Seat­tle Ge­net­ics gets ready to launch its sec­ond ADC af­ter scor­ing blad­der can­cer OK with part­ner Astel­las

Seat­tle Ge­net­ics and Astel­las have scored a tru­ly ac­cel­er­at­ed ap­proval to de­ploy their armed an­ti­body in sec­ond-line urothe­lial can­cer, adding an­oth­er promi­nent drug to the list of 2019 OKs that the FDA has been dish­ing out well ahead of sched­ule.

Pad­cev is how en­for­tum­ab ve­dotin will be known on the mar­ket, where it will be a new op­tion for pa­tients with lo­cal­ly ad­vanced or metasta­t­ic urothe­lial can­cer — the most com­mon type of blad­der can­cer — fol­low­ing treat­ment with a PD-1/L1 in­hibitor and a plat­inum-con­tain­ing chemother­a­py. Des­ig­nat­ed a “break­through” ther­a­py by the FDA, it’s the first an­ti­body-drug con­ju­gate to be ap­proved for this ag­gres­sive dis­ease, the com­pa­nies not­ed, and it’s al­so the first prod­uct Seat­tle Ge­net­ics will be bring­ing in­to the mar­ket since Ad­cetris.

Clay Sie­gall

The an­ti­body in en­for­tum­ab ve­dotin hunts down and latch­es on­to Nectin-4, a cell sur­face pro­tein high­ly ex­pressed in blad­der can­cer, then re­leas­es the chemo agent (at­tached with a link­er) to kill the cells.

The ap­proval is the cul­mi­na­tion of a co-de­vel­op­ment pact that dates back to 2007, in which Seat­tle Ge­net­ics and Astel­las are split­ting the costs and prof­its 50/50. But they are not near­ly done.

First, they need to com­plete the con­fir­ma­to­ry Phase III, a 550-pa­tient, place­bo-con­trolled tri­al mea­sur­ing over­all sur­vival as its pri­ma­ry end­point and PFS as one of the sec­on­daries. Reg­u­la­tors gave the green light af­ter re­view­ing da­ta from EV-201, a sin­gle-arm tri­al in­volv­ing 125 pa­tients, in which the con­firmed ob­jec­tive re­sponse rate hit 44% (12% com­plete, 32% par­tial). Me­di­an du­ra­tion of re­sponse was 7.6 months.

Hav­ing rolled out those num­bers at AS­CO this sum­mer, the part­ners sub­mit­ted their fil­ings for ac­cel­er­at­ed ap­proval in Ju­ly, and two months lat­er reg­u­la­tors moved the PDU­FA date up to March 2020 by grant­i­ng pri­or­i­ty re­view and cut­ting the time­frame from 10 months to 6 months.

Then there’s the push to move the ther­a­py up to front­line. Seat­tle Ge­net­ics turned up at ES­MO to show off how their drug com­bined with Keytru­da trig­gered an im­pres­sive 71% over­all re­sponse rate in front­line pa­tients — 32 of 45 pa­tients — who were in­el­i­gi­ble for chemo. The com­plete re­sponse rate came in at 13%. For ref­er­ence, Keytru­da monother­a­py was ap­proved in this set­ting two years ago with an ob­jec­tive re­sponse rate of 29%, in­clud­ing 7% com­plete re­spons­es — and there are con­cerns about low ef­fi­ca­cy for PD-L1 neg­a­tive pa­tients.

The biotech did have to con­tend with some se­ri­ous ad­verse re­ac­tions, in­clud­ing uri­nary tract in­fec­tion, cel­luli­tis, febrile neu­trope­nia, di­ar­rhea, sep­sis, acute kid­ney in­jury, dys­p­nea, and rash. The side ef­fects proved fa­tal in 3.2% of pa­tients.

But for long­time CEO Clay Sie­gall, that won’t hurt their chances in a dis­ease clear­ly still in need of new treat­ments. Seat­tle Ge­net­ics has al­ready hired the sales force for Pad­cev — and they will be gear­ing up for lots more, in­clud­ing breast can­cer drug tu­ca­tinib, now un­der pri­or­i­ty re­view.

Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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Ul­tragenyx slams the brakes on rare dis­ease study af­ter all 5 pa­tients are hit with a se­ri­ous set­back

Ultragenyx $RARE and its partners at GeneTx have been forced to halt early-stage study for a drug to treat rare cases of Angelman syndrome after all 5 of the patients being treated for the first time experienced a severe adverse event at the highest dose.

The patients in the study experienced “lower extremity weakness believed to be related to local inflammation due to GTX-102,” triggering a red light on dosing and enrollment.

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Yung Chyung, Scholar Rock CMO (Business Wire)

A dark horse en­trant in­to the spinal mus­cu­lar at­ro­phy field dou­bles its val­ue on some PhII da­ta

The last four years have seen a sudden explosion in treatments for spinal muscular atrophy, a neurodegenerative condition that once led patients — often young ones — with a grim prognosis and no options. The prognosis still isn’t rosy, but now there are three FDA-approved options, enough to make the choice of one difficult.

Now a fourth potential option has entered the mix. Today, Scholar Rock announced the results from a proof-of-concept testing their SMA drug by itself and in combination with Ionis’ Spinraza, showing that all patient cohorts improved on standard scales used for measuring motor function in people with SMA.

Three years in­to a PhI­II pro­gram for a failed Duchenne MD drug, Cataba­sis hauls down the flag and ad­mits de­feat

Three years ago, Catabasis CEO Jill Milne and the crew insisted they had found good reason for great cheer once they plumbed the data from their failed study for the Duchenne MD drug edasalonexent. Plunging into the extended open-label data, they said, you could find solid evidence of efficacy. And that justified a try in Phase III.

But they were wrong.

Monday, after the bell, the little biotech acknowledged that their pivotal attempt following the mid-stage flop was another failure. The primary, change in baseline on the North Star Ambulatory Assessment, and the secondary on timed function tests both came up short of statistical significance.

Gary Glick, Keith Flaherty

What can a biotech en­tre­pre­neur and a top sci­en­tist come up with on a cou­ple of shared flights? Well...

Just weeks before the pandemic hit the states, Gary Glick and Keith Flaherty had a chance to spend 12 hours together, talking craft and trade on board shared flights as they headed to and from a board meeting together.

It may have been the most fruitful 12 hours of Glick’s productive life. They certainly found plenty to talk about.

Glick, a well known biotech entrepreneur and inflammatory specialist, had bumped up to executive chair at IFM Therapeutics just weeks before, after running through a slate of sizable deals with some major pharma players like Novartis.