Kidney drug developer Tricida’s shares shot up on Thursday, after long-term data highlighted its lead drug’s durable efficacy and safety profile as a treatment for metabolic acidosis, in which faulty kidneys are not able to expel the acid causing a buildup in the body.
The drug — TRC101 — is designed to bind to hydrochloric acid in the GI tract, triggering the ejection of acid via excretion — thereby diminishing acid levels and stimulating blood bicarbonate. The company expects to submit an application to market the drug by the second half of this year for the condition commonly caused by chronic kidney disease (and believed to accelerate the progression of CKD), increase the risk of muscle wasting and cause the loss of bone density. If approved, it will be the first drug to win the FDA nod specifically for metabolic acidosis.
Data posted last June showed the drug induced a statistically significant increase in blood bicarbonate in 208 patients after 12 weeks in a Phase III study, hours after the company advertised plans to go public in a $150 million IPO. On Thursday, Tricida revealed data from a 40-week extension to that study in the 185 patients that completed one year as part of the trial.
The drug met all the primary and secondary goals of the extension portion of the trial. The main goal of the extension study was the assessment of the long-term safety profile of TRC101 versus placebo — and data showed 2.6% of the patients on TRC101 versus 9.8% on placebo discontinued the 40-week treatment period prematurely.
Significantly, data on the drug’s durability were also positive. Over 52 weeks, 63% of the 111 TRC101-treated patients exhibited an increase in blood bicarbonate level of at least 4 milliequivalents per liter, or achieved a blood bicarbonate level in the normal range — compared to 38% of the 74 placebo subjects (p=0.0015).
The statistical plan also called for the evaluation of TRC101 versus placebo for the composite endpoint of: all-cause mortality, dialysis/kidney transplant or a ≥50% decline in estimated glomerular filtration rate (a test used to check how well the kidneys are working) — DD50 when taken together — over the 52 week period. Of the 124 subjects given TRC101, 4% (5) had a DD50 event, including one patient who initiated dialysis. In contrast, of the 93 subjects randomized to the placebo group, 10.8% (10) subjects had a DD50 event, including four deaths and one who initiated dialysis.
While the trial was not powered to assess all-cause mortality and/or the progression of CKD outcomes, Tricida said it observed a 65% reduction in the annualized event rate of the composite endpoint of all-cause mortality and/or the progression of CKD in TRC101-treated subjects versus the placebo group.
“The 52-week…results far exceeded our expectations,” said company chief Gerrit Klaerner said in a statement. “We did not anticipate that we would observe evidence of clinical benefit beyond the increase in blood bicarbonate in patients treated with TRC101 until the read out of the results of our postmarketing trial…in the 2022 to 2023 timeframe.”
Shares of the South San Francisco-based drug developer $TCDA leapt more than 57% on Thursday, closing at $37.80.
Cowen’s Phil Nadeau, who deemed the data ‘impressive,’ expects TRC101 will achieve $1 billion in revenue by 2025.
(T)here is a major need to control metabolic acidosis and slow the progression of CKD. Though sodium bicarbonate is effective, its high sodium concentration makes most CKD patients ineligible for it…With 25 million people in the U.S. having stage 3, 4, or 5 CKD, TRC101 also addresses a large patient population, and even modest penetration could yield billions in sales.
(T)he composite endpoint data combined with the physical functioning scores show clear, clinically meaningful benefits to patients. In fact, the dramatic reduction in the composite endpoint implies that physicians need to treat at most 3 patients in order for 1 to derive a clinically meaningful reduction in CKD progression.
Separately, Tricida on Thursday said it had amended its debt facility with Hercules Capital, raising the total amount available to up to $200 million from the $100 million agreed in February 2018.
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