Sobi to Showcase Latest Rare Disease and Specialty Care Research at Multiple Spring 2019 Scientific Meetings

April 4, 2019 Off By BusinessWire

WALTHAM, Mass.–(BUSINESS WIRE)–Sobi, an international biopharmaceutical company transforming the lives
of people affected by rare diseases, announced that investigators will
present data from across its rare disease and specialty care portfolio
at multiple scientific meetings this Spring. These presentations will
facilitate the exchange of scientific research with the immunology,
hematology/oncology, pediatric and newborn screening communities.

Key presentations featuring Sobi medicines are listed in more detail
below:

Clinical
Immunology Society (CIS) 2019 Annual Meeting – Atlanta, GA
(April 4-7)

Emapalumab-lzsg for the Treatment of Pediatric Patients with Primary
Hemophagocytic Lymphohistiocytosis (Plenary Session #4:
Cross-Talk: PIDs from the Perspective of Other Specialties II);
presented by Michael Jordan, M.D., Cincinnati Children’s Hospital
Medical Center, on Friday, April 5, 10:00-11:30 a.m. ET

Association
of Public Health Laboratories (APHL) 2019 Newborn Screening and Genetic
Testing Symposium – Chicago, IL (April 7-10)

Update on the State of Newborn Screening for Hereditary Tyrosinemia
Type 1 (HT-1) in the United States (P-159); authored by A. Compton,
Pharm.D., B. Filas, Ph.D., and C. Satler, M.D., Sobi, Inc. Available
for viewing beginning Sunday, April 7, 3:30 p.m. CT

Pediatric
Academic Societies (PAS) Meeting – Baltimore, MD (April 27-30)

Neonatal Infectious Diseases/Immunology: Respiratory Viral Infections;
Monday, April 29; 5:45-7:30 p.m. ET
- The Prevalence of Diagnosed Chronic Lung Disease in US Infants by
  Gestational Age: Implications for RSV Policy (Poster; Kunjana
  Mavunda, M.D., Kidz Medical Services, et. al.)
- Impact of the 2014 American Academy of Pediatrics Policy on
  Respiratory Syncytial Virus Hospitalization Rates for Preterm
  Infants <29 Weeks Gestational Age at Birth: 2012–2016 (Poster;
  Mitchell Goldstein, M.D., Loma Linda University Children’s
  Hospital, et. al.)
- Severity and Costs of Respiratory Syncytial Virus and
  Bronchiolitis Hospitalization in Preterm and Term Infants Before
  and After the 2014 American Academy of Pediatrics Policy Change on
  Immunoprophylaxis (Poster; Leonard Krilov, M.D., NYU Winthrop, et.
  al.)
- Respiratory syncytial virus hospitalization rates among term and
  preterm infants before and after changes to the American Academy
  of Pediatrics policy on immunoprophylaxis: 2011–2017 (Poster;
  Jaime Fergie, M.D., Driscoll Children’s Hospital, et. al.)
- Why does the incidence of respiratory syncytial virus
  hospitalization peak during the second month and not the first
  month of life? Insights from a large US study of infants 29–35
  weeks gestational age (Poster; John DeVincenzo, M.D., University
  of Tennessee Health Science Center, et. al.)

The
American Society of Pediatric Hematology/Oncology (ASPHO) Conference
– New Orleans, LA (May 1-4)

Emapalumab in Pediatric Patients with Primary Hemophagocytic
Lymphohistiocytosis: Safety & Efficacy (ID: 2005; Session: Leukemia);
authored by Carl Allen, M.D., Texas Children’s Hospital, et. al.;
Thursday, May 2, 3:15-4:15 p.m. CT

