Pfiz­er vet Ger­mano makes quick ex­it from In­trex­on as hands-on bil­lion­aire Kirk re­or­ga­nizes biotech

When Geno Ger­mano left a se­nior post at Pfiz­er and lat­er signed on as pres­i­dent of In­trex­on 10 months ago, he start­ed down a path that he was told led straight to the CEO’s job. In­stead, he wound up at the ex­it door on Fri­day, leav­ing bil­lion­aire Ran­dal “RJ” Kirk at the helm of the com­pa­ny he found­ed, re­or­ga­niz­ing the com­plex biotech side of the busi­ness.

Ger­mano had been head of the glob­al phar­ma busi­ness at Pfiz­er when he was sud­den­ly odd man out in a planned merg­er with Al­ler­gan, which al­so ul­ti­mate­ly nev­er hap­pened. Kirk wel­comed him with open arms, telling the world that Ger­mano would be the next chief for day-to-day op­er­a­tions $XON as he stepped up to the ex­ec­u­tive chair­man’s role.

That put the ex-Pfiz­er ex­ec right near the top of the pyra­mid of a wide net­work of sub­sidiaries all claim­ing to be work­ing on game-chang­ing syn­thet­ic bi­ol­o­gy tech in 5 big sec­tors: health, food, en­er­gy, en­vi­ron­ment and con­sumer mar­kets.

Now Ger­mano is go­ing back to the bio­phar­ma world, and “RJ” is stay­ing at the helm of In­trex­on, which en­joys a $2.8 bil­lion mar­ket cap. From Ger­mano’s state­ment:

RJ’s vi­sion can be seen in the goals he sets and the tremen­dous re­sources – fis­cal, tech­no­log­i­cal and hu­man – as­sem­bled to achieve them.  It is with the fore­go­ing in mind that it has be­come clear to me that RJ is in­te­gral to the day to day op­er­a­tion of this com­pa­ny and that it is there­fore ap­pro­pri­ate for him to re­main in the CEO role for the fore­see­able fu­ture.  At the same time, I have come to re­al­ize that my pref­er­ence is to work with­in the in­dus­try where I spent most of my life, and there­fore am leav­ing the com­pa­ny to con­tin­ue my ca­reer in the phar­ma/bio­phar­ma in­dus­try.

Kirk an­nounced Ger­man’s de­par­ture at the same time he set up yet an­oth­er sub­sidiary, this one dubbed Pre­ci­gen, that he said would make it eas­i­er to pur­sue “po­ten­tial strate­gic op­tions to en­hance share­hold­er val­ue.”

Kirk didn’t ex­act­ly ex­plain what those strate­gic op­tions are, but he made it clear that he want­ed all the part­nered pipeline col­lab­o­ra­tions — which are con­sid­er­able — un­der one arm of the com­pa­ny.

Their pipeline in­cludes ther­a­pies for a wide va­ri­ety of dis­eases, such as ad­vanced lym­phoid ma­lig­nan­cies, acute myeloid leukemia, pe­di­atric brain tu­mors, sol­id tu­mors, oral mu­cosi­tis, type 1 di­a­betes, wet age-re­lat­ed mac­u­lar de­gen­er­a­tion, Clostrid­i­um dif­fi­cile in­fec­tion, lin­ear scle­ro­der­ma, and car­diac dis­ease. Pre­ci­gen will al­so man­age a 75% stake in Xo­genex, which is pur­su­ing ge­net­ic strate­gies on car­diac dis­ease. It’s all pre­clin­i­cal right now, with an IND com­ing be­fore the end of the year.

Said Kirk:

While we have been re­view­ing po­ten­tial op­tions for over a year, as our col­lab­o­ra­tors in­creas­ing­ly be­gin to move in­to the clin­ic, it is ap­par­ent that our col­lec­tion of health as­sets may be over­shad­owed by the breadth and com­plex­i­ty of the op­por­tu­ni­ties the en­gi­neer­ing of bi­ol­o­gy has af­ford­ed us.  We are there­fore tak­ing this struc­tur­al ac­tion now to bet­ter po­si­tion us for strate­gic de­ci­sions re­gard­ing our health busi­ness mov­ing for­ward.

The com­pa­ny has inked a bliz­zard of deals over the years, but it’s al­so been bleed­ing cash. Ac­cord­ing to its 10K filed at the be­gin­ning of this month, In­trex­on was $187 mil­lion in the red in 2016 af­ter more than dou­bling down on its loss­es from 2015.

In­trex­on has man­aged to main­tain a large val­u­a­tion with­out demon­strat­ing much in the way of clin­i­cal im­pact, so far. That can’t last for­ev­er, but it’s held up bet­ter than most over the last few years.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

Endpoints News

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.