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|
|MyoKardia Inc.||Phase I||MYK-224||Hypertrophic Cardiomyopathy (HCM)||This Phase I clinical trial is of a randomized, placebo-controlled, single and multiple-ascending dose study design that will enroll adult healthy volunteers into cohorts of eight, randomized 3:1 to MYK-224 or placebo. The primary study objectives will be to evaluate the safety, tolerability and pharmacokinetics of MYK-224.||As secondary study objectives, pharmacodynamic effects on cardiac function and dimensions will also be assessed using echocardiography. HCM is a progressive disorder in which the excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can lead to debilitating symptoms and cardiac dysfunction. MYK-224 selectively targets cardiac myosin, the sarcomeric proteins of the heart muscle, in other words the heart’s motor protein, with the aim of normalizing contractility and filling.|
|Kite Pharma||Phase I||KITE-439||Human Papillomavirus (HPV) associated cancers with HPV type 16||This Phase I, two part, open label clinical trial will evaluate the safety and efficacy of KITE-439. Adult, HLA-A201 positive patients with relapsed/refractory HPV16 solid tumors will receive cyclophosphamide and fludarabine conditioning chemotherapy followed by a single dose of KITE-439. The trial will be conducted in two parts, the first part of the study will evaluate the dose-limiting toxicity after a single dose of KITE-439. Based on the selected dose, part two of the study will evaluate the efficacy of KITE-439 by measuring the objective response rate.||HPV type 16-induced cancers, a strain that causes about 70% of all cervical cancers worldwide, are ideal candidates for targeted T cell therapy due to their nonself antigens. Those antigens can be readily recognized by immune cells inducing T cell activation. The study makes use of genetically engineered human T cells with KITE-439 receptors for the treatment of patients with relapsed or resistant HPV-associated cancers. This engineered receptor works on targeting antigens expressed in cancer cells that are infected by HPV, leading to a T cell activation against the cells.|
|NKMax America||Phase I||SNK||Plaque Psoriasis||This Phase I, single centre, dose escalation clinical trial will assess SNK administered intravenously once a week for four weeks at three different dosing levels. Nine patients will be recruited in the study to evaluate the safety and tolerability of SNK in adults with mild to severe plaque psoriasis. The clinical trial site will be at Angeles Hospital, in Tijuana, Mexico.||The sponsor's SNK product is an autologous therapy made by harvesting a small number of a patient's immune cells. After harvesting, NK cells are isolated from the blood, purified, activated, and expanded in a cGMP facility. The activated NK cells, known as "SNK," are reinfused in the patient to suppress the inflammatory response attributed to psoriasis.|
|Forendo Pharma||Phase Ib||FOR-6219||Endometriosis||In this on-going Phase Ib study, FOR-6219 will be administered to premenopausal women to evaluate its effect on the endometrium by measuring changes in endometrial thickness and endometrial estrogen levels. In addition to these local effects, systemic hormone levels will also be monitored. Secondary endpoints will be based on safety and tolerability.||The Phase Ia study results of FOR-6219 revealed single doses from 2mg up to 175mg and multiple doses up to 150mg twice daily over 10 days were found to be safe and well tolerated in 36 healthy postmenopausal women. The pharmacokinetics (PK) of FOR-6219 was dose-proportional and a steady state was attained within 3 days. The observed elimination half-life was in the range of 16 to 18 hours, inferring potential for once daily dosing. The PK profiles of FOR-6219 were similar in a fasted and fed state meaning that FOR-6219 can be administered with or without food. FOR-6219 is a first-in-class drug candidate that selectively targets endometriosis lesions without effecting systemic estrogen. The mode of action of FOR-6219 lies in its inhibitory effect of the conversion of low potency estrone into highly potent estradiol in endometrial tissues. The key differentiating pharmacology of FOR-6219 compared to currently available treatments is its selective activity and the ability to act locally in the target tissues, without adversely affecting systemic hormone levels.|
