Content analysis
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Litigous | ||
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H.S. senior Avg
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New words:
AAV, absorption, Acadia, accident, Accipiter, accreted, accretion, accrual, acquiree, Activist, adapt, adaptation, addendum, adequacy, adulterated, advocacy, advocating, affiliated, agnostic, aid, aim, AKS, Alcyone, alliance, alter, alteration, altered, altering, ameliorated, amelioration, aminotransferase, analogue, analogy, analyst, analyze, analyzed, analyzing, anatomic, annex, anniversary, antibody, antidilutive, arbitrage, Article, artificial, ascertain, ASP, aspiration, AstraZeneca, attached, Attenuation, attorned, Attornment, Auspex, autonomic, Axovant, background, backup, baculo, Baffi, baseline, batch, Batten, bearing, Becerra, bioanalytical, biology, biosafety, Bipartisan, birth, black, bold, Borderdale, brain, breakdown, breakthrough, breathing, Brexit, bribery, Bridge, bridging, Britain, brochure, built, Bulletin, burdensome, capsid, cardinal, caregiver, Carolina, cast, CAT, Catalyst, categorized, catheter, Cautionary, CBER, CDMO, Centre, Century, Cerevel, Certified, certifying, cGCP, channel, Chapel, check, childhood, CHMP, chosen, Christine, chronic, chronically, clarification, classic, clause, Clawback, cleanroom, clinician, clock, closure, CMC, CNS, Cobb, Cochener, CODM, cohort, cold, commensurate, commentary, commerce, comparison, compatibility, compatible, compensatory, compound, comprised, comprising, concomitant, constitute, construct, construe, contest, contraindication, convention, conventional, convey, convince, cord, Corhort, correctly, correlation, cortex, Cory, counsel, coupled, creative, CTR, currency, curve, Cvijic, CVR, dampened, database, dataset, DAYBUE, deadline, dealing, Dear, death, debilitating, decentralized, defective, delegated, Deloitte, depicted, deplete, depressing, depression, depriving, destroyed, destruction, devastating, developmental, diagnosing, dialogue, die, died, disaggregated, disaggregation, disciplined, disclaim, disorder, display, diverge, divergent, domain, Donna, dorsal, dossier, downstream, Dr, Drachman, draft, DRG, driven, dual, dysfunction, earliest, easily, EC, Edinburgh, EIP, electrophysiological, element, Eli, email, embedded, endorsed, endorsement, enterprise, enzyme, Equiniti, Ernst, eroding, escalating, escalation, evidentiary, exact, EXACTTM, exceeded, excessive, excise, executory, expedite, expedited, Explanation, explicitly, exploit, expressly, eye, fatal, FCA, FDCA, FDIC, FDORA, feature, female, figure, fine, flammable, Floor, fluctuate, fluid, foot, forefront, format, formation, Founder, Freedland, freedom, fulfil, fulfill, fulfilled, gait, GALC, ganglion, GCP, gene, genome, genotypically, GMP, god, gold, GPB, Great, greatest, green, GTM, handling, harnessing, hear, hearing, Hedging, herewith, highest, highlighted, Hill, hindsight, hostile, Houston, IBC, IBR, Iceland, ICV, IDTA, ImClone, immunosuppression, impermissible, Importantly, incentivize, incentivizing, incomplete, incorrect, incorrectly, indemnification, indemnify, indemnity, index, indispensable, injunction, injunctive, Inline, input, inquiring, inquiry, insect, intelligence, Intercept, interdisciplinary, intermediary, intermediate, interplay, interstate, intervention, intracerebral, intracranial, intraocular, intrathecal, intravitreal, introduce, introduced, intrusion, inventorship, invoiced, involuntary, Ireland, IRP, Israel, issuable, IVT, Johnson, Jonathan, justification, knockout, Krabbe, laid, landlord, Lastly, laundering, lawsuit, left, legacy, legitimate, lengthen, lengthier, Liechtenstein, lifecycle, lifespan, Lilly, Lincoln, linear, listen, live, LLC, LLP, logo, Lonza, lowest, LP, lumbar, MA, majeure, Maker, male, malfunction, mammalian, margin, marked, MAs, Master, mathematically, MCA, McMinn, meaningfully, median, mediated, medulla, MHRA, mice, microscopic, middle, midpoint, midyear