A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Dociparstat Sodium in Combination With Standard Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia

Phase 3 study to evaluate the efficacy and safety of dociparstat sodium in adults with newly diagnosed untreated acute myeloid leukemia (AML) with adverse or intermediate genetic risk.

Start Date30 Apr 2021

Sponsor / Collaborator

Chimerix, Inc.

NCT04389840

/ TerminatedPhase 2/3

A Phase 2/3 Study to Evaluate the Safety and Efficacy of Dociparstat Sodium for the Treatment of Severe COVID-19 in Adults at High Risk of Respiratory Failure

This was a randomized, double-blind, placebo-controlled Phase 2/3 study to evaluate the safety and efficacy of dociparstat sodium in adult patients with acute lung injury (ALI) due to Coronavirus Disease 2019 (COVID-19). This study was designed to determine if dociparstat sodium could accelerate recovery and prevent progression to mechanical ventilation in patients severely affected by COVID-19.

Start Date08 Jul 2020

Sponsor / Collaborator

Chimerix, Inc.

NCT02995655

/ CompletedPhase 1IIT

A Pilot Study of CX-01 Combined With Azacitidine in the Treatment of Relapsed or Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia

The investigators hypothesize that CX-01 will disrupt the bone marrow microenvironment and increase the cytotoxic effects of azacitidine on myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) hematopoietic stem cells by disrupting the High-mobility group box protein 1 (HMGB1) interaction with toll-like receptor 4 (TLR4) and receptors for advanced glycation end products (RAGE), the CXC chemokine CXCL12/chemokine receptor 4 (CXCR4) axis, and by disrupting other leukocyte and vascular adhesion molecules. In addition, CX-01 may also help promote count recovery after treatment given its affinity for platelet factor-4 (PF4).
The selection of CX-01 dose for study in relapsed or refractory MDS and AML has been based upon the dual requirements to have sufficient drug administered to have potential activity but without clinically significant anticoagulation. The study dose chosen (4 mg/kg bolus followed by 0.25 mg/kg/hour) fulfills both of these criteria. In addition, this dose is expected to result in serum levels of CX-01 which are significantly higher than the IC90 identified in preclinical studies for inhibition of HMGB1-RAGE, toll-like receptor 2 (TLR2) and TLR4 interaction. Therefore, the chosen dose represents a rational balance between effective dosing and safety in thrombocytopenic patients with MDS and AML. This dose was previously established to be safe and tolerable when combined with cytarabine and idarubicin in patients with AML.

Start Date07 Apr 2017

Sponsor / Collaborator

Washington University School of Medicine

[+1]

100 Clinical Results associated with Dociparstat sodium

100 Translational Medicine associated with Dociparstat sodium

100 Patents (Medical) associated with Dociparstat sodium

Literatures (Medical) associated with Dociparstat sodium

01 Dec 2021·Molecular medicine (Cambridge, Mass.)Q2 · MEDICINE

2-O, 3-O desulfated heparin (ODSH) increases bacterial clearance and attenuates lung injury in cystic fibrosis by restoring HMGB1-compromised macrophage function

Q2 · MEDICINE

ArticleOA

Author: Kennedy, Thomas P ; Wu, Jiaqi ; Daley, LeeAnne ; Lin, Mosi ; Yang, Xiaojing ; Wang, Haichao ; Mantell, Lin L ; Velagapudi, Uday Kiran ; Mun, Sung Soo ; Gauthier, Alex G ; Patel, Vivek ; Wang, Mao ; Ashby, Charles R ; Talele, Tanaji T

Abstract:

Background:

High mobility group box 1 protein (HMGB1) is an alarmin following its release by immune cells upon cellular activation or stress. High levels of extracellular HMGB1 play a critical role in impairing the clearance of invading pulmonary pathogens and dying neutrophils in the injured lungs of cystic fibrosis (CF) and acute respiratory distress syndrome (ARDS). A heparin derivative, 2-O, 3-O desulfated heparin (ODSH), has been shown to inhibit HMGB1 release from a macrophage cell line and is efficacious in increasing bacterial clearance in a mouse model of pneumonia. Thus, we hypothesized that ODSH can attenuate the bacterial burden and inflammatory lung injury in CF and we conducted experiments to determine the underlying mechanisms.

Methods:

We determined the effects of ODSH on lung injury produced by Pseudomonas aeruginosa (PA) infection in CF mice with the transmembrane conductance regulator gene knockout (CFTR−/−). Mice were given ODSH or normal saline intraperitoneally, followed by the determination of the bacterial load and lung injury in the airways and lung tissues. ODSH binding to HMGB1 was determined using surface plasmon resonance and in silico docking analysis of the interaction of the pentasaccharide form of ODSH with HMGB1.