|BioInvent International AB||Phase I/IIa||BI-1206 in combination with Pembrolizumab (Keytruda)||Solid Tumours||This Phase l/lla study will investigate the drug combination of BI-1206, one of the sponsor's proprietary anti-FcγRllB antibodies, and Pembrolizumab (Keytruda®), a leading anti-PD1 antibody, in patients with advanced solid tumours, who have been previously treated with anti-PD1 or anti-PD-L1 antibodies. This clinical trial is of a multicentre, dose-finding, consecutive-cohort, open-label study design.||The premise to initiating this clinical trial program is based on the sponsor's recent preclinical data demonstrating the ability of BI-1206 to address an important mechanism of resistance to PD1 inhibition, providing a way to enhance anti-tumour immune responses in patients with solid tumours. The sponsor's proprietary F.I.R.S.TTM technology platform simultaneously identifies both targets and the antibodies that bind to them, generating new drug candidates for investigation.|
|Rocket Pharmaceuticals, Inc.||Phase II||RP-L102||Fanconi Anaemia (FA)||This open-label Phase II study will enroll up to five paediatric patients in Europe. The clinical trial will enroll paediatric patients who have not developed severe bone marrow failure. Patients will receive a single intravenous infusion of RP-L102 that utilizes “Process B” which incorporates enhanced stem cell enrichment, transduction enhancers and commercial-grade vector and manufacturing without the use of any conditioning treatment. The study is designed to evaluate the benefit/risk profile of RP-L102. The primary endpoint of the study is the emergence of mitomycin-C (MMC) resistance in bone marrow colony forming (progenitor) cells. A surrogate endpoint is the diepoxybutane (DEB) chromosomal stability of peripheral blood T-lymphocytes. Sensitivity to DNA-damaging agents (MMC and DEB) is a phenotypic hallmark of FA. Additional outcomes include stability or increase in blood counts with no significant worsening in anaemia, neutropenia or thrombocytopenia and peripheral blood genetic correction, as demonstrated by vector copy number (VCN), including progressive increases in peripheral blood VCN over the months subsequent to infusion.||FA is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic haematopoietic stem cell transplantation (HSCT), when available, corrects the haematologic component of FA, but needs myeloablative conditioning, which is highly toxic for the patient. HSCT is frequently complicated by graft versus host disease and also increases the risk of solid tumours, particularly upper aerodigestive tract squamous cell carcinomas. Roughly 60% to 70% of patients with FA have a FANCA gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress.|
|Landos Biopharma||Phase II||BT-11||Mild to moderate Ulcerative Colitis (UC)||This global Phase II clinical trial is of a randomized, placebo-controlled, double-blind, parallel-group, multicentre study design to investigate the safety, efficacy and tolerability of BT-11 compared to placebo in patients with mild to moderately-active UC. The study will recruit 195 patients with mild to moderate UC in 11 countries with 60 sites throughout the U.S., Europe and Eastern Europe. Patients will be randomized to receive one of two doses of BT-11 (500 or 1,000 mg) or placebo for a 12-week induction phase followed by a maintenance phase. The study’s primary endpoint will assess the effect of treatment with once-daily doses of BT-11 tablets or placebo on clinical remission rate at week 12, as defined by total Mayo score ≤ 2 with all sub-scores ≤1.||UC is a chronic inflammatory disease of the large intestine caused by the body’s immune system sending white blood cells to the intestinal lining thus eliciting chronic inflammation and ulcerations giving rise to abdominal discomfort and frequent emptying of the lower intestines. BT-11 is a first-in-class, gut-restricted small molecule IND of oral formulation that targets the Lanthionine Synthetase C-Like 2 (LANCL2) pathway that regulates the immunological state of the gastrointestinal tract. By activating the LANCL2 pathway and modulating the interactions between immunological and metabolic signals in immune cells, BT-11 creates a favorable regulatory microenvironment in the gut tissue wall, decreasing the production of key inflammatory mediators and increasing anti-inflammatory markers in regulatory T cells (Treg) within the site of inflammation.|
