Full Press Release Details
All Ages June 2024 1
Disclaimer This presentation and the accompanying oral commentary
contain forward-looking statements that are based on our management's beliefs and assumptions and on information currently available to our management. Forward-looking statements are inherently subject to risks and uncertainties, some of which
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comparable terminology. Forward-looking statements include all statements other than statements of historical fact contained in this presentation, including information concerning our future financial performance, including the sufficiency of our
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the potential achievement of milestones and provision of royalty payments thereunder, timing and results of nonclinical studies and clinical trials, efficacy and safety profiles of our products and product candidates, the ability of tovorafenib to
treat pediatric low-grade glioma (pLGG) or related indications, the potential therapeutic benefits and economic value of our products and product candidates, potential growth opportunities, competitive position, industry environment and potential
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a high degree of uncertainty and risk. 2
Cancer Therapies for People of All Ages Our Approach Develop
medicines for genomically-defined cancers Establish first-in-class position through rapid registration pathways Expand to adolescent and adult populations in parallel and pursue those opportunities with the same commitment we do for
children IPO: 2021 Financial Position: Runway into 2026 Nasdaq: DAWN Founded: 2018 3
Our Pipeline Phase 3/ Recent & Anticipated Product Candidate
Therapeutic Area Preclinical Phase 1 Phase 2 Approved Registrational Milestones BRAF-altered FDA approval: FIREFLY-1 (pivotal Phase 2) April 2024 Relapsed pLGG Tovorafenib Type II RAF Inhibitor OJEMDA brand name 1 in U.S. Frontline RAF- First
patient dosed: FIREFLY-2 (pivotal Phase 3) March 2023 altered pLGG MAPK-altered Recommended Phase 2 dose & Pimasertib schedule expected: FIREFLIGHT-1 solid tumors MEK 1/2 Inhibitor 2H 2024 (Combo w/ tovorafenib)
VRK1 Program Pediatric and In-licensed : August 2023 VRK1 Inhibitor adult cancers 1 OJEMDA has received accelerated approval by the U.S. Food and Drug Administration. Pimasertib Phase 1 dose escalation and expansion
trial previously completed. Includes patients 12 years of age. Research collaboration and license agreement with Sprint Bioscience AB for exclusive worldwide rights to a research-stage program targeting VRK1. pLGG, pediatric low-grade glioma.
4 The safety and efficacy of investigational agents and/or investigational uses of approved products have not been established.
TM OJEMDA (tovorafenib) Relapsed or Refractory BRAF-altered pLGG
OJEMDA Now Approved In The U.S. OJEMDA is the first and only FDA
Approved therapy for the treatment of pediatric patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation * 6 This indication is approved under
accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial.
pLGG Impact On Patients' Lives Lily was diagnosed with an operable
brain tumor at 5 months of age 7 Lily, lives with pLGG.
Pediatric Low-Grade Glioma: The Most Common Type Of Brain Tumor In
Children A Serious and Life-Threatening Disease pLGGs are chronic and For the majority of pLGG patients in the relapsed setting, there is no standard of care and no approved therapies , with patients relentless * Up to 75% of pLGGs
have a BRAF alteration , of those ~80% suffering profound tumor 2-6 are BRAF fusions and ~20% are BRAF V600 mutations and treatment-associated Despite surgery playing a significant role in treatment, the morbidity that can impact 7,8 vast
majority of patients still require systemic therapy their life trajectory over the Due to high rate of disease recurrence, most patients will 1 long term undergo multiple lines of systemic therapy over the course of their disease 1 2
*Incidence of BRAF alterations varies across pLGG subtypes. Sievert AJ, Fisher MJ. Pediatric low-grade gliomas. J Child Neurol. 2009;24(11):1397-1408. doi:10.1177/0883073809342005. Penman CL et al. Front Oncol. 8 3 4 5 6 2015;5:54. Cohen AR., N Engl
J Med. 2020;386(20):1922-1931. Lassaletta A, et al. J Clin Oncol. 2017;35(25):2934-2941. Faulkner C, et al. J Neuropathol Exp Neurol. 2015;74(9):867-872. Packer RJ, et al. Neuro 7 8 Oncol. 2017;19(6):750-761. Ostrum QT et al., Neuro Oncol. 2015;
16(Suppl 10):x1-x36; De Blank P. et al., Curr Opin Pediatr. 2019 Feb; 31(1):21-27.
