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Welcome to PharmaShots Weekly
| May 11, 2026 Edition
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| | PharmaShots Weekly is your Monday signal check: a fast, story-driven run-through of the deals, data, approvals, and platforms that actually shift the biopharma landscape.
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UCB’s $2.2B Candid Buyout Backs BCMA
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| | ARCHIMED’s ~$1.1B Esperion Deal Adds CVR Upside
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| | Bayer’s ~$2.45B Perfuse Buyout Adds PER-001
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| | Roche’s ~$1.05B PathAI Deal Boosts AI Diagnostics
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Stay Curious.
| Stay informed!
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| | Stay ahead with PharmaShots Weekly!
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Cover Story
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| • | Angelini’s $4.1B Catalyst Buyout Builds a Transatlantic Brain Health and Rare Disease Platform
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Licensing & Collaborations
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| • | BioCryst’s ~$345M Navenibart EU License Advances Long-Acting HAE Prophylaxis
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| • | Madrigal’s ~$1B ARO-PNPLA3 License Adds Precision RNAi for Genetically Defined MASH
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| • | GSK’s ~$1.0B SA030 License Adds First-in-Disease ALK7 siRNA for Cardiometabolic Risk
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| • | Waiv’s AI Pathology Collaboration with Daiichi Sankyo Targets Early ADC Biomarker Discovery
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| • | GSK Partners with Halozyme to Develop SC Formulation of Multiple Oncology Drugs
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M&A
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| • | UCB’s ~$2.2B Candid Buyout Bets on BCMA T-Cell Engagers for Immune Reset
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| • | ARCHIMED’s Up to ~$1.1B Take-Private Deal Gives Esperion Shareholders Cash Plus CVR Upside
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| • | Bayer’s Up to ~$2.45B Perfuse Buyout Adds PER-001 to Rebuild Ophthalmology Franchise
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| • | Roche’s Up to ~$1.05B PathAI Buyout Puts AI Pathology at the Core of Precision Diagnostics
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Clinical Trials
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Oncology
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| • | Celcuity’s Phase III VIKTORIA-1 Trial Advances Gedatolisib in PIK3CA-Mutant HR+/HER2- Breast Cancer
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| • | Sensei Doses First Patient in Phase Ib/II FTH-PIK-101 Trial for Advanced Breast Cancer
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| • | ALX Oncology presented exploratory P‑Ib/II trial data for evorpacept + Ziihera (zanidatamab) in mBC patients
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Rare & Liver Diseases
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| • | Mirum’s Phase IIb VISTAS Trial Positions Volixibat for PSC-Associated Pruritus
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Nephrology
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| • | Alebund Completes Enrollment in Global Phase III RESPOND-2 Trial of AP301 for Hyperphosphatemia
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Imaging & Diagnostics
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| • | Bayer’s Phase III REVEAL Study Supports PET Imaging in Cardiac Amyloidosis
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Dermatology
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| • | Incyte Reports Durable 24-Week Phase III TRuE-AD4 Data for Opzelura in Atopic Dermatitis
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Ophthalmology & Gene Therapy
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| • | Beacon Therapeutics Reports 12-Month DAWN Data for Gene Therapy in X-Linked Retinitis Pigmentosa
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Regulatory Watch
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| • | EC Approves Rhythm’s Imcivree for Acquired Hypothalamic Obesity
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| • | FDA Approves Arvinas & Pfizer’s Veppanu for ESR1-Mutated Breast Cancer
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| • | BriaCell Receives FDA IND Clearance for Bria-BRES+ Breast Cancer Trial
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| • | FDA Approves Langlara, Biosimilar to Lantus
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| • | Harbour BioMed Gains FDA IND Clearance for HBM7004 in Solid Tumors
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MedTech Watch
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| • | Guardant360 CDx Gains FDA Approval as Companion Diagnostic for Veppanu
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| • | Ortho Development Launches Trivicta Cementless Hip Stem
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| • | Insulet Enrolls First Patient in EVOLVE Study of Omnipod 5 for Type 2 Diabetes
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| • | Caris Launches AI-Powered MI Clarity for Breast Cancer Recurrence Assessment
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| • | Multi4 Medical Secures CE Mark for Outpatient Bladder Cancer Treatment System
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| • | Artera Receives FDA Clearance for AI-Based Breast Cancer Risk Assessment Tool
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| • | Bausch + Lomb Launches Bi-Blade+ Vitrectomy Cutter in the EU on Stellaris Elite Platform
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Angelini’s $4.1B Catalyst Deal Expands CNS Reach
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Angelini Pharma has entered into a definitive agreement to acquire all outstanding shares of Catalyst Pharmaceuticals, marking Angelini’s formal entry into the U.S. market and significantly expanding its Brain Health and rare disease footprint.