About primary hemophagocytic lymphohistiocytosis (HLH)
Primary
hemophagocytic lymphohistiocytosis (HLH) is an ultra-rare, rapidly
progressive, often-fatal syndrome of hyperinflammation in which massive
hyperproduction of interferon gamma (IFNy) is thought to drive immune
system hyperactivation, ultimately leading to organ failures. It is
estimated that fewer than 100 cases of primary HLH are diagnosed each
year in the US, but this is believed to represent underdiagnosis.
Diagnosis is challenging due to the variability in signs and symptoms,
which may include fevers, swelling of the liver and spleen, severe low
red and white blood cell counts, bleeding disorders, infections,
neurological symptoms, organ dysfunction and organ failure. Primary HLH
can rapidly become fatal if left untreated, with median survival of less
than two months. The immediate goal of treatment is to quickly control
the hyperinflammation and to prepare for hematopoietic stem-cell
transplant. The current conventional treatment prior to transplant
includes steroids and chemotherapy, and is not specifically approved to
treat primary HLH.

About Emapalumab
Emapalumab is a monoclonal antibody (mAb)
that binds to and neutralises interferon gamma (IFNγ). In the US,
emapalumab is available as Gamifant and is indicated for pediatric
(newborn and older) and adult primary hemophagocytic lymphohistiocytosis
(HLH) patients with refractory, recurrent or progressive disease, or
intolerance to conventional HLH therapy. The FDA approval is based on
data from the phase 2/3 studies (NCT01818492 and NCT02069899).
Emapalumab is indicated to be administered through intravenous (IV)
infusion over one hour twice per week until hematopoietic stem cell
transplant. Emapalumab was developed and submitted for approval to the
FDA by Novimmune. Sobi acquired the global rights to emapalumab from
Novimmune through an exclusive licensing agreement announced in July
2018.

About hereditary tyrosinemia (HT-1)
Hereditary tyrosinemia
type 1 (HT-1) is an extremely rare but treatable hereditary disorder.
When a child has HT-1, their body lacks the enzymes needed to break down
the amino acid tyrosine. High levels of tyrosine can build up in the
blood and form toxic substances in the liver, kidneys and central
nervous system, which can cause liver, renal and neurological
complications. Approximately 1,000 persons worldwide are identified as
living with HT-1 today.

About Respiratory Syncytial Virus (RSV)
Respiratory
syncytial virus (RSV) is the most common cause of lower respiratory
tract infection (LRTI) in infants and young children worldwide, and 90%
of children are infected with RSV in the first two years of life. Of
those, up to 40% will experience a LRTI with the initial episode, making
the development and availability of effective prevention methods a
critical public health priority.¹

About Palivizumab
Palivizumab is a RSV F protein inhibitor
monoclonal antibody (mAb) that acts as a prophylaxis against serious RSV
disease.² In the US, palivizumab is available as Synagis and
is indicated for the prevention of serious LRTI caused by RSV in three
pediatric patient populations: 1) children born prematurely (at or
before 35 weeks) and who are 6 months of age or less at the
beginning of RSV season; 2) children who have a chronic lung condition
called bronchopulmonary dysplasia (BPD), that needed medical treatment
within the last 6 months, and who are 24 months of age or less at
the beginning of RSV season; OR 3) children born with certain types of
heart disease and who are 24 months of age or less at the
beginning of RSV season. It is the only medicine approved for the
prevention of serious RSV disease.³

About Sobi in North America
As the North American affiliate
of international biopharmaceutical company Sobi™, our team is committed
to Sobi’s vision of providing sustainable access to innovative therapies
and transforming the lives of people affected by rare diseases. We bring
something rare to rare diseases – a belief in the strength of focus, the
power of agility and the potential of the people we are dedicated to
serving. Our product portfolio includes multiple approved treatments,
focused on immunology and genetics/metabolism. With North American
headquarters in the Boston area, Canadian headquarters in the Toronto
area, and field sales, medical and market access representatives
spanning North America, our growing team has a proven track record of
commercial excellence. More information is available at www.sobi-northamerica.com.
For more information about Sobi, visit www.sobi.com.

References

1. Adamko DJ, Friesen M. Why does respiratory syncytial virus appear to
cause asthma? Journal of Allergy and Clinical Immunology.
2012;130(1):101-102. doi:10.1016/j.jaci.2012.05.024.
2. Synagis
(palivizumab) US prescribing information.
3. Villafana, T. et al. Expert
Review of Vaccines 2017