Full Press Release Details
Therapeutics Achieves Study Objective in Oxylanthanum Carbonate (OLC) Pivotal Clinical Trial
Successfully Established Favorable Tolerability and Safety of OLC -
New Drug Application (NDA) Submission Anticipated in Q3 2024 -
Webcast and Conference Call Today at 8:30 A.M. ET -
LOS ALTOS, California, June 25, 2024 -- Unicycive
Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the
"Company or "Unicycive"), today announced positive results from the Oxylanthanum Carbonate (OLC) UNI-OLC-201 pivotal
clinical trial with regard to both safety and tolerability endpoints. OLC is a next-generation lanthanum-based phosphate binding agent
utilizing proprietary nanoparticle technology being developed for the treatment of hyperphosphatemia in patients with chronic kidney disease
The study established promising tolerability of
OLC at clinically effective doses in CKD patients on hemodialysis. In terms of tolerability, OLC had a low rate of discontinuation due
to adverse events (AEs) with only 5/86 patients (6%) discontinuing from the Study. Of the 5 discontinuations, 3 were treatment-related
and 2 were not related to treatment. Importantly, the Company believes the low discontinuation rate for OLC compares favorably to a discontinuation
rate due to AEs of 14% for Fosrenol from its U.S. Food and Drug Administration (FDA)-approved Package Insert.
The primary endpoint was defined as the rate of
discontinuations due to treatment-related AEs leading to discontinuation in the maintenance period. In the UNI-OLC-201 trial, there was
only 1 discontinuation due to a treatment-related AE in the Evaluable Population (n=71), a rate of 1.4%. In the full Safety Population,
a total of 3 patients discontinued due to treatment-related AEs, a rate of 3.5%.
| Patient Population | n | Treatment-Related Discontinuations | Percent | |||||
| Evaluable | 71 | 1 | 1.4 | % | ||||
| Safety | 86 | 3 | 3.5 | % |
The secondary endpoint assessing safety was also
favorable as most treatment-related AEs were mild to moderate in severity and there were no treatment-related serious adverse events (SAEs)
reported in the Safety Population. The treatment-related AEs reported in 5% of patients were diarrhea (9%) and vomiting (6%) which
also compares favorably to Fosrenol and other phosphate binders on the market.
While the study was not designed to evaluate efficacy,
the trial enrolled patients on stable doses of approved hyperphosphatemia medications. At baseline approximately 59% of patients had phosphate
levels 5.5 mg/dL, the level recommended by KDOQI guidelines. After washout from the prior phosphate binders, 90% of patients were
able to achieve phosphate levels 5.5ng/dL at the end of titration with OLC.
"We are immensely pleased with the outcome
of the UNI-OLC-201 clinical trial as the results demonstrate extremely promising tolerability results in real-world dialysis patients,"
said Shalabh Gupta, MD, Chief Executive Officer of Unicycive. "The study was well received by investigators and patients as we were
able to successfully over-enroll the study with 71 evaluable patients. In addition, we were able to obtain phosphate control in 90% of
the Safety Population during titration and OLC proved to be highly potent at lower doses. We would like to thank our investigators, study
coordinators, and the patients who dedicated their time and energy for our clinical trial."
Dr. Gupta continued, "We believe these favorable
results confirming tolerability for OLC are the final data component needed to support submission of a New Drug Application to the FDA
utilizing the 505(b)(2) regulatory pathway. The submission package will also include the previously disclosed preclinical data and the
data establishing bioequivalence to Fosrenol. The encouraging performance of OLC gives us a high degree of confidence and provides potential
clinical validation of OLC's best-in-class commercial promise for patients suffering from hyperphosphatemia."
"Based on real world evidence, approximately
40% of patients on dialysis are unable to achieve adequate serum phosphate control as defined by established KDOQI treatment guidelines1.
The UNI-OLC-201 study was representative of the U.S. dialysis patient population. Uncontrolled hyperphosphatemia is an important problem
for patients and physicians because it can lead to other major complications including cardiovascular disease. I am encouraged by the
results from the OLC pivotal trial, and I believe that a product like OLC that improves phosphate control and reduces the number of pills
could have a meaningful impact on the overall care of CKD patients on dialysis," added Pablo Pergola, MD, PhD, Research Director,
Clinical Advancement Center, Renal Associates, P.A., and principal investigator for the UNI-OLC-201 trial
Conference Call & Webcast Details
Unicycive will host a webcast and conference call
with accompanying slides today at 8:30 a.m. ET. The live and archived webcast may be accessed on the Unicycive website under the Investors
section: Events and Presentations. The live call can be accessed by dialing +1 (646) 876-9923 with Meeting ID: 96518079674 and Passcode:
Presentation slides will be provided at the start
of the conference call on the Unicycive website under the Investors section: Events and Presentations.
