Core Insights - Actuate Therapeutics announced positive topline results from the Phase 2 trial of elraglusib in combination with gemcitabine/nab-paclitaxel for treating metastatic pancreatic ductal adenocarcinoma, indicating a significant improvement in overall survival [1][2][5] Study Results - The study achieved its primary endpoint with a median overall survival of 10.1 months for the elraglusib/GnP group compared to 7.2 months for the GnP group, resulting in a 37% reduction in the risk of death (HR = 0.63) [2][8] - The 12-month survival rate was 44.1% in the elraglusib/GnP arm versus 22.3% in the GnP arm, demonstrating a doubling of survival rates [2][8] - Continued survival benefits were observed at 18 and 24 months, with survival rates of 19.7% vs 4.4% and 13.8% vs 0% for elraglusib/GnP compared to GnP alone, respectively [8] - The overall response rate was numerically improved at 29.0% for the elraglusib/GnP arm compared to 21.8% for the GnP arm [8] Safety Profile - The trial met its primary safety endpoint, with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) in the elraglusib/GnP arm being similar to those in the GnP arm, indicating a favorable risk-benefit profile [8] - Most treatment-related adverse events (TRAEs) were Grade 1-2, with transient visual impairments being the most common [8] Mechanism of Action - Elraglusib works by inhibiting GSK-3β, which may enhance chemotherapy activity, activate anti-tumor immunity, and regulate gene expression, potentially leading to improved survival outcomes [10][11] Clinical Context - The KOL event highlighted the unmet need in mPDAC and the potential of elraglusib to reshape treatment paradigms, emphasizing the importance of new drugs with unique mechanisms [4][5]

Core Insights - Actuate Therapeutics, Inc. presented topline results from the Phase 2 trial of elraglusib in combination with gemcitabine/nab-paclitaxel for treating metastatic pancreatic ductal adenocarcinoma, demonstrating significant improvements in overall survival [1][2][5] Efficacy - The trial met its primary endpoint, showing a median overall survival (mOS) of 10.1 months for the elraglusib combination group compared to 7.2 months for the control group, with a hazard ratio (HR) of 0.63 and a log-rank p-value of 0.01, indicating a 37% reduction in the risk of death [2][4][5] - The 12-month survival rate for patients receiving elraglusib with GnP was 44.1%, which is double that of the GnP alone group at 22.3% [2][5] - Continued survival benefits were observed at 18 and 24 months, with survival rates of 19.7% vs 4.4% and 13.8% vs 0% for the elraglusib/GnP combination versus GnP alone, respectively [10] - The overall response rate was numerically improved at 29.0% for the elraglusib combination compared to 21.8% for GnP alone [10] Safety Profile - The trial met its primary safety endpoint, with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) in the elraglusib combination arm being similar to those in the GnP arm, indicating a favorable risk-benefit profile [10] - Most treatment-related adverse events (TRAEs) were Grade 1-2, with transient visual impairments being the most common [10] - Grade 3 or higher neutropenia was observed, but rates of febrile neutropenia and sepsis were similar across both treatment arms [10] Future Plans - Based on the positive clinical results and manageable safety profile, the company plans to engage with the FDA and EMA in the second half of 2025 to discuss a path towards product registration [3][5] - Actuate will host a Key Opinion Leader (KOL) event to discuss the trial results, featuring prominent experts in the field [8]

Core Insights - Actuate Therapeutics, Inc. announced the acceptance of an abstract for an oral presentation at the 2025 ASCO Annual Meeting, focusing on the Phase 2 trial data of elraglusib in combination with GnP for treating metastatic pancreatic ductal adenocarcinoma (mPDAC) [1][2] Group 1: Clinical Trial Results - The topline data from the Actuate-1801 Part 3B trial show clinically meaningful improvements in primary efficacy measures when elraglusib is combined with GnP compared to GnP alone [2] - Preliminary results indicate statistically significant increases in the 1-year survival rate and median overall survival for patients treated with the elraglusib/GnP combination versus those receiving GnP alone [3] - The combination treatment also led to higher Objective Response Rates (ORR) and Disease Control Rates (DCR) compared to the control arm [3] Group 2: Company Statements and Future Outlook - The CEO of Actuate highlighted the significance of achieving statistical significance in increased survival rates in a Phase 2 trial, emphasizing elraglusib's