Pharma Phriday (May 8, 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

• Alloy Therapeutics and IPI collaborate on antibody discovery.

• Waiv partners with Daiichi Sankyo on AI biomarker R&D.

Clinical

• Avalo posts positive mid-stage results for abdakibart in HS.

• Celcuity's Phase III VIKTORIA-1 trial achieves primary endpoint.

• Dazucorilant misses primary endpoint in Phase II ALS trial.

• J&J’s guselkumab/golimumab FDC delivers in Phase IIb.

• Mirum’s Phase IIb VISTAS study in PSC hits primary endpoint.

• Naronapride delivers in Phase IIb gastroparesis trial.

• Reading between the lines of Summit's HARMONi-3 update.

• Viridian’s elegrobart hits bullseye in Phase III chronic TED trial.

Regulatory

• Arvinas’ Veppanu gets FDA nod for ESR1m, ER+/HER2- breast cancer.

• AstraZeneca’s Truqap gets FDA ODAC nod for mHSPC.

• Axsome’s Auvelity gets FDA nod for Alzheimer's related agitation.

Commercial

• Angelini Pharma to acquire Catalyst Pharmaceuticals for $4.1 billion.

• Archimed acquires Esperion for $1.1 billion.

• Bayer to acquire Perfuse Therapeutics for up to $2.45 billion.

• GSK lays down $1 billion on siRNA asset from China’s SiranBio.

• Madrigal licenses PNPLA3 targeting MASH asset from Arrowhead.

• UCB to acquire Candid Therapeutics for $2.2 billion.

In Other News

• Arrowhead’s Redemplo gets Australian TGA nod for FCS.

• AstraZeneca exercises option to license PTX-229 from Pinetree.

• Eli Lilly’s Foundayo hits 5,612 prescriptions in third week.

• J&J drops two CAR-T cell therapy pipeline programmes.

• J&J’s Stelara gets FDA nod for paediatric Crohn’s disease.

• Roche to acquire PathAI for up to $1.05 billion.

• Samsung Bioepis announces Phase I data for SB27.

• Sanofi withdraws Tzield from FDA’s CNPV programme.

• Satellite Bio announces FDA rare paediatric designation for SB-101.

• Takeda announces positive Phase II/III results for TAK-881 in PID.

Summary

• Artificial Intelligence. Two AI partnerships featured this week. Alloy Therapeutics and the Institute for Protein Innovation announced a collaboration to create nanobody libraries for next-generation antibody discovery, targeting complex biological targets in neuroscience. Separately, Daiichi Sankyo entered its fourth AI biomarker collaboration of the year with Waiv, applying computational pathology to an ADC programme.

• Clinical. A broad set of readouts this week. Avalo's Phase II LOTUS trial of abdakibart (anti-IL-1β) met its primary endpoint in HS, validating IL-1β inhibition as an independent pathway in a disease dominated by IL-17 and TNF blockade. Celcuity's Phase III VIKTORIA-1 trial confirmed that comprehensive PAM pathway inhibition with gedatolisib outperforms alpelisib in PIK3CA-mutant advanced breast cancer. Corcept's DAZALS ALS study missed its primary functional endpoint despite an 87% mortality reduction at two years, an unusual dissociation requiring confirmatory Phase III data. Johnson and Johnson's JNJ-4804 co-antibody delivered meaningful improvements in refractory IBD, with the most pronounced benefit in patients who have exhausted prior therapy classes. Viridian's Phase III REVEAL-2 study of elegrobart in chronic thyroid eye disease met its primary endpoint, completing a two-study package for BLA submission in early 2027, and positioning the subcutaneous autoinjector as a compelling convenience advance on intravenous Tepezza. Mirum's VISTAS and Dr. Falk Pharma's MOVE-IT trials both met primary endpoints in PSC and gastroparesis, respectively, two indications with no currently approved treatments.

• Regulatory. Three notable approvals this week. Veppanu (vepdegestrant, Arvinas/Pfizer) became the first PROTAC to receive FDA approval, in ESR1-mutated advanced breast cancer. Auvelity (Axsome) was approved for Alzheimer's-associated agitation, the first non-antipsychotic approved for this indication. And AstraZeneca's Truqap received a favourable ODAC recommendation for PTEN-deficient metastatic hormone-sensitive prostate cancer.

• Commercial. Six deals dominated the week. UCB acquired Candid Therapeutics ($2.2 billion) for a BCMA T-cell engager platform targeting immune reset in autoimmune disease. Angelini Pharma acquired Catalyst Pharmaceuticals ($4.1 billion) for its first US commercial presence in rare neurology. Bayer acquired Perfuse Therapeutics (up to $2.45 billion) for a novel glaucoma asset. GSK committed up to $1 billion to a Chinese-origin ALK7 siRNA. Madrigal licensed ARO-PNPLA3 from Arrowhead as a precision complement to resmetirom in MASH. Archimed took Esperion private for up to $1.1 billion.

• In Other News. Roche agreed to acquire PathAI (up to $1.05 billion). Johnson and Johnson discontinued two CAR-T programmes in large B-cell lymphoma. Stelara received FDA approval for paediatric Crohn's disease. Eli Lilly’s Foundayo prescription growth continues to fascinate. And Sanofi withdraws Tzield from the FDA’s CNPV programme.

