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The Palvella Mission

Derived from the Finnish word for “to serve”, Palvella Therapeutics was founded with the single-minded goal of serving individuals suffering from serious rare genodermatoses, or genetic skin diseases. Of the almost 600 rare dermatologic diseases, less than 2% have an approved treatment. We’re committed to developing safe and effective therapies for people with no available treatments, or ineffective treatments, for their disease. By developing therapies that specifically target the root cause of these diseases, we believe we can dramatically enhance quality of life.

Our Focus

Pachyonychia Congenita
Gorlin Syndrome
QTORIN Platform

Pachyonychia Congenita

PTX-022 (QTORIN™ 3.9% rapamycin anhydrous gel), currently in a multi-center Phase 2/3 pivotal study, aims to be the first therapy for patients with Pachyonychia Congenita:

  • PC is a lifelong, chronically debilitating disease caused by mutations in the genes for keratins on the feet, resulting in fragile skin cells, severe blistering, and limited mobility
  • Affected individuals often require ambulatory aids (wheelchairs, crutches) or crawl on hands/knees for mobility
  • No FDA or EMA approved treatments for PC
  • Estimated 5,000 to 10,000 patients in the US
  • Fast Track Designation, FDA Orphan Drug Designation, and EMA orphan drug designation

1. Gallagher et al. (2019) Prevalence of Diagnosed/Highly Symptomatic Pachyonychia Congenita (Pc) Patients Managed Annually by US Dermatologists-National Real World Occurrence (RWO) Physician Study. J Dermatol Dis 6: 280.

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Gorlin Syndrome

PTX-367 (QTORIN™ rapamycin) is a novel topical therapy for basal cell carcinomas (BCC) associated with Gorlin Syndrome, a serious, lifelong genetic disease caused by a mutation in the PTCH1 gene. Palvella initiated internal research efforts on the potential for QTORIN™ rapamycin in Gorlin Syndrome in 2017.

  • BCCs are a malignant skin cancer requiring repeated and potentially disfiguring surgical removal.
  • Individuals afflicted with Gorlin Syndrome can develop hundreds of BCCs, oftentimes beginning in childhood.
  • No FDA approved therapies; Current therapies are limited by tumor recurrence, resistance, and tolerability issues.
  • The mTOR pathway has been recently implicated in BCC tumorigenesis in Gorlin Syndrome.1
  • Gorlin Syndrome affects an estimated 10,000 people in the US.
  • Palvella will commence a Phase 2b study in 2020.

1. Kim et al. SOX9 Transcriptionally Regulates mTOR-Induced Proliferation of Basal Cell Carcinomas. J Invest Dermatol. 2018 August; 138(8): 1716–1725.

QTORINTM Platform

Palvella’s proprietary QTORIN™ technology was developed over several years of research to overcome inherent challenges with topical delivery of mTOR inhibitors such as rapamycin – including stability, skin penetration, and skin distribution.
QTORIN™ utilizes a composition of excipients precisely selected and optimized to achieve drug stability at room temperature and enable cutaneous distribution into the stratum basale of the epidermis and the reticular dermis, where the disease pathology manifests. Two of Palvella’s programs, PTX-022 and PTX-367, leverage QTORIN™ to deliver the active agent directly to the root genetic cause of the disease.

Clinical Trials

Valo Study

The VALO Study is a Phase 2/3 multicenter clinical study evaluating the safety and efficacy of PTX-022 (QTORINTM rapamycin) in adults with moderate to severe pachyonychia congenita (PC). The Valo Study is a partnership between Palvella and PC Project, the leading patient advocacy group for Pachyonychia Congenita. Centers throughout the United States are currently recruiting individuals to participate in the trial. The trial will require up to 6 clinic visits and includes travel assistance if required.

