Akebia Therapeutics(US:AKBA)2023-03-09 16:00Financial Collaborations and Agreements - The collaboration with Otsuka Pharmaceutical included an upfront payment of $125 million and additional funding of $353 million for the vadadustat global Phase 3 development program[95]. - The termination of the Otsuka agreements resulted in a nonrefundable payment of $55 million and the transfer of marketing authorization applications for vadadustat back to the company[96]. - The collaboration with Mitsubishi Tanabe Pharma Corporation includes potential payments of up to $225 million based on development and sales milestones, along with tiered royalties of 13% to 20% on annual net sales in the MTPC Territory[99]. - CSL Vifor made an upfront payment of $25 million and contributed $40 million to a working capital facility under the Vifor Second Amended Agreement[101][102]. - The Cyclerion Agreement includes an upfront payment of $3 million and potential milestone payments of up to $222 million, along with tiered royalties on net sales[108]. - The Panion Amended License Agreement allows for royalty payments based on a mid-single digit percentage of sales of ferric citrate in licensed territories[113]. - Panion earned $13.8 million in royalty payments for the year ended December 31, 2022, related to Auryxia sales in the U.S. and Riona sales in Japan[115]. - Keryx recorded $5.3 million in license revenue from royalties on Riona net sales in Japan during the twelve months ended December 31, 2022[118]. - Keryx is eligible to receive up to an additional $55.0 million upon achieving certain annual net sales milestones for Riona in Japan[118]. Patent and Intellectual Property - The expected expiration dates for vadadustat patents range from 2027 to 2034, with additional extensions possible[124]. - Auryxia patent rights include 14 issued U.S. patents with expected expiration dates between 2024 and 2030, plus potential extensions[128]. - The sublicense with JT and Torii for Riona includes rights to several Japanese patents with expiration dates between 2025 and 2027[129]. - Keryx has entered into settlement agreements allowing generic versions of Auryxia to be marketed in the U.S. starting March 20, 2025, subject to FDA approval[131]. - The company relies on a combination of patents, trade secrets, and technological innovation to maintain its competitive position[134]. - Changes in patent laws could impact Keryx's ability to protect its inventions and enforce intellectual property rights[122]. - Keryx's commercial success is dependent on obtaining and maintaining patent protection for its current and future product candidates[120]. - The company has filed applications for patent term extensions for U.S. Patent Nos. 8,299,298, 8,093,423, 7,767,851, and 8,338,642, which cover Auryxia, due to delays caused by FDA regulatory review[143]. Market Competition - Auryxia competes in the hyperphosphatemia market against FDA-approved phosphate binders such as Renagel® and Renvela®, with competition also from over-the-counter products like TUMS®[155]. - Auryxia faces competition in the iron deficiency anemia market from various oral and intravenous iron formulations, including Feraheme® and Injectafer®[157]. - The FDA approved Jesduvroq (daprodustat) for treating anemia due to CKD, which may compete with vadadustat if approved[149]. - The company is aware of several HIF-PH inhibitor product candidates in development that may compete with vadadustat upon approval[150]. - The company faces substantial competition from larger pharmaceutical and biotechnology firms with greater resources and experience in drug development[147]. Regulatory Compliance and Drug Approval - The company must comply with extensive regulations for drug approval, which require significant time and financial resources[159]. - The FDA requires a 30-day waiting period after the submission of each IND before clinical trials may begin[165]. - The application fee for submitting an NDA under the Prescription Drug User Fee Act (PDUFA) for fiscal year 2023 is approximately $3.2 million[184]. - The annual program fee for an approved NDA is currently more than $393,000 per eligible prescription product[184]. - Sponsors must submit an initial Pediatric Study Plan (PSP) within 60 days of an end-of-phase 2 meeting[180]. - The PHSA mandates that sponsors register and disclose clinical trial information on a public registry, with potential civil monetary penalties of up to $10,000 for each day of noncompliance[170]. - Expanded access allows investigational drugs to be used outside of clinical trials for patients with serious conditions when no satisfactory alternatives exist[171]. - The Right to Try Act allows eligible patients to access investigational drugs that have completed Phase I trials without enrolling in clinical trials[173]. - The FDA may grant deferrals for pediatric data submission until after adult approval, based on specific criteria[183]. - The FDA's regulations are designed to ensure the protection of human subjects in clinical trials and the integrity of clinical data[167]. - Sponsors must submit progress reports detailing clinical trial results at least annually to the FDA[178]. - The FDA conducts a preliminary review of all applications within 60 days and must inform the sponsor whether the application is sufficiently complete for substantive review[185]. - Most NDA applications are reviewed within ten months, while priority review products are reviewed within six months[190]. - The FDA may designate a product for fast track review if it addresses an unmet medical need for a serious disease[191]. - A