January 10, 2022
The biopharma industry has started 2022 with plenty of clinical trial news. Here’s a look.
Celltrion Group announced Phase I results from its antibody cocktail of Regkirona and CT-P63. One Phase I study tested CT-P63 in 24 healthy participants in Poland. The trial hit the primary objective of safety and tolerability. It also demonstrated strong neutralizing activity against the Omicron variant. CT-P63 is a monoclonal antibody that targets the SARS-CoV-2 spike receptor binding domain (RBD) and comes in a nebulized formulation. A second drug being investigated is CT-P59 (regdanvimab), which in studies has been shown to strongly bind to RBD and neutralize the wild type and mutant variations of concern. And in in vivo models, it has effectively decreased the viral load of SARS-COV-2 and lung inflammation. Regkirona is given intravenously.
Gritstone bio announced positive Phase I data from the first cohort of its CORAL-BOOST study. The study is evaluating a 10-microgram dose of CORAL self-amplifying mRNA vaccine. The vaccine provided strong neutralizing antibody responses to the virus’s spike protein and robust CD8+ T cell responses. CORAL-BOOST is one of four clinical trials in Gritstone’s CORAL program, which is evaluating use of the vaccine as a booster shot in people who received two doses of AstraZeneca and Oxford University’s first-generation COVID-19 vaccine, Vaxzevria.
ARCH Biopartners reported the Research Ethics Board at the Sunnybrook Research Institute had approved the amendment to the CATCO protocol to include LSALT Peptide (LSALT and “Metablok”) in a new arm of the CATCO human trial. CATCO will request approval from Health Canada before beginning dosing in the new arm of the trial for COVID-19. CATCO is a study being run in up to 55 hospitals across Canada to find new treatments for people with complications caused by COVID-19.
Intravacc and Dutch Leiden University Medical Center announced a partnership for a Phase I/II trial of a new vaccine, NANOVAC, a broad coronavirus vaccine that could be effective for COVID-19, SARS, MERS and other beta coronaviruses. NANOVAC is based on minuscule soluble nano-spheres that contain synthetic mini proteins, offering broad protection in both arms of the immune system.
Clover Biopharmaceuticals dosed the first participants in its ongoing Phase II/III SPECTRA trial of SCB-2019, its COVID-19 vaccine candidate, as a homologous booster dose after primary vaccination of SCB-2019. The company reported final efficacy data for the vaccine in SPECTRA in September 2021.
Daxor Corporation completed patient enrollment and data analysis is underway for its observational prospective study of BVA-100 in hospitalized COVID-19 patients. BVA-100 is a Blood Volume Analyzer that measures capillary permeability via an albumin tracer.
First Wave BioPharma completed enrollment for Part 2 of the RESERVOIR Phase II trial of FW-COV as an outpatient treatment for COVID-19-related GI infections. FW-COV is a proprietary, oral, tablet formulation of micronized niclosamide.
Spero Therapeutics announced the FDA had lifted a clinical hold on the Phase IIa trial of SPR720. The drug is an oral antimicrobial agent being studied in patients with nontuberculous mycobacterial pulmonary disease (NTM-PD). SPR720 is from a novel class of antibacterial agents. They target enzymes required for bacterial DNA replication.
Achieve Life Sciences announced the last study follow-up visit for the last participant enrolled in the ORCA-2 Phase III trial of cytisinicline for smoking cessation. Cytisinicline is a plant-based alkaloid with high binding affinity to the nicotinic acetylcholine receptor.
Vaccinex reported positive interim safety data from the Phase Ib segment of the KEYNOTE B84 combination trial of Merck’s Keytruda (pembrolizumab) and Vaccinex’s pepinemab in advanced, recurrent or metastatic head and neck squamous cell carcinoma. They now expect to start enrollment for the Phase II segment. Pepinemab is a humanized IgG4 monoclonal antibody that inhibits SEMA4D. Keytruda is a PD-L1 checkpoint inhibitor.
AVEO Oncology entered into a clinical trial collaboration and supply deal with Merck KGaA, Darmstadt, Germany, to study ficlatuzumab with Erbitux (cetuximab) in recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC). Ficlatuzumab is AVEO’s humanized IgG1 monoclonal antibody that targets hepatocyte growth factor. Erbitux is an EGFR-targeted antibody.
