No­var­tis' late-stage block­buster push gets ma­jor boost with an­oth­er 'break­through' for MET ther­a­py

No­var­tis has just re­ceived a ma­jor boost in its race with Pfiz­er to de­vel­op a ther­a­py for a no­to­ri­ous­ly treat­ment-re­sis­tant can­cer and get one of its pro­ject­ed 2020 block­buster drugs to mar­ket.

The FDA has ex­pand­ed No­var­tis’ “break­through ther­a­py” des­ig­na­tion for cap­ma­tinib (INC280) to treat a par­tic­u­lar­ly ag­gres­sive form of non-small cell lung can­cer, MET ex­on14 skip­ping-mu­tat­ed NSCLC. Pfiz­er achieved break­through ther­a­py des­ig­na­tion to treat the same dis­ease with Xalko­ri (crizo­tinib), which is al­ready in use to treat less-re­sis­tant forms of NSCLC, in March of 2018. No known treat­ment is cur­rent­ly ap­proved for the ail­ment.

No­var­tis’ des­ig­na­tion, how­ev­er, is now broad­er than Pfiz­er’s, cov­er­ing both treat­ment-naive pa­tients and those al­ready treat­ed with plat­inum-cell chemother­a­py. Pfiz­er’s was on­ly for those al­ready treat­ed with plat­inum-lev­el chemother­a­py, as of their 2018 an­nounce­ment. Cap­ma­tinib has al­so at­tained or­phan drug sta­tus.

Orig­i­nal­ly dis­cov­ered by In­cyte and li­censed to No­var­tis in 2009, cap­ma­tinib is an oral­ly bioavail­able in­hibitor that works by bind­ing to the c-Met cells that are al­tered and dri­ve tu­mor growth in a small num­ber – around 5%, per the NIH – of NSCLC can­cer pa­tients. It is one of sev­er­al in­hibitors that has gained trac­tion in re­cent years as a vi­able treat­ment for this ther­a­py-re­sis­tant form of lung can­cer, in­clud­ing Mi­rati Ther­a­peu­tics’s gle­sa­tinib and Mer­ck KGaA’s tepo­tinib.

No­var­tis CEO Vas Narasimhan in­clud­ed the ther­a­py as one of sev­en po­ten­tial block­busters the com­pa­ny was hop­ing to bring on to the mar­ket in 2020. More broad­ly, the com­pa­ny has talked up its gene and cell ther­a­pies, pro­ject­ing this week that they will ac­count for 15-20% of its sales in the midterm, or near­ly $50 bil­lion per year – up from the pen­nies they cur­rent­ly ac­count for.

The rise has al­ready be­gun with the blood can­cer ther­a­py Kym­ri­ah and the con­gen­i­tal blind­ness treat­ment Lux­tur­na, both of which run in­to the hun­dreds of thou­sands of dol­lars in price per pa­tient.

Al­ready this year, one of its pro­ject­ed block­busters, MS drug Arz­er­ra, cleared two tri­als to put it on pace for a 2020 launch, seiz­ing head­lines from the flop of its star car­diac drug En­tresto.

If No­var­tis launch­es cap­ma­tinib, In­cyte stands to earn over half a bil­lion dol­lars in mile­stone pay­ments and roy­al­ties.

Break­through sta­tus came af­ter da­ta from No­var­tis’ in­ter­na­tion­al, 97-per­son GEOM­E­TRY mono-1 study showed what the com­pa­ny called “promis­ing” re­sults. Da­ta showed a 68% re­sponse rate from treat­ment-naive pa­tients and 40.6% from those pre­vi­ous­ly treat­ed. Sev­en of 13 pa­tients al­so saw in­tracra­nial ac­tiv­i­ty, in­clud­ing in­stances of com­plete brain le­sion heal­ing.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist partnered with Novartis, the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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Novus Ther­a­peu­tics plunges deep in­to pen­ny stock ter­ri­to­ry af­ter failed ear tri­al

After a more than 15-year run, a California-based biotech is exploring options, including a sale, after its lead experimental therapy failed an exploratory mid-stage study in patients with middle ear infections characterized by a build-up of fluid behind the eardrum.

The company, initially called Tokai Pharmaceuticals but which subsequently changed its name to Novus Therapeutics in 2017, saw its shares more than halve on Monday after the drug — OP0201— did not pass muster as an adjunct therapy to oral antibiotics in infants and children aged 6 to 24 months with acute otitis media (OM).

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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