8-K
Q32 Bio Inc. (QTTB)
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): January 28, 2022
HOMOLOGY MEDICINES, INC.
(Exact name of Registrant as Specified in Its Charter)
| Delaware | 001-38433 | 47-3468154 |
|---|---|---|
| (State or other jurisdiction of<br> <br>incorporation or organization) | (Commission<br> <br>File Number) | (I.R.S. Employer<br> <br>Identification No.) |
| One Patriots Park<br> <br>Bedford, MA | 01730 | |
| --- | --- | |
| (Address of principal executive offices) | (Zip Code) |
(781) 301-7277
(Registrant’s telephone number, include area code)
Not Applicable
(Former Name or Former Address, if Changed Since Last Report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions
| ☐ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
|---|---|
| ☐ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
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| ☐ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| --- | --- |
| ☐ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
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Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading<br> <br>Symbol(s) | Name of each exchange<br> <br>on which registered |
|---|---|---|
| Common Stock,<br> <br>$0.0001 par value per share | FIXX | Nasdaq Global Select Market |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company ☒
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 1.01. | Entry Into a Material Definitive Agreement. |
|---|
Equity Securities Purchase Agreement and Contribution Agreement
On January 28, 2022, Homology Medicines, Inc. (“Homology”) entered into an Equity Securities Purchase Agreement (the “Purchase Agreement”) with a newly formed adeno-associated virus (“AAV”) vector manufacturing company (“Newco”), Oxford Biomedica (US), Inc. (“OXB”) and Oxford Biomedica plc (“OXB Parent” and collectively with OXB, “Oxford”), pursuant to which Homology and Oxford have agreed to collaborate to operate Newco, which will provide AAV vector process development and manufacturing to pharmaceutical and biotechnology companies (the “Transaction”).
Pursuant to the terms of the Purchase Agreement and a contribution agreement (the “Contribution Agreement”) to be entered into between Homology and Newco prior to the closing of the Transaction (the “Closing”), (i) Homology has agreed to assign and transfer to Newco all of its assets that are primarily used in the manufacturing of AAV vectors for use in gene therapy or gene editing products, but excluding certain assets related to manufacturing or testing of Homology’s proprietary AAV vectors (collectively, the “Transferred Assets”) and Newco has agreed to assume from Homology, and agree to pay, perform and discharge when due, all of Homology’s duties, obligations, liabilities, interests and commitments of any kind under, arising out of or relating to the Transferred Assets and (ii) in exchange therefor, Newco will issue to Homology 175,000 common equity units in Newco (“Units”).
The Purchase Agreement further provides that, effective as of the Closing, Homology will sell to OXB, and OXB will purchase from Homology, 130,000 Units (the “Transferred Units”) in exchange for $130 million. In connection with the Closing, OXB will also contribute $50 million in cash to Newco in exchange for an additional 50,000 Units. Immediately following the Closing, (i) OXB will own 180,000 Units, representing 80 percent (80%) of the fully diluted equity interests in Newco, and (ii) Homology will own 45,000 Units, representing 20 percent (20%) of the fully diluted equity interests in Newco.
Pursuant to the Amended and Restated Limited Liability Company Agreement of Newco (the “Newco Operating Agreement”) to be executed in connection with the Closing, at any time following the three-year anniversary of the Closing, (i) OXB will have an option to cause Homology to sell and transfer to OXB, and (ii) Homology will have an option to cause OXB to purchase from Homology, in each case all of Homology’s equity ownership interest in Newco at a price equal to 5.5 times the revenue for the immediately preceding 12-month period, subject to a specified maximum amount.
