Itolizumab

Itolizumab demonstrated clinical efficacy after 12 weeks of treatment, achieving a clinical remission rate of 23.3% compared to 20.0% for adalimumab and 10.0% for placebo Itolizumab achieved key secondary endpoint of endoscopic remission of 16.7% compared to 16.7% for adalimumab and 6.7% for placebo Itolizumab was generally well tolerated consistent with prior clinical experience LA JOLLA, CA, USA I February 06, 2025 I Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging a deep understanding of immunobiology to develop novel therapeutics to treat severe autoimmune and inflammatory disorders, and Biocon Limited (BSE code: 532523, NSE: BIOCON), an innovation-led global biopharmaceutical company, today announced positive topline results from the Phase 2 study evaluating itolizumab in the treatment of moderate to severe ulcerative colitis (UC). The double-blinded, placebo- and active-controlled Phase 2 clinical study evaluated the safety and efficacy of itolizumab in biologic-naïve patients with moderate to severe active UC. A total of 90 patients were randomized 1:1:1 to receive itolizumab (fixed dose of 140 mg), placebo, or adalimumab (a global standard of care biologic treatment used as an active control) every two weeks for an initial 12-week treatment period. The primary endpoint of the study was clinical remission as defined by Total Mayo Score, and secondary endpoints included the proportion of participants who achieved clinical response and endoscopic remission (evaluated by central endoscopy). The study was co-sponsored by Equillium and Biocon Limited and conducted at multiple clinical trial sites in India. The design and conduct of the trial were a collaborative effort, with input from the gastroenterology community and leading global clinical and scientific experts in the field of inflammatory bowel disease (IBD). “The CD6-ALCAM pathway is elevated in gastrointestinal inflammation and is associated with severity of disease in both ulcerative colitis and Crohn’s patients. As such, we are delighted with the strength of data across the primary and secondary endpoints of this Phase 2 study in moderate to severe ulcerative colitis patients,” said Dr. Stephen Connelly, chief scientific officer at Equillium. “Itolizumab was well tolerated and achieved a clinical remission rate of 23 percent despite an imbalance of more severe patients in the itolizumab arm compared to the other arms of the study. While these positive results add to itolizumab’s critical mass of safety and efficacy data across different patient populations, we are particularly encouraged by this data in the context of our Phase 3 EQUATOR study in acute graft-versus-host disease, where lower gastrointestinal pathogenesis is a key driver of mortality, with topline data expected this quarter.” “Itolizumab demonstrated proof of concept with a meaningful effect size – comparable to biologic standard of care adalimumab – in this Phase 2 study in subjects with moderate to severe ulcerative colitis,” said Dr. Brian Feagan, Professor of Medicine at the Schulich School of Medicine & Dentistry at the University of Western Ontario. “Itolizumab represents a novel selective immune modifying mechanism of action with great potential in a treatment paradigm needing differentiation and improved outcomes for patients.” Summary of Key Study Results Baseline demographics of the study included a median age of 39 years, relatively equal proportions of male and female subjects evenly distributed among study arms, and a mean weight of 58 kilograms. Baseline disease severity of the study was greater in the itolizumab arm, where 23% of patients were classified as severe (Total Mayo Score of 11) versus 0% in the placebo and adalimumab arms, and 66% had left-sided colitis versus 30% and 43% in the placebo and adalimumab arms, respectively. The primary endpoint of the study was clinical remission, defined as Total Mayo Score of ≤ 2 with no individual sub-score greater than 1 at Week 12. Secondary endpoints included the proportion of participants who achieved clinical response (per Total Mayo Score) and endoscopic remission (evaluated by central endoscopy) at Week 12 and Week 24. Additional data is expected to be presented at a future scientific conference during 2025. * One patient from the adalimumab arm had ‘endoscopic remission,’ but was recorded as ‘not in clinical remission’ at week 12 due to missing sub-score data About Itolizumab Itolizumab is a clinical-stage, first-in-class immune-modifying monoclonal antibody that selectively targets the CD6-ALCAM signaling pathway to downregulate pathogenic T effector cells while preserving T regulatory cells critical for maintaining a balanced immune response. This pathway plays a central role in modulating the activity and trafficking of T cells that drive a number of immuno-inflammatory diseases. The blockade of CD6 with itolizumab has demonstrated a reduction in T effector cell proliferation and downregulation of several important pathways that contribute to T effector cell development. The downregulation of these pathways is accompanied by decreased secretion of the pro-inflammatory T effector cytokines IFN-γ, TNF-α, IL-6 and IL-17. Additionally, inhibiting the binding of ALCAM to CD6 modulates lymphocyte trafficking and results in reduced T effector cell infiltration into inflamed tissues. About Biocon Limited Biocon Limited, publicly listed in 2004, (BSE code: 532523, NSE Id: BIOCON, ISIN Id: INE376G01013) is an innovation-led global biopharmaceuticals company committed to enhance affordable access to complex therapies for chronic conditions like diabetes, cancer and autoimmune. It has developed and commercialized novel biologics, biosimilars, and complex small molecule APIs in India and several key global markets as well as Generic Formulations in the US, Europe & key emerging markets. It also has a pipeline of promising novel assets in immunotherapy under