Familial cold urticaria

– ARCALYST® (rilonacept) Q1 2025 net product revenue of $137.8 million, representing 75% year-over-year growth – – ARCALYST 2025 expected net product revenue increased to $590 - $605 million – – KPL-387 Phase 2/3 clinical trial in recurrent pericarditis on track to initiate in mid-2025; Phase 2 data expected in 2H 2026 – – Current operating plan expected to remain cash flow positive on an annual basis – – Conference call and webcast scheduled for 8:30 am ET today – LONDON, April 29, 2025 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals International, plc (Nasdaq: KNSA) (Kiniksa), a biopharmaceutical company developing and commercializing novel therapies for diseases with unmet need, with a focus on cardiovascular indications, today reported first quarter 2025 financial results and recent portfolio execution. “Kiniksa continues to drive strong growth with ARCALYST. In the first quarter of 2025, our robust commercial execution resulted in a meaningful increase in active commercial patients, driven by increases to the prescriber base, longer average total duration of treatment, and changes to Medicare Part D. As a result of strong first quarter performance, we are increasing our expected 2025 ARCALYST net sales to between $590 and $605 million from our previous guidance of between $560 and $580 million,” said Sanj K. Patel, Chairman and Chief Executive Officer of Kiniksa. “Also, we are excited about the potential of KPL-387 to be an additional treatment option for patients by enabling dosing with a single monthly subcutaneous injection in a liquid formulation. We remain on track to initiate the KPL-387 Phase 2/3 recurrent pericarditis trial in the middle of this year.” Portfolio Execution ARCALYST (IL-1α and IL-1β cytokine trap) ARCALYST net product revenue was $137.8 million for the first quarter of 2025. Since launch, more than 3,150 prescribers have written ARCALYST prescriptions for recurrent pericarditis. As of the end of the first quarter of 2025, average total duration of ARCALYST therapy in recurrent pericarditis increased to approximately 30 months, compared to approximately 27 months as of the end of the fourth quarter of 2024. KPL-387 (monoclonal antibody IL-1 receptor antagonist) Pharmacokinetic data from the single ascending dose portion of the Phase 1 study support the development plan for KPL-387; Kiniksa plans to initiate a Phase 2/3 clinical trial in mid-2025. The trial will evaluate KPL-387 in recurrent pericarditis, with a target profile of monthly subcutaneous (SC) dosing in a liquid formulation. The company expects data from the Phase 2 portion of the trial in the second half of 2026. KPL-1161 (Fc-modified monoclonal antibody IL-1 receptor antagonist) Kiniksa is conducting Investigational New Drug (IND)-enabling development activities with a target profile of quarterly SC dosing. Financial Results Total revenue for the first quarter of 2025 was $137.8 million, compared to $79.9 million for the first quarter of 2024. Kiniksa did not record any license and collaboration revenue for the first quarter of 2025, compared to $1.0 million for the first quarter of 2024. Total operating expenses for the first quarter of 2025 were $124.5 million, compared to $96.4 million for the first quarter of 2024. Total operating expenses for the first quarter of 2025 included $43.8 million in collaboration expenses, which are driven by ARCALYST collaboration profitability, compared to $20.8 million for the first quarter of 2024. Total operating expenses for the first quarter of 2025, included $7.7 million in non-cash, share-based compensation expense, compared to $7.2 million for the first quarter of 2024. Net income for the first quarter of 2025 was $8.5 million, compared to a net loss of $17.7 million for the first quarter of 2024. As of March 31, 2025, Kiniksa had $268.3 million of cash, cash equivalents, and short-term investments and no debt. Kiniksa did not record any license and collaboration revenue for the first quarter of 2025, compared to $1.0 million for the first quarter of 2024. Total operating expenses for the first quarter of 2025 included $43.8 million in collaboration expenses, which are driven by ARCALYST collaboration profitability, compared to $20.8 million for the first quarter of 2024. Total operating expenses for the first quarter of 2025, included $7.7 million in non-cash, share-based compensation expense, compared to $7.2 million for the first quarter of 2024. Financial Guidance Kiniksa expects 2025 ARCALYST net product revenue of between $590 million and $605 million, compared