- Reported $380.8 million in net product revenue for the full
year 2023, including $108.4 million in the fourth quarter
- Delivered $49.3 million in net income for the full year 2023,
including $4.7 million in the fourth quarter, and ended the year
with cash, cash equivalents and short-term investments of $371.4
million
- Management to host conference call and webcast today at 8:00
a.m. Eastern Time
Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the
“Company”) today reported financial results for the fourth quarter
and full year ended December 31, 2023.
“In 2023, we made significant strides towards transforming the
way ALS is treated. We delivered RELYVRIO, also known as ALBRIOZA
and AMX0035, the first and only ALS therapy that has been shown to
slow disease progression, help maintain functional independence,
and extend overall survival in the same clinical trial, to
thousands of people living with ALS in North America,” said Joshua
Cohen, co-CEO of Amylyx. “As we reflect on the many stories that we
have heard from people living with ALS who are taking RELYVRIO, we
are working with urgency to continue expanding our U.S. and
international presence in 2024 and bringing RELYVRIO to more people
living with ALS.”
“We look forward to sharing topline data from our Phase 3
PHOENIX trial during or before the second quarter of 2024. PHOENIX
will provide additional efficacy and safety data in a larger
population of people living with ALS, building on the robust and
positive results observed in the CENTAUR study,” said Justin Klee,
co-CEO of Amylyx. “We also remain focused on advancing our
pipeline, which includes studying AMX0035 in Progressive
Supranuclear Palsy in the Phase 3 ORION trial. We look forward to
several expected upcoming milestones for our pipeline in 2024,
including data from our Phase 2 HELIOS trial in Wolfram syndrome
and our antisense oligonucleotide, AMX0114, entering the
clinic.”
Full Year 2023 and Recent Business Highlights:
- Commercial launches of RELYVRIO® in the U.S. and ALBRIOZA™
in Canada, also known as AMX0035, continued to progress, and
eligible people living with ALS in multiple countries are also
accessing RELYVRIO through early access pathways. Net
product revenue for the three months ended December 31, 2023 was
$108.4 million, compared to net product revenue of $102.7 million
for the three months ended September 30, 2023. The vast majority of
Amylyx’ net product revenue is generated in the U.S.; revenue in
Canada and from named patient sales programs in international
markets represented an important source of revenue growth in three
months ended December 31, 2023 relative to the Company’s financial
results for the three months ended September 30, 2023.
- Amylyx announced the first patient dosed in the Global Phase
3 ORION Study of AMX0035 in Progressive Supranuclear Palsy
(PSP). ORION is a global, randomized, double-blind,
placebo-controlled Phase 3 clinical trial designed to assess the
efficacy, safety, and tolerability of AMX0035 compared to placebo.
Approximately 600 participants will be enrolled across North
America, Europe, and Japan. The first participant was dosed in
December 2023, and topline results are anticipated in 2025 or
2026.
- Amylyx continues to progress its R&D programs. The
Company announced completion of enrollment in the HELIOS study, a
Phase 2 clinical trial of AMX0035 for the treatment of Wolfram
syndrome and continues to expect data from the study in 2024. The
Company is also working on a novel composite diagnostic test to
assist in diagnosing ALS earlier. Additionally, Amylyx is working
on a new taste-masked formulation of RELYVRIO that may allow for
new intellectual property.
- Amylyx added new members to its executive leadership team
and U.S. commercial leadership team. In November 2023, the
Company announced the appointment of Camille L. Bedrosian, MD, as
Chief Medical Officer. Dr. Bedrosian has nearly 30 years of
experience addressing unmet medical needs for people with rare and
serious diseases through successful clinical and translational
research programs. In addition, during 2024, Linda Arsenault joined
as the Company’s new Chief Human Resources Officer and Dan Monahan
joined as General Manager and Head of U.S. Commercial Markets. Ms.
Arsenault has over 30 years of human resources leadership
experience, including in the biopharmaceutical sector, most
recently from Sunovion Pharmaceuticals where she served as Chief
Human Resources Officer. Mr. Monahan has over 20 years of
experience in sales, marketing, and market access, including his
most recent position as VP of CNS Marketing and Portfolio Strategy
at Otsuka Pharmaceutical Companies.
