Clinical Catch-Up: June 21-25

It was a busy week for clinical trial news. Here’s a look.

COVID-19-Related

Gilead Sciences announced positive data from three retrospectives studies of the real-world treatment of hospitalized COVID-19 patients treated with Veklury (remdesivir). They found that in the overall patient populations, patients receiving Veklury had significantly lower risk for mortality compared with matched controls, and this reduction was seen across a spectrum of baseline oxygen requirements. Two of the studies also showed patients receiving Veklury were more likely to be discharged from the hospital by Day 28.

RedHill Biopharma presented positive Phase II safety and efficacy data for Yeliva (oral opaganib) in hospitalized patients with COVID-19 pneumonia. Fifty percent of patients receiving opaganib reached room air by Day 14 compared to 22% in the placebo group. Median time to discharge in the opaganib group was six days compared to 7.5 for the placebo group. Opaganib is a first-in-class, oral spinghosine kinase-2 (SK2) selective inhibitor with dual anti-inflammatory and antiviral activity.

CytoDyn announced preliminary results from unblinding the data from its COVID-19 long-haulers clinical trial. The trial is studying the safety and efficacy of leronlimab as a weekly subcutaneous injection in patients with prolonged symptoms greater than 12 weeks of COVID-19. The primary outcome was change from baseline in daily COVID-19-related symptom severity scored through Day 56.

Ashvattha Therapeutics announced positive interim results from its ongoing Phase II PRANA trial of the HDT-OP-101 to treat hyperinflammation in hospitalized adults with severe COVID-19. A single dose of the drug in the first stage provided a significant and sustained decrease in pro-inflammatory biomarkers until discharge or Day 30. It was more effective in reducing inflammation that steroids alone.

Galecto announced preliminary topline data from an investigator-initiated trial of its galectin-3 inhibitor, GB0139. The trial included 41 hospitalized COVID-19 patients who did not require mechanical ventilation. Twenty were randomized to the GB0139 arm. It demonstrated a favorable safety profile with no treatment-related serious adverse events.

Histogen and Amerimmune announced top-line data from their Phase I trial of emricasan in mild symptomatic COVID-19 patients. The study showed it was safe and well tolerated during the 14 days of dosing and at the 45-day follow-up, compared to placebo. Emricasan is a possible first-in-class, oral, pan-caspace inhibitor that reduces the activity of enzymes that mediate inflammation and apoptosis.

Non-COVID-19-Related

Oyster Point Pharma enrolled the first volunteer in the OLYMPIA Phase II trial of OC-01 (varenicline) nasal spray for Stage 1 Neurotrophic Keratopathy (NK). OC-01 is a highly selective cholinergic agonist being developed for dry eye disease and neurotrophic keratopathy.

Anavex Life Sciences Corp. reported predictive biomarker of response established with SIGMAR1 mRNA expression correlates with responses in primary clinical efficacy endpoints from its Phase II trial of ANAVEX®2-73 (blarcamesine) in adult female patients with Rett syndrome. ANAVEX®2-73 activates the sigma-1 receptor (SIGMAR1) and appears to restore complete housekeeping function within the body and is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.

Albireo Pharma presented nine abstracts at the EASL International Liver Congress 2021 with highlights from the Phase III PEDFIC studies of Bylvay in progressive familial intrahepatic cholestasis (PFIC). Bylvay is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) under development for PFIC, biliary atresia, and Alagille syndrome.

Aligos Therapeutics presented preliminary data in healthy volunteers from the ongoing Phase Ia/b multi-part umbrella clinical trial of ALG-010133. The drug is an S-antigen transport-inhibiting oligonucleotide polymers (STOPS) being developed for hepatitis B.

CymaBay Therapeutics announced positive data from its previously completed Phase II trial and the ENHANCE Phase III study of seladelpar in primary biliary cholangitis (PBC). They are reporting on the results of a pooled analysis of a subset of 39 patients with compensated cirrhosis from the Phase II open-label study and placebo-controlled Phase III study. Seldelpar is a potent, selective, orally active PPARδ agonist.

Spruce Biosciences shared data from two Phase II trials of tildacerfont, SPR001-201, a Phase IIa dose-escalation study and SPR001-202, a Phase IIa, dose focusing on a 400mg dose on multiple biomarkers. The drug is being developed to treat adults with classic Congenital Adrenal Hyperplasia (CAH).

