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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

Amidst R&D reshuf­fle, Ver­tex ex­pands its pres­ence in Boston, aim­ing to be­come num­ber one

Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint.

At a ribbon cutting on Tuesday for its new Jeffrey Leiden Center for Cell and Genetic Therapies at the Boston Seaport, Vertex announced it would embark on a new project: The company will build a 344,000 square foot facility in the seaport to accommodate the company’s growing R&D needs, especially in its cell and gene therapies program.

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FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.

Peter Marks (Greg Nash/Pool via AP)

Even FDA's Pe­ter Marks is wor­ried about the com­mer­cial vi­a­bil­i­ty of gene and cell ther­a­pies

When bluebird bio’s gene therapy to treat beta thalassemia won European approval in 2019, the nearly $2 million per patient price tag for the potential cure seemed like a surmountable hurdle.

Fast forward two years later, and bluebird has withdrawn Zynteglo, the beta thal drug, along with the rest of its gene therapy portfolio from Europe, which the company said is generally unwilling to pay a fair price for the treatment.