Rani Therapeutics (RANI) - 2021 Q4 - Annual Report

PART I Business Rani Therapeutics is a clinical-stage biotherapeutics company developing the RaniPill capsule, an oral delivery platform for biologics, with a pipeline of product candidates and a vertically integrated manufacturing strategy Overview Rani Therapeutics is a clinical-stage company developing oral administration technologies for biologics to replace injections - Rani Therapeutics is a clinical-stage company focused on developing oral administration technologies for biologics to replace injections[22] - The company has developed the RaniPill capsule, a drug-agnostic oral delivery platform. The current version delivers up to 3 mg, while a high-capacity version (RaniPill HC) in preclinical development aims for up to 20 mg[23] - A Phase 1 trial for RT-102 (parathyroid hormone) was initiated in Australia in March 2022. A previous Phase 1 study for RT-101 (octreotide) successfully demonstrated safety and achieved oral bioavailability greater than 70%[25] Our Platform Technology The RaniPill capsule is designed for oral delivery of biologics, offering high bioavailability and patient preference over injections - The RaniPill capsule is designed to withstand stomach acid and deploy in the small intestine. A self-inflating balloon orientates a dissolvable microneedle to inject the biologic drug into the intestinal wall for absorption into the bloodstream[41][44][45] - Key advantages of the RaniPill include high bioavailability comparable to subcutaneous injection, protection of the drug from the GI environment, and reliable delivery in both fed and fasted states[47][49][50] - A third-party survey indicated a strong preference among patients and physicians for oral pills over injections, even for infrequent dosing regimens (up to every six months)[52][53][55] Our Pipeline The company's pipeline includes multiple product candidates leveraging the RaniPill platform, targeting established markets with well-characterized molecules Product Candidate Pipeline Summary | Product Candidate | Molecule | Indication(s) | Development Stage | Key Milestone/Data | | :--- | :--- | :--- | :--- | :--- | | RT-101 | Octreotide | NETs & Acromegaly | Phase 1 Completed | Bioavailability >70% achieved | | RT-102 | Parathyroid Hormone (PTH) | Osteoporosis | Phase 1 Initiated (Mar 2022) | Evaluating pharmacokinetics, safety, and tolerability | | RT-105 | Anti-TNF-alpha antibody | Psoriatic Arthritis | Preclinical | Plan to initiate Phase 1 in 2023 | | RT-109 | Human Growth Hormone (hGH) | Growth Hormone Deficiency | Preclinical | Plan to initiate Phase 1 in H2 2022 | | RT-110 | Parathyroid Hormone (PTH) | Hypoparathyroidism | Preclinical | Plan to initiate Phase 1 in 2023 | | RT-104 | GLP-1 mimetic | Type 2 Diabetes | Partnering | Proof-of-concept study showed bioavailability similar to subcutaneous injection | | RT-106 | Basal insulin | Type 2 Diabetes | Partnering | Preclinical study showed comparable efficacy to subcutaneous injection | - The pipeline focuses on well-characterized molecules with established markets to potentially accelerate approval and market acceptance[59] Our Regulatory Pathways Rani's product candidates are expected to be classified as combination products by the FDA, following BLA/NDA pathways and leveraging prior findings - Rani's product candidates are expected to be classified by the FDA as combination products (device + biologic), with the Center for Drug Evaluation and Research (CDER) likely being the lead review center[114][115] - The company plans to conduct an Investigational Device Exemption (IDE) study for the RaniPill platform alone to evaluate its safety and tolerability. Data from this study will be used to create a Master File with the FDA's Center for Devices and Radiological Health (CDRH) to support subsequent product applications[117][118][119] - Marketing applications for product candidates are expected to follow the Biologics License Application (BLA) or New Drug Application (NDA) pathway. The company may be able to leverage the FDA's prior findings on the safety and efficacy of the already-approved biologics used in its products[122][123] Manufacturing and Commercialization Rani pursues a vertically integrated manufacturing strategy and plans to commercialize products through in-house efforts or strategic partnerships - Rani pursues a vertically integrated manufacturing strategy, with current facilities in San Jose and Milpitas, California, to control product quality, enable rapid iteration, and protect intellectual property[131] - The current semi-automated manufacturing process is sufficient for planned clinical trials, with plans to fully automate the process for commercialization[134] - The company intends to either build its own commercialization infrastructure