 at ASCO 2026

• Perioperative Erleada reduces risk of metastasis and death in prostate cancer

• Roche to present new obesity portfolio data at ADA 2026

• Virion Therapeutics reports Phase Ib data for VRON-0200 in HBV

Regulatory

• AbbVie’s Decnupaz gets FDA nod for ultra-rare BPDCN

• FDA accepts Roche’s NDA for giredestrant in breast cancer

• FDA accepts Vertex’s BLA for povetacicept in IgAN

• Imfinzi gets FDA nod for BCG-naïve, high-risk NMIBC

Commercial

• Ascidian and Eli Lilly collaborate on RNA Exon R&D

• Haisco and Eli Lilly announce broad licensing and R&D collaboration

• Hanmi licenses sonefpeglutide to Eli Lilly for up to $1.260 billion

• Pfizer and Innovent announce global oncology R&D collaboration

• Servier to pay up to $2.65 billion for Edgewise’s sevasemten

• Travere licenses civorebrutinib from Everest

In Other News

• A new drug concept to treat obesity and type 2 diabetes

• AbbVie’s Aquipta approved in Europe

• CytomX expands bsAbs collaboration with Regeneron

• Dr Falk and Renexxion move naronapride into late-stage trials

• Eli Lilly’s Retevmo shows benefit in RET fusion+ NSCLC

• Gilead’s Livdelzi shows ALP normalisation benefit in PBC

• J&J’s Rybrevant Faspro shows benefit in advanced head/neck cancer

• J&J’s Tremfya gets FDA label expansion in PsA

• MannKind gets FDA nod for Afrezza in adolescents aged 6+ years

• Microsoft premieres Copilot Health

• Mirum announces new data for its liver disease portfolio at EASL 2026

• Novartis’s Pluvicto shows consistent efficacy across mHSPC sub-groups

• Pfizer’s Braftovi regimen nearly doubles mean PFS in mCRC

• Prices for branded drugs rise in the US but fall in others

• Revolution Medicines gets standing ovation at ASCO 2026

• Samsung Bioepis launches Eylea biosimilar across Europe

Summary

Artificial Intelligence

Alnylam and Inceptive Nucleics announced a three-year AI collaboration valued at up to $2 billion, pairing Alnylam's 20-plus years of proprietary siRNA data with Inceptive's biology foundation models to accelerate RNAi drug design. Inceptive was co-founded by the lead author of the transformer architecture paper that underpins modern AI. Tempus AI launched the next generation of its Lens agentic platform, integrating 8.5 million de-identified patient records with auditable AI workflows for oncology drug development. Massive Bio and Optellum integrated their platforms to route early-stage NSCLC patients flagged by AI-powered CT analysis directly into clinical trial matching.

Clinical

Conference data readouts dominate the clinical section. Ivonescimab delivered a statistically significant OS benefit over tislelizumab plus chemotherapy in squamous NSCLC (HR 0.66), the first such result against an anti-PD-1 combination in a Phase 3 trial. Selpercatinib reduced recurrence or death by 83% in adjuvant RET fusion-positive early-stage NSCLC (HR 0.17). Mezigdomide plus carfilzomib and dexamethasone reduced progression or death by 52% versus carfilzomib and dexamethasone alone in relapsed or refractory myeloma. Perioperative apalutamide reduced metastasis or death by 20% and extended time to subsequent therapy to more than six years in high-risk localised prostate cancer. Camizestrant's updated SERENA-6 data showed a 55% PFS benefit and 37% PFS2 benefit, with 51% of patients achieving ctDNA clearance. AstraZeneca's EMERALD-3 HCC trial met its primary PFS endpoint.

Regulatory

Imfinzi received FDA approval for BCG-naïve high-risk NMIBC, completing AstraZeneca's bladder cancer franchise across all disease stages. The FDA accepted Roche's giredestrant NDA for adjuvant ER-positive breast cancer and Vertex's povetacicept BLA for IgAN, both with November 2026 PDUFA dates. AbbVie's Decnupaz received approval for BPDCN.

Commercial

Eli Lilly announced three deals: a $1.9 billion RNA exon editing collaboration with Ascidian for monogenic kidney diseases, a multi-target licensing arrangement with Haisco across several therapeutic areas, and a $75 million upfront licensing deal with Hanmi for sonefpeglutide. Pfizer and Innovent entered a 12-programme oncology collaboration worth up to $10.5 billion. Servier acquired Edgewise's sevasemten franchise for up to $2.65 billion. Travere licensed civorebrutinib from Everest for $112.5 million upfront.

In Other News

Revolution Medicines' daraxonrasib received a standing ovation at ASCO for its OS benefit in pancreatic cancer (HR 0.40). Roche's giredestrant NDA acceptance and J&J's perioperative apalutamide data both signalled expansion of targeted therapy into earlier disease settings. Drug pricing data confirmed a sustained transatlantic divergence, with US branded drug prices rising 81% post-launch versus a 13% fall across 19 other high-income countries.

Artificial Intelligence

Alnylam and Inceptive forge AI-driven RNAi discovery pact

What happened? Alnylam and Inceptive Nucleics (Inceptive) have announced a three-year strategic collaboration valued at up to $2 billion, with $30 million upfront in cash and equity in the startup. Additional payments are contingent on co-discovered drugs achieving preclinical, regulatory, and commercial sales milestones. The collaboration seeks to advance siRNA design by modelling target mRNAs, exploring sequence space and novel chemical modifications to enhance potency and efficacy, and predicting top-performing therapeutic candidates for further development by Alnylam. Inceptive was founded in 2021 by Jakob Uszkoreit, who co-authored the June 2017 "Attention is all you need" paper while at Google that laid the foundational architecture for modern AI models, including GPT.

What does this mean? The core design challenge in siRNA development is optimising the oligonucleotide sequence for potency, specificity, stability and delivery. Alnylam's 20+ years of proprietary siRNA experimental data, generated across several approved drugs and dozens of pipeline programmes, is exactly the kind of domain-specific, high-quality data set that a foundation model like Inceptive’s needs to be genuinely useful rather than pattern-matching on public data. The "without retraining" claim is interesting. Most AI models in drug discovery are trained for a specific task (e.g., predicting binding affinity, or ADMET properties, or off-target effects) and can’t generalise across modalities without separate training runs. A model that can adapt from characterisation à design à sequence optimisation without retraining would represent a meaningful advance over the current state of the field.

Massive Bio and Optellum collaborate on NSCLC clinical trials

What happened? Massive Bio and Optellum are integrating their two AI platforms to connect early lung cancer detection with clinical trial access. Optellum's LCP AI, a validated, FDA-cleared SaaMD that analyses CT imaging to flag high-risk lung nodules before a formal diagnosis is confirmed, feeds de-identified patient signals into Massive Bio's AI trial matching platform, which then identifies geographically accessible, active NSCLC trials and delivers actionable reports directly to treating physicians. No financial terms are disclosed.

What does this mean? Most oncology clinical trials, including ones for NSCLC, struggle to enrol patients quickly enough. Not because eligible patients don’t exist, but because identification and referral happen too late or not at all. Most patients are referred to trials after multiple lines of therapy have failed. Early-stage patients, the ones who are the most likely to benefit from curative-intent trial participation and whose outcomes data would be most informative for the field, are rarely flagged or referred to trials at the point of initial detection. The Massive Bio/Optellum integration attempts to close that gap by flagging when a high-risk CT finding has been made but a formal diagnosis hasn’t yet been confirmed. This is before patients have started SoC therapy, before they have developed preferences or inertia about treatment, and before the narrow eligibility windows of early-stage trials have closed.

