1. WELIREG® has been approved in the EU for two new indications

On February 18, 2025, Merck announced that the European Commission has conditionally approved WELIREG® (belzutifan) as monotherapy for adult Hippel-Lindau (VHL) disease patients who developed tumors at different tissues (Eg. localised renal cell carcinoma (RCC), central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors) and adult patients with advanced clear cell RCC that progressed following two or more lines of therapy. WELIREG® is a selective hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, which promotes cell cycle progression in hypoxic conditions. The drug works in the nucleus and may reduce the transcription of certain target genes that drive tumor growth. The two new indications were approved based on the results from both LITESPARK-004 and LITESPARK-005 trials and CHMP’s positive recommendations. WELIREG® is currently the first and only oral HIF-2α inhibitor approved in the European Union.

Link: https://www.merck.com/news/welireg-belzutifan-receives-first-european-commission-approval-for-two-indications/

2. Comparable efficacy of subcutaneous guselkumab induction therapy to the intravenous dosage

On February 21, 2025, J&J announced that TREMFYA® (guselkumab) subcutaneous (SC) induction therapy showed comparable efficacy to the current FDA-approved intravenous (IV) induction regimen when compared to the placebo. TREMFYA® blocks IL-23 and binds to CD64 simultaneously, and IL-23 is a key driver for immune-mediated disease including UC. Results were obtained from a Phase III ASTRO study for adults with moderately to severely active ulcerative colitis (UC). At Week 12, patients treated with SC induction therapy achieved 27.6% clinical remission (vs. 6.5% in the placebo arm), 65.6% clinical response (vs. 34.5% in the placebo arm) and 37.3% endoscopic improvement (vs. 12.9% in the placebo arm).

Link: https://www.jnj.com/media-center/press-releases/tremfya-guselkumab-subcutaneous-sc-induction-data-support-potential-to-be-the-first-and-only-in-its-class-to-offer-the-option-of-both-intravenous-and-sc-induction-therapy-in-ulcerative-colitis

3. Priority Review granted to Dupixent® to treat bullous pemphigoid

On Feb 18, 2025, Sanofi and Regeneron Pharmaceuticals announced that the U.S. FDA has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) to treat adults with bullous pemphigoid (BP). The application is supported by an ADEPT trial evaluating the efficacy and safety of Dupixent in 106 adults with moderate-to-severe BP. Both primary and secondary endpoints were met in the trial. Bullous pemphigoid is a rare skin condition that causes large fluid-filled blisters which could cause the skin to to bleed and crust. The antibody could successfully block the IL-4 and IL-13 pathways and has been approved to treat multiple skin diseases related to immune disorders.

Link: https://www.sanofi.com/en/media-room/press-releases/2025/2025-02-18-06-00-00-3027482

4. Duvakitug successfully manage two IBD conditions in phase 2 trial

On Feb 22, 2025, Sanofi and Teva Pharmaceuticals announced results from the RELIEVE UCCD phase 2b study of duvakitug for two common forms of inflammatory bowel disease (IBD) - ulcerative colitis (UC) and Crohn’s disease (CD). Duvakitug is a human IgG1-λ2 monoclonal antibody blocking TL1A which induces inflammation and drive fibrosis associated with IBD through binding to DR3.

In the UC cohort, 36% (450 mg dose) and 48% (900 mg dose) of patients treated with duvakitug achieved the primary endpoint of clinical remission at week 14 compared to 20% treated with placebo. There were also improvements in Clinical response, Endoscopic improvement and Histological-endoscopic mucosal improvement.

  • Clinical response (mMS): 81% (450 mg) and 70% (900 mg) compared to 52% treated with placebo.

  • Endoscopic improvement (MES): 45% (450 mg) and 50% (900 mg) compared to 23% treated with placebo.

  • Histological-endoscopic mucosal improvement (HEMI): 30% (450 mg) and 33% (900 mg) compared to 16% treated with placebo.

In the CD cohort, 26% (450 mg dose) and 48% (900 mg dose) of patients treated with duvakitug achieved the primary endpoint of endoscopic response compared to 13% on placebo at week 14. Other parameters were also improved with the treatment involved.