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Why It Matters
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| • | Deal Value: ~$4.1B all‑cash acquisition at $31.50 per share, representing a premium of roughly 21% to Catalyst’s unaffected share price and about 28% to the 30‑day VWAP.
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| • | US Market Entry: Provides Angelini with an immediate commercial footprint in the U.S., anchored by Catalyst’s rare disease neurology portfolio.
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| • | Rare Disease Scale: Combines complementary rare disease assets to create a stronger, scalable platform for expanding access to life‑changing therapies.
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Strategic Focus
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| • | Brain Health Expansion: Builds on Angelini’s Brain Health strategy by adding marketed and pipeline assets in rare neurological conditions.
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| • | Commercial Synergies: Leverages Catalyst’s “exceptional” U.S. commercial infrastructure alongside Angelini’s European operations for global brand-building.
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Long-Term Growth: Positions Angelini for sustainable, diversified growth across North America and Europe in high‑value rare disease markets.
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| | Strategic Deals & Partnerships
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BioCryst’s ~$345M Navenibart EU License Advances Long-Acting HAE Prophylaxis
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BioCryst Pharmaceuticals has entered into a license agreement with an Irish affiliate of Neopharmed Gentili, granting Neopharmed Gentili exclusive rights to commercialize navenibart for hereditary angioedema (HAE) across Europe.
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Why It Matters
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| • | Deal Value: Up to ~$345M in economics, including $70M upfront and as much as $275M in regulatory and sales milestones, plus tiered 18%–30% royalties on European net sales.
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| • | Long-Acting Prophylaxis: Navenibart’s extended half-life and durable plasma kallikrein inhibition aim to reduce dosing frequency and treatment burden versus existing HAE prophylactic options.
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| • | Regional Focus: Offloads European commercialization to a regional specialist while allowing BioCryst to retain value through milestones and royalties as navenibart advances.
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Strategic Focus
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| • | Franchise Expansion: Positions navenibart as a next-generation complement to BioCryst’s oral kallikrein inhibitor Orladeyo within a broader HAE franchise.
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| • | Capital-Efficient Development: Provides non-dilutive capital to support the global Phase III program and regulatory activities ahead of the targeted 2027 filing.
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| • | European Commercial Leverage: Leverages Neopharmed Gentili’s European footprint to maximize HAE market penetration once navenibart is approved.
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Madrigal’s ~$1B ARO‑PNPLA3 License Adds Precision RNAi for Genetically Defined MASH
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Arrowhead has granted Madrigal an exclusive global license to ARO‑PNPLA3, a GalNAc‑conjugated RNAi therapeutic designed to reduce liver expression of PNPLA3 as a potential precision treatment for metabolic dysfunction‑associated steatohepatitis (MASH) in genetically defined patients.
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Why It Matters
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| • | Deal Value: Up to ~$1B in total economics, including $25M upfront and as much as $975M in development, regulatory, and sales milestones, plus high‑single‑digit to mid‑teens tiered royalties.
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| • | Precision MASH Strategy: Adds a clinical‑stage RNAi asset targeting PNPLA3 I148M, complementing Madrigal’s broader MASH therapy Rezdiffra with a genotype‑driven approach.
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| • | Compelling Early Data: Phase I results show rapid, durable liver fat reductions in homozygous patients, supporting advancement into Phase II combination and monotherapy studies.
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Strategic Focus
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| • | Franchise Expansion: Strengthens Madrigal’s MASH franchise by pairing a foundational small‑molecule therapy with a precision RNAi program for high‑risk genetic subpopulations.
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| • | Risk‑Shared Economics: Provides Arrowhead with non‑dilutive capital and substantial upside via milestones and royalties while offloading late‑stage development and commercialization to Madrigal.