Tempus AI expands its agentic AI, Lens, into oncology R&D

What happened? Tempus has launched the next generation of its Lens agentic AI platform for oncology drug development. The updated platform integrates Tempus' multimodal RWD library, which covers more than 8.5 million de-identified patient records, with oncology foundation models, validated AI agents, and scientific workflows into a single end-to-end system. It’s commercially available and is described as already used by 19 of the top 20 largest biopharma companies. Key capabilities include a co-scientist agent that generates research plans from plain-language hypotheses, executes analysis in code against the full dataset, and delivers reproducible results with full audit transparency.

What does this mean? This announcement aligns with Tempus’ recent expanded collaboration with BMS, which named Lens as the platform being deployed across BMS' clinical development programmes. The most substantive element here is the reproducibility and audit transparency feature, which is the ability to toggle to a code view and inspect the underlying analytical logic. This is not a standard feature of AI platforms, and it addresses the most legitimate concern that pharmaceutical development teams and regulators have about AI-generated evidence: it’s opaque, can’t be interrogated, and therefore can’t be trusted for regulatory submissions or critical development decisions. Making the analytical logic fully auditable in code distinguishes Lens from more black-box AI systems. The value of RWD for trial design support, biomarker validation, and patient subgroup characterisation is directly proportional to the breadth and depth of the underlying dataset. Whether the 8.5 million records are sufficiently representative, longitudinally complete, and well-annotated for the specific questions being asked remains to be seen. Volume is necessary but not a sufficient condition for quality. Tempus hasn’t publicly disclosed the composition of the dataset in enough detail to evaluate that question independently. The "co-scientist" framing is the language of a company positioning its product as a reasoning partner rather than a search tool. Whether that characterisation is accurate or aspirational depends on the quality of the hypothesis generation in practice. Placed in the broader pharma/AI landscape, this announcement is the commercial “productisation” side of a trend being seen elsewhere.

Clinical

AstraZeneca posts new data for camizestrant in breast cancer

What happened? AstraZeneca has presented a further data cut from the Phase III SERENA-6 trial, this time with longer follow-up. Please click here for the core design of the trial. The updated data adds a few additional elements. First, camizestrant delayed time to first progression by 55%, confirming and extending the PFS benefit with longer follow-up. Second, it demonstrated a statistically significant 37% delay in second progression-free survival (PFS2), meaning the benefit extends beyond the first treatment line and continues into the subsequent therapy. ctDNA data were also presented. Camizestrant in combination with a CDK 4/6 inhibitor produced a 99% median reduction in total ctDNA versus a 64% increase in patients remaining on SoC. 51% of camizestrant patients achieved total ctDNA clearance versus 1.9% on SoC, and early total ctDNA clearance was associated with improved long-term outcome.

What does this mean? In previous notes it’s been discussed that the FDA ODAC’s core issue with SERENA-6 was whether they were satisfied that a PFS benefit translated into clinical meaningfulness, in the absence of mature OS data, was sufficient for approval. The ODAC voted 6-3 against, citing concerns about whether PFS improvement from a ctDNA-guided pre-progression switch reflects genuine long-term benefit or statistical artefact. The PFS2 data directly address that concern. PFS2 measures from randomisation to progression on the next line of therapy, so it captures whether the 1L treatment advantage carries through into the subsequent treatment course. A 37% reduction in PFS2 risk, if confirmed, argues that the benefit seen in PFS1 is a genuine disruption of disease trajectory. The association between early ctDNA clearance and improved long-term outcome, if that correlation holds in larger datasets, would provide a surrogate marker pathway for future regulatory submissions and could ultimately be used as a primary endpoint in subsequent trials. The press release notes that total ctDNA clearance may serve as a new biomarker for deep disease control. This updated data with longer follow-up, PFS2 benefit, and ctDNA clearance provides a more complete and arguably more compelling evidence package. AstraZeneca has stated its intention to discuss the SERENA-6 data with the FDA, and the PDUFA date extension gives time to incorporate the longer follow-up dataset into the regulatory review.

AstraZeneca posts positive data from its Phase III EMERALD-3 trial

What happened? EMERALD-3 is a global Phase III trial of N=760 patients evaluating the STRIDE regimen (single priming dose of tremelimumab 300mg followed by regular-interval durvalumab) with or without lenvatinib, combined with TACE (transcatheter arterial chemoembolisation), versus TACE alone in patients with unresectable embolisation-eligible hepatocellular carcinoma (HCC). Patients were randomised 1:1:1 across three arms: (1) STRIDE plus lenvatinib plus TACE, (2) STRIDE plus TACE, and (3) TACE alone. In a planned interim analysis, STRIDE plus lenvatinib plus TACE demonstrated a 30% reduction in the risk of disease progression or death versus TACE alone (HR 0.70), meeting the primary PFS endpoint with statistical significance. An OS trend in favour of the combination was also observed at this interim, though OS was not formally tested at this analysis. The STRIDE-plus-TACE arm without lenvatinib showed strong trends toward improved PFS and OS but was not formally tested at this interim analysis.

What does this mean? The 30% PFS risk reduction is meaningful but not overwhelming. Embolisation-eligible HCC is a population where TACE already delivers reasonable disease control, so a 30% incremental improvement over an effective local therapy is a credible result. The OS trend at interim is reassuring, but OS is the endpoint that will matter most for regulatory approval and clinical adoption, and it’s not yet mature. The three drug combination (tremelimumab, durvalumab and lenvatinib), plus TACE, raises practical questions about tolerability, complexity, and cost in a population where TACE alone is an established, effective SoC. For AstraZeneca, EMERALD-3 is the fourth positive Phase III Imfinzi readout this year, following POTOMAC, NIAGARA and VOLGA in bladder cancer. Adding a positive HCC result to that sequence underlines how broadly AstraZeneca is building the Imfinzi franchise across tumour types and disease stages.

BMS posts more positive Phase III data for mezigdomide in R/RMM

What happened? Following on from its interim readout in March 2026, BMS has announced additional positive results from the Phase III SUCCESSOR-2 trial of its CELMoD (cereblon E3 ligase modulation) mezigdomide in combination with carfilzomib and dexamethasone (MeziKd) vs. carfilzomib and dexamethasone alone (Kd) in patients with R/RMM. Results showed MeziKd demonstrated a clinically meaningful and statistically significant improvement in PFS (95% CI: 18 months vs. 8.3 months [HR:0.48; p<0.0001]), representing a 52% reduction in the risk of disease progression or death compared with Kd. Results also showed significantly improved PFS rates with MeziKd across patients in 2L and 3L as well as those with higher-risk disease. Higher ORR (80.2% vs 53.4%) and CR or better (26.7% vs 8.9%) were also seen with MeziKd. Median OS was not yet reached. The safety profile of MeziKd was consistent with the known profile of mezigdomide and the combination regimen. Grade 3-4 treatment-emergent adverse events were seen in 83.7% vs 56.5% of patients, with neutropenia in 61.1% vs 9.1%, and infections in 34.0% vs 15.6% of patients treated with MeziKd and Kd, respectively.

What does this mean? The treatment landscape for R/RMM has changed dramatically in the last 5-7 years, shifting from simple “next-line doublets” to sequential use of triplets/quadruplets with different mechanisms, followed by targeted immune therapies. The dominant approach in early lines of therapy is triplet regimens built around drugs from the three historical backbone classes (e.g., IMiDs + proteosome inhibitor + anti-CD38 mAb). Once a patient becomes refractory to early treatment, approaches shift toward BCMA-directed therapies and other immune approaches (e.g., CAR-T, bsAbs, ADCs, etc.). CELMoDs like mezigdomide are essentially designed to reset the IMiD class and keep combination therapy viable after lenalidomide/pomalidomide failure.

