With efficacy that hit the primary endpoint but fell below what Wall Street watched for, Biocryst Pharmaceuticals Inc. hopes a sampling program and BCX-7353's oral route will help gain market share for the once-daily kallikrein inhibitor to prevent hereditary angioedema (HAE).
"This is just a tiny seed round," Detlev Mennerich, of Boehringer Ingelheim Venture Fund (BIVF), conceded, telling BioWorld however that backers of Rinri Therapeutics Ltd. "have a clear vision" how the firm can use its newly banked £1.4 million (US$1.7 million) to develop chemistry, manufacturing and controls before going back to the market in a year or so for "north of $10 million" in series A money and then taking aim at phase Ib/IIa work.
Cytokinetics Inc. CEO Robert Blum said it's "highly unlikely" that the phase III trial planned with fast skeletal muscle troponin activator reldesemtiv, also known as CK-2127107, will begin this year, "just knowing what the tasks in front of us are." But the South San Francisco-based firm, with partner Astellas Inc., of Tokyo, is moving ahead with the candidate in amyotrophic lateral sclerosis (ALS) despite results from the Fortitude-ALS trial showing that the drug missed statistical significance for the primary efficacy analysis after 12 weeks of treatment.
With abstracts presaging the customary avalanche of data due from the American Society of Clinical Oncology (ASCO) meeting next month, the mechanism of KRAS inhibition caused an expectedly large share of talk, though efforts remain far from mature, and too fresh for big conclusions.
Kymera Therapeutics Inc. CEO Laurent Audoly told BioWorld that his firm's deal with Vertex Pharmaceuticals Inc. "signals to the sector how protein degradation is broadly deployable across many diseases. We're going way beyond oncology and immunology."
As expected, Tokyo-based Daiichi Sankyo Co. Ltd. faced rough sledding in its review by the FDA's Oncologic Drugs Advisory Committee (ODAC) of quizartinib tablets for adults with relapsed or refractory acute myeloid leukemia (AML) that is FMS-like tyrosine kinase (FLT) 3-internal tandem duplication (ITD) positive, as detected by an FDA-approved test.
Novartis AG is buying from Takeda Pharmaceutical Co. Ltd. the dry eye therapy Xiidra (lifitegrast ophthalmic solution) for a $3.4 billion up-front payment, with up to $1.9 billion more if milestones are met, and the Basel, Switzerland-based firm is bringing aboard about 400 employees – mostly based in the U.S. and Canada – associated with the drug, which was approved by the FDA in the summer of 2016.
Elevatebio Inc. CEO David Hallal told BioWorld that the five-member syndicate of investors behind his firm's whopper $150 million series A round "all have been on the other side of the table" in cell and gene therapy, and thus understand the potential value of building a portfolio of new companies in both areas by way of partnerships with academic researchers, medical centers and entrepreneurs.
Although some on Wall Street kept faith that the hobbled shares of Fibrogen Inc. will make up their losses as investors pick through the data, pundits meanwhile sought to unriddle the pooled phase III findings offered from the global program with roxadustat, an inhibitor of hypoxia-inducible-factor (HIF) prolyl hydroxylase activity.
