Actuate Therapeutics (NASDAQ: ACTU) gets FDA IND ok but flags cash risk
Rhea-AI Filing Summary
Actuate Therapeutics filed an 8-K to share that the FDA has cleared an Investigational New Drug application for an oral tablet formulation of elraglusib, allowing a Phase 1/2 study in advanced solid tumors, including metastatic melanoma, NSCLC, colorectal and pancreatic cancers, with initiation planned for the second half of 2026.
The company is shifting its elraglusib program from an IV to an oral formulation after a Phase 2 trial in metastatic pancreatic ductal adenocarcinoma showed improved overall survival when IV elraglusib was added to gemcitabine plus nab-paclitaxel. Actuate also highlights nonclinical and early clinical data supporting use in RAS-driven cancers and rare pediatric cancers such as Ewing sarcoma and neuroblastoma, and notes potential value from a future pediatric Priority Review Voucher.
At the same time, the exhibits reiterate that Actuate’s financial condition raises substantial doubt about its ability to continue as a going concern and that it needs additional capital to fund operations beyond mid-2026, which could force delays or reductions in its development programs if financing is not obtained.
Positive
- None.
Negative
- Going concern and funding risk: The company states that its financial condition raises substantial doubt about its ability to continue as a going concern and that it needs additional capital to finance operations beyond July 2026, with the possibility of delaying or terminating development programs if funding is not secured.
Insights
Clinical momentum for elraglusib is offset by acute financing risk.
Actuate Therapeutics reports FDA clearance of an IND for an oral elraglusib Phase 1/2 trial in advanced cancers, building on prior IV data showing overall survival benefits in metastatic pancreatic ductal adenocarcinoma when added to gemcitabine plus nab-paclitaxel. The strategy is to move from weekly IV dosing to convenient daily tablets targeting higher drug exposure.
The exhibits also describe expansion plans into RAS-driven tumors and rare pediatric indications, with potential upside from a pediatric Priority Review Voucher valued at $150–200 million. However, these opportunities remain contingent on future clinical success and regulatory discussions with the FDA and EMA.
Critically, the company discloses substantial doubt about its ability to continue as a going concern and states it requires additional capital to finance operations beyond July 2026. Without near-term financing on acceptable terms, Actuate may need to delay, limit, reduce or terminate development and commercialization efforts, so the ultimate impact of today’s clinical milestone depends heavily on funding outcomes.
8-K Event Classification
Key Figures
Key Terms
Investigational New Drug regulatory
Phase 1/2 study medical
metastatic pancreatic ductal adenocarcinoma medical
GSK-3β inhibitor medical
Priority Review Voucher regulatory
going concern financial
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CURRENT REPORT
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| Item 7.01. | Regulation FD Disclosure. |
On May 11, 2026, Actuate Therapeutics, Inc. (the “Company”) issued a press release announcing FDA clearance of an Investigational New Drug (“IND”) application to conduct a Phase 1/2 study and future plans to advance the elraglusib development program. A copy of the press release and the Company’s updated corporate presentation is furnished herewith as Exhibit 99.1 and 99.2, respectively, to this Current Report and is incorporated herein by reference into this Item 7.01.
The information in this Item 7.01, including Exhibit 99.1 and 99.2, and the information on the Company’s website, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filings under the Securities Act of 1933, as amended, or the Exchange Act, regardless of any general incorporation language in such filings, unless expressly incorporated by specific reference in such filing.
| Item 9.01. | Financial Statements and Exhibits. |
(d) Exhibits
The following exhibits are furnished with this report:
| Exhibit No. | Description | |
| 99.1 | Press release issued by Actuate Therapeutics, Inc. on May 11, 2026 | |
| 99.2 | Corporate Presentation dated May 11, 2026 | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | |
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| Actuate Therapeutics, Inc. | ||
| Date: May 11, 2026 | By: | /s/ Daniel M. Schmitt |
| Name: Daniel M. Schmitt | ||
| Title: President and Chief Executive Officer | ||
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Exhibit 99.1
Actuate Therapeutics
Announces FDA Clearance of IND for Oral Elraglusib
and Strategic Initiatives to Advance the Elraglusib Development Program
- Elraglusib oral tablet formulation to drive next-phase clinical development and broader clinical use
- FDA cleared IND for Phase 1/2 study of oral elraglusib in advanced cancer patients, with focus on metastatic melanoma, NSCLC, colorectal, and pancreatic cancers with initiation planned for 2H 2026
- Elraglusib plus RAS inhibitor preclinical results expected in mid-2026
- Industry veteran Martin Huber, MD, joins Board of Directors
CHICAGO, Illinois and FORT WORTH, Texas, May 11, 2026 (GLOBE NEWSWIRE) — Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers, today announced key initiatives to advance and expand the potential of the elraglusib development program.