potential as a promising therapy for mPDAC [4] - The company views elraglusib as a highly valuable late-stage asset with substantial regulatory and commercial potential [4] - Actuate plans to host a KOL event on May 31, 2025, to discuss the data presented at ASCO, featuring discussions with key opinion leaders [6][8] Group 3: Study Design and Methodology - The Actuate-1801 Part 3B study is a randomized, controlled Phase 2 trial involving 286 mPDAC patients, comparing elraglusib with GnP to GnP alone [9] - Elraglusib is administered at a dose of 9.3 mg/kg via IV infusion on Day 1 of each week in a 28-day cycle, with median overall survival as the primary endpoint [9] - Secondary endpoints include DCR, ORR, progression-free survival (PFS), and adverse events (AE) [9] Group 4: Mechanism of Action - Elraglusib, a GSK-3β inhibitor, may enhance chemotherapy efficacy, activate anti-tumor immunity, and regulate gene expression, potentially leading to improved survival outcomes [10][11]

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements](index=8&type=section&id=Item%201.%20Financial%20Statements) The company reported a net loss of **$6.3 million** for the quarter ended March 31, 2025, a decrease from the **$8.3 million** loss in the prior year period, with cash and cash equivalents significantly decreasing to **$3.9 million** from **$8.6 million** at the end of 2024, leading to a working capital deficit of **$4.9 million** Condensed Consolidated Balance Sheet Highlights (Unaudited) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $3,889,405 | $8,641,622 | | Total current assets | $4,248,564 | $9,207,900 | | Total assets | $4,483,039 | $9,318,448 | | Total current liabilities | $9,153,954 | $8,802,503 | | Total liabilities | $9,570,776 | $9,214,262 | | Total stockholders' equity (deficit) | $(5,087,737) | $104,186 | Condensed Consolidated Statements of Operations (Unaudited) | Account | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Research and development | $3,220,319 | $6,860,430 | | General and administrative | $3,145,265 | $912,824 | | Total operating expenses | $6,365,584 | $7,773,254 | | Loss from operations | $(6,365,584) | $(7,773,254) | | Net loss | $(6,317,024) | $(8,296,059) | | Net loss per share, basic and diluted | $(0.32) | $(5.40) | Condensed Consolidated Statements of Cash Flows (Unaudited) | Activity | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(4,618,740) | $(5,247,582) | | Net cash provided by (used in) financing activities | $(133,477) | $4,357,230 | | Net change in cash and cash equivalents | $(4,752,217) | $(890,352) | | Cash and cash equivalents, end of period | $3,889,405 | $2,068,307 | [Notes to Financial Statements](index=12&type=section&id=Notes%20to%20Financial%20Statements) The notes detail the company's critical financial situation, emphasizing the 'going concern' issue due to recurring losses and insufficient cash reserves - The company is a clinical-stage biopharmaceutical firm focused on developing elraglusib, a GSK-3 inhibitor for cancer treatment[33](index=33&type=chunk) - There is substantial doubt about the Company's ability to continue as a going concern, with cash and cash equivalents of **$3.9 million** as of March 31, 2025, not expected to satisfy operational requirements beyond the second quarter of fiscal year 2025[37](index=37&type=chunk)[38](index=38&type=chunk) - On March 27, 2025, the Company entered into a Committed Equity Facility with B. Riley, allowing it to sell up to **$50 million** of its common stock over 36 months, subject to conditions[39](index=39&type=chunk)[63](index=63&type=chunk) - Subsequent to March 31, 2025, the company issued **72,082 shares** to B. Riley under the Committed Equity Facility for net proceeds of approximately **$597,000**[89](index=89&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=24&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's focus on developing its sole product candidate, elraglusib, for cancer treatment, highlighting a significant decrease in R&D expenses and a sharp increase in G&A expenses, while addressing critical liquidity issues and the **$50 million** Committed Equity Facility as a primary means to address near-term capital requirements [Business Overview](index=24&type=section&id=Business%20Overview) Actuate is a clinical-stage biopharmaceutical company developing elraglusib, a GSK-3β inhibitor, for difficult-to-treat cancers, with its lead program, Actuate-1801, in a Phase 2 trial for metastatic pancreatic cancer (mPDAC) - The company's lead investigational product is elraglusib, a small molecule GSK-3β inhibitor for cancer treatment[92](index=92&type=chunk) - The most advanced clinical program is a Phase 