- Third quarter 2023 product revenue of $102.7 million; bringing
total product revenue to $272.3 million in the first three full
quarters of U.S. launch
- Strong financial position supported by $20.9 million of net
income during the third quarter of 2023 and cash, cash equivalents
and short-term investments of $355.0 million at September 30,
2023
- Management to host a 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 third quarter
ended September 30, 2023.
“We are encouraged by the strong launch of RELYVRIO over the
last year. We remain confident in the design and execution of our
Phase 3 PHOENIX trial and now expect topline data in the second
quarter of 2024,” said Joshua Cohen and Justin Klee, co-CEOs of
Amylyx. “We are incredibly pleased with the rapid adoption of
RELYVRIO at major ALS centers and focused on the work that we still
need to do to reach more people living with ALS as we work towards
our goal of transforming the way that ALS is treated.”
Third Quarter 2023 and Recent Business Highlights:
- Continued progress of commercial launches of RELYVRIO® in
the U.S. and ALBRIOZA™ in Canada, also known as AMX0035. Net
product revenue for the three months ended September 30, 2023
was $102.7 million, compared to net product revenue of $98.2
million for the three months ended June 30, 2023.
- A post hoc survival analysis comparing the CENTAUR clinical
trial to historical clinical trial control was published in October
2023 in the Annals of Clinical and Translational Neurology; results
were consistent with the data previously presented at the 2023
American Academy of Neurology Annual Meeting. 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.
- RELYVRIO and AMX0114, the Company’s investigational
antisense oligonucleotide targeting calpain-2, data were
presented at 22nd Annual Northeast ALS Consortium (NEALS) meeting
in October 2023.
- 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.
- Additionally, the Company presented an update on work to
develop a composite diagnostic biomarker for ALS. The goal of this
program is to create a tool that allows for earlier diagnosis of
ALS which may result in earlier treatment and better outcomes.
- A poster detailing survey results on real-world experiences
related to RELYVRIO’s taste was also presented. Findings included
that taste did not appear to impact survey participants’
willingness to take, adherence to, or planned future use of
RELYVRIO.
- The Company also presented preliminary experience with RELYVRIO
in a U.S. Expanded Access Program (EAP). Despite differences in
study populations, the safety and tolerability of AMX0035 in the
EAP were consistent with the AMX0035 arm from CENTAUR.
- Additionally, Amylyx presented a poster outlining ongoing and
planned RELYVRIO studies, including two collaborative real-world
studies of RELYVRIO in people living with ALS currently
underway.
- Lastly, findings from a study evaluating the use of RELYVRIO
with different types of feeding tubes and containers were
presented, supporting that RELYVRIO can be used in a variety of
feeding tubes and dosing container combinations.
Full posters can be found in the “Publications” section of the
Amylyx website.
- Amylyx presented the clinical trial design of ORION, a Phase
3 global study of AMX0035 in Progressive Supranuclear Palsy (PSP),
at the Neuro2023 PSP and CBD International Research Symposium in
October 2023. 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, with study initiation anticipated by
the end of 2023 starting in the United States.
- The Committee for Medicinal Products for Human Use (CHMP) of
the European Medicines Agency (EMA) confirmed its initial negative
opinion adopted in June 2023 on the Marketing Authorisation
Application (MAA) for AMX0035 for the treatment of ALS in the
European Union (EU). The CHMP opinion will be forwarded to the
European Commission, who will adopt the final decision on this
application anticipated by the end of 2023. The Company continues
to focus on the completion of the PHOENIX Phase 3 clinical trial.
If PHOENIX is supportive, the Company plans to seek approval in the
EU as quickly as possible. Topline results are anticipated in Q2
2024.
- Amylyx continues to progress R&D programs. The
Company continues to expect data from the Phase 2 clinical trial of
AMX0035 in Wolfram syndrome in 2024. Additionally, Amylyx has been
working on a new taste-masked formulation of RELYVRIO for several
years. This formulation may allow for new intellectual property.
The Company is planning to file an IND and conduct Phase 1 testing
for this innovative formulation in 2024.
Financial Results for the Third Quarter Ended September 30,
2023
Net product revenue was $102.7 million and cost of sales were
$5.2 million for the three months ended September 30, 2023,
compared to net product revenue of $0.3 million and cost of sales
of $0.2 million for the three months ended September 30, 2022. The
increase was primarily driven by units of RELYVRIO sold in the U.S.
following regulatory approval in late September 2022.
