Pro­teosta­sis shares zoom north as their cys­tic fi­bro­sis dou­blet caus­es a stir in small study -- trig­ger­ing an on­line brawl

Some­times it’s the small­est stud­ies that gain the biggest re­ac­tions from the in­vest­ment crowd that fol­lows biotech.

Case in point: Lit­tle Pro­teosta­sis Ther­a­peu­tics hit big-time pay dirt on Thurs­day, watch­ing its stock $PTI swoop up 448% on some in­trigu­ing ear­ly da­ta on a hand­ful of pa­tients suf­fer­ing from cys­tic fi­bro­sis.

Meenu Chhabra, CEO

Break­ing 21 pa­tients ho­mozy­gous for the F508del mu­ta­tion in­to var­i­ous dose groups for their CF dou­blet ther­a­py, re­searchers teased out some sig­nif­i­cant re­sults for the few peo­ple in the high dose co­hort that was evalu­able at the time of the Phase I cut­off for their read­out.

For the 300 mg ppFEV1 co­hort there was a sta­tis­ti­cal­ly sig­nif­i­cant in­crease from base­line to day 7 of 6.3%, which reg­is­tered as a 5.9% in­crease on day 14. Com­pared to a place­bo, the same PTI-801 300 mg/PTI-808 300 mg co­hort hit +8.3% and +6.6% at 7 and 14 days. They added that they al­so tracked a sta­tis­ti­cal­ly sig­nif­i­cant de­crease in sweat chlo­ride con­cen­tra­tion from base­line.

There’s an­oth­er 400 mg co­hort that’s be­ing tracked now, with a triplet to fol­low.

The kick­er is here:

“The cur­rent im­prove­ment in ppFEV1 for stan­dard of care dou­blets is on av­er­age 3%-4% in the F508del ho­mozy­gous pa­tient pop­u­la­tion,” said Dami­an Downey, a lec­tur­er in res­pi­ra­to­ry med­i­cine at Queen’s Uni­ver­si­ty Belfast.

The num­bers got a close re­view by an­a­lysts in the field. Leerink’s Joseph Schwartz not­ed Pro­teosta­sis’ mixed re­sults as an add-on for Orkam­bi, from Ver­tex. But, he added, “PTI’s own pro­pri­etary com­bo bodes well in our view for pre­lim­i­nary triplet da­ta ex­pect­ed this quar­ter.”

Baird’s Bri­an Sko­r­ney al­so gave the da­ta a thumbs up as an in­di­ca­tor of the po­ten­tial of the triple:

The com­bo of PTI-808 and PTI-801 showed ini­tial FEV1 re­sults that are com­pa­ra­ble to Ver­tex’s Orkam­bi and Symdeko in cys­tic fi­bro­sis pa­tients. Al­though we be­lieve this com­bo would have a dif­fi­cult de­vel­op­ment path­way, giv­en the avail­abil­i­ty of the Ver­tex dou­blets, we do be­lieve it cre­ates an op­por­tu­ni­ty for it to de­vel­op its am­pli­fi­er, PTI-428, in a pro­pri­etary triple com­bi­na­tion. We will see ini­tial triple com­bo da­ta this quar­ter that we be­lieve will be a sig­nif­i­cant pos­i­tive cat­a­lyst for the stock.

Ver­tex stock dropped 3.5%, which trig­gered a de­fen­sive scrum on Twit­ter as some of the com­pa­ny’s de­fend­ers turned ug­ly on the pres­sure. So far, Ver­tex has stared down re­peat­ed chal­lenges to its fron­trun­ner sta­tus. But it didn’t get off so light­ly on this run.

Pro­teosta­sis’ KOLs, mean­while, will con­tin­ue to talk up their new-found sta­tus in CF.

”Dou­blet com­bi­na­tions, the cur­rent stan­dard of care for many CF pa­tients, are the bench­mark for cur­rent treat­ment in cys­tic fi­bro­sis.  These da­ta are the first re­sults seen us­ing an en­tire­ly new CF dou­blet, com­pare fa­vor­ably to stan­dard of care, and demon­strate the po­ten­tial of next-gen­er­a­tion CFTR mod­u­la­tors to fur­ther im­prove out­comes in this dis­ease,” said Carsten Schwarz, head of the Adult Cys­tic Fi­bro­sis Cen­tre, Lung-Trans­plan­ta­tion Pro­gram, and En­doscopy Unit, De­part­ment of Pe­di­atric Pneu­mol­o­gy and Im­munol­o­gy, Char­ité, Berlin Uni­ver­si­ty Med­ical Cen­ter. “I look for­ward to re­sults from the fourth dos­ing co­hort and to un­der­stand­ing the po­ten­tial of an­oth­er nov­el com­bi­na­tion, PTI-801, PTI-808 and PTI-428.”

Re­searchers will be adding more da­ta this quar­ter and in ear­ly 2019.

The fight­ing has just be­gun.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

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