Wedbush Morning Call - May 19 2026 6:58AM

Research evidence excerpt

Wedbush Morning Call - May 19 2026 6:58AM

step to keep GOSS advancing as cash runway remains 1Q27. While FRI data appear interesting, the pre-NDA meeting outcomes

represents the most important near-term catalyst as this dictates whether a filing can occur by ~September. We are updating

our GOSS estimates to incorporate the proposed debt restructuring and continue to assume earliest potential seralutinib

launch in FY28. Net net, our target remains at $1, and we are sticking with the sidelines until we gain clarity on seralutinib's

path forward.

BioMarin Pharmaceuticals (BMRN)

Rating: OUTPERFORM, 12-Month Price Target: $94.00, Price: $51.81

BMN 401 Disappoints In ENPP1 Deficiency; No Impact On Valuation

BMRN announced that the Ph3 ENERGY 3 study of BMN 401 in children aged 1-12 with ENPP1 deficiency met the co-primary

endpoint of plasma PPi increase through week 52 but missed the other co-primary endpoint of improvement in Radiographic

Global Impression of Change (RGI-C). Additionally, no positive trends were observed on key secondary endpoints. BMRN is

evaluating the full dataset to determine the next steps, and management expects to provide updates when they are available.

Recall BMRN acquired BMN 401 through the acquisition of Inozyme that was completed in Jul. '25, and management had

guided for the peak market opportunity to be 400-600M by mid-2030s, predominantly in adult patients and more from ex-U.S.

We have not included BMN 401 in our valuation and therefore, this morning's negative outcomes do not change our rating

or PT on BMRN shares.

The open-label Ph3 pivotal ENERGY-3 study enrolled 27 pediatric patients with ectonucleotide pyrophosphatase/

phosphodiesterase 1 (ENPP1) deficiency, who were 2:1 randomized to receive BMN 401 or continue to take conventional