HepaTx Corp.

Palo Alto, CA
Revolutionary stem cell-based therapies for liver disease
  • Liver failure is an underserved $10B+ market
  • Developed scalable, off-the-shelf cell therapy
  • Founders have 40+ years experience in drug dev.

Bioverge Participation:

Seed+ (SAFE)
Biotech
Liver Disease
Cell Therapy
Regenerative Medicine

OPPORTUNITY OVERVIEW

HepaTx is developing an allogeneic stem cell-based therapy as an alternative to liver transplant.  Their proprietary SF-Heps cells are terminally differentiated adipocyte-derived stem cells.  These cells can address multiple liver disease pathophysiologies through a variety of mechanisms of action.  HepaTx’s proprietary SF-Heps are based on a 3-part differentiation process with IP licensed from Stanford.  Their lead indication is acute liver failure, which remains a high unmet medical need.

Company Highlights

  • Liver failure is an underserved $10 billion+ market.  Only liver transplants are curative, but less than 3% of people receive one
  • HepaTx's cell therapy platforms efficiently converts fat stem cells to liver cells.  This proprietary, patented process has been exclusively licensed from Stanford. It's scalable, efficient, can be used on-demand, and is applicable to multiple liver diseases.
  • Founders have 40+ years experience in drug development and bringing products to market.  Co-founder Mark Chao, MD, PhD was a pervious Co-founder of Forty Seven, Inc. which IPO'd in 2018 ($500M valuation) and was acquired by Gilead for $4.9 billion in March 2020. 
  • Go-to-market strategy combines innovative manufacturing and regulatory approaches to shorten the time and cost needed to reach market
  • Existing/co-investors include: Grey Sky Venture Partners, Y Combinator, iSelect Fund, Stanford StartX Fund, iSprout Ventures, Savantus Ventures, Redplate Capital, and Palapa Ventures.





Investment Thesis

Regenerative medicine is a broad field that includes tissue engineering but also incorporates research on self-healing – where the body uses its own systems, sometimes with help foreign biological material to recreate cells and rebuild tissues and organs. A major goal for regenerative medicine is to enable human tissue replacement through transplantation of stem cells that can be harvested from readily accessible tissues. 

HepaTx's vision is to develop regenerative medicine solutions for complex chronic diseases that have not been addressed well by conventional drug therapy.  The Company's approach is uniquely differentiated in its ability to generate a novel, scalable, off-the-shelf and effective cell therapy approach by converting donor fat stem cells into functioning hepatocytes for patient use. Large numbers of donor fat cells - adipocyte-derived stem cells (ASCs) - can be easily obtained by liposuction and HepaTx has developed methods for inducing ASC differentiation into functioning hepatocyte-like cells that HepTx calls SF-Heps. These features make liver regeneration via transplantation of ASC-derived hepatocytes a highly attractive possibility for regenerative medicine. 

The Company’s platform consists of proprietary methods for logically and methodically modifying the phenotype of stem cells and directing their differentiation into virtually any type of cell.  The output of this platform is clinical grade cell therapies.  SF-Heps are Hepatx’s first product from this platform. Since many liver diseases share a similar pathogenesis in terms of decline in liver function/cirrhosis, the SF-Hep cell therapy product can address multiple liver diseases with the same product.  

HepaTx has achieved early preclinical proof-of-concept, demonstrating their process can produce hepatocyte-like cells.  They have further shown in vivo that these cells stably engraft in the liver, produce key liver synthetic function, and also positively modulate liver function in a liver damage prevention and treatment model.  


Problem

While liver transplants are curative and many would benefit from a transplant, less than 3% of patients receive one.  The market opportunity for HepaTx’s cells includes not only the 15,000 people who are registered on the waiting list for a liver transplant, but an additional ~200,000 people who have later stage disease and are not currently on the waiting list.  

Solution

HepaTx’s solution is to develop a novel, scalable, off-the-shelf and effective cell therapy treatment for liver disease by converting donor fat stem cells into functionally differentiated hepatocytes.  The cells would be infused via the portal vein for engraftment into the liver.  The cell’s mechanism of action would include functionally engrafting into the liver to replace lost liver function while also reducing injury/inflammation at the site of via paracrine signaling.  This solution would enable hepatocyte transplantation utilizing a virtually unlimited supply of donor cells.  


HepaTx's first program will be treating a specific indication for patients with acute liver disease given the high unmet need, speed to data readouts, and ability to achieve regulatory approval with a faster and more efficient path given the orphan nature of the disease.  After clinical proof of concept is established, HepaTx will be evaluating the cell therapy in larger indications including chronic liver diseases such as NASH and cirrhosis.   

Over the long term, the Company envisions developing additional allogeneic cell therapy products for indications outside of the liver disease space including regenerative cells from the pancreas, kidney and other organs through expansion of our technological know-how and pre-clinical platforms. 


HepaTx' currently manufactures hepatocytes in their facility. The initial production process from the platform is dialed-in and ready for GMP transition. The Company's next milestone is to show preclinical proof-of-concept in a liver disease model that is relevant to human disease which will enable proceeding to IND-enabling studies. That work is currently in progress.