Results:

CF mice given 25 mg/kg i.p. of ODSH had significantly lower PA-induced lung injury compared to mice given vehicle alone. The CF mice infected with PA had decreased levels of nitric oxide (NO), increased levels of airway HMGB1 and HMGB1-impaired macrophage phagocytic function. ODSH partially attenuated the PA-induced alteration in the levels of NO and airway HMGB1 in CF mice. In addition, ODSH reversed HMGB1-impaired macrophage phagocytic function. These effects of ODSH subsequently decreased the bacterial burden in the CF lungs. In a surface plasmon resonance assay, ODSH interacted with HMGB1 with high affinity (KD = 3.89 × 10–8 M) and induced conformational changes that may decrease HMGB1’s binding to its membrane receptors, thus attenuating HMGB1-induced macrophage dysfunction.

Conclusions:

The results suggest that ODSH can significantly decrease bacterial infection-induced lung injury in CF mice by decreasing both HMGB1-mediated impairment of macrophage function and the interaction of HMGB1 with membrane receptors. Thus, ODSH could represent a novel approach for treating CF and ARDS patients that have HMGB1-mediated lung injury.

Graphic abstract:

01 Nov 2021·Leukemia researchQ4 · MEDICINE

Combination of dociparstat sodium (DSTAT), a CXCL12/CXCR4 inhibitor, with azacitidine for the treatment of hypomethylating agent refractory AML and MDS

Q4 · MEDICINE

Article

Author: Eric Huselton ; Rizwan Romee ; Kirsten Campbell ; John F. DiPersio ; Geoffrey L. Uy ; Stephen Marcus ; Michael P. Rettig ; Feng Gao ; Iskra Pusic ; Peter Westervelt ; Meagan A. Jacoby ; Mark A. Schroeder ; Amanda F. Cashen

Leukemia stem cells utilize cell adhesion molecules like CXCR4/CXCL12 to home to bone marrow stromal niches where they are maintained in a dormant, protected state. Dociparstat sodium (DSTAT, CX-01) is a low anticoagulant heparin with multiple mechanisms of action, including inhibition of the CXCR4/CXCL12 axis, blocking HMGB1, and binding platelet factor 4 (PF-4). We conducted a pilot study adding DSTAT to azacitidine for patients with AML or MDS unresponsive to or relapsed after prior hypomethylating agent therapy, hypothesizing that DSTAT may improve response rates. Twenty patients were enrolled, with a median of 2 prior lines of therapy and 6 cycles of prior hypomethylating agents. Among fifteen patients evaluable for response, there was 1 complete remission, and 3 marrow complete remissions, for a response rate of 27 % among evaluable patients (20 % overall). Hematologic improvement was observed in 5 additional patients. The median overall survival for all enrolled patients was 205 days (95 % CI 119-302). While cytopenias and infections were common, these were not out of proportion to what would be expected in this population of patients undergoing treatment with azacitidine alone. In summary, this trial demonstrated the feasibility of combining DSTAT with azacitidine, with several responses observed, suggesting this combination warrants further study.

01 Jan 2021·Advances in therapyQ3 · MEDICINE

Design and Rationale of a Randomized, Double-Blind, Placebo-Controlled, Phase 2/3 Study Evaluating Dociparstat in Acute Lung Injury Associated with Severe COVID-19

Q3 · MEDICINE

Article

Author: Morrison, Marion E ; Melemed, Allen ; Fuloria, Jyotsna ; Lasky, Joseph A ; Brundage, Tom ; Naderer, Odin ; Lanier, Randall

INTRODUCTION:

The COVID-19 global pandemic caused by the novel coronavirus, SARS-CoV-2, and the consequent morbidity and mortality attributable to progressive hypoxemia and subsequent respiratory failure threaten to overrun hospital critical care units globally. New agents that address the hyperinflammatory "cytokine storm" and hypercoagulable pathology seen in these patients may be a promising approach to treat patients, minimize hospital stays, and ensure hospital wards and critical care units are able to operate effectively. Dociparstat sodium (DSTAT) is a glycosaminoglycan derivative of heparin with robust anti-inflammatory properties, with the potential to address underlying causes of coagulation disorders with substantially reduced risk of bleeding compared to commercially available heparin.

METHODS:

This study is a randomized, double-blind, placebo-controlled, phase 2/3 trial to determine the safety and efficacy of DSTAT added to standard of care in hospitalized adults with COVID-19 who require supplemental oxygen. Phase 2 will enroll 12 participants in each of two dose-escalating cohorts to confirm the safety of DSTAT in this population. Following review of the data, an additional 50 participants will be enrolled. Contingent upon positive results, phase 3 will enroll approximately 450 participants randomized to DSTAT or placebo. The primary endpoint is the proportion of participants who survive and do not require mechanical ventilation through day 28.

