ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
(State or other jurisdiction of incorporation or organization) |
(I.R.S. Employer Identification No.) | |
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(Address of principal executive offices) |
(Zip Code) |
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered | ||
Large accelerated filer |
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Accelerated filer |
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Non-accelerated filer |
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Smaller reporting company |
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Emerging growth company |
Page |
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PART I |
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Item 1. |
6 |
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Item 1A. |
61 |
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Item 1B. |
120 |
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Item 2. |
120 |
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Item 3. |
120 |
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Item 4. |
120 |
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PART II |
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Item 5. |
121 |
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Item 6. |
121 |
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Item 7. |
122 |
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Item 7A. |
131 |
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Item 8. |
132 |
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Item 9. |
153 |
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Item 9A. |
153 |
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Item 9B. |
153 |
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Item 9C. |
156 |
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PART III |
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Item 10. |
157 |
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Item 11. |
157 |
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Item 12. |
157 |
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Item 13. |
157 |
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Item 14. |
157 |
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PART IV |
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Item 15. |
158 |
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Item 16. |
158 |
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162 |
• | our expectation that our existing capital resources will be sufficient to enable us to fund our planned development of MGTA-117, MGTA-145 and any other product candidates we may identify and pursue; |
• | the initiation, timing and success of clinical trials of MGTA-117, MGTA-145 and any other product candidates; |
• | our ability to commence and enroll patients in our clinical trials at the pace that we project; |
• | regulatory actions with respect to our product candidates or our competitors’ products and product candidates; |
• | the outcomes of our preclinical studies; |
• | our ability to manufacture MGTA-117, MGTA-145 or any other product candidate in conformity with the U.S. Food and Drug Administration’s requirements and to scale up manufacturing of our product candidates to commercial scale, if approved; |
• | whether the results of our trials will be sufficient to support domestic or foreign regulatory approvals for MGTA-117, MGTA-145 or any other product candidates we may develop; |
• | our reliance on third parties to conduct our clinical trials; |
• | our reliance on third-party contract development and manufacturer organizations to manufacture and supply our product candidates for us; |
• | our ability to establish clinical programs moving forward in multiple indications, with a rapidly advancing portfolio and sustainable platform; |
• | our ability to obtain, including on an expedited basis, and maintain regulatory approval of MGTA-117, MGTA-145 or any other product candidates we may develop; |
• | the level of expenses related to any of our product candidates or clinical development programs; |
• | the benefits of the use of MGTA-117, MGTA-145 or any other product candidate, if approved; |
• | our ability to successfully commercialize MGTA-117, MGTA-145 or any other product candidates we may identify and pursue, if approved; |
• | the rate and degree of market acceptance of MGTA-117, MGTA-145 or any other product candidates we may identify and pursue; |
• | our expectations regarding government and third-party payor coverage and reimbursement; |
• | our ability to obtain and maintain intellectual property protection for MGTA-117, MGTA-145 or any other product candidates we may identify and pursue; |
• | our ability to obtain orphan drug designation for any of our product candidates we may identify and pursue; |
• | our ability to successfully build a specialty sales force and commercial infrastructure; |
• | our ability to compete with companies currently producing or engaged in the clinical development of treatments for the disease indications that we pursue and treatment modalities that we develop; |
• | our ability to successfully find collaborators for E478 or any of our current and future programs and product candidates; |
• | our ability to retain and recruit key personnel; |
• | our estimates of our expenses, ongoing losses, future revenue, capital requirements and our needs for or ability to obtain additional financing; |
• | our expectations regarding the time during which we will continue to be an emerging growth company or smaller reporting company as defined in federal securities regulations; |
• | our financial performance; and |
• | developments and projections relating to our competitors or our industry. |
• | We are a clinical stage company with a limited operating history, have incurred significant losses since our inception, and anticipate that we will continue to incur significant losses for the foreseeable future. |
• | We have no products approved for commercial sale and have not generated any revenue from product sales. If we are unable to raise additional capital when needed or on terms acceptable to us, we could be forced to significantly delay, scale back or discontinue our development or commercialization efforts. |
• | Although we have initiated and conducted clinical trials for some of our product candidates, including MGTA-117 and MGTA-145, we have not yet demonstrated the ability to successfully advance our clinical trials for our product candidates through the final regulatory processes and obtain marketing approvals for such products, or to conduct sales and marketing activities necessary for successful commercialization of such products. |
• | If we are unable to obtain regulatory approval for MGTA-117, MGTA-145 or any other product candidates that we may identify or develop, our business will be substantially harmed. |
• | We have not yet demonstrated an ability to manufacture or process drug product on a commercial-scale and may not be able to do so for any of our product candidates. |
• | The results of earlier studies and interim data from our ongoing studies may not be predictive of future clinical trial results, and we may fail to establish an adequate safety or efficacy profile to conduct advanced clinical trials or obtain regulatory approval for MGTA-117, MGTA-145 or any other product candidates that we may pursue. |
• | Stem cell transplant is a high-risk procedure that may result in complications or adverse events for patients in our clinical trials or for patients that use any of our product candidates, if approved. If serious adverse events, undesirable side effects, or unexpected characteristics are identified during the development of any of our product candidates, we may need to limit, delay or abandon our further clinical development of those product candidates, even if such events, effects or characteristics were the result of stem cell transplant or related procedures generally, and not directly or specifically caused or exacerbated by our product candidates. |
• | If we are not able to identify a safe and effective dose for any of our antibody-drug conjugates, or ADCs, we may need to delay, abandon or limit our development of any potential product candidates. |
• | We rely, and expect to continue to rely, on third parties to manufacture our clinical product supplies, and we intend to rely on third parties to produce and process our product candidates, if approved. |
• | We are highly dependent on intellectual property licensed from third parties and termination of any of these licenses could result in the loss of significant rights, which would harm our business. |
• | It is difficult and costly to protect our intellectual property and our proprietary technologies, and we may not be able to ensure their protection. If we are unable to obtain and maintain sufficient intellectual property protection for MGTA-117, MGTA-145 or any of our other current or any future product candidates, or our technologies, we may not be able to compete effectively in our markets. |
• | If we are unable to successfully develop our current programs into a comprehensive portfolio of product candidates, or experience significant delays in doing so, we may not realize the full commercial potential of our current and future product candidates. |
