Regeneron/Sanofi PD-1 trial halted for significant efficacy; Avadel soars on positive narcolepsy data
→ It’s a good news, bad news day for Sanofi and Regeneron. On the same morning their joint Kevzara trial produced disappointing results for Covid-19, the biotech-pharma partners announced that a Phase III trial testing their immunotherapy Libtayo on non-small cell lung cancer had been stopped early after having shown clear benefit in improving overall survival. The PD-1 inhibitor reduced the risk of death by 32% in first-line patients.
→ Irish drug developer Avadel Pharmaceuticals‘ shares $AVDL shot up nearly 33% to $13.06 in Monday premarket trading, after the company unveiled positive late-stage data on its experimental drug, FT218, under evaluation as a treatment for excessive daytime sleepiness and cataplexy in patients with narcolepsy. The REST-ON trial met its three co-primary efficacy endpoints at all three doses, inducing statistically significant improvements on measures of wakefulness and mean weekly cataplexy (sudden muscular weakness triggered by strong emotions such as laughter, anger and surprise) attacks.
Akin to Jazz Pharmaceuticals‘ cash cow narcolepsy therapy Xyrem, FT218 is also a formulation of sodium oxybate. “(P)ositive FT218 data derisk approval from a clinical perspective, and also, with the caveats of cross-trial comparisons, suggest efficacy on par with Jazz’s Xyrem,” Stifel’s Paul Matteis wrote in a note.
→ Neurocrine Biosciences, which last year joined forces with Voyager Therapeutics to work on a range of gene therapies, has scored FDA approval for Ongentys, an add-on therapy designed to supplement standard Parkinson’s therapy levodopa/carbidopa.
As Parkinson’s disease progresses, first-line treatments such as levodopa begin to lose effectiveness and the beneficial effects of the drug begin to wear off more quickly, causing more frequent and often debilitating motor fluctuations in patients — Ongentys has shown to significantly reduce that “off” time, culminating in better and more consistent motor symptom control. The drug is set to launch later in 2020, the company said.
→ Takeda‘s experimental targeted cancer drug, mobocertinib, has secured the FDA’s breakthrough therapy status for patients with a form of non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy. “Although most EGFR mutations can be targeted by currently available TKIs (Tyrosine kinase inhibitors), people with exon 20 insertion mutations often suffer and feel forgotten since available EGFR inhibitors don’t work well in their cancer,” said Jill Feldman, lung cancer patient and co-founder of EGFR Resisters, in a statement.
→ First, Harpoon got a cool $50 million for allowing AbbVie the option to license its experimental drug, HPN217. Now, as the company kicks off a Phase I/II trial testing the therapy in relapsed, refractory multiple myeloma patients, the US drugmaker is forking over another $50 million.
→ China biotech Green Valley grabbed a significant amount of attention recently when it bagged an approval in its home country for an Alzheimer’s drug. Now the FDA is allowing their researchers to conduct a US test that will decide its fate outside of China. Give this one a long lead time, though. The company doesn’t expect to file before 2025, when all the data are in.