Another data cut from Ultragenyx Pharmaceutical Inc. regarding UX-111 (rebisufligene etisparvovec), an AAV9 gene therapy for type A Sanfilippo syndrome, continued to brighten the picture for the compound, recently taken under review again by the U.S. FDA.
As the march toward a new therapy continues in metabolic dysfunction-associated steatohepatitis (MASH), new approaches are drawing Wall Street’s attention. Among them is Inventiva SA’s pan-PPAR approach.
More than four years after a phase II/III trial with Sanofi SA’s oral glucosylceramide synthase inhibitor venglustat was stopped for lack of benefit in autosomal dominant polycystic kidney disease, the company rolled out mixed phase III data from two more efforts.
Quince Therapeutics Inc. is scrapping work with dexamethasone sodium phosphate encapsulated in autologous erythrocytes (eDSP) for patients with ataxia-telangiectasia (A-T), and Boral analyst Jason Kolbert said “the core value driver has been effectively removed, and the equity appears fully impaired at this stage.”
The presbyopia space gained another player as Tenpoint Therapeutics Ltd. won the U.S. FDA’s go-ahead for Yuvezzi (carbachol and brimonidine tartrate ophthalmic solution, 2.75%/0.1%, previously known as Brimochol PF), the first and only dual-agent eye drop for the treatment of adult presbyopia.
With the PDUFA date fast approaching for Regenxbio Inc.’s gene therapy RGX-121, the U.S. FDA placed the drug on clinical hold along with another, RGX-111, after preliminary analysis of a single case of neoplasm (specifically, an intraventricular central nervous system tumor) in a participant treated in the phase I/II study with the latter treatment.
Cardiff Oncology Inc. welcomed Mani Mohindru as interim CEO and simultaneously provided what the company dubbed a “positive” but stock-denting clinical update. The firm rolled out data from the 113-patient phase II trial called CRDF-004, designed to test the oral Polo-like kinase 1 (PLK1) inhibitor onvansertib in first-line, RAS-mutated metastatic colorectal cancer (mCRC). Shares (NASDAQ:CRDF) closed Jan. 27 at $2, down 94 cents, or 32%, as Wall Street took in the results.
The structurally similar cytokines IL-2 and IL-15, and their shared beta subunit CD122, are keeping developers busy across a range of indications. Though some scientific confusion has plagued the space historically, drug candidates have drawn deals and Wall Street interest aplenty. Amgen Inc., Novartis AG, and Incyte Corp. are among those who’ve made their interest known.
CD122 – shared beta subunit of the IL-15 and IL-2 receptors, two targets for the figurative price of one – has drawn the eyes of many drug developers, among them Anaptysbio Inc., which will roll out phase Ib results in celiac disease during the fourth quarter of this year with ANB-033. The San Diego-based firm intends to weigh the candidate in a second inflammatory disease. Under consideration is eosinophilic esophagitis, an indication that has itself proven widely intriguing.
Corvus Pharmaceuticals Inc. rolled out new positive data to prove its thesis on the value of blocking IL-2-inducible T-cell kinase in atopic dermatitis (AD), and the company’s first-in-class approach “could shake up cancer and autoimmune disorders,” Wainwright analyst Swayampakula Ramakanth said when he started coverage Jan. 2.