- |||||||||| efimosfermin alfa (GSK6519754) / GSK, efruxifermin (AKR-001) / Akero Therap, pegozafermin (BIO89-100) / 89Bio
Clinical data, Preclinical, Journal: FGF21 Analogues and MASLD: A Summary of Preclinical and Clinical Data. (Pubmed Central) - Aug 6, 2025 Among various others, efruxifermin, pegozafermin, pegbelfermin, and BOS-580 are FGF-21 analogues that have resulted in significant improvements in liver fat, fibrosis, and measures of liver function in the context of phase 2 clinical trials. This review summarizes the preclinical and clinical data from FGF21 analogues for MASLD and MASH.
- |||||||||| efimosfermin alfa (BOS-580) / Boston Pharma, Rezdiffra (resmetirom) / Madrigal Pharma
4200: SOA - Update on MASLD Therapies Pipeline () - Mar 8, 2025 - Abstract #DDW2025DDW_4810; Description: This State-of-the-Art session provides a timely update on the evolving MASLD therapies pipeline, featuring insights into emerging pharmacologic agents and real-world data on current treatments. Topics include the impact of SGLT-2 and GLP-1 receptor agonists on cirrhosis outcomes, eligibility criteria for resmetirom therapy, and phase 2 results for efimosfermin alfa in MASH with fibrosis.Learning Objectives:
- |||||||||| Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex, BOS-318 / Boston Pharma
Journal: BOS-318 treatment enhances elexacaftor-tezacaftor-ivacaftor-mediated improvements in airway hydration and mucociliary transport. (Pubmed Central) - Feb 27, 2025 This altered ion transport profile effected an improved ASL height and MCT rate, which were significantly greater than improvements observed with ETI alone, demonstrating the benefits of the dual approach. Selective furin inhibition has the potential to further improve clinical outcomes for all people with CF and offers opportunity as an adjunct to improve responses to currently available CFTR modulator therapies.
- |||||||||| BOS-318 / Boston Pharma
BOS-318 attenuates Pseudomonas aeruginosa lung infection in mouse models () - Sep 4, 2024 - Abstract #NACFC2024NACFC_805; Our study introduces furin inhibition as a novel mechanism for anti-Pseudomonal therapy in animal models, protecting the lungs from injury and promoting bacterial clearance, which is highly applicable to CF lung disease and nosocomial pneumonia. Further research will target the mechanisms underlying this protective effect and extend to non-P.
- |||||||||| SLX-0528 / Xenthera, Biolexis Therap
Trial completion date, Trial primary completion date: Subjects With Advanced or Metastatic Solid Tumor Malignancies (clinicaltrials.gov) - Jul 31, 2024 P1, N=30, Not yet recruiting, Active, not recruiting --> Recruiting | Trial completion date: Dec 2024 --> Jul 2026 | Trial primary completion date: Dec 2024 --> Jul 2026 Trial completion date: Jun 2027 --> Jun 2028 | Trial primary completion date: Jan 2027 --> Jan 2028
- |||||||||| Review, Journal: Drugs of the future for diarrhea-predominant irritable bowel syndrome: an overview of current investigational drugs. (Pubmed Central) - Mar 11, 2024
Among the potential therapies, ibodutant, ibudilast, blautix, BOS-589, solabegron, vibegron, olorinab, ebastine, and ORP-101 have demonstrated possible effects but remain confirmed...Therefore, we should focus on developing new, efficient, and affordable medications for IBS-D. The government, insurers, and society must recognize this need and collaborate to ensure its fulfillment.
- |||||||||| Genomic mechanisms of RET inhibitor resistance in RET-fusion positive NSCLC (Section 23) - Mar 5, 2024 - Abstract #AACR2024AACR_8077;
Collectively, we show that individual RET fusion variants have distinct drug sensitivity profiles and that secondary resistance mutations are non-overlapping between RETi. The comprehensive characterization of RET-dependent mechanisms of resistance to RETi may provide therapeutic guidance for treating RET-fusion driven NSCLC and provide structural insights that can guide the development of new therapeutic regimens.
