Vor(VOR)

Search documents
Vor(VOR) - 2021 Q4 - Annual Report
2022-03-14 20:13
[PART I](index=6&type=section&id=PART%20I) This section details the company's business operations, strategic initiatives, associated risks, and general corporate information [Business](index=6&type=section&id=Item%201.%20Business) Vor Biopharma is a clinical-stage cell and genome engineering company focused on treating blood cancers, developing eHSCs and CAR-T therapies [Overview and Strategy](index=6&type=section&id=Overview%20and%20Strategy) Vor Bio is a clinical-stage company engineering hematopoietic stem cells for blood cancers, focusing on AML - Vor Bio is a clinical-stage company engineering hematopoietic stem cells (HSCs) to enable the use of targeted therapies post-transplant for blood cancers, with an initial focus on Acute Myeloid Leukemia (AML)[14](index=14&type=chunk)[15](index=15&type=chunk) - The company's core strategy includes demonstrating the safety and efficacy of its lead product VOR33, advancing its in-house CAR-T therapies (VCAR33), developing 'Treatment Systems' that combine eHSCs and CAR-Ts, and establishing in-house cGMP manufacturing[37](index=37&type=chunk)[41](index=41&type=chunk) - The proprietary platform aims to overcome the on-target toxicity of traditional cancer therapies by genetically modifying healthy donor HSCs to remove cell surface targets, thereby protecting them from targeted treatments[17](index=17&type=chunk)[42](index=42&type=chunk) [Pipeline and Programs](index=13&type=section&id=Pipeline%20and%20Programs) The company's pipeline includes lead candidate VOR33 in Phase 1/2a for AML, and VCAR33 CAR-T programs Product Pipeline and Anticipated Milestones | Program | Modality | Indication | Stage | Anticipated Milestones | | :--- | :--- | :--- | :--- | :--- | | **VOR33 + Mylotarg** | eHSC + ADC | AML, MDS, MPN | Phase 1/2 | 2H 2022: Initial clinical data | | **VCAR33ALLO** | CAR-T (Allogeneic) | AML Post-transplant | Preclinical | 1H 2023 IND submission | | **VCAR33AUTO** | CAR-T (Autologous) | Bridge-to-transplant AML | Phase 1/2 (NMDP-sponsored) | 2022: Initial monotherapy clinical proof-of-concept data | | **VOR33 + VCAR33 Treatment System** | eHSC + CAR-T | AML | Preclinical | IND filing following initial VOR33 and VCARALLO data | | **VOR33-CLL1 + VCAR33-CLL1 Treatment System** | Multiplex-edited eHSC + Multi-specific CAR-T | AML | Discovery/Validation | - | - The lead product, VOR33, is an eHSC where the CD33 surface target is removed. It is being evaluated in the VBP101 Phase 1/2a clinical trial for AML patients at high risk of relapse, with initial data expected in the second half of 2022[51](index=51&type=chunk)[74](index=74&type=chunk) - The VCAR33 program consists of two CAR-T therapies targeting CD33: VCAR33AUTO (autologous cells, in an ongoing NMDP-sponsored trial) and VCAR33ALLO (allogeneic cells, IND submission planned for 1H 2023)[86](index=86&type=chunk)[89](index=89&type=chunk) - The company is developing a 'Treatment System' combining VOR33 with VCAR33ALLO, using the same healthy donor cell source for both products to potentially improve persistence and reduce toxicity[97](index=97&type=chunk)[98](index=98&type=chunk) - Discovery efforts are underway for additional targets (CD123, EMR2, CD5) and multiplex engineering to address tumor heterogeneity and escape mechanisms, with the VOR33-CLL1 + VCAR33-CLL1 Treatment System as the first multiplex program[101](index=101&type=chunk)[104](index=104&type=chunk)[111](index=111&type=chunk) [Manufacturing, Commercialization, and Competition](index=28&type=section&id=Manufacturing,%20Commercialization,%20and%20Competition) The company is establishing in-house manufacturing, targeting AML transplant markets, and navigating competition - The company is building an in-house cGMP clinical manufacturing facility in Cambridge, MA, expected to be operational in 2022 to support its eHSC and CAR-T pipeline[135](index=135&type=chunk)[49](index=49&type=chunk) - The commercial strategy targets the approximately **12,000 annual allogeneic HSCTs** performed globally for AML, focusing on the concentrated network of specialized transplant centers[115](index=115&type=chunk)[116](index=116&type=chunk) - Key competitors include companies developing gene-engineered HSCs (Tmunity Therapeutics), CD33-directed therapies (Johnson & Johnson, Amgen, 2seventy bio), and various CAR-T therapies for AML[137](index=137&type=chunk)[138](index=138&type=chunk)[139](index=139&type=chunk) [Intellectual Property and Licensing](index=33&type=section&id=Intellectual%20Property%20and%20Licensing) The company holds exclusive licenses from Columbia University and NIH for its core eHSC and CAR-T intellectual property - The company holds an exclusive worldwide license from Columbia University for patents related to engineering lineage-specific cell surface antigens like CD33 in HSCs, which is central to the VOR33 program[122](index=122&type=chunk)[147](index=147&type=chunk) - An exclusive worldwide license was obtained from the National Institutes of Health (NIH) for intellectual property related to CAR therapies targeting CD33, which underpins the VCAR33 program[126](index=126&type=chunk)[155](index=155&type=chunk) - As of February 28, 2022, the company's owned patent portfolio includes approximately **88 pending U.S. and foreign patent applications**, **22 pending U.S. provisional applications**, and **one granted U.S. patent**. The licensed portfolio includes **seven granted patents** and approximately **46 pending applications**[146](index=146&type=chunk) [Government Regulation](index=36&type=section&id=Government%20Regulation) The company's cell product candidates are regulated as biologics by the FDA, requiring BLA approval and subject to healthcare laws - The company's cell product candidates are regulated as biologics by the FDA and will require the submission and approval of a Biologics License Application (BLA) for marketing authorization[163](index=163&type=chunk) - VOR33 has received Fast Track designation and Orphan Drug Designation (ODD) from the FDA for the treatment of AML, which may facilitate development and provide market exclusivity upon approval[73](index=73&type=chunk)[186](index=186&type=chunk) - The company is subject to extensive U.S. healthcare laws, including the federal Anti-Kickback Statute, False Claims Act, and HIPAA, which regulate business practices, marketing, and data privacy[208](index=208&type=chunk)[209](index=209&type=chunk)[211](index=211&type=chunk) - The regulatory landscape for genome engineering and gene therapy is novel and evolving, which creates uncertainty regarding the time, cost, and requirements for obtaining regulatory approval[369](index=369&type=chunk)[370](index=370&type=chunk) [Risk Factors](index=52&type=section&id=Item%201A.%20Risk%20Factors) The company faces substantial risks including net losses, unproven technology, clinical trial safety, manufacturing, and regulatory uncertainties - The company has a history of significant net losses (**$68.9 million in 2021**) and expects to incur losses for the foreseeable future, requiring substantial additional funding to advance its programs[250](index=250&type=chunk)[254](index=254&type=chunk) - Engineered hematopoietic stem cells (eHSCs) are a novel, unproven technology, and the company's success is highly dependent on its two most advanced candidates, VOR33 and VCAR33[273](index=273&type=chunk)[277](index=277&type=chunk) - There is a significant risk of serious adverse events or undesirable side effects from the company's product candidates, the genome engineering process (e.g., off-target effects), or the associated HSCT and CAR-T procedures (e.g., cytokine release syndrome)[282](index=282&type=chunk)[284](index=284&type=chunk)[288](index=288&type=chunk) - The company relies on third parties for manufacturing materials and conducting clinical trials, and is highly dependent on intellectual property licensed from Columbia University and the NIH, the termination of which would harm the business[397](index=397&type=chunk)[411](index=411&type=chunk) - The regulatory landscape for genome engineering technology is novel and uncertain, which could lead to unpredictable timelines, costs, and challenges in obtaining approval for product candidates[369](index=369&type=chunk) - The COVID-19 pandemic has caused and may continue to cause disruptions, including delays in clinical trial site activation and patient enrollment for the VOR33 Phase 1/2a trial[503](index=503&type=chunk)[505](index=505&type=chunk) [Unresolved Staff Comments](index=111&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments) The company reports that it has no unresolved staff comments from the SEC - Not applicable; the company has no unresolved comments from the SEC staff[547](index=547&type=chunk) [Properties](index=111&type=section&id=Item%202.