Clinical Catch-Up: Prometheus, Algernon, Seelos, Inozyme and Takeda

Quick Catch-Up

• Prometheus Biosciences completed Cohort 1 Phase II trial enrollment for ARTEMIS-UC
• Algernon Pharmaceuticals announced positive topline data from its Phase II trial of ifenprodil
• Seelos Therapeutics dosed the first patient in an open-label basket study of SLS-005
• Inozyme Pharma announced positive preliminary data from the Phase I part of its ongoing Phase I/II trial of INZ-701
• Kowa Pharmaceuticals America published Phase III trial results in Pain Practice for Seglentis
• ADC Therapeutics dosed the first patient in its Phase II LOTIS-9 trial of Zynlonta with Rituxan
• Synaptogenix dosed the first patient in its open-label dose optimization trial of Bryostatin-1
• Celldex Therapeutics dosed the first patient in its Phase II trial of barzolvolimab
• Takeda announced positive results from its Phase III ADVANCE-1 trial of Hyqvia

It’s mid-July, but that doesn’t mean clinical trial announcements are slowing down. Here’s a look at some of the more intriguing stories of the past week.

Prometheus Biosciences completed enrollment for Cohort 1 of the ARTEMIS-UC Phase II trial of PRA023 in ulcerative colitis (UC). The company recently completed enrollment for the APOLLO-CD trial of the drug for Crohn’s disease. Topline data from both trials are expected in the fourth quarter of this year. PRA023 is an IgG1 humanized monoclonal antibody that blocks tumor necrosis factor (TNF)-like ligand 1A (TL1A).

Algernon Pharmaceuticals announced positive topline data from its Phase II trial of ifenprodil for idiopathic pulmonary fibrosis (IPF) and chronic coffee. In the trial, 65% of patients had stable or improved forced vital capacity over the 12-week treatment period with statistically significant compared to an anticipated placebo effect of 40%. Ifenprodil is an N-methyl-D-aspartate (NMDA) receptor antagonist specifically targeting the NMDA-type subunit 2B (GluN2B), which prevents glutamate signaling.

Seelos Therapeutics dosed the first patient in an open-label basket study of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for patients with amyotrophic lateral sclerosis (ALS) in Australia. The drug is also being evaluated in spinocerebellar ataxia and Huntington’s disease. SLS-005 is a low molecular weight disaccharide that crosses the blood brain barrier. It is believed to stabilize proteins and activate autophagy by activating Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression.

Inozyme Pharma announced positive preliminary biomarker, safety, and pharmacokinetic data from the first three patients treated in the Phase I part of its ongoing Phase I/II trial of INZ-701 in adults with ABCC6 Deficiency, which presents as pseudoxanthoma elasticum (PXE) in older individuals. At the 0.2 mg/kg dose level, all three patients demonstrated rapid and significant increases in PPi levels. ABCC6 Deficiency is a rare, severe, inherited disorder marked by low levels of PPi.

PPi is essential for preventing harmful soft tissue calcification and regulating bone mineralization. The disease affects more than 67,000 people globally and is systemic and progressively debilitating. INZ-701 is a clinical-stage enzyme replacement therapy.

Kowa Pharmaceuticals America published Phase III trial results in Pain Practice reinforcing that Seglentis provided superior analgesic efficacy than tramadol hydrochloride or celecoxib separately. Seglentis is a unique co-crystal combination of celecoxib 56-mg and tramadol hydrochloride 44-mg and leverages four complementary mechanisms of analgesia involving peripheral and central pathways. The drug is being developed as a multimodal treatment option for managing acute pain severe enough to require an opioid.

ADC Therapeutics dosed the first patient in its Phase II LOTIS-9 trial of Zynlonta (loncastuximab tesirine-lpyl) in combination with Genentech and Biogen’s Rituxan (rituximab) for unfit and frail patients with previously untreated diffuse large B-cell lymphoma (DLBCL). These patients are often unable to tolerate the standard of care R-CHOP or even a modified R-CHOP regimen and, as a result, are often excluded from first-line studies. The study’s primary objective is to evaluate the efficacy of a response-adapted treatment of Lonca-R in unfit participants who are previously untreated tor DLBCL and ineligible for standard R-mini-CHOP. Zynlonta is a CD19-directed antibody drug conjugate. It carries a pyrrolobenzodiazepine (PBD) payload attached to an antibody against CD19.

Synaptogenix dosed the first patient in its open-label dose optimization trial of Bryostatin-1. This is in preparation for the company’s NIH-sponsored Phase II Alzheimer’s disease trial. The drug recently demonstrated a statistically significant improvement in the treatment group that was not seen in the placebo group in two pilots, placebo-controlled Phase II studies. Enrollment is complete in the first study, and topline data is expected in the fourth quarter of 2022 for the Phase IIb study in advanced and moderately severe Alzheimer’s disease patients. Bryostatin is being developed for Fragile X syndrome, multiple sclerosis, stroke, traumatic brain injury, and some cancers.

Celldex Therapeutics dosed the first patient in its Phase II trial of barzolvolimab for the two most common forms of chronic inducible urticaria (CIndU) — cold urticaria and symptomatic dermographism. The drug is a humanized monoclonal antibody that binds to the receptor tyrosine kinase KIT. Cold urticaria is marked by hives or wheals that have a specific trigger attributed to them. Cold urticaria symptoms include itching, burning wheals and angioedema when skin is exposed to temperatures below skin temperature.

Takeda announced positive results from its Phase III ADVANCE-1 trial of Hyqvia for the maintenance treatment of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The study hit the primary endpoint, demonstrating the drug reduced relapse of neuromuscular disability and impairment when used as maintenance therapy for CIDP. The drug is an Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase. CIDP is a chronic, acquired, immune-mediated disease affecting the peripheral nervous system marked by progressive, symmetric weakness in distal and proximal limbs and impaired sensory function in the extremities.

VistaGen Therapeutics announced topline results from its Phase III PALISADE-1 trial of PH94B for acute treatment of anxiety in adults with social anxiety disorder. The study failed to hit its primary endpoint, as measured by change from baseline using the Subjective Units of Distress Scale (ISUDS) compared to placebo. The company said the tolerability profile was favorable. The trial was designed to relieve anxiety symptoms in adult patients with a social anxiety disorder during a simulated public speaking challenge, measured using the patient-reported SUDS. Prior to the public speaking challenge, the participants received either a single dose of the drug or placebo.