- A post hoc survival analysis comparing the CENTAUR clinical
trial to historical clinical trial control was published in the
Annals of Clinical and Translational Neurology. The results of
this post hoc analysis demonstrated that the median overall
survival was 10.4 months longer in the CENTAUR AMX0035 group than
in the historical clinical trial control group.
- Post hoc analyses on CENTAUR trial participants were
published in Journal of Neurology, Neurosurgery and Psychiatry.
The results of the post hoc analyses demonstrated a significant
reduction in plasma concentrations of YKL-40 (also known as
chitinase-3-like protein 1) and the systemic inflammatory biomarker
C-reactive protein (CRP), two plasma neuroinflammatory biomarkers
in ALS, over 24 weeks, with reductions observed as early as Week 12
in participants from the CENTAUR trial.
- Data on RELYVRIO and AMX0114, the Company’s investigational
antisense oligonucleotide targeting calpain-2, were
presented at the 34th International Symposium on ALS/MND. An
update on kinetic profiling experiments of AMX0114 was presented as
well as results from a collaboration with Dr. Sami Barmada and his
team at the University of Michigan School of Medicine in which the
impact of AMX0114 on survival was evaluated in human iPSC-derived
motor neurons with an ALS associated mutation in TDP43. The Company
is advancing AMX0114 through investigational new drug (IND)
enabling studies and the goal is to enter the clinic during 2024.
Additional information, including copies of each of the posters
presented at the event, are available in the in the
“Publications and Presentations” section of the Amylyx
website.
Financial Results for the Fourth Quarter and Year Ended
December 31, 2023
Net product revenue: Net product revenue was $108.4
million for the three months ended December 31, 2023, compared to
net product revenue of $21.9 million for the same period in 2022.
Net product revenue for the year ended December 31, 2023 was $380.8
million, compared to net product revenue of $22.2 million for the
same period in 2022. The increase was primarily driven by units of
RELYVRIO sold in the U.S. following regulatory approval in late
September 2022.
Cost of Sales: Cost of sales were $9.4 million in the
three months ended December 31, 2023, compared to cost of sales of
$2.8 million for the same period in 2022. For the year ended
December 31, 2023, cost of sales were $25.4 million, compared to
cost of sales of $3.0 million for the year ended December 31,
2022.
R&D Expenses: Research and development expenses for
the fourth quarter of 2023 were $44.9 million, compared to $22.8
million for the same period in 2022, and $128.2 million for the
year ended December 31, 2023 compared to $93.5 million for the year
ended December 31, 2022. The increase was primarily driven by an
increase in personnel-related expenses due to added headcount to
support research and development efforts, an increase in spending
related to the initiation of the Phase 3 ORION trial of AMX0035 in
PSP, and an increase in preclinical development activities.
SG&A Expenses: Selling, general, and administrative
expenses for the fourth quarter of 2023 were $52.2 million,
compared to $40.8 million for the same period in 2022 and $188.4
million for the year ended December 31, 2023, compared to $127.1
million for the year ended December 31, 2022. The increase was
primarily driven by higher personnel-related expenses due to added
headcount to support the launch, commercialization initiatives, and
operations as a public company.
Net Income: Net income for the three months ended
December 31, 2023 was $4.7 million, or $0.07 on a fully diluted per
share basis, compared to a net loss of $42.7 million, or $0.65 per
share for the same period in 2022. Net income for the year ended
December 31, 2023 was $49.3 million, or $0.70 on a fully diluted
per share basis, compared with a net loss of $198.4 million, or
$3.39 per share for the year ended December 31, 2022.
Cash Position: Cash, cash equivalents, and short-term
investments were $371.4 million at December 31, 2023, compared to
$355.0 million at September 30, 2023.
Investor Conference Call Information
Amylyx’ management team will host a conference call and webcast
today, February 22, 2024, at 8:00 a.m. ET to discuss financial
results and provide an update on the business. To access the
conference call, please dial +1 (800) 836-8184 (U.S. & Canada)
or +1 (646) 357-8785 (international) at least 10 minutes prior to
the start time and ask to be joined into the Amylyx Pharmaceuticals
call. A live audio webcast of the call will be available under
“Events and Presentations” in the Investor section of the Company’s
website, https://investors.amylyx.com/news-events/events. The
webcast will be archived and available for replay for 90 days
following the event.