Artificial Intelligence

Alloy Therapeutics and IPI collaborate on antibody discovery

What happened? Alloy Therapeutics (Allow) has announced a strategic collaboration with the Institute for Protein Innovation (IPI), a non-profit leader in protein engineering and in vitro discovery. Together, the organisations will create bespoke VHH (nanobody) libraries designed to power next-generation antibody discovery, including bispecific and multispecific therapeutics. The collaboration combines Alloy’s drug discovery and development infrastructure and scalable platform ecosystem with IPI’s expertise in synthetic antibody discovery, high-throughput in vitro platforms, and protein engineering. Through this partnership, Alloy and IPI aim to make advanced discovery tools more widely available, enabling researchers and drug developers to tackle complex biological targets, particularly in areas like neuroscience. 

What does this mean? This complementary collaboration combines Alloy’s in vivo platforms with IPI’s synthetic in vitro discovery expertise to create bespoke nanobody libraries optimised for bispecific and multispecific therapeutic formats. The broader consideration is access and democratisation, which is like other deals in the space (see my previous note on AWS’s foray into drug discovery), as it makes advanced nanobody discovery tools available to smaller biotechs that could not build this infrastructure independently. Alloy’s track record is impressive: over 200 partnerships have generated more than 100 licensed therapeutic programmes, 22 of which have advanced to clinical development. No deal financials were disclosed, which is consistent with IPI’s non-profit structure and suggests a tool-sharing and library-building arrangement rather than a conventional licensing deal.

Waiv partners with Daiichi Sankyo on AI biomarker R&D

What happened? Waiv has announced it has entered a collaboration with Daiichi Sankyo to support digital pathology biomarker discovery for an antibody-drug conjugate (ADC) programme. Under the collaboration, Waiv will apply its end-to-end computational pathology platform to early phase data. This includes tumour microenvironment (TME) analysis across both haematoxylin and eosin (H&E) and immunohistochemistry (IHC) stained samples, as well as biomarker discovery and outcome prediction capabilities aimed at identifying biomarkers of treatment response ahead of next clinical trial phases.

What does this mean? The technical rationale for this deal makes sense. ADC efficacy depends heavily on target expression levels and tumour biology, and patient selection is increasingly a determinant of clinical success in this class. Notably, this is the fourth AI-driven biomarker collaboration Daiichi Sankyo has announced in 2026, following deals with BostonGene, Imagene AI and 4D Path. This multi-platform approach de-risks Daiichi Sankyo’s strategy by enabling multiple parallel AI biomarker programmes across its ADC portfolio, each using a different computational approach.

Clinical

Avalo posts positive mid-stage results for abdakibart in HS

What happened? Avalo Therapeutics (Avalo) has announced positive topline results from its Phase II LOTUS trial evaluating the efficacy and safety of abdakibart (anti-IL-1β mAb) in adults with moderate to severe hidradenitis suppurativa (HS). The trial randomised N=253 adults in a 1:1:1 ratio to receive a 600mg loading dose of abdakibart followed by 300mg every four weeks, a 300mg loading dose followed by 150mg every two weeks, or placebo. The trial’s primary efficacy endpoint was the proportion of patients achieving HiSCR75 at Week 16. The trial successfully met its primary endpoint at both doses studied (p=0.018 150mg, p=0.015 300mg and p=0.004 combined), demonstrating a 42.2% and 42.9% absolute improvement in HiSCR75 response rates at Week 16, respectively (42.5% combined, placebo rate 25.6%). Abdakibart regimens also demonstrated statistically significant benefit across the key secondary endpoints in HiSCR50, change in IHS4 and change in draining tunnel count. Numerically favorable responder rates were observed across all other key secondary endpoints. The HiSCR75 response was similar in patients with and without prior biologic exposure. Across the study, abdakibart was well tolerated. Based on these data, Avalo plans to advance abdakibart into a registrational Phase III programme.

What does this mean? This is good Phase II data for a mechanism that has not previously shown promise in HS. Historically, the HS treatment landscape has been dominated by IL-17 (e.g., secukinumab) and TNF (e.g., adalimumab) blockade as the established baseline. However, IL-1β inhibition has a clear biological rationale in HS, as it sits upstream of several pro-inflammatory cascades, but it has not previously produced a validated clinical result at this scale. The response was consistent in patients with and without prior biologic exposure, which strengthens the case for IL-1β as an independent pathway rather than one that overlaps with other approaches. Once-monthly dosing could be a commercial differentiator, subject to approval. Phase III trials are planned.

Celcuity's Phase III VIKTORIA-1 trial achieves primary endpoint

What happened? Celcuity has announced positive topline results from the PIK3CA mutant cohort of the Phase III VIKTORIA-1 trial evaluating gedatolisib plus fulvestrant (Faslodex; AstraZeneca) with or without palbociclib (Ibrance; Pfizer) in patients with HR+/HER2-/PIK3CA mutant locally advanced or metastatic breast cancer, following progression on or after treatment with a CDK4/6 inhibitor and an aromatase inhibitor. The primary efficacy analysis of gedatolisib combined with fulvestrant and palbociclib (the triplet) demonstrated a statistically significant and clinically meaningful improvement in PFS compared to alpelisib (Piqray; Novartis) and fulvestrant. The secondary endpoint comparing gedatolisib plus fulvestrant (the doublet) versus alpelisib plus fulvestrant, which was not part of the primary efficacy analysis in the hierarchical order, also demonstrated a statistically significant and clinically meaningful improvement in PFS compared to alpelisib and fulvestrant. Both gedatolisib regimens were generally well tolerated, with manageable safety profiles, and no new safety signals.

