Clinical Catch-Up: August 23-27
It was a busy week for clinical trial news. Here’s a look.
COVID-19-Related
PureTech Health presented data from the Phase I multiple ascending dose and food effect study of LYT-100. LYT-100 is a deuterated form of pirfenidone, an approved anti-inflammatory and anti-fibrotic drug. It is being developed for inflammatory and fibrotic disease, including lung disease and Long COVID, and disorders of lymphatic flow, such as lymphedema.
Tonix Pharmaceuticals initiated a Phase II trial of TNX-102 SL for Long COVID Syndrome. TNX-102 SL is a sublingual form of cyclobenzaprine hydrochloride designed to provide rapid transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine.
Brii Biosciences released early data from its Phase III trial of its monoclonal antibody combination for COVID-19. The combo decreased death and hospitalization in patients at high-risk of disease progression by 78%.
Noxopharm released preliminary topline data from its NOXCOVID Phase I trial of Veyonda (idronoxil) as an anti-inflammatory in patients with moderate COVID-19. The drug is a TBK1 (TANK-binding kinase 1) inhibitor. Based on initial positive results, the company has started discussions to adding the drug to one of the clinical trial programs studying effective anti-inflammatories in COVID-19.
Pardes Biosciences dosed the first patient in its Phase I trial of PBI-0451 for COVID-19. PBI-0451 inhibits Viral Main Protease, a highly conserved protein the virus requires for replication.
EOM Pharmaceuticals dosed the first patients in R1: RESCUE, a proof-of-concept Phase I/IIa open-label multicenter trial in Brazil to evaluate EOM613 in hospitalized COVID-19 patients with severe disease. EOM613 is a novel peptide-nucleic acid solution immunomodulator believed to have anti- and pro-inflammatory broad-spectrum cytokine effects.
Non-COVID-19-Related
Foghorn Therapeutics dosed the first patient in its Phase I trial of FHD-609 for synovial sarcoma. FHD-609 is a potent, selective protein degrader of BRD9.
Novartis reported that its Kymriah (tisagenlecleucel) failed to hit its primary endpoint in the Phase III BELINDA trial in aggressive B-cell non-Hodgkin lymphoma (NHL) after relapse or lack of response to first-line treatment. Kymriah is the company’s CD19-directed CAR-T therapy, which is approved for patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) or adults with relapsed or refractory large B-cell lymphoma. The primary endpoint of the BELINDA trial was event-free survival (EFS) compared to treatment with the standard-of-care (SOC). In this case, SOC was salvage chemotherapy, with patients who responded then were treated with high-dose chemotherapy and stem cell transplant.
Nouscom dosed the first patient in its Phase Ib trial of NOUS-PEV. NOUS-PEV is a personalized neoantigen cancer vaccine. It is being dosed in combination with Merck’s Keytruda (pembrolizumab) in patients with either locally advanced 1L melanoma or 1L non-small cell lung cancer (NSCLC) expressing more than 50% PD-L1.
Rafael Pharmaceuticals completed its Phase Ib trial of CPI-613 (devimistat) in combination with gemcitabine and cisplatin in biliary tract cancer. The trial successfully identified the recommended Phase II dose and randomization of the Phase II part of the trial had begun enrolling. CPI-613 is a first-in-class compound that targets enzymes involved in cancer cell energy metabolism located in the mitochondria of cancer cells.
CytoDyn announced an update to previously reported preliminary data from its Phase Ib/II trials and compassionate use of leronlimab in 30 metastatic triple-negative breast cancer (mTNBC) patients. A decrease in circulating tumor cells (CTC) was reported after induction and was associated with a 400%-660% increase in modified progression-free survival (mPFS)/12 months PFS and a 570%-980% increase in modified Overall Survival (mOS)/12-month OS.
Flexion Therapeutics expanded the Phase Ib trial of FX301 in patients undergoing bunionectomy. FXZ301 is a novel formulation of a locally administered NaV1.7 inhibitor (funapide) with a goal of providing at least three to five days of post-operative pain relief while preserving motor function.
FibroGen announced positive topline results from the WHITNEY Phase II trial of Roxadustat for chemotherapy-induced anemia. The drug is a first-in-class oral small molecule hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI). The primary efficacy endpoint was maximum change in hemoglobin within 16 weeks from baseline without red blood cell transfusion.
Merck announced topline results from the pivotal PNEU-PED study of Vaxneuvance (Pneumococcal 15-valent Conjugate Vaccine) in healthy infants. The infants were given a 4-dose regimen of either Vaxneuvance or the licensed 13-valent pneumococcal conjugate vaccine at 2, 4, 6 and 12-15 months of age. The Vaxneuvance safety profile was comparable to PCV13 and at 30 days after the third dose was non-inferior to PCV13 for all 13 shared serotypes.
Cartesian Therapeutics announced clinical data from the first cohort of the ongoing Phase I/IIa trial of Descartes-08 in generalized Myasthenia Gravis (gMG). The drug is an mRNA-modified, autologous CAR T-cell product against B-Cell Maturation Antigen (BCMA). The drug was well tolerated with no cytokine release syndrome or other serious product-related adverse events.
Hutchmed initiated a Phase Ib/II trial of fruquintinib in advanced triple negative breast cancer or advanced endometrial cancer in the U.S. Fruquintinib is a highly selective and potent oral inhibitor of VEGFR-1, -2 and -3.
Amneal Pharmaceuticals announced positive topline results from the pivotal Phase III RISE-PD trial of IPX-203 in Parkinson’s disease patients who have motor fluctuations. The drug demonstrated statistically significant improvement in efficacy compared to immediate-release CD/LD. IPX-203 is a novel, oral formulation of CD/LD extended-release capsules.
Servier and OSE Immunotherapeutics dosed the first patient in the Phase II trial of OSE-127/S95011 in Sjogren’s syndrome. The drug is a humanized monoclonal antibody targeting the CD127 receptor, the alpha chain of the interleukin-7 receptor, creating a potent antagonistic effect on effector T cells.
Quantum Genomics announced results from the Phase IIb QUORUM study of firibastat in heart failure. The drug was as efficient as ramipril, the current standard of care, in preventing the degradation of the left ventricular ejection fraction after myocardial infarction.
OSE Immunotherapeutics and ARCAGY-GINECO dosed the first patient in the Phase II trial of Tedopi alone and in combination with Merck’s checkpoint inhibitor Keytruda (prembrolizumab) as maintenance treatment in recurrent ovarian cancer after chemotherapy. Tedopi is the company’s innovative combination of neoepitodes, part of its vaccine platform.
Bayer presented results from the Phase III FIGARO-DKD study of Kerendia (finerenone) in adults with chronic kidney disease associated with type 2 diabetes. The drug reduced the risk of cardiovascular outcomes. Finerenone is a first-in-class nonsteroidal mineralocorticoid receptor antagonist.