Familial cold urticaria

14 of 15 participants enrolled achieved a clinical response 10 of 12 participants in the 120mg cohort achieved a complete response No serious adverse events; no grade 3 or higher adverse events reported Initial data from BEACON study expected week of January 6th, 2025, including 180mg Q8W cohort Company to host conference call and webinar today at 8:00 a.m. EDT Oct. 14, 2024 -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a clinical stage biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address mast cell driven diseases such as chronic spontaneous urticaria (CSU), chronic inducible urticaria (CIndU) and asthma, today reported preliminary data from the Company’s ongoing SPOTLIGHT Phase 1b/2a study of subcutaneous briquilimab in adult participants with cold urticaria (ColdU) or symptomatic dermographism (SD), the two most prevalent sub types of CIndU. 14 of 15 participants (93%) enrolled in both dose cohorts of the study (n=15) achieved a clinical response within the 6-week preliminary analysis period following administration. In the 120mg dose cohort, 10 of 12 participants (83%) experienced a complete response (CR), and 1 participant experienced a partial response (PR). Briquilimab was well tolerated in the study, with no serious adverse events (SAEs) and no grade 3 or higher adverse events (AEs) reported. In alignment with the Company’s clinical development plan, Jasper has obtained regulatory clearance to enroll a 180mg dose cohort (n=12) in the SPOTLIGHT study. Jasper expects to present full data from the SPOTLIGHT study in the first half of 2025. Jasper also announced that it expects to report initial data from all cohorts of the BEACON study in CSU during the week of January 6th, 2025, including the recently added 180mg Q8W dose cohort. “We are very excited to report positive data from the SPOTLIGHT study, our first readout from a clinical trial evaluating briquilimab in chronic urticaria, with briquilimab driving rapid complete responses in over 80% of CIndU patients enrolled in the 120mg cohort,” said Ronald Martell, President and Chief Executive Officer of Jasper. “In addition to the responses observed, we are pleased that briquilimab was well tolerated in the study. These results demonstrate the ability of briquilimab to support optimal biologic dosing by rapidly delivering robust and durable clinical benefit along with a potentially differentiated safety profile. Additionally, we look forward to presenting initial data from all cohorts of the BEACON study in CSU in early January of next year. On behalf of the entire Jasper team, I’d like to thank both the investigators and the patients who participated in both studies, along with their families and caregivers.” SPOTLIGHT Study Design and Data Summary: The SPOTLIGHT study is a Phase 1b/2a open label clinical trial evaluating a single dose of subcutaneous briquilimab in adult patients with ColdU or SD who are refractory to antihistamines. The study will enroll 27 patients across three dose cohorts, 40mg (n=3), 120mg (n=12), and 180mg (n=12). The primary endpoints are safety and tolerability of briquilimab and secondary endpoints are focused on clinical activity and PK/PD, including measurement of serum tryptase and mast cells in skin. To assess clinical activity, participants' symptoms are induced via provocation testing prior to dosing with briquilimab and at defined time points through 12 weeks post-dosing. As of the data cut date of October 10th, 2024, 15 participants have been enrolled in the study and received a single dose of subcutaneous briquilimab (n=3 at 40mg, n=12 at 120mg), with all participants having at least 6 weeks of follow-up post-dosing. 12 participants (n=4 ColdU, n=8 SD) were enrolled in the 120mg dose cohort. Participants had high disease burden as assessed by provocation threshold testing. In the 120mg cohort, mean baseline TempTest® threshold was 20.8°C (range: 15-27°C) for ColdU patients, and mean baseline FricTest® threshold was 3.9 of 4 (range: 3-4) for SD patients. 14 of 15 participants (93%) enrolled in both dose cohorts of the study (n=15) achieved a clinical response to provocation testing within the 6-week preliminary analysis period following treatment. 10 of 12 participants (83%) treated in the 120mg cohort achieved a CR with either their critical temperature threshold improving to at least 4°C for ColdU patients or their FricTest® score improving to 0 for SD patients, and 1 of 12 participants enrolled in the 120mg cohort achieved a PR as their best response. 