Heart­burn: Iron­wood shares sink af­ter block­buster con­tender dis­ap­points on a key goal in PhI­Ib

Iron­wood Phar­ma­ceu­ti­cals $IR­WD CEO Pe­ter Hecht has some block­buster ex­pec­ta­tions for one of his top clin­i­cal can­di­dates. But he’s hav­ing a hard time con­vinc­ing an­a­lysts and in­vestors of that to­day af­ter rolling out a dis­ap­point­ing batch of top-line da­ta.

The biotech re­port­ed Thurs­day morn­ing that the high dose of its ex­per­i­men­tal drug — IW-3718 — for gas­troe­sophageal re­flux dis­ease (GERD) not con­trolled by avail­able meds hit the pri­ma­ry end­point on sig­nif­i­cant­ly re­duc­ing heart­burn sever­i­ty for pa­tients in a Phase IIb tri­al.

Pe­ter Hecht

Among pa­tients tak­ing a com­bi­na­tion of 1500 mg IW-3718 and a pro­ton pump in­hibitor like Nex­i­um, there was a 58% mean de­crease in heart­burn sever­i­ty. That’s high, but there was al­so a high re­sponse for the PPI-on­ly group, with a 46% drop. That 12-point sep­a­ra­tion was sta­tis­ti­cal­ly sig­nif­i­cant, says Iron­wood, with a p-val­ue of 0.04.

An­a­lysts, though, were dis­ap­prov­ing, not­ing they were look­ing for a 15-point or greater sep­a­ra­tion from the con­trol group — as guid­ed by the com­pa­ny. That is­sue came up quick­ly on the call this morn­ing as an­a­lysts weighed the short­fall. Ex­ecs called it a clear suc­cess, even with­out hit­ting that 15% mark. In ad­di­tion, the crew at Iron­wood al­so con­ced­ed that the 500 mg and 1000 mg dos­es were not suc­cess­ful against all end­points.

On an­oth­er key score, pa­tients treat­ed with IW-3718 1500 mg plus a PPI showed a mean de­crease of 55.4% from base­line in re­gur­gi­ta­tion fre­quen­cy com­pared to 37.9% in pa­tients treat­ed with a PPI alone.

Iron­wood shares slid 7% in ear­ly morn­ing trad­ing as in­vestors weighed in on the da­ta.

Ge­off Meacham at Bar­clays sum­ma­rized the dis­en­chant­ment with Iron­wood to­day, which has billed IW-3718 as a drug worth $2 bil­lion-plus per year in peak sales.

While the da­ta and val­i­da­tion of the pa­tient re­port­ed out­comes (PRO) in­stru­ment sup­port ad­vance­ment in­to the planned Phase III (an­tic­i­pat­ed start in 2H18), we think the clin­i­cal mean­ing­ful­ness of the 12-point re­duc­tion in heart­burn sever­i­ty will re­main a point of de­bate.  More­over, even though there are no treat­ment al­ter­na­tives or com­peti­tors in the re­frac­to­ry GERD cat­e­go­ry, we be­lieve that in­vestors will per­ceive IW-3718’s com­mer­cial po­ten­tial as some­what damp­ened giv­en the low­er than ex­pect­ed dif­fer­ence.  We see a 2020 launch as more like­ly now but al­so view the com­pa­ny’s >$2B peak US sales tar­get as po­ten­tial­ly am­bi­tious ab­sent more de­tailed da­ta.

David Nieren­garten at Wed­bush al­so didn’t care for the nasty sur­prise, not­ing that the Phase III now looks “long, ex­pen­sive and high-risk.”

Armed with the da­ta, Iron­wood says that they will now take the da­ta to the FDA and plan to set up a piv­otal pro­gram to get start­ed in the sec­ond half of 2018.

This drug is a key cat­a­lyst for Iron­wood, which sees this as a big ad­di­tion to its Linzess fran­chise.

“This is a re­al sci­en­tif­ic break­through,” Hecht told an­a­lysts Thurs­day morn­ing.

“The re­sults from this tri­al, demon­strat­ing en­cour­ag­ing im­prove­ments in heart­burn sever­i­ty and re­gur­gi­ta­tion, ap­pear to val­i­date our ap­proach of tar­get­ing bile acid re­flux in pa­tients with un­con­trolled GERD in ad­di­tion to sup­press­ing acid with PPIs,” said Mark Cur­rie, chief sci­en­tif­ic of­fi­cer at Iron­wood, in a state­ment. “These da­ta were con­sis­tent and ro­bust across key end­points, and re­in­force our be­lief that IW-3718 may lead to mean­ing­ful symp­tom re­lief for pa­tients with un­con­trolled GERD.”

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

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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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

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. 

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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.