While the drug may be well-positioned for registrational development, some important caveats remain after mixed data in Phase II study cohorts.

Key cancer asset is still looking for a Phase III validation after failing to outperform Keytruda in challenging frontline NSCLC setting.

The introductions of Spinraza and Zolgensma in SMA offer new insights into how to address neurodegenerative diseases. But more real world evidence is needed.

Public Company Edition: Of five drug developers expected to go public, four have preclinical or early clinical candidates, while the one that could not find a market for its IPO is in Phase III. Also, Biohaven, Zymeworks and Kura raise $100m or more in follow-on offerings.