Rallybio Corporation, a clinical-stage biotechnology company, has been on a mission to develop and commercialize transformative therapies for patients with devastating rare diseases. Founded in 2018 by industry veterans with extensive experience in the rare disease space, Rallybio has built an impressive pipeline of promising product candidates aimed at addressing unmet medical needs in areas such as maternal-fetal health, complement dysregulation, hematology, and metabolic disorders.
Business Overview and History
Rallybio was established in January 2018 by Dr. Stephen Uden, Dr. Martin Mackay, and Dr. Jeffrey Fryer, all of whom previously held leadership roles at Alexion Pharmaceuticals, now part of AstraZeneca. The founding team's deep expertise in rare disease drug development and commercialization has been a key driver in Rallybio's swift progress over the past several years.
Since its inception, Rallybio has focused on raising capital to fund its operations and identifying, evaluating, and acquiring or in-licensing product candidates and technologies. The company received approximately $182.5 million in equity financing prior to its initial public offering (IPO) in August 2021, where it raised an additional $83 million. This was followed by a $50.8 million follow-on offering in 2022. These funds have enabled the company to advance its pipeline and establish key partnerships to further its research and development efforts.
Throughout its history, Rallybio has faced several challenges common to early-stage biotechnology companies. The company has had to carefully manage its financial resources to fund the development of its pipeline, and has had to navigate the complexities of clinical trial design and execution, regulatory interactions, and manufacturing scale-up. Despite these challenges, Rallybio has made steady progress in advancing its programs and building out its organization to support future growth.
Financials
Rallybio's financial position remains strong, with $75.1 million in cash, cash equivalents, and marketable securities as of September 30, 2024. This runway is expected to sustain the company's operations into the middle of 2026, providing ample resources to execute on its strategic priorities.
For the fiscal year 2023, Rallybio reported no revenue and a net loss of $74.6 million. The annual operating cash flow was negative $60.3 million, with a similar figure for annual free cash flow. In the third quarter of 2024, the company reported revenue of $299,000, primarily due to a collaboration agreement with Johnson & Johnson initiated in the second quarter of 2024. The net loss for Q3 2024 was $11.5 million, showing an improvement from the previous year, mainly due to decreased research and development expenses, particularly related to the RLYB116 program, and reduced general and administrative expenses.
The company's liquidity position is solid, with a debt-to-equity ratio of 0.003 as of September 30, 2024. Both the current ratio and quick ratio stand at 9.55, indicating strong short-term liquidity. Rallybio does not have any committed external sources of funds or credit facilities, relying primarily on its cash reserves and potential future financing activities to fund operations.
Pipeline and Product Candidates
Rallybio's pipeline is anchored by two lead programs, RLYB212 and RLYB116, both of which have completed Phase 1 clinical studies and are progressing through the development process.
RLYB212 is a monoclonal anti-HPA-1a antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT), a rare and potentially life-threatening hematological disorder. In the fourth quarter of 2024, Rallybio received approval from the European Medicines Agency (EMA) and the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 2 clinical trial of RLYB212 in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT. Screening for this trial is currently underway, with initial data from the second trimester expected in the second quarter of 2025 and data at the time of delivery anticipated in the third quarter of 2025. Both the FDA and EMA have granted RLYB212 orphan drug designation, highlighting its potential importance in addressing this rare condition.
RLYB116, Rallybio's second lead program, is a novel, potentially long-acting, subcutaneously injected inhibitor of complement component 5 (C5) that is being developed for the treatment of patients with complement-related diseases, such as paroxysmal nocturnal hemoglobinuria (PNH) and generalized myasthenia gravis (gMG). In December 2024, the company announced new biomarker characterization analyses and data from manufacturing process enhancements for RLYB116, indicating that the product candidate achieved complete and sustained complement inhibition in a previous Phase 1 multiple ascending dose (MAD) study. Specifically, a 100 mg once-weekly subcutaneous dose of RLYB116 achieved sustained mean reductions in free C5 of greater than 93%. Rallybio plans to initiate a confirmatory PK/PD study for RLYB116 in the second quarter of 2025, with data readouts expected in the second half of 2025.
