CNP

The prevalence of idiopathic pulmonary fibrosis has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of idiopathic pulmonary fibrosis and the growing research and development activities to develop novel therapies to treat idiopathic pulmonary fibrosis to drive the market. The companies developing the potential therapies in the last stage of development include FibroGen, Boehringer Ingelheim, and several others. LAS VEGAS, May 25, 2023 /PRNewswire/ -- DelveInsight's ' Idiopathic Pulmonary Fibrosis Pipeline Insight – 2023 ' report provides comprehensive global coverage of pipeline idiopathic pulmonary fibrosis therapies in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space, and future growth potential of the idiopathic pulmonary fibrosis pipeline domain. Key Takeaways from the Idiopathic Pulmonary Fibrosis Pipeline Report DelveInsight's idiopathic pulmonary fibrosis pipeline report depicts a robust space with 80+ active players working to develop 100+ pipeline therapies for idiopathic pulmonary fibrosis treatment. Key idiopathic pulmonary fibrosis companies such as FibroGen, United Therapeutics, Bellerophon Therapeutics, MediciNova, Novartis, Endeavor BioMedicines, Pliant Therapeutics, Nitto Denko, Kadmon Pharmaceuticals, Calliditas Therapeutics, Avalyn Pharmaceuticals, PureTech Health, Taiho Pharmaceutical, Bristol-Myers Squibb, Galecto Biotech AB, CSL Behring, Celgene Pharmaceutical, Vicore Pharma, Boehringer Ingelheim, Guangdong Raynovent, Sunshine Lake Pharma co, Suzhou Zelgen Biopharmaceuticals, Algernon Pharmaceuticals, Horizon Therapeutics, Daewoong Pharmaceutical, Metagone Biotech, AstraZeneca, Lung Therapeutics, Bridge Biotherapeutics, AstraZeneca, Kinarus AG, Insmed, Reviva Pharmaceuticals, Annapurna Bio, Guangdong Hengrui Pharmaceutical Co., Ltd, Ark Biosciences, Ocean Biomedical, and others are evaluating new idiopathic pulmonary fibrosis drugs to improve the treatment landscape. Promising idiopathic pulmonary fibrosis pipeline therapies in various stages of development include Pamrevlumab, Treprostinil, Nitric oxide inhalation - INOpulse, MN-001 (tipelukast), VAY736, ENV-101, PLN-74809, ND-L02-s0201, KD025, GKT137831, AP 01, LYT-100, TAS-115, BMS-986278, GB0139, CSL312, CC-90001, C21, BI1015550, ZSP1603, HEC585, Jaktinib Dihydrochloride Monohydrate, Ifenprodil, HZN-825, DWN12088, MG-S-2525, Saracatinib, LTI-03, BBT-877, AZD5055, KIN001-IPF, Treprostinil palmitil inhalation powder (TPIP), Brilaroxazine, ANPA 0073, SHR 1906, AK 3280, OCF 203, and others. In May 2023, Kinarus Therapeutics announced the signing of a strategic convertible loan agreement for a CHF 1.5 million investment by ChaoDian (Hangzhou) Investment Management Co., Ltd., an investment company based in Hangzhou City, China ("CDIM"). Further, this agreement forms the basis for discussions on the introduction, development and commercialization of KIN001 for the treatment of Idiopathic Pulmonary Fibrosis (IPF) in China. Great Health Companion Group Ltd (GHCG), a subsidiary of Hakim Unique Group, introduced CDIM to Kinarus. In April 2023, AGC Biologics announced it signed a service agreement with The Jikei University in Japan. Under the agreement, AGC Biologics will assume a technology transfer and feasibility study for a drug product focused on the treatment of Idiopathic pulmonary fibrosis at the CDMO's center of Cell and Gene Excellence in Milan. In February 2023, Insilico Medicine announced that the US Food and Drug Administration (FDA) had granted Orphan Drug Designation to INS018_055 for the treatment of Idiopathic Pulmonary Fibrosis (IPF). In February 2023, Arrowhead Pharmaceuticals Inc. announced that it had dosed the first subjects in a Phase I/IIa clinical trial of ARO-MMP7, the company's investigational RNA interference (RNAi) therapeutic designed to reduce the expression of matrix metalloproteinase 7 (MMP7) as a potential treatment for idiopathic pulmonary fibrosis (IPF). In February 2023, South Korea's Daewoong Pharmaceutical secured an exclusive licensing