Clinical Catch-Up: February 17-21

Biotech Research

Mid-February had a solid number of clinical trial announcements. Here’s a look.

Bristol-Myers Squibb announced five-year follow-up data from the Phase III CheckMate-025 trial, which continued to show that Opdivo (nivolumab) delivered superior overall survival (OS) and objective response rates (ORR) in patients with previously treated advanced or metastatic renal cell carcinoma (RCC) compared to those receiving everolimus. Opdivo is the company’s checkpoint inhibitor. With an extended minimum follow-up of 64 months, patients receiving Opdivo has an OS benefit with 26% of patients alive compared to 18% of patients treated with everolimus, and the objective response was 23% with Opdivo compared to 4% in the everolimus group.

Bellerophon Therapeutics announced positive topline data from its recently completed PHPF-002 Phase II clinical trial. The trial evaluated INOpulse for the treatment of Pulmonary Hypertension associated with Pulmonary Fibrosis (PH-PF). INOpulse is a proprietary pulsatile nitric oxide delivery system. The trial demonstrated that acute treatment with the INOpulse device had statistically and clinically significant improvement in hemodynamic markers, including decreased pulmonary vascular resistance by 21% with increased benefit on dose escalation from iNO30 to iNO45, a reduction in mean pulmonary arterial pressure of 12% and the iNO was well-tolerated with no safety concerns.

Foamix Pharmaceuticals announced integrated efficacy results from the FMX103 1.5% topical minocycline foam Phase III trial for rosacea. The results are from two pivotal Phase III trials for FMX103, which is under review by the FDA for moderate to severe papulopustular rosacea in adults. In the combined analysis, FMX103 showed statistically significant benefit compared to vehicle foam on both co-primary endpoints.

Mustang Bio treated the first patient with its optimized MB-106 in its ongoing Phase I/II trial in relapsed or refractory (r/r) B-cell non-Hodgkin lymphomas. MB-106 is a CD20-targeted, autologous CAR-T cell therapy developed in collaboration between Mustang and Fred Hutchinson Cancer Research Center. They also announced that the patient had achieved a complete response (CR) at the lowest starting dose.

Sunovion presented patient-reported home dosing and response diary data evaluating self-administered apomorphine sublingual film (APL-130277) for on-demand treatment of OFF episodes of Parkinson’s disease. The data suggested that self-administration of APL-130277 may lead to a full ON response for most cases of OFF episodes experienced throughout the day.

Cellectar Biosciences announced positive data from its Phase II CLOVER-1 trial of CLR 131 in r/r B-cell lymphomas. It also successfully completed its Phase I dose escalation study. CLR 131 achieved notable response rates in multiple myeloma patients, 34.5% ORR and 42% ORR in non-Hodgkin’s lymphoma (NHL) with a good safety profile. CLR 131 is a small-molecule Phospholipid Drug Conjugate that delivers iodine-131 directly to cancer cells.

Molecular Templates initiated dosing in its Phase I trial of TAK-169 in r/r multiple myeloma. TAK-169 was co-developed with Takeda Pharmaceutical and is a potential first-in-class CD38-targeting engineered toxin body (ETB).

Five Prime Therapeutics announced that Bristol-Myers Squibb reported that the Phase II trial of the company’s cabiralizumab with BMS’s checkpoint inhibitor Opdivo (nivolumab) with and without chemotherapy in advanced pancreatic cancer did not meet its primary endpoint. The trial enrolled about 160 patients with locally advanced or metastatic pancreatic cancer that had progressed during or after one line of chemotherapy. Cabiralizumab is an antibody that inhibits the CSF-1 receptor and blocks the activation and survival of monocytes and macrophages.

Kowa Research Institute randomized 10,000 patients into its international, multi-center Phase III cardiovascular outcomes trial. It is evaluating K-877 (pemafibrate) in high-risk patients with diabetes with high triglyceride and low HDL-C levels who are already taking statins. The study is evaluating the decrease of triglyceride levels and increase of functional HDL. K-877 is a selective peroxisome proliferator activator receptor-alpha (PPAR-alpha) modulator.

Teva Pharmaceutical’s deutetrabenazine for the treatment of tics in pediatric patients with moderate to severe Tourette Syndrome did not meet the primary endpoint in the Phase II/III ARTISTS 1 and Phase III ARTISTS 2 trials. Deutetrabenazine is approved by the U.S. Food and Drug Administration (FDA) under the brand name Austedo for the treatment of chorea associated with Huntington’s disease and for tardive dyskinesia in adults.

VistaGen Therapeutics announced positive results from its exploratory Phase IIa trial of PH10 for major depressive disorder (MDD). PH10 is an investigational first-in-class, rapid-onset synthetic neurosteroid nasal spray. In the single-site study, a rapid antidepressant benefit at the 6.4 microgram dose was seen by changes in HAM-D-17 scores at the end of the first week of treatment.

Advaxis released data from the monotherapy and combination arms of its ongoing Phase I/II trial of ADXS-503 in non-small cell lung cancer (NSCLC). ADXS-503 is part of its ADXS-HOT cancer-type specific immunotherapy program, which leverages the company’s Lm technology platform to target hotspot mutations. The trials evaluated the drug alone or in combination with Merck’s Keytruda. Nine patients have been dosed so far and 50% of the monotherapy arm showed stable disease, the first evaluable patient from the combination arm, who had progressed on Keytruda, showed stable disease with a 25% decrease in a site lesion.

VBL Therapeutics launched a new trial of VB-111 combined with the checkpoint inhibitor Bristol-Myers Squibb’s Opdivo (nivolumab) in metastatic colorectal cancer. The Phase II will investigate if priming with VB-111 can drive immune cells into the tumor and turn the colorectal tumor from immunologically “cold” to “hot.” VB-111 (ofranergene obadenovec) is a first-in-class, targeted anti-cancer gene-therapy agent.

Tetra Bio-Pharma initiated a proof-of-concept trial for its synthetic cannabinoid therapy (PPP003v) for the treatment of ophthalmic eye pain in the veterinary setting. Specifically, it will be tested to treat canine indolent corneal ulcers.

Brickell Biotech announced positive results from its Phase IIb trial of sofpironium bromide in primary axillary hyperhidrosis. The drug is a retro-metabolically designed, anticholinergic new molecular entity. Hyperhidrosis is where a person sweats more than the body requires to maintain its temperature. The drug applied topically at three different concentrations showed statistically significant differences relative to vehicle.

NoNO announced results from the pivotal Phase III ESCAPE-NA1 trial of intravenous nerinetide in patients with acute ischemic stroke who were chosen to undergo endovascular thrombectomy. The drug, without previous administration of alteplase (Genentech’s Activase), provided medically important improvements in the patients. The trial shows that the drug decreased the mortality rate of stroke from 19% to 11%.

CytoDyn received Institutional Review Board approval to begin its Phase II trial in about 22 different solid tumor cancers. The basket trial is a 30 patient, CCR5+ study of leronlimab. Leronlimab is a humanized IgG4 monoclonal antibody that blocks CCR5, a cellular receptor linked to HIV, tumor metastases, and other diseases.

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