BioMarin 制药（BMRN.O）：收购 Alesta 为 HPP 提供口服治疗替代方案

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18 Aug 2026 11:32:12 ET │ 10 pages

Biomarin Pharmaceutical Inc (BMRN.O)

Alesta Acquisition Sources an Oral Treatment Alternative for HPP

CITI'S TAKE

This morning BioMarin announced the acquisition of Alesta Therapeutics

and its development stage asset ALE1 for the treatment of

(HPP; terms below). At an upfront purchase price of

$275M, we view the acquisition as a disciplined use of funds toward

complementary pipeline expansion for a sizeable market opportunity of

high unmet need. Notably, if successfully developed, ALE1 would be a

first-in-class oral treatment for the spectrum of HPP, including the

underpenetrated adult-onset segment. For reference, current standardof-care ERT, generating $1.7B globally, is restricted to pediatric patients

and carries high administrative burden. Given ALE1’s early stage of

development, however, we’re not surprised by the muted BMRN share

reaction. That said, with additional clarity on the scope of a potential ALE1

phase 1 readout in 2027, we see the potential for shares to grow more

accretive on the otherwise attractive commercial opportunity over the

mid-term.

Mechanistic snapshot of ALE1 and phase 1/2a trial underway. ALE1 is an oral, small

molecule designed to reduce inorganic phosphate (PPi) levels in plasma by inhibiting

ectonucleotide pyrophosphatase/

e 1 (ENPP1), a key enzyme in

HPP pathogenesis, to restore the crucial balance of PPi to normalize bone

mineralization. HPP is a rare genetic bone disorder caused by loss-of-function

mutations in the ALPL gene leading to deficient tissue-nonspecific alkaline

phosphatase (TNAP) and subsequent PPi accumulation resulting in skeletal and

dental

on. HPP onset can occur at birth, early childhood, or even in

adulthood. While ~9K US patients are diagnosed today, HPP is still underdiagnosed

given the broad range of symptoms and severity patients present with. A proof-ofconcept phase 1/2a double-blind, placebo-controlled study in 120 healthy

volunteers and adults with HPP assessing safety and PK/PD is underway

(NCT07179640 link, primary completion Jan 2027). In our catchup, BMRN noted that

clinical results from the trial will be presented in 2027 along with supporting

…