BMS/BioNTech’s pumitamig shows encouraging efficacy in NSCLC

What happened? BioNTech and BMS have presented interim data from its Phase II ROSETTA Lung-02 trial at ASCO 2026. The trial is evaluating pumitamig (BNT327/BMS-986545) plus chemotherapy in previously untreated advanced NSCLC. Pumitamig is an anti-PD-L1xVEGF bsAb. Among N=40 response-evaluable patients with a median follow-up of 9 months, the lower dose achieved confirmed ORRs of 63.6% in non-squamous and 72.7% in squamous NSCLC subtypes. Responses were observed across all PD-L1 expression levels (i.e., TPS below 1%, TPS 1-49%, and TPS 50% or above) and across both histological subtypes. The Phase III portion of ROSETTA Lung-02 is actively enrolling, alongside two additional Phase III trials.

What does this mean? The ORR numbers from ROSETTA Lung-02 are impressive for a Phase II interim readout: 63.6% in non-squamous and 72.7% in squamous NSCLC at the lower dose. The squamous number is noteworthy, because VEGF-targeting agents (e.g., bevacizumab) have historically been avoided in squamous NSCLC due to haemorrhage risk. Pumitamig's localised VEGF blockade mechanism, if it delivers meaningful anti-VEGF activity without systemic exposure, would address that concern. The PD-L1 expression-level agnosticism is interesting. A drug that produces consistent efficacy across all PD-L1 levels simplifies patient selection and potentially broadens the treatable population. BMS has been searching for its next major oncology franchise after Opdivo and multiple pipeline failures. BMS and BioNTech have explicitly flagged combinations with ADCs as a next wave of development strategy for pumitamig, which is forward-looking and aligns with the broader field direction.

Idorsia reports positive results for its Clostridioides difficile vaccine

What happened? Idorsia has reported additional data from the high-dose cohort of its Phase I trial of IDOR-1134-2831, its synthetic glycan vaccine targeting Clostridioides difficile. The high-dose results confirmed a favourable safety and tolerability profile, with all participants showing immunogenicity. The vaccine induced a dose-dependent IgG response, with exploratory analysis showing the response was particularly pronounced for IgG1, the subclass involved in opsonisation (i.e., the process by which pathogens are marked for destruction by the immune system). These results follow initial Phase I data reported in June 2025, which provided the first clinical validation of the synthetic glycan platform. Idorsia has stated its intention to advance the programme in partnership rather than independently. Recall, the vaccine targets a glycan on the surface of C. difficile that’s present on both the vegetative bacterial form and on spores. Most existing C. difficile vaccine approaches have targeted toxins rather than the bacterium or spore surface directly, which means they may reduce disease severity but don’t necessarily prevent colonisation or transmission. A vaccine targeting both bacteria and spores could in principle prevent initial colonisation and interrupt transmission, which is a broader protective effect than toxin-targeted approaches.

What does this mean? C. difficile causes approximately 400,000 infections annually in the US, more than 25,000 deaths per year, and generates $5-6 billion in acute care costs. There is also recurrent infection in ~25% of treated patients. Standard antibiotics clear primary infection but don’t prevent relapse, because they don’t eliminate spores and further disrupt the gut microbiome that would otherwise provide colonisation resistance. A prophylactic vaccine that prevents initial colonisation in at-risk populations would address the problem upstream. Pfizer’s PF-06425090 was the most advanced C. difficile vaccine programme, but development was discontinued after the Phase III CLOVER trials failed to meet its primary efficacy endpoint. Sanofi and MSD had earlier stage C. difficile vaccine programmes that also failed. Idorsia's glycan-targeting approach is mechanistically distinct from those failed programmes, which all targeted toxins rather than the organism itself, and that distinction is the scientific basis for the claim that this represents a genuinely new approach.

Ivonescimab delivers OS benefit in squamous NSCLC

What happened? Summit Therapeutics and Akeso have presented OS data from the Phase III HARMONi-6 trial at ASCO 2026. In N=532 patients with previously untreated locally advanced or metastatic squamous NSCLC, ivonescimab plus platinum-based chemotherapy reduced the risk of death by 34% compared to tislelizumab (Tevimbra; BeOne) plus chemotherapy (HR 0.66; 95% CI 0.50-0.87; p=0.0017). Median OS was 27.89 months for ivonescimab vs. 23.69 months for tislelizumab. The 24-month OS rate was 64.7% vs. 48.6%. The benefit was consistent across PD-L1 negative (HR 0.64) and PD-L1 positive (HR 0.68) subgroups. The trial had previously met its primary PFS endpoint in April 2025. Ivonescimab is a PD-1xVEGF bsAb engineered by Akeso and licensed to Summit Therapeutics in December 2022 for North America, South America, Europe, the Middle East, Africa and Japan.

What does this mean? A positive result, but there are a few things that need highlighting. First, the comparator. HARMONi-6 used tislelizumab, not pembrolizumab. Tislelizumab is approved in China and in some other markets but is not the dominant global PD-1 standard. Would the result hold against pembrolizumab? HARMONi-3, which is comparing ivonescimab plus chemotherapy against pembrolizumab plus chemotherapy in both squamous and non-squamous NSCLC, is the trial that will answer that question. Second, the geography. HARMONi-6 was conducted entirely in China. Cross-regional differences in tumour biology, patient populations, treatment practices, and prior therapy exposure can affect outcomes. HARMONi-3's multiregional design addresses this, but again that data is pending. Third, the VEGF safety signal in squamous NSCLC. The press release explicitly acknowledges that anti-VEGF mAbs have had limited development in squamous NSCLC due to risk of serious haemorrhage. The Grade 3 or higher haemorrhage rate of 2.6% in the ivonescimab arm vs. 0.8% for tislelizumab is higher, though the data release frames this as manageable and notes that the bispecific design may produce different safety dynamics from combining separate anti-PD-1 and anti-VEGF antibodies.

Kailera posts early-stage data for its tripleG, KAI-4729, in obesity

What happened? Kailera Therapeutics (Kailera) has provided topline results from a Chinese Phase I study assessing the safety, tolerability, pharmacokinetics and pharmacodynamics of HRS-4729, an injectable GLP-1/GIP/glucagon receptor tri-agonist (tripleG). KAI-4729 (also called HRS-4729) has been developed by Hengrui and was licensed to Kailera in October 2024. The multiple ascending dose (MAD) portion of the Phase I trial enrolled N=60 participants with a mean baseline BMI of 31.4 kg/m² and mean baseline body weight of 87.3 kg. Participants received once-weekly doses of 1mg, 4mg, 8mg or 12mg HRS-4729, 4mg ribupatide as an active control, or placebo for 12 weeks. At 12 weeks, the 12mg dose achieved a mean weight loss of 16% from baseline vs. 5.4% with placebo. The drug demonstrated linear PK with a half-life of approximately 4-5 days, supporting once-weekly dosing. Safety was consistent with the GLP-1 class. Notably, dose-dependent reduction in liver fat content was demonstrated by MRI-PDFF, which is clinically relevant for the MASH indication Hengrui intends to explore in Phase II.

What does this mean? KAI-4729 is in the same class as retatrutide (Eli Lilly), UBT251 (United Biotechnology/Novo Nordisk) and efocipegtrutide (Hanmi). A 16% mean weight loss at 12 weeks from a 12mg dose in a Phase I MAD study is a positive early signal, though several important caveats apply before drawing conclusions about how KAI-4729 compares to other tripleGs. Hengrui’s study was only 12 weeks long and they recruited patients with a mean BMI of 31.4, which is meaningfully lower compared to other trials. Kailera plans to initiate a Phase I trial outside China for KAI-4729 by end of 2026, with data anticipated in 2027.