Wes Kaupinen

Wes Kaupinen

President and CEO

Wes Kaupinen

Wes Kaupinen

President and CEO

Mr. Kaupinen brings more than 17 years of experience as an operating executive and early stage investor.
Mr. Kaupinen brings more than 17 years of entrepreneurial life sciences experience as an operating executive and early stage investor, including having worked closely with several companies that have secured FDA approvals of novel treatments for high unmet indications. Prior to Palvella, Mr. Kaupinen served as Senior Vice President of Corporate Development and Commercialization at Insmed (NASDAQ: INSM), a global commercial stage biopharmaceutical company focused on rare diseases. Previously, Mr. Kaupinen was a Principal at Quaker Partners, a life sciences venture capital firm with $700mm under management, where he worked on several public and private investments, including recruiting new leadership to Insmed to execute on a rare disease strategy. Mr. Kaupinen has served as a board director or observer for more than ten companies, including leading Series A investments in Intact Vascular (backed by NEA, HIG, and Quaker) and TELA Bio (backed by OrbiMed, Quaker, and HIG). Mr. Kaupinen earned an M.B.A. from The Wharton School of the University of Pennsylvania, and a B.A. in Economics from The University of Virginia.

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Braham Shroot, Ph.D.

Chief Scientific Officer

Braham Shroot, Ph.D.

Braham Shroot, Ph.D.

Chief Scientific Officer

Dr. Shroot brings over 30 years of experience in drug research and development specializing in skin disorders, and played a key role in the development of commercially available therapeutics including Differin, Atralin, Vectical, and Diflucan.
Dr. Shroot is the recipient of the American Skin Association’s Lifetime Achievement Award and an honorary lifetime member of the Society for Investigative Dermatology. Prior to Palvella, Dr. Shroot held senior roles in the biopharma industry, including President of Galderma Research, Chief Scientific Officer of Barrier Therapeutics (sold to Stiefel), and CSO/VP of R&D for DFB Pharmaceuticals. Dr. Shroot also served as the chief executive officer of Signum Biosciences, a company developing dermatological therapeutics. Dr. Shroot is an inventor on over 50 patents, author of over 260 publications, and a member of the editorial boards of several prestigious dermatology journals. Dr. Shroot earned a B.Sc. in Chemistry and a Ph.D. in Organic Chemistry from Glasgow University in Scotland.

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Emily Cook

VP of Clinical Operations

Emily Cook

Emily Cook

VP of Clinical Operations

Ms. Cook’s 15-year career spans large and small pharmaceutical companies where she has managed clinical development programs for clinical and commercial stage products.

Prior to Palvella, Ms. Cook served as Director of Clinical Operations for Complexa, a clinical stage biopharmaceutical company focused on life-threatening fibrosis and inflammatory diseases. Prior to joining Complexa, she served as the director of clinical operations for Trevena, Inc., where she contributed to the completion of multiple Phase 1 through Phase 3 clinical trials. Emily also held clinical and sales roles at Applied Clinical Intelligence, Yaupon Therapeutics, and Sanofi-Aventis Pharmaceuticals. Emily received her B.A. in Microbiology from Miami University and earned her Clinical Research Associate certification from the Association of Clinical Research Professionals.

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Kathy Goin

VP of Medical and Patient Engagement

Kathy Goin

Kathy Goin

VP of Medical and Patient Engagement

With a positive and relentless spirit, Ms. Goin builds and coaches high performing and successful clinical development teams.
Since 1998, Ms. Goin served many patient populations by developing drugs and devices to treat different cancers, heart disease/abnormalities, and pain conditions. As Vice President, Clinical Operations at Trevena, Inc., she shaped trial execution strategies across all programs by promoting meaningful and streamlined interactions between sites and sponsor representatives and employing modern processes for lean yet highly effective trial execution. These approaches led to helping the company start and finish three Phase III studies in just under two years. In 2017, she joined colleagues to start Clinical Works, a progressive specialty consulting firm focused on helping emerging and small biotech/pharmaceutical companies start their clinical programs and execute trials. Ms. Goin holds a Master of Science in Occupational Therapy from College Misericordia.

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Kathleen McGowan

Chief Financial Officer

Kathleen McGowan

Kathleen McGowan

Chief Financial Officer

Ms. McGowan brings over 35 years of diversified financial experience in the pharmaceutical and biotech industry.
Before Palvella, Ms. McGowan served as Chief Financial Officer of VaxInnate, a private company developing vaccines for influenza, malaria, and dengue. Prior to VaxInnate, she led financial operations at Tobira Therapeutics (acquired by Allergan plc), a clinical-stage company developing and commercializing therapies for non-alcoholic steatohepatitis (NASH) and other liver diseases. Earlier in her career, Ms. McGowan held financial senior management roles at Discovery Laboratories, Inc. (now Windtree Therapeutics), Claneil Enterprises, Liposome Company (acquired by Elan Corp.), and Johnson & Johnson. She received her B.S. in finance and accounting from Drexel University and holds an M.B.A. in finance from Fairleigh Dickinson University.

