Pharma Phriday (April 24, 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.

In this week’s note:

Artificial Intelligence

• AWS launches Amazon Bio Discovery to accelerate AI-powered R&D

• Boehringer Ingelheim launches AI centre for pharma research in London

• GEn1E and Rubicon Research announced AI-driven R&D pact

• Merck & Co. partners with Google on AI transformation

• OpenAI launches GPT-Rosalind

• UK government announces launch of Sovereign AI

Clinical

• AstraZeneca posts more positive Phase III data for tozorakimab in COPD

• Cochrane review analyses the effect of anti-amyloid mAbs in Alzheimer’s

• Eli Lilly posts Phase III safety data for Foundayo

• Merck & Co.’s MK-2010: early data, no clear plan

• Moderna posts Phase I/II data for mRNA-4359 in melanoma

• Monopar posts positive Phase III data for ALXN1840 in Wilson’s disease

• Novo’s etavopivat effective in late-stage sickle cell disease trial

• Roche’s Enspryng delivers in Phase III MOGAD trial

• Roche initiates new Phase III study for Elevidys

• Roche presents new data on fenebrutinib in RMS

Regulatory

• EC approves Moderna’s combo flu/COVID-19 vaccine, mCombriax

• FDA approves Dupixent for treatment of paediatric CSU

• GSK’s Blenrep approved in China for treatment of 2L+ R/RMM

• Ipsen’s Ojemda receives EC approval for paediatric low-grade-glioma

• LEO Pharma receives China’s NMPA approval of Enstilar

• Merck & Co.’s Enflonsia approved in Europe for RSV prevention in infants

• Merck & Co.’s Idvynso gets FDA nod for the treatment of HIV-1

Commercial

• Amneal to acquire Kashiv for up to $1.1 billion

• Biogen acquires remaining rights to felzartamab from TJ Biopharma

• Eli Lilly’s Foundayo reaches 1,390 patients in first week

• Eli Lilly to acquire Kelonia Therapeutics for $7 billion

• Interna to collaborate with Daiichi Sankyo on drug delivery R&D

• UCB to acquire Neurona Therapeutics for $1.15 billion

In Brief

• AB Science claims “substantial” survival benefit for masitinib in ALS

• AstraZeneca’s Ultomiris hits primary endpoint in Phase III IgAN trial

• Biocon gets Health Canada nod for two denosumab biosimilars

• CDC won’t publish COVID vaccine report

• FDA accepts application for Roche’s obinutuzumab for SLE

• FDA approves Sanofi's Tzield to delay stage 3 T1DM in children

• FDA extends review of Sanofi’s Sarclisa subQ

• Formation Bio raises $615 million to accelerate clinical trials

• Inside the UK’s “secretive pharma” deal with the US

• Ionis presents new data for zilganersen in Alexander disease

• J&J’s Imaavy delivers long-term disease control in gMG

• Janux doses first patient with JANX014 in patients with mCRPC

• Kura reports data for its darlifarnib/cabozantinib combo in ccRCC

• Merck/Eisai’s Phase III LITESPARK-012 trial misses on PFS and OS in 1L RCC

• Novartis trims focus for anti-FXI mAb, abelacimab

• Novo’s semaglutide “might” have muscle advantage over Lilly’s tirzepatide

• Sanofi’s Nuvaxovid beats Moderna’s mNexspike on reactogenicity

• Taiho discontinues the Phase III STAR-121 trial in NSCLC due to futility

Summary

• Artificial Intelligence. AWS launched Amazon Bio Discovery and OpenAI introduced GPT-Rosalind in the same week, intensifying competition in AI-enabled drug discovery. Merck & Co. announced a $1 billion partnership with Google Cloud to deploy agentic AI across its entire enterprise, a broader in scope than the discovery-focused deals that have defined most pharma/AI collaborations. The UK government launched a £500 million Sovereign AI fund to back domestic AI startups and reduce dependence on US and Chinese platforms.

• Clinical. AstraZeneca completed a strong Phase III trifecta for tozorakimab in COPD, positioning it to extend biologic use beyond the eosinophil-high segment. Novo Nordisk's etavopivat met both co-primary endpoints in sickle cell disease, and as an oral once-daily agent could reach a far larger patient population than approved gene therapies costing $2–3 million per patient. Roche's satralizumab met its primary endpoint in MOGAD, a condition with no currently approved targeted treatments. On the negative side, fenebrutinib's Phase III data revealed a mortality imbalance and no significant benefit on disability progression, potentially weakening the mechanistic case for BTK inhibition in MS. A Cochrane review concluded anti-amyloid antibodies offer little clinically meaningful benefit in Alzheimer's, which is awkward timing for NICE's planned reassessment of lecanemab and donanemab.

• Regulatory. Moderna received EC approval for mCombriax, the first mRNA combination flu/COVID-19 vaccine, ahead of protein-based rivals from Novavax and Sanofi. The FDA approved Dupixent for children aged 2–11 years with CSU, Merck & Co.'s Enflonsia for RSV prevention in infants in Europe, and Idvynso as the first tenofovir-free two-drug HIV regimen to beat Biktarvy head-to-head. Ipsen's Ojemda received conditional EC approval as the first targeted therapy for relapsed/refractory paediatric low-grade glioma regardless of BRAF status.

• Commercial. Eli Lilly agreed to acquire Kelonia Therapeutics for up to $7 billion, adding an in vivo CAR-T generation platform to its growing genetic medicines portfolio. UCB will acquire Neurona Therapeutics for $1.15 billion, expanding into stem cell-based regenerative therapy for drug-resistant epilepsy. Amneal is acquiring Kashiv BioSciences for $1.1 billion to build a leading biosimilars company for the future. Biogen consolidated global rights to felzartamab for $100 million upfront, focusing on renal rather than oncology indications.

• In Brief. Highlights include: a CDC decision to suppress a COVID vaccine efficacy report amid concerns about political interference; the UK's pharmaceutical trade concessions to the US potentially costing up to £64 billion according to new analysis; positive Phase III data for zilganersen in Alexander disease ahead of FDA Priority Review; Merck/Eisai's LITESPARK-012 missing on both PFS and OS in first-line RCC; and Taiho/Arcus/Gilead discontinuing the STAR-121 NSCLC trial due to futility.

Artificial Intelligence

AWS launches Amazon Bio Discovery to accelerate AI-powered R&D

What happened? AWS has announced the launch of Amazon Bio Discovery, a new AI-powered application designed to help scientists design and test novel drugs more quickly and confidently. Amazon Bio Discovery gives scientists direct access to a broad catalogue of specialized AI models called biological foundation models (bioFMs) that are trained on vast biological datasets. These models generate and evaluate potential drug molecules, known as candidates, helping scientists accelerate antibody therapies during the early stages of drug discovery. With Amazon Bio Discovery, scientists can converse naturally in their preferred terminology with an AI agent to select the right models for their research goals, optimize the inputs, and evaluate candidates for experimentation. Scientists can also train models on their prior experimental data to make more accurate predictions. Furthermore, they can easily send candidates to physical labs for synthesis and testing, with results routing back to the application for rapid iteration, creating a lab-in-the-loop experimentation cycle.