• | The commercial success of any of our product candidates will depend upon the degree of market acceptance by physicians, patients, third-party payors and others in the medical community. |
• | We face substantial competition, including from companies with greater financial, technical, research, manufacturing, marketing, distribution and other resources than us, which may result in others discovering, developing or commercializing products before or more successfully than we do. |
• | We have entered into collaborations and may enter into additional collaborations, strategic alliances or additional licensing arrangements in the future, and we may not realize the benefits of such collaborations, alliances or licensing arrangements. |
• | We are developing E478 specifically to partner with gene therapy and genome editing companies, and if we are unable to find willing collaborators, this may adversely affect the development of E478 and our business. |
• | The coronavirus, or COVID-19, pandemic or any future pandemic, epidemic or outbreak of any other highly infectious disease could have a material adverse effect on our business, financial condition and results of operations. |
• | Our future success depends in part upon our ability to attract and retain highly skilled personnel, including the members of our executive team and key scientific and medical personnel employees. |
• | Changes in tax law could adversely affect our business and financial condition. |
ITEM 1. |
BUSINESS |
• | maximize the patient impact of our portfolio by finding value-creating partnerships to enable gene and cell therapies, including stem cell-based gene therapies, genome editing and CAR-T therapies; |
• | build relationships with partners to access complementary expertise and capabilities to bring our therapies as quickly as possible to all patients who can benefit; and |
• | opportunistically bring in preclinical or clinical assets that fit with our integrated portfolio. |
• | Targeted Conditioning Programs MGTA-117 program is focused on selectively depleting stem cells from patients prior to transplant or HSC-based gene therapy to lessen the need for high-dose or high-intensity chemotherapeutic agents or, in the case of gene therapy applications, to potentially eliminate the need for chemotherapeutic agents altogether. Our second targeted conditioning program, CD45-ADC, is focused on depleting both stem and immune cells to enable transplant as a single agent in autologous autoimmune disease and allogenic blood cancer transplants. |
• | Stem Cell Mobilization & Collection Program MGTA-145 program is focused on enabling rapid, reliable, predictable and safe mobilization and collection of high numbers of functional blood stem cells for transplant. |
• | Research Programs |
• | First, the antibody component of the ADC must specifically target a receptor that is expressed on the cells of interest. |
• | Second, to comply with typical stem cell transplant conditioning timelines, the ADC must have suitable potency to ensure that the agent is able to remove the target cells rapidly, in days rather than weeks or months. |
• | Third, the ADC clearance from the body needs to be accelerated so that it is eliminated by the time the transplanted cells are infused into the patient, typically within a week of starting conditioning. This requirement stems from the fact that the target receptor is expressed on cells present in the patient but also on the newly transplanted cells which should not be targeted for depletion in order for the transplant to be successful. |
• | Finally, the drug must be well-tolerated for patients at dose levels where stem cells are effectively removed. We designed the ADC with a stable linker-payload which is intended to ensure that the payload used for cell depletion is primarily released intracellularly following internalization by target cells. |
• | MGTA-117: |
• | CD45-ADC: |
• | selectively target CD117 as measured by receptor occupancy; |
• | potently deplete CD117-expressing cells such as stem cells, progenitors, and tumor cells; and |
• | rapidly clear from the body with a well-tolerated profile as determined by pharmacokinetic analysis and clinical chemistry tests, respectively. |
• | Lysosomal Storage Disorders. MGTA-117 for conditioning of patients receiving one or more of AVROBIO, Inc.’s investigational lentiviral gene therapies. |
• | Hemoglobinopathies. MGTA-117 for conditioning of patients with sickle cell disease and beta-thalassemia receiving Beam Therapeutics, Inc.’s base editing gene therapies. |
• | mobilization into the peripheral blood, which typically requires several days of injections of a drug or combination of drugs to mobilize the cells, or move them from the bone marrow into the bloodstream, where they are then collected through a process called apheresis; |
• | extraction from the bone marrow in a process known as bone marrow harvest, which requires a procedure performed under general anesthesia where cells are withdrawn directly from the bone marrow with needle aspirates; or |
• | harvesting from umbilical cord blood units, which are stored in cord blood banks. |
• | Medexus Pharmaceuticals, Inc., which is developing treosulfan (reduced intensity conditioning alkylating agent used in allogeneic stem cell transplant) to replace busulfan; and |
• | Jasper Therapeutics, Inc., which is developing an antibody to CD117 that is not conjugated to a payload. |
• | Actinium Pharmaceuticals, Inc., which is developing an antibody to CD45 that is linked to radioisotope iodine-131; and |
• | Molecular Templates Inc., which is developing an antibody to CD45 that is conjugated to engineered Shiga-toxin. |
• | BioLineRx Ltd., which is developing BL-8040, a peptide that functions as a high-affinity antagonist for CXCR4. They have Phase 3 data in multiple myeloma patients; |
• | Yifan Pharmaceutical Co., Ltd., which is developing YF-H-2015005, |
• | TaiGen Biotechnology Co., Ltd. & GPCR Therapeutics, Inc., which are developing burixafor, a CXCR4 receptor antagonist. |
• | Allogene Therapeutics, Inc., which is developing an antibody to CD52 that is not conjugated to any toxin; |
• | Telix Pharmaceuticals Ltd, which is developing a CD66-radioconjugate; |
• | Cellectis S.A., |
• | Precision Biosciences, Inc., which is developing foralumab, a humanized anti-CD3 monoclonal antibody (Phase 1). |
• | completion of extensive preclinical studies in accordance with applicable regulations, including studies conducted in accordance with good laboratory practice, or GLP, requirements; |
• | submission to the FDA of an application for an IND application, which must become effective before human clinical trials may begin; |
• | approval by an institutional review board, or IRB, or independent ethics committee at each clinical trial site before each trial may be initiated; |
• | performance of adequate and well-controlled human clinical trials in accordance with applicable IND regulations, good clinical practice, or GCP, requirements and other clinical trial-related regulations to establish the safety and efficacy of the investigational product for each proposed indication; |
• | submission to the FDA of an NDA or BLA; |
• | a determination by the FDA within 60 days of its receipt of an NDA or BLA to accept the filing for review; |
• | satisfactory completion of one or more FDA pre-approval inspections of the manufacturing facility or facilities where the drug or biologic will be produced to assess compliance with cGMP requirements to assure that the facilities, methods and controls are adequate to preserve the drug or biologic’s identity, strength, quality and purity; |
• | potential FDA audit of the clinical trial sites that generated the data in support of the NDA or BLA; |
• | payment of user fees for FDA review of the NDA or BLA; and |
• | FDA review and approval of the NDA or BLA, including consideration of the views of any FDA advisory committee, prior to any commercial marketing or sale of the drug or biologic in the U.S. |
• | Phase 1 clinical trials generally involve a small number of healthy volunteers or disease-affected patients who are initially exposed to a single dose and then multiple doses of the product candidate. The primary purpose of these clinical trials is to assess the metabolism, pharmacologic action, side effect tolerability and safety of the product candidate. |
• | Phase 2 clinical trials involve studies in disease-affected patients to evaluate proof of concept and/or determine the dose required to produce the desired benefits. At the same time, safety and further pharmacokinetic and pharmacodynamic information is collected, possible adverse effects and safety risks are identified, and a preliminary evaluation of efficacy is conducted. |
• | Phase 3 clinical trials generally involve a large number of patients at multiple sites and are designed to provide the data necessary to demonstrate the product candidate’s safety and effectiveness for its intended use, and to establish the overall benefit/risk relationship of the product candidate and provide an adequate basis for product labeling. |
• | created an annual, nondeductible fee on any entity that manufactures or imports specified branded prescription drugs and biologic products, apportioned among these entities according to their market share in certain government healthcare programs; |
