Like many aspects of healthcare business space, drug development is in a state of constant change for the purpose of better health outcomes. Here is a summarised report of new drug therapies currently in clinical trials worldwide as from this week.
|Sponsor||Study Phase||Drug Name/Code||Clinical Indication||Status||Comment|
|Tizona Therapeutics, Inc.||Phase I/Ib||TTX-030||Cancer||This Phase I/Ib clinical trial will investigate the safety, tolerability, pharmacokinetics and anti-tumor activity of TTX-030 as a single therapy, in combination with an approved anti-PD-1 immunotherapy, and in combination with standard chemotherapies in adults with advanced cancers.||TTX-030 is classed as a monoclonal antibody. Its mechanism of action is inhibition of CD39 activity. CD39 is a cell surface enzyme that converts ATP to AMP, the first step in the generation of adenosine in the tumour microenvironment (TME). CD39 is then upregulated on tumours as an immune evasion strategy, and on exhausted T cells and other suppressive cell types. By stopping the activity of CD39, TTX-030 prevents the formation of immune suppressive extracellular adenosine, which otherwise inhibits effector cells in the TME.|
|Amylyx Pharmaceuticals, Inc.||Phase II||AMX0035||Alzheimer’s Disease (AD)||This is an expanded Phase II study (PEGASUS) to assess AMX0035 in patients with AD. This PEGASUS clinical trial is of a 3:2 randomized, double-blind, multi-centre, placebo-controlled study design investigating the safety, tolerability and activity of AMX0035 in patients with late mild cognitive impairment or early dementia due to AD over 24 weeks. The study is of a biomarker-based clinical end point seeking to demonstrate the effects of AMX0035 and its potential in treating AD.||AMX0035 is Amylyx’s proprietary two-drug combination therapy in development to prevent nerve cell death and degeneration. PEGASUS is the first clinical trial of its kind to target both amyloid and tau mechanisms in tandem.|
|Odonate Therapeutics, Inc.||Phase II||Tesetaxel (in combination with Capecitabine)||Locally Advanced or Metastatic Breast Cancer (LA/MBC)||This Phase II study (CONTESSA 2) will assess the combination of Tesetaxel plus a reduced dose of Capecitabine in approximately in 125 patients with locally advanced or metastatic breast cancer (LA/MBC). CONTESSA 2 is of a multinational, multicentre study design investigating orally administered Taxane, in patients with LA/MBC. The study trial will assess tesetaxel dosed orally at 27 mg per metre squared on the first day of each 21-day cycle plus a reduced dose of Capecitabine (1,650 mg/metre-squared/day dosed orally for 14 days of each 21-day cycle) in roughly 125 patients with human epidermal growth factor receptor 2 (HER2) negative, hormone receptor (HR) positive LA/MBC not previously treated with a taxane. The primary endpoint of this study trial will be objective response rate (ORR) as assessed by an Independent Radiologic Review Committee (IRC).||Tesetaxel is an investigational, orally administered chemotherapy candidate belonging to the taxanes drug class, a commonly used drug class for cancer treatment. Tesetaxel bears several pharmacologic properties making it unique among taxanes, including: oral administration with a low pill dosage burden; an extended ( approx. 8 days) terminal plasma half-life in humans, enabling the maintenance of adequate drug levels with relatively fewer doses; no history of hypersensitivity (allergic) reactions; and significant activity against chemotherapy-resistant tumors. In MBC patients, Tesetaxel was shown to have significant, single-agent antitumor activity in two multicentre, Phase II studies. As a secondary efficacy endpoints, this study will use duration of response (DoR) as assessed by the IRC, progression-free survival (PFS) as assessed by the IRC, disease control rate (DCR) as assessed by the IRC and overall survival (OS).|
|Poxel SA||Phase IIa||PXL770||Non-Alcoholic Steatohepatitis (NASH)||This Phase IIa 12 week, multicentre, randomized, double-blind, placebo-controlled, parallel group study will investigate the efficacy and safety of PXL770, a direct adenosine monophosphate-activated protein kinase activator (AMPK), for the treatment of NASH. In this study, three doses of PXL770 versus placebo will be administered to roughly 100 nonalcoholic fatty liver disease (NAFLD) patients who are susceptible to NASH across several clinical sites in the US. The primary endpoint of tthis clinical trial will measure the change in liver fat mass based on magnetic resonance imaging-estimated proton density fat fraction (MRI-PDFF), a novel imaging-based biomarker that allows fat mapping of the entire liver. In addition to the Phase IIa study, a separate four-week PK/PD study is expected to be initiated whereby PXL770 will be administered to approximately 32 patients. This study will assess the PK profile of PXL770 in NAFLD patients and its effects on hepatic and metabolic parameters in the target population.||PXL770 is a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator. AMPK is a central regulator of multiple metabolic pathways leading to the control of lipid metabolism, glucose homeostasis and inflammation. NASH is a metabolic disease with no clear disease origin. It is characterized by the accumulation of fat in the liver causing inflammation and fibrosis. Typical risk factors for NASH include obesity, elevated levels of blood lipids e.g. cholesterol and triglycerides and diabetes.|
