1. PADCEV + KEYTRUDA achieves significant EFS improvement and higher pCR in MIBC
On Aug 12, 2025, Pfizer and Astellas Pharma announced positive results from the Phase III EV-303 clinical trial. The study evaluates PADCEV™ (enfortumab vedotin), a Nectin-4 directed antibody-drug conjugate, in combination with KEYTRUDA™ (pembrolizumab) compared to surgery in patients with muscle-invasive bladder cancer (MIBC) who are not eligible for or declined cisplatin-based chemotherapy. The trial demonstrated a clinically meaningful and statistically significant improvement in event-free survival (EFS, primary endpoint). The secondary endpoint of overall survival (OS) and pathologic complete response (pCR) rate was also met.
2. Ianalumab (BAFF-R mAb) delivers positive Phase III results across autoimmune diseases
Novartis recently announced clinical study results on different autoimmune disease regarding the use of ianalumab. Lanalumab targets the BAFF receptors on B cells. It depletes B cells through antibody-dependent cellular cytotoxicity (ADCC) and signal inhibition.
On Aug 11, 2025, Phase III trials evaluating ianalumab in adults with active Sjögren's disease showed statistically significant improvements in disease activity as a primary endpoint. Sjögren’s disease is a chronic autoimmune disorder where the body’s immune system mistakenly attacks its own glands.
On Aug 12, 2025, another set of positive results from the VAYHIT2 Phase III trial
were announced for ianalumab with eltrombopag in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids. ITP is an autoimmune disorder characterised by a low platelet count, purpura, and hemorrhagic episodes. As the primary endpoint, ianalumab with eltrombopag significantly prolonged the time to treatment failure compared to the placebo with eltrombopag. As for the secondary endpoints, patients treated with ianalumab combination improved platelet counts at six months.
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3. FDA accepts priority review of gepotidacin for urogenital gonorrhoea
On Aug 11, 2025, GSK announced that the U.S. FDA had accepted priority review for gepotidacin as a new treatment for uncomplicated urogenital gonorrhoea in patients 12 years of age and older. Urogenital gonorrhoea is a sexually transmitted infection (STI) caused by the bacterium N. gonorrhoeae. Previously, it has been approved by the FDA to treat uncomplicated urinary tract infection (UTI). The application was based on the results from the EAGLE-1 phase III trial. It showed a similar success rate of gepotidacin (oral, two doses of 3,000mg) compared to intramuscular ceftriaxone (500mg) plus oral azithromycin (1,000mg) combination at the urogenital site (92.6% vs. 91.2%). Additionally, there were no failures at the urogenital site due to bacterial persistence of N. gonorrhoeae in either treatment arm.
4. FDA approves new indication for Wegovy® in MASH with moderate to advanced liver fibrosis
On 15 Aug 2025, Novo Nordisk announced that the U.S. FDA had approved an additional indication for Wegovy® (semaglutide 2.4 mg) to treat patients with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) in adults with moderate to advanced liver fibrosis. The accelerated approval was based on results from the ESSENCE trial. At week 72, 36.8% of people treated with Wegovy® showed improvement in liver fibrosis with no worsening of steatohepatitis compared to 22.4% on placebo. Moreover, 62.9% of people in the treatment arm achieved resolution of steatohepatitis with no worsening of liver fibrosis compared to 34.3% in the placebo arm.
5. FDA approves PAPZIMEOS™ for recurrent respiratory papillomatosis
On Aug 15, 2025, Precigen announced that the U.S. FDA had approved PAPZIMEOS™ (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP). RRP is a rare, debilitating, and potentially life-threatening disease of the upper and lower respiratory tract caused by chronic HPV 6 or HPV 11 infection. The approval is based on an open-label, single-arm pivotal study. In the study, 51% of patients achieved a complete response, showing no surgeries for 12 months post-treatment and with durability beyond a year. The therapy was well tolerated, and no treatment-related adverse events were beyond Grade 2. Mechanistically, PAPZIMEOS induced HPV 6/11–specific T-cell responses, with significantly greater peripheral expansion in responders than in non-responders.
6. FDA approves BRINSUPRI for non-cystic fibrosis bronchiectasis
On Aug 12, 2025, Insmed announced the U.S. FDA has approved BRINSUPRI™ (brensocatib 10 mg and 25 mg tablets) to treat non-cystic fibrosis bronchiectasis (NCFB) in adults and children 12 years and older. NCFB is a chronic lung condition where airways are permanently widened (dilated) and prone to inflammation and infection. The FDA approval of BRINSUPRI is backed by the Phase 3 ASPEN and Phase 2 WILLOW trials. In ASPEN, BRINSUPRI 10 mg and 25 mg reduced the annual exacerbation rate by 21.1% and 19.4% vs placebo, respectively. The secondary endpoints were also met with the prolonged time to the first exacerbation and increased proportion of patients who remained exacerbation-free over 52 weeks. Moreover, patients receiving 25 mg also experienced statistically significantly less decline in lung function compared to the placebo arm.
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