Full Press Release Details
Day One s OJEMDA (tovorafenib) Receives
US FDA Accelerated Approval for Relapsed or Refractory BRAF-altered Pediatric Low-Grade Glioma (pLGG), the Most Common Form of Childhood Brain Tumor
First and only FDA-approved type II RAF inhibitor for patients with relapsed or refractory pLGG
harboring a BRAF fusion or rearrangement, or BRAF V600 mutation
RAPNO LGG overall response rate (ORR) of 51%
Day One receives rare pediatric disease priority review voucher
Conference call and webcast to be April 24, 8:30 a.m. Eastern Time
BRISBANE, Calif., April 23, 2024 Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN) ( Day One or the
Company ), a commercial-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced that the U.S. Food and Drug Administration (FDA)
has approved OJEMDA (tovorafenib), a type II RAF inhibitor, for the treatment of patients 6 months of age and older with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under
accelerated approval based on response rate and duration of response. With the approval, Day One received a rare pediatric disease priority review voucher from the FDA.
OJEMDA ushers in a new day for children living with relapsed or refractory pLGG, and we are pleased that we can deliver a new medicine for these
patients in desperate need of new treatment options. Moreover, OJEMDA is the first and only FDA-approved medicine for children with BRAF fusions or rearrangements, which are the most common molecular
alteration in pLGG, said Jeremy Bender, Ph.D., chief executive officer of Day One. We are very proud that our first approved medicine addresses this serious and life-threatening disease of childhood and adolescence. We are grateful to
the pLGG community, including patients and their families, study investigators, non-profit organizations, and advocacy groups, for their collaboration and support as we strive to close the innovation gap for
children with cancer awaiting new treatments.
pLGG is the most common brain tumor diagnosed in children, with patients suffering profound tumor-
and treatment-associated morbidities that can impact their life trajectory. BRAF is the most commonly altered gene in pLGG, with up to 75 percent of children having a BRAF alteration. Until now, there had been no medicines approved for
patients with pLGG driven by BRAF fusions.
pLGG is a chronic and relentless cancer that can devastate children and their families, often stealing
their vision, balance and speech, said Dr. Sabine Mueller, pediatric neuro-oncologist, University of California San Francisco Benioff Children s Hospitals. The goal of pLGG treatment is to stabilize or shrink the tumor without
further disrupting the child s and family s life. Historically, there has been no standard of care for children with pLGG who have relapsed. We are excited to welcome a new targeted treatment option with once-weekly oral dosing designed
specifically for these kids and their families.
OJEMDA is the only systemic therapy for pLGG that offers once-weekly dosing, with or without food, as a tablet or
Approval Based on Multiple Criteria
The accelerated approval of OJEMDA is based on data from the Company s pivotal open-label Phase 2 FIREFLY-1 trial,
which enrolled a total of 137 relapsed or refractory BRAF-altered pLGG patients across two study arms. Arm 1, which accrued 77 patients, was used for the efficacy analyses. Arm 2 provided additional safety data from an incremental 60 patients and
was initiated to enable access to tovorafenib once Arm 1 had fully accrued. Details of this trial were presented in November 2023 at the Society for Neuro-Oncology meeting through two oral plenary presentations and in parallel through a publication
The approval of OJEMDA was based, in part, on the major efficacy outcome measure of overall response rate
(ORR), defined as the proportion of patients with complete response (CR), partial response (PR), or minor response (MR) by independent review based on Response Assessment in Pediatric Neuro-Oncology Low-Grade
This is a tremendous moment not only for Day One, but also for the broader pediatric brain tumor community. Thanks to the close
collaboration between RAPNO and Response Assessment for Neuro-Oncology (RANO) working groups and the patients and families impacted by a pLGG diagnosis, the way we think about measuring response and the goals of therapy for this unique patient
population truly evolved, said Dr. Samuel Blackman, co-founder and head of research and development at Day One. As a pediatric neuro-oncologist, the approval of OJEMDA is a dream
In Arm 1, data from the 76 RAPNO LGG evaluable patients include:
The safety of OJEMDA was evaluated in 137 patients with relapsed or refractory pLGG, with the majority of adverse events being Grade 1 or Grade 2. The most
common side effects were rash, hair color changes, tiredness, viral infection, vomiting, headache, fever, dry skin, constipation, nausea, acne and upper respiratory tract infection.
This is an exciting moment for children and families living with pLGG who previously had few treatment
options if their disease progressed, said Courtney Davies, president and chief executive officer of the Pediatric Brain Tumor Foundation. The approval of OJEMDA is a testament to the power of community and industry collaboration to
address a critical unmet need for children whose day-to-day living and long-term health outcomes are significantly impacted by pLGG. The potential benefit that a new
treatment option provides children living with this disease and their families is crucial. There is so much to celebrate here.
continues its commitment to the pLGG community with the Phase 3 FIREFLY-2/LOGGIC randomized clinical trial evaluating tovorafenib as a potential front-line therapy compared to chemotherapy in patients aged 6
months to 25 years with pLGG, which it believes will satisfy certain post-marketing requirements to the FDA. This study is currently enrolling patients in the United States, Canada, Europe, Australia, and Asia.