• | expanded eligibility criteria for Medicaid programs by, among other things, allowing states to offer Medicaid coverage to certain individuals with income at or below 133% of the federal poverty level, thereby potentially increasing a manufacturer’s Medicaid rebate liability; |
• | expanded manufacturers’ rebate liability under the Medicaid Drug Rebate Program by increasing the minimum rebate for both branded and generic drugs and revising the definition of “average manufacturer price,” or AMP, for calculating and reporting Medicaid drug rebates on outpatient prescription drug prices; |
• | addressed a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected; |
• | expanded the types of entities eligible for the 340B drug discount program; |
• | established the Medicare Part D coverage gap discount program by requiring manufacturers to provide a 50% point-of-sale-discount, |
• | created a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research. |
• | The centralized MA is issued by the European Commission through the centralized procedure, based on the opinion of the Committee for Medicinal Products for Human Use, or CHMP, of the European Medical Agency, or EMA, and is valid throughout the European Union. The centralized procedure is mandatory for certain types of products, such as biotechnology medicinal products, orphan medicinal products, advanced-therapy medicines (gene-therapy, somatic cell-therapy or tissue-engineered medicines) and medicinal products containing a new active substance indicated for the treatment of |
HIV, AIDS, cancer, neurodegenerative disorders, diabetes, auto-immune and other immune dysfunctions and viral diseases. The centralized procedure is optional for products containing a new active substance not yet authorized in the European Union, or for products that constitute a significant therapeutic, scientific or technical innovation or which are in the interest of public health in the European Union. Under the centralized procedure the maximum timeframe for the evaluation of a marketing authorization application, or MAA, by the EMA is 210 days, excluding clock stops, when additional written or oral information is to be provided by the applicant in response to questions asked by the CHMP. Clock stops may extend the timeframe of evaluation of an MAA considerably beyond 210 days. Where the CHMP gives a positive opinion, it provides the opinion together with supporting documentation to the European Commission, who make the final decision to grant a marketing authorization, which is issued within 67 days of receipt of the EMA’s recommendation. Accelerated assessment might be granted by the CHMP in exceptional cases, when a medicinal product is expected to be of major public health interest, particularly from the point of view of therapeutic innovation. The timeframe for the evaluation of an MAA under the accelerated assessment procedure is 150 days, excluding clock stops, but it is possible that the CHMP may revert to the standard time limit for the centralized procedure if it determines that the application is no longer appropriate to conduct an accelerated assessment. |
• | National MAs, which are issued by the competent authorities of the European Union Member States and only cover their respective territory, are available for products not falling within the mandatory scope of the centralized procedure. Where a product has already been authorized for marketing in a European Union Member State, this national MA can be recognized in other European Union Member States through the mutual recognition procedure. If the product has not received a national MA in any European Union Member State at the time of application, it can be approved simultaneously in various European Union Member States through the decentralized procedure. Under the decentralized procedure an identical dossier is submitted to the competent authorities of each of the European Union Member States in which an MA is sought, one of which is selected by the applicant as the Reference Member State, or RMS. The competent authority of the RMS prepares a draft assessment report, a draft summary of the product characteristics, or SmPC, and a draft of the labeling and package leaflet, which are sent to the other European Union Member States (referred to as the Concerned Member States) for their approval. If the Concerned Member States raise no objections, based on a potential serious risk to public health, to the assessment, SmPC, labeling, or packaging proposed by the RMS, the product is subsequently granted a national MA in all the European Union Member States (i.e., in the RMS and the Concerned Member States). |
• | The Budget Control Act of 2011, among other things, created measures for spending reductions by Congress. A Joint Select Committee on Deficit Reduction, tasked with recommending a targeted deficit reduction of at least $1.2 trillion for the years 2013 through 2021, was unable to reach required goals, thereby triggering the legislation’s automatic reduction to several government programs. These changes included aggregate reductions to Medicare payments to providers of up to 2% per fiscal year. These reductions went into effect on April 1, 2013 and, due to subsequent legislative amendments to the statute, will remain in effect through 2030, with the exception of a temporary suspension from May 1, 2020 through March 31, 2022. Then, a 1% payment reduction will occur beginning April 1, 2022 through June 30, 2022, and the 2% payment reduction will resume on July 1, 2022. |
• | The American Taxpayer Relief Act of 2012, among other things, reduced Medicare payments to several providers, and increased the statute of limitations period for the government to recover overpayments to providers from three to five years. |
• | The Middle Class Tax Relief and Job Creation Act of 2012 required that the Centers for Medicare & Medicaid Services reduce the Medicare clinical laboratory fee schedule by 2% in 2013, which served as a base for 2014 and subsequent years. In addition, effective January 1, 2014, CMS also began bundling the Medicare payments for certain laboratory tests ordered while a patient received services in a hospital outpatient setting. |
ITEM 1A. |
RISK FACTORS |
• | the initiation, progress, timing, costs and results of research, preclinical studies and clinical trials for our product candidates; |
• | the costs to develop, maintain, and enhance a sustainable, scalable, reproducible, and transferable manufacturing process for our product candidates; |
• | the clinical development plans we establish for these product candidates; |
• | the number and characteristics of product candidates that we develop or may in-license; |
• | the cost of milestone or other payments under any current or future license, acquisition, collaboration or other strategic transaction agreements; |
• | the outcome, timing and cost of meeting regulatory requirements established by the FDA, the European Medical Agency, or EMA, and other comparable foreign regulatory authorities; |
• | the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights; |
• | the cost of defending intellectual property disputes, including patent infringement actions brought by third parties against us or our product candidates; |
• | the effect of competing technological and market developments; |
• | the cost and timing of completion of commercial-scale outsourced manufacturing activities; |
• | the cost of seeking to attract, hire and retain skilled personnel; and |
• | the cost of establishing sales, marketing and distribution capabilities for any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products on our own. |
• | identify product candidates and complete research and preclinical and clinical development of any product candidates we may identify; |
• | seek and obtain regulatory and marketing approvals for any of our product candidates for which we complete clinical trials; |
• | launch and commercialize any of our product candidates for which we obtain regulatory and marketing approval by establishing a sales force, marketing, and distribution infrastructure or, alternatively, collaborating with a commercialization partner; |
• | qualify for adequate coverage and reimbursement by government and third-party payors for any of our product candidates for which we obtain regulatory and marketing approval; |
• | develop, maintain, and enhance a sustainable, scalable, reproducible, and transferable manufacturing process for the product candidates we may develop; |
• | establish and maintain supply and manufacturing relationships with third parties that can provide adequate, in both amount and quality, products and services to support clinical development and the market demand for any of our product candidates for which we obtain regulatory and marketing approval; |
• | obtain market acceptance of any product candidates we may develop as viable treatment options; |
• | address competing technological and market developments; |
• | implement internal systems and infrastructure, as needed; |
• | negotiate favorable terms in any collaboration, licensing, or other arrangements into which we may enter and perform our obligations in such collaborations; |
• | maintain, protect, and expand our portfolio of intellectual property rights, including patents, trade secrets, and know-how; |
