Part I [Business](index=7&type=section&id=Item%201.%20Business) Rallybio is a clinical-stage biotechnology company focused on developing therapies for severe and rare diseases, leveraging strategic collaborations and intellectual property while navigating extensive government regulation - The company's mission is to develop and commercialize life-transforming therapies for patients with severe and rare diseases, focusing on maternal fetal health, complement dysregulation, hematology, and metabolic disorders[21](index=21&type=chunk) Product Pipeline as of December 31, 2024 | Molecule | Approach | Indication | Discovery | Preclinical | Phase 1 | Phase 2 | Phase 3 | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | RLYB212 | Anti-HPA-1a Monoclonal Antibody | Prevention of FNAIT | | | ✅ | ✅ | | | RLYB116 | C5 Inhibitor Affibody®-ABD Fusion | Diseases of complement dysregulation | | | ✅ | | | | REV102 | ENPP1 Inhibitor Small Molecule | Hypophosphatasia (HPP) | | ✅ | | | Partnership with Recursion | | RLYB332 | Matriptase-2 Inhibitor Monoclonal Antibody | Diseases of iron overload and severe anemias | | ✅ | | | | | RLYB114 | C5 Inhibitor Pegylated Affibody® | Ophthalmological diseases of complement dysregulation | | ✅ | | | | | Undisclosed | - | Undisclosed metabolic disease | Partnership with AbCellera | | | | | [Our Product Candidates](index=8&type=section&id=Item%201.%20Business.Our%20Product%20Candidates) Rallybio's pipeline is led by RLYB212 for FNAIT and RLYB116 for complement-mediated diseases, with preclinical assets including RLYB332 for iron overload and REV102 for HPP - RLYB212 is in a Phase 2 clinical trial in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT, with enrollment in three stages[27](index=27&type=chunk)[28](index=28&type=chunk) - Following biomarker analyses and manufacturing enhancements for RLYB116, the company plans to initiate a confirmatory clinical PK/PD trial in Q2 2025[36](index=36&type=chunk) - RLYB332, a long-acting MTP-2 inhibitor, has shown favorable preclinical PD data, supporting its potential as a best-in-class therapy for diseases of iron overload like beta thalassemia and MDS[37](index=37&type=chunk) - In collaboration with Recursion, the company advanced REV102, an ENPP1 inhibitor for HPP, and plans for additional preclinical development activities in 2025[39](index=39&type=chunk) [Competition](index=22&type=section&id=Item%201.%20Business.Competition) Rallybio faces competition across its pipeline, with no approved preventative therapies for FNAIT, and established competitors in PNH, gMG, and HPP markets - **FNAIT:** No approved therapies exist, with Johnson & Johnson evaluating nipocalimab for treatment[110](index=110&type=chunk) - **PNH:** Competes with approved drugs Soliris and Ultomiris (AstraZeneca), Empaveli (Apellis), and Fabhalta (Novartis)[111](index=111&type=chunk) - **gMG:** Competes with approved drugs from AstraZeneca, Argenx (Vyvgart), and UCB (Zilbrysq, Rystiggo)[112](index=112&type=chunk) - **HPP:** Competes with the only approved therapy, Strensiq (AstraZeneca)[113](index=113&type=chunk) [Intellectual Property](index=23&type=section&id=Item%201.%20Business.Intellectual%20Property) The company's intellectual property strategy relies on owned and in-licensed patents and trade secrets, with key patent expirations for FNAIT in 2035 and complement programs between 2033 and 2034 - The patent family covering RLYB212 and its use in preventing FNAIT includes issued patents in the U.S., Europe, and other regions, which will expire in **2035**, excluding any potential extensions[122](index=122&type=chunk) - The complement program, including RLYB114 and RLYB116, is protected by two patent families acquired from Sobi, with patents granted in the U.S., Europe, and Japan that are scheduled to expire between **2033 and 2034**[124](index=124&type=chunk) - The company has exclusively in-licensed patent rights for RLYB332 from Kymab Limited, with patent applications pending in the U.S. and over 20 other countries[125](index=125&type=chunk) [License and Collaboration Agreements](index=25&type=section&id=Item%201.%20Business.License%20and%20Collaboration%20Agreements) Rallybio has key agreements including asset purchases for RLYB212 and RLYB116/114, license agreements for RLYB332, a joint venture for ENPP1 inhibitors, and collaborations with AbCellera and Johnson & Johnson - Entered a collaboration with Johnson & Johnson in April 2024 to advance FNAIT research, involving data sharing, an upfront payment of **$0.5 million**, and potential milestone payments up to **$3.7 million**[155](index=155&type=chunk)[156](index=156&type=chunk) - Acquired RLYB212 assets from Prophylix for an upfront payment, with up to **$19.0 million** in development milestones and **$20.0 million** in sales milestones[142](index=142&type=chunk)[143](index=143&type=chunk) - Acquired RLYB116/RLYB114 assets from Sobi for a **$5.0 million** upfront payment, with up to **$51.0 million** in development milestones and **$65.0 million** in sales milestones[137](index=137&type=chunk)[138](index=138&type=chunk) - Licensed RLYB331 (now RLYB332) from Sanofi for a **$3.0 million** upfront payment, with up to **$43.0 million** in development/regulatory milestones and **$150.0 million** in commercial milestones[133](index=133&type=chunk)[134](index=134&type=chunk) [Government Regulation](index=30&type=section&id=Item%201.%20Business.Government%20Regulation) The company's operations are subject to extensive regulation by the FDA and comparable authorities, covering preclinical studies, clinical trials, manufacturing, and post-approval requirements, including pricing, reimbursement, and data privacy - The company must navigate a multi-step approval process with the FDA, including preclinical studies, an effective IND application, and adequate and well-controlled clinical trials (Phase 1, 2, 3) under GCPs[165](index=165&type=chunk)[166](index=166&type=chunk) - RLYB212 has received Orphan Drug Designation from both the FDA and EMA, which provides incentives like market exclusivity (**7 years** in the U.S., **10 years** in the EU) upon approval[214](index=214&type=chunk)[245](index=245&type=chunk) - RLYB212 has also received Rare Pediatric Disease Designation from the FDA, making it potentially eligible for a Priority Review Voucher upon approval, subject to certain conditions and statutory deadlines[218](index=218&type=chunk) - The company is subject to various U.S. healthcare laws, including anti-kickback statutes, false claims acts, and the Sunshine Act, as well as global data privacy laws like GDPR, which impose significant compliance obligations[259](index=259&type=chunk)[268](index=268&type=chunk) [Risk Factors](index=47&type=section&id=Item%201A.