DISCUSSION:

Advances in standard of care, recent emergency use authorizations, and positive data with dexamethasone have likely contributed to an increasing proportion of patients who are surviving without the need for mechanical ventilation. Therefore, examining the time to improvement in the NIAID score will be essential to provide a measure of drug effect on recovery. Analysis of additional endpoints, including supportive biomarkers (e.g., IL-6, HMGB1, soluble-RAGE, D-dimer), will be performed to further define the effect of DSTAT in patients with COVID-19 infection.

TRIAL REGISTRATION:

ClinicalTrials.gov identifier; NCT04389840, Registered 13 May 2020.

News (Medical) associated with Dociparstat sodium

25 May 2023

·www.biospace.com

Ceapro Inc. Reports Financial Results for First Quarter 2023 and Provides Corporate Update

– Q1 2023 sales of $3,500,000 vs $6,172,000 in Q1 2022 – Ended the quarter with $12.6 million in cash allowing funding for planned pipeline development EDMONTON, Alberta, May 25, 2023 (GLOBE NEWSWIRE) -- Ceapro Inc.(TSX-V: CZO; OTCQX:CRPOF) (“Ceapro” or the “Company”), a growth-stage biotechnology company focused on the development and commercialization of active ingredients for healthcare and cosmetic industries, today announced financial results and operational highlights for the first quarter ended March 31, 2023. “Our team significantly advanced toward several key milestones related to new product development as well as strategic corporate initiatives. While sales orders were lower during the first quarter of 2023 as compared to the previous all-time record first quarter in 2022, we continue to leverage our solid base business to enable the development of new products and technologies. The priorities being the assessment of avenanthramides as anti-inflammatory products in a Phase 1-2a clinical trial, the scale-up of our PGX Technology for the development of yeast beta glucan as an immune modulator and the commercial scale-up of a malting technology to enable the production and selling of enriched formulations with high concentration of avenanthramides to serve some nutraceutical market segments. In pursuing our objective to successfully position Ceapro as a biopharmaceutical company, the Company expects to advance these projects using our cash on hand while continuing our discussions to assess different market capitalization initiatives and corporate opportunities to unlock value,” commented Gilles Gagnon, M.Sc., MBA, President and CEO. Corporate and Operational Highlights Pipeline Development Clinical Development: Avenanthramides Protocol for Phase 1-2a clinical trial, “A Double-Blind, Placebo-Controlled, Randomized, Adaptive, First-in-Human Study to Assess Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Oral doses of Avenanthramide,” is ready to go at the Montreal Heart Institute. Full approval was received from the Ethics committee and GMP clinical batches were completed by Corealis. Up to 96 subjects may be included in the study and dosages will escalate from 30 mg to 960 mg according to response. Recruitment is expected to commence in Q2 2023. Pre-Clinical Development: Avenanthramides & Oat Beta Glucan Announced positive results from animal studies conducted by Angiogenesis Foundation on a wound healing model. Both avenanthramides and oat beta glucan stimulated wound healing in mice, significantly improved the speed and quality of the healing process and decreased scar formation. More specifically, avenanthramides suppressed inflammatory cells and beta glucan promoted epithelial progenitor (stem) cells at the scar level making the treated skin look more normal than the untreated skin. This is a major discovery in tissue regeneration and these results will support additional claims for commercial and new products formulated with Ceapro’s oat-based products. Yeast Beta Glucan (YBG) Subsequent to quarter, announced positive results from research collaboration with McMaster University researchers demonstrating that PGX-processed YBG is respirable and able to safely and reliably reprogram macrophages in the lungs in pre-clinical mouse models. These results mark another step forward to a go/no-go decision for further evaluation of PGX-YBG microparticles in a Phase 1 clinical study as a potential much-needed therapeutic option for a broad spectrum of fibrotic diseases. Yeast Beta Glucan/Alginate (YBG-ALG) Pursued the development of novel drug delivery systems focusing specifically on alginates (ALG) and yeast