Actuate is prioritizing the development of the elraglusib oral tablet formulation, which is intended to enhance patient convenience, broaden potential clinical utility, and improve the pharmacokinetic exposure of elraglusib across multiple oncology indications. This strategy is supported by an analysis of patient data from the recently completed Phase 2 study in metastatic pancreatic ductal adenocarcinoma (mPDAC), which identified a positive correlation between drug exposure and clinical outcomes, including meaningful improvement in overall survival.
To support this initiative, the Company recently received Investigational New Drug (IND) clearance from FDA to conduct a Phase 1/2 study designed to demonstrate that a higher overall exposure to elraglusib can be achieved with the oral formulation compared to the IV formulation. The study will evaluate the safety and potential efficacy of the oral formulation as a monotherapy in solid tumor patients, including those with metastatic melanoma, NSCLC, colorectal, and pancreatic cancers, based on evidence of efficacy of the IV formulation in prior clinical studies and machine learning therapeutic target analyses.
The Company has worked with the European Medicines Agency (EMA) to obtain specific guidance regarding elements of a trial design for a potential single registration study for IV treatment of mPDAC. As demonstrated by the positive Phase 2 results published in Nature Medicine, elraglusib combined with gemcitabine plus nab-paclitaxel (GnP) showed statistically significant improvement in overall survival in patients that received the IV formulation of elraglusib once weekly. Going forward, the Company will work with the EMA and FDA on specific guidance regarding elements of the trial design for a potential single registration study with the oral tablet formulation of elraglusib.
“Advancing the elraglusib oral tablet is an important element to the success of our overall development strategy,” said Daniel Schmitt, President & Chief Executive Officer of Actuate. “Transitioning from an IV to an oral formulation of elraglusib, which better positions the program by improving patient convenience and broadening clinical and potential commercial utility, is supported by an analysis of the completed Phase 2 data of elraglusib in mPDAC, showing higher exposures are associated with improved clinical activity. With the IND for the oral program authorized, we are planning to advance into clinical development in the second half of 2026 with a clear focus on optimizing exposure, dose, and response.”
Mr. Schmitt continued: “In parallel, we are actively exploring elraglusib’s combination potential in RAS-driven cancers, where early preclinical data show potential for synergy with RAS inhibitors. With a strong mechanistic rationale targeting key survival and resistance pathways, we believe an oral elraglusib has the potential to enhance the activity of RAS-targeted therapies and address adaptive resistance, positioning it as a potential backbone agent in combination regimens. We look forward to further maturing this data, with additional updates expected in mid-2026.
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The planned Phase 1/2 study will initially evaluate oral elraglusib as a monotherapy in patients with advanced solid tumors. The study is intended to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D), while also evaluating safety, pharmacokinetics (PK), and preliminary signals of antitumor activity. In the Phase 2 portion, the study will enroll patients with a higher likelihood of benefitting from elraglusib treatment, including those with melanoma, non-small cell lung cancer (NSCLC), colorectal, and/or pancreatic cancers.”
Finally, the addition of new board member Martin Huber, MD, a highly experienced biotech leader with extensive experience in oncology drug development, further strengthens our strategic and execution capabilities. Dr. Huber is also currently a board member of Syndax Pharmaceuticals and, most recently, was the President and Chief Executive Officer of Mersana Therapeutics until its acquisition by Day One Pharmaceuticals in January 2026. His guidance will be instrumental as we advance the oral elraglusib program and execute on its next phase of growth. “I am excited to join a team that has successfully demonstrated improved survival in patients with pancreatic cancer who were treated with elraglusib,” said Martin Huber, MD, Director, Actuate Therapeutics, Inc. “I look forward to helping the Actuate team advance the new oral formulation in the evolving pancreatic treatment landscape and potentially bring elraglusib to more patients.”
About Actuate Therapeutics, Inc.
Actuate is a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers. Actuate’s lead investigational drug, elraglusib (a novel GSK-3β inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and DNA Damage Response (DDR). Elraglusib may also mediate anti-tumor immunity through the regulation of multiple immune checkpoints and immune cell function.