%20Risk%20Factors) The company faces substantial risks including significant losses, capital needs, dependence on early-stage clinical candidates, regulatory uncertainties, reliance on third parties, intense competition, intellectual property disputes, patient recruitment challenges, and stock volatility - **Financial Risk:** The company has a history of significant losses (**$57.8 million** in 2024) and will require substantial additional capital, with failure to obtain financing risking program delays or discontinuation[276](index=276&type=chunk)[281](index=281&type=chunk) - **Clinical Development Risk:** The company is heavily dependent on the success of RLYB212 and RLYB116, which are in early-stage clinical development, and preclinical and early clinical results may not be indicative of later-stage trial outcomes[295](index=295&type=chunk)[308](index=308&type=chunk) - **Regulatory Risk:** The marketing approval process is lengthy, unpredictable, and may require companion diagnostics, and even if approved, products will face extensive ongoing regulation and potential competition from generics or biosimilars[319](index=319&type=chunk)[366](index=366&type=chunk)[368](index=368&type=chunk) - **Third-Party Reliance:** The company relies on third-party CMOs for all manufacturing and CROs for clinical trials, increasing risks related to supply, quality control, and trial execution[383](index=383&type=chunk)[393](index=393&type=chunk) - **Stock Risk:** The company received a notice from Nasdaq on February 24, 2025, for failing to maintain a minimum bid price of **$1.00** per share, posing a risk of delisting[476](index=476&type=chunk) [Cybersecurity](index=95&type=section&id=Item%201C.%20Cybersecurity) Rallybio has implemented a cybersecurity risk management program based on the NIST Cybersecurity Framework, overseen by the audit committee, and has not experienced any material incidents to date - The company's cybersecurity program is guided by the NIST Cybersecurity Framework and includes risk assessments, security controls, and an incident response plan[512](index=512&type=chunk)[513](index=513&type=chunk) - Oversight is provided by the audit committee of the board of directors, with management handled by a third-party IT service provider and internal staff[516](index=516&type=chunk) - As of the report date, the company has not experienced any cybersecurity incidents that have had a material effect on its operations or financial condition[515](index=515&type=chunk) [Properties](index=96&type=section&id=Item%202.%20Properties) The company's corporate headquarters is located in New Haven, CT, where it leases approximately 9,000 square feet of office space under a lease expiring on September 30, 2025 - Leases **9,000 sq. ft.** of office space for its headquarters at 234 Church Street, Suite 1020, New Haven, CT 06510[517](index=517&type=chunk) - The current lease expires on **September 30, 2025**[517](index=517&type=chunk) Part II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=96&type=section&id=Item%205.%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) Rallybio's common stock trades on Nasdaq under 'RLYB' since July 2021, with no dividends paid or anticipated, and proceeds from IPO and offerings supporting pipeline development - Common stock trades on Nasdaq under the symbol **'RLYB'** since its IPO in July 2021[521](index=521&type=chunk) - The company has never paid dividends and intends to retain future earnings to finance business operations and expansion[523](index=523&type=chunk) - Net proceeds from the IPO were **$83.0 million**, which are intended to support pipeline development, working capital, and general corporate purposes[528](index=528&type=chunk)[529](index=529&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=98&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) For 2024, Rallybio's net loss decreased to $57.8 million due to lower R&D and G&A expenses, with $0.6 million in new collaboration revenue, and $65.5 million in cash expected to fund operations into H2 2026 [Results of Operations](index=104&type=section&id=Item%207.%20Results%20of%20Operations) For fiscal year 2024, Rallybio's net loss decreased to $57.8 million from $74.6 million in 2023, driven by a $17.8 million reduction in total operating expenses, primarily in R&D and G&A, and initial collaboration revenue Comparison of Operations for Years Ended Dec 31, 2024 and 2023 (in thousands) | (in thousands) | 2024 | 2023 | CHANGE | | :--- | :--- | :--- | :--- | | **Collaboration and license revenue** | $ 636 | $ — | $ 636 | | **Research and development** | 41,507 | 53,544 | (12,037) | | **General and administrative** | 19,625 | 25,388 | (5,763) | | **Total operating expenses** | 61,132 | 78,932 | (17,800) | | **Loss from operations** | (60,496) | (78,932) | 18,436 | | **Net loss** | $ (57,775) | $ (74,564) | $ 16,789 | Research and Development Expenses by Program (in thousands) | (in thousands) | 2024 | 2023 | CHANGE | | :--- | :--- | :--- | :--- | | RLYB212 | $ 21,287 | $ 25,685 | $ (4,398) | | RLYB116 | 4,841 | 8,791 | (3,950) | | Other program candidates | 1,901 | 3,411 | (1,510) | | Personnel expenses | 12,488 | 14,160 | (1,672) | | **Total R&D expenses** | **$ 41,507** | **$ 53,544** | **$ (12,037)** | - The decrease in R&D expenses was primarily due to lower development costs for RLYB212 and RLYB116, and reduced payroll expenses following a workforce reduction effective March 6, 2024[570](index=570&type=chunk) - The decrease in G&A expenses was mainly due to lower consulting fees, D&O insurance premiums, and reduced payroll costs from the 2024 workforce reduction[570](index=570&type=chunk) [Liquidity and Capital Resources](index=105&type=section&id=Item%207.