Rallybio Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Updates

-- Key Data Readouts from Sentinel Participant in RLYB212 Phase 2 Clinical Trial Expected in 2Q 2025 and 3Q 2025 --

-- Initiation of RLYB116 Confirmatory PK/PD Study Expected in 2Q 2025, with Data Anticipated in 2H 2025 --

-- $65.5 Million in Cash, Cash Equivalents, and Marketable Securities as of December 31, 2024 Provides Runway into 2H 2026 --

NEW HAVEN, Conn.--(BUSINESS WIRE)--March 13, 2025-- 

Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company translating scientific advances into transformative therapies for patients with devastating rare diseases, today reported financial results for the fourth quarter and full year ended December 31, 2024, and provided an update on recent company developments.

"We are pleased with our strong execution in 2024, and look forward to reporting on our planned milestones in 2025," said Stephen Uden, M.D., Chief Executive Officer of Rallybio. "Dosing in our RLYB212 Phase 2 trial is underway, our differentiated C5 inhibitor, RLYB116, is on track to enter a confirmatory pharmacokinetic/pharmacodynamic study in the second quarter, and our potentially best-in-class ENPP1 inhibitor for patients with hypophosphatasia, REV102, is advancing toward Phase 1 in 2026. Through exceptional execution of these programs and continued financial discipline, we are laser focused on driving value for Rallybio in 2025 and positioning the Company for sustained growth and success in the future."

Recent Business Highlights and Upcoming Milestones:

RLYB212 Program

   -- In February 2025, Rallybio announced that the sentinel participant was 
      dosed in the Phase 2 trial investigating RLYB212 in pregnant women at 
      higher risk for HPA-1a alloimmunization and fetal and neonatal alloimmune 
      thrombocytopenia (FNAIT). Pharmacokinetic $(PK)$ and safety data from the 
      second trimester are expected in the second quarter of 2025, with PK and 
      safety data at the time of delivery expected in the third quarter of 
      2025. The Company received regulatory approval to begin the Phase 2 trial 
      in October 2024 and initiated screening in November 2024. 
 
   -- More than 14,300 pregnant women were screened in Rallybio's FNAIT natural 
      history study as of January 31, 2025, at which time screening was 
      concluded at sites in the United States and Canada. Natural history data 
      will continue to be collected in a sub-study of the Phase 2 trial, where 
      participants at higher risk for HPA-1a alloimmunization and FNAIT who do 
      not receive RLYB212 are eligible to enroll. 
 
   -- Rallybio expects to present interim data from the FNAIT natural history 
      study in mid-2025, including data evaluating the frequency of FNAIT risk 
      across racial and ethnic populations. 

RLYB116 Program

   -- In December 2024, Rallybio presented biomarker characterization analyses 
      indicating that RLYB116 led to a meaningfully greater degree of 
      complement inhibition in the Phase 1 multiple ascending dose $(MAD.AU)$ study 
      than initially reported and as a result, may be effective in treating a 
      broad range of complement-mediated diseases, including paroxysmal 
      nocturnal hemoglobinuria (PNH), generalized myasthenia gravis (gMG), and 
      antiphospholipid syndrome (APS). 
 
   -- Rallybio plans to initiate a confirmatory clinical 
      pharmacokinetic/pharmacodynamic (PK/PD) study in the second quarter of 
      2025, with data readouts from Cohorts 1 and 2 expected in the third and 
      fourth quarter of 2025, respectively. 

REV102 Program

   -- Rallybio advanced REV102, an ENPP1 inhibitor for the treatment of 
      patients with hypophosphatasia $(HPP)$, which is being developed through a 
      joint venture with Recursion Pharmaceuticals. 
 
   -- Investigational new drug application $(IND.AU)$-enabling studies are underway 
      to support the initiation of a Phase 1 study in 2026. 
 
   -- Presentation of data evaluating REV102 in a preclinical model of 
      later-onset HPP is expected in the second half of 2025. 

RLYB332 Program

   -- In December 2024, Rallybio presented preclinical data for RLYB332 at the 
      American Society for Hematology $(ASH)$ annual meeting, which demonstrated 
      that single intravenous injections of RLYB332 to humanized FcRn mice had 
      rapid and sustained effects on PD parameters, including serum iron, 
      unsaturated iron binding capacity (UIBC), and transferrin saturation 
      $(TSAT)$, with greater impact than those produced by comparator molecules. 
 
