Clinical Catch-Up: November 4-8

It was a busy week for clinical trial updates for the first week of November. Here’s a look.

Sanofi presented data from the EDITION JUNIOR Phase III trial at the International Society for Pediatric and Adolescent Diabetes 45th Annual Conference held in Boston. The trial compared Toujeo (insulin glargine 300 Units/mL) to insulin glargine 100 Units/mL (Gla-100) in children and adolescents aged 6 to 17 years with type 1 diabetes. The results of the trial showed that Toujeo had a comparable decrease in average blood sugar (HbA1C) and a similar risk of low blood sugar events.

Halozyme Therapeutics indicated its HALO-301 Phase III trial of PEGPH20 as first-line treatment of metastatic pancreas cancer did not hit the primary endpoint of overall survival. The treatment arm of PGPH20 in combination with gemcitabine and nab-paclitaxel did not show an improvement in median overall survival compared to gemcitabine and nab-paclitaxel alone. The company says it plans to halt PEGPH20 development activities.

Savonix and Boston University School of Public Health launched the first-ever population health Alzheimer’s Disease Discovery Study (ASSIST) to digitally collect brain health data from more than 400,000 participants. The goal is to identify connections between lifestyle choices like diet, sleep, exercise, social interactions, and other factors and the risk for developing dementia, including Alzheimer’s disease.

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CymaBay Therapeutics reached the targeted 240 patient enrollment goal for ENHANCE, its Phase III trial of seladelpar for Primary Biliary Cholangitis (PBC). Seladelpar is a selective, oral PPARδ agonist in development for PBC, primary sclerosing cholangitis and nonalcoholic steatohepatitis (NASH). The company also presented data from its Phase II trial and preclinical data for seladelpar at The Liver Meeting hosted by the American Association for the Study of Liver Diseases (AASLD).

Evelo Biosciences announced positive interim clinical data in its Phase Ib trial with EDP1815 for mild to moderate psoriasis. Eighteen patients with mild to moderate psoriasis were randomized 2:1 to receive either a high dose of EDP1815 or placebo for 18 days, with the primary endpoint of safety and tolerability. It also hit secondary efficacy endpoints. EDDP1815 is an oral monoclonal microbial, a strain of Prevotella histicola.

Vyome Therapeutics completed enrollment in its Phase II trial of VB-1953 in moderate to severe inflammatory acne. The trial has enrolled 471 patients. VB-1953 is a first-in-class topical bactericidal antibiotic.

Biohaven Pharma completed enrollment in the T2 Protect AD Study, a Phase II/III clinical trial of troriluzole in Alzheimer’s disease. Interim futility analysis of standard cognitive tests and volumetric MRI is expected before the end of 2019. Troriluzole is a third-generation prodrug and new chemical entity that modulates glutamate, an excitatory neurotransmitter.

Rhythm Pharmaceuticals announced additional data from two pivotal Phase III trials of setmelanotide for pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The data showed the drug’s effects on body mass index (BMI) and certain cardiovascular measures. The company had announced in August that the trials met their primary endpoints and all key secondary endpoints, showing a statistically significant and clinically meaningful effect on weight loss and decreases in insatiable hunger. Setmelanotide is a MC4R agonist for rare genetic disorders of obesity.

VBL Therapeutics IND for a Phase II trial of VB-111 in recurrent glioblastoma (rGBM) patients undergoing a second surgery was given the go-ahead from the FDA. VB-111 will be dosed either before and after the surgery or just after the surgery and be compared to a standard-of-care control group. VB-111 is a first-in-class gene therapy agent for a variety of solid tumors.

Syndax Pharmaceuticals dosed the first patient in its AUGMENT-101 Phase II clinical trial of SNDX-5613. SNDX-5613 is the company’s oral Menin inhibitor. It is being evaluated in relapsed/refractory acute leukemias. The Phase I part of the trial is a dose escalation study. The Phase II portion will evaluate efficacy.