%20Liquidity%20and%20Capital%20Resources) As of December 31, 2024, Rallybio had $65.5 million in cash, cash equivalents, and marketable securities, projected to fund operations into the second half of 2026, with primary liquidity from equity financings - The company projects its existing cash of **$65.5 million** (as of Dec 31, 2024) will fund operations into the **second half of 2026**[552](index=552&type=chunk)[580](index=580&type=chunk) - In April 2024, the company raised approximately **$6.6 million** in gross proceeds through a private placement of common stock to Johnson & Johnson Innovation – JJDC, Inc[551](index=551&type=chunk)[577](index=577&type=chunk) Cash Flow Summary (in thousands) | (in thousands) | 2024 | 2023 | | :--- | :--- | :--- | | Net cash used in operating activities | $ (49,282) | $ (60,265) | | Net cash provided by investing activities | 33,492 | 27,658 | | Net cash provided by financing activities | 5,199 | 143 | | **Net decrease in cash and cash equivalents** | **$ (10,591)** | **$ (32,464)** | [Financial Statements and Supplementary Data](index=110&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) The audited consolidated financial statements for 2024 show a net loss of $57.8 million and total assets of $68.1 million, with notes detailing the J&J collaboration, asset acquisitions, and a 2024 restructuring [Consolidated Financial Statements](index=118&type=section&id=Item%208.%20Consolidated%20Financial%20Statements) The consolidated financial statements show a net loss of $57.8 million for 2024, a decrease from $74.6 million in 2023, with total assets decreasing to $68.1 million primarily due to lower cash and marketable securities Consolidated Balance Sheet Data (in thousands) | | Dec 31, 2024 | Dec 31, 2023 | | :--- | :--- | :--- | | Cash and cash equivalents | $13,903 | $24,494 | | Marketable securities | $51,608 | $85,435 | | **Total Assets** | **$68,108** | **$115,620** | | Total current liabilities | $6,242 | $9,263 | | **Total Liabilities** | **$6,454** | **$9,436** | | **Total Stockholders' Equity** | **$61,654** | **$106,184** | Consolidated Statement of Operations Data (in thousands) | | Year Ended Dec 31, 2024 | Year Ended Dec 31, 2023 | | :--- | :--- | :--- | | Collaboration and license revenue | $636 | $— | | Research and development | $41,507 | $53,544 | | General and administrative | $19,625 | $25,388 | | Loss from operations | $(60,496) | $(78,932) | | **Net Loss** | **$(57,775)** | **$(74,564)** | | **Net Loss Per Share** | **$(1.33)** | **$(1.84)** | [Notes to Consolidated Financial Statements](index=122&type=section&id=Item%208.%20Notes%20to%20Consolidated%20Financial%20Statements) Key notes to the financial statements detail the J&J collaboration revenue, equity transactions including a $6.6 million stock sale to JJDC, deferred tax assets offset by a valuation allowance, and a February 2024 restructuring with a $3.3 million charge - In February 2024, the company initiated a restructuring, eliminating approximately **45%** of its positions and incurring charges of approximately **$3.3 million** for severance and benefits[749](index=749&type=chunk)[750](index=750&type=chunk) - The company has federal net operating loss carryforwards of **$167.4 million** that do not expire and federal R&D tax credit carryforwards of **$23.4 million** that begin expiring in 2039, with a full valuation allowance recorded against deferred tax assets[734](index=734&type=chunk)[735](index=735&type=chunk)[736](index=736&type=chunk) - The company recognized **$0.6 million** in revenue in 2024 related to its collaboration with Johnson & Johnson, with total consideration of **$1.2 million** from the associated equity sale premium and discount allocated to revenue to be recognized over the two-year performance period[703](index=703&type=chunk)[706](index=706&type=chunk) [Controls and Procedures](index=110&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures and internal control over financial reporting were effective as of December 31, 2024, with no material changes during the quarter - Management concluded that as of December 31, 2024, the company's disclosure controls and procedures were effective[607](index=607&type=chunk) - Management concluded that the company's internal control over financial reporting was effective as of December 31, 2024[608](index=608&type=chunk) Part III [Directors, Executive Officers, Corporate Governance, Executive Compensation, Security Ownership, and Certain Relationships](index=111&type=section&id=Items%2010%2C%2011%2C%2012%2C%2013%2C%20and%2014) Information for Items 10 through 14, covering directors, executive officers, corporate governance, executive compensation, security ownership, and certain relationships, is incorporated by reference from the 2025 Proxy Statement - The information for Items 10, 11, 12, 13, and 14 is incorporated by reference from the registrant's Definitive Proxy Statement for its 2025 Annual Meeting of Stockholders[614](index=614&type=chunk)[615](index=615&type=chunk)[616](index=616&type=chunk)[617](index=617&type=chunk)[618](index=618&type=chunk) Part IV [Exhibits, Financial Statement Schedules](index=111&type=section&id=Item%2015.%20Exhibits%2C%20Financial%20Statement%20Schedules) This section lists all exhibits filed with the Form 10-K, including corporate governance documents, material contracts such as asset purchase and license agreements, collaboration agreements, employment agreements, and officer certifications - Lists all exhibits filed with the Form 10-K, including corporate governance documents, material contracts, and officer certifications[622](index=622&type=chunk)

