Clinical Catch-up for August 5-9

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Last week was a particularly busy week for biopharma companies to release information about ongoing clinical trials. Here’s a look.

Pfizer announced that its Phase III RESET clinical trial evaluating rivipansel in sickle cell disease (SCD) failed to meet its primary and key secondary efficacy endpoints. The goal of the trial was to evaluate the efficacy and safety of the drug in patients six years of age and older with SCD who were hospitalized for a vaso-occlusive crisis (VOC) and required treatments with intravenous (IV) opioids. The primary endpoint was readiness-for-discharge and the key secondary efficacy endpoints were time-to-discharge, cumulative IV opioid consumption, and time to discontinuation of IV opioids.

Roche announced interim results from its Phase III IMvigor130 clinical trial, indicating it met its co-primary endpoint of progression-free survival (PFS). The trial evaluated the combination of Tecentriq (atezolizumab) and platinum-based chemotherapy in previously untreated locally advanced or metastatic urothelial carcinoma (mUC) compared to chemotherapy alone. In this interim analysis, the trial met the co-primary endpoint of PFS with encouraging overall survival (OS) results, but the OS data was not yet mature and will require follow-up at the next planned analysis.

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Eli Lilly and Company reported that its Emgality (galcanezumab-gnlm) met the primary and all key secondary endpoints in its Phase III CONQUER style for chronic and episodic migraine. The trial was in migraine patients who didn’t respond to two to four standard-of-care migraine preventive drugs.

The CONQUER Phase III trial was conducted in 12 countries. It enrolled 462 patients with chronic or episodic migraine with a documented history of treatment failures to two to four standard treatments. Treatment failure was defined as inadequate results after at least two months of treatment at the maximum tolerated dose or stopped taking the drug for safety/tolerability reasons.

Eureka Therapeutics launched its Phase I/II clinical trial of ET140202 in liver cancer. ET140202 utilizes the company’s proprietary ARTEMIS antibody T-cell receptor platform engineered with a proprietary human TCR-mimic antibody that targets an alpha fetoprotein (AFP)-peptide/HLA-A2 complex on HCC cancer cells.

Altavant Sciences initiated a Phase IIa ELEVATE trial of rodatristat ethyl in pulmonary arterial hypertension (PAH). The trial is designed to evaluate safety, target engagement and biomarker information. It is a double-blind, placebo-controlled, multicenter trial in 36 adults with PAH. Rodatristat ethyl is a tryptophan hydroxylase (TPH) inhibitor designed to decrease the body’s peripheral production of serotonin.

Poxel SA, based in Lyon, France, initiated a Phase IIa trial of PXL770 for non-alcoholic steatohepatitis (NASH). The four-week PK/PD trial is expected to enroll about 16 patients per dose, with the key objective to evaluate the full PK profile of the drug in nonalcoholic fatty liver disease (NAFLD) patients who probably have NASH. PXL770 is a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, a central regulator of multiple metabolic pathways that control lipid metabolism, glucose homeostasis and inflammation.

SELLAS Life Sciences Group completed enrollment in its Phase II trial of nelipepimut-S (NPS) in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) in women with ductal carcinoma in situ (DCIS) of the breast who are HLA-A2+ or A3+ positive, express HER2 at IHC 1+, 2+, or 3+ levels, and are pre- or post-menopausal. The primary endpoint is the difference in frequency of newly induced NPS-cytotoxic T lymphocytes in peripheral blood between the two arms.

Eloxx Pharmaceuticals initiated a Phase II clinical trial for ELX-02 in cystinosis in Canada. It is a single arm, open label study to evaluate the safety, tolerability, PK and PD of subcutaneous ELX-02 in patients with nephropathic cystinosis with at least one nonsense mutation in the cystinosin gene. Six patients will be enrolled and will be treated with the drug for four weeks with three escalating dose levels.

Precigen dosed the first patient in Phase I trial of PRGN-3005 UltraCAR-T in advanced, recurrent platinum resistant ovarian, Fallopian tube or primary peritoneal cancer. PRGN-3005 UltraCAR-T is an autologous CAR-T therapy manufactured using non-viral gene delivery. The trial is conducted in partnership with the University of Washington and Fred Hutchinson Cancer Research Center.

Alkahest announced positive top-line data from its Phase II trial of GRF6019 in mild-to-moderate Alzheimer’s disease dementia. The drug was safe and well-tolerated in patients with AD. Secondary endpoints showed patients receiving the drug had no decline in cognition and negligible decline in function. The patients were evaluated over six months.

Allakos announced positive results from its Phase II trial of AK002 in patients with eosinophilic gastritis and/or eosinophilic gastroenteritis. Both dose arms showed clinically meaningful and statistically significant benefits compared to placebo on all primary and secondary endpoints, including GI tissue eosinophil counts and patient reported symptoms.

