DMD drug developers stake out territory in rare disease market
This article was originally published in Scrip
Parents of boys with Duchenne muscular dystrophy (DMD) have had little good news during the past few months due to setbacks for Prosensa and Sarepta Therapeutics, but three different biotechnology company presentations at the 32nd Annual JP Morgan Healthcare Conference offered signs of hope for therapies that may keep their sons from losing the ability to walk, eat and breathe on their own.
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Several companies have DMD drug candidates waiting in the wings to follow Sarepta’s Exondys 51 into the commercial market after FDA approval of the exon-skipping therapy, which could be the backbone for future combination treatment regimens.
While Amgen said the Phase III GALACTIC-HF results for omecamtiv mecarbil did not meet its high expectations for the heart failure candidate, Cytokinetics sees a path to treating certain patients.
Olema launched this year’s 76th US biopharma IPO. Also, investments in regenerative medicines reached a new high, Qiming closed a $1.2bn China-focused VC fund, D3 Bio led recent venture deals with a $200m round, and Bausch sold $2bn worth of notes to pay down near-term debts.