Expert Opinion on Investigational Drugs

Article	Citations
Brain insulin resistance: role in neurodegenerative disease and potential for targeting	89
PD-L1, TMB, and other potential predictors of response to immunotherapy for hepatocellular carcinoma: how can they assist drug clinical trials?	74
Durvalumab: an investigational anti-PD-L1 antibody for the treatment of biliary tract cancer	74
Biomarkers for breast cancer immunotherapy: PD-L1, TILs, and beyond	68
Faricimab: an investigational agent targeting the Tie-2/angiopoietin pathway and VEGF-A for the treatment of retinal diseases	67
Atezolizumab-bevacizumab plus Y-90 TARE for the treatment of hepatocellular carcinoma: preclinical rationale and ongoing clinical trials	65
Bile acid modulators for the treatment of nonalcoholic steatohepatitis (NASH)	63
Futibatinib, an investigational agent for the treatment of intrahepatic cholangiocarcinoma: evidence to date and future perspectives	60
FAK-targeted and combination therapies for the treatment of cancer: an overview of phase I and II clinical trials	59
Lenvatinib plus pembrolizumab: the next frontier for the treatment of hepatocellular carcinoma?	51
Carbonic anhydrase inhibitors: an update on experimental agents for the treatment and imaging of hypoxic tumors	51
Immune-based combinations for metastatic triple negative breast cancer in clinical trials: current knowledge and therapeutic prospects	47
Antisense oligonucleotide therapeutics in clinical trials for the treatment of inherited retinal diseases	44
The nonalcoholic steatohepatitis (NASH) drug development graveyard: established hurdles and planning for future success	43
The role of Eph receptors in cancer and how to target them: novel approaches in cancer treatment	41
Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies	39
Latest generation estrogen receptor degraders for the treatment of hormone receptor-positive breast cancer	38
Risuteganib—a novel integrin inhibitor for the treatment of non-exudative (dry) age-related macular degeneration and diabetic macular edema	38
Linzagolix: a new GnRH-antagonist under investigation for the treatment of endometriosis and uterine myomas	37
Tislelizumab: an investigational anti-PD-1 antibody for the treatment of advanced non-small cell lung cancer (NSCLC)	37
ELX-02: an investigational read-through agent for the treatment of nonsense mutation-related genetic disease	36
Oral Ibrexafungerp: an investigational agent for the treatment of vulvovaginal candidiasis	36
Novel emerging therapies in atherosclerosis targeting lipid metabolism	36
Pamrevlumab for the treatment of idiopathic pulmonary fibrosis	35
Investigational IRAK-4 inhibitors for the treatment of rheumatoid arthritis	35

Inhibition of Bruton´s tyrosine kinase as a novel therapeutic approach in multiple sclerosis	35
The anti-DKK1 antibody DKN-01 as an immunomodulatory combination partner for the treatment of cancer	34
Epilepsy: key experimental therapeutics in early clinical development	33
Glucagon-like peptide-1 (GLP-1)-based receptor agonists as a treatment for Parkinson’s disease	33
Infigratinib (BGJ398): an investigational agent for the treatment of FGFR-altered intrahepatic cholangiocarcinoma	32
BIO 300: a promising radiation countermeasure under advanced development for acute radiation syndrome and the delayed effects of acute radiation exposure	31
New targets and therapeutics for neuroprotection, remyelination and repair in multiple sclerosis	31
Givinostat: an emerging treatment for polycythemia vera	31
Ladiratuzumab vedotin for metastatic triple negative cancer: preliminary results, key challenges, and clinical potential	31
Investigational drugs in early phase clinical trials targeting thermotransient receptor potential (thermoTRP) channels	30
Anti-integrin therapy for retinovascular diseases	30
Zilucoplan: An Investigational Complement C5 Inhibitor for the Treatment of Acetylcholine Receptor Autoantibody–Positive Generalized Myasthenia Gravis	29
Immunotherapies in clinical development for biliary tract cancer	28
CAR-T cell therapy for triple-negative breast cancer and other solid tumors: preclinical and clinical progress	28
Proteasome inhibition for the treatment of glioblastoma	27
New promising drugs for the treatment of systemic sclerosis: pathogenic considerations, enhanced classifications, and personalized medicine	27
Progress and pitfalls in the use of immunotherapy for patients with triple negative breast cancer	27
Emerging agents for the treatment of Chagas disease: what is in the preclinical and clinical development pipeline?	27
Fezolinetant in the treatment of vasomotor symptoms associated with menopause	26