%20Properties) The company's principal executive office, laboratory, and manufacturing space is located in Cambridge, Massachusetts - The company leases approximately **73,235 square feet** of office, lab, and manufacturing space in Cambridge, Massachusetts, under a lease that expires in August 2030[548](index=548&type=chunk) [Legal Proceedings](index=111&type=section&id=Item%203.%20Legal%20Proceedings) The company is not currently subject to any material legal proceedings - The company is not currently a party to any material legal proceedings[549](index=549&type=chunk) [Mine Safety Disclosures](index=111&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This section is not applicable to the company - Not applicable[550](index=550&type=chunk) [PART II](index=112&type=section&id=PART%20II) This section covers the company's stock market information, financial performance analysis, and internal controls [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=112&type=section&id=Item%205.%20Market%20for%20Registrant's%20Common%20Equity,%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company's common stock trades on Nasdaq since February 2021, with no dividends paid and IPO proceeds unutilized - The company's common stock trades on the Nasdaq Global Select Market under the symbol 'VOR', commencing on February 5, 2021[553](index=553&type=chunk) - The company has never declared or paid cash dividends and does not anticipate doing so in the foreseeable future, intending to retain earnings for business development[555](index=555&type=chunk) - The company received net proceeds of **$186.3 million** from its February 2021 IPO. As of December 31, 2021, none of these proceeds had been used[557](index=557&type=chunk)[558](index=558&type=chunk) [Selected Financial Data](index=112&type=section&id=Item%206.%20%5BReserved%5D) This section is reserved and contains no information - This item is noted as '[Reserved]' and provides no data[559](index=559&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=113&type=section&id=Item%207.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) The company reported a **$68.9 million** net loss in 2021 due to increased R&D and G&A expenses, with cash to fund operations into Q4 2023 Results of Operations (2021 vs. 2020) | (in thousands) | 2021 | 2020 | Change | | :--- | :--- | :--- | :--- | | Research and development | $47,529 | $31,618 | $15,911 | | General and administrative | $21,489 | $11,748 | $9,741 | | **Total operating expenses** | **$69,018** | **$43,366** | **$25,652** | | Loss from operations | $(69,018) | $(43,366) | $(25,652) | | **Net loss** | **$(68,899)** | **$(43,337)** | **$(25,562)** | - Research and development expenses increased by **$15.9 million** in 2021, driven by higher personnel costs from increased headcount (**$10.5 million**), increased facilities and other expenses (**$2.2 million**), and higher external costs for clinical programs and preclinical studies (**$3.2 million**)[585](index=585&type=chunk) - General and administrative expenses rose by **$9.8 million** in 2021, primarily due to increased personnel costs (**$4.6 million**), higher professional fees for legal and insurance services (**$4.3 million**), and increased facility costs (**$0.9 million**)[586](index=586&type=chunk) - As of December 31, 2021, the company had **$207.5 million** in cash, cash equivalents, and investments. This balance is expected to fund operating expenses and capital requirements into the fourth quarter of 2023[569](index=569&type=chunk)[592](index=592&type=chunk) Cash Flow Summary (2021 vs. 2020) | (in thousands) | 2021 | 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | $(69,144) | $(36,292) | | Net cash used in investing activities | $(91,651) | $(4,161) | | Net cash provided by financing activities | $232,911 | $82,526 | [Quantitative and Qualitative Disclosures About Market Risk](index=124&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, Vor Biopharma is not required to provide the information for this item - The company is a smaller reporting company and is not required to provide this information[625](index=625&type=chunk) [Financial Statements and Supplementary Data](index=125&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) This section refers to the full financial statements and supplementary data appended to the Form 10-K - This item directs to the full financial statements which are appended to the report, starting on page F-1[626](index=626&type=chunk) [Changes in and Disagreements With Accountants on Accounting and Financial Disclosure](index=125&type=section&id=Item%209.%20Changes%20in%20and%20Disagreements%20With%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure) The company reports no changes in or disagreements with its accountants on accounting and financial disclosure - None reported[627](index=627&type=chunk) [Controls and Procedures](index=125&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded that disclosure controls and internal control over financial reporting were effective as of December 31, 2021 - Management concluded that the company's disclosure controls and procedures were effective as of December 31, 2021[628](index=628&type=chunk) - Management's assessment concluded that internal control over financial reporting was effective as of December 31, 2021[629](index=629&type=chunk) - No attestation report from the independent auditor on internal control is included, as permitted for emerging growth companies[630](index=630&type=chunk) [Other Information](index=125&type=section&id=Item%209B.%20Other%20Information) The company reports no other information - None[631](index=631&type=chunk) [PART III](index=126&type=section&id=PART%20III) This section provides details on the company's governance, executive compensation, ownership structure, and related party transactions [Directors, Executive Officers and Corporate Governance](index=126&type=section&id=Item%2010.%20Directors,%20Executive%20Officers%20and%20Corporate%20Governance) This section provides biographical information for executive officers and non-employee directors, detailing board and committee composition - The company's key executive officers are Robert Ang (President & CEO), Tirtha Chakraborty (Chief Scientific Officer), Nathan Jorgensen (Chief Financial Officer), and Christopher Slapak (Chief Medical Officer)[635](index=635&type=chunk) - The Board of Directors is chaired by Matthew Patterson and includes members with extensive experience in biotechnology, finance, and medicine[635](index=635&type=chunk)[642](index=642&type=chunk) - The Audit Committee is chaired by Daniella Beckman, who is qualified as an audit committee financial expert[650](index=650&type=chunk) [Executive Compensation](index=129&type=section&id=Item%2011.%20Executive%20Compensation) This section details executive compensation for 2021, including salary, bonuses, equity awards, and severance plans 2021 Summary Compensation Table | Name and Principal Position | Year | Salary ($) | Option Awards ($) | Non-Equity Incentive Plan Compensation ($) | All Other Compensation ($) | Total ($) | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | **Robert Ang, M.B.B.S.** | 2021 | 507,760 | 919,080 | 218,705 | 17,992 | 1,663,537 | | President and CEO | 2020 | 424,173 | 1,619,356 | 211,200 | 500,015 | 2,754,744 | | **Tirtha Chakraborty, Ph.D.** | 2021 | 384,022 | — | 138,701 | 17,915 | 540,638 | | Chief Scientific Officer | 2020 | 298,439 | 718,008 | 111,784 | 2,059 | 1,177,890 | | **Christopher Slapak, M.D.