Pharma Phriday (May 15, 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

• LenioBio and Twist Bioscience collaborate on AI drug discovery.

• Nuclera launches new service to support AI-driven antibody R&D.

• Owkin partners with AstraZeneca on biopharma agent development.

Clinical

• Alkermes posts positive data from the Phase III REVITALYZ trial.

• AstraZeneca’s Phase III CALYPSO meets primary endpoint, but…

• Eli Lilly posts weight maintenance data for Foundayo and Zepbound.

• Enterprise Therapeutics posts positive Phase II data for ETD001.

• First patient dosed with Lysoway Therapeutics’ LW-1017.

• Incyte reports 24 week data from the Phase III TRuE-AD4 trial.

• Inhibrx announces interim Phase II results for INBRX-106 in 1L HNSCC.

• Ionis toplines results from Phase II CELIA study of diranersen.

• Novo Nordisk presents oral Wegovy data set at ECO2026.

• Regenxbio toplines results from the Phase III AFFINITY DUCHENNE trial.

Regulatory

• BeOne’s Beqalzi gets FDA nod for R/R mantle cell lymphoma.

• BMS’ Sotyktu gets EC nod for adult PsA.

• EC approves label expansion for Pfizer’s Hympavzi.

• Partner’s Bizengri gets FDA nod for NRG1 fusion+ cholangiocarcinoma.

Commercial

• Boehringer Ingelheim licenses preclinical antibody from Immunitas.

• Cellular Intelligence acquires STEM-PD from Novo Nordisk.

• GSK collaborates with China’s SBP Group on bepirovirsen launch.

• Hengrui and BMS announce $15.2 billion, multi-asset R&D collaboration.

• Pfizer/Arvinas off-load Veppanu to Rigel for up to $405 million.

In Other News

• AstraZeneca expands collaboration with Immunai.

• Chugai files obinutuzumab for INS in Japan.

• Eli Lilly launches donanemab in India.

• FDA approves Taiho’s Inqovi/Venclexta combo in AML.

• FDA delays review of Eisai/Biogen’s Leqembi Iqlik.

• Fosun licenses pipeline Alzheimer's disease asset from AriBio.

• Isomorphic Labs secures $2.1 billion Series B funding.

• New AI technology to speed drug development.

• Oruka licenses Halozyme’s Hypercon for ORKA-001.

• Reformulated vorinostat enters the clinic for Rett and Pitt Hopkins syndrome.

• UAE approves AstraZeneca’s Baxfendy.

• Vyome in-licenses two selective JAK inhibitor assets from Impetis.

Summary

• Artificial Intelligence. Three AI deals featured this week, each addressing a different layer of drug development. LenioBio and Twist Bioscience integrated their cell-free expression and DNA manufacturing platforms to compress antibody validation from weeks to days, addressing the rate-limiting bottleneck in generative AI discovery workflows. Nuclera launched a complementary antibody triage service using the same cell-free approach but positioned as a commercial service rather than a platform partnership. Owkin signed a three-year agreement with AstraZeneca to deploy autonomous AI agents for competitive intelligence within AstraZeneca's internal decision-making infrastructure, representing a different and arguably more commercially mature application of AI at the strategic rather than discovery layer.

• Clinical. The most significant readout was diranersen (Biogen/Ionis) in early Alzheimer's disease, where the Phase II CELIA study missed its primary dose-response endpoint but demonstrated cognitive benefit at specific doses alongside robust reductions in CSF tau and tau PET pathology. This is the first randomised evidence of a tau-directed therapy producing both biomarker and cognitive signals, and Biogen has committed to Phase III. In DMD, Regenxbio's Phase III AFFINITY DUCHENNE trial met its primary endpoint with 93% of patients achieving at least 10% microdystrophin expression at Week 12, supporting a mid-2026 accelerated approval BLA submission. Inhibrx's hexavalent OX40 agonist INBRX-106 approximately doubled confirmed response rates versus pembrolizumab monotherapy in first-line PD-L1-high HNSCC, validating valency as a key design parameter for T-cell costimulatory agonists and sharpening M&A interest in the company. In obesity, Eli Lilly presented maintenance data showing patients switching from Wegovy to Foundayo retained almost all prior weight loss, a direct counter to Novo Nordisk's competitive claims at ECO2026. Enterprise Therapeutics reported the first demonstration of efficacy for an inhaled ENaC blocker in patients with CF ineligible for CFTR modulators. Alkermes posted strong Phase III data for Lumryz in idiopathic hypersomnia, though a patent settlement prevents marketing before March 2028.

• Regulatory. Four notable approvals. The FDA granted accelerated approval to BeOne's Beqalzi (sonrotoclax) as the first BCL2 inhibitor specifically approved for relapsed or refractory mantle cell lymphoma post-BTK inhibitor, with a notable 15.8-month median duration of response. The EC approved Sotyktu (deucravacitinib) as the first TYK2 inhibitor for active psoriatic arthritis in the EU, and Hympavzi (marstacimab) received an EC label extension covering haemophilia A and B patients with inhibitors, where it holds a commercial advantage over emicizumab through its coverage of haemophilia B. Partner Therapeutics received FDA approval for Bizengri (zenocutuzumab) in NRG1 fusion-positive cholangiocarcinoma, the first targeted therapy for this molecularly defined indication, expedited by a Commissioner's National Priority Voucher.

• Commercial. The Hengrui-BMS collaboration dominated the week. At up to $15.2 billion in potential value with a $600 million upfront, it’s one of the largest China-originated deals ever announced, covering 13 early-stage programmes across oncology, haematology, and immunology. The reciprocal platform structure reflects BMS's need to replenish a pipeline facing patent cliff pressure on Eliquis, Opdivo, and Pomalyst. Pfizer and Arvinas out-licensed Veppanu to Rigel for up to $405 million. GSK secured a commercial partnership with CTTQ to distribute bepirovirsen across China's 75 million chronic hepatitis B patients at launch. Cellular Intelligence acquired Novo Nordisk's discontinued STEM-PD Parkinson's cell therapy programme, aiming to apply AI to accelerate development. Boehringer Ingelheim licensed a preclinical antibody targeting tissue-resident inflammatory cells from Immunitas.

• In Other News. Isomorphic Labs raised $2.1 billion in Series B funding. Taiho's Inqovi plus venetoclax became the first all-oral AML combination approved for transplant-ineligible patients. Eli Lilly launched donanemab in India at approximately $1,000 per vial. Leqembi Iqlik's subQ-as-starting-dose review was delayed by three months to August 2026. The UAE became the first market globally to approve baxdrostat (Baxfendy) for resistant hypertension.

