Akebia Therapeutics Announces First Patient Dosed in Phase 2 Clinical Trial of Praliciguat for the Treatment of Focal Segmental Glomerulosclerosis (FSGS)

Rhea-AI Summary

Akebia Therapeutics (Nasdaq: AKBA) announced that the first patient has been dosed in a Phase 2 randomized, double-blind, placebo-controlled study of oral once-daily praliciguat for biopsy-confirmed focal segmental glomerulosclerosis (FSGS).

The study will randomize ~60 adults on maximally tolerated ACEi/ARB therapy 1:1 to praliciguat or placebo for a 24-week double-blind period followed by a 24-week open-label extension. The primary endpoint is change in urine protein-to-creatinine ratio (UPCR) at Week 24; a key secondary endpoint is partial remission defined as 40% UPCR reduction and UPCR <1.5 g/g.

Positive

• First patient dosed in Phase 2 study of praliciguat
• Study uses randomized, double-blind, placebo-controlled design
• Primary endpoint: UPCR change at Week 24
• Prior studies showed no significant safety issues in Phase 1 and earlier Phase 2 tests

Negative

• Relatively small sample size of ~60 patients
• Primary readout at 24 weeks may limit near-term efficacy clarity

Key Figures

FSGS patients approximately 40,000 patients Estimated U.S. prevalence cited for FSGS

Phase 2 sample size approximately 60 patients Adults with biopsy-confirmed FSGS in the praliciguat trial

Double-blind duration 24 weeks Initial randomized, placebo-controlled treatment period

Open-label duration 24 weeks Extension where all participants receive praliciguat

Primary endpoint timepoint Week 24 Change from baseline in urine protein-to-creatinine ratio

Partial remission threshold 40% UPCR reduction Component of secondary endpoint at Week 24

UPCR criterion UPCR < 1.5 gram/gram Additional requirement for partial remission definition

Randomization ratio 1:1 Praliciguat versus placebo allocation in Phase 2 trial

Market Reality Check

$1.53 Last Close

Volume Volume 2,135,590 vs 20-day average 2,811,908 (relative volume 0.76), suggesting no outsized trading ahead of this news. normal

Technical Shares at $1.51, trading below the $2.68 200-day moving average and close to the 52-week low of $1.445.

Peers on Argus

Peers show mixed moves with declines in AQST (-3.56%) and ORGO (-3.92%) while SIGA, ESPR, and DVAX post modest gains. This pattern, alongside AKBA’s -2.58% move, points to stock-specific factors rather than a clear sector-wide trend.

Historical Context

Date	Event	Sentiment	Move	Catalyst
Dec 01	Inducement grants	Neutral	-2.6%	Stock option grants to new hires under Nasdaq inducement rule.
Dec 01	Pipeline expansion	Positive	-4.4%	Launch of rare kidney disease pipeline including praliciguat and AKB-097.
Nov 24	Conference participation	Neutral	+2.6%	Planned presentation at Piper Sandler healthcare conference.
Nov 10	Earnings report	Positive	-18.1%	Q3 2025 revenue growth to <b>$58.8M</b> and return to profitability.
Nov 06	Clinical data update	Positive	-5.2%	Favorable mortality and hospitalization composite for vadadustat vs ESA.

Pattern Detected

Recent history shows several negative price reactions following otherwise constructive or positive updates, including earnings strength and clinical data, suggesting a pattern of selling into good news.

Recent Company History

Over the last few months, Akebia has balanced commercial progress with strategic shifts. Q3 2025 results on Nov 10 showed higher revenue of $58.8M and net income of $0.5M, yet shares fell 18.14%. Positive vadadustat outcomes disclosed on Nov 6 and the rare kidney disease pipeline announcement on Dec 1 (praliciguat and AKB-097) also saw negative moves. Today’s FSGS Phase 2 dosing milestone extends that rare kidney pipeline story.

Market Pulse Summary

This announcement advances Akebia’s mid-stage rare kidney disease strategy by dosing the first patient in a Phase 2 FSGS trial of praliciguat. The study plans to enroll approximately 60 adults on background ACEi or ARB therapy, with a primary UPCR endpoint at Week 24. In context of earlier vadadustat data and the December 2025 pipeline update, investors may watch enrollment progress, safety signals, and proteinuria reductions as key markers of program momentum.