Market Overview

The global liver disease market opportunity was $14.5 billion in 2019 and is expected to grow to $27.6 billion by 2025, a CAGR of 11.5%. The U.S. market for SF-Heps is estimated to be $10B+:


Examples of M&A, IPOs, or licensing deals in the space

  • Semma Therapeutics was acquired by Vertex for $950M cash. (2020, https://www.fiercebiotech.com/biotech/vertex-plunks-down-950m-for-stem-cell-player-semma-therapeutics)
  • Fate Therapeutics licensing deal with Janssen for $50M upfront and $50M equity investment for stem cell derived NK cells for cancer treatment. (2020, https://ir.fatetherapeutics.com/news-releases/news-release-details/fate-therapeutics-announces-worldwide-collaboration-janssen)
  • Mesoblast licensing deal with Grunenthal for $150M upfront for a cell therapy for back pain. (2019, https://www.globenewswire.com/news-release/2019/09/09/1913118/0/en/Gr%C3%BCnenthal-and-Mesoblast-Enter-Strategic-Partnership-for-Europe-and-Latin-America-to-Develop-and-Commercialise-Innovative-Cell-Therapy-for-the-Treatment-of-Chronic-Low-Back-Pain.html)
  • Tigenix SA was acquired by Takeda Pharmaceuticals for $400M. (2016, https://www.takeda.com/newsroom/newsreleases/2018/takeda-announces-intention-to-acquire-tigenix/)

Two companies have marketed cell therapy products produced from the same stem cells.  

  • Mesoblast (Melbourne, Australia) has developed a cell therapy for pediatric graft versus host disease that is approved in Japan and under review in the US.
  • Tigenix (acquired by Takeda) has developed a cell therapy for Chron’s disease that is approved in Europe and under review in the US.



TEAM

Thumb screen shot 2020 10 21 at 8.47.17 am
Eric Schuur, PhD
Founder, President & CEO
  • Entrepreneur, molecular biologist with experience in biopharma and medical devices. Participation and leadership across the entire range of research from basic to clinical
  • Formerly Director of Clinical Affairs, Asthmatx, Inc. where he was responsible for clinical development and data analysis. Clinical study strategy, design, and operations. Management and analysis of clinical data. Developed and executed reporting strategies on clinical trial results for regulatory and marketing departments. Managed publication process.
  • Formerly President and Founder of GeneSource, Inc. Biotechnology R&D and business consulting for biotechnology companies. Development of high content genomic assays using novel technology. Consulting focused on product development, technology assessment, and business development.
  • Visiting Scholar, Stanford.  Research position in the Department of Surgery. Molecular mechanisms of oncogenesis in breast cancer; regulation of expression of the estrogen receptor alpha. Mentoring students and staff
Thumb screen shot 2020 10 21 at 8.49.12 am
Mark Chao, MD, PhD
Founder, Board Member
  • Vice President, Oncology Clinical Research at Gilead Sciences and Co-Founder, Forty Seven, Inc (sold to Gilead for $4.9B)
  • Stanford Health Care, Clinical Instructor and Hematology Fellow
  • Stanford University - worked in the lab of Drs. Irving Weissman and Ravindra Majeti, investigated antibody therapies targeting leukemia stem cells, specifically targeting of CD47, an anti-phagocytic protein expressed on many human cancers
  • Identified CD47 as a prognostic predictor and therapeutic antibody target in multiple human cancers
  • Several first author publications in the journals of Cell, Science Translational Medicine, Blood, Cancer Research; three patents, approved Investigational New Drug Application (IND) filing and Phase I trial in progress for anti-CD47 antibody therapy in hematologic and solid tumor malignancies
Thumb screen shot 2020 10 21 at 10.32.08 am
Gary Peltz, MD, PhD
Advisor
  • Professor, Anesthesiology, Perioperative and Pain Medicine
  • Member, Bio-X
  • Member, Maternal & Child Health Research Institute (MCHRI)
  • Honors & Awards: Transformative RO1 award, NIH/NIDDK (2010); One of 10 top pharmaceutical research executives, Nature Publications (2006)
  • M.D., Stanford University, Medicine (1983)
  • Ph.D., Stanford University, Structural Biology (1983)
  • Fellowship, University of California-San Francisco, Rheumatology (1989)
  • Residency, University of California-San Francisco, Internal Medicine (1985)
  • Board Certification, American Board of Internal Medicine, Internal Medicine, Rheumatology (1989)
Thumb screen shot 2020 10 21 at 10.35.29 am
Marie Csete, MD, PhD
Advisor
  • Board certifications in Anesthesiology & Critical Care Medicine
  • Specialties include perioperative care of liver transplant patients: Human embryonic stem cells, translation of stem cell therapies, Standards and accreditation of stem cell therapy facilities 
  • M.D., Columbia University (1979)
  • Ph.D., Cal Tech, Molecular and Developmental Biology (2000)
Thumb screen shot 2020 10 21 at 10.36.58 am
Shannon Dahl, PhD
Advisor
  • 2018 PharmaVOICE 100 transformational, influential, and inspirational leader.
  • Expertise in cell and gene therapy, biologics, engineered tissues.
  • Innovator who founded and advanced a "CNBC Disruptor 50" Regenerative Medicine company.
  • Deep, broad perspective spanning technology, product, reimbursement, regulatory, market development, corporate strategy, corporate development, and patents.
  • Inventor on 36 issued patents, authored 21 publications, co-authored first ever Regenerative Medicine Advanced Therapy designation granted by FDA, invited speaker at conferences.
  • Ph.D., Duke University, Biomedical Engineering (2004)
I Can Help