For additional information, please visit the Company’s website at www.actuatetherapeutics.com or follow us on LinkedIn, X, and Facebook.
Forward Looking Statements
This press release contains forward-looking statements about us, including our and other parties’ clinical trials and development plans, and our industry. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would,” or the negative of these terms or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements related to present facts or current conditions or of historical facts, contained in this press release are forward-looking statements. Accordingly, these statements involve estimates, assumptions, substantial risks and uncertainties which could cause actual results to differ materially from those expressed in them, including but not limited to that preliminary and unpublished data may be subject to change and further interpretation following the availability of more data or following a more comprehensive review of the data and should not be relied upon as a final analysis; clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, results of prior preclinical studies, early clinical trials and sub-group studies are not necessarily predictive of future results and may not correlate with improved responses, and elraglusib may not achieve positive clinical results or favorable preclinical results or receive regulatory approval on a timely basis, if at all; that we may not successfully enroll additional patients or establish or advance plans for further development, including through conversations with the FDA or EMA and the standards such bodies may impose for such development; that elraglusib could be associated with side effects, adverse events or other properties or safety risks, which could delay or preclude regulatory approval, cause us to suspend or discontinue clinical trials or result in other negative consequences; our reliance on third parties to conduct our non-clinical studies and our clinical trials; our reliance on third-party licensors and ability to preserve and protect our intellectual property rights; that we face significant competition from other biotechnology and pharmaceutical companies; our ability to fund development activities, including because our financial condition raises substantial doubt as to our ability to continue as a going concern and we require additional capital to finance our operations beyond July 2026, and a failure to obtain this necessary capital in the near term on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our development programs, commercialization efforts or other operations. In addition, any forward-looking statements are qualified in their entirety by reference to the factors discussed under the heading “Item 1A. Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2025, filed with the SEC on March 26, 2026, and our Quarterly Reports on Form 10-Q, and other filings with the SEC. Because the risk factors referred to above could cause actual results or outcomes to differ materially from those expressed in any forward-looking statements made by us or on our behalf, you should not place undue reliance on any forward-looking statements. Further, any forward-looking statement speaks only as of the date on which it is made. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Unless legally required, we do not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events.
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Investor Contact
Mike Moyer
Managing Director
LifeSci Advisors, LLC
mmoyer@lifesciadvisors.com
Media Contact
Ignacio Guerrero-Ros, Ph.D.
Russo Partners, LLC
Ignacio.guerrero-ros@russopartnersllc.com
(858) 717-231
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Exhibit 99.2
Corporate Overview May 11, 2026
This presentation contains forward - looking statements about us, including our clinical trials and development plans, and our industry, that are based on management’s beliefs and assumptions and on information currently available to our management . The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would,” o r the negative of these terms o r other comparable terminology are intended to identify forward - looking statements, although not all forward - looking statements contain these identifying words . All statements, other than statements related to present facts o r current conditions or of historical facts, contained in this presentation are forward - looking statements . Accordingly, these statements involve estimates, assumptions, substantial risks and uncertainties which could cause actual results to differ materially from those expressed in them, including bu t not limited to that we have incurred significant operating losses, and we expect that we will incur significant operating losses for the foreseeable future ; that our financial condition raises substantial doubt as to our ability to continue as a going concern and we require additional capital to finance our operations