Friday Five – The week in review

Trump nominates Gottlieb to head up FDA

US President Donald Trump's stern, oftentimes nebulous language when discussing possible changes to the drug industry and the agency in charge of regulating it has rattled the cages of bureaucrats, industry insiders, investors and regulators alike. However, his decision to nominate Scott Gottlieb as the next FDA commissioner is likely to calm many a jangled nerve.

Since taking the oath of office, Trump has on several occasions taken the pharmaceutical industry to task, primarily over what he believes is the excessively high price of drugs in the US. In doing so he has intimated that big changes could also be in store for the FDA, which the administration has in essence accused of being too slow in approving new medicines, the effect of which has been to help drive cost up.

Gottlieb's predilections certainly do not run counter to the President's desire to cut regulation, as he has been an outspoken proponent of speeding up the drug approval process for many years. But Gottlieb is also a known quantity who, as a former deputy commissioner of the FDA during the (second) Bush administration, understands the important work the agency does and is likely to pursue evolutionary – rather than revolutionary – changes within it.

Analysis – ViewPoints: Trump eschews unpredictability by tapping Gottlieb to head FDA.

If Trump has provided with one hand, however, he has taken away with another, proposing a 2018 budget, which calls for funding for the National Institutes of Health to be cut by $5.8 billion, or close to 20 percent.

PhRMA issued a response on Thursday, which prompted widespread scorn from commentators on social media, who accused industry representatives of running scared from the President. Former director of the NIH and current head of R&D at Sanofi Elias Zerhouni told Matthew Herper at Forbes that the proposed cuts would be "catastrophic," and could dramatically harm innovation in the US, while the American Society of Clinical Oncology (ASCO) issued a statement arguing the budget will 'devastate US research enterprise."

See also – Proposed US budget for 2018 sees rise in FDA user fees to over $2 billion.

 

AstraZeneca ups ante in PARP inhibitor contest

Most attention among AstraZeneca investors is focused on whether the company can leap into the highly competitive, but equally lucrative, immuno-oncology market, which will be determined by key data readouts later this year. In the meantime, other products in AstraZeneca's cancer portfolio continue to impress and this week saw presentation of positive data for the PARP inhibitor Lynparza as a maintenance treatment for BRCA mutation ovarian cancer.

With the PARP inhibitor class attracting lots of interest – and representing potential motivation for M&A, argue analysts – AstraZeneca's latest data, which suggests these products are more similar than different, could have far reaching implications.

Analysis – ViewPoints: AstraZeneca turns the screw in PARP inhibitor race and ViewPoints: Clovis gets a boost from its nemesis AstraZeneca; but could history repeat itself?

 

The expert view

FirstWord's IAV desk continues to seek out views from key opinion leaders (KOLs), physicians in the field and other industry experts regarding agenda-setting news events…

Novartis' Kisqali – a CDK4/6 inhibitor for the treatment of HR+/HER2- breast cancer – was approved by the FDA late on Monday; our snap poll of 30 US oncologists – results for FirstWord Pharma PLUS subscribers here – provides a first take on how the drug, a competitor to Pfizer's hugely successful Ibrance, may be used.

Earlier in the week, we polled 52 oncologists to gauge assessment of recently unveiled Phase III data for TG Therapeutics' ublituximab as a treatment for second-line, high risk chronic lymphocytic leukaemia (CLL) – Physician Views Poll Results: Ublituximab data impresses, but oncologists view Rituxan and Gazyva as strong, established competitors.

FirstWord also hosted an expert call this week with a professor of medicine and paediatrics to discuss the manner and extent to which new prophylactic agents like CSL's Haegarda and Shire's lanadelumab could shake up the treatment paradigm for hereditary angioedema (HAE) – KOL Views: Big changes are in store in the HAE prophylactics space, according to leading immunologist.

With data from Amgen's much anticipated FOURIER outcomes study due to be presented at the annual American College of Cardiology (ACC) meeting on Friday, FirstWord will be speaking with a KOL immediately afterwards to ascertain what detailed results mean for the anti-PCSK9 class – KOL Views: Rapid reaction to the detailed FOURIER data – where does anti-PCSK9 class go from here?

 

Reading the M&A tealeaves

With Tesaro hotly tipped as an acquisition target, it remains to be seen if positive data for AstraZeneca's Lynparza (see above) wards off potential suitors – Tesaro is developing a rival PARP inhibitor – or this week's 16 percent share price decline represents a buying opportunity. Most recently, however, the M&A spotlight has focused on Incyte with Gilead Sciences (who else) touted as an acquirer.

Regardless of whether this particular combination plays out, Incyte remains a company to watch; one that could hold a leading position in the market for a new class of backbone drug in immuno-therapy – ViewPoints: Takeover target or not – Incyte occupies an intriguing position on the immuno-oncology landscape.

 

Marathon cashes in on controversy

Controversy surrounding Marathon Pharmaceuticals' Duchenne muscular dystrophy (DMD) therapy Emflaza has not prevented acquisition of the asset by PTC Therapeutics, in a $140 million upfront deal announced on Thursday. Emflaza, otherwise known as the corticosteroid deflazacort, was approved by the FDA in February for the treatment of DMD in patients aged five years and older, irrespective of genetic mutation. While it is the first time that deflazacort has been approved for DMD in the US, the product has been available as a cheap generic for this use in many other countries for some time. This prompted controversy around Marathon's receipt of a priority review voucher from the FDA, by virtue of Emflaza's approval by the agency as a novel therapeutic, but more significantly Marathon's proposed annual list price of $89 000. In securing approval of Emflaza, Marathon has in effect halted patients' ability to import the drug cheaply from other countries; a practice that has occurred for decades.

These factors, nor requests from US senators for Marathon to justify the drug's list price and for the FDA to explain the "unusual circumstances" surrounding Emflaza's approval – notably that Marathon has secured market exclusivity for a 20-year old treatment it did no significant research and development on – have failed to dissuade PTC from acquiring the drug.

PTC management won't as yet say what they plan to charge for Emflaza, but argued in a conference call on Thursday that patient access to the drug in the US is low; a suggestion disputed by some DMD advocates. While PTC suggested Emflaza represents a "classic orphan drug launch," RBC capital markets analyst Simos Simeonidis said the company was dealing in "alternative facts," in describing Emflaza as disease modifying.

What the deal says about an ongoing pricing dispute in the US is unclear; PTC may be more desperate than most having failed to secure approval for its own DMD treatment ataluren. Marathon, on the other hand, has not only sold on the controversy it courted, but kept its priority review voucher to boot.

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