2 trial (Actuate-1801) evaluating elraglusib for metastatic pancreatic ductal adenocarcinoma (mPDAC)[93](index=93&type=chunk)[95](index=95&type=chunk) - The company has incurred significant operating losses since inception, with a net loss of **$6.3 million** for Q1 2025 and an accumulated deficit of **$138.7 million** as of March 31, 2025[98](index=98&type=chunk) [Results of Operations](index=28&type=section&id=Results%20of%20Operations) For Q1 2025, total operating expenses decreased to **$6.4 million** from **$7.8 million** in Q1 2024, driven by a **$3.6 million** reduction in R&D expenses, offset by a **$2.2 million** rise in G&A expenses Comparison of Results of Operations (in thousands) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | Change | | :--- | :--- | :--- | :--- | | Research and development | $3,220 | $6,860 | $(3,640) | | General and administrative | $3,145 | $913 | $2,232 | | **Total operating expenses** | **$6,366** | **$7,773** | **$(1,407)** | | **Net loss** | **$(6,317)** | **$(8,296)** | **$1,979** | - R&D expenses decreased by **$3.6 million**, mainly due to a **$3.3 million** reduction in external clinical trial expenses related to fewer patients on study in the randomized Phase 2 mPDAC trial (Actuate-1801 Part 3B)[116](index=116&type=chunk) - G&A expenses increased by **$2.2 million**, primarily due to a **$1.2 million** increase in personnel-related expenses (including **$920,917** in non-cash stock-based compensation) and higher costs for D&O insurance, board fees, and legal fees from operating as a public company[117](index=117&type=chunk) [Liquidity and Capital Resources](index=30&type=section&id=Liquidity%20and%20Capital%20Resources) The company's financial position is critical, with only **$3.9 million** in cash and cash equivalents as of March 31, 2025, insufficient to support operations beyond the second quarter of 2025, necessitating substantial additional capital - As of March 31, 2025, the company had cash and cash equivalents of **$3,889,405**[119](index=119&type=chunk) - Existing cash and cash equivalents are not sufficient to fund operations beyond the second quarter of fiscal year 2025[121](index=121&type=chunk)[100](index=100&type=chunk) - The company entered into a Committed Equity Facility with B. Riley, providing the right to sell up to **$50 million** in common stock over 36 months to raise capital[124](index=124&type=chunk) - Failure to raise additional funds may require the company to delay, limit, reduce, or terminate its product development programs or even cease operations[123](index=123&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=34&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a "smaller reporting company," Actuate Therapeutics, Inc. is not required to provide the information for this item - The company is not required to provide quantitative and qualitative disclosures about market risk because it qualifies as a "smaller reporting company" under SEC rules[144](index=144&type=chunk) [Item 4. Controls and Procedures](index=34&type=section&id=Item%204.%20Controls%20and%20Procedures) Management, including the CEO and CFO, evaluated the company's disclosure controls and procedures as of March 31, 2025, concluding they were effective at a reasonable assurance level - Based on an evaluation as of March 31, 2025, the company's management, including the CEO and CFO, concluded that disclosure controls and procedures were effective at a reasonable assurance level[146](index=146&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=35&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently subject to any pending legal proceedings that are considered significant - To the company's knowledge, it is not subject to any pending legal proceedings[57](index=57&type=chunk)[148](index=148&type=chunk) [Item 1A. Risk Factors](index=35&type=section&id=Item%201A.%20Risk%20Factors) This section reiterates and expands upon the significant risks facing the company, including its critical financial condition, reliance on a single drug substance manufacturer in China, and uncertainties associated with the Committed Equity Facility [Risks Related to Our Financial Condition and Capital Requirements](index=35&type=section&id=Risks%20Related%20to%20Our%20Financial%20Condition%20and%20Capital%20Requirements) The company's financial condition raises substantial doubt about its ability to continue as a going concern, requiring immediate and substantial additional capital to finance operations beyond Q2 2025 - The company's financial condition raises substantial doubt about its ability to continue as a going concern, a conclusion also noted by its independent registered public accounting