Shat­ter­ing an old record, FDA quick­ly OKs pi­o­neer­ing, site-ag­nos­tic can­cer drug larotrec­tinib from Loxo and Bay­er

The FDA has hus­tled up a quick OK for larotrec­tinib, of­fer­ing their stamp of ap­proval for a site-ag­nos­tic can­cer drug from Loxo On­col­o­gy and their part­ners at Bay­er, a prime ex­am­ple of a new wave of on­col­o­gy drugs hit­ting the field.

The la­bel for their drug, now called Vi­t­rakvi, was the first thing to hit Twit­ter af­ter the mar­ket closed, which you can see here.

Stam­ford, CT-based Loxo $LOXO — run by CEO Josh Bilenker — is one of the trail­blaz­ers in per­son­al­ized can­cer ther­a­pies where pa­tients are grouped not by the site of tu­mor de­vel­op­ment but by ge­net­ics — an ap­proach that calls for broad­er se­quenc­ing to ID the ge­net­ic mu­ta­tions un­der­pin­ning each pa­tients’ can­cer. That re­quires lots of work to iden­ti­fy pa­tients. But they have da­ta from a small piv­otal study to back up their break­through.


Specif­i­cal­ly there were 50 pa­tients — 43 adults and 12 chil­dren and adults with TRK fu­sion can­cer — with 12% com­plete and 64% par­tial re­spon­ders. That’s a high rate of suc­cess and it qual­i­fied for a pri­or­i­ty re­view at an agency that likes to press the met­al on new drug OKs — par­tic­u­lar­ly for can­cer.

Now comes the re­al­ly hard part. To find the few thou­sand US pa­tients that could ben­e­fit will re­quire can­cer pa­tients to get their DNA se­quenced, which is un­com­mon. Then theres the price. Bay­er set the whole­sale price at just un­der $400,000 a year, putting the oral drug in the most ex­pen­sive drug cat­e­go­ry. Then there’s a liq­uid for­mu­la­tion avail­able for cer­tain chil­dren at $132,000 a year.

Bay­er is al­so of­fer­ing a mon­ey-back guar­an­tee to pay­ers with as­sur­ances that pa­tient’s out-of-pock­et ex­po­sure will usu­al­ly be lim­it­ed to small sums.

An­drew Berens at Leerink ex­pects to see the num­bers add up over the years.

We see peak Vi­t­rakvi rev­enues in the US of ~$700mn by 2030, with an ad­di­tion­al ~$375mn op­por­tu­ni­ty avail­able for next-gen­er­a­tion TRK in­hibitor LOXO-195, de­signed specif­i­cal­ly to ad­dress re­sis­tance mu­ta­tions that emerge with larotrec­tinib use.

“Its ap­proval re­flects ad­vances in the use of bio­mark­ers to guide drug de­vel­op­ment and the more tar­get­ed de­liv­ery of med­i­cine,” not­ed FDA com­mis­sion­er Scott Got­tlieb, who likes to step in when the agency breaks new ground. “We now have the abil­i­ty to make sure that the right pa­tients get the right treat­ment at the right time. This type of drug de­vel­op­ment pro­gram, which en­rolled pa­tients with dif­fer­ent tu­mors but a com­mon gene mu­ta­tion, wouldn’t have been pos­si­ble a decade ago be­cause we knew a lot less about such can­cer mu­ta­tions.”

A few months af­ter the da­ta ar­rived Bay­er bought in, hand­ing over a $400 mil­lion up­front, with $450 mil­lion in mile­stones for the de­vel­op­ment and first sale of larotrec­tinib, with an­oth­er $200 mil­lion on the ta­ble for LOXO-195. There’s al­so $500 mil­lion on the books for com­mer­cial goals.

Mer­ck was the first to score in this site-ag­nos­tic field, win­ning an FDA OK last year to use Keytru­da against tu­mors that were mi­crosatel­lite in­sta­bil­i­ty-high or char­ac­ter­ized by mis­match re­pair de­fi­cien­cy.

This is the 53rd new drug ap­proval for the FDA’s CDER, which with sev­er­al new bi­o­log­ic OKs this year has shat­tered the record to­tal of 53 drugs ap­proved in 1996. With 5 weeks to go in the year, the agency has plen­ty of time to set a brand new bar for pro­duc­tiv­i­ty in bio­phar­ma R&D.


Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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Mark Mal­lon charts post-Iron­wood course by tak­ing CEO job at NeoGe­nomics; Glax­o­SmithK­line vet Feng Ren joins In­sil­i­co as CSO

Mark Mallon steps aside at Ironwood on March 12 after close to two years at the helm, and he already has a new change of scenery squared away. Beginning April 19, Mallon takes charge as CEO of cancer-focused genetic test maker NeoGenomics out of Fort Myers, FL while his predecessor, Douglas VanOort, is retiring after 12 years as NeoGenomics’ chairman and CEO.

It’s a fresh start for Mallon after what will amount to a tumultuous 23 months as Ironwood’s chief executive. Last year was marked by trial failures that spelled double trouble, leaving the Ironwood cupboard bare: first, a Linzess reformulation for irritable bowel syndrome with diarrhea (IBS-D) in May, and then the drug IW-3718 for persistent acid reflux in September. After IW-3718’s discontinuation, Ironwood chopped its staff by 35%. On Feb. 8, Mallon announced his departure at Ironwood, with president Tom McCourt getting bumped up to interim CEO.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Covid-19 roundup: Mer­ck­'s $356M sup­ply deal on hold as FDA asks for more da­ta; FDA ap­proves Pfiz­er/BioN­Tech vac­cine stor­age at stan­dard freez­er temps

Merck is pushing back plans to supply the US government with a Covid-19 drug after the FDA asked for more data to support an emergency use authorization.

The antibody, MK-7110, had looked promising in a Phase III study conducted by OncoImmune before Merck came along and bought the biotech for $425 million. At the interim analysis, investigators looked at data from 203 patients and concluded that a single dose of the drug cut the risk of death or respiratory failure by more than 50% among severe patients. And those taking the drug had a 60% higher chance of improvement in clinical status compared to placebo.

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