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 |
(I.R.S. Employer | |
incorporation or organization |
Identification No.) | |
(Address of principal executive offices) |
(Zip Code) |
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered | ||
Large accelerated filer | ☐ | Accelerated filer | ☐ | |||||
☒ | Smaller reporting company | |||||||
Emerging growth company |
Page |
||||||
PART I |
||||||
ITEM 1. |
1 |
|||||
ITEM 1A. |
39 |
|||||
ITEM 1B. |
100 |
|||||
ITEM 2. |
100 |
|||||
ITEM 3. |
100 |
|||||
ITEM 4. |
100 |
|||||
PART II. |
||||||
ITEM 5. |
101 |
|||||
ITEM 6. |
101 |
|||||
ITEM 7. |
102 |
|||||
ITEM 7A. |
113 |
|||||
ITEM 8. |
114 |
|||||
ITEM 9. |
141 |
|||||
ITEM 9A. |
141 |
|||||
ITEM 9B. |
142 |
|||||
ITEM 9C. |
143 |
|||||
PART III |
||||||
ITEM 10. |
144 |
|||||
ITEM 11. |
144 |
|||||
ITEM 12. |
144 |
|||||
ITEM 13. |
144 |
|||||
ITEM 14. |
144 |
|||||
PART IV. |
||||||
ITEM 15. |
145 |
|||||
ITEM 16. |
146 |
|||||
147 |
• |
the sufficiency of our existing cash and cash equivalents and marketable securities to fund our future operating expenses and capital expenditure requirements; |
• |
our ability to obtain funding for our operations, including funding necessary to develop and commercialize ACU193, subject to necessary regulatory approvals; |
• |
the ability of our clinical trials to demonstrate the safety and efficacy of ACU193, and other positive results; |
• |
the therapeutic potential of ACU193, including its potential for improved safety and efficacy, as compared to other monoclonal antibodies approved and or in development, as well as the expectations concerning the INTERCEPT-AD trial; |
• |
the success, cost and timing of our development activities, nonclinical studies and clinical trials; |
• |
the timing and focus of our future clinical trials, and the reporting of data from those trials; |
• |
our plans relating to commercializing ACU193, subject to obtaining necessary regulatory approvals; |
• |
our ability to attract and retain key scientific and clinical personnel; |
• |
our ability to contract with third-party suppliers and manufacturers and their ability to perform adequately; |
• |
our reliance on third parties to conduct clinical trials of ACU193, and for the manufacture of ACU193 for nonclinical studies and clinical trials; |
• |
the success of competing therapies that are or may become available; |
• |
our plans and ability to obtain or protect our intellectual property rights, including extensions of existing patent terms where available or the use of data market exclusivity to provide protection from generic or biosimilar versions of our product; |
• |
the scope of protection we are able to establish and maintain for intellectual property rights covering ACU193 and technology; |
• |
potential claims relating to our intellectual property; |
• |
existing regulations and regulatory developments in the United States and other jurisdictions; |
• |
our ability to obtain and maintain regulatory approval of ACU193, and any related restrictions, limitations and/or warnings in the label of any approved product candidate; |
• |
our plans relating to the further development and manufacturing of ACU193, including additional therapeutic indications which we may pursue; |
• |
our ability to develop and maintain our corporate infrastructure, including our ability to design and maintain an effective system of internal controls; |
• |
our financial performance; |
• |
the effects of the ongoing COVID-19 pandemic, geopolitical events such as the pending conflict with Russia and Ukraine; and |
• |
our expectations regarding the time during which we will be an emerging growth company under the JOBS Act. |
• |
We are a clinical-stage biopharmaceutical company with a limited operating history. |
• |
We have no product candidates approved for commercial sale, we have never generated any revenue from product sales and we may never be profitable. |
• |
We will require substantial additional funding to finance our operations, complete the development and commercialization of ACU193 for Alzheimer’s disease, or AD, and evaluate future product candidates. If we are unable to raise this funding when needed, we may be forced to delay, reduce or eliminate our drug development programs or other operations. |
• |
We are substantially dependent on the success of ACU193, our sole product candidate, which will require significant clinical testing before we can seek regulatory approval and potentially launch commercial sales, and which may not be successful in clinical trials, receive regulatory approval or be successfully commercialized, even if approved. |
• |
We have concentrated our research and development efforts on the treatment of AD, a field that has to date seen very limited success in drug development. |
• |
Our approach to the potential treatment of AD is based on a novel therapeutic approach, which exposes us to unforeseen risks. |
• |
Nonclinical and clinical drug development involves a lengthy, expensive and uncertain process. The results of nonclinical studies and early clinical trials are not always predictive of future results. ACU193 or any other product candidate that we advance into clinical trials may not achieve favorable results in later clinical trials, if any, or receive marketing approval. |
• |
Clinical failure can occur at any stage of clinical development and we have never completed a clinical trial or submitted a biologics license application, or BLA, or marketing authorization application, or MAA. |
• |
We may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development and commercialization of our product candidates. |
• |
We currently rely on contract manufacturing organizations, or CMOs, to manufacture and formulate ACU193. The loss of any of these CMOs or the failure of any of them to meet their obligations to us could affect our ability to develop ACU193 in a timely manner. |
• |
We intend to rely on contract research organizations, or CROs, and other third parties to conduct, supervise and monitor a significant portion of our research and nonclinical testing and clinical trials for our product candidates, and if those third parties do not successfully carry out their contractual duties, comply with regulatory requirements or otherwise perform satisfactorily, we may not be able to obtain regulatory approval or commercialize product candidates, or such approval or commercialization may be delayed, and our business may be substantially harmed. |
• |
We face significant competition in an environment of rapid technological and scientific change, and there is a possibility that our competitors may achieve regulatory approval before us or develop therapies that are safer or more effective than ours. |
• |
If we are unable to enter into a commercial collaboration or, alternatively, establish internal sales, marketing and distribution capabilities, for ACU193 or any other product candidate that may receive regulatory approval, we may not be successful in commercializing those product candidates if and when they are approved. |
• |
If we are unable to obtain and maintain sufficient intellectual property protection for ACU193 and any future product candidate, and other proprietary technologies we develop, or if the scope of the intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize products similar or identical to ours, and our ability to successfully commercialize our product candidate, and other proprietary technologies if approved, may be adversely affected. |
• | Rapidly advance ACU193 through clinical development in patients with early AD. |
• | Evaluate combination approaches to complement our core ACU193 monotherapy strategy. |
• | Selectively explore potential of ACU193 for other diseases. |
• | Expand our portfolio by developing additional molecules. |
• | Optimize value of ACU193 and future drug candidates in major markets. |
Measured Outcome** |
solanezumab EXPEDITION 3 (Phase 3) |
Aduhelm aducanumab EMERGE (Phase 3) |
Aduhelm aducanumab ENGAGE† (Phase 3) |
lecenamab BAN2401 (Phase 2) |
donanemab (Phase 2) |
|||||||||||||||
ADAS-cog |
-11 | % | -27 | % | -12 | % | -47 | % | -39 | % | ||||||||||
ADCS-ADL |
-15 | % | -40 | % | -18 | % | N.A. | -23 | % | |||||||||||
CDR-SB |
-15 | % | -23 | % | 2 | % | -26 | % | -23 | % | ||||||||||
MMSE |
-13 | % | -15 | % | 3 | % | N.A. | -21 | % | |||||||||||
iADRS |
-11 | % | N.A. | N.A. | N.A. | -32 | % |
* | Percent Slowing= [1- [(endpoint score-baseline score)active/(endpoint score-baseline score)placebo]]*100%*(-1) |
** | ADAS-cog: Alzheimer’s Disease Assessment Scale – Cognitive Subscale |
†: | ENGAGE Post-Protocol Version 4 – patients who received at least 14 doses of 10 mg/kg, High Dose cohort achieved 27% improvement on CDR-SB compared to placebo. |
Targeting Aß Monomers |
Targeting Amyloid Plaques |
|||||||||||||||||||||||||||||||||||||||||||||||
solanezumab EXPEDITION 3 (Phase 3) |
Aduhelm aducanumab EMERGE (Phase 3) |
Aduhelm aducanumab ENGAGE (Phase 3) |
Lecenamab BAN2401 (Phase2) |
donanemab (Phase 2) |
||||||||||||||||||||||||||||||||||||||||||||
PC |
Treated |
PC |
Low |
High |
PC |
Low |
High |
PC |
High |
PC |
Treated |
|||||||||||||||||||||||||||||||||||||
ARIA-E |
0.2 | % | 0.1 | % | 2.2 | % | 26.1 | % | 34.4 | % | 3.0 | % | 25.6 | % | 35.7 | % | 0.8 | % | 9.9 | % | 0.8 | % | 27.5 | % | ||||||||||||||||||||||||
ApoE e 4 carriers |
1.9 | % | 29.8 | % | 42.5 | % | 2.4 | % | 28.7 | % | 41.8 | % | 1.2 | % | 14.6 | % | 3.6 | % | 44.0 | % | ||||||||||||||||||||||||||||
ApoE e 4 non-carriers |
2.9 | % | 18.1 | % | 17.9 | % | 4.3 | % | 17.5 | % | 27.7 | % | 0.0 | % | 8.0 | % | ||||||||||||||||||||||||||||||||
Any ARIA E or H |
10.3 | % | 32.8 | % | 41.2 | % | 9.8 | % | 30.7 | % | 40.3 | % | N.A. | 8.0 | % | 38.9 | % |
* | Shows the absence of ARIA after treatment with antibodies targeting Aß monomers (solanezumab) in comparison to the increasing presence of ARIA after treatment at increasing dose levels with antibodies targeting amyloid plaques (aducanumab, BAN2401, and donanemab), indicating that ARIA results from the removal of amyloid plaques around blood vessels and likely does not result from treatment with antibodies that target other species of Aß, i.e. Aß monomers and AßOs. |
Target Selectivity+ |
ARIA Profile | |||||||||
Product Candidate |
Amyloid plaque |
Aß fibrils |
Aß monomers |
Aß oligomers |
Lack of ARIA | |||||
ACU193 |
x | untested | x | ✓ | ✓ | |||||
Aduhelm aducanumab |
✓ | ✓ | x | ✓ | x | |||||
lecanemab BAN2401 |
✓ | ✓ | x | ✓ | x | |||||
gantenerumab |
✓ | ✓ | x | ✓ | x | |||||
donanemab |
✓ | untested | x | x | x | |||||
solanezumab* |