to prior guidance of between $560 million and $580 million. Kiniksa expects its current operating plan to remain cash flow positive on an annual basis. Kiniksa continues to monitor potential implications of tariffs on pharmaceuticals imported into the United States. ARCALYST is currently manufactured in the United States by Regeneron Pharmaceuticals. Kiniksa has been in the process of transferring drug substance manufacturing to Samsung Biologics in South Korea and believes any future impact to ARCALYST gross margin would be immaterial, limited only to the cost of drug substance imported into the United States. ARCALYST is currently manufactured in the United States by Regeneron Pharmaceuticals. Kiniksa has been in the process of transferring drug substance manufacturing to Samsung Biologics in South Korea and believes any future impact to ARCALYST gross margin would be immaterial, limited only to the cost of drug substance imported into the United States. Conference Call Information Kiniksa will host a conference call and webcast at 8:30 a.m. Eastern Time on Tuesday, April 29, 2025, to discuss first quarter 2025 financial results and recent portfolio execution. Individuals interested in participating in the call via telephone may register here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. To access the webcast, please visit the Investors and Media section of Kiniksa’s website. A replay of the event will also be available on Kiniksa’s website within approximately 48 hours after the event. About Kiniksa Kiniksa is a biopharmaceutical company dedicated to improving the lives of patients suffering from debilitating diseases by discovering, acquiring, developing, and commercializing novel therapies for diseases with unmet need, with a focus on cardiovascular indications. Kiniksa’s portfolio of assets is based on strong biologic rationale or validated mechanisms and offers the potential for differentiation. For more information, please visit www.kiniksa.com. About ARCALYST ARCALYST is a weekly, subcutaneously injected recombinant dimeric fusion protein that blocks interleukin-1 alpha (IL-1α) and interleukin-1 beta (IL-1β) signaling. ARCALYST was discovered by Regeneron Pharmaceuticals, Inc. (Regeneron) and is approved by the U.S. Food and Drug Administration (FDA) for recurrent pericarditis, cryopyrin-associated periodic syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome and Muckle-Wells Syndrome, and deficiency of IL-1 receptor antagonist (DIRA). The FDA granted Breakthrough Therapy designation to ARCALYST for the treatment of recurrent pericarditis in 2019 and Orphan Drug exclusivity to ARCALYST in 2021 for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and pediatric patients 12 years and older. The European Commission granted Orphan Drug Designation to ARCALYST for the treatment of idiopathic pericarditis in 2021. IMPORTANT SAFETY INFORMATION ABOUT ARCALYST ARCALYST may affect your immune system and can lower the ability of your immune system to fight infections. Serious infections, including life-threatening infections and death, have happened in patients taking ARCALYST. If you have any signs of an infection, call your doctor right away. Treatment with ARCALYST should be stopped if you get a serious infection. You should not begin treatment with ARCALYST if you have an infection or have infections that keep coming back (chronic infection). While taking ARCALYST, do not take other medicines that block interleukin-1, such as Kineret® (anakinra), or medicines that block tumor necrosis factor, such as Enbrel® (etanercept), Humira® (adalimumab), or Remicade® (infliximab), as this may increase your risk of getting a serious infection. Talk with your doctor about your vaccine history. Ask your doctor whether you should receive any vaccines before you begin treatment with ARCALYST. Medicines that affect the immune system may increase the risk of getting cancer. Stop taking ARCALYST and call your doctor or get emergency care right away if you have any symptoms of an allergic reaction. Your doctor will do blood tests to check for changes in your blood cholesterol and triglycerides. Common side effects include injection-site reactions (which may include pain, redness, swelling, itching, bruising, lumps, inflammation, skin rash, blisters, warmth, and bleeding at the injection site), upper respiratory tract infections, joint and muscle aches, rash, ear infection, sore throat, and runny nose. For more information about ARCALYST, talk to your doctor and see the Product Information. About KPL-387 KPL-387 is an