An Endocrine News roundup of the week’s pharmaceutical news, breakthroughs, and general information. *
Spruce Biosciences Completes Enrollment in CAHmelia-204 Study for Adult Classic Congenital Adrenal Hyperplasia
Last month, Spruce Biosciences, announced completion of enrollment in its CAHmelia-204 clinical trial of tildacerfont for the treatment of adult classic congenital adrenal hyperplasia (CAH).
Spruce Biosciences is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need.
“Completing enrollment in our CAHmelia-204 clinical trial marks a significant milestone in our adult classic CAH program. This achievement reinforces the continued momentum within our tildacerfont program and positions us favorably to report topline results in the third quarter of 2024,” said Endocrine Society member Javier Szwarcberg, MD, MPH, chief executive officer of Spruce Biosciences. “Thanks to positive engagement from our patient community and study investigators alike, the CAHmelia-204 clinical trial enrolled 100 patients, exceeding target enrollment of 90 patients.”
“Looking ahead, assuming positive results from CAHmelia-203 in March and CAHmelia-204 in the third quarter, we plan to meet with the U.S. Food and Drug Administration (FDA) and comparable foreign regulatory authorities to discuss the potential registrational path forward for tildacerfont as a treatment for adult classic CAH,” Szwarcberg continued.
CAHmelia-204 is a randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of tildacerfont, a potentially novel, once-daily, non-steroidal treatment option, in reducing glucocorticoid usage in 100 adults with classic CAH on supraphysiologic doses of glucocorticoids with normal or near normal levels of androstenedione (A4) at baseline. The primary endpoint of this clinical trial is the absolute change in daily glucocorticoid dose in hydrocortisone equivalents (HCe) from baseline at week 24.
About CAHmelia-203
CAHmelia-203 is a randomized, double-blind, placebo-controlled, dose ranging Phase 2b clinical trial evaluating the safety and efficacy of tildacerfont in adults with classic CAH and highly elevated levels of A4 at baseline while on stable glucocorticoid dosing. This clinical trial enrolled 96 subjects with elevated levels of A4. For the first six weeks, patients will receive blinded placebo to assess their adherence to their existing glucocorticoid therapy. Patients who continue to meet all eligibility criteria at the end of this period will enter a three-part treatment period. During the placebo-controlled treatment period, patients will be randomized in a blinded manner to receive placebo, 50mg, 100mg, or 200mg tildacerfont once daily. Dosing in the placebo-controlled treatment period will continue for 12 weeks. The primary endpoint of the clinical trial is the percentage change in A4 from baseline to week 12. Following the placebo-controlled treatment period, all patients will receive tildacerfont following a dose-escalation protocol that allows dose increase to 200mg once daily over 12 weeks. Following the 12-week dose-escalation period, all patients will continue receiving tildacerfont at 200mg once daily for an additional 46 weeks. Patients who achieve control of A4 while on supraphysiologic glucocorticoid treatment will have the opportunity to reduce their glucocorticoid dosing in the open-label period according to a pre-specified algorithm in the protocol. For more information about the CAHmelia program, please visit https://www.sprucebio.com/cahmelia.
About CAHmelia-204
CAHmelia-204 is a randomized, double-blind, placebo-controlled Phase 2b clinical trial to evaluate the safety and efficacy of tildacerfont in reducing glucocorticoid usage in 100 adults with classic CAH on supraphysiologic doses of glucocorticoids with normal or near normal levels of A4 at baseline. This clinical trial is designed in two parts. In the first part of the clinical trial, patients will be randomized to receive 200mg tildacerfont once daily or placebo for 24 weeks. During the second part of the clinical trial, all patients will receive open-label 200mg tildacerfont once daily for 52 weeks. Throughout the trial, tapering of glucocorticoids will be guided according to a pre-specified algorithm and continue to the lowest level possible (physiologic replacement levels), as long as patients remain well controlled based on standard biomarkers and clinical assessments. The primary endpoint of this clinical trial is the absolute change in daily glucocorticoid dose in HCe from baseline at week 24. For more information about the CAHmelia program, please visit https://www.sprucebio.com/cahmelia.
Amylyx Pharmaceuticals Completes Enrollment in HELIOS, a Phase 2 Study Wolfram Syndrome Treatment
Amylyx Pharmaeuticals, Inc., today announced it has completed enrollment of its Phase 2 HELIOS trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of Wolfram syndrome (WS), a rare genetic disease that typically results in neurodegeneration and premature death, which has no known cure. The trial has enrolled 12 adult participants living with WS, and preliminary results are anticipated in the second half of 2024.