Financial Performance - Revenue for Q4 2024 was $38 thousand, and total revenue for the year ended December 31, 2024, was $0.6 million, compared to no revenue in the same periods in 2023[12] - R&D expenses decreased to $7.4 million in Q4 2024 from $15.9 million in Q4 2023, and for the full year, R&D expenses were $41.5 million compared to $53.5 million in 2023[12] - G&A expenses were $4.3 million for Q4 2024, down from $5.2 million in Q4 2023, and totaled $19.6 million for the year compared to $25.4 million in 2023[12] - Net loss for Q4 2024 was $11.0 million, or $0.25 per share, compared to a net loss of $20.2 million, or $0.50 per share, in Q4 2023[13] - Cash, cash equivalents, and marketable securities as of December 31, 2024, were $65.5 million, providing a runway into the second half of 2026[6] Clinical Development - RLYB212 Phase 2 trial dosing is underway, with key data readouts expected in Q2 and Q3 2025[6] - RLYB116 is on track to enter a confirmatory PK/PD study in Q2 2025, with data anticipated in the second half of 2025[6] - More than 14,300 pregnant women were screened in the FNAIT natural history study as of January 31, 2025[7] - The company plans to present interim data from the FNAIT natural history study in mid-2025[7] - REV102, an ENPP1 inhibitor, is advancing toward a Phase 1 study expected to start in 2026[12]

Financial Performance - Total revenue for the three months ended September 30, 2024, was $299,000, compared to $0 for the same period in 2023, indicating a significant increase[17]. - The net loss for the three months ended September 30, 2024, was $11,466,000, compared to a net loss of $18,374,000 for the same period in 2023, showing a 37.5% improvement[17]. - The company reported a net loss of $11.5 million for the three months ended September 30, 2024, compared to a net loss of $18.4 million for the same period in 2023, reflecting a decrease of $6.9 million[115]. - Net loss for the nine months ended September 30, 2024, was $46.7 million, an improvement from a net loss of $54.3 million in 2023, a reduction of $7.6 million[122]. Expenses - Research and development expenses for the nine months ended September 30, 2024, were $34,122,000, down from $37,620,000 in the same period of 2023, reflecting a 6.6% decrease[17]. - The company reported a total operating expense of $49,486,000 for the nine months ended September 30, 2024, down from $57,820,000 in the same period of 2023, a reduction of 14.5%[17]. - General and administrative expenses were $4.1 million for the three months ended September 30, 2024, compared to $6.1 million in 2023, reflecting a decrease of $2.0 million[119]. - Research and development expenses for the three months ended September 30, 2024, were $8.2 million, down from $13.3 million in the same period in 2023, a reduction of $5.0 million[115]. Cash and Liquidity - Cash and cash equivalents as of September 30, 2024, were $25,320,000, an increase from $24,494,000 as of December 31, 2023[14]. - As of September 30, 2024, the company had cash, cash equivalents, and marketable securities totaling $75.1 million, which is expected to fund operations for more than 12 months[24]. - The company expects existing cash resources to fund operations into the middle of 2026, assuming no significant changes in expenses[131]. - The company reported a net increase in cash and cash equivalents of $826,000 for the nine months ended September 30, 2024, compared to a decrease of $37.8 million in the same period of 2023[133]. Shareholder Equity and Stock - The total stockholders' equity as of September 30, 2024, was $70,818, an increase from $123,581 as of September 30, 2023[19]. - The weighted-average common shares outstanding increased to 44,593,221 for the three months ended September 30, 2024, compared to 40,531,497 for the same period in 2023[17]. - The Company had 41,487,586 shares of common stock issued and outstanding as of September 30, 2024, an increase from 37,829,565 shares as of December 31, 2023[46]. - The company issued 3,636,363 common shares upon completion of a securities purchase agreement, net of offering costs of $268[19]. Product Development and Clinical Trials - The company is heavily dependent on the success of its product candidates RLYB212 and RLYB116, which are currently in early-stage clinical development[9]. - The company plans to initiate a Phase 2 clinical trial of RLYB212 in the fourth quarter of 2024, following the completion of Phase 1 studies[23]. - RLYB212, an anti-HPA-1a antibody, is set to initiate a Phase 2 clinical trial in Q4 2024, following EMA and MHRA approval[83]. - RLYB116, a C5 inhibitor, showed a reduction in free C5 of greater than 99% within 24 hours at a 100 mg dose during the Phase 1 study[90]. Capital Requirements - The company anticipates requiring significant additional capital to fund operations and product development, which may lead to dilution for existing shareholders[9]. - The company expects to raise substantial additional capital to fund the development and commercialization of its product candidates, if approved[24]. - The company expects to finance operations through equity sales, debt financing, and collaborations, with no current credit facility or committed sources of capital[132]. Joint Ventures and Collaborations - The company funded $2.0 million for its 50% interest in the joint venture RE Ventures I, LLC for the nine months ended September 30, 2024, up from $1.5 million in the same period in 2023[62]. - The company recorded its allocable share of losses from the joint venture totaling $1.8 million for the nine months ended September 30, 2024, compared to $1.4 million for the same period in 2023[63]. - The company entered into a collaboration agreement with Johnson & Johnson, receiving an upfront payment of $0.5 million and is eligible for up to $0.7 million based on enrollment-related events[70][73]. Marketable Securities - As of September 30, 2024, the fair value of marketable securities was $68.698 million, compared to $101.963 million as of December 31, 2023, indicating a decrease of approximately 32.5%[41]. - The aggregate fair value of available-for-sale debt securities in an unrealized loss position was $2.0 million as of September 30, 2024, down from $40.0 million as of December 31, 2023[42]. Accounting and Reporting - The Company recognized revenue in accordance with ASC 606, reflecting the consideration expected to be received for goods and services[30]. - The company is currently evaluating the impact of recently issued accounting standards on its consolidated financial statements[36].