** | 2021 | 428,012 | — | 150,593 | 12,043 | 590,648 | | Chief Medical Officer | 2020 | 176,846 | 627,356 | 136,800 | 283,344 | 1,224,346 | - In 2021, target annual performance bonuses were **50% of base salary** for the CEO and **40%** for the other named executive officers[662](index=662&type=chunk) - The company has an Executive Severance and Change in Control Benefits Plan that provides for salary continuation, COBRA payments, and potential bonus payments and equity acceleration upon qualifying terminations, with enhanced benefits if the termination occurs within one year of a change in control[673](index=673&type=chunk)[674](index=674&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=140&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) Significant beneficial ownership of common stock is concentrated among key institutional investors and management as of March 1, 2022 Beneficial Ownership as of March 1, 2022 | Name of Beneficial Owner | Percentage of Shares Beneficially Owned | | :--- | :--- | | **Greater than 5% stockholders** | | | Entities affiliated with RA Capital Healthcare Fund, L.P. | 29.7% | | Entities affiliated with 5AM Ventures VI, L.P. | 17.0% | | PureTech Health LLC | 8.6% | | Entities affiliated with FMR, LLC | 8.5% | | **Management** | | | All current executive officers and directors as a group (10 persons) | 20.7% | [Certain Relationships and Related Transactions, and Director Independence](index=142&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions,%20and%20Director%20Independence) This section details related party transactions, including Series B financing and IPO participation, and confirms director independence - In 2020 and 2021, the company sold Series B preferred stock to related parties, including entities affiliated with RA Capital (**$40.0 million**), 5AM Ventures (**$20.0 million**), and FMR, LLC (**$19.4 million**)[737](index=737&type=chunk)[738](index=738&type=chunk) - Several **5% stockholders**, including funds affiliated with RA Capital, 5AM Ventures, and FMR, LLC, purchased an aggregate of **$89.5 million** of common stock in the company's IPO[742](index=742&type=chunk) - In October 2020, the company forgave a **$497,920** promissory note, including principal and accrued interest, that was issued to CEO Robert Ang in 2019[743](index=743&type=chunk) - The Board of Directors has determined that all directors are independent under Nasdaq Listing Rules, with the exception of CEO Robert Ang[753](index=753&type=chunk) [Principal Accountant Fees and Services](index=145&type=section&id=Item%2014.%20Principal%20Accountant%20Fees%20and%20Services) This section outlines the fees billed by Ernst & Young LLP for audit and tax services in 2021 and 2020 Accountant Fees (2021 vs. 2020) | (in thousands) | 2021 | 2020 | | :--- | :--- | :--- | | Audit Fees | $815,623 | $870,000 | | Tax Fees | $62,500 | $0 | | **Total** | **$878,123** | **$870,000** | [PART IV](index=147&type=section&id=PART%20IV) This section lists all exhibits filed with the annual report and confirms no 10-K summary is provided [Exhibits, Financial Statement Schedules](index=147&type=section&id=Item%2015.%20Exhibits,%20Financial%20Statement%20Schedules) This section lists all exhibits filed with the Form 10-K, including corporate documents and material contracts - This item lists all exhibits filed as part of the Annual Report, including corporate governance documents, material contracts, and required certifications[761](index=761&type=chunk)[763](index=763&type=chunk)[765](index=765&type=chunk) [Form 10-K Summary](index=149&type=section&id=Item%2016.%20Form%2010-K%20Summary) The company indicates that there is no Form 10-K summary - None[767](index=767&type=chunk) [Financial Statements](index=151&type=section&id=Financial%20Statements) This section presents the company's audited consolidated financial statements and detailed explanatory notes [Consolidated Financial Statements](index=153&type=section&id=Consolidated%20Financial%20Statements) The 2021 consolidated financial statements report a **$68.9 million** net loss, **$242.6 million** total assets, and **$216.3 million** equity Consolidated Balance Sheet Highlights (as of Dec 31) | (in thousands) | 2021 | 2020 | | :--- | :--- | :--- | | Cash and cash equivalents | $119,801 | $48,539 | | Investments | $87,668 | $0 | | **Total assets** | **$242,590** | **$75,908** | | Total liabilities | $26,327 | $27,637 | | Total stockholders' equity (deficit) | $216,263 | $(59,065) | Consolidated Statement of Operations Highlights (Year Ended Dec 31) | (in thousands, except per share data) | 2021 | 2020 | | :--- | :--- | :--- | | Total operating expenses | $69,018 | $43,366 | | **Net loss** | **$(68,899)** | **$(43,337)** | | Net loss per share, basic and diluted | $(2.10) | $(230.57) | [Notes to Consolidated Financial Statements](index=157&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) The notes detail the February 2021 IPO proceeds, license agreements, NOL carryforwards, and expanded facility lease obligations - The company completed its IPO on February 9, 2021, receiving net proceeds of **$186.3 million**. Upon the IPO, all outstanding redeemable convertible preferred stock converted into common stock[791](index=791&type=chunk)[792](index=792&type=chunk) - As of December 31, 2021, the company had **$119.3 million** in federal and **$109.2 million** in state net operating loss (NOL) carryforwards. A full valuation allowance has been recorded against the net deferred tax assets[876](index=876&type=chunk)[877](index=877&type=chunk) - The company is obligated to make potential future payments under its license agreements, including up to **$6.3 million** in regulatory and commercial milestones to Columbia University and up to **$14.0 million** in clinical, regulatory, and sales milestones to the NIH[869](index=869&type=chunk)[871](index=871&type=chunk) - In June 2021, the company amended its Cambridge, MA lease, expanding its space by over **40,000 square feet** and increasing its total future lease payment obligations[861](index=861&type=chunk)[862](index=862&type=chunk)[863](index=863&type=chunk)
Vor Biopharma (VOR) Investor Presentation - Slideshow
2021-12-01 07:02
Vor Biopharma's Platform and Vision - Vor Biopharma is focused on curing blood cancers through cell and genome engineering[11] - The company's paradigm involves engineering hematopoietic stem cells (eHSCs) to remove target expression on healthy cells, making cancer killing more specific[7] - Vor's platform combines genome engineering with hematopoietic stem cells and CAR-T-cell therapies to create treatment-resistant transplants[10] VOR33 and VCAR33 Programs - VOR33 aims to provide treatment-resistant HSC transplants[19] - VCAR33 is an allogeneic CAR-T therapy with potential advantages for healthy donor cell source and addressing tumor heterogeneity[24] - The VCAR33 construct is being studied in a Phase 1/2 clinical trial sponsored by the National Marrow Donor Program (NMDP)[26] Clinical Development and Milestones - Vor anticipates initial clinical data for VOR33 in the first half of 2022[25,73] - The company plans to file an IND for the VOR33 + VCAR33 treatment system in the second half of 2022, following initial VOR33 and NMDP clinical data[25,73] - A Phase 1/2a clinical trial (VBP101) is underway, evaluating VOR33 + Mylotarg in transplant-eligible AML patients at high risk of relapse[48,49] Market and Financial Position - Approximately 20,000 AML cases are diagnosed annually[13] - For patients who relapse post-transplant, 2-year survival is less than 20%[17] - Vor Biopharma secured $203 million in gross proceeds from its IPO in February 2021, providing a cash runway into mid-2023[73]
Vor Biopharma (VOR) Investor Presentation - Slideshow
2021-11-19 19:00
Vor Biopharma's Platform and Vision - Vor Biopharma is focused on curing blood cancers through cell and genome engineering[11] - The company's paradigm involves engineering hematopoietic stem cells (eHSCs) to remove target expression on healthy cells, making cancer killing more specific[7] - Vor's platform combines genome engineering with hematopoietic stem cells and CAR-T-cell therapies to create treatment-resistant transplants[10] VOR33 and VCAR33 Programs - VOR33 aims to provide treatment-resistant HSC transplants[19] - VCAR33 is an allogeneic CAR-T therapy with potential advantages for healthy donor cell source and addressing tumor heterogeneity[24] - The VCAR33 construct is being studied in a Phase 1/2 clinical trial sponsored by the National Marrow Donor Program (NMDP)[26] Clinical Development and Milestones - Vor anticipates initial clinical data for VOR33 in the first half of 2022[25,73] - The company plans to file an IND for the VOR33 + VCAR33 treatment system in the second half of 2022, following initial VOR33 and NMDP clinical data[25,73] - A Phase 1/2a clinical trial (VBP101) is underway, evaluating VOR33 + Mylotarg in transplant-eligible AML patients at high risk of relapse[48,49] Market and Financial Position - Approximately 20,000 AML cases are diagnosed annually[13] - For patients who relapse post-transplant, 2-year survival is less than 20%[17] - Vor Biopharma secured $203 million in gross proceeds from its IPO in February 2021, providing a cash runway into mid-2023[73]
Vor(VOR) - 2021 Q3 - Quarterly Report
2021-11-10 21:06
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from __________________ to __________________ Commission File Number: 001-39979 VOR BIOPHARMA INC. (Exact Name of Registrant as Specified in its Charter) | Delaw ...
