News Release Details

Proceeds will advance the company’s lead programme into clinical trials and expand the portfolio

Financing led by RA Capital Management, with Participation from Fidelity Management & Research Company, LLC, Pagliuca Family Office, Alexandria Venture Investments, 5AM Ventures, Johnson & Johnson Innovation — JJDC, Inc. (JJDC) and Osage University Partners

PureTech Health plc (LSE: PRTC) (“PureTech” or the “Company”), a clinical-stage biotherapeutics company dedicated to discovering, developing and commercialising highly differentiated medicines for devastating diseases, is pleased to note that its Founded Entity, Vor Biopharma, has raised $110 million in a Series B financing. Proceeds will advance the company’s lead candidate VOR33 into clinical trials, deepen its portfolio and accelerate the validation of additional targets for its scientific platform, which is designed to remove redundant proteins so that transplanted stem cells become invisible to targeted therapies while leaving diseased cells vulnerable. Vor’s treatment strategy has the potential to bring a revolutionary treatment paradigm for patients with acute myeloid leukaemia and other haematologic malignancies.

Founded by PureTech and Siddhartha Mukherjee, MD, DPhil, Vor is developing engineered haematopoietic stem cell (eHSC) therapies for the treatment of cancer.

Bharatt Chowrira, JD, PhD, PureTech’s president and chief of business and strategy, said: “Vor is breaking new ground with its eHSCs therapies for treating cancer, which are designed to hide healthy cells from targeted therapies while leaving diseased cancer cells vulnerable to attack. This financing with top tier investors is a strong testament to the strength of this scientific platform and its potential to make a difference for cancer patients.”

The full text of the announcement from Vor is as follows:

Vor Biopharma Closes $110 Million Series B Financing

Proceeds will advance the company’s lead programme into clinical trials and expand the portfolio

CAMBRIDGE, Mass., – July 7, 2020 – Vor Biopharma, an oncology company pioneering engineered haematopoietic stem cells (eHSCs) for the treatment of cancer, today announced it has raised $110 million in a Series B financing. Proceeds will advance the company’s lead candidate VOR33 into clinical trials, deepen its portfolio, and accelerate the validation of additional targets for its scientific platform, which is designed to remove redundant proteins so that transplanted stem cells become invisible to targeted therapies while leaving diseased cells vulnerable. Vor’s treatment strategy has the potential to bring a revolutionary treatment paradigm for patients with acute myeloid leukaemia and other haematologic malignancies.

“Vor has an elegant approach to engineering haematopoietic stem cells that we believe is amongst the most promising innovations in oncology,” said Joshua Resnick, MD, Managing Director at RA Capital Management. “We are proud to support the efforts of their impressive team of experienced leaders and drug developers as they work aggressively to establish a new standard of care in stem cell transplants and forge ahead into first-in-human clinical studies.”

RA Capital Management led the Series B financing, along with a diverse group of well-respected new investors: Fidelity Management & Research Company, LLC, Pagliuca Family Office, Alexandria Venture Investments, and other undisclosed investors, including additional institutional crossover investors. Existing investors 5AM Ventures, Johnson & Johnson Innovation — JJDC, Inc. (JJDC), Osage University Partners, and co-founder PureTech Health participated in the financing.

“The high caliber of investors participating in this financing underscores the tremendous potential of our eHSC platform,” said Robert Ang, MBBS, MBA, Vor’s President and Chief Executive Officer. “We have ambitious goals for the coming year, and this financing is an important step as we prepare to treat cancer patients in our first clinical trials.”

About Vor Biopharma

Vor Biopharma aims to transform the lives of cancer patients by pioneering engineered haematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumour cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.

Vor’s platform could be used to potentially change the treatment paradigm of both haematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. The results of a proof-of-concept preclinical study for Vor’s lead programme has been published in Proceedings of the National Academy of Sciences.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil.

About VOR33

Vor’s lead product candidate, VOR33, consists of engineered haematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, we believe that CD33 will become a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from acute myeloid leukaemia.

About PureTech Health

PureTech is a clinical-stage biotherapeutics company dedicated to discovering, developing and commercialising highly differentiated medicines for devastating diseases, including intractable cancers, lymphatic and gastrointestinal diseases, central nervous system disorders and inflammatory and immunological diseases, among others. The Company has created a broad and deep pipeline through the expertise of its experienced research and development team and its extensive network of scientists, clinicians and industry leaders. This pipeline, which is being advanced both internally and through PureTech’s Founded Entities, is comprised of 24 products and product candidates, including two that have been cleared by the US Food and Drug Administration (FDA). All of the underlying programmes and platforms that resulted in this pipeline of product candidates were initially identified or discovered and then advanced by the PureTech team through key validation points based on the Company’s unique insights into the biology of the brain, immune and gut, or BIG, systems and the interface between those systems, referred to as the BIG Axis.

For more information, visit www.puretechhealth.com or connect with us on Twitter @puretechh.

Ownership Information

PureTech's percentage ownership of Vor following the financing is approximately 11.8% on a diluted basis. This calculation of PureTech's holding includes issued and outstanding shares as well as options and warrants to purchase shares, but excludes unallocated shares authorised to be issued pursuant to equity incentive plans.

Forward Looking Statement

This press release contains statements that are or may be forward-looking statements, including statements that relate to the company's future prospects, developments, and strategies. The forward looking statements are based on current expectations and are subject to known and unknown risks and uncertainties that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks and uncertainties described in the risk factors included in the regulatory filings for PureTech Health plc. These forward-looking statements are based on assumptions regarding the present and future business strategies of the company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, neither the company nor any other party intends to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise.