Clinical Catch-Up: November 29-December 3
Heading out of November and into December, plenty of companies had clinical trial news to report. Here’s a look.
COVID-19-Related
BetterLife Pharma announced its wholly owned subsidiary, Altum Pharmaceuticals, and Pontificia Universidad Catolica de Chile successfully completed the Phase I part of the Phase I/II trial on AP-003 in COVID-19 patients. The drug is a proprietary inhaled interferon alpha-2b product. Of the 18 healthy patients, the therapy showed an excellent safety and tolerability profile with no serious adverse events.
Gritstone Bio announced that the SARS-CoV-2 T cell epitopes (TCEs) administered within its self-amplifying mRNA COVID-19 vaccines are minimally impacted by mutations found in the Omicron (B.1.1.529) variant. Gritstone’s CORAL program is a second-generation COVID-19 vaccine platform that delivers a stabilized Spike protein and highly conserved TCEs, which offer the potential for more durable protection and broader immunity over vaccines that focus only on the spike protein.
First Wave BioPharma announced an independent data monitoring committee recommended enrollment continue in Part 2 of the ongoing RESERVOIR Phase II trial of FW-COV for COVID-19-related GI infections. FW-COV is an oral tablet formulation of micronized niclosamide developed to remove SARS-CoV-2.
Relief Therapeutics reported that its partner NRx Pharmaceuticals completed analysis that its aviptadil patients with critical COVID-19 and Respiratory Failure showed improvement over existing therapies. Aviptadil is a synthetic form of Vasoactive Intestinal Peptide (VIP).
OSE Immunotherapeutics announced positive analysis of the first data of CoVepiT, its prophylactic vaccine candidate against COVID-19. They reported positive interim immunological data on T cell response in 100% of the treated population.
ARCA biopharma completed enrollment in ASPEN-COVID-19, a Phase IIb trial of rNAPc2 for hospitalized COVID-19 patients. rNAPc2 is a small recombinant protein, a potent, selective inhibitor of tissue factor (TF).
Humanigen published positive results in The Lancet Respiratory Medicine from its Phase III LIVE-AIR trial of lenzilumab in hospitalized COVID-19 patients. The study demonstrated the drug improved the likelihood of survival without the need for mechanical ventilation. The drug is a first-in-class monoclonal antibody that neutralizes GM-CSF, a cytokine vital to the hyperinflammatory cascade called cytokine release syndrome or cytokine storm, that is connected to COVID-19 and other disease and treatment conditions.
Revive Therapeutics announced it is expanding research with Bucillamine as a potential treatment for the Omicron variant in an ongoing Phase III trial for COVID-19. The drug is an oral anti-viral and anti-inflammatory.
Non-COVID-19-Related
SpringWorks Therapeutics hit full enrollment in its Phase IIb ReNeu trial of mirdametinib in adults and children with NF1-associated plexiform neurofibromas (NF1-PN). The drug is an MEK inhibitor. The study enrolled over 50 adults and over 50 pediatric patients with NF1-PN.
BioCryst Pharmaceuticals enrolled the first patient in the REDEEM-2 Phase III trial of BCX9930 in paroxysmal nocturnal hemoglobinuria (PNH). The drug is an oral Factor D inhibitor.
Celularity announced the FDA cleared its IND for CYNK-101 in combination with standard chemotherapy, trastuzumab and Merck’s Keytruda (pembrolizumab), in patients with first-line locally advanced unresectable or metastatic HER2/neu positive gastric or gastroesophageal junction (G/GEJ) adenocarcinoma. CYNK-101 is a genetically engineered natural killer (NK) cell therapy. The Phase I/IIa study will evaluate the efficacy of the combinations.
Krystal Biotech announced positive topline data from the GEM-3 Phase III trial of beremagene geperpavec (B-VEC, or Vyjuvek) for dystrophic Epidermolysis Bullosa. The primary endpoint was complete wound healing of topical Vyjuvek compared to placebo at six-month periods and it hit statistical significance. The therapy is a non-invasive, topical and redosable gene therapy.
Sigilon Therapeutics reported that fibrosed spheres were observed during a retrieval procedure in a patient in its Phase I/II trial of SIG-001 in severe or moderately severe hemophilia A. The trial was placed on clinical hold by the FDA in July 2021 after Sigilon reported a serious adverse event related to the development of inhibitors to Factor VIII in one of three patients treated. They performed a laparoscopic procedure to retrieve implanted spheres. They determined the spheres had fibrosed and cells within the spheres were no longer viable.
Kronos Bio announced positive early data from the ongoing Phase I/II trial of KB-0742 for MYC-amplified solid tumors. The drug is a highly selective, oral cyclin dependent kinase 9 (CDK9) inhibitor.
G1 Therapeutics initiated a new Phase II trial of Trilaciclib in combination with the antibody-drug conjugate (ADC) Trodelvy (Sacituzumab govitgecan-hziy) in unresectable locally advanced or metastatic triple-negative breast cancer. Trilaciclib (Cosela) is designed to decrease chemotherapy-induced myelosuppression.
Olema Pharmaceuticals released topline data for its Phase I/II trial of OP-1250 in metastatic breast cancer and other women’s cancers. In addition to the dose-escalation portion, there were clear efficacy signals in the early data. OP-1250 is a selective ER degrader (SERD).
Abivax reported good tolerability and promising clinical signals in its Phase I/II trial of AGX196 in heavily pre-treated hepatocellular cancer patients. The drug is administered with Bristol Myers Squibb’s checkpoint inhibitor Opdivo (nivolumab). ABX196 is a synthetic glycolipid agonist of invariant Natural Killer T cells (iNKT) in a liposomal formulation.
