Li Ning (Junshi)

A 'break­through' PD-1? From Chi­na? FDA says why not for rare can­cer

PD-(L)1 in­hibitors may be the stan­dard of care and the cor­ner­stone ther­a­py for a num­ber of can­cers, but in oth­er nich­es, the FDA is still hap­py to con­sid­er them a break­through.

Late Thurs­day — and ear­ly Fri­day in Chi­na — reg­u­la­tors grant­ed the break­through des­ig­na­tion to Jun­shi Bio­sciences. It’s a first for an an­ti-PD-1 from Chi­na, said the Shang­hai-based, Hong Kong-list­ed biotech, which al­so won the do­mes­tic race by nab­bing the first Chi­nese check­point OK back in De­cem­ber 2018.

A sec­ondary list­ing on the STAR mar­ket in Shang­hai, the for­ma­tion of a joint ven­ture fo­cused on a CD39 drug and a Covid-19 an­ti­body pro­gram part­nered with Eli Lil­ly have kept Jun­shi ex­ecs busy. But with the FDA’s help they can move even faster in this par­tic­u­lar in­di­ca­tion.

First ap­proved for the sec­ond line metasta­t­ic melanoma as Tuoyi, tori­pal­imab’s BTD cov­ers its ap­pli­ca­tion in na­sopha­ryn­geal car­ci­no­ma.

“Jun­shi pri­or­i­tizes can­cer types with high preva­lence in Chi­na such as na­sopha­ryn­geal car­ci­no­ma, lung and liv­er can­cer, but we found that great un­met med­ical needs al­so ex­ist in oth­er coun­tries where pa­tients are en­dan­gered by this dead­ly dis­ease,” CEO Ning Li said in a state­ment.

By its count, there were 129,000 new cas­es of the tu­mor, which grows in the mu­cos­al ep­ithe­li­um of the na­sophar­ynx, world­wide in 2018. Al­though most pa­tients can be cured ear­ly, few are di­ag­nosed in that stage, mean­ing most pa­tients don’t start treat­ment un­til the can­cer has metas­ta­sized.

In a Phase II open-la­bel piv­otal study in­volv­ing 190 pa­tients with ad­vanced cas­es who have failed sys­temic treat­ment, the drug met the pri­ma­ry end­point on over­all re­sponse rate.

Jun­shi sub­mit­ted a sup­ple­men­tal NDA in Chi­na based on those da­ta ear­li­er this year, and a Phase III study is on­go­ing test­ing tori­pal­imab plus chemother­a­py (ver­sus chemo alone) in the front­line set­ting for re­cur­rent or metasta­t­ic na­sopha­ryn­geal car­ci­no­ma.

The FDA boost­ed tori­pal­imab’s prospects in the US by is­su­ing an or­phan drug des­ig­na­tion — con­fer­ring the perks that come with it — this March for mu­cos­al melanoma in com­bi­na­tion with Pfiz­er’s In­ly­ta. An­oth­er ODD fol­lowed for na­sopha­ryn­geal car­ci­no­ma in May.

Fo­cus­ing on rel­a­tive­ly small pa­tient pop­u­la­tions was an in­ten­tion­al strat­e­gy for Jun­shi, ac­cord­ing to Li, who spent 13 years in the FDA as an on­col­o­gy re­view­er.

Pric­ing is an­oth­er key part of the game plan, Li said in an in­ter­view with Lon­car Funds last year — high­light­ing many pre­dic­tions that the crowd­ing out of the PD-(L)1 mar­ket could lead to com­modi­ti­za­tion and much low­er prices. Even fac­tor­ing in Mer­ck’s Chi­na dis­count, Jun­shi’s Tuoyi costs just a third of Keytru­da’s price at $29,800 per year. But Li sug­gest­ed that the land­scape in Chi­na may not be di­rect­ly trans­lat­able to the US.