beta glucan (YBG), generating composites and cross-linked polymers with tuneable properties, which can form strips, pads, masks, or fast-dissolving orodispersible films. Over 100 runs have been performed with PGX YBG-ALG on the PGX Demo scale and these new chemical complexes appear to be currently the most promising commercial application from a cost evaluation and market potential perspective. The Company believes that the exciting results obtained with YBG and/or ALG formulations impregnated with CoQ10 showing superior bioavailability data than commercially available CoQ10 products are appealing for potential licensing partners. Technology: Malted technology(Avenanthramides): Significantly advanced the scale up for the commercial production of enriched formulations with high concentration of avenanthramides to serve some nutraceutical markets. Pressurized Gas eXpanded Technology (PGX): Significantly advanced the design of the PGX-100 pilot plant in collaboration with a European specialized engineering firm and equipment manufacturer and with a local Alberta based engineering procurement and contract management firm (EPCM), which can handle the local building modifications, electrical work and regulatory requirements. This project includes three phases: (1) the design, (2) modifications of the building at Agri Food Discovery Place (AFDP) in Edmonton and detailed design and construction of the PGX modular skids; and (3) the installation testing and commissioning of the PGX-100 in the AFDP building which is anticipated to occur in the first half of 2024. The PGX-100 pilot plant will be a major step towards commercialization of products coming from the use of the Technology. Corporate: Ceapro Inc. recognized as a Top 50 TSX Venture Exchange company. Pursued licensing discussions with potential partners mostly for YBG-ALG/CoQ10 new chemical entity. Subsequent to quarter, appointed Mr. Michel Regnier as Senior Vice-President, Technical operations. Mr. Regnier is an experienced and respected Operations Executive and Professional Engineer with 20+ years of progressive technical and leadership experience in the medical device, pharmaceutical and aerospace materials manufacturing industries. Financial Highlights for the First Quarter Ended March 31, 2023 Total sales of $3,500,000 for the first quarter of 2023 compared to $6,172,000 for the comparative period in 2022. The decrease compared to last year was primarily due to a significant decrease in product sales of avenanthramides in USA and to postponed shipments of beta glucan to a major Chinese customer. Gross margin of 46% in Q1 2023 compared to 64% in Q1 2022 mostly due to inflationary costs and the lower yield derived from the oats used in this quarter as compared to the oats used in the prior quarter which were of exceptionally high quality. Net loss of $385,000 in Q1 2023 compared to a net profit of $1,813,000 in Q1 2022. Loss was incurred due to lower sales, increased R&D investments as well as increased G&A expenses mostly due to professional fees incurred for assessments of corporate opportunities. Cash on hand of $12.6 million as of March 31, 2023. Positive working capital balance of $18,496,554 as of March 31, 2023. “Despite the fact that inflation is prevailing on all fronts and that sales were mainly impacted by lower orders from USA for avenanthramides, for which we expect the reordering pattern should resume upon completion of re-organization resulting from the spinoff of the consumer division from one major customer, management believes the prospects for the Company remain positive. While we are looking for additional sources of revenues, we feel fortunate to benefit from the support of our long-time partner, Symrise, with whom we have strengthened our relationship in 2022 through the signing of a renewable three-year supply and distribution agreement. This includes minimum annual volumes providing visibility and sustainability of cash flows that will help us to pursue developments and growth opportunities,” concluded Mr. Gagnon. CEAPRO INC. Condensed Interim Consolidated Balance Sheets Unaudited March 31, December 31, 2023 2022 $ $ ASSETS Current Assets Cash and cash equivalents 12,563,469 13,810,998 Trade receivables 2,911,238 2,820,300 Other receivables 54,419 64,808 Inventories (note 4) 4,245,505 3,757,040 Prepaid expenses and deposits 186,949 135,133 Total Current Assets 19,961,580 20,588,279 Non-Current Assets Investment tax credits receivable 854,895 854,895 Deposits 76,954 