▎Edited by the WuXi AppTec content team

Soleno Therapeutics announced today,The U.S. FDA has approved Vykat XR (Diazoxide Choline, formerly known as DCCR）Sustained-release tablets are used to treat Prader-Willi Syndrome (PWS).Adults and children over 4 years oldThe patient's bulimia.Vykat XRPreviously granted Breakthrough Therapy Designation and Fast Track status by the U.S. FDA, and awarded Orphan Drug Designation in both the United States and the European Union. The press release noted,Vykat XR is a treatment forThe first approved drug for binge-eating disorder in Prader-Willi syndrome patients.

Vykat XRIs an innovative proprietary containing diazoxide cholineSustained-release formulation,Its active ingredient is continuously released over 24 hours, maintaining a stable drug concentration, and only needs to be taken once daily.The drug works by activating K in the hypothalamus, other parts of the central nervous system, the pancreas, and adipose tissue._ATPA pathway that is expected to enhance the appetite-regulating effects of leptin and insulin, reduce fat accumulation in the body, and help decrease binge eating.

The efficacy of Vykat XR is obtainedSupported by the 16-week randomized withdrawal portion of the multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial Study C602-RWP.Compared to continuing to receiveVykat XRCompared to subjects receiving treatment, subjects randomized to placebo showed a statistically significant worsening in binge eating symptoms.Before entering the randomized withdrawal period, all subjects underwent an average of 3.3 years of double-blind and/or open-label treatment.Vykat XR Treatment。

Overall Survival Extended by More Than a Year: Johnson & Johnson's Bispecific Antibody Combination Therapy Shows Positive Phase 3 Clinical Results

Johnson & Johnson today announced the overall survival (OS) results from the Phase 3 clinical trial MARIPOSA. The trial results showed that the combination of its bispecific antibody therapy Rybrevant (amivantamab) and Lazcluze (lazertinib) as a first-line treatment for patients with epidermal growth factor receptor (EGFR) Patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring exon 19 deletions (ex19del) or L858R substitution mutations demonstrated significantly prolonged overall survival.

MARIPOSA is a study evaluating the combination of Rybrevant and Lazcluze as a first-line treatment for patients with specificEGFRA Phase 3 clinical trial for patients with mutated NSCLC, with osimertinib as the active control group.Data showed that at a median follow-up time of 37.8 months, the combination of Rybrevant/Lazcluze reduced the risk of death by 25% (HR=0.75; 95% CI: 0.61–0.92) compared to the active control group. The median OS in the Rybrevant/Lazcluze group has not yet been reached, while the median OS in the active control group was 36.7 months.Among patients treated with the Rybrevant/Lazcluze combination, 56% were still alive at 3.5 years, compared to 44% in the active control group. Predictions based on survival data indicate that Rybrevant/Lazcluze can extend median OS by at least 12 months compared to the active control.

Rybrevant is a humanized EGFR/MET-targeted bispecific antibody.It has multiple mechanisms of anticancer action, not only blocking EGFR and MET-mediated signal transduction but also guiding immune cells to target tumors carrying activating and drug-resistant EGFR/MET mutations and amplifications.Lazcluze is a highly selective, third-generation oral EGFR tyrosine kinase inhibitor capable of crossing the blood-brain barrier.

PFS Extended 4 Times, Anti-Cancer Small Molecule Therapy Receives FDA Approval Again

The U.S. FDA announced today the approval of Exelixis-developedCabometyx（cabozantinib）Extended Indications for UseTreatment of adult and pediatric patients 12 years and older with locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors (pNET) and extra-pancreatic neuroendocrine tumors (epNET) that are unresectable and have progressed after prior therapy.

The efficacy of Cabometyx in treating patients with neuroendocrine tumors was evaluated in the CABINET study, a double-blind, placebo-controlled, multicenter clinical trial that enrolled 298 patients with locally advanced or metastatic pNET/epNET who had failed prior treatment and were unable to undergo surgical resection.

Data show,In the pNET patient cohort, the median progression-free survival (PFS) was 13.8 months (95% CI: 8.9–17.0) in the Cabometyx group and 3.3 months (95% CI: 2.8–5.7) in the placebo group, with Cabometyx reducing the risk of disease progression or death by 78% (HR=0.22; 95% CI: 0.12–0.41; p<0.0001).Overall survival data are not yet mature,CabometyxIn the group, 32 patients died (accounting for 48% of enrolled patients), and in the placebo group, 17 patients died (accounting for 52% of enrolled patients). Fifty-two percent of patients in the placebo group crossed over to open-label Cabometyx treatment, which may have impacted OS assessment.

In the epNET cohort. The median PFS in the Cabometyx group was 8.5 months (95% CI: 6.8–12.5), compared to 4.2 months (95% CI: 3.0–5.7) in the placebo group. Cabometyx reduced the risk of disease progression or death by 60% (HR=0.40; 95% CI: 0.26–0.61; p<0.0001).OS data are not yet mature, with 83 deaths in the Cabometyx group (63% of enrolled patients) and 40 deaths in the placebo group (60% of enrolled patients). Thirty-seven percent of patients in the placebo group crossed over to open-label Cabometyx treatment, which may have impacted the OS assessment.

Cabometyx can inhibit multiple tyrosine kinases, including MET, VEGFR-1, 2, 3, AXL, RET, ROS1, KIT, etc.These receptor tyrosine kinases are associated with tumor formation, metastasis, angiogenesis, and drug resistance. It has been approved in the United States for the treatment of advanced renal cell carcinoma, as a first-line therapy for advanced renal cell carcinoma in combination with the PD-1 antibody nivolumab, and for the treatment of hepatocellular carcinoma (HCC) patients who have previously received sorafenib, among other indications.

Bayer Acquires PRMT5 Inhibitor, Phase 1 Clinical Trial Initiated

Bayer and Suzhou Puhuo Pharmaceutical Technology Co., Ltd. (Puhuo Pharma) today announced that they have entered into a global licensing agreement for Puhuo Pharma’s oral small molecule protein arginine methyltransferase 5 (PRMT5) inhibitor, which selectively targets tumors with deletions of the metabolic enzyme 5’-deoxy-5’-methylthioadenosine phosphorylase (MTAP). Under the terms of the agreement,Bayer Obtains Global Exclusive License for the Development, Manufacturing, and Commercialization of Methythioadenosine (MTA) Synergistic PRMT5 InhibitorsBayer has enrolled the first patient in a Phase 1 first-in-human dose-escalation clinical trial to study the effects of this MTA-cooperative PRMT5 inhibitor (development code BAY 3713372) for the treatment of MTAP-deleted solid tumors.

PRMT5 and MTAP Play Key Roles in Cellular Metabolism and Are Crucial for Cell Survival.Approximately 10% to 30% of cancers involve MTAP deletion, leading to elevated MTA levels in tumor cells, andBAY 3713372 is designed to bind to the PRMT5-MTA complex, specifically targeting tumor vulnerabilities.