development. Website: www.biocon.com ; Follow-us on Twitter: @bioconlimited for company updates. About Equillium Equillium is a clinical-stage biotechnology company leveraging a deep understanding of immunobiology to develop novel therapeutics to treat severe autoimmune and inflammatory disorders with high unmet medical need. The company’s pipeline consists of the following novel first-in-class immunomodulatory assets and product platform targeting immuno-inflammatory pathways. Itolizumab: a monoclonal antibody that targets the CD6-ALCAM signaling pathway which plays a central role in the modulation of effector T cells that drive a number of immuno-inflammatory diseases. It is currently under evaluation in a Phase 3 clinical study of patients with acute graft-versus-host disease (aGVHD) and has exhibited positive data from both a Phase 2 clinical study of patients with moderate to severe ulcerative colitis and a Phase 1b clinical study of patients with lupus/lupus nephritis. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited, who also provides commercial manufacturing for the product. EQ101: a selective tri-specific cytokine inhibitor targeting IL-2, IL-9, and IL-15, has exhibited positive results in both a Phase 2 proof-of-concept clinical study of patients with moderate to severe alopecia areata and a Phase 1/2 proof-of-concept clinical study of patients with cutaneous T cell lymphoma (CTCL). EQ302: an orally delivered, selective bi-specific cytokine inhibitor targeting IL-15 and IL-21 at pre-clinical stage. For more information, visit www.equilliumbio.com . SOURCE: Equillium

Equillium retains rights to itolizumab following substantial funding from Ono partnership Evaluating accelerating Phase 3 EQUATOR study in aGVHD to completion in Q1 2025 Phase 2 ulcerative colitis study recently completed; topline data expected Q1 2025 LA JOLLA, Calif.--(BUSINESS WIRE)-- Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging a deep understanding of immunobiology to develop novel therapeutics to treat severe autoimmune and inflammatory disorders, today announced financial results for the third quarter 2024 and provided corporate and clinical updates. “At the end of October we announced that our partnership with Ono Pharmaceutical had ended following substantial non-dilutive financing that has fully funded itolizumab research and development since July 2022. This funding enabled us to advance the program through two recent milestones – positive interim review from the Phase 3 EQUATOR study in acute graft-versus-host disease and positive topline data from the EQUALISE study in lupus nephritis. Ono informed us that their decision was strategic in nature, and that the data we have delivered from these studies met expectations with no observed or reported safety concerns,” said Bruce Steel, chief executive officer at Equillium. “We have enrolled over 150 patients in EQUATOR, and as of the end of October have temporarily paused enrollment to review clinical options for the program, including the potential to accelerate our timeline to topline data to the first quarter of 2025 while preserving registrational integrity of the study. We maintain orphan drug and fast track designations for first-line acute graft-versus-host disease, where currently no drugs are approved. In addition, in collaboration with Equillium, our partner Biocon has recently completed a robust placebo-controlled Phase 2 study of itolizumab in biologic-naïve patients with moderate to severe ulcerative colitis, and we expect topline data from this study also in the first quarter of 2025. We remain enthusiastic about the therapeutic potential and commercial opportunity for itolizumab, and we are very pleased to maintain our rights to the program as we approach the two most important data events in Equillium’s history.” Highlights Since the Beginning of the Third Quarter of 2024: Retained rights to itolizumab following Ono partnership Positive interim analysis of Phase 3 EQUATOR study of itolizumab in acute graft-versus-host disease subjects Phase 2 ulcerative colitis study completed by partner Biocon Anticipated Upcoming Milestones: Itolizumab: Topline data from Phase 2 study in ulcerative colitis – Q1 2025 Itolizumab: Topline data from Phase 3 EQUATOR study in aGVHD – potential acceleration to Q1 2025 Third Quarter 2024 Financial Results Revenue for the third quarter of 2024 was $12.2 million, compared to $8.9 million during the same period in 2023. Revenue in the third quarters of 2024 and 2023 consisted entirely of itolizumab development funding and amortization of the upfront payment related to our former partnership with Ono. Research and development (R&D) expenses for the third quarter of 2024 were $9.6 million, compared to $9.0 million for the same period in 2023. The increase was primarily due to greater expenses associated with chemistry, manufacturing and controls (CMC) activities related to itolizumab, greater non-clinical research expenses, and an increase in employee compensation expenses, which were partially offset by lower clinical study expenses primarily driven by the EQUALISE and EQ101 clinical studies, partially offset by greater expenses for our EQUATOR clinical study. General and administrative (G&A) expenses for the third quarter of 2024 were $3.3 million, compared with $3.5 million for the same period in 2023. The decrease was primarily driven by lower legal, audit and tax professional fees. Net loss for the third quarter of 2024 was approximately $7,000, or $(0.00) per basic and diluted share, compared with a net loss of $3.7 million, or $(0.11) per basic and diluted share, for the same period in 2023. The decrease in net loss was primarily attributable to greater revenue related to the Ono partnership, lower income tax expense, and other income, which were partially offset by the increase in R&D expense. Cash, cash equivalents and short-term investments totaled $25.9 million as of September 