Kelonia posts Phase I R/RMM data for KLN-1010 at ASCO 2026

What happened? Investigators at ASCO 2026 have presented new data from Kelonia’s Phase I inMMyCAR trial which is assessing the effectiveness of KLN‑1010 (in vivo BCMA CAR-T) in patients with R/RMM. Recall, Eli Lilly announced in April 2026 an agreement to acquire Kelonia for $7 billion to expand its genetic medicine/cell therapy footprint. The data presented at ASCO 2026 includes N=18 treated patients across 3 dose levels (as of the April 21, 2026 cutoff) and longer follow up at the high dose cohort. Per the presentation ORR was 100% across all treated patients, with 100% bone marrow MRD‑negative responses among MRD‑evaluable patients (n=14; 10-5 or deeper). Across all patients, responses were 50% PR, 22% VGPR, 6% CR and 22% sCR; and the responses deepened over time, with the 6 patients with ≥4 months of follow up showing 33% VGPR and 67% sCR, all ongoing. The first treated patient (high dose) remained in an ongoing MRD‑negative response beyond 10 months. Moreover, KLN‑1010 generated robust CAR‑T expansion and persistence despite the absence of lymphodepletion. Median Cmax vector copies/μg genomic DNA were 65,873 at 2x107 IU/kg, 71,021 at 6x106 IU/kg and 110,032 at 4x106 IU/kg, with Day 15 CAR-positive cells by flow cytometry of 34.5%, 53.1% and 42.3%, respectively. Overall safety was typical for CAR-T therapies and manageable.

What does this mean? Analysts have posited that KLN-1010 represents a potential next‑gen competitor to autologous BCMA CAR‑T therapies for MM such as J&J/Legend’s Carvykti (approved) and Gilead/Arcellx’s anito‑cel (under review; December 23, 2026 PDUFA date). Legend is advancing in vivo CAR‑T, using lentiviral vector-based delivery, with three assets in Phase I trials: 1) LVIVO‑TaVec100, a CD19×CD20 in development for non‑Hodgkin lymphoma; 2) LVIVO‑TaVec200, a CD19×GPRC5D in development R/RMM; and (3) LVIVO‑TaVec400, an undisclosed target in development for R/RMM.

Perioperative Erleada reduces risk of metastasis and death in prostate cancer

What happened? Johnson & Johnson has presented the final analysis of the Phase III PROTEUS trial at ASCO 2026. PROTEUS recruited N=2,109 patients and assessed if treatment with apalutamide (Erleada) plus androgen deprivation therapy (ADT) before and after radical prostatectomy (RP) with pelvic lymph node dissection (pLND) in participants with high-risk localized or locally advanced prostate cancer results in an improvement in pathological complete response (pCR) rate and metastasis-free survival (MFS) as compared to placebo plus ADT. Both dual primary endpoints were met. The pCR or MRD rate at surgery was 8.9% with apalutamide vs. 1.0% with hormone therapy alone (OR 10.17; p<0.0001). Metastasis-free survival showed a 20% reduction in risk of metastasis or death (HR 0.80; p=0.02), with five-year MFS rates of 78.2% vs. 73.5%. Time to subsequent therapy exceeded six years with apalutamide vs. approximately three and a half years with hormone therapy alone (74.2 vs 41.5 months; HR 0.65; p<0.0001). At a median follow-up of 61.7 months, testosterone recovery to adequate levels occurred within 8.1 months.

What does this mean? Apalutamide is already approved in metastatic hormone-sensitive and non-metastatic castration-resistant prostate cancer, which are later-line settings where the disease has progressed beyond the curative window. PROTEUS moves it into the perioperative setting, where approximately half of patients undergoing surgery for high-risk disease will ultimately experience biochemical recurrence. Intervening before and after surgery, when the tumour is still localised and the disease burden is lowest, has the aim of eradicating micrometastatic disease that conventional imaging can’t detect but that drives eventual recurrence. A ninefold improvement is mathematically striking, yet the absolute rate of 8.9% means that nine in ten patients still have detectable residual disease at surgery. The clinical significance of the pCR endpoint in prostate cancer is less well established than in breast cancer, where pathologic complete response is a validated surrogate for long-term outcomes. The MFS data, with a more modest 20% risk reduction at 61.7 months follow-up, is the more clinically meaningful primary endpoint and supports the biological plausibility of the pCR improvement, but the hazard ratio of 0.80 is at the lower end of what might be considered practice-changing in isolation. From a safety perspective, grade 3 or 4 adverse events occurred in 39.6% of apalutamide patients vs. 31.0% on hormone therapy alone, and discontinuations were 7.4% vs. 2.7%. The testosterone recovery data will be important as prolonged testosterone suppression has implications for bone density, cardiovascular health, and quality of life in a curative-intent population with a long expected survival. Other androgen receptor pathway inhibitors (e.g., enzalutamide, darolutamide) are also being studied in perioperative prostate cancer settings. Apalutamide's PROTEUS data establish a positive signal and a potential new indication, but the comparison against surgery alone will be necessary for regulatory purposes.

Roche to present new obesity portfolio data at ADA 2026

What happened? Roche will present late-breaking Phase II data for two obesity assets at ADA 2026. Full data are not yet publicly available, so details are thin on the ground. The two assets are enicepatide (CT-388), a dual GLP-1/GIP receptor agonist, and petrelintide, a long-acting human amylin analogue. Both are moving into Phase III development, and Roche plans to initiate a Phase II multi-arm combination trial of the two together around mid-2026.

What does this mean? Roche signalled its strategic intent to compete in the obesity space via its $400 million acquisition of Carmot Therapeutics in July 2024. The acquisition gave Roche access to Carmot’s incretin pipeline, including enicepatide. Not long after, Roche licensed petrelintide, an amylin analogue, from Zealand Pharma in March 2025, signalling its intention to develop petrelintide as a standalone therapy as well as a fixed-dose combination with enicepatide. While enicepatide has the same MOA as Lilly’s tirzepatide (Mounjaro; dual GLP-1/GIP agonism), it has been designed to have potent activity at both GLP-1 and GIP receptors with minimal beta-arrestin recruitment on either receptor. Beta-arrestin recruitment drives receptor internalisation and desensitisation, which is thought to reduce the sustained pharmacological effect of GLP-1 receptor agonists over time. By minimising this process, enicepatide is designed to maintain prolonged receptor engagement without the functional tolerance that may limit other agents. As an amylin analogue, petrelintide works on a separate pathway compared to enicepatide. Amylin is co-secreted with insulin from pancreatic beta cells in response to food intake; it acts centrally to restore leptin sensitivity and promote satiety by signalling fullness rather than suppressing appetite through the same hypothalamic circuits as GLP-1. The practical significance is that petrelintide's tolerability profile is expected to differ substantially from GLP-1 agonists, as it doesn’t cause nausea and vomiting through the same mechanism, and the preclinical poster being presented shows it doesn’t delay gastric emptying, which is the primary driver of GLP-1-related GI adverse events.

Virion Therapeutics reports Phase Ib data for VRON-0200 in HBV

What happened? Virion Therapeutics has presented data from its Phase Ib trial which is assessing VRON-0200 as a potential treatment for chronic hepatitis B (HBV). VRON-0200 is a first-in-class checkpoint modifier which is designed to restore the immune system's ability to fight HBV, rather than simply suppressing viral replication the way standard antiviral drugs do. At the end of the formal study period (one year after the prime dose), 85% of patients (23 out of 27) showed HBsAg levels that were either sustained at lower levels or continued to fall. Of those, 52% had reductions of more than 50%, with four patients achieving a decline of 1 log10 IU/mL or greater. Post-study follow-up data, available for N=12 patients at up to 846 days after the prime dose, showed that N=11 (92%) had continued HBsAg declines, no patient showed HBsAg rebound, and N=2 patients (17%) achieved full HBsAg loss. HBsAg loss is one of the accepted markers of a functional cure in HBV. The safety profile remained favourable, with no serious treatment-related adverse events, no discontinuations, and no treatment-related laboratory abnormalities.