76,954 Licences (note 5) 11,847 12,588 Property and equipment (note 6) 15,741,886 16,201,755 Total Non-Current Assets 16,685,582 17,146,192 TOTAL ASSETS 36,647,162 37,734,471 LIABILITIES AND EQUITY Current Liabilities Accounts payable and accrued liabilities 1,083,571 1,730,377 Current portion of lease liabilities (note 7) 381,455 370,460 Total Current Liabilities 1,465,026 2,100,837 Non-Current Liabilities Long-term lease liabilities (note 7) 2,151,394 2,248,577 Deferred tax liabilities 990,620 1,095,968 Total Non-Current Liabilities 3,142,014 3,344,545 TOTAL LIABILITIES 4,607,040 5,445,382 Equity Share capital (note 8 (b)) 16,697,712 16,694,625 Contributed surplus (note 8 (e)) 4,847,400 4,714,404 Retained earnings 10,495,010 10,880,060 Total Equity 32,040,122 32,289,089 TOTAL LIABILITIES AND EQUITY 36,647,162 37,734,471 CEAPRO INC. Condensed Interim Consolidated Statements of Net (Loss) Income and Comprehensive (Loss) Income Unaudited 2023 2022 Restated (note 3) Three Months Ended March 31, $ $ Revenue (note 14) 3,494,811 6,171,624 Cost of goods sold 1,888,973 2,457,102 Gross margin 1,605,838 3,714,522 Research and product development 573,687 355,281 General and administration 1,521,445 769,045 Sales and marketing 8,179 5,299 Finance costs (note 10) 88,800 88,035 (Loss) income from operations (586,273 ) 2,496,862 Other (income) expense (note 11) (95,875 ) 123,038 Loss (income) before tax (490,398 ) 2,373,824 Deferred tax (benefit) expense (105,348 ) 560,895 Total net (loss) income and comprehensive (loss) income for the period (385,050 ) 1,812,929 Net (loss) income per common share (note 17): Basic (0.00 ) 0.02 Diluted (0.00 ) 0.02 Weighted average number of common shares outstanding (note 17): Basic 78,251,844 77,686,576 Diluted 78,251,844 78,333,335 CEAPRO INC. Condensed Interim Consolidated Statements of Cash Flows Unaudited 2023 2022 Restated (note 3) Three Months Ended March 31, $ $ OPERATING ACTIVITIES Net (loss) income for the period (385,050 ) 1,812,929 Adjustments for items not involving cash Finance costs 33,800 33,035 Depreciation and amortization 485,253 462,456 Deferred income tax (benefit) expense (105,348 ) 560,895 Share-based payments 134,083 14,914 162,738 2,884,229 CHANGES IN NON-CASH WORKING CAPITAL ITEMS Trade receivables (90,938 ) (1,447,715 ) Other receivables 10,389 (30,108 ) Inventories (488,465 ) 139,629 Prepaid expenses and deposits (34,397 ) (135,299 ) Accounts payable and accrued liabilities relating to operating activities (646,806 ) (77,608 ) (1,250,217 ) (1,551,101 ) Net (loss) income for the period adjusted for non-cash and working capital items (1,087,479 ) 1,333,128 Interest paid (33,800 ) (33,035 ) CASH (USED IN) GENERATED FROM OPERATIONS (1,121,279 ) 1,300,093 INVESTING ACTIVITIES Purchase of property and equipment (24,643 ) (180,897 ) Deposits relating to the purchase of equipment (17,419 ) - Accounts payable and accrued liabilities relating to investing activities - (47,754 ) CASH USED IN INVESTING ACTIVITIES (42,062 ) (228,651 ) FINANCING ACTIVITIES Stock options exercised 2,000 360 Repayment of lease liabilities (86,188 ) (71,219 ) CASH USED IN FINANCING ACTIVITIES (84,188 ) (70,859 ) (Decrease) Increase in cash and cash equivalents (1,247,529 ) 1,000,583 Cash and cash equivalents at beginning of the period 13,810,998 7,780,989 Cash and cash equivalents at end of the period 12,563,469 8,781,572 The complete financial statements are available for review on SEDAR at and on the Company’s website at . About Ceapro Inc. Ceapro Inc. is a Canadian biotechnology company involved in the development of proprietary extraction technology and the application of this technology to the production of extracts and “active ingredients” from oats and other renewable plant resources. Ceapro adds further value to its extracts by supporting their use in cosmeceutical, nutraceutical, and therapeutics products for humans and animals. The Company has a broad range of expertise in natural product chemistry, microbiology, biochemistry, immunology and process engineering. These skills merge in the fields of active ingredients, biopharmaceuticals and drug-delivery solutions. For more information on Ceapro, please visit the Company’s website at . For more information contact: Jenene Thomas JTC Team, LLC Investor Relations and Corporate Communications Advisor T (US): +1 (833) 475-8247 E: czo@jtcir.com Issuer: Gilles R. Gagnon, M.Sc., MBA President & CEO T: 780-421-4555 Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release

