Se­cre­tive? Mod­er­na de­tails its pipeline strat­e­gy at JPM but at least one crit­ic bites back

Stephane Ban­cel, Mod­er­na CEO

Mod­er­na out­lined its pipeline strat­e­gy at JP Mor­gan this week, fo­cus­ing specif­i­cal­ly on five clin­i­cal-stage pro­grams for new vac­cines as a rel­a­tive­ly low-risk ap­proach to demon­strat­ing that their mes­sen­ger RNA tech­nol­o­gy and the plat­form work. But the kick­back didn’t take long to strike, as a per­sis­tent crit­ic at Stat picked at one trou­bled pro­gram while rais­ing ques­tions about the over­all val­ue of an en­try-lev­el ap­proach fo­cused on a high vol­ume, low-mar­gin busi­ness like vac­cines.

Af­ter rais­ing $1.9 bil­lion from a host of deep-pock­et in­vestors and part­ners, the uni­corn biotech has be­come the sub­ject of in­tense in­ter­est, par­tic­u­lar­ly in Boston biotech cir­cles. And Mod­er­na’s re­luc­tance to de­tail its work in the past has left the biotech with a rep among some re­porters as a se­cre­tive, pun­ish­ing place that has seen off a string of staffers who couldn’t main­tain the fren­zied pace.

Mod­er­na CEO Stephane Ban­cel, though, says that at­tack is un­war­rant­ed. His pre­sen­ta­tion at JP Mor­gan is in­tend­ed to shine a light on what the com­pa­ny’s top prospects are as it preps for its first clin­i­cal da­ta.

Ban­cel’s strat­e­gy is sim­ple: In­stead of fo­cus­ing on a sin­gle lead ef­fort, the pri­vate biotech chose to ramp up a full pipeline of clin­i­cal pro­grams. If one fal­ters, oth­ers can step up to make the case that mR­NA is on its way to de­liv­er­ing a new ap­proach to ther­a­peu­tic de­vel­op­ment with a vast ar­ray of po­ten­tial tar­gets.

“Peo­ple are go­ing to re­al­ize one day that mR­NA is a very good way to make med­i­cines,” Ban­cel says. “We are play­ing a very long game, a 10, 20-year game. We are a very in­tense com­pa­ny. We want to get those drugs for pa­tients.” And that means tack­ling dis­eases like Zi­ka with their tech­nol­o­gy.

Mod­er­na went af­ter two pan­dem­ic flu strains ear­ly, says Ban­cel, so in­ves­ti­ga­tors could ac­cu­rate­ly mea­sure an­ti­body re­spons­es among treat­ment naive pa­tients who had nev­er been ex­posed to the virus. In ad­di­tion to Zi­ka there’s an undis­closed pro­gram part­nered with Mer­ck that’s now in the clin­ic and sev­en pre­clin­i­cal pro­grams, in­clud­ing an­oth­er Mer­ck part­ner­ship on a per­son­al­ized can­cer vac­cine.

One of the com­pa­ny’s most promi­nent crit­ics, Stat’s Dami­an Garde, re­port­ed to­day that safe­ty is­sues hob­bled a ther­a­py for Crigler-Na­j­jar syn­drome — part­nered with Alex­ion — which Ban­cel promised a year ago would get in­to the clin­ic in 2016. That safe­ty is­sue, Garde added, raised fresh ques­tions about Mod­er­na’s high-stakes gam­ble on mR­NA, un­der­min­ing its $5 bil­lion val­u­a­tion it has en­joyed while rais­ing a moun­tain of cash.

Ban­cel tells me that a long-an­tic­i­pat­ed move to cash in on their val­u­a­tion through an IPO, though, won’t be hap­pen­ing this year.

“We will not go pub­lic this year,” he tells me. “This has been dis­cussed by the board. 2017 is not the right time. 2016 and ’17 is a crit­i­cal time for the com­pa­ny to move from dis­cov­ery to a de­vel­op­ment com­pa­ny with scale.”

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 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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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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