Day One at Day One

Day One at Day One

To the patients, parents and advocates, the scientists, physicians, and nurses, and members of the pediatric oncology community:

Today we are publicly sharing our plans for a new company: Day One Biopharmaceuticals. Our name is inspired by The Day One Talk, the discussion that occurs between oncologists and families at the time of a new pediatric cancer diagnosis. Our goal is to rapidly develop new medicines for cancer, with a special focus on drugs that can help children, and our efforts build on the ongoing work of many within this remarkable community. We look forward to continuing to work with you. 

Earlier today we outlined a new financing -- $60 million from a syndicate of exceptional investors -- and a clinical-stage drug we licenced for development. More details about the company and business are available in this press release. The specifics we shared are the result of over 18 months of work. But what we are most excited about is the potential for Day One, and for what the future holds. 

Day One was founded because -- at a time when oncology drug development has accelerated over the past 20 years -- children with cancer continue to be left behind. Every day, families with a child fighting cancer find themselves needing to make treatment decisions with limited options, despite the fact that we are in the midst of a revolution in cancer biology and cancer drug discovery. 

In my previous medical practice, I treated children with a limited armamentarium of chemotherapy drugs, most used in intensive combination regimens, and almost of which have been associated with life-long side effects. The fact that this hasn’t changed much in the past 20 years highlights the lack of access to innovative therapies for pediatric cancer patients. We felt compelled to build Day One in order to address the hole left by the drugs that were never developed for children, and biological targets that were never pursued. 

We also built Day One to solve another problem - children often lack rapid access to the new therapies in development for adult cancers. On average, children with cancer in the US gain access to cutting-edge investigational treatments 6.5 years later than adults. Partly as a result of these delays, only 12 new drugs have been approved in the past 30 years for the treatment of cancers that occur primarily in children. 

Why is this the case? 

This disparity in access and innovation is driven by historical misperceptions about safety, regulatory barriers, the complexity of conducting pediatric clinical trials, and small market sizes. I have heard these arguments throughout my career, and they only get more outdated as time passess: misperceptions about the challenge of developing new cancer therapeutics for children; unfounded concerns that children are at higher risk of side effects from new drugs, compared to adults; or the corporate risk of exposing children to new therapeutics “too soon.” Others point to small markets and the difficulty making a ‘blockbuster’ drug for children. Many not-for-profit, NGO, and advocacy groups are working to change these misperceptions and respond to these challenges, and that work is essential to the fight against pediatric cancer. With Day One, we are bringing a new set of tools to this table: we believe market forces can be harnessed and focused to help advance new solutions for patients with cancer. 

Our approach is a simple one, born of two questions: how do we help the most people and how do we bring the best cancer treatments to approval and to market as fast as possible? The answer to both questions is to prioritize new products that are likely to benefit both adults and children with cancer. It sounds straightforward, but, understandably, most often targets are selected, and drugs are developed, to best address the biology of tumors that occur primarily in adults. 

After a rigorous analysis of many targets and products, we were convinced that clinical trials and new drug approvals could be accelerated by identifying programs that address targets at the intersection of adult and pediatric cancer biology. This allows us to integrate the distinct aspects of each population into a single fast-paced development plan. In many instances, we can advance products towards approval faster in pediatric indications than in adult indications because the biopharmaceutical industry has left so many disease populations - especially patients with relapsed disease - unaddressed, and without a clear standard-of-care. Regulatory agencies have encouraged approaches like this, which allow us to bring novel therapies to diseases and patient subgroups that have been overlooked and underserved. We anticipate that this trend will continue, given the potential of embracing modern, patient-centric development plans to fuel innovation 

While that’s the business case, the most important result - after the complex financial modeling, planning, and negotiating - will be that we can initiate and advance drug development programs that will be life-changing for families.  

Our first drug development program, DAY101, exemplifies this approach. DAY101 (formerly TAK-580) is a Phase 2-ready drug that is clinically active in the largest subset of pediatric brain tumors. It has strong development potential in a second pediatric indication, as well as a number of adult indications. Our current funding will enable us to advance this program into the clinic in 2020, and also will allow us to identify additional pipeline programs capable of addressing other unmet needs in pediatric oncology and make a difference for cancer patients - of all ages. We look forward to sharing news of our progress with you in the months and years to come.

Many of you have already been deeply involved in this work for many years, and we have been the beneficiaries of your advice, guidance, trust, and support. In particular, we’d like to thank Peter Adamson and Susan Weiner for introducing my co-founder, Julie Grant, to the remarkable world of pediatric oncology. We’d also like to to thank the members of ACCELERATE, especially Working Group 4, including Delphine Heenen, Jean-Charles van den Branden, Sam Daems, Andy Pearson, Gilles Vassal, Patricia Blanc, and Raphael Rousseau, for their work and extraordinary vision that informed our business model. We also own an enormous thanks to the leadership of the Pediatric Low Grade Astrocytoma Foundation, now a part of the Pediatric Brain Tumor Foundation - AJ Janower, Evan Hecker, Ken Gainey, Amy Weinstein, John Ragnoni, and Daniel Itzkovitz - and the rest of the PLGA Foundation team and their many donors and supporters. Their vision in funding the pre-clinical and clinical development of TAK-580 (now DAY101) in pediatric glioma inspired us to fight for this drug and provide it a new home and a path to approval. We are profoundly grateful to our collaborators and partners at the Dana-Farber Cancer Institute Pediatric Low Grade Astrocytoma Program and the Pacific Pediatric Neuro-Oncology Consortium for their scientific and clinical accomplishments that gave us the reason to believe in this program. We are immensely thankful to the team at Takeda, including Philip Rowlands, Dan Curran, Zach Carey, Rachel Brake, and Paul Juniewicz for their work on TAK-580 and their dedication to finding it a home and a path forward for continued development at Day One. And finally, to the teams at Canaan Partners, Atlas Venture, and Access Biotechnologies, we are grateful for your support, your expert guidance, and your belief in our team and our mission.

To all of you, and to everyone who has supported Day One to help us reach this important milestone, you have our deepest thanks. It is our honor for us to be a part of, and to work in service of, this incredible community.

My very best wishes,

Sam Blackman, Chief Medical Officer and Co-Founder

Day One Biopharmaceuticals, Inc.

Congrats Sam! This is great news.

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Allison Kemner

VP Clinical Sciences and Operations

3y

Congratulations Sam! Excited for the future!

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Dana Langworthy

Head of Talent Acquisition | HR Leader

3y

Congratulations Sam, this is incredible news!

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Rebecca E.

Owner and Chief Organizer at GAL TO THE RESCUE LLC

3y

Congrats!!!

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