Despite clinical responses undermining expectations for the placebo arm of its phase III Alzheimer's disease (AD) study, Lucidity, Taurx Pharmaceuticals Ltd. executives Oct. 6 said they still see the data as supporting their ability to pursue regulatory submissions for hydromethylthionine mesylate (HMTM), an oral tau aggregation inhibitor. For people with mild cognitive impairment especially, they said, "HMTM treatment resulted in sustained improvement in cognition over pretreatment baseline, and normalization of brain atrophy to a rate similar to healthy individuals."

Connect Biopharma Holding Ltd.’s IL-4Rα binding candidate, CBP-201, yielded positive data in a pivotal trial in patients with moderate to severe atopic dermatitis in China, positioning the company to engage with Chinese regulators in the next several months to discuss a potential NDA filing.

Biocryst Pharmaceuticals Inc. caught some industry observers by surprise early in 2022 when it upped its peak sales estimate for Orladeyo (berotralstat), its oral therapy for prevention of hereditary angioedema attacks, from $500 million to $1 billion. But blockbuster status appears more attainable following the latest setback from a potential competitor, as safety concerns prompted Kalvista Pharmaceuticals Inc. management to ax its phase II study testing KVD-824 in prevention of HAE attacks.

X4 Pharmaceuticals Inc.’s recent stock-boosting phase Ib news with lead candidate mavorixafor fueled already-strong interest in finding an alternative therapy for neutropenia and deepened esteem for the drug’s long-known mechanism of action.

Paradigm Biopharmaceuticals Ltd. reported top-line data from its phase II trial of Zilosul (pentosan polysulfate/PPS) that suggest the non-opioid treatment for knee osteoarthritis is effective in navigating pain management but also has the potential to be disease modifying.

Analysts were upbeat about positive results from Eisai Co Ltd. and Biogen Inc.’s phase III confirmatory Clarity Alzheimer’s disease (AD) trial of lecanemab that met both primary and secondary endpoints, and most agreed that the data should be enough to gain FDA approval for mild cognitive impairment due to AD.

Trefoil Therapeutics Inc. has reported promising data from a phase II trial suggesting its engineered version of fibroblast growth factor 1, TTHX-1114, may speed and improve recovery of patients with Fuchs endothelial corneal dystrophy who are undergoing a surgical procedure called Descemet stripping only.

Analysts were upbeat about positive results from Eisai Co Ltd. and Biogen Inc.’s phase III confirmatory Clarity Alzheimer’s disease (AD) trial of lecanemab that met both primary and secondary endpoints, and most agreed that the data should be enough to gain FDA approval for mild cognitive impairment due to AD.

A serious adverse event (SAE) in one participant has led the U.S. FDA to place a partial clinical hold on Avidity Biosciences Inc.’s lead program. The action is centered on the phase I/II Marina study of AOC-1001, an antibody oligonucleotide conjugate for treating myotonic dystrophy type 1, the most common form of muscular dystrophy in adults.