First in Human: Cancer-beating virus; potential stem cell 'game changer'

This is a recurring column on clinical research in the early stages of development, which is referred to as phase 1. These are treatments being used for the first time in a small number of human patients to determine safety, dosing and general pharmacological activity.

Virus targeting breast cancer

Imugene Ltd. started a first-in-human study of CHECKvacc, evaluating the safety and efficacy of the oncolytic virus in patients with triple-negative breast cancer that has spread to other parts of the body. Oncolytic viruses are designed to both selectively kill tumor cells and activate the immune system against cancer cells, with the potential to improve clinical response and survival.

"We're incredibly eager to unlock the potential of CHECKvacc," Imugene CEO Leslie Chong said. "We hope that in time, CHECKvacc provides an improved outcome for the many women who are diagnosed every year," said Chong, who previously served as senior clinical program leader at Roche Holding AG's oncology unit, Genentech.

Invented in the California-based City of Hope research and medical center, CHECKvacc also carries a gene for an anti-PD-L1 antibody, offering more than one anticancer mechanism in a single treatment, with the potential for use across many cancer indications.

The trial's principal investigator said oncolytic viruses, such as CHECKvacc, hold significant potential to improve clinical response and survival.

The Sydney-based cancer group counts Axel Hoos among its board members. Formerly head of oncology for London-based GlaxoSmithKline PLC — until moving to Boston-based biotech Scorpion Therapeutics Inc. in 2020 — Hoos was responsible for replenishing the U.K. pharmaceutical group's oncology business, which had been reduced to a single asset in development after its cancer portfolio was swapped for Novartis AG's consumer business in 2015. At the time of his departure in August 2020, GSK had 14 experimental cancer medicines in development.

Roth Capital Partners analyst Jonathan Aschoff, who rates Imugene stock a 'buy,' also noted that another early-stage trial of CHECKvacc in patients with non-small cell lung cancer is showing early signs of producing an immune response. Imugene will select one of the three phase 1 doses to test in combination with the standard-of-care therapy — possibly a PD-L1 inhibitor or another immunotherapy agent — on non-small cell lung cancer that has either progressed or not responded to prior therapy.

'Game-changing' spinal regeneration possibilities

Dutch stem cell company Neuroplast BV said its investigational Neuro-Cells treatment was safe and well-tolerated in a phase 1 study of patients suffering from traumatic spinal cord injury, or TSCI. Injected into the spinal cord, the treatment uses the patient's own stem cells to prevent further loss of function, potentially limiting the loss of mobility and independence.

Ten patients with spinal cord injuries sustained between one and five years ago received the Neuro-Cells treatment, manufactured from the patients' own bone marrow, in a study carried out in the Hospital Nacional de Parapléjicos in Toledo, Spain. Led by principal investigators Antonio Oliviero and Jörg Mey, the study reported no serious safety concerns or product-related adverse events, according to the Geleen, Netherlands-based company.

"I'm really enthusiastic about the prospects of this therapy and what it means for the future treatment of patients with TSCI," said Oliviero, a doctor at the Hospital Nacional de Parapléjicos. "Neuro-Cells might be a game-changer."

The company, which is seeking additional funding to expand its clinical trials outside of Europe, said it will embark on a phase 2 study to evaluate the efficacy and safety of the stem cell treatment in sub-acute patients.

"Proving the safety of our autologous Neuro-Cells treatment is an important step in the development of a treatment for acute TSCI patients," Neuroplast CEO Johannes de Munter said. "The functional, psychological and financial impacts of traumatic spinal cord injury are broad, and we are committed to advance our treatment for patients worldwide as soon as possible."

Crescendo's psoriasis, cancer collaborations

Crescendo Biologics Ltd., a British immuno-oncology company developing targeted T-cell-enhancing therapeutics, announced a research collaboration with the U.K.'s Institute for Cancer Research focused on its early-stage CB307 compound. The research alliance will look at the pharmacology of CB307 in both the laboratory and in humans, including studies on patient-derived prostate cancer tissues.

"Next-generation immunotherapies could offer much-needed new treatment options to patients with castration-resistant prostate cancer, as well as other cancer types with high prevalence," said Johann de Bono, head of the institute's division of clinical studies. "We expect this collaboration to provide meaningful additional insights into the mechanisms and activity of CB307 in a variety of relevant settings."

A collaboration on a different drug, called ZL-1102, between Crescendo and China's Zai Lab Ltd., reported positive results Oct. 20 from a phase 1 study on patients with psoriasis, triggering an undisclosed milestone payment to the Cambridge, U.K.-based company. The study follows Crescendo's 2018 agreement to grant a worldwide exclusive license to develop and commercialize ZL-1102 for all indications to Zai Lab. The compound, formerly known as CB001, uses the smallest part of an antibody called the Humabody to target the protein IL-17A.

In the phase 1b study of 53 patients with mild-to-moderate plaque psoriasis, ZL-1102 applied to the skin resulted in clinical improvement in the size of skin lesions, while the occurrence of side effects was similar to placebo.

"These are the first clinical data from the first patients to be treated with a Humabody, and we are very pleased with the topline results reported by our partners at Zai Lab," Crescendo's CEO Theodora Harold said. "This is also the first study to demonstrate penetration of a protein biological therapeutic through psoriatic skin resulting in clinical response, illustrating an important point of differentiation of our Humabody platform."