Pow­er­ful Dems or­ches­trat­ed cam­paign at FDA to pro­vide Bio­gen drug to a dy­ing, high-pro­file fundrais­er

Back in 2008, Dal­las tri­al lawyer and high-pro­file De­mo­c­ra­t­ic Par­ty fundrais­er Fred Baron was dy­ing of mul­ti­ple myelo­ma. Des­per­ate to gain ac­cess to Bio­gen’s Tysabri, an MS drug then be­ing test­ed for myelo­ma in ear­ly-stage stud­ies, his wife Lisa Blue Baron reached out to the com­pa­ny but ran di­rect­ly in­to a brick wall of re­sis­tance.

“[Bio­gen] won’t ap­prove for fred be­cause he is too sick and if it fails him, it could skew the out­come of the tri­als(this told to me by his wife),” not­ed a fam­i­ly friend in the er­ror-rid­den post in­clud­ed in the batch of hacked emails from John Podes­ta — the chair­man of Hillary Clin­ton’s pres­i­den­tial cam­paign — dumped by Wik­iLeaks on Sat­ur­day.

In­censed, Baron’s wife reached out to in­flu­en­tial friends in the De­mo­c­ra­t­ic Par­ty.

If I am un­able to get drug I will take out full pg add in boston globe to ceo and board mem­bers.vFred is run­ning out of time. He’s so sick and needs this drug. Thanks so much.

In short or­der, a whole line­up of high-pro­file of­fi­cials rang­ing from for­mer Pres­i­dent Bill Clin­ton to Sen­a­tor John Ker­ry and then FDA Com­mis­sion­er An­drew von Es­chen­bach were di­rect­ly lob­by­ing for­mer Bio­gen CEO Jim Mullen to pro­vide the drug for com­pas­sion­ate use.

One seg­ment of the Podes­ta email ex­change in­cludes:

I got through to Mullen.  He had al­ready had ex­ten­sive con­ver­sa­tions with Sen­a­tor Ker­ry and Lance Arm­strong and had heard from Pres­i­dent Clin­ton, too.  The an­swer he gave in every case was [no].  I be­lieve that I un­der­stood it cor­rect­ly that Ker­ry had or­ga­nized a con­fer­ence call with Mullen and FDA Com­mis­sion­er Von Es­chen­bach.  I hear that Von Es­chen­bach said it was fine with him for the drug to be used in Fred’s case.  How­ev­er, Bio­gen be­lieves that Von E does not have that au­thor­i­ty giv­en the strict FDA con­di­tions on the use of the drug.

And in the next sec­tion:

I just had a dis­ap­point­ing call with Dr. Von Es­chen­bach. FDA had a phone con­fer­ence with the med­ical and sci­ence of­fi­cers from Bio­gen. FDA told them there would be no prej­u­dice to the clin­i­cal tri­al if this drug were used for Fred. They were ap­pre­cia­tive but gave no in­di­ca­tion they would change their po­si­tion, which is that a cor­po­rate de­ci­sion has been made they would not go out­side the clin­i­cal tri­al. I am re­al­ly up­set about this, per­son­al­ly, since FDA has been great to help. I have not had the time to­day to reach out to some on the board of Bio­gen, but hope to do so yet to­day. I felt last night, af­ter talk­ing with Andy von Es­h­en­bach, that with clear­ance from FDA, the com­pa­ny would be ok.

Look­ing back over Luke Tim­mer­man’s cov­er­age at the time for Xcon­o­my, it’s in­ter­est­ing to note that Baron got the drug be­cause the FDA and the Mayo Clin­ic pro­vid­ed it to him af­ter the agency found a le­gal ba­sis to do so in an emer­gency sit­u­a­tion.

Bio­gen, though, had con­tin­ued to in­sist that any use of Tysabri for myelo­ma in Baron’s case could cre­ate a sit­u­a­tion that would cause reg­u­la­tors to re­strict its use. This was two years af­ter the FDA had al­lowed Bio­gen and Elan to put the drug back on the mar­ket af­ter it was pulled fol­low­ing the death of sev­er­al pa­tients due to PML.

The de­bate over ac­cess to drugs un­der com­pas­sion­ate use poli­cies has helped spur a whole se­ries of “Right to Try” laws around the coun­try. For many pa­tients, it can be a frus­trat­ing ex­er­cise, try­ing to con­vince a bio­phar­ma com­pa­ny to pro­vide a drug to them in their last dy­ing days. For many biotechs, though, com­pas­sion­ate use re­mains a vex­ing is­sue as well. These pro­grams can be ex­pen­sive and al­so rais­es the threat that an un­ex­pect­ed ad­verse event can force a com­pa­ny to sus­pend work on a drug.

Fred Baron got the drug, but there was no hap­py end­ing. The be­hind-the-scenes strug­gle end­ed with his death on Oc­to­ber 30, 2008, soon af­ter the con­tro­ver­sy erupt­ed.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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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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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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