Crinetics Initiates Phase 2/3 Pediatric Trial Evaluating Atumelnant in Congenital Adrenal Hyperplasia (CAH)

Rhea-AI Summary

Crinetics (Nasdaq: CRNX) announced dosing of the first patient in the BALANCE-CAH Phase 2/3 trial of atumelnant, a once-daily oral ACTH receptor antagonist for children and adolescents with classic congenital adrenal hyperplasia (CAH).

The potential registrational study will assess safety, efficacy, and pharmacokinetics across three parts: Phase 2 dose-ranging (Part A), Phase 3 double-blind randomized placebo-controlled confirmatory (Part B), and an open-label extension (Part C). The drug holds U.S. orphan drug designation for classic CAH.

Positive

• First patient dosed in pediatric BALANCE-CAH Phase 2/3 trial
• Potential registrational study evaluating safety, efficacy, pharmacokinetics
• First/only small-molecule ACTH receptor antagonist in late-stage development
• Received U.S. orphan drug designation for classic CAH

Negative

• None.

Key Figures

Trial phase: Phase 2/3 Dosing frequency: once-daily Study parts: 3 parts (A, B, C)

3 metrics

Trial phase Phase 2/3 BALANCE-CAH pediatric CAH study design

Dosing frequency once-daily Oral atumelnant regimen in BALANCE-CAH

Study parts 3 parts (A, B, C) Dose-ranging, confirmatory, and open-label extension

Market Reality Check

Price: $53.18 Vol: Volume 978,918 is below t...

low vol

$53.18 Last Close

Volume Volume 978,918 is below the 1,490,263 20-day average, suggesting muted pre-news positioning. low

Technical Price $55.41 is trading above the $37.04 200-day moving average, reflecting a pre-existing uptrend.

Peers on Argus

CRNX slipped 0.5% while close biotech peers showed mixed moves (e.g., KYMR +3.89...

CRNX slipped 0.5% while close biotech peers showed mixed moves (e.g., KYMR +3.89%, APLS -3.19%), indicating stock-specific dynamics rather than a coordinated sector move.

Historical Context

5 past events · Latest: Jan 12 (Neutral)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 12	Inducement equity grants	Neutral	-1.9%	New-hire stock options and RSUs under 2021 Inducement Plan.
Jan 06	Equity offering priced	Negative	-4.6%	Underwritten common stock offering priced at $45.95 per share.
Jan 05	Equity offering proposed	Negative	+3.1%	Announcement of proposed $350M common stock offering.
Jan 05	Positive Phase 2 data	Positive	+3.1%	Atumelnant Phase 2 trial showed A4 reduction and steroid lowering.
Jan 04	Clinical update call	Neutral	+3.1%	Planned webcast for PALSONIFY update and atumelnant topline data.

Pattern Detected

Clinical-trial updates have generally aligned with positive price reactions, while equity offerings show mixed reactions, with one pricing selloff and an earlier proposed offering uptick.

Recent Company History

Over the last few months, Crinetics combined pipeline progress with balance sheet strengthening. In November 2025, it advanced multiple clinical programs, including paltusotine’s Phase 3 CAREFNDR trial and atumelnant in CAH. An August 2025 Orphan Drug Designation for atumelnant in CAH followed prior positive Phase 2 data. In early January 2026, the company reported strong Phase 2 atumelnant results and then executed a $350 million equity raise. Today’s initiation of a Phase 2/3 pediatric CAH trial fits the pattern of ongoing clinical expansion for atumelnant.

Market Pulse Summary

This announcement extends atumelnant’s CAH program into a pediatric Phase 2/3 BALANCE-CAH trial, tar...

Analysis

This announcement extends atumelnant’s CAH program into a pediatric Phase 2/3 BALANCE-CAH trial, targeting a population with significant unmet need. Prior CAH data and Orphan Drug Designation frame this as a continuation of an established strategy rather than a new direction. Key elements to watch include enrollment progress across Parts A–C, safety and pharmacokinetic readouts in children, and how these data may complement earlier adult CAH results and the broader endocrine-focused pipeline.

