Tildacerfont

Neurocrine Biosciences has announced that the US Food and Drug Administration (FDA) has accepted its two New Drug Applications (NDA) for crinecerfont, granting them Priority Review designations. The applications cover the treatment of children, adolescents, and adults with classic congenital adrenal hyperplasia (CAH). If approved, crinecerfont would represent the first new treatment for CAH in 70 years and the first-in-class therapy offering a novel mechanism of action to address this rare and serious endocrine disorder. LAS VEGAS, Sept. 30, 2024 /PRNewswire/ -- CAH refers to a rare set of inherited autosomal recessive disorders that impair hormone production in the adrenal glands, which sit on top of the kidneys. Normally, these glands produce three types of hormones: corticosteroids, mineralocorticoids, and androgens. CAH is mostly caused by mutations in the 21-hydroxylase gene, which accounts for more than 90% of cases. In rare instances, mutations in the 11-hydroxylase gene can also lead to CAH. In the 7MM, the US accounted for the highest prevalent cases of congenital adrenal hyperplasia in 2023, with around 50% of the total prevalent cases across the 7MM; these numbers are expected to increase during the forecast period (2024–2034). DelveInsight's analysis reveals that a higher prevalence of diagnosed congenital adrenal hyperplasia is observed in the 18-year-old and above age group across the 7MM. Among mutation-based congenital adrenal hyperplasia cases, 21 OHD (CYP21A2 gene mutation) represents a higher prevalence compared to 11-Beta hydroxylase (CYP11B1 gene mutation), and others. The approach to managing CAH differs significantly based on its type and severity. While CAH cannot be completely cured, it can be managed effectively. For classical CAH, treatment begins shortly after birth and continues throughout the individual's life. Those with classical CAH should work with a team of healthcare professionals, including experts in pediatric endocrinology, uro-gynecologic surgery, psychology, and genetics. With appropriate care, individuals with classical CAH can lead normal and satisfying lives. In contrast, treatment for non-classical CAH may not be necessary, depending on the severity of symptoms. The management of classic CAH focuses on three main objectives: correcting adrenal insufficiency, lowering adrenal androgens to appropriate levels, and preventing iatrogenic hypercortisolism. Hydrocortisone is the primary glucocorticoid used, administered two to three times daily with higher doses in the morning and lower doses in the evening to align with the body's natural circadian rhythm. For NC-CAH, cortisol replacement is generally unnecessary, except in specific situations such as adult women with hyperandrogenism and fertility problems, or pediatric patients experiencing rapidly advancing precocious puberty. Currently, the only medication approved in Europe for CAH treatment is EFMODY (Hydrocortisone Modified-release Hard Capsules) by Neurocrine Biosciences. Learn more about the FDA-approved congenital adrenal hyperplasia drugs @ Drugs for Congenital Adrenal Hyperplasia Treatment EFMODY replicates the natural circadian rhythm of cortisol secretion. It compensates for low cortisol levels in patients with congenital adrenal hyperplasia, aiding in the management of the condition's symptoms. Approved for use in adolescents aged 12 and older, as well as adults, EFMODY uses hydrocortisone—a glucocorticoid that affects various tissues through intracellular steroid receptors. Containing synthetic cortisol, EFMODY gradually releases it in the intestines to mimic the body's natural cortisol release, helping to restore hormone balance and alleviate condition-related issues. In May 2021, the EMA approved EFMODY for treating congenital adrenal hyperplasia in patients aged 12 and above. Diurnal launched the drug in Germany and Austria in September 2021, and later in the United Kingdom. In November 2022, Neurocrine Biosciences acquired Diurnal for approximately USD 55 million. To know more about congenital adrenal hyperplasia treatment options, visit @ New Treatment for Congenital Adrenal Hyperplasia The congenital adrenal hyperplasia pipeline is strong with many companies working in the domain. Various potential therapies that are projected to enter during