The Promise
Outlook wins FDA appeal for ophthalmic bevacizumab
Outlook Therapeutics has won its appeal through the FDA’s Formal Dispute Resolution process for ONS-5010/LYTENAVA, an investigational ophthalmic formulation of bevacizumab-vikg for neovascular age-related macular degeneration. The biologic is a recombinant humanized anti-VEGF monoclonal antibody designed for intravitreal use in wet AMD.
The FDA’s Office of New Drugs concluded that substantial evidence of effectiveness has been established, based on the NORSE TWO trial and confirmatory evidence including NORSE EIGHT, natural history, and mechanistic and pharmacodynamic data. Outlook plans to resubmit the BLA in June 2026, with the company expecting a Class 1 resubmission and a decision within 60 days of FDA receipt. If approved, LYTENAVA would be the first FDA-approved ophthalmic formulation of bevacizumab supported by FDA-approved manufacturing, labeling, and pharmacovigilance. Source
BioMarin reports phase 3 growth data for VOXZOGO in hypochondroplasia
BioMarin has reported positive pivotal phase 3 results for VOXZOGO, also known as vosoritide, in children with hypochondroplasia. The therapy is a C-type natriuretic peptide analog given by daily subcutaneous injection, designed to increase linear growth by targeting the FGFR3 pathway involved in skeletal growth disorders.
In the CANOPY-HCH-3 study, VOXZOGO met its primary endpoint, improving annualized growth velocity by 2.33 cm per year versus placebo at week 52. Children treated with VOXZOGO also had statistically significant increases in standing height, height Z-score, and arm span, a measure linked to reach and functional independence. Safety was consistent with the established profile in achondroplasia, with no new safety signals, and BioMarin plans to submit an sNDA to the FDA in Q3 2026. Source
The Process and the Product
Image analysis speeds mRNA purification screening
Researchers have developed a high-throughput image analysis method to evaluate the filterability of mRNA precipitates during downstream purification. The approach uses automated image segmentation to assess precipitate morphology, distribution, and packing density, helping identify precipitation conditions that support more stable filtration performance.The study addresses a key bottleneck in scalable mRNA manufacturing: efficient solid-liquid separation after precipitation-based capture. Compared with conventional packing density measurements, the image-based method can reduce material consumption by up to 90 percent while accelerating screening of salts and additives. The authors say the workflow could support development of continuous precipitation-filtration strategies for mRNA and potentially other biotherapeutic modalities. Source
The Patient
Datroway wins US approval in first-line triple-negative breast cancer
AstraZeneca and Daiichi Sankyo’s Datroway has been approved in the US for adults with unresectable or metastatic triple-negative breast cancer who are not candidates for PD-1/PD-L1 inhibitor therapy. The therapy is a TROP2-directed DXd antibody-drug conjugate, making it the first TROP2-directed ADC approved for first-line treatment in this patient group.
The approval was based on the phase III TROPION-Breast02 trial, where Datroway improved median overall survival by 5.0 months versus chemotherapy and reduced the risk of disease progression or death by 43 percent. The objective response rate was 64 percent with Datroway compared with 30 percent for chemotherapy, and the safety profile was consistent with previous breast cancer trials. Source
FDA approves first treatment for chronic hepatitis delta
The FDA has approved Hepcludex, also known as bulevirtide-gmod, as the first approved treatment for adults with chronic hepatitis delta virus infection without cirrhosis or with compensated cirrhosis. The therapy is an injectable entry inhibitor that blocks HDV and hepatitis B virus entry into liver cells, offering a new option for a serious viral infection that can rapidly lead to liver fibrosis, liver cancer, liver failure, and death.
The accelerated approval was based on the phase 3 MYR301 trial, in which 48 percent of patients treated with once-daily Hepcludex achieved a combined virologic and ALT normalization response at week 48, compared with 2 percent in the delayed-treatment group. The FDA granted the therapy Breakthrough Therapy, Orphan Drug, and Priority Review designations, and awarded approval to Gilead Sciences. Source
Business Bulletin Board
Apogee secures $1.3B financing for phase 3 biologic program
Apogee Therapeutics has entered a strategic financing collaboration with Blackstone Life Sciences worth up to $1.3 billion to support phase 3 development and potential commercialization of zumilokibart. The therapy is an optimized monoclonal antibody biologic being developed for moderate-to-severe atopic dermatitis, with additional potential in asthma, eosinophilic esophagitis, COPD, and other inflammatory and immunology indications.
The non-dilutive financing includes up to $800 million in synthetic royalty funding and access to up to $500 million in senior corporate debt. Apogee says the deal, combined with its existing cash, could support zumilokibart through commercialization without future equity financing. The company also announced APEX phase 2 Part B results and plans to advance zumilokibart into phase 3 development. Source
Aptevo and Niowave partner on radiopharmaceutical oncology programs
Aptevo Therapeutics and Niowave have launched a 50/50 strategic collaboration to develop up to three radiopharmaceutical oncology programs. The therapeutic modality combines Aptevo’s multispecific immuno-oncology targeting platforms, ADAPTIR and ADAPTIR-FLEX, with Niowave’s radioisotope production and supply capabilities.
The deal marks Aptevo’s expansion into radiopharmaceutical therapy development and Niowave’s first investment in a therapeutic development program. Niowave made an at-the-market equity investment in Aptevo at closing, taking an initial 7.9 percent ownership position, with the potential to increase its stake to up to 19.99 percent through warrant exercises and future stock purchases. Source
BMS partners with Anthropic on enterprise AI platform
Bristol Myers Squibb has entered a strategic agreement with Anthropic to deploy Claude Enterprise across its research, clinical development, manufacturing, commercial, and corporate operations. This is an AI infrastructure and enterprise operations update rather than a therapeutic product milestone, with BMS positioning Claude as a shared intelligence platform for more than 30,000 employees.
The collaboration will focus on using agentic AI to accelerate software development, connect internal scientific and clinical data, support trial documentation and regulatory workflows, and improve manufacturing and quality processes. BMS says the aim is to move beyond chatbot-style AI toward systems that can integrate with company knowledge and workflows under enterprise governance and audit controls. Source
For this week’s cell and gene therapy news – including Verve’s phase 1 PCSK9 base-editing data, SynCav1 gene therapy for neurodegeneration, Lilly’s non-viral DNA delivery deal, tumour-activated CAR-T, in vivo CAR-M, and Scribe’s first-in-human PCSK9 silencing study – click here.
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