Artificial Intelligence

LenioBio and Twist Bioscience collaborate on AI drug discovery

What happened? LenioBio GmbH (LenioBio) and Twist Bioscience Corporation (Twist) are to collaborate on AI drug discovery. The collaboration will integrate LenioBio’s ALiCE platform with Twist’s DNA manufacturing and automation capabilities to accelerate the design–build–test cycle for protein expression services, thereby delivering experimental results back to customers’ and partners’ AI models faster. In AI-driven protein and antibody design, advantage comes from iteration speed and the quality of biology-native data. This collaboration will help teams run faster, higher-throughput lab-in-the-loop cycles with rapid, eukaryotic cell-free expression.

What does this mean? A platform services collaboration aimed at the growing number of biotechs using generative AI for antibody and protein design, the practical value proposition of which is speed (i.e., compressing weeks of experimental validation into days). There is little point in generating thousands of AI-designed protein sequences if validating them takes months. Collaborations like this one represent the field's response to that constraint and are becoming a standard feature of the AI drug discovery ecosystem.

Nuclera launches new service to support AI-driven antibody R&D

What happened? Nuclera has announced the launch of its antibody screening service to streamline the transition from antibody hit-to-lead selection. The hit-to-lead stage involves evaluating the most promising compounds and selecting a smaller number (i.e., the leads) that show the best combination of potency, selectivity and early safety signals. The service will support researchers seeking to identify the most promising antibody candidates to progress to costly mammalian expression and functional testing, addressing a key bottleneck in AI-driven antibody discovery. As AI-driven approaches generate increasingly large antibody libraries, the need for rapid, cost-effective triage solutions has become more critical. Nuclera’s antibody service addresses this gap by converting large AI-generated libraries into experimental binding data and enabling early elimination of non-binders, reserving expensive downstream biology for candidates proven to bind and accelerating progression to validated leads.

What does this mean? A similarly focused deal to the one above (LenioBio/Twist). As generative AI platforms produce increasingly large antibody candidate libraries, the experimental validation step has become the rate-limiting constraint on discovery productivity. Via the use of cell-free expression systems, this collaboration seeks to improve this productivity. Interestingly, Nuclera is positioning this as a service offering rather than a platform partnership, which is a different commercial model from LenioBio-Twist but targets the same problem. Taken together, these two announcements in the same week are illustrative of a maturing sub-sector (i.e., improving the validation bottleneck).

Owkin partners with AstraZeneca on biopharma agent development

What happened? Owkin, an AI focused drug discovery company, has announced an agreement with AstraZeneca to build biopharma agents as part of their three-year licensing of K Pro, Owkin’s AI Scientist for biopharma decision making. K Pro brings multimodal data and specialised biological agentic AI to each step of the value chain. Under the three-year licensing agreement, Owkin will lead the end-to-end development of AI agents to run on K Pro, integrated within AstraZeneca’s IT infrastructure and decision workflows. The new agents' functionality is intended to help AstraZeneca’s decision-making teams access timely, data-rich insights for complex competitive intelligence questions, reducing reliance on manual analysis within established governance, security, and enterprise standards.

What does this mean? This deal is meaningfully different in character from the LenioBio/Twist and Nuclera announcements. Those two are about accelerating wet-lab experimental validation, whereas this one is about applying AI at the strategic and commercial layer of pharma operations, specifically competitive intelligence. Competitive intelligence (e.g., tracking competitor pipelines, forecasting clinical outcomes, monitoring regulatory activity, etc.) has traditionally been a labour-intensive, analyst-driven function. Embedding autonomous AI agents into executive decision workflows represents a genuine operational change for a company of AstraZeneca's scale.

Clinical

Alkermes posts positive data from the Phase III REVITALYZ trial

What happened? Alkermes has announced positive topline results from the Phase III REVITALYZ trial evaluating the investigational use of Lumryz (sodium oxybate) extended-release oral suspension in adults with idiopathic hypersomnia (IH). Lumryz met the study’s primary endpoint, demonstrating statistically significant improvements in excessive daytime sleepiness compared to placebo as measured by the change in Epworth Sleepiness Scale (ESS)1 score (p<0.0001). The safety profile of Lumryz in the REVITALYZ study was generally consistent with previously observed safety data associated with Lumryz, with no new safety signals observed in this population. 

What does this mean? The data are clean and statistically significant, but under a prior patent settlement and licence agreement, Alkermes cannot market Lumryz for IH before March 2028, even if FDA approval is granted. That is a two-year commercial lockout on an approved product in a new indication, an unusual constraint that limits the near-term revenue. In the meantime, patients with IH will be limited to using wake promoting agents (e.g., modafinil, armodafinil), stimulants (e.g., Ritalin, Adderall), Xywav (calcium, magnesium, potassium, and sodium oxybates; Jazz Pharmaceuticals) or off-label options (e.g., Sunosi). Investigational options that could shape the longer-term treatment paradigm for IH include orexin receptor agonists (ORAs), including Alkermes's own alixorexton (ALKS2680) which is in development for IH.

AstraZeneca’s Phase III CALYPSO meets primary endpoint, but…

What happened? Following positive week 24 data announced in March 2025, AstraZeneca has posted additional positive Week 52 results from the Phase III CALYPSO trial. In the trial, 31.1% of patients with hypoparathyroidism treated with eneboparatide met the composite primary endpoint (normalisation of albumin-adjusted serum calcium (sCa) levels and independence from active vitamin D and oral calcium supplements) at week 24, compared with 5.9% of patients on placebo. Efficacy would have been better were it not for immunogenicity, which was observed in most patients, resulting in reduced treatment effects in some patients. The trial also met all key secondary endpoints at week 24, including normalisation of urinary calcium (uCa) excretion in patients with hypercalciuria at baseline. Eneboparatide also demonstrated statistically significant improvements in patient-reported outcomes reflecting disease-specific core physical symptoms and physical functioning, as well as SF-36-PFS. After week 24, patients continued eneboparatide or were switched to eneboparatide from placebo for an additional 28 week open-label extension (OLE) period. In the treatment continuation group, clinical benefits were maintained through week 52 with some use of oral supplements, as assessed by OLE efficacy endpoints. Eneboparatide was well

tolerated over 52 weeks of treatment.

