BMN 401 Disappoints In ENPP1 Deficiency; No Impact On Valuation

Research evidence excerpt

BMN 401 Disappoints In ENPP1 Deficiency; No Impact On Valuation

Quick Note

May 18, 2026 BioMarin Pharmaceuticals (BMRN)

Rating: BMN 401 Disappoints In ENPP1 Deficiency; No Impact OnOUTPERFORM ValuationPrice:

$51.81

12-Month Price Target: BMRN announced that the Ph3 ENERGY 3 study of BMN 401 in children aged

$94.00 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

Analysts were observed on key secondary endpoints. BMRN is evaluating the full dataset to

Yun Zhong, Ph.D. determine the next steps, and management expects to provide updates when they(917) 608-0166

yun.zhong@wedbush.com 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

therapy as clinically indicated by the patients' treatment physician (oral phosphate

supplements and calcitriol or other active forms of vitamin D3 or analogs) for

52 weeks (NCT06046820). In addition to the co-primary endpoints, secondary

endpoints include change from baseline in rickets as measured by Rickets Severity

Score (RSS) and change from baseline in growth Z-score (height/body length and

weight).