Phase­Bio touts 'break­through' badge for blood­thin­ner re­ver­sal agent li­censed from As­traZeneca, shares leap

At Phase­Bio, the game plan has been to hus­tle its Bril­in­ta re­ver­sal agent, PB2452, down what it sees as a short path to ap­proval, un­lock­ing rev­enue that will then fund more ex­pen­sive tri­als of its pul­monary ar­te­r­i­al hy­per­ten­sion drug. With a new break­through ther­a­py des­ig­na­tion, ex­ecs are march­ing on­ward with boost­ed con­fi­dence.

The “break­through” badge was be­stowed based on Phase I re­sults in which PB2452 achieved “im­me­di­ate and sus­tained re­ver­sal” of Bril­in­ta’s an­tiplatelet ef­fects. The biotech’s shares $PHAS surged 30%, reach­ing $16.4 pre-mar­ket.

Jonathan Mow

Phase­Bio first li­censed PB2452 in 2017 from As­traZeneca for $100,000 up­front, the mak­er of the blood thin­ner al­so known as tica­grelor. It’s de­signed for acute sit­u­a­tions where pa­tients are ex­pe­ri­enc­ing ac­tive bleed­ing or re­quire ur­gent surgery — akin to what Por­to­la did with An­dexxa, an an­ti-an­ti­co­ag­u­lant that re­vers­es ri­varox­a­ban and apix­a­ban.

That’s a de­par­ture from Phase­Bio’s pre­vi­ous strat­e­gy, CEO Jonathan Mow told me ahead of its Se­ries D an­nounce­ment last year, which re­volved around its tech. The elastin-like polypep­tides plat­form cre­ates ther­a­peu­tic fu­sion pro­teins that un­der­go a ful­ly re­versible phase tran­si­tion, there­by sig­nif­i­cant­ly ex­tend­ing their half lives.

In fact, Phase­Bio said on the heels of the “break­through” news, it has li­censed a long-act­ing, re­com­bi­nant GLP-1 ana­logue dubbed PB1023 to Im­muno­Forge, which is es­pe­cial­ly in­ter­est­ed in its use for sar­cope­nia. The deal doesn’t cov­er di­a­betes, obe­si­ty and NASH.

The plat­form tech is still be­ing used in the de­vel­op­ment of PB1046, an in-house as­set for PAH that’s de­signed for chron­ic use. But for now spot­light is on PB2452, which Mow con­sid­ers an or­phan first-in-class prod­uct in the car­diopul­monary space and which he pre­dicts will en­hance the safe­ty pro­file of Bril­in­ta, re­in­forc­ing its use and grad­u­al­ly ex­pand­ing Phase­Bio’s own mar­ket.

“PB2452, can be de­vel­oped in a much short­er time pe­ri­od, and it will pay for us to de­vel­op PB1046,” he said ear­li­er. “So even though it’s on­ly in Phase I, we will get to the lat­er stages of de­vel­op­ment much more quick­ly than we will with PB1046.”

He now has the FDA on his side to ac­cel­er­ate and fa­cil­i­tate the clin­i­cal progress.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.