the forecast period include Neurocrine Biosciences' Crinecerfont, Spruce Biosciences' Tildacerfont (SPR001), Crinetics Pharmaceuticals' Atumelnant (CRN04894), BridgeBio's BBP-631, Lundbeck's Lu AG13909, and others. Discover which therapies are expected to grab major congenital adrenal hyperplasia market share @ Congenital Adrenal Hyperplasia Market Report Crinecerfont is an investigational oral medication that acts as a selective antagonist for the corticotropin-releasing factor Type 1 receptor (CRF1). It's being developed to manage and regulate high levels of Adrenocorticotropic Hormone (ACTH) and adrenal androgens without relying on glucocorticoids, specifically for treating congenital adrenal hyperplasia caused by 21-hydroxylase deficiency. By blocking the CRF1 receptor, Crinecerfont reduces the overproduction of ACTH, which in turn decreases the excess production of androgens seen in CAH patients. This mechanism aims to normalize hormone levels and address the root cause of the condition. In July 2024, Neurocrine Biosciences announced that the FDA has accepted its two New Drug Applications (NDAs) for crinecerfont, both with Priority Review designations. These applications are for treating classic congenital adrenal hyperplasia in children, adolescents, and adults. If approved, crinecerfont would become the first new treatment for CAH in 70 years and a pioneering therapy with a novel approach for this rare endocrine disorder. The NDAs for crinecerfont include (1) a capsule formulation (NDA# 218808) and (2) an oral solution formulation (NDA# 218820), both addressing the efficacy and safety of crinecerfont for classic CAH. The FDA has set Prescription Drug User Fee Act (PDUFA) target action dates for these applications as December 29 and December 30, 2024, respectively. The FDA has indicated it does not plan to hold an advisory committee meeting regarding these NDAs. Crinecerfont received an Orphan Drug designation (ODD) in March 2019 and a Breakthrough Therapy designation (BTD) in December 2023. The results from the CAHtalyst Phase III trial, involving both pediatric and adult patients with CAH caused by 21-OHD, were published online in The New England Journal of Medicine on June 2 and June 1, respectively, and were also presented at ENDO 2024. BBP-631 is a gene therapy in development using adeno-associated virus (AAV) to target the root cause of congenital adrenal hyperplasia due to 21-hydroxylase deficiency. This therapy aims to deliver a functional copy of the 21-hydroxylase gene directly to the adrenal glands, where hormones are produced. Animal studies have demonstrated its effectiveness in providing consistent and long-term delivery to the adrenal glands. If successful, BBP-631 could help restore natural hormone and steroid levels in people with CAH, allowing them to produce cortisol and aldosterone—something no current treatments or clinical trials can achieve. BridgeBio envisions this gene therapy as a potential solution to correct the hormonal imbalance caused by CAH. Tildacerfont is a new-generation antagonist targeting the Corticotropin-releasing factor (CRF1) receptor. It aims to significantly enhance disease management and decrease reliance on steroids for patients with classic congenital adrenal hyperplasia and other conditions associated with high ACTH levels. By binding to CRF1 receptors, tildacerfont reduces the overproduction of ACTH and, consequently, lowers the production of adrenal androgens like androstenedione in patients with CAH and Polycystic Ovary Syndrome (PCOS). In August 2024, Spruce Biosciences announced that the top-line results from the CAHmelia-204 study, which evaluates tildacerfont in adult CAH patients, and the CAHptain-205 study, which assesses the drug in both adult and pediatric CAH patients, are expected to be released in the fourth quarter of 2024. Discover more about drugs for congenital adrenal hyperplasia in development @ Congenital Adrenal Hyperplasia Clinical Trials The anticipated launch of these emerging therapies for congenital adrenal hyperplasia are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the congenital adrenal hyperplasia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for congenital adrenal hyperplasia is expected to grow from USD 20 billion in 2023 with a tremendous CAGR of ~40% by 2034. This