AC Immune SA of Switzerland has extended its cash runway into 2027 following receipt of a milestone payment from partner Janssen Pharmaceutical Inc and contract revenue from other collaborations for its portfolio of medicines and diagnostics for Alzheimer’s and Parkinson’s diseases. Cash on 30 September was CHF 157.9 million ($178.78), up from CHF 103.1 million on 31 December 2023. This was bumped up in October with a CHF 24.6 million payment from Janssen.
Autolus Therapeutics Plc, a UK-based cell therapy company, reached its first regulatory milestone on 8 November when the US Food and Drug Administration approved its chimeric antigen receptor (CAR) T cell therapy for cancer. The therapy, Aucatzyl (obecabtagene autoleucel), targets the CD19 antigen on B cells. The approval is to treat B cell acute lymphoblastic leukaemia (ALL), an aggressive type of blood cancer. It was based on evidence from a single-arm trial in adults whose disease had relapsed after two or more prior lines of therapy.
A new treatment for acute respiratory distress syndrome (ARDS) received a positive opinion from the European Medicines Agency on 15 November paving the way for its use in patients with a coronavirus infection. The treatment, Gohibic (vilobelimab), is a monoclonal antibody directed against a component of the complement system which is part of the body’s immune system. By targeting the complement component C5a, the antibody is believed to block an inflammatory response induced by severe SARS-CoV-2 infection. The treatment has been developed by InflaRx NV of Germany.
A new gene therapy has been given an accelerated approval in the US for an inherited disorder affecting the way neurons transmit information to other cells in the body. The therapy, Kebilidi (eladocagene exuparvovec), has been shown to be effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency, a rare disease. It is caused by mutations in the DDC gene leading to shortages of the AADC enzyme which plays an important role in the nervous system.
After a second review, the main scientific committee of the European Medicines Agency has recommended approval of Leqembi (lecanemab) for the treatment of mild cognitive impairment due to early Alzheimer's disease in patients who have only one, or no copy, of the ApoE4 gene. The EMA opinion, announced on 14 November, will be forwarded to the European Commission for a decision.
Syncona Ltd, the UK investment group, has reaffirmed its goal of achieving £5 billion in net assets by 2032, but only after significant changes in the structure of its portfolio. With net assets per share down by 5.2% at the half year ended 30 September, the company has embarked on a two-pronged strategy. The first part of the plan is to rebalance the portfolio with an emphasis on companies with assets in clinical and late clinical-stage development.
AstraZeneca Plc is to increase its R&D and manufacturing presence in the US with a new investment of $2 billion at multiple sites across the country. Coupled with existing financial commitments, this will deliver total capital of $3.5 billion to the country by the end of 2026. The money will be invested in an R&D centre in Cambridge, Massachusetts; a biologics manufacturing plant in Maryland; specialty manufacturing in Texas; and cell therapy manufacturing at locations on the country’s west and east coasts.
A prospective drug for schizophrenia failed to show efficacy in two Phase 2 trials leaving the developer, AbbVie Inc, without a molecule to challenge one of its biggest competitors in the field. The drug, emraclidine, is a small molecule positive allosteric modulator of the muscarinic M4 receptor designed to harness the psychosis in schizophrenia. Current antipsychotic drugs target a direct blockade of dopamine receptors which are overactive in schizophrenia.
Mario Draghi, former president of the European Central Bank, has issued another appeal to policymakers to build an integrated capital market for the EU. In a commentary in the Financial Times on 2-3 November, Mr Draghi argues that a unified market would mobilise private capital to back projects in defence, the building of a digital infrastructure, and the mitigation of climate change. Currently the EU is home to more than 30 national and regional stock exchanges, each with its own regulations. A similar decentralised structure exists for bond markets.
Sanofi SA has become the latest pharma company to wind down its interest in consumer healthcare in order to focus on proprietary, prescription medicines. The French company is to sell a 50% controlling stake in Opella, its consumer business, to the US private equity group Clayton Dubilier & Rice. Under the deal, Sanofi will retain a significant interest in Opella, while increasing investment in new biopharma products and vaccines, according to Paul Hudson, the chief executive.