Rallybio Corp Stock Price, News & Analysis

Rallybio (NASDAQ: RLYB) has announced the discontinuation of its RLYB212 program for preventing fetal and neonatal alloimmune thrombocytopenia (FNAIT). The decision follows Phase 2 clinical trial results showing the drug's inability to achieve required target concentrations for efficacy.

The company will now focus on advancing RLYB116, a once-weekly C5 inhibitor for complement-driven diseases, with a confirmatory PK/PD study planned for Q2 2025 and data expected in 2H 2025. This program targets diseases including PNH, APS, and gMG, representing a >$6 billion commercial opportunity.

Additionally, Rallybio's pipeline includes REV102, an ENPP1 inhibitor for hypophosphatasia entering IND-enabling studies, and RLYB332, a long-acting matriptase-2 antibody for iron overload diseases.

Rallybio (NASDAQ: RLYB) reported its Q4 and full year 2024 financial results, highlighting key developments and upcoming milestones. The company ended 2024 with $65.5 million in cash and equivalents, providing runway into 2H 2026.

Key financial metrics include Q4 2024 revenue of $38,000 and full-year revenue of $0.6 million, primarily from a Johnson & Johnson collaboration. Q4 net loss was $11.0 million ($0.25 per share), while full-year net loss reached $57.8 million ($1.33 per share).

Notable pipeline updates include:

• RLYB212: Phase 2 trial dosing underway with key data expected in Q2/Q3 2025
• RLYB116: Confirmatory PK/PD study initiation planned for Q2 2025
• REV102: Advancing toward Phase 1 in 2026 for hypophosphatasia treatment
• RLYB332: Showed promising preclinical results for iron overload diseases

Rallybio (Nasdaq: RLYB), a clinical-stage biotechnology company focused on developing therapies for rare diseases, has announced that its Chief Executive Officer, Stephen Uden, M.D., will deliver a corporate overview at the TD Cowen 45th Annual Health Care Conference.

The presentation is scheduled for Tuesday, March 4, 2025 at 1:10 p.m. EST in Boston, MA. Investors and interested parties can access a live webcast of the presentation through the Events and Presentations section of Rallybio's website at www.rallybio.com.

A replay of the webcast will remain available for 30 days following the presentation, allowing those unable to attend the live event to review the company's updates.

Rallybio (RLYB) has initiated dosing in its Phase 2 clinical trial of RLYB212, targeting pregnant women at higher risk for HPA-1a alloimmunization and fetal and neonatal alloimmune thrombocytopenia (FNAIT). The trial will evaluate pharmacokinetics and safety across three stages: a sentinel participant, followed by two cohorts totaling eight participants.

The study involves subcutaneous administration of RLYB212 starting at Gestational Week 16 and continuing every 4 weeks through delivery. Key data readouts are expected in Q2 2025 for second trimester pharmacokinetic and safety data, and Q3 2025 for delivery-time data. The trial will be conducted at European sites.

Rallybio (RLYB) outlined its 2024 achievements and 2025 milestones for its rare disease therapeutics pipeline. The company highlighted progress across multiple programs:

For RLYB212, they initiated the first Phase 2 trial for FNAIT prevention, screened over 14,000 pregnant women, and published Phase 1b results. The RLYB116 program completed manufacturing improvements for better tolerability. Their REV102 program for hypophosphatasia (HPP) treatment advanced, with data showing 30% reduction in PPi biomarker.

Key anticipated 2025 milestones include: RLYB212 Phase 2 trial sentinel participant dosing in Q2 2025 with data readouts in Q3-Q4; RLYB116 confirmatory PK/PD study initiation in Q2 2025 with results in H2 2025; and REV102's progression to IND-enabling studies for Phase 1 initiation in 2026.

Rallybio (RLYB) presented promising preclinical data for two pipeline candidates at the 66th ASH Annual Meeting. The first study showed that RLYB212 successfully prevented maternal alloimmunization and FNAIT in pregnant mice at doses of 1.01 or 5.05 µg/kg, with pups maintaining normal platelet counts and showing no signs of thrombocytopenia.

The second study demonstrated that RLYB332, a long-acting anti-matriptase-2 antibody, showed rapid and sustained effects on pharmacodynamic parameters including serum iron, UIBC, and transferrin saturation in humanized FcRn mice. The treatment was well-tolerated and showed superior results compared to comparator molecules, positioning it as a potential best-in-class therapeutic for iron overload diseases.

Rallybio (RLYB) announced new findings for RLYB116, its once-weekly subcutaneous C5 inhibitor for rare diseases. New biomarker analyses revealed that RLYB116 achieved greater complement inhibition in Phase 1 than initially reported, with the original assay overestimating free C5 levels by approximately ten-fold. The company completed manufacturing process enhancements expected to improve RLYB116's tolerability profile. A confirmatory clinical PK/PD study is planned for Q2 2025, involving two cohorts testing weekly doses of 150mg and 225mg over 4 weeks with a 10-week follow-up period.

Rallybio announced the publication of a manuscript detailing target-mediated drug disposition (TMDD) modeling used to determine RLYB212 dosing for pregnant women. The model incorporates pregnancy-related physiological changes and will be evaluated in their recently initiated Phase 2 clinical trial. RLYB212 is being developed to prevent alloimmunization in pregnant women and eliminate FNAIT risk in fetuses and newborns. The company is also conducting a natural history study, which has screened over 13,000 pregnant women as of November 1, 2024.

Rallybio (Nasdaq: RLYB), a clinical-stage biotech company focused on rare disease therapies, has announced its upcoming participation in the Evercore ISI HealthCONx Conference. Stephen Uden, M.D., CEO of Rallybio, will engage in a fireside chat on Wednesday, December 4, 2024 at 12:30 p.m. ET in Coral Gables, FL. The presentation will be available via live webcast through Rallybio's website, with a replay accessible for 30 days afterward.