Pharma Phriday (May 1, 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

• Profluent and Lilly sign AI-driven genetic medicines R&D pact.

Clinical

• Alector/GSK halt Phase II nivisnebart early Alzheimer’s trial.

• Biomea Fusion reports positive Phase II data for icovamenib in T1DM.

• Boehringer Ingelheim’s survodutide delivers in Phase III obesity trial.

• First patient dosed with Merck Group’s enpatoran in Phase III CLE trial.

• Incyte reports positive Phase III data for povorcitinib in vitiligo.

• Intellia reports positive Phase III results for HAE gene editing trial.

• Veradermics reports positive results for VDPHL01 in male pattern hair loss.

• Zealand Pharma and Roche to advance petrelintide into Phase III trials.

Regulatory

• Arrowhead’s Redemplo receives CHMP nod for FCS.

• AstraZeneca’s Saphnelo subQ approved by the FDA.

• EC approves Poherdy as a biosimilar version of Perjeta.

• FDA accepts Jazz’s sBLA for Ziihera combos in 1L HER2+ GEA.

• FDA’s approval of Otarmeni is music to Regeneron’s ears.

• FDA pushes Amgen to withdraw Tavneos from the US.

• GSK’s efimosfermin wins FDA Breakthrough, EMA PRIME for MASH.

• Novartis’ Itvisma receives CHMP nod for SMA.

• Novartis’ Rhapsido gets EC approval for CSU.

• Sanofi’s Cenrifki receives CHMP nod for nrSPMS.

Commercial

• AbbVie swoops in with right-to-buy deal for Kestrel Therapeutics.

• Chiesi to acquire KalVista Pharmaceuticals for $1.9 billion.

• Eli Lilly to acquire Ajax Therapeutics for $2.3 billion.

• LEO Pharma to acquire Replay for $50 million up front.

• Ligand Pharmaceuticals buying XOMA Royalty for $739 million.

• Sun Pharma to acquire Organon for $11.75 billion.

• Teva to acquire Emalex Biosciences for $700 million.

In Other News

• AstraZeneca’s Breztri wins FDA nod for asthma in over-12s.

• BeOne Medicines licenses pre-clinical oncology asset from Huahui.

• EverythingALS launches SAVA to match patients to clinical trials.

• FDA approves label extensions for Alyftrek and Trikafta.

• FDA unveils real-time clinical trial review plan.

• GSK’s bepirovirsen gets brace of good news from the FDA.

• Is Foundayo suffering from a brand recognition problem?

• J&J’s Caplyta gets FDA nod for prevention of schizophrenia relapse.

• J&J’s Imaavy gets FDA priority review in wAIHA.

• MHRA announces significant clinical trials reform.

• Mirum’s brelovitug delivers Phase IIb win in chronic HepD.

• Novartis malaria treatment Coartem Baby receives WHO prequalification.

• Novartis pulls EMA bid for new Pluvicto indication.

• Novo Nordisk to release new obesity data at ESO.

• Pfizer’s Elrexfio improves PFS in R/RMM.

• ReSPECT for Mundipharma’s Rezzayo after Phase III win.

• Samsung Bioepis publishes its Q2 2026 biosimilars report.

• Structure Therapeutics announces multiple presentations at ADA 2026.

Summary

• Artificial intelligence. The AI-pharma deal machine continued this week, with Eli Lilly signing a multi-programme collaboration with Bezos-backed Profluent to develop AI-designed site-specific recombinases for genetic medicine. Profluent will design recombinases for multiple genomic targets, with Eli Lilly holding exclusive commercialisation rights. Total potential deal value reaches $2.25 billion in milestones plus royalties, extending Lilly's already active AI partnership portfolio.

• Clinical. A broad set of readouts this week. GSK and Alector discontinued the Phase II PROGRESS-AD trial of nivisnebart in early Alzheimer's disease following a futility analysis, effectively ending their neurology collaboration given the earlier failure of latozinemab in frontotemporal dementia. In metabolic disease, Boehringer Ingelheim reported positive Phase III data for survodutide, with 16.6% mean weight loss versus 3.2% on placebo over 76 weeks, broadly in line with semaglutide, though tolerability data are still awaited. Zealand Pharma and Roche confirmed petrelintide will advance into Phase III obesity trials despite a modest 10.7% Phase II weight loss signal. Biomea Fusion reported durable 52-week C-peptide preservation in a small Phase II cohort of type 1 diabetes patients treated with icovamenib, pointing to meaningful beta cell restoration. The most significant clinical readout was Intellia's positive Phase III HAELO trial of lonvo-z in hereditary angioedema. A single-dose in vivo CRISPR gene editing treatment, lonvo-z delivered a 62% attack-free and therapy-free rate versus 11% on placebo, with a rolling BLA submission initiated and a potential US launch targeted for the first half of 2027. Incyte also announced positive Phase III data for povorcitinib in vitiligo, though response rates fall below those seen with ruxolitinib cream and upadacitinib.

• Regulatory. A busy week, with the FDA approving AstraZeneca's Saphnelo subQ formulation for SLE, opening competition with GSK's Benlysta subQ, and granting accelerated approval to Regeneron's Otarmeni for OTOF-related genetic hearing loss, the first gene therapy approved for a genetic form of deafness, available free of charge in the US. In a markedly different direction, the FDA proposed withdrawal of Amgen's Tavneos (avacopan), alleging manipulation of the pivotal ADVOCATE trial data, one of the most serious regulatory actions in recent memory. In Europe, EMA’s CHMP adopted positive opinions for plozasiran in familial chylomicronaemia syndrome, remibrutinib in chronic spontaneous urticaria, tolebrutinib in non-relapsing secondary progressive MS, and intrathecal onasemnogene abeparvovec for SMA in patients aged two and older. The EC also approved the first Perjeta biosimilar, Poherdy.

• Commercial. Four acquisitions dominated the week. Chiesi agreed to acquire KalVista Pharmaceuticals for $1.9 billion, gaining the oral HAE acute treatment Ekterly and an early-stage Factor XIIa inhibitor. Eli Lilly agreed to acquire Ajax Therapeutics for $2.3 billion, adding a Type II JAK2 inhibitor to its portfolio. Sun Pharma announced a transformative $11.75 billion acquisition of Organon, one of the largest ever deals by an Indian pharma company. Teva agreed to acquire Emalex Biosciences for $700 million, gaining ecopipam, a first-in-class D1 receptor antagonist for Tourette syndrome with NDA submission anticipated in the second half of 2026.

• In Other News. AstraZeneca's Breztri received FDA approval for asthma in patients aged 12 and older; Vertex gained label extensions for Alyftrek and Trikafta covering additional CFTR variants, meaning approximately 95% of US cystic fibrosis patients are now eligible for a CFTR modulator; the MHRA introduced significant clinical trials reforms in the UK, effective 28 April; and analysts flagged that Foundayo (orforglipron) is trailing oral Wegovy's launch trajectory, potentially due to lower brand recognition.