Revenue Performance - Revenue for Q3 2024 was $0.3 million, compared to no revenue in Q3 2023, attributed to a collaboration agreement with Johnson & Johnson[14] - Total revenue for the three months ended September 30, 2024, was $299,000, compared to $0 for the same period in 2023[21] Expenses - R&D expenses decreased to $8.2 million in Q3 2024 from $13.3 million in Q3 2023, primarily due to reduced development costs for RLYB116 and RLYB212[15] - G&A expenses were $4.1 million in Q3 2024, down from $6.1 million in Q3 2023, mainly due to lower payroll and consulting fees[16] - Total operating expenses decreased to $12,365,000 for the three months ended September 30, 2024, from $19,363,000 in the same period of 2023, representing a reduction of approximately 36.5%[21] - Research and development expenses for the three months ended September 30, 2024, were $8,240,000, compared to $13,288,000 for the same period in 2023, reflecting a decrease of approximately 38.5%[21] - General and administrative expenses were $4,125,000 for the three months ended September 30, 2024, down from $6,075,000 in the same period of 2023, a reduction of approximately 32.2%[21] Net Loss - Net loss for Q3 2024 was $11.5 million, or $0.26 per share, compared to a net loss of $18.4 million, or $0.45 per share, in Q3 2023[17] - Net loss for the three months ended September 30, 2024, was $11,466,000, compared to a net loss of $18,374,000 for the same period in 2023, indicating an improvement of approximately 37.5%[21] Cash and Assets - As of September 30, 2024, cash, cash equivalents, and marketable securities totaled $75.1 million, providing a runway into mid-2026[17] - Cash, cash equivalents, and marketable securities totaled $75,139,000 as of September 30, 2024, down from $109,929,000 at the end of December 31, 2023[21] - Total assets decreased to $79,007,000 as of September 30, 2024, from $115,620,000 at December 31, 2023[21] - Total liabilities as of September 30, 2024, were $8,189,000, down from $9,436,000 at December 31, 2023[21] Clinical Trials and Research - RLYB212 Phase 2 trial is set to initiate screening in Q4 2024 after receiving CTA approvals from EMA and MHRA[3] - Over 13,000 pregnant women have been screened in the FNAIT natural history study as of November 1, 2024[4] - RLYB116 manufacturing enhancements completed in Q3 2024 are expected to improve tolerability[9] - More than 30,000 pregnancies annually in North America and major European countries are estimated to be at higher risk for FNAIT[8] Future Outlook - Rallybio aims to deliver multiple key value inflection points in 2025 through focused investments in its pipeline[2] - Weighted-average common shares outstanding increased to 44,593,221 for the three months ended September 30, 2024, compared to 40,531,497 for the same period in 2023[21] - The company reported a net unrealized gain on marketable securities of $240,000 for the three months ended September 30, 2024, compared to a gain of $64,000 for the same period in 2023[21]

Core Viewpoint - Rallybio Corporation (RLYB) has received an upgrade to a Zacks Rank 2 (Buy), indicating a positive trend in earnings estimates which is a significant factor influencing stock prices [1][3]. Earnings Estimates and Stock Price Impact - The Zacks rating system emphasizes the importance of earnings estimate revisions, which have shown a strong correlation with near-term stock price movements [4][6]. - For Rallybio, the increase in earnings estimates suggests an improvement in the company's underlying business, likely leading to a rise in stock price [5]. Earnings Estimate Revisions - For the fiscal year ending December 2024, Rallybio is expected to earn -$1.35 per share, reflecting a 26.6% change from the previous year's reported figure [8]. - Over the past three months, the Zacks Consensus Estimate for Rallybio has increased by 4.9%, indicating a positive trend in analyst expectations [8]. Zacks Rank System - The Zacks Rank system classifies stocks based on earnings estimate revisions, with only the top 20% of stocks receiving a 'Strong Buy' or 'Buy' rating, highlighting their potential for market-beating returns [9][10]. - The upgrade of Rallybio to a Zacks Rank 2 places it in the top 20% of Zacks-covered stocks, suggesting a favorable outlook for the stock in the near term [10].

