Clinical Catch-Up: December 2-5


It was, as usual, a pretty busy week in clinical trial news. Here’s a look.

ASLAN Pharmaceuticals announced positive preliminary data from the lowest dose cohort of its trial of ASLAN004 for moderate-to-severe atopic dermatitis. ASLAN004 is a first-in-class fully human monoclonal antibody that binds to the IL-13 receptor of the alpha1 subunit, which blocks signaling of IL-4 and IL-13. In a review of early data, three patients’ symptoms were reduced by 85%, 70% and 59%.

Rafael Pharmaceuticals expanded its Phase II trial of CPI-613 (devimistat) for relapsed or refractory Burkitt’s lymphoma/leukemia. The trial will start enrolling patients at Massachusetts General Hospital in Boston. It is currently underway at Memorial Sloan Kettering Cancer Center in New York City and at City of Hope in Duarte, California. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, which is essential to tumor cell multiplication and survival.

Pellepharm, a BridgeBio company, completed enrollment in its Phase III trial of Patidegib Topical Gel 2% compared to vehicle gel in patients with Gorlin Syndrome. Gorlin Syndrome is a rare, genetic disease marked by heritable mutations in one allele of the tumor suppressor gene encoding PATCHED1, which inhibits the hedgehog signaling pathway. This leads to multiple basal cell carcinomas, often on the face.

Alkahest presented new data from its Phase IIa trial of ALK6019-201 in mild-to-moderate Alzheimer’s Disease. The trial is completed and was designed to determine the safety, tolerability, and possible therapeutic effects of GRF6019 in this patient group over a six-month period. GRF6019 is a proprietary plasma fraction, which in animal models, enhance neurogenesis and improve age-related deficits in learning and memory, as well as decrease neuroinflammation.

Genentech published data from SAkuraSky, a pivotal Phase III trial of satralizumab for neuromyelitis optica spectrum disorder (NMOSD). Satralizumab inhibits interleukin-6 signaling pathway, which plays a role in the inflammation seen in NMOSD. The published data showed that only eight of 41 patients (20%) receiving satralizumab with baseline immunosuppressant therapy experienced a protocol-defined relapse (PDR) compared to 18 of 42 patients (43%) treated with placebo and baseline therapy.

OSE Immunotherapeutics completed and announced positive data from its Phase I trial of OSE-127. OSE-127 is a humanized monoclonal antibody against the CD127 receptor, the alpha chain of the interleukin-7 receptor. The trial showed a good safety and tolerability profile. The company expects to use the dosing information to run two planned Phase II trials in ulcerative colitis and Sjogren’s syndrome.

Tetra Therapeutics presented data from a study of electronic health records of 56 million patients, evaluating whether the systemic inflammatory diseases that are mediated by tumor necrosis factor (TNF) increased the risk of Alzheimer’s disease. It also asked whether that risk could be mitigated with an FDA-approved, TNF-blocking drug. The findings suggested that yes, the inflammatory disease risk increased and that yes, treatment with these drugs did seem to reduce the risk of Alzheimer’s disease.

Censa Pharmaceuticals announced positive data from its Phase II trial of CNSA-001 (sepiapterin) for phenylketonuria (PKU). The drug met primary and secondary endpoints, showing a statistically significant and clinically meaning decrease in blood phenylalanine levels.

CytoDyn announced additional early Phase Ib/II clinical trial data of leronlimab (PRO 140) patients with CCR5+ metastatic triple-negative breast cancer. The data from the first patient showed no detectable circulating tumor cells or EMTs in the peripheral blood. There were also additional reductions in CCR5 expression on cancer-associated cells at eight weeks. Leronlimab is a humanized IgG4 mAb that blocks CCR5.

Outlook Therapeutics received agreement on three Special Protocol Assessments from the FDA for three additional registration trials for its ongoing Phase III program for ONS-5010. ONS-5010 is an investigational ophthalmic formulation of bevacizumab. The drug is under development for intravitreal injection for wet AMD and other retinal diseases.