1 of 3 participants treated in the 40mg cohort achieved a CR and the other two participants achieved a PR as their best response. Complete responses in TempTest® or FricTest® were observed as early as 1 week following dosing. All patients will continue to be assessed for response through week 12, and Jasper expects to present full study results, including the 180mg cohort, in the first half of 2025. Mean baseline serum tryptase for participants in the 120mg cohort was 7.6 ng/ml (range: 3.6-25.7 ng/ml). Significant reductions in tryptase were observed as early as the week 1 assessment and were correlated with the onset of clinical responses. The greatest reduction in mean tryptase of 66% was observed at 2 weeks following treatment in the 120mg cohort. At the week 6 assessment, mean tryptase reduction observed was 31% below baseline and 7 of 12 participants (58%) enrolled in the 120mg cohort continued to maintain a clinical response (CR=6, PR=1). Briquilimab was well tolerated in the study. No SAEs or AEs grade 3 were reported. Furthermore, there were no reported AEs related to hair or skin color changes, hypersensitivity, or anemia. Mild decreases in neutrophil counts were observed, with no participants experiencing neutrophil counts below 1500. “It is very exciting to see initial clinical data showing that treatment with briquilimab can lead to deep clinical benefit shortly after administration, particularly given the difficult-to-treat patient population in antihistamine refractory CIndU,” said Martin Metz, M.D., Professor of Dermatology and Allergy Charité – Universitätsmedizin Berlin. “I am also encouraged by the safety and tolerability profile observed in the SPOTLIGHT study thus far, particularly the lack of hypopigmentation or hair color changes. Patients with CIndU currently have very few treatment options, and I look forward to continuing to support the development of briquilimab in chronic urticarias.” Briquilimab is a targeted aglycosylated monoclonal antibody that blocks stem cell factor from binding to the cell-surface receptor c-Kit, also known as CD117, thereby inhibiting signaling through the receptor. This inhibition disrupts the critical survival signal, leading to the depletion of the mast cells via apoptosis which removes the underlying source of the inflammatory response in mast cell driven diseases such as chronic urticaria. Jasper is currently conducting clinical studies of briquilimab as a treatment in patients with CSU or with CIndU and is initiating a clinical study in patients with asthma. Briquilimab is also currently in clinical studies as a treatment for patients with LR-MDS and as a conditioning agent for cell therapies for rare diseases. To date, briquilimab has a demonstrated efficacy and safety profile in more than 160 dosed participants and healthy volunteers, with clinical outcomes in CIndU, and as a conditioning agent in severe combined immunodeficiency (SCID), acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), Fanconi anemia (FA), and sickle cell disease (SCD). Jasper is a clinical-stage biotechnology company developing briquilimab, a monoclonal antibody targeting c-Kit (CD117) as a therapeutic for chronic mast and stem cell diseases such as chronic urticaria, asthma and lower to intermediate risk MDS and as a conditioning agent for stem cell transplants for rare diseases such as SCD, FA and SCID. To date, briquilimab has a demonstrated efficacy and safety profile in more than 160 dosed participants and healthy volunteers, with clinical outcomes in CIndU and as a conditioning agent in SCID, AML, MDS, FA, and SCD. For more information, please visit us at www.jaspertherapeutics.com. 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Enrollment in BEACON and SPOTLIGHT studies progressing faster than expected; enrollment in the 240mg single-dose cohort of the BEACON study ongoing Additional dosing cohort (180mg Q8W) added to BEACON study Initial data from BEACON study through 240mg dosing cohort to be presented in 4Q24 REDWOOD CITY, Calif., Aug. 13, 2024 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a clinical stage biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address mast cell driven diseases such as chronic spontaneous urticaria (CSU), chronic inducible urticaria (CIndU) and asthma, today announced results for the fiscal quarter ended June 30, 2024, reported recent corporate developments, and provided an update on progress in the BEACON and SPOTLIGHT studies. “We have continued to make excellent progress advancing briquilimab during the second quarter, with patient enrollment proceeding faster than initially expected in the BEACON and SPOTLIGHT studies,” said Ronald Martell, President and Chief Executive Officer of Jasper. “The strong rate of enrollment has given us the opportunity to include additional cohorts in our initial CSU data readout, and