–First-in-Human Study to Evaluate the Safety, Tolerability and
Pharmacokinetics of GFB-887, a Targeted Podocyte-protective Therapeutic
for the treatment of Focal Segmental Glomerulosclerosis,
Treatment-resistant Minimal Change Disease and Diabetic Nephropathy–
CAMBRIDGE, Mass.–(BUSINESS WIRE)–#conference–Goldfinch Bio, Inc., a biotechnology company focused on developing
precision therapies for patients with kidney diseases, today announced
it has initiated dosing in a Phase 1 clinical trial evaluating GFB-887,
its subtype-selective, small molecule TRPC5 inhibitor, as a potential
treatment for kidney diseases associated with proteinuria and
progressive renal dysfunction including focal segmental
glomerulosclerosis (FSGS), treatment-resistant minimal change disease
(TR-MCD) and diabetic nephropathy (DN). The Phase 1 study will evaluate
the safety, tolerability, and pharmacokinetic profile of GFB-887 in
healthy volunteers and in patients with chronic kidney disease. Topline
safety data from healthy volunteers are expected by year-end 2019.
“This Phase 1 study will inform the future clinical development path of
GFB-887, a targeted podocyte-protective therapy in development for
patients with rare glomerular diseases with high unmet need, as well as
patients with DN, the leading cause of end-stage kidney disease,”
said Tony Johnson, M.D., Goldfinch’s President and Chief Executive
Officer. “Initiation of this clinical program is supported by
preclinical data recently presented by Goldfinch demonstrating the
potential of GFB-887 to ameliorate proteinuric kidney disease through
the inhibition of the TRPC5-Rac1 pathway.”
The Phase 1 study will be conducted in two parts. Part 1 comprises a
single-ascending dose (SAD) escalation component and a food effect
component in healthy volunteers, and Part 2 comprises a multiple
ascending dose (MAD) component in healthy volunteers. The Phase 1
program will also include a study in renally-impaired patients. Primary
study objectives include evaluating the safety, tolerability and
pharmacokinetic (PK) profile of single and multiple doses of GFB-887.
Secondary objectives include evaluating the safety, tolerability and PK
of GFB-887 in patients with renal impairment. For more information about
the clinical trial design please visit: www.clinicaltrials.gov
(NCT03970122).
The GFB-887 clinical program is supported by preclinical data in
multiple models of progressive kidney disease, including data from human
stem-cell derived organoids and podocytes, recently presented at the
2019 ISN World Congress of Nephrology meeting in April 2019. A
copy of the poster presentation can be downloaded from the publications
section of the Goldfinch website at goldfinchbio.com/publications.
About GFB-887 and TRPC5
GFB-887 is a sub-type selective,
small molecule TRPC5 ion channel inhibitor in clinical development for
the treatment of FSGS, TR-MCD and DN. TRPC5 is a
calcium-permeable ion channel implicated in the pathogenesis of kidney
disease. Recent evidence has demonstrated that TRPC5 and Rac1, a
critical regulator of cellular motility, form a vicious cycle that
drives pathogenic remodeling of the actin cytoskeleton in podocytes.
This causes podocyte loss and breach of the filtration barrier, which
leads to proteinuria, the hallmark of progressive kidney diseases such
as FSGS, TR-MCD and DN. Inhibition of TRPC5 offers a potential point of
therapeutic intervention to restore podocyte integrity and halt
progression of these diseases.
About FSGS, TR-MCD and DN
FSGS (focal segmental
glomerulosclerosis) is a rare kidney disorder and histopathologic
diagnosis characterized by scarring of the kidney’s filtering units, or
glomeruli, leading to proteinuria, an excess of essential proteins
spilling into the urine. FSGS is associated with loss of podocytes,
terminally differentiated cells of the kidney glomeruli essential for
filtration and proper kidney function. Recent research into the genetics
of kidney disease has identified over 50 genes associated with FSGS and
implicates the podocyte as a central player in the pathogenesis of FSGS.
There are currently no FDA approved treatments available for patients
with FSGS.
Similar to FSGS, treatment-resistant minimal change disease (TR-MCD), is
a rare kidney disorder characterized by podocyte injury and is an
important cause of nephrotic syndrome in children as well as adults.
Clinical hallmarks of MCD include rapid onset of edema and weight gain.
While MCD may be managed with corticosteroids, a subset of patients fail
to respond and are considered treatment-resistant. There are currently
no FDA approved treatments available for patients with TR-MCD.
Diabetic nephropathy (DN) develops in approximately 30-40 percent of
patients who have diabetes and is a leading cause of end-stage kidney
disease, cardiovascular disease and early mortality worldwide. Despite
current therapies, the number of people with DN continues to increase,
highlighting the need for additional treatments that preserve kidney
function.
About Goldfinch Bio
Goldfinch Bio is a biotechnology company that is singularly focused on
discovering and developing precision therapies for patients with kidney
diseases. Just as the goldfinch has long been a symbol of healing and
renewal and was a prominent figure of the Renaissance, Goldfinch Bio is
leading a new age of therapeutic discovery to transform the treatment
paradigm for patients with kidney diseases. Goldfinch was launched in
2016 by Third Rock Ventures, and is headquartered in Cambridge, Mass.
For more information, please visit www.goldfinchbio.com.
Contacts
Media Contact:
The Yates Network
Gina Nugent
617-460-3579
gina@theyatesnetwork.com
Investor
Contacts:
Argot Partners
Kimberly Minarovich / Joseph Rayne
212-600-1902
kimberly@argotpartners.com or joseph@argotpartners.com