At or immediately prior to the Closing, Newco, Homology and Oxford, as applicable (each, a “Party”), will enter into certain ancillary agreements, including: (i) a license and patent management agreement (the “LPMA”) pursuant to which Newco will grant licenses under certain intellectual property to Homology, and Homology and Newco will cooperate in the management of the patents that are Transferred Assets; (ii) a manufacturing and supply agreement (the “Supply Agreement”) pursuant to which Newco will manufacture and supply certain AAV-based products and perform certain services to and for Homology, and a quality agreement pursuant to which Newco will comply with certain quality requirements in connection with certain activities under the Supply Agreement; (iii) a transitional services agreement pursuant to which (x) Homology will perform certain services for the benefit of Newco and (y) Newco will perform certain services for the benefit of Homology; (iv) a lease assignment pursuant to which Homology will assign all of its right, title and interest in, to and under a facility lease to Newco (the “Lease Assignment”); (v) a sublease agreement, pursuant to which Newco will sublease certain premises of its facility to Homology as further described therein (the “Sublease Agreement”); (vi) an employee matters agreement pursuant to which the Parties will agree to allocate certain liabilities and obligations relating to employees associated with the business of Newco; and (vii) a patent assignment agreement evidencing the recorded transfer of certain patent rights from Homology to Newco as set forth therein.
Pursuant to the terms of the Supply Agreement, Newco will manufacture for and supply to Homology specified quantities of Homology’s AAV products, manufactured in accordance with current Good Manufacturing Practices, and perform manufacturing development services for certain of Homology’s AAV-based products. The Supply Agreement will provide for an initial term of three years, which period may be extended for an additional one-year term. Homology will agree to purchase from Newco at least 50% of Homology’s clinical supply requirements of AAV-based products during the initial term of the Supply Agreement. Homology will purchase certain annual minimum amounts of batches of drug substance and drug product for such products under the Supply Agreement. After the initial term, Homology will have the right to terminate the Supply Agreement for convenience or other reasons specified in the Supply Agreement upon prior written notice. Either Party may terminate the Supply Agreement upon an uncured material breach by the other Party or upon the bankruptcy or insolvency of the other Party.
Under the terms of the Lease Assignment, Newco will be located within Homology’s existing headquarters in Bedford, Massachusetts. Newco will incorporate Homology’s AAV manufacturing capabilities and will be operated by 125 AAV manufacturing experts formerly employed by Homology. Pursuant to the terms of the LPMA, Newco will license back continued platform development to Homology for its products.
Pursuant to the terms of the Newco Operating Agreement, Homology will be entitled to designate one director on the Board of Directors of Newco (the “Board”), which shall initially be Arthur Tzianabos, Homology’s President and Chief Executive Officer. Further, Tim Kelly, Homology’s current Chief Operating Officer, will serve as the Chief Executive Officer and Chairman of the Board.
The Closing is expected to occur in the first quarter of 2022, subject to the satisfaction of certain closing conditions set forth in the Purchase Agreement, including compliance by the parties to the Purchase Agreement with the requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 1976.
The Purchase Agreement contains customary representations, warranties and covenants made by and to the parties thereto as of certain specified dates. The statements embodied in those representations and warranties were made for the purposes of the contract between the parties and were made solely for the benefit of the parties thereto, and may be subject to qualifications or limitations agreed by the parties in connection with negotiating the terms of that contract. In addition, certain representations and warranties were made as of a specific date, may be subject to a contractual standard of materiality different from those generally applicable to investors, or may have been used for the purposes of allocating risk between parties rather than establishing matters as facts.
Accordingly, the foregoing description of the Purchase Agreement is only intended to provide investors with information regarding the terms of the Purchase Agreement and not to provide investors with any other factual information regarding Homology or its business, and should be read in conjunction with the disclosures in Homology’s periodic reports and other filings with the Securities and Exchange Commission.
Homology intends to file copies of the Purchase Agreement, the Contribution Agreement, the Lease Assignment, the Sublease Agreement, the Newco Operating Agreement and the Supply Agreement, in certain cases with confidential portions redacted, with the Securities and Exchange Commission following the Closing.
| Item 5.02. | Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers. |
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Effective as of the Closing, Tim Kelly will assume the role of Chief Executive Officer and Chairman of the Board of Newco and will no longer serve as the Chief Operating Officer of Homology.
| Item 7.01. | Regulation FD Disclosure. |
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On January 28, 2022, Homology issued a press release announcing the Transaction. A copy of the press release is attached hereto as Exhibit 99.1 and incorporated into this Item 7.01 by reference.