Artificial Intelligence

Profluent and Lilly sign AI-driven genetic medicines R&D pact

What happened? Bezos-backed Profluent, an AI company focused on protein design, has announced a multi-programme strategic research collaboration with Eli Lilly (Lilly) to develop and commercialise custom site-specific recombinases to address diseases with severe unmet needs. The collaboration focuses on enabling large-scale, precise DNA editing capabilities that remain out of reach using conventional gene editing systems. By combining Profluent’s AI models with Lilly’s clinical and developmental capabilities in genetic medicines, the companies aim to expand the scope and scalability of programmable gene editing therapeutics. Under the agreement, Profluent will apply its AI models to design and optimise site-specific recombinases for multiple genomic targets. Lilly will receive an exclusive license to advance selected recombinases through in vivo research, preclinical development, clinical studies, and commercialisation. Profluent will receive an upfront payment in addition to committed R&D funding. In total, Profluent is eligible to receive up to $2.25 billion in development and commercial milestone payments plus tiered royalties on net sales. 

What does this mean? It’s rare that a week goes by without a pharma/AI deal being announced. Since the beginning of 2025, I’ve personally tracked over 70 such deals with a total disclosed and potential deal value of $87 billion. Hat tip to Tudor Oprea for helping me out with some of the details for this tracker. For Eli Lilly this deal follows similar collaborations with Insilico Medicine, NVIDIA, Nimbus Therapeutics and BigHat Biosciences. More details to follow when they become available.

Clinical

Alector/GSK halt Phase II nivisnebart early Alzheimer’s trial

What happened? Alector has confirmed that the Phase II PROGRESS-AD trial of nivisnebart (formerly AL101) in individuals with early Alzheimer’s disease (AD) will be discontinued. The decision follows a pre-specified futility analysis conducted by an IDMC, which concluded that the trial was unlikely to meet its primary endpoint of slowing disease progression at completion. Alector and GSK have been co-developing nivisnebart since entering a neurology-focused R&D collaboration in July 2021.

What does this mean? In addition to nivisnebart, GSK’s R&D partnership with Alector included AL001 (latozinemab), a mAb designed to increase progranulin levels by inhibiting the sortilin receptor. Recall, a Phase III trial assessing the efficacy of latozinemab in patients with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN) failed to meet its co-primary endpoint in October 2025 resulting in latozinemab’s development being terminated. With both programmes now terminated, this effectively ends GSK’s agreement with Alector.

Biomea Fusion reports positive Phase II data for icovamenib in T1DM

What happened? Biomea Fusion (Biomea) has announced positive 52-week results from its Phase II COVALENT-112 trial evaluating the efficacy, safety, and tolerability of icovamenib in patients with type 1 diabetes (T1D). In patients diagnosed within 0-3 years (n=5), treatment with icovamenib 200mg once daily for 12 weeks resulted in a 52% increase in mean C-peptide area under the curve (AUC) at Week 12 (p < 0.001), representing a magnitude of improvement that is not commonly reported in published studies of T1D. Importantly, the effect was durable following only 12 weeks of dosing, mean C-peptide AUC was largely preserved through Week 52, representing approximately a 7% decline from baseline. A dose response was observed, with the 200mg dose demonstrating greater activity compared to 100mg. Published natural history data suggest that patients with Stage 3 T1D typically experience substantial declines in C-peptide over time, underscoring the significance of preserved C-peptide following only a 12-week dosing period.

What does this mean? As a bit of background, C-peptide is a by-product of insulin production. When the pancreas makes insulin, it cleaves a precursor molecule (proinsulin) into insulin plus C-peptide in equal amounts. Since exogenous (injected) insulin contains no C-peptide, measuring it gives a clean picture of how much insulin the patient's own beta cells are still making. While the dataset is small, the combination of magnitude and durability points to menin inhibition driving restoration of beta cell function.

Boehringer Ingelheim’s survodutide delivers in Phase III obesity trial

What happened? Boehringer Ingelheim (Boehringer) has announced positive topline results from the Phase III SYNCHRONIZE-1 trial, in which survodutide (glucagon/GLP-1 dual agonist) met the co-primary endpoints using both the efficacy and treatment-regimen estimands. Adults living with obesity or who were overweight, without type 2 diabetes, and who were treated with survodutide experienced sustained weight loss of up to an average of 16.6% (39.2 lb or 17.8 kg) after 76 weeks using the efficacy estimand, a statistically significant decrease versus 3.2% in the placebo arm. This level of weight loss supports survodutide’s potential as a clinically meaningful treatment option for people living with obesity or overweight. The trial also met key secondary endpoints including a significant reduction in waist circumference, a clinical marker closely linked to visceral fat and cardiometabolic risk. Boehringer licensed survodutide (formerly ZP2929) from Zealand Pharma in June 2011.

What does this mean? The statistically significant weight loss of 16.6% vs. 3.2% placebo over 76 weeks is broadly similar to Wegovy (semaglutide) at 16.9% vs. 2.4% after 68 weeks. Analysts from Jefferies believe that weight loss was predominantly driven by fat loss, though details are still needed to determine whether the amount constitutes a potential differentiation vs. other treatment options. More details are needed on tolerability, which are likely to be provided at ADA 2026.

First patient dosed with Merck Group’s enpatoran in Phase III CLE trial

What happened? Merck Group has announced the first patient has been dosed in the Phase III programme (ELOWEN-1 and ELOWEN-2) which is evaluating enpatoran in people living with lupus who experience active skin manifestations (i.e., discoid lupus erythematosus and/or subacute cutaneous lupus erythematosus). The ELOWEN studies will be conducted in 266 sites in 26 countries. Each study will recruit approximately 200 lupus participants, and the primary endpoint will be a change in CLASI-A from baseline. Enpatoran is an oral selective toll-like receptor (TLR) 7/8 inhibitor designed to modulate pathways central to lupus-related inflammation. 

What does this mean? Development of enpatoran continues in CLE despite a Phase II miss in systemic lupus erythematosus (SLE). In the Phase II WILLOW trial, data showed no statistically significant benefit of enpatoran treatment in patients with SLE. However, benefit was seen in patients with CLE or SLE with active lupus rash. There are limited targeted treatment options for CLE, with treatments like anifrolumab (Saphnelo) and belimumab (Benlysta) sometimes used off label. In addition to enpatoran, other pipeline programmes that are targeting CLE include anifrolumab (anti-IFNAR1 mAb; AstraZeneca), litifilimab (anti-BDCA2 mAb; Biogen), deucravacitinib (TYK2 inhibitor; BMS) and afimetoran (Beeline Medicines).

Incyte reports positive Phase III data for povorcitinib in vitiligo

What happened? Buried in its Q1’26 earnings statement, Incyte announced positive data from the Phase III programme evaluating povorcitinib (30mg) in adult patients with nonsegmental vitiligo. In both Phase III studies (STOP-V1 and STOP-V2), povorcitinib achieved the primary endpoint of >75% reduction in Facial Vitiligo Area Scoring Index (F-VASI75) from baseline at Week 52. In STOP-V1, 18.9% of povorcitinib-treated patients achieved a >75% reduction in F-VASI75 compared to 6.8% of placebo-treated patients at Week 52 (p<0.001). In STOP-V2, 18.9% of povorcitinib-treated patients achieved a >75% reduction in F-VASI75 compared to 3.1% of placebo-treated patients at Week 52. Across both studies, statistically significant and clinically meaningful differences were also observed in key secondary endpoint measures, including T-VASI50 at Week 52. The overall safety and tolerability profile of povorcitinib through 52 weeks is consistent with prior studies.

