Upcoming events – Phase III data for Amicus and Ocular’s FDA approval

Welcome to your weekly digest of approaching regulatory and clinical readouts. Phase III data on Zorblisa in epidermolysis bullosa are expected in the second half of the year. Amicus Therapeutics gained this asset through its acquisition of Scioderm, and since suffering setbacks with its lead project Galafold its shares are in need of a boost.

By the end of next week Ocular Therapeutix could see FDA approval of its ocular pain treatment Dextenza. Phase III results were mixed, and pipeline setbacks have rocked shares since the company’s 2014 IPO, so this group too is in need of some good news to restore investor confidence.


Epidermolysis bullosa (EB) is a rare inherited condition characterised by severe blistering of the skin from even mild physical contact. Zorblisa, also known as SD-101, has US and EU orphan drug designations, and Scioderm was the first small biotech company to gain a breakthrough designation in 2013. It is a proprietary topical formulation of allantoin, a compound found in commercially available facecreams, albeit at lower doses.

In the 130-patient phase III study subjects received either Zorblisa cream at a 6% dose or placebo applied over the entire body once daily for three months. The primary endpoint is evaluation of closure of a selected target chronic wound within two months. An open-label extension trial is offered to patients completing phase III.

In a 48-patient phase II trial two doses, 3% and 6%, were tested against placebo. The higher dose showed 67% complete target wound closure after one month, the trial's primary endpoint; however, it fell short of statistical significance. It did hit statistical significance at two months, explaining the choice of primary measure for phase III.

Amicus paid up to $947m for Scioderm last year, with the bulk tied to downstream milestones. If Zorblisa gains a priority review voucher, and this is sold on, Scioderm shareholders will receive 50% of the proceeds or $100m, whichever is less (Bristol-Myers and Amicus affirm industry's love of orphans, September 1, 2015).

There are no approved medications for EB, and treatment involves bandaging and cleaning open wounds to prevent infection, alongside pain relief. Sellside consensus forecasts from EvaluatePharma point to 2022 sales of $283m. Amicus reckons that it has a $1bn-plus sales potential, with first-in-class standing and strong support from physicians and payers.

Amicus has had various setbacks with its lead asset, the Fabry disease treatment Galafold, previously called Amigal, and shares took a 54% dive when it experienced a delay in filing last year. Perhaps Zorblisa can begin to restore investor confidence.


The PDUFA date for Ocular Therapeutix's Dextenza, also known as OTX-DP, a sustained-release dexamethasone depot inserted non-invasively into the tear ducts, is July 24.

Last year Ocular reported phase IIIa results demonstrating a statistically significant reduction in pain and inflammatory cells in the anterior chamber of the eye following cataract surgery, but a phase IIIb study missed the latter co-primary endpoint, with a high placebo response not seen in previous trials. The results could have been due to the higher rate of systemic anti-inflammatory use in the placebo arm, Cowen analysts noted at the time.

The company filed the NDA for the pain endpoint and started an additional phase III study to broaden the label to include inflammation, with a primary completion date in September.

Ocular completed its $75m IPO in July 2014, but has had a number of clinical setbacks that have knocked its valuation. Last month shares plummeted 43% when Dextenza failed in phase III for chronic allergic conjunctivitis, while last year its glaucoma project OTX-TP failed to show a difference versus timolol in phase IIb, and shares fell 52%. 

As of March the company had $95.5m in cash, sufficient to fund it through the third quarter of 2017. Consensus forecasts from EvaluatePharma peg 2022 Dextenza sales at $202m, and as this is the company’s lead pipeline asset a win needs to be secured.

Project Trial ID
Zorblisa NCT02384460
Dextenza NCT02089113

To contact the writer of this story email Joanne Fagg in London at [email protected] or follow @JoEPVantage on Twitter

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