Backed by fresh phase III data showing a combination of the IDH1 gene inhibitor Tibsovo (ivosidenib) and azacitidine significantly improved event-free survival and overall survival for adults with previously untreated IDH1-mutated acute myeloid leukemia (AML) vs. azacitidine alone, Cstone Pharmaceuticals Co. Ltd. is working to bring the treatment to Chinese AML patients “as soon as possible,” a spokesperson told BioWorld.
Protagonist Therapeutics Inc. continues having a tough time. The latest is a steep stock drop after a phase II trial in ulcerative colitis (UC) missed its primary endpoint, but the company insists the data are good enough to move into phase III. Top-line data showed a 450-mg BID dose of PN-943 in treating moderate to severe UC missed its primary endpoint, which was the number of participants achieving clinical remission at week 12 compared to placebo.
More than a quarter of all clinical data reported this year are targeting cancer indications, as pandemic news dips and overall activity drops by 12.5%.
Shares of Eliem Therapeutics Inc. fell 56% on the company’s announcement that it would end development of its non-opioid palmitoylethanolamide prodrug, ETX-810, in diabetic peripheral neuropathic pain following a phase IIa failure. It was part of a double dose of bad news reported by Seattle-based Eliem, which also is delaying phase II development of depression candidate ETX-155 to resolve issues of lower-than-expected drug exposure in a phase I study of the neuroactive steroid GABAA receptor positive allosteric modulator in photosensitive epilepsy.
After rolling out positive, preliminary phase I CAR natural killer cell data, Nkarta Inc. saw its stock (NASDAQ:NKTX) soar to $18.72, up $10.95, or 140.9%, as Wall Street made known its pleasure in results from the small, independent dose-finding studies with Nkarta’s two off-the-shelf lead candidates, NKX-101 and NKX-019, in two groups of blood cancer patients: those with relapsed/refractory (r/r) acute myeloid leukemia (AML) and with r/r non-Hodgkin lymphoma (NHL), respectively.
Isofol Medical AB’s slow reveal of data from its phase III registration trial of arfolitixorin in first-line metastatic colorectal cancer (mCRC) will not reach a conclusion until the fourth quarter of this year. The news follows a December 2021 warning to investors that an unexplained level of therapy switches occurring in the absence of disease progression would impair its analysis of a key secondary endpoint, the rate of progression-free survival.
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.
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