Nabri­va goes 2 for 2 in Phase III an­tibi­ot­ic pro­gram, scor­ing on a key cat­a­lyst -- but shares plunge on safe­ty wor­ries

Nabri­va Ther­a­peu­tics $NBRV has scored a sec­ond, piv­otal Phase III win for its an­tibi­ot­ic lefa­mulin in com­mu­ni­ty ac­quired bac­te­r­i­al pneu­mo­nia — set­ting up a pitch to the FDA lat­er this year and quite like­ly clear­ing a path to its first com­mer­cial launch.

Their an­tibi­ot­ic achieved non-in­fe­ri­or­i­ty com­pared to mox­i­floxacin for ear­ly clin­i­cal re­sponse (ECR) — 72 to 120 hours fol­low­ing ini­ti­a­tion of ther­a­py in the in­tent to treat pa­tient pop­u­la­tion. In this tri­al, ECR rates were 90.8% for lefa­mulin and 90.8% for mox­i­floxacin. The tri­al al­so achieved Eu­ro­pean goals out­lined for an ap­proval.

Nabri­va’s shares rock­et­ed up 24% on the news, and then re­versed course as in­vestors wres­tled over the safe­ty da­ta, falling 17% by the end of trad­ing. Di­ar­rhea proved a more ex­pan­sive con­cern than the ef­fi­ca­cy da­ta, with the an­tibi­ot­ic arm do­ing worse than moxi. Tox­i­c­i­ty is­sues grabbed in­vestors by the throat, with plen­ty of fret­ting about po­ten­tial lim­its to its use.

A cru­cial cat­a­lyst for this biotech, lefa­mulin came out of the sec­ond late-stage study look­ing iden­ti­cal to moxi, fit­ting a pro­file reg­u­la­tors re­quire for a mar­ket­ing ap­proval. If every­thing goes ac­cord­ing to plan, and gets around the safe­ty wor­ries, that will set up a launch in 2019 as the biotech seeks to carve out a large seg­ment of a big mar­ket, and not just due to drug re­sis­tance.

Col­in Broom

“I do not see this as an an­tibi­ot­ic to keep in re­serve,” CEO Col­in Broom tells me in a pre­view of the news. “It’s a short course of ther­a­py (5 days for lefa­mulin com­pared to 7 days for moxi), high­ly ef­fec­tive, with com­plete cov­er­age of the pathogens we wor­ry about….It’s re­al­ly the on­ly an­tibi­ot­ic out there that has the op­por­tu­ni­ty to be used out of the gate.”

Mak­ing that ar­gu­ment stick with pay­ers will re­quire some mod­er­a­tion on the pric­ing, he adds, with­out spelling out the num­bers. An­a­lysts will like­ly stay mind­ful that pay­ers will keep fo­cused on price, al­ways re­quir­ing cheap gener­ics when­ev­er pos­si­ble.

The biotech al­so re­port­ed that one pa­tient in their study al­so de­vel­oped C diff dur­ing an ex­tend­ed hos­pi­tal stay.

Gary Sender

Nei­ther Broom nor CFO Gary Sender are of­fer­ing their own peak sales es­ti­mates, but the CFO notes that an­a­lysts cov­er­ing the com­pa­ny have pen­cilled in es­ti­mates rang­ing from $500 mil­lion to $700 mil­lion a year — and they ex­pect those an­a­lysts to do some re­cal­cu­la­tions in their fa­vor with the lat­est batch of piv­otal da­ta.

Even now Nabri­va has a sales force of 20 work­ing the pre-com­mer­cial­iza­tion mar­ket. That will like­ly ex­pand to the 30-to-60 range, says the CEO, and even­tu­al­ly up to around 100. Broom ex­pects to line up part­ners for the ex-US mar­ket.

In do­ing so, Nabri­va may soon find it­self go­ing up against Paratek’s an­tibi­ot­ic, which al­so suc­ceed­ed for CABP and was filed for an ap­proval in Feb­ru­ary. Broom shrugs that off, though, dis­count­ing the ri­val as a broad spec­trum al­ter­na­tive that will like­ly be held in re­serve, al­low­ing lefa­mulin to push ahead in­to a broad­er mar­ket.

It won’t hurt Nabri­va that the FDA height­ened its warn­ings against the use of flu­o­ro­quinolones like maxi a cou­ple of years ago, af­ter iden­ti­fy­ing new safe­ty is­sues that in­cludes dis­abling side ef­fects in­volv­ing ten­dons, mus­cles, joints and nerves. And he adds that the com­mon­ly used z packs have be­come lit­tle bet­ter than a place­bo for about half of all cas­es of bac­te­r­i­al pneu­mo­nia.

Still, for years now the de­vel­op­ment of new an­tibi­otics has been left to small biotechs like Nabri­va, af­ter Big Phar­ma’s large­ly bowed out of a field they iden­ti­fied with nar­row mar­gins — de­spite the grow­ing num­ber of alarms from mul­ti­tude of glob­al health agen­cies over a steadi­ly ris­ing tide of drug re­sis­tance. It’s not an easy field, as a slate of re­cent clin­i­cal mishaps un­der­score. And cheap gener­ics are typ­i­cal­ly thrown at cas­es as they arise.

Broom, though, like oth­er CEOs in the field, be­lieves the eco­nom­ics of an­tibi­otics will grad­u­al­ly im­prove as more cas­es of re­sis­tance rise up. And he plans to be there with one of the new breed when it does.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.