Conventional Treatments Can Be Disruptive To Childhood And Can Have
Significant Long-Term Consequences Surgery Chemotherapy Radiation Significant recovery times Requirement for indwelling Risk of secondary malignancy catheter and weekly infusions Risks of complications Risk of
malignant transformation Risk of neutropenia, Resection may be limited by Risk of vascular proliferation and hypersensitivity reactions, location of tumor stroke nausea and vomiting and Potential for functional
deficits Neurocognitive impact, peripheral neuropathy based on location of tumor depending on location of tumor and extent of resection and radiation field Goal of therapy is to control the tumor, minimize the burden of surgery,
chemotherapy, and radiation, and reduce the risk of life-long treatment and disease-related effects Source: 1. Heitzer AM, Raghubar K, Ris MD, et al. Neuropsychological functioning following surgery for pediatric low-grade glioma: a prospective
longitudinal study. J Neurosurg Pediatr. 2019;1-9. doi:10.3171/2019.9.PEDS19357. 2. Bryant R. Managing side effects of childhood cancer treatment. J Pediatr Nurs. 2003;18(2):113-125. doi:10.1053/jpdn.2003.11. 3. Zahnreich S, Schmidberger H.
Childhood cancer: occurrence, treatment and risk of second primary malignancies. Cancers (Basel). 2021;13(11):2607. doi:10.3390/cancers/13112607. 4. National Cancer Institute. Fertility issues in girls and women with cancer. http://www.cancer.gov.
Accessed June 13, 2022. 5. Alessi I., Caroleo A.M., de Palma L., Mastronuzzi A., Pro S., Colafati G.S., Boni A., Della Vecchia N., Velardi M., Evangelisti M., et al. Short and Long-Term Toxicity 9 in Pediatric Cancer Treatment: Central Nervous
System Damage. Cancers. 2022;14:1540. doi: 10.3390/cancers14061540.
TM Overview U.S. Prescribing Information For OJEMDA (tovorafenib)
Available in tablet formulation and pediatric-friendly powder for oral suspension INDICATION OJEMDA is indicated for the treatment of pediatric patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma harboring a
BRAF fusion or rearrangement, or BRAF V600 mutation RECOMMENDED DOSE 2 380 mg/m administered orally once weekly (not to exceed a dose of 600mg once weekly); OJEMDA can be taken with or without food For full prescribing information, visit
dayonebio.com * 10 This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory
TM Efficacy Summary From OJEMDA (tovorafenib) Prescribing Information
Overall response rate (RAPNO-LGG) in RAPNO-LGG 76 evaluable patients 51% Response (IRC) n n (%) 95% CI ORR, n (%) 76 39 (51) 40-63 BRAF fusion or rearrangement 64 33 (52) 39-64 100 Prior MAPKi BRAF V600 mutation 12 6 (50) 21-79 80 100 MAPKi-naive 60
Prior MAPKi 80 Prior MAPKi use 45 22 (49) 31-64 BRAF mutation 40 * PD MAPKi-naive 20 60 MAPKi-na ve 31 17 (55) 36-73 0 BRAF mutation SD 40 * PD * * -20 MR 20 -40 * * * Median DOR, months 39 13.8 11.3-NR -60 PR * 0 SD * -80 * * * * CR