What does this mean? Hypoparathyroidism has a significant unmet need. The only approved PTH replacement therapy currently is Yorvipath (palopegteriparatide; Ascendis Pharma) which was approved in August 2024. While eneboparatide's once daily subQ profile and breadth of data are compelling, the absolute response vs. Yorvipath needs calling out. At 24 weeks, eneboparatide achieved the composite response (normalised serum calcium plus independence from active vitamin D and oral calcium supplements) in 31.1% of patients versus 5.9% on placebo. In the PaTHway trial, palopegteriparatide achieved normocalcaemia and independence from conventional therapy in approximately 79% of patients at 26 weeks in the blinded period. By 52 weeks this had risen to 86% maintaining normocalcaemia, and by three years 88% had normal serum calcium with 96% independent of conventional therapy. This is a large gap: 31% versus 79% at comparable timepoints. Some things to flag, however. The CALYPSO composite endpoint required full independence from both active vitamin D and oral calcium supplementation, which is a demanding standard. The PaTHway trial allowed up to 600mg per day of calcium supplementation and still counted patients as achieving independence from conventional therapy, which is a meaningfully lower bar. Additionally, the trials enrolled different patient populations with potentially different baseline disease severity, and the titration protocols differed. Interestingly, immunogenicity was also observed with Natpara (PTH; Takeda) in its clinical programme, so this is not an eneboparatide-specific problem. Natpara was withdrawn from the US market at the end of 2024 due to manufacturing issues.

Eli Lilly posts weight maintenance data for Foundayo and Zepbound

What happened? Eli Lilly has announced detailed results from two Phase III trials (SURMOUNT-MAINTAIN and ATTAIN-MAINTAIN) at ECO2026. Both trials assessed whether patients could maintain weight loss after transitioning from higher-dose injectable incretin therapy to either Foundayo (orforglipron) or a lower dose of Zepbound (tirzepatide). Data from the SURMOUNT-MAINTAIN trial show that participants who stayed on Zepbound at their maximum tolerated dose lost 25.2 kg (22.4%) from baseline across the full treatment period, while those who reduced to the 5mg dose lost 19.2 kg (17.0%). Those who reduced to 5mg maintained all but 5.6 kg of their prior weight loss during the maintenance phase. The implication is that a lower-dose Zepbound could be a viable, potentially cheaper maintenance option. Data from the ATTAIN-MAINTAIN trial show that participants who switched from a maximum tolerated dose of Wegovy (semaglutide) to Foundayo maintained all but 0.9 kg of their previously achieved weight loss after one year, which is a remarkably small amount. Those switching from Zepbound to Foundayo maintained all but 5.0 kg over the same period. The larger regain from Zepbound is consistent with what you'd expect given tirzepatide's dual GIP/GLP-1 mechanism, as switching to a GLP-1-only oral agent means losing some of that additional efficacy.

What does this mean? Foundayo prescriptions have been trending upward since its April launch, but scripts have lagged oral Wegovy and analyst expectations. These data are therefore about reassuring prescribers and patients that Foundayo works as a maintenance therapy. Interestingly, Novo Nordisk has released data from the ORION trial claiming that Foundayo was associated with approximately 14 times higher odds of GI discontinuation compared with the Wegovy pill (see Novo Nordisk presents oral Wegovy data set at ECO2026). Eli Lilly is essentially countering with real-world maintenance data showing that patients can transition to Foundayo and stay on it, and with very little weight regain, at least when coming from Wegovy. Neither company's data is head-to-head in a prospective randomised trial, so both claims should be treated with due caution.

Enterprise Therapeutics posts positive Phase II data for ETD001

What happened? Enterprise Therapeutics (Enterprise) has announced that a Phase II trial for ETD001 has achieved its primary efficacy outcome. ETD001 is an investigational inhaled epithelial sodium channel (ENaC) blocker designed to increase lung function by improving mucus clearance and reducing airway obstruction. The trial assessed the efficacy, safety, tolerability and PK of ETD001 in patients with cystic fibrosis who do not benefit from treatment with CFTR modulators (e.g., Trikafta). The trial was split into two parts. Part A assessed the safety and tolerability of repeat inhaled doses of ETD001 over 7 days, through monitoring adverse events. Part B used a double-blind, placebo-controlled, cross-over design to examine the effect of repeat BID dosing of ETD001 (4.5mg) over 28 days on absolute change in ppFEV1. Participants were randomised to receive either placebo or ETD001 for 28 days in a two group, two sequence cross-over design, with a 28-day washout period. Data from the trial demonstrate a difference of 3.4% (p value = 0.0053) in percent predicted forced expiratory volume in 1 second (ppFEV1) in patients dosed with ETD001, compared to placebo. Exploratory analysis showed that participants had a three times higher likelihood of improving ppFEV1, and therefore improving lung function, when receiving ETD001 compared to placebo. The results also show that ETD001 is overall well-tolerated, with adverse events consistent with those expected in this trial population when receiving inhaled medicines.

What does this mean? Context is critical here. ETD001 is specifically targeting the ~10% of CF patients who are ineligible for or not responding to CFTR modulators; this is the hardest-to-treat CF population. A 3.4 point ppFEV1 improvement in 28 days in that group is meaningful. As the first ENaC blocker to demonstrate efficacy in CF patients, this is a notable mechanistic milestone. In terms of what comes next, non-CF bronchiectasis is a substantially larger commercial opportunity than the 10% CFTR-ineligible CF population. If the mucus clearance mechanism translates there it could expand the utility of ETD001 into other respiratory indications (e.g., COPD, primary ciliary dyskinesia, alpha-1 antitrypsin deficiency, etc.).

First patient dosed with Lysoway Therapeutics’ LW-1017

What happened? Lysoway Therapeutics (Lysoway) has announced that the first patient has been dosed with LW-1017, a selective, brain-penetrant small-molecule Transient Receptor Potential Mucolipin 1 (TRPML1) agonist being developed for neurodegenerative diseases including Alzheimer’s disease and Parkinson’s disease. The Phase I study is being conducted in Melbourne, Australia and is designed to evaluate the safety, tolerability, and pharmacokinetics of LW-1017 in healthy volunteers through single ascending dose (SAD) and multiple ascending dose (MAD) cohorts. LW-1017 is the first TRPML1 channel agonist to enter clinical development.

What does this mean? The lysosomal pathway has had strong genetic validation for years, particularly TMEM175, which has multiple genome-wide association signals linking it to Parkinson's disease risk. Up until now the barrier to moving a TRPML1 agonist into the clinic has been the difficulty of building a small molecule that is simultaneously potent, selective, orally bioavailable, and sufficiently brain-penetrant for a target expressed broadly in peripheral tissues. Lysoway's structure-guided platform using cryo-EM appears to have solved that problem for TRPML1.