What does this mean? Reading the PR, it sounds like Amazon's philosophy is to simplify and democratise access to AI solutions for drug discovery. In many ways, it’s a lot like what happened in banking and personal finance, as new players provided direct, intuitive access to investments, insurance, loans and trading, eliminating the need for traditional, complex, intermediary-heavy transactions. Traditional is still there for those that want it, but others can choose from Monzo, Starling, Revolut, Trading212 and a dozen other fintech platforms. Looking forward to seeing how this plays out, because there's significant competition from the likes of NVIDIA, Schrödinger, OpenAI (which recently partnered with Novo Nordisk), Insilico Medicine and dozens of other AI discovery companies.

Boehringer Ingelheim launches AI centre for pharma research in London

What happened? Boehringer Ingelheim has announced the launch of a new center for AI and machine learning in London, UK. This significant investment recognizes the UK’s commitment to AI and the life sciences sector. With this latest investment, Computational Innovation now has locations in Austria, Germany, UK and USA specializing in AI, machine learning, human genetics, and computational biology. The addition of London to the company’s global footprint and clear focus on AI will further understanding of the biology that drives patient outcomes and identify biological mechanisms with a higher probability of success.

What does this mean? Like many other large pharmas, Boehringer Ingelheim is investing significant resources into AI. In addition to the other locations, locating this in London’s Knowledge Quarter is a smart move as it’s already close to the Crick, The Wellcome Trust, and the British Library, offering proximity to precisely the kind of big brains Boehringer needs to ensure this investment is a success. Whether Boehringer's investment translates into tangible R&D productivity gains will take years to assess, but the direction of travel is aligned with where the science and investment is heading.

GEn1E and Rubicon Research announce AI-driven R&D pact

What happened? GEn1E Lifesciences (Gen1E) has announced a strategic partnership with Rubicon Research (Rubicon) and its US subsidiary, Validus Pharmaceuticals. GEn1E’s GRID AI platform integrates AI-powered endotyping with multi-omics and clinical data to identify biologically defined patient subgroups and enable targeted therapeutic development. The company’s lead program advanced from discovery to Phase II in approximately 2.5 years and has received FDA Fast Track designation. Rubicon Research is a global pharmaceutical company that will leverage GEn1E’s AI-powered endotyping engine to enable more precise patient stratification across its portfolio, including CNS programmes. GEn1E will, in turn, access Rubicon’s formulation development, manufacturing, and commercial capabilities to support late-stage development and commercialisation.

What does this mean? This announcement is very vague and almost entirely self-promotional. The GRID platform's claims of "order-of-magnitude cost reductions" and improved probability of regulatory success are unverified, and no clinical data from Phase II are disclosed.

Merck & Co. partners with Google on AI transformation

What happened? Merck & Co. and Google Cloud have announced a partnership to enhance Merck & Co.’s digital backbone as an AI-enabled enterprise. Valued at $1 billion, Google will deploy an agentic platform across Merck & Co.’s R&D, manufacturing, commercial, and corporate functions, and includes Google Cloud engineers working alongside Merck & Co. teams to deploy Google Cloud’s most sophisticated AI, including Gemini Enterprise.

What does this mean? Like OpenAI’s partnership with Novo Nordisk, which was announced recently, the scope of this deal is far broader than traditional pharma/AI collaborations. All of Merck & Co.’s AI deals thus far have focused on discovery/R&D (e.g., Infinimmune, Tempus AI, Mayo Clinic). As confidence in AI platforms increases, pharma companies are likely to want to integrate AI into a broader range of functions to drive efficiency and improve productivity. I do worry about how this will affect humans, however. Job losses are inevitable.

OpenAI launches GPT-Rosalind

What happened? OpenAI continues its journey into life sciences with the launch of GPT‑Rosalind, a reasoning model built to support research across biology, drug discovery, and translational medicine. This comes in the same week as Amazon announcing the launch of Amazon Bio Discovery (see above). The model supports evidence synthesis, hypothesis generation, and experimental planning across biochemistry, genomics, and drug discovery. It can query specialised databases, parse scientific literature, interact with computational tools, and suggest experimental pathways within a single interface. Alongside the model, OpenAI is also introducing a Life Sciences research plugin for Codex that connects to more than 50 scientific tools and data sources. Early customers include Amgen, Moderna, the Allen Institute, and Thermo Fisher Scientific.

What does this mean? With a nod Rosalind Franklin, whose X-ray crystallography work was foundational to understanding DNA structure, it seems fitting that OpenAI’s new model that focuses on structural biology and drug discovery is named after such a pioneer. The timing is also interesting, coming very soon after the launch of Amazon Bio Discovery and OpenAI’s partnership with Novo Nordisk. As I’ve queried before, whether general-purpose reasoning models can match the performance of domain-specific tools in areas like protein structure prediction and molecular design remains to be seen.

UK government announces launch of Sovereign AI

What happened? British AI startups working in fields that could transform everyone’s lives for the better, and that will be critical to the UK’s national security, are set to receive support through the Sovereign AI Unit, a £500 million first-of-its-kind national effort to back Britain’s smartest founders and keep the future of AI built on British shores. Sovereign AI is designed to be different from any previous government-backed unit, acting like a venture capital fund with the muscle of the state behind it, moving fast, backing ambition and cutting through the red tape that so often holds brilliant ideas back. It will invest directly in the UK’s most promising AI startups, help them scale quickly, and give them the support they need to compete with the best in the world. Sovereign AI’s first equity investment will be in the AI infrastructure startup Callosum.

What does this mean? This is an industrial strategy play for the UK government as much as a science policy one. The explicit framing (i.e., Britain as an "AI maker, not an AI taker") reflects anxiety about dependence on US and Chinese technology platforms, and a desire to capture economic value from AI domestically rather than licensing it back from abroad. For pharma and biotech, State-backed compute access and fast-track visa decisions lower the barriers to entry for UK-based AI drug discovery startups, potentially accelerating a generation of companies that larger players will eventually want to acquire or partner with.

Clinical

AstraZeneca posts more positive Phase III data for tozorakimab in COPD

What happened? AstraZeneca has announced positive high-level results from the Phase III MIRANDA trial which show treatment with tozorakimab (anti-IL-33 mAb) is associated with a statistically significant and clinically meaningful reduction in the annualised rate of moderate-to-severe COPD exacerbations, an affect seen in the primary population of former smokers and in the overall population, which included former and current smokers, and patients across all blood eosinophil counts and all stages of lung function severity. Tozorakimab was generally well tolerated with a favorable safety profile consistent with previous trials. In MIRANDA, patients received tozorakimab 300mg or placebo on top of standard of care (i.e., ICS/LABA/LAMA triple therapy, or dual therapy if triple is not considered appropriate) once every two weeks. The trial enrolled n=1,454 patients with COPD still experiencing moderate-to-severe exacerbations while on inhaled standard of care. 

What does this mean? And the hits just keep on coming for tozorakimab! This positive data from MIRANDA (tozorakimab dosed Q2W) comes only a few weeks after positive data from two other COPD trials, OBERON and TITANIA (tozorakimab dosed Q4W) were announced. Collectively, this data set should now enable AstraZeneca to broaden COPD biologic use beyond the type-2 high segment (i.e., blood eosinophils >300 cells/µl) where other treatments (e.g., Dupixent) are established.

