Pediatric gene editing specialist Logicbio Therapeutics Inc. revealed an FDA clinical hold on a planned phase I/II trial of its lead candidate, LB-001, an investigational therapy for a rare inherited metabolic disorder methylmalonic acidemia. The agency wants "resolution of certain clinical and nonclinical questions," the company said, without providing additional detail about the query. Logicbio shares (NASDAQ:LOGC), which debuted at $10 each in October 2018, fell about 31% by midday to $7.23 in trading volume four times the norm.

Pharmas break up to accelerate growth

Even as the volume of mergers and acquisitions in 2019 reached the highest level in the last 10 years, multiple big pharma companies are looking to get smaller, spinning off units into separate entities. Last week, Merck & Co. Inc. announced plans to divest its women’s health, legacy brands and biosimilars businesses into a new company. That same day, Glaxosmithkline plc made definitive plans to spin off its consumer health care joint venture with Pfizer Inc. In addition to letting go of its consumer health care business, Pfizer is also splitting off its generic drug Upjohn unit through a merger with Mylan NV.

Fate of California law could dictate future of Rx patent settlements

The Association for Accessible Medicines is garnering support in its Ninth Circuit challenge to a new California state law that seemingly thumbs its nose at a 2013 U.S. Supreme Court ruling on so-called pay-for-delay patent settlements. Going where the Supreme Court refused to go, California’s AB 824, which went into effect Jan. 1, 2020, presumes any patent settlement that offers anything of value to a would-be competitor, including a license with a period of exclusivity, is anticompetitive and, thus, illegal. With its stiff penalties, the first-of-its-kind state law undermines federal law and could delay generic and biosimilar competition if allowed to stand, according to an amicus brief filed last week by the Washington Legal Foundation, National Association of Manufacturers and the U.S. Chamber of Commerce.

Volastra launches with a $12M financing

Newly launched Volastra Therapeutics Inc. brought in a $12 million financing to develop treatments for metastatic cancers by targeting chromosomal instability, which is related to cancer metastasis and the vast majority of cancer deaths. The goal is to use Volastra’s library of organoids to determine what drives chromosomal dysfunction and to untangle the genomic pathway chaos that ultimately leads to primary tumors. Much of the science comes from Weill Cornell Medical College and Memorial Sloan Kettering Cancer Center. Polaris Partners led the financing, with help from Droia Oncology Ventures, the Global Health Sciences Fund (Quark Venture LP and GF Securities) and Arch Venture Partners.

Trial recruitment, retention improved but drop-outs persist in late studies

Multiple tactics employed by the biopharma industry to improve the recruitment and retention of participants in clinical trials seem to be paying off. More than three of four (77%) studies now fully enroll on or ahead of schedule, according to researchers at the Tufts Center for the Study of Drug Development (CSDD), reporting in the January/February Tufts CSDD Impact Report on global recruitment performance benchmarks. Compared to early stage trials, late-stage studies face continued headwinds, however, especially in participant retention, with an average drop-out rate of 19.1% across the studied indications, up from 15.3% in a 2012 Tufts CSDD study.

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