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SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )--Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases, today announced the online publication of data in The Lancet Neurology from its Phase 3 ATTeST ( A taxia- T elangiectasia T rial with the E ryDex S ys T em; #IEDAT-02-2015/ NCT02770807 ) clinical trial evaluating the safety and efficacy of its lead asset, EryDex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes), for the treatment of A-T. “The results of this study demonstrate the encouraging efficacy and safety profile of our lead asset, EryDex, for the treatment of patients with A-T, a rare pediatric disease with vast unmet need and no approved treatments,” said Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer. “These findings provide us with additional confidence in our pivotal Phase 3 study of EryDex now underway with a primary analysis population comprised of patients with A-T experiencing the most rapid clinical decline – children ages six to nine – in addition to including participants who are ten years of age or older.” The Lancet Neurology Publication Highlights The Lancet Neurology publication entitled Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia-telangiectasia (ATTeST): a multicenter, randomized, double-blind, placebo-controlled phase 3 trial describes safety and efficacy results from the Phase 3 ATTeST clinical trial. Highlights include: ATTeST was the largest completed study in A-T, with 175 participants conducted at 22 academic institutions and medical centers on five continents in 12 different countries. ATTeST was a randomized, double-blind, placebo-controlled Phase 3 clinical trial to evaluate the safety and efficacy of two dose levels of EryDex compared to placebo on neurological symptoms of patients with A-T. In the ATTeST study, following six-months of EryDex treatment, none of the serious safety concerns typically associated with chronic corticosteroid administration were observed. There were no reports of hyperglycemia, hypertension, hirsutism, or cushingoid appearance in any of the treatment groups. The primary efficacy endpoint was measured by the change in modified International Cooperative Ataxia Rating Scale (mICARS), from baseline to month six, comparing results from the active and placebo control groups. mICARS included assessment of the participant’s posture and gait, kinetic functions, and speech. The higher score denotes higher burden of neurological symptoms. In the ATTeST study, patients receiving high-dose EryDex had reduced neurological symptoms with a -1.40 change in mICARS observed in the treated group compared to placebo. This result was not statistically significant (a nominal p-value =0.077). However, patients in the per-protocol population who received high-dose EryDex demonstrated statistical significance with a -2.2 change in mICARS and a nominal p-value of 0.019. In a pre-specified subgroup analysis by age, a statistically significant reduction in neurological symptoms observed in patients ages six to nine years old receiving high-dose EryDex, notably, a -2.8 and -4.4 change in mICARS was observed in the modified intent-to-treat (mITT) and per-protocol populations compared to placebo, with nominal p-values of 0.019 and 0.002, respectively. Inability to confirm the effect of treatment in the overall population might be related to delays and omissions in treatments, as indicated by positive efficacy results in the per-protocol population, which excluded participants with delayed and missed treatments, and potential differences in treatment effect in patients ages six to nine, compared with patients 10 years or older. Contributors to delayed treatments included difficulties with travel to treatment centers and disruptions caused by the COVID-19 pandemic. Pandemic control guidelines, including travel restrictions, which varied among countries and institutions, resulted in missed and delayed treatments. In some instances, treatment was delayed because investigators delayed visits to hospital to protect immunocompromised participants in the peak of the pandemic. Because of delays in treatment and missed doses, 35% of the mITT population did not receive treatment as planned. “The publication of the ATTeST trial results in The Lancet Neurology highlights promising clinical data that demonstrate progress being made toward a potential treatment option for patients with A-T, a rare pediatric disorder characterized by progressive neurological decline, impaired motor control, speech difficulties, and a life expectancy typically in the twenties or thirties,” said Dr. William Whitehouse, Honorary Clinical Associate Professor of the School of Medicine at the University of Nottingham, and Consultant Paediatric Neurologist at Nottingham Children’s Hospital, Nottingham University Hospitals NHS Trust. “Natural history studies have shown that children between age six and 10 with classic A-T experience rapid clinical decline, after neurological symptoms worsen and patients with A-T frequently become wheelchair-bound by adolescence. The differential response in mICARS score by age seen in subgroup analyses from the ATTeST study emphasizes the need to stratify future A-T clinical trials by age.” Quince is currently enrolling the pivotal Phase 3 NEAT study ( #IEDAT-04-2022 / NCT06193200 ), which is an international, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the neurological effects of EryDex in patients with