we are now planning to present results from dosing cohorts up to 240mg in the fourth quarter of this year. While the company remains blinded to efficacy data from the study, rapid enrollment in BEACON has also given us the flexibility to expand the study to include an additional dosing cohort evaluating briquilimab at 180mg Q8W. This will enable us to generate a more robust dataset to support dose selection for our planned registrational trials in CSU without impacting their timelines.” “We are very pleased with the progress in the BEACON and SPOTLIGHT studies thus far,” said Edwin Tucker, M.D., Chief Medical Officer of Jasper. “With the support of our investigators, the efforts of the Jasper team and the timely review and approval by the Independent Data Monitoring Committee (IDMC) we have been able to quickly proceed through dose escalation on the BEACON study and are now enrolling patients at the highest dose, 240mg. This rapid progress and safety affirmation by the IDMC has enabled expansion of the BEACON study to obtain more clinical insights into the potential benefits of briquilimab for patients with CSU, without delaying the program. We look forward to reviewing and presenting initial data from both the BEACON and SPOTLIGHT studies later this year, followed in early 2025 by the full study reports to be presented at a medical conference.” Highlights for Second Quarter 2024 and Recent Weeks Completed enrollment in the 80mg Q8W, the 120mg Q8W, the 120mg Q12W and the 180mg Q12W cohorts of the BEACON study in CSU. As of August 13th, 2024, the 240mg, single dose cohort is actively enrolling patients and the newly added 180mg, Q8W cohort is expected to be open for enrollment imminently. Jasper has opened 31 clinical sites in the BEACON study across the U.S. and EU to date.Based on the favorable rate of enrollment in the BEACON study, Jasper is now planning to report data from all dosing cohorts initially included in the study in the fourth quarter of 2024, and continued enrollment at the current pace would allow for the inclusion of the recently added 180mg Q8W cohort in the initial data disclosure as well. Endpoints that the company expects to disclose in the readout include UAS7 scores, UCT scores, serum tryptase, and adverse events. Efficacy data from the study remains blinded to the Company until the interim analysis associated with the initial data readout.Expanded the Company’s Phase 1b/2a BEACON study in CSU with the addition of a cohort evaluating 180mg of briquilimab (N=8) administered on an 8-week dosing schedule. The BEACON study is a dose escalation trial evaluating repeat doses of subcutaneous briquilimab in adult CSU patients who remain symptomatic after treatment with, or who cannot tolerate, omalizumab. Parts 2 and 3 of the study are double-blind and placebo controlled, and the Company remains blinded to all efficacy data in the study until the initial data analysis is conducted, currently planned for the fourth quarter of 2024.Enrollment is ongoing in the second cohort (120mg) of the Company’s Phase 1b/2a SPOTLIGHT study in CIndU. The SPOTLIGHT study is evaluating a single administration of subcutaneous briquilimab in adult patients with cold urticaria (ColdU) or symptomatic dermographism (SD). Jasper anticipates reporting initial data from the SPOTLIGHT study in the fourth quarter of 2024.Announced the expansion of the Company’s mast cell portfolio with a new development program in asthma and hosted a KOL webinar on the potential of briquilimab in the indication. Jasper expects to begin enrolling patients in a Phase 1b/2a study in patients with asthma in the fourth quarter of 2024.Announced the appointment of Svetlana Lucas, Ph.D., to Jasper’s Board of Directors; Dr. Lucas adds particular expertise in strategic planning and business development to the Company’s Board.Presented preclinical data demonstrating the effect of briquilimab on hematopoietic stem cells at the European Hematology Association (EHA) Hybrid Congress; the presented study evaluated the molecular basis of inhibition of the stem cell factor (SCF)/c-Kit signaling pathway via briquilimab and its functional impact on healthy human HSC survival, proliferation, and differentiation. Results from the study demonstrate that blocking of SCF/c-Kit signaling by briquilimab does not cause apoptosis of HSCs, and that HSCs cultured in the presence of briquilimab differentiate directly into CD34- cells with higher c-Kit expression and without increased CD38 expression.Presented data from preclinical and healthy volunteer studies of briquilimab, as well as trial-in-progress presentations from its BEACON and SPOTLIGHT clinical studies, at the EAACI Congress 2024. The two