The majority of patients with acromegaly who switched from therapy with injected somatostatin receptor ligands to Crinetics Pharmaceuticals Inc.’s once-daily, oral drug paltusotine maintained stable levels of insulin-like growth factor 1 in a phase III trial, moving the company’s first-in-class lead closer to becoming the potential new standard of care for the rare hormonal disorder.

Daiichi Sankyo Co Ltd.’s HER3-directed antibody-drug conjugate patritumab deruxtecan showed clinically meaningful and durable responses in patients with EGFR-mutated locally advanced or metastatic non-small-cell lung cancer (NSCLC) in the Herthena-Lung1 phase II trial.

Positive phase III data from Cymabay Therapeutics Inc. with seladelpar, the peroxisome proliferator-activated receptor for primary biliary cholangitis (PBC), could mean trouble for Intercept Pharmaceuticals Inc.’s second-line therapy, the farnesoid X receptor agonist Ocaliva (obeticholic acid), which Cymabay aims to replace with its compound as the preferred choice.

Brii Biosciences Ltd.’s therapeutic vaccine for hepatitis B (HBV), BRII-179 (VBI-2601), induced functional immune responses – inducing broad antibody and T-cell responses – in patients with chronic HBV in a phase II trial that evaluated the vaccine in combination with pegylated interferon-alfa, according to interim results.

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.”

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.

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.