- |||||||||| Prolia (denosumab) / Amgen, Daiichi Sankyo, Cabometyx (cabozantinib tablet) / Takeda, Exelixis, Ipsen
[VIRTUAL] Are Multiple Brands Used to Charge Higher Prices for Orphan Drug Designations in the US? () - Apr 12, 2021 - Abstract #ISPOR2021ISPOR_257; For one month of treatment, the ratios of prices between orphan and non-orphan indications were: 11.163 for denosumab, 7.724 for everolimus, 2.707 for hydroxyurea, 2.683 for peginterferon alfa-2b, 1.062 for cabozantinib, and 0.930 for tolvaptan (ratios >1 indicate higher cost for the orphan indication)...CONCLUSIONS While certain drugs are significantly more expensive when used in an orphan indication, others do not follow this same pattern. Further research is needed to explore additional drug databases in the US and other countries.
- |||||||||| Cabometyx (cabozantinib tablet) / Takeda, Exelixis, Ipsen
Clinical, Clinical data, Retrospective data, Journal, Real-world evidence: Clinical Outcomes of Metastatic Renal Carcinoma Following Disease Progression to Programmed Death (PD)-1 or PD-L1 Inhibitors (IO): A Meet-URO Group Real World Study (Meet-Uro 7). (Pubmed Central) - Apr 10, 2021 In our real world experience after progression to IO, vascular endotelial groth factor-tyrosine kinase inhibitors, given to patients, proved to be active and safe choices. Cabozantinib was associated with a better outcome in terms of median PFS.
- |||||||||| Avastin (bevacizumab) / Roche
Enrollment closed, Enrollment change, Trial primary completion date, Metastases: Liposomal Doxorubicin, Bevacizumab, and Everolimus in Patients With Locally Advanced TNBC With Tumors Predicted Insensitive to Standard Chemotherapy; A Moonshot Initiative (clinicaltrials.gov) - Apr 2, 2021 P2, N=19, Active, not recruiting, Further evaluation of the combination in advanced RCC patients is ongoing (NCT03095040). Recruiting --> Active, not recruiting | N=37 --> 19 | Trial primary completion date: Jan 2021 --> Jan 2022
- |||||||||| everolimus / Generic mfg.
Clinical, Journal: The mTOR-inhibitor everolimus reduces hypervolemia in patients with primary aldosteronism. (Pubmed Central) - Apr 2, 2021 Recruiting --> Active, not recruiting | N=37 --> 19 | Trial primary completion date: Jan 2021 --> Jan 2022 Everolimus lowers central and peripheral blood pressure in individuals with primary aldosteronism, possibly by decreasing primary aldosteronism-induced hypervolemia and preload.
- |||||||||| cyclophosphamide / Generic mfg., everolimus / Generic mfg., mesna / Generic mfg.
Enrollment open, Trial completion date, Trial primary completion date: LITMMUS-UCSF: Liver Transplantation With Tregs at UCSF (clinicaltrials.gov) - Apr 1, 2021 P1/2, N=9, Recruiting, Everolimus lowers central and peripheral blood pressure in individuals with primary aldosteronism, possibly by decreasing primary aldosteronism-induced hypervolemia and preload. Not yet recruiting --> Recruiting | Trial completion date: Apr 2027 --> Mar 2028 | Trial primary completion date: Apr 2027 --> Apr 2025
- |||||||||| everolimus / Generic mfg.
Journal: Two novel TSC2 mutations in renal epithelioid angiomyolipoma sensitive to everolimus. (Pubmed Central) - Mar 30, 2021 After months of treatment with everolimus, Computer-Tomography (CT) scan results showed that everolimus successfully reduced tumor growth and distal metastasis and achieved partial response (PR) to everolimu according to Response Evaluation Criteria in Solid Tumors (RECIST version 1.1). Further Blood Routine Examination results showed the concentration of red cell mass, hemoglobin, white blood cell (WBC), platelets and hematocrit (HCT) significantly returned to normal levels indicating patients with these two TSC2 mutations could be effectively treated by everolimus.
- |||||||||| fulvestrant / Generic mfg.