- |||||||||| SLX-0528 / Xenthera, Biolexis Therap
Trial completion date, Trial primary completion date, Metastases: Subjects With Advanced or Metastatic Solid Tumor Malignancies (clinicaltrials.gov) - Nov 1, 2023 P1, N=30, Not yet recruiting, Phase classification: P2a --> P2 Trial completion date: Jun 2026 --> Jun 2027 | Trial primary completion date: Jan 2026 --> Jan 2027
- |||||||||| zeteletinib (BOS-172738) / Boston Pharma
Trial completion, Trial completion date, Trial primary completion date, Metastases: Safety, Efficacy, and Tolerability of BOS172738 in Patients With Advanced Rearranged During Transfection (RET) Gene-Altered Tumors (clinicaltrials.gov) - Oct 30, 2023 P1, N=117, Completed, Trial completion date: Jun 2026 --> Jun 2027 | Trial primary completion date: Jan 2026 --> Jan 2027 Active, not recruiting --> Completed | Trial completion date: Aug 2024 --> Sep 2023 | Trial primary completion date: Mar 2024 --> Sep 2023
- |||||||||| BOS-318 / Boston Pharma
Journal: W254 in furin functions as a molecular gate promoting anti-viral drug binding: Elucidation of putative drug tunneling and docking by non-equilibrium molecular dynamics. (Pubmed Central) - Oct 11, 2023 Recent experimental evidence indicated that dichlorophenyl (DCP)-pyridine "BOS" drugs (e.g., BOS-318) competitively inhibit human furin by an induced-fit mechanism in which tryptophan W254 in the furin catalytic cleft (FCC) functions as a molecular gate, rotating nearly 180 through a steep energy barrier about its chi-1 dihedral to an "open" orientation, exposing a buried (i.e., cryptic) hydrophobic pocket...Finally, interactive molecular dynamics (iMD) revealed two putative conduits of drug entry and binding into the FCC, each coupled with W254 dihedral rotation and opening of the cryptic pocket. The iMD simulations further revealed ligand entry and binding in the FCC is likely driven in part by energy fluxes stemming from disruption and re-formation of ligand and protein solvation shells during drug migration from the solution phase into the FCC.
- |||||||||| SLX-0528 / Xenthera, Biolexis Therap
Trial initiation date, Metastases: Subjects With Advanced or Metastatic Solid Tumor Malignancies (clinicaltrials.gov) - Jul 12, 2023 P1, N=30, Not yet recruiting, The iMD simulations further revealed ligand entry and binding in the FCC is likely driven in part by energy fluxes stemming from disruption and re-formation of ligand and protein solvation shells during drug migration from the solution phase into the FCC. Initiation date: May 2023 --> Nov 2023
- |||||||||| SLX-0528 / Xenthera, Biolexis Therap
Trial completion date, Trial initiation date, Trial primary completion date, Metastases: Subjects With Advanced or Metastatic Solid Tumor Malignancies (clinicaltrials.gov) - Mar 16, 2023 P1, N=30, Not yet recruiting, A Phase 1 trial is currently in development. Trial completion date: Oct 2025 --> Jun 2026 | Initiation date: Sep 2022 --> May 2023 | Trial primary completion date: Aug 2025 --> Jan 2026
- |||||||||| BOS-580 / Boston Pharma, efruxifermin (AKR-001) / Amgen
Review, Journal: Fibroblast Growth Factor-21 as a Potential Therapeutic Target of Nonalcoholic Fatty Liver Disease. (Pubmed Central) - Jan 31, 2023 However, the definite effect of FGF-21 on NAFLD may be clarified after the completion of the ongoing clinical trials with paired liver biopsies and histological endpoints. The aim of this review is to critically summarize experimental and clinical data of FGF-21 in NAFLD, in an attempt to highlight existing knowledge and areas of uncertainty, and subsequently, to focus on the potential therapeutic effects of FGF-21 and its analogs in NAFLD.