What does this mean? A decent result, and one that’s very likely to satisfy the regulators considering the dearth of treatments for vitiligo. However, the efficacy of Incyte’s Opzelura (ruxolitinib), approved as a treatment for vitiligo by the FDA in July 2022, is slightly better. In the TRuE-V Phase III programme, 29.8% of patients in TRuE-V1 and 30.9% in TRuE-V2 achieved F-VASI75 at Week 24 vs. 7.4% and 11.4% on vehicle respectively. AbbVie is also developing upadacitinib in vitiligo. Data from two Phase III trials (released in October 2025) showed that upadacitinib achieved the co-primary endpoints of 50% reduction in Total Vitiligo Area Scoring Index (T-VASI 50) from baseline and 75% reduction in Facial Vitiligo Area Scoring Index (F-VASI 75) from baseline at week 48. Interestingly, patients recruited to the upadacitinib trials were more severe vs. other trials, as approximately 70% of subjects had T-VASI >10 at baseline. Aside from vitiligo, Incyte is also developing povorcitinib in three other indications. Data from two Phase III trials in hidradenitis suppurativa (HS) were announced in March 2026, and data from Phase III trials in prurigo nodularis (PN) and a Phase II trial in asthma are expected later in 2026. AbbVie has also reported positive Phase III data for upadacitinib in vitiligo.

Intellia reports positive Phase III results for HAE gene editing trial

What happened? Intellia Therapeutics (Intellia) has announced positive topline results from the Phase III HAELO clinical trial of lonvoguran ziclumeran (lonvo-z; formerly known as NTLA-2002) in hereditary angioedema (HAE). Designed as a one-time treatment that’s administered in an outpatient setting, lonvo-z is an in vivo CRISPR gene editing candidate that is intended to inactivate the kallikrein B1 (KLKB1) gene to permanently lower kallikrein and bradykinin levels. Intellia separately announced today that it has initiated a rolling BLA submission to the FDA to seek regulatory approval. The company is preparing for a potential US launch of lonvo-z in the first half of 2027. The HAELO trial recruited n=80 patients, with n=52 receiving lonvo-z and n=28 receiving placebo. Of the total population, 49% of patients were enrolled in the US and 71% were on long-term prophylaxis (LTP) therapy at study entry (e.g., lanadelumab, berotralstat). Patients on LTP were required to discontinue those therapies in the weeks prior to dosing. The HAELO trial met its primary endpoint (reduction in the number of HAE attacks) and all key secondary endpoints with statistical significance (p<0.0001). These included a 62% rate of patients who were entirely attack free and therapy free in the lonvo-z arm for the six-month efficacy evaluation period, compared with 11% of patients in the placebo arm. The most common treatment emergent adverse events (TEAEs) during the primary observation period (infusion through week 28) were infusion-related reactions, headache and fatigue.

What does this mean? Current treatment approaches for HAE are divided into three categories: managing acute attacks, short-term prophylaxis and long-term prophylaxis. Lonvo-z will sit in the final category, so will compete with current long-term prophylactic treatments including lanadelumab and berotralstat. The limitation of all current long-term prophylaxis approaches is that they require ongoing, indefinite treatment. They manage the disease rather than addressing its underlying cause. Lonvo-z is a one-time treatment with lifelong effect. Question marks remain about liver safety of Intellia’s CRISPR programmes, and regulators will scrutinise hepatic safety carefully across all programmes. The clinical hold in the ATTR programme creates a difficult backdrop for the HAE BLA review.

Veradermics reports positive results for VDPHL01 in male pattern hair loss

What happened? Veradermics has announced positive topline results from Part A of the Phase II/III Study 302 which is evaluating VDPHL01, an ER oral minoxidil formulation, in males with mild-to-moderate pattern hair loss. Study 302 enrolled 519 patients who were randomised to receive either VDPHL01 8.5mg QD, VDPHL01 8.5mg BID, or placebo. The trial met all primary and all key secondary endpoints with statistical significance while being generally well tolerated with no treatment-related serious adverse events (SAEs) and no adverse events of special interest (AESIs) of cardiac origin. Following six months of treatment with VDPHL01, 79.3% of patients in the once daily dose arm and 86% of patients in the twice daily dose arm reported improvement in hair coverage on the Androgenetic Alopecia Impact Rating Scale (AAIRS) versus 35.6% of placebo patients. Additionally, 48.4% of patients in the once daily dose arm and 62.9% of patients in the twice daily dose arm achieved improved or much improved hair coverage on the AAIRS compared to only 13.4% of placebo patients.

What does this mean? Current treatments for androgenetic alopecia (pattern hair loss) focus on slowing hair loss and promoting regrowth. Options include topical minoxidil foam/liquid (either as Rogaine or generics) and oral finasteride (Propecia). In April 2026, Cosmo Pharmaceuticals announced positive 12-month Phase III results for clascoterone 5% topical solution in men with mild-to-moderate androgenetic alopecia (AGA). Taking both data readouts together, the next 6-12 months are likely to see two new treatments approved for AGA. Other R&D focus areas include non-hormonal approaches (e.g., PP405 from Pelage Pharmaceuticals), thyromimetics (e.g., TDM-105795 from TechnoDerma Medicines), and multiple new pathways (e.g., Wnt signalling agonists, such as dalosirvat from Biosplice Therapeutics) and cGMP-enzyme regulation (Topadur Pharma's TOP-M119).

Zealand Pharma and Roche to advance petrelintide into Phase III trials

What happened? Zealand Pharma (Zealand) and Roche have announced that petrelintide, an amylin analogue being investigated as a potential treatment for obesity, will advance into Phase III trials. The initiation is planned for H2’26. The Phase III programme for petrelintide is designed to evaluate the efficacy, safety, and tolerability of petrelintide as a Q1W subQ treatment in adults living with obesity or who are overweight with weight-related comorbidities.

What does this mean? Following Roche and Zealand announcing topline results from the Phase II ZUPREME-1 trial evaluating petrelintide vs. placebo in people living with obesity, reaction was muted. Petrelintide delivered a 10.7% mean weight loss from baseline using the efficacy estimand, compared to 1.7% with placebo, at week 42. Recall, Wegovy (semaglutide) delivered 16.9% vs. 2.4% weight loss after 68 weeks. While below expectations, this was achieved with clean tolerability (notably no vomiting), which is encouraging. The design of the Phase III trials remains to be seen, but perhaps an improved patient experience with non-inferior efficacy will be Zealand and Roche’s way to building the belief that amylin can take share in the obesity market that will eventually include biosimilar/generic semaglutide.

Regulatory

Arrowhead’s Redemplo receives CHMP nod for FCS

What happened? Arrowhead Pharmaceuticals (Arrowhead) has announced that the EMA’s CHMP has recommended the approval of Redemplo (plozasiran), a siRNA medicine, as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronaemia syndrome (FCS). FCS is a rare disease that’s characterised by triglyceride levels that can be orders of magnitude higher than normal, leading to a substantially higher risk of developing acute, recurrent, and potentially fatal pancreatitis. EC approval is expected within 60 days. Aligned with Redemplo’s FDA approval in November 2025, the CHMP’s recommendation was based on data from the Phase III PALISADE study. The PALISADE study met its primary endpoint and all multiplicity-controlled key secondary endpoints, including demonstrating significant reductions in triglycerides and apoC-III and in the incidence of acute pancreatitis in the pooled dose groups. In PALISADE, 25mg plozasiran reduced triglycerides by a median of 80% from baseline versus a 17% reduction with placebo and significantly fewer cases of acute pancreatitis were seen in patients using plozasiran compared with those using placebo. Plozasiran is self-administered via subQ injection Q3M and works by targeting mRNA in the liver to degrade apoC-III, reducing hepatic and serum ApoC3 levels and increasing the clearance of serum triglycerides.