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| • | Platform Validation: Further validates Arrowhead’s RNAi platform in liver‑targeted metabolic disease and underscores pharma appetite for genetically defined RNAi programs.
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GSK’s ~$1.0B SA030 License Adds First-in-Disease ALK7 siRNA for Cardiometabolic Risk
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Suzhou Siran Biotechnology (SiranBio) has granted GSK a global exclusive license (excluding mainland China, Hong Kong, Macau, and Taiwan) to SA030, a potential first‑in‑disease, long‑acting siRNA oligonucleotide being developed for metabolic and vascular disease.
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Why It Matters
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| • | Deal Value: Up to ~$1.005B in total economics, including a reported $55M upfront payment and substantial downstream milestones, plus tiered royalties on ex‑Greater China sales.
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| • | Novel ALK7 Targeting: SA030 is a long‑acting ALK7‑targeting siRNA designed to reduce cardiometabolic risk by lowering abdominal fat while preserving muscle mass, addressing a key limitation of current GLP‑1 therapies.
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| • | Cardiometabolic Ambition: Supports GSK’s strategy to expand in cardiometabolic disease using oligonucleotide modalities aligned with its existing RNA‑targeted pipeline.
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Strategic Focus
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| • | Risk‑Staged Development: Keeps early‑stage execution with SiranBio through Phase I, then hands off to GSK for costly late‑stage development and global commercialization.
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| • | Geographic Split: Preserves Greater China rights for SiranBio, enabling potential regional partnerships or independent commercialization while GSK builds the rest‑of‑world franchise.
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| • | Platform Validation: Validates SiranBio’s siRNA platform and ALK7 approach, potentially paving the way for additional cardiometabolic programs and deals.
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Waiv’s AI Pathology Collaboration with Daiichi Sankyo Targets Early ADC Biomarker Discovery
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Waiv has entered into a collaboration with Daiichi Sankyo to lead digital pathology biomarker discovery for an antibody–drug conjugate (ADC) program, leveraging Waiv’s end-to-end computational pathology platform.
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Why It Matters
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| • | Biomarkers from Small Datasets: Purpose-built to discover AI-derived biomarkers in early ADC studies with limited patient numbers, tackling one of pharma’s hardest translational challenges.
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| • | Slide-First Precision: Uses routine pathology slides (H&E and IHC) to predict treatment response, potentially reducing the need for more invasive or costly biomarker assays.
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| • | ADC Differentiation: Supports Daiichi Sankyo’s efforts to clinically differentiate its ADCs through refined patient selection and response prediction.
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Strategic Focus
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| • | End-to-End Capability: Positions Waiv as a partner able to engage early and carry biomarkers through to clinically validated, deployable tests.
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| • | Multimodal Integration: Combines digital pathology with clinical and trial data to deliver more informative, interpretable biomarker signatures.
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| • | Scalable AI Infrastructure: Demonstrates how foundation models trained on large histopathology datasets can be repurposed across oncology programs and sponsors.
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GSK’s Enhanze Deal with Halozyme Targets Subcutaneous Delivery for Multiple Oncology Assets
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GSK has entered into a global collaboration and license agreement with Halozyme to develop and commercialize subcutaneous (SC) formulations of multiple promising oncology targets, including antibody‑drug conjugates (ADCs), with an option for GSK to add additional future drug targets to the collaboration.
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Why It Matters
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| • | Modality Expansion: Marks Halozyme’s first Enhanze collaboration that explicitly includes ADC targets, broadening the platform’s reach into one of oncology’s fastest‑growing modalities.
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| • | Patient-Centric Delivery: Supports GSK’s effort to move suitable oncology therapies from IV infusions to more convenient SC administration, potentially enhancing quality of life and clinic efficiency.
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| • | Royalty-Driven Upside: Adds another large‑pharma partner to Halozyme’s royalty-based model, expanding the long‑term revenue pool if GSK’s SC oncology products reach the market.
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Strategic Focus
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| • | Oncology Portfolio Optimization: Gives GSK a validated SC delivery solution it can apply across multiple current and future oncology agents, including ADCs.
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| • | Platform Leverage: Builds on Enhanze’s track record across multiple commercial products and >100 markets to de‑risk SC reformulations for GSK.
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| • | Future Target Options: The option structure allows GSK to add additional assets over time, creating a scalable collaboration as its oncology pipeline evolves.
