Emily Schoenthaler

The BioPharm Brief covers three recent FDA developments shaping the autoimmune and inflammatory disease landscape. The agency cleared a subcutaneous autoinjector for anifrolumab in systemic lupus erythematosus, enabling at-home administration. It also granted Priority Review to nipocalimab for warm autoimmune hemolytic anemia, highlighting its potential as a targeted treatment for a rare condition. In addition, a supplemental application has been submitted for subcutaneous risankizumab as an induction therapy in Crohn disease, which could reduce the need for infusion-based initiation. Together, these updates reflect a growing focus on patient convenience, expanded access, and more precise treatment approaches in immune-mediated diseases.

Mid-stage data suggest Oruka Therapeutics’ ORKA-001 may deliver high levels of skin clearance with the potential for once-yearly dosing in plaque psoriasis. However, limited study details and lack of safety data make the findings preliminary.

FDA approved a AstraZeneca's subcutaneous anifrolumab autoinjector for adults with moderate to severe systemic lupus erythematosus on standard therapy.

AbbVie seeks FDA approval for subcutaneous risankizumab induction in adults with moderately to severely active Crohn disease.

This episode of The BioPharm Brief covers innovation trends in biopharma hubs, a positive CHMP opinion for Novartis’ intrathecal gene therapy in spinal muscular atrophy, and early clinical data for a novel bispecific ADC in breast cancer. Key updates highlight regulatory momentum, emerging technologies, and next-generation oncology therapeutics.

Alphamab Oncology is set to unveil Phase I data for its bispecific ADC JSKN016 at the American Society of Clinical Oncology Annual Meeting (ASCO) 2026 in HER2-negative breast cancer. The early clinical readout highlights encouraging antitumor activity and a manageable safety profile in heavily pretreated patients, supporting continued development across multiple settings.

AbbVie has received a Complete Response Letter from the FDA for trenibotulinumtoxinE (TrenibotE), citing manufacturing-related questions but no safety or efficacy concerns. The company plans a timely resubmission, underscoring continued momentum for this short-acting neurotoxin candidate in facial aesthetics.

New developments in CAR-T therapy, pediatric biologics, and oral GLP-1 treatments highlight expanding innovation across oncology, immunology, and metabolic disease. Early data and regulatory progress signal a shift toward more targeted and accessible therapies for underserved patient populations.

Phase 3a PIONEER TEENS data show oral semaglutide significantly improved glycemic control in adolescents with type 2 diabetes, addressing a major unmet need in pediatric care. Novo Nordisk plans regulatory submissions in the US and EU, positioning the therapy as a potential first oral GLP-1 option for this population.

The FDA’s approval of Dupixent (dupilumab) for the pediatric patient population extends the biologic’s use beyond its previous indication in adults and adolescents aged 12 years and older, marking the first biologic option available in the United States for younger children with uncontrolled chronic spontaneous urticaria.

Cell therapy process design, leronlimab cancer data, and Amneal’s biosimilars strategy highlight key biopharma trends in manufacturing and innovation.

CytoDyn Inc. reported early Phase 2 data at the American Association for Cancer Research Annual Meeting 2026 showing that leronlimab in combination with TAS-102 and bevacizumab demonstrated biomarker activity, including reductions in circulating tumor DNA, in patients with metastatic colorectal cancer.

Johnson & Johnson reported that nipocalimab (Imaavy) demonstrated sustained disease control and reductions in pathogenic IgG levels through more than two years in patients with generalized myasthenia gravis. Patients who achieved sustained minimal symptom expression showed greater quality-of-life improvements, while a new head-to-head trial comparing nipocalimab with another FcRn inhibitor is now underway.

AstraZeneca reported consistent Phase III results for its IL-33 biologic in COPD, while Boehringer Ingelheim continues expanding its use of AI to accelerate drug discovery and disease research. Meanwhile, the FDA has accepted a biologics license application for Gazyva as a potential treatment for systemic lupus erythematosus, signaling progress in addressing unmet patient needs.

The FDA has accepted Roche’s application for Gazyva/Gazyvaro in systemic lupus erythematosus (SLE), based on Phase III data showing significant reductions in disease activity and improved remission rates. If approved, the anti-CD20 therapy could become the first B cell–targeting treatment for SLE and a potential new standard of care.

AstraZeneca reported that its IL-33-targeting biologic tozorakimab significantly reduced exacerbations in Phase III trials of chronic obstructive pulmonary disease, reinforcing its potential as a first-in-class treatment. The results add to growing evidence that the therapy could address persistent unmet needs in COPD patients who continue to experience exacerbations despite standard care.

The FDA has granted Priority Review to the combination of Padcev and Keytruda as a perioperative treatment for muscle-invasive bladder cancer, with a decision expected by Aug. 2026. Phase 3 trial results showed the regimen significantly improves survival outcomes and could become a new standard of care regardless of cisplatin eligibility.

Eli Lilly and Company is acquiring Kelonia Therapeutics for up to $7 billion to expand its genetic medicine portfolio, centered on Kelonia’s in vivo CAR T-cell therapy platform that generates therapies directly inside the patient. The deal is driven by early clinical promise of KLN-1010 in multiple myeloma and aims to overcome manufacturing and access limitations associated with traditional CAR T treatments.

Merck’s Enflonsia (clesrovimab) has been approved by the European Commission for RSV prevention in infants. The long-acting monoclonal antibody is given as a single fixed dose and is supported by Phase 2b/3 and Phase 3 data showing reduced RSV hospitalizations.

Marengo Therapeutics reported early Phase 2 activity for its invikafusp alfa combination in metastatic breast cancer and highlighted progress across its Selective T Cell Activation Repertoire (STAR) platform at the 2026 American Association for Cancer Research (AACR) Annual Meeting.

A look inside our latest issue of BioPharm International, where we explore how the biopharma industry is leaning into technical innovation while remaining grounded in operational discipline.

Roche plans a Phase III study of Elevidys to generate additional data for European approval and expand access for patients with Duchenne muscular dystrophy. The trial will evaluate efficacy and safety in ambulatory boys, aiming to strengthen clinical evidence and enable broader approval and reimbursement.

Eli Lilly and Company reported that its oral GLP-1 therapy orforglipron met the primary cardiovascular safety endpoint in a Phase 3 trial, while also delivering superior A1C reduction and weight loss compared with insulin glargine. The drug additionally showed a substantial reduction in all-cause mortality in a pre-planned analysis, supporting plans for FDA submission in type 2 diabetes.

PrecisionLife and Ovation.io are partnering to translate multi-omic analyses of GLP-1 receptor agonist use into predictive biomarker tools, with early findings indicating distinct biological drivers of glycemic control and weight loss and supporting development of diagnostic tests to guide treatment selection and clinical trial stratification.

Updated Phase 1/2 data show trastuzumab pamirtecan achieved an objective response rate of around 50% and disease control exceeding 90% in heavily pretreated patients with HER2-expressing recurrent endometrial cancer, with responses observed across varying levels of HER2 expression and duration of response not yet reached.

Mirvetuximab soravtansine–carboplatin combination demonstrates strong response rates and manageable safety profile in FRα-positive patients.

Ifinatamab Deruxtecan is a potential first-in-class B7-H3–directed antibody-drug conjugate for this patient population, which currently faces limited treatment options and poor prognosis.

Oricell Therapeutics has raised over $110 million in a pre-IPO round to advance its CAR-T cell therapy platform aimed at treating solid tumors, including liver cancer. The company is developing its lead candidate Ori-C101 and next-generation CAR-T technologies such as in vivo and armored CAR-T, positioning itself to address longstanding challenges in solid tumor immunotherapy.