A-T. Quince plans to enroll approximately 86 patients with A-T ages six to nine years old (primary analysis population) and approximately 20 patients with A-T ages 10 years or older. Seven patients with A-T have enrolled in the Phase 3 NEAT study to date. The Phase 3 NEAT trial is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA), and the company expects to report topline results in the fourth quarter of 2025 with a potential New Drug Application (NDA) submission to the FDA and a Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) in 2026, assuming positive study results. Additionally, Quince was granted Fast Track designation by the FDA for the company’s EryDex System for the treatment of patients with A-T based on the potential for EryDex to address a high unmet medical need in A-T. About Ataxia-Telangiectasia A-T is an inherited autosomal recessive neurodegenerative and immunodeficiency disorder caused by mutations in the ATM gene, which is responsible for cell homeostatic and cell division functions including but not limited to double-stranded DNA repair. Typically, A-T is first diagnosed before the age of five as children begin to develop an altered gait and fall with greater frequency. Neurological symptoms worsen and patients with A-T frequently become wheelchair-bound by adolescence. Teenage years for patients with A-T are typically marked by repeated infections, pulmonary impairment, and malignancies. The median lifespan is approximately 25 to 30 years old with mortality due to infections and malignancy. Based on IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics information, there are approximately 4,600 diagnosed patients with A-T in the U.S. Quince estimates that there are approximately 5,000 patients with A-T in the U.K. and EU4 countries. There are currently no approved therapeutic treatments in any global market for A-T. About EryDex for A-T EryDex is comprised of dexamethasone sodium phosphate (DSP) encapsulated in a patient’s own red blood cells (autologous erythrocytes). DSP is a corticosteroid well known for its anti-inflammatory properties as well as its dose-limiting toxicity due to adrenal suppression. EryDex is designed to provide the efficacy of corticosteroids and to reduce or eliminate the significant adverse effects that accompany chronic use of corticosteroid treatment. EryDex leverages Quince’s proprietary Autologous Intracellular Drug Encapsulation, or AIDE, technology platform, which is a novel drug/device combination that uses an automated process designed to encapsulate a drug into the patient’s own red blood cells. Red blood cells have several characteristics that make them a potentially effective vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Quince’s AIDE technology is designed to harness these benefits to allow for the chronic administration of drugs that have limitations due to toxicity, poor biodistribution, suboptimal pharmacokinetics, or immune response. About Quince Therapeutics Quince Therapeutics (Nasdaq: QNCX) is a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases. For more information on the company and its latest news, visit www.quincetx.com and follow Quince Therapeutics on social media platforms LinkedIn , Facebook , X , and YouTube . Forward-looking Statements Statements in this news release contain “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the “safe harbor” created by those sections. All statements, other than statements of historical facts, may be forward-looking statements. Forward-looking statements contained in this news release may be identified by the use of words such as “believe,” “may,” “should,” “expect,” “anticipate,” “plan,” “believe,” “estimated,” “potential,” “intend,” “will,” “can,” “seek,” or other similar words. Examples of forward-looking statements include, among others, statements relating to current and future clinical development of EryDex, including for the potential treatment of Ataxia-Telangiectasia (A-T) and other potential indications, related development and commercial-stage inflection point for EryDex, the company’s proprietary Autologous Intracellular Drug Encapsulation (AIDE) technology for treatment of other rare diseases; the strategic development path for EryDex; planned regulatory agency submissions and clinical trials and timeline, prospects, and milestone expectations; the timing and success of the clinical trials and related data, including plans and the ability to initiate, fund, enroll, conduct, and/or complete current and additional studies; research and development costs; the company’s future development plans and related timing; cash position and projected cash runway; the company’s focus, objectives, plans, and strategies; and the potential benefits of EryDex, AIDE technology and the company’s market opportunity. Forward-looking statements are based on Quince’s current expectations and are subject to inherent uncertainties, risks, and assumptions that are difficult to predict and could cause actual results to differ materially from what the company expects. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, but are not limited to, the risks and uncertainties described in the section titled “Risk Factors” in the company’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 13, 2024, and other reports as filed with the SEC. Forward-looking statements contained in this news release are made as of this date, and Quince undertakes no duty to update such information except as required under applicable law.