Available Information
We periodically provide other information for investors on our
corporate website, https://amylyx.com, and our investor relations
website, https://investors.amylyx.com. This includes press releases
and other information about financial performance, information on
corporate governance, and details related to our annual meeting of
stockholders. We intend to use our website as a means of disclosing
material non-public information and for complying with our
disclosure obligations under Regulation FD. Accordingly, investors
should monitor our website, in addition to following the Company's
press releases, SEC filings, and public conference calls and
webcasts.
About RELYVRIO®/ALBRIOZA™/AMX0035
RELYVRIO® (also known as AMX0035), an oral, fixed-dose
combination of sodium phenylbutyrate and taurursodiol (known as
ursodoxicoltaurine outside of the U.S.), is approved to treat
amyotrophic lateral sclerosis (ALS) in adults in the U.S. and
approved with conditions as ALBRIOZA™ for the treatment of ALS in
Canada. AMX0035 is being studied as an investigational drug in
several other regions for the potential treatment of ALS and other
neurodegenerative diseases. The formulation of RELYVRIO, ALBRIOZA,
and AMX0035 are identical.
RELYVRIO® (sodium phenylbutyrate and taurursodiol) Safety
Information for United States
WARNINGS AND PRECAUTIONS
Risk in Patients with Enterohepatic Circulation Disorders,
Pancreatic Disorders, or Intestinal Disorders
RELYVRIO contains taurursodiol, which is a bile acid. In
patients with disorders that interfere with bile acid circulation,
there may be an increased risk for worsening diarrhea, and patients
should be monitored appropriately for this adverse reaction.
Pancreatic insufficiency, intestinal malabsorption, or intestinal
diseases that may alter the concentration of bile acids may also
lead to decreased absorption of either of the components of
RELYVRIO. Because different enterohepatic circulation, pancreatic,
and intestinal disorders have varying degrees of severity, consider
consulting with a specialist. Patients with disorders of
enterohepatic circulation (e.g., biliary infection, active
cholecystitis), severe pancreatic disorders (e.g., pancreatitis),
and intestinal disorders that may alter concentrations of bile
acids (e.g., ileal resection, regional ileitis) were excluded from
the study; therefore, there is no clinical experience in these
conditions.
Use in Patients Sensitive to High Sodium Intake
RELYVRIO has a high salt content. Each initial daily dosage of 1
packet contains 464 mg of sodium; each maintenance dosage of 2
packets daily contains 928 mg of sodium. In patients sensitive to
salt intake (e.g., those with heart failure, hypertension, or renal
impairment), consider the amount of daily sodium intake in each
dose of RELYVRIO and monitor appropriately.
ADVERSE REACTIONS
The most common adverse reactions (at least 15% and at least 5%
greater than placebo) with RELYVRIO were diarrhea, abdominal pain,
nausea, and upper respiratory tract infection.
Gastrointestinal-related adverse reactions occurred throughout the
study but were more frequent during the first 3 weeks of
treatment.
Please click here for RELYVRIO Full U.S. Prescribing
Information.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and
creating more moments for the neurodegenerative community through
the discovery and development of innovative new treatments. Amylyx
is headquartered in Cambridge, Massachusetts and has operations in
Canada, EMEA, and Japan. For more information, visit amylyx.com and
follow us on LinkedIn and X, formerly known as Twitter. For
investors, please visit investors.amylyx.com.