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Beta Bionics announced that its bionic pancreas glucose control algorithms were deployed in a pilot study in three patients with cystic fibrosis-related diabetes. Patients using the bionic pancreas experienced improved glycemic control and decreased diabetes management burden.

MediWound initiated patient treatment in its U.S. Phase II adaptive design clinical trial of EscharEx. EscharEx is a topical biologic drug to enzymatically debride chronic wounds. It is being developed for the treatment of venous leg ulcers (VLUs). The trial will enroll 174 patients at about 25 clinical sites, mostly in the U.S.

GenKyoTex announced further progress in two Phase II trials of setanaxib in diabetic kidney disease (DKD) and idiopathic pulmonary fibrosis (IPF). In the DKD trial, they expanded the investigational network by adding centers in Germany, Denmark and New Zealand. The FDA and other Institutional Review Boards have approved the protocol of the Phase II IPF trial. Setanaxib is a NOX1 and NOX4 inhibitor that’s shown anti-fibrotic activity in primary biliary cholangitis.

Cara Therapeutics announced positive topline results from its Phase II dose-ranging study of oral Korsuva for the treatment of pruritus (itching) in stage III-V chronic kidney disease. Patients receiving the 1 mg tablet hit the primary endpoint of statistically significant reduction in weekly mean of the daily WI-NRS scores compared to placebo after 12 weeks of treatments.

Zogenix published the results from its Phase III trial of Feintepla (fenfluramine) in Dravet syndrome patients whose antiepileptic drug treatment included stiripentol but who were still experiencing a high number of convulsive seizures. The study met its primary efficacy endpoint and all key secondary endpoints. Patients receiving the drug had a 54% greater reduction in mean MCSF than those receiving the placebo.

Ampio Pharmaceuticals updated progress on its Phase III AP-013 clinical trial. As of December 3, 1,034 patients had been enrolled, more than 700 were injected and another 50 have been approved for injection. The trial is evaluating Ampion for adults with severe osteoarthritis knee pain. Ampion is a low molecular weight fraction of human serum albumin (HSA).

Innovative Cellular Therapeutics announced the FDA had cleared its IND application for ICTCAR014. The drug is a next-generation CD19-targeting CAR-T therapy. The trial is expected to launch in patients with relapsed or refractory non-Hodgkin lymphoma (NHL), including patients whose tumors are PD-L1 positive, in the first part of 2020.

Biogen announced positive topline data from the Phase II LILAC trial evaluating the efficacy and safety of BIIB059. BIIB059 is a fully humanized IgG1 monoclonal antibody that targets blood dendritic cell antigen 2 expressed on plasmacytoid dendritic cells in lupus patients. The cutaneous lupus erythematosus (CLE) part of the trial met the primary endpoint of percent change from baseline in the Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity (CLASI-A) score at Week 16. It also met the primary endpoint of reducing disease activity in SLE patients measured by change from baseline in total active joint count at Week 24.

Aurinia announced the results of its pivotal AURORA Phase III trial of voclosporin with mycophenolate and low-dose corticosteroids in lupus nephritis, which is an autoimmune disease related to lupus. In the AURORA study, 357 patients with active LN were enrolled. The trial’s primary endpoint of Renal Response rates was 40.8% for voclosporin compared to 22.5% for the control group. The secondary endpoints included Renal Response at 24 weeks, Partial Renal Response at 24 and 52 weeks, time to achieve urinary protein-to-creatinine ration (UPCR) and time to 40% reduction in UPCR. The drug was well tolerated with no unusual or unexpected safety signals. Voclosporin is a potentially best-in-class calcineurin inhibitor. By inhibiting calcineurin, it blocks IL-2 expression and T-cell mediated immune responses, which stabilizes the podocyte in the kidney.

ACADIA Pharmaceuticals presented positive topline data from its Phase III HARMONY study. Pimavanserin met the primary endpoint of the study for treatment of dementia-related psychosis. Pimavanserin is a selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors.