growth can be attributed to the introduction of upcoming therapies and the rising prevalence of the disease. DelveInsight's latest published market report titled as Congenital Adrenal Hyperplasia Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the congenital adrenal hyperplasia country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The congenital adrenal hyperplasia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of Congenital Adrenal Hyperplasia Gender-specific Cases of Congenital Adrenal Hyperplasia Age-specific Cases of Congenital Adrenal Hyperplasia Mutation-based Cases of Congenital Adrenal Hyperplasia Type-specific Cases of Congenital Adrenal Hyperplasia The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM congenital adrenal hyperplasia market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis up to 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this congenital adrenal hyperplasia market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the congenital adrenal hyperplasia market. Also, stay abreast of the mitigating factors to improve your market position in the congenital adrenal hyperplasia therapeutic space. Related Reports Congenital Adrenal Hyperplasia Pipeline Congenital Adrenal Hyperplasia Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key CAH companies, including Spruce Biosciences, Lundbeck, among others. Congenital Adrenal Hyperplasia Epidemiology Forecast Congenital Adrenal Hyperplasia Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and CAH epidemiology trends. Adrenal Crisis Market Adrenal Crisis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key adrenal crisis companies including Eton Pharmaceuticals, Antares Pharma , among others. Adrenal Crisis Pipeline Adrenal Crisis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key adrenal crisis companies, including Eton Pharmaceuticals, Antares Pharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )-- Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for endocrine and neurological disorders with significant unmet medical need, today reported financial results for the second quarter ended June 30, 2024 and provided corporate updates. “ We remain committed to advancing the development of tildacerfont and opening a new chapter in the management of CAH with a potentially life-changing medicine for patients and their families,” said Javier Szwarcberg, M.D., M.P.H., Chief Executive Officer of Spruce. “ In the fourth quarter of 2024, we plan to report primary efficacy and safety data through week 24 plus interim data from the open-label extension of the CAHmelia-204 study, which is assessing glucocorticoid (GC) reduction, a potentially registrational endpoint in adult CAH patients on supraphysiologic GC doses with normal or near normal levels of androstenedione (A4).” Dr. Szwarcberg added, “ At the same time, we will also report topline data from CAHptain-205, which will include week 4 efficacy and safety measures on 200mg twice-daily (BID) and 400mg BID doses of tildacerfont in adults, adolescents and children with CAH and elevated levels of A4. Following our analysis of results from the CAHmelia-203 study, which evaluated doses of tildacerfont up to 200mg once-daily (QD) in adults with CAH and highly elevated levels of A4, we believe that tildacerfont has the potential to address severe hyperandrogenemia in CAH at higher doses taken BID. If results from CAHmelia-204 and CAHptain-205 are positive, we intend to meet with the U.S. Food and Drug Administration (FDA) and comparable foreign regulatory authorities to align on the next steps for our CAH program.” Corporate Updates Announced Strategic Collaboration with HMNC to Develop Personalized Treatment for MDD: Spruce entered into a license, development and option agreement with HMNC to develop Spruce’s investigational product candidate, tildacerfont, a second-generation CRF 1 receptor antagonist, with HMNC’s companion diagnostic, the proprietary Cortibon Genetic Selection Tool (Cortibon), as a personalized medicine with potential for the treatment of MDD. Under the terms of the agreement, HMNC will fund and conduct a Phase 2 proof-of-concept study of tildacerfont in MDD patients, who will be screened using Cortibon. Spruce has an option to in-license exclusive worldwide rights to Cortibon after completion of the