Financial StatementPhase 1Executive Change

19 Nov 2021

·www.biospace.com

Chimerix Announces Positive ONC201 Data in Recurrent H3 K27M-mutant Diffuse Midline Glioma to Be Presented at the Society for Neuro-Oncology Annual Meeting

– Plenary Session Presentation on Saturday, November 20 – – Company to Host Conference Call at 8:30 a.m. ET on Monday, November 22 – DURHAM, N.C., Nov. 19, 2021 (GLOBE NEWSWIRE) -- Chimerix, Inc. (NASDAQ:CMRX), a biopharmaceutical company whose mission it is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases, today announced the presentation of positive data from its 50-patient efficacy analysis of ONC201 for the treatment of recurrent H3 K27M-mutant diffuse midline glioma. ONC201 is an orally administered small molecule dopamine receptor D2 (DRD2) antagonist and caseinolytic protease (ClpP) agonist for the treatment of recurrent gliomas that harbor the H3 K27M- mutation. These data will be presented by Isabel Arrillaga-Romany, MD, PhD, Director of Neuro-Oncology Clinical Trials, Massachusetts General Hospital Cancer Center tomorrow, November 20, 2021, during a plenary session at the Society for Neuro-Oncology (SNO) Annual Meeting in Boston, MA. In addition, Dr. Arrillaga-Romany will join management for a conference call on Monday, November 22, 2021 to give an additional overview of the data. Key Data Highlights ONC201 monotherapy exhibited durable and clinically meaningful efficacy in recurrent H3 K27M-mutant diffuse midline glioma (DMG) patients: Response Assessment in Neuro-Oncology Criteria for High Grade Gliomas (RANO-HGG) criteria assessed by dual reader blinded independent central review (BICR) Overall response rate (ORR): 20% (95% CI: 10 – 34%); including one complete response Median duration of response (DOR): 11.2 months (95% CI: 3.8 – not reached) Median time to response: 8.3 months (range: 1.9 – 15.9) Disease control rate (DCR): 40% (95% CI: 26 – 55%) Progression-free survival (PFS): 35% (95% CI: 21 – 49%) at 6 months; 30% (95% CI: 17 – 44%) at 12 months Response Assessment in Neuro-Oncology Criteria for Low Grade Gliomas (RANO-LGG) criteria assessed by dual reader BICR ORR 26% (95% CI: 15 – 40%) Among evaluable patients (those receiving at least 4mg of dexamethasone daily at baseline), 46.7% achieved at least a 50% confirmed reduction in corticosteroid dose Among evaluable patients (those with a baseline performance status (KPS/LPS) score of 80 or lower), 20.6% achieved a confirmed improvement, indicative of improved quality of life. Overall survival 12 months: 57% (95% CI: 41 – 70%) 24 months: 35% (95% CI: 21 – 49%) "The ONC201 data to be presented at SNO show impressive and consistent results in a disease where life expectancy is exceedingly limited. There currently are no effective therapeutic options for patients with recurrent disease after radiation other than palliation. ONC201 results are particularly notable in light of extended wash-out periods required to ensure isolation of ONC201 single agent effect. It sets the stage for future study of ONC201 earlier in treatment," said Mike Sherman, Chief Executive Officer of Chimerix. "This mutation is considered high grade regardless of histology when present in diffuse midline gliomas and yet the consistency in response between RANO-HGG and -LGG criteria is important in that responses in both enhancing and non-enhancing measures of disease confer benefit to patients. Importantly, tumor responses were quite durable and associated with other measures of clinical benefit including sustained reduction in corticosteroid use, performance status improvement, and survival beyond 24 months. In fact, 11 of the 12 evaluable RANO responders (HGG or LGG) also had accompanying corticosteroid reduction or performance status improvement. In the context of an oral therapy that has demonstrated an attractive safety pro prior reports, we are very excited about the potential of ONC201 to help children and adult patients." "Given the very limited treatment options for patients with recurrent H3 K27M-mutant glioma, we are encouraged by the durable tumor regressions seen in some patients treated with ONC201," said Dr. Arrillaga-Romany. This cohort was comprised of the first 50 patients enrolled across five ONC201 clinical studies who met specific criteria based on feedback from the FDA. These patients were two years of age or older, had a measurable diffuse midline glioma with the H3 K27M- mutation, and had evidence of disease progression following prior therapy with at least radiation completed at least 90 days prior to enrollment. One serious adverse event was attributed by an investigator as possibly related to ONC201. Full safety data collection and analysis for this cohort is ongoing. Prior safety review of ONC201 identified the most commonly reported adverse events as nausea/vomiting, fatigue and decreased lymphocyte counts. The U.S. Food and Drug Administration (FDA) granted ONC201 Fast Track Designation for the treatment of adult recurrent H3 K27M-mutant HGG, Rare Pediatric Disease Designation for treatment of H3 K27M-mutant glioma, and Orphan Drug Designations for the treatment of glioblastoma and for the treatment of malignant glioma. Chimerix plans to meet with the FDA in 2022 following completion of ongoing chemistry, manufacturing and controls (CMC) clinical pharmacology studies and natural disease history evaluation. About Recurrent H3 K27M-mutant Glioma Recurrent H3 K27M-mutant glioma is a brain cancer with a particularly poor prognosis. Pediatric patients with recurrent glioma that carries the H3 K27M mutation have an even worse prognosis. Diffuse midline gliomas with this mutation are classified as Grade IV by the World Health Organization. Grade IV gliomas represent the highest grade with the worst prognosis. Conference Call and Webcast Chimerix will host a conference call and live audio webcast to discuss these data on Monday, November 22 at 8:30 a.m. ET. To access the live conference call, please dial 877-354-4056 (domestic) or 678-809-1043 (international) at least five minutes prior to the start time and refer to conference ID 9048088. The call will reference slides that can be accessed on our website under the investor tab. A live audio webcast of the call will also be available on the Investors section of Chimerix’s website, . An archived webcast will be available on the Chimerix website approximately two hours after the event. About Chimerix Chimerix is a biopharmaceutical company whose mission is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. In June 2021, the U.S. Food and Drug Administration granted approval of TEMBEXA for the treatment of smallpox as a medical countermeasure. The Company has two other advanced clinical-stage development programs, ONC201 and dociparstat sodium (DSTAT). ONC201 is in development for recurrent H3 K27M-mutant glioma. DSTAT is in development as a potential first-line therapy in acute myeloid leukemia. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include those relating to, among other things, the status of Chimerix’s oncology programs, and the results of the 50-patient efficacy analysis of ONC201. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are risks that the current clinical study data for ONC201 will not support accelerated, or any, regulatory approval; the anticipated benefits of the acquisition of Oncoceutics may not be realized; the ability to generate positive results in a Phase 3 study in acute myeloid leukemia and subsequent approval for DSTAT; risks that Chimerix will not obtain a procurement contract for TEMBEXA in smallpox in a timely manner or at all; Chimerix’s reliance on a sole source third-party manufacturer for drug supply; risks that ongoing or future trials may not be successful or replicate previous trial results, or may not be predictive of real-world results or of results in subsequent trials; risks and uncertainties relating to competitive products and technological changes that may limit demand for our drugs; risks that our drugs may be precluded from commercialization by the proprietary rights of third parties; and additional risks set forth in the Company's filings with the Securities and Exchange Commission. These forward-looking statements represent the Company's judgment as of the date of this release. The Company disclaims, however, any intent or obligation to update these forward-looking statements. CONTACT: Investor Relations: Michelle LaSpaluto 919 972-7115 ir@chimerix.com Will O’Connor Stern Investor Relations 212-362-1200 will@sternir.com

