PATIENTS & ADVOCATES
At Cantero, we work to establish and build relationships within the hyperoxaluria community. We are committed to approaching these relationships with honesty, integrity and transparency.
We respect the independence of patient organizations and the unique perspective of every advocate, patient and family member.
We actively seek to include the perspective of patients, families and advocates in the drug development process by listening and learning from them.
Resources for patients & families
For more information about patient advocacy efforts at Cantero, contact patientadvocacy@canterotx.com
RESOURCES
HYPEROXALURIA SPECIFIC
Oxalosis & Hyperoxaluria Foundation
Patient advocacy organization dedicated to finding treatments and a cure for all forms of hyperoxaluria
RARE DISEASE
EveryLife Foundation
Patient advocacy organization dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy
Global Genes
Patient advocacy organization focused on connecting, empowering and inspiring the rare disease community
National Organization for Rare Disorders (NORD)
Patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them
SCIENCE
ABOUT PRIMARY HYPEROXALURIA TYPE 1
Primary hyperoxaluria type 1 (PH1) is a rare autosomal recessive disorder driven by a mutation in the AGXT gene, resulting in a reduction of AGT enzymatic activity in the liver. The reduction of AGT activity results in accumulation of glyoxylate, which is converted into oxalate and leads to kidney stones and organ damage.
More than 150 mutations have been identified leading to a significant decrease in AGT activity1,2.
GO = glycolate oxidase
AGT = alanine-glyoxylate aminotransferase
EPIDEMIOLOGY
PH1 is estimated to affect up to 5,000 individuals in the U.S. and Europe.
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The median age of diagnosis is 7-8 years, with ~25% diagnosed in infancy, ~50% in late childhood to early adolescence and the remaining in adulthood4
PH1 diagnosis is often delayed with an estimated 43% already progressed to end stage renal disease (ESRD) at diagnosis4
PH1 DISEASE PROGRESSION
PH1 disease progression is severe with a high degree of morbidity. Excess oxalate produced in the liver results in increased excretion by the kidney. Oxalate combines with calcium to form insoluble calcium oxalate (CaOx) that aggregates as kidney stones and calcification in the kidneys.
This can progress to ESRD, which exacerbates the accumulation of oxalate in the plasma and can promote deposition of calcium oxalate in many organs and tissues3.
1
Mutation in AGXT results in excess oxalate production
2
Excess oxalate combines with calcium to form CaOx stones
3
Nephrocalcinosis (tissue calcification in kidneys) often leading to ESRD
4
Systemic, ectopic calcification affects organs throughout the body
1
Mutation in AGXT results in excess oxalate producton
2
Excess oxalate combines with calcium to form CaOx stones
3
Nephrocalcinosis (tissue calcification in kidneys) often leading to ESRD
4
Systemic, ectopic calcification affects organs throughout the body
UNMET NEED
PH1 is the most severe type of primary hyperoxaluria. The excess oxalate production that leads to kidney stones, chronic kidney disease, and systemic oxalosis strains both patients and caregivers given the frequent medical intervention required and associated financial burden.
Patients with PH1 often need to drink several liters of fluids a day, take daily pyridoxine and/or crystallization inhibitor therapy, and may require intensive dialysis. The only known curative treatment is a combined kidney-liver transplant to restore renal function and prevent further renal damage by restoring normal oxalate production in the liver1,5. A recent survey found that 94% of patients desired new therapies that reduced medical management and provided the chance to have a normal life span6.
REFERENCES
- Cochat, Pierre, and Gill Rumsby. “Primary hyperoxaluria.” New England Journal of Medicine 369.7 (2013): 649-658.
- Hopp, Katharina, et al. “Phenotype-genotype correlations and estimated carrier frequencies of primary hyperoxaluria.” Journal of the American Society of Nephrology 26.10 (2015): 2559-2570.
- Strauss, Sara B., et al. “Primary hyperoxaluria: spectrum of clinical and imaging findings.” Pediatric Radiology 47.1 (2017): 96-103.
- Harambat, Jérôme, et al. “Genotype–phenotype correlation in primary hyperoxaluria type 1: the p. Gly170Arg AGXT mutation is associated with a better outcome.” Kidney International 77.5 (2010): 443-449.
- Lieske, John C., et al. “International registry for primary hyperoxaluria.” American Journal of Nephrology 25.3 (2005): 290-296.
- Lawrence, Jennifer E., and Debra J. Wattenberg. “Primary hyperoxaluria: The patient and caregiver perspective.” Clinical Journal of the American Society of Nephrology (2020).
ABOUT RECURRENT KIDNEY STONE FORMERS
Nearly one in 10 Americans experience a kidney stone in their life and many (20-50%) will develop additional stones in the subsequent 5 years. Frequent kidney stones are painful, associated with substantial medical care and cost, and may contribute to or accelerate progression to end stage renal disease3.
DRIVERS OF KIDNEY STONE FORMATION
The majority (~80%) of kidney stones are composed of calcium oxalate (CaOx)3. Calcium oxalate stone formation is driven by several urinary components that influence the combination of calcium and oxalate into CaOx crystals. Hyperoxaluria, or excess oxalate concentrations in the urine, directly contributes to an increase in CaOx crystals that can attach to kidney tissues and aggregate to form kidney stones.
Small increases in urinary oxalate can cause an increase in CaOx stone formation, with the relative risk of forming a stone increasing by 2.5 to 3.5-fold as urinary oxalate excretion doubles from 20 to 40 mg/day4,5. It was recently shown that individuals with steatotic livers have downregulated AGXT, which can lead to endogenous overproduction of oxalate and may provide a mechanism for the increased risk of nephrolithiasis and chronic kidney disease6.