independently developed, investigational, fully human immunoglobulin G2 (IgG2) monoclonal antibody that binds human interleukin-1 receptor 1 (IL-1R1), inhibiting the signaling of the cytokines IL-1α and IL-1β. Kiniksa believes KPL-387 could expand the treatment options for recurrent pericarditis patients by enabling dosing with a single monthly SC injection in a liquid formulation. About KPL-1161 KPL-1161 is an independently developed, investigational, Fc-modified IgG2 monoclonal antibody that binds IL-1R1, inhibiting the signaling of the cytokines IL-1α and IL-1β, with a target profile of quarterly SC dosing. Kiniksa is currently engaging in IND-enabling development activities for KPL-1161. Forward-Looking Statements This press release contains forward-looking statements. In some cases, you can identify forward looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these identifying words. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation, statements regarding: our expectation that ARCALYST 2025 net product revenue will be between $590 million and $605 million; our plan to initiate a Phase 2/3 clinical trial of KPL-387 in recurrent pericarditis in mid-2025, with Phase 2 data expected in the second half of 2026, and that we remain on track to meeting such plan; our expectation that our current operating plan will remain cash flow positive on an annual basis; our target profile of monthly dosing via a single subcutaneous injection in a liquid formulation for KPL-387; the expected impact of tariff policy on our gross margins; our target profile of quarterly dosing for KPL-1161; our beliefs about the mechanisms of our assets and potential impact of their approach; and our belief that our portfolio of assets offers the potential for differentiation. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including without limitation, the following: delays or difficulty in enrollment of patients in, and activation or continuation of sites for, our clinical trials; delays or difficulty in completing our clinical trials as originally designed; potential for changes between final data and any preliminary, interim, top-line or other data from clinical trials; our inability to replicate results from our earlier clinical trials or studies; impact of additional data from us or other companies, including the potential for our data to produce negative, inconclusive or commercially uncompetitive results; potential undesirable side effects caused by our products and product candidates; our inability to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities; potential for applicable regulatory authorities to not accept our filings, delay or deny approval of any of our product candidates or require additional data or trials to support approval; our reliance on third parties as the sole source of supply of the drug substance and drug product used in our products and product candidates; raw material, important ancillary product and drug substance and/or drug product shortages; our reliance on third parties to conduct research, clinical trials, and/or certain regulatory activities for our product candidates; complications in coordinating requirements, regulations and guidelines of regulatory authorities across jurisdictions for our clinical trials; business development activities and their impact on our financial performance and strategy; changes in our operating plan, business development strategy or funding requirements; existing or new competition; and the impact of global economic policy, including any uncertainty in national and international markets. These and other important factors discussed in our filings with the U.S. Securities and Exchange Commission, including under the caption “Risk Factors” contained therein, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. Except as required by law, we disclaim any intention or obligation to update or revise any forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release. ARCALYST® is a registered trademark of Regeneron Pharmaceuticals, Inc. Every Second Counts! ® Kiniksa Investor Contact Jonathan Kirshenbaum (781) 829-3949 jkirshenbaum@kiniksa.com Kiniksa Media Contact Tyler Gagnon (781) 431-9100 tgagnon@kiniksa.com

