Icatibant Acetate

BOSTON--(BUSINESS WIRE)-- Cycle Pharmaceuticals Limited (Cycle) is pleased to announce the launch of tiopronin delayed-release tablets as a treatment option for patients with cystinuria.1 Tiopronin delayed-release tablets are available in 100 mg and 300 mg doses, and are approved by the US Food and Drug Administration (FDA).1 Generic tiopronin delayed-release tablets must be taken without food.1 Cystinuria is the most common inheritable cause of kidney stone disease. Incidence of the condition is estimated to be 1 in 7,000 worldwide.2 The launch of tiopronin delayed-release tablets is a partnership between Cycle and Torrent Pharma Inc. (Torrent) utilizing Anovo Specialty Pharmacy (Anovo) in which all three companies are committed to providing patients with rare diseases the medication and patient support they need. Tiopronin delayed-release tablets are a treatment option for patients with cystinuria who want to benefit from the support services provided by Cycle Vita™, a dedicated support platform which provides individualized product support* for patients, and ongoing dispensing and support services provided by Anovo, the exclusive specialty pharmacy for this product. “Cystinuria is a chronic lifelong condition in which stones commonly reoccur.3 In addition, close control of a patient’s diet, specifically a reduction in salt and methionine intake, can have an impact on the likelihood of stones forming.3 We are fully aware of the unique personal challenges these patients face in effectively managing their cystinuria. That’s why we’re providing individualized support including a Bridge Program* and Co-pay Assistance Program*. In addition, a Patient Nutrition Program* will be available to reinforce the clinic’s nutrition plan,” says Chikai Lai, SVP & Chief Commercial Officer, Cycle. Tiopronin delayed-release tablets are Cycle’s fifth commercial product to be launched in the US, building on the launch of: NITYR® (nitisinone) Tablets in 2017 SAJAZIR™ (icatibant) Injection in 2021 JAVYGTOR™ (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution in 2022; and TASCENSO ODT® (fingolimod) in 2023 Tiopronin delayed-release tablets are indicated in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 9 years of age and older with severe homozygous cystinuria, who are not responsive to these measures alone. Tiopronin delayed-release tablets must be taken without food.1 To find out more about Cycle Vita, please visit or call +1 (888) 360-8482 and find the Full Prescribing Information available at . Important Safety Information Important Safety Information is below. Full Prescribing Information is available at . INDICATIONS Tiopronin delayed-release tablets are a reducing and complexing thiol indicated, in combination with high fluid intake, alkali, and diet modification, for prevention of cystine stone formation in adult and pediatric patients 9 years of age and older with severe homozygous cystinuria who are not responsive to these measures alone. WARNINGS AND PRECAUTIONS Proteinuria, including nephrotic syndrome, and membranous nephropathy, have been reported with tiopronin use. Pediatric patients receiving greater than 50 mg/kg of tiopronin per day may be at increased risk for proteinuria. Hypersensitivity reactions (drug fever, rash, fever, arthralgia and lymphadenopathy) have been reported. MOST COMMON ADVERSE REACTIONS Most common adverse reactions (≥10%) are nausea, diarrhea or soft stools, oral ulcers, rash, fatigue, fever, arthralgia, proteinuria, and emesis. DRUG INTERACTIONS Alcohol: Tiopronin is released faster from tiopronin delayed-release tablets in the presence of alcohol and the risk for adverse events associated with tiopronin delayed-release tablets when taken with alcohol is unknown. Avoid alcohol consumption 2 hours before and 3 hours after taking tiopronin delayed-release tablets. USE IN SPECIFIC POPULATIONS: Pregnancy: Available published case report data with tiopronin have not identified a drug-associated risk for major birth defects, miscarriage, or adverse maternal or fetal outcomes. Renal stones in pregnancy may result in adverse pregnancy outcomes. Tell your healthcare provider immediately if you become pregnant or planning to become pregnant. Lactation: There are no data on the presence of tiopronin in either human or animal milk or on the effects of the breastfed child. A published study suggests that tiopronin may suppress milk production. Because of the potential for serious adverse reactions, including nephrotic syndrome, advise patients that breastfeeding is not recommended during treatment with tiopronin delayed-release tablets. Tell your healthcare provider immediately if you plan on breast feeding. Pediatric Use: Tiopronin delayed-release tablets are indicated in pediatric patients 9 years of age and older with severe homozygous cystinuria, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation who are not responsive to these measures alone. Tiopronin delayed-release tablets are not approved for use in pediatric patients weighing less than 20 kg or in pediatric patients unable to swallow tablets. Geriatric Use: This drug is known to be substantially excreted by the kidney, and the risk of adverse reactions to this drug may be greater in patients with impaired renal