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Item 8.01. Other Events
On October 24, 2019, Amicus Therapeutics, Inc. (the "Company") issued a press release announcing additional positive interim results from its CLN6 Batten disease gene therapy program licensed from the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital. A copy of this press release is attached hereto as Exhibit 99.1. The results are also featured in a poster presentation at the 48th Annual Meeting of the Child Neurology Society to be held October 23-26, 2019 in Charlotte, North Carolina. A copy of this poster presentation is attached hereto Exhibit 99.2. Both exhibits are incorporated herein by reference.
Item 9.01. Financial Statements and Exhibits Exhibits:
| Exhibit No. | Description | |
| 99.1 | October 24, 2019 Press Release | |
| 99.2 | 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: October 24, 2019 | By: | /s/ Ellen S. Rosenberg |
| Name: Ellen S. Rosenberg | ||
| Title: Chief Legal Officer and Corporate Secretary | ||
Exhibit 99.1
Amicus Announces Additional Positive Interim Clinical Data for CLN6 Batten Disease Gene Therapy at 48th Annual Meeting of the Child Neurology Society
New Data Support Meaningful Impact on Motor and Language Function in Children with Fatal Neurologic Disease
Evidence of Disease Stabilization in 7 of 8 Children with Data for up to 2 Years Post-Treatment
CRANBURY, NJ, and CHARLOTTE, NC, October 24, 2019 – Amicus Therapeutics (Nasdaq: FOLD) today announced additional positive interim results from its CLN6 Batten disease gene therapy program licensed from the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital. The results are featured in a poster presentation at the 48th Annual Meeting of the Child Neurology Society to be held October 23-26, 2019 in Charlotte, NC. The poster is available in the Events and Presentations section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/events-and-presentations.
AWRI is conducting the ongoing Phase 1/2 clinical study of a single one-time intrathecal administration of AAV-CLN6 gene therapy for CLN6 Batten disease. With no approved treatments, CLN6 Batten disease is a fatal neurologic disease that rapidly robs children of their ability to walk, speak, think, and see.
Clinical Data Highlights:
Interim safety data are available for 12 patients. Interim efficacy data are available for the first eight children with CLN6 Batten disease for up to 24 months post-administration of the AAV-CLN6 gene therapy.
| · | Safety (n=12): as previously announced, treatment with AAV-CLN6 gene therapy was generally well tolerated. The majority of adverse events (AEs) were mild and unrelated to treatment. Additional details are provided in the poster. |
| · | Hamburg Motor & Language Aggregate Score (n=8): as previously announced, as of the interim analysis from this ongoing study, the Hamburg Motor & Language Score, an assessment of ambulation and speech, shows a positive impact on motor and language function for 7 of 8 patients treated (16-25 months post-administration of the gene therapy as of this data cutoff). Additional details are provided in the poster. |
| · | Hamburg Motor, Language, Seizure and Vision Subscores (n=8): new data on motor, language, seizure and vision subscores suggest stabilization of these individual components in most patients from baseline to months 12 and 24, in particular those patients treated at a younger age, further supporting the positive impact of one-time intrathecal AAV gene therapy in children with CLN6 Batten disease. Additional details are provided in the poster. |
| · | Natural History and Sibling Pair Comparisons: as previously announced, the AAV-CLN6 gene therapy demonstrated a positive impact on motor and language function compared to a natural history data set, including matched comparisons, as well as in comparisons within sibling pairs. Additional details are provided in the poster. |
Jay Barth, Chief Medical Officer of Amicus Therapeutics, stated, “We are pleased to share these positive interim clinical data for our intrathecal AAV gene therapy with the CLN6 physician community for the first time at Child Neurology Society. The aggregate and individual Hamburg components, in addition to the natural history data and sibling comparisons, continue to suggest that our gene therapy in CLN6 Batten disease has the potential to halt the progression of this devastating disease that causes loss of brain function and is fatal in childhood.”
Emily de los Reyes, MD, PhD, Principal Investigator of the CLN6 clinical trial at AWRI at Nationwide Children’s and Professor of Clinical Pediatrics and Neurology at The Ohio State University College of Medicine stated, “I continue to be pleased with the progress of this trial as well as our collection of matched natural history data to further inform the results for the AAV-CLN6 gene therapy. I look forward to following these initial study participants as well as collecting new data for additional participants as we continue to advance the program in CLN6 and move additional Batten disease programs through the clinic in collaboration with Amicus.”
