It was another busy week for clinical trial announcements. Here’s a look.
It was another busy week for clinical trial announcements. Here’s a look.
COVID-19-Related
Cerecor announced final efficacy data including 60-day mortality from their U.S.-based Phase II trial of the human anti-LIGHT (TNFSF14) monoclonal antibody CERC-002. All patients in the trial were hospitalized with COVID-19 associated pneumonia and mild-to-moderate acute respiratory distress syndrome (ARDS). The trial hit its primary efficacy endpoint, proportion of patients alive and free of respiratory failure over the 28-day study period, compared to placebo in COVID-19 patients with ARDS treated with a single dose of CERC-002.
Altimmune initiated enrollment in its Phase I trial of AdCOVID, a single-dose intranasal COVID-19 vaccine candidate. It will evaluate the vaccine in up to 180 healthy adults between the ages of 18 and 55.
Sorrento Therapeutics received the go-ahead from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for its Phase I study of intranasal STI-2099 (COVIDROPS) for mild COVID-19. STI-2099 is a formulation of its antibody against COVID-19 that can be taken in a nasal spray. It is also evaluating the antibody in an intravenous form (STI-2020) in a Phase I trial. The Phase I study of STI-2099 will evaluate the safety and pharmacokinetics of the drug in both health volunteers and people with mild COVID-19.
Bharat Biotech, based in India, announced results vaccine efficacy of 81% for its COVID-19 vaccine. The interim data analysis was based on 43 recorded cases of the disease in the trial of 25,800 people. The Phase III trial was conducted in partnership with the Indian government’s medical research division. Of the 43 cases, 36 received a placebo, compared with seven who received the Bharat Biotech vaccine. The country approved the vaccine, COVAXIN, in January ahead of Phase III efficacy data.
Claritas Pharmaceuticals initiated a second development program with R-107, its nitric oxide-releasing compound. It is currently developing it for vaccine-resistant COVID-19, influenza and other viral infections. The new program will develop R-107 as a nasal spray to be used prophylactically to prevent viral infections.
Ridgeback Biotherapeutics published Phase I data of its molnupiravir, an antiviral being developed for COVID-19. Of the 130 healthy volunteers, fewer than half reported adverse events, with 93.3% reported as mild.
Brii Biosciences’ antibodies BRIIi-196 and BRII-198 failed the Phase III component of ACTIV-3. As a result, the company will halt the antibody combination’s progression into the Phase III trial in COVID-19 hospitalized patients.
Apellis Pharmaceuticals reported it will not continue to develop APL-9 in severe COVID-19. This was after an independent data monitoring committee performed an interim review of mortality data from the Phase I/II trial and found no meaningful decrease in overall mortality rate in patients receiving APL-9 with standard of care compared to standard of care alone.
Non-COVID-19-Related
Morphic Therapeutic announced positive interim results from its Phase I trial of MORF-057 for inflammatory bowel disease. MORF-057 is an oral small molecule inhibitor of the alpha4beta7 integrin. The drug was well-tolerated in all five cohorts receiving the drug in single doses with no serious adverse events and no significant lab abnormalities.
Annexon dosed the first patient in its Phase II ARCHER study of ANX-007 for Geographic Atrophy (GA). ANX007 is an anti-C1q therapy. GA is also known as atrophic age-related macular degeneration (AMD) or dry AMD.
ASLAN Pharmaceuticals announced positive interim unblinded data from the three dose cohorts of its ongoing ascending dose Phase I study of ASLAN004 for moderate to severe atopic dermatitis (AD). The drug was shown to be well-tolerated across all doses and demonstrated improvement compared to placebo in all efficacy endpoints. The drug is a potential first-in-class human monoclonal antibody that binds to the IL-13 receptor alpha1 subunit, blocking signaling of two pro-inflammatory cytokines, IL4 and IL-13.
Accure Therapeutics enrolled the first patient in its ACUITY Phase II trial in acute optic neuritis (AON). The study is evaluated ACT-01, a peptidomimetic first-in-class disease-modifying treatment also being developed for multiple sclerosis.
Transgene and BioInvent International enrolled the first patient in a Phase I/IIa trial of BG-001 in solid tumors. BT-001 is an oncolytic virus created with Transgene’s INVIR/IO platform. It encodes a Treg-depleting human recombinant anti-CTLA4 antibody generated by BioInvent’s n-CoDeR/F.I.R.S.T. platforms, and the human GM-CSF cytokine.
Solid Biosciences presented one-year efficacy and safety data from the ongoing IGNITE-DMD Phase I/II trial of SGT-001 in Duchenne muscular dystrophy. SGT-001 is a microdystrophin gene therapy.
Avadel Pharmaceuticals published data from four Phase I studies from its FT218 pharmacokinetic program. FT218 is a once-nightly formulation of sodium oxybate that includes Avadel’s MicroPump controlled-release (CR) technology. It is being developed for excess daytime sleepiness and cataplexy in adults with narcolepsy.