• | avoid and defend against third-party interference or infringement claims; and |
• | attract, hire, and retain qualified personnel. |
• | successful completion of preclinical studies and successful enrollment and completion of clinical trials, including toxicology studies, biodistribution studies and minimally efficacious dose studies in animals, where applicable, under the FDA’s current Good Clinical Practices, or cGCPs, and the FDA’s current Good Laboratory Practices; |
• | effective IND applications or Clinical Trial Authorizations that allow commencement of our planned clinical trials or future clinical trials for our product candidates; |
• | positive results from our preclinical and clinical programs that support a finding of safety and effectiveness and an acceptable risk-benefit profile of our product candidates in the intended populations; |
• | receipt of regulatory approvals from applicable regulatory authorities; |
• | establishment of arrangements with third-party manufacturers for clinical supply and, where applicable, commercial manufacturing capabilities; |
• | successful development of our internal or external manufacturing processes or transfer to larger-scale facilities operated by either a third-party contract development and manufacturing organization, or CDMO, or by us; |
• | establishment and maintenance of patent and trade secret protection or regulatory exclusivity for our product candidates; |
• | commercial launch of our product candidates, if and when approved, whether alone or in collaboration with others; |
• | acceptance of our product candidates, if and when approved, by patients, the medical community and third-party payors; |
• | effective competition against other therapies; |
• | establishment and maintenance of healthcare coverage and adequate reimbursement; |
• | enforcement and defense of intellectual property rights and claims; and |
• | maintenance of a continued acceptable safety profile of our product candidates following approval. |
• | preclinical study results may show the therapies to be less effective than desired or to have harmful or problematic side effects; |
• | clinical trial results may show the therapies to be less effective than expected (e.g., the trial failed to meet its primary endpoint or the results are not competitive compared to other therapeutic alternatives) or to have unacceptable side effects or toxicities; |
• | failure to receive the necessary regulatory approvals or a delay in receiving such approvals, which delays may be caused by, among other things, slow enrollment in clinical trials, delays due to investigations concerning safety, length of time to achieve study endpoints, additional requirements for data by regulatory agencies, additional time requirements for data analysis, or biologics license application, or BLA, preparation, discussions with the FDA, an FDA request for additional preclinical or clinical data, or unexpected safety or manufacturing issues; |
• | manufacturing costs, formulation issues, pricing or reimbursement issues, or other factors that make the therapy uneconomical; and |
• | the proprietary rights of others and their competing products and technologies that may prevent the therapy from being commercialized. |
• | regulators, IRBs, or ethics committees may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site; |
• | we may experience delays in reaching, or fail to reach, agreement on acceptable terms with prospective trial sites and prospective contract research organization, or CROs, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; |
• | clinical trials of any product candidates may fail to show safety or efficacy, produce negative or inconclusive results and we may decide, or regulators may require us, to conduct additional preclinical studies or clinical trials or we may decide to abandon product development programs; |
• | the number of patients required for clinical trials of any product candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate, or participants may drop out of these clinical trials or fail to return for post-treatment follow-up at a higher rate than we anticipate; |
• | our third-party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all, or may deviate from the clinical trial protocol or drop out of the trial, which may require that we add new clinical trial sites or investigators; |
• | we may elect to, or regulators, IRBs or ethics committees may require, that we or our investigators, suspend or terminate clinical research or trials for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks; |
• | the cost of preclinical studies and clinical trials of any product candidates may be greater than we anticipate; |
• | the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate; and |
• | our product candidates may have undesirable side effects or other unexpected characteristics, causing us or our investigators, regulators, IRBs or ethics committees to suspend or terminate the trials, or reports may arise from preclinical or clinical testing of other blood and immune reset and cell-based therapies that raise safety or efficacy concerns about our product candidates. |
• | the patient eligibility criteria defined in the protocol; |
• | the size of the patient population required for analysis of the trial’s primary endpoints; |
• | the proximity of patients to trial sites; |
• | the design of the trial; |
• | our ability to recruit clinical trial investigators with the appropriate competencies and experience; |
• | our ability to obtain and maintain patient consents; and |
• | the risk that patients enrolled in clinical trials will drop out of the trials before completion. |
• | the research methodology used may not be successful in identifying potential indications and/or product candidates; |
• | potential product candidates may, after further study, be shown to have harmful adverse effects or other characteristics that indicate they are unlikely to be effective drugs; or |
• | it may take greater human and financial resources than we will possess to identify additional therapeutic opportunities for our product candidates or to develop suitable potential product candidates through internal research programs, thereby limiting our ability to develop, diversify and expand our product portfolio. |
• | reliance on the third party for regulatory compliance and quality assurance; |
• | the possible breach of the manufacturing agreement by the third party; |
• | regulatory or judicial termination or modification of our agreement with the third party due to the third party’s insolvency or winddown, a change in regulations or other reason; |
• | the possible misappropriation of our proprietary information, including our trade secrets and know-how; and |
• | the possible termination or nonrenewal of the agreement by the third party at a time that is costly or inconvenient for us. |
• | the efficacy, durability and safety of such product candidates as demonstrated in clinical trials; |
• | the potential and perceived advantages of product candidates over alternative treatments; |
• | the cost of treatment relative to alternative treatments; |
• | our ability to offer the product for sale at competitive prices; |
• | the clinical indications for which the product candidate is approved by the FDA or the EMA; |
• | the product’s convenience and ease of administration compared to alternative treatments; |
• | the willingness of physicians to prescribe new therapies; |
• | the willingness of the target patient population to try new therapies; |
• | the prevalence and severity of any side effects; |
• | product labeling or product insert requirements of the FDA, EMA or other regulatory authorities, including any limitations or warnings contained in a product’s approved labeling; |
• | relative convenience and ease of administration; |
• | the strength of marketing and distribution support; |
• | the timing of market introduction of competitive products; |
• | publicity concerning our products or competing products and treatments; |
• | changes in the standard of care for the targeted indications for the product; and |
• | sufficient third-party payor coverage and adequate reimbursement. |
• | a covered benefit under its health plan; |
• | safe, effective and medically necessary; |
• | appropriate for the specific patient; |
• | cost-effective; and |
• | neither experimental nor investigational. |
• | the scope of rights, if any, granted under the license agreement and other interpretation-related issues; |
• | whether and the extent to which our technology and processes infringe on intellectual property of the licensor that is not subject to the license agreement; |
• | whether our licensor or its licensor had the right to grant the license agreement; |
• | whether third parties are entitled to compensation or equitable relief, such as an injunction, for our use of the intellectual property without their authorization; |
• | our right to sublicense patent and other rights to third parties under collaborative development relationships; |
• | whether we are complying with our obligations with respect to the use of the licensed technology in relation to our development and commercialization of product candidates; |
• | our involvement in the prosecution of the licensed patents and our licensors’ overall patent enforcement strategy; |
