NEW
YORK, Sept. 4, 2024 /PRNewswire/ -- BioMarin
Pharmaceutical Inc. (NASDAQ: BMRN) hosted an Investor Day earlier
today, where President and Chief Executive Officer Alexander Hardy and other members of BioMarin's
leadership team provided an overview of the company's new corporate
strategy to deliver sustained value creation and introduced
longer-term Total Revenue and Non-GAAP Operating Margin
guidance. A copy of the presentation and a replay of the
webcast are available at investors.biomarin.com.
![BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.) BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)](https://mma.prnewswire.com/media/534018/BioMarin_Pharmaceutical_Inc_Logo.jpg)
"Over the last 9 months, we have undertaken the transformation
of BioMarin's operations and ways of working with the goal of
accelerating and delivering substantial value to all our
stakeholders – patients, employees and shareholders," said Mr.
Hardy. "Our new corporate strategy harnesses BioMarin's unique
capabilities creating and leading new therapeutic markets around
the world to deliver innovative medicines to the patients we serve.
We are confident in our ability to realize the ambitious plans
outlined today and excited to deliver on our new vision for
BioMarin's future."
Corporate Strategy
The company reviewed BioMarin's differentiated strategy
including its new company structure and updated organizational
model, now built around three business units, Enzyme Therapies,
Skeletal Conditions and ROCTAVIAN®, designed to support
the focus on sustainable growth. The updated corporate
strategy also includes the implementation of a $500 million cost transformation program that
will contribute to Non-GAAP Operating margin targets set for 2026
and beyond.
Value Commitment
BioMarin provided an overview of its long-term financial
outlook, based on its strategic plan to drive revenue growth and
expand Non-GAAP Operating Margin. The summary table below includes
the company's full year 2024 guidance, reaffirmed today, and the
newly introduced outlook for 2026 with respect to Non-GAAP
Operating Margin and other metrics for 2027, shared at BioMarin's
Investor Day.
Item
|
FY 2024
(reaffirmed)
|
FY
2027
|
Total
Revenues
|
$2.750B to
$2.825B
|
$4B
|
Non-GAAP Operating
Margin (1)
|
26% to 27%
|
Low-to-mid 40%s
starting with 40% in 2026
|
Non-GAAP Diluted EPS
(1)(2)
|
$3.10 to
$3.25
|
N/A
|
Operating Cash
flow
|
N/A
|
$1.25B+
|
(1)
|
Refer to Non-GAAP
Information beginning on page 4 of this press release for the
definitions of Non-GAAP Operating Margin and Non-GAAP Diluted EPS.
Reconciliation of forward-looking Non-GAAP Operating Margin and
Non-GAAP Diluted EPS to the most directly comparable U.S. GAAP
reported financial measures is not available. Refer to
Forward-Looking Non-GAAP Financial Measures beginning on page 5 of
this press release for further information regarding
forward-looking Non-GAAP financial measures.
|
(2)
|
Non-GAAP Diluted EPS
guidance assumes approximately 200 million Non-GAAP
weighted-average diluted shares outstanding.
|
|
|
Innovation
The company highlighted its
innovation strategy that supports BioMarin's pipeline of high
impact medicines, including 11 anticipated product launches by 2034
and two by 2027. Building on its 25-year history developing
medicines that target serious genetic conditions, several programs
that are currently advancing through the pipeline, include:
- Phase 3 enrollment of VOXZOGO® for the treatment of
hypochondroplasia, with expected data readout targeted in 2026, and
potential approval in 2027
- Clinical updates on four additional skeletal conditions,
including idiopathic short stature, Noonan Syndrome, Turner
Syndrome and SHOX Deficiency, all progressing through or beginning
Phase 2 studies
- PALYNZIQ® age label expansion to include
12-17-year-olds in the United
States and 12-15-year-olds outside of the United States, with targeted filings in
late 2025 and early 2026, respectively
- BMN 390 for the treatment of phenylketonuria (PKU) with a
targeted profile that reduces immunogenicity from novel pegylation,
and targeted Investigational New Drug (IND) application planned for
late 2025
- BMN 351 for the treatment of Duchenne Muscular Dystrophy that
targets a unique novel site for exon skipping that may enable
dystrophin expression up to 40%, based on observations
preclinically. Target Proof of Concept (POC) is expected in
2025
- BMN 349 for the treatment of Alpha-1 Antitrypsin Deficiency
with a unique mechanism of action that has the potential to
transform liver health and with target POC in 2026
- BMN 370 for the treatment of von Willebrand Disease (vWD) has
the potential to normalize bleeding events, based on results from a
vWD preclinical model, and is designed to be delivered with a
single subcutaneous injection. The targeted IND is planned for late
2025.
