Cel­gene signs Jounce as its new biotech star in $2.6B ‘im­muno-on­col­o­gy 2.0’ pact

Over the past year Cel­gene has com­mit­ted $1.25 bil­lion in cash – plus bil­lions more in mile­stones – to a pair of ma­jor im­muno-on­col­o­gy deals with Juno and Agios. To­day, Cel­gene has cho­sen its new star part­ner for can­cer R&D, step­ping up with a rich­ly front-end­ed $2.56 bil­lion deal to col­lab­o­rate with up­start Jounce Ther­a­peu­tics’ on its lead I/O pro­gram, plus a pack­age of treat­ments in dis­cov­ery.

It’s a clas­sic Cel­gene deal, swing­ing for the fences for a po­ten­tial block­buster or two while ag­gres­sive­ly woo­ing up-and-com­ing de­vel­op­ers with an open check­book and an open mind about leav­ing lead re­search ef­forts on the drugs to their new dance part­ner. More such deals are on the way, as top ex­ecs at Cel­gene pave the way to new pacts de­signed to take them well past the first wave of check­point in­hibitors, as well as the T cell treat­ments now in late-stage de­vel­op­ment, to com­mand a lead­ing role for it­self in the field.

Cel­gene is pay­ing $225 mil­lion in the up­front, adding $36 mil­lion in eq­ui­ty and com­mit­ting up to $2.3 bil­lion in mile­stones to com­plete the deal. In turn the big biotech gets dibs on a 40% share of U.S. prof­its for Jounce’s lead drug, the pre­clin­i­cal JTX-2011, plus a 75% share for its un­named suc­ces­sor and a split on three more pro­grams. There’s al­so an opt-in avail­able on an ex­per­i­men­tal check­point pro­gram tak­ing shape at Jounce.

Once Cel­gene choos­es to opt in, the two com­pa­nies will divvy up ex­pens­es the same way they plan to share prof­its. And Cel­gene gets all ex-U.S. rev­enue with a roy­al­ty stream for Jounce.

Rob Her­sh­berg, CSO of Cel­gene

“We love deals like this,” Cel­gene CSO Rob Her­sh­berg tells me. “ICOS is the most ad­vanced in the (Jounce) pipeline, dri­ving the eco­nom­ics, but it is way be­yond a T-cell deal. Agios is a per­fect ex­am­ple, push­ing the en­ve­lope be­yond the con­ven­tion­al check­point sto­ry.”

Her­sh­berg sees Agios as the best in can­cer meta­bol­ics. And now he’s been deeply im­pressed by the team at Jounce – im­muno-on­col­o­gy rock star Jim Al­li­son is a sci­en­tif­ic co-founder – and its abil­i­ties to iden­ti­fy bio­mark­ers while clear­ly defin­ing pa­tient sub­groups most like­ly to ben­e­fit.

“The qual­i­ty of the sci­ence and the peo­ple is re­al­ly good,” says the CSO.

Cel­gene has a col­lab­o­ra­tion in place with As­traZeneca’s PD-L1 dur­val­um­ab, adds Her­sh­berg. But when the check­points can re­li­ably help about 25% of pa­tients as a sin­gle agent, Cel­gene has a big fo­cus on that oth­er 75% of the mar­ket.

Adds Her­sh­berg: “We don’t want to com­pete with 500 oth­er tri­als in com­bi­na­tion with a check­point.”

Rich Mur­ray, CEO Jounce Ther­a­peu­tics

“This is re­al­ly go­ing to al­low us to ex­pand the way we need to,” says Jounce CEO Rich Mur­ray. Cur­rent­ly op­er­at­ing with a staff of 60, Mur­ray ex­pects to add 20 more em­ploy­ees lat­er in the year, with the lead drug go­ing to its first, four-part adap­tive study that starts with safe­ty and PK and leads to the ef­fi­ca­cy stage in the sec­ond half of next year.