Vor Biopharma (VOR) Investor Presentation - Slideshow
2021-09-16 19:34
Company Vision and Platform - Vor Biopharma's vision is to cure blood cancer through transformative cell engineering, utilizing a VOR platform that combines Hematopoietic Stem Cells (HSCs), genome engineering, and CAR-T cell therapies[6] - The company is developing engineered HSCs (eHSCs) as a solution to remove target expression on healthy cells, making cancer killing more cancer-specific[7, 8] - Vor's technology-driven platform vision includes VOR33 + VCAR33, multiplexed HSCs + dual-specific CAR-T, exploring targets beyond CD33, and exploring next-generation technologies like genome engineering and companion therapeutics[49] Pipeline and Clinical Development - The company's pipeline includes VOR33 (eHSC targeting CD33) for AML, MDS, and MPN, and VCAR33 (CAR-T targeting CD33) as a bridge-to-transplant for AML[13] - VOR33 is expected to have initial clinical data in the first half of 2022, and a development candidate selection for MDS and MPN[13] - The VOR33/VCAR33 treatment system is planned for an IND filing in the second half of 2022, following initial VOR33 and NMDP clinical data[13] VOR33 and CD33 Target - Preclinical validation of CD33 deletion in HSCs shows successful homing, engraftment, survival, and resistance to toxic therapy[18] - VOR33 shows no observed impact on cell populations or function, including B cells, myeloid cells, and phagocytosis[21, 23, 25] - In vitro studies show VOR33 provides resistance to CD33 therapy, with approximately 70-fold resistance to Mylotarg[33] Manufacturing and Clinical Trial - Vor is building a fully integrated manufacturing facility for eHSC and CAR-T drug products, with a streamlined cell manufacturing process for VOR33 that takes approximately 3 days, with a 7-10 day vein-to-vein time[35, 37] - VBP101 is a VOR33+Mylotarg Phase 1/2a clinical trial for transplant-eligible AML patients in remission but at high risk of relapse[38, 39]
Vor(VOR) - 2021 Q2 - Quarterly Report
2021-08-09 20:03
[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Cash and investments reached **$244.6 million** by June 30, 2021, primarily from IPO and Series B financing, despite a **$32.1 million** net loss Condensed Consolidated Balance Sheet Data (in thousands) | Account | June 30, 2021 | December 31, 2020 | | :--- | :--- | :--- | | Cash and cash equivalents | $169,516 | $48,539 | | Investments | $75,118 | $0 | | Total assets | $275,143 | $75,908 | | Total liabilities | $25,035 | $27,637 | | Total stockholders' equity (deficit) | $250,108 | $(59,065) | Condensed Consolidated Statements of Operations (in thousands) | Metric | Three Months Ended June 30, 2021 | Three Months Ended June 30, 2020 | Six Months Ended June 30, 2021 | Six Months Ended June 30, 2020 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $12,970 | $5,975 | $21,911 | $12,146 | | General and administrative | $5,410 | $2,065 | $10,199 | $3,772 | | **Loss from operations** | **$(18,380)** | **$(8,040)** | **$(32,110)** | **$(15,918)** | | **Net loss** | **$(18,370)** | **$(8,040)** | **$(32,093)** | **$(15,889)** | | Net loss per share | $(0.50) | $(56.85) | $(1.13) | $(115.83) | Condensed Consolidated Statements of Cash Flows (in thousands) | Cash Flow Activity | For the Six Months Ended June 30, 2021 | For the Six Months Ended June 30, 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | $(34,214) | $(14,413) | | Net cash used in investing activities | $(77,212) | $(1,097) | | Net cash provided by financing activities | $232,403 | $81,954 | - On February 9, 2021, the company completed its IPO, selling 11,302,219 shares of common stock at $18.00 per share, resulting in net proceeds of approximately **$186.3 million** after deducting underwriting discounts and commissions[26](index=26&type=chunk) - Upon the IPO closing, all outstanding shares of redeemable convertible preferred stock automatically converted into an aggregate of 24,924,501 shares of common stock[27](index=27&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=20&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses strategic focus on VOR33 and VCAR33, increased operating expenses, and substantial IPO and Series B funding expected to last into Q1 2023 [Overview](index=20&type=section&id=Overview) Vor Biopharma develops VOR33 and VCAR33 for hematological malignancies, with VOR33's IND accepted and initial Phase 1/2a data expected in H1 2022 - The company is developing VOR33, an engineered hematopoietic stem cell (eHSC) product candidate, and VCAR33, a companion CAR-T therapy, to treat acute myeloid leukemia (AML) and other hematological malignancies[69](index=69&type=chunk)[70](index=70&type=chunk) - The Investigational New Drug (IND) application for VOR33 was accepted by the FDA in January 2021, and a no objection letter was received from Health Canada in April 2021 for the Phase 1/2a clinical trial[70](index=70&type=chunk) - Initial clinical data from the VOR33 trial is expected in the first half of 2022, and the company plans to submit an IND for the VOR33/VCAR33 Treatment System in the second half of 2022[70](index=70&type=chunk)[72](index=72&type=chunk) - The company has experienced delays in its Phase 1/2a trial for VOR33 in part due to the COVID-19 pandemic[76](index=76&type=chunk) [Results of Operations](index=24&type=section&id=Results%20of%20Operations) Operating expenses significantly increased in H1 2021, leading to a **$32.1 million** operating loss, primarily due to higher R&D and G&A costs Comparison of Operating Expenses (in thousands) | Expense Category | Six Months Ended June 30, 2021 | Six Months Ended June 30, 2020 | Change | | :--- | :--- | :--- | :--- | | Research and development | $21,911 | $12,146 | $9,765 | | General and administrative | $10,199 | $3,772 | $6,427 | | **Total operating expenses** | **$32,110** | **$15,918** | **$16,192** | - The **$9.8 million** increase in R&D expenses for the six months ended June 30, 2021, was primarily due to a **$6.0 million** increase in personnel costs from higher headcount, a **$2.2 million** increase in external clinical and preclinical study costs, and a **$1.6 million** increase in facilities and other expenses[91](index=91&type=chunk) - The **$6.4 million** increase in G&A expenses for the six months ended June 30, 2021, was mainly due to a **$3.0 million** increase in personnel costs, a **$2.6 million** increase in professional fees (consultants, insurance), and a **$0.8 million** increase in facilities and other expenses[93](index=93&type=chunk) [Liquidity and Capital Resources](index=26&type=section&id=Liquidity%20and%20Capital%20Resources) The company held **$244.6 million** in cash and investments by June 30, 2021, primarily from IPO and Series B financing, expected to fund operations into Q1 2023 - As of June 30, 2021, the company had cash, cash equivalents, and investments of **$244.6 million**[75](index=75&type=chunk)[102](index=102&type=chunk) - In the first half of 2021, the company raised approximately **$232.4 million** from financing activities, including **$45.4 million** from the final tranche of its Series B preferred stock financing and **$187.0 million** in net proceeds from its IPO[95](index=95&type=chunk)[101](index=101&type=chunk) - Existing cash is expected to fund operating expenses and capital expenditure requirements into at least the **first quarter of 2023**[75](index=75&type=chunk)[103](index=103&type=chunk) - In June 2021, the company entered into two lease amendments to expand its corporate office and laboratory space, resulting in future fixed payment commitments of **$31.3 million** not yet recorded on the balance sheet[105](index=105&type=chunk)[106](index=106&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=28&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) Primary market risk is immaterial interest rate sensitivity on short-term cash and investments, with no significant foreign currency or inflation risk - The primary market risk is interest rate sensitivity on cash equivalents and investments, but the impact is considered immaterial due to their short-term maturities[109](index=109&type=chunk) - The company is not currently exposed to significant foreign currency exchange risk as operations are located in the U.S. and expenses are denominated in U.S. dollars[111](index=111&type=chunk) [Item 4. Controls and Procedures](index=28&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of June 30, 2021, with no material changes to internal control over financial reporting during the quarter - Management concluded that as of June 30, 2021, the company's disclosure controls and procedures were effective at the reasonable assurance level[114](index=114&type=chunk) - There were no changes in internal control over financial reporting during the quarter ended June 30, 2021, that have materially affected, or are reasonably likely to materially affect, internal controls[115](index=115&type=chunk) [PART II. OTHER INFORMATION](index=30&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=30&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any material legal proceedings, though ordinary course litigation may arise - The company is not currently a party to any material legal proceedings[118](index=118&type=chunk) [Item 1A. Risk Factors](index=30&type=section&id=Item%201A.