Zai Lab announced the Phase III PRIME study of Zejula (niraparib) as maintenance therapy in Chinese patients with newly diagnosed advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer (collectively ovarian cancer) after a platinum-based chemotherapy hit the primary endpoint, regardless of biomarker status. Zejula is an oral, once-daily poly (ADP-ribose) polymerase (PARP) inhibitor.
Mereo BioPharma reported promising interim efficacy, safety and biomarker data from its Phase Ib/II ACTIVATE study of etigilimab in combination with nivolumab in select recurrent advanced/metastatic solid tumors. The drug is an anti-TIGIT antibody.
Adagene reported the FDA cleared its IND for a Phase Ib/II trial of ADG116 in combination with Merck’s Keytruda (pembrolizumab) for patients with advanced/metastatic solid tumors. ADG116 is an anti-CTLA-4 monoclonal antibody.
Blade Therapeutics announced positive topline data from a Phase I drug-drug interaction study of cudetaxestat. The drug is a non-competitive autotaxin inhibitor in development for idiopathic pulmonary fibrosis (IPF).
Longeveron entered into a deal with the National Center for Geriatrics and Gerontology and Juntendo University Hospital in Japan to evaluate Lomecel-B in older, frail Japanese subjects. It will be a Phase II trial. Lomecel-B is a cell-based therapy derived from culture-expanded medicinal signaling cells sourced form bone marrow of young, healthy adult donors. The FDA granted it rare pediatric disease designation for Hypoplastic Left Heart Syndrome (HLHS), a life-threatening heart condition in infants.
Vertex Pharmaceuticals reported its Phase II proof-of-concept trial of VX-147 hit the mark in patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS). The drug hit a statistically significant and clinically meaningful decrease of 47.6% in the urine protein to creatinine ratio (UPCR) at Week 13 compared to baseline. Based on the data, the company plans to move it into Phase III studies in APOL1-mediated kidney disease, including FSGS, in the first quarter of 2022.
QSAM Biosciences began enrolling patients in its Phase I trial of CycloSam for multiple types of bone cancer. CycloSam (Samarium-153 DOTMP) is a bone targeting radiopharmaceutical developed by IsoTherapeutics Group.
Oncotelic Therapeutics initiated a Phase II trial of OT-101 with Merck’s Keytruda (pembrolizumab) for mesothelioma, specifically malignant pleural mesothelioma (MPM). OT-101 is a TGF-beta inhibitor.
Selecta Biosciences and Sobi completed enrollment in the Phase III DISSOLVE trial of SEL-212 for chronic refractory gout. SEL-212 is a combination of Selecta’s ImmTOR immune tolerance platform and a therapeutic uricase enzyme (pegadricase).
Arch Biopartners announced that its lead drug candidate, LSALT Peptide (LSALT and Metablok”) will enter the Canadian Treatments for COVID-19 (CATCO) human trial. LSALT will be evaluated in the Canadian trial in 65 Canadian hospitals.
Sage Therapeutics and Biogen announced positive, one-year data in major depressive disorder (MDD). The data demonstrated that 50 mg of zuranolone was well-tolerated in this population. The drug is an oral neuroactive steroid GABAA receptor positive allosteric modulator.
Statera Biopharma announced the FDA had lifted the clinical hold on its entolimod research and development activity in acute radiation syndrome (ARS). It said the company had “satisfactorily addressed historical regulatory matters.”
SAB Biotherapeutics reported that its Phase IIa study of SAB-176, a challenge study in adults infected with influenza virus, achieved statistically significant reductions in viral load and clinical signs and symptoms compared to placebo. SAB-176 is a quadrivalent fully human polyclonal antibody.
Surface Oncology reported the FDA had cleared its IND for GSK4381562 for a Phase I trial. The drug is a fully human IgG1 antibody targeting PVRIG, an inhibitory protein expressed on natural killer and T cells.
Karyopharm Therapeutics completed patient recruitment in the Phase III SIENDO trial of Selinexor as front-line maintenance therapy in advanced or recurrent endometrial cancer. The drug, with the brand name Xpovio, is a first-in-class selective inhibitor of nuclear export (SINE).
Fox Chase Cancer Center opened an investigator-initiated Phase I trial of gamitrinib in advanced cancer. The drug is a first-in-class, mitochondrial-targeted inhibitor of the molecular chaperone heat shock protein-90 (HSP90).
Dyne Therapeutics submitted an IND to the FDA for a clinical trial of DYNE-251 in Duchenne muscular dystrophy (DMD) amenable to skipping exon 51. The therapy is a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin 1 receptor.
Immutep announced the first five patients have been treated in the INSIGHT-003 Phase I trial. It a study of a triple combination therapy of eftilagimod alpha, carboplatin and an anti-PD-1 checkpoint inhibitor.
Exact Sciences published data from the RxPONDER trial led by the SWOG Cancer Research Network and sponsored by the National Cancer Institute (NCI). The trial defined the benefit of chemotherapy in early-stage node-positive breast cancer patients with Oncotype DX Breast Recurrence Score results of 0 to 25.
180 Life Sciences presented topline data from the Phase IIb adalimumab for Dupuytren’s disease. The drug is an anti-tumor necrosis factor (TNF) biologic.
Bayer announced that the Phase III ARASENS trial of Nubeqa (darolutamide) in prostate cancer hit the primary endpoint. Specifically, Nubeqa, an oral androgen receptor inhibitor, tested in metastatic hormone-sensitive prostate cancer, in combination with docetaxel and androgen deprivation therapy (ADT), significantly increased overall survival compared to docetaxel and ADT.