The following excerpt is from the company's SEC filing.
July 12, 2021
successfully completes Phase 1b safety study; on track to start two Phase 2a studies in Q3
2021 with initial data expected in Q4 2021-
-Reloxaliase advancing in
Phase 3 study; revised interim analysis now targeted for Q1 2022-
NEWTON, Mass., July 12, 2021 (GLOBE NEWSWIRE) Allena Pharmaceuticals,
Inc. (NASDAQ: ALNA), a late-stage biopharmaceutical company dedicated to discovering, developing and commercializing
oral enzyme therapeutics to treat
patients with rare and severe metabolic and kidney disorders, today provided progress updates on its clinical programs:
oral urate degrading enzyme in development for the treatment of patients with hyperuricemia and gout in the setting of chronic kidney disease (CKD) and
oral oxalate degrading enzyme in development for the treatment of patients with enteric
Phase 1b Study Results and Planned Phase 2A Program
is an investigational
orally administered enzyme for the treatment of hyperuricemia and gout, a metabolic disorder
characterized by high systemic levels of uric acid that can lead to several complications, including arthritis, kidney stones, and chronic kidney disease.
The Company recently completed a Phase 1b multiple ascending dose study of
The study included 18 healthy
volunteers, who received either
or placebo (2:1 randomization) for seven days. There were two cohorts consisting of nine subjects each, with the first receiving three capsules of
three times daily, and the second receiving five capsules of
three times daily.
was well tolerated with no
evidence of systemic absorption, as confirmed by an
immunosorbent assay (ELISA). Evaluation of clinical and laboratory parameters revealed no significant safety signals and no serious adverse
events were reported.
As previously reported, the Company is preparing to initiate two randomized, double-blind, placebo-controlled Phase 2a studies to
obtain initial bioactivity data and additional safety data for
in patients with hyperuricemia and gout during the third quarter of 2021. Initial results from the Phase 2a program are expected during
the fourth quarter of 2021.
An inpatient study (Study 201) is planned to initially enroll 12 patients with hyperuricemia randomized (2:1) to receive
either five capsules of
or matching placebo three times daily during a
treatment period. Key bioactivity endpoints will include serum uric acid level,
urine uric acid level, and renal clearance of uric acid. Following evaluation of the data from the initial 12 patients, the Company will make a determination regarding potentially extending the study to
include up to an additional 12 patients, either to obtain additional data at the five-capsule, three-times-daily dose, or to evaluate a different dose.
An outpatient study (Study 202) is planned to enroll 24 hyperuricemic patients with gout and
chronic kidney disease randomized (2:1) to receive either five capsules of
or matching placebo, three
times daily, during a
treatment period. Two cohorts of 12 patients each are planned, with the first cohort consisting of patients with an eGFR (estimated glomerular filtration rate, a measure of renal
mls/minute, and the second consisting of patients with an eGFR of
mls/minute. Key bioactivity endpoints will include serum uric acid level,
The limitations of existing gout treatments were highlighted in the Companys
recent KOL Webinar
. Specifically, managing gout in the setting of
advanced chronic kidney disease remains a significant challenge for clinicians because currently available agents are either dose-limited or contraindicated in these patients. There are approximately 500,000 patients with gout and advanced chronic
kidney disease in the United States.
Reloxaliase: Revised Plan for First Interim Analysis
Reloxaliase is an investigational,
orally administered enzyme for the treatment of enteric hyperoxaluria, a metabolic disorder characterized by high levels of urinary oxalate (UOx), which can lead to kidney stone disease and chronic
kidney disease. There are currently no approved therapies for this disorder, which affects approximately 250,000 patients in the United States. Reloxaliase exerts its effect by breaking down oxalate in the gastrointestinal (GI) tract, reducing the
absorption of dietary oxalate. The Company previously completed the
trial, the first of two pivotal Phase 3 clinical trials of reloxaliase, which demonstrated a statistically significant reduction in
UOx levels during weeks
the primary endpoint of the study. The Company is currently studying reloxaliase in the adaptive-design
trial, the second of two
pivotal Phase 3 clinical trials in its URIROX program.
is a multicenter, global, randomized,
double-blind, placebo-controlled study designed to evaluate the safety and efficacy of reloxaliase in patients with enteric hyperoxaluria over a minimum treatment period of two years. The trial is designed to enroll 200 patients with
UOx excretion greater than or equal to 50 mg/day and a history of kidney stones, and includes patients with both normal kidney function and reduced kidney function up to Stage 3 chronic kidney disease (eGFR
> 30 mL/min). The primary efficacy endpoint of
is the percent change from baseline in
UOx excretion during weeks
the same primary endpoint as
Secondary endpoints in
include the percent change from baseline in
UOx excretion during weeks
and the proportion of subjects with a
20% reduction from baseline in
UOx excretion during Weeks
The primary long-term efficacy endpoint to confirm clinical benefit is kidney stone disease progression, defined as a composite of
either symptomatic kidney stones or finding of new or enlarged kidney stones using imaging, over a minimum treatment period of two years. Secondary long-term efficacy endpoints to confirm clinical benefit include change in eGFR from baseline and
resource utilization for the management of kidney stones.
incorporates adaptive design elements that could, if necessary, allow for increasing the sample size and/or duration of treatment based upon
the results of two interim analyses.