x | x | ✓ | x | ✓ | |||||
crenezumab* |
✓ | ✓ | ✓ | ✓ | ✓ | |||||
bapineuzumab* |
✓ | ✓ | ✓ | ✓ | x |
* | Phase 3 discontinued for primary AD indication |
+ | There have been no head-to-head clinical trials between any of the product candidates listed above. Study designs and protocols for each product candidate were different, and results may not be comparable between product candidates. |
• | Potentially addresses an underlying cause of AD |
• | Selectively binds to A ßO. |
• | Binds to a broad spectrum of toxic A ßOs. |
• | May provide symptomatic improvement in addition to disease modification. |
• | Selectivity for A ßOs is likely to result in greatly reduced rates of ARIA, allowing a broad therapeutic window. |
Selectivity | • Binding characterization studies of ACU193 and its murine parent ACU3B3 show significant preferential selectivity for AßOs versus monomeric and amyloid plaques. – Competitive enzyme-linked immunosorbent assay, or ELISA, data show ACU193 binding to AßOs is 556-fold greater than to Aß monomer based on monomer equivalent weight. This selectivity value is even higher when adjusted for AßOs’ molecular weight. – ACU193 binding in brain tissues of transgenic mice following IV dosing shows significant, preferential binding to non-fibrillar, thioflavin-S-negative Aß structures. The occasional co-localization observed with thioflavin-S-positive fibrillar plaques suggests binding to AßOs reported to surround amyloid plaques. • ACU193 binds a broad range of synthetic and human-derived low, mid, and higher molecular weight AßOs (dimers to 200-mers). | |
Protection from AßO-induced synaptic toxicity | • ACU193 and ACU3B3 prevent binding of AßOs to primary hippocampal neurons in vitro (the average IC50 of ACU193 is 17 nM). • ACU193 and ACU3B3 block AßOs’ inhibition of long-term potentiation ex vivo. • ACU3B3 blocks AßO mediated intracellular calcium influx in vitro. | |
In vivo pharmacology | • ACU193 crosses the blood-brain barrier and demonstrates dose-dependent target engagement in the brain following peripheral administration of antibody. • Blinded studies in transgenic mouse models for AD over a broad range of ages show that treatment with ACU3B3 reduces multiple behavioral deficits. • QPS study of nine- to ten-month-old transgenic mice treated weekly with 20 mg/kg ACU3B3 for four weeks demonstrated significant behavioral improvements during water maze learning test. • Stanford University study of five- to seven-month-old transgenic mice treated weekly with 20 and 30 mg/kg ACU3B3 showed significant improvements during open field and Y-maze tests after four to five weeks of treatment. • Gladstone Institute studies of younger three- to five-month-old transgenic mice with sub-chronic weekly administration of ACU3B3 demonstrated significant behavioral improvements to hyperactivity, emotional response alterations and procedural learning deficits. | |
Safety | • GLP studies via IV route established no observed adverse effect level, or NOAEL, of 300 mg/kg/dose in a 14-week cynomolgus monkey study and 250 mg/kg/dose in a 28-day rat study.• Studies in transgenic mice indicated low potential for cerebral microhemorrhage. |
• | Cohort 1: One IV dose of ACU193 (2 mg/kg) or placebo. |
• | Cohort 2: One IV dose of ACU193 (10 mg/kg) or placebo. |
• | Cohort 3: One IV dose of ACU193 (25 mg/kg) or placebo. |
• | Cohort 4: One IV dose of ACU193 (60 mg/kg) or placebo. |
• | Cohort 5: One IV dose of ACU193 (10 mg/kg) or placebo once every four weeks. |
• | Cohort 6: One IV dose of ACU193 (60 mg/kg) or placebo once every four weeks. |
• | Cohort 7: One IV dose of ACU193 (60 mg/kg) or placebo once every two weeks. |
• | safety and immunogenicity, including assessment for ARIA; |
• | pharmacokinetics in plasma; |
• | determination of CSF concentrations of ACU193; |
• | evaluation of central target engagement as measured by levels of ACU193 AßO complex in CSF; |
• | evaluation of possible changes in concentration of biomarkers for AD in CSF or blood; |
• | evaluation of possible changes in amyloid plaque load as determined by PET imaging; |
• | evaluation of possible changes in cerebral blood flow as determined by MRI imaging, using Arterial Spin Labeling (ASL) pulse sequence; and |
• | evaluation of possible changes in cognitive, functional, and behavioral measures using computerized testing and standard clinical measures for AD. |
• | completion of preclinical laboratory tests and animal studies performed in accordance with the FDA’s Good Laboratory Practice requirements, or GLPs; |
• | submission to the FDA of an investigational new drug application, or IND, which must become effective before clinical trials may begin and must be updated annually and when certain changes are made; |
• | approval by an institutional review board, or IRB, or independent ethics committee at each clinical site before the trial is commenced; |
• | performance of adequate and well-controlled human clinical trials in accordance with Good Clinical Practice, or GCP, requirements and other clinical trial-related regulations to establish the safety, purity and potency of the proposed biologic product candidate for its intended purpose; |
• | preparation of and submission to the FDA of a biologics license application, or BLA, after completion of all pivotal clinical trials; |
• | payment of user fees for FDA review of the BLA; |
• | satisfactory completion of an FDA Advisory Committee review, if applicable; |
• | a determination by the FDA within 60 days of its receipt of a BLA to file the application for review; |
• | satisfactory completion of an FDA pre-approval inspection of the manufacturing facility or facilities at which the proposed product is produced to assess compliance with current Good Manufacturing Practices, or cGMPs, and to assure that the facilities, methods and controls are adequate to preserve the biological product’s continued safety, purity and potency, and of selected clinical investigation sites to assess compliance with GCPs; and |
• | FDA review and approval of the BLA to permit commercial marketing of the product for particular indications for use in the United States. |
• | Phase 1—The investigational product is initially introduced into a limited population of healthy human subjects or patients with the target disease or condition. These studies are designed to test the safety, dose response, absorption, metabolism and distribution of the investigational product in humans, the side effects associated with increasing doses, and, if possible, to gain early evidence on effectiveness. |
• | Phase 2—The investigational product is administered to a limited patient population with a specified disease or condition to evaluate the preliminary efficacy, optimal dosages and dosing schedule and to identify possible adverse side effects and safety risks. Multiple Phase 2 clinical trials may be conducted to obtain information prior to beginning larger and more expensive Phase 3 clinical trials. |
• | Phase 3—The investigational product is administered to an expanded patient population to further evaluate dosage, to provide statistically significant evidence of clinical efficacy and to further test for safety, generally at multiple geographically dispersed clinical trial sites. These clinical trials |
are intended to establish the overall risk/benefit ratio of the investigational product and to provide an adequate basis for product approval. Generally, two adequate and well-controlled Phase 3 clinical trials are required by the FDA for approval of a BLA. |
• | restrictions on the marketing or manufacturing of the product, complete withdrawal of the product from the market or product recalls; |
• | safety alerts, Dear Healthcare Provider letters, press releases or other communications containing warnings or other safety information about the product; |
• | fines, warning letters, or untitled letters; |
• | clinical holds on clinical studies; |
• | refusal of the FDA to approve pending applications or supplements to approved applications, or suspension or revocation of product license approvals; |
• | product seizure or detention, or refusal to permit the import or export of products; |
• | consent decrees, corporate integrity agreements, debarment or exclusion from federal healthcare programs; |
• | mandated modification of promotional materials and labeling and the issuance of corrective information; and |
• | and the imposition of civil or criminal penalties. |
• | the federal Anti-Kickback Statute, which prohibits, among other things, individuals or entities from knowingly and willfully soliciting, receiving, offering or paying any remuneration (including any kickback, bribe or rebate), directly or indirectly, overtly or covertly, in cash or in kind in return for, or to induce, either the referral of an individual, or the purchase, lease, order or arrangement for or recommendation of the purchase, lease, order or arrangement for any good, facility, item or service for |
which payment may be made, in whole or in part, under a federal healthcare program, such as the Medicare and Medicaid programs. The term “remuneration” has been broadly interpreted to include anything of value. Although there are a number of statutory exceptions and regulatory safe harbors protecting some common activities from prosecution, the exceptions and safe harbors are drawn narrowly; |
• | the federal civil and criminal false claims laws, including, without limitation, the federal False Claims Act, which can be enforced by private citizens through civil whistleblower or qui tam actions, and civil monetary penalty laws which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment or approval from the federal government, including Medicare, Medicaid and other government payors, that are false or fraudulent or knowingly making, using or causing to be made or used a false record or statement material to a false or fraudulent claim or to avoid, decrease or conceal an obligation to pay money to the federal government. In addition, the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the federal False Claims Act; |
• | the federal Health Insurance Portability and Accountability Act, or HIPAA, which created additional federal criminal statutes which prohibit, among other things, a person from knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program, including private third-party payors and knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent statement in connection with the delivery of or payment for healthcare benefits, items or services. Similar to the federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation; |
• | HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act and their implementing regulations, also imposes obligations, including mandatory contractual terms, on “covered entities,” including certain healthcare providers, health plans, healthcare clearinghouses, and their respective “business associates,” that create, receive, maintain or transmit individually identifiable health information for or on behalf of a covered entity as well as their covered subcontractors, with respect to safeguarding the privacy, security and transmission of individually identifiable health information, as well as analogous state and foreign laws that govern the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts; |
• | the federal transparency laws, including the federal Physician Payments Sunshine Act, which requires certain manufacturers of drugs, medical devices, biologicals and medical supplies for which payment is available under Medicare, Medicaid or the State Children’s Health Insurance Program, with specific exceptions, to report annually to the Centers for Medicare and Medicaid Services, or CMS, information related to: (i) payments or other “transfers of value’’ made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners) and teaching hospitals, and (ii) ownership and investment interests held by physicians and their immediate family members; and |
• | analogous state and foreign laws and regulations; state laws that require manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers, marketing expenditures or drug pricing; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government, or that otherwise restrict payments that may be made to healthcare providers; and state and local laws that require the registration of pharmaceutical sales representatives. |
• | increased the minimum level of Medicaid rebates payable by manufacturers of brand name drugs from 15.1% to 23.1% of the average manufacturer price; |
• | required collection of rebates for drugs paid by Medicaid managed care organizations; |
• | required manufacturers to participate in a coverage gap discount program, under which they must agree to offer 70 percent point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D; and |
• | imposed a non-deductible annual fee on pharmaceutical manufacturers or importers who sell “branded prescription drugs” to specified federal government programs. |
• | the progress, costs, timing and results of INTERCEPT-AD and other potential clinical trials of ACU193, including for potential additional indications that we may pursue beyond AD; |
• | the requirements of the U.S. Food and Drug Administration, or the FDA, and European Medicines Agency, or the EMA, for clinical trials and nonclinical studies and other work, for review and approval of ACU193 for AD; |
• | the outcome, costs and timing of seeking and obtaining FDA, EMA and any other regulatory approvals; |
• | the number and characteristics of product candidates that we pursue; |
• | our ability to obtain sufficient quantities of our product candidates from our third-party manufacturers; |
• | our need to expand our research and development activities; |
• | the costs associated with securing and establishing commercialization capabilities if we were to elect to commercialize one or more products on our own; |
• | the economics and other terms, timing of and success of any collaboration, licensing or other arrangements into which we may enter for the commercialization of our products; |
• | the costs and other terms, timing and success, of acquiring, in-licensing or investing in businesses, product candidates and technologies; |
• | our ability to maintain, expand and defend the scope of our intellectual property portfolio, including the amount and timing of any payments we may be required to make, or that we may receive, in connection with the licensing, filing, prosecution, defense and enforcement of any patents or other intellectual property rights; |
• | our need and ability to retain management and hire scientific and clinical personnel; |
• | the effect of competing drugs and product candidates and other market developments; and |
• | our need to implement additional internal systems and infrastructure, including financial and reporting systems. |
• | successful patient enrollment in INTERCEPT-AD and other clinical trials of ACU193; |
• | sufficiency of our financial and other resources to complete the necessary clinical trials; |
• | the results from INTERCEPT-AD and future clinical trials of ACU193; |
• | the frequency and severity of adverse effects of ACU193; |
• | the ability of third-party manufacturers to manufacture supplies of ACU193 and to develop, validate and maintain a commercial-scale manufacturing process that is compliant with current good manufacturing practices, or cGMP; |
• | our ability to demonstrate ACU193’s safety and efficacy to the satisfaction of the FDA and foreign regulatory authorities in order to receive necessary marketing approvals for ACU193; |
• | whether we are required by the FDA to conduct additional clinical trials prior to the approval to market ACU193 and whether the FDA may disagree with the number, design, size, conduct, implementation or other aspects of our clinical trials; |
• | whether the FDA may require implementation of a Risk Evaluation and Mitigation Strategy, or REMS, as a condition of approval or post-approval; |
• | our ability to successfully commercialize ACU193, if approved for marketing and sale by the FDA or foreign regulatory authorities, whether alone or in collaboration with others; |
• | our success in educating physicians and patients about the benefits, administration and use of ACU193; |
• | acceptance of ACU193 as safe and effective by patients and the medical community; |
• | the availability, perceived advantages, relative cost, relative safety and relative efficacy of alternative and competing treatments; |
• | achieving and maintaining compliance with all regulatory requirements applicable to ACU193, including any required post-marketing approval commitments; |
• | effectively competing with other AD therapies; |
• | the effectiveness of our own or any future collaborators’ marketing, pricing, coverage and reimbursement, sales and distribution strategies and operations; |
• | our ability to maintain our existing patents and obtain newly issued patents that cover ACU193 and to enforce such patents and other intellectual property rights in and to ACU193; |
• | our ability to avoid third-party intellectual property claims; |
• | the availability of third-party coverage and adequate reimbursement for ACU193 and any other product candidates, once approved; and |
• | a continued acceptable safety, tolerability and efficacy profile of ACU193 following approval. |
• | regulatory authorities, Institutional Review Boards, or IRBs, or Ethics Committees, or ECs, may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site or we may fail to reach a consensus with regulatory authorities on trial design; for example, our initial submission of the IND for ACU193 was placed on clinical hold by the FDA until we were able to address the FDA’s initial concerns regarding potential off-target binding of ACU193 with an additional nonclinical tissue cross reactivity study, after which the FDA permitted us to initiate the Phase 1 clinical trial of ACU193 in the second quarter of 2021; |
• | regulatory authorities in jurisdictions in which we seek to conduct clinical trials may differ from each other on our trial design, and it may be difficult or impossible to satisfy all such authorities with one approach; |
• | we may have delays in reaching or fail to reach agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different contract research organizations, or CROs, and trial sites; |
• | we may be unable to add or be delayed in adding a sufficient number of clinical trial sites and obtaining IRB or independent EC approval at each clinical trial site; |
• | clinical trials of our product candidates may fail to show safety or efficacy or otherwise produce negative or inconclusive results, and we may decide, or regulatory authorities may require us, to conduct additional clinical trials or abandon drug development programs; |
• | the number of patients required for clinical trials of our product candidates may be larger than we anticipate; |
• | enrollment in our clinical trials may be slower than we anticipate or participants may drop out of these clinical trials at a higher rate than we anticipate; |
• | difficulties in having subjects complete a clinical trial or returning for post-treatment follow-up; |
• | changes to clinical trial protocols; |
• | our third-party contractors, including clinical investigators, contract manufacturers and vendors may fail to comply with applicable regulatory requirements, lose their licenses or permits, or otherwise fail, or lose the ability to, meet their contractual obligations to us in a timely manner, or at all; |
• | we might have to suspend or terminate clinical trials of our product candidates for various reasons, including a finding that the participants are being exposed to unacceptable health risks; |
• | regulatory authorities or IRBs may require that we or our investigators suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements, a finding that our product candidates have undesirable side effects or other unexpected characteristics, or that the participants are being exposed to unacceptable health risks; |
• | the cost of clinical trials of our product candidates may be greater than we anticipate, and we may lack adequate funding to continue one or more clinical trials; |
• | 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; |
• | clinical trial sites may deviate from clinical trial protocol or drop out of a clinical trial; and |
• | occurrence of serious adverse events in trials of the same class of agents conducted by other companies. |
• | regulatory authorities may withdraw, suspend or limit approval of ACU193 or any future product candidates; |
• | we may be required to recall a drug or change the way such drug is administered to patients; |
• | regulatory authorities may require additional warnings or statements in the labeling, such as a boxed warning or a contraindication or issue safety alerts, press releases or other communications containing warnings or other safety information about the product candidate, for example, field alerts to physicians and pharmacies; |
