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Item 7.01 – Regulation FD Disclosure.
On September 28, 2023, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing that the U.S. Food and Drug Administration (FDA) has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. A copy of this press release is attached hereto as Exhibit 99.1. In connection with this announcement, the Company will host a conference call on September 28, 2023 to discuss the FDA approval. A copy of the conference call presentation materials is attached hereto as Exhibit 99.2. Both exhibits are incorporated herein by reference.
The information in this Item 7.01, including Exhibits 99.1 and 99.2, is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Act”), or otherwise subject to the liabilities of that Section. The information in this Item 7.01, including Exhibits 99.1 and 99.2, shall not be incorporated by reference into any registration statement or other document pursuant to the Act.
Item 9.01 Financial Statements and Exhibits
(d) Exhibits:
| Exhibit No. | Description | |
| 99.1 | Press Release dated September 28, 2023 | |
| 99.2 | September 28, 2023 Presentation Materials | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) |
Signature Page
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| AMICUS THERAPEUTICS, INC. | ||
| Date: September 28, 2023 | By: | /s/ Ellen S. Rosenberg |
| Name: Ellen S. Rosenberg | ||
| Title: Chief Legal Officer and Corporate Secretary | ||
Exhibit 99.1
Amicus Therapeutics Announces FDA Approval and Launch of New Treatment for Pompe Disease
Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) Approved in ERT-Experienced Adults
First and Only Two-Component Therapy for Eligible Adults Living with Late-onset Pompe Disease
Amicus Therapeutics to Host Conference Call Today at 12:00 p.m. ET
PHILADELPHIA, PA, September 28, 2023 -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscle cells and glycogen buildup causes muscle damage. Disease severity ranges across a spectrum, with predominant manifestations such as skeletal muscle weakness and progressive respiratory involvement.1
Pombiliti + Opfolda is a unique two-component therapy. Pombiliti is a recombinant human GAA enzyme (rhGAA) naturally expressed with high levels of bis-M6P (Mannose 6-Phosphate), designed for increased uptake into muscle cells. Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood.
“Today’s FDA approval of Pombiliti and Opfolda is a testament to the power of science, medicine, and our passionate determination to improve the lives of people living with Pompe disease. This approval embodies our Amicus spirit, passion, and resilience and is a very meaningful step for the Pompe community. I am just so immensely proud of our team, and so very grateful to everyone who has worked to bring this medicine to this approval. Most especially to all of the people living with Pompe around the world,” said John F. Crowley, Executive Chairman of Amicus Therapeutics, Inc.
Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “The FDA approval of Pombiliti and Opfolda is a major milestone for Amicus. We are grateful to the Pompe community, particularly the patients, caregivers, families, researchers, and physicians who have contributed to the development process through their commitment to our clinical studies. Today’s approval is also a testament to Team Amicus’ extraordinary dedication to patients and our ability to execute on our vision to bring new therapies to the rare disease community. Our highly experienced team is ready to launch this medicine in the U.S., and we look forward to rapidly bringing this new treatment regimen to all eligible adults living with late-onset Pompe disease who are not improving on their current ERT.”
The FDA approval was based on clinical data observed from the Phase 3 pivotal study (PROPEL), the only trial in LOPD to study ERT-experienced participants in a controlled setting.
“The Pompe community continues to face unmet need and limited treatment options. This two-component therapy is an important new treatment for those adults living with late-onset Pompe disease and not improving on current therapies. I am encouraged by the evidence generated over many years of clinical research studying this therapy for ERT-experienced patients living with late-onset Pompe disease,” said Tahseen Mozaffar, MD, Director of the Division of Neuromuscular Diseases in the Department of Neurology at the School of Medicine at UC Irvine and Director of the UC Irvine ALS and Neuromuscular Center, as well as an investigator for the PROPEL study.
“Today’s FDA approval is an extremely important step and acknowledges the potential of Pombiliti and Opfolda,” said Priya Kishnani, MD, Professor of Pediatrics and Chief of Medical Genetics at Duke University School of Medicine and an investigator for the PROPEL study. “I am grateful that eligible patients with late-onset Pompe disease in the U.S. will now have access to additional treatment options.”