What does this mean? The persistent HBsAg decline without rebound after a single dose, out to 2+ years in some patients, is unusual in this field. Existing and investigational HBV treatments have typically failed to restore the patient's immune response against the virus, which is what allows it to return after therapy stops. That said, the numbers are small (N=12). While the field has seen some recent successes (e.g., Ionis/GSK, Precision Biosciences), the field has also seen promising small-cohort HBV cure data that hasn't held up at scale (e.g., vesatolimod, selgantolimod, inarigivir). Overall, VRON-0200’s mechanism is differentiated, the durability signal looks real, and the safety profile looks clean. But it’s still early-stage data in small numbers. The key questions going forward are whether the response rates hold as patient numbers increase, what the full virological picture looks like (i.e., cccDNA, HBV DNA), and whether the Cohort 3 combination data add meaningfully to monotherapy results.

Regulatory

AbbVie’s Decnupaz gets FDA nod for ultra-rare BPDCN

What happened? AbbVie has received FDA approval for Decnupaz (pivekimab sunirine) for the treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an ultra-rare and aggressive haematologic malignancy with limited treatment options. Pivekimab sunirine is a CD123-targeting ADC. CD123 (IL-3Rα) is overexpressed in BPDCN, making it an attractive target. The payload belongs to the indolinobenzodiazepine pseudodimer class, which alkylates DNA and causes single strand breaks without crosslinking, leading to apoptosis. The approval is supported by data from the Phase I/II CADENZA trial which showed that newly diagnosed patients with BPDCN who were treated with pivekimab sunirine demonstrated clinically meaningful and durable responses. In newly diagnosed patients with BPDCN (N=33), researchers observed a composite complete response rate of 69.7% with a median duration of response of 9.7 months, with 13 patients (39.4%) who were able to receive post-study treatment stem cell transplant. Patients with relapsed or refractory disease (N=51) had a composite complete response rate of 15.7% with median duration of response rate of 9.2 months, with six patients (11.8%) who were able to receive post-study treatment stem cell transplant. Most common adverse reactions (≥20%) were oedema, fatigue, musculoskeletal pain, haemorrhage, infusion-related reactions, nausea, and diarrhoea.

What does this mean? The prior approved treatment for BPDCN was tagraxofusp (Elzonris; Stemline Therapeutics), an IL-3 receptor-targeted cytotoxin approved in December 2018. That drug requires inpatient administration due to capillary leak syndrome risk, which has been a significant practical limitation. This makes Decnupaz’s outpatient initiation a clinically meaningful differentiator, as it reduces the burden on both patients and healthcare systems and broadens access to treatment beyond centres with inpatient haematology infrastructure. For AbbVie, this is a strategically significant approval beyond its clinical importance in a small patient population. Decnupaz is AbbVie's first approved ADC for a haematological malignancy, establishing a proof of concept for the platform in blood cancers. AbbVie has been actively building its oncology pipeline beyond Imbruvica and Venclexta, and an ADC approval (regardless of how narrow the indication is) validates the approach and creates a foundation for broader haematological oncology ambitions.

FDA accepts Roche’s NDA for giredestrant in breast cancer

What happened? The FDA has accepted Roche's NDA for giredestrant under Priority Review for the adjuvant treatment of adults with ER+/HER2- early-stage (I-III) breast cancer, with a PDUFA date of November 30, 2026. The submission is based on results from the Phase III lidERA Breast Cancer trial which showed giredestrant reduced the risk of invasive disease recurrence or death by 30% compared with SoC endocrine therapy. Roche describes this as the first significant advance in adjuvant endocrine therapy in over 20 years, with the current standard being tamoxifen or aromatase inhibitors, both established for decades. Giredestrant is an oral selective oestrogen receptor degrader (SERD) designed to degrade the oestrogen receptor protein rather than merely blocking it, as aromatase inhibitors and tamoxifen do. Degradation rather than inhibition is theoretically more complete in its suppression of ER signalling and is also designed to retain activity against ESR1-mutant tumours which can arise as a resistance mechanism to aromatase inhibitors. Crucially, this is the adjuvant early-stage setting, where more than 90% of ER+ breast cancer cases are diagnosed and where preventing recurrence has the most profound long-term impact on survival.

What does this mean? The broader competitive picture for oral SERDs in early breast cancer has shifted significantly in recent weeks. Giredestrant has a filed NDA and a PDUFA date, AstraZeneca’s camizestrant has had a US regulatory setback (negative ODAC, extension of PDUFA date), a CHMP endorsement in Europe, and pending adjuvant data from CAMBRIA-1 and CAMBRIA-2. Moreover, elacestrant (Orserdu; Stemline Therapeutics) is approved only in the pre-treated metastatic ESR1-mutant setting. Of the three most advanced oral SERDs, giredestrant is now clearly ahead in the curative-intent space that represents the largest long-term opportunity.

FDA accepts Vertex’s BLA for povetacicept in IgAN

What happened? Vertex Pharmaceuticals (Vertex) has announced the FDA has accepted its BLA submission for povetacicept, an investigational engineered fusion protein and dual BAFF/APRIL inhibitor being developed as a potential treatment for adult immunoglobulin A nephropathy (IgAN). The FDA has assigned a PDUFA target action date of November 30, 2026. If approved, povetacicept will become the first commercialised therapy in Vertex’s emerging nephrology franchise. The submission is supported by positive data from a pre-specified Week 36 interim analysis of the ongoing Phase III RAINIER trial of povetacicept in IgAN, demonstrating a statistically significant and clinically meaningful reduction in proteinuria, a key marker of kidney disease progression, compared to placebo. The RAINIER trial met its primary and secondary objectives, and Povetacicept was generally safe and well tolerated. 

What does this mean? Vertex built one of the most valuable franchises in rare disease through its cystic fibrosis portfolio, but CF is reaching its biological ceiling. Trikafta covers most eligible patients, and the pipeline of new CF modalities has limited incremental room. Vertex has been explicitly building a nephrology franchise as its next major platform, with IgAN as the entry point and primary membranous nephropathy and APOL1-mediated kidney disease as subsequent targets. With Breakthrough Therapy Designation and FDA accelerated approval in place for povetacicept, and a November 2026 PDUFA date, a US approval and commercial launch in the first quarter of 2027 is a plausible timeline. Commercially, povetacicept’s autoinjector, which allows once monthly dosing and home administration, is an important commercial differentiator. IgAN is a chronic disease requiring long-term treatment, so a subQ injection every month via a home device competes favourably on convenience against IV or more frequent dosing regimens. The expansion into generalised myasthenia gravis via the Phase II ETNA trial is worth keeping tabs on.

Imfinzi gets FDA nod for BCG-naïve, high-risk NMIBC

What happened? AstraZeneca has received FDA approval for Imfinzi (durvalumab) in combination with BCG induction and maintenance therapy for adult patients with BCG-naïve, high-risk non-muscle-invasive bladder cancer (NMIBC). The approval is based on the Phase III POTOMAC trial, in which N=1,018 patients were randomised across three arms. Adding one year of Imfinzi to BCG induction and maintenance therapy reduced the risk of high-risk disease recurrence, progression or death by 32% compared to BCG alone (HR 0.68; 95% CI 0.50-0.93; p=0.0154), with a median follow-up exceeding five years. Estimated median disease-free survival was not yet reached in either arm. The safety profile was consistent with the known profiles of the individual agents, with no new signals identified.

