http://www.raredr.com/conferences/world2018/val1221-in-pompe
Valerion Presents Data from Study of VAL-1221 in Pompe Disease

Mathew Shanley

Today at the 14th Annual WORLDSymposium in San Diego, California, Valerion Therapeutics presented initial results from the first cohort of its ongoing Phase 1/2 clinical study of VAL-1221 in patients with late-onset Pompe disease.

VAL-1221 is currently in clinical development for the treatment of patients with the late-onset variation of the disease, and the investigational fusion protein combines antibody-mediated delivery technology with recombinant human acid alpha-glucosidase (rhGAA). It is being designed to improve the delivery of rhGAA to muscle.

Pompe disease is characterized primarily by skeletal muscle weakness causing problems with ambulation and respiratory function. It is caused by a deficiency in acid alpha-glucosidase (GAA), and symptom severity varies from patient-to-patient. Most who suffer from the condition experience neuromuscular, respiratory, cardiovascular, and gastrointestinal deficiencies, however, those with the late-onset variation of the disease may not show symptoms until their third or fourth decade of life.

"We are delighted to present these initial clinical findings from the first dosing cohort of our ongoing clinical study with VAL-1221 and look forward to providing further updates as the trial progresses into higher dosing cohorts," said Deborah Ramsdell, CEO of Valerion. "By targeting and clearing both lysosomal and extra-lysosomal glycogen in the cytoplasm, VAL-1221 has the potential to provide new therapeutic options to patients with late-onset Pompe disease."

In mouse models of the disease, VAL-1221 exhibited the ability to reduce an accumulation of lysosomal glycogen as effectively as the current standard-of-care enzyme replacement therapies.

In the study, 2 cohorts of 4 patients each were randomized to either treatment with VAL-1221 or positive control (rhGAA). Each patient randomized to VAL-1221 received 7 infusions, 1 every other week, over 12 weeks. Primary endpoints included: incidences of treatment-emergent adverse events (AEs); immunogenicity outcome measures - incidences of anti-VAL-1221 antibodies; and immunogenicity outcome measure – incidences of GAA antibodies.

“We had a lot of difficulty with enrollment,” said Ramsdell in an interview with Rare Disease Report on Wednesday. “We opened our IND early last year, and we’ve had a very difficult time enrolling patients. There’s a lot of competition in the space, and there are a lot of studies that are in later stages than ours are. Once we had data that showed we had activity in our patients, it was much, much easier to go after more patients and talk to clinicians.”

“We’re trying to do more of that [at WORLDSymposium],” Ramsdell added. “It’s like ‘Look. We’ve enrolled our first cohort, we’re enrolling our second cohort, it’s safe, and we’ve seen signals. Once you do that, trials like ours become self-fulfilling prophecies.”

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