, Mikail, military, mind, miRNA, mirror, misbranded, mislabeled, missing, mission, misuse, MMDA, mobility, modality, moderately, moiety, monogenic, month, mosaic, motivate, motor, mouse, MRHA, mRNA, multidisciplinary, Multiplied, mutation, mutual, navigate, NCE, NCV, nerve, network, neurodegeneration, neurodegenerative, neurodevelopmental, neurogene, neurogenerative, neurological, neuroscience, Nevada, NGNE, NHP, NIH, NOAEL, noncash, nonrefundable, Noonberg, Northern, Norway, novelty, NPS, nucleic, NY, ocular, older, omission, onsite, OpCo, opt, overcome, overcoming, overexpression, overhaul, overlooked, ovine, owed, packed, Page, paragraph, parallel, Parliament, passionate, path, pathobiology, pathogenic, pathology, pathophysiology, Patrick, PATRIOT, PDMA, Pennsylvania, peripheral, pertinent, pervasive, PFO, Pharma, pharmacy, Pharmasset, phenotype, phenotypic, phenotypical, phenotypically, PHI, pill, pilot, PIP, pivotal, placement, plaintiff, poison, portal, post, postnatal, postponing, preeminent, prejudiced, premature, pretax, PRIME, principle, pro, proactive, proactively, professor, proportion, provoke, PRV, PSP, punishable, pure, purple, pushing, qualification, Rachel, radioactive, randomly, rapporteur, rata, ratified, rational, rationale, reaction, reagent, reassessed, recapture, recipient, recordkeeping, red, redeemable, redefine, redemption, reevaluation, refund, regenerative, regime, regular, Reimagine, reimagining, rejected, rejection, renowned, rentable, repetition, replicate, repressor, repurchase, Rescue, reside, residing, residual, restarted, restitution, resubmit, resubmitted, retinal, retrospectively, Rett, revoked, rigorously, rituximab, RMAT, RMP, Robert, root, ROU, RSA, RTT, running, SAB, sample, sampling, Sarah, scalable, score, scoring, Sean, sector, seed, segregation, seminal, sense, sequalae, serotype, settlement, sheep, shortage, shut, SI, Sigma, silenced, Silicon, simultaneously, sixty, skepticism, slight, slowdown, Smith, SmPC, solved, solving, somatic, sought, spam, span, SPC, specialty, Specimen, speech, spinal, St, stagger, staggered, staggering, standalone, Stella, step, stipulated, Stoke, straightforward, streamline, streamlined, Stuart, sublessor, subscription, substance, subtenant, subtitle, succumbed, sue, sunset, superseded, supplementary, sural, suspected, symptomatic, synthetic, systematic, Takeda, taxation, Taysha, TCJA, telephone, territory, terrorist, theft, thereof, thereon, thereunto, thermostat, tied, tight, today, Touche, tradable, transcriptional, transduction, transfection, transgene, transient, translated, Translational, translationally, treatable, Treaty, trofinetide, true, Tuning, unaffiliated, unallocated, unannounced, unavailability, unavailable, UNC, uncapped, unchanged, uncollectible, uncovered, uncured, undermine, underscoring, undersigned, understaffing, underway, unfairly, unfeasible, unified, unilateral, unintentional, unlicensed, unmodified, unpaid, unregulated, unrest, unrestricted, unsatisfactory, untreated, unvested, UPC, upstream, uptake, urgency, USA, usage, Valley, variation, vector, vehicle, velocity, ventricular, verbally, version, veterinary, vg, vialed, vibrant, Virovek, vision, voluntarily, voucher, warehouse, weakened, weigh, whichever, wild, Windsor, wrongfully, XBRL, York, Young
Removed:
abandoned, abusive, activate, activation, adult, affirmative, agonist, allege, alpha, amino, announcement, annuity, antagonist, antitumor, Apollo, approximation, arranging, ARTG, aspect, assemble, assembled, assume, attacked, attained, attempting, attend, attorney, Australian, authored, autoimmunity, automatic, avoided, awarded, back, bar, began, beta, billion, biochemical, block, blocking, boxed, breadth, bribe, broaden, bundled, Canadian, cancelable, capped, carcinogenicity, carcinoma, careful, centered, certainty, checkpoint, chemotherapy, circumvented, claiming, codified, coincide, collect, collusion, color, comment, commented, commit, communicable, computational, conceal, conceived, conclusion, conclusive, conditionally, Conference, confirmation, consistency, consume, contracting, contractually, convenient, Conversely, Conviction, cooperate, copying, correct, corrected, corruption, covertly, crystal, CTN, CTX, cultivating, custody, cytokine, danger, decoy, dedicate, default, define, deny, Depository, determinable, deterring, devise, differential, digit, diligent, diligently, dilution, disability, disaster, discounting, discovering, discrimination, disproportionate, disqualification, disrupted, dissuade, DOJ, doubled, dramatic, durable, dynamic, earthquake, ease, East, economically, effecting, emphasize, encompassing, encouraged, engineered, entirety, entrance, enumerated, erected, ethically, ethnicity, evade, exchangeable, excluded, extrapolate, facially, factual, fashion, faulty, FCPA, FDC, feel, fierce, fill, filled, fire, fix, fold, folding, forecast, formulary, fullest, gained, gamma, gender, generating, geographical, goodwill, ground, guided, harassment, harmful, harming, heavily, hinder, holding, honest, hormone, hospital, house, HREC, ICH, illegally, illustrative, immunologic, immunological, immunoregulatory, immunotherapy, imply, impossible, impressive, inapplicable, inconsistency, incurrence, indefinite, indeterminate, India, Indictment, infection, inflamed, inflammation, influencing, inhibiting, inhibition, inhibitory, injury, insolvency, insolvent, Inspector, installation, intangible, integration, intense, interact, interference, interleukin, intermolecular, Internet, investigate, invite, ipilimumab, jointly, Jumpstart, Justice, kickback, kind, language, launching, leader, learned, leasing, lieu, ligand, liquid, location, main, malignant, mandate, manmade, meant, melanoma, metastatic, methodology, microbial, microenvironment, mimetic, mimic, misappropriated, misappropriating, misrepresentation, mistake, modifiable, molecular, molecularly, moving, multicenter, native, necessarily, necessitate, neurotoxicity, newly, NLTX, NME, nominal, noncompetitive, nondeductible, nonexclusive, notified, nullification, Obama, objectively, obsolescence, oncology, operator, opposition, ordering, organizing, orientation, outbreak, output, outweighed, override, overseen, overtly, package, partner, patentable, pause, PBCIA, pedigree, people, perception, persuasive, pertaining, pharmacoeconomic, pharmacokinetic, pharmacologic, positively, practically, practicing, precisely, precursor, predecessor, predictably, predominantly, presence, prevail, problematic, propose, provisional, pursued, questioned, quorum, race, radiation, RCC, realizability, receptor, reconsider, recoup, recourse, reexamination, refined, reimbursable, reimbursed, relieve, religion, renal, repaid, reproducibility, reproductive, resale, resembling, resistance, restructured, resubmission, retroactively, ripple, rotation, routine, RSU, scaling, sequence, sequentially, sex, sexual, sharing, shift, ship, shipment, similarity, simple, solid, sophistication, speaking, spread, staffing, standing, Stanford, steric, sterilization, stopped, strain, strategically, Street, strive, subjecting, substantive, subunit, successor, sued, suggesting, suit, surgery, talented, targeting, temporarily, tenant, terminology, TGA, therefrom, thermodynamic, threatened, threshold, tolerate, tracing, transplantation, TRICARE, trillion, tumor, unapproved, unconstitutional, understood, underwriting, underwritten, undisclosed, uniformly, unit, unmarketable, unproven, unresolved, unsolicited, unstable, unused, unviable, urged, UW, vast, verified, vi, Wall, water, weather, wide, widen, workforce, workplace
Financial report summary
?Competition
Mylan • Pfizer • Acadia Pharmaceuticals • Xencor • Iovance Biotherapeutics • Allergan • Mylan II B.V. • Voyager Therapeutics • Inhibrx • Werewolf TherapeuticsRisks
- We have a limited operating history, have not completed any clinical trials, and have no products approved for commercial sale, and our results may vary from quarter to quarter.
