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.

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.

Mene Pangalos via YouTube

As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

Daniel O'Day (AP Images)

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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