Clinical Catch-Up: Amgen, AstraZeneca, Avillion Attack Asthma
Heading out of the month of May, plenty of companies presented or announced clinical trial updates and news. Here’s a look.
Synairgen announced the Phase III Sprinter trial failed to hit the primary endpoint of patient recovery and subsequent hospital discharge. The study looked at SNG001 in patients hospitalized with COVID-19 and required supplemental oxygen treatment.
Bristol Myers Squibb reported the Phase III trial of Opdivo (nivolumab) and Yervoy (ipilimumab) in patients with metastatic urothelial carcinoma failed to hit the predetermined primary endpoint of overall survival. The drugs are anti-PD-L1 and CTLA-4 checkpoint inhibitors, respectively.
POINT Biopharma Global announced the Phase I trial details of [Lu-177]-PNT6555 for patients with select solid tumors. The study is expected to start in the summer of 2022 in Canada and will use a gallium-68-based pNT6555 molecular imaging agent to select patients to receive a no-carrier-added lutetium-177-based PNT6555 therapeutic agent.
C4 Therapeutics dosed the first patient in its Phase I/II trial of CFT8634 for SMARCB1-perturbed cancers, including synovial sarcoma and SMARCB1-null tumors. CFT8634 is an orally bioavailable BiDAC degrader targeting BRD9.
AstraZeneca and Avillion published full results from the positive Mandala Phase III trial of PT027 (albuterol/budesonide) as an as-needed rescue medicine for moderate to severe asthma. The drug is a potential first-in-class inhaled, fixed-dose combination rescue medication containing albuterol, a short-acting beta2-agonist (SABA), and budesonide. It demonstrated a statistically significant reduction in the risk of a severe exacerbation versus albuterol rescue.
Neoleukin dosed the first patient in a combination arm of NL-201 in combination with Merck’s anti-PD-1 checkpoint inhibitor Keytruda (pembrolizumab) for its Phase I trial in r/r solid tumors. NL-201 is a de novo agonist of the IL-2 and IL-15 receptors.
Inhibrx announced topline results from a Phase I trial of INBRX-101 in alpha-1 antitrypsin deficiency. The drug is an optimized recombinant human AAT-Fc fusion protein. It showed a favorable safety and tolerability profile with no drug-related severe adverse events.
CG Oncology announced interim results from the Phase II CORE1 trial of CG0070 in combination with Merck’s Keytruda for Non-Muscle-Invasive Bladder Cancer (NMIBC) unresponsive to Bacillus Calmette-Guerin (BCG). CG0070 is a selective oncolytic immunotherapy based on a modified adenovirus type 5 backbone that contains a cancer-selective promoter and a GM-CSF transgene.
Jasper Therapeutics dosed the first patient in its sponsored Phase I/II trial of JSP191 for Fanconi Anemia patients in bone marrow failure requiring allogeneic transplant with non-sibling donors. JSP191 is a humanized monoclonal antibody as a conditioning agent that blocks stem cell factor receptor signaling, leading to the clearance of hematopoietic stem cells from bone marrow.
Nouscom announced that Janssen Research & Development, a Johnson & Johnson company, received clearance from the FDA for a study of VAC85135. The therapy is an off-the-shelf, viral vector-based vaccine for an oncologic indication. It has been licensed to Janssen.
Amgen and AstraZeneca presented data from the Phase III Navigator trial of tezepelumab in asthma. The data showed almost half of those enrolled achieved complete response for exacerbation reduction, asthma control, lung function, and clinician assessment.
Aerovate Therapeutics presented Phase I data of AV-101. AV-101 is a novel dry powder inhaled formulation of imatinib being developed for pulmonary arterial hypertension (PAH). The drug was generally well-tolerated with no serious adverse events.
Urovant Sciences announced new analyses from the Phase III EMPOWUR Extension Study of Gemtesa. In a subgroup analysis of adults 65 years or older with overactive bladder, Gemtesa was safe, well-tolerated and associated with sustained reductions in average daily micturition, urge urinary incontinence episodes, and urgency episodes.
Idera Pharmaceuticals halted patient enrollment early for its Phase II trial of tilsotolimod in patients with pT3-4 cN0M0 melanoma after interim data delivered positive results. They demonstrated a 70% drop in SLN-positivity rates compared to placebo. The drug is a synthetic toll-like receptor 9 agonist.
Kibow Pharmaceuticals announced the first patient completed treatment in its Phase IIb trial of US-APR2020 for chronic kidney disease. The therapy is a novel live biotherapeutic product designed to restore the gut microbiome by metabolizing and removing several uremic toxins in the bowel.
ANANDA Scientific and the University of Nebraska Medical Center received the green light from the FDA for a study of Nantheia ATL5 for treatment for post-traumatic stress disorder (PTSD). The drug uses cannabidiol using ANANDA’s proprietary delivery technology.
HUYABIO International completed the Phase I drug-drug interaction study of HBI-3000 for cardioversion of atrial fibrillation. HBI-3000 is a multi-ion channel blocker.
Entasis Therapeutics presented data from its pivotal Phase III ATTACK trial of sulbactam-durlobactam for infections caused by Acinetobacter baumannii, including carbapenem-resistant and multi-drug resistant strains. The drug was associated with lower mortality.
