Argenx SE’s top-line results from the phase II Adhere study with Vyvgart Hytrulo (efgartigimod alfa plus hyaluronidase-qvfc) in chronic inflammatory demyelinating polyneuropathy (CIDP) sparked buzz about other developers in the space with similar mechanisms of action to Vyvgart Hytrulo, such as Immunovant Inc.
Apellis Pharmaceutical Inc.’s shares nosedived by 37% July 17 after the American Society of Retina Specialists wrote to ophthalmologists warning them of the risk of serious intraocular inflammation events in patients following injection of the company’s geographic atrophy drug, Syfovre (pegcetacoplan).
Strong and complete phase III results for Eli Lilly and Co.’s donanemab for treating early Alzheimer's disease (AD) will no doubt inspire more comparisons with recently approved Leqembi (lecanemab). The newly released data for donanemab show it significantly slowed cognitive and functional decline for those with amyloid-positive early symptomatic AD, which lowered the disease-progression risk.
Camurus AB’s subcutaneous octreotide hydrochloride injection pen, CAM-2029, yielded statistically significant improvements in “multiple endpoints” in a 52-week phase III extension trial in patients with acromegaly, moving it closer to providing a more convenient treatment option for patients with the rare, chronic growth disorder than currently available therapies.
While the 12-month data from Bridgebio Pharma Inc.’s phase III ATTRibute-CM study testing acoramidis in transthyretin amyloid cardiomyopathy were deemed “puzzling” in late 2021, the 30-month results reported July 17 proved far more pleasing to investors. The trial hit all its endpoints and showed promising results on exploratory markers, sending company shares (NASDAQ:BBIO) up 76%, to close at $32.04, up $13.82.
Dose-limiting toxicities in a phase I/II study led Theseus Pharmaceuticals Inc. to quit work with lead compound THE-630, a pan-variant KIT Inhibitor for gastrointestinal stromal tumors (GIST), but the company plans to nominate another such candidate in the same indication during the first half of 2024. Meanwhile, the firm is prioritizing THE-349, a fourth-generation EGFR inhibitor for non-small-cell lung cancer, due for an IND application in the fourth quarter of this year.
Although shares of TG Therapeutics Inc. took a hit on word of positive phase III data in multiple sclerosis (MS) from competitor Genentech, a unit of Roche Holding AG, the results may not end up affecting much the market showdown between the CD20 players.
First Wave Biopharma Inc. is taking apart the top-line data from its phase II study of adrulipase for treating nutrition deficiencies in cystic fibrosis patients and seems unsure exactly what it found. The company said that while the initial data found the enhanced enteric microgranule delivery formulation of adrulipase was safe, well-tolerated and was an improvement over other formulations, the preliminary data also indicate that “it is likely the primary efficacy endpoint was not achieved.”
Nodthera Ltd. claims to be first to demonstrate it is possible to modulate the NLRP3 inflammasome in the brain, after showing there were reductions in inflammatory and disease-specific biomarkers in blood and cerebrospinal fluid after seven days of daily administration of its lead product, NT-0796, an oral NLRP3 inhibitor.
As investors continue to weigh VRDN-001’s chances against Tepezza (teprotumumab, Horizon Pharmaceuticals plc) in thyroid eye disease, Viridian Therapeutics Inc. popped the lid off positive preliminary data from the ongoing phase I/II trial with the intravenous full antagonist antibody to the insulin-like growth factor 1 receptor.
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