NGM Biopharmaceuticals dosed the first patient in an expansion of its ongoing Phase Ib proof-of-concept trial of NGM120 in patients with metastatic pancreatic cancer. NGM120 is an antagonistic antibody that binds glial cell-derived neurotrophic factor receptor alpha-like (GFRAL) and inhibits growth differentiation factor 15 signaling.
Orbus Therapeutics announced positive results from the pre-planned interim analysis for futility for its Phase III STELLAR trial of eflornithine in recurrent anaplastic astrocytomas. The Independent Data Monitoring Committee recommended the trial proceed without modifications. Eflornithine is a novel cytostatic agent that irreversibly inhibits ornithine decarboxylase, a key enzyme in mammalian polyamine biosynthesis that is up-regulated in some cancers.
Kymera Therapeutics initiated dosing in the single ascending dose portion of its Phase I trial of KT-474 for atopic dermatitis or hidradenitis suppurative. KT-474 is a potential first-in-class, highly active and selective, orally bioavailable IRAK4 degrade being developed for toll-like receptor (TLR)/interleukin-1 receptor (IL-1R)-driven immune-inflammatory diseases.
SCYNEXIS announced positive results from the third interim efficacy analysis of the ongoing open-label Phase III FURI trial and the first interim analysis of the ongoing open-label Phase III CARES study. FURI is evaluating oral ibrexafungerp as a salvage treatment for difficult-to-treat mucocutaneous and invasive fungal infections that are refractory to, intolerant of current standards of care, or require a non-azole oral step-down therapy for the treatment of azole-resistant species. Antifungal activity was consistently positive across all interim analyses. CARES is focused on hospitalized patients with invasive candidiasis caused by the emerging Candida auris, which is often multi-drug resistant and associated with high mortality. The CARES study found that 80% of patients with invasive candidiasis and candidemia due to C. auris experienced a complete response, and one patient died of an underlying condition unrelated to treatment and one patient was considered indeterminate.
Clearside Biomedical completed dosing the first cohort of its Phase I/IIa OASIS trial of CLSL-AX (axitinib injectable suspension) in neovascular age-related macular degeneration (wet AMD). CLS-AX is a proprietary suspension of axitinib for suprachoroidal injection. Axitinib is a tyrosine kinase inhibitor (TKI) currently approved for renal cell cancer that achieves pan-VEGF blockade.
Aileron Therapeutics expanded enrollment for its Phase Ib trial of ALRN-6924 in advanced non-small cell lung cancer (NSCL) patients undergoing chemotherapy. ALRN-6924 is a novel chemoprotective drug that selectively protects healthy cells in patients with cancers that harbor p53 mutations to reduce or eliminate chemotherapy-induced side effects. It is a first-in-class MDMS/MDMX dual inhibitor administered before chemotherapy.
Eli Lilly and Incyte announced topline results from BRAVE-AA2, a Phase III study of once-daily baricitinib 2-mg and 4-mg in adults with severe alopecia areata. Both doses hit the primary efficacy endpoint at Week 36, demonstrating a statistically significant improvement in scalp hair regrowth. Olumiant (baricitinib) 2-mg is indicated for adults with moderately to severely active rheumatoid arthritis. It is a JAK inhibitor.
Azura Ophthalmics announced topline results from its Phase II program of AZR-MD-001 for treatment of Meibomian gland dysfunction (MGD) and related eye diseases. The trial met its primary endpoints, demonstrating improvements in signs and symptoms of MGD. AZR-MD-001 is a topical ointment that leverages SeS2 as the active ingredient. SeS2 breaks down the bonds between abnormal keratin proteins to soften the blocks and increases the lipid produced by the Meibomian glands.
NervGen Pharma received the greenlight from the FDA to initiate a Phase I trial of NVG-291 in healthy volunteers. The drug is a specific and selective protein tyrosine phosphatase sigma inhibitor being developed for spinal cord injury, multiple sclerosis and Alzheimer’s disease.
Apollomics enrolled the first patient in its Phase I trial of APL-106 (uproleselan injection) for adults with r/r acute myeloid leukemia (AML) in China. APL-106 is designed to block E-selectin, an adhesion molecule on cells in the bone marrow, from binding with blood cancer cells, which should disrupt the mechanism of leukemic cell resistance within the bone marrow microenvironment.
Kronos Bio wrapped its End-of-Phase II meeting with the FDA and announced plans to proceed with its plan to assess measurable residual disease (MRD) negative complete response (CR) as the primary endpoint in a Phase III trial of entospletinib in newly diagnosed NPM1-mutated AML. Entospletinib is a selective inhibitor targeting spleen tyrosine kinase (SYK).
Ocuphire Pharma published results from its MIRA-1 Phase IIb trial of Nyxol in reversing mydriasis (dilation of the pupil for eye exams). The study showed Nyxol is a promising candidate to improve patient experience after dilated eye exams.