On January 28, 2022, Homology posted a slide presentation relating to the Transaction in the “Investors” portion of its website at investors.homologymedicines.com. The slide presentation also contains updates regarding Homology’s clinical programs and product pipeline. A copy of the slide presentation is attached to this Current Report as Exhibit 99.2. Homology undertakes no obligation to update, supplement or amend the materials attached hereto as Exhibits 99.1 and 99.2.
The information contained in Item 7.01 of this Current Report (including Exhibit 99.1 and Exhibit 99.2 attached hereto) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly provided by specific reference in such a filing.
Forward-Looking Statements Disclaimer
This Current Report contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this Current Report that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation, statements regarding the completion of the Transaction and the execution of the Contribution Agreement, the satisfaction of the requisite conditions precedent described herein, and the anticipated proceeds to Homology of $130 million. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we may not realize the anticipated benefits of the collaboration with Oxford; the Transaction may not close in the time frame expected or at all; the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption “Risk Factors” in Homology’s Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2021 and our other filings with the Securities and Exchange Commission could cause actual results to differ materially from those indicated by the forward-looking statements made in this Current Report. Any such forward-looking statements represent management’s estimates as of the date of this Current Report. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.
| Item 9.01. | Financial Statements and Exhibits. |
|---|
(d) Exhibits
The following exhibits relating to Item 7.01 shall be deemed to be furnished, and not filed:
| Exhibit<br>No. | Description |
|---|---|
| 99.1 | Press Release issued by Homology Medicines, Inc., dated January 28, 2022 |
| 99.2 | Corporate Slide Presentation of Homology Medicines, Inc. dated January 28, 2022 |
| 104 | Cover Page Interactive Data File (embedded within the inline XBRL document) |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| HOMOLOGY MEDICINES, INC. | ||
|---|---|---|
| Date: February 2, 2022 | By: | /s/ W. Bradford Smith |
| W. Bradford Smith | ||
| Chief Financial Officer and Treasurer |
EX-99.1
Exhibit 99.1
Oxford Biomedica broadens leading viral vector offerings by incorporating Homology Medicines’established AAV capabilities into a newly formed AAV Manufacturing and Innovation Business in the U.S. with Homology Medicines as 20% owner
Oxford Biomedica and Homology Medicines to establish Oxford Biomedica Solutions LLC, a new U.S.-based AAV Manufacturing and InnovationBusiness
Homology Medicines to receive $130 million from Oxford Biomedica to continue to advance its three clinical programsand genetic medicines platform
Oxford Biomedica to invest $50 million to fund the growth of Oxford Biomedica Solutions LLC andwill own 80%, with Homology Medicines to own 20%; Homology Medicines to secure preferred key customer status
Dr. Roch Doliveuxto become Interim CEO of Oxford Biomedica and John Dawson to retire as CEO and remain a Board Director of Oxford Biomedica
OxfordBiomedica and Homology Medicines to host webcasts today to discuss the announcement
Oxford, UK and Bedford, Mass., U.S. – January 28,2022: Oxford Biomedica plc (LSE:OXB) (“Oxford Biomedica” or “the Group”), a leading gene and cell therapy group, and Homology Medicines, Inc. (Nasdaq: FIXX) (“Homology”), a genetic medicines company, announced today that the companies have agreed to establish a high-performing, full scope Adeno-Associated Virus (AAV) Manufacturing and Innovation Business in the U.S.
As a result of the agreement, which is immediately accretive to the Group’s revenue growth with contribution from Homology and future customer pipeline, Oxford Biomedica will establish a presence in the U.S. and will offer global pharmaceutical and biotechnology clients innovative manufacturing expertise in AAV and lentiviral-based cell and gene therapies. Oxford Biomedica Solutions LLC will provide access to Homology’s proven end-to-end manufacturing capabilities. Future customers will also benefit from technical synergies brought in by the Group’s extensive innovations and know-how in viral vector manufacturing. This collaborative and complementary AAV and lentiviral vector-based approach has the potential to accelerate the mission to improve patients’ lives worldwide.
