Editor’s note: This story is part of our 2022 PharmaVoice 100 feature.
Our honoree: Dr. Helen Thackray
Title: Chief research and development officer, BioCryst Pharmaceuticals
The company’s focus: BioCryst is a commercial-stage rare disease company that develops oral, small molecule products where an “enzyme plays a key role in the biological pathway of the disease.”
In December 2020, BioCryst received FDA approval for its first rare disease product, Orladeyo (berotralstat), an oral, once-daily pill to prevent attacks of hereditary angioedema, an inherited disease that leads to extreme swelling as a result of recurrent accumulation of fluids outside blood vessels.
According to the company: “Ongoing development programs include BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases and BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva.”
Thackray’s biggest win: While at GlycoMimetics, Thackray oversaw the development program for rivipansel, a potential treatment for sickle cell disease. As the program lead during the phase 2 data readout, Thackray helped establish the validity of pain as a real and severe consequence of the disease, which was often not understood, leading to delays in treatment. Yet, the data revealed that once the biologic source of pain was addressed, patients no longer required opioids, confirming that the disease was responsible for the pain reported by patients.
“Keep practicing what’s hardest for you. That may be where the greatest growth potential lies.”
Dr. Helen Thackray
Chief research and development officer, BioCryst Pharmaceuticals
One of Thackray’s nominators says [the trial investigator] “used these data as an example to help him change the narrative and highlight pain as a legitimate, serious symptom of the disease. The learnings from this trial contributed to acceptance in the field that opioid use could be included as an endpoint in sickle cell disease trials, leaving a legacy and an impact. [Thackray] still reflects on this experience and is proud to have had a key role in driving this change.”
Her impact: Throughout her career, Thackray has adopted a patient-first philosophy and mindset. For her, this means “engaging with patients early on, designing every aspect of a clinical program by understanding patient needs and having a desire to meaningfully improve their quality of life. [Thackray’s] experience as a physician has been invaluable as she’s guided our team, always keeping patients at the forefront,” says a BioCryst colleague.
Thackray’s steadfast eye on the prize of delivering new therapies that reduce the “burden of the disease or burden of therapy” on patients is the cornerstone to her approach to clinical drug development. This pursuit will come into play as she looks to advance BioCryst’s pipeline and oversee ongoing pivotal studies. She is particularly determined to bring BioCryst’s small molecule solutions to the market to improve patient access.
“The convenience of a pill is something many patients prioritize when determining their treatment plans, which typically include burdensome infusions,” her nominator says.
Why she’s inspiring: “Since joining our leadership team as our chief research and development officer in March 2021 — a role we created with her in mind — I’ve seen firsthand how [Thackray] exemplifies the rare executive who successfully combines scientific acumen, drug development insight and clinical expertise with true empathy and an intense desire to help patients,” her nominator says. “[Thackray] gained this unique perspective through her training as a pediatrician at Children’s National Medical Center, where she still holds a position as an assistant clinical professor in pediatrics, and ultimately her transition into a seasoned biotech executive who leads R&D programs in rare disease. From my earliest conversations with [Thackray], I understood that her experiences as a pediatrician and clinical researcher compelled her to seek a career in biopharma, particularly in rare disease.”
Thackray’s hallmark as an effective leader is grounded in her willingness to take the time to invest in her team; she encourages colleagues to step up and speak up, and brings out their innate strengths to grow the organization, her nominator writes. She is also a dedicated mentor and actively offers guidance and advice to the next generation of leaders.
She is generous with her time and expertise in innovative drug development outside of BioCryst as well. Thackray was recently named to the board of ImmunoGen, an immuno-oncology company, which is developing next-generation antibody-drug conjugates (ADCs) to help advance its pipeline. Additionally, she is involved with several professional organizations, including the American Society of Hematology, the American Society of Clinical Oncology and the American Academy of Pediatrics.
In her own words: “I was drawn to the life sciences because of the nature of the work: building something — a company or a program — based on medical science, patient need and creativity,” Thackray says.
Editor’s note: In 2019, GlycoMimetics and its development partner Pfizer suspended development of rivipansel due to the phase 3 trial not meeting its key endpoints.