Financial Performance - Total revenue for the three months ended June 30, 2024, was $299,000, compared to $0 for the same period in 2023[13]. - The company reported a comprehensive loss of $16,236,000 for the three months ended June 30, 2024[13]. - Net loss for the three months ended June 30, 2024, was $16,236,000, compared to a net loss of $18,630,000 for the same period in 2023[13]. - The net loss for the six months ended June 30, 2024, was $35,265 thousand, compared to a net loss of $35,948 thousand for the same period in 2023, representing a decrease of 1.9%[17]. - The company incurred net losses of $16.2 million and $35.3 million for the three and six months ended June 30, 2024, respectively[85]. Assets and Liabilities - As of June 30, 2024, total current assets decreased to $91.436 million from $114.789 million as of December 31, 2023, representing a decline of approximately 20.3%[11]. - Cash and cash equivalents decreased to $16.671 million from $24.494 million, a reduction of about 32.0%[11]. - Total liabilities rose to $12.414 million from $9.436 million, reflecting an increase of about 31.3%[11]. - The balance of stockholders' equity as of June 30, 2024, was $80,017,000, compared to $139,257,000 as of June 30, 2023[15]. - As of June 30, 2024, the company had cash, cash equivalents, and marketable securities totaling $88.6 million, which is expected to fund operations for more than 12 months[20]. Research and Development - Research and development expenses for the three months ended June 30, 2024, were $12,946,000, a decrease from $13,130,000 in the previous quarter[13]. - Total research and development expenses for the six months ended June 30, 2024, were $25.9 million, an increase of $1.6 million from $24.3 million in the same period of 2023[106]. - RLYB212 and RLYB116 are heavily dependent on successful early-stage clinical development, with potential delays posing risks to the business[7]. - The company plans to initiate a Phase 2 clinical trial of RLYB212 in the fourth quarter of 2024, following the completion of Phase 1 studies for both RLYB212 and RLYB116[19]. - RLYB212 achieved proof-of-concept in the Phase 1b study, demonstrating a ≥ 90% reduction in mean platelet elimination half-life compared to placebo[70]. Capital and Financing - The company anticipates requiring significant additional capital to fund operations and product development, which may lead to dilution for existing shareholders[7]. - The company raised $5,405 thousand from the issuance of common stock from a securities purchase agreement during the six months ended June 30, 2024[17]. - The company raised approximately $6.6 million from the sale of 3,636,363 shares of common stock at $1.82 per share in April 2024, representing a 10% premium on the closing stock price[37]. - The company does not anticipate its current cash and marketable securities will be sufficient to fund product candidates through regulatory approval, indicating a need for substantial additional capital[20]. Operating Expenses - Total operating expenses for the three months ended June 30, 2024, were $17,334,000, compared to $20,083,000 for the same period in 2023[13]. - General and administrative expenses for the three months ended June 30, 2024, were $4,388,000, down from $6,953,000 in the previous quarter[13]. - Loss from operations improved to $(17.0) million in Q2 2024 from $(20.1) million in Q2 2023, an improvement of $3.0 million[97]. - General and administrative expenses decreased to $11.2 million for the six months ended June 30, 2024, down from $14.1 million in 2023, a reduction of $2.9 million[107]. Joint Ventures and Collaborations - The company entered into a Collaboration and License Agreement with Johnson & Johnson, receiving an upfront payment of $0.5 million and potential additional payments totaling up to $0.7 million[61]. - The carrying value of the investment in the joint venture as of June 30, 2024, was $0.6 million, reflecting the company's maximum exposure to risk[52]. - The company's allocable share of losses from the joint venture for the three months ended June 30, 2024, was $0.5 million, an increase from $0.2 million in the same period of 2023[52]. Market and Competition - The company faces significant competition from other biotechnology and pharmaceutical companies, which could adversely affect its operating results[8]. - The regulatory approval processes for product candidates are lengthy and unpredictable, posing a risk to the company's business if approvals are not obtained[7]. Stock and Shareholder Information - Weighted-average common shares outstanding for the three months ended June 30, 2024, were 44,128,059, compared to 40,363,902 for the same period in 2023[13]. - The total number of shares available for future issuance under the 2021 Employee Stock Purchase Plan (ESPP) was 834,589 as of June 30, 2024[49]. - The company had unrecognized share-based compensation expense related to unvested stock options of $11.1 million as of June 30, 2024, expected to be recognized over approximately 2.4 years[45].

Financial Performance - Rallybio reported revenue of $0.3 million for Q2 2024, compared to no revenue in the same period in 2023, attributed to a collaboration with Johnson & Johnson[8] - Total revenue for the three months ended June 30, 2024, was $299,000, compared to $0 for the same period in 2023[14] - Rallybio reported a net loss of $16.2 million, or $0.37 per common share, for Q2 2024, compared to a net loss of $18.6 million, or $0.46 per common share, in Q2 2023[10] - Net loss for the three months ended June 30, 2024, was $16,236,000, compared to a net loss of $18,630,000 for the same period in 2023, reflecting an improvement of approximately 12.8%[14] - Net loss per common share for the three months ended June 30, 2024, was $(0.37), compared to $(0.46) for the same period in 2023[14] - Interest income for the three months ended June 30, 2024, was $1,143,000, compared to $1,608,000 for the same period in 2023, a decrease of approximately 29%[14] Expenses - Research and Development (R&D) expenses were $12.9 million for Q2 2024, a slight decrease from $13.1 million in Q2 2023, primarily due to reduced payroll costs[9] - Research and development expenses decreased to $12,946,000 for the three months ended June 30, 2024, from $13,130,000 in the same period of 2023, representing a reduction of approximately 1.4%[14] - General and Administrative (G&A) expenses decreased to $4.4 million in Q2 2024 from $7.0 million in Q2 2023, mainly due to lower payroll and consulting fees[10] - General and administrative expenses decreased to $4,388,000 for the three months ended June 30, 2024, from $6,953,000 in the same period of 2023, a decrease of approximately 37%[14] Cash and Assets - As of June 30, 2024, Rallybio had cash, cash equivalents, and marketable securities totaling $88.6 million, providing a runway into mid-2026[1] - Cash, cash equivalents, and marketable securities totaled $88,614,000 as of June 30, 2024, down from $109,929,000 as of December 31, 2023[14] - Total assets decreased to $92,431,000 as of June 30, 2024, from $115,620,000 as of December 31, 2023[14] - Total stockholders' equity decreased to $80,017,000 as of June 30, 2024, from $106,184,000 as of December 31, 2023[14] Liabilities - Total liabilities increased to $12,414,000 as of June 30, 2024, compared to $9,436,000 as of December 31, 2023[14] Clinical Development - The company is on track to initiate a Phase 2 trial for RLYB212 in Q4 2024, targeting pregnant women at higher risk for fetal and neonatal alloimmune thrombocytopenia (FNAIT)[3] - An epidemiological analysis indicated that over 30,000 pregnancies each year in key geographies are at higher risk for FNAIT, representing a 40% increase from prior estimates[4] - RLYB116 manufacturing is progressing and expected to be completed in Q3 2024, with additional biomarker analysis data anticipated in Q4 2024[6] - The company is advancing preclinical programs and seeking partnerships and non-dilutive financing options to support these initiatives[7] Collaboration - Rallybio's collaboration with Johnson & Johnson included a $6.6 million equity investment and a $0.5 million upfront payment related to the collaboration[2]

