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.”

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

UP­DAT­ED: Bris­tol My­ers Squibb com­mits $300 mil­lion to com­bat racial dis­par­i­ties, but de­clines to re­lease own de­mo­graph­ic da­ta

After the police killing of George Floyd, a flurry of pharma and biotech companies, executives and investors jumped out to make statements, either expressing support for Black Lives Matter and the protests or condemning systemic racism.

Now, a Big Pharma company is publicly putting some teeth behind those statements. This morning, Bristol Myers Squibb announced they would spend $300 million on a broad effort to reduce racial health disparities, and diversify both their clinical trials and their own executive team and workforce.

Martin Shkreli (AP Images)

Mar­tin Shkre­li's in­fa­mous Dara­prim falls off top 20 most ex­pen­sive drugs list

Martin Shkreli incited a national uproar five years ago when he raised the price of Daraprim by a factor of 56 essentially overnight from $13.50 to $750 per pill. Now that the “Pharma Bro’s” high-priced project has received a generic, it no longer places among the most expensive drugs in the world.

GoodRx is back with the latest update of the top 20 most expensive drugs and Daraprim’s exclusion marks the biggest change. The drug had previously ranked seventh on the list’s last iteration, which came in February before the world went into quarantine. Another of Shkreli’s former companies, Retrophin, saw its Chenodal drug place in the top 10 again.

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