Anti-IL-23 therapy(Band Therapeutics, LLC)

Submissions are supported by two Phase 3 clinical trials demonstrating risankizumab achieved the primary endpoint of clinical remission (per Adapted Mayo Score) and key secondary endpoints as an induction and maintenance treatment1,2 Safety results were generally consistent with the known safety profile of risankizumab, with no new safety risks observed1,2 Risankizumab is an IL-23 inhibitor being evaluated as a treatment for adults with moderately to severely active ulcerative colitis and is currently approved by FDA and EMA in Crohn's disease, psoriatic arthritis and psoriasis1 NORTH CHICAGO, Ill., Aug. 28, 2023 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that it has submitted applications for a new indication to the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for risankizumab (SKYRIZI®, 1200 mg intravenous [IV] [induction dose] and 180 mg and 360 mg subcutaneous [SC] [maintenance dose]) for the treatment of adult patients with moderately to severely active ulcerative colitis. "While there has been advancement in therapies to treat ulcerative colitis, there is still an ongoing need for additional treatments to help those seeking relief from its disruptive effects," said Roopal Thakkar, M.D., senior vice president, development, regulatory affairs and chief medical officer, AbbVie. "These submissions demonstrate our continued commitment to helping people living with IBD, and we look forward to providing a potential new treatment option for the management of ulcerative colitis." Applications to the FDA and EMA are supported by data from two Phase 3 clinical trials: an induction study, INSPIRE, and a maintenance study, COMMAND.1,2 Significantly more patients treated with risankizumab 1200 mg IV at week 12 in the induction study and 180 mg or 360 mg SC at week 52 in the maintenance study achieved the primary endpoint of clinical remission (per Adapted Mayo Score), compared to patients receiving placebo.1,2 Additionally, more risankizumab-treated patients in both the induction and maintenance studies achieved the key secondary endpoints of endoscopic improvement (endoscopic subscore ≤1 without evidence of friability) and histologic endoscopic mucosal improvement (HEMI, defined as endoscopic subscore ≤1 without evidence of friability and Geboes score ≤3.1) compared to placebo.1,2 The safety results in INSPIRE and COMMAND were generally consistent with the safety profile of risankizumab observed in previous studies across other indications, with no new safety risks observed.1,2 Risankizumab (SKYRIZI) is part of a collaboration between Boehringer Ingelheim and AbbVie, with AbbVie leading development and commercialization globally. About Ulcerative Colitis Ulcerative colitis is a chronic, idiopathic, immune-mediated inflammatory bowel disease (IBD) of the large intestine that causes continuous mucosal inflammation extending, to a variable extent, from the rectum to the more proximal colon.3,4 The hallmark signs and symptoms of ulcerative colitis include rectal bleeding, abdominal pain, bloody diarrhea, tenesmus (a sense of pressure), urgency and fecal incontinence.3,4 The disease course of ulcerative colitis varies between patients and can range from quiescent disease to chronic refractory disease, which in some cases can lead to surgery or life-threatening complications.5,6 The severity of symptoms and unpredictability of disease course can lead to substantial burden and often disability among those living with the disease.7 About INSPIRE Induction Study1 INSPIRE is a multicenter, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of risankizumab 1200 mg IV administered every four weeks as induction therapy in subjects with moderately to severely active ulcerative colitis. The primary endpoint is clinical remission (per adapted Mayo Score, defined as SFS ≤1 and not greater than baseline, RBS of 0 and endoscopic subscore ≤1 without friability) at week 12. Key secondary endpoints include clinical response (decrease from baseline in the Adapted Mayo score ≥2 points and ≥30% from baseline, plus a decrease in RBS ≥1 or an absolute RBS ≤1), endoscopic improvement (endoscopic subscore ≤1 without friability), and HEMI (endoscopic subscore of 0 or 1 without friability and Geboes score ≤3.1) at week 12. Topline results of the study were shared in March 2023. More information can be found on (NCT03398148). About COMMAND Maintenance Study2 The COMMAND study is a Phase 3, multicenter, randomized, double-blind, controlled, 52-week maintenance study designed to evaluate the efficacy and safety of risankizumab 180 mg or 360 mg SC in adults with moderately to severely active ulcerative colitis. This study had a re-randomized withdrawal design in which all patients received risankizumab IV induction and those who responded to risankizumab were re-randomized to receive risankizumab 180 mg or 360 mg SC or withdrawal from risankizumab treatment (induction-only control group). For those randomized to the withdrawal from risankizumab treatment (induction-only control group), the rest of the