REDWOOD CITY, Calif., Feb. 27, 2025 (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 reported results for the fiscal quarter and year ended December 31, 2024 and provided a corporate update. “The past year has been a transformational period for Jasper, highlighted by positive data readouts from the BEACON study in CSU and the SPOTLIGHT study in CIndU, our first two clinical studies evaluating Briquilimab in mast cell diseases,” said Ronald Martell, President and Chief Executive Officer of Jasper. “Data from both studies demonstrate the ability of briquilimab to drive rapid and deep response profiles in patients with chronic urticaria, along with the potential for a favorable and differentiated safety profile. We believe the preliminary results from the BEACON study support advancing briquilimab into a pivotal program in CSU beginning with an operationally adaptive Phase 2b study that we expect to commence later this year. Final dose selection for the Phase 2b study will be further informed by a substantial array of additional clinical data at doses of 180mg and higher coming mid-year, including results from approximately 40 additional patients in the BEACON study and SPOTLIGHT study, as well as results from approximately 30 patients in the open-label extension study.” Highlights for Fourth Quarter 2024 and Recent Weeks Reported positive preliminary data from the ongoing BEACON Phase 1b/2a study of subcutaneous briquilimab in adult participants with CSU. Rapid onset of deep and durable clinical responses were observed across multiple dosing cohorts. Multiple dosing regimens ≥120mg demonstrating weekly urticaria activity score (UAS7) improvement of more than 25 points. Clinical responses observed as early as 1 week following first dose with complete responses achieved as early as week 2. Briquilimab has been well tolerated in the study, with adverse events (AEs) possibly related to c-Kit blockade being infrequent and generally limited to low grade events which resolved while still on study. No dose delays or discontinuations due to c-Kit related AEs were reported. Preliminary data support advancing briquilimab into a registrational program in CSU, beginning with a Phase 2b operationally adaptive study expected to commence in the second half of 2025. Obtained regulatory clearance to commence enrollment in the BEACON study cohorts evaluating 240mg Q8W and a 240mg loading dose followed by 180mg Q8W. Jasper expects to report data around mid-year 2025 from 4 cohorts: the 240mg single dose cohort (4 additional patients), 240mg Q8W cohort (8 patients), 240mg followed by 180mg Q8W cohort (8 patients), and the 360mg single dose cohort (8 patients). Multiple dosing regimens ≥120mg demonstrating weekly urticaria activity score (UAS7) improvement of more than 25 points. Clinical responses observed as early as 1 week following first dose with complete responses achieved as early as week 2. Briquilimab has been well tolerated in the study, with adverse events (AEs) possibly related to c-Kit blockade being infrequent and generally limited to low grade events which resolved while still on study. No dose delays or discontinuations due to c-Kit related AEs were reported. Preliminary data support advancing briquilimab into a registrational program in CSU, beginning with a Phase 2b operationally adaptive study expected to commence in the second half of 2025. Jasper expects to report data around mid-year 2025 from 4 cohorts: the 240mg single dose cohort (4 additional patients), 240mg Q8W cohort (8 patients), 240mg followed by 180mg Q8W cohort (8 patients), and the 360mg single dose cohort (8 patients). Reported positive preliminary data from the 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 included in the data-cut (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 has been well tolerated in the study, with no serious adverse events (SAEs) and no grade 3 or higher AEs reported. 14 of 15 participants (93%) enrolled in both dose cohorts included in the data-cut (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 has been well tolerated in the study, with no serious adverse events (SAEs) and no grade 3 or higher AEs reported. Commenced enrollment in the 180mg cohort of the SPOTLIGHT study. Jasper expects to report additional data from the study, including the 180mg cohort (n=12), in the first half of 2025. Commenced an open-label extension (OLE) study in chronic urticarias that will roll over patients from the BEACON and SPOTLIGHT studies upon completion of their initial follow up period. Patients enrolled in the OLE study will receive 180mg dose briquilimab on a Q8W schedule. Jasper expects to report initial data from approximately 30 patients enrolled in the OLE study around mid-year 2025. Jasper expects to report initial data from approximately 30 patients enrolled in the OLE study around mid-year 2025. Announced five poster presentations and an oral presentation of briquilimab clinical and preclinical