Sanofi SA announced the discontinuation on 17 August of its breast cancer programme AMEERA which was investigating amcenestrant, an oral estrogen receptor degrader, in several breast cancer indications. The decision follows the failure of a Phase 3 trial of the drug in patients with estrogen receptor-positive (ER+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer.
Merck & Co Inc has entered into a collaboration with the Cambridge, US biotech company Orna Therapeutics Inc to discover new therapeutics using engineered RNA molecules. Simultaneously, Orna has raised $221 million in a Series B financing round to accelerate development of its technology platform which includes a proprietary lipid nanoparticle delivery system. Orna calls its engineered RNA molecules circular RNA therapeutics (oRNA) because they have been transformed from a linear format into a circular format.
The first cell-based gene therapy for the treatment of patients with beta thalassaemia, an inherited blood disorder, was approved by the US Food and Drug Administration on 17 August. Zynteglo (betibeglogene autotemcel) is a one-time treatment administered as an ex vivo lentiviral vector gene therapy. A functional beta globin gene is added into a patient’s cells outside of the body and then infused back into the patient. The treatment process is comprised of several steps and will be administered at qualified treatment centres in the US.
H. Lundbeck A/S and its Japanese partner Otsuka Pharmaceutical Co Ltd expect to make a regulatory filing for a new indication for Rexulti, an approved drug for schizophrenia and depression, before the end of the year. The proposed indication, for agitation in patients with Alzheimer’s dementia, would be supported by positive data from a Phase 3 study showing a statistically significant reduction in agitation in patients receiving the drug compared with placebo. The trial results were reported in June.
Enhertu (trastuzumab deruxtecan) has been approved by the US Food and Drug Administration to treat non-small cell lung cancer whose tumours have activating HER2 mutations. The accelerated approval comes just a week after Enhertu, an antibody-drug conjugate (ADC), was authorised by the FDA for a new breast cancer indication. The decision, announced on 12 August, brings the total number of US regulatory approvals for the drug up to five, all of which are for cancers expressing the HER2 protein.
Verona Pharma Plc has raised $150 million from a public share offering on Nasdaq in order to support development of ensifentrine, its small molecule drug for chronic obstructive pulmonary disease. The UK company recently completed the first of two Phase 3 trials of ensifentrine in preparation for a possible regulatory submission to the US Food and Drug Administration in the first half of 2023.
A Phase 3 trial of canakinumab in patients with non-small cell lung cancer failed to meet its primary endpoint of disease-free survival compared with placebo despite earlier positive signals of the drug’s potential in the disease. The developer, Novartis, announced the results on 15 August. Canakinumab (Ilaris) is an approved treatment for arthritis and periodic fever syndromes and was being studied as an adjuvant treatment in patients with stages 2 to 3A and 3B lung cancer. The Phase 3 trial did not throw up any unexpected safety signals.
GlaxoSmithKline Plc has obtained an exclusive option to co-develop and commercialise an experimental antibody-drug conjugate (ADC) that is designed to activate the innate immune system in order to fight cancers expressing the HER2 protein. The partner is Mersana Therapeutics Inc of Cambridge, US which has a pipeline of clinical and preclinical ADCs.
Genmab A/S has raised its forecast for revenue and operating profit in 2022 after seeing a sharp increase in royalty income in the first half from Darzalex (daratumumab), a CD38 monoclonal antibody for multiple myeloma. Revenue is now expected to be between DKK 12,000 to 13,000 million and operating profit between DKK 3,800 to 5,400 million. In 2021, revenue was DKK 8,482 million (€1,140 million) and operating profit was DKK 3,018 million.
The US Food and Drug Administration has extended a clinical hold on a proposed Phase 3 clinical trial of a microbiome-based therapeutic developed by MaaT Pharma SA of France because it requires more information about the composition of the faecal-derived product. MaaT Pharma announced the clinical hold extension on 10 August.