study duration was a risankizumab washout. The objective of the Phase 3 study is to evaluate the efficacy and safety of risankizumab 180 mg or 360 mg as maintenance therapy versus withdrawal from risankizumab treatment (control) in patients with moderately to severely active ulcerative colitis who responded to risankizumab IV induction in the INSPIRE study. The primary endpoint is clinical remission (per Adapted Mayo Score, defined as SFS ≤1 and not greater than baseline, RBS of 0 and endoscopic subscore ≤1 without evidence of friability) at week 52. Key secondary endpoints include endoscopic improvement (endoscopic subscore ≤1 without evidence of friability), HEMI (endoscopic subscore of ≤1 without evidence of friability and Geboes score ≤3.1), and steroid-free clinical remission (defined as clinical remission per Adapted Mayo Score at week 52 and corticosteroid free for ≥90 days prior to week 52) at week 52. Topline results from this study were shared in June 2023. More information can be found on (NCT03398135). About Risankizumab (SKYRIZI®) SKYRIZI is an interleukin-23 (IL-23) inhibitor that selectively blocks IL-23 by binding to its p19 subunit.8 IL-23, a cytokine involved in inflammatory processes, is thought to be linked to a number of chronic immune-mediated diseases.9 SKYRIZI is approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency for the treatment of plaque psoriasis, psoriatic arthritis and Crohn's disease. The use of risankizumab in ulcerative colitis is not approved and its safety and efficacy have not been established by regulatory authorities. Phase 3 trials of risankizumab in psoriasis, psoriatic arthritis, Crohn's disease and ulcerative colitis are ongoing.9-11 U.S. Indications and Important Safety Information about SKYRIZI® (risankizumab-rzaa)12 SKYRIZI is a prescription medicine used to treat adults with: moderate to severe plaque psoriasis who may benefit from taking injections or pills (systemic therapy) or treatment using ultraviolet or UV light (phototherapy). active psoriatic arthritis (PsA). moderate to severe Crohn's disease. IMPORTANT SAFETY INFORMATION What is the most important information I should know about SKYRIZI® (risankizumab-rzaa)? SKYRIZI is a prescription medicine that may cause serious side effects, including: Serious allergic reactions: Stop using SKYRIZI and get emergency medical help right away if you get any of the following symptoms of a serious allergic reaction: - fainting, dizziness, feeling lightheaded (low blood pressure) - swelling of your face, eyelids, lips, mouth, tongue, or throat - trouble breathing or throat tightness - chest tightness - skin rash, hives - itching Infections: SKYRIZI may lower the ability of your immune system to fight infections and may increase your risk of infections. Your healthcare provider should check you for infections and tuberculosis (TB) before starting treatment with SKYRIZI and may treat you for TB before you begin treatment with SKYRIZI if you have a history of TB or have active TB. Your healthcare provider should watch you closely for signs and symptoms of TB during and after treatment with SKYRIZI. Tell your healthcare provider right away if you have an infection or have symptoms of an infection, including: – fever, sweats, or chills – cough – shortness of breath – blood in your mucus (phlegm) – muscle aches – warm, red, or painful skin or sores on your body different from your psoriasis – weight loss – diarrhea or stomach pain – burning when you urinate or urinating more often than normal Do not use SKYRIZI if you are allergic to risankizumab-rzaa or any of the ingredients in SKYRIZI. See the Medication Guide or Consumer Brief Summary for a complete list of ingredients. Before using SKYRIZI, tell your healthcare provider about all of your medical conditions, including if you: have any of the conditions or symptoms listed in the section "What is the most important information I should know about SKYRIZI?" have an infection that does not go away or that keeps coming back. have TB or have been in close contact with someone with TB. have recently received or are scheduled to receive an immunization (vaccine). Medicines that interact with the immune system may increase your risk of getting an infection after receiving live vaccines. You should avoid receiving live vaccines right before, during, or right after treatment with SKYRIZI. Tell your healthcare provider that you are taking SKYRIZI before receiving a vaccine. are pregnant or plan to become pregnant. It is not known if SKYRIZI can harm your unborn baby. are breastfeeding or plan to breastfeed. It is not known if SKYRIZI passes into your breast milk. become pregnant while taking SKYRIZI. You are encouraged to enroll in the Pregnancy Registry, which is used to collect information about the health of you and your baby. Talk to your healthcare provider or call 1-877-302-2161 to enroll in this registry. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. What are the possible side effects of SKYRIZI? SKYRIZI may cause serious side effects. See "What is the most important information I should know about SKYRIZI?" Liver problems in Crohn's disease: A person with Crohn's disease who received SKYRIZI through a vein in the arm developed changes in liver blood tests with a rash that led to hospitalization. Your healthcare provider will do blood tests to check your liver before, during, and up to 12 weeks of treatment and may stop treatment with SKYRIZI if you develop liver problems. Tell your healthcare provider right away if you notice any of the following symptoms: unexplained rash, nausea, vomiting, stomach (abdominal) pain, tiredness (fatigue), loss of appetite, yellowing of the skin and eyes (jaundice), and dark urine. The most common side effects of SKYRIZI in people treated for Crohn's disease include: upper respiratory infections, headache, joint pain, stomach (abdominal) pain, injection site reactions, low red blood cells (anemia), fever, back pain, and urinary tract infection. The most common side effects of SKYRIZI in people treated for plaque psoriasis and psoriatic arthritis include: upper respiratory infections, headache, feeling tired, injection site reactions, and fungal skin infections. These are not all the possible side effects of SKYRIZI. Call your doctor for medical advice about side effects. Use SKYRIZI exactly as your healthcare provider tells you to use it. SKYRIZI is available in a 150 mg/mL prefilled syringe and pen, a 600 mg/10 mL vial for intravenous infusion, and a 180 mg/1.2 mL or 360 mg/2.4 mL single-dose prefilled cartridge with on-body injector. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. If you are having difficulty paying for your medicine, AbbVie may be able to help. Visit AbbVie.com/myAbbVieAssist to learn more. Please click here for Full Prescribing Information and Medication Guide for SKYRIZI. Globally, prescribing information varies; refer to the individual country product label for complete information. About AbbVie in Gastroenterology With a robust clinical trial program, AbbVie is committed to cutting-edge research to drive exciting developments in inflammatory bowel diseases (IBD), like ulcerative colitis and Crohn's disease. By innovating, learning and adapting, AbbVie aspires to eliminate the burden of IBD and make a positive long-term impact on the lives of people with IBD. For more information on AbbVie in gastroenterology, visit . About AbbVie AbbVie's mission is to discover and deliver innovative medicines and solutions that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas – immunology, oncology, neuroscience, and eye care – and products and services in our Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at . Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn. Forward-Looking Statements Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2022 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law. References: AbbVie. Data on File: ABVRRTI75696. AbbVie. Data on file: ABVRRTI76012. Gajendran M, Loganathan P, Jimenez G, et al. A comprehensive review and update on ulcerative colitis. Dis Mon. 2019;65(12):100851. doi:10.1016/j.disamonth.2019.02.004 The facts about inflammatory bowel diseases. Crohn's & Colitis Foundation of America. Accessed March 8, 2023. Ulcerative colitis. Mayo Clinic. Accessed March 3, 2023. Surgery for Crohn's Disease and Ulcerative Colitis. Crohn's & Colitis Foundation of America. Accessed August 21, 2023. Mehta F. Report: economic implications of inflammatory bowel disease and its management. Am J Manag Care. 2016;22(3 Suppl):s51-60. SKYRIZI [Summary of Product Characteristics]. AbbVie Ltd. Available at: . Accessed on August 21, 2023. Pipeline. AbbVie. Accessed March 3, 2023. A study comparing risankizumab to placebo in participants with active psoriatic arthritis including those who have a history of inadequate response or intolerance to biologic therapy(ies) (KEEPsAKE2). ClinicalTrials.gov. Updated February 28, 2023. Accessed March 3, 2023. A multicenter, randomized, double-blind, placebo controlled induction study to evaluate the efficacy and safety of risankizumab in participants with moderately to severely active ulcerative colitis. ClinicalTrials.gov. Updated March 10, 2023. Accessed March 12, 2023. SKYRIZI [package insert]. North Chicago, IL: AbbVie Inc.; 2022. SOURCE AbbVie

Positive topline results from the Phase 2b FRONTIER 1 study of JNJ-2113 (formerly PN-235), the first and only IL-23 receptor antagonist oral peptide drug candidate for the treatment of moderate-to-severe plaque psoriasis, presented at the World Congress of Dermatology. JNJ-2113 now advances toward a Phase 3 study in moderate-to-severe psoriasis and a Phase 2b study in ulcerative colitis. Positive data from the randomized withdrawal portion of the Phase 2 REVIVE study of rusfertide in polycythemia vera (PV) presented as a late breaker at the annual Congress of the European Hematology Association (EHA2023); the global Phase 3 VERIFY study continues, with sites recruiting globally. NEWARK, CA / ACCESSWIRE / August 3, 2023 / Protagonist Therapeutics (Nasdaq:PTGX) ("Protagonist" or "the Company") today reported financial results