Nek­tar wants every­one to stay calm as they car­ry on with NK­TR-214, but #AS­CO18 spurs a stam­pede

CHICA­GO — Bris­tol-My­ers Squibb $BMY has gone all in on Nek­tar’s $NK­TR ap­proach to amp­ing up their check­point strat­e­gy, part­ner­ing on NK­TR-214. But in­stead of tak­ing a bow at #AS­CO18 on pos­i­tive da­ta, re­searchers spent the week­end ex­plain­ing why their lat­est da­ta read­out from a small study shouldn’t be cause for pan­ic.

It’s not what they were look­ing for, to put it mild­ly.

Mary Tagli­a­fer­ri

Un­like most of the more close­ly-watched pro­grams here at AS­CO, the re­searchers in­volved nev­er of­fered a pre­view of the da­ta that came out Sat­ur­day evening. And that was a se­ri­ous mis­step, par­tic­u­lar­ly as the re­porters cov­er­ing this ses­sion strug­gled to fig­ure out what was be­ing re­port­ed — and what it meant.

As it hap­pens, in­ves­ti­ga­tors ex­e­cut­ed a two-stage tri­al process, sign­ing up a small group of pa­tients to test the drug in dif­fer­ent can­cer types, then mov­ing on to the next stage with a new group of re­cruits. Do­ing that, says Mary Tagli­a­fer­ri, the chief med­ical of­fi­cer at Nek­tar, meshed well with their strat­e­gy for jump­ing in­to late-stage stud­ies as they com­mu­ni­cat­ed with reg­u­la­tors.

“The goal,” she tells me, “was al­ways to as­sess mov­ing very quick­ly in­to Phase III.”

In­vestors, though, weren’t hap­py with the way this all came down, and they bolt­ed when the mar­ket opened, with Nek­tar’s stock plung­ing a sting­ing 32% — ex­act­ly the kind of rout that Bris­tol-My­ers (down 1.6%) doesn’t need right now as it tries to re­store con­fi­dence in its pipeline. And it kept get­ting worse lat­er in the day, with shares down 42%.

Here’s what Nek­tar has found so far.

In their melanoma project, re­searchers saw that 11 of 13 pa­tients treat­ed with a com­bi­na­tion of Op­di­vo and NK­TR-214 re­spond­ed in round 1 — which is out­stand­ing. But, when they added 15 more pa­tients, they on­ly boost­ed their re­sponse rate by 3 pa­tients. And 3 out of 15 is not out­stand­ing.

In kid­ney can­cer, the step one re­sponse rate of 64% was fol­lowed up with an over­all step 2 rate of 46%.

In­vestors hate to see a falling re­sponse rate, for any rea­son, as it in­di­cates a prob­lem that can se­ri­ous­ly af­fect a can­cer drug’s longterm fi­nan­cial suc­cess.

Twit­ter’s day-trad­ing crew picked it up from there, with all sorts of #AS­CO18 chat­ter about a tri­al dis­as­ter in the mak­ing. And as of­ten hap­pens, Twit­ter is not a great source of re­al-time in­for­ma­tion, par­tic­u­lar­ly when the com­pa­nies failed to ex­plain what the num­bers meant with an­a­lysts.

In the fol­lowup dis­cus­sion, though, Tagli­a­fer­ri of­fered the ra­tio­nale that it’s ear­ly days for the sec­ond batch­es of pa­tients and they ex­pect to see more re­spons­es with time. And they have rea­son to be­lieve that, af­ter track­ing a 46% ORR in RCC at SITC that has now grown to 71% at AS­CO.

“You’ll see the ORR go­ing up,” says Tagli­a­fer­ri, as they have a chance to col­lect mul­ti­ple scans on the sec­ond-stage pa­tients. “It s a lit­tle frus­trat­ing that peo­ple aren’t un­der­stand­ing that.”

Al­so im­por­tant, they say, is clear ev­i­dence that the com­bi­na­tion of NK­TR-214 is do­ing what they re­al­ly want­ed: Spark­ing re­spons­es in PD-L1 neg­a­tive tu­mors where Op­di­vo is not suc­cess­ful. If that all plays out in the am­bi­tious piv­otal pro­gram now un­der­way, it will eas­i­ly jus­ti­fy Bris­tol-My­ers’ record $3.6 bil­lion wa­ger.

If not….

In melanoma, re­searchers saw 5 out of 11 PD-L1 neg­a­tive pa­tients re­spond; in kid­ney can­cer it was 9 out of 17, or 53%. Those num­bers are fine.

Right now, though, they’ve cre­at­ed an at­mos­phere of doubt that will make every turn of da­ta a cat­a­lyst for the gam­blers now lin­ing up their bets for and against the com­bi­na­tion. That will ei­ther help or hurt these com­pa­nies, which are now bet­ting heav­i­ly that step 2 will ul­ti­mate­ly play out in their fa­vor.

We’ll find out more at the next SITC con­fer­ence in No­vem­ber, Tagli­a­fer­ri adds, with ab­stracts due in Au­gust.

Ven­ture Cap­i­tal as a Strate­gic Part­ner: Fu­el­ing In­no­va­tion be­yond Fi­nance

The average level of investment required for a biotech start-up to succeed is increasing every year, elevating the pressure even further on venture capital to make smart financial investments. Financial investment alone, however, does not always guarantee that exciting innovations can be transformed into real businesses that make a meaningful difference to patients.