firm[152](index=152&type=chunk)[154](index=154&type=chunk) - Existing cash and cash equivalents are not sufficient to fund operations beyond the second quarter of fiscal year 2025[157](index=157&type=chunk) - The company requires substantial additional capital in the near term to finance operations, and failure to obtain it could force a delay, reduction, or termination of development programs or a cessation of operations[156](index=156&type=chunk)[162](index=162&type=chunk) [Risks Related to Our Reliance on Third Parties](index=37&type=section&id=Risks%20Related%20to%20Our%20Reliance%20on%20Third%20Parties) The company faces risks due to its reliance on a single manufacturer located in China for the drug substance (DS) of its sole product candidate, elraglusib, exposing it to geopolitical and trade policy vulnerabilities - The company relies on a single manufacturer in China for the drug substance (DS) of elraglusib, making it vulnerable to geopolitical and trade policy changes[163](index=163&type=chunk) - Unfavorable tariffs or other trade actions could increase the cost of the drug substance, adversely affecting the company's financial condition and results of operations[163](index=163&type=chunk)[165](index=165&type=chunk) [Risks Related to the Committed Equity Facility](index=38&type=section&id=Risks%20Related%20to%20the%20Committed%20Equity%20Facility) The company cannot predict the actual number of shares it will sell or the proceeds it will receive from the Committed Equity Facility, and sales under this facility could cause substantial dilution to existing stockholders and depress the market price of the common stock - It is not possible to predict the actual number of shares that will be sold or the gross proceeds that will be received under the **$50 million** Committed Equity Facility with B. Riley[166](index=166&type=chunk)[168](index=168&type=chunk) - Sales of a substantial number of shares to B. Riley could depress the market price of the company's common stock and cause significant dilution to other stockholders[172](index=172&type=chunk) - Management will have broad discretion over the use of any net proceeds from the facility, and investors will be relying on their judgment[171](index=171&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=39&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company completed its Initial Public Offering (IPO) on August 14, 2024, raising net proceeds of approximately **$22 million**, with no material change in the planned use of these proceeds - On August 14, 2024, the company completed its IPO, receiving net proceeds of approximately **$22 million** after expenses[173](index=173&type=chunk) - There has been no material change in the planned use of proceeds from the IPO[173](index=173&type=chunk) [Item 5. Other Information](index=39&type=section&id=Item%205.%20Other%20Information) During the quarter ended March 31, 2025, no director or officer of the company adopted or terminated any Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement - No director or officer adopted or terminated a Rule 10b5-1 trading plan during the first quarter of 2025[176](index=176&type=chunk) [Item 6. Exhibits](index=40&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the quarterly report, including the Securities Purchase Agreement and Registration Rights Agreement with B. Riley, amendments to employment agreements, and standard SEC certification filings - Exhibits filed include agreements related to the B. Riley Committed Equity Facility, amendments to executive employment agreements, and CEO/CFO certifications[178](index=178&type=chunk)

Core Insights - Actuate Therapeutics, Inc. is hosting a key opinion leader event at the 2025 ASCO annual meeting to discuss topline clinical data from the randomized Phase 2 study of elraglusib [1][3][7] - The event will feature a discussion moderated by the CEO of Actuate and include four distinguished KOLs from leading medical institutions [2][3] - The focus will be on the clinical relevance and potential impact of the study's findings on treatment paradigms for metastatic pancreatic ductal adenocarcinoma (mPDAC) [3][7] Event Details - The event is scheduled for May 31, 2025, at 6:30 PM CDT, and will be available both in-person and via live webcast [4][7] - Registration details and a replay of the event will be accessible on the Actuate website [4] Featured Speakers - The event will include notable speakers such as Dr. Tanios Bekaii-Saab, Dr. Devalingam Mahalingam, Dr. Rachna Shroff, and Dr. Colin Weekes, all of whom have significant expertise in gastrointestinal cancers [5][6][8][9] ASCO Presentation Details - Actuate will present preliminary results from the randomized Phase 2 study of elraglusib in combination with gemcitabine/nab-paclitaxel versus gemcitabine/nab-paclitaxel alone for previously untreated mPDAC patients [10][11] - The study enrolled 286 patients, randomized in a 2:1 ratio to receive elraglusib or the control treatment [13] - The primary endpoint is the 1-year survival rate, with secondary endpoints including disease control rate, overall response rate, progression-free survival, and adverse events [13] Mechanism of Action - Elraglusib, a GSK-3β inhibitor, may enhance chemotherapy activity, activate anti-tumor immunity, and regulate gene expression, potentially improving survival outcomes [14][15]

