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On April 5, 2017, the 2nd annual Clinical and Research Excellence (CARE) Awards ceremony will return to The State Room in Boston to celebrate key accomplishments in the pharma industry. In a series of blogs leading up to the event, Pharma Intelligence will reflect on recent activities within the industry, while highlighting a few of the categories included in this year’s CARE awards. The first to be showcased is a newly introduced category for the 2017 awards: ‘Excellence in Rare Disease Drug Development’.


This award will recognize efforts of an individual, team, or company demonstrating exemplary activity while developing a drug to help combat rare diseases. In addition to acknowledging those who seek to address these truly unmet medical needs, the CARE Awards is pleased to have the Histiocytosis Association as one of our sponsors, a nonprofit committed to furthering the fight against rare diseases, specifically histiocytic disorders.


Histiocytic disorders encompass various conditions caused by an over-production of histiocytes (a certain type of white blood cells), which then leads to organ damage and tumor formation, and are classified into three major groups. The first group includes dendritic cell disorders, such as the most common histiocytic condition, Langerhans cell histiocytosis (LCH), as well as juvenile xanthogranuloma (JXG), and Erdheim-Chester Disease (ECD). Macrophage cell disorders comprise the second group, primarily hemophagocytic lymphohistiocytosis (HLH) and Rosai-Dorfman Disease (RD). The last category of malignant histiocytosis includes specific types of leukemia and malignant tumors. Although all involve histiocytes, each disease has unique attributes with varying symptoms, epidemiology, diagnosis, and treatments. One common link is the global organization of the Histiocytosis Association, which connects patient and medical communities to support those affected by these disorders as well as lead the search for key research initiatives to identify effective treatments, and ultimately, a cure.


Representing the Histiocytosis Association at the CARE Awards is Chad Rubin, who will also serve on our panel of esteemed judges. Chad has been actively involved with the association following his son’s diagnosis with LCH at just two months of age, making him only one of the estimated 1-2 cases per million newborns and young infants affected by LCH. Although Chad’s son Jack is now a perfectly healthy nine-year-old, the journey was challenging to say the least. Following his diagnosis, Jack and his mother, Kristy, spent 19 months at the Children’s Hospital of Philadelphia (CHOP) where Jack underwent numerous major procedures, such as multiple chemotherapy regimens, a splenectomy, intubations following respiratory failure, a tracheotomy, two bone marrow transplants, among other surgeries and medical interventions. Jack also contracted nosocomial infections that resulted in bacteremia and sepsis, and during the course of his disease, fought a secondary form of Hemophagocytic lymphohistiocytosis (The primary form of HLH is inherited while the secondary form is associated with a severe infection, malignancy, or rheumatologic condition). His strength and resilience throughout his path to health is an inspiration, but also a call to action for more effective treatments.


Clinical trials evaluating various treatments for LCH and HLH are ongoing, but are primarily sponsored by academic medical centers.[1] Among pharma companies, GlaxoSmithKline (GSK) previously pursued development of a LCH therapeutic with afuresertib, which was divested to Novartis in January 2015. Prior to the divestment, GSK initiated a Phase IIa trial that enrolled treatment-naïve and recurrent/refractory patients with LCH. Results for this trial were published late last year in Pediatric Blood Cancer, and although clinical activity was observed in these populations, the overall response rates in evaluable subjects did not meet the predefined Bayesian criteria for efficacy.[2] Now that the product has been divested to Novartis, it appears that the path for afuresertib within LCH may have ended as Novartis’ pipeline only lists the drug for multiple myeloma, prostate cancer, and gastric cancer.


NovImmune is currently developing a monoclonal antibody, NI-0501, for the treatment of HLH. In 2016, the FDA granted breakthrough therapy designation for the treatment of patients with primary HLH with refractory disease, or with recurrent or progressive disease during conventional therapy. NI-0501 also received PRIME designation from the EMA for the treatment of primary HLH a few months later.[3] A Phase II/III study of NI-0501 is ongoing, but initial results from the pilot Phase II trial found that parameters of HLH disease activity significantly improved overall following NI-0501 treatment. The study also showed that NI-0501 was well tolerated with no identified safety concerns, and NovImmune continues to assess the drug as a first line treatment for primary HLH.[4]


LCH and HLH are just two of many rare diseases the pharma industry is attempting to address. As of October 2016, 494 Pharmaprojects indications designated as a rare disease (per definitions from the FDA and EMA) were targeted by a total of 9,626 drugs were in development, either currently or previously active.[5]  Recent activity within this realm continues; in 2016 alone, 787 unapproved drugs targeting at least one rare disease were newly identified and captured by Pharmaprojects.[6]  Which one of these programs embody ‘Excellence in Rare Disease Drug Development’? You’ll have to wait until April 5th to find out.


For more information about the Clinical and Research Excellence (CARE) Awards, including how to enter, book a table, or sponsorship opportunities, please visit



[1]Trialtrove, January 2017


[2]Pharmaprojects, January 2017


[3]Pharmaprojects, January 2017


[4]Jordon M, et al. (2015) A Novel Targeted Approach to the Treatment of Hemophagocytic Lymphohistiocytosis (HLH) with an Anti-Interferon Gamma (IFNγ) Monoclonal Antibody (mAb), NI-0501: First Results from a Pilot Phase 2 Study in Children with Primary HLH. Blood, 126, LBA-3.


[5]Shin D. (2016) A Bird’s-Eye View of the Rare Disease Landscape. Scrip.


[6]Pharmaprojects, January 2017

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