Date: 2012-05-03
Type of information: Licensing agreement
Compound: PF-655 (synthetic siRNA)
Company: Quark Pharmaceuticals (USA) Pfizer (USA - NY) Silence Pharmaceuticals (UK)
Therapeutic area: Ophtalmological diseases
Type agreement: licensing
Action mechanism: PF-655 is a synthetic siRNA designed to inhibit the expression of Quark’s proprietary target, RTP801. Quark discovered the target gene, RTP801, using its BiFAR™ target discovery platform (PMID: 11884613). Expression of RTP801 is rapidly upregulated in response to ischemia, hypoxia and/or oxidative stress. Intravitreal injection of PF-655 in preclinical animal models of laser-induced choroidal neovascularization (CNV) led to inhibition of RTP801 expression, induction of expression of anti-angiogenic and neurotrophic factors, and subsequent reduction of CNV volume, vessel leakage and infiltration of inflammatory cells into the choroid. The mechanism of action of PF-655 is different from that of different types of VEGF inhibitors. PF-655 works in cooperation with VEGF inhibitors in the CNV model, and also decreases retinal blood vessel leakage in diabetic mice. Quark owns intellectual property covering the RTP801 gene, its RNA and protein products and sequences, specific antibodies, methods of gene inhibition and siRNA drug candidates for use in treating various indications. For PF-655 the Company has siRNA structure-related technology licenses from Silence Therapeutics and from Alnylam Pharmaceuticals.
Disease: moderate and advanced open-angle glaucoma
Details: Silence Therapeutics Partner, Quark Pharmaceuticals, extends its agreement with Pfizer to develop one of its compounds containing Silence’s AtuRNAi in a new indication. Quark Pharmaceuticals and Pfizer have amended their existing exclusive Licensing Agreement and this amendment will enable Quark to perform a Phase 2a clinical study to assess the effect of PF-655 in a new indication, looking at visual function in patients with moderate and advanced Open-Angle Glaucoma (OAG).
The OAG study will be conducted in parallel with a Phase 2b study of PF-655 in diabetic macular oedema. It will evaluate the potential of PF-655 to enhance visual function in glaucoma. The OAG study will be a Phase 2a, multi-center, double-masked, randomized, repeat dose, safety, tolerability and efficacy study in up to 108 patients with moderate and advanced OAG. In addition, Quark is currently conducting a Phase 2b study (QRK202) in DME patients testing higher doses of PF-655 alone and in combination with Lucentis® to further evaluate the safety and efficacy of PF-655 in DME and to determine the optimal dose for pivotal Phase 3 studies.
Financial terms: Under the amended agreement, should Pfizer assume development and potential commercialization of PF-655 in either or both indications following review of the Phase 2a PF-655 data, Quark will receive option exercise payments and be will eligible to receive development and regulatory milestones specific to each indication, as well as sales milestones and royalties. Quark may be eligible to receive additional total payments of up to approximately $165 million associated with development and approval of PF-655 for OAG.
Silence has previously announced that it stood to receive up to $95m from Quark in relation to its licensing agreement with Pfizer. As a result of this amendment to the Quark/Pfizer license, Silence anticipates its share of these payments could now reach $120m. Silence has previously announced the receipt of $6m from Quark in relation to this licence.
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