preclinical studies presented showed the potential of briquilimab in asthma and atopic dermatitis (AD), respectively. The asthma study demonstrated that a single dose of briquilimab can deplete mast cells in both inflamed and non-inflamed tissue as well as improve lung function in an allergen-induced asthma model. In the AD study, treatment with briquilimab led to a reduction of dermal mast cells and inflammatory leukocytes, indicating that mast cells play a critical role in the pathogenesis of AD and that briquilimab has the potential to reverse AD pathology by depleting those cells. Both preclinical studies utilized Jasper’s proprietary c-Kit Mouse™, which overcomes the limitations of standard models that do not bind antibodies directed at the human c-Kit receptor.Jasper also presented data from the previously disclosed clinical study of briquilimab in healthy volunteers showing that it has a promising safety profile, appears to be well-tolerated, exhibits a favorable pharmacokinetic (PK) profile, and leads to sustained and dose-dependent depletion of mast cells in a cutaneous wound model. The PK and pharmacodynamic (PD) profiles demonstrated in the study support dose selection for Jasper’s ongoing BEACON and SPOTLIGHT clinical trials in CSU and CIndU. Second Quarter Fiscal 2024 Financial Results Cash and cash equivalents as of June 30, 2024, totaled $106.8 million.Research and development expenses for the quarter ended June 30, 2024, were $11.3 million, including stock-based compensation expenses of $0.5 million.General and administrative expenses for the quarter ended June 30, 2024, were $4.7 million, including stock-based compensation expenses of $1.0 million.Jasper reported a net loss of $15.0 million, or basic and diluted net loss per share attributable to common stockholders of $0.97, for the quarter ended June 30, 2024. About Briquilimab Briquilimab (formerly JSP191) is a targeted aglycosylated monoclonal antibody that blocks stem cell factor from binding to the cell-surface receptor c-Kit, also known as CD117, thereby inhibiting signaling through the receptor. This inhibition disrupts the critical survival signal, leading to the depletion of the mast cells via apoptosis which removes the underlying source of the inflammatory response in mast cell driven diseases such as chronic urticaria. Jasper is currently conducting clinical studies of briquilimab as a treatment in patients with CSU or with CIndU and is planning to initiate a clinical study in patients with asthma. Briquilimab is also currently in clinical studies as a treatment for patients with LR-MDS and as a conditioning agent for cell therapies for rare diseases. To date, briquilimab has a demonstrated efficacy and safety profile in more than 145 dosed participants and healthy volunteers, with clinical outcomes as a conditioning agent in severe combined immunodeficiency (SCID), acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), Fanconi anemia (FA), and sickle cell disease (SCD). About Jasper Jasper is a clinical-stage biotechnology company developing briquilimab, a monoclonal antibody targeting c-Kit (CD117) as a therapeutic for chronic mast and stem cell diseases such as chronic urticaria, asthma and lower to intermediate risk MDS and as a conditioning agent for stem cell transplants for rare diseases such as SCD, FA and SCID. To date, briquilimab has a demonstrated efficacy and safety profile in more than 145 dosed participants and healthy volunteers, with clinical outcomes as a conditioning agent in SCID, AML, MDS, FA, and SCD. For more information, please visit us at www.jaspertherapeutics.com. Forward-Looking Statements Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “plan,” “predict,” “potential,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding briquilimab’s potential, including with respect to its potential in mast cell driven diseases such as CSU, CIndU, and asthma, its ability to block SCF/c-Kit signaling and not cause apoptosis of HSCs, the potential for HSCs cultured in the presence of briquilimab to differentiate directly into CD34- cells with higher c-Kit expression and without increased CD38 expression, a single dose’s ability to deplete mast cells in both inflamed and non-inflamed tissue as well as improve lung function in an allergen-induced asthma model, its potential to lead to a reduction of dermal mast cells and inflammatory leukocytes in AD and its potential to reverse AD pathology by depleting those cells, its potential to lead to sustained and dose-dependent depletion of mast cells in a cutaneous wound model, and the promising safety profile of briquilimab in mast cell diseases; Jasper’s expectations regarding its BEACON study, including