Incyte reports 24 week data from the Phase III TRuE-AD4 trial

What happened? Incyte has announced final week 24 data from the Phase III TRuE-AD4 study evaluating the efficacy and safety of Opzelura (ruxolitinib) cream in adults with moderate atopic dermatitis (AD) who had an inadequate response, intolerance or contraindication to topical corticosteroids (TCSs) and topical calcineurin inhibitors (TCIs). As reported in October 2025, the TRuE-AD4 study met both of its co-primary endpoints at Week 8, with a statistically significantly higher proportion of patients on Opzelura versus vehicle cream achieving EASI75 (≥75% improvement in Eczema Area and Severity Index score from baseline) and, separately, IGA-TS (Investigator's Global Assessment Treatment Success, defined as an IGA score of 0 [clear] or 1 [almost clear] with at least a two-point improvement from baseline). Patients who achieved an EASI50 response at Week 8 continued double-blind treatment, as needed, through Week 24. Most patients (84.3%) in the Opzelura treatment group completed treatment through Week 24. Among those patients, 84.3% achieved EASI75 and 70.6% achieved IGA-TS at Week 24, like the frequencies at Week 8 (83.5% and 74.4%, respectively). Mean affected body surface area (BSA) remained low (2.5% at Weeks 8 and 24) and itch relief (NRS4; ≥4-point improvement in Itch Numeric Rating Scale) remained high (74.3% at Week 8 and 64.7% at Week 24) with Opzelura treatment. As-needed treatment with Opzelura was well tolerated, with few application site reactions (1.7%) and no new safety signals up to 24 weeks.

What does this mean? Recall, Opzelura was approved by the FDA for the treatment of mild/moderate AD in September 2021. In Europe, Incyte has a Type-II variation application pending with the EMA to extend Opzelura's EU label to adults with moderate AD, with regulatory feedback expected in the first half of 2026. Opzelura is only approved in the EU for non-segmental vitiligo with facial involvement in adults and adolescents aged 12 and older, so this is a label extension into the European AD treatment landscape would provide a significant growth opportunity. Moderate AD patients who have failed topical therapy, but whose prescribers or health systems prefer not to escalate immediately to targeted systemic therapy (e.g., dupilumab, oral JAK inhibitors) represent a large and currently underserved group.

Inhibrx announces interim Phase II results for INBRX-106 in 1L HNSCC

What happened? Inhibrx Biosciences (Inhibrx) has announced positive interim results from the randomised, 1L Phase II portion of the HexAgon study. The trial evaluated the safety and efficacy of INBRX-106, a hexavalent OX40 agonist, in combination with pembrolizumab (combo arm) versus pembrolizumab monotherapy (control arm) in 1L patients with treatment-naïve, PD-L1 positive (CPS ≥ 20) metastatic or unresectable recurrent Head and Neck Squamous Cell Carcinoma (HNSCC). The trial design was modelled after KEYNOTE-048, focusing on patients with high PD-L1 expression to further sharpen the ability to detect a treatment effect above checkpoint inhibition alone. In the evaluable population, 11/25 patients (44%) in the INBRX-106 combo arm achieved a confirmed objective response, compared with 6/28 patients (21.4%) in the control arm. This represents a 22.6% absolute increase in confirmed responses. Three complete responses were observed in the INBRX-106 combination arm, reflecting tumour clearance, while no complete responses were observed with pembrolizumab alone. The combination of INBRX-106 and pembrolizumab was generally manageable, with a safety profile consistent with the addition of an active immunostimulatory agent to checkpoint blockade. 

What does this mean? This is interim data from 53 of the total Phase II population, with 15 patients still pending evaluability. Response rates can shift as more patients mature. PFS data, which will be the more meaningful endpoint for regulatory purposes, are not yet available and are expected in Q4’26. Safety data are not disclosed in detail in the press release summaries; more detail is expected later in the year. OX40 agonism can drive immune-related adverse events, and the tolerability profile of the combination will matter clinically. Inhibrx plans to initiate the Phase III portion of HexAgon in Q3’26 and is also planning expansion into perioperative and front-line metastatic NSCLC settings from 2026-27. The speed of the Phase III initiation suggests the company has sufficient confidence in these interim data to proceed without waiting for PFS maturity. The broader significance relates to the mechanism itself. If a hexavalent OX40 agonist can consistently outperform PD-1 monotherapy in a randomised setting, it would validate the hypothesis that valency is the critical design parameter for T-cell costimulatory agonists, with implications for the broader costimulatory antibody field. This data is likely to sharpen acquirer interest in Inhibrx. Merck & Co. would be a logical acquirer given the dominance of Keytruda and its impending patent expiry in 2028.

Ionis toplines results from Phase II CELIA study of diranersen

What happened? Ionis Pharmaceuticals (Ionis) and its partner, Biogen, have announced topline results from the Phase II CELIA study evaluating diranersen, an investigational antisense oligonucleotide (ASO) therapy targeting tau, in individuals with early Alzheimer’s disease (AD). The CELIA results provide the first evidence that tau-directed therapy delivers both robust biomarker impact and cognitive benefit in early AD. Pre-specified analyses of cognitive endpoints demonstrated slowing of clinical decline across all studied doses, particularly in participants receiving the lowest dose of diranersen, 60mg administered every 24 weeks. Diranersen also demonstrated reductions in both cerebrospinal fluid (CSF) tau and tau pathology, as measured by positron emission tomography (PET), across all studied doses, with reductions maintained throughout the dosing period. CELIA did not meet its primary endpoint assessing dose response for change from baseline on the Clinical Dementia Rating–Sum of Boxes (CDR-SB) at Week 76.

What does this mean? Current approved Alzheimer's therapies (e.g., lecanemab and donanemab) target amyloid. The prevailing hypothesis is that Alzheimer's requires addressing both amyloid and tau, and that tau pathology is more directly linked to neurodegeneration and cognitive decline than amyloid burden. A tau-directed therapy demonstrating cognitive signals alongside tau biomarker reduction would represent a genuinely new class of potential treatment, complementary to anti-amyloid approaches rather than competing with them. The missed primary endpoint is not necessarily fatal to the programme. The primary endpoint was a dose-response assessment across all three doses, which is a high bar. Finding significant effects at specific doses within a failed dose-response test is a pattern regulators accept as supportive for Phase III planning, provided the signal is consistent and the biomarker data corroborate it. Whether that is the case here requires the full AAIC presentation to assess properly. For Biogen, this is an important result. The company has invested heavily in Alzheimer's following the controversial Aduhelm situation and the subsequent commercial struggles of Leqembi (lecanemab). A credible tau programme would give Biogen a second Alzheimer's asset with a differentiated mechanism, and potentially a combination approach with lecanemab if both amyloid and tau targeting prove necessary. Biogen has already committed to Phase III advancement based on these data.