beyond the second quarter of fiscal year 2026 , and a failure to obtain this necessary capital in the near term on acceptable terms, o r at all, could force us to delay, limit, reduce o r terminate our development programs, commercialization efforts o r other operations ; that we have a high risk of never generating revenue o r becoming profitable or, if we achieve profitability, we may not b e able to sustain it ; that clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, and results of prior preclinical studies and early clinical trials are not necessarily predictive of future results, and elraglusib may not achieve favorable results in clinical trials o r preclinical studies, and we may not be able to make regulatory submissions o r receive regulatory approval on a timely basis, if at all ; that we may not successfully enroll additional patients o r establish o r advance plans for hase 2 o r other development, including through conversations with the FDA o r EMA and the standards such bodies may impose for such development ; that regulatory approval processes may involve delays, unfavorable determinations or other challenges due to various factors, including government funding, staffing and political uncertainties ; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities and within the medical community ; that elraglusib could b e associated with side effects, adverse events o r other properties o r safety risks, which could delay o r preclude regulatory approval, cause us to suspend o r discontinue clinical trials or result in other negative consequences ; that this presentation includes preliminary and unpublished data which may be subject to change following the availability of more data o r following a more comprehensive review of the data and should not b e relied upon as a final analysis ; that w e do not have, and may never have, any approved products on the market and our business is highly dependent upon receiving approvals from various U . S . and international governmental agencies and will b e severely harmed if we are not granted approval to manufacture and sell our product candidates ; our reliance on third parties to conduct our non - clinical studies and our clinical trials ; our reliance on third - party licensors and ability to preserve and protect our intellectual property rights ; that we currently depend entirely on the success of elraglusib, which is our only product candidate, and if we are unable to advance elraglusib in clinical development, obtain regulatory approval and ultimately commercialize elraglusib, or experience significant delays in doing so, our business will b e materially harmed ; that we face significant competition from other biotechnology and pharmaceutical companies ; that we may not b e successful in our efforts to investigate elraglusib in additional indications and we may expend our limited resources to pursue a new product candidate o r a particular indication for elraglusib and fail to capitalize on product candidates o r indications that may b e more profitable o r for which there is a greater likelihood of success ; that the termination of third - party licenses could adversely affect our rights to important compounds o r technologies ; and our ability to fund development activities, including because our financial condition raises substantial doubt as to our ability to continue as a going concern and w e require additional capital to finance our operations beyond July 2026 , and a failure to obtain this necessary capital in the near term on acceptable terms, o r at all, could force us to delay, limit, reduce o r terminate our development programs, commercialization efforts o r other operations . You are cautioned not to place undue reliance on these forward - looking statements, which speak only as of the date hereof, and we undertake no obligation to update such statements to reflect events that occur o r circumstances that exist after the date hereof . In addition, any forward - looking statements are qualified in their entirety by reference to the factors discussed under the heading “Risk Factors” in our Annual Report on Form 10 - K filed with the SEC on March 26 , 2026 , our Quarterly Reports on Form 10 - Q, and other filings with the SEC . This presentation also contain estimates and other statistical data that we obtained from industry publications and research and studies conducted by third parties relating to market size and growth and other data about our industry . This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates . 1 Forward - Looking Statements
2 Elraglusib – “Pipeline in a Molecule” Therapeutic Potential Across a Broad Range of Cancers Elraglusib is a class - leading GSK - 3 β inhibitor with a novel, multimodal MOA Clinical trials in 500+ patients resulted in complete responses and significant increases in survival in multiple difficult - to - treat cancers Demonstrated synergy with multiple SOCs, and potential synergy with RAS/RAF/MEK inhibitors Oral tablet with high bioavailability is ready to enter clinical trials IP provides exclusivity to 2038 before PTE in all major markets
