Amicus Therapeutics published positive outcomes for cipaglucosidase alfa-atga + miglustat in adults with late-onset Pompe disease.
Quiver AI Summary
Amicus Therapeutics has released a post-hoc analysis from the PROPEL study, published in Muscle and Nerve, which evaluated the efficacy of cipaglucosidase alfa-atga combined with miglustat (cipa+mig) in adults with late-onset Pompe disease (LOPD). The analysis showed that patients transitioning from alglucosidase alfa to cipa+mig experienced significant improvements or stability in various outcome measures, including motor function and fatigue, while those remaining on alglucosidase alfa generally showed worsening symptoms. Specifically, patients who switched did not experience significant declines in health and demonstrated improvements in walking distance and muscle testing. The findings suggest the potential benefits of switching treatments, especially as LOPD is a progressive disorder. Amicus remains committed to providing innovative therapies for patients with rare diseases.
Potential Positives
- Publication of a post-hoc analysis in a reputable journal highlights positive outcomes for patients switching to cipaglucosidase alfa-atga + miglustat, reinforcing the therapy's efficacy.
- Significant improvements were observed in patients' walking distance, fatigue levels, and overall quality of life metrics, indicating tangible benefits of the new treatment.
- Results provide further validation of the differentiated mechanism and clinical profile of the combination therapy, strengthening the company's position in the rare disease market.
- High completion rate (over 95%) of the PROPEL study suggests strong patient engagement and reliability of data collected.
Potential Negatives
- PROPEL study results did not achieve statistical significance for the primary endpoint of superiority in change from baseline to week 52 in 6-minute walk distance in the overall population.
- Despite some positive findings in the post-hoc analysis, the reliance on a post-hoc analysis may raise concerns about the robustness and reliability of the results.
- The press release highlights a significant distinction in outcomes between patients switched to Pombiliti + Opfolda and those remaining on alglucosidase alfa, potentially highlighting deficiencies in the latter therapy's effectiveness.
FAQ
What is the main finding of the PROPEL study's post-hoc analysis?
The analysis showed improvement or stability in outcomes for patients switching to cipaglucosidase alfa-atga + miglustat compared to those remaining on alglucosidase alfa.
How many patients were involved in the PROPEL study?
The PROPEL study enrolled 123 adult patients with late-onset Pompe disease.
What is the significance of Pombiliti + Opfolda for patients?
Pombiliti + Opfolda is indicated for adults with late-onset Pompe disease not improving on their current enzyme replacement therapy.
What outcomes showed significant improvement after switching therapies?
Significant improvements were seen in 6-minute walk distance, muscle strength, and patient-reported fatigue among those switching to cipaglucosidase alfa-atga + miglustat.
What is late-onset Pompe disease?
Late-onset Pompe disease is an inherited disorder caused by deficiency in the enzyme acid alpha-glucosidase, leading to muscle weakness and other complications.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
$FOLD Insider Trading Activity
$FOLD insiders have traded $FOLD stock on the open market 1 times in the past 6 months. Of those trades, 0 have been purchases and 1 have been sales.
Here’s a breakdown of recent trading of $FOLD stock by insiders over the last 6 months:
- BRADLEY L CAMPBELL (President and CEO) sold 400 shares for an estimated $4,000
To track insider transactions, check out Quiver Quantitative's insider trading dashboard.
$FOLD Hedge Fund Activity
We have seen 154 institutional investors add shares of $FOLD stock to their portfolio, and 152 decrease their positions in their most recent quarter.
Here are some of the largest recent moves:
- FIERA CAPITAL CORP removed 3,037,248 shares (-100.0%) from their portfolio in Q1 2025, for an estimated $24,783,943
- POINT72 ASSET MANAGEMENT, L.P. added 2,985,108 shares (+inf%) to their portfolio in Q1 2025, for an estimated $24,358,481
- MILLENNIUM MANAGEMENT LLC added 2,902,375 shares (+635.1%) to their portfolio in Q1 2025, for an estimated $23,683,380
- PERCEPTIVE ADVISORS LLC removed 2,781,425 shares (-11.5%) from their portfolio in Q1 2025, for an estimated $22,696,428
- AVORO CAPITAL ADVISORS LLC removed 2,675,000 shares (-11.5%) from their portfolio in Q1 2025, for an estimated $21,828,000
- REDMILE GROUP, LLC added 2,478,779 shares (+35.1%) to their portfolio in Q1 2025, for an estimated $20,226,836
- JPMORGAN CHASE & CO removed 2,319,966 shares (-32.2%) from their portfolio in Q1 2025, for an estimated $18,930,922
To track hedge funds' stock portfolios, check out Quiver Quantitative's institutional holdings dashboard.
$FOLD Analyst Ratings
Wall Street analysts have issued reports on $FOLD in the last several months. We have seen 1 firms issue buy ratings on the stock, and 0 firms issue sell ratings.
Here are some recent analyst ratings:
- UBS issued a "Buy" rating on 05/02/2025
To track analyst ratings and price targets for $FOLD, check out Quiver Quantitative's $FOLD forecast page.