What does this mean? Gedatolisib is a pan-PI3K and mTORC1/2 inhibitor, which is mechanistically distinct from alpelisib (Piqray; Novartis), the currently approved PI3Kα-selective inhibitor in this setting. The core argument is that inhibiting the entire PAM pathway comprehensively prevents the adaptive resistance cross-activation that allows tumours to escape single-target inhibitors. VIKTORIA-1 now provides Phase III evidence in both PIK3CA mutant and wild-type cohorts that this broader inhibition translates into meaningfully better outcomes than alpelisib plus fulvestrant.

Dazucorilant misses primary endpoint in Phase II ALS trial

What happened? Corcept Therapeutics (Corcept) has announced that the Phase II DAZALS study of its selective cortisol modulator dazucorilant in patients with amyotrophic lateral sclerosis (ALS) has missed its primary endpoint. DAZAL’s primary endpoint was the difference in function, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), between patients who received dazucorilant and those who received placebo. There were improvements in overall survival, a secondary endpoint. After two years following the start of treatment, patients who received 300mg dazucorilant experienced an 87% reduction in the risk of death compared to patients who received placebo and did not switch to 300mg of dazucorilant in the extension phase (hazard ratio: 0.13; p-value: < 0.0001).

What does this mean? The headline two-year OS figure is impressive and traces back to a survival difference first observed at 24 weeks, when zero deaths were recorded in the 83-patient 300mg arm vs five deaths in the 82-patient placebo group. The benefit has now been sustained through two years of follow-up. However, despite the OS benefit, DAZALS missed its primary endpoint (i.e., functional decline on the ALSFRS-R scale). Survival benefits observed only in Phase II secondary endpoints should be interpreted cautiously and require confirmation in Phase III. The fact that the drug did not slow functional decline while apparently reducing mortality is also an unusual pattern. One potential explanation is that dazucorilant reduces the risk of acute events rather than slowing the underlying neurodegenerative process. If elevated cortisol in ALS patients drives systemic complications (e.g., immune dysregulation, cardiovascular stress, metabolic deterioration), then blocking the glucocorticoid receptor might reduce sudden death risk without meaningfully altering the rate of motor neurone loss. In effect, patients may live longer but not function better. It is also worth asking whether the ALSFRS-R is sensitive enough to detect a functional benefit over a 24-week window in a 249-patient study, particularly if the drug’s effect is concentrated in a subgroup with high cortisol levels. A properly powered Phase III trial with pre-specified survival and functional endpoints is needed for confirmation.

J&J’s guselkumab/golimumab FDC delivers in Phase IIb

What happened? Johnson & Johnson has announced Phase IIb data from two studies evaluating JNJ-4804, a fixed-dose combination of guselkumab (Tremfya; anti-IL-23) and golimumab (Simponi; anti-TNF), in patients with moderately to severely active ulcerative colitis (UC) and Crohn’s disease (CD) that is refractory to systemic therapies. In the DUET-CD study, JNJ-4804 demonstrated higher clinical remission rates (50.8%) and endoscopic response rates (38.1%) versus golimumab (25.4% for clinical remission, 19.8% for endoscopic response) at Week 48. The JNJ-4804 rates were also numerically higher than the rates achieved with guselkumab (42.5% for clinical remission and 33.9% for endoscopic response). In the DUET-UC study, JNJ-4804 demonstrated superior clinical remission rates compared to golimumab, with 41.0% of JNJ-4804-treated patients achieving clinical remission at Week 48 versus golimumab (11.5%), and numerically higher rates than guselkumab (34.0%). In both studies, safety findings were generally consistent with the known profiles of the component monotherapies.

What does this mean? The rationale for J&J’s lifecycle management strategy is that IL-23 and TNF drive partially independent inflammatory pathways in IBD, so blocking both simultaneously produces synergistic suppression that neither monotherapy can achieve. What’s most interesting is that the benefit is most pronounced in the refractory subpopulation. In Crohn's disease, clinical remission rates for JNJ-4804 were nearly double the highest comparator at Week 48, and endoscopic response rates were more than 60% higher than the closest monotherapy. In UC, clinical remission was almost 60% higher than the closest comparator in the highly refractory group. These are patients who have failed two or more prior therapy classes, a population where current treatment options are genuinely limited and outcomes with further monotherapy are poor. By positioning the combination in a higher-need, higher-value patient segment where newer entrants like risankizumab and upadacitinib are competing could pay dividends for J&J. Phase III DUET ENCORE trials in both UC and CD are now planned. One caveat is that guselkumab and golimumab individually are not the strongest available benchmarks. Head-to-head data against upadacitinib or vedolizumab in this refractory population would be more informative. The design of the Phase III DUET ENCORE trials in both UC and CD will be eagerly awaited.

Mirum’s Phase IIb VISTAS study in PSC hits primary endpoint

What happened? Mirum Pharmaceuticals (Mirum) has announced the primary endpoint was met in the Phase IIb VISTAS study which evaluated volixibat (IBAT inhibitor) in patients with primary sclerosing cholangitis (PSC). In the primary analysis cohort, treatment with volixibat demonstrated a robust 2.72 point improvement in pruritus and a placebo-adjusted difference of 1.64 points in the primary endpoint (p<0.0001), as measured by the Adult ItchRO scale, reflecting change from baseline to the average of the last 12 weeks of treatment. Statistically significant improvements in pruritus were observed within two weeks of treatment and were also observed in the secondary cohort of patients with mild itch at baseline. Volixibat’s safety profile was generally consistent with the known effects of IBAT inhibition, characterised primarily by gastrointestinal adverse events and elevations in liver laboratory parameters, including alanine aminotransferase (ALT) and bilirubin.

