Zynerba Pharmaceuticals Inc. in late May popped the lid off top-line data from the open-label phase II study called Bright with ZYN-002 in children and adolescents with autism spectrum disorder, and the findings drew adjectives from Wall Street such as “provocative” and “encouraging.”
Nan Fung Life Sciences is “looking to make a significant presence in the field,” said Engrail Therapeutics Inc. CEO Vikram Sudarsan, whose firm bagged a $32 million series A round led by the global investment platform of Hong Kong’s Nan Fung Group, and its support represents “a clear statement in that direction.”
About six months after Epizyme Inc. won FDA clearance of Tazverik (tazemetostat) for epithelial sarcoma (ES), the firm scored accelerated approval in the larger indication of relapsed or refractory (r/r) follicular lymphoma (FL). Specifically, U.S. regulators cleared the methyltransferase inhibitor for adults whose tumors test positive for an EZH2 mutation who have received at least two prior systemic therapies and for those with no satisfactory alternative treatment options – language that “gives physicians a lot of flexibility to use their clinical judgement in how to best prescribe” the drug, CEO Robert Bazemore noted.
Neurocrine Biosciences Inc.’s chief business development and strategy officer, Kyle Gano, said “there was really no playbook” for the deal in which his firm is paying Takeda Pharmaceutical Co. Ltd. $120 million up front for an exclusive license to seven programs, including three clinical-stage assets targeting the notoriously difficult indications of schizophrenia and treatment-resistant depression (TRD), as well as depression-related anhedonia.
As expected, Viela Bio Inc. won FDA clearance for the humanized anti-CD19 monoclonal antibody Uplizna (inebilizumab-cdon) to treat adults with neuromyelitis optica spectrum disorder (NMOSD), a rare neuroinflammatory disease.
As the company gears up for two NDA filings this year, Reata Pharmaceuticals Inc.’s $350 million deal with funds managed by Blackstone Life Sciences provides cash runway through the end of 2023.
San Diego-based Autobahn Therapeutics Inc.’s $76 million series B round will let the firm advance lead candidate ABX-002, a thyroid hormone receptor beta agonist therapy for multiple sclerosis and adrenomyeloneuropathy, a rare genetic disorder, plus a portfolio of central nervous system programs that leverage the company’s brain-targeting chemistry platform.
Novartis AG didn’t say why the FDA has put off action until September – a delay of three months – on the sBLA for multiple sclerosis (MS) prospect Arzerra (ofatumumab, OMB-157), first cleared in October 2009 for chronic lymphocytic leukemia, but the holdup brought renewed attention to the bustling space, and Immunic Inc. – which held its R&D Day on May 27 – is coming on strong.
A key opinion leader (KOL) consulted by SVB Leerink deemed Roche Holding AG’s anti-TIGIT drug results “really huge” and “quite amazing,” affirming the Basel, Switzerland-based firm’s lead in the field. The phase II data from the study known as Cityscape rolled out at the virtual American Society of Clinical Oncology (ASCO) meeting.
Gone from the American Society of Clinical Oncology (ASCO) meeting were the packed halls of McCormick Place in Chicago and the excited bustle of scientists reacquainting with each other at the always data-rich event. ASCO’s annual confab, held online because of the COVID-19 pandemic, still managed to pull off what former president Howard Burris called a “resounding success,” with about 40,000 registrants, similar to previous years.
