Mar 09, 2024

Pelage Presents Late-Breaking Data at AAD 2024 Meeting Demonstrating PP405 Activates Human Hair Follicle Stem Cells Ex Vivo and in Phase 1 Clinical Study

Potent topical MPC inhibitor, based on discovery of a metabolic switch, reawakens dormant hair follicle stem cells to stimulate hair growth
PP405 demonstrates statistically significant activation of Ki67 in the hair follicle, a well-established marker of stem cell proliferation

LOS ANGELES — Pelage Pharmaceuticals, a clinical-stage regenerative medicine company pioneering a new generation of treatments for hair loss, today presented positive translational and Phase 1 clinical data demonstrating the company’s novel small molecule therapy PP405 reactivates dormant hair follicle stem cells to trigger hair growth. Data were presented in a late-breaking oral session of the American Academy of Dermatology (AAD) 2024 Annual Meeting in San Diego, titled Inhibition of pyruvate oxidation activates human hair follicle stem cells ex vivo.

“The translational data derived from our ex vivo studies on human facelift skin have been further validated in a Phase 1 first-in-human clinical trial demonstrating PP405 effectively reactivates dormant hair follicle stem cells,” said Daniel Gil, Ph.D., CEO of Pelage Pharmaceuticals. “This therapy represents an exciting new option for not only treating but reversing hair loss. We look forward to advancing PP405 to Phase 2 clinical studies this year.”

PP405 is a potent topical mitochondrial pyruvate carrier (MPC) inhibitor that acts on the cellular metabolic pathway to upregulate lactate dehydrogenase (LDH), which stem cells are particularly sensitive to, resulting in their activation and hair growth.

Data presented at AAD showed in human facelift skin, single topical applications of 0.006% and 0.06% PP405 led to an increase in LDH activity in hair follicle stem cells within 24 hours. The LDH activity corresponded with a significant increase in Ki67 signal in the hair bulge, indicating a proliferative response of the hair follicle stem cells. The findings demonstrated proof of mechanism in human explanted skin consistent with the results in animal models.

The data informed Pelage’s Phase 1 clinical trial that concluded in January 2024, which demonstrated that PP405 was safe and well-tolerated, with the target pharmacokinetic profile achieved. Additionally, the Phase 1 study demonstrated proof of mechanism and target engagement in patients with androgenetic alopecia. There was a statistically significant increase in the Ki67 signal compared to baseline after just seven days of 0.05% PP405 topical treatment. Evidence of newly emerging hair germs – the hallmark of the telogen to anagen transition – was also observed. The topical molecule also received positive formulation reviews from patients.

“We are incredibly excited about this data, and PP405 offers an opportunity to change the landscape of alopecia treatment through directly targeting hair follicle stem cells at the source through a clear mechanistic pathway,” said Qing Yu Christina Weng, M.D., CMO of Pelage Pharmaceuticals. “The Phase 1b study of PP405 demonstrates a strong safety profile, confirms daily topical dosing with no detectable drug levels in the blood, and demonstrates proof of mechanism with statistically significant activation of hair follicle stem cells. We can also see the structural architecture of the follicle on biopsy moving through the hair cycle from resting to growing as the stem cells become activated.”

Pelage will begin its multi-center Phase 2a trial of PP405 in mid-2024, recruiting both men and women with androgenetic alopecia. PP405 may also have applications for other forms of alopecia, including telogen effluvium (stress-induced hair loss) and chemotherapy-induced hair loss.

About Pelage Pharmaceuticals

Pelage Pharmaceuticals is a clinical-stage regenerative medicine company developing novel treatments for hair loss including androgenetic alopecia and chemotherapy-induced-alopecia. With a focus on molecular and stem cell biology, Pelage is advancing a new class of treatments designed to reactivate dormant hair follicle stem cells and restore the body’s ability to naturally grow hair. Its lead program, PP405, is currently in clinical trials. Through its rigorous scientific foundation, topical formulation, and novel mechanism of action, Pelage is pioneering first-in-class hair growth solutions for people of all hair types experiencing hair loss.

About PP405

PP405 is a novel, non-invasive, topical small molecule designed to reactivate dormant hair follicle stem cells and restart hair growth. Through a regenerative medicine approach, the treatment focuses on addressing the metabolic processes that regulate the activation and inactivation phases of hair follicle stem cells. Early results from a Phase 1 trial show that PP405 was well-tolerated and demonstrated statistically significant activation of hair follicle stem cells. In 2018, Pelage Pharmaceuticals licensed the intellectual property to PP405 and related topical small molecules from the UCLA Technology Development Group.

About Pelage Pharmaceuticals

Pelage Pharmaceuticals is pioneering the first regenerative approach to hair loss, grounded in stem cell biology.

Pelage’s platform targets an intrinsic metabolic switch in hair follicles and is being developed for androgenetic alopecia and other forms of hair loss, with the aim of delivering a clinically tested, FDA-approved treatment suitable for all genders, skin types, and hair types.

Clinical Advisory Board

Arash Mostaghimi, M.D., M.P.A., M.P.H., F.A.A.D.

Vice Chair of Clinical Trials and Innovation, Director of the Dermatology Inpatient Service at Brigham & Women’s Hospital
Associate Professor of Dermatology, Harvard Medical School

Dr. Arash Mostaghimi is the Vice Chair of Clinical Trials and Innovation and Director of the Consult Service for the Department of Dermatology at Brigham & Women’s Hospital. With board certifications in dermatology, internal medicine, and clinical informatics, Dr. Mostaghimi is an active clinician with an emphasis on complex medical dermatology and hair loss disorders. As a clinical researcher, Dr. Mostaghimi has over 275 peer reviewed publication, and served as a principal investigator involved in the design and implementation of numerous clinical trials. He has received numerous teaching and mentorship awards and is co-director of the Complex Medical Dermatology Fellowship.

