Signals
51Thyroid cancer targeted therapy expands across differentiated, medullary, and anaplastic subtypes
RET inhibitor maturity, NTRK fusion-targeted use, BRAF V600E and MEK combinations in anaplastic disease, and emerging mechanism programs are restructuring thyroid cancer therapy.
AL amyloidosis therapy reshapes around daratumumab and emerging amyloid-targeted programs
Daratumumab plus bortezomib first-line, isatuximab follow-on programs, and emerging anti-amyloid antibody therapy are restructuring AL amyloidosis management.
Treatment-resistant schizophrenia care formalises around clozapine integration
Clozapine pathway formalisation, KarXT muscarinic mechanism integration, and emerging mechanism-targeted programs are reshaping treatment-resistant schizophrenia.
IgG4-related disease enters real prescribing territory
Inebilizumab IgG4-RD pivotal data, rituximab maintenance protocols, and structured diagnostic pathways are reshaping IgG4-related disease management.
Frontotemporal dementia therapy programs reach late-stage trials
Progranulin-replacement therapy in GRN-mutation FTD, ASO programs in C9orf72 FTD-ALS, and tau-targeted programs are emerging in a previously bare category.
Systemic sclerosis therapy options mature past hematopoietic stem cell transplant
Tocilizumab and nintedanib in SSc-ILD, anifrolumab pivotal data, and emerging mechanism-targeted programs are reshaping systemic sclerosis management.
Osteogenesis imperfecta therapy reshapes around anti-sclerostin programs
Setrusumab pivotal data, bisphosphonate maturity, and emerging gene therapy programs are reshaping osteogenesis imperfecta management.
Achondroplasia therapy options widen past vosoritide
Vosoritide commercial maturity and follow-on CNP analogue and FGFR3-targeted programs are restructuring achondroplasia management.
Hypertrophic cardiomyopathy myosin modulator class matures
Mavacamten commercial maturity, aficamten approval and pivotal data, and emerging follow-on cardiac myosin modulator programs are restructuring obstructive HCM management.
Severe COPD biologic therapy class emerges past the inhaler era
Dupilumab COPD approval in eosinophilic phenotype, mepolizumab and benralizumab COPD pivotal data, and ensifentrine entry are reshaping severe COPD management.
Congenital adrenal hyperplasia therapy reshapes around CRF1 receptor antagonism
Crinecerfont approval, follow-on CRF1 receptor antagonist programs, and structured care infrastructure are reshaping classic congenital adrenal hyperplasia management.
Cholangiocarcinoma targeted therapy widens past chemotherapy
FGFR2 inhibitor maturity, IDH1 inhibitor use, HER2-targeted therapy entry, and IO combinations are restructuring biliary tract cancer management.
Giant cell arteritis therapy options widen past corticosteroids
Tocilizumab maturity, secukinumab pivotal data, and emerging mechanism-targeted programs are reshaping giant cell arteritis management.
Phenylketonuria therapy options mature past low-phenylalanine diet
Pegvaliase commercial maturity, sapropterin use, and emerging gene therapy programs are restructuring phenylketonuria management.
Lipid management therapy widens past statins and PCSK9 monoclonals
Inclisiran maturity, bempedoic acid, lepodisiran and emerging oral PCSK9 programs, and ANGPTL3-targeted therapy are restructuring lipid management.
ANCA-associated vasculitis therapy options mature around avacopan
Avacopan commercial maturity, rituximab maintenance protocols, and emerging mechanism programs are restructuring ANCA-associated vasculitis management.
Chronic neuropathic pain therapy reshapes around novel sodium channel mechanisms
Suzetrigine (Nav1.8 inhibitor) acute pain approval and follow-on Nav1.8 and Nav1.7 programs in chronic pain are reshaping non-opioid pain management.
Allergic conjunctivitis therapy widens past topical antihistamines
Topical multi-action options, novel mechanism programs, and emerging biologic-pathway therapy are reshaping allergic conjunctivitis management.
Cannabis use disorder pharmacotherapy programs reach late-stage trials
Late-stage cannabis use disorder pharmacotherapy programs and integrated behavioural-and-pharmacological care models are emerging in a previously bare category.