- We will require substantial additional capital to finance our operations in the future. If we are unable to raise such capital when needed, or on acceptable terms, we may be forced to delay, reduce or eliminate clinical trials, product development programs or future commercialization efforts.
- We have incurred significant losses since inception, expect to incur significant losses for the foreseeable future and may not be able to achieve or sustain profitability in the future. We have no products for sale, have not generated any product revenue and may never generate product revenue or become profitable.
- Risks Related to Discovery, Development and Commercialization
- We face competition from entities that have developed or may develop programs for the diseases we plan to address with NGN-401 and NGN-101 or other product candidates.
- NGN-401, NGN-101 and our other programs are in early stages of development and may fail in development or suffer delays that materially and adversely affect their commercial viability. If we or our current or future collaborators are unable to complete development of, or commercialize, our product candidates, or experience significant delays in doing so, our business will be materially harmed.
- We currently utilize adeno-associated virus serotype 9 (“AAV9”) capsid for delivery of therapeutic transgenes to deliver our product candidates, which may limit the safety, purity, and efficacy or potency of such product candidates.
- We intend to identify and develop novel gene therapy product candidates, which makes it difficult to predict the time, cost and potential success of product candidate development.
- The disorders we seek to treat have low prevalence and it may be difficult to identify and enroll patients with these disorders. If we experience delays or difficulties in the enrollment and/or maintenance of patients in clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.
- We are substantially dependent on the success of our most advanced product candidates, NGN-401 and NGN-101, and our ongoing and anticipated clinical trials of such candidates may not be successful.
- Our programs are focused on the development of therapeutics for patients with neurological diseases, which is a rapidly evolving area of science, and the approach we are taking to discover and develop product candidates is novel and may never lead to approved or marketable products.
- If we do not achieve our projected development goals in the time frames we announce and expect, the commercialization of NGN-401 or NGN-101 or any other product candidates may be delayed and, as a result, our stock price may decline.
- Preclinical and clinical development involves a lengthy and expensive process that is subject to delays and uncertain outcomes, and results of earlier studies and trials may not be predictive of future clinical trial results. If our preclinical studies and clinical trials are not sufficient to support regulatory approval of any of our product candidates, we may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development of such product candidate.
- Preliminary, “topline” or interim data from our clinical trials that we announce or publish from time to time may change as more patient data becomes available and are subject to audit and verification procedures.
- Our current or future clinical trials may reveal significant adverse events or undesirable side effects not seen in our preclinical studies and may result in a safety profile that could halt clinical development, inhibit regulatory approval or limit commercial potential or market acceptance of any of NGN-401 or NGN-101 or any other product candidates or result in potential product liability claims.
- We may expend our limited resources to pursue a particular product candidate, such as NGN-401 or NGN-101, and fail to capitalize on candidates that may be more profitable or for which there is a greater likelihood of success.
- Even if regulatory approval is obtained, any approved products resulting from NGN-401 or NGN-101 or any other product candidate may not achieve adequate market acceptance among clinicians, patients, healthcare third-party payors and others in the medical community necessary for commercial success and we may not generate any future revenue from the sale or licensing of such products.
- We have never commercialized a product candidate and may lack the necessary expertise, personnel and resources to successfully commercialize a product candidate on our own or together with suitable collaborators.
- We have never completed any late-stage clinical trials and may not be able to file an IND application or other applications for regulatory approval to commence additional clinical trials on the timelines we expect. Even if we are able to complete such trials, the FDA or comparable foreign regulatory authorities may not permit us to proceed or could suspend or terminate any such trial after it has been initiated.