Upcoming Amicus Milestones in Next 12 Months:
| · | Collection and presentation of additional natural history data in CLN6 Batten disease |
| · | Dosing of additional patients |
| · | Advance regulatory discussions |
| · | Manufacturing of additional AAV-CLN6 gene therapy underway at Thermo Fisher (Brammer Bio) |
| · | Continued advancement of AAV gene therapy programs in CLN3, CLN8 and CLN1 Batten disease. |
About Batten Disease
Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. In these diseases, a defect in a specific gene triggers a cascade of problems that interferes with a cell’s ability to recycle certain molecules. Each gene is called CLN (ceroid lipofuscinosis, neuronal) and given a different number designation as its subtype. There are 13 known forms of Batten disease often referred to as CLN1-8; 10-14. The various types of Batten disease have similar features and symptoms but vary in severity and age of onset.
Most forms of Batten disease/NCLs usually begin during childhood. The clinical course often involves progressive loss of independent adaptive skills such as mobility, feeding, and communication. Patients may also experience vision loss, personality changes, behavioral problems, learning impairment, and seizures. Patients typically experience progressive loss of motor function and eventually become wheelchair-bound, are then bedridden, and die prematurely.
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 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 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 preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials and the prospects and timing of the potential regulatory approval of our product candidates. In particular, this press release relates to interim data from an ongoing Phase 1/2 study to investigate intrathecal administration of AAV-CLN6 gene therapy. The inclusion of forward-looking statements arising from this interim data, ongoing study and natural history preliminary data 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, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, 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 results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. The interim data and Phase 1/2 study discussed herein is inherently preliminary and early in the study, derived from a limited patient set, and later trial results with this patient set or others may not be consistent with these preliminary results. 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, 2018 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2019. 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:
Sara Pellegrino, IRC
Vice President, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
Media:
Christopher Byrne
Executive Director, Corporate Communications
cbyrne@amicusrx.com
609-662-2798
FOLD–G
Exhibit 99.2
Interim Results From the First Clinical Gene Therapy Trial for CLN6 Batten Disease de los Reyes E1, Meyer K1,2, Lehwald L1, Albright C1, Castelli J3, Jiang H3, Reha A3, Barth JA3 1Nationwide Children’s Hospital, Columbus, OH, USA; 2Department of Pediatrics, Ohio State University, Columbus, OH, USA; 3Amicus Therapeutics, Inc., Cranbury, NJ, USA BACKGROUND • Mutations in the CLN6 gene cause lysosomal dysfunction that leads to a variant late-infantile form of neuronal ceroid lipofuscinosis, or Batten disease, a rare and fatal neurodegenerative disorder1,2 • The onset of CLN6-type Batten disease is typically between the age of 2-5 years, and affected children experience language delay, motor regression, epilepsy, decline of vision, and premature death, often occurring in late childhood1,2 • There is currently no treatment for this rapidly progressive disease • Current management strategies focus on symptomatic treatment and supportive care1 OBJECTIVES • To evaluate the safety and effectiveness of CLN6 gene transfer using an adeno-associated virus serotype 