Sorrento Therapeutics received the go-ahead from the FDA for the Phase II MAVERICK study of abivertinib (Fujovee) for metastatic castrate-resistant prostate cancer. Abivertinib is a novel small molecule tyrosine kinase inhibitor (TKI) that selectively targets both mutant forms of EGFR as well as BTK.
Anji Pharma completed enrollment in its Phase II study of pradigastat in patients with functional constipation. The drug is a diacylglycerol acyltransferase 1 (DGAT1) inhibitor.
Qlaris Bio announced results from its Phase II trial of QLS-101 for glaucoma and reported favorable safety, tolerability and positive efficacy. QLS-101 is a novel adenosine triphosphate (ATP)-sensitive potassium channel modulator.
Beyond Air presented positive new interim data from the ongoing LungFit GO pilot at-home study in Australia of inhaled nitric oxide (NO) to treat severe nontuberculous mycobacterial lung disease. The data showed that high concentration inhaled NO was well tolerated after 2,323 inhalations with no treatment-related discontinuations.
4D Pharma presented data from Part A of the Phase I/II trial of MRx-4DP0004 for asthma. The data reinforced the success of using the MicroRx platform to select single strain LBPs and generate clinical candidates.
Axsome Therapeutics published promising data from its Phase II Ascend trial of AXS-05 for major depressive disorder. AXS-05 is an NMDA receptor antagonist. The drug uses a proprietary formulation and dose of dextromethorphan and bupropion. The study demonstrated rapid, substantial and statistically significant improvement in depressive symptoms and induction of remission.
Immatics dosed the first patient in the Phase Ib trial of IMA203 and Bristol Myers Squibb’s Opdivo (nivolumab) in advanced solid tumors. ACTengine IMA203 T cells are directed against an HLA-A*02-presented peptide derived from preferentially expressed antigen in melanoma.
Veloxis Pharmaceuticals dosed the first patient in a Phase I trial of VEL-101 for the prevention of acute rejection in kidney transplant recipients. VEL-101 is a novel maintenance immunosuppressive agent.
Purple Biotech initiated a Phase II trial of CM24 for multiple cancers. The drug is a first-in-class monoclonal antibody. It will be tested in combination with BMS’s Opdivo and chemotherapy in metastatic pancreatic cancer.
Adaptive Phage Therapeutics dosed the first patient in its Phase I/II Dance trial of its precision bacteriophage study in diabetic foot osteomyelitis. Phages (viruses that infect bacteria) are selected from the company’s proprietary phage bank using their phage susceptibility test.
IO Biotech dosed the first patient in its Phase III trial of IO102-IO103 in combination with Merck’s Keytruda (pembrolizumab) in previously untreated, unresectable, or metastatic melanoma. The drug is an investigational cancer immunotherapy targeting IDO and PD-L1.
Avalo Therapeutics dosed the first patient in its Phase II Peak trial of AVTX-002 for poorly controlled non-eosinophilic asthma. The primary endpoint is the proportion of subjects who experience an asthma-related event.
Genascence Corporation presented positive safety data from its Phase I study of GNSC-001 for osteoarthritis. The drug was well-tolerated. GNSC-001 is a recombinant AAV that carries a coding sequence for interleukin-1 receptor antagonist, which can inhibit IL-1 signaling.
XyloCor Therapeutics reported promising Phase I data from the ongoing Phase I/II Exact trial of XC001 (encoberminogene rezmadenovec) for refractory angina. The drug showed positive results across all doses tested and positive trends in ischemic burden and symptom reduction.
Enanta Pharmaceuticals reported its Phase IIb trial of EDP-938 failed to hit the primary endpoint for community-acquired respiratory syncytial virus (RSV) infection. The primary endpoint was the total symptom score area under the curve from day one to 14. EDP-938 is an N-protein inhibitor.
Mustang Bio announced interim data from the Phase I/II trial with the same lentiviral vector used in its MB-107 for X-linked severe combined immunodeficiency. The company says this data supports plans to initiate a pivotal Phase II trial for MB-107 in the second half of this year.
Mind Medicine reported topline results from the Phase I trial of MM-110. The drug is being developed for the treatment of opioid withdrawal.
Ocuphire Pharma announced positive topline results from the LYNX-1 Phase III trial of Nyxol for night (or dim light) vision disturbances. The study hit the primary endpoint with Nyxol subjects gaining 3 lines of low contrast distance vision under dim light conditions compared to placebo.
Indaptus Therapeutics received the green light from the FDA for a Phase I trial of Decoy20 in solid tumors. Decoy20 is an antigen-agnostic technology.
Aileron Therapeutics treated the first patients in its Phase Ib trial of ALRN-6924 to protect against chemotherapy-induced neutropenia and other bone marrow toxicities in patients with p53-mutated breast cancer being treated with doxorubicin plus cyclophosphamide and docetaxel.
Athersys announced that its partner, Healios, reported topline results for its Japan ischemic stroke study, Treasure. The study evaluated MultiStem cells (invimestrocel). Improvements were observed on pre-specified measures of functional independence and good outcomes, but the primary endpoint, Excellent Outcome at 90 days, did not reach statistical significance. MultiStem is an off-the-shelf stem cell product that promotes tissue repair and healing.