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|
|Cyxone||Phase I||T20K||Multiple Sclerosis (MS)||This is a double-blinded trial being conducted at a clinical centre in the Netherlands, where T20K will be administrated by infusion to healthy male volunteers. The study will assess the level of T20K in the blood after administering one or two doses of the drug candidate. The primary study objective is to evaluate the safety and tolerability of T20K in humans and is being conducted in collaboration with QPS Netherlands, a CRO located in Groningen, Netherlands.||T20K is being developed as a first line treatment for MS. T20K is naturally derived from plant protein and has demonstrated in a preclinical animal model to reduce MS symptoms after oral administration by inhibiting pro-inflammatory cytokines such as IL-2. To date no signs of toxicity have been observed at therapeutic dosages. Results from preclinical studies also show that T20K could be used to mitigate or even prevent MS events and with its prophylactic properties, to potentially even delay MS disease progression.|
|NantKwest Inc.||Phase I||PD-L1 t-haNK||Solid Tumours||This is a Phase I study designed to assess the safety, tolerability and efficacy of PD-L1 t-haNK cell therapy in patients with solid tumours. Upon completion of safety testing in this Phase I study, the sponsor plans to combine this PD-L1 t-haNK cell therapy with other immunomodulatory agents including NabFc-N803, a IL15 cytokine super agonist and Adenovirus or yeast vectors.||PD-L1 t-haNK cell therapy is a novel, NK cell-based immuno-oncology therapy that incorporates a PD-L1 based Chimeric Antigen Receptor (CAR) engineered into the sponsor's proprietary haNK NK cell, which also includes the high affinity variant of the CD16 receptor (V158 FcγRIIIa) to mediate antibody dependent cellular cytotoxicity. In preclinical studies, the use of this next generation PD-L1 t-haNK therapy, in a tandem-specific manner has demonstrated to significantly enhance cancer cell annihilation and improve overall response rates.|
|Pepscan||Phase I||Clinical Neoantigen Peptides||Cancer||On 25th June, last month, the sponsor announced that they have successfully in collaboration with undisclosed partners initiated patient recruitment for their Phase I study with patients receiving their first doses of the neoantigen vaccines. Early results are anticipated in the fourth quarter of 2019. The sponsor is solely responsible for production of clinical neoantigen peptides, the core active ingredient in the vaccine candidate tailored to tumours of individual patients.||Neoantigens, or tumour-specific antigens, are peptide sequences uniquely expressed in proteins from the tumour cells of individual patients. The sponsor's in vitro synthesised neoantigen peptides represent the core active ingredient of these anti-cancer vaccines. The design of the vaccines is intended to stimulate the patient's immune system to attack the tumour, thus inhibiting or reducing the proliferation of tumour cells. Neoantigen vaccination is a new generation personalized cancer treatment undergoing a huge growth phase in clinical development.|
|I-Mab Biopharma||Phase I||TJC4||Advanced Malignant Tumours||This Phase I study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of TJC4 in patients with advanced solid tumours and lymphoma when administered as a single agent and in combination with other cancer treatment agent(s).||CD47 is a glycoprotein over-expressed in a wide variety of tumours and sends a "don't engulf me" signal to tumour-engulfing macrophages via its ligand known as SIRPα. Inhibition of CD47 by TJC4 enables macrophages to engulf malignant tumour cells as a potential treatment strategy for cancers.|
|Asieris Pharmaceuticals||Phase Ib||APL-1202 in combination with BCG (Bacillus Calmette Guerin)||Non-Muscle Invasive Bladder Cancer (NMIBC)||The primary objective of this Phase Ib US study is to assess the safety, tolerability, and pharmacokinetic profile of APL-1202 when administered in combination with intravesical BCG. Recruited patients are NMIBC subjects who have undergone at least one induction course of intravesical BCG.||APL-1202 is the first oral and reversible methionine aminopeptidase II type (MetAP2) inhibitor of its class currently under clinical development worldwide. Its mechanism of action is novel in the blockade of both tumour cell growth and angiogenesis. APL-1202 is currently registered in China also for clinical development.|