%20Risk%20Factors) The company faces extensive risks including net losses, funding needs, unproven eHSC technology, manufacturing complexity, regulatory uncertainties, third-party reliance, and intellectual property challenges [Risks Related to Financial Position and Capital Needs](index=30&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) The company has significant net losses and accumulated deficit, expects continued losses, requires substantial additional funding, and its limited operating history complicates future viability assessment - The company has incurred significant net losses since inception, with a net loss of **$32.1 million** for the six months ended June 30, 2021, and an accumulated deficit of **$93.3 million** as of that date[120](index=120&type=chunk) - The company will need substantial additional funding and may be forced to delay, reduce, or eliminate research and development programs if unable to raise capital when needed[123](index=123&type=chunk) - The company has a limited operating history, has not completed any clinical trials, and has no history of commercializing products, making it difficult to assess its future viability[133](index=133&type=chunk) [Risks Related to Discovery, Development, and Commercialization](index=37&type=section&id=Risks%20Related%20to%20Discovery,%20Development,%20Manufacturing%20and%20Commercialization) Risks include unproven eHSC technology, dependence on VOR33 and VCAR33, complex manufacturing, intense competition, and potential market acceptance or reimbursement issues - Engineered hematopoietic stem cells (eHSCs) are a novel technology that is not yet clinically validated for human use, and the company's approach is unproven[142](index=142&type=chunk) - The company is substantially dependent on the success of its two most advanced product candidates, VOR33 and VCAR33[146](index=146&type=chunk) - The company's product candidates are complex and difficult to manufacture, which could lead to delays, limit supply, or otherwise harm the business[207](index=207&type=chunk) - The company faces significant competition, and competitors may develop safer, more effective, or less expensive therapies or achieve regulatory approval sooner[184](index=184&type=chunk) [Risks Related to Regulatory Review](index=54&type=section&id=Risks%20Related%20to%20Regulatory%20Review) Regulatory hurdles include potential clinical trial failures, an uncertain genome engineering landscape, and risk that trial endpoints may not be deemed clinically meaningful by authorities - Clinical trials may fail to demonstrate safety and efficacy, and the company may incur additional costs or be unable to complete development and commercialization[222](index=222&type=chunk) - Genome engineering technology is novel, and the regulatory landscape is uncertain and may change, making it difficult to predict the time and cost of obtaining regulatory approval[231](index=231&type=chunk) - There is a risk that regulatory authorities like the FDA may not consider the endpoints of the company's clinical trials to provide clinically meaningful results due to the novel technology and mechanisms of action[238](index=238&type=chunk) [Risks Related to Third-Party Relationships](index=63&type=section&id=Risks%20Related%20to%20Our%20Relationships%20with%20Third%20Parties) Heavy reliance on third parties for research, clinical trials, and manufacturing, including single-source suppliers, poses risks of performance issues, delays, and supply disruptions - The company relies on third parties like CROs to conduct clinical trials, and these parties may not perform satisfactorily, potentially delaying product development[249](index=249&type=chunk) - The company relies on third-party manufacturers, including single-source suppliers, for materials, which increases the risk of insufficient quantities or supply disruptions that could impair development[256](index=256&type=chunk) - Future collaborations for research, development, and commercialization are uncertain and, if unsuccessful, could prevent the company from capitalizing on market potential[262](index=262&type=chunk) [Risks Related to Intellectual Property](index=67&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) Success depends on in-licensed IP, with risks including license termination, challenges in acquiring key technology rights like CRISPR-Cas9, and potential third-party infringement claims - The company is highly dependent on intellectual property licensed from third parties, including Columbia University for VOR33 and the National Cancer Institute (NCI) for VCAR33, and termination of these licenses would significantly harm the business[270](index=270&type=chunk)[271](index=271&type=chunk)[272](index=272&type=chunk) - The company may not be successful in acquiring or in-licensing necessary rights to key technologies, such as CRISPR-Cas9, which are required for commercialization of its product candidates[297](index=297&type=chunk)[298](index=298&type=chunk) - Third-party claims of intellectual property infringement could prevent or delay product development, and the company is aware of third-party patents that may be construed to cover its eHSC technology[303](index=303&type=chunk)[304](index=304&type=chunk) [Risks Related to Regulatory and Other Legal Compliance](index=79&type=section&id=Risks%20Related%20to%20Regulatory%20and%20Other%20Legal%20Compliance%20Matters) Risks include challenges in foreign marketing approvals, ongoing regulatory compliance, exposure to complex healthcare laws, and potential impacts from healthcare reform on costs and pricing - Failure to obtain marketing approval in foreign jurisdictions would prevent product sales and impair the ability to generate revenue[326](index=326&type=chunk) - Relationships with healthcare providers and payors are subject to anti-kickback, fraud and abuse, and other healthcare laws, which could expose the company to criminal and civil penalties[339](index=339&type=chunk) - Healthcare reform legislation, such as the ACA, may increase the difficulty and cost of obtaining marketing approval and commercializing products, and could negatively affect prices[345](index=345&type=chunk)[346](index=346&type=chunk) [Risks Related to COVID-19, Personnel, Growth, and IT](index=87&type=section&id=Risks%20Related%20to%20COVID-19,%20Employee%20Matters,%20Managing%20Growth%20and%20Information%20Technology) Risks include COVID-19 disruptions, dependence on key personnel, challenges in managing growth, and vulnerability of IT systems to security breaches - The COVID-19 pandemic has caused delays in activating clinical sites for the VOR33 Phase 1/2a trial and could continue to disrupt clinical trials, supply chains, and regulatory reviews[356](index=356&type=chunk)[358](index=358&type=chunk) - The company is highly dependent on its key executives and its ability to attract and retain qualified scientific, clinical, and manufacturing personnel[359](index=359&type=chunk)[360](index=360&type=chunk) - Internal computer systems and those of third-party vendors are vulnerable to security breaches, which could result in material disruption of product development programs and business operations[365](index=365&type=chunk) [Risks Related to Common Stock Ownership](index=90&type=section&id=Risks%20Related%20to%20the%20Ownership%20of%20Our%20Common%20Stock) Common stock ownership risks include price volatility, lock-up expiration