• | regulatory authorities may require us to implement a REMS to ensure that the benefits of the drug outweigh its risks, which could include medication guides, physician communication plans, or elements to assure safe use, such as restricted distribution methods, patient registries and other risk minimization tools; |
• | we may be required to change the way a drug is distributed or administered, conduct additional clinical trials or be required to conduct additional post-marketing studies or surveillance; |
• | we may be subject to regulatory investigations and government enforcement actions; |
• | we may decide to remove such product candidates from the market; |
• | we could be sued and held liable for harm caused to patients; |
• | sales of the drug may decrease significantly or ACU193 or any future drug could become less competitive; and |
• | our reputation may suffer. |
• | the severity and difficulty of diagnosing the disease under investigation; |
• | the eligibility and exclusion criteria for the trial in question; |
• | the size of the patient population and process for identifying patients; |
• | our ability to recruit clinical trial investigators with the appropriate competencies and experience; |
• | the design of the trial protocol; |
• | the perceived risks and benefits of the product candidate in the trial, including relating to cell therapy approaches; |
• | the availability of competing commercially available therapies and other competing therapeutic candidates’ clinical trials for the disease or condition under investigation; |
• | the willingness of patients to be enrolled in our clinical trials; |
• | the efforts to facilitate timely enrollment in clinical trials; |
• | further disruptions caused by the ongoing COVID-19 pandemic, including further difficulties in initiating clinical sites, enrolling and retaining participants, diversion of healthcare resources away from clinical trials, travel or quarantine policies that may be implemented, and other factors; |
• | the patient referral practices of physicians; |
• | the ability to monitor patients adequately during and after treatment; and |
• | the proximity and availability of clinical trial sites for prospective patients. |
• | interruptions in our ability to obtain drug supply for our clinical trials; |
• | interruptions in our ability to obtain clinical test kits for our clinical trials; |
• | delays in receiving authorizations from regulatory authorities to initiate our planned clinical trials; |
• | further delays, difficulties or a suspension in clinical site initiation, including difficulties in recruiting clinical site investigators and clinical site staff; |
• | further delays or difficulties in enrolling and retaining patients in our clinical trials; |
• | diversion of healthcare resources away from the conduct of clinical trials, including the diversion of hospitals serving as our clinical trial sites and hospital staff supporting the conduct of our clinical trials; |
• | changes in local regulations as part of a response to the COVID-19 pandemic that may require us to change the ways in which our clinical trials are conducted, which may result in unexpected costs or to discontinue the clinical trials altogether; |
• | interruption of key clinical trial activities, such as clinical trial site monitoring, and the ability or willingness of subjects to travel to trial sites due to limitations on travel imposed or recommended by federal or state governments, employers and others; |
• | risk that participants enrolled in our clinical trials will contract COVID-19 while the trial is ongoing, which could impact the results of the clinical trial, including by increasing the number of observed adverse events; |
• | limitations in employee resources that would otherwise be focused on the conduct of our clinical trials, including because of sickness of employees or their families or the desire of employees to avoid contact with large groups of people; |
• | interruptions of, or delays in receiving, supplies of our product candidates from our contract manufacturing organizations, or CMOs, due to staffing shortages, production slowdowns or stoppages and disruptions in delivery systems; |
• | delays in necessary interactions with local regulators, ethics committees and other important agencies and contractors due to limitations in employee resources or forced furlough of government employees; and |
• | refusal of the FDA to accept data from clinical trials in these affected geographies. |
• | the clinical indications for which our product candidates are licensed; |
• | the efficacy, safety and potential advantages compared to alternative treatments; |
• | our ability to demonstrate the advantages of our product candidates over other medicines; |
• | our ability to offer our products for sale at competitive prices; |
• | the convenience and ease of administration compared to alternative treatments; |
• | product labeling or product insert requirements of the FDA, EMA or other foreign regulatory authorities, including any limitations or warnings contained in a product’s approved labeling, including any black box warning or REMS; |
• | the willingness of the target patient population to try new treatments and of physicians to prescribe these treatments; |
• | our ability to commercialize the product either in collaboration with a third party or on our own; |
• | the timing of market introduction of our product candidates as well as competitive products; |
• | the strength of marketing and distribution support; |
• | the availability of third-party coverage and adequate reimbursement for ACU193 and any other product candidates, once approved; |
• | the prevalence and severity of any side effects; and |
• | any restrictions on the use of our products together with other medications. |
• | our inability to recruit, train and retain adequate numbers of effective sales and marketing personnel; |
• | the inability of sales personnel to obtain access to physicians in order to educate physicians about our product candidates, once approved; |
• | the lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines; and |
• | unforeseen costs and expenses associated with creating an independent sales and marketing organization. |
• | decreased demand for any product candidates or drugs that we may develop; |
• | injury to our reputation and significant negative media attention; |
• | withdrawal of clinical trial participants; |
• | significant costs to defend the related litigation; |
• | substantial monetary awards paid to trial participants or patients; |
• | loss of revenue; |
• | reduced resources of our management to pursue our business strategy; and |
• | the inability to commercialize any products that we may develop. |
• | reduced control for certain aspects of manufacturing activities; |
• | termination or nonrenewal of the applicable manufacturing and service agreements in a manner or at a time that is costly or damaging to us; |
• | the possible breach by our third-party manufacturers and service providers of our agreements with them; |
• | the failure of our third-party manufacturers and service providers to comply with applicable regulatory requirements; |
• | disruptions to the operations of our third-party manufacturers and service providers caused by conditions unrelated to our business or operations, including the bankruptcy of the manufacturer or service provider; and |
• | the possible misappropriation of our proprietary information, including our trade secrets and know-how. |
• | collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations; |
• | collaborators may not perform their obligations as expected; |
• | collaborators may not pursue development and commercialization of any product candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus or available funding, or external factors, such as an acquisition, that divert resources or create 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; |
• | collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our 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; |
• | we could grant exclusive rights to our collaborators that would prevent us from collaborating with others; |
• | product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or drugs, which may cause collaborators to cease to devote resources to the commercialization of our product candidates; |
• | a collaborator with marketing and distribution rights to one or more of our product candidates that achieve regulatory approval may not commit sufficient resources to the marketing and distribution of such products; |
• | disagreements with collaborators, including disagreements over proprietary rights, contract interpretation or the preferred course of development, might cause delays or termination of the research, development or commercialization of product candidates, might lead to additional responsibilities for us with respect to product candidates, or might result in litigation or arbitration, any of which would be time-consuming and expensive; |
• | collaborators may not properly maintain or defend our or their intellectual property rights or may use our or their proprietary information in such a way as to invite litigation that could jeopardize or invalidate such intellectual property or proprietary information or expose us to potential litigation; |
• | collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability; and |
• | collaborations may be terminated for the convenience of the collaborator and, if terminated, we could be required to raise additional capital to pursue further development or commercialization of the applicable product candidates. |
• | differing regulatory requirements for product approvals internationally; |
• | potentially reduced protection for intellectual property rights; |
• | potential third-party patent rights in countries outside of the United States; |
• | the potential for so-called “parallel importing,” which is what occurs when a local seller, faced with relatively high local prices, opts to import goods from another jurisdiction with relatively low prices, rather than buying them locally; |
• | pricing pressure and differing reimbursement regimes: |
• | unexpected changes in tariffs, trade barriers and regulatory requirements; |
• | economic weakness, including inflation, or political instability, particularly in non-U.S. economies and markets, including several countries in Europe; |
• | compliance with tax, employment, immigration and labor laws for employees traveling abroad; |
• | taxes in other countries; |
• | foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country; |
• | workforce uncertainty in countries where labor unrest is more common than in the United States; |
