TORONTO, ONTARIO--(Marketwired - Dec. 21, 2016) - Revive Therapeutics Ltd. ("Revive" or the "Company") (TSX VENTURE:RVV)(RVVTF), a company focused on commercializing treatments for rare diseases such as Cystinuria and Wilson's disease, today announced it has engaged NYU School of Medicine as one of the Company's clinical sites in the U.S. for its Phase 2 clinical study for Cystinuria.
"I am very pleased to have NYU School of Medicine as a clinical site for our Phase 2 clinical study for Cystinuria," said Fabio Chianelli, President of Revive. "We are fortunate to have established a relationship with one of the leading institutions that provides treatment for Cystinuria and to have David S. Goldfarb, MD, professor of medicine and neuroscience and physiology at NYU School of Medicine, as the Principal Investigator for the entire study and Lama Nazzal, MD, assistant professor of medicine at NYU School of Medicine, as the Principal Investigator at the site."
About Cystinuria
Cystinuria is a rare autosomal recessive genetic disorder that causes high levels of cystine in the urine thus causing kidney stones to form. The resulting kidney stones are often large and recurrent and lead to significant morbidity and sometimes loss of kidney function. There are approximately between 10,000 and 12,000 patients affected with cystinuria in the U.S. and the Company estimates a market opportunity of $500 million.
Current drugs approved by the US FDA for the treatment of Cystinuria include Cuprimine® (D-penicillamine), which is a registered trademark of Valeant Pharmaceuticals International, Inc. and Thiola® (Tiopronin), which is marketed by Retrophin, Inc. Both patent protection and the seven-year period of marketing exclusivity from the orphan drug designation for Cuprimine® and Thiola® have expired. Since the approval of Thiola® in 1988, there have been no significant improvements in the treatment of cystinuria. Revive is repurposing Bucillamine as a potential new treatment in Cystinuria. Bucillamine is an oral small molecule drug prescribed for rheumatoid arthritis in Japan and South Korea for nearly 30 years. Bucillamine has a chemical structure similar to Thiola®, but has two active thiol groups versus only one for Thiola®. The Company received US FDA orphan designation status for the use of Bucillamine for the treatment of Cystinuria.
About Cystinuria Phase 2 Study
The Cystinuria Phase 2 study is a multi-center, dose escalation trial to assess the safety and effectiveness of Bucillamine on urinary cystine excretion and cystine capacity in patients with Cystinuria. The primary outcome measures are the incidence of treatment-emergent adverse events along with secondary outcome measuring 24-hr urine cysteine excretion and 24-hr urine cystine capacity, i.e., the capacity of a patient's urine to solubilize or precipitate. The study plans to enroll up to 30 subjects in at least 5 clinical sites in the U.S. and subject enrollment is expected to be completed by Q2-2017.
About Revive Therapeutics Ltd.
Revive Therapeutics Ltd. (TSX VENTURE:RVV)(RVVTF) is focused on commercializing treatments for rare diseases such as Cystinuria, which is in a Phase 2 clinical study in the U.S. Additional information on Revive is available at www.ReviveThera.com.
"I am very pleased to have NYU School of Medicine as a clinical site for our Phase 2 clinical study for Cystinuria," said Fabio Chianelli, President of Revive. "We are fortunate to have established a relationship with one of the leading institutions that provides treatment for Cystinuria and to have David S. Goldfarb, MD, professor of medicine and neuroscience and physiology at NYU School of Medicine, as the Principal Investigator for the entire study and Lama Nazzal, MD, assistant professor of medicine at NYU School of Medicine, as the Principal Investigator at the site."
About Cystinuria
Cystinuria is a rare autosomal recessive genetic disorder that causes high levels of cystine in the urine thus causing kidney stones to form. The resulting kidney stones are often large and recurrent and lead to significant morbidity and sometimes loss of kidney function. There are approximately between 10,000 and 12,000 patients affected with cystinuria in the U.S. and the Company estimates a market opportunity of $500 million.
Current drugs approved by the US FDA for the treatment of Cystinuria include Cuprimine® (D-penicillamine), which is a registered trademark of Valeant Pharmaceuticals International, Inc. and Thiola® (Tiopronin), which is marketed by Retrophin, Inc. Both patent protection and the seven-year period of marketing exclusivity from the orphan drug designation for Cuprimine® and Thiola® have expired. Since the approval of Thiola® in 1988, there have been no significant improvements in the treatment of cystinuria. Revive is repurposing Bucillamine as a potential new treatment in Cystinuria. Bucillamine is an oral small molecule drug prescribed for rheumatoid arthritis in Japan and South Korea for nearly 30 years. Bucillamine has a chemical structure similar to Thiola®, but has two active thiol groups versus only one for Thiola®. The Company received US FDA orphan designation status for the use of Bucillamine for the treatment of Cystinuria.
About Cystinuria Phase 2 Study
The Cystinuria Phase 2 study is a multi-center, dose escalation trial to assess the safety and effectiveness of Bucillamine on urinary cystine excretion and cystine capacity in patients with Cystinuria. The primary outcome measures are the incidence of treatment-emergent adverse events along with secondary outcome measuring 24-hr urine cysteine excretion and 24-hr urine cystine capacity, i.e., the capacity of a patient's urine to solubilize or precipitate. The study plans to enroll up to 30 subjects in at least 5 clinical sites in the U.S. and subject enrollment is expected to be completed by Q2-2017.
About Revive Therapeutics Ltd.
Revive Therapeutics Ltd. (TSX VENTURE:RVV)(RVVTF) is focused on commercializing treatments for rare diseases such as Cystinuria, which is in a Phase 2 clinical study in the U.S. Additional information on Revive is available at www.ReviveThera.com.