or partner with pharmaceutical companies for sales and distribution in key markets like the United States, Europe, and Asia[135][137] - Commercial success will depend on securing coverage and adequate reimbursement from third-party payors, which is a complex and challenging process[138] Intellectual Property The company's intellectual property strategy relies on patents, trademarks, and trade secrets to protect its core technology and product pipeline - The company's IP strategy relies on a combination of patents, trademarks, and trade secrets to protect its technology and products[148] - The core patent family, with a priority date in 2009, includes over 230 patents and applications globally, with terms expected to extend into at least 2030. This family covers device aspects and the delivery of various biologics[150] - A separate patent family covers the microtablets delivered by the RaniPill capsule, with a priority date in 2014 and expected patent terms extending into at least 2035[151] Risk Factors The company faces substantial risks due to its limited operating history, history of net losses, and need for significant additional capital. Development and regulatory risks are high, as its novel drug-device combination products are in early clinical stages with an uncertain and lengthy approval pathway. Commercialization success is not guaranteed and depends on market acceptance, competition, and reimbursement. The company also relies on third parties for key materials and clinical trial execution. Additional risks include potential challenges to its intellectual property, complexities of its holding company structure, and the volatility of its stock price - The company has a limited operating history, has incurred significant losses since inception ($53.1 million net loss in 2021), and expects to continue incurring losses for the foreseeable future. It may never achieve or sustain profitability[222][224] - Clinical development is lengthy, expensive, and uncertain. The company's novel technology has not been approved by the FDA, and regulatory authorities have limited experience evaluating it, making the time and cost of development difficult to predict[248][289] - The company faces significant competition from major pharmaceutical companies with injectable biologics and other companies developing oral delivery technologies. Its success depends on demonstrating advantages in efficacy, convenience, and safety[313][317] - The multi-class stock structure concentrates over 80% of voting power with certain pre-IPO owners (Continuing LLC Owners), limiting the influence of public stockholders on important corporate decisions[510][513] - The company is involved in ongoing patent opposition proceedings in Europe initiated by Novo Nordisk A/S, which could result in the revocation or amendment of key European patents, potentially impacting its ability to commercialize in Europe[469][473] Unresolved Staff Comments The company reports that it has no unresolved staff comments from the Securities and Exchange Commission - There are no unresolved staff comments[556] Properties The company leases approximately 55,000 square feet of office, R&D, and manufacturing space across California and Texas - The company's corporate headquarters are in San Jose, California[557] - It leases approximately 55,000 square feet of space across facilities in San Jose, Milpitas (California), and San Antonio (Texas) under service agreements with the related party InCube Labs, LLC (ICL)[557] Legal Proceedings The company is not currently a party to any legal proceedings expected to have a material adverse effect on its business - The company is not currently party to any litigation or legal proceedings that management believes are likely to have a material adverse effect on the business[559] Mine Safety Disclosures This item is not applicable to the company's business - Not applicable[560] PART II Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities The company's Class A common stock trades on Nasdaq, has never paid dividends, and adjusted its IPO proceeds allocation - The company's Class A common stock is traded on the Nasdaq Stock Market LLC under the symbol "RANI"[562] - The company has never declared or paid dividends and does not intend to in the foreseeable future[562] - The planned use of IPO proceeds was revised, with allocations for the internal pipeline and manufacturing scale-up being reduced from their original ranges[566] Management's Discussion and Analysis of Financial Condition and Results of Operations The company reported a significant net loss in 2021 driven by increased expenses and equity-based compensation, but maintains sufficient cash from its IPO for the next twelve months Results of Operations (2021 vs. 2020) | Metric | 2021 (in