Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a cou­ple of ma­jor reg­u­la­to­ry mile­stones for its new drug, Arde­lyx has bagged an FDA ap­proval to mar­ket Ib­srela (tena­panor) for ir­ri­ta­ble bow­el syn­drome.

The drug’s first ap­pli­ca­tion will be for IBS with con­sti­pa­tion (IBS-C), in­hibit­ing sodi­um-hy­dro­gen ex­chang­er NHE3 in the GI tract in such a way as to in­crease bow­el move­ments and de­crease ab­dom­i­nal pain. This comes on the heels of two suc­cess­ful Phase III tri­als.

But Arde­lyx, which spe­cial­izes in car­diore­nal dis­eases, has broad­er plans for their new trade­mark drug.  Last week, they un­veiled across-the-board pos­i­tive re­sults from their AM­PLI­FY study, test­ing the in­hibitor on pa­tients with chron­ic kid­ney dis­ease (CKD) with high phos­phate lev­els. And they’ve in­di­cat­ed op­ti­mism for the re­sults of a sec­ond PHREE­DOM study on pa­tients with end-stage re­nal dis­ease.

Mike Raab Arde­lyx

“With these mile­stones ac­com­plished, and the PHREE­DOM tri­al read­ing out in Q4, I have great con­fi­dence that we are well-po­si­tioned to file our NDA for hy­per­phos­phatemia next year with po­ten­tial ap­proval and launch in 2021,” Arde­lyx CEO Mike Raab said in a state­ment.

In the Phase III tri­als that fu­eled the FDA ap­proval, Arde­lyx pri­mar­i­ly ex­am­ined the por­tion of pa­tients with who re­spond­ed by the FDA de­f­i­n­i­tion: saw at least a 30% re­duc­tion in week­ly av­er­age ab­dom­i­nal pain score and, dur­ing the same week, an in­crease of at least 1 com­plete spon­ta­neous bow­el move­ment, and did those for at least 6 of the 12 weeks. In the first study, the over­all re­sponse rate was 37% com­pared to 24% in the place­bo group. In the sec­ond study, it was 27% vs 19%.

Di­ar­rhea was the most sig­nif­i­cant health risk, af­fect­ing 16% and 15% of pa­tients in each study, against 4% and 2% in the place­bo group.

An analy­sis from SVB Leerink not­ed that the place­bo-ad­just­ed re­sponse rate in these tri­als was low­er than in tri­als for a key com­peti­tor: Linzess, sold by Al­ler­gan and Iron­wood. But rates of di­ar­rhea were low­er and that if you raise the bar and look at if pa­tients re­spond­ed for 9 (rather than 6) of 12 weeks, Ib­srela out­per­forms.

“Over­all, we be­lieve these dif­fer­ences are mi­nor in the grand scheme of things and puts Ib­srela at a com­pet­i­tive po­si­tion vs on mar­ket IBS-C treat­ments,” they wrote.

Oth­er, more out­side-the-box treat­ments are in ear­ly stages at oth­er com­pa­nies. That in­cludes Is­raeli biotech Bio­mX’s ef­fort to con­coct phage cock­tails to re­move IBS-caus­ing bac­te­ria from the gut, Whole Bio­me’s pro­bi­ot­ic-style ap­proach, and cannabi­noid ther­a­pies from CB2 Ther­a­peu­tics.

Arde­lyx is still look­ing for a US part­ner to li­cense the drug.

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Fabrice Chouraqui, Cellarity CEO-partner (LinkedIn)

Drug de­vel­op­er, Big Phar­ma com­mer­cial ex­ec, now an up­start biotech chief — Fab­rice Chouraqui is ready to try some­thing new as a ‘CEO-part­ner’ at Flag­ship

Fabrice Chouraqui’s career has taken some big twists along his life journey. He got his PharmD at Université Paris Descartes and jumped into the drug development game for a bit. Then he took a sharp turn and went back to school to get his MBA at Insead before returning to pharma on the commercial side.

Twenty years later, after steadily rising through the ranks and journeying the globe to nab a top job as president of US pharma for the Basel-based Novartis, Chouraqui exited in another career switch. And now he’s headed into a hybrid position as a CEO-partner at Flagship, where he’ll take a shot at leading Cellarity — one of the VC’s latest paradigm-changing companies of the groundbreaking model that aspires to deliver a new platform to the world of drug R&D.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.

Once a gem, now just a rock, Take­da punts PhI­II IBD drug as ri­vals mus­cle ahead

Back in 2016, when then-Shire CEO Flemming Ørnskov picked up a promising clinical-stage IBD drug from Pfizer, the Boston-based biotech dubbed it SHP647 and moved it into the gem section of the pipeline, with rosy expectations of registration-worthy Phase III data ahead.

This was a drug that the EC wanted Takeda to commit to selling off before it gave their blessing to its acquisition of Shire, to settle some deep-seated concerns revolving around the potential market overlap with their blockbuster rival Entyvio. And Takeda, which took on a heavy debt load to buy Shire, clearly wanted the cash to pay down debt.