9 (AAV9) vector in the first intrathecal gene therapy clinical trial for children with CLN6-type Batten disease METHODS Study Design • This is an open-label, single-site phase 1/2 trial of single-dose CLN6 gene transfer via intrathecal injection into the lumbar spinal cord region in children with CLN6-type Batten disease (for study design see Figure 1) • CLN6 gene was delivered using a self-complementary AAV serotype 9 (scAAV9) under the control of a chicken-β-actin promoter • Efficacy assessments include the Hamburg Scale, which rates motor function and language (HM+L)3 (Figure 2), as well as vision and seizures4 Figure 1. Study Design Key Eligibility Criteria • Diagnosis of CLN6 determined by genotyping • Hamburg motor and language score ≥3 • Age ≥1 year Efficacy Evaluations • Hamburg Scale • Additional measures include: UBDRS, Cognitive and Language Ability, Vision, QOL, Ophthalmologic Assessments, Brain MRI Day 1-3 Screening/Baseline Assessments Day 7 Day 14 Day 21 Day 30 Q3 month visits thereafter Safety Evaluations • AEs • Vital signs • Physical/neurological exam • Blood and urine laboratory parameters • ECG • Laboratory parameters of immune response evaluation Month 24: completion of evaluation period Intrathecal Administration (dose: 1.5 x 1013 vg) at Day 1 AEs=adverse events; ECG=electrocardiogram; MRI=magnetic resonance imaging; Q3=every 3; QOL=quality of life; UBDRS=Unified Batten Disease Rating Scale; vg=viral genomes. Figure 2. Hamburg Motor and Language Scale3 Motor Function Language Function Normal Normal Clumsy, falls Abnormal Non-walking Minimal Immobile Unintelligible or no vocalization 3 3 3 = normal condition 2 = slight or just noticeable abnormality 1 = severe abnormality 0 = complete loss of function In each domain, the rating is structured so that a score of: 2 2 1 1 0 0 Statistical Analyses • Safety data were analyzed for all 12 treated patients; efficacy results were presented for 8 patients, including all sibling pairs with ≥1 year of data and nonsibling patients with ≥2 years of data • HM+L scores of treated patients were compared with their untreated siblings, as well as natural history data derived from an ongoing, retrospective chart review study in CLN6 Batten disease (clinicaltrials.gov: NCT03285425) • Interim data cutoff was July 31, 2019 RESULTS Baseline Characteristics • The study population (n=12) included both male and female patients ranging from 19 to 79 months in age • Baseline characteristics for the 8 patients evaluated for efficacy are shown in Table 1 Table 1. Patient Characteristics Patient Sex Age at Gene Transfer (months) Exposure Duration (months)* Hamburg Motor + Language at Baseline Time Between Baseline and Last Measure (Months) 1 F 63 41 3 25 2 F 30 39 6 23 3 M 36 38 5 24 4 M 67 30 4 24 5 F 79 29 3 24 6 M 56 28 5 24 7 M 19 22 5 22 8 M 61 18 4 16 *Calculated to July 31, 2019. Patients 1-8 were included in the efficacy results. Safety • At interim data cutoff, duration since gene transfer ranged from 8 to 41 months • 137 adverse events (AEs) were reported among the 12 patients, and most AEs were mild and unrelated to treatment. Table 2 reports all treatmentemergent AEs [TEAEs] that occurred in >1 patient • Nine Grade 3 (severe) AEs (SAEs) were reported in 4 patients; 4 of the SAEs were considered possibly related to treatment (2 events of vomiting, 1 event of epigastric pain, and 1 event of fever; all 4 recovered) • No Grade 4 (life-threatening) AEs or deaths were reported • No pattern of AEs related to AAV9 or CLN6 transgene immunogenicity was observed Table 2. Treatment-Emergent Adverse Events That Occurred in >1 Patient Adverse event N=12 n (%) Upper respiratory tract infection 7 (58.3) Viral infection 6 (50) Vomiting 5 (41.7) Hematuria 4 (33.3) Back pain 3 (25) Constipation 3 (25) Diarrhea 3 (25) Seizure 3 (25) Viral gastroenteritis 3 (25) Abnormal behavior 2 (16.7) Insomnia 2 (16.7) Myoclonus 2 (16.7) Otitis