ORYZON Genomics received approval from the Serbian Medicines and Medical Devices Agency (ALIM) for its Clinical Trial Application (IND equivalent) for a Phase IIb trial with vafidemstat in patients with Borderline Personality Disorder (9BPD) in Serbia. Vafidemstat is an oral, CNS optimized LSD1 inhibitor.
Alladapt Immunotherapeutics completed patient enrollment in the Phase I/II HARMONY trial of ADP101 for food allergy. ADP101 is a potentially best-in-class oral immunotherapy designed to desensitize patients allergic to one or multiple foods.
Viridian Therapeutics submitted an IND to the FDA for VRDN-002 for the treatment of thyroid eye disease. The drug is a humanized monoclonal antibody with half-life extension technology.
COMPASS Pathways published results from a Phase I trial of COMP360 psilocybin, which is being developed for treatment-resistant depression. The drug is a proprietary formulation of synthetic psilocybin.
ProQR Therapeutics announced the last patient completed the last visit (Month 12) in the Phase II/III Illuminate trial of sepofarsen for CEP290-mediated Leber Congenital Amaurosis 10 due to the p.Cys998X mutation. The drug is a first-in-class RNA therapy, designed to enable normal splicing resulting in restoration of normal CEP290 mRNA and subsequent production of functional CEP290 protein.
OnQuality Pharmaceuticals announced that Part 1 of the Phase II NOVA-II trial of OQL011 hit the company’s expectations of safety, efficacy and patient reported outcome of Hand-Foot Skin Reaction (HFSR)-related pain. HFSR is a common side effect of tyrosine kinase inhibitors and VEGFR inhibitors used to treat cancer. OQL011 is a proprietary ointment designed to locally activate VEGF downstream signaling pathways to treat HSFR.
The Wistar Institute and Stanford University School of Medicine initiated a Phase II trial of VK-2019 in patients with advanced Epstein-Barr Virus (EBV)-positive nasopharyngeal carcinoma (NPC) and lymphoma. VK-2019 is an oral, small molecule inhibitor of the latent form of EBV.
Zosano Pharma published Phase I data from a low dose of a trivalent influenza vaccine administered via the company’s transdermal microneedle system. It generated antibody levels that met EMEA’s three guidelines for influenza vaccine efficacy.
Hepion Pharmaceuticals presented data from the Phase II AMBITION trial of CRV431 in nonalcoholic steatohepatitis (NASH). The drug is a potent inhibitor of cyclophilins.
Oncotelic Therapeutics initiated a Phase I study of CA4P in combination with Merck’s anti-PD-1 checkpoint inhibitor Keytruda (pembrolizumab) for melanoma. CA4P is a vascular disrupting agent.
Longeveron initiated a Phase IIa trial of Lomecel-B for Alzheimer’s disease. The first patient has agreed to the trial and patient screening has begun. Lomecel-B is a cell-based therapy derived from culture-expanded medicinal signaling cells (MSCs) sourced from bone marrow of young, healthy adult donors.
InflaRx initiated the Phase III trial of vilobelimab in hidradenitis suppurative (HS) patients with active draining tunnels. Vilobelimab is a first-in-class monoclonal anti-human complement factor C5a antibody.
Connect Biopharma reported positive data from the Phase IIb trial of CBP-201 administered subcutaneously to adults with moderate-to-severe atopic dermatitis. The drug hit the primary endpoint of eczema area and severity index (EASI) percent reduction from baseline at Week 16. CBP-201 is an antibody that targets interleukin-4 receptor alpha.
Anexxon announced interim data from its ongoing Phase II trial of ANX005 in Huntington’s disease. ANX005 is a monoclonal antibody that blocks C1q and the entire classical complement pathway. Interim data demonstrated the drug was generally well-tolerated with full target engagement of C1q in both serum and CSF.
Oncternal Therapeutics and the FDA agreed on key elements of the company’s Phase III trial of zilovertamab for r/r mantle cell lymphoma. Zilovertamab is a potentially first-in-class monoclonal antibody against receptor tyrosine kinase-like Orphan Receptor 1 (ROR1).
