Clinical Catch-Up: May 31-June 5
As summer begins, we’re seeing a shift away from COVID-19-related news and a stronger output of other clinical trial news. Here’s a look.
Gilead Sciences announced topline results from the Phase III SIMPLE trial of remdesivir plus standard of care (SOC) compared to SOC alone in hospitalized patients with moderate COVID-19 pneumonia. It evaluated 5-day and 10-day courses. The trial showed that patients in the 5-day remdesivir cohort were 65% more likely to have clinical improvement at Day 11 compared to the SOC group. In the 10-day group it trended toward improvement but did not reach statistical significance.
Eli Lilly and AbCellera dosed the first patients in its trial of LY-COV555 in COVID-19. LY-COV555 is a potent, neutralizing IgG1 monoclonal antibody directed against the spike protein of SARS-CoV-2. The patients were dosed at several major medical centers in the U.S., including NYU Grossman School of Medicine and Cedars-Sinai in Los Angeles.
Relief Therapeutics and NeurRx treated the first patients with RLF-100 at the University of Miami Miller School of Medicine as part of a Phase IIb/III trial to evaluate RLF-100 for acute respiratory disease syndrome (ARDS) in COVID-19. RLF-100 (Aviptadil) is a patented formulation of Vasoactive Intestinal Polypeptide (VIP), which is known to be highly concentrated in the lungs and inhibits a variety of inflammatory cytokines.
TLC announced topline data from the TLC590 Phase II clinical trial in pain reduction after bunionectomy. TLC590 is a non-opioid, proprietary BioSeizer sustained release formulation of ropivacaine. The overall decrease in pain intensity in the patients receiving the drug was greater than that of placebo as well as bupivacaine at every time interval from 0 hours through the end of the study at 168 hours.
Takeda Pharmaceutical presented results from the Phase II Pevonedistat-2001 trial at ASCO and the European Hematology Association (EHA) Annual Meeting. The trial evaluated pevonedistat plus azacytidine compared to azacytidine alone in rare leukemias, including higher-risk myelodysplastic syndromes (HR-MDS). The data showed the combination suggested benefits in the HR-MDS subgroup across multiple clinically meaningful endpoints.
Genentech, a Roche company, announced positive results from the Phase II CITYSCAPE trial of tiragolumab plus Tecentriq (atezolizumab) compared to Tecentriq alone as a first-line treatment for PD-L1-positive metastatic non-small cell lung cancer (NSCLC). Tiragolumab is an immune checkpoint inhibitor binding to TIGIT.
Janssen, a Johnson & Johnson company, presented four different studies at ASCO related to various drugs. First, they announced final analysis of the pivotal Phase III SPARTAN trail of Erleada (apalutamide) in combination with androgen deprivation therapy (ADT), which significantly improved overall survival (OS), compared to ADT alone in non-metastatic castration-resistant prostate cancer (nmCRPC). Janssen announced updated results from the Phase Ib/II CARTITUDE-1 trial of JNK-4528, an investigational B-cell maturation antigen (BCMA)-directed T-Cell therapy in r/r multiple myeloma. All patients responded to treatment and had deep and durable responses. Third, they reported first-time results from a Phase I first-in-human dose escalation trial of teclistamab (JNJ-7957) an antibody against BCMA and CD3 receptors on T-cells in r/r multiple myeloma. Finally, Janssen announced results from the Phase I CHRYSALIS trial of amivantamab (JNJ-6372) in advanced NSCLC with EGFR Exon 20 insertion mutations. Amivantamab is an EGFR and mesenchymal epithelial transition factor (MET) bispecific antibody.
Deciphera Pharmaceuticals presented quality of life and certain safety results from the INVICTUS Phase III trial of QINLOCK (ripretinib) in adults with fourth-line advanced gastrointestinal stromal tumor (GIST) at ASCO. The drug was associated with an increase in the patients’ self-reported health status, while placebo was associated with decline.
Myovant Sciences announced additional data from its Phase III HERO trial of once-daily, oral relugolix in men with advanced prostate cancer at ASCO. The data expanded on earlier findings from the study, showing superiority of relugolix to leuprolide acetate across multiple endpoints.
