BioMarin Pharmaceuticals: BMRN Acquires Alesta to Strengthen Its Rare Disease Back"bone"

Research evidence excerpt

J P M O R G A N

North America Equity Research

18 August 2026

BioMarin Pharmaceuticals

BMRN Acquires Alesta to Strengthen Its Rare Disease

Back“bone”

Overweight

BMRN, BMRN US

Price (17 Aug 26):$66.64

Biotechnology - Large Cap

We see BMRN’s announced plan to acquire Alesta Therapeutics for $275mm

upfront as a clear strategic fit for BMRN’s rare disease/skeletal conditions

portfolio, adding ALE1, a potentially first-in-class product that entered the clinic

last year. This deal is very consistent with BMRN’s commentary that biz dev post

Amicus acquisition would focus on the pipeline. Since ALE1 remains an earlierstage asset currently in a phase I/IIa study in healthy volunteers and adults with

hypophosphatasia and no prior ERT use, we see the product representing a

potential source of topline growth for the 2030s. Stepping back, we continue to

think that the stock will work higher as the Street gains a better appreciation of

BMRN’s post-Amicus profile. Even factoring in Voxzogo competition, we see

BMRN putting up a double-digit 4-year adj EPS CAGR through 2030 and see

valuation as compelling with the stock trading at ~13.5x our 2026 adj EPS and

~10x our 2027 estimate.

On ALE1… ALE1 is a clinical-stage oral small molecule for

hypophosphatasia (or HPP), a rare genetic bone disease caused by ALPL

mutations leading to impaired bone/tooth mineralization. Still in early in

clinical development, we think that ALE1 has potential to become the first oral

therapy for HPP offering an alternative to injectable enzyme replacement

(Strensiq) which is approved for perinatal/infantile- and juvenile-onset

hypophosphatasia, while also potentially addressing both skeletal and broader

manifestations of disease through systemic correction of inorganic

pyrophosphate (PPi ) levels. With>9k pts already diagnosed in the US, BMRN

noted that HPP could potentially represent their largest addressable pt

population, despite likely underdiagnosis.

Recapping deal specifics… BMRN will fund this $275mm upfront with cash

on hand, leaving BMRN with ~$600mm of cash proforma (BMRN reported

cash and cash equivalents of $874mm as of June 30). The terms also call for

…