"Myo inositol PCOS", a topic that has attracted much attention in the medical field in recent years, reveals the subtle relationship between endocrine disorders and women's health. Polycystic ovary syndrome (PCOS) is a common gynecological disorder that affects a wide range of women, from reduced fertility to a range of health problems. The discovery of "myo inositol PCOS" offers new hope for improving the condition of PCOS patients. The pathogenesis of PCOS is complex, in which the imbalance of myo inositol PCOS plays a key role. Reduced levels of inositol, a substance that occurs naturally in the body, can lead to insulin resistance, which in turn triggers or exacerbates the symptoms of PCOS. A large number of studies have confirmed that through the supplement of "myo inositol", the normal insulin response in the body can be restored, thereby improving the symptoms of irregular menstruation, acne, hirsutism and other symptoms, and improving the quality of life of patients. However, the supplementation of "myo inositol PCOS" is only the first step in treating PCOS. In order to achieve the overall improvement of women's health, we also need to pay attention to the adjustment of diet structure and lifestyle changes. It is recommended that patients increase whole grains, vegetables, fruits and other fiber-rich foods to enhance the body's ability to process sugar, while reducing the intake of sugar and saturated fat to prevent the type 2 diabetes and cardiovascular disease complicated by PCOS. In addition, maintaining adequate sleep, a regular schedule, and proper exercise can all help improve the endocrine environment and further relieve the symptoms of PCOS. It is important to note that the physique and symptoms of each PCOS patient are different, so when implementing the treatment plan, a personalized health management plan should be developed according to the specific circumstances of the individual. This may include regular medical checkups, professional nutritional counseling, and even psychological counseling to ensure that the patient is fully supported, both physically and psychologically. The discovery of "myo inositol PCOS" opens new avenues for the treatment of PCOS, but we should also recognize that comprehensive health management is the key to defeating PCOS. Only through a comprehensive and individualized treatment strategy can we help women out of the shadow of PCOS, regain their health and confidence, and embrace a better future.

PDUFA Target Action Dates in Late December 2024 Highly Selective CRF1 Antagonist is the Potential First New Treatment for CAH in 70 Years SAN DIEGO, July 1, 2024 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the U.S. Food and Drug Administration (FDA) has accepted its two New Drug Applications (NDA) with Priority Review designations for crinecerfont in the treatment of children, adolescents and adults with classic congenital adrenal hyperplasia (CAH). If approved, crinecerfont would be the first new treatment option for CAH in 70 years and a first-in-class therapy, with a novel approach for the treatment of this rare and serious endocrine disorder. The submitted crinecerfont NDAs included: the primary presentation of efficacy and safety of crinecerfont for the treatment of classic CAH as (1) a capsule formulation (NDA# 218808); and (2) as an oral solution formulation (NDA# 218820). The agency set Prescription Drug User Fee (PDUFA) target action dates of December 29 and December 30, 2024, respectively. The FDA stated it is not currently planning to hold an advisory committee meeting to discuss these applications. Priority Review designation by the FDA accelerates the review timeline by four months – and means the agency recognizes CAH is a serious condition with high unmet medical need and crinecerfont is a treatment that provides significant benefit over current therapy. Should crinecerfont receive FDA approval, it will enable Neurocrine Biosciences to activate its Rare Pediatric Disease Designation Priority Review Voucher – a designation granted in September 2020. "Receipt of a Priority Review reflects the FDA's agreement that CAH is a serious condition and there is an urgent need for patients to have access to new treatments," said Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. "Crinecerfont's compelling efficacy results and excellent safety profile support our filing, and we look forward to working with the FDA as we head toward the PDUFA dates at the end of 2024." Crinecerfont previously was granted Orphan Drug designation in March 2019 and Breakthrough Therapy designation in December 2023. Orphan Drug designation means the company will be exempt from paying PDUFA user fees, receive tax credits for qualified clinical trials, and has the potential of seven years of market exclusivity should crinecerfont be approved. Breakthrough Therapy designation is a process developed by the FDA to expedite development and review of drugs that are intended to treat a serious condition and where clinical evidence indicates that the potential drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). Breakthrough Therapy designation was granted based on the strong results and excellent safety profile seen from the CAHtalyst™ Phase 3 Pediatric and Adult study data announced in fall 2023 and published in June. CAHtalyst Phase 3 data results in