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| | Mergers & Acquisitions (M&A)
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UCB’s ~$2.2B Candid Buyout Bets on BCMA T-Cell Engagers for Immune Reset
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UCB has entered into a definitive agreement to acquire Candid Therapeutics, expanding UCB’s immunology pipeline with a portfolio of novel T‑cell engagers (TCEs) designed for deep, targeted B‑cell depletion and immune reset in autoimmune diseases.
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Why It Matters
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| • | Deal Value: Up to ~$2.2B total consideration, including $2B upfront and up to $200M in milestone payments tied to pipeline progress and regulatory outcomes.
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| • | Autoimmune TCE Leadership: Marks one of the largest bets to date on T‑cell engagers in autoimmune disease, bringing oncology‑style immune redirection into chronic immune disorders.
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| • | Deep B-Cell Depletion: Cizutamig and follow-on assets aim for deep, targeted B‑cell depletion while optimizing CD3 binding to reduce risks such as cytokine release syndrome.
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Strategic Focus
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| • | Immunology Franchise Expansion: Builds on UCB’s existing autoimmune footprint (e.g., in rheumatology and dermatology) with a new class of antibody-based immune reset therapies.
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| • | Platform and Pipeline: Secures a multi‑program, modular TCE platform with four bispecific programs, two already in early human trials, enabling broad indication expansion.
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| • | Durable Disease Control: Targets more complete and lasting control of autoimmune disease by eliminating pathogenic B‑cell subsets rather than chronic immunosuppression.
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ARCHIMED’s Up to ~$1.1B Take-Private Deal Gives Esperion Shareholders Cash Plus CVR Upside
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Esperion Therapeutics and ARCHIMED have entered into a definitive agreement under which funds managed by ARCHIMED will acquire Esperion, with Esperion to be delisted from Nasdaq and operate as a privately held company.
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Why It Matters
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| • | Deal Value: Up to ~$1.1B in total fully diluted equity value, including $3.16 per share cash (a 58% premium to the April 30, 2026 close) plus up to $100M in CVR milestones.
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| • | Investor Upside: The CVR structure allows existing shareholders to participate in future commercialization success of bempedoic acid and ENBUMYST while realizing immediate cash value.
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| • | Strategic Sponsor: ARCHIMED brings sector-focused capital and private ownership to support Esperion’s cardiometabolic growth strategy outside the public markets.
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Strategic Focus
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| • | Cardiometabolic Execution: Aligns incentives to drive uptake of bempedoic acid products ahead of the 2027 CVR milestone year.
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| • | ENBUMYST Growth: Encourages aggressive commercialization of bumetanide products to capture the $60M CVR opportunity by 2030.
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| • | Balance-Sheet Reset: Take-private structure may give Esperion more flexibility to invest in launches and lifecycle management without public-market pressure.
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Bayer’s Up to ~$2.45B Perfuse Buyout Adds PER-001 to Rebuild Ophthalmology Franchise
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Bayer has entered into an agreement to fully acquire Perfuse Therapeutics, including its lead asset PER‑001, complementing Bayer’s ophthalmology pipeline and expertise in ischemia-driven retinal diseases.
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Why It Matters
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| • | Deal Value: Up to ~$2.45B total, including $300M upfront and as much as $2.15B in performance‑based milestones tied to PER‑001’s development and commercialization.
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| • | Post‑Eylea Strategy: Helps Bayer sustain and evolve its ophthalmology franchise as Eylea faces biosimilar competition and patent expiry.
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| • | Disease Modification: Targets vascular perfusion and ischemia in glaucoma and diabetic retinopathy, aiming beyond symptomatic IOP lowering and anti‑VEGF approaches.
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Strategic Focus
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| • | Ophthalmology Depth: Strengthens Bayer’s late‑stage pipeline in retinal and optic nerve diseases with a differentiated mechanism of action.
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| • | Long-Acting Delivery: Leverages a twice‑yearly intravitreal implant format to improve adherence and reduce treatment burden relative to frequent injections.
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| • | Platform Potential: Creates optionality to expand endothelin receptor antagonism into additional ischemia‑driven ocular conditions over time.
