Loxo takes cen­ter stage at AS­CO with its ground­break­ing shot at a bio­mark­er-based can­cer drug ap­proval

CHICA­GO — Loxo On­col­o­gy made it in­to the AS­CO show­case over the week­end, boast­ing of a small but grow­ing num­ber of cas­es where chil­dren and adults with TRK fu­sion can­cer re­spond­ed to their lead drug, larotrec­tinib (LOXO-101). And they’re pack­ag­ing it in­to a new drug ap­pli­ca­tion they ex­pect to hus­tle to reg­u­la­tors ei­ther lat­er this year or ear­ly 2018, while show­ing off a next-gen drug com­ing up the pipeline that they’re al­ready claim­ing has es­tab­lished proof-of-con­cept ev­i­dence of ef­fi­ca­cy in two cas­es.

This Stam­ford, CT-based biotech $LOXO is all about hus­tle.

For a small com­pa­ny with just 35 staffers, Loxo has enor­mous am­bi­tions that in­clude shoot­ing at some ground­break­ing ad­vances in drug de­vel­op­ment. At AS­CO, the biotech boast­ed a 76% tu­mor re­sponse rate among pa­tients with mul­ti­tude of dif­fer­ent tu­mor types. Un­like a typ­i­cal can­cer drug fo­cused on the anato­my, Loxo is go­ing af­ter a bio­mark­er — tropomyosin re­cep­tor ki­nase (TRK) fu­sions — and at AS­CO it cob­bled to­geth­er re­sponse da­ta from three ear­ly-stage tri­als.

Specif­i­cal­ly there were 50 pa­tients — 43 adults and 12 chil­dren — with 12% com­plete and 64% par­tial re­spon­ders. That’s a high rate of suc­cess and it fits with their ear­li­er state­ments on progress in the clin­ic. Small groups of pa­tients like this are al­so not un­usu­al for a can­cer type shared by on­ly a few thou­sand US pa­tients.

The in­vestor crowd ate it up. Loxo’s shares spiked 51% mid­day Mon­day.

Just days ago Mer­ck won the first pi­o­neer­ing ap­proval for the ge­net­i­cal­ly de­fined use of a can­cer drug, an­oth­er boost for its glob­al star Keytru­da. Larotrec­tinib is be­ing pushed down the same path­way, but it is ex­per­i­men­tal. And Loxo is the po­lar op­po­site of a gi­ant like Mer­ck, which has been pour­ing cash in­to its well-es­tab­lished Keytru­da pro­gram in si­mul­ta­ne­ous pur­suit of dozens of tar­gets.

Josh Bilenker, Loxo On­col­o­gy

Go­ing af­ter an FDA ap­proval for both chil­dren and adults like this “has nev­er been done be­fore,” Loxo CEO Josh Bilenker tells me. But he is ab­solute­ly con­vinced that the da­ta he has is enough to form the ba­sis of a win­ning ap­pli­ca­tion.

It’s not un­usu­al these days to see bio­phar­ma com­pa­nies use ear­ly-stage da­ta to go af­ter an ac­cel­er­at­ed ap­proval at the FDA. What was once the ex­cep­tion is now the rule. But Loxo — which part­nered with Ar­ray on the pro­gram — is press­ing the en­ve­lope, us­ing small groups of pa­tients across Phase I and II tri­als to pro­vide the ev­i­dence they need that larotrec­tinib works. That strat­e­gy is nec­es­sary to achieve their goal, they say, and they have the agency’s break­through ther­a­py des­ig­na­tion to pro­vide an open door, which Bilenker says led to a meet­ing with the FDA 18 months ago that set them on this path.

As a pub­lic com­pa­ny, Loxo al­so has tout­ed — and ben­e­fit­ed — from just about every scrap of da­ta ex­ecs can lay its hands on, with new in­stances of par­tial re­spons­es used to rev up its stock price.

In Chica­go Sat­ur­day, that strat­e­gy was on full view as they not­ed the first glim­mer of ev­i­dence that its next-gen drug LOXO-195 trig­gered two par­tial re­spons­es af­ter it was used in emer­gency cas­es in­volv­ing re­sis­tance to a TRK fu­sion in­hibitor.

Cour­tesy Loxo On­col­o­gy.

If in fact they can prove that it can work against cas­es of TRK fu­sion drug re­sis­tance, their drug could be­come a reg­u­lar part of the treat­ment land­scape for pa­tients with their bio­mark­er. And they will tout any ev­i­dence of ef­fi­ca­cy they gath­er on their next trip to reg­u­la­tors.

Loxo’s mar­ket cap is up to $1.3 bil­lion as some mar­ket an­a­lysts cheer them at every stage.

Loxo, though, al­so has com­pe­ti­tion in the field. Igny­ta $RXDX has a Phase II un­der­way for en­trec­tinib for TRK-fu­sion pos­i­tive adult and pe­di­atric pa­tients. Just a cou­ple of weeks ago Igny­ta won their own break­through stays for their drug, giv­ing them the same easy ac­cess that Loxo en­joys. And not all an­a­lysts are ig­nor­ing the po­ten­tial for a matchup as these two small out­fits com­pete for the lime­light in can­cer R&D.

“The com­pa­ny guid­ed for NDAs for Trk+ and ROS1+ pts in 2018 and a U.S. launch in 2019 (MAA sub­mis­sion in 2019),” not­ed Jef­feries re­cent­ly. “The break­through des­ig­na­tion for en­trec­tinib helps lev­el the play­ing field some with LOXO-101, which re­ceived BTD in Ju­ly 2016 (NDA sub­mis­sion ex­pect­ed in late-2017 or ear­ly-2018).”

If they do make it to the mar­ket, Bilenker says Loxo plans to set up a com­mer­cial group and launch this it­self. “We own world­wide rights to the drug and that’s our plan,” he says, shrug­ging off the sug­ges­tion of a com­pa­ny sale.

It’s a big goal.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Pos­i­tive Covid-19 vac­cine da­ta? New mouse study? OWS in­clu­sion? Yep, but some­how, the usu­al tid­bits from In­ovio back­fire

You don’t go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.

Jan van de Winkel, Genmab CEO

Seat­tle Ge­net­ics, Gen­mab turn on TV for a high­light reel in cer­vi­cal can­cer — but a ri­val biotech promis­es a bet­ter show

Seattle Genetics $SGEN and their partners at Genmab $GMAB polished up some positive Phase II numbers for their antibody drug conjugate tisotumab vedotin — you can call it TV — for recurrent cervical cancer. And while they mapped out a shortcut to a potential quick approval, the big challenge for this team is being presented by a rival biotech which muscled its way into the spotlight for the same indication a year ago.

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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.