Novartis disclosed new data demonstrating mean IgG and IgM levels remain unchanged in adults with relapsing multiple sclerosis receiving Kesimpta (ofatumumab) over 3.5 years. This is part of the ongoing open label extension of the ALITHIOS trial, which includes 1,703 people with MS taking the drug for up to five years.

Emalex Biosciences completed enrollment of its Phase IIb trial of ecopipam for pediatric patients with Tourette Syndrome. Ecopipam is a first-in-class drug that selectively blocks the actions of dopamine in the D1 receptor.

AIM ImmunoTech announced that all volunteers had completed treatment in its Phase I trial of Ampligen as a potential intranasal therapy as a broad-spectrum respiratory virus prophylaxis. There were no severe adverse events at any dose levels. Ampligen targets and amplifies the natural immune system pathways that fight viruses, and has the potential to prevent infection after exposure to respiratory viruses such as influenza, and potentially common coronaviruses and rhinoviruses like ones that cause the common cold.

Celon Pharma filed an IND for a Phase II trial of CPL’280, its second-generation GPR40 agonist in type 2 diabetes. They are planning for the trial to include 80 patients and hopes to dose the first patient in the fourth quarter of this year with top-line results in the first half of 2022.

Caladrius Biosciences reported the FDA had authorized its IND for a Phase I/II study of CLBS201 for diabetic kidney disease. The drug is a CD34+ cell therapy. The company plans to launch the trial in the next several months.

Sanofi and Translate Bio initiated a Phase I trial of its mRNA vaccine against seasonal influenza. The monovalent flu vaccine candidate codes for the hemagglutinin protein of the A/H3N2 strain of the influenza virus.

TG Therapeutics presented data from the ULTIMATE I and II Phase III trials of ublituximab for relapsing forms of multiple sclerosis. Ublituximab is compared to teriflunomide in the studies and are expected to support a BLA submission in the third quarter of this year. Ublituximab is a novel, glycoengineered anti-CD20 monoclonal antibody.

Antios Therapeutics announced data from a Phase Ib trial of ATI-2173 in chronic Hepatitis B virus-infected adults. The drug is an Active Site Polymerase Inhibitor Nucleotide (ASPIN). The drug was generally well tolerated and led to off-treatment sustained viral suppression in chronic HBV patients as monotherapy.

Brii Biosciences and VBI Vaccines announced final data from a Phase Ib/IIa trial on BRII-179 in chronic hepatitis B virus infection. BRII-179 is a novel recombinant, protein-based immunotherapeutic candidate. The data showed the vaccine induced both B-cell and T-cell responses, was well-tolerated with no safety signals observed.

Revolo Biotherapeutics received the FDA greenlight to launch a Phase II trial of ‘1104 for eosinophilic esophagitis (EoE). ‘1104 is a first-in-class peptide for allergic diseases.

Clovis Oncology opened its first clinical site for the Phase I/II LuMIERE trial of FAP-2286. FAP-2286 is its novel peptide-targeted radionuclide therapy and imaging agent targeting fibroblast activation protein (FAP). The site is at the O’Neal Comprehensive Cancer Center at The University of Alabama at Birmingham.

Karuna Therapeutics announced results from its Phase Ib study of KarXT in healthy elderly volunteers. The drug, xanomeline-trospium, is being developed for dementia-related psychosis. The company expects to initiate a Phase II trial in the first half of 2022.

Hansa Biopharma announced the study design for a trial of imlifidase as a potential desensitization therapy to enable kidney transplants in highly sensitized patients waiting for a compatible decreased donor kidney. In the study, 64 highly sensitized kidney patients with a cPRA score of greater than or equal to 99.9% will be enrolled.

Abivax announced excellent top-line data from the induction phase of its Phase IIa trial of ABX464 in combination with methotrexate for active moderate to severe rheumatoid arthritis. ABX464 is an oral drug candidate with a novel mechanism of action based on the upregulation of a single microRNA (miR-124) with potent anti-inflammatory properties.