Cochrane review analyses the effect of anti-amyloid mAbs in Alzheimer’s

What happened? A new analysis by the Cochrane Review has shown that drugs that target amyloid beta proteins in the brain likely have no clinically meaningful positive effects, while increasing the risk of bleeding and swelling in the brain. The new review examined data from 17 clinical trials with a total of 20,342 participants, all looking at the impact of anti-amyloid drugs on people with mild cognitive impairment or mild dementia due to Alzheimer’s disease. Drugs that were included in the analysis include aducanumab (Aduhelm; Biogen/Eisai), bapineuzumab (Pfizer/J&J), crenezumab (Roche/Genentech), donanemab (Kisunla; Eli Lilly), gantenerumab (Roche), lecanemab (Leqembi; Biogen/Eisai), and solanezumab (Eli Lilly). Proponents of these drugs have theorized that they would be more effective at these earlier stages before the disease has progressed.

What does this mean? The core finding of the review is that that drugs which successfully clear amyloid-beta from the brain produce little or no clinically meaningful improvement in cognition or function, a finding that calls into question the utility of the entire drug class. However, objections to the review have been quick to appear. The central objection is that the review pools seven different drugs, several of which failed in trials, alongside lecanemab and donanemab, which have received regulatory approval based on data showing measurable slowing of decline. Eisai has described the analysis as "scientifically deeply flawed" for combining failed antibodies with approved treatments. Alzheimer's Research UK noted there is no universally agreed definition of "clinically meaningful," and that current trial measures may not capture what matters most to patients and families. Notwithstanding these assertions, the amyloid hypothesis has not delivered the clinical outcomes once hoped for in Alzheimer’s disease, but the question of whether the most recent, better targeted mAbs (i.e., lecanemab and donanemab) represent genuine progress remains to be seen. For NICE, which recently announced it will reassess its rejection of lecanemab and donanemab under new cost-effectiveness thresholds, the timing is awkward.

Eli Lilly posts Phase III safety data for Foundayo

What happened? Eli Lilly has posted positive topline results from the Phase III ACHIEVE-4 trial evaluating the efficacy and safety of orforglipron (Foundayo) compared to insulin glargine in adults with type 2 diabetes and obesity or being overweight at increased cardiovascular risk (n=2,700 participants). In the trial, Foundayo met the primary endpoint by demonstrating a non-inferior risk of major adverse cardiovascular events (MACE-4), including cardiovascular death, heart attack, stroke or hospitalization for unstable and sudden chest pain, compared to insulin glargine. In addition, Foundayo showed superior improvements in A1C and body weight at 52 weeks vs. insulin glargine, which persisted through 104 weeks of therapy. While not controlled for multiplicity, the risk of all-cause death was significantly lower for Foundayo vs. insulin glargine. The overall safety and tolerability profile of Foundayo in ACHIEVE-4 was generally consistent with previous trials and with the GLP-1 class. 

What does this mean? Demonstrating non-inferior cardiovascular risk versus insulin glargine was something the FDA asked for in Foundayo’s approval letter, so this is mission accomplished for Eli Lilly. The more notable data point is the exploratory finding of lower all-cause mortality versus insulin glargine, which, if real, would be clinically significant. However, as Eli Lilly itself notes, this endpoint was not controlled for multiplicity. In a trial with multiple secondary and exploratory endpoints, a positive result on one that was not pre-specified as a primary or multiplicity-adjusted endpoint has an elevated probability of being a statistical artefact rather than a true effect. This is a hypothesis worth testing in a dedicated trial, but it should not be presented or interpreted as a confirmed mortality benefit.

Merck & Co.’s MK-2010: early data, no clear plan

What happened. Merck & Co. has shared the first clinical data for its PD-1xVEGF bsAb MK-2010 (licensed from LaNova Medicines in November 2024). In a small Chinese Phase I/II trial, the drug produced an unconfirmed ORR of 55% in 11 treatment-naïve patients with PD-L1-positive NSCLC at the 20mg/kg dose, which is broadly in line with rivals ivonescimab (50%), pumitamig (47%) and Pfizer's PF-08634404 (55-62%). Safety was broadly manageable, though the higher 30mg/kg dose showed a notably worse tolerability profile, including more SAEs. Merck indicated 20mg/kg is the preferred dose for further study.

What does this mean? Some interesting data, but this news is more notable is what Merck & Co. hasn't said: unlike Summit/Akeso, BMS/BioNTech and Pfizer, all running multiple Phase III trials with its PD-1xVEGF bsAb, Merck & Co. has disclosed no pivotal development plans. With resources increasingly directed towards its TROP2 ADC programme (sacituzumab tirumotecan), analysts are openly questioning how seriously Merck & Co. intends to compete in this space.

Moderna posts Phase I/II data for mRNA-4359 in melanoma

What happened? Moderna has announced the release of data from a Phase I/II study of mRNA-4359, an investigational cancer antigen therapy. The data reports safety, efficacy and translational data from a Phase II dose-expansion cohort of the study which evaluated mRNA-4359 in combination with pembrolizumab as a 1L therapy in patients with locally advanced or metastatic melanoma. Among 12 participants, the combination of mRNA-4359 and pembrolizumab resulted in an ORR of 83% (95% CI: 52%-98%), with two patients achieving a complete response and eight achieving a partial response, as well as a disease control rate (DCR) of 92% (95% CI: 62%-100%). The median time to response was six weeks (range, 5.6-24.0). Responses occurred across baseline tumour PD-L1 expression categories, with an ORR of 88% (95%CI: 47%-100%) among patients with PD-L1+ (TPS≥1%) tumours, and 67% (95%CI: 9%-99%) among patients with PD-L1- (TPS<1%) tumours. Consistent with mRNA-4359's mechanistic rationale, antigen-specific T-cell responses and novel expanded T-cell receptor (TCR) clonality was observed in all patients (n=7) with evaluable samples. mRNA-4359 plus pembrolizumab demonstrated a manageable safety profile, with no new immune-related adverse events.

What does this mean? An 83% ORR in a 12-patient cohort is an eye-catching number, but the numbers are too preliminary and the patient numbers too small to draw firm conclusions. Perhaps more relevant is that is the mechanistic signal that the data sends, inasmuch antigen-specific T-cell responses and novel TCR clonality were observed in all evaluable patients, which is the pharmacological effect the therapy is designed to produce. The response rate across both PD-L1-positive and PD-L1-negative subgroups is also notable, though again, patient numbers in each subgroup are very small. What Moderna needs now is a larger, randomised trial to establish whether the observed responses are durable and whether the combination genuinely outperforms checkpoint inhibition alone.

Monopar posts positive Phase III data for ALXN1840 in Wilson’s disease

What happened? Monopar Therapeutics has announced new analyses from the Phase III FoCus trial of ALXN1840 (tiomolibdate choline) showing greater neurologic benefit versus standard of care (SoC) in Wilson disease patients with neurologic symptoms at baseline. Longer-term follow-up showed that neurologic benefits in ALXN1840-treated patients continued to increase and were sustained for roughly three years, with consistent efficacy in both treatment-naïve and treatment-experienced subgroups. Across Phase II and III studies totaling 266 patients and 645 patient-years of exposure, ALXN1840 also exhibited a favourable safety profile, with low rates of drug-related SAEs and no treatment-related deaths, reinforcing its potential to reshape neurologic management in Wilson disease. Recall, Monopar licensed rights to ALXN1840 from AstraZeneca in October 2024.

