Puretech Ownership1
Therapeutic Candidate2
Initial Indication(s)
Stage of Development
8.6% Equity
VOR33 (CD33)

Acute myeloid leukemia

Phase 1/2a
Myelodysplastic syndromes, myeloproliferative neoplasms
Preclinical
VCAR33
Bridge-to-transplant AML
Phase 1/2

1 As of June 30, 2021, PureTech’s percentage ownership of Vor was approximately 8.6 percent on an outstanding voting share basis. This calculation includes outstanding shares, options, and warrants, but excludes unallocated shares authorized to be issued pursuant to equity incentive plans.

2 Therapeutic candidates are investigational and have not been cleared by the FDA for use in the United States.

Engineering hematopoietic stem cell therapies combined with targeted therapies
  • Vor is a clinical-stage cell therapy company that combines a novel patient engineering approach with targeted therapies to provide a single company solution for patients suffering from hematological malignancies. The only way for many of these patients to achieve durable remission or a cure is through hematopoietic stem cell transplant (HSCT). Despite this, approximately 40 percent of acute myeloid leukemia (AML) patients relapse following such transplant and face a prognosis with a two-year survival rate of less than 20 percent. Targeted therapies are an effective treatment for many patients in transplant settings who relapse, though they are limited by toxicities resulting from the expression of the surface targets on healthy cells, including these new transplanted cells, which is referred to as on-target toxicity.

 

 

  • Vor’s proprietary platform leverages its expertise in hematopoietic stem cell (HSC) biology and genome engineering to remove surface targets expressed by cancer cells by genetically modifying HSCs. By removing these targets, Vor makes these HSCs and their progeny treatment resistant to targeted therapies and enables these treatments to selectively destroy cancerous cells while sparing healthy cells. As a result, Vor’s engineered HSCs (eHSCs) are designed to limit the on-target toxicities associated with these targeted therapies, or companion therapeutics, thereby enhancing their utility and broadening their applicability.
  • Vor’s platform and expertise allow it to advance its goal of replacing the patient’s HSCs with next-generation, treatment-resistant eHSCs that unlock the potential of highly-potent targeted therapies.
  • Program Discovery Process by the PureTech Team
    • We were interested in approaches to treat hematological malignancies that currently have poor response rates or poor adverse event profiles despite recent advances in cell therapies and targeted therapies. We engaged leading hematological cancer specialists and we became aware of work from the laboratory of Vor Scientific Board Chair Siddhartha Mukherjee, M.D., Ph.D., Assistant Professor of Medicine at Columbia University and Pulitzer Prize-winning author of The Emperor of All Maladies: A Biography of Cancer. Dr. Mukherjee pioneered the idea of genetically engineering stem cells to eliminate a particular target such that healthy stem cells and progeny cells would be spared from targeted cancer therapy. We worked with Dr. Mukherjee on this intellectual property, which Vor exclusively in-licensed from Columbia in April 2016, and on advancing this concept through critical POC experiments. With our support, Vor secured additional intellectual property rights (both in-licensed from Columbia and owned by Vor), assembled an excellent research team and completed a round of fundraising.
    • In July 2019, Bill Lundberg, M.D., was appointed to Vor’s Board of Directors. In August 2019, Robert Ang, MBBS, MBA, was appointed President and Chief Executive Officer of Vor. In May 2020, Vor announced the appointment of Nathan Jorgensen, Ph.D., as Chief Financial Officer. In July 2020, Vor announced the closing of a $110 million Series B financing and the appointments of Daniella Beckman and David Lubner to its Board of Directors and Christopher Slapak, M.D., as Chief Medical Officer. In August 2020, Vor announced the appointment of John King as Chief Commercial Officer, and in October 2020 Vor announced the appointment of Matthew Patterson to its Board of Directors.
  • Patient Need & Market Potential
    • The prognosis for relapsed and refractory blood-borne malignancies is very poor and can be measured in a few months, depending on patient-specific risk factors. There are an estimated 42,500 new diagnoses of AML each year in the United States, Europe and Japan. The median five-year survival rate for patients with AML is less than 30 percent, but there are significant differences in prognosis depending on several factors, including the age of the patient at diagnosis.
    • Targeted therapies, such as CAR-T cells and bispecific antibodies, antibody-drug conjugates and conventional mAbs, have shown clinical activity, particularly in patients with certain hematologic malignancies expressing B cell markers. However, these targeted therapies frequently target both cancer and normal cells, causing substantial toxicities and limiting their potential. There is a need for new strategies that can enable selectively targeting cancer cells with limited impact on a patient’s normal cells.
  • Milestones Achieved & Developmental Status
    • In November 2021, Vor announced its first multi-targeted Treatment System comprising VOR33-CLL1 multiplex-edited eHSC therapy and VCAR33-CLL1 multi-specific CAR-T therapy. Vor continues to make progress on editing multiple antigens with its eHSC platform. These multiplex-edited eHSCs will allow Vor to push the frontiers of CAR-T therapies. Knocking out CD123 and CLL-1 through gene editing offers a promising new approach to treating patients with AML using Vor’s novel eHSC platform. The Company’s research demonstrates that multiplex genome editing of allogeneic hematopoietic stem cells may represent another exciting strategy to efficiently and safely edit multiple genes in blood stem cells, allowing the potential use of multi-targeted blood cancer therapies.
    • In November 2021, Vor announced it has expanded its existing license agreement with Columbia University with the addition of an exclusive license to certain base editing technologies.
    • In September 2021, Vor announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to VOR33 for the treatment of AML allowing for potential facilitated development and expedited review process.
    • In September 2021, Vor announced it is actively enrolling in its Phase 1/2a clinical trial for AML patients who currently have limited treatment options.
    • In July 2021, Vor announced the formation of a collaboration with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop eHSC transplants combined with a bi-specific antibody therapy for AML. The agreement was facilitated by Johnson & Johnson Innovation. Under the terms of the collaboration, Vor will investigate the combination of these two technologies into a treatment solution, pairing Vor's "invisible" eHSC transplant platform with one of Janssen's bi-specific antibodies in development for AML. The collaboration agreement provides that each company retains all rights and ownership to their respective programs and platforms.
    • VOR33 is Vor’s eHSC therapeutic candidate designed to transform the standard of care in AML and potentially other myeloid malignancies. To create VOR33, Vor genetically modifies donor HSCs in order to remove the CD33 surface target that is highly expressed in most AML cells. In preclinical studies, Vor observed that the removal of CD33 had no deleterious effects on the differentiation or function of hematopoietic cells, but it provided robust protection of the healthy donor HSCs from the cytotoxic effects of CD33-directed companion therapeutics. Vor intends to develop VOR33 as an HSC transplant therapeutic candidate to replace the standard of care in transplant settings. Once the VOR33 cells have engrafted, patients can potentially be treated with anti-CD33 therapies, such as Mylotarg or a CAR-T therapy therapeutic candidate, with limited on-target toxicity. The combination of VOR33 and CD33-directed therapies has the potential to lead to durable anti-tumor activity.
    • In February 2021, Vor announced the successful closing of its initial public offering of common stock on the Nasdaq Global Market under the symbol “VOR.” The aggregate gross proceeds to Vor from the offering, before deducting the underwriting discounts and commissions and other offering expenses payable by Vor, were approximately $203.4 million.

