DUBLIN – Shares in PTC Therapeutics Inc. dropped as much as 13% during premarket trading Feb. 6 on eagerly awaited 12-month data for its oral spinal muscular atrophy (SMA) drug risdiplam, which is licensed to Genentech. Although patients with type 2 or type 3 SMA demonstrated statistically significant improvements in motor function, the level of that improvement evidently disappointed some investors.
DUBLIN – Azafaros BV raised €25 million (US$27.5 million) to develop orally available azasugar drugs with potential application to multiple lysosomal storage diseases.
DUBLIN – Maat Pharma SA closed an €18 million (US$19.9 million) series B round to continue development of its standardized, off-the-shelf fecal microbiota transplant (FMT) therapy, MaaT-013.
DUBLIN – The witty Twitter account @justsaysinmice, run by Northeastern University research scientist Jim Heathers, offers a very useful corrective to the misleading and unwarranted hype that often accompanies preclinical studies in mice. What looks good in murine models is all too often lost in translation, for a whole host of reasons, and never has any useful effect in patients. That’s not a concern for a group led by Thomas Thum, of the Institute of Molecular and Translational Therapeutic Strategies at Hannover Medical School in Germany, who just published in Nature Communications the outcome of what is probably the largest ever pig study in heart failure.
DUBLIN – Bergenbio ASA raised NOK219.9 million (US$23.9 million) in a private placement priced at NOK18 per share. The fresh injection of cash will enable the Bergen, Norway-based firm to expand its broad development program for lead drug candidate bemcentinib (BGB-324), a first-in-class Axl inhibitor, which is currently in phase II development in acute myeloid leukemia (AML), non-small-cell lung cancer (NSCLC), triple-negative breast cancer and melanoma.
DUBLIN – “Welcome to the conversation,” Abbvie Inc.’s head of genomic research, Howard Jacob, an early pioneer of genomics-driven medicine, told delegates during a keynote address at the Genomics Summit 2020 event Jan. 23. Ireland is very much a latecomer to that conversation and it has yet, as a country, to figure out what the shape of its contribution to the genomic era of medicine is going to be.
PDC*Line Pharma SA raised €13.9 million (US$15.4 million) in a series B round plus another €6.1 million in loans and grants from the Walloon region of Belgium to take its allogeneic cell-based cancer immunotherapy, PDC*lung-01, into a phase I/II trial in patients with metastatic lung cancer.
DUBLIN – Shares in Targovax ASA rose as much as 26% last Wednesday on news of an option and license agreement in China involving its peptide-based KRAS-directed cancer vaccines, TG-01 and TG-02.
Adaptimmune plc continued its bright start to the year by entering an alliance worth up to $897.5 million with Astellas Pharma Inc. to co-develop and co-commercialize stem cell-based allogeneic CAR T and T-cell receptor (TCR) cell therapies.
Ten days after its JAK1 inhibitor, itacitinib, failed a phase III trial in acute graft-vs.-host-disease (GVHD), Incyte Corp. has finalized a deal that could provide an alternative growth path. It is paying Morphosys AG $750 million up front, investing $150 million in its stock and is on the hook for up to $1.1 billion in milestones for a 50% interest in U.S. rights to the CD19-targeting antibody tafasitamab (MOR-208) and for 100% of the rights in all other territories.