Applied Genetic Technologies Corporation (AGTC) reported 12-month data from its ongoing achromatopsia (ACHM) Phase I/II trials, including data from all adults and low-dose pediatric patients. The ACHM B3 candidate demonstrated biologic activity with improvements in visual sensitivity in the treated area. The ACHM A3 therapy will require additional pediatric patient data and preclinical studies.

Fulcrum Therapeutics released data from its Phase IIb trial of losmapimod for facioscapulohumeral muscular dystrophy (FSHD). It appeared to slow progress of the disease and demonstrated improved function in patients. Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor. Nonetheless, the drug failed to meet the endpoints of a reduction in DUX4-driven gene expression due to variability of DUX4 expression in patients.

Transgene enrolled the first patient in its Phase II trial of the combination of TG4001 with avelumab versus avelumab alone in patients with HPV16-positive anogenital tumors. TG4001 is a therapeutic vaccine targeting HPV-positive tumors and is engineered to express HPV16 E6 and E7 antigens and interleukin 2 (IL-2).

PTC Therapeutics presented pooled placebo data from multiple studies that confirmed the clinical benefit of Emflaza (deflazacort) over prednisone for Duchenne muscular dystrophy (DMD). The data showed that the patients receiving the drug performed better on key measures of physical function including the six-minute-walk test, key timed function tests, and the North Star Ambulatory Assessment than patients taking daily prednisone.

Gilead Sciences announced interim results from the Phase IIb and III clinical trials of Hepcludex (bulevirtide) for chronic hepatitis delta virus (HDV). The Phase III trial data supported the safety and efficacy of bulevirtide 2 mg once a day. Data from the Phase IIb trial show that treatment with bulevirtide alone or in combination with peginterferon alfa-2a is associated with a significant HDV RNA decline and improvements in biochemical disease activity at Week 24.

Kura Oncology dosed the first patient in the Phase Ib portion of the KOMET-001 Phase I/II trial of KO-539 in r/r acute myeloid leukemia. KO-539 is an oral, potent and selective menin inhibitor.

BioVie enrolled the first patient in its Phase IIb trial of BIV201 (continuous infusion terlipressin) for refractory ascites. Ascites is a common complication of advanced liver cirrhosis involving the accumulation of large volumes of fluid in the abdomen due to liver and kidney dysfunction.

Brickell Biotech announced that its development partner, Kaken Pharmaceuticals, initiated a Phase I study of sofpironium bromide gel in patients with primary palmoplantar hyperhidrosis (PPH) in Japan. PPH is excessive sweating from the palms and soles of the feet in both adults and children.

Aerami Therapeutics initiated dosing in the Phase I trial of its drug-device combination, AER-901, an inhaled imatinib for the treatment of pulmonary arterial hypertension (PAH). Oral imatinib is a tyrosine kinase inhibitor used to treat certain types of cancers, and has been previously investigated in a Phase III IMPRES as an oral formulation for PAH.

Novartis published a study showing that ¹⁷⁷Lu-PSMA-617 plus standard of care significantly improved overall survival and imaging-based progression-free survival in patients with metastatic castration-resistant prostate cancer (mCRPC) compared to standard of care alone. ¹⁷⁷Lu-PSMA-617 is a PSMA-targeted radioligand therapy that combines a targeting compound with a therapeutic radioisotope.

Arcus Biosciences announced that, at the first interim analysis of the three-arm Phase II ARC-7 study, both arms with domvanalimab-based combinations showed encouraging clinical activity measured by ORR when given as an initial treatment to people with metastatic, PD-L1≥50% non-small cell lung cancer (NSCLC). Domvanalimab is an anti-TIGIT candidate.

Orphalan announced positive topline results from a Phase III head-to-head trial comparing d-Penicillamine with trientine tetrahydrochloride in Wilson’s Disease (WD). WD is a rare inherited disorder of copper transport that primarily affects the liver and brain, which if untreated can be fatal. The trial hit the primary efficacy endpoint, demonstrating that trientine tetrahydrochloride was non-inferior to d-Penicillamine.

Vir Biotechnology announced new data from its ongoing Phase II trial of VIR-2218 and ongoing Phase I studies of VIR-3434 in patients with chronic hepatitis B virus (HBV). The results demonstrate positive safety plus a reduction in hepatitis B surface antigen for both compounds.