What does this mean? Wilson's disease is a rare, life-threatening genetic disorder of copper metabolism, and current standard-of-care options (i.e., D-penicillamine and trientine) have significant tolerability issues, particularly for patients with neurological involvement, in whom treatment can initially worsen symptoms. ALXN1840 offers a different mechanism, working as a copper chelator that acts in the gut to reduce absorption rather than mobilising copper from tissue, which is theorised to reduce the neurological deterioration risk associated with existing therapies. This Phase III data is genuinely encouraging for a condition with limited options. Regulatory submissions will be the next milestone to watch.

Novo’s etavopivat effective in late-stage sickle cell disease trial

What happened? Novo Nordisk has announced the topline results from the Phase III HIBISCUS trial which assessed the efficacy and safety of etavopivat in adults and adolescents with sickle cell disease (SCD). In the trial, people treated with etavopivat demonstrated a superior reduction in the annualised rate of vaso-occlusive crises (VOCs) of 27% compared to placebo. The time to first VOC was significantly prolonged with etavopivat, with a median time to first VOC of 38.4 weeks versus 20.9 weeks for placebo. Additionally, etavopivat demonstrated a superior increase in the proportion of people achieving a haemoglobin response greater than 1g/dL at week 24 of 48.7% compared to

7.2% with placebo, corresponding to an adjusted rate difference of 41.2%¹. Further, as an exploratory analysis, etavopivat significantly reduced the risk of blood transfusion. In the trial, etavopivat appeared to be well tolerated, with a topline safety profile in line with previous etavopivat trials. Etavopivat is an oral, once-daily, pyruvate kinase-R (PKR) activator.

What does this mean? Sickle cell disease has seen its fair share of good and bad news over the years, so this news from Novo is very much needed in an indication that continues to have high levels of unmet need. First the good: gene therapies from Vertex/CRISPR Therapeutics (Casgevy) and bluebird bio (Lyfgenia) have been approved in Europe, the US and the UK. However, here’s the rub: with list prices of $2-3 million per patient in the US, with a UK price of well over £1 million, access is severely limited. On the flip side, certain treatments have been withdrawn from the market, either by the regulators or voluntarily, including crizanlizumab (Adakveo; Novartis) and voxelotor (Oxbryta; Pfizer). There is no doubt in my mind that the SCD community will be celebrating the data for etavopivat. An oral treatment that reduces VOCs without the complexity of gene therapy or the immunosuppression burden of bone marrow transplant would address a substantially larger, more accessible patient population globally. And be cheaper. As with other late-stage assets discussed in this note, regulatory submissions will be the next milestone to watch.

Roche’s Enspryng delivers in Phase III MOGAD trial

What happened? Roche has announced new data from the Phase III METEOROID study demonstrating that satralizumab (Enspryng) reduced the risk of a new relapse by 68% compared to placebo in adults and adolescents with myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), meeting its primary endpoint (time from randomisation to the first MOGAD relapse during the double-blind treatment period). Satralizumab also reduced the annualised relapse rate (ARR) by 66%, a key secondary endpoint. The only secondary endpoint miss was on the annualised rate of inpatient hospitalisations where satralizumab was associated with a numerical 17% reduction. No new safety signals were reported.

What does this mean? No targeted treatments currently exist for MOGAD, with current management relying on two broad strategies: treating acute attacks and trying to prevent future ones. For acute attacks, high dose steroids are typically used (e.g., methylprednisolone), with immunosuppressants used to prevent relapses (e.g., azathioprine). Data from the METEROID trial shows that treatment with satralizumab caused a 73% reduction in rescue therapy use (i.e., steroids), suggesting satralizumab is meaningfully reducing the kind of acute, severe attacks that currently drive patients into hospital for IV steroids or plasma exchange. That's a massive improvement on the current standard of care.

Roche initiates new Phase III study for Elevidys

What happened? Roche has announced that it intends to initiate a new, global, pivotal Phase III study for Elevidys (delandistrogene moxeparvovec), the first approved gene therapy to treat the underlying cause of Duchenne muscular dystrophy (DMD). Following feedback from the EMA and the Duchenne community, Roche aims to generate additional placebo-controlled data required for a regulatory resubmission with the EMA and address the urgent needs for ambulatory boys living with DMD in Europe and other regions across the world. The study will evaluate the efficacy and safety of Elevidys compared to placebo over 72 weeks in approximately 100 early ambulatory boys with DMD. The primary endpoint of the study is the change in "Time to Rise" (TTR) from the floor velocity, which is a critical prognostic factor of future disease progression. Participants initially in the placebo group will be eligible to receive Elevidys after the primary 72-week period.

What does this mean? By initiating this study, Roche has sent a very clear signal to the DMD community that it’s committed to getting Elevidys approved in Europe. However, the trial’s placebo-controlled design is likely to draw criticism.

Roche presents new data on fenebrutinib in RMS

What happened? Roche has announced new data from Phase III FENhance 1 and 2 studies,  positive topline details (including the primary endpoint of annualised relapse rate) were announced in November 2025 and March 2026. What’s news in this announcement is details on the imbalance with respect to reported fatalities across both studies and secondary endpoints. In FENhance 1 and 2, there was one death (0.1%) in the teriflunomide arm and seven deaths (0.9%) in the fenebrutinib arm during the reporting period. Overall, in the fenebrutinib arm, deaths occurred at different timepoints and were caused by various causes including infections (neuro cryptococcosis gattii and pneumonia), complications of type 1 diabetes, serious bleeding, suicide, injuries from accident and death of unknown cause. A similar imbalance in deaths was reported for the Phase III FENtrepid trial which showed non-inferiority of fenebrutinib vs ocrelizumab (Ocrevus; Roche) in reducing disability progression in patients with PPMS.  Secondary endpoints showed that fenebrutinib significantly reduced disease activity in the brain, as evidenced by MRI scans. Fenebrutinib reduced markers of active inflammation by 70.7% (p<0.0001) in FENhance 1 and 77.6% (p<0.0001) in FENhance 2 compared with teriflunomide, as measured by new T1 gadolinium-enhancing (T1-Gd+) lesions. Chronic disease burden was reduced by 76.0% (p<0.0001) in FENhance 1 and 82.5% (p<0.0001) in FENhance 2 with fenebrutinib compared with teriflunomide, as measured by new or enlarging T2 lesions. However, fenebrutinib only achieved a numerical advantage over teriflunomide on a disability assessment in RMS. Recall, the PPMS trial found fenebrutinib was noninferior to ocrelizumab on a disability endpoint. 