Full Release
PRINCETON, N.J., June 03, 2025 (GLOBE NEWSWIRE) --
Amicus Therapeutics
(Nasdaq: FOLD), today announced the publication of a
post-hoc
analysis of data from the ERT-experienced cohort of the PROPEL study of cipaglucosidase alfa-atga + miglustat (cipa+mig) in adults with late-onset Pompe disease (LOPD) in
Muscle and Nerve
.
In this new publication, based on a within group effect-size analysis, subjects who switched from alglucosidase alfa to cipa+mig achieved improvements or stability in most of the outcomes measured.
In PROPEL, 77% of patients (n=95) received enzyme replacement therapy (ERT) with alglucosidase alfa before study entry, with a median ERT duration of 7.4 years. In this new publication of post-hoc analysis from PROPEL, ERT-experienced patients switched to cipa+mig (n=65), showed improvement (d≥0.2) or stability (−0.2
“Across the different domains in this analysis, patients who switched to Pombiliti + Opfolda experienced stability or improvement on many measures, including 6-minute walking distance and PROMIS-Fatigue, compared to those who remained on alglucosidase alfa who primarily stayed stable or experienced worsening,” said Hani A. Kushlaf, MD, Professor of Neurology and Pathology, University of Cincinnati. “Given that LOPD is a progressive condition, it’s imperative that physicians assess when patients should consider switching to Pombiliti + Opfolda.”
“The consistency of stability or improvement across multiple domains in the cohort of ERT-experienced patients from PROPEL – including measures of biomarkers, muscle strength, motor function, pulmonary function, and quality of life – is highly encouraging in a disease known for its relentless progression,” said Jeff Castelli, PhD, Chief Development Officer, Amicus Therapeutics, Inc. “We view these results as further validation of the differentiated mechanism and clinical profile of Pombiliti + Opfolda and remain committed to advancing therapies that make a tangible difference in the lives of people living with late-onset Pompe disease.”
About the PROPEL Study
PROPEL was a 52-week, double-blind randomized global study designed to assess the efficacy, safety, and tolerability of cipaglucosidase alfa-atga + miglustat compared to non-U.S. approved alglucosidase alfa + placebo (the comparator). The study enrolled 123 adult LOPD patients who still had the ability to walk and to breathe without mechanical ventilation.
Patients enrolled in PROPEL were randomized 2:1 so that for every two patients randomized to be treated with cipaglucosidase alfa-atga + miglustat, one was randomized to be treated with the comparator. Of the patients enrolled in PROPEL, 77% were being treated with alglucosidase alfa (n=95) for at least 2 years at study entry and 23% had never been treated with any ERT (n=28). 117 of the 123 patients (>95%) completed the PROPEL study.
Efficacy endpoints of the study included primary endpoint of change from baseline to week 52 in 6-minute walk distance (6MWD) for comparison of superiority and key secondary endpoint of change from baseline to week 52 in forced vital capacity (FVC). PROPEL did not achieve statistical significance for the primary endpoint of superiority in change from baseline to week 52 in 6MWD in the overall population. After 52 weeks, ERT-experienced patients treated with cipaglucosidase alfa-atga + miglustat (n=61) walked an estimated 17 meters (95% CI, 0.2, 33) farther than the comparator group (n=29). After 52 weeks, ERT-experienced patients treated with cipaglucosidase alfa-atga + miglustat (n=55) showed an estimated treatment difference of 3.5% (95% CI, 1.0, 6.0) in FVC compared with the comparator group (n=29).
More details about this
post-hoc
analysis
This post-hoc analysis assessed the magnitude of within-group treatment effects across measures of motor function, muscle strength, pulmonary function, patient-reported outcomes/QoL measures, and biomarkers. Standardized effect size analysis describes the absolute effect size relative to the variability of the data. It transforms the effect size into a scale with no units of measurement (Cohen’s d). The authors calculated standardized within-group effect sizes (Cohen’s d for within-group comparisons) and corresponding 95% CIs for the change from baseline to week 52 for the primary, secondary, and pharmacodynamic endpoints of the PROPEL study. Standardized within-group effect sizes and corresponding 95% CIs were calculated by dividing the mean change from baseline values and CIs at week 52 by the standard deviation (SD) of the difference scores. Consistent with medical literature, effect sizes were defined as stable (−0.2 < d < 0.2), small (0.2 ≤ d < 0.5), medium (0.5 ≤ d < 0.8), or large (d ≥ 0.8) improvement, or as small (−0.5 < d ≤ −0.2), medium (−0.8 < d ≤ −0.5), or large (d ≤ −0.8) worsening, and were considered statistically significant if the standardized 95% CIs did not cross zero.
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.
U.S. 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).
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. CONTRAINDICATION:
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
.
About Late-Onset Pompe Disease
Late-onset
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. 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.
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 diseases. For more information please visit the company’s website at
www.amicusrx.com
, and follow on
X
and
LinkedIn
.
PP-AT-ALL-0001-0525
CONTACTS:
Investors:
Amicus Therapeutics
Andrew Faughnan
Vice President, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Amicus Therapeutics
Diana Moore
Vice President, Corporate Communications
dmoore@amicusrx.com
(609) 662-5079
FOLD–G
This article was originally published on Quiver News, read the full story.
The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.