Key Terms

adrenocorticotropic hormone, acth receptor antagonist, congenital adrenal hyperplasia, orphan drug designation, +4 more

8 terms

adrenocorticotropic hormone medical

"atumelnant, a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist"

Adrenocorticotropic hormone (ACTH) is a signaling protein made by the pituitary gland that tells the adrenal glands to release cortisol and other stress-related hormones; think of it as a thermostat that triggers the body’s emergency response. For investors, ACTH matters because it is a target for diagnostic tests, drug development, and therapeutic treatments for adrenal and stress-related disorders, influencing regulatory approvals, clinical trial outcomes, and market demand for related medicines and assays.

acth receptor antagonist medical

"first in class study of an investigational ACTH receptor antagonist"

An ACTH receptor antagonist is a drug that blocks the body’s receptor for adrenocorticotropic hormone (ACTH), which stops the hormone from telling the adrenal glands to make stress hormones like cortisol. For investors this matters because such drugs can treat conditions driven by excess cortisol (for example certain endocrine disorders), so successful development or approval can create new revenue streams; think of it as cutting the signal to an overactive factory to slow production.

congenital adrenal hyperplasia medical

"for the proposed treatment of classic congenital adrenal hyperplasia (CAH)"

Congenital adrenal hyperplasia is a group of inherited disorders in which the adrenal glands lack an enzyme needed to make certain hormones, causing a chronic imbalance of cortisol, aldosterone and/or sex hormones. Think of it as a factory assembly line missing a key part, so the body overproduces some products and underproduces others, requiring lifelong monitoring or hormone treatment. For investors, it matters because diagnosis, ongoing therapy, newborn screening and potential new drugs or gene therapies can drive medical spending, regulatory approvals and market opportunity in endocrinology and rare disease care.

orphan drug designation regulatory

"Crinetics recently received Orphan Drug Designation from the U.S. Food & Drug Administration"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

phase 2 medical

"Part A is a Phase 2, open-label, semi-sequential dose-ranging portion"

Phase 2 is the mid-stage clinical trial where a new drug or treatment is tested in a larger group of patients to see if it works and to keep checking safety after initial human testing. Think of it as a field test that proves whether a product actually delivers its promised benefit. Investors watch Phase 2 closely because its results strongly influence a medicine’s chances of reaching the market, the size of its potential sales, and the company’s valuation.

phase 3 medical

"Part B is the Phase 3, double-blind, randomized, placebo controlled confirmatory portion"

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

double-blind medical

"Phase 3, double-blind, randomized, placebo controlled confirmatory portion"

A double-blind process means that neither the people conducting an activity nor the people involved know certain key details, such as who is receiving a treatment or a placebo. This approach helps prevent bias from influencing the results, making the outcome more trustworthy. For investors, it ensures that decisions or judgments are based on unbiased information rather than preconceived opinions or expectations.

open-label extension medical

"Part C is an open-label extension portion of the study"

An open-label extension is a continuation of a clinical trial where all participants and researchers know which treatment is being given, often after an initial blinded phase. It allows further study of a drug's long-term safety and effectiveness. For investors, it can indicate ongoing interest and confidence in a product's potential, influencing perceptions of its future value.

AI-generated analysis. Not financial advice.

First in class study of an investigational ACTH receptor antagonist aims to address significant unmet need in children and adolescents with classic CAH

SAN DIEGO, Jan. 22, 2026 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the first patient has been dosed in the BALANCE-CAH Phase 2/3 trial evaluating investigational candidate atumelnant, a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist candidate for the proposed treatment of classic congenital adrenal hyperplasia (CAH) in children and adolescents.

“BALANCE-CAH is designed to evaluate atumelnant for the treatment of CAH in pediatric age patients – for whom the lifelong burden of this disease begins early,” said Dr. Alan Krasner, M.D., Chief Endocrinologist, Crinetics. “For children living with CAH, correcting elevated androgen levels caused by the condition, while preserving the ability to use low glucocorticoid doses for replacement purposes only, is critical in supporting normal growth, development, and metabolic health. By studying atumelnant in this population, we aim to generate insights that could help children live less encumbered by the challenges of this serious endocrine disorder.”

Atumelnant is the first and only small molecule ACTH receptor antagonist in late-stage clinical development and is designed to block the pathway in the adrenal gland that leads to the production of excess androgens associated with classic CAH. The BALANCE-CAH Phase 2/3 study is a potential registrational study of atumelnant in pediatric patients with CAH and will evaluate safety, efficacy, and pharmacokinetics in children and adolescents, a population with significant unmet medical need.

The study will be conducted in three phases. Part A is a Phase 2, open-label, semi-sequential dose-ranging portion of the study. Part B is the Phase 3, double-blind, randomized, placebo controlled confirmatory portion of the study. Part C is an open-label extension portion of the study.

Crinetics recently received Orphan Drug Designation from the U.S. Food & Drug Administration for atumelnant in the treatment of classic CAH.