- Risks Related to Manufacturing
- Gene therapies are novel, complex and difficult to manufacture. We could experience manufacturing problems that result in delays in the development or commercialization of our product candidates or otherwise harm our business.
- Neurogene and our contract manufacturers for AAV9 are subject to significant regulation with respect to manufacturing of our products. The third-party manufacturing facilities on which we rely, our in-house manufacturing facility, and any manufacturing facility that we may have in the future, may have limited capacity or fail to meet the applicable stringent regulatory requirements.
- We depend on third-party suppliers for materials used in the manufacture of our product candidates, and the loss of these third-party suppliers or their inability to supply us with adequate materials could harm our business.
- Delays in developing our manufacturing capabilities or failure to achieve operating efficiencies from such capabilities may require us to devote additional resources and management time to manufacturing operations and may delay our product development timelines.
- Any contamination or interruption in our manufacturing process, shortages of raw materials or failure of our suppliers to deliver necessary components could result in delays in our clinical development or marketing schedules.
- We may not be able to successfully manufacture our product candidates in sufficient quality and quantity, which would delay or prevent us from developing our product candidates and commercializing resulting approved products, if any.
- Changes in methods of product candidate manufacturing or formulation may result in additional costs or delay.
- Risks Related to Our Reliance on Third Parties
- We have a number of academic collaborations, and currently rely on our collaboration with the University of Edinburgh for certain aspects of our preclinical research and development programs, including working in collaboration to discover and preclinically develop our lead product candidate for Rett syndrome and our near-term future pipeline. Failure or delay of the University of Edinburgh or any other collaborator to fulfil all or part of its obligations under our agreement, a breakdown in collaboration between the parties or a complete or partial loss of the relationship would materially harm our business.
- We currently rely, and intend in the future to rely, on third parties to conduct a significant portion of our preclinical studies and existing clinical trials and potential future clinical trials for product candidates, and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials.
- Risks Related to Our Business and Operations
- In order to successfully implement our plans and strategies, we will need to grow the size of our organization and we may experience difficulties in managing this growth.
- We are highly dependent on our key personnel and anticipate hiring new key personnel. If we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy.
- Our future growth may depend, in part, on our ability to operate in foreign markets, where we would be subject to additional regulatory burdens and other risks and uncertainties.
- Our employees, independent contractors, consultants, commercial collaborators, principal investigators, CROs, CDMOs, suppliers and vendors may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements.
- Our internal computer systems have suffered a security breach and in the future our systems, or those of any of our CROs, manufacturers, other contractors, third party service providers or consultants or potential future collaborators, may fail or suffer additional security or data privacy breaches or other unauthorized or improper access to, use of, or destruction of our proprietary or confidential data, employee data or personal data, which could result in additional costs, loss of revenue, significant liabilities, harm to our brand and material disruption of our operations.
- Our ability to utilize our net operating loss carryforwards and certain other tax attributes may be limited.
- We may be subject to adverse legislative or regulatory tax changes that could negatively impact our financial condition.
- We may acquire businesses or products, or form strategic alliances, in the future, and may not realize the benefits of such acquisitions.
- We maintain our cash at financial institutions, at times in balances that exceed federally-insured limits. The failure of financial institutions could adversely affect our ability to pay our operational expenses or make other payments.
- At the end of August 2023, we identified a material weakness in our internal control over financial reporting and may identify additional material weaknesses in the future that may cause us to fail to meet our reporting obligations or result in material misstatements of our financial statements. If our internal control over financial reporting or our disclosure controls and procedures are not effective, we may not be able to accurately report our financial results, prevent fraud or file our periodic reports in a timely manner, which may cause investors to lose confidence in our reported financial information and may lead to a decline in our share price.
- Risks Related to Intellectual Property
- Our ability to protect our patents and other proprietary rights is uncertain, exposing us to the possible loss of competitive advantage.
- We may not be successful in obtaining or maintaining necessary rights to product candidates through acquisitions and in-licenses.