agreement with CS Pharmaceuticals for a first-in-class PRS inhibitor Bersiporocin in the Greater China region, including mainland China, Hong Kong, Taiwan, and Macau. Under this agreement, CSP will in-license Bersiporocin for Idiopathic Pulmonary Fibrosis (IPF) and potentially other fibrotic indications for a total consideration of up to $336 million, including up to $76 million in upfront and development milestone payments and double-digit royalties on Net Sales. In January 2023, Insilico Medicine announced the positive topline results of safety, tolerability, and pharmacokinetics (PK) from the Phase 1 clinical trial of INS018_055, a potential first-in-class drug discovered by Insilico's end-to-end AI platform for idiopathic pulmonary fibrosis (IPF). In January 2023, Pliant Therapeutics announced 12-week interim data from the 320 mg dose group of INTEGRIS-IPF, a multinational, randomized, double-blind, placebo-controlled Phase 2a clinical trial of bexotegrast (PLN-74809) in patients with idiopathic pulmonary fibrosis (IPF). In December 2022, Vallon Pharmaceuticals announced that they entered into a definitive agreement (the "Merger Agreement") pursuant to which GRI Bio will merge with a wholly-owned subsidiary of Vallon in an all-stock transaction (the "Merger"). The combined company will focus on advancing GRI Bio's innovative pipeline of NKT cell regulators for the treatment of inflammatory, fibrotic and autoimmune diseases. Following the closing of the Merger, the combined company is expected to operate under the name "GRI Bio, Inc." Request a sample and discover the recent advances in idiopathic pulmonary fibrosis drug treatment @ Idiopathic Pulmonary Fibrosis Pipeline Report The idiopathic pulmonary fibrosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage idiopathic pulmonary fibrosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the idiopathic pulmonary fibrosis clinical trial landscape. Idiopathic Pulmonary Fibrosis Overview Idiopathic pulmonary fibrosis (IPF) is a rare, chronic, progressive fibrosing interstitial pneumonia that primarily affects middle-aged and older adults. It affects lung tissue by thickening, stiffening, or persistent and progressive scarring that worsens irreversibly over time. Scarring affects the air sacs of IPF patients, decreasing the amount of oxygen that enters the circulation. With less oxygen in the blood, ordinary tasks such as walking can cause dyspnea. This set of lung illnesses is also known as 'Diffuse Parenchymal Lung Diseases,' and it is distinguished by a broader umbrella of 'Interstitial Lung Diseases (IDLs)'. The cause of IPF is uncertain; experts believe the condition is caused by a mix of hereditary and environmental factors. There is a significant chance that genetic variations enhance a person's risk of acquiring IPF and then being exposed to environmental variables that aggravate the condition. However, much remains unknown about this new field of study. Find out more about drugs for idiopathic pulmonary fibrosis @ New Idiopathic Pulmonary Fibrosis Drugs A snapshot of the Idiopathic Pulmonary Fibrosis Pipeline Drugs mentioned in the report: Learn more about the emerging idiopathic pulmonary fibrosis pipeline therapies @ Idiopathic Pulmonary Fibrosis Clinical Trials Idiopathic Pulmonary Fibrosis Therapeutics Assessment The idiopathic pulmonary fibrosis pipeline report proffers an integral view of idiopathic pulmonary fibrosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Idiopathic Pulmonary Fibrosis Pipeline Report Coverage: Global Idiopathic Pulmonary Fibrosis Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Idiopathic Pulmonary Fibrosis Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Idiopathic Pulmonary Fibrosis Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravitreal, Subretinal, Topical Idiopathic Pulmonary Fibrosis Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy Idiopathic Pulmonary Fibrosis Therapeutics Assessment By Mechanism of Action: Connective tissue