Register Now: From Clinical to Commercial: Accelerating ADC Late-Stage Development with Technology Innovation and Integrated Platform
Logo Image
News|Podcasts|May 7, 2026

The BioPharm Brief: Breakthrough Signals in Cancer, Duchenne, and RNA Medicine

Today’s BioPharm Brief covers promising AML remission data from Aptevo, encouraging Duchenne muscular dystrophy trial results from Entrada Therapeutics, and a new GSK licensing deal expanding cardiometabolic RNA therapy development.

Welcome to The BioPharm Brief, your daily roundup of developments shaping the biopharmaceutical industry. Today’s episode focuses on encouraging clinical data in acute myeloid leukemia and Duchenne muscular dystrophy, along with a strategic licensing agreement aimed at strengthening cardiometabolic disease pipelines.

First, Aptevo Therapeutics reported strong new data from its frontline acute myeloid leukemia, or AML, RAINIER study. According to the company, its investigational therapy mipletamig, used in combination with venetoclax and azacitidine, demonstrated an 87% clinical benefit rate and an 81% remission rate across evaluable patients in the Phase 1b trial. The company says the study has now entered its final dose-optimization stage and remains on track for Phase 2 dose selection later this year. AML remains one of the more aggressive blood cancers, making remission durability and safety profiles key areas of focus for researchers and clinicians.

Meanwhile, Entrada Therapeutics announced positive topline results from its Phase 1/2 ELEVATE-44-201 study evaluating ENTR-601-44 in patients with Duchenne muscular dystrophy amenable to exon 44 skipping. The company reported statistically significant improvements in a functional movement assessment called Time to Rise velocity, along with favorable safety and tolerability findings. Researchers also observed increases in dystrophin production and exon skipping activity, two important biological markers tied to Duchenne treatment development.

And in pipeline news, GSK has expanded its cardiometabolic disease strategy through a licensing agreement for SiranBio’s siRNA candidate SA030. The deal reflects continued industry interest in RNA-based therapeutics and precision-targeted approaches for chronic metabolic conditions. GSK has increasingly emphasized pipeline growth in specialty and genetically informed disease areas.

Thanks for listening to The BioPharm Brief. For analysis and expert insights, please visit BioPharmInternational.com

KEY TAKEAWAYS

  • Aptevo’s AML program reported high remission and clinical benefit rates as the RAINIER study advances toward Phase 2.
  • Entrada Therapeutics showed early functional improvement and favorable safety data in Duchenne muscular dystrophy patients.
  • GSK continues expanding its RNA-focused cardiometabolic pipeline through strategic licensing activity.

Related to this article

View more
The BioPharm Brief: An AI-Designed Multibody ADC Enters the Clinic, Blackstone Backs Pfizer’s Monthly GLP-1, and enGene Readies a Bladder Cancer BLA
The BioPharm Brief: An AI-Designed Multibody ADC Enters the Clinic, Blackstone Backs Pfizer’s Monthly GLP-1, and enGene Readies a Bladder Cancer BLA
FDA clears the IND for Biolojic Design’s BD200, an AI-designed Trop2/Nectin-4 multibody ADC; Blackstone Life Sciences agrees to provide Pfizer up to $1.5 billion to develop its monthly GLP-1 receptor agonist berobenatide; and enGene Therapeutics plans a year-end BLA for detalimogene voraplasmid in BCG-unresponsive non-muscle invasive bladder cancer.
Read more
The BioPharm Brief: FDA Designations for a Bispecific CAR-T, a BLA for Friedreich’s Ataxia, and Phase 2b MASH Data for Efinopegdutide
The BioPharm Brief: FDA Designations for a Bispecific CAR-T, a BLA for Friedreich’s Ataxia, and Phase 2b MASH Data for Efinopegdutide
FDA grants RMAT and Fast Track designations to AbelZeta’s CD20/CD19 bispecific CAR-T prizlon-cel in relapsed or refractory large B-cell lymphoma, Larimar Therapeutics completes a BLA seeking accelerated approval of nomlabofusp for Friedreich’s ataxia, and Merck’s GLP-1/glucagon receptor agonist efinopegdutide meets its primary endpoint in a phase 2b MASH trial.
Read more
The BioPharm Brief: argenx Bets on Peptides, a Two-Bispecific Regimen in Prostate Cancer, and a New Take on T-Cell Engagers
The BioPharm Brief: argenx Bets on Peptides, a Two-Bispecific Regimen in Prostate Cancer, and a New Take on T-Cell Engagers
argenx agrees to acquire macrocyclic peptide developer Unnatural Products for up to about $1.15 billion, Johnson & Johnson’s pasritamig and JNJ-9401 bispecific regimen achieves a 73% PSA response in phase 1 in metastatic castration-resistant prostate cancer, and Janux Therapeutics’ TRBV6-targeted T-cell engager JANX011 shows deep B-cell depletion in healthy volunteers.
Read more
The BioPharm Brief: A Bispecific Approval in Head and Neck Cancer, TCR Bispecifics Head to the Clinic, and a New Antibody Target in Essential Thrombocythemia
The BioPharm Brief: A Bispecific Approval in Head and Neck Cancer, TCR Bispecifics Head to the Clinic, and a New Antibody Target in Essential Thrombocythemia
FDA grants accelerated approval to Johnson & Johnson’s subcutaneous EGFR/MET bispecific Rybrevant Faspro for previously treated head and neck squamous cell carcinoma, T-Therapeutics raises a $133 million Series B to take TCR-CD3 bispecifics into clinical trials, and Alethio Therapeutics raises a $90 million Series A to advance its first-in-class G6B antibody for essential thrombocythemia.
Read more
The BioPharm Brief: Ivonescimab Priority Review in China, Stargardt Gene Therapy Moves Toward Phase 3, and Longer Dosing Intervals for an IBD Bispecific
The BioPharm Brief: Ivonescimab Priority Review in China, Stargardt Gene Therapy Moves Toward Phase 3, and Longer Dosing Intervals for an IBD Bispecific
China’s NMPA grants priority review to Akeso’s PD-1/VEGF bispecific ivonescimab with chemotherapy for first-line biliary tract cancer, VeonGen Therapeutics aligns with FDA on a phase 3 design for its dual-AAV Stargardt gene therapy VG801, and Caldera Therapeutics’ IL-23p19×TL1A bispecific CLD-423 shows a half-life of more than 40 days in a phase 1a trial.
Read more
The BioPharm Brief: A $7 Billion Oncology Option Deal, a TL1A Pivot, and Oral Adalimumab Heads to the Clinic
The BioPharm Brief: A $7 Billion Oncology Option Deal, a TL1A Pivot, and Oral Adalimumab Heads to the Clinic
Boehringer Ingelheim licenses six preclinical ADC and T-cell engager programs from Sino Biopharmaceutical in a deal worth up to $7 billion, Spyre Therapeutics drops anti-TL1A antibody SPY072 in rheumatic diseases after phase 2 misses and turns to hidradenitis suppurativa, and Biora Therapeutics gets ethics approval for a first-in-human trial of its BioJet oral adalimumab capsule.
Read more