What does this mean? While tragic, the absolute death numbers are small. Moreover, the causes are heterogeneous, and this mix of causes makes a single drug-related mechanism less likely as the explanation. However, the two infection-related deaths (particularly neuro cryptococcosis) will attract regulatory attention. BTK inhibitors as a class carry infection risk, and a CNS-penetrant one like fenebrutinib that targets both B cells and microglia could plausibly increase susceptibility. In terms of fenebrutinib’s impact on secondary endpoints, the disability progression data are disappointing. The whole mechanistic argument for fenebrutinib, and for BTK inhibitors in MS more broadly, is that targeting both peripheral B cells and CNS microglia should address not just relapse biology but also the smouldering inflammation driving disability progression. If disability progression isn't significantly reduced, the BTK inhibitor argument loses some force.

Regulatory

EC approves Moderna’s combo flu/COVID-19 vaccine, mCombriax

What happened? Moderna has announced that EC has approved mCOMBRIAX (mRNA-1083), Moderna's combination vaccine indicated for active immunization for the prevention of influenza and COVID-19 caused by SARS-CoV-2 in individuals 50 years of age and older. The approval was supported by results from a Phase III clinical trial (NCT06097273) which evaluated the safety, reactogenicity and immunogenicity of mRNA-1083 in two independent age cohorts of approximately 4,000 adults each. One cohort included adults 65 years of age and older and compared mRNA-1083 to co-administered Fluzone HD (licensed in the European Union as Efluelda), a high-dose influenza vaccine, and Spikevax, Moderna's licensed COVID-19 vaccine. The second cohort included adults 50-64 years of age and compared mRNA-1083 to co-administered Fluarix, a standard-dose influenza vaccine, and Spikevax. All primary endpoints demonstrating the non-inferiority of immune responses were met. mRNA-1083 also demonstrated an acceptable safety and tolerability profile.

What does this mean? This is the first mRNA-based vaccine approved to target both COVID-19 and seasonal influenza in adults aged 50 and over. The approval was based on Phase III data demonstrating non‑inferiority on all primary immunogenicity endpoints and statistical superiority on many of them compared with separately administered authorised vaccines. For regulators, this represents a significant step in integrating mRNA technology into broader seasonal vaccination programmes, signalling confidence in both safety and immunogenicity. With approval in hand, mCombriax positions Moderna ahead of emerging competitors, including Novavax and Sanofi which are developing a protein-based COVID-19/influenza combination. By securing approval, Moderna establishes an important precedent for the approval pathway of future combination vaccines, potentially smoothing subsequent filings across Europe.

FDA approves Dupixent for treatment of paediatric CSU

What happened? The FDA has approved Dupixent (dupilumab) for the treatment of children aged 2-11 years with chronic spontaneous urticaria (CSU) who remain symptomatic despite histamine-1 antihistamine (H1AH) treatment. This expands the previous approval for Dupixent in adults and adolescents aged 12 years and older with CSU. The approval is based on data from two Phase III clinical studies in the LIBERTY-CUPID programme (Study A and Study C; NCT04180488). PK data from the Phase III CUPIDKids study in children aged 2 to 11 years with CSU was also used.

What does this mean? Dupixent is now the first and only targeted medicine in the US for this vulnerable young population who do not respond to standard antihistamines. This expansion of biologics into younger age groups sets the stage for a step-change in how CSU is managed, with the next phase in CSU treatment likely to be defined by increased potency and extended duration of action. Companies such as GSK and Novartis have both made big bets on longer-acting anti-IgE assets, which is another modality used in the treatment of CSU. GSK’s January 2026 acquisition of RAPT Therapeutics included ozureprubart, a long-acting anti-IgE mAb, which demonstrated comparable efficacy with less frequent dosing, and similar safety and tolerability compared with omalizumab (Xolair). And in March 2026, Novartis acquired Excellergy to gain access to EXL-111, a half-life extended, high-affinity anti-IgE mAb currently in Phase I trials.

GSK’s Blenrep approved in China for treatment of 2L+ R/RMM

What happened? GSK has announced that China’s National Medical Products Administration (NMPA) has approved Blenrep (belantamab mafodotin) in combination with bortezomib and dexamethasone (BVd) for the treatment of adults with relapsed or refractory multiple myeloma (R/RMM) who have received at least one prior line of therapy.  Approval was supported by data from the pivotal Phase III DREAMM-7 trial. These include statistically significant and clinically meaningful PFS and OS results for the Blenrep combination versus a daratumumab-based triplet combination with bortezomib and dexamethasone (DVd). The safety and tolerability profiles of the Blenrep combination were broadly consistent with the known profiles of the individual agents.

What does this mean? Blenrep’s approval in China follows approval in the US back in October 2025. While both approvals are supported by data from the DREAMM-7 trial, the FDA has taken the more conservative position vs the NMPA and approved Blenrep in the 3L+ setting compared to the NMPA’s 2L+ setting. The FDA also imposed an additional constraint on what those prior lines must have included (a proteasome inhibitor and an immunomodulatory agent), reflecting the standard of care in the US where most patients will have received agents like lenalidomide and bortezomib upfront. The NMPA label appears less prescriptive on that point, which may reflect differences in how myeloma is typically managed in China. Interestingly, in April 2025 the MHRA approved Blenrep in the 2L+ setting, aligning with China rather than the US.

Ipsen’s Ojemda receives EC approval for paediatric low-grade-glioma

What happened? Ipsen has announced that the EC has granted conditional marketing authorization for Ojemda (tovorafenib) as monotherapy for the treatment of patients 6 months of age and older with paediatric low-grade-glioma harbouring a BRAF fusion or rearrangement, or BRAF V600 mutation, who have progressed after one or more prior systemic therapies. The approval is based on data from the pivotal Phase II FIREFLY-1 study which evaluated tovorafenib in 137 children and young adults with relapsed or refractory BRAF-altered pLGG who had received at least one prior systemic therapy. Results showed a clinically meaningful tumour response, rapid and durable responses, a manageable safety profile and convenient dosing.

What does this mean? Recall, Ipsen licensed ex-US rights to tovorafenib from Day One in July 2024. As a side note, Day One was acquired by Servier in March 2026, a move that has had no noticeable impact on Ipsen’s rights to the asset. Tovorafenib is the first approved targeted therapy for R/R paediatric low-grade glioma (pLGG) regardless of BRAF alteration status in the EU, creating a new standard of care in a setting where children previously relied on chemotherapy regimens. The BRAF inhibitor dabrafenib (Novartis), used in BRAF V600-mutated pLGG, has been the nearest comparator, but tovorafenib's broader applicability across BRAF fusions and rearrangements as well as BRAF V600 mutations gives it a wider eligible population. The caveat is that the EC approval is conditional, meaning continued authorisation depends on confirmatory data, expected from the ongoing Phase III FIREFLY-2 study in the front-line setting. The FIREFLY-1 dataset, while encouraging, was a single-arm Phase II study in 137 patients without a randomised comparator.

LEO Pharma receives China’s NMPA approval of Enstilar

What happened? LEO Pharma has announced that China's National Medical Products Administration (NMPA) has approved Enstilar (calcipotriene/betamethasone dipropionate) to treat adult patients in China living with plaque psoriasis. The NMPA approval was supported by results from a Phase III trial comparing the efficacy and safety of a once daily application with Enstilar foam and Daivobet (calcipotriene/betamethasone) ointment after 4 weeks of treatment in adult Chinese subjects with stable plaque psoriasis (n=604). The trial achieved its primary and secondary endpoints, with Enstilar demonstrating superiority to Daivobet ointment.