Core Insights - Actuate Therapeutics announced that elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) met primary endpoints and achieved statistical significance in the ongoing Phase 2 trial for metastatic pancreatic ductal adenocarcinoma (mPDAC) [1][7] - The topline results indicate a substantial improvement in median overall survival compared to previous data, with details to be presented at the ASCO Annual Meeting on May 31, 2025 [2][7] Company Overview - Actuate Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for high-impact, difficult-to-treat cancers, specifically targeting GSK-3β to inhibit tumor growth [1][8] - The lead investigational drug, elraglusib, aims to enhance chemotherapy activity and activate anti-tumor immunity through various mechanisms [6][8] Clinical Trial Details - The Actuate-1801 Part 3B study is a randomized, controlled Phase 2 trial involving 286 mPDAC patients, comparing elraglusib with GnP versus GnP alone [5] - The primary endpoint of the study is median overall survival, with secondary endpoints including disease control rate (DCR), overall response rate (ORR), progression-free survival (PFS), and adverse events (AE) [5] Presentation Information - The topline data will be presented in an oral session at the ASCO Annual Meeting, with the abstract titled "Preliminary results from the randomized phase 2 study (1801 part 3B) of elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) versus GnP alone" [4][7]

Company Overview - Actuate Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β) [1][3] - The company's lead investigational drug, elraglusib, targets molecular pathways involved in tumor growth and resistance to conventional cancer treatments, including several DNA Damage Response (DDR) pathways [3] Upcoming Events - Daniel Schmitt, President & CEO of Actuate, will present at the Citizens Life Sciences Conference 2025 on May 8, 2025, at 12:30 PM ET [1][2] - The presentation will be accessible via a webcast on the Actuate website, and the management team will participate in one-on-one investor meetings during the event [2]

Core Insights - Actuate Therapeutics, Inc. announced that topline clinical data from its Phase 2 study of elraglusib in combination with gemcitabine/nab-paclitaxel for treating metastatic pancreatic ductal adenocarcinoma has been selected for oral presentation at the 2025 ASCO Annual Meeting [1][2] Company Overview - Actuate Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β) [1][3] - The lead investigational drug, elraglusib, targets molecular pathways involved in tumor growth and resistance to conventional cancer drugs, potentially mediating anti-tumor immunity [3] Presentation Details - The oral presentation will include preliminary results from the randomized Phase 2 study comparing elraglusib in combination with gemcitabine/nab-paclitaxel versus gemcitabine/nab-paclitaxel alone in previously untreated patients with metastatic pancreatic ductal adenocarcinoma [2] - The session is scheduled for May 31, 2025, from 3:00 PM to 6:00 PM CDT, under the title "Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary" [3]

Core Insights - Actuate Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for difficult-to-treat cancers through the inhibition of GSK-3β [1][4] - The company will present data on its investigational drug elraglusib for advanced salivary gland carcinoma at the AACR Annual Meeting 2025 [1][2] - Elraglusib is being studied in combination with chemotherapy and immunotherapy, showing promising response rates in specific cancer populations [2][4] Presentation Details - The poster presentation titled "Elraglusib, a glycogen synthase kinase 3 beta (GSK-3β) inhibitor, plus chemotherapy with or without immunotherapy for advanced salivary gland cancer" will take place on April 29, 2025 [2] - An independent research group will also present findings on the synergistic effects of elraglusib in combination with investigational compounds from Jazz Pharmaceuticals [3] Company Overview - Actuate's lead drug, elraglusib, targets molecular pathways that promote tumor growth and resistance to conventional therapies, potentially enhancing anti-tumor immunity [4]

