Roche (rhhby) presents encouraging data on sma drug risdiplam – october 4, 2018 – zacks.com escoliosis de convexidad derecha

Roche ( RHHBY – free report) announced interim encouraging clinical data from two pivotal studies — FIREFISH and SUNFISH — on pipeline candidate, risdiplam at the 23rd international annual congress of the world muscle society in mendoza, argentina.

We note that roche is developing risdiplam, an oral SMN2 splicing modifier, as part of a collaboration with the SMA foundation and PTC therapeutics.

FIREFISH is an open-label, two-part pivotal clinical trial in infants with type 1 SMA. Part 1 was a dose escalation study in 21 infants, while part 2 is a pivotal, single-arm study of risdiplam in approximately 40 infants with type 1 SMA for 24 months, followed by an open-label extension.

The primary objective of part 1 was to assess the safety profile of risdiplam in infants and determine the dose for part 2.


The preliminary data from part 1 of the FIREFISH study showed that infants with type 1 SMA are meeting developmental milestones, including sitting without support.

Six out of 14 infants (43%) were able to sit (with or without support), including three (21%) who achieved unassisted stable sitting after eight months of treatment. Additionally, four infants (29%) demonstrated rolling to the side along with seven (50%) kicking and six (43%) achieving upright head control.

SUNFISH is a two-part, double-blind, placebo-controlled pivotal clinical trial in children and young adults (two to 25 years old) with type 2 and 3 SMA. Part 1 determined the dose for the confirmatory part 2. Roche completed enrollment in part 2 in september 2018.

The preliminary data from part 1 of the SUNFISH study show improvements in motor function in patients with type 2/3 SMA. The follow up studies are ongoing for the confirmatory part 2 portions of both the studies.

Roche is also conducting a third study, JEWELFISH, which is an open-label exploratory trial in people aged 12-60 years with type 2 or 3 SMA, who have been previously treated with SMN-targeting therapy as part of a clinical study.

Notably, biogen’s ( BIIB – free report) spinraza is already approved to treat children and adults with SMA, which reflects the prevailing competition in the market.

Roche has a strong presence in the oncology market. In particular, the company dominates the breast cancer space with strong demand for its HER2 franchise drugs, which include herceptin, perjeta and kadcyla.

The company is making efforts to develop its portfolio beyond oncology into immunology and neuroscience. Roche has more than a dozen investigational medicines in clinical development for diseases like multiple sclerosis, alzheimer`s disease, spinal muscular atrophy, parkinson`s disease, huntington`s disease and autism spectrum disorder.

Year to date, shares of roche have declined 0.1% against the industry’s growth of 11%. Roche’s legacy drugs like herceptin and mabthera are facing competition from biosimilars. Novartis ( NVS – free report) has already launched its biosimilar version of rituxan/ mabthera in europe. Amgen ( AMGN – free report) too has got its biosimilar of avastin.

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