Sanofi SA’s high-profile phase III blow-up Sept. 9 with the oral BTK inhibitor rilzabrutinib in pemphigus brought fresh attention to the group of rare diseases – which cause blisters on the skin and mucous membranes throughout the body – and to players pushing for a new treatment.
Biogen Inc. and Samsung Bioepis Co. Ltd. gained FDA clearance for Byooviz (ranibizumab-nuna), a biosimilar that references the VEGF therapy Lucentis (ranibizumab) from Roche Holding AG, as a treatment for wet age-related macular degeneration, macular edema following retinal vein occlusion and myopic choroidal neovascularization. Byooviz is the first ophthalmology biosimilar to win the go-ahead in the U.S., and was approved in the EU on Aug. 18, 2021, followed by the U.K. on Aug. 31, 2021.
Aerie Pharmaceuticals Inc.’s wide-net endpoint approach with the phase IIb study called Comet-1 seemed less than appreciated by the stock market as the firm unveiled top-line data in dry eye disease with AR-15512.
The market’s appetite for immunology candidates in chronic disease – in particular an oral interleukin-17 (IL-17) therapy for psoriasis – was proved by the upsized IPO pulled off by Dice Therapeutics Inc., and Tyra Biosciences Inc. benefited from the ongoing appetite in precision oncology in another, bigger-than-expected debut. Preclinical-stage Pasithea Therapeutics Inc. went public as well, albeit less spectacularly.
Apellis Pharmaceuticals Inc.’s win with one phase III trial and narrow miss with an identical one testing pegcetacoplan in geographic atrophy secondary to age-related macular degeneration caused Wall Street to punish the company while rewarding competitor Iveric Bio Inc.
Lucrative partnerships lately in the targeted protein degradation (TPD) space have shone an even brighter light on the already-intriguing approach, and among the firms to spark enthusiasm is Monte Rosa Therapeutics Inc., pursuing programs aimed solely at drugs that deploy molecular “glue” – an E3 ligase enzyme affixed to a target substrate.
Proqr Therapeutics NV sealed a deal for genetic disorders in the liver and nervous system with Eli Lilly and Co., collecting $50 million in the form of an up-front payment ($20 million) and an equity investment ($30 million), with the prospect of about $1.25 billion more if the arrangement hits research, development and commercialization goals. “The milestones are pretty evenly spread out,” said Smital Shah, Proqr’s chief business and financial officer, though details weren’t provided.
Attralus Inc.’s $116 million series B will advance pan-amyloid removal (PAR) therapeutics, putting lead prospect AT-03 into a phase I biodistribution study soon in systemic amyloidosis patients while advancing other programs, including AT-01, an amyloid-specific imaging radiotracer for diagnosis in the same indication.
The phase III fizzle in autism spectrum disorder (ASD) by French firms Les Laboratoires Servier SAS and Neurochlore SAS was the latest in a disease that has proved challenging because of its heterogeneity, which – in the words of Yehezkel Ben-Ari, president of Neurochlore – “probably makes it impossible to offer a sole treatment for all autistic children.”
“Now it’s all about time to market,” Bavarian Nordic A/S CEO Paul Chaplin said as the company disclosed positive results from the phase IIa study with its respiratory syncytial virus (RSV) vaccine, MVA-BN RSV. “We’re looking at designs [for phase III] that give us the fastest approval possible” in the competitive space, he said, adding that the previously envisioned two-season setup “has gone out the window” with the latest findings.