The low­er price in Chi­na is pro­por­tion­ate with the low­er cost of clin­i­cal de­vel­op­ment and man­u­fac­tur­ing, he said, and aligns with the main goal of get­ting on the na­tion­al drug re­im­burse­ment list:

The over­all strat­e­gy for the price set­ting is that we are shoot­ing to get on­to the na­tion­al re­im­burse­ment pro­gram as fast as pos­si­ble. 200,000 ren­min­bi is al­most three to four years of the av­er­age house­hold in­come in Chi­na even in the rich cities like Shang­hai and Bei­jing. This mar­ket is not like the Unit­ed States. The out of pock­et ex­pense in the Unit­ed States is less be­cause ei­ther in­sur­ance or Medicare or Med­ic­aid will pay a lot of it. So the com­par­i­son be­tween pric­ing in the Unit­ed States and Chi­na is not com­pa­ra­ble be­cause the out of pock­et part is key here. If you don’t get on the na­tion­al re­im­burse­ment list in Chi­na, even if the drug is one tenth of U.S. price, it does not make any sense be­cause the af­ford­abil­i­ty isn’t there giv­en the av­er­age in­come lev­el in Chi­na.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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David Hung (file photo)

Mas­ter deal­mak­er David Hung re­tools a SPAC sedan in­to a fi­nanc­ing mus­cle ve­hi­cle that leaves his can­cer start­up with $850M and a place on Wall Street

It’s only right that one of the industry’s top dealmakers just completed one of the biggest SPAC-related deals in the pipeline.

David Hung, of Medivation fame, has completed a back flip into the market, merging with EcoR1 Capital’s SPAC Panacea and landing neatly on Wall Street with an $NUVB stock ticker after filling out the blank check in his name. In addition to the $144 million held in the SPAC — provided none of the investors opt out — Hung is getting ahold of $500 million more being chipped in by a slate of institutional investors who feel that Hung could have the keys to another Medivation-style success.

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Derek Chalmers, Cara Therapeutics CEO

Cara lines up a $440M deal for US rights to its late-stage drug for se­vere itch, with $150M cash on the ta­ble

With plans afoot to file an NDA for what could be its first approved drug, Cara Therapeutics is pivoting its focus to commercialization. And Swiss company Vifor Pharma is willing to surrender up to $440 million to market the candidate in the US.

Cara $CARA CEO Derek Chalmers said an NDA submission is coming this quarter for their intravenous drug Korsuva in chronic kidney disease-associated pruritus (CKD-aP), a severe itching condition. The Stamford, CT-based biotech read out positive topline data from a Phase III pivotal study back in April, and announced plans to approach EMA regulators shortly after filing with the FDA.

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Lit­tle Zosano takes an­oth­er beat­ing as the FDA slaps down their ap­pli­ca­tion for a mi­graine patch

Zosano $ZSAN has officially come up short in its bid to develop a migraine patch.

The FDA rejected the company’s application to repurpose the triptan zolmitriptan in a new delivery system as Qtrypta, Zosano said Wednesday morning, issuing a CRL for the microneedle patch. Regulators cited inconsistent exposure levels across multiple clinical trials as the main reason for the thumbs down.

Investors did not take too kindly to the news, with Zosano shares plunking down around 25%. The company is requesting a Type A meeting to “provide clarity on the next steps for the program,” CEO Steven Lo said in a statement.

Pfizer CEO Albert Bourla (Drew Angerer/Getty Images)

Pfiz­er is on the verge of claim­ing a multi­bil­lion-dol­lar first-mover ad­van­tage with their Covid-19 vac­cine — an­a­lyst

From the beginning, Pfizer CEO Albert Bourla eschewed government funding for his Covid-19 vaccine work with BioNTech, willing to take all the $2 billion-plus risk of a lightning-fast development campaign in exchange for all the rewards that could fall its way with success. And now that the pharma giant has seized a solid lead in the race to the market, those rewards loom large.

SVB Leerink’s Geoff Porges has been running the numbers on Pfizer’s vaccine, the mRNA BNT162b2 program that the German biotech partnered on. And he sees a $3.5 billion peak in windfall revenue next year alone. Even after the pandemic is brought to heel, though, Porges sees a continuing blockbuster role for this vaccine as people around the world look to guard against a new, thoroughly endemic virus that will pose a permanent threat.

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UP­DAT­ED: CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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