media 2 (16.7) Procedural pain 2 (16.7) Pyrexia 2 (16.7) Urinary tract infection 2 (16.7) Hamburg Motor and Language Score Figure 3. Mean Hamburg Motor and Language Aggregate Score, Motor Subscore, and Language Subscore Over Time in CLN6 Natural History Patients Age (months) Mean Hamburg Motor + Language Score 0 0 12 24 36 48 60 72 84 96 108 120 132 1 2 3 4 5 6 n=11 pts with late-infantile CLN6 Motor + Language Motor Language CLN6 natural history shows a progressive decline of approximately one point per year in the Hamburg Motor and Language score from age two onwards with similar decline in motor and language. Figure 4. Individual Hamburg Motor and Language Aggregate Score, Motor Subscore, and Language Subscore of AAV9-CLN6 Gene Transfer Patients 0 1 2 3 4 5 6 0 3 6 9 12 15 18 21 24 Patient 7 Treated at 19 Months HM+L MOTOR LANGUAGE HM+L MOTOR LANGUAGE Score 0 1 2 3 4 5 6 0 3 6 9 12 15 18 21 24 Patient 2 Treated at 30 Months 0 1 2 3 4 5 6 0 3 6 9 12 15 18 21 24 Patient 3 Treated at 36 Months 0 1 2 3 4 5 6 0 3 6 9 12 15 18 21 24 Patient 6 Treated at 56 Months Months on Treatment Months on Treatment 0 1 2 3 4 5 6 0 3 6 9 12 15 18 21 24 Patient 8 Treated at 61 Months Score 0 1 2 3 4 5 6 0 3 6 9 12 15 18 21 24 Patient 1 Treated at 63 Months 0 1 2 3 4 5 6 0 3 6 9 12 15 18 21 24 Patient 4 Treated at 66 Months 0 1 2 3 4 5 6 0 3 6 9 12 15 18 21 24 Patient 5 Treated at 79 Months Months on Treatment Months on Treatment Months on Treatment Months on Treatment Months on Treatment Months on Treatment HM+L MOTOR LANGUAGE HM+L MOTOR LANGUAGE HM+L MOTOR LANGUAGE HM+L MOTOR LANGUAGE HM+L MOTOR LANGUAGE HM+L MOTOR LANGUAGE HM+L=Hamburg Motor and Language aggregate score. Seven of 8 patients with ≥12 months efficacy data maintained the combined Hamburg score or had initial change (+1 to -1 points) followed by stabilization. Figure 5. Comparison of AAV9-CLN6 Gene Transfer Patients and Untreated Sibling(s) in (A) Hamburg Motor and Language Aggregate Score, (B) Motor Subscore, and (C) Language Subscore 0 1 2 3 4 5 6 36 48 60 72 84 96 Treated Patient 6 vs. Untreated Sibling Treated Patient 6 Untreated Sibling Age (Months) Age (Months) Age (Months) 0 1 2 3 4 5 6 36 48 60 72 84 Treated Patient 3 vs. Untreated Sibling Treated Patient 3 Untreated Sibling 0 1 2 3 4 5 6 36 48 60 72 84 Treated Patient 8 vs. Untreated Siblings Treated Patient 8 Untreated Sibling 1 Untreated Sibling 2 A Hamburg Motor and Language Aggregate Score Score B 0 1 2 3 36 48 60 72 84 96 Treated Patient 6 vs. Untreated Sibling Treated Patient 6 Untreated Sibling Age (Months) Age (Months) Age (Months) 0 1 2 3 36 48 60 72 84 Treated Patient 3 vs. Untreated Sibling Treated Patient 3 Untreated Sibling Hamburg Motor Score 0 1 2 3 36 48 60 72 84 Treated Patient 8 vs. Untreated Siblings Treated Patient 8 Untreated Sibling 1 Untreated Sibling 2 Score C 0 1 2 3 36 48 60 72 84 96 Treated Patient 6 vs. Untreated Sibling Treated Patient 6 Untreated Sibling Age (Months) Age (Months) Age (Months) 0 1 2 3 36 48 60 72 84 Treated Patient 3 vs. Untreated Sibling Treated Patient 3 Untreated Sibling Hamburg Language Score 0 1 2 3 36 48 60 72 84 Treated Patient 8 vs. Untreated Siblings Treated Patient 8 Untreated Sibling 1 Untreated Sibling 2 Score Treated patients demonstrated no change or initial change followed by stabilization whereas untreated sibling(s) experienced substantial declines. Figure 6. Comparison of AAV9-CLN6 Gene Transfer Patients and Natural History Patients Matched Based on Age And Baseline Hamburg Motor and Language Aggregate Scores Natural History Patients (mean) AAV9-CLN6-Treated Patient Age (months) Hamburg M+L Score Pt 7 (Month 19 to 41)a 6 4 2 0 20 30 40 80 N= 9 NH pts Age (months) Hamburg M+L Score Pt 2 (Month 30 to 53) 6 4 2 0 30 40 50 +1.7 N= 4 NH pts Age (months) Hamburg M+L Score Pt 3 (Month 36 to 61) 6 4 2 0 30 40 50 60 +2.3 N= 3 NH pts Age (months) Hamburg M+L Score Pt 6 (Month 56 to 80) 6 4 2 