FirstWord takes a look at some of the products which are likely to dominate industry news flow over the next 12 months...
Regeneron Pharmaceuticals and Sanofi's Dupixent (dupilumab) is due to be approved by the FDA for the treatment of moderate to severe atopic dermatitis by the end of March. It is one of two drugs scheduled to be launched this year (the other being Roche's Ocrevus; see below) forecast by sell-side analysts to generate 2022 sales in excess of $4 billion.
Analysis: KOL Views: Leading dermatologist believes that Eucrisa, Dupixent have vastly different profiles, growth prospects and Physician Views Poll Results: More positive feedback for Sanofi and Regeneron's dupilumab
Anticipated US approval of Roche's multiple sclerosis treatment Ocrevus was delayed last month – contributing to an overall decline in the number of novel drug approvals – but should be secured within the next three months. The delay has been triggered by the submission of new manufacturing data by Roche rather than issues relating to Ocrevus' safety or efficacy. The biologic has been submitted for the treatment of relapsing remitting MS, but is also poised to secure approval as the first treatment of primary progressive MS, following the publication of positive Phase III data in this setting.
Analysis: Spotlight On: How payers are prepping for new multiple sclerosis drugs from Roche and Novartis and Physician Views Poll Results: Expectation remains high for Roche's ocrelizumab in multiple sclerosis
The PCSK9 inhibitors Praluent and Repatha – marketed by Regeneron/Sanofi and Amgen, respectively – have been included on previous iterations of this list. To date, however, the biggest impact of these two drugs has been to exemplify the more stringent attitude of US payers. It is hoped, nevertheless, that positive cardiovascular outcomes data – due to be published later in 2017 – will lower barriers to adoption and broaden uptake.
Analysis: KOL Views: In wake of GLAGOV results, leading cardiologist lays out his expectations for anti-PCSK9 class and Physician Views Poll Results: 100-plus cardiologists' feedback on new PCSK9 data and future expectations for drug class
Approved by the FDA in December – some four months ahead of schedule – for the treatment of spinal muscular atrophy, Biogen and Ionis Pharmaceuticals' Spinraza will be one of the most keenly watched new drug launches of 2017. Encouraged by the agency's speed of approval and a broad label, which allows Spinraza to be used in all subtypes of SMA, Biogen and Ionis have announced a much higher-than-expected price for the drug; a bold move given continued debate around US drug costs.
In immuno-oncology, performance of the PD-(L)1 inhibitors will continue to attract considerable attention in 2017, particularly as Merck & Co.'s Keytruda is used increasingly in first-line non-small-cell lung cancer (NSCLC) patients. However, it is AstraZeneca's durvalumab (paired with the CTLA-4 inhibitor tremelimumab in the first-line NSCLC MYSTIC study) that will attract most interest; a successful outcome would propel AstraZeneca among the front runners in immuno-oncology, while negative data would cast considerable doubt on the company's aspirations in this market. Data from MYSTIC are expected sometime in the first half of 2017.
Analysis: ViewPoints: AstraZeneca’s lack of transparency on PD-L1 strategy is irking investors but clemency could be swift in coming and ViewPoints: With no crystal ball, AstraZeneca investors get a little jittery
The first Phase III data for PD-(L)1 inhibitor plus chemotherapy regimens in first-line NSCLC – from Merck and Roche – should also be published by the end of 2017, providing greater clarity on how this particular indication is likely to be segmented by treatment type in the coming years. Bristol-Myers Squibb has also hinted it may be in a position to file its PD-1/CTLA-4 combination of Opdivo/Yervoy in first-line NSCLC before the end of 2017.
CAR-T therapies should continue to attract an equal amount of enthusiasm and scrutiny in 2017 as Kite Pharma and Novartis file KTE-C19 (for r/r aggressive B-cell non-Hodgkin lymphoma) and CTL019 (for paediatric and young adult patients with r/r B-cell acute lymphoblastic leukaemia), respectively, with the FDA in early 2017. Agency evaluation of these cutting-edge therapies will be influenced by recent events at CAR-T competitor Juno Therapeutics.
Analysis: KOL Views: CAR-T cell therapies poised to make commercial debuts but one company is best positioned for initial success, according to leading medical oncologist and Spotlight On Interview: ARCH Venture’s Robert Nelsen outlines his vision for the CAR-T revolution
The number of marketed PARP inhibitors should expand from one to three in 2017; Clovis Oncology's Rubraca was approved by the FDA for the treatment of third-line BRCA mutation ovarian cancer in December and Tesaro is awaiting regulatory clearance for niraparib as a second-line therapy irrespective of biomarker status. Last month, Tesaro confirmed priority review status for its application, indicating the FDA will make a decision on approval by the end of June. AstraZeneca already markets the PARP inhibitor Lynparza as a third-line therapy for ovarian cancer with positive late-stage data as a maintenance therapy top-lined in October.
Approved last year for the treatment of Parkinson's disease psychosis, Acadia Pharmaceuticals' Nuplazid also appears to show promise in Alzheimer's disease psychosis, with positive top-line data from a Phase II study reported by the company late last year. With Acadia frequently cited as a potential acquisition target for a large cap pharma or biotech company, full data from this study and other trials is keenly awaited, including Nuplazid's potential activity against Alzheimer's disease agitation.
Analysis: KOL Views: Expert disagrees with black box for Acadia’s Nuplazid – but agrees the risk is real and ViewPoints: Pimavanserin’s success in Alzheimer’s psychosis could be unexpected upside – but extenuating circumstances abound
GW Pharmaceuticals has also been touted as a possible buyout, thanks predominantly to clinical success for lead asset Epidiolex in Dravet syndrome and Lennox-Gastaut syndrome (LGS), two rare forms of epilepsy. The most recent Phase III data released for Dravet syndrome, in December, fell short of some shareholder expectation, with Epidiolex shown to reduce the total number of seizures, but not a statistically significant reduction from baseline. Expectations for the drug nevertheless remain high, and 2017 will see full presentation of previously top-lined data, results from additional indications and US/EU filing for Epidiolex in in both LGS and Dravet syndrome.
As the search for a disease modifying Alzheimer's drug continues, Phase III data for Merck's BACE inhibitor verubecestat are due to be published in mid-2017. While expectations for this class remain relatively high (notwithstanding the terrible success rate for Alzheimer's therapies over the past decade), verubecestat is viewed as a higher risk agent as patients enrolled in the pivotal-stage EPOCH study were not pre-screened for beta-amyloid plaque levels. The impact of this data readout on sentiment towards the BACE inhibitor class should not, however, be underestimated.
With the threat of biosimilar erosion to its flagship oncology brands set to intensify over the next few years, a positive study result for the combination of Herceptin plus Perjeta in adjuvant HER2-positive breast cancer (from the APHINITY trial) is much needed by Roche. Particularly as shareholder expectations are high; in metastatic breast cancer, the addition of Perjeta to Herceptin has already improved the standard of care by a considerable margin.
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