What does this mean? PSC has no approved therapies for cholestatic pruritus or for the underlying disease, making this a genuine unmet need. Mirum has now reported positive Phase IIb results with volixibat in both PSC (VISTAS) and PBC (VANTAGE; met primary endpoint in 2024), positioning it as a platform IBAT inhibitor across cholestatic liver diseases. Mirum already markets maralixibat (Livmarli) in paediatric cholestatic conditions, so volixibat in adult PSC and PBC would represent a meaningful expansion into a larger patient population with the same mechanistic approach.

Naronapride delivers in Phase IIb gastroparesis trial

What happened? Dr. Falk Pharma (Dr. Falk) and Renexxion have announced positive results from the Phase IIb MOVE-IT trial which evaluated the efficacy, safety, and tolerability of naronapride in adults with gastroparesis. MOVE-IT met the primary endpoint, demonstrating statistically significant improvement versus placebo in the ANMS GCSI-DD Core Symptom Score in the 20mg TID and 40mg TID groups. Consistent with previous studies, the safety and tolerability profiles were favorable, with no new safety signals identified versus placebo, including cardiac, neuropsychiatric, or prolactin-related signals. To date, naronapride has been studied in over 1,200 subjects, with a safety profile reflecting its minimal systemic absorption.

What does this mean? Naronapride is an oral, minimally absorbed prokinetic with a dual mechanism (5-HT4 receptor agonism and dopamine D2 receptor antagonism), both of which act on the luminal surface of the gut wall. With safety issues well documented for other prokinetics (e.g., metoclopramide, cisapride, etc.) the field has been waiting for a prokinetic that works peripherally without systemic CNS or cardiac exposure. Enter naronapride. Data from the MOVE-IT trial suggest a clean safety profile: no cardiac, neuropsychiatric or prolactin-related safety signals were observed across a dataset now extending to over 1,200 subjects. However, the absolute improvement over placebo, while statistically significant, is relatively modest on the scale used. Phase III trials will be needed to confirm the benefit.

Reading between the lines of Summit's HARMONi-3 update

What happened? In its Q1’26 earnings statement, Summit Therapeutics (Summit) gave an update on the planned HARMONi-3 squamous cohort interim PFS analysis. Recall, HARMONi-3 is a Phase III trial assessing ivonescimab (anti-PD-1/VEGF bsAb) combined with chemotherapy in patients with 1L metastatic NSCLC, with two distinct cohorts to be analysed separately; squamous and non-squamous tumours. Summit disclosed that the Independent Data Monitoring Committee (IDMC) recommended the study continue as planned, with no safety concerns and no change to the planned final progression-free survival (PFS) analysis in H2’26.

What does this mean? Soon after the news, Summit’s stock was down 25%, potentially reflecting the market’s concern that the early PFS look did not produce a strong enough efficacy signal to justify earlier FDA engagement or investor disclosure. Ivonescimab has historically worked early and fast in prior datasets, including HARMONi-2, where PFS separated meaningfully at a median follow-up of 8.67 months, with mPFS of 11.14 months vs. 5.82 months for pembrolizumab. Through that lens, the absence of a stronger interim signal in HARMONi-3 suggests the H2’26 final squamous readout will not be as compelling as previous studies.

Viridian’s elegrobart hits bullseye in Phase III chronic TED trial

What happened? Viridian Therapeutics (Viridian) has announced positive topline data from the Phase III REVEAL‑2 trial of elegrobart in patients with chronic thyroid eye disease (TED). Elegrobart is a subcutaneously delivered, half‑life‑extended anti-IGFR-1 mAb. REVEAL‑2 enrolled 204 patients, randomised 1:1:1 to elegrobart Q4W (n=70), elegrobart Q8W (n=68), and placebo (n=66). REVEAL-2 met its primary endpoint for both the FDA and EMA with high statistical significance. In addition, REVEAL-2 met all its proptosis key secondary endpoints in the Q4W and Q8W treatment arms with high statistical significance, and the Q4W treatment arm showed a statistically significant diplopia responder rate at week 24. Efficacy was generally consistent regardless of baseline Clinical Activity Score (CAS). Elegrobart was generally well tolerated in REVEAL‑2 with a safety profile consisting of adverse events generally expected from the anti-IGF-1R class, the vast majority of which were mild. Viridian remains on track to submit a BLA to the FDA for elegrobart in Q1’27.

What does this mean? The headline data from REVEAL-2 are clean and replicate the pattern seen in REVEAL-1 for active TED earlier in March 2026. Viridian now has two positive Phase III trials across both the active and chronic TED populations, supporting a BLA submission anticipated in the Q1’27. Elegrobart targets IGF-1R, which is the same MOA as teprotumumab (Tepezza, Amgen/Horizon), the only currently approved treatment for TED. The key differentiation is the delivery format: Tepezza IV requires eight intravenous infusions administered in a clinical setting over approximately five months, which creates a significant access and convenience burden. Elegrobart is dosed subQ via an autoinjector and is designed for at-home self-administration in as few as three doses. With the proptosis response rates from the REVEAL trials broadly comparable to Tepezza's pivotal data, Viridian is offering similar efficacy with dramatically greater convenience. To compete against elegrobart, Amgen is developing a subQ version of Tepezza, and Phase III data were announced in April 2026.

