Amylyx Pharmaceuticals Reports Third Quarter 2023 Financial Results
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Third quarter 2023 product revenue of
$102.7 million ; bringing total product revenue to$272.3 million in the first three full quarters ofU.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 atSeptember 30, 2023 -
Management to host a conference call and webcast today at
8:00 a.m. Eastern Time
“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
Third Quarter 2023 and Recent Business Highlights:
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Continued progress of commercial launches of RELYVRIO® in the
U.S. and ALBRIOZA™ inCanada , also known as AMX0035. Net product revenue for the three months endedSeptember 30, 2023 was$102.7 million , compared to net product revenue of$98.2 million for the three months endedJune 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 2023American 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 inOctober 2023 .
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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. - 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.
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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.
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An update on kinetic profiling experiments of AMX0114 was presented as well as results from a collaboration with Dr.
Full posters can be found in the “Publications” section of the Amylyx website.
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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 acrossNorth America ,Europe , andJapan , with study initiation anticipated by the end of 2023 starting inthe United States .
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The Committee for Medicinal Products for Human Use (CHMP) of theEuropean Medicines Agency (EMA) confirmed its initial negative opinion adopted inJune 2023 on the Marketing Authorisation Application (MAA) for AMX0035 for the treatment of ALS in theEuropean Union (EU). The CHMP opinion will be forwarded to theEuropean 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 thePHOENIX Phase 3 clinical trial. IfPHOENIX 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
Net product revenue was
Research and development expenses were
Selling, general and administrative expenses were
Net income for the three months ended
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, statements regarding a final decision from the
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CONDENSED CONSOLIDATED BALANCE SHEETS |
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UNAUDITED |
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(in thousands) |
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Assets |
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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 |
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Accounts payable and accrued expenses |
|
$ |
43,416 |
|
|
$ |
44,569 |
|
Other liabilities |
|
|
4,768 |
|
|
|
6,277 |
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Total liabilities |
|
|
48,184 |
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|
|
50,846 |
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Stockholders’ equity |
|
|
418,400 |
|
|
|
340,607 |
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Total liabilities, redeemable convertible preferred stock and stockholders' equity |
|
$ |
466,584 |
|
|
$ |
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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Nine Months 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 |
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$ |
102,693 |
|
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$ |
345 |
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$ |
272,337 |
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$ |
345 |
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Operating expenses: |
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Cost of sales |
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5,218 |
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|
|
172 |
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|
|
16,081 |
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|
|
172 |
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Research and development |
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|
30,037 |
|
|
|
24,914 |
|
|
|
83,273 |
|
|
|
70,637 |
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Selling, general and administrative |
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|
48,718 |
|
|
|
29,940 |
|
|
|
136,115 |
|
|
|
86,284 |
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Total operating expenses |
|
|
83,973 |
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|
|
55,026 |
|
|
|
235,469 |
|
|
|
157,093 |
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Income (loss) from operations |
|
|
18,720 |
|
|
|
(54,681 |
) |
|
|
36,868 |
|
|
|
(156,748 |
) |
Other income, net |
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3,691 |
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|
|
800 |
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|
|
10,953 |
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|
|
1,272 |
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Income (loss) before income taxes |
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|
22,411 |
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|
|
(53,881 |
) |
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|
47,821 |
|
|
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(155,476 |
) |
Provision (benefit) for income taxes |
|
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1,518 |
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|
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(125 |
) |
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|
3,281 |
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|
|
195 |
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Net income (loss) |
|
$ |
20,893 |
|
|
$ |
(53,756 |
) |
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$ |
44,540 |
|
|
$ |
(155,671 |
) |
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Net income (loss) per share attributable to common stockholders |
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Basic |
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$ |
0.31 |
|
|
$ |
(0.92 |
) |
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$ |
0.66 |
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|
$ |
(2.77 |
) |
Diluted |
|
$ |
0.30 |
|
|
$ |
(0.92 |
) |
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$ |
0.63 |
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|
$ |
(2.77 |
) |
Weighted-average shares used in computing net income (loss) per share attributable to common stockholders |
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Basic |
|
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67,414,669 |
|
|
|
58,533,226 |
|
|
|
67,124,407 |
|
|
|
56,163,194 |
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Diluted |
|
|
69,748,547 |
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|
|
58,533,226 |
|
|
|
70,143,659 |
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|
|
56,163,194 |
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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
+1 (857) 320-6244
Investors@amylyx.com
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