…to hope for life
Rooted in our founders’ intent, our singular mission is to provide hope for life. We strive to advance medicines that can bring hope for life to as wide a range of patients as possible, and for as long as possible.
As we progress on this journey, and witness individuals experiencing a meaningful life milestone – holding a newborn grandchild, attending a loved one’s graduation, dancing at their child’s wedding – that, too, exemplifies founders’ intent. This perspective guides how we make decisions and operate every day.
…to the unmet need
When advancing assets, our primary consideration is whether the research could lead to a significant and meaningful advance in patient care. To help us ensure this, we work closely with leading expert physicians and patient advocacy groups who are closest to the unmet needs in a particular patient community.
We deeply value investigator-initiated trials (IITs) that interrogate a potential therapeutic’s impact on aspects of a disease that physician-researchers have identified as being among the most important unmet needs.
…to compassionate use
While many companies are constrained when evaluating compassionate use or expanded access programs (EAPs), with a “return on investment” lens, we welcome these programs because of our dedication to providing hope for life.
We seek to remove access barriers to promising investigational treatments for the patients who otherwise have limited options and the requesting physicians who are providing their care. Not only do these programs help patients with immediate, intractable unmet needs, but they can also provide actionable data that inform future clinical development strategies for our programs.
Working closely with xCures, a healthcare technology company, we help facilitate and implement EAPs to provide our investigational medicines to individual patients at the request of a physician when there is strong scientific rationale and dire unmet need.
We have developed a process that is as streamlined as possible to maximally support physicians and their patients.
Compassionate use in histiocytosis
Ongoing research evaluating our investigational first-in-class ERK 1/2 inhibitor ulixertinib as a potential treatment for histiocytosis was born out of a compassionate use request. Histiocytosis is a rare, cancer-like condition characterized by an abnormal proliferation of a type of white blood cells called histiocytes.
A treating physician requested ulixertinib to administer as a monotherapy for multiple histiocytosis patients whose disease was not responding to other therapeutic approaches. Evaluation of the request led to providing ulixertinib under our compassionate use program. All of the patients showed some signs of clinical benefit, with some still taking ulixertinib years later.
The early efficacy signals observed in these patients inspired a multi-site investigator-initiated trial to further evaluate ulixertinib in histiocytosis, potentially unlocking hope for an even greater number of histiocytosis patients.