Oxford Biomedica Solutions’ AAV Manufacturing and Innovation Business is to be led by Tim Kelly as Chief Executive Officer and Chair of its Board of Directors. Tim is currently the Chief Operating Officer of Homology, where he and his team were responsible for building and leading Homology’s internal technical and manufacturing operations, which will become Oxford Biomedica Solutions and incorporate the following:
| • | Proprietary ‘plug and play’ process development and manufacturing platform (i.e., same process for<br>different transgenes and capsids) protected by intellectual property (IP); |
|---|---|
| • | 125 AAV manufacturing experts who encompass the full CMC scope of upstream and downstream process development,<br>analytical development, manufacturing operations and quality control; |
| --- | --- |
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| • | Experienced team and high-quality GMP vector production capabilities that has been operating since 2019 without a<br>single failed batch; **** |
|---|---|
| • | Over 40 analytical assays developed and an established breadth of vector characterization that has met CMC<br>requirements for three cleared Investigational New Drug applications (INDs) from Homology’s pipeline; and **** |
| --- | --- |
| • | A GMP facility near Boston, Massachusetts, operating three 500-liter<br>bioreactors using a serum-free suspension process, which has also been successfully scaled to 2,000 liters. **** |
| --- | --- |
Under the terms of the agreement, the Group will pay Homology $130 million upfront and invest $50 million to fund Oxford Biomedica Solutions in return for an 80 percent ownership stake, while Homology will own 20 percent of the new company. Additionally, at any time following the three-year anniversary of the agreement, the Group will have a call option to purchase, and Homology will have a put option to require the Group to purchase, Homology’s ownership interest in Oxford Biomedica Solutions.
The transaction is expected to close in Q1 2022, subject to the satisfaction of certain closing conditions including the requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 1976.
Simultaneous to this announcement, Dr. Roch Doliveux will assume the role of interim CEO of Oxford Biomedica, as well as Chair of the Group’s Board, and John Dawson will retire from the Group as CEO with immediate effect, while remaining a Board Director and Advisor to the Group. A search for a CEO successor is underway, as previously announced by the Group.
**Dr.Roch Doliveux, Chair and Interim CEO of Oxford Biomedica,commented: “Accessing Homology Medicines’ unique AAV capabilities is a major advancement in Oxford Biomedica’s goal to become an innovative global viral vector leader that provides solutions to Cell and Gene Therapy (C>)Biotech and Biopharma companies for their process development and manufacturing needs across key viral vectors. Process Development/CMC being one of the most important critical success factors to ensure efficacy, safety and affordability ofC>, Oxford Biomedica is in a strong position to enable our customers to bring their new medicines to many more patients and change their lives. We look forward to working with Homology’s impressive team and uniquely robust processes toachieve world-leadership as a provider of AAV solutions in addition to enhancing our leadership in lentiviral vectors. Having a U.S. base brings us closer to customers, talent, innovation in academia and pools of capital all of which will allowgrowth and building a market leadership position.”
Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines,said: “We chose Oxford Biomedica for its leadership in viral-based manufacturing and prestigious global client base, and we believe that Oxford Biomedica Solutions will build upon the strengths of both companies. Our leadership in AAVprocess development and CMC, which resulted from establishing internal capabilities early on, has enabled us to advance three programs from discovery into the clinic within five years. We believe the opportunity in Oxford Biomedica Solutions furtherleverages the value we created in this broad capability, including the demonstrated expertise of our team, to provide much-needed high-quality viral vector to other companies and, importantly, more patients around the world. Additionally, the$130 million cash infusion, coupled with the reduction in operating expenses, significantly extends our runway and supports the continued advancement of our programs and genetic medicines platform. We believe our ownership stakein Oxford Biomedica Solutions, continued access to our AAV ‘plug and play’ manufacturing platform as a preferred customer, and our ability to benefit from any further manufacturing innovations, will be key value drivers for Homology.”
