Biogen Inc.’s phase III trial of tofersen in a form amyotrophic lateral sclerosis (ALS) has missed its main goal, but the company said it is still talking with regulators after seeing positive trends in the data.
PTC Therapeutics Inc. is already established as a player in rare diseases, working with Roche Holding AG to develop and market Evrysdi (risdiplam) to treat certain patients with spinal muscular atrophy. With Evrysdi now approved in the U.S. and Europe, and Translarna (ataluren) approved in Europe for Duchenne muscular dystrophy, South Plainfield, N.J.-based PTC is approaching a crucial juncture with its first gene therapy product.
Mitochondrial disease, where the tiny energy factories in cells aren’t functioning properly, can have devastating and sometimes fatal consequences. As there are no approved treatments, patients rely on strategies such as strict diets and conserving energy to try and control symptoms that can affect organs including the brain, kidneys, muscles, pancreas and heart. Netherlands-based biotech Khondrion BV is one of a group of companies that is making progress against this group of rare inherited conditions that affects around one in every 4,000 people.
Cambridge, U.K.-based Astrazeneca plc has new data from its long-acting COVID-19 antibody combination, AZD-7442, which aims to provide longer protection, potentially for up to a year. Latest data show the intramuscularly injected drug achieved a statistically significant reduction in severe COVID-19 or death compared to placebo in non-hospitalized patients with mild to moderate symptomatic disease.
The biopharma industry is full of amazing ideas, but getting funding for them and turning them into medicines for the real world is a huge challenge. As anyone in the industry knows, getting investors to back your company can be as challenging as getting the science to work and can take a significant amount of the company’s time and effort.
Intergalactic Therapeutics Inc. is aiming for the stars with a nonviral gene therapy platform, backed with $75 million in series A financing from life sciences venture capital firm Apple Tree Partners. While management are tongue in cheek about the name of the company, they are serious about their mission to produce a next generation of gene therapies that overcome the issues associated with marketed adeno-associated virus-based products.
Exscientia plc has raised $510.4 million in an upsized Nasdaq IPO and private funding round as it pushes forward with its artificial intelligence-based drug discovery mission. The Oxford, U.K.-based company aims to out-license some of its candidates to other companies, while saving others for its own pipeline.
Developing the next generation of antibiotics is proving to be a major problem, with research reaching an almost standstill due to a lack of funding because of the poor return on R&D investment from the products.
Merck & Co. Inc. and Ridgeback Biotherapeutics Inc. are planning a U.S. emergency filing for potentially the first oral antiviral for COVID-19, after a phase III trial showed molnupiravir cut risk of hospitalization or death by around 50%.