growth factor inhibitors, 5-lipoxygenase inhibitors, Leukotriene D4 receptor antagonists, Leukotriene receptor antagonists, Phospholipase C inhibitors, Thromboxane A2 receptor antagonists, Type 3 cyclic nucleotide phosphodiesterase inhibitors, Type 4 cyclic nucleotide phosphodiesterase inhibitors, Integrin alphavbeta1 inhibitors, Integrin alphaVbeta6 inhibitors, Antibody-dependent cell cytotoxicity, Interleukin 4 receptor antagonists, SMO protein inhibitors, Galectin 3 inhibitors Key Idiopathic Pulmonary Fibrosis Companies: FibroGen, United Therapeutics, Bellerophon Therapeutics, MediciNova, Novartis, Endeavor BioMedicines, Pliant Therapeutics, Nitto Denko, Kadmon Pharmaceuticals, Calliditas Therapeutics, Avalyn Pharmaceuticals, PureTech Health, Taiho Pharmaceutical, Bristol-Myers Squibb, Galecto Biotech AB, CSL Behring, Celgene Pharmaceutical, Vicore Pharma, Boehringer Ingelheim, Guangdong Raynovent, Sunshine Lake Pharma co, Suzhou Zelgen Biopharmaceuticals, Algernon Pharmaceuticals, Horizon Therapeutics, Daewoong Pharmaceutical, Metagone Biotech, AstraZeneca, Lung Therapeutics, Bridge Biotherapeutics, AstraZeneca, Kinarus AG, Insmed, Reviva Pharmaceuticals, Annapurna Bio, Guangdong Hengrui Pharmaceutical Co., Ltd, Ark Biosciences, Ocean Biomedical, and others. Key Idiopathic Pulmonary Fibrosis Pipeline Therapies: Pamrevlumab, Treprostinil, Nitric oxide inhalation - INOpulse, MN-001 (tipelukast), VAY736, ENV-101, PLN-74809, ND-L02-s0201, KD025, GKT137831, AP 01, LYT-100, TAS-115, BMS-986278, GB0139, CSL312, CC-90001, C21, BI1015550, ZSP1603, HEC585, Jaktinib Dihydrochloride Monohydrate, Ifenprodil, HZN-825, DWN12088, MG-S-2525, Saracatinib, LTI-03, BBT-877, AZD5055, KIN001-IPF, Treprostinil palmitil inhalation powder (TPIP), Brilaroxazine, ANPA 0073, SHR 1906, AK 3280, OCF 203, and others. Dive deep into rich insights for new drugs for idiopathic pulmonary fibrosis treatment; visit @ Idiopathic Pulmonary Fibrosis Medications Table of Contents For further information on the idiopathic pulmonary fibrosis pipeline therapeutics, reach out @ Idiopathic Pulmonary Fibrosis Drug Treatment Related Reports Idiopathic Pulmonary Fibrosis Market Idiopathic Pulmonary Fibrosis Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key idiopathic pulmonary fibrosis companies, including FibroGen, Hoffmann-La Roche Ltd, United Therapeutics, Boehringer Ingelheim, Pliant Therapeutics, Inc., Galecto Biotech, Horizon Therapeutics, CSL Behring, Kadmon Corporation, LLC, among others. Idiopathic Pulmonary Fibrosis Epidemiology Forecast Idiopathic Pulmonary Fibrosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and idiopathic pulmonary fibrosis epidemiology trends. Pulmonary Fibrosis Pipeline Pulmonary Fibrosis Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key pulmonary fibrosis companies, including Roche, Boehringer Ingelheim, Galecto Biotech, Cipla, Gilead Sciences, Fibrogen, Bristol-Myers Squibb, among others. Pulmonary Fibrosis Market Pulmonary Fibrosis Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key pulmonary fibrosis companies, including Roche, Boehringer Ingelheim, Galecto Biotech, Cipla, Gilead Sciences, Fibrogen, Bristol-Myers Squibb, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

-   Data demonstrated that once-weekly TransCon CNP has the potential to meet patient and caregiver needs for a safe, effective, tolerable and convenient treatment -   The primary endpoint, annualized height velocity (AHV) at Week 52, demonstrated superiority of TransCon CNP at 100 µg/kg/week compared to placebo (p=0.0218) -   TransCon CNP was generally well tolerated with low frequency of injection site reactions; all 57 randomized children continued, with the longest treatment duration beyond two years -   Data showed robust and consistent results in prespecified analyses across age groups and dose levels, supporting continued development at the selected dose of 100 µg/kg/week COPENHAGEN, Denmark I November 14, 2022 I Ascendis Pharma A/S (Nasdaq: ASND) today announced