EPIDEMIOLOGY
Lifetime prevalence of kidney stones (KS) in the United States is nearly 10% and has been rising. Similar prevalence rates are observed in Europe, ranging from approximately 5-10% depending on the region. Approximately 80% of stones are CaOx and 20-50% of patients will experience a recurrence within 5 years3,9.
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Kidney stones have historically been more common in men compared to women, though this disparity is lessening
Non-Hispanic white individuals have the highest prevalence among racial and ethnic groups
UNMET NEED
Prevention of recurrent calcium stones typically starts with behavioral or nutritional changes such as increasing water intake and balanced diets with high calcium and low oxalate. Such diets can be difficult to maintain due to the presence of oxalate in many common foods. Current guidelines for pharmacological treatments are limited and include thiazides (stone formers with hypercalciuria), allopurinol (stone formers with hyperuricosuria), and potassium or magnesium citrate (stone formers with hypocitraturia).
There are no approved treatments to address stone formers with hyperoxaluria. Current medical management is inadequate to prevent stone recurrence in many patients. There remains a significant unmet need for targeted pharmacological approaches in kidney stone disease.
REFERENCES
- Fink, Howard A., et al. “Medical management to prevent recurrent nephrolithiasis in adults: a systematic review for an American College of Physicians Clinical Guideline.” Annals of Internal Medicine 158.7 (2013): 535-543.
- Gambaro, Giovanni, Josè M. Reis-Santos, and Nagaraja Rao. “Nephrolithiasis: why doesn’t our “learning” progress?.” European Urology 45.5 (2004): 547-556.
- Khan, Saeed R., et al. “Kidney stones.” Nature Reviews Disease Primers 2.1 (2016): 1-23.
- Burns, Zachary, et al. “Future treatments for hyperoxaluria.” Current Opinion in Urology 30.2 (2020): 171-176.
- Prochaska, Megan, et al. “Relative supersaturation of 24-hour urine and likelihood of kidney stones.” The Journal of Urology 199.5 (2018): 1262-1266.
- Gianmoena, Kathrin, et al. “Epigenomic and transcriptional profiling identifies impaired glyoxylate detoxification in NAFLD as a risk factor for hyperoxaluria.” Cell Reports 36.8 (2021): 109526.
- Scales Jr, Charles D., et al. “Prevalence of kidney stones in the United States.” European Urology 62.1 (2012): 160-165.
- Osther, Palle JS. “Epidemiology of kidney stones in the European Union.” Urolithiasis. Springer, London, 2012. 3-12.
- Vaughan, Lisa E., et al. “Predictors of symptomatic kidney stone recurrence after the first and subsequent episodes.” Mayo Clinic Proceedings. Vol. 94. No. 2. Elsevier, 2019.
CANTERO APPROACH
Cantero is developing an orally delivered, small-molecule inhibitor of glycolate oxidase (GO). Targeting GO is a clinically validated approach to reduce urinary oxalate by lowering the concentration of glyoxylate, the immediate precursor to metabolic oxalate production in the liver.
CANTERO IN THE CLINIC
BBP-711 is currently being studied in a Phase 1 clinical trial in healthy adult volunteers.
PROGRAMS
PHASE 2
PHASE 3
Follow a manual added link
Primary Hyperoxaluria Type 1 (PH1)
(NCT04876924)
Follow a manual added link
Recurrent Kidney Stone Formers
PROGRAMS
Follow a manual added link
Primary Hyperoxaluria Type 1 (PH1)
(NCT04876924)
Follow a manual added link
Recurrent Kidney Stone Formers
ABOUT US
Cantero Therapeutics, previously recognized as Orfan Biotech, is a clinical stage affiliate of BridgeBio Pharma with the aim of developing new therapies for diseases of hyperoxaluria. The company is led by veteran biotechnology executives whose objective is to partner with patients and physicians to develop a safe and effective treatment for hyperoxalurias as quickly as possible.
Based on the initial discoveries of its scientific founder, Dr. Eduardo Salido at the University of La Laguna in the Canary Islands, Cantero has quickly developed new compounds for the treatment of primary hyperoxaluria and related conditions.
Our name, Cantero, is based on the Spanish term for “stone cutter” and reflects our focus while paying tribute to Dr. Salido along with other Spanish scientists whose work paved the way.
MANAGEMENT
US
Uma Sinha, Ph.D.
Chief Scientific Officer
SA
Scott Adler, M.D.
Chief Medical Officer
CH
CHRIS HENRY
Chemistry, Manufacturing, and Controls
JA
Jocelyn Ashford
Patient Advocacy
RP
Roberta Pantani
Regulatory Affairs
SR
SATISH RAO
Drug Metabolism and Pharmacokinetics
FOUNDERS
Eduardo Salido Ruiz, M.D., Ph.D.
Hans Maag, Ph.D.
Manuel López-Figueroa, Ph.D.
Simon Goldbard, Ph.D.
Miguel Xavier Fernandes, Ph.D
Gerardo Morales-Hierro
Adolfo Nemirovsky, Ph.D.
CANTERO IS A MEMBER OF THE BRIDGEBIO FAMILY
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.
NEWS
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CONTACT
1800 Owens St., STE C-1200
San Francisco, CA 94158
To receive updates or get in touch, contact us at:
info@canterotx.com