What does this mean? The core strategy for managing FCS is via dietary modification (e.g., dietary fat at 10-15% of daily calories, use of medium-chain triglycerides), with the core aim being the prevention of pancreatitis. Adjunctive treatments include triglyceride lowering therapy (e.g., statins, fibrates, niacin, omega-3 fatty acids), which are often ineffective, and apoC-III targeting siRNA therapies. Subject to EC approval, Redemplo will become the third siRNA treatment option for FCS in Europe following EC approvals for Waylivra (volanesorsen; Akcea/Ionis) and Tryngolza (olezarsen; Sobi/Ionis) in May 2019 and September 2025, respectively. While all three treatments work in the same way, Redemplo has a potential advantage in two critical areas. The first is on dosing frequency. For the first three months, Waylivra is dosed once weekly for 3 months, then every 2 weeks. Tryngolza is dosed once a month. Redemplo will be dosed every three months (Q3M). This could improve patient adherence. Secondly, patients are only eligible for Waylivra and Tryngolza if genetic testing confirms FCS. Redemplo does not require genetic confirmation of FCS, thus provides a treatment option for more adults with FCS. A full SMPC for Redemplo will be needed before any comparison vs. Tryngolza can be made in other areas (e.g., contraindications). Note, Tryngolza’s FDA label includes hypersensitivity as a warning/precaution.

AstraZeneca’s Saphnelo subQ approved by the FDA

What happened? AstraZeneca’s Saphnelo subQ (anifrolumab) has been approved by the FDA for self-administration as a Q1W autoinjector, the Saphnelo Pen, for the treatment of adult patients with SLE on top of standard therapy. The approval was based on results from the Phase III TULIP-SC trial, which showed that subQ administration of Saphnelo led to a statistically significant and clinically meaningful reduction in disease activity compared to placebo in participants with moderate to severe SLE while receiving standard therapy. The safety profile observed was consistent with the known clinical profile of Saphnelo administered as an intravenous (IV) infusion.

What does it mean? Saphnelo subQ’s regulatory progress at the FDA has been a bit bumpy, with progress in Europe much smoother (where it was approved in December 2025). In February 2026 the FDA issued a complete response letter (CRL) regarding the BLA for Saphnelo subQ, the timing of which was a little odd as AstraZeneca had already provided the information requested in the CRL. Analysts at the time suggested this was part of the FDA’s commitment to transparency where CRL’s would be released to enhance trust in agency decisions. With the US approval for Saphnelo subQ now in hand, AstraZeneca has an opportunity to take market share away from GSK’s Benlysta subQ (belimumab), the only other self-administered biologic therapy for SLE, which was approved in July 2017. How quickly could patients switch to Saphnelo subQ? Data from two case studies could inform estimates. Following its FDA approval in January 2020, the use of Darzalex subQ in the treatment of MM rose from 38% to 91% between June 2020 and June 2021. In contrast, an Italian real-world study of 238 IBD patients in stable remission on IV vedolizumab who were offered the chance to switch to subQ, only 3.7% accepted. The key variable here is that patients who are doing well on IV and have an established routine with their infusion clinic often resist switching (i.e., if it ain't broke, don’t try to fix it). The daratumumab-style rapid conversion is probably more likely in a disease like SLE than in stable IBD. The patient population is younger, predominantly female and for whom clinic visits are a real burden.

EC approves Poherdy as a biosimilar version of Perjeta

What happened? Shanghai Henlius Biotech and Organon have announced the EC has approved Poherdy (pertuzumab; 420 mg/14 mL injection for IV use), the first approved biosimilar to Perjeta (pertuzumab; Roche) in Europe, for all indications of the reference product. Poherdy was approved based on the review of a comprehensive data package, which included structural and functional analytical data, clinical PK data, and comparative clinical studies demonstrating that Poherdy is a biological medicine highly similar to the reference product based on a totality of evidence, including analytical, PK, efficacy, safety, and immunogenicity data.

What does this mean? There is growing interest in developing biosimilar versions of Perjeta (pertuzumab). With significant competition already in place for Herceptin biosimilars, Perjeta becomes the obvious next target for companies interested in gaining a foothold in the anti-HER2 breast cancer treatment market. Other programmes include EirGenix/Sandoz’s EG1206A, Henlius Biotech (HLX11), Zydus Lifesciences/Dr Reddy’s (Sigrima/Womab), Alkem/Enzene (Pertuza) and NeuClone Pharmaceuticals. EC approval of Poherdy comes after its US approval in November 2025, when the FDA approved Poherdy as the first interchangeable biosimilar of Perjeta (pertuzumab).

FDA accepts Jazz’s sBLA for Ziihera combos in 1L HER2+ GEA

What happened? Jazz Pharmaceuticals (Jazz) has announced that the FDA has accepted for filing with Priority Review the sBLA for Ziihera (zanidatamab) containing combo for the 1L treatment of adult patients with HER2+ unresectable locally advanced or metastatic gastric, gastroesophageal junction (GEJ), or gastroesophageal adenocarcinoma (GEA). The PDUFA date is August 25, 2026. The sBLA is supported by data from the Phase III HERIZON-GEA-01 trial to investigate the efficacy and safety of zanidatamab in combination with standard-of-care chemotherapy with or without the PD-1 inhibitor tislelizumab (Tevimbra; BeOne) in patients with advanced or metastatic GEA, including gastric, GEJ and oesophageal adenocarcinomas. The submission is under review via the Real-Time Oncology Review (RTOR) programme. Zanidatamab is being developed by Jazz Pharmaceuticals and BeOne under license agreements from Zymeworks, which first developed the molecule. 

What does this mean? An important milestone in the commercial repositioning of Ziihera (zanidatamab). Ziihera is currently approved in the US for previously treated, unresectable or metastatic HER2+ biliary tract cancer based on accelerated approval. 1L gastric, gastroesophageal junction, and oesophageal adenocarcinoma (collectively GEA) represents a substantially larger patient population, and approval here would establish Ziihera as a mainstream treatment rather than a niche one. Trastuzumab (Herceptin, plus various biosimilars) is the go-to targeted treatment in HER2+gastric cancer, with Keytruda (pembrolizumab) now added in PD-L1+ patients. The question is, can zanidatamab displace or complement these approaches? Zanidatamab’s broader effector MOA could help in this regard. Moreover, the competitive field is not standing still. Enhertu plus chemotherapy with or without Keytruda is being assessed in the 1L setting vs. vs. trastuzumab plus chemotherapy with or without pembrolizumab (DESTINY-Gastric05 trial).

FDA’s approval of Otarmeni is music to Regeneron’s ears

What happened? Regeneron has announced the FDA has granted accelerated approval for Otarmeni (lunsotogene parvec), the first gene therapy and second new molecular entity approved under the FDA Commissioner’s National Priority Voucher programme. Otarmeni is an AAV vector-based gene therapy indicated for the treatment of paediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. Otarmeni, formerly known as DB-OTO, is the first and only in vivo gene therapy for OTOF-related hearing loss and will be made available by Regeneron for free in the US. Otarmeni was granted accelerated approval based on results from the pivotal Phase I/II CHORD trial, in which n=20 participants (aged 10 months to 16 years) received a single dose of Otarmeni via intracochlear infusion, either unilaterally (in one ear; n=10) or bilaterally (in both ears; n=10). 16/20 patients (80%) experienced hearing improvements per pure tone audiometry assessments at a threshold of ≤70 dB HL at 24 weeks and 14/20 patients (70%) demonstrated an auditory brainstem response.

