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BridgeBio Pharma and its subsidiary ML Bio Solutions today announced the latest results
of the Phase 2 clinical trial of their oral therapy BBP-418 (ribitol).
The data showed that at 12 months of trial, BBP-418 was effective in reducing muscle damage in patients with limb girdle muscular dystrophy type 2i (LGMD2i) and maintaining motor function
.
The press release notes that this may be the first therapy proven to increase glycosylation of αDG protein in LGMD2i patients to reduce muscle wasting and improve disease progression
.
The companies expect to begin a Phase 3 clinical trial
of BBP-418 in 2023.
.
LGMD2i is a single-gene autosomal recessive disorder caused by a loss-of-function mutation in the FKRP gene, resulting in hypoglycosylation of the αDG protein associated with stabilizing muscle cells, affecting approximately 7,000 patients
in the United States and the European Union.
Most LGMD2i patients develop symptoms
between the ages of 5-18.
Clinically typical is skeletal myopathy affecting the lower extremities and thus the upper extremities, often later with respiratory and myocardial involvement
.
Patients with homozygous genotypes usually present in late childhood and progress to loss of independent walking (25%), assisted ventilation (5%), and cardiomyopathy (10%)
.
Currently, LGMD2i patients can only receive symptom improvement or palliative care, and no approved therapies can improve disease progression
.
BBP-418 is designed to restore normal function to alphaDG by properly glycosylating muscle cells and potentially improving muscle strength and function
in patients.
The therapy is fast-track and orphan drug designated by the U.
S.
FDA for LGMD2i
.
The press release states that if the development project is successful, the company believes that BBP-418 is expected to become the first therapy
approved for the treatment of LGMD2i patients.
approved for the treatment of LGMD2i patients.
▲Pharmacological mechanism of BBP-418 (Image source: Reference [3])
▲Pharmacological mechanism of BBP-418 (Image source: Reference [3]) The Phase 2 trial enrolled 14 participants, including ambulatory and ambulatory LGMD2i patients
.
This open-label study explored the safety and tolerability of treatment with escalating doses of BBP-418, as well as the feasibility and usefulness of selected clinical efficacy and pharmacodynamic evaluation tests
in 3 cohorts of LGMD2i patients.
Data observed after 12 months of treatment show that:
In all cohorts, participants increased
αDG glycosylation.
The glycosylation ratio of αDG averaged +0.
21
at day 90.
Since αDG glycosylation deficiency is a direct cause of muscle wasting, this data shows that BBP-418 has the potential to address the underlying cause of LGMD2i disease and promote improved
muscle function in patients.
αDG glycosylation.
The glycosylation ratio of αDG averaged +0.
21
at day 90.
This data shows that BBP-418 has the potential to address the underlying cause of LGMD2i disease and promote improved
muscle function in patients.
For more than 12 months, participants experienced a greater than 75% decrease
in creatine kinase (CK) levels compared to baseline.
Creatine kinase is a biomarker
of muscle damage.
in creatine kinase (CK) levels compared to baseline.
At 12 months, the NSAD score and 10MWT walk test used to check mobility in patients with limb girdle muscular dystrophy also improved
compared to baseline.
their NSAD scores and 10MWT walk tests, which were used to check mobility in patients with limb girdle dystrophy.
BBP-418 was well tolerated and no treatment-related serious adverse events and dose-limiting toxicities
were observed during 12 months of treatment.
"LGMD2i seriously affects patients' independent living
.
As the disease progresses, LGMD2i patients gradually lose the ability to live independently, and depending on the severity of the disease, they may eventually need to live
in a wheelchair or even on a ventilator.
Data from this Phase 2 show that our investigational therapies are well tolerated and have the potential to improve or slow the clinical deterioration of
the disease.
Dr.
Douglas Sproule, chief medical officer of ML Bio Solutions, said
.