Selective androgen receptor modulators (SARMs) as pharmacological treatment for muscle wasting in ongoing clinical trials	26
CTLA4-Ig (abatacept): a promising investigational drug for use in type 1 diabetes	26
Current and experimental drug therapy for the treatment of polycystic ovarian syndrome	26
Monoclonal antibody therapies in the management of SARS-CoV-2 infection	25
Targeting pituitary adenylate cyclase-activating polypeptide (PACAP) with monoclonal antibodies in migraine prevention: a brief review	25
Differentiation between emerging non-steroidal and established steroidal mineralocorticoid receptor antagonists: head-to-head comparisons of pharmacological and clinical characteristics	24
Selatogrel, a novel P2Y12 inhibitor: a review of the pharmacology and clinical development	24
The holistic approach to psoriasis patients with comorbidities: the role of investigational drugs	23
Neoantigen vaccine platforms in clinical development: understanding the future of personalized immunotherapy	23
MK-6482 as a potential treatment for von Hippel-Lindau disease-associated clear cell renal cell carcinoma	23
Huntingtin-lowering strategies for Huntington’s disease	22
Repurposing elesclomol, an investigational drug for the treatment of copper metabolism disorders	22
Investigational drugs for the treatment of diffuse large B-cell lymphoma	22
Therapies in preclinical and clinical development for Angelman syndrome	22
Invasive candidiasis: investigational drugs in the clinical development pipeline and mechanisms of action	21
Discontinued disease-modifying therapies for Alzheimer’s disease: status and future perspectives	21
Gene-based therapy in lipid management: the winding road from promise to practice	20
Immunization therapies for Parkinson’s disease: state of the art and considerations for future clinical trials	20
Bempedoic acid, an inhibitor of ATP citrate lyase for the treatment of hypercholesterolemia: early indications and potential	19
Lecanemab (BAN2401): an anti–beta-amyloid monoclonal antibody for the treatment of Alzheimer disease	19
PARP inhibitors as single agents and in combination therapy: the most promising treatment strategies in clinical trials for BRCA-mutant ovarian and triple-negative breast cancers	19
AE37: a HER2-targeted vaccine for the prevention of breast cancer recurrence	19
Targeting FGFR in intrahepatic cholangiocarcinoma [iCCA]: leading the way for precision medicine in biliary tract cancer [BTC]?	19
The dawn of targeted therapies for triple negative breast cancer (TNBC): a snapshot of investigational drugs in phase I and II trials	17
Translesion synthesis inhibitors as a new class of cancer chemotherapeutics	17
Progress in the development of kynurenine and quinoline-3-carboxamide-derived drugs	17
The time to offer treatments for COVID-19	17
Trastuzumab-deruxtecan: an investigational agent for the treatment of HER2-positive breast cancer	17
Asciminib: an investigational agent for the treatment of chronic myeloid leukemia	16
Anti-diabetic drugs and NASH: from current options to promising perspectives	16
Innovative therapies for acute bacterial skin and skin-structure infections (ABSSSI) caused by methicillin-resistant Staphylococcus aureus: advances in phase I and II trials	16

Pyoderma gangrenosum: proposed pathogenesis and current use of biologics with an emphasis on complement C5a inhibitor IFX-1	16
The emerging role of immune checkpoint inhibitors for the treatment of breast cancer	16
CD38: targeted therapy in multiple myeloma and therapeutic potential for solid cancers	16
Melflufen for relapsed and refractory multiple myeloma	15
Aromatase inhibitors for the treatment of endometriosis: a systematic review about efficacy, safety and early clinical development	15
Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA)	15
Selective α7 nicotinic receptor agonists and positive allosteric modulators for the treatment of schizophrenia – a review	15
Sprifermin: a recombinant human fibroblast growth factor 18 for the treatment of knee osteoarthritis	15
Immunotherapy for gliomas: shedding light on progress in preclinical and clinical development	14
Investigational spleen tyrosine kinase (SYK) inhibitors for the treatment of autoimmune diseases	14
Type I interferon antagonists in clinical development for lupus	14
Immunotherapy in uveal melanoma: novel strategies and opportunities for personalized treatment	14
Overview on the use of IL-6 agents in the treatment of patients with cytokine release syndrome (CRS) and pneumonitis related to COVID-19 disease	14
Therapeutic interventions for spinal muscular atrophy: preclinical and early clinical development opportunities	14
Investigational therapies targeting CD3 for prevention and treatment of type 1 diabetes	14