What does this mean? As someone who suffers from hearing loss, this is massive news. Until recently, the only treatment for genetic deafness was cochlear implantation, which is effective but doesn't restore natural hearing. Originally developed by Decibel Therapeutics (acquired by Regeneron in September 2023), Otarmeni is the first gene therapy ever approved for a genetic form of hearing loss. As for Regeneron’s commitment to making Otarmeni free in the US, this is likely to be aligned with Regeneron’s MFN deal with the US government where Regeneron will reduce the price of Praluent and all new Regeneron medicines moving forward will receive MFN prices for US patients. Another consideration is the size of the market. The condition Otarmeni treats is ultra-rare (~50 babies per year). Other companies running clinical trials targeting the same OTOF gene as Otarmeni include Eli Lilly (via AK-OTOF which it gained from its October 2022 acquisition of Akouos) and Sensorion (via SENS-501 which is in Phase I/II trials). Eli Lilly has also partnered with Seamless Therapeutics to develop and commercialise treatments for hearing loss based on gene editing techniques.

FDA pushes Amgen to withdraw Tavneos from the US

What happened? The FDA has proposed that Amgen withdraw Tavneos (avacopan) from the US market. Used in combination with glucocorticoids and other standard-of-care medications, Tavneos is indicated for the treatment of adults with severe ANCA-associated vasculitis, a group of rare diseases that cause inflammation in small-to-medium-sized blood vessels. Amgen gained Tavneos as part of its August 2022 acquisition of ChemoCentryx. These allegations surround ChemoCentryx’s development of the drug before it was approved in October 2021. The FDA has claimed that new information indicates that Tavneos has not been shown to be effective for its approved use, and because the application that resulted in FDA approval contained untrue statements of material fact. The FDA also alleges that “unblinded study personnel manipulated [Tavneos’] results” to make the drug look effective when the original analysis “did not support that conclusion.”

What does this mean? The FDA's proposal to withdraw approval of Tavneos is one of the most serious regulatory actions in recent memory, and its implications extend well beyond a single drug. The FDA's proposal alleges that unblinded study personnel manipulated results from the Phase III ADVOCATE trial to make avacopan appear effective when the original analysis did not support that conclusion, and that the original analysis was withheld from the FDA in violation of regulations. To be clear, this an allegation of clinical trial fraud, not merely insufficient evidence. Regardless of whether Tavneos is withdrawn from the market or not, physicians and patients will face an uncomfortable choice given the efficacy question has now been formally raised as it would leave a gap in the glucocorticoid-sparing ambition for ANCA patients rather than in the core treatment regimen (i.e., rituximab, cyclophosphamide and glucocorticoids).

GSK’s efimosfermin wins FDA Breakthrough, EMA PRIME for MASH

What happened? GSK has announced that efimosfermin, a once-monthly investigational fibroblast growth factor 21 (FGF21) analogue, has been granted Breakthrough Therapy Designation by the FDA and Priority Medicines (PRIME) Designation by the EMA for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The two designations were supported by data from MASH patients with moderate to advanced (F2/F3) and cirrhotic (F4) fibrosis. This includes Phase II data at 48 weeks for F2/F3 patients who showed fibrosis improvement and MASH resolution with once monthly efimosfermin versus placebo. Data also confirmed a well-tolerated safety profile with mild, transient adverse events, including nausea, vomiting, and diarrhoea. Efimosfermin is currently in Phase III trials (ZENITH-1 and ZENITH-2) which are investigating efficacy and safety in MASH patients with F2/F3 fibrosis. Phase III trials in MASH patients with F4 fibrosis are expected to start sometime in 2026. Recall, GSK acquired efimosfermin (formerly BOS-580) from Boston Pharmaceuticals in July 2025.

What does this mean? The MASH space has been moving quickly since resmetirom (Rezdiffra; Madrigal Pharmaceuticals) became the first FDA approved MASH treatment in March 2024. For GSK, the simultaneous FDA Breakthrough Therapy and EMA PRIME designations for efimosfermin represent a significant step in the company's ambition to establish a hepatology franchise. Several other companies are also pursuing MASH indications (either commercially or clinically), meaning GSK is entering a field that is becoming increasingly competitive. Novo Nordisk has positioned itself as a potential leader in MASH. In addition to Wegovy (semaglutide) gaining FDA approval in August 2025, Novo Nordisk acquired Akero Therapeutics in October 2025 to add its FGF21 analogue efruxifermin to its pipeline. Efruxifermin is currently in Phase III development for the treatment of patients with moderate to advanced liver fibrosis (F2-F3) and patients with cirrhosis (F4). Roche has also invested in MASH via its September 2025 acquisition of 89Bio which added pegozafermin, another FGF21 analogue currently in late-stage development for MASH in moderate and severe fibrotic patients (F2 and F3 stages) as well as cirrhotic patients (F4 stage).

Novartis’ Itvisma receives CHMP nod for SMA

What happened? Novartis has announced that the EMA’s CHMP has adopted a positive opinion recommending marketing authorization for Itvisma (intrathecal onasemnogene abeparvovec). The opinion supports its use for the treatment of children two years and older, teens, and adults living with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene. The CHMP opinion is based on data from the Phase III registrational STEER study, and supportive Phase IIIb STRENGTH and Phase I/II STRONG studies. In STEER, Itvisma demonstrated a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale (HFMSE) with effects sustained over 52 weeks of follow-up. The STEER and STRENGTH studies also showed clinically meaningful benefit for treatment-naïve and pre-treated patients. Recall, Itvisma was approved by the FDA in November 2025.

What does this mean? Itvisma has become the first and only gene therapy for spinal muscular atrophy (SMA) that can be given not just to infants, but also to children aged two years and older, teenagers, and adults. This dramatically expands access to a transformative treatment (i.e., Zolgensma) that was previously limited to very young patients (children under two years old). Older patients relied on chronic treatments like Spinraza (nusinersen) or Evrysdi (risdiplam), which require lifelong administration. Both Itvisma and Zolgensma work by delivering a functional copy of the human SMN gene via adeno-associated virus (AAV) vectors. While their active drug substances are identical, Itvisma is given directly to the central nervous system via an intrathecal injection in the spinal cord area, whereas Zolgensma is administered intravenously. Thanks to this new formulation, Itvisma can be given to patients with larger weight in a more concentrated dose.

Novartis’ Rhapsido gets EC approval for CSU

What happened? Novartis has announced today that the EC has approved Rhapsido (remibrutinib) for chronic spontaneous urticaria (CSU) in adult patients with inadequate response to H1-antihistamine treatment. Rhapsido will become the first oral targeted treatment approved for CSU that does not require lab monitoring. Approval was based on data from the pivotal Phase III REMIX-1 and REMIX-2 trials. Remibrutinib showed improvements in itch and hives as early as Week 1, with benefits sustained through Week 52. Improvements in quality of life and sleep were also observed early in treatment. Remibrutinib was well tolerated and demonstrated a favorable safety profile, including no liver safety concerns across both studies through Week 52.

