Neuren Pharmaceuticals Ltd.’s NNZ-2591 met the primary endpoints in a phase II trial in children with Angelman syndrome, with improvements seen in clinically important aspects of the disease, including communication, behavior, cognition and motor abilities, Neuren CEO Jon Pilcher said during an Aug. 9 conference call. Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by a loss of function of the UBE3A gene in the maternal chromosome 15, and there are no approved treatments for AS despite its severely debilitating impact on the lives of patients, as well as their parents and siblings.

Aldeyra perseveres in phase III of dry eye disease

Aldeyra Therapeutics Inc. has bounced back from a complete response letter in November to produce positive phase III data for reproxalap in treating dry eye disease. Hitting the primary endpoint helped strengthen the stock (NASDAQ:ALDX), which opened the week at $3.25 per share and has climbed about 56% since. Shares at midday were going for $5.07, up 21%. Reproxalap, a reactive aldehyde species, or RASP, modulator was found to be statistically superior to vehicle for the prespecified primary endpoint of ocular discomfort (p=0.004). Aldeyra said this is the first positive phase III study in a dry eye chamber with the primary endpoint being a symptom.

EZH, PCR2 gathering steam; it’s more than just Novartis

Novartis AG in its July 18 second-quarter earnings call talked up tulmimetostat, the enhancer of zeste homolog (EZH) 1/EZH2 inhibitor for prostate cancer. The drug came to Basel, Switzerland-based Novartis by way of the February buyout of Morphosys AG, of Munich, for €2.7 billion (then US$2.9 billion). EZH is an enzymatic component of polycomb repressive complex 2, and a small handful of competitors with Novartis in the heating-up space have rolled out news recently.

GSK, Novartis lead July deals as biopharma sector sees more than $20B for the month

In July, biopharma deal values surged to $20.68 billion, a 61% increase from June's $12.82 billion. The boost pushed the total deal value for the first seven months of 2024 to $119.99 billion, marking the highest amount raised through July in BioWorld’s records. The 2024 monthly average for deals has now climbed to $17.14 billion, surpassing the $15.08 billion monthly average recorded during the same period in 2023.

(Modest) funding increases bring endometriosis discovery impetus

After decades of being woefully under-diagnosed and all but ignored by the biotech and pharma industry, recent advances in understanding its complex etiology could be opening the way to new treatments for endometriosis.

Also in the news

Actio, Aldeyra, Aligos, Amneal, Ani, Apellis, Astrazeneca, Aventis, Bristol Myers Squibb, Citius, Conduit, Deepcure, Evotec, Exscientia, Eyenovia, Formosa, Medigene, Merck, Novartis, Palisade, Pepromene, Precigen, Purdue, Recursion, Sanofi, Satellos, Sobi, Theramex, Westgene, Wuxi