Beyond just capital

At Astellas Venture Management (AVM) – a wholly-owned venture capital organization within Astellas, headquartered in the San Francisco Bay Area – capital is just one of the ingredients we offer to add value to our biotechnology investments and partnerships. We generally take a strategic investor approach for companies in our invested portfolio, providing access to expertise, technology and/or resources in addition to the injection of finance. An equity investment from AVM can include access to Astellas’ research and development (R&D) capabilities and expertise, and a global network of partner academic institutions and biotechnology companies, to help advance and accelerate the start-up’s innovation.

UP­DAT­ED: Ver­tex joins Mer­ck, Pfiz­er — re­vamp­ing multi­bil­lion-dol­lar tri­al strat­e­gy as biotech R&D crum­bles

You can add Pfizer, Merck and — as we found out Friday morning — Vertex to the growing list of pharma giants hitting the pause button on a range of clinical trials. But not everyone in R&D is getting a red light.

Vertex says that it’s doing its best to keep working its pipeline strategy, coming up with a plan “to enable virtual clinic visits and home delivery of study drug to ensure study continuity and medical monitoring, and to facilitate study procedures.”

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Covid-19 roundup: In­ter­cept, blue­bird and a grow­ing list of biotechs feel the pain as pan­dem­ic man­gles FDA, R&D sched­ules

Around 100 staffers at Boston area hospitals have now tested positive for Covid-19, spotlighting the growing risk that the pandemic will sideline many of the most essential workers in healthcare as caseloads peak in the US and around the globe. With more than 3,400 deaths, Spain has become the latest country to surpass the official death count attributed to the new coronavirus in China, where the outbreak originated. As of Thursday morning, confirmed global cases had crossed 470,000 and the death count eclipsed 21,000.

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Af­ter crit­ics lam­bast­ed Gilead for grab­bing the FDA's spe­cial rare drug sta­tus on remde­sivir, they're giv­ing it back

Two days after Gilead won orphan drug status for remdesivir as a potential treatment for Covid-19, they’re handing it back.

The company was slammed from several sides after Gilead reported that the FDA had come through with the special status, which comes with 7 years of market exclusivity, the waiver of FDA fees and some tax credits as well. Typically, everyone who can get orphan status lands it without much of a fuss, but Democratic presidential candidate Bernie Sanders, Public Citizen and other consumer groups were outraged.

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Mod­er­na CEO Stéphane Ban­cel out­lines a short path for emer­gency use of a coro­n­avirus vac­cine

NIAID director Anthony Fauci has left no doubts that it takes 12 to 18 months to get a new vaccine tested and in commercial use, in the best of circumstances. But in times of a global emergency — like these — maybe there’s another, faster route to follow.

In an SEC filing on Tuesday, Moderna $MRNA staked out a record-setting pathway to getting their mRNA vaccine into the frontline of the healthcare response as early as this fall. The SEC filing notes that CEO Stéphane Bancel told Goldman Sachs that an emergency use approval could allow the vaccine to go to healthcare workers and certain individuals in a matter of months — presumably provided the NIH sees the safety and efficacy data they would need from the Phase I.

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Caught in a Covid-19 mael­strom, Eli Lil­ly locks down clin­i­cal tri­als as multi­bil­lion-dol­lar R&D ops de­rail

The Covid-19 pandemic has derailed Eli Lilly’s $6 billion R&D operations.

The pharma giant reported Monday morning that it has decided to hit the brakes on most new study starts and pause enrollment for most ongoing studies. Lilly adds that it is continuing dosing for ongoing studies, “but with study-by-study consideration.”

The pandemic has severely disrupted healthcare systems around the globe, says Lilly, making it difficult or impossible to conduct studies at many research sites. And there’s no timeline for when it expects to get back on track.

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As share buy­backs come un­der scruti­ny, what's in store for the bio­phar­ma in­dus­try?

Stock buybacks are not to be permitted for companies that will be bailed out in the coronavirus stimulus package, Congressional leaders have signaled. To what degree the biopharma industry has relied on buybacks for earnings growth in recent years, and if the trend continues, are the big questions as scrutiny into the practice heightens and balance sheets weaken with the coronavirus pandemic wreaking havoc on global economies.

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A Sin­ga­pore VC rais­es $200M for a new round, but will Covid-19 pre­vent it from rais­ing the rest?

A top Singaporean biotech venture fund is nearly halfway toward its largest ever fund, but in a sign of what could be in store for VCs amid a global economic freeze, said they could face headwinds raising the other half.

Vickers Venture Partners has secured $200 million out of a targeted $500 million for its 6th fund, first announced in early 2018. They’ve given themselves 13 months to complete the financing, Vickers founder Finian Tan told Deal Street Asia, but the financial frost settling amid the Covid-19 pandemic could slow efforts.

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Strug­gling Unum ex­ecs are ready to con­sid­er a sale, merg­er or any deal that comes its way

Unum $UMRX is working its way through a survival plan of sorts.

After getting hit with a trio of FDA holds in its brief public history and triggering its second pivot to a new lead drug program while laying off 60% of the staff, the troubled penny stock biotech Unum Therapeutics has hatched new plans to secure financial backing while lining up a go-forward strategy for the company.

First, Lincoln Park Capital Fund has agreed to buy up to $25 million of the long-suffering stock, as Unum directs. And the executive team — led by CEO Chuck Wilson — has put everything on the table for consideration: a sale, acquisition, merger, licensing deal, you name it. The ACTR707 program, meanwhile, is being formally wrapped up — their second failed lead program.