After August 2021 news from the U.S. FDA of deficiencies in the NDA that Axsome Therapeutics Inc. submitted for AXS-05 in major depressive disorder (MDD), word was especially welcome April 19 that the firm has come to an agreement with the agency with regard to post-marketing requirements. Meanwhile, another player in MDD, Relmada Therapeutics Inc., is due to report phase III data with REL-1017 (esmethadone) around the middle of this year.
By January 2022, 12 billion COVID-19 vaccine doses had been manufactured and, to date, roughly 20 vaccines have received either full approval or emergency use authorization in countries around the world. In places such as Europe and the U.S., most of the people who want vaccinations have gotten their jabs. And the vaccines continue to show efficacy, with the latest CDC data showing consistently less risk for testing positive for COVID-19 or dying from the infection for those who are vaccinated and boostered. Yet the development of new COVID-19 vaccines remains surprisingly robust, with BioWorld tracking 258, the majority of which are in preclinical development.
Alexion Pharmaceuticals Inc. has demonstrated the commercial potential for rare disease drugs with its complement inhibitor Soliris (eculizumab) and long-lasting follow-up Ultomiris (ravulizumab) driving blockbuster sales. A host of other companies are hoping to compete with Ultomiris, which is U.S. FDA-approved for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Astrazeneca plc led the handful of firms rolling out COVID-19-related news, as the company said detailed results from the Provent phase III pre-exposure prophylaxis (prevention) trial with Evusheld (tixagevimab and cilgavimab), were published in the New England Journal of Medicine, the “culmination of about 19 months of pretty intensive work,” said Mark Esser, vice president of microbial sciences.
Privately held Orasis Pharmaceuticals Ltd. is a step closer to challenging Abbvie Inc.’s Vuity (pilocarpine hydrochloride) ophthalmic solution for treating presbyopia, a version of farsightedness. Orasis plans to submit an NDA to the U.S. FDA in the second half of the year based on phase III results from two studies showing CSF-1 hit its primary and secondary endpoints.
Windtree Therapeutics Inc.’s positive top-line data from the phase II study called Seismic, testing istaroxime in cardiogenic shock (CS), a form of sudden heart failure (HF), add “a lot of interesting strategies and options” with the compound, also in development for acute HF, said CEO Craig Fraser.
Arcturus Therapeutics Holdings Inc. reported that ARCT-154, its self-amplifying mRNA COVID-19 vaccine, showed efficacy of 55% against infection and 95% efficacy against severe disease, meeting the primary and key secondary endpoints of the ongoing phase I/II/III trial. While the company’s stock (NASDAQ:ARCT) regained much of its initial 25% drop to close the day, investors continue to await further data to determine where Arcturus’ vaccine will fit in with available COVID-19 vaccines.
China’s Ministry of Science and Technology issued a draft rule on regulations governing sharing of human genetic resources that provides clarity on parts of the regulation that were previously ambiguous, Katherine Wang, partner at Ropes & Gray in Shanghai, told BioWorld.
With phase III data due from Karuna Therapeutics Inc. with its combo Karxt therapy for schizophrenia, interest is rising in the historically difficult space. Karxt pairs xanomeline, a muscarinic receptor agonist that preferentially stimulates M1 and M4 muscarinic receptors, with trospium, an approved muscarinic receptor antagonist that does not measurably cross the blood-brain barrier, confining its effects to peripheral tissues.
Shionogi & Co. Ltd.’s plan to make ensitrelvir, an oral antiviral for COVID-19, the first domestically approved COVID-19 treatment has hit a snag. Animal studies showed the drug, also known as S-217622, disturbed fetal development, according to media reports.
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