What does this mean? Adding China, the world's largest single market by patient numbers, to the more than 50 countries where Enstilar is already approved strengthens LEO Pharma’s position as a global leader in topical treatments for psoriasis. The Chinese topical psoriasis market has historically been dominated by generic corticosteroids and older combination products. As such, Enstilar’s approval and subsequent launch will represent a meaningful step up in treatment quality for a market increasingly receptive to innovative dermatology options. Rival topical manufacturers operating in China will face a well-evidenced, branded competitor with a strong global track record.

Merck & Co.’s Enflonsia approved in Europe for RSV prevention in infants

What happened? Merck & Co. has announced that the European Commission (EC) has approved Enflonsia (clesrovimab) for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease in neonates (newborns) and infants during their first RSV season. This follows Enflonsia’s FDA approval in June 2025. Clesrovimab is a preventive, long-acting mAb designed to provide direct, rapid and durable protection through 5 months, a typical RSV season, with non-weight-based dosing. It’s a fully human IgG1κ mAb that targets the RSV outer membrane fusion protein, preventing virus entry into cells. The EC approval is supported by results from the pivotal Phase IIb/III CLEVER trial, which evaluated the safety and efficacy of a single dose of clesrovimab administered to preterm and full-term infants (birth to 1 year of age), as well as interim data from RSV season 1 of the Phase III SMART trial evaluating the safety, efficacy and pharmacokinetics of clesrovimab versus palivizumab (Synagis) in infants at increased risk for severe RSV disease.

What does this mean? Once commercially launched, Enflonsia will be competing with AstraZeneca/Sanofi’s Beyfortus (nirsevimbab), which was approved for a similar indication in November 2022. Enflonsia will also be competing against Pfizer’s Abrysvo (RSV vaccine), which is the only vaccine with an approved maternal immunisation indication in the EU. GSK’s Arexvy and Moderna’s mResvia are currently indicated for direct adult protection only.

Merck & Co.’s Idvynso gets FDA nod for the treatment of HIV-1

What happened? Merck & Co. has announced that the FDA has approved Idvynso, a single pill combo regimen that includes islatravir (0.25mg) and doravirine (100mg) for the treatment of HIV-1 infection in adults to replace the current antiretroviral regimen in those who are virologically suppressed (HIV-1 RNA less than 50 copies per mL) on a stable antiretroviral regimen with no history of virologic treatment failure and no known substitutions associated with resistance to doravirine. While doravirine (NNRTI) has been on the US market since September 2019, islatravir (NRTTI) is a novel medicine that’s never been approved before. The approval of Idvynso was supported by Week 48 data from two Phase III, randomised, active-controlled, non-inferiority trials: Trial 052 and Trial 051. Across the two trials, a total of 708 participants received once-daily Idvynso. Both studies tested Idvynso in patients on baseline antiretroviral therapy and those who manage their HIV with Gilead’s leading Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide). The studies showed that Idvynso was non-inferior to both comparators while offering a comparable safety profile.

What does this mean? Idvynso is the first non-integrase strand transfer inhibitor (INSTI), tenofovir-free, once-daily two-drug regimen to demonstrate non-inferiority to three-drug regimen Biktarvy in a head-to-head Phase III trial. The tenofovir-free profile addresses a genuine clinical need for patients with renal concerns or bone density issues, populations where prolonged tenofovir exposure can be problematic. One potential wrinkle is islatravir's development history. Earlier development was paused following observations of CD4+ T-cell count reductions at higher doses in some studies, raising immunological questions that required resolution before this programme could proceed. Additional islatravir-based once-weekly regimens are in Phase III trials, including a combination with Gilead's lenacapavir that’s being assessed head-to-head vs. Biktarvy.

Commercial

Amneal to acquire Kashiv for up to $1.1 billion

What happened? Amneal Pharmaceuticals has announced that it has entered into a definitive agreement to acquire 100% of Kashiv BioSciences for $1.1 billion (made up of $375 million cash, $375 million equity and up to $350 million in milestone payments). A leading driver of this acquisition is to establish an integrated biosimilar platform enabling multiple new biosimilars launches each year, including biosimilar versions of Keytruda and Opdivo. By 2030, Amneal expects to have more than 12 commercial biosimilars and over 20 more products in the pipeline.

What does this mean? This is a bold vertical integration play for Amneal Pharmaceuticals as it seeks to capture a wave of biologics patent expiries. The biosimilar market is consolidating rapidly ahead of a projected $300 billion global biologics LOE opportunity through the 2030s. By 2030, Amneal projects it will have more than 12 commercial biosimilars and over 20 more products in the pipeline, a scale that would make it a serious rival to established biosimilar players such as Sandoz, Celltrion, and Pfizer's biosimilars division. Vertical integration, where Amneal will own both development and manufacturing, will enable faster timelines and better margin protection.

Biogen acquires remaining rights to felzartamab from TJ Biopharma

What happened? Biogen and TJ Biopharma have announced that the companies have entered into a definitive agreement under which Biogen will acquire TJ Bio’s exclusive rights to felzartamab (anti-CD38 mAb) in the Greater China Region. Subject to closure, Biogen will own exclusive worldwide rights to felzartamab, which is currently being evaluated in global Phase III clinical studies across multiple immune-mediated diseases, including kidney transplantation, IgAN and PMN. Under the terms of the agreement, TJ Bio will receive a $100 million upfront payment and is eligible to receive up to $750 million in potential commercial and sales milestone payments, plus mid-single-digit to low-double-digit percentage of royalties on potential net sales in the Greater China Region. Recall, felzartamab was originally developed by MorphoSys (acquired by Novartis in February 2024).

What does this mean? By acquiring TJ Biopharma's exclusive rights to felzartamab in the Greater China Region, Biogen now holds exclusive worldwide development and commercialisation rights to the asset. Felzartamab is a CD38-directed mAb, which is the same MOA as Darzalex (daratumumab; J&J) and Sarclisa (isatuximab; Sanofi), both of which are indicated for the treatment of multiple myeloma. Interestingly, Biogen is not positioning felzartamab primarily in myeloma, instead focusing on renal indications such as IgAN and PMN, where CD38-directed depletion of plasma cells offers a differentiated mechanism compared to other approved treatments (e.g., sparsentan). Assuming Biogen remains focused on renal indications, the China BLA for felzartamab in multiple myeloma, submitted in January 2025, is likely to be withdrawn.

Eli Lilly’s Foundayo reaches 1,390 patients in first week

What happened? Eli Lilly’s Foundayo (orforglipron) has hit 1,390 prescriptions in its first week on the US market, prompting some market commentators to suggest the launch has been “lukewarm” compared to the launch of Novo Nordisk’s oral version of Wegovy (semaglutide) which hit the US market on January 5, 2026.

