Clinical Research

The Food and Drug Administration is to allow Intellia Therapeutics Inc to include the US in its global trial of a genome editing therapy for hereditary angioedema (HAE), a rare protein deficiency that can cause swelling in multiple parts of the body. To date, the Phase 1/2 study has been taking place at sites in the Netherlands, New Zealand and the UK. The addition of sites in the US will increase the geographical reach of the programme, as well as the number of patients receiving the experimental therapy. Intellia is based in Cambridge, US.

Bolstered by $60 million in receipts from a private share placement, Immunic Inc ended 2022 with cash and investments of $116.4 million, enabling it to fund the clinical development of its products for multiple sclerosis, psoriasis, and celiac disease into the fourth quarter of 2024. The financing, completed in October 2022, was supported by new and existing investors including Deep Track Capital, Commodore Capital and BVF Partners LP.

Long-term data from two Phase 3 trials of Cosentyx (secukinumab), an approved drug for plaque psoriasis and other inflammatory indications, have shown sustained efficacy and symptom improvement in patients with hidradenitis suppurativa (HS). HS is a recurrent skin disease causing abscesses that can lead to wounds and scarring. The two trials enrolled more than 1,000 patients in 40 countries.

The Roche Group has reported positive data from a Phase 3 programme of the monoclonal antibody crovalimab in people with paroxysmal nocturnal haemoglobinuria (PNH), a rare and life-threatening blood condition. PNH occurs when red blood cells are destroyed by the complement system, part of the innate immune system. Participants in the second of two studies had not been previously treated with complement inhibitors.

An antibody drug developed by Boehringer Ingelheim GmbH to prevent flares in generalised pustular psoriasis (GPP), a rare skin disease, has met its primary and key secondary endpoints in a Phase 2b trial of patients with the disease. Spesolimab (Spevigo) has already been approved for the treatment of the disease.

Merck & Co Inc has stopped a trial of Keytruda (pembrolizumab) which was being investigated to treat prostate cancer as part of a combination therapy with the hormone medicine enzalutamide and androgen deprivation therapy. The decision was based on the recommendation of the trial’s independent data monitoring committee on the basis of data from the Phase 3 KEYNOTE-991 trial. The decision was announced on 25 January.

The US Food and Drug Administration has issued a complete response letter (CRL) to Eli Lilly and Company for its application for an accelerated approval of the investigative treatment for Alzheimer’s disease donanemab. A CRL is a formal notice to a company in which the agency explains why it cannot accept an application for a drug approval, usually citing deficiencies in the application itself. Donanemab is a monoclonal antibody that targets beta amyloid plaque in the brain for the treatment of patients with early symptomatic Alzheimer’s disease.

A Phase 3 trial of a candidate vaccine to prevent infection from the human immunodeficiency virus (HIV) has failed and the clinical trial is being discontinued, Janssen Pharmaceutical Companies announced on 18 January. The decision was taken on the advice of the study’s independent Data and Safety Monitoring Board which concluded that the vaccine was not effective in preventing HIV infection compared with a placebo. No safety issues related to the vaccine were identified.

The Roche Group has reported the success of a combination therapy in reducing the risk of cancer recurrence in patients with early-stage hepatocellular carcinoma. A Phase 3 trial which combined the immune checkpoint inhibitor Tecentriq (atezolizumab) and Avastin (bevacizumab) and was administered as an adjuvant treatment after surgery, showed a statistically significant improvement in recurrence-free survival in patients at an increased risk of their disease returning.

CureVac NV and its partner GSK Plc have reported promising preliminary data from ongoing Phase 1 studies of vaccine candidates directed against both Covid-19 and influenza. The experimental medicines are based on messenger RNA. The results showed that vaccine candidates using a modified second-generation mRNA backbone produced promising immunogenicity and reactogenicity profiles in both indications. The modified vaccines are directed against specific antigens.