Growth:
The company highlighted its strategy for optimizing its growing
and durable Enzyme Therapies business unit, as well as its plan for
sustainable leadership across multiple Skeletal Conditions,
building on the strength of VOXZOGO for the treatment of
achondroplasia. The combined business units are expected to
drive a targeted mid-teen Compounded Annual Growth Rate through
2034.
A replay of today's event and accompanying presentation slides
can be found at investors.biomarin.com.
About BioMarin
Founded in 1997, BioMarin is a global
biotechnology Company dedicated to transforming lives through
genetic discovery. The Company develops and commercializes targeted
therapies that address the root cause of genetic conditions.
BioMarin's unparalleled research and development capabilities have
resulted in eight transformational commercial therapies for
patients with rare genetic disorders. The Company's distinctive
approach to drug discovery has produced a diverse pipeline of
commercial, clinical, and pre-clinical candidates that address a
significant unmet medical need, have well-understood biology, and
provide an opportunity to be first-to-market or offer a substantial
benefit over existing treatment options. For additional
information, please visit www.biomarin.com.
Forward-Looking Statements
This press release and the
associated conference call and webcast contain forward-looking
statements about the business prospects of BioMarin Pharmaceutical
Inc. (BioMarin), including, without limitation, statements about:
future financial performance, including the expectations of Total
Revenues, Non-GAAP Operating Margin percentage, Non-GAAP Diluted
EPS, Operating Cash Flow and Revenue Compound Annual Growth Rate
(CAGR) for, in certain instances, the full-year 2024 and future
periods and the underlying assumptions; BioMarin's new corporate
strategy, including plans and expectations regarding innovation and
growth, the $500 million cost
optimization program, capital allocation, and organizational
redesign efforts, as well as the anticipated benefits of such
strategy, including BioMarin's ability to achieve top quartile
biopharma revenue growth, double its Non-GAAP Operating Margin,
achieve innovation and efficiencies, optimize cash flow and capital
allocation, and deliver significant and sustained value creation to
stakeholders; BioMarin's future strategy for ROCTAVIAN and its
anticipated benefits, including BioMarin's expectations regarding
reduction of annual direct ROCTAVIAN expenses beginning in 2025 and
ROCTAVIAN being profitable by the end of 2025; ability of
BioMarin's approved products, including VOXZOGO and BioMarin's
enzyme therapies, to drive long-term revenue growth; the clinical
development and commercialization of BioMarin's product candidates
and commercial products, including plans and expectations regarding
(i) the ability to expand BioMarin's leadership in achondroplasia
with VOXZOGO and leverage VOXZOGO in other skeletal conditions,
including hypochondroplasia, idiopathic short stature, Noonan
Syndrome, Turner Syndrome and SHOX deficiency; (ii) development of
BMN 333 for the treatment of achondroplasia and hypochondroplasia,
(iii) expansion of PALYNZIQ for the treatment of adolescents with
phenylketonuria (PKU), (iv) development of BMN 390 for the
treatment of PKU, (v) development of BMN 351 for the treatment of
Duchenne Muscular Dystrophy, (vi) development of BMN 349 for the
treatment of alpha-1 antitrypsin deficiency, and (vii) development
of BMN 370 for the treatment of von Willebrand disease; the
expected benefits and availability of BioMarin's product candidates
and commercial products; the timing of BioMarin's clinical
development and commercial prospects, including announcements of
data from clinical studies and trials; and potential growth
opportunities and trends, including the assumptions and
expectations regarding Total Addressable Patient Population with
respect to the conditions targeted by BioMarin's product candidates
and commercial products.
These forward-looking statements are predictions and involve
risks and uncertainties such that actual results may differ
materially from these statements. These risks and uncertainties
include, among others, those factors detailed in BioMarin's filings
with the Securities and Exchange Commission, including, without
limitation, the factors contained under the caption "Risk Factors"
in BioMarin's Quarterly Report on Form 10-Q for the quarter ended
June 30, 2024, as such factors may be
updated by any subsequent reports. You should carefully consider
that information before you make an investment decision. You should
not place undue reliance on forward-looking statements, which speak
only as of the date hereof. These forward-looking statements are
based on the beliefs and assumptions of the Company's management
based on information currently available to management and should
be considered in connection with any written or oral
forward-looking statements that the Company may issue in the future
as well as other cautionary statements the Company has made and may
make. Except as required by law, BioMarin does not undertake any
obligation to update or alter any forward-looking statement,
whether as a result of new information, future events or
otherwise.