data at the AAAAI 2025 Annual Meeting, being held February 28 - March 3, 2025, in San Diego, CA. The oral presentation and one of the poster presentations will include additional follow-up from patients included in Jasper’s January 8th, 2025, disclosure of preliminary data from the Phase 1b/2a BEACON trial. Announced dosing of first patient in the ETESIAN Phase 1b/2a clinical challenge study evaluating a single administration of subcutaneous briquilimab in patients with asthma. ETESIAN study is a single dose double-blind, placebo-controlled challenge study that is expected to enroll approximately 30 patients across as many as 7 sites in Canada with a key objective of demonstrating proof-of-concept in asthma utilizing a potential therapeutic dose to inform future trials in the broader asthma population. The study will be conducted utilizing a single 180mg dose of subcutaneous briquilimab and key assessments will include both early and late asthmatic response, changes in airway hyperresponsiveness, mast cell depletion and recovery, and safety. Jasper expects to report initial data in the second half of 2025. ETESIAN study is a single dose double-blind, placebo-controlled challenge study that is expected to enroll approximately 30 patients across as many as 7 sites in Canada with a key objective of demonstrating proof-of-concept in asthma utilizing a potential therapeutic dose to inform future trials in the broader asthma population. The study will be conducted utilizing a single 180mg dose of subcutaneous briquilimab and key assessments will include both early and late asthmatic response, changes in airway hyperresponsiveness, mast cell depletion and recovery, and safety. Jasper expects to report initial data in the second half of 2025. Fiscal 2024 Financial Results Cash and cash equivalents as of December 31, 2024, totaled $71.6 million. Research and development expenses for the three months and the year ended December 31, 2024, were $19.8 million and $55.8 million, respectively. General and administrative expenses for the three months and the year ended December 31, 2024, were $5.5 million and $20.4 million, respectively. Jasper reported a net loss of $24.3 million and $71.3 million, or basic and diluted net loss per share attributable to common stockholders of $1.62 and $4.89, for the three months and the year ended December 31, 2024, respectively. About Jasper Jasper is a clinical-stage biotechnology company focused on developing briquilimab as a therapeutic for chronic mast cell diseases. 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 and asthma. Jasper is currently conducting clinical studies of briquilimab as a treatment in patients with CSU, CIndU or asthma. Briquilimab has a demonstrated efficacy and safety profile in patients and healthy volunteers, with positive clinical outcomes in CSU and CIndU. For more information, please visit us at www.jaspertx.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 the ability to drive rapid and deep response profiles and its potential for a favorable and differentiated safety profile; Jasper’s expectations regarding advancing briquilimab into a pivotal program in CSU, including the timing for of an operationally adaptive Phase 2b study; Jasper’s expected timing for reporting data from its BEACON study, its 180mg cohort of the SPOTLIGHT study and its OLE study in chronic urticarias; and Jasper’s expectations related to its ETESIAN study, including the expected number of patients, expected number of sites and locations and expected timing for reporting initial data. 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 prior test, study and trial results may not be replicated in continuing or future studies and trials; 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, 2024 to be filed following this press release 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 ---tables to follow---