positive topline results from the ACcomplisH Trial, its Phase 2 randomized, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in children with achondroplasia aged 2 to 10 years old. TransCon CNP is an investigational long-acting prodrug of C-type natriuretic peptide (CNP), designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose. The ACcomplisH Trial evaluated 57 children with achondroplasia aged 2 to 10 years old, randomized in a 3:1 ratio to receive either sequential ascending doses of once-weekly TransCon CNP or placebo for 52 weeks. All 57 randomized children completed the blinded portion of ACcomplisH and are currently continuing in the open label extension (OLE) at the 100 µg/kg/week dose. The trial met its primary objectives, demonstrating that TransCon CNP at 100 µg/kg/week was superior to placebo on the primary efficacy endpoint of AHV at 52 weeks. A slide presentation with these data can be found on the Investor Relations & News section of the Ascendis Pharma website: https://investors.ascendispharma.com . Key data include: Additional highlights: “Development of a drug with potential to be both an accepted and a preferred treatment option for children with achondroplasia and their caregivers has a high bar related to safety, efficacy, tolerability and convenience,” said Aimee Shu, M.D., Vice President, Clinical Development, Endocrine & Rare Diseases at Ascendis Pharma. “We are excited to see once-weekly TransCon CNP demonstrated statistically superior growth compared to placebo at the 100 µg/kg/week dose level, with safety and tolerability results supporting further development in children with achondroplasia.” About Ascendis Pharma A/S Ascendis Pharma is applying its innovative TransCon platform to build a leading, fully integrated, global biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Heidelberg and Berlin, Germany; Palo Alto and Redwood City, California; and Princeton, New Jersey. Please visit www.ascendispharma.com to learn more. SOURCE: Ascendis Pharma

- Data demonstrated that once-weekly TransCon CNP has the potential to meet patient and caregiver needs for a safe, effective, tolerable and convenient treatment - The primary endpoint, annualized height velocity (AHV) at Week 52, demonstrated superiority of TransCon CNP at 100 µg/kg/week compared to placebo (p=0.0218) - TransCon CNP was generally well tolerated with low frequency of injection site reactions; all 57 randomized children continued, with the longest treatment duration beyond two years - Data showed robust and consistent results in prespecified analyses across age groups and dose levels, supporting continued development at the selected dose of 100 µg/kg/week - Ascendis to host conference call and webcast Monday, November 14 at 8:00 a.m. Eastern time COPENHAGEN, Denmark, Nov. 14, 2022 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced positive topline results from the ACcomplisH Trial, its Phase 2 randomized, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in children with achondroplasia aged 2 to 10 years old. TransCon CNP is an investigational long-acting prodrug of C-type natriuretic peptide (CNP), designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose. The ACcomplisH Trial evaluated 57 children with achondroplasia aged 2 to 10 years old, randomized in a 3:1 ratio to receive either sequential ascending doses of once-weekly TransCon CNP or placebo for 52 weeks. All 57 randomized children completed the blinded portion of ACcomplisH and are currently continuing in the open label extension (OLE) at the 100 µg/kg/week dose. The trial met its primary objectives, demonstrating that TransCon CNP at 100 µg/kg/week was superior to placebo on the primary efficacy endpoint of AHV at 52 weeks. A slide presentation with these data can be found on the Investor Relations & News section of the Ascendis Pharma website: https://investors.ascendispharma.com. Key data include: TransCon CNP Dose Group (n)AHV (cm/year)LS Mean [95% CI]p-value(TransCon CNP vs. Pooled