|Biohaven Pharmaceutical Holding Company, Ltd.||Phase II/III||BHV-3500||Migraine||This Phase II/III, double-blind, randomized, placebo-controlled, dose-ranging study of intranasally administered BHV-3500 will be used to investigate acute treatment of migraine. In this study, 3 doses of BHV-3500 (5, 10 and 20 mg) will be compared to placebo in the treatment of a migraine onset. The planned recruitment of approximately 400 randomized patients per treatment arm, will statistically power the study to provide proof of efficacy on the regulatory endpoints of pain freedom and freedom from the most bothersome migraine-associated symptom at 2 hours post-dose.||BHV-3500 is a novel, structurally distinct, third-generation calcitonin gene-related peptide (CGRP) receptor antagonist. The study is also designed to detect early onset of other clinical measures that are of great significance to patients, including relief of pain and ability to return to normal functioning.|
|Hutchison China MediTech Limited||Phase IIb/III||Surufatinib||Advanced Biliary Tract Cancer (BTC)||The study is a randomized, open-label, active-control, multi-centre design assessing the effects of Surufatinib (HMPL-012 or Sulfatinib) versus the chemotherapy agent Capecitabine, as a second-line therapy in patients with unresectable or metastatic BTC. The primary endpoint is overall survival (OS).||Surufatinib is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptor (VEGFR) and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumour-associated macrophages, promoting the body’s immune response against tumour cells. Secondary outcomes of this clinical trial include measures of tumour control such as progression free survival (“PFS”), objective response rate (ORR), disease control rate (DCR), and duration of response (DOR), quality of life, tumour biomarkers, and safety.|
|PellePharm, Inc.||Phase III||Patidegib Topical Gel, 2%||Gorlin Syndrome||This randomized, double-blind, vehicle-controlled Phase III study will investigate the safety and efficacy of Patidegib Topical Gel 2% applied topically, twice daily to the face over 12 months vs. vehicle gel. Roughly 150 adults with Gorlin Syndrome will be randomized 1:1 to Patidegib topical gel or vehicle. The primary efficacy endpoint of this study will be the number of new surgically eligible BCCs (nSEBs) that develop over the 12-month period in the two treatment arms.||Patidegib Topical Gel 2%, is an investigational treatment gel designed to reduce the BCC tumour burden in patients with Gorlin Syndrome and High Frequency BCC (HF-BCC) by blocking the disease at its source within the hedgehog signaling pathway. Secondary endpoints of this study will include the total number of new BCCs over 12 months, percentage of study participants developing ≥2 facial nSEBs over 12 months; the percentage of participants developing ≥1 facial nSEBs over 12 months; the number of nSEBs per participant over six and nine months; and change in the Advanced Basal Cell Carcinoma Index (aBCCdex) lesion scale score. An open-label safety and tolerability extension study is planned for at least 12 months for subjects who have completed the Phase III study.|
|Aldeyra Therapeutics, Inc.||Phase III||Topical Ocular Reproxalap||Dry Eye Disease||This Phase III clinical trial (RENEW Trial) is a two-part, multi-centre, randomized, double-masked, parallel-group, vehicle-controlled, adaptive study evaluating the efficacy of reproxalap ophthalmic solution (0.25%) versus vehicle in 400 patients with moderate-to-severe dry eye disease. Initial data from the first part of the study will confirm the dosing regimen and sample size for the second part. The co-primary endpoints of the trial will be ocular dryness and fluorescein nasal region ocular staining in pre-specified moderate to severe patient subsets analyzed over twelve weeks of therapy using Mixed effects Model Repeated Measures (MMRM).||Last year in September 2018, the sponsor announced results from the Phase IIb dry eye disease study, which demonstrated statistical superiority of Reproxalap versus vehicle across multiple symptoms and signs. In the Phase 2b clinical trial, the MMRM p values for the Phase III co-primary endpoints of dryness and staining were 0.0048 and 0.0007, respectively.|
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