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1. Rallybio is like an early-stage Alexion Pharmaceuticals, developing therapies for severe and rare diseases, including complement-mediated disorders.

2. Rallybio is similar to a startup BioMarin Pharmaceutical, focusing on highly unmet medical needs in rare disease populations.

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• RLYB212: An investigational antibody designed to prevent Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT).
• RLYB116: An investigational anti-C5 antibody for the treatment of severe complement-mediated diseases, such as Generalized Myasthenia Gravis.
• RLYB331: A preclinical investigational Noggin-Fc fusion protein being developed for disorders of impaired bone formation, like Hypophosphatasia.

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Rallybio (RLYB) is a clinical-stage biotechnology company focused on identifying and accelerating the development of life-changing therapies for patients with severe and rare diseases.

As of its current stage, Rallybio does not have any approved commercial products available for sale. Consequently, the company does not currently have major customers generating product revenue.

The company's financial activities primarily involve research and development expenses, supported by capital raises and, at times, collaboration revenue from partnerships related to its development pipeline. This revenue typically comes from other pharmaceutical or biotechnology companies funding specific research or development milestones, rather than from product sales to end-users or healthcare providers.

Should any of Rallybio's product candidates successfully complete clinical trials and receive regulatory approval in the future, its potential customers would likely be institutional entities within the healthcare sector, such as hospitals, specialized clinics, or other pharmaceutical companies (through licensing or acquisition agreements), rather than direct sales to individuals.

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The key risks to Rallybio's business include:

1. Clinical Trial Outcomes and Regulatory Approvals: As a clinical-stage biotechnology company, Rallybio's success is highly dependent on the successful outcome of its ongoing and future clinical trials and the subsequent receipt of regulatory approvals. The company recently discontinued its RLYB212 program due to the inability of the dose regimen to achieve predicted target concentrations for efficacy in its Phase 2 clinical trial. This demonstrates the significant impact that unfavorable clinical trial results can have on the company's programs and future prospects.
2. Financial Resources and Ability to Raise Capital: Rallybio's operations and continued development of its pipeline candidates require substantial financial resources. The company's ability to fund its operating expenses and capital expenditure requirements, as well as its success in raising additional capital, are significant risks. Recent challenges include a potential Nasdaq delisting due to not meeting the minimum bid price and a reduction in a common stock offering.
3. Competition: Rallybio operates in a highly competitive biotechnology and pharmaceutical industry. Competition from other companies developing therapies for severe and rare diseases could impact the success and market potential of Rallybio's product candidates.

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The launch and advanced clinical development of broader-mechanism therapies by larger pharmaceutical companies for conditions that Rallybio is targeting. For instance, Janssen's Nipocalimab, an FcRn inhibitor, is in Phase 2 clinical trials for Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) and Phase 3 for Hemolytic Disease of the Fetus and Newborn (HDFN), conditions for which Rallybio's RLYB212 (an anti-HPA-1a antibody) is being developed. Nipocalimab's broad mechanism to reduce maternal IgG antibodies and its advanced stage of development represent a significant emerging threat that could establish a dominant treatment paradigm and limit RLYB212's market potential.

The rapid shift in the standard of care for complement-mediated diseases with the successful development and commercialization of complement inhibitors targeting pathways other than C5. Rallybio's RLYB116 is a C5 inhibitor, but companies like Apellis Pharmaceuticals (Empaveli, a C3 inhibitor) and Novartis (Fabhalta, a Factor B inhibitor) have introduced or are advancing therapies with novel mechanisms. This diversification of successful therapeutic approaches in a crowded market means that a new C5 inhibitor will face substantial challenges in differentiation and market capture, as the therapeutic landscape is evolving beyond just C5 inhibition.