expected patient enrollment, additional cohorts, expected timing for presentation of data, expected endpoints to be disclosed in the initial readout, including UAS7 scores, UCT scores, serum tryptase, and adverse events, expected expansion of the study, expected generation of a more robust dataset to support dose selection, expected clinical insights into the potential benefits of briquilimab for patients with CSU, the Company’s expectation that the initial data readout for the BEACON will cover up to the 240mg dosing cohort, including the 180mg Q8W cohort, and the planned presentation of the full study report at a medical conference in 2025; Jasper’s expectations regarding its SPOTLIGHT study, including patient enrollment, expected timing for reporting initial data and planned presentation of the full study report at a medical conference in 2025; Jasper’s expectations regarding its planned registration trials in CSU; and Jasper’s expectations regarding a Phase 1b/2a study in asthma patients, including expecting timing of patient enrollment. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that prior study results may not be replicated; the risk that Jasper’s product candidates may not be beneficial to patients or successfully commercialized; patients’ willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jasper’s business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jasper’s business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jasper’s filings with the SEC, including its Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent Quarterly Reports on Form 10-Q. If any of these risks materialize or Jasper’s assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jasper’s assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements. Contacts: Joyce Allaire (investors)LifeSci Advisors617-435-6602jallaire@lifesciadvisors.com Alex Gray (investors)Jasper Therapeutics650-549-1454 agray@jaspertherapeutics.com Lauren Walker (media)Real Chemistry646-564-2156lbarbiero@realchemistry.com JASPER THERAPEUTICS, INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(in thousands, except share and per share data)(unaudited) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Operating expenses Research and development(1)$11,296 $13,297 $21,594 $23,102 General and administrative(1) 4,697 4,530 9,471 8,672 Total operating expenses 15,993 17,827 31,065 31,774 Loss from operations (15,993) (17,827) (31,065) (31,774)Interest income 1,450 1,436 2,836 2,532 Change in fair value of earnout liability — 420 (20) (344)Change in fair value of common stock warrant liability — — — (575)Other expense, net (40) (109) (62) (179)Total other income, net 1,410 1,747 2,754 1,434 Net loss and comprehensive loss$(14,583) $(16,080) $(28,311) $(30,340)Net loss per share attributable to common stockholders, basic and diluted$(0.97) $(1.47) $(2.00) $(3.08)Weighted-average shares used in computing net loss per share attributable to common stockholders, basic and diluted 14,986,367 10,921,239 14,160,634 9,860,392 (1) Amounts include non-cash stock based compensation expense as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Research and development$473 $491 $822 $959 General and administrative 1,009 900 1,829 1,699 Total$1,482 $1,391 $2,651 $2,658 JASPER THERAPEUTICS, INC.CONDENSED CONSOLIDATED BALANCE SHEETS(in thousands)(unaudited) June 30, December 31,Assets 2024 2023 Current assets: Cash and cash equivalents$106,819 $86,887 Prepaid expenses and other current assets 1,861 2,051 Total current assets 108,680 88,938 Property and equipment, net 2,438 2,727 Operating lease right-of-use assets 1,231 1,467 Restricted cash 417 417 Other non-current assets 1,163 1,343 Total assets$113,929 $94,892 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable$2,964 $4,149 Current portion of operating lease liabilities 1,029 972 Earnout liability 20 - Accrued expenses and other current liabilities 6,055 7,253 Total current liabilities 10,068 12,374 Non-current portion of operating lease liabilities 1,287 1,814 Other non-current liabilities 2,264 2,264 Total liabilities 13,619 16,452 Commitments and contingencies Stockholders’ equity: Preferred stock — — Common stock 2 1 Additional paid-in capital 298,219 248,039 Accumulated deficit (197,911) (169,600)Total stockholders’ equity 100,310 78,440 Total liabilities and stockholders’ equity$113,929 $94,892

