The US Food and Drug Administration has expanded the permitted medical use of a gene therapy for Duchenne muscular dystrophy (DMD), making it available to patients who are both ambulatory and non-ambulatory. DMD is a genetic disorder characterised by a progressive deterioration of muscle due to abnormalities in, or the absence of, dystrophin protein which helps keep muscle cells intact.
Formycon AG of Germany has started clinical development of a candidate biosimilar of Keytruda (pembrolizumab), the checkpoint inhibitor for melanoma and other cancers that has revolutionised medical practice by blocking the interaction between two proteins and activating the immune system to kill cancer cells. To date, Keytruda has been approved for 48 medical indications.
Pfizer Inc has entered into a partnership with a Cambridge, US, based venture capital group and its network of drug discovery and development companies, to identify proteins that might become the basis of new drugs for obesity. The venture capital group is Flagship Pioneering and the drug discovery activities are located at Pioneering Medicines, an in-house unit of Flagship, and Profound Therapeutics, a Flagship spin-off company. Pfizer has been collaborating with Pioneering Medicines since 2023.
A new research project based in Denmark is to examine whether changes in the human microbiome can potentially cause cardiometabolic diseases such as obesity, type 2 diabetes and cardiovascular disease. Announced on 22 May, the project will be initially financed by the Novo Nordisk Foundation, a philanthropic organisation whose assets are managed by Novo Holdings. Novo Holdings is the controlling shareholder of Novo Nordisk A/S, developer of the obesity drugs liraglutide and semaglutide.
GSK Plc has acquired Elsie Biotechnologies of San Diego, US, in order to expand its presence in the field of oligonucleotide drugs. These are small pieces of DNA or RNA that can block the ability of RNA to make proteins. The two companies have been collaborating since July 2023 on the development of potential therapies for chronic hepatitis B and steatotic liver disease.
Data from an early clinical trial of a small molecule drug intended to treat solid tumours by modulating an enzyme that plays a key role in the presentation of antigen to the human immune system were positive, according to the developer Grey Wolf Therapeutics Ltd. Data from the Phase 1/2 study were presented at the 2024 American Society of Clinical Oncology annual meeting on 3 June. The drug, GRWD5769 was well-tolerated after repeat dosing. It also showed an ability to modulate the immunopeptidome.
Blincyto (blinatumomab), a bispecific antibody first approved for cancer in 2014, has received a new indication from the US Food and Drug Administration, extending its coverage for the treatment of B cell acute lymphoblastic leukaemia (B-ALL). B-ALL, an aggressive leukaemia, is the most common type of acute lymphoblastic leukaemia.
The new indication is for the treatment of adults and children from the age of one month for CD19 positive Philadelphia chromosome-negative B cell precursor acute lymphoblastic leukaemia.
A gene therapy being investigated in a Phase 3 trial of boys with Duchenne muscular dystrophy (DMD) has failed to reach its primary endpoint of improvement in motor function, the developer Pfizer Inc announced on 12 June. The therapy, fordadistrogene movaparvovec, missed the primary endpoint at one year as well as key secondary endpoints compared with a placebo. The secondary endpoints included an improvement in velocity in a 10 metre run or walk. Participants were boys between the ages of four and seven who were on a daily regimen of glucocorticoids.
Donanemab, an Alzheimer’s disease drug developed by Eli Lilly and Co, is set for a review by the US Food and Drug Administration in the wake of a recommendation by the agency’s outside panel of advisers on 10 June that the drug is both safe and effective. Neither the FDA nor Lilly would comment to MedNous on a date for the final review. Nevertheless analysts who followed discussions by the panel – the Peripheral and Central Nervous System Drugs Advisory Committee – expect that whatever the timing, a formal approval is likely.
A 2019 spin-out of the Max-Planck-Institute of Colloids and Interfaces has raised additional seed finance from a venture capital syndicate to progress antibody treatments for cancer. Tacalyx GmbH announced the funding on 11 June which increases its seed finance to more than €14 million. The company is developing antibody therapeutics that target tumour associated carbohydrate antigens. These are glycan structures often found on cancer cells but which don’t usually appear on other tissues.