- Certain of our current product candidates and research programs are licensed from or based upon licenses from a third party and are field limited to certain indications. If these license agreements are terminated or interpreted to narrow our rights, our ability to advance our current product candidates or develop new product candidates based on these technologies will be materially adversely affected.
- If we fail to comply with our obligations in any agreements under which we may license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.
- We may be subject to patent infringement claims or may need to file claims to protect our intellectual property, which could result in substantial costs, liability and diversion of resources, and prevent or delay us from commercializing potential products.
- We may be subject to claims that we have wrongfully hired an employee from a competitor or that employees, consultants or independent contractors have wrongfully used or disclosed confidential information of third parties.
- Changes to patent laws in the United States and other jurisdictions could diminish the value of patents in general, thereby impairing our ability to protect our products.
- Obtaining and maintaining patent protection depends on compliance with various procedural, document submissions, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
- We may not identify relevant third-party patents or may incorrectly interpret the relevance, scope or expiration of a third-party patent, which might adversely affect our ability to develop and market our products.
- We may become subject to claims challenging the inventorship or ownership of our patents and other intellectual property.
- Patent terms may be inadequate to protect our competitive position on our product candidates for an adequate amount of time.
- Some intellectual property that we have in-licensed may have been discovered through government funded programs and thus may be subject to federal regulations such as “march-in” rights, certain reporting requirements and a preference for U.S.-based companies. Compliance with such regulations may limit our exclusive rights, and limit our ability to contract with non-U.S. manufacturers.
- Risks Related to Government Regulation
- The regulatory approval processes of the FDA and other comparable foreign regulatory authorities are lengthy, time-consuming and inherently unpredictable. If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals for our product candidates, we will not be able to commercialize, or will be delayed in commercializing, such product candidates, and our ability to generate revenue will be materially impaired.
- Because gene therapy is novel and the regulatory landscape that governs any product candidates we may develop is rigorous, complex, uncertain and subject to change, we cannot predict the time and cost of obtaining regulatory approval, if received at all, for any product candidates we may develop.
- Disruptions at the FDA and other regulatory authorities could negatively affect the review of our regulatory submissions, which could negatively impact our business.
- We may not be able to meet requirements for the chemistry, manufacturing and control of our product candidates.
- We intend to deliver our product candidates via a drug delivery device that will have its own regulatory, development, supply and other risks.
- We currently and may in the future conduct clinical trials for our product candidates at sites outside the United States, and the FDA may not accept data from trials conducted in such locations.
- Our product candidates for which we intend to seek approval as biologics may face competition sooner than anticipated.
- Even if we receive regulatory approval of NGN-401 or NGN-101 or other product candidates, we will be subject to extensive ongoing regulatory obligations and continued regulatory review, which may result in significant additional expense and we may be subject to penalties if we fail to comply with regulatory requirements or experience unanticipated problems with our product candidates.
- We may face difficulties from healthcare legislative reform measures.
- Our business operations and current and future arrangements with investigators, healthcare professionals, consultants, third-party payors, patient organizations and customers will be subject to applicable healthcare regulatory laws, which could expose us to penalties.
- Even if we are able to commercialize NGN-401 or NGN-101 or other product candidates, due to unfavorable pricing regulations and/or third-party coverage and reimbursement policies, we may not be able to offer such products at competitive prices which would seriously harm our business.
- We are subject to U.S. and certain foreign export and import controls, sanctions, embargoes, anti-corruption laws, and anti-money laundering laws and regulations. We can face criminal liability and other serious consequences for violations, which can harm our business.
- Governments outside the United States may impose strict price controls, which may adversely affect our revenue, if any.
- While we have received Fast Track designation for NGN-401 for the treatment of Rett syndrome and for NGN-101 for the treatment of CLN5 Batten disease and we may seek certain designations for our other product candidates, including Breakthrough Therapy and Priority Review designations in the United States, we may not receive such designations, and even if we do, such designations may not lead to a faster development or regulatory review or approval process.
- The RMAT designation by the FDA for any of our product candidates may not lead to a faster development or regulatory review or approval process and it does not increase the likelihood that our product candidates will receive marketing approval.