What does this mean? Never in doubt following the EMA CHMP’s positive endorsement in February 2026. Rhapsido’s oral dosing gives Novartis a potential angle for differentiation vs. other targeted treatments, including and Xolair (omalizumab; Roche/Novartis) and more recently, Dupixent (dupilumab; Sanofi/Regeneron). Dupixent received EC approval for the treatment of adult CSU in November 2025, although Dupixent’s April 2026 approval in young children aged 2-11 years enables Dupixent to be used in a wider range of patients vs. both Xolair and Dupixent. Remibrutinib is also in development for MS and CIndU. Data for remibrutinib in CIndU was announced in February 2026, and a Phase III trial in SPMS is underway.

Sanofi’s Cenrifki receives CHMP nod for nrSPMS

What happened? Sanofi has announced that the EMA’s CHMP has adopted a positive opinion recommending the approval of Cenrifki (tolebrutinib) in the EU for the treatment of secondary progressive multiple sclerosis without relapses (nrSPMS) in the last two years. Formal EC approval is expected with 60 days. The positive CHMP opinion is based on data from the Phase III HERCULES study in non-relapsing SPMS, with supporting data from the Phase III GEMINI 1 and GEMINI 2 studies in relapsing multiple sclerosis (RMS). Data from the HERCULES study was published in the NEJM in April 2025. Recall, Sanofi partnered with Principia Biopharma in November 2017 to gain exclusive, worldwide rights to develop and commercialize PRN2246 (formerly called SAR442168, now called tolebrutinib) in multiple sclerosis and other central nervous system diseases. Sanofi then acquired Principia Biopharma in August 2020 gaining full control of the SAR442168 programme, along with other pipeline assets.

What does this mean? The regulatory fortunes for tolebrutinib in Europe and the US as a potential treatment for nrSPMS could not be more divergent: provisionally approved in the UAE in July 2025, rejected in the US in December 2025, and recommended for approval in Europe in April 2026. And as far as I can tell, all three agencies looked at the same data set (i.e., HERCULES plus GEMINI 1 and GEMINI 2). The FDA's objections centred on unresolved questions about which patients benefit, questions the EMA apparently weighed differently, perhaps accepting the overall HERCULES data at face value given a dearth of alternative treatments in this population. We await a full SMPC for Cenrifki to understand exactly what data was considered.

Commercial

AbbVie swoops in with right-to-buy deal for Kestrel Therapeutics

What happened? Alongside its announcement that it had dosed the first patient in its Phase I trial evaluating KST-6051, its investigational, oral, small-molecule pan-KRAS inhibitor, in patients with advanced or metastatic solid tumours with KRAS mutations, Kestrel Therapeutics (Kestrel) also announced that it has entered into a warrant agreement with AbbVie which gives AbbVie an exclusive option to acquire Kestrel based on defined development milestones. Under the terms of the agreement AbbVie will support funding of the KST-6051 programme and based on the completion of development and regulatory milestones will have the exclusive option to acquire Kestrel. Including the upfront payment, future exercise payments and downstream development and regulatory milestones, the value of the deal could reach up to $1.45 billion.

What does this mean? KRAS inhibition has been a tough nut for pharma to crack due to the oncogene’s smooth surface and very high affinity for its natural substrate. Some called KRAS mutations, which are very common in several cancers (e.g., NSCLC, PDAC, CRC) undruggable. But in 2013 scientists discovered a binding pocket on the surface of mutant KRAS called switch pocket II which enabled the development of compounds that selectively bind to the chemically reactive cysteine residue of the KRAS G12C mutant. Fast forward to June 2021 and the first KRAS inhibitor, sotorasib (Lumakras/Lumykras; Amgen), was approved by the FDA. Others followed, including adagrasib (Krazati; Mirati/BMS) and avutometinib/defactinib (Avmapki Fakzynja Co-Pack; Verastem). Pharma continues to search for next-generation KRAS inhibitors. As of September 2025, there were over 50 programmes in development. This search is focused on programmes targeting G12D, G12V, and broader pan-KRAS approaches. Kestrel’s KST-6051 is one such pan-KRAS programme. As is Revolution Medicine’s daraxonrasib, a pan-RAS inhibitor targeting the active "RAS-on" state rather than locking the protein in its inactive form. In April 2026, Revolution announced positive Phase III data for daraxonrasib in PDAC where daraxonrasib demonstrated a median OS of 13.2 months versus 6.7 months for chemotherapy.

Chiesi to acquire KalVista Pharmaceuticals for $1.9 billion

What happened? Chiesi and KalVista Pharmaceuticals (KalVista) have announced that the companies have entered into a definitive agreement under which Chiesi will acquire KalVista for $1.9 billion. Upon completion of the acquisition, Chiesi will assume responsibility for Ekterly (sebetralstat), an orally active plasma kallikrein inhibitor which is used as on demand treatment for hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older. The therapy is approved in the US, UK, EU, Japan and other regions, with ongoing studies exploring its use for treating HAE attacks in children aged 2-11 and multiple regulatory applications under review in key global markets.

What does this mean? As discussed above (see Intellia reports positive Phase III results for HAE gene editing trial), current treatment approaches for HAE are divided into three categories: managing acute attacks, short-term prophylaxis and long-term prophylaxis. As Ekterly is indicated for the treatment of acute attacks of HAE, it will compete against other acute attack treatments like bradykinin B2 receptor antagonists (e.g., Firazyr, plus generics), C1 esterase inhibitors (e.g., Berinert, Ruconest) and other plasma kallikrein inhibitors (e.g., Takhzyro, Kalbitor). The acquisition of KalVista also brings a pipeline asset in an oral Factor XIIa inhibitor being developed for HAE prophylaxis, which would complement Ekterly's acute indication and create a vertically integrated HAE franchise under a single owner.

Eli Lilly to acquire Ajax Therapeutics for $2.3 billion

What happened? Eli Lilly (Lilly) and Ajax Therapeutics (Ajax) have announced a definitive agreement for Lilly to acquire Ajax for $2.3 billion. Ajax's lead asset, AJ1-11095, is an investigational, once-daily oral, first-in-class Type II JAK2 inhibitor currently being evaluated in the Phase I AJX-101 trial in patients with myelofibrosis who have previously been treated with a Type I JAK2 inhibitor.

What does this mean? Approved Type I JAK2 inhibitors (e.g., ruxolitinib, fedratinib, pacritinib and momelotinib) all bind JAK2 in its active DFG-in conformation. While they deliver clinical benefit in myelofibrosis (e.g., spleen volume reduction, symptom improvement, and in ruxolitinib's case, an OS benefit) and are established as second-line options after hydroxyurea failure in polycythaemia vera (e.g., ruxolitinib), the durability problem is well documented. Type II inhibitors bind the inactive DFG-out conformation, which has the potential to deliver deeper responses, potentially including reductions in the malignant clone burden, which Type I inhibitors generally do not achieve. Notwithstanding this potential benefit, the Type II hypothesis has not yet proven in late-stage clinical trials. There is also the question of whether deeper JAK2 suppression will produce a more problematic toxicity profile (e.g., anaemia and thrombocytopenia).

LEO Pharma to acquire Replay for $50 million up front

What happened? LEO Pharma has announced that it has entered into a definitive agreement to acquire Replay, a gene therapy company focused on developing treatments for rare genetic dermatological conditions. The acquisition adds expertise and a gene therapy platform to LEO Pharma’s pipeline through Replay’s high‑payload herpes simplex virus (HSV) delivery vector. The lead drug candidate in Replay’s pipeline is currently in preclinical studies for the treatment of a rare genetic condition called dystrophic epidermolysis bullosa (DEB). This condition causes the skin to be fragile and blister easily, leading to painful chronic wounds that heal slowly and poorly, often resulting in scar tissue and skin cancer. Along with the $50 million upfront payment, Replay will be eligible for an undisclosed amount in milestone payments and tiered, single-digit royalties if resulting therapies make it to market.

