1. Submission of the novel intravesical drug-releasing system to the U.S. FDA for approval
On 15 January 2025, Johnson & Johnson announced that the company has initiated the submission of an original New Drug Application with the U.S. FDA for TAR-200 to treat patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC) with carcinoma in situ (CIS), with or without papillary tumours. TAR-200 is an investigational intravesical drug-releasing system designed to provide sustained local delivery of gemcitabine into the bladder. The new submission is supported by data from the Phase 2b SunRISe-1 registration study where the complete response rate is 83.5% and 82% of responders maintained response.
2. TROP2-directed Antibody-drug conjugate has been granted priority preview in lung cancer and approved for breast cancer
On 13 January 2025, AstraZeneca and Daiichi Sankyo datopotamab deruxtecan has been granted priority review for the treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) who have received prior systemic therapies, including an EGFR-directed therapy. Datopotamab deruxtecan is an antibody-drug conjugate targeting TROP2 overexpressed on many tumours. The decision was based on data from the TROPION-Lung05 Phase II trial and TROPION-Lung01 Phase III trial. Datopotamab deruxtecan demonstrated a confirmed objective response rate (ORR) of 42.7% and a median duration of response (DoR) of 7.0 months. In the same week, on 17 January 2025, datopotamab deruxtecan was approved by the U.S. FDA to treat adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease. Approval of this indication is supported by TROPION-Breast01 Phase III trial where the drug significantly reduced the risk of disease progression or death by 37% compared to the investigator’s choice of chemotherapy and the median progression-free survival was also two months longer in the experiment arm (6.9 months vs. 4.9 months).
3. The U.S. FDA approved Calquence combined with chemoimmunotherapy for patients with mantle cell lymphoma
On 17 January 2025, AstraZeneca’s Calquence (acalabrutinib) in combination with bendamustine and rituximab (anti-CD20 antibody) has been approved in the US for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who are ineligible for autologous hematopoietic stem cell transplantation. MCL is a rare and typically aggressive form of non-Hodgkin lymphoma (NHL), which is often diagnosed at an advanced stage. Calquence is a second-generation Bruton’s tyrosine kinase (BTK) inhibitor selectively blocking the BTK signalling pathway in the B cells. This pathway is crucial for B cell proliferation, trafficking, chemotaxis and adhesion.
The new approval was based on the results from ECHO Phase III trial. In the study, Calquence plus bendamustine and rituximab reduced the risk of disease progression or death by 27% compared to the standard-of-care chemoimmunotherapy. Median PFS was 66.4 months for patients treated with the Calquence combination versus 49.6 months with chemoimmunotherapy alone.
4. Approval of Omvoh by the U.S. FDA to treat Crohn's disease
On 15 January 2025, Eli Lilly and Company announced that the U.S. FDA has approved Omvoh® (mirikizumab-mrkz) for the treatment of moderately to severely active Crohn's disease in adults. This is the second inflammatory bowel disease (IBD) indication being approved following the approval of moderately to severely active ulcerative colitis (UC) in 2023. Omvoh® reduces the local inflammation in the GI tract by targeting interleukin-23p19 (IL-23p19) which leads to intestinal inflammation. This approval is based on positive results from the randomised placebo-controlled Phase 3 VIVID-1 study. In Omvoh group, 53% of patients achieved clinical remission at one year versus 36% in the placebo arm. Moreover, 46% of patients showed visible healing of the intestinal lining with Omvoh compared to 23% on placebo. The new FDA approval provides a new treatment option for adults with Crohn's disease to achieve long-term remission and visible healing of the intestinal lining.
5. Approval of LUMAKRAS to treat metastatic colorectal cancer with specific KRAS G12C mutation
On 17 January 2025, Amgen’s LUMAKRAS® (sotorasib) was approved by the U.S. FDA to treat adult patients with KRAS G12C-mutated metastatic colorectal cancer (mCRC) who have received prior fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy in combination with Vectibix® (panitumumab, anti-EGFR antibody). The KRASG12C mutation is present in approximately 3-5% of colorectal cancers and sotorasib can bind to KRASG12C and trap it in the inactive state, therefore blocking tumour growth.
The approval is supported by the Phase 3 CodeBreaK 300 study where both 960 mg/day or 240 mg/day LUMAKRAS dose has been compared to the investigator's choice of SOC (trifluridine and tipiracil or regorafenib). The median progression-free survival in LUMAKRAS 960 mg daily plus Vectibix arm is 5.6 months compared to 2 months in the control arm. Moreover, the study demonstrated an improved overall response rate (ORR) of 26% compared to 0% with the investigator's choice.
6. Iclepertin failed to meet both primary and secondary endpoints in treating schizophrenia
On January 16 2025, Boehringer Ingelheim announced that both primary and key secondary endpoints were not met in the Phase III CONNEX clinical program focusing on treating adults with schizophrenia. There were no statistically significant effects on cognition or functioning observed in patients treated with iclepertin versus placebo at six months. Iclepertin is a novel glycine transporter-1 (GlyT1) inhibitor which increases glycine levels in the brain. Increased glycine level improves the function of NMDA receptors and could potentially address cognitive deficits associated with schizophrenia. However, this study did not prove the efficacy of the investigational drug.
7. Subcutaneous semaglutide 7.2 mg achieved 20.7% weight loss in the STEP UP obesity trial
On 17 January 2025, Novo Nordisk announced results from a global phase 3b trial investigating subcutaneous semaglutide 7.2 mg compared to semaglutide 2.4 mg and placebo over 72 weeks. The trial achieved its primary endpoint with a statistically significant weight loss at week 72 with semaglutide 7.2 mg versus placebo. From the trial, people treated with semaglutide 7.2 mg achieved a weight loss of 20.7% after 72 weeks compared to a reduction of 17.5% with semaglutide 2.4 mg and 2.4% with placebo. In addition, 33.2% of those who received semaglutide 7.2 mg achieved a weight loss of 25% or more after 72 weeks, compared to 16.7% with semaglutide 2.4 mg and 0.0% with placebo.
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