Affimed completed enrollment in the Phase II REDIRECT trial of AFM13 monotherapy in r/r CD30-positive peripheral T-cell lymphoma. AFM13 is a bispecific tetravalent Innate Cell Engager (ICE) targeting CD30 on tumor cells and CD16A on NK cells and macrophages.
Avalo Therapeutics announced its Phase Ib trial of AVTX-002 in moderate to severe Crohn’s disease demonstrated meaningful mucosal healing. AVTX-002 is a fully human anti-LIGHT monoclonal antibody.
IN8bio provided an update on its ongoing Phase I trial of INB-200 in patients newly diagnosed with glioblastoma multiforme (GBM). Cohort 1 accrual and treatment is complete with three patients receiving a single dose of DeltEx DRI via intracranial infusion concurrent with maintenance TMZ administration. INB-200 is an autologous DeltEx drug resistant immunotherapy made up of gamma-delta T cells genetically engineered to be chemotherapy resistant.
ObsEva announced positive topline data from the Phase III EDELWEISS 3 trial of linzagolix in moderate-to-severe endometriosis-associated pain (EAP). The drug is an oral GnRH antagonist. The 200 mg dose hit the co-primary efficacy objectives, showing decreases in dysmenorrhea and non-menstrual pelvic pain at three months.
Nanobiotix enrolled the first patient in the NANORAY-312 Phase III trial of NBTXR3 in head and neck cancer. NBTXR3 is a novel, potentially first-in-class oncology therapy composed of functionalized hafnium oxide nanoparticles administered by one-time intratumoral injection and activated by radiotherapy.
23andMe dosed the first patient in a Phase I trial of 23ME-00610 for advanced solid tumors. 23ME-00610 is the company’s first wholly-owned immuno-oncology antibody identified via its proprietary genetic and health survey database.
Rafael Pharmaceuticals completed the first cohort of dose escalation with no dose-limiting toxicity in the APOLLO 613 Phase I/II trial of CPI-613 (devimistat) in combination with hydroxychloroquine in patients with relapsed clear cell sarcoma. Devimistat targets the mitochondrial tricarboxylic acid (TCA) cycle.
Aligos Therapeutics has halted further development of its STOPS drug candidate, ALG-010133 being developed in a Phase I trial for chronic hepatitis B. They have halted it because emerging data show that at the projected efficacious dose of 400 mg there is no meaningful HBsAg reduction.
BioCryst Pharmaceuticals enrolled the first patient in the REDEEM-1 Phase III trial of BCX9930 in paroxysmal nocturnal hemoglobinuria (PNH). The drug is an oral Factor D inhibitor.
BioSight announced final results for its Phase IIb trial of single-agent aspacytarabine as first-line acute myeloid leukemia for patients unfit for standard induction chemotherapy. They reported final results for the primary endpoint, complete remission. Aspacytarabine is a novel anti-metabolite composed of cytarabine covalently bound to asparagine, acting as a prodrug of cytarabine.
Avita Medical completed enrollment in its pivotal trial for RECELL System for soft-tissue reconstruction. It is currently indicated in the U.S. for acute thermal burns. They expect topline data later this year.
Bolt Biotherapeutics dosed the first patient in a new combination arm of the ongoing Phase I/II trial of BDC-1001 with Bristol Myers Squibb’s PD-1 checkpoint inhibitor Opdivo (nivolumab) for patients with HER2-expressing solid tumors. The drug is a HER2-targeting Boltbody immune-stimulating antibody conjugate (ISAC)(trastuzumab biosimilar conjugated to a toll-like receptor 7 and 8 agonists).
4D Molecular Therapeutics dosed the first patient in the Phase I/II trial of 4D-150 for neovascular age-related macular degeneration (wet AMD). The drug is ai dual-transgene, intravitreal gene therapy that inhibits four distinct angiogenic factors to prevent angiogenesis and reduce vascular permeability.
Deinove announced the independent Data Safety Monitoring Board (DSMB) completed its review of the data from the first part of the Phase II trial of DNV3837 for Clostridioides difficile infection (CDI). They recommended continuing recruitment. DNV3837 is an IV antibiotic that converts to its active form DNV3681 and crosses the GI barrier and accumulates in the intestinal lumen.