Introducing EveryDay Support From Day One
Day One Biopharmaceuticals is dedicated to helping patients with pLGG access OJEMDA and supporting their families throughout the treatment journey. As part of
this commitment, we are pleased to announce EveryDay Support From Day One , a comprehensive program that offers personalized services for eligible patients and their care teams, including
insurance coverage support, financial assistance options, shipping medication to patients homes, and educational resources. Caregivers and healthcare providers can visit www.everydaysupport.com or call a patient navigator at 855-DAY1-BIO (855-329-1246) for more information.
OJEMDA will be available in the U.S. through specialty pharmacy partners Biologics and Onco360.
Day One will host a conference call and
webcast tomorrow, April 24 at 8:30 a.m. Eastern Time. To access the live conference call by phone, dial 877-704-4453 (domestic) or 201-389-0920 (international), and provide the access code 13745150. Live audio webcast will be accessible from the Day One Investors & Media page. To ensure a
timely connection to the webcast, it is recommended that participants register at least 15 minutes prior to the scheduled start time. An archived version of the webcast will be available for replay on the Events & Presentations section of
the Day One Investors & Media page for 30 days following the event.
OJEMDA (tovorafenib) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases.
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 or rearrangement, or BRAF V600 mutation. 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 and description of clinical benefit in a confirmatory trial(s).
Tovorafenib is under evaluation as a therapy for patients with pLGG requiring front-line treatment (Phase 3
FIREFLY-2/LOGGIC). It is also being studied in combination with the MEK inhibitor pimasertib for adolescent and adult patient populations with recurrent or progressive solid tumors with MAPK pathway
alterations (FIRELIGHT-1).
Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment
of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European
Commission for the treatment of glioma.
For more information, please visit www.ojemda.com.
What is OJEMDA (tovorafenib)?
OJEMDA is a prescription medicine used to treat certain types of brain tumors
(cancers) called gliomas in patients 6 months and older:
IMPORTANT SAFETY INFORMATION
Before taking or giving
OJEMDA, tell your healthcare provider about all of your or your child s medical conditions, including if you:
Females who are able to become pregnant:
Males with female partners who are able to become
pregnant should use effective non-hormonal birth control (contraception) during treatment with OJEMDA and for 2 weeks after your last dose of OJEMDA.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
avoid while taking OJEMDA?
Limit the amount of time you spend in sunlight. OJEMDA can make your skin sensitive to the sun (photosensitivity). Use sun
protection measures, such as sunscreen, sunglasses and wear protective clothes that cover your skin during your treatment with OJEMDA.
possible side effects of OJEMDA?
OJEMDA may cause serious side effects, including:
The most common side effects of OJEMDA include:
OJEMDA may cause fertility problems in males and females, which may affect your ability to have children.
These are not all the possible side effects of OJEMDA. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA
full Product Information and Instructions for Use for more information.
About Pediatric Low-Grade
Pediatric low-grade glioma (pLGG) is the most common brain tumor with an estimated incidence of 1,100
children per year who are eligible for front-line systemic therapy. BRAF is the gene most commonly altered in pLGG, of which there are two primary types of BRAF alterations a BRAF gene fusion and a BRAF point mutation. In children with
BRAF-altered pLGG, approximately 80 percent have BRAF fusions or rearrangements, while the remaining 20 percent have a V600 mutation.
low-grade gliomas can be chronic and relentless, with patients suffering profound side effects from both the tumor and the treatment, which may include chemotherapy and radiation. These side effects can impact
their life over the long term, and may include muscle weakness, loss of vision, and difficulty speaking. This type of tumor has a high risk of progression, and many children with pLGG require long-term treatment. While most children with pLGG
survive their cancer, children who do not achieve a complete resection following surgery may face years of increasingly aggressive treatment.
FIREFLY-1 is evaluating OJEMDA (tovorafenib) as once-weekly
monotherapy in patients aged 6 months to 25 years with relapsed or progressive pLGG harboring a known activating BRAF alteration. The trial is being conducted in collaboration with the Pacific Pediatric Neuro-Oncology Consortium (PNOC). The primary
endpoint is overall response rate (ORR), defined as the proportion of patients with confirmed response based upon Response Assessment for Neuro-Oncology High-Grade Glioma (RANO HGG) criteria. Secondary and exploratory endpoints include the overall
response rate based on Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO LGG) criteria, Response Assessment for Neuro-Oncology Low-Grade Glioma
(RANO LGG) criteria and volumetric analyses, progression-free survival, safety, functional outcomes, and quality of life measures. RANO HGG, RANO LGG and RAPNO LGG are assessed by blinded independent central review. Additional information about FIREFLY-1 may be found at ClinicalTrials.gov, using Identifier NCT04775485.
About the Pacific Pediatric
Neuro-Oncology Consortium
The Pacific Pediatric Neuro-Oncology Consortium (PNOC) is an international consortium with study sites within the United
States, Canada, Europe and Australia dedicated to bringing new therapies to children and young adults with brain tumors.
Day One Biopharmaceuticals is a commercial-stage biopharmaceutical company that believes when it comes to pediatric cancer, we can
do better. The Company was founded to address a critical unmet need: the dire lack of therapeutic development in pediatric cancer. Inspired by The Day One Talk that physicians have with patients and their families about an initial cancer
diagnosis and treatment plan, Day One aims to re-envision cancer drug development and redefine what s possible for all people living with cancer regardless of age starting from Day One.