0 50 70 90 +0.7 N= 1 NH pt Age (months) Hamburg M+L Score Pt 8 (Month 61 to 77) 6 4 2 0 60 70 +1.0 N= 4 NH pts Age (months) Hamburg M+L Score Pt 1 (Month 63 to 88) 6 4 2 0 60 70 80 90 +1.8 N= 1 NH pt Age (months) Hamburg M+L Score Pt 4 (Month 66 to 90) 6 4 2 0 60 70 80 90 +2.0 N= 1 NH pt Age (months) Hamburg M+L Score Pt 5 (Month 79 to 104) 6 4 2 0 70 90 110 +1.0 HM+L=Hamburg Motor and Language Aggregate Score; NH=natural history; pt=patient. aThis patient currently has no age- and baseline HM+L score-matched patients in the natural history cohort. To facilitate comparison of AAV9-CLN6-treated patients to natural history patients with respect to clinical course over time, HM+L results from the first 8 treated patients in this study were matched with HM+L results from 11 patients in a retrospective CLN6 natural history study (ClinicalTrials.gov Identifier: NCT03285425; PI: Emily de los Reyes, MD). For each treated patient, all natural history patients who had the same baseline Hamburg M+L score at the same age at which treatment started were “matched.” The average Hamburg M+L score for these matched patients was then calculated at the ages corresponding to last observation for each treated patient. Overall, these data indicate that the gene-therapy–treated patients demonstrate stabilization in Hamburg M+L score compared to matched natural history patients. 204 This study was supported by Amicus Therapeutics, Inc. Presented at the 48th Annual CNS Meeting, October 23-26, 2019; Charlotte, NC. Poster number 204. Hamburg Vision and Seizure SubScores Table 2. Individual Hamburg Vision and Seizure Subscores of AAV9-CLN6 Gene Transfer Patients Patient Age at Gene Transfer (months) Hamburg Vison Score Hamburg Seizure Score Baseline Month 12 Month 24 Baseline Month 12 Month 24 1 63 3 2 1 3 3 3 2 30 3 3 3 3 3 3 3 36 3 3 3 3 3 2 4 67 2 1 1 2 3 3 5 79 2 0 0 3 2 0 6 56 3 3 3 3 3 3 7 19 3 3 _ 3 3 _ 8 61 3 3 _ 3 3 _ Hamburg Vision Score is decoded as: 0: no reaction to visual stimuli; 1: reacts to light; 2: grabbing for objects uncoordinated; 3: recognizes desirable object, grabs at it. Hamburg Seizure Score is decoded as: 0: > 1 seizure per month; 1: 1 seizure per month; 2: 1 - 2 seizures in 3 months; 3: no seizure in 3 months. CONCLUSIONS • These interim safety and efficacy data suggest that AAV9-CLN6 gene therapy has the potential to stabilize disease progression of the variant late-infantile onset CLN6 Batten disease • Most adverse events were mild and unrelated to treatment; the most common treatment-related adverse events were vomiting and back pain, which were all transient. Therefore, intrathecal administration of AAV9-CLN6 is generally well tolerated • Efficacy results demonstrated a meaningful treatment effect in motor and language function ◦◦ AAV9-CLN6-treated patients demonstrated stabilization in HM+L score compared with untreated siblings and natural history patients matched for age and HM+L baseline score ◦◦ Comparison of treated younger and older patients further supports the potential benefit of early intervention of gene therapy with AAV9-CLN6 REFERENCES 1. Schultz A et al. Biochem Biophys Acta. 2013;1832(11):1801-1806. 2. Mink J et al. J Child Neurol. 2013;28(9):1101-1105. 3. Wyrwich KW et al. J Inborn Errors Metab Screen. 2018;6:1-7. 4. Steinfeld R et al. Am J Med Genet. 2002;112(4):347-354. ACKNOWLEDGMENTS The authors thank the patients and their families. Third-party medical writing assistance was provided by ApotheCom (Yardley, PA) and was supported by Amicus Therapeutics, Inc. DISCLOSURE Conflicts of Interest ER received research grants from Amicus and Biomarin and has been a consultant for Biomarin. KM received a research grant from Audentes and royalties from Amicus. LL attended advisory boards and received consultant fees from Amicus and Biomarin. CA received a research grant from Amicus. JC, HJ, AR and JB are employees of and hold stock in Amicus.