Amylyx Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results
- 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
“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
“We look forward to sharing topline data from our Phase 3 PHOENIX trial during or before the second quarter of 2024.
Full Year 2023 and Recent Business Highlights:
- Commercial launches of RELYVRIO® in the
U.S. and ALBRIOZA™ inCanada , 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 endedDecember 31, 2023 was$108.4 million , compared to net product revenue of$102.7 million for the three months endedSeptember 30, 2023 . The vast majority of Amylyx’ net product revenue is generated in theU.S. ; revenue inCanada and from named patient sales programs in international markets represented an important source of revenue growth in three months endedDecember 31, 2023 relative to the Company’s financial results for the three months endedSeptember 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 , andJapan . The first participant was dosed inDecember 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 the second half of 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. InNovember 2023 , the Company announced the appointment ofCamille 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 andDan Monahan joined as General Manager and Head ofU.S. Commercial Markets.Ms. Arsenault has over 30 years of human resources leadership experience, including in the biopharmaceutical sector, most recently fromSunovion 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 theUniversity 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
Net product revenue: Net product revenue was
Cost of Sales: Cost of sales were
R&D Expenses: Research and development expenses for the fourth quarter of 2023 were
SG&A Expenses: Selling, general, and administrative expenses for the fourth quarter of 2023 were
Net Income: Net income for the three months ended
Cash Position: Cash, cash equivalents, and short-term investments were
Investor Conference Call Information
Amylyx’ management team will host a conference call and webcast today,
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,
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
RELYVRIO® (sodium phenylbutyrate and taurursodiol) Safety Information for
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
About
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
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CONDENSED CONSOLIDATED BALANCE SHEETS |
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UNAUDITED |
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(in thousands) |
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2023 |
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2022 |
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Assets |
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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 |
|
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$ |
391,453 |
|
Liabilities and Stockholders’ Equity |
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Accounts payable and accrued expenses |
|
$ |
79,785 |
|
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$ |
44,569 |
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Other liabilities |
|
|
4,237 |
|
|
|
6,277 |
|
Total liabilities |
|
|
84,022 |
|
|
|
50,846 |
|
Stockholders’ equity |
|
|
433,432 |
|
|
|
340,607 |
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Total liabilities, redeemable convertible preferred stock and stockholders' equity |
|
$ |
517,454 |
|
|
$ |
391,453 |
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CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
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UNAUDITED |
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(in thousands, except share and per share data) |
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Three Months Ended |
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Year Ended |
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2023 |
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2022 |
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2023 |
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2022 |
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Product revenue, net |
|
$ |
108,449 |
|
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$ |
21,885 |
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$ |
380,786 |
|
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$ |
22,230 |
|
Operating expenses: |
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Cost of sales |
|
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9,360 |
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|
2,821 |
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|
25,441 |
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|
|
2,993 |
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Research and development |
|
|
44,914 |
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|
|
22,813 |
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|
|
128,187 |
|
|
|
93,450 |
|
Selling, general and administrative |
|
|
52,241 |
|
|
|
40,844 |
|
|
|
188,356 |
|
|
|
127,128 |
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Total operating expenses |
|
|
106,515 |
|
|
|
66,478 |
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|
|
341,984 |
|
|
|
223,571 |
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Income (loss) from operations |
|
|
1,934 |
|
|
|
(44,593 |
) |
|
|
38,802 |
|
|
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(201,341 |
) |
Other income, net |
|
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4,542 |
|
|
|
2,468 |
|
|
|
15,495 |
|
|
|
3,740 |
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Income (loss) before income taxes |
|
|
6,476 |
|
|
|
(42,125 |
) |
|
|
54,297 |
|
|
|
(197,601 |
) |
Provision for income taxes |
|
|
1,745 |
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|
|
579 |
|
|
|
5,026 |
|
|
|
774 |
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Net income (loss) |
|
$ |
4,731 |
|
|
$ |
(42,704 |
) |
|
$ |
49,271 |
|
|
$ |
(198,375 |
) |
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Net income (loss) per share |
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||||
Basic |
|
$ |
0.07 |
|
|
$ |
(0.65 |
) |
|
$ |
0.73 |
|
|
$ |
(3.39 |
) |
Diluted |
|
$ |
0.07 |
|
|
$ |
(0.65 |
) |
|
$ |
0.70 |
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|
$ |
(3.39 |
) |
Weighted-average shares used in computing net income (loss) per share |
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Basic |
|
|
67,414,669 |
|
|
|
65,416,712 |
|
|
|
67,234,465 |
|
|
|
58,495,587 |
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Diluted |
|
|
69,196,421 |
|
|
|
65,416,712 |
|
|
|
69,991,340 |
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|
|
58,495,587 |
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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
+1 (857) 320-6244
Investors@amylyx.com
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