Ipilimumab: an investigational immunotherapy for glioblastoma	14
Abicipar pegol: an investigational anti-VEGF agent for the treatment of wet age-related macular degeneration	14
Polatuzumab vedotin: an investigational anti-CD79b antibody drug conjugate for the treatment of diffuse large B-cell lymphoma	14
Breakthrough therapy designation of nirsevimab for the prevention of lower respiratory tract illness caused by respiratory syncytial virus infections (RSV)	13
Emerging myelin repair agents in preclinical and early clinical development for the treatment of multiple sclerosis	13
Investigational non-JAK inhibitors for chronic phase myelofibrosis	13
Dopamine β hydroxylase as a potential drug target to combat hypertension	13
Bladder cancer: shedding light on the most promising investigational drugs in clinical trials	13
Investigational drugs and nutrients for human longevity. Recent clinical trials registered in ClinicalTrials.gov and clinicaltrialsregister.eu	13
Targeting the tumor microenvironment in cholangiocarcinoma: implications for therapy	13
Immune checkpoint inhibitors for the treatment of myeloma: novel investigational options	13
Teprotumumab: a novel therapeutic monoclonal antibody for thyroid-associated ophthalmopathy	13
A case study of foliglurax, the first clinical mGluR4 PAM for symptomatic treatment of Parkinson’s disease: translational gaps or a failing industry innovation model?	12
Investigational drugs in clinical trials for macular degeneration	12
Systemic therapy of advanced/metastatic gastrointestinal stromal tumors: an update on progress beyond imatinib, sunitinib, and regorafenib	12
Post-pancreatitis diabetes mellitus: investigational drugs in preclinical and clinical development and therapeutic implications	12
Lorecivivint, an intra-articular potential disease-modifying osteoarthritis drug	12
Emerging agents for the treatment and prevention of stroke: progress in clinical trials	12
Emerging therapies for COVID-19 pneumonia	12
An update on investigational therapies that target STAT3 for the treatment of cancer	11
Poly (ADP-ribose) polymerase (PARP) as target for the treatment of epithelial ovarian cancer: what to know	11
Precision approaches for cholangiocarcinoma: progress in clinical trials and beyond	11
Mavacamten: a novel small molecule modulator of β-cardiac myosin for treatment of hypertrophic cardiomyopathy	11
What have we learned from past failures of investigational drugs for Alzheimer’s disease?	11
Novel systemic treatment approaches for metastatic pancreatic cancer	11
Tackling type 2 diabetes-associated cardiovascular and renal comorbidities: a key challenge for drug development	11
JAK inhibitors for rheumatoid arthritis	11
Telacebec: an investigational antibacterial for the treatment of tuberculosis (TB)	11
New immunotherapeutic drugs in advanced non-small cell lung cancer (NSCLC): from preclinical to phase I clinical trials	11
Targeting Ebola virus replication through pharmaceutical intervention	11
Low-dose aspirin for early COVID-19: does the early bird catch the worm?	11
Drugs in phase I and phase II clinical trials for systemic sclerosis	10
Novel therapeutic agents for the treatment of diabetic kidney disease	10
TP53 dysfunction in chronic lymphocytic leukemia: clinical relevance in the era of B-cell receptors and BCL-2 inhibitors	10
New treatment approaches for Alzheimer’s disease: preclinical studies and clinical trials centered on antidiabetic drugs	10
Current emerging and investigational drugs for the treatment of chronic hand eczema	10
Selective endothelin A receptor antagonism in patients with proteinuric chronic kidney disease	10
Therapeutic angiogenesis in coronary artery disease: a review of mechanisms and current approaches	10
An evaluation of KBP-5074 in advanced chronic kidney disease with uncontrolled hypertension	10
Durvalumab: an investigational agent for unresectable hepatocellular carcinoma	9
Cholangiocarcinoma: bridging the translational gap from preclinical to clinical development and implications for future therapy	9
Investigational neuroprotective compounds in clinical trials for retinal disease	9
Emerging treatments for chronic urticaria	9
Remyelination therapies for multiple sclerosis: optimizing translation from animal models into clinical trials	9
Safety and pharmacokinetics of a biosimilar of denosumab (KN012): Phase 1 and bioequivalence study in healthy Chinese subjects	9
Investigational drugs for assisting psychotherapy for posttraumatic stress disorder (PTSD): emerging approaches and shifting paradigms in the era of psychedelic medicine	9
Understanding drug resistance mechanisms in cholangiocarcinoma: assisting the clinical development of investigational drugs	9