Novo Nordisk presents oral Wegovy data set at ECO2026

What happened? Novo Nordisk has presented three updates on oral Wegovy at the European Congress on Obesity (ECO) 2026. First up was a sub-analysis of the Phase III OASIS-4 trial, providing efficacy details of oral Wegovy in early responders. Around 29% of participants on oral Wegovy (25mg) were classified as early responders, defined as achieving at least 10% weight loss by week 16. This group averaged 13.2% weight loss at that four-month mark and went on to reach 21.6% weight loss by the end of the 64-week trial. Those who did not meet the early responder threshold still achieved 11.5% weight loss, described as clinically meaningful. In a separate OASIS 4 sub-analysis, nearly 80% of participants with poor baseline physical function who took the Wegovy pill showed clinically meaningful improvements in function scores (e.g., range of motion and stamina) compared with around 43% in the placebo group. There were also some head-to-head comparisons of oral Wegovy with orforglipron (Foundayo; Eli Lilly), arguably the most commercially significant element. The ORION indirect treatment comparison showed that oral Wegovy delivered greater mean weight loss than orforglipron 36mg, and that orforglipron was associated with approximately 14 times higher odds of discontinuation due to gastrointestinal side effects. A separate patient preference study (OPTIC) found that 84% of respondents favoured a treatment profile resembling Wegovy pill over that of orforglipron.

What does this mean? First thing to clarify are the weight loss figures provided by Novo Nordisk. The headline 21.6% weight loss figure applies only to the early responder subgroup, which was approximately 29% of patients. Presenting this as the overall result (as the headline suggests) is a little misleading. The overall trial mean was 17% weight loss. The ORION indirect treatment comparison versus Eli Lilly’s Foundayo is the most commercially interesting element of this news, but it requires some caution. An indirect treatment comparison is a statistical method for comparing two drugs that have never been tested head-to-head, using a common comparator arm as a bridge. This is not a randomised trial, and the result (i.e., that orforglipron was associated with approximately 14 times higher odds of stopping due to gastrointestinal side effects) should be treated as hypothesis-generating rather than definitive. The methodology, patient populations, trial designs, and dose levels across the trials being compared may differ in ways that the statistical adjustment cannot fully account for. Moreover, the OPTIC patient preference study showing 84% of survey respondents favouring a profile like oral Wegovy over Foundayo is market research data, not clinical data, and should be treated as such.

Regenxbio toplines results from the Phase III AFFINITY DUCHENNE trial

What happened? Regenxbio has posted positive topline and interim functional data from the pivotal Phase III portion of the Phase II/III AFFINITY DUCHENNE trial of RGX-202, a gene therapy for Duchenne Muscular Dystrophy (DMD). The trial met its primary endpoint with high statistical significance (p<0.0001), with 93% of participants (28/30) reaching at least 10% microdystrophin expression at Week 12 (n=30). Additionally, RGX-202 demonstrated statistically significant correlation between microdystrophin expression and interim functional improvement.

What does this mean? 

Using a sporting analogy, this is a home run for Regenxbio. Over 90% of patients achieving the required microdystrophin expression at Week 12 is a compelling result. However, the primary endpoint is a biomarker endpoint, not a functional clinical endpoint. This is consistent with an accelerated approval pathway, where a surrogate biomarker (which is reasonably likely to predict clinical benefit) can support initial approval. The functional data reported are interim and based on very small numbers: nine patients assessed for functional outcomes so far, with the headline being a statistically significant correlation between microdystrophin expression and functional improvement. The correlation is meaningful, but the functional dataset is too small to draw firm conclusions about clinical magnitude. In terms of how RGX-202 could fit into the competitive landscape, Elevidys (delandistrogene moxeparvovec, Sarepta Therapeutics) is the dominant incumbent. Pfizer's fordadistrogene movaparvovec and Solid Biosciences' SGT-003 are also in clinical development.

Regulatory

BeOne’s Beqalzi gets FDA nod for R/R mantle cell lymphoma

What happened? BeOne Medicines (BeOne) has received FDA accelerated approval for Beqalzi (sonrotoclax), a BCL2 inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL), after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor. Beqalzi was designed to provide greater potency, selectivity, and a pharmacologic profile vs. other BCL2 inhibitors (e.g., venetoclax) with potential to improve efficacy, tolerability, and convenience. Approval was supported by efficacy and safety data from the open-label Phase I/II BGB-11417-201 study. The ORR was 52% (95% CI, 42-62) and the CRR was 16% (95% CI, 9.1-24.0).

What does this mean? Venetoclax (Venclexta; AbbVie) dominates BCL2 inhibition in CLL and AML but has no MCL label. The most relevant comparator in post-BTK relapsed MCL is pirtobrutinib (Jaypirca, Eli Lilly), a non-covalent BTK inhibitor approved by the FDA in January 2023. Pirtobrutinib achieved an ORR of 50% with a median DOR of 8.3 months in its pivotal MCL cohort. Sonrotoclax's 52% ORR is broadly comparable, but its 15.8-month median DOR is notably longer, though cross-trial comparisons should be treated with appropriate caution. Sonrotoclax is also being studied in combination with zanubrutinib (BeOne's BTK inhibitor Brukinsa) in previously untreated CLL, with data expected at ASCO 2026. That combo programme is perhaps more commercially significant. If the sonrotoclax-zanubrutinib doublet demonstrates deep, durable responses including high rates of undetectable minimal residual disease in CLL, it would challenge venetoclax-based combinations in a much larger market than MCL.

BMS’ Sotyktu gets EC nod for adult PsA

What happened? Bristol Myers Squibb (BMS) has announced that the EC has granted approval to Sotyktu (deucravacitinib), alone or in combination with methotrexate, for the treatment of active psoriatic arthritis (PsA) in adults who have had an inadequate response or who have been intolerant to a prior disease-modifying antirheumatic (DMARD) therapy. Sotyktu, a once-daily oral, selective tyrosine kinase 2 (TYK2) inhibitor, is the first TYK2 inhibitor to be approved for the treatment of active PsA in the EU. This EU approval was based on positive results from the Phase III POETYK PsA-1 and POETYK PsA-2 trials, which evaluated the efficacy and safety of Sotyktu 6mg OD in adults with active PsA. In both trials, treatment with Sotyktu resulted in significant improvement in disease activity, as measured by ACR20 (the primary endpoint) and MDA (key secondary endpoint). The overall safety profile of Sotyktu observed in individuals with active psoriatic arthritis was generally consistent with the safety profile in those with plaque psoriasis. 