• | the allocation of ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and by us and our partners; and |
• | the amounts of royalties, milestones or other payments due under the license agreement. |
• | the USPTO and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other provisions during the patent process. There are situations in which noncompliance can result in abandonment or lapse of a patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction. In such an event, competitors might be able to enter the market earlier than would otherwise have been the case; |
• | patent applications may not result in any patents being issued; |
• | patents that may be issued or in-licensed may be challenged, invalidated, modified, revoked, circumvented, found to be unenforceable or otherwise may not provide any competitive advantage; |
• | our competitors, many of whom have substantially greater resources and many of whom have made significant investments in competing technologies, may seek or may have already obtained patents that will limit, interfere with or eliminate our ability to make, use, and sell our potential product candidates; |
• | there may be significant pressure on the U.S. government and international governmental bodies to limit the scope of patent protection both inside and outside the U.S. for disease treatments that prove successful, as a matter of public policy regarding worldwide health concerns; and |
• | countries other than the U.S. may have patent laws less favorable to patentees than those upheld by U.S. courts, allowing foreign competitors a better opportunity to create, develop and market competing product candidates. |
• | others may be able to make products that are similar to any product candidates we may develop or utilize similar technology but that are not covered by the claims of the patents that we license or own; |
• | we, or our current or future licensors might not have been the first to make the inventions covered by the issued patent or pending patent application that we license or own; |
• | we, or our current or future licensors might not have been the first to file patent applications covering certain of our or their inventions; |
• | others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our owned or licensed intellectual property rights; |
• | it is possible that our pending owned or licensed patent applications or those that we may own or license in the future will not lead to issued patents; |
• | issued patents that we hold rights to may be held invalid or unenforceable, including as a result of legal challenges by our competitors; |
• | our competitors might conduct research and development activities in countries where we do not have patent rights and then use the information learned from such activities to develop competitive products for sale in our major commercial markets; |
• | we may not develop additional proprietary technologies that are patentable; |
• | the patents of others may harm our business; and |
• | we may choose not to file a patent in order to maintain certain trade secrets or know-how, and a third party may subsequently file a patent covering such intellectual property. |
• | Collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations. |
• | Collaborators may not pursue development and commercialization of any product candidates we may develop or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborator’s strategic focus, available funding or external factors such as an acquisition that diverts resources or creates competing priorities. |
• | Collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials, or require a new formulation of a product candidate for clinical testing. |
• | A collaborator’s product candidate may have a safety or efficacy profile that would impact the collaborator’s ability to continue to pursue the development and commercialization of its product candidate, which in turn, would negatively impact our ability to continue to pursue the development and commercialization of our product candidate. |
• | Collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our medicines or product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours. |
• | Collaborators with marketing and distribution rights to one or more medicines may not commit sufficient resources to the marketing and distribution of such medicine or medicines. |
• | Collaborators may not properly obtain, maintain, enforce, or defend our intellectual property or proprietary rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our proprietary information or expose us to potential litigation. |
• | Disputes may arise between the collaborators and us that result in the delay or termination of the research, development, or commercialization of our medicines or product candidates or that result in costly litigation or arbitration that diverts management attention and resources. |
• | We may lose certain valuable rights under circumstances identified in our collaborations, including if we undergo a change of control. |
• | Collaborations may be terminated and, if terminated, may result in a need for additional capital to pursue further development or commercialization of the applicable product candidates. |
• | Collaboration agreements may not lead to development or commercialization of product candidates in the most efficient manner or at all. If a present or future collaborator of ours were to be involved in a business combination, the continued pursuit and emphasis on our product development or commercialization program under such collaboration could be delayed, diminished, or terminated. |
• | The COVID-19 pandemic has had, and will likely continue to have, an adverse impact on various aspects of our ongoing and planned clinical trials, and preclinical studies. |
• | Other potential impacts of the COVID-19 pandemic on our various clinical trials include impacts on patient dosing and study monitoring, which may be paused or delayed due to changes in policies at various clinical sites; federal, state, local or foreign laws, rules and regulations, including quarantines or other travel restrictions; the prioritization of healthcare resources toward pandemic efforts, including diminished attention from physicians serving as our clinical trial investigators and reduced availability of site staff supporting the conduct of our clinical trials; and interruption or delays in the operations of the U.S. Food and Drug Administration, or FDA, among other reasons related to the COVID-19 pandemic. If the COVID-19 pandemic continues, other aspects of our clinical trials will likely be adversely affected, delayed or interrupted, including, for example, site initiation, patient recruitment and enrollment, availability of clinical trial materials and data analysis. Some patients and clinical investigators may not be able to comply with clinical trial protocols and patients may choose to withdraw from our studies or we may choose to, or be required to, pause enrollment and or patient dosing in our ongoing clinical trials in order to preserve health resources and protect trial participants. It is unknown how long these pauses or disruptions could continue. |
• | We currently rely on third parties, including CROs, CDMOs, and other contractors and consultants to, among other things, conduct our preclinical and clinical trials, manufacture raw materials, manufacture and supply our product candidates, ship investigational drugs and clinical trial samples, perform quality testing and supply other goods and services to run our business. If any such third party is adversely impacted by restrictions resulting from the COVID-19 pandemic, including staffing shortages, production slowdowns and disruptions in delivery systems, our supply chain may be disrupted, which could limit our ability to manufacture our product candidates for our clinical trials and conduct our research and development operations. |
• | We have established a work-from-home policy for all employees, as well as safety measures for those using our offices and laboratory facilities that are designed to comply with applicable federal, state and local guidelines instituted in response to the COVID-19 pandemic. Our increased reliance on personnel working from home may negatively impact productivity, or disrupt, delay, or otherwise adversely impact our business. In addition, this could increase our cyber security risk, create data accessibility concerns and make us more susceptible to communication disruptions, any of which could adversely impact our business operations or delay necessary interactions with local and federal regulators, ethics committees, manufacturing sites, research or clinical trial sites and other important agencies and contractors. |
• | Our employees and contractors conducting non-business critical research and development activities have not been able to, and may not in the future be able to, access our laboratory for an extended period of time as a result of the current work-from-home policy and the possibility that governmental authorities further modify current restrictions. This could delay timely completion of preclinical activities, including completing Investigational New Drug, or IND, enabling studies or our ability to |
select future development candidates, and initiation of additional clinical trials for our other product candidates. |