Derazantinib: an investigational drug for the treatment of cholangiocarcinoma	9
Cabozantinib plus atezolizumab for the treatment of advanced hepatocellular carcinoma: shedding light on the preclinical rationale and clinical trials	9
Oregovomab: an investigational agent for the treatment of advanced ovarian cancer	9
Baricitinib: therapeutic potential for moderate to severe atopic dermatitis	9
Batten disease: an expert update on agents in preclinical and clinical trials	9
Angiogenesis in biliary tract cancer: targeting and therapeutic potential	9
Immune checkpoint inhibitors for the treatment of sepsis:insights from preclinical and clinical development	9
Safety, pharmacokinetics and pharmacodynamics of TQ-A3334, an oral toll-like receptor 7 agonist in healthy individuals	9
Hypoxia-inducible factor prolyl hydroxylase inhibitors: a paradigm shift for treatment of anemia in chronic kidney disease?	9
Rare cancer, rare alteration: the case of NTRK fusions in biliary tract cancers	9
Ulcerative colitis: shedding light on emerging agents and strategies in preclinical and early clinical development	9
Cystic fibrosis – Ten promising therapeutic approaches in the current era of care	8
A randomized phase I clinical trial comparing the pharmacokinetic, safety, and immunogenicity of potential biosimilar recombinant human HER2 monoclonal antibody for injection and trastuzumab in health	8
Drugs in phase I and II clinical development for the prevention of stroke in patients with atrial fibrillation	8
Targeted therapies for gallbladder cancer: an overview of agents in preclinical and clinical development	8
Anti-integrin drugs in clinical trials for inflammatory bowel disease (IBD): insights into promising agents	8
Still grasping at straws: donanemab in Alzheimer’s disease	8
Clinical complexity of utilizing FGFR inhibitors in cancer therapeutics	8

A randomized, adaptive design, double-blind, 3-arm, parallel study assessing the pharmacokinetics and safety of AVT02, a high-concentration (100 mg/mL) Adalimumab biosimilar, in healthy adult subjects	8
Circadian rhythms in diabetic retinopathy: an overview of pathogenesis and investigational drugs	8
Investigational immunomodulatory drugs for enhancement of triple negative breast cancer (TNBC) immunotherapy: early phase development	8
Remdesivir, a remedy or a ripple in severe COVID-19?	8
Shedding light on developmental drugs for idiopathic pulmonary fibrosis	8
Experimental drugs for the inhibition of preterm labor	8
Adrecizumab: an investigational agent for the biomarker-guided treatment of sepsis	8
Investigational immunotherapy targeting CD19 for the treatment of acute lymphoblastic leukemia	8
Development of gamma-tocotrienol as a radiation medical countermeasure for the acute radiation syndrome: current status and future perspectives	8
Omaveloxolone: an activator of Nrf2 for the treatment of Friedreich ataxia	7
COR388 (atuzaginstat): an investigational gingipain inhibitor for the treatment of Alzheimer disease	7
Cardiac fibrosis: emerging agents in preclinical and clinical development	7
Experimental HER2- targeted therapies for biliary tract cancer	7
Treating cognitive impairment in schizophrenia with GLP-1RAs: an overview of their therapeutic potential	7
Tilsotolimod: an investigational synthetic toll-like receptor 9 (TLR9) agonist for the treatment of refractory solid tumors and melanoma	7
The impact of molecular profiling on cholangiocarcinoma clinical trials and experimental drugs	7
Enhancing venetoclax activity in hematological malignancies	7
Novel agents for mantle cell lymphoma: molecular rational and clinical data	7
New investigational agents for the treatment of major depressive disorder	7
Ritlecitinib: an investigational drug for the treatment of moderate to severe alopecia areata	7
Druggable molecular alterations in bile duct cancer: potential and current therapeutic applications in clinical trials	7
Advances in the treatment of platinum resistant epithelial ovarian cancer: an update on standard and experimental therapies	7
IgA nephropathy: an overview of drug treatments in clinical trials	7
Novel immunotherapy combinations in clinical trials for hepatocellular carcinoma: will they shape the future treatment landscape?	7
Current options and investigational drugs for the treatment of eosinophilic esophagitis	7
Single and multiple dose pharmacokinetics and safety of ZSP1273, an RNA polymerase PB2 protein inhibitor of the influenza A virus: a phase 1 double-blind study in healthy subjects	7
Ruxolitinib: a potential treatment for corticosteroid refractory acute graft-versus-host disease	7
A perspective on RNA interference-based therapeutics for metabolic liver diseases	6
Advances in targeting estrogen synthesis and receptors in patients with endometriosis	6
