Praxis Precision Medicines Inc. CEO Marcio Souza said it would be “disingenuous not to move forward” – U.S. FDA willing – into a phase III effort with an alternate design targeted for the second half of this year, given top-line results from the phase IIb Essential-1 study with ulixacaltamide (PRAX-944) for essential tremor.

Junshi Biosciences Co. Ltd.’s ongericimab, a recombinant humanized anti-PCSK9 monoclonal antibody, met the primary endpoints of reducing the levels of low-density lipoprotein cholesterol in two phase III trials in primary hypercholesterolemia and mixed hyperlipidemia.

Abbott Laboratories continues to push its presence in the cardiovascular market with offerings for the left atrial appendage (LAA) closure and transcatheter aortic valve implant (TAVI) markets, both of which generated affirmative data presented at this year’s edition of the annual meeting of Cardiovascular Research Technologies (CRT 2023) in Washington.

A shortage of efficacy compared to placebo in a phase II study of treating cognitive impairment has put Aptinyx Inc. on the defensive. The company’s oral, small-molecule NMDA receptor modulator, NYX-458, was being studied in 99 patients with mild cognitive impairment or mild dementia associated with Parkinson’s disease or Lewy body dementia. Based on the results, Aptinyx has decided to stop the therapy’s development, along with closing its phase IIb study of another oral, small molecule, NYX-783, for treating post-traumatic stress disorder.

Once-monthly subcutaneous injections of CSL Ltd.’s CSL-312 (garadacimab) significantly reduced the rate of hereditary angioedema (HAE) attacks compared to placebo, meeting both primary and secondary endpoints in the pivotal phase III Vanguard trial. Based on the trial results, CSL will file global regulatory submissions later in 2023. Based on the trial results, CSL will file global regulatory submissions later in 2023. A humanized anti-factor XIIa monoclonal antibody, garadacimab is self-administered by subcutaneous administration once monthly, which is a huge convenience for patients, Andrew Nash, CSL’s chief scientific officer and senior vice president for research, told BioWorld.

An international team of researchers has created two bat stem cell lines that reveal an unusual number of viral sequences in bat cells compared with those of other mammals. Writing in an article posted online Feb. 21, 2023, in Cell, the scientists suggested that the unusual amount of viral genetic material found in the bat stem cells could explain why these mammals are largely unaffected by most viral infections, despite being able to transmit them.