Allergan published positive results from ACHIEVE I, a Phase III trial of ubrogepant in acute migraine. The study evaluated two doses of the drug compared to placebo. The trial hit both co-primary endpoints of pain freedom and freedom from the most bothersome of three non-headache migraine-associated endpoints, sensitivity to light, sound and nausea at two hours post initial dose.

SAGE Therapeutics’ Phase III MOUNTAIN trial of SAGE-217 on depressive symptoms in adults with major depressive disorder (MDD) failed to meet its primary endpoint of statistically significant reduction in the 17-item Hamilton Rating Scale for Depression (HAM-D) at Day 15. The analysis also found that about 9% of the patients were non-compliant in taking the drug. SAGE-217 is an oral neuroactive steroid (NAS) GABAA receptor positive allosteric modulator (PAM).

Rhythm Pharmaceuticals completed enrollment of its pivotal cohort of the Phase III trial of setmelanotide for insatiable hunger and severe obesity in people with Bardet-Biedl syndrome (BBS) or Alstrom syndrome. Rhythm enrolled 32 people with BBS and six people with Alstrom in the cohort. Both are ultra-rare disorders that affect multiple organs.

Galera Therapeutics published full results from its Phase IIb trial of avasopasem manganese in patients with locally advanced head and neck cancer. The drug is a highly selective small molecule dismutase mimetic. It is designed to rapidly convert superoxide to hydrogen peroxide, which decreases mucosal damage and as a result, the incidence and severity of mucositis. The study showed that adding a 90 mg dose of the drug to a standard radiotherapy regimen significantly reduced severe oral mucositits duration from 19 days to 1.5 days.

FerGene announced positive results from the Phase III trial of nadofaragene firadenovec for high-grade, Bacillus Calmette-Guerin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC). Nadofaragene firadenovec is a gene therapy that delivers the gene interferon alfa-2b into the bladder. The trial met the primary endpoint with more than half of patients achieving a complete response at three months.

Mitsubishi Tanabe Pharma America presented new analysis of real-world clinical outcomes of treatment with Radicava (edaravone) in ALS. The data was from 163 ALS patients receiving Radicava in the U.S., evaluating the average change in physical function as measured by ALS Functional Rating Scale-Revised (ALSFRS-R) score.

Cancer Treatment Centers Of America (CTCA) in Phoenix dosed the first patient in the first-in-human study of NKTR-255. NKTR-255 is Nektar Therapeutics’ first-in-class immunotherapy. The Phase I trial will study the drug as a monotherapy in relapsed or refractory non-Hodgkin lymphoma (NHL) or multiple myeloma.

Novartis announced data from its Phase III PALLADIUM trial of QMF149 for asthma. The study was evaluating a once-daily fixed-dose combination of inadacaterol acetate and mometasone furoate (IND/MF) compared to mometasone furoate (MF) at medium and high doses in improving lung function. The trial met the primary endpoint, showing statistically significant superiority compared to MF alone, as well as a secondary endpoint of improvement in asthma control.

Ipsen placed a partial clinical hold for its two trials of palovarotene for the chronic treatment of fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO). The partial clinical hold applies to the pediatric population in the Phase II and Phase III trials at all sites following recent safety reports about cases of early growth plate closure in pediatric patients with FOP treated with the drug.

UCB announced positive results from its Phase III BE SURE study of bimekizumab in adults with moderate-to-severe plaque psoriasis. The trial met its co-primary endpoints at week 16, showing superiority of bimekizumab to adalimumab in hitting at least a 90% improvement in the Psoriasis Area and Severity Index (PASI 90) and Investigator Global Assessment (IGA) response of clear or almost clear. Bimekizumab is an IL-17A and IL-17F inhibitor.

Biohaven Pharma announced it had completed a pre-planned interim futility analysis of the T2 Protect AD Study. This is an ongoing Phase II/III trial of truriluzole in Alzheimer’s disease. Futility was not met based on pre-specified interim analysis criteria. As a result, the company said that based on it, the trial would continue. Truriluzole is an oral, once-daily glutamate modulator.

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