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Greg Coulter, Ph.D.

VP of Technical Operations

Greg Coulter, Ph.D.

Greg Coulter, Ph.D.

VP of Technical Operations

Dr. Greg Coulter brings to Palvella deep expertise in drug substance characterization, drug product formulation, scale-up and GMP manufacturing from clinical to commercial stages.
Prior to Palvella, Dr. Coulter served as president of CTM Solutions, a manufacturing and clinical supplies consultancy for early and development stage companies. Prior to CTM Solutions, Dr. Coulter led the Formulations and Analytical Services department for a prominent international contract research organization and managed all GMP manufacturing for a Seattle biopharmaceutical company. Dr. Coulter earned his Bachelor of Science (Hons) in Biochemistry from Bishop’s University in Lennoxville, Canada, followed by a Ph.D. in Biological Chemistry and a Master of Science in Organic Chemistry from the University of Guelph in Guelph, Canada.

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Stephen Yoo, MD

Senior Clinical Advisor

Stephen Yoo, MD

Stephen Yoo, MD

Senior Clinical Advisor

Dr. Yoo brings deep expertise in rare diseases and serious skin disorders from his clinical training and practice as a dermatologist and as a senior executive in drug development.

Dr. Yoo currently serves as chief medical officer of a newly incubated company focusing on allergic mediated diseases while working as an entrepreneur in residence with Atlas Ventures. Previously he was the first CMO of REGENXBIO, a leading publicly traded clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. While at REGENXBIO he helped to advance a pipeline focusing on developing AAV gene therapies for retinal, metabolic and neurodegenerative diseases, many of which were rare. Prior to joining REGENXBIO, Dr. Yoo served as medical science director of clinical development and group director of clinical development at AstraZeneca. In previous roles at MedImmune, he provided strategic clinical leadership for early phase programs. Dr. Yoo led early and late-phase clinical trials for both common and rare autoimmune diseases. Prior to MedImmune, Dr. Yoo served as associate director of clinical development at Abbott Laboratories.

Dr. Yoo earned a B.A. in molecular and cell biology from the University of California, Berkeley and an M.D. from the UCLA School of Medicine. He has served as a member of the board of directors (resident member) for the Society for Investigative Dermatology.

George Jenkins

Chairman

George Jenkins

Chairman

From 1987 to 2005, Mr. Jenkins was a general partner of Apax Partners, a leading global private equity firm where he served as chief operating officer overseeing a multi-billion dollar venture capital and private equity investment portfolio. Since 2006, Mr. Jenkins has been an active private investor primarily funding technology-based start-ups. Mr. Jenkins has been or currently serves as a board member of numerous public and private companies, including SkinMedica (acquired by Allergan), Colorescience, Flower Orthopedics, Power Survey, Sunglass Hut, and Spyder Active Sports. Prior to joining Apax, Mr. Jenkins was a banker managing banking facilities in Europe. Mr. Jenkins earned a B.A. in Economics and Accounting from Lafayette College where he currently serves as a Trustee.

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John Doux, M.D., FAAD

John Doux, M.D., FAAD

Dr. Doux joined Palo Alto Investors, a healthcare-focused investment firm, in 2004 as an analyst. Dr. Doux is a board certified dermatologist who maintained a clinical practice in medical and surgical dermatology for seventeen years. He earned his M.D. from Stanford University, where he was a Howard Hughes Medical Institute Fellow performing and publishing research in the laboratory of Dr. David Woodley. He completed training at Brigham and Women’s Hospital and Stanford Medical Center and earned an M.B.A. at the Wharton School of Business where he was a Palmer Scholar.

Elaine J. Heron, Ph.D.

Elaine J. Heron, Ph.D.

Dr. Heron served as Chair and Chief Executive Officer of Amplyx Pharmaceuticals, a clinical stage drug development company from February 2009 until October 2015, and she continues to serve as board member. She also serves as a board member of BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a therapeutics company with annual sales over $1 billion, and Vala Sciences, a private biotechnology tools company. From July 2001 to October 2008, Dr. Heron was Chair and Chief Executive Officer of Labcyte Inc., a private biotechnology tools company. Before joining Labcyte Inc., she spent six years in positions of increasing responsibility at the Applied Biosystems Group of Applera Corporation, a biotechnology tools company, including the positions of general manager and VP of sales and marketing. Dr. Heron earned a B.S. in Chemistry with highest distinction, a Ph.D. in Analytical Biochemistry from Purdue University, and an M.B.A. from Pepperdine University.