impact, substantial insider control, reduced disclosure as an 'emerging growth company,' and charter provisions hindering change of control - The market price of the company's common stock may be volatile due to factors such as clinical trial results, regulatory developments, and market conditions[370](index=370&type=chunk) - Insiders, including executive officers, directors, and 5%+ stockholders, have substantial control over the company, which could limit other stockholders' ability to influence key transactions[376](index=376&type=chunk) - The company is an "emerging growth company" and a "smaller reporting company," allowing it to rely on reduced disclosure requirements, which may make its stock less attractive to some investors[379](index=379&type=chunk)[382](index=382&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=95&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company's IPO closed February 9, 2021, raising **$186.3 million** in net proceeds, unused as of June 30, 2021, with no material change to planned use - The company closed its IPO on February 9, 2021, raising net proceeds of approximately **$186.3 million**[393](index=393&type=chunk)[394](index=394&type=chunk) - As of June 30, 2021, the company had not used any of the net proceeds from its IPO, and the planned use of proceeds has not materially changed[395](index=395&type=chunk) [Item 6. Exhibits](index=96&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed, including lease amendments and certifications from Principal Executive and Financial Officers - Exhibits filed include two amendments to the lease agreement with PPF Off 100 Cambridge Park Drive, LLC, dated June 15, 2021[397](index=397&type=chunk) - Certifications from the CEO and CFO pursuant to Sarbanes-Oxley Act Sections 302 and 906 are included as exhibits[397](index=397&type=chunk)
Vor(VOR) - 2021 Q1 - Quarterly Report
2021-05-06 20:02
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from __________________ to __________________ Commission File Number: 001-39979 VOR BIOPHARMA INC. (Exact Name of Registrant as Specified in its Charter) Delaware 81 ...
Vor(VOR) - 2020 Q4 - Annual Report
2021-03-25 20:01
PART I [Business](index=6&type=section&id=Item%201.%20Business) Vor Biopharma is a cell therapy company developing engineered hematopoietic stem cells (eHSCs) to make patients' healthy cells resistant to targeted cancer therapies, thereby reducing on-target toxicity and enhancing treatment efficacy [Overview and Pipeline](index=6&type=section&id=Overview%20and%20Pipeline) Vor Biopharma focuses on hematological malignancies, using patient engineering to make healthy cells resistant to targeted therapies, with lead product VOR33, an engineered hematopoietic stem cell for AML, entering Phase 1/2a trials - The company's core strategy is to genetically modify hematopoietic stem cells (HSCs) to remove surface targets (creating eHSCs), making healthy blood cells resistant to targeted cancer therapies and thus limiting on-target toxicity[17](index=17&type=chunk)[19](index=19&type=chunk) - The lead eHSC product candidate, VOR33, has the CD33 surface target removed. An IND application was accepted by the FDA in January 2021, with a Phase 1/2a trial in combination with Mylotarg planned to start in Q2 2021[20](index=20&type=chunk)[21](index=21&type=chunk) - VCAR33, a CAR-T therapy targeting CD33, is being developed as a bridge-to-transplant monotherapy for AML and as a companion therapeutic for VOR33. An IND for the VOR33/VCAR33 Treatment System is planned for the second half of 2022[22](index=22&type=chunk)[23](index=23&type=chunk) Vor Biopharma Pipeline and Anticipated Milestones | Program | Modality | Indication | Status | Anticipated Milestones | | :--- | :--- | :--- | :--- | :--- | | **VOR33 (CD33)** | eHSC | AML (with Mylotarg) | Phase 1/2 | Q2 2021: First patient enrolled<br>Late 2021/1H 2022: Initial engraftment and protection data | | **VCAR33 (CD33)** | CAR-T | Bridge-to-transplant AML | Phase 1/2 (NMDP-sponsored) | 2022: Initial monotherapy clinical proof-of-concept data | | **VOR33/VCAR33 Treatment System** | eHSC/CAR-T | AML | Preclinical | 2H 2022: IND filing | | **Discovery Programs** | Vor Platform | - | Discovery | Identifying additional targets (CD123, CLL-1) and multiplex engineering approaches | [Our Programs](index=14&type=section&id=Our%20Programs) The company's programs develop eHSCs by removing non-essential surface targets from donor HSCs to enable potent companion therapies, initially focusing on AML with VOR33 and VCAR33, and extending to other targets like CD123 and CLL-1 - VOR33 is an eHSC product candidate where the CD33 surface target is genetically removed from donor HSCs. This is intended to protect patients from the on-target toxicity of CD33-directed therapies like Mylotarg or VCAR33[55](index=55&type=chunk)[73](index=73&type=chunk) - Preclinical studies showed that CD33-deleted (CD33Del) cells had an approximately **70-fold increase in IC50 (resistance)** to Mylotarg compared to wild-type cells and that removal of CD33 did not negatively impact HSC differentiation or immune function[77](index=77&type=chunk)[84](index=84&type=chunk)[85](index=85&type=chunk) - VCAR33 is a CAR-T therapy targeting CD33, licensed from the NIH. It is being developed as a monotherapy for relapsed/refractory AML (bridge-to-transplant) and as a companion therapeutic for VOR33 in the VOR33/VCAR33 Treatment System[74](index=74&type=chunk)[108](index=108&type=chunk) - The company is advancing preclinical programs to create eHSCs with other targets removed, such as CD123 and CLL-1, both individually and in multiplexed combinations with CD33, to address other hematologic malignancies and potential tumor escape mechanisms[124](index=124&type=chunk)[147](index=147&type=chunk) [Commercial Strategy, Manufacturing, and Competition](index=40&type=section&id=Commercial%20Strategy%2C%20Manufacturing%2C%20and%20Competition) The commercial strategy leverages existing HSCT centers and favorable reimbursement, while manufacturing relies on CMOs with plans for in-house capabilities, facing intense competition from eHSC and CD33-targeted therapy developers - The commercialization strategy targets the approximately **12,000 annual allogeneic HSCTs** performed globally, leveraging the concentrated network of specialized transplant centers[156](index=156&type=chunk)[157](index=157&type=chunk) - The company anticipates favorable reimbursement pathways, including a Medicare carve-out for stem cell acquisition costs effective October 1, 2020, and a new MS-DRG base payment rate of approximately **$240,000 for CAR-T cases** effective January 1, 2021[159](index=159&type=chunk) - Vor currently relies on third-party contract manufacturers for materials and products but is planning to develop in-house manufacturing capabilities to support its clinical trials[174](index=174&type=chunk) - Key competition includes Tmunity Therapeutics (licensed eHSC technology from UPenn), companies with CD33-directed therapies like Johnson & Johnson and Amgen, and numerous approved AML treatments from companies like Novartis, Pfizer, and AbbVie[177](index=177&type=chunk)[178](index=178&type=chunk)[179](index=179&type=chunk) [Intellectual Property](index=44&type=section&id=Intellectual%20Property) The company secures patent protection for its product candidates, methods, and technologies, with key licenses from Columbia University for VOR33 and NIH for VCAR33, and also owns multiple patent families for engineering specific antigens and CAR technologies - The patent portfolio for the lead eHSC product, VOR33, includes three patent families exclusively licensed from Columbia University, with expected expiration dates in **2036, 2038, and 2040**[187](index=187&type=chunk)[188](index=188&type=chunk)[189](index=189&type=chunk) - The VCAR33 program is protected by a patent family exclusively licensed from the NIH related to CARs targeting CD33, with an expected expiration date in **2039**[195](index=195&type=chunk) - The company owns several patent families directed at engineering specific antigens in HSCs, including CD33, CLL-1, and CD123, as well as compositions and methods for making and using CARs[191](index=191&type=chunk)[194](index=194&type=chunk) [Government Regulation](index=48&type=section&id=Government%20Regulation) Vor Biopharma is subject to extensive FDA and