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WuXi Biologics and Amicus Therapeutics announced that the companies have entered into an exclusive commercial manufacturing partnership for Amicus' Pompe biologic ATB200. Per the strategic agreement, WuXi Biologics will be the exclusive commercial drug substance manufacturing partner and key commercial drug product supplier. WuXi Biologics will enable and support Amicus with its "Global Dual Sourcing within WuXi Biologics" strategy and manufacture both the drug substance and drug product, each at two sites across its global commercial supply network in EU, China and US. This approach effectively ensures a global and robust supply chain while at the same time minimizes the intra-company technology transfer risk associated with traditional approaches of using different suppliers. The contract has an initial five-year term and then automatically renewed every two years until cancelled. This agreement showcases the strong commercial manufacturing expertise and global premier quality system of WuXi Biologics as well as its powerful "Follow-the-Molecule" strategy that enables a global client base from discovery to commercial manufacture. The ATB200 program was initiated at WuXi Biologics in 2012 and now progresses through a pivotal study enabled by the global leading technology platform and unparalleled manufacturing capacity at WuXi Biologics. "This long-term supply agreement is another significant step forward in our relationship with WuXi Biologics," said Mr. Bradley L. Campbell, President and Chief Operating Officer of Amicus Therapeutics, Inc. "Over the past five years we have successfully collaborated in the scale up of our Pompe biologic from research to commercial scale while maintaining all key quality attributes. We look forward to continuing this important manufacturing partnership as we advance our Pompe program in a pivotal study to support global regulatory approvals, and we are confident in our ability to supply patients around the world."

Bluebird bio (BLUE) announced Chip Baird will join the company effective February 11 and assume the role of CFO. Baird joins bluebird from Amicus Therapeutics (FOLD) where he was the CFO for the past seven years and brings a demonstrated track record of successfully leading companies in all phases of discovery, development and commercialization. Jeff Walsh, who has held various roles over the last eight years at bluebird bio - including COO and, most recently, chief financial and strategy officer - will assume the new dedicated role of chief strategy officer to drive corporate development and the company's overall growth strategy. In addition, Jason Cole, who has served as general counsel since 2014 and chief legal officer since 2016, will assume the new and expanded role of chief operating and legal officer overseeing both operations and legal functions for the company.

Amicus announced additional "positive" results from a global Phase 1/2 clinical study to investigate AT-GAA in patients with Pompe disease, an inherited lysosomal storage disorder caused by an enzyme deficiency that leads to accumulation of glycogen in cells. Patients treated with AT-GAA for up to 24 months showed improvements in six-minute walk test, or 6MWT, distance and other measures of motor function and muscle strength, stability or increases in forced vital capacity, or FVC, and durable reductions in biomarkers of muscle damage and disease substrate. These clinical results, in addition to detailed results on patient-reported outcomes, or PROs, are being featured at the WorldSymposium in a poster at the poster session. An additional poster with detailed results for the non-ambulatory cohort in the ATB200-02 study is also part of this afternoon's poster session. Six-minute walk test distance, a primary measure of motor function in Pompe disease patients, improved in both ERT-naive and ERT-switch patients with continued benefit observed out to months 21 and 24, respectively. Improvements were generally consistent across both cohorts. Non-ambulatory ERT-switch patients showed improvements in upper extremity strength from baseline to month 21, as measured by quantitative muscle testing and manual muscle testing. Pulmonary function improved in ERT-naive patients and was generally stable in ERT-switch patients. An interim analysis of patient-reported outcomes from the ATB200-02 clinical study show the benefits in activities of daily living and patient well-being in patients with Pompe disease treated with AT-GAA. Safety and tolerability data reflect a maximum of 33+ months of treatment. To date, adverse events have been generally mild and transient. AT-GAA has resulted in a low rate of infusion-associated reactions following 1,110+ infusions. The clinical pharmacokinetic profile has been consistent with previously reported preclinical data. Persistent and durable reductions in key biomarkers of muscle damage and disease substrate across all patient cohorts out to month 21/24 continue to suggest a positive effect on muscle tissue.