Friday Five – Your weekly pharma review

Emicizumab is double whammy for Shire

Roche reported initial Phase III results for emicizumab in haemophilia A patients with inhibitors to factor VIII suggesting the bispecific mAb’s efficacy and convenience is good enough to take significant market share away from incumbents like Shire, which may be further stung by confirmation that all four thrombotic events observed in the study occurred in patients treated with its bypassing agent Feiba.

On December 22, emicizumab (also known as ACE910) met the primary and all secondary endpoints in the Phase III HAVEN1 trial as inhibitor patients using the agent as prophylaxis experienced significantly fewer bleeds, including those already being treated with a bypassing agent such as Shire’s Feiba and Novo Nordisk’s NovoSeven.

Physicians have said emicizumab’s once-weekly subcutaneous administration schedule is a huge improvement over intravenous alternatives (eg, Feiba or NovoSeven) that also must be dosed more frequently. As a result, Roche could get the green light to begin marketing the product as soon as next year, where it is likely to make rapid inroads among the 25 percent to 30 percent of haemophilia A patients with inhibitors. (See KOL Views: Expert excited about emicizumab’s potential in haemophilia A patients with inhibitors but caution against over-interpreting small study.)

Some analysts have predicted that the previously disclosed thrombotic events could end up slowing penetration of emicizumab, but all four occurring in patients also receiving Feiba could, if anything, end up switching more use of a bypassing agent away from the Shire product and towards NovoSeven.

Emicizumab’s prospects in the non-inhibitor haemophilia A population is less clear as the unmet need is lower, meaning the bar for approval and penetration will be higher as physicians will be more sensitive to the risk of thromboembolic events that remains an option question with the product at this point.

Findings from seven patients in a Phase I/II trial of emicizumab were encouraging but additional clinical evidence will be needed to better flesh out its profile in the non-inhibitor setting. Results from the Phase III HAVEN 3 study in non-inhibitor patients are expected in 2018.

Shire shares fell almost 2 percent on December 22.

O Actelion, Actelion, wherefore art thou, Actelion?

The competition to win Actelion’s affections appears to be taking some Hollywood-style plot twists. Johnson & Johnson was the first company to publicly vie for a tie-up with the European drugmaker but the company had reportedly dropped out as of last week when Sanofi was rumoured to be nearing a deal to buy Actelion.

Now comes word that Sanofi is yesterday’s news and Actelion has re-engaged with Johnson & Johnson, this time describing it as “exclusive communications” with the US conglomerate.

Anonymously-sourced reports pegged Johnson & Johnson’s initial offer at roughly $240-per-share, while Sanofi’s bid was believed to be somewhere in the vicinity of $275-per-share, albeit with $20 of that coming in an attached contingent value right.

It remains to be seen whether Johnson & Johnson has returned with an over-the-top bid or if Actelion, perhaps spurned by Sanofi, has come crawling back to see if the US drugmaker is still interested. Both of the pharma companies have track records of being disciplined prospective buyers, meaning an indecently lucrative proposal seems unlikely.

The big question of course is whether Johnson & Johnson and Actelion will end up consummating the relationship or if the companies will remain star-crossed lovers, thwarted perhaps by unrealistic expectations from the target company’s board or even a deep-seated reluctance to sell by its CEO Jean-Paul Clozel. (See ViewPoints: Clozel's mind-set says much about Johnson & Johnson's Actelion pursuit.)

Politicians pull punch on pricing

Anybody hoping against hope that public concern about the price of drugs might fade softly into the background in the US like it has so many times before was in for another rude awakening this week as the topic was against the source of headlines in Washington, though industry will at least be relieved that a bipartisan commission refrained from recommending in favour of the ‘nuclear option’.

Just weeks after being highlighted as a top priority by Donald Trump, Democrats confirmed on December 20 that the high cost of medicines remains one of the exceedingly few issues that politicians on both sides of the aisle may be able to find common ground on. Specifically, a group of 20 senators – 18 democrats and two independents – sent a letter to the president-elect caling for “bold administrative and legislative actions” to reduce “exorbitant” drug prices.

The following day, a Senate committee issued a report outlining the findings from an investigation into price hikes on off-patent medicines that identified single-source manufacturing and closed distribution networks of drugs for niche markets as the characteristics that allowed companies like Retrophin, Turing and Valeant to engage in price gouging.

The bipartisan group recommended a list of policy changes that they believe would prevent market manipulation by fostering increased competition, which consist mainly of ways to remove regulatory roadblocks and go so far as to suggest allowing temporary re-importation of certain products, however, the report stops short of calling for pursuing any government-led price control policies.

Biogen’s inside man

Biogen became the latest in a long line of biopharma heavyweights this year to have named a new CEO from within its own ranks rather than looking elsewhere for an injection of fresh ideas, as chief commercial officer Michel Vounatsos was tapped to succeed the departing George Scangos, effective early next year.

The promotion of Vounatsos, who joined Biogen in April after 20 years with Merck & Co., struck some investors as a bit odd after a six-month search that many thought would result in a ‘bigger name’ coming on board. Others may have been disappointed that the move lowered the chances that Biogen would look to sell itself. (See ViewPoints: New Biogen CEO may get a frosty receptor from investors, through little fault of his own.)

However, after an initial dip on December 20 the company’s shares closed the day up almost 2 percent, suggesting that the company’s decision to select a CEO with commercial experience – Scangos had a scientific background – resonated with the Street.

Vounatsos has a number of pressing tasks on his to-do list, first among them being to try and resuscitate Biogen’s multiple sclerosis (MS) franchise. On a conference call, he suggested the company would continue to look for M&A opportunities without explicitly removing the possibility that a buyer may come calling, while also indicating an interest in exploring the possibility of expanding Biogen’s neurology focus to include ophthalmology.

PARPs be poppin'

Investors have shown a keen interest in PARP inhibitors, which have been demonstrated encouraging activity in a handful of tumour types, and the class just got a tacit thumbs up from the FDA, which this week alone approved Clovis Oncology’s Rubraca (rucaparib) two months early and said an AdComm would not be necessary to discuss a newly filed application for Tesaro’s niraparib.

On December 19, Clovis said it received accelerated approval from the FDA for Rubraca to treat ovarian cancer in patients with BRCA mutations who have received two or more chemotherapies. The approval came well ahead of the February PDUFA date, which gives the company some extra time to establish the drug in a market where AstraZeneca’s Lynparza (olaparib) has the benefit of a two-year head start.

The FDA’s speedy decision may be all the more important given news on December 20 that the agency had also accepted and granted priority review to an application for Tesaro’s niraparib as second-line maintenance therapy for ovarian cancer, which would put the agent in front of Rubraca in the treatment algorithm in a setting where little is known about the utility of re-treatment with a PARP inhibitor.

Investors will inevitably begin to place bets on whether one or both of the companies may become a target for one of the many cancer-inclined pharma players, though this is likely to be a more poignant question for Clovis, which does not have additional pipeline programmes to sustain it long-term.

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