WHAT IS SARCOIDOSIS?

Sarcoidosis is an inflammatory disease that can affect any organ and involves the lungs in 90 percent of patients. The tissue biopsy of patients with sarcoidosis has a characteristic appearance under the microscope consisting of clumps of specific types of inflammatory cells, called granulomas. Granulomas are an immune response to foreign particles that enter the body, which is why sarcoidosis is thought to have an environmental cause.

Sarcoidosis may exist without symptoms but is often discovered during a routine checkup. Usually, sarcoidosis is detected by a chest radiograph (x-ray) or chest computed tomography (CT) scan, which most commonly shows enlarged lymph nodes. The disease can last just one or two years and require minimal or no treatment, or it can span decades and require interventions. The symptoms depend on which organs the disease affects. The chronic progressive form of the disease involving the lungs results in shortness of breath and decline in overall quality of life. Patients with neurologic or heart involvement have the poorest outcome.

WHOM DOES IT AFFECT?

Epidemiology, prevalence, economic burden, vulnerable populations Sarcoidosis affects people 
of all ages throughout the world, with the highest incidence in those between the ages of 20 and 40 (1). There are significant racial and gender differences in disease severity, incidence, and prevalence. Worldwide, women are more often affected than men. The highest annual incidence has been observed in northern European countries, at 5 to 40 cases per 100,000 people per year (2).

With 36 cases per 100,000 people per year, African Americans are about 3 times more likely than Caucasian Americans (11 cases per 100,000) to have sarcoidosis. African American women are the most commonly affected group, with nearly a 3 percent lifetime risk for developing sarcoidosis (1). Sarcoidosis in African Americans is also more likely to be chronic, involve several organs, and lead to death (3).

No data on the economic burden due to sarcoidosis is available. Fatigue, joint pain, and shortness of breath can lead to lost work days and necessitate transfer to a less physically demanding job. Sarcoidosis accounts for less than 1 percent of hospital admissions in the United States (4). Exposure to inorganic particles, insecticides, and moldy environments has been reported to be associated with sarcoidosis. Occupational studies have shown positive associations with metalworking, firefighting, service in the U.S. Navy, and handling of building supplies. Investigators have reported an increased incidence of sarcoidosis among New York City Fire Department rescue workers involved in the World Trade Center attacks of September 11, 2001. Socioeconomic status does not affect the risk of sarcoidosis, but low income and other financial barriers to care are associated with more severe sarcoidosis (5).

Susceptibility to sarcoidosis depends on an interaction between inherited genes and environmental exposures. Relatives of sarcoidosis patients are at higher risk of developing sarcoidosis compared to the general population. However, while family members are at higher risk, less than 1 percent of first- and second-degree relatives of sarcoidosis patients develop sarcoidosis (6). Sarcoidosis is not transmissible, and cigarette smokers are not at increased risk of developing sarcoidosis.

PATHOPHYSIOLOGY OF SARCOIDOSIS

Systemic Inflammatory Disorder characterized by:

EPIDEMIOLOGY OF SARCOIDOSIS

Prevalence: 15 -20 cases per 100,000

  • Prevalence in the U.S = ‘ 200,0001
  • Prevalence worldwide = ‘ 1.2M Active2

 Mortality: ~ 5%3

  •  Increased from 2.7 deaths per 100,000 in 1980
    to 5.5 deaths per 100,000 in 2014.

Only approved therapy: Corticosteroids and some anti-immune drugs.

UNMET NEEDS:

A reduction in the use of steroids is a significant unmet need for sarcoidosis patients

MARKET OPPORTUNITY IN SARCOIDOSIS

Comparison to Cystic Fibrosis (CF) shows
substantial market potential for Sarcoidosis

PATIENTS WORLDWIDE

70,000
CF PATIENTS

1,200,000
SARCOIDOSIS PATIENTS

PULMOZYME® APPROVED FOR CF ONLY

SM001 FOR SARCOIDOSIS

Multi-Billion Dollar Opportunity With A Single Indication

STANDARD THERAPIES & PROGRAMS IN DEVELOPMENT

STANDARD THERAPIES1

DRUG DOSAGE MAJOR TOXICITY MONITORING
Prednisone 5-40mg daily Diabetes, hypertension, weight gain, cataracts, glaucoma, Osteoporosis. Blood pressure, weight, eye examination, and bone density checks.
Hydroxychloroquine 200-400mg daily Ocular, hepatic, cutaneous. Eye examination, CBC, hepatic and renal function every 6-12 months.
Methotrexate 5-20mg weekly Hematologic, hepatotoxic, pulmonary. CBC, hepatic, and renal function every 1-3 months.
Acthar gel 40-80 units 1-2 times per week Diabetes, hypertension, weight gain, cataracts, glaucoma, Osteoporosis. Blood pressure, weight, eye examination, and bone density checks.

1 Other therapies include Azathioprine, Leflunomide, Mycophenolate, Infliximab, Adalimumab

DEVELOPMENT PROGRAMS

COMPANY DRUG TARGET DEVELOPMENT STATUS
aTyr ATYR1923 NRP2 Selective Modulator Phase 3
Novartis CMK389 IL-18 Inhibitor Phase 1 (moving to Phase 2)
SarcoMed SM001** Mitochondrial & Microbial DNA Antigens Phase 2/3 safety & POC study in Kazakhstan

**DNase 1 addresses the root cause of inflammation and is not an immune suppressor.

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