     

     

    • In January 2021, Vor announced that the FDA had accepted the company’s IND application for VOR33. In May 2021, Vor announced that it received the Canadian clinical trial application (CTA) clearance for VOR33 from Health Canada. 
    • In November 2020, Vor announced an exclusive licensing agreement with the NCI, part of the NIH, for intellectual property related to a clinical-stage anti-CD33 CAR-T therapy candidate, VCAR33. VCAR33 is a CD33-directed CAR-T therapy that Vor intends to initially develop as a bridge-to-transplant monotherapy for relapsed/refractory AML where patients have failed prior lines of therapy and need further treatment to achieve morphologic remission and, if possible, subsequent HSCT. VCAR33 is currently being evaluated in a multi-site, investigator-initiated Phase 1/2 clinical trial in young adult and pediatric patients with relapsed/refractory AML sponsored and overseen by the National Marrow Donor Program, or NMDP. If this trial is successful, Vor expects to continue development of VCAR33 both as a monotherapy treatment for relapsed/refractory AML in the bridge-to-transplant setting and in combination with VOR33 as part of the VOR33/VCAR33 Treatment System in the post-HSCT setting.
    • Vor intends to investigate the VOR33/VCAR33 Treatment System, entailing VOR33 eHSC therapy followed by VCAR33 as a companion therapeutic, initially for transplant-eligible patients suffering from AML. Vor believes VCAR33 could be a potent anticancer therapy that, when combined with VOR33, could help obviate severe on-target myeloablative toxicities and unlock the efficacy potential of VCAR33.
    • Leveraging its proprietary platform, Vor has identified additional surface targets as well as multiple genome engineering approaches. Additionally, Vor is conducting ongoing discovery efforts on undisclosed targets for non-myeloid malignancies. PureTech does not control the clinical or regulatory development of Vor’s therapeutic candidates.
  • Expected Milestones
    • In November 2021, Vor announced it remains on track to report initial clinical data from the Phase 1/2a clinical trial of VOR33 for patients with AML in the first half of 2022.
    • Vor expects initial VCAR33 monotherapy clinical data in 2022, depending on investigator’s timing of data release.
    • Vor expects its in-house manufacturing facility at the Cambridge, MA headquarters will be operational in 2022.
    • Vor expects to file an IND for the VOR33/VCAR33 Treatment System in the second half of 2022.

