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.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a plan to near­ly dou­ble its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.