As­traZeneca punts NASH drug from an ail­ing Reg­u­lus as their lead pro­gram goes un­der for the last time

Jay Ha­gan

The drum­beat of bad news at Reg­u­lus is con­tin­u­ing with a rapid tem­po to­day.

Not sur­pris­ing­ly, Reg­u­lus has opt­ed to kill off its lead RNAi pro­gram for HCV af­ter it lin­gered for months un­der a full FDA hold on safe­ty is­sues. But its painful up­date to­day al­so dis­clos­es that As­traZeneca has punt­ed back rights to a NASH drug — AZD4076 (RG-125) — two years af­ter the phar­ma gi­ant stepped in with a deal for it. And an­oth­er pro­gram for RGLS5040 has been dis­con­tin­ued as well.

Shares of Reg­u­lus $RGLS were down 16% Mon­day af­ter­noon, drop­ping clos­er to the $1 mar­ket that de­fines the bor­der to pen­ny stock ter­ri­to­ry.

Reg­u­lus says it prob­a­bly fig­ured out what trig­gered jaun­dice — the ac­cu­mu­la­tion of too much biliru­bin in tis­sue — among pa­tients tak­ing its an­ti-miR122 drug RG-101. Re­searchers pinned it on the “in­hi­bi­tion of con­ju­gat­ed biliru­bin trans­port by RG-101, im­paired base­line biliru­bin trans­port in HCV pa­tients and the pref­er­en­tial up­take of RG-101 by he­pa­to­cytes con­tributed to this mech­a­nism.”

Just two months ago the com­pa­ny trig­gered a re­struc­tur­ing and the ex­it of its sec­ond CEO af­ter the clin­i­cal hold con­tin­ued long af­ter the biotech promised it could re­solve the is­sue with reg­u­la­tors. Now Reg­u­lus says it has bet­ter pre­clin­i­cal HCV drugs in the pipeline, but wants to do a mar­ket re­view be­fore it ad­vances any of them in­to the clin­ic.

What any ca­su­al ob­serv­er of the HCV mar­ket could tell you, though, is that there are al­ready sev­er­al ef­fec­tive cures for he­pati­tis C, which is see­ing a swift fall in sales rev­enue. New drugs that promise a faster cure are in the late-stage pipeline, but they are find­ing that in­vestors have be­gun to steer clear of the dis­as­ter zone.

As­traZeneca had ini­tial­ly bagged rights for the NASH drug in ear­ly 2015 for on­ly $2.5 mil­lion un­der a $523 mil­lion pact they had signed ear­li­er. RGLS5040 was deep sixed “based on a po­si­tion­ing of the com­pound with re­spect to the com­pet­i­tive land­scape cou­pled with the re­sults from re­peat phar­ma­col­o­gy stud­ies as part of IND-en­abling work.”

Reg­u­lus has been cir­cling the wag­ons around RG-012 for Al­port syn­drome, its new lead pro­gram.

“We are square­ly fo­cused on tak­ing the steps nec­es­sary to ad­vance our pipeline and con­tin­ue build­ing share­hold­er val­ue. To that end, we rec­og­nize that we must be dis­ci­plined in our in­vest­ment choic­es and fo­cus our re­sources and cap­i­tal on our most promis­ing dis­cov­ery and de­vel­op­ment pro­grams, in­clud­ing the ap­pli­ca­tion of im­por­tant de­vel­op­ment, reg­u­la­to­ry and com­mer­cial con­sid­er­a­tions,” said CEO Jay Ha­gan in a pre­pared state­ment. Ha­gen was pro­mot­ed to CEO from the COO job in April.

Paul Hudson, Getty Images

Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Amarin CEO John Thero discussing the company's plans for Vascepa, August 2019 — via Bloomberg

Amarin wins a block­buster ap­proval from the FDA. Now every­one can shift fo­cus to the patent

For all those people who could never quite believe that Amarin $AMRN would get an expanded label with blockbuster implications, the stress and anxiety on display right up to the last minute on Twitter can now end. But new, pressing questions will immediately surface now that the OK has come through.

On Friday afternoon, the FDA stamped its landmark approval on the industrial strength fish oil for reducing cardio risks for a large and well defined population of patients. The approval doesn’t give Amarin everything it wants in expanding its use, losing out on the primary prevention group, but it goes a long way to doing what the company needed to make a major splash. The approval was cited for patients with “elevated triglyceride levels (a type of fat in the blood) of 150 milligrams per deciliter or higher. Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease.”

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Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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Paul Biondi (File photo)

Bris­tol-My­er­s' strat­e­gy, BD chief Paul Bion­di ex­it­ed the com­pa­ny — just ahead of the $74B Cel­gene deal close

Paul Biondi, who orchestrated billions of dollars in deals for Bristol-Myers Squibb over the 5 years he’s run their business development team, has exited the company. Biondi left last month, according to a company spokesperson, in pursuit of another — unspecified — external opportunity.

After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.

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Arie Belldegrun at UKBIO 2019. Shai Dolev for Endpoints News

Kite Phar­ma's ex-CEO con­tra­dicts founder as CAR-T patent tri­al heats up, with con­flict­ing val­u­a­tions

Two days after Kite Pharma founder Arie Belldegrun told a federal courtroom that a meeting he had with a Memorial Sloan Kettering executive wasn’t about licensing their immunotherapy patent, Kite’s ex-CEO Aya Jakobovits said it was.

The admission came Tuesday during cross-examination in a patent infringement case that features two of the biggest cancer biotechs and some of the most well-known names in American medicine.

Jakobovits initially said she was not in attendance, didn’t know it was going to happen and didn’t know what took place, according to Law360. But then the plaintiff’s lawyer handed her a document – whose contents were not publicly revealed – and asked again if she learned after-the-fact that the meeting involved a potential patent license.

“Yes,” Jakobovits eventually said.

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On the heels of promis­ing MCL da­ta, Kite hus­tles its 2nd CAR-T to the FDA as the next big race in the field draws to the fin­ish line

Three days after Gilead’s Kite subsidiary showed off stellar data on their number 2 CAR-T KTE-X19 at ASH, the executive team has pivoted straight to the FDA with a BLA filing and a shot at a near-term approval.

In a small, 74-patient Phase II trial reported out at the beginning of the week, investigators tracked a 93% response rate with two out of three mantle cell lymphoma patients experiencing a complete response.

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What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

Ju­ry finds Gilead li­able for $585M and big roy­al­ties in Kite CAR-T patent case

A Kite deal that’s already become a burden on Gilead’s back just got heavier as a California jury has ruled Gilead must pay Bristol-Myers Squibb and Sloan Kettering $585 million plus a 27.6% royalty for patent infringement committed by its subsidiary. The ruling is almost certain to be appealed.

Kite Pharma — founded by Arie Belldegrun, now focused on a next-gen CAR-T company — has been facing a lawsuit since the day its first CAR–T therapy won approval in October, 2017. Juno Therapeutics and Sloan Kettering filed a complaint saying Kite had copied its technology. Gilead acquired Kite in June of that year for $11.9 billion.  Juno was acquired the following year by Celgene for $9 billion, before Celgene was acquired by Bristol-Myers Squibb in 2019.

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