What does this mean? LEO Pharma’s acquisition of Replay aligns with a broader realisation in the industry; a medicine is only as good as the delivery technology behind it. Similar collaborations have been seen in areas such as ophthalmology (e.g., Boehringer/Re-Vana), RNAi medicines in metabolic diseases (e.g., Lilly/SanegeneBio), in vivo CAR-T in haematology (e.g., Lilly/Kelonia Therapeutics) and multiple therapy areas (e.g., Interna/Daiichi Sankyo). Replay’s HSV platform, which is capable of large payloads, skin targeting, and repeat dosing, addresses some of the field’s most persistent limitations.

Ligand Pharmaceuticals buying XOMA Royalty for $739 million

What happened? Ligand Pharmaceuticals (Ligand) and XOMA Royalty Corporation (XOMA) have announced that the companies have entered into a definitive agreement under which Ligand will acquire XOMA Royalty for $739 million. With this agreement, Ligand adds over 120 commercial, clinical, and preclinical stage assets to its portfolio highlighted by Roche’s Vabysmo (faricimab), Day One Pharmaceuticals’ Ojemda (tovorafenib), Zevra Therapeutics’ Miplyffa (arimoclomol), and 14 programmes in late-stage development, including Takeda’s mezagitamab and certain assets from Takeda’s externalized asset portfolio, including osavampator, volixibat and OHB-607.

What does this mean? The royalty aggregator model, which both companies use, involves taking stakes in drugs that are still in clinical development and earning royalty payments on sales if those treatments ultimately win approval. By adding over 120 assets and seven marketed products, Ligand is positioning itself to monetise innovation at scale. The deal strengthens a diversified, cash-generative portfolio that benefits from biotech’s ongoing need for non-dilutive funding. In a market where R&D risk remains high and capital is selective, royalty platforms offer a compelling middle ground: exposure to upside without binary clinical risk.

Sun Pharma to acquire Organon for $11.75 billion

What happened? Sun Pharmaceutical Industries (Sun Pharma) and Organon have announced that the companies have entered into a definitive agreement under which Sun Pharma will acquire Organon for $11.75 billion. Organon was formed through a spinoff from Merck & Co. in June 2021 with the aim of becoming a leader in women’s health.

What does this mean? Sun Pharma’s acquisition of Organon is one of the largest ever deals by an Indian pharma company. By adding a global portfolio of established women’s health brands and biosimilars, Sun is accelerating its shift away from commoditised generics toward higher-margin, branded medicines. The deal instantly expands its global footprint and positions it as a leading player in women’s health, while providing stable, cash-generative revenues.

Teva to acquire Emalex Biosciences for $700 million

What happened? Teva Pharmaceuticals (Teva) and Emalex Biosciences (Emalex) have announced a definitive agreement for Teva to acquire Emalex, including its lead asset, ecopipam, for $700 million. Positive Phase III data for ecopipam in children with Tourette syndrome were announced in February 2025, and the NDA submission is anticipated in H2’26.

What does this mean? Ecopipam is a selective dopamine D1 receptor antagonist. Currently approved Tourette syndrome medications act primarily on D2 receptors (e.g., haloperidol, pimozide, aripiprazole). The theoretical advantage of targeting D1 specifically is circuit selectivity. By acting on the direct pathway component of the cortico-striato-thalamo-cortical (CSTC) loop, ecopipam may be able to dampen the tic-generating signal without the widespread dopaminergic suppression that causes extrapyramidal effects and the other side effects associated with D2 blockade. The nigrostriatal pathway, which is the principal driver of drug-induced parkinsonism, is more dependent on D2 signalling than D1, so a selective D1 antagonist should theoretically spare it. The Phase III data for ecopipam appear to support this: the tolerability signal from clinical trials has been favourable, with no significant extrapyramidal effects reported.

In Other News

AstraZeneca’s Breztri wins FDA nod for asthma in over-12s

AstraZeneca’s fixed-dose triple-combination therapy Breztri Aerosphere (budesonide/glycopyrrolate/formoterol fumarate, or BGF) has been approved in the US for the maintenance treatment of asthma in adult and paediatric patients 12 years of age and older. Breztri was approved in the US in July 2020 to treat adults with chronic obstructive pulmonary disease (COPD). The approval by the FDA was based on efficacy and safety data from the Phase III KALOS and LOGOS trials investigating Breztri in a broad population consisting of patients with asthma, with or without a recent asthma exacerbation. Recall, AstraZeneca obtained Breztri (formerly PT010) as part of its June 2013 acquisition of Pearl Therapeutics.

BeOne Medicines licenses pre-clinical oncology asset from Huahui

Huahui Health (Huahui) has announced that it has entered a global exclusive option, license and collaboration agreement with BeOne Medicines (BeOne) for HH160, a novel tsAb that targets PD-1, CTLA-4, and VEGF-A. Under the agreement, BeOne Medicines is granted an exclusive option covering the development, manufacturing, and commercialisation of HH160 worldwide. Under the terms of the deal, Huahui is eligible for a total of $1.9 billion, including upfront and milestone payments, as well as tiered royalties on net sales.

EverythingALS launches SAVA to match patients to clinical trials

EverythingALS has announced the launch of SAVA, an AI-powered clinical trial matching tool that connects ALS patients with clinical trials they may qualify for. SAVA AI will work by using citizen-driven research and data-driven insights to directly empower patients. Available on the EverythingALS website and free app, patients now can securely provide their data, receive real-time alerts about relevant clinical trials they are eligible for, and share this information with their physicians and family members. The collective data is made available to researchers and pharmaceutical companies, strengthening the entire ALS ecosystem.

FDA approves label extensions for Alyftrek and Trikafta

Vertex Pharmaceuticals has announced the FDA has approved expanded use of Alyftrek (vanzacaftor/tezacaftor/ivacaftor) for the treatment of people with cystic fibrosis (CF) ages 6 years and older with a variant in the CFTR gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. Additionally, the U.S. FDA has also expanded the indication statement for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in patients ages 2 years and older. This label expansion was supported by clinical and/or in vitro data from 564 variants demonstrating response to Alyftrek and 521 variants demonstrating response to Trikafta. As such, approximately 800 more people with a clinical diagnosis of CF in the US are now eligible for a medicine that treats the underlying cause of their disease for the first time. This extension means approximately 95% of people with CF in the US are now eligible for treatment with a CFTR modulator.

FDA unveils real-time clinical trial review plan

The FDA has announced a new initiative to allow its reviewers to access information from clinical trials in real time, with two major industry players already taking part in a pilot programme. AstraZeneca and Amgen are both conducting trials that will report safety and efficacy signals to the FDA as they come in, the agency said, rather than waiting for data to be collected, analysed and submitted.

GSK’s bepirovirsen gets brace of good news from the FDA

GSK has announced that the FDA has accepted for priority review a NDA for bepirovirsen, an investigational antisense oligonucleotide (ASO), for the treatment of adults with chronic hepatitis B (CHB). Bepirovirsen has also received Breakthrough Therapy Designation (BTD). The BTD for bepirovirsen builds on the Fast Track Designation also provided by the US FDA in February 2024. The regulatory submission and BTD are supported by positive results from the Phase III B-Well 1 and B-Well 2 trials, where bepirovirsen demonstrated statistically significant and clinically meaningful functional cure rates. Recall, GSK licensed development rights to bepirovirsen from Ionis in August 2019.