Regulatory

Arvinas’ Veppanu gets FDA nod for ESR1m, ER+/HER2- breast cancer

What happened? Arvinas and partner Pfizer have announced that the FDA has granted approval for Veppanu (vepdegestrant) for the treatment of adults with oestrogen receptor-positive (ER+)/human epidermal growth factor receptor 2-negative (HER2-), oestrogen receptor 1 (ESR1)-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy. This approval marks the first time the FDA has approved a Proteolysis Targeting Chimera (PROTAC), a type of heterobifunctional protein degrader therapy. FDA approval was granted based on data from the Phase III VERITAC-2 trial which evaluated vepdegestrant versus fulvestrant (Faslodex; AstraZeneca). In the trial, among patients with an ESR1 mutation (n=270), vepdegestrant demonstrated a statistically significant and clinically meaningful improvement in PFS, reducing the risk of disease progression or death by 43% compared to fulvestrant. Median PFS was 5 months in the vepdegestrant arm and 2.1 months in the fulvestrant arm.

What does this mean? This is one of the most significant approvals of the week, and arguably of the year, for reasons that extend well beyond breast cancer. Vepdegestrant is the first PROTAC to receive FDA approval, which is a landmark moment for an entirely new class of medicines that has been in development for over two decades. Click here for an excellent overview of PROTACs. More broadly, this approval validates PROTAC technology as a viable regulatory and commercial pathway after years of preclinical promise, and the hope is that it will accelerate investment and development across the many PROTAC programmes targeting oncology, neurodegeneration, and other indications in the wider pipeline (e.g., BMS-986365 in mCRPC, ARV-102 in Parkinson’s disease, GT-20029 in androgenetic alopecia).

AstraZeneca’s Truqap gets FDA ODAC nod for mHSPC

What happened? AstraZeneca has announced that the FDA’s Oncologic Drugs Advisory Committee (ODAC) has recognised a favourable benefit risk profile for Truqap (capivasertib) in combination with abiraterone and androgen deprivation therapy (ADT) for the treatment of patients with PTEN-deficient metastatic hormone-sensitive prostate cancer (mHSPC). Data from the Phase III CAPItello-281 trial was used to support the decision.

What does it mean? The ODAC voted 7-1 with one abstention in favour of a favourable benefit-risk profile for capivasertib plus abiraterone and ADT in PTEN-deficient metastatic hormone-sensitive prostate cancer. That vote reflects the genuine unmet need in this patient population where PTEN deficiency, present in approximately 25% of mHSPC patients, is associated with PI3K/AKT pathway dysregulation, aggressive disease, and poor outcomes with standard of care. The efficacy data from the CAPItello-281 trial are encouraging. The 19% reduction in risk of radiographic progression or death corresponds to a median rPFS improvement of 7.5 months (33.2 vs. 25.7 months), which is clinically meaningful given the poor prognosis of this subgroup. Key secondary endpoints including time to castration resistance and PSA progression also favoured capivasertib. The OS data remain immature, which is the main gap in the evidence package and will be watched closely. In terms of safety, grade 3 or higher adverse events occurred in 67% of the capivasertib arm versus 40% in the comparator arm, with rash, hyperglycaemia, hypokalaemia, and diarrhoea as the most common serious events. This is a meaningful tolerability burden, and how it plays out in real-world use is likely to influence adoption.

Axsome’s Auvelity gets FDA nod for Alzheimer's related agitation

What happened? Axsome Therapeutics (Axsome) has announced that the FDA has approved Auvelity (dextromethorphan HBr and bupropion HCl) for the treatment of agitation associated with dementia due to Alzheimer’s disease. Auvelity is a first-in-class treatment for Alzheimer’s disease agitation which targets the N-methyl D-aspartate (NMDA) and sigma-1 receptors. The approval was based on data from two Phase III trials. ADVANCE-1 was a 5-week trial in which participants received either Auvelity or a placebo. The primary endpoint was the change from baseline to week five in the total score of the Cohen-Mansfield Agitation Inventory (CMAI), a survey that assesses the frequency of manifestations of agitated behaviours in elderly patients, based on caregiver reports. Auvelity was significantly superior to placebo in The Cohen-Mansfield Agitation Inventory score improvements. ACCORD-2 was a withdrawal study in participants who responded to Auvelity. Upon reaching a sustained clinical response to Auvelity, patients were randomly assigned to continue treatment with Auvelity or switch to placebo. The primary endpoint was time to relapse. Participants who continued Auvelity treatment had a significantly longer time to relapse of agitation symptoms compared to patients receiving the placebo. The most common side effects include dizziness, upset stomach, headache, diarrhoea, drowsiness, dry mouth, sexual dysfunction, and uncontrolled sweating.

What does this mean? Agitation affects up to 50% of people with Alzheimer's and is one of the most distressing symptoms for patients, families, and care staff. The current management has relied heavily on atypical antipsychotics used off-label (e.g., quetiapine, risperidone, olanzapine), all of which carry FDA black box warnings for increased mortality in elderly patients with dementia-related psychosis. The safety and tolerability profile of Auvelity in the ADVANCE-1 and ACCORD-2 trials was notably clean, with a treatment discontinuation rate of 1.3%, comparable to placebo. With Auvelity already generating strong sales in MDD (just over $500 million in 2025), the Alzheimer's agitation label extension opens a significantly larger addressable population: over seven million Americans are living with Alzheimer's, the majority of whom will experience agitation at some stage.