As part of the
adaptive design, the Company had previously
planned to conduct the first interim analysis after 130 subjects had been treated for at least six months and had estimated the timing of this analysis to be in the second or third quarter of 2022. Given the adverse impact of the
global pandemic on the rate of patient enrollment in this global study, and considering as well that enrollment in the study began in early 2019, the Company has modified its plans and now expects to
conduct the first interim analysis, which will include all subjects who were enrolled in the trial as of the end of November 2021, during the first quarter of 2022. Based upon the number of subjects enrolled to date, the number of subjects currently
in screening, and managements estimate of expected enrollment over the next several months through November, the Company estimates that this revised interim analysis would include UOx data during weeks
for approximately 80 patients but would not include sufficient data to evaluate UOx levels during weeks
or the blinded rate of kidney stone events, as
previously planned. The Company will submit its revised plan for the first interim analysis to the Food and Drug Administration (FDA) as part of its planned update to the
statistical analysis plan and
adaptive design charter.
The revised interim analysis, which will be conducted by an independent data monitoring committee, will assess whether the study
continues to be adequately powered to evaluate efficacy against the primary endpoint, the change in UOx levels during weeks
versus baseline, with the planned enrollment of 200 subjects, or whether the
study size should be increased. As a result of conducting an earlier interim analysis with a smaller sample size than previously planned, the Company anticipates that the ultimate size of the study may be larger than would have otherwise been
required, but that if this occurs, any such increase would be modest relative to an increase, if any, that would have been necessary had the first interim analysis been conducted with
data on 130
patients, as previously planned. Any adjustment in study size would be designed to ensure that the statistical power of the study remains sufficiently robust. As previously planned, the interim analysis will also include an assessment of futility
with respect to the primary endpoint.
The Company does not anticipate any changes to the planned second interim analysis, which will also include an
assessment of the secondary endpoint of change in UOx levels during weeks
and of unblinded kidney stone events. The second interim analysis is expected to be conducted once 200 subjects have reached six
months of treatment and is designed to enable a potential filing for accelerated approval for reloxaliase on the basis of UOx levels. The Company expects to provide revised guidance on its expectations for the timing of the second interim analysis
and topline data from the
study following completion of the initial interim analysis in the first quarter of 2022.
We are encouraged by the progress of our two clinical programs, which we have sustained despite the challenges presented by the
pandemic, stated Louis Brenner, M.D., President and Chief Executive Officer of Allena Pharmaceuticals, Inc. We are focused on continuing to advance our bacterial-derived oral enzyme product
candidates through clinical development for patients with significant unmet needs in enteric hyperoxaluria and gout with CKD. We believe that the recent attention to the potential of synthetic biology and microbiome-derived therapeutics highlights
the potential broad applicability of our technology.
In our ongoing
study in enteric
hyperoxaluria, our trial simulation analyses give us confidence that an earlier
interim analysis will not result in a material impact on the size of the trial relative to any increase in size that
might have been necessary had the first interim analysis been conducted with a larger data set as previously planned, and we believe it will provide meaningful and timely feedback for the conduct and ultimate completion of the adaptive design
trial, added Dr. Brenner. In addition, we look forward to seeing the first data from hyperuricemic subjects treated with
late this year.
About Allena Pharmaceuticals
Inc. is a biopharmaceutical company dedicated to discovering, developing and commercializing
oral biologic therapeutics to treat patients with rare and
severe metabolic and kidney disorders. Allenas lead product candidate, reloxaliase, is currently being evaluated in a pivotal Phase 3 clinical program for the treatment of enteric hyperoxaluria, a metabolic disorder characterized by markedly
elevated urinary oxalate levels and commonly associated with kidney stones, chronic kidney disease and other serious kidney disorders. Allena is also developing
for the treatment of hyperuricemia in
the setting of gout and advanced chronic kidney disease, with a Phase 1 multiple-ascending dose study recently completed and a Phase 2a program planned for the second half of 2021.
This release contains
forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, without limitation, statements concerning the future clinical, regulatory and commercial potential of reloxaliase,
statements regarding enrollment and the timing of the planned interim analysis in the
trial, the impact of an earlier initial interim analysis on the size and duration of the
trial, statements regarding Allenas strategy of pursuing a BLA submission for reloxaliase based upon data from its URIROX program using the accelerated approval regulatory pathway, which strategy is
predicated on the FDAs agreement with our predictive model supporting a relationship between UOx levels and stone formation rates, statements regarding Allenas development of
timing of planned clinical trials and the announcement of topline date for these trials, and statements regarding Allenas financial position and need for capital. Any forward-looking statements in this press release are based on
managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements.
These risks and uncertainties include, but are not limited to: market and other conditions, the timing for completion of Allenas clinical trials of its product candidates, risks associated with obtaining, maintaining and protecting
intellectual property; risks associated with Allenas ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties; the risk of competition from other companies developing products for
similar uses; risks associated with Allenas financial condition and its need to obtain additional funding to support its business activities, including the future clinical development of reloxaliase and its ability to continue as a going
concern; risks associated with Allenas dependence on third parties; and risks related to the
coronavirus. For a discussion of other risks and uncertainties, and other important factors, any of
which could cause Allenas actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Allenas Quarterly Report on Form
for the quarter ended March 31, 2021, as well as discussions of potential risks, uncertainties and other important factors in Allenas subsequent filings with the Securities and Exchange Commission. All information in this press release is
as of the date of the release, and Allena undertakes no duty to update this information unless required by law.
Investor Relations Contact:
Ashley R. Robinson
LifeSci Advisors, LLC
Berry & Company Public Relations
Source: Allena Pharmaceuticals, Inc.
The above information was disclosed in a filing to the SEC. To see the filing, click here.
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