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Roche’s Up to ~$1.05B PathAI Buyout Puts AI Pathology at the Core of Precision Diagnostics
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Roche has entered into a definitive merger agreement to acquire PathAI, a US-based digital pathology and AI company, building on their partnership established in 2021 and expanded in 2024 to include development of AI-enabled companion diagnostic algorithms.
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Why It Matters
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| • | Deal Value: Up to ~$1.05B in total consideration, including $750M upfront and up to $300M in performance-based milestones.
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| • | Digital Pathology Scale: Positions Roche to scale PathAI’s AI-powered pathology solutions globally, transforming manual slide review into fully digital, AI-supported workflows.
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| • | Companion Dx Synergy: Deepens Roche’s companion diagnostics leadership by embedding AI models that can speed biomarker discovery and CDx algorithm development.
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Strategic Focus
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| • | Integrated Diagnostics: Brings PathAI into Roche’s Diagnostics division to create an end-to-end ecosystem from tissue imaging through AI analysis to diagnostic reporting.
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| • | Biopharma Services: Enhances Roche’s offering to biopharma partners with AI-enabled clinical trial readouts and translational research services.
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| • | Personalised Healthcare: Accelerates the shift from broad intervention to personalised treatment by using AI pathology to refine diagnoses and better match patients to targeted therapies.
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| | Clinical Trials & Data Readouts
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Phase III VIKTORIA‑1
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HR+/HER2- Advanced Breast Cancer (PIK3CA-mutant cohort)
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Celcuity reports topline data from the PIK3CA mutant cohort of the Phase III VIKTORIA‑1 study evaluating gedatolisib + fulvestrant ± palbociclib vs. SoC in HR+/HER2– advanced breast cancer patients progressing on or after a CDK4/6 inhibitor plus an aromatase inhibitor.
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Key Details
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| • | Trial met its primary endpoint: the gedatolisib triplet (gedatolisib + fulvestrant + palbociclib) achieved a statistically significant and clinically meaningful improvement in PFS vs. alpelisib + fulvestrant in PIK3CA‑mutant disease.
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| • | A key secondary endpoint, outside the primary hierarchical testing strategy, also showed improved PFS with the gedatolisib doublet (gedatolisib + fulvestrant) vs. alpelisib + fulvestrant; detailed results will be presented at ASCO 2026.
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Regulatory Plans
Celcuity plans to submit these PIK3CA‑mutant data as an sNDA to the US FDA and other regulators.
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Asset Snapshot
The FDA has granted Priority Review to gedatolisib for HR+/HER2–, PIK3CA‑wild type advanced breast cancer, with a PDUFA date of July 17, 2026.
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Primary Sclerosing Cholangitis (PSC) – Cholestatic Pruritus
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Mirum reports global Phase IIb VISTAS data for volixibat 20 mg BID vs placebo in 158 patients with cholestatic pruritus due to PSC, including 111 with moderate–severe itch (primary cohort) and 47 with mild itch (secondary cohort).
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Key Details
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| • | In the primary cohort, VISTAS met its primary endpoint: volixibat achieved a mean 2.72‑point pruritus improvement on the Adult ItchRO scale vs a 1.08‑point reduction with placebo, with significant itch relief seen by Week 2 and maintained through Week 12; 55.6% of patients on volixibat achieved ≥2‑point itch reduction vs 26.3% on placebo.
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| • | Full results will be presented in a late‑breaking oral session at the EASL 2026 International Liver Congress.
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Regulatory Plans
Mirum has a pre‑NDA meeting with the US FDA for volixibat in PSC planned for summer 2026 and expects to submit an NDA in H2 2026.
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Asset Snapshot
Oral IBAT inhibitor also in Phase IIb VANTAGE for primary biliary cholangitis, with topline PBC data now expected in Q1 2027.
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HR+/HER2- Advanced/Metastatic Breast Cancer
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Sensei Biotherapeutics has dosed the first patient in the Phase Ib/II FTH‑PIK‑101 trial evaluating all‑oral PIKTOR (serabelisib + sapanisertib) with fulvestrant and/or other anticancer therapies in patients with HR+/HER2– advanced or metastatic breast cancer.
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Key Details (Prior Phase Ib data)
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| • | In a prior Phase Ib study in heavily pretreated advanced breast, endometrial, and ovarian cancers, PIKTOR + paclitaxel achieved a 47% ORR in 15 evaluable patients, increasing to 71% in tumors harboring PI3K pathway mutations, including complete responses in endometrial cancer.
