Pfizer Inc. edged closer to Dermira Inc. in the rush for an approved treatment for atopic dermatitis (AD), a field dominated by Regeneron Pharmaceutical Inc.'s FDA-approved blockbuster, Dupixent (dupilumab), as Pfizer reported positive top-line results from a phase III trial evaluating the efficacy and safety of its oral JAK1 inhibitor, abrocitinib.

Top-line results from Enanta Pharmaceuticals Inc.'s Argon-1 phase IIa study of EDP-305 for the treatment of nonalcoholic steatohepatitis (NASH) found one of the two doses of the drug tested met the study's primary endpoint, alanine aminotransferase (ALT) reduction at week 12, contributing further evidence for the drug's mechanism of action, farnesoid X receptor (FXR) agonism. But the non-biopsy study appears to have left investors without proof for something more even more valuable: a clear case for differentiation vs. FXR first-mover Ocaliva (obeticholic acid, Intercept Pharmaceuticals Inc.). Appearing to reflect the sentiment, Enanta's shares (NASDAQ:ENTA) fell 15.1% to $60.51 on Thursday.

Ritter Pharmaceuticals Inc. is reeling from the phase III failure of its lead candidate, RP-G28, designed for patients with lactose intolerance. RP-G28 failed to demonstrate statistical significance in its primary endpoint as the top-line data showed it had no or little difference on patients compared to placebo. The data also show RP-G28 missed its secondary endpoints, all of which casts a shadow on the company and its pipeline.

Glaxosmithkline plc has won FDA approval to market Nucala (mepolizumab) for use in children as young as 6 with severe eosinophilic asthma (EA). The therapy already had FDA approval as an add-on maintenance treatment for kids with the same condition ages 12 and older. Approval of the sBLA, submitted last November, catches the U.S. market up to the EU, where Nucala has been approved as an add-on treatment for children ages 6 to 17 since August 2018.

Shares of San Diego-based Tocagen Inc. (NASDAQ:TOCA) fell 77.7% to 93 cents Thursday after its two-part immunotherapy for people with recurrent brain cancer failed to surpass standard of care on overall survival (OS), the primary endpoint of the company's phase III Toca 5 trial. Secondary endpoints in the registrational study were also missed, showing no meaningful difference between study arms.

BOSTON – The gut microbiome and its prospects for drug development have been matters of debate for a while, sharpened by the high-profile phase II failure of Seres Therapeutics Inc.'s candidate, SER-109, in the summer of 2016. A panel at Biopharm America surveyed the space in light of developments since the stumble with that candidate, composed of about 50 species of firmicutes spores derived from stool specimens from healthy donors, against recurrent Clostridium difficile infection. 

Aveo Oncology Inc. is moving closer to a potential NDA filing for its renal cell carcinoma (RCC) drug, tivozanib, following a recently conducted analysis of the ongoing phase III trial Tivo-3. Updated results evidenced "durable improvements" for study participants, all of whom have refractory metastatic RCC, said primary investigator Brian Rini. Company shares (NASDAQ:AVEO) climbed 30.9% to 92 cents Tuesday as Aveo said it would discuss the data with the FDA, which rejected the company's first attempt at approval in RCC in the summer of 2013.

Good news from San Diego-based Acadia Pharmaceuticals Inc.'s phase III trial with Nuplazid (pimavanserin) in dementia-related psychosis (DRP) had analysts trotting out music metaphors in reports about the study called Harmony, while talking already about would-be sales of the drug, a selective serotonin inverse agonist and antagonist that preferentially targets the 5-HT2A receptor.

Shares of New York-based Neurotrope Inc. (NASDAQ:NTRP) fell 77.3% to close at a record low of $1 Monday after a phase II study of its lead candidate, bryostatin-1, failed to outperform a placebo in helping people with moderate to severe Alzheimer's disease (AD) achieve improvement on a standardized measure of cognition.

As Wall Street ponders how pricing and reimbursement for gene therapies might shake out, companies in the field march intrepidly on, among them Orchard Therapeutics Inc., which early this month rolled out heartening results from an ongoing proof-of-concept trial evaluating the safety and efficacy of OTL-203 for mucopolysaccharidosis type I (MPS-I), a space that's heating up.