Commercial

Angelini Pharma to acquire Catalyst Pharmaceuticals for $4.1 billion

What happened? Angelini Pharma (Angelini) and Catalyst Pharmaceuticals (Catalyst) have announced that they have entered into a definitive agreement pursuant to which Angelini has agreed to acquire Catalent for $4.1 billion. Following the completion of the acquisition, Angelini will take control of Catalyst’s portfolio, which includes products like Firsdapse (amifampridine), FDA approved for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients aged six years and older; Agamree (vamorolone), FDA approved for the treatment of Duchenne Muscular Dystrophy (DMD) in patients aged 2 and older; and Fycompa (perampanel), acquired from Eisai in December 2022, and approved for the treatment of partial-onset seizures and primary generalised tonic-clonic seizures.

What does this mean? For Angelini, a privately held, family-owned Italian company with over a century of history and a CNS and rare disease focus, this represents its largest acquisition to date and its first significant US commercial presence. The strategy will be to integrate Catalyst's portfolio and US infrastructure with Angelini's existing CNS pipeline, including assets from its January 2021 acquisition of Arvelle Therapeutics. Gaining a pre-built US commercial organisation in rare neurology rather than building one from scratch is a sensible use of capital for a company at this stage of internationalisation. For the broader rare disease competitive landscape, the deal adds another well-capitalised owner to the neuromuscular disease space, where Sarepta, Biogen, and argenx are already active. The breadth of Catalyst’s portfolio ensures immediate diversification rather than dependence on a single asset.

Archimed acquires Esperion for $1.1 billion

What happened? Healthcare-focused investment firm Archimed has announced that it has entered into a definitive agreement to acquire Esperion for up to $1.1 billion, assuming full achievement of certain commercial-based milestones. Founded in 2008, Esperion is a commercial-stage biopharmaceutical company that markets non-statin, oral, once-daily cardiovascular medicines focused on lowering LDL-C, including Nexletol (bempedoic acid) and Nexlizet (bempedoic acid and ezetimibe). In March 2026, Esperion acquired Corstasis Therapeutics and gained access to Enbumyst (bumetanide), a nasal spray diuretic for oedema.

What does this mean? While a total equity value of up to $1.1 billion sounds impressive, Esperion's share price had been severely depressed relative to the commercial potential of its assets, reflecting years of investor frustration with the bempedoic acid commercial trajectory. Esperion’s inability to translate positive data from the CLEAR Outcomes trial into commercial uptake in a market dominated by generic statins and increasingly by PCSK9 inhibitors has weighed on the company’s share price. Perhaps ownership by Archimed is what Esperion needs to deliver commercial success?

Bayer to acquire Perfuse Therapeutics for up to $2.45 billion

What happened? Bayer and Perfuse Therapeutics (Perfuse) have announced an agreement under which Bayer will fully acquire Perfuse for a potential value of $2.45 billion. With this acquisition, Bayer will hold the full rights pertaining to PER-001, a small molecule endothelin receptor antagonist (ERA) currently in Phase II clinical development for the treatment of glaucoma and diabetic retinopathy (DR). Under the terms of the agreement, the transaction carries a total potential value of up to $2.45 billion, comprising a $300 million upfront payment and additional development, regulatory, and commercial milestone payments based on success criteria.

What does this mean? The asset driving most of the value of this deal is PER-001, a small molecule ERA delivered via an intravitreal bio-erodible implant. Endothelin is one of the most potent vasoconstrictors in the human body and is upregulated in glaucoma, diabetic retinopathy, AMD and RVO. Existing glaucoma treatments (e.g., prostaglandin analogues, beta blockers, carbonic anhydrase inhibitors) almost exclusively target intraocular pressure reduction and don’t address the ischaemic and neurodegenerative components of the disease. Blocking the endothelin receptor has potential neuroprotective and vasodilatory effects that are independent of pressure lowering, a mechanistically distinct approach to glaucoma treatment. The sustained-release intravitreal implant delivery format is practical for an indication where patient compliance with eye drops is notoriously poor. Bayer has existing ophthalmology infrastructure and pipeline, including Eylea (aflibercept) in wet AMD and diabetic macular oedema, giving PER-001 a natural commercial home if it reaches approval. The usual caveats apply. This is a Phase II asset targeting diseases where clinical endpoints are complex and long-duration trials are typically required to demonstrate disease modification.

GSK lays down $1 billion on siRNA asset from China’s SiranBio

What happened? Suzhou Siran Biotechnology (SiranBio) has announced a worldwide exclusive license agreement with GSK (excluding mainland China, Hong Kong, Macau and Taiwan) for SA030, a long-acting siRNA oligonucleotide in Phase I trials as a potential treatment of metabolic and vascular disease. SA030 targets activin receptor-like kinase 7 (ALK7), a promising therapeutic target for obesity that regulates fat accumulation. Inhibition of ALK7 has been shown to reduce fat mass and improve metabolism. Preclinical studies indicate that blocking ALK7 promotes fat utilization, boosts energy expenditure, and reverses diet-induced obesity in mice. Under the terms of the deal, GSK will pay an upfront fee as well as potential future success-based development, regulatory and commercial milestones payments up to $1 billion, and tiered royalties on global product net sales (excluding mainland China, Hong Kong, Macau and Taiwan). SiranBio will lead the clinical development of SA030 through the completion of Phase I, after which GSK will assume responsibility for development, regulatory filings, and commercialisation outside of mainland China, Hong Kong, Macau and Taiwan.