Dr. Roch Doliveux commenting on John’s departure added: “I would like to express my sincere appreciation forJohn Dawson’s leadership and achievements as CEO. He and the world-class management team he has built have worked relentlessly in selecting the best AAV partner and creating this potentially transformative deal for the Group. This makes iteasier to support John’s decision to step down at this time. I am delighted that he will remain a Board member and Advisor.”
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John Dawson, CBE, added: “I am excited that this will be my final deal as CEO of theGroup. This transaction not only brings proven, scalable, high-quality AAV manufacturing capabilities to Oxford Biomedica, which was in line with our strategy, but also expands Oxford Biomedica’s presence into the U.S. and brings us a dedicatedteam in close geographic proximity to other leading gene therapy companies in Boston and more widely across the U.S.”
Oxford Biomedica Analyst briefing
Oxford Biomedica’s management team will host a briefing for analysts via conference call and webcast at 14:00 GMT (9:00 ET) today, January 28, 2022.
Please contact Oxfordbiomedica@consilium-comms.com for details.
Homology Medicines Webcast
Homology management will host a webcast today, January 28, 2022, at 8:00 a.m. ET to discuss the transaction and provide an update on its clinical programs and product pipeline. The webcast will be accessible on Homology’s website in the Investors section, and the webcast replay will be available on the website for 90 days following the presentation. To access using the conference call line, dial (866) 244-8091 (U.S./Canada toll-free) or +1 (602) 563-8623, with Conference ID 9734109.
About Oxford Biomedica
Oxford Biomedica (LSE:OXB) is a leading, fully integrated, gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford Biomedica and its subsidiaries (the “Group”) have built a sector leading lentiviral vector delivery platform (LentiVector^®^), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, CNS disorders and liver diseases. The Group has also entered into a number of partnerships, including with Novartis, Bristol Myers Squibb, Sio Gene Therapies, Orchard Therapeutics, Santen, Beam Therapeutics, Boehringer Ingelheim, Arcellx and Cabaletta, through which it has long-term economic interests in other potential gene and cell therapy products. Additionally, the Group has signed a 3-year master supply and development agreement with AstraZeneca for large-scale manufacturing of the adenoviral based COVID-19 vaccine, AZD1222. Oxford Biomedica is based across several locations in Oxfordshire, UK and employs more than 740 people. Further information is available at www.oxb.com.
About Homology Medicines, Inc.
Homology Medicines, Inc. is a clinical-stage genetic medicines company dedicated to transforming the lives of patients suffering from rare diseases by addressing the underlying cause of the disease. Homology’s clinical programs include HMI-102, an investigational gene therapy for adults with phenylketonuria (“PKU”); HMI-103, a gene editing candidate for PKU; and HMI-203, an investigational gene therapy for Hunter syndrome. Additional programs focus on metachromatic leukodystrophy (“MLD”), paroxysmal nocturnal hemoglobinuria (“PNH”) and other diseases. Homology’s proprietary platform is designed to utilize its family of 15 human hematopoietic stem cell-derived adeno-associated virus vectors (“AAVHSCs”) to precisely and efficiently deliver genetic medicines in vivo through a gene therapy or nuclease-free gene editing modality, as well as to deliver one-time gene therapy to produce antibodies throughout the body through the GTx-mAb platform. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a focus on rare diseases. Homology believes its initial clinical data and compelling preclinical data, scientific and product development expertise, internal manufacturing capabilities and broad intellectual property position Homology as a leader in genetic medicines. For more information, visit www.homologymedicines.com.