Vor has an active IND on file with the FDA for VOR33 and an active IND is on file for VCAR33. PureTech does not have a direct interest in Vor’s therapeutic candidates or its proprietary platform. PureTech’s interest in Vor’s therapeutic candidates and proprietary platforms is limited to its non-controlling equity interest in Vor and any potential appreciation in the value of such equity interest and PureTech does not control the clinical or regulatory development of Vor’s therapeutic candidates. Vor is well-protected with a robust intellectual property portfolio. Vor was incorporated in December 2015.

Vor’s proprietary platform leverages its expertise in HSC biology and genome engineering to remove surface targets expressed by cancer cells by genetically modifying HSCs. This allows Vor to advance its goal of replacing the patient’s HSCs with next-generation, treatment-resistant eHSCs that unlock the potential of highly-potent targeted therapies. Vor intends to develop VOR33 as an HSC transplant therapeutic candidate to replace the standard of care in transplant settings. Once the VOR33 cells have engrafted, patients can potentially be treated with anti-CD33 therapies, such as Mylotarg or a CAR-T therapy therapeutic candidate, with limited on-target toxicity. The combination of VOR33 and CD33-directed therapies has the potential to lead to durable anti-tumor activity.


Press Releases

PureTech Founded Entity Vor Biopharma Announces VOR33 Granted U.S. FDA Fast Track Designation for Acute Myeloid Leukemia

August 10, 2021


PureTech Founded Entity Vor Biopharma Announces Collaboration with Janssen to Develop Engineered Hematopoietic Stem Cell Transplants Combined with a Bi-Specific Antibody Therapy for Acute Myeloid Leukemia (AML)

July 9, 2021


PureTech Founded Entity Vor Biopharma Closes Over $200M Initial Public Offering

February 10, 2021


PureTech Founded Entity Vor Biopharma Announces Pricing of Initial Public Offering

February 5, 2021


PureTech Founded Entity Vor Files Public Registration Statement for Proposed Initial Public Offering

January 18, 2021


PureTech Founded Entity Vor Announces FDA Clearance of IND Application for VOR33

January 14, 2021


PureTech Founded Entity Vor Biopharma Announces Exclusive License of Clinical-Stage CD33 CAR-T from National Cancer Institute

November 11, 2020


PureTech Founded Entity Vor Biopharma Appoints Matthew R Patterson to its Board of Directors

October 20, 2020


PureTech Founded Entity Vor Biopharma Appoints John King as Chief Commercial Officer

August 26, 2020


PureTech Founded Entity Vor Biopharma Appoints David Lubner to its Board of Directors

July 30, 2020


PureTech Founded Entity Vor Biopharma Appoints Daniella Beckman to its Board of Directors

July 16, 2020


PureTech Founded Entity Vor Biopharma Appoints Dr Christopher Slapak as Chief Medical Officer

July 14, 2020


PureTech Founded Entity Vor Biopharma Raises $110 Million in Series B Financing

July 7, 2020


PureTech Founded Entity Vor Biopharma Appoints Nathan Jorgensen, PhD, as Chief Financial Officer

May 26, 2020


PureTech Affiliate Vor Biopharma Appoints Senior Cell and Gene Therapy Leaders

October 1, 2019


PureTech Affiliate Vor Biopharma Appoints Robert Ang, MBBS, MBA, as President and Chief Executive Officer

August 8, 2019


PureTech Health Affiliate Vor Biopharma Publishes Proof-of-Concept Study of its Lead Programme in the Scientific Journal Proceedings of the National Academy of Sciences

May 29, 2019


PureTech Health Affiliate Vor Biopharma Announces $42 Million Series A Financing Round to Advance Cell Therapy Product Candidate for the Treatment of Acute Myeloid Leukaemia

February 14, 2019