-20 * -100 * MR -40 Median TTR, months 39 5.3 * * * PR -60 * Range 1.6-11.2 * -80 * * CR * -100 * June 5, 2023 data cutoff. CI, confidence interval; DOR, duration of response; IRC, independent radiology review committee; LGG, low-grade glioma; NR,
not reached; ORR, overall response rate; RAPNO, Response Assessment in Pediatric Neuro-Oncology; TTR, time to response; CR, complete response; PR, partial response; MR, minor response; SD, stable disease; PD, 11 progressive disease. As of
the data cutoff, 66% remain on tovorafenib. Maximum change in tumor size (%) Maximum change in tumor size (%) Maximum change in tumor size (%)
TM Safety Summary From OJEMDA (tovorafenib) Prescribing Information
Warnings and Precautions TEAEs ( 30% of patients [n=137]) Preferred Term, n (%) Any Grade Grade 3 Hemorrhage Any AE 137 (100) 86 (63) Hair color changes 104 (76) 0 Skin toxicity, including photosensitivity Anemia 81
(59) 15 (11) Elevated CPK 80 (58) 16 (12) Hepatotoxicity Fatigue 76 (55) 6 (4) Vomiting 68 (50) 6 (4) Effect on growth Hypophosphatemia 64 (47) 0 Embryo-fetal toxicity Headache 61 (45) 2 (1) Maculo-papular rash 60 (44) 11 (8)
Use in NF1- associated tumors Pyrexia 53 (39) 5 (4) Dry skin 49 (36) 0 Elevated LDH 48 (35) 0 Increased AST 47 (34) 4 (3) No boxed warnings or Constipation 45 (33) 0 Nausea 45 (33) 0 Upper RTI 43 (31) 2 (1) contraindications Dermatitis
acneiform 42 (31) 1 (1) Epistaxis 42 (31) 1 (1) June 5, 2023 data cutoff. OJEMDA safety data (n=137). Treatment-emergent AEs 20% any grade in arms 1 & 2. AE, adverse event; AST, aspartate aminotransferase; CPK, creatine 12 phosphokinase;
LDH, lactate dehydrogenase; RTI, respiratory tract infection; TEAEs, treatment-emergent adverse events.
Estimated BRAF-Altered pLGG Patient Population In The U.S. ~26,000
Prevalence of Systemically- ~2,000-3,000 Treated Patients 1-5 ~1,100 Under 25 Recurrent/Progressive Total Addressable Incidence of Patient Population Treatment- 6 per Year at Steady Eligible * State Frontline 4,5 Patients 7-14 Up to 75% of pLGG
cases are BRAF-altered Incidence of BRAF alterations varies across pLGG subtypes of these cases have of these cases have BRAF fusion, primarily BRAF point mutations, ~80% ~20% KIAA1549-BRAF primarily BRAF V600 1 2 Selt F, van
Tilburg CM, Bison B, et al. Response to trametinib treatment in progressive pediatric low-grade glioma patients. J Neurooncol. 2020;149(3):499-510. doi:10.1007/s11060-020-03640-3. Ryall S, Tabori U, Hawkins C. Pediatric low-grade 3 glioma in the era
of molecular diagnostics. Acta Neuropathol Commun. 2020;8(1):30. doi:10.1186/s40478-020-00902-z. SEER US complete prevalence counts of patients aged under 25 with Brain and Other Nervous Systems tumors as of January 1, 4 5 2017. CBTRUS, Qaddoumi et
al 2009, Schreck et al 2019, ClearView Analysis. US Census. Estimated annual incidence, estimated prevalence, and estimated recurrent/progressive total addressable patient population are Day One calculations based 6 7 8 on publicly available data.