global regulation, with cell therapy products regulated as biologics requiring BLA approval after preclinical and multi-phase clinical trials, and ongoing compliance with cGMP, labeling, and healthcare laws - The company's cell product candidates are regulated as biologics and are considered "more than minimally manipulated," requiring clinical trials and an approved Biologics License Application (BLA) before marketing[205](index=205&type=chunk) - The U.S. product development process involves preclinical testing (GLP), submitting an IND, performing clinical trials (GCP) in three phases, and obtaining FDA approval of a BLA after demonstrating safety, purity, and potency[207](index=207&type=chunk)[208](index=208&type=chunk)[215](index=215&type=chunk) - The company may utilize expedited programs such as Fast Track, Breakthrough Therapy, Accelerated Approval, and Priority Review to facilitate the development and review of its product candidates[230](index=230&type=chunk)[233](index=233&type=chunk) - Approved biologics in the U.S. are entitled to a **12-year period of marketing exclusivity** under the BPCIA, which prevents the FDA from approving a biosimilar version during that time[244](index=244&type=chunk) - The company's operations are subject to various U.S. healthcare laws, including the federal Anti-Kickback Statute, the False Claims Act, HIPAA, and the Physician Payments Sunshine Act, which regulate interactions with healthcare providers and payors[248](index=248&type=chunk)[249](index=249&type=chunk)[251](index=251&type=chunk) [Risk Factors](index=64&type=section&id=Item%201A.%20Risk%20Factors) The company faces substantial risks including significant net losses, the need for additional funding, reliance on unproven eHSC technology and lead candidates, intellectual property disputes, dependence on third-party licenses and manufacturers, evolving regulatory pathways, and the impact of the COVID-19 pandemic - The company has a history of significant net losses (**$43.3 million in 2020**) and expects to incur losses for the foreseeable future, requiring substantial additional funding to continue operations[290](index=290&type=chunk)[293](index=293&type=chunk) - The company's eHSC technology is novel and not yet clinically validated. The approach is unproven and may never lead to marketable products, facing challenges in regulatory approval, manufacturing, and avoiding potential complications like engraftment failure[311](index=311&type=chunk)[312](index=312&type=chunk) - Success is substantially dependent on the two most advanced candidates, VOR33 and VCAR33. Any failure or delay in their development, approval, or commercialization would significantly harm the business[315](index=315&type=chunk) - The company relies on intellectual property licensed from third parties, such as Columbia University for VOR33 and the NIH for VCAR33. Termination of these licenses would result in the loss of significant rights and harm the business[455](index=455&type=chunk)[457](index=457&type=chunk) - The company relies on third parties for manufacturing and clinical trials. This dependence increases risks related to supply sufficiency, quality control (cGMP compliance), and potential delays that are outside the company's direct control[431](index=431&type=chunk)[439](index=439&type=chunk) - The COVID-19 pandemic could disrupt development efforts by delaying clinical trial initiation and enrollment, impacting the supply chain, and causing operational disruptions at regulatory agencies[553](index=553&type=chunk)[554](index=554&type=chunk) [Unresolved Staff Comments](index=129&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments) The company reports no unresolved staff comments - Not applicable[594](index=594&type=chunk) [Properties](index=129&type=section&id=Item%202.%20Properties) The company's principal executive office and laboratory space is located at 100 Cambridgepark Drive, Suite 400, Cambridge, Massachusetts, under a lease for 32,798 square feet that terminates in June 2030 - The company leases **32,798 square feet** of office and laboratory space in Cambridge, Massachusetts, with the lease set to terminate in **June 2030**[595](index=595&type=chunk) [Legal Proceedings](index=129&type=section&id=Item%203.%20Legal%20Proceedings) The company is not currently subject to any material legal proceedings - As of the report date, the company is not a party to any material legal proceedings[596](index=596&type=chunk) [Mine Safety Disclosures](index=129&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This section is not applicable to the company - Not applicable[597](index=597&type=chunk) PART II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=130&type=section&id=Item%205.%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company's common stock began trading on Nasdaq under 'VOR' on February 5, 2021, following its IPO, with approximately 54 holders of record as of March 12, 2021, and no intention to pay cash dividends, having raised approximately **$186.3 million** net from its IPO - The company's common stock began trading on the Nasdaq Global Select Market under the ticker **"VOR"** on **February 5, 2021**[600](index=600&type=chunk) - The company has never paid cash dividends and does not anticipate paying any in the foreseeable future, intending to retain earnings for business development[602](index=602&type=chunk) - On February 9, 2021, the company closed its IPO, receiving net proceeds of approximately **$186.3 million**[611](index=611&type=chunk)[612](index=612&type=chunk) [Selected Financial Data](index=131&type=section&id=Item%206.%20Selected%20Financial%20Data) As a smaller reporting company and an emerging growth company, Vor Biopharma is not required to provide selected financial data - The company is a smaller reporting company and emerging growth company and is not required to provide this information[614](index=614&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=132&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) The company reported a net loss of **$43.3 million** in 2020, up from **$10.8 million** in 2019, driven by increased R&D and G&A expenses, with no revenue generated, and expects current funds, including **$186.3 million** from its IPO, to support operations into Q1 2023 Comparison of Results of Operations (2020 vs. 2019) | (in thousands) | 2020 | 2019 | Change | | :--- | :--- | :--- | :--- | | **Research and development** | $31,618 | $6,200 | $25,418 | | **General and administrative** | $11,748 | $4,217 | $7,531 | | **Total operating expenses** | $43,366 | $10,417 | $32,949 | | **Loss from operations** | ($43,366) | ($10,417) | ($32,949) | | **Net loss** | ($43,337) | ($10,839) | ($32,498) | - Research and development expenses increased by **$25.4 million** in 2020, primarily due to a **$14.9 million** increase in external preclinical studies and consulting, and an **$8.2 million** increase in personnel costs from higher headcount[643](index=643&type=chunk) - General and administrative expenses increased by **$7.5 million** in 2020, driven by a **$3.8 million** increase in personnel costs, a **$2.4 million** increase in professional fees (legal and consulting), and a **$1.3 million** increase in facilities and other expenses[644](index=644&type=chunk) - As of December 31, 2020, the company had **$48.5 million** in cash and cash equivalents. Subsequent financing events in early 2021, including the final Series B tranche (**$45.4 million**) and the IPO (**$186.3 million net**), significantly increased liquidity[624](index=624&type=chunk)[647](index=647&type=chunk)[648](index=648&type=chunk) - The company expects its cash on hand, including proceeds from the IPO and Series B financing, to fund operating expenses and capital requirements into the **first quarter of 2023**[650](index=650&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=146&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk is interest rate sensitivity on short-term cash and equivalents, which is not expected to be material, with no outstanding debt and minimal foreign currency or inflation exposure - The primary market risk is interest rate sensitivity on cash and cash equivalents. However, due to their short-term maturities, the impact of a **100 basis point change** is not expected to be material[686](index=686&type=chunk) - The company has no debt outstanding as of December 31, 2020, and therefore has no related interest rate risk[687](index=687&type=chunk) - Foreign currency exchange risk is not significant as operations are located in the U.S. and expenses are denominated in U.S. dollars[688](index=688&type=chunk) [Financial Statements and Supplementary Data](index=147&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) This section contains the company's audited consolidated financial statements for fiscal years 2020 and 2019, including the Report of Independent Registered Public Accounting Firm, balance sheets, statements of operations, equity, cash flows, and notes - This item includes the company's audited consolidated financial statements and supplementary data, as indexed on page F-1 of the report[690](index=690&type=chunk)[843](index=843&type=chunk) [Changes in and Disagreements With Accountants on Accounting and Financial Disclosure](index=147&type=section&id=Item%209.