Financial Performance - The company experienced a net loss of $19.029 million for the three months ended March 31, 2024, compared to a net loss of $17.318 million for the same period in 2023, representing an increase in loss of 9.9%[20]. - The company reported total operating expenses of $19.787 million for Q1 2024, an increase of 7.7% compared to $18.374 million in Q1 2023[20]. - The company incurred a loss on investment in joint ventures of $685,000 in Q1 2024, compared to a loss of $563,000 in Q1 2023, marking a 21.7% increase in losses[20]. - The company reported a net loss of $19.0 million for the three months ended March 31, 2024, compared to a net loss of $17.3 million for the same period in 2023, representing an increase in loss of $1.7 million[97]. - The company has an accumulated deficit of $254.3 million as of March 31, 2024, reflecting significant operating losses since inception[83]. - The company has incurred substantial net operating losses (NOLs) and may face limitations on using these to offset future taxable income due to ownership changes[153]. Assets and Equity - As of March 31, 2024, the company reported total assets of $99.359 million, a decrease of 14.1% from $115.620 million as of December 31, 2023[18]. - Total stockholders' equity decreased to $89.157 million as of March 31, 2024, down from $106.184 million as of December 31, 2023, reflecting a decline of 16.1%[18]. - As of March 31, 2024, the fair value of marketable securities was $85,001 thousand, down from $101,963 thousand as of December 31, 2023, representing a decrease of approximately 16.6%[36]. - The aggregate fair value of available-for-sale debt securities in an unrealized loss position increased to $58.4 million as of March 31, 2024, compared to $40.0 million as of December 31, 2023[36]. - The company had cash, cash equivalents, and marketable securities totaling $94.2 million as of March 31, 2024, which is expected to fund operations for more than 12 months[29]. Research and Development - Research and development expenses increased to $12.936 million in Q1 2024, up 15.4% from $11.202 million in Q1 2023[20]. - The company plans to initiate a Phase 2 clinical trial of RLYB212 in the second half of 2024, following the completion of Phase 1 studies for both RLYB212 and RLYB116[28]. - RLYB116, a novel C5 inhibitor, demonstrated a greater than 99% reduction in free C5 levels within 24 hours of a single 100 mg dose in Phase 1 studies, indicating its potential for treating complement-related diseases[72]. - The company is advancing RLYB332, a long-acting version of RLYB331, with favorable nonclinical study results expected to be presented in the second half of 2024[76]. - The company has screened approximately 10,000 pregnant women for its FNAIT natural history study, aiming to screen up to 30,000 women to determine the frequency of higher FNAIT risk[69]. Capital and Funding - The company expects to raise substantial additional capital to fund the development and commercialization of its product candidates, if approved[29]. - The company completed a follow-on offering in November 2022, raising approximately $54.8 million, with net proceeds of about $50.8 million after costs[81]. - The company sold 3,636,363 shares of common stock to Johnson & Johnson Innovation at a price of $1.82 per share, raising approximately $6.6 million in gross proceeds[61]. - The company expects existing cash and marketable securities to fund operations into mid-2026, although this is subject to various risks and uncertainties[108]. - The company requires significant additional capital for the development and potential commercialization of product candidates, which may be raised through various means including equity offerings and collaborations[144]. Clinical Trials and Regulatory Risks - The company’s lead product candidates, RLYB212 and RLYB116, are still in early clinical development and require successful completion of registrational trials for regulatory approval[169]. - The regulatory approval processes for product candidates are lengthy and unpredictable, potentially taking many years[185]. - The FDA may require additional clinical trials or impose restrictions on marketing approvals, impacting the company's ability to commercialize products[187]. - The company may face significant delays or inability to commercialize product candidates if it does not achieve timely regulatory approvals and successful clinical trials[159]. - The company may need to identify additional formulations or routes of administration for its product candidates, which could delay clinical trials and increase costs[157]. Market Competition and Challenges - The biotechnology and pharmaceutical industries are highly competitive, with many companies engaged in similar research and development, potentially impacting the company's market position[198]. - RLYB116 faces competition from established treatments such as Soliris and Ultomiris, which may affect its market entry and acceptance[199]. - The company has no product candidates approved for sale in any jurisdiction, including international markets, which poses a significant risk to market entry[205]. - The potential market opportunities for the company's product candidates may be smaller than anticipated due to the rarity of targeted diseases[193]. - The company has not commercialized any products and has never generated revenue from product sales[136].

Financial Performance - Rallybio reported a net loss of $19.0 million, or $0.47 per common share, for Q1 2024, compared to a net loss of $17.3 million, or $0.43 per common share, in Q1 2023[13]. - R&D expenses for Q1 2024 were $12.9 million, up from $11.2 million in Q1 2023, primarily due to increased payroll and severance costs[10]. - General and administrative expenses decreased to $6.9 million in Q1 2024 from $7.2 million in Q1 2023, attributed to lower administrative costs[10]. Cash Position - As of March 31, 2024, Rallybio had $94.2 million in cash, cash equivalents, and marketable securities, providing a runway into mid-2026[1]. Clinical Development - The company is on track to initiate a Phase 2 trial for RLYB212 in pregnant women at higher risk for FNAIT in the second half of 2024[4]. - Approximately 10,000 pregnant women have been screened in the FNAIT natural history study as of May 1, 2024, which aims to provide a contemporary dataset for HPA-1a alloimmunization frequency[9]. - Rallybio expects to disclose data from an epidemiologic analysis on FNAIT risk in mid-2024, which will help identify a larger market opportunity for RLYB212[2]. Strategic Initiatives - Rallybio announced a collaboration with Johnson & Johnson, receiving a $6.6 million equity investment to advance therapeutic solutions for FNAIT[5]. - The company is prioritizing its portfolio and has implemented a 45% workforce reduction to focus on its two Phase 2 ready assets, RLYB212 and RLYB116[5]. - RLYB116 manufacturing is on track for completion in the second half of 2024, with additional biomarker development work ongoing[6].