   -- The favorable PD data relative to comparator molecules support RLYB332 as 
      a long-acting, potentially best-in-class therapy for the treatment of 
      diseases of iron overload. 

Fourth Quarter and Full Year 2024 Financial Results

   -- Revenue: Revenue was $38 thousand for the fourth quarter of 2024 and $0.6 
      million for the year ended December 31, 2024, compared to no revenue in 
      the same periods in 2023. The increase in revenue for both the fourth 
      quarter of 2024 and the year ended December 31, 2024 was related to 
      Rallybio's entrance into the collaboration agreement with Johnson & 
      Johnson in the second quarter of 2024 and the recognition of revenue 
      related to the collaboration's performance obligations. 
 
   -- Research & Development (R&D) Expenses: R&D expenses were $7.4 million for 
      the fourth quarter of 2024, compared to $15.9 million for the same period 
      in 2023. R&D expenses were $41.5 million for the year ended December 31, 
      2024 compared to $53.5 million for the year ended December 31, 2023. The 
      decrease in R&D expenses for both the fourth quarter of 2024 and the year 
      ended December 31, 2024 was primarily due to a decrease in development 
      costs related to RLYB212, RLYB116 and other program candidates, in 
      addition to a decrease in payroll and personnel-related costs, largely 
      related to the workforce reduction. 
 
   -- General & Administrative (G&A) Expenses: G&A expenses were $4.3 million 
      for the fourth quarter of 2024, compared to $5.2 million for the same 
      period in 2023. G&A expenses were $19.6 million for the year ended 
      December 31, 2024, compared to $25.4 million for the year ended December 
      31, 2023. The decrease in G&A expenses for both the fourth quarter of 
      2024 and the year ended December 31, 2024 was primarily due to a decrease 
      in other general and administrative expenses including consulting fees, 
      director and officer insurance premiums and professional fees, in 
      addition to lower payroll and personnel-related costs, largely related to 
      the workforce reduction and lower ongoing headcount in 2024 as compared 
      to 2023. 
   -- Net Loss and Net Loss Per Common Share: Rallybio reported a net loss of 
      $11.0 million, or $0.25 per common share, for the fourth quarter of 2024 
      compared to a net loss of $20.2 million, or $0.50 per common share, for 
      the same period in 2023. A net loss of $57.8 million, or $1.33 per common 
      share, was reported for the year ended December 31, 2024 compared to a 
      net loss of $74.6 million, or $1.84 per common share, for the year ended 
      December 31, 2023. 
 
   -- Cash Position: As of December 31, 2024, cash, cash equivalents, and 
      marketable securities were $65.5 million. Rallybio expects these funds 
      will be sufficient to support operations into the second half of 2026. 

About Rallybio

Rallybio $(RLYB)$ is a clinical-stage biotechnology company with a mission to develop and commercialize life-transforming therapies for patients with severe and rare diseases. Rallybio has built a broad pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of maternal fetal health, complement dysregulation, hematology, and metabolic disorders. The Company has two clinical stage programs: RLYB212, an anti-HPA-1a antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) and RLYB116, a C5 inhibitor with the potential to treat several diseases of complement dysregulation, as well as additional programs in preclinical development. Rallybio is headquartered in New Haven, Connecticut. For more information, please visit www.rallybio.com and follow us on LinkedIn and Twitter.

Forward-Looking Statements

This press release contains forward-looking statements that are based on our management's beliefs and assumptions and currently available information. All statements, other than statements of historical facts contained in this press release are forward-looking statements. In some cases, forward-looking statements can be identified by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements concerning the timing of disclosure of preliminary PK and safety data for the sentinel participant in the RLYB212 Phase 2 trial, presenting or disclosing interim data from the FNAIT natural history study and the timing of such disclosure, whether RLYB212 will be an effective therapeutic approach for FNAIT, the timing of initiating the RLYB116 confirmatory PK/PD study and the date when data is available, including data for Cohorts 1 and 2, whether RLYB116 will be effective in treating a broad range of complement-mediated diseases, the timing of initiation of IND-enabling activities for REV102, and the timing of data in a preclinical model of later-onset HPP. The forward-looking statements in this press release are only predictions and are based largely on management's current expectations and projections

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