Company News

The US Food and Drug Administration has approved a new gene therapy for spinal muscular atrophy (SMA), a neurological disorder that can lead to progressive muscle weakness and in the most severe cases, death. The therapy, Itvisma (onasemnogene abeparvovec), has the same active ingredient as the gene therapy Zolgensma, but formulated at a different concentration. Zolgensma was approved by the FDA in 2019 to treat paediatric patients with SMA. The newest therapy extends the treatment group to older individuals with a confirmed mutation in the survival motor neuron 1 (SMN1) gene.

China-based multinational Tencent and the UK’s Business Growth Fund (BGF) have joined an investor syndicate to provide new Series A funding to T-Therapeutics Ltd, a Cambridge University spin-out which is developing T cell receptor (TCR) T cell therapies. The latest funding, for $32 million, brings the total amount raised under the Series A round to $91 million. It will be used to advance the company’s prospective T cell receptor products to the clinic. The therapeutic focus is on cancer and autoimmune disease.

Gate Bioscience Inc, a US company founded in 2021 to develop small molecule drugs targeting disease-causing extracellular proteins, has received $65 million in Series B financing to take its lead programmes into clinical development. This brings total capital raised for the company to $135 million. The financing was led by Forbion of the Netherlands with Eli Lilly and Co as a new investor. 

Artios Pharma Ltd, a UK clinical-stage company with products to kill cancer cells by blocking their ability to repair damaged DNA, has raised $115 million in an oversubscribed Series D financing. The round was co led by founding investor SV Health Investors and new investor RA Capital Management. The funds will be used to advance two small molecule drugs against large cancers. The first, alnodesertib, is an inhibitor of the ATR protein which is involved in DNA repair, and the second is ART6043, an inhibitor of the DNA repair enzyme DNA polymerase theta (Pol theta).

BC Platforms AG, one of the healthcare industry’s largest data management and analytics companies, has launched a new technology platform that features artificial intelligence as its core component. This comes at a time when AI is being applied across drug discovery in order to help researchers analyse large datasets and identify potential drug targets. The Switzerland-based company says that its new technology takes AI one step further by linking  drug discovery and development data to real-world evidence. 

A three-year project to develop new strategies for tackling resistance to antibiotics will start in 2026, GSK Plc announced on 18 November. The project will receive £45 million from GSK to execute six projects which have been identified by Imperial College London and Imperial College Healthcare NHS Trust, leaders of the Fleming Initiative in the UK. The funding will support an estimated 50 UK scientific and academic jobs focusing exclusively on antimicrobial resistance.

Switzerland-based Novartis announced plans on 19 November to build a new manufacturing hub in North Carolina, US, which is part of a previously announced $23 billion investment in the country over the next five years. The outlays will feature new facilities in Durham to manufacture biologics and sterile packaging; a site in Morrisville to produce solid dosage tablets and capsules; and an expansion of an existing site in Durham to support the sterile filling of biologics into syringes and vials.

A trial for adults with major depressive disorder failed to significantly reduce the severity of the disorder compared with a placebo, according to the sponsor Neurocrine Biosciences Inc of San Diego, US. The Phase 2 study enrolled 73 adults with a diagnosis of major depressive disorder and an inadequate response to at least one antidepressant in their regular treatment paradigm. The product, NBI-1070770, is a negative allosteric modulator of a subunit of a receptor that plays a role in the working memory. Participants received the drug or a placebo for four weeks.

Germany-based biotech InflaRx NV, has published positive Phase 2a data for a new small molecule drug for the treatment of two skin diseases: hidradenitis suppurativa and chronic spontaneous urticaria. The drug, INF904, is an inhibitor of the C5a receptor which modulates the body’s inflammatory responses. In a basket study of patients with the two diseases, the drug achieved rapid disease control. In the case of hidradenitis suppurativa this involved decreases in measures of nervous system changes.

An antibody-drug conjugate, made with a toxic mushroom rather than chemotherapy, has shown promising results in a Phase 1/2a trial in patients with multiple myeloma. The drug, HDP-101 (pamlectabart tismanitin), is being developed by Heidelberg Pharma AG of Germany. In October it received a ‘fast track’ designation from the US Food and Drug Administration for its potential as a treatment for severely ill and heavily pretreated patients.