For more information, visit https://clinicaltrials.gov/study/NCT07159841

About Atumelnant
Investigational atumelnant is the first in class and only once-daily, oral adrenocorticotropic hormone (ACTH) receptor antagonist that acts selectively at the melanocortin type 2 receptor (MC2R) on the adrenal gland in late-stage clinical development. Diseases associated with excess ACTH can have a significant impact on physical and mental health. Novel atumelnant has exhibited strong binding affinity for MC2R in preclinical models and has demonstrated suppression of adrenally derived glucocorticoids and androgens that are under the control of ACTH. Data from a 12-week Phase 2 study consistently demonstrated compelling treatment benefits of atumelnant, evidenced by the rapid, substantial and sustained statistically significant reductions in key CAH disease related biomarkers, including A4 and 17-hydroxyprogesterone, in a diverse population. Currently in Phase 3 clinical development, atumelnant holds the potential to offer transformational care for individuals living with congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome. This breakthrough could revolutionize the management of these conditions, providing hope for unprecedented improvements in quality of life.
About Crinetics Pharmaceuticals
Crinetics Pharmaceuticals is a global pharmaceutical company committed to transforming the treatment of endocrine diseases and endocrine-related tumors through science rooted in patient needs. Crinetics is focused on discovering, developing, and commercializing novel therapies, with a core expertise in targeting G-protein coupled receptors (GPCRs) with small molecules that have specifically tailored pharmacology and properties.

Crinetics’ lead product, PALSONIFY™ (paltusotine), is the first once-daily, oral treatment approved by the U.S. FDA for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. Paltusotine is also in clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics’ deep pipeline of 10+ disclosed programs includes late-stage investigational candidate atumelnant, which is currently in development for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome, and CRN09682, a nonpeptide drug conjugate candidate that is being developed to treat SST2 expressing neuroendocrine tumors and other SST2 expressing solid tumors. Additional discovery programs address a variety of endocrine conditions such as neuroendocrine tumors, Graves’ disease (including Graves’ hyperthyroidism and Graves’ orbitopathy, or thyroid eye disease), polycystic kidney disease, hyperparathyroidism, diabetes, obesity, and GPCR-targeted oncology indications.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the plans and timelines for the Phase 2/3 program for atumelnant for CAH; the plans and timelines for the clinical development of our drug candidates, including the therapeutic potential and clinical benefits or safety profile thereof; the expected timing of additional research pipeline updates or the expected timing of the advancement of those programs. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential,” “upcoming” or “continue” or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including, without limitation, data that we report may change following completion or a more comprehensive review of the data related to the clinical studies; we may not be able to obtain, maintain and enforce our patents and other intellectual property rights, and it may be prohibitively difficult or costly to protect such rights; geopolitical events may disrupt Crinetics’ business and that of the third parties on which it depends, including delaying or otherwise disrupting its clinical studies and preclinical studies, manufacturing and supply chain, or impairing employee productivity; unexpected adverse side effects or inadequate efficacy of the Company’s product candidates that may limit their development, regulatory approval and/or commercialization; the Company’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; the success of Crinetics’ clinical studies and nonclinical studies; regulatory developments or political changes, including the policies related to pricing and pharmaceutical drug reimbursement in the United States and foreign countries; clinical studies and preclinical studies may not proceed at the time or in the manner expected, or at all; the timing and outcome of research, development and regulatory review is uncertain, and Crinetics’ drug candidates may not advance in development; Crinetics may use its capital resources sooner than expected or our cash burn rate may accelerate; any future impacts to our business resulting from geopolitical developments outside our control; and the other risks and uncertainties described in the Company’s periodic filings with the Securities and Exchange Commission (SEC). The events and circumstances reflected in the company’s forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Additional information on risks facing Crinetics can be found under the heading “Risk Factors” in Crinetics’ periodic filings with the SEC, including its annual report on Form 10-K for the year ended December 31, 2024. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by applicable law, Crinetics does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Media:
Natalie Badillo
Head of Corporate Communications
nbadillo@crinetics.com
(858) 345-6075

Investors:
Gayathri Diwakar
Head of Investor Relations
gdiwakar@crinetics.com
(858) 345-6340

FAQ

What did Crinetics (CRNX) announce on January 22, 2026 about atumelnant?

Crinetics announced the first patient was dosed in the BALANCE-CAH Phase 2/3 pediatric trial of atumelnant.

What is the design of the BALANCE-CAH trial for CRNX atumelnant?

The trial has three parts: Phase 2 open-label dose-ranging (Part A), Phase 3 double-blind randomized placebo-controlled confirmatory (Part B), and an open-label extension (Part C).

What will the CRNX BALANCE-CAH study evaluate in pediatric patients?

The study will evaluate safety, efficacy, and pharmacokinetics of atumelnant in children and adolescents with classic CAH.

Does atumelnant have any special regulatory status for CAH?

Yes; atumelnant received U.S. orphan drug designation for treatment of classic CAH.

Where can investors find more information about the CRNX BALANCE-CAH trial?

Study details are available on ClinicalTrials.gov under identifier NCT07159841.