Journal, IO biomarker: Practical Treatment Strategies and Future Directions After Progression While Receiving CDK4/6 Inhibition and Endocrine Therapy in Advanced HR/HER2 Breast Cancer. (Pubmed Central) - Mar 30, 2021 Everolimus-based combinations and chemotherapy appear to have continued efficacy after progression while receiving CDK4/6i, although historical data on benefit include CDK4/6i-naive patients...Tumor biopsy with genomic sequencing and repeat biomarker analysis in patients with CDK4/6i- and endocrine-resistant disease will be integral to guide subsequent treatment strategies and to inform clinical trial eligibility. Promising novel therapeutics in CDK4/6i-resistant disease including oral selective estrogen down-regulators, fibroblast growth factor receptor antagonists, and immunotherapy will be discussed.
- |||||||||| everolimus / Generic mfg.
Clinical, P2 data, Journal, Combination therapy: A Phase II Open Label Study of Everolimus in Combination With Endocrine Therapy in Resistant Hormone Receptor-Positive HER2-Negative Advanced Breast Cancer. (Pubmed Central) - Mar 30, 2021 Promising novel therapeutics in CDK4/6i-resistant disease including oral selective estrogen down-regulators, fibroblast growth factor receptor antagonists, and immunotherapy will be discussed. After progression during antiestrogen therapy, the addition of everolimus, without changing the hormonal therapy, resulted in a median PFS of 6.6 months, suggesting efficacy in patients with HR/HER2 MBC.
- |||||||||| temozolomide / Generic mfg.
[VIRTUAL] Advanced small intestine well-differentiated neuroendocrine tumors (WD-SiNET): A survey of practice on 3rd line treatment () - Mar 27, 2021 - Abstract #ENETS2021ENETS_265; Overall, 3rd-line treatment for WD-SiNETs was: everolimus (EVE) (66.7%), PRRT (18.5%), liver embolization (LE) (7.4%) and interferon (IFN) (3.7%); chemotherapy (0%); decision was based on clinical trial data (59.3%) or personal experience (22.2%)...Chemotherapy was chosen if rapid progression (within 6 months) (WA 3.35/4), Ki-67 10-20% (WA 2.77/4), negative SSTR2 imaging (WA 2.65/4) or high tumor burden (WA 2.77/4); temozolomide or streptozocin was used with capecitabine or 5-FU (57.7%), FOLFOX (23.1%)... Selection of 3rd line therapy is based on multiple factors mainly Ki-67, rate of progression, CS and tumor burden; decisions should be made within a multidisciplinary setting.
- |||||||||| everolimus / Generic mfg.
Journal: Molecular Genetic Landscape of Sclerosing Pneumocytomas. (Pubmed Central) - Mar 27, 2021 Our data further support that abnormal activation of the mTOR pathway is a consistent genetic event in sclerosing pneumocytoma. This warrants further exploration to determine if mTOR pathway inhibitors may be effective in patients with metastatic or recurrent disease.
- |||||||||| everolimus / Generic mfg.
Journal: Response to Everolimus of a Progressive Plexiform Neurofibroma in NF type 1. (Pubmed Central) - Mar 26, 2021 In patients known to be prone to mechanistic target of rapamycin inhibitor toxicity, preemptive reduction in dose may be warranted upon initiation of cannabidiol. mTOR inhibitors may represent a treatment option to promote regression of PNs associated to NF1.
- |||||||||| camizestrant (AZD9833) / AstraZeneca
New P1 trial, Combination therapy, Metastases: AZD9833 China PK Study (clinicaltrials.gov) - Mar 25, 2021 P1, N=42, Not yet recruiting,
- |||||||||| xentuzumab (BI-836845) / Boehringer Ingelheim
Enrollment closed, Combination therapy, Metastases: The XENERA (clinicaltrials.gov) - Mar 23, 2021 P2, N=103, Active, not recruiting, The study demonstrated the safety of everolimus and lenalidomide with promising efficacy signal in thyroid and adenoid cystic cancers. Recruiting --> Active, not recruiting
- |||||||||| everolimus / Generic mfg.
Journal: Tsc1 Regulates the Proliferation Capacity of Bone-Marrow Derived Mesenchymal Stem Cells. (Pubmed Central) - Mar 23, 2021 In young (28 day old) mice, Tsc1 deficiency-induced significant cell expansion of non-hematopoietic BM in vivo, and MSC colony-forming potential in vitro, that was normalized upon treatment with the mTOR inhibitor, everolimus...ShRNA-mediated knockdown of Tsc1 in BM-MSCs replicated the hyperproliferative BM-MSC phenotype and led to impaired adipogenic and myogenic differentiation. Our data show that Tsc1 is a negative regulator of BM-MSC proliferation and support a pivotal role for the Tsc1-mTOR axis in the maintenance of the mesenchymal progenitor pool.
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