AbbVie presented new safety and efficacy results for Rinvoq (upadactinib) in adults with moderate to severe active rheumatoid arthritis, primary data of the drug in psoriatic arthritis and additional data on Humira (adalimumab) in psoriatic arthritis at the European E-Congress of Rheumatology (EULAR) 2020. They presented 25 abstracts across multiple rheumatic conditions.
VBL Therapeutics presented positive interim data from the OVAL Phase III pivotal trial in platinum-resistant ovarian cancer of VB-111 and paclitaxel compared to placebo plus paclitaxel. VB-111 (ofranergene obadenovec) is a first-in-class, targeted anti-cancer gene-therapy agent. The data demonstrated an objective response rate of 53%, with the response rate in the treatment arm being 58% or higher. In patients who had post-dosing fever, a marker for VB-111 treatment, the response rate was 69%.
Celyad provided updates from its allogeneic programs, including additional data from the Phase I alloSHRINK trial of the T-cell receptor inhibitory molecule-based, non-gene edited allogeneic CAR-T candidate, CYAD-101 for metastatic colorectal cancer and its short hairpin RNA (shRNA) platform for its next-generation, non-gene edited CYAD-200 series of CAR-T candidates.
Rgenix presented data showing clinical activity in advanced colorectal tumors from a Phase I trial of RGX-202. RGX-202 is a first-in-class small molecule that inhibits the SLC6a/CKB pathway. The ongoing Phase I trial is an escalation/expansion study of the drug with or without FOLFIRI in patients with advanced GI tumors.
Rezolute announced Phase IIa data in adults and children with congenital hyperinsulinism (HI) receiving RZ358. The data showed that the drug raises glucose into a normal target range. RZ358 is an IV human monoclonal antibody that binds to an allosteric site on the insulin receptor.
Alkahest initiated a Phase II trial of AKST1210-201 to remove excess Beta-2 microglubulin (B2M) from circulation for the treatment of cognitive impairment in patients undergoing hemodialysis for end stage renal disease (ESRD). AKST1210 is an extracorporeal device that is connected in series with a standard hemodialysis circuit.
Mirum Pharmaceuticals reported an analysis from its Phase II INDIGO open-label trial of maralixibat in patients with bile acid salt export pump (BSEP) deficiency (PFIC2). The data showed that patients with long-term maralixibat treatment who achieved sBA control have five-year transplant-free survival after treatment. Maralixibat is a novel, minimally-absorbed, oral drug that inhibits the apical sodium dependent bile acid transporter.
Menlo Therapeutics reported positive data from its Phase II trial of FCD105 (3% minocycline/0.3% adapalene foam) for moderate-to-severe acne vulgaris. The study enrolled 447 patients in the U.S. who either received FCD105 foam, 3% minocycline foam, 0.3% adapalene foam, or vehicle (placebo) foam.
NKMax America received FDA clearance for a Phase I/IIa trial of SNK-01 in combination with Herceptin (trastuzumab) or Erbitux (cetuximab) for advanced/metastatic HER2- or EGFR-expressing cancers. SNK-01 is an autologous natural killer cell therapy.
TG Therapeutics announced positive topline data from the UNITY-CLL Phase III trial of umbralisib plus ublutuximab (U2) compared to obinutuzumab plus chlorambucil in patients with previously untreated and r/r chronic lymphocytic leukemia (CLL). The trial met the primary endpoint at a prespecified interim analysis, showing statistically significant improvement in PFS. It will be halted early for superior efficacy.
Satsuma Pharmaceuticals completed enrollment and randomization of its EMERGE pivotal Phase III efficacy trial of STS101 (dihydroergotamine (DHE) nasal powder) for acute treatment of migraine. The EMERGE trial is a multi-center, randomized, double-blind, placebo-controlled, parallel group trial in more than 1,140 migraine patients being conducted at 121 sites across 32 U.S. states.
Bristol Myers Squibb announced results from True North, a pivotal Phase III trial of oral Zeposia (ozanimod) as an induction and maintenance therapy for adults with moderate to severe ulcerative colitis. The trial met both primary endpoints, showing highly statistically significant data for induction of clinical remission at Week 10 and in maintenance at Week 52. It also met key secondary endpoints of clinical response and endoscopic improvement.
