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BioWorld - Thursday, February 5, 2026
Home » Newsletters » BioWorld

BioWorld

May 16, 2018

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Regulatory front

Delivering on President Donald Trump's blueprint to lower prescription drug prices, the Centers for Medicare & Medicaid Services (CMS) released a redesigned version of its Drug Spending Dashboards Tuesday. The interactive online dashboards now include year-over-year information on drug pricing and highlight which manufacturers have increased prices. Read More

Financings

Meiragtx Holdings plc, of New York, has filed to raise up to $86 million in an IPO. Once the offering has priced the company expects that its shares will trade on the Nasdaq under the symbol MGTX. Using gene therapies, it will develop a product pipeline focused on inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Read More

Other news to note

Iliad Biotechnologies LLC, of Weston, Fla., and Vanderbilt University Medical Center report that an investigational new drug (IND) application has been filed by the National Institute for Allergy and Infectious Diseases (NIAID) at the National Institutes of Health (NIH) to study the next generation pertussis vaccine BPZE1.  Read More

Clinical data for May 15, 2018

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With $11M, Syntekabio will advance bioinformatics and AI platforms

HONG KONG – South Korean bioinformatics company Syntekabio Inc. pocketed $11.2 million in a series B financing to advance two of it core platforms in bioinformatics and AI-driven drug discovery to speed up drug development process. Read More

Exploit the luck of approved drugs with more systematic repurposing

TEL AVIV – Industrializing "luck" may open the door to a motherlode that the biopharmaceutical industry may be currently ignoring. One path to faster drug development, said Daphna Leifenfeld, is repurposing of existing drugs. This not only makes sense, but is something that the pharmaceutical industry should do more of, she said at the 17th Mixiii-Biomed Conference this week. The meeting in Israel is focused on trends shaping the future of health care systems and life sciences. Read More

Xenikos raises $30M for acute GVHD candidate

LONDON – Xenikos BV has taken a huge step toward commercializing its T-Guard treatment for acute graft-vs.-host disease (GVHD), closing a $30 million series B round to fund U.S. and European phase III trials of the antibody-drug conjugate. Read More

CBER head Peter Marks weighs in on cell, gene therapy pricing debate

DUBLIN – Last week Philadelphia-based Spark Therapeutics Inc. reported $2.4 million revenue for the debut quarter of Luxturna (voretigene neparvovec-rzyl), its gene therapy for an inherited form of vision loss, which gained FDA approval last year. Novartis AG also recently reported its first quarterly sales figure for Kymriah (tisagenlecleucel). The chimeric antigen receptor T-cell (CAR-T) therapy took in $12 million in the first quarter, its second on the market since its approval last August for children and young adults with B-cell precursor acute lymphoblastic leukemia (ALL). Gilead Sciences Inc. reported Q1 sales of $40 million for Yescarta (axicabtagene ciloleucel), the CD19-directed CAR-T therapy approved for diffuse large B-cell lymphoma, following sales of $7 million in Q4 2017, its first quarter on the market. Although modest, the numbers demonstrate that the new era of cell and gene therapy has – after several false dawns in Europe – finally arrived. Read More

Daiichi hits repeat, inks $484.7M Zymeworks deal for bispecific technology

As Zymeworks Inc. prepares to unveil new phase I data from its lead bispecific antibody candidate at the upcoming American Society of Clinical Oncology meeting, its platform technology scored another win in the validation column. Less than two years after licensing rights to Zymeworks' Azymetric and EFECT technology platforms to develop one bispecific candidate, Daiichi Sankyo Co. Ltd. returned to the negotiating table for more. Read More

Celsius collects $65M series A to fund precision medicine effort

Celsius Therapeutics became the latest biopharma spin-out from Third Rock Ventures, which also led the Cambridge, Mass.-based company's $65 million series A financing. GV (formerly Google Ventures), Heritage Provider Network, Casdin Capital, Alexandria Venture Investments and other undisclosed investors participated in the round for Celsius, which is seeking to develop precision medicines in autoimmune diseases and cancer by drilling genomic insights to the single cell level to understand which cells drive disease and identify the genes that trigger their malfunction. Read More

U.S. lawmakers seek HELP for nebulous 340B Drug Discount program

The finger pointing over U.S. drug prices continued Tuesday as members of the Senate Health, Education, Labor and Pensions (HELP) Committee once again tried to grope their way through the nebulous fog of the 340B Discount Drug program. Read More

Regulatory actions for May 15, 2018

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