pediatric and adult patients with CAH due to 21-OHD were published in online issues of The New England Journal of Medicine on June 2 and June 1, respectively, and presented at ENDO 2024. About Congenital Adrenal Hyperplasia Congenital adrenal hyperplasia (CAH) is a rare genetic condition that results in an enzyme deficiency that alters the production of adrenal hormones which are essential for life. Approximately 95% of CAH cases are caused by a mutation that leads to deficiency of the enzyme 21-hydroxylase (21-OHD). Severe deficiency of this enzyme leads to an inability of the adrenal glands to produce cortisol and, in approximately 75% of cases, aldosterone. If left untreated, CAH can result in salt wasting, dehydration, and even death. Glucocorticoids (GCs) are currently used not only to correct the endogenous cortisol deficiency, but doses used are higher than cortisol replacement needed (supraphysiologic) to lower the levels of adrenocorticotropic hormone (ACTH) and adrenal androgens. However, glucocorticoid treatment at supraphysiologic doses has been associated with serious and significant complications of steroid excess, including metabolic issues such as weight gain and diabetes, cardiovascular disease, and osteoporosis. Additionally, long-term treatment with supraphysiologic GC doses may have psychological and cognitive impact, such as changes in mood and memory. Adrenal androgen excess has been associated with abnormal bone growth and development in pediatric patients, female health problems such as acne, excess hair growth and menstrual irregularities, testicular rest tumors in males, and fertility issues in both sexes. To learn more about CAH, click here. About Crinecerfont and the CAHtalyst™ Studies Crinecerfont is an investigational, oral, selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist being developed to reduce and control excess adrenocorticotropic hormone (ACTH) and adrenal androgens through a glucocorticoid-independent mechanism for the treatment of congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. Antagonism of CRF1 receptors in the pituitary has been shown to decrease ACTH levels, which in turn decreases the production of adrenal androgens and potentially the symptoms associated with CAH. Crinecerfont study data demonstrate that lowering adrenal androgen levels enables lower, more physiologic dosing of glucocorticoids to manage androgen excess and thus could potentially reduce the complications associated with exposure to greater than normal glucocorticoid doses in patients with CAH. The CAHtalyst™ Pediatric and Adult Phase 3 global registrational studies are designed to evaluate the safety, efficacy, and tolerability of crinecerfont in children and adolescents, and adults respectively, with congenital adrenal hyperplasia due to 21-hydroxylase deficiency. The primary portions of the CAHtalyst Phase 3 studies have completed and enrollment is closed, while the open-label treatment portions of both studies are ongoing. Data from the CAHtalyst Pediatric and CAHtalyst Adult Phase 3 studies supported two New Drug Application submissions to the U.S. Food and Drug Administration in April 2024. To learn more about crinecerfont and the CAHtalyst studies, click here. About Neurocrine Biosciences, Inc. Neurocrine Biosciences is a leading neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs, but few options. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, endometriosis* and uterine fibroids*, as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, X (formerly Twitter), and Facebook. (*in collaboration with AbbVie) The NEUROCRINE BIOSCIENCES Logo Lockup and YOU DESERVE BRAVE SCIENCE are registered trademarks of Neurocrine Biosciences, Inc. CAHtalyst is a trademark of Neurocrine Biosciences, Inc. Forward-Looking Statements In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements regarding the potential benefits to be derived from crinecerfont. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements include: the crinecerfont NDAs may not obtain regulatory approval, such approval may be delayed, or may not receive the benefits associated with priority review; additional regulatory submissions may not occur or be submitted in a timely manner; the FDA may make adverse decisions regarding crinecerfont; crinecerfont may not be found to be safe and/or effective or may not prove to be beneficial to patients; development activities for crinecerfont may not be completed on time or at all; clinical development activities may be delayed for regulatory or other reasons, may not be successful or replicate previous and/or interim clinical trial results, or may not be predictive of real-world results or of results in subsequent clinical trials; competitive products and technological changes that may limit demand for our products; uncertainties relating to patent protection and intellectual property rights of third parties; our dependence on third parties for development and manufacturing activities related to crinecerfont, and our ability to manage these third parties; our future financial and operating performance; risks and uncertainties associated with the commercialization of our products; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended March 31, 2024. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof. SOURCE Neurocrine Biosciences, Inc.