product may be designated as a breakthrough therapy if it shows substantial improvement over existing therapies based on preliminary clinical evidence[192]. - The FDA may issue a Complete Response Letter (CRL) outlining deficiencies in the submission, requiring substantial additional testing or information[198]. - An approval letter authorizes commercial marketing of the product with specific prescribing information and may impose post-approval requirements[199]. - The FDA may withdraw approval if compliance with regulatory requirements is not maintained or if new problems are discovered post-market[205]. - The FDA strictly regulates marketing and promotion of prescription drugs, prohibiting promotion of unapproved uses[206]. - Companies may face significant penalties for promoting off-label uses, including civil and criminal fines[208]. - The Prescription Drug Marketing Act (PDMA) and the Drug Supply Chain Security Act (DSCSA) impose regulations on the distribution of prescription pharmaceutical products, ensuring accountability and establishing national standards for wholesale distribution[209]. - Section 505(b)(2) NDAs allow sponsors to rely on previous FDA findings for similar products, potentially expediting the approval process for new formulations or uses[210]. - The Hatch-Waxman Amendments enable the FDA to approve generic drugs through abbreviated new drug applications (ANDAs), which do not require preclinical and clinical data to demonstrate safety and effectiveness[211]. - A five-year non-patent data exclusivity period is granted for new drugs containing a new chemical entity (NCE), preventing generic applications until the exclusivity period expires[212]. - The FDA must establish a priority review track for certain generic drugs, requiring review within eight months for drugs with limited competition[215]. - Pediatric exclusivity can extend marketing protection by six months if pediatric data is submitted in response to FDA requests[218]. - Patent term restoration under the Hatch-Waxman Act allows for a limited extension of up to five years for patents lost during product development and FDA review[220]. Privacy and Data Protection - The California Consumer Privacy Act (CCPA) and the California Privacy Rights Act (CPRA) impose new privacy obligations on companies handling personal data, which could impact business activities[222]. - Compliance with evolving federal and state privacy laws may increase regulatory risks and obligations for the company[223]. - The General Data Protection Regulation (GDPR) imposes strict requirements on the processing of personal data, including health data, within the EEA[245]. - The GDPR imposes potential fines of up to €20 million or 4% of annual global revenues for violations, whichever is greater[246]. - The U.K. Data Protection Act 2018 parallels GDPR but raises questions about data transfer legality post-Brexit[250]. - The U.S. President signed an executive order in October 2022 to implement a new EU-U.S. Data Privacy Framework, but its finalization remains uncertain[249]. Financial Performance and Operations - As of December 31, 2022, the company had cash and cash equivalents of $90.5 million, down from $149.8 million in 2021[695]. - The company provided over $5.3 million worth of Auryxia for free to approximately 14,000 patients in 2022[284]. - The company employs 205 individuals, with all but one being full-time[279]. - The company aims to cap Medicare out-of-pocket drug costs at an estimated $4,000 a year in 2024, reducing to $2,000 a year starting in 2025[273]. - The Inflation Reduction Act requires manufacturers to engage in price negotiations with Medicare starting in 2026 for certain drugs[272]. - A substantial portion of the company's revenues for the year ended December 31, 2022, was received in U.S. dollars, including royalty payments from Riona and Vafseo in Japanese yen[698]. - A 5.0% appreciation or depreciation of the Japanese yen against the U.S. dollar would have increased or decreased the company's revenues by approximately $0.4 million for the year ended December 31, 2022[698]. Corporate Social Responsibility and Diversity - The company is committed to diversity, with women comprising approximately 45% of its senior management team[282]. - The company has launched a platform called Modern Health to support employees' well-being, focusing on mental, physical, financial, social, and professional health[281]. - The company has developed a team called IDEA to promote inclusion, diversity, and equity within the workplace[283]. - The company is exploring options to incorporate value into payments for Medicare Part B pharmaceuticals[269]. Market Access and Pricing - The ESRD prospective payment system (PPS) includes dialysis-related drugs, with separate payment for eligible new drugs for two years based on Average Sales Price (ASP)[254]. - The U.S. government has implemented automatic reductions to Medicare payments to providers of up to 2% per fiscal year, effective since April 2013, with potential variations up to 4% under current legislation[265]. - Third-party payors increasingly challenge drug prices and may impose prior authorization requirements, affecting product sales[252]. - The European Union allows member states to restrict reimbursement for products, leading to potential barriers for new product entries[257]. - Pricing negotiations for pharmaceuticals can extend beyond regulatory approval, potentially delaying commercialization[256]. - The company may need to conduct expensive pharmacoeconomic studies to secure coverage and reimbursement for approved products[253].