Clinical Catch-Up: January 4-8

Doctor and Patient

The new year began with a fairly low level of clinical trial news. Here’s a look.

COVID-19-Related

Arcturus Therapeutics got the FDA go-ahead for its Phase II trial of its COVID-19 vaccine candidate ARCT-021. The trial will enroll 600 participants, with 450 receiving ARCT-021 and 150 receiving placebo.

Cerecor announced results from its exploratory Phase II U.S.-based trial of CERC-002 in hospitalized COVID-19 patients with associated pneumonia and mild-to-moderate ARDS. CERC-002 is a human anti-LIGHT monoclonal antibody. The trial demonstrated strong improvement in the proportion of patients alive and free of respiratory failure over the 28-day study period.

China’s Sinovac Biotech reported that its COVID-19 vaccine, CoronaVac, had a 78% efficacy rate in a Phase III clinical trial in Brazil. It also completely prevented severe cases. Turkish researchers reported interim data on the same vaccine in December showing 91.25% efficacy.

Non-COVID-19-Related

Calithera Biosciences reported that its CANTATA clinical trial of telaglenastat in patients with advanced or metastatic renal cell carcinoma (RCC) failed to hit the primary endpoint. Telaglenastat is a glutaminase inhibitor. In the study, telaglenastat in combination with cabozantinib was compared to placebo with cabozantinib in patients with advanced or metastatic RCC who had been treated with one or two previous lines of systemic therapy, including at least one vascular endothelial growth factor (VEGF)-pathway targeted therapy or the combination of Bristol Myers Squibb’s Opdivo (nivolumab) and Yervoy (ipilimumab). The primary endpoint was progression-free survival (PFS) by blinded independent review.

Ayala Pharmaceuticals completed its end-of-Phase I meeting with the FDA on AL102 for desmoid tumors. They got the go-ahead to initiate a Phase II/III pivotal trial. AL102 is a potent, selective, oral gamma secretase inhibitor (GSI).

Forge Therapeutics received FDA clearance to initiate a Phase I/II trial of its FBX-101 for patients with Krabbe disease. FBX-101 is a first-in-human AAV gene therapy. Krabbe disease is a rare and fatal pediatric leukodystrophy caused by mutations in the galactosylceramidase (GALC) gene.

Harpoon Therapeutics dosed the first patient with HPN328 in its Phase I/II trial of small cell lung cancer (SCLC) and other tumors associated with DLL3 expression. HPN328 is a delta like ligand 3- (DLL3) targeting TriTAC.

Biohaven Pharmaceutical initiated enrollment in its Phase III trial of troriluzole in obsessive-compulsive disorder (OCD). A new chemical entity, troriluzole is a third-generation glutamate modulating agent that normalizes glutamate, a key neurotransmitter associated with OCD.

Inozyme Pharma was cleared by the FDA and the UK’s MHRA for a Phase I/II trial of INZ-701 in adults with ENPP1 deficiency. INZ-701 is a soluble, recombinant protein containing the extracellular domain of native human ENPP1 fused to the Fc domain of the immunoglobulin IgG1. ENPP1 deficiency is a rare, chronic, systemic, and progressive disease.

Ilya Pharma completed recruitment in its Phase I trial of ILP100-Topical for wound treatment. ILP100 is a genetically engineered Lactobacillus that acts as a small bioreactor on site in the wound, continuously introducing a chemokine, CXCL12, into the wound tissue.

Precigen received the okay from the FDA to initiate a Phase I trial of PRGN-2012 in recurrent respiratory papillomatosis (RRP). PRGN-2012 is a first-in-class, off-the-shelf AdenoVerse immunotherapy using the company’s gorilla adenovector technology, part of its proprietary AdenoVerse platform.

BioXcel Therapeutics announced that its dexmedetomidine hit the primary and secondary endpoints of the TRANQUILITY Phase Ib/II trial for agitation in dementia. BXCL501 is the company’s proprietary, orally dissolving thin film formulation of dexmedetomidine. The primary endpoints focused on safety and tolerability; the secondary efficacy endpoints were compared to placebo and all were met for the three primary agitation scale: the Positive and Negative Syndrome Scale-Excitatory Component, the Pittsburgh Agitation Scale, and the Modified Cohen-Mansfield Agitation Inventory.

Ocuphire Pharma initiated patient recruitment and screening of its LYNX-1 Phase III trial of Nyxol in night vision disturbances (NVD). NVD is also called dim light vision disturbances and is when peripheral imperfections of the cornea scatter light when the pupil dilates in dim light. Nyxol is a once-daily preservative-free eye drop formulation of phentolamine mesylate, a non-selective alpha-1 and alpha-2 adrenergic antagonist.

Alnylam Pharmaceuticals reported positive topline data from its Phase III trial of vutrisiran in hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) amyloidosis with polyneuropathy. The drug is an RNA interference (RNAi) therapeutic. The trial met primary and both secondary endpoints at nine months. The primary endpoint was change from baseline in the modified Neuropathy Impairment Score at nine months compared to historical placebo data from the APOLLO Phase III trial of patisiran.

Theratechnologies received the go-ahead from the FDA to launch its Phase III trial for tesamorelin for adults with nonalcoholic steatohepatitis (NASH) with liver fibrosis. It expects to initiate the trial by the third quarter of 2021. The company also received an ok to launch a Phase I trial of TH1902, its lead peptide-drug conjugate.

Sarepta Therapeutics announced topline results from Part 1 of Study SRP-9001-102 for its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD). The study hit the mark on its primary biological endpoint of micro-dystrophin protein expression. However, on the primary functional endpoint, meaning how well the patients actually responded, there was an increase in NSAA total score compared to placebo at 48 weeks, but it was not statistically significant. NSAA is a 17-point rating scale that measures functional motor abilities in ambulant children with DMD.

Biogen dosed the first patient in its Phase IV RESPOND trial to evaluate Spinraza (nusinersen) in infants and children with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with gene therapy Zolgensma (onasemnogene abeparvovec). It will be conducted at about 20 sites globally and enroll up to 60 children with SMA.

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