Financial Condition and Operating Losses - The company has incurred significant operating losses since its inception in January 2015, with an accumulated deficit of approximately $132.4 million as of December 31, 2024[243]. - As of December 31, 2024, the company had approximately $8.6 million in cash and cash equivalents and working capital of approximately $0.4 million, raising substantial doubt about its ability to continue as a going concern[247]. - The company expects to continue incurring substantial operating losses for the foreseeable future as it develops its sole drug candidate, elraglusib[243]. - The company has not generated any revenue from product sales and does not expect to do so in the near future, if ever[244]. - The development of elraglusib and any future product candidates is highly capital-intensive, and the company will require substantial additional capital to finance its operations[249]. - The company plans to address its financial conditions by raising funds from public or private offerings of equity or debt securities, but there is no assurance that such funding will be available on favorable terms[248]. - The company intends to seek additional financing to achieve business objectives, but adequate financing may not be available on acceptable terms[254]. - There is no assurance that the company will be able to secure additional financing in sufficient quantities or at all, which may delay or limit product development[256]. Clinical Development and Regulatory Approval - The company may face delays or difficulties in clinical trials, which could adversely affect its ability to obtain regulatory approvals and commercialize its product candidates[237]. - The company relies on third parties for conducting non-clinical studies and clinical trials, and any failure on their part could adversely impact the company's financial condition[241]. - The company’s future capital requirements will depend on the success of clinical trials and the ability to obtain regulatory approvals, which are uncertain and time-consuming processes[253]. - Regulatory approvals are critical for the commercialization of elraglusib, and the process is expensive and time-consuming[258]. - The company may encounter delays in clinical trials that could preclude regulatory approval and commercialization of its products[259]. - The company has no experience in successfully obtaining regulatory approval, which may hinder its ability to manage related risks[268]. - The success of elraglusib depends on various factors, including successful clinical trials and regulatory submissions[266]. - The company has not yet completed a Phase 2 clinical trial, which is critical for obtaining regulatory approval and commercializing elraglusib[289]. - Safety concerns in clinical trials could adversely affect the prospects for regulatory approval of elraglusib, impacting the company's financial condition[291]. - The company anticipates developing product candidates for use in combination with other oncology drugs, which may complicate clinical trial designs and regulatory approval processes[298]. Manufacturing and Supply Chain Risks - The company relies on third-party manufacturers for the production of elraglusib, which poses risks related to quality control and regulatory compliance[305]. - The company does not control the manufacturing process and is dependent on third-party manufacturers for compliance with cGMP requirements[305]. - The company relies on third-party manufacturers for drug substances and products, with no long-term supply agreements in place, which may delay clinical trials if supplies are not sourced timely[308]. - The current manufacturer for elraglusib's drug substance is located in China, and geopolitical relationships may impact costs and regulatory approvals[317]. - Establishing new suppliers could take up to two years for regulatory approval, potentially delaying clinical development and increasing costs[318]. - The company may face higher than expected costs in manufacturing elraglusib due to inflation, supply chain issues, or component shortages[255]. - The company has not experienced significant supply chain disruptions but acknowledges the risk of future interruptions[309]. Intellectual Property and Legal Risks - The company may face challenges in obtaining licenses from third parties, which could adversely affect its ability to develop and commercialize products[375]. - The company may not have control over the prosecution and maintenance of licensed patents, which could jeopardize