Source: Internal market research conducted by EpidStrategies, A Division of ToxStrategies, Inc. on behalf of Day One. Ryall S, et al. Acta Neuropathol Commun. 2020;8(1):30. Behling F, et al. Cancers (Basel). 13 9 10 11 12 13 2019;11(6):794. Penman
CL, et al. Front Oncol. 2015;5:54. Packer RJ, et al. Neuro Oncol. 2017;19(6):750-761. Cohen AR, et al. N Engl J Med. 2022;386(20):1922-1931. Ryall S, et al. J Neuropathol Exp Neurol. 2017;76(7):562-570. Lassaletta A, 14 * et al. J Clin Oncol.
2017;35(25):2934-2941. Faulkner C, et al. J Neuropathol Exp Neurol. 2015;74(9):867-872. The estimated addressable pool of recurrent or progressive pLGG patients is based on progression free survival curves modeled from
published literature. Predominantly seen in pilocytic astrocytomas. May vary across pLGG subtypes. BRAF, V-Raf murine sarcoma viral oncogene homolog B; MAPK, mitogen-activated protein kinase; pLGG, pediatric low-grade glioma.
What Physicians & Caregivers Are Looking For In A Therapy What
HCP's are Seeking What Caregivers are Seeking Effective in stopping or shrinking tumors Live as normal of a childhood as possible Manageable safety profile Minimal impact from the disease Minimal disruption to child's life Minimal
disruption to child's life "Our time with our kids is precious and not guaranteed, so the less time with meds and "The goal is not interfering with the child's life." doctors the better." - Ped Onc, Chicago
Ad Board - Caregiver for a child under 5 yrs 14
Product Profile Aligns With What Physicians Are Looking For In A
Therapy Meaningful tumor stabilization or shrinkage may be possible with OJEMDA. In the clinical trial: Efficacy 51% of children experienced tumor shrinkage by at least 25% 82% of children saw their tumors shrink or remain stable
Generally well-tolerated therapy, with 9 out of 10 patients staying on treatment in the clinical trial Safety Most common grade 3 / 4 adverse events include: anemia, elevated CPK, maculo- papular rash, fatigue & vomiting Once-weekly, taken with
or without food conveniently from home can mean Dosing fewer daily interruptions OJEMDA is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion,
rearrangement, or BRAF V600 mutation. 15 Data from Pivotal Phase 2 FIREFLY-1 trial.
Comprehensive Approach For A Successful Launch Physicians Payers
Patients & Families TM Objective: Establish OJEMDA as Objective: Provide a positive & Objective: Rapidly establish st 1 choice in relapsed / refractory supportive experience when coverage BRAF-altered pLGG patients initiating therapy
MEDICAID 89% 40% 65% COMMERCIAL 60% SP distribution enables consistent patient Pre-launch engagement to establish Day experience AIDED INTENT TO One & provide background information AWARENESS TREAT Comprehensive patient
support programs Plans in place for rapid engagement post- address patient needs and accelerates Dedicated & experienced sales team to approval access to drug engage HCPs 16 SP, Specialty Pharmacy.
Targeted Launch With Highly Experienced Field Team Targeting ~200
centers where 90% Deep oncology experience with of pLGG patients receive treatment relationships at top-tier accounts Average experience: 18 Account 13 years of oncology Managers fully-dedicated 4 years of rare disease to OJEMDA 2 years of pediatric
oncology clinical experience Institutional experience and existing relationships with key accounts 17
Patient Support Program Supporting Access DEDICATED PATIENT NAVIGATOR
COPAY CARD PHONE & VIDEO PROGRAM CONSULTATIONS COVERAGE PATIENT DELAY ASSISTANCE PROGRAM PROGRAM COVERAGE INTERRUPTION SUPPORT 18
FIREFLY-2 / LOGGIC Pivotal Phase 3 Trial of Tovorafenib in Frontline
FIREFLY-2/LOGGIC Pivotal Phase 3 Trial Of Tovorafenib In Frontline pLGG
Trial Design Endpoints Randomized, global, registrational Phase 3 trial of monotherapy tovorafenib Primary endpoint: ORR based on RAPNO-LGG criteria, assessed by vs SoC chemotherapy blinded independent central review The ORR