Clinical Research

A male patient who was diagnosed with HIV infection in 2003 has experienced a remission from his disease after a haematopoietic stem cell transplant. The stem cell transplant was undertaken in order to treat a cancer. At the time the patient was on also on antiretroviral therapy to treat his HIV. The patient remained on antiretroviral drug therapy for 16 months after the transplant at which point the physicians stopped the drug therapy. Remarkably, the patient remained free of HIV.

Sangamo Therapeutics Inc is readying a second generation of its zinc finger nuclease technology to use in an in vivo genome editing trial later this year. This is expected to provide further support for the company’s strategy of using engineered nucleases to correct DNA in patients with Hunter syndrome and other genetic diseases. Hunter syndrome is a rare genetic disorder that can lead to tissue and organ damage.

Patients with pancreatic cancer, who were treated with Lynparza during a Phase 3 clinical trial, have shown a statistically-significant and clinically-meaningful improvement in progression-free survival, according to AstraZeneca Plc. The drug’s benefit, reported on 26 February, is the first for a PARP inhibitor in pancreatic cancer, one of the most difficult-to-treat cancers.

A gene therapy intended as a one-time treatment for dry age-related macular degeneration (AMD) has been successfully administered to a patient in the UK, marking the start of one of the first clinical trials of its kind. The therapy consists of a recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor.

An RNA oligonucleotide drug that is being tested as a combination therapy in patients with pancreatic and colorectal cancers has produced positive data in a Phase 1 clinical study. Top-line results from the combination of NOX-A12 (olaptesed pegol) and Keytruda produced responses from patients whose cancers had progressed rapidly on prior therapy.

A Chinese scientist’s reported use of the Crispr gene editing tool to alter a gene in two  embryos which were implanted into a mother’s womb has created an uproar in the scientific community. The edit was performed when the embryos were just a day old and involved alterations to the CCR5 gene in order to prevent the offspring from contracting HIV. They were reported on YouTube videos, rather in a scientific journal, after twin girls bearing the DNA changes were born.

Immunocore Ltd is expanding a 2013 collaboration with Genentech (Roche) to co-develop a bi-specific biologic for cancer. The compound is based on soluble T cell receptors that have been designed to recognize intracellular cancer antigens, and then kill cancer cells through an anti-CD3 effector function.

In this case, the targets are tumours expressing the melanoma-associated antigen A4. Called IMC-C103C, the molecule is poised to enter clinical development in early 2019.

AstraZeneca Plc’s Phase 3 trial of the Imfinzi and tremelimumab checkpoint antibodies did not meet the primary endpoint of improving overall survival compared with standard-of-care chemotherapy in previously untreated patients with Stage IV metastatic non-small cell lung cancer, the company announced on 16 November. This follows the failure of the same combination in July 2017 to reach statistical significance in progression-free survival.

AstraZeneca Plc has reported positive outcome data for its licensed diabetes medicine Farxiga (dapagliflozin) showing that the drug significantly reduces the risk of hospitalisation for heart failure or cardiovascular death among patients with Type 2 diabetes. In a Phase 3 trial, the reduction of risk was 17% compared with a placebo.

A prospective treatment for patients with schizophrenia, who have not responded to other drugs, failed to show statistical superiority in a Phase 3 trial compared with conventional therapy, H. Lundbeck A/S announced on 25 October.

The treatment, Lu AF35700, is a dopamine D1 and D2 antagonist with a high occupancy of the 5-HT2A and 5-HT6 serotonin receptors. The relatively low D2 receptor occupancy compared with the D1 receptor was expected to result in a better side effect profile.