What does this mean? This label expansion comes after several previous approvals for Sotyktu in Europe and the US. Sotyktu was first approved by the FDA in September 2022 for moderate-to-severe plaque psoriasis. The EC granted the same approval in March 2023. The FDA then approved Sotyktu for active PsA in March 2026. The PsA market is crowded with TNF inhibitors, IL-17 inhibitors, IL-23 inhibitors, and JAK inhibitors, most notably upadacitinib (Rinvoq; AbbVie) and tofacitinib (Xeljanz; Pfizer). The mechanistic distinction of TYK2 inhibition is relevant here. TYK2 sits upstream of IL-12, IL-23, and type I interferon signalling, making it particularly relevant in diseases with a strong IL-23/Th17 axis like PsA. Critically, TYK2 inhibition does not affect JAK1, JAK2, or JAK3, which are the targets implicated in the cardiovascular and thromboembolic safety signals associated with pan-JAK and JAK1/JAK2 inhibitors.

EC approves label expansion for Pfizer’s Hympavzi

What happened? Pfizer has announced that the EC has granted marketing authorisation to expand the approved indication for Hympavzi (marstacimab) to include patients 12 years of age and older weighing at least 35kg with haemophilia A (congenital factor VIII [FVIII] deficiency) with FVIII inhibitors or haemophilia B (congenital factor IX [FIX] deficiency) with FIX inhibitors. Marstacimab offers a combination of superior bleed protection compared to on-demand treatment that is well-tolerated with a straightforward, once-weekly subQ administration that does not require routine treatment-related lab monitoring. This indication extension is based on results from the Phase III BASIS trial. Recall, marstacimab is a first-in-class, non-factor rebalancing agent that reduces the action of TFPI, a protein that prevents blood from clotting, thus helping to increase thrombin generation and promote clotting.

What does this mean? Another important label expansion for Hympavzi having been previously approved in Europe for routine prophylaxis of bleeding episodes in patients 12 years of age and older, weighing at least 35 kg, with haemophilia A without inhibitors, or haemophilia B without inhibitors in November 2024. FDA approval for the expanded indication is pending. The inhibitor population has the highest level of unmet need in haemophilia, historically managed with IV bypassing agents (e.g., activated prothrombin complex concentrate and recombinant FVIIa) which require on-demand administration and produce variable results. Approved in November 2017, emicizumab (Hemlibra; Roche) transformed the haemophilia A with inhibitors landscape with its subQ prophylaxis profile. However, emicizumab is only approved for haemophilia A, not haemophilia B. Hympavzi covers both, giving it a specific commercial advantage in the haemophilia B with inhibitors population where there is no comparably convenient approved prophylactic option.

Partner’s Bizengri gets FDA nod for NRG1 fusion+ cholangiocarcinoma

What happened? Partner Therapeutics (Partner) has announced that the FDA has approved Bizengri (zenocutuzumab) for the treatment of adults with advanced, unresectable or metastatic cholangiocarcinoma harbouring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy. This marks the first targeted therapy approved specifically for NRG1+ cholangiocarcinoma. The approval was expedited by Partner’s receipt of a Commissioner's National Priority Voucher (CNPV). The FDA approved Bizengri based on safety and efficacy data from the Phase II eNRGy trial, a multicenter, open-label, multi-cohort trial in adults with advanced solid tumours harbouring NRG1 gene fusions. A total of 22 patients with unresectable or metastatic NRG1 fusion-positive cholangiocarcinoma were enrolled, with 19 evaluable for efficacy. The major efficacy outcome measures were confirmed overall response rate (ORR), which is the percentage of patients in a clinical trial whose cancer shrinks or disappears after treatment, and duration of response (DOR). The ORR was 36.8% with a DOR range of 2.8 to 12.9 months. The most common adverse reactions (≥20%), excluding laboratory findings, were fatigue, diarrhoea, musculoskeletal pain, abdominal pain, nausea, cough, dyspnoea, and decreased appetite. Bizengri previously received Breakthrough Therapy Designation and Orphan Drug Designation from the FDA for NRG1+ cholangiocarcinoma and was approved for non-small cell lung cancer and pancreatic adenocarcinoma in April 2024.

What does it mean? This is the first targeted therapy approved specifically for NRG1+ cholangiocarcinoma. The approval was expedited by a Commissioner's National Priority Voucher (CNPV), a relatively new regulatory process in the US. NRG1 fusions are mechanistically distinct from more familiar fusion oncogenes such as NTRK, RET, ROS1, ALK, and FGFR. Those fusions create chimeric kinase receptors. NRG1 fusions instead create chimeric ligands. The fused protein binds to HER3, triggering HER2/HER3 heterodimerisation and downstream pathway activation. Zenocutuzumab is a bsAb that simultaneously blocks NRG1 fusion protein binding to HER3 and prevents HER2/HER3 dimerisation, thereby suppressing the oncogenic signalling. Bizengri now holds three approved indications across NRG1+ NSCLC, pancreatic adenocarcinoma, and cholangiocarcinoma.

Commercial

Boehringer Ingelheim licenses preclinical antibody from Immunitas

What happened? Boehringer Ingelheim and Immunitas Therapeutics (Immunitas) have announced a global licensing agreement for a preclinical antibody programme being developed for chronic inflammatory and autoimmune diseases. The programme is designed to selectively target cells that play a central role in driving chronic inflammation, with the goal of achieving sustained disease control for patients who do not respond adequately to current therapies. Unlike therapies that block individual inflammatory signals, the licensed Immunitas antibody program is designed to target pathogenic cells localised at the sites of inflammation. By targeting this subset of pathogenic cells, the approach has the potential to provide deeper and more durable benefit across a range of inflammatory conditions. Under the terms of the agreement, Boehringer Ingelheim will obtain worldwide rights to develop, manufacture and commercialise the programme. Immunitas will receive an upfront payment and is eligible to receive near-term and future development, regulatory, and commercial milestone payments totaling up to €407.5 million, in addition to tiered royalties on future sales. 

What does this mean? Details are thin on the ground, as they usually are deals that include pre-clinical assets, but based on Immunitas’ expertise the antibody in question is likely to act via the CD161-CLEC2D pathway. Immunitas has published work specifically on CD161-high tissue-resident T cell populations as drivers of chronic inflammation, using single-cell transcriptomics to identify these cells as a disease-relevant subset. An antibody targeting the CD161-CLEC2D axis could plausibly modulate or deplete this tissue-enriched population without broadly depleting circulating lymphocytes.