• | production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and |
• | business interruptions resulting from geo-political actions, including war and terrorism, or natural disasters, including earthquakes, volcanoes, typhoons, pandemics, epidemics, floods, hurricanes and fires. |
• | the United States Patent and Trademark Office, or USPTO, and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other provisions during the patent process, the noncompliance with which can result in abandonment or lapse of a patent or patent application, and partial or complete loss of patent rights in the relevant jurisdiction; |
• | patent applications may not result in any patents being issued; |
• | issued patents may be challenged, invalidated, modified, revoked, circumvented, found to be unenforceable, or may otherwise not provide any competitive advantage; |
• | our competitors, many of whom have substantially greater resources than we do 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 product candidate; |
• | other parties may have designed around our claims or developed technologies that may be related or competitive to ours, may have filed or may file patent applications and may have received or may receive patents that overlap or conflict with our patent applications and/or patents, either by claiming the same composition of matter, methods or formulations or by claiming subject matter that could dominate our patent position; |
• | any successful opposition to any patents owned by or licensed to us could deprive us of rights necessary for the practice of our technologies or the successful commercialization of any product candidate that we may develop; |
• | because patent applications in the United States and most other countries are confidential for a period of time after filing, we cannot be certain that we or our licensors were the first to file any patent application related to our product candidate and other proprietary technologies and their uses; |
• | an interference proceeding can be provoked by a third party or instituted by the USPTO to determine who was the first to invent any of the subject matter covered by the patent claims of any application with an effective filing date before March 16, 2013; |
• | 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 United States for disease treatments that prove successful, as a matter of public policy regarding worldwide health concerns; and |
• | countries other than the United States 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 candidate in those countries. |
• | others may be able to make compounds that are similar to our product candidate but that are not covered by the claims of our patents; |
• | we might not have been the first to file patent applications for these inventions; |
• | others may independently develop similar or alternative technologies or duplicate any of our technologies; |
• | any patents that we obtain may not provide us with any competitive advantages; |
• | we may not develop additional proprietary technologies that are patentable; |
• | our competitors might conduct research and development activities in countries where we do not have patent rights or where patent protection is weak and then use the information learned from such activities to develop competitive products for sale in our major commercial markets; |
• | we cannot ensure that we will be able to successfully commercialize our product candidate on a substantial scale, if approved, before the relevant patents that we own or license expire; or |
• | the patents of others may have an adverse effect on our business. |
• | the scope of rights granted under the license agreement and other interpretation-related issues; |
• | whether and the extent to which our technology and processes infringe on intellectual property rights of the licensor that are not subject to the license agreement; |
• | our right to sublicense intellectual property rights to third parties under collaborative development relationships; |
• | our diligence obligations with respect to the use of the licensed technology in relation to our development and commercialization of our product candidate, and what activities satisfy those diligence obligations; and |
• | the ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners. |
• | result in costly litigation; |
• | divert the time and attention of our technical personnel and management; |
• | cause development delays; |
• | prevent us from commercializing our product candidate until the asserted patent expires or is finally held invalid, unenforceable, or not infringed in a court of law; |
• | require us to develop non-infringing technology, which may not be possible on a cost-effective basis; |
• | require us to pay damages to the party whose intellectual property rights we may be found to be infringing, which may include treble damages if we are found to have been willfully infringing such intellectual property; |
• | require us to pay the attorney’s fees and costs of litigation to the party whose intellectual property rights we may be found to be willfully infringing; and/or |
• | require us to enter into royalty or license agreements, which may not be available on commercially reasonable terms, or at all. |
• | some patent applications in the United States may be maintained in secrecy until the patents are issued; |
• | patent applications in the United States and elsewhere can be pending for many years before issuance, or unintentionally abandoned patents or applications can be revived; |
• | pending patent applications that have been published can, subject to certain limitations, be later amended in a manner that could cover our technologies, our product candidate or their uses; |
• | identification of third-party patent rights that may be relevant to our technology is difficult because patent searching is imperfect due to differences in terminology among patents, incomplete databases, and the difficulty in assessing the meaning of patent claims; |
• | patent applications in the United States are typically not published until 18 months after the priority date; and |
• | publications in the scientific literature often lag behind actual discoveries. |
• | collaborators have significant discretion in determining the efforts and resources that they will apply to collaborations; |
• | collaborators may not pursue development and commercialization of our products or may elect not to continue or renew development or commercialization programs based on trial or test results, changes in their strategic focus due to the acquisition of competitive products, availability of funding, or other external factors, such as a business combination that diverts resources or creates competing priorities; |
• | collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our product candidate; |
• | a collaborator with marketing, manufacturing, and distribution rights to one or more products may not commit sufficient resources to or otherwise not perform satisfactorily in carrying out these activities; |
• | we could grant exclusive rights to our collaborators that would prevent us from collaborating with others; |
• | collaborators may not properly maintain or defend our intellectual property rights or may use our intellectual property or proprietary information in a way that gives rise to actual or threatened litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential liability; |
• | disputes may arise between us and a collaborator that causes the delay or termination of the research, development, or commercialization of our current or future products or that results in costly litigation or arbitration that diverts management attention and resources; |
• | collaborations may be terminated, and, if terminated, may result in a need for additional capital to pursue further development or commercialization of the applicable current or future products; |
• | collaborators may own or co-own intellectual property covering our products that results from our collaborating with them, and in such cases, we would not have the exclusive right to develop or commercialize such intellectual property; and |
• | a collaborator’s sales and marketing activities or other operations may not be in compliance with applicable laws resulting in civil or criminal proceedings. |
• | the federal Anti-Kickback Statute, which prohibits, among other things, individuals or entities from knowingly and willfully soliciting, receiving, offering or paying any remuneration (including any kickback, bribe or rebate), directly or indirectly, overtly or covertly, in cash or in kind in return for, or to induce, either the referral of an individual, or the purchase, lease, order or arrangement for or recommendation of the purchase, lease, order or arrangement for any good, facility, item or service for which payment may be made, in whole or in part, under a federal healthcare program, such as the Medicare and Medicaid programs. The term “remuneration” has been broadly interpreted to include anything of value. Although there are a number of statutory exceptions and regulatory safe harbors protecting some common activities from prosecution, the exceptions and safe harbors are drawn narrowly. Practices that involve remuneration that may be alleged to be intended to induce prescribing, purchases or recommendations may be subject to scrutiny if they do not qualify for an exception or safe harbor. A person does not need to have actual knowledge of this statute or specific intent to violate it in order to have committed a violation; |
• | the federal civil and criminal false claims laws, including, without limitation, the federal False Claims Act, which can be enforced by private citizens through civil whistleblower or qui tam actions, and civil monetary penalty laws which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment or approval from the federal government, including Medicare, Medicaid and other government payors, that are false or fraudulent or knowingly making, using or causing to be made or used a false record or statement material to a false or fraudulent claim or to avoid, decrease or conceal an obligation to pay money to the federal government. A claim includes “any request or demand” for money or property presented to the U.S. federal government. Several pharmaceutical and other healthcare companies have been prosecuted under these laws for allegedly providing free product to customers with the expectation that the customers would bill federal programs for the product. Other companies have been prosecuted for causing false claims to be submitted because of the companies’ marketing of products for unapproved, and thus non-reimbursable, uses. In addition, the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the federal False Claims Act; |
