Clinical Catch-Up: December 28, 2020-January 1, 2021

Clinical Trial Patient_Compressed

It’s not particularly surprising that there wasn’t a lot of clinical trial news between Christmas and New Year’s, although there was some. Here’s a look.

COVID-19-Related

CytoDyn received guidance from the FDA on its open-label extension of its Phase III trial and for the continuation of eINDs for patients receiving Vyrologix (leronlimab-PRO 140) for COVID-19. The company completed its Phase II trial for mild-to-moderate COVID-19 patients in the U.S., which demonstrated statistically significant data and completed enrollment of 390 patients in its Phase IIb/III trial in severe-to-critically ill COVID-19 patients with data to be released mid-January 2021. Vyrologix is a CCR5 antagonist.

Novavax initiated the PREVENT-19 Phase III trial of its COVID-19 vaccine candidate, NVX-CoV2373. It is being run in the U.S. and Mexico. The vaccine contains a full-length, pre-fusion spike protein and the company’s Matrix-M adjuvant.

Capricor Therapeutics reported the independent Data and Safety Monitoring Board (DSMB) completed its safety review of its Phase II INSPIRE study of CAP-1002 for severe COVID-19. The DSMB recommended the trial continue as designed. CAP-1002 is an allogeneic cell therapy in development for Duchenne muscular dystrophy and the cytokine storm associated with COVID-19.

NeuroRx and Relief Therapeutics concluded enrollment in the Phase IIb/III trial of Zyesami (aviptadil) for Respiratory Failure in critical COVID-19 patients. No drug-related serious adverse events have been reported. Topline data are expected in late January or early February 2021. Zyesami is a formulation of vasoactive intestinal polypeptide (VIP).

Arcturus Therapeutics received approval from the Singapore Health Sciences Authority to proceed with its Phase II trial of ARCT-021, a vaccine for COVID-19. Data from the Phase I/II trial was favorable. The trial will study both single dose and prime-boost regimens in up to 600 participants.

PanThera initiated dosing in the Phase III PROVENT trial of AstraZeneca’s antibody combination, AZD7442, for potential prevention of COVID-19. The trial of a single dose inoculation will run for a year and hopes to recruit 5,000 volunteers globally.

Non-COVID-19-Related

TLC announced it had completed patient enrollment of its Phase III EXCELLENCE trial of TLC599 in osteoarthritis (OA) knee pain. TLC599 is a non-opioid, proprietary BioSeizer sustained release formulation of dexamethasone sodium phosphate (DSP) intended to manage OA pain for up to six months. It is evaluating approximately 500 OA patients at 46 sites in the U.S. and Australia.

Audentes Therapeutics announced the FDA had lifted the clinical hold for the ASPIRO trial of AT132 in patients with X-linked myotubular myopathy (XLMTM). XLMTM is a serious, life-threatening neuromuscular disease marked by extreme muscle weakness, respiratory failure, and early death. AT132 is an AAV8 vector containing a functional copy of the MTM1 gene.

Aprea Therapeutics announced its Phase III trial of eprenetapopt and azacytidine for TP53 mutant myelodysplastic syndromes (MDS) failed to meet its primary endpoint. Although the combo treatment demonstrated a higher complete remission rate than azacytidine alone, it was not deemed statistically significant. Eprenetapopt is a small molecule that reactivates the mutant and inactivated p53 protein.

VBL Therapeutics expanded its OVAL Phase III trial of ofranergene obadenovec in platinum-resistant ovarian cancer into Europe. It has enrolled the first patient in the expansion. Ofranergene obadenovec is a first-in-class, targeted anti-cancer gene therapy.

LyGenesis received the go-ahead from the FDA to run a Phase IIa trial of its first-in-class novel cell therapy for end stage liver disease (ESLD). Its lead program is an allogeneic cell therapy program focused on liver regeneration. They expect to treat the first patient in early 2021.

Protalix Biotherapeutics and Chiesi Global Rare Diseases announced final trial results from the BRIDGE Phase III study of pegunigalsidase alfa for Fabry disease. Pegunigalsidase alfa is a plant cell-expressed recombinant, PEGylated, cross-linked alpha-galactosidase-A product. Fabry disease is an X-linked inherited disorder caused by deficient activity of the lysosomal alpha-Galactosidase-A enzyme, which results in abnormal deposits of a fatty substance, globotriaosylceramide, in blood vessel walls. The data demonstrated substantial improvement in renal function.

Hepion Pharmaceuticals announced the DSMB had approved continuation of its Phase IIa AMBITION trial of CRV431 in non-alcoholic steatohepatitis (NASH). CRV431 is a pan-cyclophilin inhibitor. Cyclophilins play a central role in protein folding and contribute to inflammation and fibrosis, as well as other biological effects.

Altimmune enrolled the first patient in its Phase II trial of HepTcell for chronic hepatitis B. HepTcell is a novel peptide-based immunotherapeutic. The trial will run in the U.S., Canada and Europe.

RedHill Pharma announced preliminary data from the Phase II trial of 40 hospitalized COVID-19 patients receiving opaganib. There were consistent trends of greater improvement in reducing oxygen requirement in the opaganib-treated arm. Opaganib is a proprietary, first-in-class, orally administered, sphingosine kinase-2 (SK2) selective inhibitor with dual anti-inflammatory and antiviral activity.

Axsome Therapeutics reported positive data from the long-term, open-label Phase III MOVEMENT trial of AXS-07 for the treatment of acute migraine. The drug is a novel, oral, rapidly absorbed drug with several methods of action. A new molecular entity, it utilizes the company’s Molecular Solubility Enhanced Inclusion Complex (MoSEIC) technology, which allows rapid absorption of meloxicam while maintaining a longer half-life in the blood. The drug is a combination of meloxicam and rizaptriptan. Meloxicam is a COX-2 preferential non-steroidal anti-inflammatory and rizaptriptan is a 5-HT1B/1D agonist.

Lexicon Pharmaceuticals launched patient dosing in RELIEF-PHN 1, its Phase II trial of LX9211 for post-herpetic neuralgia. LX9211 is an oral small molecule inhibitor of adaptor associated kinase 1 (AAK1).

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