Cor­bus brags about an­oth­er PhII suc­cess for its lead drug, but shares slide ear­ly

Yu­val Co­hen, Cor­bus CEO

Cor­bus Phar­ma­ceu­ti­cals has plucked out some pos­i­tive da­ta from its mid-stage cys­tic fi­bro­sis study. But it didn’t help its stock price this morn­ing.

While the Phase II goal for its add-on drug an­aba­sum (JBT-101 or Re­sunab) was safe­ty and tol­er­a­bil­i­ty — which the biotech says they hit — it was an ef­fi­ca­cy sig­nal they picked up in a high-dose group that drew most of the at­ten­tion. There, the com­pa­ny says, a twice dai­ly 20 mg dose of the drug trig­gered a 75% re­duc­tion in the an­nu­al­ized rate of pul­monary ex­ac­er­ba­tions re­quir­ing IV an­tibi­otics com­pared to place­bo co­hort.

While Cor­bus tout­ed the da­ta as an­oth­er sign of clin­i­cal progress, there was con­sid­er­able chat­ter on Twit­ter at­tack­ing the re­sults, which ex­plains why shares are down 13% in pre-mar­ket trad­ing.

TheStreet’s Adam Feuer­stein, a fre­quent crit­ic of Cor­bus, launched a full scale as­sault on the com­pa­ny, say­ing the da­ta shows no signs of a clin­i­cal ben­e­fit and that re­searchers just got lucky on pul­monary ex­ac­er­ba­tions with a hand­ful of cas­es to com­pare.

In this four-month study of cys­tic fi­bro­sis pa­tients, the Cor­bus drug failed to im­prove lung func­tion com­pared to a place­bo. Over­all res­pi­ra­to­ry symp­toms, mea­sured by a cys­tic fi­bro­sis pa­tient ques­tion­naire, al­so showed no dif­fer­ence be­tween an­aba­sum and place­bo.

Cor­bus CEO Yu­val Co­hen re­spond­ed:

We don’t com­ment on stock move­ments but it is note­wor­thy that there is a siz­able short po­si­tion in our stock that is prob­a­bly hav­ing a bad hair day this morn­ing…

There were a to­tal of 85 pa­tients re­cruit­ed for this study, and the high dose was test­ed in the sec­ond part of the Phase II, weeks 5 through 12, with 30 pa­tients get­ting a 20 mg sin­gle dose per day and 31 get­ting the twice dai­ly dose. Eleven pa­tients in the place­bo arm were as­signed to the drug groups and 24 pa­tients con­tin­ued on place­bo.

Five pa­tients with­drew due to ad­verse events — five on place­bo, three on an­aba­sum. And the re­searchers al­so high­light­ed some pos­i­tive bio­mark­er re­sults as well.

The lat­est batch of mid-stage da­ta fol­lows an­oth­er small Phase II for sys­temic scle­ro­sis, an au­toim­mune dis­ease of con­nec­tive tis­sue, that helped add some pos­i­tive at­ten­tion for the com­pa­ny, which has been un­der an in­tense short at­tack. Us­ing a com­pos­ite score to gauge a re­sponse on a slate of key mea­sures like lung func­tion, the com­bined drug arm in­clud­ing pa­tients on a range of dos­es of re­sunab (JBT-101) post­ed a me­di­an 33% CRISS score af­ter 16 weeks of ther­a­py, com­pared to 0% in the place­bo group. And that marks a sig­nif­i­cant ben­e­fit for pa­tients, says the biotech, with a p-val­ue of 0.044 — not a great num­ber, but with­in the mar­gin need­ed to de­clare a win.

These small stud­ies and sub­group analy­ses leave a lot to be de­sired, but Cor­bus CEO Co­hen — nev­er re­luc­tant to boast about suc­cess­es — be­lieve it helps show that the com­pa­ny is on track to de­liv­er in piv­otal stud­ies.

“The re­duc­tion in acute pul­monary ex­ac­er­ba­tions along with re­duc­tions in in­flam­ma­to­ry cells and in­flam­ma­to­ry me­di­a­tors in spu­tum demon­strate the po­ten­tial for an­aba­sum as a new in­flam­ma­tion-tar­get­ing ther­a­peu­tic in cys­tic fi­bro­sis that can broad­ly tar­get pa­tients with­out re­gard to their spe­cif­ic CFTR mu­ta­tions. The out­comes of this 16-week study in­di­cate that an­aba­sum has the po­ten­tial to ad­dress the im­por­tant un­met need for treat­ments that tar­get in­flam­ma­tion in CF,” com­ment­ed prin­ci­pal in­ves­ti­ga­tor James Chmiel.

Most of these pa­tients were al­ready tak­ing Orkam­bi, a com­bo drug from Ver­tex which has proved to have a mod­est but ef­fec­tive im­pact on CF.

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.

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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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.

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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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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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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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.