Phase 3 CSU studies initiated July 2024 Positive topline Phase 2 CIndU 12 week data reported July 2024 supporting Phase 3 advancement 52 week Phase 2 CSU data and full 12 week Phase 2 CIndU data to be reported 2H 2024 Enrollment continues in Phase 2 PN and EOE studies; Phase 2 AD study to start YE 2024 Celldex’s first bispecific for inflammatory diseases, CDX-622, to enter the clinic this year HAMPTON, N.J., Aug. 08, 2024 (GLOBE NEWSWIRE) -- Celldex Therapeutics, Inc. (NASDAQ:CLDX) today reported financial results for the second quarter ended June 30, 2024 and provided a corporate update. “2024 has been a monumental year for Celldex. We presented data establishing clinical benefit and safety for barzolvolimab in two distinct forms of urticaria—chronic spontaneous urticaria and inducible urticaria—both of which support advancement to registration studies,” said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. “Last month, we initiated two of the largest, global studies to be conducted in CSU and we are now planning for the initiation of a second Phase 3 program in CIndU. With ongoing Phase 2 trials also in prurigo nodularis, eosinophilic esophagitis and, by year end, in atopic dermatitis—barzolvolimab is actively delivering on its significant pipeline in a product potential and we remain committed to our goal of bringing this potential new medicine to patients.” Mr. Marucci continued, “As the year progresses, we continue to build on our leadership in the development of mast cell-targeted therapeutics and plan to advance CDX-622, our first bispecific for inflammatory diseases, into the clinic by year end. We think this candidate is an exciting entrant to the field, targeting two complementary pathways that drive chronic inflammation, TSLP and stem cell factor. Importantly, the Company is well capitalized with more than $800M in cash to support the continued advancement and expansion of the barzolvolimab program and our growing pipeline.” Recent Program Highlights Barzolvolimab - KIT Inhibitor Program Barzolvolimab is a humanized monoclonal antibody developed by Celldex that binds the KIT receptor with high specificity and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells, which mediate inflammatory responses such as hypersensitivity and allergic reactions. KIT signaling controls the differentiation, tissue recruitment, survival and activity of mast cells. Chronic Urticarias Celldex initiated a global Phase 3 program in chronic spontaneous urticaria (CSU) in July, consisting of two Phase 3 trials (EMBARQ-CSU1 and EMBARQ-CSU2) designed to establish the efficacy and safety of barzolvolimab in adult patients with CSU who remain symptomatic despite H1 antihistamine treatment. The studies will also include patients who remain symptomatic after treatment with biologics. Both studies are actively enrolling patients. Celldex is completing Phase 2 clinical studies of barzolvolimab for the treatment of chronic spontaneous urticaria (CSU) and the two most common forms of chronic inducible urticaria (CIndU) - cold urticaria (ColdU) and symptomatic dermographism (SD). These randomized, double-blind, placebo-controlled Phase 2 studies are evaluating the efficacy and safety profile of multiple dose regimens of barzolvolimab in patients who remain symptomatic despite antihistamine therapy, to determine the optimal dosing strategies. In July 2024, the Company reported topline 12 week primary endpoint results from the Phase 2 CIndU study. Data from the 196 patients randomized in the study showed that barzolvolimab achieved the primary efficacy endpoint, a statistically significant difference between the percent of patients with a negative provocation test (complete response) compared to placebo at Week 12, in ColdU and in SD across both dose groups. Importantly, barzolvolimab demonstrated rapid, durable and clinically meaningful responses. Barzolvolimab was well tolerated with a favorable safety profile consistent with prior studies. Patients on study will continue to receive barzolvolimab for 20 weeks and the Company plans to present the full 12 week data from this study at an upcoming medical meeting in the second half of 2024.In February 2024, 12 week treatment results were reported from the Phase 2 CSU study at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting. Barzolvolimab achieved the primary efficacy endpoint of the study, a statistically significant mean change from baseline to Week 12 of UAS7 (weekly urticaria activity score) compared to placebo across multiple dosing groups, and was well tolerated. Secondary and exploratory endpoints strongly support the primary endpoint results. Importantly, barzolvolimab demonstrated rapid, durable and clinically meaningful responses, including in patients with prior omalizumab treatment. Patients on study continue to receive barzolvolimab for 52 weeks and the Company plans to report 52 week data in the second half of 2024.In June 2024, data from this study on the impact of barzolvolimab on angioedema