Trial results

ABX1100, an experimental substrate reduction therapy for late-onset Pompe disease (LOPD), was well tolerated and seemed to work as designed in an early clinical trial involving nine adults with LOPD, new data show. Developer Aro Biotherapeutics noted that, in addition to meeting the study’s primary endpoints, or goals,…

ABX1100, an experimental substrate reduction therapy being developed by Aro Biotherapeutics, showed strong and sustained biological activity and early signs of clinical benefit in people with late-onset Pompe disease (LOPD), according to preliminary trial results. The therapy was also well tolerated. The preliminary results are based on the…

Using Nexviazyme (avalglucosidase alfa) led to sustained improvements in lung function, walking ability, and other motor skills over nearly three years of treatment in people with late-onset Pompe disease (LOPD), according to newly shared results from a global clinical trial. LOPD patients who switched to Nexviazyme after one…

For adults with late-onset Pompe disease (LOPD), Pombiliti + Opfolda (cipaglucosidase alfa/miglustat) appears to offer lasting benefits. New four-year data from a Phase 3 trial show the combination treatment helped sustain improvements in motor function and disease biomarkers in patients who had previously been treated with standard…

Switching from Lumizyme (alglucosidase alfa) to Pombiliti + Opfolda (cipaglucosidase alfa and miglustat) improved or stabilized motor function, muscle strength, and lung function in adults with late-onset Pompe disease (LOPD). That’s according to a new analysis from the Phase 3 PROPEL trial (NCT03729362) that also showed…

More late-onset Pompe disease (LOPD) patients treated with Nexviazyme (avalglucosidase alfa) achieved clinically meaningful improvements in patient-reported outcome (PRO) measures related to symptoms, daily life impacts, and life quality than those treated with Lumizyme (alglucosidase alfa). That is according to new analyses from the COMET Phase 3 clinical…

Adding the enzyme stabilizer miglustat did not improve the effectiveness of Lumizyme (alglucosidase alfa) or Nexviazyme (avalglucosidase alfa) in a mouse model of Pompe disease, a new study reports. “These data demonstrate that co-administration of miglustat with either [Lumizyme or Nexviazyme] to Pompe mice provides no additional…

Treatment with AT-GAA, Amicus Therapeutics’ experimental two-part therapy for Pompe disease, has now been shown to improve motor function, and to stabilize or improve lung function, for up to four years in adults. That’s according to new analyses from the ongoing Amicus-sponsored Phase 1/2 ATB200-02 trial…

After two years being treated with AT-GAA (cipaglucosidase alfa/miglustat), adults with late-onset Pompe disease (LOPD) maintained their improvements in walking ability and had stabilized breathing function, as well as a reduction in the levels of disease biomarkers, according to…

Nearly three years of treatment with Nexviazyme (avalglucosidase alfa) led to long-term improvements in walking ability and lung function for people with late-onset Pompe disease (LOPD), according to new data from the Phase 3 clinical trial COMET. …