Proteostasis Therapeutics completed enrollment in its Phase II trial of its proprietary combinations, PTI-428, PTI-801 and PTI-808 in cystic fibrosis. PTI 428 is an investigational CFTR amplifier being developed for CF in patients with at least one F508del mutation in the CFTR gene. PTI-808 is a novel potentiator and PTI-801 is a third-generation PTFR corrector.

Polynoma presented data from its Phase III MAVIS clinical trial of seviprotimut-L for melanoma. Seviprotimut-L is an allogeneic, polyvalent, partially purified shed melanoma antigen vaccine engineered from three proprietary human melanoma cell lines. It stimulates humoral and cellular immune responses. So far, the data indicates improved outcomes in Stage IIB/C patients and a favorable safety profile.

SCYNEXIS announced positive top-line results from its Phase III VANISH-303 trial of ibrexafungerp for women with vulvovaginal candidiasis (VVC), or vaginal yeast infection. Ibrexafungerp is an antifungal agent, the first of a novel class of structurally-distinct glucan synthase inhibitors called triterpenoids. The drug showed superiority over placebo at a highly statistically significant level.

Takeda’s Phase III TOURMALINE-MM4 trial met its primary endpoint of progression free survival (PFS) of Ninlaro (ixazomib) in adults with multiple myeloma not treated with stem cell transplantation. The trial is a randomized, placebo-controlled, double-blind Phase III trial of 706 patients. Ninlaro is an oral proteasome inhibitor. It was first approved by the FDA in November 2015 in combination with lenalidomide and dexamethasone for multiple myeloma patients who have received at least one previous therapy.

EIP Pharma announced that the REVERSE-SD trial of neflamapimod in early-stage Alzheimer’s met its secondary objectives of target engagement and proof-of-mechanism, showing statistically significant decreases relative to placebo in cerebrospinal fluid levels of phosphor-tau and tau. It did not meet the primary objective of improving episodic memory at the end of 24 weeks.

Bellerophon Therapeutics presented data from Cohort 1 of its ongoing Phase II/III trial of INOpulse for pulmonary hypertension associated with interstitial lung disease (PH-ILD). It confirmed the improvement previously reported, demonstrating a consistent and clinically meaningful benefit in moderate to vigorous physical activity (MVPA). INOpulse is a proprietary pulsatile nitric oxide delivery system.

Allena Pharmaceuticals announced positive topline results from URIROX-1, its Phase III trial of reloxaliase in enteric hyperoxaluria (EH), as well as more data from Study 206, its Phase II trial of the drug in high-risk patients with EH and advanced chronic kidney disease (CKD). Both studies showed substantial reductions in oxalate burden. URIROX-1 hit its primary endpoint, a statistically significant change from baseline in 24-hour urinary oxalate (UOx) excretion compared to placebo. Reloxaliase is an oral, recombinant oxalate-degrading enzyme.

Daiichi Sankyo Company announced that its ESAX-DN Phase III trial of esaxerenone met the primary endpoint in diabetic nephropathy. Esaxerenone is a novel mineralocorticoid receptor (MR) blocker. It is approved for hypertension in Japan and marketed as Minnebro. The primary endpoint was rate of remission of microalbuminuria after 52 weeks.

Aimmune Therapeutics presented new data on its AR101 peanut allergy therapy. AR101 is an investigational, peanut-derived, biologic drug candidate for oral immunotherapy in patients with peanut allergy. It delivers a daily dose of peanut protein with a consistent protein profile. The company presented results from a new analysis of the drug in the Phase III PALISADE and ARTEMIS trials. Both met the primary efficacy endpoints, showing improvements in developing a tolerance to peanut allergen.

Angion Biomedica presented data from its Phase II trial of ANG-3777 in kidney transplant patients with acute kidney injury associated with delayed graft function. The data indicate improved short-term and long-term graft function as measured by 12-month eGFR. ANG-3777 is a small molecule hepatocyte growth factor (HGF) mimetic.

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