- We have received orphan drug designation for NGN-401 for the treatment of Rett syndrome and for NGN-101 for the treatment of CLN5 Batten disease, and we may seek orphan drug designation for certain future product candidates, but we may be unable to obtain such designations or to maintain the benefits associated with orphan drug designation, including market exclusivity, which may cause our revenue, if any, to be reduced.
- We have received Rare Pediatric Disease designation by the FDA for NGN-401 for the treatment of Rett syndrome and for NGN-101 for the treatment of CLN5 Batten disease. However, Rare Pediatric Disease designation for any of our product candidates does not guarantee that the BLA for the product will qualify for a priority review voucher upon approval, and it does not lead to a faster development or regulatory review process, or increase the likelihood that our product candidates will receive marketing approval.
- Our estimates of market opportunity and forecasts of market growth may prove to be inaccurate, and even if the markets in which we compete achieve the forecasted growth, our business may not grow at similar rates, or at all.
- We may become exposed to costly and damaging liability claims, either when testing a product candidate in the clinical or at the commercial stage, and our product liability insurance may not cover all damages from such claims.
- Litigation costs and the outcome of litigation could have a material adverse effect on our business.
- Our business could be adversely affected by economic downturns, inflation, increases in interest rates, natural disasters, public health crises such as the COVID-19 pandemic, political crises, geopolitical events, such as conflicts between Russia and Ukraine and between Israel and the surrounding regions, or other macroeconomic conditions, which could have a material and adverse effect on our results of operations and financial condition.
- The market price of our common stock may continue to be volatile.
- We have incurred, and will continue to incur additional costs and increased demands upon management as a result of complying with the laws and regulations affecting public companies.
- Once we are no longer a smaller reporting company or otherwise no longer qualify for applicable exemptions, we will be subject to additional laws and regulations affecting public companies that will increase our costs and the demands on management and could harm our operating results and cash flows.
- If we fail to maintain proper and effective internal controls, our ability to produce accurate financial statements on a timely basis could be impaired.
- Our certificate of incorporation and bylaws, as well as provisions under Delaware law, could make an acquisition of the company more difficult and may prevent attempts by our stockholders to replace or remove management.
- Our governing documents provide that, unless we consent in writing to the selection of an alternative forum, certain designated courts will be the sole and exclusive forum for certain legal actions between us and our stockholders, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors, officers, employees or agents.
- Future sales of shares by existing stockholders could cause our stock price to decline.
- Our executive officers, directors and principal stockholders have the ability to control or significantly influence all matters submitted to our stockholders for approval.
- We may be exposed to increased litigation, including stockholder litigation, which could have an adverse effect on our business and operations.
- If equity research analysts do not publish research or reports, or publish unfavorable research or reports, about us, our business or our market, our stock price and trading volume could decline.
- We may be subject to adverse legislative or regulatory tax changes that could negatively impact our financial condition.
Management Discussion
- Research and development expenses were $44.4 million for the year ended December 31, 2023, as compared to $47.5 million for the year ended December 31, 2022, a decrease of $3.1 million.
- Expenses related to the Rett syndrome program increased primarily due to a $4.2 million increase in clinical trial costs related to the Phase 1/2 clinical trial of NGN-401, offset by a $2.6 million decrease in preclinical costs. The decrease in expenses related to the Batten disease program was driven primarily by a decrease of $0.1 million in clinical development expenses. The increase in Early Discovery expenses was driven primarily by a $1.5 million increase in preclinical costs. Discontinued Programs expense decreased primarily due to a $1.3 million decrease in preclinical costs, a $1.1 million decrease in chemistry, manufacturing and controls costs, a $0.5 million decrease in clinical trial costs, and $0.5 million decrease in milestone payments. Expenses for Discontinued Programs were substantially complete by year end 2023 and are not expected to contribute meaningfully in 2024.
- The $2.5 million decrease in unallocated internal expenses was primarily driven by a $1.7 million decrease in manufacturing expenses due to lower raw material expenses and a $0.8 million decrease in personnel-related expenses primarily driven by $0.7 million in employee retention tax credits received in 2023.