Glioblastoma therapy options evolve past temozolomide and TTFields
Tumour-treating fields maturity, IDH-mutant glioma vorasidenib approval, and emerging mechanism-targeted programs are reshaping glioma management.
Vasomotor symptom therapy expands past fezolinetant
Elinzanetant approval, follow-on NK3 receptor antagonist programs, and combination NK1-NK3 approaches are restructuring vasomotor symptom management.
Presbyopia pharmacological options widen past pilocarpine
Pilocarpine maturity, follow-on miotic-class programs, and emerging non-miotic mechanism programs are reshaping presbyopia management.
Chimeric autoantibody receptor T cells enter pemphigus vulgaris
Desmoglein 3-targeted CAART cell therapy programs in pemphigus vulgaris are reading out as a precision approach to autoantibody-driven disease.
Heart failure with reduced ejection fraction therapy moves beyond four-pillar standard
Vericiguat addition, mechanism-targeted programs, and emerging genetically-defined HFrEF approaches are reshaping the post-four-pillar landscape.
Obstructive sleep apnea pharmacotherapy emerges past CPAP-and-MAD
Tirzepatide OSA approval, follow-on GLP-1 OSA programs, and upper airway pharmacological programs are reshaping obstructive sleep apnea management.
Lupus nephritis therapy options mature past induction-and-maintenance
Voclosporin and belimumab in lupus nephritis plus emerging mechanism programs are restructuring induction and maintenance therapy.
Chronic kidney disease therapy options widen in non-diabetic populations
Finerenone in non-diabetic CKD, SGLT2 expansion past diabetes, and novel mechanism programs are restructuring CKD management beyond the diabetic kidney disease frame.
Refractory chronic cough acquires a first targeted mechanism class
P2X3 antagonist class entry plus follow-on novel mechanism programs are establishing chronic cough as a real prescribing category.
Stroke prevention restructures across atrial fibrillation, lipids, and acute window
Factor XIa inhibitors entering late-stage trials, expanded thrombectomy windows, and tenecteplase displacement of alteplase are restructuring stroke prevention and acute care.
Type 1 diabetes therapy reshapes around disease modification and automation
Teplizumab disease modification, automated insulin delivery system maturity, and emerging beta-cell replacement programs are restructuring T1D management.
MASH therapy class establishes after resmetirom approval
Resmetirom commercial uptake plus follow-on FGF21 analogues, GLP-1 plus glucagon, and PPAR-pan agonists are establishing a real metabolic-dysfunction-associated steatohepatitis prescribing class.
Sjogren disease therapy enters real prescribing territory
First positive pivotal readouts in Sjogren disease are establishing a category that has had no specific systemic therapy for decades.
Inherited retinal disease gene therapy widens past RPE65
X-linked retinitis pigmentosa, choroideremia, and additional inherited retinal disease gene therapy programs are reading out.
Hepatocellular carcinoma systemic therapy options mature
Atezolizumab plus bevacizumab maturity, durvalumab plus tremelimumab adoption, and TIGIT-class programs are restructuring advanced HCC.
Sarcoidosis acquires mechanism-targeted therapy after a long quiet period
Efzofitimod late-stage data and other emerging mechanism-targeted programs are reshaping pulmonary sarcoidosis after years of corticosteroid-only first-line.
Genetically-targeted Parkinson's programs reach pivotal data
LRRK2 inhibitors and GBA-targeted programs in Parkinson's disease are reading out as the first genetically-defined Parkinson's therapy options.
Tumor-infiltrating lymphocyte therapy expands past melanoma
TIL therapy approval in metastatic melanoma is opening pathways into cervical and other solid tumour indications.
Cell therapy moves into non-malignant rare disease
Cell-therapy approaches are beginning to land in non-malignant rare-disease indications including severe lupus, scleroderma, and inherited metabolic conditions.
Choroidal melanoma therapy options widen
Approved tebentafusp and emerging programs are creating the first systemic therapy options for metastatic choroidal melanoma.
Apolipoprotein C3-targeted therapy expands lipid management
ApoC3-targeted programs are widening the triglyceride-lowering toolset and entering routine lipid practice.