• | Certain government agencies, such as health regulatory agencies and patent offices, within the U.S. or internationally have experienced, and may continue to experience, disruptions in their operations as a result of the COVID-19 pandemic. The FDA and comparable foreign regulatory agencies may have slower response times or be under-resourced to continue to monitor our clinical trials and, as a result, review, inspection and other timelines may be materially delayed. It is unknown how long these disruptions could continue. Any elongation or de-prioritization of our clinical trials or delay in regulatory review resulting from such disruptions could materially affect the development and study of our product candidates. For example, regulatory authorities may require that we not distribute a product candidate lot until the relevant agency authorizes its release. Such release authorization may be delayed as a result of the COVID-19 pandemic, which would likely result in delays to our ongoing clinical trials. |
• | The trading prices for our common stock and those of other biopharmaceutical companies have been highly volatile, partly due to the COVID-19 pandemic. As a result, we may face difficulties raising capital through sales of our common stock or such sales may be on unfavorable terms. In addition, a recession, depression or other sustained adverse market event resulting from the COVID-19 pandemic could materially and adversely affect our business and the value of our common stock. |
• | identifying, recruiting, integrating, maintaining and motivating additional employees; |
• | managing our internal development efforts effectively, including the clinical, FDA and international regulatory review process for our product candidates, while complying with our contractual obligations to contractors and other third parties; and |
• | improving our operational, financial and management controls, reporting systems and procedures. |
• | decreased demand for our products; |
• | injury to our reputation; |
• | withdrawal of clinical trial participants and inability to continue clinical trials; |
• | initiation of investigations by regulators; |
• | costs to defend the related litigation; |
• | a diversion of management’s time and our resources; |
• | substantial monetary awards to trial participants or patients; |
• | product recalls, withdrawals or labeling, marketing or promotional restrictions; |
• | loss of revenue; |
• | exhaustion of any available insurance and our capital resources; |
• | the inability to commercialize any product candidate; and |
• | a decline in our share price. |
• | the success of existing or new competitive products or technologies; |
• | regulatory actions with respect to our product candidates or our competitors’ products and product candidates; |
• | announcements by us or our competitors of significant acquisitions, strategic partnerships, joint ventures, collaborations or capital commitments; |
• | the timing and results of preclinical studies for any of our product candidates; |
• | the timing and results of clinical trials of MGTA-145, MGTA-117 and any other product candidates; |
• | commencement or termination of collaborations for E478 or any of our current and future programs and product candidates; |
• | failure or discontinuation of any of our development programs; |
• | results of clinical trials of product candidates of our competitors; |
• | regulatory or legal developments in the U.S. and other countries; |
• | developments or disputes concerning patent applications, issued patents or other proprietary rights; |
• | the recruitment or departure of key personnel; |
• | the level of expenses related to any of our product candidates or clinical development programs; |
• | the results of our efforts to develop additional product candidates or products; |
• | actual or anticipated changes in estimates as to financial results or development timelines; |
• | announcement or expectation of additional financing efforts; |
• | sales of our common stock by us, our insiders or other stockholders; |
• | variations in our financial results or those of companies that are perceived to be similar to us; |
• | changes in estimates or recommendations by securities analysts, if any, that cover us; |
• | changes in the structure of healthcare payment systems; |
• | market conditions in the pharmaceutical and biotechnology sectors; |
• | disruptions to political, governmental or regulatory systems, including shutdowns of the government and its agencies; |
• | general economic, industry and market conditions; and |
• | the other factors described in this “Risk Factors” section. |
• | establish a classified board of directors such that all members of the board are not elected at one time; |
• | allow the authorized number of our directors to be changed only by resolution of our board of directors; |
• | limit the manner in which stockholders can remove directors from the board; |
• | establish advance notice requirements for nominations for election to the board of directors or for proposing matters that can be acted on at stockholder meetings; |
• | require that stockholder actions must be effected at a duly called stockholder meeting and prohibit actions by our stockholders by written consent; |
• | limit who may call a special meeting of stockholders; |
• | authorize our board of directors to issue preferred stock without stockholder approval, which could be used to institute a “poison pill” that would work to dilute the stock ownership of a potential hostile acquirer, effectively preventing acquisitions that have not been approved by our board of directors; and |
• | require the approval of the holders of at least 66.67% of the votes that all our stockholders would be entitled to cast to amend or repeal certain provisions of our charter or bylaws. |
ITEM 1B. |
UNRESOLVED STAFF COMMENTS |
ITEM 2. |
PROPERTIES |
ITEM 3. |
LEGAL PROCEEDINGS |
ITEM 4. |
MINE SAFETY DISCLOSURES |
ITEM 5. |
MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES |
ITEM 6. |
RESERVED |
ITEM 7. |
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS |
• | initiate, enroll and conduct a Phase 1/2 clinical trial for MGTA-117 and Phase 2 clinical trials for MGTA-145 |
• | initiate and conduct preclinical studies and clinical trials of our other product candidates; |
• | develop any other future product candidates we may choose to pursue; |
• | seek marketing approval for any of our product candidates that successfully complete clinical development, if any; |
• | maintain compliance with applicable regulatory requirements; |
• | develop and scale up our capabilities to support our ongoing preclinical activities and clinical trials for our product candidates and commercialization of any of our product candidates for which we obtain marketing approval, if any; |
• | maintain, expand, protect and enforce our intellectual property portfolio; |
• | develop and expand our sales, marketing and distribution capabilities for our product candidates for which we obtain marketing approval, if any; and |
• | expand our operational, financial and management systems and increase personnel, including to support our clinical development and commercialization efforts and our operations as a public company. |
• | employee-related expenses, including salaries and related costs, and stock-based compensation expense, for employees engaged in research and development functions; |
• | expenses incurred in connection with the preclinical and clinical development of our product candidates, including under agreements with contract research organizations, or CROs; |
• | the cost of consultants and third-party contract development and manufacturing organizations, or CDMOs, that manufacture drug products for use in our preclinical studies and clinical trials; |
• | facilities, depreciation and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance and supplies; and |
• | payments made under third-party licensing agreements. |
• | the continuing impact of the COVID-19 pandemic on our industry, the healthcare system, and our current and future operations; |
• | successful completion of preclinical studies and clinical trials; |
• | receipt and related terms of marketing approvals from applicable regulatory authorities; |
• | raising additional funds necessary to complete clinical development of and commercialize our product candidates; |
• | obtaining and maintaining patent, trade secret and other intellectual property protection and regulatory exclusivity for our product candidates; |
• | making arrangements with third-party manufacturers, or establishing manufacturing capabilities, for both clinical and commercial supplies of our product candidates; |
• | developing and implementing marketing and reimbursement strategies; |
• | establishing sales, marketing and distribution capabilities and launching commercial sales of our products, if and when approved, whether alone or in collaboration with others; |
• | acceptance of our products, if and when approved, by patients, the medical community and third-party payors; |
• | effectively competing with other therapies; |
• | obtaining and maintaining third-party coverage and adequate reimbursement; |
• | protecting and enforcing our rights in our intellectual property portfolio; and |
• | maintaining a continued acceptable safety profile of the products following approval. |
