Clinical Catch-up for July 29 – August 2

Clinical Trial Data

Every week brings news of clinical trials, ranging from launches, first-patient dosing, full enrollment, interim data or final data. Here’s a look at some of the clinical trial news from last week.

Novartis announced that Entresto (sacubitril/valsartan) compared to valsartan in HFpEF patients “narrowly missed” hitting its primary endpoint in its Phase III PARAGON-HF study. The primary endpoint of the trial was the composite of total, both first and recurrent heart failure hospitalizations and cardiovascular death.

“The totality of evidence from the trial suggests that treatment with sacubitril/valsartan may result in clinically important benefits in HFpEF,” stated John Tsai, Global Drug Development and chief medical officer of Novartis.

Alkahest reported that its Phase IIa trial of AKST4290 met its primary endpoint of achieving an increase in best corrected visual acuity (BCVA) in patients with refractory wet or neovascular age-related macular degeneration (nAMD). The drug is an oral small molecule CCR3 inhibitor. It was also safe and well-tolerated. It was tested in patients who were no longer responding to anti-VEGF injections. Of the patients receiving AKST4290, 72% showed maintenance or improvement in BC VA.

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Cidara Therapeutics reported topline data from Part B of its global Phase II STRIVE trial of rezafungin in patients with candidemia and/or invasive candidiasis, which are fungal infections. Rezafungin is a novel echinocandin antifungal. The company previously reported positive topline results from Part A of the trial in March 2018. The objective of Part B was to compare efficacy and safety of the drug in once-a-week doses compared to caspofungin dosed once-daily. The topline results showed improvised outcomes compared to caspofungin across all efficacy measures.

Merck’s checkpoint inhibitor Keytruda (pembrolizumab) hit the primary endpoint in the company’s Phase III KEYNOTE-522 in triple-negative breast cancer (TNBC). Keytruda was used in combination with chemotherapy. The KEYNOTE-522 trial has 1,174 patients in it. It is a Phase III, randomized, double-blind trial studying Keytruda with chemotherapy compared to placebo plus chemotherapy as neoadjuvant therapy. The dual-primary endpoints are pCR and EFS. Secondary endpoints are pCR rate, EFS in patients expressing PD-L1, overall survival, safety and patient-reported outcomes.

Biogen and Alkermes announced positive topline data from EVOLVE-MS-2, a Phase III trial of diroximel fumarate for relapsing-remitting multiple sclerosis (RRMS) compared to Tecfidera (dimethyl fumarate). The EVOLVE-MS-2 Phase III trial ran for five weeks and was designed to evaluate the GI tolerability of diroximel fumarate 462 mg twice a day compared to Tecfidera 240 mg twice daily in 506 patients with RRMS. In the trial, diroximel fumarate was statistically superior to Tecfidera and patients receiving diroximel fumarate reported significantly fewer days of gastrointestinal symptoms. The most common adverse events for both treatment groups were flushing, diarrhea and nausea. Overall percentage of patients with AEs causing discontinuation were 1.6% for diroximel fumarate and 6% for Tecfidera.

BELLUS Health announced the first patient enrolled in the RELIEF Phase II trial of BLU-5937 for chronic cough. It also announced it is developing the drug for the treatment of chronic pruritus, which is expected to begin in 2020. The RELIEF study is a dose-escalation, placebo-controlled trial to study BLU-5937, a highly selective P2X3 antagonist, at four doses twice a day. It expects to enroll about 65 patients in the U.S. and UK at 12 clinical sites.

SELLAS Life Sciences Group dosed the first patient of its Phase I/II trial of GPS with Merck’s Keytruda in patients with selected WT1-positive advanced cancers, including solid tumors and hematologic cancers. GPS is SELLAS’ lead product candidate, licensed from Memorial Sloan Kettering Cancer Center. It targets the Wilms Tumor 1 (WT1) protein, present in numerous tumor types. The primary endpoints of the study are safety and overall response rate. It will enroll about 90 patients in up to 20 centers in the U.S.

Biohaven Pharmaceutical Holding Company enrolled the first patient in its Phase III trial to determine the efficacy and safety of verdiperstat in patients with Multiple System Atrophy (MSA). The drug is a potential first-in-class, oral, brain-penetrant myeloperoxidase inhibitor. Data from a previous Phase II trial in MSA run by AstraZeneca showed target engagement and positive trends over 12 weeks. MSA is a rare, rapidly progressive and fatal neurodegenerative disease.

Novartis announced that its Phase III MONALEESA-3 clinical trial of Kisqali (ribociclib) hit its key secondary endpoint, overall survival, in a pre-planned interim analysis. The MONALEESA-3 trial is evaluating the efficacy and safety of Kisqali plus fulvestrant in postmenopausal women with HR+/HER2- advanced or metastatic breast cancer in the first-line and second-line treatment settings.

AZTherapies completed target enrollment in its COGNITE Phase III clinical trial to determine the safety and efficacy of ALZT-OP1 for early Alzheimer’s disease. There are 620 patients in the trial after screening about 1,800 people. Patients were identified with a combination of cognition, function, and biomarker levels in the cerebrospinal fluid (CSF). The primary endpoint is change from baseline in Clinical Dementia Rating-Sum Boxes (CDR-SB) at week 72.

Bellerophon Therapeutics completed enrollment for Cohort 2 of its ongoing Phase II/III clinical trial of INOpulse to treat Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD). Cohort 2 includes 44 patients randomized 2:1 to receive either iNO45 or placebo. The company reported top-line data from Cohort 1 in January, which included 41 patients randomized 1:1 with iNO30 or placebo for 8 weeks. It showed clinically meaningful improvements in multiple activity parameters including oxygen saturation.

Amicus Therapeutics announced positive interim results from its CLN6 Batten disease gene therapy program. The Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital is running the ongoing Phase I/II clinical trial of a one-time intrathecal administration of AAV-CLN6 gene therapy for CLN6 Batten disease. It is a fatal neurologic illness that results in children losing the ability to walk, speak, think, and see. Interim efficacy data for the first eight children showed a positive impact on motor and language function compared to a natural history dataset, as well as comparisons within sibling pairs. Treatment was generally well-tolerated.

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AC Immune initiated its Phase Ib/IIa clinical trial to evaluate ACI-35.030, an anti-phospho-Tau vaccine for Alzheimer’s disease and other tauopathies. The primary objective is to assess the safety, tolerability and immunogenicity of different doses of ACI-35.030 in patients with early Alzheimer’s disease. Secondary objectives will be immunogenicity parameters while also evaluating endpoints that include notable biomarkers of progression of the disease as well as clinical assessments. The drug, as well as ACI-35, is being developed with Janssen Pharmaceuticals, a Johnson & Johnson company.

An experimental vaccine for respiratory syncytial virus (RSV) showed positive results in a Phase I clinical trial. Researchers from The University of Texas at Austin and others reported that a single dose of the vaccine produced significant evidence of RSV-neutralizing antibodies that were sustained for several months. The study was published in the journal Science and describes interim analysis of data from the first 40 adult volunteers in the trial. The data suggests the vaccine produces more than 10-fold increase in RSV-neutralizing antibodies compared to the number a person produces naturally from RSV exposure earlier in life.

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