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Oxford Biomedica’s Forward-Looking Statements
This press release may contain and Oxford Biomedica may make verbal statements containing “forward-looking statements” with respect to certain of Oxford Biomedica’s plans and its current goals and expectations relating to its future financial condition, performance, strategic initiatives, objectives and results. Forward-looking statements sometimes use words such as “aim”, “anticipate”, “target”, “expect”, “estimate”, “intend”, “plan”, “goal”, “believe”, “seek”, “may”, “could”, “outlook” or other words of similar meaning. By their nature, all forward-looking statements involve risk and uncertainty because they relate to future events and circumstances which are beyond the control of Oxford Biomedica. As a result, the actual future financial condition, performance and results of Oxford Biomedica may differ materially from the plans, goals and expectations set forth in any forward-looking statements. Any forward-looking statements made in this press release by or on behalf of Oxford Biomedica speak only as of the date they are made. The information contained in this press release is subject to change without notice and except as required by applicable law or regulation (including to meet the requirements of the Listing Rules of the Financial Conduct Authority, Regulation (EU) 596/2014 (as it forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018, as amended), the Prospectus Regulation Rules of the Financial Conduct Authority and/or the Financial Services and Markets Act 2000, as amended), Oxford Biomedica expressly disclaims any obligation or undertaking to publish any updates or revisions to any forward-looking statements contained in this press release to reflect any changes in Oxford Biomedica’s expectations with regard thereto or any changes in events, conditions or circumstances on which any such statements are based. Statements contained in this press release regarding past trends or activities should not be taken as representation that such trends or activities will continue in the future. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release. No statement in this press release is intended to be a profit forecast and no statement in this press release should be interpreted to mean that earnings per share of Oxford Biomedica for the current or future years would necessarily match or exceed the historical published earnings per share of Oxford Biomedica.
Homology Medicines’ Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our expectations regarding the transaction described above, including anticipated benefits, anticipated payments, future business and market opportunities, anticipated growth resulting from the transaction and the anticipated timing of the closing of the transaction; the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; our beliefs regarding our manufacturing capabilities; our position as a leader in the development of genetic medicines; the sufficiency of our cash and cash equivalents to fund our operations; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we may not realize the anticipated benefits of the collaboration with Oxford Biomedica; the transaction may not closed in the time frame expected or at all; the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption “Risk Factors” in our
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Quarterly Report on Form 10-Q for the quarter ended September 30, 2021 and our other filings with the Securities and Exchange Commission (“the SEC”) could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.
Oxford Biomedica plc Enquiries
T: +44 (0)1865 783 000 / E: ir@oxb.com
Stuart Paynter, Chief Financial Officer
Sophia Bolhassan, Head of Investor Relations
Consilium Strategic Communications:
Mary Jane-Elliott: +44 (0)7739 788 014
Matthew Neal: +44 (0)7720 088 468
Homology Medicines Contacts
Theresa McNeely
Chief Communications Officer
and Patient Advocate
tmcneely@homologymedicines.com
781-301-7277
Media Contact:
Cara Mayfield
Vice President, Patient Advocacy
and Corporate Communications
cmayfield@homologymedicines.com
781-691-3510
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EX-99.2
Exhibit 99.2
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this presentation that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding expectations about the transaction with Oxford Biomedica described herein, including anticipated benefits, anticipated payments, future business and market opportunities, anticipated growth resulting from the transaction and the anticipated timing of the closing of the transaction; the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; our beliefs regarding our manufacturing capabilities; our position as a leader in the development of genetic medicines; the sufficiency of our cash and cash equivalents to fund our operations; our competitive position, business strategy, prospective products, timing, design, results and likelihood of success of studies and/or clinical trials, including the Phase 1/2 pheNIX trial, the Phase 1 pheEDIT trial, the Phase 1 juMPStart trial, and IND-enabling studies and/or planned clinical studies for MLD and PNH, timing for regulatory feedback, the potential of our gene therapy and gene editing platforms, including our new GTx-mAb platform, plans and objectives of management for future operations, manufacturing facility capabilities, the market opportunity for our product candidates, and the potential future uses and effects of our product candidates. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we may not realize the anticipated benefits of the collaboration with Oxford Biomedica; the transaction may not close in the timeframe expected or at all; the fact that we have incurred significant losses since inception and expect to incur losses for the foreseeable future; our need for additional funding, which may not be available; raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or drug candidates; our limited operating history; failure to use our novel genetic medicines platform to identify additional product candidates and develop marketable products; adverse public perception of genetic medicine, and gene editing in particular, may negatively impact regulatory approval of, or demand for, our potential products; the early stage of our development efforts with all programs in the research or preclinical stage; our failure or the failure of our collaborators to successfully develop and commercialize drug candidates; the regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time-consuming and inherently unpredictable; delays or difficulties in the enrollment of patients in clinical trials; our product candidates may cause serious adverse events, side effects, toxicities or have other properties that may delay or prevent their regulatory approval; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; inability to maintain any of our collaborations, or the failure of these collaborations; our reliance on third parties to conduct our preclinical studies and manufacture our drug candidates; our inability to obtain required regulatory approvals; the fact that a Fast Track or Breakthrough Therapy designation by the FDA for our drug candidates may not actually lead to a faster development or regulatory review or approval process; the inability to obtain orphan drug exclusivity for drug candidates; failure to obtain marketing approval in international jurisdictions; failure to obtain U.S. marketing approval; ongoing regulatory obligations, continued regulatory review and any post-marketing restrictions or withdrawals from the market; effects of recently enacted and future legislation; failure to comply with environmental, health and safety laws and regulations; failure to achieve market acceptance by physicians, patients, or third-party payors; failure to establish sales, marketing and distribution capabilities on our own or in collaboration with third parties with such capabilities; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to retain key personnel and attract, retain and motivate qualified personnel; difficulties in managing our growth; the possibility of system failures or security breaches; failure to obtain and maintain patent protection for or otherwise protect our technology and products; effects of patent or other intellectual property lawsuits; the price of our common stock may be volatile and fluctuate substantially; significant costs and required management time as a result of operating as a public company; the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, ongoing and planned clinical trials and ability to access capital; and any securities class action litigation. These and other important factors discussed under the caption “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, and our other filings with the Securities and Exchange Commission could cause actual results to differ materially from those indicated by the forward-looking statements made in this presentation. Any such forward-looking statements represent management’s estimates as of the date of this presentation. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this presentation. This presentation also includes statistical and other industry and market data that we obtained from industry publications and research, surveys and studies conducted by third parties or us. Industry publications and third-party research, surveys and studies generally indicate that their information has been obtained from sources believed to be reliable, although they do not guarantee the accuracy or completeness of such information. All of the market data used in this presentation involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. While we believe these industry publications and third-party research, surveys and studies are reliable, we have not independently verified such data. The industry in which we operate is subject to a high degree of uncertainty, change and risk due to a variety of factors, which could cause results to differ materially from those expressed in the estimates made by the independent parties and by us. 2 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
Homology Medicines and Oxford Biomedica (OXB) to Form New AAV Manufacturing Company – Oxford Biomedica Solutions* OXB to pay Homology $130M upfront and invest Homology to own 20% and $50M in Oxford Biomedica Solutions OXB to own 80% Oxford Biomedica Solutions to incorporate Homology’s AAV manufacturing capabilities Tim Kelly to serve as Chief Executive Officer and team and Board Chair Oxford Biomedica Solutions has the potential to accelerate the mission to improve patients’ lives worldwide *Subject to the satisfaction of certain closing conditions including the requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 1976 5 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
