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Silence Therapeutics higher as lead medicine candidate granted orphan drug status

Orphan drug status gives drugmakers certain benefits to encourage them to develop treatments for rare diseases which might otherwise lack sufficient profit motive
A Phase Ib trial of SLN124 is due to begin later this year

Silence Therapeutics PLC’s (LON:SLN) shares rose on Tuesday after the firm revealed that its lead candidate SLN124 has been granted orphan drug designation by European regulators.

The European Medicines Agency has concluded that SLN124 will be of “significant benefit” to people affected by β-Thalassemia – an inherited and potentially life-threatening blood disorder.

READ: Silence finance chief departs

In early studies, SLN124 was shown to lower serum iron levels and modulate tissue iron distribution in rodent models of β-Thalassemia and hereditary hemochromatosis.

“We are pleased to receive Orphan Drug Designation for our iron overload disorder candidate, SLN124, which will assist in expediting clinical development as we progress our Phase Ib trial planned to begin in H2 2019,” said chief executive David Horn Solomon.

“We believe that this innovative product offers significant promise for patients with iron overload disorders such as β-Thalassemia, MDS and hereditary hemochromatosis and we look forward to rapidly advancing SLN124 through clinical development.”

What is orphan drug status?​

Orphan drug status gives drugmakers – in this case, Silence– certain benefits in order to encourage them to develop treatments for rare diseases which might otherwise lack sufficient profit motive.

For example, it can be easier to gain marketing approval for that drug or extend the exclusivity periods, while there are often also financial advantages such as tax breaks and R&D grants.

Without these incentives, companies might not pursue a drug’s development because the R&D costs would be too expensive.

Shares push higher

In a note to clients, analysts at Peel Hunt said today’s move was “a further sign of Silence’s commitment to progressing its pipeline”.

The City broker noted that it continues to have its rating and target price for Silence Therapeutics ‘under review’ as it assesses its model and valuation approach for the company following its recent litigation resolution.

In afternoon trading, shares in Silence Therapeutics were 7.4% higher at 56.80p.

 -- Adds analyst comment, share price --

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