What does this mean? Back in January 2026, Reuters reported that Novo Nordisk’s oral version of Wegovy (semaglutide) hit 18,000 prescriptions in its first week on the US market. Following the release of new data, this figure now looks more like two weeks. According to a report by UBS (April 17, 2026), Novo’s oral Wegovy only hit 3,071 prescriptions in its first week on the market (five days between January 5-9, 2026). Conversely, Foundayo made commercially available on April 9, 2026, so the 1,390 prescriptions reported were only made over the course of a 1-2 days, although this is not clear. More data that compares similar time periods will be needed before any conclusions about a “lukewarm” launch for Foundayo can be made.

Eli Lilly to acquire Kelonia Therapeutics for $7 billion

What happened? Eli Lilly (Lilly) and Kelonia Therapeutics (Kelonia) have announced a definitive agreement for Eli Lilly to acquire Kelonia. Kelonia has developed a proprietary in vivo gene placement system that uses specially engineered lentiviral-based particles designed to efficiently and selectively enter T-cells inside the body, allowing the patient's own body to generate CAR-T therapies that can treat underlying disease. Kelonia's lead program, KLN-1010, is an investigational, one-time intravenous gene therapy that generates anti-BCMA CAR-T cells, targeting the BCMA protein expressed on the surface of multiple myeloma cells. Data presented at ASH 2025 from the first three patients dosed in the ongoing Phase I inMMyCAR study demonstrated that promising clinical activity and manageable toxicities are feasible with an off-the-shelf in vivo CAR-T in MM. Under the terms of the agreement, Lilly will acquire Kelonia, and Kelonia shareholders will receive up to $7 billion in cash, inclusive of an upfront payment of $3.25 billion, and subsequent payments upon achievement of certain clinical, regulatory and commercial milestones.

What does this mean? The acquisition of Kelonia aligns with Eli Lilly’s push (via M&A and licensing) into genetic medicines by adding a broadly applicable in vivo gene delivery platform that could ultimately extend beyond MM into other haematologic malignancies and non-oncology indications. Other notable deals include a partnership with MeiraGTx focused on gene therapies in ophthalmology, the acquisition of Adverum Biotechnologies to gain access to ixoberogene soroparvovec, an intravitreal gene therapy being developed for the treatment of wet age-related macular degeneration (wAMD), and the acquisition of Orna Therapeutics to gain access to an in vivo CAR-T platform. Data for Orna’s lead programme, ORN-252 (anti-CD19 panCAR programme), was presented in December 2025.

Interna to collaborate with Daiichi Sankyo on drug delivery R&D

What happened? Interna Therapeutics has announced a research collaboration with Daiichi Sankyo to evaluate the use of Interna’s Molecular Nano Motor (MNM) technology as a delivery enhancer for targeted therapeutic modalities. Under the terms of the agreement, the collaboration will focus on integrating MNM molecules with targeting approaches to improve intracellular delivery, functional activity, and overall therapeutic performance across selected programmes. Interna’s MNM platform is designed to improve the efficiency of intracellular delivery of a broad range of modalities, including nucleic acids and other macromolecules, without reliance on traditional delivery systems. By enhancing intracellular access and tissue penetration, MNM has the potential to improve the efficacy of targeted therapies across multiple indications.

What does this mean? Details are sketchy on what the collaboration will focus on. However, given Daiichi Sankyo's position as the world's leading ADC developer, the pairing with an intracellular delivery enhancer is an obvious strategic fit.

UCB to acquire Neurona Therapeutics for $1.15 billion

What happened? UCB has announced that it has entered into a definitive agreement under which UCB would acquire Neurona Therapeutics, including lead asset NRTX-1001, adding to UCB’s epilepsy portfolio. Neurona’s platform uses regenerative pluripotent stem cell technology to deliver cells aimed at structurally and functionally restoring compromised neural circuitry. NRTX‑1001 is being investigated in Phase I/II clinical trials to evaluate the safety, tolerability, and effects treatment with NRTX-1001 on seizure frequency in drug-resistant unilateral and bilateral mesial temporal lobe epilepsy (mTLE) with and without Mesial Temporal Sclerosis (MTS). Based on encouraging preliminary data, NRTX-1001 received RMAT designation from the FDA for drug‑resistant mesial temporal lobe epilepsy (MTLE) in June 2024, and PRIME designation from the EMA for adults with drug‑resistant focal epilepsy in November 2025.

What does this mean? UCB has a 30+ year heritage in epilepsy, establishing itself as a global leader in providing medications for diverse seizure types and age groups. The company pioneered the SV2A class of medicines (e.g., levetiracetam), introducing novel mechanisms of action to the modern epilepsy landscape. The acquisition of Neurona Therapeutics signals an expansion into regenerative medicine with the aim of disease modification in epilepsy. Early data for NRTX-1001 in drug-resistant unilateral and bilateral mTLE are compelling, with large reductions in disabling seizures seen in post-treatment follow-up.

In Brief

AB Science claims “substantial” survival benefit for masitinib in ALS

Via a reprint in medRxiv, AB Science has reported a long-term survivor analysis from the AB10015 trial, suggesting a 5-year survival rate of 42.3% with masitinib vs historical benchmarks of roughly 7-28%. Among those who reached the 5-year milestone, median overall survival was 121 months versus 42 months predicted by the ENCALS model.

AstraZeneca’s Ultomiris hits primary endpoint in Phase III IgAN trial

Positive high-level results from a prespecified interim analysis of the I CAN Phase III trial showed that Ultomiris (ravulizumab) met its primary endpoint, demonstrating a statistically significant and clinically meaningful reduction of proteinuria, based on 24-hour urine protein creatinine ratio (UPCR), at week 34 in adults with immunoglobulin A nephropathy (IgAN) who are at risk of disease progression. The primary endpoint of change from baseline in estimated glomerular filtration rate (eGFR) will be measured at week 106.

Biocon gets Health Canada nod for two denosumab biosimilars

Biocon Biologics has received approval from Health Canada for Bosaya and Aukelso, biosimilars of Amgen's Prolia and Xgeva, respectively. Bosaya, a 60 mg/mL prefilled syringe for subQ use, has been approved for the treatment of postmenopausal women and men with osteoporosis at high risk of fracture, glucocorticoid-induced osteoporosis, and bone loss caused by cancer therapies. Aukelso, a 120 mg/1.7 mL single-dose vial, has been approved for the prevention of skeletal-related events in patients with multiple myeloma or bone metastases, as well as for the treatment of giant cell tumour of bone and hypercalcemia of malignancy refractory to bisphosphonate therapy. Clinical studies confirmed comparable safety, efficacy, and quality with the reference drugs.

CDC won’t publish COVID vaccine report

According to the Washington Post, a report showing the efficacy of COVID-19 vaccines that was previously delayed by the head of the Centers for Disease Control and Prevention has been blocked from being published in the agency’s flagship scientific journal. The report shows that vaccines reduced emergency department visits and hospitalizations among healthy adults by about half this past winter. The move has raised concerns among current and former officials that information about the vaccine’s benefits is being downplayed because they conflict with the views of Health Secretary Robert F. Kennedy Jr., who has been an outspoken critic of COVID-19 vaccines. Along similar lines, an article in Forbes analyses some of the challenges that vaccine developers are facing in the US due to political interference.

