Amicus Therapeutics Inc.’s results from the phase III trial called Propel with AT-GAA (cipaglucosidase alfa and miglustat) for late-onset Pompe disease (LOPD) met with split opinions, though Wall Street took a decidedly dim view and left shares (NASDAQ:FOLD) to close at $12.57, down $6.16, or 33%.
Recently published findings in JAMA Psychiatry related to the sharply increased risk of death from COVID-19 in people with schizophrenia put the spotlight on drug development in the space, which has been steadily heating up the past few years.
Panbela Therapeutics Inc. hit a speed bump with its phase I trial in the ever-challenging indication of pancreatic cancer (PC), as an independent data safety monitoring board (DSMB) recommended that dosing be held for patients until more safety information is available about polyamine analog SBP-101.
Kalvista Pharmaceuticals Inc.’s chief development officer, Christopher Yea, said the across-the-board positive phase II top-line data with KVD-900 for on-demand use against hereditary angioedema (HAE) attacks will open “a very flexible discussion with regulators” regarding the design and endpoints of the phase III study to come with the oral kallikrein inhibitor. Shares of the Cambridge, Mass.-based firm (NASDAQ:KALV) closed at $33.50, up $17.89, or 115%, after reaching a high of $45, as Wall Street digested the findings.
CEO Mitchell Steiner said Veru Inc. at first downplayed the prospects of oncology candidate VERU-111 in COVID-19 treatment but now, with positive phase II data in hand, the company has a moral obligation to push onward to late-stage research.
In the wake of Study 045’s failure with Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy, PTC Therapeutics Inc. is “trying to thread the needle between the notion of getting Study 041 completed in the third quarter of 2022 vs. getting accelerated approval now,” CEO Stuart Peltz said.
Probably no one was surprised by the FDA’s recent nod for the combination of Alameda, Calif.-based Exelixis Inc.’s tyrosine kinase inhibitor, Cabometyx (cabozantinib), which targets MET, AXL and VEGF, and PD-1 drug Opdivo (nivolumab) from Bristol Myers Squibb Co. as a first-line treatment for advanced renal cell carcinoma (RCC), but investors may feel less confident about the odds for the pairing in a competitive space.
Questions about the price tag, the extent of diligence done on intellectual property, and the staying power of Epidiolex (cannabidiol) as well as the potential of other prospects in the acquiree’s pipeline bubbled up during the conference call related to Jazz Pharmaceuticals plc’s whopping takeover of GW Pharmaceuticals plc.
Less than a month after informing the market of a slowdown caused by pandemic-related site closures in phase II studies with neonatal Fc receptor (FcRn)-targeting IMVT-1401, Immunovant Inc. said it’s voluntarily pausing dosing with the compound in thyroid eye disease (TED) in the study called Ascend Go-2 because of a worrisome physiological signal. The New York-based firm plans to investigate and, after seeking the FDA’s go-ahead on tweaks to the program, forge onward with development.
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.