What does this mean? This is the third positive Phase III readout for Imfinzi in bladder cancer, following NIAGARA in cisplatin-eligible MIBC and VOLGA in cisplatin-ineligible MIBC, which were covered in previous notes. AstraZeneca has now established Imfinzi across the full spectrum of bladder cancer: non-muscle-invasive, muscle-invasive cisplatin-eligible, and muscle-invasive cisplatin-ineligible. This is a breadth of indication coverage that no other PD-L1 inhibitor has achieved in this tumour type. This third indication (NMIBC) is commercially important as there are more than 31,000 high-risk NMIBC patients treated in the US annually. The competitive picture in BCG-naïve NMIBC is relevant. Pembrolizumab is approved for BCG-unresponsive NMIBC, which is a different and more refractory population, but hasn’t demonstrated benefit in the BCG-naïve setting. The approval therefore gives Imfinzi a first-mover advantage in the earliest and largest NMIBC population, ahead of any competing checkpoint inhibitor data in the same indication.

Commercial

Ascidian and Eli Lilly collaborate on RNA Exon R&D

What happened? Ascidian Therapeutics (Ascidian) and Eli Lilly (Lilly) have entered a global research collaboration to discover and develop therapies for undisclosed monogenic kidney diseases, with an option to expand to additional targets. Lilly receives exclusive target-specific rights to Ascidian's RNA exon editing technology. Total deal value is up to $1.9 billion, comprising an undisclosed upfront payment, development and commercial milestones, and tiered royalties on approved products. Ascidian leads the discovery phase, Lilly takes over for later preclinical development, clinical trials, manufacturing and commercialisation.

What does this mean? RNA exon editing is a novel modality that’s not yet widely understood, and its significance is easily underappreciated alongside the more familiar CRISPR and base editing approaches. Readers are advised to review Doi et al. (2024) for an overview of the science. One thing to highlight, however, is that the kilobase-scale editing capability mentioned in the PR is what makes RNA exon editing particularly relevant for kidney disease. Many monogenic kidney conditions (e.g., Alport syndrome, autosomal dominant polycystic kidney disease) involve mutations in very large genes or complex splicing errors that cannot be corrected by single-base approaches. Correcting whole exons at scale is the unique capability that positions this technology where other genetic medicine modalities cannot reach. Ascidian’s most advanced clinical programme is in Stargardt disease (an inherited retinal condition), meaning kidney disease is an expansion into a new organ system. Placing this alongside Lilly's other recent genetic medicine activity (e.g., VERVE-102 for PCSK9 base editing, the Engage Biologics non-viral DNA delivery acquisition, the ophthalmology gene therapy acquisition of Adverum, SangeneBio for metabolic RNAi therapeutics) demonstrates that Lilly believes genetic medicine will be the dominant therapeutic modality within the next decade, and that owning multiple platform positions across the modality spectrum is the right way to compete in that future.

Haisco and Eli Lilly announce broad licensing and R&D collaboration

What happened? Haisco Pharmaceutical Group (Haisco) has entered a licensing and R&D collaboration with Eli Lilly (Lilly) covering up to five innovative target programmes across multiple therapeutic areas. Haisco is responsible for the discovery and identification of the programmes, with Lilly leading IND-enabling studies, clinical development and commercialisation. Lilly obtains exclusive worldwide rights to certain programmes and exclusive ex-Greater China rights to others, with Haisco retaining rights in mainland China, Hong Kong, Macau and Taiwan for those latter programmes. Haisco is eligible for up to $87 million in upfront and near-term payments, up to $2.967 billion in downstream milestones, and single-digit tiered royalties.

What does this mean? Lilly continues to spend its GLP-1 dollars on accessing Chinese innovation. Prior to this deal with Haisco, Lilly had entered into agreements with Innovent Biologics (oncology and immunology) and SanegeneBio (metabolic RNAi therapies). The financial terms reflect that earlier stage. $87 million in upfront and near-term payments across up to five programmes is approximately $17 million per programme at full utilisation. The $2.967 billion in downstream milestones assumes all five programmes succeed through clinical development and commercialisation, which in practice is unlikely to happen. Assuming Lilly’s interests align with Haisco’s R&D focus, the therapeutic area breadth is wide enough that the deal is essentially a discovery pipeline option for Lilly. Autoimmune disorders align with Lilly's immunology franchise, and the metabolic area is an obvious connection to GLP-1 and broader metabolic disease work.

Hanmi licenses sonefpeglutide to Eli Lilly for up to $1.260 billion

What happened? Hanmi Pharm (Hanmi) has entered into a license agreement for the development, manufacturing and commercialisation of sonefpeglutide, its long-acting glucagon-like peptide 2 analogue, with Lilly. Sonefpeglutide is a novel drug candidate incorporating Hanmi's proprietary long-acting platform technology, LAPSCOVERY. The agreement covers multiple indications, including short bowel syndrome (SBS) for which Hanmi is conducting a Phase II trial. Under the terms of the deal, Hanmi will continue to conduct the ongoing Phase II trial in SBS through completion while Lilly will explore additional clinical trials for sonefpeglutide based on its nonclinical and clinical data. Lilly will obtain exclusive rights to develop, manufacture and commercialise sonefpeglutide worldwide, excluding Korea. Hanmi will receive up to $1.260 billion, including an upfront payment of $75 million and $1.185 billion in potential clinical development, regulatory approval and commercialisation milestone payments.

What does this mean? The immediate indication is SBS, a rare condition in which insufficient functional intestinal length leads to malabsorption, requiring parenteral nutrition. Teduglutide, a GLP-2 analogue originally developed by Takeda and now generically available, is already approved for SBS. Sonefpeglutide, with a longer half-life, would potentially offer less frequent dosing than teduglutide's daily injection requirement, which is a clinically and commercially relevant differentiator in a chronic rare disease setting. Beyond SBS, GLP-2 has demonstrated preclinical activity in intestinal failure, inflammatory bowel disease, gut mucosal protection during chemotherapy, and metabolic bone disease. Whether any of those indications translates into viable clinical programmes is unproven, but the half-life extension technology makes sonefpeglutide a more flexible development candidate compared to a standard GLP-2 peptide. In the broader context of Lilly's 2026 acquisitions and licensing activity (i.e., retatrutide, VERVE-102, Engage Biologics, three vaccine companies, and now sonefpeglutide) this deal is the smallest but could turn out to be the most strategic. It extends Lilly's reach into gastrointestinal biology beyond appetite and weight, into an area of genuine mucosal disease biology that connects to oncology supportive care, rare GI disease, and potentially inflammatory bowel disease.

Pfizer and Innovent announce global oncology R&D collaboration

What happened? Pfizer and Innovent Biologics have entered a global strategic licensing and collaboration agreement covering 12 early-stage oncology programmes: eight originated by Innovent and four have been proposed by Pfizer. Programmes span ADCs with novel differentiated payloads and multi-specific antibodies with differentiated immune-engaging designs. Innovent receives $650 million upfront and is eligible for up to $9.85 billion in development, regulatory and commercial milestones, plus double-digit royalties on approved products. The deal is structured across three tiers: four programmes where Pfizer takes an exclusive global licence and funds development; four where Pfizer takes ex-Greater China rights and funds the bulk of the costs; and four where both companies co-develop and co-commercialise globally, sharing profits in the US and Europe while Innovent retains Greater China rights. Innovent leads development through Phase I, after which Pfizer takes over global development. Closing is expected in Q3’26 subject to regulatory approvals.