30, 2024, compared to $33.3 million as of June 30, 2024. Equillium believes that its cash, cash equivalents and short-term investments are sufficient to fund operations into the fourth quarter of 2025, assuming certain operational changes including accelerating the completion of the Phase 3 EQUATOR study based on reduced enrollment and early unblinding of the study, pausing further development activities related to EQ101 and EQ302, the elimination of certain positions and the reduction of certain discretionary expenditures, as well as assuming no further repurchases under our stock repurchase program. About Equillium Equillium is a clinical-stage biotechnology company leveraging a deep understanding of immunobiology to develop novel therapeutics to treat severe autoimmune and inflammatory disorders with high unmet medical need. The company’s pipeline consists of the following novel first-in-class immunomodulatory assets and product platform targeting immuno-inflammatory pathways. Itolizumab: a monoclonal antibody that targets the CD6-ALCAM signaling pathway which plays a central role in the modulation of effector T cells that drive a number of immuno-inflammatory diseases; currently under evaluation in a Phase 3 clinical study of patients with acute graft-versus-host disease (aGVHD) and announced positive data from a Phase 1b clinical study of patients with lupus/lupus nephritis in April 2024. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited, who also provides commercial manufacturing for the product. EQ101: a selective tri-specific cytokine inhibitor targeting IL-2, IL-9, and IL-15; recently announced positive results from a Phase 2 proof-of-concept clinical study of patients with alopecia areata. EQ302: an orally delivered, selective bi-specific cytokine inhibitor targeting IL-15 and IL-21 at pre-clinical stage. For more information, visit www.equilliumbio.com. Forward Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking Statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words such as “anticipate”, “believe”, “could”, “continue”, “expect”, “estimate”, “may”, “plan”, “outlook”, “future”, “potential” and “project” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These statements include, but are not limited to, statements regarding Equillium’s plans and strategies with respect to developing itolizumab, including repositioning itolizumab as the top priority in the pipeline, anticipated upcoming milestones, timelines for topline data for itolizumab, the temporary pausing and potential acceleration of the EQUATOR study, the pausing of further activities related to EQ101 and EQ302, Equillium’s cash runway and related assumptions, and the potential benefits of Equillium’s product candidates. Because such statements are subject to risks and uncertainties, many of which are outside of Equillium’s control, actual results may differ materially from those expressed or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: Equillium’s ability to execute its plans and strategies; risks related to performing clinical and pre-clinical studies; whether the results from clinical and pre-clinical studies will validate and support the safety and efficacy of Equillium’s product candidates; changes in the competitive landscape; changes in Equillium’s strategic plans; uncertainties related to Equillium’s capital requirements and ability to obtain sufficient financing to fund Equillium’s strategic plans; the potential impact of the registrational integrity that would be caused by Equillium’s decision to accelerate the completion of EQUATOR; and having to use cash in ways or on timing other than expected and the impact of market volatility on cash reserves. These and other risks and uncertainties are described more fully under the caption "Risk Factors" and elsewhere in Equillium's filings and reports, which may be accessed for free by visiting the Securities and Exchange Commission’s website and on Equillium’s website under the heading “Investors.” Investors should take such risks into account and should not rely on forward-looking statements when making investment decisions. All forward-looking statements contained in this press release speak only as of the date on which they were made. Equillium undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Equillium, Inc. Condensed Consolidated Balance Sheets (In thousands) (unaudited) September 30, December 31, 2024 2023 Assets Cash, cash equivalents and short-term investments $ 25,877 $ 40,866 Accounts receivable 5,009 3,735 Prepaid expenses and other assets 3,098 5,133 Operating lease right-of-use assets 473 796 Total assets $ 34,457 $ 50,530 Current liabilities Accounts payable and other current liabilities $ 8,989 $ 11,844 Current portion of deferred revenue 2,068 15,729 Total current liabilities 11,057 27,573 Long-term operating lease liabilities 223 384 Total liabilities 11,280 27,957 Total stockholders' equity 23,177 22,573 Total liabilities and stockholders' equity $ 34,457 $ 50,530 Equillium, Inc. Condensed Consolidated Statements of Operations (In thousands, except share and per share data) (unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Revenue $ 12,161 $ 8,870 $ 36,703 $ 26,873 Operating expenses: Research and development 9,562 8,974 30,113 27,855 General and administrative 3,278 3,519 10,161 10,340 Total operating expenses 12,840 12,493 40,274 38,195 Loss from operations (679 ) (3,623 ) (3,571 ) (11,322 ) Total other income, net 672 409 1,298 893 Loss before income taxes (7 ) (3,214 ) (2,273 ) (10,429 ) Income tax expense - 496 - 564 Net loss $ (7 ) $ (3,710 ) $ (2,273 ) $ (10,993 ) Net loss per share, basic and diluted $ (0.00 ) $ (0.11 ) $ (0.06 ) $ (0.32 ) Weighted-average number of common shares outstanding, basic and diluted 35,424,388 34,878,700 35,324,092 34,582,574 Investor Contact Michael Moore Vice President, Investor Relations & Corporate Communications 619-302-4431 ir@equilliumbio.com