Cellular Intelligence acquires STEM-PD from Novo Nordisk

What happened? Meta CEO Mark Zuckerberg backed-Cellular Intelligence has announced that it has acquired STEM-PD from Novo Nordisk. STEM-PD is a Phase II-ready allogeneic, stem cell-derived therapy, designed to ​replace dopamine-producing nerve cells lost in Parkinson's disease. A Phase I trial was initiated in November 2022. Recall, Novo Nordisk terminated development of STEM-PD when it closed its cell therapy division in October 2025. In connection with the transaction, Novo Nordisk is also making a strategic investment in Cellular Intelligence. Cellular Intelligence said it aims to deploy its AI platform to accelerate the development of the therapy.

What does this mean? STEM-PD’s mechanism is disease-modifying in intent rather than symptomatic. Parkinson's disease involves progressive loss of dopaminergic neurons in the substantia nigra. The STEM-PD approach attempts to replace those lost neurons by transplanting allogeneic stem cell-derived dopaminergic progenitors that engraft and restore striatal dopamine signalling. This is conceptually analogous to the Lund foetal cell transplant work which demonstrated proof of concept for cell replacement but was limited by the impracticality of foetal tissue sourcing. Stem cell-derived allogeneic approaches remove that constraint but introduce manufacturing complexity and immunological challenges.

GSK collaborates with China’s SBP Group on bepirovirsen launch

What happened? GSK has announced an exclusive strategic collaboration with Sino Biopharmaceutical (SBP Group), through its subsidiary Chia Tai Tianqing Pharmaceutical Group (CTTQ), to accelerate the launch of bepirovirsen in mainland China. Bepirovirsen is a potential first-in-class treatment for chronic hepatitis B (CHB) under priority regulatory review in China. Bepirovirsen is an ASO licensed from Ionis Pharmaceuticals. It has a triple mechanism: inhibiting viral DNA replication, suppressing hepatitis B surface antigen (HBsAg) levels, and stimulating immune activation. The goal of treatment is functional cure, defined as undetectable HBsAg and viral DNA for at least six months after stopping treatment, which is associated with reduced long-term risk of cirrhosis and liver cancer. The regulatory submission is supported by the Phase III B-Well 1 and B-Well 2 trials, which demonstrated statistically significant functional cure rates. Under the agreement, CTTQ will be responsible for importation, distribution, hospital access, and promotional and non-promotional activities for bepirovirsen in mainland China. GSK will remain the marketing authorisation holder and retain responsibility for regulatory, quality, pharmacovigilance and global medical strategy. The agreement also grants GSK the ability to review certain early-stage pipeline assets of the SBP Group to evaluate the potential for collaboration opportunities outside China.

What does this mean? The size of the opportunity for bepirovirsen in China is significant. China accounts for approximately 75 million of the more than 250 million people globally living with chronic hepatitis B, and approximately 85% of liver cancer cases in China are associated with hepatitis B infection. Bepirovirsen, if approved, would be positioned as the first functional cure option in a market where patients currently require lifelong antiviral suppression therapy. CTTQ is a logical partner. The company is a dominant commercial player in hepatitis in China, with one of China's most comprehensive liver disease portfolios and access to more than 5,000 medical centres. For a newly launching drug in a complex market, using an established local commercial infrastructure rather than building one from scratch is a sound approach.

Hengrui and BMS announce $15.2 billion, multi-asset R&D collaboration

What happened? Hengrui Pharma (Hengrui) and Bristol Myers Squibb (BMS) have announced a multi-indication R&D collaboration which will advance a portfolio of 13 early stage programmes in oncology, haematology and immunology. The agreements include four oncology/ haematology assets from Hengrui, four immunology assets from BMS, and five innovative assets to be jointly discovered and developed by both companies, leveraging Hengrui's discovery engine and platform technologies across several modalities. Hengrui has the option to co-develop select assets and the potential to conduct certain commercialisation activities globally with BMS. Under the terms of the agreement, BMS will pay Hengrui up to $950 million, including a $600 million upfront payment, a $175 million first anniversary payment, and a second contingent anniversary payment of $175 million in 2028. The potential total value of the agreement is up to approximately $15.2 billion, including the exercise of available options for the joint discovery programmes and the achievement of applicable development, regulatory, and commercial milestones for all programmes.

What does this mean? At up to $15.2 billion in total potential value with $600 million upfront, this is one of the largest China-originated deals ever announced. The upfront alone is nearly 10x the average for China out-licensing deals in early 2026. Data from Pharmacube shows that the average upfront fee amounted to $77.7 million in early 2026, doubling from $38.8 million in 2025 and about three times the level in 2021. Deal structure is also interesting. The deal reciprocal and platform-based rather than asset-specific. BMS is not licensing specific named drugs, it’s entering a portfolio and discovery collaboration where 5/13 programmes will be jointly created using Hengrui's modality platforms. BMS is facing patent expiry pressure on several key products (e.g., Eliquis, Opdivo, Pomalyst) in the coming years and is investing heavily in pipeline renewal. Hengrui's efficiency in early-stage development and its platform capabilities in oncology and immunology provide a cost-effective way to fill that pipeline across multiple modalities and therapy areas simultaneously. But to paraphrase a headline from STAT+, is this another example of Western pharma sowing the seeds of its own destruction by seeking innovation from China?

Pfizer/Arvinas off-load Veppanu to Rigel for up to $405 million

What happened? Rigel Pharmaceuticals (Rigel) has entered an exclusive, global license agreement with Arvinas and Pfizer to develop, manufacture and commercialise Veppanu (vepdegestrant), the first FDA approved oral Proteolysis Targeting Chimera (PROTAC). Veppanu was approved by the FDA in May 2026 for the 2L treatment of adults with ER+/HER2-/ESR1-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test. Approval was granted based on data from the Phase III VERITAC-2 trial. Under the terms of the agreement, Arvinas and Pfizer will receive an upfront payment of $70 million and an additional $15 million upon successful completion of select development and manufacturing transition activities. Arvinas and Pfizer will also be eligible to receive up to $320 million in future development, regulatory, and commercial milestone payments.

What does this mean? No surprise considering both Pfizer and Arvinas announced their intention to out-license Veppanu in September 2025. Rigel is a sound choice as a home for Veppanu due to its experience in haematological and solid tumours (via Rezlidhia for AML and Gavreto for NSCLC and thyroid cancer). Rigel has an existing oncology commercial infrastructure (i.e., MSLs, market access capabilities, and oncologist relationships) that can be redeployed for a breast cancer indication without being built from scratch. Its track record of licensing in and successfully launching acquired assets, explicitly cited by both Arvinas and Rigel, also supports the decision. The NCCN Category 2A designation also gives Rigel an important clinical validation tool at launch. The main question is whether Rigel has the depth of breast cancer relationships to compete effectively against elacestrant (Orserdu), which has a head start in the same ESR1-mutated setting.

