Proqr Therapeutics NV stock lost three-quarters of its value as word got out that its pivotal phase II/III study of sepofarsen in treating a tough, rare and genetic retinal disease failed to hit the primary endpoint. CEO Daniel de Boer said he was “shocked by the unexpected outcome” based on data from earlier studies. He added that he is unsure if Proqr will continue developing its therapy for treating CEP290-mediated Leber congenital amaurosis 10 until it understands the new results. Read More
Delivering important but likely unwelcome news to Americans increasingly trying put the COVID-19 pandemic behind them, new research published Feb. 11 by the CDC suggested that "protection conferred by mRNA vaccines waned in the months after receipt of a third vaccine dose reinforces the importance of further consideration of additional doses to sustain or improve protection against COVID-19-associated [emergency department or urgent care (ED/UC)] encounters and COVID-19 hospitalizations." Read More
Cincor Pharma Inc.’s IPO at the start of the year underlined hopes for a new way to attack treatment-resistant hypertension and related diseases, as the Boston-based firm touted what one analyst called it ‘pipeline in a pill,” CIN-107, which selectively targets aldosterone synthase to lower aldosterone levels, in turn knocking down blood pressure. Read More
An increasing number and rising value of high-money biopharma deals has placed early 2022 above all recent years, even though there are fewer partnerships overall. Read More
In the bad old days of cancer treatment, it was the nasty side effects of chemotherapy that often ended up limiting treatment – and while checkpoint inhibitors have raised the bar in terms of efficacy and safety, they can have dangerous and unpleasant off-target consequences, too. Finding ways to focus the immune system on cancer and limiting any off-target effects will not only make checkpoint inhibitor therapy more tolerable but could also improve survival rates – and Sweden’s Ilya Pharma AB aims to do this with a novel approach combining synthetic biology with cell therapy. Read More
Biopharmas raising money in public or private financings, including: Adial, Aeglea, Alzecure, Enveric, Ortho Regenerative Technologies, TC, Ucello. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Biontech, Cassava, Edigene, Johnson, Legend, Metacrine, Moderna, Pfizer. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Akeso, Apex, Ardelyx, Bayer, GSK, Regeneron, Sanofi, Teva, Theragnostics. Read More
