Clinical Catch-Up: May 24-28
Heading into the U.S. Memorial Day holiday weekend and the official start of summer, it was a moderately busy week for clinical trial news. Take a look.
Organicell Regenerative Medicine presented case reports at the International Society of Cell and Gene Therapy (ISCT) on its Zofin in three severely ill COVID-19 patients. The data showed that the drug is associated with decreased levels of inflammatory biomarkers, such as CRP and IL6. Zofin is an acellular biologic therapy originating from perinatal sources and manufactured to keep naturally occurring microRNAs. It contains over 300 growth factors, cytokines, and chemokines.
Axcella presented positive data from its AXA1665-002 Phase I clinical trial of AXA1665 in 60 patients with mild and moderate hepatic insufficiency. Both doses of the drug demonstrated safety and were well tolerated. It is now initiating a 24-week Phase II trial. AXA1665 is a composition of eight amino acids and derivatives designed to target multiple metabolic pathways.
NGM Biopharmaceuticals’ aldafermin failed to hit its primary endpoint in the company’s Phase IIb ALPINE 2/3 trial for non-alcoholic steatohepatitis (NASH) with stage 2 or 3 liver fibrosis. The trial failed to hit the primary endpoint of a dose response showing improvement in liver fibrosis by greater than or equal to 1 stage with no worsening of NASH at week 24. It did achieve statistical significance compared to placebo on some secondary endpoints, including NASH resolution at the 3 mg dose and multiple non-invasive measures of NASH, including liver fat content reduction at the 1 mg and 3 mg doses. Aldafermin is an engineered analog of the human hormone FGF19.
Takeda presented long-term safety and efficacy data from its ongoing pivotal Phase III Tetravalent Immunization against Dengue Efficacy Study (TIDES) trial of TAK-003, its dengue vaccine candidate. The 36-month data demonstrated continued protection against dengue-caused hospitalization.
RedHill Biopharma presented two new analyses of Movantik Phase III trial data that demonstrated rapid onset of action and sustained and predictable improvement of key symptoms associated with opioid-induced constipation. The two studies, Kodiak 4 and 5, are identical. Movantik (naloxegol) is an opioid antagonist.
Sarepta Therapeutics announced positive 12-week expression and safety data from the first 11 patients in its Study SRP-9001-103 ENDEAVOR trial run in collaboration with Roche. SRP-9001 is an investigational gene transfer therapy designed to deliver its micro-dystrophin-encoding gene to muscle tissue for the treatment of Duchenne Muscular Dystrophy (DMD). The data showed robust expression of micro-dystrophin and no new safety signals.
GenSight Biologics published a case study of partial recovery of vision in a blind patient with late-stage retinitis pigmentosa (RP). The patient is part of the company’s ongoing PIONEER Phase I/II trial of GS030 optogenetic therapy. The research was published in Nature Medicine. RP is a leading cause of inherited blindness, caused by mutations in more than 71 different genes. GS030 uses the company’s optogenetics technology platform, delivering a gene encoding for a light-sensitive protein (ChrimsonR-tdT) into retinal ganglion cells, which makes them responsive to light and bypasses photoreceptors that were killed by the disease. A wearable medical device is needed to stimulate the treated retina.
Ultragenyx announced positive longer-term data from the Glycogen Storage Disease Type Ia (GSDIa) and Ornithine Transcarbamylase (OTC) Deficiency Phase I/II trials. The data demonstrated ongoing durability of response in addition to advancements in the company’s HeLa producer cell line (PCL) manufacturing platform.
AbbVie’s Skyrizi (risankizumab) hit the co-primary endpoints of clinical remission and endoscopic response at week 12 compared to placebo in two Phase III studies in Crohn’s disease. The company also published data from the pivotal global Phase III trials, Measure Up 1, Measure UP 2 and AD Up of Rinvoq (upadacitinib) in adults and adolescents with moderate to severe atopic dermatitis who were candidates for systemic treatment.
EyePoint Pharmaceuticals completed enrollment in its Phase I trial of EYP-1901 for wet age-related macular degeneration. EYP-1901 is a possible twice-yearly sustained delivery intravitreal anti-VEGF treatment.
Emergent BioSolutions announced positive two-year data on its chikungunya virus virus-like particle (CHIKV VLP) vaccine candidate. The vaccine demonstrated an increase in immune response as measured by anti-chikungunya virus serum neutralizing antibodies (SNA), and the mean titers were 19 times higher than pre-vaccination titers.
Jasper Therapeutics initiated a Phase I/II trial of JSP191 as a targeted, non-toxic conditioning regimen before allogeneic transplant for chronic granulomatous disease (CGD). JSP191 is a first-in-class anti-CD117 monoclonal antibody. CGD is a rare, inherited disease of the immune system that develops in infancy or early childhood.
AmMax Bio enrolled the first patient in its Phase II trial of AMB-05X for tenosynovial giant cell tumors (TGCT). AMB-05X inhibits the CSF1R pathway and is believed capable of stunting tumor growth. TGCT is an aggressive tumor of the synovium primarily in the knee, hip, and ankle joints.
Sinovant Sciences and Nabriva Therapeutics announced positive topline data from their Phase III trial of Lefamulin in Chinese adults with community acquired bacterial pneumonia (CABP). The trial hit the primary endpoint of non-inferiority versus moxifloxacin. Lefamulin, marked in the U.S. as Xenleta, is a first-in-class, semi-synthetic pleuromutilin antibiotic for systemic administration.
OSE Immunotherapeutics and the FoRT Foundation announced the Italian Medicines Agency (AIFA) and the Italian Ethics Committee approved the launch of a new Phase II trial of Tedopi in combination with Bristol Myers Squibb’s Opdivo for second-line treatment of HLA-A2 positive patients with metastatic NSCLC after first-line chemo-immunotherapy. Tedopi is a neo-epitope-based vaccine.
BioInvent International and Transgene received a greenlight from the FDA for U.S. patients to enroll in their ongoing Phase I/IIa trial of BT-001. BT-001 is a novel oncolytic virus armed with a differentiated Treg-depleting human recombinant anti-CTLA4 antibody and GM-CSF. It will be evaluated alone and in combination with Merck’s Keytruda (pembrolizumab), an anti-PD-1 checkpoint inhibitor.
Dicerna and Eli Lilly had their IND approved for LY3819469 for a Phase I trial in cardiometabolic diseases. The drug is an RNAi silencing compound that targets the LPA gene. Dicerna and Boehringer Ingelheim also received the go-ahead from the FDA for a Phase I trial for DCR-LIV2, an RNAi silencing drug for NASH.
Chiasma presented patient-reported outcomes (PROs) data from its MPOWERED Phase III trial showing that patients with acromegaly reported significantly improved quality of life and work productivity after transitioning from long-acting injectable somatostatin analogs (iSSAs) to Mycapssa. Mycapssa (oral octreotide capsules) is a somatostatin analog indicated for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide.
Zai Lab dosed the first patient in China in the Phase II portion of TRIDENT-1 of repotrectinib being run by Turning Point Therapeutics. It is being tested in patients with ROS1+ advanced non-small cell lung cancer (NSCLC) and NTRK+ advanced solid tumors. Repotrectinib is a next-generation kinase inhibitor targeting the ROS1 and TRK oncogenic drivers of NSCLC and advanced solid tumors.