“The unmet need for people living with WS remains significant. WS is progressive, often fatal, and difficult to diagnose, with current treatments focused mostly on helping to address symptoms,” said Fumihiko Urano, MD, PhD, principal investigator of the HELIOS clinical trial and professor of medicine in the Division of Endocrinology, Metabolism & Lipid Research at Washington University School of Medicine. “Our preclinical findings showed that AMX0035 may impact relevant WS disease pathways and manifestations, and we look forward to the preliminary results from HELIOS anticipated later this year.”
HELIOS is an open-label proof of biology trial designed to study the effect of AMX0035 on safety and tolerability, and various measures of endocrinological, neurological, and ophthalmologic function in adult participants living with WS. Data from this initial study will inform potential future Amylyx trials for individuals with WS.
More information on the HELIOS clinical study can be found at www.clinicaltrials.gov, NCT05676034.
In September 2022, researchers from Washington University School of Medicine in St. Louis, in collaboration with Amylyx, published preclinical data in the peer-reviewed Journal of Clinical Investigation Insight. The data explored the potential of AMX0035 as a novel therapeutic approach for WS and provided initial proof-of-concept for its therapeutic development in WS.
“Little progress has been made in the treatment of WS, and we are excited by the potential we have been seeing in the preclinical work with AMX0035,” says Endocrine Society member Camille L. Bedrosian, MD, chief medical officer of Amylyx. “These preclinical findings suggest the possibilities of AMX0035 for individuals with WS that we continue to explore, including in the HELIOS trial. We are grateful to those who partnered to complete this important clinical trial enrollment milestone, in particular, the WS community, their families and caregivers, experts, clinicians, researchers, and advocacy leaders.”
Amylyx announced that the FDA granted orphan drug designation to AMX0035 for the treatment of WS in November 2020. The FDA may grant this designation to drugs and biologics intended to treat a rare disease or condition affecting fewer than 200,000 persons in the U.S. Orphan designation qualifies a company for certain benefits, including financial incentives to support clinical development and the potential for seven years of market exclusivity in the U.S. upon regulatory approval.
About Wolfram Syndrome
Wolfram syndrome (WS) is an autosomal recessive neurodegenerative disease characterized by childhood-onset diabetes, optic nerve atrophy, and neurodegeneration. Common manifestations of WS include diabetes mellitus, optic nerve atrophy, central diabetes insipidus, sensorineural deafness, neurogenic bladder, and progressive neurologic difficulties. Genetic and experimental evidence suggest that endoplasmic reticulum (ER) dysfunction is a critical pathogenic component of WS. The prognosis of WS is poor, and many people with the disease die prematurely with severe neurological disabilities.
Enable Biosciences Enhances ‘Kit to Clinics’ with ADAP Technology for Diabetes Care
On January 23, Enable Biosciences, Inc., announced the enhancement of its ‘Kit to Clinics’ program for type 1 diabetes care, featuring the ADAP (Antibody Detection by Agglutination-PCR) technology.
According to the company, the technology has been rigorously and exhaustively vetted through multiple peer-reviewed and/or blinded studies. This includes top performances in three evaluations administered by the Islet Autoantibody Standardization Program of the Immunology of Diabetes Society, underscoring its reliability and effectiveness.
The ‘Kit to Clinics’ program, aimed at revolutionizing early detection and care of type 1 diabetes, provides clinics with this advanced testing technology and educational resources, emphasizing early intervention’s importance. With recent developments in treatments capable of delaying the onset of diabetes, early and accurate detection provided by ADAP is crucial.
ADAP technology’s precision in early detection is central to the program, having been validated in various rigorous scientific studies. This validation ensures that clinics employing the ‘Kit to Clinics’ program are equipped with one of the most reliable and innovative tools available for diabetes care.
David Seftel, MD, MBA, CEO of Enable Biosciences, comments on the program’s significance: “The ‘Kit to Clinics’ program, bolstered by our extensively vetted ADAP technology, signifies a major advancement in diabetes care. This initiative aligns with the latest therapeutic approaches, offering the possibility of changing the trajectory of type 1 diabetes.”
The program has been successfully implemented in pilot phases across several clinics, including Adams County Clinic in Idaho, University Hospital in New Jersey, and Dell’s Children Hospital in Texas. These clinics are leading the way in incorporating ADAP technology into their diabetes care services.
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