Bol­stered by promis­ing PhI­Ib snap­shot, uniQure plays catch-up with Spark in the race to a he­mo­phil­ia B gene ther­a­py

The piv­otal lead switch that uniQure ex­e­cut­ed late last year ap­pears to have paid off, as the gene ther­a­py pi­o­neer of­fers some pos­i­tive, if ear­ly, num­bers for its tweaked he­mo­phil­ia B treat­ment.

Robert Gut

Among three pa­tients treat­ed with AMT-061 in the dose-con­fir­ma­tion study, re­searchers re­port­ed a mean fac­tor IX lev­el that rep­re­sent­ed 31% of nor­mal lev­els at six weeks, ex­ceed­ing the 12% com­mon­ly re­gard­ed as suf­fi­cient to sub­stan­tial­ly re­duce spon­ta­neous bleeds. A quick break­down by in­di­vid­ual showed FIX ac­tiv­i­ty in the first pa­tient was 37% of nor­mal 10 weeks af­ter ad­min­is­tra­tion; in the sec­ond pa­tient, 23% at week 8; and the third, 30% at week 6.

Hap­py to hear that none of the pa­tients re­ceived fac­tor in­fu­sions, ex­pe­ri­enced re­port­ed bleed­ing events or re­quired im­muno­sup­pres­sion, in­vestors sent the stock up $QURE 39%.

Ed­ward Nash

It’s al­so mu­sic to the ears of some sup­port­ive an­a­lysts, who are call­ing the re­sults “very good” and “a clear pos­i­tive” and large­ly agree­ing with ex­ecs that a “mild, asymp­to­matic and tran­sient in­crease in liv­er en­zyme lev­els” ob­served in the tri­al was not a cause for con­cern.

The re­sults af­firm uniQure’s com­pet­i­tive po­si­tion with Spark Ther­a­peu­tics, which is al­so de­vel­op­ing a he­mo­phil­ia B ther­a­py to fol­low up its his­toric reti­na eye dis­ease treat­ment.

Ed­ward Nash of Sun­Trust notes:

As a ref­er­ence, SPK-9001 from Spark showed in a Phase I/II study that the range of FIX ac­tiv­i­ty lev­els was be­tween 14% and 68% at Week 6 and be­tween 16% and 78% at Week 8.

Joseph Schwartz

An­oth­er de­tail that may sep­a­rate uniQure from Spark, Leerink’s Joseph Schwartz points out, is that AMT-061 may be able to tar­get a broad­er spec­trum of pa­tients. Two of the three pa­tients in uniQure’s Phase IIb tri­al were en­rolled af­ter pre­vi­ous­ly screen-fail­ing an­oth­er gene ther­a­py study due to pre-ex­ist­ing neu­tral­iz­ing an­ti­bod­ies to a dif­fer­ent AAV vec­tor. In fact, ex­ecs were so con­fi­dent about the AAV5 vec­tor they have vowed not to ex­clude any pa­tients on the pa­tients of pre-ex­ist­ing neu­tral­iz­ing an­ti­bod­ies.

Am­s­ter­dam- and Lex­ing­ton, MA-based uniQure re­placed its lead pro­gram, AMT-060, with AMT-061 last Oc­to­ber, claim­ing that a new gene vari­ant called FIX-Pad­ua in­tro­duced in the tweaked as­set will boost FIX ac­tiv­i­ty while keep­ing the same de­liv­ery tech and man­u­fac­tur­ing process.

“Based on the long-term FIX da­ta from our Phase I/II tri­al of AMT-060, where we saw FIX ac­tiv­i­ty lev­els con­tin­ue to in­crease be­yond the lev­els achieved at six to ten weeks, we are hope­ful that we will con­tin­ue to see sim­i­lar trends in these pa­tients,” said CMO Robert Gut in a state­ment.

Catch­ing up with Spark and its Big Phar­ma al­lies at Pfiz­er, uniQure plans to be­gin dos­ing for a Phase III study in Q1 2019.

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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CEO Pascal Soriot via Getty Images

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.