that the therapy manufactured at a different scale—a tricky proposition with biological products—is as safe and effective as the therapy being produced in small batches for clinical trials.
Ingram wasn’t clear whether the second trial represented a delay, but that’s how investors interpreted it. Shares promptly fell 9 percent amid concerns that Sarepta could lose its lead over rivals Pfizer (NYSE: [[ticker:PFE]]) and Solid Biosciences (NASDAQ: [[ticker:SLDB]]).
Ingram was perplexed. “It’s all very positive,” he told Xconomy in an interview later that day. Instead of having to run one study after the other, as Sarepta first expected, the FDA will let the company run the studies in parallel. While the commercial-scale study might run longer, Ingram says Sarepta can potentially launch the product by the end of 2020 if all goes well. What’s more, Ingram claims Sarepta can’t be leapfrogged by someone else looking for a short cut.
“Before the FDA meeting we were very confident we were in the lead,” he said. “After the meeting, that’s in concrete.”
So… the program hasn’t been set back in any way? “It’s on the same track that it was,” said Ingram, who then paused, turned to Ian Estepan, head of investor relations, shook his head and added, “I really wish I had said this at the meeting…I know, shut up.”
ALNYLAM CEO: TRUMP A “DISASTER”
Remember “getting away with murder“? And hints about Medicare “bidding”? Then president-elect, Donald Trump slammed the drug industry in his first press conference two years ago, sending chills through the 2017 J.P. Morgan gathering. (“Trump pandemonium just hit stocks,” one prominent investor told Xconomy, begging out of a meeting.)
Two years later, it’s well established that the administration has done little to nothing to hamstring the industry, even with its 2018 proposal of an international index to control drug prices. If anything, drug makers keep benefiting. Many raised prices again to ring in 2019.
The Trump administration has been good for biopharma, right? Wrong, said Alnylam Pharmaceuticals NASDAQ: [[ticker:ALNY]]) CEO John Maraganore, who is also the chair of the powerful biotech lobbying group BIO. (He said his thoughts were his own, not the official position of BIO.)
Sure, there are positives, he said. Corporate tax reform has freed up cash for drug makers. The economy keeps humming. And under Trump’s FDA commissioner Scott Gottlieb, the agency has continued a brisk pace of drug approvals that began in the second half of the Obama administration.
But for Maraganore, the positives are far outweighed by constant uncertainty. Trade problems with China. Volatile markets. Populism. Anti-immigrant stances. Complete “chaos” surrounding the drug pricing debate. “We went from a blueprint in the summer that had a comprehensive list of things to, right before the election, ‘Oh, [here’s an] international pricing index,’ which wasn’t even in the blueprint,” Maraganore said, throwing his hands up. “What sort of approach is that?”
Alnylam itself is having problems hiring qualified foreign candidates because of current immigration restrictions. Maraganore is also worried that his current employees from foreign countries “don’t know if they can feel comfortable in this country.” On the financial side, Maraganore says the self-inflicted market volatility adds more burden to drug makers like Alnylam. The firm just won FDA approval of its first product but won’t see a profit for a few years and must continue to raise cash by selling equity.
“This has been a disaster administration for this industry,” says Maraganore, “and I can’t wait until it changes.”
YANCOPOULOS AND THE INNOVATION APOCALYPSE
What a difference a year makes. Or two. At #JPM17 (was it even a hashtag?), drug executives were all a-fluster with mea culpas about pricing. Allergan (NYSE: [[ticker:AGN]]) CEO Brent Saunders led the way, telling Xconomy that his industry peers were missing a “common-sense gene” about the optics of high prices. Biogen’s (NASDAQ: [[ticker:BIIB]]) $750,000 initial price for the newly approved nusinersen (Spinraza) was sticking most notably in Saunders’s newly-bearded craw.
Around the same time, Regeneron Pharmaceuticals (NASDAQ: [[ticker:REGN]]) CEO Leonard Schleifer lambasted his peers for high prices.
This year, another Regeneron founder was in no mood for in-fighting, let alone self-reflection. Just the mention of the phrase “drug pricing” sparked a heated response from Regeneron’s president and chief scientific officer, George Yancopoulos. Prices and, in turn, the value society places on biomedical innovation are “the most important thing not just for the drug industry but for the future of society and mankind.”
But wait, there’s more. “The little pricing discussions that people are worried about now are so freaking short-sighted that it drives me crazy. Society won’t be able to