Is Foundayo suffering from a brand recognition problem?

Analysts from Truist Securities have reported prescriptions for Foundayo (orforglipron) during its first full week on the US market (ending April 17) reached 3,707 compared to 18,410 prescriptions that oral Wegovy achieved during its first full week on the market. Analysts posited that Wegovy’s brand name may have aided its launch.

J&J’s Caplyta gets FDA nod for prevention of schizophrenia relapse

Johnson & Johnson has announced that the FDA has approved a sNDA based on long-term data evaluating the safety and efficacy of Caplyta (lumateperone) for the prevention of relapse in schizophrenia. In the Phase III trial supporting this update, Caplyta significantly extended time to relapse versus placebo during the 26-week double-blind treatment period (p=0.0002). Patients who received Caplyta had a 63% percent lower risk of relapse compared with placebo, and 84% of patients were relapse-free over six months. Caplyta also significantly delayed time to all-cause treatment discontinuation, including relapse. The safety profile remained consistent with the existing body of clinical data.

J&J’s Imaavy gets FDA priority review in wAIHA

Johnson & Johnson has announced today that the FDA has granted Priority Review to the sBLA for Imaavy (nipocalimab, confirming the urgent need for treatment options in warm autoimmune haemolytic anaemia (wAIHA). Imaavy is the first therapy to receive FDA Priority Review for this condition. The FDA’s decision to grant Priority Review is supported by results from the pivotal Phase II/III ENERGY study, which showed that more patients treated with Imaavy achieved a durable haemoglobin response compared with placebo, along with improvements in fatigue, a critical outcome for people living with wAIHA. The full results of the ENERGY trial will be presented at an upcoming medical conference.

MHRA announces significant clinical trials reform

The Medicines and Healthcare products Regulatory Agency (MHRA) and Health Research Authority (HRA) are introducing significant clinical trials reform in the UK. Reforms include faster assessment of first in human trials and the introduction of notifiable trials, a fast-track route to allow lower-risk trials to start sooner and modification to be approved quicker, whilst maintaining the highest safety standards. The reforms will make it simpler to start lower-risk studies, strengthen support for early-stage research and embrace new approaches, including use of early safety data from overseas studies which meets UK standards and computer model simulations which can help to predict how new medicines may behave before they are tested in patients. The reforms will come into effect on April 28, 2026.

Mirum’s brelovitug delivers Phase IIb win in chronic HepD

Mirum Pharmaceuticals has announced the primary endpoint was met in the Phase IIb portion of the Phase II/III AZURE-1 study evaluating brelovitug, an investigational monoclonal antibody designed to bind hepatitis B surface antigen (HBsAg), for the treatment of chronic hepatitis delta virus (HDV). At Week 24, treatment with brelovitug demonstrated robust antiviral activity across two dose groups. 100% of evaluable patients in the 300mg QW arm and 75% of patients in the 900mg Q4W arm achieved virologic response (≥2 log10 reduction in HDV RNA from baseline or undetectable HDV RNA [<LLOQ, TND]), as compared to 0% in the delayed treatment arm.

Novartis malaria treatment Coartem Baby receives WHO prequalification

Novartis has announced that the WHO has prequalified Coartem (artemether-lumefantrine) Baby, the first and only antimalarial developed specifically for newborns and young infants weighing from 2 to 5 kilograms. The decision is a key step towards enabling widespread access through public sector procurement. Coartem Baby is also known as Riamet Baby in some countries and was developed in collaboration with Medicines for Malaria Venture (MMV). Prequalification is a process run by the WHO to assess the quality, safety and efficacy of treatments for diseases like malaria, HIV/AIDS and tuberculosis. Prequalification outputs, including lists of prequalified products, are used by United Nations and other procurement agencies to inform funding and purchasing decisions for the public sector.

Novartis pulls EMA bid for new Pluvicto indication

Novartis has withdrawn an EMA type II variation application for Pluvicto to treat adult patients with prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) pre-chemotherapy, following CHMP feedback. The CHMP indicated that they would not support the application based on the control arm used in the PSMAfore study (which was abiraterone or enzalutamide, based on the investigator’s discretion). The withdrawal is not related to the quality, efficacy or safety of Pluvicto and does not impact ongoing clinical trials, approved indications or pending regulatory submissions inside or outside the EU.

Novo Nordisk to release new obesity data at ESO

Novo Nordisk will present new clinical data and real-world evidence at the European Congress on Obesity (ESO) 2026. The full data, 52 abstracts, span Wegovy (semaglutide 2.4 mg), higher-dose Wegovy (semaglutide 7.2 mg), Wegovy pill (oral semaglutide 25 mg), and CagriSema (cagrilintide 2.4 mg/semaglutide 2.4 mg), an investigational first-in-class combination of a GLP-1 receptor agonist and a long-acting amylin analogue.

Pfizer’s Elrexfio improves PFS in R/RMM

Pfizer has announced positive topline results from the Phase III MagnetisMM-5 study evaluating Elrexfio (elranatamab; BCMAxCD3 bsAb) as monotherapy in adults with relapsed or refractory multiple myeloma (R/RMM) who received at least one prior line of treatment. The study demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of PFS vs. standard-of-care daratumumab plus pomalidomide and dexamethasone (DPd). The safety and tolerability of Elrexfio was consistent with its known safety profile. The PFS results exceeded the pre-specified interim analysis target hazard ratio for efficacy, with most Elrexfio-treated patients remaining progression-free. The trial remains ongoing to assess overall survival, a key secondary endpoint, which was not yet mature at the time of this interim analysis.

ReSPECT for Mundipharma’s Rezzayo after Phase III win

Mundipharma has announced positive topline results from the Phase III ReSPECT trial evaluating Rezzayo (rezafungin acetate) for prophylaxis of invasive fungal diseases (IFDs) in adult patients undergoing allogeneic haematopoietic stem cell transplantation (HSCT). ReSPECT evaluated the efficacy and safety of once weekly rezafungin compared with a standard antimicrobial regimen (SAR) for the prophylaxis of IFDs, including infections caused by Candida, Aspergillus, and Pneumocystis, in adults undergoing allogeneic HSCT. Permitted comparator therapies included posaconazole (Noxafil; MSD), fluconazole or trimethoprim-sulfamethoxazole. The ReSPECT study met its primary endpoint for FDA and EMA standards for fungal-free survival at Day 90, showing non-inferiority of rezafungin vs. SAR (60.7% vs. 59.0%, respectively). Rezzayo is already approved in Europe as a treatment for adults with invasive candidiasis.

Samsung Bioepis publishes its Q2 2026 biosimilars report

Samsung Bioepis has released its Q2 2026 Biosimilar Market Report, marking the thirteenth edition of the Quarterly Biosimilar Market Report in the US. The report provides an overview of the market status of all biosimilars available in the US, including approval and launch status, pricing (both ASP and WAC), and market uptake per molecule.

Structure Therapeutics announces multiple presentations at ADA 2026

Structure Therapeutics (Structure) has announced multiple presentations at American Diabetes Association (ADA) 2026. The presentations include an oral presentation on the Phase IIb ACCESS clinical trial of aleniglipron in patients who are obese or overweight, along with presentations on ACCG-2671 (amylin receptor agonist) and combination regimens.

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.