  • Endoscopic remission (SES-CD): 17% (450 mg) and 26% (900 mg) compared to 9% treated with placebo.

  • Clinical remission (CDAI): 50% (450 mg) and 54% (900 mg) compared to 41% treated with placebo.

  • Clinical response (CDAI): 61% (450 mg) and 62% (900 mg) compared to 41% treated with placebo.

  • Clinical response (PRO2): 50% (450 mg) and 53% (900 mg) compared to 29% treated with placebo.

The drug is well tolerated with no serious adverse events observed.

Link: https://www.sanofi.com/en/media-room/press-releases/2025/2025-02-22-07-30-00-3030764

5. HIV PrEP-Lenacapavir received a new drug application from the U.S. FDA

On February 18, 2025, Gilead Sciences announced that the U.S. FDA had accepted the new drug application for submissions for lenacapavir. Lenacapavir is the company’s twice-yearly injectable HIV-1 capsid inhibitor to prevent HIV as a pre-exposure prophylaxis (PrEP). This is based on the data from the Phase 3 PURPOSE 1 and PURPOSE 2 trials. In PURPOSE 1, there were zero infections in the lenacapavir arm compared to the background HIV incidence. In PURPOSE 2, there were two HIV infections observed in the lenacapavir group, demonstrating a 96% risk reduction in HIV infections.

Link: https://www.gilead.com/news/news-details/2025/us-fda-accepts-gileads-new-drug-applications-for-twice-yearly-lenacapavir-for-hiv-prevention-under-priority-review

6. Conditional marketing authorization has been granted to seladelpar for PBC

On February 20, 2025, Gilead Sciences announced that the European Commission has granted conditional marketing authorization for seladelpar in combination with ursodeoxycholic acid for the treatment of primary biliary cholangitis (PBC). PBC is a rare, chronic, autoimmune disease of the bile ducts that might result in impaired bile flow and potential liver damage. The approval was followed by the recommendation from the CHMP based on results from the Phase 3 RESPONSE study. In the study, 62% of participants achieved the primary endpoint of composite biochemical response in the 12th Month by taking seladelpar, versus 20% of participants taking placebo. Meanwhile, 25% of participants in the treatment arm achieved normal alkaline phosphatase (ALP), but none of the participants achieved it by receiving the placebo. The current approval is conditional and the company still needs to provide updated trial data to be further approved.

Link: https://www.gilead.com/news/news-details/2025/gileads-seladelpar-granted-conditional-european-marketing-authorization-for-the-treatment-of-primary-biliary-cholangitis

7. Regenerative Medicine Advanced Therapy designation to gene therapy AB-1005 for Parkinson’s Disease

On February 19, 2025, the U.S. FDA granted investigational gene therapy AB-1005 with Regenerative Medicine Advanced Therapy designation for the treatment of Parkinson’s disease (PD). The therapy is developed by Askbio, a gene therapy company under Bayer. AB-1005 can slow the disease progression and improve motor outcomes in patients with PD. In the 36-month Phase Ib data, there is a trend for improvement or stability on several PD-relevant clinical scales compared to the baseline.

Link: https://www.bayer.com/media/en-us/askbio-receives-fda-regenerative-medicine-advanced-therapy-designation-for-parkinsons-disease-investigational-gene-therapy/

8. On February 19, 2025, Boehringer Ingelheim announced that the U.S. FDA has granted Priority Review to its new drug application for zongertinib to treat adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) mutations and treated with systemic therapy. Zongertinib is an investigational, irreversible tyrosine kinase inhibitor (TKI) that selectively inhibits HER2 while sparing EGFR, thereby off-target side effects. The application was supported by results from the Phase Ib Beamion LUNG-1 trial. In cohort 1, the study demonstrated an objective response rate (ORR) of 71%, six-month progression-free survival (PFS) of 69% and duration of response (DoR) rates of 73% in participated patients.

Link: https://www.boehringer-ingelheim.com/human-health/cancer/lung-cancer/zongertinib-priority-review-fda-non-small-cell-lung-cancer