FDA Grants Priority Review to Potential First-in-Class Hepatitis B ASO Therapy; AbbVie Enters $1.45B Pan-KRAS Inhibitor Collaboration

Potential "First-in-Class" HBV ASO Therapy Granted Priority Review

Ionis Pharmaceuticals and its partner GSK announced today,The U.S. FDA has accepted the New Drug Application (NDA) for GSK's investigational antisense oligonucleotide (ASO) therapy, bepirovirsen, for the treatment of adult patients with chronic hepatitis B (CHB), granting it Priority Review. The PDUFA date for this application is set for October 26, 2026.At the same time, this therapy has also been granted Breakthrough Therapy Designation by the FDA. Currently, bepirovirsen is under review by multiple regulatory agencies, including the European Medicines Agency (EMA), Japan's Ministry of Health, Labour and Welfare (MHLW), and China's National Medical Products Administration (NMPA).

This regulatory submission and Breakthrough Therapy Designation are primarily based on the positive results from the Phase 3 B-Well 1 and B-Well 2 clinical trials. The studies showed,Compared with standard treatment alone, bepirovirsen combined with standard treatment achieved statistically significant and clinically meaningful improvements in functional cure rates across all ranked endpoints., especially in patients with lower baseline hepatitis B surface antigen (HBsAg) levels, the efficacy is more significant. Meanwhile, the therapy shows good performance in safety and tolerability, consistent with previous study results.

Bepirovirsen is a potential “first-in-class” ASO therapy with a triple mechanism of action., aiming to identify and target the genetic components (i.e., RNA) of the hepatitis B virus, potentially enabling the patient's immune system to regain control over the viral infection. Bepirovirsen can inhibit viral replication in the body, reduce HBsAg levels in the blood, and activate the immune system, thereby increasing the chances of achieving a sustained response. GSK obtained the authorization for bepirovirsen from Ionis Pharmaceuticals in 2019 and has been collaborating with them to advance the drug’s development.

$1.45 Billion! AbbVie Reaches Collaboration on Pan-KRAS Small Molecule Inhibitor

Today, Kestrel Therapeutics announced that its investigational oral pan-KRAS small molecule inhibitor, KST-6051, has completed the first patient dosing in a Phase 1 clinical trial targeting patients with advanced or metastatic solid tumors carrying KRAS mutations. At the same time, the company also announced a strategic collaboration agreement with AbbVie. According to the agreement,AbbVie will gain the option to exclusively acquire Kestrel upon reaching predetermined R&D milestones, while supporting the project's development.

KST-6051 is a pan-KRAS inhibitor with a differentiated mechanism of action., its unique binding mode aims to achieve sustained inhibition of the target pathway, which is expected to result in better efficacy compared to other pathway inhibitors.This drug can simultaneously bind to both the active and inactive states of KRAS, covering a broader range of oncogenic signals while selectively avoiding HRAS and NRAS, thereby reducing the toxicity risk associated with non-selective pan-RAS inhibition from a mechanistic perspective.According to the agreement between both parties, AbbVie will provide financial support for the project and have exclusive acquisition rights after completing development and achieving regulatory-related milestones. This includes upfront payments, potential exercise payments, and subsequent R&D and regulatory milestone payments.The total amount of this transaction can reach up to 1.45 billion US dollars.

Boehringer Ingelheim's Peptide Weight Loss Therapy Meets Co-Primary Endpoints in Phase 3 Trial

Today, Boehringer Ingelheim announced positive topline results from its Phase 3 SYNCHRONIZE-1 clinical trial. In the trial, its therapy Survodutide (BI 456906) achieved both co-primary endpoints using two methods: the efficacy estimand and the treatment policy estimand. When evaluated using the efficacy estimand,In a 76-week treatment of adults with obesity or overweight but without type 2 diabetes, the semaglutide group achieved an average sustained weight loss of up to 16.6%, which was statistically significantly different (p<0.0001) compared to the 3.2% in the placebo group.The trial also met another co-primary endpoint: Based on the efficacy estimand, 85.1% of adults treated with sofudeptide achieved a weight reduction of ≥5% after 76 weeks of treatment, compared to 38.8% in the placebo group (p<0.0001). Preliminary analysis indicates,The weight loss brought by Sofudeptide mainly comes from the reduction of fat tissue, while the decrease in lean body mass accounts for only a small part of the total weight loss.This level of weight loss effect demonstrates the potential of sofudeptide as a clinically valuable treatment option for people with obesity or overweight.

Sofudopeptide is a glucagon/GLP-1 receptor dual agonist that can simultaneously activate glucagon receptors and GLP-1 receptors.These two receptors play an important role in regulating the body's metabolic functions. Currently, a systematic and comprehensive Phase 3 clinical development program is underway to evaluate sufotudipeptide, including the SYNCHRONIZE series of studies for overweight or obese populations, and the LIVERAGE series of studies for populations with metabolically associated steatohepatitis (MASH) and liver fibrosis. Sufotudipeptide has the potential to treat MASH in adult patients with moderate or severe (Stage 2 or 3) liver fibrosis and has been granted Fast Track designation and Breakthrough Therapy designation by the U.S. FDA.Sofdegliptide is licensed by Zealand Pharma to Boehringer Ingelheim, which has exclusive global responsibility for its development and commercialization.