Stuck on FDA hold, Reg­u­lus CEO hits the ex­it as re­or­ga­ni­za­tion slash­es staff at the be­lea­guered biotech

With their lead drug stuck on a full clin­i­cal hold and no hope of re­lief be­fore the end of this year — a year af­ter it had ini­tial­ly fore­cast the end of its spell in pur­ga­to­ry — Reg­u­lus Ther­a­peu­tics $RGLS is bid­ding good­bye to its CEO (again) and slash­ing staff.

Jay Ha­gan

Af­ter the mar­ket closed on Thurs­day the be­lea­guered com­pa­ny an­nounced plans to cut its head­count by 30%, or ap­prox­i­mate­ly 30 staffers af­ter it list­ed 97 em­ploy­ees at the end of 2016. CEO Paul Grint, who stepped up from the CMO’s job af­ter found­ing CEO Klean­this Xan­thopou­los re­signed in 2015, is leav­ing the com­pa­ny, with COO Jay Ha­gan re­plac­ing him at the helm.

Reg­u­lus was cre­at­ed in 2007 as a joint ven­ture be­tween Al­ny­lam and Io­n­is, then called Isis, with a spe­cial fo­cus on mi­croR­NA ther­a­pies.

The new CEO’s first task was to re­as­sure in­vestors that the com­pa­ny is squared away and fo­cused on hit­ting its marks with a sec­ond ther­a­py, RG-012. But the mes­sage didn’t sit well with its in­tend­ed au­di­ence.

The biotech’s stock cratered again, drop­ping 30% on the news of the lat­est set­back and chang­ing of the guard. It’s now trad­ing at a lit­tle more than a dol­lar a share, just a frac­tion of what it was two years ago.

Lead drug RG-101 went on full clin­i­cal hold last sum­mer af­ter a sec­ond pa­tient came down with jaun­dice. Grint in­sist­ed that the team could get all of the agency’s ques­tions an­swered by the 4th quar­ter of 2016, then in Jan­u­ary said that the FDA want­ed more safe­ty and ef­fi­ca­cy da­ta that wouldn’t be avail­able un­til the 4th quar­ter of this year.

Things looked con­sid­er­ably bet­ter a year ago when RG-101 ap­peared to look good in a small Phase II study for hep C, with high cure rates. Now the en­tire mar­ket is ex­pe­ri­enc­ing a swoon as lead­ers like Gilead cure pa­tients at a faster pace than they can be di­ag­nosed. RG-101 had po­ten­tial to cure pa­tients in just a few weeks, but that added val­ue is shrink­ing steadi­ly in sig­nif­i­cance to pay­ers.

Reg­u­lus had $57.5 mil­lion at the end of the first quar­ter, with plans to cir­cle its wag­ons around a Phase II study for RG-012 for Al­port syn­drome, a ge­net­ic con­di­tion that trig­gers the grad­ual loss of kid­ney func­tion. Net loss for the first quar­ter was $20 mil­lion.

“We are very grate­ful for the lead­er­ship of Dr. Grint and the many con­tri­bu­tions of our oth­er im­pact­ed em­ploy­ees who have ded­i­cat­ed them­selves to Reg­u­lus’ ef­forts in ad­vanc­ing the sci­ence of mi­croR­NAs,” said Ste­lios Pa­padopou­los, the com­pa­ny’s board chair­man.  “We are con­fi­dent that Reg­u­lus is well po­si­tioned for suc­cess un­der Jay’s guid­ance.”

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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Federal Trade Commission commissioner Rohit Chopra testifies on Capitol Hill (AP Photo/Susan Walsh)

FTC clears Bris­tol-My­ers’ $74B deal to buy Cel­gene — but Dems sig­nal a po­ten­tial hard shift against Big Phar­ma M&A

Bristol-Myers Squibb’s record $74 billion takeover of Celgene is a done deal. And it will all be over — except for the lingering complaints from die-hard Celgene investors — on Wednesday.

Like much else that’s going on in Washington these days, the vote among the 5 FTC commissioners split along party lines, with the 3 Republicans voting to clear the way and the 2 Democrats steamed over what they see as a major M&A move that will lessen competition and innovation. And that split has big implications for the M&A side of the business if the Dems take the White House in 2020.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: In a first, FDA green-lights use of a Chi­nese built can­cer ther­a­py — and more are com­ing

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

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What does $62B buy you these days? A lot, says Take­da ex­ecs as the phar­ma play­er promis­es a block­buster R&D fu­ture

First comes the $62 billion buyout. Then comes the asset auction and reorganization to pay down debt. Now comes the detailed pledge of a bigger, brighter future in drug development.

That’s where Takeda finds itself on R&D day today, about 11 months after closing on their Shire acquisition. R&D chief Andy Plump is joining CEO Christophe Weber and other top members of the team to outline a new set of priorities in the greatly expanded pipeline at Takeda, which has jumped into the top ranks of the world’s pharma giants in the wake of the Shire deal.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.

Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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