Donanemab's limited-duration treatment paradigm is reshaping the lifetime cost calculation against lecanemab
Donanemab's protocol stops dosing once amyloid clearance is achieved, typically 12-18 months. Lecanemab is continuous indefinitely. Over a 10-year treatment horizon, the implied lifetime cost difference is substantial.
ASO platform diversification beyond DMD is reshaping rare-disease commercial planning
The antisense oligonucleotide (ASO) platform has moved beyond the DMD foundation into multiple rare-disease indications including SMA, AdLD, Stargardt disease and adjacent conditions. The platform-level commercial logic is reshaping rare-disease portfolio strategy at multiple sponsors.
PROTAC oncology pipeline is transitioning from concept to commercial reality
Targeted protein degradation programs (PROTACs and molecular glues) in oncology have moved from early-stage proof-of-concept to multiple late-stage assets with pivotal readouts in the next 24 months. The mechanism's promise of drugging previously undruggable targets is closer to commercial validation than at any prior point.
Gantenerumab post-mortem: what the failure tells the field about Abeta-targeting
The gantenerumab phase 3 readout failure is a useful data point for understanding what differentiates the successful anti-amyloid antibody class from the unsuccessful programs. The implications for next-generation amyloid-targeting and adjacent neurodegeneration pipeline are material.
FDA accelerated-approval reform is changing oncology evidence requirements
Post-Aduhelm reform of the accelerated-approval pathway has tightened expectations for confirmatory trial design, enrolment timing, and surrogate-endpoint validity. Oncology programs targeting accelerated approval are seeing the practical effect first.
Antibody-drug conjugates are reshaping the HER2-low breast cancer setting
T-DXd's expansion into HER2-low has changed second-line decision-making, and the ADC class is delivering further candidates that are likely to redefine biomarker-driven sequencing across breast cancer subtypes.
The "clinically meaningful" debate around CDR-SB is not settling
The interpretive argument over whether a sub-half-point CDR-SB delta represents a clinically meaningful slowing of decline continues to shape regulatory, payer, and clinician views.
Donanemab introduces a finite-duration treatment model
Donanemab's protocol allows treatment cessation once amyloid plaque clearance is confirmed - a meaningfully different model from indefinite biologic dosing.
GLP-1 receptor agonists enter Alzheimer's clinical trials
Phase 3 readouts on semaglutide in Alzheimer's are due, with mechanistic interest in metabolic, vascular, and inflammatory pathways.
FDA's accelerated approval pathway under continued post-Aduhelm scrutiny
Aducanumab's voluntary withdrawal in 2024 left lasting institutional caution about surrogate-endpoint approvals in neurodegeneration.
Tau-targeting programs advance behind the amyloid wave
Anti-tau immunotherapies and small molecules are progressing through mid-stage trials, with the field watching for the first credible clinical signal.
Snapshots
5Anti-amyloid antibody landscape, 2026 mid-year reference
A dated reference snapshot of the anti-amyloid antibody class for Alzheimer's disease as of mid-2026: approved products, withdrawn products, late-stage pipeline, label-defining clinical evidence, and the operational pattern that defines the class.
What we are watching in Alzheimer's, as of Q2 2026
A reference list of the threads PatientSpotlight is actively tracking - clinical readouts, regulatory and reimbursement decisions, real-world evidence accumulation, and operational rollout. Each thread names what to watch and why it matters, without predicting when it will resolve.
Alzheimer's drug development pipeline, as of Q2 2026
A reference view of the late-stage Alzheimer's pipeline as of Q2 2026 - tau-directed programs, GLP-1 receptor agonists, neuroinflammation, synaptic and neuronal resilience, and genetic/protein-clearance approaches.
Clinical trial endpoints in Alzheimer's disease, as of Q2 2026
A reference view of the cognitive, functional, and biomarker endpoints used in late-stage Alzheimer's trials - what each measures and how to read a readout that uses it.
Disease-modifying therapies in Alzheimer's, as of Q2 2026
Two anti-amyloid antibodies have traditional FDA approval; subcutaneous formulations are advancing; the post-amyloid pipeline is portfolio-shaped.