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Ongoing Development
Sensei is also supporting an ongoing open‑label Phase II FTH‑PIK‑201 (GOG‑3111) trial of PIKTOR with paclitaxel (± diet intervention) in approximately 40 patients with advanced or recurrent endometrial cancer.
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Hyperphosphatemia in Dialysis-dependent CKD
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Alebund has completed patient enrollment in the global Phase III RESPOND‑2 trial of AP301, a novel iron‑based phosphate binder, in hyperphosphatemia, including 138 patients in the US and 144 in China.
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Key Details
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| • | RESPOND‑2 enrolled 282 patients (planned n=264) aged ≥12 years with CKD on maintenance dialysis and hyperphosphatemia, and includes an 8‑week double‑blind dose‑titration phase, a 24‑week open‑label phase, and a 3‑week randomized withdrawal phase comparing AP301 maintenance dose vs ineffective low dose.
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Regulatory Plans
Based on positive Phase III RESPOND‑1 results in China, Alebund plans to submit an NDA in China after alignment with the NMPA, while the US FDA has agreed that the ongoing global Phase III trial can serve as the single pivotal study to support US registration of AP301.
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Cardiac Amyloidosis – PET/CT Imaging
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Bayer has reported topline Phase III REVEAL data for IV PET/CT radiotracer I‑124 evuzamitide vs clinical standard‑of‑care diagnosis in 170 adults with suspected cardiac amyloidosis.
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Key Details
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| • | REVEAL met its co‑primary endpoints, demonstrating adequate sensitivity and specificity for the diagnosis of cardiac amyloidosis based on visual PET/CT scan interpretation; data will be shared with regulators, presented at upcoming meetings, and used to support regulatory submissions to the US FDA and other authorities.
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Regulatory Status
I‑124 evuzamitide has US FDA Breakthrough Therapy Designation for PET imaging in suspected or known cardiac amyloidosis and Orphan Drug designation for AL and ATTR amyloidosis in both the US and EU.
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Moderate Atopic Dermatitis – Adults
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Incyte has reported 24‑week Phase III TRuE‑AD4 data for Opzelura (ruxolitinib) cream BID vs vehicle in 241 adults with moderate AD and inadequate response, intolerance or contraindication to topical corticosteroids and calcineurin inhibitors; data were presented at EADV 2026.
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Key Details (Week 8 efficacy)
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| • | As previously reported, TRuE‑AD4 met its co‑primary endpoints, with 83.5% of patients on Opzelura achieving EASI75 and 74.4% achieving IGA‑TS, and 74.3% achieving ≥4‑point improvement in Itch NRS, demonstrating clinically meaningful itch relief by Week 8.
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24-week outcomes
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| • | Among patients achieving EASI50 at Week 8, 84.3% of Opzelura‑treated patients completed treatment through Week 24, maintaining robust disease control: EASI75 in 84.3%, IGA‑TS in 70.6%, low mean affected BSA of 2.5% at Weeks 8 and 24, and durable itch relief (NRS4) in 64.7% through Week 24.
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Regulatory context
These data support a Type II variation application for Opzelura in the EU, with an EMA decision expected in H1 2026.
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X-Linked Retinitis Pigmentosa (XLRP)
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Beacon Therapeutics has reported 12mos. P‑II (DAWN) trial data assessing subretinal gene therapy laruparetigene zovaparvovec (laru‑zova) in pts with XLRP who had previously been treated with an AAV vector‑based gene therapy delivering the full‑length RPGR protein.
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Key Details
DAWN showed sustained improvements in low luminance visual acuity (LLVA) and microperimetry mean sensitivity through Mos. 12, with 50% of high‑dose pts achieving ≥2‑line vision improvement and 25% achieving ≥3‑line gains from baseline, while 67% of low‑dose pts achieved ≥2‑line gain; data were presented at ARVO’26.
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Next Steps
Beacon is also evaluating pts with LLVA improvement and changes in microperimetry mean macular sensitivity in the global P‑II/III (VISTA) trial of laru‑zova for XLRP, with topline data expected in H2’26.