Basilea Pharmaceutica, based in Basel, Switzerland, announced positive topline data from its Phase III TARGET study of deftobiprole for acute bacterial skin and skin structure infections (ABSSSI). Ceftobiprole medocaril is a cephalosporin antibiotic for intravenous dosing. It has rapid bactericidal activity against a broad range of Gram-positive and Gram-negative bacteria, including methicillin-susceptible and resistant Staphylococcus aureus (MSSA, MRSA) and susceptible Pseudomonas species. It is currently approved and on sale in major European countries, Argentina, Canada, Jordan, Peru and Saudi Arabia for adults with community-acquired pneumonia (CAP) or hospital-acquired pneumonia (HAP), excluding ventilator-associated pneumonia (VAP).

The antibiotic is presently in Phase III development for a possible regulatory application in the U.S. The antibiotic hit the primary efficacy objective of non-inferiority to vancomycin plus aztreonam in the intent-to-treat (ITT) population. The primary endpoint of early clinical response was evaluated on a 20% or more decrease from baseline in lesion size at 48 to 72 hours after the antibiotic was dosed.

Regeneron and Sanofi announced positive Phase III trial of their Dupixent for severe atopic dermatitis in children six to 11 years of age. Already approved for severe atopic dermatitis in children ages 12 and up, as well as moderate-to-severe atopic dermatitis, the drug met its primary and secondary endpoints in the Phase III trial. Dupixient with standard-of-care topical corticosteroids significant improved measures of overall disease severity, including skin clearing, itching, and health-related quality of life.

Evelo Biosciences announced positive interim data from the first cohort in its Phase Ib trial of EDP1815 in mild to moderate psoriasis. The drug was well tolerated with no overall adverse effects than found with placebo. EDP1815 is a monoclonal microbial developed for inflammatory diseases. It is a strain of Prevotella histicola, which has shown potent immunomodulatory effects on human immune cells.

Leap Therapeutics announced that its anti-Dickkopf-1 (DKK1) antibody, DKIN-01, in combination with Merck’s Keytruda showed higher survival and objective response in advanced gastroesophageal junction and gastric cancer (GEJ/GC) tumors that expressed high levels of DKK1. It hit PFS in 22 weeks and median overall survival in 32 weeks, with a 50% overall response rate (ORR) and 80% disease control rate (DCR) in patients with DKK1-high GEJ/GC who had not received previous checkpoint inhibitor therapy.

Rhythm Pharmaceuticals announced positive topline data from two Phase III clinical trials of setmelanotide, its melanocortin-4 receptor (MC4R) agonist. The trials evaluated the drug for the treatment of pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities. Both trials met their primary endpoints as well as all key secondary endpoints. They showed statistically significant and clinically meaningful effect on weight loss and decreases in insatiable hunger, called hyperphagia, in both types of obesity.

AstraZeneca and Merck announced data from the Phase III PROfound trial of Lynparza (olaparib) in metastatic castration-resistant prostate cancer (mCRPC) who have a homologous recombination repair gene mutation (HRRm) and have progressed on previous treatment with new hormonal anticancer treatments. The primary endpoint was radiographic progression-free survival. The trial compared the drug against enzalutamide or abiraterone. The data showed a statistically-significant and clinically-meaningful improvement in the primary endpoint.

BioAegis Therapeutics Phase Ib/IIa trial of Recombinant Human Plasma Gelsolin in patients requiring hospitalization because of Community-Acquired Pneumonia showed favorable safety and pharmacokinetic results. There were no serious side effects and the PK supported once-a-day dosing. Although not an efficacy trial, there were signs of anti-inflammatory activities.

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Retrope completed enrollment in its Phase II/III clinical trial for RT001 for infants with Infantile Neuroaxonal Dystrophy (INAD). RT001 is a first-in-class drug in a new category called D-PUFAs (deuterated polyunsaturated fatty acids), which are designed to protect against free radical damage to omega 3 and 6 fats that cause cell death and are the hallmark of several neurodegenerative diseases, including INAD. INAD is an ultra-rare, life-shortening disorder that affects only a few hundred patients worldwide. It is caused by a genetic defect in the PLA2G6 housekeeping gene that removes damaged lipids from cell membranes.

DiscGenics reported it had treated its first patient in its clinical trial for IDCT for mild to moderate degenerative disc disease (DDD). The treatments are performed at Tokai University School of Medicine in Kanagawa, Japan. IDCT is a homologous, allogeneic, injectable cell therapy that uses engineered progenitor cells called Discogenic Cells. They have been derived from intervertebral disc tissue.

Akcea Therapeutics and Ionis Pharmaceuticals released data from the Phase III APPROACH trial evaluating Waylivra (volanesorsen) in familial chylomicronemia syndrome (FCS). Data was published in The New England Journal of Medicine. Waylivra lowered triglyceride levels below the threshold of risk for triglyceride-induced acute pancreatitis in the majority of patients. FCS is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and marked by severe high triglycerides levels, which increases the risk of acute pancreatitis.

AstraZeneca’s Tagrisso (osimertinib) showed a statistically-significant benefit in progression-free survival compared to other first-line therapies in non-small cell lung cancer (NSCLC) patients who were locally-advanced or metastatic epidermal growth factor receptor mutation-positive. The drug was compared in a Phase III clinical trial to first-line treatments gefitinib, an EGFR inhibitor, and elotinib (Tarceva), a tyrosine kinase inhibitor. Tagrisso is a third-generation EGFR and tyrosine kinase inhibitor.

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