In Other News

AstraZeneca expands collaboration with Immunai

AstraZeneca and Immunai have announced an expansion of its collaboration with Immunai eligible to receive up to $37.5 million over 2026-2027. The agreement extends and broadens a multi-year collaboration in oncology clinical development through 2027. Under the expanded agreement, AstraZeneca will continue to leverage Immunai’s AMICA-OS AI operating system, which integrates one of the largest clinical immunology databases at single-cell resolution with advanced foundation AI models of the immune system. The platform is designed to generate clinically relevant insights across oncology drug development, including biomarker discovery, patient stratification, mechanism of action analysis, and dose optimisation.

Chugai files obinutuzumab for INS in Japan

Chugai Pharmaceutical and Nippon Shinyaku have announced that Chugai has filed a regulatory application with Japan’s Ministry of Health, Labour and Welfare (MHLW) for the obinutuzumab (humanized anti-CD20 mAb) for an additional indication of idiopathic nephrotic syndrome (INS). Announced in October 2025, data from the Phase III INShore study was used to support the filing.

Eli Lilly launches donanemab in India

Eli Lilly has launched its Alzheimer’s therapy donanemab under the brand name Lormalzi in India. Lormalzi will be priced at ₹91,688 ($957) for a 350mg vial after India’s Central Drugs Standard Control Organization approved it for patients with mild cognitive impairment or ‌mild ⁠dementia due to Alzheimer's.

FDA approves Taiho’s Inqovi/Venclexta combo in AML

Taiho Pharmaceutical (Taiho) has announced that the FDA approved Inqovi (decitabine and cedazuridine) plus venetoclax (Venclexta; AbbVie) as a treatment for adults with newly diagnosed acute myeloid leukaemia (AML) who are 75 years or older or who are ineligible for intensive induction chemotherapy. Inqovi in combination with venetoclax is the first oral combination treatment regimen approved for this patient population, offering an alternative to parenteral hypomethylating agent–based regimens (e.g., azacitidine or decitabine) that require frequent clinic visits. The approval was supported by results from the Phase II ASCERTAIN-V trial, results for which were presented at ASCO 2025.

FDA delays review of Eisai/Biogen’s Leqembi Iqlik

Eisai and Biogen have announced that the FDA has extended the review period by three months for the sBLA for QW Leqembi Iqlik (lecanemab subQ) as a starting dose for the treatment of early Alzheimer’s disease. The new PDUFA date is 24 August 2026. According to Eisai and Biogen, the FDA has asked for additional information that constitutes an amendment to the marketing application and so requires additional time for review. Recall, the subQ formulation of lecanemab was approved as a maintenance therapy for those who have initially been treated with the original intravenous infusion in August 2025.

Fosun licenses pipeline Alzheimer's disease asset from AriBio

Fosun Pharma has paid $60 million for an option on AriBio’s late-phase Alzheimer’s disease programme, securing the chance to expand its rights to the asset in exchange for a further $80 million. The deal covers AR1001, a once-daily oral phosphodiesterase-5 (PDE5) inhibitor that AriBio is evaluating in a Phase III clinical trial. Viagra and Cialis, both used to treat erectile dysfunction, are the best-known PDE5 inhibitors. Preclinical studies suggest PDE5 inhibition drives neuroprotective effects

Isomorphic Labs secures $2.1 billion Series B funding

Isomorphic Labs has announced it has raised $2.1 billion in Series B funding. This latest round of investment will accelerate the company's evolution from pioneering novel AI models to applying them at scale. The financing round is led by Thrive Capital and includes participation from existing backers Alphabet and GV alongside new investors MGX, Temasek, CapitalG, and the UK Sovereign AI Fund, significantly expanding Isomorphic Labs' global capital base. The new capital will be used for the continued development and deployment of Isomorphic Labs' AI drug design engine (IsoDDE), accelerating and expanding its pipeline of therapeutic programmes towards the clinic.

New AI technology to speed drug development

University of Virginia School of Medicine scientists have developed a new approach to drug development and discovery that could accelerate the creation of new medicines. Scientists have developed a suite of AI-powered tools, called YuelDesign, YuelPocket and YuelBond, that work together to transform how new drugs are created. The centrepiece, YuelDesign, uses a form of AI called diffusion models to design new drug molecules tailored to fit their protein targets exactly, even accounting for the way proteins flex and shift shape during binding. A companion tool, YuelPocket, identifies exactly where on a protein a drug can attach, while YuelBond ensures the chemical bonds in designed molecules are accurate. Together, the approach aims to improve both how new drugs are designed and how quickly and efficiently existing drugs can be evaluated for new purposes.

Oruka licenses Halozyme’s Hypercon for ORKA-001

Halozyme Therapeutics (Halozyme) and Oruka Therapeutics (Oruka) have entered into a global exclusive collaboration and license agreement. Under the agreement, Oruka has licensed Halozyme's Hypercon technology for use with ORKA‑001, in development for psoriasis and related inflammatory diseases, and up to one additional target. Hypercon is an innovative microparticle technology that allows for hyperconcentration of drugs and biologics, reducing injection volume for a given dose and supporting more convenient, patient‑friendly administration.

Reformulated vorinostat enters the clinic for Rett and Pitt Hopkins syndrome

Unravel Biosciences (Unravel) has announced the first study participants have been dosed in Unravel’s RVL-001 clinical trials for Rett syndrome (RTT) and Pitt Hopkins syndrome (PTHS). RVL-001, a proprietary formulation of vorinostat (Zolinza; Merck & Co.), is an oral histone deacetylase (HDAC) inhibitor which will be clinically evaluated for the first time in these indications. In addition to Unravel’s RVL-001 programme for RTT and PTHS, the company has also initiated development work on RVL-002, a first-in-class novel molecule for Rett syndrome. Unravel's proprietary BioNAV drug discovery engine established a dynamic transcriptome network profile, called “Living Molecular Twins,” to identify RVL-001 as a potentially promising therapeutic drug for RTT and PTHS.

UAE approves AstraZeneca’s Baxfendy

The Emirates Drug Establishment (EDE) has approved Baxfendy (baxdrostat) to treat hypertension in patients who are unable to adequately control blood pressure levels through traditional medications. This marks the first approval for Baxfendy, which is a highly selective aldosterone synthase inhibitor. Approval was based on data from the Phase III BaxHTN hypertension trial, data from which was announced in July 2025.

Vyome in-licenses two selective JAK inhibitor assets from Impetis

Vyome Holdings has announced the signing of an agreement with India’s Impetis Biosciences Limited that gives it exclusive license to develop and commercialise a selective JAK 1/3 inhibitor and a selective JAK1 inhibitor.

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.