That lines up with a pop­u­lar strat­e­gy on can­cer drug de­vel­op­ment, where the tra­di­tion­al break­down in tri­als has now blend­ed in­to a rel­a­tive­ly fast shot at piv­otal re­sults.  The lead pro­gram will be test­ed as a sin­gle agent and in com­bo with a PD-1 check­point in­hibitor. And Jounce plans to push its own check­point pro­gram in­to the clin­ic as a “log­i­cal com­po­nent” of a larg­er on­col­o­gy ef­fort like this.

The plat­form com­pa­ny was launched by Third Rock, ring­ing up $103 mil­lion in two ven­ture rounds from in­vestors deeply im­pressed by its mar­quee sci­en­tif­ic co-founders, Al­li­son and Pam Shar­ma.

The lead drug says a lot about Jounce. It’s fo­cused on ICOS, AKA the In­ducible T cell CO-Stim­u­la­tor, a pro­tein on the sur­face of T cells Jounce — and now Cel­gene — be­lieves can spur an im­mune re­sponse against a pa­tient’s can­cer.  Im­muno-on­col­o­gy has been at the cross­roads of a mul­ti­tude of deals over the past three years, and Cel­gene clear­ly wants to be in the front end of the sec­ond wave — what Mur­ray calls im­muno-on­col­o­gy 2.0 — af­ter Bris­tol-My­ers and Mer­ck led the way with Op­di­vo and Keytru­da for check­point in­hi­bi­tion.

Cel­gene has a big fo­cus on on­col­o­gy, with fran­chis­es for Revlim­id and Abrax­ane fund­ing a large pipeline ef­fort that in­cludes pacts for Juno’s JCAR015 and JCAR017. Af­ter that comes im­munol­o­gy and in­flam­ma­tion.

Mur­ray says Jounce start­ed off 2016 with a va­ri­ety of op­tions on the ta­ble, pri­mar­i­ly re­volv­ing around an IPO or a ma­jor pact. The mar­ket hasn’t co­op­er­at­ed, yet, on the IPO, and af­ter try­ing out var­i­ous “fla­vors” of po­ten­tial deals, Jounce opt­ed for a big scoop of Cel­gene.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Roche cracks Chi­na's ADC mar­ket open as Kad­cy­la scores its first breast can­cer OK in the coun­try

Roche’s Kadcyla has become the first antibody-drug conjugate to enter the Chinese market, marking a dramatic advance for both the Swiss pharma giant and the therapeutic class.

The local arm of Roche announced the approval late Tuesday, which covers the therapy’s use in the adjuvant setting in patients with early HER-2 positive breast cancer who still have residual invasive disease after receiving paclitaxel and Herceptin as neoadjuvant treatment.

Pascal Soriot, Getty

Pas­cal So­ri­ot and As­traZeneca com­mit to car­bon neu­tral­i­ty by 2025. Where's the rest of Phar­ma?

Pascal Soriot has spent more than 20 years at the top of an industry recently found to emit more carbon than the automotive industry.

He called himself a “global citizen,” and traveled often across three-plus continents. While CEO of AstraZeneca, he commissioned a flight service — media-dubbed AstraZeneca airlines — from Cambridge to the company’s other European hub in Gothenburg. He made few, if any, public statements on the environment or his companies’ impact on it.

Fresh tri­al da­ta for­ti­fy po­si­tion of Roche's oral ther­a­py in spinal mus­cu­lar at­ro­phy bat­tle­ground

With an FDA decision date looming, Roche on Wednesday unveiled positive pivotal data on its blockbuster-bound oral spinal muscular atrophy (SMA) drug in patients with the most severe form of the muscle-wasting disease.

The FDA is set to make its decision on the therapy, risdiplam, by May 24. It is expected to compete with Biogen’s Spinraza and Novartis’ Zolgensma.

Partnered with PTC Therapeutics, the Roche drug was tested in 41 patients aged 1-7 months with type 1 SMA, a rare genetic muscle-wasting disease. The trial, dubbed FIREFISH, measured efficacy via the proportion of infants sitting without support after 12 months of treatment, and longer.

Wuhan virus out­break trig­gers in­evitable small-biotech ral­ly

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.