“The FDA approval of Pombiliti and Opfolda represents a long-awaited day for people living with late-onset Pompe disease and advocating for additional therapeutic options,” said Tiffany House, President, Acid Maltase Deficiency Association. “Amicus’ long-standing commitment to the Pompe community and rare disease research has led to the development of an important therapy for the Pompe community because patients will now have options.”
Amicus Therapeutics will launch Pombiliti + Opfolda immediately in the U.S. The FDA previously granted Breakthrough Therapy designation for Pombiliti + Opfolda. Pombiliti + Opfolda has also been approved for the treatment of adults with LOPD in the European Union and the United Kingdom.
Amicus Assist® provides support to patients and caregivers in the U.S. and can help patients access their medication and identify possible sources of financial assistance. For more information on Amicus Assist, visit the Amicus Assist website at amicusassist.com, or please call +1-833-AMICUS-A (+1-833-264-2872).
SAFETY INFORMATION
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. See PI for complete Boxed Warning. CONTRADICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) LINK and full Prescribing Information for OPFOLDA (miglustat) LINK.
Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, September 28, 2023, at 12:00 p.m. ET to discuss the FDA approval. Participants and investors interested in accessing the call by phone will need to register using the online registration form. After registering, all phone participants will receive a dial-in number along with a personal PIN to access the event.
A live audio webcast and related presentation materials can also be accessed via the Investors section of the Amicus Therapeutics corporate website at ir.amicusrx.com. Web participants are encouraged to register on the website 15 minutes prior to the start of the call. An archived webcast and accompanying slides will be available on the Company's website shortly after the conclusion of the live event.
About Pompe Disease
Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. Pompe disease ranges from a rapidly deteriorating infantile form with significant impact to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle and progressive respiratory involvement. Late-onset Pompe disease can be severe and debilitating with progressive muscle weakness throughout the body that worsens over time, particularly skeletal muscles and muscles that control breathing.1
About Pombiliti + Opfolda
Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood.
INDICATIONS AND USAGE
POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. Further information about the Company can be found at: www.amicusrx.com, and can be followed on Twitter and LinkedIn.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to approval and commercialization plans for Pombiliti + Opfolda in the United States. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that we may not be successful in commercializing Pombiliti + Opfolda in the United States, the potential that public and commercial payors will not reimburse Pombiliti + Opfolda, the potential that we may not be able to manufacture or supply sufficient commercial products; and the potential that we will need additional funding to complete all of our commercialization and manufacturing activities. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2022, as well as our Quarterly Report on Form 10-Q for the quarter ended June 30, 2023, filed with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.
CONTACTS:
Investors:
Amicus Therapeutics
Andrew Faughnan
Vice President, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Amicus Therapeutics
Diana Moore
Head of Global Corporate Communications
dmoore@amicusrx.com
(609) 662-5079
| 1. | Kishnani PS, Steiner RD, Bali D, et al. Pompe disease diagnosis and management guideline. Genet Med 2006; 8: 267–88. |
FOLD-G
PP-AT-US-0008-0623
| U.S. Approval Call: Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) September 28, 2023 |
| 2 Forward-Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to approval and commercialization plans for Pombiliti and Opfolda in the United States. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this presentation may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that we may not be successful in commercializing Pombiliti + Opfolda in the United States, the potential that public and commercial payors will not reimburse Pombiliti + Opfolda, the potential that we may not be able to manufacture or supply sufficient commercial products; and the potential that we will need additional funding to complete all of our commercialization and manufacturing activities. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2022, as well as our Quarterly Report on Form 10-Q for the quarter ended June 30, 2023, filed the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof. |