for the second quarter ended June 30, 2023, and provided a corporate update. "The second quarter of 2023 was transformative for Protagonist, marked by the disclosure of strongly positive data from FRONTIER 1, a Phase 2b study of JNJ-2113, at the World Congress of Dermatology, and the decision by our partner, Janssen, to move forward into a Phase 3 clinical program in psoriasis and a Phase 2b study in ulcerative colitis," said Dinesh V. Patel, Ph.D., President and CEO of Protagonist. "JNJ-2113 is a first-in-class and only oral IL-23 receptor antagonist peptide that has the potential to lead the field of oral therapy for psoriasis. Today, a significant number of people living with moderate-to-severe plaque psoriasis are eligible for, but are still not receiving, advanced therapies, and JNJ-2113 can potentially offer a highly convenient, effective and safe solution to a broad category of eligible individuals." "We have also made impressive strides forward in the clinical development of our fully owned asset, rusfertide, in polycythemia vera," Dr. Patel continued. "The Phase 2 REVIVE study results were presented to a large audience as a late breaker at EHA2023. The Phase 3 VERIFY clinical trial is continuing to enroll study participants at sites globally, with enrollment completion projected by the end of the first quarter of 2024. Our cash position remains strong, with estimated cash runway through end of 2025. This estimate does not include the potential $200 million in milestones associated with a successful Phase 3 study, NDA filing, and approval of JNJ-2113 in psoriasis." Q2 and Recent Corporate Highlights Positive Phase 2b FRONTIER 1 topline results were presented at the World Congress of Dermatology in Singapore in July 2023. All primary and secondary efficacy endpoints were achieved in the study, which evaluated five different dosing regimens of JNJ-2113 in adult patients with moderate-to-severe plaque psoriasis. JNJ-2113 is a novel oral IL-23R antagonist peptide which binds with high affinity to the IL-23 receptor. In the FRONTIER 1 study, a greater proportion of patients who received JNJ-2113 achieved Psoriasis Area and Severity Index (PASI) 75 (primary endpoint), as well as PASI 90 and PASI 100 (75, 90 and 100 percent improvement in skin lesions as measured by PASI, respectively), compared to placebo, at week 16. 78.6 percent, 59.5 percent and 40.5 percent of adult patients who received JNJ-2113 at 100 mg twice daily achieved PASI 75, 90 and 100, respectively, at week 16. Five treatment groups were evaluated in the study, ranging from 25 mg once daily to 100 mg twice daily. Treatment was generally well tolerated, and the proportions of patients with adverse events were comparable between patient groups. The proportion of participants experiencing one or more adverse events was 52.4 percent in the combined JNJ-2113 group and 51.2 percent in the placebo group. JNJ-2113 is advancing into Phase 3 development for moderate-to-severe plaque psoriasis and in a Phase 2b clinical trial for adults living with ulcerative colitis. Pre-clinical and Phase 1 data for JNJ-2113 were presented at the International Societies for Investigative Dermatology meeting in Tokyo, Japan in May 2023, showing selective systemic IL-23 pathway inhibition in pre-clinical rat models and systemic pharmacodynamic activity in healthy human volunteers in a Phase 1 trial. Positive topline results from the blinded, placebo-controlled, randomized withdrawal portion of the Phase 2 REVIVE study of rusfertide in polycythemia vera were presented in Frankfurt, Germany in June 2023 at EHA2023. The Company completed a $115 million equity financing in April 2023. Second Quarter 2023 Financial Results Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of June 30, 2023, were $313.4 million. License and Collaboration Revenue: License and collaboration revenue was zero for the three and six months ended June 30, 2023 as we completed our performance obligation associated with the Janssen License and Collaboration Agreement as of June 30, 2022. License and collaboration revenue for the three and six months ended June 30, 2022 was $0.9 million and $26.6 million, respectively. The six months to June 30, 2022 included a one-time $25.0 million milestone earned by the Company following the dosing of the third patient in the Janssen Phase 2b FRONTIER 1 clinical trial of JNJ-2113. Research and Development ("R&D") Expenses: R&D expenses were $33.2 million and $60.6 million for the three and six months ended June 30, 2023, respectively, as compared to $34.6 million and $70.9 million for the same periods in 2022. The decreases in R&D expenses from prior year periods was primarily due to decreases in PN-943 expenses and costs related to the completion of PN-232 Phase 1 trials, partially offset by an increase in rusfertide expenses related primarily to the Phase 3 VERIFY clinical trial. General and Administrative ("G&A") Expenses: G&A expenses were $9.2 million and $17.8 million for the three and six months ended June 30, 2023, respectively, as compared to $7.7 million and $18.2 