were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024. Barzolvolimab profoundly improved angioedema at 12 weeks, demonstrating statistically significant, rapid and durable improvements in the weekly angioedema activity score (AAS7) and additional measures of angioedema control across all doses at Week 12. Additional Indications A Phase 2 study in prurigo nodularis (PN) was initiated in early 2024 and enrollment is ongoing. This randomized, double-blind, placebo-controlled, parallel group study is evaluating the efficacy and safety profile of barzolvolimab in approximately 120 patients with moderate to severe PN who had inadequate response to prescription topical medications, or for whom topical medications are medically inadvisable, including patients who received prior biologics. A Phase 2 study in eosinophilic esophagitis (EoE) was initiated in July 2023 and enrollment is ongoing. This randomized, double-blind, placebo-controlled study is evaluating the efficacy and safety profile of barzolvolimab in approximately 75 patients with active EoE. In May 2024, Celldex announced that atopic dermatitis (AD) has been selected as the fifth indication for the development of barzolvolimab. Barzolvolimab’s novel mast cell depleting mechanism could play an important role in addressing patients with moderate to severe AD who do not achieve complete disease control on currently available systemic therapies. Celldex plans to initiate a Phase 2 study in AD by year end. Bispecific Antibody Platform CDX-622 – Bispecific SCF & TSLP CDX-622 targets two complementary pathways that drive chronic inflammation, potently neutralizing the alarmin thymic stromal lymphopoietin (TSLP) and depleting mast cells via stem cell factor (SCF) starvation. Combined neutralization of SCF and TSLP with CDX-622 is expected to simultaneously reduce tissue mast cells and inhibit Type 2 inflammatory responses to potentially offer enhanced therapeutic benefit in inflammatory and fibrotic disorders. Celldex has completed preclinical, manufacturing and IND-enabling activities for CDX-622 and plans to initiate a Phase 1 study of the candidate in healthy volunteers by the end of 2024. In preclinical studies, CDX-622 inhibits TSLP and SCF with similar potency to both its respective parental mAbs and comparator mAbs in vitro. CDX-622 was well tolerated in a multi-dose 8 week toxicology study in non-human primates and the No Adverse Event Level (NOAEL) was established to be 75 mg/kg, the highest dose level tested. In inflammatory and fibrotic disorders, TSLP is often upregulated and associated with disease severity. Similarly, mast cells drive or contribute to the pathophysiology of many of these disorders and CDX-622 contains a unique SCF neutralizing function that is expected to inhibit and deplete mast cells. CDX-585 – Bispecific ILT4 & PD-1 CDX-585 combines highly active PD-1 blockade with anti-ILT4 blockade to overcome immunosuppressive signals in T cells and myeloid cells. ILT4 is emerging as an important immune checkpoint on myeloid cells. In May 2023, the first patient was dosed in the Phase 1 study of CDX-585. This open-label, multi-center study of CDX-585 is evaluating patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy. Enrollment is ongoing in the dose-escalation portion of the study. Second Quarter 2024 Financial Highlights and 2024 Guidance Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2024 were $802.3 million compared to $823.8 million as of March 31, 2024. The decrease was primarily driven by second quarter cash used in operating activities of $29.3 million, partially offset by proceeds from issuance of stock under employee benefit plans of $3.7 million. At June 30, 2024, Celldex had 66.3 million shares outstanding. Revenues: Total revenue was $2.5 million in the second quarter of 2024 and $2.7 million for the six months ended June 30, 2024, compared to $0.3 million and $1.2 million for the comparable periods in 2023. The increase in revenue was primarily due to an increase in services performed under our manufacturing and research and development agreements with Rockefeller University. R&D Expenses: Research and development (R&D) expenses were $39.7 million in the second quarter of 2024 and $71.3 million for the six months ended June 30, 2024, compared to $26.3 million and $53.0 million for the comparable periods in 2023. The increase in R&D expenses was primarily due to an increase in barzolvolimab clinical trial and personnel expenses, partially offset by a decrease in barzolvolimab contract manufacturing expenses. G&A Expenses: General and administrative (G&A) expenses were $9.1 million in the second quarter of 2024 and $18.2 million for the six months ended June 30, 2024, compared to $7.2 million and $13.9 million for the comparable periods in 2023. The increase in G&A expenses was primarily due to an increase in stock-based compensation and barzolvolimab commercial planning expenses. Net Loss: Net loss was $35.8 million, or ($0.54) per share, for the second quarter of 2024, and $68.7 million, or ($1.10) per share, for the six months ended June 30, 2024, compared to a net loss of $30.5 million, or ($0.65) per share, for the second quarter of 2023, and $59.9 million, or ($1.27) per share, for the six months ended June 30, 2023. Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at June 30, 2024 are sufficient to meet estimated working capital requirements and fund current planned operations through 2027. About Celldex Therapeutics, Inc.Celldex is a clinical stage biotechnology company leading the science at the intersection of mast cell biology and the development of transformative therapeutics for patients. Our pipeline includes antibody-based therapeutics which have the ability to engage the human immune system and/or directly affect critical pathways to improve the lives of patients with severe inflammatory, allergic, autoimmune and other devastating diseases. Visit www.celldex.com. Forward Looking StatementThis release contains "forward-looking statements" made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as "believes," "expects," "anticipates," "intends," "will," "may," "should," or similar expressions. These forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct or that those goals will be achieved, and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, our ability to successfully complete research and further development and commercialization of Company drug candidates, including barzolvolimab (also referred to as CDX-0159), in current or future indications; the uncertainties inherent in clinical testing and accruing patients for clinical trials; our limited experience in bringing programs through Phase 3 clinical trials; our ability to manage and successfully complete multiple clinical trials and the research and development efforts for our multiple products at varying stages of development; the availability, cost, delivery and quality of clinical materials produced by our own manufacturing facility or supplied by contract manufacturers, who may be our sole source of supply; the timing, cost and uncertainty of obtaining regulatory approvals; the failure of the market for the Company's programs to continue to develop; our ability to protect the Company's intellectual property; the loss of any executive officers or key personnel or consultants; competition; changes in the regulatory landscape or the imposition of regulations that affect the Company's products; our ability to continue to obtain capital to meet our long-term liquidity needs on acceptable terms, or at all, including the additional capital which will be necessary to complete the clinical trials that we have initiated or plan to initiate; and other factors listed under "Risk Factors" in our annual report on Form 10-K and quarterly reports on Form 10-Q. All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise. Company ContactSarah CavanaughSenior Vice President, Corporate Affairs & Administration(508) 864-8337scavanaugh@celldex.com Patrick TillMeru Advisors(484) 788-8560ptill@meruadvisors.com CELLDEX THERAPEUTICS, INC.(In thousands, except per share amounts) Three Months Six MonthsConsolidated Statements of Operations Data Ended June 30, Ended June 30, 2024 2023 2024 2023 (Unaudited) (Unaudited)Revenues: Product development and licensing agreements $- $16 $2 $16 Contracts and grants 2,498 252 2,652 1,218 Total revenues 2,498 268 2,654 1,234 Operating expenses: Research and development 39,687 26,252 71,348 53,049 General and administrative 9,128 7,221 18,231 13,861 Total operating expenses 48,815 33,473 89,579 66,910 Operating loss (46,317) (33,205) (86,925) (65,676) Investment and other income, net 10,475 2,703 18,275 5,813 Net loss $(35,842) $(30,502) $(68,650) $(59,863) Basic and diluted net loss per common share $(0.54) $(0.65) $(1.10) $(1.27) Shares used in calculating basic and diluted net loss per share 66,019 47,253 62,445 47,233 Condensed Consolidated Balance Sheet Data June 30, December 31, 2024 2023 (Unaudited) Assets Cash, cash equivalents and marketable securities $802,317 $423,598 Other current assets 9,658 8,095 Property and equipment, net 3,864 4,060 Intangible and other assets, net 29,747 29,874 Total assets $845,586 $465,627 Liabilities and stockholders' equity Current liabilities $27,658 $31,125 Long-term liabilities 4,269 5,331 Stockholders' equity 813,659 429,171 Total liabilities and stockholders' equity $845,586 $465,627