Rallybio Corporation · NASDAQ:RLYB
Rallybio — Investor Presentation (March 2022)
29 slides from Rallybio's investor presentation, Mar 7, 2022
Slide 1: Corporate Presentation March 2022
MARCH 2022 TAKING A DEVASTATING DISEASE – AND DEVASTATING IT. We’re going there. Corporate Presentation
Slide 2: Forward-Looking Statements
Forward-Looking Statements–This presentation contains forward-looking statements that are based on management’s beliefs and assumptions and on information currently available to management. All statements other than statements of historical facts contained in this presentation are forward-looking st
Slide 3: Our Mission
We’ve gathered a proven team of innovators with the fearlessness to create new paths forward others haven’t tried And we want to develop therapies that shatter expectations of what’s possible Because it’s time to tackle the undone, the too difficult, the inaccessible – and change the odds for patien
Slide 4: Building for Growth
Building for Growth 4 GROUNDWORK FOR SUCCESS Diversified portfolio of five programs across three areas: maternal-fetal, complement dysregulation and metabolic Strong focus on expanding our pipeline through global business development platform Strong financial position; Completed $93M IPO in August 2
Slide 5: Rapidly Advancing Our Diversified Portfolio
Rapidly Advancing Our Diversified Portfolio THERAPEUTIC AREA PROGRAM MOLECULE APPROACH DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 DEVELOPMENT RIGHTS Maternal Fetal Blood Disorders Prevention of FNAIT RLYB212* Monoclonal HPA-1a Antibody RLYB211 Polyclonal HPA-1a Antibody Complement Dysregulation P
Slide 6: Strong Focus on Portfolio Expansion
PRODUCT CANDIDATE REQUIREMENTS Clear mechanism of action Validated modality TARGET DISEASE REQUIREMENTS Well-understood pathophysiology Significant unmet patient need Potential for transformative benefit Pursuing Transformative Science Around the Globe to Expand Our Portfolio with New Assets and Par
Slide 7: Programs for the Prevention of FNAIT: RLYB212, RLYB211
Programs for the Prevention of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) RLYB212 RLYB211
Slide 8: Potential to Eradicate a Devastating Disease in Fetuses and Newborns
Potential to Eradicate a Devastating Disease in Fetuses and Newborns Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) is a rare condition in which the mother’s immune system attacks the platelets of her fetus. Destruction of fetal platelets can lead to potentially catastrophic fetal and neonat
Slide 9: Approximately 22,700 Pregnancies Estimated at High Risk for FNAIT
~8M Each Year, Approximately 22,700 Pregnancies Estimated at High Risk for FNAIT * Estimate is based on a ~2% incidence of HPA-1a negative status in the Caucasian population Source: NCHS National Vital Statistics Report Volume 68, Number 13, November 30, 2019, Births: Final Data for 2018; World Bank
Slide 10: FNAIT is Caused by a Platelet Antigen Mismatch Between Mother and Fetus
FNAIT is Caused by a Platelet Antigen Mismatch Between Mother and Fetus Sources: 1. Kjeldsen-Kragh, J and Ahlen, M. Transfus Apher Sci. 2020 Feb; 59(1): 102707 FNAIT occurs when the mother’s immune system attacks the platelets of her fetus Caused by a mismatch in the type of Human Platelet Antigen 1
Slide 11: Potential First-in-Class Preventive Therapy for FNAIT
Potential First-in-Class Preventive Therapy for FNAIT RLYB212 Monoclonal anti-HPA-1a antibody Moved to lead candidate for prevention of FNAIT in 3Q 2021 Dosing has initiated in Phase 1 safety and PK study Proof-of-concept data from Phase 1b study expected 3Q 2022 RLYB211 Polyclonal anti-HPA-1a antib
Slide 12: Rapid Elimination of Transfused Platelets Following RLYB211
Rapid Elimination of Transfused Platelets Following Administration of RLYB211, an Anti-HPA-1a Antibody COHORT 1 RESULTS 8 participants received either single dose RLYB211 1000 IU (n=6) or placebo (n=2) to assess elimination of HPA-1a positive platelets, based on t1/2 of 10x109 transfused platelets R
Slide 13: Clinical Proof-of-Concept with RLYB211; Favorable Attributes of RLYB212
Clinical Proof-of-Concept Achieved with RLYB211; Favorable Attributes of RLYB212 Could Provide Best Option for Expectant Mothers RLYB211 RLYB212 Mechanism of Action Anti-HPA-1a antibody Anti-HPA-1a antibody Route of Administration IV bolus injection Subcutaneous injection Pharmacokinetic Profile Wid
Slide 14: Platelet Clearance and Prevention of Alloimmunization with RLYB212 in Preclinical Model
Platelet Clearance and Prevention of Alloimmunization Demonstrated with RLYB212 in Preclinical Model of FNAIT RLYB212 rapidly and completely eliminates HPA-1a positive platelets at concentrations more than 1000-fold lower than RLYB211 PREVENTION OF IMMUNIZATION PLATELET CLEARANCE pre-bleed Week 1 We
Slide 15: RLYB212 Phase 1 Safety and PK Study Underway
