Hyperandrogenism

Spruce Biosciences Reports Baseline Characteristics from CAHmelia-203 and CAHmelia-204 Studies in Adult Classic CAH

Retrieved on: 
Friday, January 5, 2024

Spruce Biosciences, Inc .

Key Points: 
  • Spruce Biosciences, Inc .
  • (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, today reported baseline characteristics of patients enrolled in the CAHmelia-203 and CAHmelia-204 clinical studies of tildacerfont for the treatment of adult classic congenital adrenal hyperplasia (CAH).
  • “The baseline characteristics of patients enrolled in our CAHmelia-203 and CAHmelia-204 studies reinforce our adult CAH program enrichment strategy and underscore the clinical significance of both hyperandrogenemia and hypercortisolemia within this patient population,” said Javier Szwarcberg, M.D., M.P.H., Chief Executive Officer of Spruce Biosciences.
  • Collectively, we are encouraged by these baseline characteristics and look forward to reporting topline results from CAHmelia-203 in March, followed by CAHmelia-204 in the third quarter.”

Spruce Biosciences Provides Clinical Program Updates and Outlook for 2024

Retrieved on: 
Thursday, January 4, 2024

“2023 was a year of exceptional clinical execution across the board, and we were pleased to reach important milestones by completing enrollment in CAHmelia-203 for adult classic CAH and CAHptain-205 for pediatric classic CAH,” said Javier Szwarcberg, M.D., M.P.H., Chief Executive Officer of Spruce Biosciences.

Key Points: 
  • “2023 was a year of exceptional clinical execution across the board, and we were pleased to reach important milestones by completing enrollment in CAHmelia-203 for adult classic CAH and CAHptain-205 for pediatric classic CAH,” said Javier Szwarcberg, M.D., M.P.H., Chief Executive Officer of Spruce Biosciences.
  • The primary endpoint of the clinical trial is the percentage change in A4 from baseline at week 12.
  • Enrollment for CAHmelia-204 Study in Adult Classic CAH to be Completed in January 2024: Enrollment in CAHmelia-204 is anticipated to be completed in January 2024, and will surpass target enrollment of 90 patients.
  • CAHptain-205 Study in Pediatric Classic CAH Completes Enrollment: Enrollment in CAHptain-205 is complete with 30 patients, surpassing target enrollment of 20 patients.

Diurnal Ltd., a Neurocrine Biosciences Company, Presents Phase 3 Extension Study Data for Modified-Release Hydrocortisone in Patients with Congenital Adrenal Hyperplasia at ENDO 2023

Retrieved on: 
Thursday, June 15, 2023

Diurnal Ltd. developed this modified-release formulation of hydrocortisone, which has been specifically designed to replicate the natural circadian release of cortisol.

Key Points: 
  • Diurnal Ltd. developed this modified-release formulation of hydrocortisone, which has been specifically designed to replicate the natural circadian release of cortisol.
  • Current treatment guidelines recommend a target daily hydrocortisone dose of ≤25 mg (or equivalent dose of prednisone, prednisolone, or dexamethasone) in adult (fully grown) patients with CAH.
  • The post hoc analyses examined total glucocorticoid daily dose and 9:00 a.m. 17-hydroxyprogesterone (17-OHP) levels in patients treated with modified-release hydrocortisone in an open-label Phase 3 extension study.
  • Of the 71 patients who completed 24 months in the Phase 3 extension study at the time of the interim cut, 64 patients had 9:00 a.m. 17-OHP data for analysis.

Brazilian Regulatory Authority Grants Approval for Waylivra™ for Familial Partial Lipodystrophy

Retrieved on: 
Thursday, December 1, 2022

SOUTH PLAINFIELD, N.J., Dec. 1, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra™ (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil. This is the first approval globally for Waylivra for the FPL indication. Waylivra is also approved in Brazil for the treatment of Familial Chylomicronemia Syndrome (FCS).

Key Points: 
  • SOUTH PLAINFIELD, N.J., Dec. 1, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Brazilian Health Regulatory Agency, ANVISA (Agncia Nacional de Vigilncia Sanitria), has approved Waylivra (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil.
  • This is the first approval globally for Waylivra for the FPL indication.
  • Waylivra is also approved in Brazil for the treatment of Familial Chylomicronemia Syndrome (FCS).
  • ANVISA's approval for Waylivra for FPL was based on results from the Phase 2/3 BROADEN study in patients with familial partial lipodystrophy.

Recordati Rare Diseases Announces Several Scientific Abstracts to Be Presented at the Upcoming Endo Society Annual Meeting

Retrieved on: 
Wednesday, June 8, 2022

Recordati Rare Diseases Inc. announced today that various scientific abstracts have been accepted and will be presented onsite at the ENDO 2022 annual meeting being held in Atlanta, Georgia from June 11 14, 2022.

Key Points: 
  • Recordati Rare Diseases Inc. announced today that various scientific abstracts have been accepted and will be presented onsite at the ENDO 2022 annual meeting being held in Atlanta, Georgia from June 11 14, 2022.
  • For more information visit www.isturisa.com
    About Recordati Rare Diseases Inc.
    Recordati Rare Diseases Inc. is a biopharmaceutical company committed to providing often-overlooked orphan therapies to the underserved rare disease communities of the United States.
  • Recordati Rare Diseases mission is to reduce the impact of extremely rare and devastating diseases by providing urgently needed therapies.
  • We work side-by-side with rare disease communities to increase awareness, improve diagnosis and expand availability of treatments for people with rare diseases.

