Treatment-emergent adverse events (TEAEs) may have soured investors on otherwise-positive new data from Cardiff Oncology Inc.’s phase Ib/II study with PLK1 inhibitor onvansertib in second-line, KRAS-mutated metastatic colorectal cancer (mCRC). Cardiff, however, is forging ahead with a pivotal experiment.

The FDA clapped a clinical hold on the IND for a clinical trial of Dyne Therapeutics Inc.’s DYNE-251 for treating Duchenne muscular dystrophy in patients amenable to skipping exon 51. The agency is asking for more clinical and non-clinical information on the therapy. A response, including data from existing and ongoing studies in the second quarter of 2022, is expected to be filed to the FDA sometime in mid-2022, Dyne said.

PERTH, Australia – Australia has recently established new research and discovery centers to study psychedelic treatments for mental health disorders. Headquartered Melbourne, the Psychae Institute is a global research collaboration supported by a AU$40 million (US$29 million) investment from a North American biotechnology company.

Targeting VEGF and DLL4 at the same time in solid tumors is an approach that continues to entice researchers, with such firms as Abbvie Inc., Compass Therapeutics Inc. and Oncxerna Therapeutics Inc. working to come up with oncology solutions.

“There isn’t a better place to be” now than in Alzheimer’s disease (AD) drug development, said Phyllis Ferrell, global head of external engagement in AD and neurodegeneration at Eli Lilly and Co., during Biotech Showcase’s panel talk titled, “Aduhelm: Stimulating the Next Generation of AD Treatment.”

Having unveiled more data from the ongoing, global phase III Gener8-1 study with Roctavian (valoctocogene roxaparvovec, also known as valrox), Biomarin Pharmaceutical Inc. remains on track to file a regulatory submission with the FDA in the second quarter of this year for the gene therapy to treat adults with severe hemophilia A. The EMA is already reviewing a marketing authorization application.

Annexon Inc. deemed promising – as did analysts – the interim phase II data with C1q protein complex inhibitor ANX-005 in Huntington’s disease (HD), but the safety profile took Wall Street aback, and shares (NASDAQ:ANNX) sank $3.75, or 34%, to close at $7.26.

Avrobio Inc., stung by variable outcomes in a phase II test of its investigational Fabry disease therapy, is quitting further enrollment in the program, one of the first from its gene therapy platform, Plato. The team's attention will shift instead to other clinical-stage lysosomal disorder programs amid "an increasingly challenging market and regulatory environment for Fabry disease," the company said.