Table 1.

Oral drugs in clinical development for multiple sclerosis

Drug	Phase 3 (indication, comparator)	Phase 2 (primary end point)	Safety profile (most relevant data from recent clinical experience)
Cladribine	(1) CIS, placebo (2) RRMS, placebo46 (3) RRMS, add-on to IFNβ	Not performed for oral formulation	Lymphocytopenia, exacerbation of herpes virus infection, one case of tuberculosis excerbation, single cases of malignancies (melanoma, pancreas carcinoma, and ovarian carcinoma)
Fingolimod	(1) RRMS, placebo47 (2) RRMS, placebo (3) RRMS, IFNβ48 (4) PPMS	MRI80 (median total number of gadolinium-enhanced lesions on MRI): 1.25 mg or 5 mg or placebo: 1 (P = 0.001) or 3 lesions (P = 0.006) or 5 lesions	Lymphocytopenia, exacerbation of herpes virus infection (2 fatal cases), macula edema, cardiovascular side effects
Teriflunomide	(1) CIS, placebo (2) RRMS, placebo (3) RRMS, IFNβ	MRI81 (mean number of CU active lesions per scan): 7 or 14 mg/day: reduction by 61%	GI symptoms, hepatotoxicity, low risk of pancytopenia, low risk of endogenous infections, teratogenicity
Laquinimod	(1) RRMS, placebo (2) RRMS, IFNβ	MRI82 (cumulative number of active lesions over 24 wk): reduction by 44%	Iritis and burning sensation; during follow-up acute tonsillitis, one case of breast cancer
BG12	(1) RRMS, placebo (2) RRMS, glatiramer acetate	MRI83 (total number of new gadolinium-enhancing lesions on MRI week 12 to 24): reduction by 69%	Abdominal pain, flushing, hot flush, headache, and fatigue

Abbreviations: CIS, clinically isolated syndrome; CU, combined unique; RRMS, relapsing-remitting multiple sclerosis; MRI, magnetic resonance imaging; PPMS, primary progressive multiple sclerosis; IFNβ, interferon beta.