thousands) | 2020 (in thousands) | Change Driver | | :--- | :--- | :--- | :--- | | Contract Revenue | $2,717 | $462 | Termination of Takeda agreement led to full recognition of deferred revenue | | Research & Development Expense | $26,482 | $12,044 | Increased headcount and $8.2M in equity-based compensation | | General & Administrative Expense | $27,834 | $4,962 | Increased headcount, public company costs, and $14.4M in equity-based compensation | | Loss from Operations | ($51,599) | ($16,544) | Higher operating expenses | | Net Loss | ($53,088) | ($16,703) | Higher operating loss and other expenses | Cash Flow Summary (2021 vs. 2020) | Metric | 2021 (in thousands) | 2020 (in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | ($32,245) | ($14,960) | | Net cash used in investing activities | ($506) | ($1,200) | | Net cash provided by financing activities | $77,146 | $72,682 | - As of December 31, 2021, the company had cash and cash equivalents of $117.5 million, which is expected to fund operations for at least the next twelve months[621][630] - The company's IPO in August 2021 raised net proceeds of $73.6 million[621] Quantitative and Qualitative Disclosures about Market Risk The company is a smaller reporting company and is therefore not required to provide market risk disclosures - As a smaller reporting company, Rani Therapeutics is not required to provide quantitative and qualitative disclosures about market risk[660] Financial Statements and Supplementary Data This section presents the company's audited consolidated financial statements for 2021 and 2020, including balance sheets, income statements, and cash flows Key Financial Data (Year Ended Dec 31, 2021) | Metric | Amount (in thousands) | | :--- | :--- | | Income Statement: | | | Contract Revenue | $2,717 | | Total Operating Expenses | $54,316 | | Net Loss | ($53,088) | | Balance Sheet (End of Period): | | | Cash and Cash Equivalents | $117,453 | | Total Assets | $124,207 | | Total Liabilities | $2,640 | | Total Stockholders' Equity | $121,567 | - The financial statements reflect the company's transition to a public entity following its IPO in August 2021, including the establishment of a non-controlling interest and a new equity structure[679][685] Changes in and Disagreements with Accountants on Accounting and Financial Disclosure The company reports no changes in or disagreements with its accountants on accounting and financial disclosure - None[820] Controls and Procedures Management concluded the company's disclosure controls were effective as of December 31, 2021, with a new stock plan system implemented - Management concluded that the company's disclosure controls and procedures were effective at a reasonable assurance level as of December 31, 2021[821] - As a newly public company, a management assessment of internal control over financial reporting is not yet required[822] - A new stock plan administration system was implemented in 2021, leading to changes in internal controls over financial reporting[823] Other Information The company reports no other information for this item - None[826] PART III Directors, Executive Officers and Corporate Governance Information for this item is incorporated by reference from the company's 2022 Proxy Statement - Information is incorporated by reference from the company's 2022 Proxy Statement[829] Executive Compensation Information for this item regarding executive compensation is incorporated by reference from the company's 2022 Proxy Statement - Information is incorporated by reference from the company's 2022 Proxy Statement[831] Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters Information for this item concerning security ownership is incorporated by reference from the company's 2022 Proxy Statement - Information is incorporated by reference from the company's 2022 Proxy Statement[832] Certain Relationships and Related Transactions, and Director Independence Information for this item detailing related party transactions and director independence is incorporated by reference from the company's 2022 Proxy Statement - Information is incorporated by reference from the company's 2022 Proxy Statement[833] Principal Accountant Fees and Services Information for this item regarding principal accountant fees and services is incorporated by reference from the company's 2022 Proxy Statement - Information is incorporated by reference from the company's 2022 Proxy Statement[834] PART IV Exhibits and Financial Statement Schedules This section lists the documents filed as part of the Annual Report on Form 10-K, including financial statements and exhibits - This item contains the list of financial statements and exhibits filed as part of the Form 10-K[835][837] Form 10-K Summary The company reports no information for this item - None[839]