Research and development expenses were $30.0 million for the
three months ended September 30, 2023, compared to $24.9 million
for the same period in 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
on AMX0035 for the treatment of PSP to support the initiation of
the Phase 3 ORION trial, and an increase in preclinical development
activities.
Selling, general and administrative expenses were $48.7 million
for the three months ended September 30, 2023, compared to $29.9
million for the same period in 2022. The increase was primarily
driven by higher personnel-related expenses due to added headcount
to support the Company’s launch, commercialization initiatives, and
operations as a public company.
Net income for the three months ended September 30, 2023 was
$20.9 million, or $0.30 on a fully diluted per share basis,
compared to a net loss of $53.8 million, or $0.92 on a fully
diluted per share basis for the same period in 2022.
Cash, cash equivalents, and short-term investments were $355.0
million at September 30, 2023, compared to $357.3 million at June
30, 2023.
Investor Conference Call Information
Amylyx’ management team will host a conference call and webcast
today, November 9, 2023, at 8:00 a.m. ET to discuss financial
results and provide an update on the business. To access the
conference call, please dial (833) 816-1395 (U.S.) or +1 (412)
317-0488 (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 and EMEA. 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, statements regarding a
final decision from the European Commission regarding whether to
approve AMX0035 for the treatment of ALS in Europe; the potential
of AMX0035 (sodium phenylbutyrate and taurursodiol) as a treatment
for ALS and other neurodegenerative diseases including Wolfram
syndrome and PSP; the ongoing commercialization of RELYVRIO and
ALBRIOZA; expectations regarding the timing of initiation of the
Company’s Phase 3 ORION trial of AMX0035 for the treatment of PSP
and of the results of the Company’s Phase 2 HELIOS trial of AMX0035
for the treatment of Wolfram syndrome; the timing of the results of
the Company’s Phase 3 PHOENIX trial of AMX0035 for the treatment of
ALS; s expectations regarding the results of the Phase 3 PHOENIX
trial and of the potential for future approval of AMX0035 in the
EU; the timing of filing IND applications for AMX0114 and for a new
formation of RELYVRIO; expectations regarding 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; that data from later-stage
trials may not reflect data from earlier-stage trials, including
other indications; regulatory developments; expectations regarding
the timing of a decision from the European Commission regarding
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’ Quarterly
Report on Form 10-Q for the quarter ended June 30, 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, except as required by
law.
AMYLYX PHARMACEUTICALS,
INC.
CONDENSED CONSOLIDATED BALANCE
SHEETS
UNAUDITED
(in thousands)
September 30, 2023
December 31, 2022
Assets
Cash, cash equivalents and short-term
investments
$
355,045
$
346,945
Accounts receivable, net
29,353
15,306
Inventories
56,703
9,769
Prepaid expenses and other current
assets
17,338
10,113
Other assets
8,145
9,320
Total assets
$
466,584
$
391,453
Liabilities, Redeemable Convertible
Preferred Stock and Stockholders’ Equity
Accounts payable and accrued expenses
$
43,416
$
44,569
Other liabilities
4,768
6,277
Total liabilities
48,184
50,846
Stockholders’ equity
418,400
340,607
Total liabilities, redeemable convertible
preferred stock and stockholders' equity
$
466,584
$
391,453
AMYLYX PHARMACEUTICALS,
INC.
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS
UNAUDITED
(in thousands, except share
and per share data)
Three Months Ended September
30,
Nine Months Ended September
30,
2023
2022
2023
2022
Product revenue, net
$
102,693
$
345
$
272,337
$
345
Operating expenses:
Cost of sales
5,218
172
16,081
172
Research and development
30,037
24,914
83,273
70,637
Selling, general and administrative
48,718
29,940
136,115
86,284
Total operating expenses
83,973
55,026
235,469
157,093
Income (loss) from operations
18,720
(54,681
)
36,868
(156,748
)
Other income, net
3,691
800
10,953
1,272
Income (loss) before income taxes
22,411
(53,881
)
47,821
(155,476
)
Provision (benefit) for income taxes
1,518
(125
)
3,281
195
Net income (loss)
$
20,893
$
(53,756
)
$
44,540
$
(155,671
)
Net income (loss) per share attributable
to common stockholders
Basic
$
0.31
$
(0.92
)
$
0.66
$
(2.77
)
Diluted
$
0.30
$
(0.92
)
$
0.63
$
(2.77
)
Weighted-average shares used in computing
net income (loss) per share attributable to common stockholders
Basic
67,414,669
58,533,226
67,124,407
56,163,194
Diluted
69,748,547
58,533,226
70,143,659
56,163,194
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231109623562/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
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