OLC-201 Data Summary
The results from the trial are focused on two
patient populations: the full Safety Population and the Evaluable Population. The Safety Population (n=86) included all patients who entered
titration and received at least one dose of OLC. The Evaluable Population (n=71) required a patient to have a serum phosphate level of
5.5 mg/dL at the end of titration and received at least one dose of OLC in the maintenance period.
Once patients were enrolled into the trial, they
went through a washout period for two weeks to clear their current phosphate binder from the body. Participants were initially dosed at
500 mg of OLC three times a day (TID) and titrated to a clinically effective dose that is defined as the dose required to achieve a serum
phosphate level of 5.5 mg/dL. The maximum dose of OLC tested was 3000 mg/day (1000 mg TID). As a reminder, all approved phosphate
binders are administered on a dose titration schedule based on the control of serum phosphate. Once titrated to a clinically effective
dose, patients were then treated in a maintenance period with OLC for four weeks to evaluate tolerability.
Demographics and Enrollment Summary
In the study, 106 patients were enrolled, of
which 86 patients entered titration and were followed as the Safety Population. Of the 86, 78 entered the maintenance period. Of the
78 patients that entered maintenance, 7 patients did not have phosphate control, leaving an Evaluable Population of 71 patients, exceeding
the planned enrollment number of 60. Of the 86 patients, the trial enrolled 47 males and 39 females with a mean age of 62. Renvela
was the most prescribed phosphate binder for patients entering the study.
Primary Endpoint - Tolerability:
The objective of the OLC-201 trial was to evaluate
the tolerability of clinically effective doses of OLC in CKD patients on dialysis. A clinically effective dose was established when a
patient achieved a serum phosphate level 5.5 mg/dL. Tolerability was assessed based on the incidence of treatment-related AEs leading
to discontinuation from the study in the maintenance period. In the OLC-201 trial, there was only 1 discontinuation due to a treatment-related
AE in the Evaluable Population, a rate of 1.4%. In the Safety Population of 86 patients there were only 3 treatment-related discontinuations,
a rate of 3.5%. In total, 5 patients discontinued due to AEs in the Safety Population, 3 were related to OLC and 2 were deemed unrelated
Secondary Endpoint - Safety:
The secondary endpoint assessing safety was reported
as the treatment-related AEs occurring in 5% of patients. The safety analysis covered all 86 patients in the Safety Population. Consistent
with the AEs observed with other phosphate binders, the AEs were gastrointestinal related with diarrhea and vomiting being the most common
at 9% and 6% respectively. There were no treatment-related serious adverse events (SAEs). Six patients experienced SAEs but those were
deemed not related to OLC treatment. Most treatment-related AEs were mild to moderate in severity with only 2 AEs reported as severe.
Unicycive continues to assess the pharmacokinetics
from this trial and those final data will be included in the NDA package.
Serum Phosphate Control
While the UNI-OLC-201 study was not designed to
evaluate efficacy, the trial enrolled patients on stable doses of approved hyperphosphatemia medications. At baseline 59% of patients
had phosphate levels 5.5 mg/dL, the level recommended by KDOQI guidelines. After washout from the prior phosphate binders, 90% of
patients were able to achieve phosphate levels 5.5ng/dL at the end of titration with OLC. This includes the last serum phosphate levels
from all patients including those that discontinued during titration: 77/86 (90%).
In addition, 69% of the 71 Evaluable Patients
achieved a target serum phosphate level of 5.5 mg/dL at OLC doses of 1500 mg/day or lower.
About Oxylanthanum Carbonate (OLC)
Oxylanthanum carbonate is a next-generation lanthanum-based
phosphate binding agent utilizing proprietary nanoparticle technology being developed for the treatment of hyperphosphatemia in patients
with chronic kidney disease (CKD). OLC has over forty issued and granted patents globally. Its potential best-in-class profile may have
meaningful patient adherence benefits over currently available treatment options as it requires a lower pill burden for patients in terms
of number and size of pills per dose that are swallowed instead of chewed. Based on a survey conducted in 2022, Nephrologists stated that
the greatest unmet need in the treatment of hyperphosphatemia with phosphate binders is a lower pill burden and better patient compliance.2 The
global market opportunity for treating hyperphosphatemia is projected to be in excess of $2.5 billion in 2023, with the United States
accounting for more than $1 billion of that total.3 Despite the availability of several FDA-cleared medications, 75 percent