Orphan DrugSmall molecular drugFast TrackAcquisition

11 Nov 2021

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Chimerix Announces Upcoming Presentations at the Society for Neuro-Oncology Annual Meeting 2021

DURHAM, N.C., Nov. 11, 2021 (GLOBE NEWSWIRE) -- Chimerix (NASDAQ:CMRX), a biopharmaceutical company whose mission it is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases, today announced upcoming data presentations at the Society for Neuro-Oncology (SNO) Annual Meeting 2021, which will be held in Boston, MA from November 19-21, 2021. Details for the late-breaking plenary oral presentation are as follows: Title: Clinical efficacy of ONC201 in recurrent H3 K27M-mutant diffuse midline glioma patients Abstract Number: LTBK-05 Date and Time: November 20, 2021 from 9:37 a.m. – 9:50 a.m. ET Presenter: Isabel Arrillaga-Romany, M.D., Ph. D., Massachusetts General Hospital Details for additional oral and poster presentations are as follows: Title: Single agent activity of ONC201 in non-midline H3 K27M-mutant diffuse gliomas Abstract Number: CTNI-27 Date and Time: November 19, 2021 from 7:30 p.m. - 9:30 p.m. ET Presenter: Yazmin Odia, M.D., M.S., FAAN, Miami Cancer Institute Title: Safety of ONC201 administered two consecutive days per week in pediatric H3 K27M-mutant glioma patients Abstract Number: CTNI-36 Date and Time: November 19, 2021 from 7:30 p.m. - 9:30 p.m. ET Presenter: Sharon L. Gardner, M.D., New York University School of Medicine Title: Dual metabolic reprogramming by ONC201/TIC10 and 2-Deoxyglucose has a strong antiproliferative effect on medulloblastoma cells Abstract Number: EXTH-68 Date and Time: November 19, 2021 from 7:30 p.m. - 9:30 p.m. ET Presenter: Georg Karpel-Massler, M.D., Ph.D., Ulm University Title: Induction of Synthetic Lethality by Activation of Mitochondrial ClpP and Inhibition of HDAC1/2 in Glioblastoma Abstract Number: TAMI-17 Date and Time: November 19, 2021 from 7:30 p.m. - 9:30 p.m. ET Presenter: Trang T. Nguyen, Ph.D., Columbia University Title: Harnessing Cellular Stress for Immune Targeting of DIPGs Abstract Number: EXTH-30 Date and Time: November 19, 2021 from 7:30 p.m. - 9:30 p.m. ET Presenter: Donghang Cheng, Ph.D., University of Texas MD Anderson Cancer Center Title: Serial plasma and CSF cell-free tumor DNA (cf-tDNA) tracking in diffuse midline glioma patients undergoing treatment with ONC201 Abstract Number: BIOM-28 Date and Time: November 21, 2021 from 11:35 a.m. – 11:45 a.m. ET Presenter: Evan Cantor, M.D., J.D., Washington University at St. Louis About Chimerix Chimerix is a biopharmaceutical company whose mission is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. In June 2021, the U.S. Food and Drug Administration granted approval of TEMBEXA for the treatment of smallpox as a medical countermeasure. The Company has two other advanced clinical-stage development programs, ONC201 and dociparstat sodium (DSTAT). ONC201 is in development for recurrent H3 K27M mutant glioma. DSTAT is in development as a potential first-line therapy in acute myeloid leukemia. CONTACT: Investor Relations: Michelle LaSpaluto 919-972-7115 ir@chimerix.com Will O’Connor Stern Investor Relations 212-362-1200 will@sternir.com