|Precigen, Inc.||Phase I/Ib||PRGN-3006 UltraCAR-T||Relapsed or Refractory Acute Myeloid Leukaemia (AML) or Higher Risk Myelodysplastic Syndrome (MDS)||This Phase I/Ib clinical trial is of a single centre, nonrandomized, investigator‐initiated study design. The safety and tolerability of PRGN‐3006 UltraCAR-T will be evaluated following intravenous administration of escalating doses in subjects with relapsed or refractory AML or higher risk MDS.||PRGN-3006 utilizes the sponsor's proprietary UltraCAR-T therapeutic platform, which eliminates ex vivo expansion and reduces manufacturing time to less than two days following non-viral gene transfer at the clinical site. PRGN-3006 UltraCAR-T is a multigenic CAR-T cell treatment which applies the sponsor's advanced non-viral gene delivery system to co-express a chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch mechanism for more precise and controlled targeting in relapsed or refractory AML and higher risk MDS. The clinical trial is being perfromed in collaboration with the Moffitt Cancer Center.|
|Syntrix Pharmaceuticals||Phase I/II||SX-682 in combination with KEYTRUDA® (Pembrolizumab)||Metastatic Melanoma||This Phase I/II clinical trial is an open-label study which will assess the safety, tolerability, immune response markers, and overall response rates achieved with SX-682 in combination with KEYTRUDA® in up to 77 subjects with metastatic melanoma. The study will assess biomarkers identified from paired biopsies taken before and after the three-weeks of monotherapy and combination treatments, as well as clinical outcomes observed over the course of the trial. The trial is being carried out at the Massachusetts General Hospital and Dana-Farber Cancer Institute.||From preclinical studies, SX-682 demonstrated that it enhances both PD-1 immune checkpoint inhibition and T cell receptor engineered T cell elicitation. Effects include a reduction of myelosuppressive cells in the tumour microenvironment and augmentation of NK and T cell infiltration into the tumour site. Clinical studies to date have revealed an inverse correlation between heamatological CXCR1/2 ligands and survival of subjects treated with anti-PD1 therapy.|
|Minovia Therapeutics||Phase I/II||Mitochondrial Augmentation Therapy (MAT)||Pearson Syndrome||This Phase I/II clinical trial is of an open label, single dose study design to assess the safety and therapeutic effects of transplantation of MNV-BM-BLD, the sponsor’s autologous CD34+ cells enriched with blood-derived mitochondria, in paediatric subjects. The study will recruit a total of seven patients by invitation and the primary outcome will be safety as determined by the number of participants with treatment-related adverse events at one year duration, as well as quality of life improvement as determined by an International Paediatric Mitochondrial Disease Scale (IPMDS), a multi-dimensional scale rating clinically relevant aspects of mitochondrial disease in children.||The secondary outcomes measured also at one year duration will include cognitive status, changes in brain MRI, changes in physical activity and muscle function, changes in body mass, and changes in peripheral blood lactate levels. As per study protocol, autologous CD34+ cells enriched with blood-derived mitochondria manufactured by the sponsor’s proprietary MAT platform will be transplanted via a single dose into paediatric subjects with Pearson syndrome to increase the levels of normal mitochondrial DNA. Pearson syndrome is a debilitating genetic disorder caused by a deletion in the mitochondrial DNA and affects approximately 100 child cases globally. Such deletions cause cellular respiration dysfunction, leading to poor energy production in affected patients. Pearson syndrome affects many parts of the body but in particular bone marrow cells and the pancreas.|
