1. KOSELUGO shows significant response improvement in neurofibromatosis type 1 patients
Alexion, AstraZeneca Rare Disease and Merck announced positive results from a global Phase 3 KOMET trial investigating the effects of KOSELUGO (selumetinib) on neurofibromatosis type 1 patients who have symptomatic, inoperable plexiform neurofibromas. Neurofibromatosis type 1 is a rare, progressive genetic condition that causes skin pigment changes and nerve tissue tumors. The study has met its primary endpoint in achieving statistically significant and clinically meaningful improvement in the overall response rate compared to the placebo group.
https://www.astrazeneca.com/media-centre/press-releases/2024/komet-phase-iii-trial-met-primary-endpoint.html
2. New indication of KEYTRUDA received positive recommendation from CHMP of EMA
European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended the approval of KEYTRUDA ® (pembrolizumab), in combination with pemetrexed and platinum chemotherapy for the first line treatment of unresectable non-epithelioid malignant pleural mesothelioma. Malignant mesothelioma is a thin tissue (mesothelium) cancer that lines the lung, chest wall, and abdomen. This recommendation was based on Phase 2/3 IND.227/KEYNOTE-483 trial results. The combination therapy was able to reduce the risk of death by around 20% compared to the platinum chemotherapy-only arm. Both overall survival (17.3 months vs. 16.1 months) and overall response rate (52% vs. 29%) were higher in the trial group.
Source: https://www.merck.com/news/merck-receives-positive-eu-chmp-opinion-for-keytruda-pembrolizumab-plus-chemotherapy-as-first-line-treatment-for-adult-patients-with-unresectable-non-epithelioid-malignant-pleural-mesothelioma/
3. Two new drugs from BMS with new indications have granted recommendation approval from CHMP
Bristol Myers Squibb announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended approval for two drugs with new indications. Firstly, repotrectinib was recommended to treat patients with ROS1+ advanced non-small cell lung cancer (NSCLC) and adult or pediatric patients 12 years of age and older with advanced solid tumors expressing an NTRK gene fusion. It is supported by results from the TRIDENT-1 and CARE trials, where the drug demonstrated clinically meaningful response rates. Another granted approval was towards the Opdivo ® (nivolumab) plus Yervoy ® (ipilimumab) for the first-line treatment of adult patients with microsatellite instability-high or mismatch repair deficient unresectable or metastatic colorectal cancer. This is supported by results from the CheckMate -8HW trial where a 79% reduction in the risk of disease progression or death was identified. Both drugs require further EMA approval to be officially indicated in Europe for the mentioned symptoms.
4. Nipocalimab largely alleviate the moderate-to-severe Sjögren’s disease symptoms
Johnson & Johnson recently announced additional results from the Phase 2 DAHLIAS study showing the efficacy of nipocalimab in adult patients with moderate-to-severe Sjögren’s disease (SjD). The primary endpoint was achieved with the significant improvement in the ClinESSDAIa score at 24 weeks, meanwhile, key secondary endpoints were also met by reducing disease activity both systemically and across multiple organ systems. There were also significant IgG reductions observed after the treatment. This therapy has also been rewarded with Breakthrough Therapy designation from the U.S. FDA recently.
Source: https://www.jnj.com/media-center/press-releases/nipocalimab-demonstrates-significant-clinical-improvement-in-disease-activity-and-igg-reduction-in-phase-2-sjogrens-disease-study
5. CHMP positive opinions granted to new anti-lung cancer drug regime
European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the marketing authorization for Johnson & Johnson’s LAZCLUZE® (lazertinib), in combination with RYBREVANT® (amivantamab), for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or exon 21 L858R (L858R) substitution mutations. From the Phase 3 MARIPOSA study, amivantamab in combination with Lazertinib has reduced the risk of disease progression or death by 30 percent compared to Osimertinib. And the median duration of response was also longer in the combination therapy arm.
Source: https://www.jnj.com/media-center/press-releases/chmp-recommends-rybrevant-amivantamab-in-combination-with-lazcluze-lazertinib-for-the-first-line-treatment-of-patients-with-egfr-mutated-advanced-non-small-cell-lung-cancer
6. Announcement of two clinical studies associated with controlling diabetes and heart failures by tirzepatide
Eli Lilly and Company announced results from the Phase 3 SURMOUNT-1 three-year study which is the current longest completed study of tirzepatide. Weekly tirzepatide (Zepbound® and Mounjaro®) injections (5 mg, 10 mg, 15 mg doses) significantly reduced the risk of progression to type 2 diabetes in adults with pre-diabetes and obesity or overweight, compared to the placebo group. Individuals treated with tirzepatide lost on average up to 23% and are able to sustain over the trial period, which reduces 94% of risk in progressing to type 2 diabetes. The study also showed benefits in glycemic control and improvements in cardiometabolic risk factors.
In another SUMMIT Phase 3 study, tirzepatide was found to significantly reduce the risk of worsening heart failure events in adults with heart failure with preserved ejection fraction (HFpEF) and obesity. There was a 38% reduction in the risk of heart failure and 56% reduction in the risk of hospitalization. Several key secondary endpoints were also improved including exercise capacity, body weight and systemic inflammation level.
Previously, Tirzepatide was approved by the U.S. FDA as Mounjaro® for adults with type 2 diabetes to improve glycemic control on May 13, 2022, and as Zepbound® for adults with obesity or those who are overweight who also have at least one weight-related medical problem on November 8, 2023.
Source: https://investor.lilly.com/news-releases/news-release-details/lillys-tirzepatide-reduced-risk-worsening-heart-failure-events;
https://investor.lilly.com/news-releases/news-release-details/treatment-tirzepatide-adults-pre-diabetes-and-obesity-or
7. Livdelzi ® (seladelpar) successfully manages primary biliary cholangitis conditions
Gilead Sciences recently announced a two-and-a-half-year interim analysis from the ongoing Phase 3 ASSURE study. The study is evaluating the long-term effects of Livdelzi ® (seladelpar) on patients with primary biliary cholangitis (PBC). Currently, there are around 500 participants recruited from 160 sites. Results showed that 81% (30 out of 37) of participants with primary biliary cholangitis receiving Livdelzi ® (seladelpar) treatment achieved a composite biochemical response, demonstrating significant improvements in key measures of PBC progression. Additionally, 41% (15 out of 37) of participants achieved normalization of alkaline phosphatase levels, which is a critical biomarker of liver function. PBC is an autoimmune disease in which the bile ducts are inflamed and slowly destroyed. Livdelzi ® (seladelpar) is an oral PPAR-delta agonist and PPAR-delta is a critical component in regulating metabolic and liver disease pathways. Current results supported sustained efficacy and long-term safety in managing the primary biliary cholangitis condition.
Source: https://www.gilead.com/news/news-details/2024/gileads-livdelzi-seladelpar-demonstrated-a-sustained-efficacy-and-long-term-safety-profile-in-management-of-primary-biliary-cholangitis
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