study, if results are positive. Poster Highlighting Final Results from Phase 2 POWER Study of Tildacerfont for the Treatment of Polycystic Ovary Syndrome (PCOS) Presented at ENDO 2024 Annual Meeting: Ricardo Azziz, M.D., M.B.A., M.P.H., Professor, Obstetrics and Gynecology at University of Alabama at Birmingham School of Medicine, presented final results from the Phase 2 POWER study, which demonstrated the ability of tildacerfont to reduce dehydroepiandrosterone sulfate levels over 12 weeks in women with PCOS. Additionally, an observed increase in serum sex hormone binding globulin demonstrated that tildacerfont may potentially lower levels of free, bioactive sex hormones such as testosterone. Tildacerfont was well-tolerated, with no safety signals observed. The majority of adverse events were mild to moderate, and no serious adverse reactions were reported. The poster presentation is available on the company’s website . Poster Highlighting Impact of Geography and Insurance on Healthcare Utilization Preferences of Individuals with CAH Presented at ENDO 2024 Annual Meeting: Prasanth Surampudi, M.D., Associate Professor, Endocrinology, Diabetes and Metabolism at U.C. Davis School of Medicine, presented findings related to the need for increased partnership between primary care physicians and endocrinologists as well as increased education among CAH patients and advocacy groups of specialty care to improve biochemical outcomes that reduce risks of morbidity and mortality in adult CAH patients. The poster presentation is available on the company’s website . Poster Highlighting Pediatric and Adult Endocrinology Practices to Improve Biochemical Outcomes in Adults with CAH Presented at ENDO 2024 Annual Meeting: Wenyu Huang, M.D., Ph.D., Associate Professor, Division of Endocrinology, Metabolism and Molecular Medicine at Northwestern University Feinberg School of Medicine, and Amir Hamrahian, M.D., Associate Professor of Medicine, Division of Endocrinology, Diabetes and Metabolism at Johns Hopkins University, presented findings exploring the risks associated with CAH diagnosis and treatment, including health impacts of hyperandrogenemia and serious adverse events with long-term use of supraphysiologic GC doses. The poster presentation is available on the company’s website . Anticipated Upcoming Milestones Topline results from the CAHmelia-204 clinical trial of tildacerfont 200mg QD in adult classic CAH patients on supraphysiologic doses of GCs with normal or near normal levels of A4 anticipated in the fourth quarter of 2024 Topline results from the CAHptain-205 clinical trial of tildacerfont 200mg BID and 400mg BID adult and pediatric cohorts anticipated in the fourth quarter of 2024 End of Phase 2 (EOP2) meeting with the U.S. FDA anticipated in the first half of 2025 Second Quarter 2024 Financial Results Cash and Cash Equivalents: Cash and cash equivalents as of June 30, 2024 were $69.7 million. Cash and cash equivalents are expected to allow the company to fund its current operating plan through the end of 2025. Collaboration Revenue: Collaboration revenue was $1.6 million and $3.6 million for the three and six months ended June 30, 2024, respectively, compared to $2.2 million and $4.1 million for the same periods in 2023. The collaboration revenue reflects the partial recognition of the $15.0 million upfront payment the company received in April 2023 in connection with the collaboration and license agreement with Kaken Pharmaceutical. Research and Development (R&D) Expenses: R&D expenses for the three and six months ended June 30, 2024 were $8.1 million and $18.4 million, respectively, compared to $13.1 million and $24.8 million for the same periods in 2023. The overall decrease in R&D expenses was primarily driven by the decrease in clinical development and manufacturing expenses related to the termination of the CAHmelia-203 study, completion of enrollment in the company’s CAHmelia-204 study, and completion of the Phase 2 POWER study in PCOS. General and Administrative (G&A) Expenses: G&A expenses for the three and six months ended June 30, 2024 were $3.6 million and $7.9 million, respectively, compared to $3.0 million and $6.5 million for the same periods in 2023. Total Operating Expenses: Total operating expenses for the three and six months ended June 30, 2024 were $11.6 million and $26.3 million, respectively, compared to $16.1 million and $31.3 million for the same periods in 2023. Operating expenses include non-cash stock-based compensation expenses of $1.7 million and $3.2 million for the three and six months ended June 30, 2024, respectively, compared to $1.2 million and $2.3 million for the same periods in 2023. Net Loss: Net loss for the three and six months ended June 30, 2024 was $9.2 million and $20.8 million, respectively, compared to $12.8 million and $25.6 million for the same periods in 2023. About Spruce Biosciences Spruce Biosciences is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for endocrine and neurological disorders with significant unmet medical need. Spruce is developing its product candidate, tildacerfont, an oral, second-generation CRF 1 receptor antagonist, for the treatment of congenital adrenal hyperplasia (CAH), polycystic ovary syndrome (PCOS) and major depressive disorder (MDD). To learn more, visit www.sprucebio.com and follow us on X , LinkedIn , Facebook and YouTube . Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the design, results, conduct, progress and timing of Spruce’s clinical trials; Spruce’s expectations regarding reporting results of its clinical trials in 2024; Spruce’s plans to meet with the FDA to discuss the potential registrational path forward of tildacerfont for adult and pediatric classic CAH; and Spruce’s product candidate, strategy and regulatory matters. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “anticipate”, “will”, “potential”, “suggest”, “plan”, “intend” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Spruce’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Spruce’s business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Spruce’s filings with the U.S. Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Spruce undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. SPRUCE BIOSCIENCES, INC. CONDENSED BALANCE SHEETS (unaudited) (in thousands, except share and per share amounts) June 30, December 31, 2024 2023 ASSETS Current assets: Cash and cash equivalents $ 69,683 $ 96,339 Prepaid expenses 2,698 3,876 Other current assets 1,531 1,968 Total current assets 73,912 102,183 Right-of-use assets 1,060 1,181 Other assets 547 582 Total assets $ 75,519 $ 103,946 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable $ 682 $ 3,332 Accrued expenses and other current liabilities 10,683 14,600 Term loan, current portion 1,622 1,622 Deferred revenue, current portion 1,298 4,911 Total current liabilities 14,285 24,465 Lease liabilities, net of current portion 880 1,019 Term loan, net of current portion 923 1,717 Other liabilities 262 236 Total liabilities 16,350 27,437 Commitments and contingencies Stockholders’ equity: Preferred stock, $0.0001 par value; 10,000,000 shares authorized and no shares issued or outstanding as of June 30, 2024 and December 31, 2023 — — Common stock, $0.0001 par value; 200,000,000 shares authorized as of June 30, 2024 and December 31, 2023; 41,302,599 and 41,029,832 shares issued and outstanding as of June 30, 2024 and December 31, 2023, respectively 4 4 Additional paid-in capital 277,203 273,737 Accumulated deficit (218,038 ) (197,232 ) Total stockholders’ equity 59,169 76,509 Total liabilities and stockholders’ equity $ 75,519 $ 103,946 SPRUCE BIOSCIENCES, INC. CONDENSED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) (in thousands, except share and per share amounts) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Collaboration revenue $ 1,610 $ 2,165 $ 3,612 $ 4,129 Operating expenses: Research and development 8,090 13,126 18,407 24,838 General and administrative 3,556 3,011 7,874 6,462 Total operating expenses 11,646 16,137 26,281 31,300 Loss from operations (10,036 ) (13,972 ) (22,669 ) (27,171 ) Interest expense (83 ) (127 ) (180 ) (258 ) Interest income and other expense, net 938 1,275 2,043 1,814 Net loss (9,181 ) (12,824 ) (20,806 ) (25,615 ) Other comprehensive gain, net of tax: Unrealized gain on available for sale securities — 133 — 503 Total comprehensive loss $ (9,181 ) $ (12,691 ) $ (20,806 ) $ (25,112 ) Net loss per share, basic and diluted $ (0.22 ) $ (0.32 ) $ (0.51 ) $ (0.71 ) Weighted-average shares of common stock outstanding, basic and diluted 41,163,209 40,547,925 41,129,719 36,247,931