Spruce Biosciences Achieves Proof of Concept in Phase 2 Study of Tildacerfont in Congenital Adrenal Hyperplasia

-Tildacerfont demonstrates meaningful reduction in key disease biomarkers adrenocorticotropic hormone (ACTH), 17-hydroxyprogesterone (17-OHP) and androstenedione (A4)

-Well-tolerated and safe, tildacerfont holds potential as a treatment for patients with congenital adrenal hyperplasia, a rare endocrine disorder

San Francisco – March 25, 2019 – Spruce Biosciences, a clinical-stage biotechnology company developing novel therapies for rare endocrine disorders, today reported positive proof of concept data from a Phase 2 multicenter, multiple-dose, dose-escalation trial of tildacerfont, an oral corticotropin-releasing factor type-1 (CRF1) receptor antagonist, for the treatment of patients with congenital adrenal hyperplasia (CAH). The Phase 2 results were presented in a late-breaking poster presentation at the Annual Meeting of the Endocrine Society (ENDO 2019), the premier conference for endocrine science and medicine.

Spruce’s Phase 2 trial involved 18 patients with uncontrolled classic CAH. Ten patients in Cohort A received two weeks of treatment at each of three doses: 200, 600 and 1,000 mg tildacerfont once per day (QD) for a total of six weeks of treatment. Eight patients in Cohort B were treated for two weeks with 400 mg per day (200 mg twice per day [BID]). The study was designed to assess safety, tolerability, pharmacokinetics and the ability of tildacerfont to reduce adrenal androgens (androstenedione [A4]), progestins (17-hydroxyprogesterone [17-OHP]) and adrenocorticotropic hormone (ACTH), the primary driver of adrenal tissue hyperplasia.

Across both cohorts, tildacerfont was well-tolerated, exhibited a predictable, dose-dependent pharmacokinetic profile and was effective in reducing excess A4, 17-OHP and ACTH. There were no serious AEs and no AEs leading to withdrawal.

In Cohort A, improvements in A4 were demonstrated in 10 of 10 patients (100 percent), and 17-OHP and ACTH were both improved in 8 of 10 patients (80 percent). In Cohort B, improvements in A4 were demonstrated in 6 of 8 patients (75 percent), 17-OHP in 6 of 7 patients (86 percent) and ACTH in 5 of 7 patients (71 percent).

Additionally, after six weeks of treatment with tildacerfont, the one male patient with confirmed testicular adrenal rest tumors (TARTs) at baseline saw a notable reduction in tumor size after 6 weeks of therapy. TARTs are ACTH-responsive, space-occupying lesions of the testes that lead to pain and infertility in men and boys as young as four years old, and represent a meaningful unmet need in males with CAH.

“These results demonstrate that a targeted therapy like tildacerfont, capable of treating the underlying disease is on the horizon for patients with CAH” said Kyriakie Sarafoglou, M.D., associate professor, department of pediatrics at the University of Minnesota and first author of the poster.

Alexis Howerton, Ph.D., CEO at Spruce Biosciences, added: “We are excited to be able to share these data with the clinical community at ENDO 2019, which reveal that the majority of patients treated with tildacerfont achieved meaningful reductions across the three key biomarkers for CAH. We are highly motivated by the potential to fulfill a therapeutic void for patients living with CAH and are optimistic that a CRF1 receptor antagonist such as tildacerfont will be the right treatment solution for this rare disease.”

CAH is a rare endocrine disease of impaired cortisol synthesis coupled with adrenal androgen excess. Although CAH is a part of the nationwide newborn screening program, there are currently no FDA-approved therapies. Patients are often prescribed supraphysiologic doses of steroids to suppress androgen production, yet the vast majority of patients continue to suffer from poorly controlled disease, along with the adverse effects associated with high dose steroids.

For more information on Spruce, please visit www.sprucebiosciences.com.

 

About Spruce Biosciences
Spruce Biosciences is a clinical-stage biotechnology company developing novel therapies for rare endocrine disorders. Spruce’s lead program, tildacerfont (formerly SPR001), is an oral, non-steroidal small molecule with the potential to be the first FDA-approved therapy for CAH. For more information on Spruce Biosciences and its lead clinical program for CAH, please visit www.sprucebiosciences.com.

Media Contact
Amanda Guisbond
Canale Communications
781-405-8775
amanda@canalecomm.com

 

 


Spruce Biosciences Announces Presentation of Positive Topline Data from Phase 2 Study of Tildacerfont (SPR001) in Congenital Adrenal Hyperplasia at the 2019 Annual Meeting of the Endocrine Society (ENDO)

Tildacerfont demonstrates potential to become the first approved therapy for congenital adrenal hyperplasia, a rare endocrine disorder.

SAN FRANCISCOMarch 8, 2019 /PRNewswire/ — Spruce Biosciences, a clinical-stage biotechnology company developing novel therapies for rare endocrine disorders, today announced that the abstract, titled “A Phase 2, Dose-Escalation, Safety and Efficacy Study of Tildacerfont (SPR001) for the Treatment of Patients with Classic Congenital Adrenal Hyperplasia,” has been selected for a late-breaking poster presentation at the 2019 Annual Meeting of the Endocrine Society (ENDO), the world’s largest event for endocrine science and medicine.

“We regularly hear from patients and physicians who are looking for new treatment options for CAH,” says Spruce CEO Alexis Howerton, Ph.D. “We are pleased to be in a position to share positive data from our Phase 2 program, which demonstrated that tildacerfont was well-tolerated and effective in reducing ACTH and adrenal steroids. We look forward to sharing the data in more detail at ENDO later this month.”

Dina Matos, Executive Director, CARES Foundation added: “There continues to be a critical need for a therapeutic to enhance quality of life for patients by improving hormone levels and reducing steroid use. We are pleased to support Spruce’s efforts as it swiftly advances its lead program.”