Homology’s Process Development & Manufacturing Platform, Team and GMP Facility to Form Basis of Oxford Biomedica SolutionsTim Kelly as Chief Executive Officer and Board Chair‘Plug and Play’ Manufacturing Process 125 AAV Manufacturing State-of-the-Art and Platform Experts • Full scope of all CMC functions GMP Facility • Allows for rapid new construct • 25,000 square feet of GMP execution and scale-up • Upstream/downstream process space development • Created significant efficiencies • 3x500-liter bioreactors and speed for Homology pipeline • Analytical development • Met CMC requirements for 3 • Manufacturing ops • Producing GMP vector since 2019 with 100% success rate cleared Homology INDs • Quality assurance & control • First to successfully scale • Extensive know-how, and • Breadth of analytical testing & HEK293 AAV to 2,000 liters platform protected by 4 patent characterization leads to high product quality • Additional 23,000 square feet of families space being built out to support • Platform delivers very high- • Preclinical, clinical and new business quality product, which is critical commercial scale experience for the industry 9 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
Oxford Biomedica Solutions – ‘Everything for AAV’ Continue our Successful Culture & Execution • Rapid decision-making, data-driven, “one team,” highly collaborative • High energy & continuous focus on platform innovation & development • Delivery focused model à On-time and 100% success • Committed to deliver cures for genetic diseases for patients • Continue leading the industry in productivity & product quality 10 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
Phase 2 Part: Plans to Report Initial Data Mid-2022 HMI-102 one-time, in vivo • Phase 2 dose expansion gene therapy candidate for • 14 clinical sites with more expected shortly adults with PKU • Positive Phase 1 dose-escalation data Designed to restore natural biochemical pathway with durability • Upcoming presentation at ACMG (Mar.) on patient-centric trial in adults adaptations for COVID-19 13 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
Phase 1 Trial: First Nuclease-Free Gene Editing Study for PKU Goal to Move to Pediatric Patients to Address Rapidly Dividing Livers HMI-103 one-time, in vivo, nuclease-free gene editing • Dose-escalation trial ongoing in adults with PKU candidate for pediatric PKU • Trial update planned for EOY 2022 • Utilizes AAVHSC15, same vector as HMI-102 Designed to integrate functional PAH gene into genome using natural DNA • Encouraging preclinical data repair process of homologous recombination (HR) 14 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
Trial Phase 1, open-label, sequential dose-escalation, first-in-human trial w 3 sequential dose cohorts; 3 adult patients with classical PKU per cohort w 21-day stagger between patient dosing w 82-day screening and run-in period Evaluating safety and efficacy of a one-time intravenous (I.V.) dose of HMI-103 15 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
Phase 1 Trial: Differentiated Strategy to Evaluate Systemic Gene Therapy Candidate for MPS II HMI-203 one-time, in vivo • Dose-escalation trial ongoing focused on unmet need in ERT-treated adults gene therapy candidate for Hunter syndrome • Trial update planned for EOY 2022 • Encouraging preclinical data in MPS II murine model Designed to deliver functional copies • Upcoming presentations at WORLDSymposium™ (Feb.) of IDS gene to peripheral organs and CNS 16 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
Trial Phase 1, open-label, sequential dose-escalation, first-in-human trial w 3 sequential dose cohorts; 3 adult patients with Hunter syndrome currently being treated with ERT per cohort w 21-day stagger between patient dosing w 47-day run-in period Evaluating safety and efficacy of a single dose of HMI-203 including potential to discontinue ERT ERT = Enzyme replacement therapy 17 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
HMI-104 GTx-mAb Development Candidate for PNH Could Overcome Burdens of Current Anti-C5 Therapeutics • IND-enabling studies underway HMI-104 one-time, in vivo • Single I.V. dose in murine model showed: GTx-mAb development • Expression of full-length antibodies consistent with anti-C5 candidate for PNH therapeutic levels • Sustained, robust IgG expression Designed to express consistent • In vivo, vector-expressed C5mAb had potent functional full-length antibody in the liver activity using ex vivo assay against C5 and reduce peaks and troughs inherent with repeated • Potential to expand HMI-104 into additional dosing of antibodies complement-mediated indications and apply GTx-mAb to other targets and into larger therapeutic areas PNH = Paroxysmal nocturnal hemoglobinuria ASGCT 2021. Sharma, et al. 18 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
2022 Anticipated Milestones: Progress Three Clinical Programs and Pipeline PKU • Phase 2 dose expansion gene therapy trial for adults with PKU Gene Therapy • Initial clinical data from Phase 2 anticipated by mid-2022 HMI-102 PKU Gene Editing • Phase 1 dose-escalation trial in PKU HMI-103 MPS II Gene Therapy • Phase 1 dose-escalation trial in MPS II HMI-203 MLD Gene Therapy • Optimizing HMI-202 vector for MLD HMI-202 PNH GTx-mAb • IND-enabling studies with HMI-104 for PNH HMI-104 19 © Copyright 2022 Homology Medicines, Inc. All rights reserved.
© Copyright 2022 Homology Medicines, Inc. All rights reserved.