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Todd Davis

Todd Davis

Mr. Davis is a founding Managing Director at HealthCare Royalty Partners, a global healthcare investment firm. He has thirty years of experience working in and investing in the pharmaceutical and biotechnology industries. Over the course of his career, Mr. Davis has been involved in over $2 billion in healthcare deals and nearly $1 billion in royalty financings. He has also led, structured and closed over 40 additional intellectual property licenses, as well as growth equity, and debt deals. Previously, Mr. Davis was a partner at Paul Capital Partners, where he co-managed that firm’s royalty investments as a member of the royalty management committee. He also served as a partner responsible for biopharmaceutical growth equity investments at Apax Partners. Prior to beginning his principal investment career in 2001, Mr. Davis held various sales and product management roles at Abbott Laboratories and worked in business development, operations and licensing at Elan Pharmaceuticals. Mr. Davis holds a B.S. from the U.S. Naval Academy and an M.B.A. from Harvard University. He currently serves on the board of Ligand Pharmaceuticals (NASDAQ: LGND).

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Tim Henkel, M.D., Ph.D.

Tim Henkel, M.D., Ph.D.

Dr. Henkel is the Chief Medical Officer of VenatoRx, a venture-backed company focused on the development of novel anti-infective agents to address antibiotic resistance. Dr. Henkel has spent 20 years in the biopharmaceutical industry successfully developing biologics, small molecules and natural products in the US and Europe across multiple therapeutic areas. Prior to joining VenatoRx, Dr. Henkel was Executive Vice President of R&D at Ceptaris Therapeutics (acquired by Actelion). At Ceptaris, Tim architected the clinical development plan and regulatory strategy for VALCHLOR, a novel topical mechlorethamine gel for the treatment of cutaneous T-cell lymphoma. Dr. Henkel was also Executive Vice President of R&D at Ception Therapeutics (acquired by Cephalon), EVP and Chief Medical Officer of Vicuron Pharmaceuticals (acquired by Pfizer), and Vice President of worldwide anti-infective clinical development for SmithKline Beecham.

Prior to joining SmithKline Beecham, Dr. Henkel was an assistant professor of internal medicine and infectious diseases at Washington University in St. Louis, where he also earned his board certifications in both internal medicine and in infectious diseases. He received his M.D. and a Ph.D. in Immunology from Washington University in St. Louis, and his B.S. in Chemistry and Biology from Rhodes College.

Michael Christopher

Michael Christopher

Mr. Christopher has more than 17 years of private equity and investment banking experience. Prior to joining ARM Energy (formerly Asset Risk Management) in 2015 in the role of chief investment officer and chief financial officer, he spent nearly 13 years at ArcLight Capital Partners where he originated and numerous structured private equity transactions. Mr. Christopher began his career in Citigroup’s Investment Banking Group, where he provided M&A and capital markets advice to public and private companies. He earned a B.S. in Commerce from The University of Virginia.

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Wes Kaupinen

President and CEO

Wes Kaupinen

President and CEO

Mr. Kaupinen brings more than 17 years of experience as an operating executive and early stage investor.
Mr. Kaupinen brings more than 17 years of entrepreneurial life sciences experience as an operating executive and early stage investor, including having worked closely with several companies that have secured FDA approvals of novel treatments for high unmet indications. Prior to Palvella, Mr. Kaupinen served as Senior Vice President of Corporate Development and Commercialization at Insmed (NASDAQ: INSM), a global commercial stage biopharmaceutical company focused on rare diseases. Previously, Mr. Kaupinen was a Principal at Quaker Partners, a life sciences venture capital firm with $700mm under management, where he worked on several public and private investments, including recruiting new leadership to Insmed to execute on a rare disease strategy. Mr. Kaupinen has served as a board director or observer for more than ten companies, including leading Series A investments in Intact Vascular (backed by NEA, HIG, and Quaker) and TELA Bio (backed by OrbiMed, Quaker, and HIG). Mr. Kaupinen earned an M.B.A. from The Wharton School of the University of Pennsylvania, and a B.A. in Economics from The University of Virginia.

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