Drug Development and Clinical Trials - The company has developed RLYB212 for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT), with over 22,000 high-risk pregnancies estimated annually in the U.S., Canada, the UK, and Australia[28]. - RLYB212 has completed two Phase 1 clinical trials, with preliminary results indicating a well-tolerated profile and a potential once-monthly dosing regimen for the upcoming Phase 2 trial[29]. - The Phase 1b trial of RLYB212 demonstrated a dose-dependent reduction in mean platelet elimination half-life, achieving a reduction of 90% or more in HPA-1a positive platelets[30]. - The company plans to initiate a Phase 2 clinical trial for RLYB212 in the second half of 2024, following the completion of Phase 1 trials[29]. - The FNAIT natural history study aims to screen up to 30,000 expectant mothers to gather data for future clinical trials, with approximately 9,400 women screened as of March 1, 2024[31]. - RLYB116, an inhibitor of complement component 5 (C5), has shown over 99% reduction in free C5 within 24 hours of a single 100 mg subcutaneous injection in Phase 1 trials[34]. - The multiple ascending dose (MAD) portion of the RLYB116 trial indicated sustained mean reductions in free C5 of greater than 93% after a once-weekly 100 mg dose[36]. - RLYB331, a preclinical monoclonal antibody targeting Matriptase-2, aims to address severe anemia and iron overload, with data expected in the first half of 2024[38]. - The company plans to initiate a Phase 2 trial of RLYB212 in expectant mothers at higher FNAIT risk in the second half of 2024, focusing on PK and safety assessments[72]. - The Phase 1 clinical trial for RLYB116 included a single-blind, placebo-controlled design with doses ranging from 2mg to 300mg, enrolling 8 subjects per cohort[90]. Market Opportunities and Collaborations - The market opportunity for RLYB212 is estimated to exceed $1 billion, targeting approximately 22,000 high-risk pregnancies annually in the U.S., Canada, the UK, and major European countries[53][54]. - Approximately 2% of the Caucasian population is HPA-1a negative, leading to an estimated 110,000 HPA-1a negative expectant mothers annually in the targeted regions[54]. - The collaboration with AbCellera aims to co-develop up to five rare disease therapeutic targets, enhancing Rallybio's product pipeline[42][43]. - The joint venture is developing a small molecule ENPP1 inhibitor for the treatment of Hypophosphatasia (HPP), a rare genetic disease with an incidence of 1 in 100,000 to 1 in 300,000 in the U.S. and Canada for severe cases[40]. - The partnership with Exscientia is focused on discovering small molecules for rare metabolic diseases, initially targeting ENPP1 for treating hypophosphatasia (HPP)[103][104]. Intellectual Property and Regulatory Compliance - As of March 1, 2024, the company owns two patent families related to its FNAIT prevention program, with patents expiring in 2035 and 2026, and one U.S. patent expiring in November 2030 due to a PTA granted by the USPTO[130]. - The company has a multi-layered approach to securing intellectual property rights, including acquiring rights through purchase or exclusive license and filing patent applications[123]. - The company is managing prosecution of a patent family relating to RLYB331 and has pending applications in over 20 countries, including the U.S. and China[133]. - The company intends to pursue relevant marketing exclusivities in the U.S. and foreign countries for its candidate products, although there is no certainty that such exclusivities will be granted[128]. - The company has confidence in its protective measures for trade secrets, but acknowledges that breaches can occur and remedies may be inadequate[129]. - The FDA application fee for an NDA or BLA in fiscal year 2024 is approximately $4.0 million, with an annual program fee exceeding $415,000 per program[185]. - The FDA may issue a Complete Response Letter (CRL) if the application does not meet regulatory criteria, outlining deficiencies that must be addressed for reconsideration[192]. - The FDA may impose post-approval requirements, including Phase 4 clinical trials to further assess safety[193]. - The FDA may designate certain products for expedited development if they address unmet medical needs in serious conditions[198]. Manufacturing and Development Strategy - The company is prioritizing enhancements to the manufacturing process of RLYB116, expected to be completed in the second half of 2024, to improve tolerability and expand treatment options[36]. - The company currently relies on third-party contract manufacturers for the manufacture of product candidates for preclinical and clinical testing[163]. - The company expects to continue developing product candidates that can be produced cost-effectively at contract manufacturing facilities[167]. - The company is considering partnerships or non-dilutive financing to support future clinical development of RLYB116[76]. - The company has acquired rights to certain C5 inhibitor compounds from Sobi, with an upfront payment of $5.0 million and potential milestone payments totaling up to $116.0 million[145]. Safety and Efficacy Data - RLYB212 demonstrated a dose-dependent, rapid, and complete elimination of transfused HPA-1a positive platelets, achieving a ≥90% reduction in mean platelet elimination half-life, with mean values of 5.8 hours (0.09mg dose) and 1.5 hours (0.29mg dose) compared to 71.7 hours for placebo[70]. - RLYB116 demonstrated a reduction in free C5 greater than 99% within 24 hours after a single 100 mg dose, with sustained mean reductions of over 93% at day 29[92][95]. - The mean elimination half-life of RLYB116 was greater than 300 hours, indicating low inter-subject variability and consistent increases in exposure relative to dose[92][96]. - The MAD portion of the Phase 1 trial included four dosing cohorts, with mild to moderate adverse events reported, primarily gastrointestinal in nature[93][96]. - RLYB116 aims to provide a more accessible treatment option for patients with gMG, addressing significant unmet needs in the market[82].