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].

Financial Performance - As of September 30, 2023, total assets decreased to $133.194 million from $180.435 million as of December 31, 2022, representing a decline of approximately 26.2%[17] - Cash and cash equivalents decreased to $19.158 million from $56.958 million, a reduction of about 66.4%[17] - Total operating expenses for the three months ended September 30, 2023, were $19.363 million, compared to $18.860 million for the same period in 2022, reflecting an increase of 2.7%[19] - The net loss for the three months ended September 30, 2023, was $18.374 million, slightly higher than the net loss of $18.370 million for the same period in 2022[19] - The company reported a net loss per common share of $0.45 for the three months ended September 30, 2023, compared to $0.60 for the same period in 2022[19] - Total stockholders' equity decreased to $123.581 million as of September 30, 2023, down from $169.317 million as of December 31, 2022, a decline of approximately 27.0%[17] - The company reported a comprehensive loss of $18.310 million for the three months ended September 30, 2023, compared to a comprehensive loss of $18.433 million for the same period in 2022[19] - For the nine months ended September 30, 2023, the net loss was $54,322 thousand, compared to a net loss of $50,451 thousand for the same period in 2022, representing an increase in loss of approximately 3.4%[27] Cash Flow and Liquidity - Cash and cash equivalents, along with marketable securities, totaled $121.4 million as of September 30, 2023, which is expected to fund operating expenses for more than 12 months[30] - The net cash used in operating activities for the nine months ended September 30, 2023, was $48,620 thousand, an increase from $42,020 thousand in the same period of 2022[27] - The company reported net cash provided by investing activities of $10,749 thousand for the nine months ended September 30, 2023, compared to a net cash used of $81,524 thousand in the same period of 2022[27] - The company reported a net decrease in cash and cash equivalents of $37,800 thousand for the nine months ended September 30, 2023, compared to a decrease of $123,623 thousand in the same period of 2022[27] - The company expects existing cash and marketable securities to fund operations into Q1 2025, but anticipates significant expenses and operating losses as product candidates advance through clinical development[103][104] Research and Development - Research and development expenses increased to $13.288 million for the three months ended September 30, 2023, from $12.110 million in the same period of 2022, marking an increase of 9.7%[19] - The company plans to increase research and development expenses significantly as it continues to develop product candidates and conduct clinical trials[82] - Research and development expenses for Q3 2023 were $13.3 million, an increase of $1.2 million (9.7%) compared to $12.1 million in Q3 2022[89] - For the nine months ended September 30, 2023, research and development expenses totaled $37.6 million, a significant increase of $7.7 million (25.7%) from $29.9 million in the same period of 2022[94] Clinical Development - The company has two clinical stage programs: RLYB212 and RLYB116, targeting severe and rare diseases[29] - RLYB212, an anti-HPA-1a antibody, achieved proof-of-concept in a Phase 1b study, demonstrating a ≥ 90% reduction in mean platelet elimination half-life compared to placebo[59] - The company expects to initiate a Phase 2 dose confirmation study for RLYB212 in the second half of 2024, targeting pregnant women at higher risk of FNAIT[61] - RLYB116, a novel C5 inhibitor, is currently in a Phase 1 multiple ascending dose study, with initial data expected to be reported in Q4 2023[65] - Clinical data from the Phase 1 study of RLYB116 showed that doses of 100 mg and 300 mg resulted in over 99% reductions in free C5 concentrations[66] Capital Requirements and Future Funding - The company expects to raise substantial additional capital to fund the development and commercialization of its product candidates, if approved[30] - The company will require significant additional capital to fund operations and may raise funds through equity offerings, debt financings, and collaborations[139] - The company anticipates incurring substantial additional costs associated with regulatory filings and marketing approvals once product candidates complete development[107] Regulatory and Market Challenges - The regulatory approval process for product candidates is lengthy and unpredictable, with no guarantee of success[189] - The company has never obtained marketing approval for any product candidate, which could significantly harm its business and profitability[196] - The company may face challenges transitioning from a research and development focus to supporting commercial activities[144] - The company may face various regulatory actions, including fines or market withdrawals, if it fails to comply with regulatory requirements[215] Competition and Market Position - The company faces significant competition from biotechnology and pharmaceutical companies, which could adversely affect its operating results if it fails to compete effectively[203] - RLYB116 faces competition from established treatments such as Soliris and Ultomiris marketed by AstraZeneca for conditions like PNH and gMG[203] - The company believes RLYB212 could be a first-in-class antibody for the prevention of FNAIT, with no direct mechanistic-based clinical competition currently existing[203] Operational Risks - The ongoing COVID-19 pandemic may cause delays and disruptions to preclinical studies and clinical trials, potentially impacting the company's ability to obtain regulatory approvals[160] - The company has encountered limited disruptions to its manufacturing and supply chain due to COVID-19, but significant disruptions could materially impact its operations[162] - Patient enrollment for clinical trials is challenging, especially for rare diseases, which may lead to increased costs and delays in product development[179]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 _____________________________________ FORM 10-Q _____________________________________ (Mark One) x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 For the transition period from ______________ to ______________ Commission File Number: 001-40693 RALLYBIO CORPORATION (Exact Name of Registrant as Specified in its Charter) ____________________________________ ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________ to ______________ Commission File Number: 001-40693 RALLYBIO CORPORATION (Exact Name of Registrant as Specified in its Charter) Delaware 85-10837 ...

Gelsen et d., Rapick Ind Compiete Clearanta: Of HPA-ico Nismatched Platelets In A Human Model Of Fetal Ara Nescratol Allanmure in Tromborgtoperio By A Nyaerimmune Plasmo De International Society on Thrombosis and Hemostasis 2021 Congress, July 17-21, 2021. Abstract #PB0969 Study Design Prospective, non-interventional, multinational natural history study conducted at ~ 20 sites in US/EU ~26-30 Weeks | --- | --- | |-------------------------|-------| | | | | ■ C5 is a proven target | | 20 21 23 ENPP1: Target T ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 Delaware 85-1083789 (State or other jurisdiction of incorporation or organization) 234 Church Street, Suite 1020 New Haven, CT 06510 (Address of principal executive offices) (Zip Code) OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE T ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Delaware 85-1083789 (State or other jurisdiction of incorporation or organization) For the quarterly period ended September 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________ to ______________ Commission File Number: 001-40693 RALLYB ...