WEEK 24 WEEK 12 RLYB212 DAY 1 Placebo Safety Follow-up Screening Cohort 1 Design RLYB212 DAY 1 Placebo Safety Follow-up Screening Cohort 2 Design WEEK 12 WEEK 2 RLYB212 Phase 1 Safety and PK Study Underway STUDY DESIGN Single-blind, placebo-controlled study investigating the safety and pharmacokinet
Slide 16: RLYB212 Phase 1b Proof-of-Concept Study to Start 2Q 2022
RLYB212 Phase 1b Proof-of-Concept Study to Start 2Q 2022 STUDY DESIGN Single-blind, placebo-controlled, proof-of-concept study investigating the capacity of RLYB212 to rapidly eliminate HPA-1a positive platelets transfused to HPA-1a negative healthy male volunteers (N=8 subjects) PRIMARY OBJECTIVE T
Slide 17: FNAIT Natural History Alloimmunization Study Underway
FNAIT Natural History Alloimmunization Study Underway STUDY OBJECTIVES Obtain better prevalence estimates of the FNAIT at-risk population in racial and ethnic groups that may have been under-represented in previously published studies Provide historical alloimmunization rate to control a future sing
Slide 18: Screening For Risk of FNAIT Fits Into Routine First Trimester Prenatal Care
Screening For Risk of FNAIT Fits Into Routine First Trimester Prenatal Care Practice * Summarized from ACOG Guidelines for Perinatal Care; ** For pregnancies identified at high FNAIT risk, physicians may conduct additional follow-up testing, e.g., maternal anti-HPA-1a antibody status, fetal HPA-1a g
Slide 19: C5 Inhibitors for Diseases due to Complement Dysregulation
C5 INHIBITORS For the Treatment of Diseases due to Complement Dysregulation
Slide 20: Applying Our Expertise to Transform Diseases of Complement Dysregulation
Complement dysregulation has been implicated in the pathogenesis of a growing number of diseases Applying Our Expertise to Transform Diseases of Complement Dysregulation Our team has designed, developed, and/or secured approval for two C5 inhibitors in four rare disease indications globally C5 repre
Slide 21: Our Approach to Treating Diseases of Complement Dysregulation
Our Approach to Treating Diseases of Complement Dysregulation RLYB116 Affibody® molecule linked to an Albumod® albumin binding domain Suitable for subcutaneous administration Phase 1 study initiated in February 2022 Single dose safety, PK, and PD data expected 2H 2022 PHARMACODYNAMIC PROPERTIES An e
Slide 22: RLYB116 Designed for Clinical and Commercial Success
RLYB116 is Designed to Possess Attributes for Clinical and Commercial Success ATTRIBUTES FOR CLINICAL AND COMMERCIAL SUCCESS Terminal complement blockade Rapid, complete, and sustained inhibition of C5 Designed to be optimized for C5 binding, stability, and long half-life in serum Small size enablin
Slide 23: RLYB116 Phase 1 Study Design: Single Ascending Dose
RLYB116 Phase 1 Study Design: Evaluating Safety, PK and PD of Single Ascending Dose STUDY DESIGN Single-blind, placebo-controlled dose escalation study investigating the safety, pharmacokinetics and pharmacodynamics of single dose RLYB116 in healthy participants STUDY COHORTS Up to 6 sequential asce
Slide 24: Lead Indication Strategy Guided by Biology
Lead Indication Strategy Guided by Biology 1. Brodsky, RA. Blood. 2014; 124 (18): 2804-2811; 2. Sahin, F. Am J Blood Res 2015; 5 (1): 1-9; 3. Howard, JF. Ann N Y Acad Sci. 2017; 1412 (1): 113-128; 4. Punga, AR. Lancet Neurol 2022; 21: 176-188 A potentially life-threatening, rare hematologic disorder
Slide 25: RLYB114 for Ophthalmic Indications of Complement Dysregulation
1. Park et al 2019 Frontier in Immunology; 2. Bakri et al 2007 Ophthalmology; 3. Park et al 2016 Inv Ophthalmol Vis Sciences, Christoforidis et al 2012 Current Eye Research C5-targeted Affibody® molecule conjugated to polyethylene glycol (PEG) Complement dysregulation implicated in the pathogenesis
Slide 26: RE Ventures I: ENPP1 Inhibitor Program for Hypophosphatasia
RE VENTURES I ENPP1 Inhibitor Program for Hypophosphatasia (HPP) Joint Venture with Industry-leading AI/ML Pharmatech Exscientia
Slide 27: Hypophosphatasia is a Rare, Potentially Life-Threatening Genetic Disease
Hypophosphatasia is a Rare, Potentially Life-Threatening Genetic Disease TNSALP: Tissue Non-Specific Alkaline Phosphatase In patients with hypophosphatasia, the reduction of PPi hydrolysis by TNSALP results in a relative increase in PPi, leading to an inhibition of mineralization, and inhibited hydr
Slide 28: ENPP1 Inhibitor Approach to Address Unmet Need for Patients with HPP
ENPP1 Inhibitor Approach to Address Unmet Need for Patients with HPP ERT: Enzyme replacement therapy UNMET NEED IN HPP Significant population of underserved patients exist due to variability in disease severity and access to approved therapy PROGRAM FOCUS & RATIONALE Small molecule inhibitors of ENP
Slide 29: Contacts
29 Media Relations Tara DiMilia Tara.Dimilia@tmstrat.com Business Development Amanda Hayward, Ph.D. ahayward@rallybio.com Corporate Development Steven Tuch stuch@rallybio.com investors@rallybio.com Investor Relations Ami Bavishi abavishi@rallybio.com