The World Health Organization held off declaring the novel coronavirus infection that has emerged in Wuhan, China, an international public health emergency, despite the fact that the number of confirmed cases tripled and the infection spread to several other countries, including the U.S. and Canada. In response to the emergence of a new coronavirus seen recently in China, and now America, Novavax Inc. has initiated development of a vaccine candidate. The Gaithersburg, Md.-based company has extensive history working with coronaviruses and developing vaccine candidates. Investors recognized the opportunity pushing company shares (NASDAQ:NVAX) up by more than 71% Jan. 21, and they ended the day at $9.82. At market close Friday, Jan. 24, their value had slipped back significantly to $7.80.
Moderna Inc., meanwhile, is collaborating with the Coalition for Epidemic Preparedness Innovations to develop an mRNA vaccine against the 2019-nCoV coronavirus.
Earlier than expected, Horizon Therapeutics plc received FDA approval for teprotumumab in thyroid eye disease, a progressive autoimmune condition that disproportionately affects women. The drug will be marketed as Tepezza and could generate more than $1 billion in peak annual sales.
Epizyme Inc. won accelerated FDA clearance for Tazverik (tazemetostat) in epithelioid sarcoma patients not eligible for curative surgery.
Deals of the week
Aum Biosciences Pte. Ltd. and Cyclica Inc. have agreed to work together to apply Cyclica’s drug discovery platform in Aum’s R&D programs for the discovery of potential new cancer therapies. Aum will deploy its drug R&D skills, including a biomarker-driven approach, while Cyclica will contribute its AI-augmented platforms, Ligand Design for multi-objective drug design, and Ligand Express for off-target profiling, systems biology linkages, and structural pharmacogenomic insights. The two platforms together design molecules that reduce off-target effects while also detailing each molecule's activity through the application of integrated systems biology and structural pharmacogenomics.
Horizon Discovery Group plc will license its gene-edited glutamine synthetase knockout Chinese hamster ovary K1 cell line to NGM Biopharmaceuticals Inc., which is focused on developing therapeutics based on scientific understanding of biological pathways underlying cardiometabolic, liver, oncologic and ophthalmic diseases.
Ncardia AG and Bluerock Therapeutics Inc. inked a deal covering process development technologies for the manufacture of induced pluripotent stem cell-derived cardiomyocytes.
After a slow start to the IPO market, it picked up during the period, with one biopharma getting out of the gate and three others adding themselves to the IPO runway. I-Mab Biopharma Co. Ltd. became the first IPO out of the gate this year, pricing its IPO of about 7.4 million American depositary shares – each 10 representing 23 ordinary shares of the company at $14 each, within the planned range of $12 at the low end and $15 at the high.
Arcutis Biotherapeutics Inc. set terms for its proposed IPO, offering 7.8 million shares priced at a range of $15 to $17, which would raise about $125 million at the midpoint. Black Diamond Therapeutics Inc. set terms for its proposed IPO, offering 8.9 million shares at a price range of $16 to $18, which would raise about $151 million at the midpoint. Revolution Medicines Inc. has filed to raise up to $100 million from an IPO.
Public offerings: Adaptimmune Therapeutics plc priced 21 million American depositary shares at $4 apiece for gross proceeds of about $84 million; Nanoviricides Inc. raised gross proceeds of $7.5 million from its offering.
Private transactions: 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; Ionctura SA has closed a €15 million (US$16.6 million) series A financing round.
Whether large or small, or based in the U.S. or abroad, life sciences companies are an increasingly attractive target for shareholder suits. The number of securities fraud class action suits filed in the U.S. in 2019 across all industries totaled 404, just one more than the 403 cases filed in 2018 and down from the peak of 412 in 2017, according to a new Dechert LLP report. However, nearly 25% of the suits filed in 2019 involved life sciences companies, compared with about 20% in the previous two years – which set records for the number of shareholder suits filed against companies in the health care industry, including drug and device manufacturers.
While machine learning holds great promise in accelerating drug development, several challenges hinder its adoption, according to a new U.S. Government Accountability Office (GAO) report. To overcome them and more fully unleash the promise of artificial intelligence in developing new drugs, the GAO developed several policy options centering around research, data access, standardization, human capital and regulatory certainty. These include government policymakers promoting basic research to generate more and better data and improve understanding of machine learning in drug development; creating mechanisms or incentives for increased sharing of high quality data while ensuring protection of patient information; collaborating with stakeholders to establish uniform standards for data and algorithms; creating opportunities for more public and private sector workers to develop machine learning skills; and collaborating with stakeholders to develop a clear, consistent message on regulating machine learning in drug development. The GAO said policymakers could just maintain the status quo, allowing companies to drive the use of machine learning in drug development. This alternative could mean that problems identified in the report would remain unresolved or be exacerbated, the agency said.
The FDA’s Center for Drug Evaluation and Research is preparing to launch its Drug Risk Management Board to ensure the seamless alignment for marketed product safety activities among offices throughout the center. The new board also is expected to facilitate rapid analysis and resolution of drug safety issues, according to CDER Director Janet Woodcock.
Word on the Street: BioWorld's favorite quotes of the week
"Today’s approval marks an important milestone for the treatment of thyroid eye disease. Currently, there are very limited treatment options for this potentially debilitating disease. This treatment has the potential to alter the course of the disease, potentially sparing patients from needing multiple invasive surgeries by providing an alternative, nonsurgical treatment option."
Wiley Chambers, deputy director of the FDA's Division of Transplant and Ophthalmology Products, in an announcement on the FDA approval for Horizon Therapeutics’ teprotumumab in thyroid eye disease
“When I invest in biotech, I have a sort of a model for the type of person I'm looking to invest in. You basically have people who are extremely conventional and will do experiments that will succeed but will not mean anything. Then you have your various people who are crazy and want to do things that are [going to] make a very big difference. You want to ... find the people who are roughly halfway in between. My challenge to biotech venture capitalists is to find some of those people who are crazy enough to try something bold, but not so crazy that it's going to be this mutation where they do 100 things differently.”
Peter Thiel, who invests across technology and the life sciences via various vehicles, including the Founders Fund, in his keynote address at the Precision Medicine World Conference
“Gold was used therapeutically for a number of decades in rheumatoid arthritis, injected into joints.”
Rob Etherington, CEO-president, Clene Nanomedicine Inc., which just completed enrollment and dosed the first patient in its phase II trial of CNM-Au8 in amyotrophic lateral sclerosis, that uses clean-surfaced faceted nanocrystalline gold suspended in sodium bicarbonate buffered, pharmaceutical-grade water