3 Elraglusib Development Strategy Develop Elraglusib Oral Tablet to expand portfolio • Initiate phase 1/2 trial to expand into additional high value / blockbuster indications Advance pathway to be the backbone of SOC in PDAC • Complete nonclinical research assessing potential synergy between elraglusib and RAS / MEK inhibitors • Bridge clinical development into oral tablet program Advance pediatric indications towards regulatory approval in EWS and neuroblastoma • Pursuing non - dilutive support to pursue topline data in two rare disease indications • Potential regulatory approvals targeted in one or more indications • Potential for pediatric review voucher worth $150 - 200 million
4 Elraglusib: Multimodal MOA Blocks Cancer Survival Pathways Elraglusib targets cancer survival pathways ERK Dabrafenib Encorafenib Vemurafenib Trametinib Binimetinib Cobimetinib Avutometinib Sotorasib Adagrasib Daraxonrasib RAS RAF MEK Ulixertinib Cancer driver pathways NF - κB Cancer cell survival GSK - 3β MHC - I Immune suppression INF γ PD - 1 PD - L1 LAG - 3 TCR Elraglusib RAS pathway inhibitors alone target driver mutations, with treatment escape nodes on NF - κ B pathway
5 Elraglusib Significantly Improves OS in a Most Difficult to Treat Cancer mPDAC: metastatic pancreatic ductal adenocarcinoma; GnP: Gemcitabine + Nab - paclitaxel; OS: Overall survival Survival Probability 0.0 1.0 0.8 0.6 0.4 0.2 Months 7.2 mos. 10.1 mos. 22.3% 12 - month OS 44.4% mOS GnP Elraglusib + GnP 0 4 8 16 20 24 28 32 36 40 44 12 Predefined Safety Population (n=233, 2:1 Elra+ GnP vs GnP alone) Data cut as of Nov 22, 2025 Doubled percentage of 1L mPDAC patients alive at one year in international Phase 2 RCT
6 Elraglusib Significantly Improves Survival with Weekly IV Dosing Randomized Phase 2 trial met primary endpoint SOC: Standard of Care Elraglusib + GnP doubled 1 - year OS vs. GnP alone (p=0.0004) 2.5x increase in 1 - year OS in patients with liver metastases (p=0.0003) Elraglusib + SOC (GnP) improved median OS by 40% ( HR: 0.62 ; p=0.02) Greater benefit seen in patients receiving at least one full cycle (4 weeks) of treatment ( HR:0.58 ; p=0.035) Excellent safety profile with TEAEs/SAEs and discontinuations balanced between treatment groups
7 Greater Elraglusib Exposure Drives Improved OS Outcomes Target Therapeutic IV Dose Identified Multivariable Cox Proportional Hazards Analysis Increased frequency of dosing could further improve OS benefit PK analyses showed patients exceeding exposure C avg of 210 ng/ml with significantly better outcomes (HR= 0.48) ~ 60% of Elra QW patients achieve target exposure
8 Oral Tablet Provides Expedited Pathway to High Dose Exposure Increased frequency of dosing could further improve OS benefit Analysis demonstrates 9.3 mpk QW as Minimally Effective Dose (MED). ~85% of elraglusib 2QW patients vs ~60% QW patients achieve target exposure Oral dosing in models suggest 210 ng/ml target achievable with ~1 - 2 tablets per day in adults. Oral elraglusib daily dosing may achieve exposures to further improve OS Proportion of Patients Achieving Exposure Threshold
9 Two tablets could replace an IV infusion and increase overall exposure with daily dosing Nonclinical studies show >95% bioavailability, enabling higher dosing IND clearance received from FDA to initiate Ph1/2 study Potential first clinical candidate indications include: Elraglusib Tablet to Expand Portfolio of Blockbuster Indications Potential to be ready for pivotal studies by 2H 2027 Refractory Metastatic NSCLC 1 st line mPDAC treatment Metastatic Colorectal Cancer CPI Refractory, Metastatic Melanoma TAM: ~$4 billion TAM: ~$ 10 billion TAM: ~$12 billion TAM: ~$27 billion PDAC: https://www.fortunebusinessinsights.com/pancreatic - cancer - treatment - market - 101989 Melanoma: https://www.thebusinessresearchcompany.com/report/metastatic - melanoma - therapeutics - global - market - report CRC: https://www.gminsights.com/industry - analysis/colorectal - cancer - therapeutics - market NSCLC: https://www.databridgemarketresearch.com/reports/global - non - small - cell - lung - cancer - market
Elraglusib in Rare Pediatric Cancers