CAH is a rare endocrine disease of impaired cortisol synthesis coupled with adrenal androgen excess. Although CAH is a part of the nationwide newborn screening program, there are currently no FDA-approved therapies for CAH.

Details of Spruce’s ENDO presentation are below.

Abstract: 8030
Title: A Phase 2, Dose-Escalation, Safety and Efficacy Study of Tildacerfont (SPR001) for the Treatment of Patients with Classic Congenital Adrenal Hyperplasia
Presenter: Kyriakie Sarafoglou, M.D., Associate Professor, Department of Pediatrics, University of Minnesota 
Date and TimeSunday, March 24, 20191:00-3:00 p.m. ET
Location: Poster Board # SUN-LB064, ENDO Expo Hall, Ernest N. Morial Convention Center

About Spruce Biosciences

Spruce Biosciences is a clinical-stage biotechnology company developing novel therapies for rare endocrine disorders. Spruce’s lead program, tildacerfont (formerly SPR001), is an oral, non-steroidal small molecule with the potential to be the first FDA-approved therapy for CAH. For more information on Spruce Biosciences and its lead clinical program for CAH, please visit www.sprucebiosciences.com.


Spruce Biosciences Appoints David Moriarty, Ph.D., as Vice President of Development Operations

Moriarty brings 18 years of clinical industry experience to Spruce to advance SPR001, the company’s lead program for congenital adrenal hyperplasia (CAH), through Phase 2 clinical trials and expand into additional indications

 

San Francisco – July 17, 2018 – Spruce Biosciences, a clinical-stage biotechnology company developing novel therapies for rare endocrine disorders, today announced the appointment of David Moriarty, Ph.D., as Vice President of Development Operations.

David Moriarty joins Spruce Biosciences’ management team

“David brings extensive experience in clinical research and operations to Spruce at the exact time we need it,” said Alexis Howerton, Ph.D., Chief Executive Officer of Spruce Biosciences. “We are currently advancing our lead clinical product candidate, SPR001, through Phase 2 clinical trials. As we continue to progress the program, we will rely on David’s operational expertise, which includes leading clinical operations and driving multiple clinical programs at large pharmaceutical companies that ultimately led to product approvals. We are excited to add David to our team as we move toward initiating pivotal clinical studies and bringing the potentially first approved therapy for congenital adrenal hyperplasia to patients.”

Spruce is currently enrolling patients in its SPR001 Phase 2 trial and has active sites in San Diego, CA, Orange, CA, Las Vegas, NV, Atlanta, GA, Indianapolis, IN, Minneapolis, MN, Philadelphia, PA and Melbourne, FL. Earlier this year, Spruce also launched its CAH Natural History Study to enable patients with CAH to contribute their experiences to advance knowledge of the disease, including its management and treatment.

Unlike many other rare diseases, CAH is part of the newborn screening program and is highly identifiable. There are currently no FDA-approved therapies for CAH, which is caused by genetic mutations resulting in the inability to produce cortisol, the critical ‘stress’ hormone. The most common form of CAH, 21 hydroxylase deficiency, affects approximately 1 in 10,00 to 15,000 people in the United States.

Prior to joining Spruce, Dr. Moriarty served as Vice President of Clinical Operations and Data Management at Jazz Pharmaceuticals, where he assembled a successful clinical operations, data management and strategic outsourcing team delivering multiple submissions in hematology/oncology and sleep medicine, leading to approvals for Defitelio and Vxyeos. Before Jazz, he was responsible for leading clinical operations in different Janssen Pharmaceutical Alzheimer’s Disease late development clinical programs over several years. Earlier at Janssen, formerly Tibotec Therapeutics, he held clinical operations roles leading to the approved HIV therapies of Prezista and Edurant. Dr. Moriarty also previously held positions of increasing responsibility at PPD, a global CRO, working primarily on the late development work of Humira for Abbott Pharmaceuticals. Dr. Moriarty earned a B.Sc. in Neuroscience from University of Central Lancashire and a Ph.D. in Neuroscience from the University of Manchester.

“I am pleased to join the talented team at Spruce with the objective of developing a new, effective therapy that will potentially benefit patients with CAH,” said Moriarty. “I am looking forward to leveraging my experience as a clinical development leader of now-approved products through the clinical trial process on behalf of Spruce, as the company advances its lead program in CAH.”

Spruce is currently conducting a Phase 2 clinical trial to assess the safety and efficacy of SPR001 in adults with classic CAH. For more information on Spruce Biosciences and its lead clinical program for CAH, please visit sprucebiosciences.com.

About Spruce Biosciences

Spruce Biosciences is a clinical-stage biotechnology company developing novel therapies for rare endocrine disorders. The Spruce team is leveraging their extensive expertise in endocrinology and orphan drug development to meet the significant unmet need of patients suffering from rare endocrine diseases. Spruce’s lead product candidate, SPR001, has a novel and clinically verified mechanism of action (MOA) and is currently in Phase 2 clinical trials for congenital adrenal hyperplasia, a rare disease that is screened in newborns and for which there is not yet an FDA-approved therapy. The Company is headquartered in San Francisco and closed a Series A Financing of $20 million in 2016. For more information on Spruce, please visit sprucebiosciences.com.

 

Media Contact
Amanda Guisbond
Canale Communications
781-405-8775
amanda@canalecomm.com


Spruce Biosciences is a clinical-stage biotechnology company developing novel therapies for rare endocrine disorders. We are leveraging our extensive expertise in endocrinology and orphan drug development to meet the significant unmet need of patients suffering from these diseases. We are committed to transforming the quality of life for patients who have been underserved by scientific innovation.