Tonix Pharmaceuticals Holding Corp.’s failure last December in the phase III study called Recovery with TNX-102 SL (cyclobenzaprine HCl sublingual tablets) 5.6 mg dented hopes for patients with civilian and military-related post-traumatic stress disorder (PTSD), but other developers remain in the game.
Innate Pharma SA’s unexpected return to partner Astrazeneca plc all rights, in the U.S. and EU, to Lumoxiti (moxetumomab pasudotox-tdfk) for hairy cell leukemia likely triggered unease in at least some investors.
Phase II data with San Diego-based Heron Therapeutics Inc.’s pain drug HTX-011 (Zynrelef) graced the online pages of the Journal of the American Podiatric Medical Association in early January, whetting investor thirst for an FDA decision regarding approval, slated by May 12. And the agency’s go-ahead, if it comes, could have special meaning for Pacira Biosciences Inc. with competing analgesic Exparel (bupivacaine).
Boston-based Nirogy Therapeutics Inc.’s $16.5 million series A round is meant to enable a pipeline of small-molecule drugs targeting the solute carrier family of transporter proteins (SLCTs) embedded in the cell membrane, and let the firm bring its front oncology runner to the clinic in 2022.
Tscan Therapeutics Inc. CEO David Southwell told BioWorld that his firm’s series C financing of $100 million will allow two IND filings in liquid tumors this year and three – possibly more – in solid tumors starting next year. “We’ll be filing a lot of INDs in solid tumors,” he said. The Waltham, Mass.-based firm works with T-cell receptor-engineered T-cell therapies.
At the recent 39th J.P. Morgan Healthcare Conference, Biomarin Pharmaceutical Inc. popped the lid off top-line results from its ongoing phase III GENEr8-1 study with valoctocogene roxaparvovec – also known as valrox, now commonly called Roctavian. Data, though encouraging, may not have quelled controversy around the prospect.
With worrisome COVID-19 variants cropping up, developers including the likes of Gritstone Oncology Inc. and Vir Biotechnology Inc. continue their efforts to invent new vaccines that may get around the drawbacks of existing shots if they turn up.
KSQ Therapeutics Inc.’s chief scientific officer, Frank Stegmeier, said that the CRISPRomics technology that drew Takeda Pharmaceutical Co. Ltd. to the table allows, “for the first time, genome-scale functional screening [in vivo as well as in vitro] across multiple disease settings. It really takes the guessing game out of your drug target selection.”
Aclaris Therapeutics Inc. CEO Neal Walker said “a lot of room within rheumatoid arthritis [RA]” remains for new drugs, such as his firm’s orally delivered ATI-450. “We see opportunities given the profile, the relative efficacy and safety that we've already demonstrated, to look at not only potentially monotherapy or earlier treatment of disease but also combo treatment, particularly given the safety profile. I think it's pretty well-known that polypharmacy is the rule in this indication.”
With the table set for phase III data due next month from a trial testing Travere Therapeutics Inc.’s sparsentan against focal segmental glomerulosclerosis (FSGS), investor appetite grows ever sharper for prospects in kidney disease.
