The following discussion should be read in conjunction with the unaudited
Consolidated Financial Statements and the notes thereto included in this
Quarterly Report on Form 10-Q and the audited Consolidated Financial Statements
and the notes thereto included in our Annual Report on Form 10-K for the fiscal
year ended
Overview
We are a global, patient-dedicated biotechnology company focused on discovering,
developing, and delivering novel medicines for rare diseases. We have a
portfolio of product opportunities including the first, oral monotherapy for
Fabry disease that has achieved widespread global approval and a differentiated
biologic for Pompe disease that is under review with the
The cornerstone of our portfolio is Galafold® (also referred to as
"migalastat"), the first and only approved oral precision medicine for people
living with Fabry disease who have amenable genetic variants. Migalastat is
currently approved under the trade name Galafold® in
The lead biologics program of our pipeline is Amicus Therapeutics GAA ("AT-GAA",
also known as ATB200/AT2221, or cipaglucosidase alfa/miglustat), a novel,
two-component, potential best-in-class treatment for Pompe disease. In
Our Strategy
Our strategy is to create, manufacture, test, and deliver the highest quality medicines for people living with rare diseases through internally developed, jointly developed, acquired, or in-licensed products and product candidates that have the potential to obsolete current treatments, provide significant benefits to patients, and be first- or best-in-class. We are leveraging our global capabilities to develop and broaden our lead franchises in Fabry and Pompe disease, with focused discovery work on next generation therapies and novel platform technologies.
Our operations have not been significantly impacted by the novel coronavirus ("COVID-19") pandemic thus far. The Company continued to observe increased lag times between patient identification and Galafold® initiation due to the resurgence of COVID-19 in certain markets. We have maintained operations in all geographies, secured our global supply chain for our commercial and clinical products, as well as maintained the operational integrity of our clinical trials, with minimum disruptions. Our ability to continue to operate without any significant disruptions will depend on the continued health of our employees, the ongoing demand for Galafold® and the continued operation of our global supply chain. We have continued to provide uninterrupted access to medicines for those in need of treatment, while prioritizing the health and safety of our global workforce. However, our results of operations in future periods may be negatively impacted by unknown future impacts from the COVID-19 pandemic.
Highlights of our progress include:
•Commercial and regulatory success in Fabry disease. For the three months ended
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continue to see a significant increase in patients from a growing and very wide prescriber base. Across all markets, we see a high rate of compliance and adherence to this oral treatment option.
•Pompe disease clinical program milestones. In
•Pipeline advancement and growth. We are leveraging our global capabilities to develop and broaden our lead franchises in Fabry and Pompe disease, with focused discovery work on next generation therapies and novel platform technologies.
•Manufacturing. We have managed our clinical and commercial supply chains during the COVID-19 pandemic such that as of the date hereof we have not experienced supply impacts. We have been able to continue to meet required commercial demand for Galafold® as well as supply our ongoing Pompe disease clinical studies and access programs including EAMS without interruption. We have secured supply for our continued needs for the Pompe disease program through a long-term supply agreement with Wuxi Biologics. The agreement allows for the continuous manufacture of our biologic to support future clinical needs and our anticipated commercial requirements should we garner regulatory approvals as planned. We have contracts in place to supply miglustat, our small molecule component of AT-GAA, to support both clinical and future commercial requirements.
•Financial strength. Total cash, cash equivalents, and marketable securities as
of
Our Commercial Product and Product Candidates
Galafold® (Migalastat HCl) for Fabry Disease
Our oral precision medicine Galafold® was granted accelerated approval by the
FDA in
As an orally administered monotherapy, Galafold® is designed to bind to and stabilize an endogenous alpha-galactosidase A ("alpha-Gal A") enzyme in those patients with genetic variants identified as amenable in a GLP cell-based amenability assay. Galafold® is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic variants, and at this time, it is not intended for concomitant use with ERT.
In early 2022, we announced the issuance of six additional patents, including
the new
Next Generation for Fabry Disease
We are committed to continued innovation for all people living with Fabry disease. Our pipeline includes a Fabry gene therapy.
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Novel ERT for Pompe Disease
We are leveraging our biologics capabilities to develop AT-GAA, a novel treatment paradigm for Pompe disease. AT-GAA consists of a uniquely engineered rhGAA enzyme, ATB200, or cipaglucosidase alfa, with an optimized carbohydrate structure to enhance lysosomal uptake, administered in combination with AT2221, or miglustat, that functions as an enzyme stabilizer. Miglustat binds to and stabilizes ATB200, or cipaglucosidase alfa, preventing inactivation of rhGAA in circulation to improve the uptake of active enzyme in key disease-relevant tissues, resulting in increased clearance of accumulated substrate, glycogen. Miglustat is not an active ingredient that contributes directly to substrate reduction ("glycogen").
In
In
In addition, we are conducting ongoing clinical studies in pediatric patients for both LOPD and infantile-onset Pompe disease ("IOPD") populations.
Next Generation for Pompe Disease
As part of our long-term commitment to provide multiple solutions to address the significant unmet needs of the Pompe disease community, we are also continuing discovery for next-generation genetic medicines for Pompe disease.
CDKL5 Deficiency Disorder
We are researching a potential first-in-class genetic medicine for CDKL5
deficiency disorder consisting of a CDKL5 protein engineered for cross
correction, delivered as either a protein replacement or as a gene therapy
through our collaboration with Penn. We are collaborating with the
Additional Next Generation Programs
We have a number of additional gene therapies in clinical and preclinical development, including potential gene therapies in multiple forms of Batten disease.
Strategic Alliances and Arrangements
We will continue to evaluate business development opportunities as appropriate to build stockholder value and provide us with access to the financial, technical, clinical, and commercial resources necessary to develop and market technologies or products with a focus on rare and orphan diseases. We are exploring potential collaborations, alliances, and other business development opportunities on a regular basis. These opportunities may include business combinations, partnerships, the strategic out-licensing of certain assets, or the acquisition of preclinical-stage, clinical-stage, or marketed products or platform technologies consistent with our strategic plan to develop and provide therapies to patients living with rare and orphan diseases.
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