function. Because elderly patients are more likely to have decreased renal function, care should be taken in dose selection, and it may be useful to monitor renal function. For more detailed information, please refer to the full Prescribing Information at . If you get any side effects, talk to your healthcare provider. This includes possible side effects not listed in this material. You may also report side effects directly by calling Torrent Pharma Inc. at: 1-800-912-9561 or FDA at: 1-800-FDA-1088 or . This material is for education purposes only. It is not intended to be, and should not be used as, a substitute for medical advice. US-TIO-2400004 (February 2024) References Tiopronin Delayed-Release Tablets. Prescribing Information. Torrent Pharma Inc. Leslie, S.W., Sajjad, H. and Nazzal, L. (2023). Cystinuria. [online] Nih.gov. Available at: [Accessed Feb 9 2024]. Cystinuria UK - Diet - what should I eat (2023). Cystinuria UK - Diet - what should I eat? [online] Cystinuriauk.co.uk. Available at: [Accessed Feb 9 2024]. * Some areas of support may not be accessible to all patients. Eligibility criteria may apply to ensure compliance with all applicable federal and state requirements, and benefits may be limited to commercially insured patients only. For more detailed information about eligibility, terms and conditions, please contact the Cycle Vita team at +1 (888) 360-8482. The US Prescribing Information provides the indications for which the product is approved in the US. Please consult the US Prescribing Information and product labeling for information about the specific uses for which the product is indicated in the US. –ENDS– Cycle Vita™, SAJAZIR™ and JAVYGTOR™ are trademarks of Cycle Pharmaceuticals Limited in the United States. NITYR® is a registered trademark of Cycle Pharmaceuticals Limited in the United States. TASCENSO ODT® is a registered trademark of Handa Neuroscience, LLC. ©2024 Cycle Pharmaceuticals Limited. All rights reserved. About Cycle Pharmaceuticals Cycle Pharmaceuticals was founded in 2012 with the sole aim of delivering drug treatments and product support to the underserved rare disease patient community. Cycle focuses on rare metabolic, immunological, and neurological genetic conditions. Within neurological conditions, we focus on multiple sclerosis. Cycle is headquartered in Cambridge, UK and has offices in Boston, Massachusetts. For more information, please visit and follow us on X, LinkedIn and Facebook. About Torrent Pharma Inc. Torrent Pharma is one of the leading Pharma companies in India. Torrent is the pioneer in initiating the concept of niche marketing in India and today is ranked amongst the leaders in therapeutic segment of cardiovascular (CV), central nervous system (CNS), gastro-intestinal (GI) and women healthcare (WHC). Torrent also has significant presence in diabetology, pain management, gynecology, oncology and anti-infective segments. Torrent Pharma is present in more than 40 countries. It is ranked first amongst Indian Companies for having largest market share in Brazil and Germany. About Anovo Anovo was founded in 2012 as an exclusive provider of comprehensive end-to-end services for manufacturers of rare disease drugs and patients who need the drugs, including specialty wholesale/3PL distribution services, specialty pharmacy services, and patient support hub programs. Anovo benefits all parties involved by navigating all the complexities of managing rare disease medications and providing the related support needed by patients, caregivers and prescribers. View source version on businesswire.com: Contacts Marketing@cyclepharma.com Cycle Pharmaceuticals Tel: +44 1223 354 118 Source: Cycle Pharmaceuticals Limited View this news release online at:

CAMBRIDGE, Mass. & SALISBURY, England--( BUSINESS WIRE )--KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today announced that it presented real-world data from US patient surveys that assessed the experience of HAE patients using injectable on-demand treatments at the 2024 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting that took place in Washington, DC. The following presentations occurred at AAAAI 2024: Characteristics of Hereditary Angioedema Attacks Among Long-Term Prophylaxis Users: Bob Geng, Allergy and Immunology, University of California, San Diego, California, United States (Poster Presentation) Among HAE patients who had treated a recent attack, the location and duration of the most recent attacks were similar between long-term prophylaxis (LTP) and on-demand only users Earlier on-demand treatment was associated with a shorter attack duration, regardless of LTP use Delayed On-demand Treatment of Hereditary Angioedema Attacks: Patient Perceptions and Associated Barriers: Sandra Christiansen, University of California San Diego, La Jolla, CA, United States (Poster Presentation) Despite their perception of treating attacks “early”, many patients did not meet guideline recommendations for prompt on-demand treatment after recognition of an HAE attack The most common barriers to earlier treatment were uncertainty if attack was real, thinking the attack would be mild, and wanting to save treatment for a severe attack Anxiety Associated with On-Demand Treatment for Hereditary Angioedema (HAE) Attacks: James