Key Terms

focal segmental glomerulosclerosis medical

"for the Treatment of Focal Segmental Glomerulosclerosis (FSGS)"

Focal segmental glomerulosclerosis is a chronic kidney disease in which some of the tiny filters in the kidneys (glomeruli) become scarred in parts, reducing the organ’s ability to remove waste and control fluid balance. For investors, it matters because the condition can drive sustained demand for specialized drugs, diagnostic tests, and treatment services, influence healthcare spending and reimbursement dynamics, and affect the commercial prospects of companies developing therapies or diagnostics for rare kidney disorders.

urine protein-to-creatinine ratio medical

"evaluating changes in urine protein-to-creatinine ratio, a widely-accepted endpoint"

A urine protein-to-creatinine ratio compares the amount of protein in a urine sample to the amount of creatinine (a stable waste product) to estimate how much protein the kidneys are leaking without needing a full 24-hour collection. Think of it like measuring how sweet a drink is relative to its concentration so you can compare samples fairly. Investors watch this measure because rising values indicate potential kidney damage, which can signal safety issues for drugs, affect patient populations, and influence regulatory and commercial outcomes.

soluble guanylate cyclase medical

"an oral, once-daily soluble guanylate cyclase (sGC) stimulator being evaluated"

Soluble guanylate cyclase is a protein in cells that makes a small signaling molecule called cyclic GMP when it senses nitric oxide; think of it as a thermostat that converts a chemical signal into a change in cell behavior. Investors track it because drugs that stimulate or activate this protein can widen blood vessels, reduce blood pressure, and alter heart or lung function, making it a key target in cardiovascular and respiratory drug development.

angiotensin-converting enzyme inhibitor medical

"receiving maximally tolerated doses of an angiotensin-converting enzyme inhibitor (ACEi)"

A angiotensin-converting enzyme (ACE) inhibitor is a class of medicine that lowers blood pressure and eases stress on the heart by blocking a chemical step that tightens blood vessels; think of it as loosening a constricting valve in the body’s plumbing. Investors care because these drugs drive sales in hypertension, heart failure and kidney protection, and changes in clinical trial results, approvals, patent status or safety signals can materially affect a drugmaker’s revenue and stock value.

angiotensin receptor blocker medical

"or an angiotensin receptor blocker (ARB) will be randomized"

An angiotensin receptor blocker (ARB) is a class of prescription medicines that lower blood pressure by preventing a natural hormone from tightening blood vessels and causing the body to retain salt and water. For investors, ARBs represent a major, steady market in cardiovascular care—sales depend on clinical trial results, regulatory approvals, patent status and pricing; think of an ARB as a key that keeps a door open so blood can flow more easily, affecting long‑term drug demand.

randomized, double-blind, placebo-controlled medical

"The Phase 2, randomized, double-blind, placebo-controlled, multicenter study is designed"

A "randomized, double-blind, placebo-controlled" process is a method used to test the effectiveness of a new treatment or intervention. Participants are randomly assigned to different groups, with one receiving the real treatment and the other a fake version, called a placebo. Neither the participants nor the researchers know who is receiving which, which helps ensure unbiased results. For investors, this rigorous approach increases confidence that the findings are accurate and not influenced by guesswork or bias.

partial remission medical

"secondary endpoint is defined as the percentage of patients with partial remission"

Partial remission is a clinical outcome where a disease’s signs or symptoms have decreased substantially but not disappeared entirely, like turning a bright light down rather than switching it off. For investors, it signals that a treatment is having a meaningful effect on patients and can influence regulatory decisions, future sales potential and stock value, because it often represents progress toward full remission or approval milestones.

biopsy-confirmed medical

"being evaluated for the treatment of biopsy-confirmed FSGS, a rare kidney disease"

A diagnosis described as "biopsy-confirmed" means a tissue sample was taken and examined under a microscope to directly verify the presence or type of disease. For investors, this matters because biopsy confirmation is considered a reliable, objective proof point that strengthens clinical trial results, regulatory submissions, and market claims—similar to having a clear photograph of evidence rather than a secondhand description.

AI-generated analysis. Not financial advice.

Clinical trial evaluating changes in urine protein-to-creatinine ratio, a widely-accepted endpoint measuring risk reduction of kidney failure

CAMBRIDGE, Mass., Jan. 06, 2026 (GLOBE NEWSWIRE) -- Akebia Therapeutics^®, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, today announced that the first patient has been dosed in a Phase 2 clinical trial of praliciguat, an oral, once-daily soluble guanylate cyclase (sGC) stimulator being evaluated for the treatment of biopsy-confirmed FSGS, a rare kidney disease, with plans to assess its use in other rare podocytopathies in the future.

“We are pleased by the timely initiation of this important Phase 2 clinical trial of praliciguat and have successfully dosed the first patient following the defined screening process,” said Dr. Steven K. Burke, Chief Medical Officer. “FSGS is a rare kidney disease that affects approximately 40,000 patients in the U.S, and there are no approved treatments. Praliciguat is a promising therapeutic candidate with no significant safety issues observed in Phase 1 studies in healthy volunteers and Phase 2 studies in heart failure and diabetic kidney disease. Praliciguat is a key component of our recently announced mid-stage rare kidney disease pipeline.”