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Metastatic HER2-positive Breast Cancer (mBC)
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ALX Oncology presented exploratory P‑Ib/II trial data for evorpacept + Ziihera (zanidatamab) in heavily pretreated HER2+ mBC pts, all previously treated with Enhertu, at ESMO Breast Cancer 2026.
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Key Details
Across 24 pts treated with evorpacept + Ziihera at 20 mg/kg (n=3) or 30 mg/kg (n=21), the combination achieved a 33% cORR and mPFS of 3.6mos., whereas pts with centrally confirmed HER2‑positive (ccHER2+) disease (n=10) had a higher 60% cORR and mPFS of 8.3mos. Among pts (n=5) with ccHER2+ disease and high CD47 expression (>20% membrane staining), all responded to the combination (100% cORR: 1 CR, 4 PRs), with mDOR of 20.2mos. and mPFS of 22.1mos., vs a 25% cORR and 3.4mos. mPFS in ccHER2+ pts with low CD47 expression.
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| | Global Regulatory Momentum in Pharma
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Regulatory Watch: This Week’s Key Approvals and Milestones
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From metabolic disorders and oncology to biosimilars, this week’s regulatory developments underscore continued progress in expanding therapeutic innovation and improving patient access across key disease areas. Here’s a quick look at the latest approvals and regulatory milestones shaping the healthcare landscape:
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| • | Rhythm Pharmaceuticals reported European Commission approval of Imcivree (setmelanotide) for acquired hypothalamic obesity, expanding treatment options for rare obesity disorders.
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| • | Arvinas and Pfizer announced FDA approval of Veppanu (vepdegestrant) for ESR1-mutated breast cancer, advancing targeted therapy options in oncology.
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| • | BriaCell Therapeutics received FDA IND clearance to initiate a Phase I/II trial of Bria-BRES+ for breast cancer, supporting the advancement of next-generation immunotherapy approaches.
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| • | Lannett Company and Sunshine Lake Pharma reported FDA approval of Langlara, a biosimilar to Lantus, reinforcing momentum in the biosimilars market.
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| • | Harbour BioMed announced FDA IND clearance for HBM7004 for the treatment of advanced solid tumors, supporting the advancement of next-generation oncology therapeutics.
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| | AI and Precision Care Propel MedTech
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Precision Medicine Fuels MedTech Momentum
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From AI-powered diagnostics and companion testing to orthopedic innovation and diabetes technology, this week’s MedTech developments highlight continued momentum in precision medicine, digital health, and patient-centered care. Here’s a snapshot of the latest launches, clearances, and clinical milestones shaping the MedTech landscape:
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| • | Guardant Health secured FDA approval for Guardant360 CDx as a companion diagnostic for Arvinas’ Veppanu, advancing precision oncology testing.
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| • | Ortho Development launched the Trivicta Cementless Hip Stem for primary hip arthroplasty, expanding orthopedic implant options for joint replacement procedures.
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| • | Insulet enrolled the first patient in the EVOLVE study evaluating its fully closed-loop Omnipod 5 System for Type 2 Diabetes management.
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| • | Caris Life Sciences launched Caris MI Clarity, an AI-powered tool designed to assess breast cancer recurrence risk and support personalized treatment decisions.
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| • | Multi4 Medical received CE Mark approval for the Multi4 System to perform outpatient bladder cancer treatment, supporting minimally invasive care approaches.
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| • | Artera secured FDA clearance for its AI-based breast cancer risk assessment tool, further advancing the integration of artificial intelligence in oncology care.
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| • | Bausch + Lomb launched the Bi-Blade+ Vitrectomy Cutter in the EU for use with the Stellaris Elite Platform, strengthening its ophthalmic surgical technology portfolio.
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Big Pharma Bets Billions on AI, RNAi, and Rare Disease Expansion
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This week’s healthcare landscape was defined by billion-dollar dealmaking, precision medicine advances, and transformative clinical milestones. Angelini’s $4.1B Catalyst acquisition expands its rare disease and brain health footprint into the U.S., while Roche, Bayer, UCB, and GSK doubled down on AI pathology, ophthalmology, autoimmune T-cell engagers, and cardiometabolic RNAi therapies. Clinical updates from Celcuity, Mirum, and Beacon Therapeutics highlighted momentum in oncology, liver disease, and gene therapy, alongside key regulatory approvals and MedTech innovations accelerating personalized healthcare worldwide.
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