The highly competitive and rapidly maturing therapeutic landscape for IgA Nephropathy. Rallybio's RLYB111 (a C4S inhibitor) is entering a market that has recently seen the approval and late-stage development of multiple therapies from competitors, including Travere Therapeutics (Filspari), Calliditas Therapeutics (Tarpeyo), and Novartis (Iptacopan, Atrasentan). These products target various pathways and have already begun to establish market share, creating a significant barrier for a new entrant like RLYB111 to differentiate itself and gain meaningful traction in an increasingly crowded treatment paradigm.

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Rallybio (RLYB) is developing several product candidates for rare diseases. The addressable markets for their main products are as follows:

• RLYB212 (Fetal and Neonatal Alloimmune Thrombocytopenia - FNAIT): Rallybio estimates the commercial opportunity for RLYB212 for the prevention of FNAIT to be greater than $1.6 billion. This market size is global, based on an epidemiological analysis across diverse ancestries, including the United States, Canada, the United Kingdom, other major European countries, and Australia.
• RLYB116 (Complement Dysregulation - Immune Platelet Transfusion Refractoriness (PTR) and refractory Antiphospholipid Syndrome (APS)): Rallybio announced that PTR and refractory APS represent a combined market opportunity of $5 billion. In addition, RLYB116 is also being developed for other complement-mediated diseases:
  • Paroxysmal Nocturnal Hemoglobinuria (PNH): The global paroxysmal nocturnal hemoglobinuria treatment market size was estimated at USD 5.75 billion in 2024 and is projected to reach USD 9.96 billion by 2030.
  • Antiphospholipid Syndrome (APS): The global antiphospholipid syndrome market reached US$ 32.87 billion in 2023 and is expected to reach US$ 68.43 billion by 2031. The global catastrophic antiphospholipid syndrome market was estimated at USD 4.7 billion in 2024 and is expected to reach USD 12.9 billion by 2034.
  • Generalized Myasthenia Gravis (gMG): The global market for generalized myasthenia gravis drugs is projected to reach $10.2 billion by 2033.
• REV102 (Hypophosphatasia - HPP): The global hypophosphatasia treatment market size was valued at USD 1.34 billion in 2024 and is projected to reach USD 1.89 billion by 2032.

Market sizes for RLYB332 (diseases of iron overload and severe anemias) and RLYB114 (ophthalmological diseases of complement dysregulation, such as age-related macular degeneration) were not explicitly identified in the search results with specific addressable market figures for Rallybio's products, beyond stating the general disease areas they target. Therefore, the market size for these products cannot be provided at this time.

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1. Successful Clinical Development and Commercialization of RLYB116: Rallybio's lead program, RLYB116, a differentiated C5 inhibitor, is central to its future revenue prospects. The company is advancing RLYB116 through clinical trials, with data from a confirmatory Phase 1 study anticipated in the fourth quarter of 2025. Rallybio views RLYB116 as a potential best-in-class therapeutic capable of addressing multiple "billion-dollar market opportunities" in complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), generalized myasthenia gravis (gMG), and antiphospholipid syndrome (APS). Positive clinical outcomes and subsequent regulatory approvals would be significant drivers of revenue growth.
2. Advancement and Potential Partnerships for Other Pipeline Candidates: Beyond RLYB116, Rallybio has other promising candidates in its pipeline, such as RLYB332, a matriptase-2 inhibitor for diseases of iron overload and severe anemias, which has shown superior preclinical data. Successful progression of these preclinical programs into clinical development, and potentially into strategic partnerships or licensing agreements, could generate future milestone payments and royalty revenue.
3. Revenue from Collaboration Agreements and Milestone Payments: Rallybio has a history of generating revenue through collaboration agreements, such as its agreement with Johnson & Johnson in 2024. More recently, the company generated $20 million from the sale of its interest in REV102 to Recursion Pharmaceuticals, which included an upfront payment and a contingent equity payment. Rallybio is also eligible for future milestone payments and low single-digit royalties on net sales from the REV102 program. Continuing to secure and execute on such collaborations, as well as achieving milestones in existing partnerships, will contribute to future revenue growth.

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