%20Changes%20in%20and%20Disagreements%20with%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure) The company reports no changes in or disagreements with its accountants on accounting and financial disclosure - None[691](index=691&type=chunk) [Controls and Procedures](index=147&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of December 31, 2020, with no material changes to internal controls reported, and a management report on internal control over financial reporting is not yet required for this newly public company - Management concluded that as of December 31, 2020, the company's disclosure controls and procedures were effective at the reasonable assurance level[692](index=692&type=chunk) - As a newly public company, a management report on internal control over financial reporting is not included in this Annual Report[693](index=693&type=chunk) [Other Information](index=147&type=section&id=Item%209B.%20Other%20Information) The company reports no other information - None[695](index=695&type=chunk) PART III [Directors, Executive Officers and Corporate Governance](index=148&type=section&id=Item%2010.%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) This section provides information on the company's executive officers and non-employee directors as of March 17, 2021, detailing their backgrounds, the composition of board committees, and the company's adopted Code of Business Conduct and Ethics - The report lists the executive officers, including Robert Ang (CEO), Tirtha Chakraborty (CSO), Nathan Jorgensen (CFO), Sadik Kassim (CTO), and Christopher Slapak (CMO), along with their professional backgrounds[698](index=698&type=chunk)[700](index=700&type=chunk)[701](index=701&type=chunk) - The non-employee directors are detailed, including Kush Parmar (Chair), Daniella Beckman, David C. Lubner, Sven (Bill) Ante Lundberg, Matthew Patterson, and Joshua Resnick[698](index=698&type=chunk)[706](index=706&type=chunk)[707](index=707&type=chunk) - The composition of the board's committees (Audit, Compensation, Nominating and Corporate Governance) is outlined, with members identified for each[698](index=698&type=chunk)[714](index=714&type=chunk) [Executive Compensation](index=151&type=section&id=Item%2011.%20Executive%20Compensation) Named executive officers for 2020 included CEO Robert Ang, CSO Tirtha Chakraborty, and CMO Christopher Slapak, with compensation comprising salary, non-equity incentives, and option awards, alongside new executive severance, equity incentive, and employee stock purchase plans adopted in early 2021, and a formal non-employee director compensation policy 2020 Summary Compensation Table | Name and Principal Position | Year | Salary ($) | Option Awards ($) | Non-Equity Incentive Plan Compensation ($) | All Other Compensation ($) | Total ($) | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | **Robert Ang, M.B.B.S.** | 2020 | 424,173 | 1,619,356 | 211,200 | 500,015 | 2,754,744 | | **Tirtha Chakraborty, Ph.D.** | 2020 | 298,439 | 718,008 | 111,784 | 2,059 | 1,177,890 | | **Christopher Slapak, M.D.** | 2020 | 176,846 | 627,356 | 136,800 | 283,344 | 1,224,346 | - In January 2021, the company adopted an Executive Severance and Change in Control Benefits Plan, providing for salary continuation, COBRA payments, and potential bonus payments and equity acceleration upon qualifying terminations[737](index=737&type=chunk)[738](index=738&type=chunk) - The company adopted a new **2021 Equity Incentive Plan** and a **2021 Employee Stock Purchase Plan (ESPP)**, which became effective in February 2021. No further grants will be made under the 2015 Plan[749](index=749&type=chunk)[769](index=769&type=chunk) - A formal non-employee director compensation policy was adopted in January 2021, providing annual cash retainers (e.g., **$35,000 base**) and initial and annual option grants[783](index=783&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=165&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) As of March 12, 2021, RA Capital Healthcare Fund, L.P. was the largest beneficial owner with **30.0%** of outstanding shares, while all current executive officers and directors as a group beneficially owned **20.5%** of the company's common stock Security Ownership of Greater than 5% Stockholders (as of March 12, 2021) | Name of Beneficial Owner | Percentage of Shares Beneficially Owned | | :--- | :--- | | Entities affiliated with RA Capital Healthcare Fund, L.P. | 30.0% | | Entities affiliated with 5AM Ventures VI, L.P. | 18.0% | | Entities affiliated with FMR, LLC | 10.6% | | PureTech Health LLC | 8.6% | - As of March 12, 2021, all current executive officers and directors as a group beneficially owned **20.5%** of the company's common stock[795](index=795&type=chunk) [Certain Relationships and Related Transactions, and Director Independence](index=168&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions%2C%20and%20Director%20Independence) The company details related party transactions, including preferred stock financings and IPO participation by major investors, a forgiven promissory note to the CEO, and a consulting agreement with the CMO, while affirming that all directors except the CEO are independent under Nasdaq rules - The company conducted Series A-2 and Series B preferred stock financings with participation from major investors and related parties, including entities affiliated with 5AM Ventures, RA Capital, and PureTech Health[803](index=803&type=chunk)[808](index=808&type=chunk) - In the company's IPO, affiliated funds of RA Capital, 5AM Ventures, and FMR LLC purchased an aggregate of **$89.5 million** worth of common stock[814](index=814&type=chunk) - In September 2019, the company issued a limited recourse promissory note for **$497,920** to CEO Robert Ang to facilitate an early exercise of stock options; this note and accrued interest were forgiven in full in October 2020[815](index=815&type=chunk) - The Board of Directors has determined that all directors are independent under Nasdaq rules, with the exception of CEO Robert Ang[825](index=825&type=chunk) [Principal Accounting Fees and Services](index=173&type=section&id=Item%2014.%20Principal%20Accounting%20Fees%20and%20Services) The company paid its independent registered public accounting firm, Ernst & Young LLP, **$870,000** in 2020 and **$50,000** in 2019, exclusively for audit services, with the audit committee pre-approving all services Principal Accounting Fees (Ernst & Young LLP) | (in thousands) | 2020 | 2019 | | :--- | :--- | :--- | | **Audit Fees** | $870 | $50 | | **Total Fees** | $870 | $50 | - The audit committee has adopted policies and procedures for the pre-approval of all audit and non-audit services provided by the independent auditor[829](index=829&type=chunk) PART IV [Exhibits, Financial Statement Schedules](index=174&type=section&id=Item%2015.%20Exhibits%2C%20Financial%20Statement%20Schedules) This section lists the financial statements, schedules, and exhibits filed as part of the Form 10-K, including corporate governance documents, material contracts, and certifications - This item provides a list of all exhibits filed with the Form 10-K, including corporate governance documents, material contracts, and certifications[834](index=834&type=chunk)[835](index=835&type=chunk) [Form 10-K Summary](index=176&type=section&id=Item%2016.%20Form%2010-K%20Summary) The company reports no Form 10-K summary - None[837](index=837&type=chunk)