THE PIPELINE REPORT 2016 (VII): Neurology / Orphan

NEUROLOGY 

Ocrelizumab Roche 

Indication: Multiple sclerosis (Ph.III) 
What the clinical trials found: OPERA I and OPERA II studies (Ph.III) met primary endpoint with a nearly 50% reduction in annualized relapse rate over a two-year period. Overall, AEs were similar to interferon beta-1a in both studies; the most common AEs were mild to moderate infusion-related reactions. 
Credit Suisse Success Probability and inThought Comment: 60%. After 15 years in development with failures in RA and lupus, this Rituxan follow-on compound appears to be a game changer in MS. In addition to being the first drug that really works in primary proressive MS, it's on track to be a serious threat to all the current relapsing remitting MS drugs. Expected launch: 2017 (Source: Credit Suisse) 
Credit Suisse revenue forecast: $2.23 billion in global annual sales by 2020 
What the physicians are saying: Based on positive Phase II and Phase III trial results, some neurologists feel that ocrelizumab's -efficacy profile could eventually top that of Tysabri (natalizumab). What may separate ocrelizumab from other effective marketed brands is its promising positive data in primary progressive MS, a form of the disease currently with no approved treatments. While trial results also show great promise in the much more prevalent form of MS, relapsing-remitting MS, the product's benefit/risk profile may deter neurologists from calling on the agent too early and reserve it only for more progressive cases of the disease. Even if this is the case, it would be neurologists' first approved therapy for these patients. —Paul Wojciak, research director, GfK 

ORPHAN 

Kyndrisa (drisapersen) BioMarin 

Indication: Duchenne muscular dystrophy (Pre-reg.) 
What the clinical trials found: A Phase III study (DEMAND III) showed a 49m difference in the six-minute walk test (6MWT) between those on continual active treatment (n = 52) and those who had been on placebo for the first 48 weeks followed by active treatment (n = 31). AEs were consistent with previous trials. Jefferies revenue forecast: $1.06 billion in global sales by 2021 Credit Suisse Success Probability and Jefferies comment: 50%. Despite imperfect data, our due diligence indicates a likely positive outcome (we assume ~75% probability for success). 
Expected launch: 2016 (Sources: Credit Suisse; Jefferies) 
What the physicians are saying: There are few treatment options for patients and families affected by Duchenne muscular dystrophy (DMD). The FDA has granted BioMarin's drisapersen priority review status and approval is expected in 2016. [Its PDUFA date is scheduled for 12/27/15. —Ed.] Drisapersen targets exon 51 by “skipping” this genetic code and thereby allowing the creation of partially functional dystrophin, the muscle protein missing in DMD. It is estimated that 13% of DMD's population will benefit from this treatment. It's not a cure, but physicians are expected to welcome this drug into their limited armamentarium as well as Sarepta Therapeutic's eteplirsen, which has a similar mechanism of action. —Joanne French, VP, new products, GfK

Ver anterior: 
THE PIPELINE REPORT 2016 (VII): Respiratory