What does this mean? ALK7 is a receptor involved in the signalling pathway that regulates fat storage in adipocytes. Inhibiting it via siRNA reduces abdominal fat accumulation, and SiranBio's preclinical data suggest that SA030, delivered directly to fat cells, reduces visceral adiposity and in turn improves insulin sensitivity, blood lipid profiles, and adipocyte-driven inflammation. This positions SA030 as a complementary approach to GLP-1 receptor agonists, as it addresses the inflammatory and metabolic consequences of visceral fat rather than simply reducing appetite and body weight. For GSK, this is the third siRNA deal with a Chinese biotech, following deals with Frontier Biotechnologies and Empirico. The pattern reflects GSK's desire to build out an oligonucleotide therapeutics portfolio across respiratory, immunology, and now cardiometabolic disease, complementing its November 2021 collaboration with Arrowhead in MASH and assets acquired from Wave Life Sciences in December 2022. ALK7 is also being pursued by Arrowhead with ARO-ALK7. More broadly, Chinese biotech innovation continues to attract major Western pharma licensing deals. Recent deals include AbbVie/Haisco (pain), AbbVie/RemeGen (oncology), AstraZeneca/CSPC (metabolic), Boehringer Ingelheim/Simcere (IBD), Eli Lilly/Innovent (oncology, immunology) and Pfizer/3SBio (oncology), to name only a few.

Madrigal licenses PNPLA3 targeting MASH asset from Arrowhead

What happened? Madrigal Pharmaceuticals (Madrigal) has announced an exclusive global license agreement with Arrowhead Pharmaceuticals (Arrowhead) for ARO-PNPLA3, a clinical-stage, siRNA asset targeting patatin-like phospholipase domain-containing protein 3 (PNPLA3) which is in development as a potential treatment for metabolic dysfunction-associated steatohepatitis (MASH). PNPLA3 I148M is a well-established genetic contributor to MASH progression, and is associated with increased liver fat, inflammation, fibrosis, cirrhosis and hepatocellular carcinoma. Approximately 30% of patients with MASH with moderate to advanced fibrosis (F2/F3 fibrosis) carry two identical copies of this variant (known as homozygous patients), and it is highly prevalent in Hispanic populations. Phase I data for ARO-PNPLA3, published in the NEJM in July 2024, showed reductions in liver fat up to 46% (as measured by MRI-PDFF) at 12 weeks following a single dose at the highest dose level tested in PNPLA3 I148M homozygous patients. Under the terms of the deal, Madrigal will make a $25 million upfront payment to Arrowhead. Arrowhead is also eligible to receive development, regulatory, and sales milestone payments of up to $975 million. Arrowhead is further eligible to receive tiered royalties on commercial sales ranging from high-single digits to the mid-teens.

What does this mean? J&J previously held this asset and returned it to Arrowhead in February 2023. Why? Were there clinical, commercial, or strategic concerns that informed that decision? Madrigal's rationale, that ARO-PNPLA3 fits specifically as a precision complement to resmetirom in a genetically defined subgroup, is a plausible answer to that question, given that J&J lacked a foundational MASH asset to combine it with. But curious, all the same.

UCB to acquire Candid Therapeutics for $2.2 billion

What happened? UCB has announced the signing of a definitive agreement under which it would acquire Candid Therapeutics (Candid). Cizutamig, Candid’s lead investigational asset, is a BCMA TCE in development for autoimmune diseases. It’s a bsAb directed to BCMA on plasma cells and CD3 on T-cells, enabling T-cell–mediated cytotoxicity against BCMA-expressing plasma cells and B-cells. Cizutamig has been clinically evaluated in over 100 patients with multiple myeloma and autoimmune diseases and is currently in multiple clinical studies in over 10 autoimmune indications. In addition to cizutamig, Candid is developing a pipeline of multi-specific TCE antibodies designed to enable targeted depletion of pathogenic B cell populations in immune mediated diseases to achieve immune reset. Under the terms of the agreement, UCB will pay $2 billion upfront and up to $200 million in potential future milestone payments.

What does this mean? The mechanistic rationale for cizutamig is the "immune reset" concept that has been gaining significant traction recently, particularly following the striking remission data from CAR-T therapy in refractory autoimmune diseases at centres in Germany and elsewhere. The idea is that deep depletion of pathogenic plasma cell and B-cell populations (i.e., the cells producing the autoantibodies driving conditions like lupus, myasthenia gravis, and systemic sclerosis) can induce prolonged, drug-free remission rather than simply controlling symptoms. T-cell engagers targeting BCMA offer a potentially more accessible and scalable route to that same goal than CAR-T, which requires complex manufacturing and carries significant toxicity. This acquisition, and UCB’s March 2026 deal with Antengene, forms part of a platform-driven B-cell targeting strategy, with UCB wanting to build complementary coverage across B-cell mechanisms rather than betting on a single approach. One thing to note is the breadth of indications being studied simultaneously (10+) suggests Candid is running an indication-finding strategy to identify where the benefit-risk profile is most compelling before committing to Phase III.