million for the same periods in 2022. The increase in G&A expenses from the prior year quarter was primarily due to increases in payroll and stock-based compensation and general expenses. The decrease in G&A expenses from the prior year was primarily due to one-time costs incurred in the first quarter of 2022. Net Loss: Net loss was $38.5 million, or $0.68 per share, for the three months ended June 30, 2023 as compared to a net loss of $41.0 million, or $0.84 per share, for the three months ended June 30, 2022. Net loss was $72.2 million, or $1.34 per share, for the six months ended June 30, 2023 as compared to a net loss of $62.0 million, or $1.27 per share, for the six months ended June 30, 2022. PROTAGONIST THERAPEUTICS, INC. Condensed Consolidated Statements of Operations (Unaudited) (Amounts in thousands except share and per share data) Three Months Ended Six Months Ended June 30, June 30, 2023 2022 2023 2022 License and collaboration revenue $ - $ 859 $ - $ 26,581 Operating expenses: Research and development (1) 33,182 34,611 60,598 70,929 General and administrative (1) 9,172 7,691 17,777 18,206 Total operating expenses 42,354 42,302 78,375 89,135 Loss from operations (42,354) (41,443) (78,375) (62,554) Interest income 3,913 484 6,404 652 Other expense, net (19) (78) (214) (65) Net loss $ (38,460) $ (41,037) $ (72,185) $ (61,967) Net loss per share, basic and diluted $ (0.68) $ (0.84) $ (1.34) $ (1.27) Weighted-average shares used to compute net loss per share, basic and diluted 56,775,742 49,049,902 53,691,965 48,902,047 Amount includes non-cash stock-based compensation expense. PROTAGONIST THERAPEUTICS, INC. Stock-based Compensation (Unaudited) (In thousands) Three Months Ended Six Months Ended June 30, June 30, 2023 2022 2023 2022 Research and development $ 4,809 $ 4,106 $ 9,391 $ 7,432 General and administrative 3,534 2,699 6,536 5,308 Total stock-based compensation expense $ 8,343 $ 6,805 $ 15,927 $ 12,740 PROTAGONIST THERAPEUTICS, INC. (Unaudited) Selected Consolidated Balance Sheet Data (In thousands) June 30, December 31, 2023 2022 Cash, cash equivalents and marketable securities$ $ 313,401 $ 237,355 Working capital 290,543 211,898 Total assets 320,466 247,928 Accumulated deficit (608,940) (536,755) Total stockholders' equity 294,068 215,608 About Protagonist Protagonist Therapeutics is a biopharmaceutical company with peptide-based new chemical entities (NCEs) rusfertide and JNJ-2113 (formerly PN-235) in advanced stages of clinical development, both derived from the Company's proprietary technology platform. Protagonist scientists jointly discovered PN-235 (now known as JNJ-2113) as part of Protagonist's Interleukin-23 receptor (IL-23R) collaboration with Janssen and followed it through IND-enabling pre-clinical and Phase 1 studies, with Janssen assuming responsibility for further clinical development. Rusfertide, a mimetic of the natural hormone hepcidin, is the Company's lead drug candidate currently in a global Phase 3 development program. The Phase 2 REVIVE study is now complete, with an open-label extension underway. The global Phase 3 VERIFY study of rusfertide in polycythemia vera is ongoing. Protagonist retains all worldwide development and commercialization rights to rusfertide. More information on Protagonist, its pipeline drug candidates and clinical studies can be found on the Company's website at protagonist-inc.com. Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements regarding the potential benefits of JNJ-2113, our expectations regarding the clinical development of JNJ-2113, our potential receipt of milestone and royalty payments under our collaboration agreement with Janssen Biotech, Inc., our forecasted cash runway and our expectations regarding enrollment in the REVIVE Phase 3 trial. In some cases, you can identify these statements by forward-looking words such as "anticipate," "believe," "may," "will," "expect," or the negative or plural of these words or similar expressions. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, our ability to develop and commercialize our product candidates, our ability to earn milestone payments under our collaboration agreement with Janssen Biotech, Inc., our ability to use and expand our programs to build a pipeline of product candidates, our ability to obtain and maintain regulatory approval of our product candidates, our ability to operate in a competitive industry and compete successfully against competitors that have greater resources than we do, and our ability to obtain and adequately protect intellectual property rights for our product candidates. Additional information concerning these and other risk factors affecting our business can be found in our periodic filings with the Securities and Exchange Commission, including under the heading "Risk Factors" contained in our most recently filed periodic reports on Form 10-K and Form 10-Q filed with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this press release. Contact Jami Taylor Email: j.taylor@ptgx-inc.com SOURCE: Protagonist Therapeutics, Inc. View source version on accesswire.com: