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Share Name | Share Symbol | Market | Type |
---|---|---|---|
Amicus Therapeutics Inc | NASDAQ:FOLD | NASDAQ | Common Stock |
Price Change | % Change | Share Price | Bid Price | Offer Price | High Price | Low Price | Open Price | Shares Traded | Last Trade | |
---|---|---|---|---|---|---|---|---|---|---|
-0.01 | -0.10% | 10.42 | 10.25 | 11.14 | 10.61 | 10.365 | 10.51 | 2,576,847 | 22:54:08 |
Patients treated with AT-GAA for 24 months showed persistent and durable effects on six-minute walk test (6MWT) distance and other measures of motor function and muscle strength, stability or increases in forced vital capacity (FVC), and reductions in biomarkers of muscle damage and disease substrate. Consistent with these 24-month results, positive impacts on the same measures of motor and pulmonary function and key biomarkers were also observed after 3-15 months of treatment in an additional group of six ambulatory ERT switch patients that had been on standard of care ERT for at least seven years prior to switching to AT-GAA. (Cohort 4).
These clinical results are being featured at the 24th International Annual Congress of the World Muscle Society in an oral platform presentation on Friday October 4, 2019 at 10:00am CEST (4:00am EDT). The presentation will be given by Professor Benedikt Schoser, senior consultant at the Friedrich-Baur-Institute, Dept. of Neurology at the Ludwig-Maximilians-University of Munich, Germany and Principal Investigator in the ATB200-02 study. These results are also available in a presentation on the Amicus corporate website and will be highlighted during the Amicus Analyst Day on October 10, 2019.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics stated, “We are very pleased to report the latest data for AT-GAA. Collectively these data continue to represent meaningful and durable improvements in functional outcomes, in addition to persistent reductions in key biomarkers of muscle damage and disease substrate. Compared to what is known about the natural history of both untreated and ERT-experienced patients, these results give great hope that AT-GAA has the potential to become the new standard of care for people living with Pompe. These results also provide further support and confidence in the overall study design and powering of our ongoing pivotal PROPEL study.”
Dr. Schoser stated, “There is a clear need for a new treatment option to address multiple aspects of Pompe disease across a broad spectrum of Pompe patients, including previously untreated and ERT-switch patients as well as non-ambulatory patients. The twenty four month results from this Phase 1/2 clinical study of AT-GAA continue to demonstrate a robust effect in adult people living with Pompe disease, including improvements in six minute walk distance among ambulatory ERT-switch and ERT-naïve participants and sustained positive changes in arm and shoulder strength among the non-ambulatory ERT-switch patients. These new data in the Cohort 4 patients are particularly impressive showing the potential for AT-GAA to change the course of the disease in these patients. I look forward to continuing to follow patients from this Phase 2 study in addition to the ongoing PROPEL pivotal study.”
ATB200-02 Study Data Highlights in ERT-Switch and ERT-Naive Patients Out to Month 24
Cohort 1 – 3 (n=22)Complete 24-month data was presented for 18 out of 22 patients enrolled in the initial three study cohorts, including ERT-switch ambulatory patients (n=11), ERT-switch non-ambulatory patients (n=6), and ERT-naïve patients (n=5).
Functional Outcomes (n=18)*: Muscle function improved in 16 out of 18 patients at 24 months.
Cohort 4 (n=6)A fourth cohort of six additional ERT-switch ambulatory patients was subsequently added to the study. At the time of the data analysis, five patients have available data at month 6. The last available timepoint includes all six patients after 3-15 months of treatment, with one subject at Month 3, two subjects at Month 6, two subjects at Month 12 and one subject at Month 15.
Safety, Tolerability and Pharmacokinetics/Pharmacodynamics (PK/PD) in Cohorts 1-4 (n=28)
AT-GAA Development and Regulatory StrategyThe Company’s strategy is to enhance the body of clinical data for AT-GAA in ongoing clinical studies, including the pivotal study (PROPEL, also referred to as ATB200-03) to deliver this potential new therapy to as many people living with Pompe disease as soon as possible. Based on feedback from US and EU regulatory authorities, Amicus expects the PROPEL study to support approval for a broad indication, including ERT-switch and treatment-naïve patients.
Anticipated Pompe Milestones:
*Discontinuations were as follows: Cohort 1 (n=2 out of 11): travel burden (after 18 weeks), withdrawal of consent (not related to treatment) (after month 18). Cohort 2 (n=1 out of 6): IAR (after month 18 - 4 IARs, generally urticarial rash, with nasopharyngeal edema on 1 occasion. Baseline values not obtained in one patient in Cohort 2. No discontinuations in Cohorts 3 or 4.
About ATB200-02 Clinical StudyThe primary objectives of the open-label ATB200-02 clinical study are to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ATB200/AT2221 over an 18-week primary treatment period followed by a long-term extension. Sixteen clinical sites in five countries participated in the ATB200-02 clinical study. The study originally enrolled a total of 20 patients across three patient cohorts: ambulatory ERT-experienced (Cohort 1, n=11), non-ambulatory ERT-experienced (Cohort 2, n=4) and ERT-naïve (Cohort 3, n=5); in addition two more patients were enrolled in cohort 2. A fourth cohort of 6 ambulatory ERT-switch patients was also been enrolled, adding to the patient data in the ambulatory ERT-switch population. Patients in Cohort 1 received escalating doses of ATB200 (5, 10, 20 mg/kg), followed by 3 doses of 20 mg/kg ATB200 plus low dose AT2221, followed by ongoing doses of 20 mg/kg ATB200 plus high dose AT2221. Patients in Cohorts 2, 3, and 4 all receive 20 mg/kg ATB200 plus high dose AT2221.
About AT-GAAAT-GAA is an investigational therapy that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In preclinical studies, AT-GAA was associated with increased tissue enzyme levels, reduced glycogen levels in muscle, and improvements in muscle strength. A global Phase 1/2 study (ATB200-02) is ongoing to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of AT-GAA.
Amicus has also initiated a global Phase 3 clinical study (ATB200-03, or PROPEL) of AT-GAA in adult patients with late onset Pompe disease. PROPEL is a 52-week, double-blind randomized study designed to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa, an enzyme replacement therapy (ERT). More information, including a list of participating sites, is available at www.clinicaltrials.gov: NCT03729362.
About Pompe DiseasePompe disease is an inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA leads to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating, and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.
About Amicus TherapeuticsAmicus 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 metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company’s website at www.amicusrx.com, and follow us on Twitter and LinkedIn.
Forward-Looking StatementsThis press release contains "forward- looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to preliminary data from a global Phase 1/2 study to investigate AT-GAA for the treatment of Pompe and the potential implications on these data for the future advancement and development of AT-GAA. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” "confidence," "encouraged," “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “could,” and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the preliminary data based on a small patient sample and reported before completion of the study will not be predictive of future results, that results of additional preliminary data or data from the completed study or any future study will not yield results that are consistent with the preliminary data presented, that the Company will be not able to demonstrate the safety and efficacy of AT-GAA, that later study results will not support further development, or even if such later results are favorable, that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully commercialize AT-GAA. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2018 and Quarterly Report on 10-Q for the Quarter ended June 30, 2019. As a consequence, actual results may differ materially from those set forth in this press release. You are cautioned not to place undue reliance on these forward looking statements, which speak only of the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof.
CONTACTS:
Investors:Amicus TherapeuticsSara Pellegrino, IRCVice President, Investor Relations spellegrino@amicusrx.com(609) 662-5044
Media:Christopher Byrne Executive Director, Corporate Communications cbyrne@amicusrx.com609-662-2798
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