What does this mean? This is one of the largest licensing deals announced between a Chinese biotech and a Western pharma, and it follows a pattern that has become one of the defining commercial dynamics in pharmaceutical dealmaking: Western pharma acquiring access to Chinese biotech innovation at scale, in exchange for global development muscle and commercial reach. Similar deals include Pfizer/3SBio (May 2025), GSK/Hengrui (July 2025), AstraZeneca/CSPC Pharmaceutical (January 2026), AbbVie/RemeGen (January 2026), Eli Lilly/Innovent (February 2026), and BMS/Hengrui (May 2026). $650 million upfront for 12 programmes is approximately $54 million per programme, which is modest on a per-asset basis but reflects the early-stage nature of the portfolio. The ~$10 billion in potential milestones is headline-grabbing but is an aggregate across all 12 programmes where all milestones are fully achieved. The co-commercialisation structure for four programmes, with profit sharing in the US and Europe, means that Innovent gains a stake in Western commercial success rather than just a royalty stream, and signals Pfizer's confidence that those four programmes have the potential to reach the market. Looking at this deal through the technology lens, ADCs are relevant to Pfizer's strategic priorities. Pfizer acquired Seagen in December 2023 for $43 billion specifically to build an ADC franchise, and Padcev (enfortumab vedotin) has become one of its most commercially important oncology assets. Innovent's ADC capabilities give Pfizer access to novel payload and linker technologies that may differentiate its ADC portfolio from what is available through purely Western R&D. The multi-specific antibody element is similarly aligned with where oncology immunotherapy is heading, as the field moves beyond single checkpoint inhibitors towards more sophisticated immune-engaging architectures.

Servier to pay up to $2.65 billion for Edgewise’s sevasemten

What happened? Servier has agreed to acquire the muscular dystrophy business of Edgewise Therapeutics for $1.55 billion upfront and up to $1.1 billion in regulatory and commercial milestones, totalling potential consideration of $2.65 billion. The deal covers sevasemten and all related intellectual property, regulatory filings, clinical data, and key agreements. Sevasemten is an oral, first-of-its-kind skeletal muscle modulator designed to protect fragile muscles by inhibiting fast skeletal muscle troponin activator, reducing the rapid, powerful contractions that cause muscle fibre damage in Becker and Duchenne muscular dystrophy. It’s being developed for both conditions. The mechanism is distinct from approved DMD therapies such as exon-skipping agents and steroid regimens. Rather than trying to restore dystrophin expression or compensate for its absence, sevasemten reduces the contractile force that tears already-vulnerable muscle fibres. The FDA declined to review an early rolling NDA application based on Phase II CANYON study data inpatients with Becker muscular dystrophy, but Edgewise had continued development towards a more conventional Phase III path.

What does this mean? For Servier, this is its second major acquisition in neurology and rare disease in 2026, following its March 2026 acquisition of Day One Biopharmaceuticals for $2.5 billion and its approved paediatric brain cancer drug Ojemda. Becker and Duchenne muscular dystrophy are conditions with significant unmet need and relatively limited competition outside the exon-skipping and gene therapy approaches, and sevasemten's mechanism is genuinely novel. For Edgewise, this is a focused strategic reset. Following the deal closing, Edgewise will become a cardiovascular-focused company, with its pipeline centred on EDG-7500 for hypertrophic cardiomyopathy, EDG-15400 for heart failure with preserved ejection fraction, and EDG-003 for an undisclosed target. The upfront proceeds, combined with existing cash, are expected to fully fund EDG-7500 through potential approval.

Travere licenses civorebrutinib from Everest

What happened? Travere Therapeutics (Travere) has licensed civorebrutinib (EVER001) from Everest Medicines (Everest) for exclusive development and commercialisation rights in all markets outside China and certain East and Southeast Asian countries. Travere will pay $112.5 million upfront, with up to approximately $1 billion in additional milestone payments across up to five indications, plus tiered royalties ranging from high single-digit to double-digit percentages based on annual net sales. Civorebrutinib is an oral, covalent reversible BTK inhibitor designed to provide differentiated efficacy, safety and convenience in immune-mediated kidney diseases. PoC data exist in primary membranous nephropathy, with planned evaluation in focal segmental glomerulosclerosis (FSGS), minimal change disease (MCD) and other indications. Most approved BTK inhibitors (e.g., ibrutinib, acalabrutinib, zanubrutinib) are covalent irreversible, meaning they permanently inactivate BTK. Reversible covalent binding provides sustained target occupancy while allowing BTK to recover when drug levels fall, which is theoretically associated with a cleaner off-target safety profile.

What does this mean? Travere has an established renal presence via sparsentan (Filspari), an endothelin and angiotensin II receptor antagonist that’s approved for the treatment for IgA nephropathy (IgAN) and focal segmental glomerulosclerosis (FSGS). Licensing civorebrutinib for development and eventual commercialisation in rare kidney disease is a logical step toward growing its renal portfolio. It also mitigates some of the competitive pressure that exists in indications like IgAN where multiple MOAs have been approved (e.g., endothelin pathway inhibitors, complement inhibitors, BAFF/APRIL-targeting biologics (see FDA accepts Vertex’s BLA for povetacicept in IgAN) and plasma cell-directed therapies. Today, BTK inhibitors are probably not the leading mechanism in rare kidney disease, but they’re one of the most important emerging "next-wave" modalities. If civorebrutinib delivers strong Phase II data in IgAN and other indications, BTK inhibition could become a significant new pillar of nephrology alongside complement and APRIL/BAFF-targeted therapies.

In Other News

A new drug concept to treat obesity and type 2 diabetes

A team led by metabolism researcher Prof Timo D. Müller at Helmholtz Munich has developed a new approach for treating obesity and type 2 diabetes: a hybrid molecule using the well-known GLP-1/GIP signalling pathway as a “door opener” which delivers an additional metabolic modulator specifically into the target cells. In laboratory experiments, mice treated with a GLP-1R-GIPR-PPARα/γ/δ “quintuple agonist” ate less, lost more weight and showed improved blood-glucose values compared with reference treatments. The researchers published their preclinical results in Nature.

AbbVie’s Aquipta approved in Europe

AbbVie has received approval from the EC for Aquipta (atogepant), an oral CGRP antagonist, for the acute treatment of migraine in adults, with or without aura. This marks the second indication for Aquipta in the EU, where it’s now approved both for acute attacks and as a once-daily preventive treatment for adults experiencing four or more migraine days each month.

CytomX expands bsAbs collaboration with Regeneron

CytomX Therapeutics (CytomX) has announced an expansion of its collaboration and licensing agreement with Regeneron to discover conditionally activated bispecific cancer therapies. The Regeneron and CytomX collaboration, initially entered in November 2022, is strategically focused on applying CytomX’s biologic masking strategies in combination with Regeneron’s bispecific antibodies to develop investigational bispecifics that remain inactive until activated by proteases in the tumour microenvironment. This technology has the potential to widen the therapeutic window and help minimize off-target effects for next-generation T-cell engaging therapies, potentially addressing tumour types that have historically been challenging for immunotherapy.

Dr Falk and Renexxion move naronapride into late-stage trials

Following the release of positive data from its Phase IIb MOVE-IT trial in May 2026, Dr Falk and Renexxion have announced that they will advance the development of naronapride beyond Phase IIb into pivotal studies in patients with gastroparesis. No late-stage trial details have been provided.