|Inovio Pharmaceuticals, Inc.||Phase II||VGX-3100||Human Papillomavirus (HPV)-related anal High-grade Squamous Intraepithelial Lesions (anal HSIL)||This Phase II clinical trial is of an open-label, multi-centre study design to evaluate the safety and efficacy of VGX-3100 administered by the Sponsor's CELLECTRA® delivery system in adult men and women with anal HSIL caused by HPV-16 and/or HPV-18. In a randomized, double-blind, placebo-controlled Phase IIb proof-of-concept study in 167 adult women with histologically documented HPV-16/18 cervical HSIL, treatment with VGX-3100 resulted in a statistically significant greater regression of cervical HSIL and clearance of HPV-16/18 infection versus placebo.||Anal HSIL or dysplasia is an orphan disease and a common precursor to developing anal cancer with an estimated mortality rate for this year so far of more than 1,280 deaths in the United States alone. Anal HSIL is estimated to occur in nearly 20,000 new cases annually. VGX-3100 is a DNA-based immunotherapy stimulating a specific immune response to HPV-16 and HPV-18, which targets the infection and causes destruction of precancerous cells.|
|Knopp Biosciences LLC||Phase II||Dexpramipexole||Eosinophilic Asthma||This 12-week biomarker Phase II clinical trial will randomize an estimated 100 patients with eosinophilic asthma to evaluate the eosinophil response to three different doses of oral Dexpramipexole. The primary outcome measure of this randomized, double-blind, placebo-controlled study is the change in blood absolute eosinophil count from Baseline to Week 12.||The secondary outcome measures of this study include changes in pre-bronchodilator FEV1 and asthma control outcomes (ACQ-7 questionnaire) from baseline to week 12. Dexpramipexole although prescribed as an oral steroid-sparing agent in hypereosinophilic syndromes, its mechanism of action is yet unclear.|
|Emalex Biosciences, Inc.||Phase IIb||Ecopipam HCl||Tourette Syndrome (TS)||This Phase IIb clinical trial of double-blind study design of Ecopipam HCl oral dosing in paediatric patients (aged >6 to <18 years) with TS is being conducted in multiple clinical sites in the United States, Canada, and the European Union. An estimated 150 patients will be randomized to either Ecopipam HCl 2 mg/kg/day or an equivalent placebo for 12 weeks. Upon completion of the study patients will be eligible to enroll in a one-year open-label safety extension study where all patients will receive Ecopipam HCl 2 mg/kg/day.||TS is a neurological disorder characterized by motor or vocal tics that begins during childhood and is not associated with any medication etiology, other medical reasons, or a confirmed neurological abnormality. Ecopipam HCl is an investigational first-in-class drug being investigated in paediatric patients for the treatment of TS and for childhood onset fluency disorder (stuttering) in adults. Ecopipam selectively blocks the actions of the neurotransmitter dopamine at the D1 receptor.|
|Satsuma Pharmaceuticals, Inc.||Phase III||STS101||Acute Migraine||This Phase 3 EMERGE efficacy clinical trial of candidate STS101 is of a multi-centre, single-dose, randomized, double-blind, placebo-controlled, parallel group study design to be undertaken in approximately 1,140 migraine patients in the United States. The EMERGE participanting patients will be randomized (1:1:1) to receive one of three treatments: STS101 DHE 3.9 mg, STS101 DHE 5.2 mg or equivalent placebo and will be instructed to treat their next migraine attack of at least moderate pain severity with the allocated blinded study medication. The two co-primary endpoints of the EMERGE trial to be assessed at two hours after STS101 administration are freedom from pain and freedom from most bothersome symptom (from among photophobia, phonophobia or nausea).||The EMERGE trial design incorporates several secondary endpoints and prospective evaluations of the clinical performance of STS101 in a number of patient subgroups that could differentiate the clinical profile of STS101. STS101 is a drug-device combination of a proprietary dry-powder formulation of Dihydroergotamine Mesylate (DHE), which can be swiftly and conveniently self-administered with a proprietary pre-filled, single-use, nasal delivery device. DHE is an antimigraine agonist to the serotonin (5-HT) -1B, -1D and -1F receptors. It also interacts with other serotonin, adrenergic and dopamine receptors.|
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