Pieris Pharmaceuticals(US:PIRS)2023-03-31 20:01Clinical Development - Pieris Pharmaceuticals has a clinical pipeline that includes elarekibep, an inhaled IL-4Rα antagonist for uncontrolled asthma, and PRS-220, an inhaled CTGF antagonist for idiopathic pulmonary fibrosis[22]. - The phase 2a asthma study for elarekibep is ongoing, with topline data expected by mid-2024[28]. - The first patient was dosed in the phase 1 study of PRS-220 in healthy volunteers in Q4 2022, with results expected in 2023[32]. - The ongoing phase 2a study of elarekibep includes a safety review and evaluation of adverse events compared to placebo[63]. - PRS-220 is in phase 1 clinical development, with a €14.2 million grant received to support its research and development[64]. - Elarekibep demonstrated a 36% relative reduction in FeNO at the 20 mg dose compared to placebo, with a p-value <0.0001, indicating significant efficacy in reducing eosinophilic airway inflammation[62]. - The inhaled route of administration for PRS-220 offers advantages over parenterally administered CTGF-targeted therapies[75]. - PRS-220 binds to CTGF with high affinity in the picomolar range, demonstrating higher affinity and more stable target engagement compared to the anti-CTGF antibody pamrevlumab[77]. - PRS-400, targeting Jagged-1, is being developed as an inhaled treatment for muco-obstructive lung diseases, aiming to improve quality of life by reducing mucus hypersecretion[83]. - Preclinical data for PRS-400 indicates potent inhibition of Jagged-1-induced Notch 2 signaling and reduction of major airway mucins, addressing key features of muco-obstructive lung diseases[84]. Financial Milestones and Collaborations - The company achieved a $13.0 million milestone related to the initiation of the phase 2a study for elarekibep and issued 3,584,230 shares to AstraZeneca for $10.0 million in a private placement[25]. - Pieris Pharmaceuticals has collaborations with major pharmaceutical companies, including a license agreement with AstraZeneca for respiratory diseases[25]. - The company has entered into a multi-program research collaboration with Genentech to develop locally delivered respiratory therapies[34]. - The company retains co-development and U.S. co-commercialization options for two discovery-stage programs in collaboration with AstraZeneca[30]. - A key development milestone was achieved in the Seagen collaboration, triggering a $5 million milestone payment for the SGN-BB228 program[97]. - Under the AstraZeneca Agreements, the company received an upfront payment of $45.0 million and is eligible for total potential milestones of up to $3.9 billion, including tiered royalties on sales[137]. - The Servier Agreements provided an upfront payment of €30.0 million (approximately $32.0 million) and potential milestone payments of up to €19.0 million during the collaboration[141]. - The Seagen Agreements included a $30 million upfront fee and potential success-based payments totaling up to $1.2 billion across three product candidates[144]. - The company entered into a product license agreement with Boston Pharmaceuticals, receiving an upfront payment of $10.0 million and potential milestone payments of up to $352.5 million[150]. - The Genentech Agreement included a $20 million upfront fee and potential additional milestone payments of approximately $1.4 billion across multiple programs[154]. Intellectual Property and Manufacturing - The company has a strong intellectual property position related to its Anticalin platform, which is crucial for addressing novel targets and diseases[24]. - The company has a patent portfolio that includes several issued U.S. patents and pending applications, with potential expiration dates ranging from 2023 to 2043[126]. - The company holds an exclusive license to multiple patents related to Anticalin proteins, with some expected to expire in 2029[128]. - The company is pursuing intellectual property protection for its drug candidates, which could provide exclusivity for up to 12 years in the U.S. for new biological entities[127]. - The company relies on third-party contract manufacturing organizations (CMOs) for the production of drug candidates, with no current plans to establish its own manufacturing facilities[116]. - The company has experienced reduced capacity from CMOs due to the COVID-19 pandemic, impacting its supply chain[118]. Market Potential and Competition - The asthma market includes over 300 million affected individuals globally, with significant economic impacts, including approximately $82 billion in costs in the U.S. in 2013[51]. - In 2021, about 55% of asthma patients over 12 years of age in the U.S. had moderate-to-severe asthma, indicating a substantial patient population for potential treatment[52]. - Omalizumab, a competitor drug, reported global sales of $3,734 million in 2022, highlighting the market potential for Anticalin-based therapies[52]. - The company faces intense competition in the pharmaceutical and biotechnology industries, with competitors developing products targeting the same diseases[102]. - The company faces significant competition from established therapies for respiratory diseases and cancer, with many competitors having greater financial and technical resources[106]. - There are multiple drugs in clinical development for respiratory diseases and cancer that may provide advantages over currently marketed therapies, posing a threat to the company's product candidates[105]. Regulatory Environment - The FDA regulates the drug approval process, requiring substantial time and financial resources for compliance with regulations[168]. - The company must submit progress reports on clinical trials to the FDA, with the potential for suspension or termination of trials based on safety concerns[180]. - The FDA conducts a preliminary review of all NDAs and BLAs within 60 days and informs the sponsor by the 74th day whether the application is sufficiently complete for substantive review[185]. - The approval process includes a pre-approval inspection of manufacturing facilities to ensure compliance with cGMPs[188]. - Products may receive accelerated approval if they provide meaningful therapeutic benefit over existing treatments, based on adequate and well-controlled clinical trials[199]. - The accelerated approval pathway is often used for serious or life-threatening diseases where traditional approval may take longer due to lengthy clinical trials[201]. - Sponsors of drugs granted accelerated approval must conduct post-marketing confirmatory studies to verify clinical benefits[202]. - The FDA may require sponsors to have a confirmatory trial underway prior to approval under recent amendments to the FDCA[203]. - The FDA has the authority to withdraw drug approval if post-approval studies fail to confirm predicted clinical benefits[204]. Strategic Development - The company aims to become a fully-integrated biotechnology company by commercializing Anticalin-based therapeutics targeting validated disease pathways[42]. - The company is actively seeking additional financing to support its operations and development activities[12]. - The company plans to actively seek additional collaboration partners that align with its corporate development strategy[134]. - The collaboration with AstraZeneca includes two undisclosed programs beyond elarekibep, with ongoing discovery work[86]. - The collaboration with Genentech focuses on locally delivered respiratory and ophthalmology therapies, with Pieris responsible for discovery research and Genentech for clinical development[87].