In a squeak­er, FDA Ad­Comm votes for an OK of Cem­pra’s an­tibi­ot­ic

Bri­an Sko­r­ney, Baird an­a­lyst

Cem­pra $CEMP just bare­ly squeaked through an Ad­Comm re­view for its pro­posed new an­tibi­ot­ic solithromycin on Fri­day, with a slim ma­jor­i­ty of 7 to 6 cast­ing votes in its fa­vor on the key ques­tion of whether the ben­e­fits out­weighed the ob­vi­ous tox­i­c­i­ty risks as­so­ci­at­ed with their prod­uct.

Past safe­ty is­sues as­so­ci­at­ed with telithromycin (Ketek) clear­ly weighed on all the mem­bers, but with grow­ing re­sis­tance to ex­ist­ing an­tibi­otics be­com­ing an ever-grow­ing pub­lic health cri­sis, there were enough ‘yes’ votes to get this one over the line.

Next stop: The FDA has to make the for­mal de­ci­sion, and it’s like­ly to con­sid­er this vote as a vir­tu­al split, leav­ing it in the po­si­tion of ei­ther throw­ing up the stop sign, or sign­ing on with a lot of strings at­tached to an ap­proval.

The com­mit­tee fre­quent­ly ze­roed in on the crux of the is­sue: New an­tibi­otics are bad­ly need­ed and solithromycin al­so met the piv­otal end­point on non-in­fe­ri­or­i­ty. But the an­timi­cro­bial drugs ad­vi­so­ry group had to bal­ance that against trou­bling signs of liv­er tox­i­c­i­ty that could harm pa­tients. And an­a­lysts were left skep­ti­cal that Cem­pra, even if it gets a for­mal OK, would be able to ef­fec­tive­ly mar­ket it. Not­ed Baird’s Bri­an Sko­r­ney:

(A)s we out­lined in our down­grade note, mar­ket­ing re­stric­tions are like­ly to be so bur­den­some, if ap­proved, based on pan­elists con­cerns over drug-in­duced liv­er in­jury and the unan­i­mous re­quests to re­strict and mon­i­tor it, the com­mer­cial po­ten­tial could be neg­li­gi­ble. Al­though we ex­pect the stock to give up some of its loss­es on short cov­er­ing, we con­tin­ue to see the soli op­por­tu­ni­ty as fun­da­men­tal­ly im­paired by safe­ty con­cerns.

How would Cem­pra track cas­es and what could they do to re­duce risks af­ter sev­er­al ad­verse events were record­ed in its tri­als?

These were three key ques­tions:

  • Did the biotech pro­vide suf­fi­cient ef­fi­ca­cy da­ta for treat­ing com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia?

The vote was unan­i­mous, 13 to 0 in fa­vor of solithromycin.

Marc Scheetz, a phar­ma­cist, felt that the an­tibi­ot­ic should be rig­or­ous­ly lim­it­ed to 5 to 7 days of ther­a­py but agreed with the rest of the com­mit­tee that the ef­fi­ca­cy da­ta were clear and that the an­tibi­ot­ic works as billed. Sev­er­al com­mit­tee mem­bers called the da­ta ad­e­quate, but not nec­es­sar­i­ly sub­stan­tial.

  • Was the risk of he­pa­tox­i­c­i­ty prop­er­ly char­ac­ter­ized?

The vote went the oth­er way, 12 to 1 vot­ing ‘no.’ Small sam­ple sizes in the tri­als drew some sharp re­spons­es and many of the com­mit­tee mem­bers were clear­ly con­cerned about how this should be dealt with in the re­al world. For sev­er­al pan­el mem­bers, the ques­tion seemed to bal­ance on whether Cem­pra should be re­quired to get a bet­ter un­der­stand­ing of the an­tibi­ot­ic in Phase III or Phase IV, af­ter an ap­proval.

Sev­er­al com­mit­tee mem­bers — though cer­tain­ly not all — leaned to­ward a Phase IV, which would re­quire a larg­er study, a point that was echoed by oth­ers.

  • Do the ben­e­fits out­weigh the risk?

This was the mon­ey shot, and it just bare­ly fell in­to Cem­pra’s fa­vor.

Tom Boy­er vot­ed no. If there was no oth­er op­tion, he’d say yes, but the risk of tox­i­c­i­ty clear­ly out­weighed the ben­e­fit.

Marc Scheetz: Yes, but… “I wish I could have vot­ed the mean, or maybe.” And its use should be se­vere­ly re­strict­ed un­til more da­ta is gath­ered.

Ellen An­drews says the bugs just won’t stop, and that’s what swayed her vote in fa­vor of an ap­proval, in spite of the risk.

The re­view to­day marks a crit­i­cal junc­ture for a hand­ful of biotechs with late-stage an­tibi­otics. The path to mar­ket is clear, as are the ob­sta­cles in the clin­ic. None of that change to­day.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

[via AP Images]

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

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.