- |||||||||| efimosfermin alfa (GSK6519754) / GSK
Enrollment closed: BOS-580-201: A Study of BOS-580 in Obese Subjects at Risk for, or With Biopsy-confirmed, Nonalcoholic Steatohepatitis (NASH) With an Extension (clinicaltrials.gov) - Sep 27, 2022 P2a, N=102, Active, not recruiting, The aim of this review is to critically summarize experimental and clinical data of FGF-21 in NAFLD, in an attempt to highlight existing knowledge and areas of uncertainty, and subsequently, to focus on the potential therapeutic effects of FGF-21 and its analogs in NAFLD. Recruiting --> Active, not recruiting
- |||||||||| zeteletinib (BOS-172738) / Boston Pharma
Trial completion date, Trial primary completion date, Metastases: Safety, Efficacy, and Tolerability of BOS172738 in Patients With Advanced Rearranged During Transfection (RET) Gene-Altered Tumors (clinicaltrials.gov) - Sep 26, 2022 P1, N=117, Active, not recruiting, Recruiting --> Active, not recruiting Trial completion date: Dec 2022 --> Apr 2023 | Trial primary completion date: Dec 2022 --> Apr 2023
- |||||||||| BOS-318 / Boston Pharma
Journal: A highly selective, cell-permeable furin inhibitor BOS-318 rescues key features of cystic fibrosis airway disease. (Pubmed Central) - Jun 22, 2022 Furin inhibition also protected ENaC from subsequent activation by neutrophil elastase, a soluble protease dominant in CF airways. Additional therapeutic benefits include protection against epithelial cell death induced by Pseudomonas aeruginosa exotoxin A. Our findings demonstrate the utility of selective furin inhibition as a mutation-agnostic approach that can correct features of CF airway pathophysiology in a manner expected to deliver therapeutic value.
- |||||||||| BOS-318 / Boston Pharma
Journal: Clearing the air: Uniquely engaging furin as an approach to cystic fibrosis therapy. (Pubmed Central) - Jun 22, 2022 describe a potent, specific, and cell-permeable furin inhibitor that interacts with a cryptic binding site to rescue hallmarks of cystic fibrosis in human ex vivo models. BOS-318 holds promise for development of therapeutics targeting an array of furin-dependent pathologies.
- |||||||||| BOS-589 / GSK, Boston Pharma
Clinical, P1 data, PK/PD data, Journal: First-in-Human Study for a Selective Rearranged During Transfection Tyrosine Kinase Inhibitor, GSK3352589, to Investigate the Safety, Tolerability, and Pharmacokinetics in Healthy Volunteers. (Pubmed Central) - Mar 25, 2022 Accumulation ratios for C , AUC , AUC , and AUC after twice-daily dosing to steady state ranged from 1.2 to 1.8 for all doses except the 200-mg dose, which had ratios between 1.9 and 2.7. Administration of GSK3352589 after SD and RD was well tolerated with no safety concerns in healthy subjects.
- |||||||||| BOS-580 / Boston Pharma
Clinical, Journal: LLF580, an FGF21 Analog, Reduces Triglycerides and Hepatic Fat in obese adults with modest hypertriglyceridemia. (Pubmed Central) - Feb 15, 2022 P1 In obese, mildly hypertriglyceridemic adults, LLF580 was generally safe and demonstrated beneficial effects on serum lipids, liver fat and biomarkers of liver injury, suggesting it may be effective for treatment of select metabolic disorders including hypertriglyceridemia and non-alcoholic fatty liver disease. Assessments of longer-term safety and efficacy are warranted.
- |||||||||| zeteletinib (BOS-172738) / Boston Pharma
Enrollment closed, Trial completion date, Metastases: Safety, Efficacy, and Tolerability of BOS172738 in Patients With Advanced Rearranged During Transfection (RET) Gene-Altered Tumors (clinicaltrials.gov) - Sep 16, 2021 P1, N=117, Active, not recruiting, Not yet recruiting --> Recruiting Recruiting --> Active, not recruiting | Trial completion date: Dec 2021 --> Jun 2022
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