After nearly a decade of labor, PIN Pharma Inc. reported success in its first-in-human, open-label, repeat-dose trial of immunomodulating precision immune stimulant (PIN)-2 in participants with advanced solid tumors. The New York-based company said rapid onset of action was signaled by a surge in circulating tumor necrosis factor (TNF)-alpha six hours after injection of PIN-2, a derivative of the HIV-1 Tat protein that activates monocyte-derived dendritic cells. Circulating TNF-alpha levels subsequently returned to baseline. A similar but more pronounced finding was observed during a second cycle of treatment, confirming proof of mechanism for the immunomodulatory effect of PIN-2 and corroborating results seen in human monocytes in vitro.
In many ways, 2018 resembled the previous year in fast motion. Political turmoil accelerated on both sides of the Atlantic, with Asia swept into the fold, while the “estrangement" previously seen between President Donald Trump and the scientific community morphed into global revulsion against a rogue research rule-breaker. Not all was well in biopharma-land, either. The groundswell of anger against rising U.S. drug prices looked likely to spill into multiple pieces of legislation in a rebalanced Congress. And the ongoing tragedy of opioid abuse and addiction prompted the FDA to put the brakes on certain drugs while the CDC warned...
Alexion Pharmaceuticals Inc. is set to go to market with Ultomiris (ALXN-1210, ravulizumab), the long-acting C5 complement inhibitor approved by the FDA Friday nearly two months ahead of its Feb. 18, 2019, PDUFA date to treat adults with the ultra-rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH).
Gilead Sciences Inc. placed another big bet in fibrosis, inking a preclinical deal with Scholar Rock Holding Corp. that includes $80 million up front – $50 million in cash and $30 million in Scholar Rock common shares – along with a one-time milestone payment of $25 million when certain preclinical studies are completed.
In a second first in the same week for China, roxadustat (previously FG-4592), developed by Fibrogen Inc. and partnered in the country with Astrazeneca plc, became the first drug advanced by a global biopharma to receive its initial nod from the National Medical Products Administration (NMPA, formerly the CFDA) before applications were submitted to the FDA and EMA.
After a courtship of more than a year, Tetra Discovery Partners LLC and Shionogi & Co. Ltd. tied the knot to develop and commercialize BPN-14770, Tetra's phosphodiesterase 4D (PDE4D) allosteric inhibitor, for the treatment of fragile X syndrome (FXS), Alzheimer's disease (AD) and other indications marked by cognitive and memory deficits.
Shares of Danish biotech Saniona AB (STOCKHOLM:SANION) closed at SEK36.65 (US$4.05) for a gain of SEK8.95 (US$.99), or 32.3 percent, Monday after partner Productos Medix S.A. de C.V. reported statistically and clinically significant weight loss for both doses of tesofensine compared to placebo in the phase III VIKING study in obesity. Medix will use the findings, which included good tolerability with a low incidence of adverse events, for filings in Mexico and Argentina, where it owns commercial rights.
Applied Genetic Technologies Corp. (AGTC) dropped two pieces of bad news following Wednesday's market close. The Gainesville, Fla.-based company said it was halting development of rAAV2tYF-CB-hRS1, its adeno-associated virus (AAV)-based RS1 gene therapy for X-linked retinoschisis (XLRS), after top-line interim data from its phase I/II trial showed no sign of clinical activity at six months. AGTC also revealed that Biogen Inc. was walking away from their three-year-old, potential $1 billion collaboration and returning full rights to the clinical-stage XLRS and X-linked retinitis pigmentosa (XLRP) programs and discovery programs in adrenoleukodystrophy (ALD) and two ophthalmic targets, effective March 8, 2019.
A somber Kevin Gorman was brutally honest with analysts Wednesday, advising them to remove Tourette syndrome (TS) from their Neurocrine Biosciences Inc. model after Ingrezza (valbenazine) missed the primary endpoint of change from baseline to week 12 in Yale Global Tic Severity Scale (YGTSS) score among children and adolescents with moderate to severe symptoms enrolled in the phase IIb T-Force GOLD study.
