Pharma regulatory and development news over Christmas


Some biopharma investors might have shut down over the holiday break, but the US FDA certainly did not, handing out approvals to Novo Nordisk and Vertex, while Novartis got its Christmas present from the Japan regulator.

And, perhaps taking advantage of the festive spirit, OncoGenex and Repros slipped out news about development setbacks on which they nevertheless put a highly positive gloss. With the Nasdaq biotech index defying most expectations, and set to end the year up 35%, who can blame companies for tapping into positive investor sentiment?

December 30

OncoGenex’s bad news had already come out in April with its lead asset, custirsen, failing to show an overall survival benefit in a phase III first-line prostate cancer study.

Yesterday came the near-inevitable decision of its partner, Teva, to pull the plug on a custirsen alliance. That said, the deal termination came with a $27m payment from Teva; OncoGenex might have used as a bargaining chip the argument that it would clearly have been unethical to halt ongoing custirsen studies prematurely.

Thus the payment is intended to fund the Affinity and Enspirit trials in second-line prostate cancer and metastatic NSCLC respectively. The latter will now involve a statistical modification to give what OncoGenex termed a “more rigorous” second futility analysis, expected in the first half of 2015.

OncoGenex was already trading below cash, and on yesterday’s news the shares crept up 3%. The $27m is subject to adjustments; under an original development alliance OncoGenex is obliged to pay 30% of non-royalty revenues to Isis Pharmaceuticals, in addition to other earnouts.

December 29

For Repros’s part the positive gloss was applied to its testosterone-replacement project Androxal, which the group wants to file with the US FDA in the first quarter of next year.

Repros said it had responded to questions raised in a meeting with the agency last month, and in what sounds like damning Androxal with faint praise stated that it believed that there were no refuse-to-file issues for its NDA.

Still, major problems remain for all makers of testosterone replacements after an FDA panel recommended that the class label should be narrowed (“Low-T” makers escape calls for broad safety trials but the damage is done, September 18, 2014).

Better news for Repros came in a Texas court ruling confirming the company as the rightful owner of two key Androxal patents. The decision looks to have ended the threat of litigation from the urologist Dr Harry Fisch, who had claimed ownership of the idea behind the project.

Already at the FDA with a resubmitted filing is the Parkinson’s disease project safinamide, its originator, Newron Pharmaceuticals, said on December 29. The first US submission, made in May, had been slapped with a rare refuse-to-file letter, based on paperwork irregularities.

With the US regulator in festive spirits it pays to strike while the iron is hot. On the same day the FDA approved Vertex’s cystic fibrosis treatment Kalydeco for additional use in patients with the rare R117H mutation.

This followed a 13-2 advisory panel vote in favour, despite the product’s failure in a phase III trial in these patients. UBS analysts called the approval an incremental positive; much more important now is the imminent readout of data from a trial of Kalydeco plus VX-661 in F508del patients.

December 26

There was good news on the other side of the world too, as the Japanese ministry gave Novartis’s secukinumab its first ever approval on Boxing Day. The product is trademarked Cosentyx for treating psoriasis and psoriatic arthritis.

It has also been filed in the US and Europe, and as a biological it will face significant pricing scrutiny. Undeterred, UBS analysts called it a blockbuster that had been overshadowed by the expected mid-2015 launch of Novartis’s congestive heart failure project LCZ696.

December 23

Three days earlier the US FDA had given Novo Nordisk an early Christmas gift by approving for obesity its injectable diabetes drug liraglutide, while the Swiss biotech Acetion submitted a US filing for selexipag (Uptravi) in pulmonary arterial hypertension.

In obesity once-daily liraglutide will be sold as Saxenda, and with a reasonably clean label Bryan Garnier analysts called approval the best possible gift from the FDA. Saxenda will have to be discontinued in patients who do not respond, and considering its injection delivery and high price the analysts expect Novo to implement free sampling to gain acceptance.

Whatever doubts are building up over pharma’s pricing power, huge amounts of cash continue to pour into the sector, as seen for instance in the $48m private investment in Conkwest made by the biotech entrepreneur Patrick Soon-Shiong on Christmas Eve.

Conkwest is working on an allogeneic cancer immunotherapy based on natural killer cells modified to express a chimaeric antigen receptor (CAR). With Kite Pharma, Juno Therapeutics and Bellicum Pharmaceuticals completing massive fund-raisings for CAR T-cell approaches, cell immunotherapies look to be one of the hottest themes of 2015.

Behind such enthusiasm the Nasdaq biotech index has climbed to record highs – notwithstanding earlier assumptions that 2014 could not possibly have bettered 2013's performance. Despite the huge threat now stalking biopharma in the form of insurers’ new-found strength in drug pricing, the sector is continuing to show unprecedented amounts of resilience.

Where we will be at this time in 2015 is, of course, the 64,000-dollar question.

To contact the writer of this story email Jacob Plieth in London at or follow @JacobEPVantage on Twitter

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