• | vendors in connection with the preclinical development activities; |
• | CROs in connection with preclinical and clinical trials; |
• | CDMOs in connection with the production of preclinical and clinical trial materials; and |
• | investigative sites in connection with clinical trials. |
Year Ended December 31, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Operating expenses: |
||||||||||||
Research and development |
$ | 46,766 | $ | 50,615 | $ | (3,849 | ) | |||||
General and administrative |
27,926 | 28,087 | (161 | ) | ||||||||
|
|
|
|
|
|
|||||||
Total operating expenses |
74,692 | 78,702 | (4,010 | ) | ||||||||
|
|
|
|
|
|
|||||||
Loss from operations |
(74,692 | ) | (78,702 | ) | 4,010 | |||||||
Interest and other income, net |
3,556 | 3,766 | (210 | ) | ||||||||
|
|
|
|
|
|
|||||||
Net loss |
$ | (71,136 | ) | $ | (74,936 | ) | $ | 3,800 | ||||
|
|
|
|
|
|
Year Ended December 31, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Direct research and development expenses by program: |
||||||||||||
Conditioning |
$ | 9,677 | $ | 16,127 | $ | (6,450 | ) | |||||
Mobilization |
5,203 | 4,066 | 1,137 | |||||||||
Cell therapy |
684 | 4,398 | (3,714 | ) | ||||||||
Unallocated expenses: |
||||||||||||
Personnel related (including stock-based compensation) |
18,418 | 14,848 | 3,570 | |||||||||
Consultant (including stock-based compensation) |
1,488 | 1,196 | 292 | |||||||||
Facility related and other |
11,296 | 9,980 | 1,316 | |||||||||
|
|
|
|
|
|
|||||||
Total research and development expenses |
$ | 46,766 | $ | 50,615 | $ | (3,849 | ) | |||||
|
|
|
|
|
|
Year Ended December 31, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Personnel related (including stock-based compensation) |
$ | 13,902 | $ | 14,219 | $ | (317 | ) | |||||
Professional and consultant |
6,555 | 7,290 | (735 | ) | ||||||||
Facility related and other |
7,469 | 6,578 | 891 | |||||||||
|
|
|
|
|
|
|||||||
Total general and administrative expenses |
$ | 27,926 | $ | 28,087 | $ | (161 | ) | |||||
|
|
|
|
|
|
Year Ended December 31, |
||||||||
2021 |
2020 |
|||||||
(in thousands) |
||||||||
Cash used in operating activities |
$ | (59,531 | ) | $ | (64,023 | ) | ||
Cash provided by (used in) investing activities |
43,428 | (10,635 | ) | |||||
Cash provided by financing activities |
89,601 | 67,739 | ||||||
|
|
|
|
|||||
Net increase (decrease) in cash, cash equivalents and restricted cash |
$ | 73,498 | $ | (6,919 | ) | |||
|
|
|
|
ITEM 7A. |
QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK |
ITEM 8. |
CONSOLIDATED FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA |
Page(s) |
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133 | ||||
134 | ||||
135 | ||||
136 | ||||
137 | ||||
138 |
December 31, |
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2021 |
2020 |
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Assets |
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Current assets: |
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Cash and cash equivalents |
$ | $ | ||||||
Marketable securities |
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Prepaid expenses and other current assets |
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Total current assets |
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Restricted cash |
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Property and equipment, net |
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Total assets |
$ | $ | ||||||
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Liabilities and Stockholders’ Equity |
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Current liabilities: |
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Accounts payable |
$ | $ | ||||||
Accrued expenses and other current liabilities |
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Total current liabilities |
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Deferred rent |
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Total liabilities |
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Commitments and contingencies (Note 8) |
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Stockholders’ Equity: |
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Preferred stock, $ |
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Common stock, $ |
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Additional paid-in capital |
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Accumulated other comprehensive loss |
( |
) | ( |
) | ||||
Accumulated deficit |
( |
) | ( |
) | ||||
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Total stockholders’ equity |
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Total liabilities and stockholders’ equity |
$ | $ | ||||||
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Year Ended December 31, |
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2021 |
2020 |
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Operating expenses: |
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Research and development |
$ | $ | ||||||
General and administrative |
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Total operating expenses |
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Loss from operations |
( |
) | ( |
) | ||||
Interest and other income, net |
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Net loss |
$ | ( |
) | $ | ( |
) | ||
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Net loss per share, basic and diluted |
$ | ( |
) | $ | ( |
) | ||
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Weighted average common shares outstanding, basic and diluted |
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Comprehensive loss: |
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Net loss |
$ | ( |
) | $ | ( |
) | ||
Other comprehensive loss: |
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Unrealized losses on marketable securities |
( |
) | ( |
) | ||||
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Total other comprehensive loss |
( |
) | ( |
) | ||||
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Total comprehensive loss |
$ | ( |
) | $ | ( |
) | ||
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Common Stock |
Additional Paid-in |
Accumulated Other Comprehensive |
Accumulated |
Total Stockholders’ |
||||||||||||||||||||
Shares |
Amount |
Capital |
Income (Loss) |
Deficit |
Equity |
|||||||||||||||||||
Balances at December 31, 2019 |
$ | $ | $ | $ | ( |
) | $ | |||||||||||||||||
Issuance of common stock upon public offering net of underwriting discounts, commissions and offering costs |
— | — | ||||||||||||||||||||||
Vesting of restricted stock |
— | — | — | — | — | |||||||||||||||||||
Issuance of common stock upon exercise of stock options |
— | — | ||||||||||||||||||||||
Issuance of common stock under Employee Stock Purchase Plan |
— | — | — | |||||||||||||||||||||
Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
Unrealized losses on marketable securities |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
Net loss |
— | — | — | — | ( |
) | ( |
) | ||||||||||||||||
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|||||||||||||
Balances at December 31, 2020 |
( |
) | ( |
) | ||||||||||||||||||||
Issuance of common stock upon private investment, net of offering costs |
— | — | ||||||||||||||||||||||
Vesting of restricted stock |
— | — | — | — | — | |||||||||||||||||||
Issuance of common stock upon exercise of stock options |
— | — | — | |||||||||||||||||||||
Issuance of common stock under Employee Stock Purchase Plan |
— | — | — | |||||||||||||||||||||
Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
Unrealized losses on marketable securities |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
Net loss |
— | — | — | — | ( |
) | ( |
) | ||||||||||||||||
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|
|||||||||||||
Balances at December 31, 2021 |
$ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||
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|
|
Year ended December 31, |
||||||||
2021 |
2020 |
|||||||
Cash flows from operating activities: |
||||||||
Net loss |
$ | ( |
) | $ | ( |
) | ||
Adjustments to reconcile net loss to net cash used in operating activities: |
||||||||
Stock-based compensation expense |
||||||||
Depreciation and amortization expense |
||||||||
Loss on disposal of property and equipment |
||||||||
Net amortization of premiums on marketable securities |
||||||||
Changes in operating assets and liabilities: |
||||||||
Prepaid expenses and other current assets |
( |
) | ||||||
Accounts payable |
( |
) | ||||||
Accrued expenses and other current liabilities |
( |
) | ||||||
Deferred rent |
||||||||
|
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|
|
|||||
Net cash used in operating activities |
( |
) | ( |
) | ||||
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|
|||||
Cash flows from investing activities: |
||||||||
Purchases of property and equipment |
( |
) | ( |
) | ||||
Purchases of marketable securities |
( |
) | ( |
) | ||||
Maturities of marketable securities |
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|||||
Net cash provided by (used in) investing activities |
( |
) | ||||||
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|
|||||
Cash flows from financing activities: |
||||||||
Proceeds from private investment |
— | |||||||
Proceeds from public offerings, net of underwriting discounts and commissions |
