Updated MRI results from Clene Inc.’s phase II Visionary-MS trial testing gold nanocrystal therapy CNM-Au8 bolsters the company’s premise that targeting energy metabolism could protect neuronal function in patients with multiple sclerosis and lays the groundwork for a phase III trial as soon as a strategic partner is found.
Adhering to the recommendation of a mixed advisory committee vote, the U.S. FDA cleared use of GSK plc’s daprodustat as the first oral hypoxia-inducible factor prolyl hydroxylase (HIF-PHI) inhibitor for treating anemia in patients with chronic kidney disease (CKD) who are on dialysis. For CKD patients not on dialysis, the agency determined the drug’s safety has not been established.
Winning the race to market with the first oral selective estrogen receptor degrader (SERD) for breast cancer is Menarini Group’s elacestrant, which gained U.S. FDA approval for use as second- and third-line therapy in patients with ER-positive/HER2-negative advanced or metastatic disease with the ESR1 mutation. It marks the first therapy approved specifically targeting ESR1, found in up to 40% of patients with ER-positive/HER2-negative disease.
Mantle cell lymphoma (MCL) patients developing resistance to existing BTK inhibitors now have a new treatment option, with the U.S. FDA’s accelerated approval of Jaypirca (pirtobrutinib) from Eli Lilly and Co.’s oncology unit, Loxo@Lilly. It is indicated for use in adults with relapsed or refractory disease who have received at least two lines of systemic therapy, including a BTK inhibitor.
Looking ahead to potential commercialization of its late-stage tau PET imaging agent, Aprinoia Therapeutics Inc. has chosen to go public via a merger with special purpose acquisition company (SPAC) Ross Acquisition Corp. II in a deal that has an equity value of $280 million. The funding is aimed at getting candidate 18F-APN-1607 to the market in China.
Leveraging a computational platform designed to exploit the full potential of a widely studied pharma target has proved successful in attracting venture investors in the last few years to Structure Therapeutics Inc. The company now is giving the public markets a go, aiming to raise up to $100 million in what marks the first biopharma U.S. IPO filing for 2023.
Juvena Therapeutics Inc. co-founder and CEO Hanadie Yousef had the company’s name picked out several years before it was officially incorporated in 2017 to combine Yousef’s work in the mechanics of aging with an underutilized class of biologics and an advanced proteomics platform to tackle chronic and age-related diseases.
Trex Bio Inc., which kicked off 2022 with a big pharma partnership, is back at it again, starting the new year with a potential $1.1 billion agreement with original backer Eli Lilly and Co. targeting immune-mediated diseases. Under the terms, Trexbio gets $55 million up front, with Lilly picking up an exclusive worldwide license for candidates from three programs.
Geron Corp. executives highlighted a potential billion-dollar market opportunity for imetelstat on the back of positive phase III data for the telomerase inhibitor in patients with lower-risk myelodysplastic syndromes (MDS), news that sent shares (NASDAQ:GERN) rising 36% and could portend the first regulatory win for a scientific approach the company has pursued since the 1990s.
Despite pipeline setbacks in 2022, TG Therapeutics Inc. ended the year on a positive note, with U.S. FDA approval of its glycoengineered CD20 monoclonal antibody, ublituximab, in relapsing multiple sclerosis (MS). Branded Briumvi, the drug is set to go up against approved anti-CD20 antibodies Kesimpta (ofatumumab, Novartis AG) and Ocrevus (ocrelizumab, Roche Holding AG).