11 EWS and NBL: Rare Diseases with High Unmet Need 1. Stahl M, Ranft A, Paulussen M, et al.: Risk of recurrence and survival after relapse in patients with Ewing sarcoma. Pediatr Blood Cancer 57 (4): 549 - 53, 2011 2. Van Mater and Wagner. Onco Targets Ther. 2019;12:2279 - 2288. 3. Bagatell R, DuBois SG, Naranjo A, et al. Children's Oncology Group's 2023 blueprint for research: Neuroblastoma. Pediatr Blood Cancer 2023;70 Suppl 6(Suppl 6):e30572. DOI: 10.1002/pbc.30572. 4 Ewing Sarcoma Treatment Market (2025 – 2035 outlook); IMARC Group. https://www.researchnester.com/reports/ewing - sarcoma - treatment - market/6962?utm 5. Neuroblastoma Market Size, Epidemiology, In - Market Drugs Sales, Pipeline Therapies, and Regional Outlook 2025 - 2035. IMARC Group. https://www.imarcgroup.com/neuroblastoma - market Ewing Sarcoma (EWS): 5 year survival: • 10 - 15% for relapsed/refractory or after recurrence 1 • 7% for patients with disease - free interval <2 years 2 Strategic and Commercial Potential Accelerated pathways to registration Combined NBL + EWS treatment market size: $700M - $1B 4,5 • Significant market upside with EWS maintenance therapy development Priority Review Voucher eligible for either NBL or EWS Neuroblastoma (NBL): • Leading cause of pediatric mortality • Survival rates ~ 50% 3 High Unmet Need No uniformly effective SOC for relapsed/refractory Ewing Sarcoma or advanced NBL
12 Durable Responses in Heavily Pre - treated Ewing Sarcoma Patients Phase 1 Solid Tumor Study (n=40): 9 elra monotherapy, 12 elra + irinotecan, 19 elra + cyclophosphamide/topotecan Elraglusib + cyclo/topo patients • 10/19 patients responded/achieved disease control • 12 EWS, 6 R/R patients responded/achieved disease control • 3 objective responses (2 CRs, 1 PR) CMR • 18 year - old EWS patient • Refractory to 6 prior lines of tx • CMR with partial response; 60% reduction in lung target legion • No evidence of recurrence after >2 years CR at First Scan • 20 year - old EWS patient • Refractory to 4 prior lines of tx • CR by CT, CMR by PET • No evidence of disease >3 years after termination of treatment EWS: Ewing Sarcoma; CMR: Complete Metabolic Response; CR: Complete Response; PR: Partial Response
13 Nonclinical Data Support Further Development in Neuroblastoma • Addition of elraglusib to SOC chemoimmunotherapy led to markedly superior survival in independent nonclinical research studies • Data submitted for publication in peer review journal in 2Q26 9 - ING - 41: Scientific name for elraglusib
14 Clinical Data Support Further Development in Neuroblastoma Background ~38% patients with advanced relapsed/refractory disease responded with stable disease or better in Phase 1 clinical study of elraglusib + chemotherapy Complete Response • Last line NBL patient with unfavorable molecular profile • Achieved CR within 6 cycles of treatment • Completed 12 cycles of treatment
15 Elraglusib Development Strategy and Timelines Develop Elraglusib Oral Tablet to expand portfolio • 2H 2026: Initiate Phase 1/2 trial to expand into additional high value / blockbuster indications Advance pathway to be the backbone of SOC in PDAC • Mid - 2026/2H 2026: Complete nonclinical research assessing potential synergy between elraglusib and RAS / MEK inhibitors • Mid - 2027: Bridge clinical development into oral tablet program Advance pediatric indications towards regulatory approval in EWS and neuroblastoma • Pursuing non - dilutive support to pursue topline data in two rare disease indications • Potential regulatory approvals targeted in one or more indications • Potential for pediatric review voucher worth $150 - 200 million
16 Andrew Dorr, MD – VP, Clinical Development • Proven oncology drug development leader with senior roles in the development of multiple blockbuster therapies • COO (Salmedix), CMO (Isis/Ionis Pharmaceuticals), Medical Research Advisor (Eli Lilly), and former NCI leader • Extensive expertise in first - in - human studies, pivotal trial planning, and service on the Steering Committee of the Tamoxifen Breast Cancer Prevention Study; >70 peer - reviewed publications in cancer therapeutics • Eulexin, Taxol, Gemzar, Alimta, Treanda, Avastin, Talzenna Seasoned and Successful Leadership Experienced leadership team with demonstrated ability to develop and commercialize cancer drugs Daniel M. Schmitt – Chief Executive Officer and Founder • 30+ years of biotechnology and pharmaceutical experience across senior executive roles • Led and contributed to the successful development and launch of multiple pharmaceutical products • Exosurf, Zovirax, Valtrex, Adenoscan, Ambisome, Duraclon, Campath, Abraxane, enTrust • Executed ~1B+ in milestone value through licensing, acquisition, and development deals Andrew Mazar, PhD – Chief Operating Officer and Scientific Co - Founder • Co - founder, Chief Scientific Officer and Director, Monopar Therapeutics, Inc. (Nasdaq: MNPR) • Entrepreneur - in - Residence; Professor of Pharmacology; Founding Director, Center for Developmental Therapeutics, Northwestern University • Chief Scientific Officer, Attenuon, LLC • Internationally recognized expert in cancer metastasis and translational oncology • Eleven drugs from discovery through Phase 2 • >250 peer - reviewed publications and book chapters and inventor on >70 patents • Serial entrepreneur with seven start - ups founded Paul Lytle – Chief Financial Officer • 30+ years of finance and accounting experience • 25+ years of public company experience for Nasdaq listed companies • Served as co - founder, CFO, and director for multiple biotech companies • Raised in excess of $500 million in net proceeds from various equity and debt offerings
Nasdaq Global Market: ACTU