In Other News

Arrowhead’s Redemplo gets Australian TGA nod for FCS

Arrowhead Pharmaceuticals (Arrowhead) has announced that the Australian Therapeutic Goods Administration (TGA) has approved Redemplo (plozasiran), a siRNA medicine, as an adjunct to diet to reduce triglyceride levels for adult patients with familial chylomicronaemia syndrome (FCS) for whom standard triglyceride lowering therapies have been inadequate.

AstraZeneca exercises option to license PTX-229 from Pinetree

AstraZeneca and Pinetree Therapeutics (Pinetree) have announced that AstraZeneca has exercised its option under the companies' previously announced agreement to obtain an exclusive global license to develop and commercialize PTX-299, a first-in-class bsAb degrader targeting EGFR. The option exercise follows encouraging preclinical progress and represents an important milestone in the collaboration between the two companies. Under the terms of the agreement, AstraZeneca will assume responsibility for global development and commercialization of the therapeutic candidate.

Eli Lilly’s Foundayo hits 5,612 prescriptions in third week

Citing IQVIA data, analysts have reported that Eli Lilly's newly launched Foundayo (orforglipron) was prescribed ~5,600 times in the US in the third week after its launch. This compares with ~3,700 in its second week, and ~26,100 in the third week for oral Wegovy (semaglutide).

J&J drops two CAR-T cell therapy pipeline programmes

Johnson & Johnson (J&J) has announced that it has terminated development of two CAR-T cell therapy pipeline candidates. The investigational CD20 mono CAR-T cell therapy (JNJ-9530) and CD19-CD20 bi-CAR-T (JNJ-4496) programmes will be discontinued. In its PR, J&J said that this is “a strategic business decision based on the Company’s portfolio priorities and an assessment of the evolving large B-cell lymphoma treatment landscape.” Recall, J&J licensed rights to both assets from Cellular Biomedicine Group in May 2023.

J&J’s Stelara gets FDA nod for paediatric Crohn’s disease

Johnson & Johnson has announced that the FDA has approved Stelara (ustekinumab) for the treatment of patients two years and older with moderately to severely active Crohn’s disease (CD). Stelara is the only FDA-approved IL-12/23 antagonist and the first non-TNF biologic to treat adult and paediatric patients two years and older with moderately to severely active Crohn’s disease (CD). The approval is based on data from the Phase III UNITI-Jr clinical study, a multicenter interventional study to evaluate the efficacy, safety, and PK of Stelara for the treatment of paediatric CD over 52 weeks (8 weeks of induction and 44 weeks of maintenance).

Roche to acquire PathAI for up to $1.05 billion

Roche has announced today that it has entered into a definitive merger agreement to acquire PathAI, a company focused on digital pathology and AI-powered technology for pathology laboratories and the biopharma industry. This acquisition builds on the successful partnership between Roche and PathAI, established in October 2021 and scaled up in February 2024 to include the development of AI-enabled companion diagnostic algorithms. The acquisition will strengthen Roche’s position in digital pathology.

Samsung Bioepis announces Phase I data for SB27

Samsung Bioepis has announced that a Phase I study of SB27, a proposed biosimilar to pembrolizumab (Keytruda; Merck & Co.), has met its primary PK endpoints. The randomised, double-blind, three-arm, parallel group, multicenter clinical trial demonstrated PK bioequivalence of SB27 to the reference product Keytruda.

Sanofi withdraws Tzield from FDA’s CNPV programme

According to Fierce Pharma, via STAT, Sanofi has asked that an application for the use of Tzield in children aged 8 and older with stage 3 Type 1 diabetes be removed from the Commissioner’s National Priority Voucher (CNPV) fast-track programme. The CNPV programme was set up to shorten the review process for products that align with national interests from the 10-12 months typically required for a standard review to just 4-8 weeks. Recall, Tzield is already approved to delay the onset of stage 3 Type 1 diabetes in adults, children ages 8 and older whose disease is at stage 2 and children at least 1 year old.

Satellite Bio announces FDA rare paediatric designation for SB-101

Satellite Biosciences (Satellite) has announced that the FDA has granted Rare Paediatric Disease (RPD) designation for SB-101 for the treatment of urea cycle disorders (UCDs). UCDs are a class of devastating conditions in which serious or life-threatening manifestations, such as neurocognitive deficits, behavioural impairment, encephalopathy, coma, seizures, organ failure, and mortality, primarily affect individuals aged from birth to 18 years.

Takeda announces positive Phase II/III results for TAK-881 in PID

Takeda has announced that the Phase II/III TAK-881-3001 trial in patients with Primary Immunodeficiency Disease (PID) has met its primary endpoint. It demonstrated PK comparability between TAK-881 (Immune Globulin Subcutaneous (Human), 20% Solution (SCIG 20%) with Recombinant Human Hyaluronidase]) and Hyqvia [Immune Globulin Infusion (Human) 10% with Recombinant Human Hyaluronidase]. Additionally, secondary endpoints showed that TAK-881 demonstrated safety, efficacy and tolerability profiles comparable to Hyqvia, an established SCIG 10% facilitated with hyaluronidase. These findings support the potential of TAK-881 to deliver the required immunoglobulin (IG) dose for PID patients in half the volume of Hyqvia, reducing infusion duration while maintaining flexible, up to once-monthly dosing for patients (every three or four weeks for PID).

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.