BioMarin®, BRINEURA®, KUVAN®,
NAGLAZYME®, PALYNZIQ®, ROCTAVIAN®,
VIMIZIM® and VOXZOGO® are registered
trademarks of BioMarin Pharmaceutical Inc., or its affiliates.
ALDURAZYME® is a registered trademark of
BioMarin/Genzyme LLC. All other brand names and service marks,
trademarks and other trade names appearing in this release are the
property of their respective owners.
Non-GAAP Information
This press release includes both GAAP information and Non-GAAP
information. Non-GAAP Income is defined by the company as GAAP Net
Income excluding amortization of intangible assets, stock-based
compensation expense and, in certain periods, certain other
specified items, as detailed below when applicable. The company
also includes a Non-GAAP adjustment for the estimated tax impact of
the reconciling items. Non-GAAP Operating Margin percentage is
defined by the company as GAAP Income from Operations, excluding
amortization of intangible assets, stock-based compensation
expense, and, in certain periods, certain other specified items,
divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by
the company as Non-GAAP Income divided by Non-GAAP weighted-average
diluted shares outstanding. Non-GAAP weighted-average diluted
shares outstanding is defined by the company as GAAP
weighted-average diluted shares outstanding, adjusted to include
any common shares issuable under the company's equity plans and
convertible debt in periods when they are dilutive under
Non-GAAP.
BioMarin regularly uses both GAAP and Non-GAAP results and
expectations internally to assess its financial operating
performance and evaluate key business decisions related to its
principal business activities: the discovery, development,
manufacture, marketing and sale of innovative biologic therapies.
Because Non-GAAP Income, Non-GAAP Operating Margin percentage,
Non-GAAP Diluted EPS and Non-GAAP weighted-average diluted shares
outstanding are important internal measurements for BioMarin, the
company believes that providing this information in conjunction
with BioMarin's GAAP information for historical results enhances
investors' and analysts' ability to meaningfully compare the
company's results from period to period and to its forward-looking
guidance, and to identify operating trends in the company's
principal business. BioMarin also uses Non-GAAP Income internally
to understand, manage and evaluate its business and to make
operating decisions, and compensation of executives is based in
part on this measure.
Non-GAAP measures are not meant to be considered in isolation or
as a substitute for, or superior to, comparable GAAP measures and
should be read in conjunction with the consolidated financial
information prepared in accordance with GAAP. Investors should note
that the Non-GAAP information is not prepared under any
comprehensive set of accounting rules or principles and does not
reflect all of the amounts associated with the company's results of
operations as determined in accordance with GAAP. Investors should
also note that these Non-GAAP financial measures have no
standardized meaning prescribed by GAAP and, therefore, have limits
in their usefulness to investors. In addition, from time to time in
the future there may be other items that the company may exclude
for purposes of its Non-GAAP financial measures; likewise, the
company may in the future cease to exclude items that it has
historically excluded for purposes of its Non-GAAP financial
measures. Because of the non-standardized definitions, the Non-GAAP
financial measure as used by BioMarin in this press release may be
calculated differently from, and therefore may not be directly
comparable to, similarly titled measures used by other
companies.
Forward-Looking Non-GAAP Financial Measures
BioMarin does not provide guidance for GAAP reported financial
measures (other than revenue) or a reconciliation of
forward-looking Non-GAAP financial measures to the most directly
comparable GAAP reported financial measures because the company is
unable to predict with reasonable certainty the financial impact of
changes resulting from its strategic portfolio and business
operating model reviews; potential future asset impairments; gains
and losses on investments; and other unusual gains and losses
without unreasonable effort. These items are uncertain, depend on
various factors, and could have a material impact on GAAP reported
results for the guidance period. As such, any reconciliations
provided would imply a degree of precision that could be confusing
or misleading to investors.
Contacts:
|
|
Investors
|
Media
|
Traci
McCarty
|
Marni Kottle
|
BioMarin Pharmaceutical
Inc.
|
BioMarin Pharmaceutical
Inc.
|
(415)
455-7558
|
(415)
218-7111
|
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SOURCE BioMarin Pharmaceutical Inc.