Shares of Corvus Pharmaceuticals Inc. (NASDAQ:CRVS) sank 21.5% on Sept. 7, even though the company confirmed a phase III plan with the U.S. FDA for its ITK inhibitor, soquelitinib, to treat relapsed peripheral T-cell lymphoma (PTCL).
While an “unexpected placebo effect” marred its primary endpoint, the pivotal phase II/III study testing AMO-02 (tideglusib) showed clinically significant benefits across a range of functional and objective assessments, according to developer Amo Pharma Ltd., which is prepping to meet with regulators to discuss potential approval for use in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), an ultra-rare subtype of myotonic dystrophy type 1 for which no treatment options are available.
Wall Street’s hoped-for phase III derisking event from Insmed Inc. materialized, and shares of the firm (NASDAQ:INSM) closed Sept. 5 at $26.37, up $3.73, or 16.5%, on positive top-line results from the study called Arise with inhaled Arikayce (amikacin) in patients with newly diagnosed or recurrent nontuberculous mycobacterial lung infection by Mycobacterium avium complex (MAC) who had not started antibiotics. “We crushed it” on culture conversion with Arikayce, CEO William Lewis said. “We could not be happier about the results of this study. It exceeded all of our expectations on every front.”
Roche Holding AG’s Genentech subsidiary has broken new ground with a victory in phase III testing of the oral, anaplastic lymphoma kinase (ALK) inhibitor Alecensa (alectinib) in early stage, ALK-positive non-small-cell lung cancer (NSCLC). The drug, well known to doctors in the advanced setting, was compared with platinum-based chemotherapy and met its primary endpoint of disease-free survival at a prespecified interim analysis.
For many multiple sclerosis patients, the approval over the past 30 years of a lengthy list of immunomodulatory therapies has helped to reduce the frequency of relapses and to slow disease progression. However, there has been little parallel progress in the development of remyelination therapies, to tackle the other key pathophysiological dimension of the disease. Patients still have no therapies that can help to repair at least some of the damage that results from flare-ups, and the resulting neuronal loss contributes to further disease progression and disability. Rewind Therapeutics NV, of Leuven, Belgium, is one of a small clutch of firms attempting to tackle this problem.
Genexine Co. Ltd.’s recombinant human growth hormone eftansomatropin alfa (GX-H9/TJ-101) met the primary endpoint in a phase III pediatric study conducted in China in children with growth hormone deficiency, and the company plans to file a BLA in 2024 in China on the data.
Blood biomarkers have been found in patients hospitalized with acute COVID-19 that are predictive of the cognitive defects of long COVID. Post COVID-19 deficits in cognition, including brain fog, are common and debilitating. They are also clinically complex, with both objective and subjective components. In the U.K., one in eight patients received their first ever neurological or psychiatric diagnosis within six months following COVID-19.
Phathom Pharmaceuticals Inc. made recent headway with its new mechanism of action in gastroesophageal reflux disease (GERD) even as further, disturbing discoveries in the space are seeing daylight. Florham Park, N.J.-based Phathom turned over to the U.S. FDA the requested six-month stability data related to reformulated vonoprazan tablets, and the numbers remained consistent with three-month data, reflecting N-nitroso-vonoprazan nitrosamine control and levels comfortably more than tenfold below the acceptable daily intake limit.
The paper published June 19 in Nature Genetics that described a genome-wide analysis to narrow down the implicated pathogenic signaling pathways and “prioritize drug targets for IgA nephropathy [IgAN]” no doubt proved of great interest to developers, plenty of which are busy in the space.
Heterozygous familial hypercholesterolemia (HeFH) patients treated with lerodalcibep achieved a 58.6% reduction in LDL-cholesterol at week 24 and a 65% reduction at the mean of weeks 22 and 24 in the phase III trial Liberate-HeFH. The developer, Cincinnati-based Lib Therapeutics Inc., was founded in 2015 when it licensed the technology from Bristol Myers Squibb Co. but is just now emerging from stealth.
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