Novus Therapeutics announced that its Phase IIa clinical trial of OPO201 in acute otitis media, or inflammation of the middle ear, failed to meet its primary endpoint. Study C-006 was evaluating the safety, tolerability and efficacy of 20 mg per day intranasal OP0201 as adjunct to oral antibiotics to treat acute otitis media in infants and children 6 to 24 months of age. Patients received the drug twice a day for 10 days with a one-month follow-up. The primary efficacy endpoints included resolution of bulging tympanic membrane at the second visit and resolution of middle ear effusion at the third visit.
Corbus Pharmaceuticals completed the final visit in the company’s RESOLVE-1 Phase III of lenabasum for systemic sclerosis. Topline data is expected in the summer 2020. Lenabasum is an oral, small molecule that selectively binds as an agonist to the cannabinoid receptor type 2 (CB2), resolves inflammation and limits fibrosis.
Horizon Therapeutics announced a series of data presentations showing Krystexxa (pegloticase injection) can be co-prescribed with three commonly used immunomodulators in rheumatology to help more people with chronic gout refractory to conventional treatments. This included new data from the MIRROR open-label trial of the drug with methotrexate, and analysis of real-world data of Krystexxa with immunomodulators.
Eli Lilly presented 29 abstracts at the virtual European Congress of Rheumatology 2020 on the use of Taltz and Olumiant in several rheumatic diseases, including psoriatic arthritis (PsA) and non-radiographic axial spondyloarthritis (nr-axSpA) for Taltz and rheumatoid arthritis (RA) and the investigational use in systemic lupus erythematosus (SLE) for Olumiant.
ChemoCentryx presented highlights from the company’s Phase III ADVOCATE trial of avacopan in ANCA-associated vasculitis. Avacopan is a first-in-class, orally-administered molecule that employs a novel, highly targeted mode of action to treat ANCA-associated vasculitis and other complement-driven autoimmune and inflammatory diseases.
Aimmune Therapeutics presented long-term data from ARC004, an open-label, rollover trial of its pivotal PALISADE trial of Palforzia Allergen Powder and data on patient-reported treatment satisfaction from the European Phase III ARTIMIST trial of the drug. Palforzia is an oral immunotherapy to mitigate allergic reactions to peanuts.
PureTech Health plans to advance its program into Phase III of Follica, to treat androgenetic alopecia, epithelial ageing and other related conditions. The Phase III is for hair loss in male androgenetic alopecia patients.
Biogen presented positive data from the 16-week cutaneous lupus erythematosus (CLE) part of the Phase II LILAC trial. The study looked at BIIB059, a fully humanized IgG1 monoclonal antibody against blood dendritic cell antigen 2 (BDCA2) expressed on plasmacytoid dendritic cell (pDCs). The CLE part of the study met its primary endpoint, showing a dose response on the percentage change from baseline in the Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity (CLASI-A) score at Week 16.
VBL Therapeutics announced new data from its anti-MOSPD2 antibodies for rheumatoid arthritis (RA). They were presented at the European League Against Rheumatism (EULAR) 2020 E-Congress. The results were encouraging, with a clear pattern across multiple indications that they are beneficial in chronic inflammatory diseases.
Hutchison China MediTech (Chi-Med) announced the Independent Data Monitoring Committee (IDMC) of the FRUTIGA trial had completed a planned interim data review and recommended the trial continue. The Phase III trial in China is evaluating fruquintinib in advanced gastric adenocarcinoma or gastroesophageal junction adenocarcinoma who have progressed after first-line standard therapy. Fruquintinib is a highly selective and potent oral inhibitor of vascular endothelial factor receptor (VEGFR) 1/2/3.
Arcturus Therapeutics dosed the first healthy volunteer in its Phase I trial of ARCT-810. ARCT-810 is a mRNA-based candidate for Ornithine Transcarbamylase (OTC) deficiency. ARCT-810 leverages the company’s LUNAR lipid-mediated delivery platform to deliver OTC messenger RNA to liver cells. OTC is a serious, urea cycle disorder.