Clinical Catch-Up: August 30-September 3
There were a fair number of clinical trial announcements last week. Here’s a look.
Relief Therapeutics and NRx Pharmaceuticals announced an additional finding in the Phase IIb/III trial of Zyesami (aviptadil) for ARDS in critical COVID-19. Patients receiving the drug demonstrated improvement in blood oxygen within a day of beginning treatment.
Rigel Pharmaceuticals published results from a Phase II trial of fostamatinib for hospitalized COVID-19 patients. The trial was sponsored by the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH), in partnership with Inova Health System. The data was from 59 patients and showed that adding fostamatinib to standard of care (SOC), including Gilead’s remdesivir and the steroid dexamethasone, was well tolerated and linked to clinically meaningful improvements in patients who required supplemental oxygen.
The National Institutes of Health (NIH) initiated a clinical trial to evaluate the antibody response to a booster dose of an authorized or approved COVID-19 vaccine in people with autoimmune diseases who did not respond to their initial COVID-19 vaccines. One of the goals is to determine whether pausing immunosuppressive therapy for their disease will improve the antibody response to a booster dose.
AlzeCure Pharma received the go-ahead from regulators in Sweden to launch a Phase I trial of ACD856 in Alzheimer’s disease. The drug comes from the company’s NeuroRestore platform, which develops compounds that simulate several important signaling pathways in the brain.
Poseida Therapeutics received the green light from the FDA for its Phase I trial of P-BCMA-ALL01, an allogeneic CAR-T candidate for relapsed/refractory multiple myeloma. It expects to follow a 3+3 design of dose-escalating cohorts. The company also presented preliminary Phase I data of P-PSMA-101, its solid tumor autologous CAR-T product for metastatic castrate-resistant prostate cancer (mCRPC). The data confirmed activity in solid tumors in the first nine patients.
Kinnov Therapeutics completed patient enrollment in its Phase II COCKTAIL study of KT-110 for alcohol use disorders. KT-110 is made up of a combination of cyproheptadine and prazosin, which has a therapeutic effect on addiction, regulating the reward system.
VBL Therapeutics resumes U.S. enrollment of new patients in its VB-111 studies. This came after the FDA Chemistry, Manufacturing and Controls (CMC) Group authorized new batches of ofranergene obadenovec (VB-111) produced in its GMP Modiin, Israel facility. The OVAL trial is studying the drug in ovarian cancer. VB-111’s mechanism of action is to blockade tumor vasculature with an anti-tumor immune response.
Boehringer Ingelheim announced results from the EMPEROR-Preserved Phase III trial of Jardiance (empagliflozin) for heart failure in adults with heart failure with preserved ejection fraction (HFpEF) compared to placebo. The drug demonstrated a 21% relative risk reduction for the composite primary endpoint.
Johnson & Johnson announced that its Imbokodo Phase IIb trial in HIV failed to hit the primary endpoint. The study was evaluating the company’s investigational HIV vaccine regimen in young women in sub-Saharan Africa at high risk of acquiring HIV. It did not provide sufficient protection against HIV. The virus uses an adenovirus design that the company also uses for its COVID-19 vaccine. As a result of the analysis, the Imbokodo trial will not continue. In parallel with the trial, Janssen, a J&J company, is sponsoring the ongoing Phase III Mosaico trial which is evaluating a different composition of the HIV vaccine regimen in men who have sex with men (MSM) and transgender individuals. The Mosaico trial is being run in the Americas and Europe, where, the company notes, there are different strains of HIV circulating than in sub-Saharan Africa. After consulting with the Mosaico study’s independent Data and Safety Monitoring Board (DSMB), the company decided Mosaico will continue.
AC Immune and Genentech announced that their Phase II Lauriet trial of semorinemab in mild-to-moderate Alzheimer’s disease hit one of its co-primary endpoints. The drug is an anti-tau monoclonal antibody. The trial hit the ADAS-Cog11 endpoint, which is the Alzheimer’s Disease Assessment Scale, Cognitive Subscale, 11-item Version at week 49. There was a statistically significant decrease in cognitive decline of 43.6% from baseline compared to placebo. However, the trial missed the other co-primary endpoint, decreasing the rate of functional decline from baseline measured by the Alzheimer’s Disease Cooperative Study-Activities of Daily Living (ADCS-ADL). It also missed secondary efficacy endpoints for the Mini-Mental State Examination (MMSE) and the Clinical Dementia Rating-Sum of Boxes (CDR-SB).
Bayer initiated Phase III development program OASIS of elinzanetant for vasomotor symptoms during menopause. The drug is under development as a potential non-hormonal, once-daily, oral, dual neurokinin-1,3 receptor antagonist.
iCAD announced encouraging results from a prospective two-center comparative study of Xoft Axxent Electronic Brachytherapy System for recurrent glioblastoma (GBM). The study evaluated 15 patients with recurrent GBM who were treated with maximal safe resection and Xoft intraoperative radiation therapy (IORT) and 15 treated with maximal safe resection and other modalities. In the IORT group, both overall survival and local progression-free survival continued to be superior to the control group.
Astellas Pharma paused the screening and dosing of its investigational gene therapy for patients with X-linked Myotubular Myopathy (XLMTM) after reports of a serious adverse event. AT132 is an AAV8 vector transporting a functional copy of the MTM1 gene.
Fractyl Health activated the tenth clinical trial site in its pivotal REVITA-T2Di trial in the U.S. and Europe. Revita DMR resurfaces the lining of the upper intestine. The study is evaluating the ability of the procedure to reduce or eliminate the need for insulin in patients with advanced type 2 diabetes.
Autolus Therapeutics published data from the Phase I ALLCAR19 trial of obecabtagene autoleucel (obe-cel). Abe-cel is a fast off-rate CD19 CAR-T therapy. The product demonstrated a good safety profile with high levels of CAR T-cell concentration in the blood at a median of 166.5 days.
Novartis announced that after conducting an interim analysis from its CIRRUS-1 trial of CFZ533 (iscalimab), it is discontinuing the trial in kidney transplant patients. The drug is dosed with tacrolimus and other immunosuppressive therapies to prevent organ rejection. The company indicated the drug will continue to be developed as a treatment for hidradenitis suppurativa and Sjögen’s syndrome and other autoimmune diseases. It is currently still being evaluated in the Phase II CONTRAIL trial in liver transplant patients, as well as other studies in type 1 diabetes and lupus nephritis.
Forte Biosciences plans to discontinue the development of FB-401 for atopic dermatitis after it failed to demonstrate statistical significance for its primary endpoint. FB-401 is made up of three therapeutic strains of a commensal gram-negative bacteria. The primary endpoint of Eczema Area and Severity Index (EASI)-50 was achieved by 58% of patients receiving the drug in comparison to 60% who received placebo.
Pfizer dosed the first patients in its Phase III study of its RSV bivalent prefusion F subunit vaccine candidate (RSVpreF) in adults 60 and older. It is expected to include 30,000 adults to assess safety and efficacy for prevention of moderate to severe respiratory syncytial virus (RSV) infections.
Relief Therapeutics announced that its Germany subsidiary, Advita Lifescience GmbH received clearance to start a Phase II trial in Germany of inhaled aviptadil for sarcoidosis. Aviptadil is a synthetic form of Vasoactive Intestinal Peptide (VIP).