its rights to develop and commercialize products[376]. - The company may face claims regarding the inventorship or ownership of its patents and intellectual property, which could lead to litigation and substantial costs, adversely affecting its business and financial condition[378]. - The company may lose valuable intellectual property rights if it is unsuccessful in interference proceedings or other disputes regarding patent validity, which could impact its ability to commercialize drug candidates[380]. - The company may need to litigate or obtain licenses from third parties to develop or market its technologies, which may not be available on commercially reasonable terms[381]. - The company faces significant risks regarding its intellectual property, as patents may be challenged, invalidated, or circumvented, potentially impacting competitive advantage[363]. - The biopharmaceutical industry is characterized by extensive litigation, which could lead to costly and time-consuming legal disputes that may adversely affect the company's business[365]. Market and Competitive Risks - The company faces significant competition in establishing strategic partnerships, which can be time-consuming and costly[325]. - The company is exposed to risks from competitors who may develop superior products or obtain regulatory approvals more quickly[394]. - The commercial success of elraglusib and future product candidates depends significantly on market acceptance among physicians, patients, and healthcare payors, with no assurance of achieving adequate revenue if acceptance is low[342]. - The company currently lacks a marketing and sales organization and must invest significant resources to develop these capabilities, which may delay revenue generation[343]. - Establishing effective marketing, sales, and distribution relationships with third parties is critical, as failure to do so could hinder product commercialization and revenue generation[345]. Compliance and Regulatory Environment - Compliance with cGMP and regulatory requirements is critical, and failure to meet these could delay product approvals and revenue generation[307]. - The company may face increased compliance costs due to various healthcare laws and regulations, which could adversely affect its business operations[399]. - The company is subject to significant risks related to product liability lawsuits, which could lead to substantial costs and reduced demand for its products[410]. - The company is classified as an emerging growth company, allowing it to take advantage of reduced disclosure requirements, which may affect investor attractiveness[404]. Data Privacy and Security Risks - The company relies on third-party vendors for manufacturing and IT infrastructure, which increases vulnerability to disruptions and unauthorized access[415]. - Compliance with health privacy and data protection laws is critical, as violations could lead to government enforcement actions and significant costs[416]. - The company is subject to various state and federal data privacy laws, including the California Consumer Privacy Act, which allows penalties of up to $7,500 per violation[418]. - International data protection laws, including the EU General Data Protection Regulation, impose strict requirements and potential fines of up to 4% of annual worldwide turnover[422]. - The evolving legal framework around privacy could impose significant limitations on data collection and usage practices[424]. Legislative and Pricing Pressures - Current healthcare reform legislation may increase difficulty and costs for the company to obtain coverage and commercialize elraglusib or future product candidates[427]. - The Inflation Reduction Act of 2022 allows HHS to negotiate prices for certain high-expenditure drugs under Medicare, which could significantly impact the pharmaceutical industry[431]. - The American Rescue Plan Act of 2021 eliminates the statutory cap on Medicaid drug rebates starting January 1, 2024, which was previously capped at 100% of a drug's average manufacturer price[430]. - Legislative changes have resulted in reduced Medicare payments to providers, which will remain in effect until 2032 unless further Congressional action is taken[430]. - Increased scrutiny of pharmaceutical pricing practices has led to congressional inquiries and proposed legislation aimed at enhancing transparency and reforming reimbursement methodologies[430]. - The company faces potential pressure on product pricing due to bidding procedures used by regional healthcare authorities and hospitals[432]. - The company anticipates that future legislative changes may further impact drug pricing and reimbursement practices[431].