Clinical Advisory Board

Amy McMichael, M.D., F.A.A.D.

Professor and Past Chair of Dermatology, Wake Forest School of Medicine

Dr. Amy McMichael is a Philadelphia native who received her medical degree from the University of Pennsylvania School of Medicine. She completed Dermatology residency training at the University of Michigan School of Medicine.

Dr. McMichael is a Professor in the Department of Dermatology at Wake Forest School of Medicine in Winston-Salem, NC. She has held leadership positions in the Department for over 20 years first as Residency Program Director for 12 years and Chair of the Department for 11 years. She has been a leader in her field, including her roles as past President of Skin of Color Society and past President of the National Medical Association Dermatology Section. She recently served as a Board member of the American Academy of Dermatology.
Dr. McMichael’s clinical and research interests include: hair and scalp disorders, psoriasis, and skin of color. She is co-editor of several medical

Clinical Advisory Board

Mathew Avram, M.D., J.D., F.A.A.D.

Director of the Dermatology Laser & Cosmetic Center, Massachusetts General Hospital
Associate Professor of Dermatology, Harvard Medical School

Dr. Mathew M. Avram is the Director of the Massachusetts General Hospital Dermatology Laser & Cosmetic Center and serves as Faculty Director for Dermatology Cosmetic Training at Harvard Medical School. He is the Past-President of both the American Society for Dermatologic Surgery and American Society for Laser Medicine & Surgery. Dr. Avram has co-authored 7 textbooks and 90 peer-reviewed publications. Dr. Avram has delivered over 800 invited lectures, co-chaired teaching sessions for the FDA for three years, and has two U.S. patents. Dr. Avram has been recognized by Newsweek as one of the Top 10 cosmetic dermatologists in America.

Clinical Advisory Board

Amelia K. Hausauer, M.D., F.A.A.D.

Director of Dermatology and Minimally Invasive Aesthetics at Aesthetx

Dr. Amelia K. Hausauer is the Director of Dermatology and Head of Aesthetic Medicine at Aesthetx, a hybrid dermatology and plastic surgery practice in Northern California. She is a key opinion leader for early pipeline innovation and development of multiple injectable and regenerative therapies as well as an active research investigator. Dr. Hausauer spearheaded one of the largest clinical trials using PRP for hair growth and served as the chief editor for Platelet Rich Plasma (PRP) and Microneedling in Aesthetic Medicine. Earning a bachelor’s degree from Stanford University and medical doctorate from University of California San Francisco, Dr. Hausauer completed residency at New York University then an American Society for Dermatologic Surgery cosmetic surgery fellowship.

Leadership

Qing Yu Christina Weng, MD

Chief Medical Officer

Chief Medical Officer

Dr. Weng is the CMO of Pelage. She is a physician-scientist and entrepreneur, board-certified dermatologist, and Lecturer at Harvard Medical School. She is experienced in corporate startup strategy as Head of Business Development & Strategy at Kira Pharma and CSO and cofounder of Mymiel Skincare. She is on the Board of Directors for the Dermatology Innovation Forum and Dermatology Summit. Dr. Weng has an undergraduate degree from the California Institute of Technology, MD from Harvard, and she completed dermatology residency and fellowship at Harvard.

Leadership

Daniel Gil, PhD

Chief Executive Officer

Chief Executive Officer

Dr. Gil is the CEO of Pelage. He is also a Venture Partner with Visionary Ventures. Until 2018, Dr. Gil was a Vice President at Allergan, overseeing the research portfolio and early clinical development. His research contributed to development of 5 marketed drugs and he led 8 clinical development projects. He is an inventor on 70+ issued US patents and author on 45 peer-reviewed research articles. Dr. Gil has an undergraduate degree in Biology from Harvard University and a PhD in Pharmacology from the University of Pennsylvania.

Leadership

Michael Jung, PHD

Co-Founder

Co-Founder
Professor, UCLA Chemistry & Biochemistry

Dr. Jung is a renowned Medicinal Chemist and co-founder of several successful startups. He is also co-inventor of two FDA approved drugs for prostate cancer (Xtandi & Erleada) with several drugs currently in clinical trials.

Leadership

Heather Christofk , PhD

Co-Founder

Co-Founder
Professor, UCLA Biological Chemistry

Dr. Christofk is a leader in the metabolism field with a research focus on how metabolism regulates cell state transitions. Her groundbreaking collaborative research with Dr. Lowry revealed that changes in metabolism can instruct hair follicle stem cell activation, forming the basis for the Pelage’s approach to treat hair loss. She is a Professor of Biological Chemistry at the David Geffen School of Medicine at UCLA and Associate Director of Basic and Translational Research at the UCLA Jonsson Comprehensive Cancer Center. She earned her bachelor’s degree from UCLA, her PhD from Harvard, and conducted postdoctoral research at UCSF before joining the faculty at UCLA in 2008.

Leadership

William Lowry, PhD

President & Co-Founder

President & Co-Founder
Professor, UCLA Molecular, Cell and Dev. Biology

Dr. Lowry is a leader in the stem cell field with 20+ years of research experience resulting in the discovery that hair follicle stem cells can be activated by metabolic manipulation. His work has led to the creation of several startups including Pelage and Sardona, as well as other efforts under development.