The Phase 2, randomized, double-blind, placebo-controlled, multicenter study is designed to evaluate the efficacy and safety of praliciguat in adults with biopsy-confirmed FSGS. Approximately 60 patients who are already receiving maximally tolerated doses of an angiotensin-converting enzyme inhibitor (ACEi) or an angiotensin receptor blocker (ARB) will be randomized 1:1 to receive praliciguat or placebo for an initial 24-week treatment period. Following this double-blind period, all participants will receive praliciguat in the open-label portion of the study for an additional 24 weeks. The primary endpoint is defined as change from baseline in urine protein-to-creatinine ratio (UPCR) measured at Week 24. The secondary endpoint is defined as the percentage of patients with partial remission at Week 24 (measured as a 40% UPCR reduction and UPCR<1.5 gram/gram). More information about this study, can be found here.

About Praliciguat 
Akebia licensed praliciguat from Cyclerion Therapeutics, Inc. No significant safety issues were observed with praliciguat in Phase 1 studies in healthy volunteers and Phase 2 studies in heart failure and diabetic kidney disease. Praliciguat adverse events were infrequent and consistent with its known blood pressure lowering effect.

About Akebia Therapeutics 
Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. Akebia was founded in 2007 and is headquartered in Cambridge, Massachusetts. For more information, please visit our website at www.akebia.com, which does not form a part of this release. 

Forward-Looking Statements
Statements in this press release regarding Akebia Therapeutics, Inc.'s ("Akebia's") strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and include, but are not limited to, statements regarding: Akebia’s plans and expectations with respect to its Phase 2 clinical trial of praliciguat, including its use in other rare podocytopathies and timing thereof; Akebia’s beliefs and statements with respect to FSGS, including the number of U.S. patients and that there are no approved treatments; Akebia’s plans and expectations with respect to praliciguat, including that is a promising therapeutic candidate with no significant safety issues observed in Phase 1 studies in healthy volunteers and Phase 2 studies in heart failure and diabetic kidney disease and that it is a key component in its mid-stage kidney disease pipeline. The terms "intend," "believe," "plan," "goal," "potential," "anticipate, "estimate," "expect," "future," "will," "continue," derivatives of these words, and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to, risks associated with: the potential therapeutic benefits, safety profile, and effectiveness of Vafseo and Akebia’s development candidates; the results of preclinical and clinical research; Akebia’s ability to enroll patients in its clinical trials; decisions made by health authorities, such as the FDA, with respect to regulatory filings and other interactions; the potential demand and market potential and acceptance of, as well as coverage and reimbursement related to, Vafseo^®, including estimates regarding the potential market opportunity; the competitive landscape for Auryxia^® and Vafseo, including generic entrants and the timing thereof; the ability of Akebia to attract and retain qualified personnel; Akebia's ability to achieve and maintain profitability and to maintain operating expenses consistent with its operating plan; manufacturing, supply chain and quality matters and any recalls, write-downs, impairments or other related consequences or potential consequences; early termination of any of Akebia's collaborations; and changes in the geopolitical environment and uncertainty surrounding U.S. trade policy on tariffs. Other risks and uncertainties include those identified under the heading "Risk Factors" in Akebia's Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, and other filings that Akebia may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and, except as required by law, Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release.

Akebia Therapeutics^®, Auryxia^® and Vafseo^® is a registered trademark of Akebia Therapeutics, Inc. and its affiliates.

Akebia Therapeutics Contact 
Mercedes Carrasco 
mcarrasco@akebia.com   

FAQ

What did Akebia (AKBA) announce on January 6, 2026 regarding praliciguat?

Akebia announced the first patient dosed in a Phase 2 trial of praliciguat for biopsy-confirmed FSGS on Jan 6, 2026.

How many patients will Akebia's AKBA Phase 2 praliciguat study enroll and how long is treatment?

Approximately 60 patients will be randomized 1:1 for a 24-week double-blind period followed by a 24-week open-label extension.

What is the primary endpoint of the AKBA praliciguat Phase 2 FSGS trial?

The primary endpoint is change from baseline in urine protein-to-creatinine ratio (UPCR) measured at Week 24.

What defines the secondary endpoint of partial remission in the AKBA FSGS study?

Partial remission is defined as a 40% UPCR reduction and UPCR <1.5 g/g at Week 24.

Are there prior safety data for praliciguat cited by Akebia (AKBA)?

Yes; the company reports no significant safety issues observed in Phase 1 studies and Phase 2 studies in heart failure and diabetic kidney disease.