Forward-Looking Statements
Statements contained in this press release and related comments
in our earnings conference call regarding matters that are not
historical facts are “forward-looking statements” within the
meaning of the Private Securities Litigation Reform Act of 1995, as
amended. Because such statements are subject to risks and
uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. Such
statements include, but are not limited to, the potential of
AMX0035 (sodium phenylbutyrate and taurursodiol) as a treatment for
ALS and other neurodegenerative diseases including Wolfram syndrome
and PSP; the Company’s beliefs regarding the benefits of AMX0035 in
ALS and other neurodegenerative diseases, the potential of AMX0035
to be a foundational therapy for ALS and a potential, future cure;
the ongoing commercialization of RELYVRIO and ALBRIOZA; the
potential to expand global approvals for AMX0035 in ALS and other
neurodegenerative diseases; expectations regarding the timing of
the announcement of results from the Phase 3 PHOENIX trial of
AMX0035 for the treatment of ALS, the Company’s Phase 3 ORION trial
of AMX0035 for the treatment of PSP, and the Company’s Phase 2
HELIOS trial of AMX0035 for the treatment of Wolfram syndrome; the
potential continued market acceptance and market opportunity for
RELYVRIO and ALBRIOZA and opportunities for growth; the potential
for new pipeline programs and clinical indications for AMX0035;
statements regarding regulatory developments; the Company’s
expectations with respect to its progress through IND enabling
studies of AMX0114 and other advancements in its pipeline; the
Company’s expectations regarding its financial performance; and
expectations regarding the Company’s longer-term strategy. Any
forward-looking statements in this press release and related
comments in the Company's earnings conference call are based on
management’s current expectations of future events and are subject
to a number of risks and uncertainties that could cause actual
results to differ materially and adversely from those set forth in
or implied by such forward-looking statements. Risks that
contribute to the uncertain nature of the forward-looking
statements include: the success, cost, and timing of Amylyx’
program development activities, Amylyx’ ability to successfully
commercialize RELYVRIO in the United States and ALBRIOZA in Canada,
Amylyx’ ability to execute on its commercial and regulatory
strategy, regulatory developments, expectations regarding the
timing of results of the global Phase 3 PHOENIX trial of AMX0035
for the treatment of ALS, Amylyx’ ability to fund operations, and
the impact that global macroeconomic uncertainty, geopolitical
instability and public health events, such as COVID-19, will have
on Amylyx’ operations, as well as the risks and uncertainties set
forth in Amylyx’ United States Securities and Exchange Commission
(SEC) filings, including Amylyx’ Annual Report on Form 10-K for the
year ended December 31, 2023, and subsequent filings with the SEC.
All forward-looking statements contained in this press release and
related comments in our earnings conference call speak only as of
the date on which they were made. Amylyx undertakes no obligation
to update such statements to reflect events that occur or
circumstances that exist after the date on which they were
made.
AMYLYX PHARMACEUTICALS,
INC.
CONDENSED CONSOLIDATED BALANCE
SHEETS
UNAUDITED
(in thousands)
December 31,
2023
2022
Assets
Cash, cash equivalents and short-term
investments
$
371,362
$
346,945
Accounts receivable, net
40,050
15,306
Inventories
83,280
9,769
Prepaid expenses and other current
assets
14,931
10,113
Other assets
7,831
9,320
Total assets
$
517,454
$
391,453
Liabilities and Stockholders’
Equity
Accounts payable and accrued expenses
$
79,785
$
44,569
Other liabilities
4,237
6,277
Total liabilities
84,022
50,846
Stockholders’ equity
433,432
340,607
Total liabilities, redeemable convertible
preferred stock and stockholders' equity
$
517,454
$
391,453
AMYLYX PHARMACEUTICALS,
INC.
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS
UNAUDITED
(in thousands, except share
and per share data)
Three Months Ended December
31,
Year Ended December
31,
2023
2022
2023
2022
Product revenue, net
$
108,449
$
21,885
$
380,786
$
22,230
Operating expenses:
Cost of sales
9,360
2,821
25,441
2,993
Research and development
44,914
22,813
128,187
93,450
Selling, general and administrative
52,241
40,844
188,356
127,128
Total operating expenses
106,515
66,478
341,984
223,571
Income (loss) from operations
1,934
(44,593
)
38,802
(201,341
)
Other income, net
4,542
2,468
15,495
3,740
Income (loss) before income taxes
6,476
(42,125
)
54,297
(197,601
)
Provision for income taxes
1,745
579
5,026
774
Net income (loss)
$
4,731
$
(42,704
)
$
49,271
$
(198,375
)
Net income (loss) per share
Basic
$
0.07
$
(0.65
)
$
0.73
$
(3.39
)
Diluted
$
0.07
$
(0.65
)
$
0.70
$
(3.39
)
Weighted-average shares used in computing
net income (loss) per share
Basic
67,414,669
65,416,712
67,234,465
58,495,587
Diluted
69,196,421
65,416,712
69,991,340
58,495,587
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240222435442/en/
Media Amylyx Media Team +1 (857) 799-7274
amylyxmediateam@amylyx.com Investors Lindsey Allen Amylyx
Pharmaceuticals, Inc. +1 (857) 320-6244 Investors@amylyx.com
Amylyx Pharmaceuticals (NASDAQ:AMLX)
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