GSK plc is preparing filings for its respiratory syncytial virus (RSV) vaccine in older people following supportive late-stage trial results, a product which could play a vital role as it spins out its consumer business arm. The vaccine could be a potential blockbuster for GSK, worth as much as $7 billion in peak annual sales if approved in all its potential uses – although trials in pregnant women remain on hold because of safety concerns.
Bluebird’s beta-thalassemia gene therapy in spotlight at US FDA adcom meeting
The U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) kicked off its second-day session to evaluate Bluebird Bio Inc.’s betibeglogene autotemcel for people with beta-thalassemia who require regular red blood cell transfusions. On June 9, CTGTAC voted 15-0 that benefits outweigh risk with another of the company’s products, elivaldogene autotemcel for early active cerebral adrenoleukodystrophy in patients without a matched sibling donor. Trading of Bluebird shares (NASDAQ:BLUE) was halted both days.
EHA 2022: Best in class? Cogent soars on early data in mastocytosis
Shares of Cogent Biosciences Inc. rocketed up more than 80% in morning trading on early data, strong clinical efficacy and a safety profile that could differentiate lead asset bezuclastinib from existing kinase inhibitors approved for advanced systemic mastocytosis. Though only 11 patients have been enrolled in the phase II study to date, all have so far achieved a 50% or greater reduction in serum tryptase levels and all eight patients who have had bone marrow exams achieved 50% or greater reduction in bone marrow mast cells. Data were presented June 10 at the European Hematology Association Congress in Vienna.
Trevi heavy as Algernon bears down in IPF cough space
A paper published March 22 in the journal Chest highlighted research that found patients with interstitial lung disease who had poor quality of life specifically related to cough were more likely to be hospitalized for respiratory issues, need a lung transplant and die. The findings in 1,447 patients matter because they back previous investigations and provide a way of predicting the advance of ILD, of which idiopathic pulmonary fibrosis (IPF) is a prominent type. A handful of companies are working on drugs to tackle cough in IPF, often an early symptom of the disease.
EMA is tracking COVID-19 medicines to avoid shortages
The EMA has a list and it’ll be checking it frequently to avoid shortages with the help of COVID-19 marketing authorization holders and EU members states. The agency’s Medicines Shortages Steering Group just issued the list of 14 approved medicines it deemed essential for handling COVID but it needs help keeping tabs on supply and demand.
Boehringer Ingelheim to develop A*STAR antibodies for targeted cancer therapies
Singapore’s Agency for Science, Technology and Research (A*STAR) has inked a deal with Boehringer Ingelheim GmbH (BI) to develop and commercialize targeted cancer therapies. BI will obtain exclusive worldwide rights to research, develop and commercialize products based on a panel of engineered antibodies from A*STAR, which may allow for specific targeting of tumor cells without binding to healthy cells.
Rznomics raises ₩37.2B in series C round
Rznomics Inc. recently secured ₩37.2 billion (US$30 million) from private investors and the Korean government in a series C financing round. “The $30 million series C proceeds will be used for the phase I/IIa clinical trial of RZ-001, and the additional R&D costs for candidates RZ-004 and RZ-003. The $6.4 million research grant will be used for RZ-004 and other candidates,” Rznomics CEO Seong-wook Lee told BioWorld.
The BioWorld Insider Podcast: New therapies vie to change fatal course of amyotrophic lateral sclerosis
As multidisciplinary care extends survival and improves the quality of life for people with amyotrophic lateral sclerosis (ALS), can a host of new ALS drugs vying to slow its progression make a difference? A surprise extension of the U.S. FDA review for Amylyx Pharmaceuticals Inc.’s AMX-0035 to review new data, plus near-term readouts from a major ALS platform trial may soon provide answers. Join the BioWorld Insider podcast as we talk with executives from Amylyx and Clene Inc., and a key Penn State ALS expert on what the future of medicine may hold for ALS patients.
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