Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced financial results for the quarter ended September 30, 2022.

Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, 'We remain laser focused on advancing our key strategic priorities for the year. The strong commercial uptake of Galafold globally is on track to deliver double-digit operational growth in 2022. In addition, we are making great progress towards gaining regulatory approvals of AT-GAA for people living with Pompe disease around the world, and are well underway with our launch readiness. Through our continued commercial success with Galafold, our highly anticipated approvals of AT-GAA in the months ahead, together with our careful expense management, we believe Amicus is well positioned to drive significant value for shareholders and further our mission to bring innovative new medicines to people living with rare diseases.'

Third Quarter 2022 Financial Results

Total revenue in the third quarter 2022 was $81.7 million, a year-over-year increase of 3% from total revenue of $79.5 million in the third quarter of 2021. On a constant currency basis, third quarter 2022 total revenue growth was 14%. Reported quarterly revenue was offset by a negative currency impact of $8.6 million, or 11%.

Cash, cash equivalents, and marketable securities totaled $354.7 million on September 30, 2022, compared to $482.5 million at December 31, 2021.

Total GAAP operating expenses of $102.1 million for the third quarter 2022 decreased 7% as compared to $110.2 million for the third quarter 2021.

Total non-GAAP operating expenses of $85.5 million for the third quarter of 2022 decreased 9% as compared to $93.6 million in the third quarter of 2021, reflecting the reprioritization of the gene therapy portfolio.3

Net loss was $33.3 million, or $0.12 per share, compared to a net loss of $50.3 million, or $0.19 per share, for the third quarter 2021, demonstrating that the Company is progressing on the path towards non-GAAP profitability.

2022 Financial Guidance

For the full-year 2022, the Company anticipates total Galafold revenue growth between 15 and 20% at CER1 driven by continued underlying demand from both switch and treatment-naive patients, geographic expansion, the continued diagnosis of new Fabry patients and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. Applying average October 2022 exchange rates, the negative currency impact on full-year 2022 Galafold reported sales would be approximately 9%.

Amicus is updating its non-GAAP operating expense guidance for the full-year 2022 from $470 million to $485 million to $430 million to $440 million, driven by prudent expense management offset by continued investment in the global Galafold launch, AT-GAA clinical studies and pre-launch activities, in addition to certain non-recurring costs for manufacturing to support the global launch of AT-GAA and committed obligations for the gene therapy portfolio. In 2023, Amicus expects non-GAAP operating expense at a level below 2021.4

About Galafold

Galafold (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body's own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of Fabry patients may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan.

U.S. INDICATIONS AND USAGE

Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.

This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

U.S. IMPORTANT SAFETY INFORMATION

ADVERSE REACTIONS

The most common adverse reactions reported with Galafold (10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.

USE IN SPECIFIC POPULATIONS

There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus.

It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.

Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.

The safety and effectiveness of Galafold have not been established in pediatric patients.

To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

EU Important Safety Information

Treatment with Galafold should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. Galafold is not recommended for use in patients with a nonamenable mutation.

Galafold is not intended for concomitant use with enzyme replacement therapy.

Galafold is not recommended for use in patients with Fabry disease who have severe renal impairment (

(C) 2022 Electronic News Publishing, source ENP Newswire