The European Medicines Agency has given a positive opinion to a new drug for Duchenne muscular dystrophy (DMD), a genetic disorder primarily affecting males, which can lead to progressive muscle degeneration and weakness. The developer is Santhera Pharmaceuticals AG of Switzerland. The drug, Agamree (vamorolone), is a small molecule anti-inflammatory medicine that is being positioned as an alternative to corticosteroids, the current standard of care.
Pfizer Inc has received regulatory approval in the US for a personalised, or precision, medicine for lung cancer that demonstrated efficacy in a single-arm trial in both treatment naïve and previously treated patients with BRAF-V600E mutant metastatic non-small cell lung cancer. The medicine, Braftovi (encorafenib) and Mektovi (binimetinib), is a combination therapy that was first approved in 2018, but has since been updated. The newest approval includes two companion diagnostics that can identify BRAF V600E mutations in plasma or tumour tissue.
In a boost for precision medicine, Bristol Myers Squibb Co is to spend up to $5.8 billion to buy Mirati Therapeutics Inc of the US which uses molecular biology to design drugs that are intended to arrest cancer growth by targeting specific genetic mutations. BMS will pay $58 per share for the precision medicine company, representing an outlay of $4.8 billion. If Mirati shareholders decide to buy additional non-tradeable contingent value rights, the value of the acquisition could rise to as much as $5.8 billion.
On 4 October, Novartis completed the spin-off of Sandoz, its generic and biosimilar business unit, enabling it to focus exclusively on the development and sale of proprietary medicines. The long anticipated transaction involved the listing of Sandoz Group AG shares on the SIX Swiss Exchange in Zurich, Switzerland and its American Depositary Receipts on an over-the-counter market in the US. Each shareholder of Novartis received one Sandoz share for every five Novartis shares held, or one Sandoz ADR for every five Novartis ADRs.
The radiopharmaceutical sector has a new player. This is Eli Lilly and Co which is to pay $1.4 billion to acquire POINT Biopharma Global Inc of Indianapolis, US, a clinical stage company with candidate radioligand therapies for the treatment of cancer. The deal, valued at $12.50 per share in cash, has been approved by the boards of both companies and is expected to close by the end of the year.
Sanofi SA is to expand its portfolio with the in-licensing of a candidate vaccine targeting extraintestinal pathogenic Escherichia coli, a gram negative bacterium that causes urinary tract infections and potentially sepsis. The vaccine was developed by Janssen Pharmaceuticals Inc and is currently in Phase 3 development
A diagnostic test for cancer was approved by the US Food and Drug Administration on 30 September which will help physicians detect hundreds of genetic variants associated with a person’s elevated risk of developing certain malignancies. Produced by Invitae Corp, the test also identifies potentially cancer-associated hereditary variants in individuals who have already been diagnosed with cancer.
France-based Nanobiotix SA is taking steps to conserve cash ahead of results from a Phase 3 trial of its lead radioenhancer NBTXR3 for the treatment of head and neck cancer. The data are expected to be reported in the second half of 2024 which, if positive, would lead to registration and unlock significant milestone payments from the company’s partner Janssen Pharmaceutica NV. Nanobiotix is developing radioenhancers for use with radiotherapy to treat patients with multiple, difficult cancers.
Ono Pharmaceutical Co Ltd, one of Japan’s largest pharmaceutical companies, is to expand its oncology pipeline with the addition of more bispecific antibodies, a therapy type that is said to enable better precision medicine for treating cancer. Under a collaboration with Adimab LLC of the US, the Japanese company will have an option for exclusive rights to novel bispecific antibody candidates for the treatment of multiple cancers. The deal involves an undisclosed upfront payment, R&D funding, and success-based milestone payments.
AstraZeneca Plc has reported positive data from a Phase 3 trial of its antibody-drug conjugate datopotamab deruxtecan in patients with metastatic hormone receptor (HR) positive, HER2-low or negative breast cancer, the most common breast cancer subtype, accounting for more than 65% of cases diagnosed globally. The trial enrolled 700 patients across five continents.