Enrollment ongoing in Phase 3 barzolvolimab CSU studies; Phase 3 program in CIndU under development Phase 2 barzolvolimab EOE study fully accrued; Phase 2 study initiated in atopic dermatitis; enrollment ongoing in Phase 2 PN study Celldex’s first bispecific for inflammatory diseases, CDX-622, Phase 1 study initiated Multiple important data read outs anticipated in 2025 HAMPTON, N.J. , Feb. 27, 2025 (GLOBE NEWSWIRE) -- Celldex Therapeutics, Inc. (NASDAQ:CLDX) today reported financial results for the fourth quarter and year ended December 31, 2024 and provided a corporate update. "In 2024, Celldex set a new bar for efficacy in chronic urticarias—presenting best-in-disease data across both our Phase 2 studies in CSU and CIndU,” said Anthony Marucci , Co-founder, President and Chief Executive Officer of Celldex Therapeutics . “Our Phase 3 studies in CSU have been met with great enthusiasm from the global medical community and are actively enrolling patients around the world. We look forward to building on this momentum later this year when we initiate our Phase 3 program in CIndU.” “We closed 2024 with the initiation of two new programs—advancing barzolvolimab into its fifth indication, atopic dermatitis, and expanding our leadership in mast cell science with the introduction of our first inflammatory bispecific candidate into the clinic, CDX-622, which targets stem cell factor and TSLP,” added Mr. Marucci . “We expect 2025 will be a year of continued execution across our robust pipeline supported by important data from our barzolvolimab Phase 2 studies in CSU, CIndU and EOE and our CDX-622 Phase 1 study in healthy volunteers. We’re excited to continue this work and share our progress over the course of the year.” 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 Phase 3 Development A global Phase 3 program in chronic spontaneous urticaria (CSU)(opens in a new tab) consisting of two Phase 3 trials (EMBARQ-CSU1 and EMBARQ-CSU2) was initiated in July and enrollment is ongoing. The studies are designed to establish the efficacy and safety of barzolvolimab in adult patients with CSU who remain symptomatic despite H1 antihistamine treatment and also include patients who remain symptomatic after treatment with biologics. EMBARQ-CSU1 and EMBARQ-CSU2 will enroll approximately 915 patients each across 40 countries and 500 sites. The Company is currently planning a global Phase 3 program in chronic inducible urticaria (CIndU), which is expected to initiate in 2025. Phase 2 Development Barzolvolimab met all primary and secondary endpoints at 12 weeks across the Company’s Phase 2 studies in CSU and CIndU. Results were highly statistically significant and clinically meaningful. 52 week data(opens in a new tab) from the Phase 2 study in CSU (n=208) were presented in a late breaking oral presentation at the EADV Congress 2024. A deepening of response was observed over the 52 week treatment period and barzolvolimab demonstrated the highest rate of complete response observed in a well controlled study in CSU with 71% of patients (150 mg Q4W) achieving a complete response at Week 52. Importantly, barzolvolimab was also well tolerated through 52 weeks. The Company anticipates presenting 76 week data (52 weeks of treatment; 24 weeks of follow up post treatment) from the study in 2025. 12 week primary endpoint data(opens in a new tab) from the Phase 2 study in CIndU (n=196) were presented in a late breaking oral presentation at the ACAAI Annual Scientific Meeting 2024. Barzolvolimab is the first drug to demonstrate clinical benefit in patients with chronic inducible urticaria in a large, randomized, placebo-controlled study. Up to 53.1% of patients with cold urticaria (ColdU) and 57.6% of patients with symptomatic dermographism (SD) treated with barzolvolimab experienced a complete response (per provocation test) compared to placebo rates of only 12.5% (p=0.0011) in ColdU and 3.2% (p<0.0001) in SD—the primary endpoint of the study. Secondary and exploratory endpoints were highly supportive of the primary endpoint. Patients on study continued to receive barzolvolimab or placebo for up 20 weeks and are being followed for an additional 24 weeks. The study also includes an Open Label Extension. The Company anticipates presenting data through week 44 in 2025 (20 weeks of treatment; 24 weeks of follow up post treatment; patients with returning symptoms can enter open label extension during follow up period). 52 week data(opens in a new tab) from the Phase 2 study in CSU (n=208) were presented in a late breaking oral presentation at the EADV Congress 2024. A deepening of response was observed over the 52 week treatment period and barzolvolimab demonstrated the highest rate of complete response observed in a well controlled study in CSU with 71% of patients (150 mg Q4W) achieving a complete response at Week 52. Importantly, barzolvolimab was also well tolerated through 52 weeks. The Company anticipates presenting 76 week data (52 weeks of treatment; 24 weeks of follow up post treatment) from the study in 2025. 