Placebo)6 µg/kg/week (n=10)4.09[3.34, 4.84]0.600420 µg/kg/week (n=11)4.52,[3.82, 5.22]0.702250 µg/kg/week (n=10)5.16[4.43, 5.90]0.0849100 µg/kg/week (n=11)5.42[4.74, 6.11]0.0218Pooled Placebo (n=15)4.35[3.75, 4.94]NA Additional highlights: TransCon CNP demonstrated a consistent dose-response in AHV across the four dose groups.Mean improvements in AHV for TransCon CNP-treated patients were consistent across age groups <5 years and >5 years, with dose response established.TransCon CNP at 100 µg/kg/week demonstrated superiority in change in ACH-specific height SDS compared to placebo.TransCon CNP was generally safe and well tolerated, with no discontinuations.No serious AEs (SAEs) related to treatment were reported; two unrelated SAEs were reported.Injections were generally well tolerated with low frequency of injection site reactions (ISRs): 11 mild ISRs (in 8 patients) out of >2,000 injections. Patients treated ≥6 months at 100 µg/kg/week in the blinded or OLE period demonstrated a consistent and sustained response, with mean AHV of 5.39 cm/year (n=40). “Development of a drug with potential to be both an accepted and a preferred treatment option for children with achondroplasia and their caregivers has a high bar related to safety, efficacy, tolerability and convenience,” said Aimee Shu, M.D., Vice President, Clinical Development, Endocrine & Rare Diseases at Ascendis Pharma. “We are excited to see once-weekly TransCon CNP demonstrated statistically superior growth compared to placebo at the 100 µg/kg/week dose level, with safety and tolerability results supporting further development in children with achondroplasia.” Conference Call and Webcast InformationAscendis Pharma will host a conference call and webcast Monday, November 14, at 8:00 a.m. Eastern Time (ET) to discuss the topline ACcomplisH Trial results. Those who would like to listen to the live webcast can access it through the following link here. To access the live teleconference, register online here. Participants are encouraged to register at least 15 minutes prior to the call. A replay of the webcast will be available on the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com shortly after conclusion of the event for 30 days. About Ascendis Pharma A/SAscendis Pharma is applying its innovative TransCon platform to build a leading, fully integrated, global biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Heidelberg and Berlin, Germany; Palo Alto and Redwood City, California; and Princeton, New Jersey. Please visit www.ascendispharma.com to learn more. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) TransCon CNP’s ability to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose, (ii) TransCon CNP’s potential to meet patient and caregiver needs for a safe, effective, tolerable and convenient treatment and to become an accepted and preferred treatment method, (iii) Ascendis’ plans to advance TransCon CNP in its new Phase 2b ApproaCH trial and to bring TransCon CNP to patients as fast as possible, (iv) Ascendis’ use of its TransCon technologies and platform to create new and potentially best-in-class therapies and build a leading, fully integrated, global biopharma company and (v) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis products and product candidates; unforeseen safety or efficacy results in its development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including inflation, and the effects on its business from the worldwide COVID-19 pandemic and the ongoing conflict in the region surrounding Ukraine and Russia. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on March 2, 2022 and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, and TransCon are trademarks owned by the Ascendis Pharma Group. © November 2022 Ascendis Pharma A/S. Investor Contacts:Media Contact:Tim LeeMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 374-6343+1 (650) 709-8875tle@ascendispharma.commedia@ascendispharma.comir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com