New drug approvals down – cause for concern?
The FDA released its New Drug Review performance report for 2016 on Wednesday, drawing attention to the fact that the number of new drug approvals in the US market this year will fall significantly below that of the previous two.
Approval of Pfizer's atopic dermatitis treatment Eucrisa, also announced on Wednesday, brings the total number of new drug and biologic approvals in 2016 to 20, compared to 41 and 45 in 2014 and 2015, respectively. There are a handful of outstanding applications that could be green-lighted by the end of December, the most notable of which is Roche's multiple sclerosis treatment Ocrevus.
There are a number of mitigating factors for the decline in 2016, says the FDA, which includes early approval of five drugs in 2015 and a higher number of complete response letters (CRL) versus recent years.
Coinciding with publication of the agency's review, Deloitte issued a new report earlier this week, which concludes that Big Pharma's return on R&D spend is dwindling at an alarming rate; a return on investment of 3.7 percent versus 10.1 percent in 2010. However, the FDA did note that it received 36 new drug applications in 2016 versus an average of 35 over the past decade. Furthermore, a number of the CRLs it issued this year have been related to manufacturing issues rather than drug-specific problems.
There are dangers in over-interpreting these data and viewing 12-month data in isolation, despite concerns raised by the Deloitte report; products such as Ocrevus and AbbVie and Roche's Venclexta – approved earlier this year for the treatment of chronic lymphocytic leukaemia – represent notable breakthroughs in their respective indications. On a related note, the FDA continues to tout the speed at which it is approving breakthrough therapies and utilising priority reviews, although there is an argument that resources to support these initiatives may be contributing to a slower overall approval rate.
Will Sanofi snag Actelion?
Securing a deal to acquire, or merge with, Actelion has proven too difficult – read expensive – for Johnson & Johnson, which confirmed this week it has walked away from negotiations with the Swiss biotech.
This decision reportedly leaves Sanofi free to make a pass at Actelion with the latest rumours indicating that talks are focused on the value of its R&D pipeline. Sanofi has been here before, most recently earlier in 2016 when it was negotiating a deal to acquire Medivation, but found itself outbid by Pfizer.
A further complication is provided by the past reluctance of CEO and founder Jean-Paul Clozel to sell Actelion. This is likely to drive up the price, which in turn could show just how desperate Sanofi is to do a deal.
What to make of events at Alexion?
Alexion Pharmaceuticals continues to argue that everything is OK, but the sudden departure of its CEO and CFO this week – amid an investigation into sales practices for the flagship Soliris franchise – would suggest otherwise.
In a note to investors, analysts at Credit Suisse said that having spoken to Alexion management the ongoing investigation is focused on sales, rather than marketing, practices – a potentially positive scenario they argue, which is less likely to impact future Soliris revenues. They caveat, however, that "while it seems the issues may not be impactful to Soliris, we really don't know and are very cautious around future surprises."
Eli Lilly becomes latest to make pricing pledge
Eli Lilly has become the latest company to assume initiative in the ongoing US drug pricing debate by announcing that it will provide its insulins at up to a 40 percent discount to those patients that pay the highest out-of-pocket costs. The discounts will be provided through a partnership with Express Scripts and will be accessed, from January, via web and mobile platforms hosted by Blink Health.
Pitched at diabetes patients who are uninsured or whom are signed to up high-deductible healthcare plans, the latter have been increasingly cited by pharma management in recent months as the reason why rebates negotiated with pharmacy benefit managers are not always passed on to end-users.
Mylan's exposure to drug pricing scrutiny this year stemmed largely from many EpiPen consumers suddenly having to pay their high deductible and Eli Lilly's scheme appears partly designed to insulate the company against any similar backlash when high deductible plans restart at the beginning of 2017.
Confirmation of the discount programme coincides with US launch of Eli Lilly's 'biosimilar' Lantus product Basaglar, which has already displaced Sanofi's basal insulin on CVS Health's preferred formulary in 2017.
Amyloid hypothesis lives on
Analysis of data from Eli Lilly's failed Phase III EXPEDITION3 study – presented at last weekend's CTAD meeting – suggests that directionally positive (but not statistically significant) effects demonstrated by the experimental Alzheimer's disease treatment solanezumab, across a variety of measures, can be taken as a positive result for those still believing in the amyloid hypothesis – not least of which being companies like Biogen, Merck & Co., Roche and even Eli Lilly, which all continue to invest in programmes targeting beta amyloid.
This view is supported by new data presented at CTAD for Biogen's aducanumab, showing a larger impact on beta-amyloid plaque burden that correlated with more robust improvements on clinical endpoints.
On the other hand, sceptics will point to aducanumab's lack of dose-concordant activity on the clinical endpoints as calling into question the trustworthiness of the results, while also reminding adherents to the amyloid hypothesis that there have been more failures going after the target than there have been successes, with Biogen's early-stage PRIME study being among the very few beacons of hope.
Continued optimism for amyloid-based programmes was reflected by a deal announced last week by Eli Lilly and AstraZeneca to expand an existing partnership to include a mAb against beta amyloid that the UK pharma has in Phase I testing right now. In fact, if any company could be forgiven for losing faith in the viability of beta amyloid as a target it would be Eli Lilly, which is fresh off its third straight Phase III disappointment with solanezumab, and yet the company is not only continuing to develop a BACE inhibitor with AstraZeneca, but it is deepening its commitment by agreeing to co-develop MEDI1814 and paying a $30-million upfront fee for the right to do so.
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