Welcome to your weekly digest of approaching regulatory and clinical readouts. Next week will see the Alzheimer’s Association International Conference in Copenhagen – one of the last scientific conferences before the traditional summer lull.
Among several mid-stage readouts, long-awaited phase II data on Roche’s crenezumab are due to be presented, but the read-across to other drug developers might be even more important than the impact on the Swiss firm. Elsewhere, Salix Pharmaceuticals' Ruconest faces a US FDA action date – put back from April – but this is unlikely to be a high-profile event for the company given its M&A move his week.
AAIC, Copenhagen, July 12–17
Roche says it is presenting phase II crenezumab data at the AAIC, though little is known about what these might entail. No abstracts are available before the meeting, but some results from two phase II studies that completed enrolment last year – Abby and Blaze – have been expected for some time.
The group has revealed little hard data on crenezumab, which is why the AAIC presentation is eagerly awaited, even though most analysts do not expect any definitive proof of the amyloid-beta hypothesis. Deutsche Bank analysts, however, do expect any data revealed to be sufficiently positive to warrant further development.
Crenezumab is a monoclonal antibody that binds to amyloid beta – an approach that has seen high-profile failures of Elan’s bapineuzumab and Eli Lilly’s solanezumab, though the latter is being taken into a phase III programme.
It is hoped the Roche candidate will be a more potent candidate, in that it seems to bind to several forms of amyloid beta, including monomer, oligomer and fibrillar, and can be dosed higher (Roche’s Alzheimer’s strategy matures as key readouts approach, May 27, 2014).
Abby, a study in 450 patients, tests intravenous and subcutaneous crenezumab against endpoints including change in cognition and clinical dementia rating. Blaze is a biomarker trial in 91 patients looking at change in brain amyloid load assessed by PET imaging at week 69 as its primary endpoint.
Should the Abby trial read out as positive, it will be seen as support for the amyloid beta hypothesis, giving hope not just to Roche’s own phase III gantenerumab but also to Lilly’s much-hyped solanezumab.
The Indiana-based group decided to keep the latter project in phase III clinical development after the failed Expedition trials showed signs of efficacy in a pre-specified group of mildly affected patients.
Backing for the amyloid beta hypothesis also should help support continued work into the beta-site APP cleavage enzyme 1 (BACE1) inhibitor class, of which AstraZeneca’s AZD3293 is a member. The UK-based group is expected to disclose biomarker data in healthy volunteers at the meeting. Merck & Co’s phase III MK-8931 and Eisai’s E2609 resulted in reduction of amyloid beta in cerebrospinal fluid, and AZD3293 would need to show similar benefit to be a candidate for advancement.
The hereditary angioedema (HAE) treatment developed by the Netherlands’ Pharming faces an FDA decision date after a delay from April. It will be the first event for US licensee Salix Pharmaceuticals since the company announced a merger with Cosmo Pharmaceuticals’ Irish-based subsidiary earlier this week.
Marketed as Rhucin outside the US, the biological treats acute disease episodes in patients who are deficient in or lack a functional plasma protein C1 inhibitor. HAE attacks are marked by swelling of the hands, feet, abdomen, face and airway, which can lead to suffocation.
If the FDA approves the Ruconest, it will be competing against Shire’s Firazyr, a product that recorded $235m in sales last year. Ruconest is forecast as a modest competitor, $41m in 2020, according to EvaluatePharma’s consensus.
Salix acquired Ruconest with its buyout of Santarus last year, a transaction driven more by the desire to buy a primary care sales force than marketed products or an R&D pipeline (Salix plays the long game with $2.6bn Santarus swoop, November 8, 2013). As EP Vantage argued at the time, Ruconest does not really fit in with the gastrointestinal focus of the combined company, and thus Salix may want to find it a home elsewhere.
FDA approval may represent the perfect catalyst for such a sale.
|Abby||Measuring cognition in 450 patients||NCT01343966|
|Blaze||Measuring biomarkers in 91 patients||NCT01397578|