In addition to its lead programs, Rallybio's pipeline includes several other promising product candidates targeting rare diseases in hematology, metabolic disorders, and complement dysregulation. One notable example is RLYB332, a long-acting version of an anti-Matriptase-2 (MTP-2) antibody, which the company obtained worldwide exclusive rights to in 2022. Preclinical data on RLYB332 were presented at the American Society of Hematology (ASH) Annual Meeting in December 2024, demonstrating favorable tolerability, dose-dependent pharmacokinetics, and sustained pharmacodynamic effects.
Partnerships and Collaborations
Rallybio has strategically partnered with several industry leaders to bolster its research and development capabilities. In 2022, the company entered into a multi-year, multi-target collaboration with AbCellera Biologics to discover and develop novel antibody-based therapeutics for rare diseases. Rallybio has also collaborated with Exscientia to develop small molecule therapeutics for rare diseases, and with EyePoint Pharmaceuticals to explore the use of Rallybio's C5 inhibitor in EyePoint's proprietary technology for sustained intraocular drug delivery.
In April 2024, Rallybio announced a two-year collaboration agreement with Johnson & Johnson, through its wholly-owned subsidiary Momenta Pharmaceuticals, to support the development of complementary therapeutic approaches aimed at reducing the risk of FNAIT. As part of this agreement, Rallybio received an upfront payment of $0.5 million and is eligible for additional payments upon the achievement of certain enrollment-related events and other triggers related to the FNAIT studies. This collaboration contributed to the company's revenue of $0.6 million for the nine months ended September 30, 2024.
Challenges and Resilience
Rallybio's journey has not been without its challenges. The biotechnology industry, particularly in the rare disease space, is highly competitive and subject to significant regulatory hurdles. The company has faced delays and setbacks, including the need to pause or modify certain development programs due to the COVID-19 pandemic and the broader economic uncertainty.
In February 2024, Rallybio announced a workforce reduction of approximately 45% in connection with a prioritization of its portfolio and cost savings plan to focus on its clinical assets. This move, while difficult, was necessary to ensure the efficient use of the company's resources and maintain its commitment to advancing its lead programs, RLYB212 and RLYB116.
Despite these challenges, Rallybio has demonstrated remarkable resilience. The company's management team has navigated the complexities of the rare disease landscape with agility, continuously evaluating and optimizing its pipeline to ensure the best allocation of capital and resources. Rallybio's strong financial position, with $75.1 million in cash, cash equivalents, and marketable securities as of September 30, 2024, provides a solid foundation to execute on its strategic priorities and weather any future storms.
Liquidity
Rallybio's liquidity position is robust, with $75.1 million in cash, cash equivalents, and marketable securities as of September 30, 2024. This financial cushion is expected to fund the company's operations into the middle of 2026, providing a stable foundation for advancing its clinical programs and strategic initiatives. The company's strong liquidity is further evidenced by its current ratio and quick ratio, both at 9.55, indicating a significant ability to meet short-term obligations.
Outlook and Catalysts
As Rallybio looks to the future, the company's focus remains on advancing its lead programs and expanding its pipeline of innovative therapies for rare diseases. The initiation of the Phase 2 trial for RLYB212 and the planned confirmatory PK/PD study for RLYB116 in the second quarter of 2025 represent significant milestones that could provide important data readouts and drive the company's progress.
Additionally, Rallybio's collaboration with Johnson & Johnson and its other strategic partnerships underscore the company's ability to leverage external expertise and resources to complement its internal capabilities. These collaborations not only provide non-dilutive funding but also have the potential to accelerate the development and commercialization of Rallybio's product candidates.
In the metabolic disorders space, Rallybio's candidate REV102, an ENPP1 inhibitor for the treatment of hypophosphatasia (HPP), is expected to enter IND-enabling studies in 2025. This program represents another promising opportunity for the company to address an area of high unmet medical need.
Rallybio's resilience and commitment to innovation have positioned the company as a promising player in the rare disease space. With a strong pipeline, strategic partnerships, and a seasoned management team, Rallybio is well-equipped to navigate the challenges and capitalize on the opportunities that lie ahead in its pursuit of transformative therapies for patients with devastating rare diseases.
As Rallybio continues to advance its clinical programs and expand its pipeline, investors and industry observers will be closely watching for key data readouts, regulatory milestones, and potential partnership announcements. The company's ability to execute on its strategic priorities while managing its cash runway will be critical in determining its long-term success in the competitive and complex landscape of rare disease drug development.