The ReDUX4 study missed its biomarker-driven primary endpoint, making it unclear if an additional trial will be needed for regulatory submission.

Pharma and the US biotech will target key regulators of cellular metabolism with focus on immune and neurological disorders. Royalty Pharma buys portion of PTC’s royalty rights to risdiplam, under development at Roche.

Private Company Edition: In addition to Erasca’s big series B round, Mabwell reportedly brought in $278m. Also, Kurma, Venrock and Section bring the number of new VC funds in April to eight, raising $5.89bn, and former AveXis execs launched the new gene therapy venture Taysha.

Novartis's Jakavi joins the likes of Roche's Actemra in targeting the life-threatening immune reaction which is killing acutely ill COVID-19 patients.