High cho­les­terol con­tender Es­pe­ri­on slips on safe­ty con­cerns, stock sinks 30%

Es­pe­ri­on’s stock is slid­ing south Wednes­day morn­ing fol­low­ing news that the com­pa­ny’s high cho­les­terol drug may have some safe­ty con­cerns.

Tim Mayleben

Over­all, the com­pa­ny re­port­ed pos­i­tive re­sults from its Phase III tri­al of the drug, called be­mpe­doic acid. The ther­a­py met its pri­ma­ry end­point on safe­ty while low­er­ing pa­tients’ bad cho­les­terol by 20% over 12 weeks. But that wasn’t enough to ap­pease in­vestors, as the safe­ty da­ta raised a few eye­brows.

In a study that in­clud­ed 2,200 pa­tients, rough­ly 1% get­ting Es­pe­ri­on’s treat­ment died (the com­pa­ny says caus­es were un­re­lat­ed to the treat­ment). But on­ly 0.3% of pa­tients on the place­bo died. That might be cause for con­cern at the FDA, which tends to err on the side of cau­tion when con­sid­er­ing drugs for a large pa­tient pop­u­la­tion — and high cho­les­terol is any­thing but rare.

As long as late-stage da­ta com­ing in Sep­tem­ber are pos­i­tive, Es­pe­ri­on plans to file its NDA in ear­ly 2019. The plan is to tack­le the mid­dle of the mar­ket, of­fer­ing a treat­ment that may be su­pe­ri­or to gener­ic (and cheap) statins, but not quite as ex­pen­sive as the PC­SK9 ther­a­pies from Re­gen­eron/Sanofi and Am­gen that go for about $14,000. In con­trast, Es­pe­ri­on’s be­mpe­doic acid might go for about $4,000, if ap­proved.

“In the com­ing months, re­sults from our three re­main­ing piv­otal Phase III stud­ies are ex­pect­ed to fur­ther val­i­date the safe­ty, ef­fi­ca­cy and tol­er­a­bil­i­ty pro­file of be­mpe­doic acid and the be­mpe­doic acid/eze­tim­ibe com­bi­na­tion pill, de­fin­i­tive­ly es­tab­lish­ing these once-dai­ly oral ther­a­pies as con­ve­nient and com­ple­men­tary to ex­ist­ing treat­ments for the 13 mil­lion peo­ple in the US with ath­er­o­scle­rot­ic car­dio­vas­cu­lar dis­ease,” said Tim Mayleben, Es­pe­ri­on pres­i­dent and CEO, in a state­ment.

Es­pe­ri­on’s stock $ES­PR is down 30% in pre-mar­ket trad­ing, sell­ing at $49.70 per share as of press time.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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