— | |||||||
Payments of offering costs |
( |
) | ( |
) | ||||
Proceeds from exercise of common stock options |
||||||||
Proceeds from issuance of common stock under Employee Stock Purchase Plan |
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|
|
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|
|||||
Net cash provided by financing activities |
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|
|||||
Net increase (decrease) in cash, cash equivalents and restricted cash |
( |
) | ||||||
Cash, cash equivalents and restricted cash at beginning of period |
||||||||
|
|
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|
|||||
Cash, cash equivalents and restricted cash at end of period |
$ | $ | ||||||
|
|
|
|
Estimated Useful Life | ||
Lab equipment |
||
Computer equipment |
||
Furniture and fixtures |
||
Leasehold improvements |
Shorter of life of lease or estimated useful life |
• | Level 1—Quoted prices in active markets for identical assets or liabilities. |
• | Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. |
• | Level 3—Unobservable inputs that are supported by little or no market activity and that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. |
As of December 31, |
||||||||
2021 |
2020 |
|||||||
Stock options to purchase common stock |
||||||||
Unvested restricted common stock and units |
||||||||
Shares of common stock issuable under Employee Stock Purchase Plan |
||||||||
|
|
|
|
|||||
|
|
|
|
Amortized Cost |
Gross Unrealized Gains |
Gross Unrealized Losses |
Estimated Fair Value |
|||||||||||||
U.S. treasury notes (due within one year) |
$ | $ | $ | ( |
) | $ | ||||||||||
U.S. treasury Notes (due after one year through two years) |
( |
) | ||||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total |
$ | |
$ | |
$ | ( |
) |
$ | |
|||||||
|
|
|
|
|
|
|
|
Amortized Cost |
Gross Unrealized Gains |
Gross Unrealized Losses |
Estimated Fair Value |
|||||||||||||
U.S. treasury notes (due within one year) |
$ | $ | $ | ( |
) | $ | ||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total |
$ | |
$ | |
$ | ( |
) |
$ | |
|||||||
|
|
|
|
|
|
|
|
Fair Value Measurements at December 31, 2021 Using: |
||||||||||||||||
Level 1 |
Level 2 |
Level 3 |
Total |
|||||||||||||
Cash equivalents: |
||||||||||||||||
Money market funds |
$ | $ | — | $ | — | $ | ||||||||||
Marketable securities: |
||||||||||||||||
U.S. treasury notes |
— | — | ||||||||||||||
Total |
$ | |
$ | |
$ | — | $ | |
||||||||
Fair Value Measurements at December 31, 2020 Using: |
||||||||||||||||
Level 1 |
Level 2 |
Level 3 |
Total |
|||||||||||||
Cash equivalents: |
||||||||||||||||
Money market funds |
$ | $ | — | $ | — | $ | ||||||||||
U.S. treasury notes |
— | — | ||||||||||||||
Marketable securities: |
||||||||||||||||
U.S. treasury notes |
— | — | ||||||||||||||
Total |
$ | |
$ | |
$ | — | $ | |
||||||||
December 31, |
||||||||
2021 |
2020 |
|||||||
Laboratory and computer equipment |
$ | $ | ||||||
Furniture and fixtures |
||||||||
Leasehold improvements |
||||||||
Less: Accumulated depreciation and amortization |
( |
) | ( |
) | ||||
$ | $ | |||||||
December 31, |
||||||||
2021 |
2020 |
|||||||
Accrued payroll and related expenses |
$ | $ | ||||||
Accrued external research and development expenses |
||||||||
Deferred rent, current portion |
||||||||
Accrued professional fees |
||||||||
Accrued other |
||||||||
$ | $ | |||||||
Year Ended December 31, |
||||||||
2021 |
2020 |
|||||||
Risk-free interest rate |
% | % | ||||||
Expected term (in years) |
||||||||
Expected volatility |
% | % | ||||||
Expected dividend yield |
% | % |
Number of Shares |
Weighted Average Exercise Price |
Weighted Average Remaining Contractual Term |
Aggregate Intrinsic Value |
|||||||||||||
(in years) |
(in thousands) |
|||||||||||||||
Outstanding as of December 31, 2020 |
$ | $ | ||||||||||||||
Granted |
$ | |||||||||||||||
Exercised |
( |
) | $ | |||||||||||||
Forfeited |
( |
) | $ | |||||||||||||
|
|
|||||||||||||||
Outstanding as of December 31, 2021 |
$ | $ | — | |||||||||||||
|
|
|||||||||||||||
Options vested and expected to vest as of December 31, 2021 |
$ | $ | — | |||||||||||||
|
|
|||||||||||||||
Options exercisable as of December 31, 2021 |
$ | $ | — | |||||||||||||
|
|
Number of Shares |
Weighted Average Grant Date Fair Value |
|||||||
Outstanding as of December 31, 2020 |
$ |
|||||||
Vested |
( |
) | $ | |||||
Forfeited |
— | |||||||
|
|
|||||||
Outstanding as of December 31, 2021 |
— | |||||||
|
|
Number of Shares |
Weighted Average Grant Date Fair Value |
|||||||
Outstanding as of December 31, 2020 |
$ | |||||||
Granted |
$ | |||||||
Vested |
( |
) | $ | |||||
Forfeite d |
( |
) | $ | |||||
|
|
|||||||
Outstanding as of December 31, 2021 |
$ | |||||||
|
|
Number of Shares |
Weighted Average Grant Date Fair Value |
|||||||
Outstanding as of December 31, 2020 |
$ | |||||||
Granted |
$ | |||||||
Vested |
( |
) | $ | |||||
Forfeited |
( |
) | $ | |||||
Outstanding as of December 31, 2021 |
$ | |||||||
Year Ended December 31, |
||||||||
2021 |
2020 |
|||||||
Research and development expenses |
$ | $ | ||||||
General and administrative expenses |
||||||||
$ | $ | |||||||
2022 |
$ | |||
2023 |
||||
2024 |
||||
2025 |
||||
2026 |
||||
Thereafter |
||||
$ |
||||
Year Ended December 31, |
||||||||
2021 |
2020 |
|||||||
Federal statutory income tax rate |
% | % | ||||||
State taxes, net of federal benefit |
||||||||
Research and orphan drug tax credits |
||||||||
Other |
( |
) | ||||||
Increase in deferred tax asset valuation allowance |
( |
) | ( |
) | ||||
|
|
|
|
|||||
Effective income tax rate |
% | % | ||||||
|
|
|
|
December 31, |
||||||||
2021 |
2020 |
|||||||
Deferred tax assets: |
||||||||
Net operating loss carryforwards |
$ | $ | ||||||
Capitalized research and development expenses |
||||||||
Research and orphan drug tax credit carryforwards |
||||||||
Stock compensation expense |
||||||||
Accrued expense |
||||||||
Other |
||||||||
|
|
|
|
|||||
Total deferred tax assets |
||||||||
Valuation allowance |
( |
) | ( |
) | ||||
|
|
|
|
|||||
Net deferred tax assets |
||||||||
|
|
|
|
|||||
Deferred tax liabilities: |
||||||||
Depreciation and amortization |
( |
) | ( |
) | ||||
|
|
|
|
|||||
Total deferred tax liabilities |
( |
) | ( |
) | ||||
|
|
|
|
|||||
Net deferred tax assets and liabilities |
$ | $ | ||||||
|
|
|
|
Year Ended December 31, |
||||||||
2021 |
2020 |
|||||||
Valuation allowance as of beginning of year |
$ | $ | ||||||
Net increases recorded to income tax provision |
||||||||
|
|
|
|
|||||
Valuation allowance as of end of year |
$ | $ | ||||||
|
|
|
|
ITEM 9. |
CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE |
ITEM 9A. |
CONTROLS AND PROCEDURES |
ITEM 9B. |
OTHER INFORMATION |
ITEM 9C. |
DISCLOSURE REGARDING FOREIGN JURISDICTIONS THAT PREVENT INSPECTIONS |
ITEM 10. |
DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE |
ITEM 11. |
EXECUTIVE COMPENSATION |
ITEM 12. |
SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS |
ITEM 13. |
CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTOR INDEPENDENCE |
ITEM 14. |
PRINCIPAL ACCOUNTING FEES AND SERVICES |
ITEM 15. |
EXHIBITS, FINANCIAL STATEMENT SCHEDULES |
ITEM 16. |
FORM 10-K SUMMARY |
Exhibit Number |
Description | |
101SCH* | Inline XBRL Taxonomy Extension Schema Document. | |
101CAL* | Inline XBRL Taxonomy Extension Calculation Linkbase Document. | |
101LAB* | Inline XBRL Taxonomy Extension Labels Linkbase Document. | |
101PRE* | Inline XBRL Taxonomy Extension Presentation Linkbase Document. | |
101DEF* | Inline XBRL Taxonomy Extension Definition Linkbase Document. | |
104* | Cover Page Interactive Data File (formatted as Inline XBRL with applicable taxonomy extension information contained in Exhibits 101). |
* | Filed herewith. |
** | Furnished herewith. |
# | Represents management compensation plan, contract or arrangement. |
† | Application has been made to the Securities and Exchange Commission for confidential treatment of certain provisions. Omitted material for which confidential treatment has been requested has been filed separately with the Securities and Exchange Commission. |
^ | Portions of this exhibit have been omitted in accordance with the rules of the Securities and Exchange Commission. |
MAGENTA THERAPEUTICS, INC. | ||||||
Date: March 8, 2022 |
By: | /s/ Stephen Mahoney | ||||
Stephen Mahoney | ||||||
Chief Financial and Operating Officer (Principal Financial and Accounting Officer) |
Signature |
Title |
Date | ||
/s/ Jason Gardner, D.Phil. |
President, Chief Executive Officer and Director (Principal Executive Officer) |
March 8, 2022 | ||
Jason Gardner, D.Phil. | ||||
/s/ Stephen Mahoney |
Chief Financial and Operating Officer (Principal Financial and Accounting Officer) |
March 8, 2022 | ||
Stephen Mahoney | ||||
/s/ Jeffrey Albers |
Director | March 8, 2022 | ||
Jeffrey Albers | ||||
/s/ Bruce Booth, D.Phil. |
Director | March 8, 2022 | ||
Bruce Booth, D.Phil. | ||||
/s/ Alexis A. Borisy |
Director | March 8, 2022 | ||
Alexis A. Borisy | ||||
/s/ Thomas O. Daniel, M.D. |
Director | March 8, 2022 | ||
Thomas O. Daniel, M.D. | ||||
/s/ Alison F. Lawton |
Director | March 8, 2022 | ||
Alison F. Lawton | ||||
/s/ Anne M. McGeorge |
Director | March 8, 2022 | ||
Anne M. McGeorge | ||||
/s/ Amy L. Ronneberg |
Director | March 8, 2022 | ||
Amy L. Ronneberg | ||||
/s/ David T. Scadden, M.D. |
Director | March 8, 2022 | ||
David T. Scadden, M.D. |