An enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD), a rare genetic disease that can cause premature death, has cleared the US Food and Drug Administration making it the first medicine to treat symptoms of the disease that are not related to the central nervous system. The FDA approval of Xenpozyme (olipudase alfa) was granted to Genzyme, a division of Sanofi SA.
The US Food and Drug Administration has approved two new bivalent vaccines to protect against Covid-19. They are to be administered as an updated single booster dose at least two months after primary or booster vaccination. The decisions were taken in response to the prevalence of the omicron variant of the SARS-CoV-2 virus and specifically to its two new subvariants BA.4 and BA.5.
The Bill & Melinda Gates Foundation has invested $5 million in a Danish company that is developing a new type of biologic to strengthen the gut microbiome and overcome resistance to antibiotics. In a statement on 30 August, Bactolife A/S said its technology is designed to reduce the risk of bacterial infections in both humans and animals. Novo Seeds, a unit of Novo Holdings A/S, led a €7 million seed financing round for Bactolife in April 2021.
Finland-based Kaiku Health Oy and Novartis have expanded their partnership in digital medicine in order to use machine learning to predict symptoms of multiple diseases. The two companies have been collaborating since June 2021 on a project involving the monitoring of patients with melanoma. Using Kaiku’s digital platform, data is collected from cancer patients and then fed back to healthcare workers to help them personalise care.
A spin-out of the Belgian university KU Leuven (Katholieke Universiteit Leuven) has raised €30 million from a large group of venture capitalists in order to develop a new generation of vaccines. AstriVax NV will use the seed funding to bring a thermostable yellow fever vaccine into clinical development and further develop candidate vaccines for rabies and chronic hepatitis B.
Novartis is to spin off Sandoz, its generics business, into a separately incorporated Swiss company by the second half of 2023 in order to focus on proprietary medicine development. The decision, announced on 25 August, follows a year-long strategic review that included the options of retaining Sandoz as an autonomous business unit within the group, or divesting it.
A Phase 3 trial of a new monoclonal antibody for rheumatoid arthritis has been shown to be at least as effective as Humira (adalimumab) and methotrexate, the current standard of care, potentially increasing the treatment options for patients who haven’t been able to control their disease with methotrexate chemotherapy alone. The experimental drug, olokizumab, targets the interleukin-6 (IL-6) cytokine directly. This distinguishes it from Humira which targets tumour necrosis factor-alpha and three other antibody drugs that target the IL-6 receptor.
Belgium-based eTheRNA NV has changed its business strategy in order to exploit the potential of its technology platform to provide services to the RNA therapeutics industry. Previously, the company’s focus was on proprietary messenger RNA (mRNA) drug development. But with the support of a new €39 million financing round, the company has changed its model to focus on services.
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.