100 Deals associated with Dociparstat sodium

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D11187	-	-	-

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Indication	Highest Phase	Country/Location	Organization	Date
Acute Myeloid Leukemia	Phase 3	United States	Chimerix, Inc.	30 Apr 2021
COVID-19	Phase 3	United States	Chimerix, Inc.	08 Jul 2020
Metastatic Pancreatic Cancer	Phase 2	United States	Chimerix, Inc.	01 Nov 2011
Pancreatic Cancer	Phase 2	United States	Cantex Pharmaceuticals, Inc.	01 Nov 2011
Pulmonary Disease, Chronic Obstructive	Phase 2	United States	Cantex Pharmaceuticals, Inc.	01 Apr 2007
Pulmonary Disease, Chronic Obstructive	Phase 2	United States	Chimerix, Inc.	01 Apr 2007
Pulmonary Disease, Chronic Obstructive	Phase 2	Belgium	Chimerix, Inc.	01 Apr 2007
Pulmonary Disease, Chronic Obstructive	Phase 2	Belgium	Cantex Pharmaceuticals, Inc.	01 Apr 2007
Pulmonary Disease, Chronic Obstructive	Phase 2	Canada	Chimerix, Inc.	01 Apr 2007
Pulmonary Disease, Chronic Obstructive	Phase 2	Canada	Cantex Pharmaceuticals, Inc.	01 Apr 2007

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04571645 (AML, CTgov)	Phase 3	Acute Myeloid Leukemia	9	Control	fppcaczbem = czqkupjsbp lldwfhlvpi (wwezgutfxt, lampxbjaxa - hcperxvnpu) View more	-	15 Apr 2024
NCT02056782 (PGX-AML, CTgov)	Phase 1	Acute Myeloid Leukemia \| Acute Megakaryoblastic Leukemia \| Acute Promyelocytic Leukemia	12	Dociparstat sodium	uepqauumqo(lorgsndwup) = dgizrlxzgw samyfoidvx (nknvilqver, hzcdeunjlu - fizqiweuxm) View more	-	21 Feb 2023
NCT04389840 (CTgov)	Phase 2/3	COVID-19	27	Dociparstat sodium (Cohort 1 Dociparstat)	eefjxtplic = jtswumkqse xbuzysvmym (ztqyhimsym, sopeczccsx - muynqhylmu) View more	-	30 Aug 2022
				Placebo (Cohort 1 Placebo)	eefjxtplic = ghmucuqynv xbuzysvmym (ztqyhimsym, rumemunwqo - flefdbothe) View more
NCT02873338 (CTgov)	Phase 2	Acute Myeloid Leukemia	75	Cytarabine+Idarubicin (Control (Idarubicin+Cytarabine))	zcjdocdbak = qunqsrgids ephytahcic (rcivgpsvwa, karubcszqp - ympjchcfki) View more	-	29 Oct 2021
				Dociparstat sodium+Cytarabine+Idarubicin (Dociparstat 0.125 mg/kg)	zcjdocdbak = woeldunwel ephytahcic (rcivgpsvwa, kuntpieunz - zgsvsuuvqh) View more
NCT02873338 (ASCO 12019 \| ASCO 22019) Manual	Phase 2	Acute Myeloid Leukemia First line	66	Idarubicin+Cytarabine+CX-01 (lower dose of CX-01 (0.125 mg/kg/hour))	phksamnhjz(kpqgjkifcp) = lttjmsyvbo ewjxijazvj (ijsexvbzsj ) View more	Positive	01 Jun 2019
				Idarubicin+Cytarabine+CX-01 (higher dose of CX-01 (0.25 mg/kg/hour))	phksamnhjz(kpqgjkifcp) = vagkkctszj ewjxijazvj (ijsexvbzsj )
NCT02995655 (ASCO 12018 \| ASCO 22018) Manual	Phase 1	Acute Myeloid Leukemia \| Myelodysplastic Syndromes FOLR1 Positive	20	azacitidine+CX-01	oqcrhulgfg(rttkagajcq) = uswdmkpojz pbnvqofewu (mropsqslhw ) View more	Positive	04 Jun 2018
NCT00457951 (COPD, CTgov)	Phase 2	Pulmonary Disease, Chronic Obstructive	158	Placebo Comparator: Placebo-Control Arm 0.9% Sodium Chloride (0.9% Sodium Chloride)	bwtxwevleb = oszodofyku gwyzeifdtr (jlkmhhygib, mjgqgfgpby - rziyojoxgi)	-	16 Dec 2016
				ODSH (Randomized, Blinded, ODSH Arm)	bwtxwevleb = sofsaefmwd gwyzeifdtr (jlkmhhygib, otizrlqqyf - ahlqqylijb)
NCT01461915 (ASCO_GI2013)	Phase 2	Metastatic Pancreatic Cancer	10	ODSH + Gemcitabine/nab-paclitaxel	xjxwysesiq(moplxnyjzq) = ngonbzmnuf oszzpddjth (jslhnkvmjy )	-	01 Feb 2013

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