• | HIPAA which created additional federal criminal statutes which prohibit, among other things, a person from knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program, including private third-party payors and knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent statement in connection with the delivery of or payment for healthcare benefits, items or services. Similar to the federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation; |
• | HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act and their implementing regulations, also imposes obligations, including mandatory contractual terms, on “covered entities,” including certain healthcare providers, health plans, healthcare clearinghouses, and their respective “business associates,” that create, receive, maintain or transmit individually identifiable health information for or on behalf of a covered entity as well as their covered subcontractors, with respect to safeguarding the privacy, security and transmission of individually identifiable health information, as well as analogous state and foreign laws that govern the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts; |
• | the federal transparency laws, including the federal Physician Payments Sunshine Act, which requires certain manufacturers of drugs, medical devices, biologicals and medical supplies for which payment is available under Medicare, Medicaid or the State Children’s Health Insurance Program, with specific exceptions, to report annually to CMS, information related to: (i) payments or other “transfers of value’’ made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners) and teaching hospitals, and (ii) ownership and investment interests held by physicians and their immediate family members; and |
• | analogous state and foreign laws and regulations; state laws that require manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers, marketing expenditures or drug pricing; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government, or that otherwise restrict payments that may be made to healthcare providers; and state and local laws that require the registration of pharmaceutical sales representatives. |
• | issue an untitled letter or warning letter asserting that we are in violation of the law; |
• | seek an injunction or impose administrative, civil or criminal penalties or monetary fines; |
• | issue a safety alert, Dear Healthcare Provider letter, press release or other communication containing warnings or safety information about the product; |
• | mandate corrections to promotional materials and labeling or issuance of corrective information; |
• | suspend or withdraw regulatory approval; |
• | suspend any ongoing clinical trials; |
• | refuse to approve a pending marketing application or supplement to an approved application or comparable foreign marketing application (or any supplements thereto) submitted by us or our strategic partners; |
• | restrict the marketing or manufacturing of the drug; |
• | seize or detain the drug or otherwise require the withdrawal of the drug from the market; |
• | refuse to permit the import or export of products or product candidates; or |
• | refuse to allow us to enter into supply contracts, including government contracts. |
• | additional clinical trials to be conducted prior to obtaining approval; |
• | changes to manufacturing methods; |
• | recalls, replacements, or discontinuance of one or more of our products; and |
• | additional recordkeeping. |
• | successfully attract and recruit new employees or consultants with the expertise and experience we will require; |
• | manage our clinical programs effectively, which we anticipate being conducted at numerous clinical sites; |
• | develop a marketing and sales infrastructure; and |
• | continue to improve our operational, financial and management controls, reporting systems and procedures. |
• | the commencement, enrollment or results of our clinical trials, including INTERCEPT-AD and any future clinical trials we may conduct, or changes in the development status of our product candidates; |
• | any delay in our regulatory filings for ACU193 or any other product candidate we may develop, and any adverse development or perceived adverse development with respect to the applicable regulatory authority’s review of such filings, including without limitation the FDA’s issuance of a “refusal to file” letter or a request for additional information; |
• | delays in, or termination of, clinical trials; |
• | adverse regulatory decisions, including failure to receive regulatory approval of our product candidates; |
• | unanticipated serious safety concerns related to the use of ACU193 or any other product candidate we develop; |
• | changes in financial estimates by us or by any equity research analysts who might cover our stock; |
• | conditions or trends in our industry; |
• | changes in the market valuations of similar companies; |
• | announcements by our competitors of new product candidates or technologies, or the results of clinical trials or regulatory decisions; |
• | stock market price and volume fluctuations of comparable companies and, in particular, those that operate in the biopharmaceutical industry; |
• | publication of research reports about us or our industry or positive or negative recommendations or withdrawal of research coverage by securities analysts; |
• | announcements by us or our competitors of significant acquisitions, strategic partnerships or divestitures; |
• | our relationships with our collaborators; |
• | announcements of investigations or regulatory scrutiny of our operations or lawsuits filed against us; |
• | investors’ general perception of our company and our business; |
• | recruitment or departure of key personnel; |
• | overall performance of the equity markets; |
• | trading volume of our common stock; |
• | disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain patent protection for our technologies; |
• | significant lawsuits, including patent or stockholder litigation; |
• | changes in the structure of healthcare payment systems; |
• | general political and economic conditions; and |
• | other events or factors, many of which are beyond our control. |
• | only one of our three classes of directors will be elected each year; |
• | stockholders will not be entitled to remove directors other than by a 66 2/3% vote and only for cause; |
• | stockholders will not be permitted to take actions by written consent; |
• | stockholders cannot call a special meeting of stockholders; and |
• | stockholders must give advance notice to nominate directors or submit proposals for consideration at stockholder meetings. |
• | not being required to comply with the auditor attestation requirements in the assessment of our internal control over financial reporting; |
• | not being required to comply with any requirement that may be adopted by the Public Company Accounting Oversight Board regarding mandatory audit firm rotation or a supplement to the auditor’s report providing additional information about the audit and the financial statements; |
• | reduced disclosure obligations regarding executive compensation in our periodic reports, proxy statements and registration statements; and |
• | not being required to hold a nonbinding advisory vote on executive compensation and stockholder approval of any golden parachute payments not previously approved. |
• | any derivative claim or cause of action brought on our behalf; |
• | any claim or cause of action asserting a breach of fiduciary duty; |
• | any claim or cause of action against us arising under DGCL; |
• | any claim or cause of action arising under or seeking to interpret our amended and restated certificate of incorporation or our amended and restated bylaws; and |
• | any claim or cause of action against us that is governed by the internal affairs doctrine. |
• | we will indemnify our directors and officers for serving us in those capacities or for serving other business enterprises at our request, to the fullest extent permitted by Delaware law. Delaware law provides that a corporation may indemnify such person if such person acted in good faith and in a manner such person reasonably believed to be in or not opposed to the best interests of the registrant and, with respect to any criminal proceeding, had no reasonable cause to believe such person’s conduct was unlawful; |
• | we may, in our discretion, indemnify employees and agents in those circumstances where indemnification is permitted by applicable law; |
• | we are required to advance expenses, as incurred, to our directors and officers in connection with defending a proceeding, except that such directors or officers shall undertake to repay such advances if it is ultimately determined that such person is not entitled to indemnification; |
• | we are not obligated pursuant to our amended and restated bylaws to indemnify a person with respect to proceedings initiated by that person against us or our other indemnitees, except with respect to proceedings authorized by our board of directors or brought to enforce a right to indemnification; |
• | the rights conferred in our amended and restated bylaws are not exclusive, and we are authorized to enter into indemnification agreements with our directors, officers, employees and agents and to obtain insurance to indemnify such persons; and |
• | we may not retroactively amend our amended and restated bylaw provisions to reduce our indemnification obligations to directors, officers, employees and agents. |
• | costs of funding research performed by third parties that conduct research and development and nonclinical and clinical activities on our behalf; |
• | costs of manufacturing drug supply and drug product; |
• | costs of conducting nonclinical studies and clinical trials of our product candidates; |
• | consulting and professional fees related to research and development activities, including equity-based compensation to non-employees; |
• | costs related to compliance with clinical regulatory requirements; and |
• | employee-related expenses, including salaries, benefits and stock-based compensation expense for our research and development personnel. |
Year Ended December 31, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
Grant and other revenue |
$ | — | $ | 1,436 | $ | (1,436 | ) | |||||
Costs and operating expenses |