Explained
13How chronic cough has emerged as a discrete indication with its own pipeline
Chronic cough was historically managed within the broader respiratory and ENT framework as a symptom rather than as an indication. The emergence of P2X3 receptor antagonists and adjacent novel mechanisms has established chronic cough as a discrete therapeutic indication with a distinct pipeline, regulatory pathway and commercial logic.
How natural history studies are being co-designed with sponsors
Natural history studies in rare disease are increasingly being designed jointly between patient organisations, academic networks, and sponsors with assets in development. The co-design model has implications for evidence quality, regulatory acceptability, and the commercial use of the resulting data.
How pediatric mental health prescribing differs from adult and why it matters
Pediatric mental health prescribing operates under different evidence frameworks, regulatory expectations, and access structures than adult prescribing. Understanding the differences is essential for any sponsor with mental health assets that are eligible for paediatric expansion or that interact with paediatric-eligible populations.
How combination regimens are restructuring late-line oncology trial design
Late-line oncology trials are increasingly testing combination regimens rather than single-agent comparisons. The trial-design conventions, the regulatory framework, and the commercial implications of combination-as-standard are different enough from single-agent design that cross-functional teams need to understand the shift.
How ophthalmic drug delivery has moved from formulation chemistry to device engineering
Ophthalmic drug delivery has shifted in the past decade from a primarily formulation-chemistry exercise (eye drops, conventional intravitreal injection) into a device-engineering one (sustained-release implants, port-delivery systems, microneedle and suprachoroidal delivery). The methodology and the implications for commercial planning are worth understanding.
How orphan drug designation reshapes commercial planning across markets
Orphan drug designation is more than a marketing-exclusivity tag. It carries fee waivers, accelerated review pathways, market-exclusivity protection, and HTA-layer implications that shape commercial planning materially across markets.
How to read a modern phase 3 oncology readout: hazard ratios, crossover and what regulators care about
Phase 3 oncology trial readouts have a vocabulary, a set of conventions, and a regulatory frame that the headline numbers obscure. This is a plain-language guide to the parts of the readout that decide approval, label and reimbursement.
Patient registries are now critical infrastructure, not nice-to-have evidence
Patient registries in rare disease have moved from research-grade data sources to operational infrastructure underpinning regulatory submissions, HTA decisions, post-marketing evidence, and patient-finding models. The framing has shifted; the funding model is still catching up.
What are GLP-1 receptor agonists, and why are they being tested in Alzheimer's?
GLP-1 receptor agonists are a class of drugs originally developed for type 2 diabetes and now widely used for obesity. The class is now in late-stage Alzheimer's trials. The mechanistic case spans metabolic, vascular, inflammatory, and direct neuronal pathways - and the access shape would be very different from anti-amyloid therapy.
What is iADRS, and how is it different from CDR-SB?
iADRS - the Integrated Alzheimer's Disease Rating Scale - is a composite endpoint that combines a cognitive score (ADAS-Cog) with a functional score (ADCS-iADL) into a single number. It is the primary endpoint donanemab used in its pivotal trial and is reported alongside CDR-SB in many late-stage Alzheimer's programs. It answers a slightly different question than CDR-SB does.
What is the FDA accelerated approval pathway, and why does it matter for Alzheimer's?
Accelerated approval is a 1992 FDA regulatory pathway that lets a drug come to market based on a surrogate endpoint reasonably likely to predict clinical benefit, with a confirmatory trial obligated to follow. In Alzheimer's, the pathway is closely associated with the aducanumab episode and a recalibrated bar for what surrogate evidence the agency now considers persuasive.
What is CDR-SB, and what does a small change on it actually mean?
CDR-SB is the Clinical Dementia Rating - Sum of Boxes, the cognitive and functional scale used as the primary endpoint in most late-stage Alzheimer's trials. It is a six-box, 0–18 scale, scored by a clinician from a structured interview with the patient and a caregiver.
What are anti-amyloid antibodies, and how do they work?
A plain-language explanation of the disease-modifying drug class that defines the current Alzheimer's treatment landscape.