Shares soar as ‘break­through’ news in biotech con­tin­ues to run hot this week

The FDA has been work­ing over­time this week hand­ing out new break­through ther­a­py des­ig­na­tions for a whole slate of biotech drugs in the clin­ic. But five years af­ter the pro­gram was launched, in­vestors are still ready to cheer on each an­nounce­ment with a big spike in stock prices.

Waltham, MA-based Pro­teon Ther­a­peu­tics nabbed a BTD for von­a­pan­i­tase for in­creas­ing ar­te­ri­ove­nous fis­tu­la sec­ondary pa­ten­cy (sur­vival of the fis­tu­la with­out aban­don­ment) and use for he­modial­y­sis in pa­tients on or ex­pect­ed to ini­ti­ate he­modial­y­sis. Its stock $PRTO surged 23% on the news Wednes­day.

This morn­ing it was Syn­thet­ic Bi­o­log­ics’ turn. The FDA hand­ed it a BTD for SYN-004 (rib­axa­m­ase), which is de­signed to pro­tect the gut mi­cro­bio­me from the hav­oc caused by some an­tibi­otics and guard against Clostrid­i­um dif­fi­cile in­fec­tion. The Rockville, MD-based biotech said that af­ter the Phase IIb tri­al came up with pos­i­tive da­ta, reg­u­la­tors were ready to give it a BTD and an open door to reg­u­la­tors who can help speed this to an ap­proval. Their stock $SYN jumped 40% in pre-mar­ket trad­ing, but it’s a pen­ny stock af­ter the com­pa­ny is­sued a huge num­ber of shares last fall, so a big spike is still just worth pen­nies a share.

Boston-based Rhythm land­ed a sec­ond BTD for set­melan­otide, the com­pa­ny’s melanocortin-4 re­cep­tor (MC4R) ag­o­nist. The ex­pand­ed BTD is for the treat­ment of obe­si­ty as­so­ci­at­ed with ge­net­ic de­fects up­stream of the MC4 re­cep­tor in the lep­tin-melanocortin path­way. The FDA had pre­vi­ous­ly grant­ed BTD to set­melan­otide for the treat­ment of POMC de­fi­cien­cy obe­si­ty. Rhythm is a pri­vate com­pa­ny.

Cor­bus Phar­ma­ceu­ti­cals $CRBP ear­li­er this week, mean­while, said that it did not get a BTD for its an­ti-in­flam­ma­to­ry an­aba­sum, as they had re­quest­ed, mak­ing it one of the few biotechs that have gone af­ter the des­ig­na­tion and pub­licly ad­mit­ted when it didn’t come through. But plen­ty of oth­ers are land­ing new or im­proved BTDs, which could help re­duce the time it takes to get a new drug through the clin­ic and on to a po­ten­tial ap­proval.

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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