||||||||||||
Research and development |
12,305 | 7,997 | 4,308 | |||||||||
General and administrative |
7,279 | 1,351 | 5,928 | |||||||||
|
|
|
|
|
|
|||||||
Total operating expenses |
19,584 | 9,348 | 10,236 | |||||||||
|
|
|
|
|
|
|||||||
Loss from operations |
(19,584 | ) | (7,912 | ) | (11,672 | ) | ||||||
Other income (expense) |
||||||||||||
Change in fair value of preferred stock tranche rights liability and preferred stock warrant liability |
(81,157 | ) | 586 | (81,743 | ) | |||||||
Interest income, net |
84 | 1 | 83 | |||||||||
Other income, net |
51 | — | 51 | |||||||||
|
|
|
|
|
|
|||||||
Total other income (expense) |
(81,022 | ) | 587 | (81,609 | ) | |||||||
|
|
|
|
|
|
|||||||
Net loss |
(100,606 | ) | (7,325 | ) | (93,281 | ) | ||||||
|
|
|
|
|
|
|||||||
Other comprehensive loss |
||||||||||||
Unrealized loss on marketable securities |
(231 | ) | — | (231 | ) | |||||||
|
|
|
|
|
|
|||||||
Comprehensive loss |
$ | (100,837 | ) | $ | (7,325 | ) | $ | (93,512 | ) | |||
|
|
|
|
|
|
Year Ended December 31, |
||||||||
2021 |
2020 |
|||||||
Net cash used in operating activities |
$ | (17,961 | ) | $ | (7,450 | ) | ||
Net cash used in investing activities |
(104,120 | ) | — | |||||
Net cash provided by financing activities |
200,466 | 44,675 | ||||||
|
|
|
|
|||||
Net change in cash and cash equivalents |
$ | 78,385 | $ | 37,225 | ||||
|
|
|
|
• | the scope, progress, results and costs of discovery, nonclinical development, laboratory testing and clinical trials for other potential product candidates we may develop, if any; |
• | the costs, timing and outcome of regulatory review of our product candidates; |
• | our ability to establish and maintain collaborations on favorable terms, if at all; |
• | the achievement of milestones or occurrence of other developments that trigger payments under any collaboration agreements we might have at such time; |
• | the costs and timing of future commercialization activities, including product sales, marketing, manufacturing and distribution, for any of our product candidates for which we receive marketing approval; |
• | the amount of revenue, if any, received from commercial sales of our product candidates, should any of our product candidates receive marketing approval; |
• | the costs of preparing, filing and prosecuting patent applications, obtaining, maintaining and enforcing our intellectual property rights and defending intellectual property-related claims; |
• | our headcount growth and associated costs as we expand our business operations and our research and development activities; and |
• | the costs of operating as a public company. |
• | Fair Value of Common Stock Common Stock Valuations |
• | Expected Term mid-point between the vesting date and the end of contractual term of the option (generally ten years). |
• | Expected Volatility |
• | Risk-Free Interest Rate |
• | Expected Dividend Yield |
• | relevant precedent transactions involving our capital stock; |
• | contemporaneous valuations of our common stock performed by third-party specialists; |
• | rights, preferences, and privileges of our redeemable convertible preferred stock relative to those of our common stock; |
• | our business, financial condition and results of operations, including related industry trends affecting our operations; |
• | likelihood of achieving a liquidity event, such as an initial public offering or a sale of our business; |
• | the lack of marketability of our common stock, and the illiquidity of stock-based awards involving securities in a private company; |
• | market multiples of comparable publicly-traded companies; and |
• | U.S. and global capital and macroeconomic conditions. |
• | Option Pricing Method, or OPM. |
• | Probability-Weighted Expected Return Method, or PWERM. |
• | an exception from compliance with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act of 2002, as amended; |
• | reduced disclosure about our executive compensation arrangements in our periodic reports, proxy statements and registration statements; |
• | exemptions from the requirements of holding non-binding advisory votes on executive compensation or golden parachute arrangements; and |
• | an exemption from compliance with the requirements of the Public Company Accounting Oversight Board regarding the communication of critical audit matters in the auditor’s report on financial statements. |
Page |
||||
Report of Independent Registered Public Accounting Firm (PCAOB ID: |
115 |
|||
116 |
||||
117 |
||||
118 |
||||
119 |
||||
120 |
December 31, |
||||||||
2021 |
2020 |
|||||||
ASSETS |
||||||||
Current assets |
||||||||
Cash and cash equivalents |
$ | $ | ||||||
Marketable securities, short-term |
— | |||||||
Grant receivable |
— | |||||||
Prepaid expenses and other current assets |
||||||||
Total current assets |
||||||||
Marketable securities, long-term |
— | |||||||
Property and equipment, net |
— | |||||||
Other assets |
— | |||||||
Total assets |
$ | $ | ||||||
LIABILITIES, CONVERTIBLE PREFERRED STOCK AND STOCKHOLDERS’ EQUITY (DEFICIT) |
||||||||
Current liabilities |
||||||||
Accounts payable |
$ | $ | ||||||
Accrued expenses and other current liabilities |
||||||||
Preferred stock tranche rights liability |
— | |||||||
Preferred stock warrant liability |
— | |||||||
Total liabilities |
||||||||
Series A convertible preferred stock, $ December 31, 2021; 2020; liquidation preference of $ |
— | |||||||
Series A-1 convertible preferred stock, $ |
— | |||||||
Series B convertible preferred stock, $ as of December 31, 2021; December 31, 2020; liquidation preference of $ |
— | |||||||
Stockholders’ equity (deficit) |
||||||||
Preferred stock, $ December 31, 2021; |
— | |||||||
Common stock, $ |
— | |||||||
Additional paid-in capital |
||||||||
Accumulated deficit |
( |
) | ( |
) | ||||
Accumulated other comprehensive loss |
( |
) | — | |||||
Total stockholders’ equity (deficit) |
( |
) | ||||||
Total liabilities, convertible preferred stock and stockholders’ equity (deficit) |
$ | $ | ||||||
Year Ended December 31, |
||||||||
2021 |
2020 |
|||||||
Grant and other revenue |
$ | — | $ | |||||
Operating expenses |
||||||||
Research and development |
||||||||
General and administrative |
||||||||
|
|
|
|
|||||
Total operating expenses |
||||||||
|
|
|
|
|||||
Loss from operations |
( |
) | ( |
) | ||||
Other income (expense) |
||||||||
Change in fair value of preferred stock tranche rights liability and preferred stock warrant liability |
( |
) | ||||||
Interest income, net |
||||||||
Other income, net |
— | |||||||
|
|
|
|
|||||
Total other income (expense) |
( |
) | ||||||
|
|
|
|
|||||
Net loss |
( |
) | ( |
) | ||||
|
|
|
|
|||||
Other comprehensive loss |
||||||||
Unrealized loss on marketable securities |
( |
) | — | |||||
|
|
|
|
|||||
Comprehensive loss |
$ | ( |
) | $ | ( |
) | ||
|
|
|
|
|||||
Net loss per common share, basic and diluted |
$ | ( |
) | $ | ( |
) | ||
|
|
|
|
|||||
Weighted-average shares outstanding, basic and diluted |
||||||||
|
|
|
|
Series A Convertible Preferred Stock |
Series A-1 Convertible Preferred Stock |
Series B Convertible Preferred Stock |
Common Stock |
Additional Paid-in Capital |
Accumulated Deficit |
Accumulated Other Comprehensive Loss |
Total Stockholders’ Equity (Deficit) |
|||||||||||||||||||||||||||||||||||||||||
Shares |
Amount |
Shares |
Amount |
Shares |
Amount |
Shares |
Amount |
|||||||||||||||||||||||||||||||||||||||||
Balance as of December 31, 2019 |
$ |
$ |
$ |
$ |
$ |
$ |
( |
) |
$ |
$ |
( |
) | ||||||||||||||||||||||||||||||||||||
Issuance of Series B convertible preferred stock for cash, net of issuance costs of $ |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
||||||||||||||||||||||||||||||||||||||
Share-based compensation |
— |
— |
— |
— |
— |
— |
— |
— |
— |
|||||||||||||||||||||||||||||||||||||||
Net loss |
— |
— |
— |
— |
— |
— |
— |
— |
— |
( |
) |
( |
) | |||||||||||||||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||||||||||
Balance as of December 31, 2020 |
( |
) |
( |
) | ||||||||||||||||||||||||||||||||||||||||||||
Issuance of milestone shares for cash, net of issuance costs of $ |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
||||||||||||||||||||||||||||||||||||||
Exercise of preferred stock warrant |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
||||||||||||||||||||||||||||||||||||||
Reclassification of preferred stock tranche rights liability upon issuance of milestone shares |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
|||||||||||||||||||||||||||||||||||||
Reclassification of warrant liability upon exercise of preferred stock warrant |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
|||||||||||||||||||||||||||||||||||||
Exercise of common stock warrants |
— |
— |
— |
— |
— |
— |
— |
— |
— |
|||||||||||||||||||||||||||||||||||||||
Conversion of convertible preferred stock into common stock upon initial public offering |
( |
) |
( |
) |
( |
) |
( |
) |
( |
) |
( |
) |
— |
— |
||||||||||||||||||||||||||||||||||
Issuance of common stock for cash, net of issuance costs of $ |
— |
— |
— |
— |
— |
— |
— |
— |
||||||||||||||||||||||||||||||||||||||||
Cashless exercise of common stock warrants |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
|||||||||||||||||||||||||||||||||||||
Stock options exercised |
— |
— |
— |
— |
— |
— |
— |
— |
— |
|||||||||||||||||||||||||||||||||||||||
Unrealized loss on marketable securities |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
( |
) |
( |
) | ||||||||||||||||||||||||||||||||||
Share-based compensation |
— |
— |
— |
— |
— |
— |
— |
— |
— |
— |
||||||||||||||||||||||||||||||||||||||
Net loss |
— |
— |
— |
— |
— |
— |
— |
— |
— |
( |
) |
— |
( |
) | ||||||||||||||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||||||||||
Balance as of December 31, 2021 |
$ |
$ |
$ |
$ |
$ |
$ |
( |
) |
$ |
( |
) |
$ |
||||||||||||||||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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: |
||||||||
Depreciation |
— | |||||||
Change in fair value of preferred stock tranche rights liability and preferred stock warrant liability |
( |
) | ||||||
Stock-based compensation expense |
||||||||
Amortization of premiums and accretion of discounts on marketable securities, net |
— | |||||||
Other non-cash expense |
— | |||||||
Changes in operating assets and liabilities: |
||||||||
Grant receivable |
— | ( |
) | |||||
Prepaid expenses and other current assets |
( |
) | ||||||
Other assets |
( |
) | ||||||
Accounts payable |
||||||||
Accrued expenses and other current liabilities |
( |
) | ||||||
|
|
|
|
|||||
Net cash used in operating activities |
( |
) | ( |