Investigational PARP inhibitors for the treatment of biliary tract cancer: spotlight on preclinical and clinical studies	6
Trabodenoson on trabecular meshwork rejuvenation: a comprehensive review of clinical data	6
Emerging neuroprotective interventions in periventricular leukomalacia - A systematic review of preclinical studies	6
KSI-301: an investigational anti-VEGF biopolymer conjugate for retinal diseases	6
Recent progress in development of cyclin-dependent kinase 7 inhibitors for cancer therapy	6
Investigational drugs in early phase development for primary biliary cholangitis	6
Does lopinavir measure up in the treatment of COVID-19?	6
Efficacy, safety, and tolerability of ulotaront (SEP-363856, a trace amine-associated receptor 1 agonist) for the treatment of schizophrenia and other mental disorders: a systematic review of preclini	6
COVID-related fibrosis: insights into potential drug targets	6
A phase 1b/2 study of PF-06747775 as monotherapy or in combination with Palbociclib in patients with epidermal growth factor receptor mutant advanced non-small cell lung cancer	6
A randomized, double-blind, parallel pharmacokinetic study comparing the trastuzumab biosimilar candidate, AryoTrust®, and reference trastuzumab in healthy subjects	6
Systemic lupus erythematosus: an expert insight into emerging therapy agents in preclinical and early clinical development	6
Prexasertib: an investigational checkpoint kinase inhibitor for the treatment of high-grade serous ovarian cancer	6
Ewing sarcoma: investigational mono- and combination therapies in clinical trials	6
Emerging HER2-directed therapeutic agents for gastric cancer in early phase clinical trials	6
Camrelizumab: an investigational agent for hepatocellular carcinoma	6
Therapeutic strategies for Parkinson’s disease: promising agents in early clinical development	6
Is retatrutide (LY3437943), a GLP-1, GIP, and glucagon receptor agonist a step forward in the treatment of diabetes and obesity?	6
Nivolumab: an investigational agent for the treatment of biliary tract cancer	6
An expert overview of emerging therapies for acute myeloid leukemia: novel small molecules targeting apoptosis, p53, transcriptional regulation and metabolism	6
Innovative therapies for invasive fungal infections in preclinical and clinical development	6
Dementia with Lewy bodies: emerging drug targets and therapeutics	6
Nonalcoholic steatohepatitis (NASH) cirrhosis: a snapshot of therapeutic agents in clinical development and the optimal design for clinical trials	5
New drugs on the horizon for cerebral edema: what’s in the clinical development pipeline?	5
Investigational drugs for the treatment of kidney transplant rejection	5
Combination therapies plus transarterial chemoembolization in hepatocellular carcinoma: a snapshot of clinical trial progress	5
Investigational treatments for chronic lymphocytic leukemia: a focus on phase 1 and 2 clinical trials	5
Investigational topical anticholinergics in clinical development for the treatment of hyperhidrosis	5
Targeted therapies for extrahepatic cholangiocarcinoma: preclinical and clinical development and prospects for the clinic	5
ALZT-OP1: an experimental combination regimen for the treatment of Alzheimer’s disease	5
Ivosidenib: an investigational drug for the treatment of biliary tract cancers	5
Novel approaches for the management of biliary tract cancer: today and tomorrow	5
Chemoresistance and resistance to targeted therapies in biliary tract cancer: what have we learned?	5
Repurposing tetracyclines for acute respiratory distress syndrome (ARDS) and severe COVID-19: a critical discussion of recent publications	5
SGLT2 inhibitors: an evidence-based update on cardiovascular implications	5
Bioavailability and pharmacokinetic profile of balovaptan, a selective, brain-penetrant vasopressin 1a receptor antagonist, in healthy volunteers	5
Novel investigational treatments for ventilator-associated pneumonia and critically ill patients in the intensive care unit	5
Results from a biomarker study to accompany a phase II trial of RRx-001 with reintroduced platinum-based chemotherapy in relapsed small cell carcinoma	5
Investigational drugs for the treatment of thymic cancer: a focus on phase 1 and 2 clinical trials	5
Galinpepimut-S (GPS): an investigational agent for the treatment of acute myeloid leukemia	5
Cholangiocarcinoma: shedding light on the most promising drugs in clinical development	5
Hepatitis D virus (HDV): investigational therapeutic agents in clinical trials	5
Tucatinib: an investigational novel therapeutic agent for the treatment of HER-2 colorectal cancer	5
Key challenges for drugs in clinical development for cholangiocarcinoma	5