FDA accepts application for Roche’s obinutuzumab for SLE

Roche has announced that the FDA has accepted the company’s sBLA for Gazyva/Gazyvaro (obinutuzumab) for the treatment of systemic lupus erythematosus (SLE). The filing acceptance is based on positive results from the Phase III ALLEGORY study. The primary endpoint was met, with 76.7% of the obinutuzumab group achieving SRI-4 response at 52 weeks versus 53.5% with placebo (adjusted difference 23.1%, p<0.001). All key secondary endpoints were also met, including BICLA response, sustained corticosteroid control, and time to first flare. Data was published in the NEJM in March 2026.  The FDA is expected to decide on an approval by December 2026. Gazyva/Gazyvaro is already approved for adults with lupus nephritis in the US and EU.

FDA approves Sanofi's Tzield to delay stage 3 T1DM in children

The FDA has approved the sBLA for Tzield (teplizumab), expanding the indication from eight years and older to as young as one year of age to delay the onset of stage 3 type 1 diabetes (T1D) in patients diagnosed with stage 2 T1D. The approval was granted under a priority review process and is supported by one-year data from the Phase IV PETITE-T1D study evaluating safety and PK in young children.

FDA extends review of Sanofi’s Sarclisa subQ

The FDA has extended by up to three months the target action date for its review of the BLA for Sarclisa subQ (isatuximab) in combination with approved standard-of-care regimens for the treatment of patients with multiple myeloma (MM) across all currently approved US indications of Sarclisa intravenous (IV) formulation. The revised target action date for the FDA decision is July 23, 2026.

Formation Bio raises $615 million to accelerate clinical trials

Formation Bio represents one of the more credible attempts to address the clinical development bottleneck rather than the earlier-stage discovery problem that attracts most AI hype. The commercial exits and the calibre of its partners give it more substance than most. Whether it can consistently turn AI-enabled efficiency into approved medicines, rather than well-priced licensing transactions, remains the primary question.

Inside the UK’s “secretive pharma” deal with the US

Keir Starmer’s trade deal with Trump for US pharmaceuticals may end up costing the UK £64 billion, according to The Bureau of Investigative Journalism, far higher than original estimates. The Bureau cites research done by Professor Karl Claxton (University of York), who previously sat on the appraisal committee for NICE. Claxton found that the UK government’s £1 billion estimate of the deal’s most immediate costs should be closer to £3.3 billion. He also argues that number will balloon to £64 billion when we take account of the government’s commitment to double its pharmaceutical spending levels by 2036.

Ionis presents new data for zilganersen in Alexander disease

After the FDA accepted zilganersen's NDA for Priority Review in March 2026, new pivotal study data was presented at AAN 2026. Zilganersen 50mg met its primary endpoint, significantly stabilizing gait speed (10MWT) in participants aged ≥5 years. GMFM-88 results suggest improved gross motor function in children aged 2-4 years. Secondary endpoints from patient, caregiver, and clinician assessments favoured zilganersen, which also showed good safety and tolerability.

J&J’s Imaavy delivers long-term disease control in gMG

Johnson & Johnson has shared new Phase III Vivacity-MG3 and ongoing OLE results showing Imaavy (nipocalimab) is effective and safe for antibody-positive adults with generalized myasthenia gravis. At 96 weeks, patients had notable reductions in symptom scores and muscle strength measures; half experienced minimal symptoms, and a third maintained that status for at least 8 weeks. IgG levels dropped over 64%, and most patients reduced corticosteroid use. Quality of life improved most in those who sustained minimal symptoms, with nipocalimab plus standard care yielding four times the sustained benefit versus placebo.

Janux doses first patient with JANX014 in patients with mCRPC

Janux Therapeutics has announced that the first patient has been dosed in a Phase I clinical trial of JANX014 in patients with metastatic castration-resistant prostate cancer (mCRPC). JANX014 is a double-masked, prostate-specific membrane antigen (PSMA) directed T cell engager (TCE) designed to leverage Janux’s tumour-activated technology platform to selectively activate T cells in the tumour microenvironment.

Kura reports data for its darlifarnib/cabozantinib combo in ccRCC

Kura Oncology has released early results from a subset of ccRCC patients previously treated with cabozantinib in the ongoing FIT-001 trial. The combination of darlifarnib (KO-2806) and cabozantinib showed strong antitumor effects and manageable safety, even at full dose levels. These results align with prior ESMO 2025 and earlier data, supporting darlifarnib's potential to improve VEGFR-targeted therapies and counter resistance.

Merck/Eisai’s Phase III LITESPARK-012 trial misses on PFS and OS in 1L RCC

Merck & Co. and Eisai have reported results from the Phase III LITESPARK-012 trial which evaluated combo regimens for 1L treatment of patients with advanced clear cell renal cell carcinoma (RCC). The study tested two combo regimens: pembrolizumab (anti-PD-1) plus lenvatinib (TKI) plus belzutifan (HIF-2α inhibitor), and MK-1308A (pembrolizumab and quavonlimab) plus lenvatinib, versus pembrolizumab plus lenvatinib. Neither combo met the primary endpoints of PFS and OS at interim analysis. Safety profiles matched those in earlier studies.

Novartis trims focus for anti-FXI mAb, abelacimab

Novartis has announced that it has discontinued development of its anti-FXI mAb, abelacimab, in cancer-associated thrombosis and prevention of venous thromboembolism specifically related to gastrointestinal or genitourinary cancer. Development in abelacimab’s lead indication (SPAF) continues. Recall, Novartis regained abelacimab by buying Anthos Therapeutics for $925 million upfront in February 2025.

Novo’s semaglutide “might” have muscle advantage over Lilly’s tirzepatide

A medRxiv preprint reports that Novo Nordisk’s semaglutide may offer better body composition effects than Eli Lilly’s tirzepatide. Researchers analysed data from nearly 8,000 patients starting GLP-1 treatment, categorizing them by weight loss patterns. In the first year, 10.3% of tirzepatide users and 6.7% of semaglutide users showed a depletive metabotype—a statistically significant difference in favour of semaglutide. For the prime metabotype, 12.3% of semaglutide users and 11.8% of tirzepatide users were identified, but this was not statistically significant. Both drugs were associated with greater lean body mass decline at higher doses and longer exposure. Note: Preprints on medRxiv have not been peer-reviewed and should not guide clinical decisions.

Sanofi’s Nuvaxovid beats Moderna’s mNexspike on reactogenicity

Sanofi’s protein-based, non-mRNA COVID-19 vaccine Nuvaxovid (COVID-19 vaccine, adjuvanted) has demonstrated statistically significant lower systemic reactogenicity to mNexspike (COVID-19 vaccine, mRNA), Moderna's latest mRNA COVID-19 vaccine, across all pre-specified endpoints in the COMPARE study. This is more of a patient experience thing, as opposed to an efficacy thing.

Taiho discontinues the Phase III STAR-121 trial in NSCLC due to futility

Based on a recommendation from the IDMC, Taiho Pharmaceutical (an Otsuka subsidiary) has discontinued the Phase III STAR-121 study due to futility. The study, which was being conducted in collaboration with Arcus and Gilead, was evaluating the combination of domvanalimab (anti-TIGIT mAb) and zimberelimab (anti-PD-1 mAb), plus chemotherapy, versus pembrolizumab plus chemotherapy as a 1L treatment for metastatic NSCLC.

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.