| 3 Executive Summary New treatment for adults living with late-onset Pompe disease 3 Late-onset Pompe disease is a rare and fatal genetic disease that is significantly underdiagnosed Pombiliti + Opfolda is a differentiated two-component therapy for adults who are not improving on their current therapy Pombiliti + Opfolda global peak revenue potential of ~$1B by resetting treatment expectations Strong and broad patent portfolio with exclusivity to end of 2030s Body of evidence demonstrates measurable improvement in ERT-experienced adults Current global Pompe market is ~$1.2B expected to grow to >$1.8B by 2027 |
| 4 Deficiency of GAA leading to lysosomal glycogen accumulation and cellular dysfunction Diagnosed at different stages of life, from childhood to adulthood Symptoms include progressive muscle weakness, particularly skeletal and respiratory muscles, that worsens over time Respiratory failure and loss of motor function are major causes of morbidity and mortality ~5,000-10,000 people diagnosed globally; Significant underdiagnosis ~$1.2B+ global Pompe ERT sales1 Majority of patients on current standard of care decline after ~2 years Late-onset Pompe Disease (LOPD) Overview 1As reported, based on 12 months ended December 31, 2022. Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA) |
| 5 Pombiliti + Opfolda: An Innovative Approach to Pompe Disease Our scientists created a uniquely glycosylated and highly phosphorylated ERT that enhances targeting to key affected muscles and is co-administered with an enzyme stabilizer & Pombiliti + Opfolda is a two-component therapy combining cipaglucosidase alfa-atga, an ERT, with miglustat, an orally administered enzyme stabilizer & Pombiliti is expressed in a unique cell line producing a naturally glycosylated and highly phosphorylated enzyme that can be properly processed to its mature form, which are both required for greater lysosomal GAA activity1 cipaglucosidase alfa-atga miglustat 1Selvan et al. 2021, J Biol Chem 2021 Jan-Jun;296:100769 ERT: Enzyme Replacement Therapy |
| 6 U.S. Label Highlights Pombiliti + Opfolda approved in the United States & Pombiliti + Opfolda indicated for the treatment of adults with late-onset Pompe (LOPD) disease weighing ≥40 kg and who are not improving on their current enzyme replacement therapy & This indication is approved based on positive data from the Phase 3 pivotal study (PROPEL): – The only controlled trial to study the real-world population of both ERT-naïve and ERT-experienced participants |
| 7 Pombiliti + Opfolda U.S. Opportunity Alaska Hawaii Universally screened Not screened Pompe Disease Newborn Screening in 44 U.S. States2 & ~80% of the 800 treated patients in the U.S. are adults1 & Currently ~40 patients in the U.S. on Pombiliti + Opfolda through clinical trials are eligible for commercial product and are expected to transition & 88% of states screen for Pompe disease in newborns which continues to support diagnosis of Pompe disease 1 Amicus Data on File from Market Mapping 2 https://www.newsteps.org/resources/data-visualizations/newborn-screening-status-all-disorders U.S. Pompe market currently represents a sizeable market opportunity of >$500M1 7 |
| 8 Pombiliti + Opfolda Launch Activities Experienced and passionate rare disease commercial and medical organizations poised for second successful launch & Market mapping and account profiling completed & Sales team visiting top accounts in first 30 days & Promotional materials developed & Product labeled and available for sale within 2 weeks & Successful transition of all clinical trial patients by year-end & Market Access: Payer ad boards and pre-approval information exchange meetings completed & Patient hub: All case managers and patient education liaisons hired and trained Representative promotional materials ready for launch |
| 9 Pricing Promise Pombiliti + Opfolda U.S. Pricing & Combined price of Pombiliti + Opfolda slightly below current treatments & Pricing PROMISE: Amicus will limit price increases to CPI-U (consumer price index) & Pledge for a Cure: Amicus pledges to reinvest a portion of our profits into R&D of new treatments for Pompe disease until there is a cure & Provide world-class patient support services, including needs-based financial support globally to ensure all who need our therapies have access to them Our medicines must be fairly - Amicus Founding Belief priced and broadly accessible” 9 |
| 2023 Strategic Priorities Double-digit Galafold® revenue growth of 14-18% at CER1 Secure FDA, EMA, and MHRA approvals for Pombiliti + Opfolda Initiate successful global launches of Pombiliti + Opfolda Advance best-in-class, next-generation Fabry and Pompe pipeline programs and capabilities Maintain strong financial position on path to profitability 10 1 2 3 4 5 1CER: Constant Exchange Rates; 2023 Galafold revenue guidance utilizes actual exchange rate as of December 31, 2022 & & |
| Thank You |