) | ||||
|
|
|
|
|||||
Cash flows from investing activities |
||||||||
Purchases of available-for-sale |
( |
) | — | |||||
Purchases of property and equipment |
( |
) | — | |||||
|
|
|
|
|||||
Net cash used in investing activities |
( |
) | — | |||||
|
|
|
|
|||||
Cash flows from financing activities |
||||||||
Proceeds from issuance of Series B milestone shares, net of issuance costs |
— | |||||||
Proceeds from issuance of convertible preferred stock, net of issuance costs |
— | |||||||
Proceeds from exercise of Series A-1 warrant |
— | |||||||
Proceeds from exercise of common stock warrants |
— | |||||||
Proceeds from issuance of common stock upon initial public offering, net of issuance costs |
— | |||||||
Proceeds from stock option exercises |
— | |||||||
|
|
|
|
|||||
Net cash provided by financing activities |
||||||||
|
|
|
|
|||||
Net change in cash and cash equivalents |
||||||||
Cash and cash equivalents at the beginning of the period |
||||||||
|
|
|
|
|||||
Cash and cash equivalents at the end of the period |
$ | $ | ||||||
|
|
|
|
|||||
Supplemental disclosure of cash flow information |
||||||||
Cash paid for income taxes |
$ | $ | ||||||
|
|
|
|
|||||
Cash paid for interest |
$ | $ | ||||||
|
|
|
|
|||||
Supplemental disclosure of noncash financing activities |
||||||||
Reclassification of preferred stock tranche rights liability upon share issuance |
$ | $ | ||||||
|
|
|
|
|||||
Reclassification of warrant liability upon exercise of preferred stock warrant |
$ | $ | ||||||
|
|
|
|
|||||
Conversion of convertible preferred stock into common stock upon IPO |
$ | $ | ||||||
|
|
|
|
Level 1 — |
Observable inputs, such as quoted prices in active markets for identical assets or liabilities. |
Level 2 — |
Inputs other than Level 1 inputs that are either directly or indirectly observable, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the instrument’s anticipated life. |
Level 3 — |
Unobservable inputs in which there is little or no market data, which require the reporting entity to develop its own assumptions. |
Fair value measurements at reporting date using |
||||||||||||||||
Quoted Prices in Active Markets for Identical Assets (Level 1) |
Significant Other Observable Inputs (Level 2) |
Significant Unobservable Inputs (Level 3) |
Fair Value at December 31, 2021 |
|||||||||||||
Assets included in: |
||||||||||||||||
Cash and cash equivalents |
||||||||||||||||
Money market securities |
$ | $ | — | $ | $ | |||||||||||
Marketable securities |
||||||||||||||||
Commercial paper |
— | |||||||||||||||
Corporate debt securities |
— | |||||||||||||||
Asset-backed securities |
— | |||||||||||||||
U.S. treasury securities |
— | |||||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total fair value |
$ | $ | $ | $ | ||||||||||||
|
|
|
|
|
|
|
|
Fair value measurements at reporting date using |
||||||||||||||||
Quoted Prices in Active Markets for Identical Assets (Level 1) |
Significant Other Observable Inputs (Level 2) |
Significant Unobservable Inputs (Level 3) |
Fair Value at December 31, 2020 |
|||||||||||||
Assets included in: |
||||||||||||||||
Cash and cash equivalents |
||||||||||||||||
Money market securities |
$ | $ | — | $ | — | $ | ||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total fair value |
$ | $ | — | $ | — | $ | ||||||||||
|
|
|
|
|
|
|
|
|||||||||
Liabilities included in: |
||||||||||||||||
Preferred stock tranche rights liability |
$ | — | $ | — | $ | $ | ||||||||||
Preferred stock warrant liability |
— | — | ||||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total fair value |
$ | — | $ | — | $ | $ | ||||||||||
|
|
|
|
|
|
|
|
Amortized Cost |
Gross Unrealized Gains |
Gross Unrealized Losses |
Fair Value |
|||||||||||||
Available-for-sale |
||||||||||||||||
Commercial paper |
$ | $ | — | $ | — | $ | ||||||||||
Corporate debt securities |
— | ( |
) | |||||||||||||
Asset-backed securities |
— | ( |
) | |||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total available-for-sale |
— | ( |
) | |||||||||||||
Available-for-sale |
||||||||||||||||
Corporate debt securities |
— | ( |
) | |||||||||||||
Asset-backed securities |
— | ( |
) | |||||||||||||
U.S. treasury securities |
— | ( |
) | |||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total available-for-sale |
— | ( |
) | |||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total available-for-sale |
$ | $ | — | $ | ( |
) | $ | |||||||||
|
|
|
|
|
|
|
|
December 31, |
||||||||
2021 |
2020 |
|||||||
Research and development service agreements |
$ | $ | ||||||
Prepaid insurance |
||||||||
Dues and subscriptions |
— | |||||||
Prepaid raw materials |
||||||||
Other |
||||||||
|
|
|
|
|||||
Total prepaid expenses and other current assets |
$ | $ | ||||||
|
|
|
|
December 31, |
||||||||
2021 |
2020 |
|||||||
Research and development |
$ | $ | ||||||
Bonuses and other employee liabilities |
— | |||||||
Legal |
— | |||||||
Professional fees |
||||||||
Other |
||||||||
|
|
|
|
|||||
Total accrued expenses and other current liabilities |
$ | $ | ||||||
|
|
|
|
Shares Authorized |
Shares Issued and Outstanding |
Weighted Average Issuance Price per Share |
Carrying Value |
Liquidation Preference |
||||||||||||||||
Series A |
$ | $ | $ | |||||||||||||||||
Series A-1 |
||||||||||||||||||||
Series B |
||||||||||||||||||||
|
|
|
|
|
|
|
|
|||||||||||||
Total |
$ | $ | ||||||||||||||||||
|
|
|
|
|
|
|
|
June 17, 2021 |
December 31, 2020 |
|||||||
Risk-free interest rate |
% | % | ||||||
Expected time to Milestone Closing (in years) |
||||||||
Probability of achievement of Milestone Closing |
% | % |
June 2021 |
December 31, 2020 | |||
Risk-free interest rate |
||||
Expected term (in years) |
||||
Expected volatility |
||||
Expected dividend yield |
Series A-1 Preferred Stock Warrant |
Series B Tranche Rights |
Total |
||||||||||
Balance, December 31, 201 9 |
|
$ |
|
|
|
$ |
— |
|
|
$ |
|
|
Fair value at issuance of Series B convertible preferred stock (November 2020) |
|
|
— |
|
|
|
|
|
|
|
|
|
Change in fair valu e |
|
|
( |
) |
|
|
( |
) |
|
|
( |
) |
Balance, December 31, 2020 |
||||||||||||
Change in fair value |
||||||||||||
Settlement of tranche liability due to issuance of Milestone Shares |
— | ( |
) | ( |
) | |||||||
Settlement of warrant liability upon exercise of |
( |
) | — | ( |
) | |||||||
|
|
|
|
|
|
|||||||
Balance, December 31, 2021 |
$ | $ | $ |
Equity Upon Exercise |
Exercise Price |
Expiration Dates |
Number of Warrants |
|||||||||||||
Warrants issued in 2014 |
Common Stock | $ | ||||||||||||||
Warrants issued in 2015 |
Common Stock | $ | ||||||||||||||
Warrants issued in 2016 |
Common Stock | $ | ||||||||||||||
Warrants issued in 2017 |
Common Stock | $ | ||||||||||||||
|
|
|||||||||||||||
Total Warrants |
Risk-free interest rate |
||
Expected term (in years) |
||
Expected volatility |
||
Expected dividend yield |
Risk-free interest rate |
||
Expected time to liquidity event (in years) |
||
Expected volatility |
||
Expected dividend yield |
Stock Options |
Weighted Average Exercise Price |
Weighted Average Remaining Contractual Life (in years) |
Aggregate Intrinsic Value (in thousands) |
|||||||||||||
Outstanding at December 31, 2020 |
$ | |||||||||||||||
Granted |
||||||||||||||||
Exercised |
( |
) | ||||||||||||||
Outstanding at December 31, 2021 |
$ | $ | ||||||||||||||
Vested and exercisable at December 31, 2021 |
$ | $ |
Year Ended December 31, |
||||||||
2021 |
2020 |
|||||||
General and administrative |
$ | $ | ||||||
Research and development |
||||||||
Total stock-based compensation |
$ | $ |
For the Year Ended December 31, |
||||||||
2021 |
2020 |
|||||||
Statutory federal income tax rate |
% | % | ||||||
State tax, net of federal benefit |
||||||||
Change in fair value of tranche and warrant liabilities |
( |
) | ||||||
Non-deductible expense |
( |
) | — | |||||
R&D credit |
( |
) | ||||||
Rate change |
( |
) | — | |||||
Other |
— | |||||||
Change in valuation allowance |
( |
) | ( |
) | ||||
Income tax provision (benefit) |
% | % | ||||||
December 31, |
||||||||
2021 |
2020 |
|||||||
Deferred tax assets: |
||||||||
Net operating loss |
$ | $ | ||||||
R&D credit |
||||||||
Stock compensation |
||||||||
Accruals and other temporary differences |
— | |||||||
Gross deferred tax assets |
||||||||
Depreciation |
( |
) | — | |||||
Valuation allowance |
( |
) | ( |
) | ||||
Deferred tax assets, net of allowance |
$ | $ | ||||||
Year ended December 31, 2022 |
$ |
|||
Year ended December 31, 2023 |
||||
Total |
$ | |||
Year Ended December 31, |
||||||||
2021 |
2020 |
|||||||
Shares issuable upon exercise of stock options |
||||||||
Shares issuable upon conversion of Series A Preferred Stock |
||||||||
Shares issuable upon conversion of Series A-1 Preferred Stock |
||||||||
Shares issuable upon conversion of Series B Preferred Stock |
||||||||
Shares issuable upon exercise of common stock warrants |
||||||||
Shares issuable upon exercise of preferred stock warrant |
||||||||
Total |
||||||||
* | Filed herewith. |
† | Pursuant to Item 601(b)(10) of Regulation S-K, certain confidential portions of this exhibit have been omitted by means of marking such portions with asterisks [***] as the identified confidential portions (i) are not material and (ii) the Registrant customarily and actually treats that information as private or confidential. |
+ | Indicates management contract or compensatory plan. |
# | These certifications are being furnished solely to accompany this annual report pursuant to 18 U.S.C. Section 1350, and are not being filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, and are not to be incorporated by reference into any filing of the registrant, whether made before or after the date hereof, regardless of any general incorporation language in such filing. |
ACUMEN PHARMACEUTICALS, INC. | ||||
Date: March 28, 2022 | By: | /s/ Daniel O’Connell | ||
Daniel O’Connell President and Chief Executive Officer |
Signature |
Title |
Date | ||
/s/ Daniel O’Connell |
Chief Executive Officer and Director (Principal Executive Officer) |
March 28, 2022 | ||
Daniel O’Connell | ||||
/s/ William Matthew Zuga |
Chief Financial Officer and Chief Business Officer |
March 28, 2022 | ||
William Matthew Zuga | (Principal Financial Officer and Principal Accounting Officer) |
|||
/s/ Nathan B. Fountain |
Director | March 28, 2022 | ||
Nathan B. Fountain, M.D. | ||||
/s/ Jeffrey L. Ives |
Director | March 28, 2022 | ||
Jeffrey L. Ives, PhD | ||||
/s/ Sean Stalfort |
Director | March 28, 2022 | ||
Sean Stalfort | ||||
/s/ Laura Stoppel |
Director | March 28, 2022 | ||
Laura Stoppel, PhD | ||||
/s/ Jeffrey Sevigny |
Director | March 28, 2022 | ||
Jeffrey Sevigny, M.D. |