Wedner, Washington University School of Medicine, St Louis, MO, United States (Poster Presentation) Both adults and adolescents with HAE reported moderate to extreme anxiety when anticipating use of parenteral on-demand treatment, irrespective of use of on-demand only or on-demand plus LTP The results of this study highlighted the association between delayed treatment and treatment-related anxiety, with a higher proportion of patients who delayed treatment experiencing moderate to extreme anxiety Anxiety Associated with Refilling On-demand Therapy for HAE Attacks Contributes to Treatment Delay and Non-Treatment: Autumn Burnette, Division of Allergy and Immunology, Howard University Hospital, Washington, DC, United States (Poster Presentation) Anxiety associated with not being able to refill on-demand treatment quickly impacted treatment decisions, which contributed to treatment delay or resulted in non-treatment of HAE attacks One-third of patients using icatibant did not treat or delayed treatment due to anxiety about refills Characterizing the Negative Impact of Delayed On-Demand Treatment of HAE Attacks: Princess Ogbogu, Division of Pediatric Allergy, Immunology, and Rheumatology, University Hospitals Rainbow Babies and Children's Hospital, Cleveland, Ohio, United States (Poster Presentation) People living with HAE understood the importance of treating HAE attacks early and recognized that earlier treatment translates to quicker recovery and resolution Delayed on-demand treatment (> 1 hour) lengthened the time to “feeling in control” of an attack and the time to feeling fully recovered The Impact of On-demand Treatment on Quality of Life of People with HAE: Paula Busse, Department of Medicine, Division of Clinical Immunology, Mount Sinai, New York, United States (Poster Presentation) People with HAE needed to make adjustments to their daily lives including avoiding situations where their injectable on-demand treatment may be discovered by others Embarrassment associated with carrying on-demand treatment was among the reasons HAE patients delayed administration of on-demand treatment Characterizing the Perspective of Patients With HAE on Prophylactic Treatment: Stephen Betschel, Division of Allergy and Immunology, Department of Medicine, St. Michael’s Hospital, University of Toronto, Toronto, Ontario, Canada (Poster Presentation) Only 35% of LTP patients always carried on-demand treatment when away from home, while 43% of prophylaxis patients cited avoiding potential triggers as a reason for not carrying on-demand treatment at all times Nearly half of patients on LTP patients experienced moderate to extreme levels of anxiety when anticipating on-demand treatment administration Treatment Patterns of Patients Requiring Redosing of an On-demand Treatment After the Return of an HAE Attack: Constance Katelaris, Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney, NSW, Australia (Poster Presentation) Almost one third of people with HAE experienced the return of an HAE attack requiring ≥1 additional dose of on-demand treatment HAE attacks treated within 1 hour returned less frequently compared with attacks treated after ≥1 hour “The results of these surveys clearly conveyed the challenges faced by patients trying to manage their HAE attacks with injectable on-demand treatments. The resulting non-compliance with treatment guidelines may lead to poor clinical outcomes, even among patients receiving LTP,” said Andrew Crockett, Chief Executive Officer of KalVista. “We believe the efficacy and safety data from our phase 3 trial for sebetralstat show a potential path forward to address these persisting unmet needs.” Links to all posters and presentations can be found on the KalVista website under “Publications”. About KalVista Pharmaceuticals, Inc. KalVista Pharmaceuticals, Inc. is a pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors for diseases with significant unmet need. KalVista disclosed positive phase 3 data for the KONFIDENT trial for its oral, on-demand therapy sebetralstat in February 2024. The Company anticipates submitting a new drug application to the U.S. FDA for sebetralstat in the first half of 2024 and expects to file for approval in Europe and Japan later in 2024. In addition, KalVista’s oral Factor XIIa inhibitor program represents a new generation of therapies that may further improve the treatment for people living with HAE and other diseases. For more information about KalVista, please visit www.kalvista.com . Forward-Looking Statements This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, timing or outcomes of communications with the FDA, our expectations about safety and efficacy of our product candidates and timing of clinical trials and its results, our ability to commence clinical studies or complete ongoing clinical studies, including the KONFIDENT-S trial, and to obtain regulatory approvals for sebetralstat and other candidates in development, the success of any efforts to commercialize sebetralstat, the ability of sebetralstat and other candidates in development to treat HAE or other diseases, and the future progress and potential success of our oral Factor XIIa program. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2023, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.