Friday Five – The pharma week in review

Is Trump backing government drug price negotiations?

US President Donald Trump has once again cited ambitions to reduce US drug prices, although details of how this could be achieved remain scarce.

On Wednesday, however, democrat representative Elijah Cummings said that Trump has endorsed a plan to repeal legislation that prevents Medicare from negotiating drug prices. If passed, a new measure would allow the government to negotiate with the pharmaceutical industry directly when procuring drugs supplied through Medicare Part D. In a statement the White House said it was hoping to use bipartisan support to make prescription pharmaceuticals more affordable, but would not provide more specific details.

Significantly, Cummings' proposal would not only allow the government to negotiate prices on behalf of Medicare, but establish a formulary, or list of covered products, which could significantly enhance its bargaining power. Trump has cited his willingness to work with democrats to reduce drug prices and Cummings described the President as "enthusiastic." Trump's main challenge will be convincing republicans to back the bill.

Deals of note – Vertex, Sanofi, Novo Nordisk?

Vertex Pharmaceuticals announced an agreement this week to acquire Concert Pharmaceuticals' experimental cystic fibrosis ​drug​​ CTP-656 ​under a deal ​potentially ​worth as much as $250 million​, including a cash payment of $160 million​. The drugmaker noted that the CFTR potentiator ​​is a longer-acting version of its cystic fibrosis drug Kalydeco, which has been approved in several markets for patients who carry specific mutations in the CFTR gene.

Vertex is paying a relative pittance to acquire CTP-656 and in the process has removed a competitive threat, while potentially providing an important boost to its own in-house cystic fibrosis combinations – ViewPoints: Vertex kills two birds with one deal.

Sanofi and AstraZeneca announced they will co-develop and commercialise MEDI8897, a next-generation monoclonal antibody treatment for the prevention of respiratory syncytial virus (RSV). The deal is interesting given Sanofi's previous decision to opt-out of co-developing a rival, albeit earlier-stage, RSV therapy discovered by long-term collaborator Regeneron Pharmaceuticals – ViewPoints: Sanofi's deal for AstraZeneca's MEDI8897 may speak volumes about Regeneron's competing RSV mAb.

Diabetes specialist Novo Nordisk, which recently outlined a new R&D strategy designed to broaden its long-term therapeutic focus, is rumoured to be interested in acquiring Global Blood Therapeutics. Speaking to Reuters on Thursday, CEO Lars Fruergaard Jorgensen reiterated the company is seeking "external innovation," primarily through bolt on deals.

The expert view

FirstWord's IAV desk is always looking for views from key opinion leaders (KOLs), physicians in the field and other industry experts…

A leading oncology KOL explains that Kite Pharmaceuticals must now move quickly to finalise its commercial plans for the CAR-T therapy axicabtagene ciloleucel, which he expects to be approved by the FDA based on newly released Phase II data.

Our separate poll of 62 oncologists illustrates broad support for axicabtagene ciloleucel, based on efficacy data from Kite's ZUMA-1 study and what appear to be some improvements in the product's toxicity profile. Cost and identifying the most suitable patients for treatment with CAR-T therapy are the most pressing concerns among oncologists who expect to have access to these types of product.

In response to recently top-line data from Roche's APHINITY study – showing that adjuvant treatment with the combination of Perjeta, Herceptin and chemotherapy achieved a significant reduction in the risk of recurrence of invasive disease or death in people with HER2-positive early breast cancer compared to Herceptin and chemotherapy alone – we polled 66 oncologists to ascertain what level of additional risk reduction will be deemed clinically meaningful, adoption rates if clinically meaningful data are presented at the ASCO annual meeting, comfort levels with biosimilar Herceptin and whether the clinical opportunity for Puma Biotechnology's neratinib in the extended adjuvant setting is now redundant.

FirstWord hosted an expert call this week with an assistant professor of medicine/director of a medical-surgical intensive care unit to discuss top-line results from the Phase III ATHOS-3 trial of La Jolla Pharmaceutical's LJPC-501, and what they might mean for the treatment of catecholamine-resistant hypotension. The critical care specialist concluded that the devil will be in detailed results for LJPC-501 as a possible therapy for refractory hypotension.

Following recent research by FirstWord's Therapy Trends team, US payers suggest biosimilar adoption will continue to occur slowly.

An executive change at Bristol-Myers Squibb

Bristol-Myers Squibb CEO Giovanni Caforio played up the forward thinking behind appointment of Thomas Lynch as the company's new chief scientific officer this week, but it is difficult to shake entirely the feeling that recent events may have sped up the retirement of his predecessor Francis Cuss. Bristol-Myers Squibb has lost a firm grip on what appeared to be leadership of the market for immuno-oncology treatments in the lucrative field of non-small-cell lung cancer. Notably, Caforio was also quick to note that Lynch "has made significant contributions to the field of targeted therapies for lung cancer."

ViewPoints: Bristol-Myers Squibb concedes more ground in PD-1 lung cancer race

An analysis of Bristol-Myers Squibb's late- to early-stage R&D pipeline by analysts at Deutsche Bank this week suggests the company needs to quickly get its PD-1 inhibitor Opdivo back on track in the lung cancer market. They forecast minimal revenue contribution from products not yet on the market until 2024.


TG Therapeutics reported positive data from its Phase III study of ublituximab and Imbruvica in chronic lymphocytic leukaemia (CLL) – ViewPoints: A short-term win, but can TG Therapeutics' ublituximab make long-term gains in the CLL market?

Ionis Pharmaceuticals' pivotal Phase III APPROACH study of volanesorsen met its primary endpoint of reducing triglyceride levels in patients with familial chylomicronaemia syndrome – ViewPoints: Ionis' volanesorsen hits Phase III endpoint but safety questions limit outlook.

Karyopharm Therapeutics announced that at a planned interim analysis the Phase II SOPRA study evaluating selinexor in relapsed/refractory acute myeloid leukaemia (AML) did not reach statistical significance for overall survival (OS), the study's primary endpoint – ViewPoints: Why Karyopharm won't cry in its Cheerios about selinexor miss in AML study.

Seattle Genetics confirmed that the FDA has lifted the clinical hold announced in December on Phase I trials of vadastuximab talirine in AML – ViewPoints: Seattle quickly moves Mylotarg successor back into the clinic.

Merck KGaA announced that for the treatment of first-line non-small-cell lung cancer, completion of the JAVELIN Lung 100 trial – which is evaluating the PD-L1 inhibitor avelumab as monotherapy setting – has slipped from August 2017 to April 2019. The study has been upsized from 420 to 1095 patients and the primary endpoint has been amended to include both progression free survival and OS. Furthermore, both endpoints will be evaluated in "high PD-L1 positive" patients, versus all PDL1+ patients in the previous protocol. 

MorphoSys confirmed that its partner Roche plans to initiate a new pivotal Phase III programme for gantenerumab in patients with prodromal to mild Alzheimer's disease. Gantenerumab is a monoclonal antibody directed against beta amyloid. MorphoSys has been informed that Roche intends to commence preparations for two studies, which it expects to begin later this year. Recent quarterly updates from Roche indicate that dose-escalation studies for gantenerumab have been ongoing. With a different mechanism of action to crenezumab – another antibody Roche is developing in late-stage trials for Alzheimer's disease – management has maintained for some time that development of both products will continue concurrently.

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