Phase III LINC 3 Study Demonstrates That ISTURISA® (osilodrostat) Improves Physical Features Associated With Hypercortisolism in Patients With Cushing’s Disease

Retrieved on: 
Friday, May 13, 2022

Recordati Rare Diseases Inc. announced today that the Phase III LINC 3 study demonstrates ISTURISA (osilodrostat) improves physical features associated with hypercortisolism in patients with Cushings disease.

Key Points: 
  • Recordati Rare Diseases Inc. announced today that the Phase III LINC 3 study demonstrates ISTURISA (osilodrostat) improves physical features associated with hypercortisolism in patients with Cushings disease.
  • Patients with Cushings disease experience multiple physical manifestations of hypercortisolism that may reduce quality of life.
  • According to the abstract entitled Osilodrostat Therapy Improves Physical Features Associated with Hypercortisolism in Patients with Cushings Disease: Findings from the Phase III LINC 3 Study, 137 adult Cushings disease patients with mUFC >1.5 x the upper limit of normal were enrolled in the 48-week core phase to evaluate the safety and efficacy of ISTURISA in patients with Cushings disease.
  • Changes to physical features caused by Cushings disease can have a significant impact on patient health and well-being.

Recordati Rare Diseases Announces Multiple Scientific Abstracts to Be Highlighted at American Association of Clinical Endocrinology

Retrieved on: 
Tuesday, May 10, 2022

Recordati Rare Diseases Inc. announced today that various scientific abstracts have been accepted and will be featured onsite at the American Association of Clinical Endocrinology (AACE) annual meeting being held in San Diego, California from May 12 14, 2022.

Key Points: 
  • Recordati Rare Diseases Inc. announced today that various scientific abstracts have been accepted and will be featured onsite at the American Association of Clinical Endocrinology (AACE) annual meeting being held in San Diego, California from May 12 14, 2022.
  • Key Onsite abstracts presented by Alberto Pedroncelli MD, Head of Clinical Development & Medical Affairs, Global Endocrinology, Recordati AG.
  • For more information visit www.isturisa.com
    About Recordati Rare Diseases Inc.
    Recordati Rare Diseases Inc. is a biopharmaceutical company committed to providing often-overlooked orphan therapies to the underserved rare disease communities of the United States.
  • Recordati Rare Diseases mission is to reduce the impact of extremely rare and devastating diseases by providing urgently needed therapies.

Organon to Acquire Forendo Pharma

Retrieved on: 
Thursday, November 11, 2021

Organon (NYSE: OGN), and Forendo Pharma today announced that the companies have entered into a definitive agreement, under which Organon will acquire Forendo, a clinical-stage drug development company focused on novel treatments in womens health.

Key Points: 
  • Organon (NYSE: OGN), and Forendo Pharma today announced that the companies have entered into a definitive agreement, under which Organon will acquire Forendo, a clinical-stage drug development company focused on novel treatments in womens health.
  • Forendo is pioneering the science of intracrinology, addressing disease through a novel, tissue-specific approach.
  • Our acquisition of Forendo further demonstrates our commitment to building a pipeline of impactful treatment options for women with unmet medical needs.
  • In addition to womens health programs, Forendo has a strategic collaboration with Novartis leveraging its unique HSD17B platform in chronic liver diseases.

Spruce Biosciences Reports Second Quarter 2021 Financial Results and Provides Corporate Updates

Retrieved on: 
Tuesday, August 10, 2021

Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, today reported financial results for the second quarter ended June 30, 2021 and provided a corporate update.

Key Points: 
  • Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, today reported financial results for the second quarter ended June 30, 2021 and provided a corporate update.
  • With clearance of the IND, Spruce remains on track to initiate a Phase 2 proof of concept study later this year.
  • Spruce management uses these non-GAAP financial measures to monitor and evaluate its operating results and trends on an on-going basis, and internally for operating, budgeting and financial planning purposes.
  • The non-GAAP financial measures should be considered in addition to results prepared in accordance with GAAP but should not be considered a substitute for or superior to GAAP results.

Spruce Biosciences Announces Publication of Phase 2 Results for Tildacerfont in Journal of Clinical Endocrinology and Metabolism

Retrieved on: 
Monday, June 21, 2021

SPR001-201 and SPR001-202 comprised the entire Phase 2a clinical development program for tildacerfont in adult classic CAH.

Key Points: 
  • SPR001-201 and SPR001-202 comprised the entire Phase 2a clinical development program for tildacerfont in adult classic CAH.
  • Tildacerfont has been evaluated in 171 patients across seven clinical trials in which it has been generally well tolerated.
  • Spruce is also developing tildacerfont for women suffering from a rare form of polycystic ovary syndrome (PCOS) with primary adrenal androgen excess.
  • Such forward-looking statements include statements regarding, among other things, the results, conduct, progress and timing of Spruces clinical trials.