12 week primary endpoint data(opens in a new tab) from the Phase 2 study in CIndU (n=196) were presented in a late breaking oral presentation at the ACAAI Annual Scientific Meeting 2024. Barzolvolimab is the first drug to demonstrate clinical benefit in patients with chronic inducible urticaria in a large, randomized, placebo-controlled study. Up to 53.1% of patients with cold urticaria (ColdU) and 57.6% of patients with symptomatic dermographism (SD) treated with barzolvolimab experienced a complete response (per provocation test) compared to placebo rates of only 12.5% (p=0.0011) in ColdU and 3.2% (p<0.0001) in SD—the primary endpoint of the study. Secondary and exploratory endpoints were highly supportive of the primary endpoint. Patients on study continued to receive barzolvolimab or placebo for up 20 weeks and are being followed for an additional 24 weeks. The study also includes an Open Label Extension. The Company anticipates presenting data through week 44 in 2025 (20 weeks of treatment; 24 weeks of follow up post treatment; patients with returning symptoms can enter open label extension during follow up period). Additional Indications A Phase 2 study in eosinophilic esophagitis (EoE) is fully accrued. This randomized, double-blind, placebo-controlled, parallel group study is evaluating the efficacy and safety profile of barzolvolimab in patients with active EoE. Data from this study is expected in 2025. Enrollment continues in the Phase 2 study in prurigo nodularis (PN). 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 atopic dermatitis (AD) was initiated in December 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 AD. 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. A Phase 1 study in healthy volunteers was initiated in November 2024 and enrollment is ongoing. This two-part randomized, double-blind, placebo-controlled, Phase 1 dose escalation study is designed to assess the safety, pharmacokinetics, and pharmacodynamics of single ascending doses (Part 1) and multiple ascending doses (Part 2) of CDX-622 in up to 56 healthy participants. The pharmacodynamic biomarkers from blood and skin will be highly informative on the ability of CDX-622 to engage and neutralize SCF and TSLP. Fourth Quarter and Twelve Months 2024 Financial Highlights and 2025 Guidance Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2024 were $725.3 million compared to $756.0 million as of September 30, 2024 . The decrease was primarily driven by fourth quarter cash used in operating activities of $32.5 million . At December 31, 2024 , Celldex had 66.4 million shares outstanding. Revenues: Total revenue was $1.2 million in the fourth quarter of 2024 and $7.0 million for the year ended December 31, 2024 , compared to $4.1 million and $6.9 million for the comparable periods in 2023. The decrease in revenue for the fourth quarter of 2024, as compared to the fourth quarter of 2023, was primarily due to a decrease in services performed under our manufacturing and research and development agreements with Rockefeller University . R&D Expenses: Research and development (R&D) expenses were $46.9 million in the fourth quarter of 2024 and $163.6 million for the year ended December 31, 2024 , compared to $30.4 million and $118.0 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 $10.3 million in the fourth quarter of 2024 and $38.5 million for the year ended December 31, 2024 , compared to $8.8 million and $30.9 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. Litigation Settlement Related Loss: During the fourth quarter of 2023, the Company announced positive topline results from its Phase 2 clinical trial of barzolvolimab in patients with moderate to severe CSU, satisfying the “Successful Completion” of a Phase 2 clinical trial of barzolvolimab milestone pursuant to the Company’s settlement agreement with SRS. During the fourth quarter of 2023, we paid the $12.5 million milestone in cash and recorded a litigation settlement related loss of $12.5 million . Net Loss: Net loss was $47.1 million , or ( $0.71 ) per share, for the fourth quarter of 2024, and $157.9 million , or ( $2.45 ) per share, for the year ended December 31, 2024 , compared to a net loss of $43.3 million , or ( $0.83 ) per share, for the fourth quarter of 2023, and $141.4 million , or ( $2.92 ) per share, for the year ended December 31, 2023 . Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at December 31, 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(opens in a new tab). 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 Contact Sarah Cavanaugh Senior Vice President, Corporate Affairs & Administration(508) 864-8337scavanaugh@celldex.com(opens in a new tab) Patrick Till Meru Advisors (484) 788-8560ptill@meruadvisors.com(opens in a new tab) Source: Celldex Therapeutics, Inc.