Emily Schoenthaler

In today’s podcast, we're seeing microbiome therapies gain FDA Fast Track status, Leo Pharma expand into gene therapy via acquisition, and automation improving AAV manufacturing efficiency.

Restore Vision Inc. reported interim Phase 1/2 data showing early vision improvements in patients with advanced retinitis pigmentosa treated with its optogenetic gene therapy RV-001.

Ensorcell has entered the cell and gene therapy manufacturing market with its VersaWeld sterile tube welding platform, unveiled at INTERPHEX 2026 where it also won Best New Product/Service.

LEO Pharma has acquired U.S.-based gene therapy company Replay to strengthen its rare disease dermatology pipeline through a novel herpes simplex virus-based platform. The deal adds a preclinical gene therapy program targeting dystrophic epidermolysis bullosa and expands LEO Pharma’s long-term strategy in genetic skin disease innovation.

Novartis has finalized its US manufacturing and R&D expansion plan with a seventh new facility in North Carolina, reinforcing domestic drug production capabilities. The investment is part of a broader $23 billion strategy to enable end-to-end manufacturing across advanced therapeutic platforms.

Zealand Pharma and Roche will advance the amylin analog petrelintide into Phase 3 trials for chronic weight management following positive mid-stage data. The move positions petrelintide as a potential differentiated obesity therapy with a focus on tolerability and sustained weight loss.

Pfizer’s Elrexfio significantly improved progression-free survival in the Phase 3 MagnetisMM-5 trial for relapsed or refractory multiple myeloma. The results strengthen its potential as an earlier-line therapy in a difficult-to-treat patient population.

Genexine’s first-in-class SOX2 degrader GX-BP1 demonstrated up to 96% tumor growth inhibition in preclinical models presented at AACR 2026. The bioPROTAC candidate also prevented tumor relapse and restored drug sensitivity, positioning it as a promising next-generation oncology therapy.

As the biopharmaceutical industry moves increasingly toward applied AI in GMP manufacturing, emphasizing real-world use, PDA Week 2026 is expected to evolve as it further dives into risk management and data-driven quality decisions.

Survodutide delivers significant Phase III weight loss results, FDA grants RMAT status to Orca-Q, and Ligand expands its portfolio with a $739 million acquisition of XOMA.

Biopharma news highlights: Henlius and Organon gain EU approval for a pertuzumab biosimilar, while Pfizer reports Phase 3 success for Elrexfio in multiple myeloma. Genexine advances GX-BP1 targeting SOX2, signaling new progress in overcoming cancer resistance and expanding oncology innovation.

Boehringer Ingelheim announced promising Phase III data showing that survodutide achieved up to 16.6% weight loss and significant metabolic improvements in adults with obesity or overweight. The dual glucagon/GLP-1 agonist may offer a new approach targeting both weight reduction and liver-related metabolic health.

The FDA has granted RMAT designation to Orca-Q, an investigational allogeneic T-cell therapy for high-risk hematologic malignancies, based on early phase 1 data.

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.