Eli Lilly’s Retevmo shows benefit in RET fusion+ NSCLC

Eli Lilly (Lilly) has presented data from its Phase III LIBRETTO-432 trial at ASCO 2026. In N=151 patients with early-stage (IB-IIIA) RET fusion+ NSCLC following surgery or definitive radiotherapy with curative intent, adjuvant selpercatinib (Retevmo) reduced the risk of disease recurrence or death by 83% vs. placebo in the primary analysis population (stage II-IIIA; HR 0.17; 95% CI 0.06-0.51; p<0.001). The 24-month EFS rate was 92% with selpercatinib vs. 61% with placebo. Median EFS was not reached for selpercatinib vs. 31.8 months for placebo. Results were consistent in the overall IB-IIIA population (HR 0.17; p<0.001). OS trended in favour of selpercatinib but was immature at this analysis. The primary Grade 3 or higher adverse events were elevated ALT (17%) and AST (19%), both manageable with dose modification. Recall, Lilly gained selpercatinib via its February 2019 acquisition of LOXO Oncology. It was initially approved as a treatment for advanced (metastatic) RET fusion+ NSCLC in May 2020.

Gilead’s Livdelzi shows ALP normalisation benefit in PBC

Gilead has announced positive results from its Phase III IDEAL trial in people with primary biliary cholangitis (PBC), showing that treatment with Livdelzi (seladelpar) led to significantly more patients achieving normalization of alkaline phosphatase (ALP), a key liver marker of disease progression, compared with placebo after 52 weeks. These findings were observed in people with inadequately controlled disease, defined as having ALP levels above the ULN and below 1.67×ULN, with an incomplete response or intolerance to ursodeoxycholic acid (UDCA). The safety profile of seladelpar observed in IDEAL was consistent with previously reported Livdelzi studies, with no new safety concerns identified.

J&J’s Rybrevant Faspro shows benefit in advanced head/neck cancer

Johnson & Johnson has announced pivotal results from the Phase Ib/II OrigAMI-4 study showing that subQ amivantamab and hyaluronidase (Rybrevant Faspro) delivered durable responses in patients with advanced head and neck squamous cell carcinoma previously treated with immunotherapy and chemotherapy. Confirmed ORR was 42%, with more than one-third of responders achieving complete responses. Median duration of response was not yet reached, with a median follow up of 11.8 months.

J&J’s Tremfya gets FDA label expansion in PsA

Johnson & Johnson has announced that the FDA has approved a sBLA to include evidence in the Tremfya (guselkumab) label for the inhibition of progression of structural joint damage in adults with active psoriatic arthritis (PsA). The inclusion of this outcome reflects that Tremfya is the only anti-IL-23 proven to help stop further structural damage, offering patients with active PsA a first-line treatment option that provides effective symptom control and no new safety signals, while significantly inhibiting irreversible joint damage.

MannKind gets FDA nod for Afrezza in adolescents aged 6+ years

MannKind has received FDA approval for Afrezza (insulin human) Inhalation Powder for use in children and adolescents aged 6 and older living with type 1 and type 2 diabetes. Afrezza is an ultra-rapid-acting inhaled insulin taken at mealtimes that more closely mimics the body’s natural insulin response. This approval expands Afrezza’s availability beyond adults. The FDA approval is supported by results from the pivotal INHALE‑1 clinical trial, along with additional safety, efficacy, and long‑term exposure data from studies evaluating inhaled insulin over the past two decades of development.

Microsoft premieres Copilot Health

Microsoft has launched Copilot Health, making it available to US users aged 18 and over with a Microsoft 365 Personal, Family or Premium subscription. Copilot Health is a dedicated space within Microsoft Copilot that integrates wearable device data, wellness app data and medical health records to provide personalised health insights and guidance. It connects initially with Apple Health, with other integrations to follow, and draws on health records from over 50,000 US provider organisations. Features include a personal health profile, intelligent insights based on that profile, care navigation for finding local healthcare providers by specialty, language, insurance and location, and guidance sourced from trusted health organisations globally in partnership with Harvard Health and in accordance with National Academy of Medicine principles.

Mirum announces new data for its liver disease portfolio at EASL 2026

Mirum Pharmaceuticals has presented three late-breaking datasets at EASL 2026 covering its rare liver disease portfolio. These include data from the Phase IIb VISTAS trial (volixibat in primary sclerosing cholangitis), the Phase IIb AZURE-1 trial (brelovitug in chronic hepatitis delta) and new long-term data for its approved therapy maralixibat in progressive familial intrahepatic cholestasis.

Novartis’s Pluvicto shows consistent efficacy across mHSPC sub-groups

Novartis has presented a subgroup analysis from the Phase III PSMAddition trial at ASCO 2026, showing consistent rPFS improvement across key subgroups, stratified by disease volume (high or low) and disease presentation (de novo or recurrent), with Pluvicto plus SoC vs. SoC alone. The primary PSMAddition endpoint was first reported in June 2025 which showed a 28% reduction in the risk of radiographic progression or death (HR 0.72; 95% CI: 0.58-0.90). Recall, the purpose of PSMAddition trial was to evaluate the efficacy and safety of 177Lu-PSMA-617 (Pluvicto) in combination with SoC, vs. SoC alone, in adult male patients with mHSPC.

Pfizer’s Braftovi regimen nearly doubles mean PFS in mCRC

Pfizer has presented Cohort 3 results from its Phase III BREAKWATER trial at ASCO 2026 in a late-breaking oral presentation. The trial evaluated encorafenib (Braftovi) plus cetuximab and FOLFIRI vs. FOLFIRI with or without bevacizumab in previously untreated BRAF V600E-mutant metastatic colorectal cancer (mCRC). Encorafenib plus cetuximab and FOLFIRI reduced the risk of disease progression or death by 56% vs. chemotherapy (HR not specified in the metadata but implied by the near doubling median PFS described in the headline). An OS benefit was also observed, with a 44% reduction in the risk of death. Full data were presented orally, and the press release metadata contains the top-line numbers but not the full data table. Recall, Pfizer reported that Cohort 3 met its primary endpoint of objective response rate in January 2026.

Prices for branded drugs rise in the US but fall in others

A report from the AARP Public Policy Institute (PPI) has found that list prices for 25 top brand name drugs have increased by an average of 81% since they first entered the US market. In contrast, lifetime list prices for the same products decreased by an average of 13% percent in 19 other high income countries.

Revolution Medicines gets standing ovation at ASCO 2026

During its presentation of data from the Phase III RASolute 302 clinical trial which evaluated daraxonrasib in 2L metastatic pancreatic ductal adenocarcinoma (PDAC), the audience gave a standing ovation, such was the excitement surround the data. As reported back in April 2026, daraxonrasib taken orally once daily demonstrated statistically significant and clinically meaningful improvements in PFS and OS compared with SoC cytotoxic chemotherapy delivered intravenously. In the overall (ITT) study population, daraxonrasib demonstrated a median OS of 13.2 months versus 6.7 months for chemotherapy, with a hazard ratio of 0.40 (p < 0.0001). Daraxonrasib was generally well tolerated, with a manageable safety profile and with no new safety signals.

Samsung Bioepis launches Eylea biosimilar across Europe

Samsung Bioepis has announced the launch of Opuviz 40 mg/mL solution for injection in a vial, a biosimilar referencing Eylea (aflibercept; Bayer), across Europe. Opuviz is the fifth biosimilar product to be directly commercialised by Samsung Bioepis. It builds on other key biosimilar milestones in Europe in 2026, including the initiation of direct commercialization of Byooviz (ranibizumab) 10 mg/mL solution for injection in a vial, as well as the launch of Xbryk (denosumab) 120 mg solution for injection in a vial in January 2026.

About the Author

Duncan Emerton is a pharmaceutical industry professional with a background spanning clinical R&D, medical affairs, and strategic consulting. He brings a commercially minded perspective to complex scientific and market developments, with experience in competitive intelligence, business analysis, and portfolio strategy across the life sciences sector. Through Pharma Phriday, Duncan provides concise, insight-driven commentary on the stories shaping the pharmaceutical and biotech landscape, helping readers filter out the noise to understand what really matters for companies, competitors, and patients alike.