|Xeris Pharmaceuticals, Inc.||Phase II||Developmental Ready-to-Use Glucagon||Post-Bariatric Hypoglycaemia (PBH) following Bariatric Surgery||This Phase II clinical trial, is of a randomized, placebo-controlled, double-blind study design which will assess the efficacy, safety and tolerability of the sponsor's ready-to-use glucagon in treating symptomatic postprandial hypoglycaemia among 12 patients with PBH initially during two in-patient clinical research centre visits, and then ongoing as part of a 12-week outpatient phase. Efficacy will be determined during confirmed hypoglycaemic episodes by plasma glucose recovery (blood glucose >70 mg/dL) at 15 minutes after dosing with ready-to-use glucagon or placebo.||PBH is a complication of bariatric surgery considered to be related to excessive insulin secretion in response to a meal. The onset of PBH can begin one to eight years after gastric bypass surgery. Glucagon is the standard of care for treating severe hypoglycaemia. The sponsor is applying its novel technology platforms to develop and commercialize ready-to-use, room-temperature stable injectable and infusible drug formulations including its ready-to-use glucagon candidate. The sponsor’s proprietary XeriSol™ and XeriJect™ formulation technologies are being evaluated for the subcutaneous (SC) and intramuscular (IM) administration of highly-concentrated, non-aqueous, ready-to-use formulations.|
|iLiAD Biotechnologies||Phase IIb||BPZE1||Pertussis (Whooping Cough)||This Phase IIb clinical trial is of a multicentre, randomized, placebo-controlled, and observer-blinded study design which will evaluate the immunological response and safety profile of single dose and two dose vaccination adminstration schedules of the BPZE1 vaccination, a live attenuated intranasal pertussis vaccine in healthy adults. Roughly 300 subjects will be randomly assigned 2:1 for the first (primary) vaccination with 200 subjects assigned to BPZE1 vaccination and 100 subjects to Boostrix™ (currently marketed vaccine). Half of the subjects in each primary vaccination group will receive a second (booster) of the BPZE1 vaccination and the other half will receive placebo. The primary immuno-efficacy outcome is the proportion of subjects who achieve seroconversion against at least 1 pertussis antigen in nasal secretions on Day 29 or 113 (primary or primary + booster). The primary safety outcomes are solicited adverse events for 7 days after each vaccination and safety laboratory results.||Key secondary clinical endpoints are systemic immunogenicity (IgG, IgA, and IgG or IgA) including the proportion of subjects that are seropositive to 2 or more pertussis antigens in serum, mucosal immunity including the proportion of subjects generating a seropositive response to 2 or more pertussis antigens in nasal secretions, and the proportion of subjects with BPZE1 nasopharyngeal colonization after the booster vaccination. BPZE1 is designed to vanquish deficiencies of current vaccines, including poor durability and failure to prevent nasopharyngeal Bordetella pertussis infections giving rise to escape mutants and thus transmission to vulnerable infants. Pertussis is a life-threatening disease caused by the highly contagious respiratory bacterium Bordetella pertussis. Despite an estimated global vaccination coverage rate of 84%, current vaccines are unable to control epidemics. Moreover, current vaccines do not fully protect infants under the age of 6 months, since multiple immunization injections are needed at 2, 4 and 6 months.|
|X4 Pharmaceuticals, Inc.||Phase III||Mavorixafor (X4P-001)||Warts, Hypogammaglobulinemia, Infections and Myelokathexis (WHIM)||This worldwide Phase III 4WHIM clinical trial is of a 52-week, randomized, double-blind, placebo-controlled, multicentre study design which will assess the safety and efficacy of Mavorixafor in genetically confirmed WHIM patients. The study is intending to recruit up to 28 patients in approximately 20 countries, followed by an open-label extension study. The primary efficacy endpoint for the study will compare the level of circulating neutrophils relative to a clinically meaningful threshold (500 cells/µL), in response to Mavorixafor versus placebo measured during multiple 24-hour periods over the duration of 52 weeks.||The secondary endpoints of the study include infection rates, wart burden, assessments of immune system function and quality of life. Mavorixafor is a potentially new generation, once-daily, oral, small molecule antagonist of the chemokine receptor, CXCR4. Proof of concept in WHIM patients has been shown with significant increases in neutrophil and lymphocyte counts, both critical biomarkers of CXCR4 signaling and immune function. Mavorixafor has been well tolerated in earlier phase clinical trials.|
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