Netherlands and U.S.-based biotech ProQR Therapeutics and Rhythm Pharmaceuticals completed public financings in the past week to support advancement of their experimental rare disease therapies.
ProQR raised $50 million in an underwritten public offering of 9.1 million shares at $5.50 a share. ProQR’s experimental RNA repair therapies target severe genetic rare diseases such as Leber’s congenital amaurosis 10, Usher syndrome type 2 and autosomal dominant retinitis pigmentosa.
ProQR recently received Rare Pediatric Disease designation for sepofarsen, its experimental treatment of Leber’s congenital amaurosis 10, the most common cause of blindness in children due to genetic disease.
Rhythm Pharmaceuticals raised $172.5 million in an underwritten public offering of 9.3 million shares at $18.50 a share. The company is developing therapies to treat rare genetic disorders of obesity.
Rhythm recently reported positive topline results from two pivotal, phase 3 clinical trials evaluating